Daniela Zarnescu, associate professor of molecular and cellular biology at the University of Arizona in Tucson, was awarded an MDA research grant totaling $405,000 over a period of three years to investigate whether anti-diabetic drugs may reduce the death of muscle-controlling nerve cells called motor neurons in amyotrophic lateral sclerosis (ALS).
Zarnescu and colleagues have developed a model of ALS in fruit flies that carry a mutant form of the TDP43 gene, a known cause of ALS. “TDP43 has emerged as a common denominator for a significant number of ALS cases,” Zarnescu says, “because mutations in TDP43 can cause ALS and, in addition, normal TDP43 protein associates with cellular inclusions found in autopsy samples from ALS patients, regardless of the genetic cause of the disease.”
She has used this model to test candidate drugs that are already approved for other uses. Her work led to the identification of several types of drugs currently used to treat diabetes that also reduced death among the ALS research flies. The drugs work to affect signaling by the hormone insulin, suggesting that the insulin pathway may be a critical survival pathway in motor neurons.
“Given that our candidate drugs are already approved for use in humans, our work in the fly model will provide a rapid and cost-effective strategy to help determine whether these drugs could be adapted for the treatment of ALS,” Zarnescu says.
Funding for this MDA grant began Feb. 1, 2013.