Welcome!

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Welcome to Four Square Clobbers Cancer!

foursquarecourt2.jpgFour-Square Clobbers Cancer is a conversational blog that is dedicated to improving the outcomes of children, adolescents, and young adults who are being treated or have been treated for cancer.  The goal is to inform, communicate and collaborate with those in the cancer community.

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Four-Square is a very popular game played by children, adolescents and even young adults.   Following the rules and reacting to the constant changing path of a big orange ball, the object is to stay in the game as long as possible.  It’s kind of like the game of life,  where the object is to react favorably to the fast, changing  things that come your way,  stay healthy, live as long as possible and have fun.

Four-Square by definition is also used as an adjective that means firm and resolute, especially in support of someone or something.  “We stand four-square in our conviction to improve the therapies and outcomes of children, adolescents, and young adults with cancer.”bounceball

Clobber is actually a term used in the game of Four-Square and,  coincidently, it means the same thing in the game of life.  It’s what everyone wants to do to cancer!

Complete information about Four Square Clobbers Cancer and it’s bloggers can be found by clicking the “About” tab on the menu bar at the top of the page.

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September is Childhood Cancer Awareness Month. There are a host of activities in Washington, DC centered around childhood cancer.

Click here to see what is happening in your Capitol and be a part of it.

Tell your Senators what you think about #MoreThan4 percent for research.

Recently heard the words,”Your child has cancer?” Please read this:

BestCare_edited-1If you found this site because you have recently heard, “Your child has cancer,” this article is a must read if you are trying to decide, “What next?”  The advice given is invaluable and will help you on your journey down this uncertain road.

Posted in Cancer, Childhood Cancer, Pediatric Cancer, Rare Disease | Tagged , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , | 3 Comments

September, 2015 in Our Capitol

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FreePocketGuide_edited-2September 2015 in our nation’s capitol will be full of activities that will highlight the need for more medical research and an opportunity to show our government that we need to make childhood cancer a national priority.

Below is a schedule of activities that you may find interesting.  As additional information is received, this page will be updated weekly.

A handy Free PocketGuide with maps and locations of all the activities below is now available to print and take with you for all the week’s activities.

Saturday, September 12, 2015, 7:00 p.m. ’till

News for this nationally savedate_edited-1televised major event to be held close to Washington, DC will be updated as soon as available. We have been sworn to total secrecy until all the plans are complete. All we can say at the moment is that it’s a first for the childhood cancer community and it will blow your mind!  Save this date!

Wednesday, September 16, 2015,  3:00 p.m. to 4:30 p.m. and 5:30 to 7:30 p.m.

Rally for Medical Research Hill Day Training Session and Reception

Screen Shot 2015-07-19 at 2.58.42 PMThe Rally for Medical Research is both a call to action for Congress to make medical research a national priority, and a means by which to raise awareness with the general public about the importance of continued investment in medical research through the National Institutes of Health.  The training session will be held at 3:00 p.m. in the Mandarin Oriental Hotel, while the reception will be held in the Kennedy Caucus Room (Russell 325) at 5:30 p.m.. For more information, contact Mary Lee Watts (marylee.watts@aacr.org) or Jon Retzlaff (jon.retzlaff@aacr.org).  Register to attend here!

 Thursday, September 17, 2015,  7:30 a.m. to 4:30 p.m.

Rally for Medical Research Hill Day Breakfast Program and Congressional Meetings.  

facebook-pictureBreakfast will be served at 7:30 a.m. in the Mandarin Oriental Hotel followed by a short meeting. Meetings will be scheduled for participants with their individual Senators and Congressional Representatives. It’s a great opportunity to meet the people from your state and district that are representing you in Washington. This is your opportunity to let them know how valuable research is to you, your family, and our economy.  For more information, you may contact by email Mary Lee Watts (marylee.watts@aacr.org) or send email to Jon Retzlaff (jon.retzlaff@aacr.org). Register here to attend.

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Thursday, September 17, 2015,  5:00 p.m. to 7:00 p.m.

A Golden Toast honoring  members of  The Congressional Childhood Cancer Caucus.

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This is a invitation only event to be held at Stanton and Greene, 319 Pennsylvania Avenue, SW, Washington, D.C.in Washington, DC.

The child friendly event marks the sixth year of the cancer caucus and will  honor the Congressmen and their staffs for the tireless work they have invested over the years. 

Dress is business casual with a lot of emphasis on Golden accessories.  For more information, contact Annette Leslie (carrymecarson@gmail.com)

Thursday, September 17, 2015,   6:30 p.m. to  9:30 p.m.

2015  A Celebration of Life Gala and Dinner Reception by Hyundai Hope on Wheels, 

IMG_2902This is a invitation only event.  Hyundai Motor Company will be celebrating seventeen years involvement and over $100,000,000 in grants and contributions to childhood cancer research they have made during that time. For this wonderful event, Hyundai will bring together some of their top participating dealers, cancer researchers, oncologists, representatives of major medical institutions and the object of everyone’s attention, the children and their families that have been affected by cancer.

Friday, September 18, 2015   9 a.m. to 10:30 a.m.

Childhood Cancer Caucus Summit

U.S. Capitol Building, Washington D.C.The 2015 Congressional Childhood Cancer Caucus is a bipartisan caucus serving as a public policy clearinghouse for information on pediatric cancer and to be a forum for Members of Congress to work together to address this critical issue. The Caucus of over 100 congressional members strives to raise awareness about pediatric cancer, advocate in support of measures to prevent the pain, suffering and long-term effects of childhood cancers, and work toward the goal of eliminating cancer as a threat to all children. Congressmen Michael McCaul and Chris Van Hollen serve as the Co-Chairs of the Childhood Cancer Caucus.  The event will be in the Congressional Auditorium, Capital Building Visitors Center. For more information, contact Andy Taylor (Andy.Taylor@Mail.House.Gov)

Saturday, September 19, 2015   4:00 p.m. ~7:00 p.m.

CureFest Welcome Reception

2014CurefestRecept_edited-1A welcoming event the night before CureFest at the J.W. Marriott Hotel, 1331 Pennsylvania Ave., Washington, DC 20004. This event is intended to be a casual family-friendly event being hosted by a group of foundations from the Washington-area. The event will feature dinner, a short speaking program, live music and activities for the children. Cost of this event is $35 and includes dinner and dessert. The welcome event is sponsored by several childhood cancer organizations which have defrayed the original expenses of the event. Following the event, we will walk as a group, two blocks to the White House for a candlelight vigil and rally. Register here.

Saturday, September 19, 2015  7:30p.m ~9:00 p.m

A Night of Golden Light

10698652_10152397941950665_5842703728529277096_nThis event will take place at Lafayette Park in front of the White House.  Night of Golden Light is a CureFest candlelight rally to honor the children taken by cancer and all those still in the fight. Children, their families and advocates will gather in front of the White House holding gold lights. The mission of the rally is to generate public awareness for childhood cancer. This event open to the public and  is free to all participants.

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Sunday, September 20, 2015  10 a.m. to 3:00 p.m.

CureFest Events

CureFest is an annual festival in Washington, DC that is the key component of the growing childhood cancer movement. CureFest is held in the Nation’s Capital each September in an effort to make childhood cancer a national priority. It is an opportunity for the childhood cancer community, the general public and elected leaders to come together as One Voice against childhood cancer. The ultimate goal is safer treatments and better outcomes for children diagnosed with cancer.

Curefestmall_edited-1Over 100 childhood cancer foundations and groups are expected to attend CureFest. Over 1,000 individuals have indicated they will be attending. It is a family-friendly event with activities for all ages. The event will include live music, entertainment, and various presentations by children and adults impacted by childhood cancer. Featured will be moving tributes for children who have or have had cancer. CureFest, again this year, will be held on the newly-renovated main Mall close to the U.S. Capitol. It is a very prominent location and is very close to public transportation, food vendors and public restrooms. Go to the CureFest Facebook page and “like” the page so you can be updated on all the information as it happens.

Registration is now open for CureFest for Childhood Cancer 2015! Be a part of the growing childhood cancer movement and attend this important event on the National Mall in Washington, DC on September 19th and 20th.  Join the CureFest Walk. Go to www.curefestdc.org to register for all events.

For the second time, CureFest will include a 3-mile walk on the National Mall. Participants will enjoy historic views of the U.S. Capitol, the Washington Monument, The World War II Memorial, The Lincoln Memorial and The White House.

CureFestWalk2015_edited-1The CureFest Walk is intended to increase the participation by the general public. Successful charity walks in the Washington-area typically start small but eventually attract several thousand participants within a few years of being established. There is a registration fee to help offset the costs of the event. Register and get a free T-Shirt.

You can register for the CureFest Walk and the Welcome Event at: www.curefestdc.org. Registration is not required for CureFest attendees not participating in the Walk and registration is not required for “A Night of Golden Lights.” These are both free.

For more information about CureFest visit the CureFest web page.

September 29, 2015  7:00 PM.

American Cancer Society Lights of Hope

The American Cancer Society Cancer Action Network (ACS CAN) will be honoring those who have been touched by cancer and remembering those lost, with a Lights of Hope 8393404216_550b3d4f64_zceremony  on September 29, 2015 in Washington D.C.

Lights of Hope  luminaries bearing the names of those touched by cancer will line the Capitol Reflecting Pool the evening of September 29th at the Nation’s Capitol. For additional information, please contact David Pugach at david.pugach@cancer.org .

This page will be updated every week, please check back for changes and additions.

Author: Joe Baber

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Posted in Childhood Cancer, Pediatric Cancer, Rare Disease | Tagged , , , , , , , , , , , , , , , | 5 Comments

As Our Children Wait – Part 4, Conclusion

AsOurChildrenWaitPart4_edited-1The rate of childhood cancer has been escalating since the 1970’s, today 1 in 285 children will be diagnosed with cancer before the age of 20. Our children continue to die, suffer and be irreparably harmed and all the while,  we hear is that childhood cancer is rare. The fact that each year over 2,000 children lose their lives to childhood cancer and that tens of thousands of children are irreparably harmed for life by current treatments demands that more be done to fund pediatric cancer research. Our kids deserve the best options we can give them and we are failing them.

TRANSPARENCY AND ACCOUNTABILITY

AsourChildrenWaitSMALL_edited-1The first three episodes of this series discussed various legislation that directs the National Institute of Health (NIH) to focus on children’s health issues and to have a transparent process that provides the public with information on NIH decisions and AsourChildrenWaitSMALLPart2_edited-1outcomes.   Congress passed the Children’s Health Act (CHA) in 2000 including initiatives requiring NIH to study risk factors for childhood cancer and carry out projects to improve outcomes among children with cancer and secondary conditions. The Act AsOurChildrenWaitPart3SMALL_edited-2required NIH to establish a Pediatric Research Initiative that would support and promote research, and called for NIH to develop the National Children’s Study (NCS) a cohort of 100,000 children to study children’s diseases. Even though these requirements are 15 years old, it is difficult to understand NIH compliance with the CHA.   After enrolling fewer than 6,000 children over the decade of design and implementation and spending over $1 billion with little to nothing to show for the effort, the National Children’s Study was abandoned in 2014.

The Government Performance & Results Act (GPRA) of 1993 and its enhancement by the Modernization Act of 2010 require an agency goal setting process, clear results and outcomes, transparency and accountability. Legislation requires that all agencies develop NIHMagnifier_edited-1strategic plans in collaboration with the public and Congress and that these strategic plans be used to assist agencies in setting priorities, articulating expected results and informing the budget process. GPRA and the Modernization Act are meant to provide the public with a clear picture of how budget decisions are made and a clear picture of what the taxpayer gets for their investment. Despite GPRA requirements and the White House open government directive, there is little transparency regarding how NIH sets priorities, how programs are evaluated, what results/outcomes are anticipated and how budget decisions are made.

In the past, Congress has left the setting of priorities to NIH, most without questioning of how decisions are made. More recently Congress has started to question how NIH sets their research priorities. In 2007, Congress began to urge NIH to increase investment in childhood cancer research in report language, in this same year the National Cancer Institute (NCI) began to indicate funds that supported grants that projected impact to childhood cancer, that percentage has stayed around 4% since 2007. (Legislation, report Plan_edited-1language and funding charts attached as other information at the end of this episode.) With 2015 appropriations Congress required that NIH develop an overarching strategic plan that incorporates all Institutes and Centers, this is due by December 2015. Congress indicates concern about whether taxpayers are getting the most for their investment asking how research agencies decide what research is deserving of federal funds and stating that there is a need for more transparency in the process. Yippee! But our children can’t wait, we must have more childhood cancer research in this decade to insure that we have the cures in the next decade, especially if the trend continues and childhood cancer rates continue to escalate.

A NUMBER’S GAME

Spinning the numbers to “defend” your position is another huge issue, a comprehensive look at the cost of childhood cancer is desperately needed. At the April 30th hearing with the Senate sub-committee, NIH was questioned about the continued rise in spending for AIDS Research, now at over $3 billion per year. At the same time, NIH has stated that this will be the first AIDS free generation. Senators questioned how NIH sets priorities and the wisdom of the increased spending in the AIDS portfolio.   Are priorities set by cost savings to the country? Dr. Fauci discussed the NIH desire to “completely end something” like we ended other epidemics like polio. He went on to say curing AIDS would mean a savings of $6 billion a year.

With a current U.S. population of 84 million children, and expectations that 1 in 285 children will be diagnosed with cancer before the age of 20, this means roughly 300,000 children will be diagnosed with cancer over the next two decades. On average the cost of treatment for childhood cancer is $500,000 per child. Using these statistics one could extrapolate that a goal of curing childhood cancer could save $7 billion per year, and this is probably a very conservative estimate. A $7 billion savings does not even consider savings that might be achieved by developing less harmful treatments for childhood cancer.   Two-thirds of survivors of childhood cancer suffer life altering impacts of treatment, heart issues, hearing problems, cognitive issues, secondary cancers, the list is long and costly. In addition, studies show that 95% of childhood cancer survivors suffer serious health impacts before the age of 45, largely as a result of treatments to cure their childhood cancers.

CONCLUSION

This four part series has been meant to examine a timeline over the past almost two decades and consider what the public should expect from NIH in regards to childhood cancer research. Two decades while our children wait for cures. NIH states childhood cancer is rare and that there are more survivors than ever before. The survival rate has increased largely due to the investment, but there is so much more to be done. Many childhood cancers have had little to no research – 20% of children are terminal on diagnosis. How is that acceptable?   It is undisputed that childhood cancer and adult cancers are different, continuing to use down-sized adult protocols to treat kids is not the answer. We need childhood cancer research, we need less harmful, less invasive treatments for kids.

