MedPub Date : 2025-05-17DOI: 10.1016/j.medj.2025.100706
Luigi Albano, Daniele Emedoli, Filippo Agnesi, Simone Romeni, Elena Losanno, Laura Toni, Veronica Fossati, Chiara Ciucci, Filippo Gasperotti, Leonardo Cociani, Giovanni Zucco, Edoardo Pompeo, Cinzia Mura, Jacopo Carpaneto, Andrea Tettamanti, Veronica Castelnovo, Jeffrey David Padul, Carlo Mandelli, Lina Raffaella Barzaghi, Federica Alemanno, Heike Caravati, Carla Butera, Ubaldo Del Carro, Antonella Castellano, Andrea Falini, Federica Agosta, Massimo Filippi, Sandro Iannaccone, Pietro Mortini, Silvestro Micera
{"title":"Epidural electrical stimulation facilitates motor recovery in spinal cord injury involving the conus medullaris: A case study.","authors":"Luigi Albano, Daniele Emedoli, Filippo Agnesi, Simone Romeni, Elena Losanno, Laura Toni, Veronica Fossati, Chiara Ciucci, Filippo Gasperotti, Leonardo Cociani, Giovanni Zucco, Edoardo Pompeo, Cinzia Mura, Jacopo Carpaneto, Andrea Tettamanti, Veronica Castelnovo, Jeffrey David Padul, Carlo Mandelli, Lina Raffaella Barzaghi, Federica Alemanno, Heike Caravati, Carla Butera, Ubaldo Del Carro, Antonella Castellano, Andrea Falini, Federica Agosta, Massimo Filippi, Sandro Iannaccone, Pietro Mortini, Silvestro Micera","doi":"10.1016/j.medj.2025.100706","DOIUrl":"https://doi.org/10.1016/j.medj.2025.100706","url":null,"abstract":"<p><strong>Background: </strong>Emerging research increasingly supports that epidural spinal cord electrical stimulation (EES) combined with neurorehabilitation can improve motor recovery in spinal cord injury (SCI) subjects. Patients with lesions involving the medullary cone may be challenging to treat with this approach, probably due to potential peripheral nervous system damage, leaving the open question of whether this large population may benefit from EES.</p><p><strong>Methods: </strong>A T11-T12 SCI patient, with medullary cone involvement, underwent EES implant in a clinical trial (NCT05926843). During three months of testing, we determined optimal stimulation protocols for improving isolated movements and integrated them to reinstate independent walking with a walker.</p><p><strong>Findings: </strong>EES substantially boosted hip flexor, spinal erector, and abdominal muscle contraction, improving the patient's performance in isolated movements. Over three months of combining continuous subthreshold EES with personalized rehabilitation, the patient progressed from being unable to walk to overground ambulation using a two-wheeled walker and bilateral knee and foot orthoses. At the time of hospital discharge, the patient managed to cover 58 m in the 6-min walking test and completed the 10-meter walking test in 40.29 s. Six months after EES implant, the patient was able to walk independently for 1 km with a walker.</p><p><strong>Conclusions: </strong>These results underscore the potential of neurorehabilitation protocols integrating EES also for patients with medullary cone lesions and pave the way for new rehabilitation prospects.</p><p><strong>Funding: </strong>This work was funded by Università Vita-Salute San Raffaele, Boston Scientific Spa, Fondazione Cariplo, Bertarelli Foundation, and the Ministry of University and Research (MUR).</p>","PeriodicalId":29964,"journal":{"name":"Med","volume":" ","pages":"100706"},"PeriodicalIF":12.8,"publicationDate":"2025-05-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144175106","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
MedPub Date : 2025-05-16DOI: 10.1016/j.medj.2025.100704
Ming-Hao Dong, Zhi-Cheng Mei, Luo-Qi Zhou, Michael Heming, Lu-Lu Xu, Yu-Xin Liu, Xiao-Wei Pang, Yun-Hui Chu, Song-Bai Cai, Huan Ye, Ke Shang, Jun Xiao, Gerd Meyer Zu Hörste, Wei Wang, Chuan Qin, Dai-Shi Tian
