Cancer drug trial brings family from Sweden to Penn State Health Children’s Hospital
By Bill Landauer
Hannah and Fredrik Rosen were running out of options.
Their daughter, Ava, was in danger of reliving the same horror again and again. She had neuroblastoma, a form of childhood cancer with a cruel resilience. Surgery, radiation and chemotherapy could knock it down, but neuroblastoma often returns - often worse than before.
Ava’s most recent round of radiation, this one aimed at cancer cells in her spine, came at a steep price. Doctors told the family Ava likely would not grow as tall as she otherwise might.
Of all the awful effects Ava had been living with for months - sores, weakness, pain and vomiting in isolation - this was the first bargain her family had to make between treatment and her future.
“That was hard,” Hannah said, “but worth it.”
The cancer seemed to be gone. But what would it take next time? And how could they stop it?
By then, the result of an exhaustive search that took them all the way from their home in Alingsås, a small town just outside of Gothenburg, Sweden, to Hershey, Pennsylvania, they had found what they hoped was the answer to their prayers.
Dr. Giselle Saulnier Sholler, division chief of Pediatric Hematology and Oncology at Penn State Health Children’s Hospital and the director of pediatric oncology research at Penn State College of Medicine, has been waging a war on neuroblastoma for decades. And she may have found her best weapon yet ― a drug called eflornithine (DFMO).
Personal battle
Sholler first encountered the disease 22 years ago while working as a resident. The first patient she ever lost - a little boy named Tyler - died from it. Since then, she’s seen the illness shatter families over and over again.
Its metastatic qualities - how it can go away and reappear in different parts of the body - is among neuroblastoma’s most challenging characteristics, she said. Another is its aggressive nature. By the time it’s usually first diagnosed, the disease has already raged through the children sickened with it. Most are 3 to 5 years old, and by the time it’s clear anything is wrong, they’ve reached Stage 4.
Neuroblastoma often starts as an abdominal tumor, like the mass doctors found inside Ava in 2022, which by the time they first spotted it had grown to the size of an orange. The cancer can appear anywhere along the spinal column, and a neuroblastoma tumor is tougher to remove than other kinds of cancer, Sholler said.
“It’s sticky,” Sholler said. “It wraps around all the vessels and nerves or around the aorta.”
From there, it spreads quickly to bone marrow, lymph nodes and organs. And while radiation works well against it, it can hide in so many places that treatment can’t always reach it. “You can’t irradiate a whole body,” Sholler said.
During years of testing the drug in clinical trials, Sholler and her colleagues discovered DFMO can stop neuroblastoma from coming back. Sholler’s Beat Childhood Cancer Consortium earned U.S. Food and Drug Administration approval for DFMO in December 2023.
‘It was everywhere’
The Rosens first discovered Ava had neuroblastoma during an already terrifying time. Like families throughout the world, they’d all come down with COVID-19 in early 2022. The couple and Ava’s little sister, Hedda, recuperated quickly. But for Ava, who was 5 at the time, the illness lingered for weeks. She complained of bad tummy aches, which turned into vomiting. Doctors didn’t understand what was wrong. All they could tell the family was that her hemoglobin, the protein in the blood that carries oxygen, was low.
A doctor in Sweden found the tumor in May. “We don’t have a clue when neuroblastoma developed in her body,” Fredrik said. “We suspect it was quite some time ago.”
“It feels like it was really long ago now,” Hannah said. “It also feels like it was yesterday.”
It was the beginning of a dark odyssey. During the height of the pandemic, the family didn’t want to give their daughter, with her weakened immune system, COVID, so they kept isolated from one another. Chemo dried and cracked Ava’s skin. Blisters riddled her mouth and throat and made swallowing impossible. “Anything she ate came right back up again,” Hannah said.
Chemo shrank the tumor, but not enough. Doctors prescribed more rounds. The family counted each agonizing moment they had to live apart, only to find more days and weeks added each time.
Finally, doctors performed a surgical procedure to remove the tumor, but by then, the cancer had spread.
“It was everywhere,” Hannah said. “Her spine, her shoulders, her face.”
Beyond borders
They tried more chemo and radiation therapy. An internet search took them outside their own borders, and eventually a website in England gave them a link that brought them to Sholler and DFMO.
“We decided this was what Ava needed,” Hannah said.
Their own oncologist agreed. Soon they found themselves talking directly with Sholler.
“She asked, “Will you come and see me?’” Hannah said. “She made it very easy. It was very easy after that.”
Hannah and Fredrik hosted fundraisers, a Halloween party, an art show and a concert to bring in money to help pay the costs.
After more treatment in Sweden confirmed Ava’s spine was free of cancer cells, the family flew to Hershey. They stayed at the local Ronald McDonald House and met with Sholler for the first time.
“She’s very professional and very caring,” Hannah said. “She makes you feel very safe as a parent, leaving your child in her hands.”
Ava and her family will make five trips in all to Pennsylvania to retrieve more DFMO tablets - she takes them twice a day - and for evaluation. In between, her medical team in Sweden will test her and keep Sholler updated of her progress.
After that, Sholler will get her updates from abroad, where she hopes to still have a view of Ava performing a miracle that everyone once feared would never happen.
In photos and in test results, Sholler will watch Ava grow up.
Their best life
So far, the cancer hasn’t returned. Ava is now 7 years old. She’s found new energy - she plays soccer every Sunday, swims every Saturday, goes to school and plays with friends in between.
Ava sometimes still gets sick. She has runny noses and colds. But the bad days locked in a room, weakened by her fight with an illness that won’t go away - those times haven’t returned.
“That’s really the goal for this medication,” Sholler said. “It’s so they can live their best life.”