As part of developing the overarching NIH strategic plan, we expect that outreach will be conducted with the childhood cancer community. We want NIH to hear our stories and understand why we believe investment in our children crucial. We want childhood cancer research to be a priority in the budget process. We want a transparent budget process – understanding how priorities are set, including goals, anticipated outcomes, taxpayer dollars invested and transparent reporting afterwards that gauges the value of investments and results. NIH has a responsibility to explain to the public what the plan is for childhood cancer. The litany of reasons for the lack of funding for childhood cancer research makes little sense to the aware community – childhood cancer is rare, we could do more if Congress provides more funding, etc. – these responses seem disingenuous. Our children are suffering and dying, they deserve the best that society and science can give them. Our children need to be a priority, they deserve action, not excuses.

PeoplePower_edited-1We need a movement, we need people power to tell Congress that childhood cancer research should be a priority.   People power is about politics and strategy – using various forces to create a movement, including the media that forces social change. In order to bring cures to kids, we need to exert our people power to force change. Please help create the movement, spread awareness about the realities of childhood cancer, use your voice to tell representatives constantly and continually that the current situation is unacceptable. Use your people power to demand that NIH follow the law provide transparency about goals, objectives, how priorities are set, results anticipated and where childhood cancer fits in agency plans. Use your people power to help effect change that will give kids hope and cures.

WHAT CAN YOU DO?

Use your People Power and help create the movement that will bring change for the kids battling cancer and the 16,000 kids that will be diagnosed with cancer this year, next year and every year.

  • 25394418_sCall, email, tweet, write, fax, facebook your representatives and senators. Get your Representatives contact information here: >Congress< Get your Senator’s contact information here: >Senate and Tell Congress that —  
  • Childhood cancer is the #1 disease related killer of kids in the U.S.
  • The incidence of childhood cancer is rising in the U.S.
  • 1 in 285 children will be diagnosed with cancer before the age of 20
  • 20% of children diagnosed with cancer are terminal on diagnosis
  • 60% of children suffer life altering impacts of treatment, a long term cost to society
  • 95% of survivors will suffer serious health impacts before the age of 45
  • NIH should make childhood cancer research  a priority
  • The public deserves transparency, deserves to understand the NIH plan for childhood cancer research, deserves to understand how priorities are set
  • Help us turn September Gold
  • In your communities, spread the awareness. Take every opportunity to spread awareness at community and social events
  • Hang gold ribbons in your community
  • Ask your town, state to light cities gold
  • Ask your local teams to wear gold ribbons, gold socks, gold shoelaces
  • Ask your employers, schools, social organizations to participate in September awareness events – crazy hair day, wear GOLD Wednesday, etc
  • Participate in social media events, fundraisers, awareness events
  • Attend the Congressional Caucus on Childhood Cancer on September 18, 2015. Urge your Representative to join the caucus
  • Attend CureFest for childhood cancer in Washington, D.C. the weekend of September 19-20, more info at http://www.curefestdc.org/

Author: Donna Carroll Carmical

OTHER INFORMATION —

  • 1993 – Government Performance & Results Act Passed requiring agencies to develop strategic plans, partner with stakeholders and members of the public to set priorities
  • 1995 – White House Task Force formed to consider children’s health issues including the “leading cause of mortality childhood cancer”
  • 2000 – Children’s Health Act – This Act required NIH to: study risk factors for childhood cancer and improve outcomes among children with childhood cancer; establish an initiative to support research directly related to diseases in children; and invest in tomorrow’s pediatric researchers.
  • 2002 – National Children’s Study – Authorized and funded by Congress to address the major effects on and high costs of child morbidity. After a dozen years and $1.2 billion of taxpayer dollars, the study was cancelled with little to show.
  • 2007 to 2014 Congressional Intent – Congress has sporadically inserted report language requiring NIH to increase funding for childhood cancer research. Since that time NCI has indicated that around 4% of the budget supported childhood cancer research.

SENATE REPORT LANGUAGE

2007 — Pediatric Cancer– The NCI is currently partially funding a portfolio of studies looking at the causes and most effective treatments for childhood cancers. The Children’s Oncology Group [COG] is conducting important laboratory research on cancer cells to discover the reasons children get cancer, developing and making available new treatments that destroy cancers, and improving the quality of life and long-term survival for pediatric cancer patients. However, the NCI is only funding approximately 50 percent of the approved collaborative pediatric cancer research projects. The Committee understands the COG acts as a cancer center without walls and reimburses hospitals for enrolling pediatric cancer patients into the best available clinical trials. The Committee encourages the NCI to increase the percentage of approved funding directed to the COG in order to open additional treatment protocols and make more treatment options available to physicians and families.

2009Pediatric Cancer– The Committee urges the NCI to expand and intensify pediatric cancer research, including laboratory research to identify and evaluate potential therapies, preclinical testing, and clinical trials through cooperative clinical trials groups. Such research should include research on the causes, prevention, diagnosis, recognition, treatment, and late effects of pediatric cancer.

2010 — Pediatric Cancer– The Committee urges the NCI to further expand and intensify pediatric cancer research, including laboratory research to identify and evaluate potential therapies, preclinical testing, and clinical trials through cooperative clinical trials groups. Such research should include research on the causes, prevention, diagnosis, recognition, treatment, and late effects of pediatric cancer.

2012 — Pediatric Cancer- The Committee notes that childhood cancer research accounts for less than 5 percent of the Institute’s annual budget and encourages NCI to increase that amount, as cancer remains the leading cause of disease-related death in children. More effective and less toxic treatments are needed.

2013 — Pediatric Cancer- The Committee continues to urge NCI to devote more of its funding specifically for research on pediatric cancer, including pediatric low-grade astrocytoma. The Committee requests an update in the fiscal year 2014 congressional budget justification, including efforts that could result in more effective, less toxic treatments.

2014 — Pediatric Cancer- The Committee encourages NCI to put a higher priority on pediatric cancer, as cancer remains the leading cause of disease-related death in children. More effective and less toxic treatments are needed, including materials-based strategies for localized drug delivery.

2015 Pediatric Brain Tumors – The Committee commends NCI and NINDS for developing the coordinated Pediatric Neuro-Oncology Section and urges NCI to devote additional resources to research projects focused on pediatric malignant brain tumors conducted in partnership with the Pharmacology and Experimental Therapeutics Section. Research should be aimed at developing novel translational and therapeutic studies. The Committee places a priority on efforts to evaluate new agents with novel mechanisms of action for the treatment of childhood brain tumors. In addition, the Committee notes that the Brain Cancer SPORE program has no center of excellence that focuses on pediatric cases and encourages NCI to address this gap. Finally, the Committee directs NIH to submit a report no later than 60 days after enactment of this act on pediatric brain cancer. The report should include: the state of science; current NIH funded research initiatives; and key research questions that need to be prioritized.

Slow-Growing Children’s Brain Tumors – the Committee is encouraged by NIH’s progress in better understanding the pathways that are active in Pediatric Low Grade Astrocytoma (PLGA) and pleased that NCI launched the first clinical trial for a targeted agent directed at genomic characteristics common in PLGA. The Committee is eager for these discoveries to be translated into effective therapies for PLGA patients. The Committee encourages NCI to continue its work on PLGA through the Specialized Programs of Research Excellence focusing on brain cancer, and to prioritize PLGA through the Cancer Therapy Evaluation Program.

NCI Operating Plan – Childhood Cancer

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Posted in Cancer, Childhood Cancer, Pediatric Cancer, Rare Disease | Tagged , , , , , , , , , , , , , , , , , , | 1 Comment

As Our Children Wait – Part 3

AsOurChildrenWaitPart3_edited-2Childhood cancer has been on the rise since the 1970s, a White House task force was formed in the 1990s to look at the underlying causes of children’s health issues.

Episode 1 provided an overview of the Children’s Health Act (CHA) of 2000, legislation authorizing expanded research regarding childhood health issues including childhood cancer. Episode 2 examined the National Children’s Study (NCS), a major initiative of the CHA. After 14 years, the NCS study design remained problematic and with next to nothing to show for the $1.2 billion in taxpayer dollars invested, the Director of NIH cancelled the children’s study.

In Episode 3 we focus on questions of priority setting, strategic planning, accountability and transparency.

GPRA & STRATEGIC PLANS

A little introduction to what is often called the Results Act and why it matters to the public. Officially named the Government Performance and Results Act (GPRA) of 1993 (and enhanced by the Modernization Act of 2010), the Act was meant to address a range of concerns about government accountability and performance.

GPRA intended to improve confidence in the Federal government and provide the American people with information about the results of government. Agencies are required to develop strategic plans that describe goals, objectives and expected outcomes of activities and to use this information to set priorities and formulate budget requests. GPRA is unique in that the agencies are required to annually analyze results, determine value of results, and integrate that information into the budget decision process.

 THE PUBLIC HAS AN INVESTMENT

Think of it this way, in the private sector corporations have investors and shareholders. In the public sector, Federal agencies are financed by our tax dollars. We are the investors and as such, agencies have a responsibility to the taxpayer financing government operations. As an investor in agencies, we should have a kind of voting right in the direction agencies are taking. GPRA requires agencies to communicate externally as they develop strategic plans. Agencies are required to conduct outreach to the public, shareholders, and with Congress as they develop strategic plans and set priorities.

Taxpayers, the investors, have ownership in Federal entities and our dividends are the results or outcomes. That is, what we expect agencies to deliver with the investment of tax dollars. Agencies are required to develop annual plans as part of their budget requests, and tie their budgets to programs and outcomes. Projected outcomes should make sense in terms of what the public needs and wants, cost benefit should be part of the equation and the public should be aware of and satisfied with their return on investment.

As an investor, we have a right to inspect the books. Current legislation requires that agencies produce financial statements and audits as part of their annual reporting process and that this information be available to the public.   All of this information, strategic plans, budget requests, annual performance plans, annual performance reports, financial statements are required to be transparent to the public and available through performance.gov.

As an investor we have a right to demand accountability for poor management. In the private sector, this is the right to sue, in the public sector this is part of the performance management process for senior managers of the government. The Senior Executive Service (SES), managers who run Federal agencies, receive annual performance ratings that are based on how they deliver on agency GPRA goals, objectives and results.

So in a nutshell, the taxpayers should be included in the priority setting process, have transparency about products, services, outcomes delivered, and a right to demand accountability for mismanagement.

GAO REVIEW OF NIH PRIORITY SETTING

The NIH is made up of 27 Institutes and Centers (ICs) and has a budget of approximately $30 billion, 80% of this supports extramural research.   In 2014 Congress asked the Government Accountability Office (GAO) to review NIH funding related to leading disease and health conditions and determine how priorities are set. The GAO is an independent, nonpartisan agency that works for Congress, often called the “congressional watchdog,” investigating, at the request of Congress, how the federal government spends taxpayer dollars.

As part of its review GAO investigated how research priorities are set at NIH and how allocation decisions about research funding are made across selected diseases and conditions. The GAO review looked at the five ICs with the largest funding base, one of the ICs scrutinized was the National Cancer Institute (NCI). Interestingly, GAO found that each individual IC sets its own research priorities. ICs revealed that since potential research projects exceed available funds, all five set priorities in a similar fashion — considering

  • scientific needs and opportunities
  • gaps in funded research
  • burden of disease
  • public health need

All five ICs advised GAO they had strategic plans in place for individual centers. ICs also indicated they considered input from stakeholders and looked to NIH leadership to provide overarching direction in terms of setting priorities.

WHERE OH WHERE IS THE NATIONAL CANCER INSTITUTE STRATEGIC PLAN

Recently we tried to find the strategic plan for the National Cancer Institute (NCI), one of the ICs studied by GAO. Even though the NCI advised GAO that they had a strategic plan, we couldn’t locate it behind the strategic plan link on the NIH website. When we inquired about the NCI strategic plan this is the response we received from the NIH Office of Advocacy Relations —

“the specific requirements of Perfomance.gov apply at the agency level—in this case to the Department of Health and Human Services (HHS), which oversees the National Institutes of Health and therefore the National Cancer Institute.  While the HHS plan needs to meet specific GPRA reporting format and requirements (and does), other levels within an agency are not required to follow that same format.  HHS streamlined GPRA reporting in 2011; NIH and other Operating Divisions/Staff Divisions no longer have to publish individual GPRA plans/reports.”

CONGRESS REQUIRES NIH STRATEGIC PLAN

Interestingly, Dr. Collins appeared before the Senate sub-committee on appropriations on April 30, 2015 and advised the Senate that NIH was working on an overarching strategic plan for the entire Institute. He went on to say that “each IC has its own strategic plan in place” but we are working on a comprehensive NIH strategic plan that will be sent to the Senate by December 2015. Actually, NIH is working on this strategic plan and submitting by December 2015 because that’s what is required by law in their 2015 appropriations Bill — not because they decided to do it themselves. In the 2015 Omnibus Appropriation Bill Congress required, “NIH shall submit to Congress an NIH-wide 5 year scientific strategic plan as outlined in sections 402(b)(3) and 402(b)(4) of the PHS Act no later than 1 year after enactment of this Act.”

Congress has increased inquiry about how NIH sets priorities and makes funding decisions. In the past, Congress has left setting priorities to NIH and has done very little questioning about how the science community at NIH determines what to fund. Thankfully,  some members of Congress have begun to pose questions regarding the soundness of this past practice.

TRANSPARENCY

Fulfilling the requirements of GPRA can be powerful strategy for solving government performance and addressing priorities. In government, as in our personal lives, we have infinite needs, wants, desires and finite resources.

Strategic plans are meant to make visible to the public the goals, objectives and expected outcomes of Federal agencies. They should serve as a roadmap for agencies in their annual planning process and help agencies determine what programs are making progress towards goals and what programs need to be ditched because they aren’t delivering or cost too much. The public should have a good idea about outcomes that agencies are moving towards, understand progress made and costs necessary to achieve results.

In addition to GPRA requirements, the White House issued an open government directive in 2009 seeking to increase transparency, participation and collaboration. Its message to agencies says, transparency promotes accountability by providing the public with information about what the Government is doing. Participation allows members of the public to contribute ideas and expertise so that their government can make policies with the benefit of information that is widely dispersed in society. Collaboration improves the effectiveness of Government by encouraging partnerships and cooperation within the Federal Government, across levels of government, and between the Government and private institutions.

In this series we’ve presented some legal requirements for strategic planning, external consultation required when setting goals and objectives, how the strategic plan ties to budgets requested, and the condition that all of this informs how agencies set priorities.

In the next episode we will finish with an overview and consider what all of this means as you look at the picture of legislation, requirements and taxpayer expectations. We will consider where we are today, how we might reinforce our argument that childhood cancer research should be a national priority and talk about what you can do to help change this picture for kids. Stay tuned…………

Author: Donna Carroll Carmical

Want to help make childhood cancer a priority? Ask lawmakers who help determine the budget for the National Cancer Institute for more than 4% of the research budget is one way to show support.

