You may remember that in 2023 we made a £10,000 grant to the Centre Scientifique de Monaco (CSM – Monaco Scientific Centre) to help fund their groundbreaking work on paediatric cancers.

You can read our previous article about it here: First donation of 2023

Dr. Vincent Picco, who heads up the Stem Cells and Brain Tumors team in the CSM’s Medical Biology Department, got back in touch to give us some great news!

The MEPENDAX clinical trial, resulting from his team’s work, is now under way. Professor Nicolas André, who is leading the trial in Marseille, is “cautiously optimistic, but very hopeful that progress will be made.”

The CSM’s laboratory research into medulloblastoma, the most common malignant children’s brain tumour, 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 other French cities will come on board as it progresses. 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.”

We’ll keep you updated with any further news!