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Chimeric antigen receptor T cell therapy: a new emerging landscape in autoimmune rheumatic diseases.

Chimeric antigen receptor T cell (CAR-T) therapy, an innovative immune cell therapy, has revolutionized the treatment landscape of haematological malignancies. The past 2 years has witnessed the successful application of CD19-targeting CAR constructs in refractory cases of autoimmune rheumatic diseases, including systemic lupus erythematosus, systemic sclerosis and anti-synthetase syndrome. In comparison with existing B cell depletion therapies, targeting CD19 has demonstrated a more rapid and profound therapeutic effect, enabling drug-free remission with manageable adverse events. These promi

B-cell maturation antigen-targeted CAR T cell as a salvage therapy for progressive multiple myeloma after anti-G proteincoupled receptor, class C group 5 member D CAR T-cell therapy.

Repeat infusion of chimeric antigen receptor (CAR) T-cell is effective for patients with refractory or relapsed multiple myeloma (RRMM) after failing a prior CAR Tcell therapy. It is unknown whether B-cell maturation antigen (BCMA) CAR T-cell is effective for patients with RRMM after anti-G protein-coupled receptor, class C group 5 member D (GPRC5D) CAR-T cell therapy. In this study, 20 subjects with RRMM after a prior anti-GPRC5D CAR T-cell therapy received BCMA-targeted CAR T-cell therapy as a salvage treatment. During a median follow-up of 13.4 months (IQR 3.6-21.2), 13 subjects showed resp

Reimbursement of licensed cell and gene therapies across the major European healthcare markets.

ObjectiveThe aim of this research is to identify the pricing, reimbursement, and market access (P&R&MA) considerations most relevant to advanced therapy medicinal products (ATMPs) in the Big5EU, and to inform their manufacturers about the key drivers for securing adoption at a commercially viable reimbursed price.MethodologyThe research was structured following three main steps: 1) Identifying the market access pathways relevant to ATMPs through secondary research; 2) Validating the secondary research findings and addressing any data gaps in primary research, by qualitative interviews with nat

Chimeric Antigen Receptor T-Cells in Myasthenia Gravis: Advances, Safety Challenges, and Future Directions.

This review examines the emerging application of chimeric antigen receptor (CAR) T-cell therapy in myasthenia gravis (MG), with emphasis on safety, efficacy signals, and future therapeutic potential in treatment-refractory disease. A comprehensive literature search was conducted across PubMed, medRxiv, bioRxiv, and Google Scholar for studies published between January 1, 2010 and July 1, 2025, using the terms "CAR T-cell," "chimeric antigen receptor," and "myasthenia gravis." Eligible reports included clinical trials, case reports, case series, and mechanistic studies describing CAR T-cell ther

The chemokines CXCL8 and CXCL12: molecular and functional properties, role in disease and efforts towards pharmacological intervention.

Chemokines are an indispensable component of our immune system through the regulation of directional migration and activation of leukocytes. CXCL8 is the most potent human neutrophil-attracting chemokine and plays crucial roles in the response to infection and tissue injury. CXCL8 activity inherently depends on interaction with the human CXC chemokine receptors CXCR1 and CXCR2, the atypical chemokine receptor ACKR1, and glycosaminoglycans. Furthermore, (hetero)dimerization and tight regulation of transcription and translation, as well as post-translational modifications further fine-tune the s

Clinical innovations and future directions of nanoparticles in the treatment of psychiatric and neurological disorders.

Nanoparticles (NPs) provide a versatile toolkit for psychiatry and neurology by leveraging tunable size, surface chemistry, and payload control to overcome long-standing barriers in central nervous system (CNS) therapy. Lipid, polymeric, inorganic, and hybrid NPs can be engineered to traverse the blood-brain barrier (BBB) via receptor/transporter pathways, target specific cell types, and deliver sustained or stimuli-responsive release. Beyond drug delivery, NPs improve imaging, enable gene/RNA therapeutics, and support anti-inflammatory and neuroprotective strategies, advancing precision medic

Treatment preferences of patients with relapsed and refractory multiple myeloma: a qualitative study.

