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The latest Paediatric Strategy Forum, organised by ACCELERATE in collaboration with the European Medicines Agency (EMA) and with participation from the FDA, has brought to light the current limitations in the development of cyclin-dependent kinase (CDK) inhibitors in paediatric oncology. Patricia Blanc, founder of the Imagine for Margo association, contributed to the publication of the Forum’s conclusions in a landmark scientific article published in the European Journal of Cancer.
Many clinical trials, yet limited meaningful outcomes
The Forum critically assessed the many studies conducted in recent years on CDK4/6 inhibitors (palbociclib, ribociclib, abemaciclib) in children with cancers such as neuroblastoma, gliomas, or sarcomas. While these molecules are widely used in adults—especially for breast cancer—the results in paediatrics remain modest, with mostly cytostatic effects (tumour growth slowdown) and very few actual tumour regressions observed.
A strong message from patients: “It’s time to change the paradigm”
Patricia Blanc and other patient advocacy representatives emphasised the lack of coordination between trials, the redundancy of study designs, and the limited value gained given the number of children enrolled.
“Exposing children to repeated, low-efficacy treatments without gaining real knowledge is a missed opportunity,” stated Patricia Blanc during the Forum. “We need to prioritise, collaborate, and most importantly, learn from the past to avoid making the same mistakes again.”
What’s next for CDK inhibitors in paediatrics?
Experts now call for:
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Reassessing the value of CDK4/6 inhibitors as monotherapy;
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Favouring combination strategies based on strong preclinical evidence;
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Prioritising transcriptional CDK inhibitors (notably CDK9, 12 and 13), which show greater promise in some paediatric cancers such as MYCN-amplified neuroblastoma or osteosarcoma;
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Improving coordination between industry, academia, and regulators, and developing global, class-level strategies rather than product-by-product approaches.
A major contribution to shift the lines
Through her active involvement in this Forum and publication, Patricia Blanc continues Imagine for Margo’s mission to accelerate access to innovative and meaningful treatments for children with cancer.
This article stands as a clear call to streamline paediatric trials, prioritise based on science, and foster stronger collaboration among all stakeholders in the field.