Among the many movement disorders treated at UC Health, Huntington’s disease (HD) stands apart. Described as a “tragic inheritance,” it is autosomal-dominant, which means that a child of a parent with Huntington’s has a 50 percent of inheriting the gene and, inevitably, the progressive neurological disease.
But this May – HD Awareness Month – families affected by Huntington’s have a milestone to celebrate with the U.S. Food and Drug Administration’s recent approval of a new drug for HD. The James J. and Joan A. Gardner Center for Parkinson’s Disease and Movement Disorders was one of 34 sites across North America and Australia whose research led to the drug’s approval.
Deutetrabenazine, to be marketed as Austedo, is only the second drug approved for use in the United States for the treatment of chorea, the involuntary jerking, dance-like movements that are a debilitating symptom of HD.
“This drug provides a significant reduction in the chorea associated with Huntington’s,” says Andrew Duker, MD, a movement disorders specialist with the Gardner Center and principal investigator of the Gardner Center’s portion of the Phase 3 trial, known as First-HD. “It has been shown to be helpful in reducing the extra movements associated with Huntington’s disease, and when compared to a placebo it showed a very good response.
“It gives us an additional treatment option. It’s an important step forward toward developing new medications and therapies and putting Huntington’s disease research on the radar of the FDA.”
Hilary Perez, PhD, who began coordinating the study for the Gardner Center in July 2014, says the drug is closely related to another drug that is already on the market for people with HD. “This is a longer-acting version,” she says. “Patients will be able to take it once or twice a day, versus three times, and hopefully have an equal or even greater response.”
The First-HD study was conducted on behalf of Teva Pharmaceuticals through the Huntington Study Group, a non-profit network of experts from more than 100 medical centers throughout North America, Europe and Australia, and the University of Rochester’s Clinical Trials Coordination Center. A second deutetrabenazine trial, ARC-HD, which has completed enrollment, is investigating the drug’s long-term safety as well as how individuals fare when they switch to it from a different drug (tetrabenazine).
The Gardner Center, a regional destination for patients with movement disorders, is part of the Huntington Study Group and has enrolled patients in both the First-HD and follow-up ARC-HD trials. “The Gardner Center is proud to be a part of the Huntington Study Group and committed to advancing knowledge about and new treatments for Huntington’s disease,” Duker says.
The Gardner Center treats approximately 100 patients with Huntington’s at any given time. The condition affects 5 to 10 of every 100,000 individuals, but the incidence varies widely by ethnicity. It is highest among people of western European descent and virtually non-existent (one of every million) among people of Asian or African descent. HD usually manifests in people in their 30s and 40s, though some people are affected as early as childhood and others experience disease symptoms much later in life. The disease brings with it an array of symptoms besides chorea, including dystonia (involuntary muscle contractions), cognitive problems, changes in personality and depression.
The Cincinnati area support group for HD caregivers, patients, and at-risk individuals meets every third Saturday at The Beechwood Home, 2140 Pogue Avenue, Cincinnati, OH 45208 >
Mary Beth Bialick, social worker for the Ohio Valley Chapter of the Huntington Disease Society of America, expressed her thanks for the patients and families who made the approval of deutetrabenazine possible. “HD families are extraordinarily enthusiastic in their willingness to participate in research,” she said. “They are generous with their time and commitment and enthusiasm for all research.”
Teva Pharmaceuticals owns the rights to develop and sell deutetrabenazine in the United States, following its purchase of Auspex Pharmaceuticals in 2015. Deutetrabenazine is an investigational, oral, small-molecule inhibitor of vesicular monoamine 2 transporter, or VMAT2, that was granted Orphan Drug Designation for the treatment of HD by the FDA.
Teva is also investigating the potential of deutetrabenazine to treat tardive dyskinesia, a disorder that causes involuntary and repetitive movements, and for tics associated with Tourette syndrome.
— Cindy Starr