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A lesson in perseverance
The happy occasion of the first edition of Vector magazine coincides with a period of reflection about the life and contributions of Judah Folkman, MD, the former director of Children's Hospital Boston's Vascular Biology Program. Dr. Folkman, who passed away on January 14, was the embodiment of research at Children's for nearly 40 years. His vision, first outlined in the New England Journal of Medicine in 1971, was to find a way to treat tumors by cutting off their blood supplies. He reasoned that tumors could only grow larger if they were able to recruit new blood vessels to nourish the new tissue, a process called angiogenesis. Consequently, finding drugs to inhibit tumor blood vessel growth should also halt the growth of the tumor.
When Dr. Folkman applied for his first grant in the 1970s based on this hypothesis, reviewers at the National Cancer Institute summarily turned him down. But after four decades of tireless work, his vision is being realized. More than 1.2 million patients worldwide are receiving anti-angiogenic therapy, not just for cancer, but for other diseases involving abnormal growth of blood vessels, including the blinding eye diseases macular degeneration and diabetic retinopathy. More than 10 angiogenesis inhibitors have been approved by the Food and Drug Administration, and nearly every major pharmaceutical company has a program to discover more.
Dr. Folkman lived only 74 years, but he worked so hard and thought so quickly that he seemed to accomplish twice as much each day as the rest of us. He essentially invented the field of angiogenesis research, and his undying scientific curiosity spawned many new lines of inquiry, some of them in unexpected areas. A true physician-scientist, he never forgot his main goal of improving the lives of patients. Day or night, he was never too busy to talk with patients, instilling hope and confidence that there were doctors who cared about their disease and were working furiously to defeat it.
We who worked with Dr. Folkman quickly learned that the best way to get his attention was to still be in the lab after midnight. That was our best chance for a long conversation, an encouraging word about our research or a general piece of advice about life from a superb mentor. The most important thing he told us was that no idea is too outrageous to be pursued. If others don't believe in your work, or in your ideas, he said, it's only because they haven't caught on yet; it is your job to do impeccable research that is so convincing, it can't be denied. Michael Rogers, PhD, and Robert D'Amato, MD, PhD, profiled here, are just two of many scientists given the room by Dr. Folkman to pursue unconventional ideas.
25% Chances an investigator will have an NIH Research Project Grant (RO1) renewed
8% Chances an investigator applying for a first R01 grant will be successful
1 Rank of Children's Hospital Boston among U.S. pediatric research centers, by amount of NIH research funding
5 Rank of Children's, among all independent U.S. hospitals, by amount of NIH funding
1,425 Number of scientists and scientific support staff at Children's
7 Laboratory space in active use, in acres
Our call is to continue this legacy, nurturing the next generation of medical researchers to think and work and find cures in the same way that Judah Folkman did. We are lucky at Children's Hospital Boston to attract terrific young scientists with deeply creative ideas. We also benefit from a hospital leadership committed to the importance of research, under the direction of President and Chief Executive Officer James Mandell, MD, and Chief Operating Officer Sandra Fenwick, and from an environment where scientists and physicians work side by side to ask and answer the most important medical questions.
Getting research funding has never been easy, but the next generation of scientists faces a particularly great challenge. Historically, the National Institutes of Health (NIH) has provided the great majority of the support for medical research in our country; getting more funding used to mean writing another NIH grant. Today, with fewer dollars being directed to the NIH, many of these grants have less than a 10 percent chance of being funded. Since it can take more than a month of effort just to write one NIH grant proposal, and multiple grants to run one laboratory, scientists are diverting more and more time to writing grants—time that could be better spent doing research.
To stay viable, research institutions are finding new sources of support and educating their young scientists to do the same. Much of the research in these pages comes from non-NIH sources. They include other federal agencies, such as the Centers for Disease Control and Prevention, the Department of Defense and a variety of environmental agencies and large foundations, such as the American Cancer Society and the American Heart Association. Biotech and pharmaceutical companies are also stepping up to support hospital-based biomedical research. With the hiring of Erik Halvorsen to direct our new Office of Technology and Business Development, Children's will cultivate more of these partnerships. And individuals and private foundations are supporting more biomedical research than ever before. This past year, 13 percent of the money spent on research at Children's came from philanthropy—our second largest funding source.
Often, the support that matters most is for our riskiest projects—the projects whose grants are routinely rejected because the work is at "too early a stage" or runs counter to established dogma, yet could spawn the next generation of treatments for patients at our hospital. In short, projects based on ideas much like Judah Folkman had nearly 40 years ago.