Does Passionate CEO and Illegal Clinical Research Lead to Good Science?
[Posted on: Thursday, 28 April , 2016]
This week, the Seattle-based gene therapy company, BioViva, made a bold claim to have invented the world’s first “successful” gene therapy for aging. The evidence for efficacy and safety of its product was generated in a manner that raises serious concerns of ethics, good scientific practices, and regulatory oversight. What is clearly a publicity stunt may be considered illegal research by most regulatory agencies in the World. However, by using this unconventional method to test its product, BioViva may give ideas to other similar small biotech companies that need critical news to attract investors. The company is developing a genetic telomerase therapy for aging based on genetically increasing the length of telomeres (ends of chromosomes). It is also working on at least 10 other disease areas using their platform technology. It is unclear, from the publicly available information, the status of research conducted by BioViva and specific non-clinical studies conducted with their lead gene therapy products, or if they have optimized their manufacturing systems, a key issue with gene therapy products. The CEO of the company, Elizabeth Parrish, traveled to Columbia to get injected with two investigational gene therapy products invented by BioViva, one for aging and another for increasing muscular mass. Six months post-injection, tests showed a 9% increase in the length of telomeres. The company claims that Ms Parrish does not suffer from any adverse effects. There are many obvious concerns with this experiment. There does not appear to be a formal clinical trial or any independent review of the experimental plan or the results. The fact that Ms Parrish traveled to Columbia for these injections shows that she was very aware that such research will not be sanctioned under the US laws or any other Western country. It also shows that BioViva does even have sufficient research data to initiate discussions with any regulatory agency. BioViva has not announced if it supported by any public funding or grants. The results did generate the desired impact in the research community with several researchers applauding the company for using an out-of-the-box approach to generate proof-of-concept (POC) data while many objecting to the credibility and reliability of the evidence presented. There are obvious bioethical issues of injecting a human being with a product of unknown safety profile for questionable claims of efficacy clearly drummed up for raising investment for the company. The company announced that it is seeking a “regulatory partner” presumably outside the US FDA and similar regulatory agencies. There exists several processes within the US and EU laws that allow testing of such novel therapies with low burden of non-clinical and manufacturing information in support of a POC study. FDA has allowed numerous POC studies with gene therapy products over the last 20 years. So, if companies believe they have promising ideas rooted in good science, they should seek a regulatory strategy with long-term goals rather than sensationalizing their research to make a quick buck that may hurt the field in the long run. Flaws in the experimental design not only kill the credibility of the results but may kill the entire area of research should there be accidents which can’t be adequately investigated owing to the flawed design.