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Preclinical rare cancer research to inform clinical trial design

Nature Reviews Cancer volume 19, pages 481–482 (2019)Cite this article

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With improved biobanking of rare cancer tissue enabling more in-depth tumour profiling, as well as generation of clinically relevant rare cancer preclinical models, treatment strategies for patients with rare cancer will rapidly advance. The future successes of rare cancer research, with alternative approaches for diagnosing, researching and conducting clinical trials, will ultimately benefit patients with all types of cancer.

Collectively, rare cancers (those with an incidence of <6 out of 100,000 per year) account for ~25–30% of all cancer diagnoses and 25% of cancer deaths (as defined by RARECARE, a Consortium for the Surveillance of Rare Cancers in Europe), representing a substantial burden of disease. However, basic science research, clinical trials and approval of new therapies for rare cancers are lacking. This translates into a worse prognosis for patients with rare cancer than for those with common cancers, with a 5-year survival of 48.5% compared with 63.4%, respectively1. As research evolves and common cancers are reclassified based on molecular and genomic markers into rare subtypes, the novel ways in which rare cancers are being studied will be extended to all cancer types. Consequently, with the number of rare cancers increasing (currently rising by 0.5% annually1), finding more appropriate solutions for diagnosing, managing and studying rare cancers is essential.

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Acknowledgements

This work was supported by fellowships from the Stafford Fox Medical Research Foundation (C.L.S. and H.E.B.); Cancer Council Victoria (Sir Edward Dunlop Fellowship in Cancer Research to C.L.S.); the Victorian Cancer Agency (Clinical Fellowships CRF10-20 and CRF16014 to C.L.S.). This work was made possible through the Australian Cancer Research Foundation, the Victorian State Government Operational Infrastructure Support and Australian Government National Health and Medical Research Council Independent Medical Research Institutes Infrastructure Support Scheme.

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Authors and Affiliations

  1. The Walter and Eliza Hall Institute of Medical Research, Parkville, Victoria, Australia

    Holly E. Barker & Clare L. Scott

  2. Department of Medical Biology, University of Melbourne, Parkville, Victoria, Australia

    Holly E. Barker & Clare L. Scott

  3. Department of Obstetrics and Gynaecology, University of Melbourne, Parkville, Victoria, Australia

    Clare L. Scott

  4. Royal Women’s Hospital, Parkville, Victoria, Australia

    Clare L. Scott

  5. Peter MacCallum Cancer Centre, Parkville, Victoria, Australia

    Clare L. Scott

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  1. Holly E. Barker
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  2. Clare L. Scott
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Correspondence to Clare L. Scott.

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CART-Wheel.org: https://www.cart-wheel.org/

International Rare Cancers Initiative: http://www.irci.info/

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Barker, H.E., Scott, C.L. Preclinical rare cancer research to inform clinical trial design. Nat Rev Cancer 19, 481–482 (2019). https://doi.org/10.1038/s41568-019-0172-2

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