Experimental Alzheimer's drug stands up to closer examination

First, there was a press release. Late yesterday, there were a scientific presentation and a NEJM paper providing details on a new treatment for Alzheimer’s that raised hopes and eyebrows when trial results were first disclosed in a September press release. The drug, lecanemab, held up to scrutiny, showing it slowed the cognitive and functional decline of patients with early Alzheimer’s by 27% relative to placebo in a roughly 2,000-volunteer clinical trial.

Its developers, partners Eisai and Biogen, said the drug dramatically reduced patients’ levels of beta-amyloid, a toxic protein in the brain thought to drive the advance of Alzheimer’s, and it showed statistically significant benefits on measures of cognition and function. Lecanemab is expected to win preliminary FDA approval in January, based on its ability to reduce amyloid. Read more from STAT’s Damian Garde and Jonathan Wosen, including Eisai’s first detailed comments on the deaths of two patients receiving lecanemab alongside blood-thinners.

Out of prision, 'CRISPR babies' scientist is back

Less than six weeks after finishing his sentence for violating Chinese regulations and medical ethics, the scientist who made headlines and earned condemnation for creating the first gene-edited children has jumped back into the lab. He Jiankui has posted videos and messages on social media this month saying he’ll focus on developing affordable gene therapies. The first rare disease he’d like to tackle is Duchenne muscular dystrophy.

Back in May, He attended a closed-door meeting hosted by the Global Observatory for Genome Editing via Zoom to respond to questions about the circumstances that led up to the controversial "CRISPR babies" experiments and his motivations in conducting them. It seems to have been just the beginning of the publicity tour now underway, STAT’s Megan Molteni reports. STAT reached out to He, but at the time of publication had not yet received a response.

Why experts say China's 'zero Covid' policy will fail

China’s "zero Covid" policy, which has kept cases and deaths in China to negligible numbers throughout the pandemic before drawing unprecedented protests, seems doomed to fail, experts believe. Just look at Hong Kong, they say, which adopted similarly draconian quarantine, tracing, and testing policies to try to stop the SARS-CoV-2 virus. It worked until the highly transmissible Omicron variant broke through Hong Kong's defenses and infected half the population in a matter of weeks.

Australia, New Zealand, and Singapore also adopted a zero Covid policy early on in the pandemic, but only until vaccines arrived. China has deployed vaccines, but not the more effective mRNA versions. Low infection rates — the very thing that has protected China from Covid — may now be rendering it more vulnerable to the disease because people lack the hybrid immunity born of infection and vaccination. STAT’s Helen Branswell explains.

Closer look: Huntington's genetic stutter sends scientists back to overlooked clues

(MIKE REDDY FOR STAT)

A landmark discovery in 1993 promised to unlock the molecular underpinnings of Huntington’s disease and provide a road map to a potential cure. That discovery revealed a lethal genetic stutter that devastates families with the brain-ravaging Huntington’s disease. But almost three decades later, effective treatments — let alone curative ones — have yet to materialize. Huntington’s researchers, like others laboring to understand complex neurological disease, have long focused their efforts on mutant proteins produced by the problematic gene, in this case HTT.

But reducing mutant proteins hasn’t helped. Now a growing number of researchers are questioning the underlying premise of that therapeutic strategy. Some are looking at a phenomenon called somatic expansion as well as DNA repair. “I don’t think we’re certain what causes the toxicity, but we know enough now to know that the earliest target is DNA instability and I’m hopeful about that,” geneticist James Gusella, who's been there from the start, told STAT’s Megan Molteni. Read more.

Single-dose oral drug for sleeping sickness sparks hope for elimination

It could be another watershed moment for sleeping sickness, a neglected tropical disease that can be fatal if not treated. A new study in The Lancet Infectious Diseases concludes that a single oral dose of acoziborole was safe and 95% effective in adults and adolescents 18 months after being given. Until 2019, the treatment for early stages of the parasitic disease known as human African trypanosomiasis required a daily injection for at least a week or an IV drip in the hospital for later-stage disease (after it crosses the blood-brain barrier), with a spinal tap to determine the stage.

Fexinidazole, a 10-day oral drug, was introduced in 2019 for both stages, but skilled care and sometimes hospitalization was still required. The new study, which drew patients from 10 hospitals in the Democratic Republic of Congo, raises hopes for meeting WHO’s goal of eliminating transmission of the disease by 2030.

Twitter has stopped monitoring Covid information. How much will it matter?

That was then.

This is now.

If you’ve been watching Twitter since Elon Musk took over — or following people who do — you know fears are growing by the day that misinformation will metastasize in the name of free speech. Now that the platform’s hands-off approach to misleading Covid-19 information is official, it’s worth looking back at what STAT’s Brittany Trang reported earlier this month. Yes, the WHO’s Mike Ryan and the FDA’s Robert Califf warned of the dangers of mis- and disinformation. But experts also told her then that while Twitter had done a better job than most other social-media platforms in developing approaches to combating misinformation, its policies seem to have been applied unevenly.

One study showed that 59% of Covid-19 claims on Twitter that fact-checkers had determined to be false still did not have misinformation labels on them. Conversely, in 2022, correct information about Covid-19 was flagged mistakenly. Still, Twitter says 11,000 accounts were suspended under the policy. Meanwhile, the #TwitterMigration continues, with many scientists fleeing for other platforms, and Musk has reinstated the accounts of several figures who had spread false information about Covid.

What we're reading

Mayor says NYC will treat mentally ill, even if they refuse, Associated Press
Can this man stop lying? New York Times
‘The more voice, the better’: How technology can help put the reality of living with a disability into health records, STAT
That knee surgery you postponed could soon hobble insurance giants, Wall Street Journal
Scientists revive 48,500-year-old ‘zombie virus’ buried in ice, Bloomberg