Principle Investigator: Dr. David Teachey
Disease: Autoimmune lymphoproliferative syndrome, Evans disease, systemic lupus erythematosus, autoimmune hemolytic anemia, idiopathic thrombocytopenic purpura, common variable immunodeficiency
Research Description: Children with autoimmune diseases that affect their blood cells require aggressive treatment with immunosuppressive drugs. These drugs are not very effective and have excessive side effects. Dr. Teachey previously investigated the use of sirolimus, a safe generic drug with few sides effects, to successfully treat autoimmune lymphoproliferative syndrome (ALPS). Based on these results. Dr. Teachey tested sirolimus in children with other autoimmune diseases. A total of 30 children were enrolled in the clinical trial, and sirolimus led to a response in 22 children. The results support sirolimus as a treatment option for children suffering from six types of autoimmune diseases. Sirolimus has the potential to improve quality of life and lower medical costs for many of these patients.
CWR funding role: Primary funder
→ Data are supporting off-label clinical use