LOS ANGELES (KABC) --

While Roeuy Garay was pregnant with her daughter Brook, she felt weak and an unusual back pain. Her doctors thought it was just part of the pregnancy. But a few weeks after her delivery, her fiance Joseph knew something was seriously wrong.

"I passed out and he took me to urgent care and said, 'Something is wrong with her. It's got to be her kidney or something. We need to do some blood tests,'" Roeuy said.

A bone biopsy and body scan revealed a diagnosis the 36-year-old Corona mother of four could not believe.

"They came in and said, 'Yeah, you have multiple myeloma, and it's about between 70 to 80 percent of your blood is cancer,'" she said.

Multiple myeloma, also called Kahler's disease, is a cancer of the plasma cells, which are in the blood stream. Her best chance at survival is a bone marrow transplant.

None of her siblings were a match and being of Cambodian descent, Roeuy's odds of finding a match are very slim. It's a fact that is hard to hide from her children.

There are 12 million people in the National Bone Marrow Registry, but only 7 percent are Asian and only a small fraction of that are Southeast Asian.

Dr. Elizabeth Budde with City of Hope National Medical Center in Duarte said it only takes a cheek swab to be part of the registry and donating stem cells can be as easy as donating blood.

For now, Roeuy is in remission so she needs a match as soon as possible.

See original here:
Mother of 4 seeks bone marrow match

LOS ANGELES (KABC) --

While Roeuy Garay was pregnant with her daughter Brook, she felt weak and an unusual back pain. Her doctors thought it was just part of the pregnancy. But a few weeks after her delivery, her fiance Joseph knew something was seriously wrong.

"I passed out and he took me to urgent care and said, 'Something is wrong with her. It's got to be her kidney or something. We need to do some blood tests,'" Roeuy said.

A bone biopsy and body scan revealed a diagnosis the 36-year-old Corona mother of four could not believe.

"They came in and said, 'Yeah, you have multiple myeloma, and it's about between 70 to 80 percent of your blood is cancer,'" she said.

Multiple myeloma, also called Kahler's disease, is a cancer of the plasma cells, which are in the blood stream. Her best chance at survival is a bone marrow transplant.

None of her siblings were a match and being of Cambodian descent, Roeuy's odds of finding a match are very slim. It's a fact that is hard to hide from her children.

There are 12 million people in the National Bone Marrow Registry, but only 7 percent are Asian and only a small fraction of that are Southeast Asian.

Dr. Elizabeth Budde with City of Hope National Medical Center in Duarte said it only takes a cheek swab to be part of the registry and donating stem cells can be as easy as donating blood.

For now, Roeuy is in remission so she needs a match as soon as possible.

Excerpt from:
Corona mother of 4 seeks bone marrow match

Inside the microscopic world of the mouse hair follicle, Yale Cancer Center researchers have discovered big clues about how stem cells regenerate and die. These findings, published April 6 in the journal Nature, could lead to a better understanding of how the stem cell pool is maintained or altered in tissues throughout the body.

Stem cells are undifferentiated cells that replenish themselves and, based on their tissue location, can become specialized cells such as blood or skin cells. The hair follicle is an ideal site for exploring stem cell behavior because it has distinct and predictable oscillations in the number and behavior of stem cells, said the studys lead author, Kailin R. Mesa, a third-year doctoral student in the lab of Valentina Greco, associate professor of genetics, cell biology, and dermatology.

Using live microscopic imaging to track stem cell behavior in the skin of living mice, researchers observed that the stem cell niche, or surrounding area, plays a critical role in whether stem cells grow or die.

Prior to this, it wasnt clear whether stem cell regulation was intrinsic or extrinsic, and now we know it is external in that the niche instructs the stem cells, Mesa said. In terms of cancer, we can next explore how we might perturb or change the niche in hopes of affecting the growth of cancer stem cells.

Also, researchers were surprised to find that the stem cells within the pool fed on other dying stem cells. This reveals a mechanism for removing dead cells, a process previously observed in mammary glands but never in the skin.

This study was supported by the Yale Dermatology Spore, National Institutes of Health, American Cancer Society, and New York Stem Cell Foundation.

Citation: Nature

(Photo via Shutterstock)

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Research in the News: Tiny hair follicle offers big clues about the life and death of stem cells

IMAGE:These are examples of 3-D neural tissue construct designs. 3D tissue and organoid models will provide incredible new tools and insights into neurological injury and disease, as well as great... view more

Credit: Richard McMurtrey / Institute of Neural Regeneration & Tissue Engineering

It is well known that neurological diseases and injuries pose some of the greatest challenges in modern medicine, with few if any options for effectively treating such diagnoses, but recent work suggests a unique approach for reconstructing damaged neural tissue. In an article published in the journal Neural Regeneration Research, several new designs for 3D tissue constructs are described for using stem cells grown on nanofiber scaffolding within a supportive hydrogel.

"The idea that neural structure can be guided in three dimensional hydrogels using nanofiber scaffolding and biochemical cues is quite unique," said Dr. Richard McMurtrey, the author of the work. "Evidence from in vitro work thus far has been fairly surprising, showing that after only a few days neurons can grow long neurite extensions that track along the coated nanofibers."

