Preprint Review Version 1 Preserved in Portico This version is not peer-reviewed

Retinoid Therapy for Neuroblastoma: Historical Overview, Regulatory Challenges, and Prospects

Atsushi Makimoto
* ORCID logo ,
Hiroyuki Fujisaki
,
Kimikazu Matsumoto
,
Yoshiyuki Takahashi
,
Yuko Cho
,
Yoshihiko Morikawa
,
Yuki Yuza
,
Tomoko Iehara
,
Tatsuro Tajiri
Version 1 : Received: 14 November 2023 / Approved: 15 November 2023 / Online: 16 November 2023 (11:37:03 CET)

A peer-reviewed article of this Preprint also exists.

Makimoto, A.; Fujisaki, H.; Matsumoto, K.; Takahashi, Y.; Cho, Y.; Morikawa, Y.; Yuza, Y.; Tajiri, T.; Iehara, T. Retinoid Therapy for Neuroblastoma: Historical Overview, Regulatory Challenges, and Prospects. Cancers 2024, 16, 544. Makimoto, A.; Fujisaki, H.; Matsumoto, K.; Takahashi, Y.; Cho, Y.; Morikawa, Y.; Yuza, Y.; Tajiri, T.; Iehara, T. Retinoid Therapy for Neuroblastoma: Historical Overview, Regulatory Challenges, and Prospects. Cancers 2024, 16, 544.

Abstract

Retinoids are vitamin A derivatives and include trans-retinoic acid, isotretinoin, tamibarotene, and bexarotene, all of which are currently available for clinical use. The clinical development of retinoid therapy for neuroblastoma has a history spanning more than four decades. The most promising agent is isotretinoin, which can contribute to improving event-free survival in patients with high-risk neuroblastoma by approximately 10 % when administered over six months as maintenance therapy. Although isotretinoin is regarded as an essential component in the standard clinical management of high-risk neuroblastoma, its use for this purpose in the US and EU is off-label. To promote isotretinoin use in Japan as a treatment for neuroblastoma, our clinical research team is planning to launch an investigator-initiated, registration-directed clinical trial. The present review article discusses the basic science behind retinoid therapy, pre-clinical / clinical evidence on neuroblastoma, the concept of the proposed clinical trial, and prospects for this therapy.

Keywords

neuroblastoma; high-risk; retinoids; retinoic acids; isotretinoin; retinoid therapy; tumor differentiation therapy; maintenance therapy; off-label issue

Subject

Medicine and Pharmacology, Oncology and Oncogenics

Copyright: This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

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