A clinical trial gives Scarlett hope

Scarlett was diagnosed with acute lymphoblastic leukemia (ALL) at just 2½ years old. While ALL is the most common form of childhood cancer and highly treatable, the type Scarlett has — Philadelphia chromosome B cell ALL — is a rare genetic mutation. Even with the most aggressive treatment, it was likely Scarlett’s cancer would return. And it did.

The pediatric cancer specialists at UW Health Kids American Family Children’s Hospital are part of a national research team that combines two scientific fields — genomics and immunotherapy — in search of cures for the most hard-to-treat cancers. Scarlett is now enrolled in a Phase One clinical trial for a promising new treatment that is offered only at the University of Wisconsin. For her family and care team, it represents real hope for a cure.

The only telltale sign of Scarlett’s nearly seven-year journey at American Family Children’s Hospital is her bald head — which she covers with a bedazzled cap accented with sparkly earrings that match her personality. She’s outgoing, a 9-year-old dynamo who charms everyone and walks through the hospital with a big smile and unexpected ease despite everything she’s experienced in her young life.

The beginning of Scarlett’s long relationship with her care team

For the first year and a half, Scarlett underwent intensive treatment — most of which involved long inpatient stays. It was difficult, but Scarlett’s mom, Tami, says the nurses and Child Life specialists make the hospital feel like home.

“They cater to children here, and Scarlett loved everything about it. There were three days she wasn’t talking because of mouth sores, but she was still up playing,” Tami said.

The nurses hung pictures of fires around the unit and Scarlett drove around in a little car, squirting syringes filled with water to put them out. Child Life specialists and facility dog Kiko have supported Scarlett through countless intravenous (IV) and nasal tube placements and needle “pokes.”

Tami and her husband Jordan also can’t say enough about how much support they received as parents.

“Sometimes the nurses would give me a hug,” Tami said. “They’d tell me something frustrating or sad. I’d say, ‘It’s OK,’ and the nurse would say 'No, it’s not.’ As parents, the nurses were just as nice to us. We needed them for sure.”

Following the long hospital stay, Scarlett had a relatively calm three years of oral chemotherapy, routine labs and bone marrow checks. But in November 2022, Scarlett’s cancer came back, and she would need a stem cell transplant to keep the cancer at bay.

“We’d cry — some of us more than others,” Tami said, “but we’ve always been optimistic. If this doesn’t work, there are so many backup plans.”

A new combination provides hope

It was not possible to find a perfect donor match, but Scarlett was the perfect candidate for a clinical trial led by Dr. Christian Capitini, a UW Health pediatric oncologist. Scarlett would receive a stem cell transplant followed by five doses of the drug Zoledronate every 28 days.

The first step was for Scarlett to undergo two weeks of “conditioning” — chemotherapy and radiation to rid her bone marrow of white blood cells in preparation for the transplant. Scarlett’s father was her stem-cell donor, but his blood required special treatment to minimize the chance his cells would reject being in Scarlett’s body (also known as graft-versus-host disease).

Scarlett had her stem cell transplant on April 7, 2023. She remained in the hospital another three weeks following the transplant, returned to the hospital weekly as an outpatient for check-ups for three months, and now comes to the hospital every other week.

The road trip is worth it

Scarlett’s clinical trial is in phase one of three, and it will be years before the treatment could be approved by the U.S. Food and Drug Administration as standard treatment, but Dr. Capitini is thrilled with how Scarlett has responded.

“After two months, Scarlett was in remission not only in terms of detectable leukemia, but she also showed no detectable Philadelphia chromosome," he said. "The medicines she had been taking before weren’t even able to do that."

Clinical nurse specialist Ryan Brown, BSN, coordinates Scarlett’s care, which also includes social work, psychology, pain and palliative care, nurses, phlebotomists and medical assistants.

“Clinical trials are a real collaboration between the family, the care team and the research team,” he said. “We are very singularly focused on her quality of life and will be following her for a very long time.”

Scarlett and her parents have driven 2 hours each way from Wisconsin Rapids for the nearly seven years she’s been in our care. Scarlett could have received care closer to home, but as Tami said: “We just gravitated to Madison and never thought about going anywhere else.”