Welcome!

Welcome_edited-1

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.

MoonShotFirst

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

The Cancer Moonshot: Are Our Kids Stepping on the Moon Too?

MoonShotAgin_edited-1On September 12, 1962 President Kennedy delivered his famous “moonshot” speech. Screen Shot 2016-02-08 at 5.33.01 PMDeclaring that as a nation, we would focus vast resources to landing a man on the moon and safely bringing that man back to earth. It was ambitious and the roadmap was not fully developed. President Kennedy of course never lived to see that dream come to fruition; however when it did it is now even more fitting and apropos that the man who ultimately first set foot on the moon lost a child to cancer himself. Armstrong’s young daughter died from what most of us in the childhood cancer community now believe was DIPG, the same type of inoperable brain tumor that ultimately took the life of my daughter Alexis.

Fast-forward to the President’s recent State of the Union address delivered on January 12, 2016. He announced that once again the United States would embark upon heading to the moon. Except this time, the moonshot refers to the Emperor of all Maladies; Cancer. With Vice President Joe Biden steering the effort, the goal is to end cancer, as we know it. The cancer moonshot was announced with the hopes of infusing $1.0 billion dollars to the effort. In the 2017 budget that the White House prepares, there will be a request for an additional $755 million increase in funding for cancer research generally as well as the oversight of an additional $195 million in new funding provided to the , . The outline of the moonshot was released by the White House and can be found here.

Screen Shot 2016-02-08 at 5.34.57 PMFor the childhood cancer community, a simple question surfaced: Are our children going to the moon? The news release the White House issued does specifically mention pediatric cancer. The outlined effort for pediatric cancer, as denoted in the release, is aimed at developing new technologies for drug libraries for screening to discover new treatments; efforts to increase tissue and tumor specimen collection for the “rarest” childhood cancers; and the collection and inclusion of increased clinical data to assist clinicians in the future course of treatments. Based upon this thumbnail outline, it is difficult to tell whether or not our children will set foot on the surface or whether they will be like Michael Collins, who stayed in the command module poised to pick up Armstrong and Buzz Aldrin after they completed the first walk on the surface in 1969. In theory the efforts outlined in the release would certainly make a positive impact in the fight against childhood cancer. Creating a larger drug library for increased efforts at drug screening is critical to determining whether or not existing drugs, compounds, and even potentially biologics, demonstrate efficacy upon specific cell lines, tumor types, genetic mutations or other identifiable targets.

There is a more pressing and underlying issue that needs to be addressed, as it exists like a pink elephant in the room. Specifically, it is the issue of drug development and availability for childhood cancer. The ability to conduct more substantial drug screening, dependent upon access to the drugs and compounds, is the first component of unlocking more effective, less toxic treatments (of course backing up even further identification of proper targets to utilize for drug screening and targeted therapies is beyond essential). The secondary component of the equation is ensuring that these drugs and compounds, once the efficacy is demonstrated in the preclinical setting, are available to be initiated in clinical trials or available for clinical use. Anything less in this overall effort, in my humble opinion, ensures that children, like Michael Collins, will remain in the capsule waiting for the others to arrive back inside.

Ensuring greater data sharing either in the research or clinical setting, collection of increased epidemiology data, and tissue collection are of course necessary components to increasing the overall understanding of biology, incidence rates and treatment efficacy. Nevertheless, the key question remains, will more clinical trials be initiated for children with the drugs that are screened if the preclinical data “de-risks” the use of any of those drugs? Children with cancer without a set frontline treatment protocol are usually left without many options. For recurrent or metastatic disease, the options are even more limited.

bicycle-wheelI view childhood cancer like a bicycle wheel full of spokes. There are many spokes on the wheel and individually the spokes represent separate issues to be addressed. No single spoke is sufficient enough that if it was the focus of the moonshot it would be a “cure” for all forms of childhood cancer. Accordingly, placing bets on which spoke to focus upon is obviously very difficult. There are some commentators that have already opined that the amount of money to be dedicated to the overall effort is insufficient. This is probably very true. And, thus as we drill down to where our children fall in this moonshot, it seems likely that the overall impact will not be significant. Of course it is impossible to provide any reasoned assessment of the overall efficacy of this effort at this point in time. It is easy to criticize, but such criticism is presently misplaced. In the end, any additional focus and resources dedicated towards childhood cancer is thoroughly welcome.

Neal Armstrong reached those first monumental steps by carrying his grief with him and allowing those emotions to keep him focused. In many respects, children with cancer have already set foot on the actual moon. It is up to us advocates to continue to ensure that children with cancer are always brought out of the command spacecraft and firmly planted on the moon. And it is up to us advocates to continue to work effectively to create opportunities for drug discovery, development and availability. An identified target is key to determining effective treatments, but there is an underlying component to the useful nature of that data. Unfortunately, if those treatments are not made available, then children with cancer can only continue to look at the moon as they circle it waiting for a turn to take that one giant step.

