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Good morning, I have a packed, stacked newsletter for you today. Highlights include a moving article from my colleague Jason Mast and a long-awaited update to a 2017 STAT story that editor Gideon Gil reported to me.
A desperate father becomes a beacon in the world of rare diseases
Terry Pirovolakis is not a scientist or doctor. He’s not rich. He is a 45-year-old IT professional, has three children and a penchant for monochrome T-shirts. He has also somehow become the first stop and last hope for many families who have watched profitable drug companies abandon the rare diseases that afflict their children.
Over the past two decades, researchers have made tremendous progress in their ability to diagnose one-in-a-million mutations and devise treatments with gene therapy, CRISPR, or RNA-based drugs. But in recent years, companies have shelved or deprioritized more than 50 gene therapies, leaving the families who counted on them adrift.
Pirovolakis’ first goal was 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 within three years. He then became an informal counselor for parents struggling to get treatment for their own children. He then founded his own biotech company.
STAT’s Jason Mast has a wonderful article about Pirovolakis and a family he helped, the Lockards, pictured above. As Jason writes in the story, there are different 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 kid selling lemonade to raise money for his own cancer treatment.
Read the story.
Anger towards 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 healthcare. You’ve probably seen a number of posts online, ranging from sad 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 found a place for it,” says bioethicist Yolonda Wilson.
Public dissatisfaction has never been greater. A survey published Friday shows that public approval of the health care system is at 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 Friday that it is instituting a mandatory milk testing program that should provide a much clearer picture of how widespread the bird flu virus is in the nation’s dairy industry. The program will begin in 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 often it will be released.
Read more from STAT’s Helen Branswell.
An ‘addicted real estate agent’ pleads guilty seven years later
Seven years after STAT first wrote about him, an “addicted broker” named Daniel Cleggett has done just that pled guilty for his involvement in a fraud scheme involving multiple sober houses 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 “addicted real estate agents.” These brokers were almost bounty hunters, recruiting people struggling with addiction from the Northeast and Midwest and then arranging transportation and insurance coverage so they could travel to Florida for treatment. But the rehabilitation centers brokers work with often offer few services and may be run by people without any actual training or expertise.
Daniel Cleggett was one of those real estate agents. Read the story about the widespread patient trafficking he was involved in, and a follow-up piece from the same year about the patients being played as pawns in the system.
Study: New treatment approach could alleviate the common relapse of childhood cancer
A new treatment approach for a type of leukemia that is the most common form of cancer in children – combining standard chemotherapy with immunotherapy – resulted in significantly better survival rates than chemotherapy alone, according to a study published this weekend in the US newspaper The Guardian published. New England Journal of Medicine.
The most common cancer in children is a blood and bone marrow cancer called B-cell acute lymphocytic leukemia, or B-ALL. Despite a fairly high survival rate – about 85% of those under 18 who develop the disease are still cancer-free five years later – relapse of B-ALL is a leading cause of cancer-related death among young people. Researchers randomized the two treatment courses among 1,440 children at moderate or high risk of relapse, but ultimately stopped the trial early because the results were so promising. Overall, the study showed a 61% risk of B-ALL relapse or death for those given the combination.
As with any treatment, there were side effects: the children who received the more effective combination treatment were also more likely to develop sepsis and catheter infections. Still, the study authors believe the combination approach could become a new standard of care.
RI will open the first supervised drug consumption site outside of New York
A Rhode Island nonprofit will open a government-sanctioned illegal drug use site this week, making it only the second organization in the country to offer officially monitored 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 about the overall effectiveness of these locations is limited, but most studies suggest that their presence is associated with a reduction in overdose deaths. While groups in other states have announced similar plans, they have been met with resistance at the local, state and federal levels.
And the barriers may only increase. President-elect Donald Trump will take office in less than two months, and conservative political leaders have generally opposed controlled consumption. Read more from STAT’s Lev Facher about how the Rhode Island site broke new ground in the legal battle for harm reduction.
How generative AI is already transforming radiology
Today, the main clinical application of artificial intelligence is in medical imaging: algorithms that help 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 be ubiquitous in radiology.
That hypothesis comes from radiologists themselves, at the annual meeting of the Radiological Society of North America. Both hallway discussions and formal sessions revolved around products using large language models to streamline radiology documentation. “I think these kinds of tools will be so powerful that at some point they will essentially read our minds,” says one radiologist who consults for Open AI and MD.ai.
Generative AI was in the spotlight at the meeting, as the technology’s applications in medicine come under increasing scrutiny. Read more from Katie Palmer about how people are talking about it.