Definition and research guide
This page combines a MeSH definition, publication statistics and selected research abstracts. It is a starting point for reading the literature, not a medical textbook or a systematic evidence review.
Content available: 4 complete abstracts (866 words), 10 references. Full article text is not stored or reproduced here; follow the PMID, DOI or PMC links to read the original sources and their access terms.
Definition
Treatments with drugs which interact with or block synthesis of specific cellular components characteristic of the individual's disease in order to stop or interrupt the specific biochemical dysfunction involved in progression of the disease.
Definition: NLM Medical Subject Headings (MeSH) scope note (Public domain (U.S. National Library of Medicine); attribution requested). View the MeSH record.
Also known as: Molecular Targeted Therapies; Targeted Therapy, Molecular; Therapy, Molecular Targeted; Targeted Molecular Therapy; Molecular Therapy, Targeted; Targeted Molecular Therapies; Therapy, Targeted Molecular.
Where it fits in the MeSH classification
- Broader topics
- Drug Therapy
- See also
- Immunoconjugates
Research landscape
PubMed indexes 41,134 records tagged with the MeSH heading “Molecular Targeted Therapy” (counts retrieved 2026-10-29). 12,697 of them (30.9%) were published from 2020 onward, and the busiest period so far is 2010-2019 with 28,754 records.
Reviews account for 25,094 records (61.0%) and meta-analyses for 355. Randomized controlled trials number 162 (0.4%) as a share of all records on the topic. Randomized trials make up only a small share of this literature, so much of what is known comes from laboratory, observational or descriptive work.
Show the data
| Period | Records |
|---|---|
| through 1999 | 0 |
| 2000-2009 | 33 |
| 2010-2019 | 28,754 |
| 2020 onward | 12,697 |
Publication-type chart data
| Type | Records | Share |
|---|---|---|
| Reviews | 25,094 | 61.0% |
| Meta-analyses | 355 | 0.9% |
| Randomized controlled trials | 162 | 0.4% |
Method: PubMed query "Molecular Targeted Therapy"[MeSH Terms] with publication-type and date filters, retrieved 2026-10-29.
Reading the evidence in pharmacology
Drug evidence builds in layers: laboratory pharmacology describes how a compound acts, pharmacokinetic studies describe how the body handles it, and randomized trials test whether it helps and what harms it causes. Post-marketing surveillance and observational studies reveal rarer adverse effects but cannot prove causation. This page is educational and is not a recommendation to start, stop or change any medicine; decisions about treatment belong with a qualified clinician.
Evidence from open-access studies
These open-access studies come from PubMed’s best-match results for this heading (papers whose main topic it is), ranked here by citation count, with one recent addition. Each abstract is reproduced in full under its recorded open licence so that you can read the authors’ own words. They illustrate the literature; they are not a systematic summary of it. Citation counts measure attention, not reliability or clinical benefit.
| Study / authors | Year | Source label / design | Europe PMC citations | Licence / source |
|---|---|---|---|---|
| Emerging Biomimetic Drug Delivery Nanoparticles Inspired by Extracellular Vesicles | 2025 | Review article Design not recorded in structured metadata | 28 | cc by PMID 40840553 · DOI · PMC12370395 |
| Claudin18.2 as a Promising Therapeutic Target in Gastric Cancer | 2025 | Review article Design not recorded in structured metadata | 18 | cc by PMID 40862764 · DOI · PMC12384651 |
| Aging microenvironment in osteoarthritis focusing on early-stage alterations and targeted therapies | 2025 | Review article Design not recorded in structured metadata | 17 | cc by PMID 41073390 · DOI · PMC12514018 |
| Leveraging large-scale biobanks for therapeutic target discovery | 2026 | Research article Design not recorded in structured metadata | 6 | cc by PMID 41376171 · DOI · PMC12799792 |
Emerging Biomimetic Drug Delivery Nanoparticles Inspired by Extracellular Vesicles [1]
Abstract reading summary — source excerpts
- Opening excerpt
- Nanoparticles (NPs) made up of cellular components such as extracellular vesicles (EVs) with a biomimetic outlook have emerged as a revolutionary approach in nanomedicine, providing significant benefits for targeted drug administration, immunotherapy, monitoring therapeutic response, and diagnostic applications.