The tissue constructs have been designed for guidance of neural connections, acting like a road map for the growth of the neurons. "One of the weaknesses with prior studies of stem cell implantation into the nervous system is that no guidance is given for what the cells should do once they are implanted," says McMurtrey. "But if we combine signaling molecules and three-dimensional topographical guidance along with the stem cells, the chances of the cells achieving their intended function is much greater." Dr. McMurtrey likens the transplantation of cells into the harsh environment of the nervous system to dropping people off in the mountains with no resources and hoping that they form a functional civilization. "What we hope to do, however, is build some of the roads, bridges, street signs, and homes that can guide and protect the cells when they are transplanted. In this case, that infrastructure includes nanofibers, biochemical cues, and hydrogel composites."

Tissue at its most basic level is made of two parts: cells and the matrix outside of cells called the extracellular matrix. The approach discussed in the article seeks to provide both of these components for more complete reconstruction of the tissue. "The idea that neurons need scaffolding guidance along with biochemical signals is not entirely foreign," McMurtrey says. "During early development, precursor cells that will become neurons must migrate along a sort of scaffolding of radial glial fibers in the nervous system, and it is during this process that many anatomical pathways and lines of communication between neurons form." The materials used in building these constructs are compatible with implantation into the tissue of the brain and spinal cord and will biodegrade after a few weeks to months. It is hoped that this will give just enough time to help the implanted cells integrate into the nervous system.

Many challenges are expected in the development and implementation of this technology. Nevertheless, there is reason for optimism Dr. McMurtrey says: "Scientists must have a bit of skepticism," he says, "but they also need to have vision to try things that haven't been done before. Prior studies have implanted cells in hydrogels without patterned scaffolding and demonstrated better cell survival than when cells were implanted alone, so the idea of combining patterned and functionalized nanofiber scaffolds within protective hydrogels really makes a lot of sense. We know there will be challenges along the way, but we hope to be able to anticipate and overcome the difficulties that will likely arise. In many ways, this may be like the search for an ideal light bulb--we are looking for the right combinations of nanofiber filaments, hydrogel polymers, and molecular signals that will enable implanted neural cells to connect and communicate across lesions of neural tissue."

Much more study will be needed before a patient's own stem cells can be used clinically for things like spinal cord injury, stroke, or neurodegenerative disease, but the first implantation of a patient's own reprogrammed stem cells has recently been performed for a patient with macular degeneration in Japan in 2014 as a collaboration of researchers at RIKEN, one of the world's leading stem cell research centers. Dr. McMurtrey says that much more research and funding would be necessary to bring guided cell therapies into clinical use for neurological diseases, and even then it would not likely be a perfect cure. "The structure and function of the nervous system is more complex than anything else in the universe," says McMurtrey, "so this is not just something like rewiring a circuit board; rather, what we are doing is laying carefully designed pathways through space that neurons can use to reconnect relay centers, but the patient will still have to learn how to use and adapt to these new connections."

The technology may also have many applications apart from just regenerative medicine. These applications include constructing and studying simple artificial neural networks, testing new drugs, and investigating models of human neurological diseases on tissue-like structures in a dish. The Institute has successfully created 3D neural structures from a patient's skin cells that were reprogrammed into stem cells ("induced pluripotent stem cells") and then transformed into 3D neural tissue analogs, which opens up numerous possibilities for exploring complex neurological processes and diseases in human cells rather than in animal models.

"We hope that this approach will give us new capabilities to guide neural extensions, to study neural functions, and ultimately to achieve functional reconstruction of neural architecture in the brain and spinal cord. Henry David Thoreau wrote that 'We are all sculptors and painters, and our material is our own flesh and blood.' In clinical medicine, the protocols are all spelled out, but there are many diseases and limitations in medicine that move you, that frustrate you, and that inspire you, and I think this is the pice de rsistance, if you will. Just the chance that this work might help alleviate the long-term suffering of so many people with neurological injuries makes it a privilege to be part of such an endeavor despite all the challenges."

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New advancements in 3-D designs for neural tissue engineering

Miami, FL (PRWEB) April 06, 2015

Regenestem Network, a subsidiary of the Global Stem Cells Group, has announced plans to attend the 23rd Annual World Congress on Anti-Aging Medicine (a4m) at the Diplomat Resort and Spa in Hollywood, Fla. Hosted by the American Academy of Anti-aging Medicine, the conference will be attended by physicians and medical practitioners from around the world.

Regenestem Network plans to showcase its upcoming stem cell training course, Adipose Derived and Bone Marrow Stem Cell course, with classes scheduled to be held May 9-10 and June 15-16, 2015 in Miami. The intensive, two-day course covers the latest technology and procedures in adipose and bone marrow stem cell therapies. Participants learn skills that can be used in their own practice and for career advancement.

A4m Conference Keynote speakers include Daniel G. Amen, MD, David Perlmutter, MD, FACN, ABIHM, and Gary Small, MD. All three will focus on disease prevention and optimized health through a proactive treatment approach. These world-renown speakers are scheduled to deliver insightful presentations, the latest research and breakthrough therapies in anti-aging medicine.

To learn more about the 23rd Annual World Congress on Anti-Aging Medicine, visit the a4m website. For more information on the Regenestem Network, visit the website at regenestemnetwork.com. For more information on the stem cell training classes, visit the http://www.stemcelltraining.net website, email bnovas(at)regenestem(dot)com, or call 849.943.2988.

