A new U study suggests that researchers can save thousands of lives by using a cholesterol-treatment drug for patients with blood vessel disorders that cause sometimes-fatal seizures and strokes.
Millions of patients take statin drugs to lower cholesterol every year, but U researchers found that the drug could help reduce leaking in a blood vessel disorder that many people are unaware they have until a stroke or paralysis hits them.
“We’ve been interested in (the disorder) for a number of years because, for one reason, it’s so common,” said Kevin Whitehead, the study’s lead author and an internal medicine professor at the U School of Medicine. “One out of every 200 people randomly put on a scanner has it.”
Whitehead said his research team began studying the drug on genetically engineered mice using a similar technology that U genetics researcher Mario Capecchi pioneered and that led him to receive the Nobel Prize in Physiology or Medicine in 2007.
Researchers injected blue dye into mice’s blood streams and also injected a stimulus to make the dye leak in a way similar to how blood would leak in humans with a blood vessel disorder. U biology graduate student Aubrey Chan said the drug-injected mice had less blue dye leaking than the control group of mice that were not given a stimulus to force leaking into the blood stream.
Whitehead said they want to generate a mouse with the same blood vessel problems as a human, but mice have a shorter life span and different blood vessel structure.
In the meantime, researchers are organizing a small clinical trial to test the drug on humans.
“I’m not sure how effective it will be on people,” Whitehead said. However, the U researcher is already inviting patients with a certain blood vessel disorder to consider a trial.
There is no existing treatment for patients with the disorder.
Other researchers have considered the possibility of using the statin drug for blood vessel leaking and have shown that the drug is effective in blocking a signaling pathway that becomes overactive in patients with the blood vessel disorder.
“People with the (disorder) break down vessel barriers and the blood goes into the head, scarring and causing seizures and strokes from the blood,” Whitehead said.
In the mice used for the tests, the statin drug began working within 24 hours to clear up the blue dye.
Although the drug seems to work well in mice, human trials could prove differently. Whitehead said trials could start soon, but he thinks they will need at least two to three years to know if the drug is working right and what the side effects might be.
“There’s still a lot of work to be done, and we plan to look at a third gene that is supposed to play a part in (the disorder),” Whitehead said.
Chan and Whitehead worked with cardiologist Dean Li, director of the molecular medicine program, as well as a number of graduate and postdoctoral students from the U, to finish the study.
The study was published Jan. 18 in the online publication Nature Medicine.