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Robin Roberts says her prayers have been answered as she recovers from bone marrow transplant

By LizaAVILA

By Daily Mail Reporter

PUBLISHED: 14:58 EST, 4 October 2012 | UPDATED: 16:07 EST, 4 October 2012

Good Morning America co-anchor Robin Roberts has revealed that her bone marrow transplant, which she underwent two weeks ago, appears to have been successful.

The procedure, which saw donor stem cells from her sister Sally Ann injected into her body, took just five minutes, and according to the 51-year-old, who wrote fans an update from her hospital in New York City this morning, her sister's cells 'feel right at home' in her body.

'My blood counts are GREAT,' she wrote on herGMA blog, after being hospitalized or 25 days now. 'It's an answer to so many prayers'.

Scroll down for video

Pulling through: Robin Roberts, 51, said her friends near and far (pictured here with Sam and Josh yesterday) have been lifting her spirits, she says

Ms Roberts, who was diagnosed with myelodysplastic syndrome, or MDS, earlier this year - a disease which attacks blood cells and bone - added: 'My doctors and rock star nurses are very pleased with my progress and I could not be more thankful for the excellent care I am receiving.

'I have had some extremely painful days and its still difficult for me to eat because of all the chemo. [But] I continue to learn so much on this journey, especially when it comes to true friendship and love.

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College student answers growing need for bone marrow transplants

By raymumme

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DURHAM -- With lives on the line, the need for bone marrow donations across the country is greater than ever.

The National Marrow Donor Program said just five out of 10 patients will receive the transplant they need survive.

Elon University student Donovan Rainey recently passed the exam of a lifetime. He's a donor match for a patient in need of a bone marrow transplant.

"To be able to give life and to be able to try to sustain someone else's is just the ultimate gift," said Rainey.

Giving that gift is easier than before.

Duke University Medical Center said many are under the false impression that the only way to donate is by surgericaly removing bone marrow from the hip.

Instead, donors can get blood removed through a machine. The stem cells found in donors blood will be used to create a new immune system for recipients.

"They don't need general anesthesia, they don't have to go to the operating room and I think there is less discomfort," said Susan Dago, a nurse at Duke's Blood and Marrow Transplant Clinic and Treatment Facility.

Rainey said the temporary discomfort is worth it because the life on the line was his dad's.

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Study Shows Epigenetics Of Adult Stem Cells Influences Organ Creation

By raymumme

October 3, 2012

Alan McStravick for redOrbit.com Your Universe Online

Ask a handful of people about their thoughts and feelings on the use of stem cells for research and therapeutic means and you will find that they each have strong and varying positions on the topic. Outside the scientific community, however, little is known about this highly complex field of research.

The politicization of stem cell research accompanied the 1998 discovery that embryonic stem cells, the building blocks of organ, tissue, bone and brain cells, could be extracted for study and medical use. In 2001, with an order to limit the lines of stem cell research to those already in possession of the scientific community, President George W. Bush largely hampered the development of this field in the United States by limiting government funding for stem cell research. Adult stem cells, or somatic stem cells, were unaffected by this order, but the prevailing wisdom of the genetic community was that adult stem cells were not as dynamic and couldnt be used in the same way as their embryonic cousins.

With a report published Monday in the American Journal of Pathology, that truth no longer seems to be the case. A team led by Manel Esteller, director of the Cancer Epigenetics and Biology Program in the Bellvitge Biomedical Research Institute (IDIBELL), was able to identify epigenetic changes that occur in the somatic stem cells to generate different body tissues.

The use of somatic or adult stem cells had been a regular occurrence since their discovery in the 1950s. It was then that researchers found that bone marrow contains two different kinds of stem cells. The first, called hematopoietic stem cells, form all the types of blood cells in the body. The second, known as bone marrow stromal stem cells, were discovered only a few years later and are effective in the generation of bone, cartilage, fat and fibrous connective tissues.

One thing that has been understood is that the genome of each cell in the human body is identical. This is true regardless of their appearance and function. It is for this reason that certain anomalies, such as cancer, are seemingly incomprehensible as they are unable to be explained by the genome of the host. To better understand such complex genetic deviations, something more is required.

Researchers in this current study offer an explanation via analogy. Epigenetics is defined as the inheritance of DNA activity that does not depend on the strict sequence of it. According to the team, if genetics is the alphabet, spelling would be the epigenetics, referring to chemical changes in our genetic material as well as the proteins that regulate and control their activity.

We now know that somatic stem cells have enormous potential to regenerate damaged organs. By investigating how to use them more effectively in different types of therapies, the research team postulates that it will become easier to steer clear of any sticky ethical complications that might arise from working with embryonic stem cells.

In this study, the team was able to isolate somatic stem cells from body fat, allowing them to transform them into muscle and bone cells. Through their study, they observed the resemblance of the cells created in the laboratory to those of the host individual. They were also able to determine that the cells were biologically secure enough that they might be implanted into waiting patients. Overall, the study was able to show that the epigenome of the cells obtained and maintained in culture closely resembled skeletal and muscle cells that are spontaneously present in nature, though not completely identical.

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Bio-Matrix Scientific Group's Regen BioPharma Subsidiary Announces HemaXellerate™ As First Product in Development

By raymumme

Company seeks to enter $18 billion market for bone marrow stimulating growth factors

San Diego, CA (PRWEB) October 03, 2012

As part of the development process, on May 18, 2012, Regen submitted a provisional patent application covering the use of placentally-derived endothelial cells for treatment of bone marrow failure. Regen has also been granted an exclusive option to enter into an agreement to be granted an exclusive, worldwide, royalty bearing license to US patent No. 6,821,513, covering a proprietary method for enhancing hematopoiesis (formation of blood cells).

Current approaches to treating bone marrow disorders involve administration of pharmaceuticals which target stem cells to produce more blood. This approach is not effective on everyone with bone marrow failure and some forms of this disease are completely resistant said J. Christopher Mizer, President of Regen BioPharma. Our strategy is to heal the bone marrow by administering cells that provide the optimum mix of growth factors to stimulate the bone marrow into producing blood cells naturally.

