ScienceDaily (Mar. 27, 2012) A team of researchers led by scientists at Weill Cornell Medical College has designed what appears to be a powerful gene therapy strategy that can treat both beta-thalassemia disease and sickle cell anemia. They have also developed a test to predict patient response before treatment.

This study's findings, published in PLoS ONE, represents a new approach to treating these related, and serious, red blood cells disorders, say the investigators.

"This gene therapy technique has the potential to cure many patients, especially if we prescreen them to predict their response using just a few of their cells in a test tube," says the study's lead investigator, Dr. Stefano Rivella, Ph.D., an associate professor of genetic medicine at Weill Cornell Medical College. He led a team of 17 researchers in three countries.

Dr. Rivella says this is the first time investigators have been able to correlate the outcome of transferring a healthy beta-globin gene into diseased cells with increased production of normal hemoglobin -- which has long been a barrier to effective treatment of these disease.

So far, only one patient in France has been treated with gene therapy for beta thalassemia, and Dr. Rivella and his colleagues believe the new treatment they developed will be a significant improvement. No known patient has received gene therapy yet to treat sickle cell anemia.

A Fresh Approach to Gene Therapy

Beta-thalassemia is an inherited disease caused by defects in the beta-globin gene. This gene produces an essential part of the hemoglobin protein, which, in the form of red blood cells, carries life-sustaining oxygen throughout the body.

The new gene transfer technique developed by Dr. Rivella and his colleagues ensures that the beta-globin gene that is delivered will be active, and that it will also provide more curative beta-globin protein. "Since the defect in thalassemia is lack of production of beta-globin protein in red blood cells, this is very important," Dr. Rivella says.

The researchers achieved this advance by hooking an "ankyrin insulator" to the beta-globin gene that is carried by a lentivirus vector. During the gene transfer, this vector would be inserted into bone marrow stem cells taken from patients, and then delivered back via a bone marrow transplant. The stem cells would then produce healthy beta-globin protein and hemoglobin.

This ankyrin insulator achieves two goals. First, it protects delivery of the normal beta-globin gene. "In many gene therapy applications, a curative gene is introduced into the cells of patients in an indiscriminate fashion," Dr. Rivella explains. "The gene lands randomly in the genome of the patient, but where it lands is very important because not all regions of the genome are the same." For example, some therapeutic genes may land in an area of the genome that is normally silenced -- meaning the genes in this area are not expressed. "The role of ankyrin insulator is to create an active area in the genome where the new gene can work efficiently no matter where it lands," Dr. Rivella says. He adds that the small insulator used in his vector should eliminate the kind of side effects seen in the French patient treated with beta-thalassemia gene therapy.

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New gene therapy approach developed for red blood cell disorders

NEW YORK, NY--(Marketwire -03/28/12)- Biotechnology stocks have been on an impressive run this year as favorable legislation out of Washington is allowing biotech companies of all sizes to more easily navigate regulations. Five Star Equities examines the outlook for companies in the Biotechnology industry and provides equity research on Advanced Cell Technology Inc. (OTC.BB: ACTC.OB - News) and PharmAthene Inc. (AMEX: PIP - News). Access to the full company reports can be found at:

http://www.fivestarequities.com/ACTC http://www.fivestarequities.com/PIP

The Biotechnology Industry Organization (BIO) recently applauded the House Energy and Commerce Committee's passage of the Medicare Decisions Accountability Act, H.R. 452, which would repeal the Independent Payment Advisory Board (IPAB) established in the health care reform law. BIO also issued a press release applauding the Senate on the passage of H.R. 3606, the Jumpstart Our Business Startups (JOBS) Act. The JOBS Act creates an "on-ramp" to the public market for emerging growth companies, allowing them five years to focus on conducting critical research that can lead to cures for debilitating diseases before having to divert funds to costly regulations, BIO reports.

Five Star Equities releases regular market updates on the biotechnology industry so investors can stay ahead of the crowd and make the best investment decisions to maximize their returns. Take a few minutes to register with us free at http://www.fivestarequities.com and get exclusive access to our numerous stock reports and industry newsletters.

Advanced Cell Technology, Inc., a biotechnology company, focuses on the development and commercialization of human embryonic and adult stem cell technology in the field of regenerative medicine. Earlier this month the company filed with the Securities and Exchange Commission a proxy statement containing a shareholder proposal for a reverse split of its common stock. "This reverse stock split, which should better align the company's capital structure with its stage of development, and an accompanying Nasdaq listing application, will represent a significant step toward creating long-term shareholder value and building ACT into a world-class player in the regenerative medicine space," said Gary Rabin, chairman and CEO of ACT.

