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Helen McNeills work in developmental biology spans birth defects to cancer

Helen McNeill, PhD, has been named a BJC Investigator at Washington University School of Medicine in St. Louis. She is the first researcher named as part of the new BJC Investigators Program.

Helen McNeill, PhD, has been named a BJC Investigator at Washington University School of Medicine in St. Louis. She is the first researcher named as part of the new BJC Investigators Program, which aims to recruit scientists who bring innovative approaches to major biological quandaries and whose discoveries stand to inform new ways of understanding disease and developing treatments.

McNeill, the first of 10 expected BJC Investigators, is currently a professor in the Institute of Medical Science and the Department of Molecular Genetics, both at the University of Toronto. She is also a senior investigator at the Lunenfeld-Tanenbaum Research Institute, part of the Sinai Health System in Toronto. Her appointment as a BJC Investigator and a professor of developmental biology at Washington University begins Jan. 1, 2018.

We are excited to begin the BJC Investigators Program with the appointment of Dr. Helen McNeill, an international leader in the field of developmental biology, said David H. Perlmutter, MD, executive vice chancellor for medical affairs and dean of the School of Medicine. We sought candidates who had already indelibly changed their fields, whose discoveries will result in new and fundamental shifts in scientific thinking and whose laboratories will become a nidus for additional innovative work across Washington University. Helens scientific accomplishments, her high standards of excellence and ability to collaborate across disciplines make her a perfect fit.

The program is designed to specifically focus on basic science and is inspired by the Howard Hughes Medical Institutes philosophy of investing in people with exceptional creative talent. It plans to bring 10 renowned researchers to Washington University School of Medicine and the life sciences ecosystem of St. Louis.

We are very excited about the BJC Investigators Program at Washington University School of Medicine, said Steven H. Lipstein, CEO of BJC HealthCare. This program represents another joint effort between BJC and Washington University to help keep the schools biomedical research at the forefront of discovery. Pioneering research here in St. Louis offers our best hope for finding solutions to societys greatest medical challenges.

McNeills work is focused on understanding the processes that govern how cells make contact and work together to form the broader architecture of whole tissues, both during development and adulthood. Her work spanning studies of fruit flies, mice and human genetic data has relevance for understanding birth defects, cancer and diseases of specific organs, such as the kidney and lungs.

McNeill earned a bachelors degree in biology from the Ramapo College of New Jersey in 1985, followed by a doctorate in molecular and cellular physiology from Stanford University in 1993. She continued research at Stanford with a postdoctoral fellowship in fruit fly genetics. McNeill later led the Developmental Patterning Laboratory at the London Research Institute, a part of the Imperial Cancer Research Fund of the United Kingdom. She joined the faculty of the University of Toronto in 2005, where she has directed the Collaborative Program in Developmental Biology and earned numerous recognitions for her research, including the Petro-Canada Young Innovator Award and the Lloyd S.D. Fogler, QC, Award of Excellence for her research in cancer biology. Last year, she was awarded a Canada Tier 1 Research Chair, a position in which a scientist is recognized by peers as a world leader in his or her field.

I am delighted that Dr. McNeill will be joining us at Washington University, said Lilianna Solnica-Krezel, PhD, the Alan A. and Edith L. Wolff Professor of Developmental Biology and head of the Department of Developmental Biology. She is a leader in the field and among the most original and creative investigators of pathways that are vital for the regulation of tissue structure and growth. The pathways she studies are among the least understood cellular pathways, with implications for a variety of birth defects and other diseases, including cancer.

Specifically, McNeill studies molecules that govern how cells make contact and communicate with one another. Called giant cadherins for their large size, these molecules play important roles in controlling the size of organs and in orchestrating how cells assemble themselves into complex tissues at precise times and with specific patterns and orientations. Her work also has implicated these molecules in cellular metabolism and the function of mitochondria, molecular powerhouses that manufacture a cells fuel supply. According to McNeills research, disruption of the giant cadherins can interfere with early embryonic development leading to, for example, neural tube defects that cause spina bifida or defects in the development of the kidney and urinary tract. Her work has identified cadherins as a culprit in congenital kidney diseases such as cystic kidney disease.

I am excited and honored to be joining Washington University School of Medicine as a BJC Investigator, McNeill said. Supporting research in the basic sciences is so important in making new discoveries and pushing the boundaries of what is known about human health and development. I thank the School of Medicine and BJC HealthCare for their commitment to supporting basic biomedical science in my own lab and in the labs of my fellow investigators.

Washington University School of Medicines 2,100 employed and volunteer faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is one of the leading medical research, teaching and patient-care institutions in the nation, currently ranked seventh in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

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First of 10 expected BJC Investigators named - Washington University School of Medicine in St. Louis

Harry Glorikian is an influential global business expert with more than three decades of experience building successful ventures in North America, Europe, Asia and the rest of the world. Harry is well known for achievements in life sciences, healthcare, diagnostics, healthcare IT and the convergence of these areas. He is a sought-after speaker, frequently quoted in the media, and regularly asked to assess, influence, and be part of innovative concepts and trends.

He is currently a General Partner at New Ventures Funds (NV). Before joining NV Funds, he served as an Entrepreneur In Residence to GE Ventures New Business Creation Group. He currently serves on the board of GeneNews Ltd. He also serves on the advisory board of Evidation Health (a digital health startup launched with support from GE Ventures), and several other companies. He is also a co-founder and an advisory board member of DrawBridge Health (a revolutionary diagnostics startup launched with support from GE Ventures).

