Alternative to stem cell therapy discovered for treating chronic brain diseases

Scientists in Korea have reported the first successful use of a drug-like molecule to transform early-forming human muscle cells into nerve cells. The researchers exposed immature mouse muscle cells called myoblasts to neurodazine, a synthetic small molecule. They observed that after a week, about 45% of the myoblasts transformed into cells that resembled both the structure and function of nerve cells, including expression of neuron-specific proteins. "In conclusion, we have developed the first small molecule that can induce neurogenesis of non-pluripotent myoblasts and the cells derived from mature, human skeletal muscle," the study authors wrote. "These studies build upon recent research illustrating the value of chemical approaches for providing tools that differentiate lineage-committed cells into other cell types." The results of this study may lead to potential treatments for stroke, Alzheimer’s disease, Parkinson’s disease and other neurological disorders. A fully developed treatment like this would be a breakthrough for chronic brain disease treatment and would provide an alternative to stem cell therapies.

Researchers from the University of Wisconsin-Madison have shown that cell-based therapies can rescue the dying neurons characteristic of amyotrophic lateral sclerosis (ALS), a fatal neuromuscular disorder also known as Lou Gehrig’s disease. The study, reported in the August 1 issue of PLoS ONE, showed that stem cells engineered to secrete a key growth factor can protect the motor neurons that waste away as a result of ALS. "At the early stages of disease, we saw almost 100% protection of motor neurons," explained Clive Svendsen, neuroscientist and lead author of the study. "But when we looked at the function of these animals, we saw no improvement: the muscles aren't responding." The researchers implanted embryonic stem cells which were engineered to secrete a chemical known as glial cell line derived neurotrophic factor (GDNF), an agent that has been shown to protect neurons. "The (GDNF secreting) cells survive beautifully. In 80 percent of the animals, we saw nice maturing transplants," said Svendsen.

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