Neurodegeneration Exploring Commonalities Across Diseases: Workshop Summary (2013) / Chapter Skim
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2 Rationale for Exploring Commonalities Across Neurodegenerative Diseases
2 Rationale for Exploring Commonalities Across Neurodegenerative Diseases
Pages 9-22
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For example, many patients with a particular disease have more than one proteinopathy, and a single type of proteinopathy can be associated with multiple diseases. • Neurodegenerative diseases have overlapping genetics.
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However, the workshop began by asking the underlying question: Is there a rationale to justify studying neurodegenerative diseases together? Presentations and discussions examined common features across diseases, including pathological and genetic overlaps, common challenges, and practical considerations related to the infrastructure needed to study these diseases.
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A single therapy aimed at one proteinopathy may be found ineffective because the underlying disease has multiple proteinopathies. Just as a single neurodegenerative disease can be associated with multiple proteinopathies, a single proteinopathy can also be associated with multiple diseases.
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argyrophilic grain disease is an increasingly recognized disorder of the elderly that affects the medial temporal lobe and is associated with an amnesic cognitive impairment. Given the pathological heterogeneity, Dickson urged researchers, for therapeutic purposes, to search for "upstream targets, rather than downstream pathologies." Genetic Overlaps Neurodegenerative diseases also have genetic overlaps (Bertram and Tanzi, 2005)
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Besides genetics and pathology, other overlaps occur in the mechanisms of disease pathogenesis. These overlaps were the prime focus of the workshop and are covered in ensuing chapters: protein aggregation, transmissibility within the central nervous system, mitochondrial dysfunction, and RNA processing errors.
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Finally, pooling resources for clinical trials brings fears in drug companies of losing intellectual property, which deters investment. Amid the formidable array of challenges, as well as the economic downturn, several pharmaceutical companies have reduced their neurodegenerative disease divisions, according to press reports.
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"I think for almost every stone you turn over, you will find an opportunity to collaborate," said Ivinson. He and other presenters acknowledged, however, that collaborative approaches to studying more than one disease have rarely been tried and may not be pertinent to all neurodegenerative diseases because of disease heterogeneity.
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. example Promising Technologies Several speakers described promising technologies to study the complex pathogenesis of neurodegenerative disease as well as to identify new treatments.
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It has become a model for studying neurodegenerative disease because many of its fundamental cell pathways are relevant, such as mitochondrial gene function and autophagy, the process of self-degradation and clearance described in Chapter 3. Gregory Petsko of Weill Cornell Medical College described two crucial areas in which yeast can be useful for the study of neurodegenerative disease: mechanistic studies of pathophysiology and drug screening.
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Research Needs and Next Steps Suggested by Individual Participants The workshop speakers identified many questions for future research and other opportunities for future action. Those related to the rationale for studying commonalities across neurodegenerative diseases are compiled here to provide a sense of the range of suggestions made (research suggestions that are specific to the topics covered in Chapters 3-6 are included at the ends of those chapters)
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(Singleton) • Catalogue ongoing efforts to produce neurodegenerative disease related induced pluripotent stem cells.
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(Games, Ivinson, Mucke) Improving Animal Models3 • Generate experimental models that better simulate the multifacto rial nature of neurodegenerative disease and use them to assess 2 PPMI refers to the Parkinson's Progression Markers Initiative, which is a clinical study aiming to identify biomarkers of Parkinson's disease progression.
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• Generate animal models, free from intellectual property constraints, that are fully validated and available for minimal cost to academia and perhaps at a premium cost to industry. (Youle)
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