Newswise LOS ANGELES (June 19, 2012) Cedars-Sinais Regenerative Medicine Institute has pioneered research on how motor-neuron cell-death occurs in patients with spinal muscular atrophy, offering an important clue in identifying potential medicines to treat this leading genetic cause of death in infants and toddlers.

The study, published in the June 19 online issue of PLoS ONE, extends the institutes work to employ pluripotent stem cells to find a pharmaceutical treatment for spinal muscular atrophy or SMA, a genetic neuromuscular disease characterized by muscle atrophy and weakness.

With this new understanding of how motor neurons die in spinal muscular atrophy patients, we are an important step closer to identifying drugs that may reverse or prevent that process, said Clive Svendsen, PhD, director of the Cedars-Sinai Regenerative Medicine Institute.

Svendsen and his team have investigated this disease for some time now. In 2009, Nature published a study by Svendsen and his colleagues detailing how skin cells taken from a patient with the disorder were used to generate neurons of the same genetic makeup and characteristics of those affected in the disorder; this created a disease-in-a-dish that could serve as a model for discovering new drugs.

As the disease is unique to humans, previous methods to employ this approach had been unreliable in predicting how it occurs in humans. In the research published in PLoS ONE, to the team reproduced this model with skin cells from multiple patients, taking them back in time to a pluripotent stem cell state (iPS cells), and then driving them forward to study the diseased patient-specific motor neurons.

Children born with this disorder have a genetic mutation that doesnt allow their motor neurons to manufacture a critical protein necessary for them to survive. The study found these cells die through apoptosis the same form of cell death that occurs when the body eliminates old, unnecessary as well as unhealthy cells. As motor neuron cell death progresses, children with the disease experience increasing paralysis and eventually death. There is no effective treatment now for this disease. An estimated one in 35 to one in 60 people are carriers and about in 100,000 newborns have the condition.

Now we are taking these motor neurons (from multiple children with the disease and in their pluripotent state) and screening compounds that can rescue these cells and create the protein necessary for them to survive, said Dhruv Sareen, director of Cedars-Sinais Induced Pluripotent Stem Cell Core Facility and a primary author on the study. This study is an important stepping stone to guide us toward the right kinds of compounds that we hope will be effective in the model and then be reproduced in clinical trials.

The study was funded in part by a $1.9 million Tools and Technology grant from the California Institute for Regenerative Medicine aimed at developing new tools and technologies to aid pharmaceutical discoveries for this disease.

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Researchers, with Stem Cells, Advance Understanding of Spinal Muscular Atrophy

ScienceDaily (June 19, 2012) A cutting-edge method developed at the University of Michigan Center for Arrhythmia Research successfully uses stem cells to create heart cells capable of mimicking the heart's crucial squeezing action.

The cells displayed activity similar to most people's resting heart rate. At 60 beats per minute, the rhythmic electrical impulse transmission of the engineered cells in the U-M study is 10 times faster than in most other reported stem cell studies.

An image of the electrically stimulated cardiac cells is displayed on the cover of the current issue of Circulation Research, a publication of the American Heart Association.

For those suffering from common, but deadly heart diseases, stem cell biology represents a new medical frontier.

The U-M team of researchers is using stem cells in hopes of helping the 2.5 million people with an arrhythmia, an irregularity in the heart's electrical impulses that can impair the heart's ability to pump blood.

"To date, the majority of studies using induced pluripotent stem cell-derived cardiac muscle cells have focused on single cell functional analysis," says senior author Todd J. Herron, Ph.D., an assistant research professor in the Departments of Internal Medicine and Molecular & Integrative Physiology at the U-M.

"For potential stem cell-based cardiac regeneration therapies for heart disease, however, it is critical to develop multi-cellular tissue like constructs that beat as a single unit," says Herron.

Their objective, working with researchers at the University of Oxford, Imperial College and University of Wisconsin, included developing a bioengineering approach, using stem cells generated from skin biopsies, which can be used to create large numbers of cardiac muscle cells that can transmit uniform electrical impulses and function as a unit.

Furthermore, the team designed a fluorescent imaging platform using light emitting diode (LED) illumination to measure the electrical activity of the cells.

"Action potential and calcium wave impulse propogation trigger each normal heart beat, so it is imperative to record each parameter in bioengineered human cardiac patches," Herron says.

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New method generates cardiac muscle patches from stem cells

wwltv.com

Posted on June 15, 2012 at 5:53 PM

Updated today at 6:12 PM

Meg Farris / Eyewitness News Email: mfarris@wwltv.com | Twitter: @megfarriswwl

NEWORLEANS- She was only in kindergarten when doctors gave her family the bad news.

Now she's one of the first in Louisiana to try a new treatment for people who get gravely ill after a bone marrow transplant.

The last three years of Sami Smith's life have been physically and emotionally painful.

"I literally, they try to scare me and they can't, because I've been through the scariest thing that you can," said Smith, 9, of Ponchatoula.

Her mother noticed she was napping more and bruising. Doctors diagnosed AML, a type of leukemia or blood cancer. Had she not gotten to the doctor then, she would not have made it much longer. A Child's Wish sent her to Disney World. The good news, one of her teen sisters Mary Hannah, 13, was a good bone marrow match. The transplant worked and Sami was cancer free.

