Clinical Trials Saving Kids

April 6, 2009 TREATMENT: Currently, the most effect treatment for SCID is a bone marrow transplant. Stem cells are taken from a healthy donor's bone marrow and injected into the patient. If the treatment goes well, the stem cells will stimulate the production of necessary immune and blood cells. Most transplants performed within the first few months of life are successful, however it is important that the donor tissue matches the patient. Siblings, or matched donors, make the best donors because they have the most similar genetic make-up. A half-matched donor, or a parent, can also provide a good match. A clinical trial that involves giving chemotherapy before the bone marrow transplant is currently underway at the University of Miami School of Medicine. "If you give chemotherapy you make it easier for the new cells to grow and we think that it actually makes the immune system come in better later down the road," Gary Kleiner, M.D., Ph.D., a pediatric immunologist at the University of Miami School of Medicine, told Ivanhoe.

FIXING COMPLICATIONS: While bone marrow transplants are effective for developing T-cells in patients with SCID, they sometimes cause complications. Veno-occlusive disease (VOD) is a condition in which some of the small vessels in the liver are blocked. The complication can arise due to the high-dose chemotherapy given before a bone marrow transplant. Symptoms include increased liver size, weight gain and clotting problems. A phase III clinical trial is currently investigating the use of the drug defibrotide as a potential treatment for VOD. Dibribrotide is believed to work by prevnting clotting in the blood vessels and by helping dissolve clots. Without treatment, VOD is often a fatal condition.


Omar Montejo, Director of Media Relations
University of Miami School of Medicine
(305) 243-5654

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