The first known attempt to prevent ALS

Jeff Vierstra takes a selfie on a mountain, with his hood up, a baseball hat on, and a scarf wrapped around his ears.

Courtesy Jeff Vierstra

When Jeff Vierstra (above) signed up for Columbia University’s ALS Families Project, he thought he would be participating as a carrier of a gene mutation that causes the devastating disease, not as a patient himself. His mother died from ALS when she was 32 years old, and decades later, only his sisters had begun to show early symptoms.

But when the doctor did an initial exam to establish a baseline for Jeff’s nerve function, he didn’t like what he saw. Jeff didn’t have ALS yet, but there were abnormalities. The doctor wanted to put him on the same experimental drug that his sisters would take — as a preventive measure. If Jeff was truly about to develop ALS, the drug might save his life. But if the doctor was wrong, it could do irreparable harm. Read more from STAT’s Jason Mast about the story that some advocates see as a case study in possibility.

public health

AAP emphasizes vaccines after devastating flu season

The flu death toll among kids during the 2024-2025 season has reached an astonishing 266, the worst single season total — except during the 2009 flu pandemic — in the 21 years that the CDC has recorded pediatric flu deaths. Five new deaths were added on Friday, and it’s likely that number could rise further before the 2024-2025 season officially closes at the end of September.

Though the vaccine advisers who guide the CDC’s vaccination policy voted in June to reiterate U.S. policy calling for everyone 6 months and older to get an annual flu shot, health secretary Robert F. Kennedy Jr. is still reviewing that recommendation. In the meantime, the American Academy of Pediatrics is urging parents to vaccinate their kids this fall. “Immunized kids are healthier kids who can focus on growing, playing, and learning,” Kristina Bryant, a member of the AAP’s infectious diseases committee, said in a statement. Flu vaccination rates among both children and adults have dropped in the wake of the Covid-19 pandemic. — Helen Branswell

from AXIOS:

GLP-1s could become the “everything drug”
By Adriel Bettelheim and Maya Goldman
Illustration of the Rx prescription symbol with a magic wand and sparkle trail on top of it
Illustration: Natalie Peeples/Axios
The biggest buzz around GLP-1 drugs these days has nothing to do with weight loss. And that might lead to some problems for patients and insurers.

The big picture: Blockbuster treatments like Ozempic have been found to lower the risk of everything from Alzheimer’s and addiction, to sleep apnea, seizures and bacterial infections. More potential uses keep surfacing.

  • It’s still not clear whether they’re a cure-all, or whether the benefits come from the fact that obesity and diabetes give rise to so many other health problems.

State of play: The injectable drugs’ potential beyond weight loss was driven home early this year by a study of almost 2 million Veterans Health Administration patient records that found GLP-1s lowered the likelihood of dozens of health conditions.

  • Researchers found the benefits were modest — about a 10% to 20% reduction in most cases — but noted that could be meaningful, especially for conditions like dementia that have few effective treatments.

Friction point: The FDA has only approved GLP-1s for obesity, Type 2 diabetes, heart risk and, under certain circumstances, sleep apnea. That means using them for any other condition is “off label” and at a physician’s discretion.

  • The designation can affect whether insurers pay for the treatments. That’s no small matter when the monthly cost of brand-name options like Wegovy and Zepbound range from $1,000 to $1,350 without coverage.
  • The hype around the drugs has sent many Americans to telemedicine companies that sell brand-name or knockoff GLP-1s. One recent study found that nearly 40% of GLP-1s approved by the FDA for diabetes are being prescribed off-label.

More here

Full-body MRIs offer peace of mind and pitfalls
By Carly Mallenbaum
Illustration of an x-rayed figure giving a thumbs-up.
Illustration: Allie Carl/Axios
Depending on whom you ask, a full-body MRI is either an early-detection breakthrough or an unnecessary procedure that harms more than helps.

Why it matters: Scans from companies like Prenuvo and Ezra have become a popular health splurge for adults focused on longevity.

  • That’s largely thanks to wellness influencers, celebrity endorsements and a distrust of the conventional medical system.

By the numbers: Prenuvo has completed 50,000 scans since December — and 150,000 total since 2018, CEO Andrew Lacy tells Axios.

What they found: Prenuvo scans spotted cancer in 2.2% of mostly asymptomatic patients, according to an ongoing study conducted by the MRI company and presented at an American Association for Cancer Research conference in April.

  • Roughly half of the biopsies prompted by scan findings turned out to reveal cancer.

Between the lines: Just because a scan detected cancer, that doesn’t mean the cancer was aggressive or that the detection extended someone’s lifespan.

  • In the study, two breast cancer cases were not detected by a whole-body MRI.

What we’re hearing: Full-body scans are “the bane of my existence,” says oncologist Marleen Meyers, director of NYU Langone’s survivorship program at the Perlmutter Cancer Center.

  • She says that most findings from full-body MRIs are false positives or benign, but “the knowledge, the stress and fact you start treatment then upends your life.”
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