In the United States these days, there are discussions of the moon, precision medicine, targeted therapies, priority review vouchers and moving into the 21st Century. These are all buzzwords that touch directly or indirectly upon the efforts to treat cancer and other diseases in our population. These efforts are intricate and complicated to say the least, and at the end of the day, the childhood cancer advocacy community is playing a game of musical chairs in an effort to find a seat at the table. Ultimately, there is no way of knowing just what role our voices will play, and more importantly, what the overall impact will be upon the childhood cancer community as a result of these initiatives. Nonetheless, the simple fact that there are these potential opportunities to see gains is enough to warrant tempered optimism.
In order to understand where this optimism springs from, it is helpful to dig into some of these initiatives, if only just to scratch the surface, in order to balance out perspective. I am always reminded of my first soccer coach in college back in 1991. He was masochistic when it came to running and doing sprints. It was to the point that some practices felt more like a forced death march. What does this have to do with the point I am trying to make here? It is for one simple pearl of wisdom that he taught us as a team that stuck for all these years. His philosophy regarding optimism was simple: Never get too high, never get too low, and keep your emotions right in the middle. We only lost one game during the 1991 regular season. I suppose we kept our optimism in check. So, as I think through the current state of comprehensive care for children with cancer in the United States and beyond, and I try to overlay these new initiatives on top of this analysis, I am keeping myself grounded and my optimism at bay for now.
The Cancer Moonshot.
I’ve previously written about this and provided my cautious optimism. The details of President Obama’s program and Vice President Biden’s leadership on this initiative are being shaped daily and thus, what takes shape from here is a matter of waiting to see how everything develops. The early release from the Whitehouse regarding pediatric specific initiatives stated that the focus would be upon the following: “New technology to develop drug libraries and screens for inhibitors against a wide variety of targets will find new therapies, which will be of particular benefit for pediatric populations. The initiative will intensify efforts to collect and analyze tumor specimens from the rarest childhood cancers, enlisting participation from the pediatric oncology community. Clinical data about course of disease and response to therapy will also be included to enable the research community to develop new approaches to treat childhood cancers.” (https://www.whitehouse.gov/the-press-office/2016/02/01/fact-sheet-investing-national-cancer-moonshot). Since this declaration, the formation of a Cancer Moonshot 2020 project for pediatrics was announced. This multi-institution consortium will lead the focus on pediatrics. It is unclear the specific projects that will be part of this consortium (beyond those that are loosely outlined in the link); however, there is some muscle behind the consortium, and its collaborative effort provides a measure of hope that some of the pre-existing impediments to moving new, less toxic and more effective treatments to the clinic will be overcome. On the Cancer Moonshot project, I am personally providing a guarded but optimistic assessment overall.
Precision Medicine Initiative.
Precision medicine is a buzzword that is often used as a catch-all phrase. Simply stated in the context of cancer, or any disease with a molecular component, the goal of precision medicine is to provide individualized treatment on a patient-by-patient basis as a result of the underlying and unique genetic characteristics of the person’s cancer. The President’s Precision Medicine Initiative, announced in his State of the Union Address in January 2015 seeks to create a greater effort for researchers, clinicians and patients to work together to develop individualized treatment and care plans. As science is able to provide greater molecular analysis of every individual’s cancer, it is imperative that the days of “one size fits all” treatments are retired. The National Cancer Institute (NCI), through the National Institutes of Health (NIH) will be utilizing what they call the NCI-MATCH Program (Molecular Analysis for Therapy Choice). Through this program, patients (pediatric and adults alike) who no longer respond to standard therapies will be enrolled on other treatments selected through the underlying molecular analysis of the person’s cancer.
Another underlying effort rolled into the Precision Medicine Initiative is based upon the growth of big data and cloud platforms that are utilized to equip clinicians and researchers alike with greater availability of relevant information to make treatment decisions. These platforms are being built to house and manage the enormous amount of clinical data that may hopefully allow clinicians to ultimately select more effective therapies. Sharing information from bio-banks is also something that is a key necessity for such an initiative. In theory, this program should hopefully provide greater access to treatments that are selected at targeting the molecular drivers of the child’s cancer. As science and technology evolve, so too do the opportunities that exist for data sharing and what scientists can ultimately do with those data. In the end, I am once again cautiously optimistic that the Precision Medicine Initiative will have a positive impact upon the childhood cancer community that.
Thankfully, there are other initiatives upon which – with loud and strong voices – our community can make itself heard. The priority review voucher program established by the Creating Hope Act and then reauthorized by the Advancing Hope Act creates a system of incentives aimed at drug developers and manufacturers to reward them for investing in rare pediatric diseases. The fight to make this legislation permanent will come to a head as there is a sunset provision, (meaning that the law goes away if not permanently reauthorized) which takes effect on September 30, 2016. Through the priority review voucher system, the childhood cancer community saw the approval of the first pediatric cancer specific drug, Unituxin, in March 2015. Hand in hand, is the 21st Century Cures Act, which is being hotly debated on Capital Hill. Passed by the House, it now sits in the Senate and the fight to move it forward is significant. It goes without saying that this is a long and complicated piece of legislation that could potentially help the mission of the childhood cancer community through some of the initiatives contained therein. (For a brief summary see here.) Of course, in the end, whether or not a specific piece of legislation and the provisions contained therein ultimately has any impact upon children with cancer is a downstream question that remains to be resolved after passage and implementation. Thus, for this legislation, it remains too uncertain to come to any conclusions on whether to be optimistic or not.
The takeaway from this thumbnail analysis is that there are initiatives that could ultimately prove to be beneficial to the childhood cancer community. And, included in some measure is the issue of drug development and drug availability. In my opinion, this is the single greatest challenge facing the childhood cancer community. All the money in the world aimed at funding research, creating bio-repositories, sharing information and understanding the individual molecular drivers and targets for specific patients will have marginal impact at best unless there is significant investment, incentives and partnerships developed for the creation of new drugs for the multiple types of childhood cancer. And, more importantly, in some cases, drugs do exist for specific molecular targets and drivers present in children, yet children are not provided with access to those drugs because of the regulatory and investment framework set up to bring drugs to market and initiate clinical trials. Accordingly, there is much to be optimistic about; however we must understand that without significant focus and inertia for the development of new therapies and drug availability directed against known molecular targets, metastatic disease, relapses and recurrences, many forms of childhood cancer and the children impacted will remain waiting for a trip to the moon or a seat at the table to obtain a better outcome.
Jonathan Eric Agin is a childhood cancer advocate, speaker and an attorney (www.jonathanagin.com) in Washington, DC. He is married to Neely Agin and is the father of Alexis, Gabriel 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 is the Director of External Affairs for the Max Cure Foundation and the General Counsel for the Children’s Cancer Therapy Development Institute. Mr. Agin, along with his wife, run a Washington, DC chapter of a pediatric brain cancer foundation. In his spare time, Jonathan participates in endurance events, such as triathlons and running races to raise increased awareness for childhood cancer.