Archive for the ‘Gene Therapy Research’ Category

DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/zzkm43/plunketts_biotech) has announced the addition of Plunkett Research Ltd's new book "Plunkett's Biotech & Genetics Industry Almanac 2012: Biotech & Genetics Industry Market Research, Statistics, Trends & Leading Companies" to their offering.

A complete market research report, including forecasts and market estimates, technologies analysis and developments at innovative firms.

You will gain vital insights that can help you shape your own strategy for business development, product development and investments.

- How is the industry evolving?

- How is the industry being shaped by new technologies?

- How is demand growing in emerging markets and mature economies?

- What is the size of the market now and in the future?

- What are the financial results of the leading companies?

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Research and Markets: Plunkett's Biotech & Genetics Industry Almanac 2012: Biotech & Genetics Industry Market Research ...

SAN MARINO, Calif.--(BUSINESS WIRE)--

Viral Genetics (VRAL) majority-owned subsidiary, VG Energy, has contracted with Eno Research Chemicals & Custom Synthesis Group for production of its LipidMax lipid enhancement compound, in sufficient capacity to meet demand from commercial and research end-users worldwide.

Orders for LipidMax can be placed at https://store.vgenergy.net/. Once approved for purchases, customers will be shipped LipidMax in 10g units. Additional information is also available at lipidmax@vgenergy.net.

LipidMax, the first product marketed by VG Energy, is a lipid-enhancement compound for use in the production of oils from algae, corn, palm, and other plants or plant-like organisms and is based on the Companys exclusively licensed Metabolic Disruption Technology (MDT).

Independent testing has shown that, when treated with LipidMax, the yields from several algae and algae-like species including Schizochytrium, Prymnesium parvum, and Chlamydomonas reinhardtii, have shown significant increases in oil (lipid) yields. Similar, if not stronger, results have been observed in palm and other oil-producing plants. The Company believes that LipidMax should generate oil-enhancing results in most other species of plants and plant-like organisms beyond those mentioned, and is continuing testing to validate and quantify this.

The oil yield increase is thought to provide a possible catalyst for changing the economic feasibility for algal-derived biofuels in the marketplace, making biofuels more cost-competitive, when compared to petroleum-based fuels. Similar cost-reduction effects in plant oils used as food, nutraceuticals and cosmetics should also result from productivity-enhancement.

The company joins a newly emerging category of businesses called intermediates1 in the biofuels and bio-chemical sectors. These products can be utilized in the production of an array of fuels, chemicals, flavors, fragrances and other end products. Intermediates constitute building blocks and intermediate steps upon which other processes and products are built, and as such can become integral to building the value chain in these rapidly growing bio-based industries.

VG Energys Chief Scientist, Dr. M. Karen Newell-Rogers, noted that the Eno Research Chemicals and Custom Synthesis Group is fully cGMP- and cGLP-compliant, has passed a rigorous set of internal testing protocols, and the product produced at the facility meets the companys standards for chemical consistency, purity and yield. When scaled up to commercially viable quantities, the LipidMax produced at this facility meets or exceeds the same quality standards we established using smaller research-grade batches at the Agrilife facilities. The process of commercial or industrial scale-up of a chemical such as our product can sometimes alter the ultimate activity of a compound, but LipidMax produced at scaled up production protocols has shown to match the results we observed during initial evaluations.

Haig Keledjian, Viral Genetics CEO said, This marks the transition to commercialization from a purely research based company and we are very pleased and excited to now commence sales of our LipidMax product to customers in the bio-fuels, and high quality edible and nutraceutical oil markets.

Mr. Keledjian continued, We expect that initial customers will be industrial laboratories and academic research institutions looking to establish and verify the economic advantages of LipidMax that we have demonstrated in our own and independent testing. As end users attain increased yields of usable oil in commercial size quantities, we anticipate a rapid and widespread awareness of LipidMax, leading to adoption of LipidMax in a host of industries worldwide including algal biofuel and palm oil, amongst others.

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Viral Genetics’ Subsidiary, VG Energy, Commencing Sales of LipidMax™

When Jessica Ainslie had her son Colton, she knew there was something wrong. Born prematurely, he weighed just 2 pounds, 9 ounces at birth, and he had to spend time in the neonatal intensive care unit before he could come home.

Thought to be out out of the woods, Colton then started developing breathing problems and was vomiting often, just a few months later.

It just kept getting worse. He wasnt eating a whole lot, and he couldnt keep anything down, Ainslie, from Peoria, Ariz., told FoxNews.com. We took him to the ER a couple times; they kept giving him fluids and sending him back home. But I knew there was something wrong with my kid. I had that mom gut feeling.

Multiple blood tests were done on Colton, but nothing definitive came up. Finally, one of Ainslies doctors said she was going to test for SCID, a possible diagnosis that made the new mom incredibly anxious.

For small children with severe combined immunodeficiency (SCID) more famously known as bubble boy syndrome disease and infection are a constant source of worry due to a weakened, or almost non-existent immune system. If left untreated, the condition is fatal in just one to two years.

Sure enough, Coltons blood work showed that he had ADA-deficient SCID, a form of SCID that affects 15 percent of SCID patients. While some treatments exist for those suffering from the disease, options are mostly limited to rare bone marrow matches or frequent drug injections that can last the entirety of the childs life. Though helpful, these treatments dont always work and can come with terrible side effects and high costs.

Devastated by the diagnosis, Ainslie was offered some hope when she was asked if they could Air Evac 10-month-old Colton to Dr. Donald Kohns office at UCLA, one of the leading doctors in ADA-deficient SCID. Now, after having participated in a new gene therapy study developed by Kohn, Colton and eventually his baby sister Abbygail are living normal lives, nearly SCID-free.

Be their own donor

Just about the time Colton was diagnosed, Kohn had been working on a novel gene therapy treatment for children with SCID. In collaboration with researchers from both the University of California at Los Angeles and that National Institute of Health, Kohn and his colleagues have spent 11 years creating and testing this new therapy, which is capable of restoring immune systems in children with ADA-deficient SCID.

What were doing with gene therapy is having the patient be their own donor, study author Kohn, a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, told FoxNews.com. We insert the gene theyre missing and transport it back through their system.

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New Therapy for Bubble Babies

Public release date: 11-Sep-2012 [ | E-mail | Share ]

Contact: Kim Irwin kirwin@mednet.ucla.edu 310-435-9457 University of California - Los Angeles Health Sciences

UCLA stem cell researchers have found that a gene therapy regimen can safely restore immune systems to children with so-called "Bubble Boy" disease, a life threatening condition that if left untreated can be fatal within one to two years.

In the 11-year study, researchers were able to test two therapy regimens for 10 children with ADA-deficient severe combined immunodeficiency (SCID). During the study, they refined their approach to include a light dose of chemotherapy to help remove many of the blood stem cells in the bone marrow that are not creating an enzyme called adenosine deaminase (ADA), which is critical for the production and survival of healthy white blood cells, said study senior Dr. Donald Kohn, a professor of pediatrics and of microbiology, immunology, and molecular genetics in Life Sciences and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.

