KAMLOOPS My curiosity was sparked when I read that a stem cell centre was opening in Kamloops (Kamloops This Week, March 21, 2017).

So I went to the location of the centre at 470 Columbia St only to find a parking lot. Thinking that the address might be wrong, I searched the directory of the medical building next door and found that no stem cell centre was listed.

The Stem Cell Centers website lists Kamloops as the only one in Canada. Dr. Richard Brownlee is named as the surgeon with more information coming soon.

Stem cell therapy, says the website, can help with orthopedic or pain management, ophthalmological conditions, cardiac or pulmonary conditions, neurological conditions, and auto-immune diseases, among many other conditions and disease that results in damaged tissue.

One of the ophthalmological conditions they treat is macular degeneration. If your vision is fading due to macular degeneration, you know its time to seek help. Our non-invasive Stem Cell Therapy treatment might be the solution for you.

I wanted get Dr. Brownlees reaction to news that an unproven stem cell treatment had resulted in blindness according to the New England Journal of Medicine as reported in the Globe and Mail, March 20, 2017.

This week, the New England Journal of Medicine (NEJM) reported on three individuals who went blind after receiving an unproven stem cell treatment at a Florida clinic. The patients paid thousands of dollars for what they thought was a clinical trial on the use of stem cells to treat macular degeneration.

The writer of the Globe and Mail article, Timothy Caulfield, Research Chair of the in Health Law and Policy at the University of Alberta, doesnt name the Florida clinic.

The Stem Cell Centers website refers optimistically to treatment for macular degeneration at a Florida clinic, although apparently not theirs since no Florida clinic appears on their list. It tells of how Doug Oliver suffered from macular degeneration before stem cells were extracted from his hip bone and injected them into his eyes. Almost immediately, Olivers eyesight started to improve. I began weeping, he said.

Caulfield encourages caution. Health science gets a lot of attention in the popular press. People love hearing about breakthroughs, paradigm shifts and emerging cures. The problem is, these stories are almost always misleading. It can also help to legitimize the marketing of unproven therapies.

Reports from the Stem Cell Centers own website are cautionary as well. It quotes an abstract from a study done by the Southern California College of Optometry on how stem cells might ultimately be used to restore the entire visual pathway.

The promise of stem cell research is phenomenal. Scientific American (Jan., 2017) reports that brains can be grown in a lab dish from stem cells taken from skin. These samples can be used to research brain disorders ranging from schizophrenia to Alzheimer's disease, and to explore why only some babies develop brain-shrinking microcephaly after exposure to the Zika virus.

However, Dr. George Daley, dean of Harvard Medical School, concludes that there are only a handful of clinical applications available and they are for skin and blood-related ailments.

Practice, it seems, has not yet matched the promise of stem cell research.

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Stem cell centre coming to Kamloops? | CFJC Today - CFJC Today Kamloops

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Stem cells are undifferentiated biological cells that can differentiate into specialized cells and can divide (through mitosis) to produce more stem cells. They are found in multicellular organisms. In mammals, there are two broad types of stem cells: embryonic stem cells, which are isolated from the inner cell mass of blastocysts, and adult stem cells, which are found in various tissues. In adult organisms, stem cells and progenitor cells act as a repair system for the body, replenishing adult tissues. In a developing embryo, stem cells can differentiate into all the specialized cellsectoderm, endoderm and mesoderm (see induced pluripotent stem cells)but also maintain the normal turnover of regenerative organs, such as blood, skin, or intestinal tissues.

There are three known accessible sources of autologous adult stem cells in humans:

Stem cells can also be taken from umbilical cord blood just after birth. Of all stem cell types, autologous harvesting involves the least risk. By definition, autologous cells are obtained from ones own body, just as one may bank his or her own blood for elective surgical procedures.

Adult stem cells are frequently used in medical therapies, for example in bone marrow transplantation. Stem cells can now be artificially grown and transformed (differentiated) into specialized cell types with characteristics consistent with cells of various tissues such as muscles or nerves. Embryonic cell lines and autologous embryonic stem cells generated through Somatic-cell nuclear transfer or dedifferentiation have also been proposed as promising candidates for future therapies.[1] Research into stem cells grew out of findings by Ernest A. McCulloch and James E. Till at the University of Toronto in the 1960s.[2][3]

The classical definition of a stem cell requires that it possess two properties:

Two mechanisms exist to ensure that a stem cell population is maintained:

Potency specifies the differentiation potential (the potential to differentiate into different cell types) of the stem cell.[4]

In practice, stem cells are identified by whether they can regenerate tissue. For example, the defining test for bone marrow or hematopoietic stem cells (HSCs) is the ability to transplant the cells and save an individual without HSCs. This demonstrates that the cells can produce new blood cells over a long term. It should also be possible to isolate stem cells from the transplanted individual, which can themselves be transplanted into another individual without HSCs, demonstrating that the stem cell was able to self-renew.

Properties of stem cells can be illustrated in vitro, using methods such as clonogenic assays, in which single cells are assessed for their ability to differentiate and self-renew.[7][8] Stem cells can also be isolated by their possession of a distinctive set of cell surface markers. However, in vitro culture conditions can alter the behavior of cells, making it unclear whether the cells will behave in a similar manner in vivo. There is considerable debate as to whether some proposed adult cell populations are truly stem cells.

Embryonic stem (ES) cells are stem cells derived from the inner cell mass of a blastocyst, an early-stage embryo.[9] Human embryos reach the blastocyst stage 45 days post fertilization, at which time they consist of 50150 cells. ES cells are pluripotent and give rise during development to all derivatives of the three primary germ layers: ectoderm, endoderm and mesoderm. In other words, they can develop into each of the more than 200 cell types of the adult body when given sufficient and necessary stimulation for a specific cell type. They do not contribute to the extra-embryonic membranes or the placenta.

Nearly all research to date has made use of mouse embryonic stem cells (mES) or human embryonic stem cells (hES). Both have the essential stem cell characteristics, yet they require very different environments in order to maintain an undifferentiated state. Mouse ES cells are grown on a layer of gelatin as an extracellular matrix (for support) and require the presence of leukemia inhibitory factor (LIF). Human ES cells are grown on a feeder layer of mouse embryonic fibroblasts (MEFs) and require the presence of basic fibroblast growth factor (bFGF or FGF-2).[10] Without optimal culture conditions or genetic manipulation,[11] embryonic stem cells will rapidly differentiate.

A human embryonic stem cell is also defined by the expression of several transcription factors and cell surface proteins. The transcription factors Oct-4, Nanog, and Sox2 form the core regulatory network that ensures the suppression of genes that lead to differentiation and the maintenance of pluripotency.[12] The cell surface antigens most commonly used to identify hES cells are the glycolipids stage specific embryonic antigen 3 and 4 and the keratan sulfate antigens Tra-1-60 and Tra-1-81. The molecular definition of a stem cell includes many more proteins and continues to be a topic of research.[13]

There are currently no approved treatments using embryonic stem cells. The first human trial was approved by the US Food and Drug Administration in January 2009.[14] However, the human trial was not initiated until October 13, 2010 in Atlanta for spinal injury victims. On November 14, 2011 the company conducting the trial announced that it will discontinue further development of its stem cell programs.[15] ES cells, being pluripotent cells, require specific signals for correct differentiationif injected directly into another body, ES cells will differentiate into many different types of cells, causing a teratoma. Differentiating ES cells into usable cells while avoiding transplant rejection are just a few of the hurdles that embryonic stem cell researchers still face.[16] Many nations currently have moratoria on either ES cell research or the production of new ES cell lines. Because of their combined abilities of unlimited expansion and pluripotency, embryonic stem cells remain a theoretically potential source for regenerative medicine and tissue replacement after injury or disease.

Human embryonic stem cell colony on mouse embryonic fibroblast feeder layer

The primitive stem cells located in the organs of fetuses are referred to as fetal stem cells.[17] There are two types of fetal stem cells:

Adult stem cells, also called somatic (from Greek , of the body) stem cells, are stem cells which maintain and repair the tissue in which they are found.[19] They can be found in children, as well as adults.[20]

Pluripotent adult stem cells are rare and generally small in number, but they can be found in umbilical cord blood and other tissues.[21] Bone marrow is a rich source of adult stem cells,[22] which have been used in treating several conditions including spinal cord injury,[23] liver cirrhosis,[24] chronic limb ischemia [25] and endstage heart failure.[26] The quantity of bone marrow stem cells declines with age and is greater in males than females during reproductive years.[27] Much adult stem cell research to date has aimed to characterize their potency and self-renewal capabilities.[28] DNA damage accumulates with age in both stem cells and the cells that comprise the stem cell environment. This accumulation is considered to be responsible, at least in part, for increasing stem cell dysfunction with aging (see DNA damage theory of aging).[29]

Most adult stem cells are lineage-restricted (multipotent) and are generally referred to by their tissue origin (mesenchymal stem cell, adipose-derived stem cell, endothelial stem cell, dental pulp stem cell, etc.).[30][31]

Adult stem cell treatments have been successfully used for many years to treat leukemia and related bone/blood cancers through bone marrow transplants.[32] Adult stem cells are also used in veterinary medicine to treat tendon and ligament injuries in horses.[33]

The use of adult stem cells in research and therapy is not as controversial as the use of embryonic stem cells, because the production of adult stem cells does not require the destruction of an embryo. Additionally, in instances where adult stem cells are obtained from the intended recipient (an autograft), the risk of rejection is essentially non-existent. Consequently, more US government funding is being provided for adult stem cell research.[34]

Multipotent stem cells are also found in amniotic fluid. These stem cells are very active, expand extensively without feeders and are not tumorigenic. Amniotic stem cells are multipotent and can differentiate in cells of adipogenic, osteogenic, myogenic, endothelial, hepatic and also neuronal lines.[35] Amniotic stem cells are a topic of active research.

Use of stem cells from amniotic fluid overcomes the ethical objections to using human embryos as a source of cells. Roman Catholic teaching forbids the use of embryonic stem cells in experimentation; accordingly, the Vatican newspaper Osservatore Romano called amniotic stem cells the future of medicine.[36]

It is possible to collect amniotic stem cells for donors or for autologuous use: the first US amniotic stem cells bank [37][38] was opened in 2009 in Medford, MA, by Biocell Center Corporation[39][40][41] and collaborates with various hospitals and universities all over the world.[42]

These are not adult stem cells, but rather adult cells (e.g. epithelial cells) reprogrammed to give rise to pluripotent capabilities. Using genetic reprogramming with protein transcription factors, pluripotent stem cells equivalent to embryonic stem cells have been derived from human adult skin tissue.[43][44][45]Shinya Yamanaka and his colleagues at Kyoto University used the transcription factors Oct3/4, Sox2, c-Myc, and Klf4[43] in their experiments on cells from human faces. Junying Yu, James Thomson, and their colleagues at the University of WisconsinMadison used a different set of factors, Oct4, Sox2, Nanog and Lin28,[43] and carried out their experiments using cells from human foreskin.

