Category Archives: Stem Cell Treatment


Emerging potential of PTL nanoformulations in tumor therapy | DDDT – Dove Medical Press

Introduction

As a medical herb, Feverfew (Tanacetum parthenium) is conventionally used in Europe to treat fever, inflammation, migraines, rheumatoid arthritis, and menstrual irregularities. Parthenolide (PTL, Figure 1) is a sesquiterpene lactone found in feverfew, which is currently considered to be responsible for the herbs therapeutical potential.1 Initially, conventional extraction methods using chloroform and petroleum ether were performed to extract PTL; various extraction methods have been developed, such as high-performance liquid chromatography (HPLC), Soxhlet extraction, supercritical fluid extraction (SFE) and microwave-assisted extraction (MAE).1,2

Figure 1 Chemical structure of PTL and DMAPT.

The anti-cancer property of PTL was firstly validated in 1973. Furthermore, its patent application for tumor suppression was approved in 2005.3 Additionally, the in vitro and in vivo antitumor potential of PTL in multiple cancer types has been confirmed by numerous researches, which mainly resulted from its cytotoxicity to the bulk population of cancer cells as well as from selectively targeting cancer stem cells (CSCs); it is a subpopulation currently believed to be responsible for chemotherapy resistance and tumor relapse.310 Further studies revealed a series of direct PTL targets [p65, IB kinase (IKK), focal adhesion kinase 1 (FAK1), and others] that indirectly affect signaling pathways, which account for cell cycle arrest, apoptosis induction, metastasis suppression, redox imbalance, and epigenetic regulation involved in PTLs antitumor properties.3,4,11,12 The potential utility of PTL as radio-sensitization agent and complementary therapy against various cancers has also been widely studied and summarized.1315 As reviewed by Malgorzata et al, PTL has been combined with various anticancer agents, such as tubulin-directed agents, anthracyclines, antimetabolites, histone deacetylase inhibitors, mTOR inhibitors, and inducers of reactive oxygen species (ROS).14

Despite its deciphered anticancer potential and mechanisms of action in pre-clinical experiments, the clinical application of PTL remains hindered because of some disadvantages, including weak aqueous solubility, low oral bioavailability, and relative instability under chemical and physiological conditions.16,17 As a result, various methods for synthesizing PTL derivatives to yield compounds with better hydrophilicity and improved potency have been proposed.4,6,18 Dimethylamino parthenolide (DMAPT, Figure 1), a representative among hydrophilic PTL analogues, showed improved water solubility and oral bioavailability. Thus, it has advanced into the first phase of a clinical trial for the treatment of acute myeloid leukemia (AML).19

Nanomedicine is a rapidly developing field that exploits nanoparticles (NPs) to facilitate the diagnosis and treatment of a wide range of diseases. Nanoparticles applied in nanomedicine generally refer to a type of colloidal drug delivery system, which comprises particles with a size range from 10 to 200 nm in diameter.20 By far, diverse types of nanoparticles have been developed as drug carriers, including but not limited to liposomes, polymeric micelles, carbon nanotubes, mesoporous silica nanoparticles, metal-based nanoparticles, and dendrimers. Moreover, these can be made of diverse materials, including lipids, phospholipids, polymers, proteins, and inorganic materials.21,22

Compared with free drug counterparts, nanoparticles entrapment has displayed distinct advantages, such as improved bioavailability, prolonged circulation time, and ease of functionalization by surface modification. Furthermore, the enhanced permeability and retention (EPR) effect caused by the large amount of leaky vascularization and impaired lymphatic drainage at the tumor site enables non-targeted nanoparticles to accumulate in tumor tissues.23 Some of these nanoparticles have been approved as cancer therapeutics by the Food and Drug Administration.21 Besides the chemical modification of PTL for property improvement, the development of nanoscale drug delivery systems offers another promising strategy to overcome the poor water solubility and bioavailability of PTL as well as to determine its efficient and selective delivery to tumor tissues; the latter of which has not been summarized as compared to the extensively reviewed bioactivities and combination treatments of PTL. Therefore, we focused on the key antitumor mechanisms of PTL as well as its efficiency in being formulated as a nanoparticle delivery system.

Current evidence demonstrates that the antitumor mechanism of PTL is multifactorial and complex, due to the high electrophilic reactivity of -methylene--lactone present in PTL, thereby resulting in the alkylation of various proteins. A number of PTL targets have thus been identified and summarized.4 Furthermore, newer potential targets of PTL, such as USP7 and EGFR, continue to be reported,2427 thereby giving rise to the regulatory effect of PTL on various signaling pathways (Figure 2) and cellular processes (Figure 3) responsible for proliferation, cell cycle regulation, stemness, cell death, angiogenesis and metastasis.3,4 Thus, it is not surprising that PTL displays diverse anticancer effects, including abrogated cell viability and angiogenesis, cell cycle arrest, cell death induction, and decrease in stemness, invasiveness, and chemoresistance (Table 1).

Figure 2 Selected signaling pathways regulated by PTL. The key components and signal transduction cascade reactions in NF-B signaling, Wnt/-catenin signaling, JAK/STAT signaling, FAK1 signaling, and p53 signaling were depicted, and the nodes targeted by PTL were labeled.

Notes: Represents the inhibitory effect, and Represents the activation effect.

Figure 3 Pivotal cellular processes affected by PTL. Schematic diagram showing the formation of microtentacle and major antioxidant machineries were plotted. The targets regulated by PTL were marked.

Note: Represents the accumulation of ROS.

As shown in Figure 2, several signaling pathways closely related to tumorigenesis and progression were suppressed by PTL; among these, the prominent NF-B signaling pathway was the first to be inhibited. Mechanism investigation illustrated that PTL inhibited NF-B signaling by alkylating cysteine 38 in p65 and cysteine 179 in IKK.28,29 Additionally, a later study predicted that tumor necrosis factor receptor-associated factor 6 (TRAF6) might have been a novel target involved in the PTL-associated inhibition of NF-B.30 Moreover, the role of PTL in preventing NF-B activation contributed to the suppression of hypoxia-inducible factor-1 (HIF-1) signaling.31 Recent studies have elucidated that PTL inhibited Wnt/-catenin signaling by targeting ubiquitin specific peptidase 7 (USP7) and ribosomal protein L10 (RPL10), a deubiquitination enzyme stabilizing -catenin and a ribosomal protein related to the synthesis of the transcriptional regulator 4/lymphoid enhancer binding factor 1 (TCF4/LEF1), respectively.24,32 Furthermore, FAK1 and janus kinase/signal transducer and activator of transcription (JAK/STAT) signaling were found to be impaired by PTL by covalently modifying specific cysteine molecules.33,34

Apart from inhibiting the aforementioned signaling pathways, PTL also activated p53 functions by promoting the ubiquitination of E3 ligase murine double minute 2 (MDM2) in an ataxia-telangiectasia mutated (ATM)-dependent manner, thus leading to either cell cycle arrest or apoptosis.35 Specially, USP7 was reported to interact with and stabilize MDM2 and p65.36,37 Thus, the inhibitory effect of PTL on USP7 might be a further step for activating p53 and inhibiting NF-B. Additionally, the activation of c-Jun N-terminal kinase (JNK) by PTL enhanced the sensitivity of human cancer cells to tumor necrosis factor- (TNF-) or tumor necrosis factor-related apoptosis-inducing ligand (TRAIL).38,39

To date, PTL has been found to exert antitumor effects on several cellular processes, including redox balance and epigenetic modulation; the targets involved in these two processes have also been revealed (Figure 3). Several studies have indicated that PTL disrupted intracellular redox homeostasis by depleting glutathione (GSH) and inhibiting its metabolic enzymes, including glutamate-cysteine ligase catalytic subunit (GCLC), thioredoxin reductase 1/2 (TrxR1/2), and GSH peroxidase 1 (GPX1), thus leading to increased ROS level.4042 Moreover, ROS enhancement by PTL seemed to elicit different forms of cell death, such as necrosis, apoptosis, and autophagic cell death, depending on the tumor cell type.40,41,43 Meanwhile, the action of PTL on ROS partially accounts for its distinctive ability to selectively induce cell death in cancer cells, while sparing the equivalent normal cells. This is due to the fact that oxidative stress in cancer cells is more frequently elevated than that in normal cells. Thus, additional ROS produced by PTL may promote tumor cell death, whereas normal cells may maintain redox homeostasis by adaptive antioxidant responses.44 Moreover, the epigenetic targets of PTL consist of various enzymes, including histone deacetylase 1 (HDAC1), DNA methyltransferase 1 (DNMT1), and lysine methyltransferase 5C (KMT5C); these regulate the transcription of various genes, such as p21 and high in normal-1 (HIN-1).4547 In addition, PTL disrupted the cyclical detyrosination/tyrosination of tubulin by inhibiting tubulin carboxypeptidase (TCP), which led to a reduced frequency of microtentacles and suppressed tumor cell reattachment to endothelial layers (Figure 2).48,49

Interactions between signaling pathways and cellular processes may be affected by PTL. For instance, the stimulated generation of ROS by PTL was able to induce autophagy and cause NF-kB downregulation.40,43 Furthermore, NF-B inhibition contributed to epigenetic regulation,47 thereby further complicating the antitumor mechanism of PTL.

To understand the pharmacological effects of PTL, in vitro studies on its efficacy, pharmacological activities, and potential molecular mechanisms in a variety of cancer cells as well as available in vivo models since 2019 are tabulated in Table 1, thus further extending and qualifying existing reviews of its biological activities.3,4,50 As shown in Table 1, PTL modulation toward the aforementioned signaling pathways and cellular processes exerts multiple pharmacological effects against a myriad of tumor cell types.

The antiproliferative activity of PTL was detected in almost all studies; the results indicated that PTL exhibited half-maximal inhibitory concentration (IC50) between the range of 2.525 M for most tumor cells listed, thus showcasing its cytotoxicity to different cancer cells.5159 Cell cycle arrest, induction of cell death, and changes in related proteins were further detected, thus further supporting the cytotoxic potential of PTL. Overall, PTL was shown to induce different effects depending on the cell type, which can be illustrated by its ability to induce cell cycle arrest at different phases as well as several types of cell death in various cancer cells. For example, PTL arrested uveal melanoma cells in the G1 phase by upregulating p21 and downregulating cyclin D1, which are two G1 phase cell cycle regulatory proteins.60 On the other hand, PTL decreased the expression of survivin promoting G2/M cell cycle transition, thereby triggering G2/M cell cycle arrest in glioblastoma cells.61 Apoptosis, necrosis, and autophagy are the three forms of cell death caused by PTL;62 among which, apoptosis is the most studied. It is well known that apoptosis is elicited by two distinct pathways, the extrinsic and mitochondria-mediated intrinsic pathways; it culminates in the activation of caspases, which function as the main apoptotic effectors.63

Numerous studies demonstrated that PTL treatment could induce extrinsic or intrinsic apoptosis in tumor cells by inhibiting the activities of the NF-B, STAT3, Wnt and JNK signaling pathways, activating the p53 signaling pathway, regulating the Bcl-2 family members, and generating ROS.24,62,6471 Furthermore, PTL-induced cell death of breast cancer and multiple myeloma cells was dramatically attenuated by co-treatment with the pan-caspase inhibitor, Q-VD-OPh or Z-VAD-fmk, thus indicating that caspases are involved in PTL-induced apoptotic cell death; it also concurs the presence of other forms of cell death.33,72 Indeed, a large number of studies reported that PTL was capable of inducing autophagic or necrotic cell death.50,62 For instance, PTL mediated cell death through ROS-mediated autophagy in human osteosarcoma (Saos-2 and MG-63) and triple-negative breast cancer (MDA-MB-231) cells.40,43 In particular, PTL was capable of simultaneously inducing mixed forms of cell death, as evidenced by observations of PTL-induced apoptosis and autophagy in Hela and HepG2 cells as well as apoptosis and necrosis in HL60 cells.7375 Additionally, the role of autophagy in cell death involves the fact that its inhibition significantly blocked PTL-induced apoptosis in pancreatic cancer cells,76 but potentiated PTL-induced apoptosis in human breast cancer cells.77

Although previous studies have elucidated the selective targeting effect of PTL on CSCs from primary or sensitive cancer cells,3 a recent study by Yi et al suggested that PTL also effectively eliminated leukemia stem cells (LSCs) from adriamycin (ADM)-resistant K562 cells (K562/ADM) by suppressing aberrantly activated NF-B.64 In addition, NF-B inactivation by PTL sensitized gastric tumor and esophageal squamous cell carcinoma (ESCC) cells to chemotherapeutic drugs including ADM and cisplatin.78,79 PTL exerted its anti-angiogenic effects by inhibiting the NF-B-mediated VEGF expression in ESCC cells.79 Moreover, several studies have pointed out that PTL inhibits migration, invasion, and metastasis, which benefits from its abilities to regulate epithelial-to-mesenchymal transition (EMT) and to inhibit FAK1, TCP, and STAT3.33,8083

In short, the ultimate outcomes resulting from the affected signaling pathways and cellular processes by PTL include, but are not limited to, impaired cell proliferation and angiogenesis, induction of cell death, and reduced stemness, invasiveness, and chemoresistance. This has been confirmed by a large body of research, thus providing a sufficient basis for studies investigating and developing a wide range of PTL nanoformulations for various cancer therapies.

