Study of ANPD001 Autologous Neuronal Cell Replacement Treatment Approach Published in Journal of Neurosurgery
Study of ANPD001 Autologous Neuronal Cell Replacement Treatment Approach Published in Journal of Neurosurgery
Primate Study Demonstrates Safety and Feasibility of Cell Transplantation Approach
Currently in Phase 1/2a First-in-Human Parkinson's Disease Clinical Trial
电芯移植方法的安全性和可行性已经得到灵长类动物研究的证明。
目前正在进行1/2a阶段的第一项帕金森氏病临床试验。
SAN DIEGO, July 29, 2024 /PRNewswire/ -- The Journal of Neurosurgery has published online a study by the Wisconsin National Primate Research Center at the University of Wisconsin, Madison regarding a novel cell transplantation approach being used for delivery of ANPD001, an autologous, dopaminergic neuronal cell replacement under investigation by Aspen Neuroscience as a potential treatment for Parkinson's Disease.
2024年7月29日圣迭戈消息/美通社/--美国威斯康星大学麦迪逊分校威斯康星国家灵长类动物研究中心在神经外科杂志上在线发布了一项研究,介绍了一种用于传递ANPD001的新型移植细胞方法,这是一种由Aspen Neuroscience正在研究的自体多巴胺能神经元移植替代物,用于治疗帕金森氏病的潜在治疗方法。研究表明, ANPD001的治疗方法在非人类灵长类动物中是安全可行的。Aspen目前正在开展ASPIRO试验,一项面向中度至重度帕金森氏病的一项首次一类,开放标签的1/2a临床试验,以调查ANPD001。
The study by the Wisconsin National Primate Research Center demonstrated the safety and feasibility of the treatment approach for ANPD001 in non-human primates. Aspen is currently investigating ANPD001 in the ASPIRO trial, a first-in-human, open-label Phase 1/2a clinical trial in people with moderate to severe Parkinson's disease.
威斯康星州国家灵长类动物研究中心的研究表明,ANPD001的治疗方法在非人类灵长类动物中是安全可行的。Aspen目前正在开展ASPIRO试验,一项面向中度至重度帕金森氏病的一项首次一类,开放标签的1/2a临床试验,以调查ANPD001。
Study Demonstrates Safety & Feasibility of Cell Transplant Approach Currently in Phase 1/2a Parkinson's Clinical Trial
目前正在进行第1/2a期帕金森氏病临床试验的移植细胞治疗方法的安全性和可行性已经得到证明。
The study was also incorporated into Aspen's Investigational New Drug (IND) application with the U.S. Food & Drug Administration (FDA), enabling the company to proceed with the clinical trial of ANPD001.
该研究还被纳入Aspen提交的美国食品和药物管理局(US FDA)的新药申请(IND)中,为该公司进行ANPD001临床试验铺平了道路。
University of Wisconsin researcher Marina Emborg, M.D, PhD, professor, Department of Medical Physics, led the study. A previous study by Dr. Emborg, and her colleagues, published in 2021, demonstrated that an autologous neuronal replacement therapy reversed Parkinsons-like symptoms in primates.
威斯康星大学马里纳·恩伯格(Marina Emborg), 博士, 医学物理系副教授带领这个研究。恩伯格博士及其同事在2021年发表的一项先前研究表明,自体神经元替代疗法能够逆转灵长类动物中类似帕金森氏病的症状。
"The results of the study established that this approach can be used to safely target neuronal replacement therapy with precision to the brain and supports its clinical investigation as a procedure for cell replacement therapy in neurodegenerative diseases," concluded Dr. Emborg.
"该研究的结果表明,这种方法可以安全地精确地靶向神经元替代疗法,支持将其作为神经退行性疾病细胞替代疗法的手段进行临床研究," 恩伯格博士总结道。
"The study was an important step in our work to bring the promise of a cell-replacement therapy to people with Parkinson's disease," said Andrés Bratt-Leal, PhD, study co-author, Aspen Neuroscience co-founder and senior vice president of research and development. "The results were instrumental in opening our first-in-human trial and informing how we deliver patients' own cells."
"该研究是我们迈向为帕金森氏病患者带来神经元替代疗法承诺的重要一步,"安德烈斯·布拉特-利尔(Andrés Bratt-Leal),博士,研究合著者,Aspen神经科学公司联合创始人,研究和开发高级副总裁说。"该结果对于开启我们的首个人类试验和研究我们如何输送患者自己的细胞具有重要作用。"
ANPD001 begins with a small sample of the patient's own skin cells, followed by reprogramming to induced pluripotent stem cells (iPSCs) which can become any type of cell. The iPSCs are then differentiated into dopaminergic neuron precursor cells (DANPCs), which are transplanted under real-time MRI guidance into the putamen region of the brain, replacing cells that were lost or damaged due to disease.
