Stem cell therapy is poised to become the next big thing in the treatment of major diseases. Even those extracted from dental pulp can be preserved for future use

Watching his five-year-old pull at his loose tooth, dad Shekar remembered something he had read in a dental clinic. Stem cells from teeth, called dental pulp stem cells (DPSCs) could be preserved and retrieved to treat his son if he had a major ailment in future. Stemade, a private company, would arrange to collect DPSCs through its Smile Clinics and store them in state-of-the-art labs in several cities across the country. His thought: Stem cell technology is the next big step in medical treatment. Banking SCs is medical bio-insurance for his kid.

Stem cell therapy didn't jump out of a box yesterday. We've heard of it being used in treating leukaemia. Patients with spinal cord injury have spent huge sums on it hoping to get up and walk. Some ask: If a house lizard can grow back its tail, why can't we get our systems to re-start with a million multiplying stem cells?

Kinds of cells

The best cells for banking are embryonic cells which are programmed to develop and grow. But harvesting these is banned. Ethical issues, you know. Adult SCs beyond the embryonic stage are classified as haematopoietic (from umbilical cord blood and bone marrow) and mesenchymal (tissues and organs). While haematopoietic cells are used in the treatment of blood-related diseases such as haemophilia, blood cancer and skin troubles, tissue cells are tried on all problems other than these. HSCs are collected only from the umbilical cord and bone marrow. Tissue cells are taken from many body sources such as bone marrow, placenta, menstrual blood, cornea, outer layer of the heart, liposuction waste and teeth pulp.

Among these DPSCs are perhaps the best option, says Shailesh Gadre, MD, Stemade Biotech. We all lose our milk teeth and cell extraction here is almost painless. As for the permanent teeth, we can harvest the pulp when people have to lose them for orthodontic (cosmetic) reasons, as when braces are fixed or teeth are extracted because of poor positioning. Of course, they need to be free of caries and other dental infections.

But as we age, our cells age too, so DPSCs are best extracted and preserved when we're very young, when the cells are virile and robust. DPSCs have extraordinary doubling properties that give them a huge advantage over other stem cells, says Dr. Julian Deepak, Medical Advisor, Stemade. They are derived from the same source as nerve cells, with the same capacity as neuron cells, making them a better option for treating Parkinson's, Alzheimer's and muscular dystrophy. Work is on to see their effectiveness in curing diabetes.

Back to the kid's tooth. After the dad's call, a dentist from Stemade will check if Milan's tooth is free of disease. At a Smile Clinic he will extract it and take a blood sample. The dentist will then place the tooth in a specially-designed vial of antibiotic solution. The vial will be packed in ice-gel to keep the temperature low during transport. At their lab (which I visited) in suburban Chennai, a visual inspection is done, the tooth is flooded with anti-bacterial solution and broken open. The pulp is extracted, divided into parts for quality control and sterility (aerobic/anaerobic) tests. The processing is done in zero-contamination conditions and the cells are put in 5 different vials and placed in the vapour phase of liquid nitrogen for cryo-preservation. It is complete, patented technology. The cells are stored in raw format and can be retrieved when needed. Shekar gets a certificate and a CR Management number which will be part of his son's medical records.

These are your own (autologous) cells and will need no matching should you need them for treatment of tissue-and-organ-related diseases such as spinal cord/bone/liver/cartilage regeneration, diabetes, eye-care, etc., says Shailesh. Adds Dr. Julian, Now for most diseases we just do maintenance therapy. With their regenerative property, stem cells will cure diseases in the future.

Fine, but for a few details. One, is the banking fee? Yes, you have to pay for the banking facility, but we can help you with EMIs, says Shailesh. Subsidies are given to the poor as part of CSR. We want to reach as many households as possible. Others are the right to will it and fool-proof identification of the cells. We may store DPSCs at six and may need them at sixty.

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It's not pulp fiction

To read Hard Cell by Mayrav Saar (PostScript, Feb. 26), one would think the only form of stem-cell therapy is the embryo-destroying kind. There wasnt a single mention of non-embryonic adult stem cells.

One attraction of embryonic versus non-embryonic research for some is political the chance to stick it to pro-lifers. But it grieves me to see ailing people used as pawns in this culture war and being denied the possible benefits of adult stem-cell research.

Flushing such an idea down the memory hole, as you help do with this article, is against the spirit of scientific inquiry.

Bob Hunt, Hillsborough, NJ

Wrong on the right

If social conservatives had won out in history, women would not be able to vote and we would still have slavery (Why Social Issues Matter, Jeffrey Bell, PostScript, Feb. 26).

Their thinking denigrates the role of science and promotes antiquated religious beliefs. Many of the causes taken up by social conservatives have been seen to be wrong in light of later progressive thought.

While social conservatives say some good things, history has shown that their views work against American freedoms an obscurantism that continues today.

Jeffrey Bell should balance his thought with facts and not be led blindly by evangelicals.

Eduardo Rodriguez, Corona

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Stem-cell pawns

Cardiac cellular therapies are undergoing global clinical trials with "encouraging early results" and these economical options will soon be available in India which could bring relief to patients who cannot afford the currently available expensive surgical treatments, says Indian American cardiac surgeon Dr Mukesh Hariawala.

Delivering a special invited plenary lecture on the "Novel Cellular Therapies for Heart Disease" at the recently concluded Healthcare India 2012 convention in New Delhi, the renowned cardiac surgeon asserted that the new developments in cardiac cellular therapies would bring down the alarming healthcare costs globally.

Dr Hariawala is internationally acclaimed as a pioneer of cardiovascular surgical techniques using Therapeutic Angiogenesis. He said Therapeutic Angiogenesis is a fast emerging science of stimulating growth of new blood vessels in the heart which acts as natural bypasses to areas lacking in blood supply.

