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Scientists have hailed an important advance in the decades-old quest for a drug to slow the impact of Parkinson’s disease, in research that highlights the potential versatility of new diabetes and weight-loss drugs.
Parkinson’s patients who took the diabetes drug lixisenatide saw no worsening of their motor symptoms after a one-year trial, according to a paper published in The New England Journal of Medicine on Wednesday.
The results from the research, which involved 156 people, were a “major breakthrough”, said Olivier Rascol, a Toulouse University Hospital professor and a co-author of the research with Prof Wassilios Meissner, his Bordeaux University Hospital counterpart.
“If our pilot data are confirmed, it is going to dramatically change the quality of life of the patients,” said Rascol.
Parkinson’s is a brain disorder that affects an estimated 10mn people worldwide and leads to symptoms including body tremors, slow movement and — in severe cases — dementia.
The results will renew attention on the possible effectiveness of lixisenatide, which is made by French pharmaceutical group Sanofi, and other so-called GLP-1 drugs against neurodegenerative conditions.
The drugs mimic the GLP-1 gut hormone to trigger the release of insulin. They include the popular new diabetes and weight-loss drugs Ozempic and Wegovy, made by Novo Nordisk, and Mounjaro and Zepbound made by Eli Lilly. Soaring sales of the products have pushed the companies’ valuations to record highs.
Novo Nordisk is trialling GLP-1 treatments on conditions including cardiovascular diseases and Alzheimer’s, but the lixisenatide trial is one of the first longer studies to suggest that a GLP-1 drug may slow progression of Parkinson’s symptoms in humans.
Rascol stressed that some GLP-1 drugs may not be effective against Parkinson’s because they “do not cross the blood-brain barrier easily”, referring to a semi-permeable membrane that can stop molecules passing between the two.
The results of the latest trial would require further research and testing to work out optimal treatments, Rascol added, along with more supplies of lixisenatide. The drug is marketed by Sanofi as Lyxumia in the EU and Adlyxin in the US, where it was discontinued last year because of low demand.
The company, which provided lixisenatide free of charge to the French research but did not sponsor it, welcomed the results. It said it was “open to a discussion with the investigators of the study on providing support for their next phase of research”.
The French trial results were “really encouraging” because they showed people who were on the drug were “significantly better off in their movements” after a year than those who didn’t take it, said Masud Husain, a neurology professor at Oxford university.
But he said the findings did not provide “conclusive evidence” that the drug helped protect the brain, while he noted the prevalence of side-effects such as nausea in more than 45 per cent of patients and vomiting in 13 per cent.
A smaller 2017 trial of exenatide, another GLP-1 diabetes drug originally marketed by Eli Lilly in 2005, showed improvement in motor symptoms for patients with moderate Parkinson’s. A phase 3 trial led by researchers at University College London is due to report its findings this year.
The trial results on exenatide so far suggested it could be even more beneficial than lixisenatide, said Prof David Dexter, director of research at the charity Parkinson’s UK.
“The Parkinson’s community urgently needs new treatments so they, and we, eagerly await the results from this ongoing phase 3 trial, later this year.”
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