131I-omburtamab for neuroblastoma with central nervous system or leptomeningeal metastasis in paediatric patients
131I-omburtamab is a medicinal product that is being developed for the treatment of children with advanced neuroblastoma that has spread to membranes lining the brain and spinal cord (‘leptomeningeal metastasis’). Neuroblastoma is a rare cancer that mostly affects babies and young children. It develops from specialised nerve cells (neuroblasts) in the foetus that do not become mature nerve cells. Instead, they continue to grow and divide becoming cancerous. When neuroblastoma spreads to the brain and spinal cord, the prognosis is poor and there are limited treatment options.
131I-omburtamab is a monoclonal antibody that binds to the surface of neuroblastoma cells. It is linked to radioactive iodine (iodine-131) that produces low-level radiation with a short range, a type of treatment known as radioimmunotherapy. As such, 131I-omburtamab delivers precision radiation to the cancer cells. This radiation from the iodine damages the DNA of the cancer cells which shrinks the tumour and therefore controls the disease. 131I-omburtamab is given by injection into cerebrospinal fluid. If licensed, 131I-omburtamab may offer a treatment option for children with neuroblastoma which has spread to the central nervous system or brain.