Earlier this week I had the opportunity to attend and participate in a conference "Turning the Tide Against Cancer Through Sustained Medical Innovation" in Washington DC. . The conference organizers brought together a stellar list of experts (present company excepted) to discuss the coming revolution in cancer care through personalized medicine, as well as the barriers and risks we face as science moves us forward towards what I consider a brave new world of cancer research and treatment.
With all of the intellect that was present at that meeting-and there was a lot-there was a theme that crystallized for me and others as the day progressed: we have developed incredible science and incredible opportunities to understand and treat cancer. But with all of the issues that have to be dealt with, the reality is that there is no singular leader-organization or individual-who has the clout and the heft to accelerate all the changes that need to happen if the vision of personalized medicine is going to be a success.
The progress we have made in the laboratory over the past 40 years has been truly incredible. As I have written previously, we have learned much about what makes a cancer cell a cancer cell. We have made the first steps in harnessing that knowledge to bring new treatments to our patients that are crafted to interfere with those internal cancer cell derangements, some having been more successful in meeting our expectations than others. We are moving into an era where traditional microscopic examination of cancer tissues will be relatively outmoded, and genomic analysis of cancers will be routine and become part of the decision-making process to guide us in choosing the best treatments for our patients.
All of this is true, and all of it is promising. But there remain many sobering realities that we have to face up to before we get to the promising land.
First and foremost, cancer is a complex disease with many ways to circumvent our discoveries and our treatments. Plug up something here, and something else pops up there. Again and again, cancer has dashed our hopes on the shoals of reality.
To move forward, we need to continue our research. But money is tight everywhere, and research budgets are being cut. It takes investigators a lifetime of work and discovery to lead us down new paths to success. But if we don't have the resources to fund our young investigators-and maintain our established research workforce-we will pay a significant penalty in years ahead.
Pharmaceutical companies and venture capitalists are being asked to make the investments in developing new drugs, engage in clinical trials, and bring new drugs to market. But the risks are mounting in these enterprises, and successes are becoming ever more expensive with a declining return on investment. As we slice and dice the cancer genome, we find that there are many individual variations in the genetic makeup of our cancers. The drugs that are developed will be useful in smaller and smaller segments of the cancer patient population. There comes a point when no one can afford to invest in the research and drug development and no patient can afford to receive a drug that relies on these outmoded processes.
Enter the universe of biomarkers, which hopefully will help guide us-as they have recently with such markers as HER2 in breast and other cancers, BRAF mutations in melanoma and ALK in lung cancer-to more precisely target patients who will benefit from our new drugs. If we can do that effectively, then we can get useful clinical trials with smaller numbers of patients and perhaps reduce the costs of development.
But additional barriers to research and drug development stand in the way of potential success.
Researchers and drug companies have traditionally been very proprietary when it comes to their research and their data. Keeping data in a silo, protected from the peering eyes of others, has been the rule. However, if we are going to take advantage of the new world, we are going to have to share data and ideas.
New models of research collaboration among experts around the country and around the world are going to have to become the norm if we are going to be successful. Data libraries of genomic data have to be designed, funded and made readily available. How we structure clinical trials will have to change. After all, finding those patients with a specific cancer and a specific genomic abnormality in that particular cancer is going to be increasingly difficult. No more herding in 50 patients with lung cancer at a major academic research center and try out a new drug. Now it will be a search for that patient with a specific genetic change in their cancer and hoping that-in addition to finding them-they and their doctors will be willing to participate in a clinical trial.
All of this means we need new information systems that are standardized and easily exchange information. It means we need to protect patient privacy but help patients understand that by allowing us to use their information we can advance our understanding of cancer treatments so much more rapidly than is the case even today. It means addressing deeply held beliefs and suspicions held by many that they don't trust the medical system to work for them, and provide them assurances their information is safe in the hands of researchers and others who want to understand more quickly how we can better treat cancer and its long term consequences.
Then there is the matter of costs.
Who is going to pay for all this? Not just the research and the researchers, but the clinical trials, the costs of the drugs, the support that our major academic centers need to advance our science and our knowledge, and the cost of caring for our patients.
Our healthcare system is undergoing major changes. I heard someone call the impact of all of these changes on patients and doctors a "waterfall." Perhaps "avalanche" or "tsunami" may be a more appropriate description of what is going on in cancer care.
There is a movement to get cancer care back into the community. But with the increasing complexities of diagnosing and treating cancer, how can we facilitate community care to be the best care? How do we make certain that there is fluid communication in the cancer care continuum to help patients get the best care wherever they may be? As accountable care organizations develop and are incentivized to keep that cancer care close to home, how do we support the missions of our cancer centers which include research, teaching, and providing complex care to complex patients who need that care (think bone marrow transplant or the treatment of acute leukemia or esophageal cancer as examples)?
As I mentioned at the conference, ultimately we have to keep patients at the center of our focus. For me, it was about access: nothing happens unless we can get patients in the door to get properly treated in the first place. As others reminded me, we have talked for years about putting patients in the center of our focus. Maybe the time has come for us to actually take that mantra seriously.
With so much going on, and so much change happening, who is in charge? Right now, for many of us, we feel like we are trying to build or fix the plane while we are flying it. The world around us is changing constantly, and with all the angst about how this impacts our everyday lives as patients and professionals, we are layering on top of that a whole new way of understanding medicine through personalization and the promise that it brings.
Can we make this work? Can we get everyone in line and on board? Can we make certain that we build in the right quality and ethical protections for our patients? Can we pay for it?
All of those are daunting tasks. There are so many moving parts and so many participants in this process that it is mind boggling to contemplate how we are going to make this happen.
One of the speakers at the conference was Dr.Siddhartha Mukherjee, the author of The Emperor of All Maladies: A Biography of Cancer (what an intellect, and what a surprise as I sat listening to him that much of his history of cancer was in fact part of my personal life experience and knowledge, which makes me feel old. But I digress...).
One of the points he made during his wonderful presentation was the fact that years ago a driving force behind advancing cancer research and care was a single individual named Mary Lasker. In a real sense, she was in fact a force of nature. She empowered researchers and Presidents-among others-to take cancer seriously, devote the funds needed for cancer research, and get treatments to patients that at the time were incredibly new, novel and powerful in their impact.
What we don't have today, Dr. Mukherjee opined, is another Mary Lasker. Where is our Mary Lasker going to come from?
That becomes a key and important question if personalized medicine is going to advance, for all the reasons cited above. Who is going to guide this revolution? Who is going to knock heads to make it happen? Who is going to make so many parts of this non-system of healthcare fed up with the status quo? Who is going to have the power to force us out of our respective comfort zones and make this happen?
Maybe that someone was sitting in the room with us on Tuesday, but I suspect not. That someone is out there somewhere. Maybe it's an organization like the various foundations and researchers who are trying to reform the way we do research and get new treatments to patients. Maybe it is organizations like the American Cancer Society and others who help people access needed care. Maybe it will be all of us who demand that things change, and that our interests as people be put front and center, before the self-interests of those who populate this process and have distorted our opportunities to get the best care we need when we need it.
Only through our own resolve to understand the potential, promise and possibilities of where we are headed with personalized medicine can we make this happen. Each and every one of us should demand the best of all who serve us in this brave new world of genomics, proteomics, and the personalization of cancer care. We need to break the barriers that are holding us back from getting where we need to go. The power of many can make a difference, and right now we need that power.