Archive for the ‘Bone Marrow Stem Cells’ Category

His name means brave in Hindi. And for four year-old Veer Gudhka that couldnt be more appropriate.

For while the bubbly little boy might look fighting fit, he actually has just months to live.

Veer suffers from a rare blood disorder called Fanconi anaemia, which results in a decreased production of all types of blood cells.

But a stem cell donor will save his life.

In a heartfelt video message, the plucky toddler asks Sunday Mirror readers: Please be my life-saver? Will you be my superhero?

And today his family are appealing to those from BAME communities to help by signing up to the Anthony Nolan stem cell register.

Mum Kirpa and dad Nirav know the odds are stacked against them getting that all-important call because they are of Indian descent.

While 69 per cent of Northern European patients find the best possible stem cell match from a stranger, this drops to just 20 per cent for those with black, Asian or ethnic minority backgrounds.

Currently only two per cent of the population is on the UK stem cell register.

And with Asians making up just six per cent of the UK population, there is a smaller pool of potential donors.

Veer was diagnosed with the blood disorder last August, after he started suffering from extreme fatigue, and was referred for tests.

Doctors said he would need a stem cell transplant within three years for a chance of survival.

They hoped to buy Veer some time by putting him on steroids to boost his blood counts. But his condition has deteriorated fast.

Recent tests at Great Ormond Street Hospital in London show he now has just three to four months to find a donor.

Kirpa and Nirav were both tested, along with Veers six-year-old sister Suhani, but none of them were a match.

A search on the global stem cell register also drew a blank.

And his dad has been trying to encourage his fellow countrymen and women in India to join the register.

They have even signed up a female battalion of the Indian Army.

Kirpa, 37, from Harrow, London, said: We just feel so scared were going to lose our cheeky, amazing little boy. To look at Veer you wouldnt know hes critically ill.

Like his name, hes been brave from the start. Hes undergone countless tests and hospital visits but has had a constant smile on his face.

"He knows he needs a superhero to step forward, but his optimism and enthusiasm are infectious and keep us all going.

She added: Going on the register is incredibly quick and donating cells if you match someone in need is painless.

You can join the Anthony Nolan stem cell register today.

Nine out of 10 people donate their stem cells through the bloodstream in a simple IV process called peripheral blood stem cell collection.

One in 10 will have their stem cells collected via the bone marrow itself, while under general anaesthetic. Doctors transplant the new, healthy cells via the patients bloodstream, where they begin to grow and create healthy red blood cells, white blood cells and platelets.

A perfect match from a donor can mean a lifelong cure.

Veers dad Nirav, 40, said: I only learned about the Anthony Nolan stem cell register two years ago and even then I assumed it would involve long and painful procedures.

We need to raise awareness to save lives in every community.

Continued here:
Boy, 4, may look fighting fit but only has months to live - unless you can save him - Mirror Online

INTRODUCTION

The ability to regenerate complex body parts varies considerably in the animal kingdom. While planarian and hydra are able to regenerate their entire bodies, many avian and mammalian species mostly stop at the wound healing stage without a reparative regeneration process (1). This disparity may result from complexity differences among organisms by nature, yet it leaves us the hope that we may learn from highly regenerative species to improve our own regenerative potential.

Zebrafish is known for its ability to regenerate multiple complex body structures (2). Among regenerable tissues, the caudal fin serves as a great model due to its faithful and rapid regeneration, ease of manipulation, and relatively low complexity. A key step in regeneration is the formation of the blastema, a layer of proliferative and undifferentiated cells that accumulates between the wound site and the wound epidermis following initial wound closure. This step occurs in response to appendage loss and is one of the key features that separates regenerative systems from nonregenerative systems. At later stages of regeneration, the blastema further proliferates and differentiates to regenerate the missing complex structures.

However, the molecular signatures of blastemal cell state transitions during regeneration in zebrafish remain elusive. The state of a cell can be represented by its collective gene expression profile, which has only been measured in bulk for all genes or in specific lineages of cells for a subset of genes during caudal fin regeneration. Prior work has shown that both proliferation of progenitors and dedifferentiation of adult lineage cells contribute to the blastema (38). Progenitors respond to injury cue and proliferate as in normal development. Cells derived from mature adult lineages, however, lose their lineage-specific markers while obtaining progenitor-like markers when they proliferate. Neither type of cell gains multipotency, but rather, they proliferate and regenerate with lineage restrictions. The limited resolution and throughput of these approaches have prevented a more systematic understanding of blastema cells. The advent of single-cell transcriptomic technologies promises to reveal signals masked at the bulk tissue level (9), granting us an opportunity to define and monitor cellular state transition in regenerating fin at an unprecedented resolution.

In this study, we generated single-cell transcriptomic maps of regenerating fin tissue. These maps allowed us to separate the contribution from different cell types and track the transcriptomic dynamics in cell state transitions during regeneration. By comparing with the profiles obtained from uninjured fin tissue, we identified cell types involved in regeneration. We demonstrated the activation of cell cyclerelated programs shared across cell types as well as cell typespecific programs. Furthermore, we defined the heterogeneity in both epithelial and blastemal populations and their functional relations to the regeneration process.

To better understand cell type involvement in fin regeneration, we characterized single-cell transcriptional landscapes for both preinjury and regenerating caudal fin tissues using the 10x Genomics platform (see Materials and Methods and table S1) (9). We sampled regenerating fins from 1, 2, and 4 days post-amputation (dpa) time points to interrogate the stages of blastema formation, outgrowth, and maintenance (Fig. 1A). Fin samples were collected from multiple fish to control for individual variation while at the same position along the proximal-distal axis to avoid positional effects. To establish the transcriptional ground states for each cell type in the fin tissue, we first focused on cells collected from the preinjury time point. Via an unsupervised clustering of 4134 cells, we identified epithelial cells (epcam and cdh1), hematopoietic cells (mpeg1.1 and cxcr3.2), and mesenchymal cells (msx1b and twist1a) (fig. S1, A and B) (1014). Epithelial cells are from three transcriptionally distinct subgroups, representing the superficial (krt4), intermediate (tp63), and basal layers (tp63 and krtt1c19e) of the epithelium (fig. S1, A and B) (15, 16).

(A) General experimental design. Zebrafish caudal fin tissues at preinjury and 1/2/4 dpa stages were collected. (B) Clustering assignments for caudal fin cells collected from each stage. Uniform Manifold Approximation and Projection (UMAP) axes were calculated from the integrated cells dataset as in (C). (C) Clustering assignments for caudal fin cells collected from both preinjury and regenerating stages. Cells were plotted on UMAP axes. Color coding is the same as in (E). (D) Percentage distribution of the major cell types captured in caudal fin, grouped by their stage of collection. Color coding is the same as in (E). (E) Differential expressions of the key marker genes by the identified major cell types. Color gradient: normalized relative expression level. Dot size: percentage of cells in the cluster that express the specified gene.

To determine whether the same cell types existed in the regenerating stages, we performed analysis using two different approaches: (i) Cells from each stage were clustered independently, and (ii) cells from both uninjured fins and injured fins were integrated through the anchoring approach (see Materials and Methods; Fig. 1, B, C, and E; and table S2) (17). For both approaches, we regressed out cell cycle effects before principal components analysis (PCA). Agreement between cluster assignments was measured using Hubert and Arabies adjusted Rand index (ARI). An average ARI of 0.86 (preinjury, 0.86; 1 dpa, 0.85; 2 dpa, 0.90; and 4 dpa, 0.83) indicated that clustering results generated using the two approaches were highly consistent. Cell types identified in the preinjury cells presented consistently across all regenerating stages, suggesting that regenerating fins contain the same cell types as the preinjury fins.

New regenerates are built up by the proliferation and migration of cells located at a number of fin segments away from the amputation plane (2). In response to injury cues, these cells gained the ability to detach from local tissue, enter cell cycle, and migrate toward the wound site while undergoing transcriptional reprogramming. We computationally separated S phase, G2-M phase, and G1-phase cells based on the expression level of cell cyclerelated genes and performed clustering analysis using only S phase cells (see Materials and Methods and fig. S2A). In this cycling cell population, we identified epithelial, mesenchymal, and hematopoietic cell groups as before (Fig. 2, A to C, and table S3). Our data support a model in which cells likely keep their original identities during proliferation.

(A) Cell type clustering of S phase cells plotted onto UMAP axes calculated by S phase cell only. Cells are colored by the general cell types merged from major cells types in Fig. 1B. (B) Stage distribution of S phase cells. Cells were plotted on the same UMAP axes as in (A) and colored by stage when the cells were collected. (C) Relative expression levels of the top 30 differentially expressed genes from each cluster of only S phase cells. (D) Venn diagrams of numbers of genes shared between the cell cycleactivated genetic programs. Left, included all genes; right, included only cell cyclerelated genes (see Materials and Methods).

Next, we asked whether different regenerating cell types exhibited similar or distinct cell cycle regulations. To this end, we identified genes up-regulated in S phase cells compared to G1 phase cells in each cell type, respectively (logFC, >0.25; minimum percentage, >10%). Of the 1098 differentially expressed genes, 161 were shared across all three groups of comparisons (Fig. 2D and table S4). Of these shared genes, at least 54 genes were related to cell cycle regulation, underscoring a shared program governing cell cycle reentry (criteria described in Materials and Methods). In contrast, hundreds of genes differentially highly expressed in S phase exhibited cell typespecific pattern, of which dozens were related to cell cycle (Fig. 2D). We next evaluated the degree of conservation of these enriched genes by asking what fraction did not have human orthologs that had been curated in the Metascape database (18). Twenty-five percent of genes in the epithelial cellspecific group had no human ortholog, whereas all shared groups had at most 15% genes without a human ortholog, suggesting that enriched genes shared by cell types were more evolutionarily conserved (fig. S2C).

Some cell typespecific S-G1 enriched genes were also expressed in a cell typespecific manner regardless of their cell cycle phases: For example, psmb8a and psmb9a shared similar epithelial-hematopoietic enrichments (fig. S2D). The human homologs of these genes (PSMB8 and PSMB9) encode 5i and 1i subunits of the immunoproteasome (19). Together with 2i and PA28 subunits of the proteasome, they turn the proteasome into immunoproteasome and take part in immune response (20). Immunoproteasome digests peptides more efficiently, promoting antigen presentation by a major histocompatibility complex (MHC) class I molecule. Although they did not pass the differential expression criteria in the S-G1 comparison, zebrafish psmb10, psme1, and psme2 shared a differential expression signature similar to that of psmb8a and psmb9a, suggesting that zebrafish might use the same group of subunits for the assembly of immunoproteasomes that might help increase immune responses during regeneration, especially in epithelial and hematopoietic cells (fig. S2, D and E). In addition, we found three genes that shared the same expression signature with the immunoproteasome subunits (psmb13a, psmb12, and psma6l) (fig. S2E) without known human or mouse homologs, suggesting that they might form zebrafish-specific proteasomes with functional relevance to regeneration (19).

Consistent with current knowledge, we observed three transcriptionally distinct subgroups in the preinjury epcam+ epithelial cells, representing the superficial, intermediate, and basal layers of the adult zebrafish epithelium (Fig. 3A and fig. S1B) (15, 16). By integrating cells from all stages during regeneration, we found clusters of cells that corresponded to all three layers of the epithelium after injury (Fig. 1, B and C). In addition, we captured a rare agr2+ population (referred to as mucosal-like epithelium herein) that was too small to be clustered by itself in the preinjury stage (Fig. 1E). These cells shared general epithelial features with the other epithelial layers but exhibited higher expression of a unique set of 200 genes. We examined the expression distribution of the orthologs of these genes in human tissues (The Human Protein Atlas, http://proteinatlas.org/) (21). Among the top 30 genes with human orthologs, 11 showed enriched expressions in proximal digestive or gastrointestinal tract and another 11 in bone marrow of blood lineages, suggesting that this population is analogous to cells in the mucosa in mammalian systems (table S2). In zebrafish, agr2 is required for the differentiation of the mucosal-producing goblet cells in the intestinal epithelium (22). To confirm the cell typespecific expression pattern of this gene in the fin tissue, we performed in situ hybridization on agr2 in both uninjured and regenerating fin tissues (see Materials and Methods). agr2 transcripts are scattered within the epithelium regardless of the sample collection stage and reflect a round morphology of the cell expressing it (fig. S3, A, C, E, and G to I). A proportion of agr2+ cells overlap with positive dark blue staining of Alcian blue in serial sections, suggesting that these cells are mucous cells that are known to exist in the caudal fin epithelium (fig. S3, B, D, and F) (23).

(A) Diagram of the stratified adult zebrafish epithelium. (B) Differential expressions of claudin family and keratin family genes in epithelial subgroups shown as a dot plot. Known epithelial markers krt4, fn1b, tp63, and krtt1c19e were included for comparison. Cells were first grouped by major cell types and then separated into preinjury and regenerating stages. Darkness of dot color: relative expression level. Dot size: percentage of cells in the cluster that express the specified gene. (C) In situ hybridization targeting krt1-19d, cldna, cldn1, and krt4 of 4-dpa fin tissues. Brown dots indicate positive RNA signals from target genes, while pale blue blocks represent hematoxylin-stained cell nuclei. Zoomed-in views are presented. Original images can be found in fig. S4. All epithelial layers are above the black dotted lines. (D) Clustering assignment of epithelial cells plotted on UMAP axes calculated with only epithelial cells. Cells are colored by their epithelial layer identity as in (A). (E) The same UMAP visualization as in (D), with cells colored by stage of collection. Arrows connect the groups of comparison, with a direction from preinjury stage to regenerating stages (1, 2, and 4 dpa). Numbers next to the green triangle: number of genes up-regulated in regenerating stage. Numbers next to the red triangle: number of genes down-regulated in regenerating stage. (F) Clustered GO enrichment for genes up-regulated in regenerating basal, intermediate, and superficial epithelial cells comparing to their preinjury counterparts. GTPase, guanosine triphosphatase; ER, endoplasmic reticulum; PKN, protein kinases N; snRNP, small nuclear ribonucleoprotein.

Although the same three-layer classification of epithelial cells could be defined when cells from regenerating stages were integrated with the preinjury cells, the expression of the commonly used layer-specific marker genes changed dramatically during regeneration: Superficial epithelial marker krt4 expanded into basal and intermediate layers of the epithelium, the intermediate layer marker fn1b was also highly expressed in the basal layer, and the basal epithelial marker krtt1c19e was barely detectable in the postinjury cell populations (Fig. 3B) (15, 16). To better understand the molecular features of the epithelial populations, we identified genes significantly more highly expressed in epithelial cells than in hematopoietic and mesenchymal cells and found that cell-cell junction genes ranked high in the list. Among these, genes from the claudin and keratin families were detected at a ratio 20-fold higher than that in randomly selected detectable genes (2 test, P value of <0.0001). We focused on expression patterns of all claudin and keratin genes in zebrafish and found that cldne, cldnf, krt1-19d, and krt17 labeled the superficial cluster; cldnh labeled the mucosal-like cluster; cldna, krt93, and krt94 labeled the intermediate cluster; and cldn1 and cldni labeled the basal cluster (Fig. 3B). Claudin genes are expressed in a tissue-specific manner in zebrafish and are generally considered to be the proteins responsible for regulating the paracellular permeability in the vertebrate epithelium (24). Their differential expression signature in both uninjured and regenerating tissues suggests that they might play important roles in maintaining the permeability in each epithelial population. On the other hand, the expression of keratin genes displayed less restriction across the three layers relative to claudin genes but stronger dependence on regenerating states (Fig. 3B). The differential expression signature suggests that they might perform epithelial subtyperelated function in regeneration. To verify their expression pattern, we performed RNA in situ hybridization targeting the known marker krt4 and new candidates, including krt1-19d, cldna, and cldn1 (Fig. 3C) as well as cldne, krt94, and cldni (fig. S4, A to H). Comparing with the known marker krt4, these genes exhibited more restricted expression patterns in epithelial layers, better representing the molecular signatures of different epithelial populations in the fin tissue regardless of regeneration status (Fig. 3, B and C).

The three epithelial layers were present across the regeneration stages albeit with varying proportions (Fig. 1D). The proportion of basal epithelial cells peaked at 2 dpa, reaching up to 42%, whereas the superficial layer epithelial cells decreased from 27 to 6% at 2 dpa (the coefficient of variations of cell proportions between biological replicates is below 15%). The observed compositional change of the two epithelial populations is consistent with a previous finding that the initial migration of superficial layer cells to the new regenerates is followed by replenishment by basal epithelial cells (25). This basal replenishment was also reflected in the two-dimensional Uniform Manifold Approximation and Projection (UMAP) calculation from only epithelial cells, in which preinjury cells were separated by their respective layers, whereas regenerating cells were closer in the projection space (Fig. 3, D and E). Superficial layer cells from before and after injury stages were in juxtaposition to each other, consistent with our knowledge that this layer of epithelial cells directly migrates to and covers the wound site (25). On the other hand, basal layer cells from before and after injury stages were more distantly separated, suggesting more dramatic changes between resting and regenerating basal epithelial cells.

To understand the mechanisms of epithelium regeneration, we compared the transcriptome between preinjury and regenerating cells for the three epithelial layers. Basal layer cells up-regulated 1271 genes and down-regulated 198 genes during regeneration; both were the highest numbers across all comparisons (numbers of differentially expressed genes were from Wilcoxon rank sum test, adjusted P value of < 0.01; Fig. 3E). We performed gene ontology (GO) enrichment analysis on genes up-regulated in the regenerating stage by layer and found both common and layer-specific programs associated with regeneration (18). All three layers were enriched for oxidative phosphorylation (dre00190), proteasome (dre03050), and cell redox homeostasis (GO:0045454). While basal and intermediate layer cells could be regulated by Rho guanosine triphosphatasemediated Wnt signaling for extracellular matrix organization and actin filament depolymerization, respectively (R-DRE-195258, R-DRE-5625740, R-DRE-195721, GO:0030198, and GO:0030042), superficial layer cells showed enrichment mainly for general transcriptional and translational regulations (Fig. 3F). When comparing the expression profiles between regenerating superficial epithelial and basal epithelial, we saw enrichment for antigen presentation and apoptosis features in the superficial layer (table S5). In addition, the superficial layer contained the lowest proportion of cells in S phase or G2-M phase, further supporting that superficial layer epithelium was most likely maintained by migration and proliferation from other layers (fig. S2B).

