Lani McLeod has had an underlying feeling of guilt for two years that her boys had access to life-changing treatment others don’t.
That changed this week.
Her sons Lincoln, 4, and Harlan, 2, have the motor neurone disease Spinal Muscular Atrophy (SMA) Type 2, but were fortunate to be granted compassionate access to Risdiplam, a drug to treat SMA, since March 2021.
Pharmac announced this week it will begin consultation on funding another SMA treatment, Nusinren, the drug branded as Spinraza, to roll it out for people aged 18 and under from January 1.
“It’s so exciting knowing it’s not just us now, as there has been that underlying guilt with the happiness of us getting [treatment]; knowing there are some many more out there that need and there was nothing we could do,” Lani said.
“It’s exciting for those who up until now have had access to nothing.
“It will make things easier for families of any newly-diagnosed kids, and take the stress out of considering moving countries or fighting for treatment away.”
The shock of an SMA diagnosis was bad enough, Lani said, without the heartbreak of being told there was a treatment available but it was out of reach.
“For a newly diagnosed parent, it’s still going to be extremely tough, but having the treatment available will take a huge weight off.
“The faster you have access to it the better.
“In our case treatment early would have saved a lot of heartache and a lot of struggles for the boys and us.
“It’s hard watching your child decline and there is nothing you can do.”
Everything changed when the brothers were granted compassionate access to Risdiplam as Lani and her husband James started seeing their boys “stop declining and making gains”.
“Within weeks we noticed the difference.
“The boys are making some amazing progress, and haven’t had any further loss of motor skills.”
Lincoln is benefiting from an improved immune system, meaning fewer trips to the hospital for minor coughs and colds, and making physical gains.
“The little things that you can take for granted are huge milestones, like how he can put his hand up so that when he goes to school next year he can do that.”
Before the treatment, the then 8-month-old Harlan was struggling to lift his head off the ground, but now “he’s off crawling, climbing up on furniture and getting into mischief”.
“It’s regular kid stuff that he never would have had the chance to do if he didn’t have access to the medication.
“So it’s an awesome feeling to know other children will have a chance at a regular childhood – as regular as it can be with SMA.
“Knowing they are not declining is massive for their families and themselves.”
As of January 1, other families and newly diagnosed families will have access to treatment as well.
“It’s a happy moment but it’s also frustrating that it took so much of a fight to get here.”
Lani said they will likely have a choice whether to remain in the programme or switch when the other drug becomes funded.
“As we are in the programme there will be no rush to make that decision, but we will speak to the specialists and parents who have experience with both to make the best decision for our boys.
“Whether we switch earlier or not, it’s a relief to know that when the 10-year programme ends we will have the option here.”
Lani understood the plan would hopefully be to roll out access at more locations than just at Auckland’s Starship Hospital.
“It’s a three times a year treatment, so for some families that access may still be a bit tricky if it’s only available from Starship.”
Having been through the stress of considering leaving the country just to get access, she said she would have had no hesitation with a few trips to Auckland a year for treatment.
“It would have been a logistical issue but it certainly wouldn’t have hindered our excitement at going to get it.”
Now the treatment is available there will be a push for screening for SMA in newborns, she said.
“The toll it will take away from parents, the child, the health system, and education system will be huge down the line.”
- By Jonathan Leask