Children's Cancer Care in China

pCAR-19B (Pulidekai®)

pCAR-19B is the world's first CD19-targeted CAR-T product using a fully humanized single-chain variable fragment (scFv) design, indicated exclusively for children and adolescents aged 3–21 with relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL). In Phase II data across 64 pediatric patients, it achieved a 90.63% complete remission rate and a minimal-residual-disease-negative rate above 90% — profound tumor clearance in the vast majority of treated children. As an original Chinese innovation, pCAR-19B has not yet been approved in Europe or the United States, making China the only place a child can currently receive it.

References [R2][R11]

Zurletrectinib (ICP-723)

Independently developed in China, zurletrectinib is a second-generation TRK inhibitor covering all NTRK1/2/3 fusion mutations, designed to overcome the acquired resistance that limits first-generation TRK inhibitors in rare pediatric and adolescent solid tumors. Registration data showed an overall objective response rate of 89.1% and a disease-control rate of 96.4%, with a 100% response rate observed in adolescent patients. It is approved for patients aged 12 and above with NTRK-fusion-positive solid tumors and is available across numerous tertiary hospitals in China.

References [R12]

Anlotinib

Anlotinib is a wholly China-developed oral multi-target tyrosine kinase inhibitor from Chia Tai Tianqing Pharmaceutical, indicated for refractory pediatric sarcoma in children aged 5–18 with recurrent or refractory solid tumors. It precisely suppresses VEGFR, FGFR, and PDGFR — core targets driving tumor angiogenesis and proliferation. The pivotal Phase I trial confirmed favorable safety and tolerability, with most adverse events grade 1–2 and clinically manageable, supporting long-term pediatric use. It is one of the few wholly domestic multi-target TKIs with established pediatric clinical evidence — reflecting China's growing capacity to develop oncology therapies tailored to pediatric populations underserved by global trials.

References [R13]

Selumetinib (Koselugo®)

Selumetinib (Koselugo) is the world's first oral MEK inhibitor approved for pediatric neurofibromatosis type 1 (NF1), developed by AstraZeneca/Merck for children aged 3 and above with symptomatic, inoperable plexiform neurofibromas — tumors that commonly cause deformity, chronic pain, and functional impairment. It received FDA approval in 2020 and NMPA approval in China in 2023. Before selumetinib, surgery was the only intervention and rarely achieved complete resection. As an ultra-rare-disease orphan therapy, its list price reaches approximately $268,678 per patient per year in Canada (US pricing comparable or higher). In China it is available through specialized pediatric oncology centers at treatment costs substantially below US/Canada list prices, though pricing varies by hospital pathway and weight-based dosing.

References [R14]

GD2-Targeted Immunotherapy (Dinutuximab Beta / Naxitamab)

Anti-GD2 monoclonal antibodies (dinutuximab, dinutuximab beta, naxitamab) are foreign-developed immunotherapies for high-risk pediatric neuroblastoma — a leading cause of childhood cancer death. By targeting the GD2 ganglioside on neuroblastoma cells, they improve long-term survival from roughly 40% to 60%. A peer-reviewed 2026 cost analysis documented full-course pricing of approximately $192,750 (dinutuximab, US), $142,695 (dinutuximab beta, EU), and $610,800 (naxitamab, US), and found anti-GD2 therapy available at 93% of high-income-country centers but only 21% of low/middle-income-country centers. Two dedicated GD2 antibodies (dinutuximab beta and naxitamab) are approved in China; domestic data show first-line maintenance with dinutuximab beta reaching a 2-year event-free survival of 80.1% and a 2-year overall survival of 97.6% in newly diagnosed high-risk neuroblastoma. For families with no domestic access, China offers a high-quality, more affordable destination.

References [R24-R25]

The Beijing Protocol: half-matched stem cell transplant that eliminates the donor-matching wait.