About Regenestem Network:

Regenestem Network, a division of the Global Stem Cells Group, Inc., is an international medical practice association committed to researching and producing comprehensive stem cell treatments for patients worldwide. Having assembled a highly qualified staff of medical specialistsprofessionals trained in the latest cutting-edge techniques in cellular medicineRegenestem continues to be a leader in delivering the latest protocols in the adult stem cell arena. Global Stem Cells Group and Regenestem Network are expanding the companys clinical presence worldwide by partnering with experienced and qualified regenerative medicine physicians to open new clinics licensed and developed under the Regenestem banner. In 2014, Global Stem Cells Group expanded the Regenestem Networks global presence to 20 countries.

Regenestem offers stem cell treatments to help treat a variety of diseases and conditions including arthritis, autism, chronic obstructive pulmonary disease (COPD), diabetes, and pain due to injuries at various facilities worldwide. Regenestem Oaxaca will have an international staff experienced in administering the latest in cellular therapies.

Regenestem is certified for the medical tourism market, and staff physicians are board-certified or board-eligible. Regenestem clinics provide services in more than 10 specialties, attracting patients from the United States and around the world.

About the Global Stem Cell Group:

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Regenestem Network Announces Plans to Attend the 23rd Annual World Congress on Anti-Aging Medicine May 7-9, 2015

Lifeline Skin Care is stem cell skin care -- the only skin care products in the world based on non-embryonic Human Stem Cell Extracts. These extracts from highly-potent stem cells -- the same stem cells active early in life -- will stimulate your skins own abilities to repair itself and create smooth, beautiful skin. Non-embryonic stem cells means no embryos are created or destroyed.

Stem Cell Extracts Help New Skin Begin Lifeline stem cell skin care uniquely helps your skin build millions of new, young, healthy skin cells. You restore volume and fullness, you fill in lines around the eyes and mouth, and help reverse the damage caused by the sun's UV rays. It's literally "out with the old skin cells, in with the new, young, healthy skin cells."

What Specific Results Does Lifeline Provide? Stem cells are special cells that go to work whenever your skin needs to repair itself. But they become less potent with age. Lifeline Skin Care has discovered a patented, ethical way to take extracts from non-embryonic human stem cells, which then help create millions of new skin cells that rejuvenate skin. An independent clinical study showed the following results:

improve skin hydration by 93% increase skin elasticity by 73% decrease wrinkles appearance by 67% improved skin tone and brightness by 63%

Profits from Lifeline Skin Care are applied to fund stem cell research for curing degenerative diseases like diabetes and Parkinson's Disease, diseases of the liver, and blinding diseases of the eye.

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Lifeline Stem Cell Skin Care | Lifeline Skin Care Coupon ...

Degenerated/herniated lumbar discs 1 year after stem cell therapy by Harry Adelson, N.D.
Bill describes his result one year after bone marrow stem cell therapy by Dr. Harry Adelson for low back pain caused by a degenerated and herniated lumbar disc.

By: Harry Adelson, N.D.

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Degenerated/herniated lumbar discs 1 year after stem cell therapy by Harry Adelson, N.D. - Video

Life-changing results: Sandra Sharman is a private stem cell patient. Photo: Meredith O'Shea

Last week, Suzie Palmer, 44, travelled from her home in NSW to the Gold Coast for her second round of stem cell treatments for multiple sclerosis. OnTuesday morning,the wheelchair-bound poet underwent liposuction.

By 2.30pm, stem cells had been partially separated from her abdominal fat, suspended in plasma, and injected intravenously. Her doctor, Soraya Felix, is a cosmetic surgeon and molecular biologist with a sideline in regenerative medicine.

Palmer, a relentlessly upbeat and positive person, says the treatments have helped her cope better with heat, improved her mobility and flexibility and otherwise made her "feel like a normal human being". She has, she says, managed a few steps with a walker, still a long way from "running about, which is my dream".

Poster girl: Suzie Palmer is undergoing stem cell therapy for MS. Photo: Edwina Pickles

The rapidly growing stem cell industry is aglow with similarly positive testimonials, notably on behalf of practitioners who offer little documented scientific evidence of their success.

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Suzie Palmer is literally the poster girl for stem cell tourism within Australia. You can find her smiling sweetly, along with Dr Felix, on the Facebook page of a group called the Adult Stem Cell Foundation. She is one of an unknown number of unwell Australians pinning their hopes on an unregulated industry that is now under review by the Therapeutic Goods Administration.

The TGA public consultation, which closed earlier this month, was prompted by long-standing concerns raised by Stem Cells Australia that a loophole in the regulations has allowed dozens of doctors across Australia to provide experimental treatments without the ethics committee oversight that registered clinical trials are subject to. These treatments invariably cost $10,000 and up. The loophole is this: while the use of donor stem cells in therapies is tightly regulated, the use of a patient's own stem cells is not.

Professor Martin Pera is the program leader of Stem Cells Australia, which is administered by the University of Melbourne and includes scientists from Monash University, the Walter and Eliza Hall Institute for Medical Research, the Florey Institute and the CSIRO, among others. They are engaged in a seven-year Australian Research Council project to answer the big questions about stem cells and the potential for reliable therapies.

Read more from the original source:
Is a loophole in stem cell law helping new therapy to thrive, or allowing dubious science?

CIRM funds many projects seeking to better understand heart disease and to translate those discoveries into new therapies.

If you want to learn more about CIRM funding decisions or make a comment directly to our board, join us at a public meeting. You can find agendas for upcoming public meetings on our meetings page.

Find Out More: Stem Cell FAQ | Stem Cell Videos | What We Fund

Find clinical trials: CIRM does not track stem cell clinical trials. If you or a family member is interested in participating in a clinical trial, please visit clinicaltrials.gov to find a trial near you.

