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Research Article

Antitumor Activity of Rapamycin in a Phase I Trial for Patients with Recurrent PTEN-Deficient Glioblastoma

  • Tim F Cloughesy equal contributor,

    equal contributor Contributed equally to this work with: Tim F Cloughesy, Koji Yoshimoto, Phioanh Nghiemphu, Paul S Mischel, Ingo K Mellinghoff, Charles L Sawyers

    Affiliation: Department of Neurology, Jonsson Comprehensive Cancer Center, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, California, United States of America

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  • Koji Yoshimoto equal contributor,

    equal contributor Contributed equally to this work with: Tim F Cloughesy, Koji Yoshimoto, Phioanh Nghiemphu, Paul S Mischel, Ingo K Mellinghoff, Charles L Sawyers

    Affiliation: Department of Pathology and Laboratory Medicine, Jonsson Comprehensive Cancer Center, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, California, United States of America

    ¤ Current address: Department of Neurosurgery, Graduate School of Medical Sciences, Kyushu University, Fukuoka, Japan.

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  • Phioanh Nghiemphu equal contributor,

    equal contributor Contributed equally to this work with: Tim F Cloughesy, Koji Yoshimoto, Phioanh Nghiemphu, Paul S Mischel, Ingo K Mellinghoff, Charles L Sawyers

    Affiliation: Department of Neurology, Jonsson Comprehensive Cancer Center, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, California, United States of America

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  • Kevin Brown,

    Affiliation: Translational Genomics Research Institute, Phoenix, Arizona, United States of America

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  • Julie Dang,

    Affiliation: Department of Pathology and Laboratory Medicine, Jonsson Comprehensive Cancer Center, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, California, United States of America

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  • Shaojun Zhu,

    Affiliation: Department of Pathology and Laboratory Medicine, Jonsson Comprehensive Cancer Center, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, California, United States of America

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  • Teli Hsueh,

    Affiliation: Department of Molecular and Medical Pharmacology, Jonsson Comprehensive Cancer Center, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, California, United States of America

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  • Yinan Chen,

    Affiliation: Department of Molecular and Medical Pharmacology, Jonsson Comprehensive Cancer Center, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, California, United States of America

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  • Wei Wang,

    Affiliation: Taylor Technology, Princeton, New Jersey, United States of America

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  • David Youngkin,

    Affiliation: Translational Genomics Research Institute, Phoenix, Arizona, United States of America

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  • Linda Liau,

    Affiliation: Department of Neurosurgery, Jonsson Comprehensive Cancer Center, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, California, United States of America

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  • Neil Martin,

    Affiliation: Department of Neurosurgery, Jonsson Comprehensive Cancer Center, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, California, United States of America

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  • Don Becker,

    Affiliation: Department of Neurosurgery, Jonsson Comprehensive Cancer Center, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, California, United States of America

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  • Marvin Bergsneider,

    Affiliation: Department of Neurosurgery, Jonsson Comprehensive Cancer Center, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, California, United States of America

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  • Albert Lai,

    Affiliation: Department of Neurology, Jonsson Comprehensive Cancer Center, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, California, United States of America

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  • Richard Green,

    Affiliation: Department of Neurology, Kaiser Permanente; Los Angeles, California, United States of America

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  • Tom Oglesby,

    Affiliation: Taylor Technology, Princeton, New Jersey, United States of America

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  • Michael Koleto,

    Affiliation: Taylor Technology, Princeton, New Jersey, United States of America

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  • Jeff Trent,

    Affiliation: Translational Genomics Research Institute, Phoenix, Arizona, United States of America

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  • Steve Horvath,

    Affiliation: Department of Biostatistics and Human Genetics, Jonsson Comprehensive Cancer Center, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, California, United States of America

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  • Paul S Mischel equal contributor,

    equal contributor Contributed equally to this work with: Tim F Cloughesy, Koji Yoshimoto, Phioanh Nghiemphu, Paul S Mischel, Ingo K Mellinghoff, Charles L Sawyers

    Affiliations: Department of Pathology and Laboratory Medicine, Jonsson Comprehensive Cancer Center, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, California, United States of America, Department of Molecular and Medical Pharmacology, Jonsson Comprehensive Cancer Center, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, California, United States of America

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  • Ingo K Mellinghoff equal contributor,

    equal contributor Contributed equally to this work with: Tim F Cloughesy, Koji Yoshimoto, Phioanh Nghiemphu, Paul S Mischel, Ingo K Mellinghoff, Charles L Sawyers

    Affiliation: Department of Molecular and Medical Pharmacology, Jonsson Comprehensive Cancer Center, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, California, United States of America

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  • Charles L Sawyers equal contributor mail

    equal contributor Contributed equally to this work with: Tim F Cloughesy, Koji Yoshimoto, Phioanh Nghiemphu, Paul S Mischel, Ingo K Mellinghoff, Charles L Sawyers

    To whom correspondence should be addressed. E-mail: sawyersc@mskcc.org

    Affiliation: Department of Human Oncology and Pathogenesis Program, Memorial Sloan Kettering Cancer Center, New York, New York, United States of America

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  • Published: January 22, 2008
  • DOI: 10.1371/journal.pmed.0050008

About the Authors

Tim F Cloughesy, Phioanh Nghiemphu, Albert Lai
Department of Neurology, Jonsson Comprehensive Cancer Center, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, California, United States of America
Koji Yoshimoto, Julie Dang, Shaojun Zhu, Paul S Mischel
Department of Pathology and Laboratory Medicine, Jonsson Comprehensive Cancer Center, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, California, United States of America
Kevin Brown, David Youngkin, Jeff Trent
Translational Genomics Research Institute, Phoenix, Arizona, United States of America
Teli Hsueh, Yinan Chen, Paul S Mischel, Ingo K Mellinghoff
Department of Molecular and Medical Pharmacology, Jonsson Comprehensive Cancer Center, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, California, United States of America
Wei Wang, Tom Oglesby, Michael Koleto
Taylor Technology, Princeton, New Jersey, United States of America
Linda Liau, Neil Martin, Don Becker, Marvin Bergsneider
Department of Neurosurgery, Jonsson Comprehensive Cancer Center, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, California, United States of America
Richard Green
Department of Neurology, Kaiser Permanente; Los Angeles, California, United States of America
Steve Horvath
Department of Biostatistics and Human Genetics, Jonsson Comprehensive Cancer Center, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, California, United States of America
Charles L Sawyers
Department of Human Oncology and Pathogenesis Program, Memorial Sloan Kettering Cancer Center, New York, New York, United States of America

Corresponding Author

Email: sawyersc@mskcc.org

Competing Interests

The authors have declared that no competing interests exist.

Author Contributions

TFC, PN, JT, PSM, IKM and CLS designed the study. TFC, KY, PN, KB, JD, SZ, TH, YC, WW, DY, LL, NM, RG, TO, MK, and CLS collected the data. TFC, KY, PN, KB, TH, DY, SH, PSM, IKM and CLS analyzed the data. TFC, PN, LL, NM, DB, AL, RG, and CLS enrolled patients. TFC, KY, PN, KB, DY, PSM, IKM, and CLS contributed to writing the paper.