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.

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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.

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.

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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Stem Cell Assay Market Analysis On Trends & Need 2025 - Health News Office

Spinal Cord Stem Cells Comments Off on Stem Cell Assay Market Analysis On Trends & Need 2025 – Health News Office | October 25th, 2019

Whether youve been recently diagnosed with multiple sclerosis (MS) or have been living with the condition for a while, chances are youll sometimes hear terms from your healthcare team that are new to you.

The following is a quick, alphabetical guide to the terminology you may need to know as you manage your condition:

Ankle-Foot Orthosis (AFO) A brace designed to support the position of the foot and motion of the ankle to compensate for nerve damage and muscle weakness in the area caused by MS and other movement disorders. An AFO is typically used to stabilize weak limbs or to reposition a limb with contracted muscles into a more normal position.

Autoimmune Disease Your immune system plays a major part of your bodys defense against bacteria and viruses by sending out cells to attack them once they enter your body. However, if you have an autoimmune disease, your immune system mistakenly attacks healthy cells in your body, causing them to weaken or break down. MS is thought to be just one example of an autoimmune disease. It has been suggested that in MS, your immune system may mistakenly attack the cells in your central nervous system.

Axon Long threadlike structures of nerve cells that send impulses to other cells in your body. Research suggests that damage to or loss of these fibers in progressive MS may be linked to worsening disability and more severe progression.

Central Nervous System (CNS) The group of organs in your body that includes the brain, spinal cord, and optic nerves. If you have MS, your bodys immune system may be working against the CNS, producing neurological symptoms such as muscle weakness and vision problems.

Cerebrospinal Fluid (CSF) A clear, colorless liquid that surrounds the brain and spinal cord to protect the CNS and assist in the circulation of nutrients and removal of waste products. In MS, damage to the myelin sheath of nerve cells causes certain types of proteins to be released into the spinal fluid. The presence of these proteins in the CSF, but not in the blood, may point to a diagnosis of MS.

Clinically Isolated Syndrome (CIS) A first episode of neurologic symptoms that lasts at least 24 hours and is caused by inflammation or demyelination (loss of the myelin that covers the nerve cells) in the CNS. People who experience CIS may or may not go on to develop MS. However, when CIS is accompanied by magnetic resonance imaging (MRI)detected brain lesions similar to those found in MS, you have a 60 to 80 percent chance of a second neurologic event and diagnosis of MS within several years, according to the National MS Society.

Cog Fog A commonly used term that refers to the cognitive changes experienced by many people with MS. According to MS Australia, approximately 50 percent of people with the condition will develop some degree of cog fog, or inhibited ability to think, reason, concentrate, or remember. For some, cognitive problems will become severe enough to interfere in a significant way with daily activities.

Corticosteroids (or Steroids) Prescription medication used to treat relapses in relapsing-remitting MS. Your doctor may prescribe intravenous (IV) corticosteroids if the symptoms of your relapse are causing significant problems, like poor vision or difficulty walking. These drugs work by suppressing the immune system and reducing inflammation in the CNS, and they may help relapse symptoms resolve more quickly. But they wont affect your ultimate level of recovery from a relapse or the long-term course of your MS. Methylprednisolone is a commonly used corticosteroid in MS.

Diplopia (or Double Vision) An eye problem in which you see two images of a single object. It may be present when only one eye is open (monocular) or disappear when either eye is closed (binocular). Diplopia is a common symptom of MS, and it occurs because of damage to the optic nerve.

Disease-Modifying Therapies (DMTs) Drugs designed to reduce new relapses, delay progression of disability, and limit new CNS inflammation in people with MS. Although there are multiple DMTs that have been approved by the U.S. Food and Drug Administration (FDA) for use in MS, these drugs generally work by reducing inflammation in nerve cells in theCNS.

Dysarthria A speech disorder caused by neuromuscular impairment and resulting in disturbances in motor control of the muscles used in speech. Its believed the demyelinating lesions in MS may result in spasticity, weakness, slowness, or ataxic incoordination of the lips, tongue, mandible, soft palate, vocal cords, and diaphragm, causing this speech impairment.

Dysphagia (Difficulty Swallowing) A condition that may occur in people with MS, leading to difficulty in eating solid foods or liquids, frequent throat clearing during eating or drinking, a feeling that food is stuck in the throat, or coughing or a choking sensation when eating or drinking. Its the result of nerve damage within the muscles that control swallowing.

Epstein-Barr Virus (EBV) A virus believed to be a possible cause or trigger for MS. Although the exact cause of MS remains unknown, researchers suggest an infectious agent may be involved in its development. Studies have found that antibodies (immune proteins that indicate a person has been exposed to a given virus) to EBV are significantly higher in people who eventually develop MS than in those who dont. Other research has noted that people with a specific immune-related gene and high levels of antibodies to EBV in their blood are 9 times more likely to develop MS than others.

Evoked Potentials A test that measures the speed of nerve messages along sensory nerves to the brain, which can be detected on your scalp using electrodes attached with sticky pads. Its sometimes used in the diagnosis of MS, because nerve damage can slow down the transmission of nerve signals. Evoked potential tests can indicate nerve pathways that are damaged prior to the onset of MS symptoms.

Exacerbation An occurrence of new symptoms or the worsening of old symptoms that may also be referred to as a relapse, attack, or flare-up. Exacerbations can be very mild, or severe enough to interfere with a person's ability to perform day-to-day activities.

Expanded Disability Status Scale (EDSS) A scale used for measuring MS disability and monitoring changes in the level of disability over time. Developed by neurologist John Kurtzke, MD, in 1983, the EDSS scale ranges from 0 to 10 in 0.5-unit increments (scoring is based on a neurological exam) and relies on walking as its main measure of disability. People with an EDSS of 1 have no disability and minimal loss of function, while those with an EDSS of 9.5 are confined to bed and totally dependent on others for functions of daily living.

Foot Drop (or Drop Foot) A symptom of MS caused by weakness in the ankle or disruption in the nerve pathway between the legs and the brain, making it difficult to lift the front of the foot to the correct angle during walking. If you have foot drop, your foot hangs down and may catch or drag along the ground, resulting in trips and falls. Foot drop can be managed with an AFO or other treatments.

Hematopoietic Stem Cell Transplantation (HSCT) A procedure designed to reboot the immune system, the National MS Society says, using hematopoietic (blood cellproducing) stem cells derived from a persons own bone marrow or blood. If your doctor recommends HSCT, youll undergo a chemotherapy regimen before these cells are reintroduced to the body via IV injection, where they will migrate to your bone marrow to rebuild the immune system.

John Cunningham (JC) Virus A common infection completely unrelated to MS that is found in as many as 90 percent of people, according to the UK's MS Trust. JC virus has no symptoms and is normally controlled by the immune system. However, if your immune system is weakened, the JC virus can reactivate, causing potentially fatal inflammation and damage to the brain known as progressive multifocal leukoencephalopathy (PML). Certain MS disease-modifying therapies have been linked with increased risk for PML.

Lhermittes Sign An electric shock-like sensation experienced by some with MS when the neck is moved in a particular way. The sensation can travel down to the spine, arms, and legs.

Lesion (or Plaque) Refers to an area of damage or scarring (sclerosis) in the CNS caused by inflammation in MS. These lesions can be spotted on an MRI scan, with active lesions appearing as white patches. With regular MRIs, a neurologist can tell how active your MS is.

Lumbar Puncture (or Spinal Tap) A procedure used for the collection of cerebrospinal fluid (CSF), sometimes done to help diagnose MS. For this procedure, your doctor will ask you to lie on your side or bend forward while seated, before cleansing an area of your lower back and injecting a local anesthetic. He will then insert a hollow needle and extract a small amount of spinal fluid using a syringe.

Magnetic Resonance Imaging (MRI) The diagnostic tool that currently offers the most sensitive noninvasive way of imaging the brain, spinal cord, or other areas of the body, according to the National MS Society. Its the preferred imaging method for diagnosis of MS and to monitor the course of the disease. MRI uses magnetic fields and radio waves to measure the relative water content in tissues, which is notable in MS because the layer of myelin that protects nerve cell fibers is fatty and repels water. In areas where myelin has been damaged by MS, fat is stripped away and the tissue holds more water. This shows up on an MRI as a bright white spot or darkened area, depending on how the images are made.

McDonald Criteria A guidance used in the diagnosis of MS, authored by an international panel of experts on the condition, originally in 2010. The guidance was updated in 2017. Among the key changes: advising for the use of brain MRI as part of the diagnostic process.

MS Hug A common symptom of MS. If you experience the MS hug, you may feel like you have a tight band around your chest or ribs, or pressure on one side of your torso. Some people find that it is painful to breathe. The MS hug can last for seconds, minutes, hours, or even longer.

Myelin A substance rich in lipids (fatty substances) and proteins that helps form the myelin sheath. In MS, particularly relapsing-remitting MS, an abnormal immune response produces inflammation in the CNS, effectively attacking the myelin in the cells.

Myelin Sheath An insulating layer of fatty substances and proteins that forms around the nerves in body, including those in the CNS. It allows electrical impulses to transmit quickly and efficiently along the nerve cells, but these impulses can be slowed if the sheath is damaged, causing MS.

Neurodegeneration Refers to the process by which the myelin sheath of cells in the CNS is damaged in MS. Its believed to be a major contributor to neurological disability in the condition, and may be the reason immune modulation treatments (disease-modifying therapy) are generally less effective in the progressive MS than in the relapsing-remitting MS.

Neurologist The point person for monitoring your MS treatment and managing MS symptoms. This specialist typically focuses on conditions affecting the CNS.

Neuropathic Pain A type of pain common in MS that results from changes or damage to the myelin sheath and the axons, or nerve fibers, it normally covers. MS-caused neuropathic pain may be chronic, intermittent, or occur only in response to a stimulus.

Neuropsychologist A specialist you may be referred to who helps you manage the cognitive effects of MS. Neuropsychological testing (or testing of the functioning of your brain) involves identifying memory or learning difficulties associated with MS. Cognitive rehabilitation may improve functioning.

Nociceptive Pain Caused by damage to muscles and joints, it can be either acute or chronic, and may not result from MS itself, but be caused by changes in posture or walking or the overuse of assistive devices in those with the condition.