Author: Jonathan Agin
Reprinted from Max Cure Foundation. Follow Jonathan on Twitter @jonathanagin

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FlyMeToo_edited-1Fly me to the Moon

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Broken Trust

BrokenTrust_edited-1I just can’t believe what I read the other day and what I am about to write about right now. This about trust—trust between patients and their healthcare providers. This is about trust that is being broken.

What I read:

The New York Times published an article on January 29, 2016, entitled: Drug Shortages Forcing Hard Decisions on Rationing Treatments. It was actually the subtitle that caught my attention: Such shortages are the new normal in American medicine. But the rationing that results has been largely hidden from patients and the public.

The article was not specific to childhood cancer or cancer in general, but many of the doctors quoted were oncologists. They talked about different approaches to rationing drugs, including decisions based on age. I encourage you to read the entire article, if you haven’t already.

What I’m writing about:

I’m going to reserve comment on the drug shortage problem itself, including the causes behind it and any proposed solutions. I am also going to reserve comment on the complex issue of rationing the drugs, other than to say that I recognize that nobody wants to be in this situation and that such decisions must be very difficult. I don’t want to get into any of that right now.

I’m writing about 1 aspect and 1 aspect only: the decision to not tell the patients and families.

Here are some specific highlights for context, taken verbatim from the piece:

CLEVELAND — In the operating room at the Cleveland Clinic, Dr. Brian Fitzsimons has long relied on a decades-old drug to prevent hemorrhages in patients undergoing open-heart surgery. The drug, aminocaproic acid, is widely used, cheap and safe. “It never hurt,” he said. “It only helps.”

Then manufacturing issues caused a national shortage. “We essentially did military-style triage,” said Dr. Fitzsimons, an anesthesiologist, restricting the limited supply to patients at the highest risk of bleeding complications. Those who do not get the once-standard treatment at the clinic, the nation’s largest cardiac center, are not told. “The patient is asleep,” he said. “The family never knows about it.[…]

Studies have associated alternative treatments during drug shortages with higher rates of medication errors, side effects, disease progression and deaths. For example, children with Hodgkin’s lymphoma who received a substitute to the preferred drug had a higher rate of relapse, researchers found, and adults with a genetic disorder called Fabry disease had decreased kidney function when their medication was cut by two-thirds. One alternative guideline adopted during a shortage of intravenous nitroglycerin “was downright scary from a clinical perspective,” according to Dr. Nicole Lurie, a senior federal health official.

Physicians say that many of the changes they are compelled to make appear to do no harm. But, they acknowledge, typically no one is tracking outcomes in patients who get a drug and others who get a substitute or delayed treatment.

Doctors and hospitals often do not tell patients about shortages and the resulting rationing because they do not want them to worry, especially when alternative drugs are available, or because they feel it would stir up too much anger.

Dr. Ivan Hsia, an anesthesiologist in Ontario, Canada, said many physicians in his field adopt what he called “the paternalistic model — like I’ll inform them when I think it’s unsafe enough to inform them.

When he and his colleagues surveyed hundreds of patients at the Mayo Clinics in Arizona and Florida and others in Canada about their preferences, the results surprised him. Most wanted to know about a drug shortage that might affect their care during elective surgery, even if there was only a minor difference in potential side effects, and many said they would delay surgery.

When the study was published last year in the journal Anesthesia and Analgesia, an accompanying editorial urged health professionals to disclose shortages and their implications. “Patients want to know and they should know,” the editorial said. “There is no ethical ambiguity.”

 Dr. Eric Kodish, a children’s cancer doctor who heads the Cleveland Clinic’s center for ethics, humanities and spiritual care, said patients should be told. “It’s their bodies and their lives that are on the line.”

Indeed, Beverly Smith, a Cleveland patient who has Crohn’s disease, said she had no idea that an important ingredient had been removed from the daily intravenous nutritional treatments she depends on until she developed side effects from the deficiency. “Why didn’t anybody tell me?” she asked. [….]

A recent shortage of a therapy for bladder cancer, BCG, demonstrates how the lack of national guidance can lead to very different decisions. One ,urologist, Dr. Andrew Stephenson, said he came up with BCG rationing guidelines that were used with dozens of patients after being shared with colleagues. “We tried to reserve the BCG for those patients who needed it the most,” he said.

Merck, the manufacturer, said it filled requests from a waiting list in the order received, and left rationing decisions to doctors. Some cancer centers reduced the length of BCG treatment from three years to one, because the benefit may be smaller after the first year. Others restricted BCG to patients whose tumors were mostly likely to spread or recur. And still others decided to reduce the typical dose so that each vial could be used for three patients instead of one, which some experts say raises questions about efficacy. Some outpatient clinics just ran out.”

Wait—what? Patients are not being told about shortages and rationing, citing reasons such as “worry” and “anger”? Is that really what I read? I seriously had to do a few takes and pinch myself.

PrescriptionAs a registered pharmacist who is trained in the hospital setting, I have to say that I am completely and utterly appalled by even the mere suggestion—and this is no mere suggestion, but what it actually says. Good grief.

