Journal of Clinical Pediatrics >
Progress and challenges in clinical research of umbilical cord blood transplantation for the treatment of premature infant diseases
Received date: 2023-07-11
Online published: 2023-10-08
Despite great progress in the treatment of neonatal diseases, complications of preterm infants such as bronchopulmonary dysplasia, perinatal brain injury and necrotizing enterocolitis are still leading causes of neonatal mortality and long term morbidity. Exploring more effective therapies for the prevention or treatment for the complications of preterm infants are the concerns in neonatology. Stem cell therapy especially umbilical cord blood transplantation, have been explored as a new therapeutic option in premature infant diseases, with very promising preclinical and clinical results over the past 20 years. This article outlined the clinical progress, prospects and challenges of stem cells derived from umbilical cord blood in preterm diseases.
Fang LI , Li WANG . Progress and challenges in clinical research of umbilical cord blood transplantation for the treatment of premature infant diseases[J]. Journal of Clinical Pediatrics, 2023 , 41(10) : 646 -653 . DOI: 10.12372/jcp.2023.23e0634
| [1] | Sun B, Shao X, Cao Y, et al. Neonatal-perinatal medicine in a transitional period in China[J]. Arch Dis Child Fetal Neonatal Ed, 2013, 98(5): F440-F444. |
| [2] | He C, Liu L, Chu Y, et al. National and subnational all-cause and cause-specific child mortality in China, 1996-2015: a systematic analysis with implications for the Sustainable Development Goals[J]. Lancet Glob Health, 2017, 5(2): e186-e197. |
| [3] | Schmidt B, Asztalos EV, Roberts RS, et al. Impact of bronchopulmonary dysplasia, brain injury, and severe retinopathy on the outcome of extremely low-birth-weight infants at 18 months: results from the trial of indomethacin prophylaxis in preterms[J]. JAMA-J Am Med Assoc, 2003, 289(9): 1124-1129. |
| [4] | Baker EK, Jacobs SE, Lim R, et al. Cell therapy for the preterm infant: promise and practicalities[J]. Arch Dis Child Fetal Neonatal Ed, 2020, 105(5): 563-568. |
| [5] | Sanchez-Petitto G, Rezvani K, Daher M, et al. Umbilical cord blood transplantation: connecting its origin to its future[J]. Stem Cells Transl Med, 2023, 12(2): 55-71. |
| [6] | Broxmeyer HE, Lee M, Hangoc G, et al. Hematopoietic stem/progenitor cells, generation of induced pluripotent stem cells, and isolation of endothelial progenitors from 21- to 23.5-year cryopreserved cord blood[J]. Blood, 2011, 117(18): 4773-4777. |
| [7] | Mcdonald CA, Fahey MC, Jenkin G, et al. Umbilical cord blood cells for treatment of cerebral palsy; timing and treatment options[J]. Pediatr Res, 2018, 83(1-2): 333-344. |
| [8] | Castillo-Melendez M, Yawno T, Jenkin G, et al. Stem cell therapy to protect and repair the developing brain: a review of mechanisms of action of cord blood and amnion epithelial derived cells[J]. Front Neurosci, 2013, 7: 194. |
| [9] | Dominici M, Le Blanc K, Mueller I, et al. Minimal criteria for defining multipotent mesenchymal stromal cells. The International Society for Cellular Therapy position statement[J]. Cytotherapy, 2006, 8(4): 315-317. |
| [10] | Bernardo ME, Pagliara D, Locatelli F. Mesenchymal stromal cell therapy: a revolution in regenerative medicine?[J]. Bone Marrow Transplant, 2012, 47(2): 164-171. |
