Local scientists, health advocates and leaders from the California Institute for Regenerative Medicine (CIRM) will gather in San Diego June 23 for a special public meeting to spotlight a new University of California (UCSD), San Diego grant to pursue novel, fast-tracked stem cell-based therapies for amyotrophic lateral sclerosis (ALS).
The San Diego meeting is part of a larger, long-term effort to inform and educate the public about research and projects funded by CIRM, which was established in 2004 following passage of Proposition 71, the California Stem Cell Research and Cures Initiative. That statewide ballot measure provided $3 billion in funding for stem cell research at California universities and research institutions.
The meeting, to be held at 8:30 a.m. at the Sheraton San Diego Hotel on Harbor Island Drive, will feature presentations by the grant project’s principal investigator Larry Goldstein, Ph.D., professor of cellular and molecular medicine at the UCSD School of Medicine, Howard Hughes Medical Institute Investigator and director of the UCSD Stem Cell Program and Don W. Cleveland, Ph.D., professor and chair of cellular and molecular medicine at the UC San Diego School of Medicine and head of the Laboratory of Cell Biology at the Ludwig Institute for Cancer Research.
Also speaking are Mark Bonyhadi, Ph.D., director of clinical business development for cell therapy systems at Life Technologies Corporation; co-principle investigator Martin Marsala, MD, professor in the UCSD School of Medicine’s department of anesthesiology and Lucie Bruijn, Ph.D., ALS Association chief scientist.
“The ALS Association is currently funding several stem cell studies, including the development of the surgical techniques utilized in the phase 1 Neuralstem study,” said Dr. Bruijn. “We are excited about the potential of stem cells in ALS and recognize the need for multiple rigorous studies in this area.
The $11.5 million CIRM “disease team” grant will fund stem cell research for ALS that exploits a special type of cell called an astrocyte progenitor. Astrocytes are glial cells, a family of cells that support the proper functioning and insulation of neurons. The particular job of astrocytes is to help with neurotransmissions and neuronal metabolism. In ALS, the decay of astrocytes and other cells eventually causes neurons to malfunction and die, leading to a host of debilitating and ultimately fatal consequences.
Previous research indicates that transplanting healthy glial cells into patients could be a possible treatment for ALS, and animal studies have shown that astrocytes possess particular promise. Researchers at UCSD, along with co-principle investigator Sam Pfaff, Ph.D., a professor in the Salk Institute’s Gene Expression Laboratory, hope to develop an ALS therapy that uses human embryonic stem cells to create astrocyte precursors that would be transplanted into patients where they would mature into new and healthy astrocytes that could halt – and perhaps reverse – the progressive ravages of ALS.
The researchers will study two methods of administering progenitors in animal models and test the safety and efficacy of these approaches, with the goal of providing proof-of-principle and laying the groundwork for clinical trials within five years. The grant is among the first by CIRM explicitly expected to result in FDA approval for clinical trials.