“Patients should know.” “Patients should be told”. Should, should, should.

With all due respect, what’s with the “should”?

Nothing like stating the obvious here, but there is no should in this argument. Unequivocally, patients must be told if a therapy is being modified in response to a drug shortage.

Then we have this part, which completely blows my mind: “And still others decided to reduce the typical dose so that each vial could be used for three patients instead of one, which some experts say raises questions about efficacy.”

Wow, raises questions about efficacy, you don’t say? Are patients being told about these potentially efficacy-reducing dose-reducing strategies of rationing drugs to spread them around, even if the spreading around is thin? I can’t even find the words for just how QuestionsAboutEfficacyunethical it would be to not tell the patient that a dose reduction to save drug was being made behind their backs. I just can’t even get my head around that so-called strategy.

For many types of cancers, not all but many, alternative regimens exist that are considered equally effective. If I was being treated for cancer or if one of my family members were being treated for cancer, no way would I accept a suboptimal dose or duration of a chemotherapy agent on the basis of a drug shortage. I would explore alternative regimens. I would explore treatment at another center, if that’s what it took. If a dose reduction was ultimately the way to go, that would be for me to decide, after a detailed discussion of risk:benefit with my doctor.

Cancer patients have a tough enough battle without unknowingly receiving a potentially less effective (and possibly more toxic) substitute.

In some cases, reducing the dose a little might be a reasonable option. In other cases, it may be unreasonable.

The bottom line is that patients must be involved in this decision-making process.

I don’t care how busy these doctors and pharmacists are; they need to find time to explain the shortages to the patients and work with them on a solution that everyone feels good about.

So that’s the reality out there. Yeah, it’s scary, but I believe that it is way better to be informed than to live with one’s head in the sand.

I have no way of knowing how pediatric oncologists and adult oncologists may differ in terms of how they are handling the communication surrounding the changes they are being forced to make in response to not having enough drugs to go around.

LackofdisclosureI am in no way trying to demonize anyone.

Again, to reiterate, my gripes are not around the tough choices—but instead about the lack of disclosure to patients and families.

Clearly, it is more important than ever to really be vigilant—like a hawk.

Know the prescribed doses of all drugs to be used over an entire treatment regimen, also verifying that the doses are within the recommended ranges.

Keep yourself abreast of the drug shortage problems. Here are links listing drug shortages by generic name: http://www.ashp.org/menu/DrugShortages and http://www.accessdata.fda.gov/scripts/drugshortages/default.cfm. Check them. Nobody should have to do this on their own, but apparently patients must stay informed about this

Daunorubicin

Daunorubicin, primarily a leukemia drug and used for a few other cancers, remained in short supply for over 18 months 2014-2015

stuff. If there is any ambiguity or any questions, I would suggest calling the pharmacy at your hospital or clinic. It might not hurt to call them anyway. Form a relationship. If you have any friends or family members who are physicians, pharmacists, or nurses, ask them to help you as well.

Everyone should be doing this anyway, but if you haven’t been then now is the time to start: routinely check the dosing on any prescription bottles or infusion bags. Make sure the dosing has not changed. If it has, ask about why it is different. Sometimes doses might be modified due to a prior toxicity or for some other justifiable reason. It may or may not have anything to do with a drug shortage.

Preventing such an encounter is a much better plan though. So next time you talk to your doctor, pharmacist, or nurse, you may want to open up the dialogue. Be direct. Tell them you saw the New York Times piece. Tell them you are concerned about something like that happening to you or your loved ones—changes being made or drugs being withheld without your knowledge. Make it clear that you want to be informed. Talk to them, openly and honestly, but still stay vigilant!

If it were me or my family member and we showed up at an outpatient cancer clinic, only to find that what we had shown up for had been intentionally modified (whether it be a substituted drug or a reduced dose) without my knowledge or approval, I would go and round up 3 chairs: 1 for the prescribing physician, 1 for the dispensing pharmacist, and 1 for the infusion nurse. All 3 of them hold a responsibility. It would be explanation time. That’s just what I would do.

I would not be happy if it happened to me or a loved one, and I’m equally as unhappy knowing that these things are happening to other patients.

If a medication is being modified in response to a drug shortage, and the response is that it won’t affect anything, then ask your doctor for the evidence. Have them back it up just to be safe.

So, be proactively aware. Don’t be afraid to ask questions or speak up. Don’t let concerns get brushed off without evidence.

Trust your instincts.

Author: Laurie Orloski, PharmD

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

Fly Me to the Moon

FlyMeToo_edited-1President Obama called for an end to cancer in his State of the Union Address to the nation this year. He tasked his own Vice President with the job and since then the buzz word “Moonshot” has offered renewed hope for millions of Americans.  EMoonshotROCKET_edited-1very one in our childhood cancer community is very excited about Joe Biden’s very aggressive initiative. He calls it, the Cancer Moonshot. Mr. Biden’s son Beau died of brain cancer a year ago. Like all of us affected directly by cancer, it’s made him extremely passionate and focused about finding cures for this terrible disease. No parent should ever see a child die from cancer.

