This is beginning of a four part series by Jonathan Agin, an attorney and well known childhood cancer advocate (www.jonathanagin.com) in Washington, DC. He is married to Neely Agin and is the father of Alexis, Gabriel Agin and Trevor Agin. Alexis was diagnosed in April 2008 with a tumor in her brainstem and battled for 33 months before passing away on January 14, 2011. Mr. Agin, along with his wife, operate a Washington, DC chapter of a pediatric brain cancer foundation. In his spare time, Jonathan runs marathons and participates in triathlons. He has become a very effective voice for the childhood cancer community.   Four Square Team Captain

Introduction

Childhood Cancer, My Road Map

It is easy for me to write about childhood cancer in many respects. It matters to me. It touched me personally through the diagnosis and loss of my amazing daughter Alexis. I have come to know so many families touched by this disease, and unfortunately, too many children who have lost their lives from this insidious killer. It matters. Where I think the struggle lies, is connecting childhood cancer to the masses and then, beyond that, changing the entire landscape with respect to fighting the disease. Childhood cancer represents many different types. Cancer of the bones, brain, nerves, soft tissues, etc. It is not simply one single type. Therein lies the problem for many of the issues that we face as a community with respect to unification. In addition, the thought of children being diagnosed with cancer and their struggles is not particularly pleasant. That is completely understandable. For those of us who have had to witness their child go through the journey though, we know just how amazing these children are. Finally, we lack a well organized lobby. I am hopeful that can change as well. I am trying not to be too self-indulgent here, my apologies for the shameless plug.

So, what can be done to ensure that childhood cancer is not fought in a vacuum by overtaxed parents? I have some thoughts. I plan to share them with my readers, and hopefully a broader audience as well with the help of all of you. Over the coming weeks, I intend to lay out several areas for collaboration and focus that I believe would be helpful for our children and our cause in general. I am certainly not an expert, I am simply a father who witnessed his amazing daughter journey from the day of her diagnosis in April 2008 to the day when she transcended this earth in January 2011. Along the way I have had the opportunity to meet many amazing children and parents who are all committed to joining this fight and winning the war. I am fortunate to have this platform, to live in Washington, D.C. and be able to have access to lawmakers, and to have a little bit of legal knowledge that makes me just scary enough to be successful. So, where do I think we can move the ball ahead?

First, we need to enter into real discussions with pharmaceutical companies on several issues. There needs to be better access to compounds for testing in research and mouse models. It simply is unacceptable that some pharmaceutical companies make it very difficult for researchers to obtain compounds. The discovery rate for the efficacy of compounds is being thwarted by the slow delivery of the drugs in the first place. Time is not an element that we have in the childhood cancer community. More importantly, despite incentives placed upon pharmaceutical companies to develop new pediatric cancer drugs, there has not been a single new childhood specific drug marketed in over twenty years. This has to change and there are more ways to create incentives and demonstrate profitability models to the pharmaceutical industry for childhood cancer specific drugs. I will tackle this in my first piece in this series.

The Food and Drug Administration needs to change the manner in which it operates. Far too often the FDA acts as an impediment to the development, marketing and delivery of drugs to our children. I have witnessed it first hand in the treatments that Alexis was offered, and more importantly, not offered. As a parent, you will do and try anything and everything to save your child. Unfortunately, you are not always provided that opportunity and frequently, it is the FDA that acts as the head of the snake denying these chances. We must enter upon a new dialogue with the FDA to make our voices heard. We must ensure that our children are provided the opportunities to fight and that we are given the chance to fight. There is a message that we must deliver. Knock knock FDA. The message is coming.

Federal funding levels must change. I have written many pieces on this topic. I have sat with members of the National Cancer Institute and discussed this issue, as well as been in many congressional offices talking about this topic. The buzzword inside and outside of the Beltway may be Sequester; however that should not be the end to our discussion about funding disparities. There is money available and we can and should continue upon our mission to change the proportions allocated to childhood cancer. From the top, Harold Varmus, M.D., to members of congress, there is a message that we can send, with appropriate asks, that I will present. The ability to message the appropriate “asks” is paramount to being successful at changing the funding landscape.

Unity. It is often discussed, it is often attempted, but it is not often achieved. With so many different types of childhood cancer, and the wealth of amazing foundations and organizations, it is a difficult task to try and gather as many under one roof or umbrella and create the common goals that are necessary to achieve a clear message and strategy. I think it can be done. In the DIPG community, the type of tumor my daughter had, we have created what is known as the DIPG Collaborative. This group has come together to pool funding and resources to gain greater collaboration among researchers. Each member continues to maintain its own identity while creating a larger voice in the community. The mandate is clear here, research funding. In the childhood cancer community in a broader sense, we must all be able to agree on a handful of smaller “asks” to ensure that we not only have success, but more importantly, can grow into a larger more organized and action oriented entity. I believe this is possible and will present my thoughts in the coming weeks on this issue.

Over the coming weeks I want to provide my thoughts on the topics outlined above. I certainly do not have all the answers, I simply have this platform. There are so many amazing people and families who are walking this path and fighting this battle. I am simply one of them. The ultimate goal for us all is to be able to ensure that the next time a family is pulled aside and told “your child has cancer,” the next words out of the doctor’s mouth will be “but thankfully we have a cure.” Those words will never be spoken to my family for Alexis. For us, it is much too late. Alexis paid the ultimate price. I want a cure. I want a better outcome for every child currently battling and who will be diagnosed in the future. It is more than time for this to be realized. I think it can be, I think.

