Pluristem Therapeutics Ltd. (Nasdaq:PSTI; DAX: PJT: PLTR)has announced that a seven year-old girl suffering from an aplastic bone marrow whose condition was rapidly deteriorating has seen a reversal of her condition. The improvement came due to a significant increase in her red cells, white cells and platelets following the intramuscular injection of Pluristem's PLacental eXpanded (PLX) cells. Aplastic bone marrow is a disease where the patient has no blood-forming hematopoietic stem cells in the bone marrow.

Hadassah Medical Center Bone Marrow Transplantation, Cell Therapy and Transplantation Research Center director Prof. Reuven Or said, "With her body rejecting all possible treatment, and with no other options, we finally turned to Pluristem's PLX cells, which literally saved her life. The results of this unique case indicate that PLX cells may be effective in treating other diseases that affect the bone marrow."

The patient has been hospitalized at the Hadassah Hebrew University Medical Center in Jerusalem since August 2011. Her aplastic bone marrow had been refractory to treatment. So she underwent allogeneic stem cell transplantation from a matched unrelated donor. The first transplant was unsuccessful and the patient remained with bone marrow failure. The patient underwent a second allogeneic stem cell transplantation from a second donor. The bone marrow function was very poor and the patient suffered from recurrent infections.

Two months after the patient's second bone marrow transplant, the child received PLX cells intramuscularly in two doses about one week apart. Some 10 days after the last administration of PLX cells, the patient's hematological parameters began to significantly increase, an effect that has persisted to date. The patient's general clinical status has also improved. Subsequent analysis has indicated that the PLX cells worked by stimulating the recovery of the hematopoietic stem cells contained in the second bone marrow transplant that she had received over two months earlier. Finally, after nine months of hospitalization, the child will be discharged from the hospital.

Pluristem chairman and CEO Zami Alberman said, "Pluristem is extremely happy that our PLX cells have helped this little girl. Remarkably, these beneficial effects were seen in the patient after our PLX cells were administered intramuscularly and correlate with the positive effects on the bone marrow when we administered our PLX cells intramuscularly (IM) in animals exposed to toxic levels of radiation. Pluristem now has several data points to indicate that our PLX cells may work for systemic diseases when given locally, away from the target organ, and without a need to give cells intravenously."

In February 2012, Pluristem announced the results of animal studies suggesting PLX cells can be potentially effective in treating the life threatening hematopoietic complications associated with Acute Radiation Syndrome (ARS). In these experiments, animals given PLX cells IM up to 24 hours post irradiation demonstrated a recovery of their red cells, white cells, platelets and bone marrow to almost normal levels. It was that announcement, and the significant deterioration of the patient following two bone marrow transplants, that led Prof. Or to contact Pluristem about the possible compassionate use of PLX cells to treat his young patient.

Pluristem recently received US FDA clearance to begin a Phase II clinical trial using the company's proprietary PLX-PAD cell product candidate intramuscularly for the treatment of Intermittent Claudication (IC), a subset of peripheral artery disease (PAD).

Published by Globes, Israel business news - http://www.globes-online.com - on May 9, 2012

Copyright of Globes Publisher Itonut (1983) Ltd. 2012

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(CBS News) Patients with brain cancer may face devastating side effects from chemotherapy, but a new study offers a possible solution: stem cells.

Yearly dental X-rays raise brain tumor risk, study finds

Memorial Hermann hospital in Houston to live tweet brain tumor surgery

The stem cells form a shield of sorts against the toxic side effects from chemo, according to the researchers behind the study. It was a small trial that involved only three patients with glioblastoma, the most aggressive and common form of a malignant brain tumor that's usually fatal.

Two of the patients survived longer than predicted with help from the stem cell treatment - an average of 22 months - and a third man from Alaska remains alive today with no disease progression almost three years following treatment.

How does it work?

Many patients with the deadly form of brain cancer possess a gene called MGMT. The MGMT gene is typically turned on and counters the effects from some chemotherapy agents, such as temozolomide, rendering them less effective. As such, people with such a gene often have a particularly poor prognosis.

A drug called benzylguanine can block the MGMT gene, thus making tumors more receptive to chemotherapy, but the combination of the drug and chemo are often too toxic for healthy bone marrow cells.

That's where the new stem cell treatment comes in. By combining bone marrow stem cells with a modified version of MGMT in the form of the new treatment, patients' cells were protected from the toxic effects of the cancer drugs and chemotherapy while keeping the tumor cells targeted.

"This therapy is analogous to firing at both tumor cells and bone marrow cells, but giving the bone marrow cells protective shields while the tumor cells are unshielded," study author Dr. Jennifer Adair, a researcher at the Fred Hutchinson Cancer Research Center in Seattle, said in a news release.

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LAS VEGAS, May 10, 2012 /PRNewswire/ -- An investigational therapyderived from a patient's own bone marrow stem cells improves heart function in some patients with progressive heart failure due to dilated cardiomyopathy (DCM), according to the results of a Phase 2a study presented today as a late-breaking clinical trial at the SCAI 2012 Scientific Sessions.

Ixmyelocel-T is developed by culturing a patient's bone marrow for 12 days to increase the numbers of immune cells including macrophages and monocytes, as well as mesenchymal cells, stem cells that can differentiate into several different cell types. The resulting cell treatment is then injected into the patient's heart muscles to encourage growth of new tissue and improve inflammation.

"An increasing number of patients have progressive heart failure due to dilated cardiomyopathy, even after treatment with drug therapy and surgical intervention," said Timothy Henry, MD, FSCAI, director of research and an interventional cardiologist at the Minneapolis Heart Institute at Abbott Northwestern Hospital, and the study's principal investigator. "In this study, patients treated with ixmyelocel-T showed repair in damaged heart muscle and some reversal in heart failure symptoms."

The trial included 22 ischemic (IDCM) and non-ischemic (NIDCM) patients with a New York Heart Association (NYHA) heart failure class of III or IV, or moderate to severe heart failure, and a left ventricular ejection fraction of 30 percent or less, which is a measure of how much blood leaves the heart with each pump. Patients were randomized to receive an injection of the treatment into their heart muscles or to a control group, and were followed at 3, 6 and 12 months.

After 12 months, no procedural complications and no difference in adverse events were reported among patients who received the treatment and the control group. IDCM patients who received the cell treatment had a lower mean number of major adverse clinical events (0.33 compared to 1.67 in the control group). IDCM patients who received the treatment were more likely to see improvement in NYHA class, six-minute walking distance and ejection fraction, compared to NIDCM patients who received the treatment and those in the control group.

"Treatment with ixmyelocel-T was well-tolerated and patients who received the cell therapy showed improved symptoms after one year," said Dr. Henry. "The results provide a strong basis for a larger clinical trial of this treatment in patients with dilated cardiomyopathy."

The study was sponsored by Aastrom Biosciences.

