Adult stem cell transplants are more commonly called bone marrow transplants, and are familiar to most of us as a treatment for leukemia. Stem cell transplants, however, can be applied as a permanent treatment or even cure for a number of both malignant and non-malignant disorders, including inherited and autoimmune disorders. The current limitations of a traditional bone marrow transplant are significant, however, and often mean that people who could benefit from a transplant will never receive one. At the present time, bone marrow or stem cell transplants require marrow donors and recipients to be a perfect genetic match. Even among siblings, the chance of finding a perfect match is less than 25%.
In addition, the risk of life threatening complications associated with a traditional bone marrow transplant can be as high as 40% even with a perfect genetic match. Graft-versus-host-disease (GVHD), the most common and potentially fatal complication of transplantation, occurs when cells in the transplanted donor marrow recognize the recipient as “foreign” and attack the recipient’s body. The liver, skin, mucosa and GI tract are the most common targets. GVHD can be either acute or chronic. Acute GVHD occurs within the first 100 days of a transplant; chronic GVHD occurs after the first 100 days. In some cases of leukemia, a mild form of GVHD is desirable because it may assist the recipient in fighting tumors.
A traditional bone marrow stem cell transplant requires a hospitalization of 30 to 60 days in a specialized center, where large doses of radiation and chemotherapy are used to destroy the recipient’s own bone marrow. This preparation to receive donated marrow is referred to as conditioning. Because bone marrow is home to the body’s immune system, the patient will have no ability to fight infection until the donated marrow is infused. Once the donated marrow “takes,” the immune system of the donor takes over.
After a bone marrow transplant has been successfully completed, the patient must stay in the vicinity of the transplant center for approximately 6 to 8 weeks for daily monitoring and testing. The cost of a bone marrow transplant typically runs between $150,000 and $200,000.
The Hope for Current Research
The NFCTR is supporting an active clinical collaboration between internationally recognized research teams at the University of Louisville, Northwestern University and Duke University to perform a significantly less toxic “mini” bone marrow transplant to:
- Treat patients diagnosed with relapsing-remitting MS
- Transplant organs without the need for life-long immunosuppressive medication
- Treat children suffering from inherited blood-forming diseases and rare metabolic or immune disorders
This “mini” bone marrow transplant does not require a perfect donor match and may be performed as an outpatient procedure as a result of the minimal conditioning required.
See what Collaborators Have to Say
Dr. Suzanne Ildstad |
Dr. Joanne Kurtzberg |
Dr. Joseph Leventhal |
Professor of Surgery, University of Louisville |
Professor of Pediatrics and Pathology, Duke University |
Associate Professor of Surgery, Northwestern University |
Director, Institute of Cellular Therapeutics |
Director, Pediatric Blood and Marrow Transplant Program |
Director, Living Donor Renal Transplant Program |
Jewish Hospital Distinguished Professor of Transplantation |
Director, Carolinas Cord Blood Bank |
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Dr. Suhag Parikh |
Dr. Kadiyala Ravindra |
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Assistant Professor of Pediatrics, Duke University |
Assistant Professor of Surgery, Division of Transplantation, University of Louisville |
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Pediatric Blood and Marrow Transplant Program Hematologist/Oncologist |
Liver, Kidney and Pancreas Transplant Surgeon |
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Useful / Helpful links
National Marrow Donor Program
http://www.marrow.org
National Cancer Institute
http://www.cancer.gov/cancertopics/understandingcancer/StemCells
American Cancer Society
http://www.cancer.org/docroot/ETO/eto_1_3_Bone_Marrow.asp
National Institutes for Health: Stem Cell Information
http://stemcells.nih.gov/info/scireport/chapter5.asp
