The Practicing Parent: Pros & cons of stem cell banking

Umbilical cord stem cell banking can be expensive and controversial, but Jamie and Ben Page decided to bank their daughter, Harlow's stem cells just in case.

"We had heard about cord blood banking and talked about it a lot and thought let's just go for it and have it just as a backup," Jamie Page said.

They did need it. Harlow had cancer in her uterus.

"On the ultrasound they immediately saw that there was a mass in her abdomen about the size of a grapefruit," Page said.

After a year of chemo, the tumor was gone. Doctors wanted to keep it that way.

"So, when the doctors found out we actually had her own stem cells, they were very excited," Page said.

Oncologist Elaine Morgan says those stem cells helped Harlow.

"I think that her umbilical cord cells were used as a boost to her own cells when we harvested her to have adequate cells for reconstitution," Dr. Morgan said.

Dr. Morgan does not advocate private cord stem cell banking at birth to be saved for a healthy baby's later use, because it's not clinically useful and it's expensive.

The Pages paid almost $2,000 for the initial banking fee, plus an extra $125 per year.

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The Practicing Parent: Pros & cons of stem cell banking

Stem cell guru walks out on Spanish science

A world leader in stem cell research is leaving the cutting-edge center he helped create in Barcelona due to a lack of support from the authorities in both Catalonia and Spain.

Juan Carlos Izpisa, a Spanish biochemist who teaches at the Salk Institute in California and has earned numerous awards for his research into organ and tissue development, had helped found the Barcelona Regenerative Medicine Center (CMRB) in 2004, bringing Spain to the forefront of stem cell research.

But now Izpisa is resigning from his director's post due to a lack of financial and political support from his two major public sponsors, the government of Spain and the regional government of Catalonia.

Sources in the scientific community are blaming the move on "the cuts, political mediocrity and a lack of empathy from Madrid." They also note that Catalan premier Artur Mas met with Izpisa 18 months ago and assured him that he would put all available means at his disposal.

Sources in the scientific community are blaming the move on "the cuts, political mediocrity and a lack of empathy from Madrid"

According to sources familiar with the months-long negotiations, the decision to let Izpisa go originated in the Catalan government, although Madrid did nothing to stop it. The explanation supplied was that, as an undesirable effect of cutbacks, the government was no longer in a condition to keep funding the center "at the quality levels required" by its director.

While his departure will not bring about the immediate closure of the center, the CMRB will lose many of its lines of research, since 18 of the 21 scientific projects it is currently carrying out are the intellectual property of Izpisa, who will take them with him.

The move also underscores how the crisis has meant further cuts for scientific projects in Spain, which is seeing many researchers move abroad to find jobs and financial support.

Shortly after the crisis hit Spain in 2008, the then-Socialist government announced a shift away from construction as the basis of the countrys economic growth. But so far this has failed to translate into any significant investment in other fields.

Over the last decade, the CMRB has published more than 200 papers, including some seminal work in the emerging field of regenerative medicine. One of Izpisa's projects, the development of "micro-kidneys" from stem cells, was described by Science magazine as one of the great advances of 2013.

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Stem cell guru walks out on Spanish science

Stem Cells Could Prove Effective in Treating Brittle Bone Disease

Durham, NC (PRWEB) January 14, 2014

A new study released in STEM CELLS Translational Medicine indicates that stem cells can be effective in treating a debilitating and sometimes lethal genetic disorder called brittle bone disease.

Brittle bone disease, or osteogenesis imperfecta (OI), is characterized by fragile bones causing some patients to suffer hundreds of fractures over the course of a lifetime. In addition, according to the OI Foundation, other symptoms include muscle weakness, hearing loss, fatigue, joint laxity, curved bones, scoliosis, brittle teeth and short stature. Restrictive pulmonary disease occurs in the more severe cases. Currently there is no cure.

OI can be detected prenatally by ultrasound. In the study reported on in STEM CELLS Translational Medicine, an international team of researchers treated two patients for the disease using mesenchymal stem cells (MSCs) while the infants were still in the womb, followed by stem cell boosts after they were born.

