Significant progress towards moving novel therapy into human clinical trials

California Stem Cell, Inc. (CSC) and Families of Spinal Muscular Atrophy (FSMA) announced that they have completed a formal pre-Investigational New Drug (Pre-IND) meeting with the Food and Drug Administration (FDA) for guidance on the clinical and regulatory pathway and requirements for submission of an IND to initiate human trials for a stem cell-derived motor neuron replacement therapy for Spinal Muscular Atrophy (SMA) Type I.
CSC, a leading stem cell therapeutics company, has developed a stem cell-derived motor neuron replacement product, for the treatment of SMA Type I. Pre-clinical studies, completed in collaboration with professor Hans Keirstead of the University of California, Irvine, have shown clinical proof of concept through the demonstration of functional benefit in animal models treated with CSC’s motor neuron replacement product, MotorGraft.

MotorGraft has the potential to provide benefit for SMA by two mechanisms: the direct replacement of the motor neurons lost during the disease course, resulting in new muscle innervation, and providing a nursing support function to remaining motor neurons.
“We are greatly encouraged by the feedback we received from the FDA” said CSC COO Chris Airriess, “It will greatly assist us in preparation of our final IND application. Completion of this key milestone is an important step towards moving our SMA program to the clinic”.
“We are very pleased with the outcome of the Pre-IND interaction with the FDA” said Kenneth Hobby, President FSMA. “FSMA has invested almost $2 Million over the last 8 years to develop this motor neuron replacement therapy for SMA. This meeting was a significant accomplishment towards the filing of the IND and ultimately bringing this therapy to patients”.

(source: FSMA website)