Dr Hariawala demonstrated angiogenesis along with bypass surgery, lasers and stem cell injections as a novel "Combo Therapy."

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The laser energy acts by creating channels in the diseased heart muscle which also triggers Angiogenesis. Stem cells are then injected directly into coronary arteries feeding the diseased territory or in the stimulated lasered muscle during the open heart surgery. This option could be very helpful in Indian patients with diffused distal small caliber coronary arteries and diabetes, who are not amicable to routinely offered current interventions, he said.

Dr Hariawala acknowledged that only a combination of these four therapies could give it the "Therapeutic Threshold Power" and bring about optimum results and relief of patients symptoms. Standalone, each of these therapies is weak to treat a large muscular pumping organ like the heart.

Stem cells have a therapeutic role and hold enormous promise for the future as they are harvested from the patient's own tissues. Currently, adult stem cell extraction is done from one's own hip bone and patients do not have to worry about rejection phenomenon occurring as they are native cells unlike transplanted from another donor. In the future, stem cell banks could proliferate allowing donors to freeze and store cells for family members who could be treated for many diseases, he added.

Harvard-trained Dr Hariawala's studies have been published in several scientific surgical journals and medical text books.

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Angiogenesis and Stem Cell Therapy Key to Treating Heart Patients: Dr Mukesh Hariawala

ScienceDaily (Mar. 1, 2012) Despite their unassuming appearance, the planarian flatworms in Whitehead Institute Member Peter Reddien's lab are revealing powerful new insights into the biology of stem cells -- insights that may eventually help such cells deliver on a promising role in regenerative medicine.

In this week's issue of the journal Cell Stem Cell, Reddien and scientists in his lab report on their development of a novel approach to identify and study the genes that control stem cell behavior in planarians. Intriguingly, at least one class of these genes has a counterpart in human embryonic stem cells.

"This is a huge step forward in establishing planarians as an in vivo system for which the roles of stem cell regulators can be dissected," says Reddien, who is also an associate professor of biology at MIT and a Howard Hughes Medical Institute (HHMI) Early Career Scientist. "In the grand scheme of things for understanding stem cell biology, I think this is a beginning foray into seeking general principles that all animals utilize. I'd say we're at the beginning of that process."

Planarians (Schmidtea mediterranea) are tiny freshwater flatworms with the ability to reproduce through fission. After literally tearing themselves in half, the worms use stem cells, called cNeoblasts, to regrow any missing tissues and organs, ultimately forming two complete planarians in about a week.

Unlike muscle, nerve, or skin cells that are fully differentiated, certain stem cells, such as cNeoblasts and embryonic stem cells are pluripotent, having the ability to become almost cell type in the body. Researchers have long been interested in harnessing this capability to regrow damaged, diseased, or missing tissues in humans, such as insulin-producing cells for diabetics or nerve cells for patients with spinal cord injuries.

Several problems currently confound the therapeutic use of stem cells, including getting the stem cells to differentiate into the desired cell type in the appropriate location and having such cells successfully integrate with surrounding tissues, all without forming tumors. To solve these issues, researchers need a better understanding of how stem cells tick at the molecular level, particularly within the environment of a living organism. To date, a considerable amount of embryonic stem cell research has been conducted in the highly artificial environment of the Petri dish.

With its renowned powers of regeneration and more than half of its genes having human homologs, the planarian seems like a logical choice for this line of research. Yet, until now, scientists have been unable to efficiently find the genes that regulate the planarian stem cell system.

Postdoctoral researcher Dan Wagner, first author of the Cell Stem Cell paper, and Reddien devised a clever method to identify potential genetic regulators and then determine if those genes affect the two main functions of stem cells: differentiation and renewal of the stem cell population.

After identifying genes active in cNeoblasts, Wagner irradiated the planarians, leaving a single surviving cNeoblast in each planarian. Left alone, each cNeoblast can form colonies of new cells at very specific rates of differentiation and stem cell renewal.

The researchers knocked down each of the active genes, one per planarian, and observed how the surviving cNeoblasts responded. By comparing the rate of differentiation and stem cell renewal to that of normal cNeoblasts, they could determine the role of each gene. Thus, if a colony containing a certain knocked down gene were observed to have fewer stem cells than the controls, it could be concluded that gene in question plays a role in the process of stem cell renewal. And if the colony had fewer differentiated cells than normal, the knocked down gene could be associated with differentiation.

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Planarian genes that control stem cell biology identified

29-02-2012 17:57 Learn more at http://www.cordblood.com CBR's team of dedicated professionals is prepared to guide you through every step of the banking process and beyond. Meet Sherry, CBR's transplant coordinator. As Sherry says, her employer is CBR, but she works for the families who need newborn stem cell medicine. She is the voice parents hear over the phone when they need to use their stored cord blood stem cells. Sherry's dedication and passion to deliver exceptional customer service to clients is one example of the many people at Cord Blood Registry who are committed to helping families live longer, healthier lives.

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Cord Blood Registery Helps Families Use Stem Cells - Video

Newswise UCLA stem cell researchers have discovered a critical placental niche cell and signaling pathway that prevent blood precursors from premature differentiation in the placenta, a process necessary for ensuring proper blood supply for an individuals lifetime.

The placental niche, a stem cell safe zone, supports blood stem cell generation and expansion without promoting differentiation into mature blood cells, allowing the establishment of a pool of precursor cells that provide blood cells for later fetal and post-natal life, said study senior author Dr. Hanna Mikkola, an associate professor of molecular cell and developmental biology and a researcher at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.

Mikkola and her team found that PDGF-B signaling in trophoblasts, specialized cells of the placenta that facilitate embryo implantation and gas and nutrient exchanges between mother and fetus, is vital to maintaining the unique microenvironment needed for the blood precursors. When PDGF-B signaling is halted, the blood precursors differentiate prematurely, creating red blood cells in the placenta, Mikkola said.

