The growing number of biological structures being grown on chips in various laboratories around the world is rapidly replicating the entire gamut of major human organs. Now one of the most important of all a viable functioning heart has been added to that list by researchers at the University of California at Berkeley (UC Berkeley) who have taken adult stem cells and grown a lattice of pulsing human heart tissue on a silicon device.

Sourced from human-induced pluripotent stem cells able to be persuaded into forming many different types of tissue, the human heart device cells are not simply separate groups of cells existing in a petri dish, but a connected series of living cells molded into a structure that is able to beat and react just like the real thing.

"This system is not a simple cell culture where tissue is being bathed in a static bath of liquid," said study lead author Anurag Mathur, a postdoctoral researcher at UC Berkeley. "We designed this system so that it is dynamic; it replicates how tissue in our bodies actually gets exposed to nutrients and drugs."

Touted as a possible replacement for living animal hearts in drug-safety screening, the ability to easily access and rapidly analyze a heart equivalent in experiments presents appealing advantages.

"Ultimately, these chips could replace the use of animals to screen drugs for safety and efficacy," said professor of bioengineering at UC Berkeley, and leader of the research team, Kevin Healy.

The cardiac microphysiological system, as the team calls its heart-on-a-chip, has been designed so that its silicon support structure is equivalent to the arrangement and positioning of conjoining tissue filaments in a human heart. To this supporting arrangement, the researchers loaded the engineered human heart cells into the priming tube, whose cone-shaped funnel assisted in aligning the cells in a number of layers and in one direction.

In this setup, the team created microfluidic channels on each side of the cell holding region to replicate blood vessels to imitate the interchange of nutrients and drugs by diffusion in human tissue. The researchers believe that this arrangement may also one day provide the ability to view and gauge the expulsion of metabolic waste from the cells in future experiments.

"Many cardiovascular drugs target those channels, so these differences often result in inefficient and costly experiments that do not provide accurate answers about the toxicity of a drug in humans," said Professor Healy. "It takes about US$5 billion on average to develop a drug, and 60 percent of that figure comes from upfront costs in the research and development phase. Using a well-designed model of a human organ could significantly cut the cost and time of bringing a new drug to market."

The use of animal organs to forecast human reactions to new drugs is problematic, the UC Berkeley researchers note, citing the fundamental differences between species as being responsible for high failure rates in using these models. One aspect responsible for this failure is to be found in the difference in the ion channel structure between human and other animals where heart cells conduct electrical currents at different rates and intensities. It is the standardized nature of using actual human heart cells that the team sees as the heart-on-a-chip's distinct advantage over animal models.

The UC Berkeley device is certainly not the first replication of an organ-on-a-chip, but potentially one of the first successful ones to integrate living cells and artificial structures in a single functioning unit. Harvard's spleen-on-a-chip, for example, replicates the operation of the spleen, but does so by using a set of circulatory tubes containing magnetic nanobeads.

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Heart-on-a-chip beats a steady rhythm

WASHINGTON: Researchers, including one of Indian-origin, have created a 'heart-on-a-chip' loaded with human cardiac muscle cells that mimic the real organ to serve as a novel tool to screen medicines. Researchers developed a network of pulsating cardiac muscle cells housed in an inch-long silicone de-vice that effectively models human heart tissue, and they have demonstrated the viability of this system as a drug-screening tool by testing it with cardiovascular medications.

This organ-on-a-chip represents a major step forward in the development of accurate, faster methods of testing for drug toxicity, researchers said. "Ultimately, these chips could replace the use of animals to screen drugs for safety and efficacy," said professor Kevin Healy from the University of California, Berkeley. The authors noted a high failure rate associated with the use of non-human animal models to predict human reactions to new drugs.

"It takes about 5 billion on average to develop a drug, and 60% of that figure comes from upfront costs in the research and development phase. Using a well-designed model of a human organ could significantly cut the cost and time of bringing a new drug to market," said Healy.

The heart cells were derived from human-induced pluripotent stem cells, the adult stem cells that can be coaxed to become many different types of tissue. Researchers designed their heart-on-a-chip so that its 3D structure would be comparable to the geometry and spacing of connective tissue fibre in a human heart.

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Drug testing on heart-on-a-chip gets a step closer

Durham, NC (PRWEB) March 11, 2015

Scientists report in the current issue of STEM CELLS Translational Medicine that they have been able to clone a line of defective stem cells behind a rare, but devastating disease called Fanconi Anemia (FA). Their achievement opens the door to drug screening and the potential for a new, safe treatment for this often fatal disease.