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Posted in Childhood Cancer, Pediatric Cancer, Rare Disease, Cancer | Tagged , , , , , , , , , , , , , | 1 Comment

As Our Children Wait – Part 2

AsOurChildrenWaitPart2_edited-1“As Our Children Wait, Part 1″  discussed the Children’s Health Act of 2000, which was passed after extensive work by a White House task force recognized concerns about children’s health. The Children’s Health Act required the National Institute of Health (NIH) to carry out many projects including focus on improving outcomes for children with cancers, creating a Pediatric Research Initiative and requiring facilitation of pediatric research.

THE NATIONAL CHILDREN’S STUDY (NCS)

The Children’s Health Act authorized the NIH to conduct the National Children’s Study. The NCS was aimed at determining environmental influences that might impact children’s health, with the intent that understanding children’s diseases would aid in finding cures or preventing conditions from occurring in children.  Congress intended that the study would address the major effects on and high costs of child morbidity due to potentially preventable conditions in the U.S. The NCS proposed to examine the effects of environmental influences on the health and development from birth to age 21, of a cohort of 100,000 children.

NCS DESIGN PHASE 2002 -2007

The preliminary development of the NCS was long and arduous, 2002 saw the establishment of the NCS Advisory Committee. In 2003 the NCS Program Office was established — many working groups were created, many white papers were produced and workshops were held. By 2005, the NCS initiated what was identified as three milestone events:

  1. NCS first-stage sample
  2. Data management
  3. Initial Vanguard Centers contract

In 2007, five years later,  the Research Plan for NCS was completed and reviewed by an outside panel and Congress appropriated funds for implementation.

NCS IMPLEMENTATION 2008 – 2012

Real implementation of the NCS and sample recruitment, was scheduled to begin in 2008 with addition of more Vanguard centers in 2009. There were delays during 2008 and 2009 and when real recruitment began in 2009-2010 it was revealed that the intended approach was going to be more costly and time consuming than planned. In 2011 the Program Office begin testing alternative recruitment methods.

In 2009 the original NCS lead was ousted for misleading Congress about the cost of the study. A new lead was appointed and many researchers blame his decision making for the ultimate collapse of the study. In 2011 the NCS lead as well as the Director of NIH were advised that the study was at high risk of scientific failure and corrections were recommended to get the study back on track. The new lead was accused of mismanagement. The NIH Director, Francis Collins, subsequently defended the NCS lead and advised that Congress deserved some of the blame saying, “There was an effort initially put forward by the Congress which maybe from the start was likely to run into serious trouble.”

NCS REVISED 2013

In 2013 the study design was revised with a 2015 start date set for the main study. The revised study was seen as having problems with concept, methodology and administrative challenges continued.   In its 2013 budget request, the NIH decided to reduce the budget and scope of the children’s study. Subsequently, expert advisors of the Advisory Committee begin to resign. Ellen Silbergeld an environmental scientist resigned charging that the study had been “significantly abrogated” by managers at NIH.

NCS ON HOLD AND REVIEWED 2014

Congress required NIH to contract with the National Academy of Sciences to conduct a review of the NCS, the Academy published this review in June 2014. They stated their “deep concern about the overall leadership and management of the NCS,”  but concluded that the study was not beyond saving and identified feasibility and pathways for success. NIH leadership ignored these recommendations.

The Director of NIH, Francis Collins, placed the launch of the main study on hold and charged a working group of the Advisory Committee to determine the viability of the NCS. That working group came up with basically the same conclusions as the National Academy of Sciences, advising that the NCS was not practical as currently designed and they too recommended that the NIH champion and support new study designs. The working group felt that utilizing advances in technology and research in a redesign could make the original goals of the NCS more “achievable, feasible and affordable.”

NCS SHUTDOWN 2015

Director Collins ignored recommendations from both groups. In December 2014, the NIH Director announced the closure of the study and the transition of the NCS program office. He stated, “I am disappointed that this study failed to achieve its goals. Yet I am optimistic that other approaches will provide answers to these important research questions.”

In the past two decades since the White House task force identified trends in children’s health issues, passage of the Children’s Health Act, design of the National Children’s Study not much has happened in terms of childhood cancer research. During this time more than 300,000 children have been diagnosed with cancer, more than 40,000 children have died, tens of thousands of children suffer life altering impacts of treatments largely due to the lack of research and related development of pediatric drugs, cures, treatments, protocols. The children wait … and without significant investment in research now, children will be in a similar place two decades into the future.

ANALYSIS PARALYSIS

Some critics of the study say that researchers spent years debating which questions to investigate. It is reported that while 100 scientific papers based on pilot research have been published many focused on methods of study design and data collection rather than the original intent of the study. An advisor from Brown University recently stated, “At some point, you have to come to grips with reality: this much time, this much money, make it happen.”   At some point we may have to consider that brilliant scientists may not be the best business managers, budget managers, the best CEOs. What we often see is analysis paralysis, over-analyzing, too many detailed options, a lack of choice. The cost of decision analysis exceeds the benefits that could be gained by enacting a decision or pursuing a plan. There is no awareness that time is money, and in this case, time is also the lives of our children.

$1.2 BILLION WITH LITTLE TO SHOW

The National Children’s Study struggled for over a decade and cost tax payers more than $1.2 billion, there’s little to show for this effort.   In the first 7 years, NIH spent $54 million on planning. In 2007 Congress approved $69 million to start the project and continued appropriations through 2015. A cohort of 100,000 children was envisioned in 2000 and 15 years later only around 5,000 children were enrolled. In the private sector there is no doubt what would happen with this type of management, heads would roll. In this instance there seems to be no accountability, the leaders not only do not get penalized, but they make the decisions on how to spend money originally intended to positively influence outcomes regarding children’s health.

 WHAT TO DO, WHAT TO DO WITH $165 MILLION

Questions remain about what NIH will do with the appropriations provided in 2015, and what happens with the original legislative intent of The Children’s Health Act.

In 2015, Congress appropriated $165 million to carry out the work of the NCS. Congress directed in Bill language for 2015 …”that $165 million shall be for the National Children’s Study or research related to the Study’s goals and mission, and any funds in excess of the estimated need shall be transferred to and merged with the accounts for the various Institutes and Centers to support activity related to the goals and objectives of the NCS: Provided further, that NIH shall submit a spend plan on the NCS’s next phase to the Committees on Appropriations of the House of Representatives and the Senate not later than 90 days after the date of enactment of this Act.” The legislative date for the spend plan was March 14, 2015.

In January 2015 NIH presented a plan for reallocation of the funds indicating that Bill and report language (there is no report language on the Congress.gov website) direct NIH to maintain the mission and goals of the NCS with flexibility on how to carry this out. A slight stretch!

The NIH, the agency that couldn’t successfully manage the NCS, has come up with three initiatives for spending the $165 million. They propose to —

  1. develop tools to enhance studies of environmental influences on pediatric diseases
  2. study the influence of the environment on in utero development with the goal of identifying the “seeds” of future diseases and conditions
  3. expand examination of environmental influences on later child development by leveraging extant programs.

The proposal to spend 2015 funds seems to be more of the same. What are the expected goals, objectives, results of these three initiatives? Are these projects already in place or new programs? Are these short term projects and is funding sufficient to deliver real results, outcomes? Why wouldn’t NIH use these funds to support pediatric research grant proposals in keeping with the original legislation? Was there consultation with other agencies, as required in the original legislation, with external entities?

IN THE MEANTIME CHILDREN WAIT FOR CURES

The legislative intent of the Children’s Health Act was concern about children’s health and rising rates of childhood cancer. There has been virtually no progress over the past 15 years. A simple analogy — we have a pipe leaking in our home, we put a bucket under the leak and go down to the water company to determine the external source that might be causing the pipe to leak. In the meantime the bucket overflows, the basement floods, the structure begins to rot and mold. Childhood cancer is in a similar boat, the children wait for cures while we seek reasons why children are getting cancer. We need cures now, studies simultaneously but don’t ignore the current problem – children are suffering, children are dying. Children need specific childhood cancer research to develop pediatric cures. Many current treatments are a guessing game, using downsized adult protocols, developed for adult cancers to treat kids.

THE INCIDENCE OF CHILDHOOD CANCER IS RISING, IS THERE A PLAN?

NIH does not seem to have a plan, or at least any plan about childhood cancer research goals that are transparent to the public. Thankfully, the Senate has started to ask questions about how NIH sets priorities.   I think the general public would agree that more taxpayer dollars should be invested in childhood cancer research.

Perhaps we should leave the study of possible environmental impacts, cancer clusters, etc. to the research community at the Environmental Protection Agency and research to develop cures — childhood cancer research to NIH, the nation’s medical research agency.

We have evidence that investment in childhood cancer research has changed the picture dramatically for childhood leukemia, but according to NCI’s own study, childhood leukemia accounts for less than one-third of childhood cancers. It is undeniable that the lack of research in all childhood cancers leads to pain, suffering and all too often a death sentence for kids.

In Part 3 we pursue legal requirements for demonstrating results, transparency and setting priorities. Stay tuned ……….

Author: Donna Carroll Carmical

Want to help make childhood cancer a priority? Ask lawmakers who help determine the budget for the National Cancer Institute for more than 4% of the research budget is one way to show support.

SenSnailMail_edited-1  SenTWEET_edited-1

Related Articles:

“As Our Children Wait, Part 1″

“Moving the Needle”

Posted in Cancer, Childhood Cancer, Pediatric Cancer, Rare Disease, Uncategorized | Tagged , , , , , | 4 Comments

As Our Children Wait – Part 1

AsOurChildrenWait_edited-1Blissfully ignorant! That was us before our grandchild Declan was diagnosed with a childhood cancer called AT/RT. On diagnosis we were told that for Declan there was no hope — no cures, treatments, protocols. We were told this particular type of childhood cancer was deemed “rare” and there had been little to no research. Thus began our journey into the world of childhood cancer and subsequently recognizing the apathy on the part of those who make decisions about funding research.

For many childhood cancers not much has changed in decades. Research, reading, inquiry, everything seemed to say, that although billions of taxpayer dollars are provided to fund research programs, childhood cancer research is not even on the radar screen and certainly not a priority in the Federal budget. There have been various pieces of legislation related to childhood cancer, Congress appropriates billions of taxpayer dollars for research each year – but childhood cancer research does not get a fair share of those funds.

Childhood cancer is the #1 disease related killer of kids in the U.S., the incidence of childhood cancer in this country is rising; 1 in 285 children will be diagnosed with cancer before the age of 20; over two thirds of survivors suffer life-altering impacts of treatments; and, 95% of survivors will suffer severe health impacts before the age of 45. Let me say it again, the incidence of childhood cancer continues to rise in this country – we need research now! Despite gripping statistics, resources available and the fact that Americans proclaim our children to be our most valuable asset — childhood cancer research is not a priority and is vastly and consistently underfunded. Why?

In the 1950s almost all children who were diagnosed with cancer, like Robin Bush, were given a death sentence at diagnosis. Robin was the 3 year old daughter of former President George H.W. Bush and his wife Barbara. During an interview a few years ago, former first lady Barbara Bush recalled  when Robin was diagnosed with leukemia in 1953. Mrs. Bush was told by the doctor’s, “she’s going to die.”

Today, thanks to research, childhood leukemia is no longer an absolute death sentence for kids. By the late 1980s, cure rates for pediatric acute lymphoblastic leukemia (ALL) climbed above 80%. Investment in research has changed the picture for childhood leukemia – still more to do but childhood cancer research has changed the diagnosis from a death sentence to a likely cure.  According to a September 2013 report by the National Cancer Institute (NCI) leukemia/lymphomas account for about one-third of leading childhood cancers. There are 16 major types of pediatric cancers and over 100 subtypes.

CHILDREN’S HEALTH ACT OF 2000

The Children’s Health Act of 2000 was signed into law on October 17, 2000. The Act intended to focus on children’s health issues, authorized the formation of federal child health funding programs and included pediatric research initiatives. The legislation called on Health and Human Services (HHS) to provide services to children whose lives have been affected by diseases such as cancer.

The Children’s Health Act of 2000 contains statutory provisions related to childhood cancer —

  • Title XI requires the Secretary of HHS acting through the NIH to study risk factors for childhood cancer and carry out projects to improve outcomes among children with childhood cancers and secondary conditions. Title XI states “Such projects shall be carried out by the Secretary directly and through awards of grants or contracts.”
  • Title X creates the Pediatric Research Initiative requiring the Secretary to establish an “initiative” at NIH to conduct and support research that is directly related to diseases, disorders and other conditions in children. The “initiative” provides funds to enable the Director to increase support for pediatric biomedical research, to enhance collaborative efforts among the institute and increase “development of adequate pediatric clinical trials and pediatric use information to promote the safer and more effective use of prescription drugs in the pediatric population.”
  • Section 1002 provides for “Investment in tomorrow’s Pediatric Researchers.” In order to ensure the future supply of researchers dedicated to the care and research needs of children, the Director of the Institute, shall support activities to provide an increase in the number and size of institutional training grants to support pediatric training an increase in the number of career development awards for professionals to support pediatric basic and clinical research.

A little overview on various organizations mentioned in this legislation. The Department of Health & Human Services (HHS) is a cabinet level agency with a mission to “enhance the health and well-being of Americans by providing for effective health and human services and by fostering sound, sustained advances in the sciences underlying medicine, public health, and social services.” HHS notes that they administer more grant dollars than all other federal agencies combined.

The National Institute of Health (NIH) is one of the 11 agencies that make up the Department of HHS. The NIH is “the nation’s medical research agency – making important discoveries that improve health and save lives.” NIH is the largest source of funding for medical research in the world, creating hundreds of thousands of jobs in research institutions in every state and around the globe. NIH indicates they are responsive to Congressional legislations that adjusts NIH programs to meet changing research needs. The NIH homepage indicates that they encourage and depend on public involvement in federally supported research and activities. (So let them hear from you.) The Office of the Director, led today by Francis Collins, is the central office at NIH. The Office of the Director sets policy for programs and activities of all NIH components. The National Cancer Institute (NCI) is a component of NIH and they are a leader in the nation’s cancer research.

The Children’s Health Act was passed 15 years ago. During this time, the children wait. In 15 years we have lost more than 30,000 children to childhood cancer. While the children wait, tens of thousands of children receive treatments that often consist of down-sized adult protocols due to the lack of research and corresponding lack of pediatric protocols, treatments and cures.

