Leaders in Pharmaceutical Business Intelligence (LPBI) Group

Reporter: Aviva Lev-Ari, PhD, RN

 

Multi-Scale Study of Cancer

Friday, February 15, 2013: 8:00 AM-9:30 AM

Room 207 (Hynes Convention Center)

Despite the identification of hundreds more potential targets by genome sequencing, the pharmaceutical industry has been faced with a decline in the production of new successful drugs. Mathematical models provide a framework in which to interpret the vast amount of experimental data generated to suggest experiments necessary to test new biological theories leading to development of efficient drugs. Genetic mutations occurring at a subcellular level manifest themselves in a complex, multi-scale cancer process as functional changes at the cellular and tissue scales. Integration of mathematical modeling approaches with statistical analysis of the experimental and clinical data leads to breakthroughs in understanding of the cancer growth, which is translated into novel patient-specific therapies and development of new drugs. When applied to experimental data, statistical techniques can reveal whether a particular intervention produces a significant response or whether a correlation exists between observable phenomena. Establishing why such correlations arise requires verification of a large number of hypotheses using modeling. For example, models offer greater scope for understanding normal and diseased colorectal crypts, for testing and identifying new therapeutic targets in colon cancer, and for predicting their impacts. Speakers have been selected for their contribution to the field and their ability to communicate to audiences with diverse backgrounds and scientific interests.

Organizer:

Mark Alber, University of Notre Dame

Co-Organizer:

Jill P. Mesirov, Broad Institute of Massachusetts Institute of Technology and Harvard University

Moderator:

Mark Alber, University of Notre Dame

Discussants:

Mark Alber, University of Notre Dame

and Jeremy Gunawardena, Harvard Medical School

Speakers:

 

Philip Maini, University of Oxford

Hybrid Approach to Multi-Scale Modeling of Cancer

Martin Nowak, Harvard University

Dynamics of Targeted Cancer Therapy

Kathleen Wilkie, Tufts University School of Medicine

Modeling in Cancer Immunology

 

The Benefits of Randomized Experiments for Science and Society

Friday, February 15, 2013: 1:00 PM-2:30 PM

Room 206 (Hynes Convention Center)

Random experiments have allowed researchers to make advances in physical science, social science, industry, and health that have led to better policy and benefits to society. Randomized experiments use random assignment of study units to allow for learning when variation exists among the units and to estimate causal effects free of confounding by the selection of people into treatment conditions in social experiments. As a result, researchers now routinely test medical, educational, behavioral, and policy interventions with randomized experiments to identify effective interventions. This session provides examples of such uses and benefits of randomized experiments. The first paper describes applications of randomized experimentation to the study of politics, how experimentation has evolved in political science, and examples of its benefits to policy. The second paper describes examples of randomized evaluations of public health and education initiatives in developing countries. These experiments found that low-cost interventions could yield substantial health benefits, but paying teachers for performance had more mixed results. In behavioral health, treatment often follows a course of care, and determining the optimal course for a patient is challenging. The third paper describes how recent statistical advances have led to new experimental designs that allow the researcher to test for courses of care that yield optimal outcomes. It also gives an example of the use of such designs.

Organizer:

Daniel McCaffrey, RAND Corp.

Speakers:

 

Arthur Lupia, University of Michigan

Experimenting with Politics

 

 

Michael Kremer, Harvard University

Experimenting with Public Health and Education in the Developing World

 

 

Susan Murphy, University of Michigan

Experimenting To Improve Clinical Practice

 

 

Clinical Trial and Error: Beauty and the Beast

Friday, February 15, 2013: 3:00 PM-4:30 PM

Room 309 (Hynes Convention Center)

Independent clinical trials are fundamental to improving the results, safety, and cost-effectiveness of health care. Because of costs, the scope of these trials is often limited to new products. Important clinical interventions, surgery, physiotherapy, and rehabilitation are of less interest to commercial sponsors seeking primarily drug and device authorization. Whereas investigator-driven studies now find support in the United States and European Union, they cannot match the resources available for commercial clinical trials. Public and charitable funding typically backs health care–orientated trials independently of manufacturers, but is often much smaller in size. Results matter. When we do get such new evidence, it allows us to look back: how good was the preclinical research to start with? While evidence-based medicine is the health care imperative of the 21st century, it remains difficult to generate the evidence base for pressing public health challenges. This session unites key stages and world authorities in the clinical trials process to explain why and what must change. With expertise on leading animal and human studies to end products, speakers will provide new insights into how clinical trials are conducted. Their common cause will be to argue that greater international cooperation and standards are required. That said, effective communication of clinical and societal benefits must be better coordinated to citizens, patients, health-care professionals, researchers, policy-makers, and society as a whole.