Data from a peer reviewed publication (Lei et al. Stem Cell Res. 2010 January; 4(1): 1724) by the inventor of the patent demonstrated that the administration of endothelial cells restores blood production and extends survival after bone marrow damage.

The HemaXellerate product aims to address the unmet medical need of patients who are non-responsive to existing growth factor therapies such as Neupogen and Leukine. These patients include those suffering from: aplastic anemia, a condition where the bone marrow produces an insufficient number of new cells to replace lost blood cells; chemotherapy/radiotherapy induced bone marrow failures; and low blood cell production after bone marrow or cord blood transplants, stated Thomas Ichim, Chief Scientific Officer of Regen BioPharma.

According to David Koos, Chairman & CEO of Bio-Matrix, HemaXellerate may provide an ideal therapeutic for bone marrow failure based upon: (1) regulating secretion of cytokines as biologically needed; (2) producing long-term, localized growth factors that alleviate the need for drugs; and (3) actively repairing the blood producing stem cell environment.

A spokesperson for the Company said Regen intends to file an Investigational New Drug (IND) Application in the fourth quarter of 2012 and conduct Phase I/II clinical trials during 2013 and 2014.

About Bio-Matrix Scientific Group Inc. and Regen BioPharma, Inc.:

Bio-Matrix Scientific Group, Inc. (OTCQB: BMSN) (PINKSHEETS: BMSN) is a biotechnology company developing regenerative medicine therapies and tools. The Company is focused on human therapies that address unmet medical needs. Specifically, Bio-Matrix Scientific Group Inc. is looking to increase the quality of life through therapies involving stem cell treatments. These treatments are focused in areas relating to cardiovascular, hematology, oncology and other indications.

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Bio-Matrix Scientific Group's Regen BioPharma Subsidiary Announces HemaXellerate™ As First Product in Development

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Study Sheds Light on Bone Marrow Stem Cell Therapy for Pancreatic Recovery

By NEVAGiles23

Newswise LOS ANGELES (Oct. 2, 2012) Researchers at Cedars-Sinais Maxine Dunitz Neurosurgical Institute have found that a blood vessel-building gene boosts the ability of human bone marrow stem cells to sustain pancreatic recovery in a laboratory mouse model of insulin-dependent diabetes.

The findings, published in a PLoS ONE article of the Public Library of Science, offer new insights on mechanisms involved in regeneration of insulin-producing cells and provide new evidence that a diabetics own bone marrow one day may be a source of treatment.

Scientists began studying bone marrow-derived stem cells for pancreatic regeneration a decade ago. Recent studies involving several pancreas-related genes and delivery methods transplantation into the organ or injection into the blood have shown that bone marrow stem cell therapy could reverse or improve diabetes in some laboratory mice. But little has been known about how stem cells affect beta cells pancreas cells that produce insulin or how scientists could promote sustained beta cell renewal and insulin production.

When the Cedars-Sinai researchers modified bone marrow stem cells to express a certain gene (vascular endothelial growth factor, or VEGF), pancreatic recovery was sustained as mouse pancreases were able to generate new beta cells. The VEGF-modified stem cells promoted growth of needed blood vessels and supported activation of genes involved in insulin production. Bone marrow stem cells modified with a different gene, PDX1, which is important in the development and maintenance of beta cells, resulted in temporary but not sustained beta cell recovery.

Our study is the first to show that VEGF contributes to revascularization and recovery after pancreatic injury. It demonstrates the possible clinical benefits of using bone marrow-derived stem cells, modified to express that gene, for the treatment of insulin-dependent diabetes, said John S. Yu, MD, professor and vice chair of the Department of Neurosurgery at Cedars-Sinai, senior author of the journal article.

Diabetes was reversed in five of nine mice treated with the injection of VEGF-modified cells, and near-normal blood sugar levels were maintained through the remainder of the six-week study period. The other four mice survived and gained weight, suggesting treatment was beneficial even when it did not prompt complete reversal. Lab studies later confirmed that genetically-modified cells survived and grew in the pancreas and supported the repopulation of blood vessels and beta cells.

Anna Milanesi, MD, PhD, working in Yus lab as an endocrinology fellow, is the articles first author. The researchers cautioned that although this and other related studies help scientists gain a better understanding of the processes and pathways involved in pancreatic regeneration, more research is needed before human clinical trials can begin.

Insulin-dependent diabetes occurs when beta cells of the pancreas fail to produce insulin, a hormone that regulates sugar in the blood. Patients must take insulin injections or consider transplantation of a whole pancreas or parts of the pancreas that make insulin, but transplantation carries the risk of cell rejection.

# # #

PLoS ONE: Beta-cell Regeneration Mediated by Human Bone Marrow Mesenchymal Stem Cells.

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Cedars-Sinai study sheds light on bone marrow stem cell therapy for pancreatic recovery

By LizaAVILA

Public release date: 2-Oct-2012 [ | E-mail | Share ]

Contact: Sandy Van sandy@prpacific.com 808-526-1708 Cedars-Sinai Medical Center

LOS ANGELES (Oct. 2, 2012) Researchers at Cedars-Sinai's Maxine Dunitz Neurosurgical Institute have found that a blood vessel-building gene boosts the ability of human bone marrow stem cells to sustain pancreatic recovery in a laboratory mouse model of insulin-dependent diabetes.

The findings, published in a PLOS ONE article of the Public Library of Science, offer new insights on mechanisms involved in regeneration of insulin-producing cells and provide new evidence that a diabetic's own bone marrow one day may be a source of treatment.

Scientists began studying bone marrow-derived stem cells for pancreatic regeneration a decade ago. Recent studies involving several pancreas-related genes and delivery methods transplantation into the organ or injection into the blood have shown that bone marrow stem cell therapy could reverse or improve diabetes in some laboratory mice. But little has been known about how stem cells affect beta cells pancreas cells that produce insulin or how scientists could promote sustained beta cell renewal and insulin production.

