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Stem Cells PRP, Acne & Skin Rejuvenation Cape Town

By Dr. Matthew Watson

PRP Skin Regeneration Therapy, a type of regenerative medicine with the patients own blood, uses components called platelets in the blood to rejuvenate the skin.

Based on an innate wound-healing ability, the therapy is performed by injecting components collected from the blood and is associated with no risk of allergy or infections. The safe therapy has been studied and applied in a variety of fields. It is indicated for a wide variety of conditions; it is used for the treatment of trauma and burn in the department of plastic surgery and also used as an adjunct to implant therapy in the department of dentistry.

Our clinics have introduced a new technology for extraction of platelet-rich plasma containing autologous white blood cells that has a PRP enrichment rate of about 6- to 10-fold (about three to five times higher than that for conventional one). It also contains white blood cells, which are not contained in conventional PRP. The therapy is found to have much greater efficacy in rejuvenating the skin, eliminating wrinkles, and reducing irregularities from acne marks compared with conventional therapy.

Indications: Expected Effects in Cosmetic Medicine

PRP is particularly effective for crepy skin under the eyes (fine wrinkles) that are difficult to treat by conventional Rejuvenation Therapy. Inducing skin regeneration, the therapy is also effective in treating wrinkles, acne marks, sags and wrinkles on the neck.

Features of the Process Include:

1. Allowing the extraction of White Blood Cells not found in conventional PRP

2. The interaction between PRP and white blood cells results in the release of growth factors that potentiate the natural healing power and tissue reorganization potential at injection sites and subsequent regeneration of the skin.

3. In conventional therapy, it takes about two months for any benefit to be seen, although the time varies among individuals. New-PRP Skin Regeneration Therapy produces noticeable symptomatic improvement in a short period of about two weeks.

Comparison with Conventional Procedures

The PRP enrichment rate is three to five times higher than that for conventional procedures, and the time to benefit is reduced to one-fourth.

Mechanism of New-PRP Skin Regeneration Therapy

PRP Skin Regeneration Therapy uses components called platelets contained in the blood. Platelets play a role in stopping bleeding and repairing damaged blood vessels and cells in the body. Platelets contain substances called growth factors that activate and rejuvenate cells in the body. The growth factors, when released, induce the production of collagen and generation of new capillaries to rejuvenate the skin.

Precautions on How to Choose PRP Therapy

Many clinics now use what they call autologous platelets to eliminate wrinkles and treat acne marks. All those clinics claim skin regeneration therapy with autologous platelets. However, the efficacy of therapy differs substantially.

Important factors for greater efficacy include the concentration and component of platelets for injection. As described previously, skin regeneration with PRP is based on the mechanism that platelets release a variety of growth factors to promote tissue repair, angiogenesis, and collagen production for skin rejuvenation.

The efficacy is enhanced by improving the quality of platelets. Our clinics use a method of injecting platelet-rich plasma containing autologous white blood cells that is prepared by mixing enriched platelets (6 to 10 fold) collected from blood and an appropriate amount of white blood cells, which are rarely contained in common PRP. Our method is found to be highly effective in a range of symptoms.

Skin Rejuvenation with PRP

1. Injection of Platelet Component (PRP):The aged skin has less collagen, low elasticity, reduced amount of hyaluronic acid, and low ability to retain moisture.

2. Release of Growth Factors From Platelet Component (PRP) cell growth is activated, and collagen is produced.

3. Regeneration and Rejuvenation of Skin Tissue:Here Collagen is produced, and skin elasticity is improved. The ability to retain moisture is restored.

For separation of platelet-rich plasma (PRP), a dedicated kit called Fibrinet AGF is used. The use of a specific filter and a centrifuge achieves a high platelet recovery rate of 97% or more and allows preparation of plasma containing six to ten times as many platelets as the common one. This is a three step process:

Step 1: Collection of Blood

Step 2:Separation of Platelet Component A specific filter and a centrifuge are used to prepare platelet-rich plasma (PRP) containing autologous white blood cells.

Step 3. Injection of Platelet Component The platelet-rich plasma (PRP) with autologous white blood cells is injected into the area of concern. It takes about 30 to 40 minutes from blood collection to injection.

Platelets and white blood cells exert a synergistic effect, resulting in the release of a variety of growth factors at the injection sites. This promotes the production of collagen and hyaluronic acid and wound healing, leading to improvement of symptoms such as wrinkles and irregularities from acne marks.

Comparison of Conventional Anti-Aging Therapy

PRP Skin Regeneration Therapy is expected to provide great benefit for crepy skin under the eyes, which are difficult to treat with conventional rejuvenating injections and laser therapy. The therapy uses the patients own blood for rejuvenation and thus poses no risk of infection or allergy. It has the advantage of a longer duration of efficacy compared with injection of hyaluronic acid and collagen that are absorbed into the body. Other features such as no need for skin incision and short downtime (swelling usually resolves in two to three hours) make this therapy a safe treatment.

NOTE: When injection is performed under the eyes, redness may persist for two to three days but resolve over time.

Consult a physician about the best procedure, depending on the sites and conditions of your wrinkles and others symptoms.

Before and After

Another feature of the therapy is that the patient will experience a natural change in the operative site, as well as minimal discomfort as the beneficial effects gradually occur after about two weeks of therapy.

As simple as giving a tube of blood, this nonsurgical treatment utilises patients own platelets and stem cells to promote wound healing. PRP can effectively improve the bodys natural collagen production, resulting in a more youthful appearance.

Neocel PRP kits are the only FDA approved stem cells harvesting kit in the world. The Wembley MediSpa in Cape Town is amongst the few clinics in South Africa to offer this world class treatment with a world class Doctor (90 120 mins).

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Stem Cells PRP, Acne & Skin Rejuvenation Cape Town

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Potential Baldness Pathway Uncovered while Studying Rare Skin Disease – Genetic Engineering & Biotechnology News

By daniellenierenberg

The researchers constructed mouse models for WNT10A-associated HED by deleting the Wnt10a gene. The mutant mice displayed the same symptoms as HED patients with severe loss-of-function mutations in the WNT10A gene. Long-term absence of WNT10A leads to miniaturization of hair follicle structures and enlargement of the associated sebaceous glands, a phenomenon that is also observed in male pattern baldness.

We showed "that -catenin pathway activity and adult epithelial progenitor proliferation are reduced in the absence of WNT10A, and identify Wnt-active self-renewing stem cells in affected tissues including hair follicles, sebaceous glands, taste buds, nails, and sweat ducts, the authors wrote. Human and mouse WNT10A mutant palmoplantar and tongue epithelia also display specific differentiation defects that are mimicked by the loss of the transcription factor KLF4. We found that -catenin interacts directly with region-specific LEF/TCF [lymphoid enhancer factor/T-cell factor] factors, and with KLF4 in differentiating, but not proliferating, cells to promote expression of specialized keratins required for normal tissue structure and integrity.

Interestingly, the UPenn team also discovered that cracking and scaling of palm and foot sole skin in WNT10A patients is due to decreased expression of a structural protein called keratin 9, which is specifically expressed in these regions of skin and contributes to its mechanical integrity.

"Our studies took us back and forth between human patients and our mouse model," said Dr. Millar. "Our goal was to find what happened to cellular components affected by the WNT10A mutation to make better treatments."

Dr. Millar and her colleagues showed that decreased proliferation and keratin 9 expression in the absence of WNT10A resulted from the failure of signaling through a well-characterized pathway that stabilizes -catenin, allowing it to enter the cell nucleus and activate gene transcription. These findings indicate that small-molecule drugs that activate the -catenin pathway downstream of WNT10A could potentially be used to treat hair thinning and palm and sole skin defects in WNT10A patients. These agents may also be useful for preventing hair loss in a subgroup of people with male-pattern baldness.

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The Vitamin That Targets and Kills Cancer Stem Cells – Care2.com

By Sykes24Tracey

While the search for possible cures for cancer continue in laboratories around the world, exciting new research turns our attention to a commonly available, inexpensive vitamin. Thats because a humble vitamin has been found to seek out and destroy cancer stem cells, which are cells that are believed to drive the creation of new cancer cells and cancer tumors.

The study, published in the medical journal Oncotarget, found that vitamin C can actually seek out and destroy cancer stem cells, thereby preventing the spread of the disease. Vitamin C was found by researchers to be up to 10 times more effective at killing cancer stem cells than experimental drugs. Thats good news considering the toll that cancer is currently taking. Cancer is currently the second leading cause of death and killed almost 9 million people in 2015 alone.

