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A New Cell-Based Therapy to Treat Leukemia

Drs. Michel Sadelain, M.D., Ph.D., Renier Brentjens, M.D., Ph.D., and Isabelle Rivière, Ph.D.,  plus a team of researchers based at Memorial Sloan-Kettering Cancer Center, developed a new treatment for patients with relapsed B-cell acute lymphoblastic leukemia (ALL).  This particular form of leukemia has a very poor prognosis, typically requiring intense chemotherapy and eventually hematopoietic stem cell (HSC) transplantation to eliminate the leukemia and rebuild the immune system.  Using a new method, the team of physicians and researchers isolated T-cells from the patients and genetically engineered them to target a protein expressed on all B-cells.  Both B and T-cells are part of the body’s immune system, so this therapy turns the immune system on itself, resulting in elimination of both leukemic and normal B-cells, which occurred with striking rapidity in all five patients treated.  Four of the five patients then received the HSC transplant, which is the standard of care for relapsed ALL and restored normal B-cell production.  Despite this arduous process, three of the five patients treated appear to have complete remission, one within eight days of the treatment, and remain so to date, up to 24 months.  A fourth patient died of an unrelated complication, free of the cancer.  The study was published in Science Translational Medicine.

Dr. Sadelain received NYSTEM funding through contract C024412 to explore the possibility of using induced pluripotent stem cells rather than blood stem cells for this treatment and, along with Dr. Rivière, is part of NYSTEM consortium aimed at developing a stem cell-based therapy for Parkinson’s disease (C028503).

Brentjens RJ, Davila ML, Riviere I, Park J, Wang X, Cowell LG, Bartido S, Stefanski J, Taylor C, Olszewska M, Borquez-Ojeda O, Qu J, Wasielewska T, He Q, Bernal Y, Rijo IV, Hedvat C, Kobos R, Curran K, Steinherz P, Jurcic J, Rosenblat T, Maslak P, Frattini M, Sadelain M.  CD19-Targeted T Cells Rapidly Induce Molecular Remissions in Adults with Chemotherapy-Refractory Acute Lymphoblastic Leukemia.  Sci Transl Med. 2013 Mar 20;5(177):177ra38.

More information on this trial can be found at or at the New York Times.