The refined gene therapy and chemotherapy regimen proved superior to the other method tested in the study, restoring immune function to three of the six children who received it, Kohn said. Going forward, an even further refined regimen using a different type of virus delivery system will be studied in the next phase of the study, which already has enrolled eight of the 10 patients needed.

The study appears Aug. 30 in the advance online issue of the peer-reviewed journal Blood.

"We were very happy that in the human trials we were able to see a benefit in the patients after we modified the protocol," Kohn said. "Doctors treating ADA-deficient SCID have had too few options for too long, and we hope this will provide them with an efficient and effective treatment for this devastating disease."

Children born with SCID, an inherited immunodeficiency, are generally diagnosed at about six months. They are extremely vulnerable to infectious diseases and don't grow well. Chronic diarrhea, ear infections, recurrent pneumonia and profuse oral candidiasis commonly occur in these children. SCID cases occur in about 1 of 100,000 births

Currently, the only treatment for ADA-deficient SCID calls for injecting the patients twice a week with the necessary enzyme, Kohn said, a life-long process that is very expensive and often doesn't return the immune system to optimal levels. These patients also can undergo bone marrow transplants from matched siblings, but matches can be very rare.

About 15 percent of all SCID patients are ADA-deficient. Kohn and his team used a virus delivery system that he had developed in his lab in the 1990s to restore the gene that produces the missing enzyme necessary for a healthy immune system. To date, about 40 children with SCID have received gene therapy in clinical trials around the world, Kohn said.

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UCLA stem cell researchers use gene therapy to restore immune systems in 'bubble babies'

ScienceDaily (Sep. 11, 2012) UCLA stem cell researchers have found that a gene therapy regimen can safely restore immune systems to children with so-called "Bubble Boy" disease, a life threatening condition that if left untreated can be fatal within one to two years.

In the 11-year study, researchers were able to test two therapy regimens for 10 children with ADA-deficient severe combined immunodeficiency (SCID). During the study, they refined their approach to include a light dose of chemotherapy to help remove many of the blood stem cells in the bone marrow that are not creating an enzyme called adenosine deaminase (ADA), which is critical for the production and survival of healthy white blood cells, said study senior Dr. Donald Kohn, a professor of pediatrics and of microbiology, immunology, and molecular genetics in Life Sciences and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.

The refined gene therapy and chemotherapy regimen proved superior to the other method tested in the study, restoring immune function to three of the six children who received it, Kohn said. Going forward, an even further refined regimen using a different type of virus delivery system will be studied in the next phase of the study, which already has enrolled eight of the 10 patients needed.

The study appears Aug. 30 in the advance online issue of the peer-reviewed journal Blood.

"We were very happy that in the human trials we were able to see a benefit in the patients after we modified the protocol," Kohn said. "Doctors treating ADA-deficient SCID have had too few options for too long, and we hope this will provide them with an efficient and effective treatment for this devastating disease."

Children born with SCID, an inherited immunodeficiency, are generally diagnosed at about six months. They are extremely vulnerable to infectious diseases and don't grow well. Chronic diarrhea, ear infections, recurrent pneumonia and profuse oral candidiasis commonly occur in these children. SCID cases occur in about 1 of 100,000 births

Currently, the only treatment for ADA-deficient SCID calls for injecting the patients twice a week with the necessary enzyme, Kohn said, a life-long process that is very expensive and often doesn't return the immune system to optimal levels. These patients also can undergo bone marrow transplants from matched siblings, but matches can be very rare.

About 15 percent of all SCID patients are ADA-deficient. Kohn and his team used a virus delivery system that he had developed in his lab in the 1990s to restore the gene that produces the missing enzyme necessary for a healthy immune system. To date, about 40 children with SCID have received gene therapy in clinical trials around the world, Kohn said.

Two slightly different viral vectors were tested in the study, each modified to deliver healthy ADA genes into the bone marrow cells of the patients so the needed enzyme could be produced and make up for the cells that don't have the gene. Four of the 10 patients in the study remained on their enzyme replacement therapy during the gene therapy study. There were no side effects, but their immune systems were not sufficiently restored, Kohn said.

In the next six patients, the enzyme therapy was stopped and a small dose of chemotherapy was given before starting the gene therapy to deplete the ADA-deficient stem cells in their bone marrow. Of those patients, half had their immune systems restored. The human findings confirmed another study, also published recently in Blood by Kohn and UCLA colleague Dr. Denise Carbonaro-Sarracino, which tested the techniques in parallel, using a mouse model of ADA-deficient SCID.

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Stem cell researchers use gene therapy to restore immune systems in 'Bubble Boy' disease

Public release date: 11-Sep-2012 [ | E-mail | Share ]

Contact: Raymond MacDougall macdougallr@mail.nih.gov 301-402-0911 NIH/National Human Genome Research Institute

Researchers have demonstrated that a refined gene therapy approach safely restores the immune systems of some children with severe combined immunodeficiency (SCID). The rare condition blocks the normal development of a newborn's immune system, leaving the child susceptible to every passing microbe. Children with SCID experience chronic infections, which usually triggers the diagnosis. Their lifespan is two years if doctors cannot restore their immunity.

The findings from facilities including the National Institutes of Health, the University of California, Los Angeles (UCLA), and the Children's Hospital Los Angeles, are reported in the Sept. 11, 2012, advanced online issue of the journal Blood, the official journal of the American Society of Hematology.

In the 11-year study, the researchers tested a combination of techniques for gene therapy, arriving at one that produced normal levels of immune function for three patients.

"Doctors who treat patients with SCID have had limited treatment options for too long," said Dan Kastner, M.D., Ph.D., scientific director of the National Human Genome Research Institute (NHGRI), part of the NIH. "The research teams and the patients who have participated in the studies have together achieved an impressive advance toward a cure that is welcome news for both the scientific and patient communities."

Gene therapy is an experimental method for treating patients with genetic diseases. It is intended to integrate functioning genes among those naturally existing in the cells of the body to make up for faulty genes. Researchers in the current study tested a set of methods to improve outcomes for children with a particular form of SCID.

"This is a highly rewarding study for those of us in the clinic and lab," said Fabio Candotti, M.D., a senior author and a senior investigator in NHGRI's Genetics and Molecular Biology Branch. "Not only have we realized an important advancement in gene therapy, but we have seen a renewal of health in our patients."

While rare, SCID became widely known because of the remarkable boy-in-the-bubble story of the 1970s. The story was based in part on a boy named David Vetter, who lived for 13 years in a plastic isolation unit to protect him from infections. He died following an unsuccessful bone marrow transplant that doctors had hoped would repair his immune system.

SCID has many causes. In one type, a gene that produces the adenosine deaminase (ADA) enzyme becomes mutated and fails to produce the normal enzyme. Without ADA, a chemically altered form of adenosine, one of DNA's building blocks, accumulates in rapidly dividing bone marrow cells, killing them and destroying the immune system in the process. Normal bone marrow makes healthy white blood cells, or lymphocytes, which are the key players in the immune response that reacts against harmful bacteria and destroys cells infected by viruses. ADA deficiency accounts for some 15 percent of SCID cases.

