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Diabetes drug could halt Parkinson's progression

Optimistic researchers say exenatide presents 'major new avenue for investigation'

Mark Gould

Friday, 04 August 2017

Exenatide, a drug normally used in type 2 diabetes, may be a potent weapon in halting the progression of Parkinson's disease, a new clinical trial* suggests.

The trial on 62 patients seems to show that the medicine halted the progression of the disease. Writing in The Lancet, a University College London (UCL) team said it was "excited", by its findings but it urged caution as any long-term benefit is uncertain and the drug needs more long-term testing to see if it holds back progression of the disease in the long term.

"There's absolutely no doubt the most important unmet need in Parkinson's is a drug to slow down disease progression, it's unarguable," Prof Tom Foltynie, one of the researchers, told the BBC.

Exenatide helps control blood sugar levels in diabetes by acting on the hormone sensor GLP-1. Those sensors are found in brain cells too. Scientists suggest that the drug makes those cells work more efficiently or helps them to survive.

In the trial, half of patients were given exenatide and the rest were given a placebo. All the patients stayed on their usual medication.

As expected, those on just their usual medication declined over 48 weeks of treatment. But those given exenatide were stable. And three months after the experimental treatment stopped, those who had been taking exenatide were still better off.

"Exenatide had positive effects on practically defined off-medication motor scores in Parkinson's disease, which were sustained beyond the period of exposure. Whether exenatide affects the underlying disease pathophysiology or simply induces long-lasting symptomatic effects is uncertain. Exenatide represents a major new avenue for investigation in Parkinson's disease, and effects on everyday symptoms should be examined in longer-term trials," the authors conclude.

Prof Foltynie told the BBC News website: "This is the first clinical trial in actual patients with Parkinson's where there has been anything like this size of effect.

"It gives us confidence exenatide is not just masking symptoms, it's doing something to the underlying disease.

"We have to be excited and encouraged, but also cautious as we need to replicate these findings."

David Dexter, the deputy director of research at Parkinson's UK, said: "The findings offer hope that drugs like exenatide can slow the course of Parkinson's -  something no current treatment can do.

"Because Parkinson's can progress quite gradually, this study was probably too small and short to tell us whether exenatide can halt the progression of the condition, but it's certainly encouraging and warrants further investigation."

Dr Brian Fiske, from The Michael J Fox Foundation for Parkinson's Research, which funded the study, said: "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."


* Athauda D, Maclagan K, Skene SS, et al. Exenatide once weekly versus placebo in Parkinson's disease: a randomised, double-blind, placebo-controlled trial. The Lancet, published: 03 August 2017. DOI: 10.1016/S0140-6736(17)31585-4

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