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New genetic subtype of lung cancer defined

ScienceDaily (Jan. 31, 2012) — A report from investigators at the Massachusetts General Hospital (MGH) Cancer Center has defined the role of a recently identified gene abnormality in a deadly form of lung cancer. Tumors driven by rearrangements in the ROS1 gene represent 1 to 2 percent of non-small-cell lung cancers (NSCLC), the leading cause of cancer death in the U.S. The researchers show that ROS1-driven tumors can be treated with crizotinib, which also inhibits the growth of tumors driven by an oncogene called ALK, and describe the remarkable response of one patient to crizotinib treatment.

"ROS1 encodes a protein that is important for cell growth and survival, and deregulation of ROS1 through chromosomal rearrangement drives the growth of tumors," says Alice Shaw, MD, PhD, of the MGH Cancer Center -- co-lead author of the paper which has been published online in the Journal of Clinical Oncology. "This finding is important because we have drugs that inhibit ROS1 and could lead to the sort of dramatic clinical response we describe in this paper."

The current findings add ROS1 to the list of genes known to drive NSCLC growth when altered -- a list that includes KRAS, mutations of which account for about 25 percent of cases; EGFR, accounting for 10 to 15 percent; and ALK, rearranged in about 4 percent. Altogether, known cancer-causing genetic changes have been found in a little more than half of NSCLC tumors. Originally identified in brain tumors, ROS1 rearrangement previously had been identified in one NSCLC patient and one NSCLC cell line. The current study was designed to determine the frequency of ROS1 rearrangement in NSCLC and to define the characteristics of patients with ROS1-rearranged tumors.

The investigators screened tumor samples from more than 1,000 NSCLC patients treated at the MGH, Vanderbilt University, the University of California at Irvine, and Fudan University in Shanghai, China. ROS1 rearrangement was identified in 18 tumor samples, for a prevalence of 1.7 percent; ALK rearrangements were identified in 31 samples, with no samples showing alterations in both genes. Patients with ROS1-positive tumors tended to be younger, never to have smoked and to have a type of lung cancer called adenocarcinoma -- characteristics very similar to those of ALK-positive patients.

An earlier MGH study of an experimental ALK inhibitor had found the drug suppressed the growth of a ROS1-positive cell line in addition to ALK-positive cell lines, suggesting that ROS1-positive tumors might be sensitive to the ALK-inhibitor crizotinib. This observation led corresponding author John Iafrate, MD, PhD, and his team to develop a diagnostic test that could identify ROS1-positive tumors. Around the time that test became clinically available, a lung cancer patient whose tumor had not responded to drugs targeting EGFR mutations was referred to the MGH Cancer Center for genetic testing. His tumor was negative for ALK but later proved to harbor a ROS1 rearrangement, and he was enrolled in an extension of the crizotinib clinical trial first reported in the October 28, 2010, New England Journal of Medicine.

"When he enrolled in the trial last April, this patient was extremely sick -- with significant weight loss and very low oxygen levels -- and was barely able to walk," says Shaw. "Within a few days of starting crizotinib, he felt better; and by the time we scanned his chest at seven weeks, the tumors had essentially disappeared from his lungs." Nine months after starting crizotinib therapy, this patient continues to do well. Additional ROS1-positive patients have been enrolled in this trial at MGH, at UC Irvine and at the University of Colorado.

Shaw is an assistant professor of Medicine and Iafrate is an associate professor of Pathology at Harvard Medical School. Co-lead authors are Kristin Bergethon, MGH Pathology, and Sai-Hong Ignatius Ou, MD, PhD, University of California at Irvine. The study was supported by grants from the National Institutes of Health and from Pfizer, which received FDA approval for crizotinib in August 2011.

Additional co-authors are Ryohei Katayama, Eugene Mark, Julie Batten, Eunice Kwak, Jeffrey Clark, Jeffrey Engelman, and Mari Mino Kenudson, MGH Cancer Center; Christina Siwak-Tapp, University of California at Irvine; Keith D. Wilner, Pfizer; Christine Lovly, Nerina McDonald, Pierre Massion, Adriana Gonzalez, David Carbone, and William Pao, Vanderbilt University Medical Center; Pierre Massion, Nashville Veterans Affairs Medical Center; Rong Fang and Hongbin Ji, Shanghai Institutes for Biological Sciences; and Haiquan Chen, Shanghai Medical College, Fudan University.

Massachusetts General Hospital, founded in 1811, is the original and largest teaching hospital of Harvard Medical School. The MGH conducts the largest hospital-based research program in the United States, with an annual research budget of more than $750 million and major research centers in AIDS, cardiovascular research, cancer, computational and integrative biology, cutaneous biology, human genetics, medical imaging, neurodegenerative disorders, regenerative medicine, reproductive biology, systems biology, transplantation biology and photomedicine.

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The above story is reprinted from materials provided by Massachusetts General Hospital.

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Journal Reference:

K. Bergethon, A. T. Shaw, S.-H. Ignatius Ou, R. Katayama, C. M. Lovly, N. T. McDonald, P. P. Massion, C. Siwak-Tapp, A. Gonzalez, R. Fang, E. J. Mark, J. M. Batten, H. Chen, K. D. Wilner, E. L. Kwak, J. W. Clark, D. P. Carbone, H. Ji, J. A. Engelman, M. Mino-Kenudson, W. Pao, A. J. Iafrate. ROS1 Rearrangements Define a Unique Molecular Class of Lung Cancers. Journal of Clinical Oncology, 2012; DOI: 10.1200/JCO.2011.35.6345

Note: If no author is given, the source is cited instead.

Disclaimer: This article is not intended to provide medical advice, diagnosis or treatment. Views expressed here do not necessarily reflect those of ScienceDaily or its staff.

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New genetic subtype of lung cancer defined

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Myriad Genetics Reports Second Quarter Fiscal Year 2012 Results

SALT LAKE CITY, Jan. 31, 2012 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (Nasdaq:MYGN - News) today announced results for its second fiscal quarter and six months ending December 31, 2011. Revenue for the second fiscal quarter was $122.8 million, an increase of 22 percent over the $100.4 million reported in the second fiscal quarter of 2011. Earnings per diluted share were $0.33, an increase of 27 percent over the same period of the prior year.

"These results represent the second quarter in a row of 20 percent or greater year-over-year revenue growth," said Peter D. Meldrum, President and Chief Executive Officer of Myriad Genetics, Inc. "As a result, I'm pleased to announce increased guidance for fiscal 2012. We remain focused on delivering strong top-line growth and implementing our broader strategic plan to diversify revenue across multiple disease indications and geographies."

Analysis of Second Fiscal Quarter 2012

Molecular diagnostic testing revenue in the second fiscal quarter equaled $117.6 million, an increase of 17 percent compared to the prior year period. This increase was driven by strong growth across all segments and products. Oncology revenue equaled $79.8 million, an increase of 15 percent over the second quarter of 2011. Women's Health revenue totaled $37.9 million, an increase of 22 percent over the same period in the prior year. Revenue from the BRACAnalysis(R) test, which represented 82.6 percent of total revenue in the second quarter, was $101.4 million, a 14 percent increase over the same period of the prior year. Revenue from the COLARIS(R) and COLARIS AP(R) tests, which represented 8.9 percent of total revenue during the quarter, was $10.9 million, an increase of 56 percent compared to the same fiscal quarter of the prior year. Myriad's remaining molecular diagnostic tests contributed $5.3 million to second quarter revenue, an increase of 24 percent over the same period in the prior year, and accounted for 4.3 percent of total revenue. Companion diagnostic service revenue in the second fiscal quarter equaled $5.2 million and represented 4.2 percent of total company revenue. There is no prior year revenue, as the Company acquired this business in May 2011. Operating income was $45.5 million, an increase of 18 percent from the prior year period. This record level of operating income included the impact of a 68 percent increase in R&D investment to support the Company's existing molecular diagnostic tests and future product opportunities. Net income for the second fiscal quarter was $28.3 million, an increase of 17 percent over the $24.2 million reported in same period of the prior year. The Company repurchased 927,709 shares of its common stock during the quarter under its previously announced stock repurchase program. Diluted weighted average shares outstanding were 86.2 million in the second fiscal quarter as compared to 93.6 million in the same period of the prior year. The Company ended the quarter with $428.3 million in cash, cash equivalents and marketable investment securities. Days sales outstanding for Myriad's accounts receivable improved to 32 days, compared with 37 days in the same period of the prior year. Bad debt expense in the second fiscal quarter equaled 5.2 percent of revenue, compared with 4.2 percent in the same period of the prior year.

Year-to-Date Performance

Total revenue for the first half of fiscal 2012 was $233.3 million, an increase of 21 percent over $192.3 million reported for the half of fiscal 2011. Operating income for the first half of fiscal 2012 was $86.9 million, an increase of 17 percent year-over-year. Net income for the first half of fiscal 2012 equaled $53.4 million, compared to $46.7 million for the first half of the prior year, an increase of 14 percent. In the first half of fiscal 2012, diluted earnings per share increased 24 percent to $0.62 from $0.50 for the same period of the prior year.

Fiscal Year 2012 Outlook

The Company has increased its expectations for fiscal year 2012 financial performance. Total revenue is now expected to be $465 million to $475 million, an increase from the $445 million to $465 million previously announced. This level of revenue is expected to result in fully diluted earnings per share of $1.24 to $1.28, up from the original guidance of $1.20 to $1.25. Molecular diagnostic revenue is now expected to range between $440 million and $450 million and companion diagnostic service revenue continues to be expected to range between $24 million and $26 million. These projections are forward looking statements and are subject to the risks summarized in the safe harbor statement at the end of this press release. The Company will provide further detail on its business outlook during the conference call it is holding today to discuss its fiscal results for the second fiscal quarter and first half of fiscal 2012.

Conference Call and Webcast

A conference call will be held on Tuesday, January 31, 2012, at 4:30 p.m. Eastern time to discuss Myriad's second fiscal quarter and first half 2012 financial results and fiscal year 2012 outlook. The dial-in number for domestic callers is (888) 225-2734. International callers may dial (303) 223-2685. All callers will be asked to reference reservation number 21566441. An archived replay of the call will be available for seven days by dialing (800) 633-8284 and entering the reservation number above. The conference call will also be available through a live Webcast at http://www.myriad.com.

About Myriad Genetics

Myriad Genetics, Inc. (Nasdaq:MYGN - News) is a leading molecular diagnostic company dedicated to developing and marketing transformative tests to assess a person's risk of developing disease, guide treatment decisions and assess a patient's risk of disease progression and disease recurrence. Myriad's portfolio of nine molecular diagnostic tests are based on an understanding of the role genes play in human disease and were developed with a focus on improving an individual's decision making process for monitoring and treating disease. With fiscal year 2011 annual revenue of over $400 million and more than 1,000 employees, Myriad is working on strategic directives, including new product introductions, companion diagnostics, and international expansion, to take advantage of significant growth opportunities. For more information on how Myriad is making a difference, please visit the Company's website: http://www.myriad.com.

