Beta Glucan Enhances Hematopoietic Stem Cell Mobilization.

Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 1348-1348
Author(s):  
Stephanie Wagner ◽  
Daniel Cramer ◽  
Richard Hansen ◽  
Ryan Reca ◽  
Mariusz Ratajczak ◽  
...  

Abstract Background: Peripheral blood stem cell infusion is the preferred method for establishing hematopoiesis in transplantation. Use of G-CSF is now the most commonly used mobilizing agent. Despite advances in stem cell techniques and agents, studies have shown that up to 20–25% of patients exhibit poor mobilization and are not able to proceed with autotransplantation. Strategies to improve mobilization include using chemotherapy alone or in conjunction with growth factor or novel agents such as AMD3100. β-glucan PGG is a soluble yeast beta glucan with a molecular mass of 150kD comprised of a β-D-(1–3)-linked glucopyranosyl backbone with a β-D-(1–6)-linked β(1–3) side chains. In previous studies, β-glucan PGG has been shown to induce hematopoietic stem and progenitor cell (HSPC) mobilization to the periphery. In this study, we examined β-glucan PGG’s ability to mobilize HSPC alone and in conjuction with G-CSF and explored its mechanism of action. Methods: Prior to our study, dose kinetic studies were done and showed peak stem cell mobilization at 24 hours and maximum results using the 9.2 mg/kg dose with β-glucan PGG alone. In this study, four groups of wildtype (WT) C57BL/6 mice (6 mice/group) were used; control (saline injection × 4 days), G-CSF only (125ug/d × 4 days), PGG only (4.8 or 9.2mg/kg × one dose), and G-CSF/PGG combination. In the combination group, G-CSF injections were given daily for four days and one PGG injection on day three. Four hours after the last G-CSF injection, the mice were sacrificed and final white blood cell count were collected. Blood was assayed for in vitro mobilization in methycellulose culture. Peritoneal macrophage were stimulated with PGG and supernatant was harvested at times indicated and concentration of MMP-9 was determined using ELISA (R&D Systems). Results: All treated groups showed increased mobilization of all major cell lines (CFU-GM, CFU-M, and CFU-G). β-glucan PGG alone was able to mobilize peripheral stem cells at both doses (4.8mg/kg–9.3 CFU/200000 PBL and 9.2mg/kg–14 CFU/200000 PBL). The combination group (G-CSF/PGG-4.8mg/kg) showed an almost two-fold increase in CFU compared to G-CSF alone (G-CSF-30.42 CFU/200000 PBL, G-CSF/PGG(4.8)-57 CFU/200000 PBL, p=0.008). Initial in vitro chemotaxis assays revealed β-glucan PGG induces HPSC mobilization independent of SDF-1 (stromal derived factor) gradient. Our previous studies have demonstrated that β-glucan can enhance bone marrow engraftment via a CR3 dependent mechanism. However, our current study indicated that β-glucan mobilized stem cells via a CR3 independent mechanism and did not induce appreciable levels of cytokine secretion. To further explore its mechanism of action, we stimulated peritoneal macrophages with β-glucan PGG. Strikingly, β-glucan PGG stimulated macrophages to produce significant amounts of matrix metalloproteinase-9 (MMP-9). Similarly, β-glucan PGG also stimulated bone marrow-derived macrophages to secrete MMP-9. Conclusion: β-glucan PGG is an agent that enhances stem cell mobilization alone and has a synergistic effect when used in conjunction with G-CSF. The mechanism of mobilization by β-glucan PGG involves MMP-9, which results from release of pro-MMP-9 from marrow macrophages. The efficacy of β-glucan PGG and lack of proinflammatory activity make it an attractive agent to supplement mobilization with G-CSF.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 2190-2190 ◽  
Author(s):  
Pieter K. Wierenga ◽  
Ellen Weersing ◽  
Bert Dontje ◽  
Gerald de Haan ◽  
Ronald P. van Os

