Event Sponsors:
The Israeli Association for the Study of Diabetes (IASD)
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Over the past century, dramatic scientific progress has been made in the field of Life Sciences which has led to the development and refinement of new research tools and important discoveries in molecular biology. In the last quarter of the 20th century, scientific discoveries have enabled the intensification of knowledge serving the understanding of diseases at the molecular level. As time passes, rapid breakthroughs in genetic research, advances in molecular biology and the knowledge of the genetic origin of metabolic diseases, whether genetic or acquired, malignant, infectious and other diseases, have enabled a deeper understanding of the mechanisms and complex processes that cause diseases. All of this brought about the natural birth of a new medical field: Gene and Cell Therapy.
Gene and Cell Therapy can be seen in a small nuclear acid sequence such as anti sense or whole genes packed in synthetic matter, viruses or even in whole cells. The future widespread application of gene and cell therapy in the treatment of various human diseases requires on the one hand, a deep understanding of the disease, the sick organ, the disease mechanism, and on the other hand, relates to problems caused by the treatment, such as an immune response. Research in Gene and Cell Therapy is interdisciplinary and requires the close and ongoing cooperation between different medical and biological fields over a long period of time, while continuously maintaining humility and also addressing the ethical and legal issues of both man and animal. During the past few years, research in Gene and Cell Therapy has pivoted backwards and forwards, from the preclinical stage to the clinical stage and back again. As a result of the move to the clinical stage, we were once again tossed back to finding answers to basic questions. This, in my opinion, is undoubtedly for the best. Over the past few years, more than 7,000 patients received genetic treatments from a wide range of clinical fields. One example of this dynamic process can be seen in patients who were treated with retroviral vectors for hematological diseases such as X-SCID, ADA-SCID and CGD. In all, 27 patients with these diseases were treated in Europe with retroviral vectors and clinically followed-up for a number of years. After receiving genetic treatment, one of the patients died from leukemia and two others developed blood cancer, received treatment, and are now exhibiting no further symptoms. With the rest of the patients, there was improvement in the quality and length of their lives. Today, many researchers are still trying to better understand the phenomenon of insertional mutagenesis which is the base cause of the appearance of malignant tumors in these patients. It would appear that we are dealing with a field of research where progress is measured by one step forward and two steps backwards, and is characterized by successes on the one hand, and the need to reflect and understand the failures, on the other.
Today, most of the financial support for research derives from governmental and philanthropic groups and only a small percentage from commercial companies. This, I believe, is for the best. Nevertheless, in some of the studies, one would like to see more of an investment from private companies. In Israel today, there are 300 researchers actively involved in the field of Gene and Cell Therapy. All of them have research groups, including students, fellows, technicians and colleagues. The aim of the Society is to help these groups advance their research in the field of Gene and Cell Therapy, mainly through exchange of information, clarification of problems, encouraging openness and expressing scientific opinion through the discussion of scientific and regulatory issues. There is a lot of important, challenging, and complex work ahead of us, demanding a great deal of imagination, creativity, diligence, frankness and love. We hope that with the help of these attributes, we will succeed in healing the sick. There is no doubt that the honest and close cooperation between members of the Society will greatly enhance the development of Gene and Cell Therapy in Israel and abroad.
Eithan Galun
ISGT President
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