Autologous dendritic cells transfected with prostate-specific antigen RNA stimulate CTL responses against metastatic prostate tumors.
Level 4 - case-series / case-control
Phase I single-arm dose-escalation study without a control group.
PubMed 11828001 · doi:10.1172/JCI14364
What was done
A phase I dose-escalation trial assessed the safety, feasibility, and immunological activity of autologous dendritic cells transfected with mRNA encoding prostate-specific antigen (PSA) in 13 patients with metastatic prostate cancer.
What was found
No dose-limiting toxicity, adverse effects, or autoimmunity were detected in the 13 subjects. Induction of PSA-specific T-cell responses occurred in all 13 patients. Vaccination was associated with a significant decrease in the log slope PSA in 6 of 7 evaluated subjects, and transient molecular clearance of circulating tumor cells was observed in all 3 analyzed patients.
Why it matters
It demonstrates initial human proof-of-concept that RNA-transfected dendritic cells can safely stimulate an immune response against a tumor self-antigen in metastatic prostate cancer.
Limits
The study is limited by a small sample size (n = 13) and an uncontrolled, single-arm design. Clinical outcomes were limited to surrogate markers evaluated in only small subsets (n = 7 for PSA kinetics, n = 3 for circulating tumor cells) without reporting hard endpoints such as survival.
Cited by
- supports The first human clinical trial using mRNA technology took place in 2001 using mRNA-induced dendritic cells.