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Drug–device combinations in rare diseases: Challenges and opportunities
IF 6.5 2区 医学
Drug Discovery Today Pub Date : 2025-03-22 DOI: 10.1016/j.drudis.2025.104343
E.A. Tataru , M. Dooms , C. Gonzaga-Jauregui , A.M.G. Pasmooij , D.J. O’Connor , A.H. Jonker
{"title":"Drug–device combinations in rare diseases: Challenges and opportunities","authors":"E.A. Tataru ,&nbsp;M. Dooms ,&nbsp;C. Gonzaga-Jauregui ,&nbsp;A.M.G. Pasmooij ,&nbsp;D.J. O’Connor ,&nbsp;A.H. Jonker","doi":"10.1016/j.drudis.2025.104343","DOIUrl":"10.1016/j.drudis.2025.104343","url":null,"abstract":"<div><div>Drug–device combinations (DDCs) are therapeutic products that integrate drugs with medical devices to enhance treatment efficacy and/or safety. These combinations hold significant promise for rare diseases, which affect millions of patients globally, by improving drug delivery, targeting specific organs, and reducing side effects. However, the regulatory framework for DDCs remains complex and lacks specific incentives for rare diseases, unlike orphan drugs. This review examines regulatory approaches and case studies of DDCs in rare diseases, and highlights specific challenges and untapped opportunities. Moreover, the publication discusses recommendations to overcome these challenges through tailored policies and incentives to unlock the potential of DDCs in the context of rare diseases.</div></div>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":"30 4","pages":"Article 104343"},"PeriodicalIF":6.5,"publicationDate":"2025-03-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143690620","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Overview of new indications for novel drugs approved in China between 2018 and 2024.
IF 6.5 2区 医学
Drug Discovery Today Pub Date : 2025-03-21 DOI: 10.1016/j.drudis.2025.104342
Yang Xu, Qixiang Guo, Ziqi Chen, Yunpeng Liu, Yue Yang
{"title":"Overview of new indications for novel drugs approved in China between 2018 and 2024.","authors":"Yang Xu, Qixiang Guo, Ziqi Chen, Yunpeng Liu, Yue Yang","doi":"10.1016/j.drudis.2025.104342","DOIUrl":"https://doi.org/10.1016/j.drudis.2025.104342","url":null,"abstract":"<p><p>Since China's regulatory reforms were initiated in 2015, the development of new indications for novel drugs has become an important trend. Between 2018 and 2024, China's National Medical Products Administration (NMPA) approved 313 new indications for 151 novel drugs. This cross-sectional study comprehensively depicts the landscape of China's new indications for novel drugs, including the characteristics of approvals, quality, and quantity of clinical trial evidence. The quality characteristics of the efficacy evidence for new indications were affected by the treatment areas and conditional approval programs. The efficacy of a novel drug for a new indication can be demonstrated by one pivotal trial or one pivotal trial plus supportive evidence in most cases.</p>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":" ","pages":"104342"},"PeriodicalIF":6.5,"publicationDate":"2025-03-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143690629","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The Six Ds of Exponentials and drug discovery: A path toward reversing Eroom's law.
IF 6.5 2区 医学
Drug Discovery Today Pub Date : 2025-03-21 DOI: 10.1016/j.drudis.2025.104341
Alexander Tropsha, Holli-Joi Martin, Artem Cherkasov
{"title":"The Six Ds of Exponentials and drug discovery: A path toward reversing Eroom's law.","authors":"Alexander Tropsha, Holli-Joi Martin, Artem Cherkasov","doi":"10.1016/j.drudis.2025.104341","DOIUrl":"https://doi.org/10.1016/j.drudis.2025.104341","url":null,"abstract":"<p><p>Many technological sectors underwent recent exponential growth because of digital disruption, a phenomenon Peter Diamantis characterized as the 'Six Ds of Exponentials': digitization, deception, disruption, demonetization, dematerialization, and democratization. In contrast, drug discovery has been marked by rising costs and modest growth, if any, of annual drug approvals. We argue that the exponential growth of drug discovery can be also achieved through digital disruption brought by data expansion, mature artificial intelligence (AI), automation of experiments, public-private partnerships, and open science. We detected the emergence of all 'Six Ds of Exponentials' within modern drug discovery and discuss how each of the 'Six Ds' can further empower the field and forcefully address the societal demand for novel, potent, affordable, and accessible medicines.</p>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":" ","pages":"104341"},"PeriodicalIF":6.5,"publicationDate":"2025-03-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143690632","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Gastroretentive drug delivery systems: A holy grail in oral delivery
IF 6.5 2区 医学
Drug Discovery Today Pub Date : 2025-03-21 DOI: 10.1016/j.drudis.2025.104340
Hossein Omidian
