Neuroblastoma. What is it and how is it treated?

To combat them, the Italian Association for the Fight against Neuroblastoma was born in 1993, an ONLUS founded by oncologists and parents determined to identify the right and effective treatments for all sick children, which is based at the G. Gaslini Institute in Genoa and is active throughout the national territory. Today it has over 115,000 members.

The goal is to finance scientific research on neuroblastoma and brain tumors, inform about pathologies, create links between treatment and research centers, strengthen the laboratories and means available for treatment, to ensure a rapid and correct diagnosis and an approach therapy aimed at the little patient

The journey of those who fight these diseases is still long: the child with the funnel, symbol of the Association, with his gaiety, joy and will to live represents all sick children and invites them not to be discouraged, in the belief that one day everyone the sick little ones will heal.

See also

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What is neuroblastoma?

Neuroblastoma is a childhood cancer that originates in the sympathetic nervous system and occurs at diagnosis in most cases with skeletal and marrow metastases. It affects 120-130 children from 0 to 15 years a year in Italy

It is a sneaky tumor, the symptoms of which can be misleading because they can often be traced back to flu (pallor, anorexia, mood change, refusal to walk); generally they precede the discovery of the tumor by weeks or months, which at that point, for more than half of the patients, presents with metastases to the skeleton and marrow.

Neuroblastoma is recognized by the scientific world as an excellent study model: given the peculiarity of its biological characteristics that justify the definition of a "set of tumors", the results on this pathology can be extended to all pediatric oncological pathologies and also to some forms of the adult.

The stadiums

The most recent neuroblastoma risk protocol classifies the disease into these stages:

Stage 1: The tumor is mostly small and confined to a single site: the surgeon can easily remove it and there are no metastases to other organs or traces of malignant cells. At this stage the disease does not require postoperative therapy.

Stage 2: the tumor is more extensive than stage 1, but still limited; Surgical removal leaves minimal residues that sometimes reach the spine. Some cancer cells can enter the lymph nodes, in which case a short period of chemotherapy is required to reduce the risk of cancer cell proliferation.

Stage 3: the tumor is confined to the area of ​​origin and there are no distant metastases, but it is too big to be removed by the surgeon and completely at the first operation, so it is essential to resort to chemotherapy to reduce the tumor mass and to be able to remove it surgically at a later time.

Stage 4: The tumor, regardless of its shape and size, has spread diseased cells to other organs, such as the bone marrow, skeleton, liver, lymph nodes. The disease has considerable biological aggression and intensive chemotherapy treatment will be required.

Stage 4 S: it is a special form of Neuroblastoma that arises in the first months of life, with a particular dissemination that mainly involves the skin and liver and not the skeleton. At this stage the disease can regress completely spontaneously and, only in some cases, a short course of chemotherapy may be necessary. The primary tumor, often small, can be removed immediately or after the metastatic lesions have at least partially regressed.


Other important elements for diagnostic purposes beyond age, stage and genetic characteristics (MYCN oncogene modified or not) are histology and response to therapy.

The cure

Recently, new therapy protocols have been developed that take into account the patient's risk. There are three in traditional protocols

well-established and well-tested treatments: surgery, radiotherapy and chemotherapy.

The new protocols have included two new aspects deriving from the study of tumor genetics and new drugs to which the Foundation has made a significant financial commitment, supporting national research projects on neuroblastoma. These new aspects are:

  • avoid treatment of the child when the tumor has little aggressive genetic characteristics
  • aggressively treat high-risk patients (metastatic disease in children over 1 year of age) with high doses of drugs, autologous hematopoietic stem cell transplantation, surgery, radiotherapy, and cis retinoic acid differentiation therapy combined with immunotherapy . This last phase would finally seem to make it possible to significantly reduce the reappearance of the disease after a few months, increasing the chances of recovery.

Clinicians and researchers are struggling to improve neuroblastoma treatment, largely supported by organizations such as the Association; thanks to these new protocols, the survival of sick children has gone from 50% in the early 1980s to the current 70%. Certainly not a satisfactory result, but the most recent discoveries on the biological events at the origin of the neoplasm give hope in the possibility of identifying new, more active and safer drugs.

