A recent report published in The Lancet calls for a coordinated strategy to reform the science and policy behind stem cells and regenerative medicine. The authors describe a combination of specious science, unclear funding models, unrealistic hopes and dodgy private clinics that deceive and exploit patients. This reality threatens regenerative medicine’s social license to operate. Despite increasing interest and an exponential growth in research in the last 10 years, there is scant evidence of the cost-effectiveness and scalability of new experimental therapies.
In other words, is it simply wishful thinking and are companies going bankrupt pursuing a pipe dream?
Regenerative medicine is a broad field that includes stem cell and gene therapy, tissue engineering, and new generation drugs; concepts that encourage the body to ‘heal itself’ by using its own systems to recreate cells and rebuild tissues and organs. It is estimated that globally, regenerative medicine will become a $10 billion industry by 2030. North America and Europe are the largest markets for regenerative medicine, owing to the fact that only wealthy countries are able to bear the cost of the research, but Asia is beginning to become a more significant player as the region seeks solutions to treat an increase in chronic disease and an ageing population. However, the investment required to develop high-quality trials is considerable and although investment in the field is substantial internationally, the therapies do not carry guaranteed commercial success. Compared with conventional drug development, these novel therapies follow a highly uncertain route to market. Significantly, new therapies expose patients to risks, many of which are difficult to predict. Stem cell therapies in particular have become a regulatory battleground as clinicians, scientists and ethicists debate whether the road to the clinic should be shorter.
The Lancet report emphasises the need to conduct rigourous and best quality basic research and to invest in more clinical scientists to help translate research successfully into the clinic. Critically, the public needs to be engaged in open dialogue as a means of bridging the gap between the expectation of patients and the clinical community, and the realities of treatment outcomes. All discoveries should fall into a strong governance framework that is protective of public interest and safety. If adopted, these recommendations may just turn this ‘pipe dream’ into the promise of better regulated, and better delivered, science.
Stem cell therapy's concept is deceptively simple: take cells from a donor and put them into a patient to treat a disease or injury. However, the reality falls far short of the dream.