Archive for the ‘Gene Therapy Research’ Category

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Gene Therapy - the impossible dream?
Gene Therapy - the impossible dream?

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10/01/2014 - 07:15:45Back to World Home

A gene therapy for Parkinsons disease has produced promising results in its first patient trial, say researchers.

The ProSavin treatment uses an inert virus to carry corrective genes directly into the striatum region of the brain that controls movement.

It is designed to convert ordinary nerve cells into factories for making dopamine, the signalling chemical that is lost in Parkinsons patients.

Lack of dopamine activity leads to the common Parkinsons symptoms of tremor, slow movement and rigidity.

The trial tested the safety, tolerability and effectiveness of three different doses of ProSavin in 15 patients aged 48 to 65 with advanced Parkinsons disease who were not responding to conventional treatments.

A standard system of rating motor functions was used, covering speech, tremors, rigidity, finger taps, posture, gait, and slow movement. Lower scores indicated better muscle control and co-ordination.

Significant score improvements were seen after six months and a year in all patients not taking medication.

Reporting their findings in The Lancet medical journal, the researchers led by Professor Stephane Palfi, from Les Hopitaux Universitaires Henri-Mondor in Creteil, France, wrote: ProSavin was safe and well tolerated in patients with advanced Parkinsons disease. Improvement in motor behaviour was observed in all patients.

They stressed that, while promising, the results at this stage were still limited and should be interpreted with caution.

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Gene therapy provides hope for Parkinson's sufferers

The ProSavin treatment uses an inert virus to carry corrective genes directly into the striatum region of the brain that controls movement.

It is designed to convert ordinary nerve cells into factories for making dopamine, the signalling chemical that is lost in Parkinson's patients.

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Lack of dopamine activity leads to the common Parkinson's symptoms of tremor, slow movement and rigidity.

The trial tested the safety, tolerability and effectiveness of three different doses of ProSavin in 15 patients aged 48 to 65 with advanced Parkinson's disease who were not responding to conventional treatments.

A standard system of rating motor functions was used, covering speech, tremors, rigidity, finger taps, posture, gait, and slow movement. Lower scores indicated better muscle control and co-ordination.

Significant score improvements were seen after six months and a year in all patients not taking medication.

Reporting their findings in The Lancet medical journal, the researchers led by Professor Stephane Palfi, from Les Hopitaux Universitaires Henri-Mondor in Creteil, France, wrote: "ProSavin was safe and well tolerated in patients with advanced Parkinson's disease. Improvement in motor behaviour was observed in all patients."

They stressed that, while promising, the results at this stage were still limited and should be "interpreted with caution".

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Gene therapy for Parkinson's produces promising results in first patient trial

PARIS: A closely-watched prototype therapy to inject corrective genes into the brain to treat Parkinson's disease has cleared an important safety hurdle, doctors said Friday.

Tested on 15 volunteers with an advanced form of the degenerative nerve disease, the technique proved safe and the results were encouraging, they said.

The experiment aims to reverse the lack of a brain chemical called dopamine, which is essential for motor skills.

It entails tucking three genes into a disabled horse virus of the family lentiviruses.

The modified virus is then injected directly into a specialised area of the brain, where it infiltrates cells. In doing so, it delivers corrective pieces of DNA, prompting defective brain cells to once again start producing dopamine.

Called ProSavin, the British-designed treatment was authorised for tests on humans after it was tried on lab monkeys.

It is being closely watched by specialists to see if it works better than conventional therapies -- the veteran drug levodopa or electrical stimulation of the brain -- or another experimental gene technique which uses a modified cold virus.

French neurosurgeon Stephane Palfi, who led the early-stage trial published in The Lancet, said 15 patients aged 48-65 were given the genes in one of three doses.

They developed better coordination and balance, had less muscle twitching and improved speech.

Assessed at least 12 months after the injection, "motor symptoms remained improved in all the patients," Palfi said.

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Novel gene therapy for Parkinson's clears hurdle

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Newswise Imagine you are building a house. You would need a team of specialists, including an architect, a general contractor, carpenters, an electrician, a plumber and many others. Now picture yourself leading an effort to develop a new therapeutic drug or device. For that, youd need a very different kind of specialized team.

The National Eye Institute (NEI), part of the National Institutes of Health, has a research program designed to support this team-based approach. NEIs Translational Research Program (TRP) on Therapy for Visual Disorders provides a lead investigator with up to $1.75 million per year for up to five years in order to assemble a multidisciplinary team, and moveor translatepotential new therapies beyond the research lab and into clinical trials.

In addition to bringing together an expert research team, investigators can use funding from the TRP to recruit experts on navigating the drug and device approval process overseen by the Food and Drug Administration (FDA). They can also use TRP funds to seek help in patenting new therapies. These steps are no less essential than lab work for bringing new therapies to patients.

The program enables investigators to assemble multidisciplinary teams that can tackle scientific, technical, and regulatory issues that are beyond the capabilities of any single research group, said Neeraj Agarwal, Ph.D., who oversees programs in research training and workforce development at NEI. The TRP began in 2000, and has funded one or two projects each year since.

Eyeing new drugs for retinal diseases

Krzysztof Palczewski, Ph.D., professor and chair of the pharmacology department at Case Western Reserve University in Cleveland, received a grant (EY021126) through the TRP in 2010. His goal is to develop new drugs for diseases that damage the retina, the light-sensitive tissue at the back of the eye.

Vision begins with cells inside the retina called photoreceptors. Chemicals called retinoids, which are derived from vitamin A, play a key role inside these cells. One type of retinoid, when combined with a protein called opsin, acts as a light-sensitive switch, converting light into electrical signals that are ultimately sent from the photoreceptors to the brain.

