By Dennis Thompson HealthDay Reporter

WEDNESDAY, July 23, 2014 (HealthDay News) -- Most blood cancer patients in the United States who need a bone marrow transplant can find an acceptable match through the National Marrow Donor Program, a new study has determined.

Depending on a patient's race or ethnic background, the study found that 66 percent to 97 percent of patients will have a suitably matched and available live donor on the registry.

Even hard-to-match ethnic groups can find a suitable donation thanks to banked stem cells drawn from umbilical cord-blood donations, said senior author Martin Maiers, director of bioinformatics research at the National Marrow Donor Program.

All told, for patients who are candidates for either bone marrow or cord-blood transplants, the likelihood of having a suitable match is as high as 91 to 99 percent, the study found.

"For almost all patients, there is some sort of product available for them," Maiers said.

The findings, said to represent the first attempt to accurately determine the successful-match rate of the bone marrow registry, are published July 24 in the New England Journal of Medicine.

Patients suffering from blood-related cancers such as leukemia or lymphoma need a stem cell transplant to help them survive their cancer treatment. The transplant is done after chemotherapy and radiation is complete.

Donation from a relative is the best option, but only about 30 percent of patients have such a donor available, researchers said in background notes. The majority must rely on the National Marrow Donor Program to match them with a live bone marrow donor or banked stem cells gathered from donated umbilical cord blood.

The National Marrow Donor Program has on hand 11 million potential bone marrow donors and 193,000 banked cord-blood donations. The number of transplants facilitated by the program has quadrupled, with nearly 6,000 transplants in 2012 compared with 1,500 a decade earlier.

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Good Odds for Those Who Need Bone Marrow Donor, Study Finds

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Newswise BOSTON July 24, 2014 Japanese biologist Shinya Yamanaka won a Nobel Prize in 2012 for discovering how to create induced pluripotent stem cells (iPSCs), cells derived from normal adult cells that have the ability to differentiate into almost any other kind of cells. Scientists at Joslin Diabetes Center now have created the first iPSCs that offer a human model of insulin resistance, a key driver of type 2 diabetes.

This is one of the very first studies of human iPSC models for type 2 diabetes, and it points out the power of this technology to look at the nature of diabetes, which is complex and may be different in different individuals, says C. Ronald Kahn, MD, Joslins Chief Academic Officer and the Mary K. Iacocca Professor of Medicine at Harvard Medical School.

Until now, scientists examining the causes and effects of insulin resistance have struggled with a general lack of human cell lines from tissues such as muscle, fat and liver that respond significantly to insulin, Kahn says. Studying insulin resistance as it progresses through pre-clinical stages of type 2 diabetes has been particularly challenging.

There have been no good human cell models to study insulin resistance, but such cells can now be made with iPSCs, says Kahn, co-senior author on a paper about the study published in the journal Diabetes.

Generation of iPSCs typically starts with fibroblasts (connective tissue cells) from skin samples. Kahn and his colleagues used fibroblasts from three patients with severe insulin resistance brought on by mutations in the gene for the insulin receptor (IR)a molecule that crosses the cell membrane and plays a key role in insulin signaling and glucose metabolism.

The Joslin researchers reprogrammed the fibroblasts into iPSCs by using viral procedures that activated four genes that together maintain cells in the iPSC state. The scientists then looked at gene activation in insulin signaling pathways for iPSCs and fibroblasts with IR mutations, and for corresponding cells derived from people without those mutations.

Among the study findings, IR mutations alter expression of many genes both in fibroblasts and iPSCs compared to normal cells, but the impact is very much dependent on the cell type, says Kahn. You see one type of expression pattern in the fibroblasts and a different type of pattern in the iPSCs.

Insulin is a key ingredient for the growth and proliferation of normal stem cells, and the study demonstrated that insulin resistance also reduces the ability of the iPSCs to grow and proliferate. That defect may represent a previously unrecognized mechanism that aids in developing diabetes, Kahn says, as well as helping to explain the problems in wound healing, tissue repair and even beta-cell growth that are common among people with diabetes.

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Joslin Scientists Create the First IPS Cells to Offer Human Model of Insulin Resistance

Stem cell agency President C. Randal Mills (left) and Chairman of the Board Jonathan Thomas.

Responding to his predecessor's ethically controversial departure, the president and chief executive of California's stem cell agency said Thursday he is taking legal steps to minimize conflicts of interests with those who have business before the agency.

C. Randal Mills said he will not take a job with any company funded by the California Institute for Regenerative Medicine for one year after he departs the agency. In addition, he also will not accept gifts or travel payments from any company, institution or person who gets agency funding.

Mills' action, announced at the agency's meeting in Millbrae, will be enforced with a legal agreement he will sign. His action comes less than a month after he replaced Alan Trounson as the agency chief. One week after his departure, CIRM-funded StemCells Inc. announced it had appointed Trounson to its board. StemCells Inc. had received an award of nearly $20 million from the agency to develop a therapy for Alzheimers disease.

While Trounson's appointment wasn't illegal, critics said it was unseemly for him to join a company that had received agency funding so soon after he left CIRM. An ethical controversy could harm the agency's chances of getting more funding from California voters, who gave the agency $3 billion with the passage of Proposition 71 in 2004.

