Since their first successful derivation in 1998, human embryonic stem cells have received almost unprecedented attention. Hailed as the next revolution for medicine, they have been described as the future of molecular biology and the biggest development since recombinant DNA.1 It has been predicted that their successful derivation will have a more profound impact on health than even the advent of anaesthesia and the development of antibiotics.2 They are set to create a whole new genre of medical therapies.3 Their potential availability has also, however, opened a Pandoras box of ethical dilemmas, ranging from ongoing issues surrounding the moral status of the human embryo to the conflicting claims of alternative stem cell sources. Although integral to ethical discourse, these dilemmas demand understanding and assessment on scientific grounds. It is our contention that the ethical debate is being hindered by failure to appreciate the subtleties of the scientific background.

Since the ethical problems accompanying destruction of human embryos are well recognised, the advantages of bypassing these by employing adult stem cells are obvious. For many, the ethical conflicts would be avoided, while all the potential benefits to patients with severe diseases would be retained. Consequently, perceived ethical problems would be resolved if it could be demonstrated that adult stem cells are superior to embryonic stem cells as therapeutic agents.

Unfortunately, resolution is far from clear, for this research field is in its infancy. Scientific uncertainty abounds, and yet societies are demanding definitive scientific answers on stem cell technology. Since the least controversial course of action would be to use adult stem cells, the pressures on scientists to emerge with evidence demonstrating that their potential is equal to, or even greater than, that of embryonic stem cells are formidable. Scientific data and interpretation have become integral to the ethical debate, perhaps in inappropriate ways.

An understanding of the most fundamental aspects of stem cell identity and function is required, from the identification of stem cells to the role of environmental factors at both the microscopic and macroscopic levels. Recognising the role of environmental factors has ramifications both clinically and ethically. Acknowledgement of these factors will provide for greater understanding of the obstacles that have to be overcome if the clinical potential of stem cells is to be realised. It will also help clarify the notions of totipotency and pluripotency, concepts central to delineating the moral value of embryonic stem cells and their parent blastocysts.

Stem cells are unspecialised cells, which have the ability to renew themselves indefinitely, and under appropriate conditions can give rise to a variety of mature cell types in the human body. Some stem cells can give rise to a wide range of mature cell types, whereas others give rise to only a few. Stem cells can be derived from a variety of sources including early embryos, fetal tissue, and some adult tissues, of which bone marrow and blood are the best known examples. Hence, there are two populations of stem cells: embryonic and adult stem cells. Of these, embryonic stem cells are derived from the inner cell mass (ICM) of the blastocyst at five to seven days after fertilisation. At this point the blastocyst has differentiated into two cell types, ICM cells (some of which will give rise to the future individual) and the surrounding trophectoderm cells (which will later form the placenta).

The distinction between embryonic and adult stem cells raises the issue of accurate identification, a prerequisite to testing the claims frequently made for the abilities of both embryonic and adult stem cells to produce a wide array of cell and tissue types. Scientifically, the problem is a fundamental one: defining stem cells solely on the basis of their structurethat is, the specific markers they carry on their outer surfaces, is inaccurate and potentially misleading. Identification mayfor example, be complicated by some stem cells expressing markers from several kinds of lineages and may be further confused by the possibility that marker expression changes throughout development.4,5 The potential for misidentification is of considerable importance for the scientific community, which has called for functional as well as structural testing.

Placing far more reliance on the functional properties of stem cells opens up a wider debate, namely, the role of the environment in an understanding of stem cell function. The ability of the structure of stem cells to change points to the existence of a dynamic relationship between stem cells and their immediate microenvironment, the stem cell niche.

The niche concept was first developed in blood cells, where proliferation, differentiation, and survival of distinct progenitor populations were found to be dependent on factors secreted by other cell types.6 This microenvironment is characterised by numerous external signals, including those derived from chemical factors, cell/cell interactions, and relationships between cells and the surrounding tissue.6 These, in their various ways, all have an impact on stem cells, affecting the precise directions in which they subsequently develop.

This microenvironment is governed by regulatory mechanisms, the molecular nature of which is complicated and elusive. Schuldiner et al,7 in their study of the effects of eight growth factors on the capacity of human embryonic stem cells to form other cell types, found that while these factors altered developmental outcome, they did not produce uniform differentiation of the stem cells. Consequently, although the structural markers and functions of stem cells appear to be dependent upon their environment, defining the nature of this environment will be far from straightforward.

An increasing awareness of the role of the niche on stem cell structure and function has led to an evolving concept of the stem cell. For instance, there is now the suggestion that stem cells should be viewed, not as undifferentiated cells, but as appropriately differentiated cells with the potential to display diverse cell types in alternative niches.8 An excellent illustration of this point is provided in a recent study by Wu et al9 where human neural stem cells were primed in a cocktail of chemical factors and then implanted into various regions of the adult rat brain. Not only did the implanted stem cells give rise to a larger number of neurons than previously reported, but most significantly they gave rise to different neuronal types depending upon the region of the brain into which they were grafted. It is possible that the distinctive nature of the local environment in each brain region instructed the neural stem cells to adopt such different fates.

Furthermore, stem cells taken out of their original niche and exposed to an entirely new environment can potentially differentiate into the cell type(s) typical of that new environment. Human neural stem cellsfor example, produced muscle cells when introduced into skeletal muscle10 and human bone marrow cells differentiated into neural cells when transplanted into a neural environment.11 The above two studies were carried out in rodents, but more recently Mezey et al12 have demonstrated that a similar scenario is possible in humans. Following bone marrow transplants in patients with various forms of cancer, bone marrow stem cells entered the brain and generated neural cell types including neurons. In many of these studies, where stem cells have been transformed into cells from different lineages, there has been some form of injury to the stem cells new environment or niche. In light of this, it is possible that various factors, signals, or chemicals normally present in damaged or disrupted tissue may play a role in governing stem cell fate.

The above findings reflect the increasing influence being attributed to environmental factors, acknowledgement of which has led to the view that stem cells are dynamic rather than static entities. This view underpins the concept of stem cell plasticity, whereby stem cells from adult sources have the ability to dedifferentiate or redifferentiate into cells from other lineages. This may blur the absolute distinction so frequently made between embryonic and adult stem cells (let alone between specific types of adult stem cells), a determinative factor in much ethical debate.

Adult stem cells include stem cells from bone marrow, blood, fat, and both fetal and adult organs. Plasticity is particularly characteristic of bone marrow. Stem cells from this source can differentiate into neural cells,11,1315 (see above for further discussion) while other research has indicated that such cells can be incorporated into skeletal muscle.16

While these reports indicate that interest in the potential of adult stem cells is justified, they should be interpreted cautiously. It would be unwise to jump to the conclusion that these studies render the use of embryonic stem cells (with destruction of human embryos) unnecessary. There are a number of reasons for this.

First, accurate identification is a prerequisite for determining the presence and extent of plasticity. For instance, although Jackson et al17 presented data to suggest that a group of muscle cells could turn into blood cells, they later found they were dealing with a subpopulation of cells that normally reside in muscle tissue.18 What is required are more rigorous standards for determining stem cell plasticity.1921 Iffor example, cardiac cells developed from stem cells are to contribute to heart function, they would have to demonstrate synchronous contraction within the heart itself. Similarly, neural cells derived from neural stem cells would have to generate electrical impulses and release and respond to chemicals normally found within the brain.19,20

A second issue concerns frequency of occurrence. Failure to replicate previous experimental work showing that blood cells are capable of differentiating into neural cells, suggests that, if transformations are occurring, they are very rare.22 Consistent with this conclusion is the work of Jackson et al,23 who demonstrated plasticity in human blood stem cells, although the change to the desired heart and blood vessel cells occurred in only 0.02% of cells. Thus, as Winston24 notes, even in apparently rich sources, the cells capable of change may be very few in number, and this may ultimately diminish their therapeutic value.

A third point of concern with clinical applications in mind, is that transformations may occur via hybrid cells, that is, by the fusion of two distinct cell types. Such spontaneous fusion was observed when embryonic stem cells were grown in the laboratory in the presence of neural cells25 or bone marrow cells.26 Such hybrids, however, show chromosomal abnormalities that may preclude them from being used in therapeutic applications.

The apparent formation of such hybrid cells may have important implications for interpretations of stem cell plasticity. Such a phenomenon presents an alternate explanation for the claims that stem cells from one tissue type are able to produce the progeny of another tissue typethat is, bone marrow into muscle, blood into brain, and vice versa. In other words, adult stem cells may not be as plastic as early reports have suggested. Thus, as pointed out by Ying et al25 future stem cell plasticity studies should ensure that any transformed cells are examined and tested to see if they display properties of both the original and the introduced cell types.

A final note of caution is that it has become clear that there is far more data to show that embryonic stem cells are capable of indefinite growth and pluripotency than adult stem cells. Mouse embryonic stem cellsfor example, have been renewing for 10 years,27 a capacity yet to be demonstrated in cells from adult sources. If adult cells have a restricted renewal potential, this will have negative implications for therapeutic applications, which rely on the ability to expand cells accurately in the laboratory in order to provide enough material for effective transplantation. Furthermore, embryonic stem cells exhibit high levels of the enzyme telomerase which indicates their immortality,28 whereas adult stem cells grown in the laboratory do not exhibit this in the same way. This property renders embryonic stem cells important in the study of cellular ageing and stem cell renewal. Work with neural stem cells from biopsies and autopsies suggests that embryonic stem cells may be easier to coax into specific cell types than adult stem cells.18

Overall, there are few confirmed reports of truly pluripotential adult human stem cells,3,29 while even apparently convincing reports30 may raise serious queries when assessed in a critical manner.3 Nearly a dozen teams have reported adult stem cell plasticity31 and it seems unlikely that random mutation or hybrid fusion can explain all these results. What is required is far more understanding of the fundamental biological issues raised by this research. Even as Winston24 outlines the advantages of embryonic stem cell researchfor example, he recognises the benefits of adult stem cells in regard to safety, possible efficacy, and accessibility. Adult sources have the added advantage of not requiring an intermediate embryo for immunocompatibility. Similarly, while the UK Department of Health32 argues that the therapeutic potential of embryonic stem cells outweighs that of adult stem cells, it acknowledges that in the long term both may be useful. The UK government reiterated this point in 2002 by stating that it wishes to advance research with stem cells from all sources.33

Scientifically, therefore, research with both adult and embryonic sources should continue, although caution should be exercised in evaluating the results. Currently, however, adult stem cells are more problematic than their embryonic counterparts. In light of this evaluation, considerable care should be employed in advocating on allegedly scientific grounds, the advantages of adult over embryonic cells as the source of replacement tissues. The impetus behind such a sentiment stems principally from a desire to protect the status of the human embryo than from any demonstrated superiority of adult stem cell sources.14,34

Confusion at this point will do nothing to advance the cause of ethical analysis, since the current state of the science and its likely future directions are integral to serious ethical assessment. In other words, it is short sighted to attempt to circumvent discussion of the moral status of the blastocyst by concentrating on the potential of adult stem cells alone. Until it is accepted that this latter approach is a cul de sac for ethical discourse, the imperatives of some ethicists will continue to come into conflict with current scientific perspectives.

It is generally asserted that totipotency denotes the ability of a cell or group of cells to give rise to a complete individual, whereas pluripotency refers to the capacity to give rise to all the cell types constituting the individualbut not the individual as a whole. Helpful as this distinction is, it is limited, in that it neither acknowledges nor emphasises the importance of environmental influences in defining these abilities.

As we have seen, embryonic stem cells are derived from the ICM of the blastocyst. These ICM cells have the capacity to form all three embryonic germ layers: endoderm, which will form the lungs, liver, and gut lining; mesoderm, which will form the bone, blood, and muscle, and ectoderm, which will form the skin, eyes, and nervous system. Outwardly, these cells appear to give rise to a complete individual and are considered by some to be totipotent.35

The claim of totipotency requires a number of conditions, however, whether this be for blastocysts or embryonic stem cells. The latter must be undifferentiated and, hence, capable of giving rise to all three germ layers, a condition that is met when embryonic stem cells are derived from the ICM of the blastocyst. In addition, there is a requirement for trophectoderm cells, which will eventually form the layers of the placenta. The extraembryonic tissues are a crucial source of signalling molecules and must function optimally for the differentiation of both embryonic somatic cells and for the establishment of germlines.36 Since both trophectoderm and ICM cells are required for successful development of the fetus, both cell types are required to establish totipotency.37 Thus, totipotency becomes a function of the immediate environment of the embryonic stem cell. If a viable fetus is to result, totipotency also requires successful implantation and development within the uterus of a woman.

In the absence of all these conditions embryonic stem cells are only pluripotent, since they are capable of creating all the cell lines of the fetus, but not the fetus itself. In the laboratory environment they are incapable of totipotency, since they have been removed from the context of the trophectoderm, let alone that of the uterus. It is inaccurate, therefore, to refer to embryonic stem cells as totipotent rather than pluripotent.38

These criteria for establishing totipotency also have ramifications for the ethical evaluation of the human blastocyst. While the blastocyst has intact trophectoderm cells and, therefore, the capacity to produce all three germ layers, plus the extraembryonic material necessary for its survival, totipotency is still dependent on the wider environmentsuccessful implantation in a uterus. Hence, blastocysts within the laboratory are only potentially totipotent, in contrast to their counterparts within the body.

A blastocyst or even a later embryo in the laboratory lacks the capacity to develop into a human individual. Unfortunately, this simple observation is frequently overlooked, and moral discussion focuses on the potential of an embryo to grow into a fully developed human without any reference to its context. Ignoring context in this manner inevitably overlooks the crucial importance of an appropriate environment necessary for the realisation of totipotency, changes to which may also alter the moral debate. Just as stem cell identity and arguably moral value depend upon the microenvironment, so too the human embryo is intimately dependent upon its wider environment.

