April 11, 2017 07:15 ET | Source: Cesca Therapeutics Inc.

RANCHO CORDOVA, Calif., April 11, 2017 (GLOBE NEWSWIRE) -- Cesca Therapeutics Inc. (Nasdaq:KOOL), a market leader in automated cell processing and point-of-care, autologous cell-based therapies, today announced that Dr. Xiaochun (Chris) Xu, Chairman and Interim Chief Executive Officer and Chairman of Boyalife Group, will present an overview of the Companys cardiovascular clinical research program at the 2017 International Symposium of Translational Medicine in Stem Cell Myocardial Repair, being held April 10-12, 2017 at the Hope Hotel in Shanghai, China.

Details of the presentation are as follows:

Despite recent therapeutic and surgical advances, the effects of peripheral arterial disease, including heart attack and critical limb ischemia (CLI), remain among the worlds leading causes of morbidity and mortality and represent a rapidly escalating public health crisis, noted Dr. Xu. I look forward to presenting a review of our latest findings, including key feasibility study results and an overview of our Phase 3 Critical Limb Ischemia Rapid Stemcell Treatment (CLIRST) trial, which we believe highlight the potential of Cesca Therapeutics proprietary AutoXpress point-of-care platform to deliver autologous cell-based therapies that may represent a new paradigm in patient treatment going forward.

About the Symposium of Translational Medicine in Stem Cell Myocardial Repair

The 2017 International Symposium of Translational Medicine in Stem Cell Myocardial Repair brings together more than 650 of the worlds cardiac disease thought leaders to discuss the potential of translational and regenerative medicine in treating myocardial infarction (MI) and cardiac failure. The symposium is co-sponsored by the Shanghai Society for Cell Biology, the Institute of Health Sciences, the Shanghai Cardiovascular Disease Institute, the Guangzhou Institutes of Biomedicine and Health, and the Key Laboratory of Stem Cell Biology, Shanghai.

About Cesca Therapeutics Inc.

Cesca is engaged in the research, development, and commercialization of cellular therapies and delivery systems for use in regenerative medicine. The Company is a leader in the development and manufacture of automated blood and bone marrow processing systems that enable the separation, processing and preservation of cell and tissue therapeutics. Cesca is an affiliate of the Boyalife Group (http://www.boyalifegroup.com), a China-based industrial-research alliance among top research institutes for stem cell and regenerative medicine.

Forward-Looking Statement

The statements contained herein may include statements of future expectations and other forward-looking statements that are based on managements current views and assumptions and involve known and unknown risks and uncertainties that could cause actual results, performance or events to differ materially from those expressed or implied in such statements. A more complete description of risks that could cause actual events to differ from the outcomes predicted by Cesca Therapeutics' forward-looking statements is set forth under the caption "Risk Factors" in Cesca Therapeutics annual report on Form 10-K and other reports it files with the Securities and Exchange Commission from time to time, and you should consider each of those factors when evaluating the forward-looking statements.

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CESCA Therapeutics to Present at the 2017 International Symposium of Translational Medicine in Stem Cell ... - GlobeNewswire (press release)

Cardiac Stem Cells Comments Off on CESCA Therapeutics to Present at the 2017 International Symposium of Translational Medicine in Stem Cell … – GlobeNewswire (press release) | April 16th, 2017

Joseph Baucum , jbaucum@pnj.com Published 4:06 p.m. CT April 16, 2017 | Updated 26 minutes ago

Andrews Institute is conducting new stem cell research that could impact the FDA approval of certain treatments. Joseph Baucumjbaucum@pnj.com

Dr. Andrew Anz, an orthopedic surgeon and sports medicine specialist at the Andrews Institute in Gulf Breeze is working on cutting-edge stem cell research for cartilage therapy.(Photo: Tony Giberson/tgiberson@pnj.com)Buy Photo

By the time most reach age 55, Adam Anz estimatesas much as 30 percent of the population will incur some form of knee degeneration, a problem that equals pain and in many cases, surgery.

Its a problem that were all going to face at some point in our lives, said Anz, orthopaedic surgeon at Andrews Institute for Orthopaedics & Sports Medicine.

But in May, a new study will begin at Andrews Institute in Gulf Breeze that could play a game-changing role in evolving the range of medicine available for treating knee injuries. In the process, the research may also help drive down patients costs.

Anz will help spearhead a study next month into increasing the amount of stem cells doctors are able to harvest from bone marrow transplants with the goal of utilizing those cells to regrow cartilage in knees. Cartilage, a tough and flexible material, is essential to the knee, because it acts as a cushion between the bones in the joint. Damaged cartilage can often necessitate knee replacement.

ADDITIONAL CONTENT:Andrews Institute expands prep athletics care in region

In the study, Anz said researchers will attempt to increase the amount of stem cells in participants bone marrow, which would then empty from the marrow into their bloodstream. Researchers would collect the blood, separate the stem cells from it and inject the cells into patients knees. Doctors would then monitor if the marrow cells transform into cartilage cells and spark regeneration.

Its about determining how can we obtain those cells in efficient quantities and put those cells in the right place at the right time to help with healing patients injuries, Anz said.

Because the Food & Drug Administration has not approved the vast majority of stem cell-based remedies, not all treatments involving the cells are available for patients, including the cartilage procedure. For the treatments that are offered, health insurance providers do not cover them without the FDAs consent. Patients who choose to undergo them must pay out-of-pocket prices.

The study at Andrews Institute could push a stem cell cartilage treatment closer to FDA approval and by extension, availability and affordability. The research is an official FDA study. It is led by Khay Yong Saw, a Malaysian physicianwho has already demonstrated conceptual proof of the treatment in an animal study in 2006. He completed a randomized control trial in 2012. This study is the next step in proving the safety and efficacy of the procedure to gain federal endorsement.

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ADDITIONAL CONTENT:Elite athletes just piece of Andrews Institute

Anz, optimistic about the studys potential, emphasized more research must be conducted into the effectiveness of stem cell treatments those already available and those still in the testing phase.

Its important to be excited about technologies, but its also important to be honest with the patients that more must be done to show these treatments are effective, said Anz, who estimated the cartilage study to require two years for participant enrollment and another two years before researchers can observe outcomes.

But some who have undergone stem cell treatments advocate for the procedures federal approval. Jody Falvey, a retired Pensacola resident, had a stem cell procedure conducted at Andrews Institute on her knee in the fall of 2012.

Falvey, 67, tore the medial and lateral meniscus in her knee during a family visit to South Florida while brewing coffee in the morning. The sensation, she said, felt like a knife slicing through her joint.

Following a consultation with Anz, who described an available stem cell treatment known as bone marrow aspirate concentrate, Falvey chose to have the procedure done. The treatment utilized cells from her own body to repair the knee. The process, from procedure to recovery, spanned about two years.

Falvey said her knee does not feel like it ever underwent surgery. The fact that it helped prevent her from having to undergo a knee replacement made the operation even better.

I did not want metal in my body, she said. This was just one of the greatest alternatives I had heard of. I would do it again in a heartbeat.

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Andrews Institute to study stem cells' impact on knee - Pensacola News Journal

Bone Marrow Stem Cells Comments Off on Andrews Institute to study stem cells’ impact on knee – Pensacola News Journal | April 16th, 2017

Stem cells have been touted as treatments for everything from hair loss to heart disease.

But are those claims scientifically sound?

Research on the technology continues to look promising, but many of its human applications are still preliminary and their effectiveness anecdotal.

Samumed, a $12 billion biotech start-up based in San Diego, profiled this month in Business Insider, exemplifies both sides of the coin.

The company has promised a bevy of age-reversing cures, including regrowing hair, treating wrinkles, and regenerating cartilage in people with osteoarthritis

However, their research isnt conclusive.

None of their treatments have received government approval yet.

Read more: Rheumatoid arthritis and stem cell treatments

Its easy to get excited about all this research.

Samumed Is Trying to Create the Fountain of Youth, says one headline.

Samumed Aims to Reverse Aging with Eternal Youth Treatments, says another.

Combined with $300 million in investment funding, the company has more than just buzz going for it in the biotech industry.

Their treatment for androgenetic alopecia (hair loss) is currently in phase II trials.

Its program to help people with osteoarthritis regrow cartilage in their knees is in phase III.

In total, the company has seven drugs in phase II trials, with plans to expand into more areas of disease research this year.

However, Samumed has raised some eyebrows in the industry with its secrecy. Some skeptics have likened the company to Theranos, a biotech start-up that was valued at $9 billion before an investigation by the Wall Street Journal led to a shutdown of the companys labs.

Samumed has been more open about presenting their data to the public but not about the actual treatments.

We're basically telling everyone, here's proof that it works, Samumed Chief Executive Officer, Osman Kibar, told Business Insider. How it works you just need to wait a little longer because we want to build as much of a head start as we can.

Read more: Stem cell treatments for multiple sclerosis

Beyond the applications of stem cells at Samumed, the technology is also being used to treat some of the United States most widespread health issues.

New research from the American Heart Association this month demonstrated the effectiveness of implanted stem cells into the hearts of people with cardiomyopathy.

Although the sample size was small (only 27 people), scientists noted function and symptomatic improvements of heart functioning as well as less frequency of hospitalization and lower medical costs. They conclude that the stem cell procedure is a feasible treatment for cardiomyopathy, but they note that a larger clinical follow-up is needed for more conclusive results.

In the past week, Newsweek reported on miracle stem cell treatments for burn victims that will promote healing without scars.

Stat News wrote about research on stem cells in mice that could potentially help cure Parkinsons disease.

