Breaking News: Diabetes Drug Shows Potential as Disease-Modifying Therapy for Parkinson’s

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University College London Press Release

A drug commonly used to treat diabetes may have disease-modifying potential to treat Parkinson’s disease, a new UCL-led study suggests, paving the way for further research to define its efficacy and safety.

The study was conducted at UCL in collaboration with The Cure Parkinson’s Trust and researchers from the National Institute on Aging, and was supported by the National Institute for Health Research University College London Hospitals Biomedical Research Centre.

The study, published in The Lancet and funded by The Michael J. Fox Foundation for Parkinson’s Research (MJFF), found that people with Parkinson’s who injected themselves each week with exenatide for one year performed better in movement (motor) tests than those who injected a placebo.

“This is a very promising finding, as the drug holds potential to affect the course of the disease itself, and not merely the symptoms. With existing treatments, we can relieve most of the symptoms for some years, but the disease continues to worsen.”

Professor Tom Foltynie (UCL Institute of Neurology).

The researchers followed 60 people with Parkinson’s disease as they used either a once-weekly injection of exenatide for 48 weeks, or a placebo, in addition to their regular medications.

They found that people who used exenatide had better motor function at 48 weeks when they came off the treatment, which persisted after the 12-week follow-up. Those who had injected the placebo showed a decline in their motor scores at both the 48- and 60-week tests. The advantage of 4 points, on a 132-point scale of measures such as tremors, agility and speech, was statistically significant.

The participants did not report noticeable improvements in their symptoms during the trial period beyond what their standard medication already did for them. They were tested while temporarily off all medication, to determine how the disease itself was progressing. The research did not determine conclusively whether the drug was modifying the disease itself, so the next stage in the research will investigate that more fully.

Parkinson’s disease affects 1 in 500 people and is the second most common neurodegenerative disease worldwide. Symptoms typically don’t become apparent until over 70% of the brain’s dopamine-producing cells have been affected. The condition results in muscle stiffness, slowness of movement, tremors, sleep disturbance, chronic fatigue and an impaired quality of life.

The saliva of the Gila monster lizard provided the inspiration for the development of exenatide, which has been used since 2005 to treat Type 2 diabetes. It activates receptors for the GLP-1 hormone in the pancreas to stimulate insulin release. GLP-1 receptors are also found in the brain, and prior research has shown that activating them can boost the function of dopamine connections, act as an anti-inflammatory, improve energy production, and switch on cell survival signals. Further research by a team led by Professor Foltynie will seek to clarify how exenatide works for people with Parkinson’s disease.

Prior evidence in animal models demonstrated that exenatide improved motor performance. Another study also found early evidence that it could be a disease-modifying agent for Parkinson’s, but it was an open-label trial, so this latest study strengthens the existing evidence as the first randomised, placebo-controlled trial of the drug for Parkinson’s patients.

“This is the strongest evidence we have so far that a drug could do more than provide symptom relief for Parkinson’s disease. ”

Professor Foltynie

“Using approved therapies for one condition to treat another, or drug repurposing, offers new avenues to speed Parkinson’s therapeutic development,” said Dr Brian Fiske, senior vice president of research programs at MJFF. “The results from the exenatide studies justify continued testing, but clinicians and patients are urged not to add exenatide to their regimens until more is known about their safety and impact on Parkinson’s.”

“While we are optimistic about the results of our trial, there is more investigation to be done, and it will be a number of years before a new treatment could be approved and ready for use. We also hope to learn why exenatide appears to work better for some patients than for others,” said the study’s first author, Dr Dilan Athauda (UCL Institute of Neurology).

The researchers say the next step will be a longer-term study with more participants, which will investigate whether there are marked improvements in quality of life.

**The groundbreaking results of this study and related research on GLP-1 agonists were discussed at a recent collaborative meeting hosted by The Cure Parkinson’s Trust during which attendees discussed drugs commonly used to treat diabetes which hold promise for repurposing as potential Parkinson’s therapies. Click here to read more…

Professor Tom Foltynie discussing the Exenatide trial results

 

Notes to Editors

For more information or to speak to the researchers involved, please contact Chris Lane, UCL press office. T: +44 (0)20 7679 9222 / +44 (0)7717 728 648, E: chris.lane@ucl.ac.uk

Dilan Athauda,  Kate Maclagan,  Simon S. Skene,  Martha Bajwa-Joseph, Dawn Letchford, Kashfia Chowdhury,  Steve Hibbert, Natalia Budnik,  Luca Zampedri, John Dickson,  Yazhou Li, Iciar Aviles-Olmos, Thomas T. Warner, Patricia Limousin, Andrew J. Lees, Nigel H. Greig, Susan Tebbs and Thomas Foltynie, ‘A randomised, double-blind, placebo controlled trial of Exenatide once-weekly in Parkinson’s disease,’ published in The Lancet

About UCL (University College London)

UCL was founded in 1826. We were the first English university established after Oxford and Cambridge, the first to open up university education to those previously excluded from it, and the first to provide systematic teaching of law, architecture and medicine. We are among the world’s top universities, as reflected by performance in a range of international rankings and tables. UCL currently has over 39,000 students from 150 countries and over 12,500 staff. Our annual income is more than £1 billion.

About the National Institute for Health Research University College London Hospitals Biomedical Research Centre

The National Institute for Health Research (NIHR) is funded by the Department of Health to improve the health and wealth of the nation through research.

The NIHR is the research arm of the NHS. Since its establishment in April 2006, the NIHR has transformed research in the NHS. It has increased the volume of applied health research for the benefit of patients and the public, driven faster translation of basic science discoveries into tangible benefits for patients and the economy, and developed and supported the people who conduct and contribute to applied health research.

The NIHR plays a key role in the Government’s strategy for economic growth, attracting investment by the life-sciences industries through its world-class infrastructure for health research. Together, the NIHR people, programmes, centres of excellence and systems represent the most integrated health research system in the world.

Published: 3rd August, 2017