Paediatric cancer: CSM reports encouraging results following clinical trials 

The findings of a two-year clinical trial of a new type of treatment protocol for children and young adults suffering from brain cancer, which was developed by the Centre Scientifique de Monaco, have been described by its backers as both “positive” and “encouraging”. 

Between January 2022 and December 2023, three paediatric oncology units in France were involved in the clinical trials of a new form of treatment for children and young adults. In total, six patients who had relapsed or refractory brain tumours received the oral axitinib and metronomic etoposide developed in the laboratories of the Centre Scientifique de Monaco (CSM).  

See more: CSM set to revolutionise treatment of childhood cancer

The data and findings of these trials, referred to as the MEPENDAX clinical trial project, have now been published in the Pediatric & Blood Cancer journal, along with a dedicated communiqué from the CSM that confirms that this type of treatment can improve the care of children and young adults suffering from brain cancer. 

“Schematically, the administration of two anticancer molecules called axitinib and etoposide is done at lower doses, but more frequently,” explains Dr. Vincent Picco (pictured), the head of the Brain Stem Cells and Tumours team within the CSM’s Department of Medical Biology. “This new therapeutic combination and this particular mode of administration, which also has the advantage of being taken orally outside the hospital setting, is capable of more effectively controlling tumour growth while limiting side effects.” 

For the six individuals who received the treatment, described by Dr. Picco as a “response to a lack of appropriate therapeutic drug alternatives”, this MEPENDAX trials represented a “last-chance” form of therapy.  

All had reached a tragic impasse in their treatment, having already pursued at least four other lines of treatment before joining the trials.  

According to a communiqué issued by the CSM on 13th June, “The treatment results are very encouraging with, as hoped, relatively low toxicity accompanied in some cases by a favorable therapeutic response and an improvement in quality of life.” 

See more: CSM researchers publish game-changing study on cancer and stem cells

Over the next three years, the CSM has ambitions to expand the trials to a further 36 patients aged four to 18. This time, eight French hospitals will be involved in the project, “with the hope of confirming the initial clinical results”. 

“For the children and their families, this clinical trial offers an immense hope,” says Denis Maccario, the president of the Monaco-based Flavien Foundation. “Although funding is now assured, the administrative constraints to start this type of trial with a new therapy delay this hope for children and families facing the tragedy of therapeutic failure.” 

Maccario, who lost his young son to cancer in 2014, has since used the foundation founded in nine-year-old Flavien’s honour, to help raise the considerable funds that allowed the trials to become a reality. Other backers included the Monaco Government, Climbers Against Cancer, the Savchuk Foundation and the Syndicat des Jeux Européens de la Société des Bains de Mer.

The Pediatric & Blood Cancer Journal report on the trials can be read in full here.  


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Photo source: CSM, Facebook