A cancer drug has succeeded in reversing Alzheimer's disease in its early stages in mice, according to a new study.
The drug, bexarotene, is designed to reduce levels of amyloid beta, the protein whose presence in the brain has been most closely tied to the development of Alzheimer's.
In a new study, mice treated with bexarotene saw their amyloid beta levels drop 25 percent within six hours and, importantly, they showed a corresponding improvement in their cognitive function.
"The data we provide here really suggest that Alzheimer's could be, in the early stages, a reversible disease," said study author Paige Cramer, a doctoral student in neuroscience at Case Western Reserve School of Medicine.
The researchers used mice that had a mouse model of Alzheimer's disease. After the researchers administered varying doses of bexarotene, they measured levels of amyloid beta in the brain and tested the mice for their abilities in maze running, nest building, smell, and fear conditioning, which is a type of learning.
"They did a lot of different tests of learning and memory and they saw an effect on every single one of them," said Michael Sasner, a research scientist and associate director at the Jackson Laboratory in Bar Harbor, Maine.
Bexarotene is already approved by the Food and Drug Administration for the treatment of cutaneous T-cell lymphoma, a type of skin cancer, and so it may be able to proceed through clinical trials more quickly than drugs not already known to be safe to administer to people.
The study appears in the Feb.10 issue of the journal Science.
A new way to target Alzheimer's
Sasner, who was not involved with the new study, said it overcomes some of the weaknesses of previous Alzheimer's work, in which only one or two tests of cognitive improvement were conducted.
Bexarotene is not the first attempted Alzheimer's treatment to target amyloid beta. But past research has aimed at removing the plaques that amyloid beta can form in the brain, which has not shown any effect on the disease itself.
The difference now, researchers say, is in a better understanding of amyloid beta and the various forms it can exist in. Rather than focusing on the plaques, researchers now think it is the active, soluble form of the protein that is at work in Alzheimer's.
While there are various views on the causes of Alzheimer's, "the predominant view right now is that it's the soluble forms of amyloid beta that are causing the impaired brain function," Cramer said. "Plaques are just sinks, just tombstones that gather amyloid beta."
Bexarotene works by promoting the production of another protein, called Apolipoprotein E, which binds to and clears amyloid beta from the brain.
"This paper lends a lot to the mechanism of how ApoE may be involved in Alzheimer's," Cramer said.
Hurdles remain
It remains to be seen whether the benefits of bexarotene in mice would translate to humans.
"Because we're using an FDA-approved drug, this allows us to translate these basic science findings to the clinic; that's our next goal," Cramer said.
Figuring out the correct dosing presents another challenge. Researchers found that in one case, giving bexarotene over several doses appeared to be less effective than giving it once. Cramer said the reason may be that the drug degrades itself within the body.
Cramer said she and Gary Landreth, the senior investigator on the study, hope to begin a preliminary trial this year, in which they will look for the same changes in beta amyloid levels in humans. If successful, the testing would move to clinical trials.
Bexarotene is currently sold as Targretin; patents on that drug will expire in April.
"There's a long way to go to prove this treatment in humans, but it seems like an exciting thing to follow up on," Sasner said.


Just what we need, rats with good memories.
You're just jealous you weren't born a laboratory rat.......Pinky!
"So, what are we going to do today Brain?"
"Same thing we do every day Pinky, plan to take over the world."
...now they'll remember where they moved their cheese?
I think this is a great breakthrough in Ratology. Now when these rats make it through the maze they will remember what they went in there for in the first place.
It isn't so much mice or rats with good memory, its the longevity for the good brain functions. (Flowers for Algernon comes to mind.) What I worry about the most is, the Pharma's will try to can the product so that people will be forced to use the medicine on a prolonged period. We need logical people making products that finish the job and cure the issues; not make us dependent indefinitly. I don't care if the Pharma's whine about their profits, I care that people are done being sick and are able to move on with their real lives (outside of drug dependency).
LONG LIVE THE BRAIN !!!
This is great news! Alzheimer's runs in my family so I'm happy for any progress.
LMAO! That's some funny stuff, guys!
Is that what you got out of this, Roger? Clearly, you've never had anyone you love unable to recognize you anymore. This is the worst disease EVER. It's worse than losing someone to death. At least with death, you have your good memories of your loved one intact. Alzheimer's leaves you the empty but living shell of someone you once loved who once loved you.
He's joking, Tracy. Jeesh lighten up. Four of my relatives died from it and I may too.