Read more: Unproved stem cell treatments offer hope and risk

Some researchers in the industry are somewhat measured in their optimism of the technologys human applications.

I want to make sure that we provide a real cautionary note, especially to those individuals and those institutions that tout stem cells as the panacea for any ill, Dr. Cato Laurencin, director of the Institute for Regenerative Engineering at the University of Connecticut, told Healthline.

Laurencin, a medical practitioner at the forefront of stem cell technology, is a firm believer in the benefits of the treatment, but also remains skeptical of some of the claims associated with it.

Much of the evidence is still preliminary or anecdotal, and when people operate on information that is preliminary or anecdotal, there is the possibility for harm, he said.

His work in regenerative engineering a term he coined several years ago looks at the healing properties of implanted stem cells in the human body.

In research published this month, Laurencin and his team concluded that stem cells effectively improved healing to torn rotator cuff tendons in rats.

Rotator cuff tendon tears are a relatively common injury in humans and can be difficult to treat.

Unlike other tendons in the body, the rotator cuff tendon is unable to heal itself, said Laurencin.

Once it is torn, it is liable to be reinjured again and again.

However, the research released this month is about more than just applying stem cells to a certain kind of injury, its about how the stem cells are applied.

Read more: Scientists use 3-D environment to speed up growth of stem cells

Laurencin describes his field as an evolution of earlier work from 30 years ago in tissue engineering: a convergence of bringing together new technologies to create new science and new possibilities.

In this case, nanotechnology is at the heart of this stem cell operation.

Currently there are a variety of ways that stem cells can be implanted into a subject, including injections and bone marrow transplants.

For his research, Laurencin and his team used biomaterial based fiber matrices a nanomaterial conducive to growing and attaching stem cells to implant into the wounded area.

The results are promising, but Laurencin and his team will have to continue working with animals for some time before the process can be applied to humans.

The key is in understanding that stem cells have the potential for more than just regrowing damaged parts of the body.

The way we commonly think about a stem cell is it becoming a new tissue. But were also understanding that the stem cell itself can secrete biological factors that help regeneration occur. Thats what we think is happening here, said Laurencin.

His research into stem cells as a medicinal element in the body could have far reaching implications for all kinds of wound therapy.

Despite his measured approach, Laurencin is still willing to hypothesize about the excitement that the future of the field undoubtedly holds with proper time, funding, and research.

There are newts and salamanders that can regenerate a limb, he told Healthline.

How do we harness the cues that are taking place in these types of animals, and can we utilize what weve learned from these types of animals in humans?

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Stem Cell Research Advancing Rapidly - Healthline

Bone Marrow Stem Cells Comments Off on Stem Cell Research Advancing Rapidly – Healthline | April 16th, 2017

Stem cells are a rapidly advancing field of biological research. Since Dr. James Thomson first cultivated human embryonic stem cells at the University of Wisconsin - Madison in the late 1990s, this field of researched has exploded with potential. The links below provide access to a curriculum developed under the supervision of Dr. Thomson as well as the co-directors and staff of the UW Stem Cell & Regenerative Medicine Center. The material has been reviewed for accuracy by the scientists actually conducting the research and was compiled and formatted by Craig Kohn, a high school teacher with research experience, for a high school audience. The PowerPoint presentation works in conjunction with the notesheet, allowing for students to work independently if preferred. More information about specific instructional practices can be found below in Teacher Notes. PowerPoint: http://bit.ly/ted-stemcells Notesheet: http://bit.ly/ted-stemcellsnotesheet Quiz: http://bit.ly/ted-stemcellsquiz Additional resources about stem cells can be found at: http://www.stemcells.wisc.edu/node/386 http://stemcells.nih.gov/Pages/Default.aspxhttp://www.stemcellschool.org/http://www.nursingdegree.net/blog/750/25-best-blogs-for-following-stem-cell-research/

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What are stem cells? - Craig A. Kohn | TED-Ed

Skin Stem Cells Comments Off on What are stem cells? – Craig A. Kohn | TED-Ed | April 16th, 2017

Salk Institute and Chinese researchers report creating a new kind of stem cell, one that is more versatile than any other normally grown in the lab.

Called an extended pluripotent stem cell, it can give rise to every cell type in the body, the researchers say in a recent study. This includes the extra-embryonic tissues such as the placenta that support the developing baby. Just one cell can generate a complete organism.

Embryonic stem cells and artificial embryonic stem cells called induced pluripotent stem cells cant make these extra-embryonic tissues. So neither embryonic nor IPS cells can give rise to a complete embryo, because the supportive tissues necessary for an embryo to survive arent there.

But the extended pluripotent stem cells can reliably give rise to both types of cells, and thus whole embryos and offspring, the scientists report.

The EPS cells were made from human and mouse embryonic stem cells. In addition, they were produced from skin cells, or fibroblasts by treating them with a chemical cocktail. IPS cells, invented in 2006, are generated from fibroblasts by a similar reprogramming process.

Use of IPS cells is regarded as morally acceptable by those who oppose use of human embryonic stem cells, because they cant form an entire embryo. This is the reasoning of the Catholic Church. But since the EPS cells can make whole embryos, at least in mice, how the church will react is unclear.

To demonstrate this ability to make all cell types, the researchers grew an entire mouse from just one EPS cell. They also grew chimeric mice, with human EPS cells integrating into the mice better than embryonic stem cells did.

The study on these new stem cells was published April 6 in the journal Cell. It can be found at j.mp/extendedstem.

Better tool

That characteristic of creating every cell in the body, called totipotency, is normally found only at the very beginning of embryonic development. Embryonic stem cells are usually extracted too late, when the cells have already divided into the embryonic and extra-embryonic lineages.

Totipotent stem cells have been observed in the lab, but they lasted briefly, and didnt yield stable totipotent cell lines.

Salk Institute stem cell researcher Juan Carlos Izpisa Bemonte was a cosenior author of the paper along with Hongkui Deng of Peking University in Beijing. The first authors were Yang Yang, Bei Liu, Jun Xu, and Jinlin Wang; all of Peking University, and Jun Wu, of the Salk Institute.

EPS cell lines provide a useful cellular tool for gaining a better molecular understanding of initial cell fate commitments and generating new animal models to investigate basic questions concerning development of the placenta, yolk sac, and embryo proper, the study stated.

Furthermore, they also provide an unlimited cell resource and hold great potential for in vivo disease modeling, in vivo drug discovery, and in vivo tissue generation in the future. Finally, our study opens a path toward capturing stem cells with intra- and/or inter-species bi-potent chimeric competency from a variety of other mammalian species.

Organs for transplant

The creation of chimeric mice is part of Izpisa Bemontes longstanding goal of growing human organs in animals for transplant.

While mice are too small to grow organs for transplant, they serve as a model to understand how cells from a different species, can be grown in a host body. In this new study, the mice served as a model of how well the EPS cells can integrate.

Izpisa Bemonte is now working to translate his research on chimeric mice to pigs, which are large enough to provide human organs. In January, a team he led reported on work with human-pig chimeras, which were not allowed to grow past the embryonic stage. They also created rat-mice chimeras.

The superior chimeric competency of both human and mouse EPS cells is advantageous in applications such as the generation of transgenic animal models and the production of replacement organs, Wu said in a Salk statement. We are now testing to see whether human EPS cells are more efficient in chimeric contribution to pigs, whose organ size and physiology are closer to humans.

We believe that the derivation of a stable stem cell line with totipotent-like features will have a broad and resounding impact on the stem cell field, Izpisua Belmonte said in the statement.

The work was funded by a number of sources. They include: the National Key Research and Development Program of China; the National Natural Science Foundation of China; the Guangdong Innovative and Entrepreneurial Research Team Program; the Science and Technology Planning Project of Guangdong Province, China; the Science and Technology Program of Guangzhou, China; the Ministry of Education of China (111 Project); the BeiHao Stem Cell and Q9 Regenerative Medicine Translational Research Institute; the Joint Institute of Peking University Health Science Center; University of Michigan Health System; Peking-Tsinghua Center for Life Sciences; the National Science and Technology Support Project; the CAS Key Technology Talent Program; the G. Harold and Leila Y. Mathers Charitable Foundation; and The Moxie Foundation.

bradley.fikes@sduniontribune.com

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Stem cell invented that can grow into any tissue in the body - The ... - The San Diego Union-Tribune

Skin Stem Cells Comments Off on Stem cell invented that can grow into any tissue in the body – The … – The San Diego Union-Tribune | April 16th, 2017

TERT Identifiers Aliases TERT, CMM9, DKCA2, DKCB4, EST2, PFBMFT1, TCS1, TP2, TRT, hEST2, hTRT, telomerase reverse transcriptase External IDs OMIM: 187270 MGI: 1202709 HomoloGene: 31141 GeneCards: TERT Genetically Related Diseases breast cancer, interstitial lung disease, adenocarcinoma of the lung, prostate cancer, se atraganto con un caramelo, testicular germ cell cancer, idiopathic pulmonary fibrosis, malignant glioma[1] RNA expression pattern More reference expression data Orthologs Species Human Mouse Entrez Ensembl UniProt RefSeq (mRNA) RefSeq (protein) Location (UCSC) Chr 5: 1.25 1.3 Mb Chr 13: 73.63 73.65 Mb PubMed search [2] [3] Wikidata View/Edit Human View/Edit Mouse

Telomerase reverse transcriptase (abbreviated to TERT, or hTERT in humans) is a catalytic subunit of the enzyme telomerase, which, together with the telomerase RNA component (TERC), comprises the most important unit of the telomerase complex.[4][5]

