About a month ago, Seattle-based biotechnology company BioViva USA Inc. claimed to have successfully administered gene therapy to lengthen telomeres in an attempt to reverse ageing. The first patient of this therapy was none other than the CEO of the company herself, Elizabeth Parrish. It was stated on the company’s official website that the therapy had “reversed 20 years of normal telomere shortening” (1).

A closer look at Telomeres

Telomeres can be visualized as the caps at the ends of long, thread-like DNA molecules that function to protect the chromosomes. The telomeres themselves are synthesized by telomerase – the enzyme that makes telomere DNA.

It was shown that when telomerase activity was high, telomeres retain their length, which subsequently delayed cellular senescence. On the other hand, when the telomeres become shorter due to low telomerase activity, the cells age, and subsequently die.

The discovery of this fundamental cell mechanism was so groundbreaking that it had stimulated the development of a new range of therapeutic targets. The scientists who discovered the telomere DNA sequences and who identified telomerase were awarded the Nobel Prize in Physiology or Medicine in 2009 (2).

Subsequent telomeres-based research

The discovery of telomeres’ function to protect cells’ viability has prompted much research into this area with hopes that these new findings could be utilized to treat diseases such as cancers, or to reverse ageing. Multiple studies have successfully increased the length of telomeres, thereby increasing the life cycle of cells. One such study was conducted by scientists at the Stanford University School of Medicine, whereby they used a modified messenger RNA to extend the telomeres (3).

However, thus far, none of the therapies involving telomeres-lengthening have progressed beyond the pre-clinical phase, i.e. laboratory testing. Till date, no study has been conducted on humans to investigate either the safety, or the efficacy of such therapies, and it will be difficult for the scientific community to conclude if such treatments would be possible at this stage.

BioViva’s Gene Therapy research

BioViva’s official announcement on the 21st April 2016 only stated that the gene therapy was targeted to lengthen the telomeres of Parrish’s leukocytes, which appeared “unusually short for her age”. The therapy was administered in September 2015, and within approximately 7 months, tests have shown that her telomeres had been “lengthened by approximately 20 years” (1).

The announcement has had mostly skeptical reception, due to very little scientifically credible data and backing offered by the company.

"The experiment seems likely to be remembered as either a new low in medical quackery or, perhaps, the unlikely start of an era in which people receive genetic modifications not just to treat diseases, but to reverse ageing", wrote Antonio Regalado, a Senior Editor of the MIT Technology Review (4).

Other researchers also stated that there is still no convincing evidence to support the causal link between lengthening telomeres and increasing longevity in clinical cases, "If it's like grey hair, dyeing your hair won't make you live longer", said Dr. Dana Glei, from Georgetown University, Washington, D.C. (5).

However, if BioViva’s claims turn out to be well-supported by valid, scientific data, they represent a whole new frontier in efforts to reverse the irreversible.

However, Parrish’s therapy has also raised some serious questions about the legality and ethics of testing a new therapy without proper regulatory supervision. It remains to be seen if the bold claim made by BioViva will remain standing amidst scrutiny by the scientific community, or if it ends up being dismissed as a disastrous marketing campaign. MIMS


References:
1. BioViva USA Inc. First Gene Therapy Successful Against Human Aging [Internet]. 2016 [cited 2016 May 15]. Available from: http://bioviva-science.com/2016/04/21/first-gene-therapy-successful-against-human-aging/
2. Nobelprize.org. he Nobel Prize in Physiology or Medicine 2009 - Press release [Internet]. Nobel Media AB. 2014 [cited 2016 May 15]. Available from: http://www.nobelprize.org/nobel_prizes/medicine/laureates/2009/press.html
3. Conger K. Telomere extension turns back aging clock in cultured human cells, study finds [Internet]. Stanford Medicine News Center. 2015 [cited 2015 May 15]. Available from: https://med.stanford.edu/news/all-news/2015/01/telomere-extension-turns-back-aging-clock-in-cultured-cells.html
4. Regalado A. A Tale of Do-It-Yourself Gene Therapy [Internet]. MIT Technology Review. 2015 [cited 2016 May 15]. Available from: https://www.technologyreview.com/s/542371/a-tale-of-do-it-yourself-gene-therapy/
5. Grens K. First Data from Anti-Aging Gene Therapy [Internet]. The Scientist. 2016 [cited 2016 May 15]. Available from: http://www.the-scientist.com/?articles.view/articleNo/45947/title/First-Data-from-Anti-Aging-Gene-Therapy/



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