PharmAthene, Inc., a biodefense company, engages in the development and commercialization of medical countermeasures against biological and chemical weapons in the United States. For the year ended December 31, 2011, PharmAthene recognized revenue of $24.3 million, compared to $21.0 million in 2010.

Five Star Equities provides Market Research focused on equities that offer growth opportunities, value, and strong potential return. We strive to provide the most up-to-date market activities. We constantly create research reports and newsletters for our members. Five Star Equities has not been compensated by any of the above-mentioned companies. We act as an independent research portal and are aware that all investment entails inherent risks. Please view the full disclaimer at: http://www.fivestarequities.com/disclaimer

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Advanced Cell Technology and PharmAthene Poised to Benefit From Positive Legislation

From being unable to move the majority of his body in 2008 to taking steps with leg braces today, Mount Shastas Corben Brooks is proving that a spinal cord injury isnt the end of the world.

Three and a half years after a high school football injury left him a quadriplegic, Mount Shastas Corben Brooks is focused on recovery while in New Delhi, India, where hes receiving a third round of stem cell treatments not yet available in the United States.

The ever-optimistic 20 year old can now stand with minimal assistance, take steps with leg braces, wiggle his toes, partially close his hands and feel the majority of his legs.

Corben said hes looking forward to Labor Day Weekend, when his family will host Thunder in the Park in Mount Shasta, an event which will include the raffle drawing for a custom built motorcycle dubbed Corbens Ride, as well as live music, a chili cookoff, pancake breakfast and a poker run. Thunder in the Park will coincide with the Mount Shasta Police Departments Show & Shine car show in attempt to keep visitors in Mount Shasta the entire weekend.

Without the support of our community and countless other people I wouldn't be where I am today, Corben said via email from India last week. I can honestly say that without the help from my family, friends, this wonderful community and all who have so generously gone out of their way to help me, I would not be in the remarkable position that I am today. Thank you is nowhere near an adequate enough word to express my thanks.

Though he knows stem cell treatments are controversial, Corben said after each treatment he sees more function and sensation in his body for up to nine months after returning home.

So far on this trip I have gained new sensation and feeling in the back of my legs and hamstrings and additional feeling on my left foot, Corben said.

The results of a recent MRI also showed encouraging results, said Corben.

What we saw was the stem cells have been reducing the amount of scar tissue in my spinal cord at the injury site, Corben said. With the scar tissue being reduced, my nerves are given the opportunity to reestablish a connection. And we believe that is why I have been seeing continual recovery during and after these treatments.

Since his last visit in 2011, Corben said his walking has improved greatly, thanks to the help of his trainer back at home, Lisa Pigoni.

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Corben Brooks continues on road to recovery

ScienceDaily (Mar. 26, 2012) A research network led by a Mayo Clinic physician found that stem cells derived from heart failure patients' own bone marrow and injected into their hearts improved the function of the left ventricle, the heart's pumping chamber. Researchers also found that certain types of the stem cells were associated with the largest improvement and warrant further study.

The results were presented March 26 at the 2012 American College of Cardiology Meeting in Chicago. They will also be published online in the Journal of the American Medical Association.

This Phase II clinical trial, designed to test this strategy to improve cardiac function, is an extension of earlier efforts in Brazil in which a smaller number of patients received fewer stem cells. For this new network study, 92 patients received a placebo or 100 million stem cells derived from the bone marrow in their hips in a one-time injection. This was the first study in humans to deliver that many bone marrow stem cells.

"We found that the bone marrow cells did not have a significant impact on the original end points that we chose, which involved reversibility of a lack of blood supply to the heart, the volume of the left ventricle of the heart at the end of a contraction, and maximal oxygen consumption derived through a treadmill test," says Robert Simari, M.D., a cardiologist at Mayo Clinic in Rochester, Minn. He is chairman of the Cardiovascular Cell Therapy Research Network (CCTRN), the network of five academic centers and associated satellite sites that conducted the study. The CCTRN is supported by the National Heart, Lung, and Blood Institute, which also funded the study.

"But interestingly, we did find that the very simple measure of ejection fraction was improved in the group that received the cells compared to the placebo group by 2.7 percent," Dr. Simari says. Ejection fraction is the percentage of blood pumped out of the left ventricle during each contraction.