Unfortunately, the supply of retinoids is limited and they need to be recycled. Moreover, the recycling process isnt 100 percent efficient. Dr. Palczewski has found that it can lead to the formation of a toxic byproduct called all-trans-retinal (atRAL), which may contribute to some diseases of the retina, such as age-related macular degeneration (AMD) and Stargardt disease. AMD is a leading cause of vision loss among people age 50 and older. Stargardt disease is a rare genetic disease that begins in childhood but has some similarities to AMD.

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Translational Research Through Teamwork

New York, NY (PRWEB) January 08, 2014

A stem cell model of familial Alzheimers disease (FAD) was successfully generated, allowing researchers to identify 14 genes potentially implicated in the disease. One gene in particular demonstrates the important role inflammation may play in the brain of Alzheimers patients. The study was completed by scientists at The New York Stem Cell Foundation (NYSCF) Research Institute in collaboration with scientists at the Icahn School of Medicine at Mount Sinai (ISMMS) and funded in part by the Cure Alzheimers Fund(CAF).

In the study published today in PLOS ONE, a team of scientists produced stem cells and neural precursor cells (NPCs), representing early neural progenitor cells that build the brain from patients with severe early-onset AD with mutations in the Presenilin 1 (PSEN1) gene. These NPCs had elevated Abeta42/Abeta40 ratios, indicating elevation of the form of amyloid found in the brains of Alzheimers patients. These levels were greater than those in adult cells that did not have the PSEN1 mutation. This elevated ratio shows that the NPCs grown in the petri dish accurately reflected the cells in the brains of FAD patients.

"The gene expression profile from the familial Alzheimers stem cells points to inflammation, which is especially exciting because we would not usually associate inflammation with this particular Alzheimer's gene," said Sam Gandy, MD, PhD, Professor of Neurology and Psychiatry and Director of the Center for Cognitive Health at the Icahn School of Medicine at Mount Sinai and a co-author on the study. Gandy is also Associate Director of the NIH-Designated Mount Sinai Alzheimers Disease Research Center and leader of the Cure Alzheimers Fund Stem Cell Consortium.

"This is the kind of innovative science that will help us better understand the cause of Alzheimers and how to approach the disease with effective therapies," said Tim Armour, President and CEO of Cure Alzheimers Fund (CAF). "It also showcases how targeted investment of critical resources can make a difference in finding solutions to this debilitating disease."

The researchers generated induced pluripotent stem (iPS) cells from affected and unaffected individuals from two families carrying PSEN1 mutations. After thorough characterization of the NPCs through gene expression profiling and other methods, they identified 14 genes that behaved differently in PSEN1 NPCs relative to NPCs from individuals without the mutation. Five of these targets also showed differential expression in late onset Alzheimers disease patients brains. Therefore, in the PSEN1 iPS cell model, the researchers reconstituted an essential feature in the molecular development of familial Alzheimers disease.

The studys co-lead authors Sam Gandy, MD, PhD and Scott Noggle, PhD are both members of CAFs Stem Cell Consortium, which supported this research. The Stem Cell Consortium is an international group of scientists collaborating on innovative research that investigates, for the first time, the brain cells from individuals with the common form of Alzheimers disease. Other members of the Consortium include Kevin Eggan, PhD, of Harvard University, Marc Tessier-Lavigne, PhD, of Rockefeller University, Doo Kim, PhD, of Harvard Medical School, and Tamir Ben-Hur, MD, PhD, of Hadassah University.

Stem cells are the least mature cells in the body. This means they can be treated with a defined cocktail of factors that can cause maturation of cells along discrete cell types. With iPS cells, which are cells that can become any cell type in the body, it now is possible to take skin cells from adults and return them to an immature state. By redirecting skin cells from Alzheimers patients and turning them into nerve cells, investigators are able to study adult Alzheimers neurons (nerve cells) in the lab.

Although the majority of Alzheimers disease cases are late onset and likely result from a mixture of genetic predisposition and environmental factors, there are genetic forms of the disease that affect patients at much earlier ages. PSEN1 mutations cause the most common form of inherited familial Alzheimers disease and are one hundred percent penetrant, resulting in all individuals with this mutation getting the disease.

Identifying genes that behaved differently in patients with the mutation provides new targets to further study and better understand their effects on the development of Alzheimers disease. One of these genes, NLRP2, is traditionally thought of as an inflammatory gene.

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Breakthrough Research Provides Valuable Insight On Cause Of Alzheimer’s

PUBLIC RELEASE DATE:

8-Jan-2014

Contact: Jim Newman jnewman@mdanderson.org 713-792-0662 University of Texas M. D. Anderson Cancer Center

HOUSTON, TX - Some surprising research findings from scientists at The University of Texas MD Anderson Cancer Center suggest it's possible a simple blood test could be developed to determine whether gene mutations associated with pancreatic cancer exist without the need of locating and testing tumor tissue. This appears possible following the discovery that tiny particles the size of viruses called 'exosomes,' which are shed by cancer cells into the blood, contain the entire genetic blueprint of cancer cells. By decoding this genomic data and looking for deletions and mutations associated with cancer, the research team believes this discovery could be translated into a test that helps physicians detect cancer and treat patients. The findings are based on research led by Raghu Kalluri, M.D., Ph.D., chairman and professor in MD Anderson's Department of Cancer Biology. The research results appear in the current online edition of the Journal of Biological Chemistry.

"At the present time, there is no single blood test that can screen for all cancer related DNA defects," said Kalluri. "In many cases, current protocols require a tumor sample to determine whether gene mutations and deletions exist and therefore determine whether the tumor itself is cancerous or benign. To procure tumor tissue, one needs to know that a tumor exists and if so, is it accessible for sample collection or removal? Finally, there are always risks and significant costs associated with surgical procedures to acquire tumor tissue."