Then devastating news. Sami got a condition called GvHD (Graft-versus-host disease) where the new marrow launches a painful attack on the recipient's body. It's the leading cause of transplant-related death.

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Stem cell treatment offers hope to those sickened after getting bone marrow

SAN DIEGO, CA--(Marketwire -06/15/12)- Bio-Matrix Scientific Group (BMSN) (BMSN) announced today the appointment of David Audley to the advisory board of Its Regen BioPharma subsidiary. Mr. Audley will advise Regen BioPharma on strategic leveraging of national and international clinical research resources. Mr. Audley is viewed by the Company as a key component in the commercialization of stem cell intellectual property. Additionally, it is anticipated that he will assist in raising international awareness for the regenerative therapies being developed by the Company.

In his function as executive director and CEO of the International Cellular Medicine Society (ICMS), Mr. Audley has spearheaded development and implementation of global guidelines for accreditation of stem cell clinics. Under his leadership, the ICMS has grown from a loose association of a handful of physicians to a major international standards organization with over 3500 members from 36 countries. He is a strong advocate for stem cell therapy development and implementation, and is the chief architect of the ICMS accreditation program that is currently evaluating the practices of nearly 20 facilities in a dozen countries. Mr. Audley also has strong professional relationships with Ministries of Health and governmental agencies in South America, Asia and the Middle East.

"My work at ICMS exposes me to the tremendous ability of stem cell therapeutics to alleviate human suffering. Unfortunately, business models have not caught up with the medical reality. Regen BioPharma is unique in that to my knowledge they are the first group to develop a model that accelerates development of stem cell therapeutics in a win-win situation for investors and patients," said David Audley.

"Mr. Audley has made a substantial impact in the clinical translation of stem cell therapeutics by establishing standards, accreditations, an Institutional Review Board (IRB), and partnerships with major organizations such as the AABB," said Christopher Mizer, President of Regen BioPharma. "We are extremely excited to work side by side with Mr. Audley in accelerating access of new stem cell therapies for patients."

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

Bio-Matrix Scientific Group, Inc. (BMSN) (BMSN) is a biotechnology company focused on the development of 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.

Through Its wholly owned subsidiary, Regen BioPharma, it is the Company's goal to develop translational medicine platforms for the rapid commercialization of stem cell therapies. The Company is looking to use these translational medicine platforms to advance intellectual property licensed from entities, institutions and universities that show promise towards fulfilling the Company's goal of increased quality of life. To follow our development, visit us at http://www.regenbiopharma.com.

Disclaimer

This news release may contain forward-looking statements. Forward-looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified. Future events and actual results could differ materially from those set forth in, contemplated by, or underlying the forward-looking statements. The risks and uncertainties to which forward-looking statements are subject include, but are not limited to, the effect of government regulation, competition and other material risks.

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Bio-Matrix Scientific Group Announces David Audley, the Founder of International Cellular Medicine Society, Has Joined ...

For the first time doctors have successfully transplanted a vein grown with a patient's own stem cells, another example of scientists producing human body parts in the lab.

In this case, the patient was a 10-year-old girl in Sweden who was suffering from a severe vein blockage to her liver. Last March, the girl's doctors decided to make her a new blood vessel to bypass the blocked vein instead of using one of her own or considering a liver transplant.

They took a 9-centimetre section of vein from a deceased donor, which was stripped of all its cells, leaving just a hollow tube. Using stem cells from the girl's bone marrow, scientists grew millions of cells to cover the vein, a process that took about two weeks. The new blood vessel was then transplanted into the patient.

Because the procedure used her own cells, the girl did not have to take any drugs to stop her immune system from attacking the new vein, as is usually the case in transplants involving donor tissue.

"This is the future for tissue engineering, where we can make tailor-made organs for patients," said Suchitra Sumitran-Holgersson of the University of Gothenburg, one of the study's authors.

She and colleagues published the results of their work online Thursday in the British medical journal Lancet. The work was paid for by the Swedish government.

The science is still preliminary and one year after the vein was transplanted, it needed to be replaced with another lab-grown vein when doctors noticed the blood flow had dropped. Experts from University College London raised questions in an accompanying commentary about how cost-effective the procedure might be, citing "acute pressures" on health systems that might make these treatments impractical for many patients.

Sumitran-Holgersson estimated the cost at between $6,000 and $10,000.

Similar methods have already been used to make new windpipes and urethras for patients. Doctors in Poland have also made blood vessels grown from donated skin cells for dialysis patients.

Patients with the girl's condition are usually treated with a vein transplant from their own leg, a donated vein, or a liver transplant. Those options can be complicated in children and using a donated vein or liver also requires taking anti-rejection medicines.

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Vein grown from girl's own stem cells transplanted

LONDON For the first time doctors have successfully transplanted a vein grown with a patients own stem cells, another example of scientists producing human body parts in the lab.

In this case, the patient was a 10-year-old girl in Sweden who was suffering from a severe vein blockage to her liver. Last March, the girls doctors decided to make her a new blood vessel to bypass the blocked vein instead of using one of her own or considering a liver transplant.

They took a 3-1/2-inch section of vein from a deceased donor, which was stripped of all its cells, leaving just a hollow tube. Using stem cells from the girls bone marrow, scientists grew millions of cells to cover the vein, a process that took about two weeks. The new blood vessel was then transplanted into the patient.

