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Intensifying our focus

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The medical school finds itself well positioned to address some of the nation's most challenging research issues: How to take up the growing slack in drug discovery and get new therapies and technologies to patients. How to produce better health outcomes at lower costs. How to advance care for big global killers like heart and infectious disease. Much of the fivefold growth in Emory's research portfolio during the past 16 years has been centered on these areas.

Drug testing and development 

As the pharmaceutical industry narrows the focus of its own discovery engines, it is turning increasingly to academic medicine for collaboration in drug testing and development. Emory and Bristol-Myers Squibb, for example, recently formed a partnership in which investigators from Emory and affiliated institutions will conduct clinical trials in metro Atlanta to support development of investigational medicines from across Bristol-Myers Squibb's portfolio, particularly in oncology, metabolics, hepatitis C, and immunoscience. The agreement builds on recent experiences between the two organizations in conducting clinical trials in organ transplantation and cancer. 

Meanwhile, the Emory Institute for Drug Development (EIDD) is collaborating with Scynexis, Inc., to develop the first antiviral drug for mosquito-borne dengue fever, which infects 50 million people a year worldwide, including recent cases in Florida. Drugs already exist for tuberculosis, another EIDD focus, but the six- to nine-month treatment course leads to poor compliance, allowing the bacterium to rebound, producing drug resistance. EIDD is searching for a compound that would cut treatment regimens to a more workable two or three weeks. A partnership with GlaxoSmithKline gives EIDD a head start: access to more than 600 patents for drugs. 

Helping investigators take the next step—Emory's technology transfer office manages more than 1,000 Emory-invented technologies. Management of inventions has led to formation of 63 new companies and introduction of 50 new products to market, with royalties earned from sales of new products by Emory licensees used to subsidize research and education. To help grow the number of new biotech companies in Georgia, Emory recently teamed up with the University of Georgia to offer 22 budding entrepreneurs from Emory, Georgia Tech, Morehouse School of Medicine, and UGA a six-week course on how to start and run a business, from market analysis to identifying and working with investors. 

Brain tumors: a new direction for progesterone—Based on Candler Professor Don Stein's pioneering research, medical centers across the country have been conducting an NIH-sponsored, Emory-led phase 3 clinical trial of progesterone for acute traumatic brain injury (TBI). In other studies in the lab, Emory researchers have noticed an additional apparent benefit of progesterone besides that for TBI: In mice the hormone not only protected healthy neurons but also caused tumor cells to die. As they reported in Molecular Medicine, high doses of progesterone kill neuroblastoma cells—the most common cancer in very young children—while leaving healthy cells unscathed. Progesterone cut tumor growth in half, decreasing proteins produced by tumor cells to attract blood vessels for nourishment and invasion of nearby healthy tissues. The scientists now are determining optimal dosage, length of treatment, and combination with radiation or chemotherapy, before moving to human clinical trials. 

New stroke drugs—Compounds called prostaglandins have been found in animal research to protect the brain from damage following stroke, and Emory pharmacologists have identified other compounds that enhance these protective effects. They believe drugs targeting a specific prostaglandin receptor, if given in the ICU, could lower risk of a repeat stroke. Creating and developing such a drug will move more rapidly, thanks to the new NIH Blueprint for Neuroscience Research, which selected Emory as one of seven teams nationwide to develop these compounds, providing unprecedented access to pharmaceutical and biotechnology industry consultants to assist throughout the drug-development process.

Translating cancer genome data into therapies—The new Emory Molecular Interaction Center for Functional Genomics (MicFG) focuses on protein-protein interactions that often get rewired in cancer, driving tumor development and progression. MicFG's goal is to map these interactions and identify molecular targets where "pathway-perturbing" drugs could disrupt them. The center's team has extensive experience in genomics data mining and analysis in the technology-rich Emory Chemical Biology Discovery Center, which tests compounds for use as potential drugs to target these protein-interaction pathways. Meanwhile, researchers in Emory's Winship Cancer Institute are working to translate MicFG discoveries into patient therapies. Funded by $4.3 million from the National Cancer Institute, MicFG is the only southeastern center selected for the NCI's nine-member Cancer Target Discovery and Development network.

