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Newswise Philadelphia, Dec. 6, 2014 An innovative cell therapy against a highly aggressive form of acute lymphoblastic leukemia (ALL) continues to show highly promising results in children treated in a pilot study. Ninety-two percent of the 39 children receiving bioengineered T cells had no evidence of cancer at one month after treatment, with this complete response persisting in some cases for more than two years. The personalized cell therapy reprograms a patients immune system and offers the potential of long-term success.

As we continue to follow children in this study, we see exciting results for patients who have exhausted their other treatment options, said study leader Stephan A. Grupp, M.D., Ph.D., a pediatric oncologist at The Childrens Hospital of Philadelphia and a Professor of Pediatrics at the Perelman School of Medicine of the University of Pennsylvania. Grupp described outcomes and follow-up results of this pilot clinical trial for pediatric patients with ALL in a press program today at the annual meeting of the American Society of Hematology (ASH) in San Francisco.

Grupp, first author Shannon Maude, M.D., Ph.D., a pediatric oncologist at CHOP and Assistant Professor of Pediatrics at the Perelman School of Medicine of the University of Pennsylvania, and colleagues reported today that of the 39 children treated, 36 (92%) had complete responses one month after treatment. Of those 36 patients, 25 (69%) remained in remission at a median follow-up of 6 months after treatment. There were 10 relapses among the 36 patients with complete responses; 5 of the relapsed patients died.

Grupp is an ongoing collaborator with colleagues at Penn Medicine, led by Carl H. June, M.D., who offer this personalized cell therapy as a treatment for adult patients with other types of cancer.

ALL is the most common childhood cancer and the most common childhood leukemia. All the patients had high-risk ALL that recurred after initial treatment or resisted that treatment from the start. Patients received bioengineered hunter T cells called CTL019 cells.

The first child to undergo this therapy, 9-year-old Emily Whitehead, remains cancer-free since her T cell treatment in April 2012, and continues to enjoy normal childhood activities like going to school and playing with her dog, Lucy. Emily has appeared prominently in news stories since her doctors announced dramatic findings during the December 2012 ASH meeting.

A relatively new approach in cancer treatment, this type of immunotherapy modifies T cells, the workhorses of the bodys immune system, to attack B cells, other immune cells that become cancerous in specific leukemias such as ALL. Bioengineered T cells, called CTL019 cells, function as cancer hunters, killing the leukemia cells that normally evade regular T cell surveillance. Researchers first extract a patients own T cells. They then use bioengineering techniques to reprogram each patients T cells into chimeric antigen receptor cellsthe CTL019 cellscustom-designed to bind to a protein called CD19 that exists only on the surface of B cells. After being returned to the patients body, the CTL019 cells proliferate and then eliminate B cells. Moreover, they persist in the circulation, helping to guard against the cancers recurrence.

As the CTL019 cells potently attack leukemia cells, they also stimulate an unwanted, toxic immune response called cytokine release syndrome in patients. The care team successfully counteracted these side effects with an immunomodulating drug that had never been used for that purpose before, an approach which now has been adopted widely by cell therapy groups. In addition, because the CTL019 therapy eliminates healthy B cells along with cancerous B cells, patients must receive infusions of immunoglobin to perform the immune function provided by normal B cells.

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Benefits Persist in T Cell Therapy for Children with Relapsed Leukemia

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The Sims 4: Perfect Genetics Legacy [Part 5] Kaydence Moves In
With this newfound romance that Marshall and Kaydence have, they had become an official couple. With them becoming a couple there was only one thing that it ...

By: Connor K Games

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The Sims 4: Perfect Genetics Legacy [Part 5] Kaydence Moves In - Video

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InWin - Quarti: EasyFix vs Superior Genetics
30 Novembre 2014.

By: 5GGBranches

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Mendelian genetics, sweet pea examples

By: BiologyVittles

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Mendelian genetics, sweet pea examples - Video

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Genetics Policy Institute (GPI) and the Regenerative Medicine Foundation (RMF) Announce Merger Plan
The Genetics Policy Institute (GPI), producer of the annual World Stem Cell Summit, and the Regenerative Medicine Foundation (RMF) have agreed to merge in order to leverage their resources...

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Hog Blog Genetics Video #2
This video is about Snake Genetics. Dominant vs Recessive, Mitosis vs Meiosis. Simple Genetics.

By: Hog Blog!

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Hog Blog Genetics Video #2 - Video

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ALGA Questionnaire promo
Visit http://www.ecancer.org for more The Personalized Medicine project is a 4 year long collaborative effort aimed at developing innovative tools to create support and sustain new knowledge...

By: ecancer medicalscience

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ALGA Questionnaire promo - Video

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SAN DIEGO--(BUSINESS WIRE)--Cytori Therapeutics, Inc. (NASDAQ: CYTX) today confirmed that two Japanese regenerative medicine laws, which went into effect on November 25, 2014, remove regulatory uncertainties and provide a clear path for the Company to commercialize and market Cytori Cell Therapy and its Celution System under the Companys existing and planned regulatory approvals.