Myriad, the Myriad logo, BRACAnalysis, Colaris, Colaris AP, Melaris, TheraGuide, Prezeon, OnDose, Panexia and Prolaris are trademarks or registered trademarks of Myriad Genetics, Inc. in the United States and foreign countries. MYGN-F, MYGN-G

Safe Harbor Statement

This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to the Company's focus on delivering strong top-line growth and implementing its broader strategic plan to diversify revenue across multiple disease indications and geographies; the Company's investment in R&D to support its existing molecular diagnostic tests and future product opportunities; the Company's increased guidance for fiscal year 2012 under the caption "Fiscal Year 2012 Outlook;"and the Company's strategic directives under the caption "About Myriad Genetics". These "forward-looking statements" are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by forward-looking statements. These risks and uncertainties include, but are not limited to: the risk that sales and profit margins of our existing molecular diagnostic tests and companion diagnostic services may decline or will not continue to increase at historical rates; the risk that we may be unable to expand into new markets outside of the United States; the risk that we may be unable to develop or achieve commercial success for additional molecular diagnostic tests and companion diagnostic services in a timely manner, or at all; the risk that we may not successfully develop new markets for our molecular diagnostic tests and companion diagnostic services, including our ability to successfully generate revenue outside the United States; the risk that licenses to the technology underlying our molecular diagnostic tests and companion diagnostic services and any future products are terminated or cannot be maintained on satisfactory terms; risks related to delays or other problems with manufacturing our products or operating our laboratory testing facilities; risks related to public concern over genetic testing in general or our tests in particular; risks related to regulatory requirements or enforcement in the United States and foreign countries and changes in the structure of healthcare payment systems; risks related to our ability to obtain new corporate collaborations and acquire new technologies or businesses on satisfactory terms, if at all; risks related to our ability to successfully integrate and derive benefits from any technologies or businesses that we acquire; the development of competing tests and services; the risk that we or our licensors may be unable to protect the proprietary technologies underlying our tests; the risk of patent-infringement and invalidity claims or challenges of our patents; risks of new, changing and competitive technologies and regulations in the United States and internationally; and other factors discussed under the heading "Risk Factors" contained in Item 1A in our most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission, as well as any updates to those risk factors filed from time to time in our Quarterly Reports on Form 10-Q or Current Reports on Form 8-K. All information in this press release is as of the date of the release, and Myriad undertakes no duty to update this information unless required by law.

MYRIAD GENETICS, INC. AND SUBSIDIARIES CONDENSED CONSOLIDATED INCOME STATEMENTS (Unaudited)

(in thousands, except per share amounts) Three Months Ended Six Months Ended
Dec. 31, 2011 Dec. 31, 2010 Dec. 31, 2011 Dec. 31, 2010

Molecular diagnostic testing $117,610 $100,440 $221,579 $192,298 Companion diagnostic services 5,201 -- 11,684 -- Total revenue 122,811 100,440 233,263 192,298

Costs and expenses:

Cost of molecular diagnostic testing 12,815 12,046 24,115 23,058 Cost of companion diagnostic services 3,302 -- 6,364 -- Research and development expense 10,243 6,092 18,748 11,853 Selling, general, and administrative expense 50,986 43,716 97,100 83,210 Total costs and expenses 77,346 61,854 146,327 118,121

Operating income 45,465 38,586 86,936 74,177

Other income (expense):

Interest income 1,382 548 1,856 1,269 Other (64) (80) (205) (214) Total other income 1,318 468 1,651 1,055

Income before income taxes 46,783 39,054 88,587 75,232

Income tax provision (benefit) 18,487 14,863 35,193 28,503

Net income $28,296 $24,191 $53,394 $46,729

Earnings per share:

Basic $0.33 $0.26 $0.63 $0.51 Diluted $0.33 $0.26 $0.62 $0.50

Weighted average shares outstanding

Basic 84,498 91,528 84,870 92,395 Diluted 86,231 93,647 86,602 94,178

Condensed Consolidated Balance Sheets
Dec. 31, 2011 Jun. 30, 2011

(In thousands)

Cash, cash equivalents, and marketable investment securities $428,259 $417,314

Trade receivables, net 42,988 50,272

Other receivables 1,083 575

Prepaid taxes 16,569 --

Inventory, net 10,294 8,218

Prepaid expenses 3,087 2,949

Equipment and leasehold improvements, net 24,329 23,080

Note receivable 17,667 --

Other assets 8,000 --

Intangibles, net 16,265 16,715

Goodwill 56,850 56,051

Deferred tax assets 35,867 35,653

Total assets $661,258 $610,827

Accounts payable and accrued liabilities $32,612 $33,040

Deferred revenue 2,434 1,347

Uncertain tax benefits 9,448 9,648

Stockholders' equity 616,764 566,792

Total liabilities and stockholders' equity $661,258 $610,827

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Genetics Pioneer Was UConn Professor, Mentor

Arthur Chovnick, a professor at the University of Connecticut, was a pioneer in the field of genetics whose influence was felt across the field of molecular genetics and biology.

"Arthur did something that has effectively jump-started enormous strides in the genetics of higher organisms," said Hal Krider, a former professor of genetics at UConn. "He was probably the most recognized, under-honored geneticist, but people with Nobel prizes would call and ask him for advice."

Chovnick, 84, of Chaplin, died Sept. 5.

One anecdote from his life reflected Chovnick's stature in the world of genetics. When his daughter Lisa was taking a biology course, she learned about Watson and Crick, who discovered the structure of DNA, but when the home phone rang one day and a caller identified himself as Francis Crick, Lisa hung up on him. "Quit joking," she told the Nobel Prize winner the next time he called.

Later that night, Arthur Chovnick picked up the phone himself. "Hello, Francis," he said.

"People of that stature were available to Arthur all the time," said Krider. "Everybody knew him. He was very, very well known and inordinately highly regarded."

Chovnick conducted experiments on drosophila melanogaster, a relative of the humble fruit fly that, rather than being a laboratory pest, is a valuable scientific specimen used for years in genetics research. First used to study heredity, the fly is now used in the study of disease as scientists search for the genes responsible for Alzheimer's or Parkinson's or Huntington's.

Drosophila genes are nearly identical to human genes. They also reproduce very quickly, meaning mutations may be studied in weeks rather than months or years. They have only four chromosomes. Even better, no groups picket against drosophila experimentation as they do against higher-order species.

"It is easy to grow and manipulate, and they have genes like us," said Christine Rushlow, a Chovnick-trained geneticist who is a professor at New York University. "We use them as a model system to see how genes work. We share so many genes."

Chovnick, known for pioneering work in gene organization and in demonstrating the way traits cross over within a gene, could look at events that were rare and re-create them.

Chovnick, born in Brooklyn, N.Y., on Aug. 2, 1927, grew up in Queens, where he graduated from Jackson High School in 1944. He was the oldest of four children born to Fannie and Herman Chovnick, who had both emigrated from Russia. He attended Indiana University for a year before joining the U.S. Navy, where he served on a hospital ship. After he was discharged, he returned to Indiana and obtained his undergraduate degree in 1949 and his master's in 1951. He got a doctorate in genetics from Ohio State University two years later, and obtained a grant from the National Institutes of Health that continued until 1995, one of the longest continuous NIH grants.

He spent two years at the University of Connecticut doing research and teaching before going to the Cold Spring Harbor Laboratory in Long Island, first as assistant director, then as director. In 1962, he returned to UConn as a professor, where he remained until he retired in 1994. He was a fellow of the American Association for the Advancement of Science and a founding member of the Connecticut Academy of Science and Engineering.

Chovnick was revered as a mentor as well as a teacher, his colleagues said.

"He left you alone, except he would always teach or help you," Rushlow said. "He was a great analytical thinker, which he could do in his head because he was so experienced."

He helped his students design experiments that would create a certain type of drosophila — with pink eyes for example, or missing a wing — to help them create their own mutations. "You see the consequences to the fly, and what it is doing to the fly," Rushlow said.

Chovnick also did early work on cloning, providing a fly with unusual chromosomes for other scientists to study. He studied how to regulate the activity of genes.

"When things go wrong because genes are out of control, you get disease," Rowlson said. "He was at the forefront, a leader in the genetics field, and famous for the work he had done." He also understood how genes recombine and how a new DNA sequence is created with potentially new effects.

Today, as scientists intensify their search for the genetic cause of disease, Chovnick's work is significant.

"He was a seminal character in the transition from classical genetics to modern genetic cloning and gene manipulation," said Krider, his former colleague.

"He was a very careful and highly creative thinker with a keenly analytical mind," said Arthur Hilliker, a professor at York University in Toronto, who studied under and later collaborated with Chovnick.

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Silkworms may help repair damaged hearts

Although people do regularly recover from heart attacks, the heart itself never entirely "gets better." This is because cardiac muscle tissue doesn't regenerate - any that dies in the event of a heart attack will only be replaced with inactive scar tissue, and the heart's performance will be permanently compromised as a result. Scientists have responded by trying to develop heart patches made of materials that act as nanoscale scaffolds, upon which new cardiomyocytes (heart cells) can grow. Materials used for these scaffolds have included fibrin, nanofiber, gold nanowires and polymer. Now, new research is suggesting that silkworm silk may be a better choice than any of those.

For some time now, scientists from Germany's Max Planck Institute for Heart and Lung Research have been among those researching ways of growing cardiac tissue on three-dimensional scaffolds. Everything that they looked at, however, had limitations.

"Whether natural or artificial in origin, all of the tested fibers had serious disadvantages," said research group leader Felix Engel. "They were either too brittle, were attacked by the immune system or did not enable the heart muscle cells to adhere correctly to the fibers."

It turned out, however, that scientists from the Indian Institute of Technology, Kharagpur had been working on an alternative - coin-sized disks made from the cocoon of the tasar silkworm. Not only is the silk coarser than other silk fibers, making it better-suited for use as a scaffold, but its surface also contains proteins that facilitate the adhesion of cardiomyocytes. When the silk was tested at the Max Planck Institute, heart cells from rats that were seeded onto it were able to remain in communication with one another, and beat synchronously for 20 days.

Before the silk patches can ever see clinical use, however, the scientists need to figure out a safe way of procuring a sufficient amount of heart cells from the patient. Using stem cells is a possibility, although finding a way of getting those to convert into heart cells still poses a challenge.

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Harper government invests in personalized medicine

Public release date: 31-Jan-2012
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Contact: Adele Blanchard
ablanchard@cihr.gc.ca
613-946-3308
Canadian Institutes of Health Research

This press release is available in French.

Ottawa, Ontario -- The Harper Government today announced an important investment that will help Canadians in getting more effective treatments and make the healthcare system more sustainable through personalized medicine. The announcement was made by the Honourable Leona Aglukkaq, Minister of Health, and the Honourable Gary Goodyear, Minister of State for Science and Technology.

"Our Government is committed to improving the quality of life of Canadians," Minister Aglukkaq said. "The potential to understand a person's genetic makeup and the specific character of their illness in order to best determine their treatment will significantly improve the quality of life for patients and their families and may show us the way to an improved health care system and even save costs in certain circumstances."

Personalized medicine offers the potential to transform the delivery of healthcare to patients. Healthcare will evolve from a reactive "one-size-fits-all" system towards a system of predictive, preventive, and precision care. Areas in which personalized approaches are particularly promising include oncology, cardiovascular diseases, neurodegenerative diseases, psychiatric disorders, diabetes and obesity, arthritis, pain, and Alzheimer's disease. In all of these fields, and others, a personalized molecular medicine approach is expected to lead to better health outcomes, improved treatments, and reduction in toxicity due to variable or adverse drug responses. For example, cancer patients would be screened to identify those for whom chemotherapy would be ineffective. In addition to saving on the costs of expensive drug treatments, this personalized treatment would prevent a great deal of suffering, while identifying and initiating earlier treatments that would be more effective.

