The enthusiasm generated for a mouse study stems from the desperation for new ideas as the number of Alzheimer's cases, now at 5.4 million in the U.S., is expected to more than double by the year 2050 as the nation's demographic profile continues to gray. A better understanding of the disease process—the knowledge that pathology begins 10 or 20 years before the first symptom—has shifted focus toward earlier drug trials. New technologies that combine brain imaging and spinal fluid tests might identify at-risk patients and test new drugs. A relatively inexpensive drug that can be ingested orally, such as bexarotene, could then be prescribed to at-risk but symptom-free patients, who would take them over the course of their lifetimes, like a cholesterol-lowering drug.
As ReXceptor moves forward with its clinical trial plans, it will inevitably have to contend with the demands of the families of Alzheimer's patients. Landreth emphasizes that calling your physician after reading an article like this one is a bad idea. "Don't try this at home," he cautions, "because we don't know we 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 would work at a middle or advanced stage of the disease, when neurodegenerative processes have already set in.
Bexarotene's genesis as an Alzheimer's treatment comes as an outgrowth of Landreth's long-time fundamental work on cell receptors. If it succeeds, it will demonstrate that new ideas for treating this seemingly intractable disease may come from beyond the sometimes narrowly focused strategies of large pharmaceutical companies.
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Add CommentWOW!! If this pans out this is big news.
Reply | Report Abuse | Link to thisWith 5.4 million people currently suffering from this horrible disease, sounds to me like they should move immediately to human trials.
If the drug is 13 years old, does that mean it will only be under patent for another 12, even if a new use is found for it? If so, that's potentially doubly-good news. The fact that it's already a well-tested drug should speed up the approval of human trials for Alzheimer's, or at least one would hope.
Reply | Report Abuse | Link to thisA quick correction - with ApoE, the allele of interest is ε4 (epsilon 4), not e4.
Reply | Report Abuse | Link to thisOf greater interest is whether amyloid β is actually the driver of AD pathology in humans. Even if having a lot of amyloid sludge in the brain is impairing memory in these mice, there is a healthy debate as to whether it is the driving force in the decades-long depletion of neurons which occurs in human AD. None of the mouse models are universally accepted as close analogies of the human disease - for starters, you have to load mice up with multiple genetic defects, each one of which triggers full-blown AD in humans, before they start to show any kinds of problems at all. Responding to the first comment, that's why they don't jump from a drug which affects a genetically damaged mouse, to trials on humans suffering a slow, subtle degeneration whose cause and mechanism are very poorly understood after decades of intensive study.
Sorry - looks like the Sci Am website can't handle standard unicode characters - please imagine epsilon and beta in place of those marks in the previous comment.
Reply | Report Abuse | Link to thisEven with it's uncertainties, this is still very promising. See what can be done when one scientist shares information with another scientist?
Reply | Report Abuse | Link to thisIts approved use if for T-cell lymphoma which has a fairly low incidence. Apparently some treatments involve topical application. I'd be very worried about off-label use presuming that the drug is "well studied" in humans. In other reports, I've seen bexarotene characterized as an "orphan drug."
Reply | Report Abuse | Link to thisIn reply to Bruce Byrne:
Reply | Report Abuse | Link to thisYou 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?"
Done deal. We'll know in a few weeks whether this works in humans or not. Predictably, there will be a lot of palaver about whether this is the right way to do science or medicine, but it won't matter. Anyone familiar with human desperation knows that, as the drug is available for purchase, no force on the planet will prevent its massive "unapproved" use.
Better bet: find the companies offering the drug for sale, and invest in them. Or start such a company yourself. Buy the stuff in bulk -- Chinese suppliers can be found on the internet -- and start "dealing" in the black, grey, and/or "white" market.
There'll be no external force stopping this flood. If the drug turns out to be ineffective, it will stop on its own. If effective, the medical community will quickly get on board. In reply to Bruce Byrne:
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?"
Done deal. We'll know in a few weeks whether this works in humans or not. Predictably, there will be a lot of palaver about whether this is the right way to do science or medicine, but it won't matter. Anyone familiar with human desperation knows that, as the drug is available for purchase, no force on the planet will prevent its massive "unapproved" use.
