Health Watch: Targeting Huntington's Disease

ORLANDO, Fla. -- About 30,000 people have Huntington's disease in the US and more than 200,000 people are at risk of inheriting it. It is a fatal genetic condition that causes the breakdown of nerve cells in the brain and leads to physical and mental deterioration. Huntington's currently has no cure, but one researcher and her team have developed an experimental therapy that may enhance patients' quality of life.

Danny and Katie Miller enjoy taking shots at life and snapping the surprises they find. But what didn't come as a surprise was Danny's diagnosis of Huntington's disease in 2017.

Danny shared, "my father had Huntington's. My sister has it too."

"The initial response was you know kind of devastation," said Katie.

With the likelihood of passing it down, the Millers decided not to have children.

"At this day and time, until there's a better answer for the quality of life, it seems like a bad idea," Danny said.

But a finding by researcher Amber Southwell, PhD at the University of Central Florida is giving them hope. Ionis Pharmaceuticals synthesized pieces of DNA, called ASOs, and Southwell injected that into the brains of mice to stop production of the mutant form of the Huntington protein that causes the disease. With the suppression of the mutant Huntington in mice and neurons in a dish ...

"We're able to either prevent the onset of symptoms or actually restore normal functions, if we start treatment after symptoms have begun," Southwell explained.

These findings are leading a pathway to human clinical trials. That's good news for Danny.

"Chances for longer and better quality of life that's not a cure, but it's a damn good start for sure," he said.

Southwell's study was the first of its kind to look at cognitive function improvement using ASOs for Huntington's disease. Right now, the life expectancy for someone with Huntington's is about 15 to 20 years after onset.