Biotechnology
Rare mutation prompts race for cholesterol drug
Health Headlines
By
Gina Kolata
She was a 32-year-old aerobics instructor from a Dallas suburb — healthy, college-educated, with
two young children. Nothing out of the ordinary, except one thing.Her cholesterol was astoundingly low. Her low-density lipoprotein, or LDL, the form that promotes heart disease, was 14, a level unheard-of in healthy adults, whose normal level is over 100.
The reason: a rare gene mutation she had inherited from both parents. Only one other person, a young, healthy Zimbabwean woman whose LDL cholesterol was 15, has ever been found with the same mutation.
The discovery of the mutation and of the two women with their dazzlingly low LDL levels has set off one of the greatest medical chases ever. It is a fevered race among three pharmaceutical companies, Amgen, Pfizer and Sanofi, to test and win approval for a drug that mimics the effects of the mutation, drives LDL levels to new lows and prevents heart attacks. All three companies have drugs in clinical trials and report that their results, so far, are exciting.
“This is our top priority,” said Dr. Andrew Plump, the head of translational medicine at Sanofi. “Nothing else we are doing has the same public-health impact.”
Dr. Gary H. Gibbons, the director of the National Heart, Lung, and Blood Institute, estimates that even if the drugs were expensive and injected, as many as 2 million Americans might be candidates. But if they could eventually be made affordable and in pill form — two very big ifs — they might be used by 1 in 4 adults, he said.
So far, people with stubbornly high cholesterol levels who are taking the drugs in preliminary studies have seen their LDL levels plunging from levels well over 100 to 50, 40 or even lower. Like insulin for diabetes, the drugs are injected, but they are taken once or twice a month.
Dr. Barry Gumbiner, who is directing Pfizer’s studies, said the company had to decide whether to set a floor for patients’ LDL levels. Pfizer is interrupting treatment when LDL levels reach 25 or lower. The people seemed fine, but the company got nervous.
“There is not a lot of experience treating people to LDL levels this low,” Gumbiner said.
And there is another concern: cost. Each company’s drug is a biologic, a so-called monoclonal antibody made in living cells at an enormous expense, like some new cancer drugs that are already straining the medical system.
Insurers generally pay for drugs approved by the Food and Drug Administration, and the number who might benefit from these cholesterol drugs dwarfs those who are helped by the biologic cancer drugs.
David Mayse, 60, who lives in South Point, Ohio, was 49 when he had his first heart attack while at work in a paper recycling plant. His doctor plied him with four different drugs to lower his cholesterol, to little avail. Then Mayse had another heart attack and bypass surgery. His doctor sent him to a cardiologist who called his cholesterol levels “outrageous” and asked Mayse if he would enter a clinical trial.
“I was willing to try anything at that point,” Mayse said.
In February 2012, he saw Dr. Evan A. Stein, who heads the Metabolic and Atherosclerosis Research Center in Cincinnati. Mayse’s LDL was 160 even though he was taking Vytorin, a combination of a statin and another drug to lower LDL levels.
Mayse enrolled in a study for Amgen’s experimental drug. His LDL fell to 42.
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