Dr. Henry will present "Safety and Efficacy ofIxmyelocel-T, An Expanded Patient-Specific Mixed Cell Therapy, in Dilated Cardiomyopathy" on Thursday, May 10, 2012, in the Late-Breaking Clinical Trials Session beginning at 12:00 p.m. (Pacific Time).

About SCAI

Headquartered in Washington, D.C., the Society for Cardiovascular Angiography and Interventions is a 4,000-member professional organization representing invasive and interventional cardiologists in approximately 70 nations. SCAI's mission is to promote excellence in invasive and interventional cardiovascular medicine through physician education and representation, and advancement of quality standards to enhance patient care. SCAI's patient education program, Seconds Count, offers comprehensive information about cardiovascular disease. For more information about SCAI and Seconds Count, visit http://www.scai.org or http://www.SecondsCount.org.

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A stem cell breakthrough at UCLA could mark a big step for a biopharmaceutical company to use its proprietary technology to forge partnerships with pharmaceutical companies and other research institutions.

Fibrocell Sciences technology isolates, purifies and multiplies a patients fibroblast cells, connective skin cells that make collagen. In a research collaboration with the company, UCLA used the technology to isolate, identify and increase the number of different skin cell types, which lead to two rare adult stem cell-like subpopulations being identified in adult human skin SSEA3-expressing regeneration-associated cells associated with skin regeneration after injuries and mesenchymal adult stem cells.

The findings could have broad applications for personalized medicine. Currently, adult stem cells are derived from adipose tissue and bone marrow. Using mesenchymal stem cells would be less invasive and could be more efficient. Mesenchymal stem cells are being used in research to develop osteoblasts, or bone cells; chondrocytes, or cartilage cells; and adipocytes, or fat cells.

David Pernock, the chairman and CEO of Fibrocell, said the move could mark a significant step in the companys growth.

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CARLSBAD, Calif.--(BUSINESS WIRE)--

International Stem Cell Corporation (OTCBB: ISCO.OB - News) http://www.internationalstemcell.com today announced that the Company has developed new technologies to commercialize the use of human parthenogenetic stem cells (hpSC) to treat human diseases. The methods announced today are capable of producing populations of stem cells and their therapeutically valuable derivatives not only to a higher level of purity but also at a cost that is approximately several times lower than previously reported techniques.

ISCOs research team has developed a new method to derive high-purity populations of neural stem cells (NSC) from hpSC and further differentiate them into dopaminergic neurons. This method is capable of generating sufficient quantities of neuronal cells for ISCOs pre-clinical and clinical studies and is highly efficient as it requires substantially less time and labor in addition to using fewer costly materials than traditional methods. ISCOs technologies make possible the creation of billions of neuronal cells necessary for conducting such studies from a small batch of stem cells.

ISCO has also announced today that it has developed a new high-throughput cell culture method for growing human parthenogenetic stem cells (hpSC) in large quantities. This new technique is easily scalable and can produce the quantities of cGMP grade hpSC necessary for commercial and therapeutic applications.

One of the most challenging issues in commercializing stem cell based treatments is creating high-purity populations of stem cell derivatives at a reasonable cost. I believe the new methods we have developed solve this important problem and help position us for future clinical studies, says Dr. Ruslan Semechkin, Vice President, R&D.

About International Stem Cell Corporation

International Stem Cell Corporation is focused on the therapeutic applications of human parthenogenetic stem cells (hpSCs) and the development and commercialization of cell-based research and cosmetic products. ISCO's core technology, parthenogenesis, results in the creation of pluripotent human stem cells from unfertilized oocytes (eggs). hpSCs avoid ethical issues associated with the use or destruction of viable human embryos. ISCO scientists have created the first parthenogenic, homozygous stem cell line that can be a source of therapeutic cells for hundreds of millions of individuals of differing genders, ages and racial background with minimal immune rejection after transplantation. hpSCs offer the potential to create the first true stem cell bank, UniStemCell. ISCO also produces and markets specialized cells and growth media for therapeutic research worldwide through its subsidiary Lifeline Cell Technology, and stem cell-based skin care products through its subsidiary Lifeline Skin Care (www.lifelineskincare.com). More information is available at http://www.internationalstemcell.com or follow us on Twitter @intlstemcell.

To receive ongoing corporate communications, please click on the following link: http://www.b2i.us/irpass.asp?BzID=1468&to=ea&s=0.

Forward-looking Statements

Statements pertaining to anticipated developments, the potential benefits of research programs and new manufacturing technologies, and other opportunities for the company and its subsidiaries, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates,") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products and technologies regulatory approvals, need and ability to obtain future capital, application of capital resources among competing uses, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the company's business, particularly those mentioned in the cautionary statements found in the company's Securities and Exchange Commission filings. The company disclaims any intent or obligation to update forward-looking statements.

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THE WOODLANDS, Texas--(BUSINESS WIRE)--

Opexa Therapeutics, Inc. (NASDAQ:OPXA - News), a company developing Tovaxin, a novel T-cell therapy for multiple sclerosis (MS), today reported financial results for the quarter ended March 31, 2012 and provided an update on recent corporate developments.

Corporate development highlights include:

We are progressing favorably in the planning and preparations for a clinical trial in Secondary Progressive MS patients, commented Neil K. Warma, President and Chief Executive Officer of Opexa. The clinical trial protocol was submitted to the FDA for final review and we received no additional comments from them. This is a key milestone for us, providing us with the necessary regulatory positioning to advance with the trial preparations and discussions with potential trial sites. Operationally, we believe we remain on track to initiate the trial in the coming months as the team continues to work diligently to finalize preparations. In order to initiate the trial, we will need to secure additional financing, either through a potential partnership or additional capital raise, and this continues to be an important focus for us.

The meeting we recently held with clinical trial investigators at the annual American Academy of Neurology conference was well attended and included some of the most influential MS opinion leaders participating in the discussions related to our clinical trial design and enrollment criteria. We are anticipating strong enrollment once we are able to begin the trial, driven by the support of the clinicians and a strong desire from patients to participate. SPMS is understood to be a very serious medical condition coupled with the fact that patients have limited treatment options. We hope that Tovaxin can eventually develop into a treatment of choice, not only for SPMS but for all MS patients.

As of the end of the first quarter, our cash and cash equivalents totaled approximately $4.7million and our monthly burn rate for the quarter was approximately $810,000, inclusive of increased cash expenditures to support preparations for the initiation of the planned Phase IIb clinical study in North America.

First Quarter Financial Results

Opexa reported no commercial revenues in the three months ended March 31, 2012 or in the comparable prior-year period.

Research and development expenses were $1,490,097 for the three months ended March 31, 2012, compared with $685,161 for the three months ended March 31, 2011. The increase in expenses was primarily related to an increase in the procurement and use of laboratory reagents and supplies, and increases in professional service fees, employee personnel costs, facility costs and non-cash stock compensation expense.

