Why the Saatchi Innovation Bill Is Important for Parents of Children With Duchenne

Harrison is now nearly 8, and we live each day with the knowledge that because he's got a duplication of an exon of his dystrophin gene, his symptoms continue to progress and he continues on a steady and rapid physical decline.

When Harrison was just past his fourth birthday, I took him to his pediatrician thinking he might have a mild physical delay and may need physical therapy. Within 2 weeks we recieved the most devastating diagnosis imaginable. Duchenne muscular dystrophy. It was 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.

Harrison is now nearly 8, and we live each day with the knowledge that because he's got a duplication of an exon 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 the shock of his legs buckling without warning, sending him tumbling to the ground. It is marked by the fatigue of exerting great effort just to do "normal" things - like keeping his balance and getting up from the floor. It is marked by my lack of words or comfort at his tears when he is unable to join his brother in his sporting endeavours. And it is marked by hanging on - to what may be the last time he'll complete a task as simple as opening a jar.

This is the time when kids are moving faster and pushing limits and growing and looking forward to the future. I'm continually terrified that Harrison may well not have the future we all have a right to.

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 monsters under their bed. But they should NOT be afraid of needles, biopsies, of falling and breaking bones. They should NOT be afraid of no longer walking or of being unable to breathe for themselves or of no longer having the strength to hug their loved one's. Above all else they should NOT be afraid of dying.

I'm writing this blog today because The Saatchi Bill is designed to help doctors innovate, so that they can advance medical science and find new and better treatments and cures for diseases and conditions. It will free your doctor to consider new treatments and ideas. And, it will allow the patient to demand innovative treatment. Armed with the legislation, a patient will be able to say to his or her doctor: 'Are you trying everything? Can you do anything differently?' The doctor will no longer need to say he or she cannot risk trying anything new.

This 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.

As patient and patient advocates there is a terrible lack of clarity and lack of certainty in the current law which is not acceptable to the real people whom the law affects; and that whatever clarity there may be in theory about the "textbook" law of medical negligence, the lack of clarity in practice affects doctors and the patients they are trying to save.

The Bill will enable our physicians and patients to plan the use of innovative treatments together, focusing on a new statutory list of criteria to consider, a processes to follow, and to ensure that the innovation is responsible. Getting this right gives our doctors confidence that it will be upheld as responsible by the courts, rather than having to take the risk, wait and battle it out with opposing teams of medics after the event in court. In no way should responsible doctors be handcuffed from innovating and thinking without tethers when the lives of their patients are in the balance.

We've all come across the 'quackery' out there with miracle cures and the framework a Bill like this will have will allow us to clearly distinguish between responsible innovators and the obsessive monomaniac lunatics.

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. 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.

We are so close to a treatment that my son Harrison is part of a generation that will either be the last to die from Duchenne or the first to survive.

With this bill, the robust ethical framework it will be built around 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.

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