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APPEAL: Delroy Anglin, star of TV's 'Cant' Pay? We'll Take it Away!' (photo credit: DCBL)

DELROY ANGLIN, a star bailiff of the TV series, 'Cant Pay? Well Take it Away!' has launched an appeal to encourage more African and Caribbean people living in the UK to join the Stem Cell Register.

The #Match4Delroy appeal which is to be led by blood cancer charity, the African Caribbean Leukaemia Trust (ACLT) is hoping to find an unmatched donor for Delroy who requires a lifesaving stem cell, specifically, a bone marrow transplant if he is to beat his battle with leukaemia.

Delroy, aged 56, was diagnosed with Acute Myeloid Leukaemia (AML) last November. Since his diagnosis, Delroy has managed with drugs and receiving two rounds of chemotherapy, but tests show the leukaemia remains. Doctors have confirmed Delroy will need an urgent stem cell transplant to beat the illness.

With siblings having a one in four chance of being a match, it was no surprise out of Delroys five siblings, none were found to be a match to help their brother.

The pain and anguish of dealing with a loved one being diagnosed with blood cancer is something Delroy and his family are all too familiar with, as it was 40 years ago, Delroys brother lost his battle against leukaemia which makes Delroys illness that much more painful for his loved ones to deal with.

Delroys sister Janet Hills, who is Chair at the Met Black Police Association (MBPS) and President at National Black Police Association, or NBPA, said:

When I tell people that Delroy from Cant Pay? Well Take it Away! is my brother, there is an immediate outpouring of warmth and love. I'm praying this appeal turns that love into action. FAMILY: Janet Hills of the Met Black Police Association and Delroy Anglin's sister (photo credit: David Sillitoe/The Guardian) ACLT is the preferred charity for the MBPS and the NBPA. Our members continually engage with the charity and organise community events to raise awareness and funds. If you love Del on the show as much as I love him as my brother, then please, please, please make that commitment today to join the stem cells (bone marrow) register.

Delroy is being supported by his loving family which includes his children and mother. His daughter Domenique Anglin said:

Dad is an active, charismatic person, he loves socialising with his family and friends. He is a fantastic father to my siblings and a wonderful grandfather too. I am appealing on his behalf to all Caribbean and African people in the UK and abroad to join the register, in the hope they might be the match that saves his life.

Anglin said:

Its going to be difficult to find me a perfect matched donor unless we have a lot more Caribbean and African people on the register. It takes 15 minutes to register and is almost painless to donate. I want to beat this illness, but I will only be able to do so, with the help of the Caribbean and African community.

I am requesting for more people of Caribbean and African heritage to join the register to help me and others like me.

Beverley De-Gale, ACLT co-founder said:

Delroys life has done a complete 360 in the last four months. From being on a popular documentary series to being diagnosed with a life-threatening illness. We hope fans of the show come together and help save the life of the individual they have come to love on-screen, in addition to Joe-public who dont watch the show.

If youre 16 55 and in good health, you could potentially be the person to save Delroys life.

You could be a #Match4Delroy. Join the Stem Cell Register now, by clicking here.

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

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

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

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

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

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

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

About GvHD

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

About ApoGraft01 study

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

About Cellect Biotechnology Ltd.

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Human embryonic stem cell colony on mouse embryonic fibroblast feeder layer

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Read more: Stem cell Wikipedia, the free encyclopedia

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Next wave of innovation and discovery

Over the past 70 years, new surgical techniques and medical therapies, some of which were developed at the Stanford School of Medicine and Packard Childrens, have evolved and greatly improved outcomes for children with almost every type ofcongenital heart disease.

Heart defects that were once universally fatal can now be surgically improved. As patients born with heart disease survive longer, there are now more adults than children in the United States with congenital heart disease. However, further advancements are still needed to ensure a healthier future for patients, many of whom continue to face a compromised quality of life and require subsequent surgeries.

Surgical intervention can repair, but it rarely can truly cure, said pediatric heart surgeonFrank Hanley, MD, who is also the Lawrence Crowley, MD, Endowed Professor in Child Health at the School of Medicine and executive director of the Betty Irene Moore Childrens Heart Center. Children who have received complex surgical intervention to repair a cardiac abnormality require careful monitoring and specialized care throughout their life span. We imagine a day when a child born with a poorly working aortic valve, rather than undergoing multiple open-heart operations throughout his lifetime, instead receives a replacement valve engineered from his own stem cells. Dr. and Mrs. Moores gift comes at a critical juncture enabling us to advance beyond surgical repair to the discovery of transformational treatments and interventions and, ultimately, to true cures.

The center has an overall survival rate of 98 percent. Beyond survival alone, the goal is now to ensure an excellent overall outcome from normal brain function for even the most fragile patients, to the ability for children to perform well in school and to exercise and enjoy an active life into adulthood.

We are committed to providingbabies and children with heart disease and their families with the happiest, healthiest lives possible, from the early identification of problems, to expert intervention, and finally to a lifetime of care and support, saidStephen Roth, MD, MPH, chief of pediatriccardiologyand director of the Betty Irene Moore Childrens Heart Center.

Dr. and Mrs. Moores incredible gift will not only bolster our clinical capabilities for children and families receiving care now in the Betty Irene Moore Childrens Heart Center, it will also accelerate basic and translational research by Stanford Medicine faculty and scientists to develop more precise techniques to predict, prevent and cure, said Lloyd Minor, MD, dean of the School of Medicine. When it comes to achieving precision health, we must think as big as we can not just about treating disease, but about making and keeping people healthy and nowhere is this more true than in children.