- Closing excerpt
- This article is categorized under: Nanotechnology Approaches to Biology > Nanoscale Systems in Biology Therapeutic Approaches and Drug Discovery > Nanomedicine for Oncologic Disease.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (273 words)
Nanoparticles (NPs) made up of cellular components such as extracellular vesicles (EVs) with a biomimetic outlook have emerged as a revolutionary approach in nanomedicine, providing significant benefits for targeted drug administration, immunotherapy, monitoring therapeutic response, and diagnostic applications. Utilizing the distinctive characteristics of natural cell membranes, membrane proteins, and cellular contents, these biomimetic NPs acquire essential biological functions from their source and biogenesis, including immune evasion, extended circulation, and target recognition, rendering them optimal candidates for therapeutic applications. This review offers a comprehensive examination of the methodologies of EVs infused with synthetic NP systems with the goal of overcoming their respective shortcomings. For instance, EVs are biogenic with cellular targeting features, but their isolation yield is limited, and their structural and colloidal stability are weak. Whereas, we have decades of experience in the mass production of highly stable synthetic NPs, they lack cellular targeting features. Therefore, the integration of these two systems as a single entity in the field of nanomedicine has gained significant attention. In this review, we emphasized the variety of EVs sources, such as erythrocytes, leukocytes, cancer cells, and stem cells, each providing unique biological benefits. Critical procedures encompassing EV's separation, coating processes, and material integration were examined while addressing the issues, including scalability, membrane stability, and preservation of functionality. Additionally, their promise in customized medicine is analyzed, highlighting their immediate medical applications. This review seeks to elucidate the existing methodologies, their constraints, and prospective advancements in the creation of EV-derived biomimetic NPs for clinical use. This article is categorized under: Nanotechnology Approaches to Biology > Nanoscale Systems in Biology Therapeutic Approaches and Drug Discovery > Nanomedicine for Oncologic Disease.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 40840553 · DOI · PMC12370395
Claudin18.2 as a Promising Therapeutic Target in Gastric Cancer [2]
Abstract reading summary — source excerpts
- Opening excerpt
- Claudin-18.
- Closing excerpt
- What seems to be crucial is that preclinical and clinical data indicate their high efficacy and safety.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (127 words)
Claudin-18.2 (CLDN18.2) is an isoform of a tight junction protein and has emerged as a promising therapeutic target in gastric cancer (GC). CLDN18.2 is responsible for gastric homeostasis and protects epithelial cells from low pH conditions. Interestingly, CLDN18.2 expression is strictly restricted to the stomach, making it an ideal tumor marker. This narrative review presents the characterization and role of claudin 18.2 (CLDN18.2) as a promising biomarker in GC and a target for clinical therapies, more specifically CLDN18.2-targeted drugs and therapies including mABs (e.g., Zolbetuximab, Osemitamab, ZL-1211), bsAB, and CAR-T cell-based immunotherapies. We also summarize numerous ongoing worldwide clinical trials that are evaluating CLDN18.2 as a target for GC treatment. What seems to be crucial is that preclinical and clinical data indicate their high efficacy and safety.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 40862764 · DOI · PMC12384651
Aging microenvironment in osteoarthritis focusing on early-stage alterations and targeted therapies [3]
Abstract reading summary — source excerpts
- Opening excerpt
- Osteoarthritis (OA) is one of the most common degenerative and age-related diseases in joints, which affects 654 million people worldwide.
