01/14/2013 12:20 pm ET | Updated Mar 16, 2013

Are We Not Recalcitrant Enough?

Per Webster's dictionary, one definition of recalcitrant is something that does not respond to treatment. Recently, the president signed into law the Recalcitrant Cancer Research Act of 2012. The purpose of this law is to direct the powers that be at the National Cancer Institute and the National Institute of Health to initially identify two forms of cancer that have a fifty percent or less chance of survival over a five-year period. The function of the designation is then to develop a research scheme for these most difficult of cancers to treat. Naturally, when I looked at this legislation I thought about the cancer that stole my daughter Alexis. As many of my readers know, Alexis was diagnosed with an inoperable brain tumor known as DIPG. DIPG, essentially, is a terminal diagnosis. The cancer has remained that way since its classification. There has not been a single effective treatment developed for DIPG, ever. Radiation, an obviously toxic treatment, is the only thing that provides temporary relief. Recalcitrant? Of course it is.

As many of the cancer lobbies line up to push for this designation, it is essentially a certainty that both pancreatic and lung cancer, the two cancers that lobbied heavily for the passage of this legislation, will almost certainly gain this designation and all other forms of cancer will wait for some time in the future for the "recalcitrant market" to be opened up for additional designations. It should be noted that the director of the National Cancer Institute, Harold Varmus, M.D., must make these designations within six month of the enactment of the legislation so only time will tell. In addition, he has the power to select other recalcitrant cancers in this period, should he see fit. Given the certain death sentence that DIPG carries as well as the potential to unlock answers to treatments for other childhood and adult brain tumors, it seems to me that obtaining this designation would be quite simple. Unfortunately, as with many things that happen in Washington, it is never that easy or straight-forward.

One of the main impediments to the implementation of this legislation as well as obtaining the designation for DIPG or any other childhood cancer as recalcitrant are the directors of the National Cancer Institute, including Harold Varmus, M.D. It is well known that Dr. Varmus does not like having mandates forced upon him by Congress or any other source. Additionally, Dr. Varmus, and some members of Congress, believe that only NIH and NCI should make any type of determination as to specific disease funding and allocation for research. This pattern has certainly been evident in terms of the overall childhood cancer funding scheme on the federal level. The disparity is enormous as I have pointed out in other pieces. And thus, many of the cancers that fit this definition will probably not be placed on this list and receive the attention that they deserve. And that is the most unfortunate aspect of the opportunities that now present themselves with this legislation. Unfortunately, legislation that impacts the childhood cancer community is often very rare. Any opportunity that arises must be seized upon with swift action.

The tug of war between the legislative, advocacy and the scientific community also has a long history. The scientific community frequently advances the argument that there should not be direct legislative mandates for disease specific funding or research. Disease priorities, it seems they suggest, should be left to the medical community to determine. Certainly Senator Tom Colburn, M.D., (R-OK) believes this to be the case and openly opposes legislation such as this. The problem with this "hands-off mentality" is that it has left childhood cancer dreadfully underfunded on the federal level in relation to the impact the disease has upon children. For those of us who fight for greater federal funding, it is simply not acceptable to allow childhood cancer to be so poorly treated and under-recognized. Although the childhood cancer lobby is growing in voice, we still lack a lot of the "power" many of the other cancer lobbies possess. Limited funding is one obvious reason for the lack a stronger voice. In addition, we are diverse, representing many different types of cancer, and often a diffuse bunch. Accordingly, our message is often not as coherent as it should be.

We may not be able to gain the designation of DIPG or another form of childhood cancer as "recalcitrant" at this point in the cycle. Obviously I am not thrilled by this almost certain realization. Nevertheless, sometimes you have to fight the battles you know you can prevail in to win the war. Accordingly, now is the time to look towards the future. By making strategic alliances with members of congress, insisting on congressional oversight of legislation like the Recalcitrant Cancer Research Act of 2012 and pressuring people like Harold Varmus, M.D., and NCI in general to do more for childhood cancer, I truly believe that the federal picture can in fact change. Of course there will always be members of congress and those at NIH and NCI who object to disease-specific legislative mandates. I firmly believe, though, that if the wheel squeaks loud enough, those objections can surely fade away. Yes, we are recalcitrant enough.