Researchers at the Gladstone Institutes have successfully transformed skin cells into a functional network of brain cells, which could offer new solutions in the fight against neurological conditions.

There has been a renewed focus on Alzheimer’s disease of late, and this research opens up several new avenues for exploration in relation to neurological conditions. Scientists involved on the project hope that the ability to reprogram cells will lead to better models for testing drugs for devastating neurodegenerative conditions, such as Alzheimer’s disease, of which there are currently no approved medications to prevent or reverse the progression of this debilitating disease.

A team of researchers in the laboratory of Gladstone Investigator Yadong Huang, MD, PhD, found a way to transfer a single gene called Sox2 into human skin cells. The cells were transformed into early-stage brain stem cells within days, which began to self-renew until they became neurons fully capable of transmitting electrical signals. After a month, the neurons had developed into neural networks.

Dr. Huang, who is also an associate professor of neurology at the University of California, San Francisco (UCSF) said: “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 reengineered skin cells – could help assess the efficacy and safety of these drugs, thereby reducing risks and resources associated with human trials.”

Dr Huang has spent several years of research in this area, but today takes a new tack to most of his other research by using one genetic factor – Sox2 – to directly reprogram one cell type into another without reverting to the pluripotent state. He now hopes to identify similar regulators that guide the development of specific neural progenitors and subtypes of neurons in the brain.

He said:”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.

“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.”