In 2017, Packard Childrens will complete its major expansion, becoming the most technologically advanced, family-friendly and environmentally sustainable childrens hospital in the nation. The Moores gift will enable the Childrens Heart Center to expand its state-of-the-art clinical and research facilities, train the future leaders of cardiovascular medicine and surgery, and improve the field of pediatric cardiology and pediatric cardiovascular surgery through innovative research. In addition, the center will expand its clinical facilities, including a newly designed outpatient center.

Packard Childrens established the Childrens Heart Center in 2001 to focus more expertise and resources on congenital heart disease, the most common type of birth defect worldwide. Each year, approximately 40,000 children in the United States are born with heart defects, and an additional 25,000 children develop some kind of acquired heart disease.

The center has gained recognition as a national and international destination program for several highly specialized surgical procedures, and is also a full-service cardiology program that cares for patients with all forms of cardiovascular conditions. Under the leadership of Hanley and Roth, the center receives more than 25,000 patient visits annually and performs 80 to 90 percent of all cardiac surgical care for children in northern and central California.

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FINDINGS

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

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

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

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

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

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

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

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

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

The research was published in the Journal of Clinical Investigation.

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

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Investopedia

Cellect Succeeds In First Stem Cell Transplant (APOP) Investopedia It includes more than half the stem cell transplant procedures, including bone marrow transplant, resulting in a serious rejection disease called Graft-versus-Host-Disease (GvHD). GvHD is a medical disorder which results from receipt of transplanted ... Cellect Announces Successful First Cancer Patient Stem Cell TransplantP&T Community Why Cellect Biotechnology Ltd. (APOP) Stock Is Soaring TodayInvestorplace.com all 28 news articles »

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Stelios Andreadis, PhD, is leading a team of researchers who have discovered how to convert adult skin cells into stem cells without modifying their genetics.

UB researchers have found that adult skin cells can be converted into neural crest cells without any genetic modification.

The discovery, which was several years in the making, proves that these stem cells can yield other cells that are present in the spinal cord and brain.

The applications could be very significant, ranging from studying genetic diseases in a dish to generating possible regenerative cures from a patients own cells.

Its actually quite remarkable that it happens, says Stelios Andreadis, PhD, professor of biomedical engineering, who recently published a paper on the results, titled Reprogramming Postnatal Human Epidermal Keratinocytes Toward Functional Neural Crest Fates, in the journal Stem Cells.

The identity of the cells was further confirmed by lineage tracing experiments, where the reprogrammed cells were implanted in chicken embryos and acted just as neural crest cells do.

This image shows Keratinocyte-derived neural crest stem cells turning into neurons as shown by typical neuronal morphology.

Stem cells have been derived from adult cells before, but not without adding genes to alter the cells. The new process yields neural crest cells without addition of foreign genetic material. The reprogrammed neural crest cells can become smooth muscle cells, melanocytes, Schwann cells or neurons.

In medical applications this has tremendous potential because you can always get a skin biopsy, says Andreadis, who is also professor and chair of the Department of Chemical and Biological Engineering in the School of Engineering and Applied Sciences.

We can grow the cells to large numbers and reprogram them without genetic modification. So, autologous cells derived from the patient can be used to treat devastating neurogenic diseases that are currently hampered by the lack of easily accessible cell sources, he says.

The process can also be used to model disease. Skin cells from a person with a genetic disease of the nervous system can be reprogrammed into neural crest cells. These cells will have the disease-causing mutation in their chromosomes, but the genes that cause the mutation are not expressed in the skin.

The genes are likely to be expressed when cells differentiate into neural crest lineages, such as neurons or Schwann cells, thereby enabling researchers to study the disease in a dish. This is similar to induced pluripotent stem cells, but without genetic modification or reprogramming to the pluripotent state.

The discovery was a gradual process, taking almost five years, Andreadis says, as successive experiments kept leading to something new.

It was one step at a time. It was a very challenging task that involved a wide range of expertise and collaborators to bring it to fruition, he says.

Collaborators include:

Andreadis credits the persistence of his then-doctoral student, Vivek K. Bajpai, for sticking with it.

He is an excellent and persistent student, Andreadis says. Most students would have given up.

The research was supported by grants from the National Institutes of Health (NIH).

Andreadis also credits a seed grant from UBs Office of the Vice President for Research and Economic Developments IMPACT program that enabled part of the work.

The work recently received a $1.7 million NIH grant to delve into the mechanisms that occur as the cells reprogram, and to employ the cells for treating the Parkinsons-like symptoms in a mouse model of hypomyelinating disease.

This work has the potential to provide a novel source of abundant, easily accessible and autologous cells for treatment of devastating neurodegenerative diseases, Andreadis says. We are excited about this discovery and its potential impact and are grateful to NIH for the opportunity to pursue it further.

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Forbes

Four Ways to Younger Skin Right Now Forbes Her Hydrating and Plumping Serum No1 combats the environmental stressors that skin faces every day to detoxify and rejuvenate the face and subsequently enacting anti-aging properties. By using plant-stem cells, hyaluronic acid, marine snail peptides ...

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Malia

March is Multiple Sclerosis Month and Hadassah leads the world in the stunning success and implementation of the research at Hadassah Hospital in Jerusalem. Malia is just one of the many stories of triumph in Prof. Dimitrios Karussis program.

Malia was a top Dallas trial attorney-a senior partner in a law firm and the mother of three children, the youngest, age five. Her energy level was high and her life was full. And then, 18 years ago, she was diagnosed with multiple sclerosis (MS).

In the beginning, her symptoms were mild. For example, she was a little unsteady in her walking. She began taking a weekly injection of Avonex because, as her doctor told her, "You wouldn't want me to treat you for high blood pressure only after you had a heart attack."

