Who has access to a sickle cell cure, a budget request for hepatitis C, & doctors who go to the 'dark side'

Victoria Gray is living proof of the power of CRISPR.

March 8, 2023 Check out STAT+

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By Elizabeth Cooney Reporter, Morning Rounds Writer

Good morning. Megan Molteni brings us a powerful story about an experimental gene therapy that can end the agony of sickle cell disease — but might be out of reach where it's needed most.

Health CRISPR cures for sickle cell may be coming, but who can access them? Courtesy Royal Society Victoria Gray (above) is living proof of the power of CRISPR. After a lifetime of sickle cell disease and its agonizing crises, she joined a clinical trial of a gene therapy created by CRISPR Therapeutics and Vertex Pharmaceuticals, one expected to become the first FDA-approved treatment of its kind. Now free of symptoms, able to be a mom and work a full-time job, Gray is able to "dream again without limitations," she told the audience at the Third International Summit on Human Genome Editing in London this week. The promise of a one-time cure offers hope to the millions of people living with sickle cell disease — a disorder long subject to scientific neglect and medical racism. But the majority of people with the disease live in sub-Saharan Africa, and face a number of barriers to accessing the new therapies. STAT's Megan Molteni has more. politics Collins hints that Biden budget request will seek funds to end hepatitis C The Biden administration will request "significant investments" to wipe out hepatitis C in its forthcoming budget proposal, former NIH director Francis Collins hinted at last night's STAT event in Washington, D.C. "I would like, if it's possible, to be as bold as possible, and contemplate not just an effort that would improve the situation, but that would eliminate hepatitis C in the United States," said Collins, currently a White House science adviser. "That's a scary word, and obviously would require a huge investment, to try to reach everybody." Rep. Hank Johnson, (D-Ga.), also at the event, said the request could reach $10 billion over five years. Collins has previously said such a plan could save $26 billion by 2050. And Sen. Bill Cassidy, a Republican from Louisiana said that if the proposal does indeed save money, it's worth pursuing. STAT's Nicholas Florko has more. chronic diseases Navigating Parkinson's: 'It was never a thing to say, Why me?' Photo illustration: Casey Shenery for STAT In 2020, Sandra Coplin traded life in Covid-ravaged New York City for retirement near family in Connecticut. She also swapped her four-inch-high pumps for "orthopedic-looking shoes" because in her last years as a legal records manager, she had begun navigating life with Parkinson's. STAT's Isabella Cueto asked her how her life has shifted since. Tell us about learning you had Parkinson's. ​​When I found out in 2017, it was never a thing to say, 'Why me?' because I felt I had been blessed in my life. What were the first signs? Fatigue. As I learned with Parkinson's, we tend to have an on-off time. I had a loss of appetite. And then the balance.  Do you have advice for others with a new diagnosis? Ask the questions. There's no question that's ridiculous. And if you're made to feel that way, I suggest getting another neurologist.  Read the full interview.

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Closer Look Why some doctors go over to the 'dark side' Anyone who's been on the wrong end of a prior authorization dispute can probably relate to last week's story on one patient's experience battling confounding insurance denials. Now STAT contributor and Mass General gastroenterologist Trisha Pasricha brings us the perspective of physician gatekeepers who make such decisions. It's easy to think of these figures as "shills for the insurance company," one doctor who made the switch told her, but there's more to the story. Some physicians jump to the "dark side" after burning out in their clinical jobs. Others are working mothers seeking better work-family balance. Once engaged in "utilization management," some doctors worry about a culture leaning toward denial or being asked to review cases outside their specialties. But one doctor who now works for an insurer argues that denials can actually protect patients. Read more about who's actually evaluating those faxed appeals for coverage. health FDA makes a rare comment on its move authorizing at-home flu and Covid test  Last week, the FDA issued an emergency authorization for the first at-home Covid-19 and flu combination test, days after its maker filed for bankruptcy. Now the FDA has released a rare comment on the lengthy authorization process that the company, Lucira, blamed for its bankruptcy. The statement fromJeff Shuren, director of the FDA's Center for Devices and Radiological Health, clarifies that the FDA ultimately authorized Lucira's test with fewer samples than the agency had previously said it would require, partly because of its partnership with the NIH Independent Test Assessment Program. The NIH program gave the FDA data that showed that even without more test samples, the company's test kits would be of the appropriate quality. While other home test company executives lauded the FDA's newfound flexibility as the right thing and a positive signal for subsequent at-home combination test authorizations, Nelson Patterson, president and CEO of at-home test manufacturer Anavasi, couldn't help but be a bit jealous. "Do I feel cheated?" Patterson told STAT. "I wish I had known that that would be the standard because I would have applied last summer."  reproductive health Battles continue over abortion and its restrictions  Legal battles centered on abortion and abortion access are playing out throughout the country: Five women in Texas are suing the state over its abortion ban, saying they were denied the procedure when their lives or the lives of their fetuses were in danger. The suit alleges that doctors are turning away pregnant women with serious complications because they fear legal penalties.  In California, Gov. Gavin Newsom said the state would no longer do business with Walgreens in response to the pharmacy chain's plans to stop dispensing abortion pills in 20 states. Walgreens now appears to have backtracked, saying in a recent statement it "plans to dispense Mifepristone in any jurisdiction where it is legally permissible to do so." Yesterday in Florida, which already bans abortion after 15 weeks, GOP lawmakers proposed banning the procedure after six weeks of pregnancy, except to save a patient's life or after rape or incest — if the patient can provide proof of those crimes. The new proposed ban is backed by Florida governor Ron DeSantis. by the numbers

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What we're reading Biden proposes expanding Medicare drug negotiation program, STAT Vulnerable people in U.K. to be offered spring Covid booster vaccine, The Guardian No, female mice are not more erratic, New York Times The company offering free health care to East Palestine? It's a right-wing, anti-vax project, Mother Jones She didn't need the risky surgery. County doctors talked her into it anyway, lawsuit says, Los Angeles Times Controversial drug for premature births is withdrawn by manufacturer after an unusual battle with FDA, STAT

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Thanks for reading! More tomorrow,

elizabeth.cooney@statnews.com

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