Lead study author Dr. Michael P. Lisanti, professor of translational medicine at the University of Salford said in an interview with Medical News Today: We have been looking at how to target cancer stem cells with a range of natural substancesbut by far the most exciting are the results with vitamin C. Vitamin C is cheap, natural, nontoxic and readily available, so to have it as a potential weapon in the fight against cancer would be a significant step.

Vitamin C is found in most fruits and vegetables, but especially in red bell peppers, strawberries, oranges, grapefruit, lemons, limes, pomegranates, black currants, spinach, beet greens, tomatoes and sprouts. Eating a plant-based or largely plant-based diet high in vitamin C-rich foods may be helpful in preventing or treating cancer, but supplementation may be necessary to achieve the study results. Vitamin C is available in a variety of forms, with ascorbic acid being the primary one, along with other buffered options such as calcium ascorbate. The Oncotarget study found that ascorbic acid effectively sought out and destroyed cancer stem cells.

It is not clear how much vitamin C is necessary to create the anti-cancer results. More research may help to determine the ideal dosage. The recommended dietary intake is 90 milligrams of vitamin C, but many natural health experts believe that this amount is extremely low and doesnt take stress or diseases like cancer into account. Stress causes the rapid depletion of vitamin C. Our stress glands, the adrenal glands, which are two small, triangular-shaped glands that sit atop the kidneys in the abdominal region, use high amounts of vitamin C, particularly when they are dealing with acute or chronic stress. Many natural health experts recommend 2000 milligrams of vitamin C daily, and sometimes even more than that if it is part of a therapeutic protocol.

Nobel Prize winner Dr. Linus Pauling first discovered vitamin C and its role in fighting cancer. This new Oncotarget study builds on Dr. Paulings research, showing that vitamin C also targets cancer stem cells, an important advancement in our knowledge of cancer and vitamin C. Other research published in the medical journal Science found that high doses of vitamin C may help in the treatment of colorectal cancer. It is a good idea to work with a naturally-minded health professional if you intend to take high doses of vitamin C, divided throughout the day.

Because vitamin C is water soluble, it is not stored in our body and must therefore be ingested on a daily basis to avoid a deficiency. Some of the symptoms of a vitamin C deficiency include: excessive hair loss, becoming exhausted easily, fragile bones, frequent nosebleeds, gums that bleed easily, skin that bruises easily, and sores or wounds that heal slowly.

Vitamin C is also crucial to the formation of bones and teeth, digestion, blood cell formation, wound healing and the production of collagen, which is involved in maintaining the skins youthful elasticity.

Related:Dont Believe in Herbal Medicine? 10 Things to Change Your MindThe 5 Best Herbs to Soothe Your NervesShould You Actually Starve a Fever?

Dr. Michelle Schoffro Cook, PhD, DNM is the publisher of the free e-news Worlds Healthiest News, president of PureFood BC, and an international best-selling and 20-time published book author whose works include: The Life Force Diet: 3 Weeks to Supercharge Your Health and Get Slim with Enzyme-Rich Foods.

Disclaimer: The views expressed above are solely those of the author and may not reflect those of Care2, Inc., its employees or advertisers.

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Origin of age-related muscle loss discovered – Bel Marra Health

By raymumme

Home General Health Origin of age-related muscle loss discovered

When were young, we feel like we can accomplish anything. We can go anywhere and do anything because we dont feel encumbered by our physical limitations. However, when we age, we start to lose this feeling of empowerment. Our once-impressive physical physique fades away, leaving us weak and defenseless.

However, new research into the reasons why we lose muscle cells as we age may lead to the development of new drugs that can slow the process of muscle decline.

It was previously thought that the driving factor behind age-related muscle decline was the loss of motor neuronsnerve cells in the spinal cord controlling muscle. Starting in our 30s, we begin to notice that activities that once came easily are now difficult.

When we reach our 70s and 80s, we become weak and frail, having to depend on others to lend us their strength on a daily basis. No amount of diet and exercise can prevent this inevitable result.

The pursuit of preserving our youth has been a goal for many researchers and scientists. Understand how and why the body ages over time is something worth pursuing in the hopes of improving lives.

Research using mouse models has found that muscle stem cells play a very important role in lifelong maintenance of muscle. This challenges the current, widely accepted theory of motor neurons.

Muscle stem cell pools were seen to reside in muscle tissue that responds to exercise and injury. These stem cells are directly involved in repair and growth of muscles. It is the dying off of these stem cells that is theorized to be the driving factor for muscle loss.

Mice that were genetically altered to prevent muscle stem cell loss maintained healthier muscles in old age.

Subsequently, no evidence was found linking motor neurons to age-related muscle loss.

I think weve shown a formal demonstration that even for aging sedentary individuals, your stem cells are doing something. They do play a role in the normal maintenance of your muscle throughout life, said study author Joe Chakkalakal, Ph.D., assistant professor of Orthopaedics in the Center for Musculoskeletal Research at the University of Rochester Medical Center.

Related Reading:

Simple fix for age-related muscle loss

Simple steps to slow down your muscle loss

https://www.urmc.rochester.edu/news/story/4788/stem-cells-may-be-the-key-to-staying-strong-in-old-age.aspx

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Asterias Biotherapeutics to Present New 9-Month Efficacy Data from AST-OPC1 SCiStar Study at the International … – PR Newswire (press release)

By Sykes24Tracey

Workshop Details

Title: Clinical Development of hESC-derived Oligodendrocyte Progenitors for the Treatment of Spinal Cord injury Date: Tuesday, June 13, 2017 Time: 10:45 - 11:10 AM Room: 205ABC

Poster Presentation Details

Title: Safety and Efficacy of Human Embryonic Stem Cell-Derived Oligodendrocyte Progenitor Cells (AST-OPC1) in Patients with Subacute Cervical Spinal Cord Injury Poster Session III-ODD Poster Board F-1225 Date: Friday, June 16, 2017 Time: 6:00 - 7:00 PM

About the SCiStar Trial

The SCiStar trial is an open-label, single-arm trial testing three sequential escalating doses of AST-OPC1 administered at up to 20 million AST-OPC1 cells in as many as 35 patients with sub-acute, C-5 to C-7, motor complete (AIS-A or AIS-B) cervical SCI. These individuals have essentially lost all movement below their injury site and experience severe paralysis of the upper and lower limbs. AIS-A patients have lost all motor and sensory function below their injury site, while AIS-B patients have lost all motor function but may retain some minimal sensory function below their injury site. AST-OPC1 is being administered 14 to 30 days post-injury. Patients will be followed by neurological exams and imaging procedures to assess the safety and activity of the product.

The study is being conducted at six centers in the U.S. and the company plans to increase this to up to 12 sites to accommodate the expanded patient enrollment. Clinical sites involved in the study include the Medical College of Wisconsin in Milwaukee, Shepherd Medical Center in Atlanta, University of Southern California (USC) jointly with Rancho Los Amigos National Rehabilitation Center in Los Angeles, Indiana University, Rush University Medical Center in Chicago and Santa Clara Valley Medical Center in San Jose jointly with Stanford University.

Asterias has received a Strategic Partnerships Award grant from the California Institute for Regenerative Medicine, which provides $14.3 million of non-dilutive funding for the Phase 1/2a clinical trial and other product development activities for AST-OPC1.

Additional information on the Phase 1/2a trial, including trial sites, can be found at http://www.clinicaltrials.gov, using Identifier NCT02302157, and at the SCiStar Study Website (www.SCiStar-study.com).

About AST-OPC1

AST-OPC1, an oligodendrocyte progenitor population derived from human embryonic stem cells, has been shown in animals and in vitro to have three potentially reparative functions that address the complex pathologies observed at the injury site of a spinal cord injury. These activities of AST-OPC1 include production of neurotrophic factors, stimulation of vascularization, and induction of remyelination of denuded axons, all of which are critical for survival, regrowth and conduction of nerve impulses through axons at the injury site. In preclinical animal testing, AST-OPC1 administration led to remyelination of axons, improved hindlimb and forelimb locomotor function, dramatic reductions in injury-related cavitation and significant preservation of myelinated axons traversing the injury site.