Because the procedure used her own cells, the girl did not have to take any drugs to stop her immune system from attacking the new vein, as is usually the case in transplants involving donor tissue.

This is the future for tissue engineering, where we can make tailor-made organs for patients, said Suchitra Sumitran-Holgersson of the University of Gothenburg, one of the studys authors.

She and colleagues published the results of their work online Thursday in the British medical journal Lancet. The work was paid for by the Swedish government.

The science is still preliminary and one year after the vein was transplanted, it needed to be replaced with another lab-grown vein when doctors noticed the blood flow had dropped. Experts from University College London raised questions in an accompanying commentary about how cost-effective the procedure might be, citing acute pressures on health systems that might make these treatments impractical for many patients.

Sumitran-Holgersson estimated the cost at between $6,000 and $10,000.

Similar methods have already been used to make new windpipes and urethras for patients. Doctors in Poland have also made blood vessels grown from donated skin cells for dialysis patients.

Patients with the girls condition are usually treated with a vein transplant from their own leg, a donated vein, or a liver transplant. Those options can be complicated in children and using a donated vein or liver also requires taking anti-rejection medicines.

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Vein grown from stem cells

For the first time, doctors have successfully transplanted a vein grown with a patients own stem cells, another example of scientists producing human body parts in the lab.

In this case, the patient was a 10-year-old girl in Sweden who was suffering from a severe vein blockage to her liver. In March, the girls doctors decided to make her a new blood vessel to bypass the blocked vein instead of using one of her own or considering a liver transplant.

They took a 3 1/2-inch section of vein from a deceased donor, which was stripped of all its cells, leaving just a hollow tube. Using stem cells from the girls bone marrow, scientists grew millions of cells to cover the vein, a process that took about two weeks. The new blood vessel was then transplanted into the patient.

Because the procedure used her own cells, the girl did not have to take any drugs to stop her immune system from attacking the new vein, as is usually the case in transplants involving donor tissue.

This is the future for tissue engineering, where we can make tailor-made organs for patients, said Suchitra Sumitran-Holgersson of the University of Gothenburg, one of the studys authors.

She and colleagues published the results of their work online Thursday in the British medical journal Lancet. The work was paid for by the Swedish government.

The science is still preliminary and one year after the vein was transplanted, it needed to be replaced with another lab-grown vein when doctors noticed the blood flow had dropped. Experts from University College London raised questions in an accompanying commentary about how cost-effective the procedure might be, citing acute pressures on health systems that might make these treatments impractical for many patients.

Ms. Sumitran-Holgersson estimated the cost at between $6,000 and $10,000.

Similar methods have already been used to make new windpipes and urethras for patients. Doctors in Poland have also made blood vessels grown from donated skin cells for dialysis patients.

Patients with the girls condition are usually treated with a vein transplant from their own leg, a donated vein, or a liver transplant. Those options can be complicated in children and using a donated vein or liver also requires taking anti-rejection medicines.

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Doctors transplant vein grown with patient's own stem cells

BOCA RATON, Fla., June 13, 2012 (GLOBE NEWSWIRE) -- Good Morning America co-host Robin Roberts' decision to go public with the fact that she has a rare blood disorder was courageous and sheds light on the need for more bone marrow donors in the United States.

"In Robin's case, her sister turned out to be a perfect match, but the fact is, about two out of every three patients who need a transplant won't find a match in their family and will need to reach out to strangers to help save their lives," said Jay Feinberg, CEO of Gift of Life Bone Marrow Foundation, an international bone marrow registry based in Boca Raton, FL.

Approximately 10,000 people are diagnosed each year with a blood disease in which a bone marrow transplant could save their life, yet only half receive one. That is why the more people who are willing to donate, the better the chance of saving a life.

Feinberg knows that all too well. He was diagnosed with Leukemia in the early 1990s. He found his match in 1995 after more than 50,000 people were tested worldwide. He turned that grassroots movement into the not-for-profit Gift of Life Bone Marrow Foundation to get more donors into the worldwide registry and educate the public on the importance of donating. For its part, Gift of Life has facilitated more than 2,500 matches in its history and entered more than 200,000 people into the registry.

"It only takes one match to save a life, and that's what keeps us passionate and focused every day," said Feinberg, who found his match from a young woman who registered at the very last marrow drive organized for him. "The fact that someone as high profile as Robin Roberts is willing to share her personal story with the world will create a lot of new interest in people willing to become donors and to that end, that's a very positive thing. We wish her well in her upcoming treatments."

Gift of Life Bone Marrow Foundation, through its network of life-saving volunteers, organizes dozens of bone marrow drives per year around the world. Feinberg said becoming a donor is easy. A cotton swab is rubbed on the inside of the mouth to collect cells used for tissue typing. That information is then entered into the registry where anyone needing a transplant can turn to see if they find someone compatible. If a match is made, the donor is notified by phone and then undergoes one more test to confirm he or she is a perfect match. If so, the donor then undergoes a complete physical exam, and then the donation procedure, which involves either the taking of blood stem cells from the arms, or bone marrow from the hip. Those life-saving cells are then transplanted into the sick patient. The donor's marrow will eventually replenish itself. On average, one in 1,000 of Gift of Life donors is asked to donate every year.

For more information on bone marrow and blood stem cell transplants, and to see answers to frequently asked questions, please log onto http://www.giftoflife.org.

