The successful treatment of severe aplastic anemia with autologous cord blood transplantation

Fruchtman, Steven; Hurlet, A.; Dracker, Robert A.; Isola, Luis; Goldman, Benjamin; Schneider, Benjamin; Emre, Sükrü

doi:10.1016/j.bbmt.2004.07.003

Cited by 54 publications

(31 citation statements)

References 7 publications

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“…Cells derived from the human umbilical cord (HUC) have been successfully used in the clinic for almost 2 decades [1-4]. Their simple and economic retrieval, enrichment for hematopoietic progenitors, enhanced proliferation rate, expansion potential [5,6], and low incidence of graft-versus-host disease [7,8] makes them a promising cell treatment for several disorders.…”

Section: Introductionmentioning

confidence: 99%

Intraarterial transplantation of human umbilical cord blood mononuclear cells is more efficacious and safer compared with umbilical cord mesenchymal stromal cells in a rodent stroke model

et al. 2014

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IntroductionStroke is the second leading cause of death worldwide, claims six lives every 60 seconds, and is a leading cause of adult disability across the globe. Tissue plasminogen activator, the only United States Food and Drug Administration (FDA)-approved drug currently available, has a narrow therapeutic time window of less than 5 hours. In the past decade, cells derived from the human umbilical cord (HUC) have emerged as a potential therapeutic alternative for stroke; however, the most effective HUC-derived cell population remains unknown.MethodsWe compared three cell populations derived from the human umbilical cord: cord blood mononuclear cells (cbMNCs); cord blood mesenchymal stromal cells (cbMSCs), a subpopulation of cbMNCs; and cord matrix MSCs (cmMSCs). We characterized these cells in vitro with flow cytometry and assessed the cells’ in vivo efficacy in a 2-hour transient middle cerebral artery occlusion (MCAo) rat model of stroke. cbMNCs, cbMSCs, and cmMSCs were each transplanted intraarterially at 24 hours after stroke.ResultsA reduction in neurologic deficit and infarct area was observed in all three cell groups; however, this reduction was significantly enhanced in the cbMNC group compared with the cmMSC group. At 2 weeks after stroke, human nuclei-positive cells were present in the ischemic hemispheres of immunocompetent stroke rats in all three cell groups. Significantly decreased expression of rat brain-derived neurotrophic factor mRNA was observed in the ischemic hemispheres of all three cell-treated and phosphate-buffered saline (PBS) group animals compared with sham animals, although the decrease was least in cbMNC-treated animals. Significantly decreased expression of rat interleukin (IL)-2 mRNA and IL-6 mRNA was seen only in the cbMSC group. Notably, more severe complications (death, eye inflammation) were observed in the cmMSC group compared with the cbMNC and cbMSC groups.ConclusionsAll three tested cell types promoted recovery after stroke, but cbMNCs showed enhanced recovery and fewer complications compared with cmMSCs.

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Section: Introductionmentioning

confidence: 99%

Intraarterial transplantation of human umbilical cord blood mononuclear cells is more efficacious and safer compared with umbilical cord mesenchymal stromal cells in a rodent stroke model

et al. 2014

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“…In the vast majority of these transplants, HLA mismatching between donor and recipient was present at 1 or 2 HLA antigens. Efficacy has been demonstrated in both children and adults with leukemias (7)(8)(9)(10)(11)(12)(13)(19)(20)(21)(22)(23) and children with hemoglobinopathies (14,15), immunodeficiency syndromes (16), bone marrow failure syndromes (24), and inborn errors of metabolism (17,18). Reported survival rates (Table 1) are similar to those seen in patients transplanted with matched bone marrow from unrelated donors despite the fact that the cord blood was generally mismatched at 1 or 2 HLA loci.…”

Section: From Experimentation To Practice: Development Of Cord Blood supporting

confidence: 58%

“…Initially, because of early results correlating cell dose with engraftment and survival, cord blood transplantation was restricted to use in children and small adults, generally weighing less than 40 kg. More recently, however, the use of cord blood has been extended to include adults, allowing for better definition of cell dose limitations and thresholds (19)(20)(21)(22)(23)(24). The results of these transplants have helped define a requirement for a minimum cell dose of 3 × 10 7 to 3.5 × 10 7 nucleated cells/kg in order to obtain acceptable clinical outcomes (25).…”

Section: From Experimentation To Practice: Development Of Cord Blood mentioning

confidence: 99%

Untying the Gordian knot: policies, practices, and ethical issues related to banking of umbilical cord blood

