Drugmakers testing Alzheimer's drugs on asymptomatic candidates

Drugmakers are pushing for earlier intervention for Alzheimer's disease. Trials are underway to test whether asymptomatic volunteers who have amyloid buildups in the brain might respond to an intravenous drug that could offset the onset of symptoms.

"I often use the cholesterol analogy," said the neurologist leading one study at Brigham and Women's. "If you give somebody a statin drug when they're in the ICU, it does nothing. But when you give states before people have a heart attack or a stroke, it really changes the morbidity and mortality. Alzheimer's disease is very similar. If we can go after this underlying process early enough, we can one day prevent dementia."

cellular therapy

A CAR-T approach for all blood cancers?

Penn researchers Carl June and Saar Gill have been crafting a way to treat virtually all blood cancers with a single CAR-T therapy. The best bet, they found, was to target the CD45 protein, which is common to all blood cancers. Unfortunately, however, CD45 is also found on every healthy blood cells — so the scientists began searching for a way to prevent cellular fratricide.

A new study in Science Translational Medicine shows that they, along with grad student Nils Wellhausen, may have figured it out — a way to kill blood cancers while leaving their healthy kin unscathed by essentially rendering the CD45 protein invisible.

The work is years away from human study, and involves a new form of genome editing and several cellular infusions, but "it's very clever and really a tour de force," one Mass General cellular immunotherapy expert told STAT.

psychedelics

A single psilocybin dose has enduring effect on depression

Just one dose of psilocybin could have enduring efficacy for major depressive disorder, a new Phase 2 study published in JAMA shows. In the trial, 104 participants in the midst of a depressive episode were either given a 25-milligram dose of psilocybin or a placebo dose of niacin. Not only did the volunteers taking the psilicybin show a statistically and clinically significant reduction in depression eight days after receiving the drug, but the effects lasted through the end of the six-week observation period.

"It's exciting to see another study that's coming out that is adding to the evidence base suggesting potential efficacy of psilocybin for depression, among other conditions," one psychedelic researcher not involved in the study told STAT.

gene therapy

AstraZeneca carrying the AAV torch

Pfizer has bought three gene therapies for muscular dystrophy that had progressed far into clinical trials. But earlier this year, it scrapped many of its early stage assets that were based on the adeno-associated viruses that are regularly used in gene therapy. The move seemed emblematic of an industry shift away from AAVs, which are being shelved in favor of new technologies like CRISPR.

But last month, AstraZeneca said it would acquire Pfizer's AAV portfolio for $1 billion. Researcher Seng Cheng, who was formerly at Pfizer, has shifted to Alexion, AstraZeneca's rare disease subsidiary. He's following the AAVs that he's built a career around — telling STAT that these gene-delivering vehicles are still best in class.

"The most mature technology that we have in hand, for which we have the most experience of success, is the AAV platform," Cheng said. "Yes, there are challenges, but there are also many attributes that we need to talk more often about as well."

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