To the best of our knowledge, several types of PTL nanocarriers, including liposomes,8489 polymeric micelles,9096 nanocrystals,97 PLGA nanoparticles,98 and nanographene,99 have been synthesized to deliver PTL and ameliorate its anti-cancer efficacy. Whether PTL is co-encapsulated into nanoparticles with other drugs or small molecules and whether the nanoparticles are modified for targeted therapy, these studies can be classified into three types. Furthermore, the types, materials, and properties of nanocarriers mentioned in these studies, as well as the in vitro and in vivo models employed to evaluate the anticancer effects of nanoformulations, are separately tabulated in Tables 24.

Table 2 Unmodified Nanoparticles Solely Incorporated with PTL

Table 3 Undecorated Nanovectors Encapsulated PTL and Other Agents

Table 4 Targeted Nanocarriers Encapsulated PTL with or without Other Agents

Baranello et al synthesized different types of micelles formed from amphiphilic diblock copolymers of PSMA-b-PS or PSMA-b-PBA; PTL was successfully loaded into these micelles. However, PTL exhibited the greatest loading efficiency and capacity in PSMA100-b-PS258 micelles, thereby indicating that the composition and hydrophobic core chemistry of micelles were significant parameters for optimization.90 Although subsequent studies suggested that PTL-loaded PSMA100-b-PS258 micelles did not exhibit better cytotoxic ability toward MV4-11 cells than free PTL, it protected sequestered PTL from damage by both cells and deactivating chemicals, such as GSH.91 Similarly, the application of stealthy liposomes and micelles fabricated by F127 or biodegradable PTL-PTMC as nanocarriers of PTL did not appear to efficiently increase its cytotoxicity against MCF-7, MDA-MB-231, and patient T-lineage acute lymphoblastic leukemia (T-ALL) or B-cell precursor acute lymphoblastic leukemia (BCP-ALL) cells.89,92,93 However, the combination of stealthy liposomal PTL slightly sensitized the antitumor effects of stealthy liposomes loaded with vinorelbine.89

Unlike the nanocarriers mentioned above, the carboxyl-functionalized nanographene (fGn) as well as the PTL-nanocrystal delivery system showed improved antiproliferation activities in comparison with individual PTL. The IC50 of PTL and PTL-fGn for Panc-1 cells were 39 and 9.5 M respectively, whereas the IC50 of PTL for HepG2 cells decreased from 50.891 M to 33.618 M when delivered with nanocrystals.97,99 Interestingly, there was no significant difference in cytotoxic activity between DMAPT and DMAPT-fGn; this inefficacy may be due to the lack of a hydrophobic interaction between DMAPT and fGn.99 Thus, whether incorporation of DMAPT into other nanovectors could enhance its cytotoxicity is worth exploring. Furthermore, the in vitro and in vivo combination of PTL-nanocrystals and sorafenib achieved remarkable synergistic anti-cancer effects, as reflected by the MTT, wound-healing and HepG2 xenograft assays.97

Given the molecular complexity of cancer, combination therapy has attracted tremendous attention because of its ability to increase drug efficacy, improve drug resistance, and reduce systemic toxicity. Kanwaldeep et al constructed a paclitaxel and PTL co-delivery system of PEG2000-DSPE/vitamin E-TPGS mixed micelles, which retained a high encapsulation efficiency (>95%) and chemical stability over a storage period of 45 days. Furthermore, co-encapsulation of these two drugs significantly suppressed the viability of sensitive and taxol-resistant A549 cells compared to their free drug counterparts in solutions and single drug-loaded micelles.94 In addition, as a component of these mixed micelles, it is noteworthy that vitamin E-TPGS aided in not only maintaining high encapsulation efficiency due to its bulky structure and large surface area, but also enhanced chemosensitization by inhibiting P-glycoprotein (P-gp) efflux.94,100 Recently, a liposome system loaded with betulinic acid, PTL, honokiol and ginsenoside Rh2 displayed in vitro and in vivo antitumor activity comparable to cisplatin, the first-line therapy for lung cancer. In addition, this cocktail liposome therapy circumvented obvious kidney damage induced by cisplatin, as revealed by hematoxylin and eosin (H&E) staining. It also did not cause any significant damage to other major organs, including the heart, liver, spleen, and lungs, thereby indicating that this cocktail is a safer alternative for lung cancer treatment.84

To exploit the advances in photothermal therapy, Jin Xin and co-workers constructed thermosensitive liposomes (TSLs), in which PTL and the photosensitizer indocyanine green (ICG) were co-loaded. ICG converts light energy into heat energy upon near-infrared light irradiation. Compared to groups treated with paclitaxel, a combination of free drugs with or without laser, or PTL-ICG TSLs without laser, groups treated with PTL-ICG TSLs with laser exhibited lower cell viability, higher ROS induction and apoptosis, and better in vivo anti-cancer effects. According to these results, the two benefits of heat energy released by ICG under near-infrared radiation at the tumor location were validated. On the one hand, the heat-promoted phase transition of these liposomes enhanced their fluidity and permeability, thus allowing loaded drugs to effectively diffuse to tumor tissues at high concentrations. On the other hand, the maximum temperature of tumor tissues treated with PTL-ICG TSLs with laser reached 47.4C 2.68C, which led to irreversible damage and further synergized with the oxidative stress of PTL.85

To further improve drug delivery and selective targeting toward cancer cells, targeted nano-encapsulation of PTL with or without other agents was developed according to the characteristics of specific types of tumor cells and their microenvironment or the suction of the magnetic field. CD44 is a pivotal receptor involved in myelopoiesis; its specific variant isoforms have been reported to be overexpressed in AML cells, thus indicating that CD44 can serve as a promising receptor for targeted delivery of anti-AML drugs.101,102 As a result, an intervention was developed by encapsulating PTL into PLGA nanoparticles conjugated with antiCD44 with higher tumor targeting efficiency than PLGA-PTL nanoparticles. Although the cytotoxic abilities of PLGA-antiCD44-PTL and PLGA-PTL nanoparticles were not compared, PLGA-antiCD44-PTL nanoparticles exhibited stronger cytotoxicity than free PTL.98

Leukemia stem cells (LSCs) within the bone marrow (BM) microenvironment are thought to be the primary mediators of relapse and chemotherapy resistance in AML. Furthermore, E-selectin expressed in the BM endothelium provides a feasible approach for targeted BM delivery. In view of the remarkable capability of PTL to eradicate cancer stem cells including LSCs, a multistage vector system (MSV) was developed by entrapping PTL in mPEG-PLA micelles coated with protective degradable porous silicon particles and an E-selectin thioaptamer. In contrast to the negligible therapeutic efficacy of PTL-loaded micelles, MSV-PTL significantly reduced the tumor burden of patient-derived AML xenografts, concurrent with the inhibition of NF-B and activation of -H2AX; this supports the effectiveness of the MSV system for targeted BM delivery. Moreover, the decreased level of secondary xenotransplants implied that the directed delivery of PTL to the BM using the MSV system led to the elimination of LSCs.95

The tLyP-1 peptide has been verified to possess cell-penetrating ability and tumor-targeting capacity, which are derived from its C-terminal structure and affinity to the neuropilin-1 receptor overexpressed in several kinds of cancer cells (eg glioma and lung cancer).103 In which tLyP-1-modified liposomes entrapped in PTL and ginsenoside compound K (CK) were synthesized, the level of ROS and induced apoptosis of A549 cells in vitro significantly increased. Besides, PTL/CK tLyP-1 liposomes exhibited greater anticancer efficacy than the combined administration of these two compounds in A549 tumor-bearing mice.87 In addition, Ran et al developed PTL-loaded PEG-PLA micelles decorated with a Y-shaped DWVAP peptide, which could guide micelles across multiple biological barriers and ultimately target glioma and its associated stem cells. Moreover, combined therapy of PTL-loaded, DWVAP-modified PEG-PLA micelles with temozolomide or DWVAP-modified PEG-PLA micelles loaded with paclitaxel achieved outstanding anti-glioma efficacy, according to the KaplanMeier survival analysis.96

Recently, by virtue of the magnetic field, magnetic nanoparticles (MNPs) were chosen to modify the surface of liposomes (lips) loaded with PTL by Gao and co-workers.86,88 In one study, PTL-ICG-Lips, similar to PTL-ICG TSLs mentioned above, were coated with MNPs. In terms of in vitro heating efficiency and drug release, there was no significant difference between the [emailprotected] group treated only with laser and [emailprotected] group treated with laser plus magnet, which were stronger than non-laser irradiation treated groups. These results suggested the dominant role of photothermal conversion mediated by ICG in these processes. However, the presence of the magnetic field increased the heating rate and percentage of drug release at an earlier stage. In addition, cells treated with [emailprotected] (magnet plus laser) exhibited the highest cellular uptake. Consistently, in vivo studies also showed that the [emailprotected] (magnet plus laser) group displayed the fastest heating rate, highest temperature, and highest intratumoral PTL concentration.86 These findings indicated that the magnetic field could enrich the magnetic liposomes in the irradiated area, thus further enhancing the efficiency of photothermal conversion and facilitating the release and uptake of PTL.

Another multifunctional delivery system generated by Gao et al was the encapsulation of PTL and glucose oxidase (GOD) into nanomagnetic liposomes coated with chitosan, named [emailprotected] The addition of chitosan endowed [emailprotected] with the capability to release drug at a slightly acidic pH, which was characteristic of the tumor microenvironment. As a result, this increased the targeting ability of the system together with the magnetic field. By consuming glucose, GOD in this system lowers the pH and generates H2O2 as well as starves the cancer cells to death. A lower pH further promotes drug release; H2O2 can be subsequently catalyzed by iron ions in MNPs to produce hydroxyl radicals (OH), a noxious ROS. Meanwhile, PTL protects OH from scavenging by depleting GSH,40 which amplifies the intracellular oxidative stress and thus leads to cell apoptosis. Under an extra magnetic field, the [emailprotected] demonstrated prominent antitumor effects in vitro and in vivo through the integration of chemo-, chemodynamic, starvation and magnetic-targeting therapies.88

As discussed above, the incorporation of PTL into nanocarriers results in increased solubility, cellular uptake and stability, prolonged circulation time, and enhanced accumulation at tumor sites. Therefore, the majority of nanocarriers encapsulated with PTL, especially when combined with other agents such as photosensitizers, anticancer drugs, and MNPs, demonstrated higher anticancer efficiency than free PTL. These mechanisms are reflected by better antiproliferative activities, more effective induction of apoptosis, higher suppression rate of migration, and xenograft tumor growth. Histological examination demonstrated the low toxicity of this novel therapeutic agent.