When the Cedars-Sinai researchers modified bone marrow stem cells to express a certain gene (vascular endothelial growth factor, or VEGF), pancreatic recovery was sustained as mouse pancreases were able to generate new beta cells. The VEGF-modified stem cells promoted growth of needed blood vessels and supported activation of genes involved in insulin production. Bone marrow stem cells modified with a different gene, PDX1, which is important in the development and maintenance of beta cells, resulted in temporary but not sustained beta cell recovery.

"Our study is the first to show that VEGF contributes to revascularization and recovery after pancreatic injury. It demonstrates the possible clinical benefits of using bone marrow-derived stem cells, modified to express that gene, for the treatment of insulin-dependent diabetes," said John S. Yu, MD, professor and vice chair of the Department of Neurosurgery at Cedars-Sinai, senior author of the journal article.

Diabetes was reversed in five of nine mice treated with the injection of VEGF-modified cells, and near-normal blood sugar levels were maintained through the remainder of the six-week study period. The other four mice survived and gained weight, suggesting treatment was beneficial even when it did not prompt complete reversal. Lab studies later confirmed that genetically-modified cells survived and grew in the pancreas and supported the repopulation of blood vessels and beta cells.

Anna Milanesi, MD, PhD, working in Yu's lab as an endocrinology fellow, is the article's first author. The researchers cautioned that although this and other related studies help scientists gain a better understanding of the processes and pathways involved in pancreatic regeneration, more research is needed before human clinical trials can begin.

Insulin-dependent diabetes occurs when beta cells of the pancreas fail to produce insulin, a hormone that regulates sugar in the blood. Patients must take insulin injections or consider transplantation of a whole pancreas or parts of the pancreas that make insulin, but transplantation carries the risk of cell rejection.

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New study sheds light on bone marrow stem cell therapy for pancreatic recovery

By raymumme

ScienceDaily (Oct. 2, 2012) Researchers at Cedars-Sinai's Maxine Dunitz Neurosurgical Institute have found that a blood vessel-building gene boosts the ability of human bone marrow stem cells to sustain pancreatic recovery in a laboratory mouse model of insulin-dependent diabetes.

The findings, published in a PLoS ONE article of the Public Library of Science, offer new insights on mechanisms involved in regeneration of insulin-producing cells and provide new evidence that a diabetic's own bone marrow one day may be a source of treatment.

Scientists began studying bone marrow-derived stem cells for pancreatic regeneration a decade ago. Recent studies involving several pancreas-related genes and delivery methods -- transplantation into the organ or injection into the blood -- have shown that bone marrow stem cell therapy could reverse or improve diabetes in some laboratory mice. But little has been known about how stem cells affect beta cells -- pancreas cells that produce insulin -- or how scientists could promote sustained beta cell renewal and insulin production.

When the Cedars-Sinai researchers modified bone marrow stem cells to express a certain gene (vascular endothelial growth factor, or VEGF), pancreatic recovery was sustained as mouse pancreases were able to generate new beta cells. The VEGF-modified stem cells promoted growth of needed blood vessels and supported activation of genes involved in insulin production. Bone marrow stem cells modified with a different gene, PDX1, which is important in the development and maintenance of beta cells, resulted in temporary but not sustained beta cell recovery.

"Our study is the first to show that VEGF contributes to revascularization and recovery after pancreatic injury. It demonstrates the possible clinical benefits of using bone marrow-derived stem cells, modified to express that gene, for the treatment of insulin-dependent diabetes," said John S. Yu, MD, professor and vice chair of the Department of Neurosurgery at Cedars-Sinai, senior author of the journal article.

Diabetes was reversed in five of nine mice treated with the injection of VEGF-modified cells, and near-normal blood sugar levels were maintained through the remainder of the six-week study period. The other four mice survived and gained weight, suggesting treatment was beneficial even when it did not prompt complete reversal. Lab studies later confirmed that genetically-modified cells survived and grew in the pancreas and supported the repopulation of blood vessels and beta cells.

Anna Milanesi, MD, PhD, working in Yu's lab as an endocrinology fellow, is the article's first author. The researchers cautioned that although this and other related studies help scientists gain a better understanding of the processes and pathways involved in pancreatic regeneration, more research is needed before human clinical trials can begin.

Insulin-dependent diabetes occurs when beta cells of the pancreas fail to produce insulin, a hormone that regulates sugar in the blood. Patients must take insulin injections or consider transplantation of a whole pancreas or parts of the pancreas that make insulin, but transplantation carries the risk of cell rejection.

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FDA Approves Stemedica Phase II Clinical Trial For Acute Myocardial Infarction With Ischemia Tolerant Mesenchymal Stem …

By Dr. Matthew Watson

Stemedica Cell Technologies, Inc., a leader in adult allogeneic stem cell manufacturing, research and development, announced today that the U.S. Food and Drug Administration (FDA) approved its application for an Investigational New Drug (IND) to assess the clinical effects of Stemedyne-MSC (Stemedicas human bone marrow-derived ischemia tolerant mesenchymal cells) in subjects with a myocardial infarct.

San Diego, CA (PRWEB) October 02, 2012

The clinical trial will address the prevalence of cardiovascular disease estimated to carry a global disease burden in excess of $400 billion each year. More than one million patients undergo PTCA and stenting in the Untied States annually; another 800,000 have the procedures each year in Europe.

Nabil Dib, M.D., MSc., F.A.C.C., Director of Cardiovascular Research at Mercy Gilbert and Chandler Regional Medical Centers, and an Associate Professor of Medicine and Director of Clinical Cardiovascular Cell Therapy at the University of California, San Diego, will serve as the principal investigator of the FDA-approved study. Dr. Nib commented, We've learned from bench top research that not all stem cells are created equally. We believe that the ischemic tolerance of Stemedica's MSCs and the robustness of their protein array will translate into significant patient benefits post myocardial infarction.