Abstract Adhesion molecules have been implicated in the interactions of hematopoietic stem and progenitor cells with the bone marrow extracellular matrix and stromal cells. In this study we examined the role of very late antigen-5 (VLA-5) in the process of stem cell mobilization and homing after stem cell transplantation. In normal bone marrow (BM) from CBA/H mice 79±3 % of the cells in the lineage negative fraction express VLA-5. After mobilization with cyclophosphamide/G-CSF, the number of VLA-5 expressing cells in mobilized peripheral blood cells (MPB) decreases to 36±4%. The lineage negative fraction of MPB cells migrating in vitro towards SDF-1α (M-MPB) demonstrated a further decrease to 3±1% of VLA-5 expressing cells. These data are suggestive for a downregulation of VLA-5 on hematopoietic cells during mobilization. Next, MPB cells were labelled with PKH67-GL and transplanted in lethally irradiated recipients. Three hours after transplantation an increase in VLA-5 expressing cells was observed which remained stable until 24 hours post-transplant. When MPB cells were used the percentage PKH-67GL+ Lin− VLA-5+ cells increased from 36% to 88±4%. In the case of M-MPB cells the number increased from 3% to 33±5%. Although the increase might implicate an upregulation of VLA-5, we could not exclude selective homing of VLA-5+ cells as a possible explanation. Moreover, we determined the percentage of VLA-5 expressing cells immediately after transplantation in the peripheral blood of the recipients and were not able to observe any increase in VLA-5+ cells in the first three hours post-tranpslant. Finally, we separated the MPB cells in VLA-5+ and VLA-5− cells and plated these cells out in clonogenic assays for progenitor (CFU-GM) and stem cells (CAFC-day35). It could be demonstared that 98.8±0.5% of the progenitor cells and 99.4±0.7% of the stem cells were present in the VLA-5+ fraction. Hence, VLA-5 is not downregulated during the process of mobilization and the observed increase in VLA-5 expressing cells after transplantation is indeed caused by selective homing of VLA-5+ cells. To shed more light on the role of VLA-5 in the process of homing, BM and MPB cells were treated with an antibody to VLA-5. After VLA-5 blocking of MPB cells an inhibition of 59±7% in the homing of progenitor cells in bone marrow could be found, whereas homing of these subsets in the spleen of the recipients was only inhibited by 11±4%. For BM cells an inhibition of 60±12% in the bone marrow was observed. Homing of BM cells in the spleen was not affected at all after VLA-5 blocking. Based on these data we conclude that mobilization of hematopoietic progenitor/stem cells does not coincide with a downregulation of VLA-5. The observed increase in VLA-5 expressing cells after transplantation is caused by preferential homing of VLA-5+ cells. Homing of progenitor/stem cells to the bone marrow after transplantation apparantly requires adhesion interactions that can be inhibited by blocking VLA-5 expression. Homing to the spleen seems to be independent of VLA-5 expression. These data are indicative for different adhesive pathways in the process of homing to bone marrow or spleen.


Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 3542-3542 ◽  
Author(s):  
Ying-Huey Huang ◽  
Yi-Chun Liu ◽  
Chi-Feng Yen ◽  
Hua-Chien Chen ◽  
Shu-Jen Chen ◽  
...  

Abstract Abstract 3542 Poster Board III-479 Background The interaction between SDF-1 and its receptor, CXCR4, is responsible for retaining of stem cells in the bone marrow. CXCR4 antagonist disrupts the SDF-1/CXCR4 interaction and mobilizes CD34+ hematopoietic stem cells (CD34+ HSCs) and CD133+ endothelial progenitor cells (CD133+ EPCs) from bone marrow into circulation, which can be used as a source for stem cell transplantation and other potential clinical indications. TG-0054 is a novel CXCR4 antagonist. In vitro CXCR4 antagonistic activity and in vivo stem cell mobilization activity of TG-0054 were determined in this study. Materials and Methods In vitro pharmacological assays, including GTP-binding assay, calcium mobilization assay, and chemotaxis assays, were performed to assess the potency of TG-0054 as a CXCR4 antagonist. In addition, receptor-binding assays against a panel of human chemokine receptors as well as other 68 cellular receptors were screened to evaluate the specificity of TG-0054. Kinetics and dose-dependent response of stem cell mobilization by TG-0054 were demonstrated in BALB/c mice. Activity of stem cell mobilization of TG-0054 when used alone or combined with G-CSF was also studied. Results TG-0054 blocked SDF-1-binding to CXCR4 receptor with an IC50 of 10 nM and inhibited SDF-1–induced GTP-binding (IC50 = 6 nM), chemotaxis (IC50 = 43 nM), and calcium mobilization (IC50 = 59 nM). TG-0054 showed greater than 3000-fold selectivity for CXCR4 receptor over other chemokine receptors. It is noteworthy that the IC50 for the inhibitory effect of TG-0054 on hERG potassium currents was greater than 1000 μM when human embryonic kidney (HEK) cells were used to examine the in vitro effects on the hERG potassium channel currents. In animal studies, TG-0054 rapidly and effectively mobilized CD34+ HSCs and CD133+ EPCs into circulation. Single intravenous (IV) administration of TG-0054 resulted in a rapid increase of total white blood cell (WBC) counts, as well as CXCR4+, CD34+, and CD133+ cells in peripheral blood between 1–3 hour post-injection and the cell counts returned to baseline within 6 hours. At their maximum tolerated doses (MTD), TG-0054 increased CXCR4+ cells by 28.7-fold in mice. Furthermore, TG-0054 efficiently mobilized CD34+ (14.5-fold) and CD133+ (7.9-fold) cells. The combined effects of TG-0054 and G-CSF on HSCs and EPCs mobilization from the bone marrow in mice were also investigated. G-CSF (100 μg/kg/day) was administered subcutaneously (SC) from Day 1 to Day 4 followed by a single IV injection of TG-0054 or AMD3100 on Day 5. Synergistic effects were observed in all cell types in mice receiving combination of G-CSF and 50 mg/kg of TG-0054 (total WBC 23.0-fold, CXCR4+ 29.0-fold, CD34+ 37.1-fold, CD133+ 110.8-fold of increase in combination group). Conclusion It was concluded that TG-0054 was a potent and selective CXCR4 antagonist intended for the use of stem cell transplant and other clinical indications. It showed strong stem cell mobilization activity comparable to G-CSF when used alone, and demonstrated synergistic effects when combined with G-CSF in a nonclinical model. Disclosures: Huang: TaiGen Biotechnology Inc.: Employment. Liu:TaiGen Biotechnology Inc.: Employment. Yen:TaiGen Biotechnology Inc.: Employment. Chen:TaiGen Biotechnology Inc.: Consultancy. Chen:TaiGen Biotechnology Inc.: Consultancy. King:TaiGen Biotechnology Inc.: Employment. Hsu:TaiGen Biotechnology, Inc.: Membership on an entity's Board of Directors or advisory committees.


Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 2433-2433
Author(s):  
Medhat Shehata ◽  
Rainer Hubmann ◽  
Martin Hilgarth ◽  
Susanne Schnabl ◽  
Dita Demirtas ◽  
...  

Abstract Abstract 2433 Chronic lymphocytic leukemia (CLL) is characterized by the clonal expansion of B lymphocytes which typically express CD19 and CD5. The disease remains incurable and recurrence often occurs after current standard therapies due to residual disease or probably due to the presence of therapy-resistant CLL precursors. Based on the growing evidence for the existence of leukemia stem cells, this study was designed to search for putative CLL precursors/stem cells based on the co-expression of CLL cell markers (CD19/CD5) with the hematopoietic stem cell marker (CD34). Forty seven CLL patients and 17 healthy persons were enrolled in the study. Twenty four patients had no previous treatment and 23 had pre-therapy. Twenty two patients were in Binet stage C and 25 patients in B. Twenty two patients had unmutated and 18 mutated IgVH gene (7: ND). Cytogenetic analysis by FISH showed that 14 patients had del 13q, 8 had del 11q, 4 had del 17p and 9 had trisomy 12. Peripheral blood and bone marrow mononuclear cells were subjected to multi-colour FACS analysis using anti-human antibodies against CD34, CD19 and CD5 surface antigens. The results revealed the presence of triple positive CD34+/CD19+/CD5+ cells in CLL samples (mean 0.13%; range 0.01–0.41) and in healthy donors (0.31%; range 0.02–0.6) within the CD19+ B cells. However, due to the high leukocyte count in CLL patients, the absolute number of these cells was significantly higher in CLL samples (mean: 78.7; range 2.5–295 cells /μL blood) compared to healthy persons (mean: 0.45: range 0.04–2.5 cells/μl)(p<0,001). These triple positive “putative CLL stem cells” (PCLLSC) co-express CD133 (67%), CD38 (87%), CD127 (52%), CD10 (49%), CD20 (61%), CD23 (96%), CD44 (98%) and CD49d (74%). FISH analysis on 4 patients with documented chromosomal abnormalities detected the corresponding chromosomal aberrations of the mature clone in the sorted CD34+/CD5+/CD19+ and/or CD34+/CD19-/CD5- cells but not in the CD3+ T cells. Multiplex RT-PCR analysis using IgVH family specific primer sets confirmed the clonality of these cells. Morphologically, PCLLSC appeared larger than lymphocytes with narrow cytoplasm and showed polarity and motility in co-culture with human bone marrow stromal cells. Using our co-culture microenvironment model (Shehata et al, Blood 2010), sorted cell fractions (A: CD34+/19+/5+, B: CD34+/19-/5- or C: CD34-/CD19+/5+) from 4 patients were co-cultured with primary autologous human stromal cells. PCLLSC could be expanded in the co-culture to more than 90% purity from fraction A and B but not from fraction C. These cells remained in close contact or migrated through the stromal cells. PCLLSC required the contact with stromal cells for survival and died within 1–3 days in suspension culture suggesting their dependence on bone marrow microenvironment or stem cell niches. RT-PCR demonstrated that these cells belong to the established CLL clone. They also eexpress Pax5, IL-7R, Notch1, Notch2 and PTEN mRNA which are known to play a key role in the early stages of B cells development and might be relevant to the early development of the malignant clone in CLL. Using NOD/SCID/IL2R-gamma-null (NOG) xenogeneic mouse system we co-transplanted CLL cells from 3 patients (5 million PBMC/mouse) together with autologous bone marrow stromal cells (Ratio: 10:1). The percentage of PCLLSC in the transplanted PBMC was 0.18% (range 0.06–0.34%). Using human-specific antibodies, human CD45+ cells were detected in peripharal blood of the mice (mean 0.9 % range 0.47–1.63%) after 2 months of transplantation. More than 90% of the human cells were positive for CD45 and CD5. Among this population, 26% (range 15–35%) of the cells co-expressed CD45, CD19, CD5 and CD34 and thus correspond to the PCLLSC. In conclusion, our data suggest the existence of putative CLL precursors/stem cells which reside within the CD34+ hematopoietic stem cell compartment and carry the chromosomal aberrations of the established CLL clone. These cells could be expanded in vitro in a bone marrow stroma-dependent manner and could be engrafted and significantly enriched in vivo in NOG xenotransplant system. Further characterization and selective targeting and eradication of these cells may pave the way for designing curative therapeutic strategies for CLL. Disclosures: No relevant conflicts of interest to declare.


Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 2476-2476
Author(s):  
Kasia Mierzejewska ◽  
Ewa Suszynska ◽  
Sylwia Borkowska ◽  
Malwina Suszynska ◽  
Maja Maj ◽  
...  

Abstract Background Hematopoietic stem/progenitor cells (HSPCs) are exposed in vivo to several growth factors, cytokines, chemokines, and bioactive lipids in bone marrow (BM) in addition to various sex hormones circulating in peripheral blood (PB). It is known that androgen hormones (e.g., danazol) is employed in the clinic to treat aplastic anemia patients. However, the exact mechanism of action of sex hormones secreted by the pituitary gland or gonads is not well understood. Therefore, we performed a complex series of experiments to address the influence of pregnant mare serum gonadotropin (PMSG), luteinizing hormone (LH), follicle-stimulating hormone (FSH), androgen (danazol) and prolactin (PRL) on murine hematopoiesis. In particular, from a mechanistic view we were interested in whether this effect depends on stimulation of BM-residing stem cells or is mediated through the BM microenvironment. Materials and Methods To address this issue, normal 2-month-old C57Bl6 mice were exposed or not to daily injections of PMSG (10 IU/mice/10 days), LH (5 IU/mice/10 days), FSH (5 IU/mice/10 days), danazol (4 mg/kg/10 days) and PRL (1 mg/day/5days). Subsequently, we evaluated changes in the BM number of Sca-1+Lin–CD45– that are precursors of long term repopulating hematopoietic stem cells (LT-HSCs) (Leukemia 2011;25:1278–1285) and bone forming mesenchymal stem cells (Stem Cell & Dev. 2013;22:622-30) and Sca-1+Lin–CD45+ hematopoietic stem/progenitor cells (HSPC) cells by FACS, the number of clonogenic progenitors from all hematopoietic lineages, and changes in peripheral blood (PB) counts. In some of the experiments, mice were exposed to bromodeoxyuridine (BrdU) to evaluate whether sex hormones affect stem cell cycling. By employing RT-PCR, we also evaluated the expression of cell-surface and intracellular receptors for hormones in purified populations of murine BM stem cells. In parallel, we studied whether stimulation by sex hormones activates major signaling pathways (MAPKp42/44 and AKT) in HSPCs and evaluated the effect of sex hormones on the clonogenic potential of murine CFU-Mix, BFU-E, CFU-GM, and CFU-Meg in vitro. We also sublethally irradiated mice and studied whether administration of sex hormones accelerates recovery of peripheral blood parameters. Finally, we determined the influence of sex hormones on the motility of stem cells in direct chemotaxis assays as well as in direct in vivo stem cell mobilization studies. Results We found that 10-day administration of each of the sex hormones evaluated in this study directly stimulated expansion of HSPCs in BM, as measured by an increase in the number of these cells in BM (∼2–3x), and enhanced BrdU incorporation (the percentage of quiescent BrdU+Sca-1+Lin–CD45– cells increased from ∼2% to ∼15–35% and the percentage of BrdU+Sca-1+Lin–CD45+ cells increased from 24% to 43–58%, Figure 1). These increases paralleled an increase in the number of clonogenic progenitors in BM (∼2–3x). We also observed that murine Sca-1+Lin–CD45– and Sca-1+Lin–CD45+ cells express sex hormone receptors and respond by phosphorylation of MAPKp42/44 and AKT in response to exposure to PSMG, LH, FSH, danazol and PRL. We also observed that administration of sex hormones accelerated the recovery of PB cell counts in sublethally irradiated mice and slightly mobilized HSPCs into PB. Finally, in direct in vitro clonogenic experiments on purified murine SKL cells, we observed a stimulatory effect of sex hormones on clonogenic potential in the order: CFU-Mix > BFU-E > CFU-Meg > CFU-GM. Conclusions Our data indicate for the first time that not only danazol but also several pituitary-secreted sex hormones directly stimulate the expansion of stem cells in BM. This effect seems to be direct, as precursors of LT-HSCs and HSPCs express all the receptors for these hormones and respond to stimulation by phosphorylation of intracellular pathways involved in cell proliferation. These hormones also directly stimulated in vitro proliferation of purified HSPCs. In conclusion, our studies support the possibility that not only danazol but also several other upstream pituitary sex hormones could be employed to treat aplastic disorders and irradiation syndromes. Further dose- and time-optimizing mouse studies and studies with human cells are in progress in our laboratories. Disclosures: No relevant conflicts of interest to declare.


Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 3728-3728
Author(s):  
Samuel Milanovich ◽  
Jeremy Allred ◽  
Jonathan Peterson ◽  
Cary Stelloh ◽  
Sridhar Rao

Abstract Stem cells play key roles in early normal development (e.g. embryonic stem cells (ESCs)), maintenance of adult organs (e.g. hematopoietic stem cells (HSCs)) and in some cancers (e.g. leukemia stem cells). To what degree these different types of stem cells rely upon shared versus distinct transcriptional programs remains controversial. Sall4 is a zinc finger transcription factor that exists in two distinct splice isoforms, Sall4a (long) and Sall4b (short). Sall4 has been implicated in embryonic, hematopoietic and malignant stem cell transcriptional regulation. Additionally, Sall4 has been proposed as a potential means of ex-vivo hematopoietic stem cell expansion prior to transplantation. Sall4 isoform-specific differences have been described in ESCs, with Sall4b shown to be critical for maintaining ESC “stemness”. Here we investigate the role of Sall4 isoforms in pediatric acute myeloid leukemia (AML) and murine hematopoiesis to unravel shared versus unique transcriptional programs across different stem cell types. Quantitative real time PCR shows that Sall4b is the predominant Sall4 isoform in murine HSCs and lin-, Sca1+, cKit+ (LSK) cells. Sall4b expression decreases in early lineage-committed progenitors, while Sall4a expression is minimal to absent across murine HSCs and progenitors. Next, we evaluated seven pediatric AML samples and found highly variable Sall4 expression across AML cases. All samples had measurable Sall4a and Sall4b; in 3/7 cases Sall4a and Sall4b expression was similar to that of ESCs, in the other 4 cases Sall4 expression was minimal (<3% of ESCs). To study overexpression of Sall4, we used a murine stem cell retrovirus system to express Sall4a or Sall4b. Bone marrow was harvested from C57/BL6 mice and lineage-committed cells were removed by magnetic column separation. Lineage-negative bone marrow was infected with either empty vector, Sall4a or Sall4b. Transduced bone marrow was then cultured in methylcellulose media to assess colony forming capacity and proliferation in vitro or transplanted in syngeneic mice to assess engraftment and hematopoietic reconstitution in vivo. Sall4a or Sall4b overexpression caused diminished colony forming capacity and cellular proliferation in vitro compared to bone marrow transduced with empty vector (Figure 1). In bone marrow transplant assays, all mice (4/4) transplanted with Sall4b-transduced bone marrow following lethal irradiation succumbed to bone marrow failure within 10 days of transplant. Transplantation of Sall4b-transduced bone marrow into sublethally irradiated mice failed to contribute to hematopoiesis as measured by peripheral blood leukocyte GFP expression (encoded by the viral vector). Together, this data shows that Sall4b-transduced hematopoietic cells fail to engraft and reconstitute hematopoiesis in vivo. We postulated that this phenotype might be mediated through the interaction of Sall4 with Bmi1. Bmi1 is a member of the polycomb complex necessary for normal hematopoiesis, and is known to be bound by Sall4. In preliminary experiments, we have found that overexpression of Sall4 leads to decreased Bmi1 expression at 48 hours post-infection compared to bone marrow infected with empty vector.Figure 1Lin- bone marrow expressing Sall4a, Sall4b or empty vector was cultured in methylcellulose; plates were flushed and replated out to three generations. Colony forming units were assessed (A) and viable cells were counted (B) after 7-10 days in culture.Figure 1. Lin- bone marrow expressing Sall4a, Sall4b or empty vector was cultured in methylcellulose; plates were flushed and replated out to three generations. Colony forming units were assessed (A) and viable cells were counted (B) after 7-10 days in culture. In conclusion, our data shows that Sall4b is expressed in murine hematopoietic stem cells and progenitors, suggesting that Sall4b but not Sall4a influences a hematopoietic cell fate. Additionally, Sall4 expression is variable in AML specimens, implicating a potential pathogenic role in some leukemias, while others are Sall4-independent. Lastly, Sall4 overexpression is associated with decreased expression of the critical hematopoietic gene Bmi1. Together this data suggests that hematopoiesis is dependent upon appropriately regulated Sall4 expression with alterations leading to impaired proliferation and self-renewal. These effects on hematopoiesis appear to be mediated at least in part through a dose-dependent effect on Bmi1 expression. Future studies will evaluate other genes targeted by Sall4 in hematopoiesis and leukemia to define Sall4-dependent gene signatures in normal versus malignant hematopoiesis. Disclosures: No relevant conflicts of interest to declare.


Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 2500-2500
Author(s):  
Tellechea Maria Florencia ◽  
Flavia S. Donaires ◽  
Tiago C. Silva ◽  
Lilian F. Moreira ◽  
Yordanka Armenteros ◽  
...  

Aplastic anemia (AA) is characterized by a hypoplastic bone marrow associated with low peripheral blood counts. In acquired cases, the immune system promotes hematopoietic stem and progenitor cell (HSPC) depletion by the action of several pro-inflammatory Th1 cytokines. The current treatment options for severe cases consist of sibling-matched allogeneic hematopoietic stem cell transplantation (HSCT) and immunosuppressive therapy (IST) with anti-thymocyte globulin, cyclosporine, and eltrombopag. However, most patients are not eligible for HSCT and, although about 85% of patients respond to IST with eltrombopag, a proportion of patients eventually relapse, requiring further therapies. Failure to respond adequately to immunosuppression may be attributed to the scarcity of HSPCs at the time of diagnosis. Induced pluripotent stem cells (iPSCs) are potentially an alternative source of patient-specific hematopoietic cells. Patient-specific HSPCs derived from in vitro iPSC differentiation may serve as a tool to study the disease as well as a source of hematopoietic tissue for cell therapies. The pyrimidoindole molecule UM171 induces ex vivo expansion of HSCs of human cord and peripheral blood and bone marrow, but the pathways modulated by this molecule are not well understood. Here we evaluated the hematopoietic differentiation potential of iPSCs obtained from patients with acquired AA. We further determined the effects of UM171 on this differentiation process. First, we derived iPSCs from 3 patients with acquired AA after treatment (1 female; average age, 31 years; 2 partial responders, 1 complete responder) and 3 healthy subjects (3 females; average age, 61 years) and induced differentiation in vitro through the embryoid body system in cell feeder and serum-free medium supplemented with cytokines. The hematopoietic differentiation of healthy-iPSCs yielded 19% ± 8.1% (mean ± SEM) of CD34+cells after 16 days in culture, in contrast with 11% ± 4.9% of CD34+cells obtained from the differentiation of AA-iPSCs, which corresponds to a 1.7-fold reduction in CD34+cell yield. The total number of erythroid and myeloid CFUs was lower in the AA-iPSC group as compared to healthy-iPSCs (12±4.2 vs.24±7.2; respectively; p<0.03). These findings suggest that erythroid-derived AA-iPSC have an intrinsic defect in hematopoietic differentiation. Next, we tested whether UM171 modulated hematopoietic differentiation of AA-iPSCs. We found that UM171 significantly stimulated the differentiation of both healthy and AA-iPSCs. In the healthy-iPSC group, the percentage of CD34+cells was 1.9-fold higher when treated with UM171 compared to controls treated with DMSO (37% ± 7.8% vs.19% ± 8.1%; respectively; p<0.03) and in AA-iPSCs the increase was 3.9-fold (45% ± 11% vs. 11% ± 4.9%; p<0.07). The clonogenic capacity of progenitors to produce erythroid and myeloid colonies also was augmented in both groups in comparison to DMSO (28±11 vs. 23±7.2) for healthy-iPSCs and for AA-iPSCs (23±8.5 vs. 12±4.2, p<0.06). We then investigated the molecular pathways influenced by UM171. The transcriptional profile of differentiated CD34+cells showed that UM171 up-regulated genes involved in early hematopoiesis from mesoderm (BRACHYURY and MIXL1) and primitive streak specification (APELA and APLNR), to hemangioblasts and primitive hematopoietic progenitor commitment (TDGF1, SOX17, and KLF5). We also observed the up-regulation of pro-inflammatory NF-kB activators (MAP4K1, ZAP70, and CARD11) and the anti-inflammatory gene PROCR, a marker of cultured HSCs and an NF-kB inhibitor. This balanced network has been previously suggested to be modulated by UM171 (Chagraoui et. al. Cell Stem Cell 2019). Taken together, our results showed that acquired AA-iPSCs may have intrinsic defects that impair hematopoietic differentiation in vitro. This defect may be atavic to the cell or, alternatively, the consequence of epigenetic changes in erythroid precursors provoked by the immune attack. In addition, our findings demonstrate that UM171 significantly stimulate the hematopoietic differentiation of AA-iPSCs and identified a novel molecular mechanism for UM171 as an enhancer of early hematopoietic development programs. These observations may be valuable for improving the achievement of de novo hematopoietic cells. Disclosures No relevant conflicts of interest to declare.


Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 5444-5444
Author(s):  
Michael Schmitt ◽  
Amy Publicover ◽  
Kim H. Orchard ◽  
Anita Schmitt ◽  
Panagiotis Tsirigotis ◽  
...  

Abstract Introduction Hematopoietic growth factors including G-CSF are manufactured by the use of recombinant technology. When biologically equivalent agents are manufactured they are termed “biosimilars”. The use of biosimilars in general and in particular for peripheral blood hematopoietic stem cell (PBSC) mobilization has stimulated an ongoing debate regarding their efficacy and safety. The use of biosimilar G-CSF (Ratiograstim®, Tevagrastim®, Biograstim®, Zarzio®, Nivestim®) was approved by the European Medicines Agency (EMA) for all the registered indications of the originator (Neupogen®) including mobilization of stem cells. Methods We performed a comprehensive review of published reports on the use of biosimilar G-CSF covering patients with hematological malignancies as well as healthy donors that underwent stem cell mobilization with a biosimilar G-CSF for autologous and allogeneic stem cell transplantation, evaluating mobilization yield and safety profile as well as engraftment and transplantation outcome. Results An extensive literature review produced 903 patients mostly with hematological malignancies as well as healthy donors that underwent successful autologous or allogeneic stem cell mobilization respectively using a biosimilar G-CSF (Ratiograstim®/Tevagrastim® or Zarzio®). A total of 519 patients or donors underwent stem cell mobilization with Ratiograstim®/Tevagrastim®, while 384 patients or donors underwent stem cell mobilization with Zarzio®. The indication for stem cell mobilization in hematology patients included 326 with multiple myeloma, 273 with Non-Hodgkin’s lymphoma (NHL), 79 with Hodgkin’s lymphoma (HL), and others. 155 sibling or volunteer unrelated donors were mobilized using either Ratiograstim®/Tevagrastim® or Zarzio®. Biosimilar G-CSF based stem cell mobilization for both autologous and allogeneic transplantation resulted in good mobilization of CD34+ stem cells with side effects similar to reference G-CSF. Post transplantation engraftment did not significantly differ from results previously documented with the originator filgrastim (Neupogen®) in historical controls. The side effects experienced by the patients or donors mobilized by biosimilar G-CSF were minimal and were comparable to those of originator G-CSF. (More detailed results regarding yield, engraftment and side effects will be disclosed in the conference.) Conclusions In summary, we present the published experience for the use of biosimilar G-CSFs in more than 900 patients and normal family related and volunteer unrelated donors. Both the toxicity profile PBSC yield and efficacy seem equivalent to historical data with the reference G-CSF. Until results from multi-center randomized clinical trials that directly compare biosimilar G-CSF with the originator G-CSF are reported, it is important to collect and summarize all of the available clinical experience in order to allow the transplant community to make informed decisions regarding the choice of G-CSF. Disclosures: Schmitt: AMGEN: Honoraria; Teva: Consultancy, Honoraria, Research Funding, Travel Grant, Research Grants Other. Publicover:Teva: Travel Grants, unrestricted educational grant Other. Orchard:Teva: Honoraria, Travel Grants, research grants Other. Schmitt:TEVA: Travel grant Other. Nagler:Teva: Honoraria, Travel grants, research grants Other.


Blood ◽  
1992 ◽  
Vol 80 (12) ◽  
pp. 3044-3050 ◽  
Author(s):  
S Okada ◽  
H Nakauchi ◽  
K Nagayoshi ◽  
S Nishikawa ◽  
Y Miura ◽  
...  

c-kit is expressed on hematopoietic stem cells and progenitor cells, but not on lymphohematopoietic differentiated cells. Lineage marker- negative, c-kit-positive (Lin-c-kit+) bone marrow cells were fractionated by means of Ly6A/E or Sca-1 expression. Lin-c-kit+Sca-1+ cells, which consisted of 0.08% of bone marrow nucleated cells, did not contain day-8 colony-forming units-spleen (CFU-S), but 80% were day-12 CFU-S. One hundred cells rescued the lethally irradiated mice and reconstituted hematopoiesis. On the other hand, 2 x 10(3) of Lin-c- kit+Sca-1- cells formed 20 day-8 and 11 day-12 spleen colonies, but they could not rescue the lethally irradiated mice. These data indicate that Lin-c-kit+Sca-1+ cells are primitive hematopoietic stem cells and that Sca-1-cells do not contain stem cells that reconstitute hematopoiesis. Lin-c-kit+Sca-1+ cells formed no colonies in the presence of stem cell factor (SCF) or interleukin-6 (IL-6), and only 10% of them formed colonies in the presence of IL-3. However, approximately 50% of them formed large colonies in the presence of IL-3, IL-6, and SCF. Moreover, when single cells were deposited into culture medium by fluorescence-activated cell sorter clone sorting system, 40% of them proliferated on a stromal cell line (PA-6) and proliferated for more than 2 weeks. In contrast, 15% of the Lin-c- kit+Sca-1-cells formed colonies in the presence of IL-3, but no synergistic effects were observed in combination with SCF plus IL-6 and/or IL-3. Approximately 10% proliferated on PA-6, but most of them degenerated within 2 weeks. The population ratio of c-kit+Sca-1+ to c-kit+Sca-1- increased 2 and 4 days after exposure to 5-fluorouracil (5-FU). These results are consistent with the relative enrichment of highly proliferative colony-forming cells by 5-FU. These data show that, although c-kit is found both on the primitive hematopoietic stem cells and progenitors, Sca-1+ cells are more primitive and respond better than Sca-1- cells to a combination of hematopoietic factors, including SCF and stromal cells.


