MDA awarded a research grant totaling $231,300 over three years to Alex Parker, assistant professor in the department of pathology and cellular biology at the University of Montreal Hospital Research Center in Montreal, Quebec (Canada). The funds will help support Parker's study of cellular stress response in neurodegeneration associated with amyotrophic lateral sclerosis (ALS).
Mutations in the genes for TDP43 and FUS proteins have been linked to ALS, Parker notes, "and they cause persistent cellular stress that ultimately overwhelms the neuron’s [nerve cell's] coping mechanisms, leading to cell death."
With colleagues, Parker is working to better understand a critical cell survival mechanism known as the endoplasmic reticulum stress response, which is known to reduce the toxicity caused by mutant TDP43 and FUS proteins. (The endoplasmic reticulum is a cellular compartment involved in the transport of proteins and other biological substances within cells.)
Using a C. elegans (nematode, or roundworm) model of ALS, Parker aims to identify small molecules that modify the ER stress response to help neurons overcome protein toxicity and stave off cell death.
Parker's work potentially could led to the identification of therapeutic targets and development of therapies for people with ALS.
"There are very few drugs available to treat neurodegeneration, and they are not very effective," Parker says. "We hope our findings accelerate drug discovery and development for ALS patients."
Funding for this MDA grant began Aug. 1, 2012.