A proposal to reanimate brain dead patients using a combination of treatments has been killed by the Indian Council of Medical Research due to concerns over regulatory lapses (1). Whether it will stay dead is unclear.
The ‘ReAnima’ trial investigators proposed mixing direct transcranial laser pulses and nerve stimulations with injections of stem cells and regenerative peptides, hypothesizing this treatment combination could return brain dead subjects to a minimally conscious state. Other medical experts note a lack of compelling evidence that simply combining these treatments will successfully restore lost brain function (1). Nonetheless, perhaps an argument that it is worth a try will still win the day. However, the chief executive officer of a biotech firm involved in the ReAnima proposal indicated that the trial would be pursued outside of India if necessary.
Threats to move biomedical research operations to locations where regulations are less restrictive have been issued before. A recent editorial in the journal Nature (2) revealed that the researchers proposing an ornithine transcarbamylase gene therapy trial disagreed with most of the conditions set forth by government overseers asserting they would increase study costs while adding no additional benefits. Expressing concerns that their work was being held to standards that are not applied to other studies and the urgency of the situation, the Chief Scientific Officer of the sponsoring company indicated they would conduct these trials overseas if necessary.
Such warnings are not necessarily empty talk. Economic considerations have encouraged corporations to shift clinical trials to less expensive locations (3). The complex regulations and monitoring demands in countries like the United States which allegedly increase the costs and time to develop products may provide additional motivations to outsource trials. Although companies seeking to market products in the U. S. have incentives to maintain high standards, transferring studies offshore raises concerns about data transparency as well as the adequacy of safety and subject protections (3). Nonetheless, despite great uncertainties regarding efficacy, frustration with the pace of genetic therapy development in the U. S. led one biotech company executive to reveal she had received treatments to combat aging and muscle degeneration in a foreign country (4).
Corporations answer to market forces and shareholders while regulatory agencies and governments move slowly in response to a different set of forces. Corporate aspirations thwarted by regulatory restrictions may sometimes be circumvented by a change in geographic location. The recent announcement of the first 3-parent baby was greeted with some concern that the team performed the procedure in Mexico, a situation that was probably worsened when one of the principals involved in the effort made the regrettable comment indicating at that location “…there are no rules” (5). A new report suggests that stem-cell treatment activities resulting in conviction on criminal charges and a prison sentence in one nation do not necessarily preclude the same work being performed in another country (6). Global biomedical research has become the new wild West of the 21st Century.
Biotechnology has enormous potential and an equally powerful allure. Anxious for miracle cures and frustrated that scientific studies can take so long due to regulatory hurdles, some entrepreneurs will seek, and find, shortcuts. Desperate patients seeking help are well advised to weigh the risks and possible benefits with the greatest of care.
(1) P. Pulla. 2016. Experiment to Raise the Dead Blocked in India. Science, 14 November 2016. http://www.sciencemag.org/news/2016/11/experiment-raise-dead-blocked-india
(2) The Editorial Board. Gene Therapy Trials Must Proceed with Caution. Nature, 28 June 2016. http://www.nature.com/news/gene-therapy-trials-must-proceed-with-caution-1.20186
(3) S. W. Glickman et al. 2009. Ethical and Scientific Implications of the Globalization of Clinical Research. The New England Journal of Medicine 360:816-823. http://www.nejm.org/doi/full/10.1056/NEJMsb0803929
(4) A. Regalado. 2015. A Tale of Do-It-Yourself Gene Therapy. An American biotech CEO claims she is the first to undergo gene therapy to reverse aging. Judge for yourself. MIT Technology Review, 14 October 2015. https://www.technologyreview.com/s/542371/a-tale-of-do-it-yourself-gene-therapy/
(5) S. Reardon. 2016. ‘Three-parent baby’ claim raises hopes — and ethical concerns. Nature, 28 September 2016. http://www.nature.com/news/three-parent-baby-claim-raises-hopes-and-ethical-concerns-1.20698
(6) A. Abbott. 2016. Disgraced stem-cell entrepreneur under fresh investigation. Nature, 14 November 2016. http://www.nature.com/news/disgraced-stem-cell-entrepreneur-under-fresh-investigation-1.20985