T cell acute lymphoblastic leukemia (T-ALL) is an aggressive hematologic malignancy with inferior outcome compared with that of B cell ALL. Here, we show that Runt-related transcription factor 2 (RUNX2) was upregulated in high-risk T-ALL with KMT2A rearrangements (KMT2A-R) or an immature immunophenotype. In KMT2A-R cells, we identified RUNX2 as a direct target of the KMT2A chimeras, where it reciprocally bound the KMT2A promoter, establishing a regulatory feed-forward mechanism. ...

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

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

We describe two unusual cases of MPE and use DNA methylation analyses to compare their signatures to try and distinguish if these represent a unique subset. ...

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

Our approach to hematopoietic progenitor cells mobilization resulted in highly effective HPC harvest in children and adolescents with high-risk cancers ...

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

Although neurocognitive, psychological and behavioural problems were noted for some patients during medical review, only 20% of patients were formally assessed. ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2013
• May 2013

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

Extensive research over the past 50 years has resulted in significant improvements in survival for patients diagnosed with leukemia. Despite this, a subgroup of patients harboring high-risk genetic alterations still suffer from poor outcomes. There is a desperate need for new treatments to improve survival, yet consistent failure exists in the translation of in vitro drug development to clinical application. ...

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

Acute leukemia continues to be a major cause of death from disease worldwide and current chemotherapeutic agents are associated with significant morbidity in survivors. While better and safer treatments for acute leukemia are urgently needed, standard drug development pipelines are lengthy and drug repurposing therefore provides a promising approach. ...

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

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

Patients whose leukemias harbor a rearrangement of the Mixed Lineage Leukemia (MLL/KMT2A) gene have a poor prognosis, especially when the disease strikes in infants. The poor clinical outcome linked to this aggressive disease and the detrimental treatment side-effects, particularly in children, warrant the urgent development of more effective and cancer-selective therapeutics. ...

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

Children with Down syndrome (constitutive trisomy 21) that develop acute lymphoblastic leukemia (DS-ALL) have a 3-fold increased likelihood of treatment-related mortality coupled with a higher cumulative incidence of relapse, compared with other children with B-cell acute lymphoblastic leukemia (B-ALL). ...

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

Our findings shed light on the mechanisms of leukemia-induced bone loss ...

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