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Morning Briefing

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Monday, May 6 2019

Full Issue

Parents Of Children With Rare Genetic Disorder See Hope In Drug Trial, But Others See Litany Of Red Flags

Ovid Therapeutics' drug for Angelman syndrome--a rare cognitive disease that currently has no treatment--saw a glimmer of success in a very small trial. The drug had beaten the placebo on only one metric and failed on a full 16 others, including measures of quality of life and ability to sleep. To investors, the ostensibly positive data looked cherry-picked. In other news at the convergence of pharma and public health: Alzheimer's, dengue fever, superbugs, statins and more.

It鈥檚 called the clinical global impressions of improvement, or CGI-I, and it鈥檚 a seven-point scale doctors use to score whether patients are generally improving. According to Ovid, that makes it ideal for a disease like Angelman, in which patients鈥 symptoms can dramatically vary in both type and severity. One may struggle to sleep but be able to walk; another might be virtually immobile but have some speech skills. (Garde, 5/6)

Scientists are setting a new course in their quest to treat Alzheimer's disease. The shift comes out of necessity. A series of expensive failures with experimental drugs aimed at a toxic protein called amyloid-beta have led to a change in approach. The most recent disappointment came in March, when drugmaker Biogen and its partner Eisai announced they were halting two large clinical trials of an amyloid drug called aducanumab. (Hamilton, 5/3)

The Food and Drug Administration has approved the first vaccine for dengue, Dengvaxia, but placed significant restrictions on its use because the vaccine has been shown to put some people at heightened risk for a severe form of the disease. In clearing the vaccine, the agency acknowledged the serious public health benefit of slowing a disease that affects hundreds of millions of people around the world. The decision may also help a struggling product whose use has stalled because of concerns over its possible risks. (Thomas, 5/3)

The U.S. Food and Drug Administration just approved one of the most sought after vaccines in recent decades. It's the world's first vaccine to prevent dengue fever 鈥 a disease so painful that its nickname is "breakbone fever." The vaccine, called Dengvaxia, is aimed at helping children in Puerto Rico and other U.S. territories where dengue is a problem. (Doucleff, 5/3)

In early 2001, Joe Ng boarded a ship in the Azores to collect samples of mud. It wasn鈥檛 just any old boat: It was the Akademik Mstislav Keldysh, the 401-foot Russian research vessel used by filmmaker James Cameron to help capture deep-sea footage for the blockbuster film 鈥淭itanic.鈥 And it wasn鈥檛 just any kind of mud: It was samples from a hydrothermal vent field thousands of feet below the Atlantic Ocean鈥檚 surface where some of the world鈥檚 tiniest and toughest organisms flourished. The $50,000 ticket bought Ng, a molecular biologist, roughly two weeks at sea. Afterward, Ng traveled back to Huntsville, Ala., with an insulated lunch cooler stuffed with plastic sandwich bags of mud. Back in his lab, he occasionally subjected these 鈥渆xtremophiles鈥 鈥 a word derived from Latin (extreme) and Greek (love) 鈥 to various tests. (Blau, 5/5)

Statin drugs like Lipitor and Crestor significantly reduce the risk for cardiovascular disease, rigorous studies have shown. But a 鈥渞eal-world鈥 analysis published in Heart suggests that in practice about half of patients do not benefit from them. British researchers studied 165,411 patients free of cardiovascular disease who started statin therapy between 1990 and 2016. They measured their LDL levels (the 鈥渂ad鈥 cholesterol) before the study began and again after 24 months, and then followed them for an average of six years. (Bakalar, 5/3)

A prominent U.S. lawmaker may be upset that AbbVie (ABBV) ties the bonus given chief executive Richard Gonzalez to sales of the Humira rheumatoid arthritis treatment, but most shareholders seem unconcerned. At the drug maker鈥檚 annual meeting on Friday, an overwhelming majority of stockholders rejected a proposal that would have required AbbVie to compile an annual report on how pricing is used to set executive compensation, according to a coalition of faith-based investor groups that submitted the proposal. On a preliminary basis, 69% of shareholders voted down the proposal. (Silverman, 5/3)

This is part of the Morning Briefing, a summary of health policy coverage from major news organizations. Sign up for an email subscription.
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