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

10 comments

This is when I miss r/science. There is always someone that critiques the study and balance expectations.

This is wonderful news!

Would be awesome if this pans out. That disease is horrific. It isn't just tremors but also dementia and depression.

Related article: https://www.frontiersin.org/articles/10.3389/fphar.2022.851654/full
Best I can deduce USP30 is in the mitochondrial wall and its function is to regulate mitochondrial apoptosis.
"At the cellular level, several studies provide evidence for the pivotal role of USP30 involved in mitochondrial cell death and apoptosis. Mitochondria are fundamental in the orchestration of cell death pathways as they play a central role in energy production and metabolism."

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.

And this is all we're ever hear about it.
Remember a few months back when a study came out about a new material that was supposed to be a room temperature superconductor.
Pepperidge farms remembers.
- We heard a lot about that. It would have been revolutionary and many teams tried to replicate it. Their failure to do so, and its subsequent discredit, are well documented.
  
  No we did not. Just because you kept up with it doesn't mean it didn't fall into obscurity regardless of its success or in this case failure.
  All revolutionary claims like this have some effect in the circles that refer to it.
  My comment is reflective of the failure of that substance.

10 comments