Archive for May, 2012

By Carolyn Y. Johnson, Globe Staff

Two teams of Boston scientists have developed new ways to turn stem cells into different types of lung tissue, surmounting a major hurdle for scientists trying to harness the power of stem cell biology to study and develop treatments for major lung diseases.

One team then used skin cells from cystic fibrosis patients to create embryonic-like stem cells, then working in lab dishes used those cells to grow tissue that lines the airways and contains a defect responsible for the rare, fatal disease. The technique -- essentially a recipe for growing such lung tissue -- could provide a powerful platform to screen drugs and study the biology of the disease.

Growing lung tissue in the laboratory has long been a goal of stem cell scientists, but has been more technically difficult than growing other types of tissues, such as brain cells or heart cells. Such lung tissue is valuable because it could be used to screen potential drugs and more closely probe the problems that underlie diseases such as asthma, emphysema, and rare genetic diseases. Such techniques may also one day help researchers grow replacement tissues and devise ways to restore or repair injured lung tissue.

A team led by Massachusetts General Hospital researchers created lung tissue from a patient with the genetic mutation that most commonly underlies cystic fibrosis and researchers hope the technique will also be a powerful tool to study other diseases that affect the airway tissue, such as asthma and lung cancer. The other team, led by Boston University School of Medicine scientists, was able to derive cells that form the delicate air sacs of the lung from mouse embryonic stem cells. The team is hoping to refine the recipe for making the cells so that they can be used to derive lung tissue from a bank of 100 stem cell lines of patients with lung disease. Both papers were published Thursday in the journal Cell Stem Cell.

Vertex Pharmaceuticals, a Cambridge biotechnology company, earlier this year received approval for Kalydeco -- the first drug to directly target the underlying cause of cystic fibrosis. That compound was discovered by screening massive numbers of potential drugs against cells engineered to carry the same defect that underlies cystic fibrosis.

We had to use engineered cells, and certainly using more native human cells ... would be potentially beneficial, said Dr. Frederick Van Goor, head of biology for Vertexs cystic fibrosis research program. We had to rely on donor tissue obtained from patients with cystic fibrosis, and its a bit more challenging, because the number of donor lungs you can get and the number of cells you can derive from there are more limited.

Van Goor said it was too soon to say whether the company would use the new technology in screening, but noted that the tests the company had used to determine whether a drug was likely to work against the disease had, in some cases, given scientists false leads. Some molecules that worked on the engineered cells did not work in the complicated biology of the lung.

Its a significant event for the lung field, said Dr. Thiennu Vu, associate professor of medicine at the University of California San Francisco, who was not involved in the research. She added that much work remains before such cells could be used to repair or replace damaged tissue, and even before such cells would necessarily be useful for drug screening. It will be important, she said, to refine the recipe to ensure that the technique yields pure populations of the specific types of functional lung cells.

In the competitive world of science, where credit for being the first to do something is crucially important, the two research teams accomplishments are an unusual example of competitors turning into collaborators -- forging a relationship that both teams felt helped speed up progress.

Originally posted here:
Boston scientists grow lung tissue from cystic fibrosis patients’ skin cells

NEW YORK--(BUSINESS WIRE)--

Pfizer Inc announced today that the Phase 3 INTORSECT (B1771003) study, evaluating TORISEL (temsirolimus) in patients with advanced renal cell carcinoma (RCC) whose disease had progressed on or after SUTENT (sunitinib malate) therapy, did not meet the primary endpoint of prolonging progression free survival (PFS) when compared to sorafenib. Although PFS was numerically higher in patients treated with temsirolimus, the difference was not statistically significant. Overall survival, a secondary endpoint in the study, showed statistical significance favoring patients randomized to the sorafenib arm. Adverse events in this study were consistent with the known safety profiles for both drugs. Full efficacy and safety data from this study will be presented at an upcoming major medical congress.

Approximately 270,000 people worldwide are diagnosed with renal cell cancer every year with about 20 percent having advanced disease at the time of diagnosis.1 Between 40 and 65 percent of patients in the U.S. who progress following first-line therapy go on to receive a second-line treatment.2,3,4

This trial advances our knowledge about TORISEL in RCC. TORISEL remains an important drug for treatment of advanced kidney cancer based on its pivotal study in first-line patients with poor prognostic risk, said Dr. Mace Rothenberg, senior vice president of clinical development and medical affairs for Pfizers Oncology Business Unit. TORISEL continues to be an important part of Pfizers portfolio of therapies for advanced kidney cancer.

About TORISEL (temsirolimus)

TORISEL is approved in the US and other countries for the treatment of advanced RCC. TORISEL is approved in the European Union for the first-line treatment of patients with advanced renal cell carcinoma (RCC) who have at least three of six prognostic risk factors. In a pivotal Phase 3 study, TORISEL demonstrated median overall survival (OS) in previously untreated patients of 10.9 months in patients with advanced RCC with poor prognostic risk, compared with 7.3 months for interferon-alpha (IFN-).

TORISEL is the only intravenous mammalian target of rapamycin (mTOR) inhibitor approved for the treatment of advanced RCC. TORISEL remains the only treatment to show a significant improvement in OS in treatment-nave poor risk patients with advanced RCC.5

Based on preclinical studies, TORISEL inhibits the activity of mTOR, an intracellular protein implicated in multiple growth-related cellular functions including proliferation, growth and survival. The inhibition of mTOR also reduces levels of certain growth factors, such as vascular endothelial growth factor (VEGF), which are overexpressed in solid tumors like kidney cancer and are thought to play a crucial role in angiogenesis, the process by which tumors acquire blood vessels, nutrients and oxygen needed for growth.

Important TORISEL (temsirolimus) Safety Information

TORISEL is contraindicated in patients with bilirubin >1.5 x ULN and should be used with caution when treating patients with mild hepatic impairment (bilirubin >1 1.5 x ULN or AST > ULN but bilirubin ULN). If TORISEL must be given to patients with mild hepatic impairment, reduce the dose of TORISEL to 15 mg/week. In a phase 1 study, the overall frequency of grade 3 adverse reactions and deaths, including deaths due to progressive disease, was greater in patients with baseline bilirubin > 1.5 x ULN.

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Pfizer Provides Topline Results From Phase 3 Study Of Torisel® As Second-Line Treatment In Advanced Renal Cell ...

LEUVEN, BELGIUM--(Marketwire -05/15/12)- TiGenix NV (TIG) a leader in the field of cell therapy, today gave a business update and announced the financial results for the first quarter ending March 31, 2012.

Business highlights

Financial highlights

"In the first quarter 2012 we continued to aggressively push our commercial efforts forward," said Eduardo Bravo, CEO of TiGenix. "As a result sales of ChondroCelect are developing in line with the improved traction we observed in the second part of last year. At the same time we are moving ahead of schedule with most of our clinical adipose stem cell programs. We closed the quarter with almost EUR 17 million cash on hand, which is sufficient to execute on our business plan and reach key inflection points."

Business update

ChondroCelect sales increase continues apaceThe Company reports net sales growth for the quarter of 123% compared with the same period of last year, and of 62% compared to Q4, 2011, a positive trend reflecting the uptake in Belgium, where we benefit from national reimbursement. In the Netherlands one of the leading private healthcare insurance companies has made treatment with ChondroCelect compulsory for its insured, and no longer reimburses non-ATMP treatments. Similarly, one of the large private insurers in the UK has expressed its intention to routinely reimburse ChondroCelect going forward. Discussions to obtain full national reimbursement keep advancing in the Netherlands, France, Spain and Germany.

Positive outcome of ChondroCelect compassionate use program published in leading journalPositive outcome data from the ChondroCelect compassionate use program (CUP), involving 43 orthopedic centers in 7 European countries, treating 370 patients with ChondroCelect over the span of four years, were published in advance online in Cartilage, the official journal of the International Cartilage Repair Society. The data show that the implantation of ChondroCelect results in a positive benefit/risk ratio when used in an unselected, heterogeneous population, irrespective of the follow-up period, lesion size and type of lesion treated. In addition, the CUP study significantly expands the data set used to obtain approval for ChondroCelect from the European Medicines Agency in 2009, increasing eight-fold, from 43 to 334, the number of patients with long-term follow up data. To date almost 700 patients have been treated with ChondroCelect.