Conventional bone marrow transplantation requires a fully matched donor, a search that can take months while a critically ill child waits. The Beijing Protocol — a haploidentical (“half-matched”) HSCT regimen developed by Prof. Huang Xiaojun at the Peking University Institute of Hematology, using GCSF-primed marrow plus post-transplant immunosuppression — makes a parent or sibling an immediate eligible donor while achieving survival comparable to fully matched transplants. It has been adopted internationally and is one of the most influential HSCT innovations to come from China. Indications include pediatric acute lymphoblastic leukemia, acute myeloid leukemia, severe aplastic anemia, thalassemia, and inherited immunodeficiency disorders. Shanghai Children's Medical Center maintains 32 Class-100 sterile transplant wards and completes over 300 pediatric HSCT procedures annually, ranking first in national pediatric transplant volume.

References [R4]

The world's largest pediatric liver transplant program — anchored at Renji Hospital, Shanghai.

Pediatric liver transplantation is the standard rescue treatment for hepatoblastoma that cannot be completely resected, as well as for biliary atresia, metabolic liver diseases (Wilson's disease, tyrosinemia), and acute liver failure. Renji Hospital, Shanghai Jiao Tong University, is documented in the medical literature as the world's largest pediatric liver transplantation program. National outcomes from the Chinese Liver Transplant Registry report 1-year graft survival of 84.7% and 5-year graft survival of 72.6% for biliary atresia transplants (509 cases), and Renji's published hepatoblastoma series shows favorable long-term outcomes in carefully selected patients. The Chinese Children's Cancer Group has developed the nationwide Wuhan Protocol for hepatoblastoma multidisciplinary management. International families gain access to China's combination of very high case volume and integrated pediatric oncology coordination.

References [R31]

Comprehensive pediatric CAR-T programs — anchored by the China-originated pCAR-19B.

CAR-T cell therapy for children spans the full clinical pathway: peripheral blood mononuclear cell collection, T-cell genetic modification, in vitro expansion and quality inspection, chemotherapy lymphodepletion, cell infusion, and long-term monitoring. The primary pediatric indication is relapsed or refractory B-cell acute lymphoblastic leukemia in children and adolescents aged 3–21, with the China-originated pCAR-19B (Pulidekai) the only fully humanized CAR-T product designed specifically for this population. Leading centers — Beijing Children's Hospital, Shanghai Children's Medical Center, and West China Hospital — have built standardized pediatric CAR-T systems covering patient screening, cell preparation, toxicity management (CRS, ICANS), and long-term immune monitoring.

References [R15][R18]

3D-printed implants designed for growing bones — limb salvage in pediatric bone cancer.

Limb-salvage surgery is now standard for pediatric osteosarcoma and Ewing sarcoma around the knee, distal femur, proximal tibia, and shoulder — replacing amputation. The challenge in children is the growing skeleton: traditional adult endoprostheses cannot accommodate ongoing growth, leading to limb-length discrepancy. China is among the leading countries integrating clinical-scale 3D printing into pediatric orthopedic oncology, with customized extendable prostheses, joint-preserving prostheses that spare the epiphyseal growth plate, and patient-specific surgical guides now in clinical use. The Musculoskeletal Tumor Center at Peking University People's Hospital reports one of the largest pediatric series (89 children/adolescents with osteosarcoma around the knee), and Shanghai Children's Medical Center, West China Hospital, and Beijing Cancer Hospital have published recent pediatric 3D-printed prosthesis outcomes.

References [R28]

Intra-arterial chemotherapy for retinoblastoma — high-volume, eye-preserving treatment.

Retinoblastoma is the most common pediatric eye cancer, and conventional intravenous chemotherapy often fails to preserve the eye. Intra-arterial chemotherapy (IAC), guided by digital subtraction angiography, delivers high-concentration chemotherapy directly to the tumor through the ophthalmic artery — maximizing anti-tumor effect while sparing systemic tissue. The technique was pioneered at Memorial Sloan Kettering Cancer Center (Abramson and Gobin, 2006–2008). Beijing Tongren Hospital now performs one of the highest IAC volumes worldwide, with a reported pediatric survival rate of 91.2% and a 2-year eyeball-retention rate of 53% versus 27% for conventional intravenous chemotherapy. For international families, China provides high-volume IAC expertise integrated with pediatric oncology, ophthalmic surgery, and rehabilitation — at access and cost terms more favorable than most Western retinoblastoma programs.

