1y ago

Groundbreaking Discovery: Inhibiting Key Enzyme May Halt Parkinson’s Disease Progression

scitechdaily.com Groundbreaking Discovery: Inhibiting Key Enzyme May Halt Parkinson’s Disease Progression

The findings have the potential to pave the way for new disease-modifying treatments for patients with Parkinson’s disease. A study published in Nature Communications by Beth Israel Deaconess Medical Center (BIDMC) investigators provides new insights into cellular mechanisms in Parkinson's disease

This study reveals that inhibiting a specific enzyme, USP30, in a mouse model can protect dopamine-producing neurons. This finding halts the disease’s progression and opens the door to new therapeutic possibilities for the 10 million people affected by Parkinson’s worldwide.

DOI: 10.1038/s41467-023-42876-1

https://www.nature.com/articles/s41467-023-42876-1

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Man, I love studies like these but I wish they would release a ‘For Dummies’ version so a dummy like me can better understand the study and its findings.
- "In conclusion, we used a strategy for upregulating mitophagy by Usp30 deletion, with similar results obtained with a pharmacological USP30 inhibitor. These strategies to reduce USP30 lead to enhanced mitophagy and potent protection against αSyn toxicity. This work validates inhibition of USP30 as a promising strategy for further testing for potential disease-modifying effects in PD."
  Mitophagy - clearing out old stuff from cells. Clears out dysfunctional mitochondria from cells.
  aSyn toxicity - alpha synuclein - a protein found in the body, esp. in the brain. [We think it helps with some neruo-regulation, and DNA repair. We think these two go together. ] In people with diseases like Alzheimers an Parkinsons there appears a 'buildup' of αlpha-synuclein in places and a corresponding degeneration of other areas.
  This study tried to crank up the clearing out of old mitochondria in Mice first by modifying their genes to removing (knocking out) the ability to make USP30 (an enzyme). They also cooked up a molecule that could just block USP30 (it gets there first and sits in the way).
  Both methods worked to protect mice when they tried to give them alpha synuclein problems. Of course there's lots of other questions about what else is affected, but this has potential to be fix this one part of the problem.

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