We asked NFXF board member, scientific advisor, doctor and clinical researcher Dr. Elizabeth Berry-Kravis to comment on the challenges inherent in a clinical trial. Here is her response:
When a lab experiment is being done for the first time it often has to be done six or more times before everything is finally correct and it finally works reproducibly. That’s distressing and frustrating, but this is actually more the rule than the exception. For a clinical trial the stakes are so much higher. It’s not just distressing and frustrating for the researchers, but it also causes real disruption and agony in the lives of real people who are a part of these trials. So when we don’t “get it right” the first time everyone suffers. Yet it’s almost impossible to get it right the first time in new science ground like this, and even tougher because the FDA and commercial interests do not want to let us have six tries to find the right way to do the experiment and get a reproducible result like we would in the lab. So we are going to have growing pains. We can’t have real targeted treatments for Fragile X syndrome that will improve lives more than current supportive treatments without doing science that’s totally new and going where no clinician, researcher, or the FDA has gone before. Short of a science miracle (which I, Seaside and families had hoped for), we won’t get everything right the first time. We will stumble and we will fall and we have to just get right back up and be wiser from our mistakes and do it over and over again until it’s right. And every time it’s not just us that falls – it’s all of the families who are our partners. So it’s going to hurt and we feel bad that families have to go through this pain. But that is the nature of making progress in science and you must remember that without you we will not make it to the goal of developing new treatments. (We can’t ever prove it works without you) So we have to ask you to go through this process with us. It’s tiring – maybe even horrifying – to think of the persistence and resilience we will all need as partners to get through all the falls and scrapes as we eventually get over all the hurdles to the finish line!