The Power of Early Investment

Early in his career, pediatric oncologist and physician-scientist Elliot Stieglitz, MD, began studying a rare childhood leukemia. His work has since changed how the disease is diagnosed and treated. We sat down with Dr. Stieglitz, the William Fries II Professor of Pediatric Oncology, to discuss how early philanthropic support helped shape his career.

What drew you to pediatric oncology?

I wanted to be a doctor for as long as I can remember. During medical school, on my first rotation in pediatrics, I saw what that could really look like. What stood out to me was the connection — not just with the patient but also with the entire family. In pediatric oncology, you become part of the patient’s inner circle, along with the parents, the grandparents, even the family dog and the next-door neighbors! It’s a real privilege to be a part of that.

Can you take us back to the moment you first encountered JMML?

During my residency, I was taking care of a patient who was suspected of having this rare type of blood cancer called JMML — juvenile myelomonocytic leukemia. As soon as this diagnosis came up as a possibility, everyone said, “Call Dr. Mignon Loh [former chief of pediatric oncology] at UCSF.” I called her, and she helped us make the diagnosis using a brand-new molecular technique. That experience led to my decision to pursue oncology training at UCSF.

What made UCSF stand out to you?

This type of work often is divided — you’re either a physician or a scientist. But UCSF excels in this area because these roles come together. As physician-scientists, we understand which discoveries are most likely to help patients, and that perspective shapes the research so what we learn in the lab can more quickly translate into better care. I realized this was exactly the career I wanted, and I don’t think I could have accomplished what our lab has done anywhere else.

How did your early research begin to change how JMML is diagnosed?

When I first started in 2011, JMML was often diagnosed by ruling out other, more common types of leukemia. If a child didn’t fit into those other categories, they would be diagnosed with JMML. We didn’t think that was good enough, so we developed a molecular test to identify JMML. We discovered that 100% of JMML patients have a mutation in the RAS family of genes. That gave us a way to definitively identify the disease and ultimately changed how JMML is diagnosed and defined around the world.

When you cure a child who is 2 years old, you’ve given them the opportunity to live an entire lifetime. There’s nothing better than that.

What does that shift mean for patients and families?

Families regularly tell me that getting to a diagnosis is a huge ordeal. Patients are often sick and moving between hospitals as doctors search for answers, and getting a diagnosis can take months. With this test, we can make a definitive diagnosis within two weeks.

Where did you take the research from there?

After improving the ability to make a rapid diagnosis, we began exploring which patients needed which treatment. Historically, every patient received a bone marrow transplant, which is the most intensive treatment we offer as oncologists. But we knew that some didn’t need that. We just didn’t have a method for identifying them.

We developed a test that analyzes patterns in DNA to better guide treatment. We’re currently running a clinical trial in which, based on the results of this test, certain patients can avoid a bone marrow transplant. Now, approximately one-third of our patients are avoiding a treatment we once thought was necessary for everyone.

How were you able to get this research off the ground so early in your career?

It really started with philanthropic support. During my fellowship, I received a series of young investigator awards from private philanthropic organizations, which gave me the time and support I needed to develop my expertise at a career stage when that kind of support is hardest to find. Those awards really helped bridge that gap between "trainee" and "independent investigator."

How does philanthropy continue to support this work today?

The clinical trial we’re running now costs between $3 million and $4 million, and the only source of funding for a clinical trial of that sort is the federal government. But to secure that level of funding, you need strong preliminary data. Philanthropic donations from individuals and organizations funded the early work that generated that data and made this trial possible. As a public institution, this becomes even more important.

Do you see pediatric cancer research as an area where private investment can make a unique difference?

Yes! Only about 4% of all federal funding for cancer research goes to pediatric cancer. Pediatric cancer is relatively rare, but when a child is diagnosed with cancer, it’s everything for that family. When you cure a child who is 2 years old, you’ve given them the opportunity to live an entire lifetime. There’s nothing better than that.

 

For more information about how you can invest in the work of a promising young researcher like Dr. Stieglitz, contact Chris Anderson, Senior Executive Director of Development, at [email protected] or (415) 994-0160.