As a result of the success of these experiments, Ian Wilmut, who helped create the first cloned animal Dolly the Sheep, has announced that he will abandon somatic cell nuclear transfer as an avenue of research.[46]

Frozen blood samples can be used as a source of induced pluripotent stem cells, opening a new avenue for obtaining the valued cells.[47]

To ensure self-renewal, stem cells undergo two types of cell division (see Stem cell division and differentiation diagram). Symmetric division gives rise to two identical daughter cells both endowed with stem cell properties. Asymmetric division, on the other hand, produces only one stem cell and a progenitor cell with limited self-renewal potential. Progenitors can go through several rounds of cell division before terminally differentiating into a mature cell. It is possible that the molecular distinction between symmetric and asymmetric divisions lies in differential segregation of cell membrane proteins (such as receptors) between the daughter cells.[48]

An alternative theory is that stem cells remain undifferentiated due to environmental cues in their particular niche. Stem cells differentiate when they leave that niche or no longer receive those signals. Studies in Drosophila germarium have identified the signals decapentaplegic and adherens junctions that prevent germarium stem cells from differentiating.[49][50]

Diseases and conditions where stem cell treatment is being investigated include:

Stem cell therapy is the use of stem cells to treat or prevent a disease or condition. Bone marrow transplant is a crude form of stem cell therapy that has been used clinically for many years without controversy. No stem cell therapies other than bone marrow transplant are widely used.[64][65]

Research is underway to develop various sources for stem cells, and to apply stem cell treatments for neurodegenerative diseases and conditions, diabetes, heart disease, and other conditions.[66]

In more recent years, with the ability of scientists to isolate and culture embryonic stem cells, and with scientists growing ability to create stem cells using somatic cell nuclear transfer and techniques to created induced pluripotent stem cells, controversy has crept in, both related to abortion politics and to human cloning.

Stem cell treatments may require immunosuppression because of a requirement for radiation before the transplant to remove the patients previous cells, or because the patients immune system may target the stem cells. One approach to avoid the second possibility is to use stem cells from the same patient who is being treated.

Pluripotency in certain stem cells could also make it difficult to obtain a specific cell type. It is also difficult to obtain the exact cell type needed, because not all cells in a population differentiate uniformly. Undifferentiated cells can create tissues other than desired types.[67]

Some stem cells form tumors after transplantation; pluripotency is linked to tumor formation especially in embryonic stem cells, fetal proper stem cells, induced pluripotent stem cells. Fetal proper stem cells form tumors despite multipotency.[citation needed]

Hepatotoxicity and drug-induced liver injury account for a substantial number of failures of new drugs in development and market withdrawal, highlighting the need for screening assays such as stem cell-derived hepatocyte-like cells, that are capable of detecting toxicity early in the drug development process.[68]

Some of the fundamental patents covering human embryonic stem cells are owned by the Wisconsin Alumni Research Foundation (WARF) they are patents 5,843,780, 6,200,806, and 7,029,913 invented by James A. Thomson. WARF does not enforce these patents against academic scientists, but does enforce them against companies.[69]

In 2006, a request for the US Patent and Trademark Office (USPTO) to re-examine the three patents was filed by the Public Patent Foundation on behalf of its client, the non-profit patent-watchdog group Consumer Watchdog (formerly the Foundation for Taxpayer and Consumer Rights).[69] In the re-examination process, which involves several rounds of discussion between the USTPO and the parties, the USPTO initially agreed with Consumer Watchdog and rejected all the claims in all three patents,[70] however in response, WARF amended the claims of all three patents to make them more narrow, and in 2008 the USPTO found the amended claims in all three patents to be patentable. The decision on one of the patents (7,029,913) was appealable, while the decisions on the other two were not.[71][72] Consumer Watchdog appealed the granting of the 913 patent to the USTPOs Board of Patent Appeals and Interferences (BPAI) which granted the appeal, and in 2010 the BPAI decided that the amended claims of the 913 patent were not patentable.[73] However, WARF was able to re-open prosecution of the case and did so, amending the claims of the 913 patent again to make them more narrow, and in January 2013 the amended claims were allowed.[74]

In July 2013, Consumer Watchdog announced that it would appeal the decision to allow the claims of the 913 patent to the US Court of Appeals for the Federal Circuit (CAFC), the federal appeals court that hears patent cases.[75] At a hearing in December 2013, the CAFC raised the question of whether Consumer Watchdog had legal standing to appeal; the case could not proceed until that issue was resolved.[76]

Read more: Stem cell Wikipedia, the free encyclopedia

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Stem cell Wikipedia, the free encyclopedia IPS Cell ...

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In Brief A Japanese man has become the first recipient of donated, reprogrammed stem cells as a treatment for macular degeneration. If the treatment proves effective against the age-related eye condition, it could halt or prevent the vision loss of the 10 million people in the U.S. who have macular degeneration. A New Treatment for Macular Degeneration

Macular degeneration is the leading cause of progressive vision loss with almost 10million Americans affected by the disease. Currently, there are no known cures for the conditionalthough stem cells might change that entirely.

Macular degeneration occurs when the central portion, the macula, of the retina is deteriorated. This is where our eyes record images and send them to the brain through the optic nerve. The macula is known for focusing our vision, controlling our ability to read, recognize faces, and see objects clearly.

A Japaneseman in his sixties is the worlds first person to receive induced pluripotent stem (iPS) cells donated by a different individual. Rather than tip-toeing around the ethics of embryonic stem cells, scientists were able to remove mature cells from a donor and reprogram them into an embryonic state, which then could be developed into a specific cell-type to treat the disease. Physicians cultivated donated skin cells that were transplanted onto the mans retina to halt the progression of his age-related macular degeneration.

While the mans first surgery was a success, the doctors have said they will make no more announcements about his progress until they have completed all five of the planned procedures. While the effectiveness of this technique cannot be evaluated until the fate of the donated cells and the progression of the patientsmacular degenerationhave been fully monitored, there is increasing interest inusing iPScells for theraputic purposes.

A similar therapy was performed at the Kobe City Medical Center General Hospital in Japan in September 2014, but with a slight difference. In this case, the patient received her own skin cells reprogrammed into retinal cells. As hoped, a year after the surgery her vision had no decline, seemingly halting the macular degeneration. Four more patients in the clinical trial are expected to receive donor cells as well.

The donor-cell procedure, if successful, could help pave the way for the iPS cell bank thatShinya Yamanaka is establishing. An iPS cell bank would allow physicians find theperfect iPS donor per each patients biological signatures. Yamanaka is a Nobel-prizewinning scientist at Kyoto University who pioneered the iPS cells.

Yamanakas idea of a iPS cell bank has the potential torevolutionize modern medicine. It would provide patients with ready-made cells immediately, givinga widespread population access to more treatment options bylower all-around costs. While the risk of genetic defects or a poor donor match still remains, the new procedurecould offer enormous advantagescompared toother alternatives.

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A Japanese Man Has Become the First Recipient of Donated ... - Futurism

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If death was imminent, would you consider cryogenically freezing yourself, with the hopes that one day future technology would bring you back to life? Battling with brain cancer, thats what 22 year old Kim Suozzi did, and there are others just like her! But does this have any basis in science? Trace has the answers!

Read More: http://www.wired.com/rawfile/2012/10/... http://www.kurzweilai.net/cryonics-ph... http://www.guardian.co.uk/science/200... http://www.kurzweilai.net/kim-suozzi-... http://ieet.org/index.php/IEET/more/V... http://abcnews.go.com/Health/life-ice... http://cryonics.org/prod.html http://www.kurzweilai.net/a-chance-to... http://www.alcor.org/donate/KimSuozzi... http://venturist.info/kim-suozzi-char... http://io9.com/5940085/futurists-set-... http://io9.com/5977640/23+year-old-ki... http://science.howstuffworks.com/dict... http://betabeat.com/2013/01/cancer-su...

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Written by Anders Sandberg

This post was originally published onAndert II

A girl dying of cancer wanted to use cryonic preservation to have a chance at being revived in the future. While supported by her mother the father disagreed; in a recent high court ruling, the judge found that she could be cryopreserved.

As the judge noted, the verdict was not a statement on the validity of cryonics itself, but about how to make decisions about prospective orders. In many ways the case would presumably have gone the same way if there had been a disagreement about whether the daughter could have catholic last rites. However, cryonics makes things fresh and exciting (I have been in the media all day thanks to this).

What is the ethics of parents disagreeing about the cryosuspension of their child?

One obvious principle is that parents ought to act in the best interest of their children.

If the child is morally mature and with informed consent, then they can clearly have a valid interest in taking a chance on cryonics: they might not be legally adult, but as in normal medical ethics their stated interests have strong weight. Conversely, one could imagine a case where a child would not want to be preserved, in which case I think most people would agree their preferences should dominate.

The general legal consensus in the West is that the childs welfare is so important that it can overrule the objections of parents. In UK law parents have the right and the duty to give consent for a minor. Children can consent for medical treatment, overriding their parents, at 16. However, if refusing treatment parents and court can override. This mostly comes into play in cases such as avoiding blood transfusions for religious reasons.

In this case the issue was that the parents were disagreeing and the child was not legally old enough.

If one thinks cryonics is reasonable, then one should clearly cryosuspend the child: it is in their best interest. But if one thinks cryonics is not reasonable, is it harming the interest of the child? This seems to require some theory of how cryonics is bad for the interests of the child.

As an analogy, imagine a case where one parent is a Jehovahs Witness and want to refuse a treatment involving blood transfusion: the child will die without the treatment, and it will be a close call even with it. Here the objecting parent may claim that undergoing the transfusion harms the child in an important spiritual way and refuse consent. The other parent disagrees. Here the law would come down on the side of the pro-transfusion parent.

On this account and if we agree the cases are similar, we might say that parents have a legal duty to consent to cryonics.

In practice the controversialness of cryonics may speak against this: many people disagree about cryonics being good for ones welfare. However, most such arguments usually seem to be based on various farfetched scenarios about how the future could be a bad place to end up in. Others bring up loss of social connections or that personal identity would be disrupted. A more rational argument is that it is an unproven treatment of dubious efficacy, which would make it irrational to undertake if there was an alternative; however since there isnt any alternative this argument has little power. The same goes for the risk of loss of social connection or identity: had there been an alternative to death (which definitely severs connections and dissolves identity) that may have been preferable. If one seriously thinks that the future would be so dark that it is better not to get there, one should probably not have children.

In practice it is likely that the status of cryonics as nonstandard treatment would make the law hesitate to overrule parents. We know blood transfusions work, and while spiritual badness might be a respectable as a private view we as a society do not accept it as a sufficient reason to have somebody die. But in the case of cryonics the unprovenness of the treatment means that hope for revival is on nearly the same epistemic level as spiritual badness: a respectable private view, but not strong enough to be a valid public reason. Cryonicists are doing their best to produce scientific evidence tissue scans, memory experiments, protocols that move the reasons to believe in cryonics from the personal faith level to the public evidence level. They already have some relevant evidence. As soon as lab mice are revived or people become convinced the process saves the connectome the reasons would be strengthened and cryonics becomes more akin blood transfusion.