We had previously reported on the prenatal transplantation for the patient with OI type III, which is the most severe form in children who survive the neonatal period, said Cecilia Gtherstrm, Ph.D., of the Karolinska Institutet and Karolinska University Hospital, Stockholm, Sweden. She and Jerry Chan, M.D., Ph.D., of the Yong Loo Lin School of Medicine and National University of Singapore, and KK Womens and Childrens Hospital, led the study that also included colleagues from the United States, Canada, Taiwan and Australia.

The first eight years after the prenatal transplant, our patient did well and grew at an acceptable rate. However, she then began to experience multiple complications, including fractures, scoliosis and reduction in growth, so the decision was made to give her another MSC infusion. In the two years since, she has not suffered any more fractures and improved her growth.

She was even able to start dance classes, increase her participation in gymnastics at school and play modified indoor hockey, Dr. Gtherstrm added.

The second child, which was experiencing a milder form of OI, received a stem cell transfusion 31 weeks into gestation and did not suffer any new fractures for the remainder of the pregnancy or during infancy. She followed her normal growth pattern just under the third percentile in height until 13 months of age, when she stopped growing. Six months later, the doctors gave her another infusion of stem cells and she resumed growing at her previous rate.

Our findings suggest that prenatal transplantation of autologous stem cells in OI appears safe and is of likely clinical benefit and that re-transplantation with same-donor cells is feasible. However, the limited experience to date means that it is not possible to be conclusive, for which further studies are required, Dr. Chan said.

Although the findings are preliminary, this report is encouraging in suggesting that prenatal transplantation may be a safe and effective treatment for this condition, said Anthony Atala, M.D., editor of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine.

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Stem Cells Could Prove Effective in Treating Brittle Bone Disease

Keeping stem cells pluripotent

PUBLIC RELEASE DATE:

13-Jan-2014

Contact: Scott LaFee slafee@ucsd.edu 619-543-6163 University of California - San Diego

While the ability of human embryonic stem cells (hESCs) to become any type of mature cell, from neuron to heart to skin and bone, is indisputably crucial to human development, no less important is the mechanism needed to maintain hESCs in their pluripotent state until such change is required.

In a paper published in this week's Online Early Edition of PNAS, researchers from the University of California, San Diego School of Medicine identify a key gene receptor and signaling pathway essential to doing just that maintaining hESCs in an undifferentiated state.

The finding sheds new light upon the fundamental biology of hESCs with their huge potential as a diverse therapeutic tool but also suggests a new target for attacking cancer stem cells, which likely rely upon the same receptor and pathway to help spur their rampant, unwanted growth.

The research, led by principal investigator Karl Willert, PhD, assistant professor in the Department of Cellular and Molecular Medicine, focuses upon the role of the highly conserved WNT signaling pathway, a large family of genes long recognized as a critical regulator of stem cell self-renewal, and a particular encoded receptor known as frizzled family receptor 7 or FZD7.

"WNT signaling through FZD7 is necessary to maintain hESCs in an undifferentiated state," said Willert. "If we block FZD7 function, thus interfering with the WNT pathway, hESCs exit their undifferentiated and pluripotent state."

The researchers proved this by using an antibody-like protein that binds to FZD7, hindering its function. "Once FZD7 function is blocked with this FZD7-specific compound, hESCs are no longer able to receive the WNT signal essential to maintaining their undifferentiated state."

FZD7 is a so-called "onco-fetal protein," expressed only during embryonic development and by certain human tumors. Other studies have suggested that FZD7 may be a marker for cancer stem cells and play an important role in promoting tumor growth. If so, said Willert, disrupting FZD7 function in cancer cells is likely to interfere with their development and growth just as it does in hESCs.

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Keeping stem cells pluripotent

Medical Board Suspends Oregon Doctor For Stem Cell Treatments …

Oregon Medical Board has issued a rare emergency suspension of a Eugene physicians license after the doctor conducted experimental stem cell treatments on patients.