In a previous Phase 1 clinical trial, five patients with neurologically complete, thoracic spinal cord injury were administered two million AST-OPC1 cells at the spinal cord injury site 7-14 days post-injury. They also received low levels of immunosuppression for the next 60 days. Delivery of AST-OPC1 was successful in all five subjects with no serious adverse events associated with AST-OPC1. No evidence of rejection of AST-OPC1 was observed in detailed immune response monitoring of all patients. In four of the five patients, serial MRI scans indicated that reduced spinal cord cavitation may have occurred. Based on the results of this study, Asterias received clearance from FDA to progress testing of AST-OPC1 to patients with cervical spine injuries, which represents the first targeted population for registration trials.

About Asterias Biotherapeutics

Asterias Biotherapeutics, Inc. is a biotechnology company pioneering the field of regenerative medicine. The company's proprietary cell therapy programs are based on its pluripotent stem cell and immunotherapy platform technologies. Asterias is presently focused on advancing three clinical-stage programs which have the potential to address areas of very high unmet medical need in the fields of neurology and oncology. AST-OPC1 (oligodendrocyte progenitor cells) is currently in a Phase 1/2a dose escalation clinical trial in spinal cord injury. AST-VAC1 (antigen-presenting autologous dendritic cells) is undergoing continuing development by Asterias based on promising efficacy and safety data from a Phase 2 study in Acute Myeloid Leukemia (AML), with current efforts focused on streamlining and modernizing the manufacturing process. AST-VAC2 (antigen-presenting allogeneic dendritic cells) represents a second generation, allogeneic cancer immunotherapy. The company's research partner, Cancer Research UK, plans to begin a Phase 1/2a clinical trial of AST-VAC2 in non-small cell lung cancer in 2017. Additional information about Asterias can be found at http://www.asteriasbiotherapeutics.com.

FORWARD-LOOKING STATEMENTS

Statements pertaining to future financial and/or operating and/or clinical research results, future growth in research, technology, clinical development, and potential opportunities for Asterias, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, need and ability to obtain future capital, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the businesses of Asterias, particularly those mentioned in the cautionary statements found in Asterias' filings with the Securities and Exchange Commission. Asterias disclaims any intent or obligation to update these forward-looking statements.

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SOURCE Asterias Biotherapeutics, Inc.

http://www.asteriasbiotherapeutics.com

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Asterias Biotherapeutics to Present New 9-Month Efficacy Data from AST-OPC1 SCiStar Study at the International ... - PR Newswire (press release)

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Human heart tissue grown from stem cells improves drug testing – Medical Xpress

By JoanneRUSSELL25

June 8, 2017 This image shows human heart muscle cells growing in the 3D tissue structure. The cells have been stained with fluorescent molecules to identify the nuclei in blue, and cardiac-specific protein, in green. Credit: Agency for Science, Technology and Research (A*STAR), Singapore

Researchers at the Institute of Bioengineering and Nanotechnology (IBN) of A*STAR have engineered a three-dimensional heart tissue from human stem cells to test the safety and efficacy of new drugs on the heart.

"Cardiotoxicity, which can lead to heart failure and even death, is a major cause of drug withdrawal from the market. Antibiotics, anticancer and antidiabetic medications can have unanticipated side effects for the heart. So it is important to test as early as possible whether a newly developed drug is safe for human use. However, cardiotoxicity is difficult to predict in the early stages of drug development," said Professor Jackie Y. Ying, Executive Director at IBN.

A big part of the problem is the use of animals or animal-derived cells in preclinical cardiotoxicity studies due to the limited availability of human heart muscle cells. Substantial genetic and cardiac differences exist between animals and humans. There have been a large number of cases whereby the tests failed to detect cardiovascular toxicity when moving from animal studies to human clinical trials.

Existing screening methods based on 2-D cardiac structure cannot accurately predict drug toxicity, while the currently available 3-D structures for screening are difficult to fabricate in the quantities needed for commercial application.

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To solve this problem, the IBN research team fabricated their 3-D heart tissue from cellular self-assembly of heart muscle cells grown from human induced pluripotent stem cells. They also developed a fluorescence labelling technology to monitor changes in beating rate using a real-time video recording system. The new heart tissue exhibited more cardiac-specific genes, stronger contraction and higher beating rate compared to cells in a 2-D structure.

"Using the 3-D heart tissue, we were able to correctly predict cardiotoxic effects based on changes in the beating rate, even when these were not detected by conventional tests. The method is simple and suitable for large-scale assessment of drug side effects. It could also be used to design personalized therapy using a patient's own cells," said lead researcher Dr Andrew Wan, who is Team Leader and Principal Research Scientist at IBN.

The researchers have filed a patent on their human heart tissue model, and hope to work with clinicians and pharmaceutical companies to bring this technology to market.

This finding was reported recently in the Biofabrication journal.

Explore further: Stem cell-based screening methods may predict heart-related side effects of drugs

More information: Hong Fang Lu et al. Engineering a functional three-dimensional human cardiac tissue model for drug toxicity screening, Biofabrication (2017). DOI: 10.1088/1758-5090/aa6c3a

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Patients with rare blood cancer brought back to normal – The Hindu – The Hindu

By Sykes24Tracey

Patients with rare blood cancer brought back to normal - The Hindu
The Hindu
Undergo bone marrow transplant at Apollo Cancer Institute.

and more »

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Harvard Biotech Spinout Lands $10M to Make Platelets From Stem Cells – Xconomy

By Sykes24Tracey

Xconomy Boston

If youve donated blood, you probably received a follow-up message asking you to consider a platelet donation. Donation centers must constantly replenish their blood supplies, but the need for platelets is particularly acute. Compared to other components of blood, platelets are fragile and short lived, which puts high demand on the small supply of these cell fragments that are key to stopping bleeding.

Plasma separated from blood can be frozen and red blood cells can be refrigerated for up to 42 days. But platelets, which must be stored at room temperature, last just five days. After pathogen screening and transport, platelets have about two days to reach a patient, says Sven Karlsson, president and co-founder of Boston-based Platelet Biogenesis.

Karlssons startup aims to bring hospitals an alternative: platelets produced as needed from stem cells. Platelet BioGenesis, a spinout from Harvard University and Brigham and Womens Hospital, now has $10 million from a Series A round of funding to support preclinical testing of its regenerative medicine technology.

The body produces the components of blood in bone marrow. Platelets are made by a type of bone marrow cell called a megakaryocyte. Platelet Biogenesis produces its platelets in a two-step process. It first develops megakaryocytes from stem cells in culture, Karlsson says. Next, the megakaryocytes are fed into a device that the company developed that puts the cells through a process the company says is similar to what occurs in natural bone marrow physiology and results in the production of platelets.

Were making real, functional platelets, Karlsson says. Instead of mimicking the platelet, were mimicking the process of making a platelet.

Platelet transfusions are needed by patients whose bodies dont produce enough platelets, as well as those who have clotting problems. These transfusions are also used to treat patients who have cancer or are undergoing surgery. Since most platelet donation centers are in urban areas, the short shelf life of platelets makes supplies tight in rural areas. Donations dont entirely solve the problem, Karlsson says. While donating whole blood takes about 15 minutes, platelet donation takes 90 minutes, which makes it harder to get donors.

Researchers have tried to address the platelet shortage problem by developing synthetic platelets. But Karlsson says that the synthetic versions can cause side effects. The idea for replicating the bodys process of producing platelets stems from more than a decade of research. Jonathan Thon, the CEO and co-founder of Platelet BioGenesis, was pursuing ways to extend the shelf life of platelets. But Karlsson says Thon concluded a better approach might be developing a way to replace human donors. As a post-doctoral researcher at Harvard, he invented a microfluidic device that mimics a human bone marrow.

Before Platelet BioGenesis can bring its platelet-making technology to the market, it will need FDA approval. The regulator treats blood products as therapeutics, which means that the company will need to conduct clinical trials. Karlsson says the funding round will support preclinical research. Within three years, he expects the startup will be able to start Phase 1 safety studies. For those trials, Platelet BioGenesis will need to raise additional funds. The current financing round was led by Qiming US Healthcare Fund and included participation from Vivo Capital, VI Ventures, Adena Partners, eCoast Angels, as well as other unidentified investors.

