A Phase II, Multicenter, Randomized and Controlled Open-Label Trial Comparing the Safety and Efficacy of Bilateral Intraputaminal (IPu) Administration of CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN]) Combined with Best Medical Therapy (BMT) versus BMT-alone in Subjects With Idiopathic Parkinson’s Disease.

开始日期2008-11-11

申办/合作机构

Ceregene, Inc.

EUCTR2007-006721-27-AT / Not yet recruitingN/A

开始日期2008-10-20

申办/合作机构

Ceregene, Inc.

NCT00400634 / Completed临床2期

Multicenter, Randomized, Double-Blind, Sham Surgery-Controlled Study of CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN]) to Assess the Efficacy and Safety of Bilateral Intraputaminal (IPu) Delivery in Subjects With Idiopathic Parkinson's Disease

The purpose of this double blind study is to determine whether CERE-120 (adeno-associated virus serotype 2 [AAV2]-neurturin [NTN]) is effective and safe in the treatment of patients with idiopathic Parkinson's Disease. CERE-120 is administered via bilateral stereotactic injections targeting the putaminal region of the brain. The design of this study involves approximately 34 patients receiving CERE-120 treatment via stereotactic surgery and approximately 17 patients receiving sham stereotactic surgery (no CERE-120 administered).

开始日期2006-11-01

申办/合作机构

Sangamo Therapeutics, Inc.

[+1]

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项与 Ceregene, Inc. 相关的文献（医药）

2015-06-01·Neurobiology of disease2区 · 医学

Post-mortem assessment of the short and long-term effects of the trophic factor neurturin in patients with α-synucleinopathies

2区 · 医学

Article

作者: Lang, A E ; Bartus, R T ; Brown, L ; Kordower, J H ; Kruegel, B R ; Baumann, T L ; Herzog, C D ; Olanow, C W ; Chu, Y ; Johnson, E M

Substantial interest persists for developing neurotrophic factors to treat neurodegenerative diseases. At the same time, significant progress has been made in implementing gene therapy as a means to provide long-term expression of bioactive neurotrophic factors to targeted sites in the brain. Nonetheless, to date, no double-blind clinical trial has achieved positive results on its primary endpoint despite robust benefits achieved in animal models. A major issue with advancing the field is the paucity of information regarding the expression and effects of neurotrophic factors in human neurodegenerative brain, relative to the well-characterized responses in animal models. To help fill this information void, we examined post-mortem brain tissue from four patients with nigrostriatal degeneration who had participated in clinical trials testing gene delivery of neurturin to the putamen of patients. Each had died of unrelated causes ranging from 1.5-to-3-months (2 Parkinson's disease patients), to 4+-years (1 Parkinson's disease and 1 multiple-system atrophy-parkinsonian type patient) following gene therapy. Quantitative and immunohistochemical evaluation of neurturin, alpha-synuclein, tyrosine hydroxylase (TH) and an oligodendroglia marker (Olig 2) were performed in each brain. Comparable volumes-of-expression of neurturin were seen in the putamen in all cases (~15-22%; mean=18.5%). TH-signal in the putamen was extremely sparse in the shorter-term cases. A 6-fold increase was seen in longer-term cases, but was far less than achieved in animal models of nigrostriatal degeneration with similar or even far less NRTN exposure. Less than 1% of substantia nigra (SN) neurons stained for neurturin in the shorter-term cases. A 15-fold increase was seen in the longer-term cases, but neurturin was still only detected in ~5% of nigral cells. These data provide unique insight into the functional status of advanced, chronic nigrostriatal degeneration in human brain and the response of these neurons to neurotrophic factor stimulation. They demonstrate mild but persistent expression of gene-mediated neurturin over 4-years, with an apparent, time-related amplification of its transport and biological effects, albeit quite weak, and provide unique information to help plan and design future trials.

