This article highlights some of the most recent drug target discoveries that could be used to develop and design a treatment for pancreatic cancer.

Scientists investigating pancreatic cancer have identified new targets which, with further research, could be the basis for developing future therapies. Listed below are five of the most recent target discoveries, in order of their journal publication dates, with the newest first.

Scientists at the Queen Mary University of London, UK and Zhengzhou University, China have developed a personalised vaccine system that may be able to delay the onset of pancreatic cancer.

Cells taken from mice, mutated chemically into pancreatic cancer cells and then infected with Adenovirus (AdV) as a prime or Vaccinia virus (VV) as a boost, create a vaccine product. The virus kills the cancerous cells in such a way that their antigens are released and are therefore able to prime the immune system to prevent pancreatic cancer returning.

Injection of the virus-infected cells into mice destined to develop pancreatic cancer doubled their survival rate, compared to their unvaccinated counterparts. The vaccine also delayed the onset of the condition in these mice.

Using cells from the recipient of the vaccine enables the immune system to respond to the exact antigens seen in tumour cells of the individual, resulting in a vaccine regime tailored to them.

Through this international collaboration, we have made progress towards the development of a prophylactic cancer vaccine against pancreatic cancer, said Professor Yaohe Wang, leader of the study, from Queen Mary University of London and the Sino-British Research Centre at Zhengzhou University in China.

Researchers at Sanford Burnham Prebys Medical Discovery Institute in the US have identified that a combination of two anti-cancer compounds, already approved for use to treat other cancers, shrank pancreatic tumours in mice.

Our study identifies a potential treatment combination that can immediately be tested against these aggressive tumours. We are already meeting with oncologists at Oregon Health & Science University, US to discuss how to advance this discovery into clinical evaluation, explained Dr Zeev Ronai, a professor in Sanford Burnham Prebys Tumor Initiation and Maintenance Program, also senior author of the study.

Scientists used L-asparaginase to starve pancreatic tumours of asparagine, an amino acid required by cells for protein synthesis. However, the tumour cells did not die, instead switching on a stress response pathway whereby they could produce asparagine themselves. Scientists then used an MEK inhibitor to block the stress response pathway, causing the pancreatic tumour to shrink.

L-asparaginase is already US Food and Drug Administration (FDA) approved to treat leukaemias and similarly the MEK inhibitor is approved for the treatment of solid tumours, including melanoma skin cancer.

This research lays the basis for the inhibition of pancreatic tumour growth by a combined synergistic attack based on asparagine restriction and MAPK signalling inhibition, says Dr Eytan Ruppin, chief of the Cancer Data Science Library at the National Cancer Institute (NCI) and co-author of the study.

Scientists from the Max Planck Institute for Biology of Ageing, Germany have identified that YME1L, a protease in the membrane of mitochondria, is activated when a cell uses glycolysis to produce energy anaerobically.

scientists were able to reduce tumour growth by switching off the glycolysis signalling pathway in the mitochondria

Cells adapt to oxygen deficiency by switching their energy supply to glycolysis, which ferments sugar without oxygen. This switch is often necessary in old age, as the cells in the body become poorly supplied with oxygen and nutrients.

Cancer cells can also face this problem; prior to angiogenesis, tumours are poorly perfused and so the tissue is deprived of oxygen. Oxidative stress in tumours drives the switch-on of multiple pathways. This includes the glycolysis pathway that alters the behaviour of the mitochondria to provide tumour cells with energy despite being starved of oxygen.

Scientists found that the YME1L protease is activated during the conversion to glycolysis. YME1L appears altered and breaks down various proteins in the organelles, preventing the formation of new mitochondria and causing the remaining organelles to change their metabolism. This process eventually stops as YME1L begins to degrade itself at high activity.

Researchers examined cancer cells originating from patients with pancreatic tumours and were able to reduce tumour growth by switching off the glycolysis signalling pathway in the mitochondria, with reproducible results both in the petri dish and in pancreatic tumours in mice.

There is currently no treatment available for pancreatic cancer. I believe that this protease can be a very interesting therapeutic target because we have seen that the signalling pathway is also active in human patients with pancreatic cancer, explained Thomas Langer, the Max Planck Director, continuing: However, there are no known substances that have an effect on this protease.

Researchers at the Crick Institute have identified cancer stem cells as a driver of pancreatic cancer growth. These cells can metastasise and differentiate into different tumour types to continue the spread of cancer.

Cancer stem cells appear at all stages of cancer growth so being able to identify where they are present could be vital in both targeting cancer and developing new treatments, according to the researchers. Analysis of gene expression in the cancer stem cells identified a protein, CD9, is present on tumour surfaces during development and when it is more established. This protein could therefore be used as a marker to help locate these cells.

A further development of the study established that this protein is not just a marker of cancer stem cells, but also promotes their malignant behaviour. By altering the amount of CD9 in tumour cells in mice, researchers found that reduced levels of this protein caused smaller tumours to form and increasing levels of CD9 created more aggressive cells able to form large tumours quickly.

These cells are vital to pancreatic cancer and if even just a few of them survive chemotherapy, the cancer is able to bounce back. We need to find effective ways to remove these cells and so stop them from fuelling cancer growth. However, we need more experiments to validate the importance of CD9 in human pancreatic cancer, says Victoria Wang, lead author and member of the Adult Stem Cell Laboratory at the Crick Institute.

A look into cancer stem cell metabolism also revealed CD9 increases the rate tumour cells take up glutamine, an amino acid which helps provide energy for cancer growth.

Now we know this protein is both linked to cancer stem cells and helps cancer growth, this could guide the development of new treatments that are targeted at the protein and so cut off the supply of glutamine to cancer stem cells, effectively starving the cancer, says Axel Behrens, corresponding author and group leader in the Adult Stem Cell Laboratory at the Crick Institute.

Scientists at Tel Aviv University, Israel have found that PJ34, a small molecule, causes human pancreatic cancer cells to self-destruct. The researchers tested PJ34 on xenografts (transplants) of human pancreatic tumours in mice.

this mechanism also exists in other types of cancer and therefore the treatment could be valuable for use on those resistant to current therapies

The mice were treated with a molecule called PJ34, which is permeable in the cell membrane but affects human cancer cells exclusively. This molecule causes an anomaly during the duplication of human cancer cells, provoking their rapid cell death. Thus, cell multiplication itself resulted in cell death in the treated cancer cells, explains Professor Malca Cohen-Armon, project lead at Tel Aviv Universitys Sackler Faculty of Medicine.

The treatment consisted of daily PJ34 injections for 14 days and four weeks later there was a relative drop of 90 percent in the number of cancer cells within the tumours of the mice. Cohen-Armon also noted there were no adverse side-effects observed in the mice.

This mechanism similarly exists in other types of cancer and therefore the treatment could be valuable for use on those resistant to current therapies. The molecule PJ34 is being tested in pre-clinical trials according to FDA regulations before clinical trials begin.

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Im hairythats just fact, and its something Ive always been aware of. Ill never forget being in summer camp as a kid, wearing shorts every day and becoming very aware that my legs were much hairier than those of the girls around me. Now, Im no longer ashamed of being hairyIve actually come to embrace it. But in the past two decades, Ive gotten very acquainted with epilators, including laser hair removal.

I got my first laser hair removal treatment two years ago. It seemed like everyone was getting it done, but as a black woman, I knew I wasnt everyone. For brown skin, laser hair removal isnt a spur-of-the-moment decision, a process that should be approached through a Groupon or the local nail salon (yes, some nail salons actually perform laser hair removal). For us, the process can be a bit more expensive and tedious, and should be approached with the utmost caution.

Why? Well, lasers target pigment, and due to the high content of melanin in our skin, risks of discoloration and hyper-pigmentation are astronomically higher. So thats why, according to Chris Karavolas, owner of Romeo And Juliette Laser Hair Removal, darker skin requires a completely different laser all together, and operators with much more experience. Darker skin complexions need to be careful because not all centers have the right lasers for dark skin, and even if they do they do, many do not have enough experience in treating dark skin, he says.

When it comes to those specialized lasers, there are two options. Its important to treat with an Nd:YAG laser, such as the Candela GentleYAG or GentleMax Pro, says Anne Chapas, M.D., medical director of Union Square Laser Dermatology in New York City. The wavelength of a YAG laser goes deeper into the skin than a diode laser and is less absorbed by the surrounding skin pigment, so it more successfully treats the stem cells of the hair follicle. Additionally, more treatments may be required than for fairer skin: Expect to receive at least six sessions, Chapas says.

Knowing all these things, I went into laser hair removal with cautious optimism. I chose to treat my Brazilian areayears of improper hair removal had left me with ingrown and severe discoloration, to the point that my wax lady refused to continue treating me because my skin had gotten so irritated. I was sure that I wanted to continue being hair-free down there, so I decided to get laser hair removal on my vagina area. I was nervous heading into my first appointment, so I made sure to ask the aesthetician at Romeo & Juliette in-depth questions about her experience with dark skin, as well as requesting to see photos of previous clients and inquiring about a patch test. Then, it was showtime.

I like to think that I have a pretty high pain tolerance. However, laser removal certainly made me question that belief. I wont lie and say that it didnt hurt. It did. It felt like being snapped, hard, by rubber bands, but it was quick, and the results I saw were almost immediate. I was advised to shave right before the treatment, in order to give the laser immediate access to the hair follicle, and because, well, nobody likes the smell of burning hair. After about 10 minutes, I was sent on my way and advised to come back in another six weeks. Laser hair removal requires a strict schedule and works in conjunction with the hair cycle for optimal results. Every appointment thereafter was just as easy, and now, completely through all six of my sessions, my hair is almost completely gone, with the occasional stubble appearing here and there.

So how do you know if your skin will need one of these specialized lasers? Different ethnicitiesregardless of skin tonereact differently to lasers. Your skin may appear to be a 3 or 4 on the Fitzpatrick scale, but if youre, say, Latin or Asian, it could react to the laser as a 6 would, Dr. Chapas says. So be sure that youre only going to facilities that have operators trained in dermatology, and are skilled enough to differentiate your skin tone.

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When embryonic Stem Cell therapy was first discovered in 1998, it changed the face of medicine. The idea of being able to regenerate and replace damaged cells seemed futuristic at the time, yet today such treatments are commonplace. Now, science has taken another quantum leap this time into the nano-sized world of exosomes, tiny bubbles that grow out of cell walls and contain much of the information contained within the cell including Growth factors, microRNA and messenger RNA. Mesenchymal stem cell (MSC) exosome therapy is currently one of the hottest trends in regenerative medicine, one that patients at AM Medical in Yelm can now experience.

Everyone has heard of stem cell therapy, but it turns out that its not the stem cells that are doing the work, says Dr. Ana Mihalcea, President of AM Medical. Its the exosomes that carry the information of regeneration. Infused stem cells, attach to blood vessel walls, and then give off exosomes.

Exosomes have several key differences from stem cells; they do not get removed from the circulation like stem cells, which are in the body for less than 72 hours before they get destroyed by the immune system; they do not produce a rejection reaction because they are not a cell and contain no DNA, and they pass the blood brain barrier, Mihalcea notes. In a study on stroke scientists fluorescently tagged exosomes, and the infused exosomes went exactly to the region where the stroke had occurred, she adds. The same was not true of stem cells as they do not cross the blood brain barrier.

