The Food and Drug Administration and the National Brain Tumor Society, joined by a strong coalition of patient advocacy and research organizations, collaborated to host a workshop on March 22, 2019, at the FDA campus, with the intent to provide an open forum to discuss key challenges including clinical trial accrual, study design, and endpoint selection for brain metastases research and treatment discovery.
Metastatic brain cancer can result from several types of primary cancers including but not limited to breast, kidney, lung, and skin cancer (melanoma). Brain and central nervous system (CNS) metastases represent late-stage cancer and are always a serious, if not a life-threatening diagnosis.
More than 80% of cancers have been associated with the ability to metastasize to the brain.
(Journal of the National Cancer Institute – JNCI) These include:
Studies cite that anywhere from 6-28% of cancer patients develop brain metastases. There are too few treatments for cancer patients with brain metastases and also too few clinical trials aimed at developing new treatments. Increased efforts to develop therapeutic products are needed to address this unmet medical need and ultimately help patients. Barriers to product development for CNS metastases include lack of clinical trials specific for this population, exclusion of patients with brain metastases from clinical trials, and lack of standardized response assessments.
In light of all this, the Food and Drug Administration and the National Brain Tumor Society, joined by a strong coalition of patient advocacy and research organizations, collaborated to host a workshop on March 22, 2019, at the FDA campus, with the intent to provide an open forum to discuss key challenges including clinical trial accrual, study design, and endpoint selection for brain metastases research and treatment discovery.
This workshop has generated results, criteria, documentation, and awareness for the challenges of brain tumor mets patients. SEE the SIDEBAR for materials and resources related to brain and CNS metastases. Also, view the pre-workshop video segments featuring key researchers, medical professionals, and clinicians that highlight key sessions of the workshop.
**The coalition includes ABC2, ABTA, Friends of Cancer Research, KCCure, LUNGevity, MBCA, MRA, RANO and SNO
David Arons, JD National Brain Tumor Society | Joohee Sul, MD, Co-Chair US Food and Drug Administration |
Nancy Lin, MD Dana Farber Cancer Institute | Patrick Wen, MD, Co-Chair Dana Farber Cancer Institute |
Wendy Selig, MSJ WSCollaborative | Al Yung, MD MD Anderson Cancer Center |
Jeff Allen, PhD Friends of Cancer Research | Debbie Robbins American Brain Tumor Alliance |
Upal Basu Roy, PhD LUNGevity Foundation | David Sandak Accelerate Brain Cancer Cure |
Dena Battle Kidney Cancer Research Alliance | Dianne Spillman US Food and Drug Administration |
Laurie Campbell Metastatic Breast Cancer Alliance | Mark Stewart, PhD Friends of Cancer Research |
Ralph DeVitto American Brain Tumor Alliance | Kirk Tanner, PhD National Brain Tumor Society |
Andrea Ferris, MBA LUNGevity Foundation | Joan Todd US Food and Drug Administration |
Chas Haynes, JD Society for Neuro-Oncology | Max Wallace, JD Accelerate Brain Cancer Cure |
Michael Kaplan Melanoma Research Alliance | Nicole Willmarth, PhD American Brain Tumor Alliance |
Kristen Mueller, PhD Melanoma Research Alliance |
Lauren Abrey, MD Novartis | Kim Margolin, MD City of Hope |
Laleh Amiri-Kordestani, MD US Food and Drug Administration | Shanthi Marur, MD US Food and Drug Administration |
Carey Anders, MD Duke University | Minesh Mehta, MD Miami Cancer Institute |
Manmeet Ahluwalia, MD Cleveland Clinic | Pallavi Mishra-Kalyani, PhD US Food and Drug Administration |
Priscilla Brastianos, MD Massachusetts General Hospital | Tatiana Prowell, MD US Food and Drug Administration |
Paul Brown, MD Mayo Clinic | Derrick Queen Patient |
