Drugs benefit Huntington’s patients’

image: Erin Furr Stimming, MD, professor of neurology at McGovern Medical School at UTHealth Houston and director of the Huntington’s Disease Society of America Center of Excellence at UTHealth Houston Neurosciences.
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Credit: Photo by UTHealth Houston

According to a recent international study conducted by UT Health Houston researcher Erin Furr Stimming, MD, who served as principal investigator on behalf of the KINECT-HD Huntington Study Group.

Fur Stimmingprofessor of neurology at McGovern Medical School at UTHealth Houston, presented results of the phase III, randomized, double-blind, placebo-controlled KINECT-HD study for the first time last week at the annual meeting 2022 from the American Academy of Neurology in Seattle, Washington.

She reported that valbenazine, a selective vesicular monamine transporter 2 (VMAT2) inhibitor that is not yet approved by the US Food and Drug Administration specifically for Huntington’s disease, was safe and effective when it was given once a day for the treatment of chorea in patients with Huntington’s disease. sickness. Chorea is involuntary, irregular movement and the cardinal motor feature of Huntington’s disease.

“We are delighted that this study has yielded positive results. We found a statistically significant decrease in Huntington’s disease-related chorea in patients taking valbenazine compared to placebo,” said Furr Stimming, who is also director of the Center of Excellence for the Huntington’s Disease Society of America. at UTHealth Houston Neurosciences. “Although chorea is not the only symptom experienced by our patients with Huntington’s disease, it can be quite problematic and can contribute to impaired daily activities like eating and drinking, and can even lead to frequent falls.”

Interestingly, a majority of people with chorea do not receive medication for the symptom, according to a separate study. abstract also presented by Furr-Stimming, who retrospectively analyzed data from the Enroll-HD dataset, a global observational study that includes more than 20,000 participants on four continents. Chorea was documented in nearly 97% of participants; however, only 36% of these participants were prescribed medication for chorea, despite the availability of FDA-approved medications such as VMAT2 inhibitors and off-label medications such as antipsychotics and benzodiazepines.

Furr Stimming said continued research is needed to better understand the optimal treatment regimens for chorea in patients with Huntington’s disease.

“We do not yet have a modifying treatment for Huntington’s disease, and therefore we must continue to focus on optimizing symptomatic treatments for the motor and non-motor symptoms of this disease,” she said. declared.

Huntington’s disease is a rare inherited condition that usually begins between the ages of 30 and 40 and causes nerve cells in the brain to break down over time. About 40,000 Americans have the deadly disease, while another 200,000 are at risk of inheriting the disease. There is no cure, but medications and physical therapy, speech therapy, and occupational therapy can help manage symptoms.

The KINECT-HD study was conducted by the Huntington Study Group, a global non-profit organization, in conjunction with its wholly owned for-profit subsidiary, HSG Clinical Research, Inc., and in cooperation with the Coordinating Center clinical trials from the Center for Health and Technology at the University of Rochester Medical Center, with funding from Neurocrine Biosciences, a biopharmaceutical company.


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