Advancing Stem Cell Therapies: Balancing science, clinical trials, regulation and consumer demand
31st May 2016
Victor Chang Cardiac Research Institute
Conjoint Associate Professor of Medicine John Moore, St Vincent’s Hospital, Clinical Haematology Assoc. Prof John Pimanda, Adult Cancer Program, UNSW
Dr Mark Young, Australasian College of Sports Physicians, Brisbane
Dr Julian Clark, Head of Business Development, Walter and Eliza Hall Institute of Medical Research
Prof Stan Gronthos, Centre of Stem Cell Research, University of Adelaide
Duncan Wallace, Executive Director and CEO of Spinal Cure Australia
Prof Richard Boyd, CSO Cartherics Pty. Ltd.
On the evening of Tuesday 31st May, The Australasian Society for Stem Cell Research hosted its third Discussion Panel at the Victor Chang Cardiac Research Institute, sponsored by Lonza and BioMeurex. The Hot Topic ‘Advancing Stem Cell Therapies: Balancing science, clinical trials, regulation and consumer demand’ attracted an audience of about 60 people from all stages of careers, including PhD students, post-docs, academic researchers, clinicians, industry and the media. The panel, discussed the pros, cons, expectations and realities surrounding the use of stem cells for cellular therapies – a hot topic of interest amongst both the medical science community and the general public. The discussion began with a presentation from Dr. Mark Young who reviewed position statements from regulatory bodies. This followed a discussion how the various regulatory statement would benefit patients. The debate covered the challenges of commercial versus research based StemCell therapy clinical trials. It was argued that ‘innovation in clinical practice is responsible for 80% medical discoveries compared to the clinical trial process is only responsible for about 20%. Clinical innovation – whether it’s a clinician trying a new clinical procedure for the first time – is a very important part of being a physician, but unfortunately it could be used to exploit patients”. This statement is particularly relevant for stem cell therapies, given the number of clinics providing unproven stem cell therapies. The question was raised if four stages of clinical trials are necessary? The Japanese legislation now states that once a phase 2 clinical trial has been completed and proven patient’s safety and effectiveness, that the company may proceed to market the Stem Cell therapy product. Can Australia move towards the way the Japanese operate in terms of clinical therapies in order to get therapies to patients faster? Responses from the panel were generally supportive, but realistic: “In theory, it looks promising”, “…if it encourages good data collection and patient monitoring”, “if clinical trials make it past phase 1 and 2, they have a safety track record, then maybe it is sufficient to take a leap of faith!” Mark Young provided data and raised concerns that often commercial interest distorts the truth by companies not publishing negative trial results. The panel agreed that transparency should be mandatory for any clinical trial. Any, regardless if it is negative or positive procedure, results and side effects should be recorded and made public. Further, concerns were raised that phase one and phase two usually deal with small numbers of patients and are seldom subjected to randomized double blinded procedures. Hence pharmaceutical companies often have to abandon the development of a drug in the trial three phase. The question was raised that if Australia was to follow the Japanese model, would that raise the potential risk of money versus evidence? There was some agreement that evidence base medicine was needed in Australia, however the discussion always returned to the challenge how to manage cost and patients benefits. Opinions were divided if patients enrolled in clinical trials should pay a service fee. Some members, including the audience were strictly against patient having to pay, some felt a small service fee to cover cost was acceptable, while others felt that a cost was acceptable if patients had been provided informed consent.
In summary, Australia is at the forefront of stem cell research – we have good technologies and solid research, but the general consensus of the night was that Australia lacks funding in a market-driven industry. Bridging the gap between the private, academic, industry and government sectors will be imperative to overcoming this issue; investing more into primary research is fundamentally important in getting better clinical outcomes; cross-disciplinary research is crucial because we just don’t understand enough of the science yet, and well-designed clinical trials and clinical innovation will move stem cell research from the bench to the bedside faster, more safely and more efficiently. To explore the topic of the evening further, it has been suggested to discuss and present data of real life examples of clinical trials and cell based therapies and opposing approaches, as well as the ethical and legal issues.