BACKGROUND: Multiple myeloma is a haematological malignancy characterized by significant morbidity and mortality. This study sought to develop an in-depth understanding of patients' lived experiences of relapsed or refractory multiple myeloma (RRMM) and its treatment, and to identify which features of treatment were most important to them. METHODS: Qualitative interviews and focus groups (FGs) were conducted with 32 people living with RRMM across Canada. In Phase 1, interviews focused on participants' accounts of their experiences with the disease and its treatment and laid the groundwork for

Not so Fast: Co-Requirements for Sonic Hedgehog Induced Brain Tumorigenesis.

The Sonic hedgehog (Shh) pathway plays an integral role in cellular proliferation during normal brain development and also drives growth in a variety of cancers including brain cancer. Clinical trials of Shh pathway inhibitors for brain tumors have yielded disappointing results, indicating a more nuanced role for Shh signaling. We postulate that Shh signaling does not work alone but requires co-activation of other signaling pathways for tumorigenesis and stem cell maintenance. This review will focus on the interplay between the Shh pathway and these pathways to promote tumor growth in brain tu

Perspectives for the Use of CAR-T Cells for the Treatment of Multiple Myeloma.

During recent years considerable progress has been made in the treatment of multiple myeloma. However, despite the current improvements in the prognosis of this malignancy, it always ends with relapse, and therefore new therapy approaches for destroying resistant cancer cells are needed. Presently, there is great hope being placed in the use of immunotherapy against refractory/relapsed multiple myeloma which is unresponsive to any other currently known drugs. The most promising one is CAR-T cell therapy which has already shown tremendous success in treating other malignancies such as acute lym

Spine surgery for metastatic spine cancer in the era of advanced radiation therapy.

Metastatic spine cancer (MSC), a common complication of advanced malignancies, poses significant challenges due to pain, neurological deficits, and mechanical instability. While radiation therapy is a cornerstone of treatment, the role of spine surgery is evolving, fueled by advances in surgical techniques and radiation modalities such as stereotactic body radiation therapy (SBRT). This review examines the evolving role of spine surgery in MSC management, focusing on separation surgery, surgical innovations, and future directions. The treatment paradigm for MSC shifted with the advent of SBRT,

Cardiac Amyloidosis Treatment.

Cardiac amyloidosis (CA) is a restrictive cardiomyopathy with a traditionally poor prognosis. Until recently, CA treatment options were limited and consisted predominantly of managing symptoms and disease-related complications. However, the last decade has seen significant advances in disease-modifying therapies, increased awareness of CA, and improved diagnostic methods resulting in earlier diagnoses. In this review, we provide an overview of current and experimental treatments for the predominant types of CA: transthyretin cardiac amyloidosis (ATTR-CA) and immunoglobulin light chain (AL)-med

Роль агентов CELMOD в Мультимиломе.

Несмотря на то, что в последние десятилетия наблюдалось дальнейшее улучшение положения в области выживания пациентов с множественными миеломами, это заболевание в значительной степени остается неизлечимым, и большинство пациентов переживают рецидивы и/или становятся невосприимчивыми к лечению. Таким образом, по-прежнему остро ощущается потребность в новых методах лечения, особенно для тех пациентов с рецидивистской или рефрактной множественной миеломой. Новейшие методы лечения, такие, как целевая деградация белка, вызывают особый интерес, поскольку они позволяют расширить диапазон поддающихся

Clinical Posters From JADPRO Live 2022

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DNA Damaging Drugs

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Clinical application of cytokine-induced killer cells in cancer immunotherapy.

