Development of effective in-office autologouscell therapy for osteoarthritis

开发治疗骨关节炎的有效诊室自体细胞疗法

• 批准号: 9909405
• 负责人: NATHAN KATZ
• 金额: $ 21.9万
• 依托单位: JOINTECHLABS, INC.
• 依托单位国家: 美国
• 财政年份: 2019
• 资助国家: 美国
• 起止时间: 2019-09-11 至 2021-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9909405
• 关键词: Address; Adipose tissue; Affect; Autologous; Automobile Driving; Biocompatible Materials; Biomimetics; Cartilage; Cell Proliferation; Cell Survival; Cell Therapy; Cells; Chronic; Clinic; Clinical; Clinical Research; Clinics and Hospitals; Data; Degenerative polyarthritis; Development; Diagnostic radiologic examination; Economic Burden; Elderly; Encapsulated; Environment; Enzyme-Linked Immunosorbent Assay; Exposure to; Fibrinogen; Foundations; Future; Gel; Gene Expression Profile; Genes; Goals; Harvest; Health; Health system; Healthcare; Histology; Human; Hyaluronic Acid; Hydrogels; In Vitro; Incubated; Inflammation; Infrastructure; Injections; Interleukin-1; Joint Instability; Joint Prosthesis; Joints; Licensing; Longevity; Lubrication; Medical; Mesenchymal Stem Cells; Methods; Modeling; Molecular; Monitor; Natural regeneration; Necrosis; Obesity; Operative Surgical Procedures; Pain; Patients; Performance; Pharmacological Treatment; Phase; Population; Preparation; Procedures; Property; Prosthesis; Protocols documentation; Recovery of Function; Regenerative Medicine; Rehabilitation therapy; Replacement Arthroplasty; Reproducibility; Research Personnel; Risk; Safety; Small Business Innovation Research Grant; Stem cells; Structure; System; Therapeutic; Therapeutic Effect; Time; Tissues; Treatment Efficacy; Universities; Validation; ankle joint; arthropathies; base; cartilage degradation; cartilage repair; cell growth; chronic pain; crosslink; cytokine; disability; effective therapy; first-in-human; glucosaminoglycans; healing; implantation; improved; in vitro Model; innovation; novel therapeutics; pain relief; point of care; portability; prevent; product development; regenerative; scaffold; treatment strategy

PROJECT SUMMARY Osteoarthritis is today a major worldwide challenge for health systems, representing the most common degenerative joint disorder and the leading cause of chronic disability among older adults. In the U.S. alone, OA affects 30 million people and it represents a $303 billion economic burden. Available pharmacological treatments only provide symptomatic pain relief and fail to arrest the progressive degeneration of cartilage associated with OA. Surgical procedures that promote cartilage repair by implantation of artificial joint structures are expensive procedures that require long rehabilitation and might expose patients to the risk of complications. JointechLabs (JTL) proposes an innovative regenerative protocol for OA that could be easily implemented at POC (Point Of Care). JTL approach to OA combines for the first time the regenerative healing properties of stem cells and the benefits of long-lasting viscosupplementation. The innovation proposed resides on the unique combination of two components developed by JTL: 1) A unique portable and disposable closed system for the preparation and harvest of Autologous Adipose-Derived Stem Cells (ADSCs) in non-controlled environments; 2) The first clinically approved viscosupplement product that can act as a scaffold to support stem cells growth. This combination will allow the articular injection of Autologous ADSCs isolated at the bedside and encapsulated in a protective hydrogel, which will support and accelerate the release of regenerative cellular factors to promote joint healing in OA. At the same time, the proprietary gel will provide lubrication for pain relief, creating a favorable environment for general therapeutic effect. The goal of this SBIR Phase I proposal is to demonstrate the feasibility of the proposed approach as a new treatment of OA. We propose the following specific aims: 1) Identification of the most favorable combination of hydrogel carrier and ADSCs concentrations to support high regenerative potential. 2) Validation of regenerative protocol using an in vitro model of inflamed human joints. At the same time, evidence of the mechanism of action will be collected at the molecular level to gain accurate information before moving forward into clinical studies. The successful completion of this SBIR Phase I project will lay the foundation of a Phase II SBIR proposal that will address key technical product developments in support of IND application that will open the way to a first-in- human clinical study to validate safety and efficacy of the treatment for OA.

项目概要 骨关节炎是当今世界范围内卫生系统面临的一项重大挑战，是最常见的疾病 退行性关节疾病是老年人慢性残疾的主要原因。仅在美国，OA 影响到 3000 万人，造成 3030 亿美元的经济负担。可用的药物治疗 只能缓解症状性疼痛，无法阻止与以下疾病相关的软骨进行性退化： 办公自动化。通过植入人工关节结构促进软骨修复的外科手术费用昂贵 需要长期康复并可能使患者面临并发症风险的手术。 JointechLabs (JTL) 提出了一种创新的 OA 再生协议，可以轻松实现 在 POC（护理点）实施。 JTL 治疗 OA 的方法首次结合​​了再生愈合 干细胞的特性以及持久粘稠补充的好处。所提出的创新在于 JTL 开发的两种组件的独特组合： 1) 独特的便携式一次性封闭式 用于在非对照条件下制备和收获自体脂肪干细胞 (ADSC) 的系统 环境； 2) 第一个临床批准的粘稠补充产品，可以作为支架支撑干 细胞生长。这种组合将允许关节注射在床边分离的自体 ADSC 封装在保护性水凝胶中，这将支持并加速再生细胞的释放 促进 OA 关节愈合的因素。同时，专有的凝胶将提供润滑作用以缓解疼痛， 为整体治疗效果创造良好的环境。 SBIR 第一阶段提案的目标是证明所提出的方法作为新方法的可行性 治疗骨关节炎。我们提出以下具体目标： 1）确定最有利的组合 水凝胶载体和 ADSC 浓度可支持高再生潜力。 2）再生验证 使用发炎的人体关节的体外模型的协议。同时，作用机制的证据 将在分子水平上收集，以便在进入临床研究之前获得准确的信息。 SBIR 第一阶段项目的成功完成将为第二阶段 SBIR 提案奠定基础 将解决支持 IND 申请的关键技术产品开发，这将为首创开辟道路 人体临床研究，以验证 OA 治疗的安全性和有效性。