Coordination: Manuel Carrondo and Cláudia Lobato da Silva

 

 

Objectives

 

The overall objective of this course is to provide the main theoretical fundamentals on developmental and stem cell biology.

 

Program

 

1. “From bed-to-bench” paradigm: identification and characterization of unmet medical needs: cancer, neurodegenerative and cardiovascular diseases, infectious diseases, among others. 2. Cell therapies. Autologous and allogeneic cell therapies. Examples. Types of cells that can be used in Cell therapies. Tissue Engineering. Immunomodulation. Stem cell-based and progenitor cell-based therapies. Development of processes for production of cell therapy products. Pre-clinical studies. Cell therapy products already approved by the regulatory agencies. 3. Current clinical applications of stem cells: haematopoietic stem cells. Clinical studies with adult stem cells (hematopoietic and mesenchymal stem cells, neural stem cells, among others) and embryonic stem cell-derived cells. Case studies. 4. From bench-to-bed paradigm: ATMP (advanced therapeutic medicinal products). ATMP Regulation: regulatory agencies and legislation. Economic challenges. Quality control, GMP and GLP. GMP facilities. Clinical studies: phases I, II, III and IV. Concepts of safety, efficacy and statistical relevance. Time and cost for approval of an ATMP product. Orphan drugs state. Distribution channels and storage of ATMP. Commercialization of ATMP. 6. Visits to clinical and GMP facilities. 7. Bioethics and life sciences: concepts and fundamentals, bioethics laws and theories. Preservation of research integrity in life sciences. Ethics and legislation in the use of human cells and tissues in research and application of cell therapies and regenerative medicine. 8. Ethics and stem cell research: legislation and ethics for the use of human embryos and embryonic stem cells and other sources of pluripotent and totipotent stem cells. Therapeutic and reproductive cloning. Private versus public investment in stem cell research. Creation of proprietary rights for the materials and products of stem cell research. The impact of religion on stem cell research. 9. Ethics of cell therapy research and application. Distinction between the concepts of research and application of cellular therapies. Relations-ship doctor-patient: informed consent and confidenciality. Concept of Good Clinical Practice (GCP). Ethical committees. 10. Private and public cell banks. 11. Ethics in Human Genetics. 12. Ethics in gene therapy. 6. Leadership and team building and Development of communication skills.

 

Evaluation Methodology

 

Essay - Critical analysis of successful and failed cell therapy and regenerative medicine clinical trials.
Development and public presentation of a monography about a case study on cell therapies and regenerative medicine ethics.