Programme / Thematic Sessions IV. d. The Ethics of Regenerative Medicine

Regenerative medicine is an emerging medical endeavor aimed at restoring tissue function via approaches ranging from small molecular drugs, biological therapies and tissue engineering devices to cell and gene therapies. While it holds great potential and offers the promise of curing intractable diseases, as Volker ter Meulen summarised in his opening thoughts, we have to think about how far we can go and what we can do with these new tools. Two main approaches of stem cell therapy and genome editing require especially focused attention with regard to ethical questions and regulations.

Robin Fears introduced an ongoing project conducted by a working group of EASAC and FEAM, which has been reviewing the opportunities and challenges of the regenerative medicine field. Their report, to be published soon, aims at providing guidelines and recommendations for an up-to-date EU regulatory framework of regenerative medicine focusing on stem cell and gene therapy, and will also serve to inform patients, medical centers and policy makers to protect future recipients of regenerative medical care. Göran Hermeén pointed out that there is an ethical obligation to perform more rigorous research to provide better scientific evidence on the unique tools of regenerative medicine. A fine balance between safety and efficacy must be found, and we have to take risks to make progress in the field. The main ethical concerns are around the uncertainty about the risks to and benefits for patients with few other treatment options, the lack of sufficient verified evidence for informed consent, problems related to information supplies from unverified sources, potential conflicts of interest for medical professionals, and issues of equity and fairness about treatment costs and payments where health service resources are limited. Anne Cambon-Thomsen emphasised the importance of harmonising regulations and the implementation of new technologies in regenerative medicine. Research technologies are far ahead compared to their regulation. Patients’ interests should be first when we are defining regulations. Balázs Sarkadi summarised the latest progress in the stem cell field, highlighting that while cell therapies differ from medical drugs, a similar regulation is required, including validated GLP and GMP protocols. He pointed out that in the United States alone, there are over 200 clinics providing unregulated stem cell treatment with little evidence and vague rationale, with the intent of making a significant commercial gain. We need more time and patience to properly characterise how to best use stem cells for therapies. Beáta Sperlágh summarised the classic principles of medical ethics and their relation to currently emerging advanced-therapy medicinal products. Although the effects of cell and gene therapy are far more complex than classic drugs, similar regulation is required. Elisa Corritore pointed out the main challenges in the stem cell biology field, including the clinical variability of treatments.

The main questions raised in the panel discussion concerned the means of input from different stakeholders in the field and the necessity of taking risks for the sake for progress. As a community, we should not follow the precautionary principle, as this will prevent advances in the field. However, proper ethical and legislative regulation is required to allow the unique treatment methods of regenerative medicine to find their place in medical care.

Rapporteur: Balázs Enyedi, Assistant Professor, Semmelweis University