Better bet: find the companies offering the drug for sale, and invest in them. Or start such a company yourself. Buy the stuff in bulk -- Chinese suppliers can be found on the internet -- and start "dealing" in the black, grey, and/or "white" market.
There'll be no external force stopping this flood. If the drug turns out to be ineffective, it will stop on its own. If effective, the medical community will quickly get on board.
Undoubtedly medical tourism will soar as offshore clinics use this information to market to Alzheimer patients. Perhaps our testing regimen is too restrictive for those patients who are willing to accept the risks.
Reply | Report Abuse | Link to thisWhy not create a category of patient that waives ALL legal recourse?
ditto
Reply | Report Abuse | Link to thisTruly this disease is such a nightmare for us.Will other cancer drugs bring similar effects to the amyloid?Does it help in all kinds of Alzheimer's diseases with different causes?Anyways,good news though.
Reply | Report Abuse | Link to thisGiven the normal course of Alzheimer's I'm sure patients and carers/relatives would be willing to take a risk of toxix side effects from small explaoratory doses.
Reply | Report Abuse | Link to thisParkinson's disease is beleived to have a similar mechanism to Alzheimer's and mice with induced PD are widley used to test therapies. Has anyone tried bexarotene on them?
A subsatnce used to prebent amhylloidosis in arthriris patients is ATNF (Anti Tumour Necrosis Factor) but any frug to be used for neurological diseases has to pass the blood brain barrier and I'm not sure that ATNF would.
Apologies for the typos in my comment. I have PD and my typing, like my speech and my writing, is too fast.
Reply | Report Abuse | Link to thisYet even more good news for mice. The catalogue of 'complete complete breakthroughs' are filled with so many epic success stories its hard to believe rodents still die.
Reply | Report Abuse | Link to thisThose researchers with brains the size of mice or at a fairly advanced stage of Alzheimers will naturally say this is yet another complete breakthrough for anthropoids , and invariably there are plenty of people with more fear than sense. We do no need any more ineffective agents on this or any other market for the present thanks.
"Why not create a category of patient that waives ALL legal recourse?"
Reply | Report Abuse | Link to thisYou don't think there might be competence and consent issues in a population of patients with dementia?
Bexarorene/Targetin has been used for treating skin cancer for more than 10 years. Skins cancers are very common in older people so some of these patients must have some degree of dementia/Alzheimers disease too. Not that this is very scientific, but did anyone notice any improvment in their mental abilities? Pat
Reply | Report Abuse | Link to thisAmong the billions of people inhabiting earth, some must have both t-cell lymphoma and AD. Find them and test them?
Reply | Report Abuse | Link to thisAmong the billions of people inhabiting earth, some must have both t-cell lymphoma and AD. Find them and test them?
Reply | Report Abuse | Link to thisPithycus Among the billions of people inhabiting earth, some must have both AD and t-cell lymphoma. Find them and test them?
Reply | Report Abuse | Link to thisAmong the billions of people inhabiting earth, some must have both AD and t-cell lymphoma. Find them and test them?
Reply | Report Abuse | Link to thisPithycus
Among the billions of people inhabiting earth, some must have both AD and t-cell lymphoma. Find them and test them?
Reply | Report Abuse | Link to thisI’m not sure where you are getting your info, but good topic. I needs to spend some time learning much more or understanding more, read this : http://strepthroatsymptomsinfo.com/
Reply | Report Abuse | Link to thisI have read many positive results about the <a href="http://www.buy-bexarotene-online.com/" rel="nofollow">drug</a>. But the clinical trials for human are still on going and we will have to wait until 2020 for the drug to be approved by the FDA.
Reply | Report Abuse | Link to thisBexarotene has a great potential to be a treatment for AD.The progression of the studies is like a glue to a broken hope. It was also found out that bexarotene kills the first sign of AD and that is loss of sense of smell. It is also proven that it can improves memory deficits and behavior and acted to reverse the symptoms of Alzheimer’s. For more click <a href="http://www.buy-bexarotene-online.com/">here</a>
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