Telomerases are part of a distinct subgroup of RNA-dependent polymerases. Telomerase lengthens telomeres in DNA strands, thereby allowing senescent cells that would otherwise become postmitotic and undergo apoptosis to exceed the Hayflick limit and become potentially immortal, as is often the case with cancerous cells. To be specific, TERT is responsible for catalyzing the addition of nucleotides in a TTAGGG sequence to the ends of a chromosomes telomeres.[6] This addition of repetitive DNA sequences prevents degradation of the chromosomal ends following multiple rounds of replication.[7]

hTERT absence (usually as a result of a chromosomal mutation) is associated with the disorder Cri du chat.[8][9]

Telomerase is a ribonucleoprotein polymerase that maintains telomere ends by addition of the telomere repeat TTAGGG. The enzyme consists of a protein component with reverse transcriptase activity, encoded by this gene, and an RNA component that serves as a template for the telomere repeat. Telomerase expression plays a role in cellular senescence, as it is normally repressed in postnatal somatic cells, resulting in progressive shortening of telomeres. Studies in mice suggest that telomerase also participates in chromosomal repair, since de novo synthesis of telomere repeats may occur at double-stranded breaks. Alternatively spliced variants encoding different isoforms of telomerase reverse transcriptase have been identified; the full-length sequence of some variants has not been determined. Alternative splicing at this locus is thought to be one mechanism of regulation of telomerase activity.[10]

The hTERT gene, located on chromosome 5, consists of 16 exons and 15 introns spanning 35 kb. The core promoter of hTERT includes 330 base pairs upstream of the translation start site (AUG since it's RNA by using the words "exons" and "introns"), as well as 37 base pairs of exon 2 of the hTERT gene.[11][12][13] The hTERT promoter is GC-rich and lacks TATA and CAAT boxes but contains many sites for several transcription factors giving indication of a high level of regulation by multiple factors in many cellular contexts.[11] Transcription factors that can activate hTERT include many oncogenes (cancer-causing genes) such as c-Myc, Sp1, HIF-1, AP2, and many more, while many cancer suppressing genes such as p53, WT1, and Menin produce factors that suppress hTERT activity .[13][14] Another form of up-regulation is through demethylation of histones proximal to the promoter region, imitating the low density of trimethylated histones seen in embryonic stem cells.[15] This allows for the recruitment of histone acetyltransferase (HAT) to unwind the sequence allowing for transcription of the gene.[14]

Telomere deficiency is often linked to aging, cancers and the conditions dyskeratosis congenita (DKC) and Cri du chat. Meanwhile, over-expression of hTERT is often associated with cancers and tumor formation.[8][16][17][18] The regulation of hTERT is extremely important to the maintenance of stem and cancer cells and can be used in multiple ways in the field of regenerative medicine.

hTERT is often up-regulated in cells that divide rapidly, including both embryonic stem cells and adult stem cells.[17] It elongates the telomeres of stem cells, which, as a consequence, increases the lifespan of the stem cells by allowing for indefinite division without shortening of telomeres. Therefore, it is responsible for the self-renewal properties of stem cells. Telomerase are found specifically to target shorter telomere over longer telomere, due to various regulatory mechanisms inside the cells that reduce the affinity of telomerase to longer telomeres. This preferential affinity maintains a balance within the cell such that the telomeres are of sufficient length for their function and yet, at the same time, not contribute to aberrant telomere elongation [19]

High expression of hTERT is also often used as a landmark for pluripotency and multipotency state of embryonic and adult stem cells. Over-expression of hTERT was found to immortalize certain cell types as well as impart different interesting properties to different stem cells.[13][20]

hTERT immortalizes various normal cells in culture, thereby endowing the self-renewal properties of stem cells to non-stem cell cultures.[13][21] There are multiple ways in which immortalization of non-stem cells can be achieved, one of which being via the introduction of hTERT into the cells. Differentiated cells often express hTERC and TP1, a telomerase-associated protein that helps form the telomerase assembly, but does not express hTERT. Hence, hTERT acts as the limiting factor for telomerase activity in differentiated cells [13][22] However, with hTERT over-expression, active telomerase can be formed in differentiated cells. This method has been used to immortalize prostate epithelial and stromal-derived cells, which are typically difficult to culture in vitro. hTERT introduction allows in vitro culture of these cells and available for possible future research. hTERT introduction have an advantage over the use of viral protein for immortalization in that it does not involve the inactivation of tumor suppressor gene, which might lead to cancer formation.[21]

Over-expression of hTERT in stem cells changes the properties of the cells.[20][23][24] hTERT over-expression increases the stem cell properties of human mesenchymal stem cells. The expression profile of mesenchymal stem cells converges towards embryonic stem cells, suggesting that these cells may have embryonic stem cell-like properties. However, it has been observed that mesenchymal stem cells undergo decreased levels of spontaneous differentiation.[20] This suggests that the differentiation capacity of adult stem cells may be dependent on telomerase activities. Therefore, over-expression of hTERT, which is akin to increasing telomerase activities, may create adult stem cells with a larger capacity for differentiation and hence, a larger capacity for treatment.

Increasing the telomerase activities in stem cells gives different effects depending on the intrinsic nature of the different types of stem cells.[17] Hence, not all stem cells will have increased stem-cell properties. For example, research has shown that telomerase can be upregulated in CD34+ Umbilical Cord Blood Cells through hTERT over-expression. The survival of these stem cells was enhanced, although there was no increase in the amount of population doubling.[24]

Deregulation of telomerase expression in somatic cells may be involved in oncogenesis.[10]

Genome-wide association studies suggest TERT is a susceptibility gene for development of many cancers,[25] including lung cancer.[26]

Telomerase activity is associated with the number of times a cell can divide playing an important role in the immortality of cell lines, such as cancer cells. The enzyme complex acts through the addition of telomeric repeats to the ends of chromosomal DNA. This generates immortal cancer cells.[27] In fact, there is a strong correlation between telomerase activity and malignant tumors or cancerous cell lines.[28] Not all types of human cancer have increased telomerase activity. 90% of cancers are characterized by increased telomerase activity.[28]Lung cancer is the most well characterized type of cancer associated with telomerase.[29] There is a lack of substantial telomerase activity in some cell types such as primary human fibroblasts, which become senescent after about 3050 population doublings.[28] There is also evidence that telomerase activity is increased in tissues, such as germ cell lines, that are self-renewing. Normal somatic cells, on the other hand, do not have detectable telomerase activity.[30] Since the catalytic component of telomerase is its reverse transcriptase, hTERT, and the RNA component hTERC, hTERT is an important gene to investigate in terms of cancer and tumorigenesis.

The hTERT gene has been examined for mutations and their association with the risk of contracting cancer. Over two hundred combinations of hTERT polymorphisms and cancer development have been found.[29] There were several different types of cancer involved, and the strength of the correlation between the polymorphism and developing cancer varied from weak to strong.[29] The regulation of hTERT has also been researched to determine possible mechanisms of telomerase activation in cancer cells. Glycogen synthase kinase 3 (GSK3) seems to be over-expressed in most cancer cells.[27] GSK3 is involved in promoter activation through controlling a network of transcription factors.[27]Leptin is also involved in increasing mRNA expression of hTERT via signal transducer and activation of transcription 3 (STAT3), proposing a mechanism for increased cancer incidence in obese individuals.[27] There are several other regulatory mechanisms that are altered or aberrant in cancer cells, including the Ras signaling pathway and other transcriptional regulators.[27]Phosphorylation is also a key process of post-transcriptional modification that regulates mRNA expression and cellular localization.[27] Clearly, there are many regulatory mechanisms of activation and repression of hTERT and telomerase activity in the cell, providing methods of immortalization in cancer cells.

If increased telomerase activity is associated with malignancy, then possible cancer treatments could involve inhibiting its catalytic component, hTERT, to reduce the enzymes activity and cause cell death. Since normal somatic cells do not express TERT, telomerase inhibition in cancer cells can cause senescence and apoptosis without affecting normal human cells.[27] It has been found that dominant-negative mutants of hTERT could reduce telomerase activity within the cell.[28] This led to apoptosis and cell death in cells with short telomere lengths, a promising result for cancer treatment.[28] Although cells with long telomeres did not experience apoptosis, they developed mortal characteristics and underwent telomere shortening.[28] Telomerase activity has also been found to be inhibited by phytochemicals such as isoprenoids, genistein, curcumin, etc.[27] These chemicals play a role in inhibiting the mTOR pathway via down-regulation of phosphorylation.[27] The mTOR pathway is very important in regulating protein synthesis and it interacts with telomerase to increase its expression.[27] Several other chemicals have been found to inhibit telomerase activity and are currently being tested as potential clinical treatment options such as nucleoside analogues, retinoic acid derivatives, quinolone antibiotics, and catechin derivatives.[30] There are also other molecular genetic-based methods of inhibiting telomerase, such as antisense therapy and RNA interference.[30]

hTERT peptide fragments have been shown to induce a cytotoxic T-cell reaction against telomerase-positive tumor cells in vitro.[31] The response is mediated by dendritic cells, which can display hTERT-associated antigens on MHC class I and II receptors following adenoviral transduction of an hTERT plasmid into dendritic cells, which mediate T-cell responses.[32] Dendritic cells are then able to present telomerase-associated antigens even with undetectable amounts of telomerase activity, as long as the hTERT plasmid is present.[33]Immunotherapy against telomerase-positive tumor cells is a promising field in cancer research that has been shown to be effective in in vitro and mouse model studies.[34]

Induced pluripotent stem cells (iPS cells) are somatic cells that have been reprogrammed into a stem cell-like state by the introduction of four factors (Oct3/4, Sox2, Klf4, and c-Myc).[35] iPS cells have the ability to self-renew indefinitely and contribute to all three germ layers when implanted into a blastocyst or use in teratoma formation.[35]

Early development of iPS cell lines were not efficient, as they yielded up to 5% of somatic cells successfully reprogrammed into a stem cell-like state.[36] By using immortalized somatic cells (differentiated cells with hTERT upregulated), iPS cell reprogramming was increased by twentyfold compared to reprogramming using mortal cells.[36]

The reactivation of hTERT, and subsequently telomerase, in human iPS cells has been used as an indication of pluripotency and reprogramming to an ES (embryonic stem) cell-like state when using mortal cells.[35] Reprogrammed cells that do not express sufficient hTERT levels enter a quiescent state following a number of replications depending on the length of the telomeres while maintaining stem cell-like abilities to differentiate.[36] Reactivation of TERT activity can be achieved using only three of the four reprogramming factors described by Takahashi and Yamanaka: To be specific, Oct3/4, Sox2 and Klf4 are essential, whereas c-Myc is not.[15] However, this study was done with cells containing endogenous levels of c-Myc that may have been sufficient for reprogramming.