General and administrative expenses for the three months ended March 31, 2012 were $816,196, compared with $592,058 for the three months ended March 31, 2011. The increase in expense is due to increases in business development activities, legal expenses, employee compensation expense and non-cash stock compensation expense.

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Opexa Therapeutics Reports First Quarter 2012 Financial Results and Provides Corporate Update

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By Elizabeth Lopatto - Wed May 09 18:00:00 GMT 2012

An experimental therapy that reprograms the immune system then spurs the growth of healthy insulin-producing cells reversed late-stage diabetes in mice and may lead to a cure for people, researchers said.

Mice with Type 1 diabetes, a form of the disease in which the bodys immune system destroys cells that secrete insulin, were free of illness after scientists shut down the immune attack, reprogrammed the errant cells and coaxed the growth of healthy, new insulin-producers. The study was published today in the journal Science Translational Medicine.

About 3 million Americans have Type 1 diabetes, which is usually diagnosed in children, according to the Juvenile Diabetes Research Foundation. The only treatment is insulin injected to replace the bodys naturally-occurring version of the hormone, which is needed to convert blood sugar into energy. The experimental immune system approach appears promising because its the first time diabetes has been cured in mice with advanced disease, said Anita Chong, a medical researcher at the University of Chicago.

Conceptually, each component isnt novel, people have thought about them, but put it together and show it can work? said Chong, who wrote an accompanying editorial to the study. Thats very exciting.

There are many more steps before the treatment will be tested in humans, starting with non-human primate models, she said in a telephone interview.

Type 1 diabetes differs from the more common Type 2 form in that it is an autoimmune disease, in which the immune system kills the cells needed to produce insulin. In Type 2 diabetes, the body produces insulin but cells no longer respond to it.

Diabetics must test their blood sugar several times a day, and sometimes experience hypoglycemia, or dangerously low blood sugar, while they sleep at night.

In the study, the mice were given antibodies to attack two kinds of immune cells that kill the pancreas insulin-producing beta cells. Then the mice had a bone marrow transplant to replenish the vanquished cells. Bone marrow is where blood cells are made, and the transplant let the mice make immune cells that wouldnt attack the beta cells. A treatment with pancreas growth factor spurred creation of new beta cells.

The study was led by Defu Zeng, an endocrinologist at City of Hope medical center in Duarte, California, and funded by the Iacocca Family Foundation, and private donations from Todd and Karen Wanek and the Davis family.

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Immune Cell Therapy Cures Diabetic Mice, Researchers Find

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Wed May 9, 2012 3:35pm BST

(Reuters) - Pluristem Therapeutics Inc said a 7-year old girl suffering from a bone marrow disease experienced a reversal of her condition after receiving its experimental stem cell therapy, sending the Israeli company's shares up 32 percent.

The girl, suffering from aplastic bone marrow in which the patient has no blood-forming stem cells, had a significant rise in her red cells, white cells and platelets following an injection of Pluristem's therapy -- PLacental eXpanded cells.

"The results of this unique case indicate that PLX cells may be effective in treating other diseases that affect the bone marrow," Reuven Or, the child's physician at Hadassah Medical Center, was quoted in a statement by Pluristem.

Last September, the company said animal studies showed that the therapy had the potential to treat blood tissue complications related with acute radiation syndrome, commonly called radiation sickness.

Last month, the U.S. health regulators gave a go ahead to the company to start a mid-stage trial of the therapy for treating Intermittent Claudication -- a subset of peripheral artery disease.

Pluristem shares, which have gained 5 percent since receiving the FDA nod for the mid-stage trial, were up 15 percent at $2.70 in morning trade on the Nasdaq. They touched a high of $3.10 earlier.

(Reporting by Esha Dey in Bangalore; Editing by Gopakumar Warrier)

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By Kate Rauch on May 11, 2012

Chronic pain affects an estimated 116 million Americans and costs $635 billion each year in medical treatment and lost productivity.

Aditi Bhargava, PhD

Aditi Bhargava, PhD, associate professor at UCSFs School of Medicine, is using a technique known as RNA interference (RNAi) to develop a gene therapy system that sends specific commands to certain neurons, or nerve cells, telling them to turn off pain, or stop inflammation.

The current treatments for pain dull everything, Bhargava said. You have a little fire in the kitchen, but your only solution is a fire hose that floods the entire house. You put out the fire, but youre affecting the whole house in the process a huge negative side effect.

Likening her method to a Trojan horse, Bhargavas novel therapeutic approach essentially hides the pain-silencing commands, carried by distinct proteins that affect cellular function, inside other proteins which bind only to the troublemaker cells. Once attached, they release their hidden power.

We want to target the small or medium neurons that sense pain, while leaving other neurons unaffected, she said. Were hoping that while you reduce pain, for example, youll still be able to chew or not drool.

The data from her proof-of-concept animal studies look promising, Bhargava said. I believe that this project has tremendous translational potential to turn what we learn into concrete benefits for patients.

Targeted pain and inflammation relief could also be used to treat illnesses, such as Inflammatory Bowel Disease (IBD), an autoimmune condition. This approach has potential to not only minimize unwanted side effects, but save costs, as it sends very small amounts of drug therapies to the targeted cells.

Targeted delivery of drug would reduce the dose required to treat, reduce cost, and most importantly, reduce unwanted side effects.

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Targeted Gene Therapy Offers Relief from Pain and Inflammation

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09-05-2012 19:22 Humanity faces increasing problems around the supply and quality of water. The dual implications of an ever expanding population, combined with the threat of climate change, makes water a precious commodity. Delving deep into the composition of water however, may help its preservation into the future. In this presentation, Professor Chris Saint will share examples from his research where gene technology has been applied to identify and record microorganisms that cause problems in our water supplies. These include blue-green algae and protozoan parasites such as Cryptosporidium. For the first time DNA tests are being developed to rapidly assess the risk that these organisms present to drinking water consumers. Research has also demonstrated that not all bugs in water are bad -- some can be used to breakdown key pollutants, leading to a possibility of using bacteria to clean up our water supplies. Professor Saint will discuss how these discoveries will lead the way in monitoring water quality, and share his thoughts on what the future holds in this area.

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Christopher Saint - Water quality management - it can be in the genes - Knowledge Works - Video

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10-05-2012 04:57 Watch in 1080p or 720p for best experience! I hope you enjoy watching the video. I do not own the music in this video! All rights reserved to DVBBS & Arkasia! Follow Arkasia: Follow DVBBS: Copyright Disclaimer Under Section 107 of the Copyright Act 1976, allowance is made for "fair use" for purposes such as criticism, comment, news reporting, teaching, scholarship, and research. Fair use is a use permitted by copyright statute that might otherwise be infringing. Non-profit, educational or personal use tips the balance in favor of fair use.

