Have you wondered what it might be like to participate in a clinical trial for Rett Syndrome. I sure did, so I sat down to speak with Mickie McCool about her experience taking her daughter Ellie to the University of Alabama to participate in the NNZ-2566 trial. Today I am really excited to share her story with you.
When the confirmation first came, Mickie was over the moon happy and excited that her daughter was accepted into the trial. Then she thought, wait, what?!?! It was a little scary. After the initial shock Mickie sat down and described to me that it just felt right in her stomach. She had known the doctors that would be doing the trial for years and trusted that loved her daughter immensely and that Ellie would be in good hands.
In the days leading up to their first trip there was a lot to manage. On top of an already full day-to-day life Mickie had to prepare to fly alone with Ellie. She was concerned about what type of physical and emotional toll this process would take. Would Ellie get upset and cry inconsolably on the airplane? Would one of them get sick? The day came and many of Mickie’s fears were realized when they both got a horrible stomach bug on the way home from their first trip but somehow, they made. What she feared most Mickie overcame and with each trip it got a little easier.
With the drama of the screening visit behind them, they headed back down, this time in a car to start treatment. Mickie described the process as surreal. The doctors counted down 3-2-1 go and with great precision the EEG and the treatment started in synchronization. Due to the double-blind nature of the trial neither Mickie or the doctors know if she was getting the placebo or the treatment, but either way, the process was awe-inspiring. In that moment, Mickie knew that she was doing what she could, to make a difference, maybe for her daughter but definitely for all the girls out there yet to be diagnosed.
After the initial excitement it was a rather mundane process of data and being in a hospital. There was the bonding that happens between a mother, daughter and friends that can only take place when you are going through something like this, it changes people and brings them together. Ellie’s team rallied around her and that was something special that wouldn’t have happened without this process.
Now that it is over, Mickie wonders, was it the drug, was it the placebo? She wonders what the data will show. Will there be a phase 3? If there is, how will they afford it? There are still a lot of questions but some things are certain. They made a difference. Ellie was part of a possible solution, her participation in this trial will make a difference for those living with Rett Syndrome in years to come. I asked Mickie what she would say to somebody thinking about enrolling in the same trial and she gasped and paused seriously. Her response blew me away, “I would tell that it was a very rewarding to have the chance to help not just your own child but all of the girls. Look at it from every angle, talk to the person in your heart.”
My sincerest thanks to Mickie for sharing her story of hope, to Ellie for enduring all of the testing and to each of the families that have bravely stepped up to participate in this trial.