Osteonecrosis or bone death is caused by reduced blood supply to a small part of the bone. Osteonecrosis is often caused by use of glucocorticoids, medications that reduce inflammation because these medications reduce bone formation and the blood supply to the bone. Our laboratory developed a compound, LLP2A-ale that was found to direct mesenchymal stem cells to the bone surface and these cells differeentiated into bone forming osteoblasts. and stimulated osteoblasts that were present at the bone surface to form bone. In addition, our preclinical studies found that mesenchymal stem cells that were directed to the bone surface by LLP2A-ale also, increased the size and number of blood vessels in the bone. Since glucocorticoids reduce bone blood supply and bone formation, and LLP2A-ale was able to reverse this by directing mesenchymal stem cells to the bone, we tested if LLP2A-ale treatment was safe and possibly effective in study subjects that were currently treated with low doses of glucocorticoids and had low bone mass.
During this award period we analyzed the clinical study data. We found LLP2A-ale was safe and well tolerated in study subjects with osteopenia and currently treated with low dose prednisone. However, the exploratory endpoints of change in bone mass and biochemical markers of bone turnover were not different between LLP2A-ale and placebo treated subjects.