Telomere length in healthy adult cells elongates and acquires epigenetic characteristics similar to those of ES cells when reprogrammed as iPS cells. Some epigenetic characteristics of ES cells include a low density of tri-methylated histones H3K9 and H4K20 at telomeres, as well as an increased detectable amount of TERT transcripts and protein activity.[15] Without the restoration of TERT and associated telomerase proteins, the efficiency of iPS cells would be drastically reduced. iPS cells would also lose the ability to self-renew and would eventually senesce.[15]

DKC (dyskeratosis congenita) patients are all characterized by the defective maintenance of telomeres leading to problems with stem cell regeneration.[16] iPS cells derived from DKC patients with a heterozygous mutation on the TERT gene display a 50% reduction in telomerase activity compared to wild type iPS cells.[37] Conversely, mutations on the TERC gene (RNA portion of telomerase complex) can be overcome by up-regulation due to reprogramming as long as the hTERT gene is intact and functional.[38] Lastly, iPS cells generated with DKC cells with a mutated dyskerin (DKC1) gene cannot assemble the hTERT/RNA complex and thus do not have functional telomerase.[37]

The functionality and efficiency of a reprogrammed iPS cell is determined by the ability of the cell to re-activate the telomerase complex and elongate its telomeres allowing for self-renewal. hTERT is a major limiting component of the telomerase complex and a deficiency of intact hTERT impedes the activity of telomerase, making iPS cells an unsuitable pathway towards therapy for telomere-deficient disorders.[37]

Although the mechanism is not fully understood, exposure of TERT-deficient hematopoietic cells to androgens resulted in an increased level of TERT activity.[39] Cells with a heterozygous TERT mutation, like those in DKC (dyskeratosis congenita) patients, which normally exhibit low baseline levels of TERT, could be restored to normal levels comparable to control cells. TERT mRNA levels are also increased with exposure to androgens.[39] Androgen therapy may become a suitable method for treating circulatory ailments such as bone marrow degeneration and low blood count linked with DKC and other telomerase-deficient conditions.[39]

As organisms age and cells proliferate, telomeres shorten with each round of replication. Cells restricted to a specific lineage are capable of division only a set number of times, set by the length of telomeres, before they senesce.[40] Depletion and uncapping of telomeres has been linked to organ degeneration, failure, and fibrosis due to progenitors' becoming quiescent and unable to differentiate.[19][40] Using an in vivo TERT deficient mouse model, reactivation of the TERT gene in quiescent populations in multiple organs reactivated telomerase and restored the cells abilities to differentiate.[41] Reactivation of TERT down-regulates DNA damage signals associated with cellular mitotic checkpoints allowing for proliferation and elimination of a degenerative phenotype.[41] In another study, introducing the TERT gene into healthy one-year-old mice using an engineered adeno-associated virus led to a 24% increase in lifespan, without any increase in cancer.[42]

The hTERT gene has become a main focus for gene therapy involving cancer due to its expression in tumor cells but not somatic adult cells.[43] One method is to prevent the translation of hTERT mRNA through the introduction of siRNA, which are complimentary sequences that bind to the mRNA preventing processing of the gene post transcription.[44] This method does not completely eliminate telomerase activity, but it does lower telomerase activity and levels of hTERT mRNA seen in the cytoplasm.[44] Higher success rates were seen in vitro when combining the use of antisense hTERT sequences with the introduction of a tumor-suppressing plasmid by adenovirus infection such as PTEN.[45]

Another method that has been studied is manipulating the hTERT promoter to induce apoptosis in tumor cells. Plasmid DNA sequences can be manufactured using the hTERT promoter followed by genes encoding for specific proteins. The protein can be a toxin, an apoptotic factor, or a viral protein. Toxins such as diphtheria toxin interfere with cellular processes and eventually induce apoptosis.[43] Apoptotic death factors like FADD (Fas-Associated protein with Death Domain) can be used to force cells expressing hTERT to undergo apoptosis.[46] Viral proteins like viral thymidine kinase can be used for specific targeting of a drug.[47] By introducing a prodrug only activated by the viral enzyme, specific targeting of cells expressing hTERT can be achieved.[47] By using the hTERT promoter, only cells expressing hTERT will be affected and allows for specific targeting of tumor cells.[43][46][47]

Aside from cancer therapies, the hTERT gene has been used to promote the growth of hair follicles.[48]

A schematic animation for gene therapy is shown as follows.

Telomerase reverse transcriptase has been shown to interact with:

Continued here:
Telomerase reverse transcriptase - Wikipedia

IPS Cell Therapy Comments Off on Telomerase reverse transcriptase – Wikipedia | April 16th, 2017

WASHINGTON D.C: Scientists have developed a revolutionary 3D-bioprinted patch that could one day be used to repair damage to the human heart.

The patch can help heal scarred heart tissue after a heart attack. The discovery is a major step forward in treating patients with tissue damage after a heart attack.

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

When the cell patch was placed on a mouse following a simulated heart attack, the researchers saw significant increase in functional capacity after just four weeks. Since the patch was made from cells and structural proteins native to the heart, it became part of the heart and absorbed into the body, requiring no further surgeries.

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

Ogle said that this research is different from previous research in that the patch is modelled after a digital, three-dimensional scan of the structural proteins of native heart tissue. The digital model is made into a physical structure by 3D printing with proteins native to the heart and further integrating cardiac cell types derived from stem cells. Only with 3D printing of this type can we achieve one micron resolution needed to mimic structures of native heart tissue.

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

Ogle said they are already beginning the next step to develop a larger patch that they would test on a pig heart, which is similar in size to a human heart.

The research study is published in Circulation Research, a journal published by the American Heart Association.

See the article here:
Breakthrough in cardiac health: 3D-printed patch can help mend a 'broken' heart - Economic Times

Cardiac Stem Cells Comments Off on Breakthrough in cardiac health: 3D-printed patch can help mend a ‘broken’ heart – Economic Times | April 15th, 2017

Having worked at University Malaya Medical Centres (UMMC) Paediatrics Department for 20 years, senior consultant paediatric oncologist Prof Dr Hany Mohd Ariffin has had to tell her fair share of parents that there is nothing more that can be done for their terminally ill child.

As head of the Paediatric Haematology-Oncology and Bone Marrow Transplantation Unit, this is usually because there is no suitable donor available for a life-saving bone marrow transplant for the child.

Bone marrow transplants, also called stem cell transplants, are used in conditions where the patients bone marrow is damaged or destroyed by disease or intensive cancer treatment, and is unable to carry out its job of producing healthy red blood cells, white blood cells and platelets.

Because white blood cells or leukocytes are part of the immune system that protects our body against foreign invaders, it is critical in such a procedure to match the so-called immunological fingerprints of the patient and the donor.

As Prof Hany explains, these fingerprints are known as human leukocyte antigens (HLAs).

HLAs help the immune system distinguish between the bodys own cells and foreign cells, usually bacteria and viruses that infect us, so that our white blood cells can find and destroy them.

It is crucial that a bone marrow donor and the patient have the same HLAs in order to minimise the chances of the donated bone marrows white blood cells considering its new host body as foreign and attacking it.

Perfect match needed

Standard bone marrow transplantations require that all 10 HLAs in both patient and donor are a match.

As HLAs are inherited half from each parent, this means that only a patients siblings are a possible perfect match.

Explains Prof Hany: If you look at statistics, out of four, one sibling will be completely matched, one sibling will be completely not matched, and two siblings would be half-matched.

So, the chances of finding a match is 25%, but that is statistical randomisation.

In the real world, you can have 10 siblings and all of them might not be matched with you.

If a patient does not have a sibling that matches perfectly with them, or does not have a sibling at all, their only other option is to check for an unrelated match in international stem cell registries or blood banks.

However, Prof Hany notes that this usually requires a sum of RM100,000 for a unit of bone marrow and at least three to four months of waiting two luxuries not all patients have.

She adds: But it is not easy to get a good match for Asians as these registries are usually Caucasian.

And its even worse if you are an Indian patient, as you cant even go to a Taiwanese blood bank.

In the case of Muhammad Yusuff Iskandar Mohd Hambali, time was a critical factor.

The firstborn of two teachers had been referred to UMMC at 10 months of age for recurrent pneumonia.

