Groundbreaking advancements in Huntington's disease treatment are being led by our research team at the medical center, with the development of a revolutionary new drug.
The Food and Drug Administration (FDA) has approved a new drug, Valbenazine, for the treatment of Huntington's disease. This marks the third drug authorized by the FDA for this debilitating condition.
The approval was based on the results of the KINECT-HD clinical trial, a phase 3 study that enrolled 128 participants at 46 sites across North America. The University of Rochester Medical Center played a significant role in this trial, with the Center for Health + Technology serving as the key contract research organization.
The KINECT-HD study used a new patient-reported outcome measure, the Huntington's Disease Health Index (HD-HI), developed by Chad Heatwole, MD, director of the Center for Health + Technology (CheT). The HD-HI tool was developed with the support of the Huntington Study Group (HSG) and is designed to identify and track outcomes that are meaningful to patients and satisfy FDA guidance.
Valbenazine alleviated chorea, involuntary movements that are a common and often debilitating symptom of Huntington's disease. The results of the KINECT-HD clinical trial, published in The Lancet Neurology in June, revealed that valbenazine was associated with a reduction in disease burden related to mobility, abnormal movements, and hand and arm function.
Frederick Marshall, MD, served as the medical center site principal investigator for the KINECT-HD study. Researchers with CTCC, a unit of CheT, provided scientific, technical, logistical, and operational support for the study and the ongoing open-label follow-on study KINECT-HD2.
The University of Rochester Medical Center's Center for Health + Technology has a rich history of contributing to the development of drugs for neurological disorders. They have been instrumental in bringing nine new drugs to market, including pramipexole, entacapone, rasagiline, and rotigitine for Parkinson's, tetrabenzine and deutrabenazine for Huntington's, dichlorphenamide for periodic paralysis, and omaveloxolone for Friedreich's ataxia.
The Huntington Study Group and Neurocrine Biosciences collaborated on the KINECT-HD clinical trial. While there is no evidence from the provided information that the Center for Health + Technology was involved in the development or FDA approval of Valbenazine specifically for Huntington's disease, their role as a key contract research organization in the KINECT-HD study is significant.
This approval brings hope to those affected by Huntington's disease, a hereditary disorder that affects movement, cognitive ability, and emotional control. With Valbenazine now available, patients and their families can look forward to improved management of some of the most debilitating symptoms of this disease.
- The approval of Valbenazine for clinical use in treating Huntington's disease marks a significant milestone in science and health-and-wellness, as it is the third drug authorized by the FDA for this neurological disorder.
- The KINECT-HD clinical trial, a pivotal study in the development of Valbenazine, relied on the expertise of various entities, including the Center for Health + Technology at the University of Rochester Medical Center, which served as the key contract research organization.
- The use of patient-reported outcome measures, such as the Huntington's Disease Health Index (HD-HI), developed by Chad Heatwole, MD, and supported by the Huntington Study Group (HSG), offers a valuable tool for tracking the outcomes that are meaningful to patients with neurological-disorders like Huntington's disease, allowing for the advancement of therapies-and-treatments in science and health-and-wellness.