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NIH researchers restore children's immune systems with refinements in gene therapy

Public release date: 11-Sep-2012 [ | E-mail | Share ]

Contact: Claire Gwayi-Chore cgwayi-chore@hematology.org 202-776-0544 American Society of Hematology

By including chemotherapy as a conditioning regimen prior to treatment, researchers have developed a refined gene therapy approach that safely and effectively restores the immune system of children with a form of severe combined immunodeficiency (SCID), according to a study published online today in Blood, the Journal of the American Society of Hematology (ASH).

SCID is a group of rare and debilitating genetic disorders that affect the normal development of the immune system in newborns. Infants with SCID are prone to serious, life-threatening infections within the first few months of life and require extensive treatment for survival beyond infancy.

Adenosine deaminase (ADA) deficiency, which accounts for approximately 15 percent of all SCID cases, develops when a gene mutation prohibits the production of ADA, an enzyme that breaks down toxic molecules that can accumulate to harmful levels and kill lymphocytes, the specialized white blood cells that help make up the immune system. In its absence, infants with ADA-deficient SCID lack almost all immune defenses and their condition is almost always fatal within two years if left untreated. Standard treatment for ADA-deficient SCID is a hematopoietic stem cell transplant (HSCT) from a sibling or related donor; however, finding a matched donor can be difficult and transplants can carry significant risks. An alternate treatment method, enzyme replacement therapy (ERT), involves regular injections of the ADA enzyme to maintain the immune system and can help restore immune function; however, the treatments are extremely expensive and painful for the young patients and the effects are often only temporary.

Given the limitations of HSCT and ERT, in the 1990s researchers began investigating the efficacy of gene therapy for ADA-deficient SCID. They discovered that they could "correct" the function of a mutated gene by adding a healthy copy into the cells of the body that help fight infectious diseases. Since then, there have been significant advances in gene therapy for SCID, yet successful gene therapy in patients with ADA-deficient SCID has been seen in only a small series of children due to the difficulty of introducing a healthy ADA gene into bone marrow stem cells and to engraft these cells back into the patients.

"Although the basic steps of gene therapy for patients with SCID have been known for a while, technical and clinical challenges still exist and we wanted to find an optimized gene therapy protocol to restore immunity for young children with ADA-deficient SCID," said Fabio Candotti, MD, one of the study's senior authors, senior investigator in the Genetics and Molecular Biology Branch of the National Human Genome Research Institute at the National Institutes of Health, and chair of the ASH Scientific Committee on Immunology and Host Defense.

To determine whether an enhanced gene therapy approach would improve immunity in children with ADA-deficient SCID, the teams of Dr. Candotti and Donald B. Kohn, MD, director of the Human Gene Medicine Program at the University of California, Los Angeles (UCLA), Professor of Pediatrics and of Microbiology, Immunology, and Molecular Genetics, and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, conducted a clinical trial in 10 patients with the disorder. For the first time, Drs. Candotti and Kohn and their team of investigators compared two different retroviral vectors, MND-ADA and GCsapM-ADA, to transport normal ADA genes into the young patients' bone marrow stem cells as well as two different treatment plans in preparation for receiving gene therapy. Following therapy, investigators found that more bone marrow stem cells were marked with the MND-ADA vector, demonstrating its superiority over the GCsapM-ADA vector.

The investigators also sought to determine whether providing a low dose of chemotherapy prior to gene therapy, known as a pre-transplant conditioning regimen, would successfully deplete the young patients' bone marrow stem cells and make room for gene-corrected stem cells. In four patients, gene therapy was performed without chemotherapy, and the patients remained on ERT throughout the entire procedure to evaluate the efficiency of ERT combined with gene therapy. While these patients did not experience any adverse effects, they also did not experience a significant increase in their levels of the ADA enzyme. They also maintained low absolute lymphocyte counts (ALC) and minimal immune system function, leading the researchers to believe that ERT may weaken the therapy's effect by diluting the number of gene-corrected lymphocytes.

The remaining six patients were treated with the chemotherapy drug busulfan prior to gene therapy and ERT was discontinued prior to the gene therapy procedure. A significant increase in ADA was observed in all six patients; half of them remain off of ERT with partial immune reconstitution findings that support results from prior trials in Italy and the United Kingdom using chemotherapy prior to gene therapy and discontinuting ERT. While the ALC of all six patients declined sharply in the first few months due to combined effects of busulfan administration and ERT withdrawal, their counts increased from six to 24 months, even in the three patients that remained off of ERT. After adjusting the chemotherapy dosage, investigators were able to determine an optimal level for enhancing the efficacy of the gene-therapy-corrected cells with minimal toxicity.

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Researchers improve gene therapy technique for children with immune disorder

ScienceDaily (Sep. 11, 2012) By including chemotherapy as a conditioning regimen prior to treatment, researchers have developed a refined gene therapy approach that safely and effectively restores the immune system of children with a form of severe combined immunodeficiency (SCID), according to a study published online September 11 in Blood, the Journal of the American Society of Hematology (ASH).

SCID is a group of rare and debilitating genetic disorders that affect the normal development of the immune system in newborns. Infants with SCID are prone to serious, life-threatening infections within the first few months of life and require extensive treatment for survival beyond infancy.

Adenosine deaminase (ADA) deficiency, which accounts for approximately 15 percent of all SCID cases, develops when a gene mutation prohibits the production of ADA, an enzyme that breaks down toxic molecules that can accumulate to harmful levels and kill lymphocytes, the specialized white blood cells that help make up the immune system. In its absence, infants with ADA-deficient SCID lack almost all immune defenses and their condition is almost always fatal within two years if left untreated. Standard treatment for ADA-deficient SCID is a hematopoietic stem cell transplant (HSCT) from a sibling or related donor; however, finding a matched donor can be difficult and transplants can carry significant risks. An alternate treatment method, enzyme replacement therapy (ERT), involves regular injections of the ADA enzyme to maintain the immune system and can help restore immune function; however, the treatments are extremely expensive and painful for the young patients and the effects are often only temporary.

Given the limitations of HSCT and ERT, in the 1990s researchers began investigating the efficacy of gene therapy for ADA-deficient SCID. They discovered that they could "correct" the function of a mutated gene by adding a healthy copy into the cells of the body that help fight infectious diseases. Since then, there have been significant advances in gene therapy for SCID, yet successful gene therapy in patients with ADA-deficient SCID has been seen in only a small series of children due to the difficulty of introducing a healthy ADA gene into bone marrow stem cells and to engraft these cells back into the patients.

"Although the basic steps of gene therapy for patients with SCID have been known for a while, technical and clinical challenges still exist and we wanted to find an optimized gene therapy protocol to restore immunity for young children with ADA-deficient SCID," said Fabio Candotti, MD, one of the study's senior authors, senior investigator in the Genetics and Molecular Biology Branch of the National Human Genome Research Institute at the National Institutes of Health, and chair of the ASH Scientific Committee on Immunology and Host Defense.

To determine whether an enhanced gene therapy approach would improve immunity in children with ADA-deficient SCID, the teams of Dr. Candotti and Donald B. Kohn, MD, director of the Human Gene Medicine Program at the University of California, Los Angeles (UCLA), Professor of Pediatrics and of Microbiology, Immunology, and Molecular Genetics, and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, conducted a clinical trial in 10 patients with the disorder. For the first time, Drs. Candotti and Kohn and their team of investigators compared two different retroviral vectors, MND-ADA and GCsapM-ADA, to transport normal ADA genes into the young patients' bone marrow stem cells as well as two different treatment plans in preparation for receiving gene therapy. Following therapy, investigators found that more bone marrow stem cells were marked with the MND-ADA vector, demonstrating its superiority over the GCsapM-ADA vector.

