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Global Panoramic analysis of clinical research in cell therapy: clinical trial landscape, marketed products, and regulatory trends.

IntroductionCell therapy is a vital field in modern medicine. This review assesses its global clinical development landscape, approved products, and regional regulatory characteristics.MethodsA comprehensive search and analysis of global clinical trial databases (e.g., https://clinicaltrials.gov/ClinicalTrials.gov), regulatory agency announcements, and relevant literature up to October 2025 was conducted. Key data points regarding trial numbers, phases, therapy types, and approved products were extracted and analyzed descriptively by region.ResultsA cumulative total of 10,373 cell therapy clin

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… Elivaldogene autotemcel (brand name Skysona®) is indicated for the treatment of early active cerebral adrenoleukodystrophy (CALD) in adolescent boys. … Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy. N. Engl. J. Med. 377 (17), 1630–1638. 10.1056/NEJMoa1700554 Fernández-Garza L. E., Barrera-Barrera S. A., Barrera-Saldaña H. A. (2023). Mesenchymal stem cell therapies approved by regulatory agencies around the world. …

Leukoencephalopathy During Daratumumab-Based Therapy: A Case Series of Two Patients with Multiple Myeloma.

Leukoencephalopathy in the setting of multiple myeloma (MM) is a rare demyelinating condition, with few reported cases in literature. Daratumumab is a CD38 targeted monoclonal antibody that has been widely used for the management of MM. In the absence of central nervous system (CNS) disease, many medication-induced leukoencephalopathy cases reported with MM, including daratumumab-induced, are associated with progressive multifocal leukoencephalopathy (PML) and John Cunningham (JC) virus. Currently, there are no reported cases of daratumumab-induced leukoencephalopathy among patients without CN

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… Adult-onset adrenoleukodystrophy presenting after chemotherapy . Neurol Clin Pract . 2014 ; 4 ( 2 ): 168 – 170 . doi: 10.1212/01.CPJ.0000435751.17621.93 29443243 PMC5765618 13. Cai X , Bhattacharyya S , Plitt A , et al. …

Наркотики, основанные на сходстве генных экспрессионных сигнатур, чтобы исследовать потенциальные признаки кексетина: тематическое исследование множественных склерозов.

Quercetin (QR) — природный флавонол, широко распространенный в королевстве растений с обширными фармакологическими последствиями. Чтобы найти потенциальные клинические показатели QR, 156 Дифференциально выраженные гены (DEG), регулируемые QR, были получены из базы данных Gene Express Omnibus, и новые потенциальные фармакологические эффекты и клинические показатели QR были перенаправлены путем интегрирования соединений с аналогичными генными сигнатурами возмущения и соответствующими сигнатурами болезни QR на основе карты связи и платформ Coexedia. Предлагаемые результаты QR оказывают главным об

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… potential indications for QR include neuromuscular diseases (muscular dystrophy), autoimmune diseases (multiple sclerosis), mouth diseases (gingivitis), joint diseases (arthritis), neoplasms (liposarcoma, leukemia, and lymphoma), metabolic diseases (diabetes mellitus and hemochromatosis), and CNS diseases (adrenoleukodystrophy …

Prevention of Bortezomib-Induced Peripheral Neuropathy in Newly Multiple Myeloma Patients Using Nervonic Acid, Curcuma Rizoma, and L-Arginine Compound: A Pilot Study.

INTRODUCTION: This is a phase II pilot study to evaluate the efficacy of a nutraceutical compound composed of nervonic acid, curcuma rizoma, and l-Arginine to prevent the onset of bortezomib-induced peripheral neuropathy (BIPN) in 16 newly diagnosed multiple myeloma (MM) patients treated with bortezomib (BTZ) over 6 months. MATERIALS AND METHODS: Assessments included neurological examination and electroneurography, Common Terminology Criteria for Adverse Events (NCI-CTCAE), reduced version of Total Neuropathic Score (TNSr), pain evaluation, functional autonomy scales, self-perceived symptoms

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… Localization of nervonic acid beta-oxidation in human and rodent peroxisomes: impaired oxidation in Zellweger syndrome and X-linked adrenoleukodystrophy. J Lipid Res. 1998;39:2161-2171. 22. Basu P, Maier C, Basu A. …

Biochemical and Metabolical Pathways Associated with Microbiota-Derived Butyrate in Colorectal Cancer and Omega-3 Fatty Acids Implications: A Narrative Review.

