About Neuroblastoma
Disease Information
This area is dedicated in particular to patients and family members as aims to provide main indications and informations on the diseases that are at the heart of our focus, on the disgnosis and therapies used.
We firmly believe that information promotes a peaceful relationship between families, patients and our doctors, creating a fundamental therapeutic alliance in order to deal with the disease in a resolute and calm way.
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What is Neuroblastoma?
Neuroblastoma is a very aggressive childhood cancer: in Italy affects about 130 children every year. It is a rare tumor and represents the primary cause of death by illness for the pre-school age group.
Neuroblastoma is a subtle tumour with similar symptoms to influence ones: for this reason is often diagnosed late, when already presents itself with metastasis and healing it becomes really difficult.
Neuroblastoma is an excellent study model: it is in fact considered “a group of tumours” since holds all the genetic characteristics of nearly all paediatric solid tumours. Finding a permanent cure for Neuroblastoma means find a cure for all paediatric tumours.
During these years important progress has been made thanks to the research supported by the Association: we went from 25% in 90’s to 65% of today chance of survival at 5 years after the diagnosis for favorable prognosis cases and from 8 to 35-40% for children with aggressive cancer and poor prognosis. Every year in Italy it affects about 130 children and teenagers.
Neuroblastoma is a childhood tumor that affects children from 0 to 10 years of age. Along with brain tumors Neuroblastoma is considered the first cause of death and the third neoplasm by frequency after leukemias and Pediatric Brain Tumors.
The term Neuroblastoma refers to a group of malignant tumors more frequent in the first years of life, originating from the remnants of autonomic nervous system that develops during embryonic life.
This tumor originates from neuroblasts, cells located in sympathetic nervous system, a series of neuronal bundles spread throughout the body that control some involuntary functions such as breathing, digestion and heartbeat. Neuroblasts are immature or developing cells that are located in nerves and are widespread throughout the body: neck, adrenal glands, chest, spine.
For this reason Neuroblastoma can arise in different parts. Most cases of Neuroblastoma occur at adrenal glands level, which are located above kidneys, or in the nerve ganglia present in abdomen.
From the clinical point of view Neuroblastoma has some peculiarities:
Prognosis is very good in children under the year of life, regardless of metastic disease extent.
In the first year of life the disease can regress on it’s own even if disseminated.
In older children prognosis is decidedly unfavorable.
Often when diagnosis are reached, Neuroblastoma occurs in an already advanced stage, with metastases to skeleton and marrow. In cases with a more favorable prognosis the survival rate is about 70% but in it’s most aggressive form known as Neuroblastoma at stage 4 survival rate in affected children does not exceed 35%.
That’s not enough for us! The Italian Association for the fight against Neuroblastoma in 20 years of funding for basic research has allowed significant advances in the biological understanding of this disease and about mechanisms that generate it. The Association commitment has guaranteed to all Italian children a correct diagnosis and framing in modern and increasingly personalized care protocols.
Thanks to the Association we have allocated 20 million euros in twenty years of activity to scientific research, of which 45 % to G. Gaslini Insitute in Genoa always considered a reference point for Neuroblastoma.
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Personalized care protocols to young patients take into account the risk of relapse of the child.
They provide:
Surgery
Radiotherapy
Chemotherapy
Well established treatments in synergy with each other, to which are added, (thanks to research results) the genetic information of tumor and those on new treatments obtained in recent years.
This allowed to:
1) Avoid treatment for the child when tumor has not aggressive genetic characteristics.
2) Treat patients with high risk (metastatic disease in children over one year of age) with high doses of drugs, autologous transplantation of hematopoietic stem cells, surgery, radiation therapy and differential therapy with retinoic acid CIS combined with immunotherapy.
This latter stage finally seems to allow a significantly reduction of the disease recurrence after discontinuation of therapy and increases chances of long-survival.
A great study model.
Neuroblastoma is an excellent study model: it’s in fact considered “a set of tumors” as it contains the genetic characteristics of almost all solid childhood tumors. Finding a definitive cure for Neuroblastoma means finding a cure for all children’s cancers.
Over the years, important advances have been made thanks to the research supported by association: we have gone from 25% of 90s to 65% of survival today, 5 years after diagnosis for cases with a more favorable prognosis and from 8 to 35-40% of survival for children with aggressive cancer and unfavorable prognosis.
