Promising clinical trial results for drug for rare disease in which patients can't eat fat

ScienceDaily | 8/8/2019 | Staff
smnth28 (Posted by) Level 3
Joseph Witztum, PhD, professor of medicine at University of California San Diego School of Medicine, recently helped lead a global research team that tested how well the drug volanesorsen reduces fat accumulation in the blood (triglycerides) of participants with familial chylomicronemia syndrome. In a randomized, double-blind Phase III clinical trial, 33 participants received the drug and 33 received a placebo.

Volanesorsen reduced triglyceride levels by an average of 77 percent. In contrast, for participants who received a placebo, triglyceride levels increased by an average of 18 percent.

Results - August - New - England - Journal

These results will publish August 8, 2019 in the New England Journal of Medicine.

Chylomicrons carry dietary triglycerides from the gut into the blood, where they are normally broken down by the enzyme lipoprotein lipase. Without that enzyme, chylomicrons accumulate and thicken the blood of people with familial chylomicronemia syndrome.

Chylomicrons - Form - Top - Milk - Witztum

"Chylomicrons are what you see when you see cream form on the top of milk," Witztum said. "For most people, when you eat a fatty meal for dinner at 6:00 p.m., the triglycerides are transported into the blood by chylomicrons and then lipoprotein lipase clears those out of the blood by 9:00 p.m. That's definitely not the case for patients with familial chylomicronemia syndrome."

Triglyceride levels are regulated by a molecule called apolipoprotein C-III, which is made in the liver, and then secreted into the blood. Volanesorsen, an antisense drug developed by Ionis Pharmaceuticals, is designed to block the mRNA in the liver that encodes apolipoprotein C-III. Reducing apolipoprotein C-III in the blood in turn reduces triglyceride levels. Triglycerides are often used as a measure for heart health, as high levels are associated with heart disease, even in...
(Excerpt) Read more at: ScienceDaily
Wake Up To Breaking News!
Do I know who I am?
Sign In or Register to comment.

Welcome to Long Room!

Where The World Finds Its News!