Berkeley Daily Planet

Stakeholders Weigh in on UC Berkeley GMO Complex Berkeley Daily Planet By Richard Brenneman A forum critical of UC Berkeley's plans to ramp up genetic engineering research at a planned massive new second campus of Lawrence Berkeley National Laboratory in Richmond drew a capacity crowd to the David Brower Center Thursday ...

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Via Scoop.it – inPharmatics

On March 29, 2012, Oracle announced that it has agreed to acquire ClearTrial, a leading provider of cloud-based Clinical Trial Operations and analytics products that make the planning, sourcing, and tracking of clinical projects and financial performance…

Via http://www.oracle.com

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The California stem cell agency has issued a statement from its president, Alan Trounson, concerning the cancellation of a Vatican stem cell conference at which Trounson was scheduled to speak.

According to the Catholic News Agency, the meeting was terminated because of the scheduled appearances of researchers such as Trounson, who support hESC research. The Catholic church opposes such research.

The news agency last week quoted one Vatican insider as saying the conference had generated a scandal within the higher echelons of the church. However, the Vatican later claimed it was cancelling the meeting because of "organizational, logistical and economic factors."

Trounson's statement said,

"I am disappointed that the decision was made to cancel the conference because it offered the opportunity for a constructive dialogue on all types of stem cell research.

"Open dialogue can enhance the field as a whole and accelerate our efforts to provide new therapies for patients in need."

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

The proposed $17.8 million operational budget for the California stem cell agency will receive more public scrutiny at a meeting next week in San Francisco, with additional public participation sites ranging from Boston to La Jolla.

The spending plan for 2012-13 is 7.2 percent higher than the estimated expenditures for the current year. By law, CIRM's budget is not subject to cuts by the legislature or the governor, who are trying to find solutions to the state's ongoing financial crisis.

Next week's hearing will be the last before approval of the budget in late May by the directors of the $3 billion research program. No significant changes are expected from the document to be presented on Monday.

Public sites where the public can participate in the CIRM directors' Finance Subcommittee meeting include South San Francisco, Stanford, Berkeley and Boston. You can find more information about the specific locations on the meeting agenda, but if you are interested in attending you need even more details from CIRM prior to the actual meeting. Email a request to info@cirm.ca.gov.

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

Myjoyonline.com

Adopt Genetically Modified crops to increase yields - Prof Nketsia-Tabiri Myjoyonline.com Professor Josephine Nketsia-Tabiri, Director of Biotechnology and Nuclear Agriculture Research Institute (BNARI), has called on farmers to embrace the application of Genetically Modified Organisms (GMOs) to increase crop yields and help sustain ... and more »

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http://news.google.com/news?q=biotechnology&output=rss

Is your child about to lose her milk tooth? Instead of throwing it away, you can now opt to use it to harvest stem cells in a dental stem cell bank for future use in the face of serious ailments. Now thats a tooth fairy story coming to life.

Still relatively new in India, dental stem cell banking is fast gaining popularity as a more viable option over umbilical cord blood banking.

Stem cell therapy involves a kind of intervention strategy in which healthy, new cells are introduced into a damaged tissue to treat a disease or an injury.

The umbilical cord is a good source for blood-related cells, or hemaotopoietic cells, which can be used for blood-related diseases, like leukaemia (blood cancer). Having said that, blood-related disorders constitute only four percent of all diseases, Shailesh Gadre, founder and managing director of the company Stemade Biotech, said.

For the rest of the 96 percent tissue-related diseases, the tooth is a good source of mesenchymal (tissue-related) stem cells. These cells have potential application in all other tissues of the body, for instance, the brain, in case of diseases like Alzheimers and Parkinsons; the eye (corneal reconstruction), liver (cirrhosis), pancreas (diabetes), bone (fractures, reconstruction), skin and the like, he said.

Mesenchymal cells can also be used to regenerate cardiac cells.

Dental stem cell banking also has an advantage when it comes to the process of obtaining stem cells.

Obtaining stem cells from the tooth is a non-invasive procedure that requires no surgery, with little or no pain. A child, in the age group of 5-12, is any way going to lose his milk tooth. So when its a little shaky, it can be collected with hardly any discomfort, Savita Menon, a pedodontist, said.