ADMIRE-CD Phase III trial (Cx601) in complex perianal fistula on schedule The ADMIRE-CD (Adipose Derived Mesenchymal stem cells for Induction of REmission in perianal fistulizing Crohn's Disease) Phase III protocol was submitted to Ethics Committees or Health Authorities in all 8 participating countries, and to date approvals have been received in four of those countries already.

Cx611 Phase IIa in RA passes last safety hurdleOn April 17, upon review of the safety data of the first three patients of the third cohort of the company's Phase IIa clinical trial in rheumatoid arthritis (Cx611), TiGenix received the go-ahead from the independent Safety Monitoring Board to recruit and dose the remaining patients of this cohort. This fact is of major importance. In RA it ensures that the product will not be held back by any dose-limiting factors and that we will be able to move forward with the optimal treatment dose. Of almost equal importance is that, if required, we can expand the dosing range in other indications that we are exploring as well. With 6 months of follow-up, the current RA trial in 53 patients is expected to report meaningful results in H1 2013.

Last patient treated in Cx621 Phase I clinical trialAll 10 healthy volunteers have been recruited and treated in the Phase I study of Cx621. Cx621 investigates the safety and feasibility of intra-lymphatic administration of stem cells. Intra-lymphatic administration of (all) stem cells is patented by TiGenix. The final report of this trial will be available at the end of June.

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TiGenix Reports Business & Financial Results for the First Quarter 2012

JERUSALEM--(BUSINESS WIRE)--

Gamida Cell announced today that it has closed an internal E financing round of $10 million. All major shareholders participated.

The financing will be used to support the global commercialization of the companys lead cell therapy product, StemEx, in development as an alternative therapeutic treatment for patients with blood cancers, such as leukemia and lymphoma, who can be cured by bone marrow transplantation but do not have a matched bone marrow donor. The company is currently seeking a strategic partner to join in the global commercialization of StemEx.

The financing will also support the continued development of the companys pipeline of products, primarily the NiCord clinical trial for sickle cell disease and thalassemia.

Mr. Reuven Krupik, chairman of the board of Gamida Cell said, The investors were unanimous in their decision to reinvest, understanding the importance of bringing StemEx to market as well as maintaining the companys leadership role in the stem cell industry. Gamida Cell is a game changer.

The international, multi-center, pivotal registration, Phase III clinical trial of StemEx completed enrollment in February 2012. Clinical outcome is expected in Q4/2012. The market launch of StemEx is planned for 2013. StemEx is likely to be the first allogeneic stem cell product in the market. StemEx is being developed by the Gamida Cell-TEVA joint venture.

Dr. Yael Margolin, president and chief executive officer of Gamida Cell said, With the continued support of our shareholders and the analysis of the clinical results of the StemEx trial just around the corner, we are now focused on submitting the BLA.

StemEx is a graft of an expanded population of stem/progenitor cells, derived from part of a single unit of umbilical cord blood and transplanted by IV administration along with the remaining, non-manipulated cells from the same unit. Competing products in development use two units. As the average cost of a cord blood unit in the U.S. is $40K, StemEx is expected to be a significantly less expensive treatment option. StemEx is also expected to be available in the market several years before any of the competing products.

About Gamida Cell

Gamida Cell is a world leader in stem cell population expansion technologies and stem cell therapy products for transplantation and regenerative medicine. The companys pipeline of stem cell therapy products are in development to treat a wide range of conditions including blood cancers, solid tumors, non-malignant hematological diseases such as hemoglobinopathies, neutropenia and acute radiation syndrome, autoimmune diseases and metabolic diseases as well as conditions that can be helped by regenerative medicine. Gamida Cells therapeutic candidates contain populations of adult stem cells, selected from non-controversial sources such as umbilical cord blood, bone marrow and peripheral blood, which are expanded in culture. Gamida Cells current shareholders include: Elbit Imaging, Clal Biotechnology Industries, Israel Healthcare Venture, Teva Pharmaceutical Industries, Amgen, Denali Ventures and Auriga Ventures. For more information, please visit: http://www.gamida-cell.com.

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Gamida Cell Closes $10 Million E Financing Round Earmarked to Support the Global Commercialization of the Company’s ...

Public release date: 14-May-2012 [ | E-mail | Share ]

Contact: Juan J. Gomez juanj.gomez@cnio.es 34-917-328-000-4060 Centro Nacional de Investigaciones Oncologicas (CNIO)

A number of studies have shown that it is possible to lengthen the average life of individuals of many species, including mammals, by acting on specific genes. To date, however, this has meant altering the animals' genes permanently from the embryonic stage an approach impracticable in humans. Researchers at the Spanish National Cancer Research Centre (CNIO), led by its director Mara Blasco, have proved that mouse lifespan can be extended by the application in adult life of a single treatment acting directly on the animal's genes. And they have done so using gene therapy, a strategy never before employed to combat ageing. The therapy has been found to be safe and effective in mice.

The results are published today in the journal EMBO Molecular Medicine. The CNIO team, in collaboration with Eduard Ayuso and Ftima Bosch of the Centre of Animal Biotechnology and Gene Therapy at the Universitat Autnoma de Barcelona (UAB), treated adult (one-year-old) and aged (two-year-old) mice, with the gene therapy delivering a "rejuvenating" effect in both cases, according to the authors.

Mice treated at the age of one lived longer by 24% on average, and those treated at the age of two, by 13%. The therapy, furthermore, produced an appreciable improvement in the animals' health, delaying the onset of age-related diseases like osteoporosis and insulin resistance and achieving improved readings on ageing indicators like neuromuscular coordination.

The gene therapy utilised consisted of treating the animals with a DNA-modified virus, the viral genes having been replaced by those of the telomerase enzyme, with a key role in ageing. Telomerase repairs the extremes of chromosomes, known as telomeres, and in doing so slows the cell's and therefore the body's biological clock. When the animal is infected, the virus acts as a vehicle depositing the telomerase gene in the cells.

This study "shows that it is possible to develop a telomerase-based anti-ageing gene therapy without increasing the incidence of cancer", the authors affirm. "Aged organisms accumulate damage in their DNA due to telomere shortening, [this study] finds that a gene therapy based on telomerase production can repair or delay this kind of damage", they add.

'Resetting' the biological clock

Telomeres are the caps that protect the end of chromosomes, but they cannot do so indefinitely: each time the cell divides the telomeres get shorter, until they are so short that they lose all functionality. The cell, as a result, stops dividing and ages or dies. Telomerase gets round this by preventing telomeres from shortening or even rebuilding them. What it does, in essence, is stop or reset the cell's biological clock.

But in most cells the telomerase gene is only active before birth; the cells of an adult organism, with few exceptions, have no telomerase. The exceptions in question are adult stem cells and cancer cells, which divide limitlessly and are therefore immortal in fact several studies have shown that telomerase expression is the key to the immortality of tumour cells.

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CNIO scientists successfully test the first gene therapy against aging-associated decline

Barbara Netter, co-founder of the Alliance for Cancer Gene Therapy, receives the first-ever ACGT Partnership Award from Savio Woo, chairman of the Alliance's scientific council and professor and founding chair of the Department of Gene and Cell Medicine at Mt. Sinai School of Medicine.

Photo credit: Courtesy of Elaine Ubia

GREENWICH, Conn. The Greenwich-based Alliance for Cancer Gene Therapy celebrated its 10th anniversary of funding cancer gene therapy research with a gala benefit last month.

Thirty of the nations top cancer research scientists and more than 450 Alliance supporters gathered on April 19 to mark the progress made in the field. The event raised more than $1.4 million, all of which will go directly to funding new grants and clinical trails.

The alliance also honored the life of its visionary co-founder, Edward Netter, who died last year, by presenting the first ever ACGT Partnership award to Netter's wife, Barbara, who co-founded the alliance with her husband.

