bims-carter Biomed News
on CAR-T Therapies
Issue of 2026–06–07
forty-nine papers selected by
Luca Bolliger, lxBio



  1. Front Immunol. 2026 ;17 1826738
      Cell therapies such as chimeric antigen receptor (CAR) T cells and T cell receptor (TCR) T cells marked transformative advances in the treatment of hematologic and solid malignancies, respectively. Thus, adoptive T cell therapy (ACT), in which autologous T cells sourced from the patient constitute the starting immune population, represents a contemporary modality for the treatment of cancers. The need of an autologous cell product poses scientific and logistical challenges that need to be overcome to develop efficacious, scalable and cost-effective ACT. Peripheral blood lymphocytes procured from healthy donors can serve as a starting population for manufacturing a universal allogeneic T cell product offering solutions to both challenges. Recent advances in gene-engineering and -editing technologies have facilitated progress in the development and large-scale manufacturing of allogeneic T cell products. A strategy in development of allogenic ACT is ablation of the TCRαβ/CD3 complex to avoid graft versus host disease mediated by unrelated donor T cells. Mitigating host allogeneic T cells recognition is a complex endeavor that may begin with HLA-I/-II ablation, avoiding recognition and rejection of "non-self" HLA molecules. However, HLA-deficient T cells are susceptible to host NK cell recognition via the "missing-self" response. Here, we discuss immune evasive strategies taken to reduce NK cell mediated rejection of HLA-deficient T cells with particular emphasis on exploitation of HLA-E, a non-classical HLA-I, with regulatory function on NK cell activity. Current progress suggests that off-the-shelf universal T cell products may evolve to become a standard of care treatment options for certain disease indications.
    Keywords:  CAR-T; HLA-E; NK cells; TCR-T; universal T cells
    DOI:  https://doi.org/10.3389/fimmu.2026.1826738
  2. Intractable Rare Dis Res. 2026 May 31. 15(2): 144-155
      Chimeric antigen receptor (CAR) T-cell therapy targeting B cells has emerged as a breakthrough treatment for hematological malignancies and shows promising potential in autoimmune diseases. While selective targeting of B-cell activation and autoantibody production represents an innovative therapeutic approach in autoimmune conditions, clinical responses remain suboptimal in many patients due to incomplete B-cell depletion in tissues and limitations in identifying ideal target antigens. Over the past four years, autologous or allogeneic CAR T-cell therapy has demonstrated remarkable efficacy in autoimmune diseases, achieving rapid and sustained B-cell depletion alongside complete clinical and serological remission. This review examines the current landscape of B cell-targeting CAR T-cell therapy, its therapeutic applications in autoimmune disorders, ongoing translational research, and future developments.
    Keywords:  CAR T-cell therapy; autoimmune disease; immunotherapy
    DOI:  https://doi.org/10.5582/irdr.2025.01057
  3. Cytotherapy. 2026 Mar 11. pii: S1465-3249(26)00737-1. [Epub ahead of print]28(8): 102776
      The development and application of chimeric antigen receptor (CAR) T-cell therapies have changed the treatment landscape of hematologic malignancies. While there are currently FDA approved products for their use in relapsed or refractory B-cell and plasma cell malignancies, ongoing research efforts strive to target difficult to treat antigens and diseases. In this review, we discuss five main challenges involving CAR-T cell products in hematologic malignancies. Additionally, we propose mechanisms to overcome these challenges that monitor adverse effects, improve universal access, enhance manufacturing platforms, and advance commercialization with the goal of optimal patient care.
    Keywords:  CAR T; T cells; cell manufacturing; chimeric antigen receptor; hematologic malignancies; patient access
    DOI:  https://doi.org/10.1016/j.jcyt.2026.102776
  4. Transl Res. 2026 Jun 02. pii: S1931-5244(26)00103-9. [Epub ahead of print]
      Chimeric Antigen Receptor (CAR) T cell therapy has emerged as a transformative modality in cancer immunotherapy. While global efforts have advanced the field, China has played a pivotal role in shaping the development and clinical deployment of CAR-T technologies. We, herein, provide a comprehensive evaluation of China's contributions in structural refinements, the antigen-binding domain and intracellular signaling modules of CAR. Moreover, Chinese preclinical studies have addressed on enhancing T cell infiltration, overcoming immune checkpoint resistance, and remodeling the tumor microenvironment. We, further, review the discovery of novel targets, strategies to minimize on-target/off-tumor toxicity, and innovative manufacturing approaches that have optimized product quality and reduced costs. Clinical translation efforts in China have accelerated rapidly, supported by a robust research infrastructure and regulatory framework, leading to a growing portfolio of CAR-T cell products in trials and practice. Future research should focus on enhancing CAR-T cell efficacy against solid tumors, integrating gene editing technologies, and expanding allogeneic platforms for scalable, off-the-shelf therapies.
    Keywords:  Antigen receptor engineering; Bispecific T cells; Gene editing; Immune checkpoint blockade; Immunotherapy; Solid tumors
    DOI:  https://doi.org/10.1016/j.trsl.2026.06.001
  5. Autoimmun Rev. 2026 Jun 01. pii: S1568-9972(26)00125-4. [Epub ahead of print]25(9): 104111
      Chimeric antigen receptor (CAR) T cells are an advanced cellular therapy produced by genetically modifying T cells to express a synthetic receptor that redirects them to specific cellular targets. Anti-CD19 CAR T cells, approved for use in patients with B cell malignancies, have demonstrated remarkable efficacy in patients with severe refractory autoimmune diseases, such as systemic lupus erythematosus. The purpose of this review is to describe the fundamental aspects of CARs and CAR T cells and the experience of their use in systemic autoimmune diseases. In addition, we discuss the mechanisms through which they achieve clinical effectiveness and present a brief overview of where the field is headed. Even though CAR T cells are a new therapy for autoimmune diseases and their long-term effects are still unknown, they represent a promise for the clinical management of severe autoimmunity and have opened a new window for understanding the pathogenesis of systemic autoimmunity.
    Keywords:  Advanced cellular therapies; CAR T cells; Chimeric antigen receptor; Idiopathic inflammatory myopathies; Immune resetting; Systemic lupus erythematosus; Systemic sclerosis
    DOI:  https://doi.org/10.1016/j.autrev.2026.104111
  6. Expert Rev Hematol. 2026 Jun 03.
       INTRODUCTION: Cytokine release syndrome (CRS) is a potentially life-threatening toxicity associated with chimeric antigen receptor (CAR)-T cell therapy, particularly in hematological malignancies. As CAR-T therapies are increasingly integrated into clinical practice, managing CRS effectively is critical to maximizing therapeutic benefit while minimizing harm.
    AREAS COVERED: This review provides a comprehensive overview of CRS in the context of CAR-T therapy for hematologic cancers. It outlines the current definitions and grading systems, highlights key aspects of CRS pathophysiology, and analyzes the incidence and severity of CRS across pivotal clinical trials in large B-cell lymphoma, acute lymphoblastic leukemia, and multiple myeloma. Standard management approaches, including anti-cytokine therapies and corticosteroids, are discussed alongside emerging strategies such as early intervention and prophylaxis using anakinra and corticosteroids. The review is informed by a structured evaluation of clinical studies and real-world data, reflecting evolving treatment paradigms.
    EXPERT OPINION: CRS remains a major challenge, therefore early recognition, standardized grading, and targeted intervention will improve outcomes. Future efforts should focus on predictive biomarkers, personalized prophylaxis, and next-generation CAR constructs to reduce toxicity. The real-world experience mirrors trial data, reinforcing the reproducibility and scalability of these evolving management strategies.