The key difference is that weak private reasons are enough to allow an experimental treatment where there is no alternative but death, but they are generally not enough to go for an experimental treatment when there is some better treatment. When disallowing a treatment weak reasons may work well against unproven or uncertain treatments, but not when it is proven. However, disallowing a treatment with no alternative is equivalent to selecting death.

When two parents disagree about cryonics (and the child does not have a voice) it hence seems that they both have weak reasons, but the asymmetry between having a chance and dying tilts in favor of cryonics. If it was purely a matter of aesthetics or value (for example, arguing about the right kind of last rites) there would be no societal or ethical constraint. But here there is some public evidence, making it at least possible that the interests of the child might be served by cryonics. Better safe than sorry.

When the child also has a voice and can express its desires, then it becomes obvious which way to go.

King Solomon might have solved the question by cryosuspending the child straight away, promising the dissenting parent not to allow revival until they either changed their mind or there was enough public evidence to convince anybodythat it would be in the childs interest to be revived. The nicest thing about cryonics is that it buys you time to think things through.

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Cross Post: Solomon's frozen judgement - Practical Ethics (blog)

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Miami Herald

Cutting-edge gene therapy provides hope for patients with inherited eye conditions Miami Herald The trial will evaluate a cutting-edge concept: gene therapy. While there are no gene therapy products currently approved for use in the U.S., researchers are experimenting to see if they can provide a solution to alleviate hereditary diseases. Gene ...

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Cutting-edge gene therapy provides hope for patients with inherited eye conditions - Miami Herald

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The development of gene therapy vectors for the hereditary immunodeficiency Chronic Granulomatous Disease (CGD) is hampered by the absence of human cell lines, necessary for rapid and effective gene therapy vector testing. A new model supported by EU funding can make the process more efficient.

CI Photos, Shutterstock

When no donor matches, a few locations globally will carry out gene therapy. But for this to be used clinically, the efficacy of the treatment has to be established. The testing process depends on cellular models and currently the process is labour intensive, lengthy and expensive.

A team of researchers headed by Dr Janine Reichenbach, a professor and Co-Head of the Division of Immunology at the University Children's Hospital Zurich, has developed a new cellular model that enables researchers to test the efficacy of new gene therapies much more efficiently.

We used Crispr/Cas9 technology to change a human cell line so that the blood cells show the genetic change typical of a specific form of Chronic Granulomatous Disease, explains Professor Reichenbach.

The standard way of testing has been to use patients skin cells that are reprogrammed into stem cells, a time consuming and expensive process. The new testing system will be more cost effective. Our system makes the process faster and cheaper which means well be able to develop new gene therapies for affected patient more efficiently, she adds.

In more detail, to test the vectors of potential benefit to people suffering from p47phox-deficient chronic granulomatous disease (CGD), the article published in the journal Scientific Reports explains researchers have generated a cellular model consisting of regularly interspaced, short palindromic repeats (CRISPR)/Cas9. This introduces a GT-dinucleotide deletion (GT) mutation in p47phox encoding NCF1 gene in the human acute myeloid leukemia PLB-985 cell line.

The research has also lead to a different approach to the transfer of healthy copies of the gene into the affected cells. So far therapies have used modified, artificial viruses as transporters, but some patients went on to develop cancer so this first generation of viral correction systems is now outdated. Dr Reichenbachs team now use lentiviral, self-inactivating gene therapy that she describes as more efficient and safer.

She believes these to be an intermediate stage only, and looks forward to the future use of genome editing to provide greater precision. However, Science Daily suggests this will take another five to six years before precision gene surgery is available clinically.

An exciting approach to treatment furthered by EU support

CELL-PID (Advanced cell-based therapies for the treatment of Primary ImmunoDeficiency) and NET4CGD (Gene therapy for X-linked Chronic Granulomatous Disease - CGD) are two projects that have received funding from the EU. The University Children's Hospital Zurich is one of three European centers able to use this new gene therapy in an international clinical phase I/II study to treat patients with Chronic Granulomatous Disease as part of NET4CGD.

For more information, please see: NET4CGD project website CELL-PID CORDIS project webpage

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Faster way to test new gene therapies - Cordis News

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Core Care adding primary care and other medical services The San Luis Obispo Tribune Cambrians soon will have more in-town choices for medical care, with family practice, primary care and regenerative medicine coming to Core Care in early May, according to the clinic's founder Kirk Azevedo. He said that CORECareMD is partnering with ...

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Core Care adding primary care and other medical services - The San Luis Obispo Tribune

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Read More: benefit, Bingo, Diversity Richmond, drag bingo, drag performers, fundraiser

Bingo: its not just for blue-haired grandmas and church basement fundraisers anymore.

Diversity Richmond is set to host its Drag Bingo and the Party Thereafter, an event that mixes grandmas bingo night with all the fabulous flare of drag.

Robyn Bentley, Special Events/Fundraising Chair for Diversity, is thrilled to have Diversity hosting Drag Bingo.

LGBT folks and their allies love bingo and drag, she said. Its a way for us to all get together and help raise money and have a lot of fun.

Bentley has a long history of LGBTQ advocacy, starting as a volunteer for the Triad Health Project on the AIDS Memorial Quilt display at the Greensboro Coliseum. Her first taste of Drag Bingo was as the Ball Girl for Philadelphia AIDS Funds GayBingo! Monthly fundraiser.

When asked about some of her favorite memories, Bentley immediately laughs and brings up O69.

Whenever O69 is called, everyone stands up and says ooohhhhh 69! she explained. So, theres a little wink-wink joke there.

This isnt the first fundraising event for Diversity Richmond, which feature events such as art shows, support groups, and social hours, and its not even its first bingo event.

We had a bingo in December at Diversity Richmond, and people liked it a lot, she said. However, Bentley explained that the previous event was hosted by traditional bingo providers, without any sequins or glitter in sight.

This event is set to have all the bells and whistles (and glitz and glam).

Its different than regular bingo because theres drag numbers during bingo, she said. Then, theres an after-party with a drag show.

Zakia McKensey, under the name Zakia Jamaceye, will be the hostess for the Drag Bingo event.

This will be my first time actually hosting a drag bingo, she said, but Ive been affiliated with Diversity Richmond for seven or eight years.

McKensey is a prominent figure in Richmond as an advocate for the transgender community and the LGBTQ community at large. She has previously worked at Health Brigade, formerly the Fan Free Clinic, in HIV prevention and was also one of the pioneers behind its transgender health clinic, providing local trans clients with hormone replacement therapy and overall healthcare.

She is also the C.E.O. of Nationz Pageantry System, which showcases talents of male entertainers and female impersonators. Not only is Mz. Nationz 2016, Tarena McCray, scheduled to make an appearance, but Nationaz Pageantry will be holding more events at Diversity in the upcoming year, including the 2017 Duke & Duchess Closetball.

In short, this is not McKenseys first event with the organization and certainly wont be the last.

Diversity Richmond provides a space where people feel safe and people can come have a good time, she said. Theyre doing a lot of great things in the community to create events that honor members in the community or provide fun, safe spaces or educational spaces for people in our community.

McKensey said attendees can expect a fun-filled night at the Drag Bingo event while supporting a good cause.

High energy performances, lots of laughter and fun, raising money for Diversity, and also winning some money with bingo.

Depending on how Drag Bingo performs, it may become a regular staple at Diversity.

Were trying different things, Bentley said. Were trying this to see how this goes and see how people like it, and if lots of people show up, it will give us a lot of incentive to do another.

Regardless, the event is set to be full of laughter, glitter, and prizes.

Its just like grandmas bingo but way more fun, she said. And, you have a chance to win $1,000 so come on out.

Drag Bingo will be held on Friday, March 31 at Diversity Richmonds Event Hall at 1407 Sherwood Ave in Richmond. The 18+ event will start at 7pm, $15 gets you seven $100 games plus entry to the Party Thereafter, while party-only entry starts at 9pm for $5. Extra game packs and Instant Bingo will be available for purchase. Wine & beer will be available after 9pm and food will be catered by Nacho Mamas.

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Arts & Events - GayRVA

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Utah's New Anti-Abortion Law Deliberately Puts Women's Lives At Risk The Daily Banter ... blocks the production of the "pregnancy hormone" progesterone. Without this hormone, the lining of the uterus breaks down and the pregnancy is no longer viable. This can happen in a few hours or a few days. Mifepristone is given to the patient at ...

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Utah's New Anti-Abortion Law Deliberately Puts Women's Lives At Risk - The Daily Banter

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Scientists have unveiled a five-organ female reproductive system on a chip small enough to hold in your hand. Photo by HyacinthEmpinao/STAT

BOSTON So-called organs on a chip small blobs of tissue growing in lab dishes that mimic the function of their human counterparts have promise for basic science and drug development. And those efforts are scaling up. On Tuesday scientists unveiled a five-organ female reproductive system on a chip small enough to hold in your hand, and showed that it could simulate a 28-day menstrual cycle.

The chip is part of an effort funded by the National Institutes of Health to build an entire human body-on-a-chip a creation that would involve all of the organ systems and allow researchers to run unprecedentedly precise experiments on human tissue. Other research groups are also working on chips that mimic multiple organs, for instance the liver, heart, and blood vessels.

In this case, the chip is about the size of a hardcover book and studded with Lego-like blocks, each of which is hollowed-out and holds bits of tissue growing on plastic scaffolding: ovary, fallopian tube, uterus, cervix, liver. (The ovary samples come from mice ovaries are rarely removed from healthy women while the tissue for the fallopian tubes, uterus, and cervix comes from women who had hysterectomies.) The blocks are connected by minuscule tubes to simulate how the real organs communicate with each other in the human body.

Those tubes allow hormones to flow between the miniature organs. By feeding the right cocktail of hormones into the ovary block, the researchers were able to coax the miniature organ to release an egg, and to produce hormones that flowed into each organ downstream, causing them to behave similarly to how they do in the human body.

READ MORE: From ovary to uterus: studying the overlooked transport in between

This is the first menstrual cycle on-a-chip, said Teresa Woodruff, the studys primary investigator and a professor of obstetrics and gynecology at Northwestern University Feinberg School of Medicine.

The results were published Tuesday in Nature Communications.

All about EVATAR

The chip dubbed EVATAR, a portmanteau of the biblical Eve and a representative avatar could someday be used to test the effects of drugs on human tissues before putting them into the human body. Right now, animals play that role but scientists wonder if animals different physiology might be one reason that so many drugs never make it into the clinic.

Building the EVATAR was a team effort, with multiple groups working to build the organ systems and a crack team of biomedical engineers in Cambridge, Mass., handling the design of the chip itself.

Jonathan Coppeta, a biomedical engineer at the Charles Stark Draper Laboratory, was part of that team, which built the piping system that moves fluid between the organ blocks. Its controlled by 62 pumps that can be turned on and off independently. Each of those pumps uses a pinky-sized electromagnet to move about a millionth of a liter of fluid at a time.

Such a precisely controllable system allows scientists to do things that wouldnt be possible to do in a real person, like change the rate at which hormones flow from one organ to another, to study the effect of that hormone on the organs.