Ed epitomized the innovative thinker and problem solver, and he felt it important to examine the status quo, Barbara Netter said in a prepared statement. He had the perseverance to pursue a better way. Our progress, our recent breakthrough, is proving to be a fitting tribute and legacy to Ed, who worked tirelessly and passionately to this end. Ed Netter was a key figure in accelerating research and bringing us to this point.

Prior to the evenings festivities many of the nations leading cancer researchers held a scientific symposium, where they discussed the alliances progress as a leader in the field, as well as how to bridge the gap between the lab and bringing science to patients.

Robert Bazell, the Emmy award-winning chief science and health correspondent for NBC News, was the keynote speaker. Dr. Dennis Clegg, professor, Department of Molecular, Cellular and Developmental Biology, University of California, Santa Barbara, California, was the featured scientist at the dinner.

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Greenwich Cancer Research Group Raises $1.4M

Diane Cook's 65th birthday was a milestone for her, but not because of her age. That was the day she was diagnosed with Parkinson's disease.

"I was stunned," she said, adding that even though she had had symptoms for four years before being diagnosed, the news was still a surprise.

That day, she could not muster the courage to learn more about what was in store for her. But the next day she pored over the Internet to learn about the disease. She quickly discovered that it would continue to get worse, and that there was no cure.

Parkinson's disease, often associated with boxer Muhammad Ali and actor Michael J. Fox, affects 1 million Americans, according to the National Parkinson Foundation.

While the exact causes largely remain a mystery, doctors know that the condition arises from the degeneration of a specific area of the brain involved in movement. As a result, those with Parkinson's experience tremors, rigidity, slowness in moving, and difficulty with balancing and walking.

Parkinson's eventually leads to mood disorders and dementia. The complications associated with the condition are the 14th largest cause of death in the United States, according to the U.S. Centers for Disease Control and Prevention.

Not only is there no cure for Parkinson's, but many patients have no way of knowing how quickly their symptoms will progress.

"We all worry about how rapidly we'll lose our abilities," Cook said. "The uncertainty is very frustrating."

A new study from UCLA may help. Researchers have found two variants on a gene already known to be associated with Parkinson's that may be able to predict how quickly patients with the condition will deteriorate. The study found that patients with one particular variant were four times as likely to have rapid decline of motor function. Those patients having both of the variants studied were even more likely to see their disease progress more quickly.

The information is important, as patients who have more severe motor disease tend to die sooner.

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Gene Predicts Parkinson's Progression

ScienceDaily (May 15, 2012) A novel mechanism for anxiety behaviors, including a previously unrecognized inhibitory brain signal, may inspire new strategies for treating psychiatric disorders, University of Chicago researchers report.

By testing the controversial role of a gene called Glo1 in anxiety, scientists uncovered a new inhibitory factor in the brain: the metabolic by-product methylglyoxal. The system offers a tantalizing new target for drugs designed to treat conditions such as anxiety disorder, epilepsy, and sleep disorders.

The study, published in the Journal of Clinical Investigation, found that animals with multiple copies of the Glo1 gene were more likely to exhibit anxiety-like behavior in laboratory tests. Further experiments showed that Glo1 increased anxiety-like behavior by lowering levels of methylglyoxal (MG). Conversely, inhibiting Glo1 or raising MG levels reduced anxiety behaviors.

"Animals transgenic for Glo1 had different levels of anxiety-like behavior, and more copies made them more anxious," said Abraham Palmer, PhD, assistant professor of human genetics at the University of Chicago Medicine and senior author of the study. "We showed that Glo1 was causally related to anxiety-like behavior, rather than merely correlated."

In 2005, a comparison of different mouse strains found a link between anxiety-like behaviors and Glo1, the gene encoding the metabolic enzyme glyoxylase 1. However, subsequent studies questioned the link, and the lack of an obvious connection between glyoxylase 1 and brain function or behavior made some scientists skeptical.

"When people discover a gene, they're always most comfortable when they discover something they already knew," Palmer said. "The alarming thing here was there was a discovery of something that nobody knew, and therefore it seemed less likely to actually be correct."

A 2009 study from Palmer's laboratory suggested that differences in Glo1 expression between mouse strains were due to copy number variants, where the segment of the genome containing the gene is repeated multiple times. To test this hypothesis, lead author Margaret Distler inserted two, eight or ten copies of the Glo1 gene into mouse lines. She then ran experiments such as the open field test, in which researchers measure how much time a mouse spends in the center of an arena versus along the walls, to detect changes in anxiety behavior.

The results confirmed a causative role for Glo1 copy number variants, as mice with more copies of the Glo1 gene exhibited higher anxiety-like behavior in their experiments.

"It's the first study to show that it's the copy number variant that has the potential to change Glo1 expression and behavior," said Distler, an MD/PhD student in the Pritzker School of Medicine's Medical Scientist Training Program. "Our study was a physiological representation of what it means to increase Glo1 expression for anxiety."

The researchers then set about answering the mystery of how Glo1 expression influences anxiety-like behaviors. The primary function of glyoxylase 1 is to metabolize and lower cellular levels of methylglyoxal, a waste product of glycolysis. Distler produced the opposite effect by injecting MG to artificially increase its levels in the brain, finding that raising MG levels quickly reduced anxiety symptoms in mice.

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Mystery gene reveals new mechanism for anxiety disorders

GRAND RAPIDS, MI An international research effort led by a Van Andel Institute scientist has identified several new genes frequently mutated in bile duct cancer, a deadly form of liver cancer.

The breakthrough came after two years of intensive research that involved scientists visiting villagers in northern Thailand, an area with a high rate of bile duct cancer, according to VAI officials.

The research was led by Bin Tean Teh, the director of the Van Andel Research Institutes Translational Cancer Research Laboratory at the National Cancer Center in Singapore.

According to a statement released by VAI, bile duct cancer accounts for 10 to 25 percent of all primary liver cancers worldwide and has a poor prognosis, the VAI says. The high incidence in Thailand is attributed to consumption of raw fish infected with parasites, called liver flukes. The flukes cause infection in the bile duct and eventually cause cancer.

The researchers analyzed bile duct cancers of Thai patients and discovered mutations in 187 genes. They focused on 15 genes that were frequently mutated.

This discovery adds depth to what we currently know about bile duct cancer, Teh said in VAIs statement. More important is that we are now aware of new genes and their effects on bile duct cancer, and we now need to further examine their biological aspects to determine how they bring about the onset of cholangiocarcinoma (bile duct cancer).

We are talking about the potential to save many lives in Thailand, said Professor Vajarabhongsa Bhudhisawasdi, director of bile duct cancer research at Khon Kaen University of Thailand.

The research findings are published online in Nature Genetics.

Email Sue Thoms at sthoms1@mlive.com and follow her on Twitter at twitter.com/suethoms

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Van Andel Institute leads breakthrough research into deadly form of liver cancer

SAN DIEGO, May 16, 2012 /PRNewswire/ -- Cardium Therapeutics (CXM) today announced a late-breaking poster presentation at the American Society of Gene & Cell Therapy (ASGCT) 15th Annual Meeting being held May 16-19, 2012 at the Pennsylvania Convention Center in Philadelphia, PA.

(Photo: http://photos.prnewswire.com/prnh/20120516/LA07787)

(Logo: http://photos.prnewswire.com/prnh/20051018/CARDIUMLOGO)

The new research findings demonstrate that cardiac ischemia plays an important role in adenovector gene transfection (delivery) in mammalian hearts. Based on this understanding, using a standard balloon angioplasty catheter, researchers have developed and tested a new method to induce transient ischemia during a non-surgical interventional cardiac procedure, which when coupled with the infusion of nitroglycerin, boosts the delivery (cell transfection) of an adenovector gene construct into heart cells. The increase in adenovector-based gene transfection with the new technique is over two orders of magnitude (>100 fold).

Cardium's new method of adenovector delivery takes advantage of the findings that transient ischemia appears to alter the permeability barrier of the vascular endothelium and may expose the blood to the coxsackie-adenovirus receptor mediating adenovector uptake by the heart. Balloon angioplasty catheters have been used for many years to dilate blocked coronary arteries, sometimes with use of a stent, and these catheters have also been used safely by cardiologists in patients with coronary artery disease to study the effects of brief ischemia. Cardium's new technique inflates the balloon in non-narrowed areas, and only enough to briefly interrupt flow using inflation pressure that is less than that used for performing angioplasty.

