Abstract
The notion that many neurodegenerative diseases are caused by seeded protein aggregation is almost 20 years old (Jarrett and Lansbury, Cell 73:1055–1058, 1993; Lansbury, Neuron 19:1151–1154, 1997). Recent data, some of it summarized here, suggest that this mechanism may account for cell-to-cell transmission throughout the brain by “proteopathic seeds.” There are many scientific questions that remain to be solved, including what is the best approach to interfere with the seeding process in vivo. But it may be more important to address the practical bottleneck that is common to all therapeutic strategies: how can one demonstrate potential efficacy of an experimental drug in a small, inexpensive clinical trial? This manuscript will address the issues that, together, have produced this bottleneck and will suggest some possible approaches to stimulate drug development for neurodegeneration.
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Lansbury, P.T. (2013). Development of Drugs That Target Proteopathic Seeds Will Require Measurement of Drug Mechanism in Human Brain. In: Jucker, M., Christen, Y. (eds) Proteopathic Seeds and Neurodegenerative Diseases. Research and Perspectives in Alzheimer's Disease. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-642-35491-5_10
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DOI: https://doi.org/10.1007/978-3-642-35491-5_10
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