Knowledge regarding the influence of the microbial community in cancer promotion or protection has expanded even more through the study of bacterial metabolic products and how they can modulate cancer risk, which represents an extremely challenging approach for the relationship between intestinal microbiota and colorectal cancer (CRC). This review discusses research progress on the effect of bacterial dysbiosis from a metabolic point of view, particularly on the biochemical mechanisms of butyrate, one of the main short chain fatty acids (SCFAs) with anti-inflammatory and anti-tumor properties

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… During the last decade, inhibition of HDACs by HDAC inhibitors (HDACIs) emerged as a target for specific epigenetic modification associated with cancer or other diseases (hemoglobinopathies; cystic fibrosis; X-linked adrenoleukodystrophy; muscular dystrophies; neurodegenerative disorders; systemic lupus …

Длиннокаменистые толстые кислоты как движущие факторы нейроинфламмации в нейродегенерации: механистические связи с липидом пероксидацией, Ферроптозом и митохондриальной дисфункцией.

Фон: Нейродегенеративные заболевания (НИ) рассматриваются главным образом как расстройства, характеризующиеся серьезным иммунометаболическим дисбалансом, характеризующимся текущей нейроинфламмацией и глиальной активацией. Хотя митохондриальная дисфункция и окислительный стресс являются хорошо известными особенностями, метаболические факторы в верхнем течении, связывающие эти патологические процессы, по-прежнему плохо понимаются. Методы: В ходе этого обзора мы изучили недавние доклинические и клинические исследования, в ходе которых были изучены связи между липидным метаболизмом, иммунометаболи

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… Mitochondrial Dysfunction and Impaired Oxidative Stress Defense as Potential Trigger of Cerebral X-Linked Adrenoleukodystrophy Free Radic. Biol. Med. 2026 242 654 666 10.1016/j.freeradbiomed.2025.10.289 41177236 52. Singh A. Tiwari V. Roy S. …

Gene therapy: principles, challenges and use in clinical practice.

INTRODUCTION: Gene therapy is an emerging topic in medicine. The first products have already been licensed in the European Union for the treatment of immune deficiency, spinal muscular atrophy, hemophilia, retinal dystrophy, a rare neurotransmitter disorder and some hematological cancers, while many more are being assessed in preclinical and clinical trials. OBJECTIVE: The purpose of this review is to provide an overview of the core principles of gene therapy along with information on challenges and risks. Benefits, adverse effects and potential risks are illustrated based on the examples of

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… Ex vivo gene therapies commonly use cells of hematopoietic origin such as hematopoietic stem cells or T cells, and are being developed for the treatment of inherited immune disorders, neurodegenerative diseases (e.g., X‑linked adrenoleukodystrophy, metachromatic leukodystrophy), β‑hemoglobinopathies …

Real-World Data Included in Post-authorisation Measures: A Case Study of Approved Advanced Therapy Medicinal Products in the European Union between 2013 and 2024.

BackgroundAdvanced therapy medicinal products (ATMPs) often require long-term monitoring to assess both safety and efficacy post-authorisation due to uncertainties identified during the approval process. This study aims to characterise the use of real-world data (RWD) in post-authorisation measures (PAMs) for ATMPs approved in the European Union.MethodsA systematic extraction of all PAMs from publicly available European Medicines Agency (EMA) regulatory documents for ATMPs approved between January 2013 and December 2024 was performed, followed by the identification of the presence and sources

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… | Conditional | 2020 | Authorised | Yes | Yes Zolgensma | Onasemnogene abeparvovec | Spinal muscular atrophy | M09AX09 | Conditional | 2020 | Authorised | Yes | Yes Abecma | Idecabtagene vicleucel | Multiple myeloma | L01 | Regular | 2021 | Authorised | Yes | Yes Skysona | Elivaldogene autotemcel | Adrenoleukodystrophy …

Market Access Challenges and Solutions in Cell and Gene Therapy in The Netherlands.

With the increasing pipeline of cell and gene therapies (CGTs) and the expected surge in the number of approvals, understanding the market access landscape becomes crucial for timely patient access. This study evaluates the challenges Dutch stakeholders encounter in CGT market access, offering insights for improving time-to-patient access. A traditional literature review was conducted to identify market access challenges and solutions for CGTs. Based on the findings, participants for semi-structured interviews, designed using an interview guide adapted to the Dutch context, were selected to ca

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… Consequently, access to treatment options for patients with respective conditions such as multiple myeloma, mantle cell lymphoma, cerebral adrenoleukodystrophy, beta-thalassemia, sickle cell disease, and haemophilia B remains constrained. …

Indications for haematopoietic cell transplantation and CAR-T for haematological diseases, solid tumours and immune disorders: 2025 EBMT practice recommendations.

