Treatment of non-traumatic osteonecrosis with endogenous Mesenchymal stem cells
Femoral head osteonecrosis (aka avascular necrosis) is a disease caused by loss of blood supply to the bone, leading to bone cell death, end stage hip arthritis and total hip replacement. There is an unmet need for treatment of this disease, that affects individuals at prime of life (peak age 35 years). This small molecule therapeutic recruits bone forming cells to site of damage, where they serve the dual function of laying down new bone, and stimulating revascularization (new blood vessel growth) to prevent further bone cell death. Should this drug prove safe and and show signs of effectiveness, it may be tested for the treatment of other bone diseases like osteoporosis.
Randomized, double-blind placebo-controlled. Single and multiple ascending dose.
Safety, tolerability. Determine PK. Determine PD effects on bone turnover, biomarkers. Determine immunogenicity.