UC Irvine Health News
UC Irvine-led stem cell study points to new treatments for the fatal disorder
May 17, 2017
“This discovery can be used for possible future treatments to seal the leaky blood vessels themselves and to evaluate drug delivery to patients with HD,” says study leader UC Irvine professor Leslie Thompson. Photo by Steve Zylius / UC Irvine Strategic Communications.
Irvine, Calif. — By using induced pluripotent stem cells to create endothelial cells that line blood vessels in the brain for the first time for a neurodegenerative disease, University of California, Irvine neurobiologists and colleagues have learned why Huntington’s disease (HD) patients have defects in the blood-brain barrier that contribute to the symptoms of the fatal disorder.
“Now we know there are internal problems with blood vessels in the brain,” said study leader Leslie M. Thompson, PhD, a UC Irvine professor in the departments of Psychiatry & Human Behavior and Neurobiology & Behavior. “This discovery can be used for possible future treatments to seal the leaky blood vessels themselves and to evaluate drug delivery to patients with HD.”
The blood-brain barrier protects the brain from harmful molecules and proteins. It has been established that in Huntington’s and other neurodegenerative diseases there are defects in this barrier that add to HD symptoms. What was not known was whether these defects come from the cells that constitute the barrier or are secondary effects from other brain cells.
To answer that, Thompson and colleagues from UC Irvine, Columbia University, the Massachusetts Institute of Technology (MIT) and Cedars-Sinai Medical Center reprogrammed cells from HD patients into induced pluripotent stem cells, then differentiated them into brain microvascular endothelial cells — those that form the internal lining of brain blood vessels and prevent leakage of blood proteins and immune cells.
The researchers discovered that blood vessels in the brains of HD patients become abnormal due to the presence of the mutated Huntingtin protein, …