Malia took Avonex for about 10 years. Still, her life became a journey of declining energy and mobility. Her biggest fear was that she would lose her vision and not be able to drive her children around. Fortunately, she didn't experience any problems with her vision, but her gait did get worse, as did the fatigue.

Ultimately, she could only walk a very short distance with a walker. Her balance became more unsteady. Her speech was harder to understand. And the fatigue got so bad that even taking a shower was so exhausting that she needed a nap afterward.

Malia tried four or five different medicines, but none seemed to help. As she explained, however, the promise of these medicines is only to slow the progression of the disease, not to improve upon one's symptoms.

At one point on this odyssey of decline, Malia fell and broke her leg and found herself in a wheelchair for six weeks. She began to research other treatment options, surfing the Internet extensively. It was how she came upon the innovative stem-cell work of Prof. Dimitrios Karussis, senior neurologist at Hadassah Hospital Ein Kerem and head of its Multiple Sclerosis Center. She learned that Prof. Karussis was conducting a unique MS clinical trial involving the injection of a patient's own bone marrow-derived stem cells directly into the spinal cord.

When she met Prof. Karussis for the first time, she was walking with a walker, but couldn't walk any real distance. It was December 2014. In the spring of 2015, Malia, who was accepted into one of Prof. Karussis' clinical trials, traveled back to Hadassah to have a bone marrow extraction, which would be the basis for her stem-cell transplant. As she explains, just a syringe of cells is extracted, but then these cells are enhanced in culture for six months. In the fall of 2016, Malia received her first infusion. The actual infusion, itself, Malia relates, took only five minutes!

"I got the infusion at 2 p.m.," Malia recalled, "and eight hours later, I did something I had not done in two to three years." She explained that before the infusion, whenever she got out of bed, to get back in, she would have to lift her right leg with her hands. This time, post infusion, after she got up to go to the bathroom, she was able to get back into bed by just lifting her right leg onto the bedwithout holding it! She called the kids; her husband, David, made a video of her new ability. When her doctors came in that next morning to see how she was doing, she showed them her new skill. "Everyone was sobbing," she recalled; "we were all so excited by how amazing it was."

Malia said: "The most important thing of all was that the level of my fatigue was so improved."

Taking herself back to the night she returned home from that first infusion, Malia recalled: "We arrived back home at midnight. The next morning at 8, I was up writing my blog. Then I unpacked, did laundry, and made a five-course dinner for my husband, after which he went to bed, exhausted from the jet lag. I, however, stayed up to crochet a new blanket until midnight."

That renewed energy has never disappeared since Malia started getting stem-cell infusions at Hadassah. She received two more in 2016 and the most recent in February 2017. The kind of improvements varied with each infusion-sometimes the improvement in her gait was most noticeable; other times, it was the clarity of her speech. With the 2017 infusion, it was her balance. A year or so ago, she could only balance herself on a "whole body vibration machine" for about a minute and a half. Then she worked this up to five minutes. After this fourth infusion, she could stay on the machine for 12 minutes and even let go of the bar with one hand.

In addition, before every infusion, a baseline measurement of her abilities is taken. The most recent baseline, Malia reported, was higher than ever before. By the same token, her overall physical improvements are the best they've been.

Malia's recovery is not without its setbacks. As the stem-cell treatment wears off, she does experience some backsliding in her improvements-perhaps in her speech or the way she walks. Nevertheless, after she has a treatment, the improvements return or surpass the previous ones.

"I had forgotten what it was like to feel like a regular person, like someone with a normal level of energy; I had forgotten what it was like to feel good," Malia said.

When she is out and about with her walker, she sees people looking at her with sympathy. But she doesn't feel sorry for herself, she notes, because she has gotten her life back. Malia feels blessed that she has come this far. "Until you've been there, you can't really understand just how important it is to feel like a regular person."

Malia had stopped practicing law when she was diagnosed with MS. But just recently she reactivated her law license. She took on a case, with a colleague. And in the beginning of April, she will be going to trial.

"When you feel like you can do things you used to do, life is as it should be," Malia said.

Malia no longer takes any medication for MS and being an MS patient is no longer a central focus of her life. But, she is now a stem-cell activist and fundraiser on behalf of Hadassah because "I feel I have been given a gift and I want to ensure that more people with MS can be helped."

While she doesn't know the other participants in the clinical trial, she did meet one man from London last time she was at the hospital for her infusion. As she relates, this man was "giddy with excitement, lifting his cane in the air with one hand." They began to talk and he explained that he just had his first stem-cell infusion and, overnight, he was able to lift his cane up in the air with his right hand when for 15 years he had been unable to even hold anything in that hand.

"What I would like Hadassah supporters around the world to understand," Malia related, "is that yes, I get the wonderful benefits of Prof. Karussis' stem-cell treatments (and, of course, she does not minimize his trailblazing hard work and miraculous success), "but I also was surprised to learn that I have this large Hadassah cheering squad that gives me hope and supports me-making me believe in possibilities for improvement. As Malia recalled, when she sits with a visitor from Hadassah and sees the look of awe on the face of this Hadassah supporter because of the leap in her improvement, it gives her renewed strength. "There are many things I want to do with the rest of my life," Malia said, "and raising money for Hadassah is at the top of the list."

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Lacey Miller will probably never forget her sons first birthday, at least not for the usual reasons. She threw him a party at a park near their home in Culver. Throughout the whole thing, she couldnt walk and could barely see, merely moving was a challenge.

I just kind of sat there and everyone cleaned up and they got me back to the house, she said.

Miller has a form of multiple sclerosis, a disease that disrupts communication between the brain and the rest of the body. The symptoms numbness, difficulty concentrating, blurred vision and fatigue come in cycles and tend to worsen over time.

