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Clinical Trials in the Brain Tumour Population: Challenges and Strategies for the Future

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Abstract

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.

Summary

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.

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Authors and Affiliations

  1. Cancer 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 Kong Gan

  2. Tumour Targeting Program and Centre for Research Excellence in Brain Cancer, Olivia Newton-John Cancer Research Institute, Austin Health, 145 Studley Road, Heidelberg, Melbourne, VIC, 3084, Australia

    Andrew Mark Scott

  3. La Trobe University School of Cancer Medicine, Heidelberg, VIC, Australia

    Hui Kong Gan & Andrew Mark Scott

  4. Sid Faithfull Brain Cancer Laboratory, Cell and Molecular Biology Department, QIMR Berghofer, 300 Herston Rd, Herston, QLD, 4006, Australia

    Bryan W. Day

  5. Department of Medicine, University of Melbourne, Heidelberg, VIC, Australia

    Hui Kong Gan & Andrew Mark Scott

  6. School of Biomedical Sciences, Queensland University of Technology, Brisbane, QLD, Australia

    Bryan W. Day

  7. Australian Brain Cancer Research Alliance, Melbourne, Australia

    Hui Kong Gan, Bryan W. Day, Terrance G. Johns & Andrew Mark Scott

  8. Faculty of Medicine, The University of Queensland, Brisbane, QLD, Australia

    Bryan W. Day

  9. Cancer & Blood Services, Royal Hobart Hospital, Hobart, TAS, Australia

    Rosemary Harrup

  10. Menzies Research Institute, University of Tasmania, Hobart, TAS, Australia

    Rosemary Harrup

  11. Oncogenic Signalling Laboratory, Telethon Kids Institute, Nedlands, WA, Australia

    Terrance G. Johns

  12. Department of Medical Oncology, Cancer Care Services, Royal Brisbane and Women’s Hospital, The University of Queensland, Brisbane, QLD, Australia

    Zarnie Lwin

  13. NHMRC Clinical Trials Centre, Locked Bag 77, Camperdown, NSW, 1450, Australia

    Hao-Wen Sim

  14. Department of Medical Oncology, Chris O’Brien Lifehouse, Camperdown, NSW, Australia

    Hao-Wen Sim

  15. Kinghorn Cancer Centre, St Vincent’s Hospital Sydney, Darlinghurst, NSW, Australia

    Hao-Wen Sim

  16. School of Clinical Medicine, Faculty of Medicine and Health, University of New South Wales, Darlinghurst, NSW, Australia

    Hao-Wen Sim

  17. South West Clinical School, Faculty of Medicine, University of New South Wales, Sydney, NSW, Australia

    Eng-Siew Koh

  18. Department of Radiation Oncology, Liverpool Cancer Therapy Centre, Liverpool, NSW, Australia

    Eng-Siew Koh

  19. Ingham Institute for Applied Medical Research, Liverpool Hospital, Locked Bag 7103, Liverpool, NSW, Australia

    Eng-Siew Koh

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  1. Hui Kong Gan
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  2. Bryan W. Day
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  3. Rosemary Harrup
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  4. Terrance G. Johns
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  7. Hao-Wen Sim
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Correspondence to Hui Kong Gan.

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Gan, H.K., Day, B.W., Harrup, R. et al. Clinical Trials in the Brain Tumour Population: Challenges and Strategies for the Future. Curr Oncol Rep 25, 589–598 (2023). https://doi.org/10.1007/s11912-023-01394-5

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