The U.S. Food and Drug Administration has fully approved a new drug to treat early stages of Alzheimer’s disease, but many Floridians are unsure if they should use it or even how to get it.
Research shows the drug, Leqembi, slowed progression of Alzheimer’s over 18 months by an average of 27%, giving people more meaningful time with their families. It’s the first medicine shown to modestly slow cognitive decline in the early stages of the disease. But Leqembi doesn’t cure Alzheimer’s and has some rare but potentially serious — even fatal — side effects.
Even before the FDA approval, Floridians had been testing the drug at 36 clinical trial sites, with as many as 600 patients potentially using Leqembi. Doctors involved in the trials say they have seen good results, and have learned how to manage the side effects.
Now that Leqembi has FDA approval, anyone with confirmed early-stage Alzheimer’s can receive the drug if they fulfill the requirements. When the Alzheimer’s Association International Conference kicks off Sunday in Amsterdam, doctors will talk about advancements in research and medicine and learn more about how Leqembi will roll out nationwide. The manufacturer, Eisai, estimates 100,000 individuals will receive their drug within three years.
Where is Leqembi available in Florida?
First, the requirements.
Anyone who wants Leqembi will need to take a test to confirm they have amyloid buildup in their brain, indicating early-stage Alzheimer’s. Either a PET scan or a lumbar puncture, otherwise known as a spinal tap, can confirm that. You can get those tests from doctors who specialize in Alzheimer’s and memory disorders.
“It’s not available to every physician that treats older people,” said Dr. Elizabeth Crocco, chief of geriatric psychiatry in the Department of Psychiatry and Behavioral Sciences at the University of Miami Miller School of Medicine. “There’s still a lot of study that has to be done on this drug before it is opened to every clinician to give.”
Once an expert confirms amyloid buildup, they would be able to prescribe Leqembi. The drug is given intravenously every two weeks for 18 months. A doctor would monitor a patient closely for side effects.
Infusion centers throughout Florida are gearing up for the demand.
“In certain areas, I anticipate we will receive probably 15% to 20% more patient referrals for this drug,” Sue Rottura, chief operating officer of Vivo Infusion told CNN. “We know in clinics in Florida that we may have to increase capacity at those clinics.”
Leqembi differs from oral medications such as donepezil (Aricept) or memantine (Namenda), which only treated symptoms but not the underlying cause, notes Dr. James Galvin, a neurologist and chief of the Division of Memory Disorders with the University of Miami Health System and the Miller School of Medicine. Leqembi works in a way that is similar to a previously approved IV medication called Aduhelm, but Leqembi had stronger clinical trial data.
As part of the rollout of Leqembi, the Centers for Medicare & Medicaid Services will maintain a registry of medical professionals in the coming months who are giving out the medication. The Alzheimer’s Association also will have a registry and a help line. (www.alz.org/alznet or 1-800-272-3900).
What is the cost of Leqembi?
The first step is getting a diagnosis. Most insurers cover the cost of a spinal tap to diagnose amyloid plaque in the brain, but they do not cover the cost of a PET scan, which is less invasive. A PET scan could cost as much as $5,000.
Keith Gibson, director of diversity, equity, and inclusion at Alzheimer’s Association in Fort Lauderdale, said efforts are underway for the federal Centers for Medicare & Medicaid Services to fully cover PET scans. For now, it could be covered for people, particularly minorities, who are willing to enroll in certain clinical trials.
Once you have a diagnosis, there likely will be additional out-of-pocket costs.
Until now, Floridians who received Leqembi had it fully paid for as part of a clinical trial. Some clinical trials sites still are involved in ongoing research studies with Leqembi, studying alternate dosing strategies and delivery methods as well as long-term monitoring of effectiveness and safety. Some of those clinical trial sites remain in Florida.
Dr. Mark Brody, founder and principal investigator at Brain Matters Research in Delray Beach, says the advantage to getting Leqembi outside of a trial is you know you are getting the medication rather than a placebo.
The medication outside of a trial may be expensive. Medicare likely will pay 80% of Leqembi’s $26,500 cost for a year’s supply of IVs every two weeks. Patients, however, could still be on the hook for between $5,000 and $6,600 in co-payments. Medicare also requires doctors to submit their patients’ information to a national registry while they are being treated with the drug. The Centers for Medicare & Medicaid Services says the information in the registry will be evaluated to learn more about Leqembi’s benefits or risks.