FA is a hereditary blood disorder that leads to bone marrow failure (FA-BMF) and cancer. Patients who suffer from FA have a life expectancy of 33 years. Currently, a bone marrow transplant offers the only possibility for a cure. However, this treatment has many risks associated with it, especially for FA patients due to their extreme sensitivity to radiation and chemotherapy.

Although various consequences in hematopoietic stem cells (the cells that give rise to all the other blood cells) have been attributed to FA-BMF, its cause is still unknown, said Megumu K. Saito, M.D., Ph.D., of Kyoto Universitys Center for iPS Cell and Application, and a lead investigator on the study. His laboratory specializes in studying the kinds of pediatric diseases in which a thorough analysis using mouse models or cultured cell lines is not feasible, so they apply disease-specific induced pluripotent stem cells (iPSCs) instead.

To address the FA issue, he explained, our team (including colleagues from Tokai University School of Medicine) established iPSCs from two FA patients who have the FANCA gene mutation that is typical in FA. We were then able to obtain fetal type immature blood cells from these iPSCs.

When observing the iPSCs, the researchers found that the characteristics of immature blood cells from FA-iPSCs were different from control cells. The FA-iPSCs showed an increased DNA double-strand break rate, as well as a sharp reduction of hematopoietic stem cells compared to the control group of non-FA iPSCs.

These data indicate that the hematopoietic consequences in FA patients originate from the earliest hematopoietic stage and highlight the potential usefulness of iPSC technology for explaining how FA-BMF occurs, said Dr. Saito. Since conducting a comprehensive analysis of patient-derived affected stem cells is not feasible without iPSC technology, the technology provides an unprecedented opportunity to gain further insight into this disease.

This work shows promise for identifying the initial pathological event that causes the disease, which would be a first step in working toward a cure, said Anthony Atala, M.D., Editor-in-Chief of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine.

###

The full article, Pluripotent cell models of Fanconi anemia identify the early pathological defect in human hemoangiogenic progenitors, can be accessed at http://www.stemcellstm.com.

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Stem Cell Clones Could Yield New Drug Treatment for Deadly Blood Disease

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Newswise LA JOLLA--For infants with severe combined immunodeficiency (SCID), something as simple as a common cold or ear infection can be fatal. Born with an incomplete immune system, kids who have SCIDalso known as bubble boy or bubble baby diseasecant fight off even the mildest of germs. They often have to live in sterile, isolated environments to avoid infections and, even then, most patients dont live past a year or two. This happens because stem cells in SCID patients bone marrow have a genetic mutation that prevents them from developing critical immune cells, called T and Natural Killer (NK) cells.

Now, Salk researchers have found a way to, for the first time, convert cells from x-linked SCID patients to a stem cell-like state, fix the genetic mutation and prompt the corrected cells to successfully generate NK cells in the laboratory.

The success of the new technique suggests the possibility of implanting these tweaked cells back into a patient so they can generate an immune system. Though the new work, published March 12, 2015 in Cell Stem Cell, is preliminary, it could offer a potentially less invasive and more effective approach than current options.

This work demonstrates a new method that could lead to a more effective and less invasive treatment for this devastating disease, says senior author Inder Verma, Salk professor and American Cancer Society Professor of Molecular Biology. It also has the potential to lay the foundation to cure other deadly and rare blood disorders.

Previous attempts to treat SCID involved bone marrow transplants or gene therapy, with mixed results. In what began as promising clinical trials in the 1990s, researchers hijacked virus machinery to go in and deliver the needed genes to newly growing cells in the patients bone marrow. While this gene therapy did cure the disease at first, the artificial addition of genes ended up causing leukemia in a few of the patients. Since then, other gene therapy methods have been developed, but these are generally suited for less mild forms of the disease and require bone marrow transplants, a difficult procedure to perform on critically sick infants.

To achieve the new method, the Salk team secured a sample of bone marrow from a deceased patient in Australia. Using that small sample, the team developed the new method in three steps. First, they reverted the patient cells into induced pluripotent stem cells (iPSCs)cells that, like embryonic stem cells, have the ability to turn into any type of tissue and hold vast promise for regenerative medicine.

Once we had patient-derived stem cells, we could remove the genetic mutation, essentially fixing the cells, explains one of the first authors and Salk postdoctoral researcher Amy Firth.

The second innovation was to use new gene editing technology to correct the SCID-related genetic deficiency in these iPSCs. To remove the mutation, the researchers used a technology called TALEN (similar to the better known CRISPR method). This set of enzymes act as molecular scissors on genes, letting researchers snip away at a gene and replace the base pairs that make up DNA with other base pairs.