As a result of their powerful cargo, exosomes can be used to address a multitude of conditions, including arthritis, autoimmune disorders, cardiovascular and neurogenerative diseases like Parkinsons and Alzheimers. Old cells can be reprogrammed by MSC exosomes as the target cells can transcribe the microRNA into functional proteins. Just like a virus, the exosome information of the young stem cells can infect the old cells with Youth, explains Mihalcea.

Spinal cord injuries are an area in which exosomes have produced dramatic results. Mihalcea cites the example of Dr. Douglas J. Spiels Interventional Pain Specialty Practice in NJ. Dr. Spiel has been able to rehabilitate spinal cord injuries with Exosome infusions into the spine and intravenously, she says. After several weeks, hes had patients regain muscle strength and sensation. These are prolonged, ongoing regenerative effects that continued to improve for months after the infusion.

When it comes to autoimmune diseases, inflammation plays a key role. Again, exosomes are able to reduce the problem by downregulating inflammation. TGF Beta 3 [Transforming growth factor beta-3] is the most important anti-inflammatory protein in the body and is abundant in MSC exosomes says Mihalcea. Many more Growth factors for blood vessel growth, neuronal and other tissue growth are present, allowing regenerative effects in all organ systems including skin wounds and burns.

The exosomes at AM Medical come from a laboratory in Florida that conducted pioneering research in the field. They come from perinatal mesenchymal stem cells and are scanned for any possible viruses to ensure their safety. Once harvested, the exosomes are concentrated so they can be infused in large doses.

For patients who qualify, the infusion process takes 10 to 15 minutes. Already, its been producing results for AM Medical patients. Weve had people with arthritis and chronic pain who had great responses, Mihalcea notes. There is an overall increase in wellbeing and sense of rejuvenation that is definitely noticeable.

Perhaps one of the largest sources of excitement over exosomes has to do with their anti-aging effects. Recently, ideas about the root causes of aging have been evolving, according to Mihalcea. Its been thought that aging occurs due to multiple different reasons like stem cell exhaustion, epigenetic changes, telomere shortening and others, she explains. It turns out that exosomes can modify almost all the hallmarks of aging. Theyre changing epigenetic expression to youthful function, and there are many potential applications. This is a new frontier in regenerative medicine that can help many people.

Learn more by watching Dr. Ana Mihalceas video on Exosomes The New Frontier Part 1: Longevity and Age reversal or reading further on the AM Medical website.

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Biomedical engineers at Duke University have developed a system that allows for real-time observations of individual cells in the colon of a living mouse.

Researchers expect the procedure to allow new investigations into the digestive systems microbiome as well as the causes of diseases such as inflammatory bowel disease and colon cancer and their treatments.

The procedure described online today (December 11, 2019) in Nature Communications involves surgically implanting a transparent window into a mouses abdominal skin above the colon. Similar setups are already being used to allow live looks into the detailed inner workings of the brain, spinal cord, liver, lungs and other organs. Imaging a live colon, however, is a slipperier proposition.

A brain doesnt move around a lot, but the colon does, which makes it difficult to get detailed images down to a single cell, said Xiling Shen, the Hawkins Family Associate Professor of Biomedical Engineering at Duke University. Weve developed a magnetic system that is strong enough to stabilize the colon in place during imaging to obtain this level of resolution, but can quickly be turned off to allow the colon to move freely.

This video shows green fluorescent colon neurons activated by neurostimulation in real-time. This is the first time that sacral nerve stimulation, an FDA approved therapy for colon motility disorders, has definitively been shown to activate neurons in the colon in live animals, explaining why the therapy might work.

Credit: Xiling Shen, Duke University

Immobilizing the colon for imaging is a tricky task for traditional methods such as glue or stitches. At best they can cause inflammation that would ruin most experiments. At worst they can cause obstructions, which can quickly kill the mouse being studied.

To skirt this issue, Shen developed a magnetic device that looks much like a tiny metal nasal strip and can be safely attached to the colon. A magnetic field snaps the colon into place and keeps it stable during imaging, but once turned off, leaves the colon free to move and function as normal.

A vital organ that houses much of the digestive systems microbiome, the colon can be afflicted by diseases such as inflammatory bowel disease, functional gastrointestinal disorders, and cancer. It also plays a key role in regulating the immune system, and can communicate directly with the brain through sacral nerves.

There is a great need to better understand the colon, because it can suffer from so many diseases and plays so many roles with significant health implications, Shen said. In the study, Shen and his colleagues conducted several proof-of-principle experiments that provide starting points for future lines of research.

The researchers first colonized a living mouse colon with E. coli bacteria, derived from Crohns disease patients, that had been tagged with fluorescent proteins. The researchers then showed they could track the migration, growth and decline of the bacteria for more than three days. This ability could help researchers understand not only how antagonistic bacteria afflict the colon, Shen says, but the positive roles probiotics can play and which strains can best help people with gastrointestinal disorders.

In the next experiment, mice were bred with several types of fluorescent immune cells. The researchers then induced inflammation in the colon and carefully watched the activation of these immune cells. Shen says, this approach could be used with various types of immune cells and diseases to gain a better understanding of how the immune system responds to challenges.

Shen and his colleagues then showed that they could tag and track colon epithelial stem cells associated with colorectal cancer throughout radiation treatment. They also demonstrated that they could watch nerves throughout the colon respond to sacral nerve stimulation, an emerging therapy for treating motility and immune disorders such as functional gastrointestinal disorders and irritable bowel disorder.

While we know electrically stimulating the sacral nerves can alleviate the symptoms of these gastrointestinal disorders, we currently have no idea why or any way to optimize these treatments, Shen said. Being able to see how the colons neurons respond to different waveforms, frequencies and amplitudes of stimulation will be invaluable in making this approach a better option for more patients.

###

Reference: An intravital window to image the colon in real time by Nikolai Rakhilin, Aliesha Garrett, Chi-Yong Eom, Katherine Ramos Chavez, David M. Small, Andrea R. Daniel, Melanie M. Kaelberer, Menansili A. Mejooli, Qiang Huang, Shengli Ding, David G. Kirsch, Diego V. Bohrquez, Nozomi Nishimura, Bradley B. Barth and Xiling Shen, 11 December 2019, Nature Communications.DOI: 10.1038/s41467-019-13699-w

This work was supported by National Institutes of Health (R35GM122465, OT2OD023849), the Defense Advanced Research Projects Agency (N66001-15-2-4059) and the National Cancer Institutes (R35CA197616).

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A Real-Time Window Into the Hidden World of the Colon of a Living Animal - SciTechDaily

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PATIENTS with life-changing injuries could walk again thanks to a pioneering electrical spinal implant. The device has proven effective in trials on macaque monkeys and researchers in Canada are hopeful it will be available for use on human patients in as little as a decade.

Lead researcher Dr Vivian Mushahwar, of the University of Albertas Neuroscience and Mental Health Institute, said: We think that intraspinal stimulation itself will get people to start walking longer and longer, and maybe even faster.

That in itself becomes their therapy. Theres been an explosion of knowledge in neuroscience over the last 20 years.

Were at the edge of merging the human and the machine.

The device features hair-like electrical wires that plunge deep into the spinal grey matter, sending electrical signals to trigger the networks that already know how to do the hard graft.

To work alongside the implant, the team created a map to identify which parts of the spinal cord trigger the hip, knees, ankles and toes, and the areas that put movements together.

People tend to think the brain does all the thinking, but the spinal cord has built-in intelligence, said Dr Mushahwar.

A complex chain of motor and sensory networks regulate everything from breathing to bowels, while the brain stems contribution is basically go! and faster!

Your spinal cord isnt just moving muscles, its giving you your natural gait. Being able to control standing and walking would improve bone health, improve bowel and bladder function, and reduce pressure ulcers, the researchers say.

For those with less severe spinal injuries, an implant could be therapeutic, removing the need for months of gruelling physical therapy regimes that have limited success, they add. The team are now focused on refining the hardware further by miniaturising an implantable stimulator and getting approval for human trials.

The first generation of the implants will require a patient to control walking and movement through physical means, but longer term, the implants could potentially include a direct connection to the brain, they say.

Dr Mushahwar said it has the power to transform lives.

Imagine the future, a person just thinks and commands are transmitted to the spinal cord. People stand up and walk. This is the dream, she said.

23million The number of lives saved globally by vaccines between 2000 and 2018, as estimated by the US Centers for Disease Control and Prevention

WHILE they may not be as talkative as Jungle Books King Louie, it turns out wild orangutans have some serious communication skills.

Researchers at the University of Exeter in Devon have identified 11 vocal signals and 21 physical gesture types that the apes use to communicate with one another.

The findings reveal orangutans are highly responsive to communication, reacting either before gesturing ended or in less than a second in 90 per cent of communications.

The team studied video footage of 16 orangutans consisting of seven mother-child pairs and a pair of siblings, noting a total of 1,299 communicative signals 858 vocal signals and 441 gestures.

The sounds included the kiss squeak a sharp kiss noise created while inhaling the grumph (a low sound lasting one or two seconds made on the inhale), the gorkum (a kiss squeak followed by a series of multiple grumphs) and the self-explanatory raspberry.

Gestures included beckoning, stamping, pushing out a lower lip, shaking objects and presenting a body part.

The eight identified goals or requests of their communications are acquire object, climb on me, climb on you, climb over, move away, decrease intensity, resume play and stop that.

Orangutans are the most solitary of all the apes, which is why most studies have been done on African apes, and not much is known about wild orangutan gestures, said University of Exeter scientist Dr Helen Morrogh-Bernard. We spent two years filming more than 600 hours of footage of orangutans in the Sabangau peat swamp forest in Borneo, Indonesia.

While some of our findings support what has been discovered by zoo-based studies, other aspects are new and these highlight the importance of studying communication in its natural context.

More signals are likely to be identified in the future, the researchers say.

1/2million The number of hermit crabs estimated to have been killed by plastic waste, according to a study by the Natural History Museum

WE MAY like to put our feet up at Christmas but for older people, even a short period of reduced physical activity could lead to significant loss of muscle mass and gains in body fat.

Just a fortnight of walking fewer than 1,500 steps a day could have a severe impact making daily tasks harder and kick-starting chronic health conditions a University of Liverpool study found.

The team measured the effect of two weeks inactivity on 47 participants, around half of whom were elderly. All lost muscle size, strength and bone mass. But the older volunteers also experienced a big drop in cardio fitness and mitochondrial function the way cells produce energy.

If the gym is hard to get to, people should be encouraged to just meet 10,000 steps, said researcher Juliette Norman. Even this can guard against reductions in muscle and bone health as well as maintaining healthy levels of body fat.

EXPERIENCING stress at a young age could help you live longer. Juvenile Caenorhabditis elegans worms that produce more oxidants and free radicals, which are associated with stress and ageing, last longer, US researchers at the University of Michigan found. Study leader Daphne Bazopoulou said early exposure may make you better able to fight stress later in life.

