Congratulations to SMA Foundation Investigator, Lee Rubin, Director of Translational Medicine at the Harvard Stem Cell Institute, and his team for publishing their article, “A Screen for Regulators of Survival of Motor Neuron Protein Levels” in Nature Chemical Biology on June 19, 2011. The article describes a novel image-based screen to identify compounds that increase SMN protein levels in subcellular compartments of intact cells. As a result of this screen, Dr. Rubin’s group discovered several classes of compounds that elevate SMN protein levels in SMA patient fibroblasts and motor neurons deficient for SMN. In particular, chemical inhibitors of glycogen synthase kinase 3 (GSK-3) were found to elevate SMN levels by stabilizing the protein. In addition to SMN upregulation, the GSK-3 inhibitors also increased survival of the motor neurons, suggesting that the compounds correct the main morphological change underlying SMA. These results document the discovery of novel molecular targets and pathways that play a role in controlling SMN protein levels. Dr. Rubin’s research represents an important achievement as it opens opportunities to discover novel therapeutic approaches for the disease. GSK-3 inhibitors have been previously pursued by pharmaceutical and biotech companies for other neurodegenerative disorders. To view the article, please visit http://www.ncbi.nlm.nih.gov/pubmed/21685895.
Dr. Rubin’s research is supported by the SMA Foundation.