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Immune System-in-a-Dish Offers Hope for "Bubble Boy Disease"

The author has posted comments on this articlePTI | Feb 23, 2015, 12.47AM IST

Ten different donor sources have been used so far and new germ-cell lines have been created from all of them, researchers said.

Page 1 of 4

Scientists at Cambridge University collaborated with Israel's Weizmann Institute of Science and used stem cell lines from embryos as well as from the skin of five different adults. Researchers have previously created live baby mice using engineered eggs and sperm, but until now have struggled to create a human version of these 'primordial germ' or stem cells.

Ten different donor sources have been used so far and new germ-cell lines have been created from all of them, researchers said.

"We have succeeded in the first and most important step of this process, which is to show we can make these very early human stem cells in a dish," said Azim Surani, professor of physiology and reproduction at Cambridge, who heads the project.

"We have also discovered that one of the things that happens in these germ cells is that epigenetic mutations, the cell mistakes that occur with age, are wiped out," said Surani, who was involved in research that led to the birth of Louise Brown, the world's first test-tube baby, in 1978.

Jacob Hanna, the specialist leading the project's Israeli arm, said it may be possible to use the technique to create a baby in just two years.

"It has already caused interest from gay groups because of the possibility of making egg and sperm cells from parents of the same sex," he said. The details of the technique were published in the journal Cell.

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Now, same-sex couples can make babies

March 12, 2015 // R. Colin Johnson

Living beating hearts on-a-chip were recently created from pluripotent stem cells discovered by 2010 Kyoto Prize Winner, Shinya Yamanaka.

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Bioengineers at the University of Berkeley aim to create all of the human organs on-a-chip then connect them with micro-fluidic channels to create a complete human-being on-a-wafer.

"We have learned how to derive almost any type of human tissue from skin stem cells as was first discovered by Yamanaka," professor Kevin Healy told EE Times. "Our initial application is drug screening without having to use animals, but putting organs-on-a-chip using the stem cells of the patient could help with genetic diseases as well."

"For instance, one drug might solve a heart problem, but create toxins in the liver," Healy told us. "Which would be much better to find out before administering to the patient."

As to creating living robots in this way, Healy said that was not their mission on the current project, since their funding in coming from the National Institutes of Health's (NIH's) Tissue Chip for Drug Screening Initiative, an interagency collaboration specifically aimed at developing 3-D human tissue chips for drug screening.

However, the technology being creating, especially the microfluidic channels connecting the organs-on-a-chip so that they interact, could someday serve as a basis for making robot-like creatures.

"What we would need for that is sensors and actuators. Sensors would be the easiest, but MIT in particular is working on artificial muscles to serve as actuators," Healy told us.

Living beating hearts on-a-chip were recently created from pluripotent stem cells discovered by 2010 Kyoto Prize Winner, Shinya Yamanaka.

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Heart on-a-chip beats

1 Control of Pluripotency Laboratory, Department of Physiological Sciences I, Faculty of Medicine, University of Barcelona, Hospital Clinic, Casanova 143, 08036, Barcelona, Spain 2 Faculty of Medicine, University of Sydney Medical School, Division of Pediatrics and Child Health, Westmead Children's Hospital, Locked Bag 4001, Westmead NSW 2145, Sydney, Australia 3 School of Anatomy Physiology & Human Biology and The Harry Perkins Institute for Medical Research (CCTRM), the University of Western Australia, 6 Verdun St, Nedlands WA 6009, Perth, Australia

* Author to whom correspondence should be addressed.

Received: 1 October 2014 / Accepted: 3 December 2014 / Published: 29 January 2015

Abstract: The use of adult myogenic stem cells as a cell therapy for skeletal muscle regeneration has been attempted for decades, with only moderate success. Myogenic progenitors (MP) made from induced pluripotent stem cells (iPSCs) are promising candidates for stem cell therapy to regenerate skeletal muscle since they allow allogenic transplantation, can be produced in large quantities, and, as compared to adult myoblasts, present more embryonic-like features and more proliferative capacity in vitro, which indicates a potential for more self-renewal and regenerative capacity in vivo. Different approaches have been described to make myogenic progenitors either by gene overexpression or by directed differentiation through culture conditions, and several myopathies have already been modeled using iPSC-MP. However, even though results in animal models have shown improvement from previous work with isolated adult myoblasts, major challenges regarding host response have to be addressed and clinically relevant transplantation protocols are lacking. Despite these challenges we are closer than we think to bringing iPSC-MP towards clinical use for treating human muscle disease and sporting injuries.

Roca, I.; Requena, J.; Edel, M.J.; Alvarez-Palomo, A.B. Myogenic Precursors from iPS Cells for Skeletal Muscle Cell Replacement Therapy. J. Clin. Med. 2015, 4, 243-259.

