8:00 am
Coffee & Registration

8:30 am Chair’s Opening Remarks

  • Kirk Tanner Chief Scientific Officer, National Brain Tumor Society (NBTS)

8:40 am Assessing the Current State of Immunotherapy for GBM

  • Michael Lim Director of the Brain Tumor Immunotherapy Program, Johns Hopkins University School of Medicine


  • Elucidating the mechanisms of immune evasion by primary brain tumors
  • Developing translational studies to develop the next generation of GBM clinical trials
  • Pioneering translation of novel therapies against glioma
  •  Insights into why immunotherapy has failed in various clinical trials to date

Realizing Immunotherapy’s Potential to Successfully Treat Recurrent Glioblastoma Patients

9:10 am Coaxing T-cells into Action: Leveraging a Rational & Logical Way to Develop Immunotherapies


  • How recent findings suggest that the neoadjuvant administration of PD-1 blockade enhances both the local and systemic antitumor immune response
  •  Combining checkpoint inhibitors that target molecules called PD-1/PD-L1 like pembrolizumab with CTLA-4 blockades
  • How this data can help our understanding of the mechanisms by which some patients generate

9:40 am Clinical Case Study: Exploring Gene & Viral based Immunotherapies for Glioblastoma


  • Developing novel genetic therapies and engineering of viruses to kill tumor cells without affecting normal brain cells
  • Combining these experimental therapies with novel pharmacological and immunotherapeutic approaches for brain cancer
  • Phase I clinical trial: Preclinical evaluation of oncolytic viruses for glioma therapy and the development of a recombinant Herpes virus

10:10 am Clinical Case Study: Using a Multi-pronged Approach to Treating Recurrent GBM: Overcoming the Tumor and its Microenvironment


  • Discuss barriers that make GBM impervious to cancer immunotherapies
  • Discuss the role of the IL4 receptor (IL4R) in GBM and the tumor microenvironment and its relevance as an immunotherapeutic target
  • Present evidence from a Phase 2b clinical trial: results to date show improved survival in rGBM patients, especially those who over-express IL4R


10:40 am
Morning Refreshments

11:10 am Clinical Case Study: Immmuno-Gene Therapy – Selectively Infecting Cancer Cells to Stimulate Robust & Durable Anti-Cancer Immune Responses


  • Exploration of ongoing clinical trials to date, both phase one and phase three
  • Details on the approaches taken and the RRV platform and lead product candidates, Toca 511 and Toca FC
  •  Reviewing the challenges and rewards for monitoring immune responses in the clinic
  • Discussing manufacturing considerations

11:40 am DCVax Technology: Leveraging Activated Dendritic Cells in Clinical Development for GBM


  • Exploring the history, biology and development of the platform technology DCVax
  •  Outlining the key aspects of the DCVax technology that contribute to the positive clinical results
  •  Designed to mobilize the entire immune system
  • Designed to target not just one but the full set of biomarkers on the patient’s tumor
  • DCVax is personalized, and targets the particular biomarkers expressed on that patient’s tumor
  • Pinpointing highs and lows of clinical development to date

12:10 pm Active Clinical Trial: Developing Personalized Cancer Vaccines for Newly Diagnosed Glioblastoma

  • Adilia Hormigo Director Neuro-Oncology Division, Icahn School of Medicine, Mount Sinai & The Tisch Cancer Institute


  • Introduction to the trial: Precision medicine in the form of a vaccine, a mutationderived tumor antigen vaccine (MTA- vaccine) in combination with standard care treatment of glioblastoma and TTFields
  •  Preparation of MTA-based personalized vaccine in the laboratory with peptides based on each patient’s own tumor mutations and immune recognition to ensure a robust response from the immune system
  •  Considerations for compliance, manufacturing quality control and costs

12:40 pm
Networking Lunch

Driving More Effective Clinical Trial Design

1:40 pm GBM Agile: Identifying More Effective Therapies for the Right Patients Faster

  • Brian Alexander Associate Professor of Radiation Oncology, Harvard Medical School & Dana-Farber/ Brigham and Women’s Cancer Center, GBM Agile Global Coalition for Adaptive Research