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

Contact: Tracey Peake tracey_peake@ncsu.edu 919-515-6142 North Carolina State University

Imagine being able to control genetic expression by flipping a light switch. Researchers at North Carolina State University are using light-activated molecules to turn gene expression on and off. Their method enables greater precision when studying gene function, and could lead to targeted therapies for diseases like cancer.

Triplex-forming oligonucleotides (TFOs) are commonly used molecules that can prevent gene transcription by binding to double-stranded DNA. NC State chemist Dr. Alex Deiters wanted to find a way to more precisely control TFOs, and by extension, the transcription of certain genes. So Deiters attached a light-activated "cage" to a TFO. When exposed to ultraviolet (UV) light, the cage is removed, and the TFO is free to bind with DNA, inhibiting transcription of the gene of interest.

"In the absence of light, transcription activity is 100 percent," says Deiters. "When we turn on the light, we can take it down to about 25 percent, which is a significant reduction in gene expression."

Additionally, Deiters fine-tuned the process by attaching a caged inhibitor strand to the TFO. In the absence of UV light, the TFO behaves normally, binding to DNA and preventing gene expression. However, when exposed to UV light, the caged inhibitor activates and stops the TFO from binding with DNA, turning gene transcription on.

"We've created a tool that allows for the light-activation of genetic transcription," Deiters says. "By giving researchers greater temporal and spatial control over gene expression, we've expanded their ability to study the behavior of particular genes in whichever environment they choose."

###

The research appears online in ACS Chemical Biology, and was funded by the National Institutes of Health. Deiters worked with NC State graduate students Jeane M. Govan, Rajendra Uprety and James Hemphill and Wake Forest University's Mark O. Lively on the research.

Note to editors: An abstract of the paper follows.

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Researchers use light to switch on gene expression

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ScienceDaily (May 10, 2012) History, science and discovery come together to help family members affected by this syndrome make informed family planning choices.

C5ORF42 was identified as the gene that causes Joubert Syndrome in a number of families in the Lower St. Lawrence region of Quebec where the causal gene had remained unknown since the initial description of the syndrome in 1969. This is what a study in the April issue of The American Journal of Human Genetics reveals. The study was conducted by researchers from the Sainte-Justine University Hospital Research Center and the Centre of Excellence in Neuromics of Universit de Montral (CENUM).

Joubert Syndrome is a condition that affects brain development and manifests itself through delayed psychomotor development, abnormal coordination of eye movements and respiratory abnormalities. Since Dr. Marie Joubert and her colleagues described it for the first time in 1969, a number of related genes have been identified in various populations, but the causal gene of the Quebec form of the syndrome has remained until now unknown.

"No studies had been done to identify the genetic origin of the disease in Quebec, more specifically in the exact area of the Lower St. Lawrence where most cases in Quebec are concentrated," Dr. Jacques Michaud, the study's principal investigator, explained. "This is the first study to present a picture of Joubert syndrome in the Quebec population. It will allow family members affected by the syndrome to assess their children's genetic risks with a simple DNA test."

The finding is interesting, both genetically and historically, since, while Joubert Syndrome is present around the world, genetic strains can vary regionally. Distribution is related to the history of various population groups.

Quebec is no exception. In fact, 6,000 French Canadian settlers from Quebec City and its surroundings settled in the Lower St. Lawrence region about the end of the 17th and beginning of the 18th centuries. The fact that most present day inhabitants of the Lower St. Lawrence region descend from this small group of settlers suggests a genetic founder effects. Indeed, certain founder mutations are transmitted to a large number of descendants, which increases the risk of genetic diseases in offspring.

As a matter of fact, Dr. Michaud's team identified three C5ORF42 mutations that are common to most of the families in the study. In all, seven families that are carriers of the gene were identified in a 400-km area along Route 132.

In the space of about two centuries, the first colonists settled in the Lower St. Lawrence region and from there other families headed out to settle along the river heading east as far as Mont-Joli and then along the Matapedia River. According to Myriam Srour, a doctoral student and co-author of the study, the mutations likely spread in the population along these migration routes.

So far 15 genes that play a role in the expression of the syndrome have been revealed elsewhere in the world. Dr. Michaud and his team will continue their research to better understand the exact function of the gene and the specific effect of each mutation.

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Gene that causes Joubert Syndrome discovered

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

Contact: William Raillant-Clark w.raillant-clark@umontreal.ca 514-343-7593 University of Montreal

This press release is available in French.

C5ORF42 was identified as the gene that causes Joubert Syndrome in a number of families in the Lower St. Lawrence region of Quebec where the causal gene had remained unknown since the initial description of the syndrome in 1969. This is what a study in the April issue of The American Journal of Human Genetics reveals. The study was conducted by researchers from the Sainte-Justine University Hospital Research Center and the Centre of Excellence in Neuromics of Universit de Montral (CENUM).

Joubert Syndrome is a condition that affects brain development and manifests itself through delayed psychomotor development, abnormal coordination of eye movements and respiratory abnormalities. Since Dr. Marie Joubert and her colleagues described it for the first time in 1969, a number of related genes have been identified in various populations, but the causal gene of the Quebec form of the syndrome has remained until now unknown.

"No studies had been done to identify the genetic origin of the disease in Quebec, more specifically in the exact area of the Lower St. Lawrence where most cases in Quebec are concentrated," Dr. Jacques Michaud, the study's principal investigator, explained. "This is the first study to present a picture of Joubert syndrome in the Quebec population. It will allow family members affected by the syndrome to assess their children's genetic risks with a simple DNA test."

The finding is interesting, both genetically and historically, since, while Joubert Syndrome is present around the world, genetic strains can vary regionally. Distribution is related to the history of various population groups.

Quebec is no exception. In fact, 6,000 French Canadian settlers from Quebec City and its surroundings settled in the Lower St. Lawrence region about the end of the 17th and beginning of the 18th centuries. The fact that most present day inhabitants of the Lower St. Lawrence region descend from this small group of settlers suggests a genetic founder effects. Indeed, certain founder mutations are transmitted to a large number of descendants, which increases the risk of genetic diseases in offspring.

As a matter of fact, Dr. Michaud's team identified three C5ORF42 mutations that are common to most of the families in the study. In all, seven families that are carriers of the gene were identified in a 400-km area along Route 132.

In the space of about two centuries, the first colonists settled in the Lower St. Lawrence region and from there other families headed out to settle along the river heading east as far as Mont-Joli and then along the Matapedia River. According to Myriam Srour, a doctoral student and co-author of the study, the mutations likely spread in the population along these migration routes.

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Discovery of a gene that causes Joubert Syndrome

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ScienceDaily (May 9, 2012) For the first time, scientists at Fred Hutchinson Cancer Research Center have transplanted brain cancer patients' own gene-modified blood stem cells in order to protect their bone marrow against the toxic side effects of chemotherapy. Initial results of the ongoing, small clinical trial of three patients with glioblastoma showed that two patients survived longer than predicted if they had not been given the transplants, and a third patient remains alive with no disease progression almost three years after treatment.