Doesn't mean I'm going to freak out and be all glum about it. It's funny. Get a life.
Toosano, you would appear to be the only one laughing.
Sounds like a promising line of research.
But when I read that the mice "showed a corresponding improvement in their cognitive function," I couldn't help but have an image of Stuart Little suddenly remembering where he put his car keys.
What does a rat really need to remember? And how did they test memory in rats? Quiz them on current evemts? Hmmm... now what was I talking about??
Seriously?! As if this is really about rats. Geez.
My sister has a close friend who has spent her career in medical research. I asked her once why new therapies, drugs etc. were always being conducted on mice, especially since, when improvement is noted, there is always a caution to people not to expect any improvement for them, since mice and people are so different. Basically, and in a very rambling way, She said, since the mice were expendable, they were used to either prove or disprove the value or error of the direction of the particular research. That was some 30 years ago. Still makes little sense to me. I continue to wonder if research using mice will ever yield any real value for humans. Course, It could be I'm just stupid. Actually, I hope that is the reason for my skepticism.
As part of a family in which alzheimers and dementia is a genetic trait, and currently watching my grandmother go through it, I can honestly say that this gives so many of us out there hope, when there has so far been none. This disease not only affects the person with it, but has a domino effect on everyone around him or her as well. Treatment such as this would be such a gift, and a true lifesaver. I hope and pray that something miraculous happens in other animal and human testing, and that it isn't simply contained in rats.
Thank you for writing so eloquently.
really this isn't a joking matter.
I agree, very heart-lifting post!
I can't agree with you more COmommy. This would be an incredible prayer answered if it does in fact work on people. It is one of the cruelest of illnesses.
When I was in nursing school in 1992, my microbiology teacher told us once that "someday, AIDS will be a chronic condition like diabetes, and Alzheimer's will be cured, in our lifetime." I keep holding on to that, and we need to realize that AIDS now IS just a chronic disease, you have to take medicine, you have to watch your health, but nobody that can get the proper medication dies of AIDS.
I had heard that the only problem with a cure for Alzheimers is the fact that just giving an injection doesn't necessarily get the medicine past the brain/blood barrier. but they were working on hooking Alzheimer's medicine with a rabies vaccine which will carry it across the barrier. they're working on it. I can imagine it will be curable within the next 10 years. Too late, for some. but a great work!
Great post, been where you are, it is so difficult.
My father had a 13 year decline before he died of Alzheimers. At the end he was bedridden, unable to talk or even swallow. There is not one dam thing funny about it.
I would not wish Alzheimer's on my worst enemy. Perhaps there is now at least some hope for those with the disease and, as importantly, the families of those with the disease.
My mother and my aunt both went though this horrifying disease. My aunt died 15 years ago. My mother died from complications of Alzheimer's last September. COmommy, you are 100 percent correct that this is the type of disease that everyone in the family suffers from even if only one person physically has it. The toll on everyone is very great. Today I have to wonder every time I forget where my car keys are or where I parked my car or a word that maybe I am seeing the beginning symptoms of this dreadful disease. I can't bare the thought of putting my family through it again.
It gladdens my heart to hope that because of this drug or maybe one like it, other's will not have to experience what our family has had to go through. Like Abraham, I couldn't possibly wish it on my worst enemy either.
Chris Rock, said it best, theres no money in the cure. Regardless if it does work, the FDA will now pull its approval for some bizarre reason of this medicne, and people that need help from this debilitating disease wil continue to suffer while pharmaceutical companies continue to profit by allowing doctors to perscribe only current medications that do not work on patients.
We constantly see stories like these about cures for cancer, alzheimers, aids, and other horrible diseases and these cures show so much success in other countires and labs throughout our country but these treatments never get fda approval.... Again Chris Rock said it best, theres no money in the cure we haven't cured anything since polio.
So sad poeple are this ignorant.
Have an example? Cures in the National Inquirer don't count.
Roger you funny!
Look at the side effects from FDA approved drugs; death, suicide, liver failure, etc.....and then google up cannabis/Alzheimers........Father Knows Best!
You gotta wonder what the feds problem with pot really is seeing that it also has cancer inhibiting properties too. That combined with the fact it is safer than alcohol, tobacco, and almost ALL other legal and prescription drugs.
Tylenol is the leading cause of liver failure in the US but pot is illegal and research is discouraged even though it is impossible to overdose on pot and it has many benefits.
This looks promising.