Mills said the new rules apply only to himself, because of his central role at CIRM.

"This specifically addresses an issue where an individual in an organization has a disproportionate amount of power, and I want to make sure it's known that power will not be abused," Mills said.

Mills made the right decision, said Jeanne Loring, a CIRM-funded stem cell researcher at The Scripps Research Institute.

"There's a difference between what is legal and what is ethical," said Loring, who attended the meeting. "And he's going to be pushing the needle a lot more toward the ethical side without worrying whether he can get away with stuff."

John Simpson of Santa Monica-based Consumer Watchdog, who has often criticized CIRM for conflicts of interest, also praised the decision.

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Stem cell agency tightens ethics rules

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WATCH:An 11-year-old girl with a rare blood disease is in need of a stem cell transplant ideally from a match within the South Asian Community. Angie Seth reports.

Stem cell and bone marrow donations are critical for hundreds of people in Canada suffering from certain types of cancers or blood diseases.

Right now there are approximately 800 people on the transplant list. Among them is 11-year-old Cierra Singh.

Cierra has a rare blood disease calledMyelodysplastic Syndrome.

Mybone marrow and my bones are not producing enough healthy cells. So there are platelets and the white blood cells and the red blood cells. My mom tells me they are not working as well as they should work, Cierra tells Global News.

We had the opportunity to meet this incredible little girl who strives to give back to others in every which way.

Everyone says its a big deal, but I dont see it as a big deal. I just try to stay positive all the time, she says.

Cierra was diagnosed with the rare blood disease in April. A trip to Sick Kids hospital because of a swollen leg led doctors to discover Cierras immune system was not functioning properly.

Her Mothers fears paint a bleak picture.

If she were to get a fever of 38.5 and up we need to rush her into emergency within the hour . The risk of infectious diseases is very high so they need to pump her body with antibiotics because she wont be able to fight it. The only cure for Myelodysplastic Syndrome is a stem cell transplant, there is no other option, KiranBenet, Cierras Mom says.

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11-year-olds critical need for a stem cell transplant

PUBLIC RELEASE DATE:

24-Jul-2014

Contact: Vera Siegler vera.siegler@tum.de 49-892-892-2731 Technische Universitaet Muenchen

This news release is available in German.

The immune system has evolved to recognize and respond to threats to health, and to provide life-long memory that prevents recurrent disease. A detailed understanding of the mechanism underlying immunologic memory, however, has remained elusive. Since 2001, various lines of research have converged to support the hypothesis that the persistence of immune memory arises from a reservoir of immune cells with stem-cell-like potential. Until now, there was no conclusive evidence, largely because experiments could only be carried out on populations of cells. This first strict test of the stem cell hypothesis of immune memory was based on mapping the fates of individual T cells and their descendants over several generations.

That experimental capability was developed through a long-term collaboration, focused on clinical cell processing and purification, between researchers based in Munich and Seattle. Since 2009, the groups of Prof. Dirk Busch at the Technische Universitt Mnchen (TUM) and Prof. Stanley Riddell at the Fred Hutchinson Cancer Research Center have combined their technological and clinical expertise under the auspices of the TUM Institute for Advanced Study. The University of Heidelberg, the University of Dsseldorf, the Helmholtz Center Munich, the German Cancer Research Center (DKFZ), and the National Center for Infection Research (DZIF) also contributed to the present study.

Homing In On The "Stemness" of T Cells

After generating an immune response in laboratory animals, TUM researchers Patricia Graef and Veit Buchholz separated complex "killer" T cell populations enlisted to fight the immediate or recurring infection. Within these cell populations, they then identified subgroups and proceeded with a series of single-cell adoptive transfer experiments, in which the aftermath of immune responses could be analyzed in detail. Here the ability to identify and characterize the descendants of individual T cells through several generations was crucial.

The researchers first established that a high potential for expansion and differentiation in a defined subpopulation, called "central memory T cells," does not depend exclusively on any special source such as bone marrow, lymph nodes, or spleen. This supported but did not yet prove the idea that certain central memory T cells are, effectively, adult stem cells. Further experiments, using and comparing both memory T cells and so-called naive T cells that is, mature immune cells that have not yet encountered their antigen enabled the scientists to home in on stem-cell-like characteristics and eliminate other possible explanations.

Step by step, the results strengthened the case that the persistence of immune memory depends on the "stemness" of the subpopulation of T cells termed central memory T cells: Individual central memory T cells proved to be "multipotent," meaning that they can generate diverse types of offspring to fight an infection and to remember the antagonist. Further, these individual T cells self-renew into secondary memory T cells that are, again, multipotent at the single-cell level. And finally, individual descendants of secondary memory T cells are capable of fully restoring the capacity for a normal immune response.

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Experiments prove 'stemness' of individual immune memory cells

Autism Protocol

Current investigative therapies for autism attempt to reverse these abnormalities through administration of antibiotics, antiinflammatory agents, and hyperbaric oxygen. Unfortunately, none of these approaches address the root causes of oxygen deprivation and intestinal inflammation.

