FAST Congress
Archived Content

Clinical Biomarkers


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Friday, September 21

7:30-8:15 am Morning Coffee or Sponsored Breakfast Presentation (Opportunity Available)

Contact Ilana Quigley at or 781-972-5457

Biomarker-Based Clinical Trial Design 

8:30-8:35 Chairperson’s Opening Remarks

8:35-9:00 Accelerating and Personalizing Clinical Trials with Biomarkers and Adaptive Design, the I-SPY 2 Example

Sonia Pearson-White, Ph.D., Scientific Program Manager, Oncology, The Biomarkers Consortium, Foundation for the National Institutes of Health

I-SPY 2 is a unique clinical trial managed as a public/private partnership by the Foundation for the NIH (FNIH) Biomarkers Consortium. I-SPY 2 employs an innovative adaptive trial design in high risk breast cancers in the neoadjuvant setting, and will advance the understanding of which drugs work best with tumor types with different biomarker profiles, and the drive toward personalized medicine. The trial includes 20 major cancer research centers across the United States, and is part of an accelerated approval strategy under development at the FDA.

9:00-9:25 Predictive Markers and Innovative Oncology Clinical Trials in Early Development: Opportunities and Challenges

James Song, Ph.D., Senior Statistical Scientist, Global Biostatistics, Genentech

There has been a paradigm shift in oncology research. Molecules are now targeted therapies with wider therapeutic index and derived from molecular biology discoveries. Furthermore, integration of molecular diagnostics with targeted therapies for integrated cancer care will be the key in the future. Thus the importance of identifying predictive markers early has been well recognized. In this presentation we will review opportunities and some of the key challenges in identifying and evaluating predictive markers in Phase I and Phase II oncology trials. Innovative designs, in particular adaptive designs, could play a significant role in such development programs. We will review a few examples of such designs from oncology trials.

9:25-9:50 Clinical Validation of Predictive Biomarkers and Next-Generation Personalized Medicine Treatment Strategies Incorporating Genetic Dynamics

Robert A. Beckman, M.D., External Faculty, Center for Evolution and Cancer, Helen Diller Family Cancer Center, University of California at San Francisco; Executive Director, Clinical Development Oncology, Daiichi Sankyo Pharma Development

The future of oncology drug development lies in personalized therapy using predictive biomarkers. However, examples of the failure of predictive biomarkers also exist. In these cases the use of biomarkers increased the costs, complexity and duration of clinical trials, and narrowed the treated population unnecessarily. Here, we present methods to adaptively integrate predictive biomarkers into clinical programs in a data-driven manner, wherein these biomarkers are emphasized in exact proportion to the evidence supporting their clinical predictive value. Next-generation personalized treatment strategies, which emphasize tumor heterogeneity, evolutionary dynamics and possible future tumor states, will also be presented.

9:50-10:15 Clinical Trial Designs for Testing Biomarker-Based Personalized Therapies

Mei-Chiung Shih, Ph.D., Senior Biostatistician, VA Palo Alto Cooperative Studies Program Coordinating Center; Assistant Professor, Health Research and Policy, Stanford University

Advances in molecular therapeutics in the past decade have opened up new possibilities for treating cancer patients with personalized therapies, using biomarkers to determine which treatments are most likely to benefit them, but there are difficulties and unresolved issues in the development and validation of biomarker-based personalized therapies. We use generalized likelihood ratio tests of the intersection null and enriched strategy null hypotheses to derive a novel clinical trial design for the problem of advancing promising biomarker-guided strategies toward eventual validation. We also investigate the usefulness of adaptive randomization and futility stopping proposed in the recent literature.

10:15-11:15 Coffee Break in the Exhibit Hall with Poster Viewing

Translational Biomarkers 

11:15-11:20 Chairperson’s Opening Remarks

11:20-11:45 Translation of Emerging Biomarkers from Pre-Clinical Species to Human Populations

Jiri Aubrecht, Pharm.D., Ph.D., Senior Director and Safety Biomarker Group Lead, Drug Safety Research & Development, Pfizer

Biomarkers of drug safety provide essential tools for refining of therapeutic index in drug development. Despite the success of routine markers, mainly biochemical parameters used in pre-clinical and clinical settings, a new cohort of safety biomarkers with better sensitivity and specificity is being considered. Since drug development relies on pre-clinical evaluation of lead compounds, the evaluation of translation across animal species including to humans is essential. In this presentation, case studies documenting recent advances in cross species translation for biomarkers of hepatotoxicity and nephrotoxicity will be provided.

11:45-12:10 pm New Safety Biomarkers on the Horizon: The IMI SAFE-T Consortium

Michael Merz, M.D., Director, Preclinical Safety, Translational Sciences, Novartis Institutes for BioMedical Research

The EU’s IMI SAFE-T consortium is a public/private partnership of 25 organizations from the pharmaceutical industry, small- to medium-sized enterprises, academic institutions and clinical units of excellence with representatives from health authorities as external observers and advisors. The key objective of the five-year project is clinical qualification of biomarkers for drug-induced kidney, liver and vascular injury in translational studies and obtaining regulatory acceptance in translational and clinical contexts. The talk will give an overview on the consortium, the qualification program, and the biomarker candidates. Preliminary data from the exploratory qualification phase will be presented for some selected biomarkers.

12:10-12:35 Progression of Biomarkers for Alzheimer’s Disease

Johan Luthman, D.D.S., Ph.D., Senior Program Leader, Neuroscience and Ophthalmology Research & Development, Merck & Co., Inc.

The development of biomarker-based readouts for neurological diseases has traditionally been limited by the inaccessibility of the brain and lack of insight into molecular pathophysiology of brain diseases. Rapid advances in neurosciences, coupled with breakthroughs in clinical application of novel bio-analytical approaches, functional measures and imaging technologies, are now opening up new opportunities to follow brain function and dysfunction; a good example of this is translational and clinical research on Alzheimer's disease. By measuring pathophysiology-associated factors, such as Aß peptide and tau protein in cerebrospinal fluid, and by imaging brain function with fluorodeoxyglucose Positron Emission Tomography (PET), functional MRI, pathology with Amyloid PET, or volumetric MRI, we can now detect very early stages of AD pathology, in prodomal and even pre-symptomatic subjects, and follow the disease progression to its end stages. Furthermore, by monitoring AD pathophysiology-associated biomarkers, the pharmacodynamic actions of putative disease-modifying therapeutic approaches can be measured in healthy volunteers and in AD patients. Ongoing clinical qualification of those measures is paving the way of using AD biomarkers as supportive evidence of treatment effect.

12:35 Close of Conference

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