    Keywords:  CAR-T; Cytokine release syndrome; cellular therapy; hematological malignancies; management
    DOI:  https://doi.org/10.1080/17474086.2026.2684519
  7. Front Immunol. 2026 ;17 1842236
      Chimeric antigen receptor T-cell (CAR-T) therapy has transformed the management of selected hematologic malignancies, particularly relapsed or refractory large B-cell lymphoma, B-cell acute lymphoblastic leukemia, and multiple myeloma. However, currently approved CAR-T strategies largely rely on lineage-associated or differentiation antigens, such as CD19 or BCMA, and therefore do not selectively distinguish malignant B cells from their normal counterparts. This limitation contributes to on-target, off-tumor toxicity, including B-cell aplasia, hypogammaglobulinemia, infectious complications, and prolonged immune dysfunction. In addition, CAR-T therapy remains associated with cytokine release syndrome, immune effector cell-associated neurotoxicity syndrome, immune effector cell-associated hematotoxicity, manufacturing complexity, and variable efficacy across disease entities, particularly in chronic lymphocytic leukemia. Chimeric autoantibody receptor T-cell (CAAR-T) therapy represents a conceptually distinct approach in which engineered T cells are designed to recognize disease-defining immunoglobulin structures, including surface immunoglobulin or B-cell receptor idiotypes. This strategy has been most extensively explored in autoimmune diseases, where CAAR-T cells can selectively eliminate autoreactive B-cell populations while sparing the broader B-cell compartment. Its application in B-cell malignancies remains largely hypothetical, but the biological principle is attractive because many B-cell neoplasms are defined by clonotypic immunoglobulin rearrangements. In this review, we provide a disease-specific and translationally oriented assessment of idiotype-directed CAAR-T therapy in B-cell malignancies. We summarize the current evidence supporting CAAR-T biology, critically evaluate its potential in chronic lymphocytic leukemia, indolent lymphomas, multiple myeloma, and minimal residual disease, and discuss key biological, economic, manufacturing, and regulatory barriers. At present, CAAR-T should not be viewed as a near-term replacement for approved CAR-T therapies, but rather as a hypothesis-generating precision platform requiring rigorous disease-specific validation.
    Keywords:  B-cell malignancies; B-cell receptor; CAAR-T; CAR-T; chronic lymphocytic leukemia; clonal targeting; indolent lymphoma; precision immunotherapy
    DOI:  https://doi.org/10.3389/fimmu.2026.1842236
  8. Zhongguo Shi Yan Xue Ye Xue Za Zhi. 2026 Apr;34(2): 612-616
      Chimeric antigen receptor T (CAR-T) cell therapy has achieved significant success in the field of hematologic oncology. Considering the similarities in the origin and circulatory distribution between hematopoietic tumor cells and autoantigen-specific immune cells, research has commenced to apply CAR-T cell therapy to autoimmune diseases, yielding encouraging preliminary findings. This article reviews the mechanisms of action and clinical research data of CAR-T cells in diseases such as systemic lupus erythematosus, multiple sclerosis, type 1 diabetes, myasthenia gravis, and rheumatoid arthritis in recent years, with the aim of providing a broader perspective for the future application of CAR-T cells in the field of autoimmune diseases.
    Keywords:  chimeric antigen receptor T-cell; immunotherapy; autoimmune diseases
    DOI:  https://doi.org/10.19746/j.cnki.issn1009-2137.2026.02.047
  9. Front Immunol. 2026 ;17 1800292
      Adoptive cell therapy (ACT) has emerged as a transformative strategy in cancer immunotherapy, offering durable clinical benefit in hematologic malignancies and expanding therapeutic potential in solid tumors. However, the translation of ACT to solid malignancies remains constrained by biological, immunological, and logistical challenges. This narrative review provides an evidence based overview of the current clinical landscape of ACT in solid tumors, with a focus on chimeric antigen receptor T cell (CAR-T), tumor-infiltrating lymphocyte (TIL), and T cell receptor-engineered T cell (TCR-T) therapies. We summarize recent clinical trial outcomes, highlight tumor-specific antigen targets, and examine key determinants of therapeutic efficacy across major solid tumor types. The review discusses central obstacles limiting ACT success in solid tumors, including antigen heterogeneity, immune evasion, inadequate T cell trafficking, limited persistence, and functional exhaustion within the immunosuppressive tumor microenvironment. Mechanisms driving treatment resistance, on-target off-tumor toxicity, and immune-related adverse events such as cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome are critically evaluated. We further examine evolving strategies designed to overcome these barriers, including multi-antigen targeting, armored and logic-gated CAR designs, metabolic and cytokine engineering, locoregional delivery approaches, and next-generation manufacturing platforms incorporating allogeneic and gene-edited products. In parallel, the role of biomarkers, tumor microenvironment profiling, and personalized treatment selection is explored as a means to optimize patient stratification and enhance therapeutic outcomes. Advances in translational research, combination immunotherapy, and precision immuno-oncology are positioned as key drivers of the next phase of ACT development. By integrating mechanistic insights with emerging clinical evidence, this review outlines the progress, limitations, and future directions of ACT in solid tumors. It aims to provide a forward-looking framework to guide ongoing research, clinical trial design, and the rational implementation of adoptive cellular immunotherapies in solid malignancies.
    Keywords:  CAR-T cells; T cell receptor therapy; adoptive cell therapy; cancer immunotherapy; immunoregulation; solid tumors; tumor microenvironment; tumor-infiltrating lymphocytes
    DOI:  https://doi.org/10.3389/fimmu.2026.1800292
  10. Zhongguo Shi Yan Xue Ye Xue Za Zhi. 2026 Apr;34(2): 605-611
      Chimeric antigen receptor (CAR) T cell immunotherapy has achieved significant clinical efficacy in lymphatic malignancies, and CAR-T therapy for acute myeloid leukemia (AML) has become a research hotspot in recent years. At present, there is no CAR-T target as specific and effective as CD19 in lymphatic system tumors in AML. However, there are more and more new single target or modified CAR-T therapies for AML.The research on combination targeting (dual target) for AML has improved the long-term control problem of AML that cannot be achieved by single spectrum targeted CAR-T in the past. Both single target and dual target CAR-T can improve efficacy and reduce toxicity. This article reviews the latest research progress of CAR-T cell therapy in AML treatment.
    Keywords:  acute myeloid leukemia; chimeric antigen receptor T cell; targeted therapy
    DOI:  https://doi.org/10.19746/j.cnki.issn1009-2137.2026.02.046
  11. Transplant Cell Ther. 2026 May 29. pii: S2666-6367(26)00417-3. [Epub ahead of print]
      Chimeric antigen receptor T (CAR T) cell therapy has improved outcomes in several hematological malignancies but is associated with immune and hematologic toxicities, including cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), immune effector cell-associated hematotoxicity (ICAHT), and immune effector cell-associated hemophagocytosis-like syndrome (IEC-HS). Although diagnostic and grading criteria have been established, there is no consensus on the optimal use of prediction tools. This review describes the incidence of these toxicities and highlights the utility of available tools for predicting and risk-stratifying patients. These studies focus on clinical scores, immunoassays by analyzing CAR T-cell expansion and imaging biomarkers of tumor burden. Among the former, the EASIX score and its derivatives have been validated for predicting severe CRS and ICANS, whereas the CAR-HEMATOTOX score has been validated for predicting prolonged cytopenia. Emerging prediction models aim to integrate these domains to allow early intervention or de-escalation of immunosuppression therapy.
    Keywords:  Chimeric antigen receptor T-cell therapy; Cytokine release syndrome; Immune effector cell toxicity; Immune effector cell-associated neurotoxicity syndrome
    DOI:  https://doi.org/10.1016/j.jtct.2026.05.027
  12. J Biopharm Stat. 2026 Jun 05. 1-15
      The past decade has seen significant advancements for cell and gene therapies (CGTs). FDA lists more than 30 approved CGTs on their Approved Cellular and Gene Therapy Products website as of August 2024. With the promising treatment effect brought by the currently approved CGTs, there are noticeable limitations, such as, manufacturing delays and durability of response, that encourage continued development in this field. New CGTs can potentially benefit patients by enhancing efficacy or addressing the limitations of currently available therapies. However, the development of new CGTs faces unique challenges as highly efficacious approved first-generation products could be potentially established as the current standard of care (SOC). When the approved CGT products are recommended as the comparator, questions arise, especially, is it feasible to set up a head-to-head comparison between the new and the approved CGT products? What could be the feasible study design options to evaluate the effectiveness of new CGT products? In this article, we introduce the challenges of developing new CGTs in conventional randomized control and single arm trials, as well as discuss some possible strategies for pivotal trials such as hybrid study designs. We believe that more dialogue about this topic among all stakeholders involved in drug development is crucial and hope that this article will contribute towards facilitating such a dialogue.
    Keywords:  CAR T-cell; Pivotal study design; cell and gene therapy; cell therapy; cell-based gene therapy; gene therapy
    DOI:  https://doi.org/10.1080/10543406.2026.2670523
  13. Med. 2026 Jun 04. pii: S2666-6340(26)00154-6. [Epub ahead of print] 101151
      Gene therapies represent a paradigm shift in healthcare, offering the potential for one-time treatment of genetic diseases that require chronic, burdensome management. However, these transformative therapies pose distinctive challenges for traditional cost-effectiveness analysis (CEA). This review examines how CEA of gene therapies is complicated by five distinguishing attributes: rarity, one-time administration, high durable efficacy, substantial upfront costs, and potential financing challenges. We analyze approved gene therapies, ranging in price from $60,000 to over $4 million per treatment, and review US CEAs that assess long-term value, which often find list prices above thresholds but are comparable to other recent therapies. Key methodological challenges include limited data on rare diseases, inappropriate health utility sources, uncertainty about treatment durability, and potential undervaluation of transformative health improvements. Emerging solutions include innovative financing mechanisms, methodological advances, and evolving value frameworks. Developing appropriate economic assessment methods for gene therapies is crucial for ensuring patient access while maintaining innovation incentives.