{"title":"Anti-BCMA CAR-T cell therapy in relapsed/refractory chronic inflammatory demyelinating polyneuropathy.","authors":"Ming-Hao Dong, Zhi-Cheng Mei, Luo-Qi Zhou, Michael Heming, Lu-Lu Xu, Yu-Xin Liu, Xiao-Wei Pang, Yun-Hui Chu, Song-Bai Cai, Huan Ye, Ke Shang, Jun Xiao, Gerd Meyer Zu Hörste, Wei Wang, Chuan Qin, Dai-Shi Tian","doi":"10.1016/j.medj.2025.100704","DOIUrl":"https://doi.org/10.1016/j.medj.2025.100704","url":null,"abstract":"<p><strong>Background: </strong>Chronic inflammatory demyelinating polyneuropathy (CIDP) presents a significant therapeutic challenge, with up to 15% of patients being refractory to first-line treatments.</p><p><strong>Methods: </strong>Anti-B cell maturation antigen chimeric antigen receptor T (CAR-T) cell therapy was applied to two patients with highly relapsed and refractory CIDP, followed by safety and efficacy evaluation. Multi-omics analyses were performed on samples of peripheral blood mononuclear cells (PBMCs) collected before and after infusion.</p><p><strong>Findings: </strong>Both patients had no severe adverse events and achieved drug-free remission within 6 months post-CAR-T therapy. Patient 1 experienced disease recurrence 12 months post infusion following a severe infection of COVID-19, while patient 2 maintained remission over 24 months. Relapse was accompanied by reactivation of pathogenic B cells and recurrence of autoantibodies/peptides targeting axons or myelin. Metabolic reprogramming of B cells characterized by overglycolysis was linked to disease relapse, which could be modulated by regulatory factor X5.</p><p><strong>Conclusion: </strong>This study demonstrates the safety and potential of anti-BCMA CAR-T cell therapy in treating refractory CIDP and provides insights into the molecular mechanisms underlying patient responses (ClinicalTrials.gov: NCT04561557).</p><p><strong>Funding: </strong>Ministry of Science and Technology China Brain Initiative grant STI2030-Major Projects 2022ZD0204700 (to W.W.), National Natural Science Foundation of China grants 82371404 and 82071380 (to D.-S.T.) and 82471353 and 82271341 (to C.Q.), Knowledge Innovation Program of Wuhan Shuguang Project 2022020801020454 (to C.Q.), and Key Research and Development Program of Hubei Provincial Department of Science and Technology 2023BCB148 (to D.-S.T.). The clinical trial was funded by Nanjing IASO Biotechnology Co., Ltd.</p>","PeriodicalId":29964,"journal":{"name":"Med","volume":" ","pages":"100704"},"PeriodicalIF":12.8,"publicationDate":"2025-05-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144162444","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"DiabetesLiver score: A non-invasive algorithm for advanced liver fibrosis and liver-related outcomes in type 2 diabetes mellitus population.","authors":"Chuan Liu, Jie Shen, Jie Li, Zhihui Li, Ming-Hua Zheng, Hua Bian, Xiqiao Zhou, Wenjing Ni, Zhongji Meng, Jiaojian Lv, Yijun Tang, Xuan Liang, Min Li, Taolong Zhou, Heng Wan, Yuping Chen, Yuxia Qi, Yuli Ge, Yan Wang, Wen-Yue Liu, Mingxing Huang, Shanghao Liu, Xiaomei Wang, Mingfeng Xia, Xuefeng Li, Yuehua Wang, Xinjie Li, Xiaoxiong Hu, Yan Wu, Huimin Ying, Jing He, Fengmei Wang, Wei Yan, Huili Wu, Qingge Zhang, Weimin Jiang, Yan Huang, Yudong Zhang, Hongliang He, Xiaofeng Wu, Yuwei Zhang, Ling Li, Terry Cheuk-Fung Yip, Gao-Jun Teng, Xiaolong Qi","doi":"10.1016/j.medj.2025.100700","DOIUrl":"https://doi.org/10.1016/j.medj.2025.100700","url":null,"abstract":"<p><strong>Background: </strong>This study aimed to develop and validate a non-invasive model for screening advanced liver fibrosis and predicting liver-related outcomes in patients with type 2 diabetes mellitus (T2DM).</p><p><strong>Methods: </strong>This study included patients with T2DM from five tertiary hospitals for the development and internal validation of a non-invasive model. Advanced liver fibrosis was defined as a liver stiffness measurement ≥12 kPa. An external validation cohort was obtained from the National Health and Nutrition Examination Survey (NHANES), and the model's predictive performance for hepatocellular carcinoma (HCC) and liver-related mortality was assessed in the UK Biobank.</p><p><strong>Findings: </strong>In total, 28,197 patients with T2DM were enrolled. In the derivation cohort (n = 1,129), waist circumference, alanine aminotransferase, aspartate aminotransferase, platelet count, and albumin were identified as independent risk factors for advanced fibrosis and were fit to develop the \"DiabetesLiver score.