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This past year, the School of Medicine rolled out a new five-year research strategic plan and looked back at progress over the past two decades to gain insight into directions for the future. From 1989 to 2009, the school had a 10-fold increase in NIH research funding. Almost every year since the mid-1990s, the school has been either the first- or second-fastest rising recipient of NIH dollars. And today its ratio of impactful scientists—those cited frequently in the literature and those whose discoveries have made them “gamechangers,” as a new recognition program calls them—is dense in relation to the relatively small size (450) of its research faculty.
What are the reasons underlying these successes? It doesn’t hurt, says Ray Dingledine, the school’s research dean, that over the past two decades endowment and donor support enabled major research construction (10 new buildings or major additions) and investment of $1.24 billion in Emory’s research enterprise. But credit also goes to stability of senior leadership, he says, which allowed time for vision to become reality and made possible numerous joint recruitments and pooled efforts among disciplines as well as a collaborative perspective that encourages, incentivizes, and nurtures team science.
In this section
1. Genetics researcher Stephanie Sherman has spent more than two decades studying causes and consequences of Down syndrome and has amassed the largest database on this disorder in the country. Sherman hopes that targeted drug therapy will soon become available to improve cognitive learning abilities for those with Down syndrome.
2. Biomedical engineers from Georgia Tech and heart surgeons from Emory have developed a device that provides access to a beating heart during surgery. The device prevents blood loss, enhances safety, and reduces costs.
3. Ioanna Skountzou (right) and Chinglai Yang (microbiology and immunology) are working with researchers at Georgia Tech on microneedle skin patches that could be used to deliver flu vaccine.
4. Transplant clinician-scientists Chris Larsen (right foreground) and Tom Pearson (left) were leaders in developing a new class of immunosuppressive drugs with fewer side effects. In June 2011 the FDA approved the first of these—Nulojix (belatacept)—to prevent rejection of kidney transplants.
5. Rita Nahta (pharmacology) studies therapeutic implications of growth factor signaling cross-talk in breast cancer and is identifying new molecular targets against which future drugs can be developed.
Drug discovery: from lab to clinic
According to a ranking that appeared in a recent New England Journal of Medicine, Emory University is the fourth largest contributor among public-sector research institutions to the discovery of new drugs and vaccines—outranked only by the NIH, University of California System, and Memorial Sloan-Kettering. Some of these Emory drugs include 3TC and FTC, which are taken in some form by more than 94% of U.S. patients (and thousands more globally) receiving therapy for HIV/AIDS.
Brain disorders—More new drugs, diagnostics, and devices are on the way. This past year, Emory researchers identified a new class of compounds that alter the function of NMDA receptors, critical players in communication between brain cells. This work may lead to new drug treatments for schizophrenia, Parkinson’s, and other disorders. Blocking one variety of NMDA receptor to mitigate brain damage from stroke is the focus of NeurOp, one of more than 50 start-up companies Emory has launched since the 1990s to move drug discovery toward clinical use. NeurOp recently announced a two-year research collaboration with Bristol-Myers Squibb.
Improved transplant drugs—This past summer, Emory clinician-scientists learned of long-awaited FDA approval of the drug Nulojix (belatacept) for prevention of graft rejection after kidney transplants. This is the first time a new class of drugs has been developed for transplant since the 1990s. Belatacept has the potential to improve and simplify the medication regimens of kidney transplant recipients and is also now being tested in experimental clinical trials for liver and pancreatic islet transplant.
The search for a universal flu vaccine got a boost when Emory Vaccine Center researchers took blood samples from nine patients infected with the 2009 H1N1 virus. Astonishingly, the patients had antibodies against a wide variety of flu strains, including all seasonal H1N1 flu strains from the past decade, the Spanish flu strain of 1918, and a pathogenic H5N1 strain. Some of these antibodies stuck to the “stalk” region of the virus, which changes less than other regions. These broadly protective, stalk-reactive proteins previously were thought to be exceedingly rare, but they were promisingly abundant in these patients, suggesting a way to produce a vaccine for permanent immunity to all flu strains.
