Metabolic Disease Niemann-Pick May Have a New Map to Treatment Soon

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Making scientific discoveries must be a lot like trying to map a coastline in the dark. There’s a lot of bumping into things and getting your feet wet in the beginning. But the more you map, the easier it becomes. Not just because you are better at doing it, but rather because you have a frame of reference.

Scientists are doing this same thing, metaphorically speaking. They’re cartographers of the cells, organisms, planets, solar systems, galaxies, universes. As theories get proposed, they get tested and refined. We see what is wrong, and we correct it. We have a reference point and we build from there.

An example of this can be seen in researchers from the National Institute of Health. A United Press International article recently showed how scientists studying cellular recycling systems might use something they learned to help people suffering from a form of Niemann-Pick disease.

Niemann-Pick is a rare genetic disease that affects an individual’s metabolic system.

The body is unable to breakdown or dispose of certain cholesterol lipids. As a result, they end up collecting in various organs throughout the body, including the brain, liver, and spleen. There are three types of the disease; each varies in severity based on the age of onset. The life expectancy for people diagnosed with Niemann-Pick is tragically short. Most people do not reach their twenties.

The NIH researchers found that a drug being tested (2-hydroxypropyl-β-cyclodextrin) activated a process that allowed for the disposal of some cholesterol from cells. It is believed that treatment of Niemann-Pick with this drug may delay the onset and lessen the symptoms of this disease.

More research is needed, but this would add another weapon to the arsenal in the fight against this horrific disease.

Click here to read the United Press International article.

Let’s continue to watch as this possible treatment goes through trials.


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