California Stem Cell Inc. Files IND to Commence Phase I Clinical Trial in Spinal Muscular Atrophy

IRVINE, Calif.--(BUSINESS WIRE)--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. Approximately 1 in every 6000 babies born is affected. 1 in 40 people, or approximately 7.5 million people in the US, are genetic carriers. SMA Type I, the most severe form of the disease, progresses very rapidly and is often fatal in the affected infants. To date, there are no treatments for this disease.

CSC, a leading stem cell therapeutics company, has developed a stem cell-derived motor neuron transplantation therapy, MotorGraft™, for the treatment of SMA Type I. Pre-clinical studies completed in collaboration with the Hans Keirstead Research Group at the University of California, Irvine have shown functional benefit and safety in animal models. CSC’s MotorGraft™ was granted orphan drug status for treatment of SMA by the FDA in late 2009.

Filing of this application is the first step in a multi-phase clinical development pathway aimed ultimately at approval of a novel therapy. The approval process for cutting-edge therapeutic approaches such as cell products may present unique regulatory challenges compared to conventional drugs, so companies and the FDA must work in close partnership to ensure safety and efficacy of these first in-human products. A cautious regulatory approach has been the norm in cell therapy applications submitted to date in other disease areas.

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.

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 30 Chapters throughout the United States and over 70,000 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.

Web site: http://www.curesma.org

About California Stem Cell, Inc.:

California Stem Cell, Inc. is a privately held company focused on the manufacture 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 cells and liver cells at its Irvine, California facility. CSC is currently developing stem cell-derived therapies for spinal muscular atrophy (SMA), amyotrophic lateral sclerosis (ALS) and spinal cord injury.