Roca I, Requena J, Edel MJ, Alvarez-Palomo AB. Myogenic Precursors from iPS Cells for Skeletal Muscle Cell Replacement Therapy. Journal of Clinical Medicine. 2015; 4(2):243-259.

Roca, Isart; Requena, Jordi; Edel, Michael J.; Alvarez-Palomo, Ana B. 2015. "Myogenic Precursors from iPS Cells for Skeletal Muscle Cell Replacement Therapy." J. Clin. Med. 4, no. 2: 243-259.

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GERMANTOWN, Md. (TheStreet) -- Neuralstem (CUR - Get Report) is providing an overly optimistic picture about its surgical stem-cell therapy for amyotrophic lateral sclerosis (ALS), the degenerative and fatal nerve disease.

Instead of disclosing the results from all 15 ALS patients enrolled in Neuralstem's phase II study of NSI-566, the company decided to only release a comparison between the patients who responded and those who didn't respond. Of course, the seven responders in the study showed more stabilization or improvements in muscle function compared with the eight patients deemed non-responders.

The scientific term for this conclusion is, "Duh."

When you work backwards and do some simple math on the muscle performance of all 15 ALS patients in the Neuralstem study, the results aren't very encouraging. Neuralstem chose to stay mum on this more customary analysis.

Neuralstem shares are down 14% to $3.21 in Thursday trading.

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Neuralstem Stock Plunges After Latest Study on ALS Drug

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Newswise Researchers at the University of California, San Diego School of Medicine have identified a gene variant that may be used to predict people most likely to respond to an investigational therapy under development for Alzheimers disease (AD). The study, published March 12 in Cell Stem Cell, is based on experiments with cultured neurons derived from adult stem cells.

Our results suggest that certain gene variants allow us to reduce the amount of beta amyloid produced by neurons, said senior author Lawrence Goldstein, PhD, director of UC San Diego Sanford Stem Cell Clinical Center and UC San Diego Stem Cell Program. This is potentially significant for slowing the progression of Alzheimers disease. AD is the most common cause of dementia in the United States, afflicting one in nine people age 65 and older.

The genetic risk factor investigated are variants of the SORL1 gene. The gene codes for a protein that affects the processing and subsequent accumulation of beta amyloid peptides, small bits of sticky protein that build up in the spaces between neurons. These plaques are linked to neuronal death and related dementia.

Previous studies have shown that certain variants of the SORL1 gene confer some protection from AD, while other variants are associated with about a 30 percent higher likelihood of developing the disease. Approximately one-third of the U.S. adult population is believed to carry the non-protective gene variants.

The studys primary finding is that variants in the SORL1 gene may also be associated with how neurons respond to a natural compound in the brain that normally acts to protect nerve cell health. The protective compound, called BDNF, short for brain-derived neurotrophic factor, is currently being investigated as a potential therapy for a number of neurological diseases, including AD, because of its role in promoting neuronal survival.

For the study, UC San Diego researchers took skin cells from 13 people, seven of whom had AD and six of whom were healthy control subjects, and reprogrammed the skin cells into stem cells. These stem cells were coaxed to differentiate into neurons, and the neurons were cultured and then treated with BDNF.

The experiments revealed that neurons that carried disease-protective SORL1 variants responded to the therapy by reducing their baseline rate of beta amyloid peptide production by, on average, 20 percent. In contrast, the neurons carrying the risk variants of the gene, showed no change in baseline beta amyloid production.

BDNF is found in everyones brain, said first author Jessica Young, PhD, a postdoctoral fellow in the Goldstein laboratory. What we found is that if you add more BDNF to neurons that carry a genetic risk factor for the disease, the neurons dont respond. Those with the protective genetic profile do.

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Boosting A Natural Protection Against Alzheimer's Disease

IMAGE:Brain tumor stem cells (orange) in mice express a stem cell marker (green). Researchers at Washington University School of Medicine in St. Louis are studying how cancer stem cells make... view more

Credit: Yi-Hsien Chen

Scientists are eager to make use of stem cells' extraordinary power to transform into nearly any kind of cell, but that ability also is cause for concern in cancer treatment. Malignant tumors contain stem cells, prompting worries among medical experts that the cells' transformative powers help cancers escape treatment.

New research proves that the threat posed by cancer stem cells is more prevalent than previously thought. Until now, stem cells had been identified only in aggressive, fast-growing tumors. But a mouse study at Washington University School of Medicine in St. Louis shows that slow-growing tumors also have treatment-resistant stem cells.

