Photo: AFP
LOS ANGELES (CNS) - Cedars-Sinai has been given nearly $12 million by California's stem cell agency to launch a clinical trial to test a potential gene and stem cell therapy for amyotrophic lateral sclerosis, commonly known as Lou Gehrig's disease, the hospital announced today.
The progressive neurodegenerative disease damages nerve cells known as motor neurons in the brain and spinal cord that control voluntary muscle movement. ALS affects an estimated 16,000 people in the U.S. and is usually fatal within five years of diagnosis. There is no known cure and only limited treatments that may slow progression in some patients.
According to a hospital statement, the $11.99 million award from the California Institute for Regenerative Medicine “builds on earlier support from the stem cell agency enabling Cedars-Sinai to investigate the use of stem cells delivered to the spinal cord to treat ALS patients.''
“This disease remains a remarkably stubborn and heartbreaking disorder. The new grant allows us to follow the science wherever it takes to best serve our patients," said Clive Svendsen, executive director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute and a professor of Biomedical Sciences and Medicine.
Aided by the CIRM grant, Cedars-Sinai plans to enroll 16 patients in a clinical trial to demonstrate the safety and preliminary efficacy of transplanting neural progenitor cells secreting a powerful growth factor into the brain cortices of ALS patients to slow the progression of the disease.
According to Cedars, the neural progenitor cells for the trial have been genetically reprogrammed to express GDNF, a protein that promotes survival of special cells called astrocytes that support motor neurons. In ALS patients, astrocytes may become sick and provide less support to motor neurons, which then gradually die, leading to paralysis.
“The trial's investigators, from the Regenerative Medicine Institute and the departments of Neurology and Neurosurgery at Cedars-Sinai, hypothesize that the engineered neural progenitor cells, when introduced into the brain cortices of ALS patients, will differentiate into astrocytes and secrete GDNF locally to keep the motor neurons healthy and ultimately slow the progression of ALS,'' according to a hospital statement.
The design of the trial builds on years of research at Cedars-Sinai that demonstrated a similar technique delayed disease progression and extended survival in rodent models of ALS.
Investigators from the Cedars-Sinai Board of Governors Regenerative Medicine Institute are currently analyzing data from a U.S. Food and Drug Administration-approved clinical trial that infused these same cells into the spinal cords of ALS patients. That trial, and the research that laid the groundwork for it, also was funded by CIRM.
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