The study, done in mouse models, appears March 1, 2012, in the peer-reviewed journal Developmental Cell.

We had previously discovered that the placenta provides a home for a large supply of blood stem cells that are maintained in an undifferentiated state. We now found that, by switching off one signaling pathway, the blood precursors in the placenta start to differentiate into red blood cells, Mikkola said. We learned that the trophoblasts act as powerful signaling centers that govern the niche safe zone.

The study found that the PDGF-B signaling in the trophoblasts is suppressing production of Erythropoietin (EPO), a cytokine that controls red blood cell differentiation.

When PDGF-B signaling is lost, excessive amounts of EPO are produced in the placenta, which triggers differentiation of red blood cells in the placental vasculature, said Akanksha Chhabra, study first author and a post-doctoral fellow in Mikkolas lab.

Mikkola and Chhabra used mouse models in which the placental structure was disrupted so they could observe what cells and signaling pathways were important components of the niche.

The idea was, if we mess up the home where the blood stem cells live, how do these cells respond to the altered environment, Chhabra said. We found that it was important to suppress EPO where blood stem cell expansion is desired and to restrict its expression to areas where red blood cell differentiation should occur.

The finding, Chhabra said, was exciting in that one single molecular change was enough to change the function of an important blood stem cell niche.

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UCLA Scientists Identify Cell and Signaling Pathway that Regulates the Placental Blood Stem Cell Niche

Public release date: 1-Mar-2012 [ | E-mail | Share ]

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

UCLA stem cell researchers have discovered a critical placental niche cell and signaling pathway that prevent blood precursors from premature differentiation in the placenta, a process necessary for ensuring proper blood supply for an individual's lifetime.

The placental niche, a stem cell "safe zone," supports blood stem cell generation and expansion without promoting differentiation into mature blood cells, allowing the establishment of a pool of precursor cells that provide blood cells for later fetal and post-natal life, said study senior author Dr. Hanna Mikkola, an associate professor of molecular cell and developmental biology and a researcher at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.

Mikkola and her team found that PDGF-B signaling in trophoblasts, specialized cells of the placenta that facilitate embryo implantation and gas and nutrient exchanges between mother and fetus, is vital to maintaining the unique microenvironment needed for the blood precursors. When PDGF-B signaling is halted, the blood precursors differentiate prematurely, creating red blood cells in the placenta, Mikkola said.

The study, done in mouse models, appears March 1, 2012, in the peer-reviewed journal Developmental Cell.

"We had previously discovered that the placenta provides a home for a large supply of blood stem cells that are maintained in an undifferentiated state. We now found that, by switching off one signaling pathway, the blood precursors in the placenta start to differentiate into red blood cells," Mikkola said. "We learned that the trophoblasts act as powerful signaling centers that govern the niche safe zone."

The study found that the PDGF-B signaling in the trophoblasts is suppressing production of Erythropoietin (EPO), a cytokine that controls red blood cell differentiation.

"When PDGF-B signaling is lost, excessive amounts of EPO are produced in the placenta, which triggers differentiation of red blood cells in the placental vasculature," said Akanksha Chhabra, study first author and a post-doctoral fellow in Mikkola's lab.

Mikkola and Chhabra used mouse models in which the placental structure was disrupted so they could observe what cells and signaling pathways were important components of the niche.

Read more:
UCLA scientists identify crucial cell and signaling pathway in placental blood stem cell niche

ScienceDaily (Mar. 1, 2012) UCLA stem cell researchers have discovered a critical placental niche cell and signaling pathway that prevent blood precursors from premature differentiation in the placenta, a process necessary for ensuring proper blood supply for an individual's lifetime.

The placental niche, a stem cell "safe zone," supports blood stem cell generation and expansion without promoting differentiation into mature blood cells, allowing the establishment of a pool of precursor cells that provide blood cells for later fetal and post-natal life, said study senior author Dr. Hanna Mikkola, an associate professor of molecular cell and developmental biology and a researcher at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.

Mikkola and her team found that PDGF-B signaling in trophoblasts, specialized cells of the placenta that facilitate embryo implantation and gas and nutrient exchanges between mother and fetus, is vital to maintaining the unique microenvironment needed for the blood precursors. When PDGF-B signaling is halted, the blood precursors differentiate prematurely, creating red blood cells in the placenta, Mikkola said.

The study, done in mouse models, appears March 1, 2012, in the peer-reviewed journal Developmental Cell.

"We had previously discovered that the placenta provides a home for a large supply of blood stem cells that are maintained in an undifferentiated state. We now found that, by switching off one signaling pathway, the blood precursors in the placenta start to differentiate into red blood cells," Mikkola said. "We learned that the trophoblasts act as powerful signaling centers that govern the niche safe zone."

The study found that the PDGF-B signaling in the trophoblasts is suppressing production of Erythropoietin (EPO), a cytokine that controls red blood cell differentiation.

"When PDGF-B signaling is lost, excessive amounts of EPO are produced in the placenta, which triggers differentiation of red blood cells in the placental vasculature," said Akanksha Chhabra, study first author and a post-doctoral fellow in Mikkola's lab.

Mikkola and Chhabra used mouse models in which the placental structure was disrupted so they could observe what cells and signaling pathways were important components of the niche.

"The idea was, if we mess up the home where the blood stem cells live, how do these cells respond to the altered environment," Chhabra said. "We found that it was important to suppress EPO where blood stem cell expansion is desired and to restrict its expression to areas where red blood cell differentiation should occur."

The finding, Chhabra said, was exciting in that one single molecular change "was enough to change the function of an important blood stem cell niche."

