Researchers at Scottish Rite for Children, Dallas, were awarded a $100,000 grant from the Department of Defense’s Neurofibromatosis Research Program. The grant will fund efforts to investigate new treatments for bone fractures in children with Neurofibromatosis Type 1 (NF1). Some children with NF1 develop orthopedic conditions requiring treatment, such as scoliosis and persistent bone fractures.
“Our team has dedicated years of research to understand why these persistent fractures occur in children with NF1,” says Jonathan Rios, Ph.D., assistant director of Molecular Genetics and lead investigator of the study. While several drugs are either approved by the FDA or currently in clinical trials to treat tumor manifestations of NF1, no such treatments exist for orthopedic conditions associated with NF1. “All of our team’s efforts have led to this moment, where we can now test new therapies in the lab. And by evaluating therapies already in clinical trials for other aspects of NF1, we hope to rapidly translate the most promising of these therapies to treat fractures in children with NF1,” Rios says.