\" The area under the curve (AUC) was 0.835 (95% confidence interval [CI]: 0.781-0.890), significantly higher than the AUCs of non-invasive tests (all p < 0.01). It maintained high AUCs of 0.870 and 0.823 in the internal validation (n = 1,000), and NHANES cross-sectional (n = 1,432) cohorts, respectively. A dual cutoff of 2.39 and 3.99 with sensitivity ≥90% and specificity ≥90%, respectively, was used to classify patients into low-, middle-, and high-risk groups. In the UK Biobank cohort (n = 24,636), the high-risk group had an elevated risk of liver-related outcomes.</p><p><strong>Conclusions: </strong>The DiabetesLiver score demonstrated good performance in identifying advanced liver fibrosis and the development of liver-related events in the T2DM population.</p><p><strong>Funding: </strong>National Natural Science Foundation.</p>","PeriodicalId":29964,"journal":{"name":"Med","volume":" ","pages":"100700"},"PeriodicalIF":12.8,"publicationDate":"2025-05-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144128876","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
MedPub Date : 2025-05-12DOI: 10.1016/j.medj.2025.100698
Yi Xu, Fen Tian, Hong Ren, Xian Yu, Xiaoyu Chen, Kun Ye, Feifei Sun, Lin Fang, Yuan Li, Rongxin Ban, Xin Jiang, Chenjing Wang, Yaping Ma, Fu Kuang, Xin Li, Zhigao Zhang, Chaobaihui Ye, Min Hu, Feng He, Chang Shu, Yanfang Zou, Rong Huang, Kai Shen, Guangqun Xing, Yu Cao
{"title":"HRS-5965, a small-molecule factor B inhibitor, in healthy participants and participants with renal insufficiency: A first-in-human, phase 1 trial.","authors":"Yi Xu, Fen Tian, Hong Ren, Xian Yu, Xiaoyu Chen, Kun Ye, Feifei Sun, Lin Fang, Yuan Li, Rongxin Ban, Xin Jiang, Chenjing Wang, Yaping Ma, Fu Kuang, Xin Li, Zhigao Zhang, Chaobaihui Ye, Min Hu, Feng He, Chang Shu, Yanfang Zou, Rong Huang, Kai Shen, Guangqun Xing, Yu Cao","doi":"10.1016/j.medj.2025.100698","DOIUrl":"https://doi.org/10.1016/j.medj.2025.100698","url":null,"abstract":"<p><strong>Background: </strong>HRS-5965 is an oral selective small-molecule inhibitor of complement factor B, a key component of the alternative pathway. This study assessed the safety, tolerability, pharmacokinetics, and pharmacodynamics of HRS-5965 in healthy participants and participants with renal insufficiency.</p><p><strong>Methods: </strong>The first-in-human, phase 1 study consisted of 3 parts (ClinicalTrials.gov: NCT05505955). Part 1 was a single-ascending-dose, randomized, double-blind study with 5 dose groups preset, including a food effect evaluation. Part 2 was a multiple-ascending-dose, randomized, double-blind study with 9 dose groups preset. Part 3 was an open-label, single-dose study on severe renal insufficiency. The primary endpoints were safety and tolerability.</p><p><strong>Findings: </strong>A total of 82 participants were enrolled and received either HRS-5965 or placebo (26 in part 1, 40 in part 2, and 16 in part 3). HRS-5965 was well tolerated. Treatment-emergent adverse events were comparable between the HRS-5965 groups and placebo groups in part 1 (17/20 [85.0%] vs. 6/6 [100.0%]) and part 2 (27/30 [90.0%] vs. 10/10 [100.0%]). No deaths were reported. HRS-5965 was absorbed rapidly, with a median time to reach peak concentration (T<sub>max</sub>) ranging from 0.75 to 1.50 h in fasted states and 2.00 h in fed states. Pharmacokinetics was nonlinear, and food delayed the absorption of HRS-5965 but did not impact the exposure. Alternative pathway activity was inhibited by over 80% with HRS-5965, compared to less than 20% with placebo.</p><p><strong>Conclusion: </strong>HRS-5965 demonstrated favorable safety and robust inhibition of alternative pathway activity, supporting further clinical development.</p><p><strong>Funding: </strong>The study was funded by Jiangsu Hengrui Pharmaceuticals Co., Ltd.</p>","PeriodicalId":29964,"journal":{"name":"Med","volume":" ","pages":"100698"},"PeriodicalIF":12.8,"publicationDate":"2025-05-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144086163","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
MedPub Date : 2025-05-10DOI: 10.1016/j.medj.2025.100702
Pauline Wiertsema, Ya Hwee Tan, John B A G Haanen, Tom T P Seijkens, Inge Jedema
