Noah’s Story: How a National Clinical Trial Gave One Mom an Extra Dose of Hope

When Maddie Harper’s toddler-aged son kept coming down sick, she and her pediatrics team chalked it up to daycare germs, a fact of life for many parents and children.

However, these seemingly simple illnesses were the start of a saga that would unexpectedly bring Maddie and her son, Noah Ludgood, face to face with leukemia. Their journey started with the pediatric oncology team at UR Medicine's Golisano Children’s Hospital, which works closely with the Wilmot Cancer Institute. Because of the expertise here, they also gained access to a clinical trial for a cutting-edge treatment before it was widely available, which might impact Noah’s survival.

“I'm just so grateful,” Maddie says. “The fact that they had the clinical trial available and the fact that we were even considered or able to have the option to decide if we wanted to do it is amazing because obviously it's not available everywhere.”

Noah's Journey

Childhood cancer is devastating but, thankfully, rare. About 15,000 children, from newborn to 19 years old, are diagnosed with cancer annually in the U.S., compared to about 2 million adults, according to the National Cancer Institute. At Golisano Children’s Hospital, about 80 to 100 new pediatric patients are diagnosed with cancer each year and about 10 to 20 of those have acute leukemia.

In February 2024, after weeks of fevers, congestion, and vomiting on and off, Maddie had taken Noah to the doctor’s office multiple times. The symptoms were thought to be caused by daycare virus exposures or perhaps a dairy allergy.

It wasn’t until Noah’s 18-month checkup that the pediatrician realized something was seriously wrong.

The doctor could feel Noah’s spleen was enlarged, and his lips looked pale. Blood work confirmed something was not right. Maddie immediately took Noah to the Emergency Department at Golisano Children’s Hospital/Strong Memorial Hospital. A few hours later, they learned Noah had leukemia. Specifically, he had a type called B-cell acute lymphocytic leukemia (B-cell ALL). He was admitted to Golisano for a stay that would last 28 days.

“Those first few days were a whirlwind,” Maddie recalls.

Not only did she need to be with Noah, but she was also 37 weeks pregnant. She delivered at Strong Memorial Hospital and then she and her new baby spent two weeks in the hospital with Noah as he received treatment.

Noah’s care team and her family’s support were critical.

“Spending a month in the hospital is not easy,” Maddie says. “Everybody that we've interacted on the medical side has been phenomenal. I don’t know how they do it. They make the best of the worst situation.”

Carol Fries, MD, assistant professor of Pediatrics, Division of Pediatric Hematology/Oncology, supervised the toddler’s care at the children’s hospital as Noah’s primary oncologist.

In this case, Noah was randomized to be in the “control” arm of the study, meaning he would get the standard treatment only and not the new immunotherapy drug. His mom was disappointed when she found out.

However, Noah wasn’t out of luck. During a scheduled interim review of the clinical trial while Noah was still in the early phases of treatment, experts saw that the results were so positive for the immunotherapy group that they closed the study early to make sure all eligible clinical trial participants could receive the new drug.

Noah got the immunotherapy after all.

“Based on compelling data that blinatumomab improves the chance of cure, it was appropriate to stop the trial immediately to make sure that every eligible child had access to blinatumomab going forward,” Fries said.

Maddie says she cried when Fries told her the news.

“The fact he was still going to get that and benefit from that, it was the best feeling. It was so unexpected,” she says.

The Clinical Trial

UR Medicine’s Golisano Children’s Hospital is part of the Children’s Oncology Group (COG). COG, funded by the National Cancer Institute, is a cooperative group of more than 200 institutions in the U.S. and internationally that treat childhood cancer and conduct clinical research.

Large randomized clinical trials are the best option available to identify effective new treatments for cancer, and a cooperative network like COG is necessary to make these large trials possible because most institutions don’t have enough patients to perform separate, individual trials.

Fries’ involvement in the ALL subcommittee for COG connects her with pediatric oncologists and leading researchers from all over the country and allows her to participate in groundbreaking clinical trials, like the one Noah was in, here at UR Medicine.

In this instance, after the study was closed early because of its success, the children in the control arm were able to quickly receive blinatumomab, which is approved by the U.S. Food and Drug Administration for leukemia. That made the next stage of treatment seamless for Noah.

“Not only is this medicine overwhelmingly effective, in many cases it is generally a lot better tolerated than many of our other, intensive chemotherapy medicines,” Fries says.

Being a part of the national COG network also made things easier for the teams administering the new treatment locally, Fries said.

“Because of the clinical trial and our institution’s familiarity with blinatumomab, our team was already trained and equipped. Our incredible inpatient and infusion center nurses were already prepared to seamlessly manage the blinatumomab infusions, and the pharmacy team was ready to rapidly scale the number of patients receiving this medicine,” Fries says. “I feel fortunate to be at a medical center where those resources were already invested, so there were no delays in making sure patients had immediate access.”

Looking Ahead

The results of the clinical trial were recently published in The New England Journal of Medicine, the world’s leading medical journal, and shared at national meetings.

Next steps include ensuring that all eligible patients who could benefit from blinatumomab have access to it and for scientists to learn how the success of this immunotherapy may help refine the other chemotherapy treatments to reduce side effects however possible.

“This kind of treatment revolution only happens once in a career, and it’s been inspiring and humbling to witness,” Fries says. “We seem to be in a new era when it comes to treating pediatric leukemia. It’s incredible to be at the dawn of my career at a time when our field is so full of hope. I feel so grateful – to the COG, to every institution that opened this trial, and to every patient and family who even considered enrollment.”