May 26, 2017

Rare mutations of gene inspire new heart drugs

What if you carried a genetic mutation that left you nearly impervious to heart disease? What
if scientists could bottle that miracle and use it to treat everyone else?

In a series of studies, the most recent published on Wednesday , scientists have described two
rare genetic mutations that reduce levels of triglycerides, a type of blood fat, far below
normal.People carrying these genes seem invulnerable to heart disease, even if they have
other risk factors.

Drugs that mimic the effects of these mutations are already on the way , and many experts
believe that one day they will become the next blockbuster heart treatments. Tens of millions
of Americans have elevated triglyceride levels. Large genetic studies have consistently
suggested a direct link to heart disease.

Added to the existing arsenal of cholesterol-reducers and blood pressure medications, the
new medications “will drive the final nail in the coffin of heart disease,“ predicted Dr. John
Kastelein, a professor of vascular medicine at the University of Amsterdam who was not
involved in the new research. These experimental triglyceride-reducers are in early stages of
development, however, and human trials have only just begun.

At the moment, the optimism of researchers is rooted less in clinical trial data than in the fact
that nature has produced strong evidence they should work. Finding people who are
impervious to a disease like heart disease can open a door to letting the rest of the population
share their genetic luck.

“It's a huge advance,“ said Dr. Christie Mitchell Ballantyne, chief of cardiology and
cardiovascular research at Baylor College of Medicine and a consultant for Regeneron
(although not for the triglyceride studies). “That doesn't mean it's easy .“ Still, he added,
“what we are seeing is a new approach toward drug development.“

Source: The Times of India

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