'There’s a lot of work that needs to be done’

Dr. Federico keeps pressing to improve outcomes for kids with challenging cancers like high-risk neuroblastoma

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  •  7 min

Clinical trials led by Dr. Federico focus on innovative approaches for treating high-risk solid tumors like neuroblastoma

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St. Jude Children’s Research Hospital has named Sara M. Federico, MD, director of the institution’s Solid Tumor Division within the Department of Oncology. 

Federico is an internationally recognized leader in pediatric oncology whose contributions have defined the landscape of treatment for high-risk solid tumors in kids, such as neuroblastoma. 

She is currently leading five clinical trials, including two pioneering new approaches to treat neuroblastoma. The first is a large international Phase 3 study for children with newly diagnosed high-risk neuroblastoma being conducted through the Children’s Oncology Group, a large pediatric cancer cooperative group. The second is a multi-site Phase 2 study evaluating a novel treatment combination for children with relapsed or refractory neuroblastoma, who historically have a survival rate less than 20 percent.

“I’m thrilled for this opportunity to lead a collaborative, dedicated team of investigators that is passionate about improving cure rates for children with solid tumors,” said Federico. “Together we hope to advance the field through the translation of critical scientific discoveries into innovative therapies and clinical trials which will ultimately lead to improved patient outcomes.”

Federico recently spoke with St. Jude donors and supporters about the work that remains to improve survival in challenging childhood cancers like high-risk neuroblastoma. 

This cancer comes from immature cells in the nervous system and is often found in the adrenal glands on top of kidneys. Neuroblastoma affects roughly 800 kids in the U.S. each year, most under the age of 5, and unfortunately the cancer has already spread to other body parts in more than half of the diagnosed patients.  

Treatment for neuroblastoma can be particularly grueling.  For patients with high-risk neuroblastoma, it requires multiple rounds of chemotherapy, radiation, surgery, two bone marrow transplants, immunotherapy and biologic agents. Unfortunately, sometimes, even all of that therapy is not enough to eliminate the disease. For decades, barely half of the kids diagnosed with high-risk neuroblastoma have survived. 

Q: You like to say: “At St. Jude, we don't care who cures cancer, we just need to advance the cure.” Could you expand on that sentiment?

A: When we think about what our founder Danny Thomas said, that “no child should die in the dawn of life,” he didn't end that sentence with “at St. Jude” or “in the United States”. He meant anywhere. We owe it to our patients and their families to work together, both within the institution and outside of it, to push that cure rate forward. It’s that kind of collaboration that gets better treatment into the hands of people that need it.

Q: You've been at St. Jude since your fellowship began in 2007, when patients with high-risk neuroblastoma had a 30 percent survival rate. By the time you finished your fellowship and joined the staff at St. Jude in 2011, 3-year event free survival rates for high-risk neuroblastoma had risen to 51 percent.

What is neuroblastoma and how does your team work to make progress with improving cure rates?

Neuroblastoma, a challenging childhood cancer, drives Dr. Federico’s dedication to advancing patient outcomes

A: Neuroblastoma is a cancer that starts in the nervous system. It's the second most common pediatric solid tumor, behind brain tumors, and in 50% of patients at the time of diagnosis their disease has already spread. Many of these patients are categorized as having high-risk neuroblastoma. 

Thankfully we've been able to advance cure rates in high-risk neuroblastoma, but it's come at a cost of having to add a lot more therapy to the patients to advance that cure.

A couple years back, we decided to test whether adding a certain type of antibody therapy to the beginning, the middle and the end of the treatment protocol would improve survival. Before that, we used to just add the antibody therapy to the end of treatment.

You may be wondering, “What is antibody therapy?” Well, imagine that tumor cells are like a circle with little flags hanging on them. An antibody goes around the body looking for that flag. Once it binds to that flag that's on a tumor cell it marks it for destruction. We then give medicines to make the immune system recognize the tumor plus antibody and destroy the cancer cell. 

So, we conducted a trial (with a group of 64 patients here at St. Jude) and identified that giving antibody therapy with chemotherapy during the beginning of treatment as well as at the end was tolerated and more patients were cured. Now, the next step is to study if this treatment regimen, in a larger setting, has the same improved survival rate. So, that's the phase three study that will open this year.

