Research: ‘reason to hope’ for paediatric brain cancers thanks to CSM
Dr Vincent sporting his Climbers Against Cancer T-shirt. The charity helped fund the CSM's research, along with Fondation Flavien © Thierry Apparu
Dr. Vincent Picco of the Centre Scientifique de Monaco (CSM – Monaco Scientific Centre) contacted us with the good news – the MEPENDAX clinical trial, resulting from his team’s work, is now under way.
We previously reported on Dr Picco and his team’s research into childhood cancers at the CSM. The hoped-for early clinical trial of a new treatment protocol for certain paediatric brain cancer tumours is now a reality. Professor Nicolas André, who is leading the trial in Marseille, is “cautiously optimistic, but very hopeful that progress will be made.”
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After young Flavien lost his battle with cancer in 2014, his father Denis Maccario embarked on a mission to help research into paediatric brain cancers. He founded the Fondation Flavien non-profit and worked closely with the CSM to launch laboratory research into medulloblastoma, the most common malignant children’s brain tumour. Having successfully demonstrated, in vivo, that the combination of two molecules could produce interesting results, they then had to convince one of the world’s leading pharmaceutical companies to use their molecule. Funding was also required, to the tune of approximately €800,000. Once the protocol for the clinical trial received administrative and ethical approval, phase 1 of the clinical trial could finally begin, at the end of last year.
The trial will involve three progressively increasing dose levels, with up to six patients per level. The progressive approach will enable the trial’s safety to be monitored, as well as gathering preliminary efficacy data. For now, it is too early to know if the trial is ‘working’. It is important to first check in Phase 1 that the children can tolerate the treatment. The hope for the second phase is that at least a third of patients stabilise over a six-month period. Each of the phases should take around 18 months. While the process may seem long, there are a number of essential stages to be observed to ensure it is both safe and effective.
Marseille is coordinating the trial, but others will come on board as it progresses. Strasbourg has opened, with Lyon, Lille, Bordeaux, Angers and Paris to follow. The aim is also to extend the protocol to patients with a third type of brain tumour.
Professeur André says, “We obviously believe in it and have hope in this treatment. If we didn’t, we wouldn’t have embarked on this trial. But we also have a major responsibility in offering a new treatment to children who have reached a therapeutic impasse. So I’d say that we have reason to hope.”