Subcluster identification within regenerating basal epithelial cells revealed two subgroups that represented different functionalities during regeneration, one labeled by distally distributed fgf24, while the other by proximally distributed lef1 (fig. S5, A to C) (26, 27). We compared expression profiles between group I (distal) and group II (proximal) cells and found that their suggested functionalities were consistent with their expected roles in regeneration: The distal subgroup (or distal wound epidermis) up-regulated genes associated with extracellular matrix degradation, and the proximal subgroup (or proximal wound epidermis) up-regulated genes associated with organization of extracellular matrix, skeletal system development, and negative regulation of locomotion (fig. S5, D and E). In addition, the increase of proximal cell proportion was accompanied by the decrease of distal cell proportion, suggesting that basal layer epithelium become gradually active in promoting blastema proliferation and differentiation during the initial regeneration process (fig. S5C). To confirm the distribution of these cells, we performed RNA in situ hybridization targeting two candidate genes, stmn1b and sema3b, one from each cluster. The expression of stmn1b was first observed at the basal layer of the wound epidermis at 1 dpa but diminished as regeneration proceeded (fig. S4, I to K). On the contrary, sema3b showed expression at later stages and was enriched in the relatively proximal portion of the basal layer epithelium (fig. S4, L to N). The expression dynamics of these two genes matched the predicted proportion changes of the two clusters (fig. S5C). While sema3b was more restricted to the basal layer, stmn1b showed low expression levels in the intermediate layer as well, potentially suggesting that this subpopulation could be labeling cells transitioning from the basal layer to the other layers of epithelium.

We next performed subcluster analysis within the hematopoietic cluster and found four subpopulations (Fig. 4, A to C and table S6). The first three populations were enriched for the macrophage marker mpeg1.1, with the cluster H1 being M1-like (lgals2+ and lcp1+) and the cluster H3 M2-like (ctsc+ and lgmn+) (Fig. 4D) (12). We speculated that the cluster H2 represented a transition state between M1-like and M2-like or a state before the macrophages differentiate toward M1-like or M2-like. From 1 to 4 dpa, the proportion of M1-like macrophages remained at a constant level, while that of M2-like macrophages expanded (Fig. 4B), potentially suggesting a shift in the function of macrophages in the new regenerates from pro-inflammatory toward anti-inflammatory as regeneration proceeded. Macrophages are important for proper blastema proliferation (28). The change in the proportions of M1/M2-like macrophage in our data matched with that observed in the larvae fin, suggesting that the adults followed a similar rule for immune cell recruitment after injury.

(A) Subcluster assignments of the hematopoietic cells. Cells were plotted on UMAP axes. The same color code is used for (B) to (D). (B) Proportion of subgroups of hematopoietic cells. (C) Expression enrichment of the top 30 differentially expressed genes in the four subclusters within hematopoietic cluster shown as a heatmap. (D) Expression distribution of genes associated with macrophage activation grouped by subclusters. Expression levels were log normalized by Seurat. y axis: cluster identity. z axis: cell density. (E) Expressions of pigment cell markers gch2 and mlpha in the hematopoietic population.

The cluster H4 enriched for genes including mlpha and gch2, both well-characterized markers for the chromatophore lineages in zebrafish (Fig. 4E) (29). Chromatophores are derived from neural crest lineage, yet here, they clustered with macrophages that were from hematopoietic lineage. One possibility is that this clustering result could be driven by features related to antigen presentation via MHC class II, a feature of pigment cells based on studies using human melanocytes (30). The proportion of this cluster decreased as regeneration started, agreeing with the known pattern of fin stripe recovery after amputation (Fig. 4B) (31).

To understand the component and function of the cells in the mesenchymal cell cluster before and during fin regeneration, we focused on genes enriched in this cluster and found previously identified blastema marker genes that are required for fin regeneration, including the muscle segment homeobox family members msx1b and msx3 and the insulin-like growth factor signaling ligand igf2b (logFC, >0.25; minimum percentage, >25%; and adjusted P value of <1 105, as listed in table S1) (2, 13). The mesenchymal cluster expressed these genes nearly exclusively, confirming their blastema identity in regenerating stages (fig. S6A). In addition, we found key genes involved in zebrafish bone development and regeneration: twist1a, the transcription factor that controls the skeletal development by regulating the expression of runx2 (14); cx43, the gap junction protein required for building the fin ray up to the right length; and hapln1a and serpinh1b, two genes downstream of cx43 (32, 33). By performing conserved marker analysis using Seurat, we found that msx1b and twist1a were also among the markers conserved across all stages, underscoring shared features that existed between regenerating and preinjury mesenchymal cells (maximum P values across stages: 4.72 1010 and 2.84 109 for msx1b and twist1a, respectively). This theme of building and supporting bone tissues in mesenchymal cells was not only reflected by a handful of genes: GO analysis of all the detected up-regulated genes in this cluster revealed significant enrichment of genes associated with GO terms, including fin regeneration (GO:0031101) and skeletal system development (GO:0001501) (fig. S6B). When more stringent criteria for differential expression were used, genes were also significantly enriched for GO terms, including skeletal system morphogenesis (GO:0048705) and extracellular matrix organization (GO:0030198) (fig. S6C).

Previous work has shown that blastema comprises bone cells and non-bone cells but has not defined the cell types and the regeneration process of each type (23, 34, 35). To better understand the regeneration process by cell type, we performed clustering analysis within the mesenchymal cluster and identified nine distinct subgroups (Fig. 5A and fig. S6D). Of the two preinjury subgroups, M-2 represented the mature bone lineage, which was enriched for expressions of bglap, mgp, and sost (fig. S6E) (36, 37). Comparing to M-2, cluster M-1 presented low expression levels of bglap, mgp, and sost and high expression levels of a group of other genes, including fhl1a, fhl2a, and tagln (fig. S6E). Mammalian orthologs of these genes are required for chondrogenesis and osteogenesis, leading us to speculate that cluster M-1 could represent the supporting non-bone cell lineage in the preinjury state (38, 39).

(A) Subclustering assignments of mesenchymal cells shown on UMAP axes. Cells are colored by their cluster assignments and connected by Slingshot-reconstructed trajectories. Lineage 1: 1-2-3-4; lineage 2: 1-2-3-5-6; lineage 3: 1-2-3-5-7-8; lineage 4: 1-2-3-5-9. (B) By-lineage highlighting of mesenchymal cells. Cells with colors other than gray represent the cells included in each corresponding lineage in (A). (C) Expression distribution of genes labeling cell lineages and cell states in mesenchymal cells. Gene feature plots were connected by estimated lineages using the same lineage color code as in (A). (D to G) In situ hybridization targeting the tnfaip6 gene in (D) preinjury, (E) 1-dpa, and [(F) and (G)] 4-dpa fin tissues. Brown dots indicate positive RNA signals from target genes, while pale blue blocks represent hematoxylin-stained cell nuclei. A zoomed-in view for the region inside the focused rectangle is provided within (D). (G) Zoomed-in view for the region highlighted by a rectangle in (F). Dotted lines indicate the amputation plane. All scale bars, 100 m.

The remaining seven populations came from regenerates. Pseudotime analysis via Slingshot (40) suggested that these subgroups formed four trajectories, all initiated from the tnfaip6+ cluster (M-3), which was composed mainly of 1-dpa cells (Fig. 5, B and C, and fig. S6D). tnfaip6 was ranked top by an adjusted P value in the differentially expressed genes labeling the regeneration initiation cluster and was also expressed exclusively in the mesenchymal cluster (Fig. 5C and fig. S6A). The mammalian ortholog of this gene is required for proliferation and proper differentiation of mesenchymal stem cells (MSCs) and balances the mineralization via osteogenesis inhibitions (41). The expression of tnfaip6 in the postinjury zebrafish fin suggested that it could also be required in the early stages of regeneration for promoting mesenchymal proliferation. To confirm the expression pattern of tnfaip6, we performed RNA in situ hybridization for uninjured and regenerating fin tissues targeting this gene (Fig. 5, D and E). In the uninjured fin, tnfaip6 was expressed in a segmental pattern, presumably enriching at joints between bone segments. At 1 dpa, tnfaip6 was expressed not only near the bony rays but also in the cavity, showing a general activation in the mesenchymal population. As regeneration proceeded from 1 to 4 dpa, mesenchymal cells divided into cdh11+ (M-4) and tph1b+ (M-5) branches, with the latter further divided into mmp13a+ (M-6), tagln+ (M-7), and vcanb+ (M-9) branches (Fig. 5C and fig. S6D). The mmp13+ (M-6) cluster maintained a high-level tnfaip6 expression, whereas all other branches had a lower but detectable tnfaip6 expression. This was consistent with the observation we made from in situ hybridization at 4 dpa targeting tnfaip6: the broad expression in the mesenchymal population and segmental enrichments similar to that in the uninjured fin (Fig. 5, F and G).

The four trajectories initiated from the tnfaip6+ cluster revealed four putative lineages representing bone and non-bone cells in the blastema. cdh11+ lineage 1 specifically expressed runx2 and osterix/sp7, which are the key transcription factors regulating osteogenesis (fig. S6E) (42). Mammalian ortholog of cdh11 could induce Sp7-dependent bone and cartilage formation in vivo, suggesting that the cdh11+ branch in the blastema represented the regenerating osteoblasts (43). Genes highly expressed at the end of this lineage (M-4) compared to the initiation point (M-3) were associated with bone mineralization and skeletal system development, further supporting their bone cell identity (table S7).

Mesenchymal cells outside the osteoblast branch shared enrichment for tph1b and aldh1a2 expressions at 2 dpa, followed by and1 expression at 4 dpa (Fig. 5C and fig. S6F). These three genes had been suggested to label joint fibroblasts, fibroblast-derived blastema cells, and actinotrichia-forming cells in the blastema, respectively (34, 35, 44). However, their expression signatures implied that instead of labeling separate populations in the blastema, they might be labeling different states of the same non-osteoblastic cells at the early stage of fin regeneration.

Upon 4 dpa, these non-osteoblastic cells diverged into three groups (Fig. 5C and fig. S6D). To understand this separation, we performed differential expression analysis for each branch between cells at the end of the lineage tree (lineage 1, M-4; lineage 2, M-6; lineage 3, M-7 and M-8; and lineage 4, M-9) and cells in the initiation cluster (M-3). Genes highly expressed at the lineage end points were included for GO analysis for functional predictions (logFC, >0.25; minimum percentage of >25%; and adjusted P value of <0.01). These three lineages were also associated with skeletal system development or extracellular matrix organizations as were the bone cell lineage; however, the association was driven by a nearly completely different set of genes (table S7). Unlike the osteoblast lineage, none of these three non-bone cell lineages showed enrichment for bone mineralization, suggesting that these cells might indirectly contribute to bone formation. In lineage 2, top differentially expressed genes mmp13a and ogn both have mammalian orthologs that are associated with bone formation (Fig. 5C and fig. S6F) (45, 46). In addition, this lineage presented up-regulation of DLX family genes, especially dlx5a, suggesting the reactivation of fin outgrowthrelated developmental programs during regeneration (fig. S6F and table S7) (47). Lineages 3 and 4 both enriched for estrogen response and expressed the retinoic acid (RA) synthesis gene aldh1a2. However, only lineage 3 displayed up-regulation of the RA-degrading enzyme cyp26b1 (fig. S6F and table S7). The cyp26b1high-aldh1a2low pattern helped to reduce RA levels in the blastema, promoting redifferentiation of the osteoblasts (44). The differentiation-promoting signature was also reflected in the enrichment of genes, including col6a1 and tagln, whose mammalian orthologs are essential for bone formation (fig. S6F and table S7) (39, 48). These genes were also enriched in the preinjury non-bone cell population, suggesting a connection between this subset of the non-bone cells and their preinjury counterparts (Fig. 5C and fig. S6F). Top up-regulated genes in lineage 4, on the other hand, were main contributors of the extracellular matrix, including and1/2, loxa, and vcanb (35, 49, 50). Enriched expression of these genes suggested that this lineage could be responsible for creating and organizing the fibrous environment. Together, the various non-osteoblastic cells could potentially work collaboratively with the osteoblasts in creating the environment for bone tissue regeneration.

Genes that had been suggested to label progenitors contributing to fin regeneration (mmp9 and cxcl12a) and several orthologs of known mammalian MSC markers (lrrc15, prrx1a/b, and pdgfra) (6, 7, 51, 52) were expressed almost exclusively in the mesenchymal cluster (fig. S6A). Consistent with the observations made in the lineage-tracing study, the mmp9 expression was associated with the regenerating bone cell lineage (lineage 1; Fig. 5B and fig. S6E) (7). However, mmp9 was detected only in a small portion of the mesenchymal cells and was highly expressed in the basal epithelium cells at similar proportions. On the other hand, we observed coenrichment of cxcl12a (previously known as sdf-1) and orthologs of the known mammalian MSC markers in the preinjury population (fig. S6E). cxcl12a-expressing cells in zebrafish were found to carry osteogenic, adipogenic, and chondrogenic characteristics in vitro like MSCs would do and contributed to the mesenchyme of the newly developing bony rays during fin regeneration (6, 53). The coenrichment pattern suggested that some of the preinjury cxcl12a-expressing cells could be MSCs in the fin tissue, which contribute to fin regeneration.

Zebrafish caudal fin is a unique regeneration system to model the injury response and regeneration of vertebrate appendages despite being a simple structure without muscular and adipose tissues. Major components of the regenerating caudal fin are epithelial cells covering the wound site and blastemal cells producing the connective tissue and bone matrices. Early studies established that actively proliferating blastema is the key to regeneration. Formed by cell migration and proliferation, this layer of cells continues in outgrowth and differentiation, rebuilding the complex body structure. Despite efforts in understanding its importance, basic questions regarding the formation of blastema remained: (i) Which type of cells contributes to the blastema and (ii) how do they shape the regeneration process?

Using single-cell transcriptomes, we defined cell types in both preinjury and postinjury fin tissues. Although regenerating cells were drastically different from their preinjury counterparts, both stage-specific and integrated clustering analysis revealed the same major cell type compositions in the fin tissues regardless of their time of collection. Common cell types detected include epithelial cells from all three layers, hematopoietic cells, and mesenchymal cells. Our data lay a foundation for lineage-targeted analysis to investigate the role of epithelial layers and subtypes in fin regeneration.

For each cell type to be a consistent component in the regenerated fin, cell cycle entry is required. We found that both common and unique cell cycle programs activated in the regenerating fin, with the shared ones appearing to be more evolutionarily conserved than the unique ones. Among the genes showing cell typespecific S phase enrichment, several immunoproteasome subunits also showed a clear cell typespecific expression. We speculated that the increasing level of immunoproteasome subunits in epithelial and hematopoietic cells specifically might accelerate antigen processing and presentation, which could be important for immune cell recruitment and tumor necrosis factorinduced blastemal proliferation (54).

Epithelial cells were the most abundant cell type in the profiled fins and could be clustered into four different subgroups, including the three layers in the adult fish epithelium and the mucosal-like cells within the intermediate layer. However, markers labeling these layers did not perform well in separating cell groups when only regenerating cells were considered. An unbiased differential expression test suggested that some members of the krt and cldn families were expressed in specific layers more consistently throughout regeneration. RNA in situ hybridization targeting cldne, krt1-19d, cldna, krt94, cldni, and cldn1 confirmed their exclusive layer-specific expression pattern, underscoring their potential to serve as markers for the distinct epithelial layers during regeneration. Our epithelium-specific analysis suggested that basal layer epithelial cells proliferate and could be the main source for replenishing the other two layers of the epithelium, similar to findings in a previous study based on genetic lineage tracing in zebrafish and echoing findings made using the axolotl limb regeneration model (25, 55). We observed higher apoptosis and lower proliferation features in the superficial epithelial layer compared to the other layers. At the same time, we observed transition patterns in gene expression, connecting the basal to the intermediate and the superficial layer during regeneration.

The behavior of mucosal-like cells during regeneration had been rarely reported for zebrafish in literature. We found in this study that this group of cells was an integral part of the regeneration process. Enrichment of foxp1b in this population and enrichment of foxp4 in basal and intermediate epithelial cells supported that zebrafish foxp homologs could be involved in regulating agr2 expression as does the Fox family in mice and, furthermore, the mucin production in the epithelium during regeneration (Fig. 1E) (56). The protein encoded by amphibian homologs of agr2, nAG (from newts) and aAG (from axolotl), are necessary and sufficient for salamander limb regeneration (57, 58). They are expressed in both dermal glands and the nerve sheaththe pattern of which has also been recovered from single-cell RNA sequencing (scRNA-seq) analysis (55). Regeneration deficiencies caused by denervation before amputation can be rescued by the ectopic expression of nAG. Although we do not have data supporting the nerve sheath expression pattern, as shown for the amphibian models, we hypothesize that agr2 could similarly mediate neuronal signals in zebrafish during regeneration.

Macrophages are critical players in the zebrafish caudal fin regeneration (28, 54). We observed subgroups of the mpeg1.1+ macrophage population in the regenerating fin tissue, resembling M1 and M2 macrophages in mammalian systems. However, we were not able to recover other immune cell population in the hematopoietic cells. This could potentially be due to the systematic bias against certain cell types during tissue dissociation and droplet incorporation in the microfluidic device. The same bias might also explain why we were not able to recover some other known players in the regenerating fin tissue, including neurons and endothelial cells (4). Increasing the number of cells sampled for scRNA-seq or performing scRNA-seq on sorted hematopoietic lineage cells would help to better understand the involvement of these populations in the regeneration process.

The expression profiles of mesenchymal cells captured from the postinjury stages resembled those of blastema in histology studies. We found four connected but distinct lineages representing both bone and non-bone cells in the blastema. All four lineages initiated from one cluster mostly consisted of 1-dpa cells and enriched for the tnfaip6 expression. A similar scenario has been observed in the axolotl limb regeneration model. By using scRNA-seq on a lineage-labeled axolotl model, Gerber et al. (58) found that connective tissue cells funnel into a progenitor state at initiation. Whether the cluster identified in our study represented a shared cell origin for the blastema or a shared state across mesenchymal cell types in the initial blastema-formation stage requires further investigation. High proportion of epithelial population in the fins could also hamper the discovery of relatively rare population with multipotency. Finer dissection before single-cell profiling might help in future study designs in capturing these populations.

While the bone cell lineage has been well studied in the regenerating fin, non-bone cells had been labeled by different markers and given different names and their intercorrelations left to be clarified. We found that tph1b, aldh1a2, and and1/and2 genes were shared among the non-bone cell lineages and could be labeling states instead of types of blastemal cells during regeneration. Meanwhile, differential analysis revealed similar enrichment for bone formation in all lineages yet distinct associations with reactivation of developmental programs, RA signaling, and collagen metabolism, underscoring their collaborative and complementary roles in the regeneration process.

