December 9, 2013
Welcome to Engaging Innovation! This multi-media forum will be a venue for leaders from the public and private sector, regulators, drug developers, researchers, providers, patients, and advocates to engage on important issues and discuss the most pressing challenges and the most compelling innovations in the fight against cancer. We are thrilled to have Dr. Francis Collins starting off the first of many conversations that we hope will spark further dialogue and innovative ideas.
Francis Collins is the Director of the National Institutes of Health (NIH), a Presidential Medal of Freedom and National Medal of Science Laureate, a geneticist, a physician, a bestselling author, and an amateur singer-songwriter. We asked him to talk about the future of cancer and biomedical research in the United States.
"Revelations about individual differences"- Francis Collins on Personalized Medicine
For most of medicine’s history, we’ve been doing one-size-fits-all because it’s the best we could do. But now the revelations about individual differences are making it possible to think about a much more personalized approach. In some people’s minds, that’s about drug prescribing—getting the right dose of the right drug. But I think actually the leading edge of this is cancer. We are learning that every cancer has a different set of drivers causing that good cell to grow when it should have stopped, and that set of drivers is predictive about how that cancer will respond to therapy. With an increasingly long list of targeted therapeutics where we know what pathway those drugs go after, we have the opportunity to begin to do a matching between what that individual’s tumor seems to be driven by and what drugs we have to counteract that. That is precision medicine that we have hoped and dreamed for and it is now coming true and cancer is in the lead.
"Scientific opportunities have never been more compelling. . .but the threats to the resources to support that research have never been greater" - Francis Collins on Sequestered Research Funding
The biomedical research community is living a paradox. Scientific opportunities have never been more compelling, especially in cancer research. And yet in the United States, the threat to the resources to support that research has never been greater. We have to marshal every resource we can to make the case for why this is a valuable investment for the public and for the economy. But we also have to make choices, some of them quite difficult, about how to continue to push forward cutting-edge, innovative approaches, even if it means tightening our belts even more so in other areas that are still highly productive but not quite at that same level of innovation. This is painful, and it is particularly painful for young scientists. We really have to make the case that if we want to see these advances occur and all the ways that is going to play out and benefit human health, we have to preserve that most important resource, which is the human talent that is going to make this come true. Right now that talent is pretty anxious. We need to figure out how to get through what has been a very difficult chapter and into a place with more stable support. Otherwise, I fear that a lot of the promises that are right in front of us will be delayed much more than they should be for the patients that are waiting.
"An absolutely essential priority" - Francis Collins on Interagency Cooperation and Public-Private Partnerships
NIH sees the need for partnership as an absolutely essential priority. Both scientifically and because of research constraints, we have close connections at the senior level with CMS, with FDA, with CDC. [Friends Founder and Chair] Ellen Sigal, by the way, is a significant part of many of those conversations. We also have much closer connections with the pharmaceutical and biotech industry in terms of pooling efforts in the pre-competitive space to try to move the agenda forward for identifying the next generation of drug targets so that we can hurry up the process of getting those new therapeutics out there without such a terribly high failure rate. All of that is happening, but it will not completely solve the shortfall in resources that we’re currently experiencing, which has really slowed down many of the things we’d like to do with or without partnerships.
We’re fortunate, at NIH, to have the Foundation for NIH as a means of serving to bring together public-private partnerships, manage projects, and figure out how to handle the finances. Ellen’s role as a member of the board of the Foundation of NIH has been essential to the strength of that institution.
"Figuring out how to get the right drug to the right person at the right dose at the right time" - Francis Collins on Friends of Cancer Research
Friends of Cancer Research has its very strong, intentional goal towards bringing new opportunities for cancer treatment to patients as quickly as possible, and that’s a very complicated ecosystem that not everybody understands. Friends of Cancer Research not only understands it, but knows how to bring parties together to make things happen in a shorter timeframe with a better outcome than otherwise would happen. When you think about all of the things that go together between basic discoveries about the causes of cancer—which are proliferating right now, and we’re really glad about that—but trying to take that forward to the development of new therapeutics and to figure out how to get the right drug to the right person at the right dose at the right time--that’s an enormously complicated challenge. It’s one that people are waiting to see successfully undertaken and Friends of Cancer Research is right in the middle of that. I think of the Master Protocol that is currently under development for lung cancer, which is really a brainchild of Ellen and Friends of Cancer Research, and will, I think, set a standard for how we want to conduct this sort of precision medicine for cancer going forward.
