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Table 2 Early phase clinical studies evaluating the role of mRNA vaccines for non-CHOL cancers

From: mRNA vaccine development for cholangiocarcinoma: a precise pipeline

Antigen

Vehicle

Combination

Indication

n

Outcomes

Clinical trial

WT1

DC

Durvalumab

Solid tumor Lymphoma

264

Increased WT1-specific CD8+ T cells

NCT03739931 [35]

WT1, PRAME, and CMVpp65

DC

Acute myeloid leukemia

13

Enhanced PRAME and WT1-specific immunity; 5 patients in CR, with an observation period of up to 840 d

NCT01734304

CEA-peptide

LNP

Oxaliplatin/Capecitabine

Colorectal cancer

30

CEA peptide-specific T-cells detected in 8/11 patients in the peptide group

NCT00228189 [36]

Tumor RNA plus synthetic CD40L RNA

DC

Sunitinib

Renal cell carcinoma

25

13 patients (62%) experienced clinical benefit (PR + CR)

NCT00678119 [37]

Tumor-associated antigens

Liposome

PD-1 inhibitor

Melanoma

119

Increased antigen-specific cytotoxic T-cell were observed

NCT02410733 [38]

Autologous tumor-mRNA

DC

IL-2

Melanoma

31

Antigen-specific immune response demonstrated in 51.6% patients; immune responders had better survival (median 14 months vs. 6 months, P = 0.030)

NCT01278940 [39]

CD40 ligand TLR4, gp100 and tyrosinase

DC

Melanoma

28

1 PR and 2 SD observed in 8 patients

NCT01530698 [40]

MageA3, MageA1, Melan-A, Tyrosinase, Survivin, and gp100

Protamine-protected

GM-CSF s.c

Melanoma

20

Antigen-specific T cells detected in 2/4 evaluable patients; 1 CR observed in 7 patients

NCT00204607 [41]

hTERT

DC

Acute myeloid leukemia

21

11 patients (58%) developed hTERT-specific T-cell responses

NCT00510133 [42]

  1. WT1 Wilms’ tumor 1 antigen, DC dendritic cell, CMV cytomegalovirus, PRAME preferentially expressed antigen in melanoma, CR complete response, CEA carcinoembryonic antigen, LNP lipid nanoparticle, RNA ribonucleic acid, PR partial response, PD-1 programmed death-1, IL-2 interleukin-2, TLR4 toll-likereceptor4, gp100 glycoprotein 100, SD stable disease, MageA melanoma-associated antigen, GM-CSF granulocyte–macrophage colony-stimulating factor, s.c. subcutaneous injection, hTERT human telomerase reverse transcriptase, “–” no combined drug