New Brain Treatments Possible After Stem Cell Breakthrough

Scientists at the Burnham Institute for Medical Research have genetically programmed embryonic stem (ES) cells to become nerve cells when they are transplanted into the brain. This breakthrough may pave the path for new treatments for neurological disorders.

Headed by Stuart A. Lipton, M.D., Ph.D., professor and director of the Del E. Webb Neuroscience, Aging, and Stem Cell Research Center at Burnham, the study has paved the way for developing new treatments for stroke, Alzheimer's, Parkinson's and other neurological conditions.

The researchers showed that mice afflicted by stroke showed tangible therapeutic improvement following transplantation of these cells. None of the mice formed tumors, which had been a major setback in earlier attempts at stem cell transplantation.

"We found that we could create new nerve cells from stem cells, transplant them effectively and make a positive difference in the behavior of the mice. These findings could potentially lead to new treatments for stroke and neurodegenerative diseases such as Parkinson's disease," said Lipton.

Conditions such as stroke, Alzheimer's, Parkinson's and Huntington's disease destroy brain cells, causing speech and memory loss and other debilitating consequences. It is believed that transplanting neuronal brain cells could restore at least some brain function, just as heart transplants restore blood flow.

Prior to this research, creating pure neuronal cells from ES cells had been problematic as the cells did not always differentiate into neurons. Lipton and his team found a solution to this problem by inducing ES cells to express a protein, discovered in his laboratory called myocyte enhancer factor 2C (MEF2C). MEF2C is a transcription factor that turns on specific genes which then drive stem cells to become nerve cells.