Local scientific discovery could lead to better treatments for Huntington’s and other diseases

KANSAS CITY, Mo. (KCTV) - Researchers at the Stowers Institute for Medical Research have made a stunning discovery that could change the way Huntington’s, Parkinson’s and even Alzheimer’s disease are treated.

The three diseases share similar traits. They all start in the brain, and they all involve clumps of proteins that spread through the brain. So, advances in treatment for one disease have clear implications for the others.

The discovery at Stowers was made studying Huntington’s disease. It’s a rare, inherited disease that causes the breakdown of nerve cells in the brain. Over time, a person with the disease loses control of motor and mental skills, and eventually dies. Currently, there is no cure. But now, there’s hope that could change.

Randal Halfmann studies molecules associated with Huntington’s disease. It’s known that the disease starts with a genetic mutation. But, for the first time, Halfmann has discovered the origin of the disease within each cell. It’s a problem that has stumped scientists around the world for decades.

“So, it’s almost like a little zombie,” explained Halfmann. “It’s bringing other proteins to it and converting them.”

Halfmann has been working on identifying what sparks the disease for 12 years. He likens the conversion to a zombie apocalypse. He first identified the “zombie attack” using yeast as a test subject.

“We used yeast as really tiny test tubes,” explained Halfmann. “We were able to see what the critical event looks like that starts the disease.”

Scientists say identifying that critical event could be the start of a cure.

“What we found is: If we bring them together before they convert, then we stop them from converting in the first place,” said Halfmann. “The idea is: If we’re able to find a molecule to do the same thing, we have a possible drug.”

Dr. Richard Dubinsky is the director of the Huntington’s Disease Society of America Center of Excellence at the University of Kansas Medical Center. He has treated people with the disease for more than three decades.

“If this comes to fruition, there would be a treatment that could halt the progression of the disease or even prevent it from starting,” said Dr. Dubinsky. “It’s very exciting and, hopefully, it will pan out.”

There are some 30 different amyloid diseases, which are those involving clumps of proteins. So, if a drug can be developed to stop Huntington’s, scientists believe the same concept could be applied to dozens of other diseases as well.

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