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.
Stat:
Scientists See Hope In Rare Disease Drug Wall Street Has Doubted
It’s called the clinical global impressions of improvement, or CGI-I, and it’s 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’ 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)
NPR:
With Alzheimer's Drugs Still Elusive, Scientists Now Look Beyond Amyloid
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 New York Times:
F.D.A. Approves The First Vaccine For Dengue Fever, But Limits Its Use
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)
WBUR:
Rush To Produce, Sell Vaccine Put Kids In Philippines At Risk
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 — 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)
The Washington Post:
Microbes Called Extremophiles Might Combat Superbugs, Biowarfare Agents
In early 2001, Joe Ng boarded a ship in the Azores to collect samples of mud. It wasn’t 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 “Titanic.” And it wasn’t just any kind of mud: It was samples from a hydrothermal vent field thousands of feet below the Atlantic Ocean’s surface where some of the world’s 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 “extremophiles” — a word derived from Latin (extreme) and Greek (love) — to various tests. (Blau, 5/5)
The New York Times:
Half Of People Miss Benefits Of Statins
Statin drugs like Lipitor and Crestor significantly reduce the risk for cardiovascular disease, rigorous studies have shown. But a “real-world” 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 “bad” cholesterol) before the study began and again after 24 months, and then followed them for an average of six years. (Bakalar, 5/3)
Stat:
Most AbbVie Shareholders Unfazed By Tying CEO's Bonus To Humira
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’s 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)
