Eugene Schiff

In what’s being called the start of a new era in treating hepatitis C, a team that includes a leading Miller School physician has discovered a drug that appears to dramatically increase the success of standard therapy.

For decades, patients with hepatitis C have faced an unpleasant reality: the standard therapy is effective in only 40 percent of cases and the side effects of the lengthy regimen can be debilitating. For that reason, many patients with genotype 1 chronic hepatitis C have hesitated taking the standard therapy of peginterferon plus ribavirin.

Eugene R. Schiff, professor of medicine and director of the Center for Liver Diseases, led the Miller School arm of a multisite phase II study, which found that adding boceprevir, an NS3 protease inhibitor, to the standard therapy of peginterferon and ribavirin doubles the sustained virological response in patients who have not been treated previously. The study, conducted at hospitals in the United States, Canada, and Europe, was published in the August 9 online edition of The Lancet.

“This is what many people have been waiting for,” says Schiff. Patients, he explains, have wanted a protease inhibitor that would effectively stop the disease progression in their liver.

Peginterferon and ribavirin work by stimulating the patient’s immune system to attack the hepatitis C virus. Boceprevir is a direct antiviral, which works by attacking the virus directly, stopping it from replicating. Adding this protease inhibitor doubled the sustained response rate.

Boceprevir is one of the first of this group of direct antivirals that will soon be licensed for treatment. Schiff expects this particular drug to be licensed in spring 2011, bringing out many more patients to be treated. “There’s no question this is a major advance,” he says.

Schiff says this is a preview of the future when he expects that patients will be given a combination of antivirals and won’t need peginterferon and ribavirin at all.

One of the reasons patients fail to respond to peginterferon is that hepatitis C has a component that blocks the activation of interferon in the body. However, when physicians use a direct antiviral that lowers the virus in the system, the interferon given and the interferon in the body work more efficiently in what’s known as a “restoration of the innate immune response.”

Paul Martin, professor of medicine and chief of the Division of Hepatology at the Miller School, is just as hopeful as Schiff, saying that “these results help usher in a new era in the treatment of hepatitis C, with cure now possible in many more patients.”

The Department of Medicine will host grand rounds on Wednesday, September 1 at 12 p.m. in the Rosenstiel Medical Science Building, third-floor Auditorium. Cynthia Levy, associate professor of medicine in the Division of Hepatology at the Miller School of Medicine, will present “Primary Biliary Cirrhosis.” For more information, please contact Cristina Orbeta at COrbeta@med.miami.edu or 305-243-6484.

From left, Dean Pascal J. Goldschmidt, Christopher Mosley, senior vice president and chief administrative officer of Jackson Health System, U.S. Senator Bill Nelson, Fabiola Surena, her father, Claude Surena, a senior official from the Haiti Ministry of Health, and Barth Green, professor and chair of neurological surgery, listen to a doctor talk about the patients at the Project Medishare hospital in Haiti via the Ryder Trauma Center's RP-7 robot.

In a visit to the Ryder Trauma Center last Friday, U.S. Senator Bill Nelson vowed to continue his efforts to secure funds for the critical care and rehabilitation hospital established in Port-au-Prince by the Global Institute and Project Medishare after Haiti’s devastating earthquake.

Originally located in four tents at the edge of the airport, the hospital moved to an existing community hospital in June and continues to serve as a vital critical care and rehabilitation hospital as well as a training center for local physicians, nurses, and allied health professionals eager to fill the large gaps in Haiti’s shattered health care system.

Barth Green, professor and chair of neurological surgery who co-founded Project Medishare to improve health care access in Haiti, said Project Medishare is counting on a $17.7 million federal grant to operate the hospital over the next 18 months and help Haiti build a sustainable health care system. Nelson, Florida’s senior senator, wrote a letter urging the U.S. State Department to direct a portion of the Haiti relief funding in the 2010 Emergency Supplemental Appropriations Act to the hospital.