In addition, a variety of naturally occurring sesquiterpene lactones structurally related to PTL with anticancer efficiency, as represented by micheliolide (MCL, Figure 4), melampomagnolide B (MMB, Figure 4), and costunolide (COS, Figure 4) have been reported.104106 Similar with PTL, most of current researches focus on structural modification of these compounds to improve their antitumor effects, stability, and sustainable release.107111 Several studies started to investigate the incorporation of MCL analogs and COS into nanoparticles.112,113 Bone-targeted PSMA-b-PS NPs entrapped with triazole MCL analog exhibited excellent serum stability and significantly reduced LSC burden in leukemic mice.112 Another study demonstrated that COS and COS-NPs, in combination with doxorubicin (DOX), stimulated the activity of caspase-3 and induced apoptosis of HCT116 and MDA-MB-231-Luc. They also suppressed the tumor growth of HCT116 and MDA-MB-231-Luc implants in nude mice. There was no significant difference in the anti-tumor activity of COS and COS-NPs; the authors deemed that this may have been due to the dose-selective approach for determining the optimal anti-cancer activity for both COS and Nano-COS. Thus, dose-response relationship will be investigated in future studies.113 In addition, Niu et al constructed pH-responsive mesoporous silica nanoparticles (MSNs) loaded with COS, which increased stability and enhanced anti-fibrotic effect of pure COS.114 In short, above-mentioned results implied that nanoparticles entrapped with sesquiterpene lactones show great promise in the treatment of cancer and other diseases.

Figure 4 Chemical structure of MCL, MMB and COS.

Studies from the past decades have validated the great potential of PTL as an anticancer agent with extremely intricate bioactivities. However, poor aqueous solubility, instability, low bioavailability, and drug-targeting property of PTL limit its in vivo anticancer efficacy and clinical application.86 As such, the development of nanoparticle-based platforms has been utilized in multiple biomedical fields, including hydrophobic drug delivery, which undoubtedly provides a promising strategic improvement.115 Indeed, several natural product-derived anticancer nanodrugs, including nanoparticle albuminbound (NAB)-paclitaxel and liposomal vincristine, have been used in clinical practice.21 However, no clinical trials have been reported for nanocomposites of PTL and its structurally related sesquiterpene lactones. Furthermore, despite multiple in vitro and in vivo experiments that have reported the benefits of nanoparticle-based formulations of other natural anti-cancer drugs in the treatment of various cancer types, including quercetin, curcumin, resveratrol, and andrographolide, only a few clinical trials have been performed, thus, suggesting that the investigation of these nanocarriers is still in its relative infancy.116118 Further optimization can be performed because the efficiency of nanoparticles can be influenced by many parameters, such as nanocarrier types, compositions (eg materials, ligand modification, co-encapsulated agents), and physical properties (eg size, shape, surface charge).21 As such, PTL and other natural product-based nanoformulations with improved properties will undoubtedly emerge; clinical trials need to be encouraged to further validate the security and therapeutic efficiency of these nanoparticles for cancer. Finally, PTL has been recently predicted to be a possible agent for the treatment of other diseases, such as Hutchinson-Gilford Progeria syndrome and hypertrophic cardiomyopathy.119,120 Thus, the therapeutic value of PTL nanoparticles for these diseases deserves further study.

The authors acknowledge funding from the Young Scientists Fund of National Science Foundation of China (No. 81803587), the Project of Science and Technology of Yunnan Province (2019FD054), PhD Start-up Fund for Tao An from Qilu University of Technology (No. 81110573), and Industry-University Cooperation Collaborative Education Project of Ministry of Education (202102403007).

The authors declare that they have no conflicts of interest in this work.

1. Vgh K, Alberti , Riethmller E, Tth A, Bni S, Kry . Supercritical fluid extraction and convergence chromatographic determination of parthenolide in Tanacetum parthenium L.: experimental design, modeling and optimization. J Supercrit Fluids. 2014;95:8491. doi:10.1016/j.supflu.2014.07.029

2. Alam P, Siddiqui NA, Rehman MT, et al. Box-Behnken Design (BBD)-Based optimization of microwave-assisted extraction of parthenolide from the stems of tarconanthus camphoratus and cytotoxic analysis. Molecules. 2021;26(7):1876. doi:10.3390/molecules26071876

3. Ghantous A, Sinjab A, Herceg Z, Darwiche N. Parthenolide: from plant shoots to cancer roots. Drug Discov Today. 2013;18(1718):894905. doi:10.1016/j.drudis.2013.05.005

4. Freund RRA, Gobrecht P, Fischer D, Arndt HD. Advances in chemistry and bioactivity of parthenolide. Nat Prod Rep. 2020;37(4):541565. doi:10.1039/C9NP00049F

5. Siveen KS, Uddin S, Mohammad RM. Targeting acute myeloid leukemia stem cell signaling by natural products. Mol Cancer. 2017;16(1):13. doi:10.1186/s12943-016-0571-x

6. Araujo TG, Vecchi L, Lima P, et al. Parthenolide and its analogues: a new potential strategy for the treatment of triple-negative breast tumors. Curr Med Chem. 2020;27(39):66286642. doi:10.2174/0929867326666190816230121

7. Dandawate PR, Subramaniam D, Jensen RA, Anant S. Targeting cancer stem cells and signaling pathways by phytochemicals: novel approach for breast cancer therapy. Semin Cancer Biol. 2016;4041:192208. doi:10.1016/j.semcancer.2016.09.001

8. Ren Y, Yu J, Kinghorn AD. Development of anticancer agents from plant-derived sesquiterpene lactones. Curr Med Chem. 2016;23(23):23972420. doi:10.2174/0929867323666160510123255

9. Mathema VB, Koh YS, Thakuri BC, Sillanpaa M. Parthenolide, a sesquiterpene lactone, expresses multiple anti-cancer and anti-inflammatory activities. Inflammation. 2012;35(2):560565. doi:10.1007/s10753-011-9346-0

10. Ghantous A, Gali-Muhtasib H, Vuorela H, Saliba NA, Darwiche N. What made sesquiterpene lactones reach cancer clinical trials? Drug Discov Today. 2010;15(1516):668678. doi:10.1016/j.drudis.2010.06.002

11. Koprowska K, Czyz M. [Molecular mechanisms of parthenolides action: old drug with a new face]. Postepy Hig Med Dosw. 2010;64:100114. Norwegian.

12. Kreuger MR, Grootjans S, Biavatti MW, Vandenabeele P, DHerde K. Sesquiterpene lactones as drugs with multiple targets in cancer treatment: focus on parthenolide. Anticancer Drugs. 2012;23(9):883896. doi:10.1097/CAD.0b013e328356cad9

13. Wyrebska A, Gach K, Janecka A. Combined effect of parthenolide and various anti-cancer drugs or anticancer candidate substances on malignant cells in vitro and in vivo. Mini Rev Med Chem. 2014;14(3):222228. doi:10.2174/1389557514666140219113509

14. Sztiller-Sikorska M, Czyz M. Parthenolide as cooperating agent for anti-cancer treatment of various malignancies. Pharmaceuticals. 2020;13(8):194. doi:10.3390/ph13080194

15. Pordanjani SM, Hosseinimehr SJ. The role of NF-kB inhibitors in cell response to radiation. Curr Med Chem. 2016;23(34):39513963. doi:10.2174/0929867323666160824162718

16. Lesiak K, Koprowska K, Zalesna I, Nejc D, Duchler M, Czyz M. Parthenolide, a sesquiterpene lactone from the medical herb feverfew, shows anticancer activity against human melanoma cells in vitro. Melanoma Res. 2010;20(1):2134. doi:10.1097/CMR.0b013e328333bbe4

17. Nasim S, Crooks PA. Antileukemic activity of aminoparthenolide analogs. Bioorg Med Chem Lett. 2008;18(14):38703873. doi:10.1016/j.bmcl.2008.06.050

18. Ren Y, Kinghorn AD. Development of potential antitumor agents from the scaffolds of plant-derived terpenoid lactones. J Med Chem. 2020;63(24):1541015448. doi:10.1021/acs.jmedchem.0c01449

19. Guzman ML, Rossi RM, Neelakantan S, et al. An orally bioavailable parthenolide analog selectively eradicates acute myelogenous leukemia stem and progenitor cells. Blood. 2007;110(13):44274435. doi:10.1182/blood-2007-05-090621

20. Wu D, Si M, Xue HY, Wong HL. Nanomedicine applications in the treatment of breast cancer: current state of the art. Int J Nanomedicine. 2017;12:58795892. doi:10.2147/IJN.S123437

21. Zhang J, Tang H, Liu Z, Chen B. Effects of major parameters of nanoparticles on their physical and chemical properties and recent application of nanodrug delivery system in targeted chemotherapy. Int J Nanomedicine. 2017;12:84838493. doi:10.2147/IJN.S148359

22. Jasinski DL, Li H, Guo P. The effect of size and shape of RNA nanoparticles on biodistribution. Mol Ther. 2018;26(3):784792. doi:10.1016/j.ymthe.2017.12.018

23. Torchilin V. Tumor delivery of macromolecular drugs based on the EPR effect. Adv Drug Deliv Rev. 2011;63(3):131135. doi:10.1016/j.addr.2010.03.011

24. Li X, Kong L, Yang Q, et al. Parthenolide inhibits ubiquitin-specific peptidase 7 (USP7), Wnt signaling, and colorectal cancer cell growth. J Biol Chem. 2020;295(11):35763589. doi:10.1074/jbc.RA119.011396

25. Zhang S, Ju X, Yang Q, et al. USP47 maintains the stemness of colorectal cancer cells and is inhibited by parthenolide. Biochem Biophys Res Commun. 2021;562:2128. doi:10.1016/j.bbrc.2021.05.017

26. Ai XY, Zhang H, Gao SY, et al. Sesquiterpene binding Gly-Leu-Ser/Lys-co-adaptation pocket to inhibit lung cancer cell epithelial-mesenchymal transition. Oncotarget. 2017;8(41):7019270203. doi:10.18632/oncotarget.19599

27. Li X, Huang R, Li M, et al. Parthenolide inhibits the growth of non-small cell lung cancer by targeting epidermal growth factor receptor. Cancer Cell Int. 2020;20(1):561. doi:10.1186/s12935-020-01658-1

28. Garcia-Pineres AJ, Castro V, Mora G, et al. Cysteine 38 in p65/NF-kappaB plays a crucial role in DNA binding inhibition by sesquiterpene lactones. J Biol Chem. 2001;276(43):3971339720. doi:10.1074/jbc.M101985200

29. Kwok BH, Koh B, Ndubuisi MI, Elofsson M, Crews CM. The anti-inflammatory natural product parthenolide from the medicinal herb Feverfew directly binds to and inhibits IkappaB kinase. Chem Biol. 2001;8(8):759766. doi:10.1016/S1074-5521(01)00049-7

30. Kong FC, Zhang JQ, Zeng C, et al. Inhibitory effects of parthenolide on the activity of NF-kappaB in multiple myeloma via targeting TRAF6. J Huazhong Univ Sci Technolog Med Sci. 2015;35(3):343349. doi:10.1007/s11596-015-1435-0

31. Kim SL, Park YR, Lee ST, Kim SW. Parthenolide suppresses hypoxia-inducible factor-1alpha signaling and hypoxia induced epithelial-mesenchymal transition in colorectal cancer. Int J Oncol. 2017;51(6):18091820. doi:10.3892/ijo.2017.4166

32. Zhu X, Yuan C, Tian C, et al. The plant sesquiterpene lactone parthenolide inhibits Wnt/beta-catenin signaling by blocking synthesis of the transcriptional regulators TCF4/LEF1. J Biol Chem. 2018;293(14):53355344. doi:10.1074/jbc.M117.819300

33. Berdan CA, Ho R, Lehtola HS, et al. Parthenolide covalently targets and inhibits focal adhesion kinase in breast cancer cells. Cell Chem Biol. 2019;26(7):10271035 e1022. doi:10.1016/j.chembiol.2019.03.016

34. Liu M, Xiao C, Sun M, Tan M, Hu L, Yu Q. Parthenolide inhibits STAT3 signaling by covalently targeting janus kinases. Molecules. 2018;23(6):1478.