The low-grade brain cancer stem cells identified by the scientists also were less sensitive to anticancer drugs. By comparing healthy stem cells with stem cells from these brain tumors, the researchers discovered the reasons behind treatment resistance, pointing to new therapeutic strategies.

"At the very least, we're going to have to use different drugs and different, likely higher dosages to make sure we kill these tumor stem cells," said senior author David H. Gutmann, MD, PhD, the Donald O. Schnuck Family Professor of Neurology.

The research appears online March 12 in Cell Reports.

First author Yi-Hsien Chen, PhD, a senior postdoctoral research associate in Gutmann's laboratory, used a mouse model of neurofibromatosis type 1 (NF1) low-grade brain tumors to identify cancer stem cells and demonstrate that they could form tumors when transplanted into normal, cancer-free mice.

NF1 is a genetic disorder that affects about 1 in every 2,500 babies. The condition can cause an array of problems, including brain tumors, impaired vision, learning disabilities, behavioral problems, heart defects and bone deformities.

The most common brain tumor in children with NF1 is the optic glioma. Treatment for NF1-related optic gliomas often includes drugs that inhibit a cell growth pathway originally identified by Gutmann. In laboratory tests conducted as part of the new research, it took 10 times the dosage of these drugs to kill the low-grade cancer stem cells.

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Stem cells lurking in tumors can resist treatment

A new study by University of Alberta law researchers reveals sometimes overly optimistic news coverage of clinical translation of stem cell therapies--and as spokespeople, scientists need to be mindful of harnessing public expectations.

"As the dominant voice in respect to timelines for stem cell therapies, the scientists quoted in these stories need to be more aware of the importance of communicating realistic timelines to the press," said researcher Kalina Kamenova, who co-authored the study with professor Timothy Caulfield in the University of Alberta's Health Law Institute, based in the Faculty of Law.

Their analysis of media coverage showed that most news reports were highly optimistic about the future of stem cell therapies and forecasted unrealistic timelines for clinical use. The study, published in the latest issue of Science Translational Medicine, examined 307 news reports covering translational stem cell research in major daily newspapers in Canada, the United States and the United Kingdom between 2010 and 2013.

While the field of stem cell research holds tremendous promise, "it has also been surrounded by tremendous hype, and we wanted to quantify that in some degree," Caulfield said. "Pop culture representations have an impact on how the public perceives the readiness of stem cell research, and that in turn feeds into stem cell tourism, marketing of unproven therapies and even the public's trust in research. We wanted to provide findings that would help inform the issue."

Their study found that 69 per cent of all news stories citing timelines predicted that therapies would be available within five to 10 years or even sooner. At the same time, the press overlooked challenges and failures in therapy translation, such as the discontinuation of the first FDA-approved clinical trial of an embryonic stem cell-derived therapy for spinal cord injuries in 2011. The biotech company conducting the trial was a leader in embryonic stem cell therapies and its decision to stop its work on stem cells was considered a significant setback for the field.

As well, ethical concerns about the use of human embryonic stem cells were displaced from the forefront of news coverage, while the clinical translation of stem cell therapies and new discoveries, such as hockey star Gordie Howe's recent treatment, grabbed the headlines instead.

"Our findings showed that many scientists have often provided either by implication or direct quotes, authoritative statements regarding unrealistic timelines for stem cell therapies and media hype can foster unrealistic public expectations about clinical translation and increased patient demand for unproven stem cell therapies," Caulfield noted.

While stem cell therapy research is progressing and has seen a dramatic increase in the past decade of clinical trials for treatments, the vast majority of these studies are still in the safety-testing stage and involve a limited number of participants, Kamenova noted.

"The approval process for new treatments is long and complicated, and only a few of all drugs that enter pre-clinical testing are approved for human clinical trials. It takes on average 12 years to get a new drug from the lab to the market, and additional 11 to 14 years of post-market surveillance," she added.

The science world is under pressure to come up with cures for what ails us, but "care needs to be taken by the media and the research community so that advances in research and therapy are portrayed in a realistic manner," Caulfield said.

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Media portray unrealistic timelines for stem cell therapies

Stem Cell Therapy Network
Stem Cell Therapy Network connects patients and providers through a global Stem Cell Therapy Network using our Patient Advocate System, Medical Tourism and Personal Injury Network. We have...

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Cardiac Stem Cells: Making a Difference in Duchenne
Dr Eduardo Marban, Director of the Cedars-Sinai Heart Institute, discusses a possible Cardiac Stem Cell breakthrough for Duchenne muscular dystrophy. Coalition Duchenne founder, Catherine ...

