ALSA Renews Funding for Two Key Strategies Toward ALS Therapy

Antisense, Stem Cell Projects Show Promise

The ALS Association (ALSA) today announced renewed funding for two key projects that intend to produce new therapies for ALS. ALSA-initiated grants were renewed to Don Cleveland, Ph.D., and colleagues working on antisense approaches to treating the disease, and to Clive Svendsen, Ph.D., and collaborators at the University of Wisconsin in Madison, who are designing stem cell strategies.

In ALS, stem cells could rescue dying nerve cells by producing, at the site of damage, the supportive proteins called trophic factors. Svendsen and colleagues at the Waisman Center have marked progress toward that goal, by implanting stem cells in a rodent spinal cord. Further experiments will test the ability of several trophic factors to improve survival in ALS rodents. Also, Svendsen will bring on board an expert in respiratory physiology, Gordon Mitchell, Ph.D., to see if stem cell therapy with trophic factors can slow or reverse the respiratory decline that is the final stage of ALS.

Antisense is another way to address the process that kills motor neurons in ALS. Cleveland ’s team has designed small molecules that stop the genetic message for production of mutant protein in inherited ALS. Renewed ALSA funding will support safety tests and studies of how long the molecules stay in the rat’s body, to pick the best candidate for clinical trials.

Brian Kaspar, Ph.D., now at the Columbus Children's Research Institute at The Ohio State University, will collaborate with the Cleveland lab to help with a method called RNA interference (RNAi), which is another way to silence the RNA message that produces mutant protein. Researcher report that mutant SOD1 mice treated with RNAi show prolonged grip strength.