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Oxford-Harrington Rare Disease Scholar Award
Development of a Novel Therapy for Osteoporosis
2015 Harrington Scholar-Innovator
David R. Clemmons, MD, is one of those fortunate people who discover their direction in life early and never veer from it. Now an endocrinologist and the Sarah Graham Kenan Professor of Medicine at the University of North Carolina at Chapel Hill, he was a young medical student there when he first experienced what would become his lifelong love of medical research. “I dropped out of medical school between my second and third years to do research in endocrinology, and that was it,” Dr. Clemmons recalls. “I knew then that research was it for me.”
The pathway to the osteoporosis project that has earned him a Harrington Discovery Institute Scholar-Innovator award has been less direct. Call it serendipity, a side effect or an accident, Dr. Clemmons discovered the osteoporosis drug he is developing when he was researching diabetes.
“We were studying diabetic nephropathy [kidney disease] in mice when we noticed that their bones were very thin and would break easily,” he explains. Those fragile bones led Dr. Clemmons and his team eventually to discover a peptide (a short amino acid chain) that plays a role in stimulating bone formation and inhibiting bone resorption.
For Dr. Clemmons that was an exciting revelation. He immediately saw the potential application in osteoporosis, a possibility that spoke to his heart as well as his mind. In that peptide, Dr. Clemmons envisioned hope for the elderly women with osteoporosis he sees every week with their pain, difficulty moving, broken bones and fractures that occur simply because their bones are so weak. “These are the very patients I want to help with this discovery,” he says. Among all the currently available osteoporosis drugs, only one regenerates bone, and it has limitations, he adds.
The Harrington Discovery Institute grant will help Dr. Clemmons and his team validate their discovery in laboratory animals and optimize the peptide structure. From there, a French pharmaceutical company has expressed interest in identifying the best form for the drug and moving it through the toxicology studies and other testing needed to advance it to clinical trials.
Although clinical trials are at least three years in the future, Dr. Clemmons already is looking forward to them. “Seeing those results is what keep me going and energized,” he says. “I can live for a week on the positive results of a clinical trial, knowing that a discovery will make a difference in people's lives.”
The osteoporosis drug would give Dr. Clemmons a trio of discoveries in clinical trials. His other successes, a monoclonal antibody for diabetic nephropathy and a compound that inhibits coronary artery disease in diabetes, are currently in clinical trials.
Dr. Clemmons is modest about his achievements as a physician-scientist, but he is quietly proud of what could be his legacy. “If all three compounds make it as therapeutics,” he considers, “that would be a pretty good accomplishment.”
“I can live for a week on the positive results of a clinical trial, knowing that a discovery will make a difference in people's lives.”