July 13, 2021
In June, the Food and Drug Administration approved the new drug Aduhelm for people with Alzheimer’s disease. It was the first new drug in nearly 20 years to be approved for Alzheimer’s, and the first approved medicine that aims to slow the progression of the disease and not just treat its symptoms. Interest in the drug has been intense. Here are some questions and answers about Aduhelm.
Who Is Eligible to Get Aduhelm?
Aduhelm, which was known by its scientific name aducanumab during testing, has been approved for anyone with Alzheimer’s disease, though it was tested only in people in the earliest stages of the disease and appeared to benefit only some patients. The company that makes it, Biogen, recently began shipping the drug out to 900 medical centers around the country.
How Do I Get a Prescription for Aduhelm?
Your doctor can discuss with you whether Aduhelm may be right for your situation. Many experts question whether the drug is truly effective, since it only appeared to benefit some patients during clinical trials. It may be most effective for people in the earliest stages of Alzheimer’s, though it can be prescribed to those in the moderate and more advanced stages of the disease. Some recent developments might limit the prescription only to men and women with early stage Alzheimer’s disease..
How Much Does Aduhelm Cost?
Biogen set a price of $56,000 for a year’s supply of the drug. Whether some or all of that cost will be covered by private insurance is still uncertain. Medicare will likely cover much of the cost, but many patients will likely need to pay a substantial amount out of pocket.
Are There Additional Costs?
In addition to the cost of the drug itself, patients will need to undergo regular MRI brain scans, beginning in the first year of treatment, to assess whether the drug is having a positive effect.
How Is Aduhelm Given?
Aduhelm is administered by an intravenous infusion every four weeks at a clinic, hospital or doctor’s office, which will further add to the cost of the drug. The infusion of the drug takes about an hour.
How Does Aduhelm Work?
The drug is what’s known as a monoclonal antibody, the first such drug to be approved for Alzheimer’s disease. It is designed to stimulate the immune system to eliminate small-sized clumps of toxic beta-amyloid component (the by-product originating from the cleavage of a protein called APP) that ultimately form amyloid plaques in the brains of people with Alzheimer’s disease. In Aduhelm testing, plaques were reduced. But some experts question whether reducing the buildup of plaques in the brain will lead to meaningful improvements in thinking and memory skills or a better ability to carry out day-to-day tasks. Smaller versions of beta-amyloid aggregates might represent the real culprit.
Who Might Benefit Most from Aduhelm?
In clinical trials, the drug was tested only in patients with mild Alzheimer’s disease, and it provided modest benefits but only in a subset of patients. Doctors still aren’t sure which patients might benefit most. Many experts believe the drug may be most effective in the earliest stages of Alzheimer’s, before damage to the brain becomes extensive.
Is Aduhelm a Cure for Alzheimer’s Disease?
Aduhelm is not a cure for Alzheimer’s. In tests, when it had an effect, the drug slowed deterioration of cognitive skills, but it did not reverse memory loss. Researchers continue to look for new and more effective drugs that may one day actually cure Alzheimer’s disease.
Does Aduhelm Have Side Effects?
In clinical trials of the drug, about 40 percent of study participants developed some degree of swelling in the brain. Symptoms included headaches, feeling dizzy, vision problems, and nausea and vomiting. Almost 20 percent of patients also developed tiny bleeds in the brain, though its uncertain how harmful they may be. Patients getting the drug will have to undergo brain scans before the seventh and twelfth infusion to monitor brain changes, which may mean they have to stop getting the drug if the scan results indicate potential problems.
How Long Do Potential Benefits of Aduhelm Last?
It’s uncertain how long benefits, if any, might last for someone receiving Aduhelm. The company is required to keep monitoring patients on the drug to assess benefits, safety and long-term effects. If Aduhelm does not show clear-cut benefit in follow-up testing, the FDA said it may withdraw approval of the drug. Other monoclonal antibodies showed promise against Alzheimer’s in early stage testing, only to show no benefits when tested in large numbers of patients.
Why Is Aduhelm So Controversial?
An outside expert advisory panel that advises the FDA thought that proof was lacking that Aduhelm provides meaningful clinical improvements in memory and thinking skills in people with Alzheimer’s disease, and recommended further studies to assess the drug’s efficacy. Ten experts voted against approval, and the last expert voted “uncertain.” Since approval, three experts quit the FDA panel. The FDA often follows the advise of advisory committees but in this case approved the drug anyway.
Are There Additional Alzheimer’s Drugs in the Pipeline?
Yes, dozens of promising new Alzheimer’s drugs based on various therapeutic approaches are in various stages of testing. Experts hope that one of these will show clear-cut evidence of benefit. The underlying causes of Alzheimer’s disease are still poorly understood. Research groups like the Fisher Center for Alzheimer’s Research Foundation continue to provide critical funding into several areas of basic research that will help unravel the causes of Alzheimer’s disease, in the hopes of one day finding a true cure.
What’s the Bottom Line?
The long-term benefits and risks of Aduhelm are still being evaluated. Whether the drug is right for you or a loved one will depend on an open discussion with your doctor. Given the uncertainties surrounding the drug, including whether it will provide any benefit while carrying risks, some people with Alzheimer’s may want to consider enrolling in a clinical trial of other promising drugs now undergoing testing. You can learn more at www.clinicaltrials.gov
By ALZinfo.org, The Alzheimer’s Information Site. Reviewed by Marc Flajolet, Ph.D., Fisher Center for Alzheimer’s Research Foundation at The Rockefeller University.
Source: Food and Drug Administration, Biogen.