Historically, researchers were aware these miniscule particles existed and that they carried nucleic acids and proteins. It was also believed that exosomes carried small portions of the person's DNA. However, upon further investigation, the MD Anderson research team was surprised to learn that the person's entire double-stranded genomic DNA spanning all chromosomes can be found in exosomes, including those mutated chromosomes that cause various cancers. Furthermore, Kalluri and colleagues discovered that DNA derived from exosomes carried the same cancer-related genetic mutations compared to the cancer cells taken from tumor.

"Because different forms of cancer are associated with different chromosomal mutations , we believe analysis of exosome DNA taken from blood samples may not only help determine the presence of a cancerous tumor somewhere in the body but also identify mutations without a need for tumor sample," added Kalluri. "We also believe this "fingerprint" will help lead us to the likely site of the tumor in the body. For instance, certain mutation spectrums would suggest pancreatic cancer or a brain-based tumor. While there is much more work to be conducted to develop such a test, having a tool such as this would increase our abilities to detect cancer in an earlier stage and therefore increase our chances of effective treatment."

"This seminal discovery paves the way for highly sensitive screening for driver mutations of cancer in the blood without the need for biopsy of tumor tissue and importantly, lays the foundation for a new method for the early detection of cancer when the chance for cure is greatest," said MD Anderson President Ronald A. DePinho, M.D.

###

The National Institutes of Health, Cancer Prevention and Research Institute of Texas and MD Anderson all provided funding to support this research.

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Research suggests a blood test to locate gene defects associated with cancer may not be far off

Jan. 8, 2014 Currently, doctors use chromosome markers and gene mutations to determine the best treatment for a patient with acute myeloid leukemia (AML). But a new study suggests that a score based on seven mutated genes and the epigenetic changes that the researchers discovered were present might help guide treatment by identifying novel subsets of patients.

The findings, published in the Journal of Clinical Oncology, come from a study led by researchers at The Ohio State University Comprehensive Cancer Center -- Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC -- James).

The epigenetic change used in the study is DNA methylation. It involves the addition of methyl groups to DNA, which can reduce or silence a gene's activity, or expression. Abnormal DNA methylation alters normal gene expression and often plays an important role in cancer development.

Overall, the findings suggest that patients with a low score -- indicating that one or none of the seven genes is overexpressed in AML cells -- had the best outcomes, and that patients with high scores -- that is, with six or seven genes highly expressed -- had the poorest outcomes.

"To date, disease classification and prognostication for AML patients have been based largely on chromosomal and genetic markers," says principal investigator Clara D. Bloomfield, MD, Distinguished University Professor, Ohio State University Cancer Scholar and Senior Adviser.

"Epigenetic changes that affect gene expression have not been considered. Here we show that epigenetic changes in previously recognized and prognostically important mutated genes can identify novel patient subgroups, which might better help guide therapy," says Bloomfield, who is also the William Greenville Pace III Endowed Chair in Cancer Research at Ohio State.

The seven-gene panel was identified in 134 patients aged 60 and older with cytogenetically normal acute myeloid leukemia (CN-AML) who had been treated on Cancer and Leukemia Group B (CALGB)/Alliance clinical trials.

The researchers computed a score based on the number of genes in the panel that were highly expressed in patients' AML cells, and retrospectively tested the score in two groups of older patients (age 60 and up) and two groups of younger patients (age 59 and under).

Patients with a low score -- indicating that one or none of the seven genes is overexpressed -- had the best outcomes. Patients with high scores -- that is, with six or seven genes highly expressed -- had the poorest outcomes.

"For this seven-gene panel, the fewer highly expressed genes, the better the outcome," says first author Guido Marcucci, MD, professor of medicine and the associate director for translational research at the OSUCCC -- James. "In both younger and older patients, those who had no highly expressed genes, or had one highly expressed gene had the best outcomes."

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AML score combining genetic, epigenetic changes might help guide therapy

PUBLIC RELEASE DATE:

8-Jan-2014

Contact: Darrell E. Ward Darrell.Ward@osumc.edu 614-293-3737 Ohio State University Wexner Medical Center

COLUMBUS, Ohio Currently, doctors use chromosome markers and gene mutations to determine the best treatment for patients with acute myeloid leukemia (AML). But a new study suggests that a score based on seven mutated genes and the epigenetic changes that the researchers discovered were also present might help guide treatment by identifying novel subsets of patients.

The findings, published in the Journal of Clinical Oncology, come from a study led by researchers at The Ohio State University Comprehensive Cancer Center Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC James).

The epigenetic change used in the study is DNA methylation. It involves the addition of methyl groups to DNA, which can reduce or silence a gene's activity, or expression. Abnormal DNA methylation alters normal gene expression and often plays an important role in cancer development.

Overall, the findings suggest that patients with a low score indicating that one or none of the seven genes is overexpressed in AML cells had the best outcomes, and that patients with high scores that is, with six or seven genes highly expressed had the poorest outcomes.

"To date, disease classification and prognostication for AML patients have been based largely on chromosomal and genetic markers," says principal investigator Clara D. Bloomfield, MD, Distinguished University Professor, Ohio State University Cancer Scholar and Senior Adviser.

"Epigenetic changes that affect gene expression have not been considered. Here we show that epigenetic changes in previously recognized and prognostically important mutated genes can identify novel patient subgroups, which might better help guide therapy," says Bloomfield, who is also the William Greenville Pace III Endowed Chair in Cancer Research at Ohio State.

The seven-gene panel was identified in 134 patients aged 60 and older with cytogenetically normal acute myeloid leukemia (CN-AML) who had been treated on Cancer and Leukemia Group B (CALGB)/Alliance clinical trials.