If Platelet BioGenesis wins FDA approval, Karlsson says the company plans to become a manufacturer, selling its platelets in the existing blood supply chain. Its early to talk about pricing, but he says that Platelet Biogenesis products could be more affordable because they are produced from a pathogen-free source. They could also be produced on demand. While the startups platelets should have the same shelf-life as platelets from human donors, Karlsson says that the parent cells that produce platelets can be stored frozen. When platelets are needed, thawed cells can be placed in the companys microfluidic device, which produces platelets within hours.

Karlsson says Platelet BioGenesis might be able offer another advantage: beating the five-day shelf life of donor platelets. In the body, a platelet lasts 10 days. If all of Platelet BioGenesis platelets are born on the same day, they should theoretically have the same 10-day life, he says. Thats one of the things the company plans to test in upcoming trials. The company will also evaluate its platelets to make sure that they are comparable to donor platelets.

Imageby Wikimedia user Erhabor Osarovia a Creative Commons license.

Frank Vinluan is editor of Xconomy Raleigh-Durham, based in Research Triangle Park. You can reach him at fvinluan [at] xconomy.com

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Stem cell treatment for lethal STAT1 gene mutation produces mixed results – Medical Xpress

By Sykes24Tracey

June 8, 2017 One example of STAT1 GOF Mutation phenotype. Credit: Hiroshima University

Researchers report the first-ever study assessing how patients with "gain of function" mutation of the STAT1 gene respond to stem cell transplantation. It involved 15 young patients from nine different countries, each suffering a range of complications caused by the gene's mutation.

Of these, only six survived a regime of stem cell transplantationwith five completely cured and disease free by the study's conclusion.

The study was carried out by Dr. Satoshi Okada (Hiroshima University), Professor Jennifer Leiding (University of Florida), Professor Tomohiro Morio (Tokyo Medical and Dental University), and Professor Troy Torgerson (University of Washington).

Dr. Okada, who first discovered the STAT1 gain of function mutation in 2011, says, "Overall, this result is disappointing but the fact that five patients were cured proves that treatment with stem cells can work, and we now need to learn from these 15 individual cases."

The STAT1 gene plays a vital role in the body's immune system. Rare mutations can lead to STAT1's over-activation (GOF) and autoimmunity.

While the majority of patients afflicted typically show mild to moderate symptoms involving fungal (mostly Candida), bacterial, and viral infectionsabout 10 percent of cases are severe and life threatening.

Until now, developing suitable treatments has been challenging; e.g. anti-fungal drugs temporarily treat the symptoms but not the source mutation, and immunosuppressive therapies often do more harm than good by knocking out already overburdened immune systems.

With only one confirmed case prior to this study of a sufferer being successfully cured using stem cell transplantation, researchers are keen to build an understanding of best practices in order to offer real hope for the typically young sufferers of this condition.

The 15 selected patients were sourced via an international appeal to transplant centers and consortiums. Their ages ranged from 13 months to 33 years at the time of treatment. Screening by HU researchers confirmed that each had the STAT1-GOF mutation, and that the mutation was the source of their ailments.

Treatment was carried out independently by centers around the world. It used chemotherapy to eradicate the host's bone marrowthe source of the damaging STAT1 mutation in these patients. Healthy stem cell cultures sourced from donors were then transplanted into the subjects with the aim of reconstituting their bone marrow to a mutation-free, disease-fighting state.

The researchers suspect three reasons for the low 40 percent success rate:

In response, the researchers have made several proposals for improving this treatment. Due to most of the patients having mild to moderate ailments, only those suffering from severe symptoms should undergo this treatment. In addition, the chemotherapy dosage should be reduced. Those who received low-dose chemotherapy reacted better.

However, a balance must be struck. Low-dose chemotherapy may not eradicate host bone marrow to the extent required for its reconditioning the chance of transplant rejection is thus increased. With this in mind, support treatment may be required to neutralize host antibodies and prevent attacks of introduced stem cells.

Finally, due to the relative success seen in younger patients, stem cell transplantation should occur at as early an age as possible. Due to recent advancements in STAT1-GOF diagnosis, early detection is now a very real possibility hopefully leading to greater success rates, and less suffering for those carrying this potentially devastating mutation.

Explore further: 'Smart' genetic library makes disease diagnosis easier

More information: Jennifer W. Leiding et al. Hematopoietic stem cell transplantation in patients with Gain of Function STAT1 Mutation, Journal of Allergy and Clinical Immunology (2017). DOI: 10.1016/j.jaci.2017.03.049

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My Turn: Do something within your power to save another life – The Recorder

By raymumme

This week alone in the news weve seen air strikes, suicide bombings and murders caused by hate. Violence over the hue of someones skin, the way they speak or how they dress. Hate-filled speech by neighbors at meetings and on Facebook. At dinner yesterday, my 8-year-old step-daughter asked her dad, Whats a bomb? My heart is heavy.

Its easy to forget that we are more alike than we are unalike. I offer to you a different perspective.

Six years ago my brother got the call he had Hodgkin Lymphoma, a cancer that starts in cells that are part of the bodys immune system. He was 28 years old. It started as a visible lump under his collar bone, and sometimes you wonder how can so much suffering be caused by such a little lump? And so my story begins.

About one year into his treatment, he reached remission, and from there he was required to undergo an autologous stem cell transplant (a transplant using his own stem cells) to replace his bone marrow and stem cells that were destroyed by chemotherapy and radiation. Fast-forward 10 or so months and my brothers cancer returned. This time the treatment plan had to change his body needed help actually fighting the cancer cells, rather than just a replenishment of normal blood cells. This time around, he required an allogeneic stem cell transplant (a transplant using the stem cells from a healthy donor) and as his sister, I needed to be tested to see if I was a tissue match.

This was all new to me and our family. You hear a lot about cancer. We all know someone who has it, if you dont have cancer yourself. But I knew nothing about stem cell transplants or what it meant to be a donor. First we had to find out if my brother and I were a match.

I received a kit in the mail and all I had to do was swab the inside of my cheek, place the swab inside a sealed bag, and mail it back to the hospital. A week or so later, my brother got the news from his doctor that changed our lives. I was, in fact, a match a near perfect match and we could move forward with his second stem cell transplant.

At this point in my story, youre probably thinking, Of course, youd be a match, youre his sister. I assumed so, as well. Read on.

On Aug. 12, 2016, my brother and I underwent our stem cell transplant at Dana Farber/Brigham and Womens Hospital in Boston. There are two different ways to donate stem cells peripheral blood stem cells (stem cells extracted from your blood after receiving five days of injections of a drug called filgrastim, used to increase the number of blood-forming cells in your bloodstream) and bone marrow (a surgical procedure where doctors use needles to withdraw liquid marrow from both sides of the back of your pelvic bone). Due to my brothers specific treatment plan, he required pure bone marrow, and my bone marrow was taken from my pelvis. Two liters worth of my bone marrow was processed at Dana Farber and then brought to my brother immediately, who received it via an IV drip.

So how does my story end? Why am I telling you all this?

My brother is thriving. My pelvis has healed. And we were absolutely blessed to find a match right within our family.

The reality is that fewer than 30 percent of patients with a blood cancer or blood disease will find a related-donor; the other 70 percent, thousands of patients with blood cancers like leukemia and lymphoma, sickle cell anemia or other life-threatening diseases, depend on the national bone marrow registry to find a match to save their life. Some day you or someone you love might depend on a complete stranger who might be a Muslim, a Republican, gay or straight. But it wont matter because from the inside, they will be the same.

I plead with you to remember that we are more alike than we are unalike, and to do something positive for humanity.

You can visit http://www.bethematch.org and join the Be The Match national bone marrow registry.

Or you can attend one of my in-person donor drives in Greenfield over the next few months. The first will be this Saturday, June 10, from 2 to 4 p.m. at the Pints in the Park event at the Greenfield Energy Park.

If you are between the ages of 18 and 44, patients especially need you. You could be someones cure.

I note the obvious differences

between each sort and type,

but we are more alike, my friends,

than we are unalike.

We are more alike, my friends,

than we are unalike.

From Human Family, a poem by Maya Angelou

Ashli Stempel is a Greenfield resident and a member of the Greenfield Town Council.

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Trends in cosmeceutical ingredients – ModernMedicine

By Sykes24Tracey

Whats trending in cosmeceuticals? Niacinamide, heparan sulfate, defensins, novel retinoids and sodium copper chlorophyllin complex, according to Vivian W. Bucay, M.D., a dermatologist in San Antonio, Texas, who presented on the topic this week at the 2017 Vegas Cosmetic Surgery and Aesthetic Dermatology meeting, in Las Vegas.