“My compliments to all of you,’‘ Senator Nelson told Green, Miller School of Medicine Dean Pascal J. Goldschmidt, and Enrique Ginzburg, professor of surgery, during his visit. “What you’ve done is heroic and it’s in the best tradition of America. I will continue to be your advocate.’‘

During his visit, Senator Nelson also witnessed firsthand the capabilities of the mobile Remote Presence RP-7 robot, which through wireless communication is allowing physicians at the hospital in Haiti to consult with specialists at the Ryder Trauma Center. Donning a mask and yellow gown, the senator also visited briefly with a Haitian patient who, more than seven months after barely surviving the earthquake, just improved enough to leave the ICU.

L.D. Britt, Brickhouse Professor and Chair of Surgery at Eastern Virginia Medical School, will present “Penetrating Trauma Management-2010: What is Evidence-Based Practice?” on Thursday, September 2 from 7:30 to 8:30 a.m. at the Rosenstiel Medical Science Building, fourth-floor auditorium. Upon completion of this lecture, participants will be able to outline the diagnostic modalities required in the evaluation of penetrating trauma and highlight evidence-based data supporting specific management paradigms. The University of Miami Leonard M. Miller School of Medicine designates this educational activity for a maximum of 1 AMA PRA Category 1 Credit. For more information, please contact Elisa Arguelles at 305-585-1280 or earguelles@med.miami.edu.

The Department of Neurological Surgery will host grand rounds on Thursday, August 26 from 8 to 9 a.m. in the Lois Pope LIFE Center, seventh-floor auditorium. Brian Lally, assistant professor of radiation oncology, will present “Radiation Options for Spinal Tumors.” For more information or to add your name to the department’s educational announcement distribution list, please contact Ingrid Menendez at 305-243-6751 or nrstraining@med.miami.edu.

The Department of Neurological Surgery will host grand rounds on Thursday, August 26 from 7 to 8 a.m. in the Lois Pope LIFE Center, seventh-floor auditorium. Sanjiv Bhatia, associate professor of neurological surgery at the Miller School of Medicine, will present “Glioneuronal Tumors.” For more information or to add your name to the department’s educational announcement distribution list, please contact Ingrid Menendez at 305-243-6751 or nrstraining@med.miami.edu.

Research associate Claudia Chambers demonstrates the proper use of a sleep monitor to a participant in the Hispanic Community Health Study/Study of Latinos.

Less than four years after the National Institutes of Health launched the largest long-term health study of Hispanic/Latino populations, researchers at the four sites, including the University of Miami, have published their first papers. The research appears in the August issue of Annals of Epidemiology.

The first study, titled “Design and Implementation of the Hispanic Community Health Study/Study of Latinos,” examines the rationale behind the study, objectives, design, and how it will be implemented. Neil Schneiderman, professor of psychology, and Leopoldo Raij, professor of medicine, were authors on the paper.

The second study, titled “Sample Design and Cohort Selection in the Hispanic Community Health Study/Study of Latinos,” looked at the sample design and cohort selection for a study designed to recruit and follow a cohort of 16,000 Hispanics/Latinos ages 18 to 74. David J. Lee, professor of epidemiology and public health, and John Ryan, research associate professor of family medicine and community health, were authors on the paper.

“Data thus far have been collected on more than 10,000 of the 16,000 participants in the largest health study ever conducted upon Hispanics/Latinos in the United States,” said Schneiderman, the James L. Knight Professor of Psychology, Medicine, Psychiatry and Behavioral Sciences, and Biomedical Engineering at the University of Miami. “The study is examining differences and similarities in health risks, practices, and outcomes in peoples from different Hispanic/Latino backgrounds.” Schneiderman is principal investigator of the Miami Field Center of the study.

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New medical student Tyler Beals listens attentively as faculty members welcome the Miller School’s newest students, the Class of 2014.

