An Innovative Strategy To Lessen The Plaques Was Also Found In The Study
The oral administration of rapamycin to an Alzheimers disease mouse model induces an increase in beta -amyloid protein plaques, according to researchers from The University of Texas Health Science Center at San Antonio . -amyloid buildup is a hallmark of Alzheimers disease.
Rapamycin is FDA-approved for the treatment of transplant and cancer patients. The drug is also referred to as sirolimus and is sold under the name Rapamune. Publicly available data suggest that the drug might also improve learning and memory in aged mice. According to publicly accessible evidence, the medication may also boost learning and memory in elderly mice. The UT Health San Antonio researchers, on the other hand, discovered that following rapamycin administration, a protein called Trem2 is drastically reduced. Trem2 is found in microglia, immune cells found in the brain and spinal cord.
Trem2 is a receptor located on the surface of the microglia, and it enables these cells to engulf and degrade -amyloid, said study senior author Manzoor Bhat, Ph.D. Loss of Trem2 in microglia impairs the vital function of amyloid degradation, which in turn causes a buildup of -amyloid plaques. Dr. Bhat is professor and chairman of the Department of Cellular and Integrative Physiology at UT Health San Antonio and vice dean for research in the universitys Joe R. and Teresa Lozano Long School of Medicine.
More investigation warranted
Will The Fda Reconvene Its Outside Experts For Upcoming Approvals
A flashpoint in the approval of aducanumab was the FDAs decision to disregard its advisory committees concerns about the antibody.
The agency convened this panel of experts one year ago to discuss Biogens complicated data set. In 2019, the company halted development of aducanumab after interim analyses of two phase III trials showed that the antibody was not helping people. Months later, it reversed course and said it would seek approval on the basis of a fresh analysis of the data that hinted at cognitive benefit.
One panellist said that Biogens statistical interpretation of its data was akin to firing a shotgun at a barn and then painting a target around the bullet holes. Ten panellists voted against approval, and one abstained.
After the FDA approved aducanumab, three members of this committee quit in protest.
High-ranking FDA officials defended their position in prominent journals, and newspapers, but the fallout has continued. Off-the-books meetings between the FDA and Biogen might have enabled the approval, STAT News reported in June. The federal watchdog at the Department of Health and Human Services is now reviewing the steps that led up to the approval, and is due to release a report in 2023. Two congressional committees in the US House of Representatives are also investigating the decision.
When Will Solid Data Be Available On Whether These Therapies Improve Cognition
One of the conditions that came with the FDAs approval of aducanumab was that Biogen run a confirmatory trial ensuring that the antibody actually helps people. The biotech firm has yet to launch that trial, and the FDA gave it nine years to collect the results a long timeline that has contributed to the uproar over the drugs approval.
For Ezekiel Emanuel, a bioethicist at the University of Pennsylvania in Philadelphia, the data need to come sooner. The accelerated approval pathway should be updated to mandate faster collection of high-quality confirmatory data, he wrote in the journal JAMA.
Assuming that the FDA approves donanemab using the same accelerated approval pathway, Lilly, too, will have to confirm the benefit of its antibody. The pharma firm is running a 1,500-patient phase III trial called Trailblazer-Alz2 of donanemab for people in early stages of Alzheimers. Results are due in the first half of 2023 after the antibodys potential approval and might provide the data needed. Another phase III trial is recruiting 3,300 patients at risk of Alzheimers, to test whether earlier use of the antibody delays the onset of dementia. This trial will run until 2027.
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How Does Aducanumab Work
Amyloid is a protein that can build up in the brain and may cause Alzheimers disease. Aducanumab travels to the brain, sticks to amyloid, and removes it.
You may be eligible to participate in clinical research studying aducanumab or other drugs that could fight Alzheimers disease and other types of dementia.
I Am Not Currently A Patient Of The Alzheimer Center Or Related Neurology Or Geriatric Psychiatry Clinics At The Brigham How Can I Be Evaluated At Your Center
The phone number to initiate becoming a patient through our Neurology Patient Access Center at the Brigham is 617-732-8060. Your current doctor will be asked to send a referral. The coordinators in our center will help you schedule an appointment with the most appropriate doctor for your care and assist you through the process of becoming a new patient.
We understand you may have additional questions about the potential of this drug. We will continue to keep you updated as soon as information becomes available, and we thank you for your patience.
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Finding Memory Care Near You
The early challenges posed by dementia-related memory decline can be taken care of at home relatively easy, up until it becomes a struggle to remember daily tasks like eating, or when to take your medication. If unpaid bills and chores begin to pile up, it can pose a health and financial risk that is truly necessary when there are so many community options available now to help aging people live in a healthy and safe environment. For those interested in seeking out dementia-friendly communities with access to medical staff and lifestyle amenities like meals and chore services, visit assistedliving.org to find out more information about memory care facilities near you. If you need assistance paying for memory care or other aging-related expenses, you might qualify for a Medicaid waiver.
