Longeveron LLC, a Miami-based regenerative medicine company, has been awarded a $3.8 million Small Business Innovation Research (SBIR) grant from the NIH to continue clinical trials of a stem cell therapy aimed at treating age-related frailty.
Frailty syndrome is a defined geriatric condition that increases susceptibility to the health harms of age-related decline in tissue function. It is associated with muscular degeneration (both muscle weakness and loss of muscle mass [sarcopenia]), osteoporosis, and elevated rates of all-causes disability and mortality. Its etiology remains unclear, but is thought to be related to multiple factors including chronic inflammation and the accumulated effects of stress over the lifespan.
"With the aging of the United States population, Aging Frailty is a growing clinical and public health problem, requiring novel ways to avert this condition," said Marco Pahor, M.D., director of the University of Florida Institute on Aging, in a statement issued by the company.
Longeveron’s mesenchymal stem cell (MSC) product, derived from the bone marrow of young, healthy donors, yielded promising results in Phase I and Phase II clinical trials reported last year. The SBIR grant will be used to fund a Phase IIb trial aimed at assessing the safety and efficacy of infusions of Longeveron MSCs, with three endpoints: functional mobility and exercise tolerance; patient-reported physical function; and levels of the inflammatory cytokine tumor necrosis factor alpha (TNF-α), a biomarker of frailty syndrome. Dr. Pahor, quoted above, is a principal investigator of the planned study.
"The pathophysiology of Aging Frailty includes increased inflammation that can promote systemic tissue damage,” said Joshua Hare, M.D., co-founder and CSO of Longeveron. Mesenchymal stem cells have potent anti-inflammatory and pro-regenerative properties, and have the potential to meet a great unmet medical need by directly addressing the biology of aging. We believe a biologically-driven cell-based therapy could have a major beneficial impact."
NIH’s SBIR and Small Business Technology Transfer (STTR) programs, which allow small businesses to engage in research and development with strong potential for commercialization, are a major source of early-stage capital for technology commercialization in the US. In FY 2018, NIH’s SBIR and STTR programs will invest more than $1 billion dollars into health and life science companies developing aimed at improving health and saving lives.