Much opposition to the use of embryonic stem cells relies upon the argument that adult stem cells could serve as a viable source of tissues for regeneration and therapy. In the light of this, the argument continues that embryonic stem cells, with their debatable ethical credentials, should no longer feature in attempts to produce replacement tissues. This stance uses alleged scientific evidence to bolster an ethical position, and stands or falls on the strength of the scientific case.

Apart from the validity or otherwise of this approach, definitive evidence will not be forthcoming for some time (possibly years), since the scientific issues are complex on-going ones. As outlined above, the potential of adult stem cells remains a matter for debate and further experimentation. Additionally, the dynamic nature of stem cells, both embryonic and adult, points to a close interrelationship between their potential and the environment in which they are located. The possibility of cell lineage change also has to be taken into account when the suitability of different stem cell types is being advocated. From a scientific perspective none of this is surprising, and yet it fits uneasily alongside any stance that is a mixture of scientific, ethical, and political rhetoric.

The necessity of paying attention to the scientific framework of the debate, such as we are doing, has implications for other stances as well. With the advance of scientific understanding and, specifically, the advent of a genetic level of understanding, has come a tendency to view the life of an individual on the basis of DNA alone. This too, however, ignores the dependence of the embryo upon a competent environment. The context within which the embryo develops, like the niche for the stem cell, is integral to all aspects of its functioning. The environment provides nutritional requirements as well as numerous cues to ensure the healthy development of the embryo and subsequent fetus. Consequently, the preservation of DNA cannot be equated with the preservation of an individuals life, as has been suggested by McGee and Caplan.39 Adherence to such a reductionist mode of thinking is only made possible by ignoring completely the contribution of the environment. Essential as DNA is for development, it requires an appropriate context if its potential is to be realised.

From this it follows that a notion such as totipotency is a function of the environment both at the microscopic and macroscopic levels. This suggests that ethical debate cannot be reduced to potential for life, since inherent within the potential of an embryo is an assumption regarding the appropriateness of its environment. This means that the context of blastocysts and later embryos is crucial, ethically as well as scientifically and clinically.

In light of this, it is appropriate to ask whether it is useful to continue thinking of the blastocyst as an independent entity with a moral status stemming entirely from its organisation and perceived potential. We have argued that neither blastocysts nor stem cells are to be viewed in isolation from their context. Given that the claim is frequently made that moral value and status are closely associated with embryonic potential, recognition of the importance of the environment will have major implications for ethical thinking.

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Okarma TB. Human primordial stem cells. Hastings Cent Rep1999;29:30.

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Vogel G . Cell biology. Stem cells: new excitement, persistent questions. Science2000;290:16724.

Matsuoka SY, Ebihara Y, Xu M, et al. CD34 expression on long term repopulating hematopoietic stem cells changes during developmental stages. Blood2001;97:41925.

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Schuldiner MO, Yanuka O, Itskovitz-Eldor J, et al. From the cover: effects of eight growth factors on the differentiation of cells derived from human embryonic stem cells. Proc Natl Acad Sci USA2000;97:1130712.

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Wu P , Tarasenko YI, Gu Y, et al. Region specific generation of cholinergic neurons from fetal human neural stem cells grafted in adult rat. Nat Neurosci2002;5:12718.

Galli R , Borello U, Gritti A, et al. Skeletal myogenic potential of human and mouse neural stem cells. Nat Neurosci2000;3:98691.

Zhao LR, Duan WM, Reyes M, et al. Human bone marrow stem cells exhibit neural phenotypes and ameliorate neurological deficits after grafting into the ischemic brain of rats. Exp Neurol2002;174:1120.

Mezey E , Key S, Vogelsang G, et al. Transplanted bone marrow generates new neurons in human brains. Proc Natl Acad Sci USA2003;100:13649.

Brazelton TR, Rossi FM, Keshet GI, et al. From marrow to brain: expression of neuronal phenotypes in adult mice. Science2000;290:17759.

Kopen GC, Prockop DJ, Phinney DG. Marrow stromal cells migrate throughout forebrain and cerebellum, and they differentiate into astrocytes after injection into neonatal mouse brains. Proc Natl Acad Sci USA1999;96:107116.

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Ferrari G , Cusella-De Angelis G, Coletta M, et al. Muscle regeneration by bone marrow derived myogenic progenitors. Science1998;279:152830.

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Morshead CM, Benveniste P, Iscove NN, et al. Hematopoietic competence is a rare property of neural stem cells that may depend on genetic and epigenetic alterations. Nat Med2002;8:26873.

Jackson KA, Majka SM, Wang H, et al. Regeneration of ischemic cardiac muscle and vascular endothelium by adult stem cells. J Clin Invest2001;107:1395402.

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Ying QL, Nichols J, Evans EP, et al. Changing potency by spontaneous fusion. Nature2002;416:5458.

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As eerily relevant as 1992s Death Becomes Her is in 2022, there is an unexpected difference. Unlike Madeline (Meryl Streep) and Helen (Goldie Hawn), we arent hiding our facelifts. Instead, some of us are live streaming the whole experience. Dermal fillers and Botox are getting as common as getting a facial in your local salon.

"There has been a shift of mindset and increased acceptability of these procedures, says Dr Madhuri Agarwal of Yavana Aesthetics, Mumbai. In the next few years, the trend is going to be more innovations and better delivery mechanisms of these minimally invasive procedures that deliver long term, healthy skin.

What you want to do to look and feel good is not up for discussion. While lasers and acids are wonderful for skin texture and even tightening, a non-surgical facelift involving needles can be more effective for the latter. For example, filler that is more natural looking, because a laser isnt doing anything to make up for the lost volume.

Our bodies are dynamic and need maintenance as we age. Even, and especially, our facial skin. But with so many options of non-surgical face lifts available, it can be overwhelming to make a choice. We spoke to a few dermatologists to help break down the details of the best non-surgical facelift treatments involving needles.

Botox involves injecting a very safe neurotoxin called Botulinum to freeze muscles, and relax them, ironing out wrinkles. Wary but curious first timers can choose to start with very minute unitsthey wont erase all wrinkles but will smoothen them out enough to look a little more natural. I suggest this only when fine lines form, says Dr Kiran Sethi, a dermatologist based in Delhi and author of Skin Sense. It lasts 3-6 months, and there isnt much downtime. Its great when combined with fillers or skin boosters. Theres also been a focus on preventive Botox. If you get it done before the lines set in, you will have fewer lines as you age, explains Dr Geetika Mittal Gupta of ISAAC Luxe Clinic in Mumbai and Delhi. You will need less and less Botox as you age, because those muscles are not contracting as much. And by early I mean, when you see certain lines of ageing.

Fillers are usually injections of hyaluronic acid that add back lost volume to parts of your face. The Indian bone structure is such that our cheekbone is a little flat on the centre part of the face, explains Dr. Chytra V Anand, founder, Kosmoderma located in Chennai and Bengaluru. So most Indians, even teenagers, get dark circles and hollows. Its a loss of volume. So you have to put a filler in there. And people are accepting of that. Its not because they want to look like someone else, or they want to look younger. They just want to maintain their body and skin. The down time for fillers is usually 2-7 days, depending on how easily you bruise. And a good treatment can last anywhere between 1 and 2 years.

The vampire facial might have shocked people a few years ago, but today its one of the most popular treatments in India. Platelet-rich plasma is extracted from your blood, rich in growth hormones that renews blood flow and tissue regeneration wherever it is injected back, including your scalp. Its usually a course of 3-4 sessions, monthly, says Dr Sethi. It treats melasma, dehydration, has a mild filler effect too. And when used on the scalp, new hair growth can show in 6 months.

Theres also stem cell therapy for hair and skin rejuvenation. We take a small biopsy of the skin, splice the cells, and use the extract for regenerative therapy, says Dr Anand. It takes less time and commitment than PRP and is great for scar healing.

Its good to remember that these treatments are addictive too, says Dr Akber Aimer, Director of Aesthetic Medicine, Maya Medi Spa. You need to understand your limit. Always look for a good doctor who is experienced and talk about your problems and ask their opinions. Understand everything clearly. Your decision-making is a multi-step procedure. You need to have done proper research on the materials used and the treatment. Understand the technology. Trust your gut. And dont forget to ask for before and after pictures!

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While historically lacking in foreign investments, Japans biotech scene is thriving with global investors showing increasing interest. Here are five of the hottest Japanese private companies innovating in the healthcare space.

Japan boasts one of the highest life expectancies in the world, and, faced with a rapidly aging population, is witnessing a growing burden of chronic conditions including cardiovascular disease and type 2 diabetes. For this reason, the Japanese healthcare authorities are encouraging research into the treatment and prevention of these diseases, in addition to promoting the potential of regenerative medicine.

In addition to having a roster of healthcare giants including Takeda, Astellas Pharma and Eisai, Japan is also an Asian hotspot for biotech companies. Upcoming startups have historically been limited in foreign funding and reliant on local venture capital players such as Nippon Venture Capital, Shinsei Capital Partners, and the University of Tokyo Edge Capital Partners.

In 2021, however, the amount of foreign investment flowing into the Japanese biotech space rose to $98 million, almost triple the haul of previous years. The most prominent global backers included Newton Biocapital, F-Prime Capital, and SoftBank Group. This trend arose as the COVID-19 pandemic triggered a wave of investor enthusiasm in biotechnology around the world.

With the help of local experts, weve listed five of the hottest private biotech companies in Japan. These firms, shown in alphabetical order, have raised large funding rounds in the last two years and are developing innovative treatments for a range of conditions including cancer, cardiovascular disease and inflammatory disorders.

Source: Shutterstock

Founded: 2017

Headquarters: Fujisawa

Chordia Therapeutics derives its name from the English term chord referring to a collection of musical notes normally played in harmony. In a similar way, the company aims to work in harmony with stakeholders and collaborators to develop first-in-class small molecule treatments for cancer.

Chordias lead program is a drug that disrupts the processing of RNA in tumor cells. In a healthy cell, RNA molecules are typically transcribed from a DNA template and spliced together to guide the production of new proteins. Some cancer cells accumulate mutations in the RNA splicing machinery and become vulnerable to Chordias drugs that interfere with this process.

Chordia raised $31 million (4 billion yen) in a Series C round in May 2022. The aim of the round was to push the companys lead drug through phase I testing and fund the preclinical development of the rest of its pipeline.

This month, the company announced interim results from the phase I trial of its lead candidate, with four of the recruited patients so far showing signs of responding to the treatment.

Founded: 2015

Headquarters: Tokyo

Heart failure occurs when the heart muscle is irreparably damaged and is unable to pump blood. While this deadly condition can be treated with a heart transplant, there is a general shortage of donors available, making a pressing need for alternatives.

In June 2021, the stem cell therapy developer Heartseed raised $36.5 million (4 billion yen) in a Series C round. The mission is to provide a regenerative route to saving the heart via stem cell therapy.

In the lab, Heartseed reprograms skin cells from the patient into a type of stem cell called induced pluripotent stem cells and grows these stem cells into heart muscle cells. The company then injects the muscle cells as a small cluster, or seed, into heart tissue to repair the muscle.

The proceedings from its Series C round will allow Heartseed to take its lead candidate into clinical development, including a phase I/II trial scheduled for later this year. Last year, Heartseed also licensed its treatment to Novo Nordisk in Denmark to co-develop the treatment outside of Japan.

Founded: 2018

Headquarters: Tokyo

LUCA Science hit the headlines in the last week for raising an impressive $30.3 million (3.86 billion yen) in a Series B round. The company is developing an unusual approach for treating a wide range of diseases: delivering a therapy based on mitochondria, the energy production plants in human cells.

One example where the technology could work well is in strokes and heart attacks, where blood flow is blocked to critical tissue in the brain and heart respectively. The reperfusion of blood to these tissues after the blockage can kill the tissue by damaging its mitochondria. Delivering healthy mitochondria could keep the tissue working properly and protect it from harm.

LUCA Science plans to use its recent Series B winnings to accelerate the preclinical development of its mitochondrial therapies and establish its manufacturing process. In May 2022, the firm also inked a collaboration deal with compatriot pharmaceutical company Kyowa Kirin Co., Ltd. to co-develop a mitochondrial therapy for rare genetic diseases.

Founded: 2016

Headquarters: Boston, U.S., and Tokyo

Modulus Discovery is a preclinical-stage drug discovery specialist. The company focuses on developing small molecule treatments for conditions such as cancer, inflammatory disorders and rare genetic conditions.

The firm uses a mixture of strategies to speed up the drug discovery process. These include simulating target proteins using a supercomputer; structural protein biology; forming collaborations such as with the peptide drug expert PeptiDream; and tapping into global networks for biological expertise. Modulus most advanced drug program is in late-stage preclinical testing for the treatment of chronic inflammatory diseases.

In March 2022, Modulus bagged $20.4 million (2.34 billion yen) in a Series C round. The cash is earmarked to advance the companys R&D programs by expanding its infrastructure, collaborations and headcount.

Founded: 2015

Headquarters: Tokyo

The name Noile-Immune is derived from blending together the phrases no illness and no immunity, no life. This company is developing CAR-T cell therapies for the treatment of cancer, which traditionally consist of extracting the patients immune T cells, engineering them in the lab to hunt down cancer cells, and reinfusing them into the patient.

Unlike approved CAR-T cell therapies, which are limited to treating forms of blood cancer, Noile-Immune aims its therapies at treating solid tumors. The company does this by engineering immune T cells to produce proteins that cause immune cells to migrate into the tumor site.

Noile-Immune is testing its lead candidate in a phase I in patients with solid tumors. The firm is also co-developing therapies with partners including Takeda and the European cell therapy specialists Adaptimmune and Autolus. Additionally, Noile-Immune has an allogeneic version of its cell therapy in the pipeline where immune T cells are sourced from healthy donors rather than the patient.

