FSMA-Funded Stem Cell Research

FSMA is investing in stem cell research for SMA, and we have made significant progress with our funding.  Cellular transplant therapy is the one possible approach that could result in replacement of motor neurons lost during disease course.  Motor neuron replacement is at the leading forefront of current scientific knowledge, and as such is also quite high risk.   However, this approach allowing for the possibility of replacing lost motor neurons holds great promise for the patients and families in our community. 

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

To advance stem cells to human clinical trials, both efficacy data showing this approach works in SMA animal model and safety data assessing risk will be required by the FDA.  Our collaboration team is currently working to get both types of data.  The team includes the laboratory of Dr. Keirstead at The University of California,  Irvine (UCI) , the laboratory of Dr. Kerr at Johns Hopkins, and California Stem Cell, Inc (CSC).


Our past investment in stem cell research, which was devoted entirely to efficacy studies, helped produce two important findings: 


1. Published proof-of-concept experiments showing that motor neurons made 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).

In early 2007, FSMA continued our commitment to this program by funding 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.

Most recently FSMA, Dr. Keirstead, and CSC announced a partnership to advance a stem cell therapy for SMA to human clinical trials. This partnership includes a specific set of animal safety studies, which will be conducted in accordance with FDA regulations.  The experiments will assess the safety of motor neuron progenitors derived from human stem cells after transplantation.


These safety studies are the critical step in advancing stem cell therapy into human trials for SMA. 

High purity human motor neuron populations for use in transplant therapies were developed by CSC and have been used successfully in proof-of-concept efficacy and preliminary safety studies in the laboratory of Dr. Hans Keirstead at UCI with funding from FSMA.  CSC employs scalable manufacturing protocols to produce and supply the large population of clinical-grade motor neuron progenitors required for these pivotal studies and future human clinical trials.  Therefore, we currently have the team in place to make progress in obtaining both the efficacy and safety data required to move towards human testing.

See the press release by FSMA, UCI, and CSC announcing our partnership to advance stem cell therapy in our news section.

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.