AGE can be predicted by protein levels in the blood. Using blood samples from more than 4,000 18- to 95-year-olds, scientists at Stanford University, California, found 373 proteins that peak and trough across a lifespan. The blood test can also show how physiological and real ages compare, which could flag those more at risk of ageing-related illnesses or reveal the ageing effect of drugs and treatments.

To brag flagrantly is, most of us realise, unbecoming, and so a favoured mitigation tactic has emerged: the humblebrag. This is a way of masking a boast, either in a feigned complaint or as fake modesty, such as its so exhausting staying in touch with all my friends or if someone told me Id pass with first-class honours, Id never have believed them. Unfortunately for humblebraggers, the tactic is too transparent to work. Last year, researchers at the University of North Carolina and Harvard Business School found humblebraggers are less liked, and perceived as less competent, because they appear insincere.

Astronomers have found magnetic field strengths near supermassive black holes can be as strong as their intense gravitational fields. These fields are able to expel material from the vicinity of the black hole to form highly energetic outflows called jets. However, this process is not acting on material that has already passed beyond the black holes event horizon, where not even light can escape. Such material would need to be accelerated to the speed of light to escape, which would require an infinite amount of energy. No magnet, however powerful, could provide this.

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Lab: Electric implant could allow people with crippling injuries to walk again - Metro Newspaper UK

Spinal Cord Stem Cells Comments Off on Lab: Electric implant could allow people with crippling injuries to walk again – Metro Newspaper UK | December 12th, 2019

A campaign has been launched to fund vital research into Multiple Sclerosis in Sheffield. Catherine Scott reports.

Sheffield Hospitals Charity is appealing for support to fund a new research project being undertaken in the city that could help find a cure for Multiple Sclerosis (MS).

MS affects more than 100,000 people in the UK and is the most common cause of physical disability in young adults. It can cause pain, fatigue, problems with memory and thinking, speech and vision problems, but most noticeably, loss of mobility.

The disease occurs when the bodys immune system becomes faulty, instead of protecting against viruses and germs, the immune system attacks the nerve fibres in the brain and spinal cord.

Currently, there is no effective treatment for MS and existing treatments focus on alleviating symptoms and their impact on long term disability. But Sheffield scientists Professor Basil Sharrack and Professor John Snowden are determined to find better treatments and even a cure for MS.

One of these 100,000 people, and a patient of Professors Sharrack and Snowden, is Allison Parfitt, 50 from Sheffield who has had MS since 2008.

Allison explained the impact this had on her life: When I was diagnosed, I felt like my whole world was falling apart around me. MS controls my life. I feel exhausted all over my whole body is tired, right down to my fingertips.

Some days, every move I make is like walking through quick sand, everything is slower. My mind feels like theres a fog inside it, stopping me from concentrating or thinking clearly. My legs wont support me anymore, so I have to use crutches or a wheelchair to get around.

Im a very independent person but that is being taken away from me. I cant even go to the shops on my own anymore, I need someone with me.

The research going on here in Sheffield gives me hope. Hope that one day, Ill receive that call to say a cures been found. It would be good to know that people in the future wont have to go through what I have.

The project will be delivered by Professor Basil Sharrack, Consultant Neurologist and Professor John Snowden, Consultant Haematologist from Sheffield Teaching Hospitals.

Professor Sharrack explained how supporting his research could help future patients like Allison.

For the first time ever, weve been able to reverse disability in some patients with MS using a brand new stem cell therapy trialled right here in Sheffield and its like nothing weve seen before.

First we destroy the faulty immune system that causes MS and then we replace it with a healthy new one, grown from a patients own stem cells. In some patients it has reversed some of the disabilities associated with their MS condition, but this isnt suitable for everyone as stem cell treatment can be quite aggressive.

The pair wants to build upon the success of their stem cell work by trying to identify a biomarker a test which will allow clinicians to predict how each patients disease is likely to progress.

Professor John Snowden explained further: Once a biomarker is identified, it can be used to measure the progression of MS and predict how the disease will develop and how it will respond to treatment.

This could help us revolutionise the way in which MS patients are treated. Doctors will be able to make more informed choices about which treatment course to follow and when to start and escalate the treatment to prevent long-term disability.

We will gain a crucial understanding about how MS works, which will help provide new information that could help identify new drugs that could potentially cure MS.

For the first time, it feels like a cure for MS could be right around the corner. Thats why it is so critical that our research goes ahead.

No other hospital in the UK combines neurology and haematology in the way we do here in Sheffield we are in a unique position. We want to get started with our research as soon as possible so that people living with MS right now can benefit from the discoveries we make. By donating to Sheffield Hospitals Charitys Multiple Sclerosis Appeal you could help create a better future for people like Allison.

Sheffield Hospitals Charity is aiming to raise 200,000 to support the research project. Anyone interested in donating or fundraising to enable the MS research to be undertaken can find out more at http://www.sheffieldhospitalscharity.org.uk/msresearch

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How research in Sheffield is giving hope to MS sufferers - Yorkshire Post

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Led by researchers at Baylor College of Medicine, a study published in the journal Cell Stem Cell reveals a new mechanism that contributes to adult bone maintenance and repair and opens the possibility of developing therapeutic strategies for improving bone healing.

Adult bone repair relies on the activation of bone stem cells, which still remain poorly characterized, said corresponding author Dr. Dongsu Park, assistant professor of molecular and human genetics and of pathology and immunology at Baylor. Bone stem cells have been found both in the bone marrow inside the bone and also in the periosteum the outer layer of tissue that envelopes the bone. Previous studies have shown that these two populations of stem cells, although they share many characteristics, also have unique functions and specific regulatory mechanisms.

Of the two, periosteum stem cells are the least understood. It is known that they comprise a heterogeneous population of cells that can contribute to bone thickness, shaping and fracture repair, but scientists had not been able to distinguish between different subtypes of bone stem cells to study how their different functions are regulated.

In the current study, Park and his colleagues developed a method to identify different subpopulations of periosteum stem cells, define their contribution to bone fracture repair in live mouse models and identify specific factors that regulate their migration and proliferation under physiological conditions.

Periosteal stem cells are major contributors to bone healing

The researchers discovered specific markers for periosteum stem cells in mouse models. The markers identified a distinct subset of stem cells that contributes to life-long adult bone regeneration.

We also found that periosteum stem cells respond to mechanical injury by engaging in bone healing, Park said. They are important for healing bone fractures in the adult mice and, interestingly, their contribution to bone regeneration is higher than that of bone marrow stem cells.

In addition, the researchers found that periosteal stem cells also respond to inflammatory molecules called chemokines, which are usually produced during bone injury. In particular, they responded to chemokine CCL5.

Periosteal stem cells have receptors molecules on their cell surface that bind to CCL5, which sends a signal to the cells to migrate toward the injured bone and repair it. Deleting the CCL5 gene in mouse models resulted in marked defects in bone repair or delayed healing. When the researchers supplied CCL5 to CCL5-deficient mice, bone healing was accelerated.

The findings suggested potential therapeutic applications. For instance, in individuals with diabetes or osteoporosis in which bone healing is slow and may lead to other complications resulting from limited mobility, accelerating bone healing may reduce hospital stay and improve prognosis.

Our findings contribute to a better understanding of how adult bones heal. We think this is one of the first studies to show that bone stem cells are heterogeneous and that different subtypes have unique properties regulated by specific mechanisms, Park said. We have identified markers that enable us to tell bone stem cell subtypes apart and studied what each subtype contributes to bone health. Understanding how bone stem cell functions are regulated offers the possibility to develop novel therapeutic strategies to treat adult bone injuries.

Reference

Ortinau et al. (2019) Identification of Functionally Distinct Mx1+SMA+ Periosteal Skeletal Stem Cells. Cell Stem Cell. DOI: https://doi.org/10.1016/j.stem.2019.11.003

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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New Mechanism of Bone Maintenance and Repair Discovered - Technology Networks

Bone Marrow Stem Cells Comments Off on New Mechanism of Bone Maintenance and Repair Discovered – Technology Networks | December 12th, 2019

Regardless of the advancements made in understanding the genetic and molecular landscapes of acute myeloid leukemia (AML) in young patients, bespoke treatment approaches are yet to be adopted. Based on the NCCN 2009 guidelines,2 AML risk stratification depends on genetic/cytogenetic abnormalities of AML cells and groups patients into favorable-risk" (FR), intermediate-risk (IR) or poor-risk (PR) categories. This will then determine the treatment pathway of whether they will receive allogeneic stem cell transplantation (allo-SCT). Currently, treatment of IR patients is less defined and often post-induction treatment with allo-SCT is adopted. Nevertheless, this approach does not appropriately take into account the heterogeneity in this group regarding the risk of disease relapse. Despite high complete remission (CR) rates in these patients, overall survival (OS) remains low due to the high rate of relapse incidence.3

Adriano Venditti et al., from the Gruppo Italiano Malattie EMatologiche dellAdulto (GIMEMA) Foundation investigated in a multicenter prospective clinical trial (NCT01452646) the benefit of risk-adapted, MRD-based therapy in young adults with AML. This study consisted of the integration of pre-treatment cytogenetics/genetics with post-treatment MRD assessment (detected using multiparametric flow cytometry [MFC]) to stratify patients to receive post-consolidation autologous stem cell transplantation (auto-SCT) or allo-SCT.1

In conclusion, the authors recognized that this study has intrinsic limitations due to scientific progression over time, such as better understanding of basic biology, new AML classification and increased MRD monitoring, which makes the historical control not wholly comparable. However, this is the first study that attempts to apply a prospective program of risk-adapted, MRD-driven treatment in patients with NCCN-IR AML, that integrates genetics and post-consolidation MRD status.

Patients that were in the NCCN-IR group demonstrated that allo-SCT can be avoided if patients are tested MRD-negative after induction therapy. In contrast, if patients are MRD-positive, allo-SCT improved OS and prolonged DFS to comparable levels as those of patients in the NCCN-FR group. Patients in the IR group, who could not be monitored for LAIP had an inferior 2-year OS of 50% when compared to the other risk groups. This suggests that, in this group, a number of patients could have benefited from an allo-SCT. Further studies that integrate baseline factors and monitor MRD are needed, as it could potentially be a promising tool to refine and customize outcome prediction in patients with AML.

Link:
GIMEMA AML1310 trial of risk-adapted, MRD-guided therapy for young patients with newly diagnosed AML - AML Global Portal

Bone Marrow Stem Cells Comments Off on GIMEMA AML1310 trial of risk-adapted, MRD-guided therapy for young patients with newly diagnosed AML – AML Global Portal | December 12th, 2019

By Aprille HansonAssociate Editor

Aprille Hanson

Deacon Butch King and his wife Debbie (left) stand with their daughter Paula Draeger (center) in front of the Seed of Hope garden at UAMS Winthrop P. Rockefeller Cancer Institute in Little Rock Dec. 4. Last month, King was able to place a seed of hope token into the garden, signifying he is cancer-free, thanks to a stem cell donation from his daughter.

Aprille Hanson

Deacon Butch King and his wife Debbie (left) stand with their daughter Paula Draeger (center) in front of the Seed of Hope garden at UAMS Winthrop P. Rockefeller Cancer Institute in Little Rock Dec. 4. Last month, King was able to place a seed of hope token into the garden, signifying he is cancer-free, thanks to a stem cell donation from his daughter.

