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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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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March 28, 2017 by Layne Cameron Kristin Parent mapped the structure of the giant Samba virus with MSUs cryo-EM microscope, which is featured on the cover of the journal Viruses. Credit: Michigan State University

In a laboratory at Michigan State University, scientists took a DIY approach to build a retrofitted cryo-electron microscope that allowed them to map a giant Samba virus one of the worlds largest viruses.

If the common cold virus is scaled to the size of a ladder, then the giant Samba virus is bigger than the Washington Monument, said Kristin Parent, assistant professor of biochemistry and molecular biology and co-author of the paper featured on the cover of the journal Viruses. Cryo-EM allowed us to map this virus structure and observe the proteins it uses to enter, or attack, cells.

It seems counterintuitive that bigger organisms are harder to see, but they are when using cryo-electron microscopy. Thats because these microscopes usually are used to look at thin specimens and cant decipher larger organisms to reveal their biological mechanisms. For thick samples, scientists see only dark gray or black blobs instead of seeing the molecular framework.

Cryo-EM allowed Parents team to image the giant Samba virus and understand the structures that allow it to enter an amoeba. Once inside, Samba opens one of its capsid layers and releases its nucleocapsid which carries the genetic cargo that sparks an infection. While Samba isnt known to cause any diseases in humans, its cousin, the mimivirus, may be a culprit for causing some respiratory ailments in humans.

If you scoop up a handful of water from Lake Michigan, you are literally holding more viruses than there are people on the planet, said Parent, who published the paper with Jason Schrad and Eric Young, MSU biochemistry and molecular biology graduate students. While scientists cant study every virus on Earth, the insights we glean from viruses like the giant Samba can help us understand the mechanisms of other viruses in its family, how they thrive and how we can attack them.

As bacteria become more resistant to antibiotics, looking for new ways to fight diseases will continue to grow in importance. Parents lab also studies how bacteria-infecting viruses enter cells using this method, which could potentially lead to new antibacterial treatments. Yet the worlds best cryo-EM microscope costs more than $5 million. Limited by funds but not drive, Parent was able to upgrade an existing microscope at MSU to do cryo-EM one that is a tinkerers dream.

This traditional transmission electron microscope was retrofitted with a cryostage, which keeps viruses frozen in liquid nitrogen while theyre being studied. Parent and her team then added a Direct Electron DE-20 detector, a powerful camera the mighty microscopes piece de resistance.

Parent didnt invent cryo-EM, but establishing it on campus serves as a viable proof-of-concept for MSU, opening the door for many interdisciplinary partnerships. This cutting-edge microscopy has applications across many fields, from those addressing a single protein to others studying entire cells. Virtually anyone studying complex molecular machines can advance their work with this tool, Parent added.

Parent has earned an AAAS Marion Milligan Mason Award for Women in the Chemical Sciences. This award, her paper in Viruses and being the co-author who performed cryo-EM work in a recent Nature Communications paper, lays the groundwork to some day have a more advanced cryo-EM microscope housed at MSU to be able to perform high-resolution structural studies.

Weve done quite a bit with our limited resources, but were primed to do more, Parent said. I think MSU could serve as a cryo-EM center and to increase the prevalence of this technology in the Midwest and beyond.

As one example, scientists from Universidade Federal de Minas Gerais (Brazil) and Universidade Federal do Rio de Janeiro (Brazil) also contributed to this study and benefitted from the technology MSU has to offer.

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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]

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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.

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But its always been clear that they could be risky too, especially if theyre not used carefully. The LCSB team has published its results in the scientific journal PLOS Biology.

This study shows that for the first time, targeting the proliferating tumor mass and dormant cancer stem cells with combination therapy effectively inhibited tumor growth and prevented metastasis compared to monotherapy in mice, said Wang, who is a member of the UCLA Jonsson Comprehensive Cancer Center and of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. However, as of April 2016, new rules on human cells and tissue require FDA oversight and approval for such procedures.

Although the women had moderate vision loss prior to the stem cell treatments, a year later their vision ranged from total blindness to 20/200, which is considered legally blind.

NPR contacted the FDA, and was told by a spokeswoman that the agency is now finalizing a series of new guidelines regulating how clinics could use stem cells for treatment purposes. So far, however, scientists only partially understand how the body controls the fate of these all-rounders, and what factors decide whether a stem cell will differentiate, for example, into a blood, liver or nerve cell. He wrote an editorial accompanying the two papers.

As reported Wednesday in the New England Journal of Medicine, one of the women, a 72-year-old, went completely blind after doctors injected stem cells into her eye in an attempt to cure the disease.

But within a week of starting the off-the-charts dangerous therapy at an American clinic, the patients suffered complications.

Two of the patients sought treatment at the universitys hospital for the complications they suffered. The agency also noted that it had previously issued a warning to patients. She said that they were treating patients with their own stem cells.

In addition to charging a fee for treatment, there were several other red flags in the Florida cases that consumers should watch for when considering participation in a clinical trial, Goldberg said. They sought treatment at a Florida clinic that had announced a study to treat the condition on clinicaltrials.gov, a federal database of research studies.

Within days of the stem cell injections she was almost blind and ultimately progressed to complete blindness. Their attorney, Andrew Yaffa of Coral Gables, said that the case was resolved to the mutual satisfaction of the parties but that neither he nor his clients could comment beyond that.

She acknowledged, however, that the clinic had been performing the stem cell procedures.

Shoddy preparation of the stem cells may have led to some of the complications, said the study authors. We feel very confident about the procedures that we do, and weve had great success in many different indications. We believe that regenerative medicine / cellular therapeutics will play a large role in positively changing the natural history of diseases ultimately, we contend, lessening patient burdens, as well as reducing the associated economic impact disease imposes upon modern society.

The body produces a variety of stem cells. It is also costly, at almost $900,000 to develop and test the iPS cells for the first trial, Takahashi adds.

Whatever happened, experts said there was no evidence to suggest the procedure would have helped restore vision, since so little study has been done on whether adipose-derived stem cells can mature into the kinds of retinal cells that are involved in macular degeneration.

This represents a landmark, says Daley. But it proved too slow and expensive, says Shinya Yamanaka of Kyoto University in Japan, who first discovered how to create iPS cells and is a co-author of the NEJM paper. The registry may be useful as a starting point, but patients should then discuss potential trials with qualified physicians, an academic medical center.

A second patient was supposed to be treated, but transplantation was called off after the cells were found to have potential genetic problems. The cells were extracted their from fat, mixed with blood plasma and injected into their eyes.

Even though the safety and effectiveness of this procedure is unknown, all three patients received injections in both eyes. Dr. Thomas Albini of the University of Miami examined the women after they were treated at a clinic in Florida.

Before the procedure, all three women still had at least some vision. Medical experts said the episode raises questions about whether the government and doctors are doing enough to protect patients from the dangers of unapproved therapies.