Among the students in the Miller School’s new Class of 2014 is Gabriella Polyak, a Miami native fluent in Russian who has yearned to be a doctor since she was a child. There’s also Angela Hippeli, a Spanish-speaker from Pennsylvania who spent time in Ecuador; she is sure she chose medicine when she was 7. And there’s Andrew Boulos, the son of a physician who hopes the medical profession can help him become “half the man” his father is.

Then there’s Tyler Beals, who tried to keep his career options open—but kept coming back to medicine.

“It’s the only thing I can really imagine myself doing and being truly happy, truly challenged, and get full satisfaction from my work,” said Beals. “This is what I have been preparing for for the past six years. It’s kind of a rush finally being here.”

Here was the fifth-floor auditorium at the Miller School’s Rosenstiel Medical Science Building, where Beals, Polyak, Hippeli and Boulos sat recently with 148 fellow students who are fulfilling long-nurtured dreams by embarking on the study of medicine.

Freshman orientation is the official five-day welcome for new students who made the Miller School’s competitive cut from 5,282 applicants.

As is customary on Day One, numerous faculty members, staff, and upper-class students used humor to welcome the group and allay first-day fears about the demands of intense studying while making new friends and learning a new city.

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Barth A. Green, professor and chair of neurological surgery at the Miller School of Medicine, will receive the prestigious Lawton’s Heart Humanitarian Award for his lifetime devotion to humanitarian causes from the Florida Association of Nonprofit Organizations (FANO). The award will be presented by Miller School Dean Pascal J. Goldschmidt during the opening reception of the organization’s annual conference.

The reception will be held on Wednesday, August 18, from 5 to 8 p.m. at the Hilton Fort Lauderdale Marina, 1881 S.E. 17th Street Causeway, Fort Lauderdale. The price for the reception is $20. The cost for the reception and two-day conference, “A Better Path: Bold Steps to Stronger Resources,” is $95.

FANO is a 20-year-old organization serving Florida nonprofits. The award program was initiated in 1992 to bring public recognition to individuals who dedicate their lives to humanitarian work. In 1999 the name of the award was changed to the Lawton’s Heart Humanitarian Award in honor of the late Florida Governor Lawton Chiles, whose entire career exemplified dedication to community service.

Green, who co-founded Project Medishare in 1994 to improve access to medical care and education in Haiti, organized the first medical team to arrive in Port-au-Prince one day after the 7.0-magnitude earthquake destroyed much of the capital city.

For more information on attending the award ceremony for Green, please call 305-557-1764 or visit www.fano.org.

Warren Kupin, professor of clinical medicine at the Miller School of Medicine, will present “Herbs and Heavy Metals: A Recipe for Nephrotoxicity” on Tuesday, August 17 from 8 to 9 a.m. in the Mailman Center for Child Development, eighth-floor auditorium. For more information, please contact Javier Salazar at 305-585-6042 or jsalazar@med.miami.edu.

Gwenn E. McLaughlin, professor of clinical pediatrics at the Miller School of Medicine, will present “Update on Quality and Safety at Holtz Children’s Hospital” on Tuesday, August 24 from 8 to 9 a.m. at the Mailman Center for Child Development, eighth-floor auditorium. For more information, please contact Javier Salazar at 305-585-6042 or jsalazar@med.miami.edu.

An educational pioneer at the Miller School, Laurence Gardner, executive dean for education and policy (at podium), led efforts to launch the new Public Health Physician curriculum, which will allow students to jointly earn an M.D. and M.P.H. after four years of school.

The Miller School of Medicine has created a new combined M.D./M.P.H. track that will award participating students both an M.D. and an M.P.H. (Master of Public Health) after four years of school. The program will be based in both Miami-Dade and Palm Beach counties and will respond directly to a severe shortage of physicians trained in public health and health care delivery.

The new Public Health Physician curriculum combines the Continuity Medical Curriculum (CMC) pioneered by UM during its joint program with Florida Atlantic University with a series of required and innovative courses and experiences in public health. The CMC curriculum has been fully accredited by the Liaison Committee on Medical Education and emphasizes small-group instruction throughout. The program, which will start in June 2011, will begin earlier in the year than the traditional medical curriculum and include public health courses and experiences throughout the four years.

