Category Archives: Stem Cell Treatment


Applications in Chronic Wound Healing | IJN – Dove Medical Press

Introduction

The skin is the largest organ in the body, accounting for 15% of the total body weight. It is the first line of defense against physical, chemical, and biological factors.1,2 In some cases, the anatomical structure and biological function of the skin are impaired due to internal (local blood obstruction, inflammation, or underlying diseases) or external factors (mechanical injury, chemical corrosion, electric injury, or thermal injury).1,3

After damage, skin can self-heal, and this process involves four phases: hemostasis, inflammation, proliferation, and remodeling (Figure 1).4,5 In the first few minutes after skin damage, the platelets accumulate around the wound and get activated, forming a scab to preventing bleeding.6 After 23 days, the inflammatory phase starts around the wound, and the immune cells remove the dead and devitalized tissues and prevent microbial infections.4 The proliferation phase occurs after the inflammation phase, and it is characterized by the activation of keratinocytes, fibroblasts, endothelial cells, and macrophages, which contribute to wound closure, matrix formation and angiogenesis.7 In the 12 or more months after the primary repair is completed, the regenerated skin tissue is remodeled. During this phase, the processes activated after injury slow down, and the healed wound reaches it maximum mechanical strength.4,5

Figure 1 Phases of wound healing, including the hemostasis, inflammatory, proliferation, and remodeling phase.

Notes: Reprinted from: Tavakoli S, Klar AS. Advanced Hydrogels as Wound Dressings. Biomolecules. 2020;10(8):1169. doi:10.3390/biom10081169.5 2020 by the authors. Licensee MDPI, Basel, Switzerland. Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/).

However, in some cases, the skins self-healing property is inadequate, leading to the formation of chronic wounds. Chronic wounds are defined as wounds that remain unhealed even after 12 weeks.8 The main factors delaying wound repair include diabetes, infections, and long-term inflammation. Diabetic mellitus damages the microenvironment of skin tissue, which is involved in wound regeneration. It causes increases in reactive oxygen species (ROS) levels and poor collagen deposition.911 The hyperglycemia weakens the functions of fibroblasts, keratinocytes, endothelial cells, and stem cells or progenitor cells involved in wound healing.12 Microbial infections deplete the energy and cells required for tissue regeneration, and the bacteria can form biofilms that display antibiotic resistance, immune evasion, and wound adherence.13,14 In unhealed skin, excess inflammation also contributes to wound chronicity owing to its cytotoxic effects and the induced tissue damage, both of which delay wound healing.1517 Traditionally, the chronic wounds are treated with wound dressing made of gauze, skin grafting, or even flap transplantation. Moreover, targeted antibiotics are administered in case of infection. However, Surgery for chronic wounds can be challenging due to limited donor sites, donor damage, scar formation, and even severe functional and psycho-social disorders.1820 Moreover, antibiotic overuse can lead to drug resistance, creating new problems for infectious chronic wounds.21,22 Moreover, chronic wounds become refractory due to infections, diabetes, ischemia, over-degradation of collagen, and other factors, leading to the failure of traditional treatment methods. Thus, novel methods for treating chronic wounds need to be explored.

Skin wounds are the most common type of tissue injury, and they can be caused by trauma, surgery, burns, chronic diseases, or cancers.4,23 Under adverse conditions, wounds often turn chronic. The acceleration of wound repair and improvement of the healing process are the primary objectives of chronic wound treatment. Nanobiotechnology, which involves the use of nano-sized particles in biological systems, represents the convergence of several scientific fields, including chemistry, biology, physics, optics, mechanics, and nanoscale Science and technology. Nanobiotechnology can provide tools and technologies for examining and modulating biological systems.24,25 By applying nanotechnology in the field of bioMedicine, several novel biomaterials, biosensors, and bio-therapies have been designed and studied. It is believed that the combination of nanotechnology and biology can aid in wound management, monitoring, and repair.26,27 Initially, the application of nanobiotechnology in chronic wound treatment was focused on the provision of scaffolds for cell migration and the replacement of traditional gauze dressing.2830 However, with the development of nanotechnology and our understanding of wound healing mechanisms, various nanobiotechnology-based wound-treatments systems including drug and gene delivery platforms, antimicrobial systems, and cell-carrying systems have been developed and found to have prospective applications.3136 Nevertheless, despite these advances, wound dressings remain largely primitive and lack functions that allow wound monitoring and dynamic wound responses. Therefore, smart hydrogels or bandage systems developed using nano-sized biomaterials, which can respond to stimuli or monitor the status of chronic wounds, have been examined.37,38

This review article provides a summary of nanobiotechnology-based scaffold, delivery, antimicrobial, cell-carrying, collagen modulating, stimuli-responsive, and wound monitoring systems for chronic wound healing. Further, the prospects of nanobiotechnology to achieve better treatment outcomes for chronic wounds are discussed.

Physiologically, the wound healing process is affected by several factors, including gene expression; cell functions such as migration, proliferation, and differentiation; the skin microenvironment; infection; ischemiahypoxia; inflammation; and collagen formation and arrangement.1,3,17,3942 These factors are used as references for the design of nanobiotechnology systems that promote chronic wound repair (Figure 2) and need to be carefully considered before designing such systems.

Figure 2 Nanoplatform for chronic wound healing.

To repair tissue defects in the wound area, a platform for cell adhesion, migration, and proliferation ie, a scaffold for cells needs to be established. Such a scaffold can also serve as a platform for multi-functional modification. Given their good biocompatibility, angiogenic capacity, and biomimetic behavior to natural human skin, nano-scaffold systems are widely used in tissue engineering.4346

Tradition treatment methods for chronic wounds that show delayed Union involve local or systemic drug administration. However, the performance of these drugs is suboptimal owing to limitations such as low solubility and low bioactivity. Nanobiotechnology has thus been leveraged for the development of drug, gene, and exosome delivery systems that can help in overcoming these limitations.34,47,48

Infections, which impede tissue repair, should receive careful attention in chronic wound treatment. Silver nano-particles, a product of nanobiotechnology, have been used clinically in the treatment of microbial infection for decades. Moreover, several more recent studies have explored new nanoplatform-based anti-infection therapies, including potential anti-infection nanoparticles (NPs).4952

Cell therapy, especially stem cell therapy, is currently a focus in regenerative medicine and diabetic wound repair. In some basic medical and preclinical studies, chronic wound treatment with stem cells has shown excellent outcomes.5355 However, despite its great potential, the clinical translation of stem cell therapy for chronic wound healing is hindered by the lack of appropriate methods for cell encapsulation and transplantation. Thus, the development of nanobiotechnology-based cell-carrying systems can provide improved therapeutic effects.56,57

With the development of precision medicine, therapeutic systems that monitor wounds and respond to individual stimuli are expected to become popular. One such system is based on ferrihydrite NPs, which can respond to blue light and are effective for antimicrobial and wound healing treatments.58 More stimuli-responsive materials and monitoring systems for chronic wound healing can be generated through nanobiotechnology.

The term scaffold system generally refers to materials that can integrate with living tissues and cells and can be implanted into different tissues where they supplement natural tissue function based on specific conditions. In order to enable seed cells to proliferate and differentiate, a scaffold composed of biological materials that acts as an artificial extracellular matrix (ECM) is required. Scaffolds are critical for tissue engineering systems, including those for bone, cartilage, blood vessels, nerves, skin, and artificial organs (eg, liver, spleen, kidney, and bladder).

Nano-scaffold systems aimed at chronic wound healing need to possess certain important features.

1. Safety and good biocompatibility: Scaffolds should be safe. Furthermore, their chemical components and degradation products should cause minimal immune or inflammatory responses in the body during a predetermined period.59

2. Appropriate size, dimensions, and mechanical strength: The chemical features of the scaffold should provide suitable microenvironments and maintain the biological activity of loaded cells or tissues for a long time.

3. Appropriate pore size and distribution: Scaffolds should have a highly and well-connected porous structure with an ideal pore size to allow cells, drugs, and bioactive molecules to get evenly distributed throughout the scaffold.60

4. Excellent biological behaviors: Scaffolds and the substances present in the scaffold should promote the proliferation and migration of fibroblasts, keratinocytes, and endothelial cells, thus promoting wound healing.61,62

5. Appropriate wound healing environment: The scaffold system should be able to absorb the wound exudate and prevent wound dehydration, reducing surface necrosis on the wound.63,64

Scaffold systems can be classified as follows based on the source and function of the materials.

When designing scaffold systems for chronic wound, an appropriate matrix source needs to be selected. Table 1 lists a few sources of nanocomposites used in wound dressing. Natural nanomaterials and their derivates have good biocompatibility and can be degraded by enzymes or water. However, their characters and quality differ from batch to batch and cannot be standardized. In contrast, synthetic biomaterials, such as polyethylene glycol (PEG) nano-scaffolds, show more stable structural properties and can be chemically modified. However, the biosafety of synthetic materials needs to be strictly examined.

Table 1 Sources of Nanocomposites

According to their functions, tissue engineering materials can be used for bones, nerves, blood vessels, skin, and other tissues (eg, tendon, ligament, cornea, liver, and kidneys).

Tissue engineering scaffolds for the skin can be of several types. These include natural polymers (chitosan, hyaluronic acid, and collagen), nanocomposite scaffolds (eg, nanobioactive glass and metal NPs), and conducting polymers (eg, polyaniline, polypyrrole, and polythiophene).7375 Taghiabadi et al synthesized an intact amniotic membrane-based scaffold for cultivating adipose-derived stromal cells (ASCs). By ASCs on an acellular human amniotic membrane (HAM), they created a neoteric skin substitute.76 Zhang et al designed a conductive and antibacterial hydrogel based on polypyrrole and functionalized Znchitosan molecules for the management of infected chronic wounds. They demonstrated the promising potential of the hydrogel in promoting the healing of the infected chronic wound after electrical stimulation. Currently, other tissue engineering scaffolds such as calcium phosphates and composite materials (eg, hydroxyapatite, -tricalcium phosphate, and whitlockite) for bone tissue engineering and amniotic membranes for corneal tissue engineering are under research.69,77

Skin tissue engineering scaffolds can be categorized as porous, fibrous, microsphere, hydrogel, composite, and acellular materials.73 Typically, natural biomaterials and their derivatives are biodegradable, absorbable, and harmless to the body, but their strength and processing performance are poor and their degradation speed cannot be controlled. Hence, in order to improve the mechanical and biological properties of scaffolds (eg, adhesion, strength, processing performance, and degradation speed) and accelerate wound healing, composite scaffolds have been developed by combining the characteristics and advantages of different materials. Depending on their constituents, these composite scaffolds can achieve specific functions. Currently, most novel scaffolds being developed use composite materials to obtain multifunctional characteristics.

Delivery systems are used to deliver drugs, cells, genes, and other neoteric bioactive molecules to the body or target area via transplantation or injection.78 Traditionally, delivery systems are broadly divided into two categories, drug delivery and cell delivery. With continuous Innovation in scientific research, new approaches, including gene delivery and the delivery of bioactive molecules such as growth factors, proteins, and peptides, are being developed.

Recently, there has been a significant increase in new biotechnology-based treatments, among which cell and gene therapies are quite sophisticated. Exosomes have shown superior therapeutic potential against various conditions, and delivery methods are being devised to maximize their therapeutic effectiveness. Moreover, exosomes are also emerging as a delivery system for other substances (eg, small molecules and miRNAs).79 NPs are essential for the delivery of these refined substances. In addition to serving as delivery vehicles, NPs can also act as diagnostic and therapeutic agents for some diseases.80 Research on nanoparticle-based drug delivery has mainly been focused on targeted drug delivery, and especially tumor-targeted drug delivery.81

A drug delivery system serves as a vehicle for therapeutic molecules. It allows drug delivery in the body, improves drug efficacy, and allows safe and controlled drug release.

The conventional routes for drug delivery80 are gastrointestinal drug delivery (eg, oral and rectal), parenteral administration (eg, subcutaneous, intramuscular, and intravenous injection) and topical administration (eg, percutaneous injection and wound dressings). Novel drug delivery systems for wound healing can be classified into the following categories: NPs, microcarriers, and tissue-engineered scaffolds.82 Skin tissue engineering scaffolds have been introduced earlier in this review, and NPs and microcarriers will be introduced in detail here (Table 2).

Table 2 Drug Delivery Systems Developed Using Nanotechnology

Drug-loaded nano-scaffolds that promote wound healing after topical administration have been developed. However, due to their poor solubility, short half-life, and other drawbacks, some drugs do not accumulate at an optimal concentration at the wound site for a long duration.83 Nano-scaffolds with varying porous structures can be used to load drugs or bioactive molecules, and the porous structure can provide a breathable environment for the wound.84 NPs carrying poorly soluble drugs are widely used to prepare controlled drug delivery systems. Nano-scaffolds typically show slow degradation, allowing long-term drug release and thereby maintaining an ideal concentration of the drug in the plasma.85 Shamloo et al developed polyvinyl alcohol (PVA)/chitosan/gelatin hydrogels to overcome the short half-life of basic fibroblast growth factor (bFGF). The biocompatibility of the hydrogel supported the continuous delivery of bFGF and significantly accelerated wound healing.86

During the treatment of chronic wounds, the drug is usually applied directly on affected region. Nanotechnology-based drug delivery systems could enable controlled drug release. Meanwhile, the degradability and stability of the drug could also be modified using nanosystems. Hence, these drug delivery systems could improve treatment compliance among patients with chronic wounds by reducing the application frequency and the cost of treatment.

