Authors
Eelke M Bos, Elena Binda, Iris SC Verploegh, Eva Wembacher, Daphna Hoefnagel, Rutger K Balvers, Anne L Korporaal, Andrea Conidi, Esther AH Warnert, Nadia Trivieri, Alberto Visioli, Paola Zaccarini, Laura Caiola, Rogier van Wijck, Peter van der Spek, Danny Huylebroeck, Sieger Leenstra, Martine LM Lamfers, Zvi Ram, Manfred Westphal, David Noske, Federico Legnani, Francesco DiMeco, Angelo Luigi Vescovi, Clemens MF Dirven
Publication date
2023/8/10
Journal
Molecular cancer
Volume
22
Issue
1
Pages
129
Publisher
BioMed Central
Description
Background
This Phase 1 study evaluates the intra- and peritumoral administration by convection enhanced delivery (CED) of human recombinant Bone Morphogenetic Protein 4 (hrBMP4) – an inhibitory regulator of cancer stem cells (CSCs) – in recurrent glioblastoma.
Methods
In a 3 + 3 dose escalation design, over four to six days, fifteen recurrent glioblastoma patients received, by CED, one of five doses of hrBMP4 ranging from 0·5 to 18 mg. Patients were followed by periodic physical, neurological, blood testing, magnetic resonance imaging (MRI) and quality of life evaluations. The primary objective of this first-in-human study was to determine the safety, dose-limiting toxicities (DLT) and maximum tolerated dose (MTD) of hrBMP4. Secondary objectives were to assess potential efficacy and systemic exposure to hrBMP4 upon intracerebral infusion.
Results
Intra- and peritumoral infusion of hrBMP4 was safe and …
Total citations
Scholar articles
EM Bos, E Binda, ISC Verploegh, E Wembacher… - Molecular cancer, 2023