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Spotlight: Research helps Heather Stefanski, MD, PhD, develop better treatments for her blood and marrow transplant patients

Pediatric Oncologist Heather Stefanski, MD, PhD, harnesses the power of research and innovation to treat young patients with cancer or rare diseases.
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Pediatric Oncologist Heather Stefanski, MD, has always wanted to help young cancer patients.

“A lot of my friends struggled with what they wanted to do after medical school and where they would be happiest,” she said. “I never struggled with that. I’ve always know that this field is exactly where I want to be.”

For Stefanski, that place is the pediatric blood and marrow transplant program at University of Minnesota Masonic Children’s Hospital. Blood and marrow transplants (BMTs) are used to treat patients with certain cancers or other rare, devastating diseases. During the transplant, a person’s abnormal or defective stem cells are replaced with new donor cells that work properly.

This year, the University of Minnesota celebrates the 50th anniversary of the world’s first successful matched, related donor bone marrow transplant—which was performed in 1968 at University of Minnesota Medical Center by Robert Good, MD.

We caught up with Stefanski to talk about her passion for BMT care, the leading-edge therapies she’s researching and the future of our blood and marrow transplant program.

How did you get interested in pediatric oncology?

I knew in medical school that I wanted to be a pediatric oncologist. Later, in residency, I worked in the pediatric BMT field and I really loved it. I enjoyed forming relationships with the families I met. BMTs can be a life-changing treatment or cure for children with cancers or rare diseases, and I enjoy being part of that effort.

I was fortunate enough to have trained at the University of Minnesota, a place with an amazing, 50-year BMT legacy. We have one of the largest and most experienced transplant centers in the country. I didn’t need to go anywhere to receive excellent training and to discover my own niche in medicine.

Tell us about your day-to-day work.

I am a BMT physician, and I administer the transplant procedure to help treat young patients with leukemia or solid tumors. I also conduct research to advance our expertise. My research is focused upon decreasing infection and relapse rates following a BMT transplant so that our patients experience better outcomes. Right now, one of our efforts revolves around CAR T-Cell therapies—a new type of treatment that recently received FDA approval. I translate our research from the ‘bench to the bedside.’ In other words, I use the latest discoveries to develop better treatments for our patients.

Learn more about CAR T-cell therapy treatment offered at University of Minnesota Health.

What is CAR T-Cell therapy and why is it so important?

CAR T-cell therapy uses a person’s own cells to fight cancer. T-cells, a vital part of a person’s immune system, are extracted and then genetically modified to produce chimeric antigen receptors, or CARs, that are specific to a patient’s cancer. When the modified T-cells are reintroduced into the body, they seek out and destroy those cancerous cells.

Leukemia and other cancers are very good at evading a patient’s normal immune system—the immune system either can’t detect them or doesn’t see them as a threat. This therapy equips a patient’s immune system with the ability to attack the cancer.

How does that play into your work?
My primary research focus is ‘T progenitors,’ which are very young T-cells. During a BMT, we essentially replace a patient’s immune system. Following a transplant, it takes time for a patient’s new immune system to rebound. T-cell immunity is very critical for the body when fighting fungi, viruses and tumors. We hope an infusion of T progenitors can help “jump start” an immune system after a BMT. If we can give the immune system an early boost, then perhaps we can reduce the risk of infections and relapse after transplant. Currently, there’s a 20 to 30 percent chance of the disease returning following a BMT.

Where do you see the BMT program going in the future?

I think we’re going to harness the power of a patient’s immune system and develop more immunotherapies and gene therapies. Eventually, these new therapies may make BMTs obsolete.

BMTs are life-saving procedures, but they don’t come without complications. In the years following a transplant, patients face a long list of side effects. Through research, I truly believe we can improve the safety of a BMT and, someday, find a more advanced and effective replacement for it.


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