{"title":"Gastroretentive drug delivery systems: A holy grail in oral delivery","authors":"Hossein Omidian","doi":"10.1016/j.drudis.2025.104340","DOIUrl":"10.1016/j.drudis.2025.104340","url":null,"abstract":"<div><div>Gastroretentive drug delivery systems (GRDDSs) offer a promising strategy for enhancing oral drug bioavailability by prolonging gastric residence time and enabling site-specific drug release. This review examines the key materials used in GRDDSs, including polymers for controlled drug release, gas-generating agents for buoyancy, and mucoadhesive components for improved retention and stability. Advances in fabrication techniques, such as 3D printing, spray drying, and nanoparticle encapsulation, have enabled precise modulation of drug release kinetics and retention properties. However, challenges such as inter-subject variability, physiological constraints, and manufacturing scalability remain. Future research will focus on smart materials, multi-drug platforms, and expanding applications to biologics and nutraceuticals, paving the way for more effective and patient-compliant oral drug delivery systems.</div></div>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":"30 4","pages":"Article 104340"},"PeriodicalIF":6.5,"publicationDate":"2025-03-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143690625","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Tubulin targeting agents and their implications in non-cancer disease management
IF 6.5 2区 医学
Drug Discovery Today Pub Date : 2025-03-19 DOI: 10.1016/j.drudis.2025.104338
Samridhi Lal , Timothy J. Snape
{"title":"Tubulin targeting agents and their implications in non-cancer disease management","authors":"Samridhi Lal ,&nbsp;Timothy J. Snape","doi":"10.1016/j.drudis.2025.104338","DOIUrl":"10.1016/j.drudis.2025.104338","url":null,"abstract":"<div><div>Microtubules act as molecular ‘tracks’ for the intracellular transport of accessory proteins, enabling them to assemble into various larger structures, such as spindle fibres formed during the cell cycle. Microtubules provide an organisational framework for the healthy functioning of various cellular processes that work through the process of dynamic instability, driven by the hydrolysis of GTP. In this role, tubulin proteins undergo various modifications, and in doing so modulate various healthy or pathogenic physiological processes within cells. In this review, we provide a detailed update of small molecule chemical agents that interact with tubulin, along with their implications, specifically in non-cancer disease management.</div></div>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":"30 4","pages":"Article 104338"},"PeriodicalIF":6.5,"publicationDate":"2025-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143673132","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Long-term follow-up after authorization of gene therapy: leveraging real-world data.
IF 6.5 2区 医学
Drug Discovery Today Pub Date : 2025-03-19 DOI: 10.1016/j.drudis.2025.104337
Karin de Haart, Keiko Asao, Quazi Ataher, Jamie Geier, Jodie Hillen, Kui Huang, Peter G M Mol, Donna Rivera, Huifen Wang, Hong Yang, Siyan Zhan, Wilhelmina E Hoogendoorn, Lourens T Bloem
{"title":"Long-term follow-up after authorization of gene therapy: leveraging real-world data.","authors":"Karin de Haart, Keiko Asao, Quazi Ataher, Jamie Geier, Jodie Hillen, Kui Huang, Peter G M Mol, Donna Rivera, Huifen Wang, Hong Yang, Siyan Zhan, Wilhelmina E Hoogendoorn, Lourens T Bloem","doi":"10.1016/j.drudis.2025.104337","DOIUrl":"10.1016/j.drudis.2025.104337","url":null,"abstract":"<p><p>Gene therapies are innovative treatments that could revolutionize healthcare. Given their unique mechanisms of action, methods of manufacturing and administration, and the potential for long-lasting impact, gene therapies require long-term follow-up (LTFU) of safety and effectiveness post authorization, often using real-world data (RWD). With over 2000 gene therapies in development, there is a need for knowledge-building regarding LTFU studies. Here, we review regulatory, scientific, and operational requirements and considerations for LTFU studies using RWD from a global perspective. The insights in this review support post-authorization LTFU studies for gene therapies, thereby contributing to their safe and effective use.</p>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":" ","pages":"104337"},"PeriodicalIF":6.5,"publicationDate":"2025-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143673103","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Emerging role of deubiquitinases in modulating cancer chemoresistance
IF 6.5 2区 医学
Drug Discovery Today Pub Date : 2025-03-19 DOI: 10.1016/j.drudis.2025.104339
Sukeerthi Dasharathy , Pranay , Santhosh K. Devadas , Ekta Tripathi , Prashanthi Karyala