Nystagmus A common eye abnormality in MS, its characterized by involuntary, rhythmic, back-and-forth motion of the eyeball, either horizontally or vertically. For those with nystagmus, the perception of the rhythmic movement of the surrounding stationary world (oscillopsia) can be disorienting and disabling.

Oligoclonal Bands (OCBs) Immunoglobulins, or proteins, that collect in blood plasma or cerebrospinal fluid (CSF). Although not every person with MS has OCBs, their presence can support a diagnosis of MS. Having OCBs is generally associated with a younger age of MS onset and a poorer prognosis.

Optic Neuritis An inflammatory condition that damages the optic nerve, a bundle of nerve fibers that transmits visual information from your eye to your brain, causing pain and temporary vision loss in one eye. Its been linked with nerve damage resulting from MS, and may be among the first symptoms a person with the condition experiences.

Pseudobulbar Affect (PBA) A neurologic effect experienced by roughly 10 percent of people with MS as well as some with Parkinsons disease or amyotrophic lateral sclerosis (ALS), according to the Multiple Sclerosis Association of America (MSAA). Its characterized by sudden, uncontrollable expressions of laughter or crying without an obvious cause, which can be distressing as well as embarrassing to those who experience it. PBA is believed to be a mood disorder related to the disruption of nerve impulses in the CNS, but its different from depression, which is also common in MS.

Pseudoexacerbation A temporary worsening of symptoms without actual myelin inflammation or damage. It is often triggered by other illnesses or infection, exercise, a warm environment, depression, exhaustion, and stress. Urinary tract infection (UTI) is the most common type of infection to cause a pseudoexacerbation.

Sclerosis A general hardening of the body tissue. The term multiple sclerosis refers to the multiple areas of scar tissue often called lesions that develop along affected nerve fibers and that are visible in MRI scans.

Spasticity A symptom of MS that causes your muscles to feel stiff, heavy, or difficult to move. When a muscle spasms, youll experience a sudden stiffening that may cause a limb to jerk. This may be painful.

Trigeminal Neuralgia (or Tic Douloureux) A type of neuropathic pain that occurs on the face (usually on one side only). Its a known symptom of MS, and you may experience it in your cheek; upper or lower jaw; inside the mouth; or in the area around your eyes, ears, or forehead. In MS, its typically caused by damage to the myelin sheath around the trigeminal nerve, which among other functions controls the muscles used in chewing. The condition is triggered by everyday activities, like tensing facial muscles while shaving or when chewing.

Vertigo An intense sensation of the surrounding environment spinning around one. In MS, vertigo is typically caused by growth of an existing lesion or development of a new lesion on the brain stem or cerebellum, the area in the brain that controls balance. It can also be a symptom of a problem with the inner ear, or it can be side effect of medication used to treat MS or other health conditions you may have.

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Speaking Multiple Sclerosis: A Glossary of Common Terms - Everyday Health

Spinal Cord Stem Cells Comments Off on Speaking Multiple Sclerosis: A Glossary of Common Terms – Everyday Health | October 25th, 2019

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Children, suffering from DMD, usually die of cardio-respiratory failure. Represtational image

Duchenne Muscular Dystrophy (DMD) is a deadly genetic disorder, 99.9 per cent people suffering from which, die between the age of 13 to 23 years. However, in a first, a 26-year-old patient from Lucknow has survived DMD by regularly taking stem cells for the last five years.

Children, suffering from DMD, usually die of cardio-respiratory failure. But with the stem cell therapy, this patient has not lost muscle power in last five years and heart and lung muscles and the upper half of the body are working well.

Dr. B.S Rajput, the surgeon who is treating this patient, said, "DMD is a type of muscular dystrophy and being a genetic disorder, it is very difficult to treat. Autologous (from your own body) bone marrow cell transplant or stem cell therapy in such cases was started in Mumbai about 10 years back.

Dr Rajput, who was recently appointed as visiting professor at GSVM Medical College, Kanpur, said he has treated several hundred DMD patients and recently this combination protocol was published in the international Journal of Embryology and stem cell research.

The patient's father is elated that his son has maintained well with this treatment and now has even started earning by working on computers.

According to Dr Rajput, this disease is endemic in eastern UP, especially Azamgarh, Jaunpur, Ballia and some of the adjoining districts of Bihar, and one out of every 3,500 male child, suffers from the disease.

Yet the disease is not given as much attention as it should be.

Dr Rajput, who is consultant bone cancer and stem cell transplant surgeon from Mumbai, said though patients in Uttar Pradesh and Bihar get financial support from the Chief Minister's Relief Funds, the treatment of autologous bone marrow cell transplant is not included in the package list of Ayushman Bharat scheme, which deprives many from getting the treatment.

The doctor further informed that efforts are being made to establish the department of regenerative medicine in the medical college, where bone marrow cell transplant and stem cell therapy would be done even for other intractable problems like spinal cord injury, arthritis knee and motor neurone disease.

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In a first, 26-year-old DMD patient in UP survives with stem cell therapy - India TV News

Spinal Cord Stem Cells Comments Off on In a first, 26-year-old DMD patient in UP survives with stem cell therapy – India TV News | October 25th, 2019

Researchers have developed a precise and non-toxic nanoscale technology that can deliver oncology drugs directly to cancer cells. The minuscule tubes are called peptoids.

The research was led by Yuehe Lin, professor at Washington State Universitys School of Mechanical and Materials Engineering and Chun-Long Chen, senior research scientist at the Department of Energys Pacific Northwest National Laboratory (PNNL) and joint faculty member at University of Washington. The study was published in the journal Small.

The peptoids are about a thousand times thinner than a human hair. The researchers took the nanotubes, which were inspired by biological models, and rolled them into nanosheet membranes. They were then able to use a variety of drugs, fluorescent dyes and cancer-targeting molecules and place them into the nanotubes, which allowed them to track the drug delivery.

The two drugs they used were a chemotherapy agent and a less-invasive photodynamic therapy. Photodynamic therapeutic compounds release reactive oxygen species (ROS) that kill cancer cells when exposed to light. The combination therapy allowed the researchers to use lower doses of the chemotherapeutic, which decreased the toxicity.

By precisely engineering these nanotubes with fluorescent dyes and cancer targeting molecules, scientists can clearly locate tumor cells and track how the drug regimen is performing, said Lin. We can also track how nanotubes enter and deliver the drugs inside the cancer cell.

They evaluated the peptoids on lung cancer cells. The chemotherapy drug was doxorubicin. The system delivered the drug directly to the cancer cells, which resulted in what it describes as highly efficient cancer killing, all while using much lower doses of doxorubicin.

This is a promising approach for precision targeting with little damage to healthy surrounding cells, Lin said.

What is new about the research is the use of the peptoids. Other research has been conducted using carbon nanotubes and other nanomaterials, but there are toxicity issues. They also werent as effective at precisely recognizing molecules.

By using these peptoids, we were able to develop highly programmable nanotubes and a biocompatible delivery mechanism, Chen said. We also harnessed the high stability of peptoid and its well-controlled packing to develop nanotubes that are highly stable.

Research into nanotechnology is making progress, although its not clear just how much of it, if any, is making it into clinical applications. In August, researchers at Rutgers University-New Brunswick published research about a nanotechnology platform that helps identify what happens to specific stem cells.

Stem cells are key building blocks that can differentiate into all the different types of cells in the body, including brain cells and heart cells and skin cells. Increasingly, researchers are utilizing adult human-induced pluripotent stem cells (iPSCs) to develop drugs and work on therapies.

The researchers monitored the creation of neurons from human stem cells by identifying next-generation biomarkers called exosomes. Exosomes are particles released by cells and they play a critical function in cell-to-cell communication.

One of the major hurdles in the current cell-based therapies is the destructive nature of the standard cell characterization step, stated senior author KiBum Lee, professor in the Department of Chemistry and Chemical Biology. With our technology, we can sensitively and accurately characterize the cells without compromising their viabilities.

The technology platform utilizes minuscule nanotubes for sensing. Specifically, the authors reported using a multifunctional magneto-plasmonic nanorid (NR)-based detection platform.

Researchers at Texas Heart Institute (THI) recently used bio-compatible nanotubes invented at Rice University to restore electrical function to damaged hearts.

Instead of shocking and defibrillating, we are actually correcting diseased conduction of the largest major pumping chamber of the heart by creating a bridge to bypass and conduct over a scarred area of a damaged heart, stated Mehdi Razavi, a cardiologist and director of Electrophysiology Clinical Research and Innovations at THI. Razavi co-led the study with Matteo Pasquali, a chemical and biomolecular engineer at Rice University.

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More Breakthroughs in Nanotechnology Could Lead to Improvements in Drug Delivery and Medicine - BioSpace

Skin Stem Cells Comments Off on More Breakthroughs in Nanotechnology Could Lead to Improvements in Drug Delivery and Medicine – BioSpace | October 23rd, 2019

Imagine youre a smiley-faced, feathery-gilled Mexican salamander called an axolotl. Youve just been born, along with hundreds of brothers and sisters. But salamanders like you live in the wild only in one lake near Mexico City, and that habitat isnt big enough for all of you. Theres not enough food. Only the strongest can survive. What do you do?

If youre an axolotl, you have two choiceseat your siblings arms, or have your arms eaten.

But even if you are the unfortunate victim of this sibling violence, not all hope is lost. In a few months, youll grow a whole new armbones, muscle, skin, nerves and all.

Its pretty gruesome, but cannibalism is a possible reason why they grow their arms back, says associate biology professor James Monaghan. His lab studies regeneration in axolotls, a peculiar species that can grow back limbs and other organs to various degrees.

When an injury occurs, some cues are released in that animal that tells cells near the injury to go from a resting state into a regenerative state, Monaghan says.

His lab is trying to figure out what those cues are, and how we might induce that response in humans, who have very limited regenerative abilities.

Humans are notoriously bad at regenerating, Monaghan says. After were done growing, the genes that tell our cells to grow new organs are turned off.

Thats a good thing because otherwise itd be chaos, he says. No one wants to spontaneously grow an extra finger.

Axolotls can turn back on those genes that we turn off permanently, Monaghan says.

Understanding the specific mechanisms that induce regenerative responses in axolotls is no small task since axolotls have the largest genome ever sequenced.

So far, the lab has identified one molecule, neuregulin-1, which is essential for regeneration of limbs, lungs, and possibly hearts.

