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

The views expressed are those of the author and are not necessarily those of Scientific American.

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PET image of an Alzheimer's brain

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.

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 About the Author: Gary Stix, a senior editor, commissions, writes, and edits features, news articles and Web blogs for SCIENTIFIC AMERICAN. His area of coverage is neuroscience. He also has frequently been the issue or section editor for special issues or reports on topics ranging from nanotechnology to obesity. He has worked for more than 20 years at SCIENTIFIC AMERICAN, following three years as a science journalist at IEEE Spectrum, the flagship publication for the Institute of Electrical and Electronics Engineers. He has an undergraduate degree in journalism from New York University. With his wife, Miriam Lacob, he wrote a general primer on technology called Who Gives a Gigabyte? Follow on Twitter @@gstix1.

The views expressed are those of the author and are not necessarily those of Scientific American.

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  1. 1. gooner 10:50 am 02/14/2012

    Let me guess, this must be a relatively inexpensive drug and now the drug companies are thinking of a way to increase the price? And I think the people that are suffering would roll the dice to try it. I know my mom would.

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  2. 2. gingerx 12:32 pm 02/14/2012

    Is there anyone who didn’t see this coming?

    These people are in a hopeless, helpless sitation and are desperate. In this situation, who wouldn’t try a hail mary pass over nothing?

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  3. 3. HowardB 1:14 pm 02/14/2012

    gingerx is right … “These people are in a hopeless, helpless sitation and are desperate.”

    They should move speedily to quick human safety trials and if it is found safe, then volunteers should be given the opportunity to test it. There are people where THERE IS NO FUTURE FOR THEM ! there is no other treatment !

    In parallel with this of course there should be more established trials. But this is not a new domestic pain killer folks !

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  4. 4. gmartfin 11:28 am 02/19/2012

    Having had a family member develop Alzheimers recently and watching the horrid decline in day to day faculties to the point that they are now put in a facility I would say give them the drug. There is no cure presently and any drug giving any improvement should be used. As there are established safety protocols for cancer start there.

    I know if I had it I would want the drug regardless of future risk.

    Regardless of anything else this is a situation that should be fast tracked.

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  5. 5. Behaviorquest 12:10 am 01/24/2014

    This is not hype. I have a patient that has a wife with Alzheimer and found the drug in Europe and it is expensive like $2700 a month. In three days here scores increased in 2 weeks she was dramatically improved. This drug is well known an has been around so long it is going off patent. This is about as good as it gets.

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