It is widely acknowledged that metal ion-based biomaterials exhibit promising antimicrobial activity when applied to wounds, making them very suitable for the management of diabetic wounds, which are prone to infection. Given their reducing properties, under oxidative stress, cuprous ions provide a promising therapeutic option for diabetic wounds. Copper ions have also been reported to promote angiogenesis.115117 Equipped with infrared absorption and efficient heat generation abilities, semiconductor cuprous sulfide (Cu2S) NPs are widely employed as photothermal agents. Wang et al utilized the photothermal effect of Cu2S and the angiogenic effect of Cu ions to prepare electrospun fibers containing Cu2S NPs, achieving a combination of advantages based on the components and successfully promoting diabetic wound healing. Moreover, their biomaterial could also effectively inhibit the growth of skin tumors both in vivo and in vitro.70 This system demonstrated the effectiveness of bifunctional tissue engineering biomaterials, providing a novel method for drug delivery for the treatment of biological conditions.

Classic gene therapy generally involves the expression of exogenous genes or the silencing of target genes via viral or non-viral delivery.118,119 In general, gene delivery via viral transfection may be carcinogenic.119 Most gene therapies for diabetic wounds are based on siRNAs. Gene therapy has become a promising strategy for the treatment of various diseases, and its effects are mediated via the regulation of RNA and protein expression.120 Many unmodified gene therapy agents, such as proteins, peptides, and nucleic acids, are rapidly degraded or eliminated from systemic circulation before they can accumulate at effective concentrations at the target site. Owing to poor pharmacokinetics, repeated administration is warranted. This, in addition to the narrow range of safe doses, often leads to adverse effects during treatment.121

Several studies on wound management and especially chronic diabetic wound management have focused on gene- or RNA-based (eg, mRNA, microRNA, circRNA, and lncRNA) therapies.122 Subcutaneous local injections can be used to directly deliver RNAs or proteins to the wound site.123 However, due to the short half-life of the therapeutic agent, repeated administration is required, often leading to pain and poor treatment compliance. Drug delivery systems not only solve these problems but also protect gene-related small molecules from degradation and eliminated from the body. The greatest challenge in gene therapy is ensuring the successful transduction or transfection of target genes into host cells by crossing extracellular and intracellular barriers. Therefore, the engineering of gene delivery vehicles is complex.118 Moreover, the materials used to encapsulate gene-related small molecules are required to have low toxicity and promote a high transfection efficiency.124 Currently, the NPs that deliver siRNAs to promote wound management are composed of lipids, polymers (eg, chitosan, PEG), hyperbranched cationic polysaccharides (HCP), and silicon.125130

Shaabani et al developed layer-by-layer self-assembled siRNA-loaded gold NPs with two different outer layers Chitosan ([emailprotected]) and Poly L-arginine ([emailprotected]).126 They compared the two types of NPs, which had a similar core structure. They found that the two polymers had different escape mechanisms: the buffering capacity of chitosan resulted in endosome disruption,131 while PLA bound to the endosome lipid bilayer and promoted escaped through pore formation. Their results indicated that an outer layer of PLA allows the endosomal escape of siRNA, thus improving transfection efficiency and delivering target molecules to promote diabetic wound healing. Given that naked siRNAs are easily eliminated from the body, Li et al and Lan et al designed four HCP derivative-based vehicles128,129 for the delivery of siRNA against MMP9. This treatment led to the knockdown of MMP9, which prevents the healing of diabetic wounds, and thus promoted diabetic wound healing. Currently, nanocomposite-based gene delivery applications are focused on siRNA. However, efforts to deliver other products such as miRNA, lncRNA, or even DNA will be required in the future.

Exosomes are endosome-derived vesicles (30 to 150 nm in size) secreted by a variety of cells, including adipose stem cells (ADSCs), bone marrow stem cells (BMSCs), and mesenchymal stem cells (MSCs).132,133 Different types of cells secrete exosomes with different specific markers, which account for their specific functions. Despite their different origins, exosomes have a similar appearance and size and often have a common composition. Once they are isolated from an extracellular medium or from biological fluids, the source of exosomes cannot be ascertained of.134 Exosomes can be employed as small molecules for wound treatment. The combination of exosomes with porous NPs can increase therapeutic effects while maintaining the advantages of a scaffold. Importantly, exosomes can also be used as nanocarriers for drug delivery and targeted therapy, and these are called engineered exosomes.133,135

Exosomes can effectively promote diabetic wound healing.136,137 Shiekh et al embedded ADSC-derived exosomes (ADSC-exo) into antioxidant polyurethane scaffolds to achieve sustained exosome release. Their nanosystem leveraged the advantages of the scaffold, including antioxidant and antibacterial effects, to accelerate diabetic wounds healing both in vivo and in vitro.71 To prolong the half-life and lower the clearance rate of exosomes, Lei et al designed an ultraviolet-shielding nano-dressing based on polysaccharides that allowed exosome delivery and had self-healing, anti-infection and thermo-sensitive properties.61 These findings indicate that exosomes can be stabilized and well-delivered to target cells by combining them with porous NPs or nanocarriers and can be applied for treating chronic wounds.

It is widely accepted that infection is an important factor to monitor during the wound healing process as it can lead to progression of the chronic wound or even sepsis.138140 Conventional prevention and treatment approaches for wound infection involve local or systemic antibiotic administration, which can lead to failed anti-infection treatment or even antibiotic resistance.141,142 Several nano-formulations that have antimicrobial ability have been developed and used in anti-infectious wound therapy, playing a critical role in infection management. Table 3 lists some antimicrobial nanobiotechnology-based systems used in wound healing.

Table 3 Nanomaterials Used in Anti-Microbial Wound Dressing

Metals have been used as inorganic antimicrobial agents for thousands of years and were even used as anti-infection agents in ancient Persia.162 Metal NPs, such as AgNPs, AuNPs, and CuNPs, have attracted great attention due to their anti-infection properties and low toxicity.163 Given that metal NPs do not cause antimicrobial resistance and release metal ions or produce ROS which can kill microorganisms they appear to be suitable alternatives to antibiotics as.164,165

AgNPs, which are the more well-known metal NPs, have been used widely in clinical practice and basic medical research. Wound treatment products containing AgNPs have been commercially available for decades.166 AgNPs can continuously generate Ag+, which reacts with proteins and nucleic acids, causing molecular defects and killing bacteria and viruses.167170 Several studies have shown that AgNPs have good potential as antiseptics. Luna-Hernndez et al found that a combination of functional chitosan and silver nanocomposites showed antibacterial effects against S. aureus and P. aeruginosa in burn wounds.152 Moreover, in mice treated with the composite dressing, silver accumulation was found to be far lower than that in mice treated with the clinically used AcasinTM nanosilver dressing. Zlatko et al demonstrated that the AgNPs hydrogel serves as a versatile platform, with features such as antibacterial efficacy, exudate absorbance, low cost, biocompatibility, hemocompatibility, and improved healing for chronic wounds.171 Huang et al constructed an organic framework-based microneedle patch containing AgNPs. The product showed transdermal delivery and could prevent S. aureus, E. coli, and P. aeruginosa infections in diabetic wounds.172 In addition, several commercialized products containing AgNPs have been developed for clinical treatment. These include Acticoat, Allevyn Ag, Aquacel Ag Surgical, Atrauman Ag, Biatain Silicone Ag, Flaminal, Mepilex Transfer Ag, SILVERCEL, and Urgo Clean Ag.

Nano-sized gold is also useful as an anti-infection agent. It has been confirmed that AuNPs bind to bacterial DNA and show bactericidal and bacteriostatic properties.173,174 Some studies show that Au nanocomposites can kill MRSA and P. aeruginosa through photothermal effects and could promote wound closure.150,175

Compared with gold and silver, copper is less expensive and more easily available. CuNPs are considered the best candidates for developing future technologies for the management of infectious and communicable diseases.49 Cai et al developed a CuNP-embedded hydrogel that accelerated wound healing and showed effective antibacterial capacity against both gram-positive and gram-negative bacteria as well as great photothermal properties.176

Inorganic non-metal nano-materials have been also considered potential antimicrobial agents owing to their intrinsic anti-infection effects.177 Based on the unique structural and physio-chemical properties of carbon nanomaterials, a research team prepared a carbon nanofiber platform that inhibits the growth of E. coli and MRSA.178 In this study, CuNPs and ZnNPs were asymmetrically distributed in carbon NFs grown on an activated carbon fiber substrate using chemical vapor deposition (CVD). The carbon NFs platform inhibited the growth of gram-positive and gram-negative bacterial strains with superior efficiency than simple metal NPs. Another study showed that carbon nanotubes can be used to prepare wound-repairing bandages with infection-preventing properties.179

The natural organic biomaterial chitosan and its derivatives are popular in biomedicine. Chitosan possesses good biocompatibility, antimicrobial properties, and low immunogenicity.180 Using nanobiotechnology, Ganji et al fabricated a nanofiber with chitosan-encapsulated nanoparticles loaded with curcumin for wound dressing. The electrospun chitosan-based nanofiber inhibited the growth of E. coli and MRSA by 98.9% and 99.3% in infected wounds in mice.50 Another type of chitosan nanofiber also showed potential in wound care owing to its antibacterial and re-epithelialization-promoting effects.181 Antibiotic-loaded chitosan nanofibers have also been used for local drug delivery and wound treatment.182 Other metalorganic framework nanorods have also shown bacterial inhibition in infectious wounds.183 Dias et al developed a series of soluble potato starch nanofibers sized 70264 nm. They incorporated carvacrol during the synthesis of the potato starch nanofibers, and the obtained nanocomposites showed great anti-pathogenic activity against S. aureus, E. coli, L. monocytogenes, and S. typhimurium, highlighting their potential as agents for wound dressing.184

With respect to organic nano-materials, anti-infection approaches focus on natural antibacterial compounds such as chitosan and its derivatives. Further, owing to the bactericidal effects of metals, metal-organic frameworks are also used. Given that metal NPs are associated with the potential risks of metal deposition, organic nano-antimicrobial materials, especially natural macromolecules with antibacterial properties, may become useful for wound dressing.

Biofilm, which are made up of surface-attached groups of microbes, are considered to be the primary cause of chronic wounds owing to their role in antibiotic resistance.141,185187 Most biofilms are formed on the surface of wounds. However, some special biofilms can get implanted into the deep layers of skin tissue, making traditional diagnose and treatment challenging.188 The clinical treatment of biofilms in wounds involves wound cleansing with polyvinylpyrrolidone or hydrogen peroxide, debridement, refashioning of wound edges, dressing, and the topical or general administration of antibiotics.189 With further insights into the mechanisms of biofilm formation and developments in nanobiotechnology, nanomaterials effective for biofilm therapy have been developed.

Nanomaterials based on metals or metal oxides are widely used against wound biofilms, including silver, copper, gold, titanium, zinc oxide, magnesium oxide, copper oxide, and iron oxide.190,191 Owing to the small size of these particles, metal or metal oxide NPs can move across bacterial membranes and rupture them. They can destroy enzyme activity and the respiratory chain in bacteria. It has been demonstrated that Ag NPs and silver oxide NPs are the most effective against microbial biofilms.192,193 Abdalla et al functionalized nano-silver with lactoferrin and incorporated them in a gelatin hydrogel, generating a dual-antimicrobial action dressing for infectious wounds and maximizing the anti-biofilm property of silver.194

Chitosan, bacterial cellulose (BC) and other natural antimicrobials have been modified using nanotechnology to treat wound biofilms. Owing to the positive charge on the polymeric chain of chitosan, chitosan NPs easily adhere to the negatively charged microbial membrane, triggering changes in permeability and preventing biofilm formation.195 Zemjkoski et al obtained chitosan NPs through gamma irradiation and encapsuled them into BC to form BC-nChiD hydrogels with excellent anti-biofilm potential. These hydrogels could provide a 90% reduction in viable biofilms and a 65% reduction in biofilm height.196 Mahtab reduced the amount of bacteria in a planktonic condition by treating bacterial biofilms with photodynamic therapy using curcumin encapsulated into silica NPs. After exposure to blue light, ROS was produced owing to the photodynamic properties of silica NPs. The ROS damaged biofilms, and the curcumin released prevented bacterial growth.197

The size of nanoparticles can be controlled, and they have a large specific area, can penetrate bacterial membranes, and show bactericidal properties. Hence, nanotechnology has great potential in destroying biofilms and treating infectious chronic wounds. In addition to providing nanoparticles with anti-infection properties, nanotechnology could also be used to provide a platform for antibiotics, enhance their solubility, prolong their half-life, and reduce the required treatment dose.

