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
-- 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
-- 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
-- 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