A new drug called aducanumab shows promise as a potential treatment for Alzheimer’s disease, but it first must overcome some regulatory hurdles.
Those involved with Alzheimer’s care and treatment still say they are buoyed by the drug’s potential, but it is unclear yet what path will ultimately emerge for aducanumab. “We have huge hope that it could work, and people need hope,” the AARP’s senior vice president for policy Sarah Lenz Lock said in an AARP article.
Lenz is also executive director of the Global Council on Brain Health from the organization. “But it’s really important for the FDA to play this very scientifically accurate. We’re relying on the FDA to make a wise decision,” Lenz added.
Alzheimer’s disease affects the brain by causing widespread damage to its neurons. Neurons are cells that send messages to other parts of the brain. Although the brain naturally undergoes some neuron changes with age, Alzheimer’s disease causes more serious and far-reaching damage to neurons, according to the National Institutes of Health. Initially, Alzheimer’s affects neurons related to memory. With time, the disease affects parts of the brain responsible for language, reasoning, and social behavior. The disease damages even more brain areas over time, causing a person with the disease to lose their ability to live and function on their own.
Alzheimer’s disease, which is the sixth leading cause of death in the U.S., has no cure. There are drugs that target certain symptoms of Alzheimer’s disease, but the last one was approved in 2004.
That’s what makes the news about aducanumab, a human monoclonal antibody, potentially exciting for medical professionals who treat Alzheimer’s and the families of those affected by the disease. Made by the company Biogen and Eisai, aducanumab targets beta-amyloid, a brain compound thought to play a major role in the disease. This makes it the only potential drug to target a cause of Alzheimer’s and not just its symptoms. Researchers believe it could potentially be used by the estimated 2 million Americans with mild Alzheimer’s related cognitive decline. Patients would take aducanumab by a monthly IV infusion at a cost of about $50,000 a year.
Aducanumab does not cure Alzheimer’s, but it could potentially provide a treatment stronger than anything else available.
Researchers have shown excitement over aducanumab despite some mixed Phase 3 trial results. Only one of two Phase 3 trials had positive results. “Those results emerged only from an analysis of additional data after the trials were stopped in March 2019 by an independent data monitoring committee because the drug didn’t appear to be working,” according to the New York Times. Later on, Biogen announced a new analysis of the data that showed patients who received the drug had a significant reduction in clinical decline in that same trial.
On Nov. 6, members of the Food and Drug Administration Peripheral and Central Nervous System Drugs Advisory Committee ultimately did not support data showing aducanumab’s effectiveness. Of the 11 panel members, 10 of them voted that the available research was not enough to show the effectiveness of the drug. (The eleventh member was uncertain.) Prior to the vote, Biogen’s stock rose 40% upon hopes of a more favorable view of the drug.
Alzheimer’s experts still raise questions about the drug, such as:
- Who can ultimately receive the drug based on their Alzheimer’s diagnosis?
- Where would patients go to get the drug infusion?
- What other non-drug interventions will play an important role? Those could include blood pressure control, sleep, diet, and exercise.
- Will insurers cover the cost of the drug?
The advisory committee’s recommendation doesn’t mean the drug won’t be approved, but it does give regulators food for thought about the drug. Food and Drug Administrators are expected to make a final decision about aducanumab in March 2021. They don’t have to follow recommendations of the advisory panel although they will usually do so, according to AARP.