Regenerative medicine, and the underlying stem cell technology on which it is based, offer considerable hope to patients suffering from trauma and acute or chronic disease. Despite this, regenerative medicine can be highly controversial in terms of claims and weaknesses relating to safety and efficacy, the regulatory aspects, the ethical and social aspects, the commercialisation of stem cell, technology and – most importantly – the scientific and medical basis of the proposed technology. Regenerative medicine is in its infancy and we must all be very aware that at present, hype and hope are the backbone of the technology. When safety and efficacy are the backbone, then we will truly be in a new trusted area of clinical practice which patients can access with confidence.
The issue of patient safety and treatment efficacy in regenerative medicine is arguably the most important factor in the future of the technology and at present, we are in a position of extremes. This is because technology such as bone marrow stem cell transplantation, peripheral blood stem cell technology (using mobilised bone marrow stem cells) and cord blood stem cell transplantation are practiced globally with a high level of safety and efficacy. There are many centres of excellence around the world where experts carry out these transplants with extensive regulatory guidance. Patients enjoy optimised safety and efficacy when they are treated by these experienced teams in a perfect setting.
In stark contrast, there are a rapidly increasing number of stem cell-based ‘treatments’ for which there is little or no safety and efficacy data. These are often provided by stem cell ‘clinics’ and prey on vulnerable patients who are often looking for a ‘cure’ when traditional medicine has been unable to help. This is the dark side of regenerative medicine. The safety and efficacy of treatments offered using stem cell-based regenerative medicine is defined and controlled by the relevant regulatory authorities. Once again, as with safety and efficacy, the regulation of regenerative medicine technology falls into two extremes.
The first extreme is in countries such as the UK and USA, where regulation is well developed and therefore patients are protected and can undergo regenerative medicine treatments with confidence. In the UK, for example, there is the Human Tissue Authority (HTA), the Human Fertilisation and Embryology Authority (HFEA) and the Medicines and Healthcare Products Regulatory Agency (MHRA). These organisations come together to regulate every aspect of stem cell technology, making the UK one of the safest places in the world to be treated using regenerative medicine technology.