Repurposing Rapamycin for 6 Autoimmune Childhood Diseases

Principal Investigator: Dr. David Teachey

Diseases: Autoimmune Lymphoproliferative Syndrome – ALPS; Evans Syndrome; Systemic Lupus Erthematosus; Autoimmune Hemolytic Anemia; Common Variable Immunodeficiency

Research Description: In 2004, Cures Within Reach funded Dr. David Teachey at Children’s Hospital of Philadelphia to repurpose sirolimus, a generic transplant drug, for a pediatric, ultra-rare blood disorder, Autoimmune Lympho-proliferative Syndrome (ALPS). In less than 36 months, Dr. Teachey demonstrated that the drug helped mice with this disease, and he showed the same with pediatric ALPS patients in a clinical trial: 85% who were treated were in remission after just 90 days on the drug. Many of these patients had failed other therapies and were slowly dying, but now many are still in remission years later with just two pills each day. Their healthcare costs have declined, and they can lead an almost normal life. Based​ on this success, CWR funded Dr. Teachey’s clinical trial testing sirolimus in five more diseases (Evans syndrome, systemic lupus erythematosus, autoimmune hemolytic anemia, and common variable immuno-deficiency) with similar results in 4 of the 5: 67% of kids with multilineage cytopenias went into remission.