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

21 Oct 2019

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.

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).

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About John Black Charitable Foundation

The John Black Charitable Foundation was set up in 2013, fulfilling the wishes of the late John Black. It funds research into Prostate Cancer, Parkinson’s Disease and linked conditions.

About the Van Andel Institute (VAI)

Van Andel Institute (VAI) is an agile biomedical research and education organization that unleashes innovation to multiply impact. Established in Grand Rapids, Michigan, by the Van Andel family, VAI collaborates around the world to advance bold ideas in biomedicine and education. With a staff of more than 400 and a growing network of partners, VAI studies the origins of cancer, Parkinson’s and other diseases and translates its findings into breakthrough prevention and treatment strategies to improve human health. The Graduate School at VAI offers a Ph.D. in molecular and cellular biology through a research-intensive, interdisciplinary program that prepares students for successful careers as independent investigators. VAI also is dedicated to creating classrooms where curiosity, creativity and critical thinking thrive, offering engaging programs for K–12 students and transformative professional development and instructional tools for teachers. Find out more about Van Andel Institute by visiting vai.org.

About the NIHR UCLH Clinical Research Facility at the Leonard Wolfson Experimental Neurology Centre

The Leonard Wolfson Experimental Neurology Centre (LWENC), an experimental neurology clinical facility and training programme, aimed at the understanding and treatment of neurodegenerative diseases, was set up as a partnership between the UCL and UCLH after the Wolfson Foundation donated a £20 million grant towards its creation in 2011. The LWENC Clinical Research Facility (CRF), based at the National Hospital for Neurology and Neurosurgery, opened its doors in November 2013. In April 2018, the LWENC CRF and NIHR UCLH CRF became a single NIHR CRF across two sites. Together the two sites support and safely implement an innovative portfolio, prioritising early phase mechanistic trials including immunotherapies, cell and gene therapies, and antisense oligonucleotide therapies across all therapeutic areas. The NIHR UCLH CRF works closely with, and is supported by the NIHR UCL/UCLH Biomedical Research Centre.

About UCLH

UCLH (University College London Hospitals NHS Foundation Trust) provides first-class acute and specialist services in five hospitals in North Central London. These include University College Hospital and the National Hospital for Neurology and Neurosurgery. UCLH is committed to education and research and forms part of UCLPartners which in March 2009 was officially designated as one of the UK's first academic health science centres by the Department of Health. UCLH works closely with UCL, translating research into treatments for patients. Please see our website www.uclh.nhs.uk for more information, we are also on Facebook (UCLHNHS), Twitter (@uclh), Youtube (UCLHvideo) and Instagram (@uclh).

About Defeat MSA Alliance

The Defeat MSA Alliance is composed of three inclusive nonprofits, two based in the United States and one in Canada. United in their missions, the Alliance aspires to balance efforts to support patients, educate medical professionals, raise public awareness and nurture promising research. The Alliance is the first global confederation of registered charities focused on ending Multiple System Atrophy. For further information about the Defeat MSA Alliance, visit: www.defeatmsa.org, Facebook (facebook.com/DefeatMSA), Instagram (instagram.com/Defeatmsa) and Twitter (twitter.com/Defeatmsa).

About Multiple System Atrophy Trust

The only charity supporting people with MSA in the UK iscommitted to supporting and funding innovative research into the cause and cure of MSA. The MSA Trust has committed to supporting any patient who registers in the trial in a practical way, including travel costs. Please visit the www.msatrust.org.uk for information on MSA Trust research strategy and patient support. The MSA Trust is also on Facebook (facebook.com/MSATrust1), Twitter (@MSAtrust), and Instagram (www.instagram.com/msatrust).

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