Spinal Muscular Atrophy Stem Cell Research

FSMA-Funded Stem Cell Research

Click here for a summary article in Regenerative Medicine on the potential of Stem Cell Therapy for ALS & SMA.

Current Project Status: -In 2010, California Stem Cell, Inc submitted an IND application to the FDA for MotorGraft to begin clinical trials in infants with SMA. Please click here for the announcement. -In 2011, the FDA placed the MotorGraft Program on clinical hold, in order to obtain additional data. Please click here for the announcement.

The filing of an IND application is the first step in a long, complicated process aimed ultimately at approval of a new therapy. A clinical hold is an order issued by the FDA to a sponsor to delay a proposed trial to obtain more data. California Stem Cell is actively working on collecting the new data requested by the FDA. Please click here for more on the clinical trial process.

Past Program Milestones: -The Kerr lab publishes studies showing motor neurons from mouse embryonic stem cells provided therapeutic benefit in a rodents. Please click here for the paper. -Production of motor neurons from human embryonic stem cells are published (See Figure 1). The product is called MotorGraft. Please click here for the paper.

Figure 1. Human motor neurons produced at greater than 95% purity.

-FSMA / SMA Europe funded Dr. Comi on "Development of SMA Therapeutic Strategies Based on Stem Cell Transplantation". Please click here to see published results in Brain. Please click here to see published results in the Journal of Clinical Investigation. -Collaboration between FSMA and CSC funds pivotal safety studies to support an IND to the FDA. Please click here for the announcement. -In October 2009, CSC has a pre-IND meeting with the FDA on filing an IND application for clinical trials in infants with SMA. Please click here for the announcement. -In December 2009, CSC receives Orphan Designation for the MotorGraft Product from the FDA. Please click here for the announcement. -In December 2010, California Stem Cell Inc. files IND for clinical trial in infants with SMA. This was placed on clinical hold. Please click here for the announcement.

More Information on the history of program can be found in the link above.

Families of SMA understands that the use of stem cells is a controversial subject. As an organization, FSMA is committed to pursuing the most promising treatment possibilities. However, we also understand that ethical and religious issues remove stem cells as an option for some of our families and donors. For that reason, any donor to FSMA may restrict their donation to non-stem-cell-related research. To do this simply indicate - not for stem cell research - when you make your donation.

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Spinal Muscular Atrophy Stem Cell Research