Japans new regenerative medicine laws substantially clarify regulatory ambiguities of pre-existing guidelines and this news represents a significant event for Cytori, said Dr. Marc Hedrick, President & CEO of Cytori. We have a decade of operating experience in Japan and Cytori is nicely positioned to see an impact both on existing commercial efforts and on our longer-term efforts to obtain therapeutic claims and reimbursement for our products.

Under the two new laws, Cytori believes its Celution System and autologous adipose-derived regenerative cells (ADRCs) can be provided by physicians under current Class I device regulations and used under the lowest risk category (Tier 3) for many procedures with only the approval by accredited regenerative medicine committees and local agencies of the Ministry of Health, Labour and Welfare (MHLW). This regulatory framework is expected to streamline the approval and regulatory process and increase clinical use of Cytori Cell Therapy and the Celution System over the former regulations.

Before these new laws were enacted, the regulatory pathway for clinical use of regenerative cell therapy was one-size-fits-all, irrespective of the risk posed by certain cell types and approaches, said Dr. Hedrick. Now, Cytoris point-of-care Celution System can be transparently integrated into clinical use by providers under our Class I device status and the streamlined approval process granted to cell therapies that pose the lowest risk. Our technology is unique in that respect.

Cytoris Celution System Is in Lowest of Three Risk Categories

The Act on the Safety of Regenerative Medicines and an amendment of the 2013 Pharmaceutical Affairs Act (the PMD Act), collectively termed the Regenerative Medicine Laws, replace the Human Stem Cell Guidelines. Under the new laws, the cell types used in cell therapy and regenerative medicine are classified based on risk. Cell therapies using cells derived from embryonic, induced pluripotent, cultured, genetically altered, animal and allogeneic cells are considered higher risk (Tiers 1 and 2) and will undergo an approval pathway with greater and more stringent oversight due to the presumed higher risk to patients. Cytoris Celution System, which uses the patients own cells at the point-of-care, will be considered in the lowest risk category (Tier 3) for most cases, and will be considered in Tier 2 if used as a non-homologous therapy.

Streamlined Regulatory Approval for Certain Medical Devices

In the near future, Cytori intends to pursue disease-specific or therapeutic claims and reimbursement for Cytoris Celution System and the Company would, at that point, sponsor a clinical trial to obtain Class III device-based approval and reimbursement. The new laws include changes to streamline regulation of Class II and some Class III devices, which will now require the approval of certification bodies rather than the PMDA, similar to the European notified body model. To date, certification bodies have only been used for some Class II devices.

Conditional Regulatory Approval and Reimbursement Potential

As a supplementary benefit to Cytori, the Company may also choose to take advantage of the new conditional approval opportunities granted under the new laws. Once clinical safety and an indication of efficacy are shown, sponsors may apply for their cell product to receive conditional approval for up to seven years and may be eligible for reimbursement under Japans national insurance coverage. Under the conditional approval, the sponsor can then generate post-marketing data to demonstrate further efficacy and cost effectiveness.

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Cytori Expects New Japan Laws to Boost Adoption of Cytori Cell Therapy

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Cordlife Medical Philippines Inc. (Cordlife), a fully owned subsidiary of Cordlife Group Ltd., a Singapore Exchange mainboard-listed consumer healthcare company catering to the mother and child segment, recently opened its newly upgraded stem cell processing and cryopreservation facility at UP-Ayalaland Technohub in Quezon City.

This expansion came less than five years from the opening of its facility in the country and was mainly driven by the growing number of parents who recognize the value of banking here their babys stem cells.

The event launch held at Crowne Plaza in Ortigas was hosted by popular DJ Delamar Arias and graced by celebrity moms, actress and beauty queen Lara Quigaman and journalist Nia Corpuz. Delamar, Lara and Nia all shared their personal experiences of being Cordlife moms themselves.

Also in attendance to underscore Cordlifes commitment to Filipino parents and their children were Cordlife Group CEO Jeremy Yee, Cordlife Philippines medical director Dr. Arvin Faundo and Cordlife Philippines director Michael Arnonobal.

The event highlighted the five essentials of mother and child that Cordlife addresses through services. These essentials are security, expertise, investment, bond and value for life.

With the newly upgraded stem cell facility that can accommodate up to 30,000 cord blood and cord lining units, more parents can enjoy a sense of security when they bank their babys stem cells at Cordlifes state-of-the-art processing and cryopreservation laboratory.

Peace of mind

Those who banked with Cordlife can have peace of mind knowing that their babys stem cells are kept in a highly-secure laboratory that was built to withstand earthquakes of up to magnitude 8, and ward off fire with its fire-retardant walls. Furthermore, the vapor-phased liquid nitrogen cryogenic tanks used to preserve the cord blood and cord lining samples are not dependent on electricity, making them 100-percent safe and reliable in case of power outages.

Cordlifes expertise in the stem cell banking arena, is proven not only by being the largest network of cord blood banks in Asia, but also the number of cord blood releases for stem cell transplant and their adherence to the stringent global standards of AABB (formerly known as American Association of Blood Banks).

Cordlife Philippines is also the countrys first and only ISO 9001:2008 and Department of Health-registered cord blood and cord lining bank.

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Cordlife launches newly upgraded stem cell facility

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Robert Vondracek has had multiple sclerosis for 20 years. His speech is starting to slur and he's been having more trouble getting around, and when he heard about a controversial stem cell therapy that might help, he got excited.

