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Fighting for a rare disease treatment, Becerra's legacy, & making deals on JPM Day One

January 9, 2024
Reporter, Morning Rounds Writer
Good morning. Today we have a reminder that the story of gene therapy is not solely one of scientific advances but also one with roadblocks.

exclusive

He helped U.S. soldiers in Afghanistan, but Texas Medicaid won't pay for his son's gene therapygenetherapytexas_illo3

Illustration: STAT; Photo Courtesy Najeebullah Pashai

Three years ago, the Pashai family feared for their lives as they left Afghanistan. The father had worked security for an elite unit of American-trained Afghan special forces, so leaving home was their only choice once the Taliban took over. Now the family, grown to have three sons, faces danger again. Two boys have a rare inherited disorder — metachromatic leukodystrophy — and while there may still be time to cure the younger one with a gene therapy donated by its maker under a compassionate use program, the family can't afford it. 

Their new home state of Texas has said it won't cover the hospital stay and other associated costs of giving the child Libmeldy, made by Orchard Therapeutics. The reason? Libmeldy is not yet approved in the U.S. and the Texas Medicaid program does not pay for experimental therapies. "We are in great distress that we are going to lose another child," their father told STAT's Megan Molteni. Read more.


biopharma

JPM has begun. So have the deals.

The J.P. Morgan Healthcare Conference has kicked off in San Francisco in fine fashion. Or, as the locals say, "the lanyards" have landed in the city by the bay. 

  • The Mayo Clinic has signed Silicon Valley tech startup Cerebras as its first generative AI partner. Mayo will give Cerebras millions of dollars to help build technology tools based on its trove of de-identified patient data, STAT's Mohana Ravindranath reports.  
  • Johnson & Johnson said it would buy Ambrx Biopharma for nearly $2 billion, picking up a company specializing in targeted chemotherapy treatments — one of the hottest areas of cancer drug development, STAT's Andrew Joseph notes
  • Merck announced it would acquire Harpoon Therapeutics and its pipeline of immune-based cancer drugs for $680 million as Merck's top brass searches for ways to bolster the company's revenue after it loses exclusivity on Keytruda, STAT's Jason Mast tells us.
  • Eric Tokat, biotech's most prolific dealmaker, told STAT's Adam Feuersetein that 2024 will be a strong year for acquisitions. Last year, Tokat's advisory firm, Centerview Partners, was involved in 19 of the 23 biotech transactions valued at $1 billion or more. Those acquisitions of Harpoon Therapeutics and Ambrx Biopharma? Both are Centerview clients. Read more.

health care

Hospital at home proves its worth, researchers say

Cast your mind back to November 2020, when Medicare launched the Acute Hospital Care at Home waiver program to help hospitals overburdened by Covid-19 patients. Since then, thousands of patients from 300 hospitals in 37 states have been treated in their homes after the program was expanded through this year. A new analysis in Annals of Internal Medicine reports the results include low death rates (0.5%), less use of skilled nursing facilities (3%), and lower need for rehospitalization (6%) among the socially vulnerable and medically complex patients it followed from July 2022 through June 2023.

Many of the observational study's more than 5,100 patients already had conditions including heart failure (43%), chronic lung diseases like emphysema (43%), and dementia (16.%) before being discharged with such conditions as Covid, sepsis, kidney or urinary tract infections, and cellulitis. The findings held true across race and ethnicity, and whether they were enrolled in both Medicare and Medicaid. The authors say their results make the case for extending the hospital-at-home Medicare waiver.



closer look

What legacy will Becerra, once 'invisible,' leave? Xavier-Becerra-Where

Illustration: Christine Kao/STAT; Photos: Getty 

When you think of Xavier Becerra, what comes to mind? He's been called the "invisible" HHS secretary, rarely spotted at White House health care events. Once, officials openly discussed who might be better for the job. But with one year left in this Biden administration, it's unclear what legacy he'll leave. That's in contrast to his tenure as California attorney general, when he took a forceful stand on abortion rights, and as a member of Congress, when he helped shape and then defend the Affordable Care Act. 

Supporters say he has been quiet about his accomplishments at HHS. What does Becerra say? "I would like to see us make it so that everyone equates Obamacare, the Affordable Care Act marketplace, with good health care," he told STAT's Sarah Owermohle, so the "ACA becomes like Medicare, Medicaid, and Social Security. They are part of the fabric of life in protecting your health." Read more.


reproductive health

An already high cesarean rate in Puerto Rico climbsScreen Shot 2024-01-08 at 12.04.54 PM

National Center for Health Statistics 

Just over half of deliveries in Puerto Rico were C-sections in 2022, continuing its trend of consistently higher rates of the procedure compared to the rest of the U.S. A new CDC report looked back at the early to mid-1990s, when just over 30% of births in Puerto Rico were C-sections, but climbed above 40% in the early 2000s. Those rates were 40% to 50% higher than in the rest of the U.S., and up to 78% higher than for Hispanic women on the U.S. mainland. 

From 2019 to 2022, today's report says, cesareans in Puerto Rico increased each year until reaching 50.5% of all births in 2022. Rates rose for every age group, accounting for nearly two out of every three births to mothers 40 and older. The report offers no explanation, but back in 2006, another analysis speculated that "differences in maternal characteristics, attitudes toward cesarean delivery, obstetric practices, or health insurance coverage" might be among the factors at play.


health

Early exposure to digital media tied to sensory issues

Screen time during early childhood is tied to differences in sensory processing, a new study suggests, adding another drawback to digital media exposure for babies and toddlers. Authors of the JAMA Pediatrics study say they can't draw a direct cause-and-effect line between watching TV or videos and developmental problems, but sensory processing issues could join a list of other potential issues linked to early screen exposure including language delay, lower cognitive development, ADHD, autism spectrum disorder, behavioral problems, emotional dysregulation, and sleep problems.

For this study, researchers asked caregivers of more than 1,400 children how many hours of screen time the children watched and how they responded to stimuli at 12, 18, and 24 months. Real-life examples could be making noise with toys versus fleeing a loud environment, or being more absorbed in twirling an object than interacting with a person. Children with more screen time were more likely to have atypical sensory processing. Another caveat: the survey was done between 2011 to 2014, when the digital media landscape was different.


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What we're reading

  • The pandemic sent hunger soaring in Brazil. They're fighting back with school lunches, NPR
  • Opinion: Why our fear of cancer is outdated — and harmful, Washington Post
  • Lawmakers open probe into asthma inhaler makers over pricing and patent maneuvers, STAT
  • Just how healthy is salmon? New York Times
  • Florida's gambit to import drugs from Canada: 'a complicated road' ahead, STAT

Thanks for reading! More tomorrow,


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