Ten-year-old Matilda Loomis strapped on a small blue backpack in mid-December, and she won’t take it off until mid-January.

The denim pack, decorated with a multicolored patch of a pretty horse, goes with her everywhere, even into the bathroom and, at night, into her bed, where she sleeps with it just inches away. When she goes to school at Eagle Hill Middle School in Manlius, she juggles it with her school backpack, her lunch pail, and, on some days, her saxophone.

Though it can get annoying sometimes, most days she doesn’t mind it. That’s because she knows that what’s inside of it is crucial to her recovery from B-cell Acute Lymphocyte Leukemia (ALL), the most common cancer among children. A clear thin tube runs from inside the backpack into a port in Matilda’s chest, delivering blinatumomab, also known as blina, a monoclonal antibody, 24 hours a day, seven days a week for 28 days.

The blina inside Matilda’s backpack and the timing of it in her treatment is being called the biggest advancement in children’s cancer treatment in a decade by doctors, researchers and cancer agencies.

Matilda receives the blina treatment at the Upstate Cancer Center through a Phase 3 global study conducted by the Children’s Oncology Group, the world’s largest organization devoted to childhood and adolescent cancer research. Upstate participated in the study as member of the COG and Matilda is the last of 25 children enrolled through Upstate’s Golisano Children’s Hospital.

Matilda is the last enrolled patient on the study at Upstate to receive the experimental treatment.  Golisano Division Chief of Pediatric Hematology/Oncology Melanie Comito, MD, said going forward, blinatumomab will be covered by insurance and offered to all eligible patients.

The clinical trial showed that adding blina to initial chemotherapy treatment significantly improves disease-free survival rates and lowers toxicity levels in newly diagnosed pediatric patients with standard-risk B-cell acute lymphoblastic leukemia with higher-risk features.

Most children with ALL are cured, however, certain subsets have a higher risk of relapse. It is those patients who will benefit from this additional treatment.

'They thought this study could be a game changer one day and it is'

The study found that the three-year disease-free rate was 96 percent for high-risk patients treated with chemotherapy plus Blinatumomab, compared to 87.9% for those treated with only chemotherapy.

Comito said she has been treating pediatric cancer for 30 years and can’t think of another study that showed such a substantial improvement.

“They thought this study could be a game changer one day and it is,” Comito said. “This will impact how we treat children with ALL going forward. We want to cure every child with the least amount of side effects. We want every child to look in the mirror as they grow up and nobody will ever know they were treated for cancer.”

B-cell ALL is a blood and bone marrow cancer that occurs when too many stem cells develop into immature white blood cells instead of mature blood cells. Symptoms leading to diagnosis include fatigue, fever, easy bruising or bleeding, bone and joint pain and shortness of breath.

Despite a high overall cure rate for B-ALL, relapsed B-cell ALL still results in many cases of childhood cancer.

Treatment for children with acute lymphoblastic leukemia (ALL) consists of induction, consolidation, intensification and maintenance phases and usually lasts for over 2 years. The first several months are more intense followed by maintenance therapy which is mostly oral medications.  This approach has resulted in overall high cure rates for B-ALL.  Despite these high overall cure rates, Comito said that relapsed B-ALL still results in too many cases of childhood cancer, which has led to the search for new treatments to add to the therapy

This COG study for standard-risk B-ALL patients started in 2019.  About half of these patients will have favorable features and extremely high cure rates with conventional therapy alone.  For those standard-risk patients with higher-risk features, they were randomly assigned to receive chemotherapy alone or chemotherapy plus two nonsequential 28-day cycles of blinatumomab during the second phase of treatment. The results were so good, the study stopped enrolling new participants early, and the results were published in December in the New England Journal of Medicine.

Comito said blina was previously used in ALL patients who had relapsed, for which it was approved by the FDA.

Once the study showed such promising results for these newly diagnosed standard-risk patients with high-risk features, it received FDA approval for that usage and became available to all study participants and then the general public.  It is now part of treatment in newly diagnosed patients with high-risk B-ALL.

