초록 ▼

뇌종양의 치료기능을 지닌 항암성 줄기세포치료제개발을 위해 다음과 같은 연구를 수행하였음.
1) 뇌종양치료제의 효력시험 기준 및 시험법 확립
2) 뇌종양치료제 마스터세포은행(MCB)의 특성분석완료
3) 임상시험제품 생산 공정 개발 및 임상시험을 위한 시제품생산 (GMP)완료
4) 임상시험제품 특성분석/안정성시험(24개월)/임상시험제품 효력 시험완료
5) 안전성평가를 위한 체내분포시험, GLP 독성평가, GLP 종양원성평가시험 완료
6) 임상시험계획서 작성 및 연구자임상시험승인 (IIT) 신청완료

뇌종양의 치료기능을 지닌 항암성 줄기세포치료제개발을 위해 다음과 같은 연구를 수행하였음.
1) 뇌종양치료제의 효력시험 기준 및 시험법 확립
2) 뇌종양치료제 마스터세포은행(MCB)의 특성분석완료
3) 임상시험제품 생산 공정 개발 및 임상시험을 위한 시제품생산 (GMP)완료
4) 임상시험제품 특성분석/안정성시험(24개월)/임상시험제품 효력 시험완료
5) 안전성평가를 위한 체내분포시험, GLP 독성평가, GLP 종양원성평가시험 완료
6) 임상시험계획서 작성 및 연구자임상시험승인 (IIT) 신청완료
7) 뇌종양의 치료기능을 지닌 항암성 줄기세포치료제개발기술의 산업체로의 기술이전완료 및 상업화임상시험 (SIT) 준비

(출처 : 보고서 요약서 3p)

Abstract ▼

Purpose&Contents
○ Glioblastoma multiform (GBM) is the most severe cancer in the nervous system. Despite the progress has been made in treating GBM, the most effective therapy including combination of chemotherapy and radiotherapy has remained as a palliative cure. The aim of this study is to dev

Purpose&Contents
○ Glioblastoma multiform (GBM) is the most severe cancer in the nervous system. Despite the progress has been made in treating GBM, the most effective therapy including combination of chemotherapy and radiotherapy has remained as a palliative cure. The aim of this study is to develop an innovative stem cell therapy for the treatment of GBM patients.

○ Genetically modified stem cells have been developed as anti-cancer drugs through 4 steps: 1)production of viral vector, 2) production of mesenchymal stem cells as drug substance 3) manufacturing of master cell bank of genetically modified stem cells with anti-cancer functions, 4) production of clinical lots. The first 1~3 steps were fullfilled in the previous studies.

○ In this projects, we produce the GMP-grade clinical lots, assess the ove the safety through preclinical studies in a GLP-facility as well as the efficacy. This projects include the final steps for TRL5: preparation of IND filing for the future Phase I clinical trial. Briefly

1) To prove the in vitro and in vivo efficacy, safety/toxicity, dose escalation, pharmacodynamics.
2) To develop manufacturing processes for GMP production and standard operating procedures
3) To determine the target product profile and develop the storage/stability evaluation methods
4) To develop large-scale cultivation methods
5) To design clinical trial: to plan protocol, target patients, enrollment criteria, safety assessments, toxicity monitoring, dose adjustment rules, adverse event recording and reporting, primary endpoints
6) IND application with submitting a new clinical protocol

Results
○ The SOP's for evaluating the efficacy/characterization/toxicity/pharmacology have been established.

○ The manufacturing processes for pilot-scale GMP-production have been established.
○ The clinical lots have been produced in a GMP-facility and the toxicity tests have been finished in a GLP-institute.

○ The target product profile and the clinical protocol has been designed.
○ IND application is on going for future phase I clinical trial
○ SCI paper, 1; Patent application 11; Patent approved, 1 (EU); Technology transfer 1

Expected Contribution
○ These allogeneic cells with anti-cancer functions may be used neoadjuvant therapy to treat GBM patients.

○ These cells can be produced a in large scale through simple expansion of master cell bank to obtain the clinical lots. The cells are packaged as off-the shelf drugs, which can be administered on-demand.

○ These cells can be applied to non-brain cancers once debulking solid-cancers.

(출처 : SUMMARY 5p)

목차 Contents

• 표지 ... 1
• 제 출 문 ... 2
• 보고서 요약서 ... 3
• 국문 요약문 ... 4
• 영문 요약문 ... 5
• CONTENTS ... 6
• 목차 ... 7
• 1. 연구개발과제의 개요 ... 8
• 1-1. 연구개발 목적 ... 8
• 1-2. 연구개발의 필요성 ... 11
• 1-3. 연구개발 범위 ... 18
• 2. 국내외 기술개발현황 ... 22
• 3. 연구수행 내용 및 결과 ... 27
• 3.1 정량적 연구성과 ... 27
• 3.2 정성적 연구성과 ... 32
• 4. 목표달성도 및 관련분야 기여도 ... 87
• 4.1 목표달성도 ... 87
• 4.2 관련분야기여도 ... 92
• 5. 연구결과의 활용계획 ... 94
• 6. 연구과정에서 수집한 해외과학기술정보 ... 95
• 7. 연구개발성과의 보안등급 ... 100
• 8. 국가과학기술종합정보시스템에 등록한 연구시설·장비 현황 ... 100
• 9. 연구개발과제 수행에 따른 연구실 등의 안전조치 이행실적 ... 100
• 10. 연구개발과제의 대표적 연구실적 ... 101
• 11. 참고문헌 ... 102
• [별첨4] 실적 증빙자료 ... 104
• 끝페이지 ... 134