READ MORE: Inside the sci-fi world of growing human tissue and organs in the lab

But because its still early days in the organ on-a-chip development, there are lots of unknowns. Researchers will inevitably face the question: If it doesnt kill the chip, does that mean its safe in a person?

Could it potentially be better than an animal model? said Jeffrey Borenstein, a biomedical engineer at Draper. Yes, because youre using human cells. Is it perfect? No, because there are always going to be limitations.

Reproductive biology researchers unaffiliated with the project noticed one particular limitation in the teams model of a uterus. The lining of a human uterus consists mainly of two types of cells but on the chip its primarily one type of cell, pointed out Warren Nothnick, vice chairman of the department of molecular and integrative physiology at the University of Kansas Medical Center. Nothnick said that could hinder the systems human veracity, because the underrepresented cell type gives rise to endometrial cancer. But altogether, he said, the paper is really groundbreaking.

Dr. Julie Kim, a Northwestern professor who led the team that built the uterus block, said that encouraging this particular type of cells known as endometrial cells to grow properly in lab-built organs is an open challenge. She hopes to build more life-like uteri in the future: My dream is to create a menstruating uterus in a dish.

Pharma showing interest

One of Woodruffs next steps is building personalized EVATARs, whose miniature organs are grown out of stem cells from individual people. That could allow researchers to test how a drug would impact a particular person, based on their biology.

Men have to wait, but perhaps not long within a year, Woodruff hopes to have more results to share about the male version of the project, nicknamed ADATAR.

Already pharmaceutical companies are starting to show interest Woodruff said that she has tested some AstraZeneca drug candidates to gauge their impact on the female reproductive system.

Drapers chip can support up to 12 organs, so researchers could use this chip to simulate different organ systems. The lab is also using similar technology to build custom chips for pharmaceutical companies like Pfizer.

Meanwhile, Woodruff is looking for participants to provide stem cells that could be used to make custom organ systems, which she said should happen over the next year.

This article is reproduced with permission from STAT. It was first published on March 28, 2017. Find the original story here.

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How to build a female reproductive system that fits in the palm of your hand - PBS NewsHour

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Having someone elses marrow or stem cells is called a donor transplant, or an allogeneic transplant. This is pronounced a-low-gen-ay-ik.

The donors bone marrow cells must match your own as closely as possible. The most suitable donor is usually a close relative, such as a brother or sister. It is sometimes possible to find a match in an unrelated donor. Doctors call this a matched unrelated donor (MUD). To find out if there is a suitable donor for you, your doctor will contact The Anthony Nolan Bone Marrow Register.

To make sure that your donors cells match, you and the donor will have blood tests. These are to see how many of the proteins on the surface of their blood cells match yours. This is called tissue typing or HLA matching. HLA stands for human leucocyte antigen.

Once you have a donor and are in remission, you have your high dose chemotherapy and radiotherapy. A week later the donor comes into hospital and their stem cells or marrow are collected.

You then have the stem cells or bone marrow as a drip through your central line.

If you've had a transplant from a donor, there is a risk of graft versus host disease (GVHD). This happens because the transplanted stem cells or bone marrow contain cells from your donor's immune system. These cells can sometimes recognise your own tissues as being foreign and attack them. This can be an advantage as the immune cells may also attack cancer cells left after your treatment.

Acute GVHD starts within 100 days of the transplant and can cause

If you develop GVHD after your transplant, your doctor will prescribe drugs to damp down this immune reaction. These are called immunosuppressants.

Chronic GVHD starts more than 100 days after the transplant and you may have skin rashes, diarrhoea, sore joints and dry eyes. Your doctor is likely to suggest that you stay out of the sun because GVHD skin rashes can often get worse in the sun.

There is more detailed information about graft versus host disease.

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Bone marrow or stem cell transplants for ALL | Cancer ...

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After a baby is born and the umbilical cord is cut, ever wonder where that umbilical cord ends up?

Most of the time, it becomes waste but that cord still has some valuable resources that can save a life.

The blood that is found in it is called umbilical cord blood or cord blood for short.

It contains all the normal elements of blood, such as red and white blood cells. It is also jam packed with stem cells, similar to the ones found in bone marrow.

Birth is pretty exciting, its pretty dramatic. A lot of things are happening, says James E. Baumgartner, M.D., Pediatric Surgeon.

One of those things that people rarely hear about is the option to donate cord blood. Bone marrow and cord blood contain the same type of stem cells, but those from cord blood have more advantages. Since stem cells from cord blood are less mature than stem cells from an adult's bone marrow, a recipient's body is less likely to reject them.

Another benefit is that taking cord blood is less invasive than a bone marrow transplant. Once an umbilical cord is clamped, it is wiped with antiseptic and a needle is inserted into one of the veins to withdraw a few ounces of blood. The procedure takes just a few minutes and is painless.

We all collect prospective data to look for risk for, you know, lung damage, kidney damage, liver damage, heart damage. Were looking at the nervous system pretty carefully and we found nothing. So that we really believe that its safe, explains Baumgartner.

About 70% of patients who need a stem cell transplant dont have a matching donor in their own family, which leads to the main advantage of cord blood. Stem cells from cord blood dont need to be exactly matched to the patient like bone marrow transplants from adult donors. One drawback to cord blood though is that the number of stem cells available is relatively small. This means young children will benefit because they need less.

Families can either save cord blood for themselves or donate it to a bank.

You need to talk to your doctor at least three months before your due date to find out if you are eligible to donate cord blood.

CORD BLOOD TREATMENT SAVES LIVES REPORT #2401

BACKGROUND: A stem cell transplant is a treatment that is used to treat cancers that affect blood and immune system like leukemia, multiple myeloma, and some types of lymphoma. Stem cell transplants are used to treat these types of cancer since the stem cells that the body naturally produces most often die due to treatments like radiation and chemotherapy. Human beings need stem cells to survive, therefore, a stem cell transplant gives patients blood cells that they cant produce anymore. Furthermore, donated cells can often find and kill the cancerous cells better than the patients own cells. Stem cells include:

* Red blood cells (RBCs) * White Blood cells (WBCs) * Platelets (Source: http://www.cancer.net/navigating-cancer-care/how-cancer-treated/bone-marrowstem-cell-transplantation/what-stem-cell-transplant-bone-marrow-transplant & https://www.cancer.org/treatment/treatments-and-side-effects/treatment-types/stem-cell-transplant/why-stem-cell-transplants-are-used.html)

CORD BLOOD: In the past, the only location where stem cells could be taken for a transplant was in the bone marrow. In recent years cord blood, the blood that is found in the umbilical cord, has been used for stem cell transplants. They possess the same quantity of stem cells as the bone marrow, and they come with more advantages. To start off, no surgery is needed like with bone marrow. Since the umbilical cord is natural in every birth, the mother can choose to donate her cord around three months before she is due. Once the cord is clamped, it is cleaned with antiseptic. Later, a needle is inserted into one of the veins in order to gather the necessary blood. Furthermore, since the cord blood stem cells are less mature than those stem cells from an adults bone marrow, the recipients body is less likely to reject the transplant. This is very important for people with ethnic backgrounds. With bone marrows stem cells, the match between the donor and the recipient has to be 8/8; with cord blood cells, on the other hand, the match can be partial. For recipients that come from an ethnic background, a perfect match can be harder to find. (Source: http://www.nationalcordbloodprogram.org/qa/what_are_advantages.html)

PROS & CONS: Other advantages that come with core blood cells are the association of lower incidence of GvHD (Graft vs. Host Disease), and the lower risk of viral infections. Nevertheless, the cord blood cells have a drawback: the amount of stem cells found in them is very small. Because of the low number, children benefit from this transplant procedure more than adults. Since childrens bodies are smaller, they need fewer cells for their body to start reproducing them naturally. On the other hand, adults naturally need more cells than the ones the cord blood produces because of their size. (Source: http://www.nationalcordbloodprogram.org/qa/how_is_it_collected.html)

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Donating the umbilical cord could save someone's life - WNDU-TV

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The National Bone Marrow registry took place in the Bluestem Room last Friday. This donor drive was hosted by Love Your Melon and Cardinal Key, with Gail Chism and Mary Kelly acting as representatives from Be The Match as well. On average, one person in 430 is called to donate, but the likelihood of being called also depends on the race of the donor. In total, 57 donors were added to the registry by the end of the event.

Thadd Simpson

Thadd Simpson

The National Bone Marrow registry took place in the Bluestem Room last Friday. This donor drive was hosted by Love Your Melon and Cardinal Key, with Gail Chism and Mary Kelly acting as representatives from Be The Match as well. On average, one person in 430 is called to donate, but the likelihood of being called also depends on the race of the donor. In total, 57 donors were added to the registry by the end of the event.

March 29, 2017 Filed under News

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Fifty-seven students registered to give DNA at the first-ever Bone Marrow Drive at Wayne State College. The drive was in the Bluestem Room of the Kanter Student Center on Friday. That puts WSC at 279 students on the bone marrow registry when combined with MAZE. The drive was put on by Be The Match, a nonprofit organization that helps people diagnosed with diseases such as leukemia and lymphoma to get them the blood that could save their life. Be The Match is operated by the National Marrow Donor Program. We want to get Wayne State on the bone marrow registry, said student Kelsi Anderson said, who runs the Love Your Melon group on campus. A donor can give someone battling blood cancer a second chance. Its crucial for them to have a donor. Those who registered simply gave a cheek swab of their DNA, which will be analyzed to determine if it matches with someone who needs a bone marrow transplant. Its all about the DNA makeup, said Gail Chism of Be The Match. The DNA needs to be as close as possible. A donor could have closer DNA to the patient than a family member. If a match is made, the donor will be sent somewhere local for the bone marrow transplant. A courier will then take the bone marrow to the patient, who could be anywhere in the country. Eighty percent of the time it is like giving plasma, Chism said. Anderson said that in other cases a needle is injected into the pelvic bone todraw the marrow out. Blood cancers such as leukemia and lymphoma produce abnormal blood cells, other than the normal red blood cells, white blood cells and platelets. Blood cells develop from stem cells in bone marrow. A bone marrow transplant helps the patient produce more normal blood cells that help the body with functions such as fightingoff infections or preventing serious bleeding. Anderson said the drive was a shared idea between herself and Jaelyn Lewis, the leader of Cardinal Key. They hope it will become an annual event in the future. I really appreciate what Kelsi has done, Chism said. Shes really been on it. It takes great leadership to put this together. What we get out of here today is priceless.

Thadd Simpson WSC student Lily Roberts swabs her mouth in order to join the National Bone Marrow registry in the Bluestem Room last Friday.

Tags: 2015, Be The Match, Cardinal Key, Gail Chism, Kelsi Anderson, Love Your Melon, NE, Neb., Nebraska, Nebraska State College System, Spring 2015, Thadd Simpson, The Wayne Stater, Wayne, Wayne State College, Wayne State Wildcats, Wildcats, WSC, WSC Cats, WSC Wildcats, WSCs first-ever Bone Marrow Drive

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WSC's first-ever Bone Marrow Drive - The Wayne Stater

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During a check-up, on his 43rd birthday, his doctor named summertime flu the most likely culprit.

