Landmark Sickle Cell Disease Trial To Investigate Unrelated Donor
Marrow And Umbilical Cord Blood Transplants
ScienceDaily (Aug. 19, 2008) — Children's National Medical Center
immunologist and blood and marrow transplant physician Naynesh
Kamani, MD, will serve as the study co-chair for a new national
clinical trial of unrelated donor marrow and umbilical cord blood
transplants for severe sickle cell disease. Children's National will
join more than 20 other medical institutions in the first-ever Phase
II clinical trial of this treatment. If successful, the Sickle Cell
Unrelated Transplant (SCURT) trial has the potential to extend a
promising and possibly curative treatment option to more severely
affected patients. Sickle cell disease affects approximately 70,000
people in the United States.
For many years, doctors have used blood or marrow transplants to
treat severe cases of sickle cell disease. However these treatments
have been available only to patients with a matched family member to
donate blood or marrow stem cells. Only 14 percent of severe sickle
cell cases have a matched donor within their family. Successful
transplants using unrelated donors could create a viable treatment
option for more sickle cell patients.
"Blood and marrow transplantation from unrelated donors has been
routinely utilized to treat treatment-refractor
lymphoma for almost two decades," said Dr. Kamani, co-principal
investigator of the study. "Preliminary results from the few
unrelated donor transplants that have been performed for other non-
malignant conditions show great promise for those who suffer a
severely diminished quality of life due to sickle cell disease.
Participants in this large-scale study will help us understand how to
better treat, and possibly even cure, sickle cell disease for more
people."
Starting in late August, the clinical trial will enroll 45 patients
ages 3 to 16 with severe symptoms of sickle cell disease, including
stroke, recurring acute chest syndrome, or frequent pain crises. For
all marrow and cord blood transplants, patients must undergo intense
chemotherapy to prevent their immune systems from rejecting donor
cells. This study will use a reduced-intensity chemotherapy to
minimize side effects and increase the likelihood that the donor
cells will be accepted by the body. After the transplant,
participants will be followed for two years to gauge the
effectiveness of the transplant at minimizing the ravages of sickle
cell disease.
The trial is facilitated by the Blood and Marrow Transplant Clinical
Trials Network, and is funded by the National Heart, Lung, and Blood
Institute, the National Cancer Institute, the National Marrow Donor
Program, the Sickle Cell Disease Clinical Research Network, and the
National Center on Minority Health and Health Disparities.
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Adapted from materials provided by Children's National Medical
Center, via EurekAlert!, a service of AAAS.
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MLA Children's National Medical Center (2008, August 19). Landmark
Sickle Cell Disease Trial To Investigate Unrelated Donor Marrow And
Umbilical Cord Blood Transplants. ScienceDaily. Retrieved August 19,
2008, from http://www.scienced
/releases/2008/
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StemCells subscribers may also be interested in these sites:
Children's Neurobiological Solutions
http://www.CNSfoundation.org/
Cord Blood Registry
http://www.CordBlood.com/at.cgi?a=150123
The CNS Healing Group
http://groups.yahoo.com/group/CNS_Healing
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