"I applaud Genome Canada and the CIHR for their leadership in supporting research in personalized medicine," said Minister Goodyear. "Innovative approaches like these lead to significant health benefits, enhance our knowledge within the medical arena and can be commercialized to help so many others worldwide."

###

Genome Canada is leading the landmark research competition, with significant collaboration from the Canadian Institutes of Health Research (CIHR) and the Cancer Stem Cell Consortium (CSCC). To qualify for funding, researchers must obtain matching funding that at is least equal to that provided through the competition. Matching funding is typically derived from provincial, academic, private sector or international sources.

Fact Sheet

Further information:

Cailin Rodgers
Office of the Honourable Leona Aglukkaq
Federal Minister of Health
613-957-0200

Stephanie Thomas
Special Assistant (Communications)
Office of the Honourable Gary Goodyear
Minister of State (Science and Technology)
613-960-7728

David Coulombe
Media Relations
Canadian Institutes of Health Research
613-941-4563

Marlene Orton
Director, Media Relations
Genome Canada
613-751-4460 x119
BlackBerry: 613-295-1476

The Canadian Institutes of Health Research (CIHR) is the Government of Canada's health research investment agency. CIHR's mission is to create new scientific knowledge and to enable its translation into improved health, more effective health services and products, and a strengthened Canadian health care system. Composed of 13 Institutes, CIHR provides leadership and support to more than 14,100 health researchers and trainees across Canada. http://www.cihr-irsc.gc.ca

Genome Canada is a non-profit corporation employing an innovative business model based on funding and managing large-scale, multidisciplinary, internationally peer-reviewed genomics research projects in areas such as agriculture, forestry, fisheries, the environment and human health. For more information, visit http://www.genomecanada.ca

The Cancer Stem Cell Consortium is a not-for-profit corporation that was incorporated in 2007 to coordinate an international strategy for cancer stem cell research and related translational activities. For more information, visit http://www.cancerstemcellconsortium.ca


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Harper government invests in personalized medicine

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Bionest and PMC Address Optimization of Personalized Medicine Strategies

NEW YORK & PARIS--(BUSINESS WIRE)-- Bionest Partners, a premier strategy and management consulting firm for life science industries, and a recognized leader in personalized medicine strategy consulting, announces the publication of an article in a supplement to the Dec. 23, 2011 issue of Science. The article, co-authored by Dr. Edward Abrahams, President of the Personalized Medicine Coalition (PMC), focuses on how best to optimize decision-making for personalized medicine research and development (R&D) and commercialization strategies.

The adoption of personalized medicine strategies could bring great benefits to patients, medical service providers, payers, and the manufacturers of personalized medicine products (e.g., pharmaceutical and diagnostic companies). The article discusses the complexities and uncertainties that personalized medicine introduces for pharmaceutical and diagnostic companies, and how they can address those challenges from commercial, operational, scientific and clinical perspectives. The authors highlight the use of quantitative modeling and decision-analysis tools. In addition, they emphasize the importance of the “soft art” of communication and consensus building within the organization.

Dr. Sean X. Hu, Head of Bionest USA and Managing Director, North America, and lead author, points out that “the key to success lies in the mastery of both the science and the art of personalized medicine strategy decision-making. This article summarizes the combined approach we employ to help companies make the right decisions in a still emerging field.”

Dr. Edward Abrahams adds: “The field of personalized medicine has come a long way over the last decade, to the point that it is now embedded in nearly every major pharmaceutical and diagnostic company research or product portfolio. Nevertheless, companies are struggling with how to navigate a still uncertain regulatory and payer environment, and formulate effective market strategies. The tools and approaches we refer to in this article are an important step toward providing a ‘GPS’ for making the right strategic decisions.”

Bionest is sponsoring the upcoming PMC reception at the InterContinental San Francisco on February 22, 2012, concurrent with the Molecular Medicine TriConference. For further discussions with Drs. Hu and Abrahams on personalized medicine strategies, either at the PMC reception or at another time, please contact Dr. Rachel Laing (rlaing@bionest.com).

Bionest has become a powerhouse in personalized medicine strategy consulting, experienced in a broad spectrum of project types, from the corporate level (personalized medicine business models, commercialization capability building, R&D and commercialization business processes, and organizational structure) to development and commercialization strategies for individual drug assets and companion diagnostics.

In addition, Bionest has been driving thought leadership on personalized medicine, with many articles published or in development on the strategic, commercial and technical aspects of personalized medicine.

For more details, please visit http://www.bionest.com, (navigate to section Strategic/Strategic Practice/Personalized Medicine Strategies).

About Bionest Partners

Bionest Partners provides advisory and management services exclusively for the life science industries, and helps pharmaceutical, biopharmaceutical, diagnostics, biotechnology, and medical device companies and their shareholders to maximize the value of their assets and investments. The company, founded in 2003, has offices in Paris and New York. Its broad customer base includes more than 100 clients ranging from large established companies to biotech start-ups and private equity firms, such as Pfizer, Sanofi, GlaxoSmithKline, Bristol-Myers Squibb, Novartis, AstraZeneca, Johnson & Johnson, Bayer, BiogenIdec, Millennium, MedImmune, Idenix, ProStrakan, Stallergenes, Innate Pharma, Genfit, Jubilant, Sequenom, Axa Private Equity, Blackstone, Candover. The company focuses on providing actionable strategies through its expertise in management consulting, including corporate, franchise, portfolio and product commercialization, organizational and marketing strategies, due diligence, surrogate management, as well as entry into European and US markets. Bionest has a global network of consultants and associate members deployed to address specific client assignments and geographical markets in the Americas, Europe, and Asia Pacific.

About the Personalized Medicine Coalition

The Personalized Medicine Coalition (PMC), representing scientists, patients, providers and payers, promotes the understanding and adoption of personalized medicine concepts, services and products to benefit patients and the health system. For more information on the Personalized Medicine Coalition, please visit http://www.PersonalizedMedicineCoalition.org.

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Bionest and PMC Address Optimization of Personalized Medicine Strategies

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'Personalized medicine' gets $67.5M research boost

The federal government is pledging up to $67.5 million for research into "personalized medicine," which tailors treatment to a patient's genetics and environment.

The funds will flow through Genome Canada, the Cancer Stem Cell Consortium and the Canadian Institutes of Health Research, the federal government's health research agency.

Federal Health Minister Leona Aglukkaq and Minister of State for Science Gary Goodyear made the announcement at the University of Ottawa's health campus Tuesday.

The field of personalized medicine is touted as having the potential to transform the way patients are treated. It looks at the genetic makeup of a person, the patient's environment and the exact course of a particular disease so that an appropriate and effective treatment can be tailored for that individual.

The idea is to move from a one-size-fits-all approach to one that is designed for a specific person and relies on the genetic signatures, or biomarkers, of both the patient and the disease.

Proponents of personalized medicine say it is likely to change the way drugs are developed, how medicines are prescribed and generally how illnesses are managed. They say it will shift the focus in health care from reaction to prevention, improve health outcomes, make drugs safer and mean fewer adverse drug reactions, and reduce costs to health-care systems.

"The potential to understand a person's genetic makeup and the specific character of their illness in order to best determine their treatment will significantly improve the quality of life for patients and their families and may show us the way to an improved health-care system and even save costs in certain circumstances," Aglukkaq said in a news release.

Research projects could last four years

The sequencing of the human genome paved the way for personalized medicine and there have been calls for more research funding so that the discoveries in laboratories can be translated further into the medical field so they will benefit patients more.

Identifying a person's genetic profile, for example, could then indicate a susceptibility to a certain disease, if the biomarkers of that disease have also been discovered. If people know they are genetically at risk of an illness they can take actions to prevent it, and their health-care providers can monitor for it.

Cancer patients could be pre-screened to determine if chemotherapy would work for them, which could not only save a lot of money on expensive treatments but also prevent pain and suffering for patients.

Genome Canada is leading the research initiative, in collaboration with Cancer Stem Cell Consortium and CIHR which on Tuesday launched its Personalized Medicine Signature Initiative. CIHR is committing up to $22.5 million to the large-scale initiative with the other two partners, but it will be providing more funding for other projects under its personalized medicine program.

The research projects are aiming to bring together biomedical, clinical, population health, health economics, ethics and policy researchers to identify areas that are best suited to personalized medicine.

Oncology, cardiovascular diseases, neurodegenerative diseases, psychiatric disorders, diabetes and obesity, arthritis, pain, and Alzheimer’s disease are all considered to be areas that hold promise for personalized medicine.

Funding will also go to projects that are aimed at developing more evidence-based and cost-effective approaches to health care.

Researchers can get up to four years of funding, but 50 per cent of their requested funding must be matched from another source, such as a provincial government or from the academic or private sectors.

Genome Canada, CIHR and the cancer consortium will invest a maximum of $5 million in each individual project.

The successful applicants for the $67.5 million worth of funding won't be announced until December.

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'Personalized medicine' gets $67.5M research boost

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Mark Walter, MD introduces Prolotherapy and PRP: The New Field of Regenerative Medicine – Video

19-10-2011 18:19 Mark Walter, MD is an internationally recognized expert in the field of Regenerative Medicine. He is McGill trained (1980) and has specialized in Sports Medicine and healing sports-related injuries for over three decades. Dr. Walter has worked with many of the top teachers in the world in Regenerative Medicine and for five years he joined the staff of the Univ. of Wisconsin Medical School to help train residents and doctors in Central America. Dr. Walter is a member of the American Academy of Orthopedic Medicine and is founder of ProloMD, located in Sarasota, FL. He has a special interest in working with golf-related injuries and can be found on the web at http://www.PRPyourpain.com and/or ProloMD.com.

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2011 Summit: Keynote Address, CIRM’s Translational Roadmap to Stem Cell Cures, Alan Trounson, PhD – Video

13-12-2011 14:45 Alan Trounson, PhD and President of the California Institute for Regenerative Medicine, offered a positive and forward looking keynote address. Trounson provided an update on CIRM's translational roadmap to regenerative medicine and outlined their accomplishments with over $300 million in funding for human embryonic stem cell research, adult stem cells, cancer stem cells, reprogrammed stem cells and endogenous stem cells for multiple treatments. He spoke about progress in these areas of research emphasizing their promise and relating how close many treatments are to becoming a reality.

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2011 Summit: Keynote Address, CIRM's Translational Roadmap to Stem Cell Cures, Alan Trounson, PhD - Video

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BioRestorative Therapies Signs License Agreement for Stem Cell Disc/Spine Procedure

JUPITER, Fla., Jan. 31, 2012 /PRNewswire/ -- BioRestorative Therapies, Inc. (OTCQB: BRTX) ("BRT") today announced that it has entered into a License Agreement with Regenerative Sciences, LLC ("RS") with respect to certain stem cell-related technology and clinical treatment procedures developed by RS. The treatment is an advanced stem cell injection procedure that may offer relief from lower back pain, buttock and leg pain, or numbness and tingling in the legs or feet as a result of bulging and herniated discs.

