Michael Fossel Michael is President of Telocyte

July 17, 2017

Walking Toward a Cure for Alzheimer’s

Sometimes things go wrong, sometimes they go remarkably right.

        In clinical medicine, Swiss cheese theory is a explanation of why medical disasters occur, even if the explanation has a grizzly sort of humor. Basically, Swiss cheese theory says that “all the holes need to line up” for something to get through the cheese and for things to go drastically wrong in patient care. For example, if the physician is a moron (the first hole in the cheese) and orders the wrong medication, then the knowledgeable pharmacist usually cancels the order. But if the pharmacist is also a moron (the second hole in the cheese) and sends the wrong medication to the nurse, then the experienced nurse refuses to give the medication and stops the mistake long before the patient is injured. But, of course, if the nurse is also a moron (the third hole in the cheese) and simply gives the wrong medication, then you have a problem. When all the morons line up in a row, like holes in adjoining slices of Swiss cheese, then mistakes get all the way through the cheese and you have the perfect setting for a medical disaster. Medical errors are rarely the result of a single stunning error on the part of a truly epic moron; medical errors usually take a grizzly sort of teamwork among morons, all working together like clockwork. Swiss cheese theory strikes again.
Oddly enough, the opposite can also happen. If everything lines up in a positive sense then we have innovation, progress, and (very rarely) a miracle or two. For example, to have a success in the case of a biotech company, you need a series of positive events to line up. Over the past few years, that’s exactly what has been happening to Telocyte. While there have been no truly stunning single events that have created a fleeting (if flashy) success, there have been a collection of positive events that line up exactly as they need to. In our case, all the holes are lining up to build toward a successful cure for Alzheimer’s disease.
I first proposed that telomerase could be successful as a clinical intervention in 1996, but my proposal wouldn’t have gotten anywhere if a whole collection of groups and individuals hadn’t continued to move the field along over these past twenty years. From a purely practical perspective, it was the work of CNIO in Madrid (and that of their director, Maria Blasco) that demonstrated a technique that can easily be applied to human clinical trials. Yet, while we saw the potential for human disease, it was our CEO, Peter Rayson, who moved us along in a practical direction. Two years ago, Peter arranged to meet me in Boston, and we founded Telocyte. Our COO, Mark Hodges, joined us and helped shape our program. We had additional support from volunteers, spouses, and researchers, all of whom saw the value and shared our vision. Investors, such as Rob Beers, joined us, asking little and seeing much. We were approached by large global corporations, such as SAP and Amazon Web Services, who offered us support. We partnered with the world’s preeminent biotech law firm, Cooley LLP, who saw the potential and wanted to help. Other investors have come on board, investors who saw what we could do and who agreed with our goals.
Recently, we signed agreements with a major investor and submitted our protocols for FDA review, and we continue to move ahead, steadily and confidently, as we plan for our human trial next year. None of this has been the result of one person, nor even one group. Instead, it has been the result of a continual concatenation of just the right people at the right time. Everything has gracefully, carefully, and steadily lined up, creating an historic opportunity to save lives and rescue human minds. There have been no miracles, no sudden champagne, no instant success, nor wild celebrations. We haven’t seen wonders, but we’ve seen workers. We haven’t seen miracles, but we’ve met milestones. We haven’t had champagne, but now we have a chance.
With every step, a door has opened, people have helped, another step was taken.
And each step brings us closer to curing Alzheimer’s. Walk with us.

April 12, 2017

We Already Know It Works

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:

  1. Animal studies that show efficacy (already done by our collaborators).
  2. Animal studies that show safety (an FDA requirement).
  3. 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.

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