Karyopharm Therapeutics is a clinical stage pharmaceutical company best known for its lead compound selinexor, currently in clinical testing for hematological malignancies and solid tumors. With new findings on the central role of C9ORF72 in nuclear-cytoplasmic transport (see Aug 2015 news), the company has announced their entry into the ALS arena with their small molecule modulators of this critical pathway. Specifically, the plan to test their inhibitor of the nuclear export protein XPO1, KPT-350, as a candidate therapy for ALS. A second compound developed by the company that also targets XPO1, called KPT-276, was used by Rothstein and colleagues to rescue the nuclear transport detects in C9ORF72 mutant flies (see Jan 2015 conference news). The company is already collaborating with several academic researchers to test the therapeutic efficacy of this compound in patient-derived iPSCs and in rodent models of ALS.
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