"We found that patients were able to tolerate the chemotherapy better and without negative side effects after transplantation of the gene-modified stem cells than patients in previous studies who received the same type of chemotherapy without a transplant of gene-modified stem cells," said Hans-Peter Kiem, M.D., senior and corresponding author of the study published in the May 9 issue of Science Translational Medicine.

Kiem, a member of the Clinical Research Division at the Hutchinson Center, said that a major barrier to effective use of chemotherapy to treat cancers like glioblastoma has been the toxicity of chemotherapy drugs to other organs, primarily bone marrow. This results in decreased blood cell counts, increased susceptibility to infections and other side effects. Discontinuing or delaying treatment or reducing the chemotherapy dose is generally required, but that often results in less effective treatment.

In the current study, Kiem and colleagues focused on patients with glioblastoma, an invariably fatal cancer. Many of these patients have a gene called MGMT (O6-methylguanine-DNA-methyltransferase) that is turned on because the promoter for this gene is unmethylated. MGMT is a DNA repair enzyme that counteracts the toxic effect of some chemotherapy agents like temozolomide. Patients with such an unmethylated promoter status have a particularly poor prognosis.

A drug called benzylguanine can block the MGMT gene and make tumor cells sensitive to chemotherapy again, but when given with chemotherapy, the toxic effects of this combination are too much for bone marrow cells, which results in marrow suppression.

By giving bone marrow stem cells P140K, which is a modified version of MGMT, those cells are protected from the toxic effects of benzylguanine and chemotherapy, while the tumor cells are still sensitive to chemotherapy. "P140K can repair the damage caused by chemotherapy and is impervious to the effects of benzylguanine," Kiem said.

"This therapy is analogous to firing at both tumor cells and bone marrow cells, but giving the bone marrow cells protective shields while the tumor cells are unshielded," said Jennifer Adair, Ph.D., who shares first authorship of the study with Brian Beard, Ph.D., both members of Kiem's lab.

The three patients in this study survived an average of 22 months after receiving transplants of their own circulating blood stem cells. One, an Alaskan man, remains alive 34 months after treatment. Median survival for patients with this type of high-risk glioblastoma without a transplant is just over a year.

"Glioblastoma remains one of the most devastating cancers with a median survival of only 12 to 15 months for patients with unmethylated MGMT," said Maciej Mrugala, M.D., the lead neuro oncologist for this study.

As many as 50 percent to 60 percent of glioblastoma patients harbor such chemotherapy-resistant tumors, which makes gene-modified stem cell transplant therapy applicable to a large number of these patients. In addition, there are also other brain tumors such as neuroblastoma or other solid tumors with MGMT-mediated chemo resistance that might benefit from this approach.

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Gene-modified stem cell transplant protects patients from toxic side effects of chemotherapy, study suggests

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Study is first to show feasibility and efficacy of a new use for autologous stem cell transplant

Newswise SEATTLE For the first time, scientists at Fred Hutchinson Cancer Research Center have transplanted brain cancer patients own gene-modified blood stem cells in order to protect their bone marrow against the toxic side effects of chemotherapy. Initial results of the ongoing, small clinical trial of three patients with glioblastoma showed that two patients survived longer than predicted if they had not been given the transplants, and a third patient remains alive with no disease progression almost three years after treatment.

We found that patients were able to tolerate the chemotherapy better and without negative side effects after transplantation of the gene-modified stem cells than patients in previous studies who received the same type of chemotherapy without a transplant of gene-modified stem cells, said Hans-Peter Kiem, M.D., senior and corresponding author of the study published in the May 9 issue of Science Translational Medicine.

Kiem, a member of the Clinical Research Division at the Hutchinson Center, said that a major barrier to effective use of chemotherapy to treat cancers like glioblastoma has been the toxicity of chemotherapy drugs to other organs, primarily bone marrow. This results in decreased blood cell counts, increased susceptibility to infections and other side effects. Discontinuing or delaying treatment or reducing the chemotherapy dose is generally required, but that often results in less effective treatment.

In the current study, Kiem and colleagues focused on patients with glioblastoma, an invariably fatal cancer. Many of these patients have a gene called MGMT (O6-methylguanine-DNA-methyltransferase) that is turned on because the promoter for this gene is unmethylated. MGMT is a DNA repair enzyme that counteracts the toxic effect of some chemotherapy agents like temozolomide. Patients with such an unmethylated promoter status have a particularly poor prognosis.

A drug called benzylguanine can block the MGMT gene and make tumor cells sensitive to chemotherapy again, but when given with chemotherapy, the toxic effects of this combination are too much for bone marrow cells, which results in marrow suppression.

By giving bone marrow stem cells P140K, which is a modified version of MGMT, those cells are protected from the toxic effects of benzylguanine and chemotherapy, while the tumor cells are still sensitive to chemotherapy. P140K can repair the damage caused by chemotherapy and is impervious to the effects of benzylguanine, Kiem said.

This therapy is analogous to firing at both tumor cells and bone marrow cells, but giving the bone marrow cells protective shields while the tumor cells are unshielded, said Jennifer Adair, Ph.D., who shares first authorship of the study with Brian Beard, Ph.D., both members of Kiems lab.

The three patients in this study survived an average of 22 months after receiving transplants of their own circulating blood stem cells. One, an Alaskan man, remains alive 34 months after treatment. Median survival for patients with this type of high-risk glioblastoma without a transplant is just over a year.

Glioblastoma remains one of the most devastating cancers with a median survival of only 12 to 15 months for patients with unmethylated MGMT, said Maciej Mrugala, M.D., the lead neuro oncologist for this study.

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Transplanted Gene-Modified Blood Stem Cells Protect Brain Cancer Patients From Toxic Side Effects of Chemotherapy

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

Contact: Dean Forbes dforbes@fhcrc.org 206-667-2896 Fred Hutchinson Cancer Research Center

SEATTLE For the first time, scientists at Fred Hutchinson Cancer Research Center have transplanted brain cancer patients' own gene-modified blood stem cells in order to protect their bone marrow against the toxic side effects of chemotherapy. Initial results of the ongoing, small clinical trial of three patients with glioblastoma showed that two patients survived longer than predicted if they had not been given the transplants, and a third patient remains alive with no disease progression almost three years after treatment.

"We found that patients were able to tolerate the chemotherapy better and without negative side effects after transplantation of the gene-modified stem cells than patients in previous studies who received the same type of chemotherapy without a transplant of gene-modified stem cells," said Hans-Peter Kiem, M.D., senior and corresponding author of the study published in the May 9 issue of Science Translational Medicine.

