The CMTA’s research is done in teams consisting of academic labs and clinical centers sponsored by the CMTA (STAR members), working together and with alliance partners in consortium team efforts. STAR Teams are sponsored by the CMTA via a rapid process of expert review, following invitation of proposals for targeted translational efforts. All sponsorship aims to directly aid the advancement of CMT therapies.
Cell based reporter assay established for screening of potential therapeutic libraries
One potential therapeutic target class identified
Multiple therapy development alliances established
Animal models licensed and in use for evaluation of candidate therapies
Expansion of assay capability to include high throughput assays, Nerve-Schwann cell co-cultures, and human patient-derived stem cells
The CMTA launched an alliance effort with the New York Stem Cell Foundation (NYSCF) in 2014 to create iPSC lines from a number of different CMT disease states. Human stem cell lines will be systematically created by the NYSCF from human patient skin cell lines banked at the University of Iowa. The work is expected to be completed in late summer of 2015 on the first four CMT diseases. NYSCF will bank the cell lines and make them available for research use. Their implementation in drug candidate evaluation is expected to be swiftly incorporated into the CMTA’s Disease Project Team efforts, where alliance partners will also have access to their use for STAR consortium work.