Skip to main content Accessibility help
×
Hostname: page-component-7c8c6479df-fqc5m Total loading time: 0 Render date: 2024-03-19T11:27:35.821Z Has data issue: false hasContentIssue false

15 - Neurodegenerative diseases

from PART II - DISORDERS OF HIGHER FUNCTION

Published online by Cambridge University Press:  05 August 2016

Stanley B. Prusiner
Affiliation:
Institute for Neurodegenerative Diseases and Departments of Neurology
Arthur K. Asbury
Affiliation:
University of Pennsylvania School of Medicine
Guy M. McKhann
Affiliation:
The Johns Hopkins University School of Medicine
W. Ian McDonald
Affiliation:
University College London
Peter J. Goadsby
Affiliation:
University College London
Justin C. McArthur
Affiliation:
The Johns Hopkins University School of Medicine
Get access

Summary

Twenty-five years ago, there was little understanding of the causes of neurodegeneration. In fact, the term degenerative disease was used as a wastebasket for illnesses of unknown etiology. But progress over the past quarter of a century in research focused on degenerative disorders of the central nervous system (CNS) has been impressive. It is now clear that neurodegenerative diseases are caused by the misprocessing of proteins. In each disease, one or more specific proteins have been identified that are misprocessed; this results in the accumulation of one or more particular proteins.

The proteins that accumulate in the CNS of patients with neurodegenerative diseases were initially identified by purifying these polypeptides from the brains of animals or humans with these diseases (Glenner & Wong, 1984; Masters et al., 1985; Prusiner et al., 1982). Subsequently, molecular genetics was used to identify the genes responsible for the familial forms of Alzheimer's and Parkinson's diseases as well as amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Similarly, molecular genetic investigations of Huntington's disease (HD) and the spinocerebellar ataxias have led to the identification of the genes responsible for the pathogenesis of these illnesses.

Of all the studies on neurodegenerative diseases, the discovery of prions has been most unexpected. The finding that a protein can act as an infectious pathogen and cause degeneration of the CNS was unprecedented (Prusiner, 1998b). The prion concept was so novel that achieving acceptance required a long and arduous battle (Prusiner, 1999). The prion concept not only explained how a disease can be both infectious and genetic, but it has also created new disease paradigms and revolutionized thinking in biology.

Although progress in the study of neurodegeneration has been impressive, there are still no curative treatments.Only for patients with Parkinson's disease is there a palliative drug with reasonable efficacy (Cotzias et al., 1967). L-dopa and related drugs do not stop the underlying degeneration, which often renders patients refractory to pharmacologic treatment in the later stages of Parkinson's disease (Marsden & Parkes, 1977). Stereotactic surgery has produced limited success in ameliorating the symptoms of Parkinson's disease when L-dopa becomes ineffective. Transplantation of cells secreting dopamine into the brains of patients with advanced Parkinson's disease is the subject of much research. It is noteworthy that many patients with Parkinson's disease develop dementia in the later stages of this disorder.

Type
Chapter
Information
Diseases of the Nervous System
Clinical Neuroscience and Therapeutic Principles
, pp. 210 - 236
Publisher: Cambridge University Press
Print publication year: 2002

Access options

Get access to the full version of this content by using one of the access options below. (Log in options will check for institutional or personal access. Content may require purchase if you do not have access.)

Save book to Kindle

To save this book to your Kindle, first ensure coreplatform@cambridge.org is added to your Approved Personal Document E-mail List under your Personal Document Settings on the Manage Your Content and Devices page of your Amazon account. Then enter the ‘name’ part of your Kindle email address below. Find out more about saving to your Kindle.

Note you can select to save to either the @free.kindle.com or @kindle.com variations. ‘@free.kindle.com’ emails are free but can only be saved to your device when it is connected to wi-fi. ‘@kindle.com’ emails can be delivered even when you are not connected to wi-fi, but note that service fees apply.

Find out more about the Kindle Personal Document Service.

Available formats
×

Save book to Dropbox

To save content items to your account, please confirm that you agree to abide by our usage policies. If this is the first time you use this feature, you will be asked to authorise Cambridge Core to connect with your account. Find out more about saving content to Dropbox.

Available formats
×

Save book to Google Drive

To save content items to your account, please confirm that you agree to abide by our usage policies. If this is the first time you use this feature, you will be asked to authorise Cambridge Core to connect with your account. Find out more about saving content to Google Drive.

Available formats
×