Health services research

In an era of increasingly stringent cost containment, emphasis on providing the highest quality of care possible within a given amount of resources makes health services research (HSR) one of the fastest-growing research areas nationwide. Emory has a natural advantage in the hot new field, thanks to a large health care system and strong clinical partnerships with access to diverse patient populations and lots of patient data. The Emory/Georgia Tech Healthcare Innovation Program (hip.emory.edu), a virtual network, exemplifies the growing culture of HSR collaboration throughout the region, while individual research projects illustrate the breadth and power of HSR. For example, Emory University Hospital's chief of gastrointestinal surgery joined forces with Georgia State University's director of experimental economics to understand why roughly six of every 100 surgery patients nationwide are readmitted within 30 days. The team of economists examined hundreds of thousands of observations on 3,000 Emory surgery patients. Results: a software risk profile, now being tested, that recommends whether a patient be discharged or stay in hospital.

Heart research

Identifying "vulnerable"plaque—Most heart attacks or strokes occur when unstable arterial plaque causes a clot. Being able to predict where such instability is most likely to occur would give clinicians a new way to intervene. In the largest published investigation of shear stress (a measure of how hard blood tugs on arterial walls) and plaque progression in humans, Emory clinicians and Georgia Tech experts in fluid mechanics were able to predict which areas of the coronary arteries develop more atherosclerotic plaque before and after six months of statin therapy. Some plaque develops in steady progression and other plaque, even in the same patient and same artery, develops in fits and spurts. The team found that plaque formed in steady progression is more stable, whereas plaque that develops in fits and spurts is more likely to rupture. The work appeared in Circulation. 

Regenerating damaged hearts—Earlier work at Emory demonstrated that high doses of a patient's own bone marrow cells can improve blood flow and help heal injury caused by heart attack. Now Emory is heading a multi-institution clinical trial to establish bone marrow cells as a therapeutic option. All 160 patients receive angioplasty and stents. Half have 10 million bone marrow cells infused into the coronary artery where blockage caused the heart attack; the other half receive placebo. Effectiveness is assessed by SPECT imaging of blood flow beginning six months after treatment and continuing 36 months.

Understanding causes of ischemia—Insufficient blood flow to the heart triggered by psychological stress produces higher risk of adverse cardiac events, even death, than ischemia brought on by physical stress. With an $11 million NIH grant, Emory cardiologists are working with heart patients to understand these differences. Studies focus on genetics and variations in response to stress of the brain and heart (as measured by PET scans) and the vascular system. For example, ischemia caused by mental stress occurs in different areas of the heart from that caused by physical stress. Hormones secreted during excitement or stress cause arteries and vessels to dilate in most people, but in other people these same chemicals cause constriction. 

Finding power in numbers—The Emory Center for Heart Failure Therapy and Transplantation was selected as one of nine members of the National Heart, Lung and Blood Institute's heart failure clinical research network. Member institutions combine patient populations and jointly design and conduct clinical trials for all forms of heart failure.

Vaccines

HIV prevention—An HIV/AIDS vaccine developed at the Emory Vaccine Center, Yerkes National Primate Research Center, and GeoVax Labs protects nonhuman primates against multiple exposures to simian immunodeficiency virus (SIV), the nonhuman primate version of HIV. In clinical trials supported by the NIH HIV Vaccine Trials Network, an earlier generation of the new vaccine produced excellent vaccine responses in more than 400 uninfected people, setting the stage for this new, second-generation vaccine to move into safety and efficacy human trials later this year in participants at high risk of exposure to HIV. The new vaccine regimen, designed for a version of the virus prevalent in the Americas, includes a DNA prime vaccine that co-expresses HIV proteins and a granulocyte-macrophage colony-stimulating factor (GM-CSF) that promotes the initiation of immune responses and thus enhances the ability of the vaccine to elicit blocking antibodies before the virus enters cells.

In other work, the Emory Vaccine Center, along with Yerkes Research Center, recently received two grants to participate in large, concerted HIV vaccine efforts. One is to conduct studies on CD4+ T cells as part of the NIH's new Centers for HIV/AIDS Vaccine Immunology & Immunogen Discovery. The other, from the Gates Foundation, is focused on inducing optimally effective anti-HIV antibodies. Both will use an Emory-developed technology using virus-mimicking nanoparticles to stimulate long-lasting immune responses.

Global collaboration—The medical school, through the Emory Vaccine Center, is the only U.S. medical school represented among 42 global partners in a new collaborative research program, Advanced Immunization Technologies, designed to accelerate human vaccine development. Led by Novartis, the partnership was launched with 4 million euros from the European Union and collaborating organizations, with the World Health Organization as a senior partner.