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Three women blinded after clinical trial went wrong - Normangee Star

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The Food and Drug Administration recommends that people who consider getting a stem cell treatment need to make sure it has been approved or is being studied in a clinical trial that federal health regulators have allowed. The kind that have generated the most excitement - and controversy - are human embryonic stem cells, which are derived from early human embryos and can be coaxed to become any kind of cell in the body. After 20 years of research, Italian scientists recently received European regulatory approval for a stem cell-based treatment for a type of blindness that results from damage to the cornea, the surface of the eye. The fat tissue was then processed with enzymes with the goal of obtaining stem cells.

However, they immediately suffered complications, including retinal detachment and hemorrhage, which caused total loss of eyesight. "It's just not the case".

None of the women are named in the study.

In 2013, the company listed a trial on ClinicalTrials.gov titled, "Study to Assess the Safety and Effects of Cells Injected Intravitreal in Dry Macular Degeneration", according to the ClinicalTrials.gov site. Both lawsuits were settled, with the women receiving payments.

He warned that patients could be misled by "rogue clinics" which failed to distinguish between the many different types of stem cell. Those efforts have stalled, however. She stated that this was not a drug it was a simple procedure. Albini says the complications could have come from injecting a contaminant into the eye, or from the fact that the stem cells may have turned into myofibroblasts after the injections, which are cells associated with scarring.

The cases show that patients need to be warned that something that "sounds too good to be true may indeed be too good to be true and may even be disgusting", Albini says.

The company appeared to have plans to do more such procedures. Small molecule inhibitors for cancer stem cells in this study are available or being utilized in clinical trials for other diseases. The two women told Albini that they did not recall signing documents other than the single page form.

"Patients and physicians in the United States should be made aware that not all "stem cell" clinics are safe, and that "stem therapy" as provided in private clinics in the U.S. is unproven and potentially harmful", says Thomas Albini at the University of Miami's Bascom Palmer Eye Institute, Florida, who subsequently treated two of the women.

Each woman paid $5,000 for the procedure.

The doctors say patients should also check if anything purporting to be a clinical trial is affiliated with an academic medical centre and inform themselves about stem cell treatments.

"These women had fairly functional vision prior to the procedure. and were blinded by the next day", said ophthalmologist Dr Thomas Albini of the University of Miami, whose team examined the women after their treatment at a clinic in Florida. In the case of the eye injections, the clinic injected both eyes at once, which is highly unusual and unsafe for an experimental treatment.

The trial was not preceded by laboratory experiments and lacked comparison between treated patients and "control group" participants given a dummy therapy.

It's still not clear what went wrong, but the report says the treatment raised several red flags from the beginning.

But even if executed correctly, there is no evidence suggesting that the procedure could help restore vision, Goldberg and Albini said. A year later, the patient suffered none of the additional vision loss that would be common with the condition. iPS stem cells have a body of research behind their potential healing abilities, while the fat-based cells used in the procedures do not. It offers stem cell treatments for a variety of diseases and injuries, according to the company's website.

"With this new and exciting study, Dr. Wang and his team have provided the building blocks for understanding the cellular and genetic mechanisms behind squamous cell carcinoma", said Dr. Paul Krebsbach, dean of the UCLA School of Dentistry. "Doing the procedure in rats might have shown whether there are safety problems".

Without regulation of stem cell isolation and delivery processes at stem cell clinics, it's hard to know whether their cocktails contain harmful chemicals leftover from the isolation process - or whether they contain stem cells at all. In a regulatory filing, it reported that its techniques can treat chronic obstructive pulmonary disease, hip and knee conditions, diabetes, multiple sclerosis, Parkinson's disease, autism, colitis, and lupus.

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Unproven Stem Cell Treatment Blinds 3 Florida Women - ClickLancashire

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Scientists used stem cells to grow human heart tissue that contracted spontaneously in a petri dish marking progress in the quest to manufacture transplant organs.

A team from the University of Pittsburgh, Pennsylvania, used induced pluripotent stem (iPS) cells generated from human skin cells to create precursor heart cells called MCPs. iPS cells are mature human cells reprogrammed into a versatile, primitive state from which they can be prompted to develop into any kind of cell of the body. The primitive heart cells created in this way were attached to a mouse heart scaffold from which the researchers had removed all mouse heart cells, they wrote in the journal Nature Communications.

The scaffold is a network of non-living tissue composed of proteins and carbohydrates to which cells adhere and grow on. Placed on the 3D scaffold, the precursor cells grew and developed into heart muscle, and after 20 days of blood supply the reconstructed mouse organ began contracting again at the rate of 40 to 50 beats per minute, said a University of Pittsburgh statement.

It is still far from making a whole human heart, added senior researcher Lei Yang. Ways have to be found to make the heart contract strongly enough to pump blood effectively and to rebuild the hearts electrical conduction system. However, we provide a novel resource of cells iPS cell-derived MCPs for future heart tissue engineering, Yang told AFP by email. We hope our study would be used in the future to replace a piece of tissue damaged by a heart attack, or perhaps an entire organ, in patients with heart disease.

According to the World Health Organisation, an estimated 17 million people die of cardiovascular ailments every year, most of them from heart disease. Due to a shortage of donor organs, end-stage heart failure is irreversible, said the study. More than half of patients with heart disease do not benefit from drugs. Heart tissue engineering holds a great promise based on the reconstruction of patient-specific cardiac muscle, the researchers wrote.

Last month, scientists in Japan said they had grown functional human liver tissue from stem cells in a similar process. Creating lab-grown tissue to replenish organs damaged by accident or disease is a Holy Grail for the pioneering field of stem cell research. Until a few years ago, when iPS cells were created, the only way to obtain stem cells was to harvest them from human embryos. This was controversial because it required the destruction of the embryo, a process to which religious conservatives and others object.

Source: http://news.sudanvisiondaily.com

As the Chief Doctor of the Institute of Cell Therapy, Y.V.Gladkikh, MD, PhD, Dr. med. sc. commented: In addition to laboratory success in obtaining the functional cardiac tissue, currently there is evidence of successful implantations of heart valves and blood vessels fragments, grown from stem cells, to patients. And in 2012, the Ministry of Health of Ukraine officially approved method of treatment of critical limbs ischemia with the use of cell preparation Angiostem, developed by the biotechnological laboratory of the Institute of Cell Therapy.

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Before we get to the therapeutic stuff, here is a reminder of the main problem people with Parkinsons disease face.

Researchers are reasonably sure that the accumulation of a protein called alpha-synuclein is responsible for neurons dying in people with PD. However, there are two competing theories as to how it builds up andspreads,the threshold theoryandthe ascending theory(also called the prion hypothesis). The ascending theory states that alpha-synuclein spreads from cell to cell, infecting cells as the protein moves up through the brain.The threshold theory recently put forward by Dr. Ole Isacson and Dr. Simone Engelender, proposes that alpha-synuclein builds up independently in each affected cell.

Regardless, an improved understanding of exactly how such proteins misfold and clump together is at the heart of the riddle that is Parkinsons as well asa long list of other diseases. Thankfully a number of labs around the world have been working on this sticky problem. Additionally, if anyone wants to help you can do so very easily from any computer, watch this video to learn how.