When we removed it, regeneration stopped. And when we added it back in, it induced the regenerative response, Monaghan says. Im not saying its a golden bullet for inducing regeneration in humans, too, but it could be part of the puzzle.

A lot of researchers study limb regeneration in axolotls. But Monaghans lab is interested in extending this research to other organs, as well.

When you think of the human condition, most of our issues with disease are with internal organs, Monaghan says.

Take retina regeneration, for example. Monaghan says we can either learn the process axolotls undergo that allows their specialized cells to return back to developmental cells, and then mimic that process in human eyes. Or, we can learn which elements of the axolotl enable their cells to behave this way, and then add those elements to human stem cell therapy.

To test the latter, Monaghan has teamed up with a Northeastern associate professor of chemical engineering, Rebecca Carrier, and her lab to figure out the best way to transplant mammalian retinal cells using molecules found in the axolotl.

In the experiment, Monaghan and Carrier used pig eyes, which are similar to human eyes. When they transplanted stem cells from the retina of one pig into the retina of another, 99 percent of the transplanted cells died. Somethings missing, Monaghan says. The cells dont have the right cues.

But when Carrier and Monaghan injected those same pig stem cells into the axolotl eye, fewer cells died. They were much happier, Monaghan says. Theres something in the axolotl retina that the mammalian cells like.

One reason axolotls are so good at receiving transplants is because, unlike humans, they dont have a learned immune system, meaning they cant distinguish between themselves and foreign entities.

Its really easy to do grafts between animals because the axolotls cant tell that the new tissue isnt theirs, he says. They dont reject it like we might.

An obvious example of this can be seen in axolotls that are genetically modified with a green fluorescent protein found in jellyfish. These naturally white axolotls glow neon green in certain lighting.

With this we can ask really basic questions, like do cells change their fate when they participate in regeneration? Monaghan says.

For example, if Monaghan grafts muscle tissue from a green fluorescent animal onto a white axolotl and then that axolotl regenerates, does the axolotl grow green muscle? Do its bones glow green, too? What about its skin?

Researchers have found, however, that cells dont actually change. Green muscle yields green muscle only.

The axolotl isnt the only animal that can regrow organs. Starfish, worms, frogs, and other species of salamanders can also regenerate. But axolotls are special because, unlike other animals, they can regrow organs that are just as robust as the originals, no matter how old they get.

For example, tadpoles can regenerate limbs. But once they undergo metamorphosis and become frogs, they can only regrow a spike, Monaghan says. They lose the ability to grow back their digits.

The axolotls ability to fully regrow organs, even as it ages, could be partially due to its perpetual juvenile state. Axolotls, unlike most other amphibians, dont undergo metamorphosis naturally, which means they never technically reach adulthood, even though they can reproduce. This condition is called neoteny.

Axolotls come from a species that used to walk on land, Monaghan says. They do have legs, after all. But some mutation occurred that keeps them in the lake and from reaching adulthood.

To test whether their neotenic state is responsible for their ability to regenerate, Monaghan took a group of axolotl siblings and induced metamorphosis in one half by exposing them to thyroid hormones, a chemical that flips on the maturity switch in these amphibians. The other half was kept in the juvenile state.

In the experiment, the juveniles regenerated normally, but all of their adult siblings regenerated slower than usual, and had deformities in their regrown limbs.

There is some association with neoteny and the ability to regenerate, Monaghan says. But its not the main factor.

That main factor is yet to be discovered. But even though some of this might sound like science fiction, you already made an arm once, Monaghan says. If we could just learn how to turn back on those programs, our bodies might do the rest of the work.

For media inquiries, please contact media@northeastern.edu.

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Meet the axolotl: A cannibalistic salamander that regenerates its limbs and might help us better understand human stem cell therapy -...

Skin Stem Cells Comments Off on Meet the axolotl: A cannibalistic salamander that regenerates its limbs and might help us better understand human stem cell therapy -… | October 23rd, 2019

CRANBURY, N.J.--(BUSINESS WIRE)--As a part of a fundraising effort by Moving Mountains for Multiple Myeloma (MM4MM), 13 individuals will traverse Patagonias awe-inspiring and incredible landscape from Nov. 9-19. MM4MM is a joint initiative between the Multiple Myeloma Research Foundation (MMRF), CURE Media Group and Celgene. The upcoming climb includes survivors, caregivers, family members, myeloma doctors and team members from the organizing partners.

Since MM4MM began with its first climb in 2016, the program has raised over $2.7 million. All the funds raised go directly to the MMRF to accelerate new treatment options for patients with multiple myeloma.

As a patient founded organization, the MMRF stands together with those who are battling multiple myeloma patients, families, physicians, researchers, and our pharmaceutical partners. This team represents a microcosm of that myeloma community and demonstrates that together, we can collaborate with ever increasing momentum towards a cure, said Paul Giusti, CEO of the Multiple Myeloma Research Foundation. We are thrilled to enter the fifth year of this inspiring program and to have Celgene join us in this effort to raise awareness and critical funds to continue our mission.

The MM4MM team will include four patients living with multiple myeloma:

We are so honored to be a part of yet another hike with the MMRF and Celgene, said Mike Hennessy Jr., president and CEO of MJH Life Sciences, parent company of CURE magazine. This initiative organized by Moving Mountains for Multiple Myeloma not only raises awareness and research funding for multiple myeloma but has brought together the myeloma community to take action and fight for a cure for myeloma patients.

The team will embark on a five-day trek of a lifetime through Patagonia and take on the rewarding and beautiful landscape that includes glaciers, deep valleys and challenging peaks. During this trek, the team will travel through El Chaltn and acclimatize while they experience the mighty range of peaks dominated by Monte Fitz Roy, an 11,020-foot tower with a sheer face of more than 6,000 feet. Next, the team will reach Lago San Martin, where they will traverse the terrain in daily treks, exploring a 10-mile peninsula, climbing to a condor rookery and reaching remote Andean lakes.

Celgene, Cure and the MMRF share an unwavering commitment to improving the lives of patients with multiple myeloma and we are very proud to continue our role in the Moving Mountains for Multiple Myeloma initiative, said Chad Saward, senior director, patient advocacy at Celgene Corp. We are amazed and inspired by all who are participating in this unique awareness program.

To learn more about MM4MM and to donate to multiple myeloma research, click here.

About Moving Mountains for Multiple Myeloma

Moving Mountains for Multiple Myeloma (MM4MM) is a collaboration between CURE Media Group and the Multiple Myeloma Research Foundation (MMRF) to raise awareness and funds for myeloma research. This year, Celgene Corporation and GSK join the effort as sponsors. In addition to Patagonia, the program also led hikes up Mt. Washington and through Iceland in 2019. To date, MM4MM has raised over $2.7 million for myeloma research and included 51 patients with multiple myeloma on 7 climbs. Funds raised go directly to research, supporting the MMRF mission. For more information, visit https://www.themmrf.org/events/.

About Multiple Myeloma

Multiple myeloma (MM) is a cancer of the plasma cell. It is the second most common blood cancer. An estimated 32,110 adults (18,130 men and 13,980 women) in the United States will be diagnosed with MM in 2019 and an estimated 12,960 people are predicted to die from the disease. The five-year survival rate for MM is approximately 50.7%, versus 31% in 1999.

About the Multiple Myeloma Research Foundation

A pioneer in precision medicine, the Multiple Myeloma Research Foundation (MMRF) seeks to find a cure for all multiple myeloma patients by relentlessly pursuing innovations that accelerate the development of precision treatments for cancer. Founded in 1998 by Kathy Giusti, a multiple myeloma patient, and her twin sister Karen Andrews as a 501(c)(3) nonprofit organization, the MMRF has created the business model around cancerfrom data to analytics to the clinic. The MMRF identifies barriers and then finds the solutions to overcome them, bringing in the best partners and aligning incentives in the industry to drive better outcomes for patients. Since its inception, the organization has collected thousands of samples and tissues, opened nearly 100 trials, helped bring 10 FDA-approved therapies to market, and built CoMMpass, the single largest genomic dataset for any cancer. Today, the MMRF is building on its legacy in genomics and is expanding into immune-oncology, as the combination of these two fields will be critical to making precision medicine possible for all patients. The MMRF has raised nearly $500 million and directs nearly 90% of the total funds to research and related programs. To learn more, visit http://www.themmrf.org.

About CURE Media Group

CURE Media Group is the leading resource for cancer updates, research and education. It combines a full suite of media products, including its industry-leading website, CUREtoday.com; innovative video programs, such as CURE Connections; a series of widely attended live events; and CURE magazine, which reaches over 1 million readers, as well as the dynamic website for oncology nurses, OncNursingNews.com, and its companion publication, Oncology Nursing News. CURE Media Group is a brand of MJH Life Sciences, the largest privately held, independent, full-service medical media company in the U.S. dedicated to delivering trusted health care news across multiple channels.

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Multiple Myeloma Experts, Patients, Advocates and Caregivers Team Up to Hike Through Patagonia - Business Wire

Skin Stem Cells Comments Off on Multiple Myeloma Experts, Patients, Advocates and Caregivers Team Up to Hike Through Patagonia – Business Wire | October 23rd, 2019

The Eastland Companion Animal Hospital in Bloomington is asking dog owners if they want to participate in research on using stem cells to treat dogs with arthritis.

Local dogs wouldjoin a double-blind, placebo-controlled studyto show the effectiveness of stem cells in treating large dogs(70 pounds or more) with arthritis in up to two joints of the knee, hip, elbow, or shoulder. The veterinary clinic has partnered with Animal Cell Therapies, who it's worked with before, to bring this study to Bloomington.

Dr. Kathy Petrucci, founder and CEO of Animal Cell Therapies, explained how dogs will receive the treatment.

The dogs that will receive the stem cells will be sedated, Petrucci said. Depending on what joints are affected, they will receive up to two injections in the joint and they will also receive an IV dose of stem cells.

The FDA oversees the cells that are received from donors for the study. Mothers donating these cells are screened for diseases, and cells are tested for any infections to ensure safety.

Stem cell therapy has been controversial, especially related to humans.

I think a lot of the controversy comes from the misunderstanding of the cell types, Petrucci said. The research in stem cells first started centered around embryonic or fetal tissue use. Its controversial to use embryos and fetal tissues for treatment for anything. The fact that we are using a disposable tissue as our cell sources makes it not controversial at all.