About the Gift of Life Bone Marrow Foundation

Gift of Life helps children and adults suffering from leukemia, lymphoma, other cancers and genetic disorders find donors for blood and marrow transplants. Headquartered in Boca Raton, Florida, Gift of Life is an internationally recognized bone marrow, blood stem cell, and umbilical cord blood registry. Through its life-saving work, Gift of Life is a world leader helping children and adults find the matches they need when they need them. For more information log on to http://www.giftoflife.org.

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Robin Roberts' Personal Story Highlights Need for More Bone Marrow Donors in the United States

Ethical, Clinical and Commercial Issues to be Navigated before Full Potential of Stem Cell Therapies can be Unleashed

LONDON, June 13, 2012 /PRNewswire-Asia/ -- Stem cells offer exciting potential in regenerative medicine, and are likely to be widely used by mid-2017. Pharmaceutical, biotech and medical device companies are showing increased interest in stem cell research.

New analysis from Frost & Sullivan (http://www.pharma.frost.com), Analysis of the Stem Cell Markets-Unlocking the New Era in Therapeutics, finds that the market will be driven by stem cell applications in drug discovery platforms and by successful academia commercial company partnership models.

"The high attrition rates of potential drug candidates has piqued the interest of pharmaceutical and biotech industries in stem cell use during the drug discovery phase," notes Frost & Sullivan Consulting Analyst Vinod Jyothikumar. "Previously, animal cell lines, tumours, or genetic transformation have been the traditional platform for testing drug candidates; however, these 'abnormal' cells have significantly contributed to a lack of translation into clinical studies."

Many academic institutes and research centres are collaborating with biotechnology and pharmaceutical companies in stem cell research. This will provide impetus to the emergence of novel cell-based therapies.

Key challenges to market development relate to reimbursement, ethics and the complexity of clinical trials.

Securing reimbursement for stem cell therapeutic products is expected to be critical for commercial success. However, stem cell therapies are likely to be expensive. Insurers, therefore, may be unwilling to pay for the treatment. At the same time, patients are unlikely to be able to afford these treatments.

"The use of embryonic stem cells raises a host of thorny ethical, legal, and social issues," adds Jyothikumar. "As a result, market prices for various products may be affected."

Moreover, many research institutes are adopting policies promoting the ethical use of human embryonic tissues. Such policies are hindering the overall research process for several companies working in collaboration with these institutes.

"In addition to apprehensions about how many products will actually make it through human-based clinical trials, companies are also worried about which financial model can be applied to stem cell therapies," cautions Jyothikumar. "Possibly low return on investment (ROI) is also resulting in pharmaceutical companies adopting a cautious approach to stem cell therapeutics."

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New Applications in Drug Discovery Platforms to Fuel Advance of Stem Cells, Says Frost & Sullivan

By Jacque Wilson, CNN

updated 1:44 PM EDT, Tue June 12, 2012

2009: Robin Roberts on her cancer

STORY HIGHLIGHTS

(CNN) -- Robin Roberts' battle against myelodysplastic syndrome, or MDS, is just beginning. The "Good Morning America" anchor will undergo chemotherapy before having a bone marrow transplant later this year.

"Bone marrow donors are scarce and particularly for African-American women," Roberts wrote Monday. "I am very fortunate to have a sister who is an excellent match, and this greatly improves my chances for a cure."

More than 10,000 people in the United States are diagnosed with blood-related disorders every year, according to the National Marrow Donor Program. Often the best treatment is a bone marrow transplant. During the procedure, a donor's stem cells are directly transfused into the sick patient's bloodstream. The patient's new cells multiply over time to create healthy bone marrow.

Unfortunately, the chance of finding a match on the national registry is as low as 66% for African-Americans and other minorities, compared with 93% for Caucasians.

Be the Match, the national registry, has 10 million potential donors, but only 7% are African-American. While the percentage is comparable to the overall African-American population in the United States (which is 12%), the registry is meeting only about a third of the needs for African-American transplants, said Dr. Jeffrey Chell, CEO of the National Marrow Donor Program.

Tuskegee's ghosts: Fear hinders black marrow donation

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Roberts found a match -- others won't be as lucky

CARLSBAD, Calif., June 12, 2012 /PRNewswire/ -- Life Technologies Corporation (LIFE) today announced a partnership with Cellular Dynamics International (CDI), the world's largest producer of human cells derived from induced pluripotent stem (iPS) cells, to commercialize a set of three new products optimized to consistently develop and grow human iPS cells for both research and bioproduction.

The partnership marries CDI's leadership in human iPS cell development with Life Technologies' expertise in stem cell research tool manufacturing and global distribution network to make these novel technologies accessible to researchers around the world. Life Technologies' commercialization of Essential 8 Medium, Vitronectin (VTN-N), and Episomal iPSC Reprogramming Vectors addresses several challenges associated with developing relevant cells for use in a wide range of studies, from basic and translational research to drug discovery efforts. The effectiveness of these products is the focus of recent validation studies published in the journals Nature Methods and PLoS One.

"The launch of these new stem cell culture products furthers CDI founder and stem cell pioneer Jamie Thomson's vision to enable scientists worldwide to easily access the power of iPSC technology, thus driving breakthroughs in human health," noted Bob Palay, CDI Chief Executive Officer.

