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History of the Stem Cell Program

FSMA started investing in stem cell research for SMA more than 5 years ago, and we have made significant progress with our funding.  Cellular therapy is at the leading forefront of current scientific knowledge, and as such is also quite high risk but also holds great promise for the patients and families in our community. 

Please click here to read the current status of the program in a our recent Drug Discovery Compass.

To advance stem cells to human clinical trials, several types of data are required:

• efficacy data showing this approach works for SMA
• quality assurance data showing implementation of standardized clinical grade manufacturing of the cell product
  
• animal safety data assessing potential risk of human exposure to the cellular product
  
• preparation of the IND package for the FDA to request permission to start human trials, which includes presentation of the above data, as well as the proposed design of the clinical trials to be conducted

Our collaboration team for this project includes the laboratory of Dr. Keirstead at The University of California, Irvine (UCI), the team at  California Stem Cell, Inc (CSC), and previously the laboratory of Dr. Kerr at Johns Hopkins University.  These groups have worked on obtaining the three types of data needed for an IND application. Progress for each area is described below.

Please click here to see a synopsis of this project by Dr. Hans Keirstead at the University of California, Irvine.

Funding for efficacy studies helped produce two important findings:

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 and their benefit in Spinal Cord Injury Models.   (See Figure 1, PLoS One, August 2010).

In addition, in early 2007, FSMA funded two additional grants to both the Kerr and Keirstead laboratories.  The Keirstead lab received a two-year grant to analyze the previously generated source of high purity motor neurons to prove they behave like actual motor neurons and are fit for use in human clinical trials.  The Kerr lab also received a 2-year grant to demonstrate that the human motor neurons generated in the Keirstead lab provide therapeutic benefit in a large animal model of motor neuron disease. 

In 2009, FSMA funded a grant to the Keirstead lab to show whether these cells can provide benefit in mouse models of SMA.  This work is ongoing. 

In 2009, FSMA funded a grant to the Keirstead lab to demonstrate whether these cells can provide functional benefit to respiration in a rat model of Spinal Cord Injury.  This work is ongoing.

California Stem Cell, Inc. developed methods for the manufacture of clinical grade human motor neurons progenitors from human embryonic stem cells.  The product is called MotorGraft.

CSC employs scalable manufacturing protocols to produce and supply the large population of clinical-grade motor neuron progenitors required for the pivotal studies and future human clinical trials. 

Families of SMA, Hans Keirsteand, and CSC collaborate to complete the pivotal animal safety study, required to support an IND application to the FDA to begin clinical trials.  This work was completed in October 2009.

These experiments assessed the safety of motor neuron progenitors derived from human stem cells after transplantation.  There  were no negative outcomes of this safety study, leading to the conclusion that motor neuron replacement should be a safe strategy in the treatment  of diseases such as SMA characterized by motor neuron loss, although this also needs to be assessed in early human clinical trials as well.  These safety studies are the critical step in advancing stem cell therapy into human trials for SMA.  Please click here to read a press release about the funding of this work by FSMA.

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.   Upon reaching this stage of the project, FSMA funding enabled the hiring of a Medical Director and a Clinical Research Coordinator at CSC. The two new positions are responsible for coordinating the preparation of the above IND application and for overseeing and monitoring the resulting clinical trials. The financial support for these positions came from a grant to CSC from Families of SMA through the generous support of the Dhont Family Foundation.  

 Please click here to see a recent press release by FSMA and CSC discussing the FDA pre-IND meeting.

 Please click here to see the press release by FSMA, UCI, and CSC announcing our partnership to advance stem cell therapy toward the clinic by hiring the needed medical personnel to file an IND application.

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. 

In December 2010, California Stem Cell Inc. Files IND to Commence Phase I Clinical Trial in Spinal Muscular.  Please click here to read the press release. 

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.