2015-01-01·Neural regeneration research2区 · 医学

Gene therapy for Parkinson′s disease: a decade of progress supported by posthumous contributions from volunteer subjects

2区 · 医学

ArticleOA

作者: Bartus, Raymond T

Over the past decade,nine separate gene therapy clinical trials for advanced Parkinson’s disease(PD)have been launched and completed,involving the dosing of nearly 12-dozen PD volunteers who incurred significant risks to hopefully reduce symptoms and

2012-11-01·Neurobiology of disease2区 · 医学

Translating the therapeutic potential of neurotrophic factors to clinical ‘proof of concept’: A personal saga achieving a career-long quest

2区 · 医学

Review

作者: Bartus, Raymond T

While the therapeutic potential of neurotrophic factors has been well-recognized for over two decades, attempts to translate that potential to the clinic have been disappointing, largely due to significant delivery obstacles. Similarly, gene therapy (or gene transfer) emerged as a potentially powerful, new therapeutic approach nearly two decades ago and despite its promise, also suffered serious setbacks when applied to the human clinic. As advances continue to be made in both fields, ironically, they may now be poised to complement each other to produce a translational breakthrough. The accumulated data argue that gene transfer provides the 'enabling technology' that can solve the age-old delivery problems that have plagued the translation of neurotrophic factors as treatments for chronic central nervous system diseases. A leading translational program applying gene transfer to deliver a neurotrophic factor to rejuvenate and protect degenerating human neurons is CERE-120 (AAV2-NRTN). To date, over two dozen nonclinical studies and three clinical trials have been completed. A fourth (pivotal) clinical trial has completed all dosing and is currently evaluating safety and efficacy. In total, eighty Parkinson's disease (PD) subjects have thus far been dosed with CERE-120 (some 7 years ago), representing over 250 cumulative patient-years of exposure, with no serious safety issues identified. In a completed sham-surgery, double-blinded controlled trial, though the primary endpoint (the Unified Parkinson's Disease Rating Scale (UDPRS) motor off score measured at 12 months) did not show benefit from CERE-120, several important motor and quality of life measurements did, including the same UPDRS-motor-off score, pre-specified to also be measured at a longer, 18-month post-dosing time point. Importantly, not a single measurement favored the sham control group. This study therefore, provided important, well-controlled evidence establishing 'clinical proof of concept' for gene transfer to the CNS and the first controlled evidence for clinical benefit of a neurotrophic factor in a human neurodegenerative disease. This paper reviews the development of CERE-120, starting historically with the long-standing interest in the therapeutic potential of neurotrophic factors and continuing with selective accounts of past efforts to translate their potential to the clinic, eventually leading to the application of gene transfer and its role as the 'enabling technology'. Because of growing interest in translational R&D, including its practice in industry, the paper is uniquely oriented from the author's personal, quasi-autobiographic perspective and career-long experiences conducting translational research and development, with a focus on various translational neurotrophic factor programs spanning 30+ years in Big Pharma and development-stage biotech companies. It is hoped that by sharing these perspectives, practical insight and information might be provided to others also interested in translational R&D as well as neurotrophic factors and gene therapy, offering readers the opportunity to benefit from some of our successes, while possibly avoiding some of our missteps.

