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Good morning, I’ve got a packed, stacked newsletter for you today. Highlights include a touching feature from my colleague Jason Mast and a long-awaited update to a 2017 STAT story that editor Gideon Gil flagged to me.
A desperate dad becomes a beacon in the rare-disease world
Terry Pirovolakis isn’t a scientist or a doctor. He isn’t wealthy. He’s a 45-year-old IT professional, with three children and a fondness for monochrome T-shirts. He has also, somehow, become the first stop and last hope for many families who have watched for-profit drugmakers abandon the rare diseases that affect their children.
In the last two decades, researchers have made huge strides in their ability to diagnose one-in-a-million mutations and devise treatments with gene therapy, CRISPR or RNA-based medicines. But over the last few years, companies have shelved or deprioritized over 50 gene therapies, leaving the families who had been counting on them adrift.
Pirovolakis’ first goal was just to save his son, Michael, who was diagnosed with hereditary spastic paraplegia-50 at the age of one. He worked with academics, toxicologists, and drug manufacturers to get Michael treated in under three years. He then became an informal advisor to parents struggling to get their own kids treated. Then, he formed his own biotech company.
STAT’s Jason Mast has a beautiful feature on Pirovolakis and one family he’s helped, the Lockards, pictured above. As Jason writes in the story, there are a couple ways to look at it. You could see Pirovolakis as a superhero, a Tony Stark of biology. Or, Jason says, it’s another tragic story — a child selling lemonade to raise money for his own cancer treatment.
Anger toward UHC reflects people’s ‘pent-up pain’
The targeted killing of UnitedHealthcare CEO Brian Thompson has become a defining moment in the zeitgeist of American health care. You’ve probably seen some of the posts online, ranging from mournful to apathetic to joyful, including morbid celebrations of Thompson’s death. “I think a lot of people have pent-up pain, and they haven’t had a place to put it,” said bioethicist Yolonda Wilson.
The public’s dissatisfaction has never been higher. A survey released Friday reveals that the public’s rating of the health care system is its lowest point since 2001. STAT’s Bob Herman and Tara Bannow wrote about the factors within the health care system that contribute to that pain and resentment. Read the excellent piece.
USDA makes milk testing mandatory
The USDA announced on Friday that it’s instituting a mandatory milk testing program that should provide a much clearer picture of how widespread the bird flu virus is in the country’s dairy industry. The program will begin with six states, California, Colorado, Michigan, Mississippi, Oregon, and Pennsylvania. The agency did not say in its statement whether the information will be released to the public, or how frequently if it is to be released.
Read more from STAT’s Helen Branswell.
An ‘addict broker’ pleads guilty, 7 years later
Seven years after STAT first wrote about him, an “addict broker” named Daniel Cleggett has pled guilty for his involvement in a fraud scheme involving multiple sober homes that he operated in Massachusetts.
In 2017, then-STAT reporter David Armstrong wrote a story with the Boston Globe’s Evan Allen about what they called “addict brokers.” These brokers were almost like bounty hunters — they’d recruit people struggling with addiction from the Northeast and Midwest, then arrange transportation and insurance coverage for them to travel to Florida for treatment. But the rehab centers that brokers work with often provide few services and can be run by people without any actual training or expertise.
Daniel Cleggett was one of those brokers. Read the story about the widespread patient trafficking he was involved in, and a follow-up piece from the same year about the patients who get played as pawns in the system.
Study: New treatment approach could ease common childhood cancer relapse
A new treatment approach to a type of leukemia that’s the most common childhood cancer — combining standard chemotherapy with immunotherapy — saw significantly better survival rates than chemotherapy alone in a study published this weekend in the New England Journal of Medicine.
The most common cancer in children is a blood and bone marrow cancer called B-cell acute lymphoblastic leukemia, or B-ALL. Despite a pretty high survival rate — about 85% of those under 18 who get the disease are still cancer-free five years later — relapse from B-ALL is a leading cause of cancer-related death for youth. Researchers randomized the two courses of treatment to 1,440 kids with an average or high risk of relapse, but ended up stopping the trial early because the results were so promising. Overall, the study showed a 61% risk of B-ALL relapse or death for those receiving the combination.
As with any treatment, there were side effects: Those kids who received the more effective combination treatment were also more likely to get sepsis and catheter infections. Still, the study authors believe the combination approach could become a new standard of care.
RI will open first supervised drug consumption site outside of NYC
A Rhode Island nonprofit is set to open a government-sanctioned site for illicit drug use this week, making it just the second organization in the country to officially offer supervised consumption, and the first to do so outside New York City.
The goal of supervised consumption is to prevent overdose deaths by allowing people to use drugs under medical supervision. Evidence on the overall effectiveness of these sites is limited, but most studies suggest their presence is linked to a reduction in overdose deaths. While groups in other states have announced similar plans, they’ve faced resistance at the local, state, and federal levels.
And the barriers may only grow. President-elect Donald Trump will take office in less than two months, and conservative political leaders have typically opposed supervised consumption. Read more from STAT’s Lev Facher about how the Rhode Island site broke new ground in the legal fight for harm-reduction.
How generative AI is already transforming radiology
These days, the main clinical application of artificial intelligence is in medical imaging: Algorithms that help to analyze CT scans, MRIs, and X-rays account for more than three-quarters of AI-based devices authorized by the FDA. But by 2030, generative AI could become ubiquitous in radiology.
That hypothesis comes from radiologists themselves, at the annual meeting of the Radiological Society of North America. Hallway discussions and official sessions alike circled around products using large language models to streamline radiology documentation.“I think that this kind of tool will be so powerful that essentially it’s going to read our minds at some point,” said one radiologist, who consults for Open AI and MD.ai.
Generative AI took the spotlight at the meeting at the same time that the technology’s applications in medicine have been receiving increasing scrutiny. Read more from Katie Palmer on how people are talking about it.