Infants with KMT2A-rearranged B-cell precursor acute lymphoblastic leukemia (ALL) have poor outcomes. There is an urgent need to identify novel agents to improve survival. Proteasome inhibition has emerged as a promising therapeutic strategy for several hematological malignancies. The aim of this study was to determine the preclinical efficacy of the selective proteasome inhibitor carfilzomib, for infants with KMT2A-rearranged ALL. ...

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

The bone marrow microenvironment (BMM) plays a key role in leukemia progression, but its molecular complexity in pre-B cell acute lymphoblastic leukemia (B-ALL), the most common cancer in children, remains poorly understood. To gain further insight, we used single-cell RNA sequencing to characterize the kinetics of the murine BMM during B-ALL progression. ...

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

Structural and numerical alterations of chromosome 21 are extremely common in hematological malignancies. While the functional impact of chimeric transcripts from fused chromosome 21 genes such as TEL-AML1, AML1-ETO, or FUS-ERG have been extensively studied, the role of gain of chromosome 21 remains largely unknown. ...

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

IL7 and glucocorticoids coordinately drive aberrant activation of PIM1 and suggests that T-ALL and T-LBL patients could benefit from PIM inhibition ...

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

Persistent regional and systemic inflammation may promote pain and hyperalgesia in complex regional pain syndrome. In this study, we investigated whether stimulation of α1-adrenoceptors on peripheral blood mononuclear cells might contribute to this inflammatory state. ...

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

Cardio-oncology is a new multidisciplinary area of expertise that seeks to pre-emptively and proactively address cardiac complications that emerge during and following cancer therapy. Modern therapies including molecular targeted therapy and immunotherapy have broadened the agents that can cause cardiac sequelae, often with complications arising within days to weeks of therapy. ...

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

We provide evidence that targeting leukemia-induced bone loss is a therapeutic strategy for pre-B ALL ...

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

We conclude that the novel chocolate-based formulation of midazolam provides improved tolerability while remaining efficacious ...

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

Children with Primary Central Nervous System Lymphoma and no immunodeficiency have a good outcome ...

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

Rare childhood cancers have not benefited to the same extent from the gains that have been made for their frequently occurring counterparts. ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2015
• February 2015

Connective tissue growth factor (CTGF/CCN2) has long been associated with human cancers. The role it plays in these neoplasms is diverse and tumour specific. ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2014
• May 2014