To finance the clinical development of its lead candidate, Noile-Immune raised $21.8 million (2.38 billion yen) in a Series C round in early 2021. The company hit a setback in January 2022 when a collaboration deal fell through with the U.S. player Legend Biotech. Nonetheless, other external companies remain interested in Noile-Immunes offering, including Japan-based Daiichi Sankyo Company Ltd., which opted to assess Noile-Immunes technology in late 2021.

Cover image via Elena Resko.

Thanks to feedback from Shiohara Azusa, VC Investor at The University of Tokyo Edge Capital, and Hironoshin Nomura, Chief Financial Officer, Sosei Group Corporation.

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The last decade of immunotherapy progress was based on decades of prior research, including other forms of immunotherapy.

Until recent years, cancer treatment revolved around surgery, chemotherapy, and radiation. But the FDA approval of ipilimumab (Yervoy) in 2011 led to a fourth leg of that treatment stool: immunotherapy. This enabled new treatment paradigms, sometimes with shocking levels of success.

The types of immunotherapy treatments available are proliferating, with approved immune checkpoint inhibitors (ICIs) and cellular therapies like chimeric antigen receptor (CAR) T cells as well as other modalities in the research and discovery phases. Some even include more established approaches like vaccines that are being revisited with new information and iterations.

The last decade of immunotherapy progress was based on decades of prior research, including other forms of immunotherapy. The Bacillus Calmette-Gurin vaccine, used to prevent tuberculosis for a century, has also been used as an immunotherapy to treat nonmuscle invasive bladder cancer since 1990.1 And rituximab (Rituxan), a monoclonal antibody therapy approved in 1997 for B-cell malignancies, is seen by some as an early immunotherapy as well.2

What many clinicians think of in terms of immunotherapy, however, are treatments targeting CTLA-4 and PD-1/PD-L1 pathways, brought from the bench by James P. Allison, PhD, and Tasuku Honjo, PhD, respectively, leading to a Nobel Prize awarded jointly to them in 2018.3

Immune responses are tightly controlled by T cells, and these T cells have on/off switches that help control their responses, according to Padmanee Sharma, MD, PhD, a professor in the Department of Genitourinary Medical Oncology in the Division of Cancer Medicine and the scientific director of the James P. Allison Institute at The University of Texas MD Anderson Cancer Center in Houston. Previously, she said, clinicians were not aware of the off switches. Sharma showed that CTLA-4 was an inhibitory pathway and that by blocking it, the T cells could stay longer to eradicate the tumors.

With 8 ICIs approved for immunotherapy in hematological and solid tumors,4 researchers are not only investigating newer forms of therapy, but also combining them to fi nd more effective and durable treatments and introducing them into earlier lines of treatment (TIMELINE). Current research is also attempting to predict who will respond to which therapy based on current and emerging biomarkers.

Ipilimumab, which kicked off the current era of cancer immunotherapy treatment with FDA approval in 2011, targets CTLA-4 for newly diagnosed or previously treated unresectable or metastatic melanoma.5 Ipilimumab blocks CTLA-4, removing its inhibitory signals. This allows the T cells to activate and launch an immune response to the tumors antigens.

CTLA-4 is basically the fi rst inhibitory pathway that comes up on the T cells, Sharma said. CTLA-4 is a member of an immunoglobulin-related receptor family responsible for some immune regulation aspects of T cells.6 It is thought to regulate T-cell proliferation mostly in lymph nodes, early in an immune response, by having an inhibitory role.7

What ipilimumab really did and what the immune checkpoint inhibitors really did is they opened up this whole different way to approach the immune system, Elizabeth Buchbinder, MD, a medical oncologist at Dana-Farber Cancer Institute and an assistant professor of medicine at Harvard Medical School in Boston, Massachusetts, said. Ipilimumab provided amazing durable responses in patients with melanoma with widely metastatic disease, some of whom were alive 10 years later, she said.

The PD-1 and PD-L1 blockades build on ipilimumabs success. Like CTLA-4, PD-1 is a negative regulator of T-cell immune function, inhibiting the target to increase immune system activation. PD-1 suppresses T cells mostly in the peripheral tissues.7 As of November 2021, 8 ICIs have been approved that target CTLA-4, PD-1, and PD-L1 pathways and treat 18 types of cancer.3

AntiPD-1 inhibitors

The percentage of people who benefi tted from ipilimumab was on the low side, Buchbinder said, with only an 11% response rate and 20% of people doing well long term in clinical trials. With PD-1 inhibition, however, there was approximately a 40% response rate and many more patients doing well long term, as demonstrated in clinical trials. So [PD-1 inhibition is] both far more effective and also less toxic, Buchbinder said.

When choosing an agent in the PD-1 class, we dont need to differentiate them. Theyre all antiPD-1, Sharma explained. There arent any data to indicate that patients will respond any differently to pembrolizumab [Keytruda] vs nivolumab [Opdivo]. The mechanism of action for both drugs [is] exactly the same.

Instead, clinicians should consider the FDA approvals for each drugs indications and combinations. But from a scientific standpoint, theres no distinguishing between [them], Sharma said.

AntiPD-L1 inhibitors

PD-1 and PD-L1 targeting drugs were found to work beyond melanoma and kidney cancer, the early indications for treatments targeting the CTLA-4 pathway, Buchbinder said. That was a huge opening up of this fi eld to all of these other cancers, like lung cancer, head and neck cancer, GI [gastrointestinal] cancer, breast [cancer], and beyond, she said.

Before receiving these immunotherapies, patients may need to show PD-1 or PD-L1 expression, although this may not identify all patients who can benefi t from the treatments. Researchers continue to try to identify additional and better biomarkers to indicate which patients may respond.13

In March, the FDA approved the newest ICI, nivolumab and relatlimab-rmbw (Opdualag), for adult and pediatric patients (12 years and older) with unresectable or metastatic melanoma. 3 Nivolumab is a PD-1 inhibitor, and relatlimab blocks LAG3 proteins on immune cells. It is being tested in a lot of other tumors, Buchbinder noted.

Another target in the discovery phase is T cell immunoglobulin and mucin domain 3, which is a checkpoint receptor expressed by many immune cells and leukemic stem cells.14 It is activated by several ligands and is being tested in different cancer types.

Also in clinical trials are tumor-infiltrating lymphocytes (TIL) that recognize cancer cells as abnormal, entering the tumor to kill the cells. TILs already recognize the targets because they originate from the tumor itself.15 Although they need to be expanded, they are not the same as CAR T cells, which must be engineered to recognize the targets.

In addition, older therapies are experiencing a resurgence, with research underway to make interleukin 2 (IL-2) help cytokines function better. That work is trying to optimize what those cytokines do in the body and the immune system, Buchbinder said. There are so many areas where the goal of the therapy is activation of the immune system.

One of these areas includes a return to vaccines. In earlier vaccine therapy, We had no idea that while we were giving therapy to turn on the cells, we were also rapidly turning off the cells because an on switch will automatically drive an off switch for the immune system, Sharma said. The yin and the yang of the immune response is very important to understand because when the immune response is driven in one direction, it will always try to control itself. With that in mind, newer vaccines might work better if given in combination with an antiCTLA-4, for example, to block the inhibitory pathways, she said.

Vaccines are taking many forms, including the mRNA vaccine used for COVID-19, peptide vaccines that include a tiny bit of protein that is expected to be expressed on the tumor surface, and vaccines constructed from dendritic cells, which stimulate T cells, Buchbinder said.

There are also viral therapies injected directly into tumor vaccines, such as talimogene laherparepvec (Imlygic) approved in 2015 for the treatment of some patients with metastatic melanoma that cannot be surgically removed.16 It is a is a modifi ed herpes virus directly injected into the tumor to bring about a local immune response, Buchbinder said.

According to Sharma, approximately 60 targets are currently being evaluated for immunotherapy development.

The FDA has approved 2 CAR T-cell therapies, both in 2017: tisagenlecleucel (Kymriah) for patients 25 years and younger with relapsed B-cell precursor acute lymphoblastic leukemia17 and axicabtagene ciloleucel (Yescarta) for the treatment of adult patients with large B-cell lymphoma that is refractory to fi rst-line chemoimmunotherapy or that relapses within 12 months of fi rst-line chemoimmunotherapy.18 These treatments involve collecting T cells from the patient and engineering them to express CARs that recognize the patients cancer cells. The cells are then enlarged and infused back into the patient, where they can target the antigen- expressing cancer cells. CARs have been shown to greatly improve clinical response and disease remission in some patients.19

I think CAR T cells are clearly building on the concept that T cells are the soldiers of immune response. They are basically engineering the cell to have an antibody that recognizes a specifi c antigen, Sharma said, adding that its important to ensure the targeted antigen is part of the cancer.

CAR T cells have had limited effectiveness in treating solid tumors, given the low T-cell infiltration and immunosuppressive environment that challenges the immune system from successfully reaching and killing solid tumor cancer cells.20

Natural killer (NK) cells are another cell type being researched to attempt tumor eradication, and this therapy is in the early stages, according to Sharma. CAR NK cells can be generated from allogenic donors, making them more attractive as off the shelf treatments compared with CAR T cells, which are collected from the patient. As of early 2021, more than 500 CAR T-cell trials and 17 CAR T-cell/NK-cell trials were in the works globally.21

A major consideration when choosing any treatment, including immunotherapies, is the adverse event (AE) profile. Immunotherapy drugs have different AEs than oncology treatments like chemotherapy or radiation. [With immunotherapy,] what we see is infl ammation because youre turning on the immune system in such a powerful way, Sharma said. Inflammatory reactions include a skin rash or dermatitis, infl ammation in the colon (colitis and diarrhea), and/or infl ammation in the lung with pneumonitis. Clinicians are now aware of these AEs and can monitor them closely, stopping therapy if needed to control them before they become severe, Sharma said.

Toxicities with ipilimumab can be severe, and patients requiring hospital admission might need high-dose steroids, Buchbinder noted. Common AEs for the CTLA-4 inhibitor are typically GI related, including diarrhea, colitis, and hepatitis. Some patients may experience fatigue or a small rash, but most generally make it through treatment with minimal AEs.

The stronger AEs with ipilimumab can be seen from a trial comparing ipilimumab plus nivolumab to nivolumab and relatlimab. Almost 60% of patients experienced AEs with the ipilimumab combination vs 20% in the latter group.17

PD-1 and PD-L1 inhibition typically involve AEs that cause lung issues rather than GI. The types of organ systems affected by immunotherapy AEs can vary based upon which checkpoint inhibitor you use but in some ways, the mechanism by which these occur is very similar, Buchbinder said. Its all an overactivation of the immune system leading to infl ammation in an organ, and there are very few organs that we have not seen toxicity from immunotherapy.

Buchbinder noted that cellular therapies can cause more severe AEs, such as cytokine release syndrome (CRS). Patients can get very sick very quickly, she said, because the therapies given with the cellsincluding the chemotherapy given before and the IL-2 given aftercause most of the AEs. With a lot of the injection therapies, the AEs are related to delivery method, like injection-site issues, but there are also potential systemic AEs like fever, chills, and reactions someone would get to a virus. Its really a huge range in terms of the different [adverse] effects, Buchbinder said.

CRS is the most common AE of CAR T-cell therapy, and it is caused by large numbers of T cells activating, which releases inflammatory cytokines. Although this demonstrates that the therapy is working, it can cause worrisome symptoms. The CRS and the related neurotoxicity can be treated with tocilizumab (Actemra).

One question in the immunotherapy world is whether the development of immune-related AEs predicts a positive or negative response to treatment. With melanoma, we think the data have been very tricky, Buchbinder said. Early trials appeared to show a higher response rate for patients who developed severe symptoms, but as trials developed, that signal was not always there. I think the overall impression is that yes, severe AEs are associated with a better response, she said. A cosmetic AE that clinicians who treat melanoma are excited to see, she said, is vitiligo. It suggests that the immune system is attacking normal melanocytes and that it is attacking cancer cells as well. Those patients generally do far better than patients who dont get vitiligo.

A meta-analysis of 30 studies on the topic, including 4971 individuals, showed that patients who developed immune-related AEs experienced an overall survival benefi t and a progression-free survival benefi t using ICI therapy compared with those who did not. The authors stated that more studies are needed and that the results are controversial.22

Melanoma has been the proving ground for ICIs, Buchbinder said, But now the bar is higher in terms of immunotherapy.

ICIs are now being tested in more immuneresistant tumors. Although there are huge hurdles in terms of some cancers where its going to be hard for immune therapy to do muchlike pancreatic cancer or prostate cancerthere are still diseases where theres opportunity and a possibility that the correct approach or combination might get to some great therapy for those diseases, Buchbinder said

Immunotherapies are being combined with conventional therapies to better integrate treatment. We dont see cancer as a death sentence anymore, Sharma said. We really do see a lot of hope, [and patients with cancer] should be encouraged to discuss immunotherapy with their physician either in a clinical trial or an FDA-approved agent. If you do have a response, its a pretty phenomenal response.