The legislation requires that the Secretary “carryout projects to improve outcomes among children with childhood cancers and secondary conditions,” it requires NIH to support research directly related to disease, it requires NIH to invest in pediatric research to increase the number and size of pediatric training grants. Since childhood cancer is the #1 disease related killer of kids in the U.S. a “reasonable person” might assume that based on compelling facts and statutory legislation, childhood cancer research would be a PRIORITY for HHS, NIH and NCI. Of course there is a great lack of transparency in how they set priorities, outcomes, budgets, etc. For many of us dealing with childhood cancer and trying to get the facts, it does not appear that childhood cancer research is any kind of priority for HHS or its components. Any mention of childhood cancer is scarce in the myriad of budget documents prepared by the HHS, NIH and NCI. Childhood cancer is rarely mentioned if at all in testimony and hearings. Although there was rather cavalier discussion of childhood cancer research by the former NCI Director during past budget hearings — childhood cancer is rare, NCI has it covered, NCI could do more with more money, but basically it’s not a priority for them.

It appears that NIH deems it statistically acceptable that over 2,000 children a year die. How many children will it take, how long will children have to wait before childhood cancer research becomes a priority in the Federal budget?

Subsequent to passage of the Children’s Health Act, NIH launched the National Children’s Study (NCS).   The study was funded by Congress for over a decade and intended to provide information on health and development of 100,000 children across the U.S.   Part 2  discusses the NCS, the collapse of the study and the investment of $1.2 billion of taxpayer dollars.

Author: Donna Carroll Carmical

Related Articles:

As Our Children Wait, Part 2

Want to help make childhood cancer a priority? Ask lawmakers who help determine the budget for the National Cancer Institute for more than 4% of the research budget is one way to show support.

SenTWEET_edited-1 SenSnailMail_edited-1

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Why Motorsports?

WhyMotorsports_edited-1Why is it important for the teams, drivers and fans of the motorsports racing communities such as NASCAR, NHRA, IndyCar, WoO, IMSA and others to display “Gold in September?”

Because motorsports races are, as a whole, the No. 1 spectator sport (over 75,000,000 fans) and the races are always among the top sporting events attended in the United States. The big  NASCAR, NHRA and IndyCar races are broadcast in many countries and attract a lot of television viewers! With this kind of reach, a lot of awareness could be raised about childhood cancer! As an example, NASCAR teams and track owners receive a lot of corporate sponsorship support and displaying Gold in September, would have the potential, to not only raise public awareness about childhood cancer, but also reach many huge corporations, that might be willing to help fund childhood cancer research. NASCAR had one major sponsor who helped in the past. Aflac which donated over 87 million dollars to childhood cancer research since 1995 played a big role in promoting awareness. Sadly, they will not be a sponsor this year. We need other big sponsors to fill the NASCAR gap left by Aflac.

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Aflac and driver Carl Edwards support Childhood Cancer Awareness Month each September

Gold in the most popular racing series, NASCAR, might get the attention of the NFL and other sports, who have also been reluctant to join in to promote “Childhood Cancer Awareness” month, inspiring them to help! One of the biggest problems, is the general public thinks of childhood cancer, as cute, smiling bald children in St. Jude commercials, that go there and get cured! And while St Jude does an amazing job treating children and doing research, many times, this is not the reality. Most people are unaware, that childhood cancer is the No. 1 disease killer of children, that childhood cancer research is grossly under funded, that it’s called rare, yet 1 in 285 children in the US will get cancer before age 20 and that there is a huge need for less toxic treatments! Most of the chemo drugs used on children are more than 20 years old and were made for adults. Even if a child survives cancer, many times they suffer life long problems or shortened lives because of the treatment. A lot of the awareness and research funding, is raised by hundreds of childhood cancer organizations, like St Baldricks, Alex’s Lemonade Stand, and Cookies for Kids Cancer, shaving heads, selling lemonade and cookies! They do an awesome job, but they need help!

DriverChart2_edited-1Jeff Gordon is dedicating his season to children with cancer by pledging $2,400 for each win and $1,000 for each top-ten finish of the 2015 NASCAR season.  Given their huge following, we should ask NASCAR teams, drivers, and sponsors to join in and help us raise awareness. Team owners and drivers can  ask their sponsors to give up space for a gold ribbon to be placed on the car or maybe “September is Childhood Cancer Awareness Month” on the bumper or something on the hood.

Wouldn’t it be great if the car manufacturers (Ford, Toyota, Chevy) involved with racing, stepped up and furnished a gold painted pace car or at least a white car with a big gold ribbon predominately displayed for all major races held in September to promote childhood cancer awareness month!  The tracks where September races are held could even promote awareness by using the gold theme.

September 1, 2013 Atlanta 500 Race

September 1, 2013 Atlanta 500 Race

The last few years, Chevy and the American Cancer Society have teamed up, to run a pink pace car (breast cancer awareness) around the beginning of September (August 31st this year). The childhood cancer community has no problem with a pink pace car and does support breast cancer awareness month, but just not in September. Gold is for September and Pink is for October!

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WoOSchedule_edited-1NASCAR, NHRA and World of Outlaw teams, their drivers and sponsors raise awareness about many great “causes,” like autism, diabetes, COPD and breast cancer. What greater tragedy and cause, than children dying from cancer and very little being done NHRASchedule_edited-1about it? Everyone has the power to make a difference in our world, some use their words, some use their talents and some use their resources. We need Gold in all September races in  2015!

How can you help? Share this post with all your friends. join our Motorsports Go Gold Facebook page and stay informed on our progress.  Since NASCAR is the largest racing series, consider writing to your favorite owners, drivers, sponsors, and tracks (Darlington, Richmond, Chicagoland, and New Hampshire) and let them know how you feel. DO NOT WAIT UNTIL SEPTEMBER. START NOW! 

Author: Greg Puryear

Join us on Facebook and help us achieve our mission for childhood cancer awareness.

FAcebookClick_edited-1Like Motorsports Go Gold Facebook page

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Posted in Cancer, Childhood Cancer, NASCAR, NHRA, Pediatric Cancer, Rare Disease, Sprint Cup, World of Outlaws | Tagged , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , | 7 Comments

Moving the Needle

MoveTheNeedle_edited-1Recently in Washington, D.C. there was a hearing to discuss the $30 billion 2016 budget requested by the National Institute of Health (NIH), these are our tax dollars at work.   The largest of our childhood cancer organizations admirably raises tens of millions of dollars for childhood cancer research, but they can only do so much. Without more childhood cancer research funding, cures will probably be decades into the future. Kids need cures now. Our children need a fair share of the billions of Federal research dollars to support childhood cancer research, this is the quest of the childhood cancer community!

WhereNCIFundingTHE BUDGET – The National Cancer Institute (NCI) budget of $5 billion is part of the larger National Institute of Health (NIH) $30 billion budget. NCI indicates that childhood cancer research grants on average receive around $185 million (3.9% of NCIs $5B).   Over the past couple of years, childhood cancer advocates have been, in a variety of ways, urging NCI to increase the amount of funding going to childhood cancer research.

On April 30, 2015 the Director of NIH, Dr. Francis Collins, presented the 2016 budget request to the Senate committee. The Senate committee makes recommendations to the full Senate and later joins with the House of Representatives to make the final decision on the NIH budget request.

SenCall_edited-1THE CAMPAIGN – Ten days prior to the NIH hearing, a group of us from the childhood cancer community began the third phase of the #morethan4 campaign. We campaigned to let Senators know how important childhood cancer research is to Americans – SenTWEET_edited-1our belief that childhood cancer research should be a priority in the Federal budget.   We felt that days of calls, tweets, facebook posts, emails would get attention and create inquiry about the fact that NIH virtually ignores childhood cancer research in its budget SenSnailMail_edited-1submission. Even though our combined posts and tweets did not trend or get media attention, we felt sure we had created sufficient interest to have Senators question NIH about childhood cancer research.

THE HEARING – On April 30th I lined up early to attend the hearing where NIH would defend their budget request to the Senate committee.   Even though I told myself that if there was any discussion about childhood cancer it would be minor, I had a great sense of optimism that Senators had heard our cry that children are suffering and dying due to the Donna in the NIH hoodlack of childhood cancer research and they would question NIH about childhood cancer research funding levels.   When the doors opened, and the brilliant leaders of NIH science took their seats I took my seat with great anticipation. The Senators entered and the hearing began. The committee chair, Senator Roy Blunt of Missouri, made his opening statements on behalf of the committee and Dr. Collins made his opening statement, and Senators took 5 minute turns to question Dr. Collins.

CollinsThe questions started — Alzheimer’s, precision medicine, the Brain Initiative, the establishment of a 1 million person cohort, more precision medicine, Barbra Streisand’s advocacy for women’s heart disease, lots more on Alzheimer’s, chronic pain and problems with opioids, more on Alzheimer’s, Ebola and the fact that 1 in 4 American’s is clinically depressed. NIH was questioned at length about how they set priorities in terms of making budget decisions and there was a somewhat lengthy discussion about the amount of funding spent on AIDS Research (currently NIH has a $3 billion research budget for AIDS). After almost 2 hours of questioning, Senator Blunt ended the hearing. Two hours of questions and not one mention of kids health or childhood cancer, I wanted to stand-up and scream.

THE LETDOWN – As I left the hearing room I felt discouraged. I reflected back to the day my grandson Declan was diagnosed with a “rare” form of cancer, AT/RT, and the horror of the words that we should take him home and make him comfortable until he died because there was no hope. There was no hope because so many of the rare pediatric cancers receive little to no research funding. When Declan died, we as a family committed to changing this picture for kids. We committed to raising funds for childhood cancer research. After many months of looking at numbers and budgets, we quickly realized that the only near term solution for childhood cancer cures was to get the Federal government to understand the realities of childhood cancer and get some of the billions of Federal research dollars donated to kids.   This is not a question of more money, but a questions of priorities and our kids deserve to be a priority.

imagesNCI states that approximately 2,000 children will die as a result of childhood cancer this year. Some believe this is a conservative estimate. It is appalling that it seems to be accepted as okay that 2,000 children are going to die, that 20% of children are terminal on diagnosis – how many deaths are enough?

So much has been written about the fact that adult cancers and childhood cancers are different, yet we continue to use downsized adult protocols to treat kids because we haven’t invested sufficiently in childhood cancer research.   Unbelievably, 60% of children suffer life altering impacts of treatment – hearing loss, heart problems, secondary cancers and much more. A St. Jude’s study indicates that 95% of childhood cancer survivors will have chronic health problems before the age of 45. There are currently 380,000 childhood cancer survivors in this country.

In the hearing I wanted to stand up and yell, the incidence of childhood cancer is rising, adult cancer research is not the answer. NCI estimates that 16,000 children will be diagnosed with cancer this year, 1 in 285 children will be diagnosed with cancer before the age of 20. Why isn’t this a terrifying number? If I bought 285 lottery tickets and 1 was a winner that would be a miracle, why is it that 1 in 285 kids getting cancer doesn’t scare NIH and Congress.

It seems illogical that all of these compelling statistics don’t make the case for an increased investment in childhood cancer research.

As these thoughts were running through my head I felt like giving up, if these statistics don’t make the case for an increased investment in childhood cancer research how can we ever convince our government to invest in kids. Then my thoughts went back to Declan and the 10,000 children that have died of childhood cancer in the 5 years since Declan’s death. When I started thinking of these kids, the ones who have died, the pain and suffering of the many kids fighting today, and the lives forever changed as kids are diagnosed today, tomorrow and every day into the future, I got furious. This insanity has to stop.

THE RENEWAL OF HOPE – Over the course of the afternoon, my anger turned to hopefulness as I started rethinking the hearing. I reflected on the questions by a couple of Senators about how NIH sets priorities. The childhood cancer community has been asking Congress about how NIH sets priorities. Over the past few days, our tweets, posts, and emails had asked Senators to urge NIH/NCI to make childhood cancer a priority. I was invigorated as I remembered the words of one Senator who said quite emphatically that the Congress has let the scientists of NIH determine priorities for research funding but that Congress needs evidence and assurance of the NIH priority setting process or Congress may change their stance on the blanket approval of the NIH process. NIH is supposed to provide information on how they establish priorities and re-balancing by December 2015.

One Senator said, “We need to fund things that we’ve never funded before, otherwise we’ll never get to the point of being successful” … remembering these words and the context in which they were said gave me great hope! Instead of the dejected feeling I had at the end of the hearing, I began to think, Congress is listening! I became reenergized thinking we have opened the window to making changes to the NIH budget. Perhaps this year it is not business as usual, that there is the possibility Congress will revisit and make legislative changes regarding the funding of childhood cancer research.

AdultKidsSelfie_edited-1THANK YOU FOR THE CALLS, EMAILS, TWEETS, POSTS – So many people joined in the selfie campaign to tweet, post, email, call and let their Senators know that childhood cancer research is underfunded by NIH/NCI and that childhood cancer research should be a priority. We think they heard you! We saw people tweeting regularly, encouraging followers to tweet, it was awesome. We heard from people that were galvanizing their friends, families, communities to get behind the issue, to email and call their Senators. We heard from one person that said, she had so many people calling her Senator’s office that she couldn’t get through herself. We know these efforts made a difference. We feel like we’ve made a dent in the armor of ambivalence surrounding childhood cancer research.

KEEP THE MOMENTUM GOING!

WHERE DO WE GO FROM HERE? – We’ve put a spotlight on the fact that kids are suffering and dying, that childhood cancer research needs to be a funding priority for NIH/NCI. We need to keep letting our Senators and Representatives know that the American people want childhood cancer research to be adequately funded in order to make curing childhood cancer a reality. Even though this will not end with the 2016 budget, Congress will make final funding decisions on the 2016 budget this summer and we must continue to let them know that Americans care about this issue. We need every voice, this is about all kids. Childhood cancer is on the rise and we need research now!

4%20X30Sign_edited-1Please continue to spread the awareness, ask everyone you know to call, write, email, tweet, facebook our Senators and Representatives on a regular basis and request a response. Organize events to send a message to your Senators and Representatives — post card campaigns, petitions in our Congressional districts and neighborhoods. Plan, organize, participate in events to support turning September gold. Help us make some noise on the Mall, come to CureFest in Washington, D.C. the weekend of September 19-20, 2015.

Together we can change this picture – make funding childhood cancer research a priority. Give kids the cures they deserve!

We never want any parent to hear the news we got when Declan was diagnosed, sorry there are no cures, treatments, protocols for this type of cancer. Rather, our dream is captured by the words of a mom who lost her little boy to cancer. Ben Towne’s mom said their dream is to find a cure for childhood cancer so that in the future you might hear, “The bad news is your child has cancer, the good news is we can cure it.”

Author: Donna Carroll Carmical 

Related Articles:

NIH_edited-1

NIH-Budget-Priorities

EmailExchange_edited-1Email Exchange

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What Not to Say When There is Nothing to Say

Team Captain:

This is a must read for all us who wish to offer words of comfort.

