Projects / Programmes
Human stem cells-advanced cell therapy III
January 1, 2017
- December 31, 2021
| Code |
Science |
Field |
Subfield |
| 3.04.00 |
Medical sciences |
Oncology |
|
| 3.01.00 |
Medical sciences |
Microbiology and immunology |
|
| Code |
Science |
Field |
| B490 |
Biomedical sciences |
Haematology, extracellular fluids |
| Code |
Science |
Field |
| 3.02 |
Medical and Health Sciences |
Clinical medicine |
| 3.01 |
Medical and Health Sciences |
Basic medicine |
stem cells, regenerative medicine, advanced therapies, bone marrow, umbilical cord blood, ex vivo expansion, markers of pluripotency, clinical trials
Researchers (22)
Organisations (3)
Abstract
In the past period, our research programme introduced advanced therapies to Slovenia. During this programe, stem cell (SC) research from various Slovenian institutions and laboratories has been successfully combined and coordinated. The new knowledge has been immediately translated into the clinics where these methods were successfully applied in more than one thousand patients, mostly at the University Clinical Centre, Ljubljana.
Novel protocols for collection of SCs from umbilical cord, peripheral blood and bone marrow were developed, followed by introduction of various cell separation and cell sorting methods, isolation of specific subpopulations of SCs and their long-term cryopreservation. Similarly, protocols for differentiation of SC into all three germ layers and protocols for various molecular methods were used to determine gene expression profiles, as well as novel culture conditions used that enable ex vivo expansion of SCs. We also acquired extensive knowledge from the transcriptomics and metabolomics of SCs.
As a logical continuation in the next period, we would like to achieve further improvement in the quality and success of cell treatments, and render them accessible to a wider range of patients in Slovenia. We intend to further optimize procedures for isolation of SCs from adult tissues, their detailed characterization and optimize their sorting on the molecular, phenotypic and functional level. Clonal lines will be isolated and protocols for directed ex vivo expansion improved. Various growth factors, especially ones derived from activated platelets preparations will be used. Our goal is to manufacture standardized stem cell products and to thoroughly determine their cell composition – we are especially interested in adult stem cells with broad differentiation potential (VSEL). The collection of autologous HSCs by apheresis will be optimized (UKC, Department for Haematology). With improved cell preparations, larger clinical studies, such as treatment of long bone fractures and the treatment of chronic wounds (UKC, Department for Surgical Infections), heart failure (UKC, Department of Cardiology), treatment of advanced alcoholic liver cirrhosis (UKC, Department of Gastroenterology), and treatment of Chrohn disease complications and fistulas will be carried out.
The final aim of our research programme remains the same: to provide the best medical service in the field of advanced cell therapies to all citizens, i.e. to open the possibilities for replacement of damaged and diseased tissues and thus to achieve high quality of life. By introducing new high end technologies, the proposed programme is also important from a scientific, technical, infrastructural and educational viewpoint.
Significance for science
Advanced cell therapies are one of the main developmental directions of biomedical research in the last decade. The new post-genomic technologies and system biology answered many basic scientific questions. It is predicted that the importance of advanced therapies in medicine in the coming years will grow rapidly. But one very important question is still unanswered - why is the benefit of cell therapies lesser then expected? Our hypothesis is that a major problem lies in the use of non-optimal cell preparations and incomplete clinical protocols. We also believe that the grafts should contain a bigger population of (pluri)potent SCs with a broader developmental potential. These cells could be obtained from the umbilical cord, bone marrow or mobilized peripheral blood of adults.
Our research program consists of two parts: pre-clinical studies and from clinical translation. So far we have shown that this model is extremely successful because it allowed immediate translation of scientific results into clinical practice, and with such novel methods more than 1,000 Slovenian patients have already been treated with success. Further findings from the first part (preclinical part) of our research programme and the development of in vitro procedures for isolation and enrichment of SC with broad differentiation potential will enable the production of better cellular products. New knowledge will also increase our understanding of the adult SCs, which would be an important step in the development of advanced cell therapies. We will define which subpopulations of stem cells are actually present in the HSC transplants and other preparations of SCs, and what composition of the graft will give the best clinical outcome. The new experimental haematology animal models will allow testing of various new ways of haematopoietic stem cell transplantation.
Findings from the second part (clinical research) will be the basis for wider clinical use of different types of SCs and other cells. Our research will answer the question whether and to what extent are the cell therapies superior to the established traditional medical treatments. We expect that a positive outcome of these studies will led to the expansion of indications for advanced cell therapies in the field of ?? degenerative diseases, trauma cases, treatment of nerve damage, gastroenterology cases, cancer and treatment of cardiovascular diseases. The results of our programme will serve both the development of basic medicine as well as clinical medicine that will be able to apply our methodology for the practical treatment of certain diseases. Both parts of research will yield a considerable amount of innovative and publishable scientific data.
Significance for the country
Direct relevance for the economy and society: the European population is aging rapidly and therefore, it is increasingly in need of a new industry called regenerative medicine. Replacement of damaged or diseased tissues will greatly contribute to successful treatment and rapid rehabilitation. Only superior and high quality medicine will grant citizens of Slovenia the effective treatments and high quality of life. Our research programme will have direct and indirect effects on patients and society.
The importance for the industry: Advanced therapies have become a new branch of medicine that in developed countries generates many new jobs. In Slovenia, the development is still lagging behind. Therefore we believe that our proposed research programme will largely contribute to the visibility, development and promotion of medical biotechnology. We estimate that new jobs will be opened as a consequence of the new programme. We can expect a rapid development of intellectual property, new international patents and the formation of spin-offs along the lines of company Educell. We can expect that similar new start-up biotech companies will be established.
The importance for social infrastructure: In the last 10 years, we were aware of the new world trends and we managed to create a solid infrastructure, educated personnel and good achievements in the field of medical biotechnology. This branch of medicine is extremely propulsive even as an industry, because it is environmentally friendly and allows for continuous development of economy, medical and health tourism, and development of University system of education, and of course, of the general science.
Importance for Public Administration, cultural development, and protection of natural and cultural heritage: Our programme promotes an ecologically pure and virtuous activity, based on high technology and knowledge. The programme will be an opportunity for international recognition of Slovenia, since it will help to incorporate Slovenia into international relations, it will open the possibility for collaborations within the EU's Horizon 2020, and it will allow the appropriate education of staff for the further development of the medical profession.
Most important scientific results
Annual report
2017,
2018,
2019
Most important socioeconomically and culturally relevant results
Annual report
2017,
2018,
2019