See more here:
Cell and signaling pathway that regulates the placental blood stem cell niche identified

MARLBOROUGH, Mass.--(BUSINESS WIRE)--

Advanced Cell Technology, Inc. (ACT, OTCBB: ACTC), a leader in the field of regenerative medicine, today announced year-end results for the year ended December 31, 2011. The Company utilized $13.6 million in cash for operations during the year, compared to $8.8 million in the year-earlier period. The increase in cash utilization resulted primarily from ACTs ongoing clinical activities in the US and Europe. ACT ended the year with cash and cash equivalents of $13.1 million, compared to $15.9 million in cash and cash equivalents in the year-earlier period.

Some of the 2011 highlights included:

2011 was a very important and successful year for ACT as we began our Phase 1/2 trials for the treatment of macular degeneration, said Gary Rabin, chairman and CEO of ACT. We are very excited about the preliminary Phase 1/2 clinical data from our dry-AMD and Stargardts disease trials, which were published in The Lancet earlier this year. The data demonstrated the safety of ACTs human embryonic stem cell (hESC)-derived retinal pigment epithelium (RPE) cells for the treatment of both diseases. The vision of both patients appears to have improved after transplantation, and no adverse safety issues have been observed. We look forward to validating these early findings as we expand these clinical activities throughout this year. Additionally, we made significant progress in advancing our scientific platform, expanding our board of directors and management team and strengthening our balance sheet.

The Company also announced today that it expects to shortly file a preliminary proxy statement with the Securities and Exchange Commission in which it will seek shareholder approval for a reverse split of between 1-for 20 and 1-for 80 shares. The Company is pursuing the reverse split for the sole purpose of meeting the requirements necessary for a listing on the Nasdaq Global Market. The Company believes that a listing on a national change will allow it to expand its shareholder base and improve the marketability of its common stock by attracting a broader range of investors.

Conference Call

The Company will hold a conference call at 9:00 a.m. EST tomorrow, during which it will discuss 2011 results and provide an update on clinical activities. Interested parties should dial (888)264-3177 followed by the reference conference ID number: 57426004. The call will be available live and for replay by webcast at: http://us.meeting-stream.com/advancedcelltechnology030212

About Advanced Cell Technology, Inc.

Advanced Cell Technology, Inc., is a biotechnology company applying cellular technology in the field of regenerative medicine. For more information, visitwww.advancedcell.com.

Forward-Looking Statements

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Advanced Cell Technology Announces 2011 Financial Results

SEATTLE, Feb. 29, 2012 /PRNewswire/ -- Omeros Corporation (NASDAQ: OMER - News) today announced that it has identified compounds that functionally interact with each of the following six orphan G protein-coupled receptors (GPCRs): GPR17, GPR153, CCRL2, LGR4, LGR6 and OPN5. Without compounds that functionally interact with orphan GPCRs, developing drugs targeting those receptors is extremely difficult. Omeros has now unlocked 33 of them, representing over 40 percent of the Class A orphan GPCRs. There are approximately 120 orphan GPCRs and Omeros expects to unlock a large percentage of them, focusing first on Class A orphan GPCRs.

GPR17 is a novel target tied to multiple sclerosis. GPR153 is associated with schizophrenia, and CCRL2 is connected to immunological disorders, such as rheumatoid arthritis. LGR4 is linked to cancer stem cells and the self-renewal and maintenance of adult stem cells. LGR4 is also tied to bone disorders, such as osteoporosis. LGR6 is expressed in the hair follicle stem cells and is involved in long-term wound repair, including the formation of new hair follicles. OPN5 is a recently discovered photoreceptor for ultraviolet light, but its physiological role is currently unknown. Omeros is in the process of filing broad patent applications around its unlocked orphan GPCRs and compound optimization efforts are in progress.

"We continue to advance rapidly through the Class A orphans and, by the end of 2012, we plan to have screened them all using our proprietary Cellular Redistribution Assay," said Gregory A. Demopulos, M.D., chairman and chief executive officer of Omeros. "For each of these receptors, the compounds uniquely identified by Omeros represent keys to drug development, and we believe that Omeros exclusively controls those keys. In parallel with our successful screening efforts, we are building our patent position for each of our unlocked orphans with the goal of protecting and capitalizing on our discoveries."

Ongoing GPCR Program

Omeros is screening orphan GPCRs against its small-molecule chemical libraries using its proprietary, high-throughput cellular redistribution assay (CRA). The CRA detects receptor antagonists, agonists and inverse agonists. Omeros has announced that it has identified and confirmed sets of compounds that interact selectively with 33 orphan receptors linked to metastatic melanoma (GPR19), esophageal squamous cell carcinoma and obesity-related type-2 diabetes (GPR39), hepatocellular carcinoma (GPR80), squamous cell carcinoma (GPR87), pancreatic cancer (GPR182), acute lymphoblastic leukemia (P2Y8/P2RY8), ovarian and prostate cancer (OGR1), arterial stiffness (GPR25), sleep disorders (OPN4), cognitive disorders (GPR12), torpor or "suspended animation" (GPR50), anxiety disorders (GPR31), schizophrenia (GPR52, GPR153), bipolar disorder and schizophrenia (GPR78), psychotic and metabolic disorders (GPR27, GPR85, GPR173), cognitive impairments (MAS1), inflammatory responses (GPR32), obesity and diabetes (GPR21), appetite control (GPR101), immunological disorders (CCRL2), rheumatoid arthritis and HIV-mediated enteropathy (GPR15), respiratory and immune disorders (GPR141), multiple sclerosis (GPR17), motor control (GPR139), congenital cataracts and birth defects of the brain and spinal cord (GPR161), cancer stem cells and the self-renewal and maintenance of adult stem cells (LGR4) and long-term wound repair, including the formation of new hair follicles (LGR6). In addition, Omeros has unlocked GPR20, GPR135 and OPN5, which have not yet been tied to any indications but are expressed preferentially in the gastrointestinal tract (GPR20), brain (GPR135) and eye, brain, testes and spinal cord (OPN5).

