Trinity College Dublin: New gene therapy to treat children with Spinal Muscular Atrophy approved in Ireland

Trinity College Dublin, St. James’s Hospital and Children’s Health Ireland have welcomed the news that the HSE has agreed a reimbursement price on Zolgensma gene replacement therapy for the treatment of Spinal Muscular Atrophy (SMA) in children.

The two healthcare institutions and the university were involved in delivering the first ever gene therapy to children in Ireland. The successful collaboration involved four Irish children and babies with severe SMA over the past 18 months. This was the first time that children in Ireland had ever received gene therapy.

SMA is a rare, progressive, life limiting disease that makes muscles weaker and causes problems with movement. In the most severe cases, children cannot walk and have problems breathing and swallowing.

The gene replacement therapy is a highly specialised advanced therapy requiring very specialised equipment and healthcare staff to administer it safely and to follow the long term outcomes of treatment. The therapy was administered at the Trinity Wellcome HRB Clinical Research Facility at St James’s Hospital in Dublin as part of a unique collaboration between Dr Declan O’Rourke and Professor Martina Hennessey.

The therapy is administered only once and alters the genetic barriers to normal muscle and nerve development that occur in a child with SMA.

Professor Martina Hennessey from CRF at St James’s Hospital, said:

We hope this is just the beginning of ensuring that Irish patients, children and adults, have access to the most innovative and life-changing treatments available anywhere in the world. Our collaboration has shown that we can deliver these new therapies successfully at St James’s Hospital which means that other cutting edge treatments for some of the most difficult conditions can be trialled here ensuring Irish patients have equal access to life saving medicines.

Dr. Declan O’Rourke, CHI Temple Street said:

This is a game-changer. For the families who receive a diagnosis of Spinal Muscular Atrophy it is a devastating blow as they know that without treatment their small babies will not survive to become teenagers. Now they will have access to a gene therapy which dramatically improves their quality and the length of their lives.

It is expected that Zolgensma will be available to children and babies who are suitable for the treatment within a matter of weeks.