项与 Ceregene, Inc. 相关的新闻（医药）

2022-07-25

·PRNewswire

NEUVOGEN Appoints Stephen A. Sherwin, M.D. to its Scientific Advisory Board

SAN DIEGO, July 25, 2022 /PRNewswire/ -- NEUVOGEN, an immuno-oncology company with a next-generation cancer vaccine platform, is pleased to announce the appointment of Stephen A. Sherwin, M.D., to the company's Scientific Advisory Board (SAB). "We are extremely honored to have Dr. Sherwin join NEUVOGEN's Scientific Advisory Board" said Todd Binder, Chief Executive Officer of NEUVOGEN. "He is both a practicing oncologist and biotech executive with broad experience in cancer immunotherapy including cancer vaccines, ranging from whole tumor cell to personalized neoantigen approaches. We believe that his joining the SAB validates the promise of NEUVOGEN's cancer vaccine platform." Binder continued, "We look forward to benefitting from the experience and expertise of a highly accomplished immuno-oncology pioneer to help guide our clinical development programs, regulatory and business strategies and partnership discussions." Stephen A. Sherwin, M.D., has spent over 35 years in the biotechnology industry, helping to create and manage companies that discover and develop new treatments for patients with cancer. Dr. Sherwin was the first medical doctor hired by Genentech, Inc. and served as its Vice President of Clinical Research. During his career, Dr. Sherwin co-founded and served as Chief Executive Officer and/or Chairman of industry-leading companies. Dr. Sherwin served as the Chief Executive Officer of Cell Genesys, Inc., an immuno-oncology company that developed GVAX (a first-generation whole tumor cell vaccine) and first-generation CAR T cells, and was Chairman from 1994 until the company's merger in 2009 with BioSante Pharmaceuticals, Inc. (now ANI Pharmaceuticals, Inc.). He co-founded and served as Chairman of Abgenix, Inc., an antibody company focused on oncology that was acquired by Amgen Inc. for $2.2 billion in 2006. He co-founded and served as Chairman of Ceregene, Inc., a gene therapy company acquired by Sangamo Therapeutics, Inc. in 2013. He served on the Board of Directors of Neon Therapeutics, Inc., an immuno-oncology company developing both personal and shared neoantigen targeted cancer vaccines and T cell modalities, until its merger with BioNTech SE in 2020. Dr. Sherwin currently serves as a member of the Scientific Steering Committee of the Parker Institute for Cancer Immunotherapy. He also serves as an Advisory Partner at Third Rock Ventures, a leading healthcare venture firm, and as a director of Biogen Inc. and Neurocrine Biosciences, Inc. In addition to his corporate experience, Dr. Sherwin has also served on the Board of Directors of the Biotechnology Innovation Organization from 2001 to 2014, serving as Chairman from 2009 to 2011, been on the staff of the National Cancer Institute, and served on the President's Council of Advisors in Science and Technology Working Group on Drug Development from 2011 to 2013. Dr. Sherwin is a Clinical Professor of Medicine at the University of California, San Francisco, and a volunteer Attending Physician in Hematology-Oncology at the Zuckerberg San Francisco General Hospital. Dr. Sherwin holds a B.A. in Biology, summa cum laude, from Yale University and an M.D. from Harvard Medical School, is board-certified in Internal Medicine and Medical Oncology, and is a Fellow of the American College of Physicians. "I am excited to join the NEUVOGEN SAB and bring my past experience in immunotherapy and cancer vaccines to NEUVOGEN. Cancer vaccines have the potential to be a fundamental and powerful modality in the fight against cancer. By building upon previous cancer vaccine efforts, NEUVOGEN hopes its vaccines can fulfill that potential" said Dr. Sherwin. A link to comprehensive biographies can be found at . About NEUVOGEN NEUVOGEN, Inc. is an immuno-oncology company focused on whole cell cancer vaccine therapies. NEUVOGEN believes its cancer vaccines have the broadest combination of tumor associated antigens and tumor specific antigens ever delivered in a cancer vaccine and as a result can directly target a greater percentage of cancer cells in a solid tumor than any previous cancer vaccine. Based in San Diego, California, NEUVOGEN's goal is to develop therapeutic cancer vaccines that activate the body's immune system to eradicate solid tumors and meaningfully extend the lives of patients while limiting side effects. To learn more, visit . SOURCE NEUVOGEN Inc.

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药物(靶点)	适应症	全球最高研发状态

CERE-120 ( NRTN )	亨廷顿舞蹈病更多	临床前
CERE-130 ( IGF-1 )	肌萎缩侧索硬化更多	临床前
Nerve growth factor(Amgen, Inc.) ( p75NTR )	阿尔茨海默症更多	终止
CERE-110 ( NGF x p75NTR )	阿尔茨海默症更多	终止
GDNF gene therapy (Ceregene)	亨廷顿舞蹈病更多	无进展