    DOI:  https://doi.org/10.1016/j.medj.2026.101151
  14. Mol Biol Rep. 2026 Jun 04. pii: 885. [Epub ahead of print]53(1):
       BACKGROUND: Multiple myeloma (MM) remains an aggressive and largely incurable plasma cell malignancy, with relapse common despite therapeutic advances. Although CAR-T cells have transformed the field, their clinical use is constrained by toxicity, complex manufacturing, and limited accessibility. These limitations have accelerated interest in CAR-engineered natural killer (CAR-NK) cells as a safer, more scalable, and potentially off-the-shelf immunotherapeutic platform.
    OBJECTIVE: This review goes beyond a descriptive summary of CAR-NK research in MM by providing an integrated framework that connects antigen targeting, synthetic engineering, tumor microenvironment adaptation, biomarker-guided response monitoring, and translational manufacturing barriers. In doing so, it highlights not only what has been achieved, but also what currently limits clinical implementation and where the field is most likely to advance next.
    METHODS: We performed a comprehensive literature analysis of preclinical and clinical studies on CAR-NK cell therapy in MM, focusing on target selection, persistence-enhancing strategies, immune evasion, biomarker development, combination approaches, and GMP-compatible manufacturing platforms.
    RESULTS: CAR-NK cells offer several advantages over CAR-T therapy, including lower risks of cytokine release syndrome, neurotoxicity, and graft-versus-host disease. Beyond summarizing currently explored targets such as BCMA, CD138, SLAMF7, and GPRC5D, this review identifies the main design principles driving next-generation CAR-NK development: cytokine armoring, genome editing, dual-targeting strategies, and nanotechnology-enabled delivery. Importantly, we also synthesize emerging translational priorities, including predictive biomarkers for patient stratification, serial monitoring of treatment response, and scalable closed-system manufacturing approaches that may determine clinical feasibility.
    CONCLUSION: CAR-NK therapy is evolving from a promising concept into a realistic therapeutic platform for MM. This review contributes a forward-looking translational roadmap by integrating engineering innovation, biomarker-based precision medicine, and manufacturing scalability, thereby defining the key steps needed to move CAR-NK cells toward durable and clinically meaningful impact in refractory MM.
    Keywords:  CAR-NK cells; Hematologic Malignancy; Immunotherapy; Multiple Myeloma; Next-generation Cellular Therapy; Tumor Microenvironment
    DOI:  https://doi.org/10.1007/s11033-026-11990-w
  15. Cytotherapy. 2026 Apr 12. pii: S1465-3249(26)00845-5. [Epub ahead of print]28(8): 102884
       BACKGROUND: Manufacturing of chimeric antigen receptor T-cell (CAR-T) therapies has matured significantly over the past decade, with improvements in automation, closed-system processing, and digital platforms enhancing reliability and scalability. Continuous process innovations and innovative manufacturing models are being explored and implemented, with an eye towards expanding patient access to this therapy and improving the patient journey. Manufacturing advances continue to be met with technical, operational, and regulatory challenges, including cost of goods, supply chain complexity, and capacity constraints, which underscore the need for continued innovation in process optimization and manufacturing models. This paper aims to cover considerations for a range of manufacturing models (including centralized, decentralized, and point-of-care manufacturing), analyze the manufacturing cost breakdown, and lay out opportunities for cost savings.
    RESULTS: Acknowledging the many variables in manufacturing, our analysis shows that, while there can be a big range of cost differentials between centralized and decentralized manufacturing approach, decentralization by itself is unlikely to be the main driver of cost reduction and may come with a decrease in manufacturing efficiency due to reduced scaling benefits. Many opportunities for cost reduction in manufacturing are present regardless of the manufacturing model.
    CONCLUSIONS: While cost reduction is a factor driving the uptake of this therapy, the potential of decentralized manufacturing lies in the improvements of access and patient experience, such as reduced and supply chain complexity associated with cell transport and shortened vein-to-vein times. The Alliance for Regenerative Medicine (ARM) supports all manufacturing models that can expand patient access to cell and gene therapies, while maintaining quality standards and regulatory compliance to ensure patient safety.
    Keywords:  CAR-T; COGs; decentralized manufacturing; manufacturing
    DOI:  https://doi.org/10.1016/j.jcyt.2026.102884
  16. Turk J Haematol. 2026 Jun 03.
       Objective: To evaluate the current status, clinical capacity, and key barriers related to chimeric antigen receptor T-cell (CAR-T) therapy in Türkiye and to inform national policy development.
    Materials and Methods: From June 2023 to March 2024, the Scientific Subcommittee on Cell and Gene Therapies of the Turkish Society of Hematology conducted two nationwide online surveys and held three multidisciplinary meetings with contributions from hematologists, basic scientists, and key stakeholders. Survey findings were integrated with the meeting outputs and recent regulatory developments.
    Results: Surveys indicate a marked gap between the estimated annual need (about 507 patients) and actual access to CAR-T therapy. Between 2019 and March 2024, only 23 patients in Türkiye received CAR-T therapy. Major barriers included high treatment costs, insufficient infrastructure, and a limited number of trained personnel. Expert meetings further highlighted challenges with regulatory pathways, reimbursement, and the need for standardized production and clinical protocols. Despite these limitations, participants demonstrated strong clinical readiness and consensus on the need to improve access.
    Conclusion: The implementation of CAR-T therapy in Türkiye continues to be impeded by structural and economic limitations. It is imperative to develop a strategic national roadmap that encompasses: 1) the establishment of a national academic CAR-T network, 2) the securing of sustainable funding and legal frameworks,3) the implementation of hospital-exemption pathways, 4) the development of a centralized registry, 5) the expansion of structured training programs, and 6) the enhancement of international collaborations. These measures are fundamental for the sustainable integration of this therapy into clinical practice.
    Keywords:  ATMP; CAR-T cell therapy; Türkiye; academic production; hospital exemption; meeting; strategy; survey
    DOI:  https://doi.org/10.4274/tjh.galenos.2026.55822
  17. Magy Onkol. 2026 May 27. 70(2): 151-156
      HER2-targeted therapies have improved outcomes in solid tumors, but their efficacy is often limited by resistance and tumor microenvironmental barriers. Over the past decade, the University of Debrecen Cell and Molecular Therapy Research Group has focused on developing CAR-engineered immune cell strategies to address these challenges. Our work spans advances in CAR-T cell design, including optimization of costimulatory signaling, development of modular targeting systems, and expansion toward off-the-shelf platforms such as CAR-NK cells. Collectively, these efforts highlight the potential of engineered immune cells to overcome key limitations of conventional therapies and support the continued evolution of CAR-based approaches for solid tumors.
  18. Front Immunol. 2026 ;17 1774471
      T cell engagers (TCEs) are a class of T cell-redirecting therapeutics that enhance antitumor immunity by bringing T cells into close proximity with malignant cells. Following their success in hematologic malignancies, TCEs are now being increasingly investigated for the treatment of solid tumors. The recent approval of tarlatamab for small cell lung cancer (SCLC) offers renewed hope in this setting. Nevertheless, clinical efficacy in solid tumors remains limited by immunosuppressive tumor microenvironments (TME), on-target/off-tumor toxicity, and intrinsic or acquired resistance to TCEs. This review summarizes recent advances in TCE development for solid tumors, including refinements in molecular design, biomarker-guided patient selection, and rational combination strategies aimed at overcoming resistance and improving therapeutic outcomes. We also discuss emerging next-generation approaches, such as engager platforms that redirect other immune effector cells. Collectively, these innovations underscore the potential of more precise and effective engager-based therapies for solid tumors.