Originally posted on Life in Portsong:

Recently, I was asked for advice about how to respond to the parents of a child diagnosed with cancer. Let me say from the outset that I am a dubious source whose council typically causes some manner of regret. However, since I have stood on the receiving end of some pretty stupid comments over the past year, I do have a fair amount of expertise in this particular area.

First, THERE ARE NO MAGIC WORDS, so don’t try to find them. When one is standing at the outset of a long, twisted road that includes the potential mortality of their child, words simply cannot soothe. They can, however, aggravate. So I thought it might be helpful to look at some things that struck us the wrong way when we were facing our crisis.

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1. Do not equate anything you’ve gone through (or had a third cousin go through) with their…

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Posted in Cancer, Childhood Cancer, Pediatric Cancer, Rare Disease, Uncategorized | Leave a comment

NIH Budget Priorities

NIH_edited-1Each decade we lose more than 27,000 children to childhood cancer, another 120,000 suffer impacts of treatments including secondary cancers. This picture has not improved much over the past 30 years and it will continue to change only incrementally, if at all, over the next 30 years, unless we do something to demand that our government invest in childhood cancer research. Each year Congress appropriates BILLIONS of our tax dollars for research, only a tiny fraction of that money goes to childhood cancer research. We must join together and let Congress know this is wrong, our children deserve better, 1 in 285 children will be diagnosed with cancer and I personally don’t like those odds. We must tell Congress that childhood cancer research should be a PRIORITY in the federal budget process.

Screen Shot 2015-03-28 at 4.33.25 AMI came to this realization about the lack of childhood cancer research too late, but I want to change this story for the children who will be diagnosed today, tomorrow and in the future. Our lives were forever changed when our grandson was diagnosed with a rare form of pediatric cancer called AT/RT (atypical teratoid/rhabdoid tumor). Hearing that your child/grandchild has cancer is devastating, but hearing Doctor’s say that there are no treatments, no cures, no hope is incomprehensible. Doctor’s advised that we should take our beautiful Declan home, make him comfortable, and that he would be dead in a few weeks. There are no words to describe the shear horror of this moment and the days and weeks following. After a brave and courageous battle that included several brain surgeries, removal of his kidney, six weeks of proton radiation and many rounds of poisonous chemo, our sweet Declan lost his battle 8 days before his first birthday.

unaware_edited-1Like so many Americans, prior to Declan’s diagnosis, I was blissfully ignorant about the reality of childhood cancer and its impacts. For over 30 years, I gave to pediatric cancer organizations and assumed that they and the federal government were doing everything possible to find cures and bring the best possible treatments to our children. I was dead wrong. Our children are paying the price for this lack of awareness and lack of childhood cancer research funding. Children have no voice, no lobby, no money. Although kids represent 26% of the U.S. population, they can’t vote and it seems their needs fall to the bottom of the budget pile, especially where funding decisions are concerned. We must be their voice and demand change.

The Facts – Childhood cancer is the #1 disease related killer of children in the U.S.   The National Cancer Institute (NCI) says that childhood cancer is rare. They indicate that annually approximately 16,000 kids are diagnosed with pediatric cancer and that childhood cancer is on the rise. The American Cancer Society indicates that 1 in 285 children will be diagnosed with cancer, 1 in 5 children are terminal on diagnosis and 2 out of 3 children suffer life altering impacts as a result of treatments. According to NCI there are currently 380,000 survivors of childhood cancer in the U.S., they project this number will increase over time.

Impacts of treatment – Many children suffer life altering impacts of treatment. If they pass the 5 year survival rate, statistically they are considered cured. Yet, many children suffer long term impacts — secondary cancers, heart problems, infertility, learning disabilities, stunted growth, hearing problems and more. Eighteen percent of those that survive the first five years will die over the next 30 years. The increase in survivors, burden of disease, growing number of survivors, as well as number of deaths each year are a great cost to society in so many ways. Adult chemotherapy is proven to help cure cancer with many side effects. Due to the lack of research to develop pediatric protocols, many children are treated with chemotherapy approved for adult cancers and provided at a lower dose for kids. As StandUp2Cancer reports on their webpage – “While childhood cancer research often yields discoveries that benefit adults with cancer, the opposite is less common. Children are not simply smaller versions of adults, and childhood cancers are very different from the cancers that strike adults.”

NIHBudgetMany childhood cancers ignored – There are 16 major types of cancer and over 100 subtypes. Many of these childhood cancers, like DIPG, AT/RT, receive little to no funding for research. The National Cancer Institute (NCI) indicates that survival rates for a few childhood cancers like acute lymphoblastic leukemia (ALL) have improved dramatically over the past decades (there is still work to be done and we should not stop research for ALL because of improved statistics). Ironically this improved statistics for ALL is due to the investment in research!   Normally when NCI talks about improved statistics for childhood cancer and justifies their lack of investment, they make little mention of statistics regarding the deadly childhood cancers where for decades there has been little to no research. It seems to be acceptable that these deadly “rare” cancers where children are dying day after day, year after year, are offered little to no research funding.

Childhood Cancer Organizations – Ironically many pediatric cancer organizations raising funds for childhood cancer research have been started by families who have children that have battled cancer or have lost a child to pediatric cancer. These families have suddenly been thrust into the horrible world of pediatric cancer and become aware of the lack of funding for childhood cancer research. Their new reality pushes them to try and find ways to fund childhood cancer research that brings cures, save kids and give them hope. Families desperately want other kids to have cures denied to their own children. The thing is that even the largest organizations are raising tens of millions of dollars. Childhood cancer research needs a huge investment, an investment of billions that will bring cures to the table in years rather than decades. This can only be accomplished by putting our tax dollars to work for kids.

The National Institute of Health and the National Cancer Institute

  • The Budget – The National Institute of Health (NIH) has a $30 billion research budget and the National Cancer Institute (NCI) receives about $5 billion of this amount for cancer research. Childhood cancer research is sadly neglected as budget decisions are made by the NIH/NCI.
    • NIH has lamely defended their lack of investment by saying that research in adult cancers brings benefits to childhood cancers, childhood cancer is rare, there are insufficient resources to increase funding levels for childhood cancer research, if given more funding they would do more, and a myriad of other reasons from the brilliant researchers that make budget decisions.
    • Another statement I find incredibly ironic are statements by the Directors of NIH, Dr. Francis Collins, about the budget stressor put on scientific opportunities, the throwing away of “innovative, talented research proposals” and the “serious risk of losing the most important resource we have, which is this brain trust, the talent and creative energies of this generation of scientists.” I would counter that NIH might consider the incredible opportunities that are being missed in developing cures for childhood cancer because of the lack of investment in research. Many innovative childhood cancer projects are being thrown away because there is no money to fund childhood cancer research. Finally, we have no idea about the brain trust of the 2,700 children that lose their lives each and every year, year in and year out, decade after decade because we are again doing no research or very little on those deadly pediatric cancers that are killing our kids everyday. They too are an important resource.
  • NIH mention of Childhood Cancer is what’s rare – Childhood cancer research is not a line item in the NIH/NCI budget and is rarely mentioned, if at all. NCI indicates that in 2013, $185 million was for childhood cancer, this amount is not actually for childhood cancer research grants but calculated by percentages from grant projects that estimate their work might benefit childhood cancer.
  • NIH Request for $1Billion budget increase – In the 2016 budget, NIH has requested a billion dollar increase, bringing their budget to $31 billion, on average a 3% increase for every institute/center. Documents indicate that key investments are for Alzheimer’s research, Precision Medicine Initiative, Antimicrobial Resistance, AIDS, the Brain initiative, etc.   There is no mention of childhood cancer research in their budget request, it is not a priority for NIH.

Following the AIDS pandemonium in the 1980s, Congress provided billions for AIDs research. Dr. Francis Collins, the Director of NIH has testified that success with HIV/AIDS research “may enable us to envision the first AIDS free generation since the virus emerged more than 30 years ago.” This success would not have been possible without the substantial investment of dollars in AIDS research, an investment of tens of billions of taxpayer dollars over the last few decades. This is not to say the level of AIDS funding is wrong, but to demonstrate that a significant investment could bring real change to the childhood cancer picture. Since 1980 approximately 75,000 children in the U.S. alone NCIFundingChart2007_2013_edited-1have lost their lives to childhood cancer. It is unlikely that this picture will change unless significant federal research dollars are invested in childhood cancer research to develop pediatric drugs, cures, treatments and protocols.

As reported by The Atlantic in a January 2013 article, “there is not enough funding for childhood cancer, specifically. The National Cancer Institute, a government organization, provides funding for researchers, but only 10 percent of them can move forward with their findings due to budget cuts. Most of the financial support researchers receive is from philanthropists. In the meantime, research that could benefit children on an individual level stays in the lab, and doctors prescribe the same regimens that can be successful, but can also hurt the patient in several ways. Researchers say they are working hard to discover new theories and treatments, but they feel they are being held back.” The article goes on to quote, Dr. William Carroll, researcher and director of the cancer institute at New York University saying, “Ninety-six percent of grants (sic childhood cancer) don’t get funded … There’s no doubt there’s less funding available, and it’s driving people out of the field.”

I would love to see Congress provide that $1billion increase to the NIH 2016 budget, but legislate that childhood cancer research should be a priority and appropriate this $1billion increase for childhood cancer research.  Even a legislative requirement for NIH to provide a negligible decrease to every line item in the NIH budget for childhood cancer research as part of developing their operating plan could make significant funds available. Childhood cancer research deserves to be a national priority. No parent should have to hear the words, there has been no funding for research, there are no cures, treatments or protocols.

Screen Shot 2015-02-15 at 10.27.38 AMWhat Can You Do? We believe that awareness about the reality of childhood cancer and the lack of research will create an outcry that will result in action! The House and Senate sub-committees on the NIH 2016 budget are currently considering budget request for fiscal year 2016 which starts on October 1, 2015. We need to garner as much support as we can, via our pediatric cancer organizations, our social groups, church organizations, wherever and however we can to get people to call, email, tweet and write Congress and let them know that the American people want childhood cancer research to become a national priority. We know the increasing power of social media, Facebook campaigns and Twitter campaigns (use hashtags #nationalpriority and #contactcongress) to members of Congress have the potential to create impact. Postcard campaigns can be effective too.   Having volumes of people getting messages to representatives to let them know that we believe childhood cancer research is not getting sufficient funding will have an impact, especially if we don’t let up. Having a representative that sits on the committee can also create awareness and questions and result in action by the committee members. Here is a sample letter you can use to make your request:  Dear Representative or Senator

HOUSE SUB-COMMITTEE                                    SENATE SUB-COMMITTEE

Tom Cole, Oklahoma, Chairman                                     Jerry Moran Kansas

Mike Simpson, Idaho                                                        Richard C. Shelby Alabama

Steve Womack, Arkansas                                                 Thad Cochran Mississippi

Chuck Fleischmann, Tennessee                                      Lamar Alexander Tennessee

Andy Harris, MD, Maryland                                              Lindsey Graham South Carolina

Martha Roby, Alabama                                                     Mark Kirk Illinois

Charlie Dent, Pennsylvania                                              Bill Cassidy Louisiana

Scott Rigell, Virginia                                                         Shelley Moore Capito West Virginia

Rosa DeLauro, Connecticut, Ranking Member              James Lankford Oklahoma

Lucille Roybal-Allard, California                                       Democrat

Barbara Lee, California                                                     Richard J. Durbin Illinois

Chaka Fattah, Pennsylvania                                            Jack Reed Rhode Island

-====—————————————                                       Barbara Mikulski Maryland

—-=——————————-                                              Jeanne Shaheen New Hampshire

—===———————————-                                           Jeff Merkley Oregon

-===————————————                                           Brian Schatz Hawaii

===———————————-                                             Tammy Baldwin Wisconsin

If you are interested in the hearing schedule for the House and Senate appropriation budget deliberations, you can find detailed information by going to house.gov and senate.gov, selecting appropriation committee, selecting appropriation sub-committee for Labor, Health & Human Services, Education and Related Agencies. Here you can find information about committee members, hearing schedules and requests for witnesses and testimony for the record.

Together we can create change that will fund research and bring cures to kids.

Author: Donna Carroll Carmical

Posted in Cancer, Childhood Cancer, Pediatric Cancer, Rare Disease | Tagged , , , , , , , , , , , , , , , , , , , , , , , , , , , , , | 6 Comments

Texas Proud!

TexasProud_edited-1Annette Leslie, Executive Director of the Carson Leslie Foundation and a Four Square Clobbers Cancer blogger, has served on The Cancer Prevention and Research Institute of Screen Shot 2015-03-25 at 11.51.32 AMTexas (CPRIT) Childhood Cancer Advisory Council since 2010 and couldn’t be more Texas Proud!

Annette explained to 4-Square, “CPRIT has deeply listened to the pleas of our Childhood Cancer Advisory Council by moving to action and funding over $17,988,088 specifically for childhood cancer research.

This investment is a whopping 31% of the funds during this investment cycle that are directed toward childhood cancer research. And this is in addition to the $3,200,000 called, The Carson Leslie Research Grants for Pediatric Brain cancer that are now a year into their 3 year study.

Testof Morality_edited-1Leslie addressed CPRIT’s Oversight Committee recently stating, “Our committee is so grateful CPRIT has heeded our pleas and has moved to action to make childhood cancer a priority.  She challenged the Oversight committee to think about it this way,“After CPRIT has carefully invested the 3 billion they’re charged with and the books are closed, CPRIT will have a legacy; I hope that lAnnetteLeslieegacy will be the change for childhood cancer patients. not only in Texas but across our whole planet.”

Bill Rice, Chairman of CPRIT’s Oversight committee recently stated, “CPRIT’s grant support is intended to vastly accelerate progress for prevention and research of rare and hard-to-treat cancers, including pediatric and adolescent cancers. We hope the awards will help provide the impetus for Texas to become a global leader in childhood cancer research.”

Annette couldn’t be more Texas proud and humbly honored to be smack dab in the middle of directing millions and millons towards childhood cancer research.

TexasGrants_edited-1

If we had nine more states like Texas, we would match the entire amount that the National Cancer Institute is spending on Childhood Cancer research today!

Author: Joe Baber

bounceballauthor

Posted in Cancer, Childhood Cancer, Pediatric Cancer, Rare Disease | Tagged , , , , , , , , , , , | Leave a comment

#MoreThan4 NCI Email Exchange

EmailExchange_edited-1The National Cancer Institute recently sent a response to an email sent to them containing a selfie and a #MoreThan4 flyer. You may have received one. Below is the NCI email and our response.

Thank you for taking the time to reach out to us. We know far too many children suffer from cancer and we are fighting this terrible disease alongside you.

