Month: December 2017

ASH 2017: Preliminary results show gene therapy gains in SCID

Ewelina K. Mamcarz, MD, presented Dec. 10, 2017, at the American Society of Hematology’s (ASH) Annual Meeting & Exposition in Atlanta, Georgia. X-linked SCID, severe combined immunodeficiency, is an inherited condition where a person cannot produce infection-fighting cells: T-cells, B-cells and natural killer cells. It’s a rare and devastating disease. Without treatment, babies born with SCID can contract infections and die by age two. The best available treatment, a stem cell transplant, is also very risk, and  approximately 30 percent who are treated with a transplant die by age 10. Studies with gene therapy, an experimental treatment, has also revealed a risk of leukemia. At this year’s ASH meeting, there has been exciting news reported on gene therapy for a variety of diseases, including our work in X-linked SCID (Abstract 523). We’ve treated seven infants who were born with X-SCID with gene therapy. By using an inactive form of HIV, we inserted the corrected gene in place of the defective gene that spurs the development of X-linked SCID. We also used finely-calibrated doses of busulfan to prepare the infants’ bone marrow to receive the corrected stem cells. So far, the treatment has resulted in the development of T-cell, B-cell and natural killer cells in these patients, but without side effects. We consider these preliminary results to be a substantial breakthrough for this disease. Five of the patients no longer have SCID precautions because their T cell numbers are...

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ASH 2017: GATA3 in Ph-like acute lymphoblastic leukemia

Jun J. Yang, PhD, highlights one of the ALL sessions at the 2017 annual meeting of the American Society of Hematology (ASH) in Atlanta, Georgia. My lab presented our work today on GATA3 in acute lymphoblastic leukemia (Abstract 475). Research has shown how the GATA3 gene spurs the development of a type of high-risk leukemia. We sequenced more than 5,000 pediatric ALL patients and found overexpression of GATA3 led to increased expression of a leukemia oncogene known as CRLF2. This oncogene is the defining molecular feature in nearly 50 percent of the high-risk leukemia know as Ph-like ALL. We’ve been working on this project for the past few years and now we’re able to put the story together and share that with our colleagues here at ASH. The next step is to further examine the biology behind GATA3 and use that information to develop better therapies for Ph-like ALL. In addition, there’s been other exciting progress in ALL research reported today. University of Southern California reported a study on clonal competition of ALL during therapy. This research could have an impact on discovery in the future using that technology (Abstract 480). The Plenary Scientific Session talks look particularly interesting, especially the presentation on the interaction between DNMT3A and splicing factors in acute myeloid leukemia (Abstract 1)....

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