Tag: News Approved

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STAT+: Drug meant to make gene therapy safer may also make it less effective

BOSTON — A medicine increasingly used to mitigate the risk of deadly gene therapy side effects could also blunt the therapy’s effectiveness, a new study finds.

The trial, from the startup Encoded Therapeutics, tested a gene therapy for Dravet syndrome, a severe form of genetic epilepsy. A key concern in gene therapy trials is that patients could mount an immune response against the engineered viruses used to deliver new genes into the brain. 

In the study, Encoded assigned most of the 21 children in the trial to receive steroids, the most commonly used immune suppressant. A subset — including most of the patients on the highest dose level — were assigned to also receive sirolimus, also known as rapamycin, a drug historically given to prevent rejection in transplant recipients. 

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STAT+: Scientists link boy’s tumor to gene therapy viruses, in rare finding

When 13-month-old Adam’s stem cell transplant failed, doctors had two options: A second transplant, which they warned carried a 10% to 15% risk of death, or a new and untested gene therapy.

His parents, Mary Beth and Dave Brennan, had watched Adam endure the first transplant. At one point, he couldn’t breathe and nurses rushed into the room to keep him alive. So they went with gene therapy — and then watched Adam, born with a life-threatening rare disease, reach milestones, learn sign language, and teach himself to read. 

Then last year a routine scan revealed a golf-ball sized tumor on his brain.

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Opinion: What can ‘blue zones’ really teach us about aging?

In 2023, Netflix released a documentary titled “Live to 100: Secrets of the Blue Zones,” which looked at some small, geographically isolated places around the world in which, reportedly, residents regularly lived to 100 or beyond.

It was just the most recent installment to a longevity franchise that began roughly 25 years ago. But how real are “blue zones”? And has the concept’s commercialization drowned out its scientific value?

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Opinion: RFK Jr.’s antidepressant deprescribing push gets one thing right — and others dangerously wrong

I recently helped a woman in her early 60s taper off fluoxetine (often known by the brand name Prozac), which she had taken for over 35 years, followed by bupropion (aka Wellbutrin), which she had taken for over a decade.

But Kennedy’s initiative conflates that genuine clinical need with claims unsupported by evidence — and some that are actively dangerous. 

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