The world of developing drugs to fight beta-amyloid has been on a rollercoaster ride the last year, but some scientists aren’t giving up. Researchers at the University of Southern California (USC) are launching a new clinical trial in the next few months to test a drug that aims to battle beta-amyloid in people who have not yet developed Alzheimer’s symptoms.
The drug, known as BAN2401, is an antibody that’s designed to stick to beta-amyloid plaques in the brain. By binding to amyloid, the drug would help the immune system pinpoint these toxic proteins and eliminate them.
In a previous phase 2 trial, researchers found that BAN2401 was able to remove amyloid from the brain, and they hope it may reduce cognitive decline associated with Alzheimer’s.
Is There Hope in Attacking Beta-Amyloid?
Beta-amyloid, the protein that’s one of the main signs and drivers behind neurodegenerative diseases, has been a big focus for researchers and drug developers working to find a treatment for Alzheimer’s.
But there have been some disappointments in recent years. In March 2019, Biogen cancelled a trial investigating its drug aducanumab, which aimed to fight amyloid. In a surprising reversal in October 2019, Biogen announced it would be bringing the drug back and submitting it for FDA approval.
In December 2019, researchers released data on aducanumab, arguing that after further analysis, it did show evidence of slowing progression of the disease by reducing amyloid. The new trial will begin recruiting participants in March 2020, but some experts remain skeptical about aducanumab’s efficacy.
Despite aducanumab’s alleged success, there have been several other letdowns. Just last week, two amyloid-fighting drugs developed by Eli Lilly and Roche failed in clinical trials.
These setbacks aren’t discouraging Dr. Paul Aisen, director of USC’s Therapeutic Research Institute, and an investigator on the new trial. What makes this trial stand out from past beta-amyloid studies, he said, is the fact that it will target amyloid in the brains of people who are in a “pre-clinical” stage of Alzheimer’s. In this stage, there’s evidence of amyloid accumulation but no symptoms yet—a state that can last years, and even decades, leading up to the disease.
“What this trial is doing is administering this antibody to individuals who are at a very early stage in the Alzheimer’s process,” Aisen said in an interview with Being Patient. “It’s based on the idea that the best way to attack the Alzheimer’s cascade is when the brain is still able to function normally.”
In a press release, he added that “amyloid deserves the attention we have given it. Amyloid accumulation begins the disease process, and it predicts progressive cognitive decline to dementia. All of the known genetic causes of Alzheimer’s disease are tightly linked to amyloid accumulation.”
Recruiting For the Alzheimer’s Trial
The researchers plan on recruiting some 9,000 participants across 100 sites initially, who will then be screened for the criteria of being in this pre-clinical stage. By using PET scans, the researchers can identify which of these cognitively normal people have elevated levels of amyloid in the brain, putting them at risk for dementia.
There will be some 60-70 sites recruiting in North America, and more sites in Japan, Singapore and Australia. Once screened and randomized, around 1,400 participants will begin receiving the drug in the next few months—likely over the summer, Aisen said. They will receive the drug over the course of four years.
“We are optimistic that the drug will slow or stop the cognitive decline that can be measured over this period of time even in clinically normal people,” Aisen said.
Aisen noted that people can sign up for the trial evaluation process online. The Alzheimer’s Prevention Trial Web study can be found here.