CHICAGO (Reuters) - U.S. scientists say a dramatic result last year suggesting that a cancer drug already approved by U.S. regulators could quickly clear out Alzheimer's plaques in mice was too good to be true.

The study, published last year in the journal Science, showed the skin cancer drug bexarotene cut the amount of an Alzheimer's-linked protein called beta amyloid by half in three days. It also reversed Alzheimer's symptoms, restoring a sense of smell in treated mice and allowing them to resume nest building activities.


The news sent patients clamoring for the drug, and some doctors began prescribing it, even though it had not been tested in people with Alzheimer's. But researchers at several U.S. centers reported in the same journal on Thursday that they were unable to reproduce the most dramatic aspects of the findings in their own labs.

Gary Landreth and colleagues at Case Western Reserve University in Cleveland, the scientists behind the original research, say the drug still has merit, noting that the latest studies confirmed other aspects of the research showing the drug cleared out soluble forms of beta amyloid from the brain.


Scientists say the controversy is a stark reminder of the need for studies to be replicated by other labs, and it underscores the desperation of Alzheimer's sufferers to find effective treatments for the fatal, brain-wasting disease that affects 5 million Americans and 38 million people worldwide.


'VERY DRAMATIC'


"I was a fan of the original study," said Dr. Samuel Gandy, associate director of the Mount Sinai Alzheimer's Disease Research Center in New York, who was not involved in any of the studies.


"It was very dramatic. It cut plaque loads by three-quarters over less than a week. No one had ever seen anything like it before."


Gandy has had several patients asking for the treatment. Although U.S. doctors are free to prescribe any treatment approved by the FDA, Gandy said the drug has substantial liver toxicity and requires very careful monitoring.


"I have universally declined and advised others to decline."


There is no cure or effective treatment for Alzheimer's disease, a progressive form of dementia steadily destroys brain cells, and several large companies have failed in late-stage clinical trials, with the most recent being Baxter International's drug Gammagard earlier this month.


Sangram Sisodia, a professor of neuroscience at the University of Chicago, said he and his colleagues were curious about the initial report in 2012.


"It was hot stuff. It was the new miracle drug for Alzheimer's. Even Doctor Oz proclaimed such," said Sisodia, referring to a popular FOX TV show hosted by Dr. Mehmet Oz.


"People were clamoring for this drug after he went on TV and was pushing it."


Sisodia said he and fellow Alzheimer's colleagues, who included Dr. Rudolph Tanzi of Massachusetts General Hospital in Boston and Dr. David Holtzman of Washington University School of Medicine, wanted to see if the stunning results could be replicated in their own labs, a standard part of the scientific process.


Researchers failed to see any effects on Alzheimer's plaques in three strains of mice that were treated with bexarotene.


"There is absolutely no reduction in amyloid levels in the brains of mice treated with this compound," said Sisodia of his group's efforts, which were published as a technical comment in the journal Science. Teams at the University of Florida and researchers at the University of Leuven in Belgium published similar findings in the same journal.


TEMPEST IN TEAPOT


Landreth, who has formed a company to study the compound, says the teams are all focusing on the dramatic changes in solid forms of beta amyloid reported in the study, which, despite the press it got, was not the study's main finding.







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