Stemedicas interest in this indication was triggered by a successful randomized study in acute myocardial infarction conducted by the National Scientific Medical Center (NSMC) in Astana, Kazakhstan using Stemedyne-MSCs. The study was conducted under clinical protocol and in compliance with the ICH-E6 (Good Clinical Practice) guidelines and local laws. All patients signed an informed consent. Nineteen (19) patients in this study received Stemedyne-MSCs after PTCA and stenting. Administration of Stemedyne-MSC resulted in a statistically-significant decrease in inflammation as judged by the level of C-reactive protein, significant decrease in end-systolic and end-diastolic volume of left ventricle, as well as significant increase in the left ventricular ejection fraction (LVEF) from 38.4% to 54.7% at 6 months post administration, bringing this parameter to a normal range for healthy individuals (50-65%).

Professor Daniyar Jumaniyazov, M.D. Ph.D., principal investigator of the NSMC study commented, The stem cell transplantation was safe and the procedure was well tolerated. No product-related adverse events were reported. Treatment of patients in this study resulted in improvement of overall and local contractive myocardium functions and also normalization of systolic and diastolic filling of the left ventricle as compared to the control group. Based upon the safety and efficacy results, we will soon conduct a Phase III myocardial infarct clinical trial at the NSMC with Stemedicas ischemia tolerant mesenchymal stem cells.

Lev Verkh, Ph.D., Stemedica Chief Regulatory and Clinical Development Officer commented, Stemedicas FDA submission included data from the NSMC clinical trial, the results of which were also reported at the annual American College of Cardiology meeting in April, 2012. These results contrasted with reports, at the same conference, of minimal improvement in studies with autologous stem cells. In addition to the United States sites, the study will be duplicated at leading hospitals in Europe, Asia and the Middle East. With regard to the spectrum of stem cell treatment for cardiovascular disease, Dr. Verkh noted that, Stemedyne-MSC has been approved for the treatment of chronic heart failure at Hospital Angeles, Tijuana, Mexico by COFEPRIS (the Mexican equivalent of the FDA).

Jackie See, M.D., F.A.C.C., founder of interventional cardiology at the University of California, Irvine, noted, "In the days and weeks following a myocardial infarction we may have the ability to intervene with stem cells to minimize scarring, enhance the amount of functional heart tissue, and restore the microcirculation. Stemedica's ischemia tolerant mesenchymal stem cells are ideal for this purpose. I can foresee the day when all coronary stenting is accompanied by stem cell injection. It is not unreasonable to postulate that the anti-inflammatory and anti-fibrotic effects of the mesenchymal stem cells may have an impact on the incidence of restenosis, a common condition caused by blockage of the stents.

The Stemedyne-MSC product is uniquely manufactured to contain increased amounts of the important growth factors that combat ischemic damage. According to Nikolai Tankovich, M.D., Ph.D., President and Chief Medical Officer of Stemedica, Our ischemia tolerant MSCs secrete increased amounts of vascular endothelial growth factor (VEGF), which is necessary for new blood vessel development and stromal cell-derived factor (SDF), which is responsible for rescuing dying cells. Stemedyne-MSCs also demonstrate significantly higher migratory abilities. As a company we are unique in our unparalleled scalability, with our master bank at two passages and the cells that go into patients having only been expanded four times. We have the ability to treat more than 500,000 patients with cells created from a single organ donation.

Stemedyne-MSC is one of the three adult allogeneic stem cell products developed by the Company. Other products include Stemedyne-NSC neural human stem cells and Stemedyne-RPE, retinal progenitor epithelial cells available in early 2013. All Stemedica products are unique in their ability to tolerate ischemic conditions.

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FDA Approves Stemedica Phase II Clinical Trial For Acute Myocardial Infarction With Ischemia Tolerant Mesenchymal Stem ...

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Bone marrow donor meets recipient

By JoanneRUSSELL25

DENVER - Can you imagine having a family member who's diagnosed with a disease and no one in your family, including yourself, is a match to donate.

That's the reality for 70 percent of patients needing bone marrow or stem cells. They have no other choice but to go through the bone marrow registry.

Those families rely on complete strangers who are willing to donate whatever they can in hope of saving someone's life.

One of those donors is Aurora resident Denise Camacho. She joined the bone marrow registry never thinking that anything would ever come of it.

"I have a family friend that works with Bonfils," Camacho said. "She emailed me and my family and said there's a huge need for minorities to join the registry. So we went down not knowing anyone of us would ever be called."

But just two years later, she was called to make a donation.

"I got a phone call that I'm a match, but I need to go in for further testing. All they told me was that there was a 13-year-old boy in Cleveland who has leukemia." Camacho said. "How do you say 'no' when there's a family out there that you can help and possibly save a life. I was going to do what I could."

That 13-year-old boy was Enrique Linares. He was diagnosed with acute lymphoblastic leukemia.

His entire family, 38 people in all, were tested to be a donor but none of them were a match.

After nearly two years, spent mostly in the hospital, there was a match. It was a match no one expected. Camacho is unrelated and has a different blood type.

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Grants Pass man meets donor who saved his life

By NEVAGiles23

GRANTS PASS This month, Jerry Condit met the only man in the world who could and did save his life.

Condit needed a stem cell transplant to replace bone marrow lost to leukemia, a blood-attacking cancer diagnosed in January 2008 on his 69th birthday.

Doctors estimated he had two years to live if it went untreated. But finding a suitable donor was difficult. In fact, the National Marrow Donor Program says only about half of the people in need of transplants ever find a donor.

After searching national and international registries of millions of potential donors, doctors found only one match: Marco Rixen, a 34-year-old resident of Germany.

He matched 11 of the 12 markers they were looking for, said Condits wife, Jan. It was enough to consider Rixen a match.

The transplant was performed in May 2008 at Oregon Health & Science University in Portland. Rixen made his donation at a medical center in Germany, where a courier rushed the stem cells to a plane bound for Portland. Less than 36 hours later, Condits transfusion was under way.