About G Protein-Coupled Receptors

GPCRs, which mediate key physiological processes in the body, are one of the most valuable families of drug targets. According to Insight Pharma Reports, GPCR-targeting drugs represent 30 to 40 percent of marketed pharmaceuticals. Examples include Claritin (allergy), Zantac (ulcers and reflux), OxyContin (pain), Lopressor (high blood pressure), Imitrex (migraine headache), Reglan (nausea) and Abilify (schizophrenia, bipolar disease and depression) as well as all other antihistamines, opioids, alpha and beta blockers, serotonergics and dopaminergics.

The industry focuses its GPCR drug discovery efforts mostly on non-sensory GPCRs. Of the 363 total non-sensory GPCRs, approximately 240 have known ligands (molecules that bind the receptors) with nearly half of those targeted either by marketed drugs (46 GPCRs) or by drugs in development (about 70 GPCRs). There are approximately 120 GPCRs with no known ligands, which are termed "orphan GPCRs." Without a known ligand, drug development for a given receptor is extremely difficult.

Omeros uses its proprietary high-throughput CRA to identify small-molecule agonists and antagonists for orphan GPCRs, unlocking them to drug development. Omeros believes that it is the first to possess the capability to unlock orphan GPCRs in high-throughput, and that currently there is no other comparable technology. Unlocking these receptors could lead to the development of drugs that act at these new targets. There is a broad range of indications linked to orphan GPCRs including cardiovascular disease, asthma, diabetes, pain, obesity, Alzheimer's disease, Parkinson's disease, multiple sclerosis, schizophrenia, learning and cognitive disorders, autism, osteoporosis, osteoarthritis and several forms of cancer.

About Omeros Corporation

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Adding Six More, Omeros Now Has a Total of 33 Unlocked Orphan GPCRs in its Portfolio

London, Feb 29 (IANS) Children shot with their own stem cells, for the very first time in a rare immune disorder, have shown improvement.

The condition, known as X-CGD, is caused by faulty genes. Doctors were able to take a sample of the children's stem cells, manipulate them in the lab and reintroduce them. This gave the children a working copy of the faulty gene and their condition improved, enabling them to temporarily fight off infections.

It is the third immune disorder that doctors at Great Ormond Street Hospital have successfully tackled. The others were the life-threatening conditions, X-SCID and ada-SCID, and 90 percent of treated children have improved, with some showing signs that their immune system has been normalised for good.

Remy Helbawi, 16, from South London, was the first child with X-CGD to be treated. The condition only affects boys and means that while his body produces the white blood cells to fight viruses it does not have the correct cells to fight off bacterial or fungal infections, The Telegraph reports.

The resulting infections can be life-threatening. Up until now the only treatment has been a bone marrow transplant which would offer a permanent cure.

Remy's brother who also had the disease was found a bone marrow match and was successfully treated that way but no match has been found for Remy and a serious lung infection was threatening his life.

Remy said: "Until I was 10 I had the same life as anyone else, except I had eczema a lot of the time. I didn't have a fungal infection until about ten, but when I got my first fungal infection my life changed. I missed a lot of school, I had lots of tests and was in hospital. I would get exhausted after climbing stairs."

Before undergoing the gene therapy, Remy had to have chemotherapy which made his hair fall out and he was kept in isolation for a month.

Remy's nurse Helen Braggins said: "Remy had been unwell for last two years and began to miss school. He had significant fungal lung disease in January of last year, which was getting worse. Without some radical treatment intervention, Remy would not have survived and was becoming increasingly short of breath."

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Children improve in rare disorder with own stem cells

Scientists are growing living brain cells from skin samples which could help research into treatments for schizophrenia and bipolar disorder.

Scientists at the University of Edinburgh are growing the cells from skin samples taken from families known to carry faulty genes, which are believed to cause mental illness.

The project, which has received 1 million in funding from the National Centre for the Replacement, Refinement and Reduction of Animals in Research (NC3Rs), aims to develop brain stem cells that could be used to test and screen drugs.

Scientists said it is not easy to understand the diseases using animal models and it is difficult to predict if possible new treatments will work.

They hope using cell-based systems derived from the skin or hair of affected patients will enable researchers to create tests that are more relevant to the disease in humans, and will reduce the dependence on animal models.

Andrew McIntosh, professor of biological psychiatry, said: "We are making different types of brain cells out of skin samples from people with bipolar disorder and schizophrenia.

"Once we have grown these in the laboratory we can then study the cells' neurological function and see how they respond to standard psychiatric treatments. Following this, we hope to be able to screen new medicines."

In the past, researchers used brain tissue from people with schizophrenia and bipolar depression from deceased donors to gain insight into these brain conditions. Scientists said that access to the living brain cells is an exciting development in studying mental illness.

The university said that between 1% and 4% of the world's population is diagnosed with bipolar disorder or schizophrenia, for which there are few highly effective treatments. Little is known about the causes of these conditions but a genetic component is involved as it can run in families.

Well over a million people in the UK are said to be affected by these conditions.

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Living brain cells used in research

Celltex hosts the largest stem-cell bank in the United States.

TYLER RUDICK

With Texas pouring millions of dollars into developing adult stem-cell treatments, doctors there are already injecting paying customers with unproven preparations, supplied by an ambitious new company.

The US Food and Drug Administration (FDA) has not approved any such stem-cell treatment for routine clinical use, although it does sanction them for patients enrolled in registered clinical trials. Some advocates of the treatments argue, however, that preparations based on a patient's own cells should not be classed as drugs, and should not therefore fall under the FDA's jurisdiction.

There are certainly plenty of people eager to have the treatments. Texas governor Rick Perry, for instance, has had stem-cell injections to treat a back complaint1, and has supported legislation to help create banks to store patients' harvested stem cells.

