Alzheimer’s disease (AD) is characterized by two types
of protein aggregates, neurofibrillary tangles and
amyloid plaques, distributed in regions of the CNS
that are involved in learning and memory. The
neurofibrillary tangles consist of twisted filaments
containing hyperphosphorylated tau whereas the
amyloid plaques contain mainly β-amyloid (Aβ)
peptide fibrils. Incomplete knowledge of the molecular
process that causes AD has hindered advances in drug
development. The available cholinergic therapies
target essentially late aspects of the disease,
improving temporarily the performance of the
undamaged neurones, but do not stop the progressive
mental decline. In the past years, important progress
has been made in the understanding of the pathogenic
mechanism of AD, and new therapeutic targets have
become available that should allow the underlying
disease process to be tackled directly. In this respect,
the ‘amyloid hypothesis’ has become the dominant
theory in the field. It is believed that Aβ accumulation
in plaques or as partial soluble filaments initiates a
The amyloid and tangle cascade hypothesis is the dominant explanation for
the pathogenesis of Alzheimer’s disease (AD). A complete knowledge of the
metabolic pathways leading to β-amyloid (Aβ) production and clearance in vivo
and of the pathological events that lead to fibril formation and deposition into
plaques is crucial for the development of an ‘anti-amyloid’ therapeutic strategy.
Important advances in this respect have been achieved recently, revealing new
candidate drug targets. Among the most promising potential treatments are
β- and γ-secretase inhibitors, Aβ vaccination, Cu–Zn chelators, cholesterol-
lowering drugs and non-steroidal anti-inflammatory drugs. Now, the major
question is w hether these drugs w ill work in the clinic.
Novel therapeutic strategies provide
the real test for the amyloid
hypothesis of Alzheimer’s disease
Diana Ines Dominguez and Bart De Strooper
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