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Stem Cell Therapy May Improve Heart Health In New Ways – TheHealthMania

By daniellenierenberg

Recently, a new study that appears in the journal Nature, focuses on stem cell therapy and shows unexpected ways in which it may be helpful in recovering the health of the heart. Stem cell therapy has become popular in the past few years due to its benefits for a big number of health conditions.

Currently, there is major ongoing research on stem cells since they are responsible for the regeneration of new cells and may play a fundamental role in understanding the development of a variety of different diseases as well as their potential treatments.

Some of the recent discoveries of medical science include using stem cells as regenerative medicine as they can be turned into particular types of cells that may be able to replace tissues damaged as a result of health issues and thereby control the disease.

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The therapy can be specifically useful for people with conditions such as type 1 diabetes, spinal cord injuries, Alzheimers disease, Parkinsons disease, stroke, cancer, burns, amyotrophic lateral sclerosis, heart disease, and osteoarthritis.

At the moment, the most successful procedure that involves stem cell therapy is performing a bone marrow transplant. This surgical operation replaces the cells which have been damaged during chemotherapy by programmed stem cells. People are usually able to maintain and live a normal life after recovery from the surgery.

Furthermore, stem cell usage in clinical trials designed for testing the effectiveness, safety, and potential negative impact of new drugs. To do so, the stem cells can be programmed into becoming the type of cells that the drug aims to target.

The new study, which was led by Jeffery Molkentin who is a professor of the Howard Hughes Medical Institute (HHMI) and the director of Molecular Cardiovascular Microbiology a Cincinnati Childrens Hospital Medical Center, takes data from a study from the same journal, Nature, from the years 2014 which was conducted by the same medical team.

In the new paper, the team with Molkentin as the principal investigator found some unexpected results. There were two types of stem cells in the clinical trial cardiac progenitor cells and bone marrow mononuclear cells.

The main objective of the new trial was to re-evaluate the results of the 2014 study, which showed that injecting c-kit positive heart stem in the heart does not help in the regeneration of cardiomyocytes, to see how the cell therapy can be made to be effective.

It was instead discovered that injecting an inert chemical called zymosan, which is designed particularly for inducing an innate immune response, or dead stem cells can also be beneficial for the recovery of heart as they may speed up the healing procedure.

Injecting either dead stem cells or zymosan led to a reduction in the development of cellular matrix connective tissue in the areas which had been damaged in the heart. In addition, the mechanical properties of the targeted scar also improved.

Another important finding was that chemical substances such as zymosan are required to be injected directly into the heart for optimum results. In previous clinical trials, direct injections were avoided for safety reasons.

Molkentin and the team state that follow-up studies and trials on this new discovery are imminent as they may be important for developing therapies in the future.

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South Carolina toddler survives rare cancer and the risky procedure used to treat it – USA TODAY

By daniellenierenberg

Hailie Hyman holds her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

GREENVILLE, S.C.Hailie and Treylin Hyman saw the bruising on their baby girls leg as a sign that the active 1-year-old was learning to walk.

But as a blood test would later reveal, little Maci was actually suffering from an extremely rare blood cancer that threatened her life without a risky treatment - atreatmentalmost as dangerous as the disease.

In the beginning, it was very scary, Hailie Hyman told The Greenville News.

I couldnt think of anything but the bad things, she confessed. It was all about the statistics. And the statistics arent good.

Terrifying months followed the diagnosis, punctuated by one critical complication after another, leaving the Boiling Springs couple to wonder if Maci would survive.

Somehow, though, the blue-eyed toddler pulled through.And now her family is looking forward to a special Thanksgiving with much to be grateful for.

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The Hymans journey began last February atMacis 1-year-old well-child checkup.

We had no idea anything was wrong, her mom said.But they did a routine (blood test) and a couple of hours later, we got a call saying her platelets were very low.

The Hymans were referred to a hematologist who found other abnormalities in Macis blood and scheduled a bone marrow biopsy to investigate further.

Hailie Hyman holds her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

During the procedure, the child suffered an aneurysm in an artery and went into cardiac arrest. The team performed CPR on her for 20 minutes before she was stabilized, her mom said.

Later, in the pediatric intensive care unit, she suffered internal bleeding, too.

It was really hard, she said. There were many nights that I would just pray and pray and pray.

Initially believing Maci had leukemia, doctors subsequently determined she had myelodysplastic syndrome, or MDS.

The condition occurs when abnormal cells in the bone marrow leave the patient unable to make enough blood, according to the American Cancer Society.

Its rare, afflicting as few 10,000 Americans a year, though the actual number is unknown.

Maci Hyman, 1, interacts with hospital staff before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

In children, its rarer still. Most people arediagnosed in their 70s.

We were told that just four out of 1 million children get it every year, Hailie Hyman said.

That made the diagnosis elusive at first, said Dr. Nichole Bryant, a pediatric hematologist-oncologist with Prisma Health-Upstate, formerly Greenville Health System.

Shes the only one Ive seen in my career, she said.

Maci had to have regular blood transfusions, antibiotics and other medications to fight the MDS, Bryant said. But the only hope for a cure was a stem cell transplant at the Medical University of South Carolina in Charleston.

When they said that was the only treatment plan for MDS, I of course went to Google, Hailie Hyman said. I read about transplant patients and ...all the complications. It was terrifying. But no matter how many bad things I saw, we had to do it. There is no other option.

The transplantis extremely risky.

Hailie Hyman looks at a fish tank with her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

First, high doses of chemotherapy are given to destroy the diseased bone marrow, leaving the patient without an immune system, so fighting infections becomes a challenge. Then healthy donor marrow is infused.

Its also fraught with potentially life-threatening complications, including graft vs. host disease, which occurs when immune cells from the donor attack the patients body, Bryant said. Other complications include permanent kidney damage and gastrointestinal problems.

They have to go to hell and back, she said. But its the only option for long-term survival.

Maci had a really rough start, suffering lots and lots and lots of complications, Bryant said.

Her kidneys failed, so she wound up on dialysis. When she couldnt breathe on her own, she was put on a ventilator. And because she couldnt eat, she had to be tube fed.

Hailie Hyman looks at a fish tank with her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

She had blistering sores in her mouth and throughout her GI tract, her mom said. Because her liver wasnt functioning properly, her abdomen filled up with fluid that had to be drained. She was bleeding so profusely in her lungs that one of them collapsed.

Maci, who was sedated through much of it, was put on full life support, she said.

That night we almost lost her, her mom said. We were in the hallway crying our eyes out. We didnt know what do to or think. It was pretty scary for a while.

Somehow, Maci made it.

There were so many times during her first months that it seemed like she would not survive, Bryant said. So the fact that she is here ... is really a miracle.

Macis family found an unrelated donor through the National Marrow Donor Program, enlisting hundreds of other people to join the registry in the process, Bryant said.

Nichole Bryant, M.D.(Photo: Provided)

It was an important part of their journey that maybe didnt directly benefit Maci, she said. But if everybody did that, we wouldnt have difficulty finding a donor for anybody.

Doctors have no explanation for why Maci got MDS. She didnt carry the genetic mutation for it and there is no family history.

She is a rare child - and not in a good way, her mom said, adding,Youve got to laugh sometimes or youre going to cry.

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Maci was admitted to MUSC on June 2 and released on Oct. 14.

The Hymans, both 22, spent the entire time in Charlestonwhile Hailies mom cared for their older daughter, Athena, now 2.

Treylins employer held his welding job open for him. And other friends and family members did what they could to help.

We had many, many people very generously donate to us to cover expenses at home and living expenses where we were, Hailie Hyman said.

We are thankful for everyone who helped us through it the cards, the gifts, the donations. Every single cent is greatly appreciated.

Maci's doing well, but recovery from a transplant can take months to years, Bryant said.

Her kidneys are functioning again so she was able to come off dialysis. But she still must take many medications, including anti-rejection drugs that suppress her immune system and leaveher at risk for infection. And she still must be tube fed.

She is miles ahead of where she was two months ago, Bryant said. But she still has a long way to go. Its a long, long road.

Macis mom says she can be up and playing one day and flopped over on the couch another. She still experiences a lot of nausea and vomiting, but is doing well compared to where she was.

Hailie Hyman pulls her daughter Maci, 1, in a wagon in the hallway before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

So as the nation pauses to give thanks this Thanksgiving, she says the family will be countingtheir many blessings family andfriends, Gods mercy, andthe doctors and nurses who saved Macis life.

She has battled a lot and overcome a lot, she said. I have no doubt she will be able to get through.

Want to know more about becoming a marrow donor? Go to bethematch.org.

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Novoheart to Co-develop First of its Kind Human Heart-in-a-Jar Model of Heart Failure with AstraZene – PharmiWeb.com

By daniellenierenberg

HONG KONG, Nov. 26, 2019 /PRNewswire/ --Novoheart("Novoheart" or the "Company") (TSXV: NVH; FWB: 3NH), a global stem cell biotechnology company, is pleased to announce a collaboration with global biopharmaceutical company AstraZeneca, in an effort to develop the world's first human-specific in vitro, functional model of heart failure with preserved ejection fraction (HFpEF), a common condition especially among the elderly and in women, with the reported prevalence approaching 10% in women over the age of 80 years.[1]

Heart failure (HF) is a global pandemic with an estimated 64.3 million cases worldwide in 2017, with an increasing trend in prevalence[2]. The annual global economic burden of HF is estimated at over US$100 billion[3]. Accounting for approximately 50% of HF cases, HFpEF in particular is a major and growing public health problem worldwide, with its pathological mechanisms and diverse etiology poorly understood. Due to these complexities, models of the disease available to date, including various animal models, have limited ability to mimic the clinical presentation of HFpEF[4]. Therefore, drug developers lack an effective tool for preclinical testing of drug candidates for efficacy, and as a result, clinical outcomes for HFpEF have not improved over the last decades, with no effective therapies available.

In collaboration with the Cardiovascular, Renal and Metabolism therapy area of AstraZeneca, the initial phase of the project aims to establish a new in vitro model, leveraging Novoheart's proprietary 3-D human ventricular cardiac organoid chamber (hvCOC) technology, that reproduces key phenotypic characteristics of HFpEF. Also known as "human heart-in-a-jar", the hvCOC is the only human engineered heart tissue available on the market to date that enables clinically informative assessment of human cardiac pump performance including ejection fraction and developed pressure. Unlike animal models, engineered hvCOCs can be fabricated with specific cellular and matrix compositions, and patient-specific human induced pluripotent stem cells (iPSCs), that allow control over their physical and mechanical properties to mimic those observed in HFpEF patient hearts. Together with Novoheart's proprietary hardware and software, this aims to provide a unique assay for understanding the mechanisms of HFpEF, identification of new therapeutic targets, and assessment of novel therapeutics for treating HFpEF patients. Novoheart will exclusively own the intellectual property rights to the newly developed HFpEF hvCOC model.

