THE FIRST patient in Europe to participate in a clinical trial for a promising new drug for motor neurone disease (MND) was enrolled in the trial by doctors at Beaumont Hospital last week.
The drug called Dexpramipexole, which has been developed by the pharmaceutical company Biogen, is now entering the final phase of testing. It is thought to work by increasing the efficiency of mitochondria, the part of the body’s cells that produce energy. Doctors here plan to sign up 30 Irish patients for the Empower trial by October.
Lead researcher, Prof Orla Hardiman, consultant neurologist at Beaumont Hospital, told The Irish Times, “Early clinical trials suggest that dexpramipexole has the potential to be a significant advance in MND treatment.
“In small groups of patients in the US, the drug seemed to slow disease progression and improve survival. A double blind placebo controlled study is now required to prove whether the beneficial effect is real.”
Hardiman, who is also a clinician scientist with the Health Research Board, added, “we are delighted that Irish patients have the opportunity to participate in this study and are proud that the first enrolment outside of the US happened this week in Ireland.
“We will know for definite whether the drug is truly effective in two years’ time.
“If it is, it will open the possibility of finding a treatment that will stop disease progression altogether.”
MND is a progressive neurological disorder that leads to muscle weakness and wasting and is also known as ALS (Amyotrophic Lateral Sclerosis).
The incidence of MND is one in 30,000 people. About 80 people in the Republic of Ireland develop MND each year, and there are about 200 people affected by the condition at any time. Life expectancy for someone with the disease is between three and five years from the onset of symptoms.
Experts suspect there are a variety of different causes of MND. It is likely that some people have a genetic susceptibility to developing the condition, and that environmental factors can interact with these genetic risks. In about 10 per cent of patients, the disease runs in families; it occurs sporadically in the majority of cases.
Currently, patients are treated with a drug called riluzole, which extends the patient’s life by about three months. But the mainstay of treatment is multidisciplinary care, which in Beaumont has been shown to improve survival by nine months.