The ongoing revolution in genetics is playing an increasingly important role in our understanding of the disease while also revealing whyit varies so much from patient to patient. There havebeen dozens of mutations and variants associated so far with the disease. We are just beginning to understand the role our genes play in the development of neurological diseases but an immense amount of progress has been made in the last 15 years since the human genome was sequenced. Now that sequencing costs have plummeted to around a thousand dollars we are on the verge of a new era in medicine that promises to give patients treatments tailored to their specific condition.

Personalized medicine is healthcare based on your unique genetic and molecular blueprint. Each individual has distinct genetic makeup, biomolecule and metabolic profiles, set of gut microbes, and so on. Similarly, there is no one-size-fit-all in healthcare. How you stay healthy or how you are treated for disease should be catered to match your unique profile. Knowledge of your genomics, proteomics, metabolomics, microbiotics, and other bioinformatics allow for the improvement in the quality of life, from disease prevention to therapy best suited to you. (from the Personalized Medicine Initiative in British Columbia.)

A better understanding ofgeneticswill help unlock a cascade of other problems that surround this disease includingmitochondrial dysfunction, lysosomal degradation, neuroinflammation,gut bacteria, andepigenetics, among others. And thankfully there is now a large interconnected global community of researchers working to solve these problems with more resources and better tools than in all of human history combined. This growth in a variety of public and private sector health initiatives across disciplines has lead a growing number of experts to believe that we will make more progress in the next decade than we did in the past century, which is good reason to be hopeful consideringwhat medicine was like a hundred years ago.

This medical revolution will be further bolstered by new and improved imaging techniques.A big part of the problem we still have with this disease is that we cant actually see what is wrong. Every person who has PDhas slightly different symptoms but we dont really know why primarily because we cant accurately see inside patients heads. Soon a new line of imaging techniques will be available that will give surgeons and researchers a much better understanding of what is going on inside the heads of each patient.

In addition, there are some immense ongoing collaborations such as theEuropean human brain projectand theU.S. brain initiativethat are trying to do for the brain what the human genome project did for our understanding of the genome. If successful it will give researchers unprecedented insight into how our minds are pieced together.

Then there are the new therapies themselves.

Levadopa For 50 years now this wonder drug has brought relief to millions. Of course, problems still persist, namely in getting it past that stubborn blood brain barrier and making sure a more steady supply is delivered to reduce on/off fluctuations. To get around some of those problems we now havepatches, slow release and extended release capsules, as well asintestinal pumps that deliver a steady flow of the drug directly into the intestines. Of course this drug is not an ideal solution as there are nasty side effects that come from long term use, predominantly dyskenisia which gives people the motor control of a blob of jelly, but for now, it is still the best stop-gap solution we have.

Deep Brain Stimulation This science-fiction wonder has become the undisputed Queen of modern treatments. It has already proven itself to be a miracle worker, re-animating hundreds of thousands with its electric wizardry. It too is steadily improving, from John Palfermans book,Brain Storms,Instead of implanting devices that simply deliver a continuous electrical stimulation, they are developing technologies that deliver stimulating jolts only when required. ..The idea is to design DBS so that the system can monitor the electrical activity in the basal ganglia, and when it detects an abnormal signal, it can respond automatically with an appropriate stimulation. A smart device

New Drugs There is along list of promising drugs that are already in clinical trial.Some of these drugs have the potential to not only offer symptomatic relief but hit the holy grail that is actual disease modifying therapies.

Neuromodulation techniques A number of novelneuromodulation techniques are being tested for clinical use. The most prevalent is called transcranialmagnetic stimulation in which magnets are attached to the outside of patients headsthat send a focused electric current deep into the target areas of the brain. Already an approved therapy for depression, TMS is now being tried in PD.

Immunotherapies The relatively recent identification of alpha-synuclein as playing a key role in disease formation has lead researchers to believe that we may be able to harness the bodies immune system to stop the protein from clumping while also mitigating the bodies natural inflammatory responses that damages neurons.

Pharmacogenetics The genetic revolutionhas spurred the development of a relatively new field of pharmacology called pharmacogenetics. Eventually, instead of making one drug for everybody, we will be able to tailor drugs to better fit each persons unique condition.

Stem Cell Therapies Though there were a series of trials in the 90s that had mixed results, recently a number of labs around the world have begun reexamining the therapeutic potential of stem cells. This is thanks in part to the 2007 discovery of anew type of stem cell called IPS cells which allow researchers to grow fully functioning stem cells from patients own skin cells. This has opened the door to a new set of therapies while also giving us better disease models. Since those first trials we have also made a series of other advances in our understanding of how to use stem cells which has lead to somestunning results in trials on other apes. Some labsare hoping to push forward with human trials starting at the end of this year.

Gene Modification Therapies As discussed earlier, the field of genetics is blowing up and one of the biggest benefits to society that will come from it is a new set of therapies called gene modification therapies.The most popular one today is called CRISPR, a technique that already allows researchers to cut and paste genetic code, changing the genome of living organisms. A number of articles have come out touting these kind of gene-editing techniques as the future of medicine. This first use ofCRISPRwas in a lung cancer patient in Chinalast fall, but it is also being used to help us understand neurodegenerative disordersincludingParkinsons disease.

Direct Programming In conjunction with gene therapy, direct programming is believed to bethe final solution to the problem of neurodegeneration. It is a subset of the new field of synthetic biologythatwill eventually allow us to change cell types in living organisms. For example, inpeople with Parkinsons disease we will be able toreprogram other healthy cells in the affected area, such as glial cells or astrocytes, and directly turn them into dopamine-producing cells.

When it comes right down to it, the reason why we have not been able to cure a lot of the diseases that are still with us today, such as neurodegeneration or cancer, is that there are an incredible number of factors to consider when trying to treat them, possibly too many for any human, or even any group of humans, to make sense of. But there might be a solution to this problem as we are now figuring out ways to export more and more of our intellectual abilities into computers. Already computers have become as good ashumans at diagnosing certain conditions, and astaggering number of healthcare companieshave now invested heavily in applyingartificial intelligence to the medical industry.This, along with further advances in nanotechnology,has a lot of potentialin helping us understand diseases such as Parkinsons and may reveal novel insights into how to treat them.

As you can see, there is plenty in the pipeline. While there may not be any magic bullet, there is no doubt that we will continue to see improvements in the treatment of Parkinsons disease that will benefit millions. While it is important to remain skeptical of all the promises being made, there is very good reason to believe that afflictions such as Parkinsons disease may one day be a thing of the past.

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We're About to Enter a New Era in Parkinson's Disease Treatments - Futurism

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A two-step method to make microglia Nature.com Microglia have been reported in some disease models to have beneficial effects; however, research into their potential as a cell therapy is limited by the lack of means to produce readily grafted, autologous microglial cells. Now, in Nature ...