Mr. Biden wants to approach cancer research with real gusto, the same way America came together to successfully put a man on the moon in the late 60’s. The Vice President has spent several months visiting a host institutions involved in finding cures and will visit more in the future. Along the way, he has found such obstacles as silos that resist information-sharing, territory wars and competition among researchers. He will also find that some government regulations meant to protect patients and improve drug development, are also a few more major impediments that will need to be cleared to get this “Moonshot” off the ground.

The Vice President identified two major goals:  1.) Increase resources — both private and public — to fight cancer.
2.) Break down silos and bring all the cancer fighters together — to work together, share information, and end cancer as we know it.

He went on to say, the goal of this initiative is simple — to double the rate of progress. To make a decade worth of advances in five years. “Over the next year, I will lead a dedicated, combined effort by governments, private industry, researchers, physicians, patients, and philanthropies to target investment, coordinate across silos, and increase access to information for everyone in the cancer community.”

We are totally on board with the Vice President! But, first we want to make sure all moon mission workers are aware of the major obstacles that childhood cancer has faced ever since President Nixon first declared War on Cancer in 1971.

The Vice President wants to hear our stories of how cancer has affected us and our families. The White House has provided a site to collect all the stories. We would be remiss to not urge everyone in the childhood cancer community to send them in. We need to let Joe Biden know that childhood cancer is not rare, but is a major national problem. One way we can make a point is to have a excellent response in submitting our stories.

Click Here to Submit Your Story

Click Here to Submit Your Story

When writing consider the following points:

First: We need to make sure that everyone on the moonshot mission  understands that childhood cancer is not the same as adult cancer. Kids are dying from twelve weirdly named cancers that have over 100 subtypes and none of them are breast, prostate, lung, colon, or other familiar sounding adult cancers.

Second: Everyone at mission control needs to be acutely aware that childhood cancer is the number one disease killer of kids. It was in 1971 and it still is. As a matter of fact, the rate of incidence has increased since Mr. Nixon declared war on cancer.

Third: When it comes to cancer research funding, all twelve of the childhood cancers lumped together receive less funding from the National Cancer Institute than prostate NCIFundingChart2007_2013_edited-1cancer alone. Nixon’s war on cancer has been going on for 45 years! Our children have been left behind. There have only been 3 drugs developed specifically for childhood cancer in the last 45 years compared to 60 new cancer drugs for adults in the last five years alone!  Today, after all those years, we have to embrace adult cancer drugs which are toxic and cause 95% of the survivors to have very serious health issues. Parents are shaving heads, selling lemonade, baking cookies, organizing walks and races to try to add more research funding. NCI has done little to address the such recalcitrant cancers as DIPG and others that are so prominent in our community and continue to take our children daily. A child whose life is taken by cancer loses nearly 70 years of life!

Forth: We have waited and waited to see progress. We are tired of waiting only to see our government continue to call this disease rare and therefore not a high priority. Children should be our highest priority. We are tired of having leaders point to the “tremendous MoonShotFirstprogress” we have made in childhood cancer survival rates in the last 50 years. Must I remind you that we are comparing today’s survival rates to fifty years ago when we were doing absolutely nothing but sending kids home to die. The truth is we have made little to no progress in the last ten years. To double this progress over the next five years as the Vice President said won’t mean much in the childhood cancer arena. We need to do much, much more than we have in the last decade.

We should take the attitude that we are not going to accept status quo on this Moonshot effort. Children deserve priority placement and a first class ticket to progress that is expected on this journey. It’s about time! We don’t have more time. Enough children have died from this horrible disease!

Author: Joe Baberbounceballauthor

A week after this post was published, the White House issued a Fact Sheet with more detailed information. While we are very pleased and grateful that pediatric cancer was identified as  one of the moonshot’s research opportunities, a fact with which we certainly agree, we will continue to question priority setting while monitoring levels of investment, and breadth of investigations. One thing Nixon’s 1971 War on Cancer taught us is to not take anything for granted.  We can not afford to let our children get lost in the shuffle.

Posted in Cancer, Childhood Cancer, Pediatric Cancer, Rare Disease, Uncategorized | Tagged , , , , , , , , , , , , , , , , , , | 2 Comments

I Gladly Spoke Up for Kids With Cancer Today

SpokeUp_edited-1I spent a brrrrrr cold Thursday afternoon in Philadelphia, at a Cancer Precision Medicine Conference in Philadelphia, hosted by the American Association for Cancer Research Screen Shot 2016-01-22 at 8.28.45 AM(AACR) and the Philadelphia Media Network. It was the first time that representatives from 6 Cancer Centers across Pennsylvania (Philly to Pittsburgh) were assembled in this fashion. It was a really cool thing because it represents a new frontier of collaboration toward cancer cures.

