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Translational Sciences Center Assists 218 Researchers in Inaugural Year
Center helps speed transformation of basic research into drugs, treatments or methods for prevention
Mitochondrial disease researcher Mary Kay Koenig, M.D., was one of 218 scientists assisted during the inaugural year of a program to fast track the application of scientific knowledge to patient care at The University of Texas Health Science Center at Houston.
The program is called the Center for Clinical and Translational Sciences (CCTS), a collaboration among the UT Health Science Center at Houston, the UT M. D. Anderson Cancer Center, and the Memorial Hermann Healthcare System. It is funded with a five-year, $36 million Clinical and Translational Science Award from the National Institutes of Health (NIH).
With administrative offices at the UT Health Science Center, the center is providing investigators like Koenig – who proposes to study a new treatment that could save the lives of children with a rare metabolic disorder – with help in many aspects of patient-centered research, including project design, human subject issues and budgets.
As part of a national initiative to transform the medical research process led by NIH Director Elias A. Zerhouni, M.D., the NIH established translational science centers at the UT Health Science Center and 11 other academic health centers in October 2006. Plans call for translational centers at up to 48 additional academic health centers.
“From the time a concept is spawned to its implementation can take 10 to 12 years,” said Pablo Okhuysen, M.D., co-director of the CCTS program and professor of medicine at the UT Medical School at Houston. “We’re trying to identify the obstacles that slow the transformation of basic research discoveries into drugs, treatments or methods for prevention.”
The CCTS provides mentors who assist young investigators through the regulatory process and offer help with bioethical issues. The center provides the statistical support necessary to manage and analyze complex multi-variable projects. There also is a career development component.
“We want to make research more efficient, more patient-oriented and more preemptive,” Okhuysen said. “The final stage of translation is to get the research findings implemented in day-to-day patient care.”
Divided into 12 components that support different aspects of the discovery process, the CCTS also funds innovative clinical and translational projects proposed by faculty and students both at the UT Health Science Center and other institutions.
“We pick people with novel ideas,” said Frank C. Arnett Jr., M.D., principal investigator, director of the CCTS and the holder of the Elizabeth Bidgood Chair in Rheumatology at the Medical School. “We’re looking for innovation in proposals.”
Koenig receives salary and research funding through the CCTS K12 program, which supports medical fellows and assistant professors. She believes she can slow the progression of mitochondrial disease, a hereditary condition affecting one in every 5,000 babies, through dietary changes. She’s testing the impact of a modified high-fat, low-carbohydrate diet in the lab and hopes to begin patient trials soon.
When she’s not in the laboratory, Koenig, an assistant professor of child and adolescent neurology at the Medical School, oversees the Mitochondrial Clinic, where she diagnoses and treats children with the disease. In addition, she’s the medical liaison for the Houston Chapter of the United Mitochondrial Disease Foundation, which provides parent support and participates in fundraising for children afflicted with mitochondrial diseases.
Another physician-scientist in the K12 program is Nicole R. Gonzales, M.D., an assistant professor of neurology at the Medical School. She’s about to begin patient trials on a medication she hopes will reduce the secondary injury caused by intracerebral hemorrhagic stroke, the type caused when a blood vessel ruptures.
“I’m a new investigator, and the CCTS program has allowed me to get my research off to a quick start,” said Gonzales, who is a member of the UT Stroke Team. “We have access to a lot of research expertise up front, which allows a new investigator to be efficient in planning and implementing a trial.”
Support from the CCTS is allowing Kenneth Helmer, M.D., assistant professor of surgery, to explore the anti-inflammatory effects of ketamine in repair of aortic aneurysms in the chest and abdominal cavities. “Ultimately, I would like to carry this over to trauma patients. If ketamine inhibits the systemic inflammation and alleviates resultant multi-organ dysfunction, we could save lives,” Helmer said.
The CCTS also supports pilot projects, including a study by Nehal A. Parikh, D.O., an assistant professor of neonatal-perinatal medicine, titled “Evaluation of Diffusion Tensor Magnetic Resonance Imaging (DTI) as a Predictor of Neurosensory Impairments in Extremely Preterm Infants.”
“This pilot CCTS grant has allowed us to benefit from the excellent research MRI facilities at the UT Medical School,” Parikh said. “We have imaged several extremely preterm infants with diffusion tensor and volumetric MRI in hopes of understanding the encephalopathy (brain disorders) associated with preterm birth.”
