NEW YORK (GenomeWeb News) – Life Technologies, the University of California, San Diego, and the Salk Institute for Biological Studies today said they are initiating studies that could lead to the development of cell transplant therapy for amyotrophic lateral sclerosis.
The effort is being led by UCSD and the Salk Institute with $11.5 million in funding from the California Institute for Regenerative Medicine. Life Tech is providing expertise in stem cell biology, cell separation, next-generation sequencing, and bioproduction through its Invitrogen Primary and Stem Cell Systems business, the company said in a statement.
Researchers at the university and institute will conduct stem cell research on a special type of cell called an astrocyte progenitor, which helps support the proper functioning and insulation of neurons. In ALS, also called Lou Gehrig's disease, insulation that is critical to neuron function decays over time. It is believed that astrocyte-based cell therapy could help regenerate neuron insulation and reverse the debilitating effects of the disease or slow down its progress.
UCSD and Salk investigators will study two methods of administering astrocyte progenitors in animal models, including cervical and lumbar spinal cord injections, Life Tech said. The researchers will test the safety and ability of these cells to prevent or slow down the deterioration of motor neurons in the hopes of providing a proof-of-principle, and bringing the approach to clinical trials within five years.
"Any novel treatment option would not only be clinically competitive, but could have a major impact for thousands of patients currently battling the disease," Don Cleveland, professor of medicine, neurosciences, and chair of the UCSD department of cellular and molecular medicine, said in a statement. "This approach has the potential to lead to the development of new therapies that could significantly extend the lifespan of individuals living with this disease, and improve their quality of life."
Currently, the only FDA-approved drug for ALS is riluzole, which is marketed by Sanofi-Aventis as Rilutek. About 30,000 people in the US have ALS.