Niacinamide

Cosmeceuticals contain three primary forms of vitamin B3, including niacinamide, nicotinic acid and nicotinate esters, according to Dr. Bucay.

[The] most studied and efficacious form is niacinamide. Nicotinic acid [is] not generally used because it causes vasodilation, redness and irritation, according to Dr. Bucays presentation. Niacinamide readily penetrates the stratum corneum, [with] little potential for irritation.

Niacinamides effects on the skin include improving the skin barrier function, pigmentation and appearance of lines and wrinkles associated with photoaging. The water-soluble vitamin has been shown in studies to have immunomodulatory and anti-inflammatory effects, and more. Niacinamide has also been shown to improve acne, by, among other things, decreasing facial sebum production. It also has been shown to reduce pigmentation and decrease UV-induced immunosuppression.

Heparan Sulfate

Heparan sulfate is a key glycosaminoglycan, which surrounds cells and is required for cell growth, according to Dr. Bucay.

Heparan sulfate is well documented in dermatology, with studies in wound repair, skin infections, atopic dermatitis, rosacea and psoriasis. As a facilitator of growth factor function, heparan sulfate is essential for collagen synthesis, she writes.

And topically applied heparan sulfate penetrates the epidermis and dermis in humans, according to Dr. Bucay.

Low molecular weight heparan sulfate is found exclusively in the Sent skincare product line (Sent), she writes.

Defensins

According to a study by Lough et al published November 2013, the immune system releases defensins that activate LGR6+ stem cells. Activated LGR6+ stem cells create new basal stem cells. And the newly-created basal stem cells produce fresh keratinocytes that last a lifetime.

Defensins, which are antimicrobial peptides, are released by neutrophils in wounds. These dermatologic stem cells generate cell lineages of the skin, according to Dr. Bucay.

Defensins have been shown to enhance wound healing and hair growth. Early studies suggest defensins reduce skin aging. Dr. Bucay is among the researchers conducting an ongoing vehicle-controlled study on the use of synthetic defensins in a cosmetic base.

While retinol stimulates old basal stem cells to make old keratinocytes, defensins activate preserved LGR6+ stem cells to make new basal stem cells. And while growth factors switch on good and bad cells, defensins activate specific cells types to do specific jobs, according to Dr. Bucay.

Defensins are found in DefenAge (DefenAge) skincare products.

A novel retinoid

Scientists have developed a bioengineered double conjugate retinoid with lactic acid, which is designed to optimize delivery of beneficial properties of both an alpha hydroxy acid (AHA) and a retinoid, with less skin irritation. The proprietary AlphaRet technology increases stability, reduces irritation and improves passage through the skin, according to Dr. Bucays presentation.

The product AlphaRet Overnight Cream (SkinBetter Science), includes the AlphaRet molecule, 0.1%, glycolic acid and a potent antioxidant blend.

Sodium copper chlorophyllin complex

Liposomal sodium copper chlorophyllin complex technology is based on chlorophyll, [a] fat soluble green pigment in plants necessary for photosynthesis, according to Dr. Bucay.

Sodium copper chlorophyllin has a long history of use in medicine, including in topical wound healing. Recently, sodium chlorophyllin has been used topically for cosmetic purposes, in the MDRejuvena Rejuvaphyl Rejuvenating complex (MDRejuvena). The product, she writes, has been shown to reduce redness and oiliness and improve signs of photodamage.

Results from a human biopsy study published July 2016 suggest retinoids and sodium copper chlorophyllin complex have beneficial effects on biomarkers of photoaged skin. Together, sodium copper chlorophyllin complex and retinols may provide a dual approach to reversing age-related changes, according to the authors.

Disclosure: Dr. Bucay reports ties to Allergan, Merz Aesthetics, Galderma, Johnson & Johnson/Neutrogena, LOreal/SkinCeuticals, NuGene, Sent Labs, Medicell Technologies, Alastin Skincare, Viviscal, BTL, Sienna Biopharmaceuticals, Syneron Candela and Miramar Labs.

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Biotech Movers: Shire Falls on Stem Cell Program Transfer; Calithera, Synergy Up on FDA Approvals – TheStreet.com

By NEVAGiles23

Shares of Shire (SHPG) were down $3.22 or 1.85% in early trading Wednesday to $170.88 after the company said it would transfer its U.S. investigational new drug application for for Graft-Versus-Host Disease candidate Alpha-1 Antitrypsin to Kamada (KMDA) . The treatment is aimed at addressing complications fromstem cell or bone marrow transplants.

Kamada is developing the drug in Europe. Kamada shares were down nearly 7% to $7.64.

Calithera Biosciences (CALA) was up to $16.10, a spike of 70 cents or 4.55%, after the FDA designated the company's lead product candidate, CB-839, in combination with Novartis' AFINITOR for Fast Track review for the treatment of metastatic renal cell carcinoma in patients who have received at least two prior lines of therapy.

Shares of Synergy Pharmaceutical (SGYP) rose 4.5% to $4.15 after the FDA accepted for review its supplemental New Drug Application for TRULANCE a candidate to treat irritable bowel syndrome with constipation. The FDA approved TRULANCE to treat chronic idiopathic constipation in January.

Over at Real Money, Bret Jensen looks at 4 Undervalued Biotech Stocks.

Also, Jim Cramer and the AAP team offer up stocks that will allow you to play it safe amid crazy politics.

In terms of volume, Johnson & Johnson (JNJ) and Sanofi (SNY) were among the most actively traded stocks midmorning but were both only down less than a percent.

Exact Sciences (EXAS) was trading at twice its daily volume and saw its shares fall about 6.6%, or $2.40, in early trading Wednesday to $34.19 apiece.

The Madison, Wisc.-basedmolecular diagnostics company said after markets closed on Tuesday that its underwriters, includingJefferies LLC, BofA Merrill Lynch and Robert W. Baird & Co. hadacuired7 million shares of commons stock with an option to buy about 1 million more.

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Biotech Movers: Shire Falls on Stem Cell Program Transfer; Calithera, Synergy Up on FDA Approvals - TheStreet.com

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Major research initiative explores how our bones and muscles age, new ways to block their decline – Medical Xpress

By NEVAGiles23

June 6, 2017 Drs. Carlos Isales, Meghan McGee-Lawrence, William D. Hill and Mark Hamrick. Credit: Phil Jones

With age, the form and function of our bones and muscles drop off, putting us as increased risk for frailty and falls.

Now researchers at the Medical College of Georgia at Augusta University are dissecting just what happens to the stem cells that make the tissues, which help keep us upright, with an eye on improving our healthspan.

Osteoporosis already is a major public health problem affecting about 44 million Americans and costing billions annually. The world's older population is growing at an unprecedented rate with 8.5 percent of the worldwide population - 617 million people - age 65 and older, a proportion estimated to reach 17 percent by 2050, according to the National Institute on Aging.

"After age 65 you start losing about 1 percent of both muscle and bone per year," said Dr. Carlos Isales, endocrinologist, Regents' professor and vice chair for clinical affairs in the MCG Department of Neuroscience and Regenerative Medicine.

"Daily exercise decreases the slope of that decline. But what we are focusing on is trying to see if we can flatten the curve even further," said Isales, principal investigator on a new $9.3 million Program Project grant from the National Institutes of Health.

Time seems to alter the dynamic between the mesenchymal stem cells making bone and muscle and the amino acids that fuel them. The MCG scientists also have evidence it changes the signals stem cells send each other.

The bottom line: Our stem cell population gets reduced and the cells we have become less efficient at making bone and muscle, often opting for the easier task of making fat instead, Isales said.

The team, which includes principal investigators bone biologist Dr. Mark Hamrick, stem cell researcher Dr. William D. Hill and biomedical engineer Dr. Meghan McGee-Lawrence, wants to keep stem cells focused on making bone and muscle.

"We are looking at stem cells as a group and what is happening to them as we age," Hill noted. "This includes a loss of direction so they aren't as functional as they were before. The other thing we are looking at is their survival and their numbers."

"We are trying to figure out why the changes are happening and if we can target those cells to make them want to make bone again," McGee-Lawrence said.

Much as the function of bone and muscle is interwoven, so is their health and the factors that promote their loss or survival also are similar, said Hamrick.

