Oddly enough, many investors don’t realize how far we are down the road to a cure.
In fact, most people don’t understand why such studies are done and – more to the point – why Telocyte is doing one. Just to clarify: we’re not doing an animal study to prove efficacy. We already know it’s effective in animals.
The reason we do an animal study is because the FDA, quite reasonably, requires an animal safety study in order to assess risks and side effects. Most people assume that animal studies are done to show that a potential therapy works in animals, so that it might work in humans as well. In fact, however, once you have shown that a therapy works in animals, as we have already, then before you can go on to human trials, you first need to do an animal safety study.
Animal studies are done to assess safety, not to assess efficacy.
For an initial human trial, the main question for the FDA isn’t efficacy, but safety. Sensibly, the FDA requires that the safety data be done carefully and credibly, to meet their careful standards. We know telomerase gene therapy works, but we still need to prove (to the FDA’s satisfaction) that telomerase gene therapy is safe enough to justify giving our therapy to human patients. So the question isn’t “Do we have a potential intervention for Alzheimer’s?” (which we do), but rather “Do we know what the risks are once we give it?” We’re fairly certain that we know those risk, but we need to document them rigorously.
In getting our therapy to human trials, you might say that there are three stages:
- Animal studies that show efficacy (already done by our collaborators).
- Animal studies that show safety (an FDA requirement).
- Human trials before release for general use (an FDA requirement).
Telocyte already has good data on the first stage: we know that telomerase is remarkably effective in reversing the behavioral decline seen in aging animals and that the same result will likely occur in aging human patients. In short, we are already confident that we can prevent and at least partially reverse Alzheimer’s disease. The FDA doesn’t need us to demonstrate efficacy: we already have good data on efficacy. What the FDA wants from us is more (and more detailed) data on the probable safety, which we’re about to provide.
While we are now ready to start on the FDA animal safety trial. Doing our FDA animal study isn’t a way of showing that telomerase gene therapy works – which is already clear from animal studies – but a detailed look at side effects, preparatory to our having permission to begin human trials next year.
Telomerase therapy works.