"We are delighted to partner with AstraZeneca, an organization which has long invested in cardiovascular research and is committed to bringing new therapeutic solutions to patients with heart failure," said Novoheart CSO, Dr. Kevin Costa. "We look forward to co-developing this new HFpEF hvCOC model into a powerful new tool in the worldwide battle against heart failure."

Regina Fritsche Danielson, Senior Vice President, Head of Research and Early Development, Cardiovascular, Renal and Metabolism, BioPharmaceuticals R&D, AstraZeneca, said, "There are significant unmet treatment needs in patients with heart failure with preserved ejection fraction. By combining Novoheart's proprietary hvCOC model with our expertise in heart failure, we aim to create the first in vitro model reproducing phenotypic characteristics of heart failure with preserved ejection fraction. This could bridge the gap between in vivo animal models and clinical trials to help accelerate the drug discovery process by providing human-specific preclinical data."

[1] Heart Fail Clin. 2014; 10(3):377-388.

[2] Lancet. 2018; 392:1789-1858.

[3] Int J Cardiol. 2014; 171(3):368-76.

[4]JACC Basic Transl Sci. 2017; 2(6):770-789.

About Novoheart:

Novoheartis a global stem cell biotechnology company pioneering an array of next-generation human heart tissue prototypes. It is the first company in the world to have engineered miniature living human heart pumps that can revolutionize drug discovery, helping to save time and money for developing new therapeutics. Also known as 'human heart-in-a-jar', Novoheart's bio-artificial human heart constructs are created using state-of-the-art and proprietary stem cell and bioengineering approaches and are utilized by drug developers for accurate preclinical testing of the effectiveness and safety of new drugs, maximizing the successes in drug discovery whilst minimizing costs and harm caused to patients. With the recent acquisition of Xellera Therapeutics Limited for manufacturing Good Manufacturing Product (GMP)-grade clinical materials, Novoheart is now developing gene- and cell-based therapies as well as next-generation therapeutics for cardiac repair or regeneration.

Neither TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release.

Cautionary Note Regarding Forward-Looking Statements

Information set forth in this news release may involve forward-looking statements under applicable securities laws. Forward-looking statements are statements that relate to future, not past, events. In this context, forward-looking statements often address expected future business and financial performance, and often contain words such as "anticipate", "believe", "plan", "estimate", "expect", and "intend", statements that an action or event "may", "might", "could", "should", or "will" be taken or occur, or other similar expressions. By their nature, forward-looking statements involve known and unknown risks, uncertainties and other factors which may cause the actual results, performance or achievements, or other future events, to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Such factors include, among others, the risks identified in under the heading "Risk Factors" in Novoheart's annual information form for the year ended June 30, 2018 or other reports and filings with the TSX Venture Exchange and applicable Canadian securities regulators. Forward-looking statements are made based on management's beliefs, estimates and opinions on the date that statements are made and the respective companies undertakes no obligation to update forward-looking statements if these beliefs, estimates and opinions or other circumstances should change, except as required by applicable securities laws. Investors are cautioned against attributing undue certainty to forward-looking statements.

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SOURCE Novoheart

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‘My daughter’s death took me to the darkest place, but I’ve learned it’s possible to come back’ – Telegraph.co.uk

By daniellenierenberg

Appiah rang Leukaemia Cares helpline from the point of diagnosis until well after the end of her daughters life. Sometimes Id call them as a means of support, she says, when things got really rough, when her medications were really powerful, and the chemo made her so unwell. She rang when she had panic attacks; an NHS psychologist had told her that these were likely, and that she should breathe into a brown paper bag, but Appiah found speaking to a person more soothing.

With a laugh, Appiah notes that shed ring the helpline at other times, too: Sometimes Id be out with Imogin, and shed be in the pram, being naughty, and all of my patience was going down the drain, and Id phone Leukaemia Cares nurses, and say: Look, Im feeling so depressed, my daughters shouting, I dont know what to do!

But I might also say: Nurse, Im actually feeling good today.

Appiah says the support of an independent person was invaluable: When your child is so ill, you need to speak to someone who doesnt know your name you need an outsider you can unload to. I didnt want anyone thinking: Here Sheila comes again!

You become self-conscious about your situation and dont want to be a burden on your friends and family. With the helpline, you wont be judged: they just listen. You get it out of your system and then go do the shopping at Sainsburys.

When Imogin was well, shed go to school. But she also spent weeks at a time in isolation in St Georges Hospital, with her mother by her side. Once, she had a bad reaction to a medication and went into cardiac arrest. She was crying and saying, Please, please! and they were giving her all sorts of medicine. The doctors were battling to keep her stable and I dived into the bed with her and told her: Youre going to be OK. I lay down with her and I started singing with her. And then, once she stabilised, she said: Now can I watch High School Musical?"

Appiah shakes her head, laughing: Thats what she was like: I was on thedoor of death, but I have something else planned. I want to watch my video and none of you are going to stop me!

Charities sent the pair to Disneyland Paris twice. The first time was fantastic, says Appiah, the second time Imogin was in and out of consciousness. But they said we should go, to make memories, Appiah explains. Imogin got to be a celebrity for a day and went to Hamleys in a limousine to get anything she wanted.

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'My daughter's death took me to the darkest place, but I've learned it's possible to come back' - Telegraph.co.uk

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Stem Cell Assay Market Demand with Leading Key Players and New Investment Opportunities Emerge To Augment Segments in Sector By 2025 – The Denton…

By daniellenierenberg

Stem Cell Assay Market: Snapshot

Stem cell assay refers to the procedure of measuring the potency of antineoplastic drugs, on the basis of their capability of retarding the growth of human tumor cells. The assay consists of qualitative or quantitative analysis or testing of affected tissues and tumors, wherein their toxicity, impurity, and other aspects are studied.

With the growing number of successful stem cell therapy treatment cases, the global market for stem cell assays will gain substantial momentum. A number of research and development projects are lending a hand to the growth of the market. For instance, the University of Washingtons Institute for Stem Cell and Regenerative Medicine (ISCRM) has attempted to manipulate stem cells to heal eye, kidney, and heart injuries. A number of diseases such as Alzheimers, spinal cord injury, Parkinsons, diabetes, stroke, retinal disease, cancer, rheumatoid arthritis, and neurological diseases can be successfully treated via stem cell therapy. Therefore, stem cell assays will exhibit growing demand.

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Another key development in the stem cell assay market is the development of innovative stem cell therapies. In April 2017, for instance, the first participant in an innovative clinical trial at the University of Wisconsin School of Medicine and Public Health was successfully treated with stem cell therapy. CardiAMP, the investigational therapy, has been designed to direct a large dose of the patients own bone-marrow cells to the point of cardiac injury, stimulating the natural healing response of the body.

Newer areas of application in medicine are being explored constantly. Consequently, stem cell assays are likely to play a key role in the formulation of treatments of a number of diseases.

Global Stem Cell Assay Market: Overview

The increasing investment in research and development of novel therapeutics owing to the rising incidence of chronic diseases has led to immense growth in the global stem cell assay market. In the next couple of years, the market is expected to spawn into a multi-billion dollar industry as healthcare sector and governments around the world increase their research spending.

The report analyzes the prevalent opportunities for the markets growth and those that companies should capitalize in the near future to strengthen their position in the market. It presents insights into the growth drivers and lists down the major restraints. Additionally, the report gauges the effect of Porters five forces on the overall stem cell assay market.

Global Stem Cell Assay Market: Key Market Segments

For the purpose of the study, the report segments the global stem cell assay market based on various parameters. For instance, in terms of assay type, the market can be segmented into isolation and purification, viability, cell identification, differentiation, proliferation, apoptosis, and function. By kit, the market can be bifurcated into human embryonic stem cell kits and adult stem cell kits. Based on instruments, flow cytometer, cell imaging systems, automated cell counter, and micro electrode arrays could be the key market segments.

In terms of application, the market can be segmented into drug discovery and development, clinical research, and regenerative medicine and therapy. The growth witnessed across the aforementioned application segments will be influenced by the increasing incidence of chronic ailments which will translate into the rising demand for regenerative medicines. Finally, based on end users, research institutes and industry research constitute the key market segments.

The report includes a detailed assessment of the various factors influencing the markets expansion across its key segments. The ones holding the most lucrative prospects are analyzed, and the factors restraining its trajectory across key segments are also discussed at length.

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Global Stem Cell Assay Market: Regional Analysis

Regionally, the market is expected to witness heightened demand in the developed countries across Europe and North America. The increasing incidence of chronic ailments and the subsequently expanding patient population are the chief drivers of the stem cell assay market in North America. Besides this, the market is also expected to witness lucrative opportunities in Asia Pacific and Rest of the World.

Global Stem Cell Assay Market: Vendor Landscape

A major inclusion in the report is the detailed assessment of the markets vendor landscape. For the purpose of the study the report therefore profiles some of the leading players having influence on the overall market dynamics. It also conducts SWOT analysis to study the strengths and weaknesses of the companies profiled and identify threats and opportunities that these enterprises are forecast to witness over the course of the reports forecast period.

Some of the most prominent enterprises operating in the global stem cell assay market are Bio-Rad Laboratories, Inc (U.S.), Thermo Fisher Scientific Inc. (U.S.), GE Healthcare (U.K.), Hemogenix Inc. (U.S.), Promega Corporation (U.S.), Bio-Techne Corporation (U.S.), Merck KGaA (Germany), STEMCELL Technologies Inc. (CA), Cell Biolabs, Inc. (U.S.), and Cellular Dynamics International, Inc. (U.S.).

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Researchers identify protein that governs human blood stem cell self-renewal – Newswise

By daniellenierenberg

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Newswise UCLA scientists have discovered a link between a protein and the ability of human blood stem cells to self-renew. In a study published today in the journal Nature, the team reports that activating the protein causes blood stem cells to self-renew at least twelvefold in laboratory conditions.