Moreover, in a number of cases, when an adolescent needs braces, the doctor recommends that his pre-molars be removed. These can also be used as a source for stem cells. And over and above that, an adults wisdom tooth can also be used for the same purpose, Gadre added.

Therefore, unlike umbilical cord blood banking which gives one just one chance - during birth - the window of opportunity in dental stem cell banking is much bigger.

Continued here:
Your child’s milk tooth can save her life

Dublin - Research and Markets (http://www.researchandmarkets.com/research/bac5f5c5/strategic_developm) has announced the addition of the "Strategic Development of Neural Stem & Progenitor Cell Products" report to their offering.

Overview: Neurogenesis is the process by which neurons are created. This process is most active during pre-natal development when neurogenesis is responsible for populating the growing brain. Neural stem cells (NSCs) are the self-renewing, multipotent cells that differentiate into the main phenotypes of the nervous system. These cell types include neurons, astrocytes, and oligodendrocytes. Neural progenitor cells (NPCs) are the progeny of stem cell division that normally undergo a limited number of replication cycles in vivo.

The terms neuronal and neural also need to be defined. Technically speaking, neuronal means pertaining to neurons, and neural means pertaining to nerves, which are the cordlike bundles of fibers made up of neurons. Since both terms ultimately are descriptive of neurons, the scientific community uses the terms "neuronal" and "neural" interchangeably. The complexity of this issue is explored from a marketing perspective within this report.

In 1992, Reynolds and Weiss were the first to isolate neural stem cells from the striatal tissue of adult mice brain tissue, including the subventricular zone, which is a neurogenic area. Since then, neural progenitor and stem cells have been isolated from various areas of the adult brain, including non-neurogenic areas like the spinal cord, and from other species, including humans. During the development of the nervous system, neural progenitor cells can either stay in the pool of proliferating undifferentiated cells or exit the cell cycle and differentiate.

This market report focuses on recent advances in NSC research applications, explores research priorities by market segment, highlights individual labs and end-users of neuronal stem cell research products, explores the competitive environment for NSC research products, and provides 5-year growth and trend analysis. It provides detailed guidance for companies that wish to offer strategically positioned NSC research products, including cells, kits, assays, and related media and reagents.

This Market Report Includes:

- Recent advances in NSC research applications

- Research priorities by market segment

- Competitive analysis of NSC research supply companies

- Segmentation of existing NSC products

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Research and Markets: Strategic Development of Neural Stem & Progenitor Cell Products

The Catholic Church has never had a particularly easy relationship with science. After all, this is the institution that sentenced Galileo Galilei as a heretic for his theories on the universe during the Roman Inquisition. Two thousand years later, the church forgave Galileo and called the whole misunderstanding a tragic mutual incomprehension but it remains safe to say the Vatican doesnt have a great track record when it comes to empirical open-mindedness.

So onlookers were surprised when the Vatican announced it would be hosting a global conference on the highly controversial issue of stem-cell research in Rome over four days in late April. The church held a similar conference in 2010 and 2011, which focused on its recommendation that stem-cell research should be limited to adult cells that can be harvested from live donors, not embryonic cells that destroy the source. But this years conference schedule featured some of the worlds foremost experts in embryonic research as keynote speakersleading some scientists to think that the Vatican might actually be looking for enlightenment on the topic.

That was not exactly case. Instead, the Vatican seems to have hoped that by including embryonic researchers in the program, it would appear that these scientists actually endorsed the Vaticans stance.

It might have worked to some extent, but after some of the speakers declined to censor their speeches, the Vatican abruptly canceled the conference altogether. According to the conference website, the event was canceled due to serious economic and logistic-organizational reasons that have completely jeopardized the success of the 3rd International Congress on Responsible Stem Cell Research. The scientists who were planning to attend say they are being stifled instead. I think the only interpretation is that we are being censored, Alan Trounson, president of the California Institute for Regenerative Medicine in San Francisco, said in a statement. It is very disappointing that they are unwilling to hear the truth.

