PITTSBURGH (TNS) — Every morning and night, 7-year old Ford Bartle drinks a smoothie with nine pills crushed up inside.

Those pills — newly approved by the Food and Drug Administration after decades of research by a Penn State professor — may be the key to helping Ford and thousands of kids like him prevent the relapse of a particularly aggressive childhood cancer called neuroblastoma.

“We don’t know what his outcome will be, but we have a lot of hope for him,” said Giselle Saulnier Sholler, division chief for pediatric hematology/oncology at Penn State Health Children’s Hospital. “This year has given us a lot of hope.”

Sholler’s work on neuroblastoma started 20 years ago, when she was a fellow at Brown University just starting her pediatric oncology rotation. Neuroblastoma is a fairly rare childhood cancer affecting the peripheral nervous system, with about 800 children in the U.S. diagnosed per year.

Despite its rarity, Sholler’s first three patients that year at Brown happened to have neuroblastoma. Doctors can treat neuroblastoma pretty well initially, sending patients into remission, but about half of those patients relapse, and those relapses are more resistant to treatment. As a result, back in 2004, neuroblastoma only had a 30% survival rate. Two of Sholler’s first three patients with the disease died, devastating the new physician.

Sholler couldn’t help but compare those patients’ outcomes to the many children she saw surviving leukemia, a much more common childhood cancer. In the span of just a few decades, advances in research had improved 5-year acute lymphoblastic leukemia survival rates from less than 10% in the 1960s to greater than 50% in the late 1970s to about 90% by 2004.

Sholler wanted to see if research could make a similar impact on neuroblastoma.

“Having seen the impact that research can have, particularly clinical research, I decided then that that was what I wanted to do,” she said.

She began to do lab research while still treating patients, and her first breakthrough came from one of those patients: a girl with neuroblastoma who had contracted Chagas disease, an infection caused by a South American parasite, through a blood transfusion. As Sholler treated her with an antiparasitic drug, her neuroblastoma got better.

Sholler began to research the drug’s effect on neuroblastoma and, after she presented her research at a conference, heard from three parents of children with neuroblastoma asking if they too could try the drug. When the FDA required a clinical trial to give the drug to more children, one of those parents gave Sholler $100,000 to fund one.

As she continued to research, looking into genetic pathways specific to neuroblastoma, she thought that there may be even more promise in another antiparasitic drug, this one called DFMO and used to treat African sleeping sickness.

With funding, again, from parents of children with neuroblastoma, she launched clinical trials of DFMO. For more than a decade, she tested and refined her approach, eventually producing impressive results: Used as a maintenance therapy, DFMO decreases the rate of neuroblastoma relapse by about 50%.

Sholler was well into her DFMO research in the summer of 2019 when Ford Bartle’s parents noticed that he seemed a little off during a family vacation to North Carolina’s Outer Banks.

“He was two and a half at the time and he was just not acting himself,” said his mom, Carly Bartle. “We thought that it was probably because we’re on vacation, all our family was there, and it was a lot of commotion.”

But when the family returned home to Middlesex, Butler County, Ford was still tired and fussy, with occasional fevers and headaches, and worsening constipation. The family followed up with their pediatrician, and brought Ford to the UPMC Children’s Hospital of Pittsburgh emergency room on a Sunday night in August when the constipation became severe.

The hospital ran bloodwork and recommended that Ford be admitted to the oncology department.

“It was just all of a sudden, anything that we thought could have been our worst nightmare was coming true,” said Carly Bartle.

By that Friday, Ford had an official diagnosis of neuroblastoma. By the following Tuesday, he and his parents were in New York City, being seen by specialists at Memorial Sloan Kettering Cancer Center. Ford had a tumor in abdomen that was causing his stomach problems, in addition to other tumors throughout his body. His parents were told that doctors use a “Curie score” to measure the prevalence of tumors in a neuroblastoma patient. The score has a maximum of 30, and Ford measured a 29.

With doctors at both UPMC and Sloan Kettering, Ford began aggressive treatment. Five rounds of chemotherapy, a nine-hour surgery and radiation brought his Curie score down to two. Multiple cycles of immunotherapy got the score down further and, in March of 2020, the Bartles were told that his score was down to zero.

“We were super excited,” said his father, Bobby Bartle. “He was considered to have no evidence of disease,” added Carly.

Ford was thriving in preschool when, about a year later, a scan found a small cancerous spot on his head in February 2021. It was treated with radiation but in October of that year, more spots appeared in his brain, which cleared with more intense radiation.

But in August 2023, another spot appeared. Ford had already tried so many treatments, and his parents wondered if they were no longer working. They had heard from other parents about the clinical trials that Sholler was running of DFMO, and its promise in preventing relapses.

And then, the next month, Sholler joined Penn State Health.

They spoke to Sholler on the phone nearly immediately. In October, they had their first meeting to officially enroll Ford in a DFMO trial.

Just two months later, in December 2023, the FDA announced that it had approved DFMO to reduce the risk of neuroblastoma relapse. The medication is designed to be used for two years as a maintenance drug after patients enter remission after their initial treatment.

“In the U.S., it’s now the standard of care,” Sholler said. “At the end of upfront treatment, every child should get DFMO.”

In the last few months, Australia and the United Kingdom have approved the drug as well. Sholler has patients coming to Penn State Health from nearly 30 counties where it is not yet approved to enroll in clinical trials here to get the drug.

The drug is officially named iwilfin, after two of her patients named Will and Finn, who participated in DFMO trials and are still cancer free.

The drug’s development was funded almost entirely by money raised by the parents of patients with neuroblastoma, through a fund called Beat Childhood Cancer. Money raised by the THON dance marathon at Penn State is also now funding more research by Sholler and her colleagues.

Since Ford began taking the drug in October 2023, he has had no relapses — an encouraging sign that DFMO is working given his three relapses in the years prior. “Usually it’s shorter and shorter between relapses, so this has been longer than expected,” Sholler said. “To see him looking wonderful and in remission is exciting.”

In the 20 years since Sholler began her quest to improve survival rates for neuroblastoma, they have indeed risen substantially — thanks to DFMO and other treatments such as the antibody immunotherapy that Ford received. The overall survival rate from the start of treatment is now about 70%, she said, and around 85% for patients who make it through their initial course of treatment.

With every visit to Penn State Health, Ford opens up more of his personality to Sholler. When a photographer took pictures of his visit this August, the two posed together showing off their muscles. The photo became a billboard that launched recently around Hershey.

Next to the photo, a caption reads, “We won’t stop until every child is cured.”