DUOC-01(DUOC-01) - 在研适应症：原发性进行性多发性硬化，肾上腺脑白质营养不良，α-甘露糖苷贮积症_专利_临床

概要

基本信息

药物类型

干细胞疗法

别名-

靶点-

作用机制

细胞替代物

治疗领域

免疫系统疾病神经系统疾病遗传病与畸形+ [4]

在研适应症

原发性进行性多发性硬化肾上腺脑白质营养不良α-甘露糖苷贮积症+ [9]

非在研适应症-

原研机构

Duke University

在研机构

Duke University

非在研机构-

最高研发阶段临床1期

首次获批日期-

最高研发阶段(中国)-

特殊审评-

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关联

2

项与 DUOC-01 相关的临床试验

NCT04943289 / Recruiting临床1期IIT

Phase IA Trial of Intrathecal Administration of Human Umbilical Cord Blood-Derived Cell Therapy (DUOC-01) in Adults With Primary Progressive Multiple Sclerosis (PPMS)

This study is a prospective Phase 1a open-label single- center trial. It will assess the safety of intrathecal administration of DUOC-01 cells to adults with Primary Progressive Multiple Sclerosis (PPMS). DUOC-01 is a population of cells expanded from donated human umbilical cord blood cells and is intended for treatment of neurodegenerative and demyelinating diseases. There will be approximately 20 participants enrolled. Exploratory objectives include changes in MS assessment scores, changes in brain MRI findings, and changes in blood biomarkers.

开始日期2021-01-24

申办/合作机构

Duke University

NCT02254863 / Recruiting临床1期IIT

Augmentation of Umbilical Cord Blood Transplantation for Inherited Metabolic Diseases with Intrathecal Administration of Human Umbilical Cord Blood-Derived Oligodendrocyte-Like Cells

The primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 as an adjunctive therapy in patients with inborn errors of metabolism who have evidence of early demyelinating disease in the central nervous system (CNS) who are undergoing standard treatment with unrelated umbilical cord blood transplantation (UCBT). The secondary objective of the study is to describe the efficacy of UCBT with intrathecal administration of DUOC-01 in these patients.

开始日期2014-09-01

申办/合作机构

Duke University

100 项与 DUOC-01 相关的临床结果

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100 项与 DUOC-01 相关的转化医学

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100 项与 DUOC-01 相关的专利（医药）

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5

项与 DUOC-01 相关的文献（医药）

2023-04-01·Stem Cells and Development

Cellular Therapies: Yesterday, Today, and Tomorrow

Article

作者: Mayani, Hector

Cellular therapy (CT) can be defined as the transference into a person of healthy cells to correct defective functions. Yesterday (1950-2010), CT consisted mostly of hematopoietic transplants for the treatment of a variety of hematological disorders. Interestingly, during that period of time other cell types with therapeutic potential-including certain lymphoid populations and other nonhematopoietic cells-were discovered and characterized; thus, CT became a promising discipline for the treatment of a broader diversity of diseases. Today (2011-2023), CT has significantly grownup through preclinical studies and clinical trials, and it is currently progressing toward its consolidation as one of the pillars of medicine in the 21st century. Indeed, different types of stem cells (e.g., hematopoietic, mesenchymal, neural, and pluripotent), as well as different lymphoid and myeloid cell populations (e.g., TILs, CAR-Ts, CAR-NKs, and DUOC-01) are being used in clinical settings or are being tested in clinical trials. For the past decade, several CT modalities have been developed, and today, many of them are being used in the clinic. Tomorrow (2024-2040), already established CT modalities will surely be improved and applied more frequently, and novel therapies (that will include cell types such as iPSCs) will enter and expand within the clinical ground. It is noteworthy, however, that despite significant advancements and achievements, problems still need to be solved and obstacles need to be overcome. Technical, ethical, and economic issues persist and they need to be addressed. Undoubtedly, exciting times of challenges and opportunities are coming ahead in the CT arena.