To date, over 40 procedures have been performed on patients. It is a minimally invasive out-patient procedure, and objective MRI data and patient outcomes for this novel injection procedure show positive results with limited patient downtime. BRT intends to utilize the existing treatment and outcome data, as well as further research, to prepare for clinical trials in the United States.

Pursuant to the agreement, BRT will obtain an exclusive license to utilize or sub-license a certain medical device for the administration of specific cells and/or cell products to the precise locations within the damaged disc and/or spine (and other parts of the body, if applicable) and an exclusive license to utilize or sublicense a certain method for culturing cells for use in repairing damaged areas. The agreement contemplates a closing of the license grant in March 2012, subject to the fulfillment of certain conditions. 

Mark Weinreb, Chairman and CEO of BRT, said, "This possible alternative to back surgery represents a large market for BRT once it begins offering the procedure to patients who might be facing spinal fusions or back surgery (which often times is unsuccessful). By delivering a particular cell population using a proprietary medical device that inserts a specialized needle into the disc and injects cells for repair and re-population, BRT hopes to revolutionize how degenerative disc disease will be treated." 

About BioRestorative Therapies, Inc.
BioRestorative Therapies, Inc.'s goal is to become a medical center of excellence using cell and tissue protocols, primarily involving a patient's own (autologous) adult stem cells (non-embryonic), allowing patients to undergo cellular-based treatments. In June 2011, the Company launched a technology that involves the use of a brown fat cell-based therapeutic/aesthetic program, known as the ThermoStem™ Program.  The ThermoStem™ Program will focus on treatments for obesity, weight loss, diabetes, hypertension, other metabolic disorders and cardiac deficiencies and will involve the study of stem cells, several genes, proteins and/or mechanisms that are related to these diseases and disorders.  As more and more cellular therapies become standard of care, the Company believes its strength will be its focus on the unity of medical and scientific explanations for clinical procedures and outcomes for future personal medical applications.  The Company also plans to offer and sell facial creams and products under the Stem Pearls™ brand.

This press release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events or results to differ materially from those projected in the forward-looking statements as a result of various factors and other risks, including those set forth in the Company's Form 10, as amended, filed with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and the Company undertakes no obligation to update such statements.

CONTACT:  Mark Weinreb, CEO, Tel: (561) 904-6070, Fax: (561) 429-5684

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RemedyMD Announces Investigate Cell Therapy, a Revolutionary Research Management System for Blood and Marrow …

SALT LAKE CITY, Jan. 31, 2012 /PRNewswire/ -- RemedyMD, the leading provider of Translational Research Informatics, will launch Investigate™ Cell Therapy at the American Society for Blood and Marrow Transplantation BMT Tandem Meetings Feb. 1 – Feb. 5 in San Diego, Calif. (Booth #604).

(Logo: http://photos.prnewswire.com/prnh/20120127/SF43320LOGO)

"Investigate Cell Therapy is designed to improve research efficiencies, facilitate pattern recognition and accelerate discoveries in BMT centers," said Hank Wu, Investigate product manager for RemedyMD.

Fully integrated with ComprehensiveBMT™, Investigate Cell Therapy is the world's only blood and marrow research management system capable of integrating next-generation sequencing, cross-disease registries, and long-term complications and outcomes to advance cell therapy research. Researchers benefit from:

Aggregation of disparate data types from bench to bedside data sources with an intelligent ontology that harmonizes biospecimen, registry and outcomes data 360-degree perspective of phenotypic and genotypic data for subjects, patients, diseases Configurability that keeps up with the pace of research, without waiting for software coding or database administration Multisite, real-time research collaboration without the cost and complexity of infrastructure building and maintenance A suite of reporting, analysis, and data visualization tools that enable drag-and-drop reporting by researchers, scientists, and clinicians without writing SQL

Investigate Cell Therapy leverages the same research platform implemented at the Blood Research Institute of the Blood Center of Wisconsin to integrate lab information management systems (LIMS), study design and tracking, patient registries, and data mining and visualization.

"Research is fundamentally an exercise in pattern recognition," said Gary Kennedy, Founder and CEO of RemedyMD. "Bringing the power of Investigate™ to bear on cell therapy research will provide blood and marrow researchers with the technology they need to recognize new patterns and increase the speed of discovery."

RemedyMD will demonstrate Investigate Cell Therapy at BOOTH #604 at the 2012 Tandem Meetings in San Diego, Calif. beginning Feb. 1 – Feb. 5.

This product is for research use only and is not intended for clinical or cell processing purposes.

About RemedyMD, Inc.

RemedyMD is the leading provider of registries and registry-based products for Life Science Research. RemedyMD's disease registry software is cutting-edge research management software which enables researchers, clinicians and scientists to collect, aggregate, harmonize, and analyze data from widely disparate sources and then use Mosaic™ Platform tools and applications to identify patterns that result in accelerated discoveries and better clinical data management. For more information visit: www.RemedyMD.com.

Media Contacts

Kristofer Beldin, RemedyMD, (801) 870-9407 (cell), kris.beldin@remedymd.com

 

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Gene Linked to Pancreatic Cancer Growth

Inactivating Kras caused tumors to disappear, suggesting possible treatment target

Newswise — ANN ARBOR, Mich. — A mutant protein found in nearly all pancreatic cancers plays a role not only in the cancer’s development but in its continued growth, according to a new study from University of Michigan Comprehensive Cancer Center researchers. The finding suggests a possible target for developing new ways to treat this deadly disease.

Researchers have known that mutations in the Kras gene are what cause pancreatic cancer to develop. These mutations are frequently seen in common precancerous lesions, suggesting it has an early role in pancreatic cancer.

The new study, published in the February Journal of Clinical Investigation, finds that in mice, mutant Kras also keeps the tumor growing and helps precancerous tumors grow into invasive cancer. When the researchers turned off Kras, the tumors disappeared and showed no signs of recurring.

The researchers were able to manipulate Kras in a mouse model that they designed to look at Kras at various points in pancreatic cancer development. In the precancerous lesions, turning off Kras eliminated the tumors in mice and the pancreas tissue returned to normal, with no signs of the cancer returning. With invasive cancer, inactivating Kras killed off the cancer but left the pancreas with fibrous areas similar to scar tissue. Tumors did not recur.

Researchers hope this finding provides the basis for future drug development.

“Right now no drugs specifically target Kras, but there are drugs that target the cellular processes downstream of Kras. We next need to figure out which of these downstream effectors of Kras are important in pancreatic cancer,” says study author Marina Pasca di Magliano, Ph.D., assistant professor of surgery and of cell and developmental biology at the U-M Medical School.

Kras is also known to play a role in lung and colon cancer. But it is likely the biggest player in pancreatic cancer, where more than 90 percent of all tumors have mutated Kras. Pancreatic cancer is one of the most deadly types of cancer: about 4 percent of patients are alive five years after their diagnosis. The disease is often diagnosed when surgery is not an option and it tends to be resistant to available chemotherapies.

“There is a dire need for new therapies for pancreas cancer based on a better understanding of the biology of this disease. My lab is now looking at the downstream inhibitors of Kras to try to find the best target,” Pasca di Magliano says.

Note to patients: This research was based in mice and needs further testing before any possible treatments are available for clinical trials. For information about current pancreatic cancer treatments, call the U-M Cancer AnswerLine at 800-865-1125.

Pancreatic cancer statistics: 43,140 Americans will be diagnosed with pancreatic cancer this year and 36,800 will die from the disease, according to the American Cancer Society

Additional authors: Meredith A. Collins, Filip Bednar, Yaqing Zhang, Jean-christophe Brisset, Stefanie Galban, Craig J. Galban, Sabita Rakshit, and Karen S. Flannagan, all from U-M; and N. Volkan Adsay from Emory University

Funding: U-M Biological Scholar Program; National Cancer Institute; U-M Gastrointestinal Specialized Program of Research Excellence (SPORE); Pancreatic Cancer Action Network; American Association for Cancer Research; Michigan Gastrointestinal Peptide Research Center

Disclosure: None

Reference: Journal of Clinical Investigation, Vol. 122, No. 2, February 2012

Resources:
U-M Cancer AnswerLine, 800-865-1125
U-M Comprehensive Cancer Center, http://www.mcancer.org
Clinical trials at U-M, http://www.UMClinicalStudies.org/cancer

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Gene Linked to Pancreatic Cancer Growth

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Genetic breakthroughs help develop cheaper biofuels: DOE

WASHINGTON, D.C. — Researchers at the U.S. Department of Energy’s (DOE’s) Joint BioEnergy Institute (JBEI) recently announced a major breakthrough in engineering systems of RNA molecules through computer-assisted design.

This could lead to important improvements across a range of industries, including the development of cheaper advanced biofuels. 

Scientists will use these new “RNA machines,” to adjust genetic expression in the cells of microorganisms. 

This will enable scientists to develop new strains of Escherichia coli (E. coli) that are better able to digest switchgrass biomass and convert released sugars to form three types of transportation fuels — gasoline, diesel and jet fuels.

“This is a perfect example of how our investments in basic science innovations can pave the way for future industries and solutions to our nation’s most important challenges,” Energy Secretary Steven Chu said in a news release.

“This breakthrough at the Joint BioEnergy Institute holds enormous potential for the sustainable production of advanced biofuels and countless other valuable goods.”

A breakthrough with E. coli could make it cheaper to produce fuel from switchgrass or other non-food biomass plants to create advanced biofuels with the potential to replace gasoline. 

While the work at the JBEI remains focused on the development of advanced biofuels, its researchers believe their concepts may help other researchers to develop many other desired products, including biodegradable plastics and therapeutic drugs. 

For example, some researchers have started a project to investigate how to use the “RNA machines” to increase the safety and efficacy of medicine therapies to treat diseases, including diabetes and Parkinson’s.

Biological systems are incredibly complex, which makes it difficult to engineer systems of microorganisms that will produce desired products in predictable amounts. 

JBEI’s work, featured in the Dec. 23 issue of “Science” magazine, is the first of its kind to set up and adjust a RNA system in a predictable way. 

Specifically, researchers focused their design-driven approach on RNA sequences that can fold into complicated three dimensional shapes, called ribozymes and aptazymes. 

By using JBEI-developed computer-assisted models and simulations, researchers then created complex RNA-based control systems that are able to program a large number of genes. 

In microorganisms, “commands” that are sent into the cell will be processed by the RNA-based control systems, enabling them to help develop desired products.

One of the major goals of synthetic biology is to produce valuable chemical products from simple, inexpensive and renewable starting materials in a sustainable manner. 

Computer-assisted models and simulations like the one JBEI developed are essential for doing so. 

Up to this point, such tools for biology have been limited, and JBEI’s breakthrough in applying computer assisted design marks an important technical and conceptual achievement for this field. 

To view additional details about this research, visit http://newscenter.lbl.gov/news-releases/2011/12/22/cad-for-rna/.

 

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Miracle Cure A Decade of the Human Genome – BBC Horizon (HD). 720p – Video

11-12-2011 09:20 A decade ago, scientists announced that they had produced the first draft of the human genome, the 3.6 billion letters of our genetic code. It was seen as one of the greatest scientific achievements of our age, a breakthrough that would usher in a new age of medicine. A decade later, Horizon finds out how close we are to developing the life-changing treatments that were hoped for. Horizon follows three people, each with a genetic disease, as they go behind the scenes at some of Britain's leading research labs to find out what the sequencing of the human genome has done for them - and the hope this remarkable project offers all of us.

