ABC Transporter Pharmacogenetics: Impact on Thiopurine Therapy

Project Description Narrative:

Thiopurines are important therapeutics used in adults and children in the therapy of many diseases, most notably in the maintenance therapy of acute lymphoblastic leukemia and such diseases as inflammatory bowel disease, auto-immune hepatitis, and in solid organ transplant recipients. Thiopurines have significant variation in the body as well as high rates of treatment failure and toxicity. Understanding the mechanisms that create variability, including genetic differences and developmental changes, is essential to improving treatment strategies.

Through this award, investigators will aim to identify the common genetic variants of key thiopurine transporters and explore their impact on thiopurine pharmacology in patients with inflammatory bowel disease in order to help improved individualized treatment strategies for patients.