One of the most common cancers in children, leukemia, may have met its match. Scientists at Roswell Park Comprehensive Cancer Center are at the forefront of a personalized cancer treatment called CAR T-cell therapy, which transforms a patient’s own cells into cancer-fighting supercells, often without invasive surgery, offering new hope for a long, healthy life.
Because Bryn’s cancer cells harbored a rare and aggressive mutation, TCF3-HLH, standard chemotherapies and surgeries proved ineffective.
On April 29, 2024, Bryn Ailinger was diagnosed with precursor B-cell acute lymphoblastic leukemia (B-ALL), the most common childhood cancer.
Because her cancer cells harbored a rare and aggressive mutation, TCF3-HLH, standard chemotherapies and surgeries proved ineffective.
TCF3-HLF-positive leukemia represents a rare subtype of B-ALL, characterized by a high treatment failure rate despite intensive treatment and hematopoietic stem cell transplantation (HSCT).[1]*
“You think the worst immediately,” noted Justin Ailinger, Bryn’s father.
“I didn’t know if I was going to have a daughter by the end of the year,” Allinger added.
CAR T-cell therapy is a real option
Bryn’s care team from the Roswell Park Oishei Children’s Pediatric Cancer and Blood Disorders Program then presented CAR T-cell therapy as an option.
“I explained to them that CAR T-cells were her cells, but now armed to go after the leukemia cells,” said Meghan Higman, MD, Ph.D, a pediatric oncologist at Roswell Park specializing in blood cancers.
The team at Roswell Park is a leader in the treatment and development of CAR T-cell therapies. “
“You have the clinicians and the scientists working side by side,” Higman noted.
“Chemotherapy is so toxic,” stressed Ajay Gupta, MD, MS, a pediatric oncologist at Roswell Park specializing in sarcomas. “CAR T-cell therapy is safer than chemotherapy,” Gupta added.
“This is especially important to think about late effects and survivorship in your youngest patients,” he further said.
After a week of treatment… “I look at her, and I see a miracle,” Higman observed.
“That’s because this kid five years ago wouldn’t have been alive, and now she’s alive, thriving, and just, wow!” she added.
Higman and her Roswell Park colleagues recommend CAR T-cell therapy even for their youngest patients who can benefit — not only for its efficacy but also its safety compared to chemo.
“We’re trying to make it so that patients have a better quality of life even after they’re done with treatment. And so I feel strongly that approaches that can change the immune system, like CAR T-cells, can actually do this without causing long-term side effects,” Gupta said.
What is CAR T-cell Therapy
CAR T-cell therapy is a type of immunotherapy that uses a patient’s own T-cells to fight cancer. In this process, T-cells are extracted from the blood and sent to a laboratory where they are genetically modified to produce ‘chimeric antigen receptors (CAR) on their surface. After this process is completed, the ‘new’ CAR T-cells, which are now able to recognize and attack cancer cells, multiply and are then returned to patients via IV infusion. [1]
CAR T-cell therapy is a complex, personalized treatment used to treat certain blood cancers that have not responded to or become resistant to other therapies, such as some types of leukemia and lymphoma.[1]
The cells are genetically modified at Roswell Park’s newly expanded Good Manufacturing Practice Engineering & Cell Manufacturing Facility (GEM), one of the most extensive facilities of its kind in the United States. It contains 20 sterile rooms across two buildings, marking a transformative step for cancer research and treatment.
Pediatric Patients
The first pediatric patient treated with a CAR T-cell therapy was Emily Whitehead, who, at five years old, was diagnosed with ALL. After a long treatment, her cancer went into complete remission, and in June 2012, at age 7, Emily was discharged from the hospital. Today, she is thriving and considered cured. [2][3]
Today, Bryn, the six-year-old, is in remission and ready for first grade.
“I’m hoping that her new immune system is still searching, that the CAR Ts and her further therapy is still searching for this mutation and wipes out every single cell. We usually say after five years of being in remission, you are cured of your leukemia,” Higman noted.
Other Children
“I hope for other children that have this mutation pop up that they’re able to collaborate with Higman and the others, and try these same therapies that were successful for Bryn,” Ailinger says.
“I hope that she’s able to help save lives for other children as well who go through this,” Higman said.
The results of CAR T-cell therapy so far have been auspicious, with acute leukemia remaining undetectable in more than 80% of patients after treatment. As of now, CAR T-cell therapy is approved only in the U.S. Food and Drug Administration (FDA) for certain types of blood cancer, but scientists at Roswell Park are also working to expand the use of the treatment against other cancers, of rare kinds like sarcoma, to the second most common cancer in kids, brain tumors.[4]
Solid tumors
“Our next job is how can we get them into more solid tumors or into places that it’s hard to get to because the immune system doesn’t really get there as well as it should,” said Higman, who also holds appointments with the Jacobs School of Medicine and Biomedical Sciences at the University at Buffalo and UBMD Physicians Group.
“Like the brain, chemo often doesn’t get there because of the way that the blood-brain barrier works. So what we really need to do is build more things to keep it around for everybody.”
Future development
“We want to see those therapies not only bring on more cures but also more durable cures. And so there’s still work to be done in the liquid space, but to bring that even in a fraction of that to the solid tumor space would be life-changing for those patients who currently don’t have any cellular therapies available to them, explained Gupta, discussing the future of CAR T-cell therapy
“The efforts that we’re putting forth in the lab are going to give rise to a whole host of CAR T-cell products. And so each of those will be an opportunity for a different group of patients. And so the whole enterprise is extremely, extremely exciting.” Guta concluded.
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Note: * The chimeric transcription factor TCF3-HLF remains an aggressive acute lymphoblastic leukemia (ALL) subtype. However, CAR T-cells can effectively treat relapsed/refractory TCF3-HLF-positive childhood B-ALL with acceptable toxicity, which could be a new treatment option for this subtype compared with chemotherapy or HSCT.
Reference
[1] CAR T-cell Therapy and Its Side Effects. American Cancer Society. Online. Last accessed on November 11, 2025.
[2]Awasthi R, Maier HJ, Zhang J, Lim S. Kymriah® (tisagenlecleucel) – An overview of the clinical development journey of the first approved CAR-T therapy. Hum Vaccin Immunother. 2023 Dec 31;19(1):2210046. doi: 10.1080/21645515.2023.2210046. Epub 2023 May 15. PMID: 37185251; PMCID: PMC10294746.
[3] Bouzianas D, Bouziana S. First pediatric B-acute lymphoblastic leukemia patient treated with anti-CD19 chimeric antigen receptor T-cell therapy: Long-term remission or early cure? Hum Vaccin Immunother. 2024 Dec 31;20(1):2321678. doi: 10.1080/21645515.2024.2321678. Epub 2024 Feb 25. PMID: 38402637; PMCID: PMC10898498.
[4] Patel KK, Tariveranmoshabad M, Kadu S, Shobaki N, June C. From concept to cure: The evolution of CAR-T cell therapy. Mol Ther. 2025 May 7;33(5):2123-2140. doi: 10.1016/j.ymthe.2025.03.005. Epub 2025 Mar 10. PMID: 40070120; PMCID: PMC12126787.
Featured image: Bryn Ailinger takes a nap during her treatment through the Roswell Park Oishei Children’s Cancer and Blood Disorders Program—Photo courtesy © 2025 Roswell Park Comprehensive Cancer Center. Used with permission.
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