Cardium's recently initiated Russian-based ASPIRE Phase 3 / registration clinical study uses transient ischemia techniques during non-surgical percutaneous catheterization with a standard angioplasty catheter together with the intracoronary infusion of nitroglycerin with the Generx [Ad5FGF-4] product candidate for the treatment of patients with myocardial ischemia and stable angina pectoris. These patients have atherosclerotic coronary artery disease, and the Company's Generx product candidate is intended to stimulate the growth of new or additional collateral blood vessels to bypass blockages.

These studies were conducted at Emory University School of Medicine, led by Jakob Vinten-Johnasen, PhD., and co-sponsored by a Small Business Innovative Research grant from the National Institutes of Health (Cardium Therapeutics) and the Carlyle Fraser Heart Center (Emory). At the conference Gabor M. Rubanyi, MD, PhD, Cardium's Chief Scientific Officer, will present the late-breaking poster entitled "Transient Ischemia is Necessary for Efficient Adenovector Gene Transfer in the Heart", on May 17, 2012 from 3:00 to 5:30 p.m. in Exhibit Hall A. The poster presentation can be viewed at http://www.cardiumthx.com/pdf/Generx-ASGCT-Poster-Presentation-May-2012.pdf.

In addition, Dr. Rubanyi will also make an oral presentation titled: "New Perspectives for Angiogenic Gene Therapy to Treat Myocardial Ischemia in Patients with Coronary Disease" to attendees at the ASGCT Meeting today, May 16. The presentation will provide a historical overview of the Generx clinical development program and how these new and important preclinical findings have been incorporated into the protocol for the 100-patient Generx ASPIRE Phase 3 registration study which was recently initiated in the Russian Federation for patients with myocardial ischemia and stable angina pectoris. The presentation is now available for viewing at http://www.cardiumthx.com/pdf/Generx-ASGCT-May-2012-Rubanyi.pdf.

About Generx and the ASPIRE Study

Generx (Ad5FGF-4) is a disease-modifying regenerative medicine biologic that is being developed to offer a one-time, non-surgical option for the treatment of myocardial ischemia in patients with stable angina due to coronary artery disease, who might otherwise require surgical and mechanical interventions, such as coronary artery by-pass surgery or balloon angioplasty and stents. Similar to surgical/mechanical revascularization approaches, the goal of Cardium's Generx product candidate is to improve blood flow to the heart muscle but to do so non-surgically, following a single administration from a standard balloon angioplasty catheter. The video "Cardium Generx Cardio-Chant" provides an overview Generx and can be viewed at http://www.youtube.com/watch?v=pjUndFhJkjM.

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Cardium Reports New Catheter-Based Methods Significantly Boost Cardiac Gene Delivery In Late-Breaking Presentation at ...

ScienceDaily (May 16, 2012) A well-known genetic risk factor for Alzheimer's disease triggers a cascade of signaling that ultimately results in leaky blood vessels in the brain, allowing toxic substances to pour into brain tissue in large amounts, scientists report May 16 in the journal Nature.

The results come from a team of scientists investigating why a gene called ApoE4 makes people more prone to developing Alzheimer's. People who carry two copies of the gene have roughly eight to 10 times the risk of getting Alzheimer's disease than people who do not.

A team of scientists from the University of Rochester, the University of Southern California, and other institutions found that ApoE4 works through cyclophilin A, a well-known bad actor in the cardiovascular system, causing inflammation in atherosclerosis and other conditions. The team found that cyclophilin A opens the gates to the brain assault seen in Alzheimer's.

"We are beginning to understand much more about how ApoE4 may be contributing to Alzheimer's disease," said Robert Bell, Ph.D., the post-doctoral associate at Rochester who is first author of the paper. "In the presence of ApoE4, increased cyclophilin A causes a breakdown of the cells lining the blood vessels in Alzheimer's disease in the same way it does in cardiovascular disease or abdominal aneurysm. This establishes a new vascular target to fight Alzheimer's disease."

The team found that ApoE4 makes it more likely that cyclophilin A will accumulate in large amounts in cells that help maintain the blood-brain barrier, a network of tightly bound cells that line the insides of blood vessels in the brain and carefully regulates what substances are allowed to enter and exit brain tissue.

ApoE4 creates a cascade of molecular signaling that weakens the barrier, causing blood vessels to become leaky. This makes it more likely that toxic substances will leak from the vessels into the brain, damaging cells like neurons and reducing blood flow dramatically by choking off blood vessels.

Doctors have long known that the changes in the brain seen in Alzheimer's patients -- the death of crucial brain cells called neurons -- begins happening years or even decades before symptoms appear. The steps described in Nature discuss events much earlier in the disease process.

The idea that vascular problems are at the heart of Alzheimer's disease is one championed for more than two decades by Berislav Zlokovic, M.D., Ph.D., the leader of the team and a neuroscientist formerly with the University of Rochester Medical Center and now at USC. For 20 years, Zlokovic has investigated how blood flow in the brain is affected in people with the disease, and how the blood-brain barrier allows nutrients to pass into the brain, and harmful substances to exit the brain.

At Rochester, Zlokovic struck up a collaboration with Bradford Berk, M.D., Ph.D.,a cardiologist and CEO of the Medical Center. For more than two decades Berk has studied cyclophilin A, showing how it promotes destructive forces in blood vessels and how it's central to the forces that contribute to cardiovascular diseases like atherosclerosis and heart attack.

"As a cardiologist, I've been interested in understanding the role of cyclophilin A in patients who suffer from cardiovascular illness," said Berk, a professor at the Aab Cardiovascular Research Institute. "Now our collaboration in Rochester has resulted in the discovery that it also has an important role in Alzheimer's disease. The finding reinforces the basic research enterprise -- you never know when knowledge gained in one area will turn out to be crucial in another."

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Alzheimer's gene causes brain's blood vessels to leak toxins and die

Public release date: 16-May-2012 [ | E-mail | Share ]

Contact: Tom Rickey tom_rickey@urmc.rochester.edu 585-275-7954 University of Rochester Medical Center

A well-known genetic risk factor for Alzheimer's disease triggers a cascade of signaling that ultimately results in leaky blood vessels in the brain, allowing toxic substances to pour into brain tissue in large amounts, scientists report May 16 in the journal Nature.

The results come from a team of scientists investigating why a gene called ApoE4 makes people more prone to developing Alzheimer's. People who carry two copies of the gene have roughly eight to 10 times the risk of getting Alzheimer's disease than people who do not.

A team of scientists from the University of Rochester, the University of Southern California, and other institutions found that ApoE4 works through cyclophilin A, a well-known bad actor in the cardiovascular system, causing inflammation in atherosclerosis and other conditions. The team found that cyclophilin A opens the gates to the brain assault seen in Alzheimer's.

"We are beginning to understand much more about how ApoE4 may be contributing to Alzheimer's disease," said Robert Bell, Ph.D., the post-doctoral associate at Rochester who is first author of the paper. "In the presence of ApoE4, increased cyclophilin A causes a breakdown of the cells lining the blood vessels in Alzheimer's disease in the same way it does in cardiovascular disease or abdominal aneurysm. This establishes a new vascular target to fight Alzheimer's disease."

The team found that ApoE4 makes it more likely that cyclophilin A will accumulate in large amounts in cells that help maintain the blood-brain barrier, a network of tightly bound cells that line the insides of blood vessels in the brain and carefully regulates what substances are allowed to enter and exit brain tissue.

ApoE4 creates a cascade of molecular signaling that weakens the barrier, causing blood vessels to become leaky. This makes it more likely that toxic substances will leak from the vessels into the brain, damaging cells like neurons and reducing blood flow dramatically by choking off blood vessels.

Doctors have long known that the changes in the brain seen in Alzheimer's patients the death of crucial brain cells called neurons begins happening years or even decades before symptoms appear. The steps described in Nature discuss events much earlier in the disease process.

