Journal Reports on Ongoing Gene Therapy Trial in Thalassemia

September 16, 2010 – A case report published today in the journal Nature discusses the current status of an ongoing gene therapy trial involving patients with thalassemia.

“Transfusion independence and HMGA2 activation after gene therapy of human B-thalassemia,” authored by a team led by Philippe Leboulch, reports on the case of a 21-year-old French male patient with e-beta-thalassemia. The patient was transplanted on June 7, 2007 and was last transfused on June 6, 2008. He is maintaining a stable hemoglobin level between 9 and 10 g dl.

The report also discusses the fact that there has been some overexpression of one gene; overexpression can in extreme cases lead to bad outcomes, such as leukemia. At this point, expression of this gene is an uncertain consequence; however, the authors indicate that the overexpression has plateaued and is now remaining stable.

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“The data from this trial was initially reported last fall at New York Academy of Science Symposium sponsored by the Cooley’s Anemia Foundation,” says CAF National Executive Director Gina Cioffi. “We consider this field and these initial results important and believe that with appropriate funding over the next several years, this work will inform the growing community of researchers working to advance gene therapy as a curative approach. We also believe that this is an area where investment made in the production of vectors and the running of clinical trials will generate a great return. Our Foundation is fully committed to funding gene therapy clinical trials and working to advance new funding mechanisms to enable this work to expand robustly.”

Click here to read an article in the Los Angeles Times about the Nature article.

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