The Neurobiology of Aging and Alzheimer Disease in Down Syndrome - Chapter 16: Clinical trials for Alzheimer’s disease in Down syndrome

The strong relationship between genetic determinants of Alzheimer’s disease (AD) and the overproduction of beta-amyloid (Aβ) provides compelling support for the amyloid cascade hypothesis and has been central to AD drug development. It is hypothesized that disease-modifying treatments for AD should begin prior to the development of extensive neurodegeneration and onset of AD-related cognitive symptoms. Understanding of the molecular basis for the near universal presence of early onset AD in Down syndrome (DS) has grown exponentially in the past decade and recent studies have provided evidence that AD in DS is remarkably similar to other forms of AD. Individuals with DS therefore comprise the largest group in the world with genetically determined AD. In this chapter, therapeutic strategies that can be employed in clinical trials for AD in DS will be discussed as well as their underlying scientific rationale.