Research teams at UC San Diego and UC Davis will share a $6.3 million state grant to study a human embryonic stem cell-based therapy to rescue and restore neurons devastated by amyotrophic lateral sclerosis, also known as Lou Gehrig’s Disease.
The funding from the Independent Citizens Oversight Committee of the California Institute for Regenerative Medicine could lead to human trials, according to UCSD Health.
ALS involves the progressive degeneration of spinal cord motor neurons, which control muscle movement, leading to paralysis and respiratory failure.
“A lot of people and institutions are working on ALS to understand it and resolve it at a cellular level, often targeting the motor neurons themselves. That’s good,” said Lawrence Goldstein, director of both the UC San Diego Stem Cell Program and Sanford Stem Cell Clinical Center.
“Our view is that it’s critically important that we develop an aggressive set of cell therapy programs so that we have multiple — shots on goal — in parallel,” said Goldstein, who’s also a professor in the departments of Neuroscience, and Cellular and Molecular Medicine. “We need to attack the disease from as many angles as possible.”
The research will focus on brain and spinal cord cells that support neurons by, among other things, providing nutrients or serving as impulse transmission regulators.
According to Goldstein, ALS progresses as those cells — astrocytes — die off.
Cellular and animal studies have shown that introducing and augmenting non-diseased astrocytes, and maybe other cell types, nearby dying motor neurons significantly enhances the latter’s survival.
Eventual human trials would involve transplanting neuronal stem cells, derived from an established and well-understood human embryonic stem cell line, into ALS patients. The scientists hope the neuronal stem cells would develop as astrocytes and provide new support for ailing motor neurons, thus extending their lives and function.
“Maintaining motor neuron survival would add years of fulfilling life to ALS patients,” Goldstein said.
The research will be conducted at the Alpha Stem Cell Clinic at UC San Diego Health.
–City News Service
