How much is our child worth?

SMILING THROUGH THE PAIN  Grace O’Malley, who lives with spinal muscular atrophy (SMA) and would benefit from Spiranza a ‘groundbreaking’ drug, is pictured after making her Holy Communion.

Mother begins fight for miracle drug to treat child with SMA

Ciara Galvin

THE mother of an eight-year-old girl living with spinal muscular atrophy (SMA), type two, is calling for a revolutionary drug to be made available in Ireland.
Lorraine O’Malley who lives in Robeen, near Hollymount, is calling for the drug, Nusinersen, marketed as Spinraza, to be made available for child, Grace.
The drug is the first treatment for the life-limiting condition, which affects mainly infants and children. Described by doctors as ‘groundbreaking’, it received its first approval in the US in late 2016. The drug has since been approved in the European Union (June 2017) but is still not available in Ireland.
Speaking to The Mayo News yesterday (Monday), Lorraine’s frustration with the situation was obvious. “I didn’t think it would boil down to having to fight for it. It’s there, and you’re just going, ‘Will ye make it available and make a decision?’,” she said.

Losing movement
Lorraine and husband Paul found out about the drug through an online SMA group in 2016. At that point they believed it could take a year before it became accessible in Ireland. Now, two years on, the O’Malleys are growing more and more exasperated and worried. They are aware that the drug is not a cure for Grace’s condition, but it would stop it progressing.
“She is losing movements that she used to have. She can’t hold a plate now, or plug in a charger. While we’re waiting for a decision she is deteriorating,”said Lorraine.
Her parents say that even if the drug just allowed Grace to be able to move herself around in her bed it would improve their current situation. Currently, they have to turn her four to five times during the night.
Consultant neurologist Dr Declan O’Rourke of Temple Street Children’s Hospital, who sees the majority of children with SMA, has been campaigning for Spinraza to be made available.
The drug is injected into the spinal canal, and a patient requires four ‘loading’ doses in the first two months, then a maintenance dose every four months thereafter.
The Spinraza list price is US$125,000 per injection, which puts the treatment cost at $750,000 dollars in the first year and $375,000 annually after that. According to the New York Times, this makes Spinraza ‘among the most expensive drugs in the world’.
Lorraine explains that for the drug to be made accessible, it must first get through a host of checks, from bodies that include The Health Products Regulatory Authority and subsequently the HSE Drugs Group.

Waiting
“We got word that it was approved in Europe and imagined it was full steam ahead. At Grace’s check-ups we keep asking if there is any news on Spiranza. Dr O’Rourke was positive, saying it cannot be denied because all the trials say it works.” But now the O’Malleys say it is boiling down to money.
“I think they are negotiating the price without thinking of the kids who are suffering. It’s so soul destroying, if this is down to money … and you see the Pope is coming, and it’s costing €5 million. This is down to getting treatment for our children. Every minute counts because they’re losing power,” said Lorraine.
In Kilmaine, another couple, also called Lorraine and Paul (Mearns), is joining the fight for access to the drug. Their six-year-old son Cillian also has type two SMA. Currently, two children with SMA type 1 are on a trial for the drug. The parents of Grace and Cillian are calling on all political representatives to back them in demanding that Health Minister Simon Harris put plans in place to facilitate access to the drug.
The fight continues, but for now, the O’Malleys are concentrating on making Grace’s ninth birthday on August 4 the most special one yet. Thanks to the Make-a-Wish Foundation, the family will be visiting Disneyland, where little Grace will spend the day having breakfast with a host of Disney princesses before enjoying the attractions with her brother, Mark, and her parents.