The idea that vascular problems are at the heart of Alzheimer's disease is one championed for more than two decades by Berislav Zlokovic, M.D., Ph.D., the leader of the team and a neuroscientist formerly with the University of Rochester Medical Center and now at USC. For 20 years, Zlokovic has investigated how blood flow in the brain is affected in people with the disease, and how the blood-brain barrier allows nutrients to pass into the brain, and harmful substances to exit the brain.

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Alzheimer's gene causes brain's blood vessels to leak, die

Viruses act as tiny piezoelectric generators

Viruses, tiny chunks of protein and nucleic acid, have long plagued mankind and its evolutionary ancestors before it. But thanks to the wonders of modern genetic engineering, researchers believe they have finally been able to instill a beneficial purpose in these deadly pests.

I. From Pest to Power

A team of researchers at Lawrence Berkeley National Laboratory -- one of 16 U.S. Department of Energy (DOE) national laboratories -- has created a special breed of virus that undergoes self-nanoassembly to form tiny piezoelectric generators -- machines which harvest mechanical energy (vibrations or pressure) to directly produce electricity.

The special "bug" is the M13 bacteriophage, a rod-shaped virus that only infects bacteria (such asE. coli bacteria)-- not humans.

Faculty researchersSeung-Wuk Lee, Ramamoorthy Ramesh, and Byung Yang Lee selected the virus due to its tendency to self-assemble into nanofilms, given its rod-like shape. The viruses tightly pack "like chopsticks in a box" and are easy to grow by the millions given a small supply of host bacteria.

II. Refining the Virus

But the effect was too weak to be of use. So the researchers spliced a quadruplet of negatively charged amino acids into one of the coat proteins. The results was a larger voltage gradient across the coat. The researchers also tested stacking films of the modifed viruses to see how thick they could layer the viruses in order to get the maximum effect.

When pressure was applied to the film a 400 millivolt, 6 nanoampere current was put off. That's about a quarter of the voltage of an AAA battery, albeit at a far smaller current. Still it was enough to power a '1' to show up on a low-power liquid crystal display.

Read this article:
Berkeley Trains "Harmless" Viruses to Harvest Human Kinetic Energy

Public release date: 16-May-2012 [ | E-mail | Share ]

Contact: Cathia Falvey cfalvey@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, May 16, 2012The inaugural issue of BioResearch Open Access, a new bimonthly peer-reviewed open access journal, was released today by Mary Ann Liebert, Inc., publishers. The Journal provides a new rapid-publication forum for a broad range of scientific topics including but not limited to molecular and cellular biology, tissue engineering and biomaterials, regenerative medicine, stem cells, gene therapy, systems biology, genetics, biochemistry, virology, microbiology, and neuroscience. The first issue is available on the BioResearch Open Access website at http://www.liebertpub.com/biores.

The premier issue includes research papers and a brief report from the U.S., U.K., Germany, and Korea on diverse topics such as tissue engineering, stem cells, HIV, and genetics. Forthcoming papers for the second issue include genetics, xenotransplantation, nuclear transfer, and cardiac research.

The Journal is under the leadership of Editor-in-Chief Jane Taylor, PhD, Senior Research Fellow, MRC Centre for Regenerative Medicine, University of Edinburgh, and seasoned journal editors as Section Editors, including James M. Wilson, MD, PhD, University of Pennsylvania; Antonios G. Mikos, PhD, Rice University; Professor Sir Ian Wilmut, OBE FRS FRSE, University of Edinburgh; Peter C. Johnson, MD, Scintellix, LLC, Raleigh, NC; Aubrey D.N.J. de Grey, PhD, SENS Foundation, Cambridge, UK; Alan J. Russell, PhD, Carnegie Mellon University; Thomas Hope, PhD, Northwestern University; Ganes C. Sen, PhD, Cleveland Clinic Foundation; Bruce A. Sullenger, PhD, Duke University Medical Center; Graham C. Parker, PhD, Wayne State University School of Medicine; Carol Shoshkes Reiss, PhD, New York University; Stephen C. Ekker, PhD, Mayo Clinic, Rochester, MN; John B. West, MD, PhD, University of California, San Diego; David L. Woodland, PhD, Chief Scientific Officer, Keystone Symposia on Molecular and Cellular Biology; Stephen Higgs, PhD, Kansas State University; Eugene Kolker, PhD, Seattle Children's Hospital; and Domenico Grasso, PhD, PE, DEE, University of Vermont.

The Journal welcomes basic science and translational research in the form of original research articles, comprehensive review articles, mini-reviews, rapid communications, brief reports, technical reports, hypothesis articles, perspectives, and letters to the editor. All articles in BioResearch Open Access will be published online within 4 weeks of acceptance. Articles will be fully open access and posted on PubMedCentral. All articles submitted through July 15, 2012 will be made open access without article processing charges. BioResearch Open Access is fully NIH-, HHMI-, and Wellcome Trust compliant.

"BioResearch Open Access is a fully refereed multidisciplinary journal and provides all the checks and balances that rigorous peer review ensures," says Mary Ann Liebert, president of Mary Ann Liebert, Inc., publishers. "An outstanding editorial team comprised of experienced journal editors guarantees the integrity of the Journal."

###

About the Publisher

Mary Ann Liebert, Inc., publishers is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including Tissue Engineering, Human Gene Therapy, Nucleic Acid Therapeutics, Stem Cells and Development, Viral Immunology, DNA and Cell Biology, and Antioxidants & Redox Signaling. Its biotechnology trade magazine, Genetic Engineering & Biotechnology News (GEN), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's 70 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc. website at http://www.liebertpub.com.

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Premier issue of BioResearch Open Access launched by Mary Ann Liebert Inc. publishers

Researchers may be closing in on diseases inherited component

Web edition : Tuesday, May 15th, 2012

Schizophrenias elusive genetic roots may finally be within grasp. A new, wide-ranging effort has uncovered a set of DNA signatures that are shared by people with the disease consistently enough that the set can be used to reliably predict whether someone has the disease. If replicated, the results may point out ways to diagnose schizophrenia and suggest new targets for treatment.

By analyzing a battery of 542 genetic variants, researchers could predict who had schizophrenia in a group of European Americans and African Americans. The confirmation of the result in people of varying ancestry suggests that the set of genes truly does detect the core features of the disorder, scientists report online May 15 in Molecular Psychiatry.

Genetic studies in psychiatry tend to produce initial excitement but are then not reproduced in independent populations, which is the most important proof that a finding is solid and real, says study coauthor Alexander Niculescu of the Indiana University School of Medicine in Indianapolis.

Niculescu and his colleagues created their gene panel by assessing a slew of earlier studies on schizophrenia: Data from humans and animals on gene variation and gene behavior all fed into the teams analysis. If a gene popped out of several different datasets, the reasoning went, it is probably important to schizophrenia. Niculescu compares this method called convergent functional genomics to an Internet search: The more links to a web page, the higher it comes up on your search list.

After sifting through all of this data, the team identified some top candidates, some already known to be related to schizophrenia (DISC1, a known culprit, sits at the top of the list) and a handful that have never before been linked to the disease.

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Schizophrenia’s core genetic features proposed

Public release date: 14-May-2012 [ | E-mail | Share ]

Contact: Jim Dryden jdryden@wustl.edu 314-286-0110 Washington University School of Medicine

Researchers at Washington University School of Medicine in St. Louis have developed a genetic test that can accurately predict whether the most common form of eye cancer will spread to other parts of the body, particularly the liver.

In 459 patients with ocular melanoma at 12 centers in the United States and Canada, the researchers found the test could successfully classify tumors more than 97 percent of the time.

The study will appear in an upcoming issue of the journal Ophthalmology, but is now online.

"When the cancer spreads beyond the eye, it's unlikely any therapy is going to be effective," says principal investigator J. William Harbour, MD. "But it's very possible that we can develop treatments to slow the growth of metastatic tumors. The real importance of this test is that by identifying the type of tumor a patient has, we can first remove the tumor from the eye with surgery or radiation and then get those individuals at high risk into clinical trials that might be able to help them live longer."

