In November 2023, board member Teri Reed sat down with Jerrod Watts, the first GSD1a gene therapy patient as he shared his inspirational experience being patient 1 of phase 1 human trials, which began July 24, 2018….

Q: What made you decide to participate in the GSD 1A gene therapy trial as patient 1?

A: When I got an email questionnaire asking about my interest in participating in a gene therapy trial, I excused myself from the marketing conference I was attending so I could respond immediately to the request! When I was a child, my goal in life was to bring about a change in the treatment of GSD so no one in the community would ever again fear dying in the middle of the night due to a missed cornstarch dose. I was so excited about possibly participating in a trial to help in GSD research.
 

Q: How has your life changed since your treatment?

A: My life has been a complete 180 since the trial. Not only is my glucose under much better control, but I have also seen improvements in my body on a molecular level. One example is that, when I originally went through puberty, my body did not have enough energy to carry out all the steps of puberty, but now, all these years later, my body is going through those changes. Another example is how quickly my body adjusts from metabolic imbalances. Recently during a medical procedure, my lactic acid reduced from 3.8 to 1.8 in only an hour without any IV dextrose intervention. That never would have happened prior to my treatment.
 

Q: Did the results meet your expectations?

A: Actually, I went into the trial with no expectations for my own treatment; my only hope was that it would not make me sick. Since I was patient 1 in Phase 1, I got a low dose of the gene therapy. My thought was that the gene therapy would not necessarily make my GSD more manageable, but it was a way to move the research further for others with the disease. I am a believer in karma, and I believe that the wonderful results I have seen personally result from my wish to do the trial to benefit only others. It has been a little over 5 years now since my infusion. I started to reduce my cornstarch intake gradually several months after the infusion, and 3 years after the infusion, I was completely off cornstarch and I am still off it now. I drink protein shakes in the morning and evening to maintain my glucose and have a snack before bed, but fortunately with that protocol and being careful about my diet, my glucose is now stable with no cornstarch at all. I feel I have finally done my part in helping others with GSD, which means my childhood dream was fulfilled!