HER2 in Non-Small Cell Lung Cancer (NSCLC): Evolution of the Therapeutic Landscape and Emerging Drugs—A Long Way to the Top

Non-small-cell lung cancer (NSCLC) can harbour different HER2 alterations: HER2 protein overexpression (2–35%), HER2 gene amplification (2–20%), and gene mutations (1–4%). The discovery of the HER2 gene in the 1980s raised great expectations for the treatment of several tumours. However, it was only...

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Main Authors: Pamela Trillo Aliaga, Gianluca Spitaleri, Ilaria Attili, Carla Corvaja, Elena Battaiotto, Panagiotis Agisilaos Angelopoulos, Ester Del Signore, Antonio Passaro, Filippo de Marinis
Format: Article
Language:English
Published: MDPI AG 2025-06-01
Series:Molecules
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Online Access:https://www.mdpi.com/1420-3049/30/12/2645
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author Pamela Trillo Aliaga
Gianluca Spitaleri
Ilaria Attili
Carla Corvaja
Elena Battaiotto
Panagiotis Agisilaos Angelopoulos
Ester Del Signore
Antonio Passaro
Filippo de Marinis
author_facet Pamela Trillo Aliaga
Gianluca Spitaleri
Ilaria Attili
Carla Corvaja
Elena Battaiotto
Panagiotis Agisilaos Angelopoulos
Ester Del Signore
Antonio Passaro
Filippo de Marinis
author_sort Pamela Trillo Aliaga
collection DOAJ
description Non-small-cell lung cancer (NSCLC) can harbour different HER2 alterations: HER2 protein overexpression (2–35%), HER2 gene amplification (2–20%), and gene mutations (1–4%). The discovery of the HER2 gene in the 1980s raised great expectations for the treatment of several tumours. However, it was only in 2004 that HER2 mutations were identified, and they currently represent a key druggable target in NSCLC. Despite numerous strengths, there is only one FDA/EMA-approved targeted therapy, an antibody-drug conjugate (ADC) called trastuzumab deruxtecan for pretreated patients with HER2 mutant NSCLC. In the first-line treatment, the standard of care (SoC) remains chemotherapy with or without immunotherapy. In the past, pan-HER tyrosine kinase inhibitors (TKIs) were extensively studied with poor results. But, two newly developed HER2-specific TKIs with low EGFR WT inhibition (BAY2927088 and zongertinib) reported encouraging results and received the breakthrough therapy designation from the FDA. Ongoing clinical trials are investigating new agents. This review focuses on HER2 alterations. Additionally, the anti-HER2 therapies explored so far will be discussed in detail, including the following: HER2 inhibitors (pan-inhibitors and selective inhibitors), monoclonal antibodies (mAbs), and ADCs. A section of this paper is dedicated to the role of immunotherapy in HER2-altered NSCLC. The last section of this paper focuses on the drugs under development and their challenges.