One company that has benefited from this buoyant climate is Celltex Therapeutics, which multiplies and banks stem cells derived from people's abdominal fat, according to chairman and chief executive David Eller. Its facility in Sugar Land, just outside Houston, opened in December 2011 and houses the largest stem-cell bank in the United States.

Celltex was founded by Eller and Stanley Jones, the orthopaedic surgeon who performed Perry's procedure, and it uses technology licensed from RNL Bio in Seoul. Because clinical use of adult-stem-cell treatments are illegal in South Korea, RNL has since 2006 sent more than 10,000 patients to clinics in Japan and China to receive injections.

Celltex says that although it processes and banks cells, it does not carry out stem-cell injections. It declined to answer Nature's questions about whether its cells have been used in patients. But there is evidence that the company is involved in the clinical use of the cells on US soil, which the FDA has viewed as illegal in other cases.

In addition to the publicity surrounding Perry's treatment, a woman named Debbie Bertrand has been blogging about her experiences during a five-injection treatment with cells prepared at Celltex. Her blog (http://debbiebertrand.blogspot.com) hosts photographs of herself alongside Jones; Jennifer Novak, a Celltex nurse; Jeong Chan Ra, chief executive of RNL Bio; and her doctor, Jamshid Lotfi, a neurologist who works for the United Neurology clinic in Houston. Another photo is captioned: My cells are being processed in here for my next infusion!!! A third shows Bertrand, Lotfi and a physician called Matthew Daneshmand, who is, according to the caption, injecting Bertrand's stem cells into an intravenous drip, ready for the infusion. Nature has been unable to contact Bertrand.

Lotfi says that he has administered cells processed by Celltex to more than 20 people. Five or six including Bertrand have multiple sclerosis and four or five have Parkinson's disease, he says. Lotfi explains that patients sign up for treatment by contacting Novak, and that cells are prepared by removing about five grams of fat containing roughly 100,000 mesenchymal stem cells from the patient's abdomen. Over a three-week period, the cells are cultured until they reach about 800 million cells. Lotfi says that patients get at least three injections of 200 million cells each, and that the cells do not take effect for a few months. According to Lotfi, Celltex charges US$7,000 per 200 million cells, and pays Lotfi $500 per injection.

Continued here:
Stem-cell therapy takes off in Texas

SILVER SPRING, Md.--(BUSINESS WIRE)--

Nuvilex, Inc. (OTCQB:NVLX), an emerging biotechnology provider of cell and gene therapy solutions, announced today Goldman Small Cap Research has reissued its buy recommendation on Nuvilex with a short term price target of $0.50 per share.

According to the research report prepared by Goldman, The current share price represents but a fraction of its true value, in our view. With recently increased interest and valuation in the pancreatic cancer treatment arena, we believe that Nuvilex is worth $0.20 just on the oncology therapies alone and that the shares will reach $0.50 in the next six months. Looking ahead, as milestone events occur, $1.00 per share is within reach over the next 12-18 months.

Goldman bases this value projection, in part, on the pending acquisition of SG Austria assets, and with it complete control over the cell encapsulation technology that forms the backbone of Nuvilexs planned biotechnology development. The report states in part the following:

Following execution of the SG Austria asset acquisition, we expect to see a flurry of events and progress on the development side which will serve as catalysts, including when management submits its protocol for the next stage pancreatic cancer trial. We would not be surprised to see the stock break through the $0.50 price on such news as well as progress on the next stage of trials for other therapies.

One reason we are so convinced of the great buying opportunity is the fact that pancreatic cancer treatments are currently at the forefront of the biotech space and are enjoying very high valuations. Although Nuvilex is a not a drug producer, but an existing therapy enhancer through the use of its live cell encapsulation enhancement platform, the timing of these milestone events could not be better for Nuvilex and a re-valuation of its offering.

The Goldman report also compares alternative oncology therapies, including Gemzar from Threshold Pharmaceuticals and Merrimack Pharmaceuticals drug encapsulation technology, noting that, contrary to these treatments, the Nuvilex live-cell encapsulation technology is not limited to one specific use, but can be adapted to use for a host of cell types. The report states, Its difficult to compare apples-to-apples in this space as Nuvilex is the only firm utilizing live-cell encapsulation therapy for cancer, while all the other treatments are based upon a particular drug usage. Contrasting the results of different Phase II clinical trials, the Goldman report comments that the pancreatic cancer therapy, based on completed Phase 1/2 data, appears to have yielded statistically greater results than competing technologies.

Commenting on The Goldman Report, Nuvilex Chief Executive Officer, Dr. Robert Ryan, stated, The report did an excellent job highlighting the value and capabilities of our cell encapsulation technology, not just for cancer therapy, but also for the vast array of treatments where live-cell encapsulation can aid multiple diseases. In the case of the completed cancer trials, it generated superior results with lower drug dosages, and reduced chemotherapeutic side effects. As we move forward with diabetes and stem cell therapy treatments, we are confident our success will, as Goldman predicts prompt leaders in multiple treatment segments to partner with Nuvilex in order to maintain their respective market shares.

Investors are recommended to study the Goldman Research Report for a detailed review and valuation methodology regarding Nuvilex.

About Nuvilex

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Nuvilex Reveals Goldman Small Cap Research Cites Groundbreaking Cancer Therapy in Updating Buy Recommendation

Scientists might have found a way for a woman to be able to produce more eggs, potentially aiding and extending her fertility. The study, published in the journal Nature Medicine, found the ovaries of young women might still contain egg-producing stem cells, according to a report by MSNBC.

How could these stem cells potentially be used?