Then the same thing happened again, and it settled into a disturbing pattern: midweek chills and an escalating fever that would break on Sunday. By Monday, Chris would feel fine, only to have the sequence repeat itself.

He joked about it with colleagues at T-Mobile, where he works in software development, "Well, I hope it's not cancer!"

On alternating weekends from May to October, Chris would volunteer as a back country ranger for the US Forest Service -- a physically demanding role that involves patrolling Washington's Cascade Mountain forests and hiking along high-altitude trails with a backpack that can weigh up to 32 kilograms.

But now, even at sea level, he was getting winded just walking his two dogs around the block. What was going on?

A medical appointment revealed a heart murmur and suspicions of endocarditis, an infection of the heart's inner lining. The scare triggered another series of tests that led Chris and his husband, Bill Sechter, to Emergency Room 4 at the University of Washington Medical Center.

A whiteboard checklist documented his Saturday morning: insertion of a large-bore IV as a potential conduit for antibiotics, a round of blood draws, and discussions with the ER doctor.

Then the phone rang and the nurse answered, listened and responded to multiple questions in quick succession: "Yes. Yes. Oh, OK. OK. Yeah." He excused himself from the room and soon returned in a "full hazmat suit", as Chris describes it. Yellow.

"And that's when we were like, 'Oh s***, it's on. Something is seriously bad.'"

Chris learned that his level of infection-fighting neutrophil cells, normally churned out by the bone marrow, had fallen so low that his defenses were in tatters. He was also severely anemic, with roughly half the normal amount of red blood cells in his blood.

It wasn't endocarditis. And when one of his doctors performed a blood smear, she saw something on the microscope slide that shouldn't be there: blasts.

These leukemic cells, stuck in adolescence, were the harbingers of the coming horde that had so astonished 19th-century surgeons.

The doctor apologetically broke the news and Chris and his sister dissolved into tears. In an emotional Facebook post later that day, he attached a picture of himself in a hospital gown and pink face mask and wrote: "this avowed agnostic could actually go for your good juju / positive thoughts or even your (gasp) prayers."

More tests, including a bone marrow biopsy of his pelvic bone, painted an increasingly disturbing picture. He had acute myeloid leukaemia, a fast-progressing cancer.

The biopsy suggested that an astonishing 80 per cent of his bone marrow cells were cancerous. Strike one.

Other results suggested that chemotherapy wouldn't be as effective on his form of leukemia. Strike two.

And genetic tests put him in the unfavorable risk category by revealing that his cancer cells carried only one copy of chromosome 21, a rare anomaly associated with "dismal" outcomes, according to recent studies. Strike three.

Chris needed to start chemotherapy immediately.

But first, he had his sperm banked. Then, with family and a close friend at his side, he celebrated his impending treatment with prime rib and cheap champagne smuggled into his hospital room.

Over three days, he received multiple doses of the anticancer drugs cladribine, cytarabine and mitoxantrone, the last a dark blue concoction often dubbed "Blue Thunder." The drug turned his urine a shade he describes as "Seahawks green" in honor of Seattle's football team. Other patients have had the whites of their eyes temporarily turn blue.

On the third night of his drug infusion, a sudden back pain grew into an intense pressure in his chest that felt like he was being stabbed. A heart attack? An emergency CAT scan instead revealed two newly formed blood clots: one in his right leg and another in his right lung -- not uncommon consequences of chemotherapy.

Over the next six months, Chris would need transfusions of blood-clotting platelets whenever his level of them dipped too low, and daily injections of a blood-thinning drug whenever it rose too high.

Thirteen days after being admitted into the hospital, he posted a more hopeful Facebook entry: "And I'm finally going home! Now the real adventure begins."

Based on his leukemia classification, Chris was braced for multiple rounds of chemotherapy. He and his husband were overjoyed when a second bone marrow biopsy suggested that the leukemia had become undetectable after only a single round.

Because of his high-risk classification, however, Chris's doctors said that the cancer was likely to return without a bone marrow transplant.

But Chris discovered that he had inherited an extremely rare set of cell-identifying protein tags. Only one bone marrow donor on the worldwide registry matched his genetic tags, and that person was unable to donate.

An umbilical cord blood transplant, Chris and his doctors agreed, was his best hope.

Like bone marrow, cord blood is unusually rich in hematopoietic stem cells -- which can give rise to every type of blood cell -- and their more developed descendants, progenitor cells, which are more limited in what they can become. But, unlike bone marrow, cord blood can be collected in advance and stored for decades in liquid nitrogen.

First, Chris would need to spend another five days in the hospital for a standard follow-up round of chemotherapy to pick off any hidden cancer cells. Chris marked the occasion with a Facebook post of himself in a grey felt Viking helmet and attached braids. "Round 2... And FIGHT!" This time, the chemo went off without a hitch.

He was a familiar face at the medical center, though, with three additional hospitalizations: twice for bacteremia, a bacterial blood infection marked by high fevers, and once so doctors could tame an allergic reaction to a transfusion of platelets, which always reminded Chris of chicken broth.

He had to steel himself again on Christmas Eve for the arrival of the "big guns": two days of conditioning chemotherapy, headlined by a derivative of mustard gas. Its name is cyclophosphamide, and it works by sabotaging the machinery that copies DNA in rapidly dividing cells. As it does this, it breaks down to form toxic chemicals, including a pungent one called acrolein, which can destroy the lining of the bladder.

To neutralize its effects, patients must take another drug, called mesna, and drink plenty of water.

After a day of rest, Chris began a radiation therapy regimen so intense that it would have killed him if delivered in a single dose. Instead, his radiologists used a particle accelerator to fire X-rays at him in multiple bursts during morning and evening sessions over four days.

"You basically get into a tanning booth made out of clear Plexiglas," he said.

Wearing nothing but a paper gown, Chris had to stay completely still behind two metal shielding blocks, each the size of a brick, positioned to protect his lungs from irreversible radiation-induced scarring. He did get a mild tan, he says, along with damaged skin that still resembles crepe paper.

Another absurdity still makes him laugh: while he requested punk rock for one of the sessions, he was instead blasted with the tune of Prince's 'Erotic City'.

When he finished the final round of total body irradiation on 30 December, the radiology team gathered for a final tribute and let Chris hit a small ceremonial gong.

The morning of New Year's Eve, Chris wrote on Facebook, "I'm as nervous as an expectant father!" An hour and a half later, he marked the delivery of his "zero birthday" with a small chocolate cake and a decorative "0" candle: the day when his own bone marrow cells, erased by radiation and chemotherapy, were replaced by roughly four tablespoons of a life-granting elixir from the cord blood of two baby girls.

Even with some of the best help that medicine can offer, transplant recipients face a daunting few weeks without functional bone marrow when nearly anything can kill them.

Chris and Bill have nicknamed the donors Amelia and Olivia based on their blood types, A-negative and O-positive. In a later post, Chris marveled at the new arrivals reseeding his bone marrow: "I use more vanilla flavoring creamer in my coffee than the volume of cells that are rebuilding my entire blood and immune system."

Four hours after the initial infusions, he received his protective bridge of blood-forming stem cells, collected and expanded from the cord blood of a third baby, a boy he and Bill have nicknamed Eddie.

Less than three weeks after the transplant, Chris's neutrophils had fully engrafted and genetic tests suggested that Amelia had decisively won the fight to form his new blood and bone marrow. He progressed so rapidly, in fact, that he had to stay in the hospital for two days after he was fit to leave, so that Bill could finish preparing the apartment.

28 January: discharge day. As his family packed up his hospital room, Chris was taking a shower when a wall of exhaustion hit him. He could no longer stand or even dry himself off and sat dripping on the shower bench until Bill heard his calls for help.

He had survived, but life had fundamentally changed.

At home, every surface had to be disinfected daily with a bleach solution. At first, Chris couldn't walk 100 feet down the apartment hallway without leaning on his brother. Until he hit the 100-day milestone after his transplant, the end of the most vulnerable period for recipients, he returned to the Seattle Cancer Care Alliance every other day for blood tests and checkups.

On the 97th day, Chris and his family celebrated a hard-fought victory when he was officially declared cancer-free: a leukemia survivor.

Despite dozens of studies documenting its curative powers, cord blood is saved after only 5 per cent of all US births. The rest is simply thrown away.

Michael Boo, chief strategy officer for the National Marrow Donor Program, estimates that only one in ten of those retained units passes the required screening tests and has enough volume to merit long-term storage.

Cord blood is also notoriously expensive, ranging from $22,000 to $45,000 per unit. Due to the relatively low demand from doctors, Boo says, public banks -- at least in the US -- are collecting as much as they can afford to keep. Beyond persuading new parents to donate, then, lowering the cost of cord blood transplants may depend upon persuading more doctors to use the cells and more insurers to cover them.

One potential use has attracted the avid interest of the Biomedical Advanced Research and Development Authority, part of the US Department of Health and Human Services. As part of Project BioShield, the federal agency has been on the lookout for medical interventions that could treat acute radiation syndrome after a dirty bomb or nuclear disaster.

Cord blood transplants in adults, still an option of last resort in the early 2000s, nearly slammed to a halt over the quandary of how to keep patients alive until their new bone marrow cells could kick in.

Some researchers reasoned that they could boost the transplant volume by giving adults two cord blood units instead of one. John Wagner and colleagues at the University of Minnesota performed the first double transplant in 2000, using cells from two infant donors.

The tactic dramatically reduced the rate of graft failure, in which the recipient's body rejects the new cells. But it barely changed the time needed to regenerate the bone marrow, and some critics have questioned whether a double cord blood transplant offers any significant benefits.

Wagner says his research suggested that transplanting enough blood-forming cells was necessary but likely not sufficient for better results. Improved patient survival, in fact, seemed to depend more upon a revised roster of drugs given pre-transplant.

To their surprise, researchers also discovered that the donors in a double cord blood transplant seem to battle for dominance, a curious "graft-versus-graft" phenomenon that almost always results in the victor dominating the recipient's new bone marrow and blood cells.

Filippo Milano, associate director of the Cord Blood Program at the Fred Hutchinson Cancer Research Center in Seattle, compares it to a pivotal scene in the 1986 movie Highlander, when the antagonist exclaims, "There can be only one!"

On a sunny morning nearly a year after Chris's transplant, he and I meet the Italian-born doctor in his lab so he can greet one of his star patients and explain the science behind the therapy that saved Chris's life. Milano is passionate about coaching soccer and cooking. On the side, he jokes, he conducts research on cord blood transplants.

Upon his arrival to "The Hutch" in 2009, Milano teamed up with Colleen Delaney, founder and director of the Cord Blood Program, to test and refine a treatment strategy that may yet prove a better option than a bone marrow transplant for people with leukemia who are at high risk of relapsing.

Based on collaborations and discussions with other experts in the field, Delaney pioneered a method to minimize the risk of infection and bleeding after a cord blood transplant by reducing the time needed for the new blood cells to kick in. The strategy relies on what she and Milano call an "expanded" blood unit.

