Proteasome subunit PSMD1 is a key therapeutic target in multiple myeloma

T Ting Du T Teng Fang (School of Airspace Science and Engineering, Shandong University 1 , Weihai 264209,) S Sindhu Pillai (1Department of Medical Oncology, LeBow Institute for Myeloma Therapeutics and Jerome Lipper Myeloma Center, Dana-Farber Cancer Institute, Boston, MA) A Arghya Ray (1Department of Medical Oncology, LeBow Institute for Myeloma Therapeutics and Jerome Lipper Myeloma Center, Dana-Farber Cancer Institute, Boston, MA) M Minxing Wang (1Department of Medical Oncology, LeBow Institute for Myeloma Therapeutics and Jerome Lipper Myeloma Center, Dana-Farber Cancer Institute, Boston, MA) X Xueping Wan (1Department of Medical Oncology, LeBow Institute for Myeloma Therapeutics and Jerome Lipper Myeloma Center, Dana-Farber Cancer Institute, Boston, MA) K Kenneth Wen (1Department of Medical Oncology, LeBow Institute for Myeloma Therapeutics and Jerome Lipper Myeloma Center, Dana-Farber Cancer Institute, Boston, MA) Y Yuntong Liu (1Department of Medical Oncology, LeBow Institute for Myeloma Therapeutics and Jerome Lipper Myeloma Center, Dana-Farber Cancer Institute, Boston, MA) J Jingyu Xu (1Department of Medical Oncology, LeBow Institute for Myeloma Therapeutics and Jerome Lipper Myeloma Center, Dana-Farber Cancer Institute, Boston, MA) M Md Abu Musa (1Department of Medical Oncology, LeBow Institute for Myeloma Therapeutics and Jerome Lipper Myeloma Center, Dana-Farber Cancer Institute, Boston, MA) X Xiangdong Liu M Mariateresa Fulciniti (Dana Farber Cancer Institute, Boston, Massachusetts, United States) N Nikhil C. Munshi F Filip Garbicz R Ruben D. Carrasco (Department of Pathology, Brigham and Women's Hospital, 75 Francis St, Boston, MA 02115, United States) Y Yao Yao Z Zhongkun Zhang (1Department of Medical Oncology, LeBow Institute for Myeloma Therapeutics and Jerome Lipper Myeloma Center, Dana-Farber Cancer Institute, Boston, MA) Y Yan Song (State Key Laboratory of Membrane Biology, School of Life Sciences, Peking University) K Kenneth C. Anderson

Abstract

Abstract We found that PSMD1, a key subunit of the 19S proteasome regulatory particle, was overexpressed and correlated with poor prognosis in multiple myeloma (MM). Genetic depletion of PSMD1 decreased cancer cell viability, induced polyubiquitinated protein accumulation, and promoted apoptosis. Proteomic analysis revealed the activation of immune-related pathways, suggesting the potential for immune modulation. Targeting PSMD1 with small interfering RNA (siRNA), delivered via lipid nanoparticles (LNPs), reduced tumor growth in MM cell lines and primary patient samples while sparing normal cells. It also overcame proteasome inhibitor resistance and the protective effects of the bone marrow milieu. In MM xenograft mouse models, PSMD1 siRNA LNPs significantly reduced tumor growth and prolonged survival. In addition, PSMD1 depletion had similar effects on other types of cancer cell lines. These findings position PSMD1 as a critical target in cancer therapy, with broad implications for overcoming drug resistance, improving therapeutic outcomes, and potentially affecting immune responses across various cancers. These findings provide a foundation for the clinical development of PSMD1-targeted therapies in myeloma and other malignancies.

Article Details

Journal Blood
Volume / Issue Vol. 147, Issue 20
Published May 14, 2026
Pages 2344-2357
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (19)

T

Ting Du

T

Teng Fang

School of Airspace Science and Engineering, Shandong University 1 , Weihai 264209,

S

Sindhu Pillai

1Department of Medical Oncology, LeBow Institute for Myeloma Therapeutics and Jerome Lipper Myeloma Center, Dana-Farber Cancer Institute, Boston, MA

A

Arghya Ray

1Department of Medical Oncology, LeBow Institute for Myeloma Therapeutics and Jerome Lipper Myeloma Center, Dana-Farber Cancer Institute, Boston, MA

M

Minxing Wang

1Department of Medical Oncology, LeBow Institute for Myeloma Therapeutics and Jerome Lipper Myeloma Center, Dana-Farber Cancer Institute, Boston, MA

X

Xueping Wan

1Department of Medical Oncology, LeBow Institute for Myeloma Therapeutics and Jerome Lipper Myeloma Center, Dana-Farber Cancer Institute, Boston, MA

K

Kenneth Wen

1Department of Medical Oncology, LeBow Institute for Myeloma Therapeutics and Jerome Lipper Myeloma Center, Dana-Farber Cancer Institute, Boston, MA

Y

Yuntong Liu

1Department of Medical Oncology, LeBow Institute for Myeloma Therapeutics and Jerome Lipper Myeloma Center, Dana-Farber Cancer Institute, Boston, MA

J

Jingyu Xu

1Department of Medical Oncology, LeBow Institute for Myeloma Therapeutics and Jerome Lipper Myeloma Center, Dana-Farber Cancer Institute, Boston, MA

M

Md Abu Musa

1Department of Medical Oncology, LeBow Institute for Myeloma Therapeutics and Jerome Lipper Myeloma Center, Dana-Farber Cancer Institute, Boston, MA

X

Xiangdong Liu

M

Mariateresa Fulciniti

Dana Farber Cancer Institute, Boston, Massachusetts, United States

N

Nikhil C. Munshi

F

Filip Garbicz

R

Ruben D. Carrasco

Department of Pathology, Brigham and Women's Hospital, 75 Francis St, Boston, MA 02115, United States

Y

Yao Yao

Z

Zhongkun Zhang

1Department of Medical Oncology, LeBow Institute for Myeloma Therapeutics and Jerome Lipper Myeloma Center, Dana-Farber Cancer Institute, Boston, MA

Y

Yan Song

State Key Laboratory of Membrane Biology, School of Life Sciences, Peking University

K

Kenneth C. Anderson