"I heard about the stem cell treatments being done right here in Phoenix," said Vondracek, 61. "It shocked me because it was not approved in this country, I didn't think."

The therapy was offered by an Arizona plastic surgeon who gives the stem cell treatments in the same clinic where he does cosmetic procedures.

But when Vondracek's neurologist heard about his interest in the therapy, which would cost $7,000 per treatment, "He went crazy," said Vondracek. He strongly advised Vondracek against it.

Plastic surgeons, other doctors and naturopaths at more than 100 clinics round the country are charging thousands of dollars for a controversial procedure called stem cell therapy to treat a range of disorders, including neurological diseases like MS and Parkinson's.

Robert Vondracek and his girlfriend, Terese Knapik.

The procedure has angered many neurologists and prominent researchers who say these doctors are preying on vulnerable people and capitalizing on the huge but still unrealized potential of stem cell research, which they say is years away from producing an approved treatment for neurological diseases.

"Peddling snake oil in the guise of stem cell therapies is really a threat to legitimate research," said Dr. George Daley, director of the Stem Cell Transplantation Program at Boston Children's Hospital, past president of the International Society for Stem Cell Research and a professor at Harvard Medical School.

"Finding cures is hard, it takes sometimes decades, it's extremely expensive and it's not something that we can just wish and hope for," he said. "It can only be achieved through very, very hard work."

Dr. George Daley is a nationally recognized expert on stem cells at Boston Childrens Hospital and Harvard Medical School.

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Use of Unproven Stem Cell Therapy Questioned

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Stem cells offer great potential in biomedical engineering due to their pluripotency, which is the ability to multiply indefinitely and also to differentiate and develop into any kind of the hundreds of different cells and bodily tissues. But the precise complexity of how stem cell development is regulated throughout states of cellular change has been difficult to pinpoint until now.

By using powerful new single-cell genetic profiling techniques, scientists at the Wyss Institute for Biologically Inspired Engineering and Boston Children's Hospital have uncovered far more variation in pluripotent stem cells than was previously appreciated. The findings, reported today in Nature, bring researchers closer to a day when many different kinds of stem cells could be leveraged for disease therapy and regenerative treatments.

"Stem cell colonies contain much variability between individual cells. This has been considered somewhat problematic for developing predictive approaches in stem cell engineering," said the study's co-senior author James Collins, Ph.D., who is a Wyss Institute Core Faculty member, the Henri Termeer Professor of Medical Engineering & Science at MIT, and a Professor of Biological Engineering at MIT. "Now, we have discovered that what was previously considered problematic variability could actually be beneficial to our ability to precisely control stem cells."

The research team has learned that there are many small fluctuations in the state of a stem cell's pluripotency that can influence which developmental path it will follow.

It's a very fundamental study but it highlights the wide range of states of pluripotency," said George Daley, study co-senior author, Director of Stem Cell Transplantation at Boston Children's Hospital and a Professor of Biological Chemistry and Molecular Pharmacology at Harvard Medical School. "We've captured a detailed molecular profile of the different states of stem cells."

Taking this into account, researchers are now better equipped to manipulate and precisely control which cell and tissue types will develop from an individual pluripotent stem cell or stem cell colony.

"The study was made possible through the use of novel technologies for studying individual cells, which were developed in part by collaborating groups at the Broad Institute, giving our team an unprecedented view of stem cell heterogeneity at the individual cell level," said Patrick Cahan, co-lead author on the study and Postdoctoral Fellow at Boston Children's Hospital and Harvard Medical School.

Researchers explored the developmental landscape of pluripotent stem cells by perturbing them with variants such as different chemicals, culture environments, and genetic knockouts. Then, they analyzed the individual genetic makeup of each cell to observe micro-fluctuations in each stem cell's state of pluripotency. They discovered many small nuances in the way stem cells are influenced by internal, chemical and environmental cues, revealing a complex "decision making" circuit of developmental regulators.

"These emerging single-cell analytics allow us to classify cells very precisely and identify regulatory circuits that control cell states," said the study's co-lead author Roshan Kumar, a former Wyss Institute Postdoctoral Fellow who is now a Senior Scientist at HiFiBiO Inc. and a Visiting Scholar at the Wyss Institute. "The real motivating force behind this study was to understand the causes and consequences of differences between individual stem cells and how the balance of key regulators within a cell can affect that cell's developmental outcome."

Looking at the findings, the researchers now believe there is a "code" that relates patterns of dynamic behavior in stem cell regulatory circuits to the developmental path a cell ends up taking. By leveraging that code, they hope to dial in precisely to specific individual cell states and to use them for a variety of purposes, such as creating certain cell types that a patient's body may be unable to produce on its own.

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New single-cell analysis reveals complex variations in stem cells

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PUBLIC RELEASE DATE:

5-Dec-2014

Contact: Peter Tarr tarr@cshl.edu 516-367-8455 Cold Spring Harbor Laboratory @CSHLnews

Cold Spring Harbor, NY - Scientists at Cold Spring Harbor Laboratory (CSHL) have devised a powerful algorithm that improves the effectiveness of an important research technology harnessing RNA interference, or RNAi.

