ABC Transporter Pharmacogenetics: Impact on Thiopurine Therapy

Identifying common genetic variants of key thiopurine transporters to improve individualized treatment strategies for patients with inflammatory bowel disease

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Full Project Name:ABC Transporter Pharmacogenetics: Impact on Thiopurine TherapyPrincipal Investigator:Michael Stephens, MD, PediatricsCo-Investigator:Ronald Hines, PhD, Pediatrics; Gail McCarver, MD, PharmacologyAward Amount:$170,607

Award Date

October2005

Project Duration:24 months

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.

Outcomes & Lessons Learned:

Project successfully earned extramural funding to accomplish project goals within first three months of award. Therefore, AHW funds were withdrawn.