Heart disease strikes in many forms, but collectively it causes one third of all deaths in the U.S. Many forms of heart disease have a common resultcardiomyopathy. While this is commonly called congestive heart failure (CHF), it is really just the heart becoming less efficient due to any number of causes, but the most common is loss of functioning heart muscle due to the damage caused by a heart attack. An estimated 4.8 million Americans have CHF, with 400,000 new cases diagnosed each year. Half die within five years.

Numerous clinical trials are underway testing a type of stem cell found in borne marrow, called mesenchymal stem cells or MSCs, to see if they are effective in treating the form of CHF that follows a heart attack. While those trials have shown some small improvements in patients the researchers have not found that the MSCs are creating replacement heart muscle. They think the improvements may be due to the MSCs creating new blood vessels that then help make the existing heart muscle healthier, or in other ways strengthening the existing tissue.

Californias stem cell agency has numerous awards looking into heart disease (the full list is below). Most of these involve looking for ways to create stem cells that can replace the damaged heart muscle, restoring the hearts ability to efficiently pump blood around the body. Some researchers are looking to go beyond transplanting cells into the heart and are instead exploring the use of tissue engineering technologies, such as building artificial scaffolds in the lab and loading them with stem cells that, when placed in the heart, may stimulate the recovery of the muscle.

Other CIRM-funded researchers are working in the laboratory, looking at stem cells from heart disease patients to better understand the disease and even using those models to discover and test new drugs to see if they are effective in treating heart disease. Other researchers are trying to make a type of specialized heart cell called a pacemaker cell, which helps keep a proper rhythm to the hearts beat.

We also fund projects that are trying to take promising therapies out of the laboratory and closer to being tested in people. These Disease Team Awards encourage the creation of teams that have both the scientific knowledge and business skills needed to produce therapies that can get approval from the Food and Drug Administration (FDA) to be tested in people. In some cases, these awards also fund the early phase clinical trials to show that they are safe to use and, in some cases, show some signs of being effective.

This team developed a way to isolate some heart-specific stem cells that are found in adult heart muscle. They use clumps of cells called Cardiospheres to reduce scarring caused by heart attacks. Initially they used cells obtained from the patients own heart but they later developed methods to obtain the cells they need from donor organs, which allows the procedure to become an off-the-shelf-therapy, meaning it can be available when and where the patient needs it rather than having to create it new each time. The company, working with the Cedars-Sinai team, received FDA approval to begin a clinical trial in June 2012.

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Heart Disease Fact Sheet | California's Stem Cell Agency

According to the Christopher and Dana Reeve Foundation, nearly one in 50 people are living with paralysis.

Until now, there wasn't much hope.

But, a new study involving stem cells has doctors and patients excited.

Two years ago, Brenda Guerra's life changed forever.

"They told me that I went into a ditch and was ejected out of the vehicle," says Brenda.

The accident left the 26-year-old paralyzed from the waist down and confined to a wheelchair.

"I don't feel any of my lower body at all," says Brenda.

Brenda has traveled from Kansas to UC San Diego to be the first patient to participate in a ground-breaking safety trial, testing stem cells for paralysis.

"We are directly injecting the stem cells into the spine," says Dr. Joseph Ciacci, a neurosurgeon at UC San Diego.

The stem cells come from fetal spinal cords. The idea is when they're transplanted they will develop into new neurons and bridge the gap created by the injury by replacing severed or lost nerve connections. They did that in animals and doctors are hoping for similar results in humans. The ultimate goal: to help people like Brenda walk again.

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Stem cell procedures for paralysis patients

Children's Healthcare of Atlanta and Winship Cancer Institute target graft-versus-host-disease through immune cell therapy

An innovative clinical trial using the science of "personalized" cellular therapy has begun enrolling children and adults suffering from graft-versus-host-disease (GVHD), a life-threatening complication of bone marrow transplantation in which donor immune lymphocytes attack the organs of the bone marrow transplant recipient.

Bone marrow transplantation is performed in some patients with cancers of the blood or bone marrow, including multiple myeloma and leukemia, as well as in some patients with sickle cell disease, thallesemia, aplastic anemia and inherited immune deficiency.

Physician-researchers at the Aflac Cancer and Blood Disorders Center at Children's Healthcare of Atlanta and Winship Cancer Institute of Emory University will harvest bone marrow cells from children and adults (12 to 65 years) with GVHD. Those cells will be used to manufacture large numbers of personalized autologous marrow mesenchymal stromal cells in the Emory Personalized Immunotherapy Center (EPIC), a dedicated pharmaceutical grade facility located within Emory University Hospital.

By infusing large doses of these personalized bone marrow cells into bone marrow transplant recipients, the physician-researchers aim to target sites of inflammation, potentially reducing GVHD in the intestine, liver and skin and limiting long-term organ damage.

Muna Qayed, MD, MSc. a pediatric hematologist-oncologist at the Aflac Cancer Center at Children's and an assistant professor at Emory School of Medicine, will lead the clinical trial, which is offered only in Atlanta and is supported by CURE Childhood Cancer.

"For patients with GVHD who do not respond to first line therapy, there is no reliable cure, and GVHD can be life threatening or a life-long disabling condition," says Dr. Qayed, "But we hope that through our clinical research, we will be able to significantly impact the course of this disease."

