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Monaco Scientific Centre makes a Breakthrough in Brain Cancer Treatment

The Monaco Scientific Centre (CSM) released good news in a press release concerning the clinical trial of a paediatric brain cancer treatment called ‘Mependax’. Results show that the treatment is likely to improve the care of children and young adults affected by brain cancer, stated the CSM on 13 June 2024.

Six children and young adults aged 12 to 21 benefited from this “last hope” therapy. These patients found themselves in a therapeutic impasse after having followed at least four different lines of treatment. The CSM laboratory administered a new combined treatment according to a something called the “metronomic” therapeutic regimen, which improved the care of young patients. The results of the treatment are very encouraging with relatively low toxicity. Certain cases also experienced a favourable therapeutic response and an improvement in quality of life.

“Schematically, the administration of two anticancer molecules called axitinib and etoposide is done at lower doses, but more frequently,” explained Dr. Vincent Picco from the medical biology department of the CSM. “This new therapeutic combination and this particular method of administration, which also has the advantage of oral intake outside of a hospital environment, is likely to control the growth of tumours more effectively while limiting undesirable effects.”

The trial has been detailed in the “Paediatric Blood and Cancer” scientific journal in an article titled, “Retrospective experience of children with relapsed brain tumours treated with oral combination of axitinib and metronomic etoposide”.

What’s next?

A group of 36 patients aged 4 to 18 years will be brought together over the next three years in eight French hospital centres with the hope of confirming these first clinical results.

The MEPENDAX1 clinical trial project was born under the aegis of Professor Nicolas André in Marseille and supported by a French network of expert centres in early phase clinical trials for paediatric cancers (CLIP, National Cancer Institute and Ligue Contre le Cancer).

“For children and their families, this clinical trial offers an immense hope,” said Denis Maccario, president of the Flavien Foundation, “Although funding is now assured, the legitimate administrative constraints to start this type of trial with a whole new therapy delays hope for children and families. Despite the treatments innovative nature, the drugs used in this trial have already been utilized in other applications, so doctors already have the possibility of administering this treatment.”

In this context, paediatric oncologists from the hospitals of Marseille, Lyon and Nice are now taking on this responsibility to help their young patients.

“This prescription remains exceptional and responds to a lack of an appropriate therapeutic medicinal alternatives,” specified Dr. Vincent Picco.

The work successfully carried out by cancer researchers at the CSM which led to the MEPENDAX trial has been supported for almost 10 years by the Flavien Foundation. Other Monegasque and French institutions and private supporters also chose to finance this clinical phase.

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