35. Gopal YN, Chanchorn E, Van Dyke MW. Parthenolide promotes the ubiquitination of MDM2 and activates p53 cellular functions. Mol Cancer Ther. 2009;8(3):552562. doi:10.1158/1535-7163.MCT-08-0661

36. Tavana O, Gu W. Modulation of the p53/MDM2 interplay by HAUSP inhibitors. J Mol Cell Biol. 2017;9(1):4552. doi:10.1093/jmcb/mjw049

37. Colleran A, Collins PE, OCarroll C, et al. Deubiquitination of NF-kappaB by Ubiquitin-Specific Protease-7 promotes transcription. Proc Natl Acad Sci U S A. 2013;110(2):618623. doi:10.1073/pnas.1208446110

38. Zhang S, Lin ZN, Yang CF, Shi X, Ong CN, Shen HM. Suppressed NF-kappaB and sustained JNK activation contribute to the sensitization effect of parthenolide to TNF-alpha-induced apoptosis in human cancer cells. Carcinogenesis. 2004;25(11):21912199. doi:10.1093/carcin/bgh234

39. Nakshatri H, Rice SE, Bhat-Nakshatri P. Antitumor agent parthenolide reverses resistance of breast cancer cells to tumor necrosis factor-related apoptosis-inducing ligand through sustained activation of c-Jun N-terminal kinase. Oncogene. 2004;23(44):73307344. doi:10.1038/sj.onc.1207995

40. DAnneo A, Carlisi D, Lauricella M, et al. Parthenolide generates reactive oxygen species and autophagy in MDA-MB231 cells. A soluble parthenolide analogue inhibits tumour growth and metastasis in a xenograft model of breast cancer. Cell Death Dis. 2013;4:e891. doi:10.1038/cddis.2013.415

41. Duan D, Zhang J, Yao J, Liu Y, Fang J. Targeting thioredoxin reductase by parthenolide contributes to inducing apoptosis of hela cells. J Biol Chem. 2016;291(19):1002110031. doi:10.1074/jbc.M115.700591

42. Pei S, Minhajuddin M, Callahan KP, et al. Targeting aberrant glutathione metabolism to eradicate human acute myelogenous leukemia cells. J Biol Chem. 2013;288(47):3354233558. doi:10.1074/jbc.M113.511170

43. Yang C, Yang QO, Kong QJ, Yuan W, Ou Yang YP. Parthenolide induces reactive oxygen species-mediated autophagic cell death in human osteosarcoma cells. Cell Physiol Biochem. 2016;40(12):146154. doi:10.1159/000452532

44. Xu Y, Fang F, Miriyala S, et al. KEAP1 is a redox sensitive target that arbitrates the opposing radiosensitive effects of parthenolide in normal and cancer cells. Cancer Res. 2013;73(14):44064417. doi:10.1158/0008-5472.CAN-12-4297

45. Liu Z, Liu S, Xie Z, et al. Modulation of DNA methylation by a sesquiterpene lactone parthenolide. J Pharmacol Exp Ther. 2009;329(2):505514. doi:10.1124/jpet.108.147934

46. Gopal YN, Arora TS, Van Dyke MW. Parthenolide specifically depletes histone deacetylase 1 protein and induces cell death through ataxia telangiectasia mutated. Chem Biol. 2007;14(7):813823. doi:10.1016/j.chembiol.2007.06.007

47. Nakshatri H, Appaiah HN, Anjanappa M, et al. NF-kappaB-dependent and -independent epigenetic modulation using the novel anti-cancer agent DMAPT. Cell Death Dis. 2015;6:e1608. doi:10.1038/cddis.2014.569

48. Fonrose X, Ausseil F, Soleilhac E, et al. Parthenolide inhibits tubulin carboxypeptidase activity. Cancer Res. 2007;67(7):33713378. doi:10.1158/0008-5472.CAN-06-3732

49. Whipple RA, Vitolo MI, Boggs AE, Charpentier MS, Thompson K, Martin SS. Parthenolide and costunolide reduce microtentacles and tumor cell attachment by selectively targeting detyrosinated tubulin independent from NF-kappaB inhibition. Breast Cancer Res. 2013;15(5):R83. doi:10.1186/bcr3477

50. Carlisi D, Lauricella M, DAnneo A, et al. Parthenolide and its soluble analogues: multitasking compounds with antitumor properties. Biomedicines. 2022;10(2):514. doi:10.3390/biomedicines10020514

51. Marino S, Bishop RT, Carrasco G, Logan JG, Li B, Idris AI. Pharmacological inhibition of NFkappaB reduces prostate cancer related osteoclastogenesis in vitro and osteolysis ex vivo. Calcif Tissue Int. 2019;105(2):193204. doi:10.1007/s00223-019-00538-9

52. Ge W, Liu Z, Sun Y, et al. Design and synthesis of parthenolide-SAHA hybrids for intervention of drug-resistant acute myeloid leukemia. Bioorg Chem. 2019;87:699713. doi:10.1016/j.bioorg.2019.03.056

53. Mehri S, Mohammadi S, Nikbakht M, Sahmani M, Zahedpanah M. Osteopontin siRNA does not confer resistance to toxic effects of parthenolide in Jurkat cells. Exp Oncol. 2020;42(3):188191. doi:10.32471/exp-oncology.2312-8852.vol-42-no-3.15180

54. Ge W, Hao X, Han F, et al. Synthesis and structure-activity relationship studies of parthenolide derivatives as potential anti-triple negative breast cancer agents. Eur J Med Chem. 2019;166:445469. doi:10.1016/j.ejmech.2019.01.058

55. De Blasio A, Di Fiore R, Pratelli G, et al. A loop involving NRF2, miR-29b-1-5p and AKT, regulates cell fate of MDA-MB-231 triple-negative breast cancer cells. J Cell Physiol. 2020;235(2):629637. doi:10.1002/jcp.29062

56. Dawood M, Ooko E, Efferth T. Collateral sensitivity of parthenolide via NF-kappaB and HIF-alpha inhibition and epigenetic changes in drug-resistant cancer cell lines. Front Pharmacol. 2019;10:542. doi:10.3389/fphar.2019.00542

57. Ding Y, Li S, Ge W, et al. Design and synthesis of parthenolide and 5-fluorouracil conjugates as potential anticancer agents against drug resistant hepatocellular carcinoma. Eur J Med Chem. 2019;183:111706. doi:10.1016/j.ejmech.2019.111706

58. Sun L, Yuan W, Wen G, et al. Parthenolide inhibits human lung cancer cell growth by modulating the IGF1R/PI3K/Akt signaling pathway. Oncol Rep. 2020;44(3):11841193. doi:10.3892/or.2020.7649

59. Luo Q, Wu X, Chang W, et al. ARID1A hypermethylation disrupts transcriptional homeostasis to promote squamous cell carcinoma progression. Cancer Res. 2020;80(3):406417. doi:10.1158/0008-5472.CAN-18-2446

60. Che ST, Bie L, Li X, Qi H, Yu P, Zuo L. Parthenolide inhibits the proliferation and induces the apoptosis of human uveal melanoma cells. Int J Ophthalmol. 2019;12(10):15311538. doi:10.18240/ijo.2019.10.03

Continue reading here:
Emerging potential of PTL nanoformulations in tumor therapy | DDDT - Dove Medical Press

Podcast: Has the First 150-Year-Old Already Been Born – Leaps

One is that there are some people that are naturally resistant to heart attack and have lifelong, low levels of LDL, the cardiologist says. Second, there are some genes that can be switched off that lead to very low LDL cholesterol, and individuals with those genes switched off are resistant to heart attacks.

Kathiresan and his team formed a hypothesis in 2016 that if they could develop a medicine that mimics the natural protection that some people enjoy, then they might identify a powerful new way to treat and ultimately prevent heart attacks. They launched Verve in 2018 with the goal of creating a one-time therapy that would permanently lower LDL and eliminate heart attacks caused by high LDL.

The medication is targeted specifically for patients who have a genetic form of high cholesterol known as heterozygous familial hypercholesterolemia, or FH, caused by expression of a gene called PCSK9. Verve also plans to develop a program to silence a gene called ANGPTL3 for patients with FH and possibly those with or at risk of atherosclerotic cardiovascular disease.

FH causes cholesterol to be high from birth, reaching levels of 200 to 300 milligrams per deciliter. Suggested normal levels are around 100 to 129 mg/dl, and anything above 130 mg/dl is considered high. Patients with cardiovascular disease usually are asked to aim for under 70 mg/dl, but many still have unacceptably high LDL despite taking oral medications such as statins. They are more likely to have heart attacks in their 30s, 40s and 50s, and require lifelong LDL control.

The goal for drug treatments for high LDL, Kathiresan says, is to reduce LDL as low as possible for as long as possible. Physicians and researchers also know that a sizeable portion of these patients eventually start to lose their commitment to taking their statins and other LDL-controlling medications regularly.

If you ask 100 patients one year after their heart attack what fraction are still taking their cholesterol-lowering medications, its less than half, says Kathiresan. So imagine a future where somebody gets a one-time treatment at the time of their heart attack or before as a preventive measure. Its right in front of us, and its something that Verve is looking to do.

In late 2020, Verve completed primate testing with monkeys that had genetically high cholesterol, using a one-time intravenous injection of VERVE-101. It reduced the monkeys LDL by 60 percent and, 18 months later, remains at that level. Kathiresan expects the LDL to stay low for the rest of their lives.

Verves gene editing medication is packaged in a lipid nanoparticle to serve as the delivery mechanism into the liver when infused intravenously. The drug is absorbed and makes its way into the nucleus of the liver cells.

Verves program targeting PCSK9 uses precise, single base, pair base editing, Kathiresan says, meaning it doesn't cut DNA like CRISPR gene editing systems do. Instead, it changes one base, or letter, in the genome to a different one without affecting the letters around it. Comparing it to a pencil and eraser, he explains that the medication erases out a letter A and makes it a letter G in the A, C, G and T code in DNA.

By making that simple change from A to G, the medication switches off the PCSK9 gene, automatically lowering LDL cholesterol.

Once the DNA change is made, all the cells in the liver will have that single A to G change made, Kathiresan says. Then the liver cells divide and give rise to future liver cells, but every time the cell divides that change, the new G is carried forward.

Additionally, Verve is pursuing its second gene editing program to eliminate ANGPTL3, a gene that raises both LDL and blood triglycerides. In 2010, Kathiresan's research team learned that people who had that gene completely switched off had LDL and triglyceride levels of about 20 and were very healthy with no heart attacks. The goal of Verves medication will be to switch off that gene, too, as an option for additional LDL or triglyceride lowering.

Success with our first drug, VERVE-101, will give us more confidence to move forward with our second drug, Kathiresan says. And it opens up this general idea of making [genomic] spelling changes in the liver to treat other diseases.

The approach is less ethically concerning than other gene editing technologies because it applies somatic editing that affects only the individual patient, whereas germline editing in the patients sperm or egg, or in an embryo, gets passed on to children. Additionally, gene editing therapies receive the same comprehensive amount of testing for side effects as any other medicine.

We need to continue to advance our approach and tools to make sure that we have the absolute maximum ability to detect off-target effects, says Euan Ashley, professor of medicine and genetics at Stanford University and founding director of its Center for Inherited Cardiovascular Disease. Ashley and his colleagues at Stanfords Clinical Genomics Program and beyond are increasingly excited about the promise of gene editing.

We can offer precision diagnostics, so increasingly were able to define the disease at a much deeper level using molecular tools and sequencing, he continues. We also have this immense power of reading the genome, but were really on the verge of taking advantage of the power that we now have to potentially correct some of the variants that we find on a genome that contribute to disease.

He adds that while the gene editing medicines in development to correct genomes are ahead of the delivery mechanisms needed to get them into the body, particularly the heart and brain, hes optimistic that those arent too far behind.

It will probably take a few more years before those next generation tools start to get into clinical trials, says Ashley, whose book, The Genome Odyssey, was published last year. The medications might be the sexier part of the research, but if you cant get it into the right place at the right time in the right dose and not get it to the places you dont want it to go, then that tool is not of much use.

Medical experts consider knocking out the PCSK9 gene in patients with the fairly common genetic disorder of familial hypercholesterolemia roughly one in 250 people a potentially safe approach to gene editing and an effective means of significantly lowering their LDL cholesterol.

Nurse Erin McGlennon has an Implantable Cardioverter Defibrillator and takes medications, but she is also hopeful that a gene editing medication will be developed in the near future.