Mesenchymal stem cells can regulate the immune system. It is thought that they may help to reverse inflammatory conditions and is currently in the final stages of clinical trials in the US for Crohns disease, a condition resembling the gut inflammation in autistic children.

Through administration of mesenchymal stem cells, we have observed improvement in subjects to whom weve administered stem cells at our facilities. The biological basis for our scientists appears in a peer-reviewed publication Journal of Translational Medicine: Stem Cell Therapy for Autism.

The adult stem cells used in the autism clinical investigation at the Stem Cell Institute come from human umbilical cord tissue (allogeneic mesenchymal). These stem cells are recovered from donated umbilical cords. Before they are approved for use, all umbilical cord-derived stem cells are screened for viruses and bacteria to International Blood Bank Standards. In some cases, we also utilize stem cells harvested from the subjects own bone marrow. Umbilical cord-derived stem cells are ideal for the autism protocol because they allow our physicians to administer uniform doses and they do not require any stem cell collection from the subject, which for autistic children and their parents, can be an arduous process. Because they are collected right after (normal) birth, umbilical cord-derived cells are much more potent than their older counterparts like bone marrow-derived cells for instance. Cord tissue-derived mesenchymal stem cells pose no rejection risk because the body does not recognize them as foreign.

Because HUCT stem cells are less mature than other cells, the bodys immune system is unable to recognize them as foreign and therefore they are not rejected. Weve performed thousands of procedures with umbilical cord stem cells and there has never been a single instance of rejection. HUCT stem cells also proliferate/differentiate more efficiently than older cells, such as those found in the bone marrow and therefore, they are considered to be more potent.

The umbilical cord-derived stem cells are administered intravenously by a licensed physician.

Below is an example of a typical autism schedule. Our investigational clinical protocol for autism (www.clinicaltrials.gov NCT02192749) has been approved by the National Institutional Review Board for Clinical Protocols.

Proper follow-up is an essential part of the autism clinical investigation process. Our primary goal is to ensure that your child is progressing safely. Regular follow-up also enables us to evaluate efficacy and improve our autism clinical protocols based on observed outcomes.

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Stem Cell Therapy for Autism || Treatment Information ...

PUBLIC RELEASE DATE:

24-Jul-2014

Contact: David McKeon dmckeon@nyscf.org 212-365-7440 New York Stem Cell Foundation

NEW YORK, NY (July 24, 2014) Scientists at The New York Stem Cell Foundation (NYSCF) Research Institute are one step closer to creating a viable cell replacement therapy for multiple sclerosis from a patient's own cells.

For the first time, NYSCF scientists generated induced pluripotent stem (iPS) cells lines from skin samples of patients with primary progressive multiple sclerosis and further, they developed an accelerated protocol to induce these stem cells into becoming oligodendrocytes, the myelin-forming cells of the central nervous system implicated in multiple sclerosis and many other diseases.

Existing protocols for producing oligodendrocytes had taken almost half a year to produce, limiting the ability of researchers to conduct their research. This study has cut that time approximately in half, making the ability to utilize these cells in research much more feasible.

Stem cell lines and oligodendrocytes allow researchers to "turn back the clock" and observe how multiple sclerosis develops and progresses, potentially revealing the onset of the disease at a cellular level long before any symptoms are displayed. The improved protocol for deriving oligodendrocyte cells will also provide a platform for disease modeling, drug screening, and for replacing the damaged cells in the brain with healthy cells generated using this method.

"We are so close to finding new treatments and even cures for MS. The enhanced ability to derive the cells implicated in the disease will undoubtedly accelerate research for MS and many other diseases," said Susan L. Solomon, NYSCF Chief Executive Officer.

"We believe that this protocol will help the MS field and the larger scientific community to better understand human oligodendrocyte biology and the process of myelination. This is the first step towards very exciting studies: the ability to generate human oligodendrocytes in large amounts will serve as an unprecedented tool for developing remyelinating strategies and the study of patient-specific cells may shed light on intrinsic pathogenic mechanisms that lead to progressive MS". said Dr. Valentina Fossati, NYSCF Helmsley Investigator and senior author on the paper.

In multiple sclerosis, the protective covering of axons, called myelin, becomes damaged and lost. In this study, the scientists not only improved the protocol for making the myelin-forming cells but they showed that the oligodendrocytes derived from the skin of primary progressive patients are functional, and therefore able to form their own myelin when put into a mouse model. This is an initial step towards developing future autologous cell transplantation therapies in multiple sclerosis patients

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NYSCF scientists one step closer to cell therapy for multiple sclerosis patients

July 24, 2014, 10:29 p.m.

A 60 MINUTES report on new multiple sclerosis stem cell therapy has thrown Wendouree mum Kathryn Johnston a potential lifeline.

A 60 MINUTES report on new multiple sclerosis stem cell therapy has thrown Wendouree mum Kathryn Johnston a potential lifeline.

Hopeful: Wendouree mum Kathryn Johnston is hoping new stem cell therapy treatment will help her be a more active mother to her daughter Dellah, 7. PICTURE: KATE HEALY

Ms Johnston, who has had MS for 15 years, is hoping the treatment will help her be a more active mother to daughter Dellah, 7.

