Principle Investigator: Dr. David Teachey
Disease: Autoimmune Lymphoproliferative Syndrome
Research Description: Autoimmune Lymphoproliferative Syndrome (ALPS) is a rare childhood autoimmune disease. Patients suffer from enlarged lymph nodes and spleen, increased infections and anemia. ALPS patients may spend as many as 5-10 days in the hospital each month and may not survive beyond their teens. Dr. Teachey tested the repurposed generic drug sirolimus, a drug used to prevent rejection in organ transplants, first in mice and then in a pilot clinical study among six patients at the Children’s Hospital of Philadelphia, in which five of the six patients were in complete remission in 90 days. Patient hospitalizations declined, and patients have continued to survive and thrive on sirolimus. The cost to treat these patients has also significantly decreased, resulting in lower medical bills.
CWR funding role: Participating funder
Publication: British Journal of Haematology
→ Data are supporting off-label clinical use
→ Findings from project led to additional applications of repurposed treatment in other pediatric autoimmune diseases.