Drug Name: KPT-350
Company: Karyopharm Therapeutics
Location: US-Massachusetts
Drug Type: Small Molecule
Conditions: ALS
Mechanism Type: Nuclear Export Blocker
Mechanism: This approach, which targets exportin 1 (XPO1), aims to protect motor neurons in ALS by blocking nuclear export. The strategy may reduce motor neuron toxicity in ALS in part, by helping keep TDP-43 from building up in the cytoplasm (see Chou et al., 2018).
U.S. Status for ALS: Preclinical

[1] TDP-43 pathology disrupts nuclear pore complexes and nucleocytoplasmic transport in ALS/FTD. Chou, CC et al., Nat Neurosci. 2018 Feb;21(2):228-239.
[2] Inhibiting cancer cell hallmark features through nuclear export inhibition. Sun, Q. et al. Signal Transduct Target Ther. 2016 Jul 1;1:16010.

Last updated February 15th, 2018

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