For the first two years after the transplant, Condit and Rixen could communicate only through the bone marrow donation agency. After that, names and e-mail addresses were released.

The two kept in touch, and one day the Condits got a message from Rixen that said he was planning to visit the United States.

I didnt know if I would ever get the chance to meet him, said Condit, who cant travel because of his vulnerable immune system. He wrote us and said he was coming here, and we just about fell over.

Rixen and his wife, Anja, spent Sept. 19 with the Condits in Grants Pass before heading to Las Vegas to renew their wedding vows in front of an Elvis impersonator and then visit the Grand Canyon.

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Stem cell transplantation for boy with thalassaemia

By NEVAGiles23

A 10-year-old boy has been infused with stem cells harvested from the bone marrow of his brother to treat him for thalassaemia a disorder caused by destruction of red blood cells. Called allogeneic transplantation of stem cells, this was done at Kovai Medical Center and Hospital.

D. Dhanush may not have to undergo expensive and excruciating blood transfusion anymore if his body accepts the donor cells. But his condition will have to be evaluated very minutely for the next two years to confirm that the cells donated by his brother have been received well and adapted him.

Presenting the boy before media persons, Clinical Haematologist and Head of the Bone Marrow Transplant Unit T. Rajasekar explained that transplantation was of two types autologous and allogeneic.

The autologous procedure involves harvesting of stem cells from the patients themselves (those suffering from thalassaemia or leukaemia). The extracted cells are frozen and stored for high dose treatment.

After being treated, these are infused into the patient through a vein. This procedure was done for one person suffering from myeloma (cancer of plasma cells or white blood cells that produce anti-bodies that help fight infections/diseases) and another with a relapsed lymphoma (cancer of the lymphocytes cells that are part of immune system).

Under the allogeneic procedure, matching stem cells from a donor are used. Mostly, these cells are from siblings or a close relative as they need to pass the human leukocyte antigen (HLA) matching test. HLA matching is required, or the cells will be rejected by the recipient. Ideally, it is sibling whose cells will match because he or she will have the HLA from both parents. It is the combination of HLAs from both parents that are found in the children.

The cells can be harvested from the marrow or from the blood. In the case presented on Tuesday, Dr. Rajasekar said the cells were brought out of the bone marrow in Dhanushs brother and into his blood, from where these were harvested.

Chairman of the hospital Nalla G. Palaniswami said the tough procedure was performed by the new Comprehensive Cancer Centre, which was gradually bringing in specialists of all sub-specialities of cancer care. Only then can this be called a comprehensive centre, he said.

The hospital would form a KMCH Foundation, which would use funds from donors to treat poor children suffering from cancer and some other disorders that required expensive treatment.

The stem cell transplantation that Dhanush, the son of a police head constable, underwent cost Rs.12 lakh. Of this, Rs.9 lakh was provided by a donor, Dr. Palaniswami said. Dean of the hospital V. Kumaran and Head of Department of Interventional Radiology Mathew Cherian spoke on how the cancer centre was established and how developments were being made.

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Gene therapy technique for children with immune disorder improved

By Dr. Matthew Watson

ScienceDaily (Sep. 11, 2012) By including chemotherapy as a conditioning regimen prior to treatment, researchers have developed a refined gene therapy approach that safely and effectively restores the immune system of children with a form of severe combined immunodeficiency (SCID), according to a study published online September 11 in Blood, the Journal of the American Society of Hematology (ASH).

SCID is a group of rare and debilitating genetic disorders that affect the normal development of the immune system in newborns. Infants with SCID are prone to serious, life-threatening infections within the first few months of life and require extensive treatment for survival beyond infancy.

Adenosine deaminase (ADA) deficiency, which accounts for approximately 15 percent of all SCID cases, develops when a gene mutation prohibits the production of ADA, an enzyme that breaks down toxic molecules that can accumulate to harmful levels and kill lymphocytes, the specialized white blood cells that help make up the immune system. In its absence, infants with ADA-deficient SCID lack almost all immune defenses and their condition is almost always fatal within two years if left untreated. Standard treatment for ADA-deficient SCID is a hematopoietic stem cell transplant (HSCT) from a sibling or related donor; however, finding a matched donor can be difficult and transplants can carry significant risks. An alternate treatment method, enzyme replacement therapy (ERT), involves regular injections of the ADA enzyme to maintain the immune system and can help restore immune function; however, the treatments are extremely expensive and painful for the young patients and the effects are often only temporary.

Given the limitations of HSCT and ERT, in the 1990s researchers began investigating the efficacy of gene therapy for ADA-deficient SCID. They discovered that they could "correct" the function of a mutated gene by adding a healthy copy into the cells of the body that help fight infectious diseases. Since then, there have been significant advances in gene therapy for SCID, yet successful gene therapy in patients with ADA-deficient SCID has been seen in only a small series of children due to the difficulty of introducing a healthy ADA gene into bone marrow stem cells and to engraft these cells back into the patients.

"Although the basic steps of gene therapy for patients with SCID have been known for a while, technical and clinical challenges still exist and we wanted to find an optimized gene therapy protocol to restore immunity for young children with ADA-deficient SCID," said Fabio Candotti, MD, one of the study's senior authors, senior investigator in the Genetics and Molecular Biology Branch of the National Human Genome Research Institute at the National Institutes of Health, and chair of the ASH Scientific Committee on Immunology and Host Defense.

To determine whether an enhanced gene therapy approach would improve immunity in children with ADA-deficient SCID, the teams of Dr. Candotti and Donald B. Kohn, MD, director of the Human Gene Medicine Program at the University of California, Los Angeles (UCLA), Professor of Pediatrics and of Microbiology, Immunology, and Molecular Genetics, and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, conducted a clinical trial in 10 patients with the disorder. For the first time, Drs. Candotti and Kohn and their team of investigators compared two different retroviral vectors, MND-ADA and GCsapM-ADA, to transport normal ADA genes into the young patients' bone marrow stem cells as well as two different treatment plans in preparation for receiving gene therapy. Following therapy, investigators found that more bone marrow stem cells were marked with the MND-ADA vector, demonstrating its superiority over the GCsapM-ADA vector.

