U.S. Gene Therapy Trial for Thalassemia Receives FDA Approval

July17, 2012 Memorial Sloan-Kettering Cancer Center will begin evaluating a new stem-cell-based gene therapy for patients with the inherited blood disorder beta (β)-thalassemia. The clinical trial is the first to receive US Food and Drug Administration approval to treat this disease with genetically engineered cells and is a culmination of more than two decades of research led by Memorial Sloan-Kettering investigators. Some of the research that has led to this trial was funded by CAF.

“Launching this trial is a major milestone for all the people at Memorial Sloan-Kettering and international

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

collaborators who have contributed to this work,” saysMichel Sadelain, who leads the Center for Cell Engineering “Our team was the first to show this approach was possible in disease models, and I’m thrilled to be able to finally start offering this potentially curative therapy to patients.”

Isabelle Rivière (left), Director of the Cell Therapy and Cell Engineering Facility, and Michel Sadelain, Director of the Center for Cell Engineering

“This is fantastic news and a huge step toward the hope that a cure can be discovered for our patients,” says CAF National President Anthony Viola. “We are very proud to be part of the funding of the research that has helped bring about this important trial. We congratulate all of those who have been a part of bringing the process to this point and celebrate the launch of this exciting trial.”

The trial, which is now enrolling patients, is led by Farid Boulad, a pediatric hematologist-oncologist and transplant specialist, together with Isabelle Rivière, , Director of Memorial Sloan-Kettering’s Cell Therapy and Cell Engineering Facility, and Dr. Sadelain.

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.

“Memorial Sloan-Kettering has one of the finest cell therapy facilities in the world to expand and engineer patient cells for clinical studies in patients with cancer and genetic disorders,” says Dr. Rivière.

Farid Boulad, MD

After receiving a low dose of chemotherapy to suppress the body’s natural production of blood cells, patients will have their own genetically engineered stem cells infused back into them.

“Treating a genetic defect with a reconstructed gene is something we dreamed about in medical school,” says Dr. Boulad. “The fact that it is now a reality is amazing. It is the holy grail of the treatment of genetic disorders.”

The trial will eventually be extended to patients at other institutions, including the National Institutes of Health and the University of Washington. Dr. Sadelain’s group is coordinating with investigators in Italy and Greece and throughout Asia to offer the treatment to patients there as well.

Only a small handful of diseases are currently treated with this type of gene-transfer therapy, and all but one of them are rare immune disorders. If the treatment proves effective, β-thalassemia would be by far the most common condition to be successfully treated in this way. Memorial Sloan-Kettering investigators are also preparing a follow-up study to eventually treat patients with sickle cell disease in a similar fashion.

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; and the Stavros Niarchos Foundation.

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