The board considers Dr. Kenneth Welkers medical practice an immediate danger to the public.

Welker can appeal the suspension, issued Thursday. He did not return calls from The Associated Press on Friday, nor did the clinic at which hes employed, Oregon Optimal Health.

According to his online biography, Welker is a trained surgeon who quit his practice to pursue alternative medicine in 2007.

In May 2013, the boards suspension order says Welker injected processed stem cells into the spine of a 62-year-old woman, and was confused when she began to sweat and feel tingling in her extremities.

Stem cells, unlike other cells in the body, have two distinct characteristics. They can renew themselves through cell division, and they are not specialized in the way that muscle cells or brain cells are. Under certain conditions, they can be induced to transform into organ- or tissue-specific cells.

In 1998, researchers discovered how to derive stem cells from human embryos, and in 2006, they determined how to induce some specialized adult cells to take on the genetic characteristics of stem cells. These are called induced pluripotent stem cells, or iPSC.

iPSC have long been used to treat cancers such as leukemia and lymphoma its what doctors are using when they do bone marrow transplants. The cells are being studied for everything from heart disease to diabetes, but its too soon to know if these approaches are safe or effective.

Advocates of alternative medicine have heaped praise on the possibility of using iPSC to treat a variety of maladies. Texas Gov. Rick Perry, for instance, had stem cells taken from fat in his own body, grown in a lab and then injected into his back and his bloodstream during a 2011 operation to fuse part of his spine.

But scientists have questioned the safety and wisdom of Perrys treatment, especially because it was not part of a clinical trial in which unproven therapies are tested in a way that helps protect patients and advances medical knowledge.

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Medical Board Suspends Oregon Doctor For Stem Cell Treatments ...

Stem Cell Treatment for Arthritis

Stem Cell Treatment for Arthritis

Mesenchymal stem cells have been shown in recent studies to have significant effects on a variety of conditions including both rheumatoid arthritis and osteoarthritis. As the mechanisms behind these two forms of arthritic degeneration differ, the potential stem cell treatment for arthritis is likely to be administered differently and make use of a variety of properties of stem cells, such as their regenerative abilities, and the capacity for some stem cell types to help regulate immune function. Patients undergoing stem cell treatments for unrelated conditions have reported significant relief from their arthritis after such therapy even though this was not the reason for them obtaining treatment initially; these stories are anecdotal however, and do not constitute evidence for applying treatment more widely. Intravenous stem cell therapy using haematopoietic stem cells has been used extensively in veterinary medicine for a number of years. Often, stem cell treatment for arthritis in companion animals and race horses with signs of the condition (both rheumatoid and osteoarthritis) direct injections of stem cells into the damaged joint have proven effective at inhibiting the autoimmune attacks consistent with RA, reducing inflammation and pain in the joint, and supporting active tissue regeneration.

Stem Cell Treatment for Arthritis brings hope to millions suffering from arthritic conditions

The conditions under which stem cells are implanted appear to make all the difference between active regeneration and hypertrophy of joint tissue. Researchers are wary of the ad hoc use of stem cells for joint repair as it may be that the growth encouraged by stem cells may be adversely influenced by pre-existing pathology in an arthritis patients joints leading to exaggerated tissue growth that may further exacerbate the problems. Using a small number of chondrocytes alongside mesenchymal stem cells may make a significant difference in cultivating healthy cartilage repair without hypertrophy (excessive growth) occurring (Aung, et al, 2011).