A major culprit in their breakdown appears to be the metabolite kynurenine, a byproduct of the essential amino acid tryptophan. Tryptophan is among the nine amino acids our body can't make and we must consume in foods like turkey and soybeans so we can perform essentials like making protein. The researchers also think the fuel sends signals to cells, ones that aging stem cells apparently don't get.

The unhealthy metabolite is the result of a natural action called oxidation, which occurs anytime cells use oxygen. Particularly with age, the free radicals produced by oxidation can also damage cells. Kynurenine results when the enzyme, indoleamine 2,3 dioxygenase, or IDO, which a variety of tissues make to help moderate an immune response, oxidizes tryptophan. Over time, kynurenine piles up and appears to alter the dynamic of bone and muscle formation.

Again, somewhat ironically, the many functions of essential amino acids include working as antioxidants, so the researchers are putting together nutrient cocktails - minus tryptophan and with reduced protein content - that they hope can reverse age-related damage. Isales notes that they may find that other amino acids produce similar problems as tryptophan in the aged environment.

So they also are taking more direct approaches like whether an IDO inhibitor - which is already in clinical trials as a cancer fighter - can reverse changes and get stem cells to regain more youthful function.

In an effort to begin to see if what they have seen in laboratory mice holds up in humans, they are trying both approaches in human stem cells retrieved during the process of a knee or hip replacement by colleagues in the MCG Department of Orthopaedic Surgery.

They have laboratory evidence that in mice at least, high kynurenine levels impact the ability of cells in the bone marrow to make bone-forming cells called osteoblasts. In fact, even relatively young mice fed kynurenine experience bone loss, an increase in bone destruction by cells called osteoclasts and increased fat in their bone marrow. Conversely, mice with IDO knocked out maintain strong bone mass.

"You can make an old mouse young and you can make a young mouse old," Hill noted.

The team also has evidence that part of how age-related increases in kynurenine does damage is by altering microRNAs - small but powerful pieces of RNA that can control expression of hundreds of genes at the same time - as well as vesicles called exosomes that are hauling the microRNAs around. Stem cells secrete exosomes as one way to communicate, and apparently aging stem cells don't communicate well with each other.

"Exosomes are one mechanism of crosstalk between cells and also between different organs," said Hamrick. "Your liver is producing exosomes, fat produces exosomes, they will hit other organs and they carry, in some cases, positive messages and in some cases bad messages," said Hamrick, who is leading this project to restore positive messaging.

They have laboratory evidence that aging alters at least two microRNAs, miR-141 and miR-183, which prompts cells to make bone-eating instead of bone-forming cells. Again, they have shown that even young stem cells exposed to older exosomes will assume this bone-reducing stance. But they also have some evidence that some of the dietary interventions Isales is looking at could reverse the ill effects.

The team recently reported in the journal Tissue Engineering that exosomes from old and younger mice were similar in size and number and both had a lot of miRNAs. But aged exosomes had significantly and specifically more mi183, an miRNA already associated with cancer. In this case, high mi183 appears to decrease cell proliferation and the ability of immature cells to become bone cells and to support the general deterioration that comes with age, called senescence. Age-related increases of reactive oxygen species and oxidative stress help increase mi183 levels and these undesirable results. When researchers treat mesenchymal stem cells from young animals with exosomes from old mice, is suppresses formation of muscle-making genes; giving mi183 directly to bone and muscle producing cells makes them start acting old. Now they want to know more about how aging changes the secretion and cargo of exosomes by mesynchymal stem cells and how that in turn contributes to bone and muscle loss.

A third project, led by Hill, will focus on the cargo, the miRNAs, to learn more about exactly how they impact bone formation and turnover. "We think that the amino acids are controlling the expression of specific sets of microRNA," Hill said. That means they may want to target and even eliminate key or critical microRNAs, which could obviously affect expression of numerous genes as a result.

They also are exploring aging's impact on stromal cell derived factor 1, or SDF-1, which is critical to helping keep stem cells in the bone marrow and focused on making bone. Age-related changes appear to make SDF-1 instead encourage stem cells to wander. The researchers note that while these cells do often need to leave the bone marrow, to say help heal an injury, these age-related travels are random and often cells don't find their way back. A consistent goal is identifying intervention targets.

"The idea is if we can change the environment and change how they are signaling to themselves and to other cells, we can modify the stem cell directly that way," Hill said.

They are looking upstream as well for earlier points of intervention, including what is happening to histone deacetylase-3, or HDAC3. They have evidence that HDAC3, another pervasive regulator in the body that can turn gene expression up or down, is important in stem cells' age-related propensity to make fat instead of bone.

At least one reason is that reduced HDAC3 means less bone, which literally makes more room for fat, said McGee-Lawrence, who is leading these studies. Her previous studies have shown that when HDAC3 is deleted from the skeleton, bones are weaker, much like what occurs with aging.

Now they have evidence that mice treated with kynurenine, for example, have suppressed HDAC3 expression in the bone. They want to know more about just how HDAC3 gets suppressed as we age and exactly what that does to bone formation and fat storage besides just making room. The new grant is allowing them to put the pieces together better, looking further at just what suppresses HDAC3 and what suppression does to bone versus fat formation. The bottom line again is identifying early points of intervention and potentially nutrients to intervene.

"Something in the microenvironment of the bone is causing the cells, instead of wanting to make bone, they are storing a lot of fat," McGee-Lawrence said. "Some of these epigenetic factors, like HDAC3, some of the environmental factors like changes in the amino acids are causing the cells to dysfunction. We are hoping to figure out what that signal is and how to reverse it and to make those cells want to start making bone again."

Identical twin studies have shown that environmental factors definitely play a role, since the bone/muscle health of these twins often is not identical even though their genes are, Isales said. Rather than changing the genes themselves, environmental factors appear to have changed their expression: which ones are turned or on off. These epigenetic changes include factors from diet to stress to sleep patterns to age.

There are 20 amino acids, which are essential to protein production and a variety of other functions from giving cells structure to helping organs functions. Kyrurenine also is associated with the degeneration of our brain and immune system as we age. Mesynchymal stem cells also produce blood, cartilage and fat cells.

Isales also is vice chair of clinical and translational research in the MCG Department of Orthopaedics and a faculty member in the MCG Department of Medicine. Hamrick, Hill and McGee-Lawrence are all faculty members in the MCG Department of Cellular Biology and Anatomy. Other scientists helping support three core laboratories for the interrelated studies include the Administrative Core with Biostatistics, Maribeth Johnson and Dr. Jie Chen, MCG Department of Biostatics and Epidemiology; the Bone Biology Core, Dr. Mohammed Elsalanty, Department of Oral Biology, Dental College of Georgia at AU; and the Bone Stem Cell Core, Dr. Xingming Shi, MCG Department of Neuroscience and Regenerative Medicine.

Explore further: Non-coding RNA molecule could play a role in osteoporosis

Researchers from Hong Kong Baptist University and colleagues have demonstrated that a molecule called miR-214-3p plays a role in inhibiting bone formation. MiR-214-3p is a microRNA (miRNA): a non-coding RNA involved in regulating ...

An international team of researchers, funded by Morris Animal Foundation, has shown that adipose (fat) stem cells might be the preferred stem cell type for use in canine therapeutic applications, including orthopedic diseases ...

Researchers want to turn back the clock on aging stem cells so they'll make better bone.

Adding just the right mixture of signaling moleculesproteins involved in developmentto human stem cells can coax them to resemble somites, which are groups of cells that give rise to skeletal muscles, bones, and cartilage ...

Imagine you have a bone fracture or a hip replacement, and you need bone to form, but you heal slowly a common fact of life for older people. Instead of forming bone, you could form fat. Researchers at the University ...

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Myelomeningocele is a severe congenital defect in which the backbone and spinal canal do not close before birth, putting those affected at risk of lifelong neurological problems. In a preclinical study published June 6th ...

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3-D skin made of stem cells treats backbone birth defect in rodents – Medical Xpress

By raymumme

June 6, 2017 ISkin (three-dimensional cultured skin) derived from human iPSCs. Immunohistochemical analysis with antibodies to KERATIN 14 (KRT14), p63, cytokeratins (Pan-CK), involucrin, laminin 5, loricrin, KRT10, and filaggrin. The multilayered epidermis expressed KRT14, involucrin, laminin 5, Pan-CK, loricrin, KRT10, and filaggrin in iSkin, indicating that iPSC-keratinocytes terminally differentiate in the skin equivalents. Scale bar is 100 m. Credit: Kazuhiro Kajiwara.