Harry holds an MBA from Boston University and a bachelor's degree from San Francisco State University. Harry has addressed the NIH, Molecular Medicine Tri-Conference, World Theranostics Congress and other audiences, worldwide. He has authored numerous articles, appeared on CBS Evening News and been quoted regularly by Dow Jones, The Boston Globe, Los Angeles Times, London Independent, Medical Device Daily, Science Magazine, Genetic Engineering News and many others.

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Harry Glorikian - MedCity News

Microgravity conditions experienced by astronauts in space induce stress and strain around human cells. Cancer cells have also been known to die under microgravity. Scientists are exploring if this knowledge can be used to develop novel ways to find new drugs.

A group of Indian scientists has figured out the mechanism of cancer cells dying under microgravity, and believe that this can be used to find new drugs in future.

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The research group at Indian Institute of Technology Madras subjected cultured colorectal cancer cell lines to microgravity and observed that they die within 48 hours. Cancer cells died due to apoptosis, which is death induced by cancer cells themselves in response to stress. For simulating microgravity conditions, an equipment called Rotational Cell Culture System-High Aspect Ratio Vessel was used.

Cancer cells initiate their own death, it is also called programmed cell death. They did so by increasing the levels of two proteins called PTEN and FOXO3 and reducing the levels of another protein, Akt, when they experienced microgravity, researchers said.

When brought back to normal gravity conditions, they stopped dying and started proliferating again which is their normal state, Professor Rama Shanker Verma, who led the study, told India Science Wire. However, the time taken for cancer cells to start proliferating again was longer- nearly three weeks as opposed to less than a week when they do not experience any microgravity, added Raj Pranap Arun, a member of the research team which published its findings in journal Scientific Reports.

We can exploit the properties of cancer cells under microgravity to find potential drug targets, believes Professor Verma. The team is now extending the work to cancer stem cells that are responsible in cases of relapse.

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Seyed Ehtesham Hasnain, professor atJamia Hamdard Institute of Molecular Medicine, New Delhi, who is not connected to the study, commented that it is a long way to find a new drug against cancer. But this study has taken an interesting route to address a fundamental problem and may help identify novel drug targets to intervene against cancer.

The research team also included Divya Sivanesan and Prasanna Vidyasekar from IIT Madras and National University of Singapore. (India Science Wire)

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For Future Cancer Drugs, Scientists Look To Clues From Outer Space - Outlook India

MANHASSET, N.Y., Aug. 3, 2017 /PRNewswire-USNewswire/ --A study published in NeuroRehabilitation by Feinstein Institute for Medical Research scientist Bruce T. Volpe, MD and Johanna L. Chang found that isolated ankle training with a robotic therapy device can improve walking speed and balance after a stroke, depending on the severity of the patient's initial impairment. Better understanding of severity-dependent recovery profiles after stroke will help medical professionals determine the best candidates for robotic rehabilitation.

Strokes happen when a patient's brain does not receive sufficient blood supply due to a blockage or rupture of a blood vessel, which can result in impairment of motor or cognitive function. Patients typically participate in rehabilitation programs that focus on specific enhanced motor activity of their limbs under the direction of physical and occupational therapists.

Robotic devices are effective tools to aid in this recovery of, for example, wrist and arm movement. The devices have been less effective in encouraging recovery of walking speed (also known as gait). Dr. Volpe's study examines whether a robotic-assist device that uses interactive ankle movement in a seated position would improve a patient's walking speed and balance.

"Exercise is one of the main ways for patients who have had a stroke to regain movement," said Ms. Chang, who is the lead author of the study. "The use of robotic assisted-devices can enhance the therapy by increasing the intensity of the motor experience. This interactive robotic device moves the paralyzed arm or leg when the patient cannot and gets out of the way when the patient powers the movement. In our study, the baseline or initial walking speed prior to therapy was an important factor in predicting the final walking speed."

Twenty-nine study participants with a foot drop and walking speed abnormalities after stroke were treated three times a week for six weeks with robot-assisted ankle training. The patients were separated into three groups: high function (walking speed greater than 3 feet per second), medium function (1 foot per second) and low function (less than 1 foot per second). During a session, patients were seated in front of a video monitor and the ankle robot was attached at the knee and foot. The patient viewed the video screen that had a cursor and used their legs and ankle to move the cursor to reach a particular target.

After 18 sessions, the high and medium function groups demonstrated significant improvements in walking speed, with the high functioning group achieving a speed that is considered normal for ambulating patients in the community (greater than 4 feet per second). A further exciting result showed that in follow-up three months after the treatment finished, the high function group continued to improve (4.39 feet per second). The low functioning group demonstrated the greatest change in improved balance.

"Much like one medication is not effective for all patients with a certain condition, not all rehabilitation is beneficial to all," said Kevin J. Tracey, MD, president and CEO of the Feinstein Institute. "By understanding who can most benefit from robotic rehabilitation medical professionals can better tailor a program that will result in the highest benefit for patients."

About the Feinstein InstituteThe Feinstein Institute for Medical Research is the research arm of Northwell Health, the largest healthcare provider in New York. Home to 50 research laboratories and to clinical research throughout dozens of hospitals and outpatient facilities, the Feinstein includes 4,000 researchers and staff who are making breakthroughs in molecular medicine, genetics, oncology, brain research, mental health, autoimmunity, and bioelectronic medicine a new field of science that has the potential to revolutionize medicine. For more information about how we empower imagination and pioneer discovery, visit FeinsteinInstitute.org

Contact:Heather E. Ball 516-465-7917 rel="nofollow">hball@northwell.edu

View original content with multimedia:http://www.prnewswire.com/news-releases/feinstein-institute-study-finds-robotic-ankle-rehabilitation-helps-post-stroke-recovery-300499220.html

SOURCE Feinstein Institute for Medical Research

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Feinstein Institute study finds robotic ankle rehabilitation helps post stroke recovery - Markets Insider

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Wednesday, 2 August 2017 13:56

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Will there be a day birth to babies free of genetic disease, such as in a science-fiction film? Genes are carriers of a hereditary disease to have been fixed in human embryos for the first time thanks to a technique that raises as many hopes as well as ethical issues.

