Using a Cancer Drug in Huntington’s Disease

Principal Investigator: Dr. Karen Anderson

Disease: Huntington’s Disease

Research Description: Huntington’s disease (HD) is a rare, inherited brain disease, which causes uncontrollable, fast movements, memory and concentration problems, emotional symptoms and cognitive decline. HD symptoms usually begin in a person’s third or fourth decade. Individuals with HD usually live about 15 to 20 years after diagnosis, and full-time care is required in the later stages of the disease. A Georgetown University research team has previously studied nilotinib, an FDA-approved cancer drug for the treatment of chronic myeloid leukemia, in a small, proof-of-concept study in people with Parkinson’s disease and found it to have an acceptable safety profile. They now propose a clinical trial with nilotinib in HD patients (subject to FDA approval). Over the course of a 3-month study, researchers will give nilotinib to 10 study participants with early to moderate HD, and will examine whether nilotinib is well tolerated and safe. They will also measure changes in biological markers and physical and emotional symptoms. This project has clinical importance and relevance due to limited current treatments for HD. Any potential new treatment resulting from this work would make an impactful difference for those living with HD and their families.

Funding Partners: Anonymous

CWR funding role: Primary funder