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Future plans are to intensify treatment for those people with aberration of 1p36 or 11q23 chromosomes as well as for those who lack early response to treatment. [83] [84] By contrast, focus the past 20 years or more has been to intensify treatment for high-risk neuroblastoma.
Treatment using these targets can supplement or replace some of the intensive chemotherapy that is used for neuroblastoma. These molecular targets of this disease include GD2 , ALK , and CD133 . GD2 is a target of immunotherapy , and is the most fully developed of these treatment methods, but is also associated with toxicities. [ 1 ]
GD2 is a disialoganglioside expressed on tumors of neuroectodermal origin, including human neuroblastoma and melanoma, with highly restricted expression on normal tissues, principally to the cerebellum and peripheral nerves in humans.
The degree of differentiation of neuroblastoma is correlated to the prognosis, with a wide variety of outcomes (from tumor regression to recurrence and mortality). The standard of care is the use of chemotherapy, surgical resection and radiation, although most aggressive neuroblastomas have demonstrated to be resistant to these therapies.
The tumor will then need to be biopsied to confirm the diagnosis. After the diagnosis of a CNS PNET is confirmed, management includes neoadjuvant chemotherapy and radiation (to reduce tumor size burden), complete surgical resection with confirmed negative margins, and/or additional adjuvant post-surgical chemotherapy.
The current (5th) edition of the WHO Classification of Tumors of the Central Nervous System classifies embryonal tumor of the central nervous system into six subtypes: medulloblastoma, cribiform neuroepithelial tumor, embryonal tumor with multilayered rosettes, CNS neuroblastoma, FOXR2-activated, CNS tumor with BCOR internal tandem duplication, and CNS embryonal tumor. [1]
Chemotherapy is used in a multimodality treatment plan generally for more advanced, unresectable or reoccurring tumors. [4] Cyclophosphamide, vincristine and doxorubicin have been used as neoadjuvant chemotherapy drugs for grade C esthesioneuroblastoma before surgical resection, producing fair outcomes.
The Neuroblastoma Alliance UK helps families access and fundraise for their child's treatment abroad. Various new treatments for neuroblastoma are being trialled in the UK, but if children do not meet the criteria for a trial, or need treatment that is only available abroad, families have to pay for their child's treatment abroad.