To eliminate the variability introduced by a mouse cell feeder layer previously used during the culture of human iPS cells, researchers have adopted "feeder-free" media. However, existing feeder-free culture media contain more than 20 interactive ingredients, many of which, such as bovine serum albumin (BSA) and lipids, are highly uncharacterized and vary significantly from lot-to-lot.This leads to variability in iPS cell growth and differentiation and impedes the progress of disease studies and potential clinical applications.

Essential 8 Medium, manufactured in a Life Technologies current Good Manufacturing Practices (cGMP) facility, overcomes this barrier. In addition, BSA and other undesirable components have been removed from the media, thus reducing the number of ingredients to just eight well-characterized elements required to support efficient growth, eliminate variability, and enable large-scale production of human iPS cells.

"Essential 8 has far fewer variables, it's more straight-forward and a lot more reproducible," said Emile Nuwaysir, Ph.D., Chief Operating Officer and Vice President of Cellular Dynamics International. "If the goal is to make a billion cardiomyocytes a day, every day, you want to make sure they're all the same. That's virtually impossible using mouse embryonic fibroblasts and it's very difficult using the more complex, feeder-free media that were available before Essential 8."

Optimized for use with Essential 8 Medium, Vitronectin (VTN-N) is a defined, human protein-based substrate that further eliminates variability during iPS cell culture unlike most existing feeder-free media that requires the use of an undefined matrix derived from mouse tumor cells for cell attachment and growth. The combination of Essential 8 Medium and Vitronectin (VTN-N) provides a defined, culture system free of non-human components for robust, cost-effective and scalable iPS cell culture.

Life Technologies is also introducing the Episomal iPSC Reprogramming Vectors, which leverages non-viral, non-integrating technology to deliver six genes to initiate the reprogramming of human somatic cells, such as blood and skin cells, to iPS cells. A non-viral approach offers a key advantage: human-derived iPS cells have more relevance for patient-specific, disease research. Traditional viral-based methods, such as lentivirus or retrovirus, require integration into the host genome for replication and can disrupt the genome of the reprogrammed cells.

"The ability to reproducibly establish andculture iPS cells using defined reagent systems is key for the advancement of stem cell research, disease modeling and drug discovery," said Chris Armstrong Ph.D, General Manager and Vice President of Primary and Stem Cell Systems at Life Technologies. "The commercialization of these exciting new products serves that purpose and underscores our commitment to provide the most innovative and relevant workflow tools to our customers."

All three products were developed at the University of Wisconsin by Dr. James Thomson, whose lab pioneered embryonic stem cell research and much of the technology surrounding stem cell culturing conditions, in vitro differentiation and iPS cell generation.

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Life Technologies and Cellular Dynamics International Partner for Global Commercialization of Novel Stem Cell ...

istock

"Good Morning America's" Robin Roberts announced this morning that she has myelodysplastic syndrome, or MDS. The syndrome is also known as preleukemia.

MDS can be broken down by its name: Myeloid refers to a type of blood cell; dysplasia means a problem with the development of those cells.

The condition occurs when "something goes wrong in your bone marrow -- the spongy material inside your bones where blood cells are made," according to the Mayo Clinic.

A healthy person's bone marrow produces stem cells that mature into blood cells. But the bone marrow of a person with MDS produces abnormal stem cells that turn into defective blood cells.

Deformed cells get into the bloodstream and eventually outnumber healthy blood cells, according to the National Cancer Institute. Often the deformed blood cells don't live as long as they should, producing a shortage in the body.

There are several types of MDS, depending on the kind of myeloid cells - red blood cells, white blood cells or platelets - that are being affected. Having too few red blood cells results in anemia; having too few white blood cells can result in frequent infections.

The term "preleukemia" is a bit misleading, as most MDS cases do not become cancerous. Certain types of MDS can progress to acute myeloid leukemia, however.

MDS can be caused by exposure to chemotherapy and radiation, common cancer treatments. (Roberts is a breast cancer survivor.)

Symptoms are rare during the early stage of the disease, but can include tiredness, shortness of breath and easy bruising/bleeding. Doctors generally diagnose through a blood test and a bone marrow biopsy.

More here:
What is preleukemia or MDS?

LEUVEN, BELGIUM--(Marketwire -06/08/12)- TiGenix (EURONEXT:TIG)

TiGenix obtains national reimbursement in the Netherlands for breakthrough cartilage therapy ChondroCelect

TiGenix (EURONEXT:TIG) announced today that its innovative cartilage repair therapy ChondroCelect has obtained national reimbursement in the Netherlands. The Dutch National Health Authority (NZa) has formally announced that ChondroCelect is to receive national reimbursement retroactively per January 1, 2012. Previously ChondroCelect was made available in the Netherlands under a risk-sharing scheme.

"We are delighted with the decision of the NZa to reimburse ChondroCelect, and look forward to working with Dutch orthopedic centers of excellence and health insurers to routinely make this breakthrough therapy available to the right patients in the Netherlands," said Eduardo Bravo, CEO of TiGenix. "Dutch clinicians and scientists have been instrumental in ChondroCelect's development and four Cartilage Expert Centers in the Netherlands have already gained extensive experience with the procedure. After having obtained national reimbursement in Belgium last year, this constitutes another major step in improving patient access to this innovative therapy. We remain optimistic that we can obtain national reimbursement in other European countries later this year."

