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International gene therapy trial for 'bubble boy' disease shows promising early results
Date:12/7/2013

(NEW ORLEANS, December 7, 2013) Hematopoietic stem cell transplantation (HSCT), once considered an effective yet risky alternative to drug therapy for blood cancer, has become more accessible and successful in a wide range of patients as a result of major advances in transplant strategies and technologies. Several studies representing these advances were presented today during the 55th American Society of Hematology Annual Meeting and Exposition in New Orleans.

HSCT is effectively used today as a form of "replacement" therapy for patients with hard-to-treat blood cancers, providing healthy cells from either the patient (autologous transplantation) or from a donor (allogeneic transplantation) to better equip patients to fight the disease on their own. Historically, clinicians evaluating a patient for transplant have sought to identify donor cells that are perfectly matched to the patient's cell type, which is considered to be the optimal approach to help ensure successful outcomes and to minimize risk of graft-versus-host disease (GVHD), a serious and potentially life-threatening complication that occurs when the donated immune cells attack the patient's cells as foreign tissue.

Today, researchers are challenging traditional assumptions about transplant eligibility and donor matching in an effort to expand the universe of patients who can benefit from this treatment while improving long-term success rates. New findings presented today indicate that with proper considerations, haploidentical (half-matched) transplants or transplants using cord blood cells can be viable, effective alternatives when a fully matched donor is not available. Researchers also contend that transplant outcomes can be further improved by identifying patients who are at high risk for certain complications, such as cognitive decline, or by employing post-transplant treatments to reduce their risk of relapse.

"The exciting research results being presented today
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Contact: Irene Sege
irene.sege@childrens.harvard.edu
617-919-3110
Dana-Farber Cancer Institute
Source:Eurekalert  

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International gene therapy trial for 'bubble boy' disease shows promising early results
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