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World's first CRISPR-based therapy wins approval in U.K. 

November 16, 2023
Reporter, Morning Rounds Writer
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biotech

World's first CRISPR-based medicine wins approval

This morning, regulators in the U.K. gave the greenlight to a CRISPR-based medicine for both sickle cell disease and beta thalassemia, making it the world's first treatment built on the revolutionary gene-editing technology to reach the market. As STAT's Andrew Joseph reports from London, the approval is not a surprise, given that clinical trials showed the one-time therapy enabled many sickle-cell patients to live free of debilitating pain crises and freed thalassemia patients from needing regular blood transfusions. But it is still a major step in the progress of genetic medicine. 

The medicine, which will be marketed as Casgevy and was developed by Vertex Pharmaceuticals and CRISPR Therapeutics, is also under review at the FDA and in the European Union. But with the approval comes the next challenge of getting it to patients. The companies will have to reach pricing and reimbursement deals for a therapy that is expected to carry a list price in the millions of dollars. Receiving the therapy is also an ordeal for patients, who have to undergo chemotherapy and spend weeks in the hospital. 

More here on the historic approval.


biotech

After four children with a rare disease died in its gene therapy trial, Astellas sees a way forward

The disorder is rare and deadly. X-linked myotubular myopathy affects roughly one in 40,000 to 50,000 newborns, who typically reach few motor milestones and require breathing support to survive. Only about half live to age 2. A clinical trial testing a gene therapy from Japanese pharmaceutical company Astellas was halted after four of 24 children died in 2020 and 2021. 

Astellas' new analysis of that trial, published yesterday in Lancet Neurology, concludes there is enough promise to go forward with the therapy. "The larger question is how do you balance the potential power of a therapy with the potential for major toxicity?" said Lindsey George, a hematologist at the Children's Hospital of Philadelphia who was not involved in the study. "I think it creates ethical questions, but also the question of how you chart this unknown territory in a way that advocates for patients but ensures an appropriate level of safety." STAT's Damian Garde has more.


politics

House moves to restrict gain-of-function research

If House lawmakers have their way, infectious disease studies known as gain-of-function research will be banned in the U.S. Attached to legislation that funds the NIH and other federal agencies, the measure would bar paying for research in which a pathogen is altered to study its spread, potentially making it more transmissible or severe. The spending bill already restricts funding this research in any "foreign adversary," including China. Scientists at the Wuhan Institute of Virology studied gain-of-function in some viruses, fueling theories — never definitively proven — that the Covid-19 pathogen was a lab leak. 

The amendment to the health department budget came by voice vote, and it's not clear when the Senate might take it up. Republican senators including Kentucky's Rand Paul have railed against the field and interrogated NIH officials such as former NIAID chief Anthony Fauci about its usefulness in understanding potential pandemic diseases. STAT's Sarah Owermohle has more on what may come next.



closer look

'We take care of our people': How a Wisconsin tribe models harm reduction amid the opioid crisis 

BRHR072Jaida Grey Eagle for STAT 

In the opening lines of Lev Facher's story about how the Bad River Tribe in Wisconsin is facing its opioid crisis, the scene is cold and bleak. A small tent city, thawing out from the first snow, is home to people left homeless by addiction to fentanyl and methamphetamine. But the script quickly flips to hope: Workers are revamping a modest shack into sturdier shelter.

"We take care of our people," said Lisa Whitebird (above), a coordinator of a specialized harm-reduction team. "It's not like they woke up one day and decided to be homeless, and a drug addict, and live in tents on the pow-wow grounds." That means instead of pushing immediate abstinence, the team offers sterile syringes and pipes. They distribute naloxone, the overdose-reversal medication, and encourage their clients to use test strips that can detect fentanyl or "tranq." For the moment, they have only one goal: To keep their neighbors alive. Read more.


infectious disease

FDA authorizes at-home tests for two STIs

In a first, the FDA has granted marketing approval to an over-the-counter home test for chlamydia and gonorrhea, the two most common sexually transmitted infections in the U.S. The LetsGetChecked's Simple 2 Test allows people to collect a sample at home that then goes to a laboratory for processing. Results are delivered online, with follow-up care provided when a diagnosis is positive or ambiguous. Before yesterday's authorization, the only approved tests for these two STIs required samples to be collected at medical facilities such as doctors' offices. 

Jodie Dionne of the University of Alabama at Birmingham welcomed the news. "If we are going to do a better job of reaching more sexually active people for STI testing as recommended … we need to be creative about how to get them tested and treated in a way that is highly effective and works for them," she told STAT's Helen Branswell. Read more.


health care

Forgoing medical care because of cost is a distinctly American thing

We know people with low or even average income in the U.S. may struggle to pay their medical bills compared to people of similar means who live in other wealthy countries. But a new Commonwealth Fund analysis says that pain extends to some high-income Americans, too, who are more likely to report health care cost challenges than people in other countries with low and average incomes. Looking at Australia, Canada, France, Germany, the Netherlands, New Zealand, Switzerland, the United Kingdom, and the United States, the researchers found income disparities in most countries (the least in German and the Netherlands), but not as wide as in the U.S.

Other points:

  • Nearly half of U.S. adults had a medical bill problem in the past year.
  • U.S. adults with lower or average income were more likely to skip mental health care because of cost compared to higher-income Americans.

Correction: Yesterday I gave you a faulty link to Anika Nyak's story on a pilot program testing food as medicine in the form of fresh produce. It's here.


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What we're reading

  • Exclusive: FDA's latest reorganization puts a new emphasis on its investigations, STAT
  • FTC cracks down on food industry for paid dietitian 'influencer' posts, Washington Post
  • V.A. recruits millionth veteran for its genetic research database, New York Times (Gotta call back this 2015 gem from the STAT archives: Inside the drive to collect DNA from 1 million veterans and revolutionize medicine)
  • Mike Johnson's resume includes being a board member of a Christian publishing house that called 'monkeypox' a penalty for being gay, Politico
  • To get things done fast at FDA: 'Don't tell anybody,' cancer chief says, STAT
  • Type 2 diabetes prevention programs can work at large scale, study finds, STAT

Thanks for reading! More tomorrow,


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