Could the Cell’s “Recycling” System Hold the Key to Rare Disease Treatment?

According to a recent study in Science Daily, scientists have found evidence that one treatment drug’s effect on the cells for a specific condition could actually be applied to several other rare disease; especially neurodegenerative ones like Parkinson’s.

Talk about multitasking!

During a study showing how an investigational drug works against Niemann-Pick type C1 (NPC1), scientists found that a related compound will activate an enzyme, AMPK, triggering a cellular “recycling” system that helps reduce cholesterol and other fats in the brains and livers of NPC1 patients.

In other words, this drug was able to turn on a cell’s ability to wash itself of bad agents that cause certain symptoms or even the disease itself.

This finding therefore can help develop treatments for other diseases where that cholesterol buildup plays a factor.

According to the study’s co-author, Wei Zheng:

“We’ve shown that a compound very similar to the repurposed drug currently in clinical testing in patients actually turns on an enzyme that jumpstarts the cell’s waste disposal system to reduce cholesterol in cells. This process, called autophagy, is what cells use to recycle their trash. The process malfunctions in NPC1 and a number of neurodegenerative diseases, making the AMPK enzyme a potential target for future drugs.”

While the treatment’s overall effect was already known, what was left unclear was exactly how the treatment works. So they’ll take a closer look at exactly why a treatment works, and apply it to other diseases that have similar profiles and causes. For example, NCP1 is characterized by too much cholesterol in the cell’s lysosomes – and the cell’s inability to break them down. This is also the case for diseases like Parkinson’s and Alzheimer’s – and a slew of other lysosomal storage and neurodegenerative diseases.

What’s good for the goose is good for the gander!

Keep researching, researchers!


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