Discovered in the late 1990s, RNAi is a naturally occurring biological mechanism in which short RNA molecules bind to and "interfere" with messages sent by genes that contain instructions for protein production. Such interference can prevent a gene from being expressed. In addition to helping regulate gene expression, the RNAi pathway in many species, including humans, acts to defend the genome from parasitic viruses and transposons.

Harnessed by scientists since the mid-2000s, RNAi has provided a way to artificially "knock down" the expression of specific genes. By preventing a gene or genes from being activated in a model organism such as a mouse, for instance, much can be learned by inference about gene function. RNAi-based technology also has been extremely useful as tool in drug discovery.

At CSHL, HHMI Professor Gregory Hannon and colleagues have made seminal discoveries over the last decade in elucidating the mechanism through which RNAi operates in nature. They have also pioneered means of harnessing the RNAi pathway, creating comprehensive libraries of small RNA molecules - called short hairpin RNAs, or shRNAs. These have enabled researchers worldwide to selectively shut down specific genes, for instance, across the entire human and mouse genomes.

In research published ahead of print November 26 in Molecular Cell, a Hannon lab team led by Research Investigator Simon Knott reported the creation of an algorithm that enables researchers to identify shRNAs that will be most effective for shutting down specific genes. Before this work, the sequence determinants responsible for optimizing shRNA efficacy were largely unknown. Thus the new research addresses an important technical issue in using RNAi for gene knockdown. The new algorithm was trained to identify sequence determinants and is able to search across each gene and distinguish the sequence targets that will produce the most effective gene knockdown.

Development of the algorithm, called shERWOOD, was based on a massive parallel assessment of the potency of some 250,000 different shRNAs. In effect, a computer was trained to make very accurate predictions, by learning what distinguished the potent shRNAs within this set from those that were less potent. After this learning process the computer was then able to predict the potency of new sequences.

"We built upon this result to design and construct next-generation shRNA libraries targeting the exomes, or protein-encoding gene set, of mice and humans," says Dr. Knott.

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CSHL team finds a way to make shRNA gene knockdown more effective

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Washington DC - infoZine - The research comes from a lab at NIH's Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), which seeks to understand growth.

"Finding the gene responsible for childhood overgrowth would be very helpful, but the much wider question is what regulates growth," said Constantine Stratakis, M.D., D.Sc., lead author of the new paper and the scientific director of the Division of Intramural Research at NICHD.

The research started with a family who came to the NIH Clinical Center for treatment in the mid-1990s. A mother who had been treated for gigantism had two sons who were also growing rapidly. People with this condition are abnormally tall and may have delayed puberty, large hands and feet, and double vision. A second family, with an affected daughter, came to NIH from Australia. The girl had the same duplication the researchers saw in the first family. Dr. Stratakis then contacted Albert Beckers, M.D., Ph.D., at the University of Liege, Belgium. For more than 30 years, Dr. Beckers has been following people with gigantism and acromegaly, a disorder in which excessive growth begins after adulthood. Dr. Beckers agreed to test these patients and identified the same duplication in a group of his patients as well.

Gigantism results from a defect in the pituitary, a pea-sized gland at the base of the brain that makes growth hormones and controls the activity of other glands in the body. Some people with gigantism have a tumor in the pituitary that secretes extra hormone; others just have an oversized pituitary. Gigantism is often treated by removing the tumor, or even the entire pituitary, but can sometimes be treated with medication alone.

"Giants are very rare. If you have three cases in the same family, that is very rare," Dr. Stratakis said. He explained that most of the pituitary gland of the mother in the first family was removed when she was 3 years old, and in adulthood she was only a little above average height. Both of her sons had gigantism, too, and had pituitary surgery.

In their study, the researchers used whole-genome analysis to find major changes in the DNA. Every person in the study who had gigantism as an infant or a toddler had the same defect, a duplication of a stretch of the X chromosome. Family members without gigantism did not have the duplication.

In all, the researchers studied 43 people with gigantism, most from the international cohort studied by Dr. Beckers.

Next the researchers sought to identify which gene might be responsible for the excessive growth. The length of the DNA duplication varied among the patients. But the researchers found the same four genes that were duplicated in all of the patients. After testing the genes, the researchers isolated the most likely suspect: a gene called GPR101. Its activity was up to 1,000 times stronger in the pituitaries of children with the duplication than in the pituitaries of typically developing children.

"We believe GPR101 is a major regulator of growth," Dr. Stratakis said.

The researchers also looked at samples from the pituitary tumors of 248 people with acromegaly, a condition in which adults produce excess growth hormone. None of the patients had the duplication that was seen in people with gigantism. However, 11 of the acromegaly patients did have a mutation in GPR101, suggesting that the gene also may play a role in that condition as well.

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Chromosome Region Linked to Gigantism

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No scientific proof that genetically modified crops are harmful - Central Governmnt
The Genetic Engineering Appraisal Committee has approved field trials of 12 genetically modified crops. Responding to queries in Rajya Sabha regarding the controversial decision, Environment...

By: Puthiya Thalaimurai TV

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No scientific proof that genetically modified crops are harmful - Central Governmnt - Video

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Genetics may play a larger role in causing Lou Gehrig's disease than previously believed, potentially accounting for more than one-third of all cases, according to one of the most comprehensive genetic studies to date of patients who suffer from the condition also known as amyotrophic lateral sclerosis, or ALS.

