Clinical Research Forum honors studies with potential to benefit human health and welfare

Ten of the most outstanding clinical research projects from institutions around the country have been selected to receive the Clinical Research Forum's Annual Top 10 Clinical Research Achievement Awards. Each of these winning projects is a compelling example of the scientific innovation that results from the nation's investment in clinical research that can benefit human health and welfare.

Awardees hail from the Massachusetts General Hospital at Harvard Medical School, Yale School of Medicine, Columbia University, Johns Hopkins University School of Medicine, the University of North Carolina at Chapel Hill, the University of Pennsylvania, and the University of Pittsburgh Medical Center. "There's never been a moment in the history of biology that's more optimistic for spectacular breakthroughs to happen. However, it will require strategic investments at a most difficult time in our history," said William F. Crowley Jr., M.D., founder and past chairman of the Clinical Research Forum and director of the Clinical Research Program at Massachusetts General Hospital. "America is a world leader in biomedical research and if we are to retain that leadership role globally, we have to continue making these national investments."

All published in 2012, these studies are the latest in a long tradition of notable health advances that have occurred through clinical research - such as eliminating polio, reducing the mortality of AIDS, and improving cancer survival rates - that were propelled by combined investment in basic science and clinical research. Collectively, the work was funded by a range of federal agencies including the National Institutes of Health, the Veterans Administration, the Defense Advanced Research Projects Agency, as well as many foundations and corporations.

"These achievements are beacons of hope that show what can be accomplished when our nation's researchers are given the freedom and resources to tackle tough clinical problems," said NIH Director Francis S. Collins, M.D., Ph.D. "The opportunities for advancing clinical research have never been better. So, we at NIH look forward to doing everything we can to light up more of these beacons for the millions who look to us for help."

The projects, which tackled difficult health problems and required extensive collaboration, are remarkable for their bold approaches, innovation and potential for alleviating human suffering. In the area of disease prevention, the winning research has made an impact through improved implantable nanotechniques in the brain to deal with the consequences of stroke. This remarkable clinical research accomplishment will be honored with the Herbert Pardes Clinical Research Excellence Award, named for the executive vice chair of New York-Presbyterian Hospital's Board of Trustees and the hospital's former president and chief executive officer, a physician who is regarded as a champion and visionary in clinical research.

"The tripartite mission of excellence in research, medical education and patient care unites all scientists in academic medical settings in their pursuit of clinical research that will expand the boundaries of medicine, increase the ability to diagnose, treat and prevent disease, and offer patients and their families hope for the future," Pardes said. "I am delighted to be able to advance this vitally important mission and support clinical researchers as they pursue game-changing medical breakthroughs."

The other outstanding research projects include much wider availability of bone marrow transplants in patients with sickle cell, treatment to restore sight in visually impaired people, diagnosis by whole-genome sequencing in prenatal patients, new promise to alleviating paralysis from neurological illnesses via sophisticated prosthetic devices, definition of new genetic loci that presumably cause inflammatory bowel disease and related GI diseases, new treatments for patients with malignant melanoma, improved understanding of the use of diagnostic tests for osteoporosis, the ability to replace defective heart valves without open heart surgery, and refining the ability to identify genetic abnormalities in fetuses during pregnancy.

The studies were published in The New England Journal of Medicine, Science Translational Medicine, Blood, Nature, Lancet, and Cell. Winning researchers are being honored April 18 during the Clinical Research Forum annual meeting in Washington, DC, where they also will present their work.