CTNI-30. MESENCHYMAL STEM CELL (MSC11FCD) BASED THERAPY ELICITED A FAVORABLE RESPONSE WITH EFFICIENT 5-FU CONVERSION IN PATIENTS WITH RECURRENT GLIOBLASTOMA: AN OPEN LABEL, PHASE I/IIA TRIAL

• DOI: 10.1093/neuonc/noad179.0312
• Publication Date: 2023-11-11T23:05:58Z
• Authors: Jaejoon Lim; Da-Young Chang; Jeongmin Sim; Jin-Hwa Jung; JeongMan Park; Yu Jin Kim; Dongkil Kim; Sojung Hwang; Sung-Soo Kim; Hea-Young Cho; Haeyoung Suh-Kim; Kyunggi Cho
• Keywords: Cytosine deaminase; Suicide gene

Abstract Mesenchymal stem cells (MSC) are being developed as therapeutic agents for treatment of various diseases due to their high tropism damaged sites and cancers, low immunogenicity. We developing technologies efficiently transfer genes including suicide that will be used suppress tumor growth or ensure safety against possible adverse events develop off-the-shelf formulations allogeneic uses. MSCs were engineered express a gene encoding non-human cytosine deaminase (CD) enzymes. aimed conduct phase I/IIa clinical trial investigate the maximum tolerated dose, safety, efficiency MSC11FCD in recurrent glioblastoma (GBM) patients. This study was single-arm, open label, investigator-initiated on 10 patients recruited between 2021. The administered via intra-tumoral injection after surgical resection. No severe dose-dependent toxicities observed. Median overall survival not reached, median progression-free over 4 months. identified efficient 5-FU production specific signature biomarkers predict response therapeutics GBM, which might provide new strategy this highly aggressive tumor. In conclusion, therapy safe showed GBM Specific can screen individuals who benefit from therapy.