Harbour believes the test should allow ocular oncologists to quickly evaluate the risks associated with particular tumors and to begin treatment the moment they can detect any spread of the cancer.

Melanoma of the eye is relatively rare, diagnosed in about 2,000 people in the United States each year. Advances in treatment have allowed surgeons to preserve patients' vision, but when cancer spreads beyond the eye, it often is deadly.

About a decade ago, Harbour, the Paul A. Cibis Distinguished Professor of Ophthalmology and Visual Sciences, began using gene expression profiling to monitor the activity of thousands of genes in and around ocular melanoma tumors.

"At the time, we were surprised to see that based on these gene expression profiles, the tumors clustered into two groups that corresponded, almost perfectly, to patients whose cancer spread and those whose cancer was confined within the eye," says Harbour, who directs Washington University's Center for Ocular Oncology. "Tumors with a class 1 gene expression profile, or 'signature,' very rarely spread, but those with a class 2 profile frequently develop into metastatic cancer."

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Genetic test identifies eye cancer tumors likely to spread

In households across the country, children conceived with donated sperm are struggling with serious genetic conditions inherited from men they have never met: heart defects, spinal muscular atrophy, neurofibromatosis type 1 and fragile-X syndrome the most common form of mental retardation in boys and others.

Donated eggs pose a risk as well, but the threat of genetic harm from sperm donation is arguably much greater. Sperm donors are no more likely to carry genetic diseases than anybody else, but they can father a far greater number of children: 50, 100 or even 150, each a potential inheritor of flawed genes.

Sharine and Brian Kretchmar of Yukon, Okla., tried a number of medical treatments to conceive a second child.

After a depressing series of failures, they were advised by a doctor to find a sperm donor. For more than a year, the Kretchmars researched sperm banks and donors. The donor they chose was a family man, a Christian like them, they were told. Most important, he had a clean bill of health. So the Kretchmars jumped in. After artificial insemination, Sharine Kretchmar became pregnant, and in April 2010, she gave birth to a boy they named Jaxon.

But the baby failed to have a bowel movement in the first day or so after birth, a sign to doctors that something was wrong. Doctors returned with terrible news: Jaxon appeared to have cystic fibrosis.

"We were pretty much devastated," Sharine Kretchmar said.

Genetic testing showed that Jaxon did carry the genes for cystic fibrosis. Sharine Kretchmar, 33, had no idea she was a carrier and was shocked to discover that so, too, was the Kretchmars' donor.

His sperm, they would discover, was decades old, originally donated at a laboratory halfway across the country and frozen ever since. Whether it was properly tested is a matter of dispute.

Experience not unique

Sadly, the Kretchmars' experience is not unique.

Read more here:
Given number of inheritors, donor sperm carries risk of genetic harm

Every few days my Google Alerts have been dropping in my inbox reviews of Harry Osters Legacy: A Genetic History of the Jewish People. The latest, is in the The Tablet, A Case for Genetic Jewishness:

For a Jewish genetics researcher, being told inprintthat Hitler would certainly have been very pleased by your work cant be pleasant. But thats what happened in 2010 toHarry Ostrer, a geneticist at the Albert Einstein College of Medicine, when he and his colleagues published astudyshowing that Jews in three different geographical areas had certain collections of genes that made them more biologically similar to one another than they were to non-Jews in the same regions. The work also showed that Jews around the world could trace their ancestry to a group of people who lived in the Middle East 2,000 years ago; that meant, however, that certain genetic signatures could be used to identify Jews, indicating that Jews share a common biological identity beyond their religious affiliationwhich is what inspired the Hitler crack.

I dont plan on reading Legacy because I already read the paper which it is based on, Abrahams Children in the Genome Era: Major Jewish Diaspora Populations Comprise Distinct Genetic Clusters with Shared Middle Eastern Ancestry. It is now open access, so you can read it too. As implied in the article in The Tablet the biggest finding in this paper is that most of the worlds Jewry seem to share tracts of the genome which are identical by descent (IBD). You dont have to be a geneticist to intuit that being IBD implies relatively recent and elevated shared descent from a common set of ancestors. In particular the authors were looking for segments of the genome where individuals shared the same sequence of genetic markers. Very long sequences indicate a relatively recent common ancestor, while many short ones suggest more distant but numerous common ancestors.

From looking at these patterns of relatedness the authors infer that despite the genetic variation in the modern Jewry, most of the worlds Jews, from Iran to Morocco to Lithuania, share common ancestry from a source population which flourished ~2,500 years ago. All that being said, genetics is only part of the puzzle here. In the discussion the authors suggest that Yet, the sharing of Iranian and Iraqi Jews of a branch on the phylogenetic tree with the Adygei suggests that a certain degree of admixture may have occurred with local populations not included in this study. I argue in my post The Assyrians and Jews: 3,000 years of common history, a clear and distinct category of Jew as opposed to generic North Levantine in the year 500 BC probably does not make biological sense, though it might make culturally sense (and generic North Levantine is obviously not accurate, as most of these individuals had strong tribal or ethnic identities at the time). Finally, I dont think I highlighted in my earlier commentary that these data imply that the rise of Christianity and Islam fundamentally stabilized the genetics of the Jewish people, insofar as much of the admixture upon the core base in the peripheral populations seems to predate the rise of these religious civilizaitons. Once Christianity and Islam marginalized the Jews, the gene flow from non-Jews to Jews diminished greatly. This is curiously analogous to the cultural involution which Jews also underwent during this period.

Read more from the original source:
Abraham’s genetic threads | Gene Expression

Cancer Genetics is pursuing a strategy that differs from Facebook's IPO plan, expert says.

A Rutherford, N.J.-based start-up developing tools to diagnose cancer is planning to launch an initial public offering this week under the long shadow of Facebook's long-awaited blockbuster IPO.

Cancer Genetics Inc. is looking to raise more than $50 million with an initial public offering of 4 million shares priced between $11 to $13 a share.

The offering comes as the IPO market is struggling back toward its pre-recession level of activity, although it remains well below the peak of the dot-com fueled boom of the 1990s. If Cancer Genetics prices this week, it will be the first IPO in North Jersey since Park Ridge-based SeaCube Container Leasing Ltd. went public in October 2010. The ticker symbol would be CGIX, and the shares would trade on the Nasdaq Stock Market.

How the Facebook IPO will affect that of Cancer Genetics is unclear. David Menlow, president of IPOfinancial.com, said he thinks it "greatly hampers their chance of success."

"Investors are not interested in paying attention to anything other than the 800-ton gorilla in the room," which is Facebook, he said.

But Jay R. Ritter, a professor of finance at University of Florida in Gainesville, who tracks IPOs, said the two companies are playing to different investors.

Cancer Genetics, which is planning its IPO for Friday, is looking to attract institutional investors interested in holding company stock for the long period it takes for a biotech company to make a medical breakthrough, he said. In contrast, many Facebook investors are mainly interested in the chance of making a quick profit if the stock takes off, he said.

"Facebook is getting huge amounts of attention," Ritter said. "Lots of investors are coming out of the woodwork who do not have an interest in buying biomedical companies."

Cancer Genetics, founded in 1999, is focused on developing and commercializing tests and services to diagnose, predict and help treat hematological, urogenital and HPV-associated cancers. The top executives at Cancer Genetics are Chairman Raju S.K. Chaganti, Chief Executive Officer Panna L. Sharma and Elizabeth A. Czerepak, chief financial officer.

Originally posted here:
Cancer Genetics IPO Faces Facebook

WESTMINSTER, Colo., May 16, 2012 /PRNewswire/ -- GeneThera, Inc. (GTHR.PK) announced today that Applied Genetics, GeneThera's majority owned subsidiary, signed a research and test validation agreement with Universidad National Autonoma de Mexico (UNAM). Scope of the agreement is to validate GeneThera proprietary Johne's disease (JD) HerdCheck Field Collection System (FCS) Molecular Assay in Mexico. Dr. Tony Milici, interim president of Applied Genetics and CEO of GeneThera, stated, "This agreement is another major milestone in establishing GeneThera and Applied Genetics' leadership in the field of Johne's disease molecular testing. UNAM's support is of fundamental importance to speed up the validation process of GeneThera's Johne's disease molecular testing, which will lead to JD test approval by the Mexican Government."

