My attention was recently directed to another new group planning patient paid human trials of telomerase gene therapy. This is a company associated with Sierra Sciences and the RAAD Festival crowd, meaning the Life Extension Foundation principals. These folk have of late started to fund a number of interesting efforts, such as the Betterhumans senolytics trials. This is another in that series.
Is telomerase gene therapy a useful treatment for aging? In mice it extends life span, most likely through effects such as greater immune activity and greater stem cell activity, but possibly also via other mechanisms. Telomerase acts to lengthen telomeres at the ends of chromosomes, but it also has a range of other functions, some of which might positively impact mitochondrial function. Average telomere length in tissues falls with age: it is a function of the rate of cell division, as telomeres shorten every time a cell divides, and stem cell activity, as stem cells produce daughter cells with long telomeres. So telomere length is very much an assessment of some of the processes of aging, not a cause of aging. In turn, telomerase gene therapy is not a means of targeting the causes of aging - rather, it is one of the more effective classes of compensatory treatment identified to date, alongside forms of stem cell therapy.
Whether telomerase gene therapy will have the same sort of risk and benefit profile in humans as it does in mice is something of an open question. Mice have very different telomere dynamics in comparison to humans, and the risk of cancer may well be quite different as well. Counterintuitively, in mice that risk actually appears to be reduced by introduction of telomerase, though the mechanisms involved are not well understood. We might hypothesize that increased immune system efficiency in removing potentially cancerous cells counterbalances the telomerase-induced tendency for those cells to become more active. Still, how do you find out other than by trying? Making the attempt is the most cost-effective means of obtaining human data.
Our mission is to reverse aging and cure all age-related diseases, starting with Alzheimer's. Libella Gene Therapeutics has exclusively licensed the technology of Sierra Sciences to conduct a human research project. We believe we have the scientist, the technology, the physicians, and the lab partners, all of which are necessary to get this done. By activating telomerase, we hope to lengthen telomeres in the body's cells. To have an effective delivery system for the telomerase to reach every cell in the body, quadrillions of gene therapy particles must be produced for each test subject. The production of enough gene therapy particles to treat one person takes anywhere from four months to a year to complete. Because of the demands on production, we will have a limited number of tests available. We anticipate having around 50 spots over the next 12 months.
We believe the most expedient way to test revolutionary evidence-based technology, such as gene therapy, is a pay to play model. The FDA passed legislation in 2009 allowing for patients to pay for their care when other viable options are not available. Libella Gene Therapeutics (LGT) strongly believes an informed choice is a right, not a privilege. LGT believes that "pay to play" is ethical. The data has continued to mount that telomerase activation and lengthening of telomeres may be the most exciting and disruptional breakthrough in the history of medicine. LGT is committed to bringing telomerase therapy to the world.
Today the majority of human clinical studies are performed outside of the United States. 65% of clinical studies are performed off shore. Typically it is cheaper, quicker, and involves less regulation. LGT believes it is most ethical to conduct our studies outside of the United States where we can move faster, and at a lower cost, as long as there is no reduction in quality or safety for our study participants.