Alzheimer’s disease is characterized pathologically by the presence of
neuritic plaques containing β-amyloid and neurofibrillary tangles containing tau. These
have become two targets of investigational drugs aimed at preventing or slowing the
disease. Early findings of extensive cholinergic degeneration inspired the development
of drugs targeting cholinergic function. Cholinesterase inhibitors were the first drugs to
be approved for treatment, followed by the NMDA receptor antagonist memantine.
This chapter will focus on the development, mechanism of action, and results of
clinical trials for drugs currently used or in development for treatment of Alzheimer’s
disease. Examples of these include drugs targeting cholinergic neurons, such as
cholinesterase inhibitors, muscarinic receptor agonists and nicotinic receptor agonists,
as well as memantine. Several drugs aimed at reducing levels of β-amyloid or tau are in
development and will be addressed. Finally, drugs directed at other targets that may be
useful in treatment of Alzheimer’s disease will be discussed.
