Diffuse 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.  ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2023
• May 2023

Raelene Nick Endersby Gottardo R NG BSc (Hons) PhD MBChB FRACP PhD Brainchild Fellow; Co-Head, Brain Tumour Research Co-Head, Brain Tumour Research raelene.endersby@telethonkids.org.au nicholas.gottardo@telethonkids.org.au Co-Head, Brain Tumour ...

• 1. Telethon Kids Institute
• Projects

Patient-derived orthotopic xenograft (PDOX) mouse models are considered the gold standard for evidence-based preclinical research in pediatric neuro-oncology. This protocol describes the generation of PDOX models by intracranial implantation of human pediatric brain cancer cells into immune-deficient mice, and their continued propagation to establish cohorts of animals for preclinical research. ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2024
• April 2024

Raelene Nick Endersby Gottardo R NG BSc (Hons) PhD MBChB FRACP PhD Brainchild Fellow; Co-Head, Brain Tumour Research Co-Head, Brain Tumour Research raelene.endersby@telethonkids.org.au nicholas.gottardo@telethonkids.org.au Co-Head, Brain Tumour ...

• 1. Telethon Kids Institute
• Projects

Medulloblastoma is the most common malignant paediatric brain tumour and a leading cause of cancer-related mortality and morbidity. Existing treatment protocols are aggressive in nature resulting in significant neurological, intellectual and physical disabilities for the children undergoing treatment. Thus, there is an urgent need for improved, targeted therapies that minimize these harmful side effects. ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2021
• June 2021

Nick Raelene Gottardo Endersby NG R MBChB FRACP PhD BSc (Hons) PhD Co-Head, Brain Tumour Research Brainchild Fellow; Co-Head, Brain Tumour Research nicholas.gottardo@telethonkids.org.au raelene.endersby@telethonkids.org.au Co-Head, Brain Tumour ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2023
• August 2023

The mitogen-activated protein kinase (MAPK) pathway signaling pathway is one of the most commonly mutated pathways in human cancers. In particular, BRAF alterations result in constitutive activation of the rapidly accelerating fibrosarcoma-extracellular signal-regulated kinase-MAPK significant pathway, leading to cellular proliferation, survival, and dedifferentiation. ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2023
• April 2023

We report the case of a 45-year-old woman who was diagnosed with a NOS based on immunohistochemical analysis of the patient's tumor at diagnosis. ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2018
• January 2018

Raelene Nick Endersby Gottardo R NG BSc (Hons) PhD MBChB FRACP PhD Brainchild Fellow; Co-Head, Brain Tumour Research Co-Head, Brain Tumour Research raelene.endersby@telethonkids.org.au nicholas.gottardo@telethonkids.org.au Co-Head, Brain Tumour ...

• 1. Telethon Kids Institute
• Projects

Raelene Nick Endersby Gottardo R NG BSc (Hons) PhD MBChB FRACP PhD Brainchild Fellow; Co-Head, Brain Tumour Research Co-Head, Brain Tumour Research raelene.endersby@telethonkids.org.au nicholas.gottardo@telethonkids.org.au Co-Head, Brain Tumour ...

• 1. Telethon Kids Institute
• Projects

Raelene Nick Endersby Gottardo R NG BSc (Hons) PhD MBChB FRACP PhD Brainchild Fellow; Co-Head, Brain Tumour Research Co-Head, Brain Tumour Research raelene.endersby@telethonkids.org.au nicholas.gottardo@telethonkids.org.au Co-Head, Brain Tumour ...

• 1. Telethon Kids Institute
• Projects

Optic pathway gliomas (OPGs) are associated with significant risk of visual and endocrine morbidity, but data on long-term outcomes in symptomatic patients is sparse. This study reviews the clinical course, disease progression, survival outcomes and long-term sequelae in pediatric patients with symptomatic OPGs in our institution over three decades. ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2023
• July 2023