    Keywords:  T cell engager; combination therapy; solid tumors; trispecific antibody; tumor microenvironment
    DOI:  https://doi.org/10.3389/fimmu.2026.1774471
  19. Sci Adv. 2026 Jun 05. 12(23): eaef5331
      Chimeric antigen receptor (CAR) T cell therapy faces many challenges against solid tumors including T cell exhaustion and poor CAR durability. Here, we show that engaging the CAR T cell endogenous T cell receptor (TCR) using an oncolytic virus enhances CAR T cell functionality, durability, and therapy. Upon combination therapy of solid tumors with CAR T cells and vesicular stomatitis virus (VSV), a subpopulation of antiviral, TCR-primed CAR T cells was generated with enhanced effector functions, altered activation states, and differential gene and protein expression when compared to non-TCR-primed CAR T cells. Single-cell RNA sequencing showed clonal expansion of anti-VSV CAR T cells and enhancement of effector-associated genes with VSV-mediated CAR T cell expansion. CD4 T cells played a pivotal role in the development of these TCR-primed CAR T cells. These results provide a strong rationale both for a novel use of systemic oncolytic virotherapy and for directly exploiting the CAR T cell TCR to fine tune the CAR T cell phenotype and function.
    DOI:  https://doi.org/10.1126/sciadv.aef5331
  20. Crit Rev Oncol Hematol. 2026 Jun 02. pii: S1040-8428(26)00301-X. [Epub ahead of print]225 105414
      Chimeric antigen receptor macrophages (CAR-Ms) are an emerging myeloid cell therapy designed to exploit the inherent plasticity of macrophages in solid tumors. By integrating antigen-specific recognition with macrophage-mediated phagocytosis, antigen presentation, and local immune remodeling, CAR-Ms coordinate tumor killing with reprogramming of the tumor microenvironment. Advances in CAR design, intracellular signaling, cellular platforms, and gene delivery have enabled preclinical evaluation in immunocompetent models and early clinical testing. CAR-Ms can enhance T cell and NK cell infiltration, promote antigen spreading, and sensitize tumors to immune checkpoint blockade. Early clinical studies, including HER2-targeted CT-0508, demonstrate feasible manufacturing, acceptable safety, tumor infiltration, and immune remodeling, though objective efficacy remains limited. Major challenges include maintaining antitumor polarization, persistence, scalable manufacturing, target selection, and safety control. Continued optimization of CAR-M design and exploitation of myeloid plasticity will be critical to realizing their potential as solid tumor immunotherapy platforms.
    Keywords:  CAR-macrophage; cellular immunotherapy; chimeric antigen receptor; macrophage engineering; solid tumors; tumor microenvironment
    DOI:  https://doi.org/10.1016/j.critrevonc.2026.105414
  21. Res Sq. 2026 May 21. pii: rs.3.rs-9610745. [Epub ahead of print]
      Background : B7-H3-directed chimeric antigen receptor (CAR) T cell therapy has demonstrated clinical safety and antitumor activity in pediatric brain tumors (pBTs) but lacks durable responses. Although preclinical studies show efficacy, the CAR designs that best support sustained function in immunosuppressive tumor microenvironments (TMEs) remain unclear. Group 3 medulloblastoma (G3MB) is a lethal pBT with poor responsiveness to immunotherapy. Within the TME, myeloid cells dominate the immune landscape and regulate T cell function through innate immune pathways, including the Toll-like receptor 7/8 (TLR7/8) axis. While TLR7/8 agonists activate antitumor myeloid programs, their integration with CAR T-cell therapy has not been explored. Therefore, we hypothesize that CAR architecture and TLR7/8-mediated myeloid activation cooperatively govern CAR T-cell function and subsequent therapeutic outcomes in G3MB. Methods : B7-H3 CARs incorporating CD28, 4-1BB, or dual CD28/4-1BB co-stimulation were evaluated in vitro and in orthotopic G3MB models. Resiquimod was formulated in a brain-penetrant poly (2-oxazoline) nanoparticle (ResiPOx) to activate TLR7/8-expressing myeloid cells. T cell and myeloid states were assessed by flow cytometry, bulk, and single-cell RNA sequencing. Results : CAR designs showed similar tumor control in immunodeficient hosts but diverged in immunocompetent models, where dual-costimulatory CAR T-cells demonstrated superior cytotoxicity and persistence. The optimal CAR induced sustained CAR T-cell cycling and TAM reprogramming while downregulating TLR7/8 in dominant myeloid clusters. ResiPOx enhanced CAR T-cell efficacy by activating myeloid cells and reducing suppressive populations. Conclusions : Optimized CAR design combined with TLR7/8-mediated myeloid reprogramming enhances T cell activity, supporting TME-guided immunotherapy for G3MB.
    DOI:  https://doi.org/10.21203/rs.3.rs-9610745/v1
  22. Biotechnol Adv. 2026 Jun 01. pii: S0734-9750(26)00148-5. [Epub ahead of print]91 108942
      Immunotherapy has transformed the landscape of cancer treatment, driving sustained innovation and investment across the biotechnology sector. Among the most prominent advances, CAR-T cell therapy and cancer nanovaccines have emerged as powerful and potentially complementary strategies with the capacity to reshape clinical practice. Despite their promise, each approach presents distinct strengths and limitations, as demonstrated across preclinical and clinical studies. This review provides a comparative analysis of these platforms, examining their underlying mechanisms, technological features, and recent developments aimed at improving therapeutic efficacy. Particular attention is given to the key challenges that continue to limit their broader clinical translation, including barriers associated with solid tumors, safety, scalability, and the durability of immune responses. We further integrate these discussions into translational clinical decision-making insights intended to support context-dependent patient stratification, therapeutic selection, and rational design of combination immunotherapies. By integrating these perspectives, this work offers an updated overview of CAR-T cell therapies and nanovaccines, emphasizing convergent and divergent features while outlining strategic directions to guide future research and translational development.
    Keywords:  CAR-T cells; Cancer; Cancer Nanovaccines; Immunotherapy
    DOI:  https://doi.org/10.1016/j.biotechadv.2026.108942
  23. Support Care Cancer. 2026 Jun 06. pii: 621. [Epub ahead of print]34(7):
      Chimeric antigen receptor T-cell (CAR-T) therapy has emerged as a highly effective treatment for refractory and relapsed hematologic malignancies. Despite its clinical benefits, CAR-T therapy is associated with a unique toxicity profile, including cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), and prolonged immunosuppression. Oral complications in this population remain underreported. This retrospective case series was aimed at describing oral manifestations and dental management in patients undergoing CAR-T therapy at a tertiary referral center. Medical and dental records of ten patients treated with CAR-T therapy between 2024 and 2025 were reviewed. Demographic data, underlying hematologic diagnoses, CAR-T-related toxicities, and oral findings were analyzed descriptively. The main oral manifestations observed were xerostomia, opportunistic infections, dental pain, and dysgeusia. Oral care focused on symptom control, prevention of secondary infections, and supportive oral management during periods of severe immunosuppression. These findings suggest that early dental involvement may contribute to supportive care strategies, particularly for the detection and management of opportunistic infections and dental infectious foci in neutropenic patients. Standardized oral assessment protocols and preventive dental measures may contribute to supportive clinical management and symptom monitoring in this patient population.
    Keywords:  CAR-T cell therapy; Hematologic malignancies; Immunotherapy; Oral complications; Supportive oral care
    DOI:  https://doi.org/10.1007/s00520-026-10824-6
  24. ESMO Open. 2026 Jun 04. pii: S2059-7029(26)01709-6. [Epub ahead of print]11(6): 107767
       BACKGROUND: Traditional phase I designs aim to identify the maximum tolerated dose, assuming higher doses increase efficacy, which may not hold in chimeric antigen receptor (CAR)-T cell therapy. We present DOSET (Dose Optimization with Simultaneous Efficacy and Toxicity) evaluation, a novel adaptive design jointly modeling safety and preliminary efficacy to identify the recommended dose, minimizing patient exposure to subtherapeutic or toxic doses, informed by a phase I CAR T lymphoma trial with a small sample.
    MATERIALS AND METHODS: DOSET is a two-part Bayesian adaptive design. Part 1 employs the continual reassessment method (CRM) with an initial 2 + 2 + 2 escalation across three dose levels to identify tolerable doses, allowing early stopping for excessive toxicity. Part 2 randomly assigns patients across selected doses, with ongoing CRM updates allowing dose adjustments as needed. Activity is evaluated concurrently with a Bayesian interim futility framework. Final dose selection is based on joint tolerability and activity. Simulations compared dose selection accuracy, patient allocation, and safety with the Bayesian optimal interval phase I/II design (BOIN12).
    RESULTS: DOSET effectively selects active and tolerable doses, allocates patients efficiently, and reliably stops when all doses are excessively toxic. Compared with BOIN12, it improves dose selection without compromising safety, maintains or enhances early stopping for futile or toxic doses, and provides greater flexibility.
