E-mail updates
Follow on Twitter
Subscribe to RSS
Robert Bazell, NBC News
A cancer drug is turning out not to be the miraculous treatment for Alzheimer’s that many had hoped. Two papers out Wednesday in the New England Journal of Medicine warn families of Alzheimer’s victims not to seek treatment with Targretin (generic name: bexarotene).
Last February a study from Case Western Reserve University reported that the drug rapidly cleared the clumps of protein known as beta-amyloid, the hallmark of Alzheimer’s, from the brains of mice with a version of the disease. Since the drug was already on the market, approved as a treatment for lymphoma, doctors could immediately prescribe it in so-called off-label use for Alzheimer’s. And thousands of families understandably asked.
But one mouse study does not prove that a drug is effective in humans. The drug is expensive – about $14,000 a year - and off-label use is often not covered by insurance. The drug can also bring on severe side-effects. In one paper in the Journal, Justin Lowenthal, Sara Hull and Steven Pearson of the National Institutes of Health and Massachusetts General Hospital conclude that for this drug “even if the patients are willing to take the risks for the potential benefit, the physician's answer should be no.” In the second paper Frank LaFerla of the University of California, Irvine observes “the field has been down this road before, as successes in preclinical models have thus far not translated well into the clinic.”
But the reasons for disappointment with Targretin appear to be stronger than these theoretical concerns. Word among Alzheimer’s scientists is that no one yet has independently replicated the Case Western results in mice. In science, a single study has little value until it is repeated. It could be just a fluke or a mistake. No funding agency would support a clinical trial in humans of a potential drug unless the mouse study were confirmed.
These are not good times for potential Alzheimer’s treatments. On Monday, Pfizer announced it was abandoning its efforts to market bapineuzumab, a drug that definitely cleared amyloid plaques in people, after a second, large clinical trial found it did not make the patients noticeably better.
The reason for this failure -- and other recent similar ones -- could be new evidence that the brain changes leading to Alzheimer’s begin decades before symptoms appear. Treatment may have to start long before there is perceptible loss of memory or other mental function. "The impending tsunami of new cases and the time required for introducing a drug to the market increase the urgency of identifying new therapeutic targets and strategies," LaFerla writes. That presents a daunting challenge, but science cannot give up trying to prevent the misery and expense from Alzheimer’s that is beginning to overwhelm us and will only get worse as the population ages.
Robert Bazell is NBC's chief science and medical correspondent. Follow him on Facebook and on Twitter @RobertBazellNBC
Related:
Leave a Comment:
Does this society truly want a prevention or cure? If so, then research should verify what is going on with chronic low level RF exposures from the wireless explosion. Initial studies show damage to blood-brain barrier from exposures LOWER than cell phone emissions. Big pharma would not benefit, big wireless would be angry. But what is more important? Who will pay for these studies? Do we want the truth or to keep throwing drug "bandaids" at this scourge?
Good thing you wear your tinfoil hat, huh?
Although anecdotal at best my father suffers from Alzheimers and has never used a cell phone, has no wireless phones or wireless internet in their home, and has declined quite recently. His father had it and I fear I too will have to grapple with it in the future.
The RF connection is not really a viable option for many aged folks that succumb to this never have used wireless devices in their life.
I don't have the answers and fear a future of memory loss myself but looking for ghosts in the closet is not going to address the disease.
Hello, and thanks for taking the time to follow up on false hopes drug companies are so good at building in the minds and hearts of people living with dementia. As more and more drugs fail that are based on the amyloid theory it doesn't seem to impact the true believers in the theory - get rid of the amyloid, get rid of amyloid. Now some true believers have decided we just have to try harder, earlier.
Lost in these press releases is the fact that Alzheimer's Disease is but one of many forms of dementia, and curing it will not dry up the tusnami of folks living with the symptoms of dementia, with millions more on the way as the baby boomers pass 65 years old.
The world of Alzheimer's hopes and fears has been created by people who should know better, but in order to promote their own self serving agenda's insist on scareing us (you're gonna die, you are fading away, you are losing your mind) into donating for cure researcher or asking the federal government to spend more money on cure research. If they really wanted to reduce the negative impacts of the disabilities associated with forgetting and confusion they would reverse their spending priorities and spend most of their research dollars on psycho/social research into the every day issues facing people with dementia and their loved ones, and what is left over on trying to cure something the cause of which no has a clue.
Richard Taylor (living with the disability, symptoms, and diagnosis of dementia, probably of the Alzheimer's type)
all i have to say is that they don't really care about us
My wife has had dementia for the last 17 years........I have been giving her 4000 mcg of sublingual vitamin B 12....methylcobalamin type of B 12....note, there are two types....use only the methylamamin type..........also, she receives 4ooo IU of vitamin D and a very good multi-vitamin...........she is now off all drugs including Aricept. After looking after her now for all these years, I firmly believe the vitamin combination that she receives is doing as well as any drug combination could..............seniors get so overdrugged, is really a crime
Google: Alzheimer Curcumin
TY Malcolm for the info. My prayers for all those that posted that have loved ones or is suffering from Alzheimer. I do believe a more natural approach seems better (with a good support network) From what I've learned of the disease, it is as upsetting for those who have to "watch" as it is for those who suffer it.
I do agree that the drug companies "gold rush" for a cure or treatment is just that. The process to get a new drug to market has been more difficult as time's gone on but in light of "theoretical" medicating the process should be scrutinized. Pharmaceuticals is the one area were regulation should not be "lightened" however to bring down drug cost the "exclusive" right not allowing "competitors" to produce "like" meds need to be revised. So many people could live a better quality of life if the medicinal cost could be lowered and Home health (a burgeoning field) standardized.
@ Malcolm West. Indeed. Turmeric and Vitamin D-3. See UCLA study from March this year.
In my opinion this a terrible article... "A cancer drug is turning out not to be the miraculous treatment for Alzheimer’s that many had hoped."? The recent articles in the NEJM simply repeat what the original study authors stated repeatedly in every piece of press that the article received in February - The first study testing Targretin was performed in mice, needs to be repeated, and off label use is STRONGLY discouraged until a clinical trial is completed. These studies take time and to imply that the original study is a potential "fluke or mistake" because it hasn't been repeated and published using mice that have to be aged 6 months is irresponsible and misleading. No one knows if this drug will work in human patients with Alzheimer's disease. It's true that many such promising treatments fail in clinical development, but to suggest that this drug has failed before it has been tested is poor journalism.
Sarah, you are SO EXACTLY RIGHT and thank you so much for making such an important point. Sometimes journalists write a story just to write something, and don't take the time to really find out the facts. I'm glad you wrote what you did and cannot wait until baxarotene is tested in humans for the purpose of helping Alzheimer's patients and JUST PRAY SO MUCH the results are positive.