How manageable are potential side effects?
Every patient will have to weigh the benefits vs. the risks, doctors say.
Some patients in clinical trials experienced infusion reactions to the medication but these were generally mild, said Dr. Brody at Brain Matters Research. “About 25 percent of people will get an infusion reaction, an allergic reaction that’s controllable. We give them antihistamines and low-dose steroids.”
Other side effects are more serious and can even be fatal, which is why doctors will monitor patients regularly with an MRI scan during the 18-month course of treatment.
“Some patients will experience brain bleeding or swelling in the brain as a side effect,” said Dr. Mark Agronin of Miami Jewish Health, an Alzheimer’s expert whose clinic participated in the Leqembi trials. “Those side effects usually improve once treatment is stopped.”
People who are more at risk of that happening are those on blood thinners, those who already have microscopic bleeds in the brain, and those with an Alzheimer’s-linked gene mutation called APOE4, he said
“You want to be in hands of an expert to have these side effects managed … that’s critical,” Agronin said. ” A primary care physician can be involved, but you need to be diagnosed and managed in a comprehensive way.”
In the national trial, about 13% of patients receiving Leqembi experienced brain swelling. Doctors say the most common signs that indicated swelling were dizziness and headaches. Brody at Brain Health Matters says the swelling generally resolved within eight to 12 weeks, and his clinic then resumed the treatment.
Because of that side effect, the FDA issued a warning in the prescribing information to alert people to the potential risks..
“For the most part, side effects are controllable if you are in experienced hands,” Brody said.
What if you have later-stage Alzheimer’s?
On its website, manufacturer Eisai makes it clear “there are no safety or effectiveness data on initiating treatment at earlier or later stages of the disease than were studied.”
Crocco, at the University of Miami, said all research on different treatments thus far shows there is no way to undo moderate to severe damage to the brain. “We have been researching for years, and nothing helps advanced Alzheimer’s,” she said. “There’s something going on at that level in which you can not reverse damage to the brain.”
She advises family members and patients to see a doctor at the first sign of memory loss.
“I encourage anyone with a family history who is starting to have memory loss not to wait,” Crocco said. “Get an evaluation early on because now there are things we can do.”
Should you start Leqembi if there’s a family history of Alzheimer’s?
Crocco advises against using the drug if there are no indications of memory loss.
“If you have a family history but no symptoms, it’s not wise to take Leqembi at that time,” she said. “There’s no guarantee a person will ever get symptoms, even if they have amyloid in the brain, so the risks might not be worth it.”
What happens after your initial 18-month course of Leqembi?
No one knows yet whether some patients will benefit from taking Leqembi longer than 18 months.
“We don’t know what would happen if you continue. The research only looked at results after 18 months. We just don’t have longer-term data,” said Brody at Brain Matters Research. “This is a terminal disease, and without intervention, it gets worse. There are no exceptions. I think doctors will look at how can we keep you as good as we can for as long as we can without making you sick.”
Each doctor may independently decide how long to treat a patient with Leqembi.
“While 18 months is recommended, I think certain physicians will make that choice to continue further based on whether the patient is benefitting,” said Crocco at University of Miami. “I don’t think it’s a hard stop.”
What role will blood tests play?
In the near future, a simple finger-prick blood test may help detect Alzheimer’s. There is a lot of buzz about new research that will be released during the Alzheimer’s International Conference about using blood tests to find biomarkers for Alzheimer’s disease.
“This is super exciting because it’s less invasive,” said Gibson with the Alzheimer’s Association. “Blood tests may eventually take the place of PET scans. We are close to having information to present to the FDA.”
Meredith Clements, 38, of Fort Lauderdale, has a family history of Alzheimer’s and a gene that shows predisposition. She said she would get a blood test if it were available. And if she shows signs of memory loss, she will consider Leqembi.
“This disease is an epidemic, and there are not a lot of treatments that have been approved,” Clements said. “Whether contributing to research or for my own benefit, I remain open to all options.”
Sun Sentinel health reporter Cindy Goodman can be reached at firstname.lastname@example.org