Novel combination therapies to combat therapy resistant neuroblastoma: translating pre-clinical investigations to clinical trials

Neuroblastoma is an aggressive paediatric solid tumour originating from the sympathetic nervous system. New combination therapies are urgently warranted as high-risk neuroblastoma has a poor prognosis.
The aims of this project are two-fold: (1) further evaluate the efficacy and toxicity of nutlin, alectinib, palbociclib, silvestrol and TMPYP4 in combination with currently used chemotherapeutic agents in neuroblastoma treatment (2) to determine the therapeutic efficacy and toxicity of the individual compounds and most promising combinations of these compounds with currently used chemotherapeutics, in vivo using patient-derived tumour xenografts and neuroblastoma zebrafish models.
The ultimate goal is to offer compelling pre-clinical data and a framework to support and initiate the clinical evaluation of the most promising compounds in combination with conventional chemotherapy in patients with neuroblastoma to improve outcome and reduce toxicity.