Erin McGlennon

Mary McGowan, MD, chief medical officer for The Family Heart Foundation in Pasadena, CA, sees the tremendous potential for VERVE-101 and believes patients should be encouraged by the fact that this kind of research is occurring and how much Verve has accomplished in a relatively short time. However, she offers one caveat, since even a 60 percent reduction in LDL wont completely eliminate the need to reduce the remaining amount of LDL.

This technology is very exciting, she said, but we want to stress to our patients with familial hypercholesterolemia that we know from our published research that most people require several therapies to get their LDL down., whether that be in primary prevention less than 100 mg/dl or secondary prevention less than 70 mg/dl, So Verves medication would be an add-on therapy for most patients.

Dr. Kathiresan concurs: We expect our medicine to lower LDL cholesterol by about 60 percent and that our patients will be on background oral medications, including statins that lower LDL cholesterol.

Several leading research centers are investigating gene editing treatments for other types of cardiovascular diseases. Elizabeth McNally, Elizabeth Ward Professor and Director at the Center for Genetic Medicine at Northwestern Universitys Feinberg School of Medicine, pursues advanced genetic correction in neuromuscular diseases such as Duchenne muscular dystrophy and spinal muscular atrophy. A cardiologist, she and her colleagues know these diseases frequently have cardiac complications.

Even though the field is driven by neuromuscular specialists, its the first therapies in patients with neuromuscular diseases that are also expected to make genetic corrections in the heart, she says. Its almost like an afterthought that were potentially fixing the heart, too.

Another limitation McGowan sees is that too many healthcare providers are not yet familiar with how to test patients to determine whether or not they carry genetic mutations that need to be corrected. We need to get more genetic testing done, she says. For example, thats the case with hypertrophic cardiomyopathy, where a lot of the people who probably carry that diagnosis and have never been genetically identified at a time when genetic testing has never been easier.

One patient who has been diagnosed with hypertrophic cardiomyopathy also happens to be a nurse working in research at Genentech Pharmaceutical, now a member of the Roche Group, in South San Francisco. To treat the disease, Erin McGlennon, RN, has an Implantable Cardioverter Defibrillator and takes medications, but she is also hopeful that a gene editing medication will be developed in the near future.

With my condition, the septum muscles are just growing thicker, so Im on medicine to keep my heart from having dangerous rhythms, says McGlennon of the disease that carries a low risk of sudden cardiac death. So, the possibility of having a treatment option that can significantly improve my day-to-day functioning would be a major breakthrough.

McGlennon has some control over cardiovascular destiny through at least one currently available technology: in vitro fertilization. Shes going through it to ensure that her children won't express the gene for hypertrophic cardiomyopathy.

Go here to see the original:
Podcast: Has the First 150-Year-Old Already Been Born - Leaps

Stem Cell Therapy Market: North America Leads the Market Owing to Rising Awareness among People – BioSpace

Albany NY, United States: Stem cells are found in all human beings, from the initial stages of human growth to the end of life. All stem cells are beneficial for medical research; however, each of the different kinds of stem cells has both limitations and promise. Embryonic stem cells that can be obtained from a very initial stage in human development have the prospect to develop all of the cell types in the human body. Adult stem cells are found in definite tissues in fully developed humans.

Read Report Overview - https://www.transparencymarketresearch.com/stem-cell-therapy-market.html

Stem cells are basic cells of all multicellular animals having the ability to differentiate into a wide range of adult cells. Totipotency and self-renewal are characteristics of stem cells. However, totipotency is seen in very early embryonic stem cells. The adult stem cells owes multipotency and difference flexibility which can be exploited for next generation therapeutic options. Recently, scientists have also recognized stem cells in the placenta and umbilical cord blood that can give rise to several types of blood cells.

Research for stem cells is being undertaken with the expectation of achieving major medical inventions. Scientists are attempting to develop therapies that replace or rebuild spoiled cells with the tissues generated from stem cells and offer hope to people suffering from diabetes, cancer, spinal-cord injuries, cardiovascular disease, and many other disorders.

Request Brochure of Report - https://www.transparencymarketresearch.com/sample/sample.php?flag=B&rep_id=28262

The stem cell therapy market is segmented on the basis of type, therapeutic applications, cell source, and geography. On the basis of type, the stem cell therapy market is categorized into allogeneic stem cell therapy and autologous stem cell therapy. Allogeneic stem cell therapy includes transferring the stem cells from a healthy person (the donor) to the patients body through high-intensity radiation or chemotherapy.

Allogeneic stem cell therapy is used to treat patients who do not respond fully to treatment, who have high risk of relapse, and relapse after prior successful treatment. Autologous stem cell therapy is a type of therapy that uses the person's own stem cells. These type of cells are collected earlier and returned in future.

Get COVID-19 Analysis on Stem Cell Therapy Market - https://www.transparencymarketresearch.com/sample/sample.php?flag=covid19&rep_id=28262

The use of stem cells is done to replace damaged cells by high doses of chemotherapy, and to treat the person's underlying disease. On the basis of therapeutic applications, the stem cell therapy market is segmented into cardiovascular diseases, wounds and injuries, musculoskeletal disorders, gastrointestinal diseases, surgeries, neurodegenerative disorders, and others. On the basis of cell source, stem cells therapy is segmented into bone marrow-derived mesenchyme stem cells, adipose tissue-derived mesenchyme stem cells, and cord blood or embryonic stem cells.

By geography, the market for stem cell therapy is segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America leads the stem cell therapy market owing to rising awareness among people, early treatment adoption, and new product innovations. Europe is the second leading market for stem cell therapy due to development and expansion of more efficient and advanced technologies.

Enquiry Before Buying - https://www.transparencymarketresearch.com/sample/sample.php?flag=EB&rep_id=28262

The Asia Pacific stem cell therapy market is also anticipated to grow at an increasing rate owing to increasing healthcare spending, adoption of western lifestyles, and growth in research and development. Asia Pacific is the fastest growing region for stem cell therapy as several players have invested in the development of new stem cell technologies. These factors are expected to drive the growth of the stem cell therapy market globally during the forecast period.

The major player in the stem cell therapy market are Regenexx, Takara Bio Company, Genea Biocells, PromoCell GmbH, CellGenix GmbH, Cellular Engineering Technologies, BIOTIME, INC., Astellas Pharma US, Inc., AlloSource, RTI Surgical, Inc., NuVasive, Inc., JCR Pharmaceuticals Co., Ltd., Holostem Terapie Avanzate S.r.l., PHARMICELL Co., Ltd, ANTEROGEN.CO., LTD., The Future of Biotechnology, and Osiris Therapeutics, Inc. Rising demand for advanced stem cell therapies will increase the competition between players in the stem cell therapy market.

Browse more Reports by Transparency Market Research:

Continuous Glucose Monitoring Market: Increase in incidence and prevalence rate of diabetes type 1 and type 2 across the globe is anticipated to boost the growth of the global market during the forecast period. Continuous glucose monitoring systems are real-time glucose sensing and monitoring devices that measure and display glucose levels of a patient at regular intervals.

Diabetes Management Market: The biggest driving factor for the market growth is the growing prevalence of both Type I as well as Type II diabetes. This condition coupled with considerable rise in the obese population across the globe is also helping the global diabetes management market to develop.

About Us

Transparency Market Research is a next-generation market intelligence provider, offering fact-based solutions to business leaders, consultants, and strategy professionals.

Our reports are single-point solutions for businesses to grow, evolve, and mature. Our real-time data collection methods along with ability to track more than one million high growth niche products are aligned with your aims. The detailed and proprietary statistical models used by our analysts offer insights for making right decision in the shortest span of time. For organizations that require specific but comprehensive information we offer customized solutions through ad hoc reports. These requests are delivered with the perfect combination of right sense of fact-oriented problem solving methodologies and leveraging existing data repositories.

TMR believes that unison of solutions for clients-specific problems with right methodology of research is the key to help enterprises reach right decision.

Contact

Rohit Bhisey Transparency Market Research State Tower, 90 State Street, Suite 700, Albany NY - 12207 United States USA - Canada Toll Free: 866-552-3453 Email: sales@transparencymarketresearch.com Website: https://www.transparencymarketresearch.com/

Read the rest here:
Stem Cell Therapy Market: North America Leads the Market Owing to Rising Awareness among People - BioSpace

TREEFROG THERAPEUTICS EXPANDS BOARD WITH NEW INDEPENDENT MEMBERS FROM THE CELL THERAPY INDUSTRY – BioSpace

Elsy Boglioli, former Chief Operating Officer at French cell therapy biotech Cellectis, founder and Chief Executive Officer of biotechnology consulting practice Bio-Up, joins as Chair of the Board

Melissa Carpenter, PhD, Chief Scientific Officer of US-based cell & gene therapy biotech ElevateBios Regenerative Medicine unit, joins as independent board member.

Bordeaux, France / April 14th, 2022 TreeFrog Therapeutics, a biotechnology company aimed at making safer, more efficient and more affordable cell therapies based on induced pluripotent stem cells (iPSCs), today announced the appointment of new independent members to its board: Elsy Boglioli, former Chief Operating Officer at Cellectis, Founder and Chief Executive Officer of healthtech consulting practice Bio-Up, and Melissa Carpenter, PhD, Chief Scientific Officer of the Regenerative Medicine business unit at ElevateBio, a technology-driven company focused on powering transformative cell and gene therapies. Elsy Boglioli will be the Chair of the Board.

Following our Series B, we welcomed new investors to our board: Bpifrance Large Venture, Leonard Green & Partners, and Bristol Myers Squibb. I am delighted to reinforce our organization with our new independent board members to guide our strategy and serve our ambition. Elsy, with her profound knowledge of the biotech industry, will chair our board, bringing a new dynamic to our governance and scale-up efforts. Melissa, a world-renowned expert in iPSCderived cell therapies, will reinforce our U.S. network and help us steer our programs toward clinical trials. Also, I would like to warmly thank our co-founder, Jean-Luc Treillou, for his work as a Chairman over the first years of the company. Frdric Desdouits, PhD, Chief Executive Officer, TreeFrog Therapeutics.

I am thrilled to join TreeFrog as the company advances its unique platform and enters its next phase of growth. Together, we aim to solve major challenges facing the development of cell therapies and craft transformative, scalable, high quality treatments that can benefit large patient pools. Melissa Carpenter, PhD, Chief Scientific Officer, ElevateBio, Regenerative Medicine

Elsy Boglioli, Founder and Chief Executive Officer, Bio-Up

Elsy Boglioli started her career in 2006 at the Boston Consulting Group, where she served as partner and managing director, leading the biotech business in Europe. In 2017, she joined the French cell therapy biotech company Cellectis as Executive Vice-President Strategy and Business Development and Chief Operating Officer, before founding the independent consulting practice Bio-Up. As part of her activities within Bio-Up, she was appointed to the boards of Gensight Biologics, OSE Immunotherapeutics, Metafora Biosystems, Inova, Kelindi and Womed. Graduate from the Ecole Polytechnique engineering school in Paris, France, Elsy Boglioli holds a masters degree in Economy and Management from the Pompeu Fabra University, Barcelona, Spain, and was trained in immuno-oncology at the Gustave Roussy Institute of Oncology, Paris, France.

Melissa Carpenter, PhD, Chief Scientific Officer, ElevateBio, Regenerative Medicine

Author of over 50 peer-reviewed articles and inventor of 34 issued patents, Melissa Carpenter has been pioneering the field of stem cell-derived cell therapies for the past 25 years. She started her career at CytoTherapeutics (became StemCells, Inc), where she derived human neural stem cells and successfully developed the media formulation used for scale-up of these cells. Carpenter then joined Californian cell therapy biotech Geron in 1998, as Director of Stem Cell Biology, managing research and preclinical programsin regenerative medicine, before taking an academic position as Principal Investigator at the Robarts Research Institute in Canada. In 2004, she joined Novocell (now Viacyte Inc.) as Vice-President of Research and Development, advancing a novel cell therapy program in diabetes, based on encapsulated human pluripotent stem cells. She then established her own consulting practice - Carpenter Group Consulting -, providing strategic, scientific and manufacturing guidance for the development of over 75 cell and gene therapies. In 2019, she was appointed Chief Scientific Officer of the Regenerative Medicine business unit of ElevateBio, a technology-driven company with integrated technology platforms, including gene editing, induced pluripotent stem cells (iPSCs) and protein, vector and cellular engineering, focused on the discovery, development and manufacturing of transformative cell and gene therapies. Initially trained in psychology, Melissa Carpenter completed a PhD in the Laboratory of Cellular and Molecular Neurobiology of the University of California, Irvine, in 1989.