The investigators also sought to determine whether providing a low dose of chemotherapy prior to gene therapy, known as a pre-transplant conditioning regimen, would successfully deplete the young patients' bone marrow stem cells and make room for gene-corrected stem cells. In four patients, gene therapy was performed without chemotherapy, and the patients remained on ERT throughout the entire procedure to evaluate the efficiency of ERT combined with gene therapy. While these patients did not experience any adverse effects, they also did not experience a significant increase in their levels of the ADA enzyme. They also maintained low absolute lymphocyte counts (ALC) and minimal immune system function, leading the researchers to believe that ERT may weaken the therapy's effect by diluting the number of gene-corrected lymphocytes.

The remaining six patients were treated with the chemotherapy drug busulfan prior to gene therapy and ERT was discontinued prior to the gene therapy procedure. A significant increase in ADA was observed in all six patients; half of them remain off of ERT with partial immune reconstitution -- findings that support results from prior trials in Italy and the United Kingdom using chemotherapy prior to gene therapy and discontinuting ERT. While the ALC of all six patients declined sharply in the first few months due to combined effects of busulfan administration and ERT withdrawal, their counts increased from six to 24 months, even in the three patients that remained off of ERT. After adjusting the chemotherapy dosage, investigators were able to determine an optimal level for enhancing the efficacy of the gene-therapy-corrected cells with minimal toxicity.

This study is the first to detail comparisons of ADA-deficient SCID patient outcomes between those treated with gene therapy who have not received pre-transplant conditioning while continuing to receive ERT with those receiving pre-transplant conditioning without the administration of ERT. This study is also the first to compare two different viral vectors to transport normal ADA genes into patient bone marrow.

"We were very happy that in this trial we were able to see a benefit in the patients after we modified the protocol," said Dr. Kohn. "Doctors treating ADA-deficient SCID have had too few options for too long, and we hope this will provide them with an efficient and effective treatment for this devastating disease."

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Gene therapy technique for children with immune disorder improved

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Stem Cells & Immune System: "Missing Link" Found

By Dr. Matthew Watson

Researchers have discovered a cell that is the "missing link" between bone marrow stem cells and all the cells of the human immune system, according to a release from the University of California, Los Angeles. This finding promises to lead to a more profound understanding of how a healthy immune system is created and as well as how disease can cause poor immune function.

The study's senior author, Dr. Gay Crooks, was quoted as saying, " We felt it was especially important to do these studies using human bone marrow, as most research into the development of the immune system has used mouse bone marrow.The few studies with human tissue have mostly used umbilical cord blood, which does not reflect the immune system of post-natal life."

Understanding the process of normal blood formation in human adults is a crucial step in shedding light on what goes wrong during the process that results in leukemias, cancers of the blood. The findings were published online in the journal Nature Immunology.

"The identification of a progenitor in human bone marrow primed for full lymphoid differentiation will now permit delineation of the molecular regulation of the first stages of lymphoid commitment in human hematopoiesis," the authors wrote. "It will also allow understanding of how these processes are affected during aberrant hematopoiesis in disease states."

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'Missing link' ties blood stem cells, immune system

By NEVAGiles23

LOS ANGELES UCLA researchers have discovered a type of cell that is the "missing link" between bone marrow stem cells and all the cells of the human immune system, a finding that will lead to a greater understanding of how a healthy immune system is produced and how disease can lead to poor immune function.

The research was done using human bone marrow, which contains all the stem cells that produce blood during post-natal life.

"We felt it was especially important to do these studies using human bone marrow, as most research into the development of the immune system has used mouse bone marrow," said the study's senior author, Dr. Gay Crooks, co-director of UCLA's Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research and a co-director of the cancer and stem-cell biology program at UCLA's Jonsson Comprehensive Cancer Center. "The few studies with human tissue have mostly used umbilical cord blood, which does not reflect the immune system of post-natal life."

The research team was "intrigued to find this particular bone marrow cell, because it opens up a lot of new possibilities in terms of understanding how human immunity is produced from stem cells throughout life," said Crooks, a professor of pathology and pediatrics.

Understanding the process of normal blood formation in human adults is a crucial step in shedding light on what goes wrong during the process that results in leukemias, cancers of the blood.

The findings appeared Sept. 2 in the early online edition of the journal Nature Immunology.

Before this study, researchers had a fairly good idea of how to find and study the blood stem cells of the bone marrow. The stem cells live forever, reproduce themselves and give rise to all the cells of the blood. In the process, the stem cells divide and produce cells in intermediate stages of development called progenitors, which make various blood lineages, like red blood cells or platelets.

Crooks was most interested in the creation of the progenitors that form the entire immune system, which consists of many different cells called lymphocytes, each with a specialized function to fight infection.

"Like the stem cells, the progenitor cells are also very rare, so before we can study them, we needed to find the needle in the haystack," said Lisa Kohn, a member of the UCLA Medical Scientist Training Program and first author of the study.

Previous work had found a fairly mature type of lymphocyte progenitor with a limited ability to differentiate, but the new work describes a more primitive type of progenitor primed to produce the entire immune system, Kohn said.

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'Missing link' between stem cells and the immune system

By NEVAGiles23

ScienceDaily (Aug. 31, 2012) UCLA researchers have discovered a type of cell that is the "missing link" between bone marrow stem cells and all the cells of the human immune system, a finding that will lead to a greater understanding of how a healthy immune system is produced and how disease can lead to poor immune function.

The studies were done using human bone marrow, which contains all the stem cells that produce blood during postnatal life.

"We felt it was especially important to do these studies using human bone marrow as most research into the development of the immune system has used mouse bone marrow," said study senior author Dr. Gay Crooks, co-director of the Eli and Edythe Broad Center of Regenerative Medicine and a co-director of the Cancer and Stem Cell Biology program at UCLA's Jonsson Comprehensive Cancer Center. "The few studies with human tissue have mostly used umbilical cord blood, which does not reflect the immune system of postnatal life."

