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Can Lixisenatide Revolutionize Parkinson's Treatment or Fall Short on Promise?


Diabetes drug lixisenatide shows promise in Parkinson's trial, but faces challenges in efficacy, side effects, and availability.

By Athena Xu

4/3, 18:39 EDT

Key Takeaway

  • Lixisenatide shows promise in slowing Parkinson's progression, but challenges like blood-brain barrier penetration and side effects remain.
  • Discontinuation of lixisenatide in the US raises concerns about its availability for further Parkinson's research.
  • Despite optimism, the community awaits conclusive evidence from ongoing trials of similar drugs.

Breakthrough or Hype?

Recent research has sparked interest in the potential of diabetes and weight-loss drugs to slow the progression of Parkinson's disease, a condition affecting an estimated 10 million people worldwide. A study involving the diabetes drug lixisenatide, made by French pharmaceutical giant Sanofi, showed that Parkinson’s patients experienced no worsening of their motor symptoms after a one-year trial. While hailed as a "major breakthrough" by some, including Olivier Rascol, a professor at Toulouse University Hospital, the findings have also raised questions about the broader applicability and side effects of such treatments.

Challenges Ahead

Despite the optimism, the study's results, involving 156 people, underscore the complexity of translating these findings into a viable treatment option for Parkinson's disease. Rascol himself cautioned that some GLP-1 drugs might not be effective against Parkinson’s due to their inability to easily cross the blood-brain barrier, a critical factor in treating neurological conditions. This limitation suggests that not all drugs in this class will be suitable for Parkinson's patients, potentially narrowing the field of viable treatments.

Moreover, the discontinuation of lixisenatide in the US market last year due to low demand raises concerns about the drug's availability and the pharmaceutical industry's commitment to providing it for Parkinson's research. While Sanofi has expressed openness to supporting further research, the path to a widely available and effective treatment remains uncertain.

Side Effects and Skepticism

The enthusiasm for lixisenatide and similar GLP-1 drugs must be tempered by the reality of their side effects. The French study reported that more than 45% of participants experienced nausea, and 13% suffered from vomiting. These side effects are not trivial and could significantly impact the quality of life for Parkinson's patients, potentially outweighing the benefits of slowed disease progression.

Masud Husain, a neurology professor at Oxford University, pointed out that the study does not provide "conclusive evidence" that the drug helps protect the brain. This skepticism is echoed by the Parkinson's community, which, according to Prof David Dexter, director of research at Parkinson’s UK, "urgently needs new treatments." The anticipation for the results from an ongoing phase 3 trial of exenatide, another GLP-1 drug, later this year, underscores the cautious optimism and hunger for definitive answers within the community.