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.

To be continued...