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Massachusetts General study defines a new genetic subtype of lung cancer

Public release date: 31-Jan-2012
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Contact: Katie Marquedant
kmarquedant@partners.org
617-726-0337
Massachusetts General Hospital

A report from investigators at the Massachusetts General Hospital (MGH) Cancer Center has defined the role of a recently identified gene abnormality in a deadly form of lung cancer. Tumors driven by rearrangements in the ROS1 gene represent 1 to 2 percent of non-small-cell lung cancers (NSCLC), the leading cause of cancer death in the U.S. The researchers show that ROS1-driven tumors can be treated with crizotinib, which also inhibits the growth of tumors driven by an oncogene called ALK, and describe the remarkable response of one patient to crizotinib treatment.

"ROS1 encodes a protein that is important for cell growth and survival, and deregulation of ROS1 through chromosomal rearrangement drives the growth of tumors," says Alice Shaw, MD, PhD, of the MGH Cancer Center ? co-lead author of the paper which has been published online in the Journal of Clinical Oncology. "This finding is important because we have drugs that inhibit ROS1 and could lead to the sort of dramatic clinical response we describe in this paper."

The current findings add ROS1 to the list of genes known to drive NSCLC growth when altered ? a list that includes KRAS, mutations of which account for about 25 percent of cases; EGFR, accounting for 10 to 15 percent; and ALK, rearranged in about 4 percent. Altogether, known cancer-causing genetic changes have been found in a little more than half of NSCLC tumors. Originally identified in brain tumors, ROS1 rearrangement previously had been identified in one NSCLC patient and one NSCLC cell line. The current study was designed to determine the frequency of ROS1 rearrangement in NSCLC and to define the characteristics of patients with ROS1-rearranged tumors.

The investigators screened tumor samples from more than 1,000 NSCLC patients treated at the MGH, Vanderbilt University, the University of California at Irvine, and Fudan University in Shanghai, China. ROS1 rearrangement was identified in 18 tumor samples, for a prevalence of 1.7 percent; ALK rearrangements were identified in 31 samples, with no samples showing alterations in both genes. Patients with ROS1-positive tumors tended to be younger, never to have smoked and to have a type of lung cancer called adenocarcinoma ? characteristics very similar to those of ALK-positive patients.

An earlier MGH study of an experimental ALK inhibitor had found the drug suppressed the growth of a ROS1-positive cell line in addition to ALK-positive cell lines, suggesting that ROS1-positive tumors might be sensitive to the ALK-inhibitor crizotinib. This observation led corresponding author John Iafrate, MD, PhD, and his team to develop a diagnostic test that could identify ROS1-positive tumors. Around the time that test became clinically available, a lung cancer patient whose tumor had not responded to drugs targeting EGFR mutations was referred to the MGH Cancer Center for genetic testing. His tumor was negative for ALK but later proved to harbor a ROS1 rearrangement, and he was enrolled in an extension of the crizotinib clinical trial first reported in the October 28, 2010, New England Journal of Medicine.

"When he enrolled in the trial last April, this patient was extremely sick ? with significant weight loss and very low oxygen levels ? and was barely able to walk," says Shaw. "Within a few days of starting crizotinib, he felt better; and by the time we scanned his chest at seven weeks, the tumors had essentially disappeared from his lungs." Nine months after starting crizotinib therapy, this patient continues to do well. Additional ROS1-positive patients have been enrolled in this trial at MGH, at UC Irvine and at the University of Colorado.

###

Shaw is an assistant professor of Medicine and Iafrate is an associate professor of Pathology at Harvard Medical School. Co-lead authors are Kristin Bergethon, MGH Pathology, and Sai-Hong Ignatius Ou, MD, PhD, University of California at Irvine. The study was supported by grants from the National Institutes of Health and from Pfizer, which received FDA approval for crizotinib in August 2011.

Additional co-authors are Ryohei Katayama, Eugene Mark, Julie Batten, Eunice Kwak, Jeffrey Clark, Jeffrey Engelman, and Mari Mino Kenudson, MGH Cancer Center; Christina Siwak-Tapp, University of California at Irvine; Keith D. Wilner, Pfizer; Christine Lovly, Nerina McDonald, Pierre Massion, Adriana Gonzalez, David Carbone, and William Pao, Vanderbilt University Medical Center; Pierre Massion, Nashville Veterans Affairs Medical Center; Rong Fang and Hongbin Ji, Shanghai Institutes for Biological Sciences; and Haiquan Chen, Shanghai Medical College, Fudan University.

Massachusetts General Hospital (http://www.massgeneral.org), founded in 1811, is the original and largest teaching hospital of Harvard Medical School. The MGH conducts the largest hospital-based research program in the United States, with an annual research budget of more than $750 million and major research centers in AIDS, cardiovascular research, cancer, computational and integrative biology, cutaneous biology, human genetics, medical imaging, neurodegenerative disorders, regenerative medicine, reproductive biology, systems biology, transplantation biology and photomedicine.


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FDA Approves KALYDECO™ (ivacaftor), the First Medicine to Treat the Underlying Cause of Cystic Fibrosis

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX - News) announced today that the U.S. Food and Drug Administration (FDA) has approved KALYDECOTM (ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF), a rare, genetic disease. KALYDECO (kuh-LYE-deh-koh) is approved for people with CF ages 6 and older who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Approximately 1,200 people in the United States, or 4 percent of those with CF, are believed to have this mutation. KALYDECO was granted approval in approximately three months, making it one of the fastest FDA approvals ever and marking the second approval of a new medicine from Vertex in less than a year. The company has established a financial assistance and patient support program to help get KALYDECO to eligible patients for whom it is prescribed. KALYDECO was discovered as part of a collaboration with Cystic Fibrosis Foundation Therapeutics, Inc., the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation.

Vertex is ready to support the introduction of KALYDECO and will begin shipping it to pharmacies in the United States this week. The company will host a conference call for investors and media today, January 31, 2012, at 12:15 p.m. ET to provide more information on KALYDECO availability, price and the financial assistance and patient support program.

“More than 13 years ago we set out to change the lives of people with cystic fibrosis by developing new medicines that address the underlying cause of this rare and devastating disease,” said Jeffrey Leiden, M.D., Ph.D., Vertex’s incoming President and Chief Executive Officer. “KALYDECO represents a major advance in the treatment of cystic fibrosis for people with a specific type of this disease. But our work isn’t done. With the ongoing support of doctors, patients and the Cystic Fibrosis Foundation, we’re making progress toward our ultimate goal of developing additional medicines to help many more people with cystic fibrosis.”

The approval of KALYDECO was based on data from two Phase 3 studies of people with CF who have at least one copy of the G551D mutation. Those who were treated with KALYDECO experienced significant and sustained improvements in lung function as well as other disease measures, including weight gain and certain quality of life measurements, compared to those who received placebo. People who took KALYDECO also experienced significantly fewer pulmonary exacerbations, which are periods of worsening in the signs and symptoms of the disease that often require treatment with antibiotics and hospital visits. Fewer people in the KALYDECO treatment groups discontinued treatment due to adverse events than in the placebo groups. The majority of adverse events associated with KALYDECO were mild to moderate. Adverse events commonly observed in those taking KALYDECO included headache, upper respiratory tract infection (common cold), stomach pain and diarrhea.

“Advances in cystic fibrosis treatment have helped manage symptoms of the disease, however people with cystic fibrosis still have a hard time staying healthy and being active,” said Bonnie Ramsey, M.D., Director of the Center for Clinical and Translational Research at Seattle Children’s Research Institute and principal investigator for one of the Phase 3 KALYDECO trials. “KALYDECO is a fundamental shift in the way cystic fibrosis is treated. In people with a specific genetic mutation, KALYDECO helped them breathe more easily, gain weight and generally feel better.”

“Together, we're changing the lives of people with cystic fibrosis,” said Robert J. Beall, Ph.D., President and CEO of the Cystic Fibrosis Foundation. “We now have a medicine that treats the underlying cause of the disease in people with the G551D mutation. KALYDECO also provides us with a roadmap for exploring additional targeted approaches to treatment for all people with cystic fibrosis.”

Cystic fibrosis is a rare, life-threatening genetic disease for which there is no cure. CF is caused by defective or missing CFTR proteins resulting from mutations in the CFTR gene. CFTR proteins act as channels at the cell surface that control the flow of salt and water across the cells. When the defective CFTR protein does not work properly at the cell surface, abnormally thick, sticky mucus builds up in the lungs. The digestive tract and a number of other organs are also affected. KALYDECO, an oral medicine known as a CFTR potentiator, helps the CFTR protein function more normally once it reaches the cell surface. KALYDECO targets the abnormal CFTR protein channels and opens them to allow chloride ions to move into and out of the cell, which helps thin the mucus so it can hydrate and protect the airways, and keeps them from getting clogged and then infected.

Because KALYDECO targets a specific genetic mutation, a person’s genotype should be known before this new medicine is prescribed. Genetic testing is widely available and FDA-cleared tests are available for people with CF whose genotype is unknown. According to the 2010 Cystic Fibrosis Foundation’s Patient Registry, nearly 92 percent of people with CF have already had their CF mutations identified.

KALYDECO by itself works in a subset of people with CF, but research is ongoing to explore a similar targeted approach using a combination of medicines, including KALYDECO, to treat the most common form of the disease.

Helping People with CF Get KALYDECO

The people who work at Vertex understand that medicines can only help patients who can get them. To that end, the company offers a comprehensive financial assistance and patient support program. A specially-trained and dedicated Vertex team will provide one-on-one support to help eligible patients who are prescribed KALYDECO understand their insurance benefits and the resources that are available to help them.

For eligible patients, the program also includes the following:

Free Medicine Program: Vertex will provide KALYDECO for free to people who do not have insurance and have an annual household income of $150,000 or less; and Co-Pay Assistance Program: For patients with commercial insurance plans that cover KALYDECO and who are enrolled in the Guidance and Patient Support, or GPS, program, there will be a minimal out-of-pocket obligation after which Vertex will help cover co-pay or co-insurance costs up to 30 percent of the list price of the medicine. There is no income limit to be eligible for this program.

Some patients are not eligible for company co-pay support because they have Medicare or Medicaid coverage or live in Massachusetts. There are independent non-profit copay assistance foundations that may be able to help those patients with their out-of-pocket costs.

More information about this program is available by calling 1-877-7-KALYDECO (877-752-5933) or visiting http://www.VertexGPS.com.

About KALYDECO

KALYDECO is the first treatment to target the underlying cause of CF. The Phase 3 studies evaluated KALYDECO in people with CF ages 6 and older who had at least one copy of the G551D mutation. PERSIST, a Phase 3, open-label, 96-week extension study, is underway to evaluate the long-term safety and durability of treatment with KALYDECO. This ongoing study enrolled people who completed 48 weeks of treatment in either Phase 3 study (placebo and KALYDECO treatment groups) and met other eligibility criteria. KALYDECO will be taken as one 150-mg tablet twice daily (every 12 hours).