2017-06-01·Cytotherapy3区 · 医学

Gene products promoting remyelination are up-regulated in a cell therapy product manufactured from banked human cord blood

3区 · 医学

Article

作者: Noeldner, Pamela ; Saha, Arjun ; Kurtzberg, Joanne ; Buntz, Susan ; Balber, Andrew E ; Scotland, Paula ; Gentry, Tracy

BACKGROUND AIMSDUOC-01, a cell product being developed to treat demyelinating conditions, is composed of macrophages that arise from CD14⁺ monocytes in the mononuclear cell (MNC) population of banked cord blood (CB). This article demonstrates that expression of multiple gene products that promote remyelination is rapidly up-regulated during manufacturing of DUOC-01 from either MNC or purified CB CD14⁺ monocytes.METHODSCell cultures were initiated with MNC or with immunoselected CD14⁺ monocytes isolated from the same CB unit. Cell products present in these cultures after 2 and 3 weeks were compared by three methods. First, quantitative polymerase chain reaction was used to compare expression of 77 transcripts previously shown to be differentially expressed by freshly isolated, uncultured CB CD14⁺ monocytes and DUOC-01. Second, accumulation of 16 soluble proteins in the culture medium was measured by Bioplex methods. Third, whole transcriptomes of the cell products were compared by microarray analysis.RESULTSKey transcripts in multiple pathways that promote remyelination were up-regulated in DUOC-01, and substantial secretion of proteins corresponding to many of these transcripts was detected. Cell products manufactured from MNC or from CD14⁺ monocytes were similar with regard to all metrics. Upregulation of gene products characteristic of DUOC-01 was largely completed within 14 days of culture.CONCLUSIONWe demonstrate that expression of multiple gene products that promote remyelination is up-regulated during the first 2 weeks of manufacturing of DUOC-01. Measuring these mechanistically important transcripts and proteins will be useful in monitoring manufacturing, evaluating manufacturing changes, and developing mechanism-based product potency assays.

2015-09-01·Cytotherapy

Reprint of: Preclinical characterization of DUOC-01, a cell therapy product derived from banked umbilical cord blood for use as an adjuvant to umbilical cord blood transplantation for treatment of inherited metabolic diseases

Article

作者: Storms, Robert W ; Wollish, Amy ; Wenger, David A ; Noeldner, Pamela ; Gentry, Tracy ; Kurtzberg, Joanne ; Balber, Andrew E ; Buntz, Susan ; Ozamiz, April ; Rusche, Benjamin

BACKGROUND AIMSCord blood (CB) transplantation slows neurodegeneration during certain inherited metabolic diseases. However, the number of donor cells in the brain of patients does not appear to be sufficient to provide benefit until several months after transplant. We developed the cell product DUOC-01 to provide therapeutic effects in the early post-transplant period.METHODSDUOC-01 cultures initiated from banked CB units were characterized by use of time-lapse photomicroscopy during the 21-day manufacturing process. Antigen expression was measured by means of flow cytometry and immunocytochemistry; transcripts for cytokines and enzymes by quantitative real-time polymerase chain reaction; activities of lysosomal enzymes by direct biochemical analysis; alloreactivity of DUOC-01 and of peripheral blood (PB) mononuclear cells (MNC) to DUOC-01 by mixed lymphocyte culture methods; and cytokine secretion by Bioplex assays.RESULTSDUOC-01 cultures contained highly active, attached, motile, slowly proliferating cells that expressed common (cluster of differentiation [CD]11b, CD14 and Iba1), M1 type (CD16, inducible nitric oxide synthase), and M2-type (CD163, CD206) macrophage or microglia markers. Activities of 11 disease-relevant lysosomal enzymes in DUOC-01 products were similar to those of normal PB cells. All DUOC-01 products secreted interleukin (IL)-6 and IL-10. Accumulation of transforming growth factor-β, IL-1β, interferon-γ and TNF-α in supernatants was variable. IL-12, IL-2, IL-4, IL-5 and IL-13 were not detected at significant concentrations. Galactocerebrosidase, transforming growth factor-β and IL-10 transcripts were specifically enriched in DUOC-01 relative to CB cells. PB MNCs proliferated and released cytokines in response to DUOC-01. DUOC-01 did not proliferate in response to mismatched MNC.CONCLUSIONSDUOC-01 has potential as an adjunctive cell therapy to myeloablative CB transplant for treatment of inherited metabolic diseases.

100 项与 DUOC-01 相关的药物交易

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研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
肾上腺脑白质营养不良	临床1期	美国	Duke University	2014-09-01
α-甘露糖苷贮积症	临床1期	美国	Duke University	2014-09-01
球样细胞脑白质营养不良	临床1期	美国	Duke University	2014-09-01
异染性脑白质病变	临床1期	美国	Duke University	2014-09-01
黏多醣贮积症III型	临床1期	美国	Duke University	2014-09-01
神经元蜡样质脂褐质沉积症	临床1期	美国	Duke University	2014-09-01
尼曼匹克症	临床1期	美国	Duke University	2014-09-01
Pelizaeus-Merzbacher病	临床1期	美国	Duke University	2014-09-01
山德霍夫病	临床1期	美国	Duke University	2014-09-01
泰-萨克斯病	临床1期	美国	Duke University	2014-09-01