Special public meeting to spotlight stem cell-based therapies for ALS

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, San Diego grant to pursue novel, fast-tracked stem cell-based therapies for amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease.

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, PhD, professor of cellular and molecular medicine at the UC San Diego School of Medicine, Howard Hughes Medical Institute Investigator and director of the UC San Diego Stem Cell Program and Don W. Cleveland, PhD, 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 (LICR).

Also speaking are Mark Bonyhadi, PhD, director of clinical business development for cell therapy systems at Life Technologies Corporation; co-principle investigator Martin Marsala, MD, professor in the UC San Diego School of Medicine's department of anesthesiology and Lucie Bruijn, PhD, chief scientist of the ALS Association.

ALS is a devastating disease marked by progressive degeneration of motor neurons - the nerve cells that form a complex system from the brain to the spinal cord to the body's muscles and which control voluntary movement. Over time, ALS leads to extreme muscle weakness, paralysis and death. Approximately 30,000 people in the United States are affected. The causes of ALS are unknown; there is no cure. Only one drug - Rilutek - is currently approved by the Food and Drug Administration for treating ALS. Rilutek extends the lifespan of ALS patients by a maximum of three months.

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-principal investigator Sam Pfaff, PhD, 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.

"CIRM issued a challenge to those of us who are interested in applying basic scientific research to the development of new treatments for terrible diseases," said Goldstein. "The challenge was simple: Rethink your assumptions about what can be accomplished in a short period of time, then come back to us with a compelling and persuasive proposal for how you might be able to fast-track a new therapeutic approach into clinical trials to test whether it will work in human patients.

"Our group - and a number of other groups around the state - accepted the challenge and are now in the process of trying to meet these incredibly aggressive goals. None of us is guaranteed to succeed, and some of us will fail, but if we don't try, all of us will fail."

Life Technologies is another partner in the ALS grant. The Carlsbad-based company will provide tools, technology and support in stem cell biology, cell separation, sequencing and bio-production.

"Our scientists will bring together diverse technologies across multiple businesses at Life Technologies to provide a clinically compliant process intended to enhance the safety profile of this innovative therapeutic approach," said Paul Pickering, general manager of the Cell Therapy Systems segment of Primary and Stem Cell Systems at Life Technologies.

"Our hope is that our involvement in the UCSD and Salk study will lead to a reproducible approach that may be applied to other diseases that have huge unmet medical need. We are pleased to be involved in this innovative investigation and hope that our efforts help accelerate the process of bringing novel therapeutics to the general public."

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.

"Proposition 71 is a patient-driven initiative to fund the efforts of the world's best scientists in the search for therapies and cures to reduce human suffering from chronic disease," said Robert Klein, chair of CIRM's governing board.

"These spotlight meetings provide a personal human and scientific understanding of the terrible burdens of disease and hope for the future. The ALS spotlight will provide insights for the board and the public into the tremendous burdens on the patients and their families and the nobility of the spirit of the patients in fighting this terrible disease. The patient view is complemented by the clinician's description of the clinical challenges, and the scientists and clinicians perspectives on the opportunities for advancement in the fight against ALS through stem cell research."

SOURCE University of California, San Diego