Breaking News

FOP patients plead for FDA approval of drug despite muddled data

August 14, 2023
Reporter, Morning Rounds Writer
Good morning. First things first: I apologize to readers of last Friday's edition, in which I jumped the gun on a fascinating First Opinion from a dentist who doesn't want us to give up on artificial sweeteners like aspartame. Her commentary is live on our site today.

rare diseases

When the data are muddled but the need is clear, patients plead with FDA for 'something'

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Mike Reddy for STAT

When trial results are ambiguous but the need is unequivocally dire, where do you draw the line? U.S. regulators will face that conundrum Wednesday about a drug that promises to slow a disease in which rigid bone grows where it shouldn't, locking bodies and limiting lives. The disease is fibrodysplasia ossificans progressiva and the medicine is Ipsen's palovarotene, which European regulators rejected earlier this year. It represents years of research, advocacy, and fundraising to find a treatment for a rare condition that has none.

"Even if it's not the perfect drug, to have something where there's data to show that it helps, that's just so important," said Emma Albee, 34, who's relied on a wheelchair since adolescence. For the FDA, whose advisory committee endorsed the treatment despite misgivings about its reliance on post-hoc analyses, the case may fuel ongoing debate about regulatory flexibility. STAT's Andrew Joseph and Damian Garde have more, from Europe and the U.S.


business of health

Some hospitals are quietly punting on VC offices 

Before the pandemic, many sizable health systems enthusiastically embraced venture capital offices, investing in new technologies and devices while offering hospitals as testing grounds for startups. That hopeful premise seems to have evaporated for some systems as they dissolve venture offices, halt new investments, or choose private equity instead. Among them: NewYork-Presbyterian, Advocate Health Enterprises in Charlotte, N.C., and Providence Ventures in Seattle.

"Most hospitals can't get enough dollars for it to make a whole lot of sense," said Parth Desai, a former investor at NewYork-Presbyterian's venture fund who now works at Flare Capital Partners. That could be bad news for the entrepreneurs who rely on hospitals to be their keystone customers, and for patients frustrated with entrenched problems that a breakthrough technology could solve. Still, some of the country's largest health systems say despite the challenges, they're going full steam ahead on venture capital. STAT's Tara Bannow has more.


shortages

As hospitals ration drugs, White House task force MIA 

Here's how bad the dearth of prescription drugs has become: Nearly every hospital pharmacy across the U.S. is experiencing shortages. The problem has become so severe — especially for chemotherapies — that one-third of hospitals are rationing, delaying, or canceling treatments or procedures, according to a new survey. The causes vary from quality control failures at manufacturers to tornadoes to surging demand. STAT's Ed Silverman has more.

Meanwhile, a White House task force formed early this year to work on drug shortages has yet to contact the lawmakers writing legislation to address the problem — and it's not clear when it will recommend policies. Sen. Bernie Sanders (I-Vt.) said he hadn't spoken to anyone on the White House task force. A spokesperson for Sen. Bill Cassidy (R-La.) said there has been no communication from the task force. The White House did not respond to questions from STAT's John Wilkerson. Read more.



Closer Look

Kim Kardashian enters the fray on full-body scans

AP23133099504171Ashley Landis/AP

This first sentence in Lizzy Lawrence and Mohana Ravindranath's takedown could be a mic drop, but keep reading: "The last time Kim Kardashian (above) posted about medical imaging, it was to prove her butt was real. Now, she's praising its ability to find aneurysms and cancers before they turn deadly." Kardashian's post on Instagram about the company Prenuvo raises serious questions about full-body MRI scans that can cost thousands of dollars and lead to false positives and unnecessary follow-ups for wealthy and largely healthy patients.

Prenuvo is just the latest in a long list of celebrity-endorsed scans, along with Ezra, Neko Health, and SimonMed. There are exceptions to false positives, but radiologists have been sounding the alarm on the dangers of over-testing for decades. MRIs can pick up legitimately threatening conditions, but they also pick up abnormalities that prove to be completely benign. Read what the companies say.


In the lab

Even amateur tackle football tied to later Parkinson's

Boxing and Parkinson's disease have been connected at least since the 1920s, long before Muhammad Ali died from the neurodegenerative disease linked to head trauma. But while American football at the professional level has been tied to chronic traumatic encephalopathy, or CTE, how tackle football might be related to Parkinson's or other symptoms called parkinsonism hasn't been established. A new cross-sectional study in JAMA Network Open found that playing tackle football, even in high school and college, raises the odds of developing Parkinson's, with the risk rising along with years of play.

Among 1,875 trial participants — 729 men who played football, mostly as amateurs, and 1,146 men who played non-football sports — those who played tackle football carried a 61% higher risk of having a Parkinson's or parkinsonism diagnosis than those who played other sports. The risk for college or professional players was 2.93 times higher than for those who played only at the youth or high school level.


health

Opinion: Don't give up on aspartame, dentist pleads 

Two weeks ago, when an agency within the WHO linked artificial sweeteners to cancer, some people clutched their Diet Cokes in fear. Aspartame, a sugar substitute in about 6,000 products worldwide, was deemed "possibly carcinogenic to humans." Other sugar substitutes (saccharin, sucralose, stevia) have come under fire, too. But the FDA strongly disagreed with the aspartame assessment, and so does dentist Melissa Weintraub.

"If non-nutritive sweeteners were to fall out of use altogether, would our health be in a better or worse position?" she asks in a STAT First Opinion. "The answer is obvious: We'd be in a worse place because of our relationship with sugar, which humans crave." That's not only down to the damage sugar can cause, but sugar-free gum's value as an oral health tool. "Helping reduce harmful mouth bacteria, prevent gum disease, and protect tooth enamel is precisely what chewing sugar-free gum can do." Read more.


The latest episode of Color Code explores how Long Island became the "eugenics capital of the world" and how the dangerous pseudoscience's shadow still affects aspects of racist policies today. Listen here.


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What we're reading

  • After Optum Health's lowest profit margin in a decade, UnitedHealth makes changes, STAT
  • Redesigning the siren, New York
  • A slightly sadistic experiment aims to find out why heat drives up global conflict, NPR

  • U.S. watchdog halts studies at N.Y. psychiatric center after a subject's suicide, New York Times

  • Opinion: Philanthropic endowments at universities can offer a way out of biotech's Valley of Death, STAT

Thanks for reading! More tomorrow,


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