{"title":"Advances in TIL therapy: Expanding the horizons beyond melanoma.","authors":"Pauline Wiertsema, Ya Hwee Tan, John B A G Haanen, Tom T P Seijkens, Inge Jedema","doi":"10.1016/j.medj.2025.100702","DOIUrl":"https://doi.org/10.1016/j.medj.2025.100702","url":null,"abstract":"<p><p>Tumor-infiltrating lymphocyte (TIL) therapy represents a breakthrough in solid tumor treatment, addressing unmet needs for patients with limited options. While its efficacy is established in advanced melanoma, TIL therapy shows early promise in non-small cell lung cancer, breast cancer, gynecological cancers, and head and neck cancers. However, challenges such as reduced T cell infiltration, lower tumor mutational burden (TMB), immunosuppressive tumor microenvironments (TME), and toxicity associated with the TIL therapy regimen hinder its broader application in these patient groups, compared with melanoma. To address these challenges, new approaches focus on the selection of tumor-reactive TIL, optimization of TIL expansion, combination of immune checkpoint inhibitors with TIL therapy to counteract immunosuppressive microenvironments, and genetic modification of TIL to enhance persistence and functionality. Larger clinical trials are essential to validate these innovations and standardize protocols. With continued advancements, TIL therapy has the potential to redefine the treatment landscape for advanced solid cancers.</p>","PeriodicalId":29964,"journal":{"name":"Med","volume":" ","pages":"100702"},"PeriodicalIF":12.8,"publicationDate":"2025-05-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144095076","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Inactivation of TACC2 epigenetically represses CDKN1A and confers sensitivity to CDK inhibitors.","authors":"Zhi-Rui Lin, Tian-Liang Xia, Meng-Yao Wang, Lan-Jun Zhang, Yan-Min Liu, Bo-Yu Yuan, Ai-Jun Zhou, Li Yuan, Jian Zheng, Jin-Xin Bei, Dong-Xin Lin, Mu-Sheng Zeng, Qian Zhong","doi":"10.1016/j.medj.2024.12.002","DOIUrl":"10.1016/j.medj.2024.12.002","url":null,"abstract":"<p><strong>Background: </strong>The genomic landscape of esophageal squamous cell carcinoma (ESCC) has been characterized extensively, but there remains a significant need for actionable targets and effective therapies.</p><p><strong>Methods: </strong>Here, we perform integrative analysis of genome-wide loss of heterozygosity and expression to identify potential tumor suppressor genes. The functions and mechanisms of one of the candidates, TACC2, are then explored both in vitro and in vivo, leading to the proposal of a therapeutic strategy based on the concept of synthetic lethality.</p><p><strong>Findings: </strong>We reveal that the inactivation of TACC2, due to copy number loss and promoter hypermethylation, is associated with poor prognosis in ESCC patients. TACC2 depletion enhances ESCC tumorigenesis and progression, as demonstrated in Tacc2 knockout mouse models and by increased growth abilities of ESCC cells. Mechanistically, TACC2 interacts with components of the NuRD and CoREST co-repressor complexes, including MTA1, MBD3, and HMG20B, in the cytoplasm. TACC2 loss leads to the translocation of these proteins into the nucleus, facilitating the formation of functional NuRD and CoREST complexes and the epigenetic repression of CDKN1A. This repression results in elevated CDK1/2 activation. Furthermore, TACC2-deficient cells and ESCC patient-derived organoids with reduced TACC2 expression show increased sensitivity to CDK inhibitors, particularly dinaciclib, which is currently in a phase III trial. Notably, the combination of TACC2-specific RNAi and dinaciclib in subcutaneous ESCC models significantly impairs tumor growth.</p><p><strong>Conclusions: </strong>The findings suggest a strategy for cancer treatment based on synthetic lethality.</p><p><strong>Funding: </strong>Funded by NKRDP, NSFC, GDIIET, GDBABRF, GDECISTP, and SYSUTP.</p>","PeriodicalId":29964,"journal":{"name":"Med","volume":" ","pages":"100568"},"PeriodicalIF":12.8,"publicationDate":"2025-05-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142967017","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
MedPub Date : 2025-05-09Epub Date: 2024-12-27DOI: 10.1016/j.medj.2024.11.014
Oliver McCallion, Amy R Cross, Matthew O Brook, Conor Hennessy, Ricardo Ferreira, Dominik Trzupek, William R Mulley, Sandeep Kumar, Maria Soares, Ian S Roberts, Peter J Friend, Giovanna Lombardi, Kathryn J Wood, Paul N Harden, Joanna Hester, Fadi Issa