Deacon Butch King was given a gift in 2017. He was diagnosed with a rare disease MDS/MPN, myelodysplastic/myeloproliferative neoplasm-unclassifiable to be exact.

The hybrid disease results when bone marrow overproduces unhealthy blood cells, according to University of Arkansas for Medical Sciences in Little Rock.

The diagnosis sent the family on a harrowing journey for the next two and a half years: four changes of insurance coverage and medical facilities, 19 rounds of chemotherapy, 430 lab results, 14 bone marrow biopsies, 11.25 gallons of donated blood and the disease progressing to Acute Myeloid Leukemia.

Looking at a deadly disease as a gift takes a radical faith in God, one that King and his wife Debbie have carried with grace to his cancer-free diagnosis Nov. 4.

It was given to us as a gift. And how do we manage gifts? We care for them, we nurture them, we polish them, show them off with pride and we give thanks to God. Those are his words, our words together. We had a gift and we had to manage it, we didnt get a choice, his wife said.

King was ordained a deacon in 2012, serving at Immaculate Conception Church in North Little Rock. The couple has four children, 12 grandchildren and six great-grandchildren, with another on the way in March. After 23 years of serving in the U.S. Air Force working in secure communications, he spent 22 years with the U.S. Postal Service.

In October 2016, he had a metal stent placed in his heart and could not have any surgeries for the following six months. In November of that year, he twisted his knee at work. When he was finally ready to have knee surgery in May, his lab work was irregular. In June they learned he had developed a rare blood disorder, MDS, which later in the year progressed to MPN. It required a stem cell transplant, with only a 30 percent chance of surviving a transplant.

I was kind of stunned at first, King said. As a deacon, he had been used to visiting the sick in nursing homes and hospitals.

This is one of the stories you can say, I know how you feel because Ive been there or were praying for you and really mean it, he said.

With every roadblock of insurance not covering the procedure or a hospital turning the transplant down because he was high risk, faith prevailed.

In December 2017, their youngest daughter Paula Draeger, 38, was a perfect match for a stem cell transplant, an extremely rare result.

OK, we can do this; were going to heal him. Weve got the perfect match. If this doesnt work, nothing will. So that was just kind of the reaction, lets do it, the married mother of two said.

Debbie King said, Shes a Spina bifida baby. We were told that she would be a vegetable when she had her spinal surgery. So shes a miracle to be here; long before this ever came God had a plan.

Once Medicare kicked in, insurance would cover a transplant if a clinical trial was available. It led the family to 13 visits to University of Oklahoma Stephenson Cancer Center in Oklahoma City, though they refused the transplant.

Debbie King said they specifically chose Oklahoma City because the family had been, and still are, praying daily for Blessed Stanley Rothers intercession.

The martyr, who grew up on a farm in Okarche, Okla., was declared blessed on Sept. 23, 2017, in Oklahoma City. He was killed in 1981 while serving his people in Guatemala.

He needed a miracle. And we said God provides miracles, Debbie King said.

Before we started any treatment we would place the entire illness and what would be happening at Blessed Stanley Rothers gravesite in Oklahoma City, visiting 11 times, she said.

Whats the miracle? The miracle is the faith. And thats what Butch has said, she said.

On March 13, the Kings were told they wouldnt be continuing the trial in Oklahoma.

We were ready to just be on maintenance and enjoy the days we had, she said. On March 14, our 44th wedding anniversary we were celebrating what we thought could be our last one.

But Dr. Appalanaidu Sasapu, hematologist oncologist with the UAMS Stem Cell Transplantation and Cellular Therapy Program, never gave up on them. Because Kings disease had progressed to leukemia in April, the stem cell procedure could now be done at UAMS and covered by insurance.

Draeger said the stem cell donations, done over a weekend via a port, were simple, with no side effects aside from building her energy up in the following week.

For what youre able to give somebody, what you have to endure pales in comparison to what hes been through and what you can give him, she said.

King no longer has the blood disease and is cancer free, though he will continue at least a years worth of chemotherapy treatments.

Since his diagnosis, they attend the smaller St. Patrick Church in North Little Rock for Mass, but he cannot yet return to ministry.

We do our prayer time in the mornings and evenings, we count our blessings every night before we go to bed and we just know, what was our blessing today? Did we see somebody that we havent seen before that God put in our path? Is it a new doctor who is going to take this on? King said.

But through this whole process weve been truly blessed, had no regrets. If I had to do it over, if thats the path of my life that God wants me to take, then Ill do it.

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Deacon Butch King learns to accept the 'gift' of cancer - Arkansas Catholic

Bone Marrow Stem Cells Comments Off on Deacon Butch King learns to accept the ‘gift’ of cancer – Arkansas Catholic | December 12th, 2019

AgeX Therapeutics, Inc. (NYSE American: AGE) and Lineage Cell Therapeutics, Inc. (NYSE American and TASE LCTX), announced today that the United States Patent and Trademark Office (USPTO) has issued U.S. Patent No. 10,501,723, entitled "Methods of Reprogramming Animal Somatic Cells" covering what is commonly designated "induced Pluripotent Stem (iPS) cells. The issued claims include methods to manufacture pluripotent cells capable of becoming any cell in the body. The patent has an early priority date, having been filed before the first scientific publication of Shinya Yamanaka, for which he won the Nobel Prize for Physiology or Medicine in 2012.

"This patent broadly describes multiple techniques for reprogramming cells of the body back to the all-powerful stem cell state," said Dr. Michael D. West, CEO of AgeX and first inventor on the patent. "Perhaps more significantly, it includes certain factors that address some of the difficulties currently encountered with iPS cells. It also reflects the foundational work our scientists have undertaken to apply reprogramming technology to age-reversal, specifically, induced Tissue Regeneration (iTR) which is currently a focus of AgeX product development." A video describing the significance of the patent in AgeXs product development is available on the AgeX website.

"The issuance of this patent highlights Lineages dominant position in the field of cell therapy," stated Brian M. Culley, CEO of Lineage. "Our efforts to develop new treatments rely on well-characterized and NIH-approved human cell lines. These lines are not genetically manipulated, which avoids the safety concerns associated with genetic aberrations arising from the creation of iPS cells. We believe the Lineage cell lines provide the safest option for our current clinical-stage programs, particularly in immune-privileged anatomical sites such as the eye (OpRegen for the treatment of dry AMD) and spinal cord (OPC1, for the treatment of spinal cord injury). However, the vast intellectual property estate which underlies our cell therapy platform has never been limited to these particular cell lines. As one example, this newly-issued patent provides us with proprietary methods for producing induced pluripotent stem cells, or, as it was practiced by us prior to Yamanaka, Analytical Reprogramming Technology (ART). In certain settings, an ART/iPS approach might offer important advantages, such as for an autologous treatment or when the selection of preferential attributes from a series of iPS lines is desirable. Questions as to which stem cell technology is preferred ultimately will be answered by clinical safety and efficacy and likely will be indication-specific, so we believe it is in the best interest of our shareholders to generate patented technology which enables us to pursue programs in either or both formats which we believe will ensure the highest probability of success."

Induced Pluripotent Stem Cells (iPS) are typically derived from adult skin or blood cells which have been "reprogrammed" or "induced" to retrace their developmental age and regain the potential to form all of the young cell and tissue types of the body. In 2010 inventors of the -723 patent issued today demonstrated that this reversal of developmental aging even extended to the telomere clock of cell aging. This reprogramming technology provides an alternate source of starting material for the manufacture of potentially any type of human cell needed for therapeutic purposes. Because iPSCs can be derived directly from adult tissues, they can be used to generate pluripotent cells from patients with known genetic abnormalities for drug discovery or as an alternative source of cell types for regenerative therapies.

U.S. Patent No. 10,501,723, entitled "Methods of Reprogramming Animal Somatic Cells" was assigned to Advanced Cell Technology of Marlborough, Massachusetts (now Astellas Institute for Regenerative Medicine) and licensed to Lineage and sublicensed to AgeX Therapeutics for defined fields of use. Inventors of the patent include Michael D. West, CEO of AgeX and previous CEO of Advanced Cell Technology, Karen B. Chapman, Ph.D., and Roy Geoffrey Sargent, Ph.D.

About AgeX Therapeutics

AgeX Therapeutics, Inc. (NYSE American: AGE) is focused on developing and commercializing innovative therapeutics for human aging. Its PureStem and UniverCyte manufacturing and immunotolerance technologies are designed to work together to generate highly-defined, universal, allogeneic, off-the-shelf pluripotent stem cell-derived young cells of any type for application in a variety of diseases with a high unmet medical need. AgeX has two preclinical cell therapy programs: AGEX-VASC1 (vascular progenitor cells) for tissue ischemia and AGEX-BAT1 (brown fat cells) for Type II diabetes. AgeXs revolutionary longevity platform induced Tissue Regeneration (iTR) aims to unlock cellular immortality and regenerative capacity to reverse age-related changes within tissues. AGEX-iTR1547 is an iTR-based formulation in preclinical development. HyStem is AgeXs delivery technology to stably engraft PureStem cell therapies in the body. AgeX is developing its core product pipeline for use in the clinic to extend human healthspan and is seeking opportunities to establish licensing and collaboration agreements around its broad IP estate and proprietary technology platforms.

Story continues

For more information, please visit http://www.agexinc.com or connect with the company on Twitter, LinkedIn, Facebook, and YouTube.

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineages programs are based on its proprietary cell-based therapy platform and associated development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally-differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed either to replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineages clinical assets include (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase I/IIa development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase I/IIa development for the treatment of acute spinal cord injuries; and (iii) VAC2, an allogeneic cancer immunotherapy of antigen-presenting dendritic cells currently in Phase I development for the treatment of non-small cell lung cancer. Lineage is also evaluating potential partnership opportunities for Renevia, a facial aesthetics product that was recently granted a Conformit Europenne (CE) Mark. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

Forward-Looking Statements

Certain statements contained in this release are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not historical fact including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates" should also be considered forward-looking statements. Forward-looking statements involve risks and uncertainties. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the business of AgeX Therapeutics, Inc. and its subsidiaries, particularly those mentioned in the cautionary statements found in more detail in the "Risk Factors" section of AgeXs Annual Report on Form 10-K and Quarterly Reports on Form 10-Q filed with the Securities and Exchange Commissions (copies of which may be obtained at http://www.sec.gov). Subsequent events and developments may cause these forward-looking statements to change. AgeX specifically disclaims any obligation or intention to update or revise these forward-looking statements as a result of changed events or circumstances that occur after the date of this release, except as required by applicable law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191210005435/en/

Contacts

Media Contact for AgeX:Bill Douglass Gotham Communications, LLCbill@gothamcomm.com (646) 504-0890

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AgeX Therapeutics and Lineage Cell Therapeutics Announce Issuance of U.S. Patent for Method of Generating Induced Pluripotent Stem Cells - Yahoo...

