Breaking News

Why FDA advisers voted against NurOwn's ALS drug

September 28, 2023
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Reporter & Podcast Producer

Hello, this is Theresa, next in line filling in for Liz this week! If you missed last night's GOP primary debate, be sure to check out my colleague Sarah's rundown of what  Republican presidential candidates had to say about health care. 

biotech

FDA advisers vote overwhelmingly against ALS drug

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A panel of independent advisers to the Food and Drug Administration voted overwhelmingly against a polarizing potential treatment for ALS on Wednesday, concluding that the medicine's messy supporting data did not meet the standard for approval.

The agency's expert advisers voted 17-1 with one abstention that the case for NurOwn, a treatment from BrainStorm Cell Therapeutics, was based too heavily on convoluted clinical trial results and compelling but unreliable anecdotal evidence.

BrainStorm's treatment has become the latest flashpoint in a societal debate over how to balance the needs of patients with grave diseases against the traditional standards of reviewing new medicines. The agency has promised to make a final decision on BrainStorm's medicine by Dec. 8.  STAT's Damian Garde has more.


MENTAL HEALTH

Shedding new light on substance-induced psychosis 

People who visit the emergency room for substance use are at higher risk of developing schizophrenia than the general population, according to new research in JAMA Psychiatry. The retrospective study looked at health records from about 400,000 people in Ontario, Canada who went to the emergency room for substance use between 2008 and 2022. Almost 14,000 went to the ER because of substance-induced psychosis. 

The study found that 18.5% of patients with substance-induced psychosis developed schizophrenia within three years of their visit to the emergency department. The researchers also found that even those who didn't experience psychosis had a 1.4% risk of developing schizophrenia over the next few years, which is much higher than the general population's 0.1% risk. Also noteworthy is that 26% of those who went to the ER experiencing psychosis after cannabis use developed schizophrenia within three years — a rate 242 times higher than the general population. These findings emphasize the need for prevention efforts aimed at reducing substance use, particularly early intervention measures, the authors wrote.


in the lab

New patch takes inspiration from popcorn and octopi 

When I heard about this new drug delivery technology — a patch that can deliver peptides and proteins by suctioning to the inside of a patient's mouth — I understood how researchers might have found inspiration in octopus tentacles. But as STAT's Annalisa Merelli reports, the researchers developing this patch were first intrigued by how Sichuan peppercorns and popcorn stuck in their mouths. So they initially aimed to re-create the food's suction-cup shape before landing on octopi as their ultimate model.

And it isn't just a fun idea — experts say there's a need for alternative ways to deliver peptides and large proteins. Needles aren't fun or convenient. Some innovations, like microneedles and nasal sprays, have attempted to make the delivery of peptides less invasive, while just a few can be delivered by tablets. Still, there's more testing needed on the suction patches before we're all chewing on them in our spare time. "The mouth is very easily accessible, so that's kind of nice. But at the same time… it's easily accessible, and that can also be a problem," Arturo Vegas, a professor of chemistry at Boston University, told Annalisa. Read more.



Closer Look

Did the U.S. get a bad deal on Covid-19 boosters?

After Pfizer and Moderna hiked the prices of their Covid-19 vaccines this year, the U.S. government will now pay nearly three times more than it did previously for each dose. It's also paying more than countries that did far less to support vaccine development, my colleague Rachel Cohrs writes.

The change is partly because the federal government ran out of money to continue to buy the vaccines in bulk to distribute across the country, so the vast majority of vaccine purchases will be paid for by a variety of different insurance plans this year, including Medicare and Medicaid. The fact that the government is purchasing fewer doses gave the government significantly less leverage.

"Taxpayers and U.S. patients are getting a raw deal, again," Public Citizen Access to Medicines Director Peter Maybarduk told STAT. Read more.


mental health

Mental health worsens during menstruation — even on the pill 

New research published in JAMA Network Open suggests that for those who take birth control pills, negative mental health symptoms can increase while menstruating in a similar way to those who aren't on birth control.

The research focused on combined oral contraceptives — the standard daily birth control pills that include both estrogen and progestogen. The pills for the last week of each monthly cycle are typically placebo sugar pills, to mimic the hormonal shifts of a menstrual cycle. The researchers found that out of 181 participants, those who took the pill (120 people) reported an almost 13% increase in negative affect and 7% increase in anxiety during the week of placebo pills compared to their baseline.

The changes were worse for those with higher baseline depression scores, but overall similar to the shifts experienced by those who don't take the pill. Researchers noted that the results made them question whether the week of placebo pills is worthwhile from a mental health perspective.


research

People with disabilities get new designation from NIH

It may sound like common sense, but people with disabilities are now officially a "population with health disparities" per a new designation from the National Institute on Minority Health and Health Disparities. Activists and disabled community members have long called for the official label, which increases research funding and resources for designated populations like racial and ethnic minorities and sexual and gender minority groups.

A recent NIH advisory council report noted that "persons living with disabilities face tremendous health disparities that impact quality of life, morbidity, and mortality, as well as discrimination, based on their abilities and identity." One in four people in the U.S. live with disabilities, according to the NIH, and that number is expected to increase as chronic medical conditions increase and the population ages.

Agency leaders concurrently announced funding opportunities for research aimed at addressing disparities around disability and its intersections with other factors, like race and socioeconomic status, that compound issues with access to health care and health disparities.


More around STAT
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What we're reading

    • Republicans aren't quite done talking about Obamacare, after all, STAT
    • 24 hours in an invisible epidemic, The Pudding
    • 6 ways the government could help address drug shortages, including in cancer, STAT
    • Fight to save a New York birth center tests state law, Wall Street Journal
    • Researchers struggle to convey the complex impacts of climate warming on infectious disease, Science

Thanks for reading! More tomorrow — Theresa


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