Bio-Markers for Developing Alzheimer's Treatments

Contributed by: Dennis Fortier, President, Medical Care Corporation

It's been a long time coming, but the FDA might finally have enough evidence to allow bio-marker measures into clinical trials for Alzheimer's treatments.  This change could potentially speed the trial process, reduce the risk of failed trials, and yield new AD therapies in the near future.

Based on an analysis of prior publications about bio-markers for Alzheimer's disease, an expert panel convened by the Alliance for Aging Research and the FDA, found ample scientific evidence for including changes in bio-markers as outcome measures in FDA clinical trials.  This means that, rather than relying solely on measures of cognition and function (which are measures of worsening symptoms), a drug could now be approved based on its ability to prevent pathological changes that are only evident through an examination of bio-marker measures.

An obvious precedent for this approach comes from the cholesterol field.  Since high cholesterol is a condition with no immediately apparent symptoms, a trial based on symptomatic improvement would always fail.  To approve cholesterol reducing drugs, the FDA allowed trial designs that used blood-based measures of cholesterol as a bio-marker, in lieu of any symptoms produced by a high cholesterol level.

It has long been known that Alzheimer's disease is characterized by a series of pathological changes.  These changes progress from shifting protein levels in the spinal fluid, then to lesions in the brain, and eventually to brain atrophy.  However, the precise relationship between the disease and these pathological changes had not been well enough understood for the FDA to allow their measures as part of a clinical trial.  With a massive research focus over the past five years and what appears to be an objective, expert review of the evidence, it appears as though future trials may well include bio-marker measures as critical trial outcomes.

Many Alzheimer's related stories get massive press coverage, despite minimal importance in the grand scheme of our efforts to thwart the disease.  This story has been relatively uncovered in the mainstream, but might well be one of the most important stories of the year.