Worms!

This is a Caenorhabditis elegans worm. Commonly known as c. elegans, this worm is very important to children with ADSLD. Coming in at approximately 1mm long, it's hard to believe such a small creature could have such a huge impact, but these little wrigglers are helping us learn how to treat ADSLD.

C. Elegans worms are simple, transparent, and very easy to replicate in a lab. For our purposes, the worms are modified to have ADSLD, and then compounds are tested on them to see if the effects of ADSLD are mitigated.

In the Rare Disease Community, there is a lot of talk about what 'models' a patient advocacy group has access to. I often hear, "Do you have a mouse model?" A mouse model (or murine model) is coveted because, as a complex mammal, a mouse gives a clearer picture of the disease, as well as what might treat it. However, mice are large, expensive, and need more upkeep.

This is where c. elegans is a true WORMY HERO! We can test countless compounds on worms to narrow down what might be worth testing in a mouse model. They are a vital step in translating laboratory research into clinical use for our kids.

Today, I am thankful for these Super Worms and the researchers who study them!