"The search for fundamental knowledge, motivated by curiosity, is as useful as the search for solutions to specific problems" -C.H. Llewellyn Smith This week, my Molecular Medicine seminar was lucky enough to hear from Dr. Bob Horvitz, a Nobel prize winner in Medicine in 2002. His story has me thinking about the usefulness of basic research. Dr. Horvitz studies C. elegans, a tiny worm that serves as a common model organism in biology labs. I have fond(ish) memories of long hours spent staring at these little guys through a microscope in undergrad lab classes. But although their ease of use recommends them as a lab model, you might wonder how useful these microscopic worms could be. Dr. Horvitz told us the story of how he began studying cell death in C. elegans. The worms are so tiny they only have 959 cells in their whole body (for reference, you have about 50 trillion). But, 131 other cells are created, and never make it to the final adult worm. Some cells die off as the animal develops. Until then, biologists mostly thought about cell death as something that happened when things went wrong. But it seemed like these cells were dying in purpose. Dr. Horvitz' lab used genetic studies of C. elegans to discover several genes that were responsible for "programmed cell death"- a purposeful, important biological process, now termed apoptosis. Cells need to die at the right time in order for worms to develop normally. And, it turns out, the same holds true for you and I. Some cells need to die off for you to stay healthy. There are some diseases that involve too much cell death (for example, neurodegenerative diseases like Huntington's), and some diseases that involve too little (for example, cancer is an outgrowth of cells that won't die off). Healthy bodies have to create a balance. Some time after discovering the cell death genes in C. elegans, Dr. Horvitz noticed human genes that were very similar to his worm genes. He and his colleagues started companies to study these human genes in more detail. It turns out that these genes act very similarly to their C. elegans counterparts - by causing (or preventing) apoptosis. Years later, those companies now have Phase II and III clinical trials in humans going on, testing drugs that target these genes in order to treat diseases of apoptosis gone wrong. It's one of the most direct stories I've heard about translating basic scientific research to human medicines. Nobody predicted that by counting all the cells in microscopic worms, they would one day invent a drug to treat leukemia. And yet, without the discovery of programmed cell death in C. elegans, it would have taken untold years before we could appreciate its importance in humans. It is a potent reminder that, although I am passionate about research with the explicit goal of curing diseases - and specifically looked for PhD projects that would allow me to do so - basic science for science's sake is just as useful an undertaking. Scientists' curiosity about C. elegans has led to countless important discoveries. Whether you like it or not, we owe a lot of thanks to a bunch of worms.
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AuthorAmanda Kedaigle's work in the Broad Institute focuses on leveraging brand new biological data modalities to study novel models of human brain development. Archives
February 2022
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