A University of Missouri-Kansas City researcher has secured funding for a project which could significantly improve the lives of people diagnosed with the deadly neurodegenerative disease ALS, commonly known as Lou Gehrig’s disease.

Thomas Menees, an associate biology professor, and his collaborator, Aaron Gitler from Stanford University, received the Frontiers Trailblazer Pilot award to further their research, which advances a promising drug therapy for the disease. Currently, there are no treatments that significantly alter the course of this disease, and death typically occurs within two to five years of diagnosis.

Most ALS cases are associated with clumping of the RNA-binding protein TDP-43 inside motor neurons of the brain and spinal cord, which leads to motor neuron death.

 Gilter and Menees were both studying enzymes that suppress TDP-43 aggregation, and thus decided to collaborate to identify these enzymes that could help treat ALS and HIV infection when inhibited. Menees plans to carry the work forward by using the best inhibitor to develop a drug for ALS.

 “We’re in the ‘Valley of Death’ stage right now in the drug development process,” Menees said. “This is where basic research discoveries that could help patients have advanced to a point where targeted, applied research funding is important, but hard to get. Our project will take our best inhibitor and design novel, more effective inhibitors that could slow or stop the progression of ALS.”

 This project is a collaboration among UMKC, Stanford University and the University of Kansas.