Myelomeningocele is a severe congenital defect in which the backbone and spinal canal do not close before birth, putting those affected at risk of lifelong neurological problems. In a preclinical study published June 6th in Stem Cell Reports, researchers developed a stem cell-based therapy for generating skin grafts to cover myelomeningocele defects before birth. They first generated artificial skin from human induced pluripotent stem cells (iPSCs), and then successfully transplanted the skin grafts into rat fetuses with myelomeningocele.

"We provide preclinical proof of concept for a fetal therapy that could improve outcomes and prevent lifelong complications associated with myelomeningoceleone of the most severe birth defects," says senior study author Akihiro Umezawa of Japan's National Research Institute for Child Health and Development. "Since our fetal cell treatment is minimally invasive, it has the potential to become a much-needed novel treatment for myelomeningocele."

Myelomeningocele, which is the most serious and common form of spina bifida, is a neural tube defect in which the bones of the spine do not completely form. As a result, parts of the spinal cord and nerves come through the open part of the spine. A baby born with this disorder typically has an open area or a fluid-filled sac on the mid to lower back. Most children with this condition are at risk of brain damage because too much fluid builds up in their brains. They also often experience symptoms such as loss of bladder or bowel control, loss of feeling in the legs or feet, and paralysis of the legs.

Babies born with myelomeningocele usually undergo surgery to repair the defect within the first few days of life. Some highly specialized centers also offer intrauterine surgery to close the defect before the baby is born. Although prenatal surgery can improve later neurological outcomes compared with postnatal surgery, it is also associated with higher rates of preterm birth and other serious complications, underscoring the need for safe and effective fetal therapies.

To address this problem, Umezawa and his team set out to develop a minimally invasive approach for generating and transplanting skin grafts that could cover large myelomeningocele defects earlier during pregnancy, potentially improving long-term outcomes while reducing surgical risks. In particular, they were interested in using iPSC technology, which involves genetically reprogramming patients' cells to an embryonic stem cell-like state and then converting these immature cells into specialized cell types found in different parts of the body. This approach avoids ethical concerns while offering the advantages of a potentially unlimited source of various cell types for transplantation, as well as minimal risk of graft rejection by the immune system.

In the new study, the researchers first generated human iPSCs from fetal cells taken from amniotic fluid from two pregnancies with severe fetal disease (Down syndrome and twin-twin3 transfusion syndrome). They then used a chemical cocktail in a novel protocol to turn the iPSCs into skin cells and treated these cells with additional compounds such as epidermal growth factor to promote their growth into multi-layered skin. In total, it took approximately 14 weeks from amniotic fluid preparation to 3D skin generation, which would allow for transplantation to be performed in humans during the therapeutic window of 28-29 weeks of gestation.

Next, the researchers transplanted the 3D skin grafts into 20 rat fetuses through a small incision in the uterine wall. The artificial skin partially covered the myelomeningocele defects in eight of the newborn rats and completely covered the defects in four of the newborn rats, protecting the spinal cord from direct exposure to harmful chemicals in the external environment. Moreover, the engrafted 3D skin regenerated with the growth of the fetus and accelerated skin coverage throughout the pregnancy period. Notably, the transplanted skin cells did not lead to tumor formation, but the treatment significantly decreased birth weight and body length.

"We are encouraged by our results and believe that our fetal stem cell therapy has great potential to become a novel treatment for myelomeningocele," Umezawa says. "However, additional studies in larger animals are needed to demonstrate that our fetal stem cell therapy safely promotes long-term skin regeneration and neurological improvement."

Explore further: Prenatal stem cell treatment improves mobility issues caused by spina bifida

More information: Stem Cell Reports, Kajiwara et al.: "Fetal therapy model of myelomeningocele with three-dimensional skin using amniotic fluid cell-derived induced pluripotent stem cells" http://www.cell.com/stem-cell-reports/fulltext/S2213-6711(17)30220-5 , DOI: 10.1016/j.stemcr.2017.05.013

Journal reference: Stem Cell Reports

Provided by: Cell Press

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3-D skin made of stem cells treats backbone birth defect in rodents - Medical Xpress

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Stem cell-based spinal cord therapy expanded to more patients – The San Diego Union-Tribune

By Sykes24Tracey

An experimental therapy to repair spinal cord injury with stem cell-derived tissue is progressing smoothly, according to a leader of that trial who spoke at a conference on stem cell therapy.

The Phase 1 safety trial is proceeding with no complications, said Dr. Joseph Ciacci, a University of California San Diego neurosurgeon. The trial is being conducted at the universitys Sanford Stem Cell Clinical Center. The conference was held last week at the Sanford Consortium for Regenerative Medicine in La Jolla.

With safety looking good, the green light has been given to treat more patients, Ciacci said. However, to produce effectiveness, more cells will need to be transplanted.

Four patients have been treated with neural stem cells, injected into the spinal cord. They had experienced complete loss of motor and sensor function below the injury. They had been injured between 1 and 2 years previously.

Moreover, the cells show signs of integrating with the surrounding tissue in animal studies, Ciacci said. If the preliminary evidence holds up, Ciacci and colleagues plan to submit a paper detailing the results.

Curing paralysis from spinal cord injury was a big selling point for those who successfully advocated Proposition 71, which authorized selling $6 billion in state bonds to establish and fund the California Institute for Regenerative Medicine, or CIRM. The institute got $3 billion, the remaining half is going for interest over the life of the bonds.

While CIRM has been under pressure to show results, doctors are taking great care to establish safety first in the spinal cord treatment, because of potential risks in the procedure.

We are now enrolling and recruiting for the second cohort, which is for chronic cervical spinal cord injuries, Ciacci said. They are medically classified as C5-C7 ASIA A Complete.

Chronic injuries need to have taken place more than 1 year before treatment. For this study, the injury must also be under two years old. The trial is being conducted at UCSD with Ciacci serving as the principal investigator.

For more information on the Phase I Chronic SCI study, contact Ciaccis research group at (619) 471-3698, nksidhu@ucsd.edu.

In addition, the researchers have been approved to start another spinal cord injury trial with a different set of cells. These oligodendrocyte progenitor cells, derived from embryonic stem cells, can turn into several different types of neural cells.

The trial, sponsored by Asterias, treats newly injured patients, between 14 and 30 days after injury.

For more information on the Asterias trial, contact the UCSD Alpha Stem Cell Clinic at 858-534-5932 alphastemcellclinic@ucsd.edu or visit http://www.scistar-study.com and j.mp/ucsdast.

Asterias acquired the technology from Geron, which had undertaken the work with a CIRM grant. Geron later canceled the work and refunded the money to CIRM. Asterias got funding from CIRM to continue the work.

The Asterias trial will use the same technique as used with the Chronic SCI trial, a technique which can improve safety, Ciacci said. The cells will be injected in a series of progressively larger amounts that may give evidence of the dose relates to effectiveness, although safety remains the main concern.

This cell line is cryopreserved, its sent to us as a single dose the day of surgery, Ciacci said. Were going to study different doses 2 million, 10 million, 20 million cells per injection. Its going to be a direct injection, just like what weve done before.

As in previous treatments, patients will also receive immune suppression to prevent rejection of the cells. Likewise, they will be monitored for many years after treatment.

Another trial coming to UCSD will test for efficacy in ALS, Ciacci said.

Ciacci said hes looking for qualified patients for these trials, and urged those in the audience to help find them.

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bradley.fikes@sduniontribune.com

(619) 293-1020

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Stem cell-based spinal cord therapy expanded to more patients - The San Diego Union-Tribune

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Bioquark aims to bring brain-dead people back to life next year – Metro – Metro

By NEVAGiles23


The Sun
Bioquark aims to bring brain-dead people back to life next year - Metro
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Stem cells regenerate external layer of a human heart – Today’s Medical Developments

By NEVAGiles23

Activating stem cells Wnt signaling pathways can drive cardiac progenitor cells to become epicardium instead of myocardium cells.

A process using human stem cells can generate epicardium cells that cover the external surface of a human heart, according to a multidisciplinary team of researchers.

