Conference Day One

Wednesday 15th March 2023

8:30 am Registration & Welcome Coffee

8:55 am Chair’s Opening Remarks

  • Kirk Tanner CSO and SVP Brain Tumor Investment Fund, National Brain Tumor Society

Reviewing Glioblastoma Clinical Updates in the Past Year: The Successes & Failures

9:00 am Examining the Current Landscape of Glioblastoma Therapies & Strategies to Improve Outcomes

  • Patrick Wen Director - Center For Neuro-Oncology, Dana-Farber Cancer Institute


  • Overview of novel therapies for GBM in development
  • Understanding reasons for past failures
  • Evaluating strategies to improve the identification of more effective therapies

9:30 am Panel Discussion: GBM AGILE Goes Worldwide: Taking a Deep Dive Into How Well It Has Worked So Far & What Promise It Brings With Expansion

  • David Arons Chief Executive Officer, National Brain Tumor Society
  • James Garner Chief Executive Officer, Kazia Therapeutics Limited
  • Robert Murray Chief Business Officer, Global Coalition for Adaptive Research
  • Meredith Buxton Chief Executive Officer, Global Coalition for Adaptive Research


  • Reflecting on more than 3 years of GBM AGILE – lessons learned to date and plans/goals for the next 3+ years
  • Pharma perspectives of participation and stakeholder perspectives
  • Learning from an annotated longitudinal database of screens from over 1300 glioblastoma patients
  • What progress have we already seen with GBM AGILE so far?
  • Conceptually, a ‘master protocol’ requires a standardized trial design across all participating arms, with limited ability to customize for specific agents. To what extent does that force compromise in the study design? Is there a risk we end up with a study that isn’t ideally suited to any of its participating drugs?
  • As a Bayesian adaptive study, participant companies in GBM AGILE are unable to know much about the ongoing performance of the study until the study is complete. What are the implications of this, particularly for smaller biotechs?
  • GBM AGILE sets pre-defined thresholds for graduation within the study and for successful completion at the end of the study. How do we balance those thresholds such that we neither take forward ineffective therapies, nor prematurely discard therapies that may prove beneficial to patients?

10:30 am Session reserved for Kintara Therapeutics

11:00 am Speed Networking Break


This session is the ideal opportunity to introduce yourself to the attendees that

you would like to have more in-depth conversations with. Use this time to get

back to face-to-face interaction with many of the brightest minds working in the

glioblastoma field and establish meaningful business relationships.

An Update On RANO 2.0 & Outlining Novel Imaging Endpoints in Phase II & III Clinical Trials

11:30 am Assessing Response in Gliomas (RANO 2.0)

  • Patrick Wen Director - Center For Neuro-Oncology, Dana-Farber Cancer Institute


  • Overview of the challenges of response assessment in gliomas
  • Current data on optimal response assessment
  • Review of RANO 2.0 for glioblastomas and other gliomas in adults

12:00 pm Delving Into Imaging Endpoint Strategies for Early & Later-stage Glioblastoma Clinical Trials


  • For early phase clinical trials involving a limited number of patients and/or complex tumor types, a tiered approach to recognizing, quantifying, and interpreting radiographic changes is recommended, including the use of digital “flipbooks” and a modified Levin criterion, in order to increase confidence in potential therapeutic changes
  • To increase the likelihood of success in later phase trials, we recommend evaluating the proportion of patients exhibiting a meaningful change in growth rate trajectory and progression-free survival (PFS), including calculation of pre-treatment growth rates and use of a “hyperacute” baseline within days of starting experimental therapy
  • Historic data suggests objective response rate (ORR) >25%, in combination with a sufficient duration of response (DOR), leads to a meaningful increase in overall survival and a high likelihood of success in late-stage survival studies

12:30 pm ONC201 in Recurrent H3 K27M-mutant Diffuse Midline Glioma


  • Overview of ONC201 discovery and early clinical development
  • Review of efficacy signal refinement for ONC201 toward H3 K27M-mutant glioma
  • Summary of ONC201 efficacy it recurrent H3 K27M-mutant diffuse midline glioma

Utilizing Ultrasounds: A Compelling Technique to Incorporate Into Glioblastoma Treatments

12:45 pm Lunch Break


Take this chance to meet the expert speakers, connect with your peers and explore our exhibition booths.

1:45 pm Understanding the World of MR-Guided Focussed Ultrasounds- Translating This Approach Into Glioblastomas

  • John de Groot Division Chief, Neuro- Oncology, UCSF Department of Neurosurgery


  • Unpacking the biological principles of Magnetic Resonance-guided Focused Ultrasounds (MRgFUS) in treating tumors
  • Exploring how MRgFUS has been successful in treating other pathologies like uterine cancers
  • Evaluating how feasible and effective this would be as a treatment for glioblastomas

2:15 pm Session Reserved for Novocure

2:45 pm Sonodynamic Therapy: A Promising Approach to Targeting the Treatment of High-Grade Glioma


  • Discussing the mechanism of action of sonodynamic therapy
  • Analyzing the use of sonodynamic in high grade glioma
  • Looking at the next steps for the use of sonodynamic therapy in high grade glioma

3:15 pm Interactive Group Discussion: Using Ultrasounds to Overcome the Blood Brain Barrier: Improving Drug Delivery to the Site of GBM Tumors & Enhancing Other Treatments


  • How useful are ultrasounds for blood-brain-barrier penetrance? Are the better alternatives?
  • How well do ultrasounds work to enhance the effect of temozolomide
  • What are the potential combination partners with FUS

3:45 pm Chair’s Closing Remarks & End of Day One

3:50 pm Poster Networking Session


After the formal presentations have finished, the scientific Poster Session is an informal part of the conference agenda, allowing you to connect with your peers in a relaxed atmosphere.

Continue to forge new and cement existing relationships while learning about the latest developments and breakthroughs in this field.