IPS Cell Therapy Comments Off on AgeX Therapeutics and Lineage Cell Therapeutics Announce Issuance of U.S. Patent for Method of Generating Induced Pluripotent Stem Cells – Yahoo… | December 12th, 2019

Lineage Cell Therapeutics and AgeX Therapeutics have been awarded a United States Patent and Trademark Office patent for Methods Of Reprogramming Animal Somatic Cells.

The issuance of this patent highlights Lineages dominant position in the field of cell therapy, stated Brian M. Culley, CEO of Lineage. Our efforts to develop new treatments rely on well-characterized and NIH-approved human cell lines. These lines are not genetically manipulated, which avoids the safety concerns associated with genetic aberrations arising from the creation of iPS cells. We believe the Lineage cell lines provide the safest option for our current clinical-stage programs, particularly in immune-privileged anatomical sites such as the eye (OpRegen for the treatment of dry AMD) and spinal cord (OPC1, for the treatment of spinal cord injury). However, the vast intellectual property estate which underlies our cell therapy platform has never been limited to these particular cell lines. As one example, this newly-issued patent provides us with proprietary methods for producing induced pluripotent stem cells, or, as it was practiced by us prior to Yamanaka, Analytical Reprogramming Technology (ART). In certain settings, an ART/iPS approach might offer important advantages, such as for an autologous treatment or when the selection of preferential attributes from a series of iPS lines is desirable. Questions as to which stem cell technology is preferred ultimately will be answered by clinical safety and efficacy and likely will be indication-specific, so we believe it is in the best interest of our shareholders to generate patented technology which enables us to pursue programs in either or both formats which we believe will ensure the highest probability of success.

This patent broadly describes multiple techniques for reprogramming cells of the body back to the all-powerful stem cell state, said Dr Michael D West, CEO of AgeX and first inventor on the patent. Perhaps more significantly, it includes certain factors that address some of the difficulties currently encountered with iPS cells. It also reflects the foundational work our scientists have undertaken to apply reprogramming technology to age-reversal, specifically, induced Tissue Regeneration (iTR) which is currently a focus of AgeX product development.

Patent 10,501,723 covers induced pluripotent stem cells which includes methods to manufacture iPSs cells that are capable of becoming any cell within the body. This patent has an early priority date having been filed before the first scientific publication, and was assigned to Advanced Cell Technology of Marlborough, Massachusetts and licenced to Lineage as well as being sublicensed to Age X for defined fields of use.

Original post:
Patent Granted To Lineage & AgeX - Anti Aging News

IPS Cell Therapy Comments Off on Patent Granted To Lineage & AgeX – Anti Aging News | December 12th, 2019

DUBLIN--(BUSINESS WIRE)--The "Global Blood and Bone Marrow Cancer Treatment Market Size, Market Share, Application Analysis, Regional Outlook, Growth Trends, Key Players, Competitive Strategies and Forecasts, 2019 to 2027" report has been added to ResearchAndMarkets.com's offering.

The global blood and bone marrow cancer treatment market was valued at US$ 38.8 Bn in 2018 and is expected to reach US$ 74.9 Bn by 2027, expanding at a CAGR of 7.7% from 2019 to 2027.

Market Insights

Blood cancer begins in the bone marrow which is the integral source of stem cells which later are differentiated in different types of blood cells in the human body. Researchers at Bristol Myers Squibb Company have stated that approximately 1.85 million new cases of blood cancer will be diagnosed by 2040 throughout the globe.

Lymphoma is the largest indication segment for blood and bone marrow cancer treatment market. It is prevalent in 2 types Hodgkin lymphoma and Non-Hodgkin lymphoma throughout the globe. The chief variables responsible for its rising prevalence worldwide are increasing prescription of immunosuppressant drugs for treating chronic infections and genetic mutations. Leukemia occurs when the DNA of immature white blood cells gets damaged due to exposure to ionizing radiation, hazardous chemicals, smoking, etc. The prevalence rate of leukemia is highly variable across different ethnic groups with men to women ratio of 1.4.

Chemotherapy is reigning the therapy segment for blood and bone marrow cancer treatment market. The key parameter hold responsible for its increasing demand is the availability of its generic version at affordable cost, drastically reducing the healthcare burden on ailing patients. Oncologists prefer to use them in combination therapy either with radiotherapy or immunotherapy to treat patients showing resistance to first-line drug therapy. Immunotherapy will be the fastest-growing segment during the forecast period owing to its promising drug pipeline for the treatment of blood cancer.

North America representing a market share of 34.6% is dominating the regional segment for blood and bone marrow cancer treatment market. The chief contributing factor for its market supremacy is a growing incidence of blood cancer. As per the research citings of the Leukemia and Lymphoma Society (CDC) figures after every 3 minutes, 1 person in the U.S. is diagnosed with blood cancer. In 2019, approximately 176,200 people in the U.S. are diagnosed with blood cancer in the United States. Europe holds a market share of 30.8% owing to the supportive regulatory framework provided by the European Medical Agency for the development and sale of medication for the treatment of blood cancer. The Asia Pacific accounts for 18.4% market share on account of rising public health awareness related to blood cancer & its treatment and developing healthcare infrastructure.

Key Market Movements:

Key Topics Covered:

Chapter 1. Preface

1.1. Report Scope and Description

1.1.1. Purpose of the Report

1.1.2. Target Audience

1.1.3. USP and Key Offerings

1.2. Research Scope

1.3. Research Methodology

1.3.1. Phase I-Secondary Research

1.3.2. Phase II-Primary Research

1.3.3. Approach Adopted

1.3.4. Top-down Approach

1.3.5. Bottom-up Approach

1.3.6. Phase III-Expert Panel Review

1.3.7. Assumptions

1.4. Market Segmentation

Chapter 2. Executive Summary

2.1. Global Blood and Bone Marrow Cancer Treatment Market Portraiture

2.2. Global Blood and Bone Marrow Cancer Treatment Market, by Indication, 2018 (US$ Bn)

2.3. Global Blood and Bone Marrow Cancer Treatment Market, by Therapy, 2018 (US$ Bn)

2.4. Global Blood and Bone Marrow Cancer Treatment Market, by Geography, 2018 (US$ Bn)

Chapter 3. Blood and Bone Marrow Cancer Treatment Market: Dynamics and Future Outlook

3.1. Market Overview

3.2. Drivers

3.3. Challenges

3.4. Opportunities

3.5. Attractive Investment Proposition, by Geography, 2018

3.6. Competitive Analysis: Global Blood and Bone Marrow Cancer Treatment Market, by Key Players, 2018

Chapter 4. Global Blood and Bone Marrow Cancer Treatment Market, by Indication

4.1. Overview

4.2. Multiple Myeloma

4.3. Leukemia

4.4. Lymphoma

4.5. Others

Chapter 5. Global Blood and Bone Marrow Cancer Treatment Market, by Therapy

5.1. Chemotherapy

5.2. Immunotherapy

5.3. Stem Cell Transplant

5.4. Radiotherapy

5.5. Pipeline Analysis

5.5.1. Phase III Drug

5.5.1.1. Eltrombopag

5.5.1.2. Avatrombopag

5.5.1.3. Hetrombopag

5.5.1.4. Omidubicel

5.5.1.5. Fedratinib

5.5.1.6. ATIR101

5.5.1.7. Pegylated Proline Interferon Alpha-2b

5.5.2. Tabular Representation of Phase II and I Pipeline Drugs

Chapter 6. Global Blood and Bone Marrow Cancer Treatment Market, by Geography

6.1. Overview

6.2. North America Blood and Bone Marrow Cancer Treatment Market Analysis, 2017- 2027

6.3. Europe Blood and Bone Marrow Cancer Treatment Market Analysis, 2017 - 2027

6.4. Asia Pacific Blood and Bone Marrow Cancer Treatment Market Analysis, 2017 - 2027

6.5. Latin America Blood and Bone Marrow Cancer Treatment Market Analysis, 2017 - 2027

6.6. Middle East and Africa Blood and Bone Marrow Cancer Treatment Market Analysis, 2017 - 2027

Chapter 7. Company Profiles

7.1. AstraZeneca, Plc.

7.1.1. Business Description

7.1.2. Financial Information (Subject to data availability)

7.1.3. Product Portfolio

7.1.4. News Coverage

7.2. Celgene, Inc.

7.3. Bristol Myers Squibb & Company

7.4. Eli Lilly & Company

7.5. Johnson & Johnson Company

7.6. F.Hoffman La-Roche Ltd.

7.7. Merck & Co., Inc.

7.8. Novartis AG

7.9. Pfizer, Inc.

7.10. Varian Medical Systems, Inc.

For more information about this report visit https://www.researchandmarkets.com/r/pi0qoz

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Global Blood and Bone Marrow Cancer Treatment Market Trends & Analysis During the Forecast Period, 2019-2027 - ResearchAndMarkets.com - Business...

Bone Marrow Stem Cells Comments Off on Global Blood and Bone Marrow Cancer Treatment Market Trends & Analysis During the Forecast Period, 2019-2027 – ResearchAndMarkets.com – Business… | December 10th, 2019

In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering -- by editing her genome.

Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research -- gene therapy.

"I have hoped for a cure since I was about 11," the 34-year-old told AFP in an email. "Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency."

Over several weeks, Gray's blood was drawn so doctors could get to the cause of her illness -- stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 -- pronounced "Crisper" -- a new tool informally known as molecular "scissors."

The genetically edited cells were transfused back into Gray's veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured.

"This is one patient. This is early results. We need to see how it works out in other patients," said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville. "But these results are really exciting."

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified.

But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

"It's all developing very quickly," said French geneticist Emmanuelle Charpentier, one of Crispr's inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy.

Scientists practising the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not -- such as making normal red blood cells, in Victoria's case, or making tumor-killing super white blood cells for a cancer patient.

Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the U.S. and a blood disease in the European Union.

They join several other gene therapies -- bringing the total to eight -- approved in recent years to treat certain cancers and an inherited blindness.

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

"Twenty-five, 30 years, that's the time it had to take," he told AFP from Paris. "It took a generation for gene therapy to become a reality. Now, it's only going to go faster."

Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a "breakthrough period."

"We have hit an inflection point," said Carrie Wolinetz, NIH's associate director for science policy.

These therapies are exorbitantly expensive, however, costing up to $2 million -- meaning patients face grueling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion -- and fighting a general infection. "You cannot do this in a community hospital close to home," said her doctor.

However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers. They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who don't necessarily share the medical ethics of Western medicine.

Last year in China, scientist He Jiankui triggered an international scandal -- and his excommunication from the scientific community -- when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process.

"That technology is not safe," said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr "scissors" often cut next to the targeted gene, causing unexpected mutations.

"It's very easy to do if you don't care about the consequences," Musunuru added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species -- malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the U.S.

The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesn't believe in the more dystopian scenarios predicted for gene therapy, including American "biohackers" injecting themselves with Crispr technology bought online.

"Not everyone is a biologist or scientist," she said.

And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies' crops?

Charpentier thinks that technology generally tends to be used for the better.

"I'm a bacteriologist -- we've been talking about bioterrorism for years," she said. "Nothing has ever happened."