Why Umbilical-Derived Cells

Petrucci explained why umbilical-derived cells are more effective in treating arthritis versus other sources.

We looked at fat, bone marrow, embryonic cells, Petrucci said. The embryonic cells are a lot more unpredictable, and the bone marrow cells are more difficult to work with and less predictable. We didnt think the fat cells are as potent as umbilical-derived cells. Umbilical-derived cells are a lot younger and theyre a little bit more predictable. They are more easy to collect. We obtain cells from donors when the tissue would be normally thrown away. Theres no surgery required, no extra biopsies to obtain fat, no bone marrow from research animals. Its a good, ethical source of stem cells.

Umbilical-derived stem cells have proven successful in past studies on treatment for arthritis, according to Petrucci.

We did a study at the University of Florida on elbows only and we had success with that study, Petrucci said. We had good success with dogs under 70 pounds and (less) success with dogs over 70 pounds, so we changed our dose, which is why were testing dogs 70 pounds and over in this study.

Criteria for eligibility includes dogs weighing 70 pounds or more, being one year of age or older, in general good health, no neurologic issues, arthritis in up to two joints of the knee, hip, elbow, or shoulder, and have all four functioning limbs.

Owners must bring their dogs back to the clinic after 30 days to check for progress and complete a questionnaire. About 50 to 100 dogs are expected to participate in the study.

People like you value experienced, knowledgeable and award-winning journalism that covers meaningful stories in Bloomington-Normal. To support more stories and interviews like this one,please consider making a contribution.

Stem Cell Treatment - Excerpt

Stem Cell Research - Full Story

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Bloomington Vet Joins Study For Stem Cell Therapy To Treat Dogs With Arthritis - WGLT News

Bone Marrow Stem Cells Comments Off on Bloomington Vet Joins Study For Stem Cell Therapy To Treat Dogs With Arthritis – WGLT News | October 23rd, 2019

United Therapeutics received a building permit Tuesday for a $9.5 million build-out of its cell therapy facility on the second floor of Mayo Clinics Discovery and Innovation Building.

The 21,843-square-foot space will house an automated stem cell manufacturing site, which is one of the first of its kind in the country. The Whiting-Turner Contracting Co. is the project contractor.

The technology, approved by the FDA in 2018, allows the Mayo Clinic Center for Regenerative Medicine to produce cells from the bone marrow of a stem cell donor in large enough quantities to be used as treatments in clinical trials. It allows for the treatment of multiple patients at the same time.

Construction began in 2017 on the $32.4 million building at 14221 Kendall Hench Drive. It held a grand opening in August.

The first floor houses three ex-vivo lung perfusion surgical suites used for lung restoration, another form of regenerative medicine. It turns donor lungs, which previously would have previously been unusable, into viable transplant organs. United Therapeutics also collaborates with Mayo Clinic on lung restoration.

The third floor houses the Life Sciences Incubator for biotech entrepreneurs, which offers coworking space, wet labs, business resources, networking and entrepreneurial training.

Read more:
United Therapeutics receives permit for cell therapy facility build-out at Mayo - Jacksonville Daily Record

Bone Marrow Stem Cells Comments Off on United Therapeutics receives permit for cell therapy facility build-out at Mayo – Jacksonville Daily Record | October 23rd, 2019

MELVILLE, N.Y., Oct. 23, 2019 (GLOBE NEWSWIRE) -- BioRestorative Therapies, Inc. (the Company") (BRTX), a life sciences company focused on stem cell-based therapies, today announced that the Australia Patent Office has issued a Certificate of Grant for the Companys patent application titled Human Brown Adipose Derived Stem Cells and Uses.

This is the second patent issued for the Companys brown fat technology in Australia and adds to three other patents related to BioRestoratives metabolic program (ThermoStem Program) that have previously been issued to the Company in the United States and other countries.

This patent will allow for the protection of a specific isolated human brown adipose tissue stem cell line capable of differentiating into multiple cell types. This particular cell line possesses strong characteristics applicable for potential therapeutic uses for treating a wide range of degenerative and metabolic disorders, including diabetes, hypertension, cardiac deficiency and obesity.

This patent, granted by the Australian Patent Office for our metabolic program, adds to our growing family of IP surrounding and protecting our brown fat metabolic cell program, said Mark Weinreb, CEO of BioRestorative Therapies. In particular, our invention relates to an isolated brown fat stem cell line that we expect to be used in our development of cell-based therapies to treat metabolic disorders.

About BioRestorative Therapies, Inc.

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

Disc/Spine Program (brtxDISC): Our lead cell therapy candidate, BRTX-100, is a product formulated from autologous (or a persons own) cultured mesenchymal stem cells collected from the patients bone marrow. We intend that the product will be used for the non-surgical treatment of painful lumbosacral disc disorders. The BRTX-100 production process utilizes proprietary technology and involves collecting a patients bone marrow, isolating and culturing stem cells from the bone marrow and cryopreserving the cells. In an outpatient procedure, BRTX-100 is to be injected by a physician into the patients damaged disc. The treatment is intended for patients whose pain has not been alleviated by non-invasive procedures and who potentially face the prospect of surgery. We have received authorization from the Food and Drug Administration to commence a Phase 2 clinical trial using BRTX-100 to treat persistent lower back pain due to painful degenerative discs.

Metabolic Program (ThermoStem): We are developing a cell-based therapy to target obesity and metabolic disorders using brown adipose (fat) derived stem cells to generate brown adipose tissue (BAT). BAT is intended to mimic naturally occurring brown adipose depots that regulate metabolic homeostasis in humans. Initial preclinical research indicates that increased amounts of brown fat in the body may be responsible for additional caloric burning as well as reduced glucose and lipid levels. Researchers have found that people with higher levels of brown fat may have a reduced risk for obesity and diabetes.

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Forward-Looking Statements

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

CONTACT:Email: ir@biorestorative.com

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BioRestorative Therapies Receives A Second Patent in Australia For Its Metabolic Program - Yahoo Finance

Cardiac Stem Cells Comments Off on BioRestorative Therapies Receives A Second Patent in Australia For Its Metabolic Program – Yahoo Finance | October 23rd, 2019

Reading Time: 2 minutesLucknow:

Duchenne Muscular Dystrophy (DMD) is a deadly genetic disorder, 99.9 per cent people suffering from which, die between the age of 13 to 23 years. However, in a first, a 26-year-old patient from Lucknow has survived DMD by regularly taking stem cells for the last five years.

Children, suffering from DMD, usually die of cardio-respiratory failure. But with the stem cell therapy, this patient has not lost muscle power in the last five years and heart and lung muscles and the upper half of the body are working well.

Dr B.S Rajput, the surgeon who is treating this patient, said, DMD is a type of muscular dystrophy and being a genetic disorder, it is very difficult to treat. Autologous (from your own body) bone marrow cell transplant or stem cell therapy in such cases was started in Mumbai about 10 years back.

Dr Rajput, who was recently appointed as visiting professor at GSVM Medical College, Kanpur, said he has treated several hundred DMD patients and recently this combination protocol was published in the International Journal of Embryology and stem cell research.

The patients father is elated that his son has maintained well with this treatment and now has even started earning by working on computers.

According to Dr Rajput, this disease is endemic in eastern UP, especially Azamgarh, Jaunpur, Ballia and some of the adjoining districts of Bihar, and one out of every 3,500 male child, suffers from the disease.

Yet the disease is not given as much attention as it should be.

Dr Rajput, who is consultant bone cancer and stem cell transplant surgeon from Mumbai, said though patients in Uttar Pradesh and Bihar get financial support from the Chief Ministers Relief Funds, the treatment of autologous bone marrow cell transplant is not included in the package list of Ayushman Bharat scheme, which deprives many from getting the treatment.

The doctor further informed that efforts are being made to establish the department of regenerative medicine in the medical college, where bone marrow cell transplant and stem cell therapy would be done even for other intractable problems like spinal cord injury, arthritis knee and motor neurone disease.

Here is the original post:
In a first, 26-yr-old DMD patient in UP survives with stem cell therapy - The Woke Journal

Spinal Cord Stem Cells Comments Off on In a first, 26-yr-old DMD patient in UP survives with stem cell therapy – The Woke Journal | October 23rd, 2019

Analysts expect Lineage Cell Therapeutics, Inc. (NYSEAMERICAN:LCTX) to report $-0.05 EPS on November, 14.They anticipate $0.02 EPS change or 28.57 % from last quarters $-0.07 EPS. After having $-0.06 EPS previously, Lineage Cell Therapeutics, Inc.s analysts see -16.67 % EPS growth. The stock decreased 0.81% or $0.0079 during the last trading session, reaching $0.9699. About shares traded. Lineage Cell Therapeutics, Inc. (NYSEAMERICAN:LCTX) has 0.00% since October 22, 2018 and is . It has by 0.00% the S&P500.

Lineage Cell Therapeutics, Inc., a clinical-stage biotechnology company, focuses on developing and commercializing therapies for the treatment of degenerative diseases in the United States and internationally. The company has market cap of $145.14 million. The company's lead product candidates include OpRegen, a retinal pigment epithelium cell replacement therapy, which is in Phase I/IIa multicenter trial for the treatment of the dry age-related macular degeneration; OPC1, an oligodendrocyte progenitor cell therapy that is in Phase I/IIa multicenter clinical trial for the treatment of acute spinal cord injuries; and VAC2, an allogeneic cancer immunotherapy of antigen-presenting dendritic cells, which is in Phase I clinical trial to treat non-small cell lung cancer. It has a 4.18 P/E ratio. It also develops Renevia, a facial aesthetics product that is in pivotal clinical trial for the treatment of HIV-associated facial lipoatrophy; ReGlyde, a HyStem product in preclinical development as a device for viscosupplementation, and as a platform for intraarticular drug delivery in osteoarthritis; and Premvia, a Hystem Hydrogel product, as well as develop bone grafting products for the orthopedic diseases and injuries.

