Bulletin of Chinese Academy of Sciences (Chinese Version)
Keywords
Cell-based medicinal products, cell manufacture, cell therapy
Document Type
Disciplinary Development
Abstract
Cell-based medicinal products are a breakthrough that has been proven to treat previously incurable diseases, making them a topic of global interest. This review delves into the current state and progress of cell therapy, highlighting the remarkable therapeutic effects of various types of cell-based medicinal products. We specifically explore the potential development of cell-based medicinal products in China, focusing on promoting clinical translation and commercialization. We propose necessary actions to achieve these goals, such as fostering cutting-edge basic research, innovative therapeutic approaches, scalable manufacturing processes, and policy changes.
First page
1434
Last Page
1446
Language
Chinese
Publisher
Bulletin of Chinese Academy of Sciences
References
1 惠利健. 细胞治疗药物:基于作用机制的研究和应用. 中国科学:生命科学, 2023, 53(8): 1067-1071.Hui L J. Cell-based medicine: Mode-of-action-based research and application. Scientia Sinica (Vitae), 2023, 53 (8): 1067-1071. (in Chinese)
2 Williams J Z, Allen G M, Shah D, et al. Precise T cell recognition programs designed by transcriptionally linking multiple receptors. Science, 2020, 370: 1099-1104.
3 Lajoie M J, Boyken S E, Salter A I, et al. Designed protein logic to target cells with precise combinations of surface antigens. Science, 2020, 369: 1637-1643.
4 Tousley A M, Rotiroti M C, Labanieh L, et al. Co-opting signalling molecules enables logic-gated control of CAR T cells. Nature, 2023, 615: 507-516.
5 He C H, Mansilla-soto J, Khanra N, et al. CD19 CAR antigen engagement mechanisms and affinity tuning. Science Immunology, 2023, 8: eadf1426.
6 Foster M C, Savoldo B, Lau W, et al. Utility of a safety switch to abrogate CD19.CAR T-cell-associated neurotoxicity. Blood, 2021, 137(23): 3306-3309.
7 Labanieh L, Majzner R G, Klysz D, et al. Enhanced safety and efficacy of protease-regulated CAR-T cell receptors. Cell, 2022, 185(10): 1745-1763.
8 Li H S, Wong N M, Tague E, et al. High-performance multiplex drug-gated CAR circuits. Cancer Cell, 2022, 40 (11): 1294-1305.
9 Liu Y, Liu G N, Wang J S, et al. Chimeric STAR receptors using TCR machinery mediate robust responses against solid tumors. Science Translational Medicine, 2021, 13: eabb5191.
10 Zhang H H, Li F L, Cao J, et al. A chimeric antigen receptor with antigen-independent OX40 signaling mediates potent antitumor activity. Science Translational Medicine, 2021, 13: eaba7308.
11 Li W T, Qiu S Z, Chen J, et al. Chimeric antigen receptor designed to prevent ubiquitination and downregulation showed durable antitumor efficacy. Immunity, 2020, 53(2): 456-470.
12 Pecher A C, Hensen L, Klein R, et al. CD19-targeting CAR T cells for Myositis and interstitial lung disease associated with antisynthetase syndrome. JAMA, 2023, 329(24): 2154-2162.
13 Van Oekelen O, Aleman A, Upadhyaya B, et al. Neurocognitive and hypokinetic movement disorder with features of Parkinsonism after BCMA-targeting CAR-T cell therapy. Nature Medicine, 2021, 27(12): 2099-2103.
14 Zhang J Q, Hu Y X, Yang J X, et al. Non-viral, specifically targeted CAR-T cells achieve high safety and efficacy in BNHL. Nature, 2022, 609: 369-374.
15 An J, Zhang C P, Qiu H Y, et al. Enhancement of the viability of T cells electroporated with DNA via osmotic dampening of the DNA-sensing cGAS-STING pathway. Nature Biomedical Engineering, 2024, 8(2): 149-164.
16 Ghassemi S, Durgin J S, Nunez-Cruz S, et al. Rapid manufacturing of non-activated potent CAR T cells. Nature Biomedical Engineering, 2022, 6(2): 118-128.
17 Wang Y, Chen X D, Cao W, et al. Plasticity of mesenchymal stem cells in immunomodulation: Pathological and therapeutic implications. Nature Immunology, 2014, 15(11): 1009-1016.
18 王瑜, 徐慕晗, 郑凡君, 等. 间充质干细胞在炎症性疾病治疗中的基础研究和临床应用. 中国细胞生物学学报, 2019, 41(4): 561-572.Wang Y, Xu M H, Zheng F J, et al. Immunoregulatory mechanisms and applications of mesenchymal stem/stromal cells in inflammatory diseases. Chinese Journal of Cell Biology, 2019, 41(4): 561-572. (in Chinese)
19 Zhou T, Yuan Z N, Weng J, et al. Challenges and advances in clinical applications of mesenchymal stromal cells. Journal of Hematology & Oncology, 2021, 14(1): 24.
