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.
Heart disease remains the leading killer of Americans, causing nearly 600,000 deaths a year.
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.
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