The investigators also sought to determine whether providing a low dose of chemotherapy prior to gene therapy, known as a pre-transplant conditioning regimen, would successfully deplete the young patients' bone marrow stem cells and make room for gene-corrected stem cells. In four patients, gene therapy was performed without chemotherapy, and the patients remained on ERT throughout the entire procedure to evaluate the efficiency of ERT combined with gene therapy. While these patients did not experience any adverse effects, they also did not experience a significant increase in their levels of the ADA enzyme. They also maintained low absolute lymphocyte counts (ALC) and minimal immune system function, leading the researchers to believe that ERT may weaken the therapy's effect by diluting the number of gene-corrected lymphocytes.

The remaining six patients were treated with the chemotherapy drug busulfan prior to gene therapy and ERT was discontinued prior to the gene therapy procedure. A significant increase in ADA was observed in all six patients; half of them remain off of ERT with partial immune reconstitution -- findings that support results from prior trials in Italy and the United Kingdom using chemotherapy prior to gene therapy and discontinuting ERT. While the ALC of all six patients declined sharply in the first few months due to combined effects of busulfan administration and ERT withdrawal, their counts increased from six to 24 months, even in the three patients that remained off of ERT. After adjusting the chemotherapy dosage, investigators were able to determine an optimal level for enhancing the efficacy of the gene-therapy-corrected cells with minimal toxicity.

This study is the first to detail comparisons of ADA-deficient SCID patient outcomes between those treated with gene therapy who have not received pre-transplant conditioning while continuing to receive ERT with those receiving pre-transplant conditioning without the administration of ERT. This study is also the first to compare two different viral vectors to transport normal ADA genes into patient bone marrow.

"We were very happy that in this trial we were able to see a benefit in the patients after we modified the protocol," said Dr. Kohn. "Doctors treating ADA-deficient SCID have had too few options for too long, and we hope this will provide them with an efficient and effective treatment for this devastating disease."

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Gene therapy technique for children with immune disorder improved

MULTIMEDIA ALERT: Video resources, including an interview with Dr. Crawford, are available for journalists at the Mayo Clinic News Network.

Newswise JACKSONVILLE, Fla. More than a cancer-causing gene is needed to trigger pancreatic cancer, a study led by Mayo Clinic has found. A second factor creates a perfect storm that allows tumors to form, the researchers say. The study, published in the Sept. 10 issue of Cancer Cell, overturns the current belief that a mutation in the KRAS oncogene is enough to initiate pancreatic cancer and unrestrained cell growth.

The findings uncover critical clues on how pancreatic cancer develops and why few patients benefit from current therapies. The findings also provide ideas about how to improve treatment and prevention of pancreatic cancer.

The research team, led by Howard C. Crawford, Ph.D., a cancer biologist at Mayo Clinics campus in Florida, and Jens Siveke, M.D., at Technical University in Munich, Germany, found that for pancreatic cancer to form, mutated KRAS must recruit a second player: the epidermal growth factor receptor, or EGFR.A third genetic participant known as Trp53 makes pancreatic tumors very difficult to treat, the study showed.

The scientists also found that EGFR was required in pancreatic cancer initiated by pancreatic inflammation known as pancreatitis.

We believe the perfect storm needed to trigger pancreatic cancer include KRAS mutations and inflammation in the organ, which then work synergistically to turn on EGFR, says Dr. Crawford.

The bottom line is, without EGFR, tumors dont form and that was never known before this study, he says. We also think that inflammation in the pancreas has a big impact on turning on EGFR.

The researchers discovered that when they blocked EGFR activity, the mice studied were protected against developing chronic pancreatitis and pancreatic cancer.

They further found that in mice that had lost expression of the TP53 tumor suppressor a situation that mirrors up to 60 percent of human pancreatic cancer cases tumors escape the dependency on EGFR for initiation and continued growth of pancreatic cancer, Dr. Crawford says.

Pancreatic cancer is a highly lethal disease; no drug has been able to target the mutant KRAS protein. The study suggests some patients, such as those with chronic pancreatitis, may be good candidates for treatment with EGFR inhibitors to fight or prevent pancreatic cancer, Dr. Crawford says.

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Cancer-Causing Gene Alone Doesn't Trigger Pancreatic Cancer

Public release date: 10-Sep-2012 [ | E-mail | Share ]

Contact: Kevin Punsky punsky.kevin@mayo.edu 904-953-2299 Mayo Clinic

JACKSONVILLE, Fla. More than a cancer-causing gene is needed to trigger pancreatic cancer, a study led by Mayo Clinic has found. A second factor creates a "perfect storm" that allows tumors to form, the researchers say. The study, published in the Sept. 10 issue of Cancer Cell, overturns the current belief that a mutation in the KRAS oncogene is enough to initiate pancreatic cancer and unrestrained cell growth.

The findings uncover critical clues on how pancreatic cancer develops and why few patients benefit from current therapies. The findings also provide ideas about how to improve treatment and prevention of pancreatic cancer.

The research team, led by Howard C. Crawford, Ph.D., a cancer biologist at Mayo Clinic's campus in Florida, and Jens Siveke, M.D., at Technical University in Munich, Germany, found that for pancreatic cancer to form, mutated KRAS must recruit a second player: the epidermal growth factor receptor, or EGFR.A third genetic participant known as Trp53 makes pancreatic tumors very difficult to treat, the study showed.

The scientists also found that EGFR was required in pancreatic cancer initiated by pancreatic inflammation known as pancreatitis.

"We believe the perfect storm needed to trigger pancreatic cancer include KRAS mutations and inflammation in the organ, which then work synergistically to turn on EGFR," says Dr. Crawford.

"The bottom line is, without EGFR, tumors don't form and that was never known before this study," he says. "We also think that inflammation in the pancreas has a big impact on turning on EGFR."

The researchers discovered that when they blocked EGFR activity, the mice studied were protected against developing chronic pancreatitis and pancreatic cancer.

They further found that in mice that had lost expression of the TP53 tumor suppressor a situation that mirrors up to 60 percent of human pancreatic cancer cases tumors escape the dependency on EGFR for initiation and continued growth of pancreatic cancer, Dr. Crawford says.

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Cancer-causing gene alone doesn't trigger pancreatic cancer, Mayo-led study finds

ScienceDaily (Sep. 10, 2012) Two different genetic mutations cooperate to induce adrenal cancer, according to a new study from researchers at the University of Michigan Comprehensive Cancer Center and University of Sao Paulo in Brazil.

The finding provides new clues to this rare and deadly cancer type, and researchers hope it will lead to better treatments by targeting both mutations.

About 600 Americans are diagnosed with adrenal cancer per year. It is typically diagnosed in late stages when there is nearly no chance of survival beyond five years.

