On June 7, the US. Food and Drug Administration (FDA) approved aducanumab for the treatment of Alzheimer’s disease. It will be sold by Biogen and called Aduhelm on the market.
It is the first Alzheimer’s treatment to be approved by the FDA since 2003 and the first to target the underlying disease process, a breakthrough for families and for scientific efforts. This milestone has renewed interest in the field but has also come with controversy, with some in the medical community questioning how much tangible benefit it will have for people living with Alzheimer’s.
What does the drug do?
It addresses a root cause of dementia—amyloid beta proteins that accumulate to form plaques in the brains of people with Alzheimer’s. These plaques disrupt and eventually lead to the death of neurons, first in the areas of the brain related to memory and later to areas that control functions like reasoning and language.
Aduhelm has shown that it can decrease the amount of amyloid protein in the brain of those with early Alzheimer’s. This may slow the loss of memory and allow people to participate more fully in daily activities longer. It’s unclear how much it will relieve day-to-day symptoms of Alzheimer’s for all patients.
Therefore, to receive the drug treatment it is necessary to demonstrate: (1) that the patient has early Alzheimer’s disease by neurological evaluation and neuropsychological tests; and (2) that there is amyloid in the brain using a positron emission tomography or PET scan of the brain.
How is it taken?
The treatment is given through an IV once a month.
How much does the treatment cost?
The cost of the treatment is $56,000 per year: it requires one intravenous infusion every four weeks at about $ 4,312 per infusion. It is anticipated that the cost will be covered—at least partially—by Medicare.
Is it a cure?
No. While it tackles a cause of the disease, versus symptoms, Aduhelm does not claim to stop or reverse dementia or Alzheimer’s. It aims to slow progression of the disease.
What’s the controversy?
Drug development often takes decades, and Aduhelm’s road has been rocky. Phase 3 trials were halted in 2019 but restarted with higher doses of the medication. As a result, clinical evidence about its impact is mixed. It appears to decrease amyloid in the brain, but this was not reflected in clear improvement of the clinical manifestations of Alzheimer’s disease.
Some scientists, including members of the FDA advisory committee that initially rejected Biogen’s application for the treatment’s approval in November 2020, are concerned that there isn’t enough evidence that the drug will benefit patients. Three members of the FDA advisory committee have resigned since the approval announcement was made.
Because it was given the go-ahead under what’s called “accelerated approval,” a Phase 4 study will be conducted and if serious efficacy concerns arrive, the drug could be eventually taken off the market.
What are the side effects?
The most critical potential side effects are brain swelling and bleeding in the brain occurring in 41% of treated patients (i.e., almost half of those treated compared with 10% of untreated subjects). Brain edema and cerebral hemorrhages are potentially lethal complications. For this reason, it is highly recommended that people taking Aduhelm also have MRIs several times a year to monitor for signs of swelling or bleeding. Other side effects may include headache, falls, diarrhea, and disorientation.
Patients with moderate and advanced Alzheimer’s disease are not eligible for the treatment, most probably because they have higher concentrations of amyloid in the brain. Other factors such as advanced age, risk from hypertension, anticoagulation, evidence of previous strokes or hemorrhages on brain imaging, as well as type and severity of the symptoms may make it unsafe for a person to take Aduhelm.
How do I know if it’s right for my spouse/parent/grandparent? What should I do to try it?
Speak to your doctor. Aduhelm is being rolled out across the US to nearly 1,000 health centers this year.
It will only be available by prescription and may be costly. While it will likely fall under Medicare Part B, there hasn’t been an announcement of whether or how much of the drug’s cost will be covered under Medicare.
Content has been reviewed by Prof. GC Roman, a member of ARARF’s medical advisory committee.
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