Multiplying blood stem cells in conditions outside the human body could greatly improve treatment options for blood cancers like leukemia and for many inherited blood diseases.

Dr. Hanna Mikkola, a member of theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLAand senior author of the study, has studied blood stem cells for more than 20 years.

Although weve learned a lot about the biology of these cells over the years, one key challenge has remained: making human blood stem cells self-renew in the lab, she said. We have to overcome this obstacle to move the field forward.

Blood stem cells, also known as hematopoietic stem cells, are found in the bone marrow, where they self-renew as well as differentiate to create all types of blood cells.Bone marrow transplants have been used for decades to treat people with some diseases of the blood or immune system. However, bone marrow transplants have significant limitations: Finding a compatible bone marrow donor is not always possible, the patients immune system may reject the foreign cells, and the number of transplanted stem cells may not be enough to successfully treat the disease.

When blood stem cells are removed from the bone marrow and placed in laboratory dishes, they quickly lose their ability to self-renew, and they either die or differentiate into other blood cell types. Mikkolas goal, making blood stem cells self-renew in controlled laboratory conditions, would open up a host of new possibilities for treating many blood disorders among them safer genetic engineering of patients own blood stem cells. It could also enable scientists to produce blood stem cells from pluripotent stem cells, which have the potential to create any cell type in the body.

To uncover what makes blood stem cells self-renew in a lab, the researchers analyzed the genes that turn off as human blood stem cells lose their ability to self-renew, noting which genes turned off when blood stem cells differentiate into specific blood cells such as white or red cells. They then put the blood stem cells into laboratory dishes and observed which genes shut down. Using pluripotent stem cells, they made blood stem cell-like cells that lacked the ability to self-renew and monitored which genes were not activated.

They found that the expression of a gene called MLLT3 was closely correlated with blood stem cells potential to self-renew and that the protein generated by the MLLT3 gene provides blood stem cells with the instructions necessary to maintain its ability to self-renew. It does this by working with other regulatory proteins to keep important parts of the blood stem cells machinery operational as the cells divide.

The researchers wondered if maintaining the level of the MLLT3 protein in blood stem cells in lab dishes would be sufficient to improve their self-renewing abilities. Using a viral vector a specially modified virus that can carry genetic information to a cells nucleus without causing a disease the team inserted an active MLLT3 gene into blood stem cells and observed that functional blood stem cells were able to multiply in number at least twelvefold in lab dishes.

If we think about the amount of blood stem cells needed to treat a patient, thats a significant number, said Mikkola, who is also a professor of molecular, cell and developmental biology in the UCLA College and a member of theUCLA Jonsson Comprehensive Cancer Center. But were not just focusing on quantity; we also need to ensure that the lab-created blood stem cells can continue to function properly by making all blood cell types when transplanted.

Other recent studies have identified small molecules organic compounds that are often used to create pharmaceutical drugs that help to multiply human blood stem cells in the laboratory. When Mikkolas team used the small molecules, they observed that blood stem cell self-renewal improved in general, but the cells could not maintain proper MLLT3 levels, and they also did not function as well when transplanted into mice.

The previous discoveries with the small molecules are very important, and were building on them, said Vincenzo Calvanese, a UCLA project scientist and the studys co-corresponding author. Our method, which exposes blood stem cells to the small molecules and also inserts an active MLLT3 gene, created blood stem cells that integrated well into mouse bone marrow, efficiently produced all blood cell types and maintained their self-renewing ability."

Importantly, MLLT3 made the blood stem cells self-renew at a safe rate; they didnt acquire any dangerous characteristics such as multiplying too much or mutating and producing abnormal cells that could lead to leukemia.

The next steps for the researchers include determining what proteins and elements within blood stem cell DNA influence the on-off switch for MLLT3, and how this could be controlled using ingredients in the lab dishes. With that information, they could potentially find ways to switch MLLT3 on and off without the use of a viral vector, which would be safer for use in a clinical setting.

The research was supported by the National Institutes of Health, the Leukemia and Lymphoma Society, the California Institute for Regenerative Medicine, the American Association for Cancer Research, the Arnold and Mabel Beckman Foundation, the Jonsson Comprehensive Cancer Center Foundation, and the UCLA Broad Stem Cell Research Center, including support from The Rose Hills Foundation Innovator Grant, as well as the centers training program.

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Stem Cell Therapy Helps Broken Hearts Heal in Unexpected Way – Newswise

By daniellenierenberg

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Research Results

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Newswise CINCINNATI -- Stem cell therapy helps hearts recover from a heart attack, although not for the biological reasons originally proposed two decades ago that today are the basis of ongoing clinical trials. This is the conclusion of a Nov. 27 study in Nature that shows an entirely different way that heart stem cells help the injured heart not by replacing damaged or dead heart cells as initially proposed.

The study reports that injecting living or even dead heart stem cells into the injured hearts of mice triggers an acute inflammatory process, which in turn generates a wound healing-like response to enhance the mechanical properties of the injured area.

Mediated by macrophage cells of the immune system, the secondary healing process provided a modest benefit to heart function after heart attack, according to Jeffery Molkentin, PhD, principal investigator, director of Molecular Cardiovascular Microbiology a Cincinnati Childrens Hospital Medical Center and a professor of the Howard Hughes Medical Institute (HHMI).

The innate immune response acutely altered cellular activity around the injured area of the heart so that it healed with a more optimized scar and improved contractile properties, Molkentin said. The implications of our study are very straight forward and present important new evidence about an unsettled debate in the field of cardiovascular medicine.

The new paper builds on a 2014 study published by the same research team, also in Nature. As in that earlier study, the current paper shows that injecting c-kit positive heart stem cells into damaged hearts as a strategy to regenerate cardiomyocytes doesnt work. The findings prompted Molkentin and his colleagues to conclude that there is a need to re-evaluate the current planned cell therapy based clinical trials to ask how this therapy might really work.

An Unexpected Discovery

The study worked with two types of heart stem cells currently used in the clinical trialsbone marrow mononuclear cells and cardiac progenitor cells. As the researchers went through the process of testing and re-verifying their data under different conditions, they were surprised to discover that in addition to the two types of stem cells, injecting dead cells or even an inert chemical called zymosan also provided benefit to the heart by optimizing the healing process. Zymosan is a substance designed to induce an innate immune response

Researchers reported that stem cells or zymosan therapies tested in this study altered immune cell responses that significantly decreased the formation of extra cellular matrix connective tissue in the injury areas, while also improving the mechanical properties of the scar itself. The authors concluded: injected hearts produced a significantly greater change in passive force over increasing stretch, a profile that was more like uninjured hearts.

Molkentin and his colleagues also found that stem cells and other therapeutic substances like zymosan have to be injected directly into the hearts surrounding the area of infarction injury. This is in contrast to most past human clinical trials that for patient safety reasons simply injected stem cells into the circulatory system.

Most of the current trials were also incorrectly designed because they infuse cells into the vasculature, Molkentin explained. Our results show that the injected material has to go directly into the heart tissue flanking the infarct region. This is where the healing is occurring and where the macrophages can work their magic.

The researchers also noted an interesting finding involving zymosan, a chemical compound that binds with select pattern recognition receptors to cause an acute innate immune response. Using zymosan to treat injured hearts in mice resulted in a slightly greater and longer-lasting benefit on injured tissues than injecting stem cells or dead cell debris.

Looking to the Future

Molkentin said he and other collaborating scientists will follow up the findings by looking for ways to leverage the healing properties of the stem cells and compounds they tested.

For example, considering how heart stem cells, cell debris and zymosan all triggered an acute innate immune response involving macrophages in the current paper, Molkentin explained they will test a theory that harnesses the selective healing properties of macrophages. This includes polarizing or biologically queuing macrophages to only have healing-like properties.

Further testing of this, he said, could therapeutically be very important for developing future treatment strategies.

The studys first author was Ronald Vagnozzi, PhD, a fellow and investigator in the Molkentin laboratory. Key collaboration also came from scientists in the Cincinnati Childrens Heart Institute, the University of Cincinnati Department Of Pediatrics and the Center for Systems Biology (Department of Imaging) and the Cardiovascular Research Center at Massachusetts General Hospital and Harvard Medical School in Boston.

Funding support for the study came in part by grants from the National Institutes of Health (R01 HL132391) and an NIH Research Service Award via the National Heart Blood and Lung Institute (F32 HL128083), the Howard Hughes Medical Institute, and a Career Development Award from the American Heart Association (19CDA34670044). Flow cytometric data were acquired using equipment maintained by the Research Flow Cytometry Core in the Division of Rheumatology at Cincinnati Childrens.

Post Embargo Study Link: https://www.nature.com/articles/s41586-019-1802-2

SEE ORIGINAL STUDY

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Translink in Armagh holding stem cell registration event in memory of Gavin McNaney – Armagh i

By daniellenierenberg

Gavin McNaney, who passed away two years ago.

Translink are hosting a blood stem cell registration event and coffee morning this Saturday as part of the Somebodys Stranger campaign, in memory of Armagh man Gavin McNaney, who passed away two years ago.

It will take place from 10am until 2pm and it is a painless process which could potentially save a life.

Former St Catherines College teacher Gavin was just 37 years of age when he passed on November 18, 2017.

He had been diagnosed with Acute Lymphoblastic Leukaemia whilst teaching in Dubai.

Gavin spent months in hospital undergoing treatment and had a bone marrow transplant in London.

But after contracting a common cold and an infection to his lungs, his life was sadly cut short and he passed away peacefully with mum and dad, Nuala and Pat, by his side.

Friend Karl McQuaid has been raising funds and awareness after the passing of his life-long friend, whom he had first met when they both attended St Patricks Grammar School in Armagh.

He has been running registration events as part of his Somebodys Stranger campaign for nine months in Gavins memory and is keen to advise people just how easy it is to register .

He who would like to thank Leanne Armstrong and her colleagues at Translink for inviting them to come along told Armagh I : Joining the stem cell register is quick, easy and pain-free.

Potential donors have a swab taken of the inside of their cheeks with the whole process taking just a few minutes. They will then be added to DKMSs worldwide database and could be contacted at any time should they be a genetic match for a blood cancer sufferer anywhere in the world.

Those lucky enough to be a match would then be asked to donate their stem cells in a pain-free procedure similar to giving blood. This could save the life of the cancer sufferer.

