Workshop A: Drug Blood-Brain Barrier Penetration
8am-10am EDT | Tuesday, January 18
Glioblastoma is one of the most common, fatal, and complicated to treat adult brain tumors.
Despite considerable attempts to improve our knowledge of GBM, there have been comparatively limited therapeutic developments ever since the early noughties and yet less therapies intended to successfully treat recurring tumors that are resilient to treatment.
While there is a urgent demand to push innovative treatments into the clinic, evolving evidence advocates that significant features exclusive to GBM location and biology, the blood-brain barrier (BBB) and intratumoral molecular heterogeneity, individually, stand as serious unanswered obstacles to successful treatment.
Consequently, classifying BBB-penetrating drugs and employing different and safer techniques to develop drug delivery past the BBB has grown into an industry of rigorous research.
In this workshop, we examine promising approaches to tactically develop drug delivery to GBM and repurpose currently approved and formerly studied drugs using rational combination strategies.
Workshop B: Clinical, Non-Clinical & Surrogate Endpoints
10.30am-12.30pm EDT | Tuesday, January 18
Recent understanding of the molecular pathways that induce glioblastoma development has led to the development of various investigational treatments specifically targeting tumor cells and the tumor microenvironment. Primary endpoints employed in the field typically tend to include OS, progression-free survival (PFS), tumor response, safety and toxicity, or pharmacokinetics/pharmacodynamics (PK/PD).
Given recent progression in understanding of GBM developing secondary & surrogate endpoints will be crucial for evaluating therapies targeting small subpopulations of patients with glioblastoma based on specific molecular aberrations. Appropriate validation and selection of such surrogate endpoints according to a specific target of test treatment is essential to achieve success in clinical trials.
Further to this, the decision to move from early signs of efficacy based on surrogate endpoints to late-stage randomized trials is crucial because there is a high potential of overestimating efficacy by using surrogate endpoints that do not correlate with OS or through selection bias and population drift by comparison with historical controls.
Workshop C: Combination Therapies for Glioblastoma Drug Development
1pm-3pm EDT | Tuesday, January 18
Combination therapy is increasingly becoming the cornerstone of current day antitumor therapy. Glioblastoma multiforme is an aggressive brain tumor with a low median survival post diagnosis and a high rate of disease recurrence. The poor prognosis can be attributed to unique treatment limitations, which include the infiltrative nature of tumor cells, failure of anti-glioma drugs to cross the blood–brain barrier, tumor heterogeneity and the highly metastatic and angiogenic nature of the tumor making cells resistant to chemotherapy.
A combination therapy approach is increasingly being touted as an alternative and promising method for treatment of GBM. Combination approaches rely on complementary mechanisms of antitumor activity and can be combined into a therapeutic regimen. Combination approaches are currently being developed against glioblastoma with new innovative combination drug regimens being tested in preclinical and clinical trials.
Workshop D: Biomarkers & Non-Invasive Predictive Tools
3.30pm-5.30pm EDT | Tuesday, January 18
Gliomas are the most common central nervous system malignancies and present with significant morbidity and mortality. Treatment modalities are currently limited to surgical resection, chemotherapy and radiotherapy. Increases in survival rate over the previous decades are negligible, further pinpointing an unmet clinical need in this field.
There is a continual struggle with the development of effective glioma diagnostics and therapeutics, largely due to a multitude of factors, including the presence of the blood–brain barrier and significant intertumoural and intratumoural heterogeneity. Importantly, there is a lack of reliable biomarkers for glioma, particularly in aiding tumor subtyping and measuring response to therapy. There is a need for biomarkers that would both overcome the complexity of the disease and allow for a minimally invasive means of detection and analysis.
To date, there are no approved biomarkers of glioma for clinical practice. There is an urgent need for biomarkers that (1) aid in diagnosis and patient stratification, (2) identify true disease recurrence, and (3) indicate response to treatment.