Evaluating a CRISPR-based sickle cell therapy, reducing health care worker burnout, & a second death after a pig heart transplant

"He knew his time with us was short, and this was his last chance to do for others."

November 1, 2023 Check out STAT+

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By Elizabeth Cooney Reporter, Morning Rounds Writer

Good morning. It's called xenotransplantation, but the word doesn't capture the human behind it. "He knew his time with us was short, and this was his last chance to do for others," Ann Faucette said in a statement about her husband, Lawrence, the second person to receive genetically altered pig heart. "He never imagined he would survive as long as he did, or provide as much data to the xenotransplant program."

genome editing FDA weighs how to assess safety in trial of CRISPR therapy for sickle cell Adobe This FDA panel's hearing was different. Not set up to conclude with a decision, the meeting of outside experts convened to review exa-cel, a CRISPR-based treatment for sickle cell disease made by Vertex Pharmaceuticals and CRISPR Therapeutics. None of the outside experts explicitly called for delaying approval to better assess the risk of off-target gene editing, STAT's Adam Feuerstein noted in the live blog he and Megan Molteni did all day. But there was some pointed criticism. Stanford's Joseph Wu said Vertex should be doing more genomic testing of samples taken from people treated in its clinical trial. "They have the samples," he said, "so I don't understand the hesitation of not doing it." Alexis Komor of the University of California, San Diego, pushed back. "Given the benefits of this treatment and what patients are dealing with without treatment, those far outweigh the risks here," she said. Read more on the day's back and forth, including patient perspectives. xenotransplantation Second patient who received a genetically modified pig heart has died Nearly six weeks after becoming the second person to receive the heart of a genetically engineered pig, a 58-year-old Navy veteran and former vaccine researcher at the National Institutes of Health has died. Lawrence Faucette was too sick for a human heart transplant, but in September doctors at the University of Maryland Medical Center performed the experimental surgery for the second time. Their first patient, David Bennett, survived for two months. Faucette began showing signs of rejection recently, and he died on Monday. "He knew his time with us was short, and this was his last chance to do for others," his wife, Ann Faucette, said in a statement. "He never imagined he would survive as long as he did, or provide as much data to the xenotransplant program."  STAT's Megan Molteni has explained how pigs are genetically engineered to make their organs better suit humans and STAT's Debbie Balthazar has more on Faucette and his death. health care New initiative to reduce health care workers' burnout puts the onus on management, not workers All too often, steps to ease the burnout health care workers seem to start and end with variations of the advice to "take care of yourself." Instead, a new anti-burnout campaign from the CDC and the National Institute for Occupational Safety and Health turns to leaders of the workplace, not the workers, for solutions. The campaign tools include a worker well-being questionnaire, a guide encouraging leaders to share their own struggles with mental health to help encourage staff to do the same, and online workshops on such topics as how to support work-life balance, veterans, and sleep. It's not enough to just offer better sleep strategies, Laura Linnan of the Carolina Center for Healthy Work Design and Worker Well-being said. "We have to look at, 'What are the schedules that we're putting these workers under? What are the number of hours that we're requiring?'"  STAT's Brittany Trang has more on the challenges of fixing burnout.

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closer look How fugitive fetal cells may affect maternal health STAT Chimeras may be the stuff of myths, from fantastical creatures in ancient Greece to classical Japanese tales. But the complex combination of different beings can also be found in human biology. Kristine Chua, a postdoctoral researcher in biology at the University of California Santa Barbara and a STAT's 2023 Wunderkind, studies one form  — microchimerism — that occurs when cells are exchanged between mother and fetus during pregnancy.  Scientists have long suspected that cells from developing offspring can escape the uterus and travel through the bloodstream into mothers' bodies, but it's only recently that they've studied how these fetal cells may affect maternal health. "There's an opportunity to ask questions that haven't been answered or to open up spaces for other folks to evolve our thinking," she said. Among the researchers developing more sophisticated tools for studying microchimerism, Chua is also advancing the practice of biological anthropology, a science that has struggled with diversity and ethical approaches. STAT contributor Justin Chen has more. health tech In a race of AI protein-folding models, it's DeepMind's turn to tout AlphaFold's new skills  In something like a horse race, Google DeepMind and Isomorphic Labs issued a significant progress update yesterday on AlphaFold, the deep learning model that in 2020 cracked the problem of predicting a protein's structure from its amino acid sequence. At the time, that advance moved it far ahead of other approaches prized in drug development. Here's what's new this time: Their model can predict not just standalone protein structures, but also what proteins look like in combination with several classes of molecules, including small molecules and nucleic acids. Three weeks ago, the University of Washington Institute for Protein Design published its own update to its protein-folding model, RoseTTAFold. In a preprint posted to bioRxiv, the group — which in 2021 paced AlphaFold2 with closely-timed releases of code and results — demonstrated similar abilities to model interactions between proteins and a range of molecules. STAT's Katie Palmer and Brittany Trang track the rivals' progress. soundings  Trust in vaccines dips, belief in misinformation rises  Annenberg Public Policy Center Americans' confidence in vaccines is falling while their belief in health misinformation is growing, according to a new poll out today from the Annenberg Public Policy Center of the University of Pennsylvania. The latest survey, conducted early last month, found the percentage who believe vaccines work to prevent a variety of illnesses slid from 77% in April 2021 to 71%. Over that same timeframe, the percentage who do not think FDA-approved vaccines are safe rose from 9% to 16%. Under two-thirds of adults think getting vaccinated against Covid-19 is safer than getting the disease, down from three-quarters. (The margin of error is 3.4 percentage points.) Other survey findings: ​​26% think ivermectin is a safe Covid treatment, up from 10% 16% believe "increased vaccines are why so many kids have autism these days," up from 10%  12% believe"vaccines in general are full of toxins and harmful ingredients like 'antifreeze,'" up from 8% On this week's episode of the "First Opinion Podcast," First Opinion Editor Torie Bosch talks with Brown's Michael Bernstein about the placebo effect (taken phenylephrine for a stuffy nose lately?) and its counterpart, the "nocebo effect" — if you tell patients something will make them feel worse, it generally comes true. Listen here.

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What we're reading Is this poop-test startup just wellness theater? Bloomberg The viral threat almost no one is thinking about, The Atlantic Medicare is urged to consider Amgen patent maneuvers when negotiating the price for a drug, STAT Pregnant farmworkers in California are eligible for paid time off — but many don't know it exists, The 19th Life science investors close nearly $6 billion in new funds, STAT

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Thanks for reading! More tomorrow,

elizabeth.cooney@statnews.com

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