The first talk was by Dr. Stephen Grupp from Children’s Hospital of Philadelphia. He talked about what’s known as CAR T-cell cancer immunotherapy, a new approach to treating leukemia — where genetically modified immune cells are generating a response rate of 93% among kids with relapsed/refractory leukemia with no other viable treatment options. This type of response is UNPRECEDENTED in cancer. 93% response in heavily pretreated chemotherapy-resistant childhood leukemia, a population destined to die of their disease before this breakthrough — first documented in 2012 and since extended to 200 patients.

After some additional discussion of adult cancers, the meeting was opened up to questions. I jumped in as question número dos. It went something like this, not exactly but close: “I work in the adult cancer field as a contractor to the pharmaceutical industry but volunteer in the childhood cancer community, so I have several friends who have either lost children or whose children are dealing with the late effects of treatment. I know through my work that immunotherapy is moving to earlier lines of treatment much quicker in adults compared with kids. There are obvious reasons for this, including the fact that chemotherapy is not a good option for things like melanoma. And then there are ethical considerations in kids, because of the high response rates to chemotherapy. Chemotherapy may be effective but causes problems later in the form of late toxicity. Although we are not there yet, I was wondering if you could address the prospects for moving immunotherapy to earlier use in children. You mentioned in your talk the possibility of avoiding bone marrow transplants, but can you comment on the possibly of avoiding chemotherapy as well?”

Well, No, I’m not exactly short and to the point. Nonetheless, I grabbed the opportunity to remind the audience that children do still die from cancer while addressing the huge problem of late effects, which include secondary cancers and the need for things like liver and heart transplants later in life. Dr. Grupp already knows these things, but who knows about the rest of the attendees? My assumption was that most of the audience was in tune with adult but not childhood cancer. Late effects are more prominent in children.

The short answer from Dr. Grupp is that researchers are indeed looking to design trials to address the issues that I raised, including the use of immunotherapy before chemotherapy in certain high-risk patients unlikely to benefit from chemotherapy. That was good to hear. That’s a good next step. I know enough to know that if it works in the high-risk patients, testing in low-risk patients may be a logical next step. We are not there yet, key work “yet”. Chemo damages young bodies, we need to do away with it someday — key word “someday”. Again, not there “yet”, but all signs point to “someday”.

It was a large audience, 100s, mostly medical professionals. I had no idea how many childhood cancer parents and advocates were there, if any, when I stood to ask my question. There were over 20 questions from the audience over the course of the meeting, but mine was the only one specific to kids. My Question #2 was the first and only one specific to childhood cancer — how about that? I was glad that I took that opportunity to speak about kid-specific stuff.

854598_1280x720As I was walking out of the meeting, I spotted a very tall guy who stood out because he was so tall. But that face, so familiar. Then it was obvious — it was Devon Still of the NFL. His daughter Leah was treated at CHOP for a poor-prognosis cancer, and she’s currently in remission after a very public battle. Yay Leah! Her dad has been a very vocal advocate for not only her but also other kids; he has done a ton to raise awareness despite such tough days and keeping a busy work schedule. However, parents of kids who have been dealt the blow of childhood cancer should not have to shoulder that burden alone — it’s not fair. People like me, with healthy kids, can and should speak up too. It’s simply the right thing to do. Being a celebrity is not required.

Of all the “Big Ideas” surrounding cancer, I personally think that replacing DNA-damaging chemotherapy and radiation in favor of cutting-edge novel therapies (immunotherapies, among others) is a HUGE one for the kiddos. It’s critical for all patients, but especially the kiddos because of the survivorship issues that stem from the late effects of toxic cancer treatments. Once again, we are not there “yet” — there is lots to learn first — but it is important to work hard today toward that “someday”, when cancer is routinely treated way more effectively AND safely. So I will continue to stand up and speak up, like I did today. I like to talk — I’m loud —I don’t care if I fumble or stumble — I don’t care about who does or does not like my questions — so might as well put those traits to good use!

Author: Laurie Orloski

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Posted in Cancer, Childhood Cancer, Pediatric Cancer, Rare Disease, Uncategorized | Tagged , , , , , , , , , , , , , , , | 2 Comments

A 10 year old, Speaking from Experience

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Natalia Sofia is a childhood cancer survivor. Two years ago, she was only eight years old and diagnosed with Ewing’s Sarcoma of her femur. She has endured surgery, intense chemotherapies and radiation treatments. She suffers from side effects of her life-saving treatments. This is her story written and presented to her school in her own words.

NataliaPresentation _edited-1Did you know that worldwide a child is diagnosed with pediatric cancer every 3 minutes?

Before a child turns 20, about 1 in every 285 children in America will have cancer. Unfortunately I was one of those kids, I had cancer. But fortunately, I am in remission, now and doing very well!

Because of my experience, I think and feel that we need better medicine for the children. The medicine for childhood cancer is from the 1970’s…I know, right?!?  That’s a loooong time ago!  That’s why we need to get chemo that is made for kids, NOT adult chemo. Because the medicine for childhood cancer is really made for adults, that does not mean the kids don’t take it. Childhood cancer medicine is very strong and makes their hair fall off.  We need to donate money, hats, and beanies. I think we should get medicine that doesn’t involve hair falling off.