Cytokine-induced killer (CIK) cells exhibit potent antitumor activity with reduced dependence on strict MHC compatibility, mediated by a combination of T-cell receptor (TCR)-dependent recognition and natural killer (NK)-like, MHC-unrestricted cytotoxicity. This narrative review synthesizes evidence from clinical trial evaluations, including 48 published studies. We examine strategies to enhance CIK function, such as engineering, combination with checkpoint inhibitors, and co-administration with dendritic cells. CIK cell therapy effectively reduces post-treatment recurrence in the adjuvant sett

Uncertainties about the benefit-risk balance of oncology medicines assessed by the European Medicines Agency.

BackgroundDrug regulators assess and describe uncertainties regarding treatment outcomes and the benefit-risk balance of newly authorised medicines. We aimed to evaluate the type and number of uncertainties described in the benefit-risk assessment for initial marketing authorisations of oncology medicines assessed by the European Medicines Agency (EMA). We also aimed to develop a systematic classification of uncertainties to contribute to improved communication about uncertainties.Materials and methodsWe included all medicines containing a new active substance assessed by the EMA and granted a

Society for Immunotherapy of Cancer (SITC) clinical practice guideline on immunotherapy for the treatment of lymphoma.

The recent development and clinical implementation of novel immunotherapies for the treatment of Hodgkin and non-Hodgkin lymphoma have improved patient outcomes across subgroups. The rapid introduction of immunotherapeutic agents into the clinic, however, has presented significant questions regarding optimal treatment scheduling around existing chemotherapy/radiation options, as well as a need for improved understanding of how to properly manage patients and recognize toxicities. To address these challenges, the Society for Immunotherapy of Cancer (SITC) convened a panel of experts in lymphoma

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… In patients with a different hematological malignancy, B cell acute lymphoblastic leukemia, anti-CD19 CAR T cell therapies have been successfully used as a bridge therapy to enable subsequent alloSCT, with promising rates of CR and no reported incidence of GVHD. 155 156 This result may serve as a blueprint …

Химерический антигенный рецептор T-клеточная терапия в нейроиммунных болезнях: механистическое обоснование, клинический опыт и проблемы с переводом.

Нейроиммунные заболевания представляют собой разнородную группу расстройств, при которых клетки В, плазменные удары, плазменные клетки, автоантитела и Т клетки поразному влияют на повреждение тканей. Хотя биологика B-клеток улучшила контроль за заболеваниями, некоторые пациенты по-прежнему сталкиваются с реанимацией, рецидивом после лечения или кумулятивной неврологической инвалидностью. Химерическая антигенная рецепторная T-клетка (CAR-T-клетка) является средством достижения более глубокого и потенциально устойчивого истощения определенных патогенных иммунных клеток. Этот описательный обзор в

Cardiotoxicity Induced by Anticancer Therapies: A Call for Integrated Cardio-Oncology Practice.

The introduction of novel oncologic therapies, including targeted agents, immunotherapies, and antibody-drug conjugates, has transformed the therapeutic landscape of cancer care. This evolution has resulted in a dual clinical scenario; while survival outcomes have markedly improved, leading to a growing population of long-term cancer survivors, an increasing incidence of previously unrecognized treatment-related toxicities has emerged. Among these, cardiovascular adverse events represent some of the most prevalent and clinically significant complications observed in both conventional chemother

Reciprocal regulation between m6 A modifications and non-coding RNAs: emerging roles in cancer therapeutic resistance.

In recent years, the interplay between N6-methyladenosine (m6A) modifications and non-coding RNAs (ncRNAs) has emerged as a pivotal research area, owing to their crucial involvement in the pathophysiological mechanisms underlying various diseases. A significant hurdle in cancer therapy is therapeutic resistance, which frequently contributes to adverse patient outcomes. Recent investigations have underscored the vital role that interactions between m6A modifications and ncRNAs play in mediating cancer therapeutic resistance via the MAPK, PI3K/Akt/mTOR, Wnt/β-catenin, HIPPO, and NF-κB pathways.