The first two years of the program will be taught on the Miami campus, while years three and four will take place predominantly in Palm Beach County at the clinical sites currently affiliated with the University. In addition, the Palm Beach County Health Department and the Florida Public Health Institute will be important sites of clinical and public health education.

“This program will be one of only a handful in the country to enable students to finish the accredited M.D./M.P.H. degree program in four years, instead of five,” said Dean Pascal J. Goldschmidt. “The leadership of the Miller School and the Department of Epidemiology and Public Health will join forces with community partners to implement the program.”

The program is designed to provide medical students with the skills to approach health problems from a population and prevention perspective by integrating the roles of the biological sciences and clinical practice with a broader knowledge base of practices used in public health.

“In the future, promoting health and preventing disease at a community and patient level will be as central to the job of the physician as treating illness is today,” said José Szapocznik, professor and chair of epidemiology and public health.

“We have already piloted the medical portion of the curriculum at our regional campus in Boca Raton and received accreditation,” said Laurence Gardner, executive dean for education and policy. “That success will enable us to more easily integrate the public health component with our current community partners. We will also be looking at a different type of applicant pool for this program, as our aim is to graduate a physician with a broader view of health, wellness, and prevention.”

The U.S. Cuban-American Golf Association (USCGA) and American Airlines announce the 17th Annual Cuba Libre Cup, August 13-15 at the Ocean Reef Club, 35 Ocean Reef Drive, in Key Largo. The USCGA is committed to giving back to the community by supporting local charities and foundations in South Florida. This year all proceeds will benefit The South Florida Fragile X Clinic at the University of Miami Miller School of Medicine. To participate in the event, please e-mail the tournament director at cubalibrecup@msn.com or call Jorge Iglesias at 305-788-4128.

BIU President Moshe Kaveh, standing at right, outlines the goals of the new medical school to the UM delegation and Israeli President Shimon Peres. Seated, from left, are Joseph Rosenblatt, UM professor of medicine; Harold Basch, BIU vice president for research; Dean Pascal J. Goldschmidt; UM President Donna E. Shalala; and President Peres.

Led by UM President Donna E. Shalala, a delegation of Miller School officials recently concluded a successful week of high-level meetings in Israel aimed at identifying areas of collaboration and synergy with Bar-Ilan University, as Israel’s largest and fastest-growing university establishes the nation’s fifth medical school.

A research powerhouse with four regional campuses, BIU was chosen earlier this year by Israel’s Council for Higher Education to address Israel’s looming physician shortage by launching a new medical school in the diverse and underdeveloped northern region of Galilee, in the ancient and holy city of Safed.

In a series of July meetings with BIU’s leadership and Israeli President Shimon Peres, Israeli Prime Minister Benjamin Netanyahu, and other Israeli officials, President Shalala, Dean Pascal J. Goldschmidt, and a delegation of prominent UM faculty expressed their eagerness to collaborate with BIU in the development of innovative curriculum, research and clinical programs, the exchange of students, faculty and patients, and the creation of a world-class medical destination in the Galilee.

Motivated by the benefits of cross-institutional collaboration, as well as their close ties to Israel, the Miller School’s Joseph Rosenblatt, professor of medicine and interim director of Sylvester Comprehensive Cancer Center, and Michael Lewis, professor of anesthesiology and director of the residency program, initiated the UM-BIU liaison two years ago.

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Ian McNiece, Juan Pablo Zambrano, Alan Heldman, and Joshua M. Hare.

Miller School researchers have demonstrated exactly how mesenchymal stem cells from bone marrow can repair the heart—a critical step in stem cell research that could in the near future help millions of patients with heart failure.

The findings, published in the July 29 issue of Circulation Research, a journal of the American Heart Association, address an area that has been of enormous interest to cardiologists since the first suggestion that bone marrow-derived mesenchymal stem cells regenerate heart muscle damaged by a myocardial infarction (heart attack). Joshua M. Hare, director of the Interdisciplinary Stem Cell Institute at the Miller School, led the discovery, which settles several major controversies in the field and shows that the stem cells used can restore heart function back to normal very rapidly after heart attack.