About TiGenix

TiGenix NV (EURONEXT:TIG) is a leading European cell therapy company with a marketed product for cartilage repair, ChondroCelect, and a strong pipeline with clinical stage allogeneic adult stem cell programs for the treatment of autoimmune and inflammatory diseases. TiGenix is based out of Leuven (Belgium) and has operations in Madrid (Spain), and Sittard-Geleen (the Netherlands). For more information please visit http://www.tigenix.com.

About ChondroCelect

ChondroCelect is the first and currently only cell therapy that has been granted market authorisation by the European Union in accordance with the Advanced Therapy Medicinal Product regulation EC1394/2007. For more information, including the European Public Assessment Report (EPAR), prescribing information, and the Summary of Product Characteristics (SPC) please visit the European Medicines Agency (EMA) website at http://www.ema.europa.eu.

Forward-looking information

This document may contain forward-looking statements and estimates with respect to the anticipated future performance of TiGenix and the market in which it operates. Certain of these statements, forecasts and estimates can be recognised by the use of words such as, without limitation, "believes", "anticipates", "expects", "intends", "plans", "seeks", "estimates", "may", "will" and "continue" and similar expressions. They include all matters that are not historical facts. Such statements, forecasts and estimates are based on various assumptions and assessments of known and unknown risks, uncertainties and other factors, which were deemed reasonable when made but may or may not prove to be correct. Actual events are difficult to predict and may depend upon factors that are beyond TiGenix' control. Therefore, actual results, the financial condition, performance or achievements of TiGenix, or industry results, may turn out to be materially different from any future results, performance or achievements expressed or implied by such statements, forecasts and estimates. Given these uncertainties, no representations are made as to the accuracy or fairness of such forward-looking statements, forecasts and estimates. Furthermore, forward-looking statements, forecasts and estimates only speak as of the date of the publication of this document. TiGenix disclaims any obligation to update any such forward-looking statement, forecast or estimates to reflect any change in TiGenix' expectations with regard thereto, or any change in events, conditions or circumstances on which any such statement, forecast or estimate is based, except to the extent required by Belgian law.

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TiGenix: National Reimbursement in the Netherlands Obtained for Breakthrough Cartilage Therapy ChondroCelect(R)

07-06-2012 07:52 Stem cells differ from other types of cells as they are unspecialized cells that are capable of differentiating into almost any type of specialised cells. Stem cells have the ability to replace the diseased and damaged tissue in the body, without the risk of rejection and any side effects. Therapy performed using stem cells is termed as "Regenerative medicine" and has many potential benefits in treating a wide variety of diseases and injuries. The journal is the major open access forum for translational research in stem cell therapies.

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OMICS Group :: Journal of Stem Cell Research

ScienceDaily (June 7, 2012) Scientists at the Gladstone Institutes have for the first time transformed skin cells -- with a single genetic factor -- into cells that develop on their own into an interconnected, functional network of brain cells. The research offers new hope in the fight against many neurological conditions because scientists expect that such a transformation -- or reprogramming -- of cells may lead to better models for testing drugs for devastating neurodegenerative conditions such as Alzheimer's disease.

This research comes at a time of renewed focus on Alzheimer's disease, which currently afflicts 5.4 million people in the United States alone -- a figure expected to nearly triple by 2050. Yet there are no approved medications to prevent or reverse the progression of this debilitating disease.

In findings appearing online June 7 in Cell Stem Cell, researchers in the laboratory of Gladstone Investigator Yadong Huang, MD, PhD, describe how they transferred a single gene called Sox2 into both mouse and human skin cells. Within days the skin cells transformed into early-stage brain stem cells, also called induced neural stem cells (iNSCs). These iNSCs began to self-renew, soon maturing into neurons capable of transmitting electrical signals. Within a month, the neurons had developed into neural networks.

"Many drug candidates -- especially those developed for neurodegenerative diseases -- fail in clinical trials because current models don't accurately predict the drug's effects on the human brain," said Dr. Huang, who is also an associate professor of neurology at the University of California, San Francisco (UCSF), with which Gladstone is affiliated. "Human neurons -- derived from reengineered skin cells -- could help assess the efficacy and safety of these drugs, thereby reducing risks and resources associated with human trials."

Dr. Huang's findings build on the work of other Gladstone scientists, starting with Gladstone Investigator, Shinya Yamanaka, MD, PhD. In 2007, Dr. Yamanaka used four genetic factors to turn adult human skin cells into cells that act like embryonic stem cells -- called induced pluripotent stem cells.

Also known as iPS cells, these cells can become virtually any cell type in the human body -- just like embryonic stem cells. Then last year, Gladstone Senior Investigator Sheng Ding, PhD, announced that he had used a combination of small molecules and genetic factors to transform skin cells directly into neural stem cells. Today, Dr. Huang takes a new tack by using one genetic factor -- Sox2 -- to directly reprogram one cell type into another without reverting to the pluripotent state.

Avoiding the pluripotent state as Drs. Ding and Huang have done is one approach to avoiding the potential danger that "rogue" iPS cells might develop into a tumor if used to replace or repair damaged organs or tissue.

"We wanted to see whether these newly generated neurons could result in tumor growth after transplanting them into mouse brains," said Karen Ring, UCSF Biomedical Sciences graduate student and the paper's lead author. "Instead we saw the reprogrammed cells integrate into the mouse's brain -- and not a single tumor developed."