These works have been published Wednesday in the journal Nature. They are at a very preliminary stage, but potentially open up the way to major advances in the treatment of genetic diseases.

They also raise the ethical concerns worthy of a brave new world of Aldous Huxley. Because theoretically, this technique could be used to produce babies genetically modified to choose the color of their hair or to increase their physical strength.

The research on human embryos is strictly regulated and there was no question of implementing those of the study in a womans uterus to initiate a pregnancy. This is why scientists have not been allowed to develop over a few days.

This method, which still requires extensive research, can potentially be used to prevent the transmission of genetic diseases to future generations, commented during a press conference call one of the authors of the study, Paula Amato.

But this prospect is still distant: additional research, as well as an ethical debate will be needed before clinical trials, has taken care to specify the professor Amato.

Typo

The study was conducted within the university of the Sciences and of the Health of Oregon (OHSU) in the United States by american scientists, the chinese and south koreans. The tool used is the technique CRISPR-Cas9, a major discovery unveiled in 2012.

It is based on an enzyme that acts like molecular scissors. These can remove unwanted parts of the genome in a very precise way to replace them with new pieces of DNA, a bit like when one corrects a typo in a word processing software.

The team of researchers has used the revolutionary tool to correct, in human embryos, the gene carrier of the cardiomyopathy hypertrophic. This hereditary cardiac disease may cause sudden deaths, in particular during the practice of a sport.

The researchers have achieved in vitro fertilization of oocytes female normal by sperm carrying the defective gene. At the same time as the sperm, they also introduced the editing tools genetic.

The purpose: to cut the defective DNA to bring about its repair.

The result has been conclusive. 72% of the embryos (42 of 58) were adjusted so that the rate would have been 50% without the famous scissors genetic (naturally, the embryos would have had a chance on two to inherit a healthy gene).

A precedent in China

These tools can be further improved to achieve a success rate of 90 or even 100%, predicted another of the authors of the study, Shoukhrat Mitalipov.

A similar experiment had been conducted in China in 2015, but with mixed results. The phenomenon of mosaicism (simultaneous presence of genes in healthy and defective in embryo) could not be avoided, the researchers of the new study have been able to do.

The question that will be most debated is whether the same principle of modifying the genes of an embryo in vitro is acceptable, analyzed by an independent expert, professor Darren Griffin (University of Kent), quoted by the Science Media Centre.

According to him, one other question must be taken into account: is it morally right not to act if we have the technology to prevent these deadly diseases?

In December 2015, an international group of scientists convened by the us national Academy of sciences (NAS) in Washington had estimated that it would be irresponsible to use the technology of CRISPR to modify the embryo for therapeutic purposes as long as issues of safety and effectiveness have not been resolved.

But in march, the NAS and the american Academy of medicine were of the view that the advances in this area, opened up the realistic possibility that deserved serious consideration. In France, a parliamentary report has expressed a similar position.

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Molecular scissors to correct the genes: the medicine taken in the crossfire between hope and ethical debate - The Sherbrooke Times

August 1, 2017

A research team at the University of Bristol has won a prestigious international award for a technology that could help in the fight against antibiotic resistance.

One of the main driving forces behind the evolution of antibiotic resistance in bacteria is the inappropriate use of antibiotics. Providing doctors with rapid diagnostics to indicate which antibiotic to prescribe for a particular infection would reduce inappropriate antibiotic use and protect this valuable resource for the future. These rapid "antibiotic susceptibility tests" would also ensure that a patient receives a working antibiotic first time around, reducing the length and severity of their infection, and potentially saving their life.

A collaborative team from the University of Bristol has received a grant from the Longitude Prize Discovery Awards, which will enable them to further develop a portable device for rapid antimicrobial susceptibility testing. The team is working on a test that uses a unique system to monitor the responses of individual infection-causing bacteria to antibiotics. They have demonstrated the test's ability to determine, within 20 minutes, the effectiveness of a collection of antibiotics to kill infection-causing bacteria. Using this Discovery Award, the team will now work on a prototype machine to assist GPs when prescribing antibiotics in their practices.

The interdisciplinary team comprises academics and researchers from the School of Physics (Drs Massimo Antognozzi and Charlotte Bermingham); School of Cellular and Molecular Medicine (Dr Matthew Avison); Merchant Venturers School of Engineering (Drs Ruth Oulton and Krishna Coimbatore Balram); Bristol Medical School (Dr Helen Baxter) and NIHR CLAHRC West (Dr Niamh Redmond). The Avoidable Hospital Admissions Health Integration Team (ITHAcA HIT) is part of the wider collaboration supporting this work.

Dr Antognozzi, Senior Lecturer from the School of Physics and lead academic, said: "The team is excited to receive this award, which confirms the originality of our approach and gives us crucial support at this development stage."

Dr Avison, Reader in Molecular Bacteriology and impact lead for the EPSRC-funded BristolBridge AMR research consortium, which provided the initial seed-funding for this project, added: "The continuing development of this potentially transformative diagnostic device, and its receipt of this highly competitive international award, is testament to the interdisciplinary excellence of Bristol's antibiotic resistance research. We all look forward to seeing this project develop further."

