Neuroblastoma treatments

Neuroblastoma is a highly heterogeneous disease, with treatment strategies varying widely depending on the risk group (low-, intermediate-, or high-risk). Care may range from simple monitoring to intensive multimodal therapy—including chemotherapy, surgery, and radiotherapy—followed by maintenance treatment.  Treatment is always tailored to the child and the specific characteristics of the tumor. Treatment decisions are discussed at a multidisciplinary consultation meeting (RCP).

This care is provided by Institut Curie's SIREDO (Care, Innovation, Research in oncology for children, adolescents and young adults), an expert cancer center specifically for young patients.

In Europe, treatments for neuroblastoma are developed through studies conducted by SIOPEN (Society of Pediatric Oncology Europe Neuroblastoma). The aim of these studies is to improve treatments while reducing side effects. Institut Curie is a founding member of SIOPEN and plays a leading role in its biology group.

 

Treatment of low- or intermediate-risk neuroblastoma

Almost half of all neuroblastomas are classified as low or intermediate risk at diagnosis. As the recovery rate for low-risk neuroblastoma is excellent, simple surveillance is often sufficient. Treatment may also consist of surgery alone or coupled with low-intensity chemotherapy.

For intermediate-risk neuroblastoma, treatment usually involves two to eight courses of chemotherapy followed by surgery to remove the tumor as completely as possible. For some patients, additional radiotherapy and maintenance therapy are required.

Chemotherapy for neuroblastoma

Chemotherapy for high-risk neuroblastoma

For high-risk neuroblastoma, treatment is more intensive and requires several stages, including induction chemotherapy, intensification with high-dose chemotherapy, and local treatment with surgery and radiotherapy. Subsequent maintenance treatment also includes immunotherapy.
Induction chemotherapy typically consists of eight cycles, given at short intervals (about every 10 days), to eliminate metastases and shrink the tumor. Depending on the response to this initial treatment, further courses of chemotherapy may be required. 
 

High-dose chemotherapy for neuroblastoma

High-dose chemotherapy is based on the use of powerful drugs, whose effect on tumor cells increases with the dose administered. The aim is to reinforce the anti-tumor effect on both the metastases and the tumor. 

These drugs are highly toxic to the hematopoietic stem cells in the bone marrow, which produce red blood cells, white blood cells, and platelets.  To avoid an excessively prolonged effect, and to allow the child to emerge from aplasia (i.e., the destruction of hematopoietic stem cells), a sample of the child's hematopoietic stem cells is taken before chemotherapy.  This is reinjected into the bloodstream after administration of the high-dose chemotherapy, gradually restoring normal bone marrow function.

Institut Curie has extensive expertise in autologous stem cell transplantation and has mastered this technique.
 

Neuroblastoma surgery

Surgery aims to remove as much of the tumor as possible.  Institut Curie collaborates with surgical teams from other hospitals in the Paris region, including AP-HP hospitals. 

In some cases, the tumor is partially removed. This decision depends on the location of the tumor and its proximity to other organs or blood vessels, which must be preserved. In some cases, surgery may also be used to remove accessible metastases.
 

Radiotherapy of neuroblastoma

Radiotherapy for neuroblastoma uses high-energy ionizing radiation to target the affected area with maximum precision. The dose and area irradiated depend on the volume of the tumor before surgery, and the existence of tumor residue afterwards. Institut Curie specializes in radiotherapy.

Maintenance treatment of neuroblastoma

In the case of high-risk neuroblastoma, maintenance treatment is necessary to limit the risk of relapse.  The use of the anti-GD2 antibody, targeting a molecule present on the cell surface in most neuroblastomas, improves survival in high-risk patients through the direct action of this antibody and the stimulation of immune system cells it enables. The anti-GD2 antibody, in the form of the drug dinutuximab beta, is used in this maintenance treatment to help the immune system fight neuroblastic cells. This treatment has become a standard part of the maintenance treatment of high-risk neuroblastoma, in combination with other therapies such as retinoic acid to make the residual tumor cells less aggressive

To improve the quality of life of young patients, Institut Curie helped develop strategies allowing this drug to be infused at home instead of in the hospital, under certain conditions. After the first two courses of treatment at Institut Curie, the child can benefit from hospitalization at home for the following three courses, thanks to the use of a small autonomous pump. 

Genetic counseling and testing for neuroblastoma

One to 2% of neuroblastomas occur in a predisposing context. It is therefore important to take an interest in family history and to suggest genealogical exploration. Genetic counseling may be offered if needed. As part of the France Médecine Génomique plan, doctors are increasingly recommending comprehensive genomic analyses. Institut Curie has an accredited France Médecine Génomique platform for such analyses at the diagnostic stage, or in the event of treatment failure.