This research, which was performed at the Roddenberry Center for Stem Cell Biology and Medicine at Gladstone, has also revealed the precise role of Sox2 as a master regulator that controls the identity of neural stem cells. In the future, Dr. Huang and his team hope to identify similar regulators that guide the development of specific neural progenitors and subtypes of neurons in the brain.

"If we can pinpoint which genes control the development of each neuron type, we can generate them in the petri dish from a single sample of human skin cells," said Dr. Huang. "We could then test drugs that affect different neuron types -- such as those involved in Parkinson's disease -- helping us to put drug development for neurodegenerative diseases on the fast track."

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Skin cells reprogrammed into brain cells

SAN DIEGO, CA--(Marketwire -06/04/12)- Medistem Inc. (MEDS) announced today positive safety data from the first 5 patients enrolled in the Non-Revascularizable IschEmic Cardiomyopathy treated with Retrograde COronary Sinus Venous DElivery of Cell TheRapy (RECOVER-ERC) trial. The clinical trial uses the company's "Universal Donor" Endometrial Regenerative Cells (ERC) to treat Congestive Heart Failure (CHF).

According to the study design, after 5 patients enter the trial, they must be observed for a two month time period before additional patients are allowed to enter the study. Patient data was analyzed by the study's independent Data Safety Monitoring Board (DSMB), which concluded that based on lack of adverse effects, the study be allowed to continue recruitment.

"Medistem is developing a treatment for CHF that uses a 30-minute catheter-based procedure to administer the ERC stem cell into the patients' hearts. The achievement of 2 month patient follow-up with no adverse events is a strong signal for us that our new approach to this terrible condition is feasible," said Thomas Ichim, CEO of Medistem.

The RECOVER-ERC trial will treat a total of 60 patients with end-stage heart failure with three concentrations of ERC stem cells or placebo. The clinical trial is being conducted by Dr. Leo Bockeria, Chairman of the Backulev Centre for Cardiovascular Surgery, in collaboration with Dr. Amit Patel, Director of Clinical Regenerative Medicine at University of Utah.

"As a professional drug developer, I am very optimistic of a stem cell product that can be used as a drug. The ERC stem cell can be stored frozen indefinitely, does not need matching with donors, and can be injected in a simple 30-minute procedure into the heart," said Dr. Sergey Sablin, Vice President of Medistem and co-founder of the multi-billion dollar NASDAQ company Medivation.

Currently patients with end-stage heart failure, such as the ones enrolled in the RECOVER-ERC study, have no option except for heart transplantation, which is limited by side effects and lack of donors. In contrast to other stem cells, ERC can be manufactured inexpensively, do not require tissue matching, and can be administered in a minimally-invasive manner. Animal experiments suggest ERC are more potent than other stem cell sources at restoring heart function. The FDA has approved a clinical trial of ERC in treatment of critical limb ischemia in the USA.

About Medistem Inc. Medistem Inc. is a biotechnology company developing technologies related to adult stem cell extraction, manipulation, and use for treating inflammatory and degenerative diseases. The company's lead product, the endometrial regenerative cell (ERC), is a "universal donor" stem cell being developed for critical limb ischemia and heart failure. A publication describing the support for use of ERC for this condition may be found at http://www.translational-medicine.com/content/pdf/1479-5876-6-45.pdf.

Cautionary Statement This press release does not constitute an offer to sell or a solicitation of an offer to buy any of our securities. This press release may contain certain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Forward-looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified. Future events and actual results could differ materially from those set forth in, contemplated by, or underlying the forward-looking information. Factors which may cause actual results to differ from our forward-looking statements are discussed in our Form 10-K for the year ended December 31, 2007 as filed with the Securities and Exchange Commission.

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Medistem Achieves Important ERC Stem Cell Clinical Trial Milestone

In 2004, I had been stationed at Aviano Air Base, Italy, for about a year. One day, while walking into the base exchange, I was approached by an individual standing by one of the many tables that we associate with trying to sell us something or peddle information.

This person was just like you and I, another military member, but the difference was that he had volunteered to try and convince us (Jane and Joe Public) to sign up to potentially help a leukemia patient by donating bone marrow or peripheral blood stem cells.

I was not opposed to the thought of being a registered donor, and in fact signed up that very day. The process only took 10 minutes to fill out the paperwork, and four swabs of the inside of my mouth for molecular matching of donor to recipient. Later I thought, probably like many people before me, What are the chances I will ever be called on to donate?

Next thing I knew it was 2008. I was in my office working on some building project updates, and planning to take some leave, when I received an email from some guy I didnt know. It was a strange name along with a strange email address. I thought to myself this has to be spam. Then I noticed the email was signed and encrypted, so I went ahead and opened it.

What I read next was both exciting and scary at the same time. Im paraphrasing here, but the email basically stated, Sgt Faulkwell, you have been identified as a potential donor for a leukemia patient. Please respond if you are still willing to donate.

Several weeks, and a few vials of blood later, I was identified as the most appropriate donor for my recipient. My trip was organized and paid for by the recipients insurance. They explained that I could have had a friend or family member come with me, or travel from anywhere else in the world to meet me and stay for the whole donation period. It is definitely not something that someone has to go through alone.

