Patupilone demonstrated activity in heavily pretreated patients with recurrent brain metastases from non-small cell lung cancer (NSCLC), a new study published online ahead of print in the journal Cancer showed.1

There are limited treatment options for patients with NSCLC with brain metastases, but patupilone, a blood brain barrier-penetrating, microtubule-targeting, cytotoxic agent, has demonstrated clinical activity in early clinical trials in patients with NSCLC.

In the first prospective study to evaluate chemotherapy for recurrent brain metastases from NSCLC, researchers enrolled 50 adult patients with NSCLC and confirmed progressive brain metastases. All participants received patupilone 10 mg/m2 intravenously every 3 weeks.

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Efficacy results showed that the 9-week progression-free survival was 36% and the early progression rate, defined as death or progression within 3 weeks, was 26%. Overall survival and median time to progression were 8.8 and 3.2 months, respectively.

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In regard to safety, grade 3 or 4 adverse events included diarrhea, pulmonary emboli, convulsions, and peripheral neuropathy. All participants discontinued treatment with patupilone, either for disease progression or adverse events.

Of the 32 deaths to occur, 25 were due to brain metastases.


  1. Nayak L, DeAngelis LM, Robins HI, et al; Multicenter phase 2 study of patupilone for recurrent or progressive brain metastases from non-small cell lung cancer [published online ahead of print August 26, 2015]. Cancer. doi: 10.1002/cncr.29636.