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Researchers in Dublin are participating in an early phase gene-based clinical trial for motor neurone disease (MND) sufferers.<\/p>\nResearchers in Dublin are participating in an early phase gene-based clinical trial for motor neurone disease (MND) sufferers.<\/p>\n
The team at Trinity College Dublin is one of only four in Europe to take part in the clinical trial for the devastating disease.<\/p>\n
The first Irish patient was entering the study on Tuesday at the Clinical Research Facility at St James\u2019s Hospital in Dublin.<\/p>\n
MND causes progressive paralysis, increasing physical disability and ultimately death within an average of two to three years.<\/p>\n
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.<\/p>\n
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.<\/p>\n
Up to 10% of people diagnosed in Ireland with MND carry the abnormal gene, which can also cause dementia.<\/p>\n
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).<\/p>\n
Only 24 patients in the world will be enrolled in the fifth section, including the first Irish patient.<\/p>\n
Professor Orla Hardiman, principal investigator in the Irish study and a leading world authority on MND, said: \u201cGene 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.<\/p>\n
\u201cWe are very hopeful that the same approach will work for those with the C9orf72 gene.<\/p>\n
\u201cWhile 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.<\/p>\n
\u201cWhile these are early days, there is now genuine hope that MND will become a treatable disease in the near future.\u201d<\/p>\n
Professor Martina Hennessy, director of the St James\u2019s Clinical Research Facility, said: \u201cOur aim is to be the leading center in the country for these types of early phase studies and we are delighted that the HPRA has pre-inspected and approved us.<\/p>\n
\u201cIreland 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 enroll an Irish patient into this study.\u201d<\/p>\n