Study principal investigators Emerson Perin, M.D., Ph.D., and James Willerson, M.D., of the Texas Heart Institute, explain that even though 2.7 percent does not seem like a large number, it is statistically significant and means an improvement in heart function for chronic heart failure patients who have no other options.

"This was a pretty sick population," Dr. Perin says. "They had already had heart attacks, undergone bypass surgery, and had stents placed. However, they weren't at the level of needing a heart transplant yet. In some patients, particularly those who were younger or whose bone marrows were enriched in certain stem cell populations, had even greater improvements in their ejection fractions."

The average age of study participants was 63. The researchers found that patients younger than 62 improved more. Their ejection fraction improved by 4.7 percent. The researchers looked at the makeup of these patients' stem cells from a supply stored at a biorepository established by the CCTRN. They found these patients had more CD34+ and CD133+ type of stem cells in their mixture.

"This tells us that the approach we used to deliver the stems cells was safe," Dr. Simari says. "It also suggests new directions for the next series of clinical trials, including the type of patients, endpoints to study and types of cells to deliver."

Other co-authors of the study are Guilherme Silva, M.D., Deirdre Smith, Lynette Westbrook; and James Chen, all of the Texas Heart Institute, St. Luke's Episcopal Hospital, Houston; Carl Pepine, M.D., R. David Anderson, M.D., Christopher Cogle, M.D., and Eileen Handberg, Ph.D., all of the University of Florida School of Medicine, Gainesville; Timothy Henry, M.D., Jay Traverse, M.D., and Rachel Olson, all of the Minneapolis Heart Institute at Abbott Northwestern Hospital; Doris Taylor, Ph.D., and Claudia Zierold, Ph.D., both of the University of Minnesota School of Medicine, Minneapolis; Stephen Ellis, M.D., James Thomas, M.D., and Carrie Geither, all of The Cleveland Clinic Foundation, Ohio; David Zhao, M.D., Marvin Kornenberg, M.D., Antonis Hatzopoulos, Ph.D., Sherry Bowman, and Judy Francescon, all of Vanderbilt University School of Medicine, Tennessee; Dejian Lai, Ph.D., Sarah Baraniuk, Ph.D., Linda Piller, M.D., Lara Simpson, Ph.D., Judy Bettencourt, Shelly Sayre, Rachel Vojvodic, and Lemuel Moye, M.D., Ph.D., all of The University of Texas School of Public Health, Houston; A. Daniel Martin, Ph.D., of the University of Florida College of Public Health and Health Professions, Gainesville; Marc Penn, M.D., Ph.D., of Northeast Ohio Medical University, Akron; Saif Anwaruddin, M.D., of Penn Heart and Vascular Hospital of the University of Pennsylvania, Philadelphia; Adrian Gee, Ph.D., and David Aguilar, M.D., of Baylor College of Medicine, Houston; Catalin Loghin, M.D., of The University of Texas Medical School, Houston; and Sonia Skarlatos, Ph.D., David Gordon, M.D., Ph.D., Ray Ebert, Ph.D., and Minjung Kwak, Ph.D., all of the National Heart, Lung and Blood Institute, Bethesda, MD.

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Bone marrow stem cells can improve heart function, study suggests

Public release date: 26-Mar-2012 [ | E-mail | Share ]

Contact: Tara Womersley tara.womersley@ed.ac.uk 44-131-650-9836 University of Edinburgh

A breakthrough using cutting-edge stem cell research could speed up the discovery of new treatments for motor neurone disease (MND).

The international research team has created motor neurones using skin cells from a patient with an inherited form of MND.

The study discovered that abnormalities of a protein called TDP-43, implicated in more than 90 per cent of cases of MND, resulted in the death of motor neurone cells.

This is the first time that scientists have been able to see the direct effect of abnormal TDP-43 on human motor neurons.

The study, led by the University of Edinburgh's Euan MacDonald Centre for Motor Neurone Disease Research, was carried out in partnership with King's College London, Colombia University, New York and the University of San Francisco.

MND is a devastating, untreatable and ultimately fatal condition that results from progressive loss of the motor nerves motor neurones that control movement, speech and breathing.

Professor Siddharthan Chandran, of the University of Edinburgh, said: "Using patient stem cells to model MND in a dish offers untold possibilities for how we study the cause of this terrible disease as well as accelerating drug discovery by providing a cost-effective way to test many thousands of potential treatments."

The study, funded by the MND Association, is published in the journal PNAS

Original post:
Stem cell study aids quest for motor neurone disease therapies

Nature | Health

A Monsignor and Officer for Studies at the Pontifical Academy for Life called the cancellation a "sad event." Attendees are set to receive an official explanation

March 26, 2012|

By Ewen Callaway of Nature magazine

The Vatican has abruptly cancelled a controversial stem-cell conference that was set to be attended by the Pope next month.

