The effort to find new treatments for ALS got a big boost recently when the ALS Association awarded two drug development contracts worth $998,500.
The agreements will fund preclinical development of new therapeutic strategies that target disease-related pathways in the central nervous system, the ALS Association said.
Amyotrophic lateral sclerosis, or ALS, attacks nerve cells in the brain and spinal cord, progressively causing the loss of muscle control and leading to paralysis and death within two to five years of diagnosis, on average.
One of the recipients is OptiKira LLC of Cleveland, which will focus on a way to block the creation of a protein that triggers cell death pathways in ALS patients. Similar work has prolonged survival in animal models that also experience programmed cell death. The ALS Association is giving OptiKira $498,500 over two years.
The other recipient, Thomas Lloyd of Johns Hopkins University, will receive $500,000 over two years to test nuclear export inhibitors for treating a mutation that is the most common genetic cause of ALS. Similar treatments have demonstrated promising results in animal models of traumatic brain injury.
“We are excited to be able to fund these important projects in new therapeutic strategies,” Lucie Bruijn, chief scientist at the ALS Association, said. “Through our Lawrence and Isabel Drug Development Program, we are able to bring the most promising new ideas in therapy development quickly through the preclinical phase, to more rapidly advance the best ones into clinical trials. That is the key to getting new treatments for ALS, which we so desperately need.”