    CONCLUSIONS: DOSET integrates dose escalation and optimization in a seamless framework, leveraging safety and efficacy data. Despite small-sample sizes, it offers an efficient and flexible approach to improve early-phase CAR T cell trials and applies broadly to non-monotonic dose-response settings, including targeted therapies and immunotherapies.
    Keywords:  Bayesian adaptive design; CAR-T cell therapy; dose finding; dose optimization; joint efficacy–toxicity evaluation; phase I trial; seamless design
    DOI:  https://doi.org/10.1016/j.esmoop.2026.107767
  25. J Biomed Sci. 2026 Jun 04. pii: 60. [Epub ahead of print]33(1):
      Chimeric antigen receptor (CAR)-T cells have demonstrated remarkable efficacy in several hematologic malignancies; however, their application in myeloid malignancies such as acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) remains limited, with no FDA-approved products to date. This limited progress largely reflects both efficacy challenges and safety concerns. CAR-T cells demonstrate poor trafficking and persistence within the bone marrow, limited activity against leukemia stem cells (LSCs), and reliance on antigens such as CD33 and CD123 that are also expressed on normal hematopoietic stem and progenitor cells, resulting in significant on-target off-tumor toxicity. Given these limitations, attention has increasingly shifted toward alternative CAR-engineered immune cells, including CAR-natural killer (CAR-NK) cells, CAR-invariant natural killer T (CAR-NKT) cells, and CAR-macrophages (CAR-Ms). These platforms offer unique advantages, such as intrinsic antitumor activity, distinct trafficking properties, reduced risk of graft-versus-host disease (GvHD), and potentially safer antigen recognition profiles, that may help overcome barriers faced by CAR-T cells. In this review, we highlight the challenges of applying conventional CAR-T cells to myeloid malignancies, examine emerging alternative CAR-cell platforms, and discuss how their unique biology and engineering strategies may provide safer, more effective, and more accessible therapeutic options for patients with these difficult-to-treat cancers.
    Keywords:  Acute myeloid leukemia (AML); Bone marrow homing; CAR-NK; CAR-NKT; CAR-T; CAR-macrophage; Chimeric antigen receptor (CAR); Leukemia stem cells (LSCs); Myelodysplastic syndromes (MDS); Myeloid malignancies; On-target off-tumor effect
    DOI:  https://doi.org/10.1186/s12929-026-01265-8
  26. J Immunother Cancer. 2026 Jun 03. pii: e014693. [Epub ahead of print]14(6):
       BACKGROUND: While chimeric antigen receptor (CAR) T cells have achieved encouraging remission rates in hematological malignancies, they have demonstrated limited success in treating glioblastoma (GBM), particularly due to high intratumoral and intertumorous heterogeneity. In this study, we identified a consistently expressed target antigen, podoplanin (PDPN), and evaluated the potential of a PDPN-CAR T cell and GD2-CAR T cell blend to target heterogeneous GBM.
    METHODS: Target antigen screening included clinical samples, cell lines and healthy tissues, as well as public RNA sequencing datasets. The anti-tumor function of CAR T cells was examined in co-culture experiments with GBM cell lines and patient-derived organoids (PDOs), and in vivo after locoregional delivery in orthotopic xenograft models.
    RESULTS: CAR T cells demonstrated strong anti-tumor activity against several cell lines and PDOs from multiple patients. PDPN and GD2 were expressed in all PDOs, and regardless of the antigen expression pattern, the CAR T cell blend induced significantly higher apoptosis levels in organoids compared with single-antigen targeting CAR T cells. In vivo, we observed efficient tumor regression after locoregional administration of monospecific CAR T cells. While heterogeneous orthotopic tumors eventually relapsed in these groups, therapy with the CAR T cell blend significantly increased overall survival and even achieved a cure in the majority of mice.
    CONCLUSION: This novel PDPN/GD2 CAR T cell blend demonstrated robust efficacy in advanced preclinical GBM models, suggesting its potential to treat heterogeneous GBM and address limitations associated with single-antigen CAR T cell therapies.
    Keywords:  Central Nervous System Cancer; Chimeric antigen receptor - CAR; Immunotherapy; Intrathecal
    DOI:  https://doi.org/10.1136/jitc-2025-014693
  27. J Clin Rheumatol. 2026 May 27.
       OBJECTIVES: This study aimed to systematically summarize available clinical data on the use of chimeric antigen receptor (CAR) T-cell therapy in autoimmune diseases (ADs).
    METHODS: A systematic review was conducted using PubMed and other literature databases to identify publications on CAR-T therapy in ADs published since 2020. Eligible studies included single-case reports and case series from clinical trials, while studies involving duplicated patient populations were excluded in accordance with PRISMA guidelines. Extractable patient-level data were analyzed for key clinical outcomes.
    RESULTS: A total of 32 studies encompassing 124 patients with ADs who received CAR-T therapy were included. The primary diseases treated were systemic lupus erythematosus (SLE) and myasthenia gravis (MG), with smaller numbers of patients reported in other ADs. In SLE trials, 83% achieved clinical remission or low disease activity following CAR-T therapy, within the first few months following infusion. In MG trials, minimal symptom expression was achieved in 73%. CAR-T disappearance and B-cell reconstitution generally occurred within the first few months following infusion and were predominantly of a naïve phenotype. Mild cytokine release syndrome was the most common adverse event; serious adverse events were reported in 4.8% of patients without treatment-related deaths.
    CONCLUSION: Preliminary clinical evidence suggests that CAR-T therapy is associated with meaningful clinical response in selected ADs. Larger studies with standardized outcome measures and longer follow-up are needed to better define its safety and efficacy.
    Keywords:  CAR-T; SLE; autoimmune disease; cellular therapy; clinical trial; immune reprogramming; immunotherapy
    DOI:  https://doi.org/10.1097/RHU.0000000000002354
  28. Front Immunol. 2026 ;17 1875877
      
    Keywords:  CAR-T cell; autoantibodies; autoimmunity; immune tolerance; targeted immunotherapy
    DOI:  https://doi.org/10.3389/fimmu.2026.1875877
  29. JCO Glob Oncol. 2026 Jun;12(6): e2600050
    St Jude Global, the European Group for Blood and Marrow Transplantation (EBMT) PDWP and the International BFM Study Group (I-BFM)
       PURPOSE: Chimeric antigen receptor T-cell therapy (CAR-T) has revolutionized the treatment of B-cell precursor ALL (B-ALL), but its global availability is limited. This study assessed current access and barriers to CAR-T CD19 therapy for children across Europe.
    MATERIALS AND METHODS: A country questionnaire developed by the European Group for Blood and Marrow Transplantation Pediatric Diseases Working Party, St Jude Children's Research Hospital, and IBFM assessed current access to advanced therapies for B-ALL in Europe using Qualtrics software.
    RESULTS: Data from 36 WHO-defined European countries (27 high-income, nine upper-middle-income) observed a median of five pediatric hematology-oncology (PHO) centers per country (0.56 PHO centers/1 million inhabitants, range, 0.05-1.83). Hematopoietic stem-cell transplantation (HSCT) facilities were available in 89% of countries (32/36). CAR-T CD19 therapy was available in 72% of countries; however, 25/36 (69%) countries lacked clinical trials or international collaborations for pediatric CAR-T CD19 therapy. Most countries accepted foreign patients, but referrals remained limited, with 1-2 foreign patients treated annually per country. Eighteen countries expressed interest in a referral network, but only six had established mechanisms for domestic or international referrals.
    CONCLUSION: Substantial disparities exist in access to advanced therapies for pediatric B-ALL across Europe. Although CAR-T CD19 therapy is available in most countries, gaps in clinical trials, collaborations, and referral systems limit equitable access. Efforts to improve infrastructure and establish referral networks are essential to enhance care for patients with pediatric B-ALL.
