2013-02-14
Welcome to “This Week in Blood,” a weekly snapshot of the hottest studies from each week’s issue of Blood, the official journal of the American Society of Hematology (ASH), hand-picked by Blood Editor-in-Chief Bob Löwenberg, MD, and Deputy Editor Nancy Berliner, MD. If you would like a PDF copy of any of the manuscripts highlighted below or would like to request an interview with the author, please email aslesinski@hematology.org.
Anti-CD22-chimeric antigen receptors targeting B cell precursor acute lymphoblastic leukemia, Haso et al.
Chimeric antigen receptor (CAR) genetically modified T-cell therapy represents an important therapeutic modality in early clinical development for lymphoid malignancies. The use of anti-CD19 CAR T cells has already led to impressive clinical effects in precursor B acute lymphocytic leukemia (B-cell ALL) and this manuscript builds upon that, presenting compelling data on the generation of T effector cells targeting a novel antigen (CD22) in this disease. These results support the use of CAR modified anti-CD22 CAR T cells in patients with B-cell ALL, a disease with a generally poor prognosis, especially in adults, and suggest that T cells might be employed therapeutically not only alone with a similar clinical potency as anti-CD19 CAR but possibly also in combination with anti-CD19 CAR. These anti CD22 CARs can have a significant impact in the treatment of various CD22+ malignancies.
Dysfunction of the PI3 kinase/Rap1/integrin IIb3 pathway underlies ex vivo platelet hypoactivity in essential thrombocythemia, Moore et al.
Patients with essential thrombocythemia (ET) are known to show a significantly increased risk of bleeding. The authors of this manuscript provide mechanistic insight into the reason why platelets from patients with ET are dysfunctional, revealing that platelets of patients with ET have a general defect in important thrombin and thrombopoietin activation pathways.
SAP gene transfer restores cellular and humoral immune function in a murine model of X-linked lymphoproliferative disease, Rivat et al.
This manuscript presents the first proof of principle of relevant, non-toxic correction of several key features of X-linked lymphoproliferative disease (XLP1) by gene therapy in a suitable mouse model. The observation, that various immune functions that are defective in this disorder have the ability to normalize, represents a major step towards the development of clinical gene therapy for infants with this severe inborn immune disorder.
Murine anti-3rd-party central-memory CD8+ T-cells promote hematopoietic-chimerism under mild conditioning: lymph-node sequestration and deletion of anti-donor T-cells, Ophir et al.
Engraftment failure of donor cells after non-myeloablative conditioning restricts the clinical utility of allogeneic stem cell transplantation (HCT) for the treatment of many diseases. In this paper, the authors used in vitro-generated anti-3rd party central memory CD8 T cells (Tcm) to promote engraftment of allogeneic or semi-allogeneic marrow in animals receiving non-myeloablative conditioning. The elegant experimental demonstration that this novel approach can promote engraftment with non-myeloablative conditioning has a potentially major impact on the use of HCT for the treatment of non-malignant disorders.
Reporters who wish to receive a copy of any of the manuscripts highlighted above or would like to request an interview with the authors may contact Andrea Slesinski at 202-552-4927 or aslesinski@hematology.org.
Blood (www.bloodjournal.org), the most cited peer-reviewed publication in the field of hematology, is available weekly in print and online. Blood is the official journal of the American Society of Hematology (ASH) (www.hematology.org), the world’s largest professional society concerned with the causes and treatment of blood disorders.
ASH ’s mission is to further the understanding, diagnosis, treatment, and prevention of disorders affecting blood, bone marrow, and the immunologic, hemostatic, and vascular systems by promoting research, clinical care, education, training, and advocacy in hematology.
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