Vertex retains worldwide rights to develop and commercialize KALYDECO. In October 2011, Vertex submitted a marketing authorization application to the European Medicines Agency (EMA) for KALYDECO and has received agreement from the EMA for accelerated assessment in Europe. The EMA regulatory review is ongoing.

Indication and Important Safety Information

KALYDECO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients ages 6 years and older who have a certain mutation in their CF gene called the G551D mutation.

KALYDECO is not for use in people with CF due to other mutations in the CF gene. It is not effective in CF patients with two copies of the F508del mutation (F508del/F508del) in the CF gene.

It is not known if KALYDECO is safe and effective in children under 6 years of age.

KALYDECO should not be used with certain medicines, including the antibiotics rifampin and rifabutin; seizure medications (phenobarbital, carbamazepine, or phenytoin); and the herbal supplement St. John’s Wort.

KALYDECO can cause serious side effects. High liver enzymes in the blood have occurred in patients taking KALYDECO. Regular assessment is recommended.

The most common side effects associated with KALYDECO include headache; upper respiratory tract infection (common cold) including sore throat, nasal or sinus congestion, and runny nose; stomach (abdominal) pain; diarrhea; rash; nausea; and dizziness.

These are not all the possible side effects of KALYDECO. Patients should tell their healthcare providers about any side effect that bothers them or doesn't go away.

Please see full Prescribing Information for KALYDECO at http://www.KALYDECO.com.

Conference Call for Media and Investors

Vertex will host a conference call and webcast today, January 31, 2012 at 12:15 p.m. ET to provide more information about today's approval, the price of KALYDECO and Vertex's new financial assistance and patient support program. The conference call will be webcast live and a link to the webcast may be accessed from the ‘Events & Presentations' page of Vertex's website at http://www.vrtx.com.

To listen to the live call on the telephone, dial 1-877-250-8889 (United States and Canada) or 1-720-545-0001 (International). To ensure a timely connection, it is recommended that users register at least 15 minutes prior to the scheduled webcast.

The conference ID number for the live call and replay is 48426093.

The call will be available for replay via telephone commencing January 31, 2012 at 3:00 p.m. ET running through 5:00 p.m. ET on February 7, 2012. The replay phone number for the United States and Canada is 1-855-859-2056. The international replay number is 1-404-537-3406.

Following the live webcast, an archived version will be available on Vertex's website until 5:00 p.m. ET on February 14, 2012. Vertex is also providing a podcast MP3 file available for download on the Vertex website at http://www.vrtx.com.

About Cystic Fibrosis

Cystic fibrosis is a rare, life-threatening genetic disease affecting approximately 30,000 people in the United States and 70,000 people worldwide. Today, the median predicted age of survival for a person with CF is approximately 38 years but the median age of death remains in the mid-20s. There are more than 1,800 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic, or genotyping test, lead to CF by creating non-working or too few CFTR proteins at the cell surface. The absence of working CFTR proteins results in poor flow of salt and water across cell membranes in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.

In some people, CFTR proteins are present at the cell surface but do not work properly. One type of this dysfunction is known as the G551D mutation. Approximately 4 percent of those with CF, or about 1,200 people in the United States, are believed to have this mutation. An estimated 1,000 people in Europe have the G551D mutation.

In people with the most common mutation in the CFTR gene, F508del, the CFTR protein does not reach the cell surface in normal amounts and the CFTR proteins that reach the surface do not work correctly. Nearly 90 percent of people with CF have at least one copy of the F508del mutation; approximately half of those with CF have two copies. KALYDECO is not effective in CF patients who have two copies of the F508del mutation in the CFTR gene.

Vertex’s Ongoing CF Research and Development Program

KALYDECO has been approved by the FDA for people with CF ages 6 and older who have at least one copy of the G551D mutation. Vertex is planning to begin additional studies this year to evaluate KALYDECO in children with CF as young as 2 years old and in people with CF who have the R117H mutation or gating mutations that were not evaluated in the previous Phase 3 studies.

Enrollment is ongoing in the second part of a Phase 2 clinical trial of combination regimens of KALYDECO and VX-809, a CFTR corrector, in people with the most common mutation in CF, known as F508del. In addition, the company plans to begin Phase 2 development of VX-661, a second CFTR corrector, in the first quarter of 2012.

Collaborative History with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT)

Vertex initiated its CF research program in 1998 as part of a collaboration with CFFT, the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation. This collaboration was expanded to support the accelerated discovery and development of Vertex's CFTR modulators.

About the Cystic Fibrosis Foundation

The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The CF Foundation is a donor-supported nonprofit organization. For more information, visit http://www.cff.org.

About Vertex

Vertex creates new possibilities in medicine. Our team discovers, develops and commercializes innovative therapies so people with serious diseases can lead better lives.

Vertex scientists and our collaborators are working on new medicines to cure or significantly advance the treatment of hepatitis C, cystic fibrosis, rheumatoid arthritis, epilepsy and other life-threatening diseases.

Founded more than 20 years ago in Cambridge, MA, we now have ongoing worldwide research programs and sites in the U.S., U.K. and Canada. Today, Vertex has more than 2,000 employees around the world, and Science magazine named Vertex number one on its 2011 list of Top Employers in the life sciences.

Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements, as defined in the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding (i) Vertex being ready to support the introduction of KALYDECO and beginning to ship it to pharmacies this week; (ii) Vertex's financial assistance and patient support programs; (iii) the progress Vertex is making toward its ultimate goal of developing additional medicines to help many more people with cystic fibrosis; (iv) the roadmap provided by KALYDECO for exploring additional targeted approaches to treatment for all people with cystic fibrosis; (v) the ongoing research to explore a targeted approach using a combination of medicines, including KALYDECO, to treat the most common form of the disease and (vi) planned additional clinical trials of KALYDECO in children as young as 2 years old and people with CF who have the R117H mutation and gating mutations that were not evaluated in previous Phase 3 clinical trials. While the company believes the forward-looking statements contained in this press release are accurate, there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, risks related to the commercialization of KALYDECO and development of additional medicines to treat cystic fibrosis and the other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission and available through Vertex's website at http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

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FDA Approves KALYDECO™ (ivacaftor), the First Medicine to Treat the Underlying Cause of Cystic Fibrosis

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Scientists Transform Skin Cells Direct To Brain Cells, Bypassing Stem Cell Stage

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Academic Journal
Main Category: Stem Cell Research
Also Included In: Neurology / Neuroscience;  Biology / Biochemistry
Article Date: 31 Jan 2012 - 2:00 PST

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Bypassing the stem cell stage, researchers at the Stanford University School of Medicine in California converted mouse skin cells directly into neural precursor cells, the cells that go on to form the three main types of cell in the brain and nervous system. They write about their findings in the 30 January early online issue of the Proceedings of the National Academy of Sciences.

The findings of this and an earlier study question the idea that pluripotency (the ability to become virtually any other cell in the body, a key characteristic of stem cells) is a necessary stage in the conversion of one cell type to another.

In the earlier study, the same team transformed mouse and human skin cells directly into functional neurons. But this study is a substantial advance on the earlier one for two reasons.

First, neural precursor cells can not only differentiate into neurons, they can also become either of the two other main types of cell in the nervous system: astrocytes and oligodendrocytes.

Astrocytes are star-shaped glia cells that hold neurons in place, get nutrients to them, and digest parts of dead neurons. Oligodendrocytes make the myelin that insulates nerve fibers that connect neurons to one another and allows them to transmit signals.

And secondly, neural precursor cells are a more useful and versatile end-product for the lab, where they can be cultivated in large numbers for transplantation or drug screening.

Together, the two studies raise the possibility that embryonic stem cell research and induced pluripotency could be replaced by a more direct way of making specific cell types for treatments and research.

The problem with embryonic stem cells, although they are considered the "gold standard" in generating new types of cell, is the ethical question of where they come from, and also because they don't come from the patient's own body, the patient has to take drugs to stop their immune system rejecting the new tissue.

Induced pluripotency, where the patient's own cells are reprogrammed into stem cells, appears to overcome the ethical and immune rejection problems of embryonic stem cells, except they introduce the risk of switching on genes that cause cancer. Although this risk can be reduced by screening out unwanted pluripotent cells, it introduces a cost.

The senior author of the new study is Dr Marius Wernig, assistant professor of pathology and a member of Stanford's Institute for Stem Cell Biology and Regenerative Medicine. He told the press he and his colleagues were "thrilled" about the medical potential of their findings.

"We've shown the cells can integrate into a mouse brain and produce a missing protein important for the conduction of electrical signal by the neurons. This is important because the mouse model we used mimics that of a human genetic brain disease," said Wernig.

However, he cautioned that more work is needed before they can show a similar conversion from human skin cells is not only possible and effective, but also safe.

For the study, Wernig and colleages infected embryonic mouse skin cells (a cell line commonly used in labs) with a virus carrying three transcription factors (Brn2, Sox2 and FoxG1) known to be present at a high level in neural precursor cells. In just over three weeks, one in ten of the skin cells had started to look and act like neural precursor cells.

In the earlier study, they had used a different set of three transcription factors (Brn2, Ascl1 and Myt1l).

They confirmed the presence of neural precursor cells in two ways: in the lab and in animals (in vitro and in vivo).

In the lab, they confirmed the transformed cells were expressing the appropriate genes and had the same shape and function as naturally derived neural precursor cells.

And to confirm them in animals, they injected the new cells into the brains of newborn mice bred to lack to ability to make the myelin sheath that surrounds nerve fibers. After ten weeks, the new cells had differentiated into oligodendroytes and had begun to coat the mice's nerve fibers with myelin.

The team is now hoping to repeat their success with skin cells from adult mice and humans.

Funds from the California Institute for Regenerative Medicine, the New York Stem Cell Foundation, the Ellison Medical Foundation, the Stinehart-Reed Foundation and the National Institutes of Health helped pay for the study.

Written by Catharine Paddock PhD
Copyright: Medical News Today
Not to be reproduced without permission of Medical News Today

Visit our stem cell research section for the latest news on this subject. "Direct conversion of mouse fibroblasts to self-renewing, tripotent neural precursor cells"; Ernesto Lujan, Soham Chanda, Henrik Ahlenius, Thomas C. Südhof, and Marius Wernig; PNAS Published online ahead of print 30 January 2012; DOI:10.1073/pnas.1121003109; Link to Abstract.
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Catharine Paddock PhD. "Scientists Transform Skin Cells Direct To Brain Cells, Bypassing Stem Cell Stage." Medical News Today. MediLexicon, Intl., 31 Jan. 2012. Web.
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Scientists Transform Skin Cells Direct To Brain Cells, Bypassing Stem Cell Stage

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Need muscle for a tough spot? Turn to fat stem cells, UC San Diego researchers say

Public release date: 27-Jan-2012
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Contact: Daniel Kane
dbkane@ucsd.edu
858-534-3262
University of California - San Diego

Stem cells derived from fat have a surprising trick up their sleeves: Encouraged to develop on a stiff surface, they undergo a remarkable transformation toward becoming mature muscle cells. The new research appears in the journal Biomaterials. The new cells remain intact and fused together even when transferred to an extremely stiff, bone-like surface, which has University of California, San Diego bioengineering professor Adam Engler and colleagues intrigued. These cells, they suggest, could hint at new therapeutic possibilities for muscular dystrophy.