Due to its superiority with respect to tissue engineering, cell-based therapy is extensively used for chronic wound treatment.198201 Stem cells derived from bone marrow, the umbilical cord, and adipose and cutaneous tissue can differentiate into various tissue types and modulate cell migration, collagen deposition, re-epithelialization, and tissue remodeling.198,202205 Nanofibers prepared using electrostatic spinning are widely used for scaffolding. Mao et al prepared polycaprolactone nanofibrous scaffolds and combined collagen with bioactive glass NPs (CPB nanofibrous scaffold). The CPB nanofibrous scaffold exerted positive effects as a cell-carrying system containing epithelial progenitor cells (EPCs). The EPC-carrying CPB bioactive complex promoted wound healing by enhancing cell proliferation, granulation tissue formation, re-epithelialization, and cell adhesion (Figure 3).206 Khojasteh et al found that curcumin-carrying chitosan/poly(vinyl alcohol) nanofibers can carry pad-derived mesenchymal stem cells and show excellent curcumin release and improve cell adhesion and proliferation, indicating that they could be useful in wound dressings.207 Kaplan et al produced an injectable silk nanofiber hydrogel embedded with BMSCs. The nanofiber hydrogel maintained the stemness of the BMSCs, successfully carrying them to the target site and promoting wound healing through increased angiogenesis and collagen deposition.57

Figure 3 Schematic of a CPB/EPC construct that promotes wound healing. CPB enhances cell proliferation, collagen deposition, and EPC differentiation via the Hif-1/VEGF/SDF-1 pathway. This results in the rapid vascularization and healing of full-thickness wounds.

Notes: Reprinted from: Wang C, Wang Q, Gao W et al. Highly efficient local delivery of endothelial progenitor cells significantly potentiates angiogenesis and full-thickness wound healing. Acta Biomaterialia. 2018;69:156169. doi:10.1016/j.actbio.2018.01.019.206 2018 Acta Materialia Inc. Published by Elsevier Ltd. All rights reserved. With permission from Elsevier. Available from: https://www.sciencedirect.com/science/article/abs/pii/S1742706118300308#f0060.

Usually, cell therapy in wound care is performed using micrometer-scale carriers as cell sizes fall in the range of microns. With the development of nanotechnology, an increasing number of nanofibers and NPs are being developed for cell therapy aimed at treating chronic wound given the excellent pro-differentiation, stemness-holding, and immunoregulation properties of the nanocomposites.

As an important component of the extracellular matrix, collagen mediates communication between cells, provides a scaffold for cell migration and adhesion, and plays a role in chronic wound healing.4 Some nanobiotechnology-based platforms have been used for collagen modulation. Sun et al loaded N-acetyl cysteine onto graphene oxide (GO) NPs to enable scarless wound healing (Figure 4).208 In their study, GO NPs decreased collagen metabolism and improved the balance between collagen formation and degradation, thus allowing the wound to heal without scarring. In another study by the same group, a polyamide nanofiber-based multi-layered scaffold was found to promote wound healing by encouraging the uniform arrangement of collagen.209 Krian et al synthesized a 3-D biomatrix with nanotized praseodymium that promotes collagen function via the stabilization of native collagen. Their rare-earth metal nanoparticles thus showed potential applications in wound care.210

Figure 4 Wound healing effect of a scaffold based on GO NPs.

Notes: Adapted from: Li J, Zhou C, Luo C et al. N-acetyl cysteine-loaded graphene oxide-collagen hybrid membrane for scarless wound healing. Theranostics. 2019;9(20):58395853. doi:10.7150/thno.34480.208 The author(s). Creative Commons Attribution License (https://creativecommons.org/licenses/by/4.0/). See http://ivyspring.com/terms for full terms and conditions.

In chronic wound treatment, deposited collagen acts as a natural scaffold for cells, and therefore, modulating collagens is synonymous with re-establishing tissue structure in the wound area. As a result, collagen-modulating nano-systems have mainly been used for accelerating tissue repair. However, the studies by Suns group are inspirational and demonstrate that this approach should also be utilized for developing chronic wound treatments that decrease scarring.

Despite the availability of dozens of commercial wound-care products, bionic systems have not yet been adopted for wound healing. There is an urgent need for smart wound-healing systems that can respond to the stimuli (temperature, pH, glucose, enzyme, etc.) at the site of the chronic wound area.211,212 Through developments in nanobiotechnology, NPs with stimuli-response characteristics have received great attention. Gong et al synthesized a nanozyme consisting of poly(acrylic acid)-coated Fe3O4 NPs (pFe3O4) and then combined them with GO to produce pFe3O4@GO NCs. The pFe3O4@GO NCs could react with glucose and function as a self-supplying H2O2 nanogenerator at the wound site, allowing the chemodynamic treatment of wound infections.157 Some researchers developed photoactive electrospun nanofibers using cellulose acetate, polyethylene oxide, methylene blue, and three-layered cellulose acetate/polyethylene oxide/silk fibroin/ciprofloxacin. The nanofibers could produce ROS after light irradiation at 635 nm, accelerating the healing of infectious wounds by inhibiting S. aureus, K. pneumoniae, and P. aeruginosa biofilms.213 Zhang et al developed a hybrid hydrogel with MnO2 nanosheets. The injectable MnO2 nanosheet hydrogel could perform thermogenesis under 808-nm laser irradiation, eliminating ROS and inflammation and promoting wound repair.214 Overall, nano-structures functionalized using stimuli-response properties could simulate the biological, chemical, and physical characteristics of natural skin, enabling tissue regeneration in refractory wounds.

Given the elucidation of mechanisms and physiological changes associated with wound healing, sensors that allow real-time monitoring of wound repair have been developed.215217 A complex smart wound-monitoring wound dressing has also been invented.218 This dressing contains a nanofiber membrane made of chitosan/collagen, and promotes proliferation and regeneration by upregulating extracellular matrix secretion and promoting integrin/FAK signaling. Olivo et al added AgNPs to a fiber-based membrane monitor to increase the active surface area in the sensor, improving the detection sensitivity for biomarkers in the wound area.219 In order to avoid secondary wound damage caused by dressing changes, Jiang et al created bacterial cellulose-based membranes with aminobenzeneboronic acid-modified gold nanoclusters (A-GNCs), which could be used for treating wounds infected with multidrug-resistant bacteria.220 A-GNCs emit bright orange fluorescence under UV light, and the intensity of this fluorescence decreases with the release of A-GNCs. This allows healthcare professionals to determine when the dressing needs to be replaced. In the past few years, dressings that can monitor the status of chronic wounds in real-time have been tested. However, this field is relatively new, and current research on nanotech-based systems for monitoring chronic wounds is scarce.

Along with advances in nanobiotechnology research, several new nanosystems have advanced from the laboratory investigation stage to the clinical trial stage. Table 4 lists some clinical trials that have tested nano-therapies for wound healing. As early as 2014, Lopes et al investigated the cost-effectiveness of using nanocrystalline silver for treating burns. Their study showed that AgNPs provided faster wound healing than traditional silver sulfadiazine, requiring fewer dressing changes and reducing the human resource burden.221 Meanwhile, some clinical trials tested the use of nano-products for treating chronic wounds (Table 4). Although metal NPs were typically used for antimicrobial therapy, one clinical trial studied the efficacy and safety of autologous nano-fat combined with platelet-rich fibrin for treating refractory diabetic foot wounds. However, overall, there were few clinical trials examining the applications of nanoplatforms in chronic wound care, likely owing to inadequate previous research on biocompatibility. Moreover, few doctors participated in research on nanotechnology-based chronic-wound treatment, and hence, several clinical requirements were ignored or misunderstood.

Table 4 List of Clinical Trials for Nanobiotechnology-Based Wound Treatment

As nanobiotechnology has developed, nano-sized biomaterials have been widely applied for treating chronic wounds. This review article highlights that the application of nanotechnology in chronic wound treatment has, so far, largely focused on scaffold construction, anti-infection treatment, and substance delivery.34,45,47,130,147

In scaffold systems, nanobiotechnology provides both materials and techniques for managing chronic wounds. Electrospinning, a nanotechnique, allows the production of biomimetic structures that mimic the natural skin and help in healing refractory wounds.50 Furthermore, some nano-scaffolds promote cell adhesion and migration by mimicking the construction of natural tissues, thus promoting chronic wound healing. Nevertheless, there is further scope to improve the quality of natural nano-biomaterials and the biocompatibility of synthetic nano-biomaterials to increase their application.

Dozens of metal NPs, and especially AgNPs, have been used in antimicrobial therapy for chronic wounds.163 However, metal deposition can cause DNA and cell damage. Hence, nanomaterials that prevent infection without causing toxicity are required. Further effort should be made to decrease the accumulation of heavy metals. Alternatively, nanocomposites without metal elements should be adopted more often in the future.

To overcome the ever-changing environment of the skin during chronic wound healing, several wound-monitoring and stimuli-responsive biomaterials have been developed.58,157,218 By leveraging specific characteristics, such as the photothermal effect, chemo-dynamic effect, fluorescence, and thermo-sensitivity, more nano-biomaterials that can be used in stimuli-responsive and dynamic monitoring systems for wound care should be developed. Most studies on wound healing have focused on migration-promoting effects, antimicrobial activity, and substance delivery. However, few nanotech-based multifunctional smart systems, such as smart dressings that show specific responses to stimuli, have been developed. Researchers in this field should work towards developing smart systems based on the mechanisms of disunion in chronic wounds, which could effectively demonstrate the potential of nanobiotechnology in promoting chronic wound repair.

Despite the decades-long history of nanotechnology research, few products and therapies based on nanobiotechnology have become available commercially or entered the clinical trial phase. One reason for this is that most basic nanotech research on chronic wound healing is performed in rodent models, such as C57BL/6 mice or SpragueDawley rats, even though the skin structure and chronic wound healing processes differ between rodents and humans.222 The wound healing effects observed in primates, such as humans, may not be as good as those in rats and mice. Meanwhile, the cost of nano-materials and processing platforms required for large-scale preparation also hinder the clinical translation of nanotechnologies.

During the past few years, numerous nano-materials and techniques have been used to repair chronic wounds. This review summarizes some nanobiotechnology-based systems and nanoplatform designs that can be used for treating chronic wounds. It highlights that a smart dressing for chronic wounds that allows real-time monitoring and has stimuli-responsive abilities is one possible direction for the future of nano-wound-repairing systems. We hope this review motivates the development of more sophisticated wound management systems based on nanobiotechnology in the future.

The authors acknowledge the support from the National Natural Science Foundation of China (81974289, 81772094), the Key Research and Development Program of Hubei Province (grant number 2020BCB031), the Guangdong Basic and Applied Basic Research Foundation (2019B1515120043), the international cooperation research project of Shenzhen, the international cooperation research project of Shenzhen (GJHZ20190822091601691), and the Key Project of Basic Research of Shenzhen (JCYJ20200109113603854).

The authors report no conflicts of interest in relation to this work.

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2. Vig K, Chaudhari A, Tripathi S, et al. Advances in skin regeneration using tissue engineering. Int J Mol Sci. 2017;18(4):789.

3. Stojadinovic A, Carlson JW, Schultz GS, Davis TA, Elster EA. Topical advances in wound care. Gynecol Oncol. 2008;111(2):S70S80.

4. Gurtner GC, Werner S, Barrandon Y, Longaker MT. Wound repair and regeneration. Nature. 2008;453(7193):314321.

5. Tavakoli S, Klar AS. Advanced hydrogels as wound dressings. Biomolecules. 2020;10(8):1169.

6. Versteeg HH, Heemskerk JW, Levi M, Reitsma PH. New fundamentals in hemostasis. Physiol Rev. 2013;93(1):327358.

7. Wilkinson HN, Hardman MJ. Wound healing: cellular mechanisms and pathological outcomes. Open Biol. 2020;10(9):200223.

8. Olsson M, Jarbrink K, Divakar U, et al. The humanistic and economic burden of chronic wounds: a systematic review. Wound Repair Regen. 2019;27(1):114125.

9. Stadelmann WK, Digenis AG, Tobin GR. Physiology and healing dynamics of chronic cutaneous wounds. Am J Surg. 1998;176(2ASuppl):26S38S.

10. Lerman OZ, Galiano RD, Armour M, Levine JP, Gurtner GC. Cellular dysfunction in the diabetic fibroblast: impairment in migration, vascular endothelial growth factor production, and response to hypoxia. Am J Pathol. 2003;162(1):303312. doi:10.1016/S0002-9440(10)63821-7

11. Duscher D, Januszyk M, Maan ZN, et al. Comparison of the hydroxylase inhibitor dimethyloxalylglycine and the iron chelator deferoxamine in diabetic and aged wound healing. Plast Reconstr Surg. 2017;139(3):695e706e. doi:10.1097/PRS.0000000000003072

12. Rodrigues M, Wong VW, Rennert RC, Davis CR, Longaker MT, Gurtner GC. Progenitor cell dysfunctions underlie some diabetic complications. Am J Pathol. 2015;185(10):26072618. doi:10.1016/j.ajpath.2015.05.003

13. Costerton JW, Stewart PS, Greenberg EP. Bacterial biofilms: a common cause of persistent infections. Science. 1999;284(5418):13181322. doi:10.1126/science.284.5418.1318

14. Versey Z, da Cruz Nizer WS, Russell E, et al. Biofilm-innate immune interface: contribution to chronic wound formation. Front Immunol. 2021;12:648554.

