Bluebird gene therapy for a rare disorder gains FDA advisors' recommendation

On the first of two days for an FDA advisory panel to review investigational gene therapies developed by Bluebird Bio, the committee unanimously endorsed a treatment for a rare and deadly disorder, despite a known risk of cancer. In a 15-0 vote, the advisers agreed to recommend for approval eli-cel, a one-time treatment for cerebral adrenoleukodystrophy, a genetic neurological disorder that affects young boys. The FDA isn’t required to follow suit, but it will make its decision by Sept. 16. “This treatment isn’t a cure, but it at least gives these boys time until hopefully one day we can come up with something better for them,” advisory committee member Stephanie Keller of Emory University said.

Up for discussion today: a Bluebird gene therapy for sickle cell disease. STAT’s Damian Garde has more on the impact for patients and for the company.

Poll captures confusion around medication abortion

(KFF)

Many public opinion polls have reported that most Americans don’t want the Supreme Court to overturn the Roe v. Wade decision establishing a constitutional right to an abortion. A KFF Health Tracking Poll out yesterday confirms that sentiment, but it also add adds something new: The week after Politico reported on the leaked draft opinion on abortion, most women of reproductive age said they were unaware of medication abortion and many confused it with emergency contraception.

While abortion using the pill mifepristone accounts for more than half of all abortions in the U.S., just 27% of adults overall and 40% of women under 50 have heard of it. Far more adults (92%) have heard of emergency contraceptive pills, but 73% incorrectly believe emergency contraceptive pills can end pregnancy in its early stages.

Substance use and psychiatric problems come together among many seeking treatment

Here’s a concerning pre-pandemic picture showing how substance misuse is intertwined with mental health issues. A CDC report looked into how many people seeking help for substance misuse also reported moderate to severe psychiatric problems. The researchers found that among more than 49,000 adults at 499 treatment centers, alcohol was the most common substance named (36%), followed by polysubstance use (33%) — any combination of alcohol, cannabis, opioids, stimulants, cocaine, or sedatives.

More than a third of people looking for help with substance misuse also said they had psychiatric problems, among other biopsychosocial issues. That term covers a lot of ground, also including family, medical, legal, and work issues. “Actions to enhance comprehensive substance use programs that incorporate polysubstance use and co-occurring mental health problems into strategies for prevention, treatment, and response are needed, as is expanded linkage to services,” the researchers say.

Closer look: From the noses of hamsters, insights into long Covid

(adobe)

Last summer, researchers started dabbing infectious coronavirus particles into the nostrils of 30 sedated hamsters in their lab cages. The scientists waited for them to develop not just Covid-19, but long Covid, the constellation of lingering symptoms that have defied understanding. Now those hamsters have yielded clues. A study in Science Translational Medicine shows that compared to flu virus, the coronavirus sparked persistent inflammation in smell-sensing regions of the brain in those hamsters and in patient samples. "It is the one place where SARS-CoV-2 differentiates itself from your average response to infection,” virologist and study co-author Benjamin Tenoever told STAT's Jason Mast.

“We are in dire need of new pathways to knowledge that can support diagnostics and therapeutics for this condition,” Harlan Krumholz, a Yale cardiologist not involved with the study, told Jason. Colleagues at Yale have developed a mouse model that mimics brain fog. “Any potential advance, especially the development of an animal model, is very welcome.” Read more.

Preliminary study links Covid in pregnancy to babies' neurodevelopment

Researchers have a moving target when they look at Covid-19, and its impact on children born after maternal infection is no exception. A new JAMA Network Open study found more neurodevelopmental diagnoses in the first year of life in babies whose mothers had positive PCR tests compared to those who didn’t. Acknowledging the preliminary nature of their work, the authors also say they can’t ignore previous connections made between viral infections and autism spectrum disorders, schizophrenia, cerebral palsy, cognitive dysfunction, bipolar disorder, and anxiety and depression.

Here’s what they found analyzing health records for 7,772 infants born in 2020: About 6% of babies whose mothers had Covid were diagnosed with a neurodevelopmental disorder — such as motor or speech delays — compared to 3% of babies whose mothers didn’t, after accounting for prematurity and demographic factors. The researchers, and a companion editorial, urge longer studies.

Antimicrobial resistance as an issue of access

If you think about antimicrobial resistance as a problem of inappropriate prescribing or an empty pipeline while pathogens evolve to escape treatment, you’re not wrong but perhaps incomplete. A new report from the Access to Medicine Foundations makes the case, as its name might suggest, that the waning power of antibiotics and antivirals to beat infectious bugs is also influenced by access to appropriate antibiotics. Its analysis explains how certain measures can overcome the current fact that low- to middle-income countries use what’s available, not necessarily what’s best. These strategies helped six pharma companies clear local bottlenecks:

Supporting local manufacturing and technology transfers
Identifying the right public-health partners
Defining local demand to introduce generic medicines
Making on-patent products available through license agreements and access programs
Harmonizing registration across regions

“All stakeholders need to step up,” the report concludes.

What to read around the web today

Vaccines for the littlest kids have already flopped, The Atlantic
With Cerner deal closed, Oracle chair pitches sweeping vision for health records and artificial intelligence, STAT
Age to buy cigarettes in England should rise every year, review says, Reuters
How high? Prices for newly launched medicines rose 20% annually, analysis finds, STAT
Raising the drinking age helped reduce crashes. Could age limits curb gun violence? NPR