Suicide gene therapy using allogeneic adipose tissue-derived mesenchymal stem cell gene delivery vehicles in recurrent glioblastoma multiforme: a first-in-human, dose-escalation, phase I clinical trial.
Oraee-Yazdani S., Tavanaei R., Rostami F., Hajarizadeh A., Mehrabadi M., Akhlaghpasand M.
Clinical Trial with a reported sample of 3, published in J Transl Med (2023) — summary generated from the PubMed abstract.
Several human studies show positive signals, while research methods and sample sizes continue to develop.
- Level A · Stronger Clinical Evidence
- Level B · Emerging clinical evidence with positive signals
- Level C · Early human research exploring benefits
- Level D · Scientific groundwork from lab and animal studies
- Emerging · Emerging topic under active research
This page is generated from the PubMed record. The Thai description is an automated summary of bibliographic fields and the abstract, not a full translation, and is not medical advice.
- Study type
- Clinical Trial
- Journal
- J Transl Med (2023)
- Country
- England
- Reported sample size
- 3
- Source database
- PubMed
- PMID
- 37245011
- PMCID
- PMC10223860
- DOI
- 10.1186/s12967-023-04213-4
- Citations
- 19
Abstract (original English)
Background Glioblastoma multiforme (GBM) is associated with remarkably poor prognosis, and its treatment is challenging. This investigation aimed to evaluate the safety of suicide gene therapy using allogeneic adipose tissue-derived mesenchymal stem cells (ADSCs) carrying herpes simplex virus-thymidine kinase (HSV-TK) gene for the first time in patients with recurrent GBM. Methods This study was a first-in-human, open-label, single-arm, phase I clinical trial with a classic 3 + 3 dose escalation design. Patients who did not undergo surgery for their recurrence were included and received this gene therapy protocol. Patients received the intratumoral stereotactic injection of ADSCs according to the assigned dose followed by prodrug administration for 14 days. The first dosing cohort (n = 3) received 2.5 × 10 5 ADSCs; the second dosing cohort (n = 3) received 5 × 10 5 ADSCs; the third dosing cohort (n = 6) received 10 × 10 5 ADSCs. The primary outcome measure was the safety profile of the intervention. Results A total of 12 patients with recurrent GBM were recruited. The median follow-up was 16 (IQR, 14-18.5) months. This gene therapy protocol was safe and well tolerated. During the study period, eleven (91.7%) patients showed tumor progression, and nine (75.0%) died. The median overall survival (OS) was 16.0 months (95% CI 14.3-17.7) and the median progression-free survival (PFS)
What this study does not prove
- • This study does not prove SVF is an approved treatment or a replacement for standard care.
Evidence level
Several human studies show positive signals, while research methods and sample sizes continue to develop.
How we grade evidenceBrowse all related research
Filter the research library by this study's title keywords, author, or publication year.