Being a Pioneer
The past two months have been difficult for me. We have started the PTC 124 extension trial. We have waited for baited breath for the last two years to start this trial.
My son, who is more than willing to try any therapy if he thinks it will help, balked at the plan. I came home from work late one night to find my son in a state of total melt down. Yes, at 15 we have melt downs. For the past two months it has been a struggle to gag down the vanilla flavored drug. Given his age and weight his dosage is pretty high. His stomach has been upset; he has not been hungry and has experienced rapid weight loss. As I walked in the door he was trying to take his PTC and screamed at me "TAKE ME OUT OF THE TRIAL".
Those words stopped me in my tracks. I lost my mind, and slowly turned to my son and said, "FINE, I have wasted 10 years of hard work helping PPMD and the Duchenne community!" "Before we do anything, you really need to think about the end result will be!" My husband and I looked at each other and without speaking had the same question written on our faces. "What do we do now?" I turned to my son and said "OK, skip the dose tonight and go to bed, we will talk about it more tomorrow after we talk to the doctor."
My focus has always been on my son and on the bigger picture, whereas my husband's focus has been our son. I have always felt deep down that if this trial was really successful would change the face Duchenne and pave the way for other Duchenne trials. My biggest concern is messing up the protocol for the trial and putting future trials in jeopardy. Therefore, my constant mantra to my husband, as we try and figure out how to get my son to take this drug, have you checked with the doctor yet?
After checking with the doctor, we have stopped giving our son Juven and nearly all of his other supplements. The only medications he is taking are the ones prescribed and of course the Vitamin D and Calcium Supplements. Scary thought to us, as all the other stuff combined have worked and worked pretty well for the past 8 years. The up side is that our son is now able to tolerate taking this drug and is not experiencing the upset stomach and his appetite is returning to normal. So we will take it a day at a time and continue down the path we have begun
My hopes right now are:
The past two months and participating in this trial will all be worth it in the end.
All the trial participants will end up with a therapy for their sons.
By participating in this trial we will have helped pioneer future successful Duchenne trials.
.
My son, who is more than willing to try any therapy if he thinks it will help, balked at the plan. I came home from work late one night to find my son in a state of total melt down. Yes, at 15 we have melt downs. For the past two months it has been a struggle to gag down the vanilla flavored drug. Given his age and weight his dosage is pretty high. His stomach has been upset; he has not been hungry and has experienced rapid weight loss. As I walked in the door he was trying to take his PTC and screamed at me "TAKE ME OUT OF THE TRIAL".
Those words stopped me in my tracks. I lost my mind, and slowly turned to my son and said, "FINE, I have wasted 10 years of hard work helping PPMD and the Duchenne community!" "Before we do anything, you really need to think about the end result will be!" My husband and I looked at each other and without speaking had the same question written on our faces. "What do we do now?" I turned to my son and said "OK, skip the dose tonight and go to bed, we will talk about it more tomorrow after we talk to the doctor."
My focus has always been on my son and on the bigger picture, whereas my husband's focus has been our son. I have always felt deep down that if this trial was really successful would change the face Duchenne and pave the way for other Duchenne trials. My biggest concern is messing up the protocol for the trial and putting future trials in jeopardy. Therefore, my constant mantra to my husband, as we try and figure out how to get my son to take this drug, have you checked with the doctor yet?
After checking with the doctor, we have stopped giving our son Juven and nearly all of his other supplements. The only medications he is taking are the ones prescribed and of course the Vitamin D and Calcium Supplements. Scary thought to us, as all the other stuff combined have worked and worked pretty well for the past 8 years. The up side is that our son is now able to tolerate taking this drug and is not experiencing the upset stomach and his appetite is returning to normal. So we will take it a day at a time and continue down the path we have begun
My hopes right now are:
The past two months and participating in this trial will all be worth it in the end.
All the trial participants will end up with a therapy for their sons.
By participating in this trial we will have helped pioneer future successful Duchenne trials.
.
Comment
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Comment by Tina on November 24, 2008 at 9:25pm -
Your son was telling you that all of the stuff he was receiving was making him sick. He knows his body all too well. Thanks for sharing this story because as more robost therapies arrive for our sons, we may be able to back off on some of the other supplements we have been giving to them as a way of bandaiding the downward spiraling effect this disease takes place on their bodies.
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Comment by Christine Piacentino on November 22, 2008 at 9:02pm -
Thank you Brian! I appreciate your additional comments, as they add further detail to the reality of participating in a clinical trial.
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Comment by Brian Denger on November 22, 2008 at 7:30pm -
Your message expresses the reality that families must confront when participating in a clinical trial Christine, which is the difficulty in balancing expectations with reality. We all want to get off this roller coaster and many will try just about anything. What is still present upon beginning a trial is everything we knew and lived with before starting. Our children still have emotional outbursts, we still need to dole out medications and help them as we did before and there still is the angst and stress that preoccupies our lives. Until we reach the stage where there is "real magic" to end Duchenne, there will be no immediate relief.
Therefore all those participating in the PTC 124 Trials are giving up as much as they are receiving. The reality is regular blood draws, biopsies, strict adherence to PTC dosing schedules and maintaining dosing of current medications and supplements. Add travel and the "pleasure" of spending the day in another clinic and you wonder why people envy what you are doing. Still, families like yours are showing the rest of us what to expect when participating in a clinical trail.
You title "Being a Pioneer" is appropriate as that is what your family and others participating in the PTC 124 Trials are doing. You are pioneers in world of therapeutic clinical trials for DMD. You have my support and I hope you all get the benefit you seek which would also be the spark to ignite the additional interest needed in DMD research.
Keep your chins up and my best to you and your entire family!
Brian Denger
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