I cant do a great deal with my daughter now but its also the unknown not knowing if Ill wake up one day and not be able to walk, Ms Johnston said.

The 35-year-old emergency nurse hopes to travel to Russia in August next year for the treatment, which involves extracting her stem cells, freezing them while she undergoes a strong course of chemotherapy and then replacing them.

It gets rid of any underlying MS and rebuilds the immune system from scratch. As a general rule, its been about 80 per cent effective.

Ms Johnston first noticed her MS symptoms as an active Ararat 20-year-old doing her nursing degree and about to marry her childhood sweetheart Andrew.

I developed numbness in both hands but thought Id just slept on them until my tummy went numb too.

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MS stem cell therapy treatment hope for mum

Tissue engineering and vascular biology expert Guohao Dai, assistant professor in the Department of Biomedical Engineering at Rensselaer Polytechnic Institute, recently won a Faculty Early Career Development Award (CAREER) from the National Science Foundation (NSF).

Dai will use the five-year, $440,000 grant to advance his research into bio-fabricating human tissues with 3-D cell printing technology. Adult neural stem cells are known to hold a great potential for treating disease and damage to the nervous system. However, these cells are both rare and difficult to use in a laboratory setting. The cells lose their potency quickly upon being removed from their native environment, making it difficult to study them.

With his CAREER Award, Dai seeks to design and develop a new way of using 3-D cell printing technology to create a "vascular niche" that replicates the native environment of adult neural stem cells. With the ability to prolong the potency of the cells and precisely control the parameters and components of its vascular niche, researchers would be better positioned to study the cells and their role in treating treat spinal cord injury and neurodegenerative diseases.

"Adult neural stem cells hold so much promise for treating injury and disease, but they are extremely difficult to work with," Dai said. "We believe that we can apply 3-D tissue printing technology to create a vascular niche that will prolong the life of the cells and, in turn, enable new opportunities for studying how they may be used to treat injury and fight disease."

The CAREER Award is given to faculty members at the beginning of their academic careers and is one of NSF's most competitive awards, placing emphasis on high-quality research and novel education initiatives. Dai will collaborate on his CAREER project with two stem cells experts, Rensselaer Associate Professor of Biomedical Engineering Deanna Thompson and Neural Stem Cell Initiative Scientific Director Sally Temple.

Most laboratory cell cultures are 2-D. This is significantly different from the human body, where most cells are in a 3-D environment. A major challenge in creating and studying 3-D tissues is the diffusion limit in the tissues, which quickly lose potency or die without a flow of blood to provide oxygen and nutrients.

To help overcome this challenge, Dai and his collaborators have spent years developing a 3-D tissue printer -- both the hardware and the software. The unique device prints biological tissue by carefully depositing cells, hydrogels, and other materials one layer at a time. Using this platform, Dai developed the technology to create perfused vascular channels, which provide nutrients and oxygen to the printed tissues.

"Blood vessels run throughout almost every part of our bodies, bringing the oxygen and nutrients that allow our cells to survive. The same is true of 3-D cell cultures. They need a vascular system in order to survive," Dai said. "Our device can print 3-D tissues with small channels that function as blood vessels. This enables us to print cells with extracellular matrices that closely replicate those found within the body."

Dai's research team used the 3-D tissue printing technology to help study how the functions of the vascular endothelium -- a thin layer of cells that line entire circulatory system -- are affected by environmental factors such as interactions with blood and smooth muscle cells. A dysfunctional endothelium is known to be a contributor to many vascular diseases including inflammation, thrombosis, and atherosclerosis.

With his CAREER Award, Dai is applying his expertise and unique 3-D tissue printing technology to replicate the native environment of adult neural stem cells. If successful, the project could significantly expand the potency and life span of the cells in laboratory settings, and lead to a better understanding of how this extracellular environment influences the behavior of the cells.

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3-D-printed tissues advance stem cell research

Renowned Israeli stem-cell researcher in Fairfield Aug. 6

By Cindy Mindell

Dr. Yaqub Hanna

A leading Israeli scientist who has pioneered groundbreaking stem-cell reprogamming research will discuss his work on Wednesday, Aug. 6 at Jewish Senior Services in Fairfield.

Together with a team of researchers at the Weizmann Institute of Science Department of Molecular Genetics in Rehovot, Israel, Dr. Jacob (Yaqub) Hanna has overcome a major roadblock in the use of human stem cells for medical purposes. Funded by a grant from the Israel Cancer Research Fund, their pioneering breakthrough was recently published in the peer-reviewed international science journal, Nature.

Its not only Hannas work that is note-worthy: the award-winning research scientist is a Palestinian living in Israel, a native of Kafr Rama in the Galilee and the son of two medical doctors.

Hanna earned a BS in medical sciences summa cum laude in 2001, an MS in microbiology and immunology in 2003, and a PhD-MD in immunology summa cum laude in 2007, all from the Hebrew University of Jerusalem, where he was among the top five percent of all Israeli medical-school graduates. After completing his PhD, Hanna decided to abandon clinical medicine and focus on research, and spent four years conducting postdoctoral research in the lab, part of the Whitehead Institute for Biomedical Research at MIT.

