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Creating Meaningful Change, With a Little Help From our Friends

There are only four FDA-approved therapies to treat brain tumors. And in the last 17 years there have been approximately 75 potential new drugs that have failed during the clinical trial process to prove they are safe and effective for patients, while only three that have won FDA approval[1]. That is a 25:1 failure ratio. The number of drugs that have failed to meet the FDA’s safety and efficacy standard is unacceptable for the approximately 700,000 patients living with a brain tumor today, the 69,000 more who will be diagnosed within the next year, and their countless friends, families, and loved ones.

But there is hope. Currently more than 45 new medicines are in development in the United States to fight brain cancer1.

The question now is how can we learn from past experience and ensure that those 45 clinical candidates, as well as the discoveries that progress in the future, are evaluated in the most optimal and sensitive manner? How can the lines on the road to FDA approval be made clearer, and the bumps flattened so that when there is a drug that demonstrates it can help a patient, it gets the green light rather than red.

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Workshop leaders (left to right): Drs. Al Yung (MD Anderson Cancer Center), Terri Armstrong (UT Health Science Center & MD Anderson Cancer Center), and Patrick Wen (Dana-Farber Cancer Center)

One way may be to make the system for which brain tumor treatments are tested, evaluated, and approved easier to navigate. The Jumpstarting Brain Tumor Drug Development Coalition, (that includes National Brain Tumor Society, Accelerate Brain Cancer Cure, Musella Foundation for Research and Information and Society for Neuro-Oncology) is striving to do that through the Clinical Trial End Points Initiative.

To accomplish a major goal of this effort – to make the regulatory process easier for biopharmaceutical companies and medical research institutions doing brain tumor clinical trials – the Coalition organized and hosted two Brain Tumor Clinical Trial Workshops that brought together key stakeholders in brain tumor drug development to create solutions that will allow for better and more standardized imaging and patient-focused measurement of drug effectiveness. Through these workshops, and the actions that follow them, the Initiative aims to build confidence and clarity among companies and institutions that are conduct brain tumor clinical trials. We hope this effort will motivate these groups to pursue brain tumor drug approvals, and also educate FDA about the state of the field so that it is better able to work with groups seeking approval for their potential new treatments.

It would not be possible for the Coalition to create this meaningful change within the clinical trial system, which will ultimately benefit patient care, without the help of the many stakeholders in the brain tumor drug development process: researchers, clinicians, biopharmaceutical company executives, leaders from the National Institutes of Health and the National Cancer Institute, regulators at the FDA, other nonprofits, patients, survivors, and caregivers.

We thank all of those who have helped build this initiative and made the workshops such a success:

