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Department of Pediatrics : Research : Hematology & Oncology

Darby Children’s Research Institute

The Darby Children's Research Institute (DCRI) opened in February 2005 as the largest and most comprehensive pediatric research facility in the Carolinas.  Its mission is to improve the lives of children, their families and communities by conducting high quality children’s research, training superior physicians and scientists, and fostering innovation through the sciences of discovery and application.  The 122,000 ft2 7-story building has 150 state-of-the-art laboratory modules providing bench space and core facilities for researchers in 11 multidisciplinary programs, representing many departments and colleges at MUSC. The open lab design and shared research space foster interaction, communication, and collaboration. Enclosed walkways connecting every floor between the DCRI and the adjoining Basic Sciences Building promote intra- and inter-programmatic synergies, along with sharing of high technology resources. The overall goals of the DCRI are to empower talented scientists, promote inclusiveness and collaboration, enable established and new investigators, and increase and diversify funding for pediatric research. The DCRI currently houses basic and translational investigators in cardiobiology, neurosciences, cancer biology, pharmacogenetics, addiction research, pulmonary biology, proteomics, vitamin D metabolism, osteoclast biology, autoimmune and rheumatic diseases, and renal biology.

Center for Gene, Cell and Vaccine Therapy
Director: David Cole, MD

The purpose of the MUSC Center for Gene, Cell and Vaccine Therapy is to provide a basic-preclinical-translational structure that supports the transition of laboratory-based investigation to preclinical Phase I research in humans. In the long term, the Center is expected to achieve national prominence in gene and vaccine therapy and develop an organizational structure for faculty achievement and expanded opportunity to compete for NIH research funds and industry support. Areas of scientific endeavor include cancer gene therapy with viral-based vectors, cancer therapies making use of immune cells, vaccine technologies to enhance innate and adaptive immune systems, and immunotherapies against infectious diseases. The Center utilizes a variety of existing resources, including the Hollings Cancer Center Cell Sorting Facility and Viral Vector Core Facility, and facilitates the development of new ones, such as the Clean Room Facility to support cell isolation and processing and vaccine development and a BSL-3 laboratory with an adjoining BSL-3 animal holding area. The Center allows for joint venture relationships with biotech companies and provides better access to the National Gene Vector Laboratories. A current objective is developing a resource to help basic scientists and participating physicians to translate their data into IND applications as investigator-initiated Phase I clinical trials.

Hollings Cancer Center 

The Hollings Cancer Center (HCC) at the Medical University of South Carolina (MUSC) is the one of the nation's newest NCI designated cancer centers, receiving its Cancer Center Support Grant in April 2009. Located in Charleston, South Carolina, the HCC was founded in 1993 and serves the state as a leader in cancer research, patient care and public and professional education. Since its opening in 1993, the Center has had significant growth in clinical and research facilities, patient care volume, research funding and in its clinical trials portfolio and accrual. Last year, the HCC had more than 80,000 outpatient visits, $33M in cancer research extramural funding, and more than 1,100 patients enrolled in cancer research studies. To accommodate the expanding clinical activities and provide additional space for the growth of basic and translational research, MUSC added a 7–story tower annex to its original HCC Building in 2006, bringing total dedicated MUSC cancer space to more than 200,000 ft2 (including 98,000 ft2 of research laboratories). 

The goals of the HCC are to:

  1. Create an environment where cancer research will be fostered from basic concept to clinical/community intervention;
  2. Develop epidemiologic, environmental, and behavioral research to reduce the cancer burden and disparities in a rural, underserved population;
  3. Train the next generation of basic clinical and prevention researchers;
  4. Lead the coordination of state–wide efforts in research, education and clinical care; and
  5. Obtain National Cancer Institute "comprehensive" status.

Toward these goals, the HCC has committed significant resources to:

  1. Recruiting and supporting basic, clinical and population–based investigators who can obtain and maintain investigator–initiated research grants from the NCI;
  2. Developing shared research resources to provide access to critical instrumentation and technologies; and
  3. Enhancing laboratory and other types of space dedicated to cancer–related activities.

HCC research membership includes more than 100 faculty researchers representing 23 departments in multiple colleges. Formal research programs include Lipid Signaling in Cancer, Cancer Genes & Molecular Regulation, Developmental Cancer Therapeutics, Cancer Immunology, and Cancer Prevention & Control. Future recruitments will be housed in two new buildings, the Drug Discovery Building and the Cancer Genomics and Bioengineering Building, to be completed in 2011 and 2012 respectively. HCC supports a robust array of shared research resources including the following:

  • Biostatistics
  • Clinical Trials
  • Flow Cytometry and Cell Sorting
  • Lipidomics
  • Cell and Molecular Imaging
  • Tissue Biorepository
  • Gene Knockout
  • Small Animal Imaging
  • Drug Discovery/Screening
  • Cellular Therapy/Immunology Monitoring
  • Drug Metabolism and Clinical Pharmacology

South Carolina Clinical and Translational Research Center

The SCTR SUCCESS Center, in collaboration with the Office of the Associate Provost for Research, has developed a new online guide, the “Research Toolkit,” to assist MUSC research personnel in navigating the research enterprise. The Toolkit addresses the steps involved with submitting, conducting, closing and disseminating results of a research study, and includes links to institutional, state, and federal resources and regulations.   

NIH Clinical and Translational Science Award  

MUSC joins 39 previously funded academic medical research institutions within a national network working together to reduce the time it takes to turn laboratory discoveries into treatments for patients, to engage communities in clinical research efforts, and to train the next generation of researchers. MUSC has been awarded the coveted Clinical Translational Science Award (CTSA) from the National Center for Research Resources, a division of the National Institutes of Health (NIH).

COBRE in Lipidomics and Pathobiology
Principal Investigator: Lina M. Obeid, MD
 Funding: National Center for Research Resources (NCRR)

The Center for Biomedical Research Excellence (COBRE) is a special P20 mechanism introduced by the National Center for Research Resources (NCRR/NIH) in 2000 to develop emerging excellence in thematic research areas through targeted mentoring of new investigators and enhancement of institutional research infrastructure in terms of access to scientific core resources and technologies. The COBRE in Lipidomics and Pathobiology supports MUSC investigators in their efforts to understand the role of a class of fatty molecules, known as sphingolipids, in regulating cell growth, cell death, and cell aging. Explaining the functions of this abundant class of molecules has been an enigma for scientists, hence the name derived from the Sphinx of Greek mythology. The members of the COBRE group at MUSC have been at the frontiers of developing an understanding of how these molecules participate in regulating several fundamental aspects of human cell function and how they contribute to the development of a wide range of human diseases, especially cancer, aging, neurologic disease and fungal pathogenesis. The COBRE in Lipidomics and Pathobiology successfully competed for a 5-year renewal grant in 2007, based on substantial accomplishments during the first phase. Achievements during the initial project period include mentoring and retention of all 10 targeted junior faculty investigators, participation by more than 25 MUSC research faculty members in COBRE programs, 239 publications by COBRE participants in peer-review journals (63 papers plus 61 oral and poster presentations by the mentored investigators), and development of highly successful scientific core resources, including a truly unique Lipidomics Core for synthesis and analysis of bioactive lipids. This COBRE has also been very successful in recruiting under-represented minority (3) and women (6) investigators. The scientific focus of the COBRE is on developing the field of lipidomics and studying the functional involvement of bioactive lipids in clinically relevant disorders and disease states, including cancer progression and metastasis, accelerated aging, inflammation, and fungal pathogenesis of infectious diseases. 

Translational Pediatric Oncology Research

As a Principal Investigator in the Center for Childhood Cancer and Blood Disorders in the Darby Children’s Research Institute, Dr. Kraveka’s research focuses on sphingolipid mediated signal transduction in pediatric malignances and the function and regulation of the enzyme dihydroceramide desaturase in human cancer. The long-term goals of Dr. Kraveka’a research are to develop novel therapeutic strategies for the treatment of neuroblastoma, medulloblastoma and leukemia.