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2014

Research Consortium to Receive Largest-Ever Federal Contract for Primary Immune Deficiency Research

$12.8 Million NIH Contract to Fund Groundbreaking Research with Potential

Applications Beyond Primary Immune Deficiency Diseases



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    TOWSON, Md., Oct. 3 /PRNewswire/ -- An international research network
 investigating life-threatening primary immune deficiency diseases will receive
 the largest federal award ever to study the diseases, according to an
 announcement today from the National Institute of Allergy and Infectious
 Diseases (NIAID), one of the National Institutes of Health (NIH), which is an
 agency of the US Department of Health and Human Services.  The Immune
 Deficiency Foundation (IDF) - a national non-profit health organization
 dedicated to improving the diagnosis and treatment of primary immune
 deficiency diseases - will support the US Immunodeficiency Network (USID Net),
 a consortium of leading researchers who will develop and oversee an ambitious
 and comprehensive research agenda and distribute funds to individual
 researchers totaling nearly $12.8 million over the next five years.  USID Net
 will be jointly sponsored by NIAID and the National Institute of Child Health
 and Human Development (NICHD).
     Primary immune deficiency disorders are caused by intrinsic or genetic
 defects in the immune system.  The result is an immune system that is either
 missing entirely or does not function properly.  The World Health Organization
 recognizes more than 100 of these disorders.  As many as 50,000 people in the
 U.S. have a clinically significant primary immune deficiency disease.
 
     New Funding will Promote New Treatments
     The research will focus on new investigative methods that show promise for
 the treatment of primary immune deficiency diseases, including:
 
     --    Broad analysis of the molecular and genetic basis of primary immune
           deficiencies
     --    Evaluation of proteins that are of crucial importance for the
           function of the immune system
     --    Design of screening tests for early diagnosis of primary immune
           deficiencies
     --    Investigate new molecularly based treatments that may lead to the
           cure of primary immune deficiencies
 
     In addition to evaluating and funding research projects, the consortium
 will mentor physicians and scientists to foster long-term interest in the
 fields of primary immune deficiency and also establish a collection of cell
 lines and DNA, which will be made available to qualified investigators to
 investigate aspects of the primary immune deficiency diseases.
 
     Far-Reaching Benefits for Other Conditions
     In addition to its direct benefit for the primary immune deficiency
 diseases community, the contract announced today could have far-reaching
 advantages for others.  Research initiated with primary immune deficiency
 patients often is the source for basic science breakthroughs and has given
 rise to innovative treatments in a wide range of disease areas.  Well-known
 examples include:
 
     --    Bone marrow transplantation - the first successful treatment of a
           disease by bone marrow transplantation involved a child with X-
           linked Severe Combined Immune Deficiency (SCID) in 1968.  Bone
           marrow transplantation is now a life-saving treatment for thousands
           of patients each year with disorders ranging from primary immune
           deficiency diseases to cancer to serious red blood cell disorders or
           inherited metabolic disorders.
 
     --    Systemic enzyme replacement therapy - the administration of missing
           enzymes to perform critical functions were first introduced as a
           treatment for a form of severe immunodeficiency resulting from
           absence of the enzyme adenosine deaminase (ADA-SCID).
 
     --    Gene therapy - an NIH team of researchers first administered gene
           therapy to primary immune deficiency disorder patients in 1990.
           Since then, international research teams have applied gene therapy
           to these diseases and continue to study further uses.
 
     --    Autoimmune diseases - The discovery that autoimmune diseases are
           often caused by Immune dysregulation has provided new therapeutic
           strategies using potent drugs that down regulate the immune system.
           These new drugs have been most successful in treating rheumatoid
           arthritis, Crohn's Disease, psoriasis and others.
 
     "Today's announcement is an unprecedented commitment to understanding and
 one day curing these debilitating diseases and recognizes that research
 conducted in the primary immune deficiency community has applications for
 patients suffering from other serious ailments," said Principal Investigator
 Hans Ochs, M.D.  "We believe this commitment by NIAID and NICHD will help us
 to take the lead in scientific discovery."
     The consortium will be administered by the Immune Deficiency Foundation
 and includes world-renowned immunologists and researchers across the country
 and around the globe.  IDF was founded over two decades ago to help
 researchers develop treatments and, potentially, cures for primary immune
 deficiency diseases.  The IDF works to advance research, advocacy and
 education for the primary immune deficiency diseases.
     The Steering Committee members represent internationally renowned
 researchers at University of Washington, Seattle, WA; Duke University, Durham,
 NC; St. Jude Children's Hospital, Memphis, TN; Mount Sinai Medical Center, New
 York, NY; Hopital Necker-Enfants Malades, Paris, France; Mattel Children's
 Hospital at UCLA, Los Angeles, CA; NIH, Bethesda, MD and others.
     "A key part of the IDF's mission has been to fund research for primary
 immune deficiency diseases and over recent decades the primary immune
 deficiency diseases research community has made incredible strides," said IDF
 President Tom Moran.  "Now, this federal funding will enable IDF and the
 country's finest researchers to investigate new approaches.  We anticipate
 great results for our community and the world."
 
     The Immune Deficiency Foundation (IDF) is the national non-profit health
 organization dedicated to improving the diagnosis and treatment of primary
 immune deficiency diseases through research, education and advocacy.  IDF was
 established more than two decades ago by concerned families of patients and
 their physicians.  Since its inception, IDF has expanded to offer medical
 education, fellowship and research opportunities and publications.  IDF
 sponsors a biennial National Conference for patients, their families and
 healthcare professionals.  More information about primary immune deficiency
 diseases and IDF can be found at www.primaryimmune.org.
 
 

SOURCE Immune Deficiency Foundation

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