Makary is officially out as FDA commissioner

President Trump yesterday confirmed that Marty Makary is resigning from his role leading the FDA. Kyle Diamantas, the top food regulator at the agency, will step in as acting commissioner.

The announcement comes after the Wall Street Journal and others last week reported that Trump planned to fire Makary, fueling speculation that it was only a matter of time before he would leave.

His tenure of a little over a year was tumultuous. It involved announcements of big initiatives — but also personnel drama, departures of long-time staff, and controversy over political pressure impeding the FDA’s scientific process.

politics

Cassidy's seat at risk as he wavers on health policies

SAUL LOEB / AFP via Getty Images

Bill Cassidy, the Louisiana senator leading the health committee, is facing a fierce primary fight, with President Trump and the Make American Healthy Again movement endorsing one of his rivals. The primary is Saturday.

Cassidy got to this position in large part because he's tried to play both sides: voting with his conscience as a doctor while also trying to support the president, his friends said.

As a result, some in his party accuse him of being a Republican in name only, while the health care establishment thinks he's betrayed his responsibility as a physician.

Read more from STAT's Chelsea Cirruzzo and Daniel Payne, who traveled to Louisiana for this deep dive.

rare disease

Gene therapy viruses linked to a boy's tumor

Earlier this year, Regenxbio reported that a boy who received its gene therapy for a rare neurodegenerative disease called Hurler Syndrome developed a brain tumor.

In a study published today, scientists linked the tumor to adeno-associated viruses (AAVs), the engineered viruses used most commonly in gene therapies.

It's the first report linking a tumor to these viruses, but the researchers cautioned the finding is not cause for alarm. The boy is doing well, and surgeons have excised the tumor.

Also, the risk of gene therapy driving cancer is likely quite low — over 6,000 patients have now received AAV gene therapy, with no other conclusively documented cases.

biotech

A dilemma in trying to make gene therapies safer

An ongoing concern with gene therapies is that patients could mount an immune response against the viruses used to deliver new genes into the body. To address the concern, researchers have increasingly been giving patients an immunosuppressant called sirolimus.

The dilemma, though, is that sirolimus may blunt the effectiveness of the therapy, one biotech has observed.

This morning, Encoded Therapeutics said that in a trial testing its gene therapy for Dravet syndrome, a severe form of genetic epilepsy, patients who were on sirolimus experienced a smaller decline in seizures than patients who were on steroids alone.