The Third International Congress on Responsible Stem Cell Research, scheduled for 25-28 April, was to focus on clinical applications of adult and reprogrammed stem cells. But a number of the invited speakers, including Alan Trounson, president of the California Institute for Regenerative Medicine in San Francisco, and keynote speaker George Daley, a stem-cell scientist at Children's Hospital Boston in Massachusetts, are involved in research using human embryonic stem cells, which the Catholic Church considers unethical. The previous two congresses had also included scientists who worked on such cells, without generating much controversy.

Father Scott Borgman, secretary of the Church's Pontifical Academy for Life, one of the conference organizers, says that logistical, organizational and financial factors forced the cancellation, which was announced on 23 March. The academy weighs in on bioethical and theological issues that are relevant to Church teachings.

The Catholic News Agency, an independent news service based in Englewood, Colorado, quoted an unnamed academy member who called the cancellation an "enormous relief to many members of the Pontifical Academy for Life, who felt that the presence on its program of so many speakers, including the keynote speaker, committed to embryonic stem cell research, was a betrayal of the mission of the Academy and a public scandal".

"I think the only interpretation is that we are being censored. It is very disappointing that they are unwilling to hear the truth," says Trounson. He had hoped to provide a "balanced perspective" on the potential clinical applications of stem cells, both adult and embryonic.

Meanwhile, some European scientists, who had called for a boycott because they believed the conference unfairly maligned embryonic stem cell research, cheered its cancellation.

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Vatican Calls Off Stem-Cell Conference

Columbia, SC (WLTX) --What if your pet couldn't walk anymore? One Midlands vet is using stem cell therapy to help.

For Beth Phibbs it's almost like a turning back of the hands of time.

"I call her my little miracle dog, because she's doing things she used to do," said Phibbs. "Now she's not on any medication, and she can go up and down the steps and she runs and jumps and things that she used to do when she was five."

Phibbs has spent the last 13 years loving and looking after her pet dog Maggie, and when she pet began to develop arthritis and a limp she had to take action. But when the first treatments stopped working, Phibbs and Maggie had to look to another options, dog stem cell therapy.

"I had no idea that animals were able to have they type of procedures," she said.

Dr. Kenneth Banks a veterinarian with the Bank Animal Hospital, performed the surgery for Maggie using her own stem cells in the one day procedure.

Banks said the stem cell therapy not only cost less than some other options, but was less invasive and had a quicker recovery time as well.

Still with about three similar procedures under his belt, even he didn't expect to see a such change in maggie just 40 days after the surgery.

"I wasn't sure we were gonna get the results this fast, we were expecting results, maybe not a good as she's done. We're real happy with her results," said Banks.

Now, after three years on medication and walking with a limp, Maggie's getting used to a new way of life -- one with out pain in her golden years.

Continue reading here:
Midlands Vet Uses Stem Cell Therapy for Pets in Pain

March 27, 2012

According to a new study, using a patients own bone marrow may help repair damaged areas of the heart caused by heart failure.

Researchers found that left ventricular ejection fraction increased by 2.7 percent in patients who received stem cell therapy.

The study, which was presented at the American College of Cardiologys 61st Annual Scientific Session, revealed that the improvement in ejection fraction correlated with the number of CD34+ and CD133+ cells in the bone marrow.

This is the kind of information we need in order to move forward with the clinical use of stem cell therapy, Emerson Perin, MD, PhD, director of clinical research for cardiovascular medicine at the Texas Heart Institute and the studys lead investigator, said at the event.

The study included 92 patients who were randomly selected to receive stem cell treatment or placebo. The patients all had chronic ischemic heart disease and an ejection fraction of less than 45 percent along with heart failure.

Doctors placed a catheter in the hearts left ventricle to inject 3 ccs, or 100 million stem cells, into an average of 15 sites of the stem cell patients hearts.

The doctors used electromechanical mapping of the heart to measure the voltage in areas of the heart muscle and create a real-time image of the heart.

With this mapping procedure, we have a roadmap to the heart muscle, said Dr. Perin. Were very careful about where we inject the cells; electromechanical mapping allows us to target the cell injections to viable areas of the heart.

The trial was designed to determine whether left ventricular end systolic volume and myocardial oxygen consumption improved in patients who received stem cell treatment.

