Repurposing a Blood Cancer Treatment to Treat Muscle Contractures in Cerebral Palsy Patients
Principal Investigator: Dr. Richard Lieber
Disease: Cerebral palsy
Research Description: Cerebral palsy (CP) is the most common cause of childhood motor disability, affecting approximately 1 in 600 infants, with approximately 17 million affected people worldwide. Muscle contractures affect about one-third of CP children and cause loss of movement, decreased strength, and pain. While a variety of treatment methods such as splinting, exercise and stretching may slightly reduce disability, they do not prevent or resolve the underlying contracture. The research team recently showed that muscles from children with CP have lost most of their stem cells and the stem cells that remain cannot develop into normal muscle. In addition, the team has showed that these stem cells can return to “normal” when treated with the FDA-approved cancer drug 5-azacitidine (AZA). This clinical trial will establish the dose of AZA that is safe and effective in CP patients and could lead to a new nonsurgical treatment of muscle contractures in these children.
Funding Partners: The Searle Funds at The Chicago Community Trust
CWR funding role: Primary funder