More notable recent Lineage Cell Therapeutics, Inc. (NYSEAMERICAN:LCTX) news were published by: Businesswire.com which released: Lineage Cell Therapeutics Conducts Sale of Shares in OncoCyte Corporation Business Wire on September 11, 2019, also Businesswire.com with their article: Lineage Cell Therapeutics Announces Issuance of U.S. Patent for Method of Treating Spinal Cord Injury With Stem Cell-Derived Oligodendrocyte Progenitor Cells Business Wire published on September 04, 2019, Finance.Yahoo.com published: Lineage Cell Therapeutics to Present at 2019 Cantor Global Healthcare Conference on October 4, 2019 Yahoo Finance on September 26, 2019. More interesting news about Lineage Cell Therapeutics, Inc. (NYSEAMERICAN:LCTX) were released by: Businesswire.com and their article: Lineage Cell Therapeutics to Present at H.C. Wainwright 21st Annual Global Investment Conference on September 9, 2019 Business Wire published on September 05, 2019 as well as Finance.Yahoo.coms news article titled: Lineage Cell Therapeutics to Present at 2019 Cell & Gene Meeting on the Mesa on October 3, 2019 Yahoo Finance with publication date: September 23, 2019.

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$-0.05 EPS Expected for Lineage Cell Therapeutics, Inc. (LCTX) - The Lamp News

Spinal Cord Stem Cells Comments Off on $-0.05 EPS Expected for Lineage Cell Therapeutics, Inc. (LCTX) – The Lamp News | October 23rd, 2019

CALGARY, Alberta, Oct. 21, 2019 (GLOBE NEWSWIRE) -- Hemostemix Inc. (Hemostemix or the Company) (TSX VENTURE: HEM; OTCQB: HMTXF), a biotechnology company developing and commercializing blood-derived stem cell therapies for unmet medical conditions, is pleased to provide a summary of the presentation entitled Autologous Stem Cell Treatment for CLI Patients with No Revascularization Options: An Update of the Hemostemix ACP-01 Trial With 4.5 Year Followup. Lead investigator Dr. York Hsiang, Professor of Vascular Surgery, University of British Columbia gave this update at the 41st Annual Canadian Society for Vascular Surgery Meeting on September 14, 2019.

Dr. Hsiang reported on the blinded results from the long-term follow-up of the first cohort of patients enrolled at two trial sites, Vancouver Coastal Health Research Institute (VCHRI) located in Vancouver, BC, led by principal investigator, Dr. York N. Hsiang, MB, ChB, MHSc, FRCSC and University Health Network, Peter Monk Cardiac Centre located in Toronto, Ontario, led by principal investigator Dr. Thomas Lindsay, MDCM, MSc, FRCSC, FACS.

Following is a summary of the results and conclusion:

In addition, the Companys Data Safety Monitoring Board (DSMB) recently met to review patient safety data in the ongoing Phase II clinical trial for CLI. The DSMB did not find safety concerns with ACP-01 and recommended continuing to enroll patients in the trial. The clinical trial is ongoing at 13 clinical sites in the US and Canada, with several additional sites in the process of being initiated. To date, 46 of the planned 95 patients have been enrolled and treated in the study.

We are very pleased with these blinded long term follow up results, and the recommendation of the DSMB, which are consistent with the findings reported in our two previous published studies of ACP-01 in CLI patients, said Dr. Alan Jacobs, President and Chief Medical Officer of Hemostemix. Patients with critical limb ischemia face a high rate of amputation when revascularization treatment options are exhausted, so seeing this level of improvement, and outcomes maintained for up to 4.5 years after treatment, is extremely encouraging.

ABOUT HEMOSTEMIX INC.

Hemostemix is a publicly traded clinical-stage biotechnology company that develops and commercializes innovative blood-derived cell therapies for medical conditions not adequately addressed by current treatments. It is one of the first clinical-stage biotech companies to test a stem-cell therapy in an international, multicenter, Phase II clinical trial for patients with critical limb ischemia (CLI), a severe form of peripheral artery disease (PAD) caused by reduced blood flow to the legs. The Phase II trial targets a participants diseased tissue with proprietary cells grown from his or her blood that can support the formation of new blood vessels. The Companys intellectual property portfolio includes over 50 patents issued or pending throughout the world. Hemostemix has a manufacturing contract with Aspire Health Science, LLC (Aspire), for the production of ACP-01 and for research and development purposes at Aspires Orlando, Florida, facility. Building towards commercialization, Hemostemix has also licensed the use, sale and import of ACP-01 for certain indications to Aspire in certain jurisdictions. The Company is continuing research and development of its lead product, ACP-01 with other applications, including cardiovascular, neurological and vascular indications.

For more information, please visit http://www.hemostemix.comor email office@hemostemix.com.

Contact:

Kyle Makofka, CEOSuite 2150, 300 5th Avenue S.W.Calgary, Alberta T2P 3C4Phone: (403) 506-3373E-Mail: kmakofka@hemostemix.com

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

Forward-Looking Statements

This release may contain forward-looking statements. Forward-looking statements are statements that are not historical facts and are generally, but not always, identified by the words expects, plans, anticipates, believes, intends, estimates, projects, potential, and similar expressions, or that events or conditions will, would, may, could, or should occur. Although Hemostemix believes the expectations expressed in such forward-looking statements are based on reasonable assumptions, such statements are not guarantees of future performance and actual results may differ materially from those in forward-looking statements. Forward-looking statements are based on the beliefs, estimates, and opinions of Hemostemix management on the date such statements were made. By their nature forward-looking statements are subject to known and unknown risks, uncertainties, and other factors which may cause actual results, events or developments to be materially different from any future results, events or developments expressed or implied by such forward-looking statements. Such factors include, but are not limited to, the Companys stage of development, future clinical trial results, long-term capital requirements and future ability to fund operations, future developments in the Companys markets and the markets in which it expects to compete, risks associated with its strategic alliances and the impact of entering new markets on the Companys operations. Each factor should be considered carefully and readers are cautioned not to place undue reliance on such forward-looking statements. Hemostemix expressly disclaims any intention or obligation to update or revise any forward-looking statements whether as a result of new information, future events, or otherwise.

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Hemostemix Announces Positive Results and Conclusions Reported in Phase II CLI Trial Abstract - Yahoo Finance

Cardiac Stem Cells Comments Off on Hemostemix Announces Positive Results and Conclusions Reported in Phase II CLI Trial Abstract – Yahoo Finance | October 22nd, 2019

WHIPPANY, N.J., Oct. 21, 2019 /PRNewswire/ -- Bayer today announced recipients of the inaugural Pulmonary Hypertension Accelerated Bayer (PHAB) Awards, a U.S.-based research grant program created to support clinical research in pulmonary hypertension (PH), with a focus on pulmonary arterial hypertension (PAH) and chronic thromboembolic pulmonary hypertension (CTEPH). The recipients will receive a combined total of $1 million in grants over a two-year period, making the PHAB Awards one of the largest industry-funded grant programs focused on PAH and CTEPH in the U.S. The eight award recipients were formally announced at a ceremony during the American College of Chest Physicians (CHEST) Annual Meeting in New Orleans on Sunday, October 20, 2019.

"Supporting a new generation of researchers is imperative to ensure we continue the progress that has been made during the past decade in pulmonary hypertension and its related conditions," said Aleksandra Vlajnic, M.D., Senior Vice President & Head Medical Affairs Americas at Bayer. "Our hope is that the PHAB Awards program will encourage researchers to think creatively about solving the significant treatment and patient care challenges that remain, knowing Bayer is committed to providing the support needed to help bring those ideas to fruition. We want to congratulate all of the applicants on their winning proposals."

The recipients are:

The PHAB Awards recipients were selected by an independent Grants Review Committee, consisting of the following eminent PH leaders:

"I would like to thank and recognize the Grants Review Committee for their time and commitment, and the PH community in the U.S. for their overwhelming response to the inaugural PHAB Awards," said Sameer Bansilal, M.D., M.S., Medical Director, U.S. Medical Affairs at Bayer. "We look forward to an even greater response next year and encourage eligible applicants to start thinking about submitting their research proposals."

The PHAB Award eligibility, review and category criteria were modeled after the National Institutes of Health (NIH) system; entries were graded on significance, investigator(s), innovation, approach, and environment.

For more information on the PHAB Awards visit: https://www.phab-awards.com/awards/or e-mail PHAB.awards@bayer.com.

Grants were made on the merits of the research, and research must be posted on ClinicalTrials.gov. Every effort should be made to publish or present study outcomes. If the research is not conducted the grant must be returned.

About Pulmonary Arterial Hypertension (PAH)Pulmonary Arterial Hypertension (PAH, WHO Group 1) is defined by elevated pressure in the arteries going from the right side of the heart to the lungs. Typical symptoms of PAH include shortness of breath on exertion, fatigue, weakness, chest pain and syncope. PAH is caused by abnormalities in the walls of the pulmonary arteries.1,2

About Chronic Thromboembolic Pulmonary Hypertension (CTEPH)Chronic Thromboembolic Pulmonary Hypertension (CTEPH, WHO Group 4) is a progressive type of pulmonary hypertension, in which it is believed that thromboembolic occlusion (organized blood clots) of pulmonary vessels gradually lead to an increased blood pressure in the pulmonary arteries, resulting in an overload of the right heart.3,4 CTEPH may evolve after prior episodes of acute pulmonary embolism, but the pathogenesis is not yet completely understood. The standard and potentially curative treatment for CTEPH is pulmonary thromboendarterectomy (PTE), a surgical procedure in which the blood vessels of the lungs are cleared of clot and scar material.5,6 However, a considerable number of patients with CTEPH (20%-40%) are not operable and in up to 35 percent of patients, the disease persists or reoccurs after PTE.7

About BayerBayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to benefit people by supporting efforts to overcome the major challenges presented by a growing and aging global population. At the same time, the Group aims to increase its earning power and create value through innovation and growth. Bayer is committed to the principles of sustainable development, and the Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2018, the Group employed around 117,000 people and had sales of 39.6 billion euros. Capital expenditures amounted to 2.6 billion euros, R&D expenses to 5.2 billion euros. For more information, go to http://www.bayer.us.

Our online press service is just a click away: http://www.bayer.us/en/newsroomFollow us on Facebook: http://www.facebook.com/pharma.bayer Follow us on Twitter: https://twitter.com/Bayerus

Media Contact:David Patti, +1-973-452-6793Bayer, U.S. Product Communications david.patti@bayer.com

Forward-Looking StatementsThis release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer's public reports which are available on the Bayer website at http://www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.