His mother, secondary school physical education teacher, Aduratun Nasyihin Mokhtar shares: He started falling sick at the age of seven months he had a persistent cough.

Initially, the doctor thought it was pertussis, but it didnt get better after three months as pertussis should, so he was admitted to the hospital.

However, none of the antibiotics they tried worked, so he was referred to UMMC to check his lungs.

This filepic shows a thalassaemia patient with his infusion pump machine for iron-chelating therapy. Thalassaemia is one of the conditions curable by a bone marrow transplant.

It was in UMMC that Yusuff, as he is called, was discovered to have X-linked severe combined immunodeficiency (SCID).

This rare genetic condition, also known as bubble boy disease, results in the malfunction or lack of two specialised white blood cells called T and B cell lymphocytes.

This means that Yusuff effectively had a non-existent immune system.

This was the reason he could not fight off the pneumonia. In fact, his lungs had deteriorated so badly that he was on oxygen therapy from the age of eight months.

In addition, the Mycobacterium bovis in his BCG vaccination had spread to his back, he had chronic diarrhoea and he was very much underweight.

Yusuff needed a bone marrow transplant, and he needed it fast.

Having reached out to her international colleagues at that time, Prof Hany says: One thing constant in all their advice was that if we delayed the procedure, he would never get better from his disseminated BCG, his pneumonia would just worsen, and once you reach a critical point, there would be no turning back.

He would have been dead by six months.

The problem was that Yusuff was then an only child.

Although his mother was pregnant with his younger sister at that time, she would not have been born in time to help him, assuming that she was a match for him in the first place.

With no time to waste, Prof Hany and her team decided to try a procedure called haplo-identical bone marrow transplantation.

On whether she and her team were ready to carry out the new procedure, Prof Hany says that you will never be ready until a life is dangling precariously in front of you. Photo: The Star/Samuel Ong

In this procedure, only five out of 10 HLAs need to be matched in order for the donor to be able to give bone marrow to the patient.

The beauty of this procedure is that you always have two parents (to donate), says Prof Hany.

So, Yusuffs father, sports science and physical education teacher Mohd Hambali Din @ Ismail, could now donate his bone marrow cells to his son.

First though, Yusuff needed to be fattened up via nutritional fluids infused into his veins, his pneumonia brought under control and his M. bovis infection treated with anti-tuberculosis therapy.

This was so that he would be in a decent enough condition to withstand the procedure.

Following the protocol established by Johns Hopkins University in the United States, but modified to suit Yusuffs condition, Prof Hany and her team first killed off Yusuffs remaining bone marrow cells through chemothera-py, before infusing 30ml of his fathers donated bone marrow into him.

Prof Hany explains that it takes two to three weeks for the new bone marrow cells to grow, during which time the patient is completely vulnerable to any infection.

This is why they remain in a completely sealed room where the air is hepa-filtered, they receive no visitors, and their food and linen are completely sterile, she says.

He was also treated with high-dose cyclophosphamide, a chemotherapy drug that targets T cell lymphocytes.

This was in order to destroy the half-matched mature T cells that came with his fathers donated bone marrow.

T cells are your soldier cells. His fathers T cells would recognise Yusuff as foreign and destroy everything in their wake.

And that is what has precluded mismatched transplants all this while, explains Prof Hany.

After the mature T cells are destroyed, she says: What you then get are T cells from stem cell origin, which learn to tolerate the environment of being in Yusuffs body, and therefore, they will be less aggressive and more friendly to these cells that they consider foreign.

Despite that, Yusuff still experienced graft-versus-host disease (GvHD) where his new white blood cells attacked the cells of his skin, gut and lungs.

In between, he also had two episodes of sepsis and he had to go to the ICU once.

He also had to go on the ventilator at one point, says Prof Hany.

She explains that GvHD, which is due to aggressive donor white blood cells, and infections, which are due to the still incomplete immune system, can co-exist, creating a dilemma for the medical team.

On the one hand, to ameliorate GvHD, you have to give steroids (in addition to standard immunosuppresants) to dampen down the immune system.

You dampen down the immune system, then you allow bacteria and fungi to grow.

And that is why it is very challenging, she says.

She admits: For the first 20 days, it was all very smooth and you think, Wah, Im a hero, but then the challenges came.

There were certain moments when I thought, Thats it, were going to lose him.

It took 149 days after the transplant before Yusuff was deemed well enough to be sent home.

And it was one year before Prof Hany and her team felt confident enough to declare him cured.

We estimate anything between six months to a year for the new bone marrow cells to grow and propagate.

So usually, after a year, if the GvHD doesnt appear anymore, it is very unlikely to suddenly appear, she explains.

This first anniversary of Yusuffs transplant, celebrated at UMMC on April 6, was not just sweet because of Yusuffs survival, it was also the opening of a new path for Prof Hany and her team.

On a personal note, there were many times when you have this period of self-doubt.

So, you think that we are just a bunch of stupid, gung-ho people, who are unrealistic; this is not America, this cannot be done that sort of feeling.

There were some moments when you think, have I done a disservice to this child? Would if it have been better to just let go, for the parents to just let go? Is God just testing me? shares Prof Hany.

However, a few months after Yusuffs transplant, she received the case of a baby boy with myelodysplastic syndrome.

Myelodysplastic children will progress to develop acute myeloid leukaemia within a year, and it is only curable with transplant, or not it is certain death by two years, she explains.

And this patient had two siblings, both of whom were only half-matched.

But we were able to offer a transplant to this child, because we knew that from the experience of Yusuff, if he has very bad GvHD of the gut, skin, lung, we would be able to handle it been there, done that.

We were already scarred for life, she says with a laugh. And in fact, due to their prior experience, Prof Hany and her team were able to more precisely determine the amount of donated bone marrow cells needed for transplant.

As a result, she says: The second patient sailed through and was discharged after only five weeks, as opposed to five months for Yusuff.

Explaining the potential impact of having this treatment option available, Prof Hany shares that bone marrow transplantation is a cure for conditions like leukaemia, blood disorders like thalassaemia, congenital defective immune systems and certain rare congenital metabolic conditions.

The major reason why transplants are not being done is because of the lack of an available donor, she says.

But haplo-identical bone marrow transplantation now opens the way for many more potential donors to help the patient.

The learning curve is steep, Prof Hany admits, but adds that after Yusuff, they were able to apply what they learnt to their second patient with great effect.

Im not saying it is easy, but I think it is worth developing further, because it can solve one of the greatest health problems in our country, which is inherited blood disorders.

Giving the example of thalassaemia, she estimates that it costs some RM3.5mil to treat a patient with regular blood transfusions and iron-chelating therapy for 30 years.

A haplo-identical bone marrow transplant costs approximately RM45,000 and will cure the patient.

The risk of dying from this procedure usually because of infections and GvHD during the period when the patient has no working immune system is estimated to be about 10%.

This is at the upper limit for standard bone marrow transplants, where the risk ranges from 5% to 10%.

She adds that studies have shown that the risk of severe GvHD is similar for haplo-identical transplants and sibling-matched transplants, which are both lower than transplants from an unrelated donor.

Although Yusuff is the first successful haplo-identical bone marrow transplant patient in the country, to the best of Prof Hanys knowledge, she believes that the procedure can be easily done in other major hospitals around the country.

The facilities are already there and specialists trained in bone marrow transplants need only learn the procedure once before they should be able to conduct it, she says.

So its not just having a big celebration to tell the world that we saved one boy with SCID, its having the ability to tell parents that there is always hope, as we can now do haplo-identical transplants in our centre, says Prof Hany.

It is about no longer having to tell parents that nothing more can be done for their terminally ill child.

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New hope with haplo-identical bone marrow transplant - Star2.com

Bone Marrow Stem Cells Comments Off on New hope with haplo-identical bone marrow transplant – Star2.com | April 15th, 2017

Sumitomo Dainippon Pharma Co Ltd has ordered cell culture technologies from Hitachi as part of its effort to develop a treatment for Parkinsons disease.

The order financial terms of which were not provided will see Hitachi supply automated cell culturing technologies designed for the manufacture of induced pluripotent stem cells (iPS).

Dainippon is developing a cell therapy for Parkinsons-related dopamine neuron loss and neurodegeneration in collaboration with both Hitachi and Center for iPS Cell Research and Application, Kyoto University (CiRA).

Part of the project which is funded by the Japanese Agency of Medical Research and Development (AMED) - involves the development of processing methods and technologies for the production of stem cells for regenerative therapies.

The Japanese drug firm has announced several regenerative medicine-based research projects in recent years, beginning in 2015 when it partnered with Sanbio to develop SB623, an allogenic cell therapy for ischemic stroke to improve motor abilities.

Regenerative meds

Regenerative medicine which engineers or replaces damaged cells within human patients has become a popular area of research in Japan sinceShinya Yamanaka won the 2012 Noel Prize for medicine for the discovery that mature cells can be reprogrammed to become pluripotent.

Regenerative medicine is also a big focus for the Japanese Government.

Laws introduced in November 2014 therevised pharmaceutical affairs law and newregenerative medicines legislation mean such products could be reviewed and approved in just two years, if deemed to be effective.

Japans Government further underlined its commitment to regenerative medicine in its budget in January 2015, allocating Y2.5bn ($20.8bn) to the industrialisation of regenerative medicine evaluation fundamental technology development business.

Read more:
Sumitomo Dainippon buys cell therapy processing tech from Hitachi - In-PharmaTechnologist.com

IPS Cell Therapy Comments Off on Sumitomo Dainippon buys cell therapy processing tech from Hitachi – In-PharmaTechnologist.com | April 15th, 2017

A desperate mum-of-two says she is losing her memory so fast she may soon be unable to recognise her young daughters.