The research team was "intrigued to find this particular bone marrow cell because it opens up a lot of new possibilities in terms of understanding how human immunity is produced from stem cells throughout life," said Crooks, a professor of pathology and pediatrics.

Understanding the process of normal blood formation in human adults is a crucial step in shedding light on what goes wrong during the process that results in leukemias, or cancers of the blood.

The study appears Sept. 2 in the early online edition of Nature Immunology.

Before this study, researchers had a fairly good idea of how to find and study the blood stem cells of the bone marrow. The stem cells live forever, reproduce themselves and give rise to all the cells of the blood. In the process, the stem cells divide and produce intermediate stages of development called progenitors, which make various blood lineages like red blood cells or platelets. Crooks was most interested in the creation of the progenitors that form the entire immune system, which consists of many different cells called lymphocytes, each with a specialized function to fight infection.

"Like the stem cells, the progenitor cells are also very rare, so before we can study them we needed to find the needle in the haystack." said Lisa Kohn, a member of the UCLA Medical Scientist Training Program and first author in the paper.

Previous work had found a fairly mature type of lymphocyte progenitor with a limited ability to differentiate, but the new work describes a more primitive type of progenitor primed to produce the entire immune system, Kohn said

Once the lymphoid primed progenitor had been identified, Crooks and her team studied how gene expression changed during the earliest stages of its production from stem cells.

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'Missing link' between stem cells and the immune system

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UCLA researchers discover missing link between stem cells and immune system

By daniellenierenberg

Public release date: 2-Sep-2012 [ | E-mail | Share ]

Contact: Kim Irwin kirwin@mednet.ucla.edu 310-206-2805 University of California - Los Angeles Health Sciences

UCLA researchers have discovered a type of cell that is the "missing link" between bone marrow stem cells and all the cells of the human immune system, a finding that will lead to a greater understanding of how a healthy immune system is produced and how disease can lead to poor immune function.

The studies were done using human bone marrow, which contains all the stem cells that produce blood during postnatal life.

"We felt it was especially important to do these studies using human bone marrow as most research into the development of the immune system has used mouse bone marrow," said study senior author Dr. Gay Crooks, co-director of the Eli and Edythe Broad Center of Regenerative Medicine and a co-director of the Cancer and Stem Cell Biology program at UCLA's Jonsson Comprehensive Cancer Center. "The few studies with human tissue have mostly used umbilical cord blood, which does not reflect the immune system of postnatal life."

The research team was "intrigued to find this particular bone marrow cell because it opens up a lot of new possibilities in terms of understanding how human immunity is produced from stem cells throughout life," said Crooks, a professor of pathology and pediatrics.

Understanding the process of normal blood formation in human adults is a crucial step in shedding light on what goes wrong during the process that results in leukemias, or cancers of the blood.

The study appears Sept. 2 in the early online edition of Nature Immunology.

Before this study, researchers had a fairly good idea of how to find and study the blood stem cells of the bone marrow. The stem cells live forever, reproduce themselves and give rise to all the cells of the blood. In the process, the stem cells divide and produce intermediate stages of development called progenitors, which make various blood lineages like red blood cells or platelets. Crooks was most interested in the creation of the progenitors that form the entire immune system, which consists of many different cells called lymphocytes, each with a specialized function to fight infection.

"Like the stem cells, the progenitor cells are also very rare, so before we can study them we needed to find the needle in the haystack." said Lisa Kohn, a member of the UCLA Medical Scientist Training Program and first author in the paper.

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UCLA researchers discover missing link between stem cells and immune system

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UCLA Researchers Discover "Missing Link" Between Stem Cells and the Immune System

By Dr. Matthew Watson

Newswise UCLA researchers have discovered a type of cell that is the missing link between bone marrow stem cells and all the cells of the human immune system, a finding that will lead to a greater understanding of how a healthy immune system is produced and how disease can lead to poor immune function.

The studies were done using human bone marrow, which contains all the stem cells that produce blood during postnatal life.

We felt it was especially important to do these studies using human bone marrow as most research into the development of the immune system has used mouse bone marrow, said study senior author Dr. Gay Crooks, co-director of the Eli and Edythe Broad Center of Regenerative Medicine and a co-director of the Cancer and Stem Cell Biology program at UCLAs Jonsson Comprehensive Cancer Center. The few studies with human tissue have mostly used umbilical cord blood, which does not reflect the immune system of postnatal life.

The research team was intrigued to find this particular bone marrow cell because it opens up a lot of new possibilities in terms of understanding how human immunity is produced from stem cells throughout life, said Crooks, a professor of pathology and pediatrics.

Understanding the process of normal blood formation in human adults is a crucial step in shedding light on what goes wrong during the process that results in leukemias, or cancers of the blood.

The study appears Sept. 2 in the early online edition of Nature Immunology.

Before this study, researchers had a fairly good idea of how to find and study the blood stem cells of the bone marrow. The stem cells live forever, reproduce themselves and give rise to all the cells of the blood. In the process, the stem cells divide and produce intermediate stages of development called progenitors, which make various blood lineages like red blood cells or platelets. Crooks was most interested in the creation of the progenitors that form the entire immune system, which consists of many different cells called lymphocytes, each with a specialized function to fight infection.

Like the stem cells, the progenitor cells are also very rare, so before we can study them we needed to find the needle in the haystack. said Lisa Kohn, a member of the UCLA Medical Scientist Training Program and first author in the paper.

Previous work had found a fairly mature type of lymphocyte progenitor with a limited ability to differentiate, but the new work describes a more primitive type of progenitor primed to produce the entire immune system, Kohn said

Once the lymphoid primed progenitor had been identified, Crooks and her team studied how gene expression changed during the earliest stages of its production from stem cells.

