Elliot Stieglitz, MD

About Dr. Elliot Stieglitz, MD

Elliot Stieglitz, MD is a pediatric oncologist at UCSF Benioff Children’s Hospital. His research is focused on developing innovative new targeted therapies for patients with juvenile myelomonocytic leukemia (JMML).

About Dr. Stieglitz’s Research

JMML is a type of blood cancer that affects infants and young children, causing such symptoms as stomach pain, difficulty breathing and bleeding problems. If untreated, JMML is nearly always fatal.

The only way to cure JMML is via stem cell transplantation, an intensive medical procedure with numerous potential side effects, including liver disease, kidney disease and an increased risk of severe infections. Equally concerning is that this intensive treatment only works half the time with few children surviving in instances where the procedure fails.

Since 2019, the Pediatric Cancer Research Foundation has helped fund Dr. Stieglitz’s research into JMML treatment and risk stratification through yearly Designated Grant awards. Over the past several years, Dr. Stieglitz has developed a diagnostic tool designed to predict which patients are likely to respond positively to treatment and which patients are not. Dr. Stieglitz’s risk stratification research is now a clinical test that can be ordered by doctors across the country to help them decide the best course of treatment for patients with JMML.

In conjunction with his risk stratification research, the Pediatric Cancer Research Foundation has also helped fund Dr. Stieglitz’s study of trametinib, an oral medication that shows promise as a less-intensive treatment option for JMML patients who haven’t responded to other forms of treatment. Following successful testing of trametinib in combination with other drugs using mice models, Dr. Stieglitz recently received additional funding from the National Institute of Health to take his work into a clinical trial with human subjects.

This clinical trial, known as T2020-004: Risk stratified treatment for patients with newly diagnosed juvenile myelomonocytic leukemia: A Phase I/II non-randomized study of trametinib and azacitidine with or without chemotherapy (IND #164058), is being conducted through the Therapeutic Advances in Childhood Leukemia and Lymphoma (TACL) Consortium and supported by the Foundation.

This clinical trial, which is now open to enrollment, combines Dr. Stieglitz’s risk stratification techniques with his trametinib research and helps pinpoint which patients are most likely to benefit from the medication.

“This is the first time we will be able to risk-stratify patients with JMML and tailor treatments based on an individual patient’s risk of relapse. In prior clinical trials for JMML, all patients have received the same exact treatment, a stem cell transplant. Now we have the clinical biomarker we need to tell us how to treat patients differently,” Dr. Stieglitz explains. 

Dr. Stieglitz believes this research has the potential to greatly improve the lives of children diagnosed with JMML.