Debra and Paul McAlenney were at their home in Simsbury on a recent Tuesday morning watching their 5-year-old grandson, Hudson, while his parents worked.
Hudson spends a couple of days during the week with his grandparents, or as he refers to them, Nana and Bacon.
“Paul was sitting across the living room from us and Hudson said, ‘Bacon – dah dah dah dah dah,’ and Paul didn’t respond. And then he went, ‘Bacon.’ And Paul still didn’t respond,” Debra said. “And then he went, ‘Bacon!’ And I said, Paul, he’s calling you bacon and you need to just accept that and respond to it.”
The older couple laughed.
“So, he’s been Bacon ever since,” Debra said.
“It’s stuck,” Paul said, laughing.
It’s these moments, these memories, that the McAlenneys are fighting to hold on to as Paul lives with Alzheimer’s disease, which affects nearly 6 million Americans today, including about 80,000 Connecticut residents.
With a disease that has no known cure and is projected to triple in the population by 2060, many patients and advocates were quick to praise the U.S. Food and Drug Administration’s recent decision to approve a new Alzheimer’s drug treatment for the first time in nearly two decades.
But the move has been controversial -- scientists and researchers are divided on whether the drug actually works and is worth its estimated $56,000 annual price tag per patient. Some say it puts them in a difficult position going forward.
“Patients and their families, you know, they’re desperate for a cure. It’s an awful position to watch your loved one decline cognitively,” said Dr. Joseph Ross, a professor of medicine and public health at Yale School of Medicine. “Of course, those who have the means and resources are going to want to pay for this, but there’s not clear evidence that it’s going to help them.”
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Alzheimer’s disease is most common in people aged 65 years and older, although some are diagnosed with it younger. It’s the sixth-leading cause of death in Connecticut and the United States.
Early signs can include memory loss, confusion, difficulty in problem solving or planning and changes in mood and personality. Specialists will order imaging scans to see whether patients have a buildup of amyloid plaque in the brain, a biomarker for Alzheimer’s disease.
There are a handful of drugs that can help people manage those dementia symptoms, but they don’t address the underlying disease.
Then pharmaceutical company Biogen developed a drug called aducanumab, which aimed to remove or clear away amyloid plaque from the brain and thus delay the progression of someone’s cognitive decline.
Clinical trials were halted in 2019 as researchers saw poor and mixed outcomes -- plaque was successfully removed, but proof that it resulted in cognitive benefits was murkier. Ultimately, the drug won FDA approval June 7 to become the only disease-modifying treatment for Alzheimer’s on the market.
“We’re hopeful this is just the beginning,” said Kristen Cusato, communications director for the Connecticut chapter of the Alzheimer’s Association. “Someday, could we be close to diagnosing people even earlier and maybe having something so that this disease doesn’t take everyone? Wouldn’t that be amazing?”
But many in the medical community consider the trial data suspect. Ross, who leads a federally funded center on regulatory science research, said the findings did not meet the level typically needed for widespread approved use.
“The FDA’s role is to independently adjudicate the quality of the evidence and only approve products that are safe and effective for use that they expect patients to benefit from,” he said. “And if they are approving products on the basis of marginal benefits that still contain safety concerns, they’re selling a false hope to patients. It’s not fair to them.”
The McAlenneys are familiar with the drug treatment, which is a monthly infusion. Shortly after Paul’s diagnosis nearly seven years ago at age 72, he entered into one of aducanumab’s double-blind clinical trial sites at Yale.
He eventually confirmed that he had been receiving one of the highest doses of the drug, not a placebo.
“I always felt that whenever something was put in front of us that there was no question what you’d do,” Paul said.
Debra said they went into the trial hopeful, but not overly optimistic.
“For the majority of the time, things were going pretty well, except when they decided to end the study for a while, that was devastating,” she said. “And I think during that time Paul did decline a bit, which also, to me, showed that when he was on aducanumab it was helping, so we were thrilled when they brought it back.”
Dr. Dennis Selkoe, a neurologist who teaches at Harvard Medical School, joined those in the medical community who had hoped for federal approval of the drug, though he did acknowledge the less-than-ideal circumstances.
“I know some of my colleagues are very disappointed in the FDA, for a variety of reasons, because they thought the data was imperfect,” he said. “But you have to walk before you can run.”
Selkoe is the co-director of the Ann Romney Center for Neurologic Diseases at Brigham and Women’s Hospital in Boston. He has spent nearly 40 years researching Alzheimer’s disease.
“I can understand that doctors and patients can be anxious, but we’ve been waiting for this moment where we have something that looks like it modifies the disease,” he said.
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Aducanumab will be sold under the brand name Aduhelm. The drug will likely be covered, or at least partially covered, under federal insurance programs like Medicare, though most of those enrollees are still responsible for paying about 20% of their health care costs.
Other private health insurers may place stipulations or restrictions on the coverage they’re willing to offer for aducanumab.
Either way, Ross said it’ll fall on physicians to explain the possible financial burdens, which could mean significant medical debt or bankruptcy for some families.
“And they’re [clinicians] going to say that this therapy is going to cost substantial sums, not even to account for the visits to the physician, paying for the physician, paying for the radiologist to read the [brain] scan,” he said.
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It’s only been a week since the FDA’s announcement, and Selkoe said he’s already fielding calls and emails from interested patients and their families.
There will be a priority, he said, on making sure people understand the scope and limitations of the treatment, including possible side effects like swelling in certain areas of the brain, which occurred in about 40% of trial participants.
“As long as we’re clear with our patients, tell them the pluses and the minuses, I think quite a lot of patients will say, ‘I want to take a chance, I want to see if this helps me,’” Selkoe said.
That’s certainly the path that the McAlenneys have taken.
Paul, who had a career as an attorney for more than 40 years before his retirement at age 70, restarted infusion treatments six months ago.
He knows it’s not a permanent solution, and that his disease will one day become more severe, but he hopes the drug gives him more time with his wife, their children and grandchildren.
“If they give you some options, you know -- do I want a triple or is it still all right if it’s just to get to first base?” he said, laughing. “And so, we were always definitely open to doing it.”
Pharmaceutical manufacturer Biogen said it’ll begin shipping Aduhelm to health centers later this month. The drug maker is required to conduct an ongoing trial study that could take place for the next nine years in order to prove the treatment’s effectiveness for it to stay on the market.