"This trial represents one of the most innovative clinical trials to arise from the growing partnership between the Hematology & Medical Oncology and Pediatrics departments at Emory School of Medicine, Emory Healthcare, and Children's Healthcare of Atlanta," says William (Bill) G. Woods, MD, director of the Aflac Cancer Center.

Blood and bone marrow cells have been used for more than a quarter century to treat life-threatening hematological conditions and are now used in established therapies worldwide. The current clinical trial will use mesenchymal stromal cells from the bone marrow. These cells have been studied more recently for treatment of a wide array of diseases, including autoimmune diseases.

"The beginning of this clinical trial is the culmination of two years' of collaborative effort by a terrific multidisciplinary team at Emory Healthcare, Children's Healthcare of Atlanta and the Aflac Cancer Center," says Edmund Waller, MD, director of Winship's Bone Marrow and Stem Cell Transplant Program and investigator on this trial.

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Clinical trial uses patients' own cells for treatment after bone marrow transplant

This Saturday when the University of Kentucky basketball team faces off with the University of Wisconsin in the NCAA tournament semi-finals, die-hard Kentucky fan Scott Logdon may think twice about rooting against the Wisconsin Badgers.

Nearly two years ago, Logdon was given a life-saving donation of stem cells that helped combat his acute myeloid leukemia. The donor of those cells turned out to be 22-year-old Chris Wirz, a student at the University of Wisconsin.

Logdon, 44, learned the identity of his donor last April, more than a year after the stem cell treatment and just days after the University of Kentucky squeaked past the University of Wisconsin at the NCAA semi-finals with a score of 74 to 73.

Logdon remembers feeling mixed emotions when the Kentucky wildcats won. Later, when he found out about his donor, he joked, That must have been the Badger blood in me.

Courtesy Angela Logdon

PHOTO: Chris Wirz gave life saving stem cells to Scott Logdon, who was suffering from leukemia.

Logdons ordeal started in the fall of 2012, when he was diagnosed with acute myeloid leukemia after mistaking early symptoms for strep throat. Logdon said his doctors told him chemotherapy could only keep the cancer at bay. A full stem cell transplant would be needed to cure him of the deadly disease.

Logdons doctors hoped one of his two siblings might be a match, but neither was able to donate. Longons family and community rallied in the small town of Saldasia, Kentucky, and registered over 120 people who would be willing to donate stem cells or bone marrow.

But no one who registered was a good match for Logdon.

[The doctors] went to the national bone marrow registry to try and find the match, the father of four said. I had to go back to the hospital every 30 days [for] maintenance chemo; it was a very long wait.

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Kentucky Fan Gets Life-Saving Stem Cell Donation From Univ. of Wisconsin Student

CHICAGO, April 2, 2015 /PRNewswire-USNewswire/ --After surgery failed to relieve extreme pain caused by peripheral artery disease in her right leg, Denise Hopkins-Glover was facing a bleak outlook she might never walk again.

"They said they had done everything they could and the only option was amputation of the right leg from the knee down," she said.

Undeterred, Hopkins-Glover chose to participate in an investigational trial at Northwestern Medicine called the MOBILE Study, which makes use of a device called the MarrowStim PAD Kit. In the trial, a randomized group of patients receive injections of their own stem cells retrieved through a bone marrow extraction to try to restore blood flow to the leg.

"MarrowStim offers a new approach for patients with a grim prognosis," said principal investigator Melina Kibbe, MD, a vascular surgeon at Northwestern Memorial Hospital and Edward G. Elcock Professor of Surgical Research at Northwestern University Feinberg School of Medicine. "We're pleased to be part of this national trial to see if there might be a significant chance of improving treatment for patients with few choices left for treatment."

Hopkins-Glover, a 55-year-old grandmother of two, suffers from peripheral artery disease (PAD), a condition affecting 20 percent of Americans where cholesterol and fatty plaque pool in blood vessels, restricting blood flow to the limbs. In its most severe form, PAD causes critical limb ischemia (CLI), which can cause pain in resting legs, sores or ulcers that don't heal, thickening of the toenails and gangrene, which can eventually lead to amputation.

The Chicago resident worked as a phlebotomist before her PAD worsened, and had to stop working because she could no longer walk or stand for extended stretches of time.

"I can walk only a certain distance before the circulation stops getting to certain parts of the body," she said. "It feels like a terrible leg cramp, like a jabbing, stabbing pain."

During the procedure, patients are put under general anesthesia as bone marrow is harvested through a needle from the hip. The bone marrow is loaded into the MarrowStim PAD Kit, an investigational device, where it is processed in a centrifuge. This spinning separates the marrow into different layers, with one of the layers containing the stem cells. Immediately following the separation, the stem cells are injected in 40 different spots on the affected limb, delivering concentrated bone marrow in each one. The entire procedure takes about 90 minutes. Patients follow up with investigators at different intervals in the year following the injections.

Karen Ho, MD, a Northwestern Medicine vascular surgeon who is also an investigator on the trial, said the exact reason the bone marrow injections might help chronic limb ischemia is still a mystery.

"Nobody really knows the exact mechanism," said Dr. Ho, who is also an assistant professor in vascular surgery at Feinberg. "The idea is that it might improve or enhance new blood vessels in the calf."

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Northwestern Medicine Investigates Using Stem Cells to Save Limbs from Amputation

Using patient-derived stem cells known as induced pluripotent stem cells (iPSC) to study the genetic lung/liver disease called alpha-1 antitrypsin (AAT) deficiency, researchers have for the first time created a disease signature that may help explain how abnormal protein leads to liver disease.

