The Food and Drug Administration recently approved a new drug called Aduhelm for treating Alzheimer’s disease. It’s the first therapy approved for the disease since 2003. But will it provide the relief that patients and their families are seeking?
According to Yale New Haven Hospital geriatrician Gerard Kerins, MD, patients and families may want to hold off on celebrating just yet.
Alzheimer’s disease, which afflicts more than 6 million Americans, is a progressive and irreversible condition that robs people of their memory and ability to do everyday tasks. Scientists believe a buildup of a sticky plaque called amyloid beta in the brain is a hallmark of Alzheimer’s. “These clumps of plaque become toxic, which is thought to affect memory and correlate with dementia symptoms,” said Dr. Kerins.
Most of the handful of medications approved for Alzheimer’s treat symptoms and show modest effects at best. Aducanumab, also called Aduhelm, is the first drug designed to slow the Alzheimer's progression, not just ease symptoms. The treatment is a monoclonal antibody – a laboratory-made protein that can bind to substances in the body – originally derived from the cells of older people without signs of cognitive problems.
“The thought is that by binding to the plaque and breaking up the clumps of amyloid beta, Aduhelm will reduce inflammation and slow cognitive decline,” said Dr. Kerins.
So why isn’t Aduhelm being hailed as a miracle breakthrough? According to Dr. Kerins, there are still a lot of questions about the drug. In two large clinical trials for early-stage patients, evidence did not convincingly show dramatic clinical benefits. However, the trials appear to indicate that Aduhelm may be effective at removing plaque. The FDA concluded there was a “reasonable likelihood” that a reduction in amyloid would slow disease progression, so it approved the drug using its “accelerated approval” program. That program allows for earlier approval of a drug for a serious or life-threatening illness, even though more study might be needed. Aduhelm is the first Alzheimer’s drug to receive FDA accelerated approval.
“There hasn’t been an effective treatment for Alzheimer’s approved in almost 20 years, so patients and families are understandably anxious for new drugs,” Dr. Kerins said. “There was a lot of pressure on the FDA to approve this.”
There are challenges associated with Aduhelm, he said. Patients receive the drug by IV transfusion every four weeks, a process that takes about 45 minutes. Patients must have regular MRI scans because of side effects, which may include headaches, vision changes and hemorrhaging or swelling in the brain. Aduhelm can cost up to $56,000 a year per patient, and there are still questions about whether insurance will cover it.
The biggest challenge may be that the drug was developed only for patients with mild cognitive impairment, not severe dementia.
“This may limit the number of patients who might actually benefit from it, because it takes time to evaluate patients to determine whether their memory loss is caused by Alzheimer’s or another type of dementia,” Dr. Kerins said.
Asked if he would use the drug with his patients, he replied, “It will depend on the patient, their support, their level of functioning and the expectations of their families. If it helps some patients and gives them valuable time with their families, it will be a wonderful thing.”
Overall, Alzheimer’s care is best provided using a team-based approach, he said. “This includes caring for the caregivers and being available as issues arise. Medications are just one aspect of the care regimen.”
This article appeared in Advancing Care, a monthly, electronic publication with information from YNHHS experts on a variety of medical topics, along with upcoming lectures, health screenings and more.
