FSMA, Invitrogen Corporation, and deCODE Chemistry announced today they have identified a protein that is a potential molecular target for the treatment of Spinal Muscular Atrophy (SMA).  In its most severe form, SMA often leads to death in infancy, and there is currently no treatment or cure.  Research published today in the journal ACS Chemical Biology of the American Chemical Society, entitled “DcpS as a Therapeutic Target for Spinal Muscular Atrophy,” details the identification and characterization of a protein that offers a novel biological mechanism for designing new SMA therapeutics.

Previously, researchers at deCODE, with funding from Families of SMA, had developed a class of compounds called C-5 substituted quinazolines, which increased expression of SMN protein, potentially giving clinical investigators a new class of compounds to utilize for the treatment of SMA.  However, the mechanism behind this increase in SMN production was unknown.

“While the identification of compounds that increase SMN expression represents significant hope to patients with SMA, we still did not understand the mode of action of these compounds in SMA,” noted Jill Jarecki, Ph.D., Research Director at Families of SMA. “The results outlined in the paper represent a new understanding of the physiological mechanisms that can increase SMN expression and will allow us to move forward in advancing potential treatments for SMA.  This discovery gets to the level of really understanding how SMN deficiency can be corrected in the cells of the body, which in turn will open up many new ways of developing therapies.”

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