Explore further: Fast break for antibiotic resistance testing

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Researchers develop a new test to assist GP antibiotic prescribing - Medical Xpress

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Foundation Medicine, Inc. (NASDAQ:FMI) today reported financial and operating results for its second quarter ended June 30, 2017. Results and business highlights for the quarter included:

Foundation Medicine delivered a strong second quarter, highlighted by record clinical volume and total revenue, increased adoption of our molecular information solutions by both clinicians and biopharma partners, and importantly, improved cancer care for the patients we serve, said Troy Cox, chief executive officer of Foundation Medicine. In addition, we advanced the parallel review with FDA and CMS for FoundationOne CDx with the submission of our final module to the FDA in June, a significant milestone in the process. If approved, FoundationOne CDx would become the first pan-cancer universal companion diagnostic, a highly differentiated and valuable solution for patients, oncologists and biopharma partners.

Foundation Medicine reported total revenue of $35.0 million in the second quarter of 2017, compared to $28.2 million in the second quarter of 2016. Revenue from biopharmaceutical customers was $22.1 million in the second quarter of 2017, compared to $18.9 million in the second quarter of 2016. The results of 4,762 tests were reported to biopharmaceutical customers in this years second quarter.

Revenue from clinical testing in the second quarter of 2017 was $12.9 million, compared to $9.4 million in the second quarter of 2016. The company reported 15,924 tests to clinicians in the second quarter of 2017, a 55% increase from the same quarter last year. This number includes 12,442 FoundationOne tests, 1,608 FoundationOne Heme tests, 1,594 FoundationACT tests, and 280 FoundationFocus CDxBRCA tests.

Total operating expenses for the second quarter of 2017 were approximately $57.7 million, compared with $45.5 million for the second quarter of 2016. The increase in operating expenses was partially driven by investments in the companys universal companion diagnostic assay, certain non-recurring cash and non-cash expenses, and investments in the companys technology infrastructure. Net loss was approximately $44.3 million in the second quarter of 2017, or a $1.24 loss per share. At June 30, 2017, the company held approximately $71.5 million in cash, cash equivalents and marketable securities.

The company will now be reporting revenue in two components: Molecular Information Services and Pharma Research and Development Services. Molecular Information Services is revenue derived from commercially available platforms and services such as sample profiling, data access and SmartTrials, and includes revenue from both clinical and biopharma customers. Pharma Research and Development Services is revenue derived from work funded primarily by biopharma partners to develop new assays and other services. This new disclosure is intended to provide additional information related to the revenue and cost of revenue specifically related to the companys commercially available platforms and services. During the second quarter, Molecular Information Services revenue was $30.3 million, including $12.9 million in revenue generated from our clinical customers, and $17.4 million in revenue generated from our biopharma customers. Pharma Research and Development Services revenue was $4.7 million.

On July 31st, Foundation Medicine entered into an agreement to expand its credit facility with Roche Finance from $100 million to $200 million. Any outstanding balance of the credit facility will convert to a term loan payable over a five-year period beginning on February 2, 2021. No funds were drawn under the credit facility at the time of the expansion. The company intends to use the proceeds to further fund product development, commercialization, corporate development initiatives and working capital.

2017 Outlook

Foundation Medicines business and financial outlook for 2017 is the following:

Conference Call and Webcast Details

The company will conduct a conference call today, Tuesday, August 1st at 4:30 p.m. Eastern Time to discuss its financial performance for the 2017 second quarter and other business activities, including matters related to future performance. To access the conference call via phone, dial 1-877-270-2148 from the United States or dial 1-412-902-6510 internationally. Dial in approximately ten minutes prior to the start of the call. The live, listen-only webcast of the conference call may be accessed by visiting the investors section of the companys website at investors.foundationmedicine.com. A replay of the webcast will be available shortly after the conclusion of the call and will be archived on the company's website for two weeks following the call.

About Foundation Medicine Foundation Medicine (NASDAQ:FMI) is a molecular information company dedicated to a transformation in cancer care in which treatment is informed by a deep understanding of the genomic changes that contribute to each patients unique cancer. The company offers a full suite of comprehensive genomic profiling assays to identify the molecular alterations in a patients cancer and match them with relevant targeted therapies, immunotherapies and clinical trials. Foundation Medicines molecular information platform aims to improve day-to-day care for patients by serving the needs of clinicians, academic researchers and drug developers to help advance the science of molecular medicine in cancer. For more information, please visit http://www.FoundationMedicine.com or follow Foundation Medicine on Twitter (@FoundationATCG).

Foundation Medicine, FoundationOneand FoundationACTare registered trademarks, and FoundationOne CDxand FoundationFocusare trademarks, of Foundation Medicine, Inc.

