Researchers begin trial of drug to slow progression of neurodegenerative condition Multiple System Atrophy 

04/11/2019 00:00 
Researchers at UCL Queen Square Institute of Neurology (IoN) and the UCLH National Hospital for Neurology and 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.

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

 Latest news

 Contact details

Communications unit
2nd floor central
250 Euston Road
London NW1 2PG

Media enquiries

Switchboard: 020 3456 7890
Media enquiries: 020 3447 7542 / 020 3447 9506

Out of hours
The normal working hours for the Communications Unit are Monday to Friday 9am – 5pm. The only media enquiries that will be answered outside of these working hours are urgent enquiries and those relating to major incidents. To access the out-of-hours service call switchboard on 0845 155 5000.

Share this story