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Abnormal development of the brain in an intractable disease, thanatophoric dysplasia Science Daily It is only possible, by using appropriate animal model that reproduces relevant pathophysiology, to uncover the process of pathogenesis and to develop therapy. Since the research on abnormalities of bones in TD is progressing with iPS cells at Kyoto ...

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Abnormal development of the brain in an intractable disease, thanatophoric dysplasia - Science Daily

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iPS cells may help halt failing vision

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By Andy Coghlan

A woman in her 80s has become the first person to be successfully treated with induced pluripotent stem (iPS) cells. A slither of laboratory-made retinal cells has protected her eyesight, fighting her age-related macular degeneration a common form of progressive blindness.

Such stem cells can be coaxed to form many other types of cell. Unlike other types of stem cell, such as those found in an embryo, can be made from adult non-stem cells a discovery that in 2012.

Now, more than a decade after they were created, these stem cells have helped someone. at the RIKEN Laboratory for Retinal Regeneration in Kobe, Japan, and her team took skin cells from the woman and turned them into iPS cells. They then encouraged these to form retinal pigment epithelial cells, which are important for supporting and nourishing the retina cells that capture light for vision.

The researchers made a slither of cells measuring just 1 by 3 millimetres. Before in 2014, they first removed diseased tissue on her retina that was gradually destroying her sight. They then inserted the small patch of cells they had created, hoping they would become a part of her eye and stop her eyesight from degenerating.

Now the results are in. Published today, they show that the treatment hasnt made the womans vision any sharper, but it does seem to have prevented further deterioration with her vision now stable for more than two years. Since the graft, the woman says her vision is brighter.

Takahashi and her team have done incredible work, and deserve all the praise they get for this project, says , director of the Center for iPS Cell Research and Application at Kyoto University, who won the Nobel prize for and collaborated on this work. This is a landmark study and opens the door to similar treatments for many diseases, he says.

This first iPSC-derived retinal graft is an important landmark in the field of retinal regeneration, says at University College London, and head of a trial at Moorfields Eye Hospital in London of similar grafts made instead from human embryonic stem cells.

One worry about this approach is that turning the stem cells into new tissues could lead to cancer-causing genetic mutations though the team found no evidence of this in the treated woman. However, a trial of the technique in another person was cancelled in 2015, after tests revealed that the cells intended to be given to the man had developed genetic abnormalities.

But although it has taken many years to bring , many private centres around the world have been advertising unregulated treatments purporting to use stem cells for some time.

A second study published today shows just how badly some unregulated treatments described as stem cell therapies can go wrong. Three case reports of women given such treatments for age-related macular degeneration detail how one woman went blind and the vision of the other two became much worse.

All three ended up seeking emergency treatment in 2015, after each paid $5000 to a private clinic to receive injections of their own fatty tissue into their eyes.

Patients and physicians in the US should be made aware that not all stem cell clinics are safe, and that stem therapy as provided in private clinics in the US is unproven and potentially harmful, says at the University of Miamis Bascom Palmer Eye Institute, Florida, who subsequently treated two of the women.

Albini advises people to be suspicious of any procedure involving payment. Most legitimate research in the US does not require patients to pay for the experimental procedures, he says, adding that people should check whether a trial has been registered with the US Food and Drug Administration. Be aware that if it sounds too good to be true, it may indeed not be true.

Journal reference: New England Journal of Medicine, DOI: ;

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Masayo Takahashi (second from left) treated macular degeneration with retinal tissue grown from iPS cells.

Kyodo News/Contributor/getty images

By Dennis NormileMar. 15, 2017 , 5:00 PM

Its official: The first use of induced pluripotent stem (iPS) cells in a human has proved safe, if not clearly effective. Japanese researchers reported in this weeks issue of The New England Journal of Medicine (NEJM) that using the cells to replace eye tissue damaged by age-related macular degeneration (AMD) did not improve a patients vision, but did halt disease progression. They had described the outcome at conferences, but publication of the details is an encouraging milestone for other groups gearing up to treat diseased or damaged organs with the versatile replacement cells, which are derived from mature tissues.

This initial success is pretty momentous, says Alan Trounson, a stem cell scientist at the Hudson Institute of Medical Research in Melbourne, Australia. But the broader picture for iPS therapies is mixed, as researchers have retreated from their initial hopes of creating custommade stem cells from each patients tissue. That strategy might have ensured that recipients immune systems would accept the new cells. But it proved too slow and expensive, says Shinya Yamanaka of Kyoto University in Japan, who first discovered how to create iPS cells and is a co-author of the NEJM paper. He and others are now developing banks of premade donor cells. Using stocks of cells, we can proceed much more quickly and cost effectively, he says.

Even so, clinical work is progressing more quickly than I had expected, says Yamanaka, who did his groundbreaking work just a decade ago. His collaborator on this trial, Masayo Takahashi of the RIKEN Center for Developmental Biology in Kobe, Japan, had a head start. An ophthalmologist, Takahashi was familiar with the ravages of AMD, a condition that progressively damages the macula, the central part of the retina, and is the leading cause of blindness in the elderly.

Takahashi started investigating treatments for AMD in 2000, a time when the only cells capable of developing into all the tissues of the body had to be extracted from embryos. But she was stymied by immune reactions to these embryonic stem (ES) cells. When Yamanaka announced that he could induce mature, or somatic cells, to return to an ES celllike state, Takahashi quickly changed course to develop a treatment based on iPS cells.

Her team finally operated on the first patient, a 77-year-old Japanese woman with late-stage AMD, in September 2014. They took a sample of her own skin cells, derived iPS cells, and differentiated them into the kind of retinal cells destroyed by the disease. A surgeon then slipped a small sheet of the cells into the retina of her right eye.

An operation on a second patient was called off because a number of minor genetic mutations had crept into his iPS cells during processing, and uncontrolled growthcancerhas been a worry with such cells. These changes do not directly induce cancer, but we wanted to make safety the first priority, Yamanaka says. Also, Takahashi says, AMD drugs had stabilized the patients condition so there was no urgency in subjecting him to the risks of surgery, which include hemorrhaging and retinal damage.

Immediately after surgery the first patient reported her eyesight was brighter. Takahashi says the surgery halted further deterioration of her eye, even without the drug injections still being used to treat her other eye, and there were no signs of rejection of the graft as of last December.

Clinical work is progressing much more quickly than I expected.

The result is a proof of principle that iPS cellbased therapy is feasible, says Kapil Bharti, a molecular cell biologist at the U.S. National Institutes of Healths National Eye Institute in Bethesda, Maryland, who is also developing iPS cells for treating AMD. Takahashi says once her team gains more experience with the technique they will extend it to patients with earlier-stage AMD in an effort to preserve vision.

Last month, Takahashi won approval to try the procedure on another five patients with late-stage AMD. But this time, instead of using iPS cells derived from each patient, the team will draw on banked cells from a single donor. It takes time to create iPS cells, and a lot of time for the safety evaluation, Yamanaka says. It is also costly, at nearly $900,000 to develop and test the iPS cells for the first trial, Takahashi adds.

