1286th OGM and Open Lecture

Professor John Rasko AO“The Dawn of Molecular Medicine—Gene Therapy: past, present and future”

Professor John Rasko AO
Royal Prince Alfred Hospital and Centenary Institute, University of Sydney

Date: Wednesday, 2 September 2020, 6.30pm AEST
Venue: Zoom webinar
Video recording: YouTube Video

Summary: Over the next five years a possible 900% increase in Gene and Stem Cell Therapy approvals has been forecast. The convergence of substantial incremental technical advances towards combined cell and gene therapy has led to improved clinical outcomes in immune deficiencies, haemoglobinopathies, blindness, immunotherapies and other inherited diseases. An audit of cell, tissue and gene products with marketing authorization in 2018 worldwide identified 44 unique products, 37 of them are cell and tissue therapies (84%) and mainly autologous (55%).

The challenge of realizing the full potential of genetic understanding has been vital in overcoming the hurdles of efficient gene therapy. Since the first human clinical trial using gene technology in 1989, there have been nearly 3,000 approved clinical trials worldwide. The overwhelming majority of human clinical trials involves short-term gene expression or random integration of a therapeutic gene. Emerging technologies require controlled development in compliance with safety, regulatory and GMP requirements.  More precise gene targeting tools were first described in the early 2000s. Targeted gene editing or replacement using Zinc Finger Nucleases or TALENS have been tested in about a dozen clinical trials since 2009. 

In parallel with objectively proven therapies, ‘stem cell tourism’ has become a billion dollar industry with increasing examples of false claims. Embryonic and induced pluripotent stem cells have been mired in controversy and clinical development has been forestalled. We reported an analysis of the global distribution of more than 400 unique businesses marketing stem cell-based interventions. Many of these online entities promote clinical applications of ‘stem cells’ beyond present-day standards of care. These data should be of immediate concern to governments and ethicists being lobbied to amend laws governing the manufacture, distribution and clinical use of human cell-based medical products. Unregulated, untested or unsafe stem cell ‘therapies’ place the field at a difficult crossroad. Blurring the lines that distinguish evidence-based cell therapies from those that are not remains a fundamental public health concern.

Highlights in the clinical cell & gene therapy field will be discussed with special reference to haemophilia, thalassemia, graft versus host disease and cancer.

 Professor John Rasko is an Australian pioneer in the application of adult stem cells and genetic therapy. Since 1999 he has directed the Department of Cell and Molecular Therapies at Royal Prince Alfred Hospital and the Gene and Stem Cell Therapy Program at the Centenary Institute, University of Sydney. He is the President (2018-20) of the prominent International Society for Cell & Gene Therapy.

John Rasko is a clinical haematologist, pathologist and scientist with an international reputation in gene and stem cell therapy, experimental haematology and molecular biology. In over 160 publications he has contributed to the understanding of stem cells and blood cell development, gene therapy technologies, cancer causation and treatment, human genetic diseases and molecular biology.

He serves on hospital, state and national bodies including Chair of GTTAC, Office of the Gene Technology Regulator — responsible for regulating all genetically-modified organisms in Australia — and immediate past Chair of the Advisory Committee on Biologicals, Therapeutic Goods Administration. Contributions to scientific organisations include co-founding (2000) and past-President (2003-5) of the Australasian Gene & Cell Therapy Society; Vice President (2008-12) and President-Elect (2016-18) International Society for Cell & Gene Therapy; Scientific Advisory Committees and Board member for philanthropic foundations; and several Human Research Ethics Committees. He is a founding Fellow of the Australian Academy of Health and Medical Sciences. In 2018, the Board of the ABC honoured him as the sixtieth Boyer Lecturer. He is the recipient of national (RCPA, RACP, ASBMB) and international awards in recognition of his commitment to excellence in medical research, including appointment as an Officer of the Order of Australia.

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