The study, conducted by investigators at Cedars-Sinai and Washington University in St. Louis, also showed that patients with defects in two or more ALS-associated genes experience disease onset about 10 years earlier than patients with single-gene mutations.

"These findings shed new light on the genetic origins of ALS, especially in patients who had no prior family history of the disease," said Robert H. Baloh, MD, PhD, director of neuromuscular medicine in the Department of Neurology and director of the ALS Program at Cedars-Sinai. Baloh is senior author of the study, published online in Annals of Neurology.

Typically, researchers classify 90 percent of ALS cases as "sporadic," meaning they occur in patients without a family history of the disease. In their study, however, the researchers found a significant degree of genetic involvement in patients with no family history. Examining DNA from 391 individuals, they identified numerous new or very rare ALS gene mutations in such people. Added to the 10 percent of cases already known to be genetic because of family history, the study suggested that more than one-third of all ALS could be genetic in origin.

Baloh said the presence of the new and rare mutations, found among 17 genes already known to be associated with ALS, does not necessarily mean they all cause the disease. But they are considered likely suspects -- especially in combination. ALS often is caused by well-known defects in single genes, but recent studies have suggested that some cases could be brought on by the simultaneous occurrence of two or more "lesser" genetic defects. In theory, each mutation alone might be tolerated without initiating disease, but in combination they exceed the threshold required for disease development.

This study strengthens that possibility: Fifteen patients -- nine of whom had no previous family history of ALS -- had mutations in two or more ALS-associated genes. The research also takes an important next step, showing that multiple genetic defects can influence the way disease manifests in individual patients. Those with mutations in two or more genes had onset about 10 years earlier than those with defects in only one gene.

Matthew B. Harms, MD, assistant professor of neurology at Washington University and co-corresponding author of the article, said that unknown factors still accounted for the majority of ALS cases.

"This tells us that more research is needed to identify other genes that influence ALS risk, and that ultimately, individuals may have more than one gene contributing toward developing disease," Harms said.

ALS is an incurable, virtually untreatable neurodegenerative disease that attacks motor neurons -- nerve cells responsible for muscle function -- in the brain and spinal cord. It causes progressive weakness and eventual failure of muscles throughout the body; patients typically survive three to five years after onset.

Investigators in this study used new-generation technology that quickly and efficiently determines the organizational structure of large numbers of genes. They expect this and similar research to usher in personalized medicine in ALS that will allow healthcare teams to analyze a patient's entire genetic makeup and deliver gene-specific therapies to correct detected defects. Cedars-Sinai researchers recently conducted a disease-in-a-dish study with cells from patients with defects in a gene that commonly causes ALS. Using small segments of genetic material to target the defects, they showed that this type of gene therapy can improve neurons from patients with the disease.

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More patients with Lou Gehrig's disease have genetic origin than previously thought

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THURSDAY, Dec. 4, 2014 (HealthDay News) -- Many American parents would be interested in having their newborn baby undergo in-depth genetic screening to learn about potential health risks, a new study reveals.

Newborns currently get a blood test to screen for at least 30 heritable, treatable conditions. But in-depth genetic screening, known as genomic testing, has the potential to provide more comprehensive personal information, according to the Boston-based researchers.

The researchers surveyed 514 parents within 48 hours of their baby's birth. Parents were given a brief explanation of genes and how they can affect health and medical care, and then were asked what they thought about genomic testing of newborns.

Nearly 83 percent of the parents said they were either extremely (18 percent), very (28 percent) or somewhat (36 percent) interested in in-depth newborn genetic testing, the study found.

"Several other studies have measured parents' interest in newborn genomic screening, but none focused on new parents in the first 48 hours," senior author Dr. Robert Green, a geneticist and researcher at Brigham and Women's Hospital and Harvard Medical School, said in a hospital news release.

"Since this is when genomic testing would be of the greatest value, it is especially important to study parents' attitudes immediately post-partum," he added.

The results were similar regardless of parents' age, gender, race, ethnicity, level of education, family history of genetic disease, or whether their newborn was their first child, the researchers said.

"Parents' strong interest in genomic screening for their newborns, as demonstrated by this study, underscores the importance of further research exploring the public health impacts of actually providing this testing," said Green, "particularly as it continues to become less expensive and more widely available."

Parents who'd had concerns about the health of their newborn were less likely to be interested in genomic testing, according to the study published Dec. 4 in the journal Genetics in Medicine.

-- Robert Preidt

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New Parents Favor In-Depth Genetic Testing, Survey Finds

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Practical Genetics for Basenji Breeders Part 1
Lecture on genetics for basenji breeders presented by Dr Jerold Bell DVM at 2014 BCOA Margaret Greenlee Health Seminar. This is Part 1 (1 hour 20 minutes).

By: BasenjiClubofAmerica

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Practical Genetics for Basenji Breeders Part 1 - Video

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Updated with new information.

SAN FRANCISCO (TheStreet) -- The use of Seattle Genetics' (SGEN) Adcetris as a maintenance therapy after a stem-cell transplant in "high risk" Hodgkin lymphoma patients led to a 20 percentage point improvement in relapse rates compared to a placebo after two years of follow-up, researchers reported Saturday.

