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December 15, 2014

by Mark Dunning

There are several phases of a clinical trial but phase I is the hardest. It’s that terrifying and exhilarating moment when a potential treatment that has only been tried in animals is tried in a human being for the first time. Phase I is often referred to as a safety trial because the main purpose is to find out if the treatment is safe in human beings. The risks go from something minor and reversible, like a rash that goes away when the treatment stops, to something horrible like permanent damage to vital organs and even death. It is also done to determine the dosage of a particular drug/treatment that is safe. Note that nowhere in this paragraph is an improvement in the condition mentioned. That’s not the goal of a safety trial. Phases II and III test efficacy. Improvements in the condition in a phase I trial are secondary.

A picture of the process of coming up with a cure

Research Continuum

Often participants in phase I clinical trials are people in advanced stages of a disease. In the case of vision treatments, these are usually folks who have lost their vision. The idea is that if the treatment turns out to be harmful, researchers would not be damaging useful vision in the process. 

Participation in a clinical trial often precludes a participant from joining a future clinical trial. A trial, after all, is intended to replicate how the treatment will work in a typical person. A person who has participated in a previous clinical trial is not a good measure of a typical person. The previous trial treatment might skew the results of the next trial.

Gene therapy is the first Usher specific treatment to reach clinical trial. Gene therapy rescues living cells. It does not replace cells that have already died. In other words, it will offer little improvement to people in advanced stages of the disease. So those that participate in phase I of clinical trials do so with the knowledge that it will be unlikely that the treatment will help them significantly.

Stop and think about all of that for a moment. The people participating in phase I clinical trials are willfully trying a potentially dangerous treatment. They have an advanced case of the disease but know there is little hope that the treatment will help them. And they are choosing to do this at the possible expense of participation in a future trial. 

The phase I trials have begun for UshStat, the Usher 1b gene therapy. My daughter has Usher 1b. These trials affect me personally. My daughter’s future depends on the participation of people in the phase I trials. 

I know the doctors involved and know they are beyond reproach and yet when I weigh all I know against the requirements, the risks, and the benefits, I can’t say that I would be willing to participate. Not in phase I. Probably not in phase II. Call me when you get to phase III. Unfortunately, we never get to phase III without phase I. That means if there are going to be treatments, someone has to step up, in the face of all that I have described, and choose to participate. Someone I don’t know has to be willing to sacrifice everything for little personal gain to save my daughter. 

The confidentiality of the process means I’ll never know the name of those that do participate. I’ll never shake their hand. I’ll never collapse in gratitude before them. They will never know what they mean to me, to my daughter, to the hundreds of thousands of people with Usher syndrome.

Great personal sacrifice in anonymity for the good of others. Is there a better description of a hero than that? 

There are very few true heroes in this world. I know I am not one. I’m just a regular guy. The vast majority are like me. That’s why we need everyone to join the International Usher Syndrome Registry. We need a big pool of people to identify those few out there willing to participate in a phase I trial. It’s hard to find heroes. 

If this reads like a thank you, it is. To those that have participated in a phase I clinical trial or will in the future, you are, and forever will be, my heroes. It’s hard to imagine a more noble calling than to sacrifice, anonymously, for the greater good of the community. To save a child you’ll never meet. There is nothing I can offer in return that is of equal value. I only hope my words illuminate your sacrifice for others. It’s meager, but it’s the best I can do. After all, I’m no hero.

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