New Treatment Hope for Rare Childhood Leukemia

In Episode 49 of the Game Over: c*ncer, hosts Dana Nichols and Val Solomon sit down with Dr. Elliot Stieglitz, an associate professor of pediatrics at the UCSF School of Medicine and a pediatric hematologist-oncologist who cares for children with difficult-to-treat leukemias, shares what he has learned in his time studying JMML, or juvenile myelomonocytic leukemia. From one diagnosis to creating treatment options that significantly improve survival rates for children, CKc is proud to have sponsored Dr. Stieglitz’s research not once, but twice.

You can watch his episode here: Game Over: c*ncer EP49: Dr. Elliot Stieglitz

1. The Reality Behind “Cancer Success Rates”

Not all cancers are created equal, and this episode makes that clear right away. While some childhood leukemias have survival rates nearing ninety percent or higher, JMML, or juvenile myelomonocytic leukemia, tells a very different story. It is an extremely rare disease, affecting only a small number of children each year, and historically has had far lower survival outcomes. Treatment often requires intense interventions such as stem cell transplants. This contrast reveals how general cancer statistics can obscure the urgent need for research focused on rare and difficult cases.

2. A Life-Changing Patient Encounter

Dr. Stieglitz’s journey into JMML research began with a single patient during his residency. At the time, doctors struggled to confidently diagnose the condition, relying heavily on outside experts. That moment left a lasting impression and ultimately shaped his career path. It serves as a powerful reminder that one patient experience can influence the direction of medical research and lead to long-term change.

3. The Breakthrough: JMML Is Driven by RAS Mutations

A major turning point came when Dr. Stieglitz and his team analyzed the DNA of one hundred JMML patients. They discovered that every patient had a mutation in the RAS gene pathway. This mutation causes cells to grow uncontrollably, which is a defining feature of cancer. This finding transformed JMML from a poorly understood disease into one with a clear biological cause, opening the door for more precise diagnosis and treatment.

4. From Guesswork to Precision Diagnosis

Before this discovery, JMML was diagnosed largely by excluding other diseases, which often led to long periods of uncertainty. Now, doctors can examine a patient’s DNA directly and identify the mutation responsible. This allows for a much faster and more accurate diagnosis, often within a couple of weeks. For families facing a cancer diagnosis, this clarity can make a significant difference during an already overwhelming time.

5. Not Every Child Needs the Same Treatment

Another important advancement came from studying epigenetics, which looks at chemical markers that sit on top of DNA. Researchers found patterns that help predict which patients truly need aggressive treatments like stem cell transplants and which may not. This development moves treatment toward a more personalized approach, ensuring that children receive care tailored to their specific condition rather than a one-size-fits-all solution.

6. A New Treatment Strategy Emerges

With the discovery that RAS mutations drive JMML, researchers began exploring ways to interrupt that process. While directly targeting RAS has proven difficult, they found success by targeting a related pathway using a drug called trametinib. In early trials with children whose cancer had returned after treatment, a majority showed meaningful improvement. This represents an important step toward more targeted therapies that go beyond traditional chemotherapy and transplant methods.

7. The Long Game of Research

One of the most important takeaways from the episode is that meaningful progress takes time. Research projects can span years before results are published, and even longer before they change clinical practice. Each discovery builds on previous work, creating a continuous cycle of learning and improvement. This process relies on collaboration, funding, and the willingness to invest in long-term outcomes.

Why It Matters

CKc is inspired by innovative researchers like Dr. Elliot Stieglitz. His work embodies CKc’s mission: creating new options for pediatric cancer patients by funding new, groundbreaking research. We do our work, so he can do his work, creating tangible hope.   

If you are as excited about Dr. Stieglitz’s research as CKc is and want to join our fight, visit cannonballkidscancer.org to learn more, donate, or get involved.

Know a survivor or advocate whose story needs to be shared? Email us at info@cannonballkidscancer.org to nominate a guest!