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

Glioblastoma in adults, and medulloblastoma and pineoblastoma that mainly affect children, are aggressive brain tumors. ...

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

Medulloblastoma is the most common malignant childhood brain tumor, and 5-year overall survival rates are as low as 40% depending on molecular subtype, with new therapies critically important. As radiotherapy and chemotherapy act through the induction of DNA damage, the sensitization of cancer cells through the inhibition of DNA damage repair pathways is a potential therapeutic strategy. ...

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

Medulloblastoma (MB) is the most common malignant brain tumor in children and a leading cause of cancer-related mortality and morbidity. ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2011
• September 2011

Genetic predisposition to proteome instability may be a determinant in the pathogenesis of paediatric brain cancers ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2020
• April 2020

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

Ependymomas (EPN) are the third most common malignant brain cancer in children. Treatment strategies for pediatric EPN have remained unchanged over recent decades, with 10-year survival rates stagnating at just 67% for children aged 0-14 years. Moreover, a proportion of patients who survive treatment often suffer long-term neurological side effects as a result of therapy. It is evident that there is a need for safer, more effective treatments for pediatric EPN patients. ...

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

This multi-center study provides valuable information on the success rate of establishing patient-derived pre-clinical models of diffuse intrinsic pontine glioma ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2019
• January 2019

Medulloblastoma is the most common childhood brain cancer. Mainstay treatments of radiation and chemotherapy have not changed in decades and new treatment approaches are crucial for the improvement of clinical outcomes. To date, immunotherapies for medulloblastoma have been unsuccessful, and studies investigating the immune microenvironment of the disease and the impact of current therapies are limited. ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2022
• March 2022

The induction of DNA damage has been employed as an anticancer strategy for more than 100years, first starting with the use of radiation to treat stomach cancer followed by the first uses of DNA-damaging chemotherapy to treat childhood leukemia. ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2022
• January 2022

The functional effects of increased ERBB4 activation identify ERBB4 as a potential prognostic and therapeutic target ...

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

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