Author: Jonathan Agin      Parts 1 to 4 are continued  below

Part 1 of 4 

Engaging the Pharmaceutical Industry in the Fight Against Childhood Cancer  

This is the first piece in a four piece series that I hope will lay out a plausible road-map for the fight against childhood cancer. Please share your ideas with me as we journey together.

The fight against childhood cancer is not possible without innovative drugs that are available for delivery without significant restrictions. This statement, given the lack of development of childhood specific cancer drugs is difficult to mesh with the current state of affairs, yet I think is completely true. There are obvious problems with respect to the overall access to these drugs both for testing in the laboratory as well as in the realm of patient availability. In addition, there is little movement in the development of childhood specific drugs. Part of these issues lie squarely with the Food and Drug Administration (FDA), or with institutional review boards who think they are protecting our children by failing to grant access. That’s a discussion for down the road in this series.

We hold the pharmaceutical industry in this country in a unique place. In many instances, we deal with and approach them at arm’s length and never try to engage them on the ground as advocates. This is a mistake. The childhood cancer community needs the pharmaceutical industry and the companies as partners, not as foes. First, we need to engage them to ensure that they are willing to allow compounds into the laboratory for testing. This is a significant issue in the brain tumor community in general. One of the methods researchers have for finding breakthroughs is to be provided with drugs to test. Yet, there seems to be the sense that this is not occurring at a fast enough pace or that access is not being provided in general. Part of the reason for this is that the drug manufacturers are concerned over the viability of compounds for commercial sale and use. And thus, if a drug fails in a research setting, that could suggest that the drug will not be viable in patient trials. Therefore, manufacturers are afraid to allow further testing and release compounds. This is completely understandable. For profit companies are entitled to continue to pursue business models that generate profits. Given this conundrum, we must discuss this lack of access amid the fear that failure will negatively impact the bottom line of the company. In addition, as suggested below, we must try and partner funding with drug manufacturers to obtain the compounds for testing in labs.

It is costly to bring a drug to the market. This cost, given the perception that a low patient population does not equate to a profit model is one of the largest stumbling blocks for the development and marketing of new childhood cancer specific treatments. Therefore, the childhood cancer community must demonstrate to the pharmaceutical companies that creation of child specific drugs can be used for upstream profits in the more populated adult cancer community, as well as other disease specific communities. Because childhood cancers do act differently than most adult cancers, and children are sometimes better able to withstand the toxicities and rigors of treatments, there is the theory that focusing on children could create greater numbers of efficacious drugs for adults. Therefore, patience in the creation phase of new drugs could pay off in the end for greater profitability. Children will always get the “hand me downs” but if we can demonstrate success in the creation of drugs that help children but also help larger adult populations, we can provide adults with “hand me ups.” Of course, this involves drug manufacturers taking a certain leap of faith, but it also embraces the theory that there are greater rewards through greater risks.

There have been several attempts to legislatively impact this lack of drug development and access. The most recent is the Creating Hope Act. This act, that Nancy Goodman and many in the childhood cancer community worked so hard to obtain passage of, creates incentives for drug manufacturers to develop new compounds for childhood specific diseases, including cancer. It is a milestone in the legislative landscape and should prove to be a significant tool for the advancement of our cause. Unfortunately, since its passage, I am unaware of a single drug manufacturer taking advantage of this legislation despite the ability to utilize it to increase profits by obtaining favorable fast-track review for another compound. We in the community must find ways to push the pharmaceutical companies to utilize this tool. Its existence simply is not sufficient. We must seek to meet with the executives of these companies and engage them in a truly personal manner. This has worked in the past for various compounds including one called Xerecept, a steroid alternative that was withheld from pediatric trials until the community confronted the executives of the manufacturer with evidence of the horrors of steroid use in children with cancer. Ultimately, with the help of the FDA, the compound was placed in childhood trials and it is proving effective. We must attempt to open the doors and discuss the landscape and the incentives. These discussions necessarily should include the ultimate discussion of profits from the creation of childhood specific drugs.

Finally, we must approach and treat the drug manufacturers as partners. This can be done by trying to join forces with private monetary interests to help ensure that clinical trials do not losing funding, and, more importantly, matching researchers with compounds and then supporting those research trials by removing the fears that the repercussions of failure bring. By combining forces, we can create a lasting partnership that keeps in mind the objectives of both the childhood cancer community as well as the drug manufacturers.

This is a long and evolving issue that is not going to change overnight. For those kids fighting cancer at the present time, and children like my daughter Alexis who lost her battle, this portion of the road map comes too little too late. For many, that will not be palatable. I completely understand and frankly I do not disagree. However, if the childhood cancer community is going to be the tip of the spear that thrusts itself broadside into the issue of drug development and access, then we must exercise a mixture of steadfast resolve and patience at the same time. Opening the doors is not an easy task, and, in all honesty I do not have all the answers. I am fortunate to have this platform. I simply know that at some point, the door will open and we must be prepared to walk through with the ideas. Altruism and profitability are not that far removed from one another.

The collective voice of the community is rising. I have witnessed this happen over the last number of years. It is through the motivation and singular conviction of all of these amazing parents and children that the balance is shifting. I just do not know whether the drug manufacturers have heard our voices enough.

Author: Jonathan Agin       Part 2 is continued  below