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No Mortality or Toxicity Noted With CryoStor Deployed as Vehicle Solution for Cells and Placebo in Direct Intramyocardial Injections

BioLife Solutions, Inc., a leading developer, manufacturer and marketer of proprietary clinical grade hypothermic storage and cryopreservation freeze media and precision thermal shipping products for cells and tissues (BioLife or the Company), recently announced its CryoStor cell freeze media was utilized in a porcine animal study of umbilical cord blood-derived mononuclear cells (UBC-MNC) to evaluate the safety and feasibility of these cells for cardiac regeneration in pediatric congenital heart disease (CHD).

The safety and feasibility study was performed at the Mayo Clinic in Rochester, Minnesota, with the results recently published in an article titled Safety and Feasibility for Pediatric Cardiac Regeneration Using Epicardial Delivery of Autologous Umbilical Cord Blood-Derived Mononuclear Cells Established in a Porcine Model System, which appeared in the peer reviewed clinical journal Stem Cells Translational Medicine.

Umbilical cord blood-derived mononuclear cells were frozen in protein-free, serum-free CryoStor CS10, containing 10% dimethyl sulfoxide (DMSO). Thawed cells were administered to piglets via intramyocardial injections, with follow up extended to three months. CryoStor CS10 was also used as the placebo control solution without cells, which was injected into randomized piglets.

The authors observed no mortality or toxicity in any study animals and concluded:

Mike Rice, BioLife President & CEO, stated, The data from this study further support the use of our proprietary biopreservation media products in clinical applications. We are quite pleased to have an institution as renowned as the Mayo Clinic evaluate and use our products in their important clinical research.

BioLife management estimates that the Companys CryoStor cryopreservation freeze media andHypoThermosol storage and shipping media have been incorporated into the manufacturing and clinical delivery protocols of more than 175 cell and tissue-based regenerative medicine clinical trials for new products and therapies.

About BioLife Solutions BioLife Solutions develops, manufactures and markets hypothermic storage and cryopreservation solutions and precision thermal shipping products for cells, tissues, and organs. BioLife also performs contract aseptic media formulation, fill, and finish services. The Companys proprietary HypoThermosol and CryoStor platform of solutions are highly valued in the biobanking, drug discovery, and regenerative medicine markets. BioLifes biopreservation media products are serum-free and protein-free, fully defined, and are formulated to reduce preservation-induced cell damage and death. BioLifes enabling technology provides commercial companies and clinical researchers significant improvement in shelf life and post-preservation viability and function of cells, tissues, and organs. For more information, visit http://www.biolifesolutions.com.

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Lindsey Caldwell

Heart disease is the leading cause of death among Americans. Recently the bio-tech industry has been exploding with cardiac research like last week's heart attack preventing nanobots. New research by the team at the University of California, Berkley has created working human heart cells on a tiny chip designed to test the efficacy of new drugs in clinical trials. This heart-on-a-chip is officially known as a cardiac microphysiological system, or MPS. Using this heart-on-a-chip, scientists can measure the potential cardiac damage of a drug before it reaches expensive human trials.

Drug trials can take years, and to mitigate risk these drugs undergo testing in non-human subjects. Animals are often used in place of humans, but animal models can be problematic. Specifically, they are less effective at predicting cardiotoxicity, wherein a drug damages the heart. This is important because one-third of drugs withdrawn from testing are pulled due to cardiotoxic effects.

Drugs that are first tested in animal models can succeed to future testing stages without setting off alarms. After successful early stages more time and money is invested and the drugs progress to human trials, only to be stopped in their tracks because they are found to be toxic to human hearts.

The cells on this tiny MPS chip are human heart cells that were created from pluripotent stem cells. These cells react to drugs the same way as a human heart inside a living person. By creating a portable, low-risk, and accurate drug testing environment, scientists may be able to advance clinical trials of new drugs and bring them to market sooner.

Here is a video by the UC Berkley research team of their heart cells actually beating.

Source: Berkeley

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Heart-on-a-chip tests drugs cardiotoxicity with its real heartbeat

March 10, 2015

These are human blood cells grown in the lab from genetically edited stem cells. (Credit: Ying Wang/Johns Hopkins Medicine)

Provided by Shawna Williams, Johns Hopkins Medicine

Researchers at Johns Hopkins have successfully corrected a genetic error in stem cells from patients with sickle cell disease, and then used those cells to grow mature red blood cells, they report. The study represents an important step toward more effectively treating certain patients with sickle cell disease who need frequent blood transfusions and currently have few options.

The results appear in an upcoming issue of the journalStem Cells.