The stem cell field has been plagued by a controversy surrounding exactly how bone marrow cells repair damaged hearts. In this study Hare’s group shows that the cells not only turn into new heart muscle themselves (acting as true stem cells) but also stimulate the body’s own cardiac stem cells to go into overdrive to contribute to the repair process. These findings settle one of the most controversial areas of medicine—why the heart’s own stem cells are not able to fully repair heart damage. By showing that the MSCs can stimulate the heart’s own cells, the researchers have shown that in fact the body can repair itself if given the right kind of help.

The research is of particular importance as these cells have already been tested for safety in patients with heart attack and are undergoing additional testing right now for patients with heart failure.

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Mauricio G. Cohen, associate professor of medicine

Percutaneous coronary intervention (PCI), more commonly known as angioplasty, is a widely used method of restoring blood flow to the heart. During those procedures, a certain amount of anticoagulation is necessary to prevent blood clots during the procedure. The challenge for physicians is that anticoagulants used for PCI often increase the risk of bleeding, especially at femoral access points in the thigh.

A multisite study led by a Miller School cardiologist has provided the first clinical application of a unique medication protocol consisting of an anticoagulant that thins the blood followed by a control agent that quickly reverses the process after the intervention.

Mauricio G. Cohen, associate professor of medicine in the Cardiovascular Division, led the Phase IIa study, which he calls “an important milestone in the search for safer anticoagulants for patients undergoing procedures in the catheterization lab.” The study results are published in the July 26 online issue of Circulation, a journal of the American Heart Association.

The study represented the first clinical application of REG1, a unique RNA drug system made up of an anticoagulant/blood thinner (RB006) and a control agent (RB007) to reverse anticoagulation. The REG1 system, developed by Regado Biosciences, Inc., is the first of its kind to enter clinical testing. This Phase IIa, multisite randomized study compared REG1 with unfractionated heparin during angioplasty.

The 26-patient trial was conducted between October 2007 and October 2008 at four U.S. sites and one in Argentina. The study indicated the use of the anticoagulant (RB006) provided a rapid and measurable response in patients. The control agent of REG1, RB007, which reverses the anticoagulation effect, was then used to control the step-down process. Cohen says the product can be administered according to the specific needs of the patient.

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Foundations of Translational Research “Bootcamp” is an intensive 40-hour course taught by University of Miami faculty that provides an introduction and foundation for clinical and translational research. Topics include an introduction to the tools of research, clinical study design, basic statistics, an update on molecular biology and genetics for the clinician, protection of human subjects, the how to’s of the IRB, scientific writing, and grantsmanship. All ACGME fellows, non-accredited fellows who will engage in research, research-intensive graduate students and medical students, postdoctoral fellows, new faculty, and other early career researchers are invited to attend. This learning opportunity will be offered September 20-24 at the Rosenstiel School of Marine and Atmospheric Science Auditorium, Key Biscayne Campus, and will be free unless the registrant fails to complete at least 90 percent of the coursework. Registration deadline is August 15. For more information and registration forms, click here.

Ian McNiece, left, Juan Pablo Zambrano, Alan Heldman, and Joshua M. Hare.

Stem cell researchers at the Miller School of Medicine have received a grant from the National Institutes of Health that will greatly accelerate their breakthrough findings in the arena of heart disease. The team, led by Joshua M. Hare, director of the Interdisciplinary Stem Cell Institute at the Miller School, has been awarded a $1 million grant from the National Heart, Lung and Blood Institute of the NIH to study cardiac stem cells.

With Hare, Alan Heldman, professor of medicine in the Cardiovascular Division, Juan Pablo Zambrano, associate professor of medicine in the Cardiovascular Division, and Ian McNiece, director of experimental and clinical therapies at the Interdisciplinary Stem Cell Institute, make up the rest of the team.

