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In Vitro Tumor Cell Rechallenge For Predictive Evaluation of Chimeric Antigen Receptor T Cell Antitumor Function
Published on: February 27, 2019
CD56-chimeric antigen receptor T-cell therapy for refractory/recurrent rhabdomyosarcoma: A 3.5-year follow-up case
Chiyi Jiang1, Wen Zhao1, Maoquan Qin1
1Beijing Key Laboratory of Pediatric Hematology Oncology, National Discipline of Pediatrics, Ministry of Education, MOE Key Laboratory of Major Diseases in Children, Hematology Oncology Center, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing.
Rationale:
Rhabdomyosarcoma (RMS) is a common soft tissue sarcoma in children with high malignancy. The prognosis of refractory recurrent RMS is extremely poor, and the 5-year survival rate is less than 20%.
Patient Concerns:
We reported a 2-year-old male patient with RMS who underwent 3 operations and 2 recurrences while being treated with regular multidisciplinary therapy.
Diagnoses:
A diagnosis of embryonal rhabdomyosarcoma with primary bladder (IIIa, TNM stage 2, and medium risk group) was made.
Interventions:
After repeated recurrence, the patient was treated with chimeric antigen receptor T (CAR-T) cells, which had a safety mechanism and specifically bound the CD56 antigen in the fourth generation.
Outcomes:
The process of CAR-T cell transfusion was smooth, and there were no significant cytokine release syndrome manifestations after reinfusion. The patient was in complete remission at last follow-up visit after 3.5 years.
Conclusion:
CD56-CAR-T cell therapy is a safe and effective approach and may be an option for children with solid tumors who are nonresponsive to conventional radiotherapy and chemotherapy, or are unsuitable for hematopoietic stem cell transplantation.
Insights
Chimeric antigen receptor T (CAR-T) cell therapy targeting CD56 shows promise for children with recurrent rhabdomyosarcoma. This innovative treatment led to complete remission in a young patient, offering hope for difficult-to-treat solid tumors.
Area of Science:
- Pediatric Oncology
- Immunotherapy
- Cancer Research
Background:
- Rhabdomyosarcoma (RMS) is a highly malignant pediatric soft tissue sarcoma with a poor prognosis for refractory recurrent cases.
- Existing treatments offer limited success, with a 5-year survival rate below 20% for advanced disease.
Observation:
- A 2-year-old male diagnosed with embryonal rhabdomyosarcoma experienced multiple recurrences despite standard multidisciplinary care and surgical interventions.
- The patient received fourth-generation CD56-targeting chimeric antigen receptor T (CAR-T) cell therapy after disease progression.
Findings:
- The CD56-CAR-T cell transfusion was well-tolerated, with no significant cytokine release syndrome.
- The patient achieved complete remission and remained disease-free at a 3.5-year follow-up.
Implications:
- CD56-CAR-T cell therapy presents a safe and effective treatment option for pediatric solid tumors, including rhabdomyosarcoma.
- This immunotherapy may benefit children unresponsive to chemotherapy and radiotherapy or ineligible for stem cell transplantation.
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