REFERENCES:

1. Lobo N, Brooks NA, Zlotta AR, et al. 100 years of Bacillus Calmette- Gurin immunotherapy: from cattle to COVID-19. Nat Rev Urol. 2021;18(10):611-622. doi:10.1038/s41585-021-00481-1

2. Pierpont TM, Limper CB, Richards KL. Past, present, and future of rituximab-the worlds fi rst oncology monoclonal antibody therapy. Front Oncol. 2018;8:163. doi:10.3389/fonc.2018.00163

3. Kruger S, Ilmer M, Kobold S, et al. Advances in cancer immunotherapy 2019 - latest trends. J Exp Clin Cancer Res. 2019;38(1):268. doi:10.1186/s13046-019-1266-0

4. Lee JB, Kim HR, Ha SJ. Immune checkpoint inhibitors in 10 years: contribution of basic research and clinical application in cancer immunotherapy. Immune Netw. 2022;22(1):e2. doi:10.4110/in.2022.22.e2

5. FDA approves Yervoy (ipilimumab) for the treatment of patients with newly diagnosed or previously-treated unresectable or metastatic melanoma, the deadliest form of skin cancer. News release. Bristol Myers Squibb. March 25, 2011. Accessed May 11, 2022. https://bit.ly/3PFp7q2

6. Rowshanravan B, Halliday N, Sansom DM. CTLA-4: a moving target in immunotherapy. Blood. 2018;131(1):58-67. doi:10.1182/ blood-2017-06-741033

7. Buchbinder EI, Desai A. CTLA-4 and PD-1 pathways: similarities, differences, and implications of their inhibition. Am J Clin Oncol. 2016;39(1):98-106. doi:10.1097/COC.0000000000000239

8. Keown A. Keytruda approvals: a timeline. BioSpace. Aug 13, 2019. Accessed May 11, 2022. https://bit.ly/3yHvfrL

9. Stewart J. Opdivo FDA approval history. Drugs.com. Updated March 15, 2022. Accessed May 20, 2022. https://bit.ly/3lnmtar

10. Markham A, Duggan S. Cemiplimab: fi rst global approval. Drugs. 2018;78(17):1841-1846. doi:10.1007/s40265-018-1012-5

11. FDA grants accelerated approval to dostarlimab-gxly for dMMr endometrial cancer. FDA. Updated April 22, 2021. Accessed May 20, 2022. https://bit.ly/38BSJns

12. Pierpont TM, Limper CB, Richards KL. Past, present, and future of rituximab-the worlds first oncology monoclonal antibody therapy. Front Oncol. 2018;8:163. doi:10.3389/fonc.2018.00163

13. Opdualag becomes fi rst FDA-approved immunotherapy to target LAG-3. National Cancer Institute. April 6, 2022. Accessed May 11, 2022. https://bit.ly/3FZWaAp

14. Acharya N, Sabatos-Peyton C, Anderson AC. TIM-3 finds its place in the cancer immunotherapy landscape. J Immunother Cancer. 2020;8(1):e000911. doi:10.1136/jitc-2020-000911

15. Boldt C. TIL Therapy: 6 things to know. MD Anderson Cancer Center. April 15, 2021. Accessed May 11, 2022. https://bit.ly/3wmguJb

16. FDA approves talimogene laherparepvec to treat metastatic melanoma. National Cancer Institute. November 25, 2015. Accessed May 20, 2022. https://bit.ly/3woTDwA

17. OLeary MC, Lu X, Huang Y, et al. FDA approval summary: tisagenlecleucel for treatment of patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia. Clin Cancer Res. 2019;25(4):1142-1146. doi:10.1158/1078-0432.CCR-18-2035

18. FDA approves CAR-T cell therapy to treat adults with certain types of large B-cell lymphoma. News release. FDA. Oct. 18, 2017. Accessed May 11, 2022. https://bit.ly/3wpECL1

19. FDA approves fi rst CAR T-cell therapy the evolution of CAR T-cell therapy. Cell Culture Dish. October 24, 2017. Accessed May 10, 2022. https:// bit.ly/3LlDD2B

20. Albinger N, Hartmann J, Ullrich E. Current status and perspective of CAR-T and CAR-NK cell therapy trials in Germany. Gene Ther. 2021;28:513-527. doi:10.1038/s41434-021-00246-w

21. Ahmad A, Uddin S, Steinhoff M. CAR-T cell therapies: an overview of clinical studies supporting their approved use against acute lymphoblastic leukemia and large B-cell lymphomas. Int J Mol Sci. 2020;21(11):3906. doi:10.3390/ijms21113906

22. Zhou X, Yao Z, Yang H, Liang N, Zhang X, Zhang F. Are immune-related adverse events associated with the efficacy of immune checkpoint inhibitors in patients with cancer? a systematic review and meta-analysis. BMC Med. 2020;18(1):87. doi:10.1186/s12916-020-01549-2

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10 Years of Immunotherapy: Advances, Innovations, and Better Patient Outcomes - Targeted Oncology

Skin Stem Cells Comments Off on 10 Years of Immunotherapy: Advances, Innovations, and Better Patient Outcomes – Targeted Oncology | June 20th, 2022

An estimated 1,240,000 blood cancer cases emerge annually worldwide, accounting for approximately 6 per cent of all the cancer cases. Meanwhile 720,000 people die of blood cancer every year, accounting for 7 per cent of the cancer deaths.

These statistics were shared by Dr Munira Borhany, haematologist and associate professor at the National Institute of Blood Diseases & Bone Marrow Transplant (NIBD) at a public awareness seminar held recently in collaboration with the Neurospinal & Cancer Care Postgraduate Institute.

The event was titled Rising Burden of Blood Cancers in Pakistan.

"Cancers of blood, bone marrow and lymphatic system are collectively referred to as blood cancers ranging from slow-growing to very aggressive. When the body's red blood cells, white blood cells or platelet production is unusual or abnormal, blood cancer develops. It normally begins in the bone marrow, which is responsible for the production of blood. The normal functioning, growth and development of blood cells that fight infection and make healthy blood cells are disrupted by this type of cancer. There are 137 types of blood cancers and related disorders, she explained, adding that blood cancer was among the most common form of cancers to affect children and adolescents.

The haematologist said that the symptoms of blood cancer could be quite variable depending upon its type. The common symptoms included unexplained fatigue, fever, weakness, and tiredness which could be fast develop in conditions such as acute leukaemia. There may be bleeding manifestations, bone pains, occurrence of swellings in the entire body, loss of appetite, weight loss and abdominal pain, she added.

She said that in general, the symptoms could be quite non-specific such as flu-like symptoms to more dramatic ones such as bleeding manifestations and severe infection. Highlighting the efficacy of bone marrow transplant (BMT) for such patients, she said the BMT was a highly effective therapy and often the only hope for a cure or a longer life for patients with blood cancers.

Dr Munira explained BMT was a procedure to replace disordered bone marrow with healthy bone marrow stem cells. Transplant physicians use this procedure to eliminate cancer or defective stem cells and restore a patient's blood and immune systems.

She added that not all patients with blood cancer required BMT. The need for a bone marrow transplant is evaluated case-wise based on the individual patientss underlying diagnosis, treatment response and disease genetic profile. She informed the event that patients' response to treatment in cases of acute leukemia had improved, due to the cutting-edge genetic profiling technologies combined with innovative medication.

Highlighting the benefits of BMT, Dr Munira said the procedure had two major advantages over other forms of transplants firstly, the donors did not lose any vital part of their body for life and secondly, the recipients had to take the immunosuppressive drug only for nine months.

The bone marrow transplant unit at the NIBD has successfully performed 750 BMT, including 690 allogeneic and 60 autologous blood stem cell transplants with the success rate of more than 80 per cent despite the pressure placed on the healthcare system due to Covid-19 pandemic as well as national economic crisis and escalation of the dollar value against the rupee, she said.

The expert said a bone marrow transplant surgery cost more than Rs4 million in Pakistan but the entire procedure was performed at the NIBD free of charge. She requested the industrial sector, philanthropists and NGOs to support the NIBD for this noble cause.

Blood cancer treatment success rates are improving drastically, and patients are living longer than ever before. There are now various effective and targeted therapeutic agents that have been effective such as chemotherapy, radiotherapy, targeted therapy, bone marrow transplantation and immunotherapy in beating cancer. There is a better chance of a complete cure with early diagnosis, she maintained.

Earlier, NIBD Chief Executive Officer Usama Sultan Shamsi told the seminar that there was a need for increasing awareness among the public as well as medical fraternity to help realise that blood cancer and related disorders were just another forms of disease with a potential for high cure rates provided that they were investigated properly and specific treatment instituted.

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Success rate of blood cancer treatment drastically improving, seminar told - The News International

Bone Marrow Stem Cells Comments Off on Success rate of blood cancer treatment drastically improving, seminar told – The News International | June 20th, 2022

First prospective controlled trial in the world on treating chronic back pain with stem cells shows 70% of participants helped by the treatment

Dr. Sairam Atluri has successfully treated more than 400 patients at his StemCures clinic in Cincinnati over the past five years

Proper use of bone marrow mesenchymal stem cells, or BM-MSCs, for chronic pain treatment follows FDA protocols

CINCINNATI, June 15, 2022 /PRNewswire/ --Local Cincinnati pain physician Dr. Sairam Atluri led the first prospective controlled clinical trial on using bone marrow mesenchymal stem cells (BM-MSCs) for chronic pain. 70% of study participants gained significant pain relief and improved physical and mental function. The first of its kind in the world, the study was completed last month in Ohio.

"Evaluation of the Effectiveness of Autologous Bone Marrow Mesenchymal Stem Cells in the Treatment of Chronic Low Back Pain Due to Severe Lumbar Spinal Degeneration: A 12-Month, Open-Label, Prospective Controlled Trial"was published in the official publication of the American Society of Interventional Pain Physicians, Pain Physician Journal. It is the only scientifically accepted controlled study in the world. 15 other physicians participated in the study.

"When I saw the results my patients were getting at my StemCures health facility, I and my colleagues decided the only way to get physicians to accept this treatment as a mainstream therapeutic for chronic pain was to get a study published in a respected journal," said Dr. Atluri. "About 90 percent of physicians have little to no knowledge of this treatment. They, and their patients, need to know."

40 patients were in the BM-MSC treatment group for the study, and 40 were in a control group, receiving traditional pain modalities such as injections, physical therapy, nerve ablations, and pain medications. The research group then followed their progress for one year:

Almost 70% of those receiving BM-MSCs had significant pain relief and improved physical and mental function. All had cut down or eliminated their pain medication.

Only 8% in the controlled group (not receiving BM-MSCs) had any improved functioning.

Story continues

BM-MSC treatment is a constructive pain therapeutic, as the patient's own cells, that are proven to be safe and effective, are extracted and then injected back into the area that needs repair and healing. The painless procedure takes about 90 minutes and is typically performed just one time for each area affected by chronic pain.

According to Dr. Atluri, it's important the public knows what BM-MSCs are, and what they aren't.

Mesenchymal stem cells are designed to repair and heal. They are present in every tissue, ready to spring into action if you cut your finger, or suffer an acute muscle injury.

BM-MSC treatment, done properly, follows allowed FDA protocols.

BM-MSCs are not embryonic or fetal stem cells. There are almost no cell therapies currently performed with these cell types.

These are not amniotic stem cells. This is important because there are many "pseudo clinics" advertising stem cell procedures that use "off-the-shelf" amniotic stem cells. Most are not performed by qualified physicians, are violating FDA protocols, and are a waste of money. They may also adversely affect your health.

BM-MSCs are not hematopoietic stem cells derived from blood platelets for treatments such as leukemia.

Dr. Atluri, who has successfully treated over 400 patients over the last five years at his clinic, also pointed out that by making the treatment more accessible to chronic pain sufferers, more patients can wean themselves off prescription painkillers. The consequences of that could significantly impact this country's opioid addiction problem.

"These results of this treatment are astonishing and now, irrefutable," said Dr. Atluri. "I travel around the world educating and teaching other physicians about BM-MSCs and now I can do it with scientific proof in hand. Every physician treating chronic pain patients should be identifying their BM-MSC candidates, which are those who suffer from arthritis or joint degeneration for more than six months and don't have contraindications such as cancer.

"It's changing people's lives and in many cases, giving them a future to look forward to for the first time in years."

For more information about the study and bone marrow mesenchymal stem cells treatment, contact Dr. Atluri at hisStemCures clinic at 513-624-7525. The clinic address is 7655 Five Mile Rd., Ste. 117, Cincinnati.

Cision

View original content:https://www.prnewswire.com/news-releases/cincinnati-pain-management-physician-leads-first-successful-stem-cell-controlled-trial-with-70-of-participants-seeing-positive-results-301568002.html

SOURCE Dr. Sairam Atluri

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Cincinnati Pain Management Physician Leads First Successful Stem Cell Controlled Trial with 70% of Participants Seeing Positive Results - Yahoo...

Bone Marrow Stem Cells Comments Off on Cincinnati Pain Management Physician Leads First Successful Stem Cell Controlled Trial with 70% of Participants Seeing Positive Results – Yahoo… | June 20th, 2022

-- First Site Will Enroll First Patient in the Clinical Study--

MELVILLE, NY., June 13, 2022 (GLOBE NEWSWIRE) -- BioRestorative Therapies, Inc. (the Company or BioRestorative) (NASDAQ: BRTX), a clinical stage company focused on stem cell-based therapies, today announced site initiation for its Phase 2 clinical trial targeting chronic lumbar disc disease (cLDD). The Denver Spine and Pain Institute is the first clinical site to be initiated. Additional selected sites are expected to be initiated in 2022.

BioRestoratives Phase 2 trial is a double-blind controlled, randomized study to evaluate the safety and preliminary efficacy of a single dose intradiscal injection of the Companys autologous investigational stem cell-based therapeutic, BRTX-100. A total of up to 99 eligible patients will be randomized at up to 15 centers in the United States to receive either the investigational drug (BRTX-100) or control in a 2:1 fashion.

Currently there are no approved, cell-based therapies for cLDD. While there is encouraging data that suggests that patients with cLDD could benefit from autologous stem cell transplants, the low oxygen micro-environment of the disc makes cell-based therapies challenging. BRTX-100 is manufactured under low oxygen conditions and engineered to survive this environment, said Scott Bainbridge, M.D., Principal Investigator for the BRTX-100 trial at The Denver Spine and Pain Institute. Positive proof-of-concept data in this trial could be disruptive and support the potential applicability of BRTX-100 to other spine and musculoskeletal disorders where low oxygen micro-environments are found.