We also recognize that children are not just small adults and children with cancer need new approaches and tailored treatments that allow them to live long and healthy lives. In addition to our efforts to understand this disease so that we may better prevent and treat all cancers, NCI has a number of research initiatives specifically for childhood cancer. To learn more, please visit http://www.cancer.gov/researchandfunding/areas/childhood and follow us on Twitter.

Thank you again for your passion and commitment. We never lose sight of the patients we serve and your picture is a powerful reminder that there is still much work to be done.

Kelley Landynci-logo-english.gif
Director
Office of Advocacy Relations
National Cancer Institute
31 Center Drive, Suite 10A28
Bethesda, MD 20892
(301) 594-3194 | NCIadvocacy@nih.gov

Dear Ms. Landy,

Thank you for your response to our email where we expressed our displeasure with the NCI only devoting 4% to childhood cancer research.

First, we want to let you know that we are very appreciative of all of the efforts made daily of the hardworking men and women of the National Cancer Institute in the fight against all cancers.

Regarding childhood cancer:

We applaud the developments that are being generated under the TARGET program.

We support, and very much appreciate the great work that the Children’s Oncology Group does and we look forward to the benefits to be gained from the Pediatric MATCH program.

With nearly half of a million-childhood cancer survivors alive, and most suffering from late affects of their treatments, our community surely welcomes and supports the Childhood Cancer Survivor Study (CCSS).

Immunotherapy work that you are doing offers tremendous opportunities for our children! We are extremely excited and fully support this area. We appreciate the hard work your researchers are doing to get effective results as quickly as possible.

We want to see all the above programs operate at FULL potential. For years, childhood cancer has been the unintentional victim of budgets developed using such factors as best scientific merit, potential impact, and likelihood of success. What we are saying is this: With all the potential that has been unlocked in genomics, and the vast area of knowledge that has been obtained in the last few years, you are not applying enough financial resources to the above areas that will hasten impactful successes that will generate major increases in life years for children affected by cancer.

In short, we have not been satisfied with the 4% in the past several years when NCI used the same budget methodology as outlined above. We are not going to be satisfied in the future unless NCI either changes its priorities, or changes the way they determine how to finance their “best science.” Once that is done, we are certain that the result will be more than the 4% we have seen in the past.

4%20X30Sign_edited-1We look forward to the day we see change.  We look forward to real progress. We also look forward to a day where the increasing number of survivors will not have to fear living only to 35 or 40 years of age, but can look forward to living a full life rather than an abbreviated one because we did not invest properly in the opportunities that have been uncovered in the last few years since we mapped the human genome.

We respectfully remain unsatisfied,

#MoreThan4

How_edited-2

 

 

Posted in Cancer, Childhood Cancer, Pediatric Cancer, Rare Disease, Uncategorized | Tagged , , , , , , , , | 4 Comments

Father, Daughter, Date Night

FatherDaughter_edited-1Bella_RobThe other day I decided to take my 7 year old daughter, Bella, out on a date. I found a traveling circus that was in town and recalled how much I enjoyed the circus as a kid and thought it would be a great opportunity to make some memories with my little princess. On the way I asked her to choose what she wanted for dinner and that we were in a hurry so it couldn’t be somewhere that takes too long. She choose Dairy Queen for some unknown reason? Ya right, she couldn’t wait until after she ate her burger for her ice cream. Then I remembered since it was a date, I shouldn’t be scolding her about eating her ice cream first. Shortly after getting back in the car she asked “how did Caleb die?”

Bella_Caleb copyYou see, Bella doesn’t remember her only brother. Caleb was diagnosed with a rare form of Leukemia that sadly took him away just after his 4th birthday when Bella was only 9 months old. After the question sunk in, I asked her why she wanted to know how Caleb died. She said “because he was my brudder”. Touche’ I thought. Then I asked what she wanted to know? She knows he died of Leukemia. She said “what happened to him to make him die?” I asked do you mean what happened inside of his body? She exclaimed “Yes.”

Against my better judgement, and after probing with a few anatomy and physiology questions to see at what level I would be communicating, I reluctantly explained. “Caleb had Leukemia which is a blood cancer”. Bella said, “I know, his bones make his blood and his bones were making blood that was making him sick.” Then I explained “blood carries all the food and energy to all the cells in your body and that when your blood is sick it doesn’t do that.” “What happened was the blood wasn’t supplying the necessary stuff to keep the organs in his body going, so what happened was his system began to shut down and organs started failing and stopped working.” Bella wanted more answers “then what happened daddy?” I explained, “he was sleeping and breathing very shallow” and I demonstrated how he was just barely puffing with his breath, “and he just stopped Bellabreathing and was gone to heaven.” She said “that’s very sad and I wish I was older so I could remember my big brudder.”

I don’t share these personal thoughts and moments of my family to make you sad. Its just real. Childhood cancer has devastated my family but it did not win. We continue to fight this beast and someday hopefully in my lifetime, if not, certainly in my daughter’s lifetime, we will make a difference and bring better life saving treatments to families like ours. We do this in hope that future families will not have to have a conversation like this with their 7 year-old child. We had an amazing night at the circus with a keepsake photograph of a real life mermaid that was wearing a fake tail, ice cream and a story about big brudder Caleb.

Author: Rob Whan

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Dear CalebDearCalibHeader_edited-1

Posted in Cancer, Childhood Cancer, Pediatric Cancer, Rare Disease, Uncategorized | Tagged , , , | Leave a comment

Immunotherapy

immunotherapy_edited-2I am not sure how many people outside of the cancer research community know this, but it is well recognized that the human body is equipped to fight off cancer on its own. The same immune system that we have to fight infections should be able to fight off cancerous cells and tumors. While all cancers arise from an individual’s DNA damage and thus from our own cells, there are things about cancerous cells that are very unusual. Sometimes they express very high levels of certain types of proteins and other things which make  them stand out from normal cells, and explains why they grow and progress as they do. Our immune system should be able to recognize such cells as foreign invaders and zap them before they become a problem. There is actually a theory, one which I happen to believe, that everybody develops cancer at various points in our lifetime, but that the tumors are eradicated by our immune system before we even know they are there. We all develop DNA damage, every single day, and so it makes sense that cancers come and go in uneventful fashion. Immune system activity is also a very logical explanation for why some patients will have an unexplained “spontaneous remission” in the absence of treatment. The immune system is likely behind such miracles.

immunesys_edited-1But 1 in 2 men, 1 in 3 women, and 1 in 285 children will get diagnosed with cancer, so we know that the immune system is not bullet proof. We know that patients who are in some way immunocompromised, such as patients who are undergoing immunosuppressive therapy because of an organ transplant or autoimmune condition and those with immunosuppressive conditions such as HIV/AIDS, have very elevated risks of developing cancer simply because they are immunosuppressed. But why does someone who is otherwise perfectly healthy, with no predisposition for cancer, get cancer? A deteriorating immune system with aging is thought to play a role in why cancer is so common in elderly patients, but to what extent does the immature immune system of childhood play a role in pediatric cancers? There are no clear answers to these questions, and the topic goes way beyond that of the immune system. However, we know more than we used to when it comes to the human immune system. Medical research has uncovered something really sneaky about tumors: that some tumors develop in ways that in essence get the immune system to look the other way. Unfortunately or fortunately, depending on your perspective, we now know that tumors are sometimes protected from the immune system via checkpoints like “PD1” (stands for programmed death-1; you may have heard of it, it is big news in cancer right now), leading to a new class of immunotherapy drugs called “checkpoint inhibitors”. Think of PD1 and other checkpoints (there are others, like CTLA-4, which was described before PD1) as being a brake to the immune system, telling the T-cells of our immune system to leave the cancer alone. We have these cellular checkpoints in our body to prevent our body from attacking itself (as happens in autoimmune diseases), but they are also offering protection to tumors. These new checkpoint inhibitor drugs are leading to major breakthroughs in advanced/metastatic melanoma, an aggressive skin cancer that had typically been considered a death sentence, usually within 1 year of diagnosis. In September 2014, the first PD1 checkpoint inhibitor was approved in the United States for advanced melanoma (Merck’s Keytruda® [generic is pembrolizumab so just call it Keytruda]); also another PD-1 agent, Bristol-Myers Squibb’s nivolumab (Opdivo®) has  been approved for advanced melanoma in Japan and was just approved for use in the US by the FDA. Other similar agents are aggressively being explored as single-agent therapy and combination therapy melanoma and various other malignancies. The approval of Keytruda® was based on a 24% response rate in this heavily pretreated population in which most patients had stage IV metastatic disease, but even EveryPharmmore noteworthy was that the responses tended to be durable in patients who responded. PD1 agents are not without side effects but are generally more tolerable than chemotherapy, producing mostly low-grade toxicity and not the types of things seen with chemotherapy. This is a big breakthrough for those whom chemotherapy has offered little hope but lots of toxicity.

You might be thinking, why the upfront focus on advanced and metastatic melanoma for these new immunotherapies? Without getting into specifics regarding the biology of melanoma, there are things about melanoma that make it a very good target for immunotherapy, and is one of the cancers for which there has been some success with past attempts with older immunotherapy approaches, known as cytokines (the interleukins and interferons). So starting with advanced melanoma makes a whole lot of sense, from a biologic perspective. Anti-PD1 therapy is still in its infancy overall, but it is important for those reading this to know that pediatric studies of these PD1 agents are planned, per publically available information on the FDA’s website. The manufacturers of these products all recognize the potential of these agents for kids, and studies will be coming. Here is a link for more information; a heads up that there is a lot of info within the documents and slides:  FDA Link

But the subject of PD1 immunotherapy will be continued at a later time, as it was not PD1 that prompted me to write this post. It is something else, called CAR T-cell therapy, for which pediatric data are already available.

Before I get into the data that have come to light for CAR T-cell immunotherapy for childhood leukemia, I think it is important to jump back and offer a little bit of context. I am an oncology writer who works as a contractor for the pharmaceutical/biotech industry. I have been writing about cancer drugs since 1998, and have witnessed the evolution of cancer therapy and the ups and downs with things like immunotherapy. In the early 2000s, cancer immunotherapy was the greatest thing since sliced bread. One of the big focus areas was therapeutic cancer vaccines. Unlike preventative vaccines (including those that are aimed at preventing cancer, like Gardasil), therapeutic cancer vaccines are designed to eradicate an existing cancer, by boosting the immune system as discussed earlier. In the early 2000s, several different therapeutic cancer vaccines were under development, including some complex personalized approaches that were based on the patient’s own unique cancer cells and others that were designed to be more “out-of-the-box”. Several showed promising early activity, but around the same time several different ones failed advanced-stage clinical trials. The populations that were mainly being studied were very heavily pretreated poor-prognosis cancers, like metastatic melanoma and advanced pancreatic cancer. Although many were speculating that these might not have been the best populations, that better results may have been possible if these vaccines had been tried earlier in the disease or in different types of tumors, many companies jumped off of the immunotherapy bandwagon entirely. Some small biotech companies, which were very dedicated to this therapeutic cancer vaccine technology, were forced to close their doors. I watched it happen. Some of these were my clients. My thought at that time was “Nooooooooo, don’t do it, stick with immunotherapy, we are on the right track but wrong train”. The concept of immunotherapy just makes sense on so many levels, and holds such promise for true cancer cures. But failed trials are an automatic death sentence for products, especially for small companies. Trying to manufacturer personalized cancer vaccines, a new and very complex and expensive process, just isn’t possible without money TreatrmentChart_edited-1and lots of it.

So starting around the mid-2000s, things got very quiet on the immunotherapy front. Cancer became a bigger and bigger area for both large and small pharmaceutical/ biotech companies, but the focus wasn’t immunotherapy but something called targeted therapy. You may have heard terms like small-molecule inhibitors, molecularly targeted agents, tyrosine kinase inhibitors (TKIs), monoclonal antibodies — these terms all fall under the umbrella of the novel concept of targeting the ability of cancers to grow and proliferate by targeting cell survival pathways, rather than killing them directly   via chemotherapy.

Chemotherapy basically works by causing damage to DNA, killing the tumors via this DNA damage (but unfortunately also damaging healthy DNA in the process). On the other hand, targeted agents work by indirect killing of the tumor, not by damaging its DNA but more like starving it by taking away something(s) that it should need to survive.  As I mentioned earlier, cancers express high levels of things that normal cells express at low levels, so modern medicine has been able to exploit those differences and develop these new “targeted agents” that represent “personalized medicine”. However, the term “targeted” is kind of a misnomer. For example, there is a modern class of drugs called EGFR inhibitors, which act against tumors that have identifiable mutations in this thing called EGFR. EGFR mutations commonly occur in certain patients including non-smokers with lung cancer, and these EGFR-targeted agents are being used as a substitute for chemotherapy in these patients. These agents are way more effective than chemotherapy in these patients, assuming that the patient has a tumor that carries an EGFR mutation (this is where genomic profiling comes into play, to be discussed another time),  and more tolerable to

WatchT-Cells_edited-2boot. However, they are not devoid of toxicity. For example, EGFR is highly expressed in normal skin, so these “targeted agents” can lead to skin toxicity, which can be severe and complicate treatment. So the term targeted does not mean that the activity of these drugs is confined to the tumor per se. At the same time, however, these drugs are NOT chemotherapy and generally do not produce the types of severe toxicity commonly seen with chemotherapy, and many of them are available as once-daily oral therapy which is a welcome convenience. Overall, molecular targeted therapy is a good thing and represents a huge leap forward in cancer therapy. It has revolutionized the entire approach to treatment, allowing for a personalized approach that can lead to better efficacy and better tolerability. However, a big barrier to targeted therapy is tumor resistance, either at the start of treatment or after a while of being on treatment. As I said earlier, tumors are sneaky. If you block a cell survival pathway (like EGFR or something else, there are many!), tumors can find ways to eventually adapt and grow without that particular pathway. Tumor resistance is considered inevitable for most patients treated with targeted therapy, although some patients will continue to respond over time. Researchers are still trying to figure out ways to stop this resistance, such as by combining agents. Sometimes this seems to work; other times, not so much. Always remember, every single solitary tumor is unique, being derived by our own DNA. This explains why patient experiences and outcomes vary so much.