When she was diagnosed in 2012 at age 27, Millers symptoms werent so bad: little things like numbness in her legs and blurry vision. It wasnt until three years later, when she became pregnant with her son, that things went downhill fast. She had to be induced into labor early because she had so much trouble walking. After that, there was vertigo, vision loss and walking became more difficult. Her worsening symptoms culminated in an emergency room trip following her sons first birthday party.

I was going to be in a wheelchair in probably a year, she said.

Internet exploration brought her to a study at Northwestern University in Chicago that is further exploring the idea of, in a sense, resetting the immune systems of multiple sclerosis patients. Multiple sclerosis develops when a persons immune system attacks the protective covering of nerve fibers in the central nervous system, making it difficult for the brain to communicate with the rest of the body.

Miller applied to join the study in June 2016 and was approved in September. She started her testing and treatment in early January, embarking on what would be months of grueling treatments and travel.

Through it all, Miller thought about her now-18-month-old son, Emmerick.

Even on the days when it was like, This sucks. This is really hard, it was like, Im doing this for him so he can have a mom who can run down to the park with him and do things with him, she said.

Thats a win

A handful of prominent studies in recent years have raised hope that a procedure called hematopoietic stem cell transplantation can prevent further disability among patients with multiple sclerosis.

A study on 25 multiple sclerosis patients published last month gave further hope. Three years after undergoing stem cell transplants, disability symptoms in nearly 80 percent of the patients had not worsened, according to the study, published in the in the Journal of the American Medical Association Neurology. At five years, nearly 70 percent of the patients symptoms still had not worsened.

Dr. Linda Griffith, an author on the study and a medical officer at the National Institute of Allergy and Infectious Diseases, which sponsored the study, said being able to halt existing symptoms is a big advance.

To us, thats a win if they dont get worse, she said. We have no notion of being able to cure MS here and make it go away. Its not going to go away. Its a bad disease. But medical investigators are really thrilled and excited when they can find that the disease isnt getting worse.

Dr. Richard Burt, a professor of medicine and chief of immunotherapy at Northwesterns Feinberg School of Medicine, found similar results in a 2015 study he and his team published in the Journal of the American Medical Association. Five years after undergoing stem cell transplants, only 10 percent of multiple sclerosis patients had worsened symptoms.

The majority got better and stayed better, he said. Thats a paradigm change.

Creating hope

The stem cell transplant procedure involves extracting a patients bone marrow stem cells using a long needle and using chemotherapy and other toxic medications to clear out the rest of the cells. In multiple sclerosis patients, the patients own stem cells are then put back into the body.

It is now immature, just like if you were a child again and your immune system was learning all over again whats its supposed to be reacting against versus not, Griffith said.

While it sounds exciting, Griffith cautions the research is far from conclusive. She hearkens back to the hype around early studies that showed the procedure could be helpful for breast cancer patients. It ultimately wasnt.

What needs to happen next, she said, is a randomized study that compares groups of multiple sclerosis patients who either receive the transplant or the medications typically used to treat the condition, following them over time.

Not for everyone

Burt, the first doctor to perform these stem cell transplants for multiple sclerosis patients in the U.S., performed the procedure on Miller.

He cautions that only a specific subset of multiple sclerosis patients will benefit from the procedure. They must have a common form of the condition called relapse-remitting, meaning symptoms come in waves that recur over time. He doesnt perform the procedure on people who have progressed to a later stage, as it would be too late to help them, he said. Imaging on their brain and spine must show new lesions, indicating the disease is active.

Its frustrating because people learn about this on Facebook and want to get it, she said. Theyre upset when we decline them.

Burt is confident, however, that he knows who his procedure can help and who it cant help.

We developed this over many decades, he said.

When Miller, who is now 32, went in for testing with Burt, it was in the middle of an attack. She could barely walk into the office at Northwestern; her dad had to hold her arm for balance.

Although she was accepted into the study, getting her health insurance carrier on board proved to be a struggle and logistical issues delayed her ability to officially participate. Burt is still treating her under the study protocols, however, on whats referred to as a compassionate basis.

Millers first trip to Chicago was a short one for pre-study testing: imaging to check the progression of her multiple sclerosis, among other evaluations. She flew back to Chicago on Jan. 21 for what would be a two-week trip. First, they gave her chemotherapy to kill her stem cells and suppress her immune system. That meant she lost her hair.

Then they put a long needle into her neck to remove the remaining stem cells. She still has a tiny, round scar where the needle went in.

On Feb. 12, she flew back to Chicago again to have her stem cells put back into her body. She was discharged on March 1.

Sometimes, Burt said his patients notice right away the so-called MS fog, the fatigue and inability to think clearly, is lifted once they finish the procedure.

Miller said that was the case for her. She noticed other things right away, too. Walking around downtown Chicago was much easier after the surgery than before. She no longer had to use a cane or wall to guide her and she no longer had to stop and rest.

The other day, her fiance, Chris, asked her what the score of the basketball game was. She told him.

He was like, Can you see that? she said. I was like, Yeah! I didnt realize things are just so crisp and clear.

The near-constant numbness in her legs also isnt as bad as it used to be.

We were kind of joking the other day because my fiance touched my foot, she said. I was like Oh my god, I can feel that! I hadnt been able to feel my feet.

No more drugs

Burt theorizes that in addition to helping peoples quality of life, stem cell transplants could also help them financially, a point he hopes to flesh out in his current research.

A 2015 study in the journal Neurology found multiple sclerosis drugs cost patients roughly $60,000 per year. The price of the drugs increased annually between 1993 and 2013 at rates of five to seven times higher than the rate of prescription drug inflation, according to the study.