Starting with an extra batch of cord blood, they separate out the minuscule fraction of blood-forming stem cells and their early descendants and expand that population in the lab.

The hundreds of millions -- even billions -- of resulting stem and progenitor cells can jump start the generation of protective blood cells in the recipient. When infused along with a more traditional transplant, they can act like a temporary bridge until the replacement bone marrow takes over. "The net gain was that you didn't have those very prolonged periods of recovery," Wagner said.

One crucial component, Delaney discovered, is a protein called Notch ligand.

When added to the blood-forming stem cells, Notch ligand lets them divide quickly in the lab but temporarily pauses their development by preventing them from maturing into the normal range of cell types. Critically, they never give rise to T or B immune cells, which would seek out and destroy any perceived threats lacking the proper "self" ID tags.

Putting a donor's T cells into an unmatched recipient, Delaney says, would trigger fatal graft-versus-host disease. "That's the key: we get rid of all those bad parts of the immune system that need to be matched or they can kill you."

The "bridge of recovery" lasts only so long before the full contingents of other donor cells begin attacking and dismantling it. But, with no cells checking IDs initially, the early flood of blood-forming stem cells need not be matched to the recipient at all, meaning that the "expanded" cord blood unit could be created well ahead of time and used whenever needed as a universal donor.

Other researchers are working on strategies toward the same end, and Mary Laughlin describes the overall progress as "very exciting".

Delaney's work, she says, "is very important, saving lives and improving the tolerability of these transplants and the success of these transplants".

This is an edited extract from an article first published by Wellcome on Mosaic. It is republished here under a Creative Commons license.

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March 27, 2017 07:02 ET | Source: Cellect Biotechnology Ltd.

Cellects technology, ApoGraft, aims to become a game changerin stem cells transplantations for cancer treatments

Company gets green light from DSMB Board for enrolling additional 2 cancer patients for ApoGraft transplantation treatments

TEL AVIV, Israel, March 27, 2017 (GLOBE NEWSWIRE) -- Cellect Biotechnology Ltd. (Nasdaq:APOP) (TASE:APOP), a developer of stem cell selection technology, announced today that the first stem cell transplant procedure has been successfully performed using its ApoGraft technology in the Companys Phase I/II clinical trial in a blood cancer patient.

Up to 50 percent of stem cell transplant procedures, such as bone marrow transplants, result in life-threatening rejection disease, known as Graft-versus-Host-Disease (GvHD). Cellects ApoGraft technology is aiming to turn stem cell transplants into a simple, safe and cost effective process, reducing the associated severe side effects, such as rejection and many other risks.

Dr. Shai Yarkoni, Cellects CEO said, After 15 years of research, this is the first time we have used our technology on a cancer patient suffering from life-threatening conditions. It is a first good step on a road that we hope will lead to stem cell based regenerative medicine becoming a safe commodity treatment at every hospital in the world.

Based on the successful transplantation results, the independent Data and Safety Monitoring Board (DSMB) approved the enrollment of 2 additional patients for ApoGraft treatment to complete the first study cohort as planned.

About GvHD

Despite improved prophylactic regimens, acute GvHD disease still occurs in 25% to 50% of recipients of allogeneic stem cell transplantation. The incidence of GvHD in recipients of allogeneic stem cells transplantation is increasing due to the increased number of allogeneic transplantations survivors, older recipient age, use of alternative donor grafts and use of peripheral blood stem cells. GvHD accounts for 15% of deaths after allogeneic stem cell transplantation and is considered the leading cause of non-relapse mortality after allogeneic stem cell transplantation.

About ApoGraft01 study

The ApoGraft01 study (Clinicaltrails.gov identifier: NCT02828878), is an open label, staggered four-cohort, Phase I/II, safety and proof-of-concept study of ApoGraft process in the prevention of acute GvHD. The study, which will enroll 12 patients, aims to evaluate the safety, tolerability and efficacy of the ApoGraft process in patients suffering from hematological malignancies undergoing allogeneic stem cell transplantation from a matched related donor.

About Cellect Biotechnology Ltd.

Cellect Biotechnology is traded on both the NASDAQ and Tel Aviv Stock Exchange (NASDAQ:APOP)(NASDAQ:APOPW)(TASE:APOP). The Company has developed a breakthrough technology for the isolation of stem cells from any given tissue that aims to improve a variety of stem cell applications.

The Companys technology is expected to provide pharma companies, medical research centers and hospitals with the tools to rapidly isolate stem cells in quantity and quality that will allow stem cell related treatments and procedures. Cellects technology is applicable to a wide variety of stem cell related treatments in regenerative medicine and that current clinical trials are aimed at the cancer treatment of bone marrow transplantations.

Forward Looking Statements This press release contains forward-looking statements about the Companys expectations, beliefs and intentions. Forward-looking statements can be identified by the use of forward-looking words such as believe, expect, intend, plan, may, should, could, might, seek, target, will, project, forecast, continue or anticipate or their negatives or variations of these words or other comparable words or by the fact that these statements do not relate strictly to historical matters. For example, forward-looking statements are used in this press release when we discuss Cellects aim to make its ApoGraft technology a game changer in stem cell transplantations for cancer treatments and procedures, Cellects Apograft technology aiming to turn stem cell transplants into a simple, safe and cost effective process, reducing the associated severe side effects, such as rejection and many other risks, Cellects hope that stem cell based regenerative medicine will become a safe commodity treatment at every hospital in the world and that Cellects technology is expected to provide pharma companies, medical research centers and hospitals with the tools to rapidly isolate stem cells in quantity and quality that will allow stem cell related treatments and procedures. These forward-looking statements and their implications are based on the current expectations of the management of the Company only, and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. In addition, historical results or conclusions from procedures, scientific research and clinical studies do not guarantee that future results would suggest similar conclusions or that historical results referred to herein would be interpreted similarly in light of additional research or otherwise. The following factors, among others, could cause actual results to differ materially from those described in the forward-looking statements: changes in technology and market requirements; we may encounter delays or obstacles in launching and/or successfully completing our clinical trials; our products may not be approved by regulatory agencies, our technology may not be validated as we progress further and our methods may not be accepted by the scientific community; we may be unable to retain or attract key employees whose knowledge is essential to the development of our products; unforeseen scientific difficulties may develop with our process; our products may wind up being more expensive than we anticipate; results in the laboratory may not translate to equally good results in real clinical settings; results of preclinical studies may not correlate with the results of human clinical trials; our patents may not be sufficient; our products may harm recipients; changes in legislation; inability to timely develop and introduce new technologies, products and applications, which could cause the actual results or performance of the Company to differ materially from those contemplated in such forward-looking statements. Any forward-looking statement in this press release speaks only as of the date of this press release. The Company undertakes no obligation to publicly update or review any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws. More detailed information about the risks and uncertainties affecting the Company is contained under the heading Risk Factors in Cellect Biotechnology Ltd.'s Annual Report on Form 20-F for the fiscal year ended December 31, 2016 filed with the U.S. Securities and Exchange Commission, or SEC, which is available on the SEC's website, http://www.sec.gov and in the Companys period filings with the SEC and the Tel-Aviv Stock Exchange.

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Cellect Announces Successful First Cancer Patient Stem Cell Transplant - GlobeNewswire (press release)

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The newest skin-care secrets of 2017 are blazing trails, blowing minds and changing the face of skin care. Here are the eight most important takeaways from the coolest breakthroughs happening right now.

It may not be the sexiest of anti-aging ingredients, but dermatologists are realizing that cholesterol a component of the material holding our skin cells together is one of the most important ingredients to look for in a moisturizer (especially by age 40, when levels can plummet as much as 40 percent). Its most effective when combined with fatty acids and ceramides, which also help hold skin cells firmly in place so your skin looks smoother and more radiant. Find the trio in __ Elizabeth Arden Advanced Ceramide Capsules Daily Youth Restoring Serum . But no one is suggesting you need to start eating butter by the stick: Theres no evidence that ingesting more cholesterol will do anything for the skin, says Jordana Herschthal, a dermatologist in Boca Raton, Florida.

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Were not throwing shade at walking or lifting weights they beat sitting on the couch but if you want smoother, fresher-looking skin, you need to get your heart rate up. In one study, people ages 20 to 86 who exercised at a high intensity (running, cycling, whatever youre into) for four or more hours a week for at least ten years had thinner stratum corneum layers and more energetic mitochondrial cells than participants who didnt regularly work out. In plain English: Their skin appeared and acted younger. And its never too late to reverse course. Even previously sedentary 65- to 86-year-olds who began moderate aerobic exercise for 45 minutes twice a week had a change in their skin signs of aging began to reverse on a molecular level after just three months.

We used to think UV rays were the main culprit for melasma, but data is also indicating that visible light and heat may cause dark patches, too, says Doris Day , a clinical associate professor of dermatology at NYU Langone Medical Center in New York City. This means that Bikram or standing over a hot stove can create inflammation and exacerbate hyperpigmentation . The risk for melasma is increased for anyone with a job where theyre exposed to constant heat, like bakers, says Herschthal. In addition to using sunscreen, she recommends incorporating a product that regulates skin temperature into your routine to minimize the damage; she likes Colorescience Even Up Clinical Pigment Perfector SPF 50 , which has a marine extract called venuceane. Its one of the few ingredients that keeps skin cool for an extended period of time, says Herschthal. (Studies suggest that applying it twice daily regulates skin temperature.) We also like running the metal ReFa S Carat roller over skin for a temporary cooling effect.

From left: Elizabeth Arden Advanced Ceramide Capsules Daily Youth Restoring Serum, SkinCeuticals Triple Lipid Restore 2:4:2, ReFa S Carat roller, Olay Eyes Ultimate Eye Cream, and Colorescience Even Up Clinical Pigment Perfector SPF 50. Photo by Josephine Schiele.

Theres no rule that says you have to use one product on your entire face. In fact, the new thinking is that you shouldnt. I have patients who use retinoids only on their cheeks and forehead and prescription Soolantra on their chin, where theyre prone to rosacea, says Dendy Engelman, a dermatologist in New York City. Another combo she recommends: Thick creams around the delicate eye area and on the lips, and salicylic acid or glycolic acid products on the T-zone to minimize breakouts (try Paulas Choice Skin Perfecting 2% BHA Liquid Exfoliant ). Use anti- redness products only across the bridge of the nose, on the cheeks, and on the chin to soothe areas prone to flushing. (Engelman likes the Eau Thermale Avene Antirougeurs and Aveeno Ultra-Calming lines.)

When the bones in your face shrink with age yes, it happens it contributes to sagging and a generally flat appearance, says Ava Shamban, a dermatologist in Beverly Hills. Dermatologists have long turned to hyaluronic acid fillers to make the face look fuller, but inject too much of them in the wrong places and suddenly its hello, weird blowfish face. Now dermatologists are learning that injecting hyaluronic acid deeper onto the top layer of bone, instead of in wrinkles seems to reverse some of the bone shrinkage. For chins, if you inject on the top layer of bone, youre stimulating stem cells and actually getting chin augmentation over time, says Shamban, adding that the technique appears to work for the cheekbones and jawbone, as well.

