The Monaco Scientific Centre (CSM) has announced promising news regarding a clinical trial for a pediatric brain cancer treatment called 'Mependax'. On June 13, 2024, the CSM reported that this treatment is likely to significantly improve the care of children and young adults affected by brain cancer.
Six patients aged 12 to 21, who had exhausted at least four different lines of treatment, benefited from this "last hope" therapy. The CSM laboratory administered a new combined treatment using a "metronomic" therapeutic regimen, resulting in improved care. The treatment showed very encouraging results with relatively low toxicity. Some patients experienced a favorable therapeutic response and an improvement in their quality of life.
"Essentially, we administer two anticancer molecules, axitinib and etoposide, at lower doses but more frequently," explained Dr. Vincent Picco from the medical biology department of the CSM. "This new therapeutic combination and particular method of administration, which can be taken orally outside a hospital setting, is likely to control tumor growth more effectively while minimizing undesirable effects."
The trial has been detailed in the scientific journal "Paediatric Blood and Cancer" in an article titled, “Retrospective experience of children with relapsed brain tumours treated with oral combination of axitinib and metronomic etoposide.”
Over the next three years, a group of 36 patients aged 4 to 18 years will be gathered in eight French hospital centers to confirm these initial clinical results.
The MEPENDAX1 clinical trial project was initiated under the leadership of Professor Nicolas André in Marseille and supported by a French network of expert centers in early-phase clinical trials for pediatric cancers (CLIP, National Cancer Institute, and Ligue Contre le Cancer).
"For children and their families, this clinical trial offers 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 new therapy delay hope for children and families. Despite the innovative nature of the treatments, the drugs used in this trial have already been utilized in other applications, so doctors can already administer this treatment."
In this context, pediatric oncologists from the hospitals of Marseille, Lyon, and Nice are now taking on the responsibility to help their young patients.
"This prescription remains exceptional and addresses the lack of appropriate therapeutic medicinal alternatives," specified Dr. Vincent Picco.
The successful work by cancer researchers at the CSM, which led to the MEPENDAX trial, has been supported for nearly 10 years by the Flavien Foundation. Other Monegasque and French institutions, along with private supporters, have also chosen to finance this clinical phase.