Miller, who works as a juvenile parole officer in Jefferson County, estimates she was spending about $500 a month on the drugs after her insurance paid its portion. She had to quit one medication a shot she gave herself three times per week because it gave her flu-like symptoms. Not having to take medications anymore was one of the main reasons Miller said she wanted to enroll in Burts study.

Im kind of one of those people that I dont even like taking Tylenol, she said. Id rather just not take anything.

Miller isnt currently on any multiple sclerosis medications. She said she hasnt felt this good in years. She returned to work part time this week.

Burt routinely travels to medical centers around the world explaining the procedure in hopes other neurologists will perform the stem cell transplants on multiple sclerosis patients. The older ones tend to be more set in their ways, but he believes the technique will catch on among younger doctors.

The procedure is currently being performed in England, Sweden and Brazil, Burt said. A hospital in India recently expressed interest, too.

At the end of the day, we want to help people throughout the world, Burt said, and in fact this is spreading throughout the world.

Reporter: 541-383-0304,

tbannow@bendbulletin.com

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A small stem cell trial in which patients with severe spinal injuriesappeared to make remarkable progress is still showing excellent results, according to the company conductingthe research.

One of the patients in the trial is 21-year-old Kris Boesen, from Bakersfield, California, whose story we reported on last year.A car crash had left theBakersfield, California native with three crushed vertebrae, almost no feeling below his neck, and a grimprognosis. Doctors believed he would live the rest of his life as a paraplegic.

Enter stem cell therapy. Most treatments for serious spinal injuries concentrate on physical therapy to expand the range of the patients remainingmotor skills and to limit further injury, not to reverse the actual damage. But last April, as part of an experimental phase 2 clinical trial called SCiStar, researchers injected Boesen with 10 million stem cells. By July, hehad recovered use of his hands to the point where he could use a wheelchair, a computer and a cellphone, and could take care of most of his daily living needs.In recent months his progress has continued, says his father.

Boesen is not the only patient to have improved in the trial, according toAsterias Biotherapeutics, which is conducting the research. Boesen is part of a cohort of six patients who were experiencing various levels of paralysis and were injected with the 10 million stem cell dose. In a Jan. 24update, the company saidfive of those patientshad improved either one or twolevels on a widely used scale to measuremotor function in spinal injury patients.

On Tuesday, Asterias issued a newupdate, announcingthat the sixth patient in the cohort has experienced a similar improvement.

While spontaneous recovery for spinal injury patients does occur,the likelihood of all six patients recovering to the degree they haveis less likely, researchers say.

This is as good as you could hope at this point, said Charles Liu, Boesens neurosurgeon and director of the USC Neurorestoration Center. So far all the evidence is pointing in the right direction.

To measure improvement in spinal injury patients, researchers use two yardsticks: the Upper Extremity Motor Scale, or UEMS, and the International Standards for Neurological Classification of Spinal Cord Injury, or ISNCSCI. On the UEMS scale,patients are scored from 0 to 5 on theirability to use five key muscles in the wrists, elbows and fingers. The ISNCSCI scale assesses where damage has occurred along the different levels of the cervical vertebrae, which generally determines the scope of impairment to the body and the level of care needed.

For instance, if a patient has sustained damage at the fourth cervical vertebra down, known as C-4, at the base of the neck, it generally means that person is paralyzed from the neck down, requiring round-the-clock care and a ventilator to breathe.A patient with a C-5 injury may not be able to move her arms or hands, requiring about 6 to 12 hours per day of assisted care; and at the C-6 level, better motor function mayallow a patient to take care of most of herdaily living needs on her own.

Which is all to say that even one level of recovery could substantially improve the daily life ofa spinal injury patient.

According to Asterias, all six patients in the 10million-cell cohort have improved their general UEMS scores, and jumped at least one motor level on the ISNCSCI scale on one or both sides of their body.

Two patients have improvedtwo motor levels on one side; and one patient,Boesen, has improved two motor levels on both sides.

Steve Cartt, president and CEO of Asterias, said anotherpatient, Jake Javier of Danville, California, has gonefrom partial paralysis to being able to use his hands well enough to considerpursuing a computer science career.

Throws Like a Regular Throw

In September, Boesens father, Rod Boesen, told us how excited he wasthat his son had regained some feeling in one of his feet. Last week, at11 months post-injection, the elder Boesensaid Kris has continued to improve.

Now he can move his toe and his knee together at the same time, Boesen said. Theyre about to give him a manual wheelchair now [instead of a motorized one]. He can grip with his hands enough to use a manual one.

Boesen said the movement in his sons arms and hands has greatlyimproved since September.Kris, a formerhigh school pitcher, had beenflinging a ball to his dog like people throw hand grenades, Boesen said. They kind of cradle them and thats how Kris would do it. But now he throws like a regular throw, tosses that ball down the hall, has that release point down, and just wings it.

Asterias is currently recruiting patients for a trial in which theyll receive 20 million stem cells, the optimal dose, according to company researchers. Two patients have already started the 20 million stem cell therapy, and six-month results from those patients will be released in the fall, Cartt said.

Patients who received 2 million stem cells in an earlier phase of the study have not shown much change in their condition, according to the Jan. 24 update.

Guarded Optimism

While Boesens father is impressed with the results, the optimism of researchers inside and outside the studyhas been guarded.The trial is still in its early stages, and the sample size is small, said Paul Knoepfler, a cell biology professor and stem cell researcher at UC Davis, who is not involved in the SCiStar study.

As a scientist, I still would want to wait for more data, Knoepfler said. Its certainly interesting, but its still early. Its a phase 2 trial.

To address the issue of small sample size, Asterias islooking at historical data to determinethe level of improvement for patients in similar circumstances who did not receive stem cell therapy. The company has said it found a meaningful difference in the recovery of its study patients compared to the norm.

