Potential For An Alzheimers Treatment Appears To Be Closer Than Ever
There may be nothing quite as gut-wrenching as sitting at the bedside of a loved one, watching helplessly as their life comes to an end. Perhaps the only thing more painful is when that loved one no longer recognizes you, slipping away not knowing who it was holding their hand.
Alzheimers is the only disease among the top 10 causes of death in America that cannot be prevented, cured or even slowed. Over a decade and billions of dollars have been spent on research to find a cure for Alzheimers, yet none have thus far proved real clinical benefit. If the elusive cure for Alzheimers isnt found soon, scientists estimate that by 2050 someone in the United States will develop the disease every 33 seconds.
While these statistics paint a pretty bleak picture, Cynthia Lemere, Ph.D., Associate Professor of Neurology at Brigham and Womens Hospital and Harvard Medical School, assured BioSpace that hope is not lost. Despite the many failed drug trials, Lemere says the horizon is finally looking optimistic and expects to see multiple treatments approved within the next few years.
Any therapy developed at this point is going to be probably good for one subsection of that 5.8 million people with Alzheimers disease. And maybe not even that population, but maybe the population that doesnt yet have Alzheimers disease but has the changes going on in their brain of Alzheimers, so we know that theyre on the track, Lemere said.
Dementia Cure Less Than 10 Years Away Claim Experts
A cure for dementia could be less than a decade away, according to experts in the brain-wasting disease. World-renowned neuroscientist, Professor Bart De Strooper, who works at University College London , has spoken out saying that an effective treatment could become available by 2028.
Despite there being no definitive route to finding a way to combat the disease at present, he said that researchers were heading in the right direction. Recent studies have made it clear that the key to overcoming dementia is to target it in the early stages, before irreparable damage has occurred.
Among the strategies that are currently being developed are looking at the genetic causes of the disease and preventing the build-up of plaques in the brain. Recently, scientists revealed that a blood test can now predict dementia ten years before symptoms develop.
Approximately 850,000 people in the UK have been diagnosed with dementia, but the condition has been steadily rising in recent years. And its not just this country in which it is a problem, with America being home to 5.7 million dementia patients and cases prevalent across the world.
Professor De Strooper heads up a team of 270 scientists at the Dementia Research Centre at UCL. Expertise has been gathered from six universities in order to bring together those at the top of their field to try and find a cure.
Preventing And Targeting Plaques And Tangles
As with all diseases, knowing exactly what causes Alzheimers is key to identifying ways to prevent and treat the condition.
Past research has indicated that Alzheimers occurs when two abnormal brain structures plaques and tangles damage and kill nerve cells, causing the memory, thinking and behavioral problems associated with the disease.
Plaques are fragments of a protein called beta-amyloid, which build up in areas between nerve cells. Tangles are twisted fibers of a protein called tau, which accumulate inside brain cells.
Although the jury is still out on the exact roles plaques and tangles play in the development of Alzheimers, studies have suggested that build up of these proteins begins long before symptoms develop.
Evidence suggests that the process of Alzheimers disease begins more than a decade before clinical symptoms appear, suggesting we may need to intervene earlier to have a major impact on the course of the disease, particularly when using therapies designed to prevent the development of abnormal protein structures plaques and tangles that are abundant in the brains of people with Alzheimers, says Snyder.
Other research has suggested that targeting these abnormal structures could treat Alzheimers. Earlier this year, Medical News Today reported on a study by researchers from the University of California-Irvine, suggesting that increasing brain cell connections could reduce plaque accumulation.
Fdas Accelerated Approval Program
Aducanumab was approved through the FDAs Accelerated Approval Program, which provides a path for earlier approval of drugs that treat certain serious conditions. This helps people living with the disease gain earlier access to the treatment. The approval of aducanumab was based on the ability of the drug to reduce amyloid in the brain. When using the accelerated approval pathway, drug companies are required to conduct additional studies to determine whether there is in fact clinical benefit after the drug is approved. If the follow-up trial fails to verify clinical benefit, the FDA may withdraw approval of the drug. Results of the phase 4 clinical trial for aducanumab are expected to be available by early 2030.
Q: How Will Aducanumab Impact The Future Of Alzheimers Care
A: I think its going to herald a new era in Alzheimers therapy. There are now two other monoclonal antibodies for treating Alzheimers lecanemab and donanemab that are showing similar promise in early studies. In the next two to three years, as more clinical-trial results are published, a lot of the controversy and smoke around these drugs should clear. We will have a clearer picture of what these drugs do, who they help, and who they dont help.
Consider the history of treatment for HIV and many cancers. The first drugs that were used to treat these diseases had serious toxicity and modest benefit a lot like aducanumab. Yet we now have highly effective drug cocktails that allow people with HIV to live normal lives and keep the virus under control, and we have extremely sophisticated ways of understanding individual tumors and developing targeted therapies to treat them. With Alzheimers, we have just reached the precipice of this precision approach to treatment, and aducanumab is the first drug, hopefully, of many.
