The good stem cells

A decade of battling Parkinson’s disease left 69-year-old George Lopez unable to bike, swim, fish, or even tie his shoes. Constant pain plagued the physician, businessman, and inventor, and he struggled just to push himself out of a chair, Stat News reported. But thanks to an experimental new procedure, Lopez can once again enjoy many of those activities.

Parkinson’s, a degenerative brain disease that affects 6 million people worldwide, progressively destroys the brain cells that produce the neurotransmitter dopamine. As dopamine levels drop, patients experience worsening symptoms such as tremors, slow movement, stiffness, loss of balance, and speech difficulties.

In the late 1980s, surgeons stooped to transplanting dopamine-producing brain cells from aborted babies into the brains of scores of Parkinson’s patients. They used as many as 16 aborted infants per transplant, but the cells died in the patients’ brains.

Now researchers have developed a way to use a patient’s own skin cells to accomplish what fetal cells could not. They reprogrammed Lopez’s skin cells into stem cells designed to develop into dopamine-producing neurons. In 2017, doctors transplanted about 4 million of the stem cells into the three regions of his brain involved with movement. By using Lopez’s own cells, the researchers avoided the ethical problems of using fetal tissue, as well as the likelihood of triggering an immune response in which the body would reject the foreign cells.

Two years later, brain scans show the transplanted cells functioning as normal, dopamine-producing transmitters, the scientists reported on May 14 in The New England Journal of Medicine. The cells have even formed long-term connections with other cells. Lopez experienced no side effects and can swim, ski, bike, and tie his shoes again.

Though the experiment avoided the ethical and moral problems of using cells from aborted babies, it has raised other concerns about whether Lopez had an unfair advantage in accessing the treatment. He gave $2 million to support early research that laid the groundwork for the stem cell transplant he received.

The researchers cautioned that, even though the results are exciting, the procedure did not cure Lopez. He still needs to take nearly as much medication as before. And they don’t know if the procedure will work for everyone. The researchers will need to demonstrate the technique’s efficacy in much larger human clinical trials.

“Parkinson’s patients should understand that this therapy is not currently available and there is a lot of work still required to prove this is an effective treatment,” said Todd Herrington, the study’s lead neurologist.