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New Alzheimer's Disease Trial Will Focus on Preventive Research

A large extended family in Colombia, where an elderly mother has become the caregiver for her 4 adult children with Alzheimer's, will be one of the main focuses of the trial.

For a large extended family in Colombia, early-onset Alzheimer’s disease has reversed the familial roles and turned an elderly mother into the primary caregiver for four of her middle-aged children. Intermarriage among larger families has caused the disease to spread quickly. Now, this family—part of thousands of others among a sprawling group of relatives in the same Andes mountains region—may become the center of a new clinical trial in both the United States and Colombia that will focus on preventive research.

Preventive research on Alzheimer’s disease has been difficult thus far, according to Dr. Neil Buckholtz, chief of dementias of aging at the National Institute on Aging, because participating individuals need to be guaranteed, or highly likely, to develop dementia, “and with common Alzheimer’s identifying such people is challenging because the disease’s cause is unknown.” Also, because people would not be sick when they are treated, researchers are particularly worried about serious side affects. Thus, according to Buckholtz, Colombia “seems to be the best option” for such a trial, because mutation carriers among the extended family always lead to Alzheimer’s disease, and the affected people are younger, allowing for clearer brain images.

Scientists in Colombia are recruiting participants and hope to start testing early next year. Family members, who would roughly be age 38-45 years, would be gene-tested, given treatment or placebo, and monitored with memory tests, brain scans, and other forms of evaluation. A companion American trial will include individuals age 60-80 years with what the researchers say is another rare trait: they have two copies of a different gene that substantially increases the risk of common late-onset Alzheimer’s disease. The treatment will attack the beta-amyloid protein, either in the form of a drug that destroys plaques or as a vaccine “that contains or encourages production of anti-amyloid antibodies.”

According to Dale Schenk, chief scientific officer for the Elan Corporation, if the project prevents even one early-onset patient from developing the disease, that is a major victory.

“It’s not even debateable whether this should be done,” Schenk said. “It has to be done.”