15. Wang X, Coradin T, Helary C. Modulating inflammation in a cutaneous chronic wound model by IL-10 released from collagen-silica nanocomposites via gene delivery. Biomater Sci. 2018;6(2):398406.

16. Xu Z, Liang B, Tian J, Wu J. Anti-inflammation biomaterial platforms for chronic wound healing. Biomater Sci. 2021;9(12):43884409.

17. Zhao R, Liang H, Clarke E, Jackson C, Xue M. Inflammation in chronic wounds. Int J Mol Sci. 2016;17(12):2085.

18. Boateng JS, Matthews KH, Stevens HNE, Eccleston GM. Wound healing dressings and drug delivery systems: a review. J Pharm Sci-Us. 2008;97(8):28922923.

19. Schiestl C, Stiefel D, Meuli M. Giant naevus, giant excision, eleg(i)ant closure? Reconstructive surgery with Integra Artificial Skin to treat giant congenital melanocytic naevi in children. J Plast Reconstr Aesthet Surg. 2010;63(4):610615.

20. Schiestl C, Neuhaus K, Biedermann T, Bottcher-Haberzeth S, Reichmann E, Meuli M. Novel treatment for massive lower extremity avulsion injuries in children: slow, but effective with good cosmesis. Eur J Pediatr Surg. 2011;21(2):106110.

21. Eming SA, Wynn TA, Martin P. Inflammation and metabolism in tissue repair and regeneration. Science. 2017;356(6342):10261030.

22. Mookherjee N, Anderson MA, Haagsman HP, Davidson DJ. Antimicrobial host defence peptides: functions and clinical potential. Nat Rev Drug Discov. 2020;19(5):311332.

23. Eming SA, Martin P, Tomic-Canic M. Wound repair and regeneration: mechanisms, signaling, and translation. Sci Transl Med. 2014;6(265):265sr6.

24. Paradise J, Wolf SM, Kuzma J, RamacHandran G, Kokkoli E. Introduction: the challenge of developing oversight approaches to nanobiotechnology. J Law Med Ethics. 2009;37(4):543545.

25. Roco MC. Nanotechnology: convergence with modern biology and medicine. Curr Opin Biotechnol. 2003;14(3):337346.

26. Wang B, Kostarelos K, Nelson BJ, Zhang L. Trends in micro-/nanorobotics: materials development, actuation, localization, and system integration for biomedical applications. Adv Mater. 2021;33(4):2002047.

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Applications in Chronic Wound Healing | IJN - Dove Medical Press

Blood, sweat, and many miles for cellular therapy – Martha’s Vineyard Times

From the left, Theresa Janeczek (Edgartown), Cathy Mayone (Edgartown), Lisa Conroy Murphy (Edgartown), Jacki Reich, Bob Falkenberg, Chuck Klaniecki, and Matt Berg (right) at a rest stop. Stefanie Cronin

The team met at the Steamship Authority in Vineyard Haven in the morning to get ready to ride. Natalie Aymond

The team of bikers who met in the morning (left to right): Bruce Rayvid (Cycling Club of MV), Bob Falkenberg, Elle Crofton, Chuck Klaniecki, Jacki Reich, Matt Berg, Cathy Mayone, Harry Crofton, Roger Moffat (Cycling Club of MV), and Lisa Conroy Murphy (Cycling Club of MV). Natalie Aymond

Harry Crofton (left) and Bob Falkenberg arriving at the Edgartown Memorial Wharf. Natalie Aymond

Roger Moffat (left), Harry Crofton, Elle Crofton, and Bob Falkenberg cheering on the rest of the team as they arrive. Natalie Aymond

Matt Berg (left), Bob Falkenberg, Elle Crofton, Harry Crofton, Cathy Mayone (right), and Theresa Janeczek (below) at the Edgartown Memorial Wharf. Natalie Aymond

Bob Falkenberg receiving his transplant through Be The Match. Be The Match

Bob Falkenberg, a 13-year leukemia survivor, is just one example of a transplant recipient that Be The Match (BTM) has been able to treat and save by connecting him with a blood marrow donor. But while Falkenberg is a face of success in matching patients with a donor, the 12,000 patients diagnosed with life-threatening blood cancers or other blood cell diseases each year are not always as easily treated, especially with financial barriers and a disparity in match rates for non-white patients.

Just two years after his transplant, and with a push from a 100-mile bike ride challenge from his friend, Falkenberg has been biking to raise money and awareness for the need for transplant donors for the past 11 years. He and his team of nine riders took to the Island July 20 to continue this support for Be The Match, part of their month-long East Coast ride event, Tour De TC. This annual bike ride raises critical funds for Be The Match, with this years ride aimed at raising enough funds to financially support 25 families in need of treatment.

In his past years of biking for Be The Match, Falkenberg has embarked on rides from Boston to Key West, Vancouver to San Francisco, and Vancouver to Florida. His hope is to hit all 50 states with future rides. For the second half of this years July tour, the crew has already gone from Boston to the Cape and Marthas Vineyard, but will continue on via ferry to Rhode Island and Connecticut. From there, the crew will ferry to the east end of Long Island and into New York City to meet people from the transplant center there. Finally, the riders will head to the childrens hospital in Philadelphia.

In past years, the rides have been more family and friends oriented, according to Falkenberg. But this year, he said that the rides have been more open to participation. From this, he added, they have gotten a larger response and have already raised $100,000 for this year, three times as much as last years raised funds. This is just the start, as he expects they will double all raised funds from this year next year.

Funds raised go toward adding more donors to the Be The Match registry, research to ease the safety of transplant procedures, as well as financial assistance for families and patients in need of transplants and in post-transplant recovery. For adding donors, there is a cost to do the human leukocyte antigen (HLA) tests in order to add donors to the registry and match them to patients. Funds for researching the transplant procedures include identifying and preventing issues that can impact chance of survival, which has increased from 30% to closer to 50% in the past 13 years, according to Falkenberg. For many people, the journey of treatment and recovery can even be such a financial toll it can prevent patients from moving forward with potentially life-saving procedures if careers are put on hold, decreasing household income, says BTM in an information sheet.

Leukemia is the number one childhood cancer, so a lot of this is for kids. When the parents travel, they have to stay there for a long time sometimes, Falkenberg says, which takes time away from work and puts financial strain on family support. There are also financial grants through BTM that increase this family and patient support while covering costs that insurance will not.

Money is not the only thing that decreases the success of patient survival, as there has grown to be a disparity in the diversity of donors. According to BTM, out of almost 300,000 potential U.S. donors added to the registry last year, only 31% were ethnically diverse. Falkenberg commented on this issue saying, Theres about 20 million people on the registry, but if youre Black you only have about a 29% chance right now of finding a single donor on the registry because there just arent enough Black donors and its tied to your DNA and ethnicity. Falkenberg also said that there is a similar struggle for Asian or Pacific Islander patients, Hispanic or Latino patients, and Native American patients, though not quite as bad as the odds for Black or African American patients.

Elle Crofton, a first year rider diagnosed with a blood cancer nine years ago, works as an advocate for BTM alongside Falkenberg and spoke to The Times about this issue of ethnic disparity in donors. Like Falkenberg, Crofton was able to find multiple full matches on the BTM registry that allowed her to get a transplant seven years ago, but said, For people who are not white, they have a lot less likelihood of finding a match. She added that the team is trying to get the word out to get more people of color on the registry saying, We hope we can make the need for everyone to have that equal ability to find a match smaller.

Beyond volunteering and riding, Falkenberg and Crofton have begun legislative advocacy work, lobbying congress to provide legal support to donors. The two reached out to Joe Neguse, the U.S. representative for Colorados 2nd congressional district for support. Falkenberg and Crofton had a virtual meeting surrounding their current work on a Life Saving Leave Act, to which Neguse co-sponsored the next day. The act would work to allow 40 hours of non-consecutive time off of work and protect workers from being fired while undergoing the donation process. This process includes a physical exam and an injection to increase stem cell production, for which the travel and donation can take up to two days to complete. So while not a paid leave, the act would mitigate the fear for potential donors to lose their job.

Alongside the act, Be The Match reimburses for associated costs with the donation process. Its common sense stuff, just not the law right now, Falkenberg said and added, Unfortunately, the people that are more likely to say Im worried about losing my job also line up with the groups underrepresented on the registry. So, every donor that donates matters.

To donate, become a donor and join the registry, or find out other ways to support the organization visit BeTheMatch.org. Eligibility to become a donor is met if you meet the health guidelines and are between 18 and 40 years old. For registration, completion of a health history form and a swab of cheek cells is needed. The swab kit is mailed to the registrants home.

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Blood, sweat, and many miles for cellular therapy - Martha's Vineyard Times

Inhibition of pancreatic EZH2 restores progenitor insulin in T1D donor | Signal Transduction and Targeted Therapy – Nature.com

Human samples

Rapid harvesting of cadaveric pancreatic tissues was obtained with informed consent from next of kin, from heart-beating, brain-dead donors, with research approval from the Human Research Ethics Committee at St Vincents Hospital, Melbourne. Pancreas from individuals without and with diabetes, islet, acinar and ductal samples were obtained as part of the research consented tissues through the National Islet Transplantation Programme (at Westmead Hospital, Sydney and the St Vincents Institute, Melbourne, Australia), HREC Protocol number: 011/04. The donor characteristics of islet cell donor isolations are presented in Table 1.

Islets were purified by intraductal perfusion and digestion of the pancreases with collagenase AF-1.24 (SERVA/Nordmark, Germany) followed by purification using Ficoll density gradients.25 Purified islets, from low-density gradient fractions and acinar/ductal tissue, from high-density fractions, were cultured in Miami Media 1A (Mediatech/Corning 98021, USA) supplemented with 2.5% human serum albumin (Australian Red Cross, Melbourne, VIC, Australia), in a 37C, 5% CO2 incubator.

Total RNA from human ex vivo pancreatic cells was isolated using TRIzol (Invitrogen) and RNeasy Kit (QIAGEN) including a DNase treatment. First-strand cDNA synthesis was performed using a high-capacity cDNA Reverse Transcription Kit (Applied Biosystems) according to the manufacturers instructions. cDNA primers were designed using oligoperfect designer (Thermo Fisher Scientific), as shown in Table 2. Briefly, quantitative RT-PCR analyses were undertaken using the PrecisionFast 2 qPCR Master Mix (Primerdesign) and primers using Applied Biosystems 7500 Fast Real-Time PCR System. Each qPCR reaction contained: 6.5l qPCR Master Mix, 0.5l of forward and reverse primers, 3.5l H2O and 2l of previously synthesised cDNA, diluted 1/20. Expression levels of specific genes were tested and normalised to 18s ribosomal RNA housekeeping gene.

Modification of Histone H3 and histone-associated Ezh2 protein signals were quantified in human pancreatic ductal epithelial cells (AddexBio) by the LI-COR Odyssey assay. The cells were treated with 5 or 10M of GSK 126 (S7061, Selleckchem) for 48h. Histones and their associated proteins were examined using an acid extraction and immunoblotting as described previously.18 Protein concentrations were determined using Coomassie Reagent (Sigma) with BSA as a standard. Equal amounts (3g) of acid extract were separated by Nu-PAGE (Invitrogen), transferred to a PVDF membrane (Immobilon-FL; Millipore) and then probed with antibodies against H3K27me3 (07449, Millipore), H3K27ac (ab4729, Abcam), H3K9me3 (ab8898, Abcam), H3K9me2 (ab1220, Abcam), H3K4me3 (39159, Active Motif), Ezh2 (#4905, Cell Signaling Technology), and total histone H3 (#14269, Cell Signaling Technology). Protein blotting signals were quantified by an infra-red imaging system (Odyssey; LI-COR). Modification of Histone H3 and histone-associated Ezh2 signals were quantified using total histone H3 signal as a loading control.

Chromatin immunoprecipitation assays in human exocrine cells were performed previously described.26,27 Cells were fixed for 10min with 1% formaldehyde and quenched for 10min with glycine (0.125M) solution. Fixed cells were resuspended in sodium dodecyl (lauryl) sulfate (SDS) lysis buffer (1% SDS, 10mM EDTA, 50mM Tris-HCl pH 8.1) including a protease inhibitor cocktail (Roche Diagnostics GmBH, Mannheim, Germany) and homogenised followed by incubation on ice for 5min. Soluble samples were sonicated to 200600bp and chromatin was resuspended in ChIP Dilution Buffer (0.01% SDS, 1.1% Triton X-100, 1.2mM EDTA, 16.7mM Tris-HCl pH 8.0, and 167mM NaCl) and 20l of Dynabeads Protein A (Invitrogen, Carlsbad, CA, USA) was added and pre-cleared. H3K27me3 antibody was used for immunoprecipitation of chromatin and incubated overnight at 4C as previously described.28 Immunoprecipitated DNA were collected by magnetic isolation, washed low salt followed by high salt buffers and eluted with 0.1M NaHCO3 with 1% SDS. Protein-DNA cross-links were reversed by adding Proteinase K (Sigma, St. Louis, MO, USA) and incubation at 62C for 2h. DNA was recovered using a Qiagen MinElute column (Qiagen Inc., Valencia, CA, USA). H3K27me3 content at the promoters of the INS, INS-IGF2, NGN3 and PDX1 genes were assessed by qPCR using primers designed from the integrative ENCODE resource.29 ChIP primers are shown in Table 3.