I wish it were a better drug. I wish it were less controversial. But I do think its a step in the right direction.
Disclosures: UCSF served as an enrollment site for the clinical trials of aducanumab. Dr. Rabinovici was not involved in the trials. He has done consulting work unrelated to aducanumab for Eisai Co. Ltd., which has a partnership with Biogen Inc. to develop and commercialize aducanumab and other treatments for Alzheimers disease.
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On the importance of sleep
We know that sleep has hugely beneficial effects for the brain. When you sleep your brain essentially cleans itself it uses cerebral spinal fluid to pump away the plaques and tangles that we think cause the disease. And so lots of research is now looking into ways of using sleep to treat Alzheimer’s disease, and seeing if a certain level of sleep can somehow affect the symptoms or somehow slow the disease down.
On how research into neural stem cells may affect Alzheimer’s treatment
There is a lot of really exciting research coming out now into neural stem cells that suggests that actually there are populations of cells in the brain that may provide regeneration, that may actually give birth to new neurons in the brain. And some scientists think that if we can figure out where these populations of neural stem cells are and if we can figure out what the biochemical and genetic messages are that activate these cells that we can develop a drug to switch those on, essentially, to allow the brain to heal itself. So it’s a very exciting field at the moment, because a lot is happening.
Sam Briger and Mooj Zadie produced and edited the audio of this interview. Bridget Bentz and Molly Seavy-Nesper adapted it for the Web.
Q: What Are The Drugs Side Effects
A: Mainly swelling or bleeding in the brain. This sounds really scary, but the bleeds are tiny what we call microbleeds. In the trials, fewer than 1 percent of people treated with aducanumab had what we would consider very severe symptoms, including seizures or stroke-like episodes. Most participants had no symptoms or only mild ones, like dizziness or a little more confusion.
These side effects are more serious than with other Alzheimers drugs, but they can be managed. They resolve when patients stop taking aducanumab, and in most instances, patients can safely restart the drug later. However, people who are on blood thinners or who otherwise might be prone to bleeding should avoid taking aducanumab altogether.
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Q: Are You Concerned About The High Price Tag
A: Biogen has set the wholesale price for aducanumab at $56,000 a year. Thats too high. Even if the drug is covered by Medicare, patients might still be liable for up to 20 percent in copays. A lot of people wont be able to afford that. There is huge concern that this will exacerbate the disparities that already exist in access to dementia care.
People talk about new dementia drugs like aducanumab bankrupting our health care system. But if we do nothing, we are going to see an explosion in the prevalence of dementia as the population ages. That too will bankrupt the system. I think the solution is for companies to price these drugs more reasonably and offer robust patient-assistance programs so no one is denied treatment because they can’t afford it.
Researchers Around The World Are Working To Develop Effective Treatments For Dementia And Eventually To Find A Cure
Much of this work is focussed on Alzheimers disease, the most common form of dementia.
There is currently no cure for Alzheimers disease. Available medications can reduce symptoms and improve quality of life in some people, but they do not stop the progress of the disease.
The potential treatments discussed below are in the early stages of research and are not currently available. However, they are all part of the research effort to find more effective treatments for Alzheimers disease and ultimately a cure.
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Therapy Manipulates The Immune System To Slow Neurodegeneration Without Triggering Dangerous Inflammation In The Process
A new therapy prompts immune defense cells to swallow misshapen proteins, amyloid beta plaques and tau tangles, whose buildup is known to kill nearby brain cells as part of Alzheimers disease, a new study shows.
Led by researchers at NYU Grossman School of Medicine, the investigation showed that elderly monkeys had up to 59 percent fewer plaque deposits in their brains after treatment with CpG oligodeoxynucleotides , compared with untreated animals. These amyloid beta plaques are protein fragments that clump together and clog the junctions between nerve cells .
Brains of treated animals also had a drop in levels of toxic tau. This nerve fiber protein can destroy neighboring tissue when disease-related changes to its chemical structure cause it to catch on other cells.
Our findings illustrate that this therapy is an effective way of manipulating the immune system to slow neurodegeneration, says Akash Patel, MS, an assistant research scientist in the Center for Cognitive Neurology at NYU Langone Health.
The investigators say the treatment led to cognitive benefits as well. When presented with a series of puzzles, elderly monkeys given the drug performed similarly to young adult animals and much better than those in their age group that had remained untreated. The treated monkeys also learned new puzzle-solving skills faster than their untreated peers.
Smell Test May Help Detect Alzheimers
Theres no cure for Alzheimers, the most common form of dementia, which affects more than 5 million Americans and its projected to soar to 13 million over the next 35 years. A study published earlier this month suggested its a big killer, taking down more than 500,000 Americans every year.
Many people might not want to know they have an incurable disease that will take away their identity. But Carrillo said many will want to know. There are family planning issues for the future, a lot of things to put in place, she said.
Researchers are working on a blood test for Alzheimers, and there are clinical tests that help detect fairly advanced disease. Current biological markers for early disease, including levels of abnormal proteins in spinal fluid, MRI scans of the brain, and brain PET amyloid imaging, are not very accurate, are invasive and can be expensive.