NataliaTreatmentDid you know that childhood cancer is more deadly than asthma, cystic fibrosis, and pediatric AIDS combined. The month of childhood cancer awareness is in September, the ribbon is gold and people wear gold tshirts to support childhood cancer. But when that month is gone, the awareness is gone and so is all the support from the people wearing the gold tshirts. But that doesn’t mean pediatric cancer leaves…IT DOES NOT STOP.

That’s why I am here today. I am asking YOU to help bring more awareness to our community. What can you do to help raise awareness? You can first promote more research to be done in the community. More research brings better medicines for kids like me. Lastly, you can organize a fundraiser in your neighborhood. We need more funding to help cure pediatric cancer. Most kids don’t die from cancer, they die from the chemo side NataliaSupergirleffects. That is how strong and harsh it is. Chemo side effects are mostly kidney failure, heart failure, and liver failure. And for most part secondary cancers. Also, only 4% of 100% of funds go to funding for childhood cancer research. Let’s make that into a 100%!!!
Put yourself in someone else’s shoes that has been through cancer or is still in it. See what it’s like for yourself. Think about it, seeing and going to doctors almost every day. Missing friends at school. If you have a fever, you’re in the hospital, sometimes for a month! It’s NOT fun. So, if you were an 8year old girl or boy in the hospital on Valentine’s or Halloween or Christmas Day…what would you feel like??

CHEMO DOESN’T STOP FOR THE HOLIDAYS AND NEITHER DOES CANCER.

Author: Natalia Sofia

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Baby Steps in Congress

One Step_edited-1

Getting things done for childhood cancer in Congress is not easy. For one, even as a community, we have very little political clout. One of the best things going for us is that  we have the Childhood Cancer Caucus in the House of Representatives and that is certainly a huge help. At the present time, almost one fourth of the House are members. The caucus was formed in 2009 with a mission to serve as a clearinghouse for information on pediatric cancer and a forum to aid Members of Congress in working together to address pediatric cancer. One of it’s major accomplishments was to get the Creating Hope Act passed.

The Creating Hope Act offers the incentive of issuing FDA priority review vouchers for those companies that produce a drug for a rare disease. While it costs the government very little to administer the program, the voucher is valuable because it allows a company to speed up FDA approval on another drug of their choice. The vouchers can also be sold to other companies.  So far, in three years, the FDA has approved three drugs for rare diseases. The vouchers earned have produced over $650 million in revenue for the developers. One drug approved this year was for neuroblastoma. It’s the first new drug developed for a childhood cancer in twenty years. That sounds great and it is, but now we have a problem.

The award of the third pediatric voucher for the neuroblastoma drug triggered a little-known sunset clause contained in the 2012 law which will cause the Creating Hope Act to expire on March 17, 2016.  The Creating Hope Act reauthorization was put in an all encompassing 21st Century Act that sailed through the House. Now, this big bill is tied up in the Senate. Unless something changes soon, it appears the 21st Century Cures Act will not pass the Senate in time to save the Creating Hope Act by March, 2016.

AdvancingHopeHeader_edited-1Prior to the passage of the 21st Century Cures Act, Rep. George “G.K.” Butterfield Jr. of North Carolina, introduced the Advancing Hope Act.  His bill expanded the cost-neutral Food and Drug Administration (FDA) priority review voucher (PRV) program, and eliminated the sunset clause altogether. The bill has had some push backs. By making the Act permanent, the FDA feels it will cause problems in that more and more companies will be using it and therefore causing a strain on their normal work flow and ability to approve all drugs for use.

All the drug developers now see the uncertainty in using priority review vouchers and have therefore delayed any work that may earn a voucher until the incentive program is reinstated.

We can not allow this program to have a gap or much worst die after so much success in only three years. One strategy is to ask Congress to approve the Advancing Hope Act (even if the FDA does not like it) and by doing so, show that there is a real need for these vouchers. Another strategy is to quickly re-authorize the present bill now, but we are not seeing an effort to do this since it is in the 21st Century Cures Act. What else has produced three new drugs for rare diseases? The answer is, nothing! Without the incentive of a voucher, it’s just not profitable for companies to invest in rare disease drug development.

Please help by asking your Representative and Senators to co-sponsor the Advancing Hope Act. Click HERE for more information on the Act and how you can help. Maybe we can force Congress and the FDA to come together and develop a real solution to get this effective program up and running for rare disease and childhood cancers!

We don’t have time for baby steps when we need to be running. We need Creating Hope, Advancing Hope or an alternative  to improve drug development for children with cancer.

Author: Joe Baber

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

Bereavement Meeting

Bereavement_edited-1Last night I went to a bereavement group. It’s for parents that have had a child die. I actually like to go to these meetings. There are no pretenses. We are all walking down the same path. We understand each other’s pain. When we ask, “how are you?” it has quite a different meaning and context than when someone who hasn’t had a child die asks the same question of us.