Link:
2019: the year gene therapy came of age - Japan Today

Bone Marrow Stem Cells Comments Off on 2019: the year gene therapy came of age – Japan Today | December 10th, 2019

DURHAM, N.C., Dec. 10, 2019 (GLOBE NEWSWIRE) -- Chimerix, Inc. (Nasdaq: CMRX), a biopharmaceutical company focused on accelerating the development of medicines to treat cancer and other serious diseases, today announced that data relating to its dociparstat sodium (DSTAT) program, formerly known as CX-01, were presented at the 61st American Society of Hematology Annual Meeting, in Orlando, FL.

The poster, titledUpdated Study Results for CX-01, an Inhibitor of CXCL12/CXCR4, With Azacitidine for the Treatment of Hypomethylating Agent Refractory AML and MDS, was presented byEric Huselton, M.D., Assistant Professor of Medicine at the University of Rochester on December 9, 2019.

As reported in the published study abstract, 20 patients with refractory myelodysplastic syndrome (MDS) (n = 9) or refractory acute myeloid leukemia (AML) (n = 11) were enrolled of which 15 were considered evaluable for response with a bone marrow biopsy after cycle 2. Patients received a 7-day continuous infusion of DSTAT (CX-01) at a dose of 0.25 mg/kg/hour, and azacitidine 75 mg/m2 daily days 1-7, in 28-day cycles. The primary objective of this trial was to assess the overall response rate. Half of the patients had high risk cytogenetic abnormalities and 3 had TP53 mutations. Patients had a median of 2 prior lines of therapy (range 1-3) with median of 6 prior cycles of hypomethylating agent (HMA) therapy (range 4-20). Only 4 patients had a confirmed response to prior HMA therapy.

The 15 evaluable patients received a median of 3 cycles of CX-01 and azacitidine (range 2-9). Of 15 evaluable patients, there was 1 CR (complete remission) and 3 bone marrow CRs (mCR, with incomplete peripheral blood count recovery), 9 stable disease, and 2 progressive disease for an overall response rate of 27%. Of the 3 patients with a mCR after cycle 2, two had hematologic improvement of their neutrophil and platelet counts, respectively, by the end of cycle 4. A patient with stable disease also had hematologic improvement in platelets.

The median overall survival of evaluable patients was 221 days. The median overall survival was not significantly different between AML patients at 221 days and MDS patients at 248 days.

Following a minimum of 4 cycles of prior HMA therapy, one would not expect to observe response to subsequent HMA therapy, said Dr. Huselton. These results demonstrate DSTATs potential to improve HMA therapy outcomes in terms of both response and overall survival.

"DSTATs mechanism of action is intended to enhance patient benefit when combined with an active agent, so to observe these results in HMA-refractory patients is promising. In addition to our planned Phase 3 pivotal trial in newly diagnosed AML, this study highlights the potential to develop DSTAT to enhance the benefit of multiple therapies such as azacitidine, in AML and MDS in both front-line and recurrent settings," said Mike Sherman, Chief Executive Officer of Chimerix.

AboutChimerix

Chimerixis a development-stage biopharmaceutical company dedicated to accelerating the advancement of innovative medicines that make a meaningful impact in the lives of patients living with cancer and other serious diseases. The two clinical-stage development programs are dociparstat sodium (DSTAT) and brincidofovir (BCV).

Dociparstat sodium is a potential first-in-class glycosaminoglycan biologic derived from porcine heparin that has low anticoagulant activity but retains the ability to inhibit activities of several key proteins implicated in the retention and viability of AML blasts and leukemic stem cells in the bone marrow during chemotherapy (e.g., CXCL12, selectins, HMGB1). Mobilization of AML blasts and leukemic stem cells from the bone marrow has been associated with enhanced chemosensitivity and may be a primary mechanism accounting for the observed increases in EFS and OS in Phase 2 with DSTAT versus placebo. Randomized Phase 2 data suggest that DSTAT may also accelerate platelet recovery post-chemotherapy via inhibition of platelet factor 4, a negative regulator of platelet production that impairs platelet recovery following chemotherapy. BCV is a lipid conjugate DNA polymerase inhibitor in development as a medical countermeasure for smallpox.For further information, please visit the Chimerix website,www.chimerix.com

CONTACT:

Investor Relations:Michelle LaSpaluto919-972-7115ir@chimerix.com

Will OConnorStern Investor Relations212-362-1200will@sternir.com

Link:
Chimerix Presents Updated Results from Phase 2 Clinical Trial of DSTAT in Refractory Myelodysplastic Syndrome and Acute Myeloid Leukemia at American...

Bone Marrow Stem Cells Comments Off on Chimerix Presents Updated Results from Phase 2 Clinical Trial of DSTAT in Refractory Myelodysplastic Syndrome and Acute Myeloid Leukemia at American… | December 10th, 2019

The global bone marrow aspirate concentrates market was valued around US$ 130.0 Mn in 2016 is anticipated to register a stable CAGR of over 5.0% during forecast period of 2017 to 2025, according to a new report published by Transparency Market Research (TMR) titled Bone Marrow Aspirate Concentrates Market Global Industry Analysis, Size, Share, Growth, Trends, and Forecast, 20172025.

Growth of the global bone marrow aspirate concentrates market is driven by increased prevalence of and incidences of orthopedic diseases, and sports injuries, along with high growth of the cosmetic surgery industry and increasing applications of the BMAC products in the cosmetic and orthopedic surgeries. The bone marrow aspirate concentrates market in Asia Pacific is expanding with a high potential to grow registering a CAGR above 6.0% on the backdrop of unmet clinical needs, rising geriatric population, large patient pool, favorable government regulations, development in health care sector, and increased focus on research and developmental activities.

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Increase in incidences of Osteoarthritis on the backdrop of rising geriatric population to drive market growth

According to a collaborative survey conducted by the United Nations and the World Health Organization, 1.2 billion people in China are suffering from OA, of which more than 55% are aged 60 years or above. On the backdrop of such a huge patient base, there has been several developments in the field orthopedic surgery. Bone marrow-derived stem cell treatment is considered a promising and advanced therapy. It reduces the injury healing time in orthopedic diseases to five to six weeks from four to six months in case of surgery.

Reduction in the healing time is a factor likely to propel the Bone Marrow Aspirate Concentrates market during the forecast period. However, pain associated with the treatment, lack of product approval, and preference for alternative treatments are negatively affecting the market growth. Moreover, high investments in R&D and clinical trials, slow approval processes entailing sunken costs, and marginal returns on investment (RoI) for stakeholders are primary concerns faced by manufacturer further hampering growth of the market.

Rise in the Number of BMAC Assisted Procedures to Boost Growth of Bone Marrow Aspirate Concentrates Accessories Segment

The product type segment is fragmented into bone marrow aspirate concentrates systems and bone marrow aspirate concentrates accessories. The bone marrow aspirate concentrates accessories segment is anticipated to carry major share of the market on the backdrop of rise in number of BMAC assisted procedures. Cell therapies have been used extensively over the past decade for a variety of medical applications to restore cellular function and enhance quality of life. Owing to the differentiation property, stem cells are being used for repair and regeneration of bone. Moreover, increase in awareness about hygiene and risk of cross-contamination in developing countries such as Brazil, China and India are expected to increase the use of single-use Jamshidi needles for bone marrow stem cell procedures. This is likely to fuel the growth of the accessories segment in the near future.

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Orthopedic Surgery Application to Dominate the Global Bone Marrow Aspirate Concentrates Market

The application segment of global bone marrow aspirate concentrates market is divided into orthopedic surgery, wound healing, chronic pain, peripheral vascular disease, dermatology, and others applications. Of which, orthopedic surgery segment is anticipated to dominate the market owing to rising geriatric population, and surge in incidences of osteoarthritis around the globe.

The dermatology segment is anticipated to expand at the highest CAGR of over 6.0% during forecast period of 2017 to 2025 owing to current boom in the industry, increase in disposable income, and technological advancements in the market. The utilization of the regenerative ability of fibroblasts and keratinocytes from human skin has formed new ways to develop cell-based therapies for patients. Moreover, capacity of bone marrow derived extra-cutaneous cells is being researched for its plasticity in regenerating skin; it is likely to lead to the future growth of cell therapies in dermatology.

Rise in Healthcare Expenditure to Fuel Growth of Hospitals & Clinics End-user Segment

In terms of end-users, market is divided into hospitals & clinics, pharmaceutical & biotechnology companies, Contract Research Organizations (CROs) & Contract Manufacturing Organizations (CMOs), and academic & research institutes. The hospitals & clinics segment dominated the bone marrow aspirate concentrates market in 2016. The trend is expected to continue during the forecast period. The hospitals & clinics segment is likely to be followed by the biotechnology & biopharmaceutical companies segment in terms of market share during the forecast period. The segment is anticipated to hold more than 8.0% of market share in 2016. Growth of the segment is attributed to increasing number of biotechnology companies and rising partnerships among the market players to expand global presence.

About Us

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

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

ContactMr. Rohit BhiseyTransparency Market ResearchState Tower,90 State Street,Suite 700,Albany NY 12207United StatesTel: +1-518-618-1030USA Canada Toll Free: 866-552-3453Email: [emailprotected]Website: http://www.transparencymarketresearch.comBlog: https://theglobalhealthnews.com/

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Bone Marrow Aspirate Concentrates Market to Expand at an Outstanding CAGR of 5% from 2017 to 2025 - VaporBlash

Bone Marrow Stem Cells Comments Off on Bone Marrow Aspirate Concentrates Market to Expand at an Outstanding CAGR of 5% from 2017 to 2025 – VaporBlash | December 10th, 2019

Researchers in China are laying claim to the first ever pig-monkey chimeras to be born in what they hope will be a breakthrough for biomedicine, not just fuel for your nightmares.

The worlds first monkey-pig hybrids have been born in a Chinese lab amid scientific attempts to grow human organs inside animals. The incredible experiment saw two chimera piglets born with DNA from both pigs and cynomolgus monkeys. However, the experiment was short-lived as the two piglets both died within a week.

According to the researchers, the baby piglets had genetic material from cynomolgus monkeys in their heart, liver, spleen, lung and skin.

However, the success rate was significantly low as only 10 piglets were born from more than 4,000 implanted in sows.

From those, only two were chimeras in a result that has been deemed discouraging by other leading stem cell scientists.

Lead researcher Tang Hai at the State Key Laboratory of Stem Cell and Reproductive Biology in Beijing said: This is the first report of full-term pig-monkey chimeras.

Ms Hai confirmed that it was unclear why the piglets died, but the non-chimeric pigs died as well.

The Chinese research team suspect that the deaths could have been due to the IVF process instead than the chimerism

According to research study, stem cells from macaque monkeys were grown in a lab and then injected into pig embryos five days after fertilisation.

Ms Hai said that the next step in their research was to create healthy animals with a higher proportion of monkey cells before creating pigs in which one organ is composed almost entirely of primate cells.

The ultimate research goal is to grow human organs inside live animals as a way to resolve the crisis of organ transplantation.

University of California stem cell biologist Paul Knoepfler said: Given the extremely low chimeric efficiency and the deaths of all the animals, I see this as fairly discouraging.

Reaction to the study has prompted many social media users to question if this is a step too far.

