February 17, 2015
The Japan Prize Foundation recently awarded Alain Fischer, director of the Imagine Institute, and Theodore Friedmann the Japan Prize for their pioneering work on gene therapy. Both have been instrumental in bringing the cutting edge technology of gene therapy currently pursued by researchers and pharmaceutical companies as treatments for genetic disorders. Prof. Friedmann, considered the “father of gene therapy”, overcame many adversities to propose the concept of gene therapy. His laboratory worked tirelessly to demonstrate the effectiveness of hematopoietic stem cell gene therapy in an animal model during the 1970s. The work that Dr. Friedmann pioneered has contributed to the development of core technologies surrounding today’s transgenic vectors. Not only did Dr. Friedmann pioneer gene therapy, he is also at the forefront of addressing the ethical issues that circumscribe this field.
Appropriately, Prof. Fischer was also awarded the Japan Prize for discovering the world’s first clinically proven gene therapy for children with fatal X-linked severe combined immunodeficiency (X-SCID). Along with his colleague Marina Cavazzana-Calvo, Prof. Fischer published the first account of the breakthrough clinical success of this therapy in early 2000. The effectiveness of gene therapy was shown to surpass traditional hematopoietic stem cell transplantation. Despite early setbacks, Dr. Fischer identified the random insertion into a proto-oncogene gene drug that causes leukemia in some patients and continued to work on validating the safety and effectiveness of this therapy.