The new Adcetris data comes from a phase III study known as AETHERA, which is being presented in full at the American Society of Hematology annual meeting underway here. Next year, Seattle Genetics intends to seek approval from regulators to expand the use of Adcetris into a larger pool of Hodgkin lymphoma patients based on the AETHERA study results.

Must Read: Jim Cramers 4 Best Stock Picks for the Health Care Sector

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Seattle Genetics Makes Case for Expanded Use of Adcetris as Lymphoma Maintenance Therapy

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By RTT News, December 06, 2014, 11:20:00 AM EDT

(RTTNews.com) - Seattle Genetics, Inc. ( SGEN ) and Takeda Pharmaceutical Company Limited reported data demonstrating that Hodgkin lymphoma (HL) patients at risk of relapse following an autologous stem cell transplant (ASCT) who received ADCETRIS (brentuximab vedotin) as consolidation therapy immediately after ASCT had significant improvement in progression-free survival (PFS) compared to patients who received placebo (median of 43 months versus 24 months, respectively; hazard ratio=0.57; p-value=0.001).

ADCETRIS is an antibody-drug conjugate (ADC) directed to CD30, a defining marker of classical HL. ADCETRIS has been approved in more than 45 countries for the treatment of relapsed or refractory HL and systemic anaplastic large cell lymphoma (sALCL). ADCETRIS is currently not approved in the AETHERA treatment setting.

The Phase 3 AETHERA trial was designed to evaluate the potential of single agent ADCETRIS to extend PFS post-ASCT in patients with HL who have at least one risk factor for progression. In addition to the primary endpoint of PFS, secondary endpoints included overall survival (OS), safety and tolerability.

A total of 329 HL patients at risk of relapse were enrolled, including 165 on the ADCETRIS arm and 164 on the placebo arm. Patients received a median of 15 cycles of treatment on both arms, with an average of 12 cycles on the ADCETRIS arm and 11 cycles on the placebo arm.

Key findings include: The trial achieved its primary endpoint and demonstrated a significant increase in PFS per independent review facility (IRF), with a hazard ratio of 0.57 and a p-value of 0.001. Median PFS per IRF was 43 months for patients who received ADCETRIS versus 24 months for patients who received placebo. The two-year PFS rate per IRF was 63 percent in the ADCETRIS arm compared to 51 percent in the placebo arm.

Per investigator, the hazard ratio was 0.50. The two-year PFS rate per investigator was 65 percent in the ADCETRIS arm compared to 45 percent in the placebo arm. The median PFS per investigator has not yet been reached for patients who received ADCETRIS versus 16 months for patients who received placebo. Very few progression events have been observed beyond two years.

One death occurred within 30 days of ADCETRIS treatment from treatment-related acute respiratory distress syndrome (ARDS) associated with pneumonitis. One death occurred on the ADCETRIS arm at Day 40 from ARDS following an episode of treatment-related acute pancreatitis, which had resolved at the time of death.

Submission of safety data from the AETHERA trial to the FDA is a post-marketing requirement that Seattle Genetics will fulfill in its planned supplemental BLA. Takeda plans to submit data from the AETHERA trial to regulatory agencies in its territories.

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Seattle Genetics, Takeda Report Phase3 AETHERA Clinical Trial Data From ADCETRIS

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HGF properties gene therapy

By: Sumin Sin

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HGF properties & gene therapy - Video

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High costs keep patients from using stem cells harvested from umbilical cords

Scientists are studying ways to treat HIV, cerebral palsy and other diseases using umbilical cord blood, although little of the collected blood will actually be used. Credit:Banc de Sang via flickr

Youd think doctors and patients would be clamoring for cells so versatile they could help reboot a body suffering from everything from leukemia to diabetes. But a new report shows that an important source of these stem cellsdiscarded umbilical cordsis rarely used because of high costs and the risk of failure.

Stem cells drawn from newborns umbilical cord blood are sometimes used to treat medical conditions, especially bone and blood cancers like multiple myeloma or lymphoma by replacing dysfunctional blood-producing cells in bone marrow. Generally the diseased cells are destroyed with chemotherapy and irradiation. Then new stem cells are transplanted into the patient to restore function. Cord blood stem cells are an alternative to bone marrow transplants and peripheral blood transplants, in which stem cells are gathered from the blood stream. Cord blood tends to integrate better with the body and it is easier to find a suitable donor than the alternatives.

Yet less than 3 percent of cord blood collected in the U.S. is ever used whereas the rest sits uselessly in blood banks, according to a recent report in Genetic Engineering & Biotechnology News. Immunologist Enal Razvi is author of the report and managing director of Select Biosciences, a biotechnology consulting agency. Razvi found that public cord blood banks, which store donated frozen units for transplants as needed, have only a 1 to 3 percent turnover annually. Most of their inventory sits unused year after year. For example, at Community Blood Services in New Jersey, patients have only used 278 of its 13,000 cords since it opened in 1996, according to business development director Misty Marchioni. Usage is even lower at private cord blood banks, which charge clients thousands of dollars to store a cord in the event a family member one day needs it.