  • Steering Committee: Dr. Patrick Wen (Chair –Dana-Farber Cancer Institute), Dr. Terri Armstrong (Vice-Chair – UT Health Science Center and MD Anderson Cancer Center), Dr. Brian Alexander (Dana-Farber Cancer Institute), Dr. Donald Berry (MD Anderson Cancer Center), Dr. Giovanni Parmigiani (Dana-Farber Cancer Institute), Dr. Tito Mendoza (MD Anderson Cancer Center), Dr. Benjamin Ellingson (UCLA), Dr. David Reardon (Dana-Farber Cancer Institute), Dr. Evanthia Galanis (Mayo Clinic), Dr. Howard Fine (NYU Langone Medical Center), Dr. Jaishri Blakeley (Johns Hopkins Hospital), Dr. Jan Buckner (Mayo Clinic), Dr. Jeffrey Wefel (MD Anderson Cancer Center), Dr. Mark Gilbert (MD Anderson Cancer Center), Dr. Martin Klein (VU University Medical Center), Dr. Martin Taphoorn (Medical Center Haaglanden), Dr. Michael Prados (University of California, San Francisco), Dr. Martin van den Bent (Erasmus Medical University), Dr. Susan Chang (University of California, San Francisco), Dr. Timothy Cloughsey (UCLA), Dr. Al Yung (MD Anderson Cancer Center), Dr. Whitney Pope (UCLA), and Dr. Wolfgang Wick (University Heidelberg).
  • Workshop Panelists: Terri Armstrong (UT Health Science Center and MD Anderson Cancer Center), Dr. Jaishri Blakeley (Johns Hopkins Hospital), Dr. Mark Gilbert (MD Anderson Cancer Center), Dr. Patrick Wen (Dana-Farber Cancer Institute), Allison Bishof (Patient Advocate), Dr. Paul Brown (MD Anderson Cancer Center), Dr. Martin Klein (VU University Medical Center), Dr. Joohee Sul (FDA), Dr. Martin Taphoorn (Medical Center Hagglanden), Dr. Christina Theodore-Oklota (Genentech), Dr. Stephen Joel Coons (Critical Path Institute), Dr. John Corboy (University of Colorado Medical School), Dr. Ruthann Giusti (FDA), Dr. Nancy Kline Leidy (Evidera), Dr. Tito Mendoza (MD Anderson Cancer Center), Dr. Elektra Papadopoulos (FDA), Dr. Jeffrey Wefel (MD Anderson Cancer Center), Dr. Martha Donoghue (FDA), Dr. Remi Kaleta (EMD Serono), Dr. Glen Lesser (Wake Forest University), Dr. Lawrence Rubinstein (NCI), Dr. Rajeshwari Sridhara (FDA), Dr. Patricia Keegan (FDA), Dr. Lauren Abrey (Genentech), Dr. Tim Cloughesy (University of California, Los Angeles), Dr. Daniel Krainak (FDA), Dr. Martin van den Bent (Erasmus Medical University), Dr. Martin Bendszus (Heidelberg University Hospital), Dr. Benjamin Ellingson (UCLA), Dr. Christian Graff (FDA), Dr. Whitney Pope (UCLA), Dr. Greg Sorensen (Siemens Healthcare North America), Dr. Karla Ballman (Mayo Clinic), Dr. Peter Bross (FDA), Dr. Jan Buckner (Mayo Clinic), Dr. Susan Chang (University of California, San Francisco), Suzanne Demko (FDA), Dr. Lou Marzella (FDA), Dr. Reardon (Dana-Farber Cancer Institute), Dr. Howard Fine (NYU Langone Medical Center), Dr. Michael Prados (University of California, San Francisco), and Dr. W.K. Alfred Yung (MD Anderson Cancer Center).
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The second Brain Tumor Clinical Trial Endpoints Workshop

  • Workshop Presenters: Dr. Paul Kluetz (FDA), Dr. Martha Donoghue (FDA), Dr. Lee Pai-Scherf (FDA), Dr. Benjamin Ellingson (UCLA), Virginia Kwitkowski (FDA) and Dr. Rajeshwari Sridhara (FDA)
  • Workshop Participants from: GlaxoSmithKline, DelMar Pharmaceuticals, Novartis, Mayo Clinic, Northwest Biotherapeutics, Agenus, Pfizer, Arbor Pharmaceuticals, Genentech, Bristol-Myers Squibb, Celldex Therapeutics, StemCycle, Cortice Biosciences, AstraZeneca, RTI, AbbVie, St. Baldrick’s Foundation, Tocagen, Eli Lilly and Company, Morgan, Lewis & Bockius LLP, Philips, ICON Medical Imaging, Henry Ford Health System, Friends of Cancer Research, ImmunoCellular Therapeutics, American Cancer Society Cancer Action Network, and CERN Foundation.
  • The 1,800+ Brain Tumor Patients and Caregivers who participated in the Brain Tumor Patient & Caregiver Survey.

Again, a BIG thank you to all. We look forward to continuing to work with the field to move this initiative forward.

 

[1] 2014 PhRMA report, “Researching Cancer Medicines: Setbacks and Stepping Stones”

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