Get your daily dose of health and medicine every weekday with STAT’s free newsletter Morning Rounds. Sign up here.
Good morning, everyone. Now that we’ve all narrowly survived a workday without the World Cup, we can at last return to our new normalcy this afternoon for France 3-1 Morocco.
A father’s quest to cure his daughter
Matt Wilsey has committed a decade of tireless labor and about $70 million to find a medicine for his daughter’s ultra-rare genetic condition. After a scientific odyssey, Wilsey is agonizingly close to realizing his goal. His next challenge — winning over a skeptical Food and Drug Administration — will reverberate throughout an entire field of medicine.
Wilsey’s daughter, Grace, has NGLY1 deficiency, a fatal disease that brings a cascade of profound developmental challenges. His life’s work has been fashioning a gene therapy that might allow her to live longer, a process that pulled in Nobel laureates, well-heeled investors, and families around the world affected by the disease. Ten children, including Grace, have been treated with the gene therapy. Wilsey believes it’s working.
The FDA might not approve it. As STAT’s Jason Mast and Matthew Herper write, Wilsey is asking for an extraordinary amount of flexibility from regulators, who typically demand more data on manufacturing and efficacy than he can currently provide. And, after tapping out his wealthy network, Wilsey can’t afford to generate more evidence.
What happens next could be pivotal for the rare disease community and the dozens, if not hundreds, of parents following in Wilsey’s footsteps and trying to develop treatments of their own. Read more.
Who’s going to run the FDA?
We could find out soon, as finalists for the job of FDA commissioner have been sent to the White House for final review. As STAT’s Daniel Payne reports, the short list includes Heidi Overton, a White House adviser; Jeffrey Vacirca, an oncologist and health system executive; and Stephen Ferrara, a health affairs official at the Defense Department.
Whoever gets the job will inherit an agency beset by turmoil following mass layoffs, policy flip-flops, and a revolving door of high-profile regulators under previous commissioner Marty Makary. The FDA, which regulates nearly a fifth of the nation’s economy, is currently led by top food regulator Kyle Diamantas on an acting basis. Read more.
Professional football players are nearly four times more likely to die from neurodegenerative disease than the average American, according to a study led by Mass General Brigham in Boston. In the largest study of its kind to date, researchers looked at data from nearly 20,000 former National Football League players, finding that the longer a player’s career, the more likely he was to die of dementia, Parkinson’s, or another form of neurodegeneration.
And the study, published Wednesday in eClinicalMedicine, may actually underrepresent the dangers of pro football, according to the researchers. Elite athletes have genetic, environmental, and behavioral characteristics associated with long lifespans and lower risk of brain disease, making the data from NFL players all the more dramatic.
RFK Jr. is making a list of Covid vaccine injuries
Federal authorities are compiling a list of injuries that can be tied to Covid-19 vaccines, a process that will make it easier for people to receive compensation through a federal program.
On its face, that’s a perfectly normal administrative action for the Department of Health and Human Services to take. But given health secretary Robert F. Kennedy Jr.’s long history of making unfounded claims about vaccine safety, experts told STAT’s Chelsea Cirruzzo they’re concerned his HHS will come up with an overly broad list of purported Covid-vaccine-related injuries to foster doubts about the shot. Read more.
The plot to cement control of American science
The first year of this Trump administration brought unprecedented upheaval to the world of science, with mass firings and sweeping funding cuts. The effect was destabilizing, researchers said, but many of those initiatives have since been overturned in court, and yet more could still be undone.
The next round of policy changes is likely to be more durable. As STAT’s Anil Oza reports, the administration is now going through a more protracted — and legally defensible process — to impose new rules on federally funded research, including changes that would expand the power of the executive branch to direct research, terminate grants at will, and ignore the guidance of external scientific reviews.
“What the Trump administration is trying to do is to set those priorities in stone, and they become part of the regulatory infrastructure going forward instead of temporal guidances,” said Andrew Twinamatsiko, director of the Center for Health Policy and the Law at Georgetown University. Read more.
‘MHFH’ doesn’t exactly roll off the tongue
In any case, it stands for “Make Hospital Food Healthier,” and it’s Kennedy’s latest effort to change the way America eats. The initiative is a voluntary pledge, inviting hospitals around the country “to lead by example by serving nutritious, minimally processed meals that help patients heal, reduce chronic disease, and help Make America Healthy Again,” Kennedy said in a statement Wednesday.
The word “voluntary” is important. Earlier this year, HHS sent notices to hospitals around the country urging them to adopt the recently revamped dietary guidelines “in order to enjoy continued eligibility for Medicare and Medicaid payments,” as Kennedy put it. The implied threat — that hospitals would lose federal funding if they don’t embrace the virtues of beef tallow — went well beyond the agency’s legal authority, experts said, and the softer language of Wednesday’s announcement seems to reflect that.
What we’re reading
- Outbreak of diarrhea-causing parasite grows to more than 1,000 cases, AP
- Bryan Johnson’s chronic disease is notoriously difficult to diagnose, STAT
- Patients face a thicket of red tape trying to maintain consistent health coverage, KFF Health News
- Opinion: Who benefits from classifying obesity as a disease? STAT