Development of radiopharmaceutical therapy for neuroblastoma in children

Neuroblastoma is the most common and most devastating solid tumor in children.

Despite intensive multimodal therapy, long-term survival in high-risk disease is only 50% and chances of survival after a relapse are dismal.

Neuroblastoma is a radio-sensitive tumor, making targeted radiopharmaceutical therapy attractive, by delivering radiation to the cancer cells, wherever they reside in the body.

We are leading a European multicenter trial where the efficacy of radiopharmaceutical therapy with 177-Lutetium, is being assessed in high-risk neuroblastoma in children (LuDO-N trial).

One of the greatest challenges is that patients who experience a relapse are severely weakened by the cancer and by previous therapy, and optimally this type of treatment should be given at an earlier time point.

For this purpose, we aim to develop radio-pharmaceutical therapy based on 225-actinium or 211-astathine, that emits a significantly higher radiation energy within a very congined space, to deplete single, or small clusters of metastatic cells and thus prevent metastatic relapses.

The overriding aim is to generate data to support a future clinical trial that utilises targeted α-particle therapy early in the course of the disease.

By controlling the systemic disease early, we believe that the cure rates in high-risk neuroblastoma could be significantly improved. We aim to translate our results into a clinical trial in humans, within a time period of 5-10-years.