First phase 1 clinical study of olaparib in pediatric patients with refractory solid tumors.
Masatoshi TakagiChitose OgawaTomoko IeharaYuki Aoki-NogamiEri IshibashiMinoru ImaiToshimi KimuraMasashi NagataMasato YasuharaMitsuko MasutaniKenichi YoshimuraDaisuke TomizawaAtsushi OgawaKan YonemoriAoi MorishitaSatoshi MiyamotoJunko TakitaTetsuro KiharaKiyoshi NoboriKazuhisa HasebeFuyuki MiyaSadakatsu IkedaYoko ShiodaKimikazu MatsumotoJunya FujimuraShuki MizutaniTomohiro MorioHajime HosoiRyuji KoikePublished in: Cancer (2022)
This Phase 1 trial evaluated the efficacy and safety of olaparib in patients with refractory childhood solid tumors. Olaparib was well tolerated, achieving objective response in 2/15 patients. The DNA damage response was attenuated in nearly one-half of advanced neuroblastoma patients, demonstrating the utility of the PARP inhibitor. The results support further investigation of olaparib as a new treatment for DNA damage-response or repair-defective pediatric cancers.