Modifying stem cells to treat Parkinson’s

Stem cells, with their ability to regenerate and take on different functions, have long been viewed as a potential source for treating Parkinson’s disease.

Unanswered questions remain, however, about how best to deliver stem cells to the areas of the brain Parkinson’s damages, and how to make sure the new cells survive and thrive once they are transplanted.

Tiago Cardoso, a post-doctoral fellow at Laval University, is among the researchers who believes stem cells could be a vital source of new brain cells to replace the dopamine-generating cells that die as Parkinson’s disease progresses.

Working in petri dishes, he’s using cultured stem cells derived from the skin of donors to see if he can transform them into these dopamine-producing neurons.

He will then transplant the stem cells into mouse models to see if they can reduce or eliminate the motor symptoms of Parkinson’s disease.

“We hope that we get a stem cell product that is safe and good – that works – that can in the future be used for actual transplantation in order to treat people with Parkinson’s.”

But before Cardoso gets to the transplantation stage, he’s using genetic engineering techniques to improve the stem cells.

“We would modify them, first to make them more prone to survive the transplantation procedure, and second to help them connect better to the damaged areas, to rewire the damaged circuits or pathways in the brain,” Cardoso says.

If Cardoso’s modifications work, and his altered stem cells can replace damaged dopamine neurons in mice, that would suggest further clinical trials.

Clinical trials are already under way in other jurisdictions involving stem cells that have not been modified the same way Cardoso plans to change them. His work could build on this other research.

“We hope that we get a stem cell product that is safe and good – that works – that can in the future be used for actual transplantation in order to treat people with Parkinson’s,” Cardoso says. “We hope to show this is a valid strategy.”

Even as a youth growing up in Portugal, Cardoso enjoyed learning about the natural sciences and chemistry. But his fascination with the biological processes that underlie our complex behaviour drew him to study neuroscience.

The brain is “like the universe of the human body, in terms of how much there is left to be known and how complex it is,” he says.