    DOI:  https://doi.org/10.1200/GO-26-00050
  30. Pediatr Res. 2026 Jun 03.
      The United States Food and Drug Administration has approved 44 cell and gene therapies as of May 2025, including 14 for pediatric use (excluding cord blood). This signifies an advancement in the treatment of diseases and conditions that, as of yet, cannot be effectively managed with available small-molecule drugs and therapeutic proteins. This review explores the pharmacokinetic and first-in-human dosing considerations unique to cell and gene therapies. For in vivo gene therapies, development considerations include vector shedding and biodistribution. Traditional pharmacokinetic principles do not apply to ex vivo gene therapies, given the modification of cells outside the body before re-infusion. Cellular kinetics such as expansion and persistence are relevant in cell therapies like Chimeric Antigen Receptor T cells and Tumor Infiltrating Lymphocytes. A common trend of cell and gene therapy development is the reliance on preclinical models to inform safety, efficacy, and dose selection, with first-in-human dosing further guided by data from related therapies. Rare disease and pediatric drug development present additional challenges due to small patient populations, emphasizing the importance of preclinical evidence and safety considerations. The article outlines the key considerations for cell and gene therapies that are vital for expanding their use. IMPACT: This article provides a synthesis of pediatric rare disease clinical pharmacology considerations for recently approved cell and gene therapies, highlighting how advanced therapeutic products compare with traditional pharmacokinetic and pharmacodynamic frameworks used for small-molecule drugs. The development of advanced therapeutics depends on preclinical modeling, first-in-human dosing strategies, clinical trial design, and regulatory guidance for pediatric use, emphasizing the need for a practical primer on emerging advanced therapeutic products for researchers.
    DOI:  https://doi.org/10.1038/s41390-026-05138-6
  31. Front Cell Dev Biol. 2026 ;14 1827716
      T cell exhaustion is a central framework for explaining why antitumor T cell responses often fail despite persistent antigen exposure and immune infiltration. Rather than a single dysfunctional endpoint, exhaustion is increasingly understood as a structured and dynamic continuum of antigen-experienced CD8+ T cell states that differ in proliferative capacity, effector potential, epigenetic constraint, metabolic fitness, and spatial distribution within tumors. This view has major therapeutic implications because clinically relevant interventions can remodel exhausted-state composition and function without fully restoring a non-exhausted identity. In this review, we examine the organization of exhausted T cell states from progenitor-like to terminal compartments and discuss how TOX-linked survival programs, epigenetic fixation, and tumor-imposed metabolic and spatial constraints stabilize exhausted fate under chronic stimulation. We highlight the role of progenitor exhausted T cells in sustaining therapeutic responsiveness, explain why reinvigoration after checkpoint blockade is often partial rather than transformative, and evaluate emerging strategies to modulate exhaustion dynamics, including combination immunotherapy and engineered control systems in CAR T cells. Together, these concepts support a shift from viewing exhaustion as a binary defect to understanding it as a constrained state system that can be measured, preserved, and selectively redirected. Defining which exhausted states remain productively controllable, and under what conditions, will be essential for developing more durable and mechanistically informed cancer immunotherapies.
    Keywords:  cancer immunotherapy; immune checkpoint blockade; progenitor exhausted T cells; tumor microenvironment; t cell exhaustion
    DOI:  https://doi.org/10.3389/fcell.2026.1827716
  32. Cancer Immunol Immunother. 2026 Jun 05.
      Chimeric antigen receptor (CAR)-T cells have achieved remarkable success against hematologic malignancies; however, their application is associated with risks such as cytokine release syndrome (CRS) and neurotoxicity. In contrast, CAR-NK cells not only avoid these toxicities but also retain the natural cytotoxic activity of NK cells, demonstrating great potential for cancer immunotherapy.Our previous study demonstrated the potent efficacy of folate receptor alpha (FOLR1)-targeted CAR-NK92 cells against ovarian cancer (OC). Nevertheless, the application of CAR-NK therapy in solid tumors, including OC, still faces challenges such as tumor antigen heterogeneity and the immunosuppressive tumor microenvironment (ITME). A key mediator of ITME is programmed death-ligand 1 (PD-L1), which not only correlates with poor prognosis in OC but also drives T cell exhaustion via the PD-1/PD-L1 axis. Moreover, its inducible upregulation under NK cell-based therapy supports PD-L1 as a viable therapeutic target in OC.To enhance the therapeutic potential of CAR technology for OC, we engineered two novel third-generation bispecific CAR-NK92 cells based on our prior FOLR1-CAR design. These constructs-Tandem PD-L1/FOLR1-CAR (Tan-CAR1) NK92 and Tandem FOLR1/PD-L1-CAR (Tan-CAR2) NK92-were designed to simultaneously target FOLR1 and PD-L1. Notably, we confirmed that PD-L1 expression was significantly upregulated in the co-culture supernatant of effector and target cells. In vitro, Tan-CAR2 NK92 cells exhibited markedly superior cytotoxicity against FOLR1highPD-L1low OC cells and enhanced degranulation compared with FOLR1-CAR NK92 cells. In xenograft mouse models, Tan-CAR2 NK92 cells showed effective tumor infiltration and induced significantly greater tumor regression than both FOLR1-CAR NK92 and PD-L1-CAR NK92 groups. In conclusion, the tandem bispecific CAR-NK92 cells developed in this study represent a meaningful advance in immunotherapy. Their dual mechanism-broader antigen recognition and enhanced immune infiltration-effectively addresses two major therapeutic barriers in OC, offering a promising strategy for treating refractory cases.
    Keywords:  FOLR1; Ovarian cancer; PD-L1; Tandem-CAR NK92; Tumor immune microenvironment
    DOI:  https://doi.org/10.1007/s00262-026-04443-7
  33. Inflamm Regen. 2026 Jun 05.
      Disease models are used to evaluate drug candidates, and compounds that are highly effective in vivo models have traditionally been prioritized for development. While conventional 'gold standard' animal models have been central to autoimmune drug discovery, there is increasing recognition that addressing unmet medical needs requires models capable of capturing patient pathophysiology beyond the scope of these classical systems. Accordingly, models that reflect human disease mechanisms not reproducible in conventional animals are becoming increasingly important. Humanized mice are immunodeficient mice transplanted with human immune cells, hepatocytes, thymic tissue, and other components to create a human-like biological environment that cannot be replicated in wild-type mice. Research on humanized mice has advanced through efforts to reconstitute a diverse human immune system in mice, together with accumulating knowledge of patient-specific factors such as autoantibodies and autoreactive T cells. Additionally, single-cell analyses and human tissue studies are underway to recreate the human-specific disease phenomena in humanized mice. In this review, immune-system-humanized mice are used to provide a comprehensive overview of recent advances in immune-system-humanized mouse technologies, their applications to immune-related disease models, and their current utilization in drug discovery research.
    Keywords:  Autoimmune diseases; Disease models; Humanized mice; Immune reconstitution
    DOI:  https://doi.org/10.1186/s41232-026-00427-3
  34. Bone Marrow Transplant. 2026 Jun 04.
      Autologous chimeric antigen receptor (CAR)-T cell therapy is effective in relapsed or refractory B-cell acute lymphoblastic leukemia (R/R B-ALL), but its clinical application is restricted by manufacturing failures, high costs, and limited accessibility. Gene-edited allogeneic CAR-T cells offer an off-the-shelf alternative, yet concerns remain regarding genomic instability and graft-versus-host disease (GVHD). We conducted a phase 1 study to assess the safety and efficacy of ThisCAR-T, a non-gene-edited, CD19-directed allogeneic CAR-T cell product, in patients with R/R B-ALL. Eleven patients were treated with escalating doses of ThisCAR-T at 1 × 106, 3 × 106, and 5 × 106 cells/kg. One patient withdrew after infusion. Among the remaining ten patients, eight developed cytokine release syndrome, including seven with grade 1-2 and one with grade 3. Immune effector cell-associated neurotoxicity syndrome occurred in two patients (grade 1 and grade 4, respectively). No GVHD was observed. At day 28, 8 of 9 evaluable patients achieved complete remission (CR) or CR with incomplete hematologic recovery (CRi). At a median follow-up of 31 months, the 1-year progression-free survival and overall survival rates were 30% and 40%, respectively. ThisCAR-T demonstrated a favorable safety and promising antileukemic activity, supporting its potential as a novel therapy for R/R B-ALL.
    DOI:  https://doi.org/10.1038/s41409-026-02929-7
  35. Front Immunol. 2026 ;17 1813730
      Chimeric antigen receptor (CAR) T-cell therapy has revolutionized the treatment of hematologic cancers but encounters challenges, including severe treatment-related toxicities, a highly suppressive tumor microenvironment (TME), limited long-term persistence, and poor trafficking/infiltration into solid tumors. This review outlines recent genetic engineering strategies to address these issues and enhance the safety, durability, and efficacy of CAR-T cell therapy. To reduce cytokine release syndrome and neurotoxicity, methods such as affinity-tuned and humanized scFvs, hinge/TM optimization, and ITAM calibration have been developed, along with programmable "switch-off" and "switch-on" systems that include suicide genes, antibody-bridging switches, and optogenetic or hypoxia-gated circuits. TME remodeling strategies utilize nanomaterials for targeted cytokine delivery, cell-surface "backpack" systems, and engineered oncolytic viruses that release cytokines or checkpoint-blocking agents. For durability and resistance to exhaustion, precise genome engineering techniques, including CRISPR-based editing and multiplexed shRNA platforms, were employed to target inhibitory receptors and exhaustion-driving transcriptional programs. Additionally, chemokine-receptor engineering and local biomaterial-based delivery systems are discussed as ways to enhance CAR-T trafficking and intratumoral persistence. These innovations collectively point toward integrated, patient-specific CAR-T platforms that incorporate safety controls, metabolic and transcriptional flexibility, and enhanced trafficking through the TME to broaden clinical use.