Donations are at your own discretion at the event with all proceeds going to Leukaemia & Lymphoma NI Northern Irelands only charity dedicated to fighting blood cancers.

Those willing to join the register should be in general good health and aged between 18 and 55.

All are urged to come along on Saturday morning, when the city will be full of revellers for the annual Georgian Day event. Please take time to come along to the bus station and help make a huge difference.

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The Best Thanksgiving – Financialbuzz.com

By daniellenierenberg

Bethesda, Maryland, Nov. 26, 2019 (GLOBE NEWSWIRE) One Texas family has lots to be thankful for this Thanksgiving. Their daughter, now 13, is doing well after undergoing a bone marrow transplantthe only chance for a cure for her rare and deadly disease. But Emis story is not only a story about the triumph of medical research that is making her cure possibleits also a story about extraordinary parental love and sacrifices by her birth mom and her adoptive family that are giving this very ill girl the best chance at life. Emis birth mom donated her stem cells to make the lifesaving transplant possible.

We are most thankful for an answer to years of prayers, Emis adoptive mom says. Emi got a new start at life, a rebirth day. Every holiday this year will be like the first. Were so grateful to the doctors, nurses and The Childrens Inn.

Emi and her family will be celebrating Thanksgiving at The Childrens Inn at NIH, a nonprofit hospitality house that provides free lodging and a wide variety of support services to families of children with rare and serious diseases whose best chance for a treatment is a clinical research study at the National Institutes of Health. Emi and her mom have spent several months at The Childrens Inn so far and bonded with other families. On Thanksgiving Day, families staying at The Childrens Inn who cannot go home for the holiday will be served a traditional Thanksgiving meal prepared by a group of dedicated volunteers.

It took two moms who love this little nugget to fight for her right to life, Emis adoptive mom says. We finally are getting to see that beautiful part of the story that we always knew was there.

Read Emis full story.

See photos of Emi and her family.

About The Childrens Inn at NIH:

The Childrens Inn at NIH provides free lodging and a wide range of supportive services to more than 1,500 children and their families every year whose best chance for a treatment is a clinical trial at the National Institutes of Health. Opened in 1990 and located across from the NIH Clinical Center, the worlds largest hospital dedicated entirely to medical research, The Childrens Inn has welcomed children from all 50 states and 94 countries. Children staying at The Childrens Inn are making important contributions to rare disease and cancer research, including the successful treatment of childhood leukemia, as well as treatments for HIV/AIDS, childhood asthma, bone and growth diseases, childhood onset schizophrenia and other mental health issues, neurofibromatosis type 1 and a wide variety of genetic and rare diseases. For more information, visit http://www.childrensinn.org. To support The Childrens Inn, make a donation at http://www.childrensinn.org/donate.

###

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Stem Cell Banking Market Size, by Source (Placental Stem Cells), by Application (Personalized Banking Applications), by Service Type (Sample…

By daniellenierenberg

Stem Cell Banking Market 2019 Industry report provides detailed statistics and accurate market figures, viz. market share, CAGR, gross margin, and those related to revenue, production, consumption, and sales. It also provides a regional analysis of the global Stem Cell Banking market to unveil key opportunities available in different parts of the world. This all analyzed data will help a new player and existing players to take a critical decision.

Get Sample Copy of this Report @ https://www.orianresearch.com/request-sample/1369382

The key players profiled in the market include: Cord Blood Registry Systems, Cordlife, Cryo-Cell International, Cryo-Save AG, LifeCell International, StemCyte, ViaCord, Global Cord Blood, Smart Cells International and Vita34

The global Stem Cell Banking market was estimated to be valued at USD XX million in 2018 and is projected to reach USD XX million by 2026, at a CAGR of XX% during 2019 to 2026. Growing awareness on the therapeutic potential of stem cells coupled with the increasing investments in stem cell-based research will aid in augmenting the market growth. However, high operational costs of stem cell banking and stringent regulations will impede the market growth during the analysis period.

The global stem cell banking market is segmented on the basis of source, application, service type and region. Based on source the market is segmented into Placental Stem Cells (PSCs), Human Embryo-derived Stem Cells (hESCs), Bone Marrow-derived Stem Cells (BMSCs), Adipose Tissue-derived Stem Cells (ADSCs), Dental Pulp-derived Stem Cells (DPSCs) and other stem cell sources. Based on application the market is segmented into personalized banking applications, clinical applications, hematopoietic disorders, autoimmune disorders, other diseases, research applications, disease treatment studies, life science research and drug discovery. Based on service type the market is segmented into sample collection & transportation, sample processing, sample analysis and sample preservation & storage. Based on region, it is studied across North America Europe, Asia-Pacific, South America and Middle East and Africa.

No of Pages: 121

Key Benefits of the Report:

* Global, regional, by type, storage capacity, and by end user wise market size and their forecast from 2015-2026

* Identification and detailed analysis on key market dynamics, such as, drivers, restraints, opportunities, and challenges influencing growth of the market

* Detailed analysis on product outlook with market specific Porters Five SSDs analysis, PESTLE analysis, and Value Chain, to better understand the market and build expansion strategies

* Identification of key market players and comprehensively analyze their market share and core competencies, detailed financial positions, key product, and unique selling points

* Analysis on key players strategic initiatives and competitive developments, such as joint ventures, mergers, and new product launches in the market

* Expert interviews and their insights on market shift, current and future outlook, and factors impacting vendors short term and long term strategies

* Detailed insights on emerging regions, by type, storage capacity, and by end user with qualitative and quantitative on premise and facts

The encrypted hard drives market is primarily segmented based on type, by storage capacity, by end user, and region.

Order a copy of Global Stem Cell Banking Market Report @ https://www.orianresearch.com/discount/1369382

On the basis of source, the market is split into:

* Placental Stem Cells (PSCs)

* Human Embryo-derived Stem Cells (hESCs)

* Bone Marrow-derived Stem Cells (BMSCs)

* Adipose Tissue-derived Stem Cells (ADSCs)

* Dental Pulp-derived Stem Cells (DPSCs)

* Other Stem Cell Sources

Based on end user, the market is divided into:

* Personalized Banking Applications

* Clinical Applications

* Hematopoietic Disorders

* Autoimmune Disorders

* Other Diseases

* Research Applications

* Disease Treatment Studies

* Life Science Research

* Drug Discovery

On the basis of service type, the market is split into:

* Sample Collection & Transportation

* Sample Processing

* Sample Analysis

* Sample Preservation & Storage

These enterprises are focusing on growth strategies, such as, technological advancements, expansions, acquisitions, and agreements & partnerships to expand their operations across the globe.

Target Audience:

* Stem Cell Banking Manufacturers

* Industry Participants and Associations

Research Methodology:

The market is derived through extensive use of secondary, primary, in-house research followed by expert validation and third party perspective, such as, analyst reports of investment banks. The secondary research is the primary base of our study wherein we conducted extensive data mining, referring to verified data sources, such as, white papers, research and regulatory published articles, technical journals, trade magazines, and paid data sources.

For forecasting, regional demand & supply factors, recent investments, market dynamics including technical growth scenario, consumer behavior, and product trends and dynamics, and product capacity were taken into consideration. Different weightages have been assigned to these parameters and quantified their Market impacts using the weighted average analysis to derive the Market growth rate.

The market estimates and forecasts have been verified through exhaustive primary research with the Key Industry Participants (KIPs), which typically include:

* Manufacturers

* Suppliers

* Distributors

* Government Body & Associations

* Research Institutes

About Us:

Orian Research is one of the most comprehensive collections of market intelligence reports on the World Wide Web. Our reports repository boasts of over 500000+ industry and country research reports from over 100 top publishers. We continuously update our repository so as to provide our clients easy access to the worlds most complete and current database of expert insights on global industries, companies, and products. We also specialize in custom research in situations where our syndicate research offerings do not meet the specific requirements of our esteemed clients.

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Vice President Global Sales & Partner Relations

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Bone Marrow Aspirate Concentrates Market to Expand at an Outstanding CAGR of 5.0% from 2017 to 2025 – Statsflash

By daniellenierenberg

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.

Request a PDF Sample of Bone Marrow Aspirate Concentrates Market Report https://www.transparencymarketresearch.com/sample/sample.php?flag=S&rep_id=40451

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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Makeup brand offers spray-on ‘skin’ to cover up zits and scars – New York Post

By daniellenierenberg

Soon youll be able to cover your imperfect flesh with more flesh.

Japanese cosmetics company Kao Corporation has developed a custom synthetic spray-on skin to cover unwanted blemishes, moles or other marks on the natural epidermis.

The artificial product, called est, is composed of tiny, liquid fibers. When sprayed, the substance adheres to human skin, transforming into an extremely thin, derma-like material, the Daily Mail reports.

It has a similar elasticity to skin, and its porous, too. Water vapor and air can pass through this second skin to moisten the living dermis beneath. At its edges, est forms an even thinner bond, helping it blend in with natural flesh.

Est is set to hit the market exclusively in Japan beginning Dec 4. and will sell for roughly $532 as a diffuser and potion combination, with diffuser refills priced at $73. A lotion version will sell for $110, and everything will become available online in January, according to Japanese publication the Asahi Shimbun.

Japanese-language advertisements for the product call it Future Skin, which uses Fine Fiber Technology. Kao has plans to expand the line beginning next year and hopes to soon enter the medical market.

Until then, American consumers can check out the SkinGun by RenovaCare, which shoots a liquid mist infused with human stem cells and can help burn victims skin.

Kao Japan

Kao Japan

Kao Japan

Kao Japan

Kao Japan

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Inside the Beltway: Abortion, immigration among forbidden topics at Thanksgiving table – Washington Times

By daniellenierenberg

The warnings are already up in the popular press: Conversations during the Thanksgiving feast can be hazardous if they veer into political territory. But political talk can take place in theory: A data research company has now determined what topics are safe to talk about on the holiday.

With some qualitative analysis and a little common sense, weve created a cheat sheet that will help you blaze a path through Thanksgiving dinner that steers clear of treacherous political pitfalls and dangerous inter-uncle conflicts, reports Ranker.com, a Los Angeles-based media company which uses crowdsourcing to rank public opinion on multiple topics, typically at the rate of 15 million votes a month.

They have determined what political topics are the least and the most likely to set off a Thanksgiving dinner squabble. Their judgment is based on 300,000 votes from 40,000 respondents.

