BACE inhibitor for Alzheimer's disease goes on trial in at-risk adults

By John Murphy, MDLinx
Published June 17, 2016

Key Takeaways

A clinical trial is recruiting subjects to test a novel drug aimed at stopping Alzheimer’s disease before it starts to damage the brain, according to researchers at the University of Southern California Alzheimer’s Therapeutic Research Institute (USC ATRI), in San Diego, CA.

The drug—beta-secretase (BACE)—is an enzyme that inhibits the cleavage of amyloid precursor protein (APP), which prevents the creation of amyloid-beta plaques in the brain. The researchers presume this would slow or stop Alzheimer’s disease.

For this phase II/III randomized trial, they’re recruiting older adults who don’t have symptoms of Alzheimer’s but are at risk of developing the disease.

“We are now looking at the stage of Alzheimer’s that precedes even mild symptoms,” said Paul Aisen, MD, Founding Director of USC ATRI and Professor of Neurology at the Keck School of Medicine of USC. “It is our view that drugs such as BACE inhibitors may be most effective at the earliest stages of the disease.”

In March 2015, researchers from Janssen Pharmaceuticals (which is jointly managing this phase II/III trial with USC ATRI) reported phase I results of the BACE inhibitor in humans. The smallest dose of the drug (5 mg) reduced by half the most common isoform of amyloid-beta in the cerebrospinal fluid. A 25 mg dose brought amyloid-beta down by 80%, investigators reported.

The current clinical trial is now enrolling more than 1,600 people at sites in Europe, Japan, and Australia, as well as 660 participants at 75 sites in North America. To be eligible, participants must be age 60 or older and have no clinical symptoms of Alzheimer’s disease.

Applicants will be tested for amyloid accumulation in the brain and, if positive, will be invited to participate in the trial. The study will assess cognitive performance, along with other measures related to Alzheimer’s, over time.

Further details of the study can be found on

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