Kiem, a member of the Clinical Research Division at the Hutchinson Center, said that a major barrier to effective use of chemotherapy to treat cancers like glioblastoma has been the toxicity of chemotherapy drugs to other organs, primarily bone marrow. This results in decreased blood cell counts, increased susceptibility to infections and other side effects. Discontinuing or delaying treatment or reducing the chemotherapy dose is generally required, but that often results in less effective treatment.

In the current study, Kiem and colleagues focused on patients with glioblastoma, an invariably fatal cancer. Many of these patients have a gene called MGMT (O6-methylguanine-DNA-methyltransferase) that is turned on because the promoter for this gene is unmethylated. MGMT is a DNA repair enzyme that counteracts the toxic effect of some chemotherapy agents like temozolomide. Patients with such an unmethylated promoter status have a particularly poor prognosis.

A drug called benzylguanine can block the MGMT gene and make tumor cells sensitive to chemotherapy again, but when given with chemotherapy, the toxic effects of this combination are too much for bone marrow cells, which results in marrow suppression.

By giving bone marrow stem cells P140K, which is a modified version of MGMT, those cells are protected from the toxic effects of benzylguanine and chemotherapy, while the tumor cells are still sensitive to chemotherapy. "P140K can repair the damage caused by chemotherapy and is impervious to the effects of benzylguanine," Kiem said.

"This therapy is analogous to firing at both tumor cells and bone marrow cells, but giving the bone marrow cells protective shields while the tumor cells are unshielded," said Jennifer Adair, Ph.D., who shares first authorship of the study with Brian Beard, Ph.D., both members of Kiem's lab.

The three patients in this study survived an average of 22 months after receiving transplants of their own circulating blood stem cells. One, an Alaskan man, remains alive 34 months after treatment. Median survival for patients with this type of high-risk glioblastoma without a transplant is just over a year.

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Gene-modified stem cell transplant protects patients from toxic side effects of chemotherapy

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

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

New Rochelle, NY, May 9, 2012Disorders of consciousness such as coma or a vegetative state caused by severe brain injury are poorly understood and their diagnosis has relied mainly on patient responses and measures of brain activity. However, new functional and imaging-based diagnostic tests that measure communication and signaling between different brain regions may provide valuable information about the potential for consciousness in patients unable to communicate. These innovative approaches are described and compared in a Review article in the groundbreaking neuroscience journal Brain Connectivity, a bimonthly peer-reviewed publication from Mary Ann Liebert, Inc., publishers. The article is available free on the Brain Connectivity website at http://www.liebertpub.com/brain.

Mlanie Boly and coauthors from University of Lige (Belgium), University of Milan (Italy), and University College London (UK) compare the benefits and limitations of three methods for studying the dynamics of brain communication and connectivity in response to internal and external stimulation: functional magnetic resonance imaging f(MRI); transcranial magnetic stimulation (TMS) combined with electroencephalograpy (EEG); and response to neuronal perturbation, measuring, for example, sensory evoked potentials (ERP). They report their findings and propose future research directions in the article "Brain Connectivity in Disorders of Consciousness."

"In recent years, there has been a tremendous interest in gaining a better understanding of the various disorders of consciousness. A variety of methods including fMRI and PET have been used to study these disorders," says Bharat Biswal, PhD, Co-Editor-in-Chief of Brain Connectivity and Associate Professor, University of Medicine and Dentistry of New Jersey. "This article provides a comprehensive analysis using three new and innovative methods to study disorders of consciousness."

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About the Journal

Brain Connectivity is the journal of record for researchers and clinicians interested in all aspects of brain connectivity. The Journal is under the leadership of Founding and Co-Editors-in-Chief Christopher Pawela, PhD, Assistant Professor, Medical College of Wisconsin, and Bharat Biswal, PhD. It includes original peer-reviewed papers, review articles, point-counterpoint discussions on controversies in the field, and a product/technology review section. To ensure that scientific findings are rapidly disseminated, articles are published Instant Online within 72 hours of acceptance, with fully typeset, fast-track publication within 4 weeks. Tables of content and a sample issue may be viewed on the Brain Connectivity website at http://www.liebertpub.com/brain.

About the Publisher

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

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Can new diagnostic approaches help assess brain function in unconscious, brain-injured patients?

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Hematoxylin and eosin (H&E) staining of sections of wild-type (top row) and H1 triple-knockout (bottom row) embryoid bodies. After 14 days in rotary suspension culture, the wild-type embryoid bodies showed more differentiated morphologies with cysts forming (black arrows) and the knockout embryoid bodies failed to form cavities (far right). (Credit: Yuhong Fan)

(Phys.org) -- New research findings show that embryonic stem cells unable to fully compact the DNA inside them cannot complete their primary task: differentiation into specific cell types that give rise to the various types of tissues and structures in the body.

Researchers from the Georgia Institute of Technology and Emory University found that chromatin compaction is required for proper embryonic stem cell differentiation to occur. Chromatin, which is composed of histone proteins and DNA, packages DNA into a smaller volume so that it fits inside a cell.

A study published on May 10, 2012 in the journal PLoS Genetics found that embryonic stem cells lacking several histone H1 subtypes and exhibiting reduced chromatin compaction suffered from impaired differentiation under multiple scenarios and demonstrated inefficiency in silencing genes that must be suppressed to induce differentiation.

While researchers have observed that embryonic stem cells exhibit a relaxed, open chromatin structure and differentiated cells exhibit a compact chromatin structure, our study is the first to show that this compaction is not a mere consequence of the differentiation process but is instead a necessity for differentiation to proceed normally, said Yuhong Fan, an assistant professor in the Georgia Tech School of Biology.

Fan and Todd McDevitt, an associate professor in the Wallace H. Coulter Department of Biomedical Engineering at Georgia Tech and Emory University, led the study with assistance from Georgia Tech graduate students Yunzhe Zhang and Kaixiang Cao, research technician Marissa Cooke, and postdoctoral fellow Shiraj Panjwani.

The work was supported by the National Institutes of Healths National Institute of General Medical Sciences (NIGMS), the National Science Foundation, a Georgia Cancer Coalition Distinguished Scholar Award, and a Johnson & Johnson/Georgia Tech Healthcare Innovation Award.

Enlarge

Phase contrast images showing that H1 triple-knockout (bottom) embryonic stem cells were unable to adequately form neurites and neural networks compared to wild-type embryonic stem cells (top). (Click image for high-resolution version. Credit: Yuhong Fan)

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Genetic packing: Successful stem cell differentiation requires DNA compaction, study finds

Recommendation and review posted by Bethany Smith

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

Contact: Nicole Davis ndavis@broadinstitute.org 617-714-7152 Broad Institute of MIT and Harvard

Melanoma the deadliest and most aggressive form of skin cancer has long been linked to time spent in the sun. Now a team led by scientists from the Broad Institute and Dana-Farber Cancer Institute has sequenced the whole genomes of 25 metastatic melanoma tumors, confirming the role of chronic sun exposure and revealing new genetic changes important in tumor formation.

