Source: California Stem Cell, Inc. and Families of Spinal Muscular Atrophy
Date: December 1, 2010
Summary:
IRVINE, Calif. - California Stem Cell, Inc. (CSC) and Families of Spinal Muscular Atrophy (FSMA) announced today that CSC has filed an investigational new drug (IND) application with the US Food and Drug Administration (FDA) for approval to commence a Phase I safety study on a jointly-developed stem cell-derived motor neuron transplantation therapy for Spinal Muscular Atrophy (SMA) Type I.
SMA is the leading genetic cause of death of infants. It is a disorder that results from a chronic deficiency in the production of the SMN protein, which is essential to the proper functioning of the motor neurons in the spinal cord. SMA is typically marked by the deterioration of the muscles that control crawling, walking, swallowing and breathing.
The trial will study the safety of MotorGraft™ and the surgical procedure required to deliver these cells directly into the spinal cords of patients with SMA Type I and will enroll a very limited number of patients. This IND filing is a major milestone in the search for a treatment for SMA patients.
