UC Irvine News Brief: Clinical trial of UCI stem cell-based therapy for spinal muscular atrophy proposed
California Stem Cell Inc. has applied to the federal Food & Drug Administration to start a Phase I safety study on the treatment, created in collaboration with Hans Keirstead’s research group.
Irvine stem cell therapeutics company California Stem Cell Inc. has applied to the federal Food & Drug Administration to start a Phase I safety study on a stem cell-derived motor neuron transplantation treatment for spinal muscular atrophy. The therapy was created in collaboration with Hans Keirstead’s research group at UC Irvine’s Sue and Bill Gross Stem Cell Research Center and with Families of Spinal Muscular Atrophy. The leading genetic cause of death in infants, SMA impairs proper functioning of motor neurons in the spinal cord and is marked by the deterioration of muscles that control crawling, walking, swallowing and breathing. To date, there are no treatments. Preclinical studies led by Keirstead – chair of California Stem Cell’s scientific advisory board, which includes other UCI stem cell researchers – have demonstrated the safe repair of these damaged neurons.