A further study by Abedi (et al, 2010) appears to show that the use of scaffolding material alongside mesenchymal stem cell administration improves the healing process in induced articular cartilage defects in animal models (rabbits) in comparison to the stem cells alone. By encouraging differentiation into cartilage tissue types the almost universal progression of articular cartilage damage to osteoarthritis could, theoretically, be prevented using stem cell therapy. MSCs from osteoarthritis patients used in research has also led to the development of intelligent surfaces which can effectively prevent hypertrophy of such stem cells in the laboratory whilst facilitating cartilage regrowth (Petit, et al, 2011); further research in vitro is required to assess their use for therapeutic purposes however. A review of current research into synoviocytes and chondrogenesis has also highlighted a potential source of stem cells which may actively aid repair of avascular meniscal injuries that are otherwise unresponsive to treatment and commonly lead to osteoarthritis (Fox, et al, 2011).

Mesenchymal stem cells, which can differentiate into bone, cartilage, and a number of other cell types appear to hold great potential for easing osteoarthritis joint pain and possibly regenerating damaged tissue. However, MSC therapy alone is unlikely to address the systemic issue of autoimmune rheumatoid arthritis. Just as haematopoietic stem cells can help combat leukaemia, their use following myeloablation (the destruction of the bodys immune-forming cells in the bone marrow) looks promising for treatment of RA (Sykes, et al, 2005).

A study by Bhattacharya (et al, 2001) to test the safety of using umbilical cord blood for those with a variety of conditions found that the transfusions were well tolerated by all 62 of the patients and that the cord blood had the advantages of a higher oxygen carrying capacity, many growth factors and cytokines, and was also less likely to cause an immune reaction than adult whole blood transfusions. The study did not aim to assess the potential benefits of the stem cell treatment, only the safety of such procedures for those with rheumatoid arthritis, arthritis in the neck, ankylosing spondylitis, and systemic lupus erythematosus, amongst other conditions.

The ability to derive stem cells from patients themselves is also opening up a whole new realm of possible treatments which do not require the use of human embryonic stem cells or cord blood stem cells which are less readily available and mired in some ethical controversy. Autologous stem cell transplants are also advantageous as they do not trigger an immune response causing rejection of the material in the body; immunosuppressant drugs are, therefore, not necessary. Jorgensen (et al, 2004) stated that mesenchymal stem cells appeared to be good candidates for the regeneration of arthritic tissue and that more research was required to assess their viability. This research has been carried out in subsequent years by a whole host of scientists such as Mao (2005), Gonzlez (et al, 2009), and Tyndall (et al, 2010).

Some studies have looked at animal models with induced rheumatoid arthritis and their reaction to mesenchymal stem cell transplantation. Across a number of studies the effects have been positive, with the Th1-induced autoimmune response down-regulated by the stem cell treatment. Human adipose-derived mesenchymal stem cells also decreased inflammatory cytokines and chemokines in the mouse models and actively increased the production of antiinflammatory substance interleukin-10 in lymph nodes and joints. A, perhaps unexpected, benefit of human AD-MSCs was the de novo generation of antigen-specific CD4+CD25+FoxP3+ Treg cells, which were then able to suppress those immune system cells which react against the bodys own tissue.

According to Passweg and Tyndall (2007), more than a 1000 patients with autoimmune diseases have been treated with haematopoietic stem cells between 1996 and 2007. Most of these patients had Multiple Sclerosis, systemic lupus erythematosus, RA, or systemic sclerosis and many of those treated have enjoyed long-term disease-free remissions and immune reconstitution since treatment. Unfortunately, there remains a risk of treatment related mortality with such stem cell therapy as it relies on the destruction initially of the patients immune system in order to reset it with the infused stem cells and remove the autoimmune components. This temporarily opens the patients up to increased risks of infection which can be fatal. Improvements in patient care during the treatments have reduced this risk substantially, but it is still a major consideration, particularly for those otherwise doing well on conventional medications. Tyndall and Laar (2010) found that incomplete, low immunoablative intensity, early conditioning was related to patient relapse. This is most likely due to residual lesional effector cells; the patients faulty immune system effectively repopulated itself with self-reactive immune cells when only partially destroyed by initial myeloablative treatment.

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Stem Cell Treatment for Arthritis

Patients now endorsing key stem cell research

New York, Jan 6 : Despite some ethical concerns, most patients are now broadly endorsing stem cell research.

