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Gene therapy returns hearing to two deaf children

May 08, 2024
May 8, 2024
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Morning Rounds Writer and Podcast Producer
Do you like coconut cake? How about coconut flakes? This was the big debate at STAT headquarters yesterday after someone brought in their own version of the Tom Cruise cake. News is perhaps the only thing we take more seriously than food around here, so read on for that.

disability

Two deaf children can hear after an experimental gene therapy treatment

ADOBE

Two congenitally deaf children can hear for the first time after being treated with gene therapy, according to data presented at a conference Wednesday. Researchers made a small incision behind an ear on each child, snaked a catheter through, and injected the therapeutic gene — ferried along by a viral vector — into the inner ear. The results were "jaw-droppingly good," as one investigator on the study put it. (The researchers had provided the gene therapy in one ear and a cochlear implant in the other, just in case.)

The work is part of a larger study funded by Regeneron that will test the treatment in nearly two dozen children with a rare form of hearing loss caused by variations in the otoferlin gene. In the last year, scientists have successfully enabled hearing in several children with this type of deafness — the culmination of decades of work. And researchers are already making plans to target other genes that affect more deaf people. Read more on the state of the research from STAT's Timmy Broderick.

health tech

Apple Watch gets a nod from the FDA for clinical trials

Since 2022, millions of Apple Watch wearers have been able to look at their history of atrial fibrillation, an irregular heart rhythm that can cause shortness of breath and fatigue. Now, the FDA has qualified that feature to be used in clinical trials — potentially making the smartwatch an appealing tool for medical device companies testing a common heart procedure.

The qualification allows companies to use the history feature, which estimates a user's time spent in atrial fibrillation, as a secondary endpoint in clinical trials of cardiac ablation devices intended to reduce the burden of AFib. The market for clinical trials is limited compared to Apple's reach as a consumer technology company. But the qualification helps build the case that the Apple Watch should be taken seriously in health care. Read more from STAT's Mario Aguilar and Lizzy Lawrence.

cancer

Vulnerable U.S. counties get fewer cancer clinical trials

Experts have long pushed to increase diversity in clinical trials as a way to combat health disparities entrenched in cancer care. But there's still lots of room for improvement, as a new study shows. Researchers analyzed national cancer clinical trial data and social vulnerability index scores for over 3,100 U.S. counties between 2007 and 2022. They found that the most vulnerable counties (measured using socioeconomic and demographic factors, along with housing and transportation data) were less likely than others to host any clinical trials, and on average had fewer trials in proportion to their population size.

The study, published yesterday in JAMA Network Open, did not track patient enrollment in trials, a more direct measure of access. Still, the authors believe the work demonstrates a need to focus on the social determinants of health and increasing access in vulnerable communities. 

first opinion

How easy is it to buy synthetic DNA fragments to recreate a deadly flu virus?

Too easy, according to Kevin M. Esvelt, a professor at the MIT Media Lab. And he would know. In a new First Opinion essay, Esvelt writes about an experiment that two Ph.D. students in his lab performed, purchasing DNA fragments that could be used to generate the 1918 flu virus that killed more than 50 million people. They placed orders using the name of a company that doesn't perform lab experiments, and provided shipping addresses that clearly weren't laboratories. 

Despite these red flags, 36 out of 38 vendors for the DNA shipped them the fragments. It's not a problem with those companies, Esvelt said, but rather a concerning lack of government-mandated security. Read more on the experiment and what we should learn from it.

events

STAT on the road: Dispatches from the Milken Institute Global Conference

The Milken Institute held its 27th annual conference in Los Angeles this week, an event that draws some of the richest and most powerful people in the world. STAT's Nick St. Fleur, who was there moderating panels and reporting, told me that in his first two days, he walked by "Jane the Virgin" actor Justin Baldoni and billionaire Byron Allen, saw Kellyanne Conway and Reed Jobs in a greenroom, and stood next to Marc Tessier-Lavigne in an overflow room during an Elon Musk interview. But it's not a space where people fawn over celebrities, Nick emphasized. Big names simply blend into the sea of navy and gray suits — that is, until they get up on stage to speak. Catch up on the news from the conference:

  • CDC director Mandy Cohen spoke about public health preparedness, including the critical role of data collection. But she didn't speak about the CDC's role in monitoring the ongoing spread of H5N1 bird flu in dairy cows, or the slow release of sequencing data on the virus. Read more from STAT's Nalis Merelli.
  • Artificial intelligence was top of mind at the conference, Nick said, including at a panel on health care applications moderated by STAT executive editor Rick Berke. "We've sort of anthropomorphized AI and said it has to be a thing that's going to replace a human," said MD Anderson Cancer Center chief data officer Caroline Chung. "Why are we not reenvisioning that it's something that can assist a human?" Read more from STAT's Brittany Trang.
  • Biomedical experts spoke about the long, expensive process of drug development and how to improve it, including new ways of identifying and testing therapies, a more precise understanding of disease, and a renewed sense of urgency to address longstanding public health issues. Read more from STAT's Jonathan Wosen. 
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What we're reading

  • A Kansas Republican voted for a gender-affirming care ban. But then she flipped, The 19th

  • Young boy dies in trial for Pfizer Duchenne gene therapy, STAT
  • A mother's loss launches a global effort to fight antibiotic resistance, Los Angeles Times
  • House panel eyes PBM reform to pay for telehealth extensions, STAT

Thanks for reading! More tomorrow,

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