    Keywords:  CAR-T cell therapy; chemokine trafficking; chimeric antigen receptor-T cells; cytokine release syndrome; genome engineering; immune checkpoint regulation; immunotherapy; tumor microenvironment
    DOI:  https://doi.org/10.3389/fimmu.2026.1813730
  36. J Immunother Cancer. 2026 Jun 03. pii: e014840. [Epub ahead of print]14(6):
      Natural killer (NK) cells are critical effectors of antitumor immunity, however their cytotoxic function is frequently impaired within the tumor microenvironment (TME). Tumor-associated macrophages (TAMs), the most abundant immune stromal population in solid tumors, play a central role in shaping NK cell responses through a broad range of mechanisms, including receptor-ligand interactions, immunosuppressive cytokine signaling, metabolic reprogramming, and engagement of immune checkpoint pathways. Here, we review current insights into the bidirectional crosstalk between TAMs and NK cells and discuss therapeutic strategies aimed at restoring NK cell activity by targeting TAMs. These include macrophage depletion and reprogramming approaches, modulation of metabolic and transcriptional pathways, and interventions targeting cytokine networks and immune checkpoints. We further examine emerging strategies that reshape the TME to enhance NK-macrophage cooperation, such as induction of inflammatory cell death, modulation of innate immune signaling pathways, and the development of synthetic NK cell engagers. In addition, we highlight the impact of macrophage ontogeny, tissue residency, and spatial organization on NK cell function, emphasizing how distinct microanatomical niches within the TME regulate immune cell interactions and influence therapeutic responses. Finally, we summarize translational advances and ongoing clinical efforts aimed at integrating TAM-targeted therapies with NK cell-based approaches. Collectively, these findings provide a conceptual and mechanistic framework for the rational design of combination immunotherapies that leverage macrophage-NK cell interactions to enhance innate immune responses and improve cancer treatment outcomes.
    Keywords:  Immunosuppression; Immunotherapy; Macrophage; NK Cell Lectin-Like Receptor Subfamily K; Tumor microenvironment - TME
    DOI:  https://doi.org/10.1136/jitc-2026-014840
  37. Nat Rev Drug Discov. 2026 Jun 03.
      mRNA technology, which has enabled the rapid development of vaccines for infectious diseases, also holds great promise for a new generation of therapies for a host of rare and common diseases. A decade of clinical trials are beginning to clarify the key barriers to unlock the transformative potential of mRNA drugs, which are being addressed with novel interdisciplinary technical advances that, in some cases, are integrating the fields of gene, cell and mRNA therapies. Here, we review the scientific insights from a select group of clinical studies on mRNA-based drugs, including enzyme replacement therapies for rare diseases, cancer immunotherapies, genome-modifying therapies, and immune cell reprogramming therapies for cancer and autoimmune diseases. Several innovative approaches such as clinically tractable in vivo delivery systems, the development of completely 'immune-silent' mRNA-vehicle formulations that allow repeated administration and the development of approaches for preferential delivery to organs other than the liver would expedite the development of mRNA therapeutics 2.0.
    DOI:  https://doi.org/10.1038/s41573-026-01459-7
  38. Immunol Rev. 2026 Jul;340(1): e70136
      Advances in high-throughput sequencing, single-cell profiling, and genome engineering have transformed the study of T cell receptors (TCRs), enabling the identification and functional interrogation of antigen-specific repertoires at an unprecedented scale. This review discusses how recent methodological developments-including high-dimensional TCR discovery strategies, physiological receptor engineering, and longitudinal in vivo analyses-have reshaped our understanding of TCR-driven immune responses. Recruitment into immune responses originates from diverse naïve precursor pools and results in polyclonal populations in which multiple clonotypes contribute to antigen recognition. Within such populations, receptor properties, such as TCR avidity, influence the likelihood of recruitment, expansion, and persistence. However, the impact of these parameters depends strongly on biological context, including antigen availability, cellular competition, and tissue environment. Physiological engineering approaches, such as orthotopic TCR replacement, now enable causal interrogation of receptor function while preserving endogenous regulatory control. Together with advances in spatial and longitudinal profiling of human immune responses, these approaches allow increasingly precise analyses of connections between TCR identity and T cell fate. Integrating insights across antigen discovery, receptor engineering, and in vivo dynamics suggests that TCR biology is shaped by the interplay of receptor sequence, regulatory context, and tissue environment across time. Understanding these relationships will be essential for interpreting immune responses and for guiding the rational design of T cell-based immunotherapies.
    DOI:  https://doi.org/10.1111/imr.70136
  39. Cancer. 2026 Jun 01. 132 Suppl 1 e70416
      Antibody-drug conjugates (ADCs) represent a major advance in precision oncology, combining the selectivity of monoclonal antibodies with the cytotoxic potency of chemotherapy. By enabling targeted intracellular delivery of highly potent payloads, ADCs aim to maximize antitumor activity while minimizing systemic toxicity. This review summarizes the structural principles, mechanisms of action, and pharmacokinetic features that define ADC performance, and provides an updated overview of their clinical impact across solid tumors. The authors discuss pivotal trials that established ADCs as standards of care in breast, gynecologic, gastrointestinal, and lung cancers, and highlight emerging targets such as HER3, CLDN18.2, and B7-H3. Mechanisms of resistance, including antigen loss, impaired internalization, and payload-specific adaptations, are examined alongside strategies to overcome them, such as bispecific and dual-payload designs, rational combinations with immunotherapy or targeted agents, and next-generation linker technologies. Finally, the authors explore future directions, including biomarker-driven patient selection, integration into earlier lines of therapy, and innovative formats designed to widen the therapeutic window. With continued advances in engineering and clinical development, ADCs are poised to expand their role from salvage settings to frontline treatment, offering the potential for deeper and more durable responses across a broad spectrum of solid tumors.
    Keywords:  antibody–drug conjugates (ADCs); breast cancer; gastrointestinal cancers; gynecologic cancers; non–small cell lung cancer (NSCLC); small cell lung cancer (SCLC); targeted therapy
    DOI:  https://doi.org/10.1002/cncr.70416
  40. N Engl J Med. 2026 Jun 04. 394(21): 2117-2125
      HLA sensitization poses a major challenge to kidney transplantation for patients with end-stage kidney disease, especially for highly sensitized candidates. Attempts at antibody elimination (desensitization) have had inconsistent efficacy and have often failed to produce sustained reductions in anti-HLA antibodies in patients with the highest level of sensitization (calculated panel-reactive antibody score, ≥99.9%). We now report the results for the safety run-in cohort of a multicenter phase 1 clinical study evaluating the safety and efficacy of combined CD19-targeted and B-cell maturation antigen (BCMA)-targeted chimeric antigen receptor (CAR) T cells in eliminating the cellular sources of preformed anti-HLA antibodies (ClinicalTrials.gov number, NCT06056102). Kidney transplantation was performed in two highly sensitized candidates after desensitization with the use of dual CAR T-cell therapy.
    DOI:  https://doi.org/10.1056/NEJMoa2513428
  41. bioRxiv. 2026 May 24. pii: 2026.05.22.726983. [Epub ahead of print]
      Chimeric antigen receptors (CARs) can induce T cells to kill cancer cells but also to kill normal cells that express the same antigens 1 . Designing CARs to recognize combinations of antigens, via Boolean logic, can simultaneously expand the scope of targetable antigens and make CAR T cells more specific to cancer 2 . For example, one antigen may be expressed on cancer cells and normal bone marrow cells, while a second antigen may be present on the same cancer cells but only in normal lungs. If recognition of both antigens is required for T cell activation, only the cancer cells will be killed. Creating such AND-gated CAR T cells has been challenging given the need to engineer non-natural signaling mechanisms that integrate two ligand binding events into a single T cell activation stimulus 3-6 . Here, we design a fundamentally new AND-gated receptor called M ulti- AN tigen T riggered I mmune S ynapse (MANTIS), which leverages differences in extracellular receptor dimensions to regulate CAR signaling. MANTIS initially prevents CAR activity by steric blocking with a bulky extracellular domain. Upon engagement of the first antigen, MANTIS sheds this blocking domain, releasing a free CAR that can bind a second antigen and activate the T cell in an AND-gated manner. This work demonstrates how differences in extracellular receptor size can be leveraged to spatially regulate intracellular signaling pathways in response to antigen patterns, paving the way for new applications in synthetic biology and cell engineering.