We are pleased to initiate the first of several sites across the United States that will be enrolling for the trial, said Lance Alstodt, Chief Executive Officer of BioRestorative Therapies. Our sites have been carefully reviewed and selected and have clinical expertise in treating patients who could potentially benefit from BRTX-100. We look forward to working with the principal investigators and their clinical trial teams.

About BioRestorative Therapies, Inc.

BioRestorative Therapies, Inc. (www.biorestorative.com) develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. Our two core programs, as described below, relate to the treatment of disc/spine disease and metabolic disorders:

Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events or results to differ materially from those projected in the forward-looking statements as a result of various factors and other risks, including, without limitation, those set forth in the Company's latest Form 10-K filed with the Securities and Exchange Commission and other public filings. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and the Company undertakes no obligation to update such statements.

CONTACT:Email: ir@biorestorative.com

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BioRestorative Therapies Announces Clinical Site Initiation for the Company's Phase 2 Clinical Trial to Treat Chronic Lumbar Disc Disease (cLDD) -...

Bone Marrow Stem Cells Comments Off on BioRestorative Therapies Announces Clinical Site Initiation for the Company’s Phase 2 Clinical Trial to Treat Chronic Lumbar Disc Disease (cLDD) -… | June 20th, 2022

Symptoms of the disease are usually visible at the age of 5 months and change over time. Some of the common symptoms include pain, anaemia, frequent infections, swelling of hands and feet and vision problem

Sickle-shaped cells and normal blood cells in human blood. Image courtesy: Wikimedia Commons/Dr Graham Beards

World Sickle Cell Day is marked every year on 19 June with an aim to raise awareness about sickle cell disease. Sickle Cell Disease is a group of disorders that impact haemoglobin, the molecule in red blood cells which deliver oxygen to cells throughout the body.

Individuals who live with this disease have haemoglobin S, an atypical haemoglobin molecule which distorts red blood cells into a sickle or a crescent shape. The disease is usually transmitted from parents to children.

What are the symptoms?

Symptoms of the disease are usually visible at the age of 5 months and change over time. Some of the common symptoms include pain, anaemia, frequent infections, swelling of hands and feet and vision problem.

What are the different types of Sickle Cell Disease?

If one of the parents has a problem gene, then the child will not have symptoms but will possess sickle cell trait.

What is the treatment?

The disease can be detected in an infant during the screening process of a newborn. In case, there is a family history of the Sickle Cell disease, it can even be diagnosed at the time of pregnancy.

The only way to cure it is either stem cell or a bone marrow transplant. The symptoms can also be dealt with the use of antibiotics, periodic blood transfusion, pain killers, and vaccinations.

Read all the Latest News, Trending News,Cricket News, Bollywood News,India News and Entertainment News here. Follow us on Facebook, Twitter and Instagram.

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World Sickle Cell Day 2022: Know all about symptoms and treatment of the disease - Firstpost

Bone Marrow Stem Cells Comments Off on World Sickle Cell Day 2022: Know all about symptoms and treatment of the disease – Firstpost | June 20th, 2022

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The report explains the market definition, classifications, applications, engagements, and global Rheumatoid Arthritis Stem Cell Therapy industry trends are.It gives a realistic picture of the current market position incorporating original and predicted market estimates.The report gives a thorough analysis of their product portfolios to investigate the products and applications they focus on while working in the worldwide Rheumatoid Arthritis Stem Cell Therapy market. The report offers valuable suggestions to new just as set up players of the market.

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The product types covered in the report include:

The application types covered in the report include:

The countries covered in the market report are:

The key and emerging market players in the global market include:

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Global Rheumatoid Arthritis Stem Cell Therapy Market 2022 Swot Analysis by Top Key Vendors, Demand And Forecast Research to 2028 Designer Women -...

Bone Marrow Stem Cells Comments Off on Global Rheumatoid Arthritis Stem Cell Therapy Market 2022 Swot Analysis by Top Key Vendors, Demand And Forecast Research to 2028 Designer Women -… | June 20th, 2022

DUBLIN--(BUSINESS WIRE)--Horizon Therapeutics plc (Nasdaq: HZNP) today announced that it has submitted a regulatory filing to the Brazil National Health Surveillance Agency (ANVISA) for UPLIZNA for the treatment of adult patients with anti-aquaporin-4 immunoglobulin G seropositive (AQP4-IgG+) neuromyelitis optica spectrum disorder (NMOSD).

This regulatory submission is an important milestone as we continue to expand our commitment to NMOSD patients around the world, said Vikram Karnani, executive vice president and president, international operations, Horizon. NMOSD is a devastating disease with unpredictable attacks, which can result in potential loss of vision and motor function. We are hopeful that we can bring a potential new treatment option to the estimated ten thousand people living with NMOSD in Brazil.

In the N-MOmentum Phase 3 clinical trial, the largest NMOSD trial to date, UPLIZNA demonstrated a significant reduction in the risk of an NMOSD attack with only two infusions per year, following the initial two loading doses. Additionally, 89% of patients in the AQP4-IgG+ group remained attack-free during the six-month period post-treatment and 83% of patients on treatment remained attack-free for at least four years.1,2

UPLIZNA was approved by the U.S. Food and Drug Administration (FDA) in June 2020, by the Japanese Ministry of Health, Labor and Welfare in March 2021 and by the European Commission (EC) in April 2022. Mitsubishi Tanabe Pharma Corporation has the rights to develop and commercialize UPLIZNA in Japan, Thailand, South Korea, Indonesia, Vietnam, Malaysia, the Philippines, Singapore and Taiwan. Hansoh Pharmaceutical Group Company Limited, another strategic partner to Horizon, has also recently received manufacturing and marketing approval from the National Medical Products Administration of the Peoples Republic of China for UPLIZNA.

About Neuromyelitis Optica Spectrum Disorder (NMOSD)

NMOSD is a unifying term for neuromyelitis optica (NMO) and related syndromes. NMOSD is a rare, severe, relapsing, neuroinflammatory autoimmune disease that attacks the optic nerve, spinal cord, brain and brain stem.3-4 Approximately 80% of all patients with NMOSD test positive for anti-AQP4 antibodies.5 AQP4-IgG binds primarily to astrocytes in the central nervous system and triggers an escalating immune response that results in lesion formation and astrocyte death.6

Anti-AQP4 autoantibodies are produced by plasmablasts and plasma cells. These B-cell populations are central to NMOSD disease pathogenesis, and a large proportion of these cells express CD19.7 Depletion of these CD19 B cells is thought to remove an important contributor to inflammation, lesion formation and astrocyte damage. Clinically, this damage presents as an NMOSD attack, which can involve the optic nerve, spinal cord and brain.6-8 Loss of vision, paralysis, loss of sensation, bladder and bowel dysfunction, nerve pain and respiratory failure can all be manifestations of the disease.9 Each NMOSD attack can lead to further cumulative damage and disability.10,11 NMOSD occurs more commonly in women and may be more common in individuals of African and Asian descent.12,13

About UPLIZNA (inebilizumab-cdon)

INDICATION

UPLIZNA is indicated for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive.

IMPORTANT SAFETY INFORMATION

UPLIZNA is contraindicated in patients with:

WARNINGS AND PRECAUTIONS

Infusion Reactions: UPLIZNA can cause infusion reactions, which can include headache, nausea, somnolence, dyspnea, fever, myalgia, rash or other symptoms. Infusion reactions were most common with the first infusion but were also observed during subsequent infusions. Administer pre-medication with a corticosteroid, an antihistamine and an anti-pyretic.

Infections: The most common infections reported by UPLIZNA-treated patients in the randomized and open-label periods included urinary tract infection (20%), nasopharyngitis (13%), upper respiratory tract infection (8%) and influenza (7%). Delay UPLIZNA administration in patients with an active infection until the infection is resolved.

Increased immunosuppressive effects are possible if combining UPLIZNA with another immunosuppressive therapy.

The risk of Hepatitis B Virus (HBV) reactivation has been observed with other B-cell-depleting antibodies. Perform HBV screening in all patients before initiation of treatment with UPLIZNA. Do not administer to patients with active hepatitis.

Although no confirmed cases of Progressive Multifocal Leukoencephalopathy (PML) were identified in UPLIZNA clinical trials, JC virus infection resulting in PML has been observed in patients treated with other B-cell-depleting antibodies and other therapies that affect immune competence. At the first sign or symptom suggestive of PML, withhold UPLIZNA and perform an appropriate diagnostic evaluation.

Patients should be evaluated for tuberculosis risk factors and tested for latent infection prior to initiating UPLIZNA.

Vaccination with live-attenuated or live vaccines is not recommended during treatment and after discontinuation, until B-cell repletion.

Reduction in Immunoglobulins: There may be a progressive and prolonged hypogammaglobulinemia or decline in the levels of total and individual immunoglobulins such as immunoglobulins G and M (IgG and IgM) with continued UPLIZNA treatment. Monitor the level of immunoglobulins at the beginning, during, and after discontinuation of treatment with UPLIZNA until B-cell repletion especially in patients with opportunistic or recurrent infections.

Fetal Risk: May cause fetal harm based on animal data. Advise females of reproductive potential of the potential risk to a fetus and to use an effective method of contraception during treatment and for 6 months after stopping UPLIZNA.

Adverse Reactions: The most common adverse reactions (at least 10% of patients treated with UPLIZNA and greater than placebo) were urinary tract infection and arthralgia.

For additional information on UPLIZNA, please see the Full Prescribing Information at http://www.UPLIZNA.com.

About Horizon

Horizon is a global biotechnology company focused on the discovery, development and commercialization of medicines that address critical needs for people impacted by rare, autoimmune and severe inflammatory diseases. Our pipeline is purposeful: We apply scientific expertise and courage to bring clinically meaningful therapies to patients. We believe science and compassion must work together to transform lives. For more information on how we go to incredible lengths to impact lives, visit http://www.horizontherapeutics.com and follow us on Twitter, LinkedIn, Instagram and Facebook.

Forward-Looking Statements

This press release contains forward-looking statements, including, but not limited to, statements related to potential regulatory approval of UPLIZNA in Brazil and the potential benefits of UPLIZNA to patients in Brazil. These forward-looking statements are based on management expectations and assumptions as of the date of this press release, and actual results may differ materially from those in these forward-looking statements as a result of various factors. These factors include the risk that UPLIZNA does not receive regulatory approval in Brazil, whether, if regulatory approval is received, UPLIZNA will be successfully commercialized in Brazil, and those risks detailed from time-to-time under the caption "Risk Factors" and elsewhere in Horizons filings and reports with the SEC. Horizon undertakes no duty or obligation to update any forward-looking statements contained in this press release as a result of new information.

References

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Horizon Therapeutics plc Submits Regulatory Filing for UPLIZNA (inebilizumab) in Brazil - Business Wire

Spinal Cord Stem Cells Comments Off on Horizon Therapeutics plc Submits Regulatory Filing for UPLIZNA (inebilizumab) in Brazil – Business Wire | June 20th, 2022

Abstract: Background Avascular necrosis of the femoral head (ANFH) is a common orthopaedic disease due to bone defects. However, few clear methods to treat ANFH in clinical practice are known. Many findings suggest that promoting the osteogenic differentiation and directional migration of stem cells may be a key method for bone regeneration. Over time, an increasing number of researchers have begun to focus on Chinese medicine and Chinese medicinal extracts; Epimedium is the Chinese herbal medicine studied in the most detail in the field of bone regeneration, and Icariin (ICA) is one of the main active ingredients in Epimedium. Objective This study aimed to explore the effects of ICA on the osteogenic differentiation and migration of bone marrow mesenchymal stem cells (BMSCs) and reveal its mechanism to provide a theoretical basis for the treatment of ANFH. Methods After primary BMSCs were freshly isolated from a normal rabbit and treated with various concentrations of ICA, the activity and proliferation of BMSCs were detected by CCK-8 assay, and the mineralized nodules was detected by Alizarin Red staining, to determine the optimal concentration of ICA. qPCR and western blot were used to demostrate the effects of ICA on the osteogenic differentiation and migration of BMSCs. Osteoblasts-derived exosomes (OB-exos) were extracted and analysed by high-throughput sequencing. Effect of ICA combined with OB-exos were analysed. And miRNA mimic and an miRNA inhibitor were synthetised to verify the osteogenic differentiation and migration of BMSCs alone or co-cultured with ICA. Results The CCK-8 assay and Alizarin Red staining showed that the optimal concentration of ICA is 1107 M. ICA could effectively promote the osteogenic differentiation and migration of BMSCs, and this could be enhanced after co-culturing with OB-exos. The four miRNAs with the greatest concentrations in the OB-exos were let-7a-5p, miR-100-5p, miR-21-5p and miR-122-5p. qPCR and western blot showed that miR-122-5p mimic has positive effects on the osteogensis and migration, and its inhitiotr has negative effects. Simliarly, they could enhance or inhitor the effects of ICA, which means miR-122-5p may be the target of ICA. Conclusion Like the OB-exos, ICA could obviously promote the osteogenic differentiation and migration of BMSCs. The combination of ICA and OB-exos enhanced these effects, in which miR-122-5p plays a pivotal role.

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Mechanism of Icariin regulation of the effect of miR-122-5p in osteoblast-derived exosomes on osteogenesis and migration of bone marrow mesenchymal...

Bone Marrow Stem Cells Comments Off on Mechanism of Icariin regulation of the effect of miR-122-5p in osteoblast-derived exosomes on osteogenesis and migration of bone marrow mesenchymal… | June 11th, 2022

A new study has identified a possible target for treating juvenile myelomonocytic leukemia, a highly aggressive blood cancer.