In the case of induced pluripotent stem cells (iPSCs), which are stem cells made from skin or other tissues, researchers at the Johns Hopkins University found patients were largely in favour of participating in iPSC research even if personal benefit was unlikely.

The patients, however, raised concerns about consent, privacy and transparency.

"Bioethicists as well as stem cell researchers and policy-makers have discussed ethical issues at length but till date, we didn't have any systematic information about what patients think about these issues," said Jeremy Sugarman, the Harvey M. Meyerhoff professor of bioethics and medicine at Johns Hopkins Berman Institute of Bioethics.

Unlike human embryonic stem cells, iPSCs are derived without destroying a human embryo. Research with human iPSCs is valuable for developing new drugs, studying disease, and perhaps developing medical treatments, said the study published in the journal Cell Stem Cell.

According to the study, consent was highly important for patients. Some patients even suggested that proper informed consent could compensate for other concerns they had about privacy, the "immortalisation" of cells and the commercialisation of stem cells.

There was a "strong desire among participants to have full disclosure of the anticipated uses, with some participants wanting to be able to veto certain uses of their cells", the study added.

"The idea that donated cells would potentially live forever was unnerving to some participants," the report stated.

"This study is a first step in getting crucial information about what values are factored into a decision to participate in iPSC research, and what those participants expect from the experience," said Sugarman.

--IANS (Posted on 06-01-2014)

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Patients now endorsing key stem cell research

Patients endorse key stem cell research

Despite some ethical concerns, most patients are now broadly endorsing stem cell research.

In the case of induced pluripotent stem cells (iPSCs), which are stem cells made from skin or other tissues, researchers at the Johns Hopkins University found patients were largely in favour of participating in iPSC research even if personal benefit was unlikely.

The patients, however, raised concerns about consent, privacy and transparency.

"Bioethicists as well as stem cell researchers and policy-makers have discussed ethical issues at length but till date, we didn't have any systematic information about what patients think about these issues," said Jeremy Sugarman, the Harvey M. Meyerhoff professor of bioethics and medicine at Johns Hopkins Berman Institute of Bioethics.

Unlike human embryonic stem cells, iPSCs are derived without destroying a human embryo. Research with human iPSCs is valuable for developing new drugs, studying disease, and perhaps developing medical treatments, said the study published in the journal Cell Stem Cell.

According to the study, consent was highly important for patients. Some patients even suggested that proper informed consent could compensate for other concerns they had about privacy, the "immortalisation" of cells and the commercialisation of stem cells.

There was a "strong desire among participants to have full disclosure of the anticipated uses, with some participants wanting to be able to veto certain uses of their cells", the study added.

"The idea that donated cells would potentially live forever was unnerving to some participants," the report stated.

"This study is a first step in getting crucial information about what values are factored into a decision to participate in iPSC research, and what those participants expect from the experience," said Sugarman.

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Patients endorse key stem cell research

Stanford gets $90 million cancer study grant

By Lisa M. Krieger lkrieger@mercurynews.com

Stanford has received a vast sum of money to study a tiny population of deadly cancer cells, a gift that could help combat the heartbreak of phoenixlike disease recurrence.

The $90 million from the New York City-based Ludwig Fund will boost research at the Ludwig Center for Cancer Stem Cell Research and Medicine at Stanford's School of Medicine, where scientists are studying cancer stem cells for ways to tear out the roots of tumors.

Stanford is one of six institutions to share in Ludwig's $540 million contribution to the field of cancer research, announced Monday. Endowed by the late billionaire Daniel K. Ludwig, a self-made American shipping and real estate magnate who died in 1992, the fund supports cancer research that might be otherwise dependent on the whims of government or corporate support.

"This extraordinary gift will spur innovation well into the future," said Stanford president John Hennessy, calling it "a tremendous vote of confidence."

Billions of dollars have been spent on cancer research since President Richard M. Nixon declared war on the disease in 1971. Yet the fight is going slower than most had hoped, with small changes in the death rate over the decades since.

