California Stem Cell, Inc. and Families of Spinal Muscular Atrophy Announce Completion of Pre-IND Meeting with FDA for Stem Cell-Derived Therapy for the Treatment of Spinal Muscular Atrophy Type I

Significant Progress made Towards Moving Novel Therapy into Human Clinical Trials

IRVINE, CA, USA | October 14, 2009 | California Stem Cell, Inc. (CSC) and Families of Spinal Muscular Atrophy (FSMA) announced today 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.

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. There are no approved therapies for the treatment of SMA. Approximately 1 in every 6000 babies born is affected. 1 in 40 people are genetic carriers, indicating approximately 7.5 million carriers in the United States.

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, MotorGraftTM.

MotorGraftTM has the potential to provide benefit for SMA by two mechanisms: 1) direct replacement of the motor neurons lost during the disease course, resulting in new muscle innervation, and 2) 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.”

About Families of Spinal Muscular Atrophy (

Families of Spinal Muscular Atrophy is dedicated to creating a treatment and cure by: Funding and advancing a comprehensive research program; Supporting SMA families through networking, information and services; Improving care for all SMA patients; Educating health professionals and the public about SMA; Enlisting government support for SMA; Embracing all touched by SMA in a caring community. FSMA’s vision is a world where Spinal Muscular Atrophy is treatable and curable.

Families of SMA is a non-profit 501(c)3 tax exempt organization with 26 Chapters throughout the United States and over 65,000 members and supporters. Families of SMA funds and directs the leading SMA research programs. The successful results and progress from basic research to drug discovery programs to clinical trials provide real hope for families and patients.

About California Stem Cell, Inc (

California Stem Cell, Inc is a privately held company focused on the manufacturing of high-purity human cells for therapeutic development and screening applications. Since its founding in 2005, CSC has developed and has intellectual property surrounding methods for scalable production of human motor neurons, neuronal progenitors, cardiac muscle and sino-atrial node cells at its Irvine, California facility. CSC is currently in the pre-clinical development stage of stem cell-derived therapies for spinal muscular atrophy (SMA), ALS and spinal cord injury.

SOURCE: California Stem Cell, Inc.

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