Ross Camidge, MD University of Colorado, Denver | Greg Riely, MD Memorial Sloan Kettering Cancer Center |
Mike Davies, MD MD Anderson Cancer Center | Corey Ritchings, PhD Bristol-Myers Squibb |
Ben Ellingson, PhD University of California, Los Angeles | Donna Roscoe, PhD US Food and Drug Administration |
Eva Galanis, MD Mayo Clinic | Barbara Scepura, NP US Food and Drug Administration |
Maitreyee Hazarika, MD US Food and Drug Administration | Jeffrey Wefel, PhD MD Anderson Cancer Center |
Eric Jonasch, MD MD Anderson Cancer Center | Chana Weinstock, MD US Food and Drug Administration |
Emilie LeRhun, MD University Hospital Lille, France | Michael Weller, MD University of Zurich |
Minetta Liu, MD Mayo Clinic | Peggy Zuckerman Patient |
March 22, 2019
8:00am to 4:00pm
FDA White Oak Campus Building 31, Great Room
8:00 – 8:30AM
Registration
8:30 – 8:35AM
Opening Remarks:
David Arons, JD, National Brain Tumor Society
Richard Pazdur, MD, US Food and Drug Administration
Wendy Selig, MSJ, WSCollaborative
8:35 – 8:45AM
Presenting the Challenge
Joohee Sul, MD, Workshop Co-Chair, US Food and Drug Administration
Patrick Wen, MD, Workshop Co- Chair, Dana Farber Cancer Institute
8:45 – 9:45AM
Session I: Defining the Problem of CNS Metastases
Laleh Amiri-Kordestani, MD, Co-Chair, US Food and Drug Administration
Greg Riely, MD, Co-Chair, Memorial Sloan Kettering Cancer Center
Discussant Presentation:
Clinical Management of CNS Metastasis, 2019: Current Strategies, Investigations, and Key Challenges
Mike Davies, MD, MD Anderson Cancer Center
Panel:
Laleh Amiri-Kordestani, MD, & Greg Riely, MD, Moderators
Manmeet Ahluwalia, MD, Cleveland Clinic
Mike Davies, MD, MD Anderson Cancer Center
Victoria Ebiana, MD, Merck
Derrick Queen, Patient
Luke Walker, MD, Seattle Genetics
9:45 – 11:00AM
Session II: Key Issues for Clinical Development for Brain Mets
Nancy Lin, Co-Chair, Dana Farber Cancer Institute
Chana Weinstock, Co-Chair, US Food and Drug Administration
Discussant Presentations:
Q&A
Session Recap: Chana Weinstock, US Food and Drug Administration
11:00 – 11:15AM
Break
11:15 – 2:30PM
Session III: Clinical Benefit in Patients with Brain Mets
Carey Anders, Co-Chair, Duke University
Tatiana Prowell, Co-Chair, US Food and Drug Administration
11:15-12:30PM
Discussant Presentations:
Designing an Endpoints Framework for CNS Metastases
Carey Anders, Moderator
Panel: Terri Armstrong, National Cancer Institute
Shelly Engfer-Triebenbach, Patient
Chitkala Kalidas, Bayer Pharmaceuticals
Ben Levy, Sibley Memorial Hospital
Jeff Wefel, MD Anderson Cancer Center
Arvin Yang, Bristol-Myers Squibb
Q&A
Panel Recap: Carey Anders, Duke University
12:30 – 1:00PM
Lunch
1:00 – 2:30PM
Discussant Presentations:
Rethinking Trial Designs for Stimulating Product Development for CNS Metastases
Tatiana Prowell, Moderator
Panel: Michael Atkins, Georgetown University
Kim Blackwell, Eli Lilly and Company
Vinai Gondi, Northwestern Medicine
Pallavi Mishra-Kalyani, US Food and Drug Administration
Hussein Tawbi, MD Anderson Cancer Center
Lynda Weatherby, Patient
Q&A
Panel Recap: Tatiana Prowell, US Food and Drug Administration
2:30 – 3:30PM
Session IV: Therapy Development: Challenges & Opportunities
Joohee Sul, Co-Chair, US Food and Drug Administration
Patrick Wen, Co-Chair, Dana Farber Cancer Institute
Defining Strategies to Advance Product Development
Panel: Lauren Abrey, Novartis
Caroline Chung, MD Anderson Cancer Center
Nancy Lin, Dana Farber Cancer Institute
Kim Margolin, City of Hope
Tatiana Prowell, US Food and Drug Administration
Greg Riely, Memorial Sloan Kettering Cancer Center
Peggy Zuckerman, Patient
Q&A
3:30 – 3:40PM
Overview of the American Brain Tumor Association’s Metastatic Brain Tumor Initiative
Ralph DeVitto, American Brain Tumor Association
Nicole Willmarth, American Brain Tumor Association
3:40 – 4:00PM
Summary & Next Steps
David Arons, National Brain Tumor Society
Joohee Sul, US Food and Drug Administration
Patrick Wen, Dana Farber Cancer Institute
4:00PM
Adjourn