    One sentence summary: Differences in extracellular size can be leveraged to regulate CAR T cell activity for precise recognition of antigen patterns.
    DOI:  https://doi.org/10.64898/2026.05.22.726983
  42. Cell Commun Signal. 2026 Jun 01.
      In recent years, chimeric antigen receptor-macrophages (CAR-Ms) have emerged as a pivotal branch of adoptive cell therapy. They are distinguished by their innate tumor infiltration capacity, phagocytic activity, and immunomodulatory functions, attributes that render them particularly promising for the treatment of solid tumors and non-malignant diseases. Nevertheless, the widespread clinical translation of CAR-M therapy has been constrained by the complexity, high cost, and potential genotoxicity inherent to traditional ex vivo manufacturing processes. As a transformative paradigm, in vivo CAR-M therapy bypasses these limitations by directly delivering CAR-encoding sequences to myeloid cells using advanced platforms such as viral vectors, lipid nanoparticles, extracellular vesicles, or biomaterials. This strategy enables the in situ genetic programming and functional remodeling of macrophages, thereby streamlining therapeutic workflows, reducing production costs, and broadening patient accessibility. This review systematically summarizes recent advances in in vivo CAR-M therapy, focusing on its applications across diverse solid tumors and non-malignant conditions. We compare in vivo engineering strategies with conventional adoptive CAR-M approaches from both mechanistic and translational perspectives and critically examine key challenges, including delivery specificity, immunological safety, and controllability of transgene expression. Collectively, in vivo CAR-M represents a paradigm shift from ex vivo manufacturing toward in vivo cellular programming, offering a more accessible and scalable next-generation immunotherapeutic platform for solid tumors and inflammation-related diseases.
    Keywords:   In vivo engineering; CAR-Macrophage; Immunotherapy; Solid tumor; Targeted gene delivery
    DOI:  https://doi.org/10.1186/s12964-026-02962-5
  43. Intractable Rare Dis Res. 2026 May 31. 15(2): 120-128
      Globally, the prevention and treatment of rare diseases is still constrained by limited diagnostic and therapeutic capacity, restricted drug accessibility, and disparities in medical security systems. In response, China has developed a distinct "China Model" of rare disease governance, characterized by national policy leadership and coordinated local implementation. This study systematically reviews policies issued between 2009 and 2026 and it analyzes five domains: i) prevention and screening, ii) list-based governance, iii) clinical diagnosis and treatment systems, iv) drug accessibility, and v) payment guarantees. Shandong Province is examined as a representative case. Findings show that the central government has established unified standards through two nationally endorsed rare disease lists covering 207 conditions, supported by clinical guidelines and a national collaborative network for diagnosis and treatment of those diseases. Regulatory incentives for drug review and approval have facilitated the inclusion of 126 treatments for patients with rare diseases in the National Basic Medical Insurance reimbursement list, forming an integrated policy framework spanning identification, diagnosis, treatment, and financial protection. At the provincial level, Shandong is aligned with national directives by integrating its case registration system with the national platform, enhancing quality control across its clinical network and developing a multilevel payment mechanism. The core of the "China Model" is the enhancement of clinical capacity through standardized systems and networked organizations, combined with multilevel risk-sharing mechanisms. However, governance challenges persist, including weak inter-organizational policy coordination, barriers to drug accessibility, fragmented coverage schemes, and an underdeveloped data governance infrastructure. Addressing these challenges requires enhanced end-to-end policy implementation and institution of effective local practices at the national level.
    Keywords:  China model; case registration; collaborative network for diagnosis and treatment; drug accessibility; medical security; rare diseases
    DOI:  https://doi.org/10.5582/irdr.2025.01060
  44. Curr Opin Biotechnol. 2026 Jun 01. pii: S0958-1669(26)00086-8. [Epub ahead of print]100 103521
      Red-cell therapeutics are often discussed as a single class, but current products use erythroid biology in distinct ways. Intact red blood cells (RBCs) are long-lived, deformable cells, circulating for about 120 days in humans and roughly 40-60 days in mice, and repeatedly contact immune, endothelial, and reticuloendothelial cells. They are best suited to applications that benefit from intravascular persistence and repeated surface interactions, including enzyme carriage, durable ligand or antigen display, immune modulation, and hitchhiking designs. RBC-derived extracellular vesicles (RBC-EVs) share erythroid membrane lineage at a smaller scale but operate by a different logic. Rather than relying on prolonged circulation, RBC-EVs derive their value from hours-scale routing to recipient cells, cargo protection, uptake, endolysosomal release, antigen processing, and functional intracellular cargo activity after entry. This review argues that red-cell therapeutics are best understood by engineering strategy rather than disease indication alone. For intact RBCs, the central task is to add therapeutic function while preserving deformability, membrane organization, antigen integrity, immune compatibility, and acceptable clearance. Major strategies include covalent conjugation, affinity anchoring, lipid insertion, enzymatic ligation, genetic or precursor-cell engineering, and hitchhiking-based surface association, each balancing functional gain against biological burden. For RBC-EVs, the design problem shifts to vesicle generation, cargo loading and retention, surface functionalization, targeting, intracellular delivery, storage stability, potency, and batch reproducibility. Future progress will require engineering routes that increase efficacy while reducing perturbation and manufacturing complexity, supported by scalable erythroid sources, standardized downstream processing, and release criteria matched to each platform.
    DOI:  https://doi.org/10.1016/j.copbio.2026.103521
  45. Cancer. 2026 Jun 01. 132 Suppl 1 e70421
      Antibody-drug conjugates (ADCs) represent an important therapeutic modality for the treatment of various hematologic malignancies. These include gemtuzumab ozogamicin and inotuzumab ozagamicin for acute leukemias; brentuximab vedotin, polatuzumab vedotin, and loncastuximab tesirine for lymphoid malignancies; and belantamab mafadotin for multiple myeloma. Although ADCs do provide an effective off-the-shelf approach to selectively deliver cytotoxic agents by targeting specific cell surface antigens, many ADCs have also been associated with unique toxicities related to the antigen target, linker, or payload that have limited their development. In this review, the clinical development and current role of ADCs in the therapeutic landscape of hematologic malignancies are discussed.
    Keywords:  antibody–drug conjugates; hematologic malignancies; leukemia; lymphoma; multiple myeloma
    DOI:  https://doi.org/10.1002/cncr.70421
  46. J Nanobiotechnology. 2026 Jun 04.
      Cancer continues to be one of the leading causes of death worldwide. Although immunotherapy has transformed oncologic treatment by mobilizing the host immune system, the immunosuppressive tumor immune microenvironment (TIME) still constitutes a major barrier to durable clinical responses. Recent studies indicate that iron-based magnetic nanoparticles (Fe-MNPs) offer capabilities that extend beyond those of conventional drug delivery systems, acting instead as active regulators of the TIME. In this review, we outline current progress in Fe-MNP-mediated cancer immunotherapy and propose a conceptual framework to clarify their immunomodulatory effects. Specifically, Fe-MNPs can generate multiple physicochemical stimuli under magnetic field control, including magnetic hyperthermia, magnetically induced mechanical perturbation, and Fenton reaction-mediated oxidative stress. These upstream events converge to induce immunogenic stress, including ferroptosis-associated injury, which in turn promotes hypoxia alleviation, tumor-associated macrophage (TAM) reprogramming, dendritic cell activation, and enhanced effector T-cell responses. Together, these findings support a framework in which Fe-MNP-mediated immunomodulation is understood as an integrated cascade rather than a collection of isolated effects. However, challenges remain regarding biosafety, long-term iron metabolism, delivery efficiency, and large-scale manufacturing. Overall, this review provides a systematic overview of Fe-MNP-mediated immunomodulation and discusses the translational prospects of these materials as multifunctional platforms for cancer immunotherapy.
    Keywords:  Effector T cells; Ferroptosis; Immunosuppression; Iron-based magnetic nanoparticles; Tumor immune microenvironment; Tumor-associated macrophages
    DOI:  https://doi.org/10.1186/s12951-026-04629-2