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UCLA Researchers Discover "Missing Link" Between Stem Cells and the Immune System

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Osteoporosis Clue Found In Stem Cell Signalling Protein

By Sykes24Tracey

Featured Article Academic Journal Main Category: Bones / Orthopedics Also Included In: Stem Cell Research Article Date: 23 Aug 2012 - 11:00 PDT

Current ratings for: Osteoporosis Clue Found In Stem Cell Signalling Protein

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These are the implications of a new study led by Harvard Medical School (HMS) that was published online in The Journal of Clinical Investigation on 13 August.

Senior author Bjorn Olsen, Hersey Professor of Cell Biology at HMS, told the press about what they found:

"It shifts the thinking about what controls the differentiation of stem cells to bone cells instead of fat cells, and how to make sure this mechanism stays active with aging."

Bone is not a dead material: it is living tissue that is changing all the time, as it is continuously formed and reabsorbed.

Osteoporosis is a common bone disease where bone tissue becomes progressively thinner, resulting in higher risk of fracture. It affects about 1 in 5 American women and is thought to be caused by stem cells that normally differentiate into bone-forming cells becoming fat cells instead over time.

For the study, Olsen, who is professor of developmental biology and dean for research at Harvard School of Dental Medicine, and colleagues, decided to investigate the role of vascular endothelial growth factor, or VEGF, a common signalling protein that plays a key role in the development of blood vessels that are important in early bone growth and skeletal maintenance in mammals. The protein works by activating receptors on the surface of cells.

Soon after they were born, the mice's skeletons began to show osteoporosis-like qualities, such as reduced bone tissue and a build up of fat in the bone marrow.

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Should you be allowed to sell organs?

By raymumme

Some worry that a ruling giving donors the ability to sell their bone-marrow tissue will encourage legal sale of other body parts.

STORY HIGHLIGHTS

(Time.com) -- How much would it take for you to consider selling your bone marrow? A U.S. appeals court puts the price at about $3,000 in a ruling that now makes it legal to pay donors for their bone-marrow tissue.

The court's decision may well help thousands of sick patients who need bone-marrow transplants to survive, but it also begs the question, What other body parts might next be up for sale?

The ruling came about at the end of 2011, in a decision to an October 2009 lawsuit brought by a group of cancer patients, parents and bone-marrow-donation advocates against the government over the federal law banning the buying and selling of bodily organs. The plaintiffs were led by Doreen Flynn, who has three daughters who suffer from Fanconi anemia, a blood disorder that requires bone-marrow transplants to treat.

Flynn and the other plaintiffs said that too many such patients die waiting for transplants and argued that we should be allowed to pay people to donate their marrow as a way of ensuring a more reliable supply. The U.S. Court of Appeals for the Ninth Circuit agreed.

Time.com: Facebook now lets organ donors tell their friends

At the core of the plaintiffs' argument was the National Organ Transplantation Act (NOTA), which since 1984 has forbid the buying and selling of human organs, including bone marrow. But new developments in bone-marrow extraction have made marrow donation not much different from donating blood.

Traditionally, bone-marrow donation required anesthesia and long needles to extract the marrow from the hip bones of donors. Now, a technique called peripheral apheresis allows doctors to extract blood stem cells directly from the blood, instead of the bone -- patients first take a drug that pulls stem cells from the bone and into the blood -- meaning that the marrow cells should be considered a fluid like blood, rather than an organ, the plaintiffs argued. NOTA doesn't prohibit payments for blood or other fluids, such as plasma or semen.

U.S. Attorney General Eric Holder decided not to ask the Supreme Court to review the appellate court's decision, which would have been the next step in overturning it. That means the ruling stands -- and that people can now be paid up to $3,000 for their marrow, as long as it is collected by apheresis. In a concession to the spirit of NOTA, however, the compensation can't be in cash; it needs to be in the form of a voucher that can be applied to things such as scholarships, education, housing or a donation to a charity.

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Should you be allowed to sell organs?

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Adult stem cells from bone marrow: Cell replacement/tissue repair potential in adult bone marrow stem cells in animal …

By daniellenierenberg

ScienceDaily (July 3, 2012) searchers from the University of Maryland School of Maryland report promising results from using adult stem cells from bone marrow in mice to help create tissue cells of other organs, such as the heart, brain and pancreas -- a scientific step they hope may lead to potential new ways to replace cells lost in diseases such as diabetes, Parkinson's or Alzheimer's.

The research in collaboration with the University of Paris Descartes is published online in the June 29, 2012 edition of Comptes Rendus Biologies, a publication of the French Academy of Sciences.

"Finding stem cells capable of restoring function to different damaged organs would be the Holy Grail of tissue engineering," says lead author David Trisler, PhD, assistant professor of neurology at the University of Maryland School of Medicine.

He adds, "This research takes us another step in that process by identifying the potential of these adult bone marrow cells, or a subset of them known as CD34+ bone marrow cells, to be 'multipotent,' meaning they could transform and function as the normal cells in several different organs."

University of Maryland researchers previously developed a special culturing system to collect a select sample of these adult stem cells in bone marrow, which normally makes red and white blood cells and immune cells. In this project, the team followed a widely recognized study model, used to prove the multipotency of embryonic stem cells, to prove that these bone marrow stem cells could make more than just blood cells. The investigators also found that the CD34+ cells had a limited lifespan and did not produce teratomas, tumors that sometimes form with the use of embryonic stem cells and adult stem cells cultivated from other methods that require some genetic manipulation.

"When taken at an early stage, we found that the CD34+ cells exhibited similar multipotent capabilities as embryonic stem cells, which have been shown to be the most flexible and versatile. Because these CD34+ cells already exist in normal bone marrow, they offer a vast source for potential cell replacement therapy, particularly because they come from a person's own body, eliminating the need to suppress the immune system, which is sometimes required when using adults stem cells derived from other sources," explains Paul Fishman, MD, PhD, professor of neurology at the University of Maryland School of Medicine.

The researchers say that proving the potential of these adult bone marrow stem cells opens new possibilities for scientific exploration, but that more research will be needed to see how this science can be translated to humans.

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