Huntington’s Study Recognized for Potential to ‘Shape Medicine’

The journal Nature Medicine has identified a phase 3 study of pridopidine as a treatment for Huntington’s disease as one of 11 clinical trials that will shape medicine in 2022.   The URMC Clinical Trials Coordination Center (CTCC) is providing global operational support for the study, which is being conducted at more than 50 sites across the U.S., Canada, the U.K., and Europe.   

The journal notes that the PROOF-HD clinical trial is one of several ongoing studies of pridopidine as a potential therapy for Huntington’s, ALS, and other neurodegenerative diseases.  Pridopidine is an oral small-molecule that binds and activates the Sigma-1 receptor (S1R), which is present at high levels within the brain. By activating S1R, the drug helps boost production of brain-derived neurotrophic factor, a protein with neuroprotective properties.  These protein levels are found at reduced levels in people with Huntington’s disease. 

The PROOF-HD study is being conducted by Prilenia, the drug’s manufacturer, and the Huntington Study Group – a global network of more than 400 investigators, coordinators, scientists, and Huntington’s disease experts.  The CTCC has collaborated with the HSG on a number of clinical trials, including the First-HD study which led to the FDA’s approval of deuterated tetrabenezine for Huntington’s in 2017.  

The CTCC is providing scientific, technical, logistical, and operational logistical support for the PROOF-HD study, which announced in November 2021 that it had met its enrollment goal of 480 participants and is anticipated to run through April 2023.   Elise Kayson, M.S., R.N.C., A.N.P., director of CTCC Clinical and Strategic Initiatives, is serving as project lead for the PROOF-HD study.  Kayson is also co-chair of HSG.

The CTCC is part of the Center for Health + Technology and is a unique academic-based research organization with decades of experience working with industry, foundations, and governmental researchers in bringing new therapies to market for neurological disorders. Since its inception in 1987, the CTCC has played a central role in bringing seven new drugs to market to treat Parkinson’s disease, Huntington’s disease, and periodic paralysis.