The team at Trinity College Dublin is one of only four in Europe to take part in the clinical trial for the devastating disease.
The first Irish patient was entering the study on Tuesday at the Clinical Research Facility at St James’s Hospital in Dublin.
MND causes progressive paralysis, increasing physical disability and ultimately death within an average of two to three years.
There are more than 350 people in Ireland with MND, and one person is diagnosed every three days with the condition. There is currently no effective treatment.
We have many people carrying the abnormal C9orf72 gene, and for these people, the current trial could be a true game changer
The clinical trial targets an abnormal expansion of an MND-associated gene, C90rf72, using a novel therapy designed to switch off the abnormal part of the gene.
Up to 10% of people diagnosed in Ireland with MND carry the abnormal gene, which can also cause dementia.
The first phase of the trial, which has been divided into five sections involving 75 patients worldwide, was recently approved by the Health Products Regulatory Authority (HPRA).
Only 24 patients in the world will be enrolled in the fifth section, including the first Irish patient.
Professor Orla Hardiman, principal investigator in the Irish study and a leading world authority on MND, said: “Gene therapy is an exciting new approach and considerable progress has already been made in turning off another MND associated gene, SOD1, as the results of early clinical trials have shown.
“We are very hopeful that the same approach will work for those with the C9orf72 gene.
“While we do not have any patients with SOD1 mutations in Ireland, we have many people carrying the abnormal C9orf72 gene, and for these people, the current trial could be a true game changer.
“While these are early days, there is now genuine hope that MND will become a treatable disease in the near future.”
Professor Martina Hennessy, director of the St James’s Clinical Research Facility, said: “Our aim is to be the leading centre in the country for these types of early phase studies and we are delighted that the HPRA has pre-inspected and approved us.
“Ireland has traditionally not been able to compete on the world stage for these types of very early clinical trials, and it is a testimony to our staff, and to the international reputation of the Irish MND Research Group, that we have been selected and approved to enrol an Irish patient into this study.”