Here is the original post:
AML score that combines genetic and epigenetic changes might help guide therapy

Jan. 8, 2014 Some surprising research findings from scientists at The University of Texas MD Anderson Cancer Center suggest it's possible a simple blood test could be developed to determine whether gene mutations associated with pancreatic cancer exist without the need of locating and testing tumor tissue. This appears possible following the discovery that tiny particles the size of viruses called 'exosomes,' which are shed by cancer cells into the blood, contain the entire genetic blueprint of cancer cells. By decoding this genomic data and looking for deletions and mutations associated with cancer, the research team believes this discovery could be translated into a test that helps physicians detect cancer and treat patients.

The findings are based on research led by Raghu Kalluri, M.D., Ph.D., chairman and professor in MD Anderson's Department of Cancer Biology. The research results appear in the current online edition of the Journal of Biological Chemistry.

"At the present time, there is no single blood test that can screen for all cancer related DNA defects," said Kalluri. "In many cases, current protocols require a tumor sample to determine whether gene mutations and deletions exist and therefore determine whether the tumor itself is cancerous or benign. To procure tumor tissue, one needs to know that a tumor exists and if so, is it accessible for sample collection or removal? Finally, there are always risks and significant costs associated with surgical procedures to acquire tumor tissue."

Historically, researchers were aware these miniscule particles existed and that they carried nucleic acids and proteins. It was also believed that exosomes carried small portions of the person's DNA. However, upon further investigation, the MD Anderson research team was surprised to learn that the person's entire double-stranded genomic DNA spanning all chromosomes can be found in exosomes, including those mutated chromosomes that cause various cancers. Furthermore, Kalluri and colleagues discovered that DNA derived from exosomes carried the same cancer-related genetic mutations compared to the cancer cells taken from tumor.

"Because different forms of cancer are associated with different chromosomal mutations , we believe analysis of exosome DNA taken from blood samples may not only help determine the presence of a cancerous tumor somewhere in the body but also identify mutations without a need for tumor sample," added Kalluri. "We also believe this "fingerprint" will help lead us to the likely site of the tumor in the body. For instance, certain mutation spectrums would suggest pancreatic cancer or a brain-based tumor. While there is much more work to be conducted to develop such a test, having a tool such as this would increase our abilities to detect cancer in an earlier stage and therefore increase our chances of effective treatment."

"This seminal discovery paves the way for highly sensitive screening for driver mutations of cancer in the blood without the need for biopsy of tumor tissue and importantly, lays the foundation for a new method for the early detection of cancer when the chance for cure is greatest," said MD Anderson President Ronald A. DePinho, M.D.

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Blood test to locate gene defects associated with cancer may not be far off

PUBLIC RELEASE DATE:

8-Jan-2014

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, January 7, 2014Packaging replacement genes in viruses is an effective method to deliver them to target tissues, but the human body mounts an immune response against the virus. The systemic and local immune reactions induced by an adeno-associated virus (AAV)-based gene therapy to treat lipoprotein lipase deficiency, approved for use in Europe, does not affect the safety of gene therapy or expression of the replacement gene for at least one year after delivery, according to a study published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available on the Human Gene Therapy website.

Valeria Ferreira and coauthors, uniQure BV and Academic Medical Center, Amsterdam, the Netherlands, and University of Montreal and Chicoutimi Hospital, Quebec, Canada, evaluated measures of inflammation and adverse clinical events and the expression of a replacement lipoprotein lipase (LPL) gene that was injected intramuscularly into patients with LPL deficiency. The gene was packaged in an AAV vector, as described in the article "Immune responses to intramuscular administration of alipogene tiparvovec (AAV1-LPLS447X) in a phase II clinical trial of Lipoprotein Lipase deficiency (LPLD) gene therapy."

"The clinical data published in this paper were critical to the approval of Glybera," says James Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia. "Furthermore, they provide context for laboratory measurements of immune responses which apparently did not impact product performance."

###

About the Journal

Human Gene Therapy, the official journal of the European Society of Gene and Cell Therapy, British Society for Gene and Cell Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies, is an authoritative peer-reviewed journal published monthly in print and online. Human Gene Therapy presents reports on the transfer and expression of genes in mammals, including humans. Related topics include improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, as well as ethical, legal, and regulatory issues related to the gene transfer in humans. Its sister journals are Human Gene Therapy Methods, published bimonthly and focused on the application of gene therapy to product testing and development, and Human Gene Therapy Clinical Development, published quarterly and featuring data relevant to the regulatory review and commercial development of cell and gene therapy products. Tables of content for all three publications and a sample issue may be viewed on the Human Gene Therapy website.

About the Publisher

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Does the body's immune response to viral vector delivery systems affect the safety or efficacy of gene therapy?

I have frequently pointed out that pharmaceutical companies acknowledge that animal models are not predictive for human response in terms of efficacy or toxicity. More evidence for this position comes from Robert G. Hunter in an article in Genetic Engineering & Biotechnology News.[1] Hunter: Having developed over the past 20 years into a global market recently estimated at $5 billion, in vitro and in silico products and services are now about the same size as the in vivo services (contract research organization) industry. If animal models worked well, there would be no need for industry to look at other options. Pharma does not love bunnies. Pharma loves money.

Matthew Herper addressed the problems in drug development in an article in Forbes.[2] Herper:

Theres one factor that, as much as anything else, determines how many medicines are invented, what diseases they treat, and, to an extent, what price patients must pay for them: the cost of inventing and developing a new drug, a cost driven by the uncomfortable fact than 95% of the experimental medicines that are studied in humans fail to be both effective and safe.

