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Professor Nick GottardoHead of Paediatric and Adolescent Oncology and Haematology, Perth Children’s Hospital; Co-head, Brain Tumour Research Program, The Kids Research Institute Australia
Research
Integrated Analysis of miRNA and mRNA Expression in Childhood Medulloblastoma Compared with Neural Stem CellsMedulloblastoma (MB) is the most common malignant brain tumor in children and a leading cause of cancer-related mortality and morbidity.
Research
Integrated analysis of miRNA and mRNA expression in childhood MedulloblastomaMedulloblastoma (MB) is the most common malignant brain tumor in children and a leading cause of cancer-related mortality and morbidity.
Research
A novel transcriptional signature identifies T-cell infiltration in high-risk paediatric cancerMolecular profiling of the tumour immune microenvironment (TIME) has enabled the rational choice of immunotherapies in some adult cancers. In contrast, the TIME of paediatric cancers is relatively unexplored. We speculated that a more refined appreciation of the TIME in childhood cancers, rather than a reliance on commonly used biomarkers such as tumour mutation burden (TMB), neoantigen load and PD-L1 expression, is an essential prerequisite for improved immunotherapies in childhood solid cancers.
Research
ONC201 in Combination with Paxalisib for the Treatment of H3K27-Altered Diffuse Midline GliomaDiffuse midline gliomas (DMG), including diffuse intrinsic pontine gliomas (DIPG), are the most lethal of childhood cancers. Palliative radiotherapy is the only established treatment, with median patient survival of 9 to 11 months. ONC201 is a DRD2 antagonist and ClpP agonist that has shown preclinical and emerging clinical efficacy in DMG.
Research
Coupling of response biomarkers between tumor and peripheral blood in patients undergoing chemoimmunotherapyPlatinum-based chemotherapy in combination with anti-PD-L1 antibodies has shown promising results in mesothelioma. However, the immunological mechanisms underlying its efficacy are not well understood and there are no predictive biomarkers to guide treatment decisions.
Research
Evaluation of age-dependent treatment strategies for children and young adults with pineoblastoma: Analysis of pooled European Society for Paediatric Oncology (SIOP-E) and US Head Start dataPineoblastoma is a rare pineal region brain tumor. Treatment strategies have reflected those for other malignant embryonal brain tumors.
Research
A Phase I Study of the CDK4/6 Inhibitor Ribociclib (LEE011) in Pediatric Patients with Malignant Rhabdoid Tumors, Neuroblastoma, and Other Solid TumorsIn this. i study the MTD and RP2D, safety, PK, and preliminary activity of single-agent ribociclib were investigated in patients with neuroblastoma.
Research
Contemporary survival endpoints: An international diffuse intrinsic pontine glioma registry studyThis study defines PFS and OS, and is the first describe post-progression survival in a large cohort of children with DIPG.
Research
'Walking their walk': Reducing conflict between families of ill children and the medical professionI review recent high-profile court cases to underscore the changing landscape of conflict and the central role that the media (and social media in particular) can play