References:1Galie et al. 2015 ESC/ERS Guidelines for the diagnosis and treatment of pulmonary hypertension. Eur Heart. 2016;37:67119.2American Lung Association. Pulmonary Hypertension. Accessed November 22, 2017. http://www.lung.org/lung-health-and-diseases/lung-disease-lookup/pulmonary-hypertension.3Piazza G and Goldhaber SZ. Chronic thromboembolic pulmonary hypertension. N Engl J Med. 2011; 364: 351-360.4 Simonneau G et al. Updated Clinical Classification of Pulmonary Hypertension. Journal of the American College of Cardiology. 2013; 62(25):5 D'Armini M. Diagnostic advances and opportunities in chronic thromboembolic pulmonary hypertension. Eur Respir Rev. 2015; 24: 253262.6 Kim et al. Chronic thromboembolic pulmonary hypertension. J Am Coll Cardiol. 2013; 62: D92-9.7 Mathai et al. Quality of life in patients with chronic thromboembolic pulmonary hypertension. Eur Respir J. 2016 Aug; 48(2): 526537.

SOURCE Bayer

http://www.bayer.us

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Bayer Announces Recipients of the Pulmonary Hypertension Accelerated Bayer (PHAB) Awards at CHEST Annual Meeting 2019 - PRNewswire

Cardiac Stem Cells Comments Off on Bayer Announces Recipients of the Pulmonary Hypertension Accelerated Bayer (PHAB) Awards at CHEST Annual Meeting 2019 – PRNewswire | October 22nd, 2019

In a first, 26-yr-old DMD patient in UP survives with stem cell therapy

Lucknow, Oct 22 (IANS) Duchenne Muscular Dystrophy (DMD) is a deadly genetic disorder, 99.9 per cent people suffering from which, die between the age of 13 to 23 years. However, in a first, a 26-year-old patient from Lucknow has survived DMD by regularly taking stem cells for the last five years.

Children, suffering from DMD, usually die of cardio-respiratory failure. But with the stem cell therapy, this patient has not lost muscle power in last five years and heart and lung muscles and the upper half of the body are working well.

Dr. B.S Rajput, the surgeon who is treating this patient, said, "DMD is a type of muscular dystrophy and being a genetic disorder, it is very difficult to treat. Autologous (from your own body) bone marrow cell transplant or stem cell therapy in such cases was started in Mumbai about 10 years back.

Dr Rajput, who was recently appointed as visiting professor at GSVM Medical College, Kanpur, said he has treated several hundred DMD patients and recently this combination protocol was published in the international Journal of Embryology and stem cell research.

The patient''s father is elated that his son has maintained well with this treatment and now has even started earning by working on computers.

According to Dr Rajput, this disease is endemic in eastern UP, especially Azamgarh, Jaunpur, Ballia and some of the adjoining districts of Bihar, and one out of every 3,500 male child, suffers from the disease.

Yet the disease is not given as much attention as it should be.

Dr Rajput, who is consultant bone cancer and stem cell transplant surgeon from Mumbai, said though patients in Uttar Pradesh and Bihar get financial support from the Chief Minister''s Relief Funds, the treatment of autologous bone marrow cell transplant is not included in the package list of Ayushman Bharat scheme, which deprives many from getting the treatment.

The doctor further informed that efforts are being made to establish the department of regenerative medicine in the medical college, where bone marrow cell transplant and stem cell therapy would be done even for other intractable problems like spinal cord injury, arthritis knee and motor neurone disease.

--IANS

amita/rtp

Disclaimer :- This story has not been edited by Outlook staff and is auto-generated from news agency feeds. Source: IANS

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In a first, 26-yr-old DMD patient in UP survives with stem cell therapy - Outlook India

Spinal Cord Stem Cells Comments Off on In a first, 26-yr-old DMD patient in UP survives with stem cell therapy – Outlook India | October 22nd, 2019

Lucknow: Duchenne Muscular Dystrophy (DMD) is a deadly genetic disorder, 99.9 per cent people suffering from which, die between the age of 13 to 23 years. However, in a first, a 26-year-old patient from Lucknow has survived DMD by regularly taking stem cells for the last five years.

Children, suffering from DMD, usually die of cardio-respiratory failure. But with the stem cell therapy, this patient has not lost muscle power in last five years and heart and lung muscles and the upper half of the body are working well.

Dr. B.S Rajput, the surgeon who is treating this patient, said, "DMD is a type of muscular dystrophy and being a genetic disorder, it is very difficult to treat. Autologous (from your own body) bone marrow cell transplant or stem cell therapy in such cases was started in Mumbai about 10 years back.

Dr Rajput, who was recently appointed as visiting professor at GSVM Medical College, Kanpur, said he has treated several hundred DMD patients and recently this combination protocol was published in the international Journal of Embryology and stem cell research.

According to Dr Rajput, this disease is endemic in eastern UP, especially Azamgarh, Jaunpur, Ballia and some of the adjoining districts of Bihar, and one out of every 3,500 male child, suffers from the disease.

Yet the disease is not given as much attention as it should be.

Dr Rajput, who is consultant bone cancer and stem cell transplant surgeon from Mumbai, said though patients in Uttar Pradesh and Bihar get financial support from the Chief Minister's Relief Funds, the treatment of autologous bone marrow cell transplant is not included in the package list of Ayushman Bharat scheme, which deprives many from getting the treatment.

The doctor further informed that efforts are being made to establish the department of regenerative medicine in the medical college, where bone marrow cell transplant and stem cell therapy would be done even for other intractable problems like spinal cord injury, arthritis knee and motor neurone disease.

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Lucknow: In a first, 26-yr-old DMD patient in UP survives with stem cell therapy - ETHealthworld.com

Bone Marrow Stem Cells Comments Off on Lucknow: In a first, 26-yr-old DMD patient in UP survives with stem cell therapy – ETHealthworld.com | October 22nd, 2019

NewsReal LifeLydia Burgess-Gamble has helped a sick woman in her twenties after they were genetically matched

Tuesday, 22nd October 2019, 10:38 am

Lydia Burgess-Gamble felt a lump in her throat when she got the letter saying her stem cells were a match for someone sick.

The 42-year-old had signed up to the register to be a donor almost three years earlier and hadn't given it much thought since.

Ahead of her 40th birthday, she'd wanted to do something altrusistic. Now she had the chance to potentially save someone's life who was battling a blood cancer or blood disorder.

Donating stem cells today is almost as easy as giving blood. "It was a straightforward and painless process and being able to relax and read a book for a few hours was a luxury," she said.

Patients face difficult odds

Every 20 minutes someone in the UK is diagnosed with a blood cancer, such as leukaemia, myeloma or lymphoma. That's more than 30,000 people a year. Worldwide, it's one every 30 seconds.

Patients suffering with these types of cancers can have their bone marrow damaged by the cancer itself, or from chemotherapy and radiation treatments. A stem cell transplant lets the new stem cells take over from the damaged marrow so the body can produce healthy, cancer-free blood cells.

Even though there are over 27 million people on the worldwide register, this isnt enough, according to charity DKMS. At any one time there are around 2,000 people in the UK in need of a transplant.

Matching donors and patients isnt easy because it's determined by tissue type, not blood group. There are thousands of different human leukocyte antigen (HLA) characteristics, in millions of combinations. Doctors look to relatives for a match but two out of three of those in need are unable to find one, and so must rely on the generosity of strangers.

Most donations are day cases at hospital

Lydia, an environmental research scientist from Brighton, became aware of the process involved through a Facebook post. "A friend shared an appeal for a loved one who needed a donor," she said. "I remember watching a documentary about donating bone marrow in the 90s and I hadn't realised it mainly doesn't involve an invasive procedure until I read this post."

The donation usually involves a nonsurgical procedure called peripheral blood stem cell (PBSC) donation for around 90 per cent of all cases, which is the method Lydia used.

With this method, blood is taken from one of the donors arms and a machine extracts the blood stem cells from it. The donors blood is then returned to their body through their other arm. It is an outpatient procedure that usually takes four to six hours.

'I had no side effects, other than I felt a little more tired than usual the next day'

Lydia Burgess-Gamble

This procedure doesn't "deplete" a donor's supply of stem cells, as a donor's stem cells will completely replenish themselves within two to four weeks afterwards.

"I had no side effects, other than I felt a little more tired than usual the next day but within 24 hours I was completely back to normal," said Lydia.

"All I know about my recipient is that it's a woman in her twenties who lives in Turkey. I'd love to make contact one day. I'm not expecting anything but I'm hoping she gets well and we may be able to meet."

The other 10 per cent of donations are made through bone marrow, where donors give cells from the bone marrow in their pelvis. This is under general anaesthetic so that no pain is experienced. The collection itself takes one to two hours and most donors return to their regular activities within a week. Two weeks after donation, your bone marrow will have recovered fully, and the hip bone will have fully healed within six weeks.

Donating: the process

To become a potential blood stem cell donor first check your eligibility on the DKMS website and request a swab kit for your cheek.

Complete the swabs posted to you at home and send them back. Then yourtissue type will be analysed and your details will be added to the UK stem cell registry. Your details can be searched for a genetic match for people all over the world who need a second chance at life.

The odds are you may never be called upon, but if you are, you will have a blood test at your local GP or hospital and will be asked to complete a medical questionnaire and consent form. If you're deemed fit and healthy enough, you'll have a further medical assessment and consultation at a specialist collection centre (where you will later donate your blood stem cells).

It's important to read about the methods used to collect blood stem cells PBSC and bone marrow donation because if youre on the register, you should be willing to donate in either way. The method will be determined by what the doctors believe will be best for the patient. However, you will of course always have the final decision on whether you are happy to proceed.

When a donor is matched with a patient, DKMS will cover the costs (including any travel, meals, or accommodation expenses that may be necessary and lost wages if you are not covered by your employer).

Your blood stem cells will never be stored, they last for around 72 hours and are delivered straight to the person in need by a special courier.

You will be allowed to meet the patient, if they consent, eventually UK guidelines state this can happen two years after the donation (and tules vary by country).Contact through anonymous letters can be established before this time via DKMS.

You will stay on the register until your 61st birthday.

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'I've potentially saved a stranger's life by donating my blood stem cells and it was painless' - inews

Bone Marrow Stem Cells Comments Off on ‘I’ve potentially saved a stranger’s life by donating my blood stem cells and it was painless’ – inews | October 22nd, 2019

The volume of blood needed every day by the NHS, and around the world, is staggering. But what about growing blood in a lab, could we do that? Chris Smith was joined by Cedric Ghevaert from the department of haematology at the University of Cambridge, to find out more about lab grown blood...