The study, which appears in Stem Cell Reports, also found that liver cells derived from AAT deficient iPSCs are more sensitive to drugs that cause liver toxicity than liver cells derived from normal iPSCs. This finding may ultimately lead to new treatments for the condition.

IPSC's are derived from the donated skin or blood cells of adults and, with the reactivation of four genes, are reprogrammed back to an embryonic stem cell-like state. Like embryonic stem cells, iPSC can be differentiated toward any cell type in the body, but they do not require the use of embryos. Alpha-1 antitrypsin deficiency is a common genetic cause of both liver and lung disease affecting an estimated 3.4 million people worldwide.

Researchers from the Center for Regenerative Medicine (CReM) at Boston University and Boston Medical Center (BMC) worked for several years in collaboration with Dr. Paul Gadue and his group from Children's Hospital of Philadelphia to create iPSC from patients with and without AAT deficiency. They then exposed these cells to certain growth factors in-vitro to cause them to turn into liver-like cells, in a process that mimics embryonic development. Then the researchers studied these "iPSC-hepatic cells" and found the diseased cells secrete AAT protein more slowly than normal cells. This finding demonstrated that the iPSC model recapitulates a critical aspect of the disease as it occurs in patients. AAT deficiency is caused by a mutation of a single DNA base. Correcting this single base back to the normal sequence fixed the abnormal secretion.

"We found that these corrected cells had a normal secretion kinetic when compared with their diseased, parental cells that are otherwise genetically identical except for this single DNA base," explained lead author Andrew A. Wilson, MD, assistant professor of medicine at Boston University School of Medicine and Director of the Alpha-1 Center at Bu and BMC.

They also found the diseased (AAT deficient) iPSC-liver cells were more sensitive to certain drugs (experience increased toxicity) than those from normal individuals. "This is important because it suggests that the livers of actual patients with this disease might be more sensitive in the same way," said Wilson, who is also a physician in pulmonary, critical care and allergy medicine at BMC.

According to Wilson, while some patients are often advised by their physicians to avoid these types of drugs, these recommendations are not based on solid scientific evidence. "This approach might now be used to generate that sort of evidence to guide clinical decisions," he added.

The researchers believe that studies using patient-derived stem cells will allow them to better understand how patients with AAT deficiency develop liver disease. "We hope that the insights we gain from these studies will result in the discovery of new potential treatments for affected patients in the near future," said Wilson.

Story Source:

The above story is based on materials provided by Boston University Medical Center. Note: Materials may be edited for content and length.

Continued here:
iPSC model helps to better understand genetic lung/liver disease

(Boston)--Using patient-derived stem cells known as induced pluripotent stem cells (iPSC) to study the genetic lung/liver disease called alpha-1 antitrypsin (AAT) deficiency, researchers have for the first time created a disease signature that may help explain how abnormal protein leads to liver disease.

The study, which appears in Stem Cell Reports, also found that liver cells derived from AAT deficient iPSCs are more sensitive to drugs that cause liver toxicity than liver cells derived from normal iPSCs. This finding may ultimately lead to new treatments for the condition.

IPSC's are derived from the donated skin or blood cells of adults and, with the reactivation of four genes, are reprogrammed back to an embryonic stem cell-like state. Like embryonic stem cells, iPSC can be differentiated toward any cell type in the body, but they do not require the use of embryos. Alpha-1 antitrypsin deficiency is a common genetic cause of both liver and lung disease affecting an estimated 3.4 million people worldwide.

Researchers from the Center for Regenerative Medicine (CReM) at Boston University and Boston Medical Center (BMC) worked for several years in collaboration with Dr. Paul Gadue and his group from Children's Hospital of Philadelphia to create iPSC from patients with and without AAT deficiency. They then exposed these cells to certain growth factors in-vitro to cause them to turn into liver-like cells, in a process that mimics embryonic development. Then the researchers studied these "iPSC-hepatic cells" and found the diseased cells secrete AAT protein more slowly than normal cells. This finding demonstrated that the iPSC model recapitulates a critical aspect of the disease as it occurs in patients. AAT deficiency is caused by a mutation of a single DNA base. Correcting this single base back to the normal sequence fixed the abnormal secretion.

"We found that these corrected cells had a normal secretion kinetic when compared with their diseased, parental cells that are otherwise genetically identical except for this single DNA base," explained lead author Andrew A. Wilson, MD, assistant professor of medicine at Boston University School of Medicine and Director of the Alpha-1 Center at Bu and BMC.

They also found the diseased (AAT deficient) iPSC-liver cells were more sensitive to certain drugs (experience increased toxicity) than those from normal individuals. "This is important because it suggests that the livers of actual patients with this disease might be more sensitive in the same way," said Wilson, who is also a physician in pulmonary, critical care and allergy medicine at BMC.

According to Wilson, while some patients are often advised by their physicians to avoid these types of drugs, these recommendations are not based on solid scientific evidence. "This approach might now be used to generate that sort of evidence to guide clinical decisions," he added.

The researchers believe that studies using patient-derived stem cells will allow them to better understand how patients with AAT deficiency develop liver disease. "We hope that the insights we gain from these studies will result in the discovery of new potential treatments for affected patients in the near future," said Wilson.