For over two decades, the EBMT has updated recommendations on indications for haematopoietic cell transplantation (HCT) practice based on clinical and scientific developments in the field. This is the ninth special EBMT report on indications for HCT for haematological diseases, solid tumours and immune disorders. Our aim is to provide guidance on HCT indications according to prevailing clinical practice in EBMT countries and centres. In order to inform patient decisions, these recommendations must be considered with the risk of the disease, risk of HCT procedure and non-HCT strategies, includi

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… in DQB1), PSD peroxisomal storage diseases, S standard of care (generally indicated in suitable patients), SAA severe aplastic anaemia, SCD sickle cell disease (high risk), SCID severe combined immunodeficiencies, TDT Transfusion dependent Thalassaemia, TKI tyrosine kinase inhibitors, X-ALD X-linked adrenoleukodystrophy … Among PSD, allo-HCT is effective in preventing disease progression in childhood, (or in rare instances adult) forms of X-linked adrenoleukodystrophy (X-ALD), a cerebral inflammatory disease. …

Defibrotide for prophylaxis of sinusoidal obstruction syndrome/veno-occlusive disease (SOS/VOD) in pediatric high-risk patients: consensus guidelines from the European Society for Blood and Marrow Transplantation (EBMT).

SOS/VOD is a life-threatening complication of hematopoietic stem cell transplantation, especially in children, with incidences reaching up to 15-20%. Despite efforts, SOS/VOD remains unpredictable with significant morbidity and mortality. High-risk criteria are clearly defined, and the pediatric EBMT diagnostic criteria have improved sensitivity, reducing treatment delays and enhancing outcomes. A meta-analysis combining retrospective and prospective studies found a risk ratio of 0.30 for SOS/VOD with defibrotide (DF) prophylaxis. Additionally, two prospective trials were conducted: the pediat

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… Adjudication Committee (EPAC) Inclusion criteria | • 2nd myeloablative HSCT • Allogeneic HSCT for leukemia > 2nd relapse • Pre-existing Liver disease • Prior Abdominal irradiation • Prior treatment with Gemtuzumab • Busulfan/Melphalan Conditioning • Osteopetrosis • Familiar macrophage activating syndrome • Adrenoleukodystrophy …

EU Regulatory Pathways for ATMPs: Standard, Accelerated and Adaptive Pathways to Marketing Authorisation.

Advanced therapy medicinal products (ATMPs) require evaluation by the European Medicines Agency's Committee for Advanced Therapies prior to being placed on the European market, subject to a Marketing Authorisation granted by the European Commission. In common with other medicinal products, various regulatory pathways are available for taking ATMPs through clinical trials to market authorisation, and the regulatory pathway taken will depend on a product's characteristics and the target patient population. With the industry poised to deliver more late-stage clinical and commercial ATMPs for seri

Exploration and Development of PPAR Modulators in Health and Disease: An Update of Clinical Evidence.

Peroxisome proliferator-activated receptors (PPARs) are nuclear receptors that govern the expression of genes responsible for energy metabolism, cellular development, and differentiation. Their crucial biological roles dictate the significance of PPAR-targeting synthetic ligands in medical research and drug discovery. Clinical implications of PPAR agonists span across a wide range of health conditions, including metabolic diseases, chronic inflammatory diseases, infections, autoimmune diseases, neurological and psychiatric disorders, and malignancies. In this review we aim to consolidate exist

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… However, bezafibrate failed to improve the clinical symptoms of patients with X-linked adrenoleukodystrophy [ 137 ], CPT2, and very long-chain acyl-CoA dehydrogenase deficiency [ 138 ]. … small-sized HDL | [ 135 ] Bezafibrate + Statins | NA (15) | ▪ Bezafibrate with statin reduced post-fat load triglyceride, APOB and estimated glomerular filtration rate. ▪ Fasting levels of non-HDL-cholesterol, total cholesterol, and HDL-cholesterol were improved with combined therapy. | [ 136 ] X-linked adrenoleukodystrophy …

Применение мезенхимальных ячеек и их эксзоом в нейродегенеративных заболеваниях и лизосомных заболеваниях.

Мезенхимальные стромальные клетки (МСЦ) стали перспективной терапевтической платформой для расстройств центральной нервной системы, включая нейродегенеративные заболевания и лизосомные расстройства хранения (ЛСД). В настоящем обзоре рассматриваются механизмы КБМ, связанные с активностью паракрина, иммуномодуляцией, воздействием антиоксидантов, регулированием с помощью ТФЭБ, а также с ферментатической перекрестной корректировкой в ЛСД, хотя и критически оцениваются проблемы перевода. Мы проводим сравнительный анализ источников КБМ, административных маршрутов, дозирующих режимов и характеристик

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… In cerebral adrenoleukodystrophy, IT administration of allogeneic MSCs in two patients was feasible and uncomplicated; however, demyelination progression continued [ 123 ]. …

The Contribution of Spanish Science to Patents: Medicine as Case of Study.