Liu said one of the most importantresults is the lack of significant side effects or other negative outcomes resulting from the treatment to date.

Thats very significant to me, Liu said. Thats the first thing you look for, is anyone hurt from this therapy.

There was also a concern, he said, that some patients might regress over time, once the initial injection of stem cells wore off. Thathasyet to occur.

No one has lost anything theyve gained, Liu said. We were very happy to see that. This is all very promising.

The next step for the SCiStartrial will be to establish a control group, Cartt said.

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Strong Progress for Paralyzed Patients After Stem Cell Therapy, Company Says - KQED

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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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For a soldier who suffered a spinal cord injury on the battlefield, the promise of regenerative medicine is to fully repair the resulting limb paralysis. But that hope is still years from reality.

Not only powerful, but efficient. Studying diseases in lab-created tissue may help reduce the price tag now roughly $1.8 billion for bringing a new drug to market, which is one of the reasons Ashton received a National Science Foundation CAREER Award for advancing tissue engineering of the human spinal cord. During the projects five-year funding period, his lab in the Wisconsin Institute for Discovery will fine-tune the technology for growing a neural tube, the developmental predecessor of the spinal cord, from scratch.

As the neural tube matures and diversifies during the development of a human embryo, it gives rise to the two core parts of our central nervous system: the brain and spinal cord. By replicating this process in a dish, Ashton hopes to develop a platform for research that is highly reproducible and can be broadly disseminated. Biologists could simply add their cells to Ashtons starter tissue to build a model of whichever spinal cord disease they desire.

By starting with cells from an individual patient, researchers will be able to target disease therapies to a particular genetic background a concept known as personalized medicine. Drug tests in engineered spinal cords may become an intermediate step between animal models and clinical trials of patients affected by Lou Gehrigs disease, multiple sclerosis or spinal cord injuries, helping to bridge the differences between a human and rodent spinal cord.

Weve cured spinal cord diseases in a lot of rodents over the years, but only a small percentage of those drugs work in humans, Ashton says. If we can make the engineered tissue as close as possible to whats in our body, this will eventually translate to better drugs.

The recipe Ashton and fellow UWMadison engineering professors Lih-Sheng (Tom) Turng and David Beebe plan to follow to coax stem cells into forming a neural tube goes something like this: First, they use water-soluble Lego-type molds to create microscale cavities within a jelly-like substance. Then they add human neural stem cells into these cavities, and let the cells coalesce as they do naturally to form neural tube-resembling tissue.

Next, they add signaling molecules whose variable concentration instructs the stem cells to turn into different types of neurons and neuron-supporting cells. Last comes the greatest challenge: getting these specialized cells to connect to one another and form electric circuits that give the spinal cord its function.

The complexity of the central nervous system exists because specific circuits have to form over very long distances. If any part of a circuit goes awry, you lose function, Ashton explains. The biggest open question is whether the tissue we create in vitro will have the proper wiring of different cell types to yield circuits similar to those in our body.

Ashtons CAREER Award will also fund educational activities that range from expanding outreach programs for underrepresented minority K-12 students, to creating a website and exhibit for the public, to educating Madison-area high school teachers about tissue engineering.

Ashton, who is African-American, comes from a socially active family background his grandfather was a minister and president of the Virginia chapter of the NAACP. He has assisted the nonprofit group 100 Black Men of Madison with its K-12 mentoring programs for years.

He plans to use the new grant to develop virtual interactive lab experiments and matching teaching modules.

I hope to attract more students to the fast-growing field of regenerative medicine, Ashton says, and to motivate people to continue to fund this kind of research so that we can develop therapies to cure diseases, instead of just treating their symptoms.

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

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Growing a Neural Tube in a Dish | Technology Networks - Technology Networks

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Twice a year, I strip down to my underwear, don a paper gown and subject myself to a full-body examination at the dermatologists office. These are done twice as often as most other patientsand for good reason. Not only am I freckly and fair-skinned, Ive had an unhealthy relationship with the sun, which makes me more susceptible to skin cancer.

During my teens and 20s, when I was a lifeguard and camp counselor, I spent the majority of my summers outdoors. Like my peers, Id wanted to achieve the perfect tan. Id worn sunscreen, but it was SPF 4barely any protection, compared with what doctors recommend today.

Now, Im paying the price. This past decade, Ive had a handful of suspicious-looking moles removed. Recently, my dermatologist sent me to a medical photographer for a full-body photo session to document my moles, in case they change.

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My situation isnt unique. Countless people worldwide didnt protect themselves adequately from the suns ultraviolet rays during their youth. Decades ago, doctors didnt preach about sun protection, and researchers didnt realize that the suns ultraviolet rays could cause skin changes that can lead to melanoma, the deadliest form of skin cancer.

The most important reason for the increase in melanomas is thought to be due to increased exposure to ultraviolet radiation from sun and artificial tanning sources, says John J. DiGiovanna, staff clinician in the dermatology branch of the National Cancer Institutes Center for Cancer Research in Bethesda, Maryland.

Melanoma is only the ninth most commonly diagnosed cancer across Europe, but its rates have been rising sharply since the 1980s, six-fold among some groups.

Every year, 100,000 new cases of melanoma are diagnosed in Europe, says John Haanen, head of medical oncology at the Netherlands Cancer Institute in Amsterdam. Caucasians are at greatest risk, especially those with fair skin, red hair and freckles. Risk rises after age 40especially sun worshippers. Many experts refer to the increased prevalence as an epidemic.

I would not call it a melanoma epidemic but a skin cancer epidemic, says Reinhard Dummer, director of the Skin Cancer Centre at University Hospital Zrich. We expect in Switzerland that one out of five persons will develop skin cancers once in their lives.

