Specially engineered neural cells transplanted into the brains of patients with amyotropic lateral sclerosis (ALS) delayed disease progression and extended survival in animals, according to researchers at Cedars-Sinai.
In the study, published recently in the journal Stem Cells, investigators genetically reprogrammed neural progenitor cells to secrete a special protein known as GDNF and then transplanted the cells into the brain cortices of animal models of ALS.
GDNF helps sustain glial cells, which support the body’s motor neurons. In ALS patients, glial cells lack certain proteins and become sick, and the motor neurons gradually die off, causing paralysis.
Once inside the cortex, the transplanted cells in the study matured into glial cells and released GDNF into the brain. Laboratory rats that received the transplants lived 8% longer and were free of paralysis 10% longer than were untreated animals. Motor neurons in the spine, which control muscle movement, also survived longer in the experimental group, explains a media release from Cedars-Sinai.
“If we are able in the future to reproduce our research results in humans, we could improve both the quality and length of life for patients diagnosed with this devastating disease,” says the study’s first author Gretchen Thomsen, PhD, assistant professor of Biomedical Sciences and a research scientist at the Cedars-Sinai Board of Governors Regenerative Medicine Institute.
While the research results showed promise, more preclinical studies are needed to determine which treatment levels may be adequate and safe, Thomsen adds. Cedars-Sinai investigators currently are performing these studies as preliminary steps to creating proposals for clinical trials, the release continues.
[Source(s): Cedars-Sinai, Newswise]
