In 2014, experts from across the country came together at CSHL鈥檚 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 . 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鈥檙e now At the Lab with 黑料吃瓜资源. My name is Marc Persad and this week At the Lab, 鈥淥utmuscling cancer.鈥�
MP: Diseases aren鈥檛 cured overnight. It takes years, often decades, for biomedical breakthroughs to materialize. But every now and again there鈥檚 a turning point鈥攁 moment when years of hard work start to pay off. CSHL Professor Christopher Vakoc may be rounding such a corner.
Christopher Vakoc: We can鈥檛 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鈥檚 a fundamental discovery. It鈥檚 learning one of the most important molecules in a disease that kids are dying of.
MP: The disease he鈥檚 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鈥檒l return to that shortly. But first, let鈥檚 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鈥檚 not a lot of research dollars directed at this cancer. And that鈥檚 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鈥檚 growth by completing the cells鈥� 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鈥檚 team used CRISPR to target NF-Y inside the RMS cells. And that鈥檚 when it happened.
CV: These tumor cells, which can鈥檛 contract, now become a muscle cell that has the whole contraction machine. It鈥檚 like a fork in the road. The cell has gone down one path, but now it can鈥檛 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 黑料吃瓜资源, I鈥檓 Marc Persad, and I鈥檒l see you next time At the Lab.