The topics to avoid this year: Abortion, immigration, terrorism and gender equality. The topics which are safe for dinnertime discussion: Ineffective government, health care reform and education.

If there are millennials present, they will be triggered, the organization says, by talk of abortion, police brutality and pollution. Generation X members will be set off by such topics as homelessness, affordable housing and campaign finance reform. Baby boomers will go to battle over terrorism, immigration and the moral decline of the nation.

The organization also has warnings for dinner hosts in certain states. If they live in Florida, their guests will be particularly sensitive about discussions of vaccines. In Indiana, its gender equality while Georgia diners are prone to fight over police brutality. Beware of talking about gun control at dinner tables in both California and Missouri; Texans get feisty over moral decline. New Yorkers get upset over transgender issues.

We examined each issue on a case-by-case basis to find the topics that are most likely to cause disagreement, as well as the ones on which people tend to either agree or not care about, Ranker.com explains.

A VERY SPECIALIZED MEAL

While most of us are enjoying turkey and pumpkin pie on Thanksgiving, the staff at one laboratory at Cedars-Sinai Medical Center in Los Angeles will be busy serving a meal to stem cells.

Stem cells do not observe national holidays, says Loren Ornelas-Menendez, manager of the very specialized lab that converts samples of adult skin and blood cells into stem cells which the human body uses to make our cells in the first place.

These special cells help medical scientists learn how diseases develop and how they might be cured. The lab is tending millions of them. Oh, but they have needs.

Stem cells are living creatures that must be hand-fed a special formula each day, monitored for defects and maintained at just the right temperature. And that means the cell lab is staffed every day, 52 weeks a year, the lab notes in a public advisory.

Many people have dogs. We have stem cells, says Ms. Ornelas-Menendez.

Derived from hundreds of healthy donors and patients, the resident induced pluripotent stem cells or iSPCs are keys to potential treatments for diabetes, breast cancer, Alzheimers disease, blindness, Parkinsons disease and Crohns disease, among other conditions. Ten lab technicians monitor the cells through microscopes each day and cull out any cells which have gone awry for one reason or another.

But what do they eat even on Thanksgiving?

While the cells get sorted, a special feeding formula is defrosting in a dozen bottles spread around a lab bench. The formula includes sodium, glucose, vitamins and proteins. Using pipettes, employees squeeze the liquid into food wells inside little compartments that contain the iPSCs. Afterward, they return the cells to their incubators, the lab advises.

Lab director Dhruv Sareen suggests that people consider offering a toast to the stem cells on Thanksgiving.

One day the cells they tend could lead to treatments for diseases that have plagued humankind for centuries, he says. And thats something to be truly thankful for.

THE GIPPERS FAVORITE

Back by popular demand, Inside the Beltway again shares this little known but historic recipe for President Reagans Favorite Macaroni and Cheese enjoyed by Ronald Reagan and his family on Thanksgiving and other holidays. What follows is a step-by-step shared by Mrs. Ronald Reagan, Washington, D.C., Wife of the President in a spiral-bound community cookbook published by the American Cancer Societys Northern Virginia division in 1983. The recipe serves six and is baked at 350 degrees F for 45 minutes.

The directions are from the cookbook reflecting the style, perhaps, of another era:

1/2 pound macaroni, 1 teaspoon butter, 1 egg, beaten; 1 teaspoon salt, 1 teaspoon dry mustard, 3 cups grated cheese, sharp; 1 cup milk.

Boil macaroni in water until tender and drain thoroughly. Stir in butter and egg. Mix mustard and salt with 1 tablespoon hot water and add to milk. Add cheese leaving enough to sprinkle on top. Pour into buttered casserole, add milk, sprinkle with cheese. Bake until custard is set and top is crusty.

Curious about what transpired at a Reagan Thanksgiving? A 1985 Los Angeles Times account noted this:

President and Mrs. Reagan gathered with their family for a quiet Thanksgiving dinner at their fogbound ranch in the Santa Ynez mountains, where the main topic of conversation was the weather. The Reagans did not seem to mind the enforced seclusion as they sat down to a traditional turkey dinner, prepared by Ann Allman, the Reagan familys longtime cook in California. It was an all-American menu that included cornbread dressing, cranberries, string beans, mashed potatoes, salad, pumpkin pie and monkey bread, a family favorite.

POLL DU JOUR

46% of Americans say long standing family tensions are the cause of family fights during holidays.

37% say general politics is the cause; 33% cite the 2020 presidential race.

24% say someones future plans cause the fights; 24% say money.

22% say the behavior of guests; 21% say drinking and alcohol.

18% say holiday cooking is the cause.

Source: A YouGov poll of 1,310 U.S. Adults conducted Sept. 25-26 and released Tuesday.

Have a happy Thanksgiving and thank you for reading Inside the Beltway.

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Time to Try Again: Gene-Based Therapy for Neurodegeneration – Alzforum

By daniellenierenberg

27 Nov 2019

Twenty years ago, researchers took fibroblasts from the skin of eight Alzheimers patients, engineered them to produce nerve-growth factor, and slid them into each volunteers basal forebrain. They hoped the neurotrophin would halt or slow the neurodegeneration that robbed them of their memories, indeed their lives. The gamble failed and since then, scientists have shown little zest for gene therapy in neurodegenerative disorders. That is changing. As evident at this years Society for Neuroscience conference, held October 1923 in Chicago, gene therapy is back. Buoyed by success in treating spinal muscular atrophy in infants, scientists are flush with new ideasand funding.

What was once considered risky, expensive, and unlikely to succeed is now seen by many as risky, expensiveand quite likely to succeed. A growing number of scientists think gene-based therapies may have the best chance of slowing, or even preventing, neurodegeneration, especially for disorders caused by mutations in a single gene. SfN hosted a press briefing on gene therapy, plus many projects are active throughout the field beyond those showcased at the conference. There was no breaking clinical trial news at the annual meeting, but the scope and challenges of such therapies were outlined at the briefing moderated by Rush University s Jeff Kordower, Chicago, as well as a translational roundtable moderated by Asa Abeliovich, Columbia University, New York. Abeliovich recently co-founded Prevail Therapeutics, New York.

Going viral. Researchers are tweaking the capsid of adeno-associated viruses to optimize gene therapies for a multitude of disease. Shown here, AAV2.

From Zolgensma to Alzheimers? If the failure of the nerve growth factor therapy tempered enthusiasm for gene therapy (Mar 2018 news), then the success of AVXS-101, aka Zolgensma, reignited it. Developed by scientists at Nationwide Childrens Hospital, Columbus, Ohio, and AveXis, Bannockburn, Illinois, AVXS-101 uses an adeno-associated virus to deliver billions of copies of the survival motor neuron 1 gene to the brain. A small pilot trial tested the therapy in babies with spinal muscular atrophy (SMA) Type 1, the severest form of this neurodevelopmental disease. Lacking functional SMN1, these infants face progressive muscle weakness. Most die before their second birthday; those who live need a ventilator to breathe.

In Phase 1, AVXS-101 dramatically improved motor function of 15 treated infants; all were living 20 months later when historical data predicted only one would survive. Twelve babies who received the highest dose grew stronger within months, most sitting independently and rolling over. They hit the highest score on a scale of motor function, whereas untreated babies deteriorated. By 20 months, two of the treated babies had begun to walk (Mendell et al., 2017). The Food and Drug Administration approved zolgensma in May 2019. At SfN in Chicago, Petra Kaufmann, AveXis, played videos of the first patients treated with AVXS-101. Some four years later, they are walking, running, and appear to be playing almost normally. A video of a little girl walking downstairs with nary a hint of having SMA Type I visibly moved the audience.

Scientists say its a game-changer. It is really the tremendous success with SMA that has renewed interest in gene therapy, said Clive Svendsen, Cedars-Sinai Regenerative Medicine Institute, Los Angeles. Speaking with Alzforum before SfN, Bart De Strooper, Dementia Research Institute, London, said the same. The success in SMA patients of both gene therapy and antisense therapy has revived interest in the whole area, De Strooper said. Nowadays, researchers tend to lump gene therapy and antisense therapy under one moniker, i.e., gene-based therapy. The SMA antisense therapy nusinersen also works in babies with SMA Type 1 and is FDA-approved (Nov 2016 news; May 2018 conference news). Unlike gene therapy, antisense therapy needs to be delivered indefinitely.

How About Neurodegenerative Disease?At SfN, scientists outlined strategies for treating adults who face years of decline due to Alzheimers, amyotrophic lateral sclerosis, frontotemporal dementia, Huntingtons (HD) and Parkinsons diseases (PD), or other synucleinopathies. Some are being tested in clinical trials, others are in preclinical development. Some target specific losses or gains of function, others aim to rescue dying neurons more broadly. Scientists also believe that working on rare childhood diseases of lysosomal storage may give them an opening to treat this common phenotype in age-related neurodegeneration, as well.

Just this October, an ApoE gene therapy trial started enrolling. Led by Ronald Crystal at Weill Cornell Medical College, New York, it will inject adeno-associated virus carrying the gene for ApoE2 into patients with early to late-stage AD who inherited two copies of ApoE4. The idea is to flood their brains with the protective allele of this apolipoprotein to try to counteract the effects of the risk allele. AAV-rh10-APOE2 will be injected directly into the subarachnoid cisternae of participants brains. The Phase 1 trialwill recruit 15 patients with biomarker-confirmed AD. Beverly Davidson, Childrens Hospital of Philadelphia, has a similar ApoE2 gene therapy in preclinical development.

At SfN, Abeliovich detailed Prevails programs for forms of PD and for frontotemporal dementias that are caused by risk alleles. A trial has begun for a glucocerebrosidase-based gene therapy. The enzyme GCase is essential for lysosomes to function properly. People who have loss-of-function mutations in both copies of the GBA1 gene develop Gauchers, a lysosomal storage disease. The severest form starts in babies, most of whom die before age 2. Milder forms cause later-onset Gauchers, while heterozygous mutations in GBA1 increase risk for Parkinsons, making restoration of GCase an obvious strategy for PD. Some researchers are trying to develop ways to boost activity of the mutated enzyme (e.g., Oct 2019 news), whereas Abeliovich and colleagues have constructed AAV-9 vectors to deliver normal GBA1 into the brain to restore GCase production.