We had such a large crowd last night that we had to break into two different groups of moms and dads. Some with a spouse, others without. Some were young, first time parents whose child died as an infant. Some, like me, have more than one child. Others had a child(ren) after the death of their first. We had several elderly people (one gentleman was 90 and his daughter died years ago when she was 55).  Some kids died from disease while others were from car or motorcycle accidents. We have quite a few suicides in our group. We would have never met outside of this bereavement group. Our lives wouldn’t have crossed paths. But tragedy brought us together.

This group has made me more compassionate. I had read about the death of a teenager who jumped off the ninth floor of a parking garage a couple of months ago. Now, her mother and I are sitting next to one another. Her daughter is no longer some abstract story that I read about in the paper.  Now she is real. Her mom is a living (or trying to), breathing person that can’t fully grasp her circumstances. I feel her pain rolling off her. I understand her need, to avoid, acknowledge her new reality. She looks to me for solace. I’ve walked her path.

Some of the children have been gone for 15, 20 or more years. I look to those parents for solace and guidance. These are the people that give me the most comfort. I’ve had more people than I could count tell me to “give it time,” “it’ll get easier as time goes on,” “time heals all wounds”…  I could go on and on with all of the asinine cliques that have been said to me about feeling better the further away we get from Gabriella’s death. The simple truth is that it doesn’t get better. I think that “it gets easier” for the people that are saying those things to me because it’s not so fresh and raw for them. But, as I turn to the parents whose 11013206_957904817565003_3095121134372874505_nchild has died a decade or more ago and as I hold their hand or pass them a tissue as they cry, I feel relief with their grief. Relieved, because my feelings are validated that my love for Gabriella won’t fade as we get further from her death.

All different but, the same. The same grief. The same despair. The same longing to hear our child’s voice and laughter, to hug and kiss them, to have another conversation. We miss our kids.

#GabriellaMiller #TalkIsBullshit #SmashingWalnuts

Author: Ellyn Miller

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

Open Letter to Congress

OpenLetter_edited-1Dear Member of Congress,

As you are preparing to make decisions about 2016 funding for the National Institute of Health (NIH) please consider a few things.

  • Childhood cancer is the #1 disease related killer of kids in the U.S.
  • The incidence of childhood cancer has been steadily rising over the past several decades
  • Currently 1 in 285 children will be diagnosed with cancer before they reach the age of 20
  • 20% of children diagnosed are terminal on diagnosis
  • 60% of children diagnosed suffer life altering impacts of treatment
  • 95% of survivors will suffer serious health impacts before they reach the age of 45

Despite the fact that thousands of children are suffering and dying each year, the NIH continues to defend its position of basically ignoring childhood cancer research. They say that childhood cancer is rare, they hold childhood cancer research hostage – telling Congress they could do more with more funding.

NIH supports their position by using a 90% cure rate statistic for one childhood cancer to distort the entire childhood cancer picture. Ironically, this statistic is related to a childhood cancer that has been the recipient of the majority of childhood cancer research since the 1960s. The picture is not the same for other childhood cancers where there has been little to no investment, these childhood cancers continue to kill and maim our children.

NIH recognizes that childhood cancers and adult cancers are different. They acknowledge that studying adult cancers does not result in treatment options for kids, but often the opposite is true. In spite of this awareness, we continue to use downsized adult protocols to treat the majority of childhood cancers, often with devastating impacts because there are few, if any, other options. The lack of research in childhood cancer means there are few pediatric protocols, treatments and drugs.   We need significant Federal investment in childhood cancer research now to address the growing incidence of cancer, to reduce tremendous costs to kids and society as a result of secondary impacts of childhood cancer treatments, and most importantly to give kids cures they deserve.

Congress has been aware of the steadily rising rate of cancer in children for the past couple of decades. Congress passed the Children’s Health Act of 2000 as a direct result of government’s concern about the steady increase in childhood diseases.   Title XI of this act required the Secretary of HHS through the NIH to study risk factors for childhood cancer and improve outcomes among children with childhood cancers and secondary conditions. Title X created the Pediatric Research Initiative requiring the Secretary to establish an initiative at NIH conducting and supporting research directly related to diseases in children. Since childhood cancer is the #1 disease related killer of children in the U.S., it seems reasonable to assume that the Pediatric Research Initiative would be fostering childhood cancer research and increasing pediatric clinical trials. This initiative has been funded by Congress for the last decade. It is difficult, if not impossible, to understand the outcomes. The public deserves transparency in order to understand how HHS has complied with this Act.