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spelling doaj-art-e8e96c6dce5d4173bcddead6de3866362025-08-20T02:21:14ZengMDPI AGMolecules1420-30492025-06-013012264510.3390/molecules30122645HER2 in Non-Small Cell Lung Cancer (NSCLC): Evolution of the Therapeutic Landscape and Emerging Drugs—A Long Way to the TopPamela Trillo Aliaga0Gianluca Spitaleri1Ilaria Attili2Carla Corvaja3Elena Battaiotto4Panagiotis Agisilaos Angelopoulos5Ester Del Signore6Antonio Passaro7Filippo de Marinis8Division of Thoracic Oncology, IEO—European Institute of Oncology, IRCCS, Via Ripamonti 435, 20141 Milan, ItalyDivision of Thoracic Oncology, IEO—European Institute of Oncology, IRCCS, Via Ripamonti 435, 20141 Milan, ItalyDivision of Thoracic Oncology, IEO—European Institute of Oncology, IRCCS, Via Ripamonti 435, 20141 Milan, ItalyDivision of Thoracic Oncology, IEO—European Institute of Oncology, IRCCS, Via Ripamonti 435, 20141 Milan, ItalyDivision of New Drugs and Early Drug Development for Innovative Therapies, European Institute of Oncology, IRCCS, Via Ripamonti 435, 20141 Milan, ItalyDivision of New Drugs and Early Drug Development for Innovative Therapies, European Institute of Oncology, IRCCS, Via Ripamonti 435, 20141 Milan, ItalyDivision of Thoracic Oncology, IEO—European Institute of Oncology, IRCCS, Via Ripamonti 435, 20141 Milan, ItalyDivision of Thoracic Oncology, IEO—European Institute of Oncology, IRCCS, Via Ripamonti 435, 20141 Milan, ItalyDivision of Thoracic Oncology, IEO—European Institute of Oncology, IRCCS, Via Ripamonti 435, 20141 Milan, ItalyNon-small-cell lung cancer (NSCLC) can harbour different HER2 alterations: HER2 protein overexpression (2–35%), HER2 gene amplification (2–20%), and gene mutations (1–4%). The discovery of the HER2 gene in the 1980s raised great expectations for the treatment of several tumours. However, it was only in 2004 that HER2 mutations were identified, and they currently represent a key druggable target in NSCLC. Despite numerous strengths, there is only one FDA/EMA-approved targeted therapy, an antibody-drug conjugate (ADC) called trastuzumab deruxtecan for pretreated patients with HER2 mutant NSCLC. In the first-line treatment, the standard of care (SoC) remains chemotherapy with or without immunotherapy. In the past, pan-HER tyrosine kinase inhibitors (TKIs) were extensively studied with poor results. But, two newly developed HER2-specific TKIs with low EGFR WT inhibition (BAY2927088 and zongertinib) reported encouraging results and received the breakthrough therapy designation from the FDA. Ongoing clinical trials are investigating new agents. This review focuses on HER2 alterations. Additionally, the anti-HER2 therapies explored so far will be discussed in detail, including the following: HER2 inhibitors (pan-inhibitors and selective inhibitors), monoclonal antibodies (mAbs), and ADCs. A section of this paper is dedicated to the role of immunotherapy in HER2-altered NSCLC. The last section of this paper focuses on the drugs under development and their challenges.https://www.mdpi.com/1420-3049/30/12/2645NSCLCadenocarcinomaHER2ERBB2targeted therapyantibody–drug conjugate
spellingShingle Pamela Trillo Aliaga
Gianluca Spitaleri
Ilaria Attili
Carla Corvaja
Elena Battaiotto
Panagiotis Agisilaos Angelopoulos
Ester Del Signore
Antonio Passaro
Filippo de Marinis
HER2 in Non-Small Cell Lung Cancer (NSCLC): Evolution of the Therapeutic Landscape and Emerging Drugs—A Long Way to the Top
Molecules
NSCLC
adenocarcinoma
HER2
ERBB2
targeted therapy
antibody–drug conjugate
title HER2 in Non-Small Cell Lung Cancer (NSCLC): Evolution of the Therapeutic Landscape and Emerging Drugs—A Long Way to the Top
title_full HER2 in Non-Small Cell Lung Cancer (NSCLC): Evolution of the Therapeutic Landscape and Emerging Drugs—A Long Way to the Top
title_fullStr HER2 in Non-Small Cell Lung Cancer (NSCLC): Evolution of the Therapeutic Landscape and Emerging Drugs—A Long Way to the Top
title_full_unstemmed HER2 in Non-Small Cell Lung Cancer (NSCLC): Evolution of the Therapeutic Landscape and Emerging Drugs—A Long Way to the Top
title_short HER2 in Non-Small Cell Lung Cancer (NSCLC): Evolution of the Therapeutic Landscape and Emerging Drugs—A Long Way to the Top
title_sort her2 in non small cell lung cancer nsclc evolution of the therapeutic landscape and emerging drugs a long way to the top
topic NSCLC
adenocarcinoma
HER2
ERBB2
targeted therapy
antibody–drug conjugate
url https://www.mdpi.com/1420-3049/30/12/2645
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