In preclinical studies, the AAV9-GBA1 construct PR001 rescued both lysosomal and brain function in models of GCase deficiency and of Parkinsons, Abeliovich said. In mice fed the GCase inhibitor conduritol epoxide (CBE), PR001 injected into the brain ventricles beefed up GCase activity and reduced glycolipid accumulation, which is a sign that lysosomes are functional. A single dose worked for at least six months. Similar results were seen in a commonly used model of Gauchers that expresses the V394L GBA mutation and only weakly expresses prosaposin and saposins, lysosomal proteins that metabolize lipids. In these 4L/PS-NA mice, PR001 made increased levels of active GCase, fewer lipids accumulated, and the mice were more mobile on a balance beam. 4L/PS-NA mice also accumulate -synuclein, the major component of Lewy bodies in PD and other synucleinopathies. In these mice, and also in A53T -synuclein mice made worse with CBE, PR001 halved the amount of insoluble -synuclein, Abeliovich reported at SfN.

In search of the right dose for humans, the scientists next turned to nonhuman primates. They injected PR001 into the cisterna magna in hopes AAV9 would broadly distribute throughout the brain. At the highest dose, 8 x 1010 capsids per gram of brain weight, exposure in the brain was similar to that seen in the mice. The virus permeated the spinal cord, frontal cortex, hippocampus, midbrain, and putamen.

Also in October, Prevail scientists began recruiting for a Phase 1/2 double-blind, sham-controlled trial to test this gene therapy in 16 people with moderate to severe PD, who have mutations in one or both copies of their GBA1 genes. Six patients each will receive a low or high dose of PR001A. Blood and CSF biomarkers to be analyzed at three and 12 months, and at follow-up, include GCase, lipids, -synuclein, and neurofilament light chain. Participants will also undergo cognitive, executive, and motor-function tests and brain imaging. A Phase 1/2 trial of PR001 in neuronopathic Gauchers, which affects the brain and spinal cord, will start soon, Abeliovich said.

Other groups are boosting dopamine production in Parkinsons by way of gene therapy. VY-AADC,developed by Voyager Therapeutics, Cambridge, Massachusetts, packages the gene for L-amino acid decarboxylase (AADC), which converts L-dopa into dopamine, in an AAV-2 vector that is delivered into the brain. Two Phase 1 open-label trials are testing safety and efficacy. Both the PD-1101 and PD-1102 trials use MRI to guide injections of the vector bilaterally into the putamina of 15 or 16 patients, respectively. According to preliminary results presented at the annual meeting of the American Academy of Neurology this past May, the virus penetrated half of the putamen and AADC activity, as judged by 18F-DOPA PET, increased by 85 percent in the latter study. Seven of eight treated patients reported improvement after a year, along with longer on time on L-DOPA, and shorter off time. Off time is the period when L-DOPA effects wear off and patients experience loss of motor control. RESTORE-1, a Phase 2 study of 42 patients, started in 2018 and will run to the end of 2020.

Long-Lived Gene Therapy. When a Parkinsons disease patient died eight years after neurturin gene therapy, the trophin was still being expressed in their putamen (top left) and substantia nigra (bottom left), where it corresponded with tyrosine hydroxylase activity (right). [Courtesy of Jeff Kordower.]

Also in PD, Kordower and colleagues plan to re-evaluate neurturin-based gene therapy. Previously, the gene for this neurotrophin was delivered in an AAV2 vector into the brains of Parkinson patients in Phase 1 and 2 trials. This did not improve motor function. Even so, in Chicago Kordower showed that in two patients who died eight and 10 years later, the inserted gene was still expressing neurturin and that dopamine levels were higher on the injected than the contralateral side of the substantia nigra/putamen. This shows us that long-term gene expression can be achieved in the human brain, said Kordower (see image above). He believes that by focusing delivery with ultrasound, or tweaking the capsid itself, he may be able to generate enough gene expression to improve function.

Separately, AAV-GAD, a gene therapy for PD that showed promise in Phase 2 (Mar 2011 news) was acquired by MeiraGTx, New York, which will continue to develop it in the U.S. and Europe, according to founder Samuel Waksal (Nov 2018 news).

For its part, Prevail has a gene transfer construct for frontotemporal dementia in the pipeline, as well. Called PR006, it carries GRN, the gene encoding progranulin, on an AAV9 vector. GRN mutations cause familial FTD and, much like GBA mutations, do their dirty work via lysosomal dysfunction. In Chicago, Abeliovich reported that PR006 boosted progranulin release from neurons derived from FTD-GRN patients, nearly doubling their levels of mature Cathepsin D, the lysosomal protease that chops progranulin into granulins and indicates healthy lysosomes. In progranulin knockout mice, PR006 restored brain GRN expression and progranulin secretion into the CSF. Abeliovich said he expects a Phase 1/2 clinical trial in FTD patients to start in early 2020.

The biotech company Passage Bio, Philadelphia, is planning for clinical trials early next year with its AAV-GRN vector. MeiraGTx, New York, is banking on a different approach for FTD. They have developed an AAV carrying UPF1, which encodes regulator of nonsense transcripts 1. This protein helps clear out aberrant RNAs through a process call nonsense-mediated decay. MeiraGTx hopes this will restore homeostasis to RNA processing. AAV-UPF1 will be trialed for FTD and all forms of ALS bar those caused by mutations in SOD1. For SOD ALS, Novartis, Basel, Switzerland, and REGENXBIO, Rockville, Maryland, have a vector in preclinical testing.

For his part, Svendsen is taking a different approach. His lab tackles ALS with ex vivo gene therapy. The idea is to engineer clinical-grade human stem cells to produce glial-derived growth factor, and inject them into the spinal cord, much like the early NGF studies did in AD. Svendsen hopes the cells will churn out enough of the neurotrophin to protect spinal cord motor neurons. In a Phase 1/2a trial, 18 ALS patients have received these cells into one side of their spinal cords, such that each person serves as his or her own control. If this works, they would regain mobility only on the injected side. The trial finished in October; Svendsen expects results to come out in a few months. In a follow-up study, the scientists are trying to do the same with induced pluripotent stem cells. This would allow them to transplant autologous cells into patients, avoiding immune rejection

Other groups are deploying gene therapy as a way to improve immunotherapy, shield neurons from stress, or even generate neurons from astrocytes to make up for those lost to neurodegeneration.Tom Fagan

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Scientists find a cell that helps tadpoles tails regrow – Vryheid Herald

By daniellenierenberg

Aristotle already observed in the fourth century B.C. that some animals can regrow their tails after losing them, but the mechanisms that support this kind of regeneration remain difficult to understand.

Using single-cell genomics, scientists at the Wellcome Trust / Cancer Research UK Gurdon Institute at the University of Cambridge developed an innovative strategy to show what happens in different tadpole cells when they regenerate their tails.

Recent advances at Cambridge in next-generation single-cell sequencing mean that scientists can now track which genes are turned on throughout a whole organism or tissue, at the resolution of individual cells. This technique, known as single-cell genomics, makes it possible to distinguish between cell types in more detail based on their characteristic selection of active genes.

These groundbreaking discoveries are beginning to reveal a map of cellular identities and lineages, as well as the factors involved in controlling how cells choose between alternative pathways during embryo development to produce the range of cell types in adults.

Using this technology, Can Aztekin and Dr Tom Hiscock under the direction of Dr Jerome Jullien made a detailed analysis of cell types involved in regeneration after damage in African clawed frog tadpoles (Xenopus laevis). Details were published in the journal Science.

Dr Tom Hiscock said: Tadpoles can regenerate their tails throughout their life; but there is a two-day period at a precise stage in development where they lose this ability. We exploited this natural phenomenon to compare the cell types present in tadpoles capable of regeneration and those no longer capable.

The researchers found that the regenerative response of stem cells is orchestrated by a single sub-population of skin cells, which they named Regeneration-Organizing Cells, or ROCs.

Can Aztekin said: Its an astonishing process to watch unfold. After tail amputation, ROCs migrate from the body to the wound and secrete a cocktail of growth factors that coordinate the response of tissue precursor cells. These cells then work together to regenerate a tail of the right size, pattern and cell composition.

In mammals, many tissues such as the skin epidermis, the intestinal epithelium and the blood system, undergo constant turnover through life. Cells lost through exhaustion or damage are replenished by stem cells. However, these specialised cells are usually dedicated to tissue sub-lineages, while the ability to regenerate whole organs and tissues has been lost in all but a minority of tissues such as liver and skin.

Professor Benjamin Simons, a co-author of the study said: Understanding the mechanisms that enable some animals to regenerate whole organs represents a first step in understanding whether a similar phenomenon could be reawakened and harnessed in mammalian tissues, with implications for clinical applications.

This research was funded by the University of Cambridge, the Cambridge Trust andthe Wellcome Trust;and supported by theEuropean Molecular Biology Organization, the Royal Society,theEuropean Molecular Biology Laboratory, and Cancer Research UK.

Source: University of Cambridge Research

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Activation of the Immune System Underlies Cardiac Cell Therapies – The Scientist

By daniellenierenberg

Injections of stem cellseither a patients own or from a donorinto the hearts of people with cardiac conditions has been shown in some cases to improve heart function. How the cells help has been a mystery. A paper in Nature today (November 27) shows that activation of an innate immune response can explain, and even recapitulate, the beneficial effects of stem cell transplants in the hearts of mice.

The findings suggest stem cells may not be required to boost cardiac repair, but some researchers argue that, by finally providing a mechanistic explanation, the study supports the use of cell therapy.

This work is paradigm-shifting because it demonstrates a mechanism to explain a perplexing phenomenon that has intrigued cardiologists as a result of decades of cardiac stem cell trials, writes cardiologist Jonathan Epstein of the University of Pennsylvanias Perelman School of Medicine in an email to The Scientist. Now the focus can shift from injecting cells into the heart to understanding how to modulate the immune system so that heart function is improved, continues Epstein, who was not involved in the study.

The idea of applying stem cells, derived from the bone marrow or elsewhere, to the heart to fix damage caused by myocardial infarction or cardiovascular disease has been the subject of intense pre-clinical and clinical investigations for the best part of two decades, and yet the field is highly controversial. Aside from the retractions of fraudulent papers that misguided the larger heart regeneration community for years, the observed benefits of cell transplant therapies are generally modest and, because the underlying mechanism of repair is unknown, there is a lack of consensus about which of the many types of stem cells and delivery approaches might work best, as well as which types of patients may benefit.