ANPD001始于患者自己的小样皮肤细胞,随后重新编程为诱导性多能干细胞(iPSCs),这些干细胞可以变成任何类型的细胞。iPSCs随后分化为多巴胺能神经元前体细胞(DANPCs),在实时磁共振成像引导下移植到脑的壳核区域,取代因疾病而失去或受损的细胞。
"By the time of diagnosis, it is common for people with Parkinson's to have lost the majority of dopaminergic neurons, leading to progressive loss of motor and neurological function," explained Edward Wirth III, MD, PhD, study co-author and Aspen's chief medical officer. "To replace these lost cells, we must target a very specific area of the brain with a high degree of surgical precision. Utilizing the latest advances in intraoperative MRI guided techniques, the patient's new cells are transplanted, a few microliters at a time, to the exact area where they are most needed."
"在诊断时,帕金森氏病患者通常已经失去了大多数多巴胺能神经元,导致进行性失去运动和神经功能。"Edward Wirth III, MD, PhD,研究合著者,Aspen首席医疗官解释道。"为了替换这些失去的细胞,我们必须以非常具体的方式,在高度手术精度的条件下定位到脑的一个非常特定的区域。利用内部手术中磁共振引导技术的最新进展,患者的新细胞被移植,每次只有几微升,到它们最需要的确切区域。"
Affecting more than one million Americans, PD is a neurodegenerative disorder that causes walking and motor problems, as well as impaired balance and coordination. Existing therapies alleviate symptoms but do not treat the underlying disease process, leading to a significant unmet medical need for those suffering from this chronic condition.
作用于一百多万美国人的帕金森氏病是一种神经退行性疾病,会导致步行和运动问题,以及平衡和协调能力受损。现有的治疗方法可以减轻症状,但不能治疗潜在的疾病过程,导致这种慢性病的治疗还存在诸多医学需求。
About the ASPIRO Trial
关于ASPIRO试验
The Autologous-derived Study of a Parkinson's Investigational Regenerative therapy in an Open-label trial (ASPIRO) is a Phase 1/2a clinical trial to assess the safety, tolerability, and potential efficacy of ANPD001 in patients with moderate to severe Parkinson's disease. The primary study endpoint is safety and tolerability of ANPD001. Secondary endpoints include improvement in "on" time, when patients experience periods of symptom control, and improvements in motor symptoms and quality of life based on standard Parkinson's disease rating scales.
ASPIRO(自体来源的帕金森氏病疗法研究)是一项1/2a期的临床试验,旨在评估ANPD001在中度至严重帕金森氏病患者中的安全性、耐受性和潜在疗效。主要研究终点是ANPD001的安全性和耐受性。次要终点包括基于标准帕金森氏病评分表的"ON"时间改善、运动症状和生活质量的改善。
About Aspen Neuroscience
Aspen Neuroscience,总部位于圣地亚哥,致力于自体再生医学。该公司的患者衍生iPSC平台用于创建个性化治疗方法,这是处理具有高未满足医学需求疾病的关键步骤,首先是为帕金森氏病进行自体神经元替代。Aspen将细胞生物学与最新的机器学习和基因组学方法相结合,研究特定于患者的,恢复性的细胞治疗方法。该公司的一流平台表现出卓越的质量,包括内部生物信息学、制造和质量控制,用于创建和优化多能干细胞衍生的细胞治疗方法。
Aspen Neuroscience, Inc., headquartered in San Diego, is dedicated to autologous regenerative medicine. The company's patient-derived iPSC platform is used to create personalized therapies, a crucial step in addressing diseases with high unmet medical needs, starting with autologous neuron replacement for Parkinson's disease.
Aspen Neuroscience,总部位于圣迭戈,致力于自体再生医学。该公司的患者衍生iPSC平台用于创建个性化治疗方法,这是处理具有高未满足医学需求疾病的关键步骤,首先是为帕金森氏病进行自体神经元替代。Aspen将细胞生物学与最新的机器学习和基因组学方法相结合,研究特定于患者的,恢复性的细胞治疗方法。该公司的一流平台表现出卓越的质量,包括内部生物信息学、制造和质量控制,用于创建和优化多能干细胞衍生的细胞治疗方法。
Aspen combines cell biology with the latest machine learning and genomic approaches to investigate patient-specific, restorative cell treatments. The company's unwavering commitment to quality is evident in its best-in-class platform to create and optimize pluripotent-derived cell therapies, which includes in-house bioinformatics, manufacturing, and quality control.
Aspen将细胞生物学与最新的机器学习和基因组学方法相结合,研究特定患者的、恢复性的细胞治疗方案。该公司在创建和优化诱导多能细胞治疗方案的平台方面保持了无与伦比的质量承诺,包括内部生物信息学、制造业、和质量控制。
SOURCE Aspen Neuroscience, Inc.
资料来源Aspen Neuroscience, Inc。