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High-Tech Advances In Gene Therapy Overcome Challenges, Offer Hope For Patients With Hard-to-Treat Blood Disorders

Other advances in cell engineering reported today include a new generation of gene "vector" therapy that self-destructs once it delivers critical, missing genetic material to a patient, solving the issue of T cell overgrowth observed in previous studies. Finally, genetic modifications of haploidentical (or half-matched) stem cells prior to transplant could expand the utility of this treatment approach to a much wider range of patients in the coming years by reducing the risk of transplant infections. 

"It's exciting to see these encouraging initial results with engineered immune cells, particularly such a durable response among patients who have very aggressive disease that has relapsed after standard treatments," said Laurence Cooper, MD, of The University of Texas MD Anderson Cancer Center in Houston. "With the right technology and laboratory expertise, the process of cell engineering is feasible for many patients. One remaining challenge is determining why some patients benefit and others have less durable responses. Does 'one size fits all' therapy work or do we need personalized or individualized T cell treatments? Further, we need to extend these studies to other tumor types, particularly solid tumors, to evaluate their potential in other clinical settings."

This press conference will take place on Saturday, December 7, 2013 at 8:00 a.m. CST.

Removal of Alpha/Beta+ T Cells and of CD19+ B Cells From the Graft Translates Into Rapid Engraftment, Absence of Visceral Graft-Versus-Host Disease and Low Transplant-Related Mortality in Children With Acute Leukemia Given HLA-Haploidentical Hematopoietic Stem Cell Transplantation [ 157 ]

Transplants of haploidentical, or half-matched, blood-forming stem cells may be an effective option for patients in need of a transplant without a fully matched donor; however, in the past, in comparison to transplant from a fully matched donor, this treatment has been associated with an increased risk of infection and disease recurrence. This study tested the effectiveness of manipulating in the lab these half-matched donor stem cells. 

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