Dr. Gilberto Chaves Griz, Professor of Veterinary Pathology, in the Department of Veterinary medicine at UNAM and Director of the Johne's Disease Center, who is one of the world's most renowned expert in the field of Johne's disease declared, "We are very pleased to partner with Applied Genetics and GeneThera to work on this project. It is extremely critical that we can diagnose JD in Mexico using the most advanced technology available. It is also of paramount importance to establish a Johne's disease program at the national level. Mexico has one of the largest cows, goat and sheep populations in Northern and Central America, yet no data exists to the extent of Johne's infection in these animals. It is our firm intention to establish a National Testing Program for Johne's Disease in Mexico with the help of Applied Genetics and GeneThera's state of the art technology." UNAM is the largest University in Mexico. Applied Genetics is a molecular diagnostic company that focuses on commercializing molecular testing for Johne's disease in Mexico.

Johne's disease is a global devastating and incurable disease of dairy cows, sheep and goats caused by a bacterium called Mycobacterium Paratuberculosis sub. Avium, (MAP). Dairy products, contaminated with MAP, are the vehicles by which the infection spreads in the human intestine triggering the onset of Crohn's disease. Applied Genetics employs the use of GeneThera HerdCheck to test and control the spread of Johne's disease in Mexico. HerdCheck is a proprietary molecular diagnostic system based on the use of high throughput robotics and Real time PCR.

About GeneThera, Inc.

GeneThera, Inc. is a molecular biotechnology company located in Westminster, Colorado. The Company's proprietary diagnostic solution is based on a genetic expression system (GES) and Johne's disease management system, HERDCHECK, designed to function on a highly automated Fluorogenic PCR platform. This platform enables GeneThera to offer tests that are presently not available from other technologies. The GES and HERDCHECK systems are designed for a host of individual diseases, the current priority being Johne's disease. For more information, contact Dr. Tony Milici at 720 439-3011.

This press release contains forward-looking statements, which are made pursuant to the Safe-Harbor provisions of the Private Securities Litigation Reform Act of 1995. Words such as "intends," "believes," and similar expressions reflecting something other than historical fact are intended to identify forward-looking statements, but are not the exclusive means of identifying such statements. These forward-looking statements involve a number of risks and uncertainties, including the timely development and market acceptance of products and technologies, the ability to secure additional sources of finance, the ability to reduce operating expenses, and other factors described in the Company's filings with the Securities and Exchange Commission. The actual results that the Company achieves may differ materially from any forward-looking statement due to such risks and uncertainties. The Company undertakes no obligation to revise or update any forward-looking statements in order to reflect events or circumstances that may arise after the date of this release.

http://www.genethera.net

The rest is here:
Applied Genetics Signs Agreement With The National University Of Mexico (UNAM)

Recently, there was a gathering of scientists at the Hyatt Regency in Greenwich. They were there to discuss their latest findings in cancer research -- especially in the use of gene therapy in cancer treatment. There was tangible excitement in the room. Among those in attendance was Dr. Carl June of the University of Pennsylvania, who had, using gene therapy, actually eliminated all signs of cancer in two patients he was treating. Also there was Dr. Hui Hu, who works in the same city as June, at the Wistar Institute, and who is having similar success treating mice with cancer with his gene therapy, which he wants to apply to human patients.

The reason for this writing, however, is another reason for the gathering -- the celebration of the 10th anniversary of the Alliance for Cancer Gene Therapy (ACGT), a Greenwich non-profit that has helped fund these scientists and others like them; and the legacy of Ed Netter, of Greenwich, co-founder of ACGT, who died last year, just short of Dr. Carl June's news of his gene therapy treatment.

Barbara Netter, Ed Netter's widow and cofounder of ACGT, was feeling the excitement in the scientific meeting held before the gala dinner honoring her late husband and ACGT.

"We have so much energy now with Carl June," she said. "We're receiving a lot more applications for grants. There's a lot of collaboration and partnering happening."

It was the death of the Netters' daughter-in-law from breast cancer that inspired Ed and Barbara Netter to create ACGT 10 years ago as a vehicle to raise money for research into cancer gene therapy.

"The vision that Ed had is a new way to really get the science into clinical trials that will show the (gene therapy) concept at work," said Barbara Netter.

Last August, Dr. Carl June reported to the world the success of his clinical trial, supported by ACGT, in which he genetically modified the T-cells of three patients with chronic lymphocytic leukemia to target and kill their tumors. Two of the three patients remain cancer free, with the third patient's cancer significantly reduced.

June's treatment, he said, was still only in the first phase. But he's now adding more patients. "We've taken in our first pediatric patient -- a 6-year-old girl with leukemia. She was infused today," June said.

This is the first of 400 patients June wants to treat within the next year or two.

"The gene therapy approach is fundamentally different from current therapies," said June, "and these ideas need to move to human treatment."

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Greenwich couples' legacy lives on in ACGT

Public release date: 16-May-2012 [ | E-mail | Share ]

Contact: John Pastor jdpastor@ufl.edu 352-273-5815 University of Florida

Using gene transfer techniques pioneered by University of Florida faculty, Taiwanese doctors have restored some movement in four children bedridden with a rare, life-threatening neurological disease.

The first-in-humans achievement may also be helpful for more common diseases such as Parkinson's that involve nerve cell damage caused by lack of a crucial molecule in brain tissue. The results are reported today (May 16) in the journal Science Translational Medicine.

The children in the study, who ranged in age from 4 to 6, inherited a rare disease known as aromatic L-amino acid decarboxylase deficiency, or AADC. Patients with AADC are born without an enzyme that enables the brain to produce the neurotransmitter dopamine. They generally die in early childhood.

In a phase 1 clinical trial led by Paul Wuh-Liang Hwu, M.D., of the National Taiwan University Hospital, surgeons used a delivery vehicle called an adeno-associated virus type 2 vector to transport the AADC gene into localized areas of the brains of three girls and a boy.

Before therapy, the children showed practically no spontaneous movement and their upper eyelids continually drooped. After receiving the corrective gene, the children gradually gained some head movement. Sixteen months afterward, the children's weight had increased, one patient was able to stand and the other three were able to sit up without support.

The study shows gene therapy that targets AADC deficiency is well-tolerated and leads to improved motor development and function, according to co-authors Barry Byrne, M.D., Ph.D., director of UF's Powell Gene Therapy Center, and Richard O. Snyder, Ph.D., director of UF's Center of Excellence for Regenerative Health Biotechnology. Both are members of the UF Genetics Institute.

"The children in this study have the most severe form of inherited movement disorder known, and the only treatments so far have been supportive ones," said Byrne, a pediatric cardiologist and associate chairman of the department of pediatrics in the College of Medicine. "It is gratifying to see it is possible to do something to help them, other than providing feeding tubes and keeping them safe. This absolutely opens the door to the possibility of even earlier treatment of neurological diseases by direct gene transfer, and has implications for Parkinson's disease, ALS and even cognitive diseases such as dementia when caused by gene defects."

The Powell Gene Therapy Center provided expertise to the Taiwanese physicians on treating the patients and engineering the corrective gene that spurs production of the absent AADC enzyme. UF's Center of Excellence for Regenerative Health Biotechnology manufactured the vector, packaging genetic material it received from Taiwan into virus particles that were purified, characterized and tested for sterility and stability before being shipped to the clinic for use in patients.

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Children with rare, incurable brain disease improve after gene therapy

ScienceDaily (May 14, 2012) A new study consisting of inducing cells to express telomerase, the enzyme which -- metaphorically -- slows down the biological clock -- was successful. The research provides a "proof-of-principle" that this "feasible and safe" approach can effectively "improve health span."

A number of studies have shown that it is possible to lengthen the average life of individuals of many species, including mammals, by acting on specific genes. To date, however, this has meant altering the animals' genes permanently from the embryonic stage -- an approach impracticable in humans. Researchers at the Spanish National Cancer Research Centre (CNIO), led by its director Maria Blasco, have demonstrated that the mouse lifespan can be extended by the application in adult life of a single treatment acting directly on the animal's genes. And they have done so using gene therapy, a strategy never before employed to combat aging. The therapy has been found to be safe and effective in mice.

