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Clinical Trials in the Brain Tumour Population: Challenges and Strategies for the Future
Hui Kong Gan1,2,3,4, Bryan W Day5,6,7,8, Rosemary Harrup9,10
1Cancer Therapies and Biology Group, Centre for Research Excellence in Brain Cancer, Olivia Newton-John Cancer Research Institute, Austin Health, 145 Studley Road, Heidelberg, Melbourne, VIC, 3084, Australia. hui.gan@onjcri.org.au.
Purpose Of Review:
This review identifies challenges and barriers to successful development of drugs in neuro-oncology trials at the preclinical, clinical and translational stages that we believe has contributed to poor outcomes for patients over the last 30 years.
Recent Findings:
Several key strategies have been proposed by leading groups to address these and improve patient outcomes. Better preclinical testing using more sophisticated and clinically relevant models is needed. A greater focus on assessing blood-brain barrier penetrance and targeting key biological processes such as tumour heterogeneity and immune response is vital. Adopting innovative trial designs permitting faster results and addressing key issues (including molecular heterogeneity and combinatorial approaches) is highly desirable. A stronger translational focus is also clearly needed. Implementation of these strategies is already starting to occur. Maintaining and increasing these novel approaches will require coordinated efforts between clinicians, scientists, industry and funding/regulator bodies.
Insights
Developing new neuro-oncology drugs faces significant preclinical, clinical, and translational hurdles, hindering patient progress for decades. Addressing these challenges requires innovative models, better blood-brain barrier assessment, and improved trial designs for better brain tumor treatment outcomes.
Area of Science:
- Neuro-oncology
- Drug Development
- Translational Science
Background:
- Neuro-oncology drug development has faced persistent challenges over the past 30 years.
- This has led to suboptimal outcomes for patients with brain tumors.
Purpose of the Study:
- To identify key challenges and barriers in preclinical, clinical, and translational neuro-oncology drug development.
- To propose strategies for improving patient outcomes in brain tumor treatment.
Main Methods:
- Review of existing literature and identification of critical obstacles in neuro-oncology trials.
- Analysis of proposed strategies by leading research groups.
Main Results:
- Significant barriers exist at preclinical, clinical, and translational stages.
- Key strategies include improved preclinical models, focus on blood-brain barrier penetration, targeting tumor heterogeneity and immune response, and innovative trial designs.
Conclusions:
- Addressing neuro-oncology drug development challenges requires a multi-faceted approach.
- Enhanced collaboration between researchers, clinicians, industry, and regulatory bodies is crucial for advancing brain tumor therapies.
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