closer look
Despite FDA approval, insurance doesn't always cover gene therapy
Courtesy Deb Jenssen
Sarah Jenssen, 15, is one of the few girls who have Duchenne muscular dystrophy. Though she can still use the bathroom independently and dress herself, she has lately started relying on a wheelchair. Her family celebrated when the FDA threw open the gates on a gene therapy for the condition earlier this year, widening the approval for Sarepta's Elevidys to nearly everyone regardless of age or disease progression.
But getting insurance to pay for Sarah's $3.2 million gene therapy hasn't been easy. The family's insurer first denied it because Sarah wasn't able to walk independently anymore, even though that went against the FDA's label. The issue is complicated: There's limited data on the treatment's efficacy for people who are older than 5 years old or use wheelchairs. But such patients also have few options.
"You spend all this time to get the FDA to approve it, and then you have to spend even more time with insurance companies to get them to approve it," said Jonathan Soslow, one of Sarah's doctors. Sarah and her family were relieved to hear the insurer recently agreed to cover the therapy. Read more from STAT's Andrew Joseph.
Science
Have you ever almost choked on food? You have these cells to thank for your survival
You never really think about how miraculous it is that we don't continuously choke on stuff (why do our air tube and our food tube share an entrance??) until you get some water down the wrong pipe or inhale a clump of rice and start hacking and coughing like crazy.
Scientists knew that nerve sensors in the trachea and larynx in our throats helped detect objects that were not supposed to be there. But earlier this year, Laura Seeholzer, a postdoc at UC San Francisco, showed that special kinds of epithelial cells called neuroendocrine cells help those nerves by recognizing water and acid and triggering a coughing or swallowing response.
Seeholzer is this year's recipient of the Eppendorf & Science Prize for Neurobiology, which recognizes outstanding young neurobiologists for their research and comes with $25,000.
Biotech
ARPA-H grant goes to lab using AI to discover new antibiotics
The latest grant from the Advanced Research Projects Agency for Health, also known as ARPA-H, is for AI-powered antibiotics.
The agency announced yesterday that it is awarding a $27 million grant to Phare Bio and Jim Collins' lab at MIT/Harvard's Wyss Institute. (The lab was recently profiled in a New Yorker article). Using AI, the team plans to develop new classes of antibiotics and also create an open-source database for AI-based antibiotic discovery so that other scientists can use the information the team generates along the way. The goal is to have 15 new, AI-generated preclinical antibiotic candidates by the end of the five-year project.
Phare Bio describes itself as a "social venture" rather than a drug development startup. Its plan to get antibiotics past the "valley of death" in early stage development where most drugs fail is to first use donor and grant funding, then turn to more traditional commercial partnerships and company spinouts to take the drugs through clinical development.
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