Groundbreaking advancements made by our medical center's clinical researchers lead the path toward a novel Huntington's disease medication.
The Food and Drug Administration (FDA) has announced the approval of a new drug, Valbenazine, for the treatment of Huntington's disease. This development marks a significant milestone in the fight against this debilitating neurodegenerative disorder.
Valbenazine is only the third drug authorized by the FDA to treat Huntington's disease. 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 trial was a collaborative effort between the Huntington Study Group and Neurocrine Biosciences.
The KINECT-HD clinical trial used a new patient-reported outcome measure, the Huntington's Disease Health Index (HD-HI), developed by Chad Heatwole, MD. The HD-HI tool revealed that valbenazine was associated with a reduction in disease burden related to mobility, abnormal movements, and hand and arm function. Valbenazine was found to alleviate chorea, involuntary movements that are a common and often debilitating symptom of Huntington's disease.
The University of Rochester Medical Center (URMC) played a significant role in the approval process of Valbenazine. The Center for Health + Technology at URMC served as the key contract research organization for the KINECT-HD clinical trial. Cynthia Casaceli, MBA, and the Clinical Trials Coordination Center (CTCC) operational team members provided scientific, technical, logistical, and operational support for the study.
The CTCC, a unit of CHeT, is a unique academic-based research organization with decades of experience working in a regulated environment. Researchers with CTCC have played a central role in bringing nine new drugs to market, including those for Parkinson's, Huntington's, periodic paralysis, and Friedreich's ataxia.
Frederick Marshall, MD, served as the medical center site principal investigator for the KINECT-HD study. The results of the KINECT-HD clinical trial were published in The Lancet Neurology in June. The CTCC is currently managing the KINECT-HD study and the ongoing open-label follow-on study KINECT-HD2.
The University of Rochester Medical Center's contributions extend beyond the KINECT-HD trial. Through its Department of Neurology and translational neuroscience programs, URMC is actively engaged in clinical and translational neuroscience research that broadly supports the development of new neurological therapies. This research could potentially include diseases like Huntington's.
In conclusion, the approval of Valbenazine for the treatment of Huntington's disease is a significant step forward in the fight against this devastating disease. The collaboration between research institutions, pharmaceutical companies, and regulatory bodies is crucial in bringing new treatments to patients. The University of Rochester Medical Center, through its role in the KINECT-HD clinical trial and its ongoing neurological research, continues to play a vital role in this process.
- This approval of Valbenazine for Huntington's disease treatment, following the KINECT-HD clinical trial results, signifies a significant advancement in science, particularly in medical-conditions like neurological-disorders.
- The Health-and-wellness sector witnessed another landmark achievement with the approval of Valbenazine, as it is only the third drug authorized for Huntington's disease treatments, demonstrating the efficacy of therapies-and-treatments in addressing such medical-conditions.
- The University of Rochester Medical Center, through its active involvement in the KINECT-HD clinical trial and the ongoing research in neurological therapies and translational neuroscience programs, continues to contribute significantly to science and health-and-wellness, especially in the development of treatments for neurological-disorders like Huntington's disease.
