The Australian and New Zealand Children's Haematology/Oncology Group (ANZCHOG) and the Australian Brain Cancer Mission (ABCM) are strategically deploying funds and fostering collaborations to tackle barriers hindering clinical trial execution for pediatric brain cancer. These efforts aim to improve outcomes for children and families affected by this devastating disease, which remains the most significant cause of cancer-related deaths in the 0-14 age group, accounting for 40% of fatalities despite representing only 14% of diagnoses.
Overcoming Barriers to Clinical Trials
Conducting clinical trials in pediatric oncology presents unique challenges, particularly in regions with small, geographically dispersed populations. The rarity of specific diagnoses necessitates international collaboration to achieve sufficient patient accrual within feasible timelines. Limited grant funding further complicates matters, as the time required for patient accrual and meaningful outcome data often exceeds funding periods. The specificity of diagnoses and the need for complex, multi-agent therapies also reduce the appeal of these trials to pharmaceutical companies, which tend to favor single-drug therapies. Consistent funding is therefore essential to maintain a skilled workforce for clinical trial conduct and monitoring.
ABCM and ANZCHOG: A Collaborative Approach
ABCM provides the financial support needed for partnerships and trial capacity enhancement, while ANZCHOG leads in navigating the challenges of pediatric oncology trials, building a network that overcomes logistical and financial constraints to facilitate progress and discovery. Initiated in 2017, ABCM aims to double brain cancer survival rates through strategic resource mobilization, collaborations, and research. This mission is supported by a detailed strategic framework that blends substantial financial backing with extensive stakeholder engagement and a co-funded model uniting government, philanthropic, and private sectors.
The ANZCHOG Central Nervous System (CNS) Tumour Group complements this aim by leading the prioritization of research areas and creating a clinical trial development pipeline encompassing identification, development, and funding application for trials. This vision aims to unlock new therapeutic avenues by harnessing a multidisciplinary approach and leveraging the ABCM.
Impact of Collaboration and Funding
The collaboration between ABCM and ANZCHOG CNS Tumour Group has led to the initiation of or expansion to additional sites of 11 clinical trials. These trials address various tumor types, including newly diagnosed and recurrent cases, signifying a move forward in the field and demonstrating the impact of collaborative efforts to surpass traditional research barriers. These trials range from testing innovative treatments to improve cognitive and neurological benefits, as well as the quality of life of survivors. Trials such as SJ-ELiOT, COZMOS (NCT03206021), and INFORM2 NivEnt (NCT03838042) exemplify ABCM's impact. COZMOS evaluated new combinational treatment protocols designed to improve survival rates for children with aggressive brain cancers, while INFORM2 NivEnt pioneers a precision medicine approach, tailoring treatments based on the genetic characteristics of individual tumors.
ANZCHOG's involvement in international consortia has significantly expanded, with six new partnerships in addition to existing collaborations with international cooperative groups such as the Children's Oncology Group (COG) and the International Society of Paediatric Oncology (SIOP). This ensures that Australian and New Zealand children have access to an increased number of global clinical trials, especially innovative early-phase trials, keeping Australasian pediatric oncology at the forefront of global research efforts.
Challenges and Future Directions
Despite the strides made, resource constraints remain a significant hurdle, with approximately 67% of clinical research staff funding derived from external, non-operational resources (ANZCHOG data), highlighting a dependency on philanthropy and external grants for sustainability. The transient nature of funding, with a significant portion of clinical research associates and clinical research nurses operating on short-term contracts, exacerbates the challenges of recruitment and retention. These challenges underscore the necessity for sustained and strategic funding mechanisms that go beyond the ABCM to maintain the current momentum.
The need for diverse treatment options, particularly investigational therapies when standard care falls short, underscores the importance of sustained and increased investment in research and clinical trials. Such commitment is vital to fulfill community expectations for accessible clinical trials, especially for conditions with challenging prognoses, ensuring families can find hope without the burden of seeking treatments abroad. To build on this progress, it is essential to navigate the existing challenges with innovative funding models, regulatory agility, and strengthened international partnerships.