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Funding was provided by an ARRA stimulus grant (1RC2HL101535-01) awarded by the National Institutes of Health (NIH) to Boston University School of Medicine, Boston Medical Center and the Children's Hospital of Philadelphia. Additional funding was provided by K08 HL103771, FAMRI 062572_YCSA, an Alpha-1 Foundation Research Grant and a Boston University Department of Medicine Career Investment Award. Additional grants from NIH 1R01HL095993 and 1R01HL108678 and an ARC award from the Evans Center for Interdisciplinary Research at Boston University supported this work.

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Researchers produce iPSC model to better understand genetic lung/liver disease

Researchers map chromosomal changes that must take place before stem cells can be used to produce pancreatic and liver cells

IMAGE:These are pancreatic cells derived from embryonic stem cells. view more

Credit: UC San Diego School of Medicine

Stem cells hold great promise for treating a number of diseases, in part because they have the unique ability to differentiate, specializing into any one of the hundreds of cell types that comprise the human body. Harnessing this potential, though, is difficult. In some cases, it takes up to seven carefully orchestrated steps of adding certain growth factors at specific times to coax stem cells into the desired cell type. Even then, cells of the intestine, liver and pancreas are notoriously difficult to produce from stem cells. Writing in Cell Stem Cell April 2, researchers at University of California, San Diego School of Medicine have discovered why.

It turns out that the chromosomes in laboratory stem cells open slowly over time, in the same sequence that occurs during embryonic development. It isn't until certain chromosomal regions have acquired the "open" state that they are able to respond to added growth factors and become liver or pancreatic cells. This new understanding, say researchers, will help spur advancements in stem cell research and the development of new cell therapies for diseases of the liver and pancreas, such as type 1 diabetes.

"Our ability to generate liver and pancreatic cells from stem cells has fallen behind the advances we've made for other cell types," said Maike Sander, MD, professor of pediatrics and cellular and molecular medicine and director of the Pediatric Diabetes Research Center at UC San Diego. "So we haven't yet been able to do things like test new drugs on stem cell-derived liver and pancreatic cells. What we have learned is that if we want to make specific cells from stem cells, we need ways to predict how those cells and their chromosomes will respond to the growth factors."

Sander led the study, together with co-senior author Bing Ren, PhD, professor of cellular and molecular medicine at UC San Diego and Ludwig Cancer Research member.

Chromosomes are the structures formed by tightly wound and packed DNA. Humans have 46 chromosomes - 23 inherited from each parent. Sander, Ren and their teams first made maps of chromosomal modifications over time, as embryonic stem cells differentiated through several different developmental intermediates on their way to becoming pancreatic and liver cells. Then, in analyzing these maps, they discovered links between the accessibility (openness) of certain regions of the chromosome and what they call developmental competence - the ability of the cell to respond to triggers like added growth factors.

"We're also finding that these chromosomal regions that need to open before a stem cell can fully differentiate are linked to regions where there are variations in certain disease states," Sander says.

In other words, if a person were to inherit a genetic variation in one of these chromosomal regions and his or her chromosome didn't open up at exactly the right time, he or she could hypothetically be more susceptible to a disease affecting that cell type. Sander's team is now working to further investigate what role, if any, these chromosomal regions and their variations play in diabetes.

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'Open' stem cell chromosomes reveal new possibilities for diabetes

DALLAS, April 2, 2015 /PRNewswire/ --

Lifescienceindustryresearch.com adds "Complete 2015-16 Induced Pluripotent Stem Cell (iPSC) Industry Report" in its store. Recent months have seen the first iPSC clinical trial in humans, creation of the world's largest iPSC Biobank, major funding awards, a historic challenge to the "Yamanaka Patent", a Supreme Court ruling affecting industry patent rights, the announcement of an iPSC cellular therapy clinic scheduled to open in 2019, and much more. Furthermore, iPSC patent dominance continues to cluster in specific geographic regions, while clinical trial and scientific publication trends give clear indicators of what may happen in the industry in 2015 and beyond.

Is it worth it to get informed about rapidly-evolving market conditions and identify key industry trends that will give an advantage over the competition?

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Induced Pluripotent Stem Cell (iPSC) Industry Complete Report 2015 - 2016

Damage to neural tissue is typically permanent and causes lasting disability in patients, but a new approach has recently been discovered that holds incredible potential to reconstruct neural tissue at high resolution in three dimensions. Research recently published in the Journal of Neural Engineering demonstrated a method for embedding scaffolding of patterned nanofibers within three-dimensional (3D) hydrogel structures, and it was shown that neurite outgrowth from neurons in the hydrogel followed the nanofiber scaffolding by tracking directly along the nanofibers, particularly when the nanofibers were coated with a type of cell adhesion molecule called laminin. It was also shown that the coated nanofibers significantly enhanced the length of growing neurites, and that the type of hydrogel could significantly affect the extent to which the neurites tracked the nanofibers.

"Neural stem cells hold incredible potential for restoring damaged cells in the nervous system, and 3D reconstruction of neural tissue is essential for replicating the complex anatomical structure and function of the brain and spinal cord," said Dr. McMurtrey, author of the study and director of the research institute that led this work. "So it was thought that the combination of induced neuronal cells with micropatterned biomaterials might enable unique advantages in 3D cultures, and this research showed that not only can neuronal cells be cultured in 3D conformations, but the direction and pattern of neurite outgrowth can be guided and controlled using relatively simple combinations of structural cues and biochemical signaling factors."

