Synthetic lethality between RB-loss and E2F3 inhibition in small cell cancers targeted by pyrimidine synthesis blockade

E Evan R. Abt (Department of Molecular and Medical Pharmacology, University of California) L Liang Wang G Grigor Varuzhanyan (Department of Microbiology, Immunology, and Molecular Genetics, University of California) J Jack Freeland (Department of Molecular and Medical Pharmacology, University of California) T Tian He (Department of Molecular and Medical Pharmacology, University of California) G Guadalupe M. Peña-Garcia (Department of Microbiology, Immunology, and Molecular Genetics, University of California) L Lauryn Ruegg (Department of Obstetrics and Gynecology, University of California) J Jami McLaughlin (Department of Microbiology, Immunology, and Molecular Genetics, University of California) D Donghui Cheng (Department of Microbiology, Immunology, and Molecular Genetics, University of California) N Nikolas G. Balanis (Department of Molecular and Medical Pharmacology, University of California) C Chia-Chun Chen (Department of Chemistry) Y Yang Xu Y Yi Xing (School of Energy and Environmental Engineering) S Sanaz Memarzadeh (Jonsson Comprehensive Cancer Center, University of California at Los Angeles) C Caius G. Radu (Department of Molecular and Medical Pharmacology, University of California) T Thomas G. Graeber O Owen N. Witte (Department of Molecular and Medical Pharmacology, University of California)

Abstract

Small cell carcinoma is a highly lethal cancer variant often found with neuroendocrine (NE) features, as exemplified by small cell lung cancer and small cell NE prostate cancer (SCPC). A genome-wide CRISPR dependency screen using SCPC models generated through human prostate cell transformation identifies a requirement for the transcription factor E2F3. E2F3 dependency is linked to RB inactivation, a near universal occurrence across small cell cancers. The requirement for E2F3 is shared by RB-deficient cells originating from the prostate, lung, and adnexa. In RB-deficient cancer cells, E2F3 inhibition restrains cell cycle progression, proliferation, and tumor growth in vivo. Inhibition of de novo pyrimidine synthesis limits E2F3 expression and suppresses small cell carcinoma proliferation in culture. Directly or indirectly targeting E2F3 to leverage a pan-cancer synthetic lethality resulting from RB inactivation represents a potential treatment strategy.

Article Details

Volume / Issue Vol. 123, Issue 12
Published March 24, 2026
ISSN 0027-8424
Publisher National Academy of Sciences

Authors (17)

E

Evan R. Abt

Department of Molecular and Medical Pharmacology, University of California

L

Liang Wang

G

Grigor Varuzhanyan

Department of Microbiology, Immunology, and Molecular Genetics, University of California

J

Jack Freeland

Department of Molecular and Medical Pharmacology, University of California

T

Tian He

Department of Molecular and Medical Pharmacology, University of California

G

Guadalupe M. Peña-Garcia

Department of Microbiology, Immunology, and Molecular Genetics, University of California

L

Lauryn Ruegg

Department of Obstetrics and Gynecology, University of California

J

Jami McLaughlin

Department of Microbiology, Immunology, and Molecular Genetics, University of California

D

Donghui Cheng

Department of Microbiology, Immunology, and Molecular Genetics, University of California

N

Nikolas G. Balanis

Department of Molecular and Medical Pharmacology, University of California

C

Chia-Chun Chen

Department of Chemistry

Y

Yang Xu

Y

Yi Xing

School of Energy and Environmental Engineering

S

Sanaz Memarzadeh

Jonsson Comprehensive Cancer Center, University of California at Los Angeles

C

Caius G. Radu

Department of Molecular and Medical Pharmacology, University of California

T

Thomas G. Graeber

O

Owen N. Witte

Department of Molecular and Medical Pharmacology, University of California