More than 4,000 patients nationwide enrolled in the randomized trial. Comito said Upstate was able to enroll its 25 patients thanks to funding from the COG, the St. Baldrick’s Foundation and Paige’s Butterfly Run Foundation.

Comito said it takes a significant amount of infrastructure, including two full-time clinical research assistants to participate in such a trial, and meeting those needs is challenging at a small institution like Upstate, which relies solely on outside funding for pediatric cancer trials.

She said funding from the COG helps pay salaries but isn’t enough to cover two people. The rest of Upstate’s salary money was funded by an annual infrastructure grant from St. Baldrick’s. Comito pointed out that the Syracuse area has traditionally been one of the top fundraising areas nationally for St. Baldrick’s, highlighted by the annual head-shaving event at Kitty Hoynes.

“Without the St. Baldrick’s grant, we would likely only have one clinical research assistant which then makes it difficult to participate in such a cumbersome trial,” said Comito, the Paige Yeomans Arnold Endowed Professor in Pediatric Oncology.

In addition to paying salaries, Upstate had to purchase the materials for the study, including the backpacks, pumps, battery packs and tubing. Those materials, along with gas money for participants, were funded by annual donations from Paige’s Butterfly Run, a local foundation that has raised over $5 million devoted to fund local research and help local families. Paige’s main event is an annual 5k run in June.

The study enrolled 4,264 newly diagnosed standard-risk B-ALL patients, of whom 2,334 were risk stratified at the end of induction therapy as either average standard risk or high standard risk. From there, 1,440 of the eligible and evaluable patients were randomized, with about half getting the drug and half not.

'It is nightmare to hear that you child has cancer'

Matilda enrolled in the study after her diagnosis in November 2023 but was not randomly selected to receive blina. Once the study showed the superiority of blina, she was offered the study drug as she was still early enough in her treatment. She was able to start her first round of it in August 2024 as it aligned with the correct timing of her treatment phases.

Matilda’s father Chad said the family’s initial disappointment over not getting the drug in the random coin flip didn’t last long, as the timing of it becoming available worked perfectly for her. Study participants stay overnight in the hospital when the infusion starts and then come in twice a week to change out the blina bag in their backpacks.

In early November 2023, Matilda started exhibiting symptoms, such as fever, lethargy and loss of appetite, but most alarmingly, struggled to walk. She was being treated for strep throat when bloodwork results sent the family immediately to the emergency room at Upstate. Four days after her first symptoms appeared, she was diagnosed with leukemia.

“It is a nightmare to hear that your child has cancer,” said Chad Loomis, Matilda’s dad. “Matilda is the kindest person I know. She is so selfless; she checks in on my wife, Lisa, and I to see how we’re doing. To know that she’s got that much of a better chance not only now but in the future is something that we are so thankful for. We are so blessed to be here and to be part of this, to be able to improve her chances of quality of life that much, as well as the quality of life of all kids with this disease.”

Matilda started 5^th grade at Eagle Hill Middle School in the fall in the middle of her treatment and when she had no hair. She said some kids looked at her funny when she was bald, and then thought she was a boy when her hair started growing in, but she mostly doesn’t care. With her hair just coming back in this fall, she was the spitting image of Eleven, the main character from Stranger Things, who she dressed as for Halloween. She loves art, reading and Disney World.

Her treatment is scheduled to end in March 2026 after completing the maintenance phase. She only learned a few days ago about being part of a ground-breaking study that will improve the lives of kids with ALL everywhere.

“I think it’s really cool and amazing,” she said, “and I feel really thankful to be part of this.”

 

Caption: Matilda Loomis and her care team at the Upstate Cancer Center. Loomis is seated at center with her drug-carrying backpack. Also seated are Gloria Kennedy, MD, left, and Melanie Comito, MD. Standing from left are Gail Nolan, RN; clinical research associates Karen Bylinski, RN, and Suzanne Majewski and child life specialist Beth Kinsley.