| [11] | Zhang X, Hirai M, Cantero S, et al. Isolation and characterization of mesenchymal stem cells from human umbilical cord blood: reevaluation of critical factors for successful isolation and high ability to proliferate and differentiate to chondrocytes as compared to mesenchymal stem cells from bone marrow and adipose tissue[J]. J Cell Biochem, 2011, 112(4):1206-1218. |
| [12] | Brown JA, Boussiotis VA. Umbilical cord blood transplantation: Basic biology and clinical challenges to immune reconstitution[J]. Clin Immunol, 2008, 127(3): 286-297. |
| [13] | Carrelha J, Meng Y, Kettyle LM, et al. Hierarchically related lineage-restricted fates of multipotent haema-topoietic stem cells[J]. Nature, 2018, 554(7690): 106-111. |
| [14] | Li J, Ma Y, Miao X, et al. Neovascularization and tissue regeneration by endothelial progenitor cells in ischemic stroke[J]. Neurol Sci, 2021, 42(9): 3585-3593. |
| [15] | Chirumbolo S, Ortolani R, Veneri D, et al. Lymphocyte phenotypic subsets in umbilical cord blood compared to peripheral blood from related mothers[J]. Cytometry B Clin Cytom, 2011, 80B(4): 248-253. |
| [16] | Saha A, Buntz S, Scotland P, et al. A cord blood monocyte-derived cell therapy product accelerates brain remyelination[J]. JCI Insight, 2016, 1(13): e86667. |
| [17] | Jakubzick CV, Randolph GJ, Henson PM. Monocyte differentiation and antigen-presenting functions[J]. Nat Rev Immunol, 2017, 17(6): 349-362. |
| [18] | Sullivan MJ. Banking on cord blood stem cells[J]. Nat Rev Cancer, 2008, 8(7): 555-563. |
| [19] | Batsali AK, Kastrinaki M, Papadaki HA, et al. Mesenchymal stem cells derived from Wharton's Jelly of the umbilical cord: biological properties and emerging clinical applications[J]. Curr Stem Cell Res Ther, 2013, 8(2): 144-145. |
| [20] | Malhotra A, Thebaud B, Paton MCB, et al. Advances in neonatal cell therapies: Proceedings of the First Neonatal Cell Therapies Symposium (2022)[J]. Pediatr Res, 2023. doi: 10.1038/s41390-023-02707-x. |
| [21] | Willis GR, Reis M, Gheinani AH, et al. Extracellular vesicles protect the neonatal lung from hyperoxic injury through the epigenetic and transcriptomic reprogramming of myeloid cells[J]. Am J Respir Crit Care Med, 2021, 204(12): 1418-1432. |
| [22] | van Haaften T, Byrne R, Bonnet S, et al. Airway delivery of mesenchymal stem cells prevents arrested alveolar growth in neonatal lung injury in rats[J]. A Am J Respir Crit Care Med, 2009, 180(11): 1131-1142. |
| [23] | Rubinstein P, Dobrila L, Rosenfield RE, et al. Processing and cryopreservation of placental/umbilical cord blood for unrelated bone marrow reconstitution[J]. Proc Natl Acad Sci U S A, 1995, 92: 10119-10122. |
| [24] | Walter J, Ware LB, Matthay MA. Mesenchymal stem cells: mechanisms of potential therapeutic benefit in ARDS and sepsis[J]. Lancet Respir Med, 2014, 2(12): 1016-1026. |
| [25] | Gluckman E, Broxmeyer HA, Auerbach AD, et al. Hema-topoietic reconstitution in a patient with Fanconi's anemia by means of umbilical-cord blood from an HLA-identical sibling[J]. N Engl J Med, 1989, 321(17): 1174-1178. |
| [26] | Kotowski M, Safranow K, Kawa MP, et al. Circulating hematopoietic stem cell count is a valuable predictor of prematurity complications in preterm newborns[J]. BMC Pediatrics, 2012, 12: 148. |
| [27] | Borghesi A, Massa M, Campanelli R, et al. Circulating endothelial progenitor cells in preterm infants with bronchopulmonary dysplasia[J]. A Am J Respir Crit Care Med, 2009, 180(6): 540-546. |
