A tough call: Should our sons switch Duchenne clinical trials?
As a parent to children with Duchenne MD, sometimes there are no easy answers
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While very serious and sometimes grim, the prognosis today for Duchenne muscular dystrophy (DMD) is the best it has ever been. This is because so many new therapies are in the clinical trial process, and some have already been approved. As a result, parents of children living with DMD are presented with more treatment options today than ever before.
A friend of mine, a Duchenne mom whose son has been involved in the same clinical trial as my three sons with DMD, Max, Rowen, and Charlie, once said something that has stuck with me for years. She said we are doing things now that have never been done before, and we could be changing the outcome for our sons.
Last month, while I was in the clinic with my sons, we were presented with information for a clinical trial that will open sometime in the late summer or early fall. It could be a good match for my sons.
We’re in the process of deciding if this new trial is something we are interested in. It will be a tough decision. The boys are already in a clinical trial, and participating in a new one requires the boys to wash out of the trial drug they are currently taking.
This situation poses many questions, some of which are impossible to answer. For example, how much is the current treatment helping them? Will the boys decline during the washout period? What if we wash them out and then they don’t qualify for the new trial?
While we want to do things in a way that can guarantee our success, there are simply no guarantees. We want to be able to screen for the new trial to see if the boys can make it before taking them off their current trial drug, but that isn’t possible. Trials are set up with distinct criteria and instructions, and I understand why it has to be that way, but it doesn’t help an anxious mom.
And then there are the risks, as with any trial drug. All we can do as parents is make the best decision possible with the information we have at the time. But this decision is nearly impossible to make. Our sons have been stable and are doing well, but could they be faring even better against Duchenne with something different? Or do we take this massive chance only to find out later that the current option was the right one?
Where is the magic eight ball or the mirror on the wall? I wish so badly that I could ask someone with all the answers what to do, but that’s not in the cards.
Honoring the families who came before us
We still have time to make this decision. The boys are part of the process and will ultimately decide what they want to do, but as parents, it’s our job to help them collect information and weigh it all. We will use prayer and conversations with other DMD families and medical professionals to guide us.
As we grapple with the enormity of this decision, I will not let myself be overcome by the stress of fear but rather focus on gratitude. My family can only choose between two promising treatments because many families before us made hard decisions. They decided to participate in trials with no promise of success, only hope for a better future.
I won’t take for granted the families whose sacrifices have made it possible for my own family to have choices and decisions to make. Without the brave families who came before us, our future would not look the same.
Note: Muscular Dystrophy News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Muscular Dystrophy News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to muscular dystrophy.
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Comments
Mindy Cameron
Thank you for acknowledging the boys and families who were dedicated to research in the past. They are too often forgotten and many of the boys have since passed. I will read your comments to my 21-year old son who has participated in many studies that helped with our collective understanding of DMD.
Betty Vertin
I am so thankful to you Mindy and your son. We would not be where we are without you and all the families like yours!