{"title":"Regulatory T cell therapy is associated with distinct immune regulatory lymphocytic infiltrates in kidney transplants.","authors":"Oliver McCallion, Amy R Cross, Matthew O Brook, Conor Hennessy, Ricardo Ferreira, Dominik Trzupek, William R Mulley, Sandeep Kumar, Maria Soares, Ian S Roberts, Peter J Friend, Giovanna Lombardi, Kathryn J Wood, Paul N Harden, Joanna Hester, Fadi Issa","doi":"10.1016/j.medj.2024.11.014","DOIUrl":"10.1016/j.medj.2024.11.014","url":null,"abstract":"<p><strong>Background: </strong>Adoptive transfer of autologous regulatory T cells (Tregs) is a promising therapeutic strategy aimed at enabling immunosuppression minimization following kidney transplantation. In our phase 1 clinical trial of Treg therapy in living donor renal transplantation, the ONE Study (ClinicalTrials.gov: NCT02129881), we observed focal lymphocytic infiltrates in protocol kidney transplant biopsies that are not regularly seen in biopsies of patients receiving standard immunosuppression.</p><p><strong>Methods: </strong>We present 7 years of follow-up data on patients treated with adoptive Treg therapy early post-transplantation who exhibited focal lymphocytic infiltrates on a 9-month protocol biopsy. We phenotyped their adoptively transferred and peripherally circulating Treg compartments using CITE-seq and investigated the focal lymphocytic infiltrates with spatial proteomic and transcriptomic technologies.</p><p><strong>Findings: </strong>Graft survival rates were not significantly different between Treg-treated patients and the control reference group. None of the Treg-treated patients experienced clinical rejection episodes or developed de novo donor-specific antibodies, and three of ten successfully reduced their immunosuppression to tacrolimus monotherapy. All Treg-treated patients who underwent a protocol biopsy 9 months post-transplantation exhibited focal lymphocytic infiltrates. Spatial profiling analysis revealed prominent CD20<sup>+</sup> B cell and regulatory (IKZF2, IL10, PD-L1, TIGIT) signatures within cell-therapy-associated immune infiltrates, distinct from the pro-inflammatory myeloid signature associated with rejection biopsies.</p><p><strong>Conclusions: </strong>We demonstrate for the first time that immune cell infiltrates in transplanted kidneys can occur following adoptive Treg therapy in humans, potentially facilitating within-graft T:B cell interactions that promote local immune regulation.</p><p><strong>Funding: </strong>This work was funded by the 7<sup>th</sup> EU Framework Programme, grant/award no. 260687, and the National Institute for Health Research (NIHR).</p>","PeriodicalId":29964,"journal":{"name":"Med","volume":" ","pages":"100561"},"PeriodicalIF":12.8,"publicationDate":"2025-05-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142898728","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
MedPub Date : 2025-05-09DOI: 10.1016/j.medj.2025.100697
Erika Moore, Shreya A Raghavan
{"title":"ElevateHER: Engineering a new era in women's health.","authors":"Erika Moore, Shreya A Raghavan","doi":"10.1016/j.medj.2025.100697","DOIUrl":"https://doi.org/10.1016/j.medj.2025.100697","url":null,"abstract":"<p><p>Women's health operates in fragmented silos despite encompassing all unique biological and physiological factors affecting women across their lifespan. Consequently, healthcare decisions and technological advances take place under a universal approach, leading to data deficiency and lack of robust models to predict or tailor care for women's health. Engineering innovations hold immense potential to revolutionize health but require broader awareness of their relevance to the spectrum of challenges that fall under the umbrella of women's health. The ElevateHER (Engineering Innovations in Women's Health Discovery) conference convened scientists to identify critical areas for strategic research investment in women's health. Calls for action additionally focused on resource sharing and standardization, streamlining funding opportunities, science communication and outreach, and future workforce development. This perspective highlights these calls for action to engineer a new era in women's health with a cohesive approach that seamlessly integrates women's health into the fabric of overall health.</p>","PeriodicalId":29964,"journal":{"name":"Med","volume":"6 5","pages":"100697"},"PeriodicalIF":12.8,"publicationDate":"2025-05-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144064877","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
