Karyopharm Therapeutics Initiates Treatment of Patients With Acute Myeloid Leukemia (AML) in Phase 1 Trial With Oral KPT-330, the First Selective Inhibitor of Nuclear Export (SINE)
Published: Jan 17, 2013
NATICK, Mass., Jan. 17, 2013 (GLOBE NEWSWIRE) -- Karyopharm Therapeutics Inc., a leader in the new field of nuclear transport modulators, announced the dosing of patients with AML in the ongoing Phase 1 clinical trial of KPT-330 in patients with advanced hematologic malignancies. KPT-330 is the first oral SINE Exportin 1 (XPO1/CRM1) antagonist to enter human studies. XPO1 mediates the nuclear export and inactivation of key tumor suppressor proteins. XPO1 blockade causes nuclear accumulation and functional activation of the tumor suppressor proteins, leading to potent and selective tumor cell apoptosis while sparing normal cells. Recent publications from the laboratories of Dr. Romero Garzon at the Ohio State University (Columbus, OH) and Dr. Thomas Look at the Dana Farber Cancer Center (Boston, MA) demonstrate the potent activity of SINE compounds in preclinical models of AML.