Consequences of Brain Amyloidosis

( Mechanisms of Disease )

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"Neurodegenerative diseases have long been defined by the properties of the neuropathological lesions observed in the brain. We now know that these lesions are not just markers for neurodegenerative diseases, but are tied intrinsically to their pathogenesis. Moreover, the striking similarities between many neurodegenerative diseases suggest common mechanisms in the etiology of these disorders. The repeated theme of protein misfolding leading to amyloid formation and neurotoxicity is reinforced by a striking message from genetic studies: In each case of dominantly inherited neurodegeneration, the disease-causing mutations can be linked directly to amyloid formation. These clues have now come into sharp focus and neurodegenerative properties are better understood, and they have provided a strong rationale for the in vitro experiments, mouse models, and epidemiological studies that have gone a long way towards verifying this hypothesis. These experiments have laid a solid groundwork for a number of potential therapeutics that are now in preclinical and clinical development. Over the next five years the efficacy of a large number of these drugs will be tested, and there is a growing optimism that the results of these trials will confirm current notions of the etiology of neurodegenerative diseases". Read more: annualreviews.org

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