"If you're a researcher, how has sequester begun to affect you?" - Francis Collins Reaches Out Over Twitter
So I’ve had a Twitter account for a little over a year and I do tweet, pretty much several times a week, usually about new developments in science, trying to spread the word about what an exciting time this is. But back in May, I did send out a tweet to my roughly 20,000 followers saying: ‘Tell me your stories about what’s happening. If you’re a researcher, how has sequester begun to affect you?’
I didn’t know what I would hear because sequester was still pretty new and there is a delay factor between when something like this happens—namely the loss of $1.5 billion to the NIH budget—and its consequences to people out there. But it was clearly already being felt. I got more than 2000 responses and they were heartbreaking in many instances. The ones that bothered me most were from young scientists, some graduate students or post-docs or junior faculty. They wrote about such things as having to decide not to do the definitive experiment to really nail down a conclusion that they were pretty close to, but that new definitive experiment was going to be a little more expensive and their grant had just been cut.
I got notes from people who had been offered a position in a laboratory as a post-doc and just had that position rescinded because the principal investigator heard their grant was not going to get funded because of the sequester and they couldn’t afford to bring on a new person, even though there was a great project there to be done.
I heard about people who were running clinical trials who said they were going to have to shrink the clinical trial they had planned to run and they were worried about whether it would still be large enough to give a compelling conclusion or whether it would be just on the edge of statistical significance and therefore the whole trial might end up being a waste because you couldn’t really say what happened.
And I heard many other sort of heartbreaking stories from people who looked at the situation and began to wonder about their own futures and whether they should think about doing something else or continuing science in some other country. We ought to take those concerns with great seriousness. A recent survey done by ASBMB (the American Society for Biochemistry and Molecular Biology) indicates that 18% of the scientists they polled were considering moving to another country to do research. That’s frightening when you consider how much we depend on that talent to drive our own country’s advances forward. Other countries have looked at our success story over the last 50 years and they’re expanding their support for biomedical research. We alone out of the most well developed, high income countries seem to be going the other way. It’s very puzzling and potentially very destructive to our future.
"We are going to come up with answers to mysteries that have puzzled us for all of human history" - Francis Collins to Aspiring Scientists
To a young person thinking about getting into biomedical research, I would say “Come on! It’s going to be a great ride!”
The science that we have the opportunity to do now is breathtaking. We are going to come up with answers to mysteries that have puzzled us for all of human history, and to be part of that adventure is going to be a wonderful way to spend your life.
Yes, we are under stress right now. But I am optimistic that this is a nation that ultimately, as Winston Churchill said, decides to do the right thing after exhausting all the other options. It’s about time to get to that point now and put ourselves back on a stable track to do something that really has benefited our nation dramatically over the last 60 years, whether it’s in advances in longevity and health or stimulating our economy—namely, supporting biomedical research. We’ll get there, so people shouldn’t be too anxious about the current stress on the system. I can’t imagine that this will go on indefinitely. At the same time, the sooner we can get past it, the sooner I’ll be able to say with more confidence that this is a great way to come and spend your career.
Right now, I’m mostly concerned about people who are already involved, particularly scientists who are just at the beginning of their productive phase. They’re independent researchers, they’re starting their labs, they have great ideas, they are fearless and taking on risks, and yet their chances of getting funded if they come to the NIH looking for grant support is at its lowest in history—about 15%. Sequester plus ten years of flat budgets have done that. We need to do better for them. We need to give them a chance to flex their intellectual muscles and come up with those answers that we’re all hoping for.
We still have the greatest universities in the world and those universities are absolutely a wonderful place to do the best biomedical research in the world. We have such a great situation and such a great track record, and yet it’s all under threat.
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