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SPRING 2011 Accelerating Science, Advancing Medicine Dean’s Quarterly SPRING 2011 ACCELERATING SCIENCE, ADVANCING MEDICINE Becoming an Engine of Discovery I am pleased to outline the strategic plan for the Center for Discovery and Innovation (CDI) at The Mount Sinai Medical Center. This entity, designed to capitalize on the unprecedented opportunities of 21st century biomedicine, will bring together some 30 renowned translational scientists, many of whom are successful drug developers and patent-holders already, with the charge of translating discovery into therapeutics and generating new intellectual property. Under the direction of a senior leader with Experimental Therapeutics Institute in five key international stature in translational medicine areas: small-molecule drug discovery, monoclonal and the successful development of therapeutic antibodies and purified proteins, high-content interventions, the CDI discovery group will screening/RNAi, induced pluripotent stem cells, identify the most promising research within all of and systems pharmacology and network analysis Mount Sinai’s disease-focused institutes with the facilities. goal of illuminating new disease targets and the The success of the CDI enterprise and the Dennis S. Charney, MD, is the Anne and Joel Ehrenkranz molecules that treat those targets. scientists it recruits and supports will ultimately Dean of Mount Sinai School of Medicine and the Executive Vice President for Academic Affairs of The Mount Sinai This new era of growth will span years and will be measured by the discoveries made, and the Medical Center. require great institutional commitment and applications developed, to predict, prevent, investment, and the results will be breathtaking. and treat disease. I look forward to sharing with The CDI is structured to focus on five areas that you the progress and results of CDI throughout To learn more, visit www.mountsinai.org/Charney represent the most pressing global disease burden: the coming years. cardiovascular disease, cancer, neurological See the Drug Discovery Roadmap on page 2. disorders, immune disorders, and virology/ corE facIlITIES of THE ExpErImENTal THErapEUTIcS INSTITUTE (ETI) DISEaSE focUS arEaS vaccines. To explore these of ETI corE facIlITIES areas in the greatest depth 5 Small- Monoclonal High-content Induced Systems carDIovaScUlar DISEaSE and breadth possible, we molecule drug antibodies screening/ pluripotent pharmacology caNcEr will expand the facilities discovery and purified RNAi stem cells and network NEUrologIcal DISorDErS and capabilities of our proteins analysis ImmUNE DISorDErS vIrology/vaccINES INSIDE THIS ISSUE DISTINGUISHED BY SERVICE 2 Medical Milestones Department of Rehabilitation Medicine Anniversary 3 Research Frontiers In 1910, The Mount Sinai Medical Center established one Kristjan T. Ragnarsson, MD, Lucy G. Moses Professor 3 Young Pioneers of the nation’s first departments devoted to rehabilitating and Chair of the Department of Rehabilitation Medicine, 4 New Faces patients with disabling injuries and medical conditions. Today, and Wayne A. Gordon, PhD, the Jack Nash Professor 5 Philanthropy the Department of Rehabilitation Medicine is consistently of Rehabilitation Medicine, and an Associate Director 5 Patient-Centered Care ranked one of the best in the nation by U.S. News & World of the Department of Rehabilitation Medicine, lead an Report in its annual Best Hospitals survey. The Department interdisciplinary team of professionals. 6 Photo Essay commemorated its 100th anniversary in November. Continued on page 2 7 Commentary ACCELERATING SCIENCE MEDICAL MILESTONES The Drug Discovery Roadmap A New Alternative to Open Heart Surgery Mount Sinai and other academic Three leading physicians at Mount Sinai Heart Approximately 100,000 people in the United medical centers are the new have performed the first U.S.-based percutaneous States are diagnosed with severe aortic stenosis implantation of a novel device that replaces a each year, but one-third of them do not qualify engines of research and discovery diseased aortic heart valve, providing a viable for surgery due to their advanced age, the fact for the pharmaceutical industry. treatment for patients too elderly or high risk that they’ve suffered a stroke, or have lung disease. to undergo open heart surgery. The device, the In the past, physicians have relied on a balloon BaSIc ScIENcE rESEarcH Medtronic CoreValve® Transcatheter Aortic Valve procedure to open the heart valve, but this has Prosthesis, treats severe aortic stenosis with a proven to be a temporary fix, with results lasting Scientists pursue basic research catheter-based technology that does not require up to nine months before the symptoms return. questions in the laboratory to understand an incision in the chest or