- Closing excerpt
- Importantly, we evaluate recent therapeutic targets and promising interventions against these components, as well as the challenges and prospects for precise and individualized therapies of OA patients, which we believe would guide the construction of novel combined strategies targeting aging-related factors against OA for better treatments in the future.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (209 words)
Osteoarthritis (OA) is one of the most common degenerative and age-related diseases in joints, which affects 654 million people worldwide. Current therapies could not fundamentally reverse the pathologic process of OA due to the complex pathogenesis. Although OA mechanisms have been investigated on a large scale over the past decade, the OA pathology correlated with aging-associated changes is still largely unrevealed. Therefore, in-depth analysis of the aging microenvironment and aging-related molecular mechanisms in OA may offer additional strategies for clinical prevention and treatment. In this review, we discuss the potential pathogenesis of OA in light of aging-associated changes and summarize three main components of the aging microenvironment of the OA joint: immune homeostatic imbalance, cellular senescence, and stem cell exhaustion, which could be induced by aging and further exacerbate OA progression. Additionally, it is emphasized that immune homeostatic imbalance appears before established OA, which occurs in the early stage and is the therapeutic window of opportunity for better clinical outcomes. Importantly, we evaluate recent therapeutic targets and promising interventions against these components, as well as the challenges and prospects for precise and individualized therapies of OA patients, which we believe would guide the construction of novel combined strategies targeting aging-related factors against OA for better treatments in the future.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 41073390 · DOI · PMC12514018
Leveraging large-scale biobanks for therapeutic target discovery [4]
Abstract reading summary — source excerpts
- Opening excerpt
- Large biobanks, including the Million Veteran Program (MVP), the UK Biobank, and FinnGen, provide genetic association results for more than 1 million individuals for hundreds of phenotypes.
- Closing excerpt
- We make our results publicly available in CIPHER.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (257 words)
Large biobanks, including the Million Veteran Program (MVP), the UK Biobank, and FinnGen, provide genetic association results for more than 1 million individuals for hundreds of phenotypes. To select targets for pharmaceutical development, as well as to improve the understanding of existing targets, we harmonized these studies and performed two-sample Mendelian randomization (MR) on 2,003 phenotypes using genetic variants associated with gene expression (derived from GTEx and eQTLGen) and plasma protein levels (derived from ARIC, Fenland, and deCODE) as proxies of target modulation. We found 69,669 gene-trait pairs with evidence (p ≤ 1.6 × 10-9) for causal effects. From the selected gene-trait pairs, we observed 6,447 genes with strong causal evidence for at least one of 2,003 investigated traits. As expected, being identified as a gene-trait pair in our approach was significantly associated with higher odds of being an approved drug target and indication. We were able to rediscover 9% of approved drug targets in ChEMBL 34. Moreover, identified gene-traits were significantly associated with higher odds of being previously described as a gene-trait pair in OMIM, ClinVar, mouse knockout data, and rare variant burden studies. To enhance the translational potential of the resource, we developed a predictive ranking model trained using approved drug targets described in ChEMBL 34 as well as several different biological annotations. This model was able to accurately predict the odds of a particular significant MR result being developed into an approved drug and its clinical indication (precision-recall area under the receiver operating characteristic curve 0.79). We make our results publicly available in CIPHER.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 41376171 · DOI · PMC12799792
Key reviews and meta-analyses
The most cited review-type papers among PubMed’s best-match results for this heading, listed with bibliographic details only. Follow the links for the full text and access terms.
