Parkinson's, Huntington's disease research makes advances with stem cells: Discoveries

CLEVELAND, Ohio-- Imagine cooking up a new recipe for carrot cake and trying to figure out what it tastes like by feeding it to your dog. You might be able to learn something from his reactions -- Does he eat some? A lot? Does he, heaven forbid, keel over afterward? -- but you'd be pretty limited by some basic differences between you and your canine friend. Even if he could somehow tell you what he thinks, there's just no telling if cake tastes the same to a dog.

This is something like the problem faced by researchers who are trying to understand and treat devastating human brain diseases like Parkinson's and Huntington's by working with mice.

The mouse brain has told us a lot about the diseases, but, in the end, it's only a stand-in for working with the real thing.

Now the real thing is here. Two groups of Parkinson's and Huntington's researchers working in 13 labs nationwide have used advanced stem-cell technology to make human brain cells from skin cells donated by patients with those diseases. The brain cells look and act like cells affected by the diseases, and they can be manipulated in a petri dish.

Working with the new cells in a petri dish is a little like taking a bite of your recipe and getting your own reaction, without the potential of making yourself sick.

It's a first for the field, says Dr. Christopher Ross, one of the Huntington's disease study's lead researchers and professor of psychiatry and behavioral sciences at the Johns Hopkins University School of Medicine in Baltimore.

"It's going to be a tremendous opportunity to study the disease, to understand it, and particularly to develop therapeutics," he says.

Huntington's disease is inherited and caused by a defect in a single gene. The disease is progressive and fatal, causing twitching and jerking movement, dementia and brain-cell death. It affects about 30,000 people in the United States. Parkinson's, while not fatal, affects about 1 million Americans and causes progressively worsening movement problems as well as mood and sleep disruptions.

The technology that made the recent advance possible, called induced pluripotent stem cells, or iPSCs, was developed about four years ago simultaneously at the University of Wisconsin and in Japan.

In short, iPSCs are adult cells (usually skin or blood cells) taken from a donor with the disease and then genetically reprogrammed, or induced, back to their most primitive state. Once they are turned into stem cells, they can be forced to develop into any cell in the human body.

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Parkinson's, Huntington's disease research makes advances with stem cells: Discoveries