Part of the problem, scientists think, it that some cancers are driven by hidden cancer stem cells -- which remain tenacious even after treatment, reasserting themselves and continuing to grow. Their discovery by Canadian scientist John Dick in 1994 profoundly altered our concept of cancer biology.

If such cells are proven to be the determinant of relapse, the implication for cancer therapy is enormous. Any treatment that leaves behind residual cancer stem cells would inevitably lead to a relapse.

"These are the subset of cells that self-renew -- they're the dangerous one," said Dr. Irving Weissman, who directs Stanford's Ludwig Center, the only cancer stem cell center of its kind.

Weissman and Dr. Michael Clarke have isolated these cells in many different types of cancers and identified ways they might be vulnerable.

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Stanford gets $90 million cancer study grant

Solution to problem limiting stem cell therapies

Biologists at UC San Diego have discovered an effective strategy that could prevent the human immune system from rejecting the grafts derived from human embryonic stem cells, a major problem now limiting the development of human stem cell therapies. Their discovery may also provide scientists with a better understanding of how tumors evade the human immune system when they spread throughout the body.

The achievement, published in a paper in this week's early online edition of the journal Cell Stem Cell by a collaboration that included scientists from China, was enabled by the development of "humanized" laboratory mice that contained a functional human immune system capable of mounting a vigorous immune rejection of foreign cells derived from human embryonic stem cells.

Because human embryonic stem cells are different from our own body's cells, or "allogenic," a normally functioning human immune system will attack these foreign cells. One way to reduce the body's "allogenic immune response" is to suppress the immune system with immunosuppressant drugs.

"For organ transplantation to save patients with terminal diseases that has been quite successful," says Yang Xu, a professor of biology who headed the team of researchers that included Ananda Goldrath, an associate biology professor at UC San Diego. "But for stem cell therapies, the long term use of toxic immunosuppressant drugs for patients who are being treated for chronic diseases like Parkinson's disease or diabetes pose serious health problems."

Researchers had long been searching for a human immunity relevant model that would allow them to develop strategies to implant allogenic cells derived from embryonic stem cells safely. "The problem is that we only had data from mouse immune system and those are not usually translatable in humans, because human and mouse immune systems are quite different," explains Xu. "So what we decided to do was to optimize the humanized mouse that carries a functional human immune system."

To do that, the biologists took immune deficient laboratory mice and grafted into their bodies human fetal thymus tissues and hematopoietic stem cells derived from fetal liver of the same human donor. "That reconstituted in these mice a normally functioning human immune system that effectively rejects cells derived human embryonic stem cells," says Xu. With these "humanized" mouse models, the biologists then tested a variety of immune suppressing molecules alone or in combination and discovered one combination that worked perfectly to protect cells derived from human embryonic stem cells from immune rejection.

That combination was CTLA4-lg, an FDA-approved drug for treating rheumatoid arthritis that suppresses T-cells responsible for immune rejection, and a protein called PD-L1 known to be important for inducing immune tolerance in tumors. The researchers discovered that the combination of these two molecules allowed the allogeneic cells to survive in humanized mice without triggering an immune rejection.

"If we express both molecules in cells derived from human embryonic cells, we can protect these cells from the allogenic immune rejection," says Xu. "If you have only one such molecule expressed, there is absolutely no impact. We still don't know exactly how these pathways work together to suppress immune rejection, but now we've got an ideal system to study this."

He and his team of researchers also believe their discovery and the development of their humanized mouse models may offer the much needed tools to develop ways to activate immune response to tumors, because these molecules are known to be important in allowing tumors to evade the human immune system.

"You're dealing with the same exact pathways that protect tumors from our immune system," says Xu. "If we can develop strategies to disrupt or silence these pathways in tumors, we might be able to activate immunity to tumors. The humanized mouse system is really a powerful model with which to study human tumor immunity."

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Solution to problem limiting stem cell therapies