News and Events 
FAMILIES OF SPINAL MUSCULAR ATROPHY FUNDED STEM CELL MOTOR NEURON REPLACEMENT PROGRAM
FSMA has invested $1.5 Million to develop a motor neuron replacement therapy for SMA, resulting in significant progress.
Libertyville, Ill., December 30, 2008
FSMA has invested significant resources in alternative approaches that show promise to cure Spinal Muscular Atrophy rather than just treat the symptoms. In particular, we have invested $1.5 Million to develop a motor neuron replacement therapy for SMA, and we have made significant progress with our investment.
Our initial investment in stem cell research in 2000 funded efficacy studies using motor neurons from mouse stem cells. Results show that this therapy can provide benefit to rodents with motor neuron disease: a highly significant finding. In 2005, additional FSMA funding lead to the first, highly-pure therapeutic population of human motor neurons for cellular replacement therapy for SMA. This program is now progressing on the path to IND in collaboration with the biotech firm California Stem Cell, Inc. (CSC), and leading research centers at University of California-Irvine, and Johns Hopkins University. These motor neurons recently completed a series of critical animal safety studies prior to advancing into human trials for SMA.
Read the full article here.
Motor neuron replacement is at the leading forefront of current scientific knowledge, and as such is very high risk. However, this approach allows for the possibility of replacing lost motor neurons and so holds great promise for the patients and families in our community.
"FSMA provided the first financial support for my research program investigating the development of high purity human motor neuron populations from stem cells, and their application to animal models of SMA. This work has grown into a multi-tiered program that now involves several funding agencies, an industry collaborator, a clinical collaborator and FDA relations. I will always consider FSMA my partner in pioneering this technology, and moving it towards human use." Hans S. Keirstead, Ph.D., Associate Professor of Anatomy and Neurobiology, Co-Director of the Sue and Bill Gross Stem Cell Research Center, University of California at Irvine and Chairman of the CSC Scientific Advisory Board.
Pre-clinical efficacy studies have been completed, demonstrating correct localization of CSC motor neurons in the ventral spinal cord, cell growth from the spinal cord toward the limbs, synapse formation with target muscle and functional reinnervation leading to restoration of limb function in animal models of motor neuron loss.
