Cardiac medication could offer relief for some neurological patients 

02/10/2012 00:00 

A study led by UCLH could change the way patients with a rare debilitating neurological disorder are treated.

The leading medical journal JAMA (The Journal of the American Medical Association) has published the findings of the study which established that reprofiling of a decades-old drug previously used to treat abnormal heart rhythms could treat the symptoms of a genetic muscle disorder known as nondystrophic myotonia. The genetic defect lies in a key muscle protein (known as an ion channel) which controls how muscles respond to electrical signals from their nerve supply.

The condition is not usually life-threatening, but can be very debilitating, as patients’ muscles contract and will not relax, causing functionally limiting pain, stiffness and fatigue

The experimental medicine phase II study was carried out in 59 patients at seven neuromuscular referral centres in four countries. The largest number were recruited through the MRC Centre for Neuromuscular Diseases linked to NIHR Biomedical Research Centre at University College London Hospitals NHS Foundation Trust (UCLH) and UCL.

Professor Michael Hanna at the Queen Square Medical Research Council (MRC) Centre for Neuromuscular Diseases, and the senior author on the paper, led a UCLH/UCL team.

He said: “These disorders can be extremely severe and the effects can stop people from working. Patients who took the repurposed drug, mexilitine, reported that their stiffness significantly improved, they had less pain and they also performed better on a range of detailed functional tests measuring muscle electrical excitability and muscle strength including handgrip tests. The findings are exciting because this treatment can make the difference between patients being able to work and not, but further research is required.

“It is much harder to do experimental clinical trials in rare diseases such as nondystrophic myotonias because individual researchers aren’t able to see the volume of patients required. However, if you are a patient with a rarer disease – something which is harder to study – you should still have access to research and treatment. That’s why we set up this international collaboration to pool our resources and share information. Finding treatments for rarer diseases is an important goal for the NIHR University College London Hospitals Biomedical Research Centre.”


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