Our scRNA-seq data also provided more details about the fish system we are working with. For all sample collections, we used the transgenic strain Tg(sp7:EGFP)b1212, which specifically labels osteoblast lineage in the fish (59). It was reported that green fluorescence signal could be detected in the fish skin after 72 hours post-fertilization. This ectopic expression, however, does not interfere with confocal imaging of skeletal structures of fish at any stage due to the fact that they lie in different planes of focus. What these cells are and why they expressed the transgene were unclear. In this study, we obtained a holistic view of the transgene expression pattern in the fin region regardless of whether that was associated with the cell type of interest, i.e., osteoblasts in this context. Unsupervised clustering on the expression profiles from single fin cells suggested that green fluorescent protein (GFP) is not only expressed in the mesenchymal but also highly enriched in the superficial layer epithelium (table S2). A closer examination of this classic reporter gene construct revealed that the regulatory region of sp7 used for the construction of the transgene did not exactly represent the endogenous sp7 regulatory region. Tg(sp7:EGFP)b1212 was generated from bacterial artificial chromosome transgenesis using CH73-243G6 as the backbone, which did not contain the first exon of sp7 according to the annotation of the current genome assembly (chr6:58630884-58720045 and GRCz10), leading to the usage of a regulatory sequence different from the endogenous version. Whether this usage difference contributed to the ectopic expression pattern of the transgene requires further study. This finding points to the potential of using single-cellbased approaches in reporter line validation and more thorough analysis of the transgene behavior.

All zebrafish were used in accordance with protocol no. 20190041 approved by the Washington University Institutional Animal Care and Use Committee. Wild-type and Tg(sp7:EGFP) strains are maintained under standard husbandry in the Washington University Fish Facility, with the system water temperature at 28.5C and a day-night cycle controlled as 14-hour light/10-hour dark. For fin amputation, we anesthetized 1-year-old fish with MS-222 (0.16 g/liter) in the system water and then removed the distal half of their caudal fin with sterilized razor blades. The fish were then sent back to circulating water system for recovery. We collected regenerating fin tissue from 39 fish by doing secondary fin amputation at the primary cutting plane with the same anesthesia and recovery procedures.

Collected fin tissues were digested by Accumax (Innovative Cell Technologies), filtered through 40-m cell strainers, and washed with 1 Dulbeccos phosphate-buffered saline (DPBS)0.04% bovine serum albumin to generate single-cell suspensions. Libraries were constructed from these cell suspensions following the instruction of the Chromium Single Cell Gene Expression Solution 3 v2 (10x Genomics) and were subsequently sequenced on HiSeq2500 (Illumina) with read lengths of 26 + 75 (Read1 + Read2). Raw reads were processed by Cell Ranger (10x Genomics) with default parameters for read tagging, alignment to zebrafish reference genome (GRCz10), and feature counting based on Ensembl release 91 (cellranger count). EGFP sequence was added into the reference genome as a separate chromosome for mapping reads from the reporter gene.

We performed unsupervised clustering using Seurat v3.0 following the procedure of normalization (SCTransform), highly variable gene detection, dimensional reduction (principal components analysis), and cells clustering (Louvain clustering at resolutions from 0.1 to 0.6) (17). For integrating the four stages in finding conserved cell types, we used the anchoring approach provided by Seurat v3. Cell clustering was based on the top principal components that account for most of the cell-cell variances. The same set of principal components was used in UMAP calculation for visualization as well.

We found differentially expressed genes in each cluster by comparing the expression profiles of them with those of the rest of the cells using Wilcoxon rank sumbased approach with the criteria of log fold change more than 0.25 and a minimum cell percentage of 0.25. The same criteria were applied to all pairwise comparisons, unless stated otherwise. We made functional connections between the list of differentially expressed genes and the type of cell that they most likely represent by testing for GO term enrichment (18) and manual curation by searching The Zebrafish Information Network database and PubMed. Certain cell clusters were taken as independent samples for secondary clustering following the same unsupervised clustering procedures.

We calculated the by-cell average expression level of a set of S phase or G2-M phase markers suggested by Seurat that are detected in our zebrafish dataset and normalized by subtracting aggregated expression of control genes. Although G1 phase cells are also within cell cycle, they are hardly separable from G0 cells. To avoid false-positive labeling for active cycling cells, we set stringent thresholds and only included cells with |S.score G2M.score| > 0.1 in the S or G2-M group, while cells with both S.score and G2M.score below zero as G1. Other cells were not included in this part of the analysis. Differentially expressed genes were also identified by Wilcoxon rank sumbased approach. These differentially expressed genes were considered to be cell cycle related if they were in the list of genes associated with R-DRE-1640170 Cell Cycle and/or cycling marker genes used for cell cycle phase score calculations.

We collected uninjured and regenerating fin tissues from casper (nacrew2/w2;roya9/a9) fish and fixed in 4% paraformaldehyde overnight (60). Fixed tissues were subsequently submerged in 10% sucrose in 1 PBS, 20% sucrose in 1 PBS, and 30% sucrose in 1 PBS for 4 hours each. After sucrose exchange, tissues were embedded in Optimal Cutting Temperature (O.C.T.) compound (Fisher Healthcare Tissue-Plus) and snap frozen on dry ice. The frozen tissue blocks were then processed into 15-m sections on a Leica CM1950 cryostat. We performed RNA in situ hybridization targeting krt4, cldne, krt1-19d, cldna, krt94, cldni, cldn1, agr2, sema3b, stmn1b, and tnfaip6 for mRNA detection using an RNAscope kit (Advanced Cell Diagnostics, Hayward, CA, USA). Alcian blue/periodic acidSchiff (PAS) staining was subsequently performed on the same section or separately on a consecutive serial section following the manufacturers protocol (Newcomer Supply). Microscopic images were taken by ZEISS Axio Observer.

Cell trajectories were constructed using Slingshot v1.3.1 (40). Through initial subclustering and cell type identifications, we found one subcluster with high epcam expression, potentially a doublet cell contamination from the major cell type classifications. We removed this group of cells from all downstream analysis within the mesenchymal cluster. We used UMAP embedding and subclustering assignments as input for the Slingshot calculation.

We performed nonparametric Wilcoxon rank sum test to identify differentially expressed genes across cell groups as implemented in Seurat. P values were adjusted by all features in the dataset using Bonferroni correction.

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Cellular diversity of the regenerating caudal fin - Science Advances

Organ Transplant Rejection Medications Market: Introduction

According to the report, the globalorgan transplant rejection medications marketwas valued atUS$ 4.7 Bn in 2018and is projected to expand at a CAGR of~3% from 2019 to 2027. Rise in healthcare spending and improvements in healthcare infrastructure, and increase in the global geriatric population are the major factors anticipated to drive the organ transplant rejection Medication market from2019 to 2027.

Rise in Healthcare Spending and Improvement in Healthcare Infrastructure to Drive Global Market

Increase in patient awareness about personal health boosts the demand for medical devices. Patients are more aware and proactive about their health and are willing to seek a physician's advice at an early stage. Increase in per capita disposable income is encouraging people to spend more on healthcare facilities, which, in turn, fuels the global organ transplant rejection medications market. Advertisements have increased public visibility of new technology, thereby generating interest among chronic patients for organ transplant rejection medications.

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Changing demographics in emerging economies such as China, India, Brazil, and South Africa are expected to provide significant opportunities for organ transplant rejection medications. Moreover, public and private healthcare expenditure is expected to increase in these countries, which is likely to drive the organ transplant rejection medications market.

Increase in healthcare expenditure, rise in global per capita income, and improvement in healthcare infrastructure and government reimbursement programs in developed as well as developing countries are likely to propel the organ transplant rejection medications market in the near future.

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Kidney Transplant to Offer Lucrative Opportunities

The report offers detailed segmentation of the global organ transplant rejection medications market based on drug class, transplant type, distribution channel, and region. In terms of drug class, the organ transplant rejection medications market has been segmented into calcineurin inhibitors, antiproliferative agents, mTOR inhibitors, antibodies, and steroids. Based on transplant type, the market has been segmented into kidney transplant, bone marrow transplant, liver transplant, heart transplant, lung transplant, and other transplants. Kidney transplant is a highly preferred treatment for end-stage renal disease (ESRD). It is comparatively more cost-effective than dialysis and is associated with a long-term mortality improvement.

Based on distribution channel, the global organ transplant rejection medications market has been classified into hospital pharmacies, retail pharmacies, and online pharmacies. Hospitals are major clinical settings wherein a large number of surgeries are conducted. This makes hospital pharmacies a prominent segment of the organ transplant rejection medications market.

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North America to Lead Organ Transplant Rejection Medications Market

North America was the largest market for organ transplant rejection medications in 2018, due to presence of the maximum number of living as well as deceased donors and better organ-preserving practices in the region. However, high costs and complex procedures would adversely affect the organ transplant rejection medications market during the forecast period. The organ transplant rejection medications market in Asia Pacific is projected to expand at a relatively high CAGR of4.1%during the forecast period. Transplantation procedures vary substantially from region to region and country to country, due to factors such as difference in the rate of end-organ damage, economic differences in terms of ability to provide transplants or other treatments, cultural differences that can support or hinder organ donation and transplant, and reporting of legal transplants across regions.

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Major Market Players

The report provides profiles of leading players operating in the global organ transplant rejection medications market. These includeGlaxoSmithKline plc, Novartis AG, F. Hoffmann-La Roche Ltd., Astellas Pharma, Inc., Pfizer, Inc., AbbVie, Inc., Allergan plc, Bristol-Myers Squibb Company (BMS), and Sanofi.

Novartis AG is a leading company that specializes in the development and manufacture of branded as well as generic pharmaceutical and biopharmaceutical drugs. It is evaluating the experimental Facilitating Cell Therapy (FCR001), which involves taking stem cells of a kidney donor and grafting them in the transplant recipients bone marrow. This combination would trick the recipients immune system by accepting the donated kidney as its own.

Pfizer, Inc. is a global pharmaceutical company that develops, manufactures, and markets prescription medicines in 11 therapeutic segments, including cardiovascular, oncology, neuroscience, pain, and infectious diseases. The company offers a range of medicines and vaccines as well as consumer healthcare products for the prevention and treatment of infectious and chronic diseases for all age groups.

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Organ Transplant Rejection Medications Market: Drug Companies Focus on Improving Long-term Outcome of New Drugs - BioSpace

The latest report published by Regal Intelligence on Hematopoietic Stem Cell Transplantation (HSCT) market provides crucial market insights along with detailed segmentation analysis. The report examines key driving factors that are expected to drive the growth of the market.

Global Hematopoietic Stem Cell Transplantation (HSCT) Market Research Report gives knowledgeable information on various market situations, for example, potential development factors, factors controlling the development, market opportunities and dangers to the worldwide market. Also, the report broadly centers around competitive analysis of Hematopoietic Stem Cell Transplantation (HSCT) Market. The competitive analysis segment incorporates key manufacturers, fresh players, providers, market strategies, potential chances, operation landscape and analysis of the trends of the Hematopoietic Stem Cell Transplantation (HSCT) market. The market results are centered around current market scenario. To gauge and predict the degree of competition in this market. This report will likewise support all the manufacturers and speculators to have a superior comprehension of the investments to know where the market is heading.

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Leading players of Hematopoietic Stem Cell Transplantation (HSCT) including:Regen Biopharma Inc, China Cord Blood Corp, CBR Systems Inc, Escape Therapeutics Inc, Cryo-Save AG, Lonza Group Ltd, Pluristem Therapeutics Inc, ViaCord Inc

For Product type segment the report listed main product type:

AllogeneicAutologous

For Application segment the report listed main types:

Peripheral Blood Stem Cells Transplant (PBSCT)Bone Marrow Transplant (BMT)Cord Blood Transplant (CBT)

Key Highlights of the Hematopoietic Stem Cell Transplantation (HSCT) Market

Key Strategies adopted by major players Global driving factors of the market Developed and emerging markets Comprehensive description of the international players Market dynamic factors affecting the global market Evaluation of niche business areas Driving and restraining factors of the market growth Market share analysis

Moreover, the report briefly studies the performance of both historical records along with the recent trends. It includes a complete analysis of different attributes such as manufacturing base, type, and size. This report evaluates the market segmentation along with the competitive landscape at global as well as regional level. The report also discusses about the rising need for Hematopoietic Stem Cell Transplantation (HSCT) market.

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Report on Global Hematopoietic Stem Cell Transplantation (HSCT) Market 2020 comprises of 10 Sections in Table as follows:

The Hematopoietic Stem Cell Transplantation (HSCT)-market report reads pin-direct analysis for changing serious dynamics with reference towards changing elements that drives or limits market development. The report is comprehensively visualized to forecast the market point of view and opportunities where it has an extension to develop in future. Basically, the report segregates the ability of market in the present and the future possibilities from various edges in detail.

About Us:We, Regal Intelligence, aim to change the dynamics of market research backed by quality data. Our analysts validate data with exclusive qualitative and analytics driven intelligence. We meticulously plan our research process and execute in order to explore the potential market for getting insightful details. Our prime focus is to provide reliable data based on public surveys using data analytics techniques. If you have come here, you might be interested in highly reliable data driven market insights for your product/service,reach us here 24/7.

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Hematopoietic Stem Cell Transplantation (HSCT) Market 2020: Regen Biopharma Inc, China Cord Blood Corp, CBR Systems Inc, Escape Therapeutics Inc,...

Detailed Analysis & SWOT analysis, Stem Cell Therapy Market Trends 2020, Stem Cell Therapy Market Growth 2020, Stem Cell Therapy Industry Share 2020, Stem Cell Therapy Industry Size, Stem Cell Therapy Market Research, Stem Cell Therapy Market Analysis, Stem Cell Therapy market Report speaks about the manufacturing process. The process is analyzed thoroughly with respect three points, viz. raw material and equipment suppliers, various manufacturing associated costs (material cost, labor cost, etc.) and the actual process of whole Enterprise Stem Cell Therapy Market.

Stem Cell Therapy market 2020 is a professional and in-intensity look at on the modern state of the key-word industry. The document provides a simple review of the key-word marketplace together with definitions, classifications, programs and chain shape. The key-word enterprise evaluation is supplied for the worldwide marketplace which include improvement records, competitive landscape evaluation, and principal local development popularity.

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The Stem Cell Therapy marketplace file elaborates Stem Cell Therapy industry evaluation with various definitions and category, Product kinds & its packages and chain shape. Stem Cell Therapy market document presentations the manufacturing, sales, charge, and market proportion and boom rate of every type as following.

2020 Short Detail of this Stem Cell Therapy market report:

Stem-cell therapy is the use of stem cells to treat or prevent a disease or condition. Bone marrow transplant is the most widely used stem-cell therapy, but some therapies derived from umbilical cord blood are also in use.

In the last several years, global stem cell therapy market developed fast at a average growth rate of 46.81%.

Market Analysis and Insights: Global Stem Cell Therapy Market

In 2019, the global Stem Cell Therapy market size was USD 403.6 million and it is expected to reach USD 1439.9 million by the end of 2026, with a CAGR of 19.7% during 2021-2026.

Global Stem Cell Therapy Scope and Market Size

Stem Cell Therapy market is segmented by Type, and by Application. Players, stakeholders, and other participants in the global Stem Cell Therapy market will be able to gain the upper hand as they use the report as a powerful resource. The segmental analysis focuses on revenue and forecast by Type and by Application in terms of revenue and forecast for the period 2015-2026.

Segment by Type, the Stem Cell Therapy market is segmented into Autologous, Allogeneic, etc.

Segment by Application, the Stem Cell Therapy market is segmented into Musculoskeletal Disorder, Wounds & Injuries, Cornea, Cardiovascular Diseases, Others, etc.

Regional and Country-level Analysis

The Stem Cell Therapy market is analysed and market size information is provided by regions (countries).

The key regions covered in the Stem Cell Therapy market report are North America, Europe, China, Japan, Southeast Asia, India and Central & South America, etc.

The report includes country-wise and region-wise market size for the period 2015-2026. It also includes market size and forecast by Type, and by Application segment in terms of revenue for the period 2015-2026.

Competitive Landscape and Stem Cell Therapy Market Share Analysis

Stem Cell Therapy market competitive landscape provides details and data information by vendors. The report offers comprehensive analysis and accurate statistics on revenue by the player for the period 2015-2020. It also offers detailed analysis supported by reliable statistics on revenue (global and regional level) by player for the period 2015-2020. Details included are company description, major business, company total revenue and the revenue generated in Stem Cell Therapy business, the date to enter into the Stem Cell Therapy market, Stem Cell Therapy product introduction, recent developments, etc.

The major vendors include Osiris Therapeutics, NuVasive, Chiesi Pharmaceuticals, JCR Pharmaceutical, Pharmicell, Medi-post, Anterogen, Molmed, Takeda (TiGenix), etc.

This report focuses on the global Stem Cell Therapy status, future forecast, growth opportunity, key market and key players. The study objectives are to present the Stem Cell Therapy development in North America, Europe, China, Japan, Southeast Asia, India and Central & South America.

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Stem Cell Therapy Market by Product Type:

Stem Cell Therapy Market by Applications:

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Next part of the Stem Cell Therapy Market analysis report speaks about the manufacturing process. The process is analysed thoroughly with respect three points, viz. raw material and equipment suppliers, various manufacturing associated costs (material cost, labour cost, etc.) and the actual process. Stem Cell Therapy market competition by top manufacturers, with production, price, and revenue (value) and market share for each manufacturer as per following;

Top Manufacturer Included in Stem Cell Therapy Market:

And More

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After the basic information, the Stem Cell Therapy report sheds light on the production, production plants, their capacities, global production and revenue are studied. Also, the Stem Cell Therapy Market growth in various regions and R&D status are also covered.

Stem Cell Therapy Market Report by Key Region:

The global Stem Cell Therapy market is anticipated to rise at a considerable rate during the forecast period, between 2020 and 2026. In 2020, the market was growing at a mild rate and with the rising adoption of strategies by key players, the market is predicted to rise over the projected horizon. The report also tracks the most recent market dynamics, like driving factors, restraining factors, and industry news like mergers, acquisitions, and investments.

The report can help to know the market and strategize for business expansion accordingly. Within the strategy analysis, it gives insights from market positioning and marketing channel to potential growth strategies, providing in-depth analysis for brand fresh entrants or exists competitors within the Stem Cell Therapy industry. Global Stem Cell Therapy Market Report 2020 provides exclusive statistics, data, information, trends and competitive landscape details during this niche sector.

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Further in the report, Stem Cell Therapy Market is examined for price, cost and gross revenue. These three points are analysed for types, companies and regions. In prolongation with this data sale price for various types, applications and region is also included. The Stem Cell Therapy Industry consumption for major regions is given. Additionally, type wise and application wise consumption figures are also given.