Going for the constant—Emory researchers who study virus-like proteins (VLPs) also are focusing attention on components of the flu virus that remain relatively constant from one strain to another. They combined a standard strain of flu with VLPs containing the relatively constant viral protein M2. Used by itself, M2 offers little immune protection. The combination, however, provided a high level of protection to mice exposed to pandemic H1N1 and an H5N1 strain, suggesting that supplementation of seasonal flu vaccines may overcome limits of strain-specific vaccines.
Less pain, more antigens—A $10 million NIH grant will advance technology developed by Georgia Tech and Emory for painless self-administration of flu vaccine using patches with tiny microneedles that dissolve in the skin. The ability to painlessly immunize large numbers of people without need for medical personnel would increase the number of people being vaccinated, especially children and the elderly. Better yet, the microneedle patch appears to be more effective at vaccine delivery than traditional syringes because of the large number of antigen-presenting cells that reside in the skin.
Glioblastoma multiforme—This most common and primary brain tumor often recurs because cancer cells hide in surrounding brain tissue and survive initial treatment. Mouse studies in Emory’s brain tumor nanotechnology lab show that tiny, antibody-linked particles of iron oxide bind to and kill human glioblastoma cells without causing toxicity to normal brain cells. The particles also make tumor cells more visible on MRI. Clinical trials are on the horizon.
Solid tumors—Complete tumor removal is the most important predictor of patient survival. Biomedical engineers at Emory, Georgia Tech, and University of Pennsylvania have developed a handheld SpectroPen that allows surgeons to visualize tumor edges and spot cancer cells in lymph nodes during surgery. The pen detects light from tiny gold particles coupled to fluorescent dye and an antibody that sticks to tumor cells more than normal ones. Investigators hope to begin clinical trials in lung cancer soon, but the SpectroPen already is being used at the University of Georgia College of Veterinary Medicine to treat dogs with naturally occurring tumors.
Head and neck cancer—Emory and Georgia Tech researchers are coupling gold nanoparticles with antibodies against a growth factor common to this type of cancer. When the nanoparticles travel to cancers in the soft tissues of the mouth and throat, researchers use a laser to apply energy. The gold particles absorb this energy and convert it to heat, selectively killing cancer cells and sparing normal ones. Animal studies of toxicity and efficacy are required before clinical testing can be done in humans.
Apica Cardiovascular, a company started by Emory and Georgia Tech to develop a proprietary device that provides access to a beating heart during surgery, recently received $5 million in venture capital funding. The technology attaches a conduit to the beating heart so surgeons can deliver therapeutic devices, such as aortic or mitral valves, without loss of blood that occurs with conventional sutures, thus improving safety and decreasing procedure time and costs. Apica recently was named “start-up of the year” by Emory’s Office of Technology Transfer.
Researchers at Georgia Tech and Emory received a $14.6 million contract from NIH to translate relatively mature nanotechnologies into clinical applications for heart disease. Their goals are four-fold: (1) developing nanoparticle probes to image and characterize atherosclerotic plaques to detect early-stage disease and determine which plaques will grow and rupture; (2) determining presence or levels of protein markers, reactive oxygen species, or micro-RNAs as indicators of the presence and stage of atherosclerosis; (3) using nanoparticles to deliver therapeutic agents in a specific, sufficient, and sustained manner to localized vascular lesions; and (4) delivering patient-specific stem cells to vasculature and heart tissue damaged by atherosclerosis.
Collaboration between Emory and Children’s Healthcare of Atlanta grew this year with establishment of the Emory-Children’s Pediatric Research Center, linking investigators in cancer, transplant, cystic fibrosis, and other areas with pediatricians treating these diseases.
Another new collaboration, the Center for Pediatric Nanomedicine, includes physicians and scientists from Georgia Tech, Emory, and Children’s Healthcare, who are working to develop nanoscale structures to better diagnose and treat diseases and repair damaged tissues.