About TreeFrog Therapeutics

TreeFrog Therapeutics is a French-based biotech company aiming to unlock access to cell therapies for millions of patients. TreeFrog Therapeutics is developing a pipeline of therapeutic candidates using proprietary C-StemTM technology, allowing for the mass production of induced pluripotent stem cells and their differentiation into ready-to-transplant microtissues with unprecedented scalability and cell quality. Bringing together over 80 biophysicists, cell biologists and bioproduction engineers, TreeFrog Therapeutics raised $82M over the past 3 years to advance its pipeline of stem cell-based therapies in the field of neurodegeneration, cardio-metabolic disorders, and immuno-oncology. The company is currently opening technological hubs in Boston, USA, and Kobe, Japan, to drive the adoption of C-StemTM and initiate co-development partnerships with leading academic, biotech and industry players in the field of cell therapy. http://www.treefrog.fr

Media Contact

Pierre-Emmanuel Gaultier

TreeFrog Therapeutics

+ 33 6 45 77 42 58

pierre@treefrog.fr

Read the rest here:
TREEFROG THERAPEUTICS EXPANDS BOARD WITH NEW INDEPENDENT MEMBERS FROM THE CELL THERAPY INDUSTRY - BioSpace

Versant-backed startup launches with plans to broaden cell therapy’s reach – BioPharma Dive

Stem cell transplants can effectively cure a wide range of diseases, from blood cancers to rare genetic disorders. They've been used for decades and are considered standard treatment for certain conditions.

But for a good number of patients, stem cell transplants are out of reach. Drug regimens used to prepare the body for a transplant are toxic and can cause serious side effects. The transplanted cells don't always "engraft," or take root in the bone marrow. Even when they do, patients' disease may linger or recur.

A biotech startup launching Wednesday with $50 million in funding hopes that, by combining cell, antibody and gene editing technologies, at least some of these problems can be overcome. Called Cimeio Therapeutics, the new company is led by a team of pharmaceutical industry veterans and an advisory board filled with scientific luminaries, including immunologist Jeffrey Bluestone and gene editing pioneer Fyodor Urnov.

Cimeio's approach involves "shielding" transplanted cells by genetically editing them in ways that allows paired immunotherapies to be safely used both before and after a transplant.

Thomas Fuchs

Courtesy of Cimeio Therapeutics

"We think that this can really unleash the power of hematopoietic stem cell transplant and make a lot more patients eligible for it," said Thomas Fuchs, Cimeio's CEO and a former Genentech executive.

The "shielding" technology used by Cimeio was developed in Switzerland at the laboratory of Lukas Jeker, a physician-scientist from Basel University Hospital who will join Cimeio as head of gene editing.

Jeker's lab discovered that protein receptors on the surface of cells could be genetically edited in such a way that prevented antibodies from binding to them, while leaving their function intact. In preclinical testing, these edits could cloak, or "shield," the cells from being depleted by antibody drugs and T cell therapies.

The work could have powerful implications for improving stem cell transplant and adoptive cell therapy, according to Fuchs.

Once a stem cell or T cell is shielded, a complementary immunotherapy could be used to either help ready patients for a transplant or to further treat disease afterwards, he said. "Maybe you could give a cycle or two of the paired immunotherapy, implant the shielded cells and then continue to administer the immunotherapy," he added.

If the shielding works as intended, Cimeio could develop treatments for conditioning that are more tolerable than the chemotherapy or radiation-based regimens currently in use. Shielding might also allow existing drugs that target cell proteins on healthy as well as diseased cells to be used more flexibly with transplants, such as to treat residual disease that lingers afterwards.

For example, Cimeio could engineer stem cells that are protected against binding via a protein called CD19 that's often the target for CAR-T therapies that treat lymphoma, but is also found on healthy B cells that help the immune system fight off threats.

"One benefit could be that you could prevent a lifetime of B cell depletion, which happens when you give a CAR-T," said Fuchs.

Alex Mayweg

Courtesy of Cimeio Therapeutics

Cimeio was built from Jeker's lab by Versant Ventures at the company's "Ridgeline" incubator in Basel, which has previously produced companies like Monte Rosa Therapeutics and Black Diamond Therapeutics. The initial $50 million Versant provided will fund Cimeio through next year, said Alex Mayweg, a managing director at the venture firm and a Cimeio board member. Additional investors will be brought on later this year or early next, Mayweg said.

Cimeio will need the money, as its research and development plans are expansive. The company has identified four drug candidates already and envisions a dozen more behind those, said Fuchs. Its research spans blood cancers, rare genetic diseases and autoimmune disorders.

In some cases, Cimeio will develop paired immunotherapies to go with the shielded cells. In others, it will use existing treatments. Three of the first four candidates involve protecting hematopoietic stem cells, while the fourth involves T cells. The company hopes to begin human testing next year.

Cimeio plans to choose gene editing technologies based on the type of alteration it needs to make to shield cells. "Rather than building up an internal editing capability," Mayweg said, "we wanted to stay as flexible as possible."

That might mean partnerships or alliances with other companies, some of which have reached out to Cimeio already, according to Mayweg.

Cimeio is aided by a group of scientific advisers notable for their work in areas the company is focusing on. Urnov, of the University of California, Berkeley, is well known for his research in gene editing using zinc finger nucleases and CRISPR. Bluestone previously led the Parker Institute for Cancer Immunotherapy and is CEO of the cell therapy-focused biotech Sonoma Biotherapeutics.

Suneet Agarwal, a co-program leader of the stem cell transplant center at Boston Children's Cancer and Blood Disorders Center, is also on the advisory board, while Cimeio has a research collaboration in place with Matthew Porteus, a gene editing specialist at Stanford University.

About 20 people currently work at Cimeio directly, a number Fuchs expects will grow as the company's research advances. Another 15 are currently supporting Cimeio from Versant's Ridgeline group.

Read more:
Versant-backed startup launches with plans to broaden cell therapy's reach - BioPharma Dive

Be wary of companies offering stem cell therapy for arthritis, joint pain, COVID, and more. Heres why – WATN – Local 24

ABC24 talked with Randy Hutchinson from the Better Business Bureau of the Mid-South about a lawsuit against one company and what consumers need to watch out for.

MEMPHIS, Tenn. The Federal Trade Commission and Georgia Attorney General have sued the founders of a company they claim has made unsubstantiated claims its stem cell therapy can treat arthritis, joint pain, and other orthopedic ailments.

The company is called Stem Cell Institute of America. It claimed its treatments are comparable to or better than surgery, steroid injections, and painkillers. The FTC said the company charged up to $5,000 per injection. It said a related company taught chiropractors and other healthcare professionals how to offer stem cell therapy.

ABC24 talked with Randy Hutchinson from the Better Business Bureau of the Mid-South about the claims and what consumers need to watch out for.

They're sometimes called the body's "master cells" because they develop into blood, brain, bones, and other organs.

Stem cells from bone marrow or blood are used to treat certain kinds of cancer and disorders of the blood and immune system. But other uses have not been properly studied and approved.

The FDA cites these potential risks from unproven treatments:

There could be safety risks even using a persons own stem cells.

The FTC has also looked into companies claiming their stem cell therapies can treat Parkinson's, multiple sclerosis, COVID, and a host of other ailments. They're sometimes referred to as "regenerative medicine."

Take miracle health care claims with a grain of salt.

Check out a company and treatment online using terms like "complaints," "scam" and "reviews."

Consult your own health care provider before using any product or treatment.

Read more:
Be wary of companies offering stem cell therapy for arthritis, joint pain, COVID, and more. Heres why - WATN - Local 24

CAR NK-Cell Therapy Is Quickly Growing in Immunotherapy – Targeted Oncology

The introduction of CAR engineering to adoptive cell therapy has led to immune effector cell treatments with improved cytotoxicity.

In hematologic oncology, advancements in chimeric antigen receptors (CARs) for T-cell therapy have led to new investigations and an emerging role for CAR-natural killer (NK) cell therapy. Here we review why CAR-NK cell therapy is an area of interest, how it differentiates from CAR T-cell therapy, its potential challenges, and the current stage of development of this form of treatment.

The introduction of CAR engineering to adoptive cell therapy has led to immune effector cell treatments with improved cytotoxicity. This has been a major advancement in treatment for many patients with relapsed or refractory hematologic malignancies.1

Autologous CAR T cells were used in pioneering therapies, and their efficacy has led to FDA approvals in hematologic malignancies.1 For example, tisagenlecleucel (Kymriah), a CAR T-cell therapy, was approved for patients with relapsed/refractory acute lymphoblastic leukemia based on trial results showing an overall response rate of 81%, with 60% of patients achieving complete remission.2,3

However, despite their clinical efficacy, CAR T cells have limitations.4 Not all patients are candidates for CAR T-cell therapy. For example, heavily pretreated patients may not have sufficient autologous T cells to achieve clinically relevant doses of CAR T cells.1,4 Also, generating individualized autologous CAR T-cell products for each patient can take weeks, which can lead to unacceptable treatment delays in patients with rapidly progressive disease. Furthermore, patients receiving CAR T cells are at risk of developing graft-vs-host disease (GVHD) even if human leukocyte antigen (HLA) matching between donor and recipient is performed.

This form of therapy has also yet to make significant headway in treating patients with solid tumors. Few patients with solid malignancies have achieved complete responses to date, potentially due to limited expansion or persistence of CAR T cells and the inability of these cells to penetrate solid tumors.5

Moreover, long-term persistence of CAR T cells may cause cytokine release syndrome (CRS) and immune effector cellassociated neurotoxicity syndrome (ICANS), which can be life threatening.1,4,6 Lastly, target antigen loss after therapy can render CAR T immune cells ineffective due to their dependence on antigens for efficacy.

However, interest in CAR NK-cell therapy has resulted in ongoing research.1,7

Peter Riedell, MD, assistant professor of medicine, Section of Hematology/Oncology, University of Chicago Medicine, provided his insights in an interview with Targeted Therapies in OncologyTM.

CAR T-cell therapies take time, and this can be problematic for patients [who] have more rapidly progressive disease and need therapy urgently, said Riedell in an interview comparing CAR T-cell therapy to CAR-NK cell therapy. Having a cellular therapy product which is off-the-shelf is very attractive as it means we may be able to treat patients sooner rather than later with this therapy, he added.

NK cells, which were discovered almost 50 years ago, can defend against tumors in most tissues without requiring detection of specific tumor antigens.7 Potent innate anti-tumor activity and favorable safety profile features have promoted interest in CAR-NK cell immunotherapy.

NK toxicity against tumor cells involves both innate and adaptive immunity.7,8 For example, unlike T cells, NK cells can kill tumor cells without expression of major histocompatibility complex (MHC) molecules. As a result, CAR-expressing NK cells can eradicate heterogeneous malignancies that CAR T cells cannot, due to CAR T-cell dependence on MHC expression.8 Furthermore, NK cells are able to perform CD16-mediated anti- body-dependent cell-mediated cytotoxicity, giving them an added killing mechanism.8,9

CAR-NK cells also differ from CAR T cells by having a shorter lifespan in the blood-stream. Less potential for long-term off-tumor toxicities in CAR-NK cells is theorized as a result.9 Healthy cells express CD19 as well as malignant cells. While having CAR T cells remain in the body for longer periods may be associated with continued clinical benefit in maintaining remissions, when CAR T cells remain in the body for prolonged periods, this may also lead to B-cell aplasia and hypogammaglobulinemia, explained Riedell.

