Researchers doing early-stage investigation into potential new ways to halt neuron death seen in amyotrophic lateral sclerosis (ALS) have received a grant from the Alabama Power Foundation.
Rita Cowell, PhD, a researcher at Southern Research’s Drug Discovery division, and her team have identified certain compounds that in lab tests prevented neuron death.
The funding will allow the researchers to continue to investigate the compounds’ potential to halt the loss of neurons, a hallmark of ALS.
Researchers think the loss of motor neurons in ALS is linked with the accumulation of stress molecules known as reactive oxygen species (ROS).
“Certain cells, like the motor neurons that are lost in ALS, have been shown to be sensitive to this stress response over time,” Cowell said in a press release. “The compounds we have identified could boost the ability of these neurons to counteract the stress response and protect them from cell death.”
She added, “We don’t have additional resources to devote to this work, so this grant allows us to expand our studies, which have a lot of potential.”
Currently the U.S. Food and Drug Administration (FDA) has approved only three treatments to slow the progression of ALS. These are Rilutek (riluzole tablet), Tiglutik (riluzole suspension), and Radicava (edaravone).
Rilutek acts on glutamate release in order to keep this neurotransmitter from reaching toxic levels that damage neurons. Radicava is a potent antioxidant that helps control the imbalance of oxidative stress molecules, namely ROS (reactive oxygen species). Tiglutik has a proposed mechanism of action similar to Rilutek. However, these therapies are not effective for all patients.
“There is a desperate need for new drugs for ALS,” Cowell said.
Myla Calhoun, president of the Alabama Power Foundation, said the grant will contribute to a deeper understanding of ALS disease mechanics and help fuel the discovery of new drugs.
“Health and human services is one of the five focus areas of the foundation and we hope this grant will help position our state as a leader in medical research and development,” Calhoun said. “Southern Research is uniquely positioned to make progress in this area, and our grant will allow its Drug Discovery team to continue moving therapies closer to patients in need.”
Cowell’s team will test several compounds in cells to evaluate how these substances can act against ALS progression.
The team hopes these results will fuel public and commercial investments to test the compounds’ potential in mouse models of ALS and, hopefully, in Phase 1 clinical trials.
“This drug discovery research is in its very early stages, so we don’t expect to have a lead compound in a clinical trial after one year of funding,” Cowell said. “But the grant from the Alabama Power Foundation will help us move toward our long-term goals of discovering new approaches and therapeutics to treat ALS.”