Unlike bone marrow, the main alternative stem cell source, cells transplanted from cord blood carry little risk of graft-versus-host disease, a deadly condition in which the body rejects a transplant. Scientists believe this is because a babys immune system is closer to a blank slate, so their stem cells can integrate with the patients body more easily. But cord blood transplants also take longer to start working, requiring longer hospital stays and upping the bill. Due to storage and testing costs, the cords themselves also get pricy. The cost of the cord is prohibitively high, Razvi explains. Each unit of cord blood costs between $35,000 and $40,000 and most adults require two units for a successful transplant. Insurance companies will generally pay a set amount for a stem cell transplant regardless of where the cells come from. The price tag on a cord blood transplant can run up to $300,000, which may not be fully covered.

Cord blood stem cell transplants also have a higher failure rate than other transplant methods. If the transplant fails, it leaves patients with a compromised immune system in addition to their original disease and medical bills. Because the preparation for transplant includes wiping out the patients original bone marrow, the entire body has to be repopulated with stem cells able to replace it. There are not many stem cells in each cord. Compared with bone marrow or peripheral blood there is a greater chance that there will not be enough stem cells that actually implant and begin producing blood and bone marrow. Its like spreading a small amount of seeds in a big garden, says Mitchell Horwitz, who teaches cell therapy at Duke University Medical Center. Sometimes it just doesnt take.

Martin Smithmyer, chief executive of the private bank Americord, claims that more clients will eventually use their cords, especially as more applications are found for cord blood stem cells. But some scientists disagree. Steven Joffe, a professor of medical ethics at the University of Pennsylvania Perelman School of Medicine, says that many treatments cannot be done with a patients own stem cells because genetic diseases would already be present in the cord blood and that bone marrow might be a better option for relatives. The likelihood they are ever going to use that product is vanishingly small, he says.

Despite the low usage, advocates say cord blood programs have been crucial in improving transplant options for racial minorities, because it can be hard to find a bone marrow match for some groups. Cord blood does not need to match the patient as perfectly as bone marrow. This has transformed the treatment of minority patients, says Andromachi Scaradavou, medical director of the National Cord Blood Program, a public bank based in New York City. In the past we didnt have good donors to offer them. Community Blood Servicess Marchioni also maintains that cord blood is a good emergency option, because finding a compatible bone marrow or peripheral blood donor can take months or years. If you need a transplant quickly, she says, its easy to get cord blood off of a shelf.

Still, experts are working on more efficient ways of ensuring widespread availability of cord blood without having so much of it sit forever unused. Researchers are also continuing to look for ways to improve transplant success and to increase the number of stem cells obtained from each cord, potentially bringing down costs and making cord blood transplants feasible for more patients. If the cost could be lowered, Scaradavou says, it would help a lot of patients get the treatment they need.

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PUBLIC RELEASE DATE:

4-Dec-2014

Contact: John Wallace wallacej@vcu.edu 804-628-1550 Virginia Commonwealth University @vcunews

Is the human immune system similar to the weather, a seemingly random yet dynamical system that can be modeled based on past conditions to predict future states? Scientists at VCU Massey Cancer Center's award-winning Bone Marrow Transplant (BMT) Program believe it is, and they recently published several studies that support the possibility of using next-generation DNA sequencing and mathematical modeling to not only understand the variability observed in clinical outcomes of stem cell transplantation, but also to provide a theoretical framework to make transplantation a possibility for more patients who do not have a related donor.

Despite efforts to match patients with genetically similar donors, it is still nearly impossible to predict whether a stem cell transplant recipient will develop potentially fatal graft-versus-host disease (GVHD), a condition where the donor's immune system attacks the recipient's body. Two studies recently published by the online journal Frontiers in Immunology explored data obtained from the whole exome sequencing of nine donor-recipient pairs (DRPs) and found that it could be possible to predict which patients are at greatest risk for developing GVHD and, therefore, in the future tailor immune suppression therapies to possibly improve clinical outcomes. The data provides evidence that the way a patient's immune system rebuilds itself following stem cell transplantation is representative of a dynamical system, a system in which the current state determines what future state will follow.

"The immune system seems chaotic, but that is because there are so many variables involved," says Amir Toor, M.D., member of the Developmental Therapeutics research program at Massey and associate professor in the Division of Hematology, Oncology and Palliative Care at the VCU School of Medicine. "We have found evidence of an underlying order. Using next-generation DNA sequencing technology, it may be possible to account for many of the molecular variables that eventually determine how well a donor's immune system will graft to a patient."

Toor's first study revealed a large and previously unmeasured potential for developing GVHD for which the conventional approach used for matching DRPs does not account. The conventional approach for donor-recipient compatibility determination uses human leucocyte antigen (HLA) testing. HLA refers to the genes that encode for proteins on the surface of cells that are responsible for regulating the immune system. HLA testing seeks to match DRPs who have similar HLA makeup.