In sickle cell disease, a genetic variant causes patients blood cells to take on a crescent, or sickle, shape, rather than the typical round shape. The crescent-shaped cells are sticky and can block blood flow through vessels, often causing great pain and fatigue. Getting a transplant of blood-making bone marrow can potentially cure the disease. But for patients who either cannot tolerate the transplant procedure, or whose transplants fail, the best option may be to receive regular blood transfusions from healthy donors with matched blood types.

[STORY: New injection helps stem traumatic blood loss]

The problem, says Linzhao Cheng, Ph.D. , the Edythe Harris Lucas and Clara Lucas Lynn Professor of Hematology and a member of the Institute for Cell Engineering, is that over time, patients bodies often begin to mount an immune response against the foreign blood. Their bodies quickly kill off the blood cells, so they have to get transfusions more and more frequently, he says.

A solution, Cheng and his colleagues thought, could be to grow blood cells in the lab that were matched to each patients own genetic material and thus could evade the immune system. His research group had already devised a way to use stem cells to make human blood cells. The problem for patients with sickle cell disease is that lab-grown stem cells with their genetic material would have the sickle cell defect.

To solve that problem, the researchers started with patients blood cells and reprogrammed them into so-called induced pluripotent stem cells, which can make any other cell in the body and grow indefinitely in the laboratory. They then used a relatively new genetic editing technique called CRISPR to snip out the sickle cell gene variant and replace it with the healthy version of the gene. The final step was to coax the stem cells to grow into mature blood cells. The edited stem cells generated blood cells just as efficiently as stem cells that hadnt been subjected to CRISPR, the researchers found.

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Building custom blood cells to battle sickle cell disease

Coronary heart disease is the countrys leading cause of death A new treatment was designed to treat damaged heart muscle The capsule contains stem cells derived from the patients bone marrow

By Roger Dobson for the Daily Mail

Published: 17:33 EST, 9 March 2015 | Updated: 18:07 EST, 9 March 2015

A new treatment using a tiny grow-bag has been designed to treat damaged heart muscle

A tiny grow-bag could be a new way to mend hearts damaged by disease or heart attack.

The capsule, which is pea-sized, contains stem cells that trigger the growth of new cells.

An estimated 2.3 million people in Britain have coronary heart disease the countrys leading cause of death.

It occurs when the arteries supplying the heart become blocked by fatty substances, reducing the flow of blood.

If a bit of this fatty substance breaks off, it can trigger a blood clot, which in turn cuts off the blood supply to heart muscle, causing it to die off. This is what triggers a heart attack.

Heart disease and heart attacks can also lead to heart failure, where the heart becomes too weak to pump blood around the body properly.

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The tiny grow-bag that could mend a heart damaged by disease

Researchers have created a "heart on a chip" using actual cardiac muscles to help test the effects of heart medication.

Anurag Mathur/Healy Lab

A new device could help make drug testing safer, faster, cheaper -- and eliminate the need for animal testing. It's just an inch long, but inside its silicone body is housed a small piece of cardiac muscle that responds to cardiovascular medications in exactly the same way heart muscle does inside a living human body.

"Ultimately, these chips could replace the use of animals to screen drugs for safety and efficacy," explained Kevin Healy, UC Berkeley professor of engineering, who led the research team that designed the device.

The problems with using animals to test human heart medication aren't merely ethical -- such concerns about lab animals rarely enter scientific discussions. Rather, there are some serious physiological problems -- namely, that drugs designed for humans will not have the same effect on a species that is biologically different from a human.

"These differences often result in inefficient and costly experiments that do not provide accurate answers about the toxicity of a drug in humans," Healy explained.

"It takes about $5 billion on average to develop a drug, and 60 percent of that figure comes from upfront costs in the research and development phase. Using a well-designed model of a human organ could significantly cut the cost and time of bringing a new drug to market."

The chips were created using heart muscle grown in a lab from adult human induced pluripotent stem cells -- stem cells that can be coaxed to grow into many other types of cell. The team then carefully designed the structure to be similar to the geometry and spacing of connective tissue fibre in a living human heart.

Microfluidic channels carved into the silicone on either side of the cell matrix act the same way as blood vessels, mimicking the exchange of nutrients and drugs with human tissue as it would happen in the body.

The cells start beating on their own within 24 hours of being loaded into the chamber at a healthy resting rate of 55 to 80 beats per minute. In order to test the system, the team then administered four well-known cardiovascular drugs -- isoproterenol, E-4031, verapamil and metoprolol. By monitoring the beat rate, the team was able to observe -- and accurately predict -- the chip's response to the drugs. Isoproterenol, for example -- a drug used to treat slow heart rate -- caused the muscle's beat rate to increase from 55 beats per minute to 124 beats per minute half an hour after being administered.

