Discoveries In Genetics, Stem Cell Therapies And New Treatments For Childhood Diseases Explored By World Community Of Pediatricians

Main Category: Pediatrics / Children's Health
Also Included In: Aid / Disasters;  Genetics;  Acid Reflux / GERD
Article Date: 10 Nov 2008 - 1:00 PDT

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On Nov. 8 and 9, Morgan Stanley Children's Hospital of NewYork-Presbyterian and Columbia University Medical Center hosted an "Innovations in Pediatric Medicine" conference at the Grand Hyatt New York, which featured lectures by international leading authorities in pediatric biomedical research, genetic findings and stem cell therapy breakthroughs.

Key topics included discoveries about congenital and primary immunodeficiencies; gene therapy in children; and the genetic basis for common childhood infections. In addition, there was a unique presentation on pediatric emergency care during disasters and the lessons learned from Hurricane Marilyn on St. Thomas; the 2001 attack on the World Trade Center in New York; the 2003 earthquake in Bam, Iran; and Hurricane Katrina.

"Medical breakthroughs have greatly increased the range of treatment options for pediatric diseases, making it vital to bring together medical professionals who are on the frontline of pediatric care for this opportunity to learn the latest progress and to share best practices," said the conference's course director, Dr. Mitchell Cairo, director of pediatric blood and marrow transplantation at Morgan Stanley Children's Hospital of NewYork-Presbyterian and professor of pediatrics, medicine and pathology at Columbia University College of Physicians and Surgeons.

Listed below are some key presentations by leaders in their field:

• Dr. Alain Fischer of Descartes University Hospital Necker-Enfants Malades, Paris, France, discussed gene therapy for inherited disorders based on research on the treatment of severe combined immunodeficiency. Introducing genes into bone marrow stem cells led to sustained correction of the disease for almost 10 years, providing evidence that the approach can be effective and could be used to treat other genetic diseases of blood cells. One challenge is the viral vector used to introduce the gene has been linked to cancer. In response, new vectors are being designed.
  
  
• Dr. Gary Fleisher of the Children's Hospital Boston and Harvard Medical School presented a framework for planning for disaster management, highlighting unique pediatric aspects. He discussed the conditions likely to be encountered by providers arriving in the first 24 to 72 hours and the skills necessary for success. In terms of response teams, Dr. Fleisher described the structure of PST-1 (Pediatric Specialty Team-1), the first team developed by the NDMS (National Disaster Medical System) dedicated to treating children in disasters. He shareed lessons learned from Hurricane Marilyn on St. Thomas; the 2001 attack on the World Trade Center in New York; the 2003 earthquake in Bam, Iran; and Hurricane Katrina.
  
  
• Dr. Margaret K. Hostetter of Yale-New Haven Children's Hospital presented on advances in the genetic origins of common childhood infections, focusing on newly discovered genes that are linked to early onset staphylococcal infection, recurrent pneumococcal infections, and rarer disorders such as Bruton's agammaglobulinemia, hyper IgE syndrome, chronic granulomatous disease and severe combined immunodeficiency.
  
  
• Dr. Jennifer M. Puck of the University of California, San Francisco, presented evidence that early diagnosis of primary immunodeficiencies is critical for optimal treatment. The challenge is that these disorders are rare and hard detect until serious complications have developed; a life-threatening situation usually has to occur before a correct diagnosis was made. As a solution, Dr. Puck suggests that all newborns be screened for severe combined immunodeficiencies, with the goal of improving timely diagnosis and outcomes. Her laboratory has developed a screening test that can be done on the dried blood spots routinely used for screening for other serious conditions.

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Columbia University Medical Center

Columbia University Medical Center provides international leadership in basic, pre-clinical and clinical research, in medical and health sciences education, and in patient care. The medical center trains future leaders and includes the dedicated work of many physicians, scientists, public health professionals, dentists, and nurses at the College of Physicians & Surgeons, the Mailman School of Public Health, the College of Dental Medicine, the School of Nursing, the biomedical departments of the Graduate School of Arts and Sciences, and allied research centers and institutions. Established in 1767, Columbia's College of Physicians & Surgeons was the first institution in the country to grant the M.D. degree and is now among the most selective medical schools in the country. Columbia University Medical Center is home to the largest medical research enterprise in New York City and state and one of the largest in the United States. For more information, please visit http://www.cumc.columbia.edu.

Morgan Stanley Children's Hospital of NewYork-Presbyterian

Ranked by U.S.News & World Report as one of the top children's hospitals in the country, Morgan Stanley Children's Hospital of NewYork-Presbyterian offers the best available care in every area of pediatrics -- including the most complex neonatal and critical care, and all areas of pediatric subspecialties -- in a family-friendly and technologically advanced setting. Building a reputation for more than a century as one of the nation's premier children's hospitals, Morgan Stanley Children's Hospital of NewYork-Presbyterian is affiliated with Columbia University College of Physicians and Surgeons, and is Manhattan's only hospital dedicated solely to the care of children and the largest provider of children's health services in the tri-state area with a long-standing commitment to its community. Morgan Stanley Children's Hospital of NewYork-Presbyterian is also a major international referral center, meeting the special needs of children from infancy through adolescence worldwide. For more information, visit http://www.nyp.org/.

Source: Belinda Mager
New York- Presbyterian Hospital/Columbia University Medical Center

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