Cedric - So platelets are one of the three main blood components. Theyre actually the smallest cell in the body, if you look at a millimetre on a ruler you can line 250 platelets in that millimetre. The interesting thing is that some people argue they're not even a cell, because they don't contain a nucleus, they don't have DNA.

Chris - When I was learning biology at school they said platelets are bits of cells.

Cedric - That's absolutely true. They are fragments of a parent cell that lives in the bone marrow, and called a megakaryocyte, and one megakaryocyte will release about 1000 to 2000 of these little fragments called the platelets.

Chris - Every day?

Cedric - Every day we produce 10 to the 11 platelets

Chris - 100 billion Every day?

Cedric - Thats right.

Chris - And so these cells are just budding off little bits of themselves, which then go circulating around in the bloodstream.

Cedric - That's absolutely right.

Chris - And what are they doing there? What is their role?

Cedric - The main role of the platelets is to monitor your blood vessels, so they contain two things: Their outer layer, which is called the membrane. And on that membrane they have all sorts of little receptors, and these receptors will tell the platelets that the blood vessel has been damaged. When the platelet detects that it does two things, it will attach to the damaged blood vessel, and it will become activated and by that, it will then tell other platelets around, you need to come and help. These platelets will then stick to each other and form a plug to literally block the hole.

Chris - Do they change shape or anything? Do they become, sort of spiky or anything, to become more jagged so they jam in the hole.

Cedric - So they do become spiky, indeed you see that under the microscope when you activate the platelets. They go from a disc to this sort of spider, and that allows them to indeed interact with each other even better. The thing that they also do, is to then pull. They literally pull the wound together to try to stem the blood flow.

Chris - And they're presumably the first responders when you have a wound. They're there first because they're at the scene of the crime already because they're in the blood. And then as more come along, they're recruiting more of their mates from the numbers that come in the blood flow, and what do they provide the initial foundation of a blood thrombus or a clot.

Cedric - That's absolutely right. So once they become activated, inside the platelets there are granules, and those granules contain things that tell the blood, you need to clot. These things are released when the platelets become activated. And that leads to an amplification of the blood clotting that proteins are linked together, they form a polymer, and that polymer is a sort of a mesh that will capture more platelets and really plug the hole.

Chris - So the platelets are pulling more raw materials that are dissolved in the blood into that wound site, and then turning it into this dense mesh work that's gonna be a stable repair.

Cedric - That's absolutely right.

Chris - So they're really critical aren't they?

Cedric - Absolutely.

Chris - We can't do without them. And what's the problem with just growing them in a dish, because we can grow loads of things in dishes these days. We can you know, cells grow in dishes easily. So why can't you just churn out platelets in a dish?

Cedric - The main challenge with producing platelets in a dish is to do it so efficiently that actually we have a product that can be used, for example by the NHS and cost efficient. So if you look at a bag of platelets which we give for a transfusion, it contains three times ten to the eleven.

Chris - So 300 billion platelets.

Cedric - So where the platelets score as it were, is that we only need to produce one megakaryocyte, to produce a thousand platelets. And we can grow the megakaryocyte from stem cells. So the idea is that we can take stem cells, grow them into a megakaryocyte, and then right at the last stage of production, suddenly you have this massive amplification, a thousand times more platelets than you had megakaryocytes.

Chris - But if it's that easy to just grow these things in a dish, why are we not doing it? What is the what's the problem at the moment.

Cedric - The main problem is that particular last step. When the megakaryocyte is in the bone marrow it gets its cue from its environment. And it will detect the blood flow. It will be talked to by the cells that are around it, and that, it's very difficult to reproduce in the dish. If we produce megakaryocytes in liquid, in a culture dish they can produce 1 to 10 platelets, so we are at least a hundred times below what a megakaryocyte can do.

Chris - So you've got to have some way of recreating that very specialised three dimensional relationship in the bone marrow, where all these cells are in contact in a particularly special arrangement which seems to be the cue to them, to churn out platelets with the efficiency that they do when they're inside the body.

Cedric - And that's exactly the challenge that my group and several other groups across the world are trying to answer. And there are two ways to do this. First we need to tell the megakaryocyte theres a flow. They sense the flow, and that makes them release the platelets. So we put them in a bioreactor where they're exposed to shear, which is basically fluid going along them.

Chris - That's kind of mimicking the blood flowing through the bone marrow. So that would normally be bending and distorting the cells a bit, I presume, and that's what makes them churn off or snap off bits.

Cedric - That's right. So they produce these long digits which we call proplatelets, and these long digits elongate in the bloodstream and then snap off these platelets.

Chris - And when you make them, having mimicked this as best you can, do the platelets that you produce in the dish look like, and critically work like, the ones that are made naturally in the bone marrow?

Cedric - That's the critical thing that we are trying to address at the moment. They are bigger when we produce them in the dish, and they don't seem to quite react like normal platelets. However that doesn't mean that it wont work really well. What we need to do is to test them through a range of assays to really make the statement; these platelets are good, they will monitor your blood vessel, they will last in circulation.

Chris - Is your aim to make platelets bespoke for a patient? Or would you make off the shelf platelets, a bit like we'd currently do with transfusion medicine, where we just make a big bag of platelets collected from a range of donors?

Cedric - So at the moment we can produce platelets from either four donations, from four different donors, or we take them off a special machine where we have one pool of platelets coming from one donor, but we've talked the blood group before, one of the challenges with platelets, is that some people are immunised and they need platelets of a very specific blood type.

Chris - When you say immunisation, you mean that they've made an immune reaction to certain types in the past, so you've got to basically restrict what types you give them?

Cedric - Exactly. The beauty of working with stem cells is that we can edit the DNA somewhat, and because we can edit the DNA we can actually make platelets that don't express blood group, that are universal platelets. The one we produce in the dish can go to anyone. And that's one of the beauties of this technology.

Chris - And are you far away?

Cedric - We are not that far away. We are looking at human clinical trials in the next two to three years.

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Making blood in the lab | Interviews - The Naked Scientists

Bone Marrow Stem Cells Comments Off on Making blood in the lab | Interviews – The Naked Scientists | October 22nd, 2019

BOSTON and LONDON, Oct. 22, 2019 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced initial results from a clinical trial with a cryopreserved formulation of OTL-200, a gene therapy in development for the treatment of metachromatic leukodystrophy (MLD) at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. The initial data show that cellular engraftment with OTL-200 using a cryopreserved formulation is similar to that observed using a fresh formulation with the longest patient having 12 months of follow-up since treatment. The data are being featured this week in a poster session at the European Society of Gene & Cell Therapy (ESGCT) Annual Congress in Barcelona, Spain.

MLD is a devastating and rapidly progressing disease with no standard treatment options. In its most severe forms, patients will not survive beyond their first decade of life.

These data compare the initial results of OTL-200 in the first four MLD patients treated using a cryopreserved formulation to a previously presented integrated analysis of 29 patients treated with a fresh formulation that demonstrated meaningful clinical outcomes. Hematopoietic stem cells are collected, purified and transduced in the same way for both formulations. For the cryopreserved formulation, following transduction, the gene-corrected cells are placed in a specific medium that allows them to be stably frozen. After successful testing and release, the cryopreserved cells are shipped to the site of care where they are thawed and administered to patients who have received conditioning.

Presenting the first supportive data on OTL-200 using a cryopreserved formulation represents a cross-functional effort involving our clinical, CMC and regulatory teams as we prepare for the upcoming European regulatory submission for MLD followed by a BLA in the U.S., said Mark Rothera, president and chief executive officer of Orchard. If approved, a cryopreserved formulation of OTL-200 would more readily facilitate global commercialization and patient access efforts, which are key elements in our mission to deliver potentially curative therapies to patients suffering from often-deadly rare diseases.

Mr. Rothera continued, With over 40 patients now treated using a cryopreserved formulation across our pipeline of six clinical-stage programs, we are confident our approach is supported by a robust set of evidence.

Study Results At the time of the analysis, four early-onset MLD patients (two late infantile and two early juvenile) have been treated with the cryopreserved formulation of OTL-200. All patients are alive and were followed for a minimum of one month, with the longest follow-up out to 12 months in the first patient treated (median follow-up of 0.38 years). The age at the time of treatment ranged from seven months to 42 months.

The initial results in patients receiving the cryopreserved formulation (n=4) demonstrated the following:

Figure 1. Profiles of VCN in bone marrow CD34+ cells: OTL-200 cryopreserved vs. OTL-200 fresh

https://www.globenewswire.com/NewsRoom/AttachmentNg/83f41457-927b-4b1b-9ac2-9d48ac10353a

Figure 2. ARSA activity profile in peripheral blood: OTL-200 cryopreserved vs. OTL-200 fresh

https://www.globenewswire.com/NewsRoom/AttachmentNg/393ca5f0-98ad-47f8-b723-35c5c6c08d8f

c = cryopreserved; f = fresh; Sbj. = subject

We are pleased that these initial data suggest that using gene-corrected cells that have been cryopreserved has a similar impact on clinical biomarkers for early-onset MLD patients as the OTL-200 fresh formulation, said Dr. Valeria Calbi, a hematologist at San Raffaele Scientific Institute and SR-Tiget and an investigator of the study. The four treated patients showed good levels of engraftment of gene-corrected cells and reconstitution of ARSA activity at multiple time points, as well as encouraging early trends in GMFM scores that we look forward to evaluating with additional follow-up. We believe that these data further support the positive benefit / risk profile of OTL-200 as a therapy with potential lifelong benefit for patients with MLD.

Next Steps for OTL-200 Orchard remains on track to submit a marketing authorization application, or MAA, in Europe for MLD in the first half of 2020, as well as a biologics licensing application, or BLA, in the U.S. approximately one year later.

About MLD and OTL-200Metachromatic leukodystrophy (MLD) is a rare and life-threatening inherited disease of the bodys metabolic system occurring in approximately one in every 100,000 live births. MLD is caused by a mutation in the arylsulfatase-A (ARSA) gene that results in the accumulation of sulfatides in the brain and other areas of the body, including the liver, the gallbladder, kidneys, and/or spleen. Over time, the nervous system is damaged and patients with MLD will experience neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures, finding it more and more difficult to move, talk, swallow, eat and see. Currently, there are no effective treatments for MLD. In its late infantile form, mortality at 5 years from onset is estimated at 50% and 44% at 10 years for juvenile patients.1 OTL-200 is an ex vivo, autologous, hematopoietic stem cell-based gene therapy being studied for the treatment of MLD. OTL-200 was acquired from GSK in April 2018 and originated from a pioneering collaboration between GSK and the Hospital San Raffaele and Fondazione Telethon, acting through their joint San Raffaele-Telethon Institute for Gene Therapy in Milan, initiated in 2010.