Theoretically, they could be used to develop new treatments for women who are struggling with infertility issues. The lead researcher on the study, Jonathan Tilly, has said that the stem cells could potentially be used to preserve the fertility of younger women who have struggled with serious diseases, like cancer, that may require harsh treatments that destroy the viability of their available eggs. He also speculated that they may be able to be used to restore egg production for an older woman that is no longer fertile.

What did the study involve?

Tilly, who works through Harvard-affiliated Massachusetts General Hospital, had first discovered these stem cells in mice. He then went looking for them in donated ovaries that he acquired through a partnership with a Japanese hospital.

The stem cells had to be isolated in order for Tilly to test them for their ability to produce new eggs. After being injected with a gene that would change them to a particular color, the stem cells were placed in part of a human ovary and grafted under the skin of mice to monitor the effect, according to My Health News Daily. The grafted stem cells did in fact appear to begin to grow new, albeit immature, eggs.

What are the potential challenges facing this study?

Mostly, skepticism. Some experts that have reviewed the study, including Dr. Mario Conti of the Center for Reproductive Sciences at the University of California, San Francisco, have pointed out that Tilly has failed to prove that these cells can be used to grow eggs in humans rather than mice. Other criticism concerns the stem cells themselves, which appear to make up a very small amount of the cells of the ovaries, and whether or not they are capable of producing a mature egg that can be fertilized and grow into a human being.

What are the next research steps?

Tilly plans on conducting more studies to test the potential of these stem cells. WebMD reported that he has already partnered with cell biologist Dr. Evelyn Telfer at the University of Edinburgh in Scotland to begin developing techniques to take the immature, or "seed" eggs and encourage them to become fully-mature eggs which may be able to be used.

More here:
Stem Cells Might Have the Potential to Produce New Eggs

These human eggs (oocytes) can now be made from adult ovaries; Credit: Shutterstock

Yvonne A R White, Dori C Woods, Yasushi Takai, Hiroyuki Seki and Jonathan L Tilly worked hard in 2004. When this intrepid team revealed that some mammals (eg. mice) can produce eggs into their adult life, there was hope that stem cells could now become a staple of medical ideas. That hope has been fully justified. Published in the March issue of Nature Medicine, the same team have explored human female ovary capabilities and performed what was thought the impossible.

We may all know that female babies are born with their full and finite complement of oocytes or eggs, but we are only partly correct. Now the possibilities have enlarged. The proof that you could find egg-producing stem cells in the ovary of adult women was paramount for this team of scientists.

Dr. Jonathan Tilly directs the Vincent Center in Massachusetts General Hospital: "The discovery of oocyte precursor cells in adult human ovaries, coupled with the fact that these cells share the same characteristic features of their mouse counterparts that produce fully functional eggs, opens the door for development of unprecedented technologies to overcome infertility in women and perhaps even delay the timing of ovarian failure." Presumably, stem cell researchers will read much more into this.

Shanghai mouse research provided support with proof of egg-producing stem cells in 2009 and then the Vincent team developed a more precise green fluorescence-activated cell-sorting technique(GFP), whereby no possibility of contamination from other cells was possible. The verified eggs they produced could then be fertilised and developed into blastocysts. Now for human tissues. The resultant oocytes (eggs) not only looked like and grew like those in human ovaries, but some had the required haploid number of chromosomes, presumably after meiosis (all true eggs of course have to double up their DNA later, when fertilised.)

This cross-section of a human ovary shows potential areas for stem cells -which can now be converted to oocytes - even in adult women; Credit: Shutterstock

The final step, to date, involved using mouse recipients for the human tissue. Immature human follicles and oocytes were found after 7-14 days, and possibly were present before the mouse skin graft. Dr. Tilly and the team are now exploring the freezing of these cells in human OSC banks, as human eggs cannot be frozen and thawed without damage.

Likewise, factors such as hormones that influence the marvellous transformation from OSC to oocyte need to be identified with IVF and other infertility possibilities become let us say, "improved" spectacularly by these discoveries. Women's health generally could also be improved by maintaining some functions in the ovary throughout life. Let us be clear that with even more from these particular stem cells, a fascinating transformation of the whole of medicine lies ahead.

Read this article:
Eggs from Stem Cells excite the imagination

Allegheny-Kiski Health Foundation will present "Stem-Cell Therapy: Defeating the Aging Process" from 6 to 8 p.m. March 13 in the William & Grayce Walker conference Room at Allegheny-Kiski Health Foundation, Charles and Mary Lou Young Non-Profit Center, 1 Acee Drive, Natrona Heights.

Guest speaker will be Dr. Valerie Donaldson of the Individualized Advanced Medical Center of Pittsburgh. She is active in destressing the body and focusing on the anti-aging process.

Donaldson completed her undergraduate education at Colorado College where she earned a bachelor's degree in biology and obtained her doctorate at Rush Medical College in Chicago.

Registration is requested. Call 724-294-3157. Admission is free.

The seminar is sponsored through the Dr. H.W. Fraley Health and Wellness Fund.

Programs set at Destination Wellness

Upcoming programs at Allegheny Valley Hospital's Destination Wellness at Pittsburgh Mills, Frazer, include:

Pittsburgh North Restless Legs Syndrome Support Group will meet from 6:15 to 7:45 p.m. Dr. Avinash Aggarwal will discuss "Is it RLS or Something Else?" To register, call Destination Wellness at 724-274-5202.

Heartsaver First Aid, part one will be from 9 a.m. to noon March 10 and is the basic first-aid course. A two-year certification card will be given after completion of skills and written testing. Fee is $35 per part and includes a required student manual. Call 724-274-5202 to register. Space is limited to eight participants.

Heartsaver AED/CPR, part two will be from 1 to 4 p.m. March 10 and includes adult, child and infant CPR and automated external defibrillator use. A two-year certification card will be given after completion of skills and written testing. Fee is $35 per part and includes required student manual. Call 724-274-5202 to register. Space is limited to eight participants.