What are Pediatric Brain Tumors?

Children's brain tumors are a very heterogeneous group of diseases with over 15 different histological types, affecting the central nervous system. Between 400 and 450 children, from zero to 15 years, fall ill with a CNS tumor every year in Italy.

Access to treatment is mediated not only by clinical diagnosis but also by radiological diagnosis, which now makes use of harmless and highly targeted imaging techniques, such as Computerized Axial Tomography and Nuclear Magnetic Resonance.

The treatments are always multidisciplinary and require close collaboration, mutual knowledge and trust, and mutual respect for the skills of neurosurgeon, pathologist, oncologist, radiotherapist, radiologist and all other figures: neurologist, physiatrist, ophthalmologist, ENT, social worker , neuropsychiatrist, psychologist, endocrinologist, orthopedist who, to different degrees and at different times, will participate in determining the diagnosis, treatment and recovery of the young patient. Multidisciplinary treatments have resulted in an improvement in 5-year survival in Europe from 57% to 65% for children treated from 1983 to 1994, with a 3% reduction in the risk of death per year (Gatta G, 2005).

What are the research projects funded by the Association?

  • ALK Project (Neuroblastoma Foundation Laboratory and National Cancer Institute of Milan; Dr. Longo)

The discovery of the ALK gene marked a fundamental step for the progress of neuroblastoma research, because it can potentially prove to be a fundamental gene for the development of cancer cells. It is currently being studied to determine whether it can be the target of drugs that are effective in inhibiting their activity.

  • GENUS friendly drug project (CIBIO di Trento; Prof. Alessandro Quattrone)

Based on the analysis of the composition of molecules of various drugs in use and not, the project aims to find new drugs suitable for children, and even more, a treatment that can be adapted to the various stages (neuroblastoma is a "changeable" tumor).

  • Histo-pathological clinical project on Neuroblastoma (Institute "G. Gaslini di Genova"; Dr. Alberto Garaventa)

The aim of the project is to optimize diagnostics and ensure high and homogeneous quality in the framing and treatment of children affected by Neuroblastoma. Furthermore, we want to define new and more powerful prognostic factors and re-evaluate the current ones. Thirty-six hours to diagnose and define the appropriate therapeutic protocol for each child who falls ill with neuroblastoma.

  • Thought Project (Umberto I Hospital in Rome and others; Dr. Giangaspero)

The aim of the project is to make it possible to centralize the histological material of children suffering from brain tumors at the Policlinico Umberto 1 ° in Rome, where the decisive analysis of the samples is carried out, thus avoiding diagnostic errors (often lethal in brain tumors) and / or confirming the first classification.

In 20% of cases the project even guarantees the first and correct diagnosis.

  • Combined Immunotherapy Project for disseminated neuroblastoma (National Institute for Cancer Research-Genoa; Dr. Croce)

The project aims to highlight new therapies aimed at stimulating the immune system of the little patient, so that this is strengthened and is able to fight the disease.

What are the results obtained?

Its own laboratory dedicated to the study of neuroblastoma, the discovery of the ALK gene responsible for the predisposition to neuroblastoma, the centralization of neuroblastoma and brain tumor samples to facilitate a correct diagnosis in 36 hours: these are just some of the results achieved in recent years. Currently there are about 50 people throughout Italy who are financed by the Neuroblastoma Foundation.

What is there to do and what can you do?

The best researchers need a continuous supply of financial resources and expensive equipment to achieve the most complete biological knowledge of cancer. The goal is to identify molecular targets that can be targets of new drugs, in the identification and study phase. Furthermore, the work of collecting and creating databases that can be used by the scientific community is a work in progress that opens up great potential for future progress.

To contribute to the research progress, you can make a donation directly.

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Also for anniversaries and important events, you can choose favors, parchments and solidarity tickets that allow you to celebrate, doing good.

For more information: http://www.neuroblastoma.org

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