Families of Spinal Muscular Atrophy and California Stem Cell, Inc. Motor Graft TM Project 2010 Update

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Families of SMA in collaboration with California Stem Cell, Inc. and the University of California, Irvine, make significant progress in Motor Neuron Program for Spinal Muscular Atrophy.

Families of SMA, in collaboration with California Stem Cell, Inc. and the University of California, Irvine, have made significant progress in the Motor Neuron Program for Spinal Muscular Atrophy.

Families of Spinal Muscular Atrophy (FSMA) has funded $2 Million to date for development of a Cellular Therapy for SMA. In collaboration with California Stem Cell, Inc. (CSC), we have completed an important, 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.

Importance of this Program
SMA is a disorder that results from a chronic deficiency in a protein that is essential to the proper functioning of the motor neurons in the spinal cord. CSC, a leading stem cell therapeutics company, has developed a stem cell-derived motor neuron product, for the treatment of SMA Type I.

Pre-clinical studies, completed in collaboration with Dr. Hans Keirstead, Ph.D. 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 product, MotorGraftTM.

Dr. Keirstead stated, “In 2006, two spinal cord scientists shared a stage at a meeting organized by Families of Spinal Muscular Atrophy. I spoke of what would become the world’s first FDA approved human embryonic stem cell treatment, for spinal cord injury. Dr. Douglas Kerr spoke of his groundbreaking research using motor neurons. Inspired by each other’s work, we left the stage and eagerly sketched out an aggressive strategy to develop a treatment for SMA.”

Progress Made
Families of SMA has been involved in the MotorgraftTM Program at UCI and CSC since the early days of the program. FSMA has funded projects within every stage of the program. The program plan was to generate a population of human motor neurons in a manner that allowed for enormous quantities of cells to be generated, in a purity that permitted human use. This vision was shared, and then enabled, by Families of Spinal Muscular Atrophy.

This population of cells would be tested in animal models to ensure that they work, and in other animal models to ensure that they were safe. A clinical team would then be assembled, a clinical plan would be agreed upon, and the FDA would be presented with the world’s first strategy to treat spinal muscular atrophy using stem cells.

Families of Spinal Muscular Atrophy has dedicated monetary resources to the program, which has successfully:

  •     Completed FDA required safety studies - on tumorigenicity, toxicity, biodistribution, and pain
  •     Completed manufacturing protocols for clinical-grade cells
  •     Completed a pre-IND meeting in October 2009
  •     Obtained Orphan Drug Designation from FDA in December 2009

According to Dr. Keirstead, “In the summer of 2010, the world’s first strategy to treat SMA using stem cells is planned to be presented to the FDA. This rate of progress is extraordinary, and is a testimony to the determination of FSMA in their efforts to conquer this insidious disease.

Families of Spinal Muscular Atrophy literally and figuratively set the stage for this vision. It provided the meeting places for the exchange of ideas. It provided intellectual guidance during the grant review process. It provided funding to Dr. Keirstead’s laboratory at the University of California at Irvine, and to Dr. Kerr’s laboratory at Johns Hopkins University, and to the cell manufacturer California Stem Cell, to realize this vision.”

Funding:
FSMA has invested $2 Million over the last 8 years to develop this motor neuron therapy for SMA, awarding grants through our Scientific Advisory Board, and with significant generous support from the Dhont Family Foundation.
Our objective is to dedicate targeted funds towards programs such as this, which show great promise toward creating a treatment and cure for SMA. We are confident in the process of advancing programs and continue to build a broad base of treatment discovery programs.

Jill Jarecki, Research Director for FSMA, states, "The FSMA strategy for therapy development has been to help build and a robust and diverse drug pipeline for SMA. We believe it is critical to move diverse types of approaches towards the clinic in order to find a effective treatment for SMA in the quickest and most efficient way possible. The MotorGraftTM Cellular Therapy Program is a key component of this strategy, complimenting our other approaches aimed towards enhancing SMN protein levels, which are chronically depleted in SMA."

About Families of Spinal Muscular Atrophy:
FSMA 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 a; Embracing all touched by SMA in a caring community. http://www.curesma.org

About California Stem Cell, Inc:
Irvine, CA based California Stem Cell, Inc is a privately held company focused on the manufacturing of high purity human cells for therapeutic development and clinical application. Founded in 2005, CSC has developed proprietary methods for scalable production of human motor neurons, neuronal progenitors, cardiac muscle and sino-atrial node cells.

About the University of California, Irvine:
The University of California, Irvine is a top-ranked university dedicated to research, scholarship and community service. Founded in 1965, UCI is among the fastest-growing University of California campuses, with more than 27,000 undergraduate and graduate students, and nearly 2,000 faculty members. The third-largest employer in dynamic Orange County, UCI contributes an annual economic impact of $3.6 billion.

Contact Information:
Families of SMA
925 Busse Road
Elk Grove Village, IL 60007
Phone (800) 886-1762
Fax (847) 367-7623
http://www.curesma.org

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Laura Franzen

Kenneth Hobby