See more here:
Program looks at stem-cell therapy to defeat aging

Regenerative Medicine Institute, Mexico has been granted full accreditation for its clinical stem cell trials

Portland, Oregon (PRWEB) February 29, 2012

We are pleased that RMI undertook this process, says David Audley, executive director of the ICMS. The clinic understood that patient safety can only be assured through strict evaluation and rigorous oversight. From day one they have embraced the transparency that this program requires.

RMI is the first clinic to achieve this status under the ICMS Accreditation Program. The clinic has undergone two separate site audits as well as an institutional review board review evaluation. Most importantly, the clinic has placed in excess of 50 patients into the Treatment Registry for long-term outcome tracking. The safety profile has been excellent, continued Audley. We have tracked patients over at least two follow-ups and a minimum of six months and not seen a single cell-related adverse event.

The ICMS is currently evaluating nearly a dozen clinics worldwide. Accreditation is based upon the Guidelines for the Practice of Cell-Based Medicine developed and published by the ICMS. Key components of these guidelines are the ethical recruitment of patients, proper consent of patients and compliance with local laws and regulations in the treatment of patients.

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Mr. David Audley International Cellular Medicine Society 503-884-6590 Email Information

Excerpt from:
International Cellular Medicine Society Grants First Worldwide Accreditation to Tijuana Clinical Trial

28-02-2012 14:48 Albert Rodriguez, MD administers stem cell therapy for Hereditary Spastic Paraplegia. stemcelldrR.com, email airpainmd@aol.com

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Stem Cell Therapy Procedure and Outcome - Video

Study Aims to Deliver Adult’s Own Cells As Treatment for Chronic Myocardial Ischemia

DEERFIELD, Ill.--(BUSINESS WIRE)--Baxter International Inc. (NYSE:BAX) announced today that it has initiated a phase III pivotal clinical trial to evaluate the efficacy and safety of adult autologous (an individual’s own) CD34+ stem cells to increase exercise capacity in patients with chronic myocardial ischemia (CMI).

Chronic myocardial ischemia (CMI) is one of the most severe forms of coronary artery disease, causing significant long-term damage to the heart muscle and disability to the patient. It is often diagnosed based on symptoms of severe, refractory angina, which is severe chest discomfort that does not respond to conventional medical management or surgical interventions.

“The prospect of using a person’s own adult stem cells to restore and repair blood flow in CMI is a very exciting concept based on a biological regenerative approach,” said Norbert Riedel, Ph.D., Baxter’s chief science and innovation officer. “The goals of this phase III trial are aligned with Baxter’s overall mission to develop life-saving and life-sustaining therapies and it will help us determine if the therapy can make a meaningful difference for CMI patients.”

The trial will enroll approximately 450 patients across 50 clinical sites in the United States, who will be randomized to one of three arms: treatment with their own autologous CD34+ stem cells, treatment with placebo (control), or unblinded standard of care. The primary objective is to evaluate the efficacy of treatment with CD34+ stem cells to improve the functional capacity of patients with CMI, as measured by a change in total exercise capacity at 12 months following treatment. Secondary objectives include reduced frequency of angina episodes at 12 months after treatment and the safety of targeted delivery of the cells.

After stem cell mobilization, apheresis (collecting the cells from the body) and cell processing, participants will receive CD34+ stem cells or placebo in a single treatment via 10 intramyocardial injections into targeted areas of the heart tissue. Efficacy will be measured by a change in total exercise capacity during the first year following treatment and safety data will be collected for two years. Stem cell processing will be conducted in GMP facilities in the United States by Progenitor Cell Therapy (PCT), a subsidiary of NeoStem, Inc. To learn more or enroll, visit http://www.renewstudy.com or http://www.clinicaltrials.gov.

This trial is being initiated based on the phase II data, which indicated that injections of patients’ own CD34+ stem cells may improve exercise capacity and reduce reports of angina episodes in patients with chronic, severe refractory angina.

“The phase II trial provided evidence that this strategy, leveraging the body’s own natural repair mechanisms, can improve exercise capacity and reduce chest pain, the first time these endpoints have been achieved in a population of patients who have exhausted conventional treatment options,” said Douglas Losordo, MD, vice president of new therapeutic development at Baxter.

CD34+ cells, which are blood-forming stem cells derived from bone marrow, are comprised of endothelial progenitor cells (EPCs), which develop into new blood vessels. Previous preclinical studies investigating these cells have shown an increase in capillary density and improved cardiac function in models of myocardial ischemia.

About Baxter

Baxter International Inc., through its subsidiaries, develops, manufactures and markets products that save and sustain the lives of people with hemophilia, immune disorders, infectious diseases, kidney disease, trauma, and other chronic and acute medical conditions. As a global, diversified healthcare company, Baxter applies a unique combination of expertise in medical devices, pharmaceuticals and biotechnology to create products that advance patient care worldwide.

This release includes forward-looking statements concerning the use of adult autologous stem cells to treat CMI, including expectations with respect to the related phase III clinical trial. These statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those in the forward-looking statements: clinical results demonstrating the safety and effectiveness of the use of autologous stem cells to treat CMI; timely submission of regulatory filings; satisfaction of regulatory and other requirements; actions of regulatory bodies and other governmental authorities; the enrollment of a sufficient number of qualified participants in the phase III clinical trial; the successful provision of stem cell processing by PCT, a third party; and other risks identified in Baxter’s most recent filing on Form 10-K and other SEC filings, all of which are available on Baxter’s website. Baxter does not undertake to update its forward-looking statements.

Photos/Multimedia Gallery Available: http://www.businesswire.com/cgi-bin/mmg.cgi?eid=50183372&lang=en

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Baxter Initiates Phase III Adult Stem Cell Clinical Trial for Chronic Cardiac Condition