When Harrison was just 4 years old, I took him to his pediatrician thinking he might have a mild physical delay and may need physical therapy. Within 2 weeks, a blood test and a visit with a neurologist provided us with the most devastating diagnosis imaginable. Duchenne muscular dystrophy. The neurologist explained to us that Harrison’s muscles would rapidly deteriorate, he would lose the ability to walk, to use his arms, to bathe himself, to go to the bathroom on his own. Eventually Duchenne would attack his heart and lungs and the disease would take his life. We have nothing to stop it he told us. It’s 100 % fatal, I wish I could tell you it was cancer.
I swung in to action, I challenge myself daily…mentally and physically to do the best I can. I’ve started a charity, Harrison’s Fund, to generate as much cash as I can to put into breakthrough Duchenne research to find a treatment for this generation of children and young adults. We have already invested hundreds of thousands of pounds. I’ve become a networker, at all times, this never really switches off, I’ve started a not for profit race team, Harrison’s Racing, I’ve literally gone from Average Man to Ironman, completing my first Ironman triathlon in October last year. But, becoming just an Ironman doesn’t seem quite enough for me now… next in the challenge list is to take Harrison on an Ironman with me. Drag him in a boat behind me for 3.8km, take him on the 180km bike leg and then push him the no small matter of a 42.2km marathon, all back to back in under 13hrs and 47mins, a world record time. Harrison will also be an Ironman… that’s just cool! I am in the process of assembling the team to get this done, from building a bespoke catamaran to drag, to building the bike and Harrison machine and the running chair needed to take a child that no longer really fits in any of the running chairs out there anymore.
Harrison is now nearly 8, and we live each day with the knowledge that because he’s got a duplication of exon 51 of his dystrophin gene, his symptoms continue to progress and he continues on a steady and rapid physical decline.
Harrison’s doing incredibly well considering but he has reached a plateau all children with Duchenne do around there seventh and eighth birthdays.
This is a stage of progression that is marked by an accelerated decline… It is marked by …
Shock – the shock of his legs buckling without warning, sending him tumbling to the ground. It is marked by the shock of not being able to open the jar he used to be able to
Fatigue – he is plagued by the fatigue of exerting great effort just to do “normal” things – like keeping his balance and getting up from the floor.
Strategizing – thinking about how we organise and prioritize a day for the family that will not wear him out yet still let him experience all the world has to offer.
Disappointment – the searing realization that he can’t play organized sports, my lack of words or adequate comfort when he walks into the house fighting back tears because his brother or friends just rode off on their bikes and he can’t be with them.
Routine – daily stretching sessions, and regularly physical therapy appointments and doctor appointments.
Hanging on – to what may be the last time he’ll complete a task or have the energy to help me coach his brother Williams under 6 rugby team.
This is the time when kids are moving faster and pushing limits and growing and looking forward to the future. And I’m terrified that Harrison may not have one. Annual events like birthdays and the end of a school year are marked by conflicting emotions – they’re marked by relief that we were given the gift of another year. And by grieving one less year we have together.
While these symptoms are heartbreaking, I am also filled with the sobering reality that this is MILD compared to what we will face in the very near future. There is a train racing toward my little boy and I’m running as fast as I can to scoop him up and save him, but I’m acutely aware that I may not make it in time. We need to affect massive change to help stop the train.
Children at any age – whether they’re 3 or 5 or 7, like Harrison, should be afraid of the dark. Of the bully on the playground. Of the monsters under their bed. But they should NOT be afraid of needles and biopsies and surgeries and falling and breaking bones. They should NOT be afraid of no longer walking or of being unable to feed themselves or of losing so much strength in their arms that they can no longer hug their mum’s and dad’s. Most importantly, they should NOT be afraid of dying.
I’m writing this blog today because this medical innovation bill has the very real potential to help doctors and clinicians slow down the train. In our case the risk of doing nothing is not nothing, the risk of doing nothing is fatal. Fatal every single time. You never survive this.
In any innovation in medicine there is an element of risk, and between clinicians and patients there has to be a level of permissible risk particularly when the population is small and the need is great. In terms of acceptance of risk – our community has already demonstrated our philosophy on this – we have one option right now – it causes cataracts and growth stoppage and osteoporosis, and weight gain and immune system suppression – and this drug is still being studied in the Duchenne population. There are debates over dosing and efficacy and this drug doesn’t even change the final outcome for Duchenne patients. Yet, our children’s doctors prescribe it, and in fact encourage its use. It’s part of the standard of care for Duchenne. Through this use of steroids, we’ve already shown that we’re willing to assume risk. We understand that the long-term risks of steroid therapy are known, and the long-term risks of a new therapy would be unknown. But it’s also quite straightforward and simple logic that helps us understand that long-term risks can only become clear when something has been used for the long term – and the only way to get to the long term is to begin. We have to begin somewhere. Sometime. And the time has to be now.
What we are not willing to do is assume the risk of doing nothing. If a potential therapy shows promise of stabilization or improvement over what would be expected without any treatment, and it shows safety, then patients and parents should be given a choice to try it with long-term studies taking place concurrently. Because, at the end of every discussion and assessment of a therapy, we must never lose sight of the reality that the risk of having Duchenne far outweighs the risk of most potential treatments. And our children must be the beneficiaries of our best effort, of our most noble intentions, and of our greatest commitment to safety AND speed. Because at the end of the day, these children are not a statistic. They are not a commodity. They are not someone’s science experiment.
They could be YOUR boys or grandchildren…..and they may not be, but the responsibility for saving them belongs to all of us. I believe we are close to a treatment. We are so close that my son Harrison is part of a generation that will either be the last to die from Duchenne or the first to survive. We must have a great sense of urgency and we must always remember that the children should NOT serve the science, but the science must always serve the children.
With this bill and the innovation it would encourage we have the potential to move forward and reduce Duchenne from a 100% fatal condition to that of a chronic condition. The time is now, we don’t have time to waste, #maketime.