Highlighting the need for reliable clinical trials in glioblastoma

Jacob J. Mandel, Michael Youssef, Ethan Ludmir, Shlomit Yust-Katz, Akash J. Patel, John F. De Groot

Research output: Contribution to journalReview articlepeer-review

10 Scopus citations


Introduction: Several recent phase III studies have attempted to improve the dismal survival seen in glioblastoma patients, with disappointing results despite prior promising phase II data. Areas covered: A literature review of prior phase II and phase III studied in glioblastoma was performed to help identify possible areas of concern. Numerous issues in previous phase II trials for glioblastoma were found that may have contributed to these discouraging outcomes and discordant results. Expert commentary: These concerns include the improper selection of therapeutics warranting investigation in a phase III trial, suboptimal design of phase II studies (often lacking a control arm), absence of molecular data, the use of imaging criteria as a surrogate endpoint, and a lack of pharmacodynamic testing. Hopefully, by recognizing prior phase II trial limitations that contributed to failed phase III trials, we can adapt quickly to improve our ability to accurately discover survival-prolonging treatments for glioblastoma patients.

Original languageEnglish (US)
Pages (from-to)1031-1040
Number of pages10
JournalExpert Review of Anticancer Therapy
Issue number10
StatePublished - Oct 3 2018
Externally publishedYes


  • Clinical trials
  • adaptive trial design
  • glioblastoma
  • phase II
  • phase III

ASJC Scopus subject areas

  • Oncology
  • Pharmacology (medical)


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