Chantelle Fox was diagnosed with multiple sclerosis (MS) last May after suffering fatigue and "a little numbness" in her arm.

Since the devastating diagnosis, the 41-year-old's condition has quickly deteriorated, leaving her fearful for the future.

She has 79 lesions on her brain and her short-term memory is fading, meaning she often forgets where she has put things.

She also forgets promising her "beautiful" daughters, Lilly, five, and Edie, three, that she will take them somewhere special.

Her worst fear is that in just four years, she may not even remember the little girls at all.

Now, she faces a race against time to fund radical stem cell treatment in Russia - which she believes could halt the progress of her disease.

MS is a chronic condition, for which there is currently no cure.

The disease is caused by the immune system malfunctioning and mistakenly attacking nerve cells in the brain and spinal cord.

It can lead to patients suffering from a range of mild or severe symptoms.

In a bid to stop the progress of the "monster" condition, Chantelle, from Australia, plans to undergo surgery in Moscow.

MS Australia has deemed the treatment risky, while one neurologist told the mum there is no evidence it works and it could be dangerous.

However, another neurologist reportedly told her she would be a great candidate for a haematopoietic stem cell transplant (HSCT) trial.

But Chantelle, who lives in Melbourne, said waiting a long time for the chance to take part in a clinical trial in her home country wasn't an option.

Instead, she plans to travel abroad in June to undergo surgery.

I have two beautiful kids and I might not remember them in four years time if I dont go to Russia," she told the Herald Sun .

She added: I have to fight for my kids. I want to help them study, to see them married, to be a grandparent."

Chantelle has been accepted into an autologous haematopoietic stem cell transplant programme in Moscow.

She claimed the treatment has an "86 per cent success rate" in halting the progress of the neurological condition.

However, her family said the costs involved are "crippling".

They are trying to raise $150,000 (120,000) to cover the price of the treatment and transport to and from Russia.

Chantelle's sister, Maxine Parker, has set up dedicated GoFundMe and Facebook pages to help raise money for the surgery.

On the GoFundMe page, she describes how her younger sibling was "devastated" when she was diagnosed with MS.

She writes: "Chantelle was first diagnosed in May 2016. What started out as fatigue and a little numbness in her left arm, she put it down to just being tired from being a full time working mum of two young girls...

"A trip to her doctor one Sunday afternoon changed her and her family life forever. She was told to head straight to hospital, the dr believes she may have had a minor stroke.

"24 hours later, sitting in the hospital bed the neurologist suggests its either a brain tumour, motor neurone disease or MS and the only way to confirm is with a lumbar puncture and full MRI of her brain & spine.

"I will never forget sitting there holding my baby sister's hand as she lays on the bed with the nurse injecting a large needle into her spine to obtain spinal fluid. Almost an hour and half goes by and they confirm its been unsuccessful and they will need to try again.

"Next is the MRI and after two hours my sister returns to her hospital bed waiting for the news that will change her life forever."

She adds: "That neurologist returns to deliver the news, Chantelle you have multiple sclerosis... Chantelle is devastated, all she can think about is her two young girls and if she will be around to watch them grow up."

In a post on the page, Chantelle herself pays tribute to her sister, her husband Dara O'Donoghue and her two little girls.

Addressing Lilly and Edie, she writes: "You are the reason, I will never give up fighting this terrible disease, MS.

"You are my world and I will love you for eternity."

She also expresses her gratitude to her other relatives and friends.

It is estimated that around 100,000 people in the UK have MS.

HSCT involves the intravenous infusion of stem cells derived from peripheral blood, bone marrow or umbilical cord blood.

In autologous cases, the patient's own stem cells are used.

Their immune system is usually wiped out with chemotherapy treatment before it is regrown using their stem cells.

To visit Chantelle's GoFundMe page, click here .

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Desperate mum's race against time to fund treatment before she forgets her two little girls - Mirror.co.uk

Spinal Cord Stem Cells Comments Off on Desperate mum’s race against time to fund treatment before she forgets her two little girls – Mirror.co.uk | April 14th, 2017

A quick glance at your calendar will reveal that we're now in 2017. 2017, you may recall, is the year when contraversial surgeon Sergio Canavero has promised to perform the world's first human head transplant.

But just how feasible is a human head transplant? Is it the stuff of science fiction, or does it have a basis in current sceintific thinking? Read on for everything you need to know about 2017 most alarming scientific development.

A human head transplant is exactly what it sounds like taking one living head and putting it onto a new body.

But actually, thats a little misleading. In real terms, its a body transplant, as the head will be gaining a new body to control. However, as the term whole body transplant is already used to mean transferring the brain between bodies, calling it a head transplant makes it clear that the whole head is to be switched, brain included.

Until recently, a head transplant seemed totally implausible, but the Italian scientist Dr Sergio Canavero believes its possible, and intends to conduct the first surgery in 2017.

Canavero outlines the procedure in detail here, but these are the basics of the process. Remember: dont try this at home, kids.

The donor body and the head to be attached are first cooled down to 12-15C to ensure that the cells last longer than a few minutes without oxygen. The tissue around the neck is then cut, with the major blood vessels linked with tiny tubes. The spinal cord on each party is then severed cleanly with an extremely sharp blade.

"Post coma, Canavero believes the patient would immediately be able to move, feel their face and even speak with the same voice."

At this point, the head is ready to be moved, and the two ends of the spinal cord are fused using a chemical called polyethylene glycol, encouraging the cells to mesh. This chemical has been shown to prompt the growth of spinal cord nerves in animals, although Canavero suggests that introducing stem cells or olfactory ensheathing cells into the spinal cord could also be tried.

After the muscles and blood supply are successfully connected, the patient is kept in a coma for a month to limit movement of the newly fused neck, while electrodes stimulate the spinal cord to strengthen its new connections.

Following the coma, Canavero anticipates that the patient would immediately be able to move, feel their face and even speak with the same voice. He believes physiotherapy would allow the patient to walk within a year.

He explains his suggested methods in the TED talk below.

Sceptical would be a nice way of putting it. Horrified would, in most cases, be more accurate.

Dr Hunt Batjer has attracted headlines for being particularly blunt: I would not wish this on anyone. I would not allow anyone to do it to me as there are a lot of things worse than death.

Dr Jerry Silver witnessed the 1970s monkey head transplant experiment more on which later and describes the procedure as bad science, adding that just to do the experiments is unethical. This is a particular blow to Canavero, as he states that Silvers own work in reconnecting rats spinal cords should give hope to the human head transplant. Silver dismisses this: To sever a head and even contemplate the possibility of gluing axons back properly across the lesion to their neighbours is pure and utter fantasy in my opinion.

Dr Chad Gordon, professor of plastic and reconstructive surgery and neurological surgery at Johns Hopkins University, agrees that Canaveros claims are scientifically implausible. He told BuzzFeed: Theres no way hes going to hook up somebodys brain to someones spinal cord and have them be functional.

On the conservative side, were about 100 years away from being able to figure this out, he continued. If hes saying two, and hes promising a living, breathing, talking, moving human being? Hes lying.

Dr Paul Myers, associate professor of biology at the University of Minnesota at Morris, puts it even more explicitly: This procedure will not work... Try it with monkeys first. But he cant: the result would be, at best, a shambling horror, an animal driven mad with pain and terror, crippled and whimpering, and a poor advertisement for his experiment. And most likely what hed have is a collection of corpses that suffered briefly before expiring.

Others wonder whether Canavero might simply be enjoying the limelight with a PR stunt, including Dr Arthur Caplan, director of ethics at the NYU Langone Medical Centre. Describing the doctor as nuts, he explained to CNN: Their bodies would end up being overwhelmed with different pathways and chemistry than theyre used to, and theyd go crazy.

"We'll probably see a head on a robot before we see it on [another] body," he told Live Science.

Dr John Adler of Stanford University's school of medicine is slightly more optimistic... but not much more. "Conceptually, much of this could work, but the most favourable outcome will be little more than a Christopher Reeve level of function," he told Newsweek.

Canavero is aware of this criticism, claiming that silently hes received a lot of support from the medical community. Of Dr Batjers comments that the surgery would be a fate worse than death, Canavero is scathing. Hes a vascular surgeon. A vascular surgeon of the brain, yes, but he knows nothing, he argued. How can you say such a thing? Its incredible.

"The world is moving, the critics are dwindling. Of course, there will always be critics. Science teaches us that when you propose something groundbreaking, you must be confronted by criticism. If no critics really step forward, you are saying nothing special," he told Medical News Today.

No-one has ever attempted a human head transplant before, and attempts on animals have to put it charitably had limited success.

Image: from Motherboard, uploaded under fair use from a 1959 issue of Life

The photo above really does show a dog with two heads and its not a fake. This was the work of Soviet scientist Vladimir Demikhov, and for four days the hybrid of two dogs lived as normally as such a scientific horror could be expected to. Then they died.

Demikhov tried the experiment more than 24 times, but was unable to find a way of avoiding the dogs dying shortly after surgery. Although the results are horrifying to see, Demikhovs research did pave the way for human organ transplants.

"For four days this hybrid of two dogs lived as normally as such a scientific horror could be expected to. Then they died."

But back to the topic of head transplants. The first time a straight swap was successful, was by Dr Robert White, in an experiment on a rhesus monkey in 1970. I feel the need to qualify the word successful with quotation marks, because although the monkey did live, he didnt live very long. Eight days, to be exact, and as the spinal cord wasnt attached to its new body, the monkey was paralysed for its remaining days. However, it could indeed see, hear, smell and taste before the body rejected the foreign head.

