For the first time, researchers have used a cocktail of small molecules to transform human brain cells, called astroglial cells, into functioning neurons for brain repair. The new technology opens the door to the future development of drugs that patients could take as pills to regenerate neurons and to restore brain functions lost after traumatic injuries, stroke, or diseases such as Alzheimer’s. Previous research, such as conventional stem cell therapy, requires brain surgery and therefore is much more invasive and prone to immune-system rejection and other problems. The research, led by Gong Chen, Professor of Biology and the Verne M. Willaman Chair in Life Sciences at Penn State University, will be published online in the journal Cell Stem Cell on Oct. 15th, 2015.
“We have discovered a cocktail of small molecules that can reprogram human brain astroglial cells into neuron-like cells after eight-to-ten days of chemical treatment,” Chen said. The neurons the researchers reprogrammed survived for more than five months in cell culture, where they formed functional synaptic networks. The scientists also injected the reprogrammed human neurons into the brains of living mice, where they integrated into the neural circuits and survived there for at least one month.
Astroglial cells before treatment with small-molecule cocktails in the lab of Gong Chen at Penn State University Credit: Gong Chen lab, Penn State University
Astroglial cells before treatment with small-molecule cocktails in the lab of Gong Chen at Penn State UniversityCredit: Gong Chen lab, Penn State University”The small molecules are not only easy to synthesize and package into drug pills, but also much more convenient for use by patients than other methods now being developed,” Chen said. Before the promise of the new technology results in pills at a pharmacy, the new research effort must first succeed through much development and testing in the laboratory and then through a series of clinical trials.
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