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The University of Miami Miller School of Medicine has been ranked the 25th best medical school in the country at meeting its social mission, according to the first study of its kind published in the June 15 issue of Annals of Internal Medicine.

Researchers from George Washington University defined social mission in three ways: the percentage of graduates practicing primary care, the number of graduates from underrepresented minorities, and the number working in areas with a shortage of health professionals. They then developed a metric called the social mission score to evaluate institutions in the three areas.

Three historically black colleges and universities with medical schools—Morehouse College, Meharry Medical College, and Howard University—had the highest social mission rankings. The Miller School finished first among Florida medical schools.

“The Miller School of Medicine is committed to a strong social mission that informs everything we do in medical education,” said Pascal J. Goldschmidt, senior vice president for medical affairs and dean. “Our medical student body is one of the most diverse of any medical school in the country. And from day one, our students learn the importance of taking care of the underserved, while being encouraged to pursue a career in primary care.”

The study looked at more than 60,000 physicians in active practice who graduated from one of 141 allopathic and osteopathic medical schools in the United States between 1999 and 2001. The researchers used data from the 2008 American Medical Association Physician Masterfile to calculate the percentage of graduates practicing primary care and located in areas with a shortage of health professionals.

The researchers wrote that “medical schools in the United States serve many functions, but one of their most basic purposes is to educate physicians to care for the national population.” The authors said institutions are falling short because of an insufficient number of primary care physicians, uneven geographic distribution of physicians, and low numbers of racial and ethnic minorities in medical schools and in practice.

The authors concluded that all medical schools “examine their educational commitment regarding the service needs of their states and the nation. A diverse, equitably distributed physician workforce with a strong primary care base is essential to achieve quality health care that is accessible and affordable, regardless of the nature of any future health care reform.”

Ralph Sacco, right, receives his AHA presidential pin from AHA past president Clyde W. Yancy during an induction ceremony in Dallas.

Miller School of Medicine Department of Neurology chair Ralph L. Sacco is the new president of the American Heart Association (AHA) for its 2010-11 fiscal year, beginning July 1. He became the first neurologist to hold the position when he was inducted into office during a ceremony in Dallas on June 22. As president, Sacco will be chief volunteer scientific and medical officer, responsible for medical, scientific, and public health matters.

“Clearly, one of my goals as president and as a neurologist is to broaden the organization’s focus on stroke, especially through the American Stroke Association, a vital part of the AHA,” Sacco said. “We’ll also continue to broaden our mission to build healthier lives free of cardiovascular diseases and stroke. With our nation’s constantly changing demographics, we not only want to reach out to all population groups through our programs, but also get more people from diverse backgrounds involved in our mission as volunteers.”

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James and Esther King and Bankhead-Coley grants help advance cancer research.

The battle against cancer being waged in numerous Miller School labs is getting a $12 million boost from the Florida Department of Health’s Biomedical Research Program grants supporting research in cancer and tobacco-related diseases. Twenty-three UM and Sylvester Comprehensive Cancer Center researchers collectively won about 27.5 percent – the largest share – of the $45.5 million in grants awarded through the James and Esther King Biomedical Research Program and the Bankhead-Coley Cancer Research Program.

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From left, William Donelan; Laz Pagan, chief nursing and operating officer for UMHC and Sylvester; David Zambrana, chief nursing and operations officer for UMMG; Hector Jimenez, medical director for University of Miami Hospital; Dean Pascal J. Goldschmidt; Fredrick Moffat, professor of surgery; William O'Neill; and Michele Chulick.

Miller School Dean Pascal J. Goldschmidt was named Business Leader of the Year in the hospital category by Business Leader Media at the Second Annual Business Leader of the Year Awards ceremony held at The Bankers Club in downtown Miami on June 14.

“It is often said that it takes a village to make progress and positive change,” said Dean Goldschmidt. “The same can be said for UHealth-University of Miami Health System. I am very proud of our team of dedicated professionals, who provide leading-edge health care to our South Florida community and beyond.”