Investments in research and development (R&D) and innovation are expensive, and one wishes to be assured that there is positive feedback and to receive guidance on how to direct investments in the future. The social or economic benefits of investments in R&D are of particular interest to policymakers. In this regard, public expense in research, especially through universities, is sometimes being questioned. This paper establishes a measure of how research in Spain, and specifically in its universities, is involved. In this study, we have analyzed all the literature cited in the period 1998-201

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… Alzheimer Disease, Tau oligomers | 12 Platelet-Rich Plasma, Blood Platelets, Intercellular Signaling Peptides and Proteins | 10 Deep Brain Stimulation, Parkinson Disease, Microelectrode recording | 8 Alpha-Synuclein, Parkinson Disease, Protein α- synuclein | 7 Lipids, Lipolysis, Adipose triglyceride | 6 Adrenoleukodystrophy …

Current landscape of clinical development and approval of advanced therapies.

Advanced therapy medicinal products (ATMPs) are innovative therapies that mainly target orphan diseases and high unmet medical needs. The uncertainty about the product's benefit-risk balance at the time of approval, the limitations of nonclinical development, and the complex quality aspects of those highly individualized advanced therapies are playing a key role in the clinical development, approval, and post-marketing setting for these therapies. This article reviews the current landscape of clinical development of advanced therapies, its challenges, and some of the efforts several stakeholde

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… autotemcel | other nervous system drugs/N07 | leukodystrophy, metachromatic/standard approval Abecma (orphan medicine) | idecabtagene vicleucel | antineoplastic agents/L01 | multiple myeloma/conditional approval Skysona (orphan medicine) | elivaldogene autotemcel | other nervous system drugs/N07 | adrenoleukodystrophy …

Comprehensive analysis of plasma cell heterogeneity and immune interactions in multiple myeloma.

This study focused on the role of plasma cells in multiple myeloma (MM) and the associated potential mechanisms. Transcriptomic data of MM and various gene sets from several public databases were downloaded for subsequent analyses. Through single-cell sequencing, 10 major cell types were identified and annotated. The differential gene expression and pathway enrichment between different plasma cell subtypes as well as cell communication analysis, transcriptional regulation analysis, and enrichment analysis in conjunction with the malignant subpopulation were performed. Next, the samples were cl

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… Toxic effects of X-linked adrenoleukodystrophy-associated, very long chain fatty acids on glial cells and neurons from rat hippocampus in culture. Hum Mol Genet. (2008) 17:1750–61. doi: 10.1093/hmg/ddn066 55. Salemi M, Ravo M, Lanza G, Schillaci FA, Ventola GM, Marchese G, et al. …

Barriers to Treg therapy in Europe: From production to regulation.

There has been an increased interest in cell based therapies for a range of medical conditions in the last decade. This explosion in novel therapeutics research has led to the development of legislation specifically focused on cell and gene based therapies. In Europe, the European medicines agency (EMA) designates any medicines for human use which are based on genes, tissues, or cells as advanced therapy medicinal products or advanced therapy medicinal products (ATMPs). In this article we discuss the hurdles to widespread adoption of ATMPs in Europe, with a focus on regulatory T cells (Tregs).

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… Yes | | NCT02122952 Libmeldy | GTMP | Lentiviral based gene therapy for metachromatic leukodystrophy | 17/12/20 | Yes | | NCT01560182 Tecartus | GTMP | CAR-T cell therapy for mantle cell lymphoma | 14/12/20 | Yes | | NCT02601313 Skysona | GTMP | Lentiviral transduced HSC for treatment of cerebral adrenoleukodystrophy …

A systems-pharmacology analysis of herbal medicines used in health improvement treatment: predicting potential new drugs and targets.

For thousands of years, tonic herbs have been successfully used all around the world to improve health, energy, and vitality. However, their underlying mechanisms of action in molecular/systems levels are still a mystery. In this work, two sets of tonic herbs, so called Qi-enriching herbs (QEH) and Blood-tonifying herbs (BTH) in TCM, were selected to elucidate why they can restore proper balance and harmony inside body, organ and energy system. Firstly, a pattern recognition model based on artificial neural network and discriminant analysis for assessing the molecular difference between QEH an

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… The most common metabolic disorders include diabetes, obesity, adrenoleukodystrophy, glucose galactose malabsorption and phenylketonuria, and so forth. …

Indications for haematopoietic cell transplantation for haematological diseases, solid tumours and immune disorders: current practice in Europe, 2022.

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… MUD well-matched unrelated donor (8/8, 10/10, or 9/10 if mismatched is in DQB1), PSD peroxisomal storage diseases, S standard of care (generally indicated in suitable patients), SAA severe aplastic anaemia, SCD sickle cell disease (high risk), SCID severe combined immunodeficiencies, X-ALD X-linked adrenoleukodystrophy … Among PSD, allo-HCT is effective at preventing disease progression in the childhood, or rarely adult, presentation of cerebral inflammatory disease of X-linked adrenoleukodystrophy (X-ALD). …