Li Niu, professor and chair of chemistry at the State University of New York at Albany, was awarded an MDA research grant totaling $405,000 over a period of three years to develop new drugs as potential therapies for amyotrophic lateral sclerosis (ALS).

One mechanism believed to be involved in ALS is overactivity of a protein called the AMPA receptor on the surface of muscle-controlling nerve cells called motor neurons. Too much receptor activity leads to too much excitation of the neuron, and eventually the neuron dies.

“Finding inhibitors to control the excessive receptor activity has been a long-pursued strategy for developing ALS drugs,” says Niu.

Niu is developing AMPA receptor inhibitors made from a cell chemical called RNA. These inhibitors, called RNA aptamers, have been shown to prevent receptor activity, but are vulnerable to cellular enzymes that break them down. Niu plans to chemically modify the aptamers to make them resistant to these enzymes. “That is the first step to translate these powerful AMPA receptor aptamers into clinically useful drugs,” he says.

He has already developed several chemically modified aptamers, and will now test their neuroprotective effectiveness in ALS cellular and animal models. “These studies are key preclinical experiments to advance these RNA inhibitors as a new ALS drug,” he says.

Funding for this MDA grant began Feb. 1, 2013.