Selinexor, a first-in-class oral selective inhibitor of nuclear export, demonstrated preliminary activity and manageable toxicity in patients with advanced soft tissue or bone sarcoma, according to study published in the Journal of Clinical Oncology.1

For this phase 1b study, researchers evaluated the pharmacokinetics, pharmacodynamics, safety, and efficacy of selinexor in patients with advanced soft tissue or bone sarcoma with progressive disease.

Of 52 evaluable patients, none achieved an objective response per RECIST version 1.1 criteria, though 33% exhibited durable stable disease, including 7 of the 15 patients evaluable with dedifferentiated liposarcoma.

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The most frequently reported treatment-related adverse events were nausea, vomiting, anorexia, and fatigue, which were successfully managed with supportive care. The most common grade 3 to 4 toxicities were fatigue, thrombocytopenia, anemia, lymphopenia, and leukopenia.

Patients who received the flat dose of 60 mg on an intermittent schedule tolerated treatment significantly better than those treated with other doses and schedules.

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Food intake did not appear to have a clinically meaningful impact on the pharmacokinetics of selinexor.

The efficacy and safety of selinexor is also being evaluated in patients with acute myeloid leukemia, B-cell lymphoma, breast cancer, glioma, multiple myeloma, pancreatic cancer, and rectal cancer.                    


  1. Gounder MM, Zer A, Tap WD, et al. Phase IB study of selinexor, a first-in-class inhibitor of nuclear export, in patients with advanced refractory bone or soft tissue sarcoma. J Clin Oncol. 2016 Jul 25. doi: 10.1200/JCO.2016.67.6346. [Epub ahead of print]