Accelerated Approval By The Fda
The FDA announced that it gave accelerated approval to Aduhelm because it expects that the drugs well-demonstrated ability to reduce levels of aggregated amyloid in the brain will yield cognitive clinical benefit to patients. Because Aduhelms phase 3 clinical trials did not achieve these benefits for participants under their original design, the drug did not meet the FDAs usual standards for unconditional approval.
The accelerated approval program is designed to allow drugs to reach the market quickly that have demonstrated that they affect a surrogate biomarker in a way that scientific evidence strongly suggests will lead to clinical benefit to patients, even if the benefit was not achieved during the course of the trials. As an example, in cancer, tumor size reduction may be a surrogate biomarker for improving a cancer patients prognosis. Under accelerated approval, a drugs sponsor, like Biogen for Aducanumab, must run a confirmatory post-approval study demonstrating that patients benefit cognitively from the drug or the FDA can act to remove it from the market. The FDA gave Biogen until 2030 to complete this trial Biogen has announced its intention to act faster but has not provided a timeline.
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The Drug Will Cost $56000 A Year
But will Medicare and other insurers cover it?
Not long after the FDA announced aducanumab’s approval, Biogen put a price on its new therapy: $56,000 a year. While there are plenty of drugs on the market with price tags that tower over this, the decision has drawn criticism from some advocacy groups, including the Alzheimer’s Association, which calls the cost simply unacceptable.
Insurers are still deciding whether to cover aducanumab. Medicare would likely bear the biggest burden, since most of the 6.2 million Americans living with Alzheimer’s disease are 65 and older. If just 500,000 beneficiaries went on the drug, total spending for aducanumab in one year would be nearly $29 billion, a Kaiser Family Foundation analysis found.
That amount far exceeds spending on any other drug covered under Medicare Part B or Part D , the KFF report states. To put this $29 billion amount in context, total Medicare spending for all Part B drugs was $37 billion in 2019, the authors add. If 1 million beneficiaries receive aducanumab, spending will exceed $57 billion a year.
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Who Is A Candidate For Aducanumab
Based upon the clinical trials in which it was studied, the drug can be potentially considered for use in individuals with early-stage Alzheimers disease. This means individuals who have Alzheimers in its mild cognitive impairment or mild dementia stage. Individuals in this early stage of Alzheimers may be able to function normally, or they may require a little bit of help with complicated activities like paying bills, grocery shopping, preparing meals, or balancing their checkbook. Individuals who need help with dressing, bathing, or other basic activities would be beyond the early stage and the drug would not be indicated for them.
Note that not everyone with mild cognitive impairment or mild dementia has Alzheimers disease. There are other causes of these problems as well. To determine if Alzheimers is present, you need to get either an amyloid PET scan or a lumbar puncture to be sure you have the amyloid plaques of Alzheimers. Currently, a lumbar puncture is generally covered by insurance, but an amyloid PET scan is not.
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Other Less Targeted Dementia Medications
There are several types of dementia, and each person will be affected differently by their specific disease as well as the side effects that might be present. While not designed with the intention to treat dementia, many medications are available that treat the often-debilitating effects of dementia. There are many symptoms associated with dementia, as well as associated with the medications taken to stall the progression of dementia, such as pain, nausea, insomnia, or general fatigue.
Its important to go over every possible side effect of medications you are considering taking, and carefully weighing the risks versus the benefits of using that drug. While it may seem like a juggling act, the good news is that there is a wealth of options available to treat any symptoms of dementia or medication-related side effect, so no matter your unique body chemistry, your doctor will be able to help you figure out the best way to improve your overall quality of life while living with dementia.
New Drug For Alzheimers Disease Aduhelm Update
Posted April 8, 2022
***** On April 7, 2022, the Centers for Medicare & Medicaid Services released its final national coverage decision regarding the anti-amyloid immunotherapy drug Aduhelm. The language of the coverage determination is complex and legal and regulatory experts are still working to understand all of its implications. This page will soon be updated to reflect what this decision will mean for access to Aduhelm. *****
On June 7, 2021, the FDA announced the accelerated approval for Aduhelm for the treatment of Alzheimers disease. On July 8, following controversy and upon application by Biogen, the FDA revised the label to indicate that Aduhelm should be prescribed only for patients with mild cognitive impairment or mild dementia. Although currently hard to obtain, Aduhelm is now legally available to patients.