Its an even better anti-ager than we thought. A study published in Dermatologic Surgery (and sponsored by the Johnson & Johnson Skin Research Center) found that when people applied a moisturizer with SPF 30 daily for a year without any other anti-aging products (in other words: zero, nada, nothing else), they ended up with clinically measured improvement in mottled skin tone (by 52 percent), texture (by 40 percent), and clarity (by 41 percent). And the self-reported results were even stronger, which means those numbers translated to younger- looking skin. Its some of the best evidence yet that sunscreen doesnt just prevent aging; it may actually reverse it.

A version of this article on the newest skin-care secrets of 2017 originally appeared in the April 2017 issue of Allure. To get your copy, head to newsstands or subscribe now.

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The 8 Newest Skin-Care Secrets of 2017 - Allure Magazine

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March 29, 2017 (A) Bio-imaging analysis to evaluate the therapeutic effect of iPS-ML producing IFN- on metastatic liver cancer. (B) Quantification of the image data shown in A. (C) Histological data indicating migration of iPS-ML (PKH26, red) into intrahepatic tumor tissues (GFP, green). Adapted from M. Sakisaka, M. Haruta, Y. Komohara, S. Umemoto, K. Matsumura, T. Ikeda, M. Takeya, Y. Inomata, Y. Nishimura, and S. Senju, "Therapy of primary and metastatic liver cancer by human iPS cell-derived myeloid cells producing interferon-," Journal of Hepato-Biliary-Pancreatic Sciences, vol. 24, pp. 109-119, Feb. 2017. DOI: 10.1002/jhbp.422

Causes of the most common form of liver cancer, hepatocellular carcinoma (HCC), include hepatitis B or C, cirrhosis, obesity, diabetes, a buildup of iron in the liver, or a family of toxins called aflatoxins produced by fungi on some types of food. Typical treatments for HCC include radiation, chemotherapy, cryo- or radiofrequency ablation, resection, and liver transplant. Unfortunately, the mortality rate is still quite high; the American Cancer Society estimates the five-year survival rate for localized liver cancer is 31 percent.

Hoping to improve primary liver cancer outcomes, including HCC and metastatic liver cancer, researchers from Japan began studying induced pluripotent stem (iPS) cell-derived immune cells that produce the protein interferon- (IFN-). IFN- has antiviral effects related to immune response, and exhibits two antitumor activities, the JAK-STAT signaling pathway and p53 protein expression. IFN- has been used for some forms of cancer, but problems like rapid inactivation, poor tissue penetration, and toxicity prevent widespread use. To overcome that hurdle, Kumamoto University researchers used iPS cell-derived proliferating myelomonocytic (iPS-ML) cells, which they developed in a previous research project. These cells were found to mimic the behavior of tumor-associated macrophages (TAMS), which inspired the researchers to develop them as a drug delivery system for IFN- and evaluate the therapeutic effect on liver cancer in a murine model in vivo.

The researchers selected two cancer cell lines that were sensitive to IFN- treatmentone that easily metastasized to the liver after injection into the spleen, and another that produced a viable model after being directly injected into the liver. After injection, mice that tested positive for cancer (~80 percent) were separated into test and control groups. iPS-ML/IFN- cells were injected two to three times a week for three weeks into the abdomens of the test group subjects.

Livers with tumors were found to have higher levels of IFN- than those without. This was likely due to iPS-ML/IFN- cells penetrating the fibrous connective tissue capsule surrounding the liver and migrating toward intrahepatic cancer sites. The iPS-ML/IFN- cells did not penetrate non-tumorous livers, but rather stayed on the surface of the organ. Furthermore, concentrations of IFN- from 24 to 72 hours after iPS-ML/IFN- injections were found to be high enough to inhibit proliferation or even cause the death of the tumor cells.

Due to differences between species, mouse cells are not adversely affected by human IFN-, meaning that side effects of this treatment are not visible in this model. Thus, the researchers are working on a new model with the mouse equivalent of human iPS-ML/IFN, and testing its therapeutic abilities.

"Our recent research into iPS-cell derived, IFN- expressing myeloid cells should be beneficial for many cancer patients," says research leader Dr. Satoru Senju. "If it is determined to be safe for human use, this technology has the potential to slow cancer progression and increase survival rates. At this point, however, we still have much work ahead."

This research may be found in the Journal of Hepato-Biliary-Pancreatic Sciences.

Explore further: Scientists stimulate immune system, stop cancer growth

More information: Masataka Sakisaka et al, Therapy of primary and metastatic liver cancer by human iPS cell-derived myeloid cells producing interferon-, Journal of Hepato-Biliary-Pancreatic Sciences (2017). DOI: 10.1002/jhbp.422

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Immune cell therapy on liver cancer using interferon beta produced with stem cells - Medical Xpress

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Skin stem cells gain traction for skin repair and regeneration ... FinancialsTrend Although a tremendous progress has been made, large full-thickness skin defects are still associated with mortality due to a low availability of donor skin areas. and more »

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Skin stem cells gain traction for skin repair and regeneration ... - FinancialsTrend

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On March 28, a Japanese man in his 60s became the first person to receive cells derived from induced pluripotent stem (iPS) cells that had been donated by another person.

The surgery is expected to set the path for more applications of iPS cell technology, which offers the versatility of embryonic stem cells without the latters ethical taint. Banks of iPS cells from diverse donors could make stem cell transplants more convenient to perform, while slashing costs.

iPS cells are created by removing mature cells from an individual (from their skin, for example), reprogramming these cells backto an embryonic state, and then coaxing them to become a cell type useful for treating a disease.

In the recent procedure, performed on a man from Hyogo prefecture, skin cells from an anonymous donor were reprogrammed and then turned into a type of retinal cell that was transplanted onto the retina of thepatient who suffers from age-related macular degeneration. Doctors hope the cells will stop progression of the disease, which can lead to blindness.

In a procedure performed in September 2014at the Kobe City Medical Center General Hospital, a Japanese woman received retinal cells derived from iPS cells. They were taken from her own skin, though, and then reprogrammed. Such cells prepared for a second patient were found to contain genetic abnormalities and never implanted.

The team decided to redesign the study based on new regulations, and no other participants were recruited to the clinical study. In February 2017, the team reported that the one patient had fared well. The introduced cells remained intactand vision had not declined as would usually be expected with macular degeneration.

In todays procedure performed at the same hospital and by the same surgeon Yasuo Kurimoto doctors used iPS cells that had been taken from a donors skin cells, reprogrammed and banked. Japans health ministry approved the study, which plansto enroll 5 patients, on 1 February.

Using a donor's iPS cells does not offer an exact genetic match, raising the prospect of immune rejection. But Shinya Yamanaka, the Nobel Prize-winning stem-cell scientist who pioneered iPS cells, has contended that banked cells should be a close enough match for most applications.

Yamanaka is establishing an iPS cell bank, which depends on matching donors to recipients via three genes that code for human leukocyte antigens (HLAs) proteins on the cell surface that are involved in triggering immune reactions. HisiPS Cell Stock for Regenerative Medicine currently has cell lines from just one donor. But by March 2018, they hope to create 5-10 HLA-characterized iPS cell lines, which should match 30%-50% of Japans population.

Use of these ready-made cells has advantages for offering stem cell transplants across an entire population, says Masayo Takahashi, an ophthalmologist at the RIKEN Center for Developmental Biology who devised the iPS cell protocol deployed in todays transplant. The cells are available immediately versus several months wait for a patients own cells and are much cheaper. Cells from patients, who tend to be elderly, might have also accumulated genetic defects that could increase the risk of the procedure.

At a press conference after the procedure, Takahashi said the surgery had gone well but that success could not be declaredwithout monitoring the fate of the introduced cells. She plans to make no further announcements about patient progress until all five procedures are finished. We are at the beginning, she says.

This article is reproduced with permission and wasfirst publishedon March 28, 2017.

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Japanese Man Is First to Receive 'Reprogrammed' Stem Cells from Another Person - Scientific American

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All causes of the most common form of liver cancer, hepatocellular carcinoma (HCC), are not yet known, but the risk of getting it is increased by hepatitis B or C, cirrhosis, obesity, diabetes, a buildup of iron in the liver, or a family of toxins called aflatoxins produced by fungi on some types of food. Typical treatments for HCC include radiation, chemotherapy, cryo- or radiofrequency ablation, resection, and liver transplant. Unfortunately, the mortality rate is still quite high, with the American Cancer Society giving a 5-year survival rate for localized liver cancer at 31%.

Hoping to improve primary liver cancer including HCC and metastatic liver cancer therapies, researchers from Japan began studying induced pluripotent stem (iPS) cell-derived immune cells that produced the protein interferon-? (IFN-). IFN- exhibits antiviral effects related to immune response, and two different antitumor activities, the JAK-STAT signaling pathway and p53 protein expression. IFN- has been used for some forms of cancer but problems like rapid inactivation, poor tissue penetration, and toxicity have kept it from being used extensively. To get over that hurdle, Kumamoto University researchers used iPS cell-derived proliferating myelomonocytic (iPS-ML) cells, which they developed in a previous research project. These cells were found to mimic the behavior of tumor associated macrophages (TAMS), which inspired the researchers to develop them as a drug delivery system for IFN- and evaluate the therapeutic effect on liver cancer in a murine model in vivo.

The researchers selected two cancer cell lines that were sensitive to IFN- treatment, one that easily metastasized to the liver after injection into the spleen and the other that produced a viable model after being directly injected into the liver. After injection, mice that tested positive for cancer (~80%) were separated into test and control groups. iPS-ML/IFN- cells were injected two to three times a week for three weeks into the abdomen of the test groups.

Livers with tumors were found to have higher levels of IFN- than those without. This was likely due to iPS-ML/IFN- cells penetrating the fibrous connective tissue capsule surrounding the liver ?serous membrane?and migrating toward intrahepatic cancer sites. The iPS-ML/IFN- cells did not penetrate non-tumorous livers, but rather stayed on the surface of the organ. Furthermore, concentrations of IFN- from 24 to 72 hours after iPS-ML/IFN- injections were found to be high enough to inhibit proliferation or even cause the death of the tumor cells.

Due to differences between species, mouse cells are not adversely affected by human IFN-, meaning that side effects of this treatment are not visible in this model. Fortunately, the researchers are working on a new model with the mouse equivalent of human iPS-ML/IFN, and testing its therapeutic abilities.

"Our recent research into iPS-cell derived, IFN- expressing myeloid cells should be beneficial for many cancer patients," says research leader Dr. Satoru Senju. "If it is determined to be safe for human use, this technology has the potential to slow cancer progression and increase survival rates. At this point, however, we still have much work ahead."

This research may be found in the Journal of Hepato-Biliary-Pancreatic Sciences online.

Story Source:

Materials provided by Kumamoto University. Note: Content may be edited for style and length.