Meanwhile, as the pharmaceutical/biotech progress was being made with targeted agents, the field of immunotherapy was not entirely dead, as illustrated by the PD1 example earlier. At the University of Pennsylvania, researchers were focusing on an entirely different type of cancer immunotherapy, termed CAR (chimeric antigen receptor) T-cell therapy. Our immune system is composed of a mixture of T-cells and B-cells, which act in different ways to fight infections and other invaders. The concept here is that you take T-cells from a patient, send them to a lab and use a virus to genetically program the native T-cells to target cancer cells, and re-inject them into the patient in hopes that these “serial killer cells” will do their thing and knock off the cancer. On a fascinating note, the reprogramming is taking place via use of the HIV virus, which is a very effective means of delivering information to T-cells. The HIV virus is of course deactivated so that it cannot 

Screen Shot 2014-12-19 at 2.10.08 PMcause disease. As it turns out, these serial killer cells are actually doing their thing. Not only are we seeing positive results, but a focus to date has been treatment-refractory pediatric acute lymphoblastic leukemia (ALL)!! Check out this video, “Fire With Fire”, but be prepared to be blown away:

The experiences with CAR T-cell therapy have been published in the New England Journal of Medicine, based on a study conducted at the Children’s Hospital of Philadelphia (CHOP).1,2 The first report described the first 2 patients achieving complete remission, whereas the most recent one reported a 90% complete response rate among 30 children or adults with relapsed or refractory ALL, with some durable remissions up to 24 months. Recently, updated data were reported at the Annual Meeting of the American Society of Hematology, with a 92% complete remission rate among 39 children and young adults. Results like this, in such heavily pretreated patients with no other treatment options and such a poor prognosis, are pretty much unprecedented. CAR T-cell therapy is not without toxicity issues. Because you are boosting the immune system so strongly, all patients experience something called cytokine-release syndrome, which can lead to life-threatening complications. The “serial TCellVideo2_edited-1killer cells” also kill healthy B-cells in the process, requiring patients to receive B-cell replacement therapy indefinitely. Also, this particular therapy is targeted against CD19, a certain tumor marker that is not expressed in all ALL. Finally, as explained by Dr. Stephen Grupp from CHOP in the video to the right, patients who are so heavily pretreated have a risk of not having enough T-cells to do this type of therapy. It begs the question, of just how early something like this could be tried? And what will the long-term results be, in terms of “immunity” against recurrence? Answers to questions like that will take further studies, and a multicenter study for pediatric ALL is already planned (yay!). TCellVideo_edited-1Also, because of the excitement around this product, it really resonated with the pharmaceutical/biotech industry, with the University of Pennsylvania’s technology being licensed by Novartis Pharmaceuticals. Novartis is working on building a manufacturing facility to facilitate the expansion of this therapy within the United States and abroad. Other companies have ventured into the CAR T-cell space. This is a huge deal and speaks volumes to the promise for this technology, as this type of personalized product is not the typical type of treatment for large companies to take on. This type of technology is very complex and requires a lot of money and patience, to keep it alive. Research hospitals, even major ones like UPenn/CHOP, certainly cannot go down this road alone, so it is a good thing for everyone that pharma came knocking. It took some time to generate interest. It took groundbreaking results, but CAR T-cell therapy found the home it needs to take it to the next level.

So right now, everybody in the cancer world is talking about immunotherapy as the greatest thing since sliced bread. I tend to agree. We have a long road ahead though. The experience with CAR T-cell therapy has been so positive so far, but responses like that will not be achieved for every patient in every trial. Long-term efficacy and safety data are critical, but that takes time. Nonetheless–immunotherapy is BACK and seemingly here to stay, and I am personally so excited to see what’s next. I follow this area of immunotherapy very closely, not just for children but for adults, and I will be sure to keep you posted too!

Author: Laurie Orloski

References:

  1. Maude SL, Frey N, Shaw PA, et al. Chimeric antigen receptor T cells for sustained remissions in leukemia. N Engl J Med. 2014 Oct 16;371(16):1507-17.
  2. Grupp SA, Kalos M, Barrett D, et al. Chimeric antigen receptor-modified T cells for acute lymphoid leukemia. N Engl J Med. 2013 Apr 18;368(16):1509-18.

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Five year cure, …really?

5yearcure_edited-1Five-year relative survival rates describe the percentage of patients with cancer that are alive five years after their disease is diagnosed. Use of 5-year survival statistics is more useful in aggressive cancers that have a shorter life expectancy following diagnosis (such as lung or pancreatic cancer) and less useful in cases with a long life expectancy such as prostate cancer.

5yearChart_edited-1When a patient achieves that magical 5 year survival mark, everyone celebrates the milestone and they are considered by most people to be “cured.” That is not necessarily accurate. What it means is exactly what it implies: They have survived five years since being diagnosed, nothing more. The average age at diagnosis of cancer is 6 for children and 66 for adults. While the chart at the right shows great progress in five year survival rates in childhood cancer from one period to another, it is only comparing five year survival rates, nothing more. While it gives some comfort that we appear to be making good progress, it does not really measure survival beyond a five year period. Using a standard five year survival rate for cancer will take an adult diagnosed with cancer up to age 71, but it only takes a child up to age 11. Has the child really “survived” and beaten cancer? Sadly, the answer for a large number of children is, “No.” We need a more accurate “survival” measurement for children. Kids cancer is not the same as adult cancer and survival for children must last much, much longer than just five years.  As we do today, using a five year survival rate for childhood cancer grossly discounts the pitfalls that lay ahead for up to 95% of the survivors. When it is communicated in a way that  others outside of the childhood cancer community can fully understand the long term impact cancer has on children, only then will we finally make childhood cancer a real priority. Let’s stop hiding from the reality of childhood cancer by using  unrealistic five year “survival” rates.  True progress in surviving childhood cancer can not be measured by only looking at the five year period from diagnoses. Today, advocates and medical professionals frequently cite 83% as being  the average survival rate for children.  In reality, the 30 year survival rate is only 65% overall.  A long term survival rate such as this gives a much different and more realistic perspective to the general public who we rely on to help fund research. We’d love to see the American Cancer Society or even the National Cancer Institute to step up and address the need for looking at a more realistic method of measuring survival for children. 

The following article was written by CJ Colton in February 2013. Her son is still fighting cancer today and it appears there is no end in sight. It highlights what a child goes through just to get to the five year “survival” point. Studies have proven that survivors of childhood cancer actually are fighting effects of their “life-saving” treatments for the remainder of their lives:

Five Years Is Not Enough

This article is about how children with cancer are surviving longer. However it also points out the lack of research and funding for pediatric cancer. What the article does not say is that a major number of the children do not live to be adults. 

I am not angry and I am not bitter. I refuse to become that way. Through our Faith we stay strong. However, our children are dying. How can you tell a parent of a 1 year old your child may live for another 5 years and that is considered good, or a mother of a 6 year old your child has 5 years. Both these children would die at a young age. These children would also go through horrible medical treatment to keep them alive. Five years is NOT ENOUGH for our children!

I know this because my son, Tony, was diagnosed with Clear Cell Sarcoma of the kidney at age 11 in July 2011. He finished treatment in February 2012. He lost his hair, had multiple surgeries, had a kidney removed, and was given medication that caused him to be so sick he lost 25 pounds. He was given radiation treatments, he hurt all the time. It hurt to walk or play and he never felt like eating because he was nauseous all the time. He was so sick. He smiled though most days. Most of the children do smile most of the time.

For one year after treatment he still had to go to the clinic numerous times and have radio- active or nuclear meds put in his body along with numerous scans, MRIs, and CTs. He made it one year with no sign of cancer. On February 8th 2013 we were told that Tony had a mass where his kidney use to be. On February 12 he had a biopsy of the mass. His liver bled during this biopsy and he was put in the hospital. On February 13 we were told Tony’s cancer had returned. We were referred to the National Institute of Health for a Phase 1 treatment called Ipilimumab. This treatment will make his T-cells over work to try and attack cancer cells. The effects of this medication are bad autoimmune side effects. This, we hope is his cure. This medication has been used on adults, but not many children. He will be the 8th child to receive this medication at NIH and the first child with Clear Cell Sarcoma to receive it. He may have to have a chemo that is called ICE next.

He will have treatment every 21 days at NIH which means we have to fly to Bethesda, Maryland for treatment. In between treatment he goes to All Children’s hospital in St Petersburg, FL several times during the week. His only complaint since re-diagnosed is he wants to have his port back. You see he has had over 20 blood draws and/or IV’s since February 8th.

Last Friday, March 8th 2013, while we were at clinic for a blood draw and a physical check to see about side effects I listened to what was going on there. I have listened in the past, but this day I realized something, it sounded like we were hearing a torture chamber of children and babies. I heard babies screaming, I heard toddlers yelling and saying “help me”, I heard school age children screaming and yelling “please don’t hurt me.” I heard a child yell “Mommy let me go, help!” I saw children trying to run away and I saw children sobbing. These children were getting port accessed, blood draws, large needles stuck in both their legs at the same time for chemo, and finger pricks to check counts. These are just a few of things these children and babies go through that have cancer. These brave and strong children have to go through this over and over and they just might survive past 5 years, or they might not. If they do survive they will have long term side effects from this treatment.

I will say again I am not angry or bitter. I hurt though, a hurt I could never imagine feeling to my soul. This hurt is out of fear for the life of my son, but also for many other children we have met going through treatment. The pain and suffering these children go through is tremendous and you witness it over and over.

As a nation we need to make sure our children receive the best possible research and treatment for pediatric cancer. It is our responsibility. Cancer is the number one disease that kills our children. We need to save our children for longer than 5 years.
I am just one mother with one child.

fiveyearcurerate_edited-1There are hundreds of foundations out there trying to bring awareness to childhood cancer and raise research money. We all need to unite as one strong group to fight for our children. Numbers bring strength.

What these kids go through to survive the 5 years is horrible and a lot less make it much further than 5 years. Many children do not make it to 5 years. Why would they think a 5 year survival is enough for a child? 5 Years IS NOT ENOUGH!

Author: CJ Colton

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My Calling

MyCalling_edited-1I came into the childhood cancer community in 2013 as a member of the “general public”, having no personal connection to a child with cancer. My journey is different but not without appreciation for what cancer is and how devastating the journey can be, not only for the patient but also those who serve the role as primary caregiver. My mom always said that losing one’s parents makes you feel like an orphan, and she was right on point there. The pain of losing 2 parents by age 35 may pale in comparison to the pain of losing a child, but the pain and loss are enormous nonetheless. My mother was the second to go, right as I was starting my own family, after a 9-month battle with an exceptionally rare carcinosarcoma of the uterus. They are so rare that most oncologists will never see a case. When they do occur, they tend to occur in women with certain risk factors. My mom had none of those risk factors. Many cancers are actually explainable; the unexplainable, random ones (which include most pediatric tumors but also some unusual adult tumors) are just flat-out baffling. I just have to keep reminding myself that sometimes things happen for a reason.

When my mother was suddenly sidelined by cancer, my relationship with her transitioned from being a daughter to being a daughter-cologist, aka patient advocate. I have a professional background in cancer, so I took care of almost everything—getting second opinions, getting discharged from the hospital at the right time, getting her nausea/vomiting and pain medications adjusted, making decisions on an investigational treatment, asking questions, asking more questions. My mom didn’t have to worry about a thing, and she liked it that way. I let her make her own decisions; believe me though, she did not want to manage the rest. She was very sick from the time of diagnosis. When she would start getting upset about the possibility of dying, I would just transition into some sort of scientific discussion about tumor biology and data, yada yada. My intent was to get her mind into thinking “wow, how annoying is my daughter, why won’t she shut up” rather than thinking about dying. There were times in her journey when things weren’t going

Literally 1 single week from this photo op, perfectly healthy Ma called crying.  Blood work came back.  Her "definitely benign" polyp wasn't looking so innocent.  She needed to see an oncologist STAT, and so the journey began...

Literally 1 single week from this photo op, perfectly healthy Ma called crying. Blood work came back. Her “definitely benign” polyp wasn’t looking so innocent. She needed to see an oncologist STAT, and so the journey began…

right, when answers to questions weren’t making sense. And I knew enough to know that she was in big trouble. So I would speak up, I would ask questions that nobody would ever think to ask—very difficult questions. I fought for her life in a way that nobody else on the planet would. “I wish I had a daughter like that”, I would hear her nurses say, with genuine awe that I would jump through so many hoops for my mom despite having a baby at home. Of course I would, she was my mother and only living parent. She needed to be saved. In the end though, her tumor was too aggressive, there really wasn’t anything that could have been done to save her. At the same time, I left that cancer world feeling very disgusted, not only that she died but how she died. I could go on and on but I won’t, other than to say that sometimes things take a life of their own; sometimes things happen that are simply out of one’s control. Sometimes doing the right things isn’t good enough. Sometimes mistakes are made that cannot be undone. If she had had a different tumor, maybe she would have been OK, or maybe not.

When I came out of that fog and then the fog of having my second daughter, I felt this unrelenting pull to get involved in cancer awareness and advocacy—stemming from my ordeal with my mom. I kept thinking about children, but I had no personal experience to childhood cancer so wasn’t sure if/how to get involved. Back in 1997, I had spent 6 weeks at St. Jude as part of my oncology pharmacy training. I remember that most of the kids were extremely sick, being beaten down by their treatments, but that most were considered curable. Two children passed away with solid tumors during my 6-week stay. Those losses were devastating. They were the exception and not the rule. In a lot of adult cancers, certain types, death was the rule and not the exception (still is, in some cases).

From 1997-2013, I had fallen out of touch with pediatric cancer research, as my career wound up being focused on adult oncology; however, I had assumed that things must be really good now. In the year 2014, the era of novel targeted agents that began about a decade earlier, those exceptions must be even rarer. I thought that kids must be really benefiting from the numerous new biologic and targeted drugs that had entered the marketplace since 1998, from the time when Herceptin first came onto the scene for breast cancer. I knew that many of these targeted drugs had applicability to numerous cancers and was sure that they were being aggressively studied in childhood cancer.

Then I did some quick on-line searches, to gauge the state of pediatric cancer treatment, and found that nothing had changed at all since 1998. These new cancer drugs that I had been writing about for adults had not been making their way to children. Those kids whose bodies were being literally annihilated with chemotherapy in 1997, many undergoing bone marrow or stem cell transplants, were likely suffering some sort of long-term complications. Those very same regimens continued to be used today, in many cases. In the year 2014, there were still pediatric tumors that are regarded as terminal on diagnosis. Not only was my industry, the pharmaceutical industry, paying little to no attention to childhood cancer, but the American Cancer Society and the National Cancer Institute weren’t picking up the slack in any way shape or form. It takes a lot to shock me, but I was floored. How, in the year 2014, were treatments for childhood cancer stuck in circa-1970s and 1980s? How did this happen, and who let this happen?

For me, there was no going back. If I was going to give up time that I don’t even have to volunteer in the cancer world, I was going to devote it to raising awareness and research CAC2Join2_edited-1funding for childhood cancer.

What was I going to do exactly? Well, I had some big ideas but they were not doable on my own. Luckily I made my way to the Coalition Against Childhood cancer (CAC2), which was just in the process of forming. One person told me to talk to this person, that person told me to connect with another person and so on.

