Trial of drug to slow progression of rare neurodegenerative condition begins

Researchers at UCL Queen Square Institute of Neurology (IoN) and the UCLH National Hospital for Neurology & Neurosurgery (NHNN) have begun a trial looking at whether a drug can slow progression of the devastating neurodegenerative condition Multiple System Atrophy (MSA).

Eight participants have been enrolled so far onto the study, with the first patient enrolled in October 2020.

The study team were able to recruit and continue this important trial during the Covid-19 pandemic as MSA is a serious and life-threatening disease, so progressing research and finding new treatments is critical if outcomes are to be improved for affected patients. Initiation of this trial was allowed in compliance with the UCLH Research Directorate Covid-19 and Research Policy.

Symptoms of MSA are similar to those found in Parkinson’s disease – including slowness, stiffness and tremor. MSA tends to progress more rapidly than Parkinson’s and responds poorly to Parkinson’s medications, meaning there is a huge unmet need for new MSA treatments.

Professor Tom Foltynie (UCL IoN and UCLH NHNN) is leading the pilot study looking at whether the drug exenatide – currently licensed for type 2 diabetes – can slow progression of symptoms.

For the pilot study, participants are being randomised into 2 groups, with one group adding exenatide to their regular medication. The other group will continue their regular medication alone.

Participants receiving exenatide are self-administering 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 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: “There is a huge unmet need for therapies for MSA and we hope that this trial will help to progress the search for new and effective treatments. We are also glad to be able to continue the study while in the midst of the Covid-19 pandemic. We have put in place measures to ensure the trial can continue running – and I want to thank participants for continuing to come in for study appointments, and all staff on the ground for enabling us to run this trial safely.”

For the trial, Professor Tom Foltynie is working with Professor Henry Houlden, Professor Huw Morris and Dr Dilan Athauda. Dr Sonia Gandhi, Dr Viorica Chelban, Dr Christine Girges and Dr Nirosen Vijiaratnam are also supporting 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).

During the peak of Covid-19 infections at the start of 2021 the team paused enrolment of new recruits.