Nanoparticle delivery of a prodrug-activating bacterial enzyme leads to anti-tumor responses

S Sebastian G. Huayamares (Wallace H. Coulter Department of Biomedical Engineering, Emory University School of Medicine and Georgia Institute of Technology) L Liming Lian (Wallace H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology and Emory University School of Medicine) R Regina Rab Y Yuning Hou A Afsane Radmand (Petit Institute for Bioengineering and Biosciences, Georgia Institute of Technology) H Hyejin Kim (Wallace H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology and Emory University School of Medicine) R Ryan Zenhausern (Wallace H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology and Emory University School of Medicine) B Bhagelu R. Achyut M Melissa Gilbert Ross M Melissa P. Lokugamage D David Loughrey (Wallace H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology and Emory University School of Medicine) H Hannah E. Peck E Elisa Schrader Echeverri (Wallace H. Coulter Department of Biomedical Engineering, Emory University School of Medicine and Georgia Institute of Technology) A Alejandro J. Da Silva Sanchez A Aram Shajii A Andrea Li K Karen E. Tiegreen P Philip J. Santangelo (Wallace H. Coulter Department of Biomedical Engineering, Emory University School of Medicine and Georgia Institute of Technology) E Eric J. Sorscher J James E. Dahlman (Wallace H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology and Emory University School of Medicine)

Abstract

Abstract Most cancer patients diagnosed with late-stage head and neck squamous cell carcinoma are treated with chemoradiotherapy, which can lead to toxicity. One potential alternative is tumor-limited conversion of a prodrug into its cytotoxic form. We reason this could be achieved by transient and tumor-specific expression of purine nucleoside phosphorylase (PNP), an Escherichia coli enzyme that converts fludarabine into 2-fluoroadenine, a potent cytotoxic drug. To efficiently express bacterial PNP in tumors, we evaluate 44 chemically distinct lipid nanoparticles (LNPs) using species-agnostic DNA barcoding in tumor-bearing mice. Our lead LNP, designated LNP intratumoral (LNPIT), delivers mRNA that leads to PNP expression in vivo. Additionally, in tumor cells transfected with LNPIT, we observe upregulated pathways related to RNA and protein metabolism, providing insight into the tumor cell response to LNPs in vivo. When mice are treated with LNPIT-PNP, then subsequently given fludarabine phosphate, we observe anti-tumor responses. These data are consistent with an approach in which LNP-mRNA expression of a bacterial enzyme activates a prodrug in solid tumors.

Article Details

Volume / Issue Vol. 16, Issue 1
Published April 12, 2025
ISSN 2041-1723
Publisher Nature Portfolio

Journal Info

Nature Communications

Nature Portfolio

ISSN: 2041-1723 Open Access Life Sciences

Authors (20)

S

Sebastian G. Huayamares

Wallace H. Coulter Department of Biomedical Engineering, Emory University School of Medicine and Georgia Institute of Technology

L

Liming Lian

Wallace H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology and Emory University School of Medicine

R

Regina Rab

Y

Yuning Hou

A

Afsane Radmand

Petit Institute for Bioengineering and Biosciences, Georgia Institute of Technology

H

Hyejin Kim

Wallace H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology and Emory University School of Medicine

R

Ryan Zenhausern

Wallace H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology and Emory University School of Medicine

B

Bhagelu R. Achyut

M

Melissa Gilbert Ross

M

Melissa P. Lokugamage

D

David Loughrey

Wallace H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology and Emory University School of Medicine

H

Hannah E. Peck

E

Elisa Schrader Echeverri

Wallace H. Coulter Department of Biomedical Engineering, Emory University School of Medicine and Georgia Institute of Technology

A

Alejandro J. Da Silva Sanchez

A

Aram Shajii

A

Andrea Li

K

Karen E. Tiegreen

P

Philip J. Santangelo

Wallace H. Coulter Department of Biomedical Engineering, Emory University School of Medicine and Georgia Institute of Technology

E

Eric J. Sorscher

J

James E. Dahlman

Wallace H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology and Emory University School of Medicine