"Because adrenal cancer is so rare, it has been challenging to find enough patients who can provide tissue samples for research. Only through collaboration can we do this," says senior study author Gary Hammer, M.D., Ph.D., the Millie Schembechler Professor of Adrenal Cancer at the University of Michigan Comprehensive Cancer Center.

The partnership between U-M and Sao Paulo has allowed researchers to collect tissue samples from 118 people with benign or cancerous adrenal tumors.

"Our goal is to understand these tumors and the genes that are critical lynchpins so that we can develop treatments that extend patients' lives," Hammer says.

By studying both benign and cancerous adrenal tissue samples, the researchers found aberrations in two genetic pathways: beta-catenin and insulin-like growth factor 2, or IGF-2. The benign tumors had a high percentage of mutations in beta-catenin, but IGF2 up-regulation was rare. On the other hand, most of the adrenal cancers exhibited IGF-2 up-regulation. Cancers that also had beta-catenin mutation were associated with high-grade disease and worse survival, compared to tumors with only IGF-2 up-regulation.

The researchers additionally tested this finding experimentally by inducing individual or combined mutations in beta-catenin and IGF-2 in the mouse adrenal. The mice developed cancer only when both mutations were present.

Results of the study appear in the September issue of the American Journal of Pathology.

The next step is to develop treatments that block both beta-catenin and IGF2.

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Two gene mutations drive adrenal cancer

Public release date: 10-Sep-2012 [ | E-mail | Share ]

Contact: Nicole Fawcett nfawcett@umich.edu 734-764-2220 University of Michigan Health System

This press release is available in Portuguese.

ANN ARBOR, Mich. Two different genetic mutations cooperate to induce adrenal cancer, according to a new study from researchers at the University of Michigan Comprehensive Cancer Center and University of Sao Paulo in Brazil.

The finding provides new clues to this rare and deadly cancer type, and researchers hope it will lead to better treatments by targeting both mutations.

About 600 Americans are diagnosed with adrenal cancer per year. It is typically diagnosed in late stages when there is nearly no chance of survival beyond five years.

"Because adrenal cancer is so rare, it has been challenging to find enough patients who can provide tissue samples for research. Only through collaboration can we do this," says senior study author Gary Hammer, M.D., Ph.D., the Millie Schembechler Professor of Adrenal Cancer at the University of Michigan Comprehensive Cancer Center.

The partnership between U-M and Sao Paulo has allowed researchers to collect tissue samples from 118 people with benign or cancerous adrenal tumors.

"Our goal is to understand these tumors and the genes that are critical lynchpins so that we can develop treatments that extend patients' lives," Hammer says.

By studying both benign and cancerous adrenal tissue samples, the researchers found aberrations in two genetic pathways: beta-catenin and insulin-like growth factor 2, or IGF-2. The benign tumors had a high percentage of mutations in beta-catenin, but IGF2 up-regulation was rare. On the other hand, most of the adrenal cancers exhibited IGF-2 up-regulation. Cancers that also had beta-catenin mutation were associated with high-grade disease and worse survival, compared to tumors with only IGF-2 up-regulation.

The rest is here:
Researchers find 2 gene mutations drive adrenal cancer

ScienceDaily (Sep. 10, 2012) More than a cancer-causing gene is needed to trigger pancreatic cancer, a study led by Mayo Clinic has found. A second factor creates a "perfect storm" that allows tumors to form, the researchers say. The study, published in the Sept. 10 issue of Cancer Cell, overturns the current belief that a mutation in the KRAS oncogene is enough to initiate pancreatic cancer and unrestrained cell growth.

The findings uncover critical clues on how pancreatic cancer develops and why few patients benefit from current therapies. The findings also provide ideas about how to improve treatment and prevention of pancreatic cancer.

The research team, led by Howard C. Crawford, Ph.D., a cancer biologist at Mayo Clinic's campus in Florida, and Jens Siveke, M.D., at Technical University in Munich, Germany, found that for pancreatic cancer to form, mutated KRAS must recruit a second player: the epidermal growth factor receptor, or EGFR.A third genetic participant known as Trp53 makes pancreatic tumors very difficult to treat, the study showed.

The scientists also found that EGFR was required in pancreatic cancer initiated by pancreatic inflammation known as pancreatitis.

"We believe the perfect storm needed to trigger pancreatic cancer include KRAS mutations and inflammation in the organ, which then work synergistically to turn on EGFR," says Dr. Crawford.

"The bottom line is, without EGFR, tumors don't form -- and that was never known before this study," he says. "We also think that inflammation in the pancreas has a big impact on turning on EGFR."

The researchers discovered that when they blocked EGFR activity, the mice studied were protected against developing chronic pancreatitis and pancreatic cancer.

They further found that in mice that had lost expression of the TP53 tumor suppressor -- a situation that mirrors up to 60 percent of human pancreatic cancer cases -- tumors escape the dependency on EGFR for initiation and continued growth of pancreatic cancer, Dr. Crawford says.

Pancreatic cancer is a highly lethal disease; no drug has been able to target the mutant KRAS protein. The study suggests some patients, such as those with chronic pancreatitis, may be good candidates for treatment with EGFR inhibitors to fight or prevent pancreatic cancer, Dr. Crawford says.

"The clinical implications of this study are exciting. It suggests that pancreatic cancer patients with normal p53 activity, as well as patients with chronic pancreatitis, may be good candidates for treatment with EGFR inhibitors," Dr. Crawford says.

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Cancer-causing gene alone doesn’t trigger pancreatic cancer, research finds

CPG manufacturers may be on the cusp of monumental change as voters in California contemplate a hotly contested ballot initiative to require labeling of genetically modified foods.

Food marketers will face tough choices should the measure pass, as about 70% of processed foods sold in supermarkets contain GM ingredients like corn and soy. Some estimate that 100,000 or more foods sold in California contain some level of GE ingredients and would therefore be affected.

The mandate would be limited to the Golden State, but the implications for companies that choose not to move away from GM ingredients in advance of the July 1, 2014, deadline could be as far-reaching as consumer awareness spreads.

While the government deems genetically modified organisms safe, Californians want to judge for themselves. A Pepperdine University poll found that if the election were held last month, Californians would pass the proposition by a 3-1 margin.

To avoid the partially produced with genetic engineering label and possible consumer backlash, suppliers will likely reformulate product with more costly non-GE foods or organic ingredients, just as theyve done in countries where genetic modification disclosure is required.

Read more: Prop 37 Battle Rages in California

A recent study commissioned by the No on 37, Stop the Deceptive Food Labeling Scheme campaign, of which the Grocery Manufacturers Association is a chief sponsor, bears this out.

It projects that reformulations to non-GE and organic ingredients, which by law cannot be genetically modified, will be the most likely course taken by food producers.

Read more: California GMO Bill Is Top Priority for GMA

Retailers might also adjust their sourcing policies to gain consumer favor by incorporating more organic foods and those that have been verified under the Non-GMO Project and labeled with its seal.

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Mandatory GM Labeling Would Require Major Change

Public release date: 10-Sep-2012 [ | E-mail | Share ]

Contact: John Sterling jsterling@genengnews.com 914-740-2196 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, September 10, 2012-- Therapies for retinal diseases are expected to overtake those for glaucoma by 2014, reports Genetic Engineering & Biotechnology News (GEN). Because current retinal disease treatments only improve vision for six to eight weeks, there is a critical need for new remedies, according to a recent issue of GEN.

