Category Archives: Genetic Therapy

NDTV

Yeddy praises Sonia and Cong Times Now.tv The factional feud in Karnataka's ruling BJP touched a new low on Sunday (May 13) with former Karnataka chief minister BS Yeddyurappa calling his successor Sadananda Gowda a "betrayer" as he lavished praise on Sonia Gandhi and the Congress. Eswarappa holds talks with Yeddy, says crisis will be resolvedBusiness Standard Karnataka crisis: Gowda in Delhi, to meet BJP high commandIBNLive.com BJP calls feuding Karnataka factions for a meetRediff Newstrack India -NDTV -Deccan Herald all 221 news articles »

Source:
http://news.google.co.in/news?pz=1&ned=in&hl=en&q=Genetic+Therapy&output=rss

The Hindu

Supremacy of Parliament must be preserved: Leaders Daily News & Analysis Place: New Delhi | Agency: PTI Concern over repeated disruptions that have raised questions over the efficacy of Parliament's functioning today marked the 60th anniversary of its first sitting in free India with leaders pressing for "sincere" ... PM for serious introspection on Parliament functioningHindustan Times Parliament resolves to uphold, maintain dignityTimes of India Make 100 sittings a year mandatory, demand MPsZee News India Today -Rediff all 346 news articles »

Source:
http://news.google.co.in/news?pz=1&ned=in&hl=en&q=Genetic+Therapy&output=rss

IBNLive.com

Nobody expects mid-term poll: Prakash Karat Daily News & Analysis CPI(M) General Secretary Prakash Karat on Sunday said that nobody is expecting mid-term Lok Sabha elections, reacting to Trinamool Congress chief Mamata Banerjee's comment that there were reports that general elections were imminent. "I don't know. Nobody expecting early elections: KaratBusiness Standard TMC union celebrates May Day despite MamataMSN India 'Queen of Democrazy' Mamata Banerjee at the crossroads of changeTimes of India Reuters Canada all 356 news articles »

Source:
http://news.google.co.in/news?pz=1&ned=in&hl=en&q=Genetic+Therapy&output=rss

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

Contact: Krista Conger kristac@stanford.edu 650-725-5371 Stanford University Medical Center

STANFORD, Calif. The cells that slough off from a cancerous tumor into the bloodstream are a genetically diverse bunch, Stanford University School of Medicine researchers have found. Some have genes turned on that give them the potential to lodge themselves in new places, helping a cancer spread between organs. Others have completely different patterns of gene expression and might be more benign, or less likely to survive in a new tissue. Some cells may even express genes that could predict their response to a specific therapy. Even within one patient, the tumor cells that make it into circulating blood vary drastically.

The finding underscores how multiple types of treatment may be required to cure what appears outwardly as a single type of cancer, the researchers say. And it hints that the current cell-line models of human cancers, which showed patterns that differed from the tumor cells shed from human patients, need to be improved upon.

The new study, which will be published online May 7 in PLoS ONE, is the first to look at so-called circulating tumor cells one by one, rather than taking the average of many of the cells. And it's the first to show the extent of the genetic differences between such cells.

"Within a single blood draw from a single patient, we're seeing heterogeneous populations of circulating tumor cells," said senior study author Stefanie Jeffrey, MD, professor of surgery and chief of surgical oncology research.

For over a century, scientists have known that circulating tumor cells, or CTCs, are shed from tumors and move through the bloodstreams of cancer patients. And over the past five years, there's been a growing sense among many cancer researchers that these cells accessible by a quick blood draw could be the key to tracking tumors non-invasively. But separating CTCs from blood cells is hard; there can be as few as one or two CTCs in every milliliter of a person's blood, mixed among billions of other blood cells.

To make their latest discovery, Jeffrey, along with an interdisciplinary team of engineers, quantitative biologists, genome scientists and clinicians, relied on a technology they developed in 2008. Called the MagSweeper, it's a device that lets them isolate live CTCs with very high purity from patient blood samples, based on the presence of a particular protein EpCAM that's on the surface of cancer cells but not healthy blood cells.

With the goal of studying CTCs from breast cancer patients, the team first tested whether they could accurately detect the expression levels of 95 different genes in single cells from seven different cell-line models of breast cancer a proof of principle since they already knew the genetics of these tumors. These included four cell lines generally used by breast cancer researchers and pharmaceutical scientists worldwide and three cell lines specially generated from patients' primary tumors.

"Most researchers look at just a few genes or proteins at a time in CTCs, usually by adding fluorescent antibodies to their samples consisting of many cells," said Jeffrey. "We wanted to measure the expression of 95 genes at once and didn't want to pool our cells together, so that we could detect differences between individual tumor cells."