2022 ◽  
Vol 13 (1) ◽  
Author(s):  
Francesco Da Ros ◽  
Luca Persano ◽  
Dario Bizzotto ◽  
Mariagrazia Michieli ◽  
Paola Braghetta ◽  
...  

Abstract Background Dissection of mechanisms involved in the regulation of bone marrow microenvironment through cell–cell and cell–matrix contacts is essential for the detailed understanding of processes underlying bone marrow activities both under physiological conditions and in hematologic malignancies. Here we describe Emilin-2 as an abundant extracellular matrix component of bone marrow stroma. Methods Immunodetection of Emilin-2 was performed in bone marrow sections of mice from 30 days to 6 months of age. Emilin-2 expression was monitored in vitro in primary and mesenchymal stem cell lines under undifferentiated and adipogenic conditions. Hematopoietic stem cells and progenitors in bone marrow of 3- to 10-month-old wild-type and Emilin-2 null mice were analyzed by flow cytometry. Results Emilin-2 is deposited in bone marrow extracellular matrix in an age-dependent manner, forming a meshwork that extends from compact bone boundaries to the central trabecular regions. Emilin-2 is expressed and secreted by both primary and immortalized bone marrow mesenchymal stem cells, exerting an inhibitory action in adipogenic differentiation. In vivo Emilin-2 deficiency impairs the frequency of hematopoietic stem/progenitor cells in bone marrow during aging. Conclusion Our data provide new insights in the contribution of bone marrow extracellular matrix microenvironment in the regulation of stem cell niches and hematopoietic progenitor differentiation.


Blood ◽  
1999 ◽  
Vol 93 (1) ◽  
pp. 80-86 ◽  
Author(s):  
Shai Erlich ◽  
Silvia R.P. Miranda ◽  
Jan W.M. Visser ◽  
Arie Dagan ◽  
Shimon Gatt ◽  
...  

Abstract The general utility of a novel, fluorescence-based procedure for assessing gene transfer and expression has been demonstrated using hematopoietic stem and progenitor cells. Lineage-depleted hematopoietic cells were isolated from the bone marrow or fetal livers of acid sphingomyelinase–deficient mice, and retrovirally transduced with amphotropic or ecotropic vectors encoding a normal acid sphingomyelinase (ASM) cDNA. Anti–c-Kit antibodies were then used to label stem- and progenitor-enriched cell populations, and the Bodipy fluorescence was analyzed in each group after incubation with a Bodipy-conjugated sphingomyelin. Only cells expressing the functional ASM (ie, transduced) could degrade the sphingomyelin, thereby reducing their Bodipy fluorescence as compared with nontransduced cells. The usefulness of this procedure for the in vitro assessment of gene transfer into hematopoietic stem cells was evaluated, as well as its ability to provide an enrichment of transduced stem cells in vivo. To show the value of this method for in vitro analysis, the effects of retroviral transduction using ecotropic versus amphotropic vectors, various growth factor combinations, and adult bone marrow versus fetal liver stem cells were assessed. The results of these studies confirmed the fact that ecotropic vectors were much more efficient at transducing murine stem cells than amphotropic vectors, and that among the three most commonly used growth factors (stem cell factor [SCF] and interleukins 3 and 6 [IL-3 and IL-6]), SCF had the most significant effect on the transduction of stem cells, whereas IL-6 had the most significant effect on progenitor cells. In addition, it was determined that fetal liver stem cells were only approximately twofold more “transducible” than stem cells from adult bone marrow. Transplantation of Bodipy-selected bone marrow cells into lethally irradiated mice showed that the number of spleen colony-forming units that were positive for the retroviral vector (as determined by polymerase chain reaction) was 76%, as compared with 32% in animals that were transplanted with cells that were nonselected. The methods described within this manuscript are particularly useful for evaluating hematopoietic stem cell gene transfer in vivo because the marker gene used in the procedure (ASM) encodes a naturally occurring mammalian enzyme that has no known adverse effects, and the fluorescent compound used for selection (Bodipy sphingomyelin) is removed from the cells before transplantation.


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