Researchers begin trial of drug to slow progression of neurodegenerative condition multiple system atrophy

NOTE: This release was originally published by University College London, here.

Researchers at UCL Queen Square Institute of Neurology (IoN) and the UCLH National Hospital for Neurology & Neurosurgery (NHNN) are set to test whether a drug can slow progression of the devastating neurodegenerative condition multiple system atrophy (MSA).

MSA can cause symptoms such as slowness, stiffness and tremor – similar to those found in Parkinson’s disease. In some patients it mainly affects balance, and patients can develop problems with low blood pressure, speech, and bladder and bowel control. The disease tends to progress more rapidly than Parkinson’s disease and responds poorly to Parkinson’s medications. Researchers said there is a huge unmet need for new MSA treatments.

In a pilot study led by Professor Tom Foltynie (UCL IoN and UCLH NHNN) researchers will test whether the drug exenatide – currently licensed for type 2 diabetes – can slow progression of these symptoms.

“Patients with MSA are in urgent need of therapies that slow down or stop the progression of this disease.”

Two small trials have already indicated the drug might slow decline in Parkinson’s disease – although these positive results need to be confirmed in larger studies.

In addition, lab data using animal models of MSA indicate exenatide may have positive effects, and post mortem data from people with MSA show changes in their brains which may have been treatable with the drug.

For the pilot study, patients with a diagnosis of MSA for less than 5 years – including patients with suspected MSA whose diagnosis is confirmed after a detailed clinical examination – will be randomised into 2 groups. One group will add exenatide to their regular medication; the other will continue their regular medication alone.

Participants receiving exenatide will self-administer the drug via a once-weekly injection under the skin. Researchers will see all participants every 12 weeks for a total of 48 weeks at the National Institute of Health Research (NIHR) UCLH Clinical Research Facility at the Leonard Wolfson Experimental Neurology Centre to assess whether the drug affects the rate of progression of MSA.

Professor Foltynie said: “Patients with MSA are in urgent need of therapies that slow down or stop the progression of this disease. The limited effectiveness of existing drug treatments, as well as the rapid rate of progression means that at the moment some patients can develop major disability and need for carer support even within the first few years after diagnosis.”

For the trial, Professor Tom Foltynie will work with Professor Henry Houlden and Professor Huw Morris. Dr Sonia Gandhi, Dr Viorica Chelban, Dr Christine Girges and Dr Nirosen Vijiaratnam will support the trial.

The study is funded by the John Black Charitable Foundation in the UK and Van Andel Institute and the Defeat MSA Alliance in the USA. The study is supported by the MSA Trust (UK).