Organizer:

Aidan Gilligan, SciCom–Making Sense of Science

Co-Organizer:

Thomas Hartung, Johns Hopkins University

Moderator:

Klaus Bock, Danish Ministry of Science, Technology & Innovation

Discussants:

Wilson M. Compton, U.S. Department of Health and Human Services

and Anne Glover, European Commission

Speakers:

 

Thomas Hartung, Johns Hopkins University

Look Back in Anger? What Clinical Trials Tell Us About Preclinical Research

 

 

Jay Siegel, Janssen Research and Development

Product-Orientated Versus Health Care–Orientated Clinical Trials

 

 

Paul J. Kenny, The Scripps Research Institute

The Latest Clinical Trial Research on Brain Reward Systems

 

 

Stroke Research: New Concepts and Innovative Solutions

Friday, February 15, 2013: 3:00 PM-4:30 PM

Room 208 (Hynes Convention Center)

Stroke has become a major challenge for medicine and health policy. It is the third largest cause of death in many countries and the largest single cause of severe disability. It is predicted that stroke will account for 6.2 percent of the total burden of illness in 2020. In the last two decades, research and new technology have brought significant advances in the diagnosis and treatment of stroke. For instance, thrombolytic drugs have revolutionized stroke care and improved recovery from the disease. New imaging techniques are used to study structural and functional changes at the cellular and molecular level in the brain. Nevertheless, improvement of reperfusion, reduction of brain damage, and induction of endogenous protection and repair deserve increased efforts to overcome the “translational roadblock“ and bring new therapies. By pooling together multidisciplinary and cross-sectorial expertise, the European Stroke Network drives the generation of new hypothesis and solutions for stroke, with a view toward developing successful strategies for brain protection and repair. The network has established important transatlantic research cooperation with the Canadian Stroke Network and other groups. The symposium will give an opportunity to exchange ideas and identify common scientific challenges that need to be solved to speed up the discovery of new treatments.

Organizer:

Virginija Dambrauskaite, European Commission, Directorate General for Research and Innovation

Co-Organizer:

Ruxandra Draghia-Akli, European Commission, Directorate General for Research and Innovation

Moderator:

Ruxandra Draghia-Akli, European Commission, Directorate General for Research and Innovation

Discussant:

Walter J. Koroshetz, National Institute of Neurological Disorders and Stroke

Speakers:

 

Costantino Iadecola, Weill Cornell Medical College

Great Expectations: The Promise of the Neurovascular Unit for Stroke Therapy

 

 

Molly Shoichet, University of Toronto

Engineering Meets Medicine: Innovative Strategies To Overcome Stroke

 

 

Stephen Meairs, University Medical Center Mannheim, University of Heidelberg

The European Stroke Network: A Platform for Overcoming the Translational Roadblock

 

Stem Cell–Based Bioartificial Tissues and Organs

Monday, February 18, 2013: 9:45 AM-11:15 AM

Room 207 (Hynes Convention Center)

Transplantations of artificial organs have become a reality. They require skills not only in surgery but also in biomaterials, bioengineering, stem cell culture and biology, tumor biology, and molecular biology. In this session, a team behind artificial trachea transplantation in a patient and the analysis thereof will talk about the efforts made to take modern medicine to the next level.

Organizer:

Sabina Bossi, Karolinska Institute

Moderator:

Ana Teixeira, Swedish Medical Nanoscience Center

Discussant:

Doris Taylor, University of Minnesota

Speakers:

 

Paolo Macchiarini, Karolinska Institutet

Regenerative Biotechnological Treatment

 

 

Ola Hermanson, Karolinska Institutet

Molecular and Cellular Effects of Transplanting Artificial Organs

 

 

Philipp Jungebluth, The Cardiothoracic Surgery Network and Karolinska Institutet; Paolo Macchiarini, Karolinska Institute

Clinical Transplantation of a Tissue-Engineered Airway

 

 

Engineering the Nervous System: Solutions to Restore Sight, Hearing, and Mobility

Sunday, February 17, 2013: 1:30 PM-4:30 PM

Room 302 (Hynes Convention Center)

The fields of neuroprosthetics and bioengineering offer hope for many that they may one day be able to see the world around them, walk on their own, or grasp, touch, and hear normally. To restore or enable these functions, a multitude of interfaces and approaches are necessary. This symposium highlights pioneering researchers from Europe and the United States who view the nervous system as engineerable and are working to make a new generation of bionic devices feasible. They are decoding motor commands and developing soft, flexible electronics that interface with the spinal cord and auditory brainstem; genetically engineering the human auditory and visual systems; and coming close to perfecting cortico-spinal prosthetics to reclaim voluntary locomotion after complete spinal cord injury. The scientists are optimistic for the future, yet recognize the challenges in getting these technologies out of the lab and into clinical settings. Live demonstrations at the intersection of engineering and neuroscience will show how close we are to entering a new era in the way science interfaces with the nervous system.

Organizer:

Sanna Fowler, Ecole Polytechnique Fédérale de Lausanne

Speakers:

 

Silvestro Micera, Ecole Polytechnique Fédérale de Lausanne

Controlling a Prosthetic Hand with Peripheral Neural Interfaces

 

 

Grégoire Courtine, Ecole Polytechnique Fédérale de Lausanne

Walking Again After Spinal Cord Injury

 

 

Konstantina M. Stankovic, Harvard Medical School

Treating Deafness with Better Vision: Cellular-Level Optical Imaging of the Inner Ear

 

 

Joan Miller, Harvard Medical School

Saving Sight in Retinal Disease

 

 

Philippe Renaud , Microsystems Lab, LMIS4

Challenges: The Electrical Neurostimulation and Recordings with Microelectrode Arrays