Libella Gene Therapeutics Plans Human Telomerase Gene Therapy Trial

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.



I wonder what 'quadrillions of gene therapy particles must be produced for each test subject', means? Perhaps they are using nanoparticles rather than a viral vector?

I think it is too black and white to say telomeres are either causal in aging or just an assessment of other causes of aging. Even if most cellular senescence in the human body is premature, i.e. not due to critically short telomeres, shortening telomeres are still likely to be a contributory factor. This is because the shorter telomeres are the slower the cell proliferates, and the higher the burden of other damage it will be carrying. This means that it is more likely to become senescent due to other stressors.

Agree though, let's see what the results are in human testing and then we'll know more.

Posted by: Mark at November 15th, 2017 9:24 AM

I really wish these guys would use the paid model outlined by Alexander Masters in which rich individuals pay to fund people to go on the trial, then recieve the treatment themselves outside the trial:

This allows the use of a placebo group. If the effect being tested for is expected to be large, then a placebo group may not be needed to detect the effect, as a comparison of the trial members clinical endpoints before and after the treatment can be enough to prove something is going on. But even if the treatment has a large positive effect, the side effects may not be large, and a placebo group would be needed to detect and quantify them, or more importantly, prove that side effects in the treatment group are not actually side effects.

This is why companies are not keen to give their unapproved treatments to patients, even in the situation where it might save a sick patient's life. If the patient dies or suffers some negative effect, how do you know if it was the treatment that caused it or just some other problem the patient already had, or just bad luck? If some people in the Libella trial have heart attacks or get cancer (which we we expect to happen to some in the trial group anyway) how can Libella prove that these were not related to the treatment? If there is attention/money to be had by the media telling a tale of how an non FDA approved trial overseas is giving people cancer, then this story will get told, unless the facts to prove otherwise are available.

Another story that will get told is that Libella is just taking rich fools or vulnerable individual's money for a treatment that doesn't work. Having the money go to conducting a trial with a placebo group and without the rich individuals in it will go a long way to countering this story. It would also help if Libella was set up as a transparent charity and not a for profit organisation (I don't know whether or not it is a charity).

Posted by: Jim at November 15th, 2017 5:20 PM

All good points Jim. I think they are shooting for the Moon and hoping for a big result, using Bill Andrews to sell this to the old, super rich, possibly in Asia. I wonder what they are charging.

Posted by: Mark at November 16th, 2017 3:27 AM

If telomere attrition is not a fundamental cause of aging, then I don't know what is!

Telomere attrition is not necessarily in direct proportion to the rate of cell division. Post-mitotic cells like neurons and cardiomyocytes undergo severe attrition, perhaps because telomeres are G-rich and prone to oxidative stress.

Furthermore, the ability of stem and germ cells to resist aging is dependent upon telomerase expression.

Gene therapy is not amenable to amateur self-experimentation! And crowdfunding proper clinical trials is unlikely. Check out Telocyte, a very promising company that aims to do telomerase gene therapy the right way (i.e., to get regulatory approval so that the public can actually benefit from the therapy).

Posted by: Sebastian A at November 20th, 2017 6:47 AM

So is Bill Andrews no longer working with Bioviva? I checked the Bioviva site and he is no longer listed on their advisory board.

From the Libella website FAQ:

"Libella is the only company that is exclusively licensed with Sierra Sciences and Dr. Bill Andrews. If others had his technology, we would not be alone in offering this care."

So then what does Bioviva have, if Andrews is no longer helping them?

Posted by: Eric at November 27th, 2017 3:46 PM

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