In 2012, we discovered that if we treated human stem cells with chemicals that sequentially activate and inhibit the Wnt signaling pathway, they become myocardium muscle cells, says Xiaojun Lance Lian, assistant professor of biomedical engineering and biology, who is leading the study at Pennsylvania State University (Penn State). Myocardium, the middle of the hearts three layers, is the thick, muscular part that contracts to drive blood through the body. The Wnt signaling pathway is a group of signal transduction pathways made of proteins that pass signals into a cell using cell-surface receptors.

We needed to provide the cardiac progenitor cells with additional information in order for them to generate into epicardium cells, but prior to this study, we didnt know what that information was, Lian says. Now, we know that if we activate the cells Wnt signaling pathway again, we can re-drive these cardiac progenitor cells to become epicardium cells, instead of myocardium cells.

Lance Lian/Penn State

The groups results bring researchers one step closer to regenerating an entire heart wall. Through morphological assessment and functional assay, the researchers found that the generated epicardium cells were similar to epicardium cells in living humans and those grown in the laboratory.

The last piece is turning cardiac progenitor cells to endocardium cells (the hearts inner layer), and we are making progress on that, Lian says.

The groups method of generating epicardium cells could be useful in clinical applications, for patients who suffer a heart attack.

Heart attacks occur due to blockage of blood vessels, Lian says. This blockage stops nutrients and oxygen from reaching the heart muscle, and muscle cells die. These muscle cells cannot regenerate themselves, so there is permanent damage, which can cause additional problems. These epicardium cells could be transplanted to the patient and potentially repair the damaged region.

In addition to generating the epicardium cells, researchers can keep them proliferating in the lab after treating them with a cell-signaling pathway Transforming Growth Factor Beta (TGF) inhibitor.

After 50 days, our cells did not show any signs of decreased proliferation. However, the proliferation of the control cells without the TGF Beta inhibitor started to plateau after the tenth day, Lian says.

Pennsylvania State University http://www.psu.edu

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SpaceX launches CU-built heart, bone health experiments to space station – CU Boulder Today

By Dr. Matthew Watson

Editors note: The SpaceX Falcon 9 rocket scheduled to launch today from Florida was delayed due to weather conditions. The launch occured on Saturday, June 3.

A SpaceX rocket wasslated to launch two University of Colorado Boulder-built payloads to the International Space Station (ISS) from Florida on Thursday, including oneto look at changes in cardiovascular stem cells in microgravity that may someday help combat heart disease on Earth.

The Dragon spacecraft

The second payload will be used for rodent studies testing a novel treatment for bone loss in space, which has been documented in both astronauts and mice. The two payloads were developed by BioServe Space Technologies, a research center within the Ann and H.J Smead Department of Aerospace Engineering,

We have a solid relationship with SpaceX and NASA that allows us to regularly fly our flight hardware to the International Space Station, said BioServe Director Louis Stodieck. The low gravity of space provides a unique environment for biomedical experiments that cannot be reproduced on Earth, and our faculty, staff and students are very experienced in designing and building custom payloads for our academic, commercial and government partners.

The experiments will be launched on a SpaceX Falcon 9 rocket from Cape Canaveral, Florida, and carried to the ISS on the companys Dragon spacecraft. The SpaceX-CRS-11 mission launching Thursday marks BioServes 55th mission to space.

The cardiovascular cell experiments, designed by Associate Professor Mary Kearns-Jonker of the Loma Linda University School of Medicine in Loma Linda, California, will investigate how low gravity affects stem cells, including physical and molecular changes. While spaceflight is known to affect cardiac cell structure and function, the biological basis for such impacts is not clearly understood, said BioServe Associate director Stefanie Countryman.

As part of the study, the researchers will be comparing changes in heart muscle stem cells in space with similar cells simultaneously cultured on Earth, said Countryman. Researchers are hopeful the findings could help lead to stem cell therapies to repair damaged cardiac tissue. The findings also could confirm suspicions by scientists that microgravity speeds up the aging process, Countryman said.

For the heart cell experiments, BioServe is providing high-tech, cell-culture hardware known as BioCells that will be loaded into shoebox-sized habitats on ISS. The experiments will be housed in BioServes Space Automated Bioproduct Lab (SABL), a newly updated smart incubator that will reduce the time astronauts spend manipulating the experiments.

The second experiment, created by Dr. Chia Soo of the UCLA School of Medicine, will test a new drug designed to not only block loss of bone but also to rebuild it.

The mice will ride in a NASA habitat designed for spaceflight to the ISS. Once on board, some mice will undergo injections with the new drug while others will be given a placebo. At the end of the experiments half of the mice will be returned to Earth in SpaceXs Dragon spacecraft and transported to UCLA for further study, said Stodieck, a scientific co-investigator on the experiment.

BioServes Space Automated Byproduct Lab

In addition to the two science experiments, BioServe is launching its third SABL unit to the ISS. Two SABL units are currently onboard ISS supporting multiple research experiments, including three previous stem cell experiments conducted by BioServe in collaboration with Stanford University, the Mayo Clinic and the University of Minnesota.

The addition of the third SABL unit will expand BioServes capabilities in an era of high-volume science on board the ISS, said Countryman.

BioServe researchers and students have flown hardware and experiments on missions aboard NASA space shuttles, the ISS and on Russian and Japanese government cargo rockets. BioServe previously has flown payloads on commercial cargo rockets developed by both SpaceX, headquartered in Hawthorne, California, and Orbital ATK, Inc. headquartered in Dulles, Virginia.

Since it was founded by NASA in 1987, BioServe has partnered with more than 100 companies and performed dozens of NASA-sponsored investigations. Itspartners include large and small pharmaceutical and biotechnology companies, universities and NASA-funded researchers, and investigations sponsored by the Center for the Advancement of Science in Space, which manages the ISS U.S. National Laboratory. CU-Boulder students are involved in all aspects of BioServe research efforts, said Stodieck.

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SpaceX launches CU-built heart, bone health experiments to space station - CU Boulder Today

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3D-Printed Patch Mends Hearts – Photonics.com

By raymumme

Photonics.com Jun 2017 MINNEAPOLIS, June 6, 2017 A new 3D-laser-printed patch has been developed that can help heal scarred heart tissue after a heart attack.

Researchers from the University of Minnesota-Twin Cities, University of Wisconsin-Madison, and University of Alabama-Birmingham used laser-based 3D bioprinting techniques to incorporate stem cells derived from adult human heart cells on a matrix that began to grow and beat synchronously in a dish in the lab.

"This is a significant step forward in treating the No. 1 cause of death in the U.S.," said Brenda Ogle, an associate professor of biomedical engineering at the University of Minnesota. "We feel that we could scale this up to repair hearts of larger animals and possibly even humans within the next several years."

The patch is modeled after a digital 3D scan of the structural proteins of native heart tissue. It is then made into a physical structure by 3D printing with proteins native to the heart and further integrating cardiac cell types derived from stem cells.

"We were quite surprised by how well it worked, given the complexity of the heart," Ogle said. "We were encouraged to see that the cells had aligned in the scaffold and showed a continuous wave of electrical signal that moved across the patch."

The researchers will soon begin working on a larger patch and testing it on a pig heart, which is similar to a human heart.

The research study is published in the American Heart Association journal Circulation Research (doi: 10.1161/CIRCRESAHA.116.310277).

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Mice with ALS improve with stem cell therapy – The Ledger

By Dr. Matthew Watson

TAMPA Researchers at the University of South Florida show in a new study that bone marrow stem cell transplants helped improve motor functions and nervous system conditions in mice with the disease amyotrophic lateral sclerosis (ALS) by repairing damage to the blood-spinal cord barrier.

In a study recently published in the journal Scientific Reports, researchers in USFs Center of Excellence for Aging and Brain Repair say the results of their experiment are an early step in pursuing stem cells for potential repair of the blood-spinal cord barrier, which has been identified as key in the development of ALS.

USF Health Professor Svitlana Garbuzova-Davis, PhD, led the project.

Using stem cells harvested from human bone marrow, researchers transplanted cells into mice modeling ALS and already showing disease symptoms. The transplanted stem cells differentiated and attached to vascular walls of many capillaries, beginning the process of blood-spinal cord barrier repair.

The stem cell treatment delayed the progression of the disease and led to improved motor function in the mice, as well as increased motor neuron cell survival, the study reported.

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Mice with ALS improve with stem cell therapy - The Ledger

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