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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

s of SMA and UCI and optimistic, based on preliminary data, about the very real potential for success in developing a cell transplant therapy for SMA, which devastates so many families." said Chris Airriess, Chief Operating Officer, CSC.

Studies are being conducted in parallel at both the Keirstead laboratory at UCI and the laboratory of Dr. Douglas Kerr at Johns Hopkins University, with funding from FSMA, to show the benefit of human motor neuron progenitor replacement in animal models of motor neuron disease. This builds on the pioneering work published by Dr. Kerr in the Annals of Neurology in 2005 showing that transplants of mouse embryonic stem cell-derived motor neurons into the spinal cord can connect with muscles and partially restore function in paralyzed rats.

"This program holds a tremendous amount of promise for our patients and families. While FSMA makes significant investments in traditional and relatively low-risk drug approaches to find a treatment for SMA, alternative therapies like stem cells may hold the key to a cure. Our strategy is to fund both approaches in parallel to reach our goals as quickly as possible while minimizing risks" said Kenneth Hobby, Executive Director, FSMA. "Families of SMA is extremely grateful to The Dhont Family Foundation for the support of $200,000 given to help move this stem cell program forward."

Stem cell therapy for SMA has the potential to replace the motor neurons lost during the disease course. While other types of therapies have the potential to slow disease progression and possibly increase strength, motor neuron replacement through the use of stem cells is the only means to replace motor neurons once they are gone.

This strategy may be useful for treating multiple disorders such as spinal cord injury, transverse myelitis, and Amyotrophic Lateral Sclerosis (ALS) in addition to Spinal Muscular Atrophy.

About Families of Spinal Muscular Atrophy:

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SOURCE Families of Spinal Muscular Atrophy
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