Insulin and glucagon localisation in human islets were assessed using paraffin sections (5m thickness) of human pancreas tissue fixed in 10% neutral-buffered formalin and stained with hematoxylin and eosin (H&E) or prepared for immunohistochemistry. Insulin and glucagon were detected using Guinea Pig anti-insulin (1/100, DAKO) or mouse anti-glucagon (1/50) mAbs (polyclonal Abs, Sigma-Aldrich).

Pharmacological inhibition of EZH2, human pancreatic exocrine cells were kept untreated or stimulated with 10M GSK-126 (S7061, Selleckchem) at a cell density of 1105 per well for 24h. After 24h of treatment, fresh Miami Media was added to the cells, which were treated again with 10 GSK-126 and cultured for a further 24h. All cell incubations were performed in Miami Media 1A (Mediatech/Corning 98-021, USA) supplemented with 2.5% human serum albumin (Australian Red Cross, Melbourne, VIC, Australia), in a cell culture incubator at 37C in an atmosphere of 5% CO2 for 48h using non-treated six-well culture plates (Corning).

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Inhibition of pancreatic EZH2 restores progenitor insulin in T1D donor | Signal Transduction and Targeted Therapy - Nature.com

Bristol Myers Squibb Receives Positive CHMP Opinion Recommending Approval for LAG-3-Blocking Antibody Combination Opdualag (nivolumab and relatlimab)…

PRINCETON, N.J.--(BUSINESS WIRE)--Bristol Myers Squibb (NYSE: BMY) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval of the fixed-dose combination of nivolumab and relatlimab for the first-line treatment of advanced (unresectable or metastatic) melanoma in adults and adolescents 12 years of age and older with tumor cell PD-L1 expression < 1%. The European Commission (EC), which has the authority to approve medicines for the European Union (EU), will now review the CHMP opinion.

We are very proud of the role we have played in progressing the treatment of advanced melanoma over the years. As part of our mission to deliver new medicines for patients, we have continued to develop new dual immunotherapy combinations, said Paul Basciano, development lead, relatlimab, Bristol Myers Squibb. This positive CHMP opinion marks the first step toward the potential approval of the first LAG-3 blocking antibody combination and the third distinct checkpoint inhibitor for BMS for advanced melanoma patients in the EU.

The positive opinion is based upon efficacy and safety results from the Phase 2/3 RELATIVITY-047 trial. The trial showed that treatment with the fixed-dose combination of nivolumab and relatlimab more than doubled the median progression-free survival (PFS), including in patients with tumor cell PD-L1 expression < 1%, when compared to nivolumab monotherapy an established standard of care. The proposed indication for the EU is based upon an exploratory analysis of the data in patients with tumor cell PD-L1 expression < 1%. No new safety events were identified with the combination when compared to nivolumab monotherapy.

On March 18, 2022, the U.S. Food and Drug Administration (FDA) approved the fixed-dose combination of nivolumab and relatlimab as Opdualag (nivolumab and relatlimab-rmbw) for the treatment of adult and pediatric patients 12 years of age or older with unresectable or metastatic melanoma. Please see important safety information from the U.S. prescribing information below.

Bristol Myers Squibb thanks the patients and investigators involved in the RELATIVITY-047 trial.

About RELATIVITY-047

RELATIVITY-047 is a global, randomized, double-blind Phase 2/3 study evaluating the fixed-dose combination of nivolumab and relatlimab versus nivolumab alone in patients with previously untreated metastatic or unresectable melanoma. Patients were enrolled regardless of tumor cell PD-L1 expression. The trial excluded patients with active autoimmune disease, medical conditions requiring systemic treatment with moderate or high dose corticosteroids or immunosuppressive medications, uveal melanoma, and active or untreated brain or leptomeningeal metastases. The primary endpoint of the trial is progression-free survival (PFS) determined by Blinded Independent Central Review (BICR) using Response Evaluation Criteria in Solid Tumors (RECIST v1.1) in the all-comer population. The secondary endpoints are overall survival (OS) and objective response rate (ORR) in the all-comer population. A total of 714 patients were randomized 1:1 to receive a fixed-dose combination of nivolumab (480 mg) and relatlimab (160 mg) or nivolumab (480 mg) by intravenous infusion every four weeks until disease progression, unacceptable toxicity or withdrawal of consent.

About LAG-3

Lymphocyte-activation gene 3 (LAG-3) is a cell-surface molecule expressed on effector T cells and regulatory T cells (Tregs) and functions to control T-cell response, activation and growth. Preclinical studies indicate that inhibition of LAG-3 may restore effector function of exhausted T cells and potentially promote an anti-tumor response. Early research demonstrates that targeting LAG-3 in combination with other potentially complementary immune checkpoints may be a key strategy to more effectively potentiate anti-tumor immune activity.

Bristol Myers Squibb is evaluating relatlimab, its LAG-3-blocking antibody, in clinical trials in combination with other agents in a variety of tumor types.

About Melanoma

Melanoma is a form of skin cancer characterized by the uncontrolled growth of pigment-producing cells (melanocytes) located in the skin. Metastatic melanoma is the deadliest form of the disease and occurs when cancer spreads beyond the surface of the skin to other organs. The incidence of melanoma has been increasing steadily for the last 30 years. In the United States, 106,110 new diagnoses of melanoma and about 7,180 related deaths are estimated for 2021. Globally, the World Health Organization estimates that by 2035, melanoma incidence will reach 424,102, with 94,308 related deaths. Melanoma can be mostly treatable when caught in its very early stages; however, survival rates can decrease as the disease progresses.

Bristol Myers Squibb: Creating a Better Future for People with Cancer

Bristol Myers Squibb is inspired by a single vision transforming patients lives through science. The goal of the companys cancer research is to deliver medicines that offer each patient a better, healthier life and to make cure a possibility. Building on a legacy across a broad range of cancers that have changed survival expectations for many, Bristol Myers Squibb researchers are exploring new frontiers in personalized medicine, and through innovative digital platforms, are turning data into insights that sharpen their focus. Deep scientific expertise, cutting-edge capabilities and discovery platforms enable the company to look at cancer from every angle. Cancer can have a relentless grasp on many parts of a patients life, and Bristol Myers Squibb is committed to taking actions to address all aspects of care, from diagnosis to survivorship. Because as a leader in cancer care, Bristol Myers Squibb is working to empower all people with cancer to have a better future.

OPDUALAG U.S. INDICATION

Opdualag (nivolumab and relatlimab-rmbw) is indicated for the treatment of adult and pediatric patients 12 years of age or older with unresectable or metastatic melanoma.

OPDUALAG IMPORTANT SAFETY INFORMATION

Severe and Fatal Immune-Mediated Adverse Reactions

Immune-mediated adverse reactions (IMARs) listed herein may not include all possible severe and fatal immune-mediated adverse reactions.

IMARs which may be severe or fatal, can occur in any organ system or tissue. IMARs can occur at any time after starting treatment with a LAG-3 and PD-1/PD-L1 blocking antibodies. While IMARs usually manifest during treatment, they can also occur after discontinuation of Opdualag. Early identification and management of IMARs are essential to ensure safe use. Monitor patients closely for symptoms and signs that may be clinical manifestations of underlying IMARs. Evaluate clinical chemistries including liver enzymes, creatinine, and thyroid function at baseline and periodically during treatment. In cases of suspected IMARs, initiate appropriate workup to exclude alternative etiologies, including infection. Institute medical management promptly, including specialty consultation as appropriate.

Withhold or permanently discontinue Opdualag depending on severity (please see section 2 Dosage and Administration in the accompanying Full Prescribing Information). In general, if Opdualag requires interruption or discontinuation, administer systemic corticosteroid therapy (1 to 2 mg/kg/day prednisone or equivalent) until improvement to Grade 1 or less. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Consider administration of other systemic immunosuppressants in patients whose IMARs are not controlled with corticosteroid therapy. Toxicity management guidelines for adverse reactions that do not necessarily require systemic steroids (e.g., endocrinopathies and dermatologic reactions) are discussed below.

Immune-Mediated Pneumonitis

Opdualag can cause immune-mediated pneumonitis, which may be fatal. In patients treated with other PD-1/PD-L1 blocking antibodies, the incidence of pneumonitis is higher in patients who have received prior thoracic radiation. Immune-mediated pneumonitis occurred in 3.7% (13/355) of patients receiving Opdualag, including Grade 3 (0.6%), and Grade 2 (2.3%) adverse reactions. Pneumonitis led to permanent discontinuation of Opdualag in 0.8% and withholding of Opdualag in 1.4% of patients.

Immune-Mediated Colitis

Opdualag can cause immune-mediated colitis, defined as requiring use of corticosteroids and no clear alternate etiology. A common symptom included in the definition of colitis was diarrhea. Cytomegalovirus infection/reactivation has been reported in patients with corticosteroid-refractory immune-mediated colitis. In cases of corticosteroid-refractory colitis, consider repeating infectious workup to exclude alternative etiologies.

Immune-mediated diarrhea or colitis occurred in 7% (24/355) of patients receiving Opdualag, including Grade 3 (1.1%) and Grade 2 (4.5%) adverse reactions. Colitis led to permanent discontinuation of Opdualag in 2% and withholding of Opdualag in 2.8% of patients.

Immune-Mediated Hepatitis

Opdualag can cause immune-mediated hepatitis, defined as requiring the use of corticosteroids and no clear alternate etiology.

Immune-mediated hepatitis occurred in 6% (20/355) of patients receiving Opdualag, including Grade 4 (0.6%), Grade 3 (3.4%), and Grade 2 (1.4%) adverse reactions. Hepatitis led to permanent discontinuation of Opdualag in 1.7% and withholding of Opdualag in 2.3% of patients.

Immune-Mediated Endocrinopathies

Opdualag can cause primary or secondary adrenal insufficiency, hypophysitis, thyroid disorders, and Type 1 diabetes mellitus, which can be present with diabetic ketoacidosis. Withhold or permanently discontinue Opdualag depending on severity (please see section 2 Dosage and Administration in the accompanying Full Prescribing Information).

For Grade 2 or higher adrenal insufficiency, initiate symptomatic treatment, including hormone replacement as clinically indicated. In patients receiving Opdualag, adrenal insufficiency occurred in 4.2% (15/355) of patients receiving Opdualag, including Grade 3 (1.4%) and Grade 2 (2.5%) adverse reactions. Adrenal insufficiency led to permanent discontinuation of Opdualag in 1.1% and withholding of Opdualag in 0.8% of patients.

Hypophysitis can present with acute symptoms associated with mass effect such as headache, photophobia, or visual field defects. Hypophysitis can cause hypopituitarism; initiate hormone replacement as clinically indicated. Hypophysitis occurred in 2.5% (9/355) of patients receiving Opdualag, including Grade 3 (0.3%) and Grade 2 (1.4%) adverse reactions. Hypophysitis led to permanent discontinuation of Opdualag in 0.3% and withholding of Opdualag in 0.6% of patients.

Thyroiditis can present with or without endocrinopathy. Hypothyroidism can follow hyperthyroidism; initiate hormone replacement or medical management as clinically indicated. Thyroiditis occurred in 2.8% (10/355) of patients receiving Opdualag, including Grade 2 (1.1%) adverse reactions. Thyroiditis did not lead to permanent discontinuation of Opdualag. Thyroiditis led to withholding of Opdualag in 0.3% of patients. Hyperthyroidism occurred in 6% (22/355) of patients receiving Opdualag, including Grade 2 (1.4%) adverse reactions. Hyperthyroidism did not lead to permanent discontinuation of Opdualag. Hyperthyroidism led to withholding of Opdualag in 0.3% of patients. Hypothyroidism occurred in 17% (59/355) of patients receiving Opdualag, including Grade 2 (11%) adverse reactions. Hypothyroidism led to the permanent discontinuation of Opdualag in 0.3% and withholding of Opdualag in 2.5% of patients.

Monitor patients for hyperglycemia or other signs and symptoms of diabetes; initiate treatment with insulin as clinically indicated. Diabetes occurred in 0.3% (1/355) of patients receiving Opdualag, a Grade 3 (0.3%) adverse reaction, and no cases of diabetic ketoacidosis. Diabetes did not lead to the permanent discontinuation or withholding of Opdualag in any patient.

Immune-Mediated Nephritis with Renal Dysfunction

Opdualag can cause immune-mediated nephritis, which is defined as requiring use of steroids and no clear etiology. In patients receiving Opdualag, immune-mediated nephritis and renal dysfunction occurred in 2% (7/355) of patients, including Grade 3 (1.1%) and Grade 2 (0.8%) adverse reactions. Immune-mediated nephritis and renal dysfunction led to permanent discontinuation of Opdualag in 0.8% and withholding of Opdualag in 0.6% of patients.

Withhold or permanently discontinue Opdualag depending on severity (please see section 2 Dosage and Administration in the accompanying Full Prescribing Information).

