Current Japanese Regulation for Cell Therapy and NCC strategy
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1 Current Japanese Regulation for Cell Therapy and NCC strategy National Cancer Center Hospital Department of Medical Oncology Division of Cell and Gene Therapy Yuji Heike
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3 Today s topics 1)Current Regulation of Cellular Therapy in Japan 2)Preclinical studies for cellular therapy clinical trials in NCC
4 Laws and Guidelines related to cell therapies in Japan..... The Guidelines for Gene Therapy Clinical Trials The Law Concerning Regulation Relating to Human Cloning Techniques and Other Similar Techniques The Guidelines for Handling of a Specified Embryo The Guidelines for Derivation and Utilization of Human Embryonic Stem Cells Clinical Research The Guidelines for Clinical Trials using Human Stem Cells Basic Research By The Ministry of Health, Labour and Welfare By The Ministry of Education, Culture, Sports, Science and Technology
5 Two styles of Cellular Therapy Clinical Studies Academia Trial Sponsored Clinical Trial Purpose Verification of Treatment Efficacy & Improvement GCP Study of the investigational new drug Procedure Acceptance from Institution s Ethical Committees IND Filing (same as the ordinary drugs) Institution s IND review board Application to Authorities -Application for Clinical Research Plan of Gene Therapy -Confirmation application for Cartagena Protocol - Confirmation Application for the Verification of Quality & Safety -Confirmation Application for Cartagena Protocol
6 Regulation for Cellular Therapy in two types of Clinical Trial Academia Clinical Trials Sponcered Clinical Trials Somatic Cell Therapy No rule Stem Cell Therapy Adult Stem Cell Embryonic Stem Cell Follow the guideline No clinical guideline Follow the law--central Review are required after IRB (Basic Research) IND Filing Cellar Gene Therapy Follow the Guideline and Central Review after IRB approved
7 Review Process of Sponsored Cellular Therapy Clinical Trials Regular Drugs Investigational New Drug (IND) Application Clinical trial New Drug Application Non-clinical studies Cellular Therapy Confirmation Application
8 Five projects of cellular therapy in NCC 1) Donor lymphocyte infusion against relapsed hematological malignancy after allogeneic stem cell transplantation (Sponsered Trial) 2) Haploidentical CD34 positive cell transplantation combined with HSV-TK gene modified lymphocyte infusion (Academia Trial) 3) αgalcer stimulated mononuclear cell therapy against chemotherapy resistant cancers (Academia Trial) 4) Adoptive Transfer of RetroNectin Stimulated Lymphocyte against chemotherapy resistant cancers (Academia Trial) 5) RetroNectin Stimulated Donor Lymphocyte infusion against relapsed-hematological malignant patients after allogeneic stem cell transplantation. (Academia Trial)
9 Modeling Translational Research in Cellular Therapy (1) Donor lymphocyte infusion (DLI) therapy against relapsed hematological malignancy after allogeneic stem cell transplantation (Sponsored by Takara Bio. Co Ltd)
10 Therapeutic Approach Lymphocyte Isolation and Activation Infusion Lymphocyte Expansion Transduction with HSV-TK gene and ΔLNGFR gene by a retrovirus vector Selection with specific mab
11 Review Process of Sponsored Cellular Therapy Clinical Trials Cellular Therapy Investigational New Drug (IND) Application Clinical trial New Drug Application Non-clinical studies Confirmation Application
12 Procedure for IND filing of Cellular/Gene Therapy Gene Vectors Gene Manipulated Cells Cellular Drugs Notification # 1062 Notification #1314 Notification #906
13 Cell Processing Room Zone A: Class100 Zone B: Class10,000 Zone B: Class100,000
14 Modeling Translational Research in Cellular Therapy (2) Adoptive transfer of αgalcer+il2 stimulated MNCs against chemo-resistant solid trumors (Academia Tirals)
15 Regulation for Cellular Therapy in two types of Clinical Trial Academia Clinical Trials Sponcered Clinical Trials Somatic Cell Therapy No rule Stem Cell Therapy Adult Stem Cell Embryonic Stem Cell Follow the guideline No clinical guideline Follow the law--central Review are required after IRB (Basic Research) Confirmation Application Cellar Gene Therapy Follow the Guideline and Central Review after IRB approved
16 Basic Research Pre-clinical Research Phase Ⅰ Ⅱa Ⅱb Ⅲ Pharmaceutical Company In case of Japan The same inspector follows all process of one IND as a package In case of USA Academia FDA review all INDs (GMP products) in all sponcered- and academia trals
17 Therapeutic Approach of agalcer+il2 stimulated MNC Therapy Infusion Monocyte Isolation Expansion αgalcer + IL-2
18 Basic Research Pre-clinical Research Phase Ⅰ Ⅱa Ⅱb Ⅲ Pharmaceutical Company Academia
19 National Cancer Center Hospital Yoichi Takaue MD, Yuji Heike MD PhD Takara Bio. Kohjin Bio. Fukoku Mynervatec Kirin Lab. in clinic Immune Monitoring Team Atsuko Soeda MD Clinical Trial Team Clinic Clinical Staff Fellows Nursing team Pharmacologist National Cancer Research Institute Teruhiko Yoshida MD Kazunori Aoki MD Shizuka Yamagata Molecular Biology Team Yukie Hotate Rui Kuroda Hiroaki MakiyamanMD Noriko Takahashi Cell Processing Team In-house Data Center Minuko Tajima Harumi Takahashi Shinobu Saito Tokyo SCT consotium Toranomon Hospital etc
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