A better knowledge of the mechanism would drive better clinical trial design, says Jeffery Molkentin, a cardiovascular biologist at Cincinnati Children's Hospital Medical Center who led the latest project. Indeed, he says, if mechanistic studies had been done up-front then we would have been much further along in the clinical trials [at this point].

For transplanted cells to produce functional benefits in the heart, its likely the cells would need to remain there after injection. So Molkentins team studied a variety of stem cell types injected into mice to see if any of them ever engrafted in the heart, he says. We had a list of five of the most prominent ones and none of the five ever engrafted, and they were all cleared within less than two weeks and sometimes within five or six days. But, the team did spot something else happening. In all [cases], he says, there was this really noticeable inflammatory response.

The team then showed that whether they injected live stem cells, dead stem cells, or zymosana potent activator of the innate immune systeminto the hearts of mice that had been given an experimental myocardial infarction, functional improvement of the heart occurred. By contrast, an injection of cyclosporinewhich suppresses the innate immune systemafter the cell delivery eliminated the beneficial effects.

The team went on to show that in the injured hearts of mice that received cell therapy or zymosan treatment there was evidence of improved muscle mechanical properties as well as scar remodeling and reduction. Both treatments recruited certain subtypes of macrophages that experiments indicated were driving this remodeling.

A heart attack triggers innate immunity automatically, prompting the essential scarring without which the heart would rupture, says Molkentin. The cell therapy (or zymosan treatment), being delayed by one week, does not exacerbate this initial inflammation, he says, but instead somehow realigns the healing process and makes for a better scar.

It seems like it optimize[s] the properties of the area around the scar and the contractility of that area, Molkentin says, but we dont know exactly why yet. . . . Were trying to figure this out.

Whatever the precise mechanism, the study shows the importance of the immune system, says Paul Riley of the University of Oxford who studies regenerative medicine but was not involved in the research. Its certainly very important for the field to be aware of this [finding], he continues. It will stimulate further interest in targeting or modulating, or thinking about the way the immune response . . . can effect more optimal function and repair after acute myocardial infarction.

If the results hold true in humans, it could have implications for any future trials in which patients might receive immunosuppression to prevent cell rejection, suggests Riley. Although its not thought any such trials are currently underway, according to Molkentin and Joshua Hare, a cardiologist and stem cell researcher at the University of Miami who was not involved in the study, if embryonic stem cells were ever approved for trial they would require immunosuppressives, Hare says.

Hare has been involved in a number of stem cell therapy trials and sees the paper not as evidence that the stem cells themselves arent necessary, but instead as a mechanistic explanation for the fact that they do work. It is often the case in medicine, he says, that once a treatment is in use, we change our perspective on how they work. Fundamentally, he says, we know that the cells are working, and that theyre safe. He therefore thinks the paper supports the field and . . . substantiates that we were on the right track. That said, he adds, If someone takes these findings and comes up with a better approach, a safer approach, a more efficacious approach, thats great.

R.J. Vagnozzi et al., An acute immune response underlies the benefit of cardiac stem-cell therapy,Nature, doi:10.1038/s41586-019-1802-2,2019.

Ruth Williams is a freelance journalist based in Connecticut. Email her atruth@wordsbyruth.comor find her on Twitter @rooph.

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Cell Separation Technology Market Size Projected to Rise Lucratively During 2019 2027 – News Description

By daniellenierenberg

Transparency Market Research (TMR)has published a new report on the globalcell separation technology marketfor the forecast period of 20192027. According to the report, the global cell separation technology market was valued at ~US$ 5 Bnin 2018, and is projected to expand at a double-digit CAGR during the forecast period.

Overview

Cell separation, also known as cell sorting or cell isolation, is the process of removing cells from biological samples such as tissue or whole blood. Cell separation is a powerful technology that assists biological research. Rising incidences of chronic illnesses across the globe are likely to boost the development of regenerative medicines or tissue engineering, which further boosts the adoption of cell separation technologies by researchers.

Expansion of the global cell separation technology market is attributed to an increase in technological advancements and surge in investments in research & development, such asstem cellresearch and cancer research. The rising geriatric population is another factor boosting the need for cell separation technologies Moreover, the geriatric population, globally, is more prone to long-term neurological and other chronic illnesses, which, in turn, is driving research to develop treatment for chronic illnesses. Furthermore, increase in the awareness about innovative technologies, such as microfluidics, fluorescent-activated cells sorting, and magnetic activated cells sorting is expected to propel the global cell separation technology market.

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North America dominated the global cell separation technology market in 2018, and the trend is anticipated to continue during the forecast period. This is attributed to technological advancements in offering cell separation solutions, presence of key players, and increased initiatives by governments for advancing the cell separation process. However, insufficient funding for the development of cell separation technologies is likely to hamper the global cell separation technology market during the forecast period. Asia Pacific is expected to be a highly lucrative market for cell separation technology during the forecast period, owing to improving healthcare infrastructure along with rising investments in research & development in the region.

Rising Incidences of Chronic Diseases, Worldwide, Boosting the Demand for Cell Therapy

Incidences of chronic diseases such as diabetes, obesity, arthritis, cardiac diseases, and cancer are increasing due to sedentary lifestyles, aging population, and increased alcohol consumption and cigarette smoking. According to the World Health Organization (WHO), by 2020, the mortality rate from chronic diseases is expected to reach73%, and in developing counties,70%deaths are estimated to be caused by chronic diseases. Southeast Asia, Eastern Mediterranean, and Africa are expected to be greatly affected by chronic diseases. Thus, the increasing burden of chronic diseases around the world is fuelling the demand for cellular therapies to treat chronic diseases. This, in turn, is driving focus and investments on research to develop effective treatments. Thus, increase in cellular research activities is boosting the global cell separation technology market.

Increase in Geriatric Population Boosting the Demand for Surgeries

The geriatric population is likely to suffer from chronic diseases such as cancer and neurological disorders more than the younger population. Moreover, the geriatric population is increasing at a rapid pace as compared to that of the younger population. Increase in the geriatric population aged above 65 years is projected to drive the incidences of Alzheimers, dementia, cancer, and immune diseases, which, in turn, is anticipated to boost the need for corrective treatment of these disorders. This is estimated to further drive the demand for clinical trials and research that require cell separation products. These factors are likely to boost the global cell separation technology market.

According to the United Nations, the geriatric population aged above 60 is expected to double by 2050 and triple by 2100, an increase from962 millionin 2017 to2.1 billionin 2050 and3.1 billionby 2100.

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Productive Partnerships in Microfluidics Likely to Boost the Cell Separation Technology Market

Technological advancements are prompting companies to innovate in microfluidics cell separation technology. Strategic partnerships and collaborations is an ongoing trend, which is boosting the innovation and development of microfluidics-based products. Governments and stakeholders look upon the potential in single cell separation technology and its analysis, which drives them to invest in the development ofmicrofluidics. Companies are striving to build a platform by utilizing their expertise and experience to further offer enhanced solutions to end users.

Stem Cell Research to Account for a Prominent Share

Stem cell is a prominent cell therapy utilized in the development of regenerative medicine, which is employed in the replacement of tissues or organs, rather than treating them. Thus, stem cell accounted for a prominent share of the global market. The geriatric population is likely to increase at a rapid pace as compared to the adult population, by 2030, which is likely to attract the use of stem cell therapy for treatment. Stem cells require considerably higher number of clinical trials, which is likely to drive the demand for cell separation technology, globally. Rising stem cell research is likely to attract government and private funding, which, in turn, is estimated to offer significant opportunity for stem cell therapies.

Biotechnology & Pharmaceuticals Companies to Dominate the Market

The number of biotechnology companies operating across the globe is rising, especially in developing countries. Pharmaceutical companies are likely to use cells separation techniques to develop drugs and continue contributing through innovation. Growing research in stem cell has prompted companies to own large separate units to boost the same. Thus, advancements in developing drugs and treatments, such as CAR-T through cell separation technologies, are likely to drive the segment.

As per research, 449 public biotech companies operate in the U.S., which is expected to boost the biotechnology & pharmaceutical companies segment. In developing countries such as China, China Food and Drug Administration(CFDA) reforms pave the way for innovation to further boost biotechnology & pharmaceutical companies in the country.

Global Cell Separation Technology Market: Prominent Regions

North America to Dominate Global Market, While Asia Pacific to Offer Significant Opportunity

In terms of region, the global cell separation technology market has been segmented into five major regions: North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa. North America dominated the global market in 2018, followed by Europe. North America accounted for a major share of the global cell separation technology market in 2018, owing to the development of cell separation advanced technologies, well-defined regulatory framework, and initiatives by governments in the region to further encourage the research industry. The U.S. is a major investor in stem cell research, which accelerates the development of regenerative medicines for the treatment of various long-term illnesses.

The cell separation technology market in Asia Pacific is projected to expand at a high CAGR from 2019 to 2027. This can be attributed to an increase in healthcare expenditure and large patient population, especially in countries such as India and China. Rising medical tourism in the region and technological advancements are likely to drive the cell separation technology market in the region.

Launching Innovative Products, and Acquisitions & Collaborations by Key Players Driving Global Cell Separation Technology Market

The global cell separation technology market is highly competitive in terms of number of players. Key players operating in the global cell separation technology market include Akadeum Life Sciences, STEMCELL Technologies, Inc., BD, Bio-Rad Laboratories, Inc., Miltenyi Biotech, 10X Genomics, Thermo Fisher Scientific, Inc., Zeiss, GE Healthcare Life Sciences, PerkinElmer, Inc., and QIAGEN.

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Phase 2 Trial Data on ALS NurOwn Therapy, Supporting Safety And Early Efficacy, Published – ALS News Today

By daniellenierenberg

NurOwn showed a good safety profile, as well as potential efficacy in a Phase 2 clinical trial that included people with rapidly progressing amyotrophic lateral sclerosis (ALS).

Trial results, which have been previously reported, have now been published in the journalNeurology in a paper titled, NurOwn, phase 2, randomized, clinical trial in patients with ALS.

NurOwn, which is being developed by BrainStorm Cell Therapeutics, is a stem cell-based therapy. It involves taking mesenchymal stem cells (MSCs), a type of cell capable of differentiating into other cell types, from a person.

These MSCs are modified so they produce more neurotrophic factors (NTFs) compounds that help drive the growth and survival of nervous tissue. The cells are then re-introduced to the body by injection into muscles and/or the spinal canal (termed intramuscular and intrathecal injection, respectively).

