Weide · Journal of immunotherapy (Hagerstown, Md. : 1997) 2008 · single-arm phase I/II clinical trial · n=15

Results of the first phase I/II clinical vaccination trial with direct injection of mRNA.

Cited 262 times in the scientific literature.

Level 4 - case-series / case-control

Single-arm phase I/II trial (case series) without a control group

PubMed 18481387 · doi:10.1097/CJI.0b013e31815ce501 · record verified 2026-08-31

What was done

Fifteen patients with metastatic melanoma were enrolled in a phase I/II trial testing direct intradermal vaccination of autologous naked mRNA. For each patient, growing metastases were resected, total RNA was extracted, reverse-transcribed, amplified, and cloned into cDNA libraries to generate in vitro-transcribed mRNA. Autologous mRNA preparations were administered intradermally alongside granulocyte-macrophage colony-stimulating factor (GM-CSF) adjuvant to assess feasibility, phase I safety criteria, and immune responses.

What was found

The abstract reports no numeric values, percentages, or statistical metrics. The procedure was reported as feasible and safe based on phase 1 criteria, and an increase in antitumor humoral immune response was observed in an unspecified subset of patients. Direct clinical effectiveness for antitumor immunotherapy was not demonstrated.

Why it matters

This trial provided early proof-of-concept in humans that direct intradermal injection of naked mRNA is safe, feasible, and capable of eliciting humoral immune responses against autologous tumor antigens.

Limits

The study is an uncontrolled, single-arm trial with a very small sample size (n = 15). The abstract reports no quantitative immunological or clinical data, and therapeutic efficacy could not be established.

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