When Jeff Borghoff saw the article Tuesday morning, the first thing he did was double-check the date it was published. He thought it might be years old, not a breaking story.
In Pennsylvania, Phil Gutis saw a Facebook (FB) post about the announcement. “What the hell?” he wondered.
Before March, both had been enrolled in a clinical trial for an experimental Alzheimer’s therapy from the drug maker Biogen (BIIB). When the studies were halted because the drug, aducanumab, did not appear to be working, they had been devastated.
On Tuesday, however, the company announced that a new data analysis showed the treatment reduced some patients’ decline; it said it would ask the Food and Drug Administration to approve the drug. Alerts about the news ricocheted around clinics, Wall Street, and the broader community of Alzheimer’s patients, caregivers, and researchers.
But just as with the cancellation of the trials, the resurrection of aducanumab was particularly jolting for the roughly 3,200 participants and their families:
“I don’t even know what to think.”
“It’s dizzying. Truly dizzying.”
“What does it mean to us now?”
Biogen’s reversal also restored a cautious sense of optimism that an effective Alzheimer’s treatment could be within reach. Some patients wondered if perhaps the experimental treatment they had been taking had actually been slowing the progression of their disease.
“I did feel like it was working,” said Debby Rosenkrantz, 66, of Cambridge, Mass.
For many, there was also the urgent question of what comes next. Patients wanted to know if they could start receiving aducanumab again, and when, and how. Even if the FDA were to approve the drug, the process could take a year. And then there would be a process of determining whether insurance companies would cover the therapy.
As Susan Woskie, Rosenkrantz’s wife, said, “This is precious time for folks with cognitive decline.”
In an interview with STAT, Samantha Budd Haeberlein, Biogen’s lead Alzheimer’s scientist, said the company was working to open a new study for former trial participants. The study will be open label, meaning there will be no placebo arm and all patients will get the treatment. She said patients should reach out to their trial sites so that they can start receiving the drug once regulators and review boards have given the study a green light.
Haeberlein said she did not want to put a timeline on how long that process might take, but that “we will work to get that study up and available for patients as soon as possible.” U.S. sites are expected to open before other locations because talks with regulators were already underway.
Some patients were already on the case. Gutis, 58, had emailed his trial site Tuesday morning and received a quick reply: “Holy smokes, big news!”
Gutis, who was diagnosed more than three years ago, said he had been feeling good lately, a fact he attributed to his increased exercise regimen. Now, though, he wonders whether he may be benefiting from a lasting effect of the drug. “In some ways, you want to stay positive, but it’s so disappointing that they yanked the trial and caused so much anguish back in March, when clearly they didn’t have all the data,” said Gutis, who described feeling a mix of frustration and elation.
Other patients echoed that sentiment. They were grappling with the cognitive function they had lost in just the last seven months, and feared it could not be recovered. At the same time, the mixed messages from Biogen were so confounding that some patients said that, as hopeful as they were, they couldn’t help but wonder if the company was trying to jam a drug through the regulatory process for financial gain.
Since the trial ended, many of the patients had been told whether they were in the treatment arm or the placebo arm. Families of some patients in the treatment arm said that the therapy seemed to be slowing the progression of the disease and that their conditions deteriorated faster when the monthly infusions were stopped. They knew that the apparent benefits seen in their family members did not prove the drug was effective overall, but the revival of the drug caused many to believe again that it was at least possible.
Deb, 61, who lives in Maine and asked that her last name not be used because her colleagues didn’t know she had Alzheimer’s, said she has had more trouble thinking clearly since the trial ended. “There’s just this void,” she said. “It feels like it goes on forever. And it scares me.”
And Borghoff, 55, of Forked River, N.J., said he thought he had become more easily agitated and anxious since March.
“I’m sort of scratching my head and jumping for joy at the same time,” he said about the Biogen decision. “I hope there’s not a lot of time wasted for us to get back on it,” he added.
Dick Carroll, 75, said his cognitive scores actually improved while he was on the trial. But the onetime teacher knew that those results might just have been him getting better at taking those tests, not a benefit of the drug.
Still, his outlook on Tuesday was one of pure optimism. He was looking forward to figuring out how to get back on the drug, even if it meant fighting through the Houston traffic to get to the trial site. “Some hope is better than no hope,” he said.
In North Carolina, Debbie Salerno, who heard the news from her mother-in-law over the phone — and who confirmed it herself after hanging up because she found it so surprising — was eager to learn more because her husband, Tony Milazzo, 56, had been in the trial. She said she felt his disease progression had taken off since the trial was halted, and he no longer wanted to socialize or engage with others as much.
She said that she had told Milazzo about the return of aducanumab but that she didn’t know how he felt. “He’s sitting right next to me, but his reactions don’t always come out in his expression,” she said by phone.
She then started addressing Milazzo. She told him the drug he had been on in the trial might be coming back, and asked how that made him feel.
“I would like to be on the medication,” he said.
“Do you think it was helpful?” Salerno asked.
“I don’t remember that part of it.”
“Does the thought of the medication that would be helpful to you, does that give you a little bit of hope?”