Elation, pride, and a slew of questions: Clinical trial participants reflect on their own complicated reactions to Aduhelm approval

For the roughly 3,500 people who volunteered as clinical trial participants for the Alzheimer’s therapy Aduhelm, Monday’s divisive approval spurred a complicated brew of emotions.

There was elation, yes — that the endorsement of the therapy by the Food and Drug Administration was a validation that maybe it really had been working for them, that it was letting them take the dog for a walk, or embrace new projects, or have a simple conversation with their spouse for more years than they could have otherwise.

“There hasn’t been much good news for a long time, so it’s good to have,” said Ron DeChant, 68, of Avon Lake, Ohio, who was diagnosed with Alzheimer’s in 2016.

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There, too, was pride. Their participation in these trials enabled a milestone that the whole dementia field has been tracking obsessively. This is the first new Alzheimer’s therapy approved in nearly two decades, and they were part of that — something giving other people in their shoes, and their caregivers, a reason for hope.

“Right now, it’s all we’ve got, so it’s my favorite,” said Geri Taylor, 78, who received her first dose of Aduhelm more than six years ago.

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But there were ever more dangling questions about the decision. They knew lots of experts doubted the therapy worked at all, and that even the FDA referees who approved the therapy acknowledged “some uncertainty about the drug’s clinical benefit.”

With the therapy now approved, did they need to go through the process of getting their insurers to cover the treatment like any other patients — or would they keep receiving their monthly infusions as they had as trial participants? The FDA also called for Biogen, the company behind Aduhelm, to conduct another trial to confirm a clinical benefit, so what did that mean for them? Could the therapy be yanked away once more?

And even if the therapy did work as its supporters claimed it did, it still just amounted to something that could slow the progression of the disease. That was no small thing, but Aduhelm is not a cure, nor does it reverse cognitive losses.

Doctors said Monday they knew they were going to have to try to set realistic expectations for patients who would be starting the therapy now that it was approved, but the participants in the trials are well aware of the fact that disease progression did not halt entirely when they started their infusions.

“As I reflect on all this work and time — and all these needles and these blood draws and these MRIs — I’m delighted that I’m a part of it and have helped move the field forward, but I’m also cognizant that we have so much more to do,” said Phil Gutis, 59, of New Hope, Pa. “We moved the needle, but this is not a dance-around-the-Christmas-tree moment.”

Patients - Phil Gutis
Phil Gutis, 59, of New Hope, Pa. STAT/Courtesy

Gutis, a onetime New York Times reporter, has been feeling well enough lately that he’s picked up some proofreading work. Alzheimer’s hasn’t just been robbing him of his memories, but of his identity as well, so it felt good that he could devote his mind to a project.

But there were humbling moments too. He had a cognitive assessment two weeks ago, and he couldn’t remember the date, got confused about what the big hand on a clock does, and only came up with five words that started with F in one minute. He had gone in thinking he was going to knock his test out of the park that day, he said.

“And I did not knock it out of the park.”

Theirs is a group of clinical trial participants that has already experienced a far more chaotic journey than your average drug trial volunteer. They felt the devastation in March 2019 when Biogen said it was pulling the plug on the trials because the therapy, then known as aducanumab, did not appear to work. They were whipsawed seven months later when Biogen revised its stance and said that further analysis showed the therapy could in fact slow patients’ decline — resulting in a strange mix of confusion, anger, but also renewed hope. It meant the study participants could once again start receiving monthly infusions of the therapy.

For them and their caregivers, it is difficult to say for sure whether and how Aduhelm might have helped them. They only knew their experiences as they were; they couldn’t say what the alternative might have been, if there hadn’t been the therapy.

When Susan Woskie’s wife Debby Rosenkrantz was first diagnosed with Alzheimer’s, “we grasped for any straws, any incremental help,” Woskie said.

But over the years, as Rosenkrantz participated in the studies, the Cambridge, Mass. couple came to recognize both the potential benefits and the limitations of what the therapy could do.

“In the beginning of the trial, she had higher hopes for the medication than it was able to provide, but I think now it just feels like, there’s not many things we can do,” Woskie said. “Exercise, eat right, don’t do this, do this, don’t drink alcohol in excess, take your vitamins. But there’s not many things we can do. Having something that at least provides some benefit is a positive.”

Rosenkrantz said that with the approval, she hoped that the other people in her Alzheimer’s support group could now access the therapy she’s been getting for years, even as they hope that this is just a first step, with more treatments to come.

“To me, there’s now something to offer them, which makes this feel better as a community, as people who live with Alzheimer’s,” she said.

Patients - Debby Rosenkrantz
Debby Rosenkrantz of Cambridge, Mass. STAT/Courtesy

If Aduhelm really did slow the disease process, that amounted to a meaningful benefit, participants stressed. For DeChant and his wife Colleetta, it was more time for him to be there — not just physically, but also, to whatever extent possible, cognitively — for the big events in the lives of their three children.

“We have a wedding and a grandbaby coming,” Colleetta said. “We have all sorts of good things with our kids.”

Ron, who had to give up his career as a pharmacist because of his Alzheimer’s, felt Aduhelm “changes what I can do and what I can’t do, and how I can talk and I can’t talk.”

But he then asked his wife: “Did I say that right?”

Cathy Schaefer of Fort Myers, Fla., said she felt aducanumab helped her husband Warren, a trial participant, with his everyday functioning. He could remember what he needed at the grocery store, and where in the grocery store those items were, she said. She hopes other people will see the same effects.

“I’m excited for them,” she said.

After Biogen in October 2019 revived aducanumab and said trial participants could restart their infusions, it took months for some volunteers to begin them again, with the Covid-19 pandemic adding another hurdle.

Warren wasn’t able to pick back up with his infusions until June 2020, Cathy said, and just a few months later, in November, he died at 74. The official cause was Covid-19, Cathy said, but she couldn’t help but think that his Alzheimer’s, which she believed accelerated during the break in his infusions, played a role.

With Warren’s Alzheimer’s, Cathy knew they weren’t going to have all that many years left together. But on Monday, she spoke by phone from a trip to Arizona. She was there for a wedding, and had just played golf, and part of her wondered whether there was some other reality — if the course of aducanumab’s odyssey had been different — in which Warren could have been there with her.

Source: STAT