We'll never see it, no profit for the medical establishment for CURES, they only treat the symptoms, never the cause. A cure would imply no need for further medication, they certainly don't want that!
You're confusing medical establishment with pharmaceutical companies. Many hospitals might fall into that money grabbing stereotype, too. But colleges, on the other hand, aren't so greedy. Students and professors work towards cures. After all, the prestige of a major cure would boost a college's standing, not to mention the patent would make the college a tidy sum as well. As the recent headlines have shown, colleges work hard to raise their names and stand out. That's what increases their bottom lines.
As if a cure can be hidden. Such BS.
One of the researchers for MS, whom I met at a board meeting, has MS himself, and so wants to find a treatment for it.
(Another doctor told me, "Hams are cured. People are healed.)
This is fantastic news, I sure hope it pans out. Alzheimer's is a terrible disease that steals a persons mind, slowly. There's nothing worse. Its terribly hard on the victim, and equally hard on their family. Lets hope it end's up being the cure to this terrible affliction
So how could they tell? Did the rats remember where they left their cheese the day before?
I've talked to a lot of rats in my day but I couldn't
tell if it was alzheimers or they were just pulling my leg
I had a clever comment, but I can't remember what it was.
This is exciting news!!
Mice are going to take over the world? How fitting...
Obviously most of the losers posting on this site have NOT dealt with Alzheimer's.
While I wouldn't wish it upon anyone, I know it will hit some of these losers or their families at some point.
Then it won't be so funny.
Okay, so this new anti-Alzheimer's drug works on mice. I'm sure Mickey and Minnie Mouse will be thrilled to know that. But will it work on people? That's the big question and, yes, I'm serious about it.
Well, they'll test it on other animals before clinical trials in humans, but they wanted to make sure that 1) it had effect, though not the one they were looking for (e.g. anti-cancer), 2) it didn't outright kill the mice due to acute toxicity, and 3) the associated biochemistry could be measured.
All-in-all, I'd say it's a promising start, but I'm with you -- we should wait and see what it does in humans, because reversing Alzheimer's in mice is one thing -- getting the same effect in humans without killing them is another.
Matt, the good news is this is a cancer drug. Its been used on people before. And the difference in plaques in the brain of rats and the plaques in the brain of humans should be the same. it should work. Fingers crossed!!
You will be surprised to learn how close human and mouse DNA is. 90% the same.
Dog DNA is 95% the same and Chimp DNA is 97% the same.
FOr comparison, 50% of human DNA is the same as a Banana and a fruit fly.
Oh gee ScoMata, don't confuse idiots (particularly idiots who would joke about a potential breakthrough for a heartwrenching disease) with facts!
I am a caretaker for my Mother who got Alzheimer's around age 70. It has been 14 years now and she is still with us in our home (for the past 10 yrs). I worked full-time all my life until about 7 yrs. ago and then worked part-time for 2 yrs. Now she needs full-time care so that is what I do. I am exhausted, both mentally and physically. Can't tell you how much this new research finding means to me. Both my Sister and I are both worried about getting the disease because my Grandmother (Mom's ) Mother had it in her 80's. They say it is "the long goodbye" and it truly is as Mom does not know who I am anymore and hasn't for a couple years.
I'm sorry to hear this, Moco. Take care of yourself because caregiving someone with any kind of dementia is exhausting. You need regular breaks, just to get out of the house and relax. If you don't take care of you, you can't take care of Mom. Your Mother knows what you have done for her. Trust me. I've worked in long term care for over 20 years. And while she may not know its "you", she knows deep down inside. And I can't tell you how many times in that 20 years that an Alzheimer's patient, as their mind is slowing down as they begin the dying process, that for 1-2 minutes, they are totally cognizant of what is around them. and they will turn and see their family sitting there, and they realize all that dreaming was real. so your Mom will know. Take care of you. its a hard job.
Remember to care for yourself as well as your mother. After my sister-in-law passed away while caring for her Mother we were told that care-givers often pre-decease those they are taking care of.
I never really cared a lot for my mother-in-law, but after watching her die of alzheimers, I wouldn't wish that terrible disease on anyone.
Take Charlse advice, my aunt almost died while ignoring her own health while caring for her mother.
This is heartbreaking. I am so lucky my mom still recognizes me, but went through the disease with my stepdad who for several years did not. Hope that doesn't happen with my mom!
