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

ffer services to help these patients regain that function after transplant."

Ms. Bosworth will present this study during an oral presentation at 7:30 a.m. CST on Tuesday, December 10, in Riverside Rooms R06-R07 of the Ernest N. Morial Convention Center.

Donor-Derived Anti-CD19 Chimeric-Antigen-Receptor-Expressing T Cells Cause Regression of Malignancy Persisting After Allogeneic Hematopoietic Stem Cell Transplantation [151]

This study examined the efficacy and safety of genetically modified T cells as treatment for B cell malignancies (blood cancers that affect the B cells, including certain types of leukemia and lymphoma) persisting after allogeneic hematopoietic stem cell transplantation (HSCT). The study focused on the use of these carefully designed attack cells in 10 patients who had persistent, aggressive disease after receiving one or more allogeneic HSCTs. Investigators removed T cells from each of the patients' healthy donors and then, in the laboratory, outfitted them with genetic machinery designed to target a protein expressed on the persisting malignancy (B cell antigen CD19) when infused in the patient. After receiving one infusion of modified cells with no other anti-cancer therapy, three of 10 patients experienced significant disease regression, with one patient achieving ongoing complete remission at nine months post treatment. A fourth patient had stable disease at 11 months post infusion. Importantly, none of the patients experienced graft-versus-host disease (GVHD), and the most prominent toxicities were fever and low blood pressure, which resolved within two weeks.

"These results are particularly exciting because they show that small numbers of these modified T cells can cause regression of highly treatment-resistant B cell malignancies after transplant without causing GVHD, indicating a possible new treatment approach for thos
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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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