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OSUCCC – James Blog

To Beat Cancer, We Must Start Taking Risks

Dr Michael Caligiuri at AACR

This blog post was written by Dr. Michael Caligiuri, director of The Ohio State University Comprehensive Cancer Center and CEO of James Cancer Hospital and Solove Research Institute (OSUCCC – James); President-Elect, American Association for Cancer Research (AACR)


To make breakthrough progress in cancer research and cancer care, our nation must take risks. We must find and fund the bold new ideas put forth by top scientific minds and allow them to ask the question: Will this work?

Most cutting edge ideas are so novel they lack the data to justify a sizable grant from the traditional government funding model. It's a chicken-and-egg challenge, in order to get funding, you must have data, but to generate meaningful data you need a reliable funding source. 

In spite of the overall budget constraints and challenges, we are hopeful that U.S. House of Representatives and Senate will support a significant budget increase for National Institutes of Health (NIH) in fiscal year 2017. However, so much more funding is needed to support high-risk research and the high-talent scientists who will grow the field.

In order to make progress in cancer research, we must find new mechanisms for funding big, bold ideas. Such ideas potentially bring the highest risk because they are truly unique and, therefore, relatively untested, but they also carry the highest potential reward in accelerating the development of new treatments for patients.

While a tremendous force for medicine, thousands of researchers compete for a piece of the NIH annual budget, which since 2008 has barely fluctuated from an annual appropriation of $30 billion. Its National Cancer Institute (NCI), the primary funding agency for cancer-focused efforts, gets about $5 billion of that, a number largely stagnant since 2004. Out of the 12,000 grant proposals NCI will receive this year, only about 2,000 will get funds – and only a small portion of those are set aside to jumpstart those high-risk ideas.

Research progress toward understanding and treating cancer has been steady and important, but treatment breakthroughs can take decades to arrive at a patient’s bedside. This is simply unacceptable. 

Finding support for untested ideas

The private sector is currently trying to fill that void with varying levels of success. At The Ohio State University Comprehensive Cancer Center — Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC – James), we have been boosted by the tremendous support of our giving community. One example, Pelotonia, an annual grassroots cycling event, has generated more than $106 million for cancer research since the event began in 2009. 100 percent of all rider-raised dollars directly support cancer research efforts at the OSUCCC – James.

A significant portion of that money is allocated to fund Idea Grants — these are the projects that typically would not win traditional federal grants because they would have been deemed “too risky.” Any project idea can be submitted for funding, but using a rigorous peer-review process that involves investigators within and outside the institution to evaluate each application, only those that score the best in terms of being innovative and impactful are funded. Through the Idea Grant program, more than 80 projects have been funded to date in all areas of cancer research from drug development to immunotherapy to nanotechnology to precision medicine and beyond.

As an example, in 2012 and with Pelotonia funding, John Byrd, MD, a senior investigator at the OSUCCC – James was able to conduct the first clinical testing of a drug called ibrutinib in relapsed chronic lymphocytic leukemia (CLL), a very lethal form of leukemia in adults. The results were amazing – 80 to 90 percent of patients responded and went into remission. These initial results helped Dr. Byrd gain additional federal, industry and private funding for this important research culminating in the FDA approving ibrutinib for the treatment of CLL. Ibrutinib has converted a devastating form of blood cancer into a chronic illness that can be — for the most part  — managed with just one pill a day. Thousands of people are alive today because of this drug, and our team is working on a second-generation BTK inhibitor shown to be just as effective.

The fact that cancer patients nationwide are living longer with their disease or are disease-free is a testament to the power of innovative research and, in Ohio, many of them now ride in Pelotonia as survivors.

While the lives saved from such research is powerful, we long ago committed to supporting bold new research concepts knowing that this is where progress will be made. High-risk experiments can never replace the consistent, extensive and carefully vetted research that provides the foundation for American medicine, but the explosive breakthroughs that drive quantum leaps in science and drug development are too few, and too rarely funded.

We are committed to making this investment in bold ideas to fill the funnel of strong, meaningful cancer discoveries worthy of investment from the larger funding agencies. However, such private-sector efforts will always be limited. As a global cancer community we must be creative and strive to secure funding to support innovative research if we hope to achieve our vision of a cancer-free world.