  •  Transforming clinical trials by designing an adaptive platform trial: Adaptive Global Innovative Learning Environment for Glioblastoma (GBM AGILE)
  • How adaptive platform trials maintain several “treatment arms” to simultaneously and dynamically study the effects of multiple unique drugs for one given disease
  • How GBM AGILE is creating a rich biomarker repository and paving the way for Bayesian analysis of multiple GBM drugs
  • GCAR aims to run similar adaptive Bayesian trials for other rare or deadly diseases: trailblazing adaptive platform trials

2:10 pm Discovery & Preclinical Development of a PI3K Inhibitor for the Treatment of Glioblastoma


  • Evaluating how the PI3K pathway is altered in a majority of GBM patients
  • Analyzing how the PI3K inhibitor GDC-0084 was optimized for brain penetration and specifically developed as a potential treatment of GBM
  • Assessing the evidence of brain penetration, target modulation and efficacy in preclinical models of GBM led to its evaluation in clinical trials

2:40 pm IL-12 as a Drug for Recurrent Glioblastoma: Genetic Engineering to Control this Master Regulator of the Immune System


  • Interleukin 12 (IL-12) is a powerful immune-stimulatory cytokine with known anti-tumor effects
  • The expression of IL-12 can be controlled by genetically engineering adenovirus to conditionally express this cytokine from within recurrent glioblastoma (rGBM) using a switch technology governed by an oral activator
  • Approximately 50 patients with rGBM have received “Controlled IL-12” with all producing IL-12 and serial biopsies demonstrating new intra-tumoral infiltration and activation of immune response
  • Phase 1 trials have demonstrated an apparent survival advantage for “Controlled IL-12” as monotherapy with subset of patients surviving more than 17 months on average after recurrence of GBM
  • Ongoing phase 1 and 2 trials are expanding the numbers of patients receiving Controlled IL-12 as monotherapy and exploring the combination of this cytokine with PD-1 inhibitors

3:10 pm
Afternoon Refreshments

3:40 pm Realizing Potential: Taking GDC-0084 Forward in Glioblastoma

  • James Garner Chief Executive Officer & Managing Director, Kazia Therapeutics Limited


  • Identifying a target population: newly-diagnosed versus recurrent patients
  • Addressing the challenges and opportunities in designing a definitive clinical proof-of-concept for GBM
  • Defining a path-to-market for GDC-0084
  • Establishing the commercial prospects for a new drug in GBM

4:10 pm Clinical Case Study: Driving a New Therapeutic Strategy to Accelerate to the Clinic

  • Sharon Shacham President & Chief Scientific Officer, Co-founder, Karyopharm


  • Exportin 1 (XPO1) levels are elevated in most glioblastomas (GBM) versus glial cells and tumors that express higher XPO1 levels have poor prognosis with shorter survival; similar observations apply to other tumors
  •  Selinexor is first-in-class, oral, Selective Inhibitor of Nuclear Export (SINE) compound that forces the nuclear retention and activation of most tumor suppressor proteins (e.g., p53, p21, p27, pRB) and reduces levels of certain oncoproteins (e.g., c-myc, cyclin D1)
  • Selinexor was recently approved by the FDA to treat refractory multiple myeloma (XPOVIO™) and has good brain penetration with significant activity in xenograft and patient-derived orthotopic GBM models
  •  Selinexor has shown single agent activity in phase II (“KING” Study) in patients with relapsed or refractory GBM following ≥1 prior regimen with an ORR of ~10% and 6-cycle PFS of 30%, or 6-months PFS of 19%; dosing is once weekly oral with low grade gastrointestinal effects and mild thrombocytopenia

4:40 pm Clinical Case Study: Inovio’s Innovative Combination Trial


  • Optimizing T cell-generating therapies in combination with PD-1/PD-L1 inhibitors for GBM
  • Overview of the INO-5401 clinical trial and challenges to overcome in translation to phase 2 trials

5:10 pm
Chair’s Closing Remarks

5:20 pm
End of 1st Glioblastoma Drug Development Summit