Mary Ruppe, M.D., assistant professor in the Division of Endocrinology, has received both pilot funding and K12 support to jump start her studies of X-linked hypophosphatemic rickets, a genetic disease that affects one in every 20,000 live births.
“It’s exciting. I have already recruited 42 pediatric patients and completed the first round of testing,” Ruppe said. “We want to understand the path-ways better so we can create better treatments in order to prevent some of the long term complications of the disease.”
Ruppe said she is taking full advantage of a host of CCTS services, including genetic profiling, microarray analyses, database development and statistical analysis.
Clinical Research Units
Patient trials can generate volumes of paperwork for investigators. They also can involve time-consuming follow-up tests, including blood work, vital signs and a battery of other technical procedures.
The CCTS facilitates research at three Clinical Research Units (CRUs), which provide these and other services to investigators. Staffed by experienced research nurses and phlebotomists, the CRUs also have room for on-site medical record storage. The CRUs are located at Memorial Hermann Hospital - Texas Medical Center, M. D. Anderson Cancer Center and Valley Baptist Hospital in Brownsville.
Researchers also can utilize the services of the Genetics Core Lab, which provides DNA harvesting and banking, DNA genotyping and DNA sequencing. Under the direction of Dianna Milewicz, M.D., Ph.D., the staff has the expertise to collect, analyze and store data from biological materials. Milewicz holds the President George Bush Chair in Cardiovascular Medicine at the Medical School and UT Graduate School of Biomedical Sciences at Houston (GSBS)
Other services include: the Microarray Core Lab, the Proteomics Core Lab, the Quantitative Genomics Core Lab, the Laboratory for Developmental Biology, the Image Core Lab, and an Immune Monitoring Imaging Laboratory.
Collaboration is an integral part of the CCTS and involves teamwork both between researchers and between institutions, Arnett said.
Arnett likens researchers to silos in that they are good at storing information but not necessarily good at sharing it. He’s trying to “desilosize” the research process by scheduling meetings where investigators can share ideas and discuss possible collaborations.
“To me, this is one of strongest areas of the CCTS,” said Sandeep Agarwal, M.D., Ph.D., assistant professor in the Division of Rheumatology. “The center is offering wonderful opportunities to bring scientists together for collaboration – scientists who normally wouldn’t meet.”
At a recent CCTS dinner for new investigators, Agarwal discovered numerous opportunities for collaborations. He learned about complementary work Jayasimha Murthy, M.D., assistant professor in the Division of Pulmonology and Critical Care Medicine, is conducting four floors down from his own laboratory. Agarwal also learned about research being conducted at M. D. Anderson that could be beneficial to his work in the search for an effective treatment for scleroderma.
“We’ve spent zillions on research that has led to very important basic discoveries. But we now want to become more efficient in utilizing this information quicker to improve health,” George M. Stancel, Ph.D., GSBS dean, told a recent gathering of CCTS investigators. “Groups like this will help us do exactly that by thinking how we are going to pull all this information together and apply it more quickly than in the past.”
UT Health Science Center at Houston researchers also are collaborating with their counterparts at the M. D. Anderson Cancer Center on a new way to detect melanoma, the most serious type of skin cancer, which will cause most of the 10,850 deaths due to skin cancer this year. Razelle Kurzrock, M.D., professor of medicine at M. D. Anderson and GSBS, is a co-director of the CCTS program.
Nancy Murray, Dr.P.H., co-director of the community engagement component of CCTS and assistant professor of behavioral sciences at the UT School of Public Health, has organized a 90-person community advisory board that includes Houston Chronicle Science Reporter Eric Berger to provide input on science education initiatives.
The board met and concluded that obesity and diabetes were the main health problems with which the CCTS could help. To this end, the component has created a quarterly lecture series on cutting-edge CCTS research for science teachers and an initial 56 podcasts (in both English and Spanish) on health and research for the public.
CCTS research for science teachers and an initial 56 podcasts (in both English and Spanish) on health and research for the public.
Next spring, CCTS moves into its new administrative offices on the 11th floor of the UT Professional Building, 6410 Fannin, across the street from the UT Medical School.
Maureen Goode, Ph.D., administrative director of the CCTS, said the move to the UT Professional Building will further streamline the process of clinical and translational research. “Our consultative services will be located in one area. With just a quick walk down the hall, we’ll be able to immediately bring together resources, solve problems and keep the research moving in the right direction. The ultimate goal is to accelerate discovery and improve people’s health.”
For more information on the CCTS and its services call 713-500-7900.
By Rob Cahill, Institutional Advancement
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