20 Galipeau J, Sensébé L. Mesenchymal stromal cells: Clinical challenges and therapeutic opportunities. Cell Stem Cell, 2018, 22(6): 824-833.
21 Ren G W, Zhang L Y, Zhao X, et al. Mesenchymal stem cellmediated immunosuppression occurs via concerted action of chemokines and nitric oxide. Cell Stem Cell, 2008, 2(2): 141-150.
22 Zhang Y N, Wang J C, Huang W J, et al. Nuclear Nestin deficiency drives tumor senescence via lamin A/C-dependent nuclear deformation. Nature Communications, 2018, 9(1): 3613.
23 Yang Y K, Ogando C R, Wang S C, et al. Changes in phenotype and differentiation potential of human mesenchymal stem cells aging in vitro. Stem Cell Research & Therapy, 2018, 9(1): 131.
24 Zhang T, He Y Q, Shu X, et al. Photomodulation alleviates cellular senescence of aging adipose-derived stem cells. Cell Communication and Signaling, 2023, 21(1): 146.
25 Yu J Y, Vodyanik M A, Smuga-Otto K, et al. Induced pluripotent stem cell lines derived from human somatic cells. Science, 2007, 318: 1917-1920.
26 Hering B J, Bellin M D. Transplantation: Sustained benefits of islet transplants for T1DM. Nature Reviews Endocrinology, 2015, 11(10): 572-574.
27 Millman J R, Xie C H, Van Dervort A, et al. Generation of stem cell-derived β-cells from patients with type 1 diabetes. Nature Communications, 2016, 7: 11463.
28 Pagliuca F W, Millman J R, Gürtler M, et al. Generation of functional human pancreatic beta cells in vitro. Cell, 2014, 159(2): 428-439.
29 Rezania A, Bruin J E, Arora P, et al. Reversal of diabetes with insulin-producing cells derived in vitro from human pluripotent stem cells. Nature Biotechnology, 2014, 32(11): 1121-1133.
30 Du Y Y, Liang Z, Wang S S, et al. Human pluripotent stemcell-derived islets ameliorate diabetes in non-human Primates. Nature Medicine, 2022, 28(2): 272-282.
31 Cheng X, Ying L, Lu L, et al. Self-renewing endodermal progenitor lines generated from human pluripotent stem cells. Cell Stem Cell, 2012, 10(4): 371-384.
32 Wu J Y, Li T, Guo M, et al. Treating a type 2 diabetic patient with impaired pancreatic islet function by personalized endoderm stem cell-derived islet tissue. Cell Discovery, 2024, 10(1): 45.
33 Ma X J, Lu Y K, Zhou Z Y, et al. Human expandable pancreatic progenitor-derived β cells ameliorate diabetes. Science Advances, 2022, 8: eabk1826.
34 Zhu S Y, Russ H A, Wang X J, et al. Human pancreatic betalike cells converted from fibroblasts. Nature Communications, 2016, 7: 10080.
35 Wang Y F, Qin J H, Wang S Y, et al. Conversion of human gastric epithelial cells to multipotent endodermal progenitors using defined small molecules. Cell Stem Cell, 2016, 19(4): 449-461.
36 Wang D S, Wang J Q, Bai L Y, et al. Long-term expansion of pancreatic islet organoids from resident Procr+ progenitors. Cell, 2020, 180(6): 1198-1211.
37 Gornalusse G G, Hirata R K, Funk S E, et al. HLA-Eexpressing pluripotent stem cells escape allogeneic responses and lysis by NK cells. Nature Biotechnology, 2017, 35(8): 765-772.
38 Vegas A J, Veiseh O, Gürtler M, et al. Long-term glycemic control using polymer-encapsulated human stem cell-derived beta cells in immune-competent mice. Nature Medicine, 2016, 22(3): 306-311.
39 Basta G, Montanucci P, Calafiore R. Microencapsulation of cells and molecular therapy of type 1 diabetes mellitus: The actual state and future perspectives between promise and progress. Journal of Diabetes Investigation, 2021, 12(3): 301-309.
40 Obernier K, Alvarez-Buylla A. Neural stem cells: Origin, heterogeneity and regulation in the adult mammalian brain. Development, 2019, 146(4): dev156059.
41 Kim T W, Piao J H, Koo S Y, et al. Biphasic activation of WNT signaling facilitates the derivation of midbrain dopamine neurons from hESCs for translational use. Cell Stem Cell, 2021, 28(2): 343-355.
42 Fitzgerald M, Sotuyo N, Tischfield D J, et al. Generation of cerebral cortical GABAergic interneurons from pluripotent stem cells. Stem Cells, 2020, 38(11): 1375-1386.
43 Wang Y K, Zhu W W, Wu M H, et al. Human clinical-grade parthenogenetic ESC-derived dopaminergic neurons recover locomotive defects of nonhuman primate models of Parkinson’s disease. Stem Cell Reports, 2018, 11(1): 171-182.
44 Kriks S, Shim J W, Piao J H, et al. Dopamine neurons derived from human ES cells efficiently engraft in animal models of Parkinson’s disease. Nature, 2011, 480: 547-551.
