The AHEPA Cooley’s Anemia Foundation welcomes news that the first U.S.-based clinical trial in gene therapy for beta-thalassemia has begun treating trial participants, announced Supreme President Dr. John Grossomanides. The trial received FDA approval in the summer of 2012 and began recruiting study subjects soon thereafter.
The Phase 1 study, officially titled, “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 New York City. Dr. Michel Sadelain, director of Memorial Sloan-Kettering’s Center for Cell Engineering and whose research the AHEPA Cooley’s Anemia Foundation has supported with grant-funding, is a member of the research team. In addition to Dr. Sadelain, the trial 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.
“We are very excited about this next important step forward for the trial,” said Ike Gulas, chairman, AHEPA Cooley’s Anemia Foundation, whose son suffers from Cooley’s Anemia. “Our foundation is proud to support researchers such as Dr. Sadelain, whose expertise and knowledge contributed greatly to the advancement of this clinical trial in gene therapy to treating trial participants. Our commitment remains steadfast to supporting medical research in the hope that one day a cure will be found for Cooley’s Anemia.”
According to the Cooley’s Anemia Foundation, 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.
Photo 1st page: Ο επανεκλεγείς ύπατος πρόεδρος Δρ. Τζον Γροσομανίδης (Δεξιά) και εκτελεστικός διευθυντής Βασίλης Μωσαϊδης.