‘I think this is a very big deal.’ New Alzheimer’s drug gets full FDA approval

Nicholas Milano, M.D., can’t wait to give the new Alzheimer’s drug, lecanemab, brand name Leqembi, to qualified patients at MUSC Health. “I think this is a very big deal,” the neurologist said of the drug’s full approval by the Food and Drug Administration.

“This is technically the second medication that's been approved as a treatment in recent years. Aducanumab, brand name Aduhelm, got accelerated pathway approval two years ago. But it has not yet received traditional approval because questions remain about its clinical benefit. Leqembi, the newer medication, has much clearer evidence of an actual clinical benefit.”

Milano, an associate professor of neurology at the Medical University of South Carolina, described how Leqembi appears to work. “This medication is a monoclonal antibody. An antibody is something that your immune system makes to go after infections or other diseases. We're giving the body this antibody, which then attaches to amyloid, the abnormal protein believed to be the primary driver of Alzheimer's disease,” he said.

“When the antibody latches onto the amyloid, that is a signal for your immune system to clear it out. Studies have shown that this medication does clear out amyloid from the brain.”

 

But that’s not the whole story, Milano said. “The question is how much clinical benefit a patient gets from this drug. Because when you clear out amyloid, in theory, it should stop or slow down the disease. And the studies on Leqembi do show that the disease does slow down, but it doesn't stop.”

With that in mind, Milano said the medication shouldn't be seen as a cure for Alzheimer's. “I think this is a starting point for additional medications in the future to really continue to fight the disease.”

But that starting point is welcome news for some Alzheimer’s patients, their families and their doctors who previously could only treat the disease’s symptoms. So MUSC Health has been gearing up for Leqembi’s approval. “We're working on this really intensely. We're collaborating with different specialists – radiologists, psychiatrists, primary care physicians – to come up with a comprehensive plan,” Milano said. 

In fact, with funding from the MUSC President’s Council, Milano and his team have been looking at best practices in Alzheimer’s disease care in the United States and the European Union. They’ve also proposed a network approach – currently known as the South Carolina Alzheimer’s Network, or SCAN – which they hope to begin building this summer. “We are getting ready to respond to the needs of our patients and their caregivers,” Milano said.

For now, they’re focused on getting the new Alzheimer’s medication to the people who need it, a process that requires multiple steps. “The general idea is that we're going to ask that patients have the required initial studies, which would include an MRI scan of their brain, along with laboratory studies, looking for alternative causes of memory loss before we even see them.”

If there’s an alternative cause, the patient wouldn’t qualify for Leqembi. Even if there isn’t one and the patient does have Alzheimer’s, Milano said timing is important.

“Once someone has progressed to a moderate or severe dementia, unfortunately, they're no longer a candidate for this medication. So right off the bat, we would be focusing on patients that have either mild cognitive impairment, which is the predementia stage, or early-mild dementia. And then if you meet the initial criteria, you'll get a special brain scan called an amyloid PET scan, which looks for amyloid in the brain,” Milano said.

“If you have a positive amyloid PET scan, your history fits and you don't have any other contraindications, you would be a candidate for the medication. We would bring you in, and you would be evaluated by a neurologist, who would then prescribe the medication.”

Milano said academic medical centers such as the Medical University of South Carolina are good sites for giving the medication because they have the neurology expertise, support staff and systems in place to meet Leqembi’s requirements.

“Any patient who gets the medication will have to be part of a registry. Their physician will have to enter information into this registry so that we can learn about the disease and its long-term benefits and side effects. So there is going to be a little bit of a hurdle there, as the prescribing physician will have to do some extra work to get all the data into the registry.”

Patients will also need to come in every other week for an infusion. Doctors will monitor them closely. 

“Patients will have to have multiple MRI scans of their brain during the first six months of getting this medication because of the potential significant side effects. These medications should be prescribed by physicians who can recognize and handle these unique side effects in the brain, and in most cases that will be a neurologist.”

When it comes to paying for the drug, Milano said it’s unclear how Medicare will handle the specifics. “We also don't know what other insurance companies are going to do with it. So we are still looking at probably later in the summer before we'll be able to actually offer this medication. But I would assure everyone that we're going to offer it as quickly as possible.

He said if patients think they may be candidates, they should get in touch with their regular doctors as soon as possible.

“Their primary doctors can then contact us, and we'll determine if they are appropriate candidates or not. But don’t wait. I think it is important for people to know that, like with a stroke, ‘time is brain.’ So the longer you don't receive this medication, the further the disease will progress."