The next step will be replicating more complex structures using a patient's own induced stem cells to reconstruct damaged or diseased sites in the nervous system. These 3D reconstructions can then be used to implant into the damaged areas of neural tissue to help reconstruct specific neuroanatomical structures and integrate with the proper neural circuitry in order to restore function. Successful restoration of function would require training of the new neural circuitry over time, but by selecting the proper neurons and forming them into native architecture, implanted neural stem cells would have a much higher chance of providing successful outcomes. The scaffolding and hydrogel materials are biocompatible and biodegradable, and the hydrogels can also help to maintain the microstructure of implanted cells and prevent them from washing away in the cerebrospinal fluid that surrounds the brain and spinal cord.

McMurtrey also noted that by making these site-specific reconstructions of neural tissue, not only can neural architecture be rebuilt, but researchers can also make models for studying disease mechanisms and developmental processes just by using skin cells that are induced into pluripotent stem cells and into neurons from patients with a variety of diseases and conditions. "The 3D constructs enable a realistic replication of the innate cellular environment and also enable study of diseased human neurons without needing to biopsy neurons from affected patients and without needing to make animal models that can fail to replicate the full array of features seen in humans," said McMurtrey.

The ability to engineer neural tissue from stem cells and biomaterials holds great potential for regenerative medicine. The combination of stem cells, functionalized hydrogel architecture, and patterned and functionalized nanofiber scaffolding enables the formation of unique 3D tissue constructs, and these engineered constructs offer important applications in brain and spinal cord tissue that has been damaged by trauma, stroke, or degeneration. In particular, this work may one day help in the restoration of functional neuroanatomical pathways and structures at sites of spinal cord injury, traumatic brain injury, tumor resection, stroke, or neurodegenerative diseases of Parkinson's, Huntington's, Alzheimer's, or amyotrophic lateral sclerosis.

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The work was carried out at the University of Oxford and the Institute of Neural Regeneration & Tissue Engineering, a non-profit charitable research organization.

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

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3-D neural structure guided with biocompatible nanofiber scaffolds and hydrogels

CardioCel has been initially well received with surgeons in Australia and overseas. Photo: Geoff Fisher

For 10 years researchers at Admedus worked day and night trying to work out how to improve soft tissue repair in the human body.

And with the vital help of CSIRO they have been to develop CardioCel, a life-saving heart patch for the repair and reconstruction of cardiovascular defects.

According to the Children's Heart Foundation, congenital heart disease occurs in one out of 100 births and in at least half of those cases surgery is required and a patch is needed. They state it is the leading cause of birth defect related deaths.

Research undertaken with CSIRO investigated new, potentially ground-breaking applications for CardioCel. The research focused on using stem cells. It found the heart patch has the potential to deliver stem cells and help tissue heal better than other existing products, when used for cardiac repair.

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Derived from animal tissue, the CardioCel patch is engineered over 14 days.

"The first unique feature of this product is that it doesn't calcify in young patients," Professor Leon Neethling, Admedus technical director and heart researcher says.

The flexible patch works like human tissue to cover holes in the heart thereby eliminating the need for repeat surgery.

"In the cardiac repair field it has long been recognised that strong, flexible, biocompatible and calcification-resistant tissue scaffolds would be ideal tissues for repair of heart defects," Admedus' chief operating officer Dr Julian Chick, says.

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Local innovation repairs holes in the heart

Low doses of the anti-cancer drug imatinib can spur the bone marrow to produce more innate immune cells to fight against bacterial infections, Emory researchers have found.

The results were published March 30, 2015 in the journal PLOS Pathogens.

The findings suggest imatinib, known commercially as Gleevec , or related drugs could help doctors treat a wide variety of infections, including those that are resistant to antibiotics, or in patients who have weakened immune systems. The research was performed in mice and on human bone marrow cells in vitro, but provides information on how to dose imatinib for new clinical applications.

"We think that low doses of imatinib are mimicking 'emergency hematopoiesis,' a normal early response to infection," says senior author Daniel Kalman, PhD, professor of pathology and laboratory medicine at Emory University School of Medicine.

Imatinib, is an example of a "targeted therapy" against certain types of cancer. It,blocks tyrosine kinase enzymes, which are dysregulated in cancers such as chronic myelogenous leukemia and gastrointestinal stromal tumors.

Imatinib also inhibits normal forms of these enzymes that are found in healthy cells. Several pathogens - both bacteria and viruses - exploit these enzymes as they transit into, through, or out of human cells. Researchers have previously found that imatinib or related drugs can inhibit infection of cells by pathogens that are very different from each other, including tuberculosis bacteria and Ebola virus.

In the new PLOS Pathogens paper, Emory investigators show that imatinib can push the immune system to combat a variety of bacteria, even those that do not exploit Abl enzymes. The drug does so by stimulating the bone marrow to make more neutrophils and macrophages, immune cells that are important for resisting bacterial infection.

"This was surprising because there are reports that imatinib can be immunosuppressive in some patients," Kalman says. "Our data suggest that at sub-clinical doses, imatinib can stimulate bone marrow stem cells to produce several types of myeloid cells, such as neutrophils and macrophages, and trigger their exodus from the bone marrow. However, higher doses appear to inhibit this process."

The authors note that imatinib appears to stimulate several types of white blood cells, which may provide a limit on inflammation, rather than increasing neutrophils only, which can be harmful. The authors go on to suggest that imatinib or related drugs may be useful in treating a variety of infections in patients whose immune system is compromised, such as those receiving chemotherapy for cancer.

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Anticancer drug can spur immune system to fight infection