Also in attendance were William O’Neill, executive dean for clinical affairs and chief medical officer, UHealth; William Donelan, vice president for medical affairs and chief operating and strategy officer; and Michele Chulick, associate vice president and executive director of clinical operations.

Dean Goldschmidt was selected from among three finalists in the hospital category and will be featured in the June 2010 issue of Business Leader Magazine (formerly South Florida CEO). Awards were given out in ten business sectors from a record number of nominations.

Omaida Velazquez

Omaida Velazquez, professor of surgery and chief of the Division of Vascular and Endovascular Surgery at the Miller School of Medicine, has been accepted into the 2010-11 class of the prestigious Hedwig van Ameringen Executive Leadership in Academic Medicine (ELAM) Program for Women at Drexel University College of Medicine in Philadelphia. ELAM is the only national program dedicated to preparing senior women faculty for leadership roles at academic health centers.

“It is an honor to be selected for participation in the ELAM program and I intend to represent the University of Miami Miller School of Medicine with complete dedication,” Velazquez said. “Through this outstanding program, I look forward to establishing new interpersonal networks and gaining important skills that will allow me to better address the unique challenges facing academic health centers today and thus better serve the missions of the DeWitt Daughtry Family Department of Surgery, the University of Miami Health System, and the Miller School of Medicine.”

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Sanjiv Bhatia, associate professor of clinical neurosurgery, and Mohammad Ali Aziz-Sultan, assistant professor of clinical neurological surgery, will present “Ischemic Cerebrovascular Disease in Children” on Tuesday, June 1 from 8 to 9 a.m. in the Mailman Center for Child Development, eighth-floor auditorium. For more information, please contact Javier Salazar at 305-585-6042 or jsalazar@med.miami.edu.

Eli Gilboa, Fernando Pastor, and Despina Kolonias.

Harnessing the immune system is emerging as one of the most promising new ways to fight cancer. Most cancer cells are eliminated by the immune system; however, over a lifetime, a few may escape this immune surveillance and lead to tumors and metastases. Hence a formidable opportunity has been to find ways to make the immune system recognize the tumor as a foreign body and trigger a response.

A team of researchers at the Sylvester Comprehensive Cancer Center at the University of Miami Miller School of Medicine overcame this challenge and developed an entirely new method to induce the expression of antigens (the proteins recognized by the immune system) on the surface of tumor cells, thereby spurring a much more robust immune response. The finding could lead to a new therapy for patients with most forms of cancer, even the more aggressive tumors, in the foreseeable future.

Eli Gilboa, Dodson Professor of Microbiology and Immunology and co-leader of the Tumor Immunology Program at Sylvester, led the study that has been published in the May 13 issue of the journal Nature. The research team consisted of Fernando Pastor, postdoctoral associate at Sylvester, Despina Kolonias, senior research associate at Sylvester, and Paloma Giangrande, assistant professor of internal medicine at the University of Iowa.

Pathogens such as bacteria and viruses elicit an immune response because they express antigens that are recognized as foreign. This response revs up the body’s natural defenses and eliminates the microorganisms. Because tumor cells are similar to normal cells in our bodies, they don’t express many foreign antigens. Therefore, they manage to escape the immune surveillance and grow unchecked to form cancers.

Gilboa’s team devised a method to force tumor cells to express new antigens on their surface, making tumors appear more like a “pathogen,” and consequently, to trigger a more potent immune response. “We’ve developed what could become an alternative to vaccines,” says Gilboa, “that would be simpler, broadly applicable, and potentially more effective.”

Normally, DNA information is converted to RNA, which in turn translates into a protein, the final product. During RNA synthesis there is a process known as nonsense mediated mRNA decay, which detects and eliminates defective RNAs. If a defect is present, the RNA is degraded and the protein is not formed. However, if the nonsense mediated mRNA process is not functional, the defective RNAs are not degraded and they will generate aberrant proteins, which will be viewed as foreign by the immune system and hence stimulate an immune response. With that in mind, Gilboa and his team focused on inhibiting the RNA degradation process, but only in tumor cells.

