Gene Therapy for Blindness, EMA's Bold Move (Abstract Science: Oct. 17-21)
Eyeing gene therapy, slaying the common cold and making clinical data more transparent.
(MIT Technology Review, 10/18/16, Emily Mullin)
The gene therapy field has seen its share of clinical failures, including a patient death in 1999 due to complications of an experimental treatment. But a novel treatment targeting mutations that cause blindness is propelling the field forward once again. Speaking at EmTech MIT 2016 on Tuesday, Katherine High, the founder of Spark Therapeutics, the company developing the gene therapy product, said they plan to submit their application to the US Food and Drug Administration next year. If they get the green light, it would mark the gene therapy for an inherited disease to be approved in the US. Two other gene therapies, Strimvelis and Glybera, have already been approved in Europe.
(STAT, 10/20/16, Carl Zimmer)
Since the 1960s, when scientists discovered the rhinovirus, researchers have been trying, without luck, to develop a vaccine to prevent it. That could be changing. Rather than design vaccines that induce antibodies again a virus’ surface proteins, the traditional avenue in vaccine development, researchers have been targeting the rhinovirus’ internal proteins, which spill into our cells and are used to make new viruses. Researchers from Emory have found success with their vaccine in animals, and are hoping to move soon to clinical trials. Other labs are also pursuing the same strategy.
(Nature, 10/20/16, Alison Abbott)
In a move lauded as a landmark for transparency in medical science, the London-based European Medicines Agency (EMA) has begun to publish details of the full clinical-trial data that it receives from pharmaceutical companies. These clinical study reports (CSRs) are far more detailed than the papers that drug firms publish in scientific journals – which studies have shown are an “incomplete source of information on new medicines”, says Larry Peiperl, the chief editor of PLoS Medicine. The CSRs include both positive and negative results, and details of drugs’ adverse effects.
—Compiled by Senior Scientific Writer Regina McEnery