To provide information on competitive landscape, this report includes detailed profiles of Stem Cell Therapy Market key players. For each player, product details, capacity, price, cost, gross and revenue numbers are given. Their contact information is provided for better understanding.

Other Major Topics Covered in Stem Cell Therapy market research report are as follows:

And another component .

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Q: What's the connection between the novel coronavirus and the thymus gland? A friend of ours who is a doctor says it's probably what keeps young kids from getting so sick. I've never even heard of the thymus. What does it have to do with coronavirus?

A: From the earliest days of the novel coronavirus pandemic, the data revealed a puzzling disparity. Older adults were at increased risk of grave illness when infected with the virus, but children seemed to have a certain level of protection. And while it has since become clear that children can indeed become seriously ill if they become infected, they do so at far lower rates than adults. The reasons for this are still being investigated, but some researchers have recently suggested the role of the thymus gland as a possible factor.

If you place your finger at the notch at the top of your breast bone and draw a vertical line downward a few inches, you've traced the location of your thymus. It's made up of two roughly triangular lobes, which sit behind the breastbone and between the lungs. The thymus has several functions, but perhaps its most important role is to help produce the cells that will become T-lymphocytes, or T-cells. (The "T" stands for thymus-derived.) These are white blood cells that protect the body from bacteria, fungi, viruses and other pathogens.

T-cells, which are the ninjas of the immune system, start out in the bone marrow as stem cells. The immature stem cells exit the marrow, move through the blood and enter a specific region of the thymus. There, they undergo a complex process that teaches them how to recognize a wide range of potentially dangerous and deadly invaders. As T-cells, their job is to circulate throughout the body and, when they encounter the molecular signature of the pathogen they've been trained to recognize, to attack. T-cells also activate other immune cells, produce proteins known as cytokines and have a role in regulating immune response.

The thymus is unique in that it reaches maturity in utero and is at its largest and most active in children. Starting at puberty, it gradually becomes less active, and the glandular tissue begins to shrink. This continues throughout a person's life. By the time someone has reached their mid-60s, the thymus is largely inactive. By their mid-70s, the gland has been mostly replaced with fat. This decrease in thymus function is believed to be one of the reasons that, in their later years, older adults become more susceptible to disease and infection.

Emerging research into COVID-19 has shown a marked decrease in the number of T-cells in some gravely ill patients. Scientists are now asking whether age-related thymus decline, which means T-cells aren't quickly replaced, may play a role in the severity of illness seen in older adults. The flip side of this is whether, due to their robust production of T-cells, children's immune systems are able to stay one step ahead of the novel coronavirus. It's only a working theory, but it shows promise, and research into how this may affect and inform treatment continues.

Dr. Eve Glazier is an internist and associate professor of medicine at UCLA Health. Dr. Elizabeth Ko is an internist and assistant professor of medicine at UCLA Health. Send your questions to askthedoctors@mednet.ucla.edu.

More:
Theory suggests thymus plays role in severity of COVID - Chicago Daily Herald

You could be Jadens cure

Jaden lives in Hilo, just turned 18 and has been diagnosed with a blood cancer. He is in dire need of a bone marrow or blood stem transplant. You could be Jadens cure.

If you are between the ages of 18-44 you can register online and add your name to the Hawaii marrow donor registry. A sample kit is mailed to you, you touch one swab in your mouth and send it back. Your tissue type is registered for worldwide use until you reach age 61.

I registered in Flint, Michigan, in 2006. I got the call in 2010 here in Kona at age 60. My donation of stems cells was done at St. Francis in Honolulu. It was totally painless and easy and the stem cells were sent to the mainland where the patient was cured of caner. The BeTheMatchHawaii health care professionals at St. Francis are very good, very friendly and like family to all the donors and patients.

Every three minutes there is a new blood or bone cancer diagnosis on our planet. Every one should go to http://www.BeTheMatchHawaii.org, where all of your questions can be answered and most importantly you can get tested and registered. You have the opportunity to change the world and save a life. How could anybody refuse to offer the gift of life when you learn that you are a match?

Since 1990, Hawaii citizens have donated to 468 cancer patients worldwide: The U.S., Brazil, Korea, Japan, Italy, Poland, actually 28 countries so far, including eight patients in Hawaii.

Please check it out, sign up and spread the word to all of your family, friends, coworkers, classmates, etc. Jaden is counting on us and you may never be match for any cancer patient, but if you are a match you can literally give someone the gift of life. How cool is that?

Larry Boucher

Kailua-Kona

Letters policy

Letters to the editor should be 300 words or less and will be edited for style and grammar. Longer viewpoint guest columns may not exceed 800 words. Submit online at http://www.westhawaiitoday.com/?p=118321, via email to letters@westhawaiitoday.com or address them to:

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Letters to the Editor: Aug. 3, 2020 - West Hawaii Today

DARZALEX SC reduces administration time from hours to minutes and demonstrates consistent efficacy with a reduction in administration-related reactions compared to intravenous DARZALEX (daratumumab)

TORONTO, Aug. 4, 2020 /CNW/ - The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that Health Canada has approved DARZALEX SC (daratumumab), a new subcutaneous formulation of daratumumab.1 DARZALEX SC is approved in four regimens across five indications in patients with multiple myeloma, most notably newly diagnosed, transplant-ineligible patients as well as relapsed or refractory patients. As a fixed-dose formulation, DARZALEX SC can be administered over approximately three to five minutes, significantly less time than intravenous (IV) DARZALEX, which is administered over hours.2 DARZALEX SC is the only subcutaneous CD38-directed antibody approved in the treatment of multiple myeloma.

In the Phase 3 COLUMBA study published in The Lancet, DARZALEX SC demonstrated a consistent overall response rate (ORR) and pharmacokinetics and a similar safety profile compared with IV DARZALEXin patients with relapsed or refractory multiple myeloma. In addition, there was a nearly two-thirds reduction in systemic administration-related reactions (ARRs) for DARZALEX SC compared to IV DARZALEX(13 per cent vs. 34 per cent, respectively).3

"DARZALEX has become a backbone therapy in the treatment of multiple myeloma, supported by a robust body of evidence in both the frontline and relapsed and refractory settings," says Dr. Darrell White, Hematologist, Queen Elizabeth II Health Sciences Centre, Halifax. "With this new subcutaneous formulation, not only is treatment much more convenient for patients, but it will also play a very important role in reducing wait times and the burden on our busy healthcare system, especially during this time."

The approval is based on data from the Phase 3 COLUMBA and Phase 2 PLEIADES studies.4,5In the COLUMBA study, the ORR was non-inferior for patients taking DARZALEX SC as monotherapy compared to those taking IV DARZALEXas monotherapy (41 per cent vs. 37 per cent, respectively).6 Additionally, in the Phase 2 PLEIADES study evaluating the efficacy and safety of DARZALEXSC in combination therapies, objective responses were demonstrated in combination with bortezomib, melphalan and prednisone (D-VMP) in newly diagnosed transplant ineligible patients. In addition, objective responses were demonstrated in combination with lenalidomide and dexamethasone (D-Rd) in relapsed or refractory patients who received one prior line of therapy.7In a pooled safety population of 490 patients who received DARZALEXSC as monotherapy or in combination, the ARR rate was 11 per cent.8

DARZALEX SC is approved in all current IV indicationsincluding (1) in combination with bortezomib, melphalan and prednisone in newly diagnosed patients who are ineligible for autologous stem cell transplant, (2) in combination with lenalidomide and dexamethasone in newly diagnosed patients who are ineligible for autologous stem cell transplant and in patients with relapsed or refractory multiple myeloma who have received at least one prior therapy, (3) in combination with bortezomib and dexamethasone in patients who have received at least one prior therapy, and (4) as monotherapy, in patients who have received at least three prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory agent or who are double-refractory to a PI and an immunomodulatory agent.9

Active discussions are ongoing with public insurers to determine how DARZALEX SC can be made accessible for both relapsed or refractory patients as well as newly diagnosed, transplant ineligible patients.

"This approval exemplifies Janssen's mission and commitment to bringing together passion, science and ingenuity to advance novel solutions for patients," said Mathai Mammen, M.D., Ph.D., Global Head, Janssen Research & Development, LLC.

About the COLUMBA Study The randomised, open-label, multicenter Phase 3 COLUMBA study included 522 patients (median age of 67 years) with multiple myeloma who had received at least three prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory drug (IMiD), or whose disease was refractory to both a PI and an ImiD. In the arm that received DARZALEX SC (n=263), patients received a fixed dose of DARZALEX SC 1,800 milligrams (mg), co-formulated with recombinant human hyaluronidase PH20 (rHuPH20) 2,000 Units per milliliter (U/mL), subcutaneously weekly for Cycles 1 2, every two weeks for Cycles 3 6 and every four weeks for Cycle 7 and thereafter. In the IV DARZALEXarm (n=259), patients received DARZALEXfor IV infusion 16 milligrams per kilogram (mg/kg) weekly for Cycles 1 2, every two weeks for Cycles 3 6 and every four weeks for Cycle 7 and thereafter. Each cycle was 28 days. In the arm that received DARZALEX SC, it was given in a fixed volume of 15 mL over three to five minutes; the median injection time was five minutes. In the arm that received the IV administration, the median durations of the first, second and subsequent IV DARZALEXinfusions were 7.0, 4.3 and 3.4 hours, respectively. Patients in both arms continued treatment until disease progression or unacceptable toxicity.10,11

About the PLEIADES Study The non-randomised, open-label, parallel assignment Phase 2 PLEIADES study included adults with multiple myeloma, including 67 patients with newly diagnosed multiple myeloma who were treated with 1,800 mg of DARZALEX SC in combination with bortezomib, melphalan, and prednisone (D-VMP) and 65 patients with relapsed or refractory disease who were treated with 1,800 mg of DARZALEX SC plus lenalidomide and dexamethasone (D-Rd). The primary endpoint for the D-VMP and D- Rd cohorts was overall response rate.12

About DARZALEXand DARZALEX SCDARZALEX is the first CD38-directed monoclonal antibody (mAb) approved to treat multiple myeloma and in 2020, DARZALEX SC (daratumumab) follows as the only subcutaneous CD38-directed antibody approved to treat patients with multiple myeloma.13It binds to CD38,a surface protein highly expressed across multiple myeloma cells.14 DARZALEX induces tumor cell death through cell lysis via multiple immune-mediated mechanisms of action, including complement-dependent cytotoxicity (CDC), antibody-dependent cellular cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP).15DARZALEX has also demonstrated immunomodulatory effects such as increasing CD4+ and CD8+ T-cells counts, which may contribute to clinical response.16

In August 2012, Janssen Biotech, Inc. and Genmab A/S entered a worldwide agreement, which granted Janssen an exclusive license to develop, manufacture and commercialize DARZALEX. Janssen Inc. commercializes DARZALEX and DARZALEX SC in Canada. For full Prescribing Information and more information about DARZALEX and DARZALEX SC, please visit http://www.janssen.com/canada.

About Multiple MyelomaMultiple myeloma is an incurable blood cancer that affects a type of white blood cell called plasma cells, which are found in the bone marrow.17 When damaged, these plasma cells rapidly spread and replace normal cells with tumors in the bone marrow. In 2020, it is estimated that 3,400 Canadians will be diagnosed with multiple myeloma and there will be 1,600 deaths associated with the disease.18 While some patients with multiple myeloma have no symptoms in the early stages, patients are diagnosed due to symptoms that can include bone disease or pain, anemia, calcium elevation, and kidney problems.19

About the Janssen Pharmaceutical Companies of Johnson & Johnson At Janssen, we're creating a future where disease is a thing of the past. We're the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular & Metabolism, Immunology, Infectious Diseases & Vaccines, Neuroscience, Oncology, and Pulmonary Hypertension.

Learn more at http://www.janssen.com/canada. Follow us at @JanssenCanada. Janssen Inc. is a member of the Janssen Pharmaceutical Companies of Johnson & Johnson.

*All trademark rights used under license. **Dr. White was not compensated for any media work. He has been compensated as a consultant.

Cautions Concerning Forward-Looking StatementsThis press release contains "forward-looking statements" as defined in the Private Securities Litigation Reform Act of 1995 regarding DARZALEX SC. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Janssen Inc., any of the other Janssen Pharmaceutical Companies and/or Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behavior and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson's Annual Report on Form 10-K for the fiscal year ended December 29, 2019, including in the sections captioned "Cautionary Note Regarding Forward-Looking Statements" and "Item 1A. Risk Factors," and in the company's most recently filed Quarterly Report on Form 10-Q, and the company's subsequent filings with the Securities and Exchange Commission. Copies of these filings are available online at http://www.sec.gov, http://www.jnj.com or on request from Johnson & Johnson. None of the Janssen Pharmaceutical Companies nor Johnson & Johnson undertakes to update any forward-looking statement as a result of new information or future events or developments.

References:

1

[DARZALEX SC Product Monograph, Janssen Inc., July 29, 2020]

2

[DARZALEX SC Product Monograph, Janssen Inc., July 29, 2020]

3

Mateos MV, et al. Subcutaneous versus intravenous daratumumab in patients with relapsed or refractory multiple myeloma (COLUMBA): a multicentre, open-label, non-inferiority, randomised, phase 3 trial [published online ahead of print March 23, 2020]. Lancet Haematol doi.org/10.1016/S2352-3026(20)30070-3.

4

Mateos M-V et al. Efficacy and Safety of the Randomized, Open-Label, Non-inferiority, Phase 3 Study of Subcutaneous (SC) Versus Intravenous (IV) Daratumumab (DARA) Administration in Patients (pts) With Relapsed or Refractory Multiple Myeloma (RRMM): COLUMBA. 2019 American Society of Clinical Oncology Annual Meeting. June 2019.

5

Janssen Research & Development, LLC. A Study to Evaluate Subcutaneous Daratumumab in Combination With Standard Multiple Myeloma Treatment Regimens. In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000 [cited July 5, 2019]. Available at: https://clinicaltrials.gov/ct2/show/NCT03412565. Identifier: NCT03412565.

6

Mateos MV, et al. Subcutaneous versus intravenous daratumumab in patients with relapsed or refractory multiple myeloma (COLUMBA): a multicentre, open-label, non-inferiority, randomised, phase 3 trial [published online ahead of print March 23, 2020]. Lancet Haematol doi.org/10.1016/S2352-3026(20)30070-3.

7

Chari A, M. J., McCarthy H, et al Subcutaneous daratumumab plus standard treatment regimens in patients with multiple myeloma across lines of therapy: PLEIADES study update. Poster presented at: 61st American Society of Hematology (ASH) Annual Meeting. Orlando, FL.

8

[DARZALEX SC Product Monograph, Janssen Inc., July 29, 2020]

9

[DARZALEX SC Product Monograph, Janssen Inc.,July 29, 2020]

10

[DARZALEX SC Product Monograph, Janssen Inc., July 29, 2020]

11

Mateos MV, et al. Subcutaneous versus intravenous daratumumab in patients with relapsed or refractory multiple myeloma (COLUMBA): a multicentre, open-label, non-inferiority, randomised, phase 3 trial [published online ahead of print March 23, 2020]. Lancet Haematol doi.org/10.1016/S2352-3026(20)30070-3.

12

Janssen Research & Development, LLC. A Study to Evaluate Subcutaneous Daratumumab in Combination With Standard Multiple Myeloma Treatment Regimens. In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000 [cited July 5, 2019]. Available at: https://clinicaltrials.gov/ct2/show/NCT03412565. Identifier: NCT03412565.

13

Janssen Research & Development, LLC. A Study to Evaluate Subcutaneous Daratumumab in Combination With Standard Multiple Myeloma Treatment Regimens. In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000 [cited July 5, 2019]. Available at: https://clinicaltrials.gov/ct2/show/NCT03412565. Identifier: NCT03412565.

14

[DARZALEX SC Product Monograph, Janssen Inc., July 29, 2020]

15

[DARZALEX SC Product Monograph, Janssen Inc., July 29, 2020]

16

[DARZALEX SC Product Monograph, Janssen Inc., July 29, 2020]

17

Kumar, SK et al. Risk of progression and survival in multiple myeloma relapsing after therapywith IMiDs and bortezomib: a multicenter international myeloma working group study. Leukemia. 2012 Jan; 26(1):149-57.

18

Canadian Cancer Society. "Signs and Symptoms of Multiple Myeloma." Available at: https://www.cancer.ca/en/cancer-information/cancer-type/multiple-myeloma/statistics/?region=on.Accessed June 2020.

19

Canadian Cancer Society. "Signs and Symptoms of Multiple Myeloma." Available at: http://www.cancer.ca/en/cancer-information/cancer-type/multiple-myeloma/signs-and-symptoms/?region=on.Accessed June 2020.

SOURCE Janssen Inc.

For further information: Media Contact: Janssen Inc., Jennifer McCormack, Office: (416) 382-5121; Investor Contact: Jennifer McIntyre, Office: (732) 524-3922

http://www.janssen.ca/

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Janssen Announces Health Canada Approval of DARZALEX* SC, a New Subcutaneous Formulation for the Treatment of Patients with Multiple Myeloma - Canada...

Adrenoleukodystrophy (ALD) is a rare genetic condition that causes the buildup of very-long-chain fatty acids (VLCFAs) in the brain. The defective gene in ALD, commonly referred to as a genetic mutation, can cause several different but related conditions: adrenomyelopathy (AMN), Addisons disease, and the most common and most devastating form cerebral ALD. Cerebral ALD strikes boys between ages 4 and 10, leading to permanent disability and death usually within four to eight years.

Signs and symptoms of the adrenomyeloneuropathy type appear between early adulthood and middle age. Affected individuals develop progressive stiffness and weakness in their legs (paraparesis), experience urinary and genital tract disorders, and often show changes in behavior and thinking ability. Most people with the adrenomyeloneuropathy type also have adrenocortical insufficiency. In some severely affected individuals, damage to the brain and nervous system can lead to an early death.

For detailed analysis on the symptoms of Global Adrenoleukodystrophy Pipeline Analysis browse through https://univdatos.com/report/global-adrenoleukodystrophy-pipeline-analysis-2019

The diagnosis of the disease includes genetic counseling, differential diagnosis, biochemical and molecular diagnosis. Genetic counseling must be offered to the parents of affected boys, adult males, and women with X-ALD and their family to detect: carriers who can be offered prenatal diagnosis, and asymptomatic or pre-symptomatic men or women who can benefit from therapeutic interventions. Regular follow-up in presymptomatic males can prevent serious morbidity and mortality.