Q: That’s promising work. What drives you? 

A: When I went into medicine, I really wanted to be able to look back and say, “Hmm, did I participate in anything that really helped children?” not just on an individual level, but as a group. When I selected oncology as my career, my dream was to advance cure rates, so that more children will have the chance to grow up and change the world.  This is what drives me and it's been thrilling to be a part of the research that helps advance cure rates for high-risk neuroblastoma. 

But there is so much more work that needs to be done. As I said earlier, there are approximately 800 cases of neuroblastoma diagnosed per year in the United States. But worldwide, there are thousands of patients diagnosed with neuroblastoma every year and many of those patients don't have access to the therapy that we have here within the United States. Additionally, about 30 percent of patients who are diagnosed in the U.S. with high-risk neuroblastoma will relapse. Sadly, when patients relapse, it's much harder to get rid of the cancer. So, we can’t slow down; there are still many more children that need better treatment. 

Q: What is your why? What keeps you coming to St. Jude to work every single day?

A: (laughs) How much time do you have?

There's one patient story that sums up my why. This patient of mine was diagnosed with Ewing Sarcoma as a teen. Ewing sarcoma is the second most common bone tumor in children and young adults. Unfortunately, if at the time you are diagnosed with Ewing sarcoma it's already spread, your chance of cure is only about 30 percent.

Well, that's the situation that this young man was in. He was diagnosed with metastatic Ewing sarcoma and unfortunately his aggressive cancer relapsed multiple times.  He also happened to be a talented concert pianist who loved to play music at churches and travel around the country performing concerts. So, as we treated him in our clinic we always factored in his love for music and concert schedule. During the time he was treated for relapsed disease, through a partnership with our basic scientists, we identified a new therapy that had just come from the lab to the clinical space.

Thankfully it worked for a long period of time, and we were able to get his disease to stabilize. But eventually the Ewing sarcoma came back. And I will never forget when we got a call that he was travelling and was short of breath and needed to come back to St Jude.

I knew in my heart that this wasn't going to be good. He arrived that night and went straight to the intensive care unit. The next morning, we got a CT scan and I had to share with him that he was not going to survive his cancer much longer. 

We were all having a very sad, tough moment. So, I asked him, “What can we do for you? I know I can't get rid of your cancer now, but what can we do to make your day better?”

And he looked at me and said, “I need a piano.” 

So, I paused a moment and thought, “Okay, we're sitting in an ICU, but you need a piano, so…let’s do it.” This is the spirit of St. Jude; you are able to walk out of that room, look around at your colleagues and say, “Okay, we’ve got to find him a piano.”

A few hours later they were able to locate a piano and get it on wheels up to his room. That afternoon I was on phone call when I received a page that said that he was giving a concert in the ICU.  So, I raced up to that room. 

What a sight...there was my patient, sitting on the edge of that bed playing the piano. Outside his room there was an arc of people: doctors, nurses, respiratory therapists and different staff that were all standing there soaking up the music. As he finished playing his song everybody erupted in applause and cheer. When he looked up and saw me, he invited me into his room to hear his own rendition of “Amazing Grace.” That was a life-changing moment for me and one that reminded me of what makes St. Jude unique and so incredibly special. We never give up. We never gave up on this patient. He was fighting for his life, and we were in it with him. We don't give up at the end. We take care of the whole patient, not just the disease. 

While this is not the ending that we wanted for this patient, I know that it was important to him to know that some of those clinical trials he participated in, while battling his relapses, helped other patients and have been developed into new clinical trials. That was his legacy and the amazing grace that he extended to us all. 

These patients and these moments are my why. 

Q: What would you say to supporters who give to St. Jude and provide funding that fuels your research and patient care? 

A: When I think of St. Jude there are two sets of heroes that pop into my mind. Of course, first and foremost, are the patients and their families for having the courage to face what they go through every moment of every day. The second group of heroes, in my mind, are the St. Jude supporters because they give selflessly to advance cure rates and quality care for patients that they have never had the opportunity to meet. That is not lost on me, and I am so grateful for the supporter’s dedication to provide patients the opportunity to celebrate more birthdays and grow up. And so, St. Jude supporters are always second on my hero list; we just couldn't do it without them.

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