Alzheimer’s: US approves new drug that could slow the disease | Science and health

Leqembi, of Japan’s Eisai and US partner Biogen, has achieved rare success in a field accustomed to failed experimental treatments for a disease seen as incurable. Although the drug only manages to delay cognitive decline by a few months, some experts say the drug can still significantly improve people’s lives.

“This drug is not a cure. It doesn’t stop people from getting worse, but it does slow the progression of the disease considerably,” said Joy Snyder, MD, a neurologist at Washington University in St. Louis. Louis. “This could mean someone could have another six months to a year to be able to drive, for example,” he says. Snyder stressed, however, that the drug comes with drawbacks, including the need for injections twice a month and potential side effects such as brain swelling.

In late November, data released provided the first detailed look at the drug’s effects. A preview of the results, which ran into September, sent shares of the two companies soaring at the time.

At the time, experts were also excited about Leqembi’s results, but were also reticent about the drug’s effects. “The benefit is real, and so are the risks,” Jason Karlawish, co-director of the Penn Memory Center at the University of Pennsylvania, said at the time. He was not involved in the research.

Express approval

The FDA approval came via a bureaucratic shortcut that allows drugs to be released based on preliminary results, before they are confirmed to benefit patients. The agency’s use of this approach has been increasingly criticized by government oversight agencies and congressional investigators.

Last week, a congressional report found that the Food and Drug Administration’s approval of a similar Alzheimer’s drug called Aduhelm — also from Biogen and Eisai — was “riddled with irregularities,” including a series of meetings with drug company officials that were not documented. Closer scrutiny of the new drug, known chemically as lecanemab, likely means most patients won’t start taking it for months while insurance companies decide if and how to cover it.

The drug will cost about $26,500 USD (R$138,590) for a typical year of treatment. Eisai said the price reflects the drug’s benefit in terms of improving quality of life, reducing burden on caregivers, and other factors. The company has valued it at more than $37,000 (R$193,500) annually, but said it has lowered its prices to reduce costs to patients and insurers. An independent group recently claimed that the price of lecanemab would have to be less than $20,600 USD (107,730 BRL) per year to be profitable.

About 6 million people in the United States, and many more around the world, suffer from Alzheimer’s disease, a disease that gradually attacks areas of the brain needed for memory, thinking, communication, and everyday tasks.

reservations

The FDA approval was based on a mid-stage study of 800 people who showed early signs of Alzheimer’s disease and were still able to live independently or with minimal assistance. Eisai has since published the results of a larger 1,800-patient trial that the Food and Drug Administration will review to confirm the drug’s benefit, paving the way for full approval later this year.

The larger study tracked patients’ outcomes on an 18-point scale that measures memory, judgment and other cognitive skills. Clinicians compile the rating from patient interviews and close contact. After 18 months, the participants who received Leqembi had a slower progression of the disease — by less than half a point on the scale — than patients who received a placebo infusion. The “delay” was just over five months.

There is little consensus on whether this difference translates into real benefits for patients, such as increased independence. “Most patients won’t notice the difference,” said Matthew Schrag, a neuroscience researcher at Vanderbilt University. “This is actually a very small effect and is probably below the threshold of what we would call clinical significance.” Schrag and some other researchers believe that a significant improvement requires at least a whole point difference on the scale.

Leqembi works by removing a glial protein in the brain called amyloid, which is a hallmark of Alzheimer’s disease, and the reason for this is still not clear. Many other amyloid-targeting drugs have failed, and many researchers now feel that combination therapies will be necessary.

Aduhelm, the similar drug, has been marred by controversy over its effectiveness. The Food and Drug Administration approved this drug in 2021 against advice from the agency’s outside experts. Doctors were reluctant to prescribe the drug, and insurance companies tightened coverage. The FDA did not consult the same expert panel before approving Leqembi.

While there is “less drama” surrounding the new drug, Schrag said many of the same concerns apply. He asks, “Is this mild, measurable benefit worth the high price of side effects patients may experience?” “I have serious doubts.”

Aduhelm, the similar drug, has been marred by controversy over its effectiveness. The Food and Drug Administration approved this drug in 2021 against advice from the agency’s outside experts. Doctors were reluctant to prescribe the drug, and insurance companies tightened coverage. The FDA did not consult the same expert panel before approving Leqembi.

While there is “less drama” surrounding the new drug, Schrag said many of the same concerns apply. He asks, “Is this mild, measurable benefit worth the high price of side effects patients may experience?” “I have serious doubts.”

Side effects

About 13% of patients in Eisai’s study experienced brain swelling and 17% experienced minor bleeding in the brain, side effects seen with previous amyloid-targeting drugs. In most cases, these problems do not cause symptoms that can include dizziness and vision problems. In addition, several Leqembi users have died while taking the drug, including two who were taking blood-thinning medications.

Esay said the deaths could not be attributed to drugs. The FDA label warns doctors to use caution when prescribing Leqembi to patients taking blood thinners. Insurance companies are likely to only cover the drug for people like those in the company’s study — patients with mild symptoms and confirmed amyloid buildup. This usually requires expensive brain scans. A separate type of scan will be needed to monitor brain swelling and bleeding periodically.

A major issue in the drug’s release will be the coverage decision for Medicare, the federal health plan that covers 60 million seniors and other Americans. The agency severely restricted coverage of Aduhelm, essentially eliminating its market in the United States and prompting Biogen to abandon plans to market the drug.

Eisai executives said they spent months discussing their drug data with Medicare officials. Coverage is not expected until the Food and Drug Administration confirms the drug’s benefit, likely later this year. “Once we make a decision about Medicare, we can really roll it out across the country,” said Eisai US CEO Evan Cheung.

Betsy Groves, 73, of Cambridge, Massachusetts, was diagnosed with Alzheimer’s disease in 2021. The former Harvard School of Education professor noted that she was having trouble remembering the names of some students and answering questions. Her initial diagnosis, based on cognitive examination, was later confirmed by a positive test for amyloid.

She says she’s “more than willing” to try Leqembi, despite the potential side effects and need for injections. “For me, the moment a drug hits the market — and I get my doctor’s approval — I’m going to take it,” he says.