In the face of the growing worldwide Alzheimers disease epidemic, there is a pressing need for simple, less invasive diagnostic tests that will identify the risk of Alzheimers much earlier in the disease process, said the Alzheimers Associations Heather Snyder.
We want to get people on those treatments as soon as possible, while they are still useful.
Alzheimers is marked by the accumulation of beta-amyloid “plaques in the brain. Early study has suggested these proteins also can build up in the eye, perhaps traveling along the optic nerve from the brain.
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Q: Does That Mean The Amyloid Hypothesis Is Completely Wrong
The amyloid hypothesis is that amyloid is the trigger of everything in Alzheimers. That seems now to be wrong.
New studies from the past decade tell us that amyloid is part of the story of Alzheimers disease, but its the smoke, not the fire. Weve learned that the single-gene and more common, complex forms of Alzheimers are not identical, though they do overlap.
Theres been a lot of backlash against the amyloid hypothesis lately, but in the 90s, it was the right idea. The pharmaceutical industry was right to jump on the amyloid bandwagon. And theyre now right to give it up, I think.
A Clue To A Cure For Alzheimers Disease
- By Andrew E. Budson, MD, Contributor
Are you worried about Alzheimers disease? Does one of your parents or siblings have the disease? If so, your risks are between two and four times that of the general public. What about people without a family history of the disease? Unfortunately, everyone is at risk for it. By age 85, half of you reading this article today will have developed Alzheimers disease, with or without a family history.
Sounds pretty scary, doesnt it?
Im writing today to give you some good news. A new study from the lab of Harvard researcher Yakeel Quiroz, PhD, has suggested a new target for drugs that might have the potential to slow down or even stop Alzheimers disease in its tracks.
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Q: Who Is Likely To Benefit
A: Patients who are in an early stage of Alzheimers whats called mild cognitive impairment. Their memory is not as good as we expect for their age, but they are still able to function independently. Based on what we know about the disease, it is unlikely that people who are in more advanced stages of Alzheimers are going to benefit.
Physicians also should verify that patients have amyloid plaques before starting this treatment. Its not enough to have the clinical symptoms you want to have evidence of the molecular changes in the brain that define Alzheimers disease.
Stem Cells And Dementia
Stem cells are “building block” cells. They can develop into many different cell types, including brain or nerve cells.
Scientists have taken skin cells from people with certain types of dementia, such as Alzheimer’s disease, and “reprogrammed” them into stem cells in the lab. They’ve then triggered these stem cells to become brain cells.
These brain cells can also be used to test potential treatments at a very early stage.
Will We Ever Cure Alzheimers
Few drugs have been approved for treatment of this dementia, and none works very well. It has become one of the most intractable problems in medicine.
By Pam Belluck
Its a rare person in America who doesnt know of someone with Alzheimers disease. The most common type of dementia, it afflicts about 44 million people worldwide, including 5.5 million in the United States.
Experts predict those numbers could triple by 2050 as the older population increases. So why is there still no effective treatment for it, and no proven way to prevent or delay its effects?
Why is there still no comprehensive understanding of what causes the disease or who is destined to develop it?
The answer, you could say, is: Its complicated. And that is certainly part of it.
For nearly two decades, researchers, funding agencies and clinical trials have largely focused on one strategy: trying to clear the brain of the clumps of beta amyloid protein that form the plaques integrally linked to the disease.
But while some drugs have reduced the accumulation of amyloid, none have yet succeeded in stopping or reversing dementia. And amyloid doesnt explain everything about Alzheimers not everyone with amyloid plaques has the disease.
Not all trials have targeted amyloid. Some have focused on tau, a protein that, in Alzheimers, forms threads that stick together in tangles inside neurons, sandbagging their communications with one another.
New Alzheimers Disease Treatment Approved
‘cure’ Could Take Many Forms
As varied as the research pipeline is, most experts agree on one thing: When it comes to finding a way to stop, slow or prevent dementia, it wont boil down to one drug treatment or even one drug target. Rather, it will be a combination approach, perhaps involving drugs that clear the amyloid plaques, knock out the tau tangles, target problem proteins and improve the synaptic health of the nerve cells in the brain.
Patients may also receive nonpharmacological prescriptions from their doctors. Some of the most recent research has shown that cardiovascular health and cerebral vascular health play a critical role in overall brain health throughout ones lifetime. Exercise, diet and sleep have all been shown to reduce risk of cognitive decline in adults. Whats more, a landmark study in 2018 showed that intensive blood pressure control significantly lowered the chances that participants developed mild cognitive impairment.
The mishmash of therapies likely wont cure dementia, but as Rafii explains, we have very few cures in medicine. He and others in the field, including the DDFs Grant, are optimistic, however, that the ongoing advancements will lead to treatments that can delay the disease and improve the lives of millions.
What Im seeing is great progress in the building blocks, the foundation of new future therapeutic approaches, Grant says.
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