Animal models are relied on for the evaluation of both efficacy and safety.[3-9] Herper continues:

A new analysis conducted at Forbes puts grim numbers on these costs. A company hoping to get a single drug to market can expect to have spent $350 million before the medicine is available for sale. In part because so many drugs fail, large pharmaceutical companies that are working on dozens of drug projects at once spend $5 billion per new medicine. . . . This is crazy. For sure its not sustainable, says Susan Desmond-Hellmann, the chancellor at UCSF and former head of development at industry legend Genentech, where she led the testing of cancer drugs like Herceptin and Avastin. Increasingly, while no one knows quite what to do instead, any businessperson would look at this and say, You cant make a business off this. This is not a good investment. I say that knowing that this has been the engine of wonderful things.

This, in part, is why disease-specific drugs like Kalydeco, a drug for cystic fibrosis (CF) patients that have a specific genetic mutation, costs $294,000 per patient per year.

The reason animal models fail for drug development is that animals and humans are evolved systems that are differently complex. While morphological similarities exist, very small differences in the genetic make-up between species and between individuals of the same species means the predictive value for extrapolation is nil in the real world. (For more on this see Trans-Species Modeling Theory.) Moreover, if the concept of evolved, complex systems invalidates trans-species extrapolation in drug development, it is going to do the same when trans-species extrapolation involves any perturbation that affects higher levels of organization. So just based on the evidence from drug development we can safely say that disease research on mice, monkeys, or dogs is not going to result in knowledge that has predictive value for human patients. The literature confirms this.[10-21][[22]p19-33, 73-77] [23-25]

Compare the above to this recent statement from Michael E. Goldberg published in the Wisconsin State Journal: Nearly every medical advance from the last century is a product of responsible animal research, and animal models will continue to be important to medical progress. . . . Activists who claim animal research does not benefit humans are wrong. Animals are essential to medical progress in all fields of human disease. [26] This illustrates the dichotomy regarding animal models. Dr Goldberg is an animal modeler who does basic research, which he sells as applied research. Not surprisingly, Goldberg thinks animal modeling is great. He does not suffer loss of income or prestige when the knowledge from animal modeling fails to translate to human patients.

Pharma on the other hand, can actually measure the success or lack thereof of animal models in the form of drugs successfully brought to market and Pharma says it doesnt work. Remember, Pharma is a business and they do not care how they develop new drugs they just want to develop new drugs so they can make money. Also remember that there are not two different theories of evolution: one for drug development and another for basic science research or basic research masquerading as applied research. If animal modeling in drug development fails to be consistent with evolutionary biology, then it fails in general as well.

Image courtesy of Wkipedia Common http://en.wikipedia.org/wiki/File:Chromosomes_mutations-en.svg

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There is Only One Evolution

Health

Linda Carroll NBC News contributor

19 hours ago

A rare genetic mutation that disrupts the production of histamine may help researchers unravel the mystery that surrounds Tourette syndrome.

The mutation discovered by Yale researchers can cause the kinds of tics and other abnormalities that are the hallmark of the syndrome, according to a study published Wednesday in the journal Neuron.

Thus far the genetic anomaly has been discovered only in nine members of a single family: a father and all eight of his children who have both the mutation and Tourette syndrome.

We know that Tourette is about 90 percent genetic, said study coauthor Dr. Christopher Pittenger, an associate professor of psychiatry and psychology at the Yale University School of Medicine and director of the Yale OCD research clinic. But its been incredibly hard to find any genetic abnormalities that cause the syndrome. We have proven that this gene really is the cause of Tourette in this family and also looked at some of its downstream effects.

Courtesy Jeffrey Kramer

Jeffrey Kramer and his three sons. Kramer and two of his grown-up sons have been living with Tourette for decades. Hes excited by the new findings, but realistic about their impact on patients with the syndrome.

What isnt known yet is how, or if, this finding can be extended to other people with Tourette, Pittenger and other experts said.

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Rare genetic mutation discovered in Tourette syndrome family

(MENAFN - Muscat Daily) A Weill Cornell Medical College study that analysed the DNA of Qatar's native population has discovered genetic variations that could help doctors target interventions to reduce the prevalence of a variety of debilitating hereditary disorders

Researchers at Weill Cornell Medical College in Qatar (WCMC-Q) and Weill Cornell Medical College in New York (WCMC-NY), working with colleagues from Cornell University in Ithaca and Hamad Medical Corporation, identified 37 genetic variants in 33 genes known to play causal roles in a total of 36 diseases, including such devastating conditions as cystic fibrosis, sickle cell anemia and muscular dystrophy. The study points the way to more comprehensive screening for a host of inherited diseases, which could significantly reduce their incidence.

The project titled, 'Exome Sequencing Identifies Potential Risks Variants for Mendelian Disorders at High Prevalence in Qatar' sequenced the DNA of 100 Qatari nationals representing the three major ethnic subgroups of the country the Bedouin (termed Q1 for the purposes of the study), those of Persian-South Asian descent (Q2), and those of African descent (Q3). By analysing the individuals' exomes important sections of the DNA containing the code that is translated into proteins and comparing them to the genetic data of the participants in the worldwide 1,000 Genomes Project (1000G), the researchers were able to identify the variations that cause disease among the Qatari population.

All the conditions targeted in the study were so-called Mendelian diseases'. Named after Gregor Mendel, the 19th century researcher widely regarded as the founder of genetic science, Mendelian diseases are those caused by a single mutated gene and are also known as monogenic disorders.

Dr Khalid Fakhro, postdoctoral associate in genetic medicine at WCMC-Q, and Dr Juan L Rodriguez-Flores of WCMC-NY, were co-lead principal investigators in the study, which is part of a group of research projects investigating the Qatari genome led by Dr Ronald Crystal, chairman of Genetic Medicine at Weill Cornell Medical College in New York. The study has been accepted for publication in the journal Human Mutation, appearing online in December 2013 and in print in January 2014.