Cautionary Note Regarding Forward-Looking Statements This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding the value of the companys business; the benefits of our products to physicians, biopharmaceutical companies, payers and patients in the treatment of cancer and personalized cancer care; the companys financial and operational forecasts, including projections regarding the generation of revenue, the number of tests to be conducted, the incurrence of operating expenses, the timing of product development, and the expansion of reimbursement progress, including any changes to any earlier guidance; the benefits provided by aFDA-approved and CMS-covered FoundationOne CDx and progress with the Parallel Review process withFDAand CMS; the scope and timing of any approval of our universal companion diagnostic assay as a medical device by theFDAand any coverage decision by CMS; strategies for achievingMedicarecoverage decisions at the local or national level and new and expanded coverage from third-party payers; and use of any funds from its credit facility.All such 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 such forward-looking statements. These risks and uncertainties include the risks thattheFDA does not approve our universal companion diagnostic assay as a medical device or that CMS does not decide to offer our universal companion diagnostic assay as a covered benefit underMedicare; theFDAor CMS is delayed in the completion of the Parallel Review process; the company's new facilities inNorth CarolinaandGermanydo not facilitate the company's ability to achieve it business objectives; the company's distribution partner outsidethe United Statesis not able to achieve market penetration in new and existing markets as quickly or as extensively as projected;Foundation Medicine'srelationships withthird-party or government payers do not increase or expand;Foundation Medicineis unable to sustain or grow relationships with biopharmaceutical partners; the company's revenue, test or operating expense projections may turn out to be inaccurate because of the preliminary nature of the forecasts; the company's expectations and beliefs regarding the future conduct and growth of the company's business are inaccurate; Foundation Medicineis unable to achieve profitability, to compete successfully, to manage its growth, or to develop its molecular information platform;and the risks described under the caption Risk Factors in Foundation Medicines Annual Report on Form 10-K for the year ended December 31, 2016, which is on file with the Securities and Exchange Commission, as well as other risks detailed in Foundation Medicines subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Foundation Medicine undertakes no duty to update this information unless required by law.

- Financial Tables to Follow -

FOUNDATION MEDICINE, INC.

Condensed Consolidated Balance Sheets

(In thousands)

(unaudited)

FOUNDATION MEDICINE, INC.

Condensed Consolidated Statements of Operations

(In thousands, except share and per share data)

(unaudited)

(44,258)

(28,994)

(46,301)

(1.24)

(0.84)

(2.55)

(1.34)

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Foundation Medicine Announces 2017 Second Quarter Results and Recent Highlights - Business Wire (press release)

August 1, 2017 Credit: martha sexton/public domain

A study by a research team at the Max Delbrck Center in Berlin that appears in the journal PNAS has found a group of neuronal cells in the brain stem that coordinate exhalation and tension of muscles in the larynx of baby mice without which they are mute. The cries of human babies may well depend on similar connections, which could also be impaired in speech disorders.

Almost immediately after birth, mouse pups that are separated from their mother are able to make calls to summon her. The generation of these calls requires vigorous exhalation and the tensioning of laryngeal muscles, which requires the coordinated activity of two muscle groups. This is achieved by neurons in the brainstem, according to a study by Carmen Birchmeier's lab at the Max Delbrck Center for Molecular Medicine in the Helmholtz Association (MDC).

In series of experiments, the researchers have shown that the cells of the nucleus tractus solitarii (NTS) are linked to cells that control tension in the abdominal muscles, enabling vigorous exhalation, and the muscles in the larynx. The nucleus also receives sensory information from the vocal folds, the tongue and the lung. During vocalization, it coordinates sensory inputs and motor outputs. However, if the genes for the transcription factors Olig3 or Tlx3 are mutated, the nerve cells in this particular nucleus cannot mature properly in the fetus. Without it, the pups cannot vocalize after birth.

The mother ignores mute offspring

Newborn mice need proximity to their mother for survival. As soon as a newborn mouse pup escapes the safety of the nest, it emits salvos of four to six calls with a frequency of 75 kHz. These sounds are not audible to the human ear. During each call, the newborn mouse exhales deeply. The mother responds immediately, looking for her lost pup and reuniting it with the rest of the litter. Even recorded ultrasound calls will prompt her to seek her offspring. If a baby mouse in distress is unable to emit these calls, the mother cannot respond.

"We suspect that the calls are an evolutionarily conserved signal that indicates the offsprings' fitness and health," Carmen Birchmeier says. "The mute mice are also a model for investigating the importance of vocalization for the interaction between mother and baby," first author Luis Hernandez-Miranda says.

Another theory is that the functional faults in the nucleus could be involved in the development and manifestation of speech disorders, which are often seen in patients after strokes, those who have tumors or are suffering from neurodegenerative diseases.

Explore further: Gut microbiome of mother found to impact immunity of mice pups

More information: Luis Rodrigo Hernandez-Miranda et al, Genetic identification of a hindbrain nucleus essential for innate vocalization, Proceedings of the National Academy of Sciences (2017). DOI: 10.1073/pnas.1702893114

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The importance of vocalizations between mice and their offspring - Medical Xpress

(Reuters Health) - - People with severe symptoms of chronic fatigue syndrome have a molecular signature in their blood made up of 17 immune system signaling molecules that are elevated, which may provide insight into how inflammation contributes to the mysterious condition.

Inflammation is the immune systems response to injuries and illnesses. The study identified variations in molecules known as cytokines that are responsible for sending messages and sounding an alarm, including several that can trigger inflammation.

Chronic fatigue syndrome can involve years of crippling pain and exhaustion, but is a controversial diagnosis because patients cant take a test to prove they have it. The current study results build on earlier experiments linking inflammation to the condition and might one day help create a blood test to diagnose it or lead to effective treatments, researchers say.

What is at stake here is proof of concept that this disease is real, said lead study author Dr. Jose G. Montoya of Stanford University School of Medicine in California.

Patients have been humiliated, ostracized, and ignored, Montoya said by email.

Millions of people worldwide, including more than 1 million in the U.S. alone, suffer from chronic fatigue syndrome, the researchers note in the Proceedings of the National Academy of Sciences.

