Company: Karyopharm Therapeutics
Drug Type: Small Molecule
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
 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.
 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