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Human heart on a chip could replace animal drug testing

Scientists at Tuft University in Massachusetts grew bone marrow on silk They were able to generate functioning platelet cells that form blood clots The cells could be used to stop bleeding in injured patients in ER rooms It has raised hopes that man-made blood can be created for transfusions However some say it could be up to 15 years before stem cells can be used to create blood that can be safely used for transfusions during surgery

By Richard Gray for MailOnline

Published: 11:46 EST, 19 February 2015 | Updated: 12:50 EST, 23 February 2015

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A major component of blood has been grown in the laboratory by scientists, bringing man-made blood transfusions a step closer.

Biomedical engineers have for the first time produced functional blood platelets - the cells that cause clots to form - from human bone marrow grown in the laboratory.

The achievement raises hopes that it will soon be possible to produce fully functional blood in a similar way.

Scientists have managed to grow fully functioning platelets like the one above surrounded by red blood cells

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Could we soon have man-made blood?

Orphan Designation Provides 7-Year Post Approval Marketing Exclusivity, Tax Credits and Elimination of FDA Prescription Drug User Fees

SAN DIEGO, CA--(Marketwired - February 10, 2015) - Targazyme Inc., a clinical-stage biopharmaceutical company developing enzyme technologies and products to improve efficacy outcomes for stem cell transplantation, immunotherapy, gene therapy and regenerative medicine, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to TZ101 to prevent and reduce the severity and incidence of graft vs. host disease (GVHD) in patients eligible for hematologic stem cell transplant.

GVHD is a serious, life-threating complication of stem cell transplantation.Orphan drug status confirms the importance of Targazyme's novel treatment approach to prevent and reduce the incidence and severity of GVHD in patients with blood cancers where stem cell transplant is prescribed.TZ101 could potentially transform hematopoietic stem cell transplantation by reducing patient morbidity and mortality from GVHD, which occurs in a large percentage of these patients and is very difficult to manage clinically.

"Our work with TZ101 demonstrates impressive increases in the persistence and activity of regulatory T cells in preclinical models of GVHD," said Dr. Elizabeth J. Shpall, Deputy Chair of the Department of Stem Cell Transplantation and Cellular Therapy at The University of Texas MD Anderson Cancer Center."We are looking forward to beginning clinical trials on this promising modality for preventing GVHD in our patients undergoing stem cell transplantation."

Orphan Drug Designation by FDA confers financial benefits and incentives, such as potential Orphan Drug grant funding to defray the cost of clinical testing, tax credits for the cost of clinical research, a 7 year period of exclusive marketing after Approval and a Waiver of Prescription Drug User Fee Act (PDUFA) filing fees which are now greater than $2 million.

"The granting of Orphan Drug status for TZ101 for prevention of GVHD in stem cell transplant patients, as well as our previous Orphan Drug designation of TZ101 for cord blood transplantation, provides additional validation of our innovative platform technologies," said Lynnet Koh, Chairman & Chief Executive Officer of Targazyme."TZ101 and our second product, TZ102 are enabling technologies for improving efficacy outcomes for multiple cell-based therapeutic approaches used to prevent and treat a variety of different diseases for which there is a high unmet medical need.In addition to initiating our registration trial with TZ101 in hematopoietic stem cell transplantation, we plan to embark on our cancer immunotherapy trial later this year."

About Targazyme, Inc.

Targazyme Inc. is a San Diego-based, clinical-stage biopharmaceutical company developing novel enzyme-based platform technologies and products to improve clinical efficacy outcomes for stem cell medicine, auto-immunotherapy, gene therapy and regenerative medicine.

The company's clinical-grade fucosyltransferase enzymes and small molecule products (TZ101 and TZ102) are off-the-shelf products used at the point-of-care to treat therapeutic cells immediately before infusion into the patient using a simple procedure that is easily incorporated into existing medical practice.The company has received a number of world-wide patents, multiple FDA orphan drug designations and major medical/scientific awards and grants.

Targazyme has partnerships and collaborations with Kyowa Hakko Kirin and Florida Biologix, as well as various medical research institutions including The University of Texas MD Anderson Cancer Center, Oklahoma Medical Research Foundation, Texas Transplant Institute, Case Western/University Hospitals, Scripps Hospitals, Fred Hutchinson Cancer Research Center, UCLA Medical Center, Stanford University Medical Center, University of Minnesota Medical Center, University of California San Diego, Sanford-Burnham Medical Research Institute, Indiana University, Memorial Sloan Kettering Cancer Center, and New York Blood Center.For more information please go to http://www.targazyme.com.

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Targazyme Inc. Receives Orphan Drug Designation to TZ101 for Use With Regulatory T Cells to Prevent & Reduce the ...