Memorial Sloan-Kettering Cancer Center cell engineering researchers and their clinician colleagues have been in the news recently for a successful experimental cell therapy. Called targeted immunotherapy, a patient's T cells are genetically altered in the lab, then reintroduced with the directive to target and kill cancer cells. The treatment was carried out on a group of adults who all suffered from a rapidly progressing form of leukemia that had not responded to chemotherapy. All five went into remission after the novel cell treatment, and three have stayed that way for a number of months. Results of the ongoing clinical trial appeared in the March 20 online edition of the journal Science Translational Medicine, along with an article in the New York Times.
B cell acute lymphoblastic leukemia (ALL) is particularly deadly in adults, with a cure rate of only 40% from the traditional treatment combo of chemotherapy and a subsequent bone marrow stem cell transplant. When chemotherapy fails to cause remission, the ALL patient is not able to progress to the stem cell therapy and the end usually comes quickly.
In the immunotherapy procedure pioneered at Memorial Sloan-Kettering, a patient's white blood cells are removed and a new gene is introduced into the cell using an engineered viral vector. When returned to the leukemia patient's body, the altered cells recognize a protein called CD19, which is present in B cell ALL tumor cells. The immune system attack is then full-blown, and patients needed to be treated for extreme weakness with steroids during the battle period, but the result was remission within a matter of days. The three treated individuals who remained in remission (one relapsed, and one died of an unrelated blood clot) went on to have bone marrow transplants, not because they necessarily needed it (there was no cancer in their blood at that point), but because it is standard procedure.
[Video outlining immune cell engineering, courtesy of Memorial Sloan-Kettering]
The principal researcher for the laboratory phase of this landmark gene therapy is Dr. Michel Sadelain, who heads the Gene Transfer and Gene Expression Laboratory at Sloan-Kettering. His research-physician colleagues include Drs. Renier Brentjens, Marco L. Davila, and Isabelle Rivière, who is director of the Cell Therapy and Cell Engineering Facility within the Center for Cell Engineering, where all four doctors are on faculty. Sadelain directs the Center. He said of the team's work:
“We’re creating living drugs. It’s an exciting story that’s just beginning.”
In another recent blog of ours, we highlighted 4 Manhattan researchers (two from Rockefeller, one from Memorial Sloan-Kettering, and one from Weill Cornell) who each won the newly-established $3M Life Sciences Breakthrough Prize.
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