Early Trial Offers Hope Treating Rare 'Brittle Bone' Disease
HealthDay reports on an early study published in the Journal of Clinical Investigation looking at disease mechanism and a drug’s potential to build bone density for the treatment of osteogenesis imperfecta (OI).
HSS pediatric orthopedic surgeon Cathleen L. Raggio, MD, who was not involved in the study, provided external perspective on the clinical implications of the findings.
Even though no medications are currently approved by the U.S. Food and Drug Administration specifically for the disease, Dr. Raggio explained that bisphosphonates, a mainstay of treatment for children with osteogenesis imperfecta, can increase kids' bone density and help them be more active.
According to Dr. Raggio, who researches and treats osteogenesis imperfecta, bisphosphonates are not specifically designed for OI, which means they target a symptom and not the disease process itself.
Dr. Raggio agreed that having medications that target a "pathway" in the OI process could "treat the core problem, rather than symptoms."
She noted she was "cautiously optimistic" about the findings but more work has to be done.
She explained larger studies of adults are needed to look at the safety and longer-term effectiveness of fresolimumab -- and to better understand why some patients may respond, while others do not.
Read the full article at Healthday.com.