Mouse Study Shows Potential of Bexarotene to Eradicate Beta Amyloid Plaque and Reverse Alzheimer’s Symptoms

In February 2012, researchers at Case Western University revealed results from a study using the anti-cancer drug bexarotene (brand name Tagretin) in mice that investigated what the drug would do to mice suffering from the equivalent of Alzheimer’s Disease. In particular, the study examined the impact on the amyloid beta proteins in the brains of the mice. To the surprise of the researchers, after about three days of treatment the mice recovered much if not most of their normal brain function as shown by improved behavior and cognition. Measurements of the amyloid beta plaque area in the brains showed a reduction of more than 50%. Given this, it appears bexarotene can rapidly reverse one of the main biochemical features of Alzheimer’s and reverse much of the behavioral and cognitive deficits, too.

Bexarotene was approved by the US FDA as a drug for treating cancer in 1999, so toxicity and side effects are already fairly well understood. If the drug can work similarly at fighting Alzheimer’s in humans, something that has not yet been established, it could become a short-term blockbuster drug for its owner, Japanese pharmaceutical company Eisai. And with the bexarotene patents set to expire in 2016, if it works safely it may become an affordable means to help prevent Alzheimer’s Disease within the next decade.

While these results are really exciting for people with a family history of Alzheimer’s, it’s important to note that nobody knows what the effective dosage is for this use of bexarotene. Rsearchers are hoping to have a small human trial underway within a few months to help determine dosages necessary for effectiveness against the amyloid beta plaque in human brains. They caution that mouse models of Alzheimer’s have often shown to be reversible with other drugs but the effects were not as potent in humans.

One major concern is that the drug may not work to cure Alzheimer’s even if bexarotene is highly effective at clearing amyloid beta buildup in human brains. Brain cells are severely damaged by this toxic protein and there’s no way to revive dead nerve cells. As a result, it may be the best use for bexarotene could turn out to be for treating the early stages of Alzheimer’s to keep brain cells from dying off in greater numbers. If that approach works well in future human trials, then bexarotene may turn out to be a helpful preventive medication for those with family history or elevated risk for Alzheimer’s.

Given the need for further human study, it seems likely it may be at least a couple of years before bexarotene can become a mainstream drug for preventing and treating Alzheimer’s. In the meantime, it’s possible for doctors to prescribe it for Alzheimer’s patients. For those with Alzheimer’s, you might be able to find a doctor willing to try it on you today, but don’t expect your insurance to cover it.

Further Reading

Alzheimer’s research is called ‘stunning’

Alzheimer’s Disease

Bexarotene appears to help clear out excess plaque in Alzheimer’s

Alzheimer’s Disease Symptoms Reversed in Mice

ApoE-Directed Therapeutics Rapidly Clear β-Amyloid and Reverse Deficits in AD Mouse Models

Bexarotine (Tagretin)


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