Using donor cells to create the iPS cells will make it more difficult to ensure immune compatibility. But Yamanaka says that donor iPS cells can be matched to patients based on human leukocyte antigen (HLA) haplotypessets of cell-surface proteins that regulate immune reactions. HLA-matched cells should require only small doses of immunosuppressive drugs to prevent rejection, Takahashi saysand perhaps none at all for transplantation into the immune-privileged eye.

Kyoto Universitys Center for iPS Cell Research and Application, which Yamanaka heads, has been developing an iPS cell bank. Just 75 iPS cell lines will cover 80% of the Japanese population through HLA matching, he says. Trounson, a past president of the California Institute for Regenerative Medicine, a stem cell funding agency, says banked iPS cells have advantages. Donor iPS cells may be safer than cells derived from older patients, whose somatic cells may harbor mutations. And Jordan Lancaster, a physiologist at the University of Arizona in Tucson, likes the speed of the approach. He is devising patches for heart failure patients based on iPS-derived myocardial cells that will be premanufactured, cryopreserved, and ready to use at a moments notice.

Patient-specific iPS cells will still have clinical uses. For one thing, Bharti says it will be difficult for cell banks to cover all HLA haplotypes. And a patients own iPS cells could be used to screen for adverse drug reactions, says Min-Han Tan, an oncologist at Singapores Institute of Bioengineering and Nanotechnology, who recently published a report on the approach.

Other human trials are not far behind. Yamanaka says his Kyoto University colleague Jun Takahashi (Masayo Takahashis husband) will launch trials of iPS-derived cells to treat Parkinsons disease within 2 years. Bharti hopes to start human trials of iPS cells for a different type of macular degeneration next year. And as techniques for making and growing iPS cells improve, researchers can contemplate treatments requiring not just 100,000 cells or sothe number in Takahashis retinal sheetsbut millions, as in Lancasters heart patches.

As clinical use approaches, Takahashi cautions that researchers need to keep public expectations realistic. For now, iPS treatments may help but wont fully reverse disease, she says. Regenerative medicine is not going to cure patients in the way they hope.

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Cutting-edge stem cell therapy proves safe, but will it ever be effective? - Science Magazine

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The new report says the three women, in their 70s and 80s, paid $5,000 to be treated in 2015 for age-related macular degeneration. Participants can also report their concerns to the Office for Human Research Protections within the U.S. Department of Health and Human Services.

The "devastating outcomes" experienced by the women raise the "need for oversight of such clinics and for the education of patients by physicians and regulatory bodies", the paper said.

The women all suffered detached retinas, vision loss, and hemorrhages in their eyes.

"We don't mean to say all stem cell clinical studies are risky", coauthor Dr. Thomas Albini of the University of Miami told Reuters Health in a telephone interview.

Paul Knoepfler, a stem-cell scientist at the University of California at Davis who is a frequent critic of the clinics, said he didn't understand why the FDA and the NIH have not moved more aggressively to ensure patient safety. They sought treatment at a Florida clinic that had announced a study to treat the condition on clinicaltrials.gov, a federal database of research studies. Two out of the three patients found the trial through the website, which doesn't fully vet trials for scientific soundness. "Platelet count increased to 1.01m3 following the treatment and there were remarkable improvements in other symptoms", said Geeta Shroff, Stem Cell Specialist, Director, Nutech Mediworld. Stem cell clinics have cropped up all over the United States in recent years and are operating in a self-perceived regulatory loophole. Stem cells were then extracted from the fat and injected into their eyes. Albini says the complications could have come from injecting a contaminant into the eye, or from the fact that the stem cells may have turned into myofibroblasts after the injections, which are cells associated with scarring.

The Japanese case marks the first time anyone has given induced pluripotent stem (iPS) cells to a patient to treat any condition.

Legitimate medical research seldom requires patients to pay and, in the case of eye treatments, only one eye is treated at a time so doctors can gauge its effectiveness, the Kuriyan team said.

Although the women had moderate vision loss prior to the stem cell treatments, a year later their vision ranged from total blindness to 20/200, which is considered legally blind.

And even if the interventions were done well, they say, there is no evidence that they could have restored the patients' vision. They first cultivate stem cells to form the retinal pigmented epithelial cells that are needed to restore a damaged retina.

Shoddy stem cell preparation may have led to some of the complications, said the study authors.

The episode, described Wednesday in an article in the New England Journal of Medicine, represents one of the most egregious examples of patient injury involving a stem-cell clinic. The company also noted that it does not now treat eye patients.

The paper also mentions that the women believed that they were taking part in a clinical trial because they were aware of the clinic's work on the ClinicalTrials.gov website run by the U.S. National Library of Medicine. In other words, the company claims the study was stopped before patients were enrolled. In fact, doctors have done bone marrow transplant, a procedure where stem cell transplantation is performed.

"There's this perception that there are all these stem cell therapies out there that are close to clinical application that. are being held back by regulators and if they just step back, there would be all these treatments", he said. However, it can be hard for patients to distinguish between trials that are legitimate, and those that are not, the authors wrote.

"There's no excuse for not designing a trial properly and basing it on preclinical research", added study Jeffrey Goldberg, also a study author, of Stanford University's School of Medicine.

Researchers from the Luxembourg Centre for Systems Biomedicine (LCSB) of the University of Luxembourg and an global team have now identified an ingenious mechanism by which the body orchestrates the regeneration of red and white blood cells from progenitor cells.

See if a trial is affiliated with an academic medical center - that's a good sign it is legitimate, they say.

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3 women blinded after receiving stem cell therapy for macular degeneration - ClickLancashire

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March 17, 2017 Left: Cross-sectional view of the cerebrum in normal ferret. Neurons are localized in the cerebral cortex, the surface layer of the cerebrum. Since the surface of the cerebrum has folds (gyri), the layer containing neurons winds on its way. Right: Cross-sectional view of the cerebrum in TD ferret. Clusters of neurons (indicated by arrows) are found deep in the cerebrum, which are not detected in the cerebrum of normal ferret. They are called 'periventricular nodular heterotopia,' PNH. In addition, in the surface layer, a larger number of smaller folds (gyri) are seen than normal (indicated by asterisks). They are called polymicrogryri. Credit: Kanazawa University

Thanatophoric dysplasia (TD) is an intractable disease causing abnormalities of bones and the brain. In a recent study of ferrets, which have brains similar to those of humans, researchers using a newly developed technique discovered that neuronal translocation along radial glial fibers to the cerebral cortex during fetal brain development is aberrant, suggesting the cause underlying TD.

In TD cases, the limb and rib bones are shorter than normal, and brain abnormalities manifest, including polymicrogyria and periventricular nodular heterotopia. Previous research has determined that a gene, fibroblast growth factor receptor 3 (FGFR3), is responsible. However, as a result of TD rarity and the difficulty of obtaining brain samples from human patients, the pathophysiology of TD is largely unknown, and effective therapy has not been established.

