Towards potential stem cell therapy

Families of Spinal Muscular Atrophy (FSMA), University of California, Irvine (UCI) Stem Cell Scientist; and California Stem Cell, Inc. (CSC) announce an extension to their collaboration to advance a potential stem cell therapy for SMA to human clinical trials.
The next stages of the collaboration will include the hiring of a Medical Director and a Clinical Research Coordinator at California Stem Cell, Inc. The two new positions will be responsible for coordinating the preparation of Investigational New Drug (IND) application and for overseeing and monitoring the resulting clinical trials. Together, the two individuals will spearhead and coordinate all aspects of the application for approval to begin, and preparations for, early phase clinical trials in SMA Type I.

The pivotal animal safety studies, required to support submission of an IND Application to the FDA, have recently been completed utilizing clinical grade human motor neurons manufactured by CSC. It is the intent of CSC to gain approval to begin human clinical trials for the use of these cells in development of a cell replacement therapy for SMA Type I. CSC is preparing for a final FDA pre-IND meeting to take place in the first half of 2009, keeping on track for a formal application by the end of 2009 for a Phase I/IIA clinical trial in SMA Type I.

“We are extremely pleased to continue our collaboration with Families of SMA on the development of a stem cell based treatment for SMA” said Chris Airriess, Chief Operating Officer of California Stem Cell; “this additional support will allow us to reach our ultimate goal of helping families afflicted by this devastating disease more quickly”.
Stem cell therapy for SMA has the potential to replace the motor neurons lost during the disease course. While other types of therapies have the potential to slow disease progression and possibly increase strength, motor neuron replacement through the use of stem cells is the only means to replace motor neurons once they are gone. This strategy may also be useful for treating multiple disorders such as spinal cord injury, transverse myelitis, and Amyotrophic Lateral Sclerosis (ALS) in addition to Spinal Muscular Atrophy.

FSMA has invested significant resources in alternative approaches that show promise to cure Spinal Muscular Atrophy rather than just treat the symptoms. In particular, FSMA has invested over $1.5 Million to develop a motor neuron replacement therapy for SMA.

(source: FSMA website)