Neil has to find a strength every day that most 10-year-old children will never have to muster.
For the past seven years, Neil has swallowed a daily dose of steroids at breakfast. They stunt his growth, but they keep him alive, and they help his weak and slowly wasting muscles to function.
I meet Neil in the lounge of his Auckland home. He bursts into the room like a pint-sized paparazzi, firing questions at me with big, warm eyes and a wide, infectious smile.
“Hi! I’m Neil. Is that a camera? Can I take a picture? Where are you from? You have a microphone! Are you on YouTube?”
Neil is fiercely intelligent, but his brilliant mind is outperforming his small body.
He has Duchenne Muscular Dystrophy, a severe and progressive genetic disorder which leads to muscle degeneration, difficulties with movement and breathing, and ultimately premature death. It is most common in boys with the first symptoms usually occurring between the ages of 2 and 3. By the age of 12, most have lost the ability to walk.
Neil’s father, Kuldeep speaks in the gentlest of voices.
“He’s a little bit of a chatterbox,” he says, smiling. It is quite the understatement.
Kuldeep softly pats the sofa beside him, urging his excited young son to sit down. Neil is having none of it. He bounces on his tippy-toes, peers into the camera lens and belly-laughs.
“Who will see my picture? Will I be famous? This camera is so cool! I love this camera!”
Neil’s big brown eyes never stop sparkling and his curious mind is at least three highly articulate questions ahead of our conversation. He’s got me on the ropes, and I can’t get a word in so, in fits of laughter, I start firing questions back at him.
He’s been to swimming lessons that day and he tells me he will soon be moving up a level. Children with Duchenne’s are encouraged to swim to strengthen their muscles while being supported in water.
“His neurologist encourages us to get him in the pool and he loves it,” says Yasha, Neil’s mum.
I tell Neil that I bet I can guess his favourite subject at school.
Neil gasps. “No! What is it?”
I tell him I reckon it’s maths.
“What?! How did you guess?” he says, throwing his head back in a fit of giggles.
“I like spelling too! I can spell adjective!” And then to prove his point, he barks out every letter at lightning speed. He is a voracious reader, and it’s obvious every time he speaks. The questions keep coming and I realise I am in the presence of a truly brilliant young mind.
There is no cure for Duchenne Muscular Dystrophy, but Neil takes Deflazacort, a steroid to reduce inflammation and change the way his immune system works.
His parents believe it has fewer side effects than the publicly funded steroid available here, so they pay for it themselves. It’s not hugely expensive, a few hundred dollars a month, but it all adds up when you’re supporting a child with a severe genetic disorder.
“We believe it’s the best medicine for Neil, so we pay for it. What parent wouldn’t?” says Yasha.
However, Yasha and Kuldeep’s greatest fear is our health system’s lack of preparedness for the new generation of medicines steaming towards us.
By far, the greatest hope lies in gene therapy. It offers the possibility of a permanent cure for more than 10,000 human conditions triggered by a single-gene defect. CRISPR, a genome-editing tool, allows scientists to insert or delete DNA to correct the mutation which is causing the disease. It effectively ‘turns off’ diseases like Duchenne Muscular Dystrophy.
Kuldeep says the CRISPR technology, currently used in advanced research projects all over the world, is scientifically heralded as lifesaving, and could provide a permanent cure for his son and other children just like him.
“But here is the question,” Kuldeep says.
“If you ask me what I’m frightened of, I will tell you. What is our strategy? Is our health system ready to respond to these modern medicines and treatments? I see thousands of New Zealanders fundraising to get medicines for their children and every time I see them, I know I will soon be among them. I will have to do that for Neil because I haven’t seen any evidence that our health system is even thinking about responding to today’s modern medicines or treatments, let alone tomorrow’s.”
In terms of preparedness, the Government is standing flat-footed. There is no sector-wide plan. No horizon-scanning to consider gene-editing or gene-silencing technology. No Government-supported strategy or expert team considering how our health system will respond to what has been described as one of “the greatest technologies in the history of biomedicine”. There is nothing. Nothing at all.
Last year, the Ministry of Health’s five-year Medicines Strategy expired. It hasn’t been renewed. Remarkably, New Zealand’s health system does not have a policy governing how we manage, access, and use today’s medicines, let alone tomorrow’s.
For Neil and his family, the situation amplifies an already challenging and uncertain future. Neil is now using a wheelchair at school. It’s easier for him to get around and he doesn’t tire so easily. Kuldeep says he has noticed that Neil is slowing down. The condition is slowly traveling up his body, restricting movement and muscle function, and Kuldeep fears his son will soon be permanently confined to his wheelchair.
I ask Kuldeep about his hopes for Neil’s future. He pauses for a moment.
“For my son to live. That is all. Please. Just let him live.”
Neil’s future, and the future of thousands of New Zealanders, relies on the Government’s willingness to not just acknowledge the speed of progress in medical science, but to develop a First World strategy to respond to it.