Families of Spinal Muscular Atrophy (FSMA) has given a one-year award of $150,000 to a research program at Harvard University’s Department of Stem Cell & Regenerative Biology.
The research, led by Dr. Lee Rubin, professor of stem cell and regenerative biology at Harvard and director of translational medicine at the Harvard Stem Cell Institute, seeks “to identify novel SMA therapeutics by using patient-derived motor neurons for drug discovery and preclinical testing,” FSMA said in a news announcement.
“The researchers have made induced pluripotent stem cells from type I and type II SMA patient skin biopsies, and produced large numbers of motor neurons that can be used to model SMA,” the announcement said. “They will use these motor neurons in a screen for drugs that increase SMN (protein) levels.”
FSMA added that researchers hope to identify the compounds that can increase functional SMN protein levels in the type of cell that’s most affected by spinal muscular atrophy. That discovery could lead to future discoveries of drugs that could be used as therapies for SMA, a motor neuron disease that leads to muscle wasting and weakness.
