Paediatric Strategy Forum for medicinal product development of multi-targeted kinase inhibitors in bone sarcomas: ACCELERATE in collaboration with the European Medicines Agency with participation of the Food and Drug Administration

Andrew DJ Pearson, Nathalie Gaspar, Katherine Janeway, Quentin Campbell-Hewson, Elizabeth R. Lawlor, Chris Copland, Dominik Karres, Koen Norga, Fawzi Benzaghou, Susan Weiner, Brenda Weigel, Aaron R. Weiss, Sandra J. Strauss, Malcolm Smith, Bhuvana A. Setty, Nita Seibel, Nicole Scobie, Alberto Pappo, Chinyere E. Okpara, Karsten NysomJoe McDonough, Lynley V. Marshall, Donna Ludwinski, Franca Ligas, Giovanni Lesa, Steen Knudsen, John Kauh, Antony Hsieh, Delphine Heenen, Douglas S. Hawkins, Ann Graham, Edward Garmey, Steven G. DuBois, Elizabeth Fox, Martha Donoghue, Teresa de Rojas, John Chung, Michela Casanova, Bernadette Brennan, Michael Bishop, Vickie Buenger, Gregory Reaman, Gilles Vassal

Research output: Contribution to journalReview articlepeer-review

4 Scopus citations


The eighth Paediatric Strategy Forum focused on multi-targeted kinase inhibitors (mTKIs) in osteosarcoma and Ewing sarcoma. The development of curative, innovative products in these tumours is a high priority and addresses unmet needs in children, adolescents and adults. Despite clinical and investigational use of mTKIs, efficacy in patients with bone tumours has not been definitively demonstrated. Randomised studies, currently being planned or in progress, in front-line and relapse settings will inform the further development of this class of product. It is crucial that these are rapidly initiated to generate robust data to support international collaborative efforts. The experience to date has generally indicated that the safety profile of mTKIs as monotherapy, and in combination with chemotherapy or other targeted therapy, is consistent with that of adults and that toxicity is manageable. Increasing understanding of relevant predictive biomarkers and tumour biology is absolutely critical to further develop this class of products. Biospecimen samples for correlative studies and biomarker development should be shared, and a joint academic-industry consortium created. This would result in an integrated collection of serial tumour tissues and a systematic retrospective and prospective analyses of these samples to ensure robust assessment of biologic effect of mTKIs. To support access for children to benefit from these novel therapies, clinical trials should be designed with sufficient scientific rationale to support regulatory and payer requirements. To achieve this, early dialogue between academia, industry, regulators, and patient advocates is essential. Evaluating feasibility of combination strategies and then undertaking a randomised trial in the same protocol accelerates drug development. Where possible, clinical trials and development should include children, adolescents, and adults less than 40 years. To respond to emerging science, in approximately 12 months, a multi-stakeholder group will meet and review available data to determine future directions and priorities.

Original languageEnglish (US)
Pages (from-to)71-90
Number of pages20
JournalEuropean Journal of Cancer
StatePublished - Sep 2022

Bibliographical note

Funding Information:
The meeting was held virtually on 30 November and 1 December 2021 with 180 participants: 107 international paediatric and adult oncology experts and scientists investigating the biology of mTKIs from Europe, USA, Canada and Australia; 22 representatives from eight pharmaceutical companies in Europe and USA (Allarity, Bayer, Blueprint Medicines, Eisai GmbH, Exelixis, Ipsen Pharma, HUTCHMED, and Oncoheroes); 21 patient advocates from Europe, USA and Canada (Andrew McDonough B+ Foundation, Ac2orn and Kindred Foundation, Childhood Cancer Canada, Children's Cancer Cause, Coalition Against Childhood Cancer, Euro Ewings Consortium, Karkinaki Awareness for Childhood and Adolescent Cancer, KIDS V CANCER, KickCancer, Imagine for Margo, MIB Agents, The Myrovlytis Trust, Osteosarcoma Institute, PORT, Solving Kids' Cancer, Solving Kids' Cancer UK, Swedish Childhood Cancer Fund, Zoé4life and Childhood Cancer International); 29 regulators from the EMA (including Paediatric Committee [PDCO]) and national competent authorities within the EU regulatory network, US FDA and Health Canada as observers and ACCELERATE as organiser. An overview of the existing trials of mTKIs in bone sarcomas was followed by a review of the relevant biology, and then a presentation of the current plans and needs for mTKIs in osteosarcoma and Ewing sarcoma by academic experts. Lessons learnt from soft tissue sarcomas and a perspective from adult oncology provided context to the discussion. Details of seven mTKIs were highlighted by industry representatives (Table 1). The Forum concluded with the patient advocate perspective and a multi-stakeholder strategic discussion.The authors declare the following financial interests/ personal relationships which may be considered as potential competing interests: FB is an employee and stockholder of Ipsen Pharma. JC is an employee of Bayer Healthcare Pharmaceuticals. MC has served as an advisor for Astra-Zeneca, Bayer, BMS, Pfizer, and Servier. SGD has received consulting fees from Amgen, Bayer, and Loxo Oncology and has received travel reimbursement from Roche and Salarius. EG is an employee of Oncoheroes Biosciences. AH is an employee of Blueprint Medicines. KJ has received consulting fees from Bayer and Ipsen and honoraria fromTakeda and Foundation Medicine. JK is an employee of Allarity Therapeutics. SK is an employee of HUTCHMED International Corporation. CO is an employee of Eisai GmbH. ADJP has consulted for Lilly, Norgine and Developmental Therapeutics Consortium Limited and been an advisor for Amgen. All remaining authors have declared no conflicts of interest.

Funding Information:
Andrew McDonough B+ Foundation for financial support of ACCELERATE.

Publisher Copyright:
© 2022 The Author(s)


  • Bone tumours
  • Cancer therapeutics
  • Drug development
  • Ewing sarcoma
  • Multi-targeted kinase inhibitors
  • Osteosarcoma
  • Paediatric Strategy Forum
  • Paediatric oncology

PubMed: MeSH publication types

  • Journal Article
  • Review
  • Research Support, Non-U.S. Gov't


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