Specific proteins are present on the surface of tumor cells, such as PSMA on prostate cancer cells. Using nucleic acid technology, the researchers developed a small interfering RNA (siRNA), which inhibits the nonsense mediated mRNA decay process. The siRNA was then linked to a targeting ligand, also made of nucleic acid called “aptamer,” which binds to selected proteins present only on the surface of tumor cells. In this research, it bound to PSMA expressed on prostate tumors. Acting as a missile, the aptamer targets the siRNA to tumor cells and spares normal cells. In mice, it eliminated the tumor.

Joseph Rosenblatt, professor of medicine and interim director of Sylvester, says Gilboa has developed a “very clever way of embracing the body’s immune response” and his research “represents a completely new approach to immunotherapy of cancer.”

Pascal J. Goldschmidt, senior vice president for medical affairs and dean of the Miller School of Medicine, describes the work as “groundbreaking and possibly a brand new opportunity for the millions of patients who are victims of cancers.”

Gilboa is the Dodson Professor of Microbiology and Immunology thanks to an extraordinary gift from Eugenia Dodson following her death in 2006. Because of her battle with lung cancer, she dedicated one-third of her $35.6 million estate to be used for cure-focused cancer research at Sylvester.

Gilboa, who worked in vitro and with mice, describes his findings as “a potentially significant discovery toward a new therapy.” The next step, he believes, is clinical trials at Sylvester Comprehensive Cancer Center. Gilboa says they would likely start with prostate cancer since the reagents are available, but adds that breast cancer expressing HER2 would be another candidate.

As a radio reporter recorded, Superintendent Alberto Carvalho talked to a patient at a nearby clinic. He is surrounded by, clockwise from left, Joycelyn Lawrence, Anne Burdick, Kim Greene, of the Dr. John T. Macdonald Foundation, and Arthur Fournier.

Late last Tuesday afternoon, when Anne Burdick examined the right foot of a kindergartener with a history of eczema, she could clearly see the girl’s skin was dry and leathery from constant scratching.

While the exam was nothing out of the ordinary for the professor of dermatology, the video-conferencing mode by which Burdick conducted it was – as it ushered in a new era of convenient, cost-reducing, health-promoting telehealth in the Miami-Dade County Public Schools.

Burdick, associate dean for telehealth and clinical outreach, was in the school clinic at North Miami Beach Senior High, and the 5-year-old girl was more than a mile away at Greynolds Park Elementary. The two were connected by a high-definition video conferencing system and were viewing each other via two wide-screen monitors.

“The images were very clear – diagnosis quality,” said Burdick. “It was as good as seeing it in person.”

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With support from close to $90 million in stimulus-backed grant awards—and counting—from various federal agencies, University of Miami investigators have embarked on research projects aimed at finding solutions to some of the world’s most pressing problems.

Stimulus-backed research is being conducted on UM's Coral Gables, Miller School, and Rosenstiel School campuses.

The bioreactor that will enable Weiyong Gu to analyze the growth characteristics of intervertebral tissue without having to remove samples from the device hasn’t even been invented yet. But the University of Miami biomedical engineer is in a race against time, working long hours in his lab to build the instrument that could help pave the way for advanced techniques in the engineering of human tissue to replace organs.

At UM’s Rosenstiel School of Marine and Atmospheric Science, the challenge that confronts geochemist Peter Swart lies not in building a new device, but in using existing tools to determine whether the technique of carbon capture can actually help solve the global-warming crisis.

Both investigators are conducting their research with the blessing and backing of Uncle Sam. They are beneficiaries of hefty grants that have been flowing from the federal government’s coffers ever since the American Recovery and Reinvestment Act (ARRA) pumped $10.4 billion into the National Institutes of Health, with $8.2 billion earmarked for scientific research priorities.

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