In diseases like muscular dystrophy or a heart attack, "muscle begins to die and undergoes its normal wounding processes," said Engler, a bioengineering professor at the Jacobs School of Engineering at UC San Diego. "This damaged tissue is fundamentally different from a mechanical perspective" than healthy tissue.

Transplanted stem cells might be able to replace and repair diseased muscle, but up to this point the transplants haven't been very successful in muscular dystrophy patients, he noted. The cells tend to clump into hard nodules as they struggle to adapt to their new environment of thickened and damaged tissue.

Engler, postdoctoral scholar Yu Suk Choi and the rest of the team think their fat-derived stem cells might have a better chance for this kind of therapy, since the cells seem to thrive on a stiff and unyielding surface that mimics the damaged tissue found in people with MD.

In their study in the journal Biomaterials, the researchers compared the development of bone marrow stem cells and fat-derived stem cells grown on surfaces of varying stiffness, ranging from the softness of brain tissue to the hardness of bone.

Cells from the fat lineage were 40 to 50 times better than their bone marrow counterparts at displaying the proper proteins involved in becoming muscle. These proteins are also more likely to "turn on" in the correct sequence in the fat-derived cells, Engler said.

Subtle differences in how these two types of cells interact with their environment are critical to their development, the scientists suggest. The fat-derived cells seem to sense their "niche" on the surfaces more completely and quickly than marrow-derived cells. "They are actively feeling their environment soon, which allows them to interpret the signals from the interaction of cell and environment that guide development," Choi explained.

Perhaps most surprisingly, muscle cells grown from the fat stem cells fused together, forming myotubes to a degree never previously observed. Myotubes are a critical step in muscle development, and it's a step forward that Engler and colleagues hadn't seen before in the lab.

The fused cells stayed fused when they were transferred to a very stiff surface. "These programmed cells are mature enough so that they don't respond the environmental cues" in the new environment that might cause them to split apart, Engler says.

Engler and colleagues will now test how these new fused cells perform in mice with a version of muscular dystrophy. The cells survive in an environment of stiff tissue, but Engler cautions that there are other aspects of diseased tissue such as its shape and chemical composition to consider. "From the perspective of translating this into a clinically viable therapy, we want to know what components of the environment provide the most important cues for these cells," he said.

###

Co-authors for the Biomaterials study "Mechanical derivation of functional myotubes from adipose-derived stem cells" include Ludovic G. Vincent and Andrew R. Lee in the Department of Bioengineering at the UC San Diego Jacobs School of Engineering, and Marek K. Dobke from the Division of Plastic Surgery, UC San Diego School of Medicine. The research was funded by the Human Frontier Science Program and the National Institutes of Health Common Fund.


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Promising gene therapy treats Leukemia – Video

11-08-2011 09:02 Dr. Jennifer Ashton discusses an experimental gene therapy that used modified T-cells to attack tumor cells.

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Promising gene therapy treats Leukemia - Video

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Cytori Receives Approval From FDA to Initiate U.S. Cardiac Cell Therapy Trial; Investigational Device Exemption (IDE …

SOURCE: Cytori Therapeutics, Inc.

SAN DIEGO, CA--(Marketwire - Jan 30, 2012) - Cytori Therapeutics (NASDAQ: CYTX) has received an Investigational Device Exemption (IDE) approval from the U.S. FDA to begin the ATHENA trial. ATHENA will investigate the use of the Celution® System, an innovative medical device to prepare adipose-derived stem and regenerative cells (ADRCs) to treat a form of coronary heart disease, chronic myocardial ischemia (CMI). The IDE application was originally submitted to the FDA in December 2011.

ATHENA is a multi-center, randomized, double blind, placebo controlled, pilot trial to investigate the use of autologous, clinical-grade ADRCs, processed at the point-of-care with Cytori's proprietary Celution® System. The trial will enroll up to 45 patients with no-option CMI who have limited therapeutic options. It will evaluate a variety of clinical and functional outcomes, including safety, peak oxygen consumption (mVO2), and clinical outcomes at 12-months.

"Following our pre-IDE meeting with the FDA, we received constructive guidance and implemented the Agency's recommendations, ultimately resulting in rapid approval to initiate the ATHENA trial," said Christopher J. Calhoun, chief executive officer for Cytori. "We look forward to working with the FDA on further defining our clinical strategy in the U.S."

Previously, Cytori reported six and 18-month trial data from PRECISE, a European clinical trial for this same indication showing improvement in mVO2. In Europe, Cytori has applied to expand its Celution® System CE Mark to include no-option CMI claims based on data from the PRECISE trial. Cytori is also enrolling ADVANCE, a European pivotal trial investigating the Celution® System for acute myocardial infarction (heart attacks).

In the U.S., it is estimated that 120,000 to 250,000 patients are diagnosed each year with chronic myocardial ischemia, a subset of the approximate 5.8 million patients who currently have some form of heart failure. CMI patients typically have undergone multiple revascularization procedures that have not improved their condition and are at a stage where they have few therapeutic options remaining.

About Cytori
Cytori is a leader in providing patients and physicians around the world with medical technologies that harness the potential of adult regenerative cells from adipose tissue. The Celution® System family of medical devices and instruments is being sold into the European and Asian cosmetic and reconstructive surgery markets and available in the United States only for use as an investigational device under Cytori's FDA approved IDE. Our StemSource® product line is sold globally for cell banking and research applications. Our PureGraft® products are available in North America and Europe for fat grafting procedures. http://www.cytori.com

Cautionary Statement Regarding Forward-Looking Statements
This press release includes forward-looking statements regarding events, trends and business prospects, which may affect our future operating results and financial position, such as the successful initiation of a clinical trial of the Company's Celution® System for chronic myocardial ischemia, our efforts to expand our CE Mark. Such statements are subject to risks and uncertainties that could cause our actual results and financial position to differ materially. Some of these risks include clinical and regulatory uncertainties, such as those associated with the ATHENA clinical trial, including risks in the collection and results of clinical data, final clinical outcomes, dependence on third party performance, and other risks and uncertainties described under the "Risk Factors" in Cytori's Securities and Exchange Commission Filings. We assume no responsibility to update or revise any forward-looking statements to reflect events, trends or circumstances after the date they are made.

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Cytori Receives Approval From FDA to Initiate U.S. Cardiac Cell Therapy Trial; Investigational Device Exemption (IDE ...

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ThermoGenesis Announces Management Transition in Conjunction With Tactical Realignment of Company

RANCHO CORDOVA, Calif., Jan. 30, 2012 /PRNewswire/ -- ThermoGenesis Corp. (NASDAQ: KOOL - News), a leading supplier of innovative products and services that process and store adult stem cells, today announced that the Company has implemented a number of changes in corporate management responsibilities to maximize the Company's progress toward its strategic goals.

Matthew Plavan, currently Chief Financial Officer and Executive Vice President, Business Development, will become Chief Executive Officer and a member of the Board of Directors, while retaining his position as the Company's Chief Financial Officer. Plavan has served as Chief Financial Officer since 2005 and served as Chief Operating Officer from 2008-2010.

Plavan replaces J. Melville Engle, who has retired from his position as Chairman and Chief Executive Officer.  Engle joined the Company as Chief Executive Officer in 2009 and was named Chairman of the Board in 2010. "ThermoGenesis has made important strides during Mel's tenure, as he was instrumental in building the ThermoGenesis management team and in the expansion of the Company's distributor network. We appreciate his contributions to the Company," said Patrick McEnany, a member of the board of directors.

"Given the uncertain duration of today's challenging global economy, we chose to reorganize the Company.  The board believed changes were necessary if the Company was going to achieve its short-term objectives and be positioned for long-term growth. We believe Matt's experience, knowledge of our market and proven track record at ThermoGenesis makes him the most qualified person to lead the Company," McEnany added.

"I appreciate the board's confidence in me and believe our streamlined management is a strong and dedicated group of individuals that will drive the Company to success," Plavan stated.  "Our current organization was staffed to support a faster entry of our products into new markets than we have achieved as of today, including China and India. The changes made today recalibrate the Company's resources to our current revenues and the cadence of new market opportunities, while maintaining the strong support our growing customer base has come to expect. We believe we are now optimally positioned to grow the business and maximize shareholder value, even in these turbulent economic times," Plavan added.

The Company indicated it has also eliminated eight additional positions. The Company will provide additional details on its new operating structure and objectives during its second quarter fiscal 2012 conference call on Thursday, February 9th.

The Company said it expects to record one-time expenses of approximately $500,000 related to the reorganization announced today in the third quarter of fiscal 2012.  The restructured operations should result in an annualized expense reduction of approximately $2 million.

About ThermoGenesis Corp.

ThermoGenesis Corp. (www.thermogenesis.com) is a leader in developing and manufacturing automated blood processing systems and disposable products that enable the manufacture, preservation and delivery of cell and tissue therapy products. These include:

The BioArchive® System, an automated cryogenic device, used by cord blood stem cell banks in more than 30 countries for cryopreserving and archiving cord blood stem cell units for transplant. AXP® AutoXpress® Platform (AXP), a proprietary family of automated devices that includes the AXP and the MXP® MarrowXpress® and companion sterile blood processing disposables for harvesting stem cells in closed systems. The AXP device is used for the processing of cord blood. The MXP is used for the preparation of cell concentrates, including stem cells, from bone marrow aspirates in the laboratory setting. The Res-Q® 60 BMC/PRP (Res-Q), a point-of-care system designed for the preparation of cell concentrates, including stem cells, from bone marrow aspirates and whole blood for platelet rich plasma (PRP). The CryoSeal® FS System, an automated device and companion sterile blood processing disposable, used to prepare fibrin sealants from plasma in about an hour. The CryoSeal FS System is approved in the U.S. for liver resection surgeries. The CryoSeal FS System has received the CE-Mark which allows sales of the product throughout the European community.

This press release contains forward-looking statements.  These statements involve risks and uncertainties that could cause actual outcomes to differ materially from those contemplated by the forward-looking statements. Several factors including timing of FDA and foreign regulatory approvals, changes in customer forecasts, our failure to meet customers' purchase order and quality requirements, supply shortages, production delays, changes in the markets for customers' products, introduction timing and acceptance of our new products scheduled for fiscal year 2012, and introduction of competitive products and other factors beyond our control could result in a materially different revenue outcome and/or in our failure to achieve the revenue levels we expect for fiscal 2012.  A more complete description of these and other risks that could cause actual events to differ from the outcomes predicted by our forward-looking statements is set forth under the caption "Risk Factors" in our annual report on Form 10-K and other reports we file with the Securities and Exchange Commission from time to time, and you should consider each of those factors when evaluating the forward-looking statements.

ThermoGenesis Corp.
Web site: http://www.thermogenesis.com
Contact: Investor Relations
+1-916-858-5107, or
ir@thermogenesis.com

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ThermoGenesis Announces Management Transition in Conjunction With Tactical Realignment of Company

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Stem cells may shed light on hepatitis, MIT researchers find

Sangeeta Bhatia, MIT professor of health sciences and technology and electrical engineering and computer science

Researchers at MIT and their colleagues said they have devised a way to produce liver-like cells from stem cells, a key step in studying why people respond differently to Hepatitis C.        
     