In the Phase 2 trial (NCT02017912), which was funded by BrainStorm, 48 people with ALS were enrolled; 36 were treated with NurOwn, and 12 were given a placebo. Participants received the treatment after a three-month pretransplant period, and were followed for six months after treatment.

The participants were predominantly (72.9%) male, and their average age was 51.1 years.

The studys primary goal of the study was to evaluate safety, measured by number of patients with adverse events to treatment, and this goal was met. The use of NurOwn was found to be safe and well-tolerated.

Eleven participants nine in the treatment group and two in the placebo group developed 16 serious adverse events (SAEs).

All treatment-emergent SAEs [those that occurred after start of treatment] were deemed to be related to ALS disease progression, and none was considered possibly, probably, or definitely related to study treatment, the researchers noted.

Data were also analyzed for early indications of treatment efficacy. Researchers specifically looked at rate of disease progression, as measured by the slope (that is, the change over time) in scores on the Revised ALS Functional Rating Scale (ALSFRS-R).

Overall, these rates were not significantly different between the NurOwn-treated and placebo groups.

However, in a subset of 21 patients with particularly rapid disease progression (15 given NurOwn and six a placebo), the average rate of disease progression showed a significantly improvement at two weeks (+3.3 vs. 1.3) and four weeks (+2.0 vs. 0.1) following treatment for those that got NurOwn.

Rapid progressors were defined in this study, at enrollment, as those with a decline of more than 2 points in ALSFRS-R scores during the pretreatment period.

This positive trend continued for all study time points, but it was not statistically significant after four weeks.

The researchers also looked at the proportion of patients with an improvement of at least 1.5 points each month, based on the reasoning that, responder analyses may more accurately capture individual treatment responses than changes in mean slope alone. That is, because each individual with ALS is different, some might be more likely to respond to treatment than others.

At four weeks post-treatment, a significantly greater proportion of those given NurOwn compared to placebo met this responder threshold (47% vs. 9%). In the rapid progression group, there were significant differences at both week four and week twelve (80% vs. 0%, and 53% vs. 0%, respectively).

For all of the above efficacy measurements, the greatest response was seen shortly following the injection, with decreasing response over time. This may suggest the need for repeated treatments to maintain a sustained therapeutic effect, the researchers wrote.

Cerebrospinal fluid (CSF) was collected just before and two weeks after the injection. Analysis of this fluid, broadly, showed an increase in levels of NTFs and a decrease in inflammatory markers, which suggests that NurOwn works as intended. (CSFfluid surrounds the brain and spinal cord.)

Specifically, the levels of monocyte chemoattractant protein-1 (MCP-1), a marker of immune cell infiltration and neuroinflammation, were significantly lower post-treatment in patients given NurOwn, while no significant change was observed in the placebo group. This correlated with ALSFRS-R slope improvement at all time points.

[W]e observed a clear biological effect of the treatment on CSF biomarkers to support its proposed mechanism of action in ALS, Robert H. Brown Jr., PhD, MD, of the University of Massachusetts Medical School and a study co-author, said in a BrainStorm press release.

We met our primary endpoint and demonstrated that a single dose of NurOwn was safe and well-tolerated while supporting NurOwns mechanism of action on neuroprotection and neuroinflammation pathways in ALS, added Ralph Kern, MD, MHSc, chief operating officer and chief medical officer of BrainStorm.

We look forward to completing the current Phase 3 study to confirm the promising Phase 2 findings and expand our understanding of the potential of MSC-NTF cell therapy in ALS, Kern added.

A fully enrolled, placebo-controlled Phase 3 study (NCT03280056) is underway in the U.S. in 200 ALS patients, and a secondary safety analysis found no new concerns. The trial is expected to conclude in late 2020, with results announced shortly thereafter.

Results from the [Phase 2] study underscore the importance of conducting a larger Phase 3 clinical trial that will build upon the data collected in our Phase 2 study, said Chaim Lebovits, Brainstorms president and chief executive officer. We look forward to reporting our clinical results in the scientific literature and through corporate announcements.

Marisa holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.

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Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

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Stem Cells Market 2019 Global Growth Analysis and Forecast Report by 2025 – Markets Gazette 24

By daniellenierenberg

New York, November 26, 2019: The global stem cells market is expected to grow at an incredible CAGR of 25.5% from 2018to 2024and reach a market value of US$ 467 billion by 2024. The emergence of Induced Pluripotent Stem (iPS) cells as an alternative to ESCs (embryonic stem cells), growth of developing markets, and evolution of new stem cell therapies represent promising growth opportunities for leading players in this sector.

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Due to the increased funding from Government and Private sector and rising global awareness about stem cell therapies and research are the main factors which are driving this market. A surge in therapeutic research activities funded by governments across the world has immensely propelled the global stem cells market. However, the high cost of stem cell treatment and stringent government regulations against the harvesting of stem cells are expected to restrain the growth of the global stem cells market.

This report will definitely help you make well informed decisions related to the stem cell market. The stem cell therapy market includes large number of players that are involved in development of stem cell therapies of the treatment of various diseases. Mesoblast Ltd. (Australia), Aastrom Biosciences, Inc. (U.S.), Celgene Corporation (U.S.), and StemCells, Inc. (U.S.) are the key players involved in the development of stem cell therapies across the globe.

This market research report categorizes the stem cell therapy market into the following segments and sub-segments:

The Global Stem Cell Market this market is segmented on the basis of Mode of Therapy, Therapeutic Applications and Geography.

By Mode of Therapy this market is segmented on the basis of Allogeneic Stem Cell Therapy Market and Autologous Stem Cell Therapy Market. Allogeneic Stem Cell Therapy Market this market is segmented on the basis of CVS Diseases, CNS Diseases, GIT diseases, Eye Diseases, Musculoskeletal Disorders, Metabolic Diseases, Immune System Diseases, Wounds and Injuries and Others. Autologous Stem Cell Therapy Market this market is segmented on the basis of GIT Diseases, Musculoskeletal Disorders, CVS Diseases, CNS Diseases, Wounds and Injuries and Others. By Therapeutic Applications this market is segmented on the basis of Musculoskeletal Disorders, Metabolic Diseases, Immune System Diseases, GIT Diseases, Eye Diseases, CVS Diseases, CNS Diseases, Wounds and Injuries and Others.

By Regional Analysis this market is segmented on the basis of North America, Europe, Asia-Pacific and Rest of the World.

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Table of Contents

1 INTRODUCTION

2 Research Methodology

2.1 Research Data2.1.1 Secondary Data2.1.1.1 Key Data From Secondary Sources2.1.2 Primary Data2.1.2.1 Key Data From Primary Sources2.1.2.2 Breakdown of Primaries2.2 Market Size Estimation2.2.1 Bottom-Up Approach2.2.2 Top-Down Approach2.3 Market Breakdown and Data Triangulation2.4 Research Assumptions

3 Executive Summary

4 Premium Insights

5 Market Overview

6 Industry Insights

7 Global Stem Cell Therapy Market, By Type

8 Global Stem Cell Therapy Market, By Therapeutic Application

9 Global Stem Cell Therapy Market, By Cell Source

10 Stem Cell Therapy Market, By Region

11 Competitive Landscape

12 Company Profiles

12.1 Introduction

12.1.1 Geographic Benchmarking

12.2 Osiris Therapeutics, Inc.

12.3 Medipost Co., Ltd.

12.4 Anterogen Co., Ltd.

12.5 Pharmicell Co., Ltd.

12.6 Holostem Terapie Avanzate Srl

12.7 JCR Pharmaceuticals Co., Ltd.

12.8 Nuvasive, Inc.

12.9 RTI Surgical, Inc.

12.10 Allosource

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Stem Cells Market 2019 Global Growth Analysis and Forecast Report by 2025 - Markets Gazette 24

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New Link Discovered Between Cells That Burn Fat and Colon Cancer – Clinical OMICs News

By daniellenierenberg

A new study by Rutgers University researchers suggests that two genes expressed in the intestinal cells that line the inside of the colon may also be involved in cancer development.

Recent studies have shown that intestinal stem cells can increase in animals on a high fat Western diet, potentially explaining an elevated cancer risk from such a diet.Diet being able to control cell proliferation is an interesting research development, particularly the convergence of dietary factors and dysregulated gene signaling driving malignant transformations and promoting an adenoma-to-adenocarcinoma progression.

This new study suggests a novel connection between HNF4A and HNF4G genes, diet and cancer.Genetic expression of HNF4 has previously been shown by to be heavily influenced by the gut microbiota, which in turn can influence a multitude of intestinal disorders.

Non-host gene regulation was further explored in this study by using a high fat diet to test how these genes work, and the researchers discovered they help co-regulate stem cell proliferation, as well as help intestine cells burn dietary fat. This was done by isolating cells from knockout and control mice and observing intestine stem cell proliferation under conditions of high fat and control. Mice that had both HNF4A and HNF4G knocked out were unable to have their stem cells proliferate under high fat conditions.

Intestinal stem cells undergo constant renewal and fuel the continuous turnover of the lining of the intestine. People naturally lose millions of intestinal cells daily, much like they lose skin cells. If this rate of replication is not closely controlled, it can quickly lead to malignancy. Lack of proliferation can be very problematic for the colon and damaging to lower layers of cells.

This [research] is important because scientists have shown that when theres too much dietary fat in the intestine, stem cell numbers increase, boosting susceptibility to colon cancer, said senior author Michael Verzi, an associate professor in the Department of Genetics in the School of Arts and Sciences at Rutgers UniversityNew Brunswick.

Rutgers scientists believe HNF4A and HNF4G help stem cells burn fat, providing them energy. By linking gene activation, cell replication number, diet and cancer risk, scientists might be able to better understand the cancer development process in high risk patients. Going forward, the researchers plan to continue studying whether these two genes alter stem cell numbers and cancer risk alongside a high fat diet, said Verzi.

Colorectal cancer (of the colon or rectum) is the third most common cancer diagnosed in both men and women in the United States. According to the American Cancer Society, over 100,000 Americans will be diagnosed with colon cancer this year. This cancer is also the second most deadliest in the United States, but due to a combination of increased screening and heightened awareness the death rate has been dropping. However, in patients under the age of 55, the death rate of colon cancer has increased each year by 1% since 2007. Approximately 50,000 colon cancer patients are expected to die in 2019.

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New Link Discovered Between Cells That Burn Fat and Colon Cancer - Clinical OMICs News

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