In 2014, experts from across the country came together at CSHL’s Banbury Center to discuss rhabdomyosarcoma (RMS), a tissue cancer mainly diagnosed in children. Now, CSHL Professor Christopher Vakoc could be at a turning point. You may have seen Vakoc on FOX. This week At the Lab, he brings us an RMS breakthrough 10 years in the making.
Read the related story: Once rhabdomyosarcoma, now muscle
Transcript
Marc Persad: You’re now At the Lab with Cold Spring Harbor Laboratory. My name is Marc Persad and this week At the Lab, “Outmuscling cancer.”
MP: Diseases aren’t cured overnight. It takes years, often decades, for biomedical breakthroughs to materialize. But every now and again there’s a turning point—a moment when years of hard work start to pay off. CSHL Professor Christopher Vakoc may be rounding such a corner.
Christopher Vakoc: We can’t see the future. But every successful drug has its origin story. And studies like this are the soil out of which new drugs are born. It’s a fundamental discovery. It’s learning one of the most important molecules in a disease that kids are dying of.
MP: The disease he’s talking about is rhabdomyosarcoma, or RMS, a form of cancer that mostly occurs in children and teenagers. The molecule is a protein called NF-Y. We’ll return to that shortly. But first, let’s talk about RMS and how the Vakoc lab came to study it.
CV: The name rhabdomyosarcoma refers to the similarity of the tumor to muscle stem cells. There’s not a lot of research dollars directed at this cancer. And that’s a big part of how we got into this. There are families that have lost children or spouses to this disease on Long Island. And they all came together and funded us to try to find a new therapeutic strategy for this disease.
MP: About 10 years ago, the Vakoc lab had a radical idea. If RMS cells are essentially muscle stem cells that never transformed into muscles, maybe they could stop the cancer’s growth by completing the cells’ transformation. It took years to develop the genetic screening tests needed to pinpoint the molecular machinery that would cause RMS cells to change. But finally, NF-Y emerged. Vakoc’s team used CRISPR to target NF-Y inside the RMS cells. And that’s when it happened.
CV: These tumor cells, which can’t contract, now become a muscle cell that has the whole contraction machine. It’s like a fork in the road. The cell has gone down one path, but now it can’t go back to its multiplying state because all of its energy and resources are devoted to contraction.
MP: A fork in the road for RMS cells and just maybe a turning point for RMS research, and it all happened here, At the Lab.
MP: Thanks for listening. To hear more fascinating science stories like this one, please subscribe wherever you get your podcasts and visit us online at CSHL.edu. For Cold Spring Harbor Laboratory, I’m Marc Persad, and I’ll see you next time At the Lab.