During his postdoctoral work, Hanna was the first non-American to receive a prestigious Novartis Fellowship from the Helen Hay Whitney Foundation. He joined the Weizmann Institute Department of Molecular Genetics upon his return to Israel in 2011. That year, he received the Clore Prize for distinguished new faculty at the Weizmann Institute and was accepted as a Yigal Alon Program Scholar for junior faculty in Israel. He is also the recipient of the Wolf Foundations Krill Prize for Excellence in Scientific Research and the 2013 Rappaport Prize in Biomedical Research.

Hanna has had to find a way to navigate between his personal and professional identities.

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Stem Cells: Promises and Reality

Surgeons have pioneered a new knee operation that could prevent the development of arthritis and extend sporting careers.

The procedure, which is currently being trialled at Southampton General Hospital, involves coating damaged cartilage with stem cells, taken from a patient's own hip, and surgical glue.

Known as Abicus (Autologous Bone Marrow Implantation of Cells University Hospital Southampton), the technique, if successful, will regenerate the remaining tissue and create a permanent "like-for-like" replacement for the first time.

Cartilage is a tough, flexible tissue that covers the surface of joints and enables bones to slide over one another while reducing friction and acting as a shock absorber.

Damage to the tissue in the knee is common and occurs mainly following sudden twists or direct blows, such as falls or heavy tackles playing sports such as football and rugby, but can also develop over time through gradual wear and tear.

Around 10,000 people a year in the UK suffer cartilage damage serious enough to require treatment due to pain, "locking" and reduced flexibility. If left untreated, it can progress to arthritis and severely impair leg movement.

Currently, the most commonly used procedure to repair the injury - microfracture - involves trimming any remaining damaged tissue and drilling holes in the bone beneath the defect via keyhole surgery to promote bleeding and scar tissue to work as a substitute.

However, the technique has variable results, with studies in the US suggesting the procedure offers only a short term benefit (the first 24 months after surgery), and does not lead to the formation of new cartilage.

Patients who undergo the Abicus operation have the cartilage cut and tidied and undergo microfracture, but their cartilage tissue is then coated with a substance made up of bone marrow cells, platelet gel and hyaluronic acid.

During the 30-minute procedure, the bone marrow sample is spun in a centrifuge in the operating theatre to give a concentrated amount of the patient's own stem cells.

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High hopes for new knee operation

Denver, CO (PRWEB) July 23, 2014

Daily Face & Body is excited to announce that they have released a cheaper and safer alternative to Botox called Stem Cell Technology Facial Serum.

Stem Cell Technology Facial Serum is an anti-aging product used to help people smooth, tone, and rejuvenate dead skin cells..

Stem Cell Technology Facial Serum can be used as a safe alternative to Botox, a popular cosmetic injection, because the Stem Cell does not have any toxins or health risks as opposed to Botox. In addition, it is Alcohol, Ammonia, Paraben, Perfume, and Sulfate free, and it has not been tested on Animals.

According to the Daily Face & Body website, their Stem Cell Technology Facial Serum uses 100% active plant stem cell ingredient (All Even Sweet Iris) which has been clinically tested to reduce wrinkles with overall anti-aging effects.

Jason Palmer, a representative of Daily Face & Body, says that the clinical test results showed that after 28 days of treatment, 84% of women noted their wrinkles seem to have decreased. It also decreased the total surface by 35%, decreased the number of wrinkles by 26%, and decreased the length of wrinkles by 33%.

Ingredients The ingredients in Stem Cell Technology Facial serum are as follows:

Active ingredient: All Even Sweet Irs (Iris pallida). The other ingredients are: Water, Cyclomethicone, Avena sativa (Oat) Kernel Extract, Cichorium Intybus (Chicory) Root, Oligosaccharides (and) Glycerin (and) Caesalpinia Spinosa Gum, Dimethicone, Iris Pallida Leaf Cell Extract, Lauramidoyl Inulin, Oleth-10, Carbomer, Phenoxyethanol (and) Ethylhexylglycerin, Potassium Sorbate, Tromethamine.

About Daily Face & Body is a locally owned Denver company that has been operating since 2012. They sell Skin Care products and accessories as well as home Spa therapy products and weight loss supplements. To receive more information about Daily Face & Body please visit their website http://www.dailyfaceandbody.com.

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Local Denver Skin Care Company Releases Safer Alternative to Botox

New York, NY (PRWEB) July 23, 2014

The New York Stem Cell Foundation (NYSCF) and Beyond Batten Disease Foundation (BBDF) have partnered to develop stem cell resources to investigate and explore new treatments and ultimately find a cure for juvenile Batten disease, a fatal illness affecting children.

