Grant Award Details
Grant Application Details
- Phase I Study of IL13Rα2-Targeting CAR T Cells After Lymphodepletion for Children with Refractory or Recurrent Malignant Brain Tumors
Therapeutic Candidate or Device
Autologous chimeric antigen receptor T cells derived from naive/memory T cells and engineered to target IL13Rα2 on pediatric malignant brain tumors.
Recurrent/refractory malignant pediatric brain tumors that express the tumor-associated antigen IL13Rα2.
Naive and memory T cells are harvested from patients and reprogrammed to express chimeric antigen receptors for targeted tumor killing. Upon adoptive transfer, the CAR product recognizes and destroys malignant brain tumor cells expressing IL13Rα2. Lymphodepleting chemotherapy is included in this trial to augment the proliferation, persistence, and efficacy of these cells.
Unmet Medical Need
This proposal addresses the critical unmet medical need for effective therapies to treat aggressive pediatric brain tumors, including glioblastoma, medulloblastoma, atypical teratoid/rhabdoid tumor, diffuse midline glioma, and others.
Phase I trial completed and Phase II trial enabled
Major Proposed Activities
- manufacture and release of IL13BBζ-Tn/mem CAR T cells
- evaluate safety and feasibility of intraventricularly-delivered CAR T cells administered after lymphodepletion in pediatric patients
- develop and establish methods and target populations for Phase II clinical trial
This proposal will benefit California residents by developing more effective and less toxic therapies for aggressive pediatric brain tumors. This will result in lives prolonged or saved, fewer missed days of work, and less economic and emotional burden to families and caregivers. Additionally, it will create and support skilled jobs, serve to recruit talent from elsewhere in the country/world, and cement California's leadership in this field.