I started writing blogs here, from the perspective of the “informed general public”, but also made time to travel to the CAC2 Annual Summit, where I got to meet some of my comrades for the very first time. There was no way that I was going to miss the 2014 Congressional Childhood Cancer Caucus, and I made plans to attend CureFest the second year in the row.

WhiteHouse_LogoThen, despite initially deeming it as spam, I got an e-mail invite from the White House for a closed-door meeting with pediatric cancer advocates. I seriously thought it was spam at first, but it seemed too specific. I thought those hackers were really smart. Then, after deeming it not spam, I just assumed it was some sort of mistake—but no, it was intended for me. I was humbled to have even made it onto the invite list and was not going to miss it, even though it would require some complicated travel logistics.LaurieCapital_edited-1

I won’t go into details now, about what I learned in those meetings in DC. I’ll write about those things later. I will just say that I feel a genuine sense of optimism that good things are going to come for children with cancer. That while there is a ton of work to be done, the door (actually multiple doors, those of pharma, the NCI, and the White House) has been kicked open—now as advocates, we need to open it wider and keep it wide open.

Anyhow, when I was at the CAC2 meeting and sitting in the Caucus and White House, I will admit to getting a tad emotional. The misty-eyed kind of emotion, and it is happening to me right now gosh darn it. I always say that I came into this community without a connection to a child with cancer, but I had a connection to another patient: my mom. In the time it takes to have a baby, her life was taken away in cruel fashion. Her journey, her KidsKeyboardGoldawful journey, took me to a place where I am speaking up for kids with cancer. Sometimes things happen for a reason, right? The reality is, if I had not lost my mom and in the way that I did, I would not be in the childhood cancer community right now. It took a really weird tumor and a really unusual journey to get me here. In the end, I no longer consider myself a member of the “general public” but instead call myself a “childhood cancer advocate”. The last conversation I had with my mom, we joked about how her doctors hated me (some did not like questions), how she was proud of me for that, and how I missed my calling as a patient advocate. As it turns out, I did not miss my calling after all.

Author: Laurie Orloski

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Fixing the Faults

FixingFaults_edited-1This summer I wrote a raw piece entitled “The Fault in Our Systems”. Our family’s efforts to get Nathalie access to immunotherapy treatment (available to adults) seemed to be going nowhere. The inequities were everywhere and the playing field was tilted against us. In high school on the Big Island, I dreaded playing travel soccer games at Hilo High. The soccer field was on a laughable slope. Thank goodness for halftime field switches. Nathalie is still playing up hill – there is no other playing pitch with ASPS, but gaining access to a new therapy is significant.

facebook-pictureLast weekend I was able to take a look under the hood at some of those faulty systems in the world of childhood cancer. Our family traveled to Washington D.C. with the express intent of leveling playing fields- one determined step at a time. Vicki took the redeye flight ahead of us, landed in DC and lasered to the Capitol for the “Hill Day for Medical Research”. She was able to meet with a number of lawmakers, urging support of policies to increase research funding, incentivize pediatric cancer drug development, and remove barriers to access.

The girls and I arrived in the afternoon, quickly changed into DC level attire and joined Vicki at a “Golden Toast: Reception for Congressmen McCaul (R) and Van Hollen (D) Co-Chairs of the Childhood Cancer Caucus“ It was the movers and shakers of pediatric cancer NathanReceptadvocacy. A certain pharmaceutical pediatric oncologist was there, eager to meet Nathalie. Our first introduction had been long ago when he had been on the other side of the phone letting our oncologist know that the the immunotherapy drug would not be available at this time. Through many phone calls and communications we gained a mutual respect, and even as I wished the answer for Nathalie would change, I supported his efforts to bring treatments for all pediatric patients sooner. Today we were both in DC as partners with a similar goal.

He announced that his company had enacted some wide policy changes in the default age limit for their future clinical trials. Unless specifically necessary, twelve would be the new age limit on the “adult” trials. The future pediatric trial of PDL1 will include all tumor types – also a nod to rare cancers like Nathalie’s.

At one point a few months back, Nathalie remarked that it can be lonely in front, pushing for something new, something you believe is right. I told her, “It always is.” I am thankful NathanAutoriumwe get to see this door widen for others.

So many other scenes float past – sitting as a family in the richly appointed congressional auditorium listening to the head of NIH, MD Anderson, Johnson and Johnson give their take on the challenges of pediatric cancer, compassionate use, immunotherapies, and science advancing faster than access. So much had been part of our story – I bit my tongue on the Q & A’s till I couldn’t anymore. The CEO of St. Baldrick’s turned around to thank Nathalie for the trail she had blazed and the reporter who took her story national was typing away in the row behind. The seats were filled with the names of many we’d met from afar on Nathalie’s journey.

Later that day I learned of the triplicate security measures involved in attending a meeting at the White House. The briefing/discussion for Pediatric Cancer Advocates was held in the Indian Treaty Room. I wasn’t in the mood to make treaties (in fact if the suit wasn’t required, I’d have preferred one of StillBrave.org’s “Keep Calm, My A$$! Cure Childhood Cancer” T-shirts) but I sat quietly down in the front row a few feet from the government officials best poised to advance childhood cancer efforts.

RobVarmusDr. Harold Varmus, director of the National Cancer Institute shared how budget instability was turning away young researchers from pursuing funding. A new pediatric MATCH Trial was set to begin, using genetic analysis of non-responding tumors to match them with existing market and developmental medicines – which hopefully pharma would make available as they have with the adult version of the MATCH trial. He was of course pressed on the 4% of the total cancer budget devoted to pediatric cancers. I took some solace in the words of Dr. Gregory Reaman in charge of the FDA’s oncology drug development. He is a former chairman of the Children’s Oncology Group and is very well-placed to question pointless barriers. I overheard the phrase “It’s time to stop “protecting” children from research, and use research to protect them.” What was historic about this meeting was that the administration had made available just about every government official connected to pediatric cancer and had given a forum for the diverse collection of pediatric cancer advocates to have a conversation. There is so much to be done. The many drums are starting to find the same beat.

IMG_4700There were lighter scenes as well, accidentally taking the “Senators Only” elevator, enjoying the “schmoozing” food at receptions, taking advantage of the Smithsonians sandwiched between our business at the Capitol and White House, screening the animated film addressing children’s cancer “Henry and Me” and chatting with the directors, bumping into one of Nathalie’s former NIH doctors who demanded a pic for the team there rooting Traller_HenryAndMefor her, debating the merits of “half smokes” versus normal hot dogs, and participating in the most scenic fundraising walk ever (“so head down to the capitol, turn around The Mall and then circle around the Washington Monument”). We took cabs among the alphabet and number streets and were inspired by the families and foundations that have been in this advocacy effort for years watching it slowly grow. Humbled and inspired was the theme.

Childhood cancer is the number one disease killer of children. It’s time to stop calling it rare. I used to think the lack of progress in pediatric cancers (other than blood cancers) was due to the tremendous complexity of the task. Now after I’ve looked under the hood, I know that it is really no surprise based on the paltry stream of resources. Imagine large NASCAR vehicles representing the most prevalent adult cancers – their hoods are plastered with corporate sponsors such as Merck and Bristol Myers Squibb. Now imagine NathanCar_edited-1a small car with only two small decals, that of the National Cancer Institute and Children’s Oncology Group. Call me crazy but I feel if those big companies want to keep racing to the profit line on the adult cancers, perhaps they can all agree to a small share of sponsorship of the pediatric cancer car that represents future dreams. You won’t find a company that doesn’t profess to be patient first, let’s stop defining that patient by age. Let’s make sure our government removes the speed bumps for the companies with the character and courage to join in. I saw engagement by Genentech, GlaxoSmithKline and Johnson and Johnson this weekend – Know someone with a company that was missing? Encourage them to get in the game. I can personally guarantee that the Traller family will keep pushing up hill. Sometimes it’s the difficult things are the most worth doing.

Author: Nathan Traller

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A Pair of Shoes

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One day in September, after my wife and I visited the Hart building, we met up with Ellyn Miller on a beautiful blue-sky morning in front of the Cannon House Building in Washington, DC. Ellyn had walked there from Union Station. We were there to lobby for medical research funds. During the day, we walked from the Cannon to the Rayburn, back to the Cannon, then to the Rayburn again and on to the Russell and back to the Cannon, not to mention all the walking inside those huge buildings that house our Congressional offices.  For each word we uttered on behalf of medical research, we must have spent a hundred steps walking from one place to another. By that afternoon, we all talked about our shoes and how much they hurt our feet. About a month later, I found this piece and then I realized how much Ellyn’s shoes must hurt her every day. This is dedicated to her and to others who have had a child taken by cancer.
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Hope

hopeHeader_edited-1From his perspective, my 10 year-old grandson Conor has always had a somewhat normal ConorSkate_edited-1childhood. He lives in California, is good in school, a cool blond-headed surfer dude and a wild and radical skateboarder. In fact, I am amazed when I watch him on his board as he navigates a particular skateboard park in Coronado popping in and out of the deep pools “getting air” and landing safely on the rim. This has been the life his parents wanted for him and even prayed for him to have about nine years ago when he was diagnosed with high-risk neuroblastoma.

Conor’s ordinary life started to unravel about a year ago when his natural curiosity got the best of him. He picked up a Nicholas Conor Institute leaflet on his mom’s desk and asked, “Hey mom, why are they using my name?” Conor didn’t remember, and my daughter Beth Anne and her husband Nick never really let him know that they almost lost him to cancer. They wanted him to have a normal life and did everything they could to make that happen, but now Conor was getting curious and really noticing what his mom was working on.

When Conor asked, Beth Anne told him in a very gentle manner and as time passed, sheConorRibbons gave him more and more details about those early years. He never liked the idea of being a cancer survivor and avoided any and all conversations outside of the family. He did not want to be considered “special.” He felt awkward. He really did not care for the reactions he got from strangers when they found out he was a survivor.

One day in school, during a health lesson with a large group of kids, the instructor asked if any one there had cancer or knew someone that did. Conor could not raise his hand. He did not want to be different from the others. Across the room, a boy in his class stood up and said he had neuroblastoma when he was four and talked about it. BINGO! Conor connected with his new best friend who, like him, was a cancer survivor. Afterwards they compared port and surgery scars and so forth. They are very close friends today and the relationship helped Conor be able to talk about cancer.

Beth Anne decided to bring Conor to Childhood Cancer Awareness Week in September  and involve him in all the activities. It was time to immerse him into the world of childhood cancer. She brought Conor and his sisters, Grace and Cami to Washington, DC where my wife Ellie and I joined up with them. We made a week of participating in cancer 2014-09-24 07.52.52 pmrelated events from the American Cancer Society’s Lights of Hope on Tuesday to CureFest on Sunday.

All during the week Conor watched, listened and talked about cancer. At first there was a lot of hesitation and apprehension. By Thursday, he was doing better. At a Golden Toast reception attended by 150 people for Congressmen McCaul and Van Hollen, co-chairs of the Congressional Caucus on Childhood Cancer, Conor gave a little talk on the gold flashlights that were being given to the guests as a symbol of shining a light on cancer. That was a first for him and he did it well.

ConorWhiteHouse_edited-1It all came together on Saturday night for Conor at a candlelight vigil in Lafayette Park, just across the street from the White House. It was held to honor the children who died because of cancer. First, our little man and other survivors were recognized. Afterwards, I watched Conor listen intently to each parent who spoke about the child they lost. There was complete silence in the huge crowd of about 1,000 as one by one gave very compassionate testimony what their child and their family went through in their own battle against cancer only to lose their child in the end. It was chilling. From there, we all moved silently to the gates of the White House where all of us in unison raised golden flame candles and sang Amazing Grace. It was the most moving and chilling event I have ever seen.

When the vigil ended, we sat on a park bench in front of the White House facing the park ConorBenchwhere all the parents had talked about their experiences. Conor looked up at me with his eyes full of innocence and said, “Grandpa Joe, what do you think about all of this?” Trying to not let my voice waiver, I said, “No one…no one person who was here tonight will ever forget this particular night for the rest of their lives.” I told Conor he was providing hope for others whose children are diagnosed with cancer. He is a sign of hope for others and in order for them to have courage to fight they needed hope and he could provide that sign by just being himself. While rubbing the top of his head, I asked him, “What do you think about all of this?” He looked up and replied, “ I am so very, very lucky.” We hugged, I held him close so he could not see the tears in my eyes and at that moment all I could say was simply, “You’re right.”

On the way back to our hotel we talked more and the next day at CureFest we talked again. We will talk a lot in the coming days, weeks and years.  Not only is Conor lucky, but so are we.

Author: Joe Baber, Conor’s Grandfather

Related Articles:  Survivor and Survivor 2nd Edition

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September’s Child

SeptChild_edited-2In my household, the month of September and the passing of Labor Day used to mean tucking away my white jeans, getting the kids settled at school and ordering Halloween costumes.

After my son’s cancer diagnosis in 2013, September will never be the same …EVER. The month now carries a sacred significance for our family and for all families touched each year with childhood cancer.

September is the designated month to raise National Awareness of Childhood Cancer.   Each September there are families working their hardest to reach out and awaken the general consciousness about this elusive malady that touches the most beautiful and vulnerable part of our society.

goldawarenessRibbonblue_edited-1Yet most people don’t know what the month’s “golden ribbon” signifies: a desperate, unmet need for greater funding to find treatments. Headlines about advances in cancer therapies for adults obscure the astonishing fact that, for kids, there is no treatment revolution. Only 2 new pediatric cancer drugs have been approved for children over the past 30 years, and national and pharmaceutical funding for new therapies is virtually non-existent.

I don’t want to share with you the statistics, the numbers about the stark reality of childhood cancer.  I do want to ask you, have you ever been a child?

I want to ask you to reflect back and remember if you trusted the grownups in your world to make your world safe and just. I want to ask you if you ever felt the presence of a love so enveloping that you knew that no matter what, goodness would prevail and children would fatherSon_edited-1always be a nation’s priority. I want to ask you to remember that grown ups have a sacred duty to be stewards of our children , especially the ones that don’t have a voice or wherewithal to change the status quo.

If you recall the innocence and faith that once defined you…then you must already know much about why raising awareness this month of childhood cancer is imperative. This September, you can do your part in forming an invincible circle of light and love around the child and family affected by cancer.

September summons a golden opportunity for all of us be better advocates for children and to be informed about the varied challenges facing childhood cancer research. Septgirl_edited-1Awareness can come in many different ways. It can be something as lovely and powerful as a whispered prayer for a child and the medical team taking care of the child, being fully present for the family, raising funds for research that is desperately needed, reaching out to the teacher, the politician, the artist, the corporate sector, your social media friend and challenging them to support this cause. If you can summon the child in you, then your efforts will come from an authentic call in your heart and perhaps, as the school year marches on, September will not just quietly slip away.

Author: Asha Virani

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