$33M NIH study will explore a new drug to prevent Alzheimer's

Over the past year the repeated failure of late-stage Alzheimer's drugs has pushed the R&D community away from patients with a fully developed case of the disease toward early-stage or high-risk patients who could benefit the most from therapies that would prevent the disease from wreaking havoc in the brain. To that end, the Banner Alzheimer's Institute in Phoenix has landed a $33 million NIH grant to see if an anti-amyloid therapy can prevent the memory-wasting condition from getting a grip on patients.

The study will be part of the Alzheimer's Prevention Initiative, a collaboration of prominent players looking for something that can work against the disease. Investigators will recruit 650 healthy patients between the ages of 60 and 75 with two copies of the apolipoprotein E (APOE4) gene--a genetic marker for people at high risk of developing Alzheimer's.

"We are now looking at potential treatments to prevent both the early and late onset forms of the disease," said Dr. Pierre Tariot, BAI director. "This kind of comprehensive approach could prove the tipping point in our long, arduous effort to find a way to end this devastating disease."

The new work builds on a plan to test 300 Colombians--all from the same family tree and all sharing an uncommon risk of developing Alzheimer's. Hoping to show that its amyloid-inhibiting antibody crenezumab could slow down progression of Alzheimer's in at-risk patients who don't yet have dementia, Genentech has been actively engaged in this effort. Genentech's antibody is part of a wider 5-year trial funded by the biotech giant, the NIH and Banner Alzheimer's Institute. The groups announced plans for the trial in 2012.

Investigators have been making slow but steady progress in the global effort to determine exactly what causes Alzheimer's and how drug developers could effectively target the disease. Just today a team of investigators at the National Institute of Radiological Sciences in Chiba, Japan, reported that they had developed a chemical that binds to tau--a toxic protein long thought to be linked to Alzheimer's--lighting up the tangles that grow in the brain and making them visible through PET imaging technology. Similar work has also been done already on amyloid beta, which has been the favorite culprit studied most closely by the pharma groups engaged in the field.

While billions of dollars have been spent on Alzheimer's therapies, no drug has made it through Phase III. Most notably solanezumab from Eli Lilly ($LLY) and bapineuzumab from Johnson & Johnson ($JNJ) and Pfizer ($PFE) flunked late-stage testing, sending investigators back to the drawing board to try and figure out new approaches to the disease.

- here's the press release
- read the story on tau imaging from the BBC