Vanessa's Comment:

A Phase 1 trial from Children's National in Washington, DC tested an experimental autologous (patient-derived), non-genetically engineered T-cell therapy targeting WT1, PRAME, and survivin in children with aggressive brain tumors. The study enrolled 11 children with newly diagnosed DIPG and 22 with recurrent non-brainstem brain tumors who received the treatment. The therapy was generally well tolerated, but there was one potentially treatment-related death from tumor swelling in a child with DIPG, which led to a study protocol change. Median overall survival in the DIPG group was 13.7 months. Among the patients with recurrent non-brainstem tumors, three patients (14%) remain disease-free for 31.8-51.6 months without additional treatment, including one patient who had a complete MRI response. The three long-term survivors had recurrent medulloblastoma, anaplastic ependymoma, and high-grade glioma. While this study was designed mainly to assess safety rather than efficacy, the results support further investigation of this personalized T-cell approach. 


Posted on: 07/20/2026

New autologous T cell therapy offers hope for children with brain tumors

 


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