Kurtzberg

Lyerly

Sugarman

2005

Journal of Clinical Investigation

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Since the first successful transplantation of umbilical cord blood in 1988, cord blood has become an important source of hematopoietic stem and progenitor cells for the treatment of blood and genetic disorders. Significant progress has been accompanied by challenges for scientists, ethicists, and health policy makers. With the recent recognition of the need for a national system for the collection, banking, distribution, and use of cord blood and the increasing focus on cord blood as an alternative to embryos as a source of tissue for regenerative medicine, cord blood has garnered significant attention. We review the development of cord blood banking and transplantation and then discuss the scientific and ethical issues influencing both established and investigational practices surrounding cord blood collection, banking, and use.In 1988, a 6-year-old boy from North Carolina with Fanconi anemia was transplanted in Paris with HLA-matched umbilical cord blood from his baby sister (1). Most scientists and physicians at the time were highly skeptical, doubting that a few ounces of cord blood contained sufficient stem and progenitor cells to rescue bone marrow after myeloablative therapy. However, this child engrafted without incident, fully reconstituting his blood, bone marrow, and immune system with donor cells. He remains well and durably engrafted with donor cells 17 years following the original transplant (J. Kurtzberg, personal communication). From experimentation to practice: development of cord blood transplantationOver the 5-6 years following the first cord blood transplant, approximately 60 additional transplants between HLA-matched siblings were performed worldwide. Reports of results to a volunteer registry (2) demonstrated that cord blood contained sufficient numbers of stem and progenitor cells to reconstitute the entire hematopoietic system of a child after myeloablative therapy and that the incidence of graft-versus-host disease (GVHD) was 10-fold lower than that seen after transplantation with HLA-matched bone marrow obtained from a sibling.At this time it was becoming apparent that the diversity of HLA alleles and antigens was vast and that it was never going to be possible to find fully matched related and unrelated adult donors for all patients in need of allogeneic transplantation therapy from then-available sources. The National Marrow Donor Foundation (NMDP) and other international registries successfully recruited, typed, and listed millions of volunteer unrelated adult donors, but only 25-50% of patients in need could locate sufficiently matched donors in a timely fashion. Donors for patients of minority ethnic backgrounds were even scarcer and more difficult to locate. To provide donors for all patients in need, transplant physicians needed to find a way to transplant partially mismatched grafts.

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“…The child had previously donated cord blood at birth because her brother had leukemia [45]. There have been several cases describing the use of autologous cord blood for aplastic anemia [46,47]. In addition, a recent report indicated the use of autologous cord blood for a 3 year old girl with an isolated CNS relapse of acute lymphoblastic leukemia [48].…”

Section: The Great Debate: Public or Private Donationmentioning

confidence: 95%

Challenges in Umbilical Cord Blood Stem Cell Banking for Stem Cell Reviews and Reports

Ballen

2009

Stem Cell Rev and Rep

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Twenty years has passed since the first report of a successful cord blood transplant was reported in 1989 in a child with Fanconi's anemia. During these 20 years, the cord blood field has had dramatic growth, with over 400,000 cord blood units donated and stored worldwide for unrelated use. Approximately, 14,000 unrelated cord blood transplants have been performed to date for patients with hematologic malignancies and bone marrow disorders, and who do not have matched family or unrelated donors. In contrast, about 900,000 cord blood units have been stored privately for personal use, with about 100 autologous transplants performed. Twenty years ago, due to the low cell dose, cord blood transplants were only performed in children. Today, with the use of better banking techniques, reduced intensity transplants, and double cord blood transplantation, the majority of cord blood transplants are being performed in adults. In this chapter, we review the scientific basis for cord blood transplantation, and outcome data in both pediatric and adult transplantation. We will then focus on the recent concerns regarding private and public cord blood banking. Finally, we discuss the future of cord blood transplantation, and the exciting work beginning outside of oncology.

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The successful treatment of severe aplastic anemia with autologous cord blood transplantation

Cited by 54 publications

References 7 publications

Intraarterial transplantation of human umbilical cord blood mononuclear cells is more efficacious and safer compared with umbilical cord mesenchymal stromal cells in a rodent stroke model

Intraarterial transplantation of human umbilical cord blood mononuclear cells is more efficacious and safer compared with umbilical cord mesenchymal stromal cells in a rodent stroke model

Untying the Gordian knot: policies, practices, and ethical issues related to banking of umbilical cord blood

Challenges in Umbilical Cord Blood Stem Cell Banking for Stem Cell Reviews and Reports

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