Doctors may diagnose the condition, also called myalgic encephalomyelitis, when people have at least six months of debilitating fatigue that cant be explained by other causes and make it difficult for them to keep up with school, work or social activities. Symptoms can appear in different combinations and intensities and may include sleep problems, cognitive impairment, fever, sore throat, or sensitivity to noise, light or certain foods.

The National Academy of Medicine recently proposed a new name for the condition, systemic exertion intolerance disease (SEID), because of its hallmark symptom of crushing fatigue after any kind of exertion, the authors note.

Many, but not all, chronic fatigue syndrome patients experience flu-like symptoms common in diseases caused by inflammation, they add.

For the current study, researchers examined blood samples from 192 patients with chronic fatigue syndrome and a control group of 392 healthy people.

The scientists found that some cytokine levels were lower in patients with mild forms of chronic fatigue syndrome than in the group of healthy individuals, but higher in people with severe symptoms of chronic fatigue syndrome.

One protein in particular, tumor growth factor beta (TGF-beta), was higher in patients with chronic fatigue syndrome than in the healthy controls, while another protein, resistin was lower.

However, the study team also found that the concentrations of 17 of the 51 cytokines they examined were associated with disease severity. Thirteen of those 17 cytokines are pro-inflammatory, the authors note.

Taken together, the findings build on previous research demonstrating that chronic fatigue syndrome is a real illness, and not something patients make up or experience only for psychological reasons, experts say.

Many people with (chronic fatigue syndrome) report that their illness began with symptoms associated with a typical respiratory infection including fever, sore throat, swollen lymph nodes, muscle weakness and fatigue, but the clinical course was atypical and they never fully recovered, said Dr. Ian Lipkin, a researcher at the Mailman School of Public Health at Columbia University in New York.

Many of these symptoms can be explained by circulating immune system molecules described by Montoya and colleagues in this paper, as well as by others who have reported similar findings, Lipkin, who wasnt involved in the study, said by email.

The development of a biomarker such as a diagnostic blood test for chronic fatigue syndrome would be a major advance in understanding the condition and treating it, said Dr. Shaheen Lakhan, a neurology researcher at California University of Science and Medicine in San Bernardino who wasnt involved in the study.

We currently do not have such a test, Lakhan said by email. Not only will it permit objective diagnosis, it may guide new drug development and be used to monitor disease activity and responsiveness to treatments.

SOURCE: bit.ly/2tY29nF Proceedings of the National Academy of Sciences, online July 31, 2017.

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Molecular profile hints at inflammatory processes in chronic fatigue - Reuters

Newswise SAN DIEGO AACC announced today that it will launch a new laboratory medicine conference and expedition next spring AACC Middle East. AACC Middle East will showcase AACCs globally-renowned education and scientific programs paired with a dynamic exposition in Abu Dhabi, United Arab Emirates (UAE), March 22-24, 2018.

With this new event, a partnership between AACC and Al Borg Medical Laboratories, AACC continues its strategy to provide gold standard, actionable information about the latest in clinical testing to a global audience -- advancing patient care and improving health outcomes worldwide.

The program and exposition floor of AACC Middle East will feature the latest breakthroughs in every area of clinical testing, including mobile health, molecular diagnostics, mass spectrometry, next generation sequencing, point of care, and automation. The scientific program will feature experts from the U.S. and the Middle East, sharing knowledge and innovation. Attendees will meet and network with experts in the field, and engage with their peers during the scientific program highlighting recent advances in laboratory medicine.

"Healthcare is increasingly global and interconnected," said AACC CEO Janet B. Kreizman. "The AACC leadership is excited to bring forward this new opportunity for laboratory experts from a range of healthcare settings to learn about the latest innovations in clinical testing and engage with like-minded professionals from the Middle East and nearby regions. We expect that all the major players in the IVD industry will want to be a part of this groundbreaking event.

About AACC Middle East

AACC Middle East offers 3 days packed with opportunities to connect with global leaders in clinical chemistry, molecular diagnostics, mass spectrometry, translational medicine, lab management, and other areas of breaking science in laboratory medicine. The meeting will occur from March 22-24, 2018, at Etihad Towers in Abu Dhabi, UAE.

About AACC

Dedicated to achieving better health through laboratory medicine, AACC brings together more than 50,000 clinical laboratory professionals, physicians, research scientists, and business leaders from around the world focused on clinical chemistry, molecular diagnostics, mass spectrometry, translational medicine, lab management, and other areas of progressing laboratory science. Since 1948, AACC has worked to advance the common interests of the field, providing programs that advance scientific collaboration, knowledge, expertise, and innovation. For more information, visit http://www.aacc.org.

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AACC Launches AACC Middle East in Abu Dhabi - Newswise (press release)

Researchers at Delhis CSIR-Institute of Genomics and Integrative Biology (CSIR-IGIB) have found the mechanism by which controlling the levels of telomerase can help in reining in the growth of cancer cells and probably prevent cancer metastasis. The results were published in the Journal of Biological Chemistry.

Unlike normal cells, most cancer cells have high levels of telomerase and this leads to more than normal length of the telomere. Telomeres protect chromosome ends somewhat like the plastic clips at the end of shoelaces that prevent fraying of the ends. While cells die when the telomere becomes shorter beyond a certain limit, in the case of cancer cells the length of the telomere is maintained thereby ensuring extended life span of the cells.

In normal cells the telomerase is kept under tight control. But in about 85% of all cancers the telomerase levels are more than normal leading to malignant transformation and aggressive metastasis in many cases. It is not clearly understood how telomerase is kept under tight control in normal cells and how the telomerase levels gets increased in cancerous cells, says Dr. Shantanu Chowdhury from the Genomics and Molecular Medicine Unit at IGIB and the corresponding author of the paper.

