Researchers at UC Davis Health System in Sacramento have received approval for a $20 million grant proposal to refine a stem cell therapy for osteoporosis patients and test its effectiveness in clinical trials over the next four years.
The state stem cell agency, the California Institute for Regenerative Medicine (CIRM), approved UC Davis' proposed project today as part of its overall Disease Team Grants program, which was established as a catalyst for obtaining U.S. Food and Drug Administration approval of clinical trials using regenerative, stem cell therapies.
Nancy Lane, professor of internal medicine and an expert on bone health, is leading a research team that will use a small molecule (LLP2A-Ale) it developed to direct endogenous mesenchymal stem cells to the surface of bone. The novel approach has the potential to promote new bone growth and provide an effective answer for men and postmenopausal women who suffer from osteoporosis, a condition causing increased bone fragility and often leading to fractures that significantly decrease quality of life.
"The only currently approved therapy for osteoporosis that increases bone formation requires costly and inconvenient daily injections for two years," said Lane, who also serves as director of the UC Davis Musculoskeletal Diseases of Aging Research Group. "I'm very optimistic that with our new Disease Team grant from CIRM, we will be able to successfully, and fairly quickly, develop a bone-building treatment for osteoporosis that only requires an occasional infusion."
Osteoporosis results from estrogen deficiencies and aging. It is the most common type of bone disease, and is expected to affect more than 61 million Americans by 2020. The disease occurs when the equilibrium between the formation of new bone and the reabsorption of old bone is disrupted in the body. In the U.S., 40 percent of women and 13 percent of men over the age of 50 will suffer an osteoporosis-related fracture in their lifetimes. Lane's grant proposal noted that California has one of the nation's largest over-age-65 populations and can expect to experience greatly increased fracture rates because of the disease.
In addition to research being done on UC Davis' Sacramento campus, the osteoporosis project also represents economic development opportunities for California businesses. The research team plans to contract with several companies to perform crucial preclinical studies that will ensure the small-molecule drug used to direct the bone-forming mesenchymal stem cells is safe. Another specialty company will synthesize and package the novel compound for clinical trials. And yet another firm will assist the disease team in working with the FDA when the clinical trial gets under way, which is expected in 2014. The private-sector partnerships are valued at approximately $3.5 million per year.
Lane, who received federal grants and philanthropic support during earlier stages of her osteoporosis research, said the CIRM funding is an important turning point in treating the disease.
"We've got a very detailed flight plan and pathway to clinical trials to validate a promising therapy for patients," Lane said. "This will advance our knowledge of osteoporosis and enable us to answer many key questions. I also hope to leverage the CIRM grant for additional support, which will also help us to address the challenges of bone disease and quality of life as we age."