Immune-Mediated Dermatologic Adverse Reactions

Opdualag can cause immune-mediated rash or dermatitis, defined as requiring use of steroids and no clear alternate etiology. Exfoliative dermatitis, including Stevens-Johnson syndrome, toxic epidermal necrolysis, and Drug Rash with eosinophilia and systemic symptoms has occurred with PD-1/L-1 blocking antibodies. Topical emollients and/or topical corticosteroids may be adequate to treat mild to moderate non-exfoliative rashes.

Withhold or permanently discontinue Opdualag depending on severity (please see section 2 Dosage and Administration in the accompanying Full Prescribing Information).

Immune-mediated rash occurred in 9% (33/355) of patients, including Grade 3 (0.6%) and Grade 2 (3.4%) adverse reactions. Immune-mediated rash did not lead to permanent discontinuation of Opdualag. Immune-mediated rash led to withholding of Opdualag in 1.4% of patients.

Immune-Mediated Myocarditis

Opdualag can cause immune-mediated myocarditis, which is defined as requiring use of steroids and no clear alternate etiology. The diagnosis of immune-mediated myocarditis requires a high index of suspicion. Patients with cardiac or cardio-pulmonary symptoms should be assessed for potential myocarditis. If myocarditis is suspected, withhold dose, promptly initiate high dose steroids (prednisone or methylprednisolone 1 to 2 mg/kg/day) and promptly arrange cardiology consultation with diagnostic workup. If clinically confirmed, permanently discontinue Opdualag for Grade 2-4 myocarditis.

Myocarditis occurred in 1.7% (6/355) of patients receiving Opdualag, including Grade 3 (0.6%), and Grade 2 (1.1%) adverse reactions. Myocarditis led to permanent discontinuation of Opdualag in 1.7% of patients.

Other Immune-Mediated Adverse Reactions

The following clinically significant IMARs occurred at an incidence of <1% (unless otherwise noted) in patients who received Opdualag or were reported with the use of other PD-1/PD-L1 blocking antibodies. Severe or fatal cases have been reported for some of these adverse reactions: Cardiac/Vascular: pericarditis, vasculitis; Nervous System: meningitis, encephalitis, myelitis and demyelination, myasthenic syndrome/myasthenia gravis (including exacerbation), Guillain-Barr syndrome, nerve paresis, autoimmune neuropathy; Ocular: uveitis, iritis, and other ocular inflammatory toxicities can occur. Some cases can be associated with retinal detachment. Various grades of visual impairment, including blindness, can occur. If uveitis occurs in combination with other IMARs, consider a Vogt-Koyanagi-Haradalike syndrome, as this may require treatment with systemic steroids to reduce the risk of permanent vision loss; Gastrointestinal: pancreatitis including increases in serum amylase and lipase levels, gastritis, duodenitis; Musculoskeletal and Connective Tissue: myositis/polymyositis, rhabdomyolysis (and associated sequelae including renal failure), arthritis, polymyalgia rheumatica; Endocrine: hypoparathyroidism; Other (Hematologic/Immune): hemolytic anemia, aplastic anemia, hemophagocytic lymphohistiocytosis, systemic inflammatory response syndrome, histiocytic necrotizing lymphadenitis (Kikuchi lymphadenitis), sarcoidosis, immune thrombocytopenic purpura, solid organ transplant rejection.

Infusion-Related Reactions

Opdualag can cause severe infusion-related reactions. Discontinue Opdualag in patients with severe or life-threatening infusion-related reactions. Interrupt or slow the rate of infusion in patients with mild to moderate infusion-related reactions. In patients who received Opdualag as a 60-minute intravenous infusion, infusion-related reactions occurred in 7% (23/355) of patients.

Complications of Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

Fatal and other serious complications can occur in patients who receive allogeneic hematopoietic stem cell transplantation (HSCT) before or after being treated with a PD-1/PD-L1 receptor blocking antibody. Transplant-related complications include hyperacute graft-versus-host disease (GVHD), acute GVHD, chronic GVHD, hepatic veno-occlusive disease after reduced intensity conditioning, and steroid-requiring febrile syndrome (without an identified infectious cause). These complications may occur despite intervening therapy between PD-1/PD-L1 blockade and allogeneic HSCT.

Follow patients closely for evidence of transplant-related complications and intervene promptly. Consider the benefit versus risks of treatment with a PD-1/PD-L1 receptor blocking antibody prior to or after an allogeneic HSCT.

Embryo-Fetal Toxicity

Based on its mechanism of action and data from animal studies, Opdualag can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with Opdualag for at least 5 months after the last dose of Opdualag.

Lactation

There are no data on the presence of Opdualag in human milk, the effects on the breastfed child, or the effect on milk production. Because nivolumab and relatlimab may be excreted in human milk and because of the potential for serious adverse reactions in a breastfed child, advise patients not to breastfeed during treatment with Opdualag and for at least 5 months after the last dose.

Serious Adverse Reactions

In Relativity-047, fatal adverse reaction occurred in 3 (0.8%) patients who were treated with Opdualag; these included hemophagocytic lymphohistiocytosis, acute edema of the lung, and pneumonitis. Serious adverse reactions occurred in 36% of patients treated with Opdualag. The most frequent serious adverse reactions reported in 1% of patients treated with Opdualag were adrenal insufficiency (1.4%), anemia (1.4%), colitis (1.4%), pneumonia (1.4%), acute myocardial infarction (1.1%), back pain (1.1%), diarrhea (1.1%), myocarditis (1.1%), and pneumonitis (1.1%).

Common Adverse Reactions and Laboratory Abnormalities

The most common adverse reactions reported in 20% of the patients treated with Opdualag were musculoskeletal pain (45%), fatigue (39%), rash (28%), pruritus (25%), and diarrhea (24%).

The most common laboratory abnormalities that occurred in 20% of patients treated with Opdualag were decreased hemoglobin (37%), decreased lymphocytes (32%), increased AST (30%), increased ALT (26%), and decreased sodium (24%).

Please see U.S. Full Prescribing Information for OPDUALAG.

OPDIVO U.S. INDICATIONS

OPDIVO (nivolumab), as a single agent, is indicated for the treatment of adult patients with unresectable or metastatic melanoma.

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the treatment of adult patients with unresectable or metastatic melanoma.

OPDIVO (nivolumab) is indicated for the adjuvant treatment of adult patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.

OPDIVO (nivolumab), in combination with platinum-doublet chemotherapy, is indicated as neoadjuvant treatment of adult patients with resectable (tumors 4 cm or node positive) non-small cell lung cancer (NSCLC).

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab) and 2 cycles of platinum-doublet chemotherapy, is indicated for the first-line treatment of adult patients with metastatic or recurrent non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

OPDIVO (nivolumab) is indicated for the treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving OPDIVO.

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable malignant pleural mesothelioma (MPM).

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the first-line treatment of adult patients with intermediate or poor risk advanced renal cell carcinoma (RCC).

OPDIVO (nivolumab), in combination with cabozantinib, is indicated for the first-line treatment of adult patients with advanced renal cell carcinoma (RCC).

OPDIVO (nivolumab) is indicated for the treatment of adult patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.

OPDIVO (nivolumab) is indicated for the treatment of adult patients with classical Hodgkin lymphoma (cHL) that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin or after 3 or more lines of systemic therapy that includes autologous HSCT. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

OPDIVO (nivolumab) is indicated for the treatment of adult patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum-based therapy.

OPDIVO (nivolumab) is indicated for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

OPDIVO (nivolumab), as a single agent, is indicated for the adjuvant treatment of adult patients with urothelial carcinoma (UC) who are at high risk of recurrence after undergoing radical resection of UC.

OPDIVO (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric (12 years and older) patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the treatment of adult patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

OPDIVO (nivolumab) is indicated for the treatment of adult patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy.

OPDIVO (nivolumab) is indicated for the adjuvant treatment of completely resected esophageal or gastroesophageal junction cancer with residual pathologic disease in adult patients who have received neoadjuvant chemoradiotherapy (CRT).

OPDIVO (nivolumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of adult patients with unresectable advanced or metastatic esophageal squamous cell carcinoma (ESCC).

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable advanced or metastatic esophageal squamous cell carcinoma (ESCC).

OPDIVO (nivolumab), in combination with fluoropyrimidine- and platinum- containing chemotherapy, is indicated for the treatment of adult patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma.

IMPORTANT SAFETY INFORMATION

Severe and Fatal Immune-Mediated Adverse Reactions

Immune-mediated adverse reactions listed herein may not include all possible severe and fatal immune-mediated adverse reactions.

Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue. While immune-mediated adverse reactions usually manifest during treatment, they can also occur after discontinuation of OPDIVO or YERVOY. Early identification and management are essential to ensure safe use of OPDIVO and YERVOY. Monitor for signs and symptoms that may be clinical manifestations of underlying immune-mediated adverse reactions. Evaluate clinical chemistries including liver enzymes, creatinine, adrenocorticotropic hormone (ACTH) level, and thyroid function at baseline and periodically during treatment with OPDIVO and before each dose of YERVOY. In cases of suspected immune-mediated adverse reactions, initiate appropriate workup to exclude alternative etiologies, including infection. Institute medical management promptly, including specialty consultation as appropriate.

Withhold or permanently discontinue OPDIVO and YERVOY depending on severity (please see section 2 Dosage and Administration in the accompanying Full Prescribing Information). In general, if OPDIVO or YERVOY interruption or discontinuation is required, administer systemic corticosteroid therapy (1 to 2 mg/kg/day prednisone or equivalent) until improvement to Grade 1 or less. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Consider administration of other systemic immunosuppressants in patients whose immune-mediated adverse reactions are not controlled with corticosteroid therapy. Toxicity management guidelines for adverse reactions that do not necessarily require systemic steroids (e.g., endocrinopathies and dermatologic reactions) are discussed below.

Immune-Mediated Pneumonitis

OPDIVO and YERVOY can cause immune-mediated pneumonitis. The incidence of pneumonitis is higher in patients who have received prior thoracic radiation. In patients receiving OPDIVO monotherapy, immune-mediated pneumonitis occurred in 3.1% (61/1994) of patients, including Grade 4 (<0.1%), Grade 3 (0.9%), and Grade 2 (2.1%). In patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg every 3 weeks, immune-mediated pneumonitis occurred in 7% (31/456) of patients, including Grade 4 (0.2%), Grade 3 (2.0%), and Grade 2 (4.4%). In patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg every 3 weeks, immune-mediated pneumonitis occurred in 3.9% (26/666) of patients, including Grade 3 (1.4%) and Grade 2 (2.6%). In NSCLC patients receiving OPDIVO 3 mg/kg every 2 weeks with YERVOY 1 mg/kg every 6 weeks, immune-mediated pneumonitis occurred in 9% (50/576) of patients, including Grade 4 (0.5%), Grade 3 (3.5%), and Grade 2 (4.0%). Four patients (0.7%) died due to pneumonitis.

In Checkmate 205 and 039, pneumonitis, including interstitial lung disease, occurred in 6.0% (16/266) of patients receiving OPDIVO. Immune-mediated pneumonitis occurred in 4.9% (13/266) of patients receiving OPDIVO, including Grade 3 (n=1) and Grade 2 (n=12).

Immune-Mediated Colitis

OPDIVO and YERVOY can cause immune-mediated colitis, which may be fatal. A common symptom included in the definition of colitis was diarrhea. Cytomegalovirus (CMV) infection/reactivation has been reported in patients with corticosteroid-refractory immune-mediated colitis. In cases of corticosteroid-refractory colitis, consider repeating infectious workup to exclude alternative etiologies. In patients receiving OPDIVO monotherapy, immune-mediated colitis occurred in 2.9% (58/1994) of patients, including Grade 3 (1.7%) and Grade 2 (1%). Inpatients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg every 3 weeks, immune-mediated colitis occurred in 25% (115/456) of patients, including Grade 4 (0.4%), Grade 3 (14%) and Grade 2 (8%). In patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg every 3 weeks, immune-mediated colitis occurred in 9% (60/666) of patients, including Grade 3 (4.4%) and Grade 2 (3.7%).

Immune-Mediated Hepatitis and Hepatotoxicity

OPDIVO and YERVOY can cause immune-mediated hepatitis. In patients receiving OPDIVO monotherapy, immune-mediated hepatitis occurred in 1.8% (35/1994) of patients, including Grade 4 (0.2%), Grade 3(1.3%), and Grade 2 (0.4%). In patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg every 3 weeks, immune-mediated hepatitis occurred in 15% (70/456) of patients, including Grade 4 (2.4%), Grade 3 (11%), and Grade 2(1.8%). In patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg every 3 weeks, immune-mediated hepatitis occurred in 7% (48/666) of patients, including Grade 4 (1.2%), Grade 3 (4.9%), and Grade 2 (0.4%).

OPDIVO in combination with cabozantinib can cause hepatic toxicity with higher frequencies of Grade 3 and 4ALT and AST elevations compared to OPDIVO alone. Consider more frequent monitoring of liver enzymes as compared to when the drugs are administered as single agents. In patients receiving OPDIVO and cabozantinib, Grades 3 and 4 increased ALT or AST were seen in 11% of patients.