Despite significant mortality risk, allogeneic HCT remains the only therapeutic intervention that can arrest the progression of cerebral demyelination in X-ALD, provided the procedure is performed very early, i.e., when affected boys or men have no or minor symptoms due to cerebral demyelinating disease. In the future, transplantation of autologous hematopoietic stem cells that have been genetically corrected with a lentiviral vector before re-infusion might become an alternative to autologous HCT, once the very encouraging results obtained in the first two treated patients would have been extended to a larger number of patients with cerebral X-ALD.

For detailed analysis on the management in Global Adrenoleukodystrophy Pipeline Analysis browse through https://univdatos.com/report/global-adrenoleukodystrophy-pipeline-analysis-2019

There are currently only two available treatments for childhood cerebral ALD: Lorenzos oil and stem cell transplantation, using either umbilical cord stem cells or bone marrow stem cells. Both treatment approaches have shown promise and been effective for some boys with ALD, but they also both have drawbacks. The therapeutic pipeline of Adrenoleukodystrophy consists of approximately 9+ products in different stages of development. Currently, 3+ drugs are in Phase III development and major drugs are in the late stage.

Request for Sample of the report browse through https://univdatos.com/request_form/form/170

Some of the key players include Applied Genetic Technologies Corporation; Bluebird bio; Magenta Therapeutics; MedDay Pharmaceuticals; Minoryx Therapeutics; NeuroVia; Orpheris; ReceptoPharm; SOM Biotech; and Viking Therapeutics. Several M&As along with partnerships have been undertaken by these players to facilitate costumers with hi-tech and innovative products.

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Global Adrenoleukodystrophy Treatment Trends and Highlights - Bulletin Line

For many patients with multiple myeloma, a new generation of drugs and drug combinations is producing better outcomes and fewer side effects. In recent months, several novel therapies studied and tested by Dana-Farber scientists have gained approval from the U.S. Food and Drug Administration (FDA) or taken a step toward approval after posting solid results in clinical trials.

The drugs are the fruit of years of research into improving treatment for multiple myeloma, a cancer of white blood cells known as plasma cells in the bone marrow. Many of the new agents are biologically derived made from substances such as proteins and antibodies found in living things and target biological mechanisms in a very specific, targeted fashion. Dana-Farber researchers have played a key role in these efforts.

These are each powerful examples of how next-generation novel therapies translated here at Dana-Farber from bench to bedside are further improving outcomes for our patients, and at a remarkable pace, says Paul G. Richardson, MD, clinical program leader and director of clinical research at the Jerome Lipper Multiple Myeloma Center at Dana-Farber.

Following a Dana-Farber-led clinical trial, the FDA recently approved the novel drug isatuximab in combination with pomalidomide and dexamethasone for adults with relapsed or refractory (non-responsive) myeloma who have received at least two prior therapies, including lenalidomide and drugs known as proteasome inhibitors. The drug went into trials after laboratory work by Dana-Farbers Yu-Tzu Tai, PhD, and Kenneth Anderson, MD, showed it was active against myeloma cells. In the clinical trial, the three-drug combination lowered the risk that the disease would progress by 40%, compared to pomalidome and dexamethasone alone.

Dana-Farber investigators conducted laboratory research and led the first clinical trial of the drug melflufen plus dexamethasone in patients with relapsed or refractory myeloma. Melflufen is a peptide conjugate drug made of a stub of protein, or peptide, joined to a chemotherapy agent and delivers a toxic payload directly to myeloma cells in a selective, time-sparing approach.

Results from an early-phase clinical trial published in Lancet Oncology showed the drug is active in patients with myeloma and is safe at recommended doses. Unlike the previously used standard drug melphalan, it doesnt cause mucositis inflammation of membranes within the digestive tract or hair loss. The results prompted investigators to launch two larger trials, some of whose results are being processed and are due to be published soon.

In a major study published in Blood, Dana-Farber researchers and their associates found that in patients newly diagnosed with myeloma who are eligible for a stem cell transplant, adding the drug daratumumab to the standard three-drug regimen produced more responses, and deeper responses, than in patients receiving the three-drug therapy alone.

Dana-Farber researchers were involved in the development and initial testing of the drug belantamab mafodotin, which has shown considerable promise in clinical trials and has been granted priority review for approval by the FDA.

An antibody conjugate drug consisting of an antibody that specifically targets myeloma cells and an agent that disrupts cell division, its use was informed by a preclinical trial at Dana-Farber involving Yu-Tzu Tai, PhD, and Kenneth Anderson, MD. Balantamab mafodotin was tested in studies led by Paul Richardson, MD, in patients with relapsed or refractory multiple myeloma whose disease continued to worsen after a stem cell transplant, chemotherapy, or other treatment. In the DREAMM-1 and -2 trials, the drug showed strong anti-myeloma activity with manageable side effects.

After certification in Internal Medicine, Hematology and Medical Oncology, as well as working in Cancer Pharmacology from 1994 onwards at Dana-Farber Cancer Institute (DFCI), Dr. Paul Richardson joined the Jerome Lipper Myeloma Center in 1999, was appointed Clinical Director in 2001, and led the development of several first-generation novel drugs including bortezomib, lenalidomide and pomalidomide for the treatment of multiple myeloma. Subsequent studies have focused on next-generation novel drugs including panobinostat and second-generation proteasome inhibitors including ixazomib. More recently, his clinical innovations have been in the development of the breakthrough monoclonal antibodies elotuzumab and daratumumab for the treatment of both untreated and relapsed myeloma, as well as isatuximab and more broadly, antibody drug conjugates including belantamab mafodotin, as well as other immunotherapeutic strategies. In addition to these agents, he is leading the development of melflufen, a targeted cytotoxic and an first-in-class small molecule inhibitor selinexor, which inhibits XPO-1, a key nuclear export protein, as well as first-in-human studies of cereblon E3 ligase modulators (so called CELMoDs) for the treatment of relapsed and refractory myeloma.

Over the last decade, his major effort has been focused on the development of lenalidomide, bortezomib and dexamethasone (so-called RVD), and its incorporation as part of the Intergroup Francophone Myelome (IFM)/DFCI clinical trial in newly diagnosed patients eligible for stem cell transplant treated with RVD. This regimen has generated an unprecedented response rate, leading to its adoption in this international study, as well as others in the United States and elsewhere. This particular trial incorporates genomic and proteomic evaluation to establish a future platform for tailored therapy and the optimal positioning of stem cell transplant, with results anticipated in 2021-22. Furthermore, RVD has been established as a backbone to which next-generation agents are being added, including elotuzumab, daratumumab and isatuximab, as well as panobinostat.

He has published extensively, having authored or co-authored over 400 original articles and 330 reviews, chapters, and editorials in peer-reviewed journals. In addition to holding positions on the Editorial Boards of leading journals, he is prior Chairman of the Multiple Myeloma Research Consortium (MMRC), Clinical Trials Core, a position held for 5 years as part of a rotating tenure, and for which he continues as a member of the Steering and Project Review Committee. He was also a member of ASCO Hematologic Malignancies Subcommittee for the required one-year term, and then for one year on the ASCO Internet Cancer Information Committee during 2017. He was appointed Chair of the Alliance Myeloma Committee in 2011 and continues in this role.

Honors include the George Canellos Award for Excellence in Clinical Research and Patient Care, and The Tisch Outstanding Achievement Award for Clinical Research, as well as an honorary Fellowship of the Royal College of Physicians (UK), given in recognition for international contributions in multiple Myeloma and stem cell transplantation. He was a co-recipient of the prestigious Warren Alpert Foundation Prize in recognition of the successful therapeutic targeting of the ubiquitin-proteasome pathway in 2012. He was also a co-recipient of the Accelerator Award for contributions to clinical research and patient enrollment in MMRC studies, as well as for the Research Center of the Year Award in 2009, followed by the second award for Center of the Year in 2017. He was ranked by Thomson Reuters Science Watch amongst the top 19 investigators at DFCI for the most highly cited research in 2016. He was the co-recipient of the ASH Ernest Beutler Prize for clinical science and translational research in the development of proteasome inhibition as an effective treatment strategy for multiple myeloma in 2015; the COMY Award for MM research (Paris, France) in 2016, and the prestigious IMF Robert A. Kyle Lifetime Achievement Award in 2017, and the Morse Research Award in 2019.

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Wave of New Therapies Improve Outcomes for Patients with Multiple Myeloma - Dana-Farber Cancer Institute

Dear Doctor: What's the connection between the novel coronavirus and the thymus gland? A friend of ours who is a doctor says it's probably what keeps young kids from getting so sick. I've never even heard of the thymus. What does it have to do with coronavirus?

Dear Reader: From the earliest days of the novel coronavirus pandemic, the data revealed a puzzling disparity. Older adults were at increased risk of grave illness when infected with the virus, but children seemed to have a certain level of protection. And while it has since become clear that children can indeed become seriously ill if they become infected, they do so at far lower rates than adults. The reasons for this are still being investigated, but some researchers have recently suggested the role of the thymus gland as a possible factor.

If you place your finger at the notch at the top of your breast bone and draw a vertical line downward a few inches, you've traced the location of your thymus. It's made up of two roughly triangular lobes, which sit behind the breastbone and between the lungs. The thymus has several functions, but perhaps its most important role is to help produce the cells that will become T-lymphocytes, or T-cells. (The "T" stands for thymus-derived.) These are white blood cells that protect the body from bacteria, fungi, viruses and other pathogens.

T-cells, which are the ninjas of the immune system, start out in the bone marrow as stem cells. The immature stem cells exit the marrow, move through the blood and enter a specific region of the thymus. There, they undergo a complex process that teaches them how to recognize a wide range of potentially dangerous and deadly invaders. As T-cells, their job is to circulate throughout the body and, when they encounter the molecular signature of the pathogen they've been trained to recognize, to attack. T-cells also activate other immune cells, produce proteins known as cytokines and have a role in regulating immune response.

The thymus is unique in that it reaches maturity in utero and is at its largest and most active in children. Starting at puberty, it gradually becomes less active, and the glandular tissue begins to shrink. This continues throughout a person's life. By the time someone has reached their mid-60s, the thymus is largely inactive. By their mid-70s, the gland has been mostly replaced with fat. This decrease in thymus function is believed to be one of the reasons that, in their later years, older adults become more susceptible to disease and infection.

Emerging research into COVID-19 has shown a marked decrease in the number of T-cells in some gravely ill patients. Scientists are now asking whether age-related thymus decline, which means T-cells aren't quickly replaced, may play a role in the severity of illness seen in older adults. The flip side of this is whether, due to their robust production of T-cells, children's immune systems are able to stay one step ahead of the novel coronavirus. It's only a working theory, but it shows promise, and research into how this may affect and inform treatment continues.

Eve Glazier, M.D., MBA, is an internist and associate professor of medicine at UCLA Health. Elizabeth Ko, M.D., is an internist and assistant professor of medicine at UCLA Health.

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Theory suggests thymus plays role in severity of COVID - Times Record

Companies, Organizations Needed to Host Events

MONTVALE, NJ Because of the current, nationwide surge in COVID-19 infections throughout the Sun Belt, along with the constant importance of bolstering the local blood supply, theres now significant need for residents to donate blood and, if possible, convalescent plasma.

The nonprofit, blood-collection organization Vitalantis offering an open-to-the-public Mark Spatola Memorial Drive on Tuesday, August 11 from 2 p.m. to9 p.m. at St. Agnes Parish Center, 55 South Avenue,Atlantic Highlands.

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Vitalant is also seeking companies and organizations throughoutMonmouth Countyto host blood donation events. Information on hosting a donation event is available by clicking here.

Individuals who have recovered from COVID-19 are urged to donate blood plasma. Known as convalescent plasma, this blood component contains antibodies that may provide seriously ill patients an extra boost in fighting the disease.

Vitalant is providing an antibody test which is authorized by the U.S. Food and Drug Administration to all donors; results will be available in private, online donor accounts, approximately two weeks after a donation.There is also a great need for blood platelets small cells in the blood that form clots to prevent bleeding, while also helping with anemia and low blood countsand type O-negative,the universal blood type.

To promote the increase of blood, convalescent plasma, and platelet donations,regular event host companies and organizations many of which put their events on pause due to the pandemic are asked toconsider returning to a consistent schedule of donation events.

FEMA has specifically identified blood donation as an essential and integral component of the emergency support function. Of note, coronavirus cannot be transferred through the blood. And, as always, the blood collection process is safe with noimpact on the donor's immune system. Vitalant staff follows rigorous safety and disinfection protocols at its blood drives and donation centers and have always required individuals to be in good health to donate blood.

Vitalant also maintains four New Jersey blood centers, with hours and street addresses as follows:

Healthy individuals age 16 or older, who weigh at least 110 pounds, may donate blood; 16- and 17-year-olds must have proof of birth date and signed consent forms, either in English or Spanish. Donors should eat a moderate meal prior to donating, and also bring identification featuring their signature.

On occasion, last-minute changes to scheduling for a donation event will occur. As a result, it is recommended that anyone planning to donate blood at a Vitalant donation event call 201-251-3703, toll free, to confirm timing and location details. Additional information about donating blood is also available by visitingwww.vitalant.org.

About Vitalant in New Jersey

A not-for-profit organization that supplies blood and blood products to hospitals in the New Jersey/New York region, Bergen County-based Vitalant (previously Community Blood Services) has been devoted to serving the communitys transfusion medicine needs since 1953. Donations of blood and blood products, umbilical cord blood, stem cells, and bone marrow help to join individuals, organizations, businesses, and entire communities together in partnership to help save lives.

About Vitalant

Arizona-based Vitalant is among the nations oldest and largest transfusion medical organizations in the U.S. Founded in 1943, its blood centers division serves some 700 hospitals across the United States. A founding member of Americas Blood Centers and the AABB (formerly the American Association of Blood Banks), Vitalant also operates biological products distribution services, a quality consulting group, and a world-renowned transfusion medicine research institute. It also is a partner in the operation of high-volume donor testing laboratories.

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Vitalant to Hold Blood Donation Drive on August 11 in Atlantic Highlands - TAPinto.net

CRISPR is a catchy acronym that originally described a naturally occurring gene editing tool, derived from a bacterial defense mechanism against viruses. Its the name on everybodys lips in the intersecting realms of science, medicine, ethics, and politics. From the moment of its discovery, CRISPR-Cas9 looked like a miraculous solution to all of the problems that gene editing efforts have experienced over decades of trial and error. This revolutionary new gene editing technique has opened the doors to both massive scientific progress and ethical controversy. Now more than ever, were seeing that CRISPR still has massive kinks to work out. Can we ever fully understand the social and scientific implications of gene editing, and should we use it in humans before we learn how to properly harness it?

What is gene editing?

The 20th century saw genetic scientists increasingly focus their pursuits on the sub-microscopic. As science delved deeper into the human body in an attempt to uncover the molecular minutiae of life, the possibility of reaching into the cell and manipulating its genetic material began to look more and more real. Even by the 1950s, evidence had been mounting for decades that deoxyribonucleic acid (DNA), an unassuming molecule residing in a central cellular compartment called the nucleus, was the physical genetic material that passed information from parent to child. Finally, in 1953, landmark work by Kings College biochemist Rosalind Franklin allowed Cambridge researchers to reveal the structure of DNA and confirm its role in heredity once and for all.

Starting from a hesitant foundation, molecular genetics exploded in both scope and popularity over subsequent decades. With the secrets of heredity increasingly out in the open, human ambition demanded that we try to bend DNA to our willand now we can. These days, targeted gene editing techniques revolve around artificially-engineered molecular tools known as nucleases, whose earliest use was in 1996not even 50 years after the discovery of DNAs structure. Engineered nucleases are often described as molecular scissors. Fundamentally, they have two main parts: one part that finds and grabs onto the target DNA within a cell, and one part that snips a piece out of that DNA.

How CRISPR works

CRISPR is similar to other directed nucleases, but its much better at its job. The CRISPR part is secondary to the systems gene editing applications; the truly important discovery, which Jennifer Doudna made in 2012, was a protein that she called CRISPR-associated protein 9, or Cas9. This protein is the nuclease tool, the pair of molecular scissors that finds, sticks to, and snips target DNAand its more accurate than anything weve ever seen before.

In bacteria, CRISPR is a section of the genome that acts as an immune memory, storing little snippets of different viruses genetic material as DNA after failed infections, like trophies. When a once-active virus attempts to invade a bacterium, the mobile helper Cas9 copies down the relevant snippet from CRISPR in the form of ribonucleic acid, or RNA. RNA is a molecule thats virtually identical to DNA, except for one extra oxygen atom. Because of this property, the RNA sequence that Cas9 holds can pair exactly, nucleotide by nucleotide, with the viral targets DNA, making it extremely efficient at finding that DNA. With a freshly transcribed RNA guide, the bacterium can deploy Cas9 to findand cut outthe corresponding section of viral genetic material, rendering the attacker harmless.

The existence of CRISPR in bacteria was old news by 2012, but Doudnas discovery of Cas9s function was revolutionary. With a little creativity and ingenuity, such a simple and accurate nuclease can be modified to be much more than just a pair of scissors. Using synthetic RNA guides and certain tweaks, Cas9 can be used to remove specific genes, cause new insertions to genomes, tag DNA sequences with fluorescent probes, and much more.

The possibilities seem endless.What if we could go into the body of a human affected by a hereditary disease and change that persons DNA to cure them? What if we could modify reproductive germ cells in human bodies (which give rise to sperm and eggs), or make targeted genetic edits in the very first cell of an embryo? Nine months of division and multiplication later, that cell would give rise to a human being whose very nature has been deliberately tweakedand their childrens nature, and their childrens. With the accuracy and accessibility of the CRISPR/Cas9 system, these ideas arent hypotheticals. In 2019, CRISPR edits in bone marrow stem cells were successfully used to cure sickle cell anemia in a Mississippi woman. Beta thalassaemia, another genetic disease of the blood, has also been treated this way. In 2018, Chinese scientist He Jiankui even claimed that he had conferred HIV immunity upon twin girls using embryonic editing.

CRISPRs complications

At first glance, CRISPR looks like a miraclebut it isnt perfect. What if some cells were affected by edits, but others werent, creating a strange genetic mosaic in a human body? What if, in trying to modify a specific gene, we accidentally hit a different section of DNA nearby? What if we got the right gene, but it also affected a different part of the body that we didnt know about?

These problems arent hypotheticals either. So-called mosaicism and off-target editing are huge concerns among CRISPR scientists. Mosaicism is of particular concern in embryonic editing. Though CRISPR injections are carried out when an embryo is single-celled, CRISPR doesnt always appear to work until after several rounds of cell divisionand it doesnt work in every cell. If not all the cells in the body are affected by gene editing that is intended to eliminate a genetic disease, the disease could remain in the body. It may be possible to combat mosaicism with faster gene editing (so that cells dont replicate before theyve had a chance to become CRISPR-modified), altering sperm and egg cells before they meet to form an embryo, and developing more precise CRISPR gene editing which is in itself a challenge, thanks to off-target editing.

