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First in-utero cerebrovascular surgery, RSV vaccine approval, & do we still need Covid and mpox state of emergencies?

May 4, 2023
Reporter, Morning Rounds Writer
Good morning. We have two firsts to tell you about: the first surgery to repair a serious cerebrovascular problem before birth and the first FDA-approved RSV vaccine.

Health

In a first, surgeons repair a deadly vascular malformation in the brain before birth

It's called in-utero cerebrovascular surgery and a team deployed it to prevent an aggressive vascular malformation in an infant's brain before birth. The first-ever case, described in Stroke, is part of a clinical trial to treat vein of Galen malformation. The rare condition, in which misshapen arteries in the brain connect directly to veins instead of capillaries, can cause widespread harm and death. By the time newborns are treated, in many cases brain damage has already occurred.

A team at Brigham and Women's Hospital and Boston Children's Hospital used ultrasound-guided transuterine embolization to repair the malformation. The baby was born without the signs of trouble usually seen right away and now 6 weeks old, she is eating normally, gaining weight, and back home. "Just to see her look fine, really on no medication and not in a breathing tube or anything like that, that was really fantastic," Darren Orbach, pediatric neurointerventionalist at Children's, told STAT's Ambar Castillo. Read more.


infectious disease

FDA approves first RSV vaccine 

After decades of research, the FDA has approved the first vaccine for adults against respiratory syncytial virus, or RSV, a disease whose typical seasonality was bumped into public awareness by the pandemic, calling attention to an illness most harmful to the very young and the old. GSK's Arexvy was approved for adults ages 60 and older. It joins a crowded field of competitors, including a vaccine developed by Pfizer for adults expected to be approved by the end of the month. 

The vaccines must be recommended by the CDC, which appears likely to happen after its expert panel meets on June 21 and 22. Another Pfizer vaccine against RSV — this one for infants in their first 6 months of life is up for approval later this year, as is an antibody treatment from Sanofi for children under 1. STAT's Helen Branswell has more, including the scientific challenges developers had to overcome.


health

Two studies prompt second thoughts on beta blockers for heart patients

Two new studies challenge the standard wisdom that patients with heart conditions should take beta blockers to prevent further problems, especially if they have strong heart function. One study, posted Tuesday in Heart, followed people who'd had a heart attack but didn't develop heart failure or other pumping problems. For them, long-term beta blocker use wasn't associated with better cardiovascular outcomes.

The other study, out yesterday in JACC: Heart Failure, focused on people with heart failure who had mildly reduced or normal ejection fraction (a measure of a heart's squeezing power). It turned out that beta blockers were linked to a greater risk of hospitalization in patients with higher squeezing function. The data are "all pointing to a similar direction that not all patients that we used to historically reflexively put on beta blockers benefit from beta blockers," Lakshmi Sridharan of Emory University told STAT's Elaine Chen. Read more.



Closer Look

Covid and mpox aren't gone, but WHO's emergencies may soon be

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Anja Niedringhaus/AP

Is it time to flip the emergency switch to "off" for Covid and mpox? Public health experts told STAT's Helen Branswell yes, while suggesting such a black-and-white scenario doesn't capture the nuance of what it means to respond to such serious, sudden, unusual, or unexpected disease events. The WHO will likely declare an end to the two emergencies in the next week or so when an independent panel meets for the 15th time to consider Covid. The mpox emergency committee will meet Wednesday.

"I think that's the right thing to do and the wise course of action. All emergencies have to come to an end," said Lawrence Gostin, director of the WHO Collaborating Center on National and Global Health Law. The Covid public health emergency has been in effect since Jan. 30, 2020, and for mpox, since July 23, 2022. Read more on what's involved.


cancer

Endocrine therapy after breast cancer can be safely paused for pregnancy, study says

Women diagnosed with hormone-positive early breast cancer face another hurdle after they complete treatment. Years of endocrine therapy can follow, designed to cut their risk of the cancer coming back but also ruling out pregnancy. "It's both an emotional and kind of a physical loss for these women if they're told, 'Put your lives on hold and you know, by the time you get there, both with the chemo and time, you may not be a candidate for actually getting pregnant,'" Ann Partridge, vice chair of medical oncology at Dana-Farber Cancer Institute, told me.

Partridge is the lead author of a study in NEJM yesterday that says there's another option. Women with this form of breast cancer who interrupted their endocrine therapy to become pregnant had about the same risk of cancer recurrence as similar breast cancer patients who didn't pause their treatment, the study found. I have more here.


STAT breakthrough summit

Reflecting on firsts and making connections

On day one of STAT's Breakthrough Summit in San Francisco, we heard from the first child (now 18) to receive CAR-T cell therapy, the scientist leading that research, the developer of the latest Alzheimer's drug, and researchers studying how viruses might be implicated in Alzheimer's. A sample:

  • On donanemab, Eli Lilly's amyloid-targeting therapy, which met its goals in a pivotal trial of slowing the cognitive decline of Alzheimer's relative to placebo in a study that will likely lead to FDA approval: "I've been pursuing the same enemy for 25 years," Daniel Skovronsky, head of research at Lilly, said. Read more.
  • On being the first child to receive CAR-T cell therapy, at 6 years old: "It feels a little bit like a double life, because I'll go into these different conferences and I'll speak," Emily Whitehead, now 18, said. "And then the other time I'm in high school at my hometown." Read more.
  • On what's ahead for CAR-T: "For all blood cancers, it's pretty much an engineering problem now. We need to find the right targets," said Carl June, who led the Penn trial of the CAR-T Emily Whitehead got and stuck around for a panel on what's ahead. "It's going to be solved, but it's not going to be as fast as we thought back in 2010." Read more.
  • On the connections between latent herpes simplex virus and Alzheimer's: Ruth Itzhaki of the University of Manchester said, "I think it's important people realize viruses and bacteria can cause chronic disease and not just acute infection." Read more.

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What we're reading

  • Gene therapy has helped boys like Conner walk. Scientists are trying to keep it that way, STAT
  • WHO dismisses lead Covid origins investigator for sexual misconduct, Reuters
  • DEA will delay telemedicine restrictions for buprenorphine, Adderall, and other drugs, STAT
  • Saving Antonio: Can a renowned hospital keep a boy from being shot again? Washington Post
  • New Eli Lilly Alzheimer's data poses Medicare coverage conundrum, STAT

Thanks for reading! More tomorrow,


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