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FOR IMMEDIATE RELEASE
NFCTR Funded Researcher Makes Significant
Progress in Treatment of Sickle Cell Disease
Collaborative Funding for Stem Cell Therapy Cure
May Benefit 100,000 in U.S. and Millions Worldwide
The National Foundation to Support Cell Transplant Research (NFCTR) today announced that a clinical trial collaboration it is helping to fund – between Dr. Suzanne Ildstad at the University of Louisville and Dr. Joanne Kurtzberg at Duke University – has shown promising results as a potential cure for sickle cell disease (SCD).
Based on the discoveries of Dr. Ildstad, Director of the Institute for Cellular Therapeutics at the University of Louisville, the treatment involves a “mini” bone marrow transplant that is performed as an outpatient procedure and does not require a perfect donor match.
SCD is a painful, debilitating and ultimately fatal inherited disease that affects approximately one out of every 500 African-American children and one out of every 1,000 Hispanic-American children born in this country. At present, there are between 70,000 and 100,000 diagnosed cases of SCD in the United States. Worldwide, several million children and young adults suffer from SCD.
Normal bone marrow produces red blood cells that are round and tumble through blood vessels as they carry oxygen to organs and tissue. The bone marrow of a child with SCD produces red blood cells that are sickle (crescent) shaped. Instead of tumbling, they stack up in the small vessels of the body. Stroke, acute pain and episodes of asphyxia are hallmarks of the disease.
Dr. Ildstad is internationally known for her discovery of a graft “facilitating” cell that allows bone marrow stem cells to be transplanted from one person to another without life-threatening rejection. As a result, it became possible to transplant small amounts of normal, healthy bone marrow into children with red blood cell disorders (like SCD) without the risk of graft-versus-host disease, the too often fatal consequence of traditional bone marrow transplant. It also eliminated the need for a perfect donor match.
The transplanted healthy bone marrow then provides an ongoing supply of normal red blood cells sufficient to eliminate any symptom of disease.
In a recent Louisville Courier-Journal article (February 21, 2010) Dr. Ildstad said study results are promising – with two of six patients basically cured and a third recent one doing well – and it shouldn't be long before new, safe transplants are available to many of the nation's 70,000 sickle cell patients.
The potential for a cure has generated excitement among other scientists nationwide. Researchers at Northwestern University in Chicago plan to begin treating sickle cell patients through the study in about six months.
Ildstad’s work has generated continuous funding to the University of Louisville from the National Institutes of Health (NIH), the U.S. Department of Defense (DoD) and the National Foundation to Support Cell Transplant Research (NFCTR).
“We are extremely encouraged by this exciting research,” states Dr. Paula Grisanti, Chair of NFCTR, “and by the progress being made to cure sickle cell disease and a number of other conditions that may benefit from Dr. Ildstad’s discoveries.”
About the National Foundation to Support Cell Transplant Research (NFCTR)
Formed in December 2005, the National Foundation to Support Cell Transplant Research (NFCTR) was founded by a small group of individuals – each personally impacted by the potential for this critical research. It was established to support recognized, peer-reviewed research and early stage clinical trials in the field of adult stem cell transplantation. Stem cell research has the potential to revolutionize the treatment of a large number of diseases and conditions affecting people worldwide. The NFCTR funds research in a number of strong academic settings and supports both collaboration and information sharing wherever possible. Through the funding of these collaborations, the Foundation hopes to create stronger opportunities for advancements in the field. For more information visit www.nfctr.org or call (502) 379-8544
Click here for NFCTR logo: http://nfctr.org/press/NFCTR-Logo.jpg
Contact:
National Foundation to Support Cell Transplant Research (NFCTR)
Dr. Paula P. Grisanti
(502) 379-8544
pgrisanti@nfctr.org
Source: National Foundation to Support Cell Transplant Research
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