Additionally, preclinical and phase 1/2 trials have shown that allogeneic CAR-NK- cell infusions decrease the risk of GVHD.1,9,10 This allows the expansion of NK-cell production beyond autologous cells or only 1 cell line source. Persistence of allogeneic CAR NK cells has been observed in patients for at least 1 year despite HLA mismatching.11,12

Furthermore, NK cells can be administered without a requirement for full HLA matching.10 This allows for the use of allogeneic sources for CAR NK cells, including healthy donors, umbilical cord blood units, or induced pluripotent stem cells, Riedell noted. Importantly, manufacturing failures and out-of-specification products can also be avoided with off-the-shelf therapy.10

This allows for the use of allogeneic sources for CAR NK cells, including healthy donors, umbilical cord blood units, or induced pluripotent stem cells. Products are able to come off the shelf without the need to navigate collection of patients T cells and await their engineering and manufacture, which can take weeks, Riedell noted. Importantly, manufacturing failures and out-of-specification products can also be avoided with off-the-shelf therapy.10

CAR-NK cell therapy may be associated with a lower incidence and severity of CRS and neurologic toxicity, which is another reason this therapy is being explored, explained Riedell. These less severe adverse events may be due to the release of milder cytokines such as granulocyte-macrophage colony-stimulating factor and interferon-.9 CAR T cells induce the release of more cytotoxic cytokines, such as interleukin-1 (IL-1) and IL-6, that are associated with CRS.

Despite the safety and promising clinical efficacy of unmodified allogeneic NK cells, several challenges to using CAR-NK cells have emerged from clinical trials.

While CAR-NK cell therapy has been shown to be technically feasible, there is overall limited data in regard to the efficacy and safety of this treatment approach. Given that these cellular therapy products are allogeneic, there is a concern for emergence of graft-vs-host disease, Riedell said. There are many current clinical trials being conducted that evaluate CAR-NK cell therapy and we eagerly await the results of these trials to better understand the impact of this treatment approach, he added.

Furthermore, NK cells have a short lifespan of only 1 to 2 weeks, and without cytokine support, infused cells do not persist in the donor, which restricts efficacy.13

It is unknown if responses seen with this treatment may be durable and associated with continued remissions or if this therapy may be better utilized to induce responses and remissions in patients and then consolidate those remissions with an allogeneic stem cell transplant, explained Riedell.

Techniques to enhance the stability of CAR-NK cells include incorporation of transgenes encoding exogenous cytokines, such as IL-15.11 However, exogenous cytokines have undesirable adverse effects and can promote the activation of other immune sub- sets, such as regulatory T cells, which may suppress the effector functions of NK cells.14

Another challenge with CAR-NK cells is that NK cells are limited in number and often require ex vivo expansion and actiation. NK cells represent a minor fraction of peripheral blood leukocytes, and thus the generation of sufficient numbers of NK cells remains a major challenge for adoptive immunotherapy.

NK-92 is an established NK cell line that can be used as a source of cells for CAR- NK therapies, representing an alternative to patient- or donor-derived NK cells. An advantage of this process is easier manufacture of off-the-shelf CAR-NK products; however, a drawback is that NK-92 cells are from a tumor cell line and have a potential tumorigenicity risk.15

Lastly, CAR NK approaches are limited by approaches to gene transfer in NK cells. Gene transduction may lead to random intergration of DNA into the target cell genome, and can encourage off-target effects, including the silencing of essential genes or expression of tumor suppressor genes.9

Viral transduction results in low levels of transgene expression in NK cells and adversely impacts their survival. Nonviral vectors have been explored and are considered safe alternatives, but their relative overall benefits remain unclear.11

Several phase 1 and 2 trials for CAR-NK therapy are ongoing, with some published results.

In a phase 1/2 study (NCT03056339), patients with B-cell lymphoid malignancies were administered cord bloodderived, HLA-mismatched, anti-CD19 CAR-NK cells.12 The cells were transduced with a retroviral vector that expressed genes encoding anti- CD19 CAR, IL-15, and inducible caspase 9 (safety switch).

Of 11 heavily pretreated patients with CD19-positive lymphoma or chronic lymphocytic leukemia (CLL), 8 had an objective response (73%) and 7 had complete remission (64%) without major toxic effects. There were no recorded events of CRS, neurotoxicity, hemophagocytic lymphohistiocy- tosis, or GVHD.

Myelotoxicity was observed, which the investigators attributed to the lymphodeplet-ing chemotherapy prior to infusion. Many responses were seen within 30 days of infusion. Also, the CAR-NK cells expanded and persisted for at least 12 months.

A second study, a phase 1 trial (NCT04245722), evaluated the safety and efficacy of FT596, a multi-antigentargeted, pluripotent stem cellderived, off-the-shelf, anti-CD19 CAR-NK cell therapy. In the study, 20 heavily pretreated patients with relapsed/ refractory B-cell lymphoma or CLL were treated with FT596, either alone or in combination with rituximab (Rituxan).

Responses were seen in 8 of 11 efficacy-evaluable patients, 7 of which were complete respons- es. No GVHD or ICANS was observed in any of the 20 treated patients, and only 2 cases of CRS were reported.16

Several other clinical trials of interest are ongoing. A phase 1 study (NCT05247957) evaluating NKG2D, a cord bloodderived CAR-NK therapy, in patients with relapsed/refractory acute myeloid leukemia is expected to conclude at the end of 2022.

Another phase 1 study (NCT04887012) of HLA haploidentical anti-CD19 CAR-NK cells in relapsed/refractory B-cell non-Hodgkin lymphoma is ongoing. Finally, an early phase 1 study (NCT05215015) of CAR-NK cells targeting CD33 in patients with acute myeloid leukemia is ongoing.

CAR-NK cell therapy will likely become much more common and an area of increasing research focus should we be able to gain a better understanding that this treatment approach is safe and efficacious, Riedell noted. Additional studies are needed in order to understand optimal CAR-NK cell constructs, the best antigens to target, and strategies to bolster CAR-NK cell manufacturing, storage, and delivery, he added.

REFERENCES:

1. Basar R, Daher M, Rezvani K. Next-generation cell therapies: the emerging role of CAR-NK cells. Blood Adv. 2020;4(22):5868-5876. doi: 10.1182/bloodadvances.2020002547

2. FDA approves tisagenlecleucel for B-cell ALL and tocilizumab for cytokine release syndrome. FDA. September 7, 2017. Accessed March 23, 2022. https://bit.ly/38mmisI

3. Maude SL, Laetsch TW, Buechner J, et al. Tisagenlecleucel in children and young adults with B-cell lymphoblastic leukemia. N Engl J Med. 2018;378(5):439-448. doi:10.1056/NEJMoa1709866

4. Sterner RC, Sterner RM. CAR-T cell therapy: current limitations and potential strategies. Blood Cancer J. 2021;11(4):69. doi:10.1038/s41408-021-00459-7

5. Wagner J, Wickman E, DeRenzo C, Gottschalk S. CAR T cell therapy for solid tumors: bright future or dark reality? Mol Ther. 2020;28(11):2320-2339. doi:10.1016/j.ymthe.2020.09.015

6. Morris EC, Neelapu SS, Giavridis T, Sadelain M. Cytokine release syndrome and associated neurotoxicity in cancer immunotherapy. Nat Rev Immunol. 2022;22(2):85-96. doi:10.1038/s41577-021-00547-6

7. Marofi F, Saleh MM, Rahman HS. CAR-engineered NK cells; a promising therapeutic option for treatment of hematological malignancies. Stem Cell Res Ther. 2021;12(1):374. doi:10.1186/s13287-021-02462-y

8. Farag SS, Caligiuri MA. Human natural killer cell development and biology. BloodRev. 2006;20(3):123-137.doi:10.1016/j.blre.2005.10.001

9. Xie G, Dong H, Liang Y, Ham JD, Rizwan R, Chen J. CAR-NK cells: A promising cellular immunotherapy for cancer. EBioMedicine. 2020;59:102975. doi:10.1016/j.ebiom.2020.102975

10. Lupo KB, MatosevicS. Natural killer cells as allogeneic effectors in adoptive cancer immunotherapy. Cancers. 2019;11(6):769.doi:10.3390/cancers11060769

11. Gong Y, Klein Wolterink RGJ, Wang J, Bos GMJ, GermeraadWTV. Chimeric antigen receptor natural killer (CAR-NK) cell design and engineering for cancer therapy. J Hematol Oncol. 2021;14(1):73. doi:10.1186/s13045-021-01083

12. Liu E, Marin D, Banerjee P. Use of CAR-transduced natural killer cells in CD19-positive lymphoid tumors. N Engl J Med. 2020;382(6):545-553. doi:10.1056/NEJMoa1910607

13. Malmberg KJ, Carlsten M, Bjrklund A, Sohlberg E, Bryceson YT, Ljunggren HG. Natural killer cell-mediated immunosurveillance of human cancer. Semin Immunol. 2017;31:20-29. doi:10.1016/j.smim.2017.08.002

14. Pedroza-Pacheco I, Madrigal A, Saudemont A,et al. Interaction between natural killer cells and regulatory T cells: perspectives for immunotherapy. Cell Mol Immunol. 2013;10(3):222-229.doi:10.1038/cmi.2013.2

15. Zhang C, Oberoi P, Oelsner S, et al. Chimeric antigen receptor-engineered NK-92 cells: an off-the-shelf cellular therapeutic for targeted elimination of cancer cells and induction of protective antitumor immunity. Front Immunol. 2017;8:533.doi:10.3389/fimmu.2017.00533

16. Bachanova V, Ghobadi A, Patel K, et al. Safety and efficacy of FT596, a first-in-class, multi-antigen targeted, off-the-shelf, iPSC-derived CD19 CAR NK cell therapy in relapsed/refractory b-cell lymphoma. Blood. 2021;138(suppl 1):823. doi:10.1182/blood-2021-151185

Excerpt from:
CAR NK-Cell Therapy Is Quickly Growing in Immunotherapy - Targeted Oncology

Global Dry Eye Products Markets Report 2022-2028: Ongoing Research and Growing Pipeline & Opportunities in Stem Cell Therapy -…

DUBLIN--(BUSINESS WIRE)--The "Dry Eye Products Market Forecast to 2028- COVID-19 Impact and Global Analysis - by Product; Type" report has been added to ResearchAndMarkets.com's offering.

The dry eye products market is projected to reach US$ 9,681.73 million by 2028 from US$ 6,837.26 million in 2021; it is estimated to grow at a CAGR of 5.1% from 2021 to 2028.

Factors such as rising incidences of dry eye and growing geriatric population are drive the growth of the market. However, the side effects caused due to eye drops hamper the market growth.

The Asia Pacific region is a highly affected region in the world as the outbreak of COVID-19 has come from China. Countries such as China, India, and South Korea have registered the highest number of COVID-19 positive patients. The countries have imposed lockdowns for several months.

Few countries such as South Korea and China have recovered from the pandemic. However, India is still struggling to fight against COVID-19. Therefore, there has been a great economic drop in the country, which is trying to recover. The fast recovery from the situation was seen due to the shift of focus of various companies operating in the ophthalmic division.

Companies have shifted their focus towards supplying reliefs to these countries as corporate social responsibility (CSR) activity. Also, governments are seeking help from the private sectors to contribute during the tough situation. Therefore, market players have strengthened their efforts to supply personal protective equipment.

For instance, in January 2020, Allergan plc has donated US$ 9,514 million (one million yen) to the Chinese Red Cross Foundation to provide medical masks, robes, gloves, eyewear, and other protective equipment for medical providers in Wuhan.

The incidence of dry eye is commonly seen in older people of age 65 and above. The number of geriatric population is increasing rapidly across the world. The older population is becoming a significant social transformation such as depending on the other for financial needs and wellness on the younger people in the present century.

Older people are significantly contributing to the development of the medical sector as it is helps by offering various innovative techniques to treat older people. Various healthcare companies design their products by focusing on geriatric population. As the degeneration process among older people is much faster, they are highly prevalent to dry eye conditions.

Therefore, the high prevalence of dry eye among older population is likely to promote manufacturers to introduce new dry eye products in the market, which, in turn, contributes to the growth of the market. For instance, according to the Department of Economic and Social Affairs, the United Nations report for 2019, there were 703 million individuals of age 65 years and above and is estimated to grow double to 1.5 billion by 2050.

Product Insights

The dry eye products market is segmented on the basis of product and type. Based on the product, the market is segmented into artificial tears, antibiotic drops, hormone drops, and others. The artificial tears segment held the largest share of the market in 2021, and the hormone drops segment is estimated to register the highest CAGR of 5.9% in the market during the forecast period.