It is clear that the 50 % of cures achieved with latest protocols in the advanced form of this disease are still not enough.
Molecula characterization of Neuroblastoma made it possible to identify in high-risk tumors the most aggressive forms. The latter are associated with presence of specific genes, for example:
-MYCN altered compared to that of healthy children, which is responsible for a very unfavorable prognosis.
-ALK Gene, present in sick children, which predisposes to the disease.
These important research results have been enabled researchers to identify hypothetical non-toxic drugs (now experimentally) that inhibit these genes activity.
Much remains to be done and the commitment regarding the most aggressive form is total by all researchers within the group engaged in fighting Neuroblastoma.
Neuroblastoma anb Pediatric Brain Tumors are considerate a rare and “orphan” disease because at the moment there are no pediatric treatments able to give hope children affected by it. The children are treated with drugs for adults.
Today our commitment is summarized in the program of development of clinical-biological activity, genetic and translational research, as well as for the immunotherapy project, all studies aimed at detecting molecules and solving drugs that allow the young patient to have the upper hand on Neuroblastoma.
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Paediatric Brain Tumours
What are paediatric brain tumours?
In addition to Neuroblastoma research, our Association supports “Progetto Pensiero” for help against paediatric brain tumours: thanks to our contribution misdiagnoses decreased from an initial 36% of ten years ago to 0%. However, there is still much to be done before finding a permanent cure for these diseases.
Paediatric Brain Tumors are a very diverse group of diseases, different from those of adults and much more rare. The diagnosis of a pediatric brain tumor can be difficult since there are over fifteen different histological types. In Italy the number of new cases per year is about 400.
The incidence of pediatric brain tumors is slightly higher among males than females and is higher in young children.
These include Medulloblastoma and ependymomas.
Medulloblastoma, which is the most frequent and common among pediatric brain tumors, forms in cerebellum. Due to alterations in Dna, some healthy nerve cells degenerate into cancer cells and begin to multiply uncontrollably.
Survival of Medulloblastoma at 5 years after diagnosis is around 60-70 percent. Ependymomas, on the other hand, are part of a group of tumors called “gliomas” that occur mainly within 6 years. Overall they account for 10% of all childhood tumors of central nervous system.
Survival at 5 years from diagnosis varies depending on the degree of ependymoma and, in the most favorable cases, can reach 75%. Relapses over 5 years are not uncommon.
The first symptoms of Pediatric Brain Tumors are generally related to an increase in pressure inside sthe skull, because those tumors obstructs brain’s cerebrospinal flow.
Symptoms of brain tumors can therefore be: intracranial hypertension, headache, vomiting, instability of walking, vision problems etc..
To check for pediatric brain tumors, clinical diagnosis and radiological investigations must be combined, which use harmless and highly targeted imaging techniques, such as computed axial tomography and Nucleat Magnetic Resonance.
Sometimes it is to make a diagnosis by performing a lumbar puncture to take the cerebrospinal fluid to be examined then under a microscope.
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Treatments are always multidisciplinary and provide for close collaboration, mutual knowledge, trust and mutual respect of neurosurgeon, pathologist, oncologist, radiotherapist, radiologist skills. This concerns the above- mentioned practitioners as well as all other figures who to different extent and at different times, will partecipate in determing diagnosis, care, healing of young patients and will provide targeted assistance after therapies.
The nature of Pediatric Brain Tumors (some more malignant than others) and whether or not they can be removed altogether play a big role in survival. Sometimes this is not possible, so subsequent and integrated treatment with radiotherapy and sometimes chemotherapy is expected.
Thanks to biological and clinical research and the use of increasingly effective national and international cooperative care protocols, the probability of survival has risen from 40% in the 70s to more than 80% in 2020s pediatric brain tumors-cures.
The end of care does not coincide with the end of “caring” for the patient, because children suffering from brain neoplasms need to return to their social and school context to the best of their ability.
For this reason, already from diagnosis, it is important to establish a rehabilitaiton path that integrates skills and functionality of the child that may have been lost to the offect of tumor or surgery and, subsequently, all the therapeutic steps provided.