In nature, a little bit of off-target editing could actually make the CRISPR-Cas9 defense system stronger with the principle of redundancy. Flexibility in the form of imprecision could allow a bacterium to neutralize viruses whose exact genetic sequences have not yet been encountered: viruses related to, but not identical to, previous attackers. In clinical and therapeutic applications, on the other hand, precision is everything. And unfortunately, as time passes, CRISPRs level of precision seems further and further off. Preprints released just this year reveal that the frequency and magnitude of CRISPRs off-target edits in human cells may be worse than we had previously known. Large proportions of cells with massive unwanted DNA deletions, losses of entire chromosomes in experimental embryos, and shuffling of genetic sequences were observed.

Of course, not only do scientists need to avoid off-target edits, but they also need to know when such undesired edits have occurred. Off-target effects can be detected by genome sequencing and computer prediction tools, but theres no perfect way to do it yetthere may still be editing misses that were, well, missing. Off-target edits themselves could be minimized by altering the RNA transcript that Cas9 carries to make it more accurate, altering Cas9 itself, or reducing the actual amount of Cas9 protein released into the cell (though this could also reduce on-target effects). Replacing Cas9 itself with other Cas variants, like smaller and more easily deliverable CasX and CasY proteins, is a promising possibility for more efficient editing, but these candidates still run into many of the same problems as Cas9. More strategies are constantly being discovered, proposed, and explored, but were still nowhere near perfect.

Perhaps most importantly, even barring any purely technical problems, is that humans remain in sheer ignorance of much of the extent and consequences of pleiotropy, a phenomenon where a genes presence or deletion has more than one effect in the human body. Even genes whose function we think we know well might have totally unexpected additional functions. On the other side of the coin, we dont have a comprehensive understanding of how many different genetic contributors there are to any given trait or disease, much less where they lie in the genome. We dont understand the way that thousands of variations across the entire genome contribute to appearance, personality, and health. Assuming that some genes are good and others are bad is morally dangerous, and scientifically reprehensible. In reality, we are not ready for genetic determinism, and may never be.

A great responsibility

Humanity has discovered a great power, but we all know what comes with great power. Questions of which edits are necessary for health (is mild Harlequin syndrome a disease or a cosmetic concern?), whether edits are ethical (should autism and homosexuality be considered curable conditions?), and the possibility of designer babies, among others, are pertinent and require thorough discussion. We also need to realize that making these types of changes isnt our decision until we can get CRISPR right, and understand the genome well enough to target particular phenotypes. Though most scientists are aware of the difficulties of CRISPR and its use is generally tightly regulated, some scientistsand laypeopleare less careful. He Jiankuis apparent miracle HIV cure led to his arrest and imprisonment for unapproved and unethical practice. Its no great surprise that his work likely fell prey to off-target effects and mosaicism; even if he got it right, his intended change could alter cognitive function, and who knows what else?

Non-scientists are getting involved too: in 2018, self-proclaimed biohacker Josiah Zayner publicly injected his own arm with what he claimed was muscle-enhancing CRISPR. Though Zayner is one of the most vocal, hes not the only one of his kind. Quieter biohackers, untrained people without a scientific background or a good understanding of how CRISPR can go wrong, are attempting to edit themselves and even their pets.

Laypeople have an unquestionable place in science: the scientific discipline needs fresh perspectives and creativity that stuffy academics cant offer. CRISPR is still in its infancy, though. Before we know much, much more about its capabilities and consequences, there can be no place for black market gene editing kits, rogue scientists altering human embryonic and germline DNA, or basement geneticists injecting Cas9 into their dogs. Who can say what effects these interventions might have, not just on edited individuals, but on the futures of entire species?

Some say that gene editing is an act of hubris, destined to backfire spectacularly and horrendously. Others believe that its our responsibility to use CRISPR to improve lives. Which of these opinions is true depends on how science walks a narrow tightrope, though Im inclined to agree with the latterand add that our responsibility is not just to master gene editing, but to make clear and public its many faults and failings. The truth, in all its complexity, needs to overcome pop sciences oversimplification and sensationalism. Promising new advances and techniques are on the horizon, but we have a long way to go. Gene editing is no joke; humanity is playing with fire. With an incredibly accurate and accessible nuclease making its way into labs and garages across the world (while its flaws continue to be uncovered year by year), it is more important than ever for the world to understand and discuss the long-reaching consequences and responsible use of gene editing technology. CRISPR is not a miracle, but gene editing may very well be the future of humanityand its on us to keep it under control.

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The Trouble With CRISPR - Strand

The report offers a systematic presentation of the existing trends, growth opportunities, market dynamics that are expected to shape the growth of the Orthopedic Regenerative Medicine Market. The various research methods and tools were involved in the market analysis, to uncover crucial information about the market such as current & future trends, opportunities, business strategies and more, which in turn will aid the business decision-makers to make the right decision in future.

Whats keeping Curasan, Inc., Carmell Therapeutics Corporation, Anika Therapeutics, Inc., Conatus Pharmaceuticals Inc., Histogen Inc., Royal Biologics, Ortho Regenerative Technologies, Inc., Swiss Biomed Orthopaedics AG, Osiris Therapeutics, Inc., and Octane Medical Inc. Ahead in the Market? Benchmark yourself with the strategic moves and findings recently released by CMI

Request a Sample Copy:https://www.coherentmarketinsights.com/insight/request-sample/3566

List of Companies Mentioned:Curasan, Inc., Carmell Therapeutics Corporation, Anika Therapeutics, Inc., Conatus Pharmaceuticals Inc., Histogen Inc., Royal Biologics, Ortho Regenerative Technologies, Inc., Swiss Biomed Orthopaedics AG, Osiris Therapeutics, Inc., and Octane Medical Inc.

1) Does Study provides Latest Impact on Market due to COVID & Slowdown?

Yes study have considered a chapter on Impact Analysis and this 2020 Edition of the report provides detailed analysis and its impact on growth trends and market sizing to better understand current scenario.

2) How companies are selected or profiled in the report?

List of some players that are profiled in the the report includes Curasan, Inc., Carmell Therapeutics Corporation, Anika Therapeutics, Inc., Conatus Pharmaceuticals Inc., Histogen Inc., Royal Biologics, Ortho Regenerative Technologies, Inc., Swiss Biomed Orthopaedics AG, Osiris Therapeutics, Inc., and Octane Medical Inc.. list is sorted to come up with a sample size of atleast 50 to 100 companies having greater topline value to get their segment revenue for market estimation.

** List of companies mentioned may vary in the final report subject to Name Change / Merger etc.

3) Is it possible to narrow down business segments by Application of this study?

Yes, depending upon the data availability and feasibility check by our Research Analyst, further breakdown in business segments by end use application in relation to type can be provided (If applicable) by Revenue Size or Volume*.

4) What is the base year of the study? What time frame is covered in the report?

Furthermore, the years considered for the study are as follows:

Historical year 2014 2018

Base year 2018

Forecast period** 2019 to 2027 [** unless otherwise stated]

**Moreover, it will also include the opportunities available in micro markets for stakeholders to invest, detailed analysis of competitive landscape and product services of key players.

Detailed Segmentation:

By Procedure Cell TherapyTissue EngineeringBy Cell TypeInduced Pluripotent Stem Cells (iPSCs)Adult Stem CellsTissue Specific Progenitor Stem Cells (TSPSCs),Mesenchymal Stem Cells (MSCs)Umbilical Cord Stem Cells (UCSCs)Bone Marrow Stem Cells (BMSCs)By SourceBone MarrowUmbilical Cord BloodAdipose TissueAllograftsAmniotic FluidBy ApplicationsTendons RepairCartilage RepairBone RepairLigament RepairSpine RepairOthers

Regions included:

o North America (United States, Canada, and Mexico)

o Europe (Germany, France, UK, Russia, and Italy)

o Asia-Pacific (China, Japan, Korea, India, and Southeast Asia)

o South America (Brazil, Argentina, Colombia)

o Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria, and South Africa)

Global Orthopedic Regenerative Medicine Market What to expect from this report:

Focused Study on Niche Strategy and Market Development & penetration Scenario

Analysis of M&As, Partnership & JVs in Global Orthopedic Regenerative Medicine Industry in United States & Other Emerging Geographies

Top 10 Global Orthopedic Regenerative Medicine Companies in Global Market Share Analysis: Leaders and Laggards in 2017, 2019

Gain strategic insights on competitor information to formulate effective R&D moves

Identify emerging players and create effective counter-strategies to outpace competitive edge

Identify important and diverse product types/services offering carried by major players for market development

And many more .

TABLE OF CONTENTS

Report Overview:It includes the Orthopedic Regenerative Medicine market study scope, players covered, key market segments, market analysis by application, market analysis by type, and other chapters that give an overview of the research study.

Executive Summary:This section of the report gives information about Orthopedic Regenerative Medicine market trends and shares, market size analysis by region and analysis of global market size. Under market size analysis by region, analysis of market share and growth rate by region is provided.

Profiles of International Players:Here, key players of the Orthopedic Regenerative Medicine market are studied on the basis of gross margin, price, revenue, corporate sales, and production. This section gives a business overview of the players and shares their important company details.

Regional Study:All of the regions and countries analyzed in the Orthopedic Regenerative Medicine market report is studied on the basis of market size by application, the market size by product, key players, and market forecast.

Download PDF Brochure @https://www.coherentmarketinsights.com/insight/request-pdf/3566

Thanks for reading this article; you can also get individual chapter wise section or region wise report version like North America, Europe or Asia.

About Author:

Coherent Market Insights is a global market intelligence and consulting organization focused on assisting our plethora of clients achieve transformational growth by helping them make critical business decisions. We are headquartered in India, having office at global financial capital in the U.S. Our client base includes players from across all business verticals in over 150 countries worldwide. We are uniquely positioned to help businesses around the globe deliver practical and lasting results through various recommendations about operational improvements, technologies, emerging market trends and new working methods.

Mr Raj ShahCoherent Market Insights 1001 4th Ave,#3200 Seattle, WA 98154, U.S.Phone +1-206-701-6702[emailprotected]

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Orthopedic Regenerative Medicine Market Insights and Business Outlook By Top Players Curasan, Inc., Carmell Therapeutics Corporation, Anika...

Roots Analysis has announced the addition of Global Stem Cells Market: Focus on Clinical Therapies, 20202030 (Based on Source (Allogeneic, Autologous); Origin (Adult, Embryonic); Type (Hematopoietic, Mesenchymal, Progenitor); Lineage (Amniotic Fluid, Adipose Tissue, Bone Marrow, Cardiosphere, Chondrocytes, Corneal Tissue, Cord Blood, Dental Pulp, Neural Tissue Placenta, Peripheral Blood, Stromal Cells); and Potency (Multipotent, Pluripotent)) report to its list of offerings.

There is a growing body of evidence supporting the vast applicability and superiority of treatment outcomes of stem cell therapies, compared to conventional treatment options. In fact, the unmet needs within this domain have spurred the establishment of many start-ups in recent years.

To order this 500+ page report, which features 185+ figures and 220+ tables, please visit this link

Key Market Insights

Over 280 stem cell therapies are under development, most of which are allogeneic products

More than 50% of the pipeline candidates are in the mid to late phase trials (phase II and above), and allogenic therapies (majority of which are derived from the bone marrow) make up 65% of the pipeline.

70% of pipeline candidates are based on mesenchymal stem cells

It is worth highlighting that the abovementioned therapies are designed to treat musculoskeletal (22%), neurological (21%) and cardiovascular (15%) disorders. On the other hand, hematopoietic stem cell-based products are mostly being evaluated for the treatment of oncological disorders, primarily hematological malignancies.

Close to 85% stem cell therapy developers are based in North America and Asia-Pacific regions

Within these regions, the US, China, South Korea and Japan, have emerged as key R&D hubs for stem cell therapies. It is worth noting that majority of the initiatives in this domain are driven by small / mid-sized companies

Over 1,500 grants were awarded for stem cell research, since 2015

More than 45% of the total amount was awarded under the R01 mechanism (which supports research projects). The NCI, NHLBI, NICHD, NIDDK, NIGMS and OD emerged as key organizations that have offered financial support for time periods exceeding 25 years as well.

Outsourcing has become indispensable to R&D and manufacturing activity in this domain

Presently, more than 80 industry / non-industry players, based in different regions across the globe, claim to provide contract development and manufacturing services to cater to the unmet needs of therapy developers. Examples include (in alphabetical order) Bio Elpida, Cell and Gene Therapy Catapult, Cell Tech Pharmed, GenCure, KBI Biopharma, Lonza, MEDINET, Nikon CeLL innovation, Roslin Cell Therapies, WuXi Advanced Therapies and YposKesi.

North America and Asia-Pacific markets are anticipated to capture over 80% share by 2030

The stem cell therapies market is anticipated to witness an annualized growth rate of over 30% during the next decade. Interestingly, the market in China / broader Asia-Pacific region is anticipated to grow at a relatively faster rate.

To request a sample copy / brochure of this report, please visit this link

Key Questions Answered

The USD 8.5 billion (by 2030) financial opportunity within the stem cell therapies market has been analyzed across the following segments:

The report features inputs from eminent industry stakeholders, according to whom stem cell therapies are currently considered to be a promising alternatives for the treatment of a myriad of disease indications, with the potential to overcome challenges associated with conventional treatment options. The report includes detailed transcripts of discussions held with the following experts:

The research covers brief profiles of several companies (including those listed below); each profile features an overview of the company, financial information (if available), stem cell therapy portfolio and an informed future outlook.

For additional details, please visit

https://www.rootsanalysis.com/reports/view_document/stem-cells-market/296.html or email [emailprotected]

You may also be interested in the following titles:

Contact:

Gaurav Chaudhary

+1 (415) 800 3415

+44 (122) 391 1091

[emailprotected]

Link:
With over 280 therapies under evaluation, the stem cell therapy market is estimated to be worth USD 8.5 Billion by 2030, claims Roots Analysis -...

New Delhi: Cancer takes innumerable lives each year, and so does the unavailability of cancer treatment due to lack of funds. 33-year-old Nushafreen Palsetia, a software engineer based in Mumbai, was recently diagnosed with a very aggressive form of cancer. Despite a relapse, what helped was life-saving support pouring from over 1,700 donors.

Nushafreen was first diagnosed with Non-Hodgkin Lymphoma in April 2019. After a year of enduring aggressive treatment, she tried to get back to her normal life and work. Unfortunately, Non-Hodgkin Lymphoma, Diffuse Large B Cell Lymphoma (DLBCL), cancer relapsed in her liver in May 2020 which was an unexpected major shock, leaving her and all of her family overwhelmed.

Doctors planned to perform an autologous (her own stem cells) bone marrow transplant in India after the chemotherapy but further tests showed involvement of the bone marrow as well. Hence, her treating doctor recommended the modern CAR-T Cell therapy treatment, available only in the USA, UK, Israel, and a few European countries.

Nushafreens family found Israel as the most affordable option as compared to all other countries offering the treatment. They reached out to Sheba Medical Centre in Israel which estimated the medical expenses as 200,000 USD (approximately Rs 1.5 crore). The treatment will require Nushafreen to be hospitalized for a month or more for the response to treatment and immediate follow up.

As Nushafreens family couldnt afford the high medical expenses, an ImpactGuru crowdfunding campaign was initiated. In two weeks, Nushafreens ImpactGuru.com Page has raised over Rs 1 crore from 1750 donors. The platform raises money online for medical expenses via crowdfunding such as cancer, transplants, and accidents.

According to the co-founder and CEO of the healthcare crowdfunding platform, Piyush Jain, Crowdfunding is driven by a culture of generosity, it allows people to raise money quickly in a hassle free manner without any payback liability. This is a new record for our platform with a single patients family being able to raise more than Rs 1 crore. We hope Nushafreen recovers soon and more patients come forward to utilize our platform in their time of need to get the best available treatment for critical illnesses.

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Crowdfunding to the rescue for this cancer patient - Telangana Today

DetailsCategory: AntibodiesPublished on Sunday, 02 August 2020 16:34Hits: 344

COPENHAGEN, Denmark I July 31, 2020 I Genmab A/S (Nasdaq: GMAB) announced today that the European Myeloma Network (EMN) in collaboration with Janssen Research & Development, LLC (Janssen) reported positive results from the Phase 3 APOLLO (MMY3013) study of the subcutaneous (SC) formulation of daratumumab in combination with pomalidomide and dexamethasone (Pd) versus Pd alone as treatment for patients with relapsed or refractory multiple myeloma who have previously been treated with lenalidomide (an immunomodulatory drug) and a proteasome inhibitor (PI). The study met the primary endpoint of improving progression-free survival (PFS). Overall, the safety profile of daratumumab SC in combination with Pd was consistent with the safety profile for each therapy separately.

We are pleased with these positive results for daratumumab, administered as a subcutaneous formulation, in combination with pomalidomide and dexamethasone. The corresponding intravenous regimen was previously approved by the U.S. FDA based on the Phase1 single-arm EQUULEUS study, said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

Janssen Biotech, Inc., which obtained an exclusive worldwide license to develop, manufacture and commercialize daratumumab from Genmab in 2012, intends to discuss the data with health authorities in preparation for regulatory submissions and plans to submit the data for presentation at an upcoming medical conference.

The APOLLO study was designed to confirm the results from the Phase 1 EQUULEUS (MMY1001) study, which investigated intravenous (IV) daratumumab plus Pd in the same indication. In June 2017, the U.S. Food and Drug Administration (U.S. FDA) approved the use of DARZALEX in combination with Pd for the treatment of patients with multiple myeloma who have received at least two prior therapies including lenalidomide and aPI based on the results of the EQUULEUS study.

About the APOLLO (MMY3013) studyThis Phase 3 (NCT03180736), randomized, open-label, multicenter study included 304 patients with multiple myeloma who have previously been treated with lenalidomide and a PI. Patients were randomized 1:1 to either receive daratumumab in combination with Pd or Pd alone. In the original design of the study, patients in the daratumumab plus Pd arm were treated with the IV formulation of daratumumab. As of Amendment 1, all new subjects in the experimental arm were dosed with the SC formulation of daratumumab and patients who had already begun treatment with IV daratumumab had the option to switch to the SC formulation. The primary endpoint of the study was PFS. The study was conducted in Europe under an agreement between Janssen, EMN and Stichting Hemato-Oncologie voor Volwassenen Nederland (HOVON).