The Children’s Health Act authorized NIH to conduct the National Children’s Study (NCS) which was funded for over a decade at a cost of over $1 billion to taxpayers. One might wonder, at the possibilities if there might now be more pediatric treatments for childhood cancer if these funds had gone to childhood cancer research instead of the administrative debacle that was the result of this taxpayer funded investment. Where is the accountability? In 2014, the same Director of NIH that provided oversight of the NCS, cancelled the study discounting advice of internal and external panels that completed reviews of the study. Congress allowed that same Director to provide a plan for reallocation of funds appropriated for the NCS.   In the private sector, if a CEO mismanaged $1 billion, I think it would be fair to say that the CEO would not be making decisions about funding investments moving forward. It seems that the reallocation might have gone directly to childhood cancer research since this was one of the original intentions of the legislation but as far as we know, childhood cancer research did not receive any of the reallocated funds. Despite much external pressure on NIH over the past couple of year, NIH seems to dig in its heels even more about their position that childhood cancer is rare.

Congress has been asking questions about how NIH sets priorities. Over 20 years ago Congress passed legislation requiring agencies to develop strategic plans that helped agencies set priorities, identify goals, objectives, and outcomes of funding decisions. Legislation intended to improve public confidence in government, provide accountability and transparency to the public about investment of taxpayer dollars.   In 2014 Congress required GAO to investigate how NIH set priorities and that same year required that NIH complete an overarching strategic plan by December 2015. It is hard to understand why an agency with a $30 billion budget does not have a comprehensive strategic plan. NIH goals, priorities, and transparency seems to be obscured by the giant HHS budget. Stakeholders waited patiently for NIH to conduct stakeholder outreach as they were developing the strategic plan but that outreach was limited to a 3 week comment period at the end of July. It feels like the outreach was only a façade, going through the motions without considering stakeholder thoughts.

How does NIH set priorities with the $30 billion provided by taxpayers?  NIH, our country’s largest research organization estimated funding for 2016 (RCDC) shows

  • Drug Abuse = $1 billion
  • Obesity = $1 billion
  • Alcoholism = $0.5 billion
  • AIDS = $3 billion

We understand the importance of these issues, but it is difficult for taxpayers to understand how these research investments would be of a higher priority than the childhood cancer that is killing our kids year in and year out. Let me say again, childhood cancer is the #1 disease related killer of kids in the U.S.

Since 2007 Congress has provided report language and encouraged NIH to increase funding for childhood cancer research. Around this time NIH began to show that childhood cancer research received around $180 million per year. Even this amount is not a true representation of childhood cancer research. The number is derived by asking grant recipients to denote any percentage that might benefit childhood cancer and that is calculated as childhood cancer research.

NIH may continue to defend their position in spite of devastating impacts to kids and families, and society as a whole. Statistics can be twisted and turned to assist the defense, but there is no doubt that the incidence of childhood cancer is increasing. We need significant investment of Federal research dollars into childhood cancer research now – the kids are waiting.   Members of Congress we ask you to stand up for children and make childhood cancer research a legislative priority in the NIH budget.

Sincerely,

Donna Carroll Carmical

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

September, 2015 in Our Capitol

SeptinWash_edited-1

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.

Friday and Saturday, September 11, 12, 2015, 7:00 p.m. ’till

88RichmondTweet_edited-1Richmond International Raceway will host two races where Childhood Cancer Awareness will be seen. On Friday, Richmond will run the Virginia College529 Xfinity 250. JR Motorsports will have three cars entered in the race. Each will feature Gold Ribbons in the rear quarter panels and the Childhood Cancer Awareness Month banner JRMS_edited-1on the trunk lid. Look for the #7 Tax Slayer Bowl car, the #9 Napa Auto Parts car, and the #88 Speedco car to raise awareness for childhood cancer. Saturday the Raceway is Going GOLD for Childhood Cancer Awareness and to honor Jeff Gordon’s work in pediatric cancer advocacy and funding. The track will have a gold start/finish line, gold ribbons on the walls, staff will be decked out in gold shirts and a lot of other pre-race activities to bring attention to Childhood Cancer. Toyota will have a pace car with gold ribbons.

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.

Space

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

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

Toast

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 1:30 p.m. to 3:30 p.m. 

Rally’s #MoreThan4 Walk to Capitol

Screen Shot 2015-08-22 at 5.16.36 AMThis is an unofficial CureFest event that is being organized by Rally Foundation for Childhood Cancer Research. 
Here is a preliminary schedule:
1:00-1:30 p.m.: Gather at J.W. Marriott hotel to make signs and discuss the rally
1:30 -2:00 p.m.: Walk to Capitol as a group
2:00-2:15 p.m.: Take group photo
2:15-2:45 pm.: Walk back to hotel
The majority of rally participants will be attending the Welcome Event which begins at 4 p.m. at the same hotel where this Rally walk begins.
Please RSVP with names of those attending to info@curefestdc.org. Rally is required to have a firm headcount for the rally.  This is very important.

Saturday, September 19, 2015   4:00 p.m. to 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 to 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.

 Space

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

NCI4percent 2007_2013_edited-2

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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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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.

Related Articles:

“As Our Children Wait, Part 1”

“Moving the Needle”

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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.

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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.

CarlEdwards_edited-1

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!

RaceSchedules_edited-1

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.

THE 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 – our 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 submission. 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:

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

What Not to Say When There is Nothing to Say

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

Life in Portsong By Mark Myers

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.

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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

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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

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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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