Read the original here:
Cell Therapy Improves Damaged Heart In Study

Public release date: 24-Mar-2012 [ | E-mail | Share ]

Contact: Traci Klein newsbureau@mayo.edu 507-284-5005 Mayo Clinic

CHICAGO -- A research network led by a Mayo Clinic physician found that stem cells derived from heart failure patients' own bone marrow and injected into their hearts improved the function of the left ventricle, the heart's pumping chamber. Researchers also found that certain types of the stem cells were associated with the largest improvement and warrant further study.

The results were presented today at the 2012 American College of Cardiology Meeting in Chicago. They will also be published online in the Journal of the American Medical Association.

This Phase II clinical trial, designed to test this strategy to improve cardiac function, is an extension of earlier efforts in Brazil in which a smaller number of patients received fewer stem cells. For this new network study, 92 patients received a placebo or 100 million stem cells derived from the bone marrow in their hips in a one-time injection. This was the first study in humans to deliver that many bone marrow stem cells.

"We found that the bone marrow cells did not have a significant impact on the original end points that we chose, which involved reversibility of a lack of blood supply to the heart, the volume of the left ventricle of the heart at the end of a contraction, and maximal oxygen consumption derived through a treadmill test," says Robert Simari, M.D., a cardiologist at Mayo Clinic in Rochester, Minn. He is chairman of the Cardiovascular Cell Therapy Research Network (CCTRN), the network of five academic centers and associated satellite sites that conducted the study. The CCTRN is supported by the National Heart, Lung, and Blood Institute, which also funded the study.

"But interestingly, we did find that the very simple measure of ejection fraction was improved in the group that received the cells compared to the placebo group by 2.7 percent," Dr. Simari says. Ejection fraction is the percentage of blood pumped out of the left ventricle during each contraction.

Study principal investigators Emerson Perin, M.D., Ph.D., and James Willerson, M.D., of the Texas Heart Institute, explain that even though 2.7 percent does not seem like a large number, it is statistically significant and means an improvement in heart function for chronic heart failure patients who have no other options.

"This was a pretty sick population," Dr. Perin says. "They had already had heart attacks, undergone bypass surgery, and had stents placed. However, they weren't at the level of needing a heart transplant yet. In some patients, particularly those who were younger or whose bone marrows were enriched in certain stem cell populations, had even greater improvements in their ejection fractions."

The average age of study participants was 63. The researchers found that patients younger than 62 improved more. Their ejection fraction improved by 4.7 percent. The researchers looked at the makeup of these patients' stem cells from a supply stored at a biorepository established by the CCTRN. They found these patients had more CD34+ and CD133+ type of stem cells in their mixture.

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Bone marrow stem cells improve heart function, study finds

Public release date: 24-Mar-2012 [ | E-mail | Share ]

Contact: Beth Casteel bcasteel@acc.org 240-328-4549 American College of Cardiology

CHICAGO -- A new study found that using a patient's own bone marrow cells may help repair damaged areas of the heart caused by heart failure, according to research presented today at the American College of Cardiology's 61st Annual Scientific Session. The Scientific Session, the premier cardiovascular medical meeting, brings cardiovascular professionals together to further advances in the field.

Millions of Americans suffer from heart failure, the weakening of the heart muscle and its inability to pump blood effectively throughout the body. If medications, surgery, or stents fail to control the disease, doctors often have few treatment options to offer.

This is the largest study to date to look at stem cell therapy, using a patient's own stem cells, to repair damaged areas of the heart in patients with chronic ischemic heart disease and left ventricular dysfunction. Researchers found that left ventricular ejection fraction (the percentage of blood leaving the heart's main pumping chamber) increased by a small but significant amount (2.7 percent) in patients who received stem cell therapy. The study also revealed that the improvement in ejection fraction correlated with the number of CD34+ and CD133+ cells in the bone marrow information that will be helpful in evaluating and designing future therapies and trials.

"This is the kind of information we need in order to move forward with the clinical use of stem cell therapy," said Emerson Perin, MD, PhD, director of clinical research for cardiovascular medicine at the Texas Heart Institute and the study's lead investigator.

This multi-center study was conducted by the Cardiovascular Cell Therapy Research Network and took place between April 2009 and 2011. At five sites, 92 patients were randomly selected to receive stem cell treatment or placebo. The patients, average age 63, all had chronic ischemic heart disease and an ejection fraction of less than 45 percent along with heart failure and/or angina, and were no longer candidates for revascularization.

