Drug Type: Small Molecule
Conditions: Alzheimer’s disease, Parkinson’s disease
Mechanism Type: Protein aggregate clearance
Mechanism: In collaboration with Biogen, the company developed novel inhibitors of Usp14, a deubiquitinating enzyme, to enhance clearance of aggregation-prone proteins such as alpha synuclein and tau. Inhibition of Usp14 promotes clearance of TDP-43 by retaining ubiquiting chains, suggesting this could be a promising therapeutic approach in ALS. However, Amgen recently reported that the company could not reproduce these published results. The status of this approach is unknown.
U.S. Status for ALS: N/A
 Enhancement of proteasome activity by a small-molecule inhibitor of USP14. Lee, BH et al. Nature. 2010;467:179–184.
 If you fail to reproduce another scientist’s results, this journal wants to know. Kaiser, J. Science, 4 Feb 2016. Accessed 11 Mar 2016 from http://www.sciencemag.org/news/2016/02/if-you-fail-reproduce-another-scientist-s-results-journal-wants-know.
 Proteostasis Therapeutics Announces Collaboration with Biogen Idec for Research, Development and Commercialization of Therapies for Neurodegenerative Diseases. Harvard Office of Technology Development. 27 Feb 2016. Accessed 11 Mar 2016 from http://otd.harvard.edu/news-events/proteostasis-therapeutics-announces-collaboration-with-biogen-idec-for-rese/.
Last updated February 8th, 2018