Father hails approval of MLD drug

The father of two children with Metachromatic leukodystrophy – or MLD – has said he is very glad to hear that the drug Libmeldy has been approved for use in Ireland.

The drug, which is the most expensive in the world, is set to become available for use in Ireland following negotiations with the manufacturer on price.

The drug is used to treat MLD, which is a rare and life-threatening inherited disease that affects the metabolic system in children.

The condition seriously damages the brain and the nervous system in children.

Les Martin’s eldest son, Cathal, died from the disease three years ago at the age of six.

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The disease had progressed too far for him to receive treatment by the time he was diagnosed, but his younger brother, Ciaran, was able to avail of it successfully.

Speaking on RTÉ’s Today with Claire Byrne programme, Mr Martin called for MLD to also make up part of the heel prick test at birth which screens for a number of diseases.

He explained that MLD is so rare that the disease often progresses past the point of treatment by the time it is diagnosed.

“It’s not something that is generally suspected. And by the time the diagnosis comes around, if the child is too symptomatic, the therapy is not effective and shouldn’t be used. It really has to be pre-emptive. It works best when the children are young and unaffected by the condition,” he said.

“I hope that they’re amongst the last to die of MLD in this country.”

He said that he hoped the legacy of his son, and other children who have died from MLD, is to ensure that no more children in Ireland die from the condition.

“I’m very proud of my own son’s role in all of this,” he said, and cited other families who have had children die from the condition.

For those children who did from the condition he said “[it’s] their legacy, I suppose, and the change that will make from this generation on”.

“I hope that they’re amongst the last to die of MLD in this country. The solution is two parts – approval of the therapy, and the introduction of screening,” he added.

Mr Martin said that his eldest son was born fit and healthy but that he started missing milestones as he got older.

“He was born healthy and fit and happy, a great little boy, a gorgeous little guy until about the age of a year-and-a-half, two, when he struggled to meet milestones. And it took us around a year-and-a-half to be diagnosed with MLD because of its rarity.”

Mr Martin’s younger son, Ciaran, was diagnosed with MLD at the same time as Cathal but was eligible for a trial in Italy because he was younger.

“It was a life-or-death situation so we travelled immediately,” he said, adding that they stayed there for nine months so Ciaran could receive the treatment which stops the disease from progressing any further.

Ciaran was just over a year old when he received the therapy, and has some nerve damage and mild disability, he said, but he has not declined in any way since he received the treatment.