MedPub Date : 2025-05-09DOI: 10.1016/j.medj.2025.100695
Harald Hampel, Gang Li, Michelle M Mielke, James E Galvin, Miia Kivipelto, Emiliano Santarnecchi, Claudio Babiloni, Viswanath Devanarayan, Rifky Tkatch, Yan Hu, Ricky Kurzman, Min Cho, Jo Vandercappellen, Yosuke Nakamura, Joanne Bell, Soeren Mattke, Nicola Toschi
{"title":"The impact of real-world evidence in implementing and optimizing Alzheimer's disease care.","authors":"Harald Hampel, Gang Li, Michelle M Mielke, James E Galvin, Miia Kivipelto, Emiliano Santarnecchi, Claudio Babiloni, Viswanath Devanarayan, Rifky Tkatch, Yan Hu, Ricky Kurzman, Min Cho, Jo Vandercappellen, Yosuke Nakamura, Joanne Bell, Soeren Mattke, Nicola Toschi","doi":"10.1016/j.medj.2025.100695","DOIUrl":"https://doi.org/10.1016/j.medj.2025.100695","url":null,"abstract":"<p><p>Real-world evidence (RWE) can complement clinical trials by addressing gaps in how approved anti-amyloid therapies for early Alzheimer's disease (AD) are used in everyday practice. This article outlines strategies to generate RWE that bridge three key challenges in AD care: low detection rates of mild cognitive impairment (MCI), limited data on long-term safety and effectiveness, and a lack of personalized treatment strategies. With MCI detection rates among primary care providers as low as 6%-15%, we propose cost-effective triage tools using electronic health records to enhance early diagnosis and intervention. We also highlight the importance of understanding anti-amyloid therapy outcomes in diverse, real-world populations. Supported by FDA initiatives, pragmatic trials and observational studies using real-world data (RWD) can help develop predictive models that incorporate biomarkers and support precision medicine. These approaches aim to move AD care beyond one-size-fits-all treatment, guiding more tailored, effective strategies for patients.</p>","PeriodicalId":29964,"journal":{"name":"Med","volume":"6 5","pages":"100695"},"PeriodicalIF":12.8,"publicationDate":"2025-05-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144045544","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
MedPub Date : 2025-05-09DOI: 10.1016/j.medj.2025.100699
Catherine M Klapperich, Gordana Vunjak-Novakovic, Jenny Robinson, Renita Horton, Pamela K Kreeger, Morteza Mahmoudi, Samit Shah, Antonina Frolova
{"title":"Bioengineering solutions to improve women's health.","authors":"Catherine M Klapperich, Gordana Vunjak-Novakovic, Jenny Robinson, Renita Horton, Pamela K Kreeger, Morteza Mahmoudi, Samit Shah, Antonina Frolova","doi":"10.1016/j.medj.2025.100699","DOIUrl":"10.1016/j.medj.2025.100699","url":null,"abstract":"<p><p>In recognition of The International Day of Action for Women's Health on May 28<sup>th</sup>, we bring together a collection of Voices highlighting the need for innovative solutions to tackle long-standing women's health challenges from a bioengineering perspective. Many common diseases manifest differently in women, and an insufficient understanding of the underlying mechanisms as well as a lack of tailored treatment strategies continue to impact health outcomes. Bioengineered organoids, organ-on-chip systems, and biomaterials hold promise as tools to dissect pathological mechanisms in reproductive health, cancer, autoimmune diseases, and musculoskeletal health, while tailored diagnostic and therapeutic strategies can enhance the efficacy of clinical interventions. An interdisciplinary approach uniting technological, translational, and clinical research is essential to realize the full potential of bioengineering solutions to improve women's health.</p>","PeriodicalId":29964,"journal":{"name":"Med","volume":"6 5","pages":"100699"},"PeriodicalIF":12.8,"publicationDate":"2025-05-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144001035","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}