Targeting the low-hanging fruit of neurodegeneration

Neurodegenerative diseases are the sixth leading cause of death in the United States,1 but unlike other major causes of mortality, no significant disease-modifying therapies exist. Moreover, the number of patients with neurodegenerative disease is expected to nearly triple by 2050.2 Without effective therapies, these disorders will devastate our health care systems, families, and millions of patients. The lack of treatments is not for lack of effort. In fiscal year 2011, the NIH spent $713 million on neurodegenerative disease research ( ) and PubMed lists over 227,000 studies on neurodegeneration. So why are there no effective treatments? Acknowledgment: A. Mason, A. Ziemann, and S. Finkbeiner thank R. Albin, G. Block, L. Bruijn, J. Dunlop, S. Freedman, S. Landis, R. Mahley, G. Seabrook, M. Sutherland, R.S. Williams, and members of the Finkbeiner Laboratory for discussions or comments on this article, G. Howard for editorial assistance, and K. Nelson for administrative assistance.