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From 1993 to the present the research sustained with significant sums by authorative laboratories and outstanding care institutions has allowed us to get to more and more satisfactory results in terms of quality of life and long-term survival of young patients.
Neuroblastoma has a distintive trait which differentiates it from adult cancer. Since it’s onset occurs very early during development, in some rare cases even in uterus, cells that produce it have had little time to develop and accumulate mutations. This knowledge comes from the possibility of analysing the entire genome sequence. Neuroblastoma is known as the tumor with the least mutated genome ever. It is therefore evident that children affected by Neuroblastoma are genetically predisposed to develop the disease and therefore carry risk mutations inherited from their parents. The GENEDREN project aims to identify these alterations, incrase knowledge about the development of Neuroblastoma and transfer them to clinic. The project can be realized in three to five years and requires to be done a total investment of 1,152,000 euros, of which 791,000 are borne by the FNb and 361,000 by co-financing of other organisations.
The goal is the development of a new, innovative and promising immunotherapy approach for patients with high-risk to develop Neuroblastoma (NB). To date, only immunotherapy based on the use of ant-GD2 antibody (indicates the drug produced by “dinutuximab”) that recognizes GD2 protein expressed on the surface of NB cells, is used in the United States and Europe for patiens with high-risk NB. In this context, one should keep in mind that a cellular protein, in order to be considered a target that can “strike” safely, should be abundantly expressed by cancer cells but it shouldn’t be on healthy cells. These traits have been identified in the B7-H3 protein, which becomes again a new potential immunotherapy target for NB. With these premises I twill be possible to develop an innovative treatment, already effective in several adult tumour models: the aim is to verify if also on pediatric tumors, particulary on NB same results can be obtained. If the already existing studies will confirm it we can move on to clinical trials on young patients to improve prognosis in patients affected by NB, relapsing or refractory to chemotherapy.
“Pensiero Project” was born in 2008 from the need to offer the scientific community information and tools to reduce diagnostic discrepancies as to say those that determine a change in therapy for the young patient.
The percentage of such discrepancies has decreased from an initial 35% to today’s 0.1%.
This has been possible thanks to the histopathological centralization of pediatric Central Nervous System (CNS) tumor cases. The synergy and collaboration between treatment centers of excellence such as Bambino Gesù Hospital in Rome, Santobono Hospital in Naples, Meyer Hospital in Florence, Bellaria Hospital, City of Science and Health in Turin, Gaslini Institute in Genoa, Besta Hospital in Milan and others coordinated by dr. Giangaspero from the University La Sapienza of Rome has allowed the drafting and expansion of CNS registry of tumors, as well as updating the “virtual” biological bank that represents about 32,5%(884/2723) of centralized cases of which we have biological material available for basic and translational studies.
An average of approximately 240 new cases are enrolled each year.
Neuroslatoma (NB) is the most frequent extracranial solid tumor of pediatric age (8-10% of all tumors in the age group 0-16 years) with about 130 diagnosed cases every year in Italy.
Since 1976, researchers from Giannina Gaslini Institute have been coordinating, within AIEOP, what is currently called as the “Neuroblastoma Working Group” (GdLNB), which has the task of activating national and international therapy protocols and planning scientific research about this neoplasm.
Institute Gaslini in 1994 was one of the 4 founding centers of the European Cooperative Group, which today can count 26 member states. In 2016 it was recognized by Liguria region as a reference center for Neuroblastoma and in 2017 it was included among the European reference centers within the Paediatric Cancer European Reference Network (PaedCan ERN).