Stem Cells: A weapon for Huntington's?


SAN FRANCISCO, Cali – Huntington's is a deadly, inherited disease that affects about 30,000 Americans. 150,000 more are at risk. Until now, there has been no hope for these patients who typically die of the disease within 15 years of diagnosis. But for the first time, scientists are studying a therapy that could slow down this killer, and stem cells are the weapon. 

Mike and Katie have been a couple since college, but they've known each other much longer.

"We've been together forever; I've actually known Mike since I was 5 years old," said Katie.

A marriage and three kids later, they've been through good times and bad. The worst came nine years ago when mike found out he had Huntington's disease. 

"My father had it, he died from it" Mike explained.

Huntington's causes uncontrollable movements and mental decline. There's no cure.

"Unfortunately, it ends in death. It's a fatal disease," said Vicki Wheelock, M.D., Neurologist, Health Sciences Clinical Professor of Neurology and Director of HDSA Center of Excellence at UC Davis.

Now, researchers are gearing up for a new trial in humans. Patients will have special bone marrow stem cells injected directly into their brains.

"We've engineered them to make a growth factor that's like a fertilizer for the neurons," explained Jan Nolta, Ph.D., professor and director of the Institute for Regenerative Cures at UC Davis.

That growth factor, BDNF, restored healthy brain cells and reduced behavior deficits in mice. Researchers hope the stem cells will also be the answer to slowing the disease in humans. 

"To be able to work on this for the Huntington's community, it really feels like the most important thing in my career ever," Nolta said.

Mike is signed up to receive the stem cells. He's excited about the possibilities, especially for his kids. They each have a 50/50 chance of inheriting Huntington's.

"Kids, that's the biggest thing, I can deal with it myself, but if they get it, I'll die," he said.

For now, Mike and Katie are thankful there's hope.

This study will be a pilot trial and the injection will be given one time, after MRI imaging. In mice, the stem cells delayed progression of Huntington's for up to a year, which is about half of a mouse's lifetime. Researchers are unsure what the effect will be in humans.