Cultural changes over several decades are likely to blame. Bathing suits have gotten skimpier, and seaside vacations have become more common, exposing pale office workers to intense sunlight for short periods.

In Europe, low-cost air travel has increased the ability for people to travel to sunny, warmer climates for a week here and there, says Alex Menzies, medical oncologist at Melanoma Institute Australia, the country with the highest melanoma rates in the world. Intermittent exposure to the sun with burning is a major risk for melanoma.

Even if youve endured decades worth of sun exposure, there is hope.

The earlier you notice melanoma, the greater your chances are of being cured. Surgery is the primary treatment. If you picked up an early-changing mole, you could have a virtually normal life expectancy, says Girish Patel, lead investigator for the Skin Cancer Stem Cell Research Program at Cardiff University.

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Regular skin checks and meaningful lifestyle changes to limit further damage from the sun help improve the odds. Since Imogen Cheese, 37, of Gloucestershire, England, was diagnosed with stage II melanoma in 2013, shes screened by her medical team every three months. I cover up to avoid the midday sun, says Imogen. I wear high factor SPF, I am active and eat a healthy balanced diet. So far, her cancer has not progressed.

Researchers have made great strides in the treatment of advanced melanoma. One option: Targeted therapy, which can be given to stage IV patients with specific genetic mutations.

Melanoma researchers in Australia have been involved with targeted therapy research since the beginning, about seven years ago. We do testing on their tumors to look if there are any mutations in certain genes in the tumor, says Menzies. We have targeted therapy that can attack the BRAF mutation, which is found in about 50 percent of tumors from patients. If we give tablets for BRAF-mutant melanoma, almost every patient will have shrinkage of the tumor. On average, it will keep things under control for one year, and the one-year survival rate has improved to 70 percent, from 30 percent five years ago.

Five years after John Ambrose, 67, of New South Wales, Australia, had a grade IV skin melanoma removed he began coughing up blood. His disease had spread to both lungs and his prognosis was poor. He joined a targeted therapy clinical trial in 2013, and within three months, his tumors shrank by 70 percent. After 18 months, he had clear scans. Today, John travels, plays golf and spends time with his grandchildren.

My situation has not stopped me living a normal life, he says.

Texas native Jesse Thomas, 57, also benefited from targeted therapy after being diagnosed with stage IV melanoma in 2013, with tumors on his neck, liver and spine. Genomic testing revealed Jesse had an uncommon V600K BRAF mutation, and his oncologist was able to pinpoint a targeted therapy for him.

They expected the cancer to stop growing, but it actually shrank, Jesse says. Theres no way to cure it, but I am controllable.

Targeted therapy is only for stage IV patients, but researchers are studying its effects on stage III patients. We should know within a couple of years whether these treatments are beneficial, says John Haanen.

Researchers have been able to stimulate the T-cells in some melanoma patients immune systems to fight cancer, with astounding results.

T-cells kill off viruses and other things, Menzies says, but with cancer, theyre sitting there around the tumor, asleep. They know that the tumor is foreign, but the tumor has turned them off, stopping them from killing it. Immunology drugs turn on the T-cells and they kill the tumor.

Melanoma researchers consider immunology the biggest breakthrough in decades.

This is our penicillin moment in oncology, Menzies says. Melanoma can be turned into a chronic disease, and many people will not die from it in the near future if we continue to go the way were going.

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Immunotherapy doesnt work for everyone, but it can be quite effective. Cardiff Universitys Patel says, In the 45 or so percent of people who respond, they can respond for very long periods of time.

In 2013, Cardiff resident Vicky Brown, 62, was shocked to learn that a lump in her breast was actually melanoma, not breast cancer. Shed had early-stage melanoma in 2006, which returned in her breast and lungs.

Through a clinical trial, Brown received intravenous doses of two immunotherapy drugs. Within weeks, her tumors shrank. She discontinued the drugs due to side effects, but it kept the melanoma in check for a year. In 2015, after new lung tumors appeared, she received more immunology treatments. The drugs again shrank her tumors.

I am hoping this couple of doses will give me more time again, Vicky says. My grandson is now nine months old. I want to be able to make memories for him, as well as my four-year-old granddaughter.

Researchers are working to get more patients to have a positive response to the treatment. The notion is that clearly, if we can do it in a few, we should be able to do it in the majority, says Patel.

For years, researchers tried creating a melanoma vaccine, to no avail. Now, researchers are combining the success of immunotherapy with the concept of vaccines, leading to personalized melanoma treatments.

As we better understand how the immune system recognizes the melanoma cells, we are developing so-called personalized vaccines, Haanen says. We are starting now in metastatic patients and if this concept works well move to earlier stages.

Hein Jambroers, 50, of Roermond, Netherlands, has benefited from a personalized treatment called adoptive cell therapy (ACT). He was diagnosed with stage II melanoma in 2009, but a year later, he had stage IV disease, with tumors on his right leg and liver, and was told that he had less than six months to live.

After getting some short-term benefit from targeted therapy, Hein was referred to an ACT clinical trial in 2011. Doctors at the Netherlands Cancer Institute harvested some of his white blood cells, then monitored them in a laboratory to identify the healthiest T-cells to fight melanoma. They were replicated in large numbers. Hein received chemotherapy to kill his existing T-cells, then got an infusion of the laboratory-created T-cells, which basically gave him a new immune system that shrank his tumors within three months.

Hes what doctors call a complete responder. Hes had clean scans ever since; no trace of melanoma.

Complete responders have an excellent prognosis, says Haanen, who treated Hein. Cure is always difficult to say, but very long-term remissions which could be cureare seen in the majority of complete responders and in some partial responders.

