Grant for Development of CMT 1A Medication Treatment

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CMTA Funds New Three-Year Grant to Develop Treatment for CMT

One application for a three-year grant of $100,000 per year was

chosen for funding by the scientific review committee of the CMTA's

Medical Advisory Board and a vote of the CMTA's Board of Directors.

The recipient of funding to begin in the fall is Dr. Scherer,

M.D., Ph.D, the N. Kelley Professor of Neurology, University

of Pennsylvania School of Medicine.

Dr. Scherer's grant is entitled, " A high-throughput screen for Kv3.1b

antagonists. " The purpose of his research is to identify new

medications to treat CMT1A and other demyelinating forms of CMT by

looking for drugs that inhibit potassium channels in peripheral

nerves. His summary of the work states that:

We have unpublished evidence that a substantial fraction of

myelinated axons do not function in the nerves of Trembler J mice, a

genetically authentic animal model of CMT1A. Furthermore, the

function of these axons can be restored with a drug (4-aminopyridine;

4-AP) that blocks many kinds of potassium channels. We have evidence

that a single kind of potassium channel, Kv3.1b, is the main target

of 4-AP. Because there are no specific blockers of Kv3.1b, and

general potassium blockers like 4-AP are difficult to use in humans,

our results motivate us to search for drugs that inhibit Kv3.1b

selectively, and test the effects of these drugs on Trembler J

nerves.

This study will be supported by the CMTA for three years, and have

periodic reports on the progress of the study will be presented.