World Health Organization, Royal Society of Medicine, Brazilian Research Association for Vision and Ophthalmology, Canadian Dry Eye Summit, International Dacryology Society, National Health Service, Royal Society of Medicine are among the primary and secondary sources referred to while preparing the report on the dry eye products market.

Key Market Dynamics

Market Driver

Market Restraints

Market Opportunities

Future Trends

Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/osg3y6

Read the original post:
Global Dry Eye Products Markets Report 2022-2028: Ongoing Research and Growing Pipeline & Opportunities in Stem Cell Therapy -...

O-Shot Therapy Raises Hope of Enhanced Sexual Experience in Genitally Mutilated Females – THISDAY Newspapers

Mary Nnah

There is hope for full conjugal experience with their partners for over 20 million Nigerian girls and women who have undergone female genital mutilation (FGM) with O-Shot therapy.

O-Shot, a regenerative medicine procedure that is done through Adult Stem Cell Therapy (non-invasive) or Platelet Rich Plasma (PRP) therapy (non-invasive) is a cosmetic procedure to enhance female sexual wellness.

According to Dr. David Ikudayisi, Medical Director of Glory Wellness and Regenerative Centre, Lekki, Lagos, O-Shot (aka orgasm shot) treatment, Involves injecting the Clitoris, Labia and G-spot with Platelets-substances in your blood that contains healing proteins called growth factor-extracted from your own blood.

Speaking during a recent webinar titled: Female Genital Mutilation (FGM): How to Enhance Your Sexual Experience Through Adult Stem Cell as part of the activities to mark the International Women Day, 2022, Dr. Ikudayisi explained: We use Platelet Rich Plasma (PRP) from your own blood, in addition to the use of Adult Stem Cells (such as Umbilical Blood Stem Cells, Fat-derived Stem Cells, Bone Marrow Stem Cells, Stem Cells Exosomes) and Shockwave Therapy to enhance female sexual wellness.

Ikudayisi, who is United States of America Board Certified Internist and Regenerative Medicine Specialist, said the application of PRP around the genital organ promotes blood flow and cell growth which leads to regeneration within the Clitoris. The result is new, healthy tissues (clitoris) that is permanent, he added.

Dr. Ikudayisi decried the high prevalence of female genital mutilation in Nigeria and in many African countries saying that apart from denying women the opportunity of enjoying sex it has huge health implications.

These include abscesses, cysts, excessive scar tissue, painful sex and menstruation, Hepatitis and other blood-borne diseases, urinary tract infections, infertility, and increased risk of bleeding during childbirth.

Ikudayisi said a survey on February 1, 2022, by Statista Research Department, shows that as of 2018, 20 per cent surveyed women in Nigeria had undergone female genital mutilation. Compared to 2013, this figure decreased by five percentage points (25 per cent)..

According to him, Nigeria ranks the third-highest globally in the prevalence of FGM, accounting for 10 per cent of the global total.

Ikudayisi identified age-long cultural and religious practices as the factors promoting FGM in Nigeria.

He said several civil society organisations and international non-governmental organisations are involved in strong advocacy against FGM while those females who have come under the knife can get their genital area, especially the clitoris back, and hence have full sexual experience through the O-Shot therapy.

Like Loading...

Follow this link:
O-Shot Therapy Raises Hope of Enhanced Sexual Experience in Genitally Mutilated Females - THISDAY Newspapers

The Tendonitis Market to Exhibit Growth at a CAGR of 2.9% In The 7MM During The Study Period (2018-30), Assesses DelveInsight – PR Newswire

The tendonitis market is expected to grow significantly owing to an increase in the targeted patient pool and novel treatment approaches by the leading companies such as R3 Stem Cell, TRB Chemedica, InGeneron, MiMedx Group, among others.

LAS VEGAS, April 14, 2022 /PRNewswire/ -- DelveInsight's Tendonitis Market Insightsreport includes a comprehensive understanding of current treatment practices, Tendonitis emerging drugs, market share of individual therapies, and current and forecasted market size from 2018 to 2030, segmented into 7MM (the United States, the EU5 (the United Kingdom, Italy, Spain, France, and Germany), and Japan.

Key Takeaways from the Tendonitis Market

Discover more about therapies set to grab major Tendonitis market share @Tendonitis Market Landscape

Tendonitis Overview

Tendonitis is an inflammation of a tendon, which is a thick, flexible cord of tissue that connects muscles to bone and helps the bone or structure move. Tendonitis most frequently affects the shoulder, bicep, elbow, hand, wrist, thumb, calf, knee, or ankle. The most common types of tendonitis include Tennis elbow tendonitis, golfer's elbow tendonitis, patellar tendonitis, Achilles tendonitis, supraspinatus tendonitis, trigger finger, or trigger thumb, wrist tendonitis, peroneal tendonitis, and posterior tibial tendonitis.

Tendonitis causes inflammation, soreness, and discomfort in the tendons, ligaments, and muscles around a joint. Different types of tendonitis might strike quickly and continue for days or weeks. The discomfort is exacerbated by movement and normally improves with rest or medication. Tendonitis symptoms might reoccur in the same body part.

Tendonitis symptoms often include discomfort and are commonly characterized as a dull aching (particularly while moving the afflicted limb or joint), soreness, and minor swelling at the place where a tendon joins to a bone.

Tendonitis can be diagnosed if there is discomfort at a specific place in the tendon. If the patient does not react to therapy, the doctor may order various testing. An x-ray can reveal calcium deposits surrounding the tendon, which can aid in Tendonitis diagnosis. Other imaging procedures for Tendonitis diagnosis, such as ultrasound or MRI, may indicate tendon sheath edema.

Tendonitis Epidemiology Segmentation

As per DelveInsight, the total Tendonitis incident caseswere found to be more than 11 million in the 7MM in 2020.

The total Tendonitis treated cases was more than 5 million in the 7MM in 2020.

The Tendonitis Market Reportproffers epidemiological analysis for the study period 2019-2032 in the 7MM segmented into:

Download the report to understand which factors are driving Tendonitis epidemiology trends @ Tendonitis Epidemiological Insights

Tendonitis Treatment Market

Tendonitis may go away over time. If this is not the case, the doctor will offer therapies to minimize pain and inflammation while preserving mobility. Severe symptoms of tendonitis may necessitate the services of a rheumatologist, orthopedic surgeon, or physical therapist. Most tendonitis symptoms, when properly treated, do not result in lasting joint damage or disability. RICE (Rest, Ice, Compression, Elevation) therapy; physical or occupational therapy; splints, braces, or slings these assistive Tendonitis devices allow the affected area to rest until the pain subsides; over-the-counter (OTC) and prescription medications; and corticosteroids injections are all Tendonitis treatment options.

Treatment for Tendonitis aims to alleviate pain and minimize inflammation. Failure to rest will almost certainly prolong the symptoms. If the problem is in a hip, leg, or foot, they may need to refrain from strenuous weight-bearing activities for a brief period of time. This allows the irritation to subside. Ice may aid in the reduction of inflammation and discomfort. Once or twice a day, freeze the affected region for 1015 minutes. If the pain persists, patients may require nonsteroidal anti-inflammatory drugs (NSAIDs) such as aspirin, ibuprofen, or naproxen. Topical NSAIDs are now available and may relieve pain and inflammation without causing stomach problems. In addition, acetaminophen (Tylenol) can help with pain relief.

Discover more about therapy set to grab substantial Tendonitis market share @ TendonitisTreatment

TendonitisPipeline Therapies and Key Players

To know about more new drugs for Tendonitis treatment available in the tendonitis market visit @ Best Anti-inflammatory Drugs forTendonitis Treatment

Tendonitis Market Dynamics

Tendinopathy is becoming more common in the developed world as a result of greater engagement in leisure activities and an older population. As a result, the Tendonitis market is anticipated to increase significantly owing to an increase in the targeted patient pool. Moreover, the anticipated launch of upcoming targeted therapy options and novel treatment approaches by leading companies such as, TRB Chemedica, CollPlant, MiMedx Group, among others for tendonitis treatment would provide hope to patients and have a beneficial impact on the Tendonitis market.

Nevertheless, there are certain limitations associated with the current Tendonitis treatment which are impeding the Tendonitis market growth. The lack of awareness of the biology and clinical importance of the condition may limit the right diagnosis, perhaps leading to a delayed diagnosis and misdiagnosis, the greatest problem of tendonitis treatment and thus limiting the growth of the Tendonitis market. Furthermore, one of the key causes of poor patient care is a breakdown in communication between doctors and patients. It may stifle the expansion of the Tendonitis market and access to treatments. In addition, despite several clinical advancements in the treatment for Tendonitis, there is still an unmet need for effective therapeutic options to supplement intrinsic and surgical healing. These above-mentioned factors are acting as a barrier to Tendonitis market growth.

Scope of the TendonitisMarket Report

Discover more about the future Tendonitis market share of treatment therapies @Drugs forTendonitis Treatment

Table of Contents

1.

Tendonitis Market Key Insights

2.

Tendonitis Market Report Introduction

3.

Tendonitis Market Overview at a Glance

4.

Tendonitis Market Executive Summary

5.

Disease Background and Overview

6.

Treatment and Management

7.

Epidemiology and Patient Population

8.

Patient Journey

9.

Tendonitis Marketed Therapies

10.

Tendonitis Emerging Drugs

11.

7 Major TendonitisMarket Analysis

12.

Tendonitis Market Outlook

13.

Potential of Current and Emerging Therapies

14.

KOL Views

15.

Tendonitis Market Drivers

16

Tendonitis Market Barriers

17.

Unmet Needs

18.

SWOT Analysis

19.

Appendix

20.

DelveInsight Capabilities

21.

Disclaimer

22.

About DelveInsight

Get in touch with our Business executive @Healthcare Due Diligence Services

Related Reports

Tendonitis Epidemiology

Tendonitis Epidemiology Forecast to 2032report delivers an in-depth understanding of the disease, historical, and forecasted Tendonitis epidemiology in the 7MM.

Tendonitis Pipeline

Tendonitis Pipeline Insight, 2022report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Tendonitis companies involved such as Dobecure, Causeway Therapeutics, among others.

Tendinopathy Market

Tendinopathy Market Insights, Epidemiology, and Market Forecast-2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Tendinopathy companies such as TRB Chemedica, CollPlant, MiMedx Group, among others.

Tendinopathy Epidemiology

Tendinopathy Epidemiology Forecast to 2032report delivers an in-depth understanding of the disease, historical, and forecasted Tendonitis epidemiology in the 7MM.

Tendinopathy Pipeline

Tendinopathy Pipeline Insight, 2022report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Tendinopathy companies involved such as Orthocell, InGeneron, Causeway Therapeutics, among others.

Other Trending Reports

Systemic Inflammatory Response Syndrome Market

Systemic Inflammatory Response Syndrome Market Insights, Epidemiology, and Market Forecast-2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Systemic Inflammatory Response Syndrome companies such as Cidera Therapeutics, ContraFect, Scynexis, among others.

Primary Ciliary Dyskinesia Market

Primary Ciliary Dyskinesia Market Insights, Epidemiology, and Market Forecast-2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Primary Ciliary Dyskinesia companies such as Parion Sciences, ReCode Therapeutics, Translate Bio, among others.

Exanthema Market

Exanthema Market Insights, Epidemiology, and Market Forecast-2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Exanthema companies in the domain.

Motor Neuron Disease Market

Motor Neuron Disease Market Insights, Epidemiology, and Market Forecast-2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Motor Neuron Disease companies in the domain.

Paraganglioma Market

Paraganglioma Market Insights, Epidemiology, and Market Forecast-2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Paraganglioma companies in the domain.

Latest Healthcare Blogs

Treatment-resistant Hypertension Market

Top Drugs Losing Patent in 2022

Fabry Disease Market

AboutDelveInsight

DelveInsight is a leading Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platformPharmDelve.

Connect With Us atLinkedIn|Facebook |Twitter

Here is the original post:
The Tendonitis Market to Exhibit Growth at a CAGR of 2.9% In The 7MM During The Study Period (2018-30), Assesses DelveInsight - PR Newswire