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FSMA-Funded Stem Cell Research

In March 2011 authors, Eva Feldman, Nicholas Boulis, and colleagues published a review article In Regenerative Medicine explaining the potential of Stem Cell Therapies for ALS and SMA. Click here to see this article. 

In December 2010, FSMA was pleased to see the first novel therapy for SMA initiating the official FDA process to begin human clinical trials with the filing of an IND application to the FDA for the California Stem Cell MotorGraft Program. This IND filing was a milestone moment for our community.  The first proposed trials were for safety trials to be conducted in a very small numbers of patients and were not primarily intended to test the efficacy of the drug product.  

The filing of an IND application is just the first step in a long and complicated process aimed ultimately at approval of a new therapy.  A likely outcome from this application was that the trial would be placed on clinical hold by the FDA.  A clinical hold is an order that the FDA issues to a sponsor to delay a proposed trial.   Each of the previous applications that have been submitted in this very new field of investigational stem cell treatments has been placed on clinical hold for approximately a year after the IND was filed.  FDA caution is not unusual for new therapeutic approaches.  Please click here for more on the clinical trial process. 

In January, the FDA placed the MotorGraft Program on clinical hold, in order to obtain additional data.  Please click here for that announcement.  

Program Milestones Over the Past Five Years, Enabled with FSMA Funding (please see funding tab above for specific details):

1. Proof-of-concept experiments showing motor neurons from mouse embryonic stem cells provided therapeutic benefit in a rodent model of motor neuron disease from the Kerr lab (Annals of Neurology, July 2006, 60: 32-44).

2. The successful production of motor neurons made from human embryonic stem for therapeutic use in humans from the Keirstead lab (See Figure 1, PLoS One, August 2010).

3. CSC develops methods for the manufacture of clinical grade human motor neurons from human embryonic stem cells.  This product is called MotorGraft.

4. In 2009, FSMA and SMA Europe co-funded a project reviewed by both our respective scientific advisory boards to the Comi Lab at the University of Milan called "Development of Therapeutic Strategies Based on Stem Cell Transplantation for SMA". Using two different sources of neural cell stem cells, this group has published two studies in peer-reviewed journals showing that a nursing effect of these cells when transplanted into the spinal cord of severe SMA mice (Delta7) prolongs survival and motor function. Please click here to see their papers in Brain and the Journal of Clinical Investigation. 

5. In a collaboration between Families of SMA, Hans Keirstead, and California Stem Cell, Inc. (CSC), the pivotal animal safety study, required to support an IND application to the FDA to begin clinical trials, was completed in October 2009.

6. In October 2009, CSC holds a pre-IND meeting with the FDA to discuss formal Investigational New Drug (IND) application to begin a Phase I/IIA clinical trial in SMA Type I. Please click here to see a recent press release by FSMA and CSC discussing the FDA pre-IND meeting.

7. In December, 2009 California Stem Cell, Inc. receives Orphan Designation for the MotorGraft Product from the FDA.  Please click here to read the press release. 

8. In December 2010, California Stem Cell Inc. Files IND to Commence Phase I Clinical Trial in Spinal Muscular, which was subsequently placed on clinical hold.  Please click here to read the press release. 

More detailed Information can be found in the history of program tab 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.