It is already known that when the amount of a particular protein that suppresses the spread of cancer (metastasis) called nonmetastatic 2 (NME2) is high the tendency of the cancer to spread is low. But what came as a surprise is the role of this protein in controlling the telomerase levels as well. How NME2 controls metastasis is not clearly understood. But surprisingly we found that NME2 controls the levels of telomerase, Dr. Chowdhury says.

The mechanism

The researchers found that NME2 binds to a DNA structure (G-quadrauplex) found in the telomerase promoter. Once bound, the NME2 facilitates a well known suppressor of gene expression (REST complex) to bind to the telomerase promoter and control the production of telomerase.

Experiments show that if you dont have NME2 then the REST suppressor cannot bind to the telomerase promoter and control the production of telomerase, says Dhurjhoti Saha from IGIB and one of the first authors of the paper.

We used proteomics approach to study the protein-protein interactions. We could identify protein members of the REST complex that interact with NME2. The IGIB team then confirmed the role of the REST complex and its function, says Dr. Ramesh Ummanni, from the Centre for Chemical Biology at the CSIR-Indian Institute of Chemical Technology (CSIR-IICT), Hyderabad and a co-author of the paper.

Drug target

We established that the DNA structure (G-quadrauplex) could be a possible drug target once we understood the mechanism of NME2 binding to the promoter followed by the REST suppressor complex, Dr. Chowdhury says. The involvement of a DNA structural architecture allowed the team to use small molecules that recognised the specific structure.

Since the amount of NME2 is low in many metastatic cancerous cells, the researchers used small molecules that were able to function like NME2 by recognising and binding to the DNA structure. We screened 20 molecules and 11 were able to bring down the telomerase level in fibrosarcoma cancer cells, Dr. Chowdhury says.

Based on the initial lead from the small molecules, the researchers are planning to synthesise new molecules to optimise for drug-like characteristics for therapeutic use. The molecules will then be tested on animals.

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IGIB researchers rein in cancer cells - The Hindu

COLUMBIA Mun Choi, the UM System president, was authorized Friday to send in a state funding request for educational buildings at each campus.

The Board of Curators met Friday morning in Ellis library's teleconference room, and they voted unanimously for Choi to send in the request. The request is for fiscal year 2019, which will begin on July 1, 2018, so the talks revolved around potential funding after that date.

The request listed priority projects for each of the four UM system campuses. The main item of discussion, and the most expensive of the projects, was MU's planned Translational Precision Medicine Complex.

Translational medicine focuses on research to discover new ways of diagnosing or treating health problems and also instituting those new techniques on actual patients.

With the recent big cuts made to the Missouri higher education budget, the curators said it is unlikely that these projects will be immediately funded, but they believe it is important for their request to be officially made nonetheless.

MU's translational medicine complex is expected to cost about $250 million, $100 million coming from the school and $150 million asked for from the state. It is estimated to generate over $500 million in economic impact and create 3,860 jobs, according to the appropriations request.

MU has taken preliminary steps for the new translational medicine complex to be built on the site of the former International Institute of Nano and Molecular Medicine,which was closed at the end of June.

MU is one of the 62 members of the Association of American Universities. This means it is considered a leading research institution across the U.S. and Canada. The other three UM System campuses benefit from MU's status as an AAU university, so investing in a new research facility at MU helps the whole system indirectly, curators said. For this reason, the translational medicine complex is the top capital funding priority for the system, they said.

Several times throughout the meeting the curators referenced their July 18 and 19 retreat in Columbia, which Maurice Graham, the chair of the board of curators, described as one of the most constructive curator meetings he has ever participated in. At Friday's meeting, Graham emphasized the need for the capital appropriations request to include projects from all four campuses in order to continue the spirit of intra-system cooperation that was a focus of that retreat.

All four campuses are represented in the appropriations request. The Kansas City campus and Missouri University of Science and Technology are each requesting funds for renovations of their chemistry and biological sciences buildings, and the St. Louis campus is requesting funds for "space consolidation and infrastructure."

Ryan Wrapp, the system's chief financial officer, emphasized the need for the system to move away from a reliance on state funding for school buildings. In the past, the state would fund new buildings entirely, he said, but now they need to move toward targeted fundraising, partnerships with private businesses, or fund-matching with the state.

Curator Jeffrey Layman pointed out the possibility of a future federal infrastructure bill that could provide additional funding for public universities. President Donald Trump has repeatedly called for such a bill, though lately Congress has been mainly focused on health care, and the president has not unveiled any major infrastructure bill.

Choi described the request to the state as the first step in a dialogue and as a "give and take." With the four campuses' top priorities formally submitted to the state, when there is money to spend the state will know exactly what the campuses hope to use it for, he said.

It was pointed out that, historically, funding has come to the system often when it is not expected, so the process of requesting the funding is still important even if it feels extremely unlikely.

Supervising editor is Sky Chadde.

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UM Board of Curators asks state for $150 million for MU project ... - Columbia Missourian

Facebook CEO Mark Zuckerberg and his wife, Dr. Priscilla Chan, will contribute $10 million to UCSF to help fund an effort to merge data on 15 million patients across five UC medical campuses into one database.

The investment highlights the interest from investors and researchers in applying artificial intelligence to health data. The goal is to detect patterns in disease development and to allow doctors to better develop treatment plans for patients.

In oncology, for instance, computers could mine patient data to try to predict whether women diagnosed with ovarian cancer who stop responding to one type of drug may be more likely to respond to another type of treatment, based on previous cases.