About multiple myelomaMultiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterized by an excess proliferation of plasma cells.1 Multiple myeloma is the third most common blood cancer in the U.S., after leukemia and lymphoma.2 Approximately 26,000 new patients were estimated diagnosed with multiple myeloma and approximately 13,650 people were expected to have died from the disease in the U.S. in 2018.3 Globally, it was estimated that 160,000 people were diagnosed and 106,000 died from the disease in 2018.4 While some patients with multiple myeloma have no symptoms at all, most patients are diagnosed due to symptoms which can include bone problems, low blood counts, calcium elevation, kidney problems or infections.5

About DARZALEX (daratumumab)DARZALEX (daratumumab) intravenous infusion is indicated for the treatment of adult patients in the United States: in combination with bortezomib, thalidomide and dexamethasone as treatment for patients newly diagnosed with multiple myeloma who are eligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy; in combination with pomalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor (PI); and as a monotherapy for the treatment of patients with multiple myeloma who have received at least three prior lines of therapy, including a PI and an immunomodulatory agent, or who are double-refractory to a PI and an immunomodulatory agent.6 DARZALEX is the first monoclonal antibody (mAb) to receive U.S. Food and Drug Administration (U.S. FDA) approval to treat multiple myeloma.

DARZALEX is indicated for the treatment of adult patients in Europe via intravenous infusion or subcutaneous administration: in combination with bortezomib, thalidomide and dexamethasone as treatment for patients newly diagnosed with multiple myeloma who are eligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with bortezomib, melphalan and prednisone for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; for use in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least one prior therapy; and as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a PI and an immunomodulatory agent and who have demonstrated disease progression on the last therapy7. Daratumumab is the first subcutaneous CD38-directed antibody approved in Europe for the treatment of multiple myeloma. The option to split the first infusion of DARZALEX over two consecutive days has been approved in both Europe and the U.S.

In Japan, DARZALEX intravenous infusion is approved for the treatment of adult patients: in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone for the treatment of relapsed or refractory multiple myeloma. DARZALEX is the first human CD38 monoclonal antibody to reach the market in the United States, Europe and Japan. For more information, visit http://www.DARZALEX.com.

DARZALEX FASPRO (daratumumab and hyaluronidase-fihj), a subcutaneous formulation of daratumumab, is approved in the United States for the treatment of adult patients with multiple myeloma: in combination with bortezomib, melphalan and prednisone in newly diagnosed patients who are ineligible for ASCT; in combination with lenalidomide and dexamethasone in newly diagnosed patients who are ineligible for ASCT and in patients with relapsed or refractory multiple myeloma who have received at least one prior therapy; in combination with bortezomib and dexamethasone in patients who have received at least one prior therapy; and as monotherapy, in patients who have received at least three prior lines of therapy including a PI and an immunomodulatory agent or who are double-refractory to a PI and an immunomodulatory agent.8 DARZALEX FASPRO is the first subcutaneous CD38-directed antibody approved in the U.S. for the treatment of multiple myeloma.

Daratumumab is a human IgG1k monoclonal antibody (mAb) that binds with high affinity to the CD38 molecule, which is highly expressed on the surface of multiple myeloma cells. Daratumumab triggers a persons own immune system to attack the cancer cells, resulting in rapid tumor cell death through multiple immune-mediated mechanisms of action and through immunomodulatory effects, in addition to direct tumor cell death, via apoptosis (programmed cell death).6,9,10,11,12

Daratumumab is being developed by Janssen Biotech, Inc. under an exclusive worldwide license to develop, manufacture and commercialize daratumumab from Genmab. A comprehensive clinical development program for daratumumab is ongoing, including multiple Phase III studies in smoldering, relapsed and refractory and frontline multiple myeloma settings. Additional studies are ongoing or planned to assess the potential of daratumumab in other malignant and pre-malignant diseases in which CD38 is expressed, such as amyloidosis and T-cell acute lymphocytic leukemia (ALL). Daratumumab has received two Breakthrough Therapy Designations from the U.S. FDA for certain indications of multiple myeloma, including as a monotherapy for heavily pretreated multiple myeloma and in combination with certain other therapies for second-line treatment of multiple myeloma.

About Genmab Genmab is a publicly traded, international biotechnology company specializing in the creation and development of differentiated antibody therapeutics for the treatment of cancer. Founded in 1999, the company is the creator of three approved antibodies: DARZALEX (daratumumab, under agreement with Janssen Biotech, Inc.) for the treatment of certain multiple myeloma indications in territories including the U.S., Europe and Japan, Arzerra (ofatumumab, under agreement with Novartis AG), for the treatment of certain chronic lymphocytic leukemia indications in the U.S., Japan and certain other territories and TEPEZZA (teprotumumab, under agreement with Roche granting sublicense to Horizon Therapeutics plc) for the treatment of thyroid eye disease in the U.S. A subcutaneous formulation of daratumumab, known as DARZALEX FASPRO (daratumumab and hyaluronidase-fihj) in the U.S., has been approved in the U.S. and Europe for the treatment of adult patients with certain multiple myeloma indications. Daratumumab is in clinical development by Janssen for the treatment of additional multiple myeloma indications, other blood cancers and amyloidosis. A subcutaneous formulation of ofatumumab is in development by Novartis for the treatment of relapsing multiple sclerosis. Genmab also has a broad clinical and pre-clinical product pipeline. Genmab's technology base consists of validated and proprietary next generation antibody technologies - the DuoBody platform for generation of bispecific antibodies, the HexaBody platform, which creates effector function enhanced antibodies, the HexElect platform, which combines two co-dependently acting HexaBody molecules to introduce selectivity while maximizing therapeutic potency and the DuoHexaBody platform, which enhances the potential potency of bispecific antibodies through hexamerization. The company intends to leverage these technologies to create opportunities for full or co-ownership of future products. Genmab has alliances with top tier pharmaceutical and biotechnology companies. Genmab is headquartered in Copenhagen, Denmark with sites in Utrecht, the Netherlands, Princeton, New Jersey, U.S. and Tokyo, Japan.

1 American Cancer Society. "Multiple Myeloma Overview." Available at http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-what-is-multiple-myeloma.Accessed June 2016.2 National Cancer Institute. "A Snapshot of Myeloma." Available at http://www.cancer.gov/research/progress/snapshots/myeloma. Accessed June 2016. 3 Globocan 2018. United States of America Fact Sheet. Available at http://gco.iarc.fr/today/data/factsheets/840-united-states-of-america-fact-sheets.pdf.4 Globocan 2018. World Fact Sheet. Available at http://gco.iarc.fr/today/data/factsheets/populations/900-world-fact-sheets.pdf. Accessed December 2018.5 American Cancer Society. "How is Multiple Myeloma Diagnosed?" http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-diagnosis. Accessed June 20166 DARZALEX Prescribing information, September 2019. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2019/761036s024lbl.pdf Last accessed September 20197 DARZALEX Summary of Product Characteristics, available at https://www.ema.europa.eu/en/medicines/human/EPAR/darzalex Last accessed June 20208 DARZALEX FASPRO Prescribing information, May 2020. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/761145s000lbl.pdf Last accessed May 20209 De Weers, M et al. Daratumumab, a Novel Therapeutic Human CD38 Monoclonal Antibody, Induces Killing of Multiple Myeloma and Other Hematological Tumors. The Journal of Immunology. 2011; 186: 1840-1848.10 Overdijk, MB, et al. Antibody-mediated phagocytosis contributes to the anti-tumor activity of the therapeutic antibody daratumumab in lymphoma and multiple myeloma. MAbs. 2015; 7: 311-21.11 Krejcik, MD et al. Daratumumab Depletes CD38+ Immune-regulatory Cells, Promotes T-cell Expansion, and Skews T-cell Repertoire in Multiple Myeloma. Blood. 2016; 128: 384-94.12 Jansen, JH et al. Daratumumab, a human CD38 antibody induces apoptosis of myeloma tumor cells via Fc receptor-mediated crosslinking.Blood. 2012; 120(21): abstract 2974.

SOURCE: Genmab

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Genmab Announces European Myeloma Network and Janssen Achieve Positive Topline Results from Phase 3 APOLLO Study of Daratumumab in Combination with...

FDA Approves Tafasitamab-cxix in Combination With Lenalidomide for the Treatment of DLBCL

A humanized Fc-modified cytolytic CD19 targeting monoclonal antibody, tafasitamab-cxix received accelerated approval based on overall response rate (ORR). Continued approval may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Globally, DLBCL is the most common type of non-Hodgkin lymphoma in adults. The aggressive disease is characterized by rapidly growing masses of malignant B-cells in the lymph nodes, spleen, liver, bone marrow or other organs, and about 1 in 3 patients do not respond to initial therapy or relapsing thereafter.In the United States, approximately 10,000 patients are diagnosed with relapsed or refractory DLBCL who are not eligible for ASCT, each year.

The FDA approval was based on data from the MorphoSys-sponsored Phase 2 L-MIND study, an open label, multicenter, single arm trial of tafasitamab-cxix in combination with lenalidomide as a treatment for adult patients with relapsed or refractory DLBCL. Results from the study showed an ORR of 55% (primary endpoint), including a complete response (CR) rate of 37%, and a partial response rate (PR) of 18%. The median duration of response (mDOR) was 21.7 months (key secondary endpoint).

Warnings and precautions for tafasitamab-cxix included infusion-related reactions (6%), serious or severe myelosuppression including neutropenia (50%), thrombocytopenia (18%), and anemia (7%); infections (73%), and embryo-fetal toxicity. Neutropenia led to treatment discontinuation in 3.7% of patients. The most common adverse reactions ( 20%) were neutropenia, fatigue, anemia, diarrhea, thrombocytopenia, cough, pyrexia, peripheral edema, respiratory tract infection, and decreased appetite.

The FDA previously granted Fast Track and Breakthrough Therapy Designation for the tafasitamab-cxix and lenalidomide combination in treatment of relapsed or refractory DLBCL. FDA.

Tafasitamab-cxix is expected to be commercially available in the United States shortly, according to MorphoSys and Incyte, which plan to co-commercialize this therapy in the US. Incyte has exclusive commercialization rights outside the US.

REFERENCE

FDA Approves Monjuvi(tafasitamab-cxix) in Combination With Lenalidomide for the Treatment of Adult Patients With Relapsed or Refractory Diffuse Large B-cell Lymphoma (DLBCL) [news release]. Planegg & Munich, Germany, and Wilmington, DE; July 31, 2020: MorphoSys and Incyte.https://www.businesswire.com/news/home/20200731005497/en.

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FDA Approves Tafasitamab-cxix in Combination With Lenalidomide for the Treatment of DLBCL - Pharmacy Times

The Stem Cell Market report 2020 covers all the significant developments which are recently being adopted across the global market. The prime objective of the Stem Cell market report is to provides an in-depth analysis of all market dynamics including drivers and restraints, and trends, and opportunities. The Stem Cell market report covers both the demand and supply aspects of the market. The report also highlighted the future trends in the Stem Cell market that will impact the demand during the forecast period.

The scope of this market is limited to tracking the stem cell market. As per the scope of this report, stem cells are biological cells that can differentiate into other types of cells. Also, various types of stem cells are used for therapeutic purposes.

Report Highlights:

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Key Market Trends:

Oncology Disorders Segment is Expected to Exhibit Fastest Growth Rate Over the Forecast Period

Cancer has a major impact on society in the United States and across the world. As per the estimation of National Cancer Institute, in 2018, 1,735,350 new cases of cancer were anticipated to get diagnosed in the United States, and 609,640 deaths were expected from the disease. This increasing medical burden is due to population growth. Bone marrow transplant or stem cell transplant is a treatment for some types of cancers, like leukemia, multiple myeloma, multiple myeloma, neuroblastoma, or some types of lymphoma.

Embryonic stem cells (ESC) are the major source of stem cells for therapeutic purposes, due to their higher totipotency and indefinite lifespan, as compared to adult stem cells with lower totipotency and restricted lifespan. However, the use of ESCs for research and therapeutic purposes is restricted and prohibited in many countries throughout the world, due to some ethical constraints. Scientists from the University of California, Irvine, created the stem cell-based approach to kill cancerous tissue while preventing some toxic side effects of chemotherapy by treating the disease in a more localized way.

Although the market shows positive growth, due to the growing focus of stem cell-based research that can further strengthen the clinical application, its expensive nature for stem cell therapy may still hamper its growth.

North America Captured The Largest Market Share and is Expected to Retain its Dominance

North America dominated the overall stem cell market with the United States contributing to the largest share in the market. In 2014, the Sanford Stem Cell Clinical Center at the University of California, San Diego (UCSD) Health System, announced the launch of a clinical trial, in order to assess the safety of neural stem cell-based therapy in patients with chronic spinal cord injury. Researchers hoped that the transplanted stem cells may develop into new neurons that could replace severed or lost nerve connections, and restore at least some motor and sensory functions. Such numerous stem cell studies across the United States have helped in the growth of the stem cell market.

The Report Covers:

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Detailed TOC of Stem Cell Market Report 2020-2024:

1 INTRODUCTION1.1 Study Deliverables1.2 Study Assumptions1.3 Scope of the Study

2 RESEARCH METHODOLOGY

3 EXECUTIVE SUMMARY

4 MARKET DYNAMICS4.1 Market Overview4.2 Market Drivers4.2.1 Increased Awareness about Umbilical Stem Cell4.2.2 Increase in the Approval for Clinical Trials in Stem Cell Research4.2.3 Growing Demand for Regenerative Treatment Option4.2.4 Rising R&D Initiatives to Develop Therapeutic Options for Chronic Diseases4.3 Market Restraints4.3.1 Expensive Procedures4.3.2 Regulatory Complications4.3.3 Ethical and Moral Framework4.4 Industry Attractiveness- Porters Five Forces Analysis4.4.1 Threat of New Entrants4.4.2 Bargaining Power of Buyers/Consumers4.4.3 Bargaining Power of Suppliers4.4.4 Threat of Substitute Products4.4.5 Intensity of Competitive Rivalry

5 MARKET SEGMENTATION5.1 By Product Type5.1.1 Adult Stem Cell5.1.2 Human Embryonic Cell5.1.3 Pluripotent Stem Cell5.1.4 Other Product Types5.2 By Therapeutic Application5.2.1 Neurological Disorders5.2.2 Orthopedic Treatments5.2.3 Oncology Disorders5.2.4 Diabetes5.2.5 Injuries and Wounds5.2.6 Cardiovascular Disorders5.2.7 Other Therapeutic Applications5.3 By Treatment Type5.3.1 Allogeneic Stem Cell Therapy5.3.2 Auto logic Stem Cell Therapy5.3.3 Syngeneic Stem Cell Therapy5.4 By Banking Service and Technology5.4.1 Stem Cell Acquisition and Testing5.4.2 Cell Production5.4.3 Expansion5.4.4 Sub-culture5.4.5 Cryopreservation5.5 By Type of Banking5.5.1 Public5.5.2 Private5.6 Geography5.6.1 North America5.6.1.1 US5.6.1.2 Canada5.6.1.3 Mexico5.6.2 Europe5.6.2.1 UK5.6.2.2 Germany5.6.2.3 France5.6.2.4 Italy5.6.2.5 Spain5.6.2.6 Rest of Europe5.6.3 Asia-Pacific5.6.3.1 China5.6.3.2 Japan5.6.3.3 India5.6.3.4 Australia5.6.3.5 South Korea5.6.3.6 Rest of Asia-Pacific5.6.4 Middle East & Africa5.6.4.1 GCC5.6.4.2 South Africa5.6.4.3 Rest of Middle East & Africa5.6.5 South America5.6.5.1 Brazil5.6.5.2 Argentina5.6.5.3 Rest of South America

6 COMPETITIVE LANDSCAPE6.1 Company Profiles6.1.1 Osiris Therapeutics Inc.6.1.2 Pluristem Therapeutics Inc.6.1.3 Thermo Fisher Scientific6.1.4 Qiagen NV6.1.5 Sigma Aldrich Corporation6.1.6 Becton, Dickinson and Company6.1.7 Stem Cell Technologies Inc.6.1.8 AllCells LLC6.1.9 Miltenyi Biotec6.1.10 International Stem Cell Corporation

7 MARKET OPPORTUNITIES AND FUTURE TRENDS

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Stem Cell Market Analysis and Forecast by Rapid Growth Rate 2020 Value Share Analysis by Regions, Industry Size, Key Insights till 2024 - Bulletin...

Stem Cell Therapy Market 2020 Research Report provides Emerging Market trends, Market Segmentation, regional outlook and comprehensive analysis on different market segments. It also provides key analysis on the market status of the Stem Cell Therapy manufacturers with best facts and figures, meaning, definition, SWOT analysis, expert opinions and the latest developments across the globe.This report also studies the global market competition landscape, market drivers and trends, opportunities and challenges, risks and entry barriers, sales channels, distributors and Porters Five Forces Analysis.

COVID-19 can affect the global economy in three main ways: by directly affecting production and demand, by creating supply chain and market disruption, and by its financial impact on firms and financial markets.

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Short Description About Stem Cell Therapy Market :

Stem-cell therapy is the use of stem cells to treat or prevent a disease or condition. Bone marrow transplant is the most widely used stem-cell therapy, but some therapies derived from umbilical cord blood are also in use.

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The research covers the current Stem Cell Therapy market size of the market and its growth rates based on 5-year records with company outline ofKey players/manufacturers:

Scope of the Stem Cell Therapy Market Report:

In the last several years, global stem cell therapy market developed fast at a average growth rate of 46.81%. In 2017, the global stem cell therapy market size was 235 million USD, and the market is expected to be 277 million USD.

On the basis of therapeutic application, the global stem cell therapy market is segmented into Musculoskeletal Disorder, Wounds & Injuries, Cornea, Cardiovascular Diseases, and other applications. The musculoskeletal disorders and Wounds & Injuries are expected to dominated the market.

The global Stem Cell Therapy market is valued at 280 million USD in 2018 and is expected to reach 710 million USD by the end of 2024, growing at a CAGR of 20.7% between 2019 and 2024.

The Asia-Pacific will occupy for more market share in following years, especially in China, also fast growing India and Southeast Asia regions.

North America, especially The United States, will still play an important role which cannot be ignored. Any changes from United States might affect the development trend of Stem Cell Therapy.

Europe also play important roles in global market, with market size of xx million USD in 2019 and will be xx million USD in 2024, with a CAGR of xx%.

This report studies the Stem Cell Therapy market status and outlook of Global and major regions, from angles of players, countries, product types and end industries; this report analyzes the top players in global market, and splits the Stem Cell Therapy market by product type and applications/end industries.