The results were recently published in the journal EMBO Molecular Medicine. The CNIO team, in collaboration with Eduard Ayuso and Fatima Bosch of the Centre of Animal Biotechnology and Gene Therapy at the Universitat Autonoma de Barcelona (UAB), treated adult (one-year-old) and aged (two-year-old) mice, with the gene therapy delivering a "rejuvenating" effect in both cases, according to the authors.

Mice treated at the age of one lived longer by 24% on average, and those treated at the age of two, by 13%. The therapy, furthermore, produced an appreciable improvement in the animals' health, delaying the onset of age-related diseases -- like osteoporosis and insulin resistance -- and achieving improved readings on aging indicators like neuromuscular coordination.

The gene therapy consisted of treating the animals with a DNA-modified virus, the viral genes having been replaced by those of the telomerase enzyme, with a key role in aging. Telomerase repairs the extreme ends or tips of chromosomes, known as telomeres, and in doing so slows the cell's and therefore the body's biological clock. When the animal is infected, the virus acts as a vehicle depositing the telomerase gene in the cells.

This study "shows that it is possible to develop a telomerase-based anti-aging gene therapy without increasing the incidence of cancer," the authors affirm. "Aged organisms accumulate damage in their DNA due to telomere shortening, [this study] finds that a gene therapy based on telomerase production can repair or delay this kind of damage," they add.

'Resetting' the biological clock

Telomeres are the caps that protect the end of chromosomes, but they cannot do so indefinitely: each time the cell divides the telomeres get shorter, until they are so short that they lose all functionality. The cell, as a result, stops dividing and ages or dies. Telomerase gets around this by preventing telomeres from shortening or even rebuilding them. What it does, in essence, is stop or reset the cell's biological clock.

But in most cells the telomerase gene is only active before birth; the cells of an adult organism, with few exceptions, have no telomerase. The exceptions in question are adult stem cells and cancer cells, which divide limitlessly and are therefore immortal -- in fact several studies have shown that telomerase expression is the key to the immortality of tumour cells.

It is precisely this risk of promoting tumour development that has set back the investigation of telomerase-based anti-aging therapies.

Read more here:
First gene therapy successful against aging-associated decline: Mouse lifespan extended up to 24% with a single ...

SOUTH SAN FRANCISCO, CA--(Marketwire -05/14/12)- VistaGen Therapeutics, Inc. (OTC.BB: VSTA) (VSTA.OB), a biotechnology company applying stem cell technology for drug rescue, today issued the following letter to its stockholders and the investment community from its CEO, Shawn Singh.

To our valued Stockholders:

Since becoming a public company one year ago, we have progressed to perhaps the most exciting time in our company's 14-year history. To arrive at this point, more than $45 million, obtained through various strategic collaborations, investments and grant awards, has been carefully employed. We believe our pluripotent stem cell technology platform, Human Clinical Trials in a Test Tube, combined with the network of strategic relationships we have announced, will allow us to secure additional capital and the large market drug rescue opportunities that can deliver value to our stockholders.

Since the beginning of the year, our team has carefully reviewed our Top 10 drug rescue opportunities and narrowed our focus to our Top 5 candidates. Now we intend to launch our initial drug rescue program and secure strategic capital necessary to support it, as well as launch our second drug rescue program by year-end. We also are working on validation of LiverSafe 3D, our bioassay system for drug rescue involving liver toxicity and drug metabolism issues, for launch during the first half of next year.

The pharmaceutical industry continues to face extremely high barriers in bringing new medicine to market. The number of drugs approved by the FDA over the past decade has dropped precipitously, by over 50%, in spite of staggering increases in resources devoted to R&D by pharmaceutical companies. Based on the progress we have made with CardioSafe 3D and our efforts to build our strategic drug rescue ecosystem of collaborators, we believe our core business model -- to use our stem cell technology and strategic relationships to develop less toxic variants of drugs that have already been proven in vitro to be effective -- is now more commercially promising than at any other point in our history. We believe we will be able to help major pharmaceutical companies avoid the loss of years of time and millions of dollars spent in developing new therapies that have positive efficacy data, but must be discontinued due to later discovery of unsafe toxicity levels for human heart and liver tissue.

Over the past year, we have secured additional intellectual property protection and entered into strategic relationships with leading biotech firms and academic researchers to support development of our stem technology and our drug rescue-based commercialization initiatives:

Over the next 12 months, we have an ambitious agenda to work closely with our advisors and collaborators to secure capital and achieve these transformative milestones:

Our goals are reachable, with strategic financing. We believe we have the right technology, intellectual property, development teams and specialized focus to deliver on our founding mission -- "putting humans first" -- bringing clinically relevant human biology to the front end of the drug development process, long before standard animal and human testing, and using better cells to make better medicine.

We would like to thank our partners, advisors, employees and each of you, our loyal stockholders, for helping support us in our efforts to deliver long-term value for you.

Sincerely,

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VistaGen CEO Issues Update Letter to Stockholders

DENVER, May 14, 2012 /PRNewswire/ -- Regenerative Sciences, Inc., a company dedicated to advancing orthopedic care through non-surgical adult stem cell procedures, today announced that it has secured a $2M investment from philanthropist, visionary and businessman John C. Malone, PhD, chairman of Liberty Media Corporation. In addition to advancing Regenerative Sciences' clinical and lab-based stem cell research, the investment will help support the national expansion of their Regenexx Physician Network.

Regenerative Sciences' Regenexx procedures utilize a patient's own stem cells to help repair a broad range of common injuries and degenerative conditions, including cartilage lesions, torn ligaments and tendons, osteoarthritis and bulging spinal discs. For many, the procedures offer a viable alternative to arthroscopic surgery, open-joint surgery, or joint replacement surgery. Regenexx patients experience little or no downtime from the procedures and avoid the lengthy rehabilitation period associated with most surgical procedures.

"We are proud of our accomplishments in the field of regenerative interventional orthopedics and it's exciting that our work has drawn the attention of such a noted entrepreneur and philanthropist," said Christopher J. Centeno, M.D., Chief Executive Officer of Regenerative Sciences. "Dr. Malone shares our vision for forging the next generation of minimally invasive regenerative treatments. This investment will not only bolster our existing stem cell research programs and make our procedures available in all regions of the U.S., but it will help us maintain a leadership role in clarifying the regulatory space for physician stem cell use."

Regenerative Sciences is at the forefront of regenerative orthopedic medicine within the United States and the company is bringing the future of orthopedic treatments to patient care today.

About Regenerative Sciences

Regenerative Sciences is an outgrowth of the Centeno-Schultz clinic, where we are reinventing orthopedic care for the 21st century using key biologics such as stem cells, next generation tools and devices, and unique therapeutic approaches. Our signature initiative, Interventional Orthopedics, allows doctors to treat orthopedic conditions through injection, rather than traditional invasive surgery. The Regenexx Physician Network brings together like-minded physicians from around the country to offer more patients access to our innovative procedures. For more information on Regenerative Sciences and Regenexx procedures, visit: http://www.regenexx.com

About John C. Malone, PhD

Dr. John C. Malone holds a bachelor's degree in electrical engineering and economics from Yale University, where he was a Phi Beta Kappa and merit scholar. He also holds a master's degree in industrial management and a Ph.D. in operations research from Johns Hopkins University.

Dr. Malone is Chairman of Liberty Media Corporation, a position he has held since 1990. Dr. Malone is also the Chairman of the Board of Liberty Global, Inc. (LGI), a position he has held since June, 2005. From 1996 to March 1999 when Tele-Communications, Inc. (TCI) merged with AT&T Corp., he was also Chairman and Chief Executive Officer of TCI. Previous to that, from 1973 to 1996, Dr. Malone served as President and CEO of TCI. He currently serves on the Board of Directors for CATO Institute, Expedia, Inc., Discovery Communications, Inc., and SiriusXM.

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Originally posted here:
Regenerative Sciences Receives $2M Investment for Orthopedic Stem Cell Initiatives