Research into a type of blood cancer called juvenile myelomonocytic leukemia suggests anti-inflammatory treatment as a possible new therapy for the disease

Juvenile myelomonocytic leukemia (JMML) is most common in children under the age of four. 10% of all cases occur in newborns under the age of three months. Every year, one to two children out of a million are diagnosed with JMML. The disease accounts for approximately 1.6 percent of all blood-related cancers. It is more common in males, with boys being nearly twice as likely as girls to develop JMML.

The exact underlying causes of JMML are unclear, however, virtually all patients will have a mutation in a RAS gene or a gene that affects the activation levels of Ras proteins that modifies the DNA within their blood cells. Currently, allogeneic hematopoietic stem cell transplantation is the only successful treatment for the majority of patients, although recent advancements show promise.

According to a report published on May 10th, 2022, in the journal eLife, scientists have identified a potential new target for treating patients with the blood cancer juvenile myelomonocytic leukemia (JMML).

Their findings in zebrafish and JMML patients suggest that anti-inflammatories may be a new approach to fighting the disease.

JMML is a highly aggressive blood cancer with poor patient outcomes. Children with Noonan Disease (NS), a relatively common developmental syndrome, are at a high risk of having a disorder similar to JMML called myeloproliferative neoplasm, which may later progress to JMML. A mutation in the PTPN11 gene, which encodes the protein-tyrosine phosphatase SHP2, is the most common genetic cause of JMML and NS.

Hematopoietic stem and progenitor cells are considered to be the cells of origin for JMML, says first author Maja Solman, Postdoctoral Fellow at the Hubrecht Institute, Utrecht, Netherlands. Currently, hematopoietic stem cell transplantation is the only treatment for the disease, but it has a relapse rate of 50%. With such limited treatment options for JMML, we wanted to gain a better understanding of how the disease develops to identify other possible ways of targeting it.

This image shows the macrophages (red) and neutrophils (green) in a zebrafish embryo with a mutation in SHP2. The head of the embryo is on the left, the tail on the right. Similar to the situation in JMML patients, this fish has more macrophages and neutrophils compared to fish without a mutation in SHP2. Credit: Maja Solman

To do this, Solman and the team used a novel zebrafish model with a mutation in SHP2 equivalent to the most common mutation in NS patients which can cause JMML. They used a technique called single-cell transcriptomics to examine the level of gene expression in the animals hematopoietic stem and progenitor cells. The analysis showed an increase in the number of monocyte and macrophage progenitor cells in the fish embryos, and that these cells expressed genes associated with the immune response.

The team next compared these results with their analysis of hematopoietic stem and progenitor cells, which contained SHP2 mutations, from the bone marrow of JMML patients. They found a similar pattern of proinflammatory gene expression in these cells to the one they identified in the zebrafish.

Finally, they treated the zebrafish embryos with an anti-inflammatory drug called dexamethasone. They found that the drug helped rescue JMML-like blood defects in the fish, suggesting that anti-inflammatories could one day be an important treatment strategy for JMML.

Our work reveals striking similarities in the proinflammatory response of human and zebrafish cells containing SHP2 mutations, and shows that inhibiting this response can improve JMML-like symptoms in a zebrafish model, concludes senior author Jeroen den Hertog, Group Leader and Managing Director at the Hubrecht Institute, and Professor of Molecular Developmental Zoology at Leiden University, Netherlands. Together, these findings lay the groundwork for future studies to verify the effectiveness of anti-inflammatories as a potential new treatment approach for JMML patients.

Reference: Inflammatory response in hematopoietic stem and progenitor cells triggered by activating SHP2 mutations evokes blood defects by Maja Solman, Sasja Blokzijl-Franke, Florian Piques, Chuan Yan, Qiqi Yang, Marion Strullu, Sarah M Kamel, Pakize Ak, Jeroen Bakkers, David M Langenau, Hlne Cav and Jeroen den Hertog, 10 May 2022, eLife.DOI: 10.7554/eLife.73040

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New Treatment for Highly Aggressive Blood Cancer - SciTechDaily

Bone Marrow Stem Cells Comments Off on New Treatment for Highly Aggressive Blood Cancer – SciTechDaily | June 11th, 2022

Myeloproliferative neoplasms (MPNs) are a diverse group of chronic hematological diseases caused by the clonal expansion of abnormal hematopoietic stem cells, of which polycythemia vera (PV), essential thrombocythemia (ET), and primary myelofibrosis (PMF) have been extensively studied in terms of clonal expansion, fibrosis, and other phenotypes. For a study, researchers sought to evaluate current research on the impact of different types of MPN on bone health.

Human data were used in research to show that different types of MPN influence bone density, osteoblast proliferation, and differentiation. The majority of data revealed that bone volume is frequently raised in patients with PMF. In contrast, it was slightly decreased or not affected in patients with ET or PV; however, probable distinctions between male and female phenotypes in most MPN subtypes have not been thoroughly examined.

Osteosclerosis in PMF patients was a significant consequence that could result in bone marrow failure, and bone loss seen in some ET or PV patients can result in osteoporotic fractures. Some MPN types were associated with an increase in the number of megakaryocytes (MKs), and various MK-related MPN variables are known to impact bone formation.Investigators discussed known mechanisms involved in the processes, emphasizing the function of MKs and secreted factors. Understanding MPN-related alterations in bone health should lead to better early intervention and treatment of this pathologys adverse effects.

Reference:ashpublications.org/blood/article-abstract/139/21/3127/476662/Myeloproliferative-disorders-and-their-effects-on?redirectedFrom=fulltext

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Myeloproliferative Disorders & Bone Homeostasis: The Role of Megakaryocytes - Physician's Weekly

Bone Marrow Stem Cells Comments Off on Myeloproliferative Disorders & Bone Homeostasis: The Role of Megakaryocytes – Physician’s Weekly | June 11th, 2022

Pervez Musharraf served as Pakistan's president from 2001 to 2008.

The family of former Pakistan President Pervez Musharraf (retired) has said that he has been hospitalised for the last three weeks after his condition worsened. In a statement posted on Twitter on Mr Musharraf's official Twitter account, his family said that he is dealing with Amyloidosis and prayed for ease in his daily living.

"He is not on the ventilator. Has been hospitalized for the last 3 weeks due to a complication of his ailment (Amyloidosis). Going through a difficult stage where recovery is not possible and organs are malfunctioning. Pray for ease in his daily living," the tweet said.

According to National Health Service (NHS) of the UK, Amyloidosis is the name for a group of rare, serious conditions caused by a build-up of an abnormal protein called amyloid in organs and tissues throughout the body.

Amyloid deposits can build up in the heart, brain, kidneys, spleen and other parts of the body.

The build-up of these protein deposits can make it difficult for the organs and tissues to work properly. Without treatment, this can lead to organ failure, the NHS further said.

Mr Musharraf was diagnosed with the life-threatening health condition in 2018 in the UAE.

Amyloidosis may be secondary to a different health condition or can develop as a primary condition, according to Johns Hopkins Medicine. Sometimes it is due to a mutation in a gene, but other times the cause of amyloidosis remains unknown, it added.

There are different types of the Amyloidosis, which are prevalent:

Light-chain (AL) Amyloidosis: It can affect the kidneys, spleen, heart, and other organs. People with conditions such as multiple myeloma or a bone marrow illness called Wadenstrom's macroglobulinemia are more likely to have AL amyloidosis.

AA Amyloidosis: It is caused by fragments of amyloid A protein, and affects the kidneys in about 80 per cent of cases. It can complicate chronic diseases characterized by inflammation, such as rheumatoid arthritis (RA) or inflammatory bowel disease (IBS).

Transthyretin Amyloidosis (ATTR): It can be inherited from a family member. Transthyretin is a protein that is also known as prealbumin. It is made in the liver. Excessive normal (wild-type ATTR) or mutant transthyretin can cause amyloid deposits.

Though there are some common symptoms of the disease, they vary greatly depending on where the amyloid protein is collecting in the body.

The general symptoms include (listed by Johns Hopkins on its website):

As Amyloidosis progresses, the deposits of amyloid can harm the heart, liver, spleen, kidneys, digestive tract, brain or nerves.

The diagnosis of Amyloidosis is hard, as the symptoms are generic. The doctors take a small sample of the tissue (biopsy) from the affected part to understand what kind of Amyloidosis is present. Depending on that, the treatment is done.

There is not currently a cure for amyloidosis. The amyloid deposits cannot be directly removed. So the doctors may discuss:

Chemotherapy, which is used to kill cancer cells or stop them from growing and similar method can be adopted to stop the growth of cells that are making abnormal protein.

Bone marrow transplant: In this procedure, healthy stem cells are taken from a patient's body. They are then infused back into their body to replace the unhealthy ones destroyed in chemotherapy.

There are some medicines too, which are used to treat Amyloidosis. These medicines are approved by the Food and Drug Administration (FDA) in the United States.

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What Is Amyloidosis, The Health Condition Pervez Musharraf Is Suffering From - NDTV

Bone Marrow Stem Cells Comments Off on What Is Amyloidosis, The Health Condition Pervez Musharraf Is Suffering From – NDTV | June 11th, 2022

The new report by Expert Market Research titled, Global Live Cell Imaging Market Report and Forecast 2022-2027, gives an in-depth analysis of the global live cell imaging market, assessing the market based on its segments like product type, application, technology, and major regions. The report tracks the latest trends in the industry and studies their impact on the overall market. It also assesses the market dynamics, covering the key demand and price indicators, along with analysing the market based on the SWOT and Porters Five Forces models.

Request a free sample copy in PDF or view the report [emailprotected] https://bit.ly/3mtMGEU

The key highlights of the report include:

Market Overview (2017-2027)

As the number of stem cell research projects grows, so does the use of live cell imaging tools to analyse the location, purity, and amount of cells and their components, boosting market growth. The use of live cell imaging tools to precisely detect protein levels for optimal medication therapy is rising, as it is critical to determine the interaction between stem cells and tissues during stem cell research. The introduction of numerous government initiatives to support research and development (R&D) activities is fueling the live cell imaging industrys expansion. For example, in March 2020, the Canadian government announced a $6.9 million investment to promote stem cell research efforts in the country through the Stem Cell Networks research financing programme.

Furthermore, the increasing use of live cell imaging in the discovery of new medications is propelling the market forward. The development of new technologies that allow for the precise analysis of RNA, nucleic acid, proteins, and DNA, among other things, is driving demand for many diagnostic methods, moving the market forward. Furthermore, the rise in the prevalence of chronic diseases like cancer is driving up demand for live cell imaging in both diagnosis and treatment. The expanding research and development (R&D) activities to detect cancer cells in bone marrow while also allowing for the identification of specific cancer cells are likely to boost market growth.

Industry Definition and Major Segments

The study of living cells using microscope technology to obtain images of live cells and tissues is known as live cell imaging. It is essential in a variety of laboratory operations in biological and biomedical research because it gives real-time and reliable information on cells and tissues, making it suitable for stem cell research and regenerative medicine development.

Explore the full report with the table of [emailprotected] https://bit.ly/3tpEoSd

By technology, the market can be divided into:

The market can be categorised based on its applications into:

The major product types of live cell imaging are:

The regional markets include:

Market Trends

Artificial intelligence (AI), deep learning, and 3D printing are progressively being integrated into live cell imaging techniques, as technology improvements are a key antecedent of scientific research and development efforts. The expanding use of artificial intelligence (AI) allows for more precise, simpler, and time-efficient cell imaging. Furthermore, AI-based microscopy can recognise and analyse minor cell components like nuclei, allowing researchers to analyse data more quickly and effectively. AI-based microscopes also automate and optimise many functions for quantifying live cells, resulting in increased cell viability and faster image capture. This is fueling the expansion of the live cell imaging sector by increasing demand for such microscopes in research centres.

Furthermore, the increasing use of 3D printing in a variety of medical and biological applications is fueling market expansion. Because air bubbles are a common problem in perfusion chambers used in live cell imaging, the demand for fluidic devices made with 3D printing technology is increasing dramatically. Furthermore, the cost-effectiveness of 3D printing is increasing the affordability of live cell imaging research operations, which is propelling the market forward. In the forecast future, the development of portable and low-profile devices that can be directly installed on optical microscopes to improve cell imaging precision is expected to drive market expansion for live cell imaging.

Latest News on Global Live Cell Imaging [emailprotected] https://bit.ly/3HaaQ0z

Key Market Players

The major players in the market are Carl Zeiss AG, Leica Microsystems GmbH, Nikon Instruments Inc., Becton, Dickinson and Company, GE Healthcare and Others.

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Expert Market Research is a leading business intelligence firm, providing custom and syndicated market reports along with consultancy services for our clients. We serve a wide client base ranging from Fortune 1000 companies to small and medium enterprises. Our reports cover over 100 industries across established and emerging markets researched by our skilled analysts who track the latest economic, demographic, trade and market data globally.

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Global Live Cell Imaging Market to be Driven by Growing Stem Cell Research Market in the Forecast Period of 2022-2027 mbu timeline - mbu timeline

Bone Marrow Stem Cells Comments Off on Global Live Cell Imaging Market to be Driven by Growing Stem Cell Research Market in the Forecast Period of 2022-2027 mbu timeline – mbu timeline | June 11th, 2022

The BioIntelliSense BioSticker. Photo courtesy of BioIntelliSense.

James Byrne hadnt been out of the hospital for long when a call came in after midnight. He answered to the voice of a triage nurse asking him how he was feeling. He was pretty tired, actually, having been through radiation treatment and chemotherapy designed to eradicate his acute myeloid leukemia and then an allogeneic bone marrow transplant (his son Logan had been the key donor; his cells were augmented with donated umbilical-cord stem cells). That and having been asleep until his cell phone rang. But otherwise, just fine, he said.

