At frighteningly young ages, in their 40s, four of Laura Cuartas’s children began forgetting and falling apart, assaulted by what people here have long called La Bobera, the foolishness. It is a condition attributed, in hushed rumors, to everything from touching a mysterious tree to the revenge of a wronged priest.
Since all our efforts so far to treat Alzheimer’s — in people already suffering from the disease – had pretty much come to naught, it seemed like a goldmine for scientists to find this family group plagued by an inherited genetic mutation that caused the debilitating dementia by middle age. Could researchers tap this troubled gene pool and figure a way to PREVENT the disease altogether, in a population known in advance to be acutely susceptible? The possibilities seemed fascinating, and encouraging.
Last fall, the New York Times updated its story. The new, large, and novel study to assess the possibility of preventing the disease was underway. Family members had begun travelling from their homes around Medellin, Colombia, to the Banner Alzheimer’s Institute in Phoenix, AZ, for PET scans to determine the presence of amyloid plaques, those tell-tale protein accumulations that typically accompany AD, and are thought to play a causative role.
Study partners from the National Institutes of Health, biotech giant Genentech, and the Banner Alzheimer’s Institute created their methodology. About 100 people who carry the presenilin 1 mutation would receive the drug therapy. About 100 mutation carriers would receive a placebo. And about 100 non-carriers would also receive the placebo. Most participants have chosen not to know their mutation status, a fact that helps keep results “clean.” The study also includes a small group of Americans whose families have shown a predisposition toward AD.
The Attack Drug: Crenezumab
A final piece in the study was selecting the drug therapy. Scientists decided to use crenezumab, described on the Genentech site as a “humanized monoclonal antibody… designed to bind to amyloid beta (Abeta), the main constituent of amyloid plaque in the brains of patients with Alzheimer’s disease.” Quite simply, the drug is designed to prevent the plaques from forming in the brain.
I’m excited to learn the outcome of this study, one of the most interesting I’ve encountered in my research. It shifts the current paradigm from reversing the symptoms of Alzheimer’s in people already afflicted to preventing the disease in healthy people likely to develop the dementia. And the study – if all the pieces fall into place – might just prove once and for all the role played by amyloid plaques in the development of the disease.