The present research team of Kanazawa University generated an animal model of TD using ferrets that reproduces the brain abnormalities found in human TD patients. By using this animal model, the team elucidated the formation process of polymicrogyria, one of the abnormalities found in the TD brain. The team has also investigated the formation process of PNH, the other brain abnormality found in human TD patients.

First, PNH was analyzed in terms of composing cell types to reveal that a large number of neurons but few glial cell exist in PNH. In a healthy brain, neurons are found in the cerebral cortex near the brain surface. The researchers believe that during fetal brain development, PNH formation might be induced by the inability of neurons to translocate themselves to the cerebral cortex. The researchers found that the spatial arrangement of radial glial cells was distorted; radial glial fibers are believed to serve as the "track" for neurons to translocate themselves. Thus, the distortion of radial glial fibers seems to be a reason for aberrant localization of neurons.

Research on abnormalities of bones in TD is progressing with iPS cells at Kyoto University, and it is expected that the whole aspect of TD with brain and bone abnormalities would be elucidated and that the therapeutic methods would be developed. The present study on PNH was only possible using the experimental technique for ferrets developed by the research team. This animal model technique could also contribute to studies of other neurological diseases that have been difficult to investigate with conventional model animals.

Explore further: Researchers discover a gene's key role in building the developing brain's scaffolding

More information: Naoyuki Matsumoto et al, Pathophysiological analyses of periventricular nodular heterotopia using gyrencephalic mammals, Human Molecular Genetics (2017). DOI: 10.1093/hmg/ddx038

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Researchers develop new animal model to study rare brain disease - Medical Xpress

IPS Cell Therapy Comments Off on Researchers develop new animal model to study rare brain disease – Medical Xpress | March 18th, 2017

Luxembourg (Scicasts) Stem cells are unspecialized cells that can develop into any type of cell in the human body. So far, however, scientists only partially understand how the body controls the fate of these all-rounders, and what factors decide whether a stem cell will differentiate, for example, into a blood, liver or nerve cell. Researchers from the Luxembourg Centre for Systems Biomedicine (LCSB) of the University of Luxembourg and an international team have now identified an ingenious mechanism by which the body orchestrates the regeneration of red and white blood cells from progenitor cells. "This finding can help us to improve stem cell therapy in future," says Dr. Alexander Skupin, head of the "Integrative Cell Signalling" group of LCSB. The LCSB team has published its results in the scientific journal PLOS Biology.

Although all cells in an organism carry the same genetic blueprints -- the same DNA -- some of them act as blood or bone cells, for example, while others function as nerve or skin cells. Researchers already understand quite well how individual cells work. But how an organism is able to create such a diversity of cells from the same genetic template and how it manages to relocate them to wherever they are needed in the body is still largely unknown.

In order to learn more about this process, Alexander Skupin and his team treated blood stem cells from mice with growth hormones and then watched closely how these progenitor cells behaved during their differentiation into white or red blood cells. The researchers observed that the cells' transformation does not occur in linear, targeted fashion, but rather more opportunistically. Each progenitor cell adapts to the needs of its environment and integrates itself into the body where new cells are needed. "So, it is not as though the cell takes a ticket at the beginning of its differentiation and then travels straight to its destination. Rather, it gets off frequently to look around and see which line is best to take," Alexander Skupin explains. By this clever mechanism, a multicellular organism can adapt the regrowth of new cells to its current needs. "Before progenitor cells differentiate once and for all, they first lose their stem cell character and then check, as it were, which cell line is currently in demand. Only then do they develop into the cell type that best suits their characteristics and which prevails in their environment," Alexander Skupin says.

The researcher likens this step to a game of roulette, where the different types of cells can be thought of as the differently numbered slots in the roulette wheel that catch the ball. "When the cells lose their stem cell character, they are quasi thrown into the roulette wheel, where they first bounce around aimlessly. Only when they have found the right environment do the cells then drop into that niche - like the roulette ball falling into a numbered slot - and differentiate definitively." This way, the body can orchestrate its cell regeneration and at the same time prevent stem cells from being misdirected too early. "Even if a cell takes a wrong turn, it is ultimately sorted out again if its characteristics are unsuitable for the niche, or slot, it has landed in," says Skupin.

With their study, Alexander Skupin and his team have shown for the first time that a progenitor cell's fate is not clearly predetermined and does not follow a straight line. "This observation contradicts the current doctrine that stem cells are programmed to follow a certain lineage from the beginning," Alexander Skupin says. The researcher is furthermore convinced that the processes are similar for other progenitor cells. "In the lab, we have observed the same differentiation pattern in so-called iPS cells, or induced pluripotent stem cells, which can transform into many different types of cells."

This knowledge can help the researchers to improve the effectiveness of therapies in future. Stem cell therapy involves administering a patient his or her own body's stem cells in order to replace other cells that have died as a result of an affliction such as Parkinson's disease. While this promising treatment method has been intensively researched over many years, there has so far been only limited practical success in endogenous stem cell therapy. It is also highly controversial, since it is frequently accompanied by severe side effects and it cannot be ruled out that some cells might degenerate and lead to cancer. "Because we now have a better understanding of how the body influences the direction in which stem cells differentiate, we can hopefully control this process better in future," Alexander Skupin concludes.

Article adapted from a University of Luxembourg news release.

Publication: Cell Fate Decision as High-Dimensional Critical State Transition. Mitra Mojtahedi et al. PLoS Biol. (2016): Click here to view.

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Team Deciphers How the Body Controls Stem Cells - Scicasts (press release) (blog)

IPS Cell Therapy Comments Off on Team Deciphers How the Body Controls Stem Cells – Scicasts (press release) (blog) | March 18th, 2017

Scientists have long hoped that stem cells might have the power to treat diseases. But it's always been clear that they could be dangerous too, especially if they're not used carefully.

Now a pair of papers published Wednesday in the New England Journal of Medicine is underscoring both the promise and the peril of using stem cells for therapy.

In one report, researchers document the cases of three elderly women who were blinded after getting stem cells derived from fat tissue at a for-profit clinic in Florida. The treatment was marketed as a treatment for macular degeneration, the most common cause of blindness among the elderly. Each woman got cells injected into both eyes.

In a second report, a patient suffering from the same condition had a halt in the inexorable loss of vision patients usually experience, which may or may not have been related to the treatment. That patient got a different kind of stem cell derived from skin cells as part of a carefully designed Japanese study.

The Japanese case marks the first time anyone has given induced pluripotent stem (iPS) cells to a patient to treat any condition.

"These two reports are about as stark a contrast as it gets," says George Q. Daley, Harvard Medical School's dean and a leading stem cell researcher. He wrote an editorial accompanying the two papers. "It's really striking."

The report about the three women in their 70s and 80s who were blinded in Florida is renewing calls for the Food and Drug Administration to crack down on the hundreds of clinics that are selling unproven stem cell treatments for a wide variety of medical conditions, including arthritis, autism and stroke.

"One of the big mysteries about this particular case and the mushrooming stem cell clinic industry more generally is why the FDA has chosen to effectively sit itself out on the sidelines even as this situation overall grows increasingly risky to patients," says Paul Knoepfler, a University of California, Davis, stem cell researcher who has studied the proliferation of stem cell clinics.