Good to hear, the nurse said because Byrnes body temperature had just jumped to 98.9 degrees. While thats just a shade higher than the standard 98.6 degrees, his temperature had been hanging around 97 degrees, so what would otherwise seem normal might instead have been an indication of a brewing infection. Given that his immune system had been wiped out and was just starting to rebuild itself, an infection would pose a serious risk to the 45-year-old resident of Albuquerque, N.M. This was, fortunately, a red flag that could be safely lowered again.

Byrne would have had a hard time taking his own temperature while sound asleep. A three-inch by one-inch, U.S. Food and Drug Administration-approved medical device called a BioSticker had done that and much more.

Before Byrne left UCHealth University of Colorado Hospital on the Anschutz Medical Campus, the UCHealth and University of Colorado School of Medicine advanced practice practitioner leading the study, Glen Peterson, had asked him if he might be interested in joining one of two cancer-related clinical trials using the remote-monitoring gadget developed by Golden-based BioIntelliSense. This one would involve sticking the BioSticker to his upper chest and largely forgetting about it for 30 days. The BioSticker would collect a steady stream of his vital signs body temperature, respiratory rate, resting heart rate, skin temperature, gait, body position and more and transmit it to UCHealth providers via a wireless hub and Reimagine Care, a startup focused on home cancer care. The goal would be to spot emerging health problems before Byrne himself did.

Its not the first work UCHealth has done with BioIntelliSense. Their collaborations through the UCHealth CARE Innovation Center have most notably involved remote monitoring of high-risk COVID-19 patients. But Dr. Richard Zane, UCHealths chief innovation officer and chair of the CU School of Medicines Department of Emergency Medicine, says the technology could apply to a wide range of health conditions.

Its about bringing remote patient monitoring to wherever the patient is, understanding how to ingest key vital signs, and building predictive and prescriptive algorithms on top of those data so that we can intervene before we otherwise would have, Zane said.

Technologies such as BioIntelliSenses BioSticker and the recently announced BioButton an even-smaller stick-on medical device that captures much of the same data as its predecessor have the potential to supplant an outdated system of discharged patients self-reporting their own health status, as Dr. Clay Smith, director of the UCHealth Blood Disorders and Cell Therapies Center and associate chief of the CU School of medicines Division of Hematology, put it.

Byrnes example of having a lower-than-usual body temperature is a case in point. The typical approach of having people take their temperature a couple of times a day and report fevers to caregivers assumes 98.6 as the normal temperature. But it can vary from person to person, as it did with Byrne; it can be lowered by medications; it can go up on a hot day or even when wearing a hat. A long-term monitoring device such as the BioSticker, which captures and transmits 1,440 measurements every 24 hours, day and night, can establish baselines from which to judge abnormalities.

We see people who are quite ill who have a normal temperature and those with a higher temperature who are fine, Smith said.

The second UCHealth-BioIntelliSense study also relates to body temperature. That study, which Peterson also leads, involves cancer patients whose chemotherapy has left them with low numbers of white blood cells called neutrophils that help fight infections. These patients are susceptible to febrile neutropenia a fever caused by an infection that exploits the weakened immune system. Febrile neutropenia cases account for about 5% of all adult cancer-related hospitalizations in the United States, with an average stay of more than eight days.

The sooner one can spot a fever or other signs of nascent illness, the faster the patient can get treatment.

We know that, the longer you wait from the time infection starts, the worse the outcomes, Smith said. Its important to detect it as soon as possible, figure out what type of infection it is, and start antibiotics.

Fevers are only the beginning, Smith says. He sees technologies such as the BioSticker feeding diverse readings into artificial-intelligence-powered systems capable of synthesizing body temperature, heart rate, respiratory rate, movement characteristics, and other factors to detect a whole range of problems and alert health care providers to nip health problems in the bud.

Zane says BioIntelliSenses work with UCHealth is already moving in that direction. Theyve identified patterns in the data that seem to predict fevers while a patients temperature is still normal. Such insight could one day let providers prescribe antibiotic pills for patients at home to beat back infections before theyre serious, thereby avoiding hospitalization for intravenous antibiotics.

James Byrnes BioSticker has triggered a few calls over the month he has worn it, he says. One involved a 3:30 a.m. call from a nurse practitioner triggered by a change in his breathing patterns and indeed, he had been coughing for much of the night. The nurse had him check his pulse oxygen level; it was normal.

Another had to do with a leap in his heart rate. He happened to have been walking around quite a bit at the time, he says, and he isnt in the best shape of his life for obvious reasons. The triage nurse explained why she was calling and asked, You doing alright?

Im doing just fine, he answered.

But it was nice to get that call.

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BioSticker detects more than just fever in cancer patients - UCHealth Today

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InThe Moment, a new podcast series from The Athletic,Kelly Cates and Geoff Thomas speak to sportspeople about experiencing and overcoming times of adversity that changed their lives forever.

In this weeks episode, former England footballer Thomas discusses his own recovery from leukaemia and his determination to raise millions of pounds for blood cancer research by completing the route of cyclings arduous Tour de France, just 18 months after undergoing a stem cell transplant.

When Geoff Thomas was told he had three years to live, it seemed like a lifetime.

Just 24 hours earlier, hed believed it was no more than three months.

It was July 2003 and the former Crystal Palace, Wolves and England footballer had been diagnosed with chronic myeloid leukaemia. Id only been retired from football about six to eight months, he says. Id started suffering from fatigue and I was having night sweats and various things but, a typical man, was putting off going to see a doctor.

On the morning of July 4, Thomas finally went to see his doctor. Blood was taken for testing and he was sent home with the message that the results would come back in three weeks time.

About three or four hours later, I got the phone call that changed my life. It was a call that told me I had a form of leukaemia. I was told I could be in the blast phase which, when I stupidly went on the internet, meant it looked like I could only have three months to live.

The following day, Thomas was introduced to bone-marrow specialist Professor Charlie Craddock, who attached him to a machine which helped confirm that, in fact, he had three years to live. The only slim chance of him surviving his leukaemia was to find a stem cell match for a transplant.

Back in 2003, chronic myeloid leukaemia was more or less a death sentence if you couldnt find a match to have a stem cell transplant. And thats only 20 per cent of the population that can do that.

When youre told the brutal honesty about whats ahead of you, you know, the chances of you not surviving is really pretty high. So youre looking for any positives to put you on that positive track.

I read a book about Lance Armstrongs journey the second day I was diagnosed, and I know his name has been tarnished since (Armstrong was stripped of his seven Tour de France and banned from pro cycling for life for doping offences), but his fightback and his desperation to find the best way of surviving his cancer put me on that track. I wanted to turn every stone to give myself the opportunity of surviving this.

Its almost 19 years now since that diagnosis but, mentally and emotionally, Thomas can put himself back on that ward in an instant. I can know exactly what the conversations were and exactly how myself and my wife were handling it: not very good for the first three days, but we had to protect our 10- and seven-year-old little girls, and make sure they were OK.

For the next couple of weeks from being diagnosed, I was looking at what their future looked like without me, to be honest. I was looking to see if they were insured properly, if the house was safe if I disappeared. That was extra comfort, but Ive realised that so many other people dont have that opportunity.

Four days after he was diagnosed, Thomas sister Kay went for blood tests to see if she was a stem cell match. It would take over a month for the results to come back and they were told there was just a one-in-five chance she would be suitable. In the meantime, Thomas and wife Julie made a list of trips they wanted to go on with their two girls. We went off to places like Disneyland Paris and just tried to shield them as much as we could by enjoying ourselves.

Bizarrely, I was in a really good space in my mind because for the first time I realised what was really important in life. And it wasnt the fast cars, it wasnt the bigger house, it wasnt getting a bigger garden. It was just purely your health and your family and being able to be around them.

Thats something I try to keep. When Im starting to have little down days, I try and keep that message within that things could be a hell of a lot worse.

Thomas was fortunate that Kay was a match. Not a very good match, he qualifies. So the doctors had to do some tweaking. But it was good enough for me to go into isolation for five to six weeks and really be beaten up with chemotherapy and radiotherapy. They more or less take everything down to ground level and build you back up again. You purely put your life in the hands of the great guys and nurses at the hospital.

He was put on an intensive course of chemotherapy and radiotherapy to prepare his body for the transplant. At that point, he says your immune system is gone. A simple cold could kill you. Then, all of a sudden, they bring this bag in. Its only about eight inches long, and its just full of a browny, horrible colour.

They hang it up on the little pole and you see it going down the tube and going through your body that was my sisters stem cells. It took a good eight hours to be put in.

The next day, I was really, really poorly. The nurses warned Julie, It doesnt look good. Hes been struggling. And it was probably the lowest point of everything I went through. I didnt think I was going to make it. But Julie just put her arm around me and gave me a hug for about a couple of hours.

After a few days, there were indications that his immune system was starting to work. It was a positive sign. But it would take around six months for Thomas to feel normal again.

It was January 2004 when Thomas had the transplant, and a year later when he received the momentous news that he was in remission, albeit with the knowledge that the treatment hed received could put him at risk of developing different cancers later in life.

The attitude and mentality he developed during a 20-year long football career has played a key role in helping Thomas through the difficult times he suffered. In football, youve got to have good coaches. Youve got to have good trainers to make you the sportsperson you become, he says. But most importantly, you need to have your own determination to make that happen and to be willing to keep learning and keep improving on everything.

Theres so many parallels; the manager turned into the professor. Your team-mates turned into fellow patients. And the nurses were coaches.

And so my mindset was not to try and say, Im going to beat this. I was being honest with everybody. I said, I dont know if Im going to. Like in football, I dont know if were going to win on Saturday, but well try our damnedest to make sure that we do everything to get a good result.

In sport, you find that any negative thought is detrimental to your performance on the Saturday. And I think any negative thought when youre battling an illness is detrimental to your battle as well.

Ive even seen people who have had a positive outcome not able to move away from the illness itself, so they live with that journey theyve been on. And rather than taking it into a positive, theyve kept it like a shroud of darkness around them. Its tough watching these sorts of people.

Thomas still has days when the enormity of what he faced gives him the shivers. It hits me when I see other people going through it, he says. Selfishly, I dont sometimes respond to their story its like it brings my story back. There probably were times where I should have been a lot more open about my feelings but I wasnt. I just buried it.

Ive met a number of footballers who have been touched with blood cancer since. Stiliyan Petrov I met when he was just diagnosed and I just said, Listen, open yourself up to everybody and let your emotions out, because I didnt cry for a while and I felt so much better when I did.

From almost the minute he was diagnosed, Thomas was looking for ways to utilise the profile he had from football to help those who were helping him. On the second day after he met Professor Craddock, he asked him what he could do. I always remember his answer, Thomas says. Let me get you better first, then Ill come back to you. And thats what hes been doing ever since.

A sponsored run or walk is the first port of call for most people who want to find a way to say thank you to the doctors and nurses who have helped them. But as a former professional footballer, Thomas felt he needed to do something more.

Hed already seen former England cricketer Sir Ian Botham walk hundreds of miles to raise funds for leukaemia research and the competitive streak in him told him he needed to surpass that.

A good friend of mine now, Neil Ashton, was following my story he was a reporter back then. He said, Why dont you do the Tour (de France)?. I didnt know what the Tour was, really. I watched it now and then without knowing what was really going on.

Thomas said hed think about it and get back to him. That was February 2005. Id gone into remission in January 2005. And by July, I was doing exactly the same mileage as a professional cyclist just a hell of a lot slower. I found myself doing nearly two and a half thousand miles on a bike and riding some horrendous climbs in the Alps and the Pyrenees.

That was the moment I got my life back. There were stages in there that were so tough that it definitely brought tears to my eyes. But the strength I took on was of the people Id met over the previous two years who hadnt made it; all these stories Ive got deep in the back of my mind.

They always come to the fore when Im going through a little struggle myself, because these people would love to be in my situation when I was going up these climbs. Theyre the people I still call on now and then to keep me going.

Was it medically advisable to put his body through such an intense experience: 21 stages and 2,233 miles? Thomas smiles. Charlie said that in a similar sort of timeline, people normally come and ask him, Is it OK to start swimming?.

He raised over 150,000 that year from cycling the Tour route, and two years later set up the Geoff Thomas Foundation to fund clinical research into leukaemia. In the years since, he has raised hundreds of millions of pounds, helping to fund clinical trial networks, get more drugs into the treatment system and build an infrastructure that allows work to flow and benefits patients.

We need an awful lot more to enable us to fulfill the vision of the blood cancer professors up and down this country because they believe we can beat this in 10 to 15 years eradicate blood cancer. Its such a strong message to get out there and people can see the improvement over the last 15 years that we are getting there.

Collectively, we can beat this.

Listen and subscribe to The Athletics The Moment podcast, including the first episode with Gary Lineker.

(Lead graphic: Sam Richardson)

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The Moment: Geoff Thomas After being diagnosed with leukaemia, I was looking at what my girls future looked like without me - The Athletic

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TAIPEI--(BUSINESS WIRE)--PharmaEssentia Corporation (TPEx:6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, today announced a series of data presentations will illustrate outcomes with ropeginterferon alfa-2b (marketed as BESREMi) among adults with polycythemia vera (PV) during the European Hematology Associations Hybrid Congress (EHA2022), June 9-17 in Vienna, Austria.

Ongoing evaluations of ropeginterferon alfa-2b expand the depth and duration of data on this innovative therapeutic supporting its ability to control the effects of polycythemia vera (PV), said Albert Qin, MD, PhD, Chief Medical Officer, PharmaEssentia. We believe these important new data offer greater clarity and confidence to physicians that this therapeutic tool represents an approach to effectively and durably treat PV.

