U.S.-Based Gene Therapy Study Begins Treating Participants

January 10, 2013 – CAF has learned that the first U.S.-based clinical trial in gene therapy for beta-thalassemia has begun treating trial participants.

The Phase 1 study, the official title of which is “ß-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ß-Globin Gene,” is being conducted at Memorial Sloan-Kettering Cancer Center in Manhattan. The trial received FDA approval in the summer of 2012 and began recruiting study subjects soon thereafter. It is led by Dr. Farid Boulad, Medical Director of Memorial Sloan-Kettering’s Pediatric Day Hospital; Dr. Isabelle Rivière, Director of Memorial Sloan-Kettering’s Cell Therapy and Cell Engineering Facility; and Dr. Michel Sadelain, Director of Memorial Sloan-Kettering’s Center for Cell Engineering.

In the trial, patients will have their blood stem cells extracted from circulating blood — a process in which the stem cells are filtered out of the patients’ blood while their other blood cells are returned to them. Investigators will then use a vector to introduce a functional version of the β-globin gene into patients’ stem cells. Vectors are disabled viruses that cannot replicate but efficiently shuttle their genetic cargo into host cells.

According to Dr. Sadelain, one patient has already begun receiving treatment and two others are completing the enrollment process and will begin treatment soon. The trial is an international collaboration including multiple centers in the US, Italy and other countries.

“We provided scientific proof-of principle for this approach over a decade ago, and have since focused on resolving numerous clinical, biological and manufacturing challenges to open this trial under the best possible conditions. The approval of our cell manufacturing process and the design of our clinical trial by the US FDA in 2012 was an important milestone not only for thalassemia, but also for future studies in sickle cell disease and other blood disorders”, said Dr Sadelain. “Capping 18 years of research supported in part by the Cooley’s Anemia Foundation, this trial is a big step towards eventually providing thalassemia patients with a curative option designed to obviate the need for life long transfusions.”


Anthony J. Viola, National President of the Cooley’s Anemia Foundation, says that “The Cooley’s Anemia Foundation is very excited that this important trial is moving forward at this pace. Certainly the expertise and knowledge of Drs. Sadelain, Boulad and Riviere are significant factors in the progress of the study, which is responding to a demonstrated need for a potential curative treatment for thalassemia. The Foundation is proud to have played a part in supporting research that has helped lead to this trial and acknowledges the many years of dedicated research undertaken by both the scientists involved in this trial and in other areas of gene therapy which have added to the fund of knowledge in gene therapy.

“This study, and other studies in this area which will launch in the future, are providing an invaluable service both for individuals with thalassemia and for all who are interested in the future of gene therapy for a wide range of disorders.”

To learn more about this trial, including eligibility requirements, click here.

The research that led to this FDA-approved trial was supported by the National Institutes of Health under award numbers HL53750, HL57612, and HL66952; Errant Gene Therapeutics LLC; the Cooley’s Anemia Foundation; the Leonardo Giambrone Foundation; Cooley’s Anemia International; the Stavros Niarchos Foundation; and Franco and Piera Cutino Foundation.



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