Get a Sample Copy of the Stem Cell Therapy Market Report 2020

Report further studies the market development status and future Stem Cell Therapy Market trend across the world. Also, it splits Stem Cell Therapy market Segmentation by Type and by Applications to fully and deeply research and reveal market profile and prospects.

Major Classifications are as follows:

Major Applications are as follows:

Geographically, this report is segmented into several key regions, with sales, revenue, market share and growth Rate of Stem Cell Therapy in these regions, from 2014 to 2024, covering

This Stem Cell Therapy Market Research/Analysis Report Contains Answers to your following Questions

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Major Points from Table of Contents:

1. Market Overview1.1 Stem Cell Therapy Introduction1.2 Market Analysis by Type1.3 Market Analysis by Applications1.4 Market Dynamics1.4.1 Market Opportunities1.4.2 Market Risk1.4.3 Market Driving Force

2.Manufacturers Profiles

2.4.1 Business Overview2.4.2 Stem Cell Therapy Type and Applications2.4.2.1 Product A2.4.2.2 Product B

3.Global Stem Cell Therapy Sales, Revenue, Market Share and Competition By Manufacturer (2019-2020)

3.1 Global Stem Cell Therapy Sales and Market Share by Manufacturer (2019-2020)3.2 Global Stem Cell Therapy Revenue and Market Share by Manufacturer (2019-2020)3.3 Market Concentration Rates3.3.1 Top 3 Stem Cell Therapy Manufacturer Market Share in 20203.3.2 Top 6 Stem Cell Therapy Manufacturer Market Share in 20203.4 Market Competition Trend

4.Global Stem Cell Therapy Market Analysis by Regions

4.1 Global Stem Cell Therapy Sales, Revenue and Market Share by Regions4.1.1 Global Stem Cell Therapy Sales and Market Share by Regions (2014-2019)4.1.2 Global Stem Cell Therapy Revenue and Market Share by Regions (2014-2019)4.2 North America Stem Cell Therapy Sales and Growth Rate (2014-2019)4.3 Europe Stem Cell Therapy Sales and Growth Rate (2014-2019)4.4 Asia-Pacific Stem Cell Therapy Sales and Growth Rate (2014-2019)4.6 South America Stem Cell Therapy Sales and Growth Rate (2014-2019)4.6 Middle East and Africa Stem Cell Therapy Sales and Growth Rate (2014-2019)

5.Stem Cell Therapy Market Forecast (2020-2024)5.1 Global Stem Cell Therapy Sales, Revenue and Growth Rate (2020-2024)5.2 Stem Cell Therapy Market Forecast by Regions (2020-2024)5.3 Stem Cell Therapy Market Forecast by Type (2020-2024)5.3.1 Global Stem Cell Therapy Sales Forecast by Type (2020-2024)5.3.2 Global Stem Cell Therapy Market Share Forecast by Type (2020-2024)5.4 Stem Cell Therapy Market Forecast by Application (2020-2024)5.4.1 Global Stem Cell Therapy Sales Forecast by Application (2020-2024)5.4.2 Global Stem Cell Therapy Market Share Forecast by Application (2020-2024)

6.Sales Channel, Distributors, Traders and Dealers6.1 Sales Channel6.1.1 Direct Marketing6.1.2 Indirect Marketing6.1.3 Marketing Channel Future Trend6.2 Distributors, Traders and Dealers

7.Research Findings and Conclusion

8.Appendix8.1 Methodology8.2 Data Source

Continued..

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Stem Cell Therapy Market 2020 : Top Countries Data with Revenue, Top Manufacturers, Market Size and 2024 Forecast Research Report - Market Research...

Cambridge, Mass.-based AlloVir raised $276 million in an upsized initial public offering. The funds will be used to advance the companys efforts to develop an allogeneic cell therapy to control infections in immunocompromised patients.

Shares of AlloVir began trading on the Nasdaq exchange Thursday. The initial public offering was at $17 per share. The company stock closed at $23.59 per share on its first day of trading. The stock is trading under the ticker symbol ALVR.

AlloVir, formerly known as ViraCyte, is developing allogeneic, off-the-shelf virus-specific T Cell (VST) therapy candidates targeting 12 devastating viruses, including COVID-19. AlloVir collects blood from third-party donors whose T-cells have been exposed to viruses. The company then grows those cells in its laboratory to develop its antiviral therapy. Instead of performing a type of genetic engineering on the T-cells, AlloVir searches for donors whose immune system has been exposed to the corresponding viruses and then using a screening process to identify bone marrow donors. AlloVir then gives the T-cells to patients who are a match in order to kill the virus without harming the healthy cells.

Funds from the IPO will be used in part to advance the companys lead clinical product through late-stage development and potentially to commercialization. AlloVirs lead product is Viralym-M, an allogeneic, off-the-shelf, multi-virus specific T-cell therapy targeting six common viral pathogens in immunocompromised individuals: BK virus, cytomegalovirus, adenovirus, Epstein-Barr virus, human herpesvirus 6, and JC virus. Viralym-M is aimed to begin Phase III in three different indications in immunocompromised patients post allogeneic hematopoietic stem cell transplantwho have complications that have been linked to cytomegalovirus, adenovirus and virus-associated hemorrhagic-cystitis. In a Phase II proof-of-concept study, Viralym-M hit the mark. More than 90% of patients who failed conventional treatment and received Viralym-M, demonstrated a predefined criteria for a complete or partial clinical response, most with complete elimination of detectable virus in the blood and resolution of major clinical symptoms, the company said.

In addition to the Phase III trials, AlloVir intends to begin three Phase II studies with Viralym-M as a preventative treatment for multi-virus infections in HSCT patients, and the treatment of BK and cytomegalovirus in kidney and solid organ transplant recipients.

AlloVir will also use some of the funds to advance some preclinical assets into Phase I studies, including ALVR106 and ALVR109, a potential treatment for COVID-19. In March, AlloVir and its development partner Baylor College, announced plans to expand their development program to include COVID-19. With AlloVirs proprietary technology, in addition to targeting SARS-CoV-2, the investigational virus specific T-cell therapy may also address other coronavirus (CoV) strains including SARS-CoV, MERS-CoV, and also the endemic CoVs that commonly afflict immunocompromised patients. AlloVir aims to develop a therapy for CoVs that can be used as a stand-alone treatment or incorporated into the companys multi-respiratory virus investigational therapy, ALVR106, which is designed to address other devastating and life-threatening community-acquired respiratory viruses, the company said.

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AlloVir Raises $276 Million in IPO to Advance Cell Therapy Programs - BioSpace

Be wary of stem cell therapy as a preventative treatment for COVID-19, warns Laertis Ikonomou, a UB expert on stem cell and gene therapies.

While stem cell therapy, such as bone marrow transplantation, may be used to treat a limited number of diseases and conditions, there are currently no clinically tested or government-approved cell therapies available for the treatment or prevention of COVID-19, says Ikonomou, associate professor of oral biology in the School of Dental Medicine.

He urges the public to exercise caution as the nation experiences a rise in businesses offering direct-to-consumer, unproven and unsafe stem cell therapies that promise to prevent COVID-19 by strengthening the immune system or improving overall health.

What these patients are actually sold is false hope, he says. These businesses are continuously transforming and reinventing themselves, but the common thread is that they offer potentially dangerous treatments based on unproven science.

Ikonomou is also the chair of the International Society for Cell and Gene Therapy (ISCT) Presidential Task Force on the Use of Unproven and/or Unethical Cell and Gene Therapy.

Stem cell therapy involves the conversion of stem cells into specific types of cells, such as heart or blood cells. These cells are then transplanted into a patient to promote healing.

While there are companies that carefully develop cell-based treatments following established regulatory and ethical standards, there has also been an explosion of businesses since the mid-2000s that advertise directly to consumers and evade regulations to provide unsafe and ineffective treatments, he says.

These businesses operate in gray regulatory areas, frequently branding stem cell therapies as medical interventions rather than therapeutic drugs to avoid the need for U.S. Food and Drug Administration (FDA) approval, Ikonomou says, adding that according to published research, there are more than 1,000 of these unsafe businesses in the U.S.

They offer purported stem cell therapies for nearly every condition imaginable, from diabetes and autism to Alzheimers disease. There are also reports of people suffering physical harm including blindness and death from unsafe stem cell interventions, such as drawing and reinjecting patients with their own fat cells, he says.

Im not surprised that a lot of these businesses went into COVID treatments, says Ikonomou. They went where the money is and took advantage of peoples fears.

The treatments range in price from a few thousand to tens of thousands of dollars, and often patients are encouraged to receive the expensive infusions every few months. Many people go into severe debt to acquire these ineffective treatments, he says.

This year, the FDA has issued several letters to offending businesses, including those advertising cell therapies for COVID-19, says Ikonomou. The Federal Trade Commission has also cracked down on misleading advertising from stem cell therapy clinics, he says.

However, many of these clinics are small and difficult to track. Patient prudence is key to avoiding harmful interventions, he says.

Ikonomou shares a list of steps the public can take to ensure a stem cell therapy is safe, proven and ethical.

Ikonomou also urges patients to share any questions they have with their physicians, who often are the gatekeepers for medical treatment. His best advice to patients: If something sounds too good to be true, it probably isnt true.

For information on safe and ethical cell therapies, visit the ISCT website.

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Unproven 'stem cell' therapies for COVID-19 pose harm to public, says UB expert - UB Now: News and views for UB faculty and staff - University at...

The first patient to undergo a bone marrow transplant in the UAE has told of how the stem cell treatment saved his life.

Abdullah Muhammad had blood cancer diagnosed in 2018 after he began to vomit blood.

Doctors said the health of the 49-year-old electrician from Pakistan had begun to rapidly deteriorate and his only option was to undergo an expensive bone marrow transplant.

I didnt have money to get a transplant and the UAE sponsored my treatment.

"I am happy to be first one to undergo this transplant in the UAE. It wouldnt have been able to afford it in Pakistan, said Mr Muhammad, whose family live in Khanewal District. The father of four received his transplant on July 18 at Sheikh Khalifa Medical City, with the involvement of Abu Dhabi Stem Cell Centre, which is also developing a treatment for Covid-19 patients.

I didnt have money to get a transplant and the UAE sponsored my treatment. I am happy to be first one to undergo this transplant in the UAE

Abdullah Nazir Ahmad Muhammad

The transplant procedure will bring hope to cancer patients in the UAE, who can now seek treatment closer to home.

Known as regenerative medicine, stem cell therapy promotes the repair of abnormal or injured tissue.

Doctors can manipulate the cells into the type the patient needs and inject them where repair is necessary. The cells can be taken from a matching donor or harvested from the patient, treated, and then reintroduced to the body.

The UAE has begun to harness stem cell therapy in recent months to fight the coronavirus, but this was the first time it was used in a transplant in the Emirates.

In Mr Muhammads case, stem cells were harvested from his own blood and were injected back into him after he underwent a short course of chemotherapy. This is called an autologous bone marrow transplant.

Dr Fatima Al Kaabi, executive director of Abu Dhabi Bone Marrow Transplant Programme, said the treatment was a milestone for the UAE.

Most of these cases travel abroad so, in the near future, we will be self-sufficient and efficient to take care of our own with the highest calibre of medical care and international standards," she said.

Dr Al Kaabi said that to harvest the cells, Mr Muhammad was injected with a stimulant that prompted the stem cells to leave his bone marrow and enter his bloodstream.

His blood was drawn using a machine similar to one used in kidney dialysis to separate the plasma containing the stem cells from the blood.

The plasma was safely stored while Mr Muhammad underwent chemotherapy to wipe out the bone marrow and give way to the new cells, she said.

Mr Muhammad was kept in a sterile area for 10 to 15 days to prevent him from catching infections until the stem cells were returned to his body. The reintroduction of cells to his body took about 20 minutes.

After the successful autologous bone marrow transplant, Abu Dhabi Stem Cells Centre aims to begin carrying out transplants from related donors.

For now only we are doing autologous transplants, where the patient and donor are the same person, but in the near future we will not only do related transplantation but more complicated ones where donors are not related to the patient, said Dr Yendry Ventura, general manager of the stem cell centre and director of Abu Dhabi Bone Marrow Transplant Programme.

Sheikh Khalifa Medical City and the Department of Health Abu Dhabi are working on a stem cell donor registry.

The next transplant is scheduled for a few weeks' time, with the centre aiming to perform 10 transplants before the related transplant programme. It also plans to begin a transplant programme for children.

We want to provide the people of UAE a programme that is not only comparable with any other programme in the rest of the world but also offers customised and personalised treatment, Dr Ventura said.

Mr Muhammad, who has been in hospital since July 8, will be released in a few days and plans to return to Pakistan in October to see his family.

Updated: July 29, 2020 03:10 PM

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UAE's first bone marrow transplant patient tells of life-saving treatment - The National

Be the Match: Every three minutes, someone is diagnosed with a life-threatening blood cancer or blood disease, such as leukemia, lymphoma or sickle cell disease, and you might be able to help.

For those thousands, a cure exists, though about 70% of patients dont find it within their own families.

Thats why the College of the Canyons Biology Club is partnering with Be The Match to host a drive-in community registry event, where the community is invited to register to be a potential match for someone in need of a life-saving stem cell transplant through a simple swab test.

The science of stem cells is incredible, and it can actually cure over 70 different life-threatening diseases, said Christine Mantilla, member engagement, enrollment and experience specialist at Be The Match. A small population of stem cells from a donor can regenerate an entire bodys worth of bone marrow.

Biology Club President Brian Estarella-Murphy has been on the registry for two years, eagerly awaiting the day hes a match.

For Estarella-Murphy, its personal, as he has had two close friends with blood disorders and has seen firsthand the struggle of some of these patients.

I interned and shadowed at a hospital and a clinic, he added. Ive seen many patients come in that are on their last chance of life, and Id love to be able to give someone that possibility of living their full life without having to worry.

Being involved with the organization has actually changed his career aspirations, motivating him to go into the research side of medicine.

Im a cellular biology major, and the type of research that they do on these swab tests are exactly what I want to do in the future, he added. I want to pick apart these cells to see how I can help advance medicine (to create) life-saving treatment.

So upon taking up presidency of the club, Estarella-Murphy knew the first thing he wanted to do was partner with Be The Match.

Not only has the current health crisis been an extremely difficult time for those in need of transplants, as they are immunocompromised, but without community registry events, less have been joining.

This is an action that people can take in 10 minutes for free that can directly save someones life, Mantilla said.

Those interested in being a possible match, can drive up to the event from the safety of their car, where volunteers assist them in registering and administering the 10-second swab test on the inner cheeks while following all health and safety measures.

Were just doing a quick, little swab, and then we send your data out, and we do some research to see if youre possibly even a match, Estarella-Murphy said. Our goal out of this event is to sign up as many individuals as possible, so that through further research, we can then match them to patients awaiting dire transfusions.

Only about 1-in-430 U.S. registry members go on to donate bone marrow or peripheral blood stem cells to a patient.

Thats 0.2% and just emphasizes the need for as many people as possible to be available on the registry because it is so challenging to find that match, Mantilla said, adding that not everyone has an equal chance at finding a match, as ethnic heritage plays a significant role. Right now, the registry is overwhelmingly white. Its a health disparity that is pretty serious as far as equity of access to treatment.

The most common way to harvest stem cells is through a peripheral blood stem cell donation, involving a specialized blood draw, where stem cells are processed from your blood before the blood is returned to the donor.

Your stem cells are a renewable resource, so the donors stem cells actually regenerate in about four to six weeks, Mantilla added.

The event is scheduled 2-6 p.m. Aug. 14, with the location to be determined. For more information, visit biologyclubcoc.org/bethematch. To join the registry, visit join.bethematch.org/COCBio or text COCBio to 61474.

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It's Time to 'Be The Match,' Help Save a Life - SCVNEWS.com

DEAR DOCTOR:Whats the connection between the novel coronavirus and the thymus gland? A friend of ours who is a doctor says its probably what keeps young kids from getting so sick. Ive never even heard of the thymus. What does it have to do with coronavirus?

DEAR READER: From the earliest days of the novel coronavirus pandemic, the data revealed a puzzling disparity. Older adults were at increased risk of grave illness when infected with the virus, but children seemed to have a certain level of protection. And while it has since become clear that children can indeed become seriously ill if they become infected, they do so at far lower rates than adults. The reasons for this are still being investigated, but some researchers have recently suggested the role of the thymus gland as a possible factor.

If you place your finger at the notch at the top of your breast bone and draw a vertical line downward a few inches, youve traced the location of your thymus. Its made up of two roughly triangular lobes, which sit behind the breastbone and between the lungs. The thymus has several functions, but perhaps its most important role is to help produce the cells that will become T-lymphocytes, or T-cells. (The T stands for thymus-derived.) These are white blood cells that protect the body from bacteria, fungi, viruses and other pathogens.

T-cells, which are the ninjas of the immune system, start out in the bone marrow as stem cells. The immature stem cells exit the marrow, move through the blood and enter a specific region of the thymus. There, they undergo a complex process that teaches them how to recognize a wide range of potentially dangerous and deadly invaders. As T-cells, their job is to circulate throughout the body and, when they encounter the molecular signature of the pathogen theyve been trained to recognize, to attack. T-cells also activate other immune cells, produce proteins known as cytokines and have a role in regulating immune response.

The thymus is unique in that it reaches maturity in utero and is at its largest and most active in children. Starting at puberty, it gradually becomes less active, and the glandular tissue begins to shrink. This continues throughout a persons life. By the time someone has reached their mid-60s, the thymus is largely inactive. By their mid-70s, the gland has been mostly replaced with fat. This decrease in thymus function is believed to be one of the reasons that, in their later years, older adults become more susceptible to disease and infection.

Emerging research into COVID-19 has shown a marked decrease in the number of T-cells in some gravely ill patients. Scientists are now asking whether age-related thymus decline, which means T-cells arent quickly replaced, may play a role in the severity of illness seen in older adults. The flip side of this is whether, due to their robust production of T-cells, childrens immune systems are able to stay one step ahead of the novel coronavirus. Its only a working theory, but it shows promise, and research into how this may affect and inform treatment continues.

Eve Glazier, M.D., MBA, is an internist and associate professor of medicine at UCLA Health. Elizabeth Ko, M.D., is an internist and assistant professor of medicine at UCLA Health. Send your questions to askthedoctors@mednet.ucla.edu, or write: Ask the Doctors, c/o UCLA Health Sciences Media Relations, 10880 Wilshire Blvd., Suite 1450, Los Angeles, CA, 90024. Owing to the volume of mail, personal replies cannot be provided.

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Thymus may play role in severity of coronavirus - Cumberland Times-News