The Promise and Disappointment of Exenatide for Parkinson's Disease

(The AI LLM perplexity.ai was prompted to make the prior post more readable. I lightly edited its output.)

Exenatide, a drug used for diabetes and weight loss, showed promise for Parkinson's disease (PD) in early studies. In 2017, a small trial found that it improved motor symptoms in PD patients after a year of treatment. This exciting result led to larger studies to confirm its effectiveness.

Why Were People Excited? 

1. Early results looked good: doctors saw improvements in patients' movement.

2. The drug could get into the brain: this is crucial for treating brain disorders.

3. Lab studies suggested it might protect dopaminergic brain cells: This could potentially slow down the disease.

What Happened in the Latest Trial?

Unfortunately, the larger, more definitive trial (called Phase 3) showed disappointing results. Exenatide didn't seem to help PD patients - neither the doctors nor the patients themselves saw improvements.

What Does This Mean?

1. Complex disease: Parkinson's is complicated, and what works in small studies doesn't always pan out in larger ones.

2. Conflicting effects: The drug might have both helpful and harmful effects in the brain, canceling out any benefits. More studies are needed to know if this is the case, and if so, how to maximize the benefit relative to any risk.

3. Need for caution: Early positive results should be viewed carefully until confirmed in larger studies.

What's Next?

1. Wait for full results: We need to see all the data to understand why the drug didn't work as hoped.

2. Learn from failure: Even unsuccessful trials can teach us about PD and how to design better studies.

3. Explore other approaches: This setback doesn't mean all new treatments will fail. It emphasizes the need to try different strategies (of course, I am thinking here about RB-190).

Conclusion

While the results with GLP-1 agonists are disappointing in Parkinson's, they don't mean we should give up hope. Perhaps these drugs can be modified to maximize any potential benefit on the brain cells and minimize any risk. These studies highlight the challenges in treating Parkinson's and the importance of rigorous research. Each study, successful or not, brings us closer to understanding and potentially conquering this complex disease.