The MPD-RC Projects

The MPD-RC program has six major projects.

The program, developed with the efforts of all the members of the Consortium, consists of basic, translational and clinical studies. All the projects are considered highly meritorious, very strong in preliminary data and research design, and with great potential to produce significant results.



Laboratory Projects

Project 1: (Genetic Basis of Polycythemia Vera, Principal Investigator: Josef T. Prchal) Biomarkers derived from families of PV patients will be studied and compared to those found in sporadic forms of the disease. Identifying unique biomarker profiles may help to distinguish the two forms of PV, thus avoiding unnecessary chemotherapy in patients with inherited forms.

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Project 2: (Mechanisms and Effects of NF-E2 and PRV-1 Overexpression in PV: Role of Jak2V617F, Principal Investigator: Heike L. Pahl) will investigate the genetic basis of altered gene expression in the haematopoietic cells of patients with Polycythemia Vera. This project aims to clarify the mechanism by which the Jak2V617F mutation exerts its effect. In particular, the consequences of overexpression of transcription factor NF-E2 will be determined.

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Project 3: (Animal Models ofPolycythemia Vera, Principal Investigator: Jerry Spivak) will seek to understand Polycythemia Vera by creating the same mutation, observed in the human form of the disease, to animal models. This lays the basis for consequently testing of new drugs on blood forming (haematopoietic) progenitor cells expressing the selected abnormalities.

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Project 4: (Mouse Models of Myelofibrosis, Principal Investigator: Anna Rita Migliaccio) will utilize mutant mice, as an animal model of myelofibrosis, that express reduced levels of a transcription factor that plays an important role in differentiation of the precursors of red cells (erythroblasts) and platelets (megacaryocytes). This unique model system will permit greater understanding of abnormal differentiation of stem cells and disease progression in myelofibrosis.

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Project 5: (Abnormal Stem Cell Trafficking in Myelofibrosis, Principal Investigator: Ronald Hoffman) addresses the pathogenetic mechanisms that result in sustained mobilization of primitive hematopoietic progenitor cells and stem cells into the peripheral blood (PB) of patients with IM. Greater understanding of this abnormal stem cell trafficking in IM will likely lead to the identification of therapeutic targets against which novel drugs might be directed.

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Clinical Projects

(MPD Clinical Consortium, Co- Principal Investigator Lewis Silverman and Alessandro Rambaldi)
These Projects involve studies with patients (clinical studies) who have been diagnosed with Essential thrombocythemia, Polycythemia Vera and Idiopathic Myelofibrosis. The studies are based on some of the findings from the previous projects, the arrival of new therapies and strategies that need to be evaluated. The opportunity of starting clinical studies that directly address the biomarkers responsible for the progression of the disease will offer a chance to these patients to receive best care available with the hope of a better prognosis. The unique link between laboratory and clinical studies will speed up the time needed to test drugs and other therapies and to get effective ones integrated in standard care. Within the clinical studies, tissue samples from patients with Ph- MPD are obtained that-with the consent of the patients involved-will be stored at the Tissue Bank so that they can be shared with researchers of Laboratory Projects 1 to 5.

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Posted on September 11 2006 10:38:12 Print