Link:
Not all tumor cells are equal: Stanford study reveals huge genetic diversity in cells shed by tumors

OSLO, Norway--(BUSINESS WIRE)--

Regulatory News:

DiaGenic ASA (OSE:DIAG.OL - News): DiaGenic today reports on the finalization of data collection and database lock of a blinded study in a Norwegian cohort of 80 patients with familial Parkinsons disease (PD). The majority of these patients are carrying a mutation in the parkin 8 gene (also called LRRK2) that significantly increases the risk of developing PD. Patients recruited from St Olavs University Hospital under the lead of Principal Investigator Professor Jan Aasly are all LRRK2 mutation carriers with or without the disease or healthy relatives. Unblinding of the study is set to the May 16th and analysis and reporting is expected to be completed during summer 2012.

As part of the overarching program on development of diagnostic biomarkers for PD, the objective of the study is to identify a biomarker for individuals at risk of developing the disease using material from LRRK2 individuals and relatives. The LRRK2 study is unique being the first blood based RNA analysis in pre symptomatic PD. The study is also expected to allow independent validation of DiaGenics European multicentre study that previously this year reported a high accuracy (88%) in PD patients reported February 8th 2012.

The collection of data from the Norwegian individuals with this rare mutation has been ongoing at St. Olav`s University Hospital in Trondheim, Norway, since 2009 and includes collection of blood for gene signature analysis, clinical variables and imaging of the brain. By taking blood samples from individuals with LRRK2 mutations and identifying a gene signature before signs of the disease occur, a general blood based diagnostic test for early PD can be developed.

The gene analyses of 96 samples including technical controls and samples of 82 participants from families with a LRRK2 gene mutation are performed on an Illumina whole genome platform and aims to identify disease related gene probes (i.e. gene transcripts) for disease specific diagnostic models. The gene analyses were completed May 7th and the study now enters into the phase of final bioinformatical analysis.

Professor Jan Aasly, Dept Neurology, St Olavs University Hospital, Trondheim, Norway comments: This unique study represents an opportunity to make new scientific discoveries in Parkinson`s Disease. Should we be able to identify a gene signature that is present before the onset of neurological signs in PD, in this form of familial PD, then we may be one step closer to identifying a diagnostic test for pre symptomatic PD.

DiaGenic CEO Dr Henrik Lund said: We are very pleased collaborate with St. Olavs University Hospital and Professor Aasly. We are especially grateful to the families that are committed to support scientific advancements in PD. With their support we are in a good position to start identifying novel biomarkers that in DiaGenics product development programs can mature into diagnostic tools to support early diagnosis and development of new drugs to treat PD. DiaGenic is committed to develop novel diagnostic tools for severe disorders, especially in PD and Alzheimers disease.

About PD

Parkinsons disease (PD) is the second most common neurodegenerative disorder after Alzheimers disease with more than 5 million patients worldwide, whereas 1 million are in the US. PD is a disorder of the central nervous system that results from the loss of cells in various parts of the brain. No cure is yet available, but several pharmaceutical companies are currently developing medicines many of which target disease modification in PD. There is no objective test for Parkinson's, so the rate of misdiagnosis can be relatively high, especially when the diagnosis is made by a non-specialist at an early stage of the disease.

Originally posted here:
DiaGenic ASA: DiaGenic Reports Completion of Data Collection and Genetic Analyses in a Unique Study on Familial ...