About OrchardOrchard Therapeutics is a fully integrated commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies.

Orchards portfolio of ex vivo, autologous, hematopoietic stem cell (HSC) based gene therapies includes Strimvelis, a gammaretroviral vector-based gene therapy and the first such treatment approved by the European Medicines Agency for severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID). Additional programs for neurometabolic disorders, primary immune deficiencies and hemoglobinopathies are all based on lentiviral vector-based gene modification of autologous HSCs and include three advanced registrational studies for metachromatic leukodystrophy (MLD), ADA-SCID and Wiskott-Aldrich syndrome (WAS), clinical programs for X-linked chronic granulomatous disease (X-CGD), transfusion-dependent beta-thalassemia (TDT) and mucopolysaccharidosis type I (MPS-I), as well as an extensive preclinical pipeline. Strimvelis, as well as the programs in MLD, WAS and TDT were acquired by Orchard from GSK in April 2018 and originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy initiated in 2010.

Orchard currently has offices in the U.K. and the U.S., including London, San Francisco and Boston.

Forward-Looking StatementsThis press release contains certain forward-looking statements which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, Orchards expectations regarding the timing of regulatory submissions for approval of its product candidates, including OTL-200 for the treatment of metachromatic leukodystrophy, the timing of interactions with regulators and regulatory submissions related to ongoing and new clinical trials for its product candidates, the timing of announcement of clinical data for its product candidates, including OTL-200, and the likelihood that such data will be positive and support further clinical development and regulatory approval of its product candidates, and the likelihood of approval of such product candidates by the applicable regulatory authorities. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include, without limitation: the risk that any one or more of Orchards product candidates, including OTL-200, will not be successfully developed or commercialized, the risk of cessation or delay of any of Orchards ongoing or planned clinical trials, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates, the delay of any of Orchards regulatory submissions, the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates, the receipt of restricted marketing approvals, and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading Risk Factors in Orchards annual report on Form 20-F for the year ended December 31, 2018 as filed with the U.S. Securities and Exchange Commission (SEC) on March 22, 2019, as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

1Mahmood et al. Metachromatic Leukodystrophy: A Case of Triplets with the Late Infantile Variant and a Systematic Review of the Literature. Journal of Child Neurology 2010, DOI: http://doi.org/10.1177/0883073809341669

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaMolly CameronManager, Corporate Communications+1 978-339-3378media@orchard-tx.com

Figure 1

Profiles of VCN in bone marrow CD34+ cells: OTL-200 cryopreserved vs. OTL-200 fresh

Figure 2

ARSA activity profile in peripheral blood: OTL-200 cryopreserved vs. OTL-200 fresh; c = cryopreserved; f = fresh; Sbj. = subject

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Orchard Therapeutics Presents Data from OTL-200 in Patients with Metachromatic Leukodystrophy Using Cryopreservation - BioSpace

Bone Marrow Stem Cells Comments Off on Orchard Therapeutics Presents Data from OTL-200 in Patients with Metachromatic Leukodystrophy Using Cryopreservation – BioSpace | October 22nd, 2019

Blood stem cells protect themselves against viruses with structures known as 'interferon-induced transmembrane proteins,' seen here in green. These normally useful proteins are problematic for gene therapy treatments, as they work to keep therapeutic lentiviral vectors from infiltrating cells. Scripps Research scientists found a natural compound that lets down this shield, boosting the success rate of gene delivery.Credit: Image courtesy of the Torbett lab at Scripps ResearchLA JOLLA, CA Gene therapy has broadened the treatment possibilities for those with immune system deficiencies and blood-based conditions, such as sickle cell anemia and leukemia. These diseases, which once would require a bone marrow transplant, can now be successfully treated by modifying patients' own blood stem cells to correct the underlying genetic problem.

But today's standard process for administering gene therapy is expensive and time-consuminga result of the many steps required to deliver the healthy genes into the patients' blood stem cells to correct a genetic problem.

In a discovery that appears in the journalBlood, scientists atScripps Researchbelieve they have found a way to sidestep some of the current difficulties, resulting in a more efficient gene delivery method that would save money and improve treatment outcomes.

"If you can repair blood stem cells with a single gene delivery treatment, rather than multiple treatments over the course of many days, you can reduce the clinical time and expense, which removes some of the limitations of this type of approach," says Bruce Torbett, PhD, associate professor in the Department of Immunology and Microbiology, who led the research.

The new finding centers on caraphenol A, a small molecule closely related to resveratrol, which is a natural compound produced by grapes and other plants and found in red wine. Resveratrol is widely known as an antioxidant and anti-inflammatory agent. Similar to resveratrol, caraphenol A is anti-inflammatory, but in this study, it served a different role.

Torbett and his team became interested in the unique chemical properties of resveratrol and similar types of molecules and wondered if they could enable viral vectors, used in gene therapy to deliver genes, to enter blood stem cells more easily. This would be momentous because stem cellsand in particular, self-renewing hemopoietic stem cellshave many barriers of protection against viruses, making them challenging for gene therapy to infiltrate.

Related Article:Solution to 50-Year-Old Mystery Could Lead to Gene Therapy for Common Blood Disorders

"This is why gene therapy of hemopoietic stem cells has been hit-or-miss," Torbett says. "We saw a way to potentially make the treatment process significantly more efficient."

The gene therapy treatment process currently requires isolating a very small population of hemopoietic stem cells from the blood of patients; these young cells can self-renew and give rise to all other types of blood cells. Therapeutic genes are then delivered to these cells via specially engineered viruses, called "lentiviral vectors," which leverage viruses' natural knack for inserting new genetic information into living cells.

However, hemopoietic stem cells are highly resilient to viral attacks. They protect themselves with structures known as interferon-induced transmembrane (IFITM) proteins, which intercept lentiviral vectors. Because of this, it can take many attemptsand a large quantity of expensive gene therapy vectorsto successfully delivery genes into hemopoietic stem cells, Torbett says.

Torbett and his team found that by adding the resveratrol-like compound, caraphenol A, to human hemopoietic stem cells, along with the lentiviral vector mix, the cells let down their natural defenses and allowed vectors to enter more easily. Once the treated stem cells were placed into mice, they divided and produced blood cells containing the new genetic information.

Another key benefit of the approach is time: If gene delivery treatment of blood stem cells can be accomplished in less time, the cells can be re-administered to the patient sooner. This not only makes treatment more convenient for the patient, but it helps to ensure the stem cells don't lose their self-renewing properties, Torbett says. The longer stem cells exist outside of the body and are manipulated, the more likely it is they will lose their ability to self-generate and ultimately correct disease.

Torbett and his team are continuing to study the underlying reasons for stem cells' inherent resistance to genetic modification, with the goal of further improving treatment efficiency and reducing cost. Because many of the diseases treatable with gene therapy affect children, Torbett says he feels a special urgency to advance this discovery from the lab into the clinic.

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When Added to Gene Therapy, Plant-Based Compound May Enable Faster, More Effective Treatments - Lab Manager Magazine

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The combination of trilaciclib and chemotherapy was generally welltolerated with promising activity in a phase II trial of patients with metastatic triple-negative breast cancer. However, it did not offer improvements in measures of myelosuppression compared with chemotherapy alone.

Chemotherapy-induced myelosuppression commonly leads to dose reductions that can restrict therapeutic dose intensity, wrote study authors led by Antoinette R. Tan, MD, of the Levine Cancer Institute in Charlotte, North Carolina. Introducing therapy that can protect the immune cells and bone marrow from the cytotoxic effects of chemotherapy has the potential to optimize antitumour activity while minimizing myelotoxicity.

Triliciclib is an inhibitor of cyclin-dependent kinases-4/6; it can enhance antitumor immunity and preserve hematopoietic stem and progenitor cells during chemotherapy. The new phase II study included a total of 142 patients with metastatic triple-negative breast cancer randomized to receive either gemcitabine (Gemzar) plus carboplatin alone (group 1) or 1 of 2 regimens with those agents plus trilaciclib. One regimen included intravenous trilaciclib along with the chemotherapy agents on days 1 and 8 of 21-day cycles (group 2), while the other included gemcitabine and carboplatin on days 2 and 9 along with trilaciclib on days 1, 2, 8, and 9 (group 3). The results of the study werepublishedonline ahead of print on September 28 inLancet Oncology.

The median follow-up for the three groups was 8.4 months, 12.7 months, and 12.9 months, respectively. During the first cycle, the mean duration of severe neutropenia was 0.8 days in the chemotherapy alone group, 1.5 days in group 2, and 1.0 days in group 3; these differences were not significant.

A total of 9 of 34 patients (26%) in group 1, compared with 12 of 33 patients (36%) in group 2 and eight of 35 patients (23%) in group 3 (P= 0.70). There were no differences between the groups with regard to all-cause dose reductions, patients requiring G-CSF, or patients requiring red blood cell transfusion, among other outcomes. The most common treatment-emergent adverse events included anemia, neutropenia, and thrombocytopenia in groups 1 and 2, and in group 3 they included neutropenia, thrombocytopenia, and nausea.

Overall survival outcomes in the 2 trilaciclib groups were significantly better than in the chemotherapy alone group. The combined trilaciclib groups had a median OS of 20.1 months, compared with 12.6 months without trilaciclib.

Though the trilaciclib regimens did not show significant improvements with regard to myelosuppression compared with chemotherapy, the authors highlighted the improvement in anti-tumor activity.

Together with the safety profile reported, the clinically meaningful improvements in overall survival support further studies of trilaciclib in patients with metastatic triple-negative breast cancer, they wrote.

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Can Triliaciclib Improve Chemotherapy Tolerance in Triple-Negative Breast Cancer? - Cancer Network

Bone Marrow Stem Cells Comments Off on Can Triliaciclib Improve Chemotherapy Tolerance in Triple-Negative Breast Cancer? – Cancer Network | October 22nd, 2019