pH-dependent dissociation from CTLA-4 in early endosomes improves both safety and antitumor activity of anti-CTLA-4 antibodies

M Meiyu Zhang (Department of Immunology and Microbiology, Shanghai Institute of Immunology, Shanghai Jiao Tong University School of Medicine) J Jinmei Li (NHC Key Laboratory of Biotechnology for Microbial Drugs, State Key Laboratory of Bioactive Substance & Function of Natural Medicines, Institute of Medicinal Biotechnology) K Kepeng Yan (Department of Immunology and Microbiology, Shanghai Institute of Immunology, Shanghai Jiao Tong University School of Medicine) H Haoyue Zhou (Department of Immunology and Microbiology, Shanghai Institute of Immunology, Shanghai Jiao Tong University School of Medicine) S Song Mei (Department of Immunology and Microbiology, Shanghai Institute of Immunology, Shanghai Jiao Tong University School of Medicine) B Benyu Wang (Department of Immunology and Microbiology, Shanghai Institute of Immunology, Shanghai Jiao Tong University School of Medicine) D Dongyang Li (Department of Materials Science and Engineering) X Xuexiang Du (Division of Immunotherapy, Institute of Human Virology, University of Maryland School of Medicine) M Mingyue Liu (Division of Immunotherapy, Institute of Human Virology, University of Maryland School of Medicine) P Peng Zhang J James K. Fields (Division of Immunotherapy, Institute of Human Virology, University of Maryland School of Medicine) L Lei Ye (Innovative Centre for Flexible Devices (iFLEX), Max Planck-NTU Joint Lab for Artificial Senses, School of Materials Science and Engineering, Nanyang Technological University, 50 Nanyang Avenue, Singapore 639798, Republic of Singapore) P Pan Zheng (Key Laboratory of Superlight Materials & Surface Technology of Ministry of Education, College of Material Sciences and Chemical Engineering, Harbin Engineering University, Harbin 150001, P. R. China) Y Yang Liu M Michael J. Lenardo Y Yan Zhang

Abstract

Anti-CTLA-4 Abs (ACAs) are a breakthrough for cancer therapy, but their potential is limited by immunotherapy-related adverse events (irAE). We previously reported that ACAs with acidic pH-sensitive binding to CTLA-4 exhibit higher antitumor activity with fewer irAE. We now test a panel of variants of Ipilimumab (Ipi), the first ACA cancer therapeutic, for tumoricidal efficacy and irAE. Surprisingly, not all pH-sensitive Ipi variants exhibited an enhanced therapeutic index. Ipi13, which retained binding to CTLA-4 at pH 6.0 but dissociated at lower pH, showed no enhancement. By contrast, Ipi25, which dissociates from CTLA-4 at pH 6.0, the pH of the early endosome (EE), showed greater tumor regression and less severe irAE. Confocal microscopy showed that Ipi13 maintained colocalization with CTLA-4 at the late endosomes (LE) and lysosomes resulting in lysosomal degradation of CTLA-4. Conversely, Ipi25 did not colocalize with CTLA-4 in LE or lysosomes after endocytosis but allowed both proteins to transfer to recycling endosomes. EE dissociation was also characteristic of variants of Tremelimumab (Treme), another clinical ACA, that showed better efficacy and fewer side effects. Thus, our data reveal the significance of early intracellular dissociation from CTLA-4 to improve ACAs for safer and more effective cancer immunotherapy.

Article Details

Volume / Issue Vol. 122, Issue 8
Published February 25, 2025
ISSN 0027-8424
Publisher National Academy of Sciences

Authors (16)

M

Meiyu Zhang

Department of Immunology and Microbiology, Shanghai Institute of Immunology, Shanghai Jiao Tong University School of Medicine

J

Jinmei Li

NHC Key Laboratory of Biotechnology for Microbial Drugs, State Key Laboratory of Bioactive Substance & Function of Natural Medicines, Institute of Medicinal Biotechnology

K

Kepeng Yan

Department of Immunology and Microbiology, Shanghai Institute of Immunology, Shanghai Jiao Tong University School of Medicine

H

Haoyue Zhou

Department of Immunology and Microbiology, Shanghai Institute of Immunology, Shanghai Jiao Tong University School of Medicine

S

Song Mei

Department of Immunology and Microbiology, Shanghai Institute of Immunology, Shanghai Jiao Tong University School of Medicine

B

Benyu Wang

Department of Immunology and Microbiology, Shanghai Institute of Immunology, Shanghai Jiao Tong University School of Medicine

D

Dongyang Li

Department of Materials Science and Engineering

X

Xuexiang Du

Division of Immunotherapy, Institute of Human Virology, University of Maryland School of Medicine

M

Mingyue Liu

Division of Immunotherapy, Institute of Human Virology, University of Maryland School of Medicine

P

Peng Zhang

J

James K. Fields

Division of Immunotherapy, Institute of Human Virology, University of Maryland School of Medicine

L

Lei Ye

Innovative Centre for Flexible Devices (iFLEX), Max Planck-NTU Joint Lab for Artificial Senses, School of Materials Science and Engineering, Nanyang Technological University, 50 Nanyang Avenue, Singapore 639798, Republic of Singapore

P

Pan Zheng

Key Laboratory of Superlight Materials & Surface Technology of Ministry of Education, College of Material Sciences and Chemical Engineering, Harbin Engineering University, Harbin 150001, P. R. China

Y

Yang Liu

M

Michael J. Lenardo

Y

Yan Zhang