Read more here:
Researchers Manage to Create Pig-Monkey Chimeras for the First Time - Tdnews

Skin Stem Cells Comments Off on Researchers Manage to Create Pig-Monkey Chimeras for the First Time – Tdnews | December 10th, 2019

Theglobal market for cell harvestingshould grow from $885 million in 2018 to reach $1.5 billion by 2023 at a compound annual growth rate (CAGR) of 11.3% for the period of 2018-2023.

Report Scope:

The scope of the report encompasses the major types of cell harvesting that have been used and the cell harvesting technologies that are being developed by industry, government agencies and nonprofits. It analyzes current market status, examines drivers on future markets and presents forecasts of growth over the next five years.

The report provides a summary of the market, including a market snapshot and profiles of key players in the cell harvesting market. It provides an exhaustive segmentation analysis of the market with in-depth information about each segment. The overview section of the report provides a description of market trends and market dynamics, including drivers, restraints and opportunities. it provides information about market developments and future trends that can be useful for organizations, including wholesalers and exporters. It provides market positionings of key players using yardsticks of revenue, product portfolio, and recent activities. It further includes strategies adopted by emerging market players with strategic recommendations for new market entrants. Readers will also find historical and current market sizes and a discussion of the markets future potential. The report will help market players and new entrants make informed decisions about the production and exports of goods and services.

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Report Includes:

41 data tables and 22 additional tables Description of segments and dynamics of the cell harvesting market Analyses of global market trends with data from 2017, 2018, and projections of compound annual growth rates (CAGRs) through 2023 Characterization and quantification of market potential for cell harvesting by type of harvesting, procedure, end user, component/equipment and region A brief study and intact information about the market development, and future trends that can be useful for the organizations involved in Elaboration on the influence of government regulations, current technology, and the economic factors that will shape the future marketplace Key patents analysis and new product developments in cell harvesting market Detailed profiles of major companies of the industry, including Becton, Dickinson and Co., Corning, Inc., Fluidigm Corp., General Electric Co., Perkinelmer, Inc., and Thermo Fisher Scientific, Inc.

Summary

Stem cells are unspecialized cells that have the ability to divide indefinitely and produce specialized cells. The appropriate physiological and experimental conditions provided to the unspecialized cells give rise to certain specialized cells, including nerve cells, heart muscle cells and blood cells. Stem cells can divide and renew themselves over long periods of time. These cells are extensively found in multicellular organisms, wherein mammals, there are two types of stem cells embryonic stem cells and adult stemcells. Embryonic stem cells are derived from a human embryo four or five days old that is in the blastocyst phase of development. Adult stem cells grow after the development of the embryo and are found in tissues such as bone marrow, brain, blood vessels, blood, skin, skeletal muscles and liver. Stemcell culture is the process of harvesting the exosomes and molecules released by the stem cells for the development of therapeuticsfor chronic diseases such as cancer and diabetes. The process is widely used in biomedical applications such as therapy, diagnosis and biological drug production. The global cell harvesting market is likely to witness a growth rate of REDACTED during the forecast period of 2018-2023.The value of global cell harvesting market was REDACTED in 2017 and is projected to reach REDACTED by 2023. Market growth is attributed to factors such as increasing R&D spending in cell-based research,the introduction of 3D cell culture technology, increasing government funding, and the growing prevalence of chronic diseases such as cancer and diabetes.

The growing incidence and prevalence of cancer is seen as one of the major factors contributing to the growth of the global cell harvesting market. According to the World Health Organization (WHO), cancer is the second-leading cause of mortality globally and was responsible for an estimated 9.6 million deaths in 2018. Therefore, there is an increasing need for effective cancer treatment solutions globally. Cell harvesting is the preferred method used in cancer cell-related studies including cancer cell databases (cancer cell lines), and other analyses and drug discovery in a microenvironment. The rising prevalence of such chronic diseases has led governments to provide R&D funding to research institutes and biotechnology companies to develop advanced therapeutics. Various 3D cell culture technologies have been developed by researchers and biotechnology companies such as Lonza Group and Thermo Fischer Scientific for research applications such as cancer drug discovery. The application of cell culture in cancer research is leading to more predictive models for research, drug discovery and regenerative medicine applications.

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Platelet-rich plasma (PRP) therapy, a new biotechnology solution that has a heightened interest among researchers in tissue engineering and cell-based therapies, has various applications in the treatment of tissue healing in tendinopathy, osteoarthritis and muscle injury. It has been conventionally employed in orthopedics, maxillofacial surgery, periodontal therapy and sports medicines. PRP therapy can be used in the treatment of fat grafting, acne scars, and hair regrowth.

Major factors driving market growth include increasing healthcare costs and the high rate of adoption for modern medicines in emerging economies such as China and India. It has been estimated that India will witness a CAGR of REDACTED in the cell harvesting market during the forecast period. The active participation of foreign pharmaceutical companies has tapped the Indian healthcare sector with a series of partnerships and mergers and acquisitions, which in turn is positively impacting the growth of the market in this region. Consistent development and clinical trials for stem cell therapies, plus contribution from the government and private sectors through investments and cohesive reimbursement policies in the development of cancer biomarkers, is further fueling market growth. InSweden, a research team at Lund University has developed a device to collect fluid and harvest stem mesenchymal stem cells (MSCs). The device is developed with 3D-printed bio-inert plastics which, when used by doctors, can result in the safe extraction of fluids (medical waste) from the patients body. The liquid is then passed through a gauze filter for purifying thoroughly and MSCs are separated from the fluid by centrifugation and are grown in culture.

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Cell Harvesting Market a compound annual growth rate (CAGR) of 11.3% for the period of 2018-2023 - Crypto News Byte

Skin Stem Cells Comments Off on Cell Harvesting Market a compound annual growth rate (CAGR) of 11.3% for the period of 2018-2023 – Crypto News Byte | December 10th, 2019

WALTHAM, Mass., Dec. 10, 2019 /PRNewswire/ --Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, today announced that it plans to commence a Phase 2 expansion cohort based on encouraging clinical activity and a well-tolerated safety profile observed to date in the ongoing Phase 1 dose escalation trial of SNDX-6352 in patients with chronic graft versus host disease (cGVHD). SNDX-6352 is the Company's anti-CSF-1R monoclonal antibody.

The ongoing Phase 1, open-label, modified 3+3 dose escalation trial is designed to evaluate the safety and preliminary efficacy of SNDX-6352 in up to 30 patients with cGVHD who have received at least two prior lines of therapy. As of a November 25, 2019 data cutoff date, a total of five patients, all of whom received prior treatment with ibrutinib, steroids, and a calcineurin inhibitor, have been enrolled across three dose cohorts: one patient was treated at 0.15 mg/kg every two weeks (Q2W, Cohort 1), one is receiving a dose of 0.5 mg/kg Q2W (Cohort 2), and three patients are receiving 1.0 mg/kg Q2W (Cohort 3).

Responses have been observed in all evaluable patients as of the data cutoff date, with no dose limiting toxicities (DLTs) reported. Among the three patients dosed in Cohort 3 (1.0 mg/kg Q2W), one patient recently cleared the DLT period and has not yet been evaluated for efficacy, two patients experienced a partial response, and all three patients remain on therapy. The patient in Cohort 2 experienced a partial response and is currently in their ninth month of treatment with SNDX-6352 following prior treatment with ibrutinib and both Jakafi (ruxolitinib) and KD025, two agents currently being investigated for the treatment of cGVHD. The first patient (Cohort 1) achieved a partial response but discontinued in their third cycle due to elevated LFTs attributed to progression in their liver cGVHD. Cohort 4, which will explore a 3.0 mg/kg Q2W dose, is now open for enrollment.

"The initial results from our Phase 1 trial underscore the potential of SNDX-6352 to serve as an effective therapy for patients with cGVHD who are lacking alternative options," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "We had not anticipated commenting on data from this initial trial until the second half of 2020, so it is quite encouraging to see the early signs of activity in patients with this difficult to treat disease. Based on these results, we have decided to advance into a Phase 2 expansion cohort to evaluate additional patients at the 1.0 mg/kg dose while we continue the dose escalation to 3.0 mg/kg. We continue to expect to present the Phase 1 trial results in the second half of 2020.

"Published preclinical data have demonstrated that CSF-1R blockade can prevent and treat disease in animal models of cGVHD1," said Peter Ordentlich, Ph.D., Chief Scientific Officer and Co-founder of Syndax."The initial data from our trial provide the first clinical evidence that targeting CSF-1R dependent macrophages may benefit patients with cGVHD."

To date, SNDX-6352 has been safe and well-tolerated, with no DLTs observed. Dose escalation is ongoing in the Phase 1 portion of the trial. The Phase 2 expansion cohort is expected to enroll up to 22 patients to further characterize the safety and efficacy at an initial dosing schedule of 1.0 mg/kg of SNDX-6352 administered every two weeks.

About Chronic Graft Versus Host Disease

Chronic graft versus host disease (cGVHD), an immune response of the donor-derived hematopoietic cells against recipient tissues, is a serious, potentially life-threatening complication of allogeneic hematopoietic stem cell transplantation (HSCT) which can last for years. cGVHD is estimated to develop in approximately 40% of transplant recipients, and affect approximately 14,000 patients in the US. 2-4 cGVHD typically manifests across multiple organ systems, with the skin and mucosa being commonly involved, and is characterized by the development of fibrotic tissue.

About SNDX-6352

SNDX-6352 is an investigational monoclonal antibody that targets colony stimulating factor-1 receptor, or CSF-1R, a cell surface protein thought to control the survival and function of monocytes and macrophages. In pre-clinical models, inhibition of signaling through the CSF-1 receptor has been shown to reduce the number of disease-mediating macrophages and the development of cutaneous and pulmonary chronic graft versus host disease (cGVHD), as well as to lead to the depletion of cells known as Tumor Associated Macrophages, or TAMS. SNDX-6352 is currently being evaluated in a Phase 1 multiple ascending dose clinical trial in cGVHD, and a Phase 1 multiple ascending dose clinical trial as monotherapy and in combination with Infinzi (durvalumab) in solid tumors. SNDX-6352 has the potential to treat a variety of solid tumor and immune-related diseases.

About Syndax Pharmaceuticals, Inc.

Syndax Pharmaceuticalsis a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies. The Company's lead product candidate, entinostat, a once-weekly, oral, small molecule, class I HDAC inhibitor, is being tested in a Phase 3 combination trial with exemestane for treatment of advanced HR+, HER2- breast cancer and has been evaluated in combination with several approved PD-1/PD-(L)1 antagonists. The Company's pipeline also includes SNDX-6352, a monoclonal antibody that blocks the colony stimulating factor 1 (CSF-1) receptor, and SNDX-5613, a highly selective inhibitor of the MeninMLL binding interaction. For more information, please visitwww.syndax.comor follow the Company on TwitterandLinkedIn.