the use of a heart Dr. Sharma says the balloon procedure can be the fundamental mechanisms of lung machine. performed only a few times before it actually molecular, cell, and systems biology. begins to weaken the patient’s heart valve. David H. Adams, MD, Marie-Josée and Henry R. Kravis Professor and Chair of the Department Implantation of the CoreValve, a self-expanding THE cENTEr for DIScovEry of Cardiothoracic Surgery; Samin K. Sharma, stent measuring slightly more three centimeters, aND INNovaTIoN (cDI) MD, the Zena and Michael A. Wiener Professor is guided over a wire and a catheter placed in TraNSlaTIoNal ScIENcE and Director of the Cardiac Catheterization a peripheral artery through the diseased aortic Scientists use animal models and human Laboratory; and Annapoorna S. Kini, MD, valve, and once it expands in this position, the patients to explore disease mechanics. Associate Professor and Associate Director of new aortic valve begins functioning immediately. the Cardiac Catheterization Laboratory, have begun using the CoreValve DIScovEry of poTENTIal System as part of the FDA’s NovEl THErapEUTIcS Pivotal Clinical trial. Dr. Scientists collaborate with research Adams is the trial’s national staff in ETI core facilities to develop Co-Principal Investigator. new molecules with the goal of creating new therapies, including mAbs, small “We think more than 70 molecules, percent of patients receiving and biologics. Industry partnerships with the CoreValve will survive biotech and pharmaceutical more than two years,” says companies begin to form. Dr. Sharma, whose estimates Industry partners assist in are based on the 13,000 devices the final steps of therapy that have been implanted in discovery to determine safety patients throughout Europe since 2007. pHaSE 1 and efficacy, and to incorporate fDa SafETy therapy into medical practice. TrIalS David H. Adams, MD (LEFT), and Researchers test new treatments for Samin K. Sharma, MD (RIGHT), with the CoreValve device. safety and, in some cases, efficacy in humans through standard FDA trials. pIvoTal fDa clINIcal TrIalS Physician-scientists conduct large-scale, well-controlled efficacy trials in order to DISTINGUISHED BY SERVICE gain FDA approval. Department of Rehabilitation Medicine Anniversary, continued from page 1 mEDIcal pracTIcE IS cHaNgED The new therapy becomes available to The department has become a world-renowned center of excellence providing comprehensive patients. Large-scale effectiveness trials rehabilitation for patients with spinal cord injury, brain injury, stroke, limb amputations, multiple traumas, and joint replacement surgery. It is the only federally designated Model System of Care are conducted in patients with more facility in New York State for spinal cord injury and traumatic brain injury (TBI). Among its many complicated diseases to guide medical research accomplishments is the development of an assessment tool for diagnosing subtle brain damage, practice. and an intervention to address impairment of executive function, such as goal-setting, problem-solving, and planning abilities in patients with traumatic brain injury. Thousands of patients have reclaimed their lives through care and support from Mount Sinai practitioners. Notable examples include ABC News Reporter Robert Woodruff, who in 2006 sustained TBI after being hit by a roadside bomb while on assignment in Iraq, and Dennis Byrd, a former New York Jets football player who broke his neck during a game in 1992, and now can walk on his own. 2 MEDICAL MILESTONES RESEARCH FRONTIERS A New Alternative to Open Heart Surgery $17.2 Million Grant for Research New Discovery into the on Immune Modeling and Origins of Brain Cells Experimentation Miriam Merad, MD, PhD, Associate Professor of Gene and Cell Medicine at Mount Sinai School of Medicine recently received a five-year, $17.2 Mount Sinai School of Medicine, and her million grant from the National Institute of Allergy and Infectious team of researchers at The Mount Sinai Diseases, part of the National Institutes of Health (NIH), to improve Medical Center have made a significant the collection and computation of data used to study immunology and discovery that microglia, the small, infectious diseases. immune cells that reside in the central nervous system, have a unique origin, This is the second round of funding and are formed shortly after conception. that MSSM’s Program for Research on Microglia had been thought to develop Immune Modeling and Experimentation at birth, and originate at the same time (PRIME) has received since 2005, when the as macrophages, the body’s other immune program—directed by Stuart C. Sealfon, cells. The findings were published in MD, Glickenhaus Professor and Chair of the Miriam Merad, MD, PhD Science in November. Department of Neurology—was
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