Dr Crystal explained the study: ''There are about 3.2bn letters that comprise the human genome and about two percent of those letters code for the actual proteins. This two per cent is found in regions called exomes,'' he said. ''A Mendelian or monogenic disease is caused by a change in a single letter out of the 3.2bn.

''The reason this is relevant for Qatar is that the structure of the society encourages a high degree of consanguineous marriage, so the frequency of these monogenic diseases is quite high,'' he said.

Pre-marital counseling and screening is one method of decreasing the likelihood of children being born with monogenic diseases. Parents undergo screening to see if either or both carry genetic variations that cause disease before having children. The individuals that carry the disorder do not necessarily have the conditions themselves, but may carry them on recessive genes.

Dr Crystal added, ''Disorders are present in all populations around the world, so Qatar is no different. Qatar is only different in that its variations and the frequency with which they occur are unique to its population. By finding out what these variations are and taking appropriate action we can save people from the trauma of some very unpleasant disorders. We're talking here about things like brain malformation, diabetes, blindness, deafness, cardiovascular disorders, inflammatory disorders and many other conditions. While these conditions are not common, they do occur, some are untreatable and many are very difficult to live with, for both the sufferer and their families.''

Currently, pre-marital counseling in Qatar screens for four genetic variations out of the 37 identified by the study, so incorporating the newly discovered variations into the screening process could have a significant impact.

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Mapping the Qatari shows way to prevention of inherited diseases

Dragon Genetics Game
Simple arcade game which focuses strictly on dragon genetics. It #39;s pretty simple and there isnt that much variation but interesting. Royalty Free Music by: h...

By: IamJaylew

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Dragon Genetics Game - Video

Originally published January 8, 2014 at 11:38 AM | Page modified January 8, 2014 at 6:13 PM

NEW YORK Seattle Genetics said Wednesday it expanded a cancer treatment collaboration with drugmaker AbbVie.

The Bothell company said it will receive a $25 million upfront payment from AbbVie, and it could get $255 million in license and milestones payment per drug. AbbVie also agreed to pay Seattle Genetics royalties in the single digits on sales of any drugs that are approved.

Shares of Seattle Genetics rose $1.04, or 2.6 percent, to $41.04 Wednesday.

Seattle Genetics and Abbott Laboratories started working together in March 2011 on experimental antibodies designed to fight cancer. The antibodies are designed to selectively deliver anticancer drugs to tumor cells. Abbott made an $8 million upfront payment. In October 2012 the companies agreed to expand their partnership, and Abbott paid Seattle Genetics an additional 25 million.

AbbVie was spun off from Abbott a year ago. It sells branded prescription drugs, including the blockbuster anti-inflammatory drug Humira, which is used to treat rheumatoid arthritis, psoriasis and Crohns disease.

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Seattle Genetics, AbbVie broaden partnership

By Cassandra Leger

Cancers are not created equal nor do they respond the same to treatment and scientists have sought after the reason why for decades. In particular, research has focused on uncovering why certain cancers are very rebellious and stubborn in response to radiation therapy. The answer may lie in studying the mitochondriathe energy power plant of the cell.

A research team lead by Maxim Frolov at the University of Illinois at Chicago has shed light on the previously unknown role that mitochondria play in cell death induced by radiation therapy, as described in a November press release.The team studied the E2F gene, a key player in the natural process of cell death. Fruit flies were fitted with a mutated version of the E2F gene which caused the cells mitochondria to be misshapen. When misshapen, mitochondria produce less energy. Importantly, the fruit flies with abnormal mitochondria were resistant to radiation therapy. The study was later introduced to human cells and yielded similar results.

This discovery may help explain why cancer patients react so differently to radiation therapy and may be a building block for pharmaceutical companies to develop drugs which target mitochondrial function to improve radiation therapy outcomes.

Other research teams at the University are focused on understanding the role of mitochondrial dysfunction in cancer, including Michelle Boland and colleagues. A recently published review in the journal Frontiers in Oncology covers research on the role of dysfunctional mitochondria in multiple aspects of cell growth and the relation to cancer diagnosis and treatment.

Source: http://news.uic.edu/dysfunctional-mitochondria-may-underlie-resistance-to-radiation-therapy

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Abnormal Mitochondria May Contribute To Radiation Resistant Cancer

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7-Jan-2014

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, January 7, 2014African American men have an increased risk of prostate cancer and are two times more likely than Caucasian American men to die from the disease. Despite recent questions about the overall usefulness of prostate-specific antigen (PSA) testing to detect prostate cancer, should PSA screening be used to detect early-stage disease to help save lives in this at-risk population? The controversy is explored in a Review article in Journal of Men's Health, a peer-reviewed publication from Mary Ann Liebert, Inc., publishers. The article is available free on the Journal of Men's Health website at http://www.liebertpub.com/jomh.

In the Review "PSA Screening for the African American Male: When and Why?" Tyler Luthringer, Ilija Aleksic, Vladimir Mouraviev, and David Albala, Associated Medical Professionals of NY, PLLC, and SUNY Upstate Medical University, Syracuse, support the American Urological Association's position that early detection of prostate cancer should include multiple parameters to assess personal risk. Together with their physicians, men should decide on an individualized approach to risk assessment and screening, which may include PSA testing and digital rectal examination.

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Increased risk of prostate cancer in African American men; implications for PSA screening

Carp species are found to live successfully in the Great Lakes, but at what price?

Blocking Asian carp from invading the Great Lakes could require an engineering marvel that rivals the reversal of the Chicago River more than a century ago, according to a new federal study that promises to reignite a fierce debate about the region's waterways.