"As increasing numbers of baby-boomers continue to grow older, many will have to deal with eye diseases such as age-related macular degeneration," said John Sterling, Editor-in-Chief of GEN. "Some estimates put the current AMD and diabetic retinopathy drug segment of the market at $3 billion, and this is expected to increase to about $5 billion in two years."

Standard therapy has been Genentech's VEGF inhibitors Lucentis and the off-label use of Avastin. Regeneron, in collaboration with Bayer HealthCare, is challenging these drugs with a similar VEGF inhibitor, Eyela. The FDA approved the drug last November for wet AMD.

In another approach, Acucela is in Phase II trials using visual cycle modulators to lighten the metabolic load on the retina by reducing the activity of the rod visual system. This protects the retina from light damage, improves retinal vasculature, and reduces the accumulation of A2E and other retinal-related toxic by-products.

GlaxoSmithKline has two drugs in Phase II trials for ocular therapy: darapladib, an oral Lp-PLA2 inhibitor for diabetic macular edema, and Votrient, a multi-kinase angiogenesis inhibitor in eye drop form for AMD. Early-stage work also is under way for neovascular AMD, dry AMD, diabetic retinopathy, diabetic macula edema, uveitis, and glaucoma, as well as for technologies for drug delivery.

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Other companies covered in the GEN article include Acucela, pSiveda, Sanofi, NeuroTech, QLT, Applied Genetic Technologies, RetroSense Therapeutics, Aerie Pharmaceuticals, Kowa Pharmaceuticals America, Novartis, Senju Pharmaceutical, Can-Fite Biopharma, Inotek Pharmaceuticals, Otsuka Pharmaceutical, and Santen Pharmaceutical.

For a copy of the September 1 issue of GEN, please call (914) 740-2146, or email: pbartell@genengnews.com

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GEN reports on ocular therapeutics targeting the retina

ScienceDaily (Sep. 10, 2012) The protein GATA2 is known as a "master regulator" of blood cell development. When a mutation occurs in the gene that makes GATA2, serious blood diseases such as acute myeloid leukemia can result.

Zooming in on the GATA2 gene, UW-Madison researchers and their collaborators at the National Institutes of Health (NIH) have discovered unexpectedly that a small DNA sequence drives this powerful master regulator. The sequence plays an essential role in controlling GATA2 production and generating self-renewing blood stem cells responsible for the earliest steps in the development of blood cells of all kinds -- red cells to transport oxygen and white cells to fight infection.

The researchers also found that the DNA sequence, which they call the +9.5 GATA2 switch site, ensures that blood vessels function properly to prevent hemorrhaging. Until now, GATA2 had not been implicated in blood vessel integrity.

The study appears in The Journal of Clinical Investigation (online Sept. 10, 2012).

Although the study was performed in mice, it should have significant clinical relevance, particularly to physicians and scientists aiming to understand certain types of leukemia and related disorders involving disruptions in the blood and immune systems. The research indicates that downstream genes are impacted when the switch site is altered, leading to abnormal development of the adult blood system, says senior author Dr. Emery Bresnick, professor of cell and regenerative biology at the School of Medicine and Public Health.

"There's every reason to believe that we can use these findings as a foundation to discover key factors and signals that can be modulated therapeutically for the treatment of specific blood and blood vessel disorders," he says.

Bresnick has studied GATA proteins for more than a decade. Among other things, his team discovered five "hot spots" on the GATA2 gene where special activity involving both GATA1 and GATA2 appeared to be taking place. They named these GATA switch sites. Over the last four years, the group has focused on their third GATA2 switch site, +9.5, a small sequence of about 25 base pairs located in a region of the gene called the intron.

"While introns can contain regulatory sequences that control gene activity, until now we and others have been unable to find the mechanisms that control the GATA2 gene, despite years of studying this problem," Bresnick says.

Senior scientist Dr. Kirby Johnson from the Bresnick laboratory led experimental efforts to genetically engineer and breed mice in which the site was knocked out, and then studied the consequences of the mutation.

Without the sequence, the embryos died on day 14, but before that they developed massive hemorrhaging. Closer analysis showed that large veins in the embryo leaked embryonic red blood cells.

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New genetic mechanism for controlling blood cell development and blood vessel integrity found

By Leila B. Salaverria Philippine Daily Inquirer

Former President and now Pampanga Rep. Gloria Macapagal Arroyo: Stem cell treatment

MANILA, PhilippinesLike her predecessor, former President and Pampanga lawmaker Gloria Macapagal Arroyo has turned to stem cell therapy in an effort to improve her health.

Arroyo said in her official Twitter account that she would have her fourth stem cell intravenous treatment with her alternative medicine doctor on Monday.

Arroyo said her treatment would involve cultured stem cells, and it would be much more modest in price than the one coming from sheep or ones own body.

A close friend and ally of Arroyo, Quezon Representative Danilo Suarez, confirmed that the President has started stem cell therapy, and that she told him that the stem cells she has been using did not come from lamb placenta, and was the less costly form of stem cell treatment.

If you have a lot of health problems, you will try such things, Suarez said on Sunday.

Suarez said he has even filed a resolution to investigate the practice of stem cell treatments in the country, as well as the claims being made about it, considering that it has been gaining popularity.

The public needs to be better informed about it. It might have setbacks that we need to know about, he said.

The therapy involves the use of fresh cells, which are injected into the body to regenerate cells to treat illnesses or reverse aging.

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Arroyo undergoes 4th stem cell treatment

Public release date: 9-Sep-2012 [ | E-mail | Share ]

Contact: Julia Evangelou Strait straitj@wustl.edu 314-286-0141 Washington University School of Medicine

A nationwide consortium of scientists has reported the first comprehensive genetic analysis of squamous cell carcinoma of the lung, a common type of lung cancer responsible for about 400,000 deaths each year.

"We found that almost 75 percent of the patients' cancers have mutations that can be targeted with existing drugs -- drugs that are available commercially or for clinical trials," says one of the lead investigators, Ramaswamy Govindan, MD, an oncologist at Washington University School of Medicine in St. Louis and co-chair of the lung cancer group of The Cancer Genome Atlas.

The research appears online Sept. 9 in Nature.

The Cancer Genome Atlas project combines efforts of the nation's leading genetic sequencing centers, including The Genome Institute at Washington University, to describe the genetics of common tumors with the goal of improving prevention, detection and treatment. The Cancer Genome Atlas is supported by the National Cancer Institute and the National Human Genome Research Institute, both parts of the National Institutes of Health (NIH).

The other lung cancer co-chairs are the study's senior author Matthew Meyerson, MD, PhD, of the Broad Institute of Massachusetts Institute of Technology and Harvard University, and Stephen Baylin, MD, of Johns Hopkins University.

The study examined the tumors and normal tissue of 178 patients with lung squamous cell carcinoma. The investigators found recurring mutations common to many patients in 18 genes. And almost all of the tumors showed mutations in a gene called TP53, known for its role in repairing damaged DNA.