In the end, I was asked to donate stem cells. The process took five days, in which I received two shots every day to boost my blood stem-cell production. Essentially, I was mass producing blood stem cells, which are neither red nor white cells yet. The cells were harvested on the fifth day.

It was a fairly painless process, but is highly dependent on each individuals own body composition, health, etc. Stem-cell harvesting is similar to having a transfusion. They pull your blood out, spin it in a machine to withdrawal the stem cells, and then return your blood to you. There were some minor side effects, but nothing compared to what my recipient must have been going through.

My donation went extremely well, and I found out roughly one year later that my recipient had graphed with my stem cells, and that he was doing better. I never received another update, but I hope one day to get the chance to meet the person.

There are too many myths and facts out there for me to get into, but the next time you have someone approach you to become a registered bone-marrow donor, I hope you will take the time to register. You could very well save someones life!

Originally posted here:
Are you a bone-marrow donor? You could save someone’s life today

Health

May 30, 2012

by Suzanne Kurtz, JTA

For nearly a year, Julie Gavrilov has been trying to find a match for her father, Mark.

Diagnosed with a rare and aggressive blood cancer, he needs a stem cell transplant to survive the disease.

A Bukharian Jew born in Uzbekistan, he will have the best chance of survival if he finds a donor from within his own ethnic community.

Since learning of her 58-year-old fathers diagnosis, Gavrilov, an attorney in New York, has organized a donor drive at a Bukharian Jewish community center in the Queens borough of the city, written heartfelt messages for local synagogue newsletters and posted her plea on Facebook.

A compatible donor has yet to be identified, but Gavrilov, 32, is hopeful that the person who can save her fathers life will be found.

It just takes one person, she said.

Finding that person for Jews of non-Ashkenazi descent can be especially difficult.

Excerpt from:
Israeli, U.S. drives aiming to increase number of non-Ashkenazi bone marrow donors

28 May 2012 Last updated at 09:14 ET

A 54m cutting-edge stem cell research centre in Edinburgh has been officially opened by the Princess Royal.

The Royal opened the Scottish Centre for Regenerative Medicine as well as the 24m bio-incubator facility, Nine, in the Edinburgh BioQuarter.

Research into conditions such as multiple sclerosis and heart and liver disease will benefit from the new facilities in Little France.

The Princess Royal unveiled plaques at the centres.

Edinburgh University's Scottish Centre for Regenerative Medicine is the first large-scale, purpose-built facility of its kind and provides accommodation for up to 250 stem cell scientists.

The centre, funded by Edinburgh University, Scottish Enterprise, the Medical Research Council (MRC) and the British Heart Foundation through its Mending Broken Hearts Appeal, is being opened by the Princess Royal in her role as Chancellor of Edinburgh University.

It includes the most up-to-date facilities in the UK, which meet the highest guidelines, to manufacture stem cell lines that could be used for patient therapies.

Nine, which has been jointly funded by Scottish Enterprise and the UK government's department for business, innovation and skills, has 85,000 sq ft of laboratory and office space for both established biotechnology companies and start-up ventures.

The Edinburgh BioQuarter is in the city's Little France area and includes the Royal Infirmary of Edinburgh and Edinburgh University's Queen's Medical Research Institute and Chancellor's Building.

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Princess opens stem cell centre

On a special surface that could help advance stem cell therapies, UM researchers have turned human skin cells into adult-derived stem cells, coaxed them into bone cells and then transplanted them into holes in the skulls of mice. The cells produced four times as much new bone growth as in the mice without the extra bone cells. In this pink-stained image, the black outline partially encloses the new bone growth in the skull. Image credit: Villa-Diaz, L.G., Brown, S.E., Liu, Y. Ross, A.M., Lahann, J.M., Krebsbach, P.H., University of Michigan

ANN ARBOR University of Michigan researchers have proven that a special surface, free of biological contaminants, allows adult-derived stem cells to thrive and transform into multiple cell types. Their success brings stem cell therapies another step closer.

To prove the cells regenerative powers, bone cells grown on this surface were then transplanted into holes in the skulls of mice, producing four times as much new bone growth as in the mice without the extra bone cells.

An embryos cells really can be anything they want to be when they grow up: organs, nerves, skin, bone, any type of human cell. Adult-derived induced stem cells can do this and better. Because the source cells can come from the patient, they are perfectly compatible for medical treatments.

In order to make them, Paul Krebsbach, professor of biological and materials sciences at the UM School of Dentistry, said, We turn back the clock, in a way. Were taking a specialized adult cell and genetically reprogramming it, so it behaves like a more primitive cell.

Specifically, they turn human skin cells into stem cells. Less than five years after the discovery of this method, researchers still dont know precisely how it works, but the process involves adding proteins that can turn genes on and off to the adult cells.

Before stem cells can be used to make repairs in the body, they must be grown and directed into becoming the desired cell type. Researchers typically use surfaces of animal cells and proteins for stem cell habitats, but these gels are expensive to make, and batches vary depending on the individual animal.

You dont really know whats in there, said Joerg Lahann associate professor of chemical engineering and biomedical engineering.

For example, he said that human cells are often grown over mouse cells, but they can go a little native, beginning to produce some mouse proteins that may invite an attack by a patients immune system.

The polymer gel created by Lahann and his colleagues in 2010 avoids these problems because researchers are able to control all of the gels ingredients and how they combine.

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UM: Stem-Cell-Growing Surface Enables Bone Repair