Nick Gottardo NG MBChB FRACP PhD Co-Head, Brain Tumour Research nicholas.gottardo@telethonkids.org.au Co-Head, Brain Tumour Research Professor Nick ...

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

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

While chemotherapy remains the first-line treatment for many cancers, it is still unclear what distinguishes responders from non-responders. Here, we characterize the chemotherapy-responsive tumor microenvironment in mice, using RNA sequencing on tumors before and after cyclophosphamide, and compare the gene expression profiles of responders with progressors. ...

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

Children with Down syndrome (DS) are at a significantly higher risk of developing acute myeloid leukemia, also termed myeloid leukemia associated with DS (ML-DS). In contrast to the highly favorable prognosis of primary ML-DS, the limited data that are available for children who relapse or who have refractory ML-DS (r/r ML-DS) suggest a dismal prognosis. There are few clinical trials and no standardized treatment approach for this population. ...

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

Chronic lymphocytic leukemia patients have an increased bleeding risk with the introduction of Bruton tyrosine kinase (BTK) inhibitors. BTK is a signaling effector downstream of the platelet GPVI receptor. Innate platelet dysfunction in CLL patients and the contribution of the leukemia microenvironment to the anti-platelet effect of BTK inhibitors are still not well defined. ...

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

Pineoblastoma is a rare brain tumor usually diagnosed in children. Given its rarity, no pineoblastoma-specific trials have been conducted. Studies have included pineoblastoma accruing for other embryonal tumors over the past 30 years. ...

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

Acute Lymphoblastic Leukemia (ALL) occurring in the first year of life is rare. ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2012
• September 2012

Childhood brain tumors (CBT) are the leading cause of cancer death in children, yet their causes are largely known. This study investigated the association... ...

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

This study contributes to our understanding of the genetic diversity of NMC, an important step towards finding therapeutic targets for a disease that is... ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2012
• November 2012

An Australian study of childhood leukaemia (Aus-ALL) previously reported that control participation was positively associated with socio-economic status... ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2012
• May 2012