| [28] | Rudnicki J, Kawa MP, Kotowski M. Clinical evaluation of the safety and feasibility of whole autologous cord blood transplant as a source of stem and progenitor cells for extremely premature neonates: preliminary report[J]. Exp Clin Transplant, 2015, 13(6): 563-572. |
| [29] | Chang YS, Ahn SY, Yoo HS, et al. Mesenchymal stem cells for bronchopulmonary dysplasia: phase 1 dose-escalation clinical trial[J]. J Pediatr, 2014, 164(5): 966-972. |
| [30] | Powell SB, Silvestri JM. Safety of intratracheal administration of human umbilical cord blood derived mesenchymal stromal cells in extremely low birth weight preterm infants[J]. J Pediatr, 2019, 210: 209-213. |
| [31] | Xia Y, Lang T, Niu Y, et al. Phase I trial of human umbilical cord-derived mesenchymal stem cells for treatment of severe bronchopulmonary dysplasia[J]. Genes Dis, 2023, 10(2): 521-530. |
| [32] | Nguyen LT, Trieu TTH, Bui HTH, et al. Allogeneic administration of human umbilical cord-derived mesen-chymal stem/stromal cells for bronchopulmonary dysplasia: preliminary outcomes in four Vietnamese infants[J]. J Transl Med, 2020, 18(1): 398. |
| [33] | Ahn SY, Chang YS, Lee MH, et al. Stem cells for bronchopulmonary dysplasia in preterm infants: a randomized controlled phase II trial[J]. Stem Cells Transl Med, 2021, 10(8): 1129-1137. |
| [34] | Ren Z, Xu F, Zhang X, et al. Autologous cord blood cell infusion in preterm neonates safely reduces respiratory support duration and potentially preterm complications[J]. Stem Cells Transl Med, 2020, 9(2): 169-176. |
| [35] | Zhuxiao R, Fang X, Wei W, et al. Prevention for moderate or severe BPD with intravenous infusion of autologous cord blood mononuclear cells in very preterm infants-a prospective non-randomized placebo-controlled trial and two-year follow up outcomes[J]. EClinicalMedicine, 2023, 57: 101844. |
| [36] | Inder TE, Warfield SK, Wang H, et al. Abnormal cerebral structure is present at term in premature infants[J]. Pediatrics, 2005, 115(2): 286-294. |
| [37] | Sabir H, Bonifacio SL, Gunn AJ, et al. Unanswered questions regarding therapeutic hypothermia for neonates with neonatal encephalopathy[J]. Semin Fetal Neonatal Med, 2021, 26(5): 101257. |
| [38] | Ahn SY, Chang YS, Sung SI, et al. Mesenchymal stem cells for severe intraventricular hemorrhage in preterm infants: phase I dose-escalation clinical trial[J]. Stem Cells Transl Med, 2018, 7(12): 847-856. |
| [39] | Cotten CM, Murtha AP, Goldberg RN, et al. Feasibility of autologous cord blood cells for infants with hypoxic-ischemic encephalopathy[J]. J Pediatr, 2014, 164(5): 973-979. |
| [40] | Tsuji M, Sawada M, Watabe S, et al. Autologous cord blood cell therapy for neonatal hypoxic-ischaemic encephalopathy: a pilot study for feasibility and safety[J]. Sci Rep, 2020, 10(1): 4603. |
| [41] | Malhotra A, Novak I, Miller SL, et al. Autologous transplantation of umbilical cord blood-derived cells in extreme preterm infants: protocol for a safety and feasibility study[J]. BMJ Open, 2020, 10(5): e36065. |
| [42] | Akduman H, Dilli D, Ergün E, et al. Successful mesenchymal stem cell application in supraventricular tachycardia-related necrotizing enterocolitis: a case report[J]. Fetal Pediatr Pathol, 2021, 40(3): 250-255. |
| [43] | Kotowski M, Litwinska Z, Klos P. Autologous cord utologous cord blood transfusion in preterm infants-could its humoral effect be the key to control prematurity-related complications? a preliminary study[J]. J Physiol Pharmacol, 2017, 68(6): 921-927. |