"The inaction by the FDA not only puts many patients at serious risk from unproven stem cell offerings, but also it undermines the agency's credibility," Knoepfler wrote in an email.

In response to a query from Shots, an FDA spokeswoman wrote in an email that the agency is in the process of finalizing four new guidelines aimed at clarifying how clinics could use stem cells as treatments. The agency also noted that it had previously issued a warning to patients.

In the meantime, "consumers are encouraged to contact FDA and the appropriate state authorities in their jurisdictions to report any potentially illegal or harmful activity related to stem cell based products," the FDA email says.

Other researchers say the cases should stand as a warning to patients considering unproved stem cell treatments, especially those tried outside carefully designed research studies.

"Patients have to be wary and tell the difference between the snake oil salesmen who are going to exploit them and the kind of slow, painstaking legitimate clinical trials that are also going on," Daley says.

The New England Journal of Medicine report did not name the Florida clinic, but noted that the treatment was listed on a government website that serves as a clearinghouse for research studies. The sponsor is listed as Bioheart, Inc., which is part of U.S. Stem Cell Inc. in Sunrise, Fla.

Kristen Comella, the scientific director of U.S. Stem Cell, would not discuss the cases. "There were legal cases associated with eye patients that were settled under confidentiality, so I am not permitted to speak on any details of those cases due to the confidentiality clause," Comella said by phone.

She acknowledged, however, that the clinic had been performing the stem cell procedures. They were discontinued after at least two patients suffered detached retinas, she says.

But Comella defended the use of stem cells from fat tissue to treat a wide variety of other health problems.

"We have treated more than 7,000 patients and we've have had very few adverse events reported. So the safety track record is very strong," Comella says. "We feel very confident about the procedures that we do, and we've had great success in many different indications."

According to the New England Journal of Medicine report, The Florida clinic was using adult stem cells, which circulate in various parts of the body, including in fat tissue. While those cells may someday be turn out to be useful for treating disease, none have been proven to work.

The body produces a variety of stem cells. The kind that have generated the most excitement and controversy are human embryonic stem cells, which are derived from early human embryos and can be coaxed to become any kind of cell in the body.

Scientists are also excited about iPS cells, which can be made in the laboratory by turning any cell in the body, such as skin cells, into cells that resemble embryonic stem cells.

Those are the cells that were tested by the Japanese scientists. The stem cells were converted into retinal pigment epithelium (RPE) cells, which are the cells that are destroyed by macular degeneration.

"This represents a landmark," says Daley. "It's the first time any patient has been treated with cellular derivatives of iPS cells. So it's definitely a world first."

Daley noted that the scientists only treated one of the patient's eyes in case something went wrong, to ensure remaining vision would not be threatened in the other eye.

After at least a year, no complications had occurred and the patient had not experienced any further deterioration of vision in the treated eye. While that is promising, more patients would have to be treated and followed for much longer to know whether that approach is successful, Daley says.

"Given that macular degeneration is the most frequent cause of vision loss and blindness in the elderly and our population is aging, the prevalence of macular degeneration is going up dramatically," Daley says. "So to be able to preserve or even restore sight would be a really remarkable medical advance."

Despite the potentially encouraging results with the first patient, Daley noted that the Japanese scientists decided not to treat a second patient and suspended the study. That's because they discovered worrisome genetic variations in the RPE cells they had produced for the second patient.

"They weren't certain these would cause problems for the patient, but they were restrained enough and cautious enough that they decided not to go forward," Daley says. "That's what contrasts so markedly with the approach of the second group, who treated the three patients with an unproven stem cell therapy that ended up have devastating effects on their vision."

In this case, the New England Journal of Medicine report says, patients paid $5,000 each to receive injections of solutions that supposedly contained stem cells that were obtained from fat removed from their abdomens through liposuction.

Even though the safety and effectiveness of this procedure is unknown, all three patients received injections in both eyes.

"That's what led to these horrible results," says Thomas Albini, a retina specialist at the University of Miami's Bascom Palmer Eye Institute, who helped write the report.

Before the procedure, all three women still had at least some vision. Afterwards, one woman was left completely blind while the other two were effectively blind, Albini and his colleagues reported.

The cases show that patients need to be warned that something that "sounds too good to be true may indeed be too good to be true and may even be horrible," Albini says.

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3 Women Blinded By Unproven Stem Cell Treatments - NPR

IPS Cell Therapy Comments Off on 3 Women Blinded By Unproven Stem Cell Treatments – NPR | March 16th, 2017

Guiding a recent tour of a Kyoto University lab, a staff member holds up a transparent container. Inside are tiny pale spheres, no bigger than peas, floating in a clear liquid. This is cartilage, explains the guide, Hiroyuki Wadahama. It was made here from human iPS cells.

A monitor attached to a nearby microscope shows a mass of pink and purple dots. This is the stuff from which the cartilage was grown: induced pluripotent stem cells, often called iPS cells. Scientists can create these seemingly magical cells from any cell in the body by introducing four genes, in essence turning back the cellular clock to an immature, nonspecialized state. The term pluripotent refers to the fact iPS cells can be reprogrammed to become any type of cell, from skin to liver to nerve cells. In this way they act like embryonic stem cells and share their revolutionary therapeutic potentialand as such, they could eliminate the need for using and then destroying human embryos. Also, iPS cells can proliferate infinitely.

They can also give rise, however, to potentially dangerous mutations, possibly including ones that lead to cancerous tumors. Thus, iPS cells are a double-edged swordtheir great promise is tempered by risk. Another problem is the high cost of treating a patient with his or her own newly reprogrammed cells. But now Japanese researchers are trying a different approach.

When Kyoto University researcher Shinya Yamanaka announced in 2006 that his lab had created iPS cells from mouse skin cells for the first time, biologists were stunned. In 2007, along with James Thomson of the University of WisconsinMadison, Yamanaka repeated the feat with human skin cells. Many hailed the opening of an entirely new field of personalized regenerative medicine. Need new liver cells? No problem. Patients could benefit from having their own cells reprogrammed into ones that could help treat disease, potentially eliminating the prospect of immune rejection. In 2012 Yamanaka shared the Nobel Prize in Physiology or Medicine with John Gurdon for discovering that mature cells can be converted to stem cells. By reprogramming human cells, scientists have created new opportunities to study diseases and develop methods for diagnosis and therapy, the Nobel judges wrote. To capitalize on the discovery, Kyoto University set up the $40-million Center for iPS Cell Research and Application (CiRA), which Yamanaka directs.

