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Families of Spinal Muscular Atrophy; University of California, Irvine Stem Cell Scientist; and California Stem Cell Announce Collaboration for Safety Studies for Stem Cell Therapy in Spinal Muscular Atrophy
Date:2/19/2008

Safety Studies Leading to FDA Application for Stem Cell Therapy in SMA will begin in early 2008

LIBERTYVILLE, Ill., Feb. 19 /PRNewswire-USNewswire/ -- Families of Spinal Muscular Atrophy (FSMA); a stem cell scientist at University of California, Irvine (UCI); and California Stem Cell, Inc. (CSC) are pleased to announce a partnership to advance a potential stem cell therapy for SMA to human clinical trials. The specific set of animal studies planned, which will be conducted in accordance with FDA regulations, will assess the safety of motor neuron progenitors derived from human stem cells after transplantation.

SMA is a genetic disorder with no current treatment that is the leading genetic killer of children younger than two years of age. SMA typically is marked by the degeneration of voluntary muscle movement including the muscles that control crawling, walking, swallowing or breathing due to the dysfunction or death of motor neurons. It is a debilitating and often fatal disease.

These safety studies are the critical steps in advancing stem cell therapy into human trials for SMA. High purity human motor neuron populations for use in transplant therapies were developed by CSC and have been used successfully in proof of concept efficacy and preliminary safety studies in the laboratory of Dr. Hans Keirstead at UCI with funding from FSMA. CSC employs scalable manufacturing protocols to produce and supply the large population of clinical-grade motor neuron progenitors required for these pivotal studies and future human clinical trials.

"This collaboration illustrates the breadth of skills that are required to take a potential treatment from the bench to the bedside. I am confident that we have assembled the right team and the right plan to move this treatment forward with both diligence and speed," said Hans S. Keirstead, Professor, UCI.

"California Stem Cell is very pleased to be part of this collaboration with Familie
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SOURCE Families of Spinal Muscular Atrophy
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