Syndax's Cautionary Note on Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "may," "will," "expect," "plan," "anticipate," "estimate," "intend," "believe" and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are based on Syndax's expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties. Actual results may differ materially from these forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to, statements about the progress, timing, clinical development and scope of clinical trials and the reporting of clinical data for Syndax's product candidates, and the potential use of our product candidates to treat various cancer indications. Many factors may cause differences between current expectations and actual results including unexpected safety or efficacy data observed during preclinical or clinical trials, clinical trial site activation or enrollment rates that are lower than expected, changes in expected or existing competition, changes in the regulatory environment, failure of Syndax's collaborators to support or advance collaborations or product candidates and unexpected litigation or other disputes. Other factors that may cause Syndax's actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Syndax's filings with the U.S. Securities and Exchange Commission, including the "Risk Factors" sections contained therein. Except as required by law, Syndax assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.

References

1. Alexander, KA. et al. J Clin Invest. 2014;124(10):42664280.

2.Kantar GVHD Expert Interviews N=8 interviews

3. SmartAnalyst 2017 SmartImmunology Insights chronic GVHD report.

4. Bachier, CR. et al. ASH annual meeting 2019; abstract #2109 Epidemiology and Real-World Treatment of Chronic Graft-Versus-Host Disease Post Allogeneic Hematopoietic Cell Transplantation: A U.S. Claims Analysis.

Syndax Contacts

Investor ContactMelissa ForstArgot Partnersmelissa@argotpartners.com212.600.1902

Media ContactCraig HeitGCI Healthcraig.Heit@gcihealth.com347.451.4733

SNDX-G

SOURCE Syndax Pharmaceuticals, Inc.

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Syndax Pharmaceuticals Announces Plans to Commence Phase 2 Expansion Cohort of SNDX-6352 for the Treatment of Chronic Graft Versus Host Disease -...

Skin Stem Cells Comments Off on Syndax Pharmaceuticals Announces Plans to Commence Phase 2 Expansion Cohort of SNDX-6352 for the Treatment of Chronic Graft Versus Host Disease -… | December 10th, 2019

Brian Bowman, who presides over a city increasingly defined by rising crime, chaos, and a spiralling meth epidemic, apparently thought it would be smart to tweet out a photo of him looking happy to meet Chinas ambassador to Canada, Cong Peiwu.

Pleased to visit with Chinese Ambassador to Canada, Cong Peiwu. Thank you for a productive discussion about our Sister City Chengdu, trade, and Winnipegs goal of becoming a leader in the protection and promotion of human rights.

Pleased to visit with Chinese Ambassador to Canada, Cong Peiwu. Thank you for a productive discussion about our Sister City Chengdu, trade, and Winnipeg's goal of becoming a leader in the protection and promotion of human rights. pic.twitter.com/xe4hqpHrUI Mayor Brian Bowman (@Mayor_Bowman) December 5, 2019

You can already see the problems here.

First, if youre going to talk about the protection and promotion of human rights, you probably shouldnt be meeting with an ambassador from a country that has a horrific human rights record like China.

China has at least one million innocent Muslim people in concentration camps, is committing a cultural genocide, is breaking their One Country, Two Systems pledge in Hong Kong, and is threatening their neighbours.

Also, to thank Chinas ambassador after that same ambassador threatened Canada, and while China continues to jail the two Michaels is an absolute disgrace.

Unsurprisingly, Bowman is being ripped following the Tweet.

Below, you can see how badly Bowman got ratioed, with just 26 retweets and 482 comments.

Here is some of what people said:

How much money did communists pay you to make you praise their human rights disaster?! Swear you dont know Tiananmen Square Massacre, live organ harvesting, Uyghurs in concentration camps, HK protests being brutally cracked down! Where is your conscience sir?!

How much money did communists pay you to make you praise their human rights disaster?! Swear you dont know Tiananmen Square Massacre, live organ harvesting, Uyghurs in concentration camps, HK protests being brutally cracked down! Where is your conscience sir?! pic.twitter.com/RvowTsYR0C

Whose going to tell him they have two of our people in torture like conditions?

#TheMichaels will appreciate the irony in the Human Rights part

#TheMichaels will appreciate the irony in the Human Rights part Mr. Omega Man -1965 (@Keepershiny) December 7, 2019

Human rights and industrial scale murder for organs are not compatible

Its time to drop Chengdu as a sister city. And certainly time to revoke any future invitations to ANYONE who denies the existence of these concentration camps. Oh, and youve officially lost my vote.

Its time to drop Chengdu as a sister city. And certainly time to revoke any future invitations to ANYONE who denies the existence of these concentration camps. Oh, and youve officially lost my vote. Long time listener, first time caller (@EdVorst) December 7, 2019

Bowman was also ripped by some Canadian leaders, including Conservative MP James Bezan, and former Canadian Ambassador to China David Mulroney:

Statements like this play into the PRCs agenda of obfuscation, and actually undermine efforts to protect human rights. They reflect the false assumption that diplomacy cannot cope with plain-speaking and hard truths

Statements like this play into the PRC's agenda of obfuscation, and actually undermine efforts to protect human rights. They reflect the false assumption that diplomacy cannot cope with plain speaking and hard truths David Mulroney (@David_Mulroney) December 7, 2019

Too bad @Mayor_Bowman was played. The only discussions with Chinese officials should be about the release of Michael Kovrig & Michael Spavor, removing Chinese sanctions on our agriculture products, & respecting human rights in China. Until then, everything else is off the table.

Too bad @Mayor_Bowman was played. The only discussions with Chinese officials should be about the release of Michael Kovrig & Michael Spavor, removing Chinese sanctions on our agriculture products, & respecting human rights in China. Until then, everything else is off the table. https://t.co/v0H999derC James Bezan (@jamesbezan) December 8, 2019

Bowman has made a fool of himself, and sent a message that will encourage China to treat Canada even worse, as they once again see that the only response from Canadas political elites is pathetic weakness.

If Bowman had any integrity he would listen to Yaakov Pollak and resign.

Read the original:
Canadians are filing for multiple bankruptcies and it's costing everyone - The Post Millennial

Spinal Cord Stem Cells Comments Off on Canadians are filing for multiple bankruptcies and it’s costing everyone – The Post Millennial | December 10th, 2019

The issuance of this patent highlights Lineages dominant position in the field of cell therapy, stated Brian M. Culley, CEO of Lineage. Our efforts to develop new treatments rely on well-characterized and NIH-approved human cell lines. These lines are not genetically manipulated, which avoids the safety concerns associated with genetic aberrations arising from the creation of iPS cells. We believe the Lineage cell lines provide the safest option for our current clinical-stage programs, particularly in immune-privileged anatomical sites such as the eye (OpRegen for the treatment of dry AMD) and spinal cord (OPC1, for the treatment of spinal cord injury). However, the vast intellectual property estate which underlies our cell therapy platform has never been limited to these particular cell lines. As one example, this newly-issued patent provides us with proprietary methods for producing induced pluripotent stem cells, or, as it was practiced by us prior to Yamanaka, Analytical Reprogramming Technology (ART). In certain settings, an ART/iPS approach might offer important advantages, such as for an autologous treatment or when the selection of preferential attributes from a series of iPS lines is desirable. Questions as to which stem cell technology is preferred ultimately will be answered by clinical safety and efficacy and likely will be indication-specific, so we believe it is in the best interest of our shareholders to generate patented technology which enables us to pursue programs in either or both formats which we believe will ensure the highest probability of success.

This patent broadly describes multiple techniques for reprogramming cells of the body back to the all-powerful stem cell state, said Dr. Michael D. West, CEO of AgeX and first inventor on the patent. Perhaps more significantly, it includes certain factors that address some of the difficulties currently encountered with iPS cells. It also reflects the foundational work our scientists have undertaken to apply reprogramming technology to age-reversal, specifically, induced Tissue Regeneration (iTR) which is currently a focus of AgeX product development.

Induced Pluripotent Stem Cells (iPS) are typically derived from adult skin or blood cells which have been reprogrammed or induced to retrace their developmental age and regain the potential to form all of the young cell and tissue types of the body. In 2010 inventors of the -723 patent issued today demonstrated that this reversal of developmental aging even extended to the telomere clock of cell aging. This reprogramming technology provides an alternate source of starting material for the manufacture of potentially any type of human cell needed for therapeutic purposes. Because iPSCs can be derived directly from adult tissues, they can be used to generate pluripotent cells from patients with known genetic abnormalities for drug discovery or as an alternative source of cell types for regenerative therapies.

U.S. Patent No. 10,501,723, entitled Methods of Reprogramming Animal Somatic Cells was assigned to Advanced Cell Technology of Marlborough, Massachusetts (now Astellas Institute for Regenerative Medicine) and licensed to Lineage and sublicensed to AgeX Therapeutics for defined fields of use. Inventors of the patent include Michael D. West, CEO of AgeX and previous CEO of Advanced Cell Technology, Karen B. Chapman, Ph.D., and Roy Geoffrey Sargent, Ph.D.

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineages programs are based on its proprietary cell-based therapy platform and associated development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally-differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed either to replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineages clinical assets include (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase I/IIa development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase I/IIa development for the treatment of acute spinal cord injuries; and (iii) VAC2, an allogeneic cancer immunotherapy of antigen-presenting dendritic cells currently in Phase I development for the treatment of non-small cell lung cancer. Lineage is also evaluating potential partnership opportunities for Renevia, a facial aesthetics product that was recently granted a Conformit Europenne (CE) Mark. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

About AgeX Therapeutics

AgeX Therapeutics, Inc. (NYSE American: AGE) is focused on developing and commercializing innovative therapeutics for human aging. Its PureStem and UniverCyte manufacturing and immunotolerance technologies are designed to work together to generate highly-defined, universal, allogeneic, off-the-shelf pluripotent stem cell-derived young cells of any type for application in a variety of diseases with a high unmet medical need. AgeX has two preclinical cell therapy programs: AGEX-VASC1 (vascular progenitor cells) for tissue ischemia and AGEX-BAT1 (brown fat cells) for Type II diabetes. AgeXs revolutionary longevity platform induced Tissue Regeneration (iTR) aims to unlock cellular immortality and regenerative capacity to reverse age-related changes within tissues. AGEX-iTR1547 is an iTR-based formulation in preclinical development. HyStem is AgeXs delivery technology to stably engraft PureStem cell therapies in the body. AgeX is developing its core product pipeline for use in the clinic to extend human healthspan and is seeking opportunities to establish licensing and collaboration agreements around its broad IP estate and proprietary technology platforms. For more information, please visit http://www.agexinc.com or connect with the company on Twitter, LinkedIn, Facebook, and YouTube.

Forward-Looking Statements

Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements, in some cases, can be identified by terms such as believe, may, will, estimate, continue, anticipate, design, intend, expect, could, plan, potential, predict, seek, should, would, contemplate, project, target, tend to, or the negative version of these words and similar expressions. Such statements include, but are not limited to, Lineages exploration of alternative cell therapy platforms. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineages actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including risks and uncertainties inherent in Lineages business and other risks in Lineages filings with the Securities and Exchange Commission (the SEC). Lineages forward-looking statements are based upon its current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Further information regarding these and other risks is included under the heading Risk Factors in Lineages periodic reports with the SEC, including Lineages Annual Report on Form 10-K filed with the SEC on March 14, 2019 and its other reports, which are available from the SECs website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Lineage undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

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Lineage Cell Therapeutics and AgeX Therapeutics Announce Issuance of US Patent for Method of Generating Induced Pluripotent Stem Cells - BioSpace

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