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We’ve Had Terrible Luck Trying to Cure Alzheimer’s. But What If We Could Prevent It?

November 2, 2017, 9:42 PM UTC

The story of potential Alzheimer’s cures has been pretty depressing over the last couple of decades. But what if, instead of focusing on just treating Alzheimer’s and dementia after the brain-eating conditions have already set in, we could actually prevent it in the first place?

That’s the marquee goal that drug making giants Novartis and Amgen are striving for in a project with the Banner Alzheimer’s Institute (BAI). The groups are expanding their collaboration on the Alzheimer’s Prevention Initiative (API) by launching its second stage, they announced on Thursday.

Novartis, Amgen, and BAI are trying to figure out whether or not an experimental treatment called CNP520 can delay Alzheimer’s in the people at high risk for the disease—or prevent it altogether. This is the second generation of the API undertaking and will extend the study to a larger share of people who have the APOE4 gene, which is a major genetic risk factor for Alzheimer’s.

“Expanding our collaboration with Banner Alzheimer’s Institute stands testament to our belief that preventing amyloid buildup is one of the most promising approaches to treating Alzheimer’s disease,” said Novartis’ chief medical officer, global head of drug development, and soon-to-be CEO Dr. Vas Narasimhan in a statement. “If we determine that our [treatment] can prevent or delay the onset of symptoms in healthy yet high-risk populations, this would represent a tremendous breakthrough for those that may face this debilitating disease.”

The Alzheimer’s space hasn’t seen a new approved therapy in nearly two decades and has suffered a slew of high-profile failures in the last two years alone. Both Eli Lilly and Merck ditched late-stage Alzheimer’s drugs they’d spent tens or even hundreds of millions of dollars developing after late-stage clinical trial failures over the past 12 months. Biotech giant Biogen is hoping to break the curse with an all-in bet on its own experimental treatment aducanumab.

One of the biggest problems in Alzheimer’s drug development is uncertainty over the so-called “amyloid hypothesis”—the proposed model that a buildup of a kind of plaque called amyloid-β in brain tissue is at the root of Alzheimer’s and other dementia-related diseases. Even experimental treatments that target this plaque have had mixed results in preventing or reversing cognitive decline in patients.

But another approach could involve stopping the amyloid plaque from building up in the first place—and that’s what Novartis, Amgen, and BAI hope CNP520 may be able to do. The second generation of the Alzheimer’s prevention initiative will recruit 2,000 healthy participants at high genetic risk for the disease to see if the experimental treatment is more effective than placebo in blocking or stopping symptoms by targeting an enzyme that’s key to the production of amyloid-β.

“It is our hope that by targeting people earlier, we will have a better chance of delaying or preventing the onset of the disease,” said BAI director Dr. Pierre N. Tariot in a statement.