- Research article Genetics of rheumatoid arthritis contributes to biology and drug discovery [5]
- Research article Prognostic value of TP53 concurrent mutations for EGFR- TKIs and ALK-TKIs based targeted therapy in advanced non-small cell lung cancer: a meta-analysis [6]
- Research article Short-Term Efficacy and Safety of IL-17, IL-12/23, and IL-23 Inhibitors Brodalumab, Secukinumab, Ixekizumab, Ustekinumab, Guselkumab, Tildrakizumab, and Risankizumab for the Treatment of Moderate to Severe Plaque Psoriasis: A Systematic Review and Network Meta-Analysis of Randomized Controlled Trials [7]
- Research article Discordance of immunotherapy response predictive biomarkers between primary lesions and paired metastases in tumours: A systematic review and meta-analysis [8]
- Research article Palliative chemotherapy and targeted therapies for esophageal and gastroesophageal junction cancer [9]
- Research article Pruritus in patients treated with targeted cancer therapies: systematic review and meta-analysis [10]
References
- Sundaram V, Aryal S. Emerging Biomimetic Drug Delivery Nanoparticles Inspired by Extracellular Vesicles. . 2025. PMID 40840553 · DOI · PMC12370395
- Poniewierska-Baran A, Plewa P, Żabicka Z, Pawlik A. Claudin18.2 as a Promising Therapeutic Target in Gastric Cancer. . 2025. PMID 40862764 · DOI · PMC12384651
- Dang Y, Liu Y, Zhang B, Zhang X. Aging microenvironment in osteoarthritis focusing on early-stage alterations and targeted therapies. . 2025. PMID 41073390 · DOI · PMC12514018
- Ferolito BR, Dashti H, Giambartolomei C, Peloso GM, Golden DJ, Gravel-Pucillo K, et al.. Leveraging large-scale biobanks for therapeutic target discovery. . 2026. PMID 41376171 · DOI · PMC12799792
- Okada Y, Wu D, Trynka G, Raj T, Terao C, Ikari K, et al.. Genetics of rheumatoid arthritis contributes to biology and drug discovery. Nature. 2014. PMID 24390342 · DOI · PMC3944098
- Qin K, Hou H, Liang Y, Zhang X. Prognostic value of TP53 concurrent mutations for EGFR- TKIs and ALK-TKIs based targeted therapy in advanced non-small cell lung cancer: a meta-analysis. BMC Cancer. 2020. PMID 32299384 · DOI · PMC7164297
- Bai F, Li GG, Liu Q, Niu X, Li R, Ma H. Short-Term Efficacy and Safety of IL-17, IL-12/23, and IL-23 Inhibitors Brodalumab, Secukinumab, Ixekizumab, Ustekinumab, Guselkumab, Tildrakizumab, and Risankizumab for the Treatment of Moderate to Severe Plaque Psoriasis: A Systematic Review and Network Meta-Analysis of Randomized Controlled Trials. J Immunol Res. 2019. PMID 31583255 · DOI · PMC6754904
- Zou Y, Hu X, Zheng S, Yang A, Li X, Tang H, et al.. Discordance of immunotherapy response predictive biomarkers between primary lesions and paired metastases in tumours: A systematic review and meta-analysis. EBioMedicine. 2021. PMID 33310681 · DOI · PMC7736926
- Janmaat VT, Steyerberg EW, van der Gaast A, Mathijssen RH, Bruno MJ, Peppelenbosch MP, et al.. Palliative chemotherapy and targeted therapies for esophageal and gastroesophageal junction cancer. Cochrane Database Syst Rev. 2017. PMID 29182797 · DOI · PMC6486200
- Ensslin CJ, Rosen AC, Wu S, Lacouture ME. Pruritus in patients treated with targeted cancer therapies: systematic review and meta-analysis. J Am Acad Dermatol. 2013. PMID 23981682 · DOI · PMC4181350
About this article
- Sources
- NLM Medical Subject Headings (definition, synonyms); NCBI PubMed (counts, identifiers); Europe PMC (open-access abstracts and citation counts).
- Verification
- Every reference was resolved against PubMed on 2026-10-31; title, year and DOI matched.
- Review state
- automated source verified; no human expert review has been recorded yet.
- Version
- 1, last updated 2026-10-29.
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Evidence strength
EvidenceHelix's computed view of the study designs behind this topic: how common meta-analyses, randomized trials and reviews are, how recent the literature is, and a plain-language reading. It describes the design mix of the literature. It is not a quality appraisal of any study, and it is not medical advice.