Immune-Mediated Endocrinopathies

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Bristol Myers Squibb Receives Positive CHMP Opinion Recommending Approval for LAG-3-Blocking Antibody Combination Opdualag (nivolumab and relatlimab)...

Krabbe disease, which mostly affects newborns causes, symptoms, and treatment – CNBCTV18

Krabbe disease is one of many hundreds of inherited metabolic disorders. Named after the Danish neurologist Knud Krabbe, the disease causes progressive damage to the nervous system, eventually resulting in the death of the individual. The disease is common in newborns before they reach six months of age and treatment must start at the earliest. Most newborns affected by Krabbe disease do not reach the age of two.

Krabbe disease is caused due to genetic mutation on the 14th chromosome in an infant. A child needs to inherit two copies of the abnormal genome from both its parents, after which it has a 25 percent chance of inheriting both the recessive genes and developing the disease.

On inheriting the defective genome, the body doesnt produce enough of the enzyme galactosylceramidase (GALC). Galactosylceramidase is essential for breaking down unmetabolised lipids like glycosphingolipid and psychosine in the brain. These unmetabolised lipids are toxic to some of the non-neuron cells present in the brain.

Late-onset Krabbe disease, however, can be caused by a different genetic mutation which leads to a lack of a different enzyme, known as active saposin A.

Symptoms between early-onset and late-onset Krabbe disease differ slightly. Infants suffering from early-onset Krabbe disease suffer from symptoms like excessive irritability, difficulty swallowing, vomiting, unexplained fevers, and partial unconsciousness. Other common neuropathic symptoms include hypersensitivity to sound, muscle weakness, slowing of mental and motor development, spasticity, deafness, optic atrophy, optic nerve enlargement, blindness, and paralysis.

Late-onset Krabbe disease emerges with symptoms like the development of cross-eyes, slurred speech, slow development, and loss of motor functions.

The disease is diagnosed after a physician conducts a primary physical exam. A blood or skin tissue biopsy can test for GALC levels in the body and low levels can indicate the presence of Krabbe disease. Further testing through imaging scans (MRI), nerve conduction studies, eye examination, genetic testing and amniocentesis can also help diagnose the disease.

There is no cure for Krabbe disease. Treatment is mostly palliative in nature with a focus towards dealing with symptoms and providing supportive care. Experimental trials using hematopoietic stem cell transplant (HSCT), bone marrow transplantation, stem cell therapy, and gene therapy have seen some results in the small number of patients that they have been used on.

(Edited by : Shoma Bhattacharjee)

First Published:Jul 15, 2022, 06:32 AM IST

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Krabbe disease, which mostly affects newborns causes, symptoms, and treatment - CNBCTV18

Gut bacteria nurture the immune system for cancer patients, a diverse microbiome can protect against dangerous treatment complications – The…

One promising treatment for patients with blood cancers is stem cell transplantation. Doctors completely eliminate the patients immune system by aiming chemotherapy, radiation or both at their bone marrow before replacing it with a donors immune system. Because the bone marrow produces blood and immune cells, completely substituting cancerous bone marrow with healthy cells could help the body reestablish a functioning immune system and replace cancerous blood cells.

This procedure is not without risks. A key complication hematologists like me worry about is graft-versus-host disease, where the donors immune system recognizes the patients body as foreign and launches an attack. Up to 50% of patients who receive a stem cell transplant develop graft-versus-host disease.

One unexpected part of the body that may play a key role in protecting transplant patients from complications, however, is their gut bacteria.

Alongside my colleagues Hana Andrlova and Marcel van den Brink, I study how the composition of your microbiome, or the microorganisms living in your body, can affect how well cancer treatments work. While previous studies have shown that disruptions to the diversity of organisms in the gut microbiome is linked to a higher risk of death after transplantation, the precise reasons for this are not clear.

In our recently published study, we found that gut bacteria help the immune system recover from stem cell transplants by nurturing two special types of immune cells that protect against complications.

To explore the relationship between gut bacteria and the immune system, we first needed to identify the types of bacteria present in a given microbiome. So we sequenced all the bacterial genes in the stool samples of 174 stem cell transplant patients. We then took blood samples from the same patients to identify which types of immune cells were circulating and how they were functioning.

We learned that a diverse intestinal microbiome after transplantation is associated with expansion of a particular type of cell called MAIT, or mucosal-associated invariant T cells. MAIT cells are linked to improved transplant outcomes like a lower risk of graft-versus-host disease and longer survival in both mice and people. We found that the more MAIT cells patients had in their blood after transplant, the longer they survived and the fewer their complications. Patients with the highest levels of MAIT cells had the lowest incidence of graft-versus-host disease.

The precise mechanism behind the protective effects of MAIT cells is unclear. But researchers do know that these cells require molecules that come from the process of producing riboflavin, or vitamin B2, in the body to develop and multiply. Turns out, these riboflavin derivatives are produced by the microbes in the gut.

We also found that high MAIT cell numbers were linked to the presence of another special population of T cells, V-delta-2, that are also stimulated by bacterial byproducts. Above-average levels of these cells were also associated with better survival and less graft-versus-host disease in transplant patients.

These findings suggest that one of the reasons why a healthy, diverse microbiome is linked to good results for stem cell transplant recipients could be that gut bacteria support the development of immune cells that protect against transplant complications like graft-versus-host disease.

Our next step was to figure out how these special T cells protect against transplant complications. We took blood samples from five patients who had high numbers of MAIT and V-delta-2 cells. We then used a technique called single-cell RNA sequencing to analyze thousands of individual cells and explore all the potential functions any particular cell type may have in the body.

When we compared the MAIT and V-delta-2 cells of transplant patients and healthy people, our findings were very surprising. We had originally hypothesized that genes linked with tissue repair would be active in these T cells that would explain why patients with high numbers of these cells do better after such intense treatment thats so tough on the body. Instead, we found that these cells had highly expressed genes involved in inflammatory processes with the capacity to induce cell damage sometimes necessary to fight off infections when the patients immune system is still recovering. This suggests that MAIT and V-delta-2 cells may be protecting patients from transplant complications in ways that we havent previously been aware of or understood.

Its possible that T cells that are activated by the microbiome like MAIT and V-delta-2 help reduce transplant complications by killing infected cells or cells involved in graft-versus-host disease. While we arent able to confirm this hypothesis with our study, future work may help scientists better understand the important links between the microbiome, the immune system and successful stem cell transplants for cancer patients.

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Gut bacteria nurture the immune system for cancer patients, a diverse microbiome can protect against dangerous treatment complications - The...

Global Induced Pluripotent Stem Cell (iPSC) Market Report 2022: Rising Applications of iPSCs Fueling Industry Growth – ResearchAndMarkets.com -…

DUBLIN--(BUSINESS WIRE)--The "Global Induced Pluripotent Stem Cell (iPSC) Industry Report, 2022" report has been added to ResearchAndMarkets.com's offering.

Since the discovery of induced pluripotent stem cell (iPSC) technology in 2006, significant progress has been made in stem cell biology and regenerative medicine. New pathological mechanisms have been identified and explained, new drugs identified by iPSC screens are in the pipeline, and the first clinical trials employing human iPSC-derived cell types have been initiated.

iPSCs can be used to explore the causes of disease onset and progression, create and test new drugs and therapies, and treat previously incurable diseases.

Other applications of iPSCs include their use as research products, as well as their integration into 3D bioprinting, tissue engineering, and clean meat production. Technology allowing for the mass-production and differentiation of iPSCs in industrial-scale bioreactors is also advancing at breakneck speed.

iPSC Derived Clinical Trials

The first clinical trial using iPSCs started in 2008, and today, that number has surged worldwide. Most of the current clinical trials do not involve the transplant of iPSCs into humans, but rather, the creation and evaluation of iPSC lines for clinical purposes. Within these trials, iPSC lines are created from specific patient populations to determine if these cell lines could be a good model for a disease of interest.

The therapeutic applications of induced pluripotent stem cells (iPSCs) have also surged in recent years. Since the discovery of iPSCs in 2006, it took only seven years for the first iPSC-derived cell product to be transplanted into a human patient in 2013. Since then, iPSC-derived cells have been used within a rapidly growing number of preclinical studies, physician-led studies, and formal clinical trials worldwide.

Key Topics Covered:

1. Report Overview

2. Introduction

3. Current Status of iPSC Industry

3.1 Progress Made in Autologous Cell Therapy Using iPSCs

3.2 Manufacturing Timeline for Autologous iPSC-Derived Cell Products

3.3 Cost of iPSC Production

3.4 Automation in iPSC Production

3.5 Allogeneic iPSCs Gaining Momentum

3.6 Share of iPSC-Based Research Within the Overall Stem Cell Industry

3.7 Major Focus Areas of iPSC Companies

3.8 Commercially Available iPSC-Derived Cell Types

3.9 Relative Use of iPSC-Derived Cell Types in Toxicology Testing Assays

3.10 Currently Available iPSC Technologies

4. History of Induced Pluripotent Stem Cells (iPSCs)

5. Research Publications on iPSCs

6. iPSC: Patent Landscape Analysis

6.1 Legal Status of iPSC Patents

6.2 Patents by Assignee Organization Type

6.3 Ownership of Patent Families by Assignee Type

6.4 Top Inventors of iPSC Patents

6.5 Top Ten iPSC Inventors

6.6 Most Cited Five iPSC Patents

6.7 Leading Patent Filing Jurisdictions

6.8 Number of Patent Families by Year of Filing

6.9 Patents Representing Different Disorders

6.10 iPSC Patents on Preparation Technologies

6.11 Patents on Cell Types Differentiated from iPSCs

6.12 Patent Application Trends Disease-Specific Technologies

7. iPSC: Clinical Trial Landscape

7.1 Literature and Database Search

7.2 Number of iPSC Clinical Trials by Year

7.3 iPSC Study Designs

7.4 iPSC-Based Clinical Trials With Commercialization Potential

8. Research Funding for iPSCs

8.1 Value of NIH Funding for iPSC Research

8.2 Partial List of NIH Funded iPSC Research Projects in 2022

9. M&A, Collaborations & Funding Activities in iPSC Sector

10. Generation of Induced Pluripotent Stem Cells: An Overview

10.1 Reprogramming Factors

10.2 Integrating iPSC Delivery Methods

10.3 Non-Integrative Delivery Systems

10.4 Comparison of Delivery Methods for Generating iPSCs

10.5 Genome Editing Technologies in iPSC Generation

11. Human iPSC Banking

11.1 Cell Sources for iPSC Banking

11.2 Reprogramming Methods Used in iPSC Banking

11.3 Factors Used in Reprogramming in Different Banks

11.4 Workflow in iPSC Banks

11.5 Existing iPSC Banks

12. Biomedical Applications of iPSCs

12.1 iPSCs in Basic Research

12.2 iPSCs in Drug Discovery

12.3 iPSCs in Toxicology Studies

12.4 iPSCs in Disease Modeling

12.5 iPSCs in Cell-Based Therapies

12.6 Other Novel Applications of iPSCs

12.7 iPSCs in Animal Conservation

13. Market Overview

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/mg6l5h

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Global Induced Pluripotent Stem Cell (iPSC) Market Report 2022: Rising Applications of iPSCs Fueling Industry Growth - ResearchAndMarkets.com -...

Ola Landgren, MD, PhD, Highlights How DETERMINATION Trial Results Inform Use of RVd/Transplant in Newly Diagnosed Myeloma – Cancer Network

C. Ola Landgren, MD, PhD, a professor and leader of Experimental Therapeutics and Myeloma Service at the Sylvester Comprehensive Cancer Center, University of Miami Health System, in an interview with CancerNetwork highlighted key efficacy findings from the phase 3 DETERMINATION trial (NCT01208662) assessing the use of lenalidomide (Revlimid), bortezomib (Velcade), and dexamethasone (RVd) plus autologous stem cell transplant vs RVd alone, both with continuous lenalidomide maintenance, in patients with newly diagnosed multiple myeloma.1 Moreover, he highlights how the findings compare with similar research such as the phase 3 IFM/DFCI2009 trial (NCT01191060) which previously assessed RVd alone or with high-dose transplant followed by 1 year of lenalidomide maintenance in newly diagnosed multiple myeloma.2

Patients treated on DETERMINATION who received RVd alone had a median progression-free survival (PFS) of 46.2 months compared with 67.5 months in the transplant group (HR, 1.53; 95% CI, 1.23-1.91; P <.0001). The rates of partial response or better were 95.0% and 97.5% in each respective group. No overall survival benefit was noted in either arm (HR, 1.10; 95% CI, 0.73-1.65; P = .99).

Transcript:

The DETERMINATION study showed very similar [findings to the phase 3 IFM/DFCI2009 trial in] that there is a progression-free survival benefit following bone marrow transplant; it was found to be around 21 months. Thats a quite long time. But also, they showed that there is no survival difference [between the 2 treatment arms]. The follow-up time is only around 5 years in the DETERMINATION trial, which is slightly shorter [than IFM/DFCI2009] but confirms very similar results.