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Interferon-beta producing stem cell-derived immune cell therapy on liver cancer - Science Daily

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Treating human diseases by utilizing gene therapy strategies have taken the scientific world by storm. Imporved delivery tools (i.e. AAVs) and novel therapeutic strategies are prooving that gene therapy has the promise of successfully threating a wide spectrum of diseases that were once uncurable. Read more about what is gene therapy, what are the tools and current strategies scientists use to advance the field and cure disease.

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The Faculty of the Horae Gene Therapy Center is dedicated to develop therapeutic approaches for rare inherited diseases for which there is no cure utilizing state of the art technologies to correct the genetic mutations.Our focus is on AAT Deficiency,Amyotrophic Lateral Sclerosis (Lou Genrig's Disease),Canavan Disease,Cystic Fibrosis,Tay-Sachs&Sandhoff diseases,Retinitis Pigmentosa,Huntington's disease,HypercholesterimiaandCardiac arrhythmia.

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Under the umbrella of the ATC, UMass Medical School formed the Gene Therapy Center (GTC) emphasizing the promise that lies within the application of the recombinant adeno-associated virus;RNA Therapeutics Institute (RTI)featuring novel strategies for using the RNAi mechanism to silence the action of individual genes and The Center for Stem Cell Biology and Regenerative Medicine seeking to unlock the enormous promise to elucidate disease mechanisms inherent in humans.

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The Horae Gene Therapy Center is always interested in possible partnerships with both indivituals and organizations.Some possible partnerships includeknowledge exchange,consulting,collaborations with academia and/or industry,reagent exchange,sponsored research, andspinout activities.We encourage you to contact us and learn more how we can partner together to advance the area of gene therapy.

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Horae Gene Therapy Center UMass Medical School

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FINDINGS

UCLA researchers have developed a stem cell gene therapy cure for babies born with adenosine deaminase-deficient severe combined immunodeficiency, a rare and life-threatening condition that can be fatal within the first year of life if left untreated.

In a phase 2 clinical trial led by Dr. Donald Kohn of theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Researchat UCLA, all nine babies were cured. A 10th trial participant was a teenager at the time of treatment and showed no signs of immune system recovery. Kohns treatment method, a stem cell gene therapy that safely restores immune systems in babies with the immunodeficiency using the childs own cells, has cured 30 out of 30 babies during the course of several clinical trials.

Adenosine deaminase-deficient severe combined immunodeficiency, also known as ADA-SCID or bubble baby disease, is caused by a genetic mutation that results in the lack of the adenosine deaminase enzyme, which is an important component of the immune system. Without the enzyme, immune cells are not able to fight infections. Children with the disease must remain isolated in clean and germ-free environments to avoid exposure to viruses and bacteria; even a minor cold could prove fatal.

Currently, there are two commonly used treatment options for children with ADA-SCID. They can be injected twice a week with the adenosine deaminase enzyme a lifelong process that is very expensive and often does not return the immune system to optimal levels. Some children can receive a bone marrow transplant from a matched donor, such as a sibling, but bone marrow matches are rare and can result in the recipients body rejecting the transplanted cells.

The researchers used a strategy that corrects the ADA-SCID mutation by genetically modifying each patients own blood-forming stem cells, which can create all blood cell types. In the trial, blood stem cells removed from each childs bone marrow were corrected in the lab through insertion of the gene responsible for making the adenosine deaminase enzyme. Each child then received a transplant of their own corrected blood stem cells.

The clinical trial ran from 2009 to 2012 and treated 10 children with ADA-SCID and no available matched bone marrow donor. Three children were treated at the National Institutes of Health and seven were treated at UCLA. No children in the trial experienced complications from the treatment. Nine out of ten were babies and they all now have good immune system function and no longer need to be isolated. They are able to live normal lives, play outside, go to school, receive immunizations and, most importantly, heal from common sicknesses such as the cold or an ear infection. The teenager, who was not cured, continues to receive enzyme therapy.

The fact that the nine babies were cured and the teenager was not indicates that the gene therapy for ADA-SCID works best in the youngest patients, before their bodies lose the ability to restore the immune system.

The next step is to seek approval from the Food and Drug Administration for the gene therapy in the hopes that all children with ADA-SCID will be able to benefit from the treatment. Kohn and colleagues have also adapted the stem cell gene therapy approach to treat sickle cell disease and X-linked chronic granulomatous disease, an immunodeficiency disorder commonly referred to as X-linked CGD. Clinical trials providing stem cell gene therapy treatments for both diseases are currently ongoing.

Kohn is a professor of pediatrics and microbiology, immunology and molecular genetics at the David Geffen School of Medicine at UCLA and member of the UCLAChildrens Discovery and Innovation Institute at Mattel Childrens Hospital. The first author of the study is Kit Shaw, director of gene therapy clinical trials at UCLA.

The research was published in the Journal of Clinical Investigation.

The research was funded by grants from the U.S. Food and Drug Administrations Orphan Products Clinical Trials Grants Program (RO1 FD003005), the National Heart, Lung and Blood Institute(PO1 HL73104 and Z01 HG000122), the California Institute for Regenerative Medicine (CL1-00505-1.2 and FA1-00613-1), the UCLA Clinical and Translational Science Institute (UL1RR033176 and UL1TR000124) and the UCLA Broad Stem Cell Research Center.

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Pioneering stem cell gene therapy cures infants with bubble baby disease - UCLA Newsroom

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STAMFORD, Conn. Stamford-based Alliance for Cancer Gene Therapy (ACGT), the nations only nonprofit dedicated exclusively to cell and gene therapies for cancer, will celebrate its anniversary with a gGala on Wednesday, April 19, 2017, at 6:30 p.m. at The Harvard Club of New York City.

Dr. John Lahey, president of Quinnipiac University, will be honored at the event with the first ever Edward Netter Award for Business and Industry for his contributions to ACGT and the community. Gala speakers also include: ACGT research fellow Dr. Robert Vonderheide of the University of Pennsylvania, who will speak on the exciting breakthroughs using immunotherapy for the treatment of solid cancers; and Doug Olson, one of the first three patients treated in the groundbreaking cancer immunotherapy CAR-T clinical trial developed by ACGT research fellow and Scientific Advisory Council member, Dr. Carl June.

Tickets for the ACGT Gala are $750 and are available at acgtfoundation.org/events/ or by emailing Barbara Gallagher, ACGT national director of philanthropy at bgallagher@acgtfoundation.org.

Barbara Netter of Greenwich, ACGTs honorary chairman of the Board and co-founder, will present the first-ever Edward Netter Award for Business and Industry to Dr. Lahey, an ACGT Board member since 2004. Dr. Lahey embodies the qualities prized by ACGT co-founder Edward Netter: intellect, creativity, tenacity, curiosity and compassion.

He is the eighth President of Quinnipiac University, a private university located in Hamden. Upon his arrival at Quinnipiac in March of 1987, Dr. Lahey initiated a strategic planning process that has resulted in the growth of student enrollment from 2,000 to nearly 10,000 students. He also expanded Quinnipiac from a college to a university, which now offers more than 100 programs in its nine schools and colleges: Arts and Sciences, Business, Communications, Education, Engineering, Health Sciences, Law, Medicine and Nursing.

ACGT research fellow, Dr. Robert Vonderheide, will speak about his current work in immunotherapy that is targeting pancreatic cancer and other solid tumors at the Abramson Cancer Institute at the University of Pennsylvania. Dr. Vonderheides translational work tests novel approaches such as vaccines, antibodies, and adoptive T cells for the treatment of patients with melanoma, pancreatic cancer and other cancers. ACGT funded Dr. Vonderheides early research work in immunotherapy.

Doug Olson of Tinicum Township, Pa. will share the details of his experience as patient #2 in the first CAR-T 19 immunotherapy trial in September 2010 at the University of Pennsylvania. The treatment for the trial was developed by ACGT research fellow, Dr. Carl June. Doug was diagnosed with chronic lymphocytic leukemia (CLL) in July 1996 and has remained in remission since completing the trial.

This Gala offers the opportunity to celebrate the successes of ACGT research fellows and honor those who make it all possible, said John Walter, CEO and president of ACGT. With our donors support, ACGT has been able to be a part of finding and funding some of the most exciting cancer treatment breakthroughs, several of which we anticipate coming to market this year.

ACGt was founded in 2002 by Greenwich residents Barbara and Edward (1933-2011) Netter. As a national non-profit, ACGT has provided nearly $27 million in funding for cancer cell, gene and immunotherapy research in North America.

Photo: The Alliance for Cancer Gene Therapy (ACGT), gala event committee is (left to right) Sharon Phillips, Margaret Cianci, ACGT executive director, Barbara Gallagher, Jacquie Walter, Barbara Netter, ACGT co-founder and event chair, John Walter, ACGT CEO and president, Martha Zoubek, Jenifer Howard and Tracy Holton. Not pictured is Sabrina Raquet.

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Stamford-Based Cell And Gene Therapy Nonprofit Plans New York Gala - Stamford Daily Voice

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From left: Richard Allsopp, David Watumull and Bradley Willcox

Promising anti-aging results have been shown by a study of the compound Astaxanthin by researchers at the University of Hawaii John A. Burns School of Medicine in partnership with the life sciences company Cardax Inc.

The Astaxanthin compound CDX-085, developed by Cardax, showed the ability to significantly increase the expression of the FOXO3 gene, which plays a proven role in longevity.

All of us have the FOXO3 gene, which protects against aging in humans, said Bradley Willcox, professor and director of research at the Department of Geriatric Medicine, JABSOM, and principal investigator of the National Institutes of Health-funded Kuakini Hawaii Lifespan and Healthspan Studies. But about one in three persons carry a version of the FOXO3 gene that is associated with longevity. By activating the FOXO3 gene common in all humans, we can make it act like the longevity version. Through this research, we have shown that Astaxanthin activates the FOXO3 gene, said Willcox.

Richard Allsopp in his lab

This preliminary study was the first of its kind to test the potential of Astaxanthin to activate the FOXO3 gene in mammals, said Richard Allsopp, associate professor, and researcher with the JABSOM Institute of Biogenesis Research.

In the study, mice were fed either normal food or food containing a low or high dose of the Astaxanthin compound CDX-085 provided by Cardax. The animals that were fed the higher amount of the Astaxanthin compound experienced a significant increase in the activation of the FOXO3 gene in their heart tissue.

We found a nearly 90 percent increase in the activation of the FOXO3 Longevity Gene in the mice fed the higher dose of the Astaxanthin compound CDX-085, said Allsopp.

This groundbreaking University of Hawaii research further supports the critical role of Astaxanthin in health and why the healthcare community is embracing its use, said David G. Watumull, Cardax CEO. We look forward to further confirmation in human clinical trials of Astaxanthins role in aging.

We are extremely proud of our collaborative efforts with Cardax on this very promising research that may help mitigate the effects of aging in humans, said UH Vice President of Research Vassilis L. Syrmos. This is a great example of what the Hawaii Innovation Initiative is all aboutwhen the private sector and government join forces to build a thriving innovation, research, education and job training enterprise to help diversify the states economy.

Read more about the research at the JABSOM website.

See more images from the JABSOM-Cardax press conference.

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Promising anti-aging gene therapy developed through innovative ... - UH System Current News

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