References [R22-R23]

Pediatric proton therapy from age four — at one-third to one-half of US cost.

Proton therapy delivers radiation with sharp Bragg-peak targeting and zero exit dose, minimizing damage to children's developing brains, bones, and endocrine glands — critical for patients whose organs are still maturing. It is indicated for medulloblastoma, ependymoma, craniopharyngioma, retinoblastoma, rhabdomyosarcoma, and other pediatric brain or head-and-neck tumors near critical neural structures. Proton therapy hardware was developed primarily in the US, Switzerland, and Japan; China's edge is access, scale, and cost. More than ten proton therapy centers are now operational across China, with accumulated pediatric experience extending to children aged four and above. The cost of proton therapy in China is roughly one-third to one-half of US pricing — a major advantage for families whose home countries lack pediatric proton facilities or face long waiting lists.

References [R3][R6]

A domestically developed 360° rotating proton system for complex-position pediatric tumors.

Conventional proton devices support only fixed or limited-angle irradiation, making it difficult to optimally treat pediatric tumors adjacent to the brainstem, spinal cord, and orbital tissues — areas at high risk of vital-organ damage. The domestically developed 360° rotating proton therapy system rotates fully around the patient, delivering the beam from the optimal incident angle to precisely target deep, complex lesions while reducing the risk of long-term complications such as hearing loss, growth retardation, and endocrine disorders. The Ruijin Hospital Proton Center in Shanghai has adopted this system, specializing in precise radiotherapy for pediatric head, neck, and intracranial tumors.

References [R3][R26]

A domestic single-port robot — scarless tumor resection through one micro-incision.

Children's narrow body cavities and densely distributed vital organs make traditional open surgery especially traumatic — significant bleeding, prolonged recovery, and obvious scarring. The domestic single-port robotic surgical system completes precise tumor resection through a single 3–4 cm incision under high-definition 3D visualization, applicable to pediatric nephroblastoma, neuroblastoma, hepatoblastoma, and mediastinal tumors. Clinical data from Shanghai Children's Medical Center show significantly less intraoperative bleeding, lower postoperative pain scores, and shorter hospitalization versus traditional open surgery — minimizing the impact of surgical trauma on children's physical and psychological development.

References [R27]

Patient-specific 3D-printed guides — millimeter-level navigation for pediatric bone-tumor surgery.

Built from fused 3D anatomical data from preoperative MRI and CT (integrating bone, nerves, blood vessels, and tumor boundaries), customized 3D-printed surgical guides enable precise preoperative path planning and millimeter-level osteotomy during surgery — addressing the accuracy limits and healthy-tissue damage of traditional pediatric bone-tumor surgery. The technology controls osteotomy accuracy within one millimeter, improving resection completeness and limb-salvage rates when combined with 3D-printed bionic reconstruction scaffolds. For children with growing skeletons, extendable prostheses can be customized for minimally invasive periodic adjustment, effectively preventing postoperative limb-length discrepancy.

References [R28-R30]

In-country CAR-T manufacturing — turnaround as short as 24–36 hours at leading centers.

Pediatric CAR-T therapy depends critically on cell-processing manufacturing capacity. Conventional Western commercial CAR-T (e.g., tisagenlecleucel) typically takes 3–4 weeks from cell collection to infusion — a critical window during which rapidly progressing pediatric leukemias can deteriorate. Several Chinese centers and biotech partners have built domestic CAR-T manufacturing platforms that compress this turnaround to 24–36 hours in some cases, using fresh (non-cryopreserved) cell processing, decentralized GMP facilities, and hospital-integrated manufacturing. This is the infrastructure that enables the pediatric CAR-T pathway — domestically approved products such as pCAR-19B (Pulidekai) plus in-house clinical-trial CAR-T programs at Shanghai Children's Medical Center, Beijing Children's Hospital, and Boao Lecheng-affiliated facilities. For children where standard chemotherapy has failed, this manufacturing speed can be the difference between treatment in time and treatment too late.