hAEmatology and oncology
Anti-GD2 mAbs for
the treatment of high-risk
neuroblastoma
Anti-GD2 monoclonal antibodies have recently been approved
by the EU and US regulatory agencies and are currently the only formally
approved drugs for the treatment of high-risk neuroblastoma
Jaume Mora MD PhD
Department of
Pediatric Oncology and
Hematology, Hospital
Sant Joan de Déu,
Barcelona, Spain
Neuroblastoma is the most common extracranial
paediatric solid tumour in Europe. Close to 40% of
all neuroblastoma patients are classified as high
risk (HR) because the chance of relapse or death
within two years of diagnosis is close to 50%.
Immunotherapy with anti-GD2 monoclonal
antibodies (mAbs) is now considered the only
important therapeutic advance in the treatment
of HR neuroblastoma in the last decade. The
availability and sustainability of these antibodies
in the market as well as the cost they represent
within an already expensive treatment for
neuroblastoma patients are relevant issues
affecting parents, physicians, regulatory bodies
and insurers.
Neuroblastic tumours
Neuroblastic tumours (NBTs) derive from
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HHE 2018 | hospitalhealthcare.com
primordial neural crest cells and are the most
common extracranial solid childhood tumours
in Europe. Under normal conditions, neural crest
cells delaminate and migrate from the dorsal
neural tube, and those neuroblastic precursor
cells differentiate upon reaching their final
embryonic location into tissues and organs that
will constitute the sympathetic nervous system.
In vitro and in vivo studies have shown that NBTs
originate from a block in the process of normal
differentiation of these precursor cells. 1,2
Histologically, NBTs are classified in three
categories: neuroblastoma (NB);
ganglioneuroblastoma (GNB); and
ganglioneuroma (GN). By definition, Schwannian
stroma should comprise less than 50% of the
tumour tissue to be NB. Undifferentiated NB is
composed of neuroblastic cells without