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Patients Clamor for Cancer Drug That Shows Promise for Alzheimer's in Mice

This article was published in Scientific American’s former blog network and reflects the views of the author, not necessarily those of Scientific American


The pharmaceutical industry has beat a concerted retreat from developing drugs for diseases that affect the brain, stymied by the lengthy development times for these agents and a string of failures. Despite the evident risks, a new study shows how industry leaders should perhaps be taking the long view.

The report online last week in Science that an already approved cancer drug showed promise in mice in correcting both the molecular pathology and cognitive decline of Alzheimer's has patients and their families clamoring for the compound.

Those suffering are asking by the hundreds for the drug despite warnings that evidence in mice often does not translate into later success in humans. Gary Landreth of Case Western Reserve University received a flood of requests from desperate families.


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Landreth, the lead researcher on the study, did not hype the results. He acknowledged that bexarotene rapidly cleared the toxic amyloid peptides and seemed to improve cognition in mice. But he also emphasized that rodents differ from humans—and that examining whether the drug can eliminate amyloid in a small human trial must be demonstrated now before moving forward to a larger test to ascertain whether cognition improves as well. In our story last Thursday, Landreth cautioned:

"Don't try this at home because we don't know what dose to give, we don't know how frequently to give it, and there are a few nuances to its administration. So one shouldn't be prescribing it off-label."

It is also unclear whether a drug like bexarotene, even if it were a success for patients in the early stages of the disease, would work later as the pathology progresses and nerve cells start to perish.

The fallout from this story turned up in our comments section. One reader, identified only as Jeff_Davis, responded to another's remarks by saying:

"You write: ...'I'd be very worried about off-label use...'

"I guarantee you, this is way past 'worry.' A tsunami of off-label use is underway even as we speak. Friends, already in the grip of the Alzheimer's horror -- loved ones in their care, mostly -- are already in contact with their physician, saying, 'Will you prescribe this drug, or do I have to find someone who will?'"

The Case Western Reserve researchers are heading up an effort to move the drug quickly into human trials. Things should move along at a good clip because the safety profile is relatively well known for this nearly 13-year-old drug.

Patients and their families should hold tight because without drug trials that conform to well-established testing protocols, it will be impossible to know whether a drug originally approved for cutaneous T cell lymphoma will work for Alzeheimer's. Using the drug off label now will be be like ingesting nothing more than a sophisticated dietary supplement. At the same time, drug manufacturers should take notice of the huge pent-up demand and think twice about scrapping their neuro development programs.

 

Image: Wikipedia Commons

 

 

 

 

 

 

Gary Stix, Scientific American's neuroscience and psychology editor, commissions, edits and reports on emerging advances and technologies that have propelled brain science to the forefront of the biological sciences. Developments chronicled in dozens of cover stories, feature articles and news stories, document groundbreaking neuroimaging techniques that reveal what happens in the brain while you are immersed in thought; the arrival of brain implants that alleviate mood disorders like depression; lab-made brains; psychological resilience; meditation; the intricacies of sleep; the new era for psychedelic drugs and artificial intelligence and growing insights leading to an understanding of our conscious selves. Before taking over the neuroscience beat, Stix, as Scientific American's special projects editor, oversaw the magazine's annual single-topic special issues, conceiving of and producing issues on Einstein, Darwin, climate change, nanotechnology and the nature of time. The issue he edited on time won a National Magazine Award. Besides mind and brain coverage, Stix has edited or written cover stories on Wall Street quants, building the world's tallest building, Olympic training methods, molecular electronics, what makes us human and the things you should and should not eat. Stix started a monthly column, Working Knowledge, that gave the reader a peek at the design and function of common technologies, from polygraph machines to Velcro. It eventually became the magazine's Graphic Science column. He also initiated a column on patents and intellectual property and another on the genesis of the ingenious ideas underlying new technologies in fields like electronics and biotechnology. Stix is the author with his wife, Miriam Lacob, of a technology primer called Who Gives a Gigabyte: A Survival Guide to the Technologically Perplexed (John Wiley & Sons, 1999).

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