Clinical Report: CAR T Breaks New Ground in AML and Solid Tumors

Overview

The University of Colorado Anschutz has received FDA clearance for a CAR T-cell therapy targeting CD64 in adults with relapsed or refractory acute myeloid leukemia (AML). Additionally, a new study highlights the potential of uPAR-targeted CAR T cells to overcome barriers in solid tumors by dismantling immunosuppressive microenvironments.

Background

Chimeric antigen receptor (CAR) T-cell therapy has revolutionized treatment for certain hematological malignancies, yet challenges remain in solid tumors and specific leukemias like AML. The FDA clearance for a campus-developed CAR T program represents a significant advancement in the field, particularly for patients with limited treatment options. Understanding the mechanisms of CAR T efficacy and safety is crucial for optimizing therapeutic strategies.

Data Highlights

No numerical data available in the provided source material.

Key Findings

• The University of Colorado Anschutz has launched the first U.S.-cleared CAR T program targeting CD64 for adults with relapsed or refractory AML.
• The Phase 1 trial will assess the safety, tolerability, and optimal dosing of the CAR T therapy.
• uPAR was identified as a unifying marker in aggressive solid tumors, associated with poor prognosis and present in both tumor and surrounding cells.
• CAR T cells engineered to target uPAR showed broad efficacy across multiple cancer models, including metastatic disease.
• The redesigned base editor ABE8eY149V significantly reduces off-target mutations while maintaining high editing efficiency.
• Base editing therapy has shown promise in reversing seizures in a mouse model of SCN8A epilepsy.

Clinical Implications

Highlight specific patient populations that may benefit from the new CAR T therapy.

Conclusion

Reiterate the importance of ongoing research and potential future directions for CAR T therapies.

Related Resources & Content

1. University of Colorado Anschutz Gates Institute, News, 2026 -- First-in-U.S. FDA Clearance for Campus-Built CAR T-Cell Therapy
2. Nature, 2025 -- Indications for haematopoietic cell transplantation and CAR-T for haematological diseases, solid tumours and immune disorders: 2025 EBMT practice recommendations
3. PMC, 2025 -- CAR-T Cell Therapy for Acute Myeloid Leukemia: Where Do We Stand Now?
4. Franklin / MDSpire — Current Ex Vivo CAR-T Benchmarks Define the Bar for Emerging In Vivo CRISPR-Based T Cell Engineering
5. The ASCO Post — New Data on ALK Inhibitors and CAR T-Cell Therapies
6. The ASCO Post — CAR T-Cell Therapy Makes Further Inroads in Lymphoma and Myeloma
7. Journal of Neuro-Oncology — Advancements in Chimeric Antigen Receptor T-Cell Therapy for Glioblastoma Multiforme: Historical Context, Current Insights, and Future Directions
8. Indications for haematopoietic cell transplantation and CAR-T for haematological diseases, solid tumours and immune disorders: 2025 EBMT practice recommendations
9. CAR-T Cell Therapy for Acute Myeloid Leukemia: Where Do We Stand Now? - PMC
10. The University of Colorado Anschutz Gates Institute Achieves First-in-U.S. FDA Clearance for Campus-Built CAR T-Cell Therapy