Scientists at the Gladstone Institutes have for the first time transformed skin cells into a functional network of brain cells. The research offers new hope in the fight against many neurological conditions because scientists expect that such a transformation of cells may lead to better models for testing drugs for devastating conditions such as Alzheimer's disease.
This research comes at a time of renewed focus on Alzheimer's disease, which currently afflicts 5.4 million people in the United States alone--a figure expected to nearly triple by 2050. Yet there are no approved medications to prevent or reverse the progression of this debilitating disease.
Investigator Yadong Huang, MD, PhD, was quoted as saying, "Many drug candidates, especially those developed for neurodegenerative diseases; fail in clinical trials because current models don't accurately predict the drug's effects on the human brain. Human neurons--derived from re-engineered skin cells--could help assess the efficacy and safety of these drugs, thereby reducing risks and resources associated with human trials."
Karen Ring, UCSF Biomedical Sciences graduate student and the paper's lead author explained, "We wanted to see whether these newly generated neurons could result in tumor growth after transplanting them into mouse brains. Instead we saw the reprogrammed cells integrate into the mouse's brain--and not a single tumor developed."
"If we can pinpoint which genes control the development of each neuron type, we can generate them in the petri dish from a single sample of human skin cells," said Dr. Huang. "We could then test drugs that affect different neuron types such as those involved in Parkinson's disease, helping us to put drug development for neurodegenerative diseases on the fast track."