Updated with stock price, analyst reaction. CAMBRIDGE, Mass. (TheStreet) -- Vertex Pharmaceuticals(VRTX) released preliminary data Monday showing two drugs -- the experimental VX-809 and currently marketed Kalydeco -- significantly improved lung function in patients with the most common genetic mutation causing cystic fibrosis. The interim analysis announced by Vertex is limited in scope, covering only about half of the cystic fibrosis patients who completed treatment in the phase II study to date, and then only those patients responding to treatment with VX-809 and Kalydeco compared to a placebo. The study results are also a bit confounding because improvements in lung function were not matched by significant reductions of salt in the sweat of patients treated with the two drugs compared to placebo. So-called sweat chloride is thought to be a laboratory marker for the underlying cause of cystic fibrosis. Complete results from the phase II study are expected later this summer. Nonetheless, Vertex said improvements in lung function observed so far were strong enough to warrant moving ahead with a pivotal phase III study of VX-809 and Kalydeco in cystic fibrosis patients with two copies (homozygous) of the F508del gene. The company will begin discussions soon with the FDA and European regulators about the specific designs for the phase III trial. Vertex stands to grow its nascent cystic fibrosis business substantially if a VX-809-Kalydeco combination therapy is ultimately approved since about half of the 70,000 cystic fibrosis patients worldwide carry two copies of the F508del gene. Kalydeco was approved in January as a single-drug therapy for about 4% of cystic fibrosis patients carrying the G551D genetic mutation. Vertex shares jumped 44% to $53.88 in early Monday trading. ISI Group analyst Mark Schoenebaum upgraded Vertex to buy with a $68-per-share price target, estimating the company's cystic fibrosis sales could reach $4 billion annually. Monday's results are based on an interim analysis of 37 homozygous F508del patients treated with three different dosing regimens of VX-809 and Kalydeco for 56 days compared to 11 patients with one or two copies of the F508del mutation who received placebo for 56 days. The entire study enrolled 108 patients. A statistically significant improvement in lung function was observed across the VX-809-Kalydeco treatment groups relative to baseline and compared to placebo, Vertex said. Of those patients treated with the two-drug therapy, 46% (17 of 37 patients) experienced an absolute improvement in lung function of 5% or more. A subset of 11 patients, or 30%, saw their lung function rise by at least 10%. By comparison, none of the 11 patients in the placebo arm of the study achieved a 5% improvement in lung function. Lung function was assessed by FEV1, which measures the amount of air a patient can forcibly exhale in one second. Patients' lung function was measured at the start of the trial and then compared to measurement taken after 56 days of treatment. FEV1 is the measure of clinical benefit accepted by FDA and European regulators for the approval of new cystic fibrosis drugs. Vertex did not disclose Monday the mean absolute improvement in lung function for all VX-809-Kalydeco patients compared to placebo, nor did the company break out lung function improvements by the three doses of VX-809 used in the phase II trial. However, patients in all three of the dose groups responded to treatment with VX-809 and Kalydeco, and the statistically significant improvement in lung function overall was not driven by a relatively small number of "super responders," said Eric Olson, Vertex's vice president for cystic fibrosis franchise, in an interview Sunday morning. Analysis was also conducted on a group of 21 cystic fibrosis patients with a single copy (heterozygous) of the F508del gene. Vertex did not disclose the results from this group, however, because not enough patients reached the end of the 56-day treatment period. Vertex officials are at a loss to explain why the combination of VX-809 and Kalydeco did not result in a statistically significant reduction in sweat chloride levels between Day 28 and Day 56 compared to placebo, one of two co-primary endpoints of the phase II study. [The second primary endpoint was safety; the drugs were generally well tolerated, Vertex said.] Investors watching and handicapping results of the phase II study expected the VX-809-Kalydeco combination to demonstrate a statistically significant, 15-20% reduction in sweat chloride levels with a corresponding, smaller, non-statistical trend toward better lung function. The interim results announced Monday turned these expectations on their head -- a statistically significant improvement in lung function but only a non-statistical trend towards lower sweat chloride. "I think we need to wait for the final study results but it seems as if we're still learning about sweat chloride as a biomarker in cystic fibrosis and whether it's truly correlated with lung function," said Vertex's Olson. Olson adds that from a regulatory perspective, improvements in lung function is what matters most, and it was the significant improvement in lung function observed which prompted Vertex to publicly disclose the interim results. Another intriguing finding from the interim analysis, sweat chloride levels were reduced by a statistically significant amount from the start of the trial through Day 28 when patients were treated with VX-809 alone. This raises the question of whether the addition of Kalydeco to the treatment of these patients is necessary or perhaps even detrimental. Olson believes patients more likely benefit from both drugs but Vertex plans to examine closely the final data from the study before making any judgments, Olson says. Cystic fibrosis is caused by genetic mutations that result in a malfunctioning or missing protein known as CFTR required for the regulation of sweat production, mucus and certain aspects of digestion. Defective or missing CFTR proteins in lung cells results in the formation of thick, sticky mucus that leads to restricted airflow, chronic infections and lung damage. Patients with the most common F508del mutation have missing or insufficient CFTR protein on the surface of cells. VX-809 is designed as a "corrector" drug that increases the amount of CFTR protein on the cell surface. Kalydeco, on the other hand, is a "potentiator" designed to improve the function of the damaged CFTR proteins. Kalydeco was approved in January for cystic fibrosis patients with the G551D mutation. These patients have sufficient CFTR proteins on the surface of cells but the proteins are damaged and don't work correctly. The phase II combination study treated cystic fibrosis patients with the F508del mutation with four weeks of VX-809 alone to increase the amount of CFTR protein, followed by another four weeks of VX-809 combined with Kalydeco to improve the function of the new CFTR protein. Three doses of VX-809 were studied along with a higher dose of Kalydeco. --Written by Adam Feuerstein in Boston. >To contact the writer of this article, click here: Adam Feuerstein. >To follow the writer on Twitter, go to http://twitter.com/adamfeuerstein. >To submit a news tip, send an email to: tips@thestreet.com. Follow TheStreet on Twitter and become a fan on Facebook.>To order reprints of this article, click here: Reprints

Excerpt from:
Vertex: Two-Drug Cystic Fibrosis Therapy Improves Lung Function