Specifically, Toor and his colleagues used whole exome sequencing to examine variation in minor histocompatibility antigens (mHA) of transplant DRPs. These mHA are protein fragments presented on the HLA molecules, which are the receptors on cells' surface to which these fragments of degraded proteins from within a cell bind in order to promote an immune response. Using advanced computer-based analysis, the researchers examined potential interactions between the mHA and HLA and discovered a high level of mHA variation in HLA-matched DRPs that could potentially contribute to GVHD. These findings may help explain why many HLA-matched recipients experience GVHD, but why some HLA-mismatched recipients experience none remains a mystery. This seeming paradox is explained in a companion paper, also published in the journal Frontiers in Immunology. In this manuscript, the team suggests that by inhibiting peptide generation through immunosuppressive therapies in the earliest weeks following stem cell transplantation, antigen presentation to donor T cells could be diminished, which reduces the risk of GVHD as the recipients reconstitute their T-cell repertoire.

Following stem cell transplantation, a patient begins the process of rebuilding their T-cell repertoire. T cells are a family of immune system cells that keep the body healthy by identifying and launching attacks against pathogens such as bacteria, viruses or cancer. T cells have small receptors that recognize antigens. As they encounter foreign antigens, they create thousands of clones that can later be called upon to guard against the specific pathogen that presented the antigen. Over the course of a person's life, they will develop millions of these clonal families, which make up their T-cell repertoire and protect them against the many threats that exist in the environment.

This critical period where the patient rebuilds their immune system was the focus of the researchers' efforts. In previous research, Toor and his colleagues discovered a fractal pattern in the DNA of recipients' T-cell repertoires. Fractals are self-similar patterns that repeat themselves at every scale. Based on their data, the researchers believe that the presentation of minor histocompatability antigens following transplantation helps shape the development of T-cell clonal families. Thus, inhibiting this antigen presentation through immunosuppressive therapies in patients who have high mHA variation can potentially reduce the risk of GVHD by influencing the development of their T-cell repertoire. This is backed by data from clinical studies that show immune suppression soon after transplantation improves outcomes in unrelated DRPs.

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Predicting the storm: Can computer models improve stem cell transplantation?

Recommendation and review posted by Bethany Smith

Boston, MA (PRWEB) December 04, 2014

In a new report published in the online journal Cell Death and Disease, the Adult Stem Cell Technology Center, LLC (ASCTC) continues to demonstrate its special expertise in uncovering unknown properties that are unique to adult tissue stem cells. In particular, the new study continues to build the companys portfolio of technologies that make previously invisible adult stem cells not only identifiable, but also countable.

The studies were performed with mouse hair follicle stem cells. Because of the universal nature of adult tissue stem cell properties, the new findings are predicted to apply to stem cells in a wide range of human tissues as well.

For the past half century since the experimental demonstration of their existence, it has not been possible to identify adult tissue stem cells exclusive of other related cell types. Consequently, counting them has been impossible, too. Established stem cell therapies like bone marrow transplantation are suboptimal because of this limitation; and the current worldwide flood of thousands of clinical trials of tissue stem cell transplantation therapies has the same problem. Without being able to count potentially curative adult tissue stem cells, there is no way to optimize and standardize successful treatments.

The new report presents a discovery made during studies employing one of the ASCTCs recently defined biomarkers for detecting tissue stem cells. The new biomarker is a member of a family of cell factors called histones that package the cellular DNA into chromosomes. One of the less abundant members of this family is called H2A.Z. In 2011, the ASCTC discovered that H2A.Z is only accessible on the set of chromosomes that segregates to the stem cell sister when a stem divides to produce a non-stem sister cell. The non-stem sister differentiates to replenish lost mature tissue cells. Before a stem cell divides in this manner, the stem cell chromosomes and the non-stem cell chromosomes are distinct because of this difference in their H2A.Z access. This unique feature, called H2A.Z asymmetry, is a highly specific biomarker for identifying adult tissue stem cells.

Because detection of H2A.Z asymmetry does not disrupt other features of stem and non-stem chromosomes, it can be used as a specific landmark to discover other molecular differences between chromosomes destined for the stem cell sister and chromosomes destined for the non-stem sister. The new report describes how two well-known gene regulation modifications of an abundant histone family member, H3, also display asymmetry between stem cell chromosomes and differentiating cell chromosomes.

The newly discovered asymmetric chromosomal patterning of gene regulation modifications in adult tissue stem cells may reveal a long sought mechanism to explain how stem cell fate is maintained in mammalian tissues. This new insight into the function of tissue stem cells addresses a fundamental question in the field of stem cell biology research. ASCTC Director James L. Sherley anticipates that the new report will give stem cell scientists and bioengineers a new lead idea and new research tools for extending knowledge on the molecular workings of adult tissue stem cells. Such advances in knowledge are greatly needed currently to improve the scientific foundation for the increasing number of regenerative medicine clinical trials.

******************************************************************************************** The Adult Stem Cell Technology Center, LLC is a Massachusetts life sciences company. ASCTC Director and founder, James L. Sherley, M.D., Ph.D. is the foremost authority on the unique properties of adult tissue stem cells. The companys patent portfolio contains biotechnologies that solve the three main technical problems production, quantification, and monitoring that have stood in the way of successful commercialization of human adult tissue stem cells for regenerative medicine and drug development. In addition, the portfolio includes novel technologies for isolating cancer stem cells and producing induced pluripotent stem cells. Currently, ASCTC is employing its technological advantages to pursue commercialization of facile methods for monitoring adult tissue stem cell number and function.

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The Adult Stem Cell Technology Center, LLCs New Report on Asymmetric Character of Stem Cell Chromosomes Advances ...

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