A decade after the Yamanaka teams groundbreaking discoveries, however, iPS cells have retreated from the headlines; to the layperson, progress seems scant. There has only been one clinical trial involving iPS cells, and it was halted after a transplant operation on just one patienta Japanese woman in her 70s with macular degeneration, a condition that can lead to blurry vision or partial blindness. Doctors at Kobe City Medical Center General Hospital used her skin cells to grow iPS cells, which were reprogrammed into retinal cells and implanted in her eye. The treatment stopped the degeneration but the trial was halted in 2015 because genetic mutations were detected in another batch of iPS cells intended for another patient. Regulatory changes, under which the Japanese government allowed the distribution of iPS cells for clinical use, also prompted researchers to switch the study to a more efficient process of using cells from third-party donors instead of using a patients own cells. The Japanese government has a lot of incentives to considerwere developing a new science, a new technology and also a new economic market, says CiRA spokesperson Peter Karagiannis. So theres the ethical issues, but theres also money to be made. How do we balance the two?

The Kobe clinical trial had a lot riding on it. And the setback followed a major stem cell scandal in which biologist Haruko Obokata of the Riken Center for Developmental Biology was found to have falsified data in studies, published in 2014, that claimed a new method of achieving pluripotency. Then, earlier this year, Yamanaka had to apologize at a news conference after it was discovered that a reagent used to create iPS cells at CiRA was mislabeled, which could mean the wrong reagent was used. Although the mix-up is being examined, the center has halted supplies of some of its iPS cells to researchers across Japan; the error also set back by a few years a CiRA project to produce clinical-grade platelets from iPS cells.

But Yamanaka says he remains focused on the bigger picture of iPS cells and is still optimistic they can not only help researchers but may be key to transformative clinical therapies. CiRA still has a bank of tens of millions of iPS cells that have already been reset and checked for safety, so they can be used in patient applications. In terms of regenerative medicine, things have gone quicker than I expected, Yamanaka says, adding, iPS cells have exceeded expectations because of their potential for disease modeling, which allows us to elucidate unknown disease mechanisms, and drug discovery.

Those hoping for quick clinical success should remember it takes time for revolutionary treatments to go from lab bench to bedside, says Andras Nagy, a stem cell researcher at Mount Sinai Hospitals LunenfeldTanenbaum Research Institute in Toronto, who has not been directly involved in Yamanakas work. If you fully appreciate the paradigm-shifting nature of iPS cells, tremendous progress has in fact been made over the past 10 years, says Nagy, who in 2009 established a method of creating stem cells without using viruses (which had initially been used to deliver reprogramming genes into targeted cells). By comparison, penicillin was discovered as an antibiotic in 1928, but it was not available in the clinic until the early 1940s.

Researchers in Japan are meanwhile using iPS cell technology to pave the way to better drugs. For instance, CiRAs Kohei Yamamizu recently reported developing a cellular model of the bloodbrain barrier made entirely from human iPS cells. It could become a useful tool for testing drugs for brain diseases.

All eyes, however, are back on Kobe City Medical Center General Hospital, which is resuming its retina trialthis time with iPS cells from donors instead of cells from patients themselves. Using CiRAs bank of iPS cells, there are significant time and cost savingsit could be one fifth the cost of cell preparation and patient transplant or less. The initial study, with its personalized approach, reportedly cost about $875,000 for just one patient. We plan to evaluate the efficacy of transplanting the [donor] cells and consider the feasibility of using this method as a routine treatment in the future, accessible to the wider society, study co-leader Masayo Takahashi of the RIKEN Center for Developmental Biology said at a February press conference in Kobe. Her husband Jun Takahashi, a researcher at CiRA, is also planning to use donor-derived iPS cells for a clinical applicationto help treat patients with Parkinsons disease.

Nagy admits the promise of personalized cell regeneration is probably too costly for mainstream use, and he believes genomic editingin which DNA is inserted or deletedis key to safe iPS cell implants. For his part, Yamanaka is cautiously optimistic about iPS cells as a therapeutic tool.

Regenerative medicine and drug discovery are the two key applications for iPS cells, Yamanaka says. With the use of iPS cell stock, we are now able to work quicker and cheaper, so thats the challenge going forward.

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Waiting to Reprogram Your Cells? Don't Hold Your Breath - Scientific American

IPS Cell Therapy Comments Off on Waiting to Reprogram Your Cells? Don’t Hold Your Breath – Scientific American | March 15th, 2017

Researchers decipher how the body controls stem cells Science Daily "In the lab, we have observed the same differentiation pattern in so-called iPS cells, or induced pluripotent stem cells, which can transform into many different types of cells." This knowledge can help the researchers to improve the effectiveness of ... and more »

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Researchers decipher how the body controls stem cells - Science Daily

IPS Cell Therapy Comments Off on Researchers decipher how the body controls stem cells – Science Daily | March 15th, 2017

MILPITAS, Calif.--(BUSINESS WIRE)--

Applied StemCell (ASC), a leading stem cell and genome-editing company with a goal to advance genome editing and stem cell technologies for biomedical research and clinical applications, welcomes Dr. Michele Calos as a member of the companys Scientific Advisory Board (SAB).

Dr. Michele Calos is a Professor of Genetics at the Stanford University School of Medicine, Vice President of the American Society of Gene and Cell Therapy, and has served as an Advisory Committee member for the US FDA, grant review panels for the NIH and NSF, and on numerous editorial review committees of scientific journals. She is a leader in the field of molecular genetics and has developed several novel vector systems for genetic manipulation of mammalian cells. In particular, she developed novel methods for sequence-specific integration in mammalian cells using the C31 phage integrase system. A similar integrase system was also successfully used in site-specific integration in human ES and iPS cells. For this work, Dr. Calos holds a joint patent application with Applied StemCells Chief Scientific Officer, Dr. Ruby Yanru Chen-Tsai and several other Stanford researchers. Dr. Calos pioneering work with C31 integrase also set the scientific stage for ASCs TARGATT integrase technology, which was co-developed by Dr. Chen-Tsai and Dr. Liqun Luo of Stanford University for gene modification in mouse models.

We are extremely pleased to have Dr. Calos join as a member of our scientific advisory board. With her impressive background in integrase gene modification technology and gene therapy, Dr. Calos will be an invaluable guide in furthering expansion of our genome editing platforms and our gene/cell therapy pipeline, said Ruby Yanru Chen-Tsai, Ph.D., Co-founder and Chief Scientific Officer of Applied StemCell.

Dr. Calos and her research team are currently focused on gene therapy and genome engineering for the treatment of Duchenne and Limb Girdle Muscular Dystrophies and developing further novel strategies for gene and cell therapy.

About Applied StemCell, Inc.

Applied StemCell, Inc. is a leading stem cell and gene-editing company focused on the development of products and therapeutics that are enabled by its proprietary gene editing platform technologies TARGATT and CRISPR/Cas9. For more information, please visit http://www.appliedstemcell.com.

View source version on businesswire.com: http://www.businesswire.com/news/home/20170306005063/en/

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Applied StemCell Announces the Appointment of Dr. Michele Calos, Stanford Professor and Vice President of the ... - Yahoo Finance

IPS Cell Therapy Comments Off on Applied StemCell Announces the Appointment of Dr. Michele Calos, Stanford Professor and Vice President of the … – Yahoo Finance | March 12th, 2017