I know exactly how you feel. My mother, grandmother and three of her siblings all came down with Alzheimer's. It's awful to watch how it affects people. My mother is in the last stages and it breaks my heart to see her bedridden, not able to communicate, and unable to feed herself. There is no joy at all left in her life. My sister and I, also, are frightened of what there is in store for us in the next 10 years. What a horrible, horrible disease.
Yeah, all theses jokesters with the not funny comments. Take a walk in a family caretakers shoes for a while and I don't think you will find any laughter on this subject matter.
I am legend anyone??? I'll hold my applause until the long term side effects of such treatments are known...
The drug is already being used, it just isn't used for Alzheimer's.
I am not sure there is any side effect that could possibly be worse than this disease. Frankly a fatal side effect wouldn't be any worse since this disease is fatal anyway.
One of my friends said that if he was ever diagnosed with Alzheimer's, that he'd take up sky-diving.
If I'm ever diagnosed with it I thought I might take up hiking but skydiving works too.
Well my main concern is if it cures Alzheimers, then wonderful, but what good will it do if it causes organ failure or whatnot. I'm not sure what else it's being used to treat, I'd have to look into it.
@ kayla - I understand your concern, but Alzheimer's is already fatal. If there is a slight chance of a side effect that kills you, is that really worse than the disease that will?
I'm rather curious as to whether or not anyone who has received Bexarotene as a cancer treatment has had Alzheimer's disease. It would indicate the efficacy in humans quite well. A case study should be done in such a context to support the findings in the mice.
Interesting point, too bad reporters don't think of and ask those kinds of questions anymore.
Hey, researchers, here's a thought: Go around the country and check on all the seniors who are currently taking this cancer drug to find out if any suffer from alzheimers. If you don't find any, then it must work in humans (I know, bit of a stretch, but that's why its called research). Might speed up your process.
They might be, but like said above, with the quality of reporting these daysyou cant assume they are not just because this reporter did not think to ask such an obvious and interesting question.
Reporter probably didn't get to ask any questions. A lot of articles are recycled press releases.
Sounds like a good idea until you check the side effects of this drug, bexarotene.
I think I'd rather get alzheimers than suffer the severe side effects from this toxic chemical compound.
You have never experienced the effects of Alzheimers or you would not say that.
Foreigner...you have obviously never knows anyone with Alzheimers or you would not say that!
I took the liberty of researching this further. The drug bexarotene or Targretin is a retinoid. A form of vitamin A. Look it up and follow the links to retinoid. Start drinking carrot juice, eating fish, eggs, and cod liver oil. I would go to the doctor and get blood work for D3 deficiency, any vitamin deficiency, mineral deficiency, and hormonal levels. This drug may help in one instance, but will give you other side effects. The reason why drugs are made is because herbs, plants, and flowers cannot be patented. So, in order to make money, the chemical is extracted, and mixed with other chemicals to make it a product that can be patented. However, nature has provided food that works biologically with your body.
"The reason why drugs are made is because herbs, plants, and flowers cannot be patented." In general, this is not true, not only from the stand point that processes, even if not plants, can be patented. Drugs derived from natural sources are not exceptional... you can google polyketides for one very widely used example.
While I agree with the general statement that good nutrition is commonly our best defense against disease, it is not always the best, nor the only. Any extreme position, "all drugs are bad" or "eat bananas and you'll never need to take antibiotics" are almost always wrong. We can jump to quickly to the drug counter; sure... We need to inprove our diets; no doubt! But taking the approach that someone with cancer or other life threatning diseases can be cured with raisins is often a dangerous proposition.
"A form of vitamin A"
Well unless you can name some veggies with the exact same chemical structure of bexarotene, then there is no guarantee that they will have any similar effect just because they have a similar substance. A single trans bond instead of a cis bond is what makes trans fatty acids so much more deadly than unsaturated fats, even though they may have the same chemical formula. A very small change in a molecule can lead to a very big difference in activity. There are even some chemicals that a single change in bonding or an atom or two can cause a beneficial substance to become deadly toxic.
Don't give advice you don't have.
That's not to say that eating healthy is a bad idea. An ounce of prevention is worth a pound of cure... but you might end up needing that cure anyway. Good thing we have consistent, effective drugs. Now if only we could do away with the greedy pharmaceutical companies that extort the good people so...
Oh, and go look up Taxol while you're at it. Lovely little drug from a humble little tree... but to meet the needed supply would require the destruction of the entire species... not feasible. Good thing we have these things called chemists that learned how to synthesize it.