According to Canavero in his paper on human head transplants, the monkey lived eight days and was, by all measures, normal, having suffered no complications. However, Dr Jerry Silver who worked in the same lab as Dr White has more haunting memories. He toldCBS: I remember that the head would wake up, the facial expressions looked like terrible pain and confusion and anxiety in the animal. The head will stay alive, but not very long. It was just awful. I dont think it should ever be done again.

More recently, Chinese doctor Xiaoping Ren claims to have conducted head transplants on more than 1,000 mice. The Wall Street Journal reports to have witnessed a mouse with a new head moving, breathing, looking around and drinking. But, crucially, none of these mice have lived longer than a few minutes.

Still, Dr Rens studies continue, and the latest reports are said to be promising, offering a possible answer to the risk of severe blood loss (or brain ischemia) during transplantation. The experimental method that we have described can allow for long-term survival, and thus assessment of transplant rejection and central nervous system recovery, bringing us one step closer to AHBR in man, the researchers wrote.

Ren himself has not ruled out taking part in the first human head transplant operation, according to the Daily Mail. "A human head transplant will be a new frontier in science. Some people say it is the last frontier in medicine. It is a very sensitive and very controversial subject but if we can translate it to clinical practice, we can save a lot of lives," he said.

"Many people say a head transplant is not ethical. But what is the essence of a person? A person is the brain not the body. The body is just an organ," he added.

In January 2016, Canavero told New Scientist that a head transplant had been successfully completed on a monkey in China, although details were sparse. "The monkey fully survived the procedure without any neurological injury of whatever kind," he said, although the article notes that the monkey only kept alive for 20 hours after the surgery for "ethical reasons," limiting its use as a comparison somewhat.

In September 2016, Canavero revealeda further trial of the head transplant on dogs.New Scientisthas seen video footage of a dog appearing to walk three weeks after its spinal cord was severed, with Canavero claiming that the outcome is the result of the same techniques he plans to use on Spiridonov next year.

However, speaking to a number of scientists for their view on the new evidence, New Scientistcould find few sceptics converted. "These papers do not support moving forward in humans," said Jerry Silver a neuroscientist at Cape Western Reserve University in Ohio.

"The dog is a case report, and you cant learn very much from a single animal without controls. They claim they cut the cervical cord 90 per cent but theres no evidence of that in the paper, just some crude pictures," added Silver.

You could say so, though Canavero doesn't see it quite like that. In fact, controversially he sees it more as a failure of other types of medicine, telling Medical News Today, "It will be about curing incurable neurological disorders for which other treatments have failed big time, so gene therapy,stem cells- they all just came to nothing. We have failed despite billions of dollars being poured into this sort of research."

"So actually, head transplant or body transplant, whatever your angle is, is actually a failure of medicine. It is not a brilliant success, a brilliant advancement to medical science. When you just haven't tackled biology, you don't know how to treat genes, you don't really understand, and you really need to resort to a body transplant, it means that you've failed. So this must not be construed as a success of medical research," he added.

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A team of biomedical engineering researchers, led by the University of Minnesota, has created a revolutionary 3D-bioprinted patch that can help heal scarred heart tissue after a heart attack. The discovery is a major step forward in treating patients with tissue damage after a heart attack.

The research study is published today in Circulation Research, a journal published by the American Heart Association. Researchers have filed a patent on the discovery.

According to the American Heart Association, heart disease is the No. 1 cause of death in the U.S. killing more than 360,000 people a year. During a heart attack, a person loses blood flow to the heart muscle and that causes cells to die. Our bodies cant replace those heart muscle cells so the body forms scar tissue in that area of the heart, which puts the person at risk for compromised heart function and future heart failure.

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

Watch a video of the cells beating on the patch.

When the cell patch was placed on a mouse following a simulated heart attack, the researchers saw significant increase in functional capacity after just four weeks. Since the patch was made from cells and structural proteins native to the heart, it became part of the heart and absorbed into the body, requiring no further surgeries.

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

Ogle said that this research is different from previous research in that the patch is modeled after a digital, three-dimensional scan of the structural proteins of native heart tissue. The digital model is made into a physical structure by 3D printing with proteins native to the heart and further integrating cardiac cell types derived from stem cells. Only with 3D printing of this type can we achieve one micron resolution needed to mimic structures of native heart tissue.

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

Ogle said they are already beginning the next step to develop a larger patch that they would test on a pig heart, which is similar in size to a human heart.

The research was funded by the National Science Foundation, National Institutes of Health, University of Minnesota Lillehei Heart Institute, and University of Minnesota Institute for Engineering in Medicine.

In addition to Ogle, other biomedical engineering researchers who were part of the team include Molly E. Kupfer, Jangwook P. Jung, Libang Yang, Patrick Zhang, and Brian T. Freeman from the University of Minnesota; Paul J. Campagnola, Yong Da Sie, Quyen Tran, and Visar Ajeti from the University of Wisconsin-Madison; and Jianyi Zhang, Ling Gao, and Vladimir G. Fast from the University of Alabama,

To read the full research paper entitled Myocardial Tissue Engineering With Cells Derived from Human Induced-Pluripotent Stem Cells and a Native-Like, High-Resolution, 3-Dimensionally Printed Scaffold, visit the Circulation Research website.

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3D-printed patch can help mend a broken heart - UMN News

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Theraputic Stem Cell-Sheet Transplantation May Improve ... The Cardiology Advisor (registration) A small phase I study identified positive benefits from utilizing stem cell-sheet transplantation as a therapy for ischemic and dilated cardiomyopathy. SCOTS Eye Stem Cell Study Exceeds Research GoalsMilTech all 23 news articles »

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'Cradle of life' stem cells taken from skin samples were developed into three-dimensional brain-like organisms capable of exchanging signals between each other in a network.

The petri dish cells behave in a similar way to the brain cells which produce messenger dopamine from neurons - and scientists hope they will be able to use them to come up with a cure for Parkinson's.

Dopamine maintains smooth body movements, but when the neurons die off, tremors, rigid muscles and other Parkinson's disease symptoms begin to take over.

The new developments mean scientists can now use the cells to test what environmental factors like pollutants have on the onset of the disease and potentially find a cure.

Lead author Professor Jens Schwamborn said: "Our cell cultures open new doors to brain research.

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"We can now use them to study the causes of Parkinson's disease and how it could possibly be effectively treated."

Our cell cultures open new doors to brain research

Professor Jens Schwamborn

The stem cells can be transformed into any cell type of the human body but cannot produce a complete organism.

PHD student Anna Monzel developed a procedure to convert the stem cells into brain cells as part of her doctoral thesis.

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Tremor - One of the most noticeable signs of Parkinson's is a tremor that often starts in the hands or fingers when they are relaxed

She said: "I had to develop a special, precisely defined cocktail of growth factors and a certain treatment method for the stem cells, so that they would differentiate in the desired direction."

Prof Schwamborn from the Luxembourg Centre for Systems Biomedicine at Luxembourg University said: "Our subsequent examination of these artificial tissue samples revealed that various cell types characteristic of the midbrain had developed."

"The cells can transmit and process signals.

"We were even able to detect dopaminergic cells - just like in the midbrain."

The scientists say their petri dish study can also reduce the amount of animal testing in brain research.

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Because cell cultures in the petri dishes are of human origin in some aspects they resemble human brains more than the brains of lab animals such as rats or mice.

Professor Schwamborn added: "There are also attractive economic opportunities in our approach.

"The production of tissue cultures is highly elaborate.

"In the scope of our spin-off Braingineering Technologies Sarl, we will be developing technologies by which we can provide the cultures for a fee to other labs or the pharmaceutical industry for their research."

The study was published in the Stem Cell Reports journal.

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It would seem the beautiful foxglove plant has more uses than just the garden.

A novel drug screen in liver-like cells shows that cardiac glycosides, which are found in the leaves of the digitalis or foxglove plant, could reduce low-density lipoprotein (LDL) cholesterol differently than statins, potentially providing a new treatment for patients.

The foxglove plant in bloom on MUSC's campus.

These findings were reported by the Medical University of South Carolina researcher Stephen A. Duncan, D.Phil., SmartState Chair of Regenerative Medicine at MUSC, and colleagues in the April 6 issue of Cell Stem Cell.

Duncan said the glycosides were identified through a stem cell screen for compounds that could be used off-label for the treatment of high cholesterol. The nice thing about finding new uses for drugs already on the market is that they can be used relatively quickly in patients because most of the needed safety trials have already been completed.

Not everyone with high LDL cholesterol responds to statins. Statins increase levels of a cell surface receptor that removes LDL cholesterol from the bloodstream. However, statins do not work in patients with familial hypercholesterolemia (FH), who have a rare mutation in that receptor. It is an inherited disorder that leads to aggressive and premature cardiovascular disease. FH patients have very high cholesterol and can die of cardiovascular disease by their forties. The existing drugs for FH can cause fatty liver disease, and the best treatment is a liver transplant.

Duncan and his graduate student Max Cayo, who is finishing his M.D. at the Medical College of Wisconsin, developed a drug screen to identify an alternative to statins. Apolipoprotein B (apoB) is a molecule that liver cells use to make LDL. Drugs that decreased apoB could potentially lower cholesterol independently of the LDL receptor in FH patients and also in patients with other forms of high cholesterol.