The $10 million contribution is separate from the commitment by the couples limited liability company, the Chan Zuckerberg Initiative, to invest $3 billion over the next decade to cure disease.

It will go toward UCSFs Institute of Computational Health Sciences. In addition to merging data from health records, it will be used to hire faculty members for the institute over the next five years, said Dr. Atul Butte, the institutes director.

Big data and machine learning is hot in medicine right now, Butte said. If you want machine learning to work, you need to see many, many cases before you can learn the patterns.

The soon-to-be-merged data is from electronic health records that are housed separately at UCSF, UCLA, UC Irvine, UC San Diego and UC Davis, dating back between five and nine years, Butte said.

While UCSF would have access to identifiable patient information, such as names, patient privacy laws require researchers to get authorization from patients, or approval from UCSFs Institutional Review Board, before accessing any identifiable data.

Artificial intelligence in health and drug development is a booming area. Emerging companies like Londons Benevolent AI, San Brunos Numerate and Menlo Parks NuMedii co-founded by Butte have attracted hundreds of millions of dollars from investors over the last several years.

Alphabets health subsidiary Verily, formerly Google Life Sciences, recently launched a study to track health information from 10,000 people. IBMs Watson Health uses algorithms to sift through patient records and research papers from medical journals to help doctors diagnose and treat diseases. Amazon has assembled a team to build tools for electronic health records data, CNBC reported this week.

The major tech titans are moving into this space at full tilt, said Dr. Eric Topol, a professor of molecular medicine at the Scripps Research Institute. They realize this has unparalleled growth potential.

Artificial intelligence in medicine is taking off because until recently there wasnt enough data to draw meaningful conclusions, experts said. But improvements in genomic sequencing and medical monitoring technology are quickly changing that. Every persons genomic sequence alone generates billions of data points. Add that to the data collected by wearable devices such as monitoring tools that track glucose levels, blood pressure, heart rhythm and other measures and researchers have a rich pool of health information to parse.

Thats why this is a particularly exciting era in medicine, Topol said. Its really about having enormous data sets, not just a one-off, but on a continuous basis.

The challenge, though, is cutting through the noise in ways that will enable physicians to zero in on individualized screening, treatment and prevention plans for patients.

Just having all this data is not so important, Topol said. Its processing it, working with it to change the future of medicine. Artificial intelligence could result in this promise of true prevention or far better treatments.

Catherine Ho is a San Francisco Chronicle staff writer. Email: cho@sfchronicle.com Twitter: @Cat_Ho

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Zuckerberg, Chan give UCSF $10 million for health data research - San Francisco Chronicle

KOCHI: A two-day conference, beginning on August 6, on integrative Ayurveda and modern medicine, titled Amrita Samyogam 2017, is being held in collaboration with Amrita Universitys School of Ayurveda. More than 60 experts and 1,000 delegates from around the world will be taking part. It will be inaugurated by the Union Minister of State for AYUSH, Shripad Yasso Naik.

The event will bring together allopathic doctors, Ayurveda practitioners and modern scientists on a common platform. It will identify strategies for integrating Ayurveda with Allopathy in the management of cancer, auto-immune diseases like arthritis, diabetes, neuro-degenerative diseases, and mental health. The conference will demonstrate how integrative medicine can be made a reality through examples of clinical integration, basic science studies, and application of new technologies.

Said Prof. Shantikumar Nair, Director, Centre for Nanosciences & Molecular Medicine, Amrita University: Integrating Indias ancient tradition of Ayurveda with evidence-based modern medicine has the potential to revolutionise world healthcare. Integrative medicine is becoming a popular specialty among physicians in Western countries because of the myriad ways in which it can benefit patients. Dr Nair says that it focuses on healing the person in his entirety rather than merely treating the symptoms by investigating the root cause of illness. It is much more patient-centric and can positively impact chronic and lifestyle diseases for which modern medicine has no answer. Western medicine and Indian ancient healing sciences can be a win-win combination to effectively tackle the enormous healthcare challenges facing humanity, says Nair.

The event is expected to trigger important collaborations across the world in the field of integrative medicine, especially academic collaborations and funding opportunities.

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Conference on integrative ayurveda - The New Indian Express

An experimental technique created a model that could kick-start the development process for new drugs targeting Clostridium difficile infections (C. Diff).

Scientists based at Virginia Techs Biocomplexity Institute harnessed a mix of algorithms, simulations, and machine learning to test and predict the efficacy of novel treatments for infectious and immune-mediated diseases.

A modeling system of this nature could be particularly important when it comes to predicting the progression and treatment response to C. Diff. Antibiotics are the standard form of treatment for C. Diff, but they run the risk of perpetuating drug-resistant bacterial strains.

High rates of recurrence can lead to an uptick in healthcare costs and mortality.

The research team used this model to identify a potential alternative treatment for C. Diff, which is a protein called lanthionine synthetase c-like or LANCL2.

Our modeling shows that we do not need to remove the pathogen nor directly influence inflammation in the case of CDI to have an effective treatment, said one of the study authors Andrew Leber, scientific director at BioTherapeutics, in a statement. Simply restoring immune tolerance in the gut through an LANCL2, or similar immunoregulatory pathway, or boosting the gut microbiome to allow it to naturally outcompete pathogenic C. difficile strains is effective in the absence of antibiotics.

This new model could be the first step in constructing a personalized disease treatment process for these conditions by translating preclinical results in animal models to clinical outcomes, pinpointing effective treatments, analyzing dosage effects, and forecasting patient reactions to combination therapies.

The convergence of advanced data analytics, modeling, and artificