Gene therapy is a fascinating and growing research field of translational medicine. The basic biological understanding of tissue function, cellular events, metabolic processes, stem cell function, are all linked to the genetic code and to the genetic material in all species.
In mammalians as in more simple creatures, each and every phenotype structural characterizations, function and probably behavior is dependent on the special nature and timing of genetic material and events. In altering the genetic material of somatic cells, gene therapy may correct the underlying specific disease pathophysiology.
In some instances, it may offer the potential of a one-time cure for devastating, inherited disorders. In principle, gene therapy should be applicable to many diseases for which current therapeutic approaches are ineffective or where the prospects for effective treatment appear exceedingly low. Our desire for deep understanding of human nature will encourage us to develop new therapeutic platforms in the years to come.
To this end, The Goldyne Savad Institute of Gene Therapy at the Hadassah Medical Organization was established in 1999 thanks to a generous contribution from Ms. Goldyne Savad. The Institute is headed by Prof. Eithan Galun, M.D. The Institute was recognized by the Ministry of Science as the "Gene Therapy National Knowledge Center."
The rationale for gene therapy in human disease:
The concept that gene transfer might be applied to treat disease stems from the spectacular advances of the past two decades in the area of recombinant DNA technology. Humans have some 35,000-40,000 genes each. A defect in virtually any one can lead to disease. Human gene therapy involves inserting a repaired gene into the DNA of a patient to replace a missing or defective gene. Day by day, we get closer to a time when doctors can anticipate and treat the root cause of illness, rather than merely struggle to keep up with its symptoms. The potential use of gene therapy to treat disease is thus a natural extension of recent significant biomedical research.
In the coming decade, it is foreseen that molecular therapy in general and gene therapy in particular will be used to treat patients on an individual basis. Hadassah has chosen to take the lead in Israel's thrust into this new arena of medicine.
Role of the Institute
The Goldyne Savad Institute of Gene Therapy has successfully recruited topnotch scientists with high scientific achievements, each of whom is afforded full scientific independence and is responsible for the development of her/his research. Cross-fertilization of ideas among these scientists not only at the Institute but with scientists from other institutions and disciplines has culminated into important scientific activities.
Research: Research at the Institute covers a wide range of diseases along three main tracks: cellular gene therapy, viral vectors and non-viral vectors. In addition, the Institute is engaged in the development of research and biotechnological tools for gene therapy, which we hope will facilitate the use of gene therapy in the clinical setting.
A substantial proportion of research at the Institute is done in cooperation with researchers from the Hebrew University, Weizmann Institute of Science and with researchers from other research centers in Israel and worldwide. Thus, the Institute provides the environment and activities necessary to facilitate cross-fertilization of ideas through the creation of an interdisciplinary community of researchers supporting one-another in the conception, initiation and development of research projects.
Translation: The mission of the Institute, as recommended by the Scientific Steering Committee and adopted by the Director of the Hadassah University Hospital, is to translate the fruits of laboratory research into therapies that will directly benefit patients. Hadassah's research laboratories are an inseparable part of the hospital's clinical department. The Institute is strategically placed to transfer new knowledge from the laboratory to the patient and back to the laboratory.
Education: The Institute seeks to train a new generation of scientists to explore the common elements of genetic-based disorders and to extend the concepts of basic genetics to their diagnosis and management. The aim of the Institute is not only to provide education to future scientists, but we expect our graduates to play leadership roles in the field of gene therapy. The Institute provides education and teaching to students pursuing M.Sc. or Ph.D. studies.
For example, the Institute is a major player in the research training of undergraduates, graduate students, medical students and post-doctoral fellows. Presently, the Institute has 28 students (graduates and postgraduates) working with the PIs on different research projects. Because of the overlap in research interests among faculty, the trainees have the opportunity to interact with each other and with preceptors within a program representing a wide range of interests in gene therapy, from the most basic to the applied sciences, enhancing the breadth and depth of their training experience.
In an effort to expedite clinical application of gene therapy, the Institute has adopted a number of simple general guidelines:
- Recruiting Principal Investigators (PI's) with high scientific achievements, each of whom is afforded full scientific independence, and is responsible for the development of her/his research group;
- Focusing on areas in which the PIs have proven experience and expertise;
- Targeting and solving specific gene therapy bottlenecks by developing new technologies
- Conducting a tight collaborative effort between the groups.
In order to afford cross-fertilization of ideas on an international level, The Israeli Society of Gene Therapy was established on May 2002 as a medical research society which promotes studies in the field of gene therapy. The ISGT conducts annual meetings in the field of gene therapy. For more information click www.isgt.org.il.
The institute comprises three independent units, each focusing on a different stage in the development of a given gene therapy project:
The research laboratories currently accommodate 11 independent research groups. The laboratories conduct in vitro and in vivo studies, including the development of animal models and assessment of platform technologies.
The National-Hadassah gene therapy production facility. Once a project achieves reasonable maturity with a proven potential in an animal model it moves to the production step at the National-Hadassah Gene Therapy Production Facility. This facility is able to produce molecular therapeutics, including proteins, DNA, viral vectors and cell therapeutics, all meeting the current good manufacturing practice (cGMP) guidelines of the EMA and FDA. The facility is able to produce three lines of biological therapeutics simultaneously. Additional projects awaiting the production stage include the generation cGMP human embryonic stem cells for future research and potential clinical use, and a multiple sclerosis cell based vaccine project. The products will later be assessed in toxicological studies and applied in phase I clinical studies.
Clinical Research Ward. - When the production process is completed, and the investigator's new drug file is approved, the product enters the clinical stage. The clinical Research Ward is currently conducting a number of Clinical studies.. Some phase I studies are part of research projects conducted by investigators at the institute. The facility also supports other research groups both at the Hadassah medical center and elsewhere.
GMP laboratory (English) (Hebrew)
The GMP world class laboratory in Israel is located within Hadassah University Hospital campus with easy access to the Hospital labs, Clinical Ward, P3 Labs. This arrangement provides a unique key advantage of facilitating collaborative networks and accelerating the manufacturing for translational research associated with Phase I/II clinical trials. With the establishment of the new Biotechnology Park within the Hadassah complex, companies can rent laboratory and office space, thus creating a unique opportunity for the investigator to personally be involved in all phases of development from production at the GMP facility to clinical trial. The head of the GMP Laboratory is Linda Rasooly, Ph.D. with broad skills and experience in development projects and manufacturing of pharmaceutical preparations. Investigators are welcome to bring their own personnel to be trained and work with Dr. Rasooly and experienced technical GMP specialists to ensure fast and efficient development from concept to proof-of-principle.
For further information about GMP services, contact:
Phone: +972-2-677-8270, 677-6793