| [44] | Burkhart HM, Qureshi MY, Rossano JW, et al. Autologous stem cell therapy for hypoplastic left heart syndrome: Safety and feasibility of intraoperative intramyocardial injections[J]. J Thorac Cardiovasc Surg, 2019, 158(6): 1614-1623. |
| [45] | Ciubotariu R, Scaradavou A, Ciubotariu I, et al. Impact of delayed umbilical cord clamping on public cord blood donations: can we help future patients and benefit infant donors?[J]. Transfusion, 2018, 58(6): 1427-1433. |
| [46] | Segler A, Braun T, Fischer HS, et al. Feasibility of umbilical cord blood collection in neonates at risk of brain damage—a step toward autologous cell therapy for a high-risk population[J]. Cell Transplant, 2021, 30: 1503988442. |
| [47] | Zarrabi M, Akbari MG, Amanat M, et al. The safety and efficacy of umbilical cord blood mononuclear cells in individuals with spastic cerebral palsy: a randomized double-blind sham-controlled clinical trial[J]. BMC Neurology, 2022, 22(1): 123. |
| [48] | Cohen S, Roy J, Lachance S, et al. Hematopoietic stem cell transplantation using single UM171-expanded cord blood: a single-arm, phase 1-2 safety and feasibility study[J]. Lancet Haematol, 2020, 7(2): e134-e145. |
| [49] | Qin M, Guan X, Wang H, et al. An effective ex-vivo approach for inducing endothelial progenitor cells from umbilical cord blood CD34+ cells[J]. Stem Cell Res Ther, 2017, 8(1): 25. |
| [50] | Ortiz LA, Gambelli F, Mcbride C, et al. Mesenchymal stem cell engraftment in lung is enhanced in response to bleomycin exposure and ameliorates its fibrotic effects[J]. Proc Natl Acad Sci U S A, 2003, 100(14): 8407-8411. |
| [51] | Li J, Yawno T, Sutherland A, et al. Preterm white matter brain injury is prevented by early administration of umbilical cord blood cells[J]. Exp Neurol, 2016, 283: 179-187. |
| [52] | Sun JM, Song AW, Case LE, et al. Effect of autologous cord blood infusion on motor function and brain connectivity in young children with cerebral palsy: a randomized, placebo-controlled trial[J]. Stem Cells Transl Med, 2017, 6(12): 2071-2078. |
| [53] | Ahn SY, Chang YS, Sung DK, et al. Optimal route for mesenchymal stem cells transplantation after severe intraventricular hemorrhage in newborn rats[J]. PLoS One, 2015, 10(7): e132919. |
| [54] | Luan Y, Zhang L, Chao S, et al. Mesenchymal stem cells in combination with erythropoietin repair hyperoxia-induced alveoli dysplasia injury in neonatal mice via inhibition of TGF-β1 signaling[J]. Oncotarget, 2016, 7(30): 47082-47094. |
| [55] | Park WS, Sung SI, Ahn SY, et al. Hypothermia augments neuroprotective activity of mesenchymal stem cells for neonatal hypoxic-ischemic encephalopathy[J]. PLoS One, 2015, 10(3): e120893. |
| [56] | Herz J, K?ster C, Reinboth BS, et al. Interaction between hypothermia and delayed mesenchymal stem cell therapy in neonatal hypoxic-ischemic brain injury[J]. Brain Behav Immun, 2018, 70: 118-130. |
| [57] | R?sland GV, Svendsen A, Torsvik A, et al. Long-term cultures of bone marrow-derived human mesenchymal stem cells frequently undergo spontaneous malignant transformation[J]. Cancer Res, 2009, 69(13): 5331-5339. |
| [58] | He J, Yao X, Mo P, et al. Lack of tumorigenesis and protumorigenic activity of human umbilical cord mesenchymal stem cells in NOD SCID mice[J]. BMC Cancer, 2022, 22(1): 307. |
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