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IDH mutant high-grade gliomas

Gliomas are the most common type of malignant primary central nervous system (CNS) tumors, resulting in significant morbidity and mortality in children and adolescent and young adult (AYA) patients. The discovery of mutations in isocitrate dehydrogenase (IDH) genes has dramatically changed the classification and understanding of gliomas.  IDH mutant gliomas have distinct clinical, pathological, and molecular features including a favorable prognosis and response to therapy compared to their wildtype counterparts.

Conventional Therapies Deplete Brain-Infiltrating Adaptive Immune Cells in a Mouse Model of Group 3 Medulloblastoma Implicating Myeloid Cells as Favorable Immunotherapy Targets

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.

“If you build it, they will come”: the convergence of funding, research and collaboration in paediatric brain cancer clinical trials

Each year, approximately 1000 children in Australia and New Zealand, aged 0–14 years, are diagnosed with cancer. Despite paediatric cancer accounting for less than 1% of all cancer cases, the impact on their families and communities is profound and disproportionate.

In their own words: advice from parents of children with cancer

Approximately 770 children are diagnosed with cancer in Australia every year. Research has explored their experiences and developed recommendations for improving support provided to families. These have included the provision of psychology services, improved communication between healthcare professionals and parents, and increased information for families. 

Clinical, Radiologic, Pathologic, and Molecular Characteristics of Long-Term Survivors of Diffuse Intrinsic Pontine Glioma

We report clinical, radiologic, and molecular factors that correlate with survival in children and young adults with diffuse intrinsic pontine glioma

The Kids researchers awarded Raine Medical Research Foundation funding

Congratulations to three The Kids Research Institute Australia researchers, who have been awarded funding from the Raine Medical Research Foundation.

Gene-based outcome prediction in multiple cohorts of pediatric T-cell acute lymphoblastic leukemia: a Children's Oncology Group study

Continuous complete clinical remission in T-cell acute lymphoblastic leukemia (T-ALL) is now approaching 80% due to the implementation of aggressive...

Influence of wild-type MLL on glucocorticoid sensitivity and response to DNA-damage in pediatric acute lymphoblastic leukemia

Rearrangement of the mixed-lineage leukemia gene (MLL) is found in 80% of infant acute lymphoblastic leukemia (ALL) and is associated with poor prognosis and re

Diffuse Intrinsic Pontine Glioma

This chapter summarizes recent advances in diffuse intrinsic pontine glioma and potential novel therapies

Developing and characterising juvenile models of aggressive paediatric brain cancers for the evaluation of novel immunotherapies.

While profound treatment responses have been realised using immunotherapy for some cancer types, this is yet to be seen for paediatric brain cancer patients.

Finding new, safer and targeted therapies for paediatric brain cancer that amplify responses to radiation therapy

Radiation therapy is an essential component of brain cancer treatment. However, the high doses currently required are extremely damaging to the growing brains and bodies of children.

Developing new immune based therapies for neuroblastoma

Neuroblastoma is a complex childhood cancer of the nerve cells and the most common solid tumour in children outside of the brain. The average age of diagnosis is 1-2 years and tragically 50% of children with high-risk neuroblastoma lose their battle within five years.

Irreversible growth plate fusions in children with medulloblastoma treated with a targeted hedgehog pathway inhibitor

We report on 3 children treated with vismodegib who developed widespread growth plate fusions that persist long after cessation of therapy.