Aduhelm is the brand name for aducanumab, a monoclonal antibody developed by Biogen that is designed to bind to aggregated forms of beta amyloid in the brain to trigger their degradation and removal by the brains immune system. Plaques of aggregated beta amyloid are one of three widely recognized elements of Alzheimers pathology the other two are neurofibrillary tau tangles and neuroinflammation. Together, these lead to neurodegeneration, the loss of brain cells and structures, that initially manifests as cognitive and memory dysfunction and eventually leads to physical decline as well.
What Are Potential Side Effects Of Aducanumab
As with any medication, aducanumab may have side effects. Since aducanumab works by helping the immune system remove amyloid deposits from the brain, some patients may experience an exaggerated immune response in the brain. If this occurs, it may cause blood vessel leakiness leading to localized brain swelling, small spots of bleeding in the brain, or both. These side effects can be seen using MRI imaging of the brain and are called amyloid-related imaging abnormalities . As a result, patients receiving aducanumab will require regular monitoring with MRI scans. During the aducanumab clinical trials, those trial patients who developed ARIA had clearing of the focal brain swelling within 2-3 months and most did not develop symptoms. Another potential side effect is an allergic response during the infusion of aducanumab.
This Approval Was Not Without Controversy Can You Explain What Happened
The studies, referred to as ENGAGE and EMERGE were stopped prematurely do to a late-stage interim analysis that suggested the studies failed based on a futility analysis. After all the data was collected the findings from the studies went in two different directions. One study was convincingly negative, and the other was convincingly positive. Furthermore, limitations were found in the data this treatment needs to be given with care, over a long period of time to people who meet very particular benchmarks, and has potential side effects that need to be monitored closely, such as swelling of the brain. Yet the clinical end sees hope in this drug delaying the disease progression in patients who meet those specific requirements necessary to receive the drug when compared to placebo. Currently, the FDAs approval is provisional and is based on aducanumabs ability to lower amyloid propensity. Clinical efficacy needs to be validated with at least another confirmatory controlled study and Biogen has committed to completing that study. Our lab is one of the locations that expects to collect data for this study.
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When Will Aducanumab Be Available
At this time, the specific date it will be available to patients at the Brigham is not known. However, we anticipate it may take a few months from the time it was approved by the FDA. Experts from across the Mass General Brigham system are working together to ensure that the medication can be delivered as safely and effectively as possible to eligible patients.
Paving The Way For Clinical Trials
This study is the first to examine the role of Galectin-3 in the development of glaucoma and to show the value of inhibiting this molecule in order to prevent retinal ganglion cell death.
Future studies by this research team will look more closely at Galectin-3 inhibitors as treatments for glaucoma, testing them in additional animal models as well as looking at more minimally invasive approaches to inhibitor delivery, such as oral administration or in a slow-release gel.
Ongoing studies are also examining eye fluid and serum samples collected from patients during glaucoma surgery to better understand the patient population in which Galectin-3 inhibition would be a potential therapeutic approach.
For example, patients could be candidates for treatment with inhibitor medication if Galectin-3 is found to be elevated in their biofluids. Such studies can pave the way to translate this research into human clinical trials, according to the authors.
Glaucoma remains a blinding disease for millions of people around the world, and these exciting findings by doctors Margeta and Butovsky and team provide insight into the role of genetic variants and the promise of translation to a treatment for patients, said Joan Miller, the David Glendenning Cogan Professor of Ophthalmology and chair of ophthalmology at HMS and chief of ophthalmology at Mass Eye and Ear, Massachusetts General Hospital, and Brigham and Womens.
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How Is Alzheimer’s Disease Treated
Alzheimers disease is complex, and it is therefore unlikely that any one drug or other intervention will ever successfully treat it in all people living with the disease. Still, in recent years, scientists have made tremendous progress in better understanding Alzheimers and in developing and testing new treatments, including several medications that are in late-stage clinical trials.
Several prescription drugs are already approved by the U.S. Food and Drug Administration to help manage symptoms in people with Alzheimers disease. And, on June 7, 2021, FDA provided accelerated approval for the newest medication, aducanumab, which helps to reduce amyloid deposits in the brain and may help slow the progression of Alzheimers, although it has not yet been shown to affect clinical symptoms or outcomes, such as progression of cognitive decline or dementia.
Most medicines work best for people in the early or middle stages of Alzheimers. However, it is important to understand that none of the medications available at this time will cure Alzheimers.
How Much Does It Cost And How Soon Can I Get It
Biogen, the company that makes aducanumab, estimates its cost at $56,000 per year. It is not yet clear whether Medicare, Medicaid, or private insurance companies will pay for this medication. In its press release, Biogen noted that they are working on an agreement with the Veterans Health Administration to cover this medication for veterans enrolled in VA healthcare. Aducanumab is not currently available, but the company is working to make the drug available quickly, perhaps in a few months.
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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.