A Method to Slow or Prevent the Progression Peripheral Neuropathies
Gavriel David
Problem
Charcot-Marie-Tooth disease (CMT) is one of the most common inherited neurological disorders, affecting approximately 1 in 2,500 people in the United States. While there are many types and subclasses of CMT, all are characterized by the progressive degeneration of the motor and sensory nerves. This progressive degeneration is gradual, and can be accompanied by mild to severe pain. There are currently no effective therapeutic approaches to treat CMT.
Solution
A therapeutic treatment strategy has been discovered through the identification of a previously unknown pathway involved in the pathogenesis of CMT; in CMT-Type-1A animal models, intraaxonal calcium concentration was observed to be larger in magnitude and more spatially widespread in comparison to controls. Given that excessive levels of intracellular calcium are recognized to be toxic, calcium channel blockers were tested and shown to substantially (over 50%) reduce abnormal intraaxonal calcium influx in the tissues of CMT animal models.
Competitive Advantage
Currently, there is no generally recognized therapeutic strategy for preventing or slowing the progression of CMT and other peripheral neuropathies.
Applications
Treatment of CMT-Type-1A.
Potentially Other Peripheral Neuropathies
Patent Status
United States Provisional Patent Filed August 15, 2008.
Licensing Opportunity
Available for exclusive-license and/or collaborative co-development agreement.
About the Inventors
Gavriel David, M.D., Ph.D., is currently a Research Associate Professor in the Department of Physiology and Biophysics at the University of Miami. Here, he leads a team of scientists studying the function of myelinated axons and motor nerve terminals and the mechanisms by which neurodegenerative diseases disrupt this function.
