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The first base-editing trial in people, 'adult failure to thrive,' & more evidence on inaccurate pulse oximeters

 

 

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Verve launches trial of 'base editing,' a new gene therapy aimed at heart disease

This is news in more ways than one: Today Verve Therapeutics announced the start of a clinical trial testing a base editor, a newer way of utilizing CRISPR for gene editing, aimed at a different problem than rare disorders typically targeted by gene therapy. Someone in New Zealand has gotten the first dose of a therapy designed to be a one-time treatment for familial hypercholesterolemia, which affects about a million people in the U.S. alone and leads to high levels of “bad cholesterol,” increasing the risk of heart attacks and strokes.

Base editing uses CRISPR enzymes to make single letter changes to a gene without cutting both strands of DNA, in theory with less chance of off-target mutations. It's delivered with mRNA encased in a lipid nanoparticle, as are mRNA-based Covid vaccines. STAT’s Matthew Herper and Megan Molteni explain.

Study: Having darker skin meant less supplemental oxygen in ICU after faulty pulse oximeter readings

In further damning evidence against pulse oximeters, a new study shows patients with darker skin who received less accurate readings of their oxygen levels from the ubiquitous fingertip devices also received less supplemental oxygen during ICU stays. The new paper, out yesterday in JAMA Internal Medicine, follows research in BMJ last week suggesting that imperfect readings from pulse oximeters may hurt care of Black patients broadly, not merely those who are critically ill. Evidence is accumulating that faulty readings in darker-skinned patients may be one factor explaining racial disparities, such as higher rates of limb loss and death for Black and Hispanic ICU patients.

Both studies predate Covid, when using pulse oximeters to determine oxygen levels has played a critical role in deciding which Covid patients are admitted to the hospital and given supplemental oxygen and other therapies. STAT’s Usha Lee McFarling has more.

FDA is asked to approve first over-the-counter birth control pill

In a first, a pharmaceutical company is seeking permission to sell a birth control pill over the counter in the U.S. The French drugmaker HRA Pharma says the timing of its application was unrelated to the Supreme Court’s recent decision overturning Roe v. Wade, but the move sets up a high-stakes decision for health regulators amid legal and political battles over reproductive health. 

Since their introduction in the 1960s, hormone-based pills — long the most common form of birth control — have required a prescription, so health professionals can screen for conditions that raise the risk of rare, but dangerous, blood clots. HRA Pharma hopes to convince the FDA that women can screen themselves in order to use its version, sold as Opill. The pill contains a single synthetic hormone, progestin, which prevents pregnancy by blocking sperm from the cervix. Read more.

Closer look: Retiring 'adult failure to thrive' as a label

(Maria Fabrizio for STAT)

It's a phrase to wrinkle your eyebrows: “Adult failure to thrive,” the geriatric version of a term first used to describe children not growing well or missing their developmental milestones. Its form for elders is a diagnostic code that fits medical billing systems and, in the past, could secure hospice care. But the label, vague in its cloud of physical and cognitive symptoms, sometimes speaks to a bias in medicine that dismisses older people’s troubles as just normal aging, STAT’s Eric Boodman reports.

“When an older adult is called ‘failure to thrive,’ we see a lot of ageism come in, and we see the health care system start to ignore their problems,” Sharon Brangman, chair of geriatrics at the State University of New York Upstate, told Eric. “It’s used to totally disregard a person. … You lump everybody in one basket, and then you don’t take care of them.” Read more.

More people who inject drugs are getting treatment for hepatitis C

Hepatitis C infections can be cured, but getting people tested and treated remains challenging. The U.S. and WHO have set a 2030 target of reducing deaths by 65%, a goal hampered by low uptake of treatment among people who inject drugs. A new study in the Annals of Internal Medicine followed more than 1,300 people in a community program who were infected with HCV via drug use. It found that by 2019, 48% of participants had the disease, down from 100% in 2006.

“The critical question was not whether HCV treatment will work but whether it will be used in a community setting" of people who inject drugs, the authors write. “Lower mortality in persons who are cured might reflect not simply the effect of HCV cure but a constellation of social, environmental, and medical differences compared with those who are not cured.”

Intense efforts helped smokers quit before lung cancer screening, but only for a while

Quitting smoking is hard. Counseling and nicotine patches help, but for many people that doesn't work. A new study in JNCI compares intense with minimal counseling just before getting CT scans to detect lung cancer, screening that is recommended for people with 20 or more pack-years of smoking. Smokers are routinely offered help to quit before they get their scans.

For the study, researchers randomly assigned 818 people at eight medical centers across the U.S. to receive either intensive, more frequent counseling and nicotine patch shipments or standard counseling and patches before they were screened — including people who had no plans to quit. After three months, twice as many people in the intensive group had quit than in the standard group. The differences disappeared by six months, so “methods to maintain short-term effects are needed,” the authors write.

 

What to read around the web today

  • Hospitals must provide abortions in emergency situations, Biden administration says, Wall Street Journal
  • Abortion providers are trying to open new clinics as close as possible to states with bans, The 19th
  • 5 things to know about the brain-eating amoeba that infected a swimmer in Iowa, NPR
  • Why BA.5 feels different, The Atlantic
  • Opinion: FDA rule would facilitate prescription-to-OTC switches but nix a third class of drugs, STAT

Thanks for reading! More tomorrow,

@cooney_liz
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