Q: What are the challenges in brain cancer that Accelerate Brain Cancer Cure can help address?
The first and most obvious challenge is a shortage of research funding -- that is true for all disease-focused organizations. But it is not just about money. In brain cancer, biology itself presents an especially difficult challenge. The brain is contained and protected by both the skull and the blood brain barrier, making it very difficult to access. It also has many different cell types, all electrically connected, that work interactively to control our key bodily functions. This calls for uniquely specific and selective treatment approaches. Because patients with brain tumors often have life expectancies of a year or less, there may not be time for multiple diagnostic or therapeutic interventions, and this makes both treatment and research difficult. For drug companies it also means that there is not the same type of long-term market that might be available with a more chronic disease.
Q: ABC² has an entrepreneurial approach to medical research. How does it "buy down the risk" in the drug discovery process?
First, we work to identify the scientific and commercial challenges that serve as barriers to the discovery and development of new treatments. We then work not only with academic researchers but also with scientists at pharmaceutical, biotech, IT, and other companies to supply the resources (monetary and otherwise) that enable new approaches to address these. ABC² was built to take risks, to do things that might be so new or so difficult or so uncertain in outcome that corporate or government support might not be available. In essence, we are designed to be a Special Forces team for brain cancer. We bring unique resources to bear to make the newest new things come to life. We often then find that the results generated by this early work validate the idea so that further resources can more safely flow into the project. This has proven true not only with government research funding but also with corporate and private investment.
Q: Tell us about your focus on working with partners who share your vision.
Nobody can do this alone. The scientific, medical, commercial, cultural, political, and social challenges of bringing new treatments to life and into the marketplace for patients can't be addressed by any one group. You have to have good and trusted partners. We think that building and maintaining this network is perhaps the most important thing that we do. We see ourselves largely as matchmakers – using our resources and skills to bring people, companies, and institutions together to do things they would never have been able to do alone. The African proverb says "If you want to go fast, go alone. If you want to go far, go together." We have far to go — but we need to do it quickly. This means that we must have well-conceptualized, well-managed, high-trusting, high-functioning partnerships. Our mission is to identify, form, tend, and drive those partnerships. Teams win. We look to build good teams.
Q: What do you consider the greatest accomplishments of ABC²?
We measure ourselves by three metrics – Impact, Leadership, and Leverage. Did we change things for the benefit of patients? As Steve Jobs asked: "Did we dent the universe?" Did we lead in the opening of new frontiers, making it possible for others to follow? Did we bring extra resources to bear to solve problems by smart thinking, risk taking, creative structuring, and good partnering? This is a constant process and test, so it is hard to say that any one thing is a "greatest accomplishment," but here are some things we are proud to have had a hand in. We helped establish the first pre-competitive "head to head" screening initiative to evaluate and compare brain cancer drug candidates. We played a key role in bringing the first new drug for brain cancer in over a decade into the market. We helped bring The Cancer Genome Atlas project to life with glioblastoma multiforme as the first tumor type sequenced. We have worked with academia and industry to bring 11 new treatments into clinical trials, including new vaccines and immune therapies and new and repurposed drugs. We have enabled and built partnerships that continue to drive some of the newest and most important work being done in our field today. This is just a start, and there is so much more to do. I'm confident that our greatest accomplishment will actually be tomorrow.
Q: What are your top research goals in 2015, and what will it take to reach those goals?
We are strong believers in the use of the new tools of high-end molecular profiling to develop highly specific treatments and then use those targeted therapies to better treat patients. Many people talk about doing this, but the approach has yet to be broadly deployed for the benefit of patients. The work is early, the utility of such an approach is still being figured out, it is expensive, insurance doesn't yet broadly cover it, medical centers can't afford to cover the cost, many patients can't afford to pay for it on their own, and many physicians don't have the background to make full use of the information generated. We see this as a perfect place for us to operate. We are working with several key brain tumor centers to provide the funds, build teams, and develop a system that will use this approach in a broad-based and coordinated way. We believe that this will accelerate the creation of a more standardized and effective approach, improve research, enhance clinical trials, and improve patient care. It will take money, effort, good will, creativity, patience, and persistence to get there, but we are excited about the progress so far.