Transcription Factor Targeting Advances Pediatric Cancer Treatment

Because childhood cancers are rare, finding funding and developing new treatments for them is incredibly difficult; in fact, the standard chemotherapy for Ewing sarcoma (a rare bone and tissue cancer) hasn’t fundamentally changed in over 40 years. To break this standstill, Dr. John Bushweller at UVA Health is working with an international group called C-Further to design a brand-new type of drug. Instead of using harsh, traditional treatments that can cause long-term damage to a child’s heart or brain, they are creating a targeted therapy that attacks the specific roots of the cancer while leaving the rest of the body alone.

This new approach focuses on “transcription factors,” which are special proteins that act like master switches to turn genes on and off inside our cells. Scientists used to think these proteins were impossible to target with medicine, but Dr. Bushweller’s lab discovered that Ewing sarcoma cells completely depend on a specific switch called ETV6 to stay alive. By creating a drug that safely flips this specific switch to “off,” they can starve and kill the cancer cells. While manipulating these powerful master switches carries a risk of disrupting other proteins, the team is carefully designing the drug to be highly precise, aiming to give children a much safer treatment with far fewer toxic side effects.

Read the full article