Unleashing the power of immunotherapy for kids with neuroblastoma in relapse

This project was selected in the framework of our European call: FIGHT KIDS CANCER 2022. It is jointly financed by Imagine for Margo, Kriibskrank Kanner Fondatioun and KickCancer.

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Despite the current intensive treatments, the chances of survival for children with neuroblastoma that has come back (in relapse) remain unacceptably low, with less than 20% of children achieving a long-term cure.

To this day, only one new type of drug has been developed specifically for patients with neuroblastoma and it is critical to identify new, more efficient and less toxic treatments.

The BEACON 2 trial will test two new promising anti-cancer medicines that will be combined with chemotherapy.

The first drug (bevacizumab) specifically targets the blood vessels that help tumours grow. By blocking the growth of the blood vessels, the tumour will not have access to its high needs in nutrients, which will slow or prevent its growth and eventually lead to the death of the tumour cells.

The second drug (dintutuximab beta) is an immunotherapy drug that enables the white blood cells (our immune system) to recognise the cancer cells as strangers in a patient’s body. Indeed, as cancer cells are mutated cells derived from our own cells, our immune system is not capable of detecting them as dangerous and, as a result, does not attack them. All of our own cells (normal or mutated, cancerous cells) possess “checkpoints”, which help our white blood cells to recognise them as “own cells” and to prevent their attack. This second drug binds on a molecule found on the surface of neuroblastoma cells, which will hide its “checkpoint”. When the patient’s white blood cells will pass by neuroblastoma cells, they will no longer recognise them as “own cells” and, as a result, will kill them.

As the Beacon2 trial aims at recruiting 160 patients, it will confirm which of these two treatments is superior in a large group of patients. This is an essential step to change practice, so that the treatment can be given by doctors widely across Europe after the trial. It will also develop new combinations of drugs that improve the efficacy of immunotherapy, curing more children with fewer side effects.