Interestingly, the researchers noted that lung squamous cell carcinoma shares many mutations with head and neck squamous cell carcinomas, supporting the emerging body of evidence that cancers may be more appropriately classified by their genetics rather than the primary organ they affect.

"We clearly see mutations in lung cancer that we see in other human cancers," says Richard K. Wilson, PhD, director of The Genome Institute at Washington University. "This reinforces something that we've been seeing in a lot of our cancer genomics work. It's really less about what type of tissue the tumor arises in lung, breast, skin, prostate and more about what genes and pathways are affected."

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Large lung cancer study shows potential for more targeted therapies

A genetic cause for a rare form of epilepsy-associated autism has been identified by UC San Diego and Yale scientists.

Moreover, symptoms of the newly discovered form have been reversed in mouse models by altering diet. This gives rise to the possibility that similar treatment might help people, the researchers said.The study was published online Thursday in the journal Science.

Researchers led by Gaia Novarino and Joseph G. Gleeson of UCSD studied two families, one of Egyptian descent and another of Turkish origin. They examined the genome of patients and healthy relatives for exons, gene sequences that code for proteins. The researchers found that patients shared an exon mutation on a gene called BCKDK. The mutant gene is recessive, meaning that it must be inherited from both mother and father to manifest.

Moreover, the researchers found that the mutation caused patients to produce abnormally low levels of certain types of amino acids, the building blocks of proteins. They were able to boost levels of these amino acids to normal with a nutritional supplement from a health food store. Research is now ongoing as to whether this supplementation will reduce symptoms of epilepsy and autism in these patients.

Those who might be helped are only a small fraction of people with autism, Novarino said in an Tuesday interview. Those without the metabolic defect wouldn't benefit from the supplementation.

The study illustrates how scientists have become more sophisticated in using knowledge of the human genome to crack the puzzle of previously intractable diseases.The genome is the complete set of hereditary information encoded in DNA.

Narrowing the search

The vast majority of DNA does not code for proteins, the body's workhorse molecules. This "non-coding" DNA was ignored in the new method of DNA analysis, called "whole exome" sequencing, which looks only at the exons. An advantage of whole exome sequencing is that it focuses exclusively on proteins, which are altered or missing in genetic diseases.

Whole exome sequencing can find previously undiscovered genetic diseases, according to another study performed by some of the same UCSD researchers. They examined 118 patients diagnosed with neurological disorders who had no known genetic disease causes. In addition to the newly discovered genetic causes, in about 10 percent of cases the researchers even found a known disease-causing gene that had previously escaped detection.

That study was published in June in Science Translational Medicine, a journal devoted to getting research discoveries into the hands of doctors more quickly.

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SCI-TECH: Autism gene found by UCSD researchers

DONNA WALSH/Fairfax NZ

NEW GENES: Monsanto says orchard owners like Cambridge couple Mark and Robyn Gardiner, whose kiwifruit were struck by Psa in 2010, could be helped by the introduction of GE crops.

A kiwifruit industry crippled by Psa disease is ripe for introducing genetic modification, says a visiting American biotechnology advocate.

It's estimated Psa disease will damage the kiwifruit industry to the tune of $310-$410 million over the next five years.

So a genetically-engineered solution to the disease could have significant economic benefits, said Robert Reiter, vice-president of American agrochemical giant Monsanto.

New Zealand's not a large market for any given crop. But kiwifruit might be something because there's a market in multiple places beyond New Zealand," Robert Reiter told the Star-Times.

Monsanto is regarded by detractors as a global peddler of destruction, creating foods that threaten the health of both people and the planet. Its supporters see its innovation as the world's best hope of tackling a global food crisis.

With the world's population expected to reach nine billion by 2050, and with about 40 per cent of the earth's land used for agriculture, corporations like Monsanto tout biotechnology as the best way to develop crops that are resistant to pests, drought and weeds.

Reiter said although New Zealand didn't yet need GM crops like soy and wheat, it could use the technology.

''It's giving growers a choice of different chemistries."

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Monsanto wants the GE genie out of the bottle

Former President and now Pampanga Rep. Gloria Macapagal-Arroyo will undergo stem cell therapy on Monday with an alternative medicine doctor.

Arroyo, in a post on her Twitter account Saturday morning, said Monday's session will be her fourth intravenous treatment.

"This Monday I will have my fourth stem cell intravenous treatment with my alternative medicine doctor," she said.

Also she said, "It's cultured stem cell and much more modest in price than the one coming from sheep or one's own body."

But she did not elaborate on how much the treatment will cost.

Stem cell therapy is type of intervention strategy that introduces new adult stem cells into damaged tissue in order to treat disease or injury.

Earlier this week, Arroyo said she continues to search for alternative solutions to an anatomic problem that prompted her to be rushed to a government hospital last month.

Arroyo said she had seen at least two "alternative medicine practitioners," and has initiated communication with a "neurocervical spine purist."

She said she also had her thrice-weekly therapy at the Veterans Memorial Medical Center (VMMC) in Quezon City Thursday.

Arroyo underwent treatment last August for an anatomic problem that caused her to choke on her food.

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Gloria Arroyo to have stem cell treatment Monday

Public release date: 6-Sep-2012 [ | E-mail | Share ]

Contact: Sally Stewart Sally.stewart@cshs.org 310-248-6566 Cedars-Sinai Medical Center

LOS ANGELES Sept. 6, 2012 A team of Cedars-Sinai Heart Institute stem cell researchers today was awarded a $1.3 million grant from the California Institute of Regenerative Medicine to continue study of an experimental stem cell therapy that treats heart attack patients with heart-derived cells. Earlier this year, data from the first clinical trial of the stem cell treatment showed the therapy helped damaged hearts regrow healthy muscle.

To date, this cell therapy, developed by Eduardo Marbn, MD, PhD, director of the Cedars-Sinai Heart Institute and Mark S. Siegel Family Professor, is the only treatment shown to regenerate the injured human heart. In this therapy, human heart tissue is used to grow specialized heart stem cells, which then are injected back into the patient's heart. The new research will focus on understanding the cellular mechanisms that have produced favorable outcomes.

"We have seen encouraging results in patients with this treatment, and it has the potential to revolutionize how we treat heart attack patients," Marbn said. "This further study will allow us to better understand how it works, which we hope will lead us to even more stem-cell based treatments for the heart."

During a heart attack, clots form suddenly on top of cholesterol-laden plaques, which block the flow of blood to the heart muscle. This causes living heart tissue to die and be replaced by a scar. The larger the scar, the higher the chance of death or disability from the heart attack.

Conventional treatments aim to limit the initial injury by opening the clogged artery and prevent further harm with medications. Regenerative therapy aims to regrow healthy heart muscle and dissolve the heart tissue -- an approach that, according to a study by Marbn published in The Lancet, led to an average 50 percent reduction in scar size.

Early study by Cedars-Sinai researchers indicates that much of the benefit in the experimental therapy is due to an indirect effect of the transplanted cardiac-derived cells. These cells seem to stimulate proliferation of the surrounding undamaged heart cells -- a previously unrecognized means of cardiac regeneration in response to cell therapy.

"This is vital basic science work that we believe will ultimately open pathways to new treatments in the fight against heart disease, the leading cause of premature death and disability," Marbn said.

