Leqembi gets full approval from the FDA

As expected, the FDA has granted a full approval to the Alzheimer's drug Leqembi — broadening patient access to it via Medicare. The drug, sold by Eisai and Biogen, is the first to clearly slow cognitive decline associated with the neurodegenerative disease. A large clinical trial showed that Leqembi slowed the rate of cognitive and functional decline by 27% compared to placebo, dramatically reducing levels of beta-amyloid plaques.

Regulators had granted conditional approval to Leqembi in January, but this new status will likely boost sales as hundreds of thousands more people will now be eligible for it. The drug will now carry a black-box warning that clearly states the risks of brain swelling or bleeding.

The approval opens up the Alzheimer's arena, as physicians will now have to decide which patients are the right fit for these new medicines — because many more are under development. Right now, a definitive diagnosis requires a full PET amyloid scan or a spinal tap. So there's a pressing need for better Alzheimer's diagnostics and other ancillary tools to allow more comprehensive treatment of the disease, opines Howard M. Fillit, chief scientific officer of the Alzheimer's Drug Discovery Foundation.

"Ultimately, it will take a combination therapy approach, similar to those used in cancer treatment, to slow down the progression of the disease and stop Alzheimer's in its tracks," he writes.

psychedelics

The political right is on board with psychedelics

On stage at the largest-ever psychedelics conference, former Texas governor Rick Perry recently described himself as the "knuckle-dragging, right-wing Republican former governor of the state of Texas. He went on to elaborate on why he's a staunch advocate of drugs like MDMA and other psychedelics to help treat veterans with post-traumatic stress disorder.

Psychedelic drugs used to be associated with hippies, the anti-war movement, and the far left — but no longer. The right side of the aisle is warming up to these mind-altering substances, listening to testimony from veterans about their efficacy.

"Anyone that considers themselves a patriot, or would do anything for the veterans or service members in this country, is really starting to listen and come around to the idea that this is what it takes," one veteran mental health advocate told STAT. "These men and women deserve it."

crispr

Gene editing helps validate an old cancer hypothesis

More than a century ago, scientists theorized that cells with unusual numbers of chromosomes are a primary cause of cancer. The concept was shelved as other discoveries about the disease emerged, even though more than 90% of cancers have these chromosomal imbalances. "For many decades it was kind of ignored. It was like an elephant in the cancer room," one molecular genetics researcher told the Washington Post.

But thanks to CRISPR technology, this hypothesis has been resurrected and validated, a new paper in Science shows. The work shows that without extra chromosomes, some cancer cells can't proliferate in animal models. One Yale cancer biologist CRISPRed a herpes virus gene into a cancer cell's extra chromosomes, then used the herpes drug ganciclovir to target the modified chromosomes.

This method killed the herpes-infected cells with extra copies, leaving behind only the cancer cells that had a normal number of chromosomes. When researchers tried to grow these right-sized cancer cells, they could not — suggesting that the extra chromosomes are drivers of cancer. The hope here is that this new insight could develop new therapeutic approaches to kill cancer cells.

drug pricing

Generics lobby sues Minnesota over new law

The Association for Accessible Medicines, a trade group that lobbies for the generic drug industry, has sued Minnesota, arguing that the state's new law aimed at increasing transparency for drug pricing is unconstitutional.

"Under the new price-control law, AAM's members will be exposed to massive civil penalties and other monetary liability for selling their products at prices deemed by the (law) to be unacceptable, even if charged wholly outside Minnesota," the lawsuit alleges. It called the state's regulations "draconian," particularly when the generic drug industry is "already undergoing severe financial strain."