{"title":"Emerging role of deubiquitinases in modulating cancer chemoresistance","authors":"Sukeerthi Dasharathy ,&nbsp;Pranay ,&nbsp;Santhosh K. Devadas ,&nbsp;Ekta Tripathi ,&nbsp;Prashanthi Karyala","doi":"10.1016/j.drudis.2025.104339","DOIUrl":"10.1016/j.drudis.2025.104339","url":null,"abstract":"<div><div>Chemotherapy remains a gold standard in cancer treatment by targeting the rapidly dividing cancer cells. However, chemoresistance is a major obstacle to successful cancer treatment, often leading to recurrence, metastasis, and high mortality. Deubiquitinases (DUBs), enzymes that remove ubiquitin and stabilize proteins, have been implicated in chemoresistance and can either promote therapeutic resistance or enhance sensitivity depending on their targets. In this review, we highlight the chemoresistance mechanisms of DUBs in various cancers, including breast, lung, liver, gastrointestinal, colorectal, ovarian, prostate, and blood cancers. Given these mechanisms, the development of DUB inhibitors has gained considerable attention in cancer therapeutics and combination therapies involving these inhibitors show potential to overcome drug resistance and improving treatment outcomes.</div></div>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":"30 4","pages":"Article 104339"},"PeriodicalIF":6.5,"publicationDate":"2025-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143673100","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Artificial intelligence in anti-obesity drug discovery: unlocking next-generation therapeutics
IF 6.5 2区 医学
Drug Discovery Today Pub Date : 2025-03-17 DOI: 10.1016/j.drudis.2025.104333
Amit Gangwal , Antonio Lavecchia
{"title":"Artificial intelligence in anti-obesity drug discovery: unlocking next-generation therapeutics","authors":"Amit Gangwal ,&nbsp;Antonio Lavecchia","doi":"10.1016/j.drudis.2025.104333","DOIUrl":"10.1016/j.drudis.2025.104333","url":null,"abstract":"<div><div>Obesity, a multifactorial disease linked to severe health risks, requires innovative treatments beyond lifestyle changes and current medications. Existing anti-obesity drugs face limitations regarding efficacy, side effects, weight regain and high costs. Artificial intelligence (AI) is emerging as a pivotal tool in drug discovery, expediting the identification of novel drug candidates and optimizing treatment strategies. This review examines AI’s potential in developing next-generation anti-obesity therapeutics, with a focus on glucagon-like peptide-1 receptor agonists (GLP-1 RAs) and their role in discovering anti-obesity peptides. Additionally, it explores integration challenges and offers future perspectives on leveraging AI to reshape the landscape of anti-obesity drug discovery.</div></div>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":"30 4","pages":"Article 104333"},"PeriodicalIF":6.5,"publicationDate":"2025-03-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143661875","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
IRE1α-mediated UPR activation in gastrointestinal cancers: adaptive mechanisms and therapeutic potential.
IF 6.5 2区 医学
Drug Discovery Today Pub Date : 2025-03-15 DOI: 10.1016/j.drudis.2025.104335
Valappan Veetil Soumya, Baby Jisna, Chevookaren Francis Binoy, Thekkekara Devassy Babu
{"title":"IRE1α-mediated UPR activation in gastrointestinal cancers: adaptive mechanisms and therapeutic potential.","authors":"Valappan Veetil Soumya, Baby Jisna, Chevookaren Francis Binoy, Thekkekara Devassy Babu","doi":"10.1016/j.drudis.2025.104335","DOIUrl":"10.1016/j.drudis.2025.104335","url":null,"abstract":"<p><p>The endoplasmic reticulum (ER) plays a crucial part in protein synthesis, folding and quality control. Disruptions in these processes lead to ER stress (ERS) and activate the unfolded protein response (UPR) to restore cellular homeostasis. In gastrointestinal cancers, inositol-requiring enzyme 1α (IRE1α) is a key regulator of the UPR, helping cancer cells adapt to hostile conditions such as hypoxia, oxidative stress and chemotherapy. Elevated IRE1α activity supports tumor survival, progression and metastasis by mitigating ERS-induced apoptosis. However, targeting IRE1α signaling presents a promising therapeutic strategy, potentially impairing cancer cell adaptation to stress and enhancing treatment efficacy. Targeting IRE1α offers promising therapeutic opportunities for gastrointestinal cancer.</p>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":" ","pages":"104335"},"PeriodicalIF":6.5,"publicationDate":"2025-03-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143646727","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Enhancing clinical trial outcome prediction with artificial intelligence: a systematic review
IF 6.5 2区 医学
Drug Discovery Today Pub Date : 2025-03-15 DOI: 10.1016/j.drudis.2025.104332
Long Qian , Xin Lu , Parvez Haris , Jianyong Zhu , Shuo Li , Yingjie Yang
{"title":"Enhancing clinical trial outcome prediction with artificial intelligence: a systematic review","authors":"Long Qian ,&nbsp;Xin Lu ,&nbsp;Parvez Haris ,&nbsp;Jianyong Zhu ,&nbsp;Shuo Li ,&nbsp;Yingjie Yang","doi":"10.1016/j.drudis.2025.104332","DOIUrl":"10.1016/j.drudis.2025.104332","url":null,"abstract":"<div><div>Clinical trials are pivotal in drug development yet fraught with uncertainties and resource-intensive demands. The application of AI models to forecast trial outcomes could mitigate failures and expedite the drug discovery process. This review discusses AI methodologies that impact clinical trial outcomes, focusing on clinical text embedding, trial multimodal learning, and prediction techniques, while addressing practical challenges and opportunities.</div></div>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":"30 4","pages":"Article 104332"},"PeriodicalIF":6.5,"publicationDate":"2025-03-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143646648","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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