NYSCF scientists will create induced pluripotent stem (iPS) cell lines from skin samples of young people affected by juvenile Batten disease as well as unaffected family members. IPS cell lines are produced by artificially turning back the clock on skin cells to a time when they were embryonic-like and capable of becoming any cell in the body. Reprogramming juvenile Batten iPS cells to become brain and heart cells, will provide the infrastructure needed to investigate what is going wrong with the cells adversely affected by the disease. Thus far, efforts to study juvenile Batten disease have been done using rodent models or human skin cells; neither of which accurately mimic the disease in the brain, leaving researchers without proper tools to study the disease or a solid platform for testing drugs that prevent, halt, or reverse its progression. This will be the largest and first genetically diverse collection of human iPS cells for a pediatric brain disease.*

In addition to working with BBDF to actively recruit patients and families to donate skin samples, Batten Disease Support and Research Association (BDSRA) is providing resources and technical support, spreading awareness among academic scientists, and notifying its Pharmaceutical partners. Together, BBDF and BDSRA will ensure that juvenile Batten disease and other researchers are aware of and utilize the 48 stem cell lines resulting from this collaboration to further juvenile Batten disease research worldwide.

We know the genetic mutations associated with juvenile Batten disease. This partnership will result in stem cell models of juvenile Batten, giving researchers an unprecedented look at how the disease develops, speeding research towards a cure, said Susan L. Solomon, NYSCF Chief Executive Officer.

Working with NYSCF to generate functional neuronal subtypes from patients and families is a stellar example of one of our key strategies in the fight against juvenile Batten disease: creating resource technology with the potential to transform juvenile Batten disease research and accelerate our timeline to a cure, said Danielle M. Kerkovich, PhD, BBDF Principal Scientist.

Juvenile Batten disease begins in early childhood between the ages of five and ten. Initial symptoms typically begin with progressive vision loss, followed by personality changes, behavioral problems, and slowed learning. These symptoms are followed by a progressive loss of motor functions, eventually resulting in wheelchair use and premature death. Seizures and psychiatric symptoms can develop at any point in the disease.

Juvenile Batten disease is one disorder in a group of rare, fatal, inherited disorders known as Batten disease. Over 40 different errors (mutations) in the CLN3 segment of DNA (gene) have been attributed to juvenile Batten disease. The pathological hallmark of juvenile Batten is a buildup of lipopigment in the body's tissues. It is not known why lipopigment accumulates or why brain and eventually, heart cells are selectively damaged. It is, however, clear that we need disease-specific tools that reflect human disease in order to figure this out and to build therapy.

NYSCF is a world leader in stem cell research and production with a mission to find cures for the devastating diseases of our time, including juvenile Batten disease. NYSCF has developed the NYSCF Global Stem Cell ArrayTM, an automated robotic technology that standardizes and scales stem cell production and differentiation, enabling the manufacture and analysis of large numbers of identical cells from skin samples of patients. The Array technology allows for the production of large-scale iPS cells that have the potential to become any cell type in the body.

This collaboration brings together the expertise of these two leading non-profit organizations, the support of BDSRA, and the participation of affected families, to create and make available to researchers, juvenile Batten disease iPS cell lines. Building on the NYSCF Research Institutes leading stem cell expertise and unique automated technology and analytics, while taking advantage of the tremendous resources and expertise of BBDF, BDSRA and affected families, this collaboration will move research

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The New York Stem Cell Foundation Partners With Beyond Batten Disease Foundation to Fight Juvenile Batten Disease

PUBLIC RELEASE DATE:

23-Jul-2014

Contact: David McKeon dmckeon@nyscf.org 212-365-7440 New York Stem Cell Foundation

NEW YORK, NY -- The New York Stem Cell Foundation (NYSCF) and Beyond Batten Disease Foundation (BBDF) have partnered to develop stem cell resources to investigate and explore new treatments and ultimately find a cure for juvenile Batten disease, a fatal illness affecting children.

NYSCF scientists will create induced pluripotent stem (iPS) cell lines from skin samples of young people affected by juvenile Batten disease as well as unaffected family members. IPS cell lines are produced by artificially "turning back the clock" on skin cells to a time when they were embryonic-like and capable of becoming any cell in the body. Reprogramming juvenile Batten iPS cells to become brain and heart cells will provide the infrastructure needed to investigate what is going wrong with the cells adversely affected by the disease. Thus far, efforts to study juvenile Batten disease have been done using rodent models or human skin cells, neither of which accurately mimic the disease in the brain, leaving researchers without proper tools to study the disease or a solid platform for testing drugs that prevent, halt, or reverse its progression. This will be the largest and first genetically diverse collection of human iPS cells for a pediatric brain disease.

In addition to working with BBDF to actively recruit patients and families to donate skin samples, Batten Disease Support and Research Association (BDSRA) is providing resources and technical support, spreading awareness among academic scientists, and notifying its Pharmaceutical partners. Together, BBDF and BDSRA will ensure that juvenile Batten disease and other researchers are aware of and utilize the 48 stem cell lines resulting from this collaboration to further juvenile Batten disease research worldwide.

"We know the genetic mutations associated with juvenile Batten disease. This partnership will result in stem cell models of juvenile Batten, giving researchers an unprecedented look at how the disease develops, speeding research towards a cure," said Susan L. Solomon, NYSCF Chief Executive Officer.

"Working with NYSCF to generate functional neuronal subtypes from patients and families is a stellar example of one of our key strategies in the fight against juvenile Batten disease: creating resource technology with the potential to transform juvenile Batten disease research and accelerate our timeline to a cure," said Danielle M. Kerkovich, PhD, BBDF Principal Scientist.

