BRUSSELS - In an article put online by the Lancet Oncology on February 14, 2023, the eve of International Childhood Cancer Day, we urge that promising drugs, need to be evaluated rigorously using state-of-the-art methods and, if proven valuable, made available worldwide.

There is a need to increase survival for children with aggressive brain tumours, but the development and access of new promising treatments is often far from optimal.

This is illustrated in the article in the Lancet Oncology, entitled 'Access to new drugs in paediatric oncology: can we learn from the ongoing ONC201 saga?'

For this article, the Anticancer Fund was fortunate to team up with a number of renowned experts in the field of paediatric brain cancer: Nicolas André (first author from France), Eric Bouffet (Canada), David Walker (UK) and Matthew D Dun (Australia).

Through our information service, My Cancer Navigator, we often are confronted with doctors offering treatments that are not approved and where the clinical evidence has not yet been properly established. Desperate parents want access to drugs promoted as promising and they can not judge the quality of the product. This also happened with ONC201. Therefore we must help our patients by providing them evidenced based information.

“The Anticancer Fund wants to go even one step further by committing us to assist those who want to improve drug development in oncology and ensuring broad but safe and affordable access to promising treatments in a controlled setting.”, said Guy Buyens, medical director at the Anticancer Fund and co-author of the article.

The ONC201 saga in short

ONC201 is a drug candidate that showed promising results in clinical trials for patients with certain brain tumours. ONC201 then went further in clinical development including in certain paediatric brain tumours, however access was almost exclusively possible for patients in the US and Japan. Because of the aggressive nature of the tumours and the lack of access to breakthrough innovation that have been made in years, parents of children with these brain tumours who could not access the trials or compassionate use programmes in the US were desperate to find other ways to get the treatment.

Capitalising on a legal loophole, a German doctor and pharmacy made and delivered the drug to the guardians of patients fighting a lethal and otherwise untreatable brain tumour that currently lack effective medicines. Over the last five years, parents from around the world have travelled to Germany to purchase it. The father of a French patient and French associations got in touch with the French Authorities who reacted and started a national procedure to provide ONC201 to both adult & paediatric patients for certain indications. After validation by a national molecular board, it is sent to treating centres in France.

In the meantime, the commercial drug development of ONC201 remains very slow and clinicians are still awaiting sufficient published evidence in paediatric brain tumours.

The whole story is much more complex and is described in the Lancet Oncology.

Lessons should be learned

The German initiative is unacceptable and should not be repeated. The French initiative is a step in the right direction but far from optimal.

For the development of a drug like ONC201, much more centres worldwide should be involved in early phase trials. Dialogue with the industry should allow the set up of compassionate programmes where data can be obtained. The importance of collecting data through clinical trials conducted with international academic consortia interacting with pharmaceutical companies to incorporate or test new anticancer agents in a multi-stakeholder international platform is the way forward.

We should not underestimate the creativity of stakeholders and the emerging driving force of families. However it should not compromise safe access to treatment as well as state of the art evaluation of activity and toxicity. In any case children’s needs must be given higher priority in cancer drug development than they already do.

More information

The Lancet Oncology: Access to new drugs in paediatric oncology: can we learn from the ongoing ONC201 saga? Nicolas André1, Guy Buyens2, Eric Bouffet3, David Walker4, Matthew D Dun5

Published: February 14, 2023

DOI: https://doi.org/10.1016/S1470-2045(23)00070-0

1. Department of Paediatric Oncology, La Timone University Hospital of Marseille, APHM, Marseille 13005, France (NA); SMARTc Unit, CRCM Inserm 1068- CNRS UMR 7258 Aix-Marseille University, Marseille, France
2. The Anticancer Fund, Belgium
3. Division of Neuro-oncology, Department of Pediatric Hematology-Oncology, Hospital for Sick Children,Toronto, ON, Canada
4. University of Nottingham,Nottingham, UK
5. University of Newcastle, Cancer Signalling Research Group, School of Biomedical Sciences and Pharmacy, College of Health, Medicine & Wellbeing, Callaghan, NSW, Australia; Precision Medicine Research Program, Hunter Medical Research Institute, New Lambton Heights, NSW, Australia