Families of SMA Announces New Research Grant

Families of SMA (FSMA) have announced funding for more research dedicated to ultimately finding a treatment and a cure for spinal muscular atrophy (SMA).

The organization has awarded $75,000 to Dr. Chien-Ping Ko, who is a faculty member at the University of Southern California's department of biological sciences, neurobiology section.

In announcing the research funding, FSMA said Ko would be investigating the involvement of two types of glial cells - called astrocytes and microglia, they're cells that support neurons - in the development of SMA.

"The results of the proposed studies would provide a novel concept that, in addition to motor neurons, glial cells may also play a key role in SMA pathogenesis," FSMA said. "The elucidation of new roles of astrocytes and microglia in SMA would in turn lead to new therapeutic approaches by targeting these glial cells."

Ko, who has a Ph.D. in physiology from Washington University in St. Louis, has been studying neuromuscular junctions (NMJ) - connections between the nervous system and muscles - for years and has examined whether they might be involved in motor neuron diseases. His earlier work involved amyotrophic lateral sclerosis (ALS), and after reading about different types of motor neuron diseases, "We were intrigued by the possibility of NMJ involvement in SMA," he said.

FSMA also provided an April 15 update on its funding of a project being conducted by the California Institute of Biomedical Research.

The project is called "Optimization of Small Molecules That Increase SMN2 Levels for the Treatment of Spinal Muscular Atrophy" and is being headed by Dr. Peter G. Schultz.

Initial funding, awarded in 2012, gave $700,000 to Schultz and his team, who'd conducted research on the SMN2 gene. In its literature, FSMA says Chromosome 5 spinal muscular atrophy is caused by "a deficiency of a motor neuron protein called SMN, for 'survival of motor neuron.' That deficiency, the organization explains, occurs due to a mutation on chromosome 5 and can be somewhat alleviated: "Neighboring SMN1 genes can in part compensate for nonfunctional SMN1 genes. Recent evidence suggests that a lack of SMN also may affect muscle cells directly."

Among the research team's initial goals were to confirm that SMA protein levels were elevated, and to make compounds that need less drug to increase SMN levels, FSMA said. With those first two tasks accomplished, FSMA noted, "We anticipate achievement of aim three in the coming months."

The third goal is to make compounds with greater amounts of drug reaching the brain. Eventually, the team seeks to ensure the compounds are safe in preliminary tests, and then to test the compounds in mice.

FSMA, whose annual conference takes place in Washington, D.C., in June, says it's invested more than $19 million in drug development since 2000.

About the Author

Laurie Watanabe is the editor of Mobility Management. She can be reached at lwatanabe@1105media.com.