Juvenile Batten disease begins in early childhood between the ages of five and ten. Initial symptoms typically begin with progressive vision loss, followed by personality changes, behavioral problems, and slowed learning. These symptoms are followed by a progressive loss of motor functions, eventually resulting in wheelchair use and premature death. Seizures and psychiatric symptoms can develop at any point in the disease.

Juvenile Batten disease is one disorder in a group of rare, fatal, inherited disorders known as Batten disease. Over 40 different errors (mutations) in the CLN3 segment of DNA (gene) have been attributed to juvenile Batten disease. The pathological hallmark of juvenile Batten is a buildup of lipopigment in the body's tissues. It is not known why lipopigment accumulates or why brain and eventually, heart cells are selectively damaged. It is, however, clear that we need disease-specific tools that reflect human disease in order to figure this out and to build therapy.

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NYSCF partners with Beyond Batten Disease Foundation to fight juvenile Batten disease

La Jolla, CA (PRWEB) July 23, 2014

StemGenex, the leading resource for adult adipose stem cell therapy in the US aimed at improving the lives of patients dealing with degenerative diseases today announced their newest clinical study for Parkinsons disease. StemGenex believes that a commitment to the safety and efficacy of stem cell therapy are paramount when providing care to patients with degenerative diseases.

This clinical study makes stem cell therapy accessible to the millions of individuals currently living with Parkinsons disease. The protocol used in these stem cell treatments is unique to StemGenex, having the possibility of being more effective than other stem cell treatments currently available. StemGenex has developed a multiple administration protocol for patients suffering from Parkinsons disease which includes targeted methods of stem cell delivery. Among these methods is a novel approach for delivering stem cells past the blood brain barrier an issue most stem cell treatments have been challenged by.

Principal Investigator Dr. Jeremiah McDole, Ph.D. stated, As is the case with most neurodegenerative conditions, there are few available drugs to treat Parkinsons disease. The handful of drugs that are available can only ameliorate symptoms and unfortunately, prolonged usage can create terrible side-effects. Further, these drugs do not halt disease progression or aid in the repair of established damage. Our goal is to provide regenerative medicine applications that address these critical issues. The study we are conducting is designed to provide us with a large amount of rigorously collected data so that we can better understand the clinical benefit of Parkinsons patients treated with stem cells.

This study is registered through The National Institutes of Health which can be found at http://www.clinicaltrials.gov and is being conducted under IRB approval. According to StemGenex Director of Patient Advocacy, Joe Perricone, It is important patients have access to top-tier stem cell therapy. By providing access to registered clinical studies through The National Institutes of Health, we are providing patients with the ability to choose a stem cell treatment center with the highest standard of care.

Rita Alexander, founder and president of StemGenex stated, Parkinson's disease affects a very small part of the brain but anyone suffering with this disease understands the negative impact on his or her life is very big, actually, enormous. Over the last several years we have observed significant improvement in the symptoms of Parkinsons patients through stem cell treatment. We are determined to be part of the solution and are eager to document and publish our findings in the next few years.

Stem cell treatment studies are currently being offered by StemGenex to patients diagnosed with Parkinsons disease and other degenerative neurological diseases. StemGenex takes a unique approach of compassion and empowerment while providing access to the latest stem cell therapies for degenerative neurological diseases including Multiple Sclerosis, Alzheimers disease, stroke recovery and others.

To find out more about stem cell therapy, contact StemGenex either by phone at (800) 609-7795 or email Contact(at)stemgenex(dot)com.

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StemGenex Gives Hope to Parkinsons Patients through New Stem Cell Clinical Study

The First Minister has given his support to the Teles campaign for bone marrow donors.

Alex Salmond urged people to consider joining the national bone marrow registers after he heard about Menzieshill baby Faith Cushnies cancer battle.

Mr Salmond said: Faiths story highlights the need for more bone marrow donors, and I commend the Evening Telegraph for their excellent campaign.

It is vital that those willing to donate their blood stem cells or bone marrow sign up to the Anthony Nolan Trust and British Bone Marrow Registers, to help people who desperately need lifesaving transplants.

I would encourage everyone eligible to consider saving lives by joining the register.

More than 180 people from Tayside have registered to be donors with the bone marrow and stem cell charity Anthony Nolan since the Tele published Faiths story on Tuesday.

The nine-month-old, needed a bone marrow donation to beat leukaemia, but after her donor backed out she relapsed before a replacement could be found.

Doctors have told Faiths devastated parents there is now nothing they can do for her.

Every year around 1,800 people in the UK need a bone marrow or stem cell transplant to treat cancers such as leukaemia, lymphoma and myeloma and blood disorders like sickle cell disease.

Bone marrow, the spongy tissue found inside some bones, contains stem cells that produce blood cells to carry oxygen around the body, fight infection and stop bleeding.

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Alex Salmond says Yes to the Teles bone marrow campaign

Patients' virus levels became undetectable after a bone-marrow therapy with stem cells

Scanning electron microscope (SEM) image of a lymphocyte with HIV cluster. Credit: National Cancer Institute via Wikimedia Commons

Scientists have uncovered two new cases of HIV patients in whom the virus has become undetectable.

