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Pierre Tariot, MD Headshot

Unraveling Alzheimer's: A Step Forward

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Imagine living with the knowledge that inside your brain you carried a flaw that meant that you could be stricken with Alzheimer's as early as age 45, or even earlier.

This highly unlikely scenario is actually the harsh reality for an extraordinarily large family kindred of several thousand people in Colombia who carry a rare genetic mutation. But the misfortune that has haunted this clan for generations could hold a key to the eventual prevention of this frightening disease.

Recently, we have begun a groundbreaking research trial involving more than 300 cognitively healthy volunteers from these unfortunate families. The trial, which the National Institutes of Health has called a cornerstone in the National Plan to Address Alzheimer's Disease, relies on genetic clues that could help stave off the disease long before symptoms would manifest.

This unique effort arises from a paradigm shift within the Alzheimer's community that has resulted in a new level of cooperation among members of the scientific world, regulatory agencies, industry partners and Alzheimer's advocates. Researchers have long expressed hope for the so-called amyloid hypothesis, which suggests that an accumulation of this protein may play a key role in the disease's progression. Our aim in the clinical trial is to block the accumulation of amyloid. Our NIH-assisted study represents a collaboration among the Banner Alzheimer's Institute in Phoenix, the highly regarded University of Antioquia in Colombia, and California-based Genentech, a member of the Roche Group. This exciting partnership crosses the usual public-private boundaries and has led to innovative ways of leveraging resources and data sharing to a degree previously unthinkable.

The Colombian volunteers will receive an injection of an experimental antibody called crenezumab, or a placebo, at set intervals for up to five years. Researchers will use advanced imaging techniques, cerebrospinal fluid tests and sensitive cognitive measures to monitor whether the accumulation of amyloid and other tell-tale proteins in the brain is reduced, whether brain size and function is maintained, and, most importantly, whether mental performance is preserved.

This is only the first step in a long journey and we know the stakes -- and the potential rewards -- are very high. More than 5 million Americans currently live with Alzheimer's and a new case is diagnosed approximately every 69 seconds. Nationally and globally, the disease is accelerating at an alarming rate. The fiscal threat to Medicare and Medicaid due to this burden of patients is extraordinary, in addition to the heartbreaking toll it takes on entire families around the world.

We are cognizant of the responsibility that we face, not just to the scientific community but to the families involved. This unprecedented trial would be nothing but a theory without the individuals who have stepped forward and bravely agreed to be part of such a demanding study. If this approach to fighting Alzheimer's is successful, their risk could help transform all future prevention and treatment research, and herald the beginning of the end of this devastating disease.