This Little Molecule Has Big Potential to Stop Gaucher

When it comes to science and medicine, big things seldom come in big packages (get your minds out of the gutter). Groundbreaking discoveries that change our understanding of a disease or the way we treat operate on a molecular level.

So, if you want to think big, you have to start by thinking small.

The researchers at the National Institutes of Health (NIH) understand that. Every day are working to discover new frontiers in medicine by looking deep into the building blocks of disease.

And now they’ve found something that could have a huge impact on the way we’ll treat Gaucher disease and Parkinson’s disease in the future.

Using robotic tools, NIH researchers have discovered a molecule that could one day be “weaponized” as a treatment. Dr. Ellen Sidransky, a Senior Investigator at the NIH’s National Human Genome Research Institute, explains that he molecule could do what other Gaucher and Parkinson’s treatments have been unable to do:

  • cross from the bloodstream to the brain and
  • target neurons affected by the disease.

Researchers already knew people with Gaucher disease are at higher risk for Parkinson’s disease. So, they worked in the lab to create something called pluripotent stem cells. Those are “blank slate” stem cells, which can develop into different types of cells throughout the body.

The did this by using skin cells taken from different Gaucher patients: some with Parkinson’s, and some without.

The stem cells were changed into neurons that would be found in people with Gaucher disease. By examining the neurons created in the Parkinson’s versus non-Parkinson’s group, researchers were able to zero in on a protein called alpha-synuclein.

Working backwards, they started searching for a molecule that could help lower levels of the protein and, hopefully, reverse the damage caused by it.

And that’s what brings us to NCGC607. It may not look like much—in fact, it doesn’t look like anything at all because duh, it’s a molecule. But one day, with a LOT more research it could revolutionize the way we treat Gaucher and Parkinson’s.

At a minimum, it shows that there’s still a lot we have to learn. Both about the interconnections between bodies and disease, and how research is one disease can have a positive impact on another. And that’s a pretty big deal.

Go here to read more about this new discover, then check out the rest of the research going at the NIH!

Ronald Ledsen

Ronald Ledsen

After emigrating from his native Sweden, Ronald spent a stint in the Merchant Marines while trying to work out what he wanted to do with his life. He discovered a love of writing while helping a friend write anonymous Harry Potter fan-fiction online; he discovered meaning to his writing when he began journaling after an anxiety disorder diagnosis. Ronald is most relaxed when spending quiet time with his wife, two sons, and hyperactive cat.

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