Center for Research on Cell-Based Therapy

Resumo

Description of the Center and its characteristic features. The Center is composed of researchers in the fields of human cytology, molecular and population, genetics, immunology, protein chemistry, cell culture, blood coagulation, transplantation biology, transfusion medicine and hematology, who have collaborated in different associations in the past and work in three closely located facilities in the University of S. Paulo, campus of Ribeirao Preto: 1. Regional Blood Center and Biocenter; 2. Hematology Laboratories, Bone Marrow Transplant Unit and Hematology Wards at the University Hospital; 3. Protein Chemistry Laboratory. All researchers share an interest in the cellular and molecular aspects of the blood tissue, its function, diseases and therapeutics, and now propose to join efforts to answer questions related to the use of cells to treat human diseases, especially hematological diseases. Most of the researchers of the center are already using cells to treat human diseases (transfusions, allogeneic and autologous transplantation) or are studying features of these cells, of the donors and of the recipients, which may have a bearing on the evolution of the disease or the effect of treatment. b) Definition of research focus and of its multidisciplinary connections. The research focus is cell therapy for treatment of diseases; this includes allogeneic bone marrow transplantation, therapy with autologous stem cells obtained from peripheral blood, and novel approaches such as the use of umbilical cord blood. The cells can be transplanted immediately after collection, or they can be processed and cryopreserved for long periods. There is great interest in further processing of these cells, such as purging the neoplastic cells, 'ex vivo' expansion and gene transfer for gene therapy. The initial research program includes evaluation of immunologic, structural and genetic characteristics of transplantable hematopoietic stem cells from different sources, cell-to-cell interactions and signaling, the setting up of a cord cell bank and evaluation of properties of cord blood stem cells, especially after 'ex vivo' expansion, the genetic manipulation of cells, the evaluation of some disease characteristics of the recipients, and the setting up of animal models for the study of the diseases and transplant treatment. c) Description of planned technology transfer and educational activities and of their relationship to the research focus. The Regional Blood Center is a facility with an independent administration jointly supported by the university, the university hospital and the state Department of Health; since its establishment in 1985 it gradually took over the direct processing and supervision of blood collection and distribution of blood components in a region containing 4.2 million inhabitants (167 municipalities), the training of specialists and technicians, and the definition of public policies in the area of blood transfusion. Three of the principal researchers who participate in the Center have had previous experience with the transfer of research results to the biotechnology industry or have applied for patents in this area. The planned technology transfer will include: 1. Creation and operation of a cord cell blood bank; 2. Development of new diagnostic approaches to diseases related to the treatment of blood-derived products (for instance, AIDS and Chagas disease); 3. Investigation of critical aspects of therapy with blood or cell products, which may be transferred to the health authorities and serve as the basis of public policies in this area; 4. The transfer of modern know-how in cell therapy by training of medical personnel (medical doctors, nurses and technicians). Educational activities will include: 1. A long-term joint program with the regional branches of the state and the city Departments of Education for high-school teachers and students has been negotiated. The program includes continuing education classes for teachers, short courses in specific subjects for students, and the development of introductory research projects in the laboratories of the Center and in the school laboratories for a number of selected high-school students and teachers, the results of which will be presented in mini-congresses. The subjects for this program are cytology, genetics, immunology. biochemistry and health education. 2. The collaboration with high school teachers for the development of educational resources (virtual museums and libraries, films, printed material, CD for personal computers). 3. Training of biologists, pharmacists, nurses and technicians in the laboratory methods involved in cell collection, processing and cryopreservation, donor selection, the clinical support of donors and recipients, laboratory methods for cell separation and culture, the immunologic, genetic and biochemical strategies of research. 4. Introductory research programs for at least 10 medical students a year. 5. Training of residents and graduate students (MSc and DSc) as part of the university's graduate program. d) Brief justification for the creation of a research center. To study at least part of the phenomena involved in cell-based therapy it is necessary to combine efforts of medical doctors doing clinical research in hematology and stem cell transplantation with those of basic researchers in the fields of cytology, cell separation and culture, immunology, molecular genetics, and protein chemistry. Adequate clinical facilities, laboratories for basic work and facilities for cell collection, processing and storing are also necessary. The center includes researchers and technicians who are trained (or in training) for the use of all the proposed methods, the researchers have worked together in small groups, there are considerable resources already available (buildings, scientific equipment, administrative support), the researchers have interactions with other relevant groups in the field, and they carry out both clinical work and basic research in the same environment. e) Description of facilities offered as counterpart by the participating Institutions. Physical installations of the Center include the Regional Blood Center (2200 m2 of laboratories + 1800 m2 administrative and educational area) and the adjacent Biocenter (4000 m2 laboratories, in final phases of construction) which are located 300 m from the University Hospital which contains the Hematology Laboratories (300 m2) and Wards (11 beds), and a Bone Marrow Transplant Unit (5 especially isolated rooms); the Protein Chemistry Laboratory (300 m2, will be transferred to the Biocenter in a 500 m2 area). Personnel: 8 principal researchers (salaries paid by the university or the Regional Blood Center); 33 medical doctors or biologists/technicians (salaries paid by the university, the university hospital or the Regional Blood Center); 17 biologists, technicians or medical students in training (scholarships paid by the Regional Blood Center). Large equipment already in place (3 Facs-sorters, 2 automatic DNA-sequencers, 2 automatic amino acid-analyzers, 1 ESI MS/MS mass spectrometer, 1 automatic protein sequencer, 1 capillary HPLC, 1 preparative ultracentrifuge, 5 thermocyclers for PCR, 2 nitrogen-freezers for storing cryopreserved cells, 3 apheresis instruments for obtaining peripheral blood cells from patients, 1 Electra ML1600 coagulometer, facilities for cell culture). The Regional Blood Center is presently implementing an IS09002 quality program. (AU)