NIDCR Grand Rounds: Women in Science - Targeted therapy for fibrous dysplasia/McCune-Albright syndrome: Lessons learned and new approaches

Fibrous dysplasia/McCune-Albright syndrome (FD/MAS) is a mosaic disease resulting in fractures, pain, and disability. G α s mutations disrupt differentiation of skeletal stem cells, leading to marrow fibrosis and increased bone-resorbing osteoclasts. The resulting bone develops one or many expansile, deforming lesions. There are no effective treatments for FD/MAS, and clinical studies have been challenging due to knowledge gaps in pathogenesis, natural history, and clinically relevant endpoints for clinical trials. Anti-resorptive therapy with bisphosphonates has been advocated due to prominent osteoclastogenesis in FD tissue; however, studies have failed to demonstrate a clear benefit. Emerging evidence indicates that RANKL, a central mediator of bone resorption, may play a role in FD pathogenesis by promoting both bone resorption and skeletal stem cell proliferation. RANKL inhibition with the monoclonal antibody denosumab may represent an exciting potential therapeutic strategy to treat patients and prevent lesion development.Air date: 9/25/2020 11:00:00 AM
Source: Videocast - All Events - Category: General Medicine Tags: Upcoming Events Source Type: video