A setback for a muscular dystrophy drug, yellow fever’s comeback, beads that mimic human eggs, and a boost for HIV vaccine research. This week in Abstract Science.
(STAT, 4/25/2016, Ed Silverman)
An advisory panel for the US Food and Drug Administration ruled this week that Sarepta’s experimental drug eteplirsen wasn’t effective against the incurable inherited disorder Duchenne muscular dystrophy (MD). The decision runs contrary to the testimonials of dozens of families claiming that the drug worked well in clinical trials. MD is caused by an absence of the protein dystrophin that keeps muscles intact. It usually strikes males. The fate of the Sarepta drug has been closely watched as a litmus test for an intensifying struggle between the FDA and patient groups that want the agency to take a more expansive view toward approving medicines for unmet medical needs.
(Wired Science, 4/25/2016, Sarah Zhang)
An outbreak of yellow fever in Angola that has sickened nearly 1,000 people and claimed 258 lives highlights what can occur when routine vaccinations lag. A years-long shortage of vaccine has left millions of people vulnerable to the mosquito-borne virus in sub-Saharan Africa. Only about 70% of the population is protected, far lower than the threshold needed to prevent outbreaks like this. A lack of suppliers is to blame for the shortages. Only four places make the yellow fever vaccine, and a government-run facility in Dakar, Senegal is shutting down soon for renovations.
(New Scientist, 4/27/2016, Sam Wong)
Polymer beads that mimic human eggs by tricking sperm to bind to them could work as a new form of contraceptive, while also helping fertility doctors select the best sperm for IVF. A team from the US National Institute of Diabetes and Digestive and Kidney Diseases have tested the beads in mice, with good results. The beads are coated in a protein called ZP2, the same one that sperm recognize and bind to on the egg’s surface so that they can fertilize it. Since binding to ZP2 is a vital step for fertilizing an egg, the beads can be used to select the best possible sperm for IVF.
(Nature, 4/27/2016, Erika Check Hayden)
A study led by researchers from the US National Institute of Allergy and Infectious Diseases has found that a single infusion of antibodies protected a large animal species from HIV for up to six months. While animals are not humans, the findings support the use of passive antibodies as a long-term prevention against HIV. Researchers have struggled to produce an effective vaccine against HIV, and the scientists behind this study say that administering periodic doses of antibodies might provide a stopgap measure while vaccine research continues. The findings were published this week in Nature.
—Compiled by Senior Scientific Writer Regina McEnery