Campus Connection: UW research hints at potential for Huntington’s treatment

Todd Finkelmeyer has been covering higher education for the Capital Times since April 2008. He started contributing to the newspaper in 1990, was hired full-time in 1994 and has since covered everything from the Super Bowl to stem cell research. Follow his Campus Connection blog for the latest on higher education news in the Madison area.

Researchers working on the University of Wisconsin-Madison campus have found a way to use neurons derived from human embryonic stem cells to restore muscle coordination in mice inflicted with a Huntingtons disease-like condition.

The findings, which were reported Thursday in the journal Cell Stem Cell by a team of scientists who work at the university's Waisman Center, could one day help lead to new therapies for Huntington's disease, the debilitating disorder that affects both muscle coordination and cognitive ability. There currently are no effective treatments.

This is very exciting, and next well try to move onto different models, particularly in primates, to see whether this actually works in a larger brain, says Su-Chun Zhang, a UW-Madison neuroscientist and the senior author of the study.

Zhang, who specializes in producing different types of brain cells from stem cells, explains that this particular research focused on GABA neurons -- the cells that deteriorate and thus disrupt the brains circuitry that leads to the loss of motor function in patients with Huntington's. He notes the GABA neurons make a chemical that plays an important role in helping to link the communication network in the brain with movement.

Zhang and co-workers in the lab figured out how to produce large amounts of these GABA neurons from human embryonic stem cells, which they tested in mice with a Huntingtons-like ailment. Initially, the researchers were just trying to see whether or not these cells could safely integrate into the mouse brain. They were stunned to see that the cells not only successfully merged into the brain, but that they also eventually sent signals to the proper targets and rewired broken circuitry to restore motor functions.

This was quite surprising, says Zhang. These human neurons actually projected a long distance to another place in the brain and hooked up at the circuit, which is essential for our movement coordination. This is very critical, because in order for cell therapies to work for Huntingtons, the circuit has to be re-formed.

He notes scientists generally didnt believe it was possible for this circuitry to be rewired in older brains. In the mature brain, the nerve cells do not project a long distance, particularly into the correct target, Zhang explains. But that happened with these cells. So the point is, these stem cells somehow know where to go.

Its not clear how relevant this information will be in finding treatments for Huntingtons, but the UW-Madison scientists are hopeful their research can be used as another building block of information that can one day lead to treatments for the debilitating disease.

According to the Huntington's Disease Society of America, more than a quarter of a million Americans have the ailment or are at risk of inheriting the disease from an affected parent.

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Campus Connection: UW research hints at potential for Huntington’s treatment