FH was a perfect model for testing alternatives to statins. Yet the rarity of FH meant these liver cells were scarce. Duncans group made induced pluripotent stem cells out of skin fibroblasts taken from a single patient with FH. Stem cells continually double their numbers while in culture. This meant that a sample of converted skin cells from a single patient with FH provided a renewable source of liver-like cells that retained the mutation.

The group tested these liver-like cells with the SPECTRUM library, a collection of 2,300 pharmaceuticals, many of which have reached clinical trials. Surprisingly, all nine cardiac glycosides in the collection, some widely prescribed for heart failure, reduced apoB in liver-like cells from the patient with FH. In further tests, they also lowered apoB in human hepatocytes and in mice engineered to grow normal human livers without the FH mutation.

Next, the team combed through more than five thousand medical records of patients prescribed cardiac glycosides for heart failure who also had LDL cholesterol records. Similar drops in LDL levels were observed in these patients as in a matching group of patients prescribed statins.

This study provides the first evidence that cardiac glycosides could potentially reduce LDL cholesterol independently of the LDL receptor, where statins act, by reducing apoB.

The cardiac glycosides are always prescribed with care, as they are known to be toxic at high doses. However, they could offer inexpensive life-saving options for patients with FH. Additionally, a cardiac glycoside in a low dose could conceivably provide an added benefit to patients already taking a statin. Duncan is exploring plans for a clinical trial that would determine the correct dose in hypercholesterolemia patients.

Using patient stem cells to screen drugs that are already on the market is a great way to investigate treatments for liver diseases.

There are so few livers available for transplant, Duncan said. Having the stem cell model where we make liver cells in the culture dish opens up a possibility of using this not only to investigate a disease, but also as a way to discover drugs that could fix a disease.

This article has been republished frommaterialsprovided by theMedical University of South Carolina. Note: material may have been edited for length and content. For further information, please contact the cited source.

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GERMANTOWN, Md., April 12, 2017 (GLOBE NEWSWIRE) -- Neuralstem, Inc. (CUR), a biopharmaceutical company focused on the development of nervous system therapies based on its neural stem cell technology, announced that a new cohort of four patients will be added to its ongoing Phase 1 human clinical trial evaluating the safety and feasibility of using NSI-566 spinal cord-derived neural stem cells to repair chronic spinal cord injury (cSCI). The amended protocol was approved by the U.S. Food and Drug Administration and the Institutional Review Board at the study site, University of California San Diego (UCSD). NSI-566 is Neuralstems lead stem cell therapy candidate.

Under the amended protocol, updated on clinicaltrials.gov (NCT01772810), four qualifying patients with AIS-A complete, quadriplegic, cervical injuries involving C5-C7 cord will be added to the study. The injury must have occurred 1-2 years prior to the time of stem cell treatment, which is a one-time surgery involving six injections of NSI-566 into the affected area of the cord. The study has begun active recruitment of patients.

About 250,000 Americans are living with cSCI, and approximately 11,000 new injuries are reported each year1. Roughly 52% of these individuals will be considered paraplegic and 47% will be considered quadriplegic1. cSCI is a permanent and disabling condition with few to no treatments. Its devastating effect can be measured from social, healthcare, and economic perspectives.

This expansion of the study to cervical injuries builds on the results demonstrating that the implantation of NSI-566 stem cells in the first four patients with AIS-A complete thoracic cSCI was safe and feasible with no serious adverse events, said Karl Johe, Ph.D., Chief Scientific Officer, Neuralstem. There is a tremendous unmet need in the treatment of cSCI and we are privileged to have the experts at UCSD School of Medicine and the Sanford Stem Cell Clinical Center at UC San Diego Health conducting the research. We look forward to further evaluating NSI-566 neural stem cells in chronic complete cervical injuries."

Long-term safety data from the first cohort of chronic complete thoracic injuries is currently being analyzed by the study team at UCSD School of Medicine.

About Neuralstem Neuralstems patented technology enables the commercial-scale production of multiple types of central nervous system stem cells, which are being developed as potential therapies for multiple central nervous system diseases and conditions.

Neuralstems technology also enables the discovery of small molecule compounds by systematic screening chemical compounds against its proprietary human hippocampal stem cell line. The screening process has led to the discovery and patenting of molecules that Neuralstem believes may stimulate the brains capacity to generate new neurons, potentially reversing pathophysiologies associated with certain central nervous system (CNS) conditions.

The company has completed Phase 1a and 1b trials evaluating NSI-189, a novel neurogenic small molecule product candidate, for the treatment of major depressive disorder or MDD, and is currently conducting a Phase 2 efficacy study for MDD.

Neuralstems stem cell therapy product candidate, NSI-566, is a spinal cord-derived neural stem cell line. Neuralstem is currently evaluating NSI-566 in three indications: stroke, chronic spinal cord injury (cSCI), and Amyotrophic Lateral Sclerosis (ALS).

Neuralstem is conducting a Phase 1 safety study for the treatment of paralysis from chronic motor stroke at the BaYi Brain Hospital in Beijing, China. In addition, NSI-566 was evaluated in a Phase 1 safety study to treat paralysis due to chronic spinal cord injury as well as a Phase 1 and Phase 2a risk escalation, safety trials for ALS. Subjects from all three indications are currently in long-term observational follow-up periods to continue to monitor safety and possible therapeutic benefits.

Cautionary Statement Regarding Forward-Looking Information This news release contains forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements relate to future, not past, events and may often be identified by words such as expect, anticipate, intend, plan, believe, seek or will. Forward-looking statements by their nature address matters that are, to different degrees, uncertain. Specific risks and uncertainties that could cause our actual results to differ materially from those expressed in our forward-looking statements include risks inherent in the development and commercialization of potential products, uncertainty of clinical trial results or regulatory approvals or clearances, need for future capital, dependence upon collaborators and maintenance of our intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in Neuralstems periodic reports, including the Annual Report on Form 10-K for the year ended December 31, 2015, and Form 10-Q for the nine months ended September 30, 2016, filed with the Securities and Exchange Commission (SEC), and in other reports filed with the SEC. We do not assume any obligation to update any forward-looking statements.

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Germantown, Md.-based Neuralstem is involved in a Phase 1 human clinical trial testing the safety and feasibility of using NSI-566 spinal cord-derived neural stem cells to repair chronic spinal cord injury.

A biopharmaceutical company, Neuralstem develops nervous system therapies derived from neural stem cell technology.

Here are six takeaways:

1. NSI-566 represents the company's lead stem cell therapy candidate.

2. University of California San Diego serves as the study site, and just added a new cohort of four patients.

3. The four new patients all have AIS-A complete, quadriplegic, cervical injuries involving C5-C7 cord.

4. The patients suffered the injury one to two years before undergoing stem cell treatment.

5. The treatment involves six injections of NSI-566 into the spinal cord's affected area.

6. UCSD researchers are analyzing long-term safety data from the study's first cohort on chronic complete thoracic injuries.

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UC San Diego adds 4 patients to Neuralstem's neural stem cell study for spinal cord injury: 6 takeaways - Becker's Orthopedic & Spine

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'Neuron-reading' nanowires could accelerate development of drugs for neurological diseases Science Daily "We envision that this nanowire technology could be used on stem-cell-derived brain models to identify the most effective drugs for neurological diseases," said Anne Bang, director of cell biology at the Conrad Prebys Center for Chemical Genomics at ... and more »

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It will be several weeks before Jonathan Pitre finds out if his second stem-cell transplant was successful. Tina Boileau / -

The perilous wait now begins for Jonathan Pitre.

Pitre, 16, was transfused with blood and marrow drawn from his mothers hip late Thursday afternoon. The stem-cell rich material holds the power to alter the course of Pitres aggressive skin disease, epidermolysis bullosa (EB), and change his life.

So far, so good, saidPitres mother, Tina Boileau.

It will be several weeks before Pitre finds out whether the transplant has worked its magic.

While waiting for that answer, theRussell teenager will have to travel the most difficult part of his medical journey: a time when his immune system is at its lowest ebb, and when he feels the full effects of high-dose chemotherapy and radiation.

His physician, Dr. Jakub Tolar, has warned that the period represents the highest risk for complications, the most common of which are infections and graft-versus-host disease (GVHD). It is a potentially life-threatening situation in which the implanted stem cells produce T-cells that attack normal cells.

In about two weeks time, doctors will start to look for signs that Boileaus stem cells have successfully established themselves in Pitres bone marrow.The presence of white blood cells is one of the earliest signs of stem-cell growth; an improvement in the condition of Pitres skin could also signal that the stem cells have started to work.

Last year, after his first stem-cell transplant, Pitre and his mother were thrilled when doctors discovered new white cells in his bloodstream. But their hopes were crushed when tests showed Pitres own stem cells had recolonized his bone marrow, and were producing the cells.

This time, Boileau said, they will wait to see more lab results before getting their hopes too high.

I think we will have that uncertainly until we know for sure through skin and bone marrow biopsies that the engraftment worked, she said.

Boileau went into surgery early Thursday morning to have blood and bone marrow drawn from her hip. She was at her sons bedside later in the afternoon to watch as the stem cells dripped through an intravenous tube connected to the right atrium of her sons heart.

If the transplant works, Boileaus stem cells will establish themselves in her sons bone marrow, grow, divide and make new blood cells equipped with the power to provide Pitre with the key protein he needs to rebuild his damaged skin.

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His stem-cell transplant complete, the wait begins for Jonathan Pitre - Ottawa Citizen

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Brain tissue from a petri dish: Stem cell research -- ScienceDaily Science Daily The most complex organ in humans is the brain. Due to its complexity, it is extremely difficult to do scientific experiments on it -- ones that could help us to ... and more »

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