Another very important difference between the 2 studies was that in the DETERMINATION study, of the patients on the non-transplant arm [who progressed], a much lower proportion of those patients went to transplant [vs IFM/DFCI2009]. In the DETERMINATION study, it was in the range of 20% to 25% while in the IFM/DFCI2009 study it was 70% to 80%. Despite the fact that there were fewer patients who went to transplant at the time of relapse in the non-transplant up-front arm, you still see no survival difference. Of course, this raises the question [as to whether] you need to do a transplant upfront, do you need to delay it, or do you never need to do the transplant?

This is exactly what Joseph Mikhael, MD, [of the Translational Genomics Research Institute], talked about as the discussant at ASCO. He made a very good, balanced, and fair evaluation when he said that you can make a case for transplant. If you want to extend PFS, you can make a case against the transplant not showing survival difference. There were a lot of other nuances, [such as] the onset of second malignancies. There were 10 cases of [acute myeloid leukemia and myelodysplastic syndrome] in the transplant arm and none in the non-transplant arm. There were quality-of-life differences in favor of no transplant; patients had several months of worsening as expected of their quality of life [following] transplant. Mikhael summarize saying, Welcome to the future of myelomathe era of choice. It is no longer mandatory for patients to do transplant. And I agree with that.

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Ola Landgren, MD, PhD, Highlights How DETERMINATION Trial Results Inform Use of RVd/Transplant in Newly Diagnosed Myeloma - Cancer Network

CAR T-Cell Therapy Appeared Safe, With No Signs of GVHD in Patients With T-Cell Lymphoma/Leukemia – DocWire News

A study presented at the 2022 American Society of Clinical Oncology Annual Meeting found that an autologous CD7 chimeric antigen receptor (CAR) T-cell therapy was effective for patients with relapsed/refractory T-cell acute lymphoblastic leukemia and lymphoma (ALL/LBL), with no signs of graft-versus-host disease (GVHD) reported.

The phase I study included patients with CD7+ relapsed/refractory T-cell ALL/LBL with no leukemic cells in the peripheral blood. Following a 3+3 dose escalation process, the CD7 CAR construct included an endoplasmic reticulum anchor domain fused to a CD7 binding domain to prevent CD7 expression on cell surface, which contributed to minimizing CAR T-cell fratricide. CAR T product was checked to ensure lack of tumor contamination before infusion.

Between September 2021 and January 2022, 5 patients (median age, 3.8 years; range, 1.9-13.0 years) were enrolled in the study. Of those patients, 1 had mediastinal mass and blasts in pleural fluid, 1 had central nervous system-3 status, and 3 had marrow disease with a median burden of 1.35% (range, 0.07%-7.31%).

Patients received CAR T-cell therapy at the following doses: 5 105 cells/kg (n = 3) and 1 106 cells/kg (n = 1). One patient received cells below the target dose.

A total of 3 patients had cytokine release syndrome (CRS), and 1 patient experienced grade 3 CRS. Median onset to CRS was 5 days (range, 1-9 days), with a median duration of 4 days (range, 3-14 days). There were no reports of neurotoxicity, GVHD, or infection.

All patients experienced grade 3/4 hematologic toxicities, which recovered to grade 2 within 30 days.

At 1 month post-infusion, 4 patients achieved complete remission, and 1 patient still had leukemia cells in the cerebrospinal fluid. At a median follow-up of 62 days (range, 35-136 days), 1 patient underwent hematopoietic stem cell transplantation (HSCT) at 2.9 months post-infusion and had a CD7 relapse at 1.4 months post-HSCT. The other 3 patients who experienced a response were in minimal residual disease-negative complete remission.

In the 4 patients who received target dose, the median peak CAR T-cell count in peripheral blood was 4.27 102/L (range, 2.49-5.61) by flow cytometry. All patients had detectable CAR transgene by polymerase chain reaction at their last visits.

Longer follow-up with more patients is needed to further evaluate this CAR T-cell therapy, the researchers noted.

Zhao L, Pan J, Tang K, et al. Autologous CD7-targeted CAR T-cell therapy for refractory or relapsed T-cell acute lymphoblastic leukemia/lymphoma. Abstract #7035. Presented at the 2022 American Society of Clinical Oncology Annual Meeting; June 3-7, 2022; Chicago, IL.

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CAR T-Cell Therapy Appeared Safe, With No Signs of GVHD in Patients With T-Cell Lymphoma/Leukemia - DocWire News

New cancer treatment changing outlook for those with blood cancers – WBAL TV Baltimore

Ten percent of all diagnosed cancers in the United States are blood cancers and they can be deadly. There are exciting new treatments and research happening in Baltimore that are giving patients hope."These therapies cure the patients that have no other treatment options. It's been a remarkable breakthrough," Dr. Aaron Rapoport, of the University of Maryland School of Medicine, said.Cutting-edge technology in cancer treatment will treat many types of cancers such as leukemia, lymphoma, and myeloma. Traditional treatments include chemotherapy, radiation, and stem cell therapy, but what if those treatments don't work? Now there is an immunotherapy for aggressive blood cancers that is seeing remarkable results.Chip Baldwin has a big laugh and immense love for his grandchildren."This is Kyle, he's about 3 1/2 years old and he lives in Florida. (My) granddaughter Maple. She and her family live in Fells Point. And this is (my) granddaughter Rosemary and she's a doll, and they call me Pop-pop," Baldwin said.Baldwin almost never met two of his grandchildren. In January 2018, he was told chemotherapy was no longer working to treat his lymphoma. He thought it was the end."Leaving (my wife) Angela and leaving the family, trying to figure out how they're going to get by," Baldwin said. He was out of options, or so he thought. Not willing to give up, his wife, Angela Baldwin, began researching and came across a promising new treatment."Probably the last treatment that I could have received. Had I not received it and had it not been positive to put me in remission, I probably wouldn't be talking to you today," Baldwin said.The treatment he received had just been approved by the U.S. Food and Drug Administration (FDA) months earlier. It's called "CAR T-cell Therapy." It uses the patient's own, re-engineered, immune cells to kill cancer. Rapoport helped pioneer the development of CAR T-cell at the University of Maryland Greenebaum Comprehensive Cancer Center. Baldwin was just the second patient here to receive it."The notion that one could perhaps harness the immune system, or educate the immune system, to better protect us from cancer, but also to recognize and fight against cancer, has been a goal for decades - centuries really," Rapoport said.It appears that goal has been reached. Here's how it works: The medical team extracts immune cells, called T-cells, out of the patient's blood. The cells are sent to a special lab in California, where scientists change the cells' DNA to put receptors on them called "CAR" - Chimeric Antigen Receptors. They enable the immune cells to recognize, hunt down and kill the cancer cells. The California lab then sends the now-re-engineered immune cells back to the Greenebaum Comprehensive Cancer Center."These are CAR T-cells growing in the flask here. These are CAR T-cells that were made in the lab," Dr. Djordje Atanackovic of the University of Maryland Medical Center, said. Under a microscope you can see spots on a cancer cell - those spots are the killer CAR T-cells. "You could use these right now to treat a patient, actually," Atanackovic said.For the final step, patients are admitted to the hospital and the medical team puts the T-cells back into the patient, where they multiply by the millions and destroy the cancer. For Baldwin, that was the day after Easter 2018."And, then about four months later, they determined that all the cancer cells had died, " Baldwin said."Being told that their scans are negative is a really overwhelming experience, not just for the patients, but for the families and also the nurses and physicians. The team members that are involved in their care," Rapoport said.When looking at CT scan images of two other lymphoma patients, you see black areas in the images on one is extensive cancer. The other image shows the same patient after CAR T-cell therapy and the cancer is gone. Right now, CAR T-cell Therapy is approved to treat aggressive blood cancers Lymphoma, B-cell Leukemia and Myeloma. But Atanackovic believes that's just the beginning."I'm pretty optimistic that in 10 years from now we'll have novel immunotherapies that we can't even imagine at this point for everyone, or at least most of our patients with cancer," Atanackovic said.Four years after his treatment and Baldwin is still in remission. He doesn't like the word "cure" because he's afraid it's bad luck. The word he keeps coming back to is: "Unbelievable. And even to this day, I kind of can't believe I'm in remission and I'm able to live my life. Since then, I've had two grandchildren and it's been wonderful. Had it not been for the University and the treatment, I would never have seen the two kids," Baldwin saidSo far, 250 patients have been treated with CAR T-cell Therapy at the University of Maryland, but it's not perfect and researchers are still working to improve it. The success rate for patients with aggressive lymphoma for example is 50% and some patients have side effects like flu-like symptoms, so they typically stay in the hospital for days or even weeks.Many may be wondering is this covered by insurance? The answer is yes. Keep in mind, right now it is approved by FDA as a second-line therapy, so you do have to try a different treatment first. But, immunotherapy like CAR-T is the future of cancer treatment and you're going to see more of it.

Ten percent of all diagnosed cancers in the United States are blood cancers and they can be deadly. There are exciting new treatments and research happening in Baltimore that are giving patients hope.

"These therapies cure the patients that have no other treatment options. It's been a remarkable breakthrough," Dr. Aaron Rapoport, of the University of Maryland School of Medicine, said.

Cutting-edge technology in cancer treatment will treat many types of cancers such as leukemia, lymphoma, and myeloma. Traditional treatments include chemotherapy, radiation, and stem cell therapy, but what if those treatments don't work? Now there is an immunotherapy for aggressive blood cancers that is seeing remarkable results.

Chip Baldwin has a big laugh and immense love for his grandchildren.

"This is Kyle, he's about 3 1/2 years old and he lives in Florida. (My) granddaughter Maple. She and her family live in Fells Point. And this is (my) granddaughter Rosemary and she's a doll, and they call me Pop-pop," Baldwin said.

Baldwin almost never met two of his grandchildren. In January 2018, he was told chemotherapy was no longer working to treat his lymphoma. He thought it was the end.

"Leaving (my wife) Angela and leaving the family, trying to figure out how they're going to get by," Baldwin said.

He was out of options, or so he thought. Not willing to give up, his wife, Angela Baldwin, began researching and came across a promising new treatment.

"Probably the last treatment that I could have received. Had I not received it and had it not been positive to put me in remission, I probably wouldn't be talking to you today," Baldwin said.

The treatment he received had just been approved by the U.S. Food and Drug Administration (FDA) months earlier. It's called "CAR T-cell Therapy." It uses the patient's own, re-engineered, immune cells to kill cancer.

Rapoport helped pioneer the development of CAR T-cell at the University of Maryland Greenebaum Comprehensive Cancer Center. Baldwin was just the second patient here to receive it.

"The notion that one could perhaps harness the immune system, or educate the immune system, to better protect us from cancer, but also to recognize and fight against cancer, has been a goal for decades - centuries really," Rapoport said.

It appears that goal has been reached. Here's how it works:

The medical team extracts immune cells, called T-cells, out of the patient's blood. The cells are sent to a special lab in California, where scientists change the cells' DNA to put receptors on them called "CAR" - Chimeric Antigen Receptors. They enable the immune cells to recognize, hunt down and kill the cancer cells. The California lab then sends the now-re-engineered immune cells back to the Greenebaum Comprehensive Cancer Center.

"These are CAR T-cells growing in the flask here. These are CAR T-cells that were made in the lab," Dr. Djordje Atanackovic of the University of Maryland Medical Center, said.

Under a microscope you can see spots on a cancer cell - those spots are the killer CAR T-cells.

"You could use these right now to treat a patient, actually," Atanackovic said.

For the final step, patients are admitted to the hospital and the medical team puts the T-cells back into the patient, where they multiply by the millions and destroy the cancer. For Baldwin, that was the day after Easter 2018.

"And, then about four months later, they determined that all the cancer cells had died, " Baldwin said.

"Being told that their scans are negative is a really overwhelming experience, not just for the patients, but for the families and also the nurses and physicians. The team members that are involved in their care," Rapoport said.

When looking at CT scan images of two other lymphoma patients, you see black areas in the images on one is extensive cancer. The other image shows the same patient after CAR T-cell therapy and the cancer is gone.

Right now, CAR T-cell Therapy is approved to treat aggressive blood cancers Lymphoma, B-cell Leukemia and Myeloma. But Atanackovic believes that's just the beginning.

"I'm pretty optimistic that in 10 years from now we'll have novel immunotherapies that we can't even imagine at this point for everyone, or at least most of our patients with cancer," Atanackovic said.

Four years after his treatment and Baldwin is still in remission. He doesn't like the word "cure" because he's afraid it's bad luck.

The word he keeps coming back to is: "Unbelievable. And even to this day, I kind of can't believe I'm in remission and I'm able to live my life. Since then, I've had two grandchildren and it's been wonderful. Had it not been for the University and the treatment, I would never have seen the two kids," Baldwin said

So far, 250 patients have been treated with CAR T-cell Therapy at the University of Maryland, but it's not perfect and researchers are still working to improve it.

The success rate for patients with aggressive lymphoma for example is 50% and some patients have side effects like flu-like symptoms, so they typically stay in the hospital for days or even weeks.

Many may be wondering is this covered by insurance? The answer is yes. Keep in mind, right now it is approved by FDA as a second-line therapy, so you do have to try a different treatment first. But, immunotherapy like CAR-T is the future of cancer treatment and you're going to see more of it.

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New cancer treatment changing outlook for those with blood cancers - WBAL TV Baltimore