Neuroscience Institute at Hackensack University Medical Center Studying Experimental Treatments for Charcot-Marie-Tooth DiseaseGoal Is to Gain Insights to Enhance Diagnosis and Treatment of This Degenerative Nerve Disease
The Hackensack University Medical Center Charcot-Marie-Tooth (CMT) Hereditary Neuropathy Center, led by Florian P. Thomas, M.D., Ph.D., Founding Chair and Professor, HUMC Department of Neurology & Hackensack Meridian School of Medicine, has started enrolling patients in the international, multicenter PXT3003-06 clinical trial for the treatment of Charcot-Marie-Tooth Disease type 1A (CMT1A), the most common subtype of hereditary neuropathy.
With his team of neurologists, rehab therapists, research pharmacists and research coordinators, Dr. Thomas is trying to establish whether an experimental polydrug consisting of three ingredients can improve and/or delay progression of arm and leg weakness and sensory changes for this condition that affects some 70,000 people in this country.
While there are no FDA-approved treatments for CMT, this is the third such study that Dr. Thomas is involved in, having been the national lead investigator for the two first Phase II & III clinical trials of pharmaceutical agents to treat CMT disease in the U.S. since 2015. Dr. Thomas is an international leader in the treatment of CMT disease, and he has more than 30 years of both clinical and research experience with patients diagnosed with CMT.
“CMT is a disabling condition that starts in childhood,” says Dr. Thomas. “It leads to people retiring earlier because of trouble walking and using their hands. It is our hope that this experimental treatment will allow patients to avoid such complications.”
The Hereditary Neuropathy Center at the Neuroscience Institute at Hackensack University Medical Center, part of the Hackensack Meridian Health network, was recently awarded a research grant from the University of Pennsylvania Orphan Disease Center in partnership with the Hereditary Neuropathy Foundation to analyze data from a unique patient registry, the Global Registry for Inherited Neuropathies (GRIN), to help better diagnose and treat CMT neuropathies and to inform future clinical research focused on the condition.
“GRIN provides a way for us to identify knowledge gaps to support drug developers with data to complement clinical data that will help to ensure well thought-out clinical trial designs and protocols,” said Dr. Thomas.
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