엄장현 (에스바이오메딕스)

한빛사논문

조회388

엄장현Jang Hyeon Eom

에스바이오메딕스

Cell Stem Cell, December 11, 2023 | https://doi.org/10.1016/j.stem.2023.11.009 Preclinical and dose-ranging assessment of hESC-derived dopaminergic progenitors for a clinical trial on Parkinson's disease

Sanghyun Park,^1,7 Chan Wook Park,^1,7 Jang Hyeon Eom,^2,7 Mi-Young Jo,² Hye-Jin Hur,^1,2 Sung Kyoung Choi,² Jae Souk Lee,¹ Seung Taek Nam,² Ki-Sang Jo,² Young Woo Oh,^1,3 Jungil Lee,^1,3 Sieun Kim,^1,3 Do-Hun Kim,^1,2 Chul-Yong Park,^1,2 Su Jin Kim,⁴ Ho-Young Lee,⁴ Myung Soo Cho,² Dae-Sung Kim,^5,6,* and Dong-Wook Kim^1,2,3,8,*

¹Department of Physiology, Yonsei University College of Medicine, Seoul 03722, Republic of Korea
²S. Biomedics Co., Ltd., Seoul 04797, Republic of Korea
³Brain Korea, 21 PLUS Program for Medical Science, Yonsei University College of Medicine, Seoul 03722, Republic of Korea
⁴Department of Nuclear Medicine, Seoul National University Bundang Hospital, Seongnam 13620, Gyeonggi-do, Republic of Korea
⁵Department of Biotechnology, College of Life Sciences and Biotechnology, Korea University, Seoul 02841, Republic of Korea
⁶Department of Pediatrics, Korea University College of Medicine, Guro Hospital, Seoul 08308, Republic of Korea
⁷These authors contributed equally
⁸Lead contact

^*Corresponding authors: correspondence to Dae-Sung Kim or Dong-Wook Kim

Abstract

Human embryonic stem cell (hESC)-derived midbrain dopaminergic (mDA) cell transplantation is a promising therapeutic strategy for Parkinson’s disease (PD). Here, we present the derivation of high-purity mDA progenitors from clinical-grade hESCs on a large scale under rigorous good manufacturing practice (GMP) conditions. We also assessed the toxicity, biodistribution, and tumorigenicity of these cells in immunodeficient rats in good laboratory practice (GLP)-compliant facilities. Various doses of mDA progenitors were transplanted into hemi-parkinsonian rats, and a significant dose-dependent behavioral improvement was observed with a minimal effective dose range of 5,000–10,000 mDA progenitor cells. These results provided insights into determining a low cell dosage (3.15 million cells) for human clinical trials. Based on these results, approval for a phase 1/2a clinical trial for PD cell therapy was obtained from the Ministry of Food and Drug Safety in Korea, and a clinical trial for treating patients with PD has commenced.

논문정보

형식Research article
게재일2023년 12월 (BRIC 등록일 2023-12-12)
연구진국내 연구진
분야 의약학 > 재생의학

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