45 Kikuchi T, Morizane A, Doi D, et al. Human iPS cell-derived dopaminergic neurons function in a primate Parkinson’s disease model. Nature, 2017, 548: 592-596.
46 Schweitzer J S, Song B, Herrington T M, et al. Personalized iPSC-derived dopamine progenitor cells for Parkinson’s disease. New England Journal of Medicine, 2020, 382(20): 1926-1932.
47 Loring J F. Autologous induced pluripotent stem cell-derived neurons to treat Parkinson’s disease. Stem Cells and Development, 2018, 27(14): 958-959.
48 Piao J H, Zabierowski S, Dubose B N, et al. Preclinical efficacy and safety of a human embryonic stem cell-derived midbrain dopamine progenitor product, MSK-DA01.Cell Stem Cell, 2021, 28(2): 217-229.
49 You Z W, Wang L Y, He H, et al. Mapping of clonal lineages across developmental stages in human neural differentiation. Cell Stem Cell, 2023, 30(4): 473-487.
50 Feng L, Li D, Tian Y, et al. One-step cell biomanufacturing platform: Porous gelatin microcarrier beads promote human embryonic stem cell-derived midbrain dopaminergic progenitor cell differentiation in vitro and survival after transplantation in vivo. Neural Regeneration Research, 2024, 19(2): 458-464.
51 Zhang T, Ke W, Zhou X, et al. Human neural stem cells reinforce hippocampal synaptic network and rescue cognitive deficits in a mouse model of Alzheimer’s disease. Stem Cell Reports, 2019, 13(6): 1022-1037.
52 Bershteyn M, Bröer S, Parekh M, et al. Human pallial MGEtype GABAergic interneuron cell therapy for chronic focal epilepsy. Cell Stem Cell, 2023, 30(10): 1331-1350.
53 Curtis E, Martin J R, Gabel B, et al. A first-in-human, phase I study of neural stem cell transplantation for chronic spinal cord injury. Cell Stem Cell, 2018, 22(6): 941-950.
54 Sun Z, Yuan X, Wu J Q, et al. Hepatocyte transplantation: The progress and the challenges. Hepatology Communications, 2023, 7: e0266.
55 Feng S S, Wu J Y, Qiu W L, et al. Large-scale generation of functional and transplantable hepatocytes and cholangiocytes from human endoderm stem cells. Cell Reports, 2020, 33 (10): 108455.
56 Chen S T, Wang J L, Ren H Z, et al. Hepatic spheroids derived from human induced pluripotent stem cells in bioartificial liver rescue porcine acute liver failure. Cell Research, 2020, 30(1): 95-97.
57 Wang S Y, Wang X, Tan Z L, et al. Human ESC-derived expandable hepatic organoids enable therapeutic liver repopulation and pathophysiological modeling of alcoholic liver injury. Cell Research, 2019, 29(12): 1009-1026.
58 Huang P Y, Zhang L D, Gao Y M, et al. Direct reprogramming of human fibroblasts to functional and expandable hepatocytes. Cell Stem Cell, 2014, 14(3): 370-384.
59 Zhang K, Zhang L D, Liu W M, et al. In vitro expansion of primary human hepatocytes with efficient liver repopulation capacity. Cell Stem Cell, 2018, 23(6): 806-819.
60 Fu G B, Huang W J, Zeng M, et al. Expansion and differentiation of human hepatocyte-derived liver progenitorlike cells and their use for the study of hepatotropic pathogens. Cell Research, 2019, 29(1): 8-22.
61 Yuan X, Wu J Q, Sun Z, et al. Preclinical efficacy and safety of encapsulated proliferating human hepatocyte organoids in treating liver failure. Cell Stem Cell, 2024, 31(4): 484-498.
62 Wang Y F, Zheng Q, Sun Z, et al. Reversal of liver failure using a bioartificial liver device implanted with clinicalgrade human-induced hepatocytes. Cell Stem Cell, 2023, 30 (5): 617-631.
63 Li W J, Zhu X J, Yuan T J, et al. An extracorporeal bioartificial liver embedded with 3D-layered human liver progenitor-like cells relieves acute liver failure in pigs. Science Translational Medicine, 2020, 12: eaba5146.
64 Glorioso J M, Mao S A, Rodysill B, et al. Pivotal preclinical trial of the spheroid reservoir bioartificial liver. Journal of Hepatology, 2015, 63(2): 388-398.
Recommended Citation
HUI, Lijian; YUAN, Xiang; and WU, Jingqi
(2024)
"Cell-based medicinal products: Progress and perspectives,"
Bulletin of Chinese Academy of Sciences (Chinese Version): Vol. 39
:
Iss.
8
, Article 13.
DOI: https://doi.org/10.16418/j.issn.1000-3045.20240221003
Available at:
https://bulletinofcas.researchcommons.org/journal/vol39/iss8/13
Included in
Cell Biology Commons, Pharmaceutics and Drug Design Commons, Science and Technology Policy Commons