Hein, who expected to die, is cautiously optimistic. Im very positive about my future, but Im always on a state of alert, he says. I sit in the shade. I cream up with sunscreen. I even do it for my child and my wife. I dont want to tempt the fates.

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Soon, doctors may defeat cancer by attacking stem cells.

Skin stem cells make thousands of healthy skin cells. Melanoma stem cells work similarly, except they make thousands of malignant melanoma cells. Researchers are targeting melanoma stem cells to stop tumors from spreading.

Its like killing off the queen bee, Patel says. The whole hive then dies away, because youve gotten to the cell thats giving rise to everything.

This is vastly different from chemotherapy, which aims to kill as much cancer as possible. Stem cells make up only one to three per cent of some skin cancers.

If you got rid of the cancer stem cell population, the whole tumor could not proliferate, Patel says. If you take the bulk of a tumor and regrow it in a mouse without stem cells, it cant form. But if you take a small part of the cancer stem cell population, it grows back fully.

Researchers have begun clinical trials, and treatments could be available in a decade.

Despite sun damage that I endured during my youth, Im optimistic that Im doing everything that I can to stay ahead of any problems that may crop up. Ive got photos of all of my moles and freckles now, which I use for monthly self-exams. Ill bring them to my dermatologist for my next full-body examination. Ive also been raising my children with 21st century values for sun exposureplenty of high-SPF sunscreen, hats and time in the shadeso hopefully the next generation wont have the melanoma worries that my generation does.

If youve been diagnosed with advanced melanoma, heres what patient advocates recommend:

See a specialist

Seek a facility where doctors specialize in melanoma. Our recommendation for patients is to get into a melanoma center of excellence, says Bettina Ryll, founder of Melanoma Patient Network Europe in Uppsala, Sweden. The new immunotherapies have very different side effects from anything weve ever had before, so you dont want to have a physician who has never seen this.

Consider a clinical trial

Availability of immunotherapy and targeted therapy varies in Europe. Cost is a factor in many countries. Many patients enter clinical trials to receive these drugs. A promising clinical trial may be farther from home than youd prefer, but the extra drive could be worth it. Rory Bernard, 47, of Clermont-Ferrand, France, travels four hours to Paris for targeted therapy treatments, which have shrunk his tumors and extended his life. The dermatologist said, If you stay here, youre dead in six months, says Rorys wife, Gilly Spurrier-Bernard, founder of Melanoma France. My aim is to inform patients that if they want to get the best treatment, they may need to move around. Translation translation transl translation translation transl translation translation.

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First patient cured of rare blood disorder Science Daily The transplant technique is unique, because it allows a donor's cells to gradually take over a patient's bone marrow without using toxic agents to eliminate a patient's cells prior to the transplant. ... treatment options have been limited because they ... Stem Cell Transplant Cures a Man With Rare Blood DisorderNature World News Doctors cure first patient with rare blood disorderIANS all 3 news articles »

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Electroacupuncture releases stem cells to relieve pain, promote tissue repair, study finds Science Daily "The acupuncture stimulus we're giving these animals has a rapid effect on neuroanatomical pathways that connect the stimulus point in the arm to responsive neurons in the spinal cord and into a region in the brain called the hypothalamus. In turn, the ...

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Clinical Pain Advisor (registration)

Stem Cell Therapies for Degenerative Disc Disease Clinical Pain Advisor (registration) MSCs derived from bone marrow have been successfully differentiated into cardiopoietic cells and used in treatment of heart failure. Fourth- and fifth-generation techniques use genetically modified MSCs and induced pluripotent stem cells (iPSCs), ...

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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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Scientists at The University of Queensland have taken a significant step forward in cardiac disease research by creating a functional beating human heart muscle from stem cells.

Dr James Hudson and Dr Enzo Porrello from the UQ School of Biomedical Sciences collaborated with German researchers to create models of human heart tissue in the laboratory so they can study cardiac biology and diseases in a dish.

The patented technology enables us to now perform experiments on human heart tissue in the lab, Dr Hudson said.

This provides scientists with viable, functioning human heart muscle to work on, to model disease, screen new drugs and investigate heart repair.

The UQCardiac Regeneration Laboratoryco-leaders have also extended this research and shown that the immature tissues have the capacity to regenerate following injury.

In the laboratory we used dry ice to kill part of the tissue while leaving the surrounding muscle healthy and viable, Dr Hudson said.

We found those tissues fully recovered because they were immature and the cells could regenerate in contrast to what happens normally in the adult heart where you get a dead patch.

Our goal is to use this model to potentially find new therapeutic targets to enhance or induce cardiac regeneration in people with heart failure.

Studying regeneration of these damaged, immature cells will enable us to figure out the biochemical events behind this process.

Hopefully we can determine how to replicate this process in adult hearts for cardiovascular patients.

UQ scientists create beating human heart muscle from The University of Queensland on Vimeo.

Each year, about 54,000 Australians suffer a heart attack, with an average of about 23 deaths every day.

The UQ research has been supported by the National Health and Medical Research Council (NHMRC) and the National Heart Foundation.

Heart Foundation Queensland CEO Stephen Vines said the charity was excited to fund such an important research project.

Heart attack survivors who have had permanent damage to their heart tissue are essentially trying to live on half an engine, Mr Vines said.

The research by Dr Hudson and Dr Porello will help unlock the key to regenerating damaged heart tissue, which will have a huge impact on the quality of life for heart attack survivors.

Dr Hudson and Dr Porello are deserved recipients of our highest national research accolade the Future Leader Fellowship Award.

The research is published in Circulation and Development.

Media: Dr James Hudson, j.hudson@uq.edu.au; Kim Lyell, k.lyell@uq.edu.au, 0427 530 647.

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Scientists create 'beating' human heart muscle for cardiac research - UQ News

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