Among the options outlined Monday by the Army Corps of Engineers to thwart the voracious fish and other invasive species from spreading is permanently separating Lake Michigan from the river and its connected waterways. Such a project would restore the once natural divide between the Great Lakes and rivers southwest of Chicago that drain into the Mississippi River.

Chicago blasted through that hydrological barrier when it dug the Sanitary and Ship Canal and Cal-Sag Channel at the turn of the last century to divert the region's sewage away from its source of drinking water. It also created a shipping link between two of the nation's major trade routes.

Separating the lake and the river again could cost more than $18 billion and take up to 25 years, the Corps' study concluded, making that option the most expensive of the eight studied. Another option, carrying a $15 billion price tag, would allow portions of the Chicago River, Sanitary and Ship Canal and Calumet River to flow into Lake Michigan.

Reflecting the development of Chicago from a swampy prairie outpost to a sprawling city, both of those options would require a multibillion investment in giant stormwater tunnels and massive flood-control reservoirs, according to the study.

Several political leaders said the potential cost and time frame could scuttle the plan before Congress considers it.

"I've seen too many of these long-term Corps projects languish for years and fall victim to congressional inaction," said U.S. Sen. Dick Durbin, the Illinois Democrat and assistant Senate majority leader. "We can't gamble with the threat of Asian carp ... or risk severe flood damage to the Chicago metropolitan area by pursuing a risky plan at the expense of our current efforts."

A lobbyist for one of the industries that still relies on the river system was even more blunt. The study "clearly indicates that physical separation is too expensive, too slow and too uncertain to be a viable solution to the spread of invasive species," said Mark Biel, executive director of the Chemical Industry Council of Illinois.

Two years in the making, the study comes amid a series of alarming findings that raise the possibility it might be too late for new federal action to stop Asian carp from getting into the Great Lakes and threatening the region's $7 billion fishing industry.

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Engineering marvel may be needed to stop Asian carp

FARGO Denny Sanford was recovering from possibly fatal blood clots in his lungs when he decided to invest $125 million to bring genetic medicine into the mainstream.

Sanford became ill on a hunting trip in south-central South Dakota in October, about 140 miles west of Sioux Falls.

Doctors there suspected he had pneumonia, but Sanfords personal physician, Dr. Eric Larson of Sanford Health, suspected a pulmonary embolism a blood clot in the lungs and arranged for an air ambulance to whisk him to Sioux Falls.

He really saved my life, Sanford said in a telephone interview with The Forum, referring to Larson, an internal medicine doctor and one of the champions of the new genetic medicine initiative Sanford Health announced Tuesday.

Sanford, who is in his late 70s, did not attend Tuesdays announcement, which was made in Sioux Falls, and simulcast to Sanford medical centers in Fargo, Bismarck and Bemidji, Minn.

While recuperating in his namesake hospital in Sioux Falls, Sanford reminded Kelby Krabbenhoft, Sanford Healths top executive, that his team was preparing a genetic medicine proposal.

He invited them to make their pitch two days later, when he was convalescing at home. Sanfords recent medical emergency made him receptive to the idea of placing results of genetic testing tools in the hands of primary care physicians.

It was an opportune time to lay it out on me, Sanford said, chuckling about the timing and his gratitude for the care he received.

I believe that is the medicine of the future, added Sanford, referring to the use of genetic information in tailoring health care. He recently donated $100 million to a stem cell research program in California.

Sanford, a St. Paul native who founded Premier Bank, now has donated more than $1 billion, much of it to Sanford Health, beginning with a $400 million gift in 2007.

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Denny Sanford believes genetic medicine is 'the medicine of the future'

FARGO Sanford Health is embarking on a major initiative to integrate genetic information with primary care enabled by a $125 million gift from namesake benefactor T. Denny Sanford.

The new effort, announced Tuesday, is billed by Sanford as the first of its kind in the nation to marry genetic screening with internal medicine doctors throughout its clinic system.

The so-called Sanford Imagenetics program will begin offering patients the opportunity for precise genetic testing and genetic counseling later this year.

This is the frontier of medicine, Sanfords top executive, Kelby Krabbenhoft, said in making the announcement. This is whats going to change everything for everybody.

Sanfords gift will allow the health system to hire and train teams of specialists in a collaboration that also will involve academic centers in the Sanford service area for research, and training physicians and other providers.

Each of Sanfords four regional hubs Sioux Falls, S.D., Fargo, Bismarck, and Bemidji, Minn. will have specialists who will work closely with internal medicine doctors.

Sanford already has seven genetic counselors, including two in Fargo, and five medical geneticists.

Through telemedicine and targeted outreach efforts, the program will be available to patients throughout Sanfords sprawling service area that includes North Dakota, South Dakota, Minnesota, Iowa and Montana.

A portion of Sanfords gift will be used for a new genetic medicine center at its campus near downtown Sioux Falls, with construction to begin in spring 2015.

We will have the same resources, said Dr. Julie Blehm, a senior internal medicine doctor at the Sanford Medical Center in Fargo. Were going to have the same opportunities.

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Sanford announces $125 million genetic medicine initiative

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Newswise (Sioux Falls, SD) Sanford Health announced today Denny Sanford, the preeminent health care philanthropist in the United States, will gift the organization $125 million to establish Sanford Imagenetics, a first-of-its-kind program in the country that integrates genomic medicine into primary care for adults.

Mr. Sanfords generosity to this organization is humbling, said Kelby Krabbenhoft, president and CEO of Sanford Health. Including this $125 million gift, Denny has given Sanford Health nearly a billion dollars. Its an incredible honor as well as a tremendous responsibility.

