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CIRM Awards Samuel L. Pfaff $15.6 Million to Develop Stem Cell-Based ALS Therapy

Samuel Pfaff

Samuel Pfaff

Salk researcher Samuel L. Pfaff has been awarded a $15.6 million grant by the California Institute for Regenerative Medicine (CIRM) for translational research focusing on developing a novel stem cell-based therapy for Amyotrophic Lateral Sclerosis (ALS) – or Lou Gehrig's Disease.

Pfaff, a professor in the Salk's Gene Expression Laboratory and an investigator for the Howard Hughes Medical Institute, will lead the group of researchers who will work on the four-year project, which marks the first CIRM funding explicitly expected to result in FDA approval for clinical trials.

The research team will focus on astrocytes, the star-shaped support cells that provide nutrients for nearby motor neurons. Working with six different lines of human embryonic stem cells (hESC), the team will grow clinical-grade astrocyte precursor cells and identify the line that is best suited for implantation in laboratory models.

They hypothesize that the transplanted human astrocyte precursors (hAP) will mature into astrocytes in vivo and provide support for diseased spinal motor neurons. Astrocytes are also capable of clearing excess neurotoxic glutamate and could thereby slow or halt the progression of ALS by preventing motor neuron degeneration.

Once the astrocyte precursors are tested for efficacy and safety to minimize the possibility of tumorigenesis, the next step will be to move forward with human clinical trials after approval by the FDA, says Pfaff.

"This team grant is a natural fit for San Diego because it capitalizes on the strength of neuromuscular disease research from the local scientific community," says Pfaff. "The standard has been set very high on this project because we are aiming to grow a safe population of astrocytes that can be introduced into patients. Our success will be measured by whether it will help extend the lives of patients suffering from ALS."