Ropeginterferon alfa-2b presentations during EHA2022 will include:

The data presentation regarding the final results of studies leading to marketing authorization of BESREMi in Europe are a result of clinical development work of AOP Health, Vienna. PharmaEssentia has licensed ropeginterferon alfa-2b in Europe to AOP.

About Polycythemia Vera

Polycythemia Vera (PV) is a cancer originating from a disease-initiating stem cell in the bone marrow resulting in a chronic increase of red blood cells, white blood cells, and platelets. PV may result in cardiovascular complications such as thrombosis and embolism, and often transforms to secondary myelofibrosis or leukemia. While the molecular mechanism underlying PV is still subject of intense research, current results point to a set of acquired mutations, the most important being a mutant form of JAK2.1

About BESREMi (ropeginterferon alfa-2b)

BESREMi is an innovative monopegylated, long-acting interferon. With its unique pegylation technology, BESREMi has a long duration of activity in the body and is aimed to be administered once every two weeks (or every four weeks with hematological stability for at least one year), allowing flexible dosing that helps meet the individual needs of patients.

BESREMi has orphan drug designation for treatment of polycythemia vera (PV) in adults in the United States. The product was approved by the European Medicines Agency (EMA) in 2019, in the United States in 2021, and has recently received approval in Taiwan and South Korea. The drug candidate was invented by PharmaEssentia and is manufactured in the companys Taichung plant, which was cGMP certified by TFDA in 2017 and by EMA in January 2018. PharmaEssentia retains full global intellectual property rights for the product in all indications.

BESREMi was approved with a boxed warning for risk of serious disorders including aggravation of neuropsychiatric, autoimmune, ischemic and infectious disorders.

About PharmaEssentia

PharmaEssentia Corporation (TPEx: 6446), based in Taipei, Taiwan, is a rapidly growing biopharmaceutical innovator. Leveraging deep expertise and proven scientific principles, the company aims to deliver effective new biologics for challenging diseases in the areas of hematology and oncology, with one approved product and a diversifying pipeline. Founded in 2003 by a team of Taiwanese-American executives and renowned scientists from U.S. biotechnology and pharmaceutical companies, today the company is expanding its global presence with operations in the U.S., Japan, China, and Korea, along with a world-class biologics production facility in Taichung. For more information, visit our website.

1 Cerquozzi S, Tefferi A. Blast Transformation and Fibrotic Progression in Polycythemia Vera and Essential Thrombocythemia: A Literature Review of Incidence and Risk Factors. Blood Cancer Journal (2015) 5, e366; doi:10.1038/bcj.2015.95.

2022 PharmaEssentia Corporation. All rights reserved.

BESREMi and PharmaEssentia are registered trademarks of PharmaEssentia Corporation, and the PharmaEssentia logo is a trademark of PharmaEssentia Corporation.

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New Data on Ropeginterferon Alfa-2b to Be Featured at EHA2022 - Business Wire

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Umoja Biopharma, Inc.

Partnership combines Umojas technologies in gene-edited iPSCs and immune differentiation for persistent anti-tumor activity with TreeFrog Therapeutics biomimetic platform for the mass-production of iPSC-derived cell therapies in large-scale bioreactors

Umoja Biopharma and TreeFrog Therapeutics Announce Collaboration to Address Current Challenges Facing Ex Vivo Allogeneic Therapies in Immuno-Oncology

Mass-production of human induced pluripotent stem cells in a 10L bioreactor using TreeFrog Therapeutics C-Stem technology. Photo credits: TreeFrog Therapeutics

SEATTLE and PESSAC, France, June 10, 2022 (GLOBE NEWSWIRE) -- Umoja Biopharma, Inc., an immuno-oncology company pioneering off-the-shelf, integrated therapeutics that reprogram immune cells to treat patients with solid and hematologic malignancies, and TreeFrog Therapeutics, a biotechnology company aimed at making safer, more efficient and more affordable cell therapies based on induced pluripotent stem cells (iPSCs), announced today that they have entered into a collaboration to evaluate Umojas iPSC platform within TreeFrogs C-Stem technology for scalable expansion and immune cell differentiation in bioreactors.

Together, the successful pairing of Umojas RACR engineered iPS cells and TreeFrogs C-Stem technology could overcome several challenges facing ex vivo allogeneic therapies, said Ryan Larson, Ph.D., Vice President and Head of Translational Science at Umoja. Two major industry-wide challenges include the ability to scale iPSC-based culture while maintaining cell health, quality, and efficient immune cell differentiation. TreeFrogs biomimetic C-Stem technology is the perfect complementary development platform for our RACR technology, a pairing which could result in controlled, efficient iPSC expansion and differentiation into immune cells, with improved yields and quality. In addition to enhancing the differentiation and yield of immune cells within the manufacturing process, our RACR system should bring therapeutic benefit to patients, allowing for safe in vivo engraftment and persistence of tumor-killing cells without requirements for toxic lymphodepleting chemotherapy.

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Umoja is developing an engineered iPSC platform that addressesmany challenges associated with ex vivo cell therapy manufacturing, including limited scalability and manufacturing complexity.Umojas iPSCs are engineered with a synthetic rapamycin-activated cytokine receptor (RACR) to drive differentiation to, and expansion of innate cytotoxic lymphoid cells, including but not limited to natural killer (NK) cells in the absence of exogenous cytokines and feeder cells. TreeFrogs proprietary C-Stem technology relies on the high-throughput encapsulation (>1,000 capsules/second) of hiPSCs within biomimetic alginate shells, which promote in vivo-like exponential growth and protect cells from external stress. In 2021, C-Stem was demonstrated to allow for unprecedented iPSC expansion in 10L bioreactors, while preserving stem cell quality. Also enabling direct in-capsule iPSC differentiation, C-Stem constitutes a scalable, end-to-end, and GMP-compatible manufacturing platform for iPSC-derived cell therapies.

Frdric Desdouits, Ph.D., Chief Executive Officer at TreeFrog added, Our primary goal is to bring the benefits of the C-Stem technology to patients as fast as possible, either through in-house programs or strategic alliances with cell therapy leaders. Partnering with Umoja is an important step forward in immuno-oncology. Besides scale-up and cell quality, the in vivo persistence of allogeneic therapies remains a critical challenge in the industry. We believe Umojas platform will allow for safer and more efficient allogeneic cell therapies in immuno-oncology. We look forward to rapidly advancing this joint approach to clinic and contributing to the future of off-the-shelf cancer treatments.

About Umoja BiopharmaUmoja Biopharma, Inc. is an early clinical-stage company advancing an entirely new approach to immunotherapy. Umoja Biopharma, Inc. is a transformative multi-platform immuno-oncology company founded with the goal of creating curative treatments for solid and hematological malignancies by reprogramming immune cells in vivo to target and fight cancer. Founded based on pioneering work performed at Seattle Childrens Research Institute and Purdue University, Umojas novel approach is powered by integrated cellular immunotherapy technologies including the VivoVec in vivo delivery platform, the RACR/CAR in vivo cell expansion/control platform, and the TumorTag targeting platform. Designed from the ground up to work together, these platforms are being developed to create and harness a powerful immune response in the body to directly, safely, and controllably attack cancer. Umoja believes that its approach can provide broader access to the most advanced immunotherapies and enable more patients to live better, fuller lives. To learn more, visithttp://umoja-biopharma.com/.

About TreeFrog TherapeuticsTreeFrog Therapeutics is a French-based biotech company aiming to unlock access to cell therapies for millions of patients. TreeFrog Therapeutics is developing a pipeline of therapeutic candidates using proprietary C-Stem technology, allowing for the mass production of induced pluripotent stem cells and their differentiation into ready-to-transplant microtissues with unprecedented scalability and cell quality. Bringing together over 80 biophysicists, cell biologists and bioproduction engineers, TreeFrog Therapeutics raised $82M over the past 3 years to advance its pipeline in regenerative medicine and immuno-oncology. The company is currently opening technological hubs in Boston, USA, and Kobe, Japan, to drive the adoption of C-Stem and build strategic alliances with leading academic, biotech and industry players in the field of cell therapy.

Umoja Biopharma Media Contact:Darren Opland, Ph.D.LifeSci Communicationsdarren@lifescicomms.com

TreeFrog Therapeutics Media Contact:Pierre-Emmanuel GaultierTreeFrog Therapeuticspierre@treefrog.fr

A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/012ae87d-b7c6-4fa2-81dc-c769877b182c

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Umoja Biopharma and TreeFrog Therapeutics Announce Collaboration to Address Current Challenges Facing Ex Vivo Allogeneic Therapies in Immuno-Oncology...

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Robotic innovations are accelerating at a startling rate, with the development of our humanoid counterparts taking sometimes hitting very close to the real thing. Consequently, the integration of these human-like robots into our society is a priority for many research groups across the globe. Now, a research team from Tokyo University has brought us even closer to this goal by growing human skin on a robotic skeleton to create a biohybrid robot.

The development of robots made to look like humans has sparked a fiery debate in research circles, prompting some to call for a clearer line between inanimate machines and autonomous robots. To illustrate this distinction, picture a ceiling fan whirling around at a constant speed when turned on manually this is an automated machine. But when we add a temperature sensor and a processor capable of storing user preferences and environmental data, the fan can then avoid obstructions and function autonomously based on the local temperature. The machine becomes an intelligent robot attuned to its environment a first step towards becoming more human.

At present, engineers are taking this premise even further, working on robots that have more and more in common with humans. If robots do become human-like, they could become widely used in any number of applications, but developing robots that feel like humans do isnt an easy feat.

The authors of a new study explain that blurring the line between humans and robots is one of the top priorities for humanoids tasked to interact with humans. But, presently, silicone skin used in robotics falls short when it comes to the delicate textures and expressions perceived by the human derma and underlying muscles. Additionally, synthetic skin cant heal, with patches or a silicone sealant used to repair rather than regenerate worn or torn areas.

To overcome this challenge, researchers have fashioned living skin sheets that can bond to the robots frames. However, conforming these biological coverings to the frameworks uneven surfaces and sharp, dynamic joints has proven extremely challenging. It got even worse when the humanoid moves the 3-dimensional (3D) metal chassis and joints damage the skin even further, causing gross failure.

So a new solution was needed. In the new study, the team cleared this hurdle using a novel technique that can grow living human skin onto a three-jointed robotic finger. The human-like skin consists of living cells and an extracellular matrix-a 3d support system holding cells in place-exhibiting self-healing properties while allowing the jointed structure underneath to move freely.

Our goal is to develop robots that are truly human-like, first author Professor Shoji Takeuchi, from the University of Tokyo, told ZME Science in an email. The silicone rubber covers that are commonly used today may look real from a distance or in photos or videos, but when you actually get up close, you realize that it is artificial. We think that the only way to achieve an appearance that can be mistaken for a human being is to cover it with the same material as a human being, i.e., living cells. Using cells would also allow the robot to work with the excellent biological functions of skin, such as its ability to self-repair.

To fashion the biohybrid robotic finger, the team first assembled the framework and coated it with parylene, a polymer used to protect implanted medical devices from moisture and contamination in the body. Similarly, the coating prevented any toxic materials in the robotic skeleton from leaching into the human skin equivalent and damaging it.

After this, they engineered a living dermis (the middle layer of skin responsible for protecting the human body from the outside world) that can feel different sensations and produce sweat. Once this was done, they then seeded the epidermis (the outermost layer of skin in the human body that protects against foreign substances and excessive water loss).

Expanding on this, the team explains that they placed the coated robotic finger in an outsized mold to engineer the dermis. Inside the mold, there was a solution of collagen and human dermal fibroblasts, the two main components that make up this connective tissue in the human body. To ensure the dermis was seeded correctly, the framework was cultured for 14 days, and an anchor was attached to the fingers base.

Takeuchi explains how the studys success hinges on this anchor because the collagen naturally shrinks, covering the robotic substructure tightly. Conversely, if there were no anchor at the base of the finger, the collagen would contract, retreating up the stem of the robotic digit. Like a primer, the dermis equivalent provided a uniform foundation for the next coat of cells (called keratinocytes) to form the epidermis.

This time, enough room was left in the mold to form a cap at the top of the structure to add extra tensile strength to the materials, enabling a uniform thickness of living skin across the frame. Results showed that this cap prevented damage to the human-like skin once the finger and joints were in motion.

One particular difficulty was culturing the skin to match its three-dimensional aspect. We found that we could adapt the skin to the curved 3D surface shape by culturing it on site, rather than making it elsewhere and attaching to the surface. By installing an appropriate anchor structure, the entire surface could be covered, Takeuchi told ZME Science.

This method can be used to cover the 3D surface of a robotic finger while controlling tissue shrinkage through anchor fixation. In addition, multidirectional seeding of keratinocytes enables us to uniformly form the epidermis layer, the team stated in their paper.

When testing the human-skin equivalent for tensile strength and water resistance, these layers produced a skin-like texture possessing moisture-retaining properties. Additionally, the biohybrid structure had enough strength and elasticity to allow curling and stretching movements and could handle electrostatically charged polystyrene foam packing balls when allocated a task.

The team also used a skin graft technique to evaluate their skins self-healing properties. To accomplish this, they cut a hole in the biohybrid fingers skin and applied a collagen bandage to the wound. Subsequently, this patch was integrated with the human-like skin to withstand continuous movement.

Despite these promising results, the group cautioned that their crafted skin is much weaker than human skin, and they dont expect this robot human-skin-equivalent to survive for very long. The team now plans to incorporate more biological structures into their skin to address these issues, such as sensory neurons, hair follicles, nails, and sweat glands.

Speaking to ZME Science about their results overall, Takeuchi concludes that It was exciting to find that a robotic finger, completely covered with skin, could stretch and contract when it moved, without breaking, and that it could repair itself by cell proliferation when damaged. We believe this is a great step toward a new biohybrid robot with the superior functions of living organisms.

The study is published in the journalMatter.

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