Many of you may still have this phrase ringing in your ears if you traveled with children for any length of time this past holiday season. Representatives from the ACT-AD Coalition, chaired by the Alliance for Aging Research, heard these same words almost two years ago, not uttered from the mouths of babes, but rather by prominent officials at the Food and Drug Administration (FDA) in a conversation regarding evidence to support the use of biomarkers for Alzheimer’s disease in clinical trials for “disease-modifying” therapies.
A group of top Alzheimer’s experts systematically surveyed peer-reviewed literature in this area and determined that yes, “we are there” with several established biomarkers. Cerebrospinal fluid assays that detect amyloid-beta 42 and tau, positron emission tomography (PET) that detects the AB deposits in the brain, and magnetic resonance imaging and PET that detect AD-related neuronal loss in the brain, are sufficiently validated for incorporation in AD trials. The Working Group has published their review in the Neurobiology of Aging. This is good news for those waiting for better treatments and a cure, as well as those conducting research. Biomarkers can improve the way clinical trials are designed by providing greater precision in measuring dementia progression, allowing for selective enrollment of patients in trials requiring fewer participants and less time to demonstrate a treatment effect, and enhancing confidence that patients are accurately diagnosed with Alzheimer’s Disease. Since all clinical trials to date of “disease-modifying” compounds in AD patients have failed to show benefit, we hope the Working Group's review will serve as a guide for the FDA and industry on how best to proceed with designing and executing future AD trials.