Internal medicine physicians assist adult patients by diagnosing and managing complex health issues. Later this year, Sanford Imagenetics will offer patients the opportunity to undergo precise genetic testing and genetic counseling which will provide internal medicine physicians with unprecedented patient-specific information. Arming these physicians with their patients genetic information will improve their ability to prescribe the right medication, appropriate dose or most effective treatment with drugs such as statins and blood thinners.

Most physicians can only dream of what it would be like to practice not only on the cutting edge of medical advancement but also work to fundamentally change how patients are treated. The creation of this environment does not occur spontaneously. It requires great leadership and generosity at a level not previously seen, said Eric Larson, MD, board-certified internal medicine physician with Sanford Health.

Sanford Health has a long-standing history of providing comprehensive genetic health care to the region. With Sanford Imagenetics, Sanfords MD geneticists, genetic counselors and diagnostic clinical genetics laboratories will move hand in hand with the organizations internal medicines physicians. There are currently no organizations in the country that similarly embed genetics health care professionals into these primary care practices.

Thanks to Mr. Sanfords continued generosity, Sanford Health will take a national lead role in using existing genetic markers and incorporating future discoveries for internists to individualize care for patients with cancer, diabetes, hypertension, coronary artery disease and other conditions, said Dan Blue, MD, president Sanford Clinic.

Sanford Imagenetics will include development of a rigorous research program to define the genomic markers most successful in managing primary care for adults.

We will also study the outcomes to evaluate the efficacy of this approach as well as in-depth bioinformatics research focused on the practical clinical interpretation of the complicated biological data, said Gene Hoyme, MD, president of Sanford Research and board certified geneticist.

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Sanford Health Announces $125M Gift to Fund Genomic Initiative for Internal Medicine

Christmas was two weeks ago but Sanford Health received quite the gift Tuesday morning. $125 Million, that's how much philanthropist Denny Sanford has pledged to help launch a brand new system of patient treatment.

A lot of people will probably just look at the $125 Million and be blown away by the amount, but what that money will do is far more amazing. With the gift, Sanford is launching Imagenetics to fighting disease on the molecular level. This is personalized medicine where your DNA helps determine the best course of treatment. This will help cut down on the number of medications, limit side-effects, and could even help doctors treat the condition before symptoms show up. Genetic medicine is already being utilized by Sanford doctors to treat cancers and other conditions, but this will take those practices to the clinic as well.

"This really gets to what we call precision medicine that is using the tools of genetics to precisely take care of you as an individual." Said Dr. H. Eugene Hoyme, a geneticist and president of Sanford Research.

"Once again he's taken what was possible and today has made it practical for everybody. Thank you Mr. Sanford." Said Kelby Krabbenhoft, president and CEO of Sanford Health.

The money will be spread throughout the Sanford Health system to build facilities for genetic research and testing. Those projects are expected to break ground starting in the spring of 2015.

In addition to the donation, Sanford announced partnerships with Augustana College, and the Universities of North Dakota and South Dakota to train the next wave of genomic health professionals. Krabbenhoft says this donation puts Mr. Sanford over the $1 Billion mark in charitable gifts. His largest donation came back in 2007, giving $400 million dollars to Sanford Health.

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Sanford donates $125M to launch genetic medicine program

Although we don't believe in timing the market or panicking over daily movements, we do like to keep an eye on market changes -- just in case they're material to our investing thesis.

How is Wall Street supposed to follow up a year like 2013? Boosted by low interest rates, last-minute congressional compromises, and surging real estate markets, stocks posted their highest gains since the 1990s. Unfortunately, the first economic data of 2014 saw four-week average jobless claims jump by 8,500 to more than 357,000, triggering some investors to cash in on last year's profits. After a series of all-time highs to close out December, the Dow Jones Industrial Average (DJINDICES: ^DJI) fell 135 points, or 0.8%, to end at 16,441.

McDonald's (NYSE: MCD) stock, although it fell 0.6% today, wasn't a terrible performer relative to its peers, as 28 of 30 blue chips ended in the red Thursday. The year 2014 should, however, be an interesting one for the international fast-food giant, as a fiercely competitive industry forces McDonald's to rethink its offerings, pricing, and efficiency. The Wall Street Journal called out CEO Don Thompson as one of six head honchos with a lot on the line this year, as slowing sales and so-so new product launches caused the stock to vastly underperform the red-hot stock market.

Elsewhere in the broad-encompassing services sector, Myriad Genetics (NASDAQ: MYGN) stood out as one of the top gainers, adding 3.5%, and resolving to hopefully never repeat 2013's performance. The stock is more than 20% lower than it was just a year ago, although it's worth noting that Myriad Genetics is down more than 10% in the last five days of trading alone. That's because it looks like Medicare won't be willing to dole out what it used to for Myriad's services; the proposed new rate for its BRACAnalysis gene test was actually cut in half. As you might imagine, this didn't go over well with investors.

Another big mover on the day, DryShips (NASDAQ: DRYS) stock, slumped 8.3% to begin 2014 on a very sour note. It was evident that the Greek shipper would get off to a poor start this year after the company announced -- after market close on the last day of the year -- that it would resume its $200 million equity offering. This is an odd turn of events, since just last month, DryShips mysteriously cancelled these same plans, only to reintroduce them weeks later, when the stock was trading at a much higher price.

Two game-changing biotechs revolutionizing the way we treat cancer The best way to play the biotech space is to find companies that shun the status quo and, instead, discover revolutionary, groundbreaking technologies. In the Motley Fool's brand-new FREE report, "2 Game-Changing Biotechs Revolutionizing the Way We Treat Cancer," you will find out about a new technology that big pharma is endorsing through partnerships, and the two companies that are set to profit from this emerging drug class. Click here to get your copy today.

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Myriad Genetics Rebounds, DryShips Craters on Surprise ...