In an article published online May 9 in Nature, the authors provide the first high-resolution view of the genomic landscape of human melanoma tumors. Previous genetic analyses have focused on the exomes of many types of cancer tumors, concentrating on the tiny fraction of the genome that provides the genetic code for producing proteins. Whole genomes contain a wealth of genetic information, and by sequencing and analyzing 25 metastatic melanoma tumors a significant technical and computational feat scientists can learn vastly more about the variety of genetic alterations that matter in melanoma.

"Sequencing the whole genome certainly adds a richness of discovery that can't be fully captured with a whole exome," said Levi A. Garraway, a senior associate member of the Broad Institute, an associate professor at Dana-Farber Cancer Institute and Harvard Medical School, and co-senior author of the paper.

"By looking across the entire genome you can more accurately determine the background mutation rate and the different classes of mutations, and more confidently describe the pattern of ultraviolet-induced mutagenesis in melanoma," said Michael F. Berger, co-first author of the paper. He worked in the Broad's cancer genome analysis group and with Garraway as a research scientist and computational biologist before moving to Memorial Sloan-Kettering Cancer Center.

When the scientists explored the whole genome data generated and analyzed at the Broad, they found that the rates of genetic mutations rose along with chronic sun exposure in patients, confirming the role of sun damage in disease development.

"Whole-genome analysis of human melanoma tumors shows for the first time the existence of many structural rearrangements in this tumor type," said Lynda Chin, a senior associate member of the Broad and co-senior author of the paper. Formerly at Dana-Farber and Harvard Medical School, she is now chair of the Department of Genomic Medicine at the University of Texas MD Anderson Cancer Center.

As expected, the scientists detected known BRAF and NRAS mutations in 24 of the 25 tumors. Both genes are involved in sending signals important in cell growth.

One other gene leaped out: PREX2, previously implicated in breast cancer for blocking a tumor-suppressor pathway, was altered in 44 percent of patients. In a larger validation cohort of 107 tumors, the frequency of the mutation was 14 percent.

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New under the sun: Recurrent genetic mutations in melanoma

Recommendation and review posted by Bethany Smith

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

Contact: Dr. Hilary Glover hilary.glover@biomedcentral.com 44-020-319-22370 BioMed Central

Chemotherapy is a major first line defense against breast cancer. However a patient's response is often variable and unpredictable. A study published in BioMed Central's open access journal BMC Medical Genomics shows that 'gene expression signatures' for TOP2A and -tubulin can be used to predict the outcome of chemotherapy.

The goal of personalized medicine in cancer treatment is to target therapy to the characteristics of the individual tumor. For example Herceptin treatment is of most benefit to patients whose cancer is driven by HER2 and antiestrogens benefit patients whose breast cancer is hormonally driven. Gene signatures are increasingly available for different cancer types and can be used to predict patient prognosis.

Researchers from McMaster University, in association with the Juravinski Hospital and Cancer Center, analyzed the expression of the enzyme TOP2A (DNA topoisomerase) and -tubulin, which are the targets of commonly used chemotherapy drugs (anthracycline and taxane) in hundreds of breast tumors. Combining the results from the tumor samples analyses allowed the researchers to build gene expression 'signatures' that measure the susceptibility of tumor cells to chemotherapy.

Both of the 'signatures' were separately able to predict which patients achieved a complete response (where invasive or metastatic cancer could no longer be detected) and together the two indices together were even more accurate at predicting response to chemotherapy.

Prof John Hassell, who led this study, commented, " Our results clearly demonstrate the practicality of using gene expression to personalize chemotherapy treatment for breast cancer patients. Identifying patients who will not benefit from a specific treatment means that they can be moved to a different treatment plan, and the earlier appropriate treatment is started the more likely it is that the patients will benefit from it."

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Media Contact Dr Hilary Glover Scientific Press Officer, BioMed Central Tel: +44 (0) 20 3192 2370 Mob: +44 (0) 778 698 1967 Email: hilary.glover@biomedcentral.com

Notes to Editors

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Genetic predictor of breast cancer response to chemotherapy

Recommendation and review posted by Bethany Smith

By Sean Williams | More Articles May 9, 2012 |

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

What: Shares of biotech company Seattle Genetics (Nasdaq: SGEN) have had a wild ride this morning following its first-quarter earnings results. After being down 10% early in the trading session, shares are up more than 2% as of this writing.

So what: For the quarter, Seattle Genetics reported a near-quadrupling in sales to $48.2 million as the company sold $34.5 million worth of its cancer drug Adcetris, which was approved by the Food and Drug Administration in August for the treatment of Hodgkin's lymphoma and systemic anaplastic large cell lymphoma. Its quarterly loss came in at $0.11 per share. Although the loss met Wall Street's estimates, sales estimates for Adcetris fell about $3 million shy of the consensus. For the year, Seattle Genetics forecast Adcetris sales of $140 million-$150 million and collaboration/licensing revenue of $55 million-$65 million.

Now what: Despite the sales miss, RBC Capital told investors this morning that any dip in Seattle Genetics shares is a buying opportunity, as it expects the company to report positive data at the ASCO conference. That may be partly responsible for the strength in its share price since it opened significantly lower this morning. As for me, I see Seattle Genetics as largely range-bound until it can produce that elusive quarterly profit.

Craving more input? Start by adding Seattle Genetics to your free and personalized watchlist so you can keep up on the latest news with the company.

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Why Seattle Genetics Shares Dropped Then Rallied

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LOS ANGELES--(BUSINESS WIRE)--

Response Genetics Inc. (Nasdaq:RGDX - News), (the Company), a company focused on the development and sale of molecular diagnostic tests for cancer, today announced its consolidated financial results for the first quarter ended March 31, 2012.

Total revenues for the quarter ended March 31, 2012 were $4.0 million, compared to $5.9 million for the quarter ended March 31, 2011 was largely as a result of the expected decrease in pharmaceutical client revenue. The Companys ResponseDX revenue decreased slightly to $3.0 million for the quarter ended March 31, 2012, compared to $3.1 million for the quarter ended March 31, 2011.

The Companys first quarter 2012 net loss increased compared to the first quarter of 2011, while the sequential net loss decreased for the quarter ended March 31, 2012. Net loss for the quarter ended March 31, 2012 was $3.1 million, compared with a net loss of $0.3 million for the quarter ended March 31, 2011 and net loss of $3.9 million for the quarter ended December 31, 2011.

Cash and cash equivalents at March 31, 2012, were $5.7 million, compared to $1.7 million at December 31, 2011.

In the first quarter of 2012 we accomplished what needed to be done first we strengthened our balance sheet when we raised $7.8 million of cash, said Thomas Bologna, the Companys recently appointed Chairman & Chief Executive Officer. We also began implementing programs to put our company on the right track and took action to recover from the transition that the Company faced in 201l. While we appreciate that it will take time to achieve the results that we fully expect to deliver to our shareholders, we believe we are making good progress on what needs to be done and putting the team in place to make it happen. We believe our company is in the right space and we have the foundation and wherewithal to capitalize on our strengths and the opportunities in front of us.

