The Food and Drug Administration’s approval of a new drug to treat Alzheimer’s disease, Aduhelm, sparked reactions ranging from jubilation to dismay. Some cheered the approval of the first new Alzheimer’s drug in nearly two decades. Others denounced the FDA decision as premature, based on insufficient evidence.
People with the illness and their loved ones may feel caught in the middle, unsure what to do next. Here are the key points to understand.
It’s not clear whether Aduhelm will have a significant effect on the course of the illness — but it might. The FDA approved the drug because it reduces a sticky protein, amyloid beta, that clumps in the brains of people with Alzheimer’s. It’s logical to think that might help, because the disease worsens as more plaques develop. But it’s not known whether the amyloid causes Alzheimer’s — or is merely a sign of it. And the studies showed mixed results on the drug’s effects on cognitive decline.
In approving the treatment last week, the FDA didn’t require the high level of evidence that it usually does. Instead, it made its approval conditional on the completion of another clinical trial to prove the drug works, a process that will take years. Critics called this unusual approach reckless.
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But Jim Wessler, CEO of the Alzheimer’s Association’s Massachusetts/New Hampshire chapter, pointed out the other side: “There is a bit of difference between some of the academically based specialists and the general public that are saying, ‘Give me a chance here. I have a progressive degenerative disease.’ ”
The FDA faced big risks no matter what it did, said C. Michael White, head of the Department of Pharmacy Practice at the University of Connecticut.
If it had rejected the drug now, and years later a study showed that it worked, in the interim millions of people would have missed an opportunity to stave off a horrible illness. Instead, the FDA chose the opposite risk — that the drug might later be found ineffective, and meanwhile billions of dollars get wasted.
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White said he’s “cautiously optimistic” that Aduhelm will have some benefit, but “people should know that they are gambling.”
Wessler used a baseball analogy. “This is a hit. Everyone wants to see a home run or a grand slam,” he said. “This is not a grand slam. This is a single. A significant step in the right direction.”
The drug, Wessler said, could allow an Alzheimer’s patient to stay healthy long enough to enjoy a child’s wedding or graduation, or meet a grandchild, or take a bucket-list trip. “This drug promises to provide more time,” he said.
That was another important reason for the FDA’s approval: Alzheimer’s is a terrible illness, and there’s nothing else available to treat it.
Even if Aduhelm does work, it probably won’t help people with late-stage disease. Although the FDA approved the drug for anyone with Alzheimer’s disease, the clinical trials enrolled only those with early, mild cases. Once the disease has advanced, it is questionable whether this drug can have an effect on slowing further decline — and it definitely cannot reverse the course of the illness.
“We know from other failed drug attempts to control amyloid beta that by the time severe Alzheimer’s disease occurs, it is too late,” White said.
Aduhelm has some risks. Three in 10 participants in the clinical trials experienced brain bleeds or swelling. These side effects were transient and can be easily managed, said Dr. Stephen Salloway, director of neurology and the Memory and Aging Program at Butler Hospital in Providence, and one of the leading researchers studying Aduhelm. Frequent MRIs can detect early signs of problems, and the dosage can be adjusted or paused, he said.
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But others expressed worries that once millions of people are taking the drug, more dire side effects could emerge.
It’s not just a pill. To take Aduhelm, you have to travel every four weeks to an infusion center and sit for an hour while the drug is delivered into a vein in your arm. Additionally, you will likely have to undergo a PET scan or lumbar puncture beforehand to confirm the presence of amyloid. And you will need regular MRIs to monitor for side effects.
Aduhelm is costly, and it’s not clear how much patients will have to pay. Biogen, the manufacturer, set a price of $56,000 a year for each patient. Most people with Alzheimer’s disease are enrolled in Medicare Part B, which pays 80 percent of the cost. Many Medicare beneficiaries have supplemental insurance that picks up some or all of those co-pays. For people under 65 with private insurance, their costs will vary depending on their coverage. Some insurers may even decide not to cover the drug at all.
If you’re interested in this drug, here’s what to do. Get diagnosed, Wessler said. That may seem obvious, but national data show that half of people with dementia never get diagnosed, and half of those diagnosed are never told about it, he said. The availability of Aduhelm is sure to change that, he said.
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If you’re concerned about your memory or that of someone you love, you can start by reading up on the 10 warning signs of Alzheimer’s. Then see your doctor and begin the discussion of risks and benefits. It will take time before Aduhelm is widely available, but it’s not too soon to start talking about it.
Felice J. Freyer can be reached at felice.freyer@globe.com. Follow her @felicejfreyer.