Alzheimer’s therapies
Potential Alzheimer’s therapies could result from research into bexarotene, an anti-cancer drug that can suppress the onset of Alzheimer’s.

To date, there’s been no such thing as a catch-all treatment, but researchers might be zeroing in on something nearly as impressive. It’s suspected that bexarotene, an anti-cancer retinoid, might play a part in Alzheimer therapy too.

Researchers from the University of Cambridge, Lund University, and the University of Groningen published a study in the journal Science Advances called, “An anti-cancer drug suppresses the primary nucleation reaction that initiates the production of the toxic Aβ42 aggregates linked with Alzheimer’s disease.” It’s a mouthful, but as the title illustrates, the researchers had a lot to talk about.

The Study

The basis of the study isn’t as complicated as the name. The team of researchers took worms that were genetically modified to develop Alzheimer’s disease, and they introduced bexarotene into their systems. They observed something they didn’t expect –the treatment did nothing in the worms that had already developed symptoms of Alzheimer’s, but in those that hadn’t yet started showing signs of the disease, the bexarotene was completely effective in keeping the Alzheimer’s at bay.

The Cause of Alzheimer’s

Alzheimer’s disease is a degenerative neurological affliction. It’s brought on by the clumping of proteins called amyloid-beta, which form plaques and damage neurons in the brain. According to the study, over the next 35 years, the number of Alzheimer’s sufferers is expected to nearly quadruple from 40 million to 130 million people.


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Most of those afflicted, research shows, are projected to be citizens of middle- or low-income countries. With that amount of people projected to contract the disease, any potential solution would need to be preventative, because hospitals don’t have the infrastructure in place to offer prolonged treatment to more than 100 million people.

With that said, the bexarotene breakthrough hints at a preventative treatment. The study’s senior author, Professor Michele Vendruscolo, said, “The body has a variety of natural defenses to protect itself against neurodegeneration, but as we age, these defenses become progressively impaired and can get overwhelmed. By understanding how these natural defenses work, we might be able to support them by designing drugs that behave in similar ways.”

The researchers discovered that bexarotene puts a stop to primary nucleation – the clumping of proteins into plaques – which is the precursor to the death of a brain cell. This theory had been tested in the past, without success, but the team speculated that the failures had been a product of the wrong approach rather than the wrong treatment. They suspected that nucleation occurred a little differently than the medical community had initially thought, and they reengaged with a few treatments that had previously come up short, namely bexarotene.

The second attempt proved successful, but only on nematodes, without Alzheimer’s symptoms, who were in the larva stage of their lifecycles. Regardless, it is progress, and the team is optimistic that bexarotene will eventually be used to treat the disease in humans.

Dr. Rosa Sancho, the head of research at Alzheimer’s Research UK, offered her thoughts on the potential of a bexarotene treatment. “A recent clinical trial of bexarotene in people with Alzheimer’s was not successful,” she said, “but this new work in worms suggests the drug may need to be given very early in the disease.”

Professor Vendruscolo and team have a long way to go before they can manufacture a treatment for Alzheimer’s, but the different approach they’ve taken may help get them there faster than anyone ever expected.

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