Excluding cost of revenue, total operating expenses for the first quarter were $4.4 million, compared to $3.5 million for the same period last year. The increase in total operating expenses of $0.9 million was due to an increase in general and administrative expenses of $0.5 million and research and development expenses of $0.4 million.

The increase in general and administrative expenses was a result of an increase of $0.2 million in stock based compensation expense, $0.1 million in bad debt expense, $0.1 million in consulting expense, $0.1 million of equipment/maintenance expense and $0.1 million in legal fees, offset by a decrease in business tax of $0.1 million.

The increase in research and development expenses was due to an increase in personnel expenses and associated laboratory supplies and reagents related to increased research and development activity.

CONFERENCE CALL DETAILS

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Response Genetics, Inc. Announces First Quarter Financial Results

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BOTHELL, Wash.--(BUSINESS WIRE)--

Seattle Genetics, Inc. (Nasdaq: SGEN - News) today announced that interim results from an investigator-sponsored phase II clinical trial of ADCETRIS (brentuximab vedotin) in patients with relapsed cutaneous T-cell lymphoma (CTCL) were presented at the Society for Investigative Dermatology annual meeting being held May 9-12, 2012 in Raleigh, NC. ADCETRIS is an antibody-drug conjugate (ADC) directed to CD30. ADCETRIS has not been approved for use in CTCL.

The trial enrolled CTCL patients with mycosis fungoides (MF) or Sezary syndrome. At the time of data analysis, 17 patients had been enrolled, including 16 with MF and one with Sezary syndrome. Patients had received a median of six prior therapies, including a median of four prior systemic therapies. The primary endpoint of the trial is clinical response rate. Secondary endpoints include correlation of clinical response with CD30 expression levels, duration of response, progression-free survival and safety. The study is led by principal investigator Dr. Youn H. Kim, Professor, Department of Dermatology, and Director, Multidisciplinary Cutaneous Lymphoma Program at Stanford University School of Medicine in Stanford, CA. Key findings include:

This is the second data set reported with ADCETRIS in CTCL patients. At the T-Cell Lymphoma Forum in January 2012, interim data were presented from a phase II investigator-sponsored trial in CD30-positive CTCL patients, including lymphomatoid papulosis, primary cutaneous anaplastic large cell lymphoma (pcALCL) or MF. In the trial, which is being conducted by Dr. Madeleine Duvic at The University of Texas MD Anderson Cancer Center, 11 of 17 evaluable patients (65 percent) achieved an objective response, including seven complete remissions (CRs) and four partial remissions (PRs). The most common adverse events were Grade 1, including diarrhea, chest tightness, alopecia, nausea, elevated liver enzymes and peripheral neuropathy.

Seattle Genetics and Millennium: The Takeda Oncology Company recently initiated a randomized phase III clinical trial of ADCETRIS for relapsed CD30-positive CTCL patients. The trial will assess ADCETRIS versus investigators choice of methotrexate or bexarotene in patients with CD30-positive CTCL, including those with pcALCL or MF. The primary endpoint of the study is overall response rate lasting at least 4 months. Approximately 124 patients will be enrolled in the pivotal trial. The phase III trial is being conducted under a Special Protocol Assessment agreement from the U.S. Food and Drug Administration (FDA). The study also received European Medicines Agency scientific advice.

About CTCL

Lymphoma is a general term for a group of cancers that originate in the lymphatic system. There are two major categories of lymphoma: Hodgkin lymphoma and non-Hodgkin lymphoma. Cutaneous lymphomas are a category of non-Hodgkin lymphomas that primarily involve the skin. According to the Cutaneous Lymphoma Foundation, CTCL is the most common type of cutaneous lymphoma and typically presents with red, scaly patches or thickened plaques of skin that often mimic eczema or chronic dermatitis. Progression from limited skin involvement is variable and may be accompanied by tumor formation, ulceration and exfoliation, complicated by itching and infections. Advanced stages are defined by involvement of lymph nodes, peripheral blood and internal organs. According to published literature, up to 50 percent of CTCL patients lesions express CD30.

About ADCETRIS

ADCETRIS (brentuximab vedotin) is an ADC comprising an anti-CD30 monoclonal antibody attached by a protease-cleavable linker to a microtubule disrupting agent, monomethyl auristatin E (MMAE), utilizing Seattle Genetics proprietary technology. The ADC employs a linker system that is designed to be stable in the bloodstream but to release MMAE upon internalization into CD30-expressing tumor cells.

ADCETRIS (brentuximab vedotin) received accelerated approval from the U.S. Food and Drug Administration for two indications: (1) the treatment of patients with Hodgkin lymphoma after failure of autologous stem cell transplant (ASCT) or after failure of at least two prior multi-agent chemotherapy regimens in patients who are not ASCT candidates, and (2) the treatment of patients with systemic anaplastic large cell lymphoma (sALCL) after failure of at least one prior multi-agent chemotherapy regimen. The indications for ADCETRIS are based on response rate. There are no data available demonstrating improvement in patient-reported outcomes or survival with ADCETRIS.

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Seattle Genetics Announces Data from Investigator Trial of ADCETRIS™ in Relapsed Cutaneous T-Cell Lymphoma

Recommendation and review posted by Bethany Smith

AMES, Iowa, May 10, 2012 (GLOBE NEWSWIRE) -- NewLink Genetics Corporation (Nasdaq:NLNK - News), a biopharmaceutical company focused on discovering, developing and commercializing cancer therapeutics, today reported consolidated financial results for the first quarter of 2012, and provided an update on the progress of its clinical development programs.

"During the first quarter of 2012 we continued to advance our pivotal trial in pancreatic cancer and are also moving forward on a number of other HyperAcute immunotherapy development programs including those to treat melanoma and lung and kidney cancers," commented Dr. Charles Link, Chairman and Chief Executive Officer of NewLink. "We also made strides in our IDO pathway inhibitor program and are looking forward to presenting new data for a number of these programs at both DDW and ASCO. We believe that the receipt of patent allowances in both of our core programs will also facilitate our business development activities."

First quarter 2012 Financial Results

Financial Guidance

NewLink is maintaining its financial guidance and continues to expect to end 2012 with about $20 million in cash, cash equivalents and marketable securities. NewLink continues to anticipate that this capital should allow it to fund its operations through 2013 based on its current operating plans.

2012 Key Accomplishments to Date

Upcoming Activities

NewLink expects to present at the following investor conferences:

NewLink expects to present at the following oncology and pharmacology meetings:

About NewLink Genetics Corporation

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NewLink Genetics Corporation Reports First Quarter 2012 Financial Results

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