The process to grow the cardiac-derived stem cells involved in the study was developed by Marbn when he was on the faculty of Johns Hopkins University. The university has filed for a patent on that intellectual property, and has licensed it to a company in which Dr. Marbn has a financial interest.

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Cedars-Sinai Heart Institute awarded $1.3 million to study cardiac stem cells

Public release date: 7-Sep-2012 [ | E-mail | Share ]

Contact: Matt Shipman matt_shipman@ncsu.edu 919-515-6386 North Carolina State University

New research led by researchers at North Carolina State University shows that exposure to the chemical bisphenol A (BPA) early in life results in high levels of anxiety by causing significant gene expression changes in a specific region of the brain called the amygdala. The researchers also found that a soy-rich diet can mitigate these effects.

"We knew that BPA could cause anxiety in a variety of species, and wanted to begin to understand why and how that happens," says Dr. Heather Patisaul, an associate professor of biology at NC State and lead author of a paper describing the work. BPA is a chemical used in a wide variety of polycarbonate plastics and epoxy resins, and is used in consumer products such as some food containers.

In the study, rats were exposed to low doses of BPA during gestation, lactation (nursing) and through puberty. One group of animals was fed only soy; one group was fed a soy-free diet; one group was fed only soy and exposed to BPA; and one group was fed no soy and exposed to BPA. Blood tests showed that the animals exposed to BPA had BPA levels well within the range found in humans. Similarly, blood tests of animals fed soy showed levels of genistein, an estrogen-like chemical found in soy, were at levels within the human range for vegetarians and others who regularly consume soy foods.

Among adolescent rats on the soy-free diet, both males and females that had been exposed to BPA exhibited significantly higher levels of anxiety. The researchers also found, for the first time, gene changes within the brain associated with this elevated anxiety.

Specifically, the study reveals that gene expression changes in the amygdala, a brain region known to play a role in mediating responses to fear and stress, are associated with the behavioral changes. Two of the affected genes were estrogen receptor beta and the melanocortin receptor 4. Both are required for oxytocin release, thus changes in oxytocin/vasopressin signaling pathways may underpin the behavioral changes. Oxytocin is a hormone and neurotransmitter that has been linked to social behavior.

However, the researchers also found that adolescent rats on the soy-rich diet did not exhibit anxiety suggesting that the soy-rich diet may mitigate the effects of BPA. But a soy-rich diet raises questions of its own.

"Soy contains phytoestrogens that can also affect the endocrine system, which regulates hormones," Patisaul says. "It is not clear whether these phytoestrogens are what mitigate the effect of BPA, or if it is something else entirely. That's a question we're hoping to address in future research."

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Study finds how BPA affects gene expression, anxiety; Soy mitigates effects

Sept. 7, 2012 -- In what scientists call the biggest breakthrough in genetics since the unraveling of the human genome, a massive research effort now shows how the genome works.

The human genome contains 3 billion letters of code containing a persons complete genetic makeup.

The biggest surprise is that most of the DNA in the genome -- which had been called "junk DNA" because it didn't seem to do anything -- turns out to play a crucial role. While only 2% of the genome encodes actual genes, at least 80% of the genome contains millions of "switches" that not only turn genes on and off, but also tell them what to do and when to do it.

Eleven years ago, the Human Genome Project discovered the blueprint carried by every cell in the body. The new ENCODE project now has opened the toolbox each cell uses to follow its individual part of the blueprint. The effort is the work of more than 400 researchers who performed more than 1,600 experiments.

The genome, with its 3-billion-letter code, reads from beginning to end like a book. But in real life, the genome isn't read like a book. The ENCODE data shows it's an intricate dance, with each step carefully choreographed.

Ewan Birney, PhD, associate director of the European Molecular Biology Laboratory, was one of the leaders of the Human Genome Project. He also helped lead the ENCODE project.

"The ENCODE data is just amazing. It shows how complex the human genome is," Birney said at a news conference. "This is the science for this century. We are going to be working out how we make humans, starting out from a simple instruction manual."

"We think this will lead to changes in medicine and therapeutic treatment of disease," Michael Snyder, PhD, director of Stanford University's Center of Genomics and Personalized Medicine, said the conference.

Eric D. Green, MD, PhD, director of the NIH's National Human Genome Research Institute (NHGRI), notes that most known disease-causing DNA mutations are in the small part of the genome that encodes genes.

"Most of these known mutations cause rare diseases," Green said. "But for the great majority of diseases, it's changes in the switches themselves. Diseases that are more common probably involve multiple DNA variants outside the genes. Common diseases like hypertension, heart disease, and diabetes are probably caused by things sitting in these switches."

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Genetics Breakthrough Changes Thinking About DNA

Public release date: 7-Sep-2012 [ | E-mail | Share ]

Contact: Cathy Kolf ckolf@jhmi.edu 443-287-2251 Johns Hopkins Medicine

A team of scientists from Johns Hopkins and other institutions report that restoring tiny, hair-like structures to defective cells in the olfactory system of mice is enough to restore a lost sense of smell. The results of the experiments were published online this week in Nature Medicine, and are believed to represent the first successful application of gene therapy to restore this function in live mammals.

An expert in olfaction, Randall Reed, Ph.D., professor of molecular biology and genetics and co-director of the Center for Sensory Biology at the Johns Hopkins Institute for Basic Biomedical Sciences, cautions that researchers are still years away from applying the same therapy in people, and that if and when it comes, it will likely be most effective for those who suffer from anosmia (lack of smell) due to inherited genetic disorders. "But our work has already contributed to a better understanding of the cellular factors involved in anosmia, and that will give us insights into other neurological disorders, as well," he says.

The mice used in the current study carried a genetic mutation that destroyed the production of a protein critical for the functioning of cilia in the cells responsible for smell, called olfactory sensory neurons. These specialized cells each display several of the protruding, hair-like structures that contain receptors for odorants. Without functional cilia, the cells become a broken link in the chain of events necessary for proper odor detection in the environment, the researchers explained.

Beginning with a common cold virus, which readily infects the cells of the nasal cavity, researchers replaced some of the viral genes with a corrected version of the defective cilia gene. They then infected smelling-impaired mice with the altered virus, delivering the corrected gene to the olfactory neural cells that needed it.

At the cellular level, scientists saw a restoration of proper chemical signaling between nerve cells after the treated mice were stimulated with various odorants. Perhaps even more indicative of their success, Reed says, was the 60 percent increase in body weight that the mice experienced once they could smell their meals, leading to increased appetite. Many people with anosmia lose weight because aromas play a significant part in creating appetite and food enjoyment.

Researchers are optimistic about the broader implications of this work, Reed notes, because cilia are not only important to olfactory cells, but also to cells all over the body, from the kidney to the eye. The fact that they were able to treat live mice with a therapy that restored cilia function in one sensory system suggests that similar techniques could be used to treat cilia disorders elsewhere.

"We also hope this stimulates the olfactory research community to look at anosmia caused by other factors, such as head trauma and degenerative diseases," says senior author Jeffrey Martens, Ph.D., an associate professor of pharmacology at the University of Michigan. "We know a lot about how this system works now have to look at how to fix it when it malfunctions."

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The nose knows: Gene therapy restores sense of smell in mice