The two patients, both Australian men, became apparently HIV-free after receiving stem cells to treat cancer. They are still on antiretroviral therapy (ART) as a precaution, but those drugs alone could not be responsible for bringing the virus to such low levels, says David Cooper, director of the Kirby Institute at the University of New South Wales in Sydney, who led the discovery. A year ago, a different group of researchers had reported cases with a similar outcome.

Cooper presented details of the cases today at a press briefing in Melbourne, Australia, where delegates are convening for next week's 20th International AIDS Conference. The announcement came just a day after the news that at least six people heading to the conference died when aMalaysia Airlines flight was shot down in Ukraine.

Cooper began searching for patients who had been purged of the HIV virus after attending a presentation by a US team last year at a conference of the International AIDS Society in Kuala Lumpur. At that meeting, researchers from Brigham and Womens Hospital in Boston, Massachusetts, reported that two patients who had received stem-cell transplants were virus-free.

Cooper and his collaborators scanned the archives of St Vincents hospital in Sydney, one of the largest bone-marrow centres in Australia. We went back and looked whether we had transplanted [on] any HIV-positive patients, and found these two, says Cooper.

The first patient had received a bone-marrow transplant for non-Hodgkin's lymphoma in 2011. His replacement stem cells came from a donor who carried one copy of a gene thought to afford protection against the virus. The other had been treated for leukaemia in 2012.

Unfortunately, several months after the 'Boston' patients stopped taking ART,the virus returned. An infant born with HIV in Mississippi who received antiretroviral therapy soon after birth, then stopped it for more than three years,was thought to have been cured, buthas had the virus rebound, too.

Natural resistance At the moment, there is only one person in the world who is still considered cured of HIV:Timothy Ray Brown, the 'Berlin patient', who received a bone-marrow transplant and has had no signs of the virus in his blood for six years without ART. The bone marrow received by the Berlin patient came from a donor who happened to have a natural genetic resistance to his strain of HIV.

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HIV Cleared in 2 Patients via Cancer Treatment

Scientists have discovered a new way to make human platelets, which could help patients worldwide who need blood transfusions.

Platelets are the cells we use to form blood clots. They're traditionally created in our bone marrow. But scientists are now using a machine called a platelet bioreactoralong with human stem cells to create platelets outside the human body.(ViaYouTube / ThrombosisAdviser,American Society of Hematology)

Essentially, this"next-generation"device asBoston Magazinecalls it features the same characteristics asbone marrow. The crucial difference: It's able to carry out a reaction on an industrial scale.

An author of the study said in a press release published byHealthDay,"The ability to generate an alternative source of functional human platelets with virtually no disease transmission represents a paradigm shift in how we collect platelets that may allow us to meet the growing need for blood transfusions."

Brigham and Women's Hospital reports more than 2 million donor platelet units are transfused each year in the U.S. to help patients in need.

That includestrauma patients and those undergoing chemotherapy, organ transplants and surgery. (Getty Images)

But platelet shortages are common due to increased demand, a short shelflife and the possibility of contamination, rejection and infection. (Getty Images)

The problem lab-created platelets have runinto in the past istime: Growing new platelets took too long.

A doctor not associated with this researchsaid,"This study addresses that gap, while contributing to our understanding of platelet biology at the same time."(ViaHealthDay /Brigham and Women's Hospital)

Butthe rules are tough on blood products, so the platelets will undergo safety tests over the next three years. Clinical human trials likely won't start until 2017. (Getty Images)

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Scientists find new way to make human platelets

Scientists have already successfully coaxed stem cells into becoming red blood cells, which could be used to create "man-made" blood for transfusion. Red blood cells, however, aren't the only component of human blood. Now, researchers at Harvard-affiliated Brigham and Womens Hospital have also created functional human platelets, using a bioreactor that simulates the medium in which blood cells are naturally produced bone marrow.

The main role of platelets (also known as thrombocytes) is to stop wounds from bleeding, by essentially "plugging the hole" in the skin with a clot. Without sufficient numbers of them in the blood, spontaneous and excessive bleeding can occur. Such shortages can be caused by diseases, as a result of undergoing chemotherapy, or by other factors. In these situations, transfusions of platelets harvested from donated blood are often necessary.

In previous studies, scientists have successfully gotten induced pluripotent stem cells to change into megakaryocytes these are the cells that ordinarily sit in the bone marrow and release platelets into the bloodstream. Unfortunately, it's proven difficult to get those lab-grown megakaryocytes to produce platelets outside of the body.

That's where Brigham and Womens new "bioreactor-on-a-chip" comes into the picture. By mimicking bone marrow's extracellular matrix composition, stiffness, micro-channel size and shear forces, it persuades the megakaryocytes to produce anywhere from 10 to 90 percent more platelets than was previously possible.

It is hoped that once the technology is scaled up, platelets made with it could be used to address shortages of donated natural platelets, and to minimize the risk of diseases being transmitted between donors and recipients. Human clinical trials are planned to begin in 2017.

The research was led by Dr. Jonathan Thon, and is described in a paper recently published in the journal Blood.

Source: Brigham and Womens Hospital

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Human blood platelets grown in bone marrow-replicating bioreactor