Only five physicians have served as president of the University of Texas MD Anderson Cancer Center since its founding in 1941. Dr. Ronald DePinho, who took the reins on Sept. 1, 2011, spent the previous 14 years as a member of the Harvard Medical School faculty, where he founded and ran the Belfer Institute for Applied Cancer Science at the Dana-Farber Cancer Institute. Today, he faces a far larger challenge in running the Houston-based $4.4 billion institution with a 654-bed flagship hospital and $736 million research budget. Modern Healthcare Editor Merrill Goozner recently spoke with DePinho about the scientific promise and financial problems in cancer care today. This is an edited excerpt.
Modern Healthcare: Why did MD Anderson hire someone deeply engaged in the nuts-and-bolts element of the early stage of cancer research?
Dr. Ronald DePinho: I am a physician. I have a science background. I also have a business background. That, collectively, has enabled me to serve the institution. The field has now entered an era where the bench is at the bedside. When I started my career in the 1980s, the divide between what was going on in the laboratory and what was going on in the clinic was quite significant.
But as the technology came online to allow us to analyze what was going on in humans through molecular biology, genomics, computational biology, advances in imaging physics, we have entered into this era where the laboratory science and the clinical care are one and the same. In fact, the core principle of MD Anderson is research-driven patient care. Two-thirds of patients do very well with today's standard of care. But for one-third of patients, they will not do well. For them, the standard of care is clinical trials. MD Anderson has over 1,000 clinical protocols, and that provides our patients with the drugs of tomorrow. We don't know if they are going to work, but it provides them with access to potentially life-saving drugs.
MH: There has been an evolution toward personalized or precision medicine, based on the understanding that tumors are about the mutations, rather than the sites where cancer occurs. Is this a reality in day-to-day cancer care, or is this still a research project?
DePinho: I would say that (personalized medicine) is evolving toward standard of care. As a result of major investments in the National Institutes of Health over half a century, our knowledge of the disease has reached a point of maturity that has enabled us to develop strategies to directly address the root causes of the disease. And one of the most celebrated examples of this was CML (chronic myeloid leukemia) and Gleevec. That led to near-transformative results for patients with that disease. Now there are many more examples. We are not fully there yet, but we have many examples where knowledge reached a point where drugs were developed and where patients are now matched more effectively with those drugs.
About five years ago, there were a number of things that occurred that were disruptive in nature that allowed us to more aggressively apply this to clinical decisionmaking. The costs of gene sequencing went down exponentially. We gained the ability to aggregate data through big-data platforms and analyze those data through very powerful analytical tools. That has matured to a point where we can analyze somebody's genetic makeup … within a timeframe that allows us to make clinical decisions that would influence the care of the patient, or concierge that patient into the right clinical trial.
MH: Where we have targeted therapy now, the prices of the drugs are extremely high. It has created consternation for both payers and patients.
DePinho: This is a critical issue for our patients, and an issue we have to solve. The industry needs to look at this and decide how they can make sure that the cost of drugs is matched to the quality impact of that drug. At the same time, they need to preserve the capital to invest in the development of new drugs. This is a very complex issue that our government, industry and patient advocates all need to come together to solve.
We have a list of programs that enable us to reduce the financial burden of patients that come to us. We work on this at many levels, not just the cost of drugs, but scholarships for children, ability to pay for lodging, things of that nature. The financial toll that cancer extracts on patients and families is great, so we do many things to try to minimize the impact on the patients.
A component of this is what is actually driving the high cost of these drugs? Ninety-five percent of drugs entering into clinical trials in cancer ultimately fail. We are, in part, paying for the failures. So the question is, why do we fail? We have analyzed this in great depth and we have launched a program to address these problems.
In drug development, you have a relay race in which you have siloed activities where discovery, biology, and knowledge of mechanism occur in academia, while the drug development enterprise largely occurs in the private sector. Once a drug is developed, it is placed into another world, which is clinical trials and your clinics.
MD Anderson has developed a program to integrate biology, clinical expertise and drug development. We are hoping that is going to improve the success rates of drugs. If it is successful, it will significantly reduce the cost of developing drugs because you will kill the failures early.
MH: Much of early stage basic science is funded by government. Yet NIH budgets have not grown during the past couple of years. Are you able to bring other resources to bear to replace the diminished role of government?
DePinho: The NIH has been one of the great investments that the nation has had in its history. Over a half century of investment has led to whole new industries and insights where we now can make a decisive assault on the cancer problem. So it is very important for the nation to continue its robust investment in science.
Over the past 10 years, the NIH budget has not kept up with inflation. Essentially, its purchasing power has gone down 25% at the time when the opportunities to take advantage of knowledge and apply it more aggressively to save lives has never been greater. There are things that we can do to help attenuate the NIH decline. They may come in the form of philanthropy or commercialization revenue from intellectual property. But it is important to appreciate that those sources are not the same as NIH, which is peer-reviewed research and has the wisdom of other experts saying, “These ideas are great ideas.”