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Proof-of-concept rare cancers in drug development: the case for rhabdomyosarcoma

Oncogene volume 33, pages 1877–1889 (2014)Cite this article

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Abstract

Rare diseases typically affect fewer than 200 000 patients annually, yet because thousands of rare diseases exist, the cumulative impact is millions of patients worldwide. Every form of childhood cancer qualifies as a rare disease—including the childhood muscle cancer, rhabdomyosarcoma (RMS). The next few years promise to be an exceptionally good era of opportunity for public–private collaboration for rare and childhood cancers. Not only do certain governmental regulation advantages exist, but these advantages are being made permanent with special incentives for pediatric orphan drug-product development. Coupled with a growing understanding of sarcoma tumor biology, synergy with pharmaceutical muscle disease drug-development programs, and emerging publically available preclinical and clinical tools, the outlook for academic–community–industry partnerships in RMS drug development looks promising.

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Acknowledgements

This work was funded by R01CA133229 (CK), R01CA15923 (DML), R21CA156056 (DML), the Harvard Stem Cell Institute and a gift from the Ethan Jostad Foundation for Childhood Cancer. We thank M Imran Aslam, Lara Davis, Ailin Jiang, Leanna Mah, Marissa Peterson, Victoria Reinke, Teagen Settlemeyer, Suman Malempati and R Lor Randall for their contributions to and discussions regarding this topic. Important input on cell lines was made by Karoly Szuhai, Beat Schäfer, Frederic Barr, Javed Khan, Corinne Linardic and Peter Houghton. We are grateful for the critical comments of Douglas Hawkins, Malcolm Smith, Idriss Bennani-Baiti, M Simone Nsouli, Parkash Gill, Kathryn Gould, Sandra Smith, Steven Townson, Max Wallace, John Mackintosh, Tommy Pham, Jay Scott, Nadim Nsouli, Christopher Winter and Owen Lockerbie.

Author countributions: CK concepted and designed the study. CK and KK developed the methodology of the study. ES, CBT, KK and CK performed the acquisition of data (generation of Tables). CK and KK performed the analysis and provided interpretation of data. KK, DML and CK wrote, reviewed and/or revised the manuscript. CK supervised the study.

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Author notes

  1. E Sokolowski and C B Turina: These authors contributed equally to this work.

Authors and Affiliations

  1. Department of Student Affairs, Oregon State University, Corvallis, OR, USA

    E Sokolowski & C B Turina

  2. Department of Pediatrics, Pediatric Cancer Biology Program, Papé Family Pediatric Research Institute, Oregon Health and Science University, Portland, OR, USA

    C B Turina, K Kikuchi & C Keller

  3. Division of Molecular Pathology and Cancer Center, Massachusetts General Hospital, Boston, MA, USA

    D M Langenau

  4. Harvard Medical School and Harvard Stem Cell Institute, Boston, MA, USA

    D M Langenau

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  1. E Sokolowski
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  2. C B Turina
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  3. K Kikuchi
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  5. C Keller
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Correspondence to C Keller.

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Competing interests

CK has received single-time honoraria for scientific presentations at Novartis, Millennium/Takeda Pharmaceutical and GlaxoSmithKline, and has research joint ventures or sponsored research with Novartis and Johnson & Johnson. CK is also a paid consultant to the NCI/CTEP Pediatric Preclinical Testing Program (PPTP). The remaining authors declare no conflict of interest.

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Sokolowski, E., Turina, C., Kikuchi, K. et al. Proof-of-concept rare cancers in drug development: the case for rhabdomyosarcoma. Oncogene 33, 1877–1889 (2014). https://doi.org/10.1038/onc.2013.129

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  • DOI: https://doi.org/10.1038/onc.2013.129

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