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Good morning everyone. Having barely survived a weekday without a World Cup, we can finally get back to our new normal this afternoon with France 3-1 against Morocco.
A father’s quest to cure his daughter
Matt Wilsey has spent 10 years of tireless work and nearly $70 million trying to find a cure for his daughter’s ultra-rare genetic disease. Through his scientific adventures, Wilsey is painfully close to achieving his goal. His next challenge, convincing a skeptical Food and Drug Administration, will have implications for the entire field of medicine.
Wilsey’s daughter Grace has NGLY1 deficiency, a fatal disease that poses a series of severe developmental challenges. His life’s work was to develop a gene therapy that could extend her life, a process that attracted Nobel Prize winners, wealthy investors, and families around the world suffering from the disease. Ten children, including Grace, are undergoing gene therapy. Wilsey believes it’s working.
The FDA may not approve it. As STAT’s Jason Mast and Matthew Harper write, Willsey is asking for extraordinary flexibility from regulators, who typically require more data on manufacturing and efficacy than they can currently provide. And after leveraging his wealthy network, Willsey can’t afford to produce any further evidence.
What happens next could be crucial for the rare disease community and the dozens, if not hundreds, of parents who are following in Willsey’s footsteps and developing their own treatments. read more.
Who will run the FDA?
We may find out soon, as the finalists for the FDA commissioner job are sent to the White House for final review. As STAT’s Daniel Payne reports, the list of candidates also includes White House adviser Heidi Overton. Jeffrey Vasilka, oncologist and health system executive; and Pentagon Health Officer Stephen Ferrara.
Whoever wins the job will take over an agency hit by turmoil following mass layoffs, policy reversals and a high-profile revolving door of regulators under former Secretary Marty McCulley. The FDA, which regulates nearly one-fifth of the U.S. economy, is currently led by Kyle Diamantas, the top food regulator. read more.
Professional football players are nearly four times more likely to die from neurodegenerative diseases than the average American, according to a study led by Army Gen. Brigham of Boston. In the largest study of its kind to date, researchers looked at data from nearly 20,000 former National Football League players and found that the longer a player’s career was, the more likely they were to die from dementia, Parkinson’s disease, or another form of neurodegeneration.
And the study, published Wednesday in eClinicalMedicine, may actually underestimate the dangers of playing professional football, researchers say. The data from NFL players is even more dramatic because elite athletes have genetic, environmental, and behavioral characteristics that are associated with longer lifespans and lower risk of brain disease.
RFK Jr. on COVID-19 vaccine injury list
Federal officials are compiling a list of injuries that may be related to coronavirus vaccines, making it easier for people to receive compensation through federal programs.
On the face of it, this is a perfectly normal administrative action for the Department of Health and Human Services to take. But experts told STAT’s Chelsea Siluzzo that given Health Secretary Robert F. Kennedy Jr.’s long history of making unsubstantiated claims about vaccine safety, they fear that his Department of Health and Human Services will compile an overly broad list of injuries allegedly related to COVID-19 vaccines, fueling doubts about the shots. read more.
A conspiracy to solidify the dominance of American science
The first year of the Trump administration brought unprecedented upheaval to the world of science, including mass layoffs and drastic funding cuts. The effects have been destabilizing, researchers say, but many of these efforts have since been overturned in court, and many more may be reversed.
The next policy change is likely to be more permanent. As STAT’s Anil Oza reports, the administration is currently undergoing a longer and legally defensible process to impose new rules on federally funded research, including changes that expand the executive branch’s authority to direct research, terminate grants at will, and ignore the guidance of outside scientific review.
“What the Trump administration is trying to do is articulate these priorities, and they will not be temporary guidelines, but will be part of the regulatory infrastructure going forward,” said Andrew Tuinamatsko, director of Georgetown University’s Center for Health Policy and Law. read more.
“MHFH” is not something to roll your tongue at.
Either way, it stands for “Make Hospital Food Healthy,” and it’s Kennedy’s latest effort to change the way America eats. The initiative is a voluntary pledge that calls on hospitals across the country to “lead by example by providing nutritious, minimally processed meals that help patients heal, reduce chronic disease and make America healthy again,” Kennedy said in a statement Wednesday.
The word “voluntary” is important. Earlier this year, HHS sent a notice to hospitals across the country urging them to adopt recently revised dietary guidelines “in order to continue to receive Medicare and Medicaid payment eligibility,” as President Kennedy put it. The implicit threat of losing federal funding if hospitals don’t embrace the benefits of tallow goes far beyond the agency’s legal authority, and the soft language of Wednesday’s announcement appears to reflect that, experts said.
what we are reading
- Outbreak of diarrhea-causing parasite rises to more than 1,000, Associated Press
- Brian Johnson’s chronic illness is notoriously difficult to diagnose, STAT
- Patients face red tape trying to maintain consistent health coverage, KFF Health News
- Opinion: Who benefits from classifying obesity as a disease?Status
