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Research

The critical role of the bone marrow stromal microenvironment for the development of drug screening platforms in leukemia

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.

Research

Pharmacokinetics of PEGasparaginase in Infants with Acute Lymphoblastic Leukemia

PEGasparaginase is known to be a critical drug for treating pediatric acute lymphoblastic leukemia (ALL), however, there is insufficient evidence to determine the optimal dose for infants who are less than one year of age at diagnosis. This international study was conducted to identify the pharmacokinetics of PEGasparaginase in infants with newly diagnosed ALL and gather insight into the clearance and dosing of this population.

Research

FDA-approved disulfiram as a novel treatment for aggressive leukemia

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.

Research

Cancer therapies inducing DNA damage

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.

Research

Antifungal use in children with acute leukaemia: state of current evidence and directions for future research

Invasive fungal disease (IFD) remains a common and serious complication in children treated for leukaemia. Antifungal prescription in children with leukaemia presents unique challenges, particularly due to variation in IFD risk between and within leukaemia treatment protocols, drug toxicities and interactions between antifungals and chemotherapeutic agents.

News & Events

Childhood cancers not on the rise in SA and NT, but disparity remains for Indigenous children in the NT

Despite national and global reports of rising incidences of cancer affecting children and young people, a new analysis has found rates of childhood cancer have remained unchanged over the last 30 years in South Australia and the Northern Territory.

News & Events

Precision medicine offering new hope for kids with aggressive cancers

A world-first study involving more than 100 cancer researchers and clinicians across Australia – including from The Kids Research Institute Australia and Perth Children’s Hospital – has shown that precision medicine can significantly improve outcomes for children with high-risk cancer.

Research

ONC201 in Combination with Paxalisib for the Treatment of H3K27-Altered Diffuse Midline Glioma

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. 

Research

“I Don’t Get to Play With My Mum Anymore”: Experiences of Siblings Aged 8–12 of Children With Cancer: A Qualitative Study

Siblings of children with cancer have been shown to experience disruption in multiple domains including family, school, and friendships. Existing literature on siblings' experiences focuses on older children or on a broad range of ages.

Research

Population-level 5-year event-free survival for children with cancer in Australia

Event-free survival considers other adverse events in addition to mortality. It therefore provides a more complete understanding of the effectiveness and consequences of treatment than standard survival measures, but is rarely reported at the population level for childhood cancer.