49. New Treatment Hope for Rare Childhood Leukemia

What does it really take to develop new treatments for pediatric cancer?In this episode of Game Over: c*ncer, we sit down with Dr. Elliot Stieglitz, a leading pediatric hematologist-oncologist and researcher at UCSF, to break down the reality behind pediatric cancer research, clinical trials, and breakthrough treatments for rare diseases like juvenile myelomonocytic leukemia (JMML).JMML is a rare and aggressive form of childhood leukemia affecting just 50–100 children per year in the United States, yet it presents some of the biggest challenges in diagnosis and treatment. Historically, survival rates have been low, and treatment options have been limited.Dr. Stieglitz shares how his groundbreaking research into RAS gene mutations and DNA profiling has:Revolutionized how JMML is diagnosedEnabled faster, more accurate testing worldwideHelped doctors predict which patients need aggressive treatmentOpened the door to targeted therapies and clinical trialsWe also dive into the reality of medical research timelines, why innovation takes years, and how nonprofit funding plays a critical role in bringing life-saving treatments to children.If you’ve ever wondered how pediatric cancer breakthroughs actually happen—or what it takes to move from research to real-world impact—this episode is for you.----------------------------------Connect with Dana: https://www.linkedin.com/in/danaknichols/Connect with Val: https://www.linkedin.com/in/valerie-solomon/Upcoming Ckc Events: https://cannonballkidscancer.org/category/make-an-impact/events/----------------------------------Podcast Produced by Hi Hello Labs: Website: https://www.hihellolabs.com/