An infectious disease that can cause inflammation and organ failure, Hepatitis C has different effects on different people, but no one is sure why, the researchers said in a press release from MIT. Some people are very susceptible to the infection, while others are resistant.

The researchers said that by studying liver cells from different people in the lab, they may determine how genetic differences produce these varying responses. However, liver cells are hard to get and very difficult to grow in a lab dish because they tend to lose their normal structure and function when removed from the body.

The researchers, from MIT, Rockefeller University and the Medical College of Wisconsin, have come up with a way to produce liver-like cells from induced pluripotent stem cells (iPSCs), which are made from body tissues rather than embryos. Those liver-like cells can then be infected with Hepatitis C and help scientists study the varying responses to the infection.

The scientists claim this is the first time an infection has been made in cells derived from iPSCs. Their new technique is described in the Jan. 30 issue of the Proceedings of the National Academy of Sciences. The development, they said, may also eventually enable personalized medicine, in which doctors could test the effect of different drugs on tissues derived from the patient being treated and then customize therapy for that patient.

The new study is a collaboration between Sangeeta Bhatia, professor of health sciences and technology and electrical engineering and computer science at MIT; Charles Rice, professor of virology at Rockefeller; and Stephen Duncan, professor of human and molecular genetics at the Medical College of Wisconsin.

The iPSCs are derived from normal body cells, often skin cells. By turning on certain genes in those cells, the scientists can revert them to an immature state that is identical to embryonic stem cells, which can turn into any cell type. Once the cells become pluripotent, they can be directed to become liver-like cells by turning on genes that control liver development.

The researchers’ goal is to take cells from patients who have unusual reactions to hepatitis C infection, transform them into liver cells and study their genetics to see why people respond as they do. “Hepatitis C virus causes an unusually robust infection in some people, while others are very good at clearing it. It’s not yet known why those differences exist,” Bhatia said in a statement.

Bhatia is a 2009 Mass High Tech Women to Watch honoree.
 

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Stem cells may shed light on hepatitis, MIT researchers find

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Scientists use skin samples to create human brain cells

Sixteen years after Dolly the sheep was cloned in Edinburgh, scientists in Scotland have made another startling medical breakthrough.

Researchers at Edinburgh's Centre for Regenerative Medicine have created brain tissue from patients suffering mental illnesses such as schizophrenia and depression.

"A patient's neurones can tell us a great deal about the psychological conditions that affect them, but you cannot stick a needle in someone's brain and take out its cells," the center's director, Professor Charles ffrench-Constant, told the Guardian.

"However, we have found a way round that. We can take a skin sample, make stem cells from it and then direct these stem cells to grow into brain cells. Essentially, we are turning a person's skin cells into brain."

The scientists hope that studying these manufactured brain cells will reveal clues to the conditions of patients with mental illnesses—a task that had been challenging in the past.

"It is very difficult to get primary tissue to study until after a patient has died," said the Royal Edinburgh Hospital's Professor Andrew McIntosh, who is collaborating with the center on the project.

"Even then, that tissue is affected by whatever killed them and by the impact of the medication they had been taking for their condition, possibly for several decades. So having access to living brain cells is a significant development for the development of drugs for these conditions," McIntosh said.

If successful, the same methods could be used for other organs, including the liver and heart.

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Scientists use skin samples to create human brain cells

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Skin transformed into brain cells

30 January 2012 Last updated at 19:13 ET By James Gallagher Health and science reporter, BBC News

Skin cells have been converted directly into cells which develop into the main components of the brain, by researchers studying mice in California.

The experiment, reported in Proceedings of the National Academy of Sciences, skipped the middle "stem cell" stage in the process.

The researchers said they were "thrilled" at the potential medical uses.

Far more tests are needed before the technique could be used on human skin.

Stem cells, which can become any other specialist type of cell from brain to bone, are thought to have huge promise in a range of treatments. Many trials are taking place, such as in stroke patients or specific forms of blindness.

One of the big questions for the field is where to get the cells from. There are ethical concerns around embryonic stem cells and patients would need to take immunosuppressant drugs as any stem cell tissue would not match their own.

An alternative method has been to take skin cells and reprogram them into "induced" stem cells. These could be made from a patient's own cells and then turned into the cell type required, however, the process results in cancer-causing genes being activated.

Continue reading the main story “Start Quote

We are thrilled about the prospects for potential medical use of these cells”

End Quote Prof Marius Wernig Stanford University School of Medicine Direct approach

The research group, at the Stanford University School of Medicine in California, is looking at another option - converting a person's own skin cells into specialist cells, without creating "induced" stem cells. It has already transformed skin cells directly into neurons.

This study created "neural precursor" cells, which can develop into three types of brain cell: neurons, astrocytes and oligodendrocytes.

These precursor cells have the advantage that, once created, they can be grown in a laboratory into very large numbers. This could be critical if the cells were to be used in any therapy.

Brain cells and skin cells contain the same genetic information, however, the genetic code is interpreted differently in each. This is controlled by "transcription factors".

The scientists used a virus to infect skin cells with three transcription factors known to be at high levels in neural precursor cells.

After three weeks about one in 10 of the cells became neural precursor cells.

Lead researcher Prof Marius Wernig said: "We are thrilled about the prospects for potential medical use of these cells.

"We've shown the cells can integrate into a mouse brain and produce a missing protein important for the conduction of electrical signal by the neurons.

"More work needs to be done to generate similar cells from human skin cells and assess their safety and efficacy."

Dr Deepak Srivastava, who has researched converting cells into heart muscle, said the study: "Opens the door to consider new ways to regenerate damaged neurons using cells surrounding the area of injury."

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Skin transformed into brain cells

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Researchers turn skin cells into neural precusors, bypassing stem-cell stage

The multiple successes of the direct conversion method could refute the idea that pluripotency (a term that describes the ability of stem cells to become nearly any cell in the body) is necessary for a cell to transform from one cell type to another. Together, the results raise the possibility that embryonic stem cell research and another technique called "induced pluripotency" could be supplanted by a more direct way of generating specific types of cells for therapy or research.

This new study, which will be published online Jan. 30 in the Proceedings of the National Academy of Sciences, is a substantial advance over the previous paper in that it transforms the skin cells into neural precursor cells, as opposed to neurons. While neural precursor cells can differentiate into neurons, they can also become the two other main cell types in the nervous system: astrocytes and oligodendrocytes. In addition to their greater versatility, the newly derived neural precursor cells offer another advantage over neurons because they can be cultivated to large numbers in the laboratory — a feature critical for their long-term usefulness in transplantation or drug screening.

In the study, the switch from skin to neural precursor cells occurred with high efficiency over a period of about three weeks after the addition of just three transcription factors. (In the previous study, a different combination of three transcription factors was used to generate mature neurons.) The finding implies that it may one day be possible to generate a variety of neural-system cells for transplantation that would perfectly match a human patient.

"We are thrilled about the prospects for potential medical use of these cells," said Marius Wernig, MD, assistant professor of pathology and a member of Stanford's Institute for Stem Cell Biology and Regenerative Medicine. "We've shown the cells can integrate into a mouse brain and produce a missing protein important for the conduction of electrical signal by the neurons. This is important because the mouse model we used mimics that of a human genetic brain disease. However, more work needs to be done to generate similar cells from human skin cells and assess their safety and efficacy."

Wernig is the senior author of the research. Graduate student Ernesto Lujan is the first author.

While much research has been devoted to harnessing the pluripotency of embryonic stem cells, taking those cells from an embryo and then implanting them in a patient could prove difficult because they would not match genetically. An alternative technique involves a concept called induced pluripotency, first described in 2006. In this approach, transcription factors are added to specialized cells like those found in skin to first drive them back along the developmental timeline to an undifferentiated stem-cell-like state. These "iPS cells" are then grown under a variety of conditions to induce them to re-specialize into many different cell types.

Scientists had thought that it was necessary for a cell to first enter an induced pluripotent state or for researchers to start with an embryonic stem cell, which is pluripotent by nature, before it could go on to become a new cell type. However, research from Wernig's laboratory in early 2010 showed that it was possible to directly convert one "adult" cell type to another with the application of specialized transcription factors, a process known as transdifferentiation.

Wernig and his colleagues first converted skin cells from an adult mouse to functional neurons (which they termed induced neuronal, or iN, cells), and then replicated the feat with human cells. In 2011 they showed that they could also directly convert liver cells into iN cells.

"Dr. Wernig's demonstration that fibroblasts can be converted into functional nerve cells opens the door to consider new ways to regenerate damaged neurons using cells surrounding the area of injury," said pediatric cardiologist Deepak Srivastava, MD, who was not involved in these studies. "It also suggests that we may be able to transdifferentiate cells into other cell types." Srivastava is the director of cardiovascular research at the Gladstone Institutes at the University of California-San Francisco. In 2010, Srivastava transdifferentiated mouse heart fibroblasts into beating heart muscle cells.

"Direct conversion has a number of advantages," said Lujan. "It occurs with relatively high efficiency and it generates a fairly homogenous population of cells. In contrast, cells derived from iPS cells must be carefully screened to eliminate any remaining pluripotent cells or cells that can differentiate into different lineages." Pluripotent cells can cause cancers when transplanted into animals or humans.

The lab's previous success converting skin cells into neurons spurred Wernig and Lujan to see if they could also generate the more-versatile neural precursor cells, or NPCs. To do so, they infected embryonic mouse skin cells — a commonly used laboratory cell line — with a virus encoding 11 transcription factors known to be expressed at high levels in NPCs. A little more than three weeks later, they saw that about 10 percent of the cells had begun to look and act like NPCs.

Repeated experiments allowed them to winnow the original panel of 11 transcription factors to just three: Brn2, Sox2 and FoxG1. (In contrast, the conversion of skin cells directly to functional neurons requires the transcription factors Brn2, Ascl1 and Myt1l.) Skin cells expressing these three transcription factors became neural precursor cells that were able to differentiate into not just neurons and astrocytes, but also oligodendrocytes, which make the myelin that insulates nerve fibers and allows them to transmit signals. The scientists dubbed the newly converted population "induced neural precursor cells," or iNPCs.

In addition to confirming that the astrocytes, neurons and oligodendrocytes were expressing the appropriate genes and that they resembled their naturally derived peers in both shape and function when grown in the laboratory, the researchers wanted to know how the iNPCs would react when transplanted into an animal. They injected them into the brains of newborn laboratory mice bred to lack the ability to myelinate neurons. After 10 weeks, Lujan found that the cells had differentiated into oligodendroytes and had begun to coat the animals' neurons with myelin.

"Not only do these cells appear functional in the laboratory, they also seem to be able to integrate appropriately in an in vivo animal model," said Lujan.

The scientists are now working to replicate the work with skin cells from adult mice and humans, but Lujan emphasized that much more research is needed before any human transplantation experiments could be conducted. In the meantime, however, the ability to quickly and efficiently generate neural precursor cells that can be grown in the laboratory to mass quantities and maintained over time will be valuable in disease and drug-targeting studies.

"In addition to direct therapeutic application, these cells may be very useful to study human diseases in a laboratory dish or even following transplantation into a developing rodent brain," said Wernig.

Provided by Stanford University Medical Center (news : web)

Read more:
Researchers turn skin cells into neural precusors, bypassing stem-cell stage

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