"Studies such as these are able to be completed much faster because of the team approach of the network," said Sonia Skarlatos, PhD, deputy director of the division of cardiovascular sciences at the National, Heart, Lung and Blood Institute, and program director of the network.

Bone marrow was aspirated from the patients and processed to obtain just the mononuclear fraction of the marrow. In patients randomly selected to receive stem cell therapy, doctors inserted a catheter into the heart's left ventricle to inject a total of 3 ccs comprising 100 million stem cells into an average of 15 sites that showed damage on the electromechanical mapping image of the heart. Dr. Perin said the procedure is relatively quick and painless, involving only an overnight stay at the hospital.

The study used electromechanical mapping of the heart to measure the voltage in areas of the heart muscle and create a real-time image of the heart.

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Stem cell therapy possibly helpful in heart failure patients

25-03-2012 10:22 Dr Adelson aspirates bone marrow for concentration for stem cell therapy for musculoskeletal pain conditions

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bone marrow aspiration for stem cell therapy by Dr Adelson - Video

Published on Mar 25, 2012

(KOREA HERALD/ASIA NEWS NETWORK) - A joint research team from South Korea and Germany said on Friday they have created stem cells that have the potential to help treat people suffering from dementia and spinal cord trauma.

Scientists from Konkuk University and the Max Planck Institute said they have successfully used somatic cells from mice to create so-called induced neural stem cells (iNSCs) that can be cultivated for over a year under laboratory conditions.

The iNSCs have also been injected into the brains of mice and differentiated into various nerve cells without growing into malignant tumors.

'The discovery marks the first time ordinary somatic cells have been artificially engineered to become adult stem cells,' said Prof Han Dong Wook, a professor of stem cell biology at Konkuk, who led the research.

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Scientists develop stem cells that may help treat dementia

By Prue Salasky

3:48 p.m. EDT, March 23, 2012

UVA recently performed the first two stem cell transplants in Virginia, using non-embryonic stem cells from umbilical cord blood. The Stem Cell Transplant Program offers both bone marrow and stem cell transplants, with a focus on cord blood, to treat leukemia, lymphoma, Hodkin's disease and other blood diseases.

The outcome isn't known yet, but in both patients the stem cells began producing new cells 14 days after the transplant instead of the 24 to 28 days it usually takes.

The cord blood comes from placentas that otherwise would be discarded following childbirth; its benefits include sidestepping ethical issues of embryonic stem cells; they're easier and faster to collect than stem cells from other sources; and they are immune tolerant (this means that they won't attack other cells in the body and match doesn't have to be exact).

Speed is important because there is a narrow window of opportunity to perform a transplant when a patient's disease is in remission.

The program is led by Mary Laughlin, who heads up a team of 29, including 4 other transplant physicians who started seeing patients in September. The program had anticipated doing 15 transplants in first year; now expects to do 100.

Link:
Health Notes: UVA performs first stem cell transplants in Virginia

HOUSTON -

Doctors from the Texas Heart Institute at St. Luke's Episcopal Hospital have found that patients with heart failure may be able to repair the damaged areas of the heart with stem cells from the patient's own bone marrow.

Doctors presented the findings at the American College of Cardiologys 61st Annual Scientific Session Saturday.

The results are from a multi-center clinical study that measured the possible benefits of using a patients own bone marrow cells to repair damaged areas of the heart suffering from severe heart failure, a condition that affects millions of Americans.

The study, which was the largest such investigation to date, found that the hearts of the patients receiving bone marrow derived stem cells showed a small but significant increase in the ability to pump oxygenated blood from the left ventricle, the hearts main pumping chamber, to the body.

The expectation is that the study will pave the way for potential new treatment options and will be important to designing and evaluating future clinical trials.

This is exactly the kind of information we need to move forward with the clinical use of stem cell therapy, said Emerson Perin, MD, PhD, Director of Clinical Research for Cardiovascular Medicine at THI, and one of the studys lead investigators.

The bone-marrow derived stem cells are helpful to the injured heart when they are themselves biologically active, added Dr. James T. Willerson, the studys principal investigator and President and Medical Director of THI.

This study moves us one step closer to being able to help patients with severe heart failure who have no other alternatives.

The study was conducted by the Cardiovascular Cell Therapy Research Network, the national consortium to conduct such research funded by the National Institutes of Healths National Heart, Lung, and Blood Institute.

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Stem cell, heart heath study

ScienceDaily (Mar. 22, 2012) Researchers at the University of Bonn artificially derive brain stem cells directly from the connective tissue of mice.

Scientists at the Life & Brain Research Center at the University of Bonn, Germany, have succeeded in directly generating brain stem cells from the connective tissue cells of mice. These stem cells can reproduce and be converted into various types of brain cells. To date, only reprogramming in brain cells that were already fully developed or which had only a limited ability to divide was possible. The new reprogramming method presented by the Bonn scientists and submitted for publication in July 2011 now enables derivation of brain stem cells that are still immature and able to undergo practically unlimited division to be extracted from conventional body cells. The results have now been published in the current edition of the journal Cell Stem Cell.

The Japanese stem cell researcher Professor Shinya Yamanaka and his team produced stem cells from the connective tissue cells of mice for the first time in 2006; these cells can differentiate into all types of body cells. These induced pluripotent stem cells (iPS cells) develop via reprogramming into a type of embryonic stage. This result made the scientific community sit up and take notice. If as many stem cells as desired can be produced from conventional body cells, this holds great potential for medical developments and drug research. "Now a team of scientists from the University of Bonn has proven a variant for this method in a mouse model," report Dr. Frank Edenhofer and his team at the Institute of Reconstructive Neurobiology (Director: Dr. Oliver Brstle) of the University of Bonn. Also involved were the epileptologists and the Institute of Human Genetics of the University of Bonn, led by Dr. Markus Nthen, who is also a member of the German Center for Neurodegenerative Diseases.

Edenhofer and his co-workers Marc Thier, Philipp Wrsdrfer and Yenal B. Lakes used connective tissue cells from mice as a starting material. Just as Yamanaka did, they initiated the conversion with a combination of four genes. "We however deliberately targeted the production of neural stem cells or brain stem cells, not pluripotent iPS multipurpose cells," says Edenhofer. These cells are known as somatic or adult stem cells, which can develop into the cells typical of the nervous system, neurons, oligodendrocytes and astrocytes.

The gene "Oct4" is the central control factor

The gene "Oct4" is a crucial control factor. "First, it prepares the connective tissue cell for reprogramming, later, however, Oct4 appears to prevent destabilized cells from becoming brain stem cells" reports the Bonn stem cell researcher. While this factor is switched on during reprogramming of iPS cells over a longer period of time, the Bonn researchers activate the factor with special techniques for only a few days. "If this molecular switch is toggled over a limited period of time, the brain stem cells, which we refer to as induced neural stem cells (iNS cells), can be reached directly," said Edenhofer. "Oct4 activates the process, destabilizes the cells and clears them for the direct reprogramming. However, we still need to analyze the exact mechanism of the cellular conversion."

The scientists at the University of Bonn have thus found a new way to reprogram cells, which is considerably faster and also safer in comparison to the iPS cells and embryonic stem cells. "Since we cut down on the reprogramming of the cells via the embryonic stage, our method is about two to three times faster than the method used to produce iPS cells," stresses Edenhofer. Thus the work involved and the costs are also much lower. In addition, the novel Bonn method is associated with a dramatically lower risk of tumors. As compared to other approaches, the Bonn scientists' method stands out due to the production of neural cells that can be multiplied to a nearly unlimited degree.

Low risk of tumor and unlimited self renewal

A low risk of tumor formation is important because in the distant future, neural cells will replace defective cells of the nervous system. A vision of the various international scientific teams is to eventually create adult stem cells for example from skin or hair root cells, differentiate these further for therapeutic purposes, and then implant them in damaged areas. "But that is still a long way off," says Edenhofer. However, the scientists have a rather urgent need today for a simple way to obtain brain stem cells from the patient to use them to study various neurodegenerative diseases and test drugs in a Petri dish. "Our work could form the basis for providing practically unlimited quantities of the patient's own cells." The current study was initially conducted on mice. "We are now extremely eager to see whether these results can also be applied to humans," says the Bonn scientist.

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New shortcut for stem cell programming

24-03-2012 07:46 This is the harvest of adipose tissue for combination with bone marrow aspirate concentrate for stem cell therapy

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Adipose harvest for stem cell therapy by Dr Adelson - Video

3/25/2012 10:50 AM ET (RTTNews) - An important clinical trial, which evaluated the use of autologous bone-marrow-cell therapy in patients with chronic ischemic heart failure, has failed to meet the prespecified end points of improvement in most measures of heart function, according to the results presented at the American College of Cardiology 2012 Scientific Sessions.

The trial dubbed, FOCUS - a phase II study, is the largest study to date to investigate if a patient's own bone marrow cells improved myocardial perfusion, reduced left ventricular end-systolic volume or enhanced maximal oxygen consumption in patients with coronary artery disease or LV dysfunction, and limiting heart failure or angina. The FOCUS trial was undertaken by the National Heart, Lung, and Blood Institute-sponsored Cardiovascular Cell Therapy Research Network.

Ninety two patients with chronic ischemic heart disease , having a left ventricular ejection fraction of 45% or less, a perfusion defect by single-photon emission tomography, or SPECT, who were no longer candidates for revascularization, were enrolled in the trial. Sixty one patients in the study were administered bone marrow cells through transendocardial injections while thirty one patients were administered placebo.

An assessment of primary endpoints at 6 months has revealed that there is no statistically significant difference between the treatment group and placebo arm in left ventricular end-systolic volume assessed by echocardiography, maximal oxygen consumption, and reversibility on SPECT. The secondary outcomes, including percent myocardial defect, total defect size, fixed defect size, regional wall motion, and clinical improvement, also has not exhibited any difference between the two arms.

However, according to the study authors, exploratory analyses have revealed that left ventricular ejection fraction improved in the treatment group compared with the placebo group by 2.7%.

The authors, led by Emerson Perin, concluded that the findings provide evidence for further studies to determine the relationship between the composition and function of bone marrow product and clinical end points. Understanding these relationships will improve the design and interpretation of future studies of cardiac cell therapy, the authors noted.

The results were published online March 24 in the Journal of the American Medical Association.

by RTT Staff Writer

For comments and feedback: editorial@rttnews.com

Read more:
Bone Marrow Stem Cell Therapy Trial - Clues, But No Answers

(HealthDay News) -- A new method to prevent recurrence of deadly glioblastoma brain cancer shows promise, say U.S. scientists.

Radiation can temporarily shrink a glioblastoma tumor, but the cancer nearly always recurs within weeks or months. Few people with this type of brain cancer survive more than two years after diagnosis.

In a study on mice, Stanford University School of Medicine researchers found that blocking access to oxygen and nutrients prevents tumor recurrence.

The first step, they said, was discovering that tumors blasted with radiation use a secondary pathway to generate blood vessels needed for regrowth.

"Under normal circumstances, this pathway is not important for growth of most tumors," senior author Martin Brown, a professor of radiology, said in a Stanford news release. "What we hadn't realized until recently is that radiation meant to kill the cancer cells also destroys the existing blood vessels that nourish the tumor. As a result, it has to rely on a backup blood delivery pathway."

The Stanford team used a molecule called AMD3100 to block the secondary glioblastoma tumor growth process in mice.

The study was published online Feb. 22 in the Journal of Clinical Investigation. Read more...

Immunice for Immune Support

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Biotechnology is undoubtedly the technology of the future for it not only presents exceptional opportunities, but also gives hope for a better future with better diagnosis and treatment of diseases. In terms of potential and growth, it is not much different from the mystical dragon, and since 2012 is the year of the dragon, it is expected that it would bring in loads of good news and prosperity for this new branch of science.
The good news
For biotechnology, the last year has been strong and monumental with Tax Incentive Legislation being passed in Australia and a very strong and consistent growth in the sector which was recently followed by good news, the Senate Inquiry of the gene patents bill. Since the last year, the Australian Biotechnology has been included amongst the fifth most innovative biotech nations in the world (according to the Scientific American World View). This trend is continuing in the current year, as Australia has shown great potential in developing biotech related agricultural, medical and even environmental research.
The companies of Australian Biotech are confident that the New Year would definitely be the best and until now with tremendous growth in the sector it has proved this. Even the new startup biotech companies in Australia now stand a better chance with the Tax Incentive’s 45% refundable component, even the large corporations would now be able to reduce their R&D expenses by as much as 10%. Such a healthy growth favoring environment has allowed the Australian Biotech companies to make a mark globally and have a steadily rising status even in the competitive markets of US and Europe.
Conclusion
The Australian Biotech industry now needs to revamp itself and embrace a more authentic and transparent management. There should be better communication between the management and the stakeholders. The opportunities are in plenty and the industry environment very supportive, hence the companies should make the most of it and truly let the biotech dragon rise in this year of the dragon.

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Ethanol Producer Magazine

Mendel Biotechnology, BP Biofuels to conduct miscanthus trials Ethanol Producer Magazine based dedicated energy crop developer Mendel Biotechnology Inc., together with its wholly owned subsidiary Mendel Bioenergy Seeds, and BP Biofuels have signed a four-year agreement to conduct a demonstration field trial of Mendel's trademarked ...

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