Just what was the Vatican thinking? Inviting embryonic stem-cell researchers to a conference and then denying them the right to talk about their field of expertise was a major gamble. Had the speakers agreed to avoid reference to embryonic research, it would have given the disingenuous impression that they endorse the Holy Sees recommendation on adult stem-cell research only. Did the Vatican really think they could control the scientific community? Apparently so. Father Scott Borgman of the Pontifical Academy for Life, which co-organized the conference, had reportedly asked the speakers to limit their discussions to adult stem-cell research only. George Daly, a leading embryonic researcher with the Childrens Hospital in Boston, says he was actually told not to make embryonic researchhis field of expertisea focal point of his talk. When he told Borgman that he would still be touching on the topic in a historical context, higher-ups in the Vatican reportedly panicked. I had been encouraged to think that the Congress would be a forum for discussion of many areas of common interest to the Vatican and stem cell scientists, regardless of the disagreements over embryonic stem cells, Daly told The Daily Beast. We should all agree that clinical trials of new medical treatments based on stem cells should proceed according to rigorous principles to ensure patients are kept as safe as possible and free from exploitation. And we should all agree that premature claims of therapeutic efficacy and direct marketing of unproven interventions to vulnerable patients is a threat to legitimate attempts to develop experimental stem cell medicines.

Pope Benedict looks on during the mass in solemnity of the chair of St. Peter with new Cardinals in St. Peter's basilica at the Vatican on February 19, 2012. The Vatican stands by its decision to cancel the controversial conference as having a purely business motive. , Alberto Pizzoli, AFP / Getty Images

With the cancelation of the event, discourse between the two diverse entities will not have a venue. One Vatican official told the Catholic News Service that many of the Vaticans leaders were secretly glad the conference failed. I am infinitely relieved that the church has avoided a major blunder which would have confused the faithful for decades to come, the unnamed source said. The Holy Spirit has certainly shown to be present through those faithful members who drew attention to the ambiguity of the choice of speakers. I hope and pray that a review will be affected of the basis on which these congresses are planned.

Some stem-cell researchers are also relieved the conference wont go on. I personally am very uncomfortable with a scientific meeting run by a church, and one at which only certain types of science and scientists are allowed to attend, blogged Paul Knoepfler, an associate professor of Cell Biology and Human Anatomy at UC Davis School of Medicine who blogs about stem cell research at IPCell.com. Also I cant help but wonder, what would be the reaction if someone like Daley spent a few minutes of his talk discussing his embryonic cell research in a very nonconfrontational way? Would he be tasered or drop through some trap door straight to Hell?

Still, Knoepfler was hopeful. I view the canceled Vatican stem-cell meeting as a missed opportunity for a very much needed, open dialogue about stem cells, he told The Daily Beast. More specifically, I believe the reasons for the cancellation reflect an anti-scientific attitude by the highest level of Vatican leaders. More simply put, the attitude might be summed up by the phrase If you do not think like us, you are not welcome at our meeting, and well go so far as to cancel the whole thing to avoid your presence.

Inviting embryonic stem-cell researchers to a conference and then denying them the right to talk about their field of expertise was a major gamble.

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Vatican’s Stem-Cell Censorship Sham

As the basketball frenzy that accompanies March Madness draws to the fever pitch of the Final Four, it brings to mind that basketball is a high contact sport. A quick peek at the NBA injured list reveals a catalog of breaks and tears that affect tendons, ligaments and bones.

The pressure to improve performance and search for quick recoveries has led some celebrity athletes to seek out stem cell treatments overseas and in the U.S. Among NBA players to get stem cell treatments are Jason Kidd, Tracy McGrady, Amar Stoudemire, Allan Houston and Kenyon Martin, according to a Sports Illustrated article.

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Dragoo said in a phone interview that the publicity has actually had a negative impact on the development of clinically proven stem cell therapies for orthopedic medicine and how it is perceived. Because of this market pressure, private clinics have been offering stem cells treatments both here in the USA as well as around the world. Often, these treatments have not been studied and are not regulated in any way. FDA [U.S. Food and Drug Administration] regulations have also severely limited new clinical trials in stem cell therapy in the USA.

The ethical debate of using embryonic stem cells taken from fetuses has been sidestepped to some extent by the viability of adult stem cells for stem cell therapy. Although the FDA permits cells being extracted from individuals and transformed into stem cells, and re-inserted back into the same person, it requires that the conversion involve no more than water, preservatives and storage products. Anything more than that, the FDA policy states, would be classified as a drug therapy and need to go through the proper application protocol.

But a much-awaited decision by the U.S. District Court in Washington, D.C. expected in May that may resolve a four-year-old battle between the FDA and Regenerative Science in Colorado could represent a sea change in how autologous adult stem cell treatments are regulated. The FDA is seeking to prevent the company from providing autologous adult stem cell treatment for musculoskeletal and spinal injuries. If the FDA were to lose, anyone with a medical license could develop autologous stem cells and inject them back into patients, without any regulatory oversight, according to a Cell Press article.

Although stem cells are the focus of numerous clinical trials, they are mainly for cancer and rare diseases, most are being conducted outside the United States. While there have been some developments for sports medicine applications produced by research from academic institutions, there have been no clinical trials for stem cell treatments in sports medicine in the United States because of the FDAs reservations about using adult stem cells. Despite the laxer regulations in Japan, China and Europe, its not in the financial interest of companies there to spend the money to do clinical trials if they dont have to.

Among the most interesting applications for orthopedic medicine are the restoration of articular cartilage and patching defects in joint cartilage, with the hope of resurfacing arthritic joints in the future, Dragoo said. Stanford is preparing to initiate its own clinical trial next year looking at inducible stem cells.

This technique takes adult cells and makes them young again by inserting four genes, which makes the cells immature and allows them to be directed into different types of tissues, Dragoo said.

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Basketball’s influence on stem cell treatments in sports medicine

28-03-2012 10:17 Dr Joshua Hare is Professor of Medicine and Director of the Interdisciplinary Stem Cell Institute at the University of Miami. The interview was conducted on 25 March 2012 at the American College of Cardiology's (ACC's) 61st Annual Scientific Session & Expo in Chicago. See more ACC.12 Coverage: http://www.getinsidehealth.com

Originally posted here:
Stem cell therapy for the repair of myocardium in heart failure patients - Video

ROCKVILLE, Md., March 28, 2012 /PRNewswire/ --Neuralstem, Inc. (NYSE Amex: CUR) announced that safety results from the first 12 patients with amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease) to receive its stem cells were reported online in the peer-reviewed publication, STEM CELLS, on March 13th. "Lumbar Intraspinal Injection of Neural Stem Cells in Patients with ALS: Results of a Phase I Trial in 12 Patients" (http://www.ncbi.nlm.nih.gov/pubmed/22415942.1) reports that one patient has shown improvement in his clinical status, even though researchers caution that the study was not designed to show efficacy. Additionally, there was no evidence of accelerated disease progression due to the intervention in any of the 12 patients, who were followed from 6-18 months after they were transplanted with the cells. All of the patients, who received transplants in the lumbar (lower back) region, tolerated the treatment without any long-term complications related to either the surgery or the cells.

(Logo: http://photos.prnewswire.com/prnh/20061221/DCTH007LOGO )

The 12 patients, part of the ongoing Phase I trial to evaluate the safety of Neuralstem's stem cells and transplantation procedure in patients with ALS, were the first in the world to receive intraspinal stem cell injections. Results from these patients were also were reported at the American Academy of Neurology Annual Meeting last September.

Based on a positive safety assessment, the trial has now been approved by the FDA to progress to transplanting ALS patients in the cervical (upper back) region of the spine, where the goal is to protect the motor neurons which affect respiratory function, and possibly prolong life. The fourteenth patient was transplanted earlier this month. All patients were treated at Emory University Hospital in Atlanta, Georgia.

"For these first 12 patients, we have met the objective of the Phase I trial, demonstrating safety for both the procedure of intraspinal injection and the presence of the neural stem cells in the spinal cords of ALS patients," said Jonathan Glass, MD, lead author of the publication. "We are encouraged by these results and have now advanced our trial to injections into the cervical spinal cord, targeting the motor neurons that control respiratory function." Dr. Glass is Professor of Neurology and Pathology at Emory University School of Medicine, as well as the Director of the Emory ALS Center.

"This important peer-reviewed publication reinforces our belief that we have demonstrated a safe, reproducible and robust route of administration into the spine for these spinal cord neural stem cells," said Eva Feldman, MD, PhD, Director of the A. Alfred Taubman Medical Research Institute and Director of Research of the ALS Clinic at the University of Michigan Health System. "The publication covers data up to 18 months out from the original surgery. However, we must be cautious in interpreting this data, as this trial was neither designed nor statistically powered to study efficacy." Dr. Feldman is senior author on the study, principal investigator (PI) of the ALS trial and serves as a consultant to Neuralstem as part of her University of Michigan activities.

"As this article points out, our experience in the lumbar spinal cord has been overwhelmingly positive," commented Karl Johe, PhD, study author and Neuralstem Chairman and Chief Scientific Officer. "We have already transplanted two patients in the cervical spinal cord, where we believe we can affect patients' lives the most by improving their breathing. We are in active discussions with the FDA to increase the number of cells and the number of injections as well."

"We wish to thank the teams at Michigan and Emory for the tireless efforts required to refine this breakthrough method of administration of our neural stem cells. We'd also like to thank the patients and families involved in the trial," said Richard Garr, Neuralstem CEO and President. "The progress we have made to date is both substantial, and a true team effort."

About the Study

Safety results were reported on the first 12 patients in an ongoing Phase I study to evaluate the safety of Neuralstem's spinal cord stem cells (HSSC's), as well as the transplantation technique, in the treatment of ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease).

Read more from the original source:
Neuralstem ALS Stem Cell Trial Interim Results Reported in the Journal, STEM CELLS

Arkansas State University-Newport will host a Bone Marrow Donor Drive on campus Thursday, March 29 from 10am until 7pm and Saturday, March 31 from 9am until 1pm in the Student/Community Center, Merchants & Planters Insurance and Investments room. A bone marrow transplant is a lifesaving treatment for people with leukemia, lymphoma and many other diseases. First, patients undergo chemotherapy and sometimes radiation to destroy their diseased marrow. Then a donor's healthy blood-forming stem cells are transfused directly into the patient's bloodstream, where they can begin to function and multiply. For a patient's body to accept these healthy cells, the patient needs a donor who is a close match. Seventy percent of patients cannot find a matching donor within their family and depend on the national registry to find an unrelated bone marrow donor. Even with a registry of millions, 6 out of 10 patients NEVER receive the lifesaving transplant they need. Donors of all ethnicities are needed to change this. To see if you can be a bone marrow donor and to read about the process of testing and donating, go to http://www.dkmsamericas.org and click on Get Educated.

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ASUN to host Bone Marrow Donor Drive

Displaying rare unanimity on an issue, the full U.S. 9th Circuit Court of Appeals on Tuesday rejected a request by the federal government thatit reconsidera rulingthat most bone marrow donors can be compensated for providing life-saving marrow stem cells from their blood.

A three-judge panel of the appeals court ruled on Dec. 1 that the process of harvesting marrow cells by filtering a donor's blood wasn't covered by the 1984 National Organ Transplant Act's prohibitionof payment for organs or organ parts.The statute was enacted by Congress before the blood-filtering process was developed and donors were subjected to painful and medically risky surgical extraction of marrow by insertion of a siphoning needle into the hip bone. Compensation for that form of donation remains illegal.

Atty. Gen. Eric H. Holder Jr., on behalf of the federal government, petitioned the court in Januaryfor a new hearing by an 11-judge panel. Department of Justice lawyers argued that the December ruling ignored the clear intent of Congress to prevent money from influencing donation decisions.

The 9th Circuit panel said in its latest ruling thatall 25 active judges on the court were informed of the government's request and none called for a vote on it, signaling their agreement with the December decision. That unusualaccord among the judges who span a broad ideological spectrum might also indicate that the U.S. Supreme Court will be unlikely to take the case for review.

The lawsuit challenging the ban on bone marrow compensation was brought by a group of cancer patients and their families, as well as a marrow transplant specialist and a California nonprofit organization, MoreMarrowDonors.org, aiming to expand the registry of available donors by offering up to $3,000 in housing assistance or scholarships for promising genetic matches.

Violation of the organ transplant act's prohibition on sales of organs or parts thereofcarries heavy fines and up to five years in prison.The 1984 act defined bone marrow as an organ part, while the 9th Circuit's ruling said it was a blood part and not subject to theban on compensation.

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Appeals court stands united on compensation for bone marrow donors

ScienceDaily (Mar. 26, 2012) A breakthrough using cutting-edge stem cell research could speed up the discovery of new treatments for motor neuron disease (MND).

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

Role of protein

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 said Professor Siddharthan Chandran, Director of the University's Euan MacDonald Centre for MND Research.

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 neuron 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 Neuron Disease Research, was carried out in partnership with King's College London, Columbia University, New York and the University of San Francisco.

Motor neuron disease

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

The study, funded by the MND Association, is published in the journal Proceedings of the National Academy of Sciences.

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Stem cell study aids quest for motor neuron disease therapies

MADISON, Wis., March 28, 2012 /PRNewswire/ --Cellular Dynamics International, Inc. (CDI) today announced an expansion of its existing distribution agreement with iPS Academia Japan, Inc. to include iCell Neurons and iCell Endothelial Cells. The original distribution agreement, announced on June 8, 2011, covered the distribution of CDI's iCell Cardiomyocytes, the first commercially available product based on induced pluripotent stem cells (iPSCs), in Japan.

CDI is the world's largest manufacturer of human cellular tools for drug discovery and safety derived from iPSCs. The company currently manufactures iCell Cardiomyocytes, iCell Neurons and iCell Endothelial Cells with several other cell types, including liver cells, in development.

iPS Academia Japan was originally established to manage the patents and technology arising from the work of Shinya Yamanaka, MD, PhD of Kyoto University. CDI was the first foreign company granted a license to Yamanaka's iPSC patent portfolio by iPS Academia Japan, announced in May 2010.

"The reliability and consistent quality of CDI's cardiomyocytes have proven to be a valuable product offering to our academic and pharmaceutical customers," said Shosaku Murayama, President and CEO of iPS Academia Japan. "We're already seeing demand for additional human cell types manufactured by CDI by our Japanese customers."

Robert Palay, CEO and chairman of the board of CDI, noted, "We view the expansion of our distribution agreement with iPS Academia Japan as a vote of confidence in our ability to provide human iPSC-derived cells in the quantity, quality and purity required for scientists to realize the full potential of their experiments. We look forward to future growth of our relationship with iPS Academia Japan as we launch new human cell types and in vitro human disease models."

About Cellular Dynamics International Cellular Dynamics International, Inc. (CDI) is a leading developer of next-generation stem cell technologies for drug development, cell therapy, tissue engineering and organ regeneration. CDI harnesses its unique manufacturing technology to produce differentiated tissue cells from any individual's stem cell line in industrial quality, quantity and purity. CDI is accelerating the adoption of pluripotent stem cell technology, adapting its methods to fit into standard clinical practice by the creation of individual stem cell lines from a standard blood draw. CDI was founded in 2004 by Dr. James Thomson, a pioneer in human pluripotent stem cell research at the University of Wisconsin-Madison. CDI's facilities are located in Madison, Wisconsin. See http://www.cellulardynamics.com.

About iPS Academia Japan, Inc. iPS Academia Japan, Inc. (AJ) is an affiliate of Kyoto University, and its main role is, among other activities, to manage and utilize the patents and other intellectual properties held/controlled by Kyoto University and other universities in the field of iPSC technologies so that the research results contribute to health and welfare worldwide.

AJ was established in Kyoto in June 2008. AJ's patent portfolio consists of about 60 patent families (the total number of patent applications is about 220 cases) in the iPSC technology as of March 2012, and about 50 license arrangements have been executed with domestic or international enterprises. See http://ips-cell.net.

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Cellular Dynamics Expands Distribution Agreement with iPS Academia Japan, Inc. to Include Distribution of iCell ...

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

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Stem cell study aids quest for motor neurone disease therapies