July 7, 2021 -- Rolfe Johnson is a glass-half-full kind of guy, but also a realist. As a longtime attorney in Houston, only recently retired, he specialized in taking companies public, so he is also a man who knows how to do his homework.
Johnson also has Alzheimer’s disease. So, when his neurologist called to tell Rolfe and Carol, his wife of 60 years, that he would like to prescribe aducanumab (Aduhelm), the controversial new Alzheimer's drug just approved by the FDA on June 7, he didn't hesitate. "I agreed to it right away," he says.
But in true lawyer fashion, he also had a laundry list of questions.
"I certainly wanted to know what he knew about the company, the maker, what their history was," says Rolfe, 82. Assured that the company, Biogen, was solid, he asked other questions about what he could expect from the drug. The couple both expressed concern about the cost -- a staggering $56,000 a year list price -- and were assured that an arrangement was being worked on.
Rolfe had his first monthly infusion June 30.
"There are a number of side effects they warn about, and so far, I haven’t had any of those," he says.
He's keeping his expectations in check. He knows it won't reverse the damage already done since his diagnosis 5 years ago. "If it works well, it prevents the propagation of Alzheimer's," he says, beginning "from when you start taking it."
His wife, 81 and a psychotherapist, chimes in: "That's as good as a cure for us."
As advocates and critics debate the FDA's approval of the new drug, and a chorus of experts and patient advocates protest the price, many patients and their families are excited about it, hopeful that it will stop the debilitating disease from getting worse and help them function and enjoy time with family and friends.
Aduhelm: Sticking Points
The price of the drug isn’t the only point of debate. So is the FDA's accelerated approval, which was based on clinical trial data that found a reduction in amyloid plaques in those given the drug, compared to those in the placebo group. The plaques build up in the brain and are thought to lead to the memory loss and eventually the inability to do the tasks of daily living. But reduction of the plaque, critics say, doesn't prove there is a clinical benefit, such as remembering a grocery list or the route home.
The FDA also approved it broadly for use in Alzheimer's patients, although some experts say it works best in early stages and should be preserved for those patients.
Patient Interest is High
Despite the debate and the unknowns, Rolfe's neurologist, Paul Schulz, MD, a professor of neurology at McGovern Medical School at the University of Texas Health Science Center in Houston, says he's been inundated with emails and calls from patients and families, asking about the new option.
Ever since the approval, he says, he's gotten numerous emails and phone calls, some from patients he hasn't seen in 3 years. "We get a lot of them every day," says Schulz, who also directs the UT Health Neurocognitive Disorders Center and is an adviser for Biogen.
According to Biogen, more than 900 sites across the country are ready and prepared to give the monthly infusions.
Addressing the Cost Barrier
For the Johnsons, one of the controversial points -- price -- was solved. Officials at Memorial Hermann Hospital, UT Health's teaching hospital, agreed to donate the money to Schulz's dementia program so that Johnson can get 6 months of infusions without cost to him. "The hospital is very supportive," Schulz says.
By the end of that period, Medicare may make a decision on coverage, experts say. Once Medicare decides, other insurance plans typically follow its lead.
Schulz has found another avenue to solving the prohibitive cost issue. Several large pharmacies in his area have agreed to infuse patients now and bill Medicare "if and when they approve it."
While smaller pharmacies can't take that potential financial hit, some of the larger ones are able to, he says.
Treat Now, Pay Later?
Marc Archambault, 70, of South Kingstown, RI, was the first person in the world to receive the drug outside of clinical trials. He doesn't know if he is on the hook for that fee or not. So far, he hasn't paid anything but says he isn’t sure about how coverage will work out.
That didn't stop his resolve.
"There was no way I didn't want to do it," he says.
He received his first infusion June 16 at Butler Hospital in Providence, RI. And he half-jokes: "I think the first person in the world to get it [after approval] should not have to pay."
He, too, is a realist about what the drug can and can't do. "This is a shot for me to stay hopefully as I am now," he says. "I said yes right away."
Diagnosed in 2014, Archambault is a longtime real estate agent in South Kingstown, a town of about 30,000, some 30 miles from Providence. He is still active, but he gave up managing the office in 2017 and asks for help when he needs it. "I started to have trouble with words pretty early," he says. "All of a sudden, I can't get the word out."
He's open about his diagnosis and asks for help from those close to him. For instance, if he is trying to talk about a car but can't get the words out, he'll tell his daughter or a colleague to tell him the word for "the thing you sit in and it gets you around town."
He's had no second thoughts about the decision, he says.
"It is my turn to do this,” he says.
He had already been a research participant at the Memory and Aging Program at Butler Hospital. At a news conference on the day of the infusion, Stephen Salloway, MD, director of neurology and the Memory and Aging Program, said that, "Today we open a new era in the treatment of Alzheimer's," with a focus on slowing the progression. Salloway was co-chair of the global investigator steering committee for Aduhelm's phase III studies.
"If this works, I feel like I may not have to go through those last stages when I don’t know anybody," Archambault says. "It isn't perfect, but I'm giving it a shot."
Waiting Out the Reimbursement Issue
Others, like Jay Reinstein, say they will be ready for the treatment once the reimbursement issue is decided. A former assistant city manager in Fayetteville, NC, he was diagnosed in 2018 at age 57 with younger-onset Alzheimer's disease. Originally planning to work until age 65, he had to retire early.
"My neurologist has said that I would be a good candidate for Aduhelm, and the only delay is waiting on a decision by Medicare to determine the amount they will cover for the infusion."
The list price, he says, is "more than I can afford at the moment. Once a decision is made, even if it does take months, I plan to move forward with Aduhelm."
With the new drug, he says, "I understand there is a potential for swelling of the brain, a potential for bleeding. To me, the benefits outweigh the [potential] side effects."
He and his wife, Angela, celebrated their 30-year anniversary in May. They have two daughters, a son, and four grandchildren. "I want to be around for my grandkids and kids," he says.
To cope, he goes to therapy and a support group and remains hopeful that things will work out. "I'm just waiting on the green light. I'm just crossing my fingers."
Aduhelm as Step One
Patients' passion and excitement to start the treatment don't surprise Schulz. The approval of the drug "is giving people a lot of hope," he says. He agrees the effects are far from perfect and the drug can’t reverse the damage. "With no disrespect meant to Biogen, it’s giving us a 25 to 40% less decline on different parameters [measured]. That's way better than no effect."
The approval, he says, "is the first important step of finding things that are effective." After then-President Richard Nixon declared a war on cancer in 1971, "we started getting medicines that had better effects. I think this is the same way."
Family, Friends on Board
Support from family and friends seems to convince those who have begun the drug or want to get it that their decision is sound -- and to keep their spirits up. "My daughter is excited about it," Reinstein says, "as my parents are."
While his wife, Angela, worries more about the potential downsides of the treatment than he does, she is also on board.
None of these patients talk about a "cure," but more about holding onto what they have. As they cautiously anticipate a future they can function in, they're also enjoying life in the moment.
Archambault laughs about how popular he's become since news of his infusion was publicized. "I've heard from a ton of old friends." He's booked fairly solid for lunch and dinner. "None of my friends said, 'What are you doing?' 'Are you nuts?'"
Rolfe needs to dust off his dancing shoes. The Johnsons, who were high school sweethearts, have three adult children and 11 grandchildren. "Our oldest grandson just got engaged," says Carol, who says she is hoping for great-grandchildren soon -- and isn't shy about letting family members know that. "There's a huge family wedding next February."
Rolfe smiles as he chimes in: "We're going to dance at that wedding. My high school sweetheart is going with me."