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Bearings - Winter 2012

Children’s Research Institute Leads in Developing New Treatments for Children

Children’s National Medical Center is known throughout the Washington metropolitan region and across the country as a world-class children’s hospital – a destination for families seeking the highest quality care for their children. Children’s National is also quickly building a reputation for cutting edge pediatric research that takes place within the research arm of the hospital: Children’s Research Institute (CRI).

CRI conducts and promotes translational and clinical medical research and education programs that lead to improved understanding, prevention, treatment, and care of childhood diseases. The institute is divided into five specialty research centers:

There are more than 450 federally and privately funded research projects underway at Children’s National, led by more than 130 faculty members. Children’s junior faculty collectively has 26 National Institutes of Health (NIH) awards, which are prestigious grants to engage future leaders in translational research and medicine.

The research conducted at Children’s National is highly collaborative and multi-disciplinary with laboratories and clinical divisions working together to answer questions about childhood diseases. The research space is located atop the Children's National Medical Center - Sheikh Zayed Campus for Advanced Children's Medicine building, which creates an environment where scientists and physicians can more easily collaborate on innovative projects that will improve patient care.

When it first opened in 1990, CRI research space totaled 40,000 square feet. Through multiple expansions, the research space has grown to 100,000 square feet.

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The Sheikh Zayed Institute for Pediatric Surgical Innovation

Launched in September 2009, the Sheikh Zayed Institute for Pediatric Surgical Innovation (the Institute) at Children’s National Medical Center is redefining what is possible in surgery through innovative, integrated research.

Despite tremendous medical advances over the past few decades, surgery can still be a difficult experience for children and their families. The goal of the Institute is to make pediatric surgical care more precise, less invasive, and less painful. By combining research and clinical work in this area, the Institute is developing knowledge, tools, and procedures that will benefit children in the Washington region, across the country, and around the world.

The Institute brings together pediatric surgeons, anesthesiologists, and researchers to improve children’s lives before, during, and after surgery. The Institute focuses on four initiatives that together will open a new era for pediatric surgery: pain, immunology, bioengineering and personalized medicine.

Pain is one of the most challenging and misunderstood issues in pediatric care — due in part to the difficulty of measuring it in newborns and young patients who cannot effectively communicate their experience. The Pain Medicine Initiative brings us closer to a solution through its focus on the development of a novel device capable of providing an objective measure of a child’s pain. Once Institute researchers can measure pain accurately, they will be able to begin to eliminate it with more effective medications and treatments. At the same time, they will work to unravel the genetic markers that show how a child will react to existing medications for optimal and safe results. The benefit to children will be improved surgical outcomes through the elimination of needless pain and suffering.

Immunology researchers want to use a child’s own immune system to fight disease, especially the recurrence of cancers after initial surgical intervention; Bioengineering researchers are building new ways to visualize surgery and use robotics to enhance precision and accuracy; and finally the Personalized Medicine program wishes to tailor treatment for every child based on their own genetic makeup to optimize outcomes.

The four initiatives of the Institute are unique and critical to the transformation of pediatric surgery. None of these initiatives, however, can be transformational on its own. It is the synergies and collaborations among these initiatives that will facilitate meaningful discoveries, create an environment where the best physician-scientists can thrive, and solve the key challenges of the field. To successfully realize the development of new ideas and set the stage for new standards of performance, the Institute is implementing principles of innovation management borrowed from successful business models — focused leadership; defined tools and processes for selecting promising research paths, as well as tracking and measuring advances; all aimed at fostering creativity, discovery, and invention. Applying these principles across all areas, with a sense of urgency and a common goal, the Institute’s physician-scientists are re-imagining surgery to allow children to live longer, healthier lives.

In August, Children’s National announced the appointment of Peter C.W. Kim, MD, CM, PhD, as vice president of the Institute. An internationally known pediatric surgeon and scientist, Dr. Kim will implement the Institute’s vision of innovative, multidisciplinary research and development in pediatric surgery.

“The Sheikh Zayed Institute creates unprecedented opportunity to make fundamental differences in pediatric surgery, in real time, and truly affect the well being of children worldwide,” said Dr. Kim. “The Institute’s model creates an ecosystem where care, education, and research work simultaneously together.”

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The Center for Genetic Medicine Research

The Center for Genetic Medicine Research investigates, diagnoses, and treats genetic disorders--diseases and conditions in children that are caused by inherited genes that do not function normally. Founded in 1999, the Center emphasizes translational research (bench-to-bedside) and harnesses emerging technologies for genetic and proteomic analysis. Approximately 150 scientists (including 48 faculty) stress open, collaborative, multidisciplinary research.

Center Director Eric P. Hoffman, PhD, leads a collaborative research team that works with clinical and research programs throughout Children’s National to investigate some of the most challenging pediatric disorders, including:

“We have had some major successes in attracting large federally-funded research projects,” said Dr. Hoffman of recent work in his Center. “We are one of four NIH Centers of Pediatric Pharmacology, one of two children’s hospitals in the new national neurology clinic trail network, and an initial awardee from the new NIH Therapeutics for Rare and Neglected Diseases (TRND) program.”

Drug development and personalized medicine has become an increasing focus of the Center’s cutting edge research. One drug development program involves using small pieces of DNA as drugs to correct a patient’s gene mutation. The NIH and Department of Defense have recently provided $25 million in drug development for the personalized medicine drug development programs for muscular dystrophy patients, and these efforts are being used as a model for many other disorders.

Dr. Hoffman leads one of the largest muscular dystrophy research groups worldwide. He is credited with discovering the cause of the most common type of muscular dystrophy (dystrophin deficiency). Dr. Hoffman’s work was featured in Parade Magazine on August 30, 2009.

The Center has recently spun off a Montgomery County-based start-up company, ReveraGen BioPharma, Inc. This biopharmaceutical company has developed a potential replacement for the commonly-used glucocorticoids (steroids), with loss of side effects.  Their lead compound, VBP15, is a novel dissociate glucocorticoid compound where the first application is for the treatment of Duchenne muscular dystrophy (DMD).

Edward Connor, MD, serves as interim CEO of ReveraGen. Dr. Connor is Director of the Office of Investigational Therapeutics at Children’s National and Professor of Pediatrics at the George Washington University School of Medicine and Health Science in Washington, DC. He was most recently Executive Vice President & Chief Medical Officer at MedImmune, one of the nation’s leading biotechnology companies. Dr. Hoffman is a founder and member of ReveraGen’s management team.

In August 2011, ReveraGen’s VBP15 received a grant from the TRND program to partner on the development of a new treatment for DMD. The TRND program is a new initiative for NIH to assist in the development of new drugs for rare and neglected diseases. To develop new medicines, TRND establishes partnerships with academic, government, biopharmaceutical, and patient advocacy groups to focus on the discovery, optimization, and pre-clinical testing of new drugs. VBP15 was selected through a national competitive process as one of a select few inaugural compounds in the TRND program.

“This partnership with NIH/TRND provides ReveraGen with the opportunity to facilitate the next steps in the research and development of VBP15,” said Dr. Connor. “Public-private partnerships are an important way of advancing therapeutics in orphan diseases and the team at TRND provides significant experience in orphan drug development.”

“Glucocorticoids are important in the treatment of many diseases, yet their broad mechanism of action has been challenging to refine to more targeted drugs,” offered Dr. Peter Kim, director of the Institute. “The pioneering work that the ReveraGen staff has been doing on these dissociative glucocorticoids is the type of innovative research we foster at the Sheikh Zayed Institute. We look forward to seeing the work that comes from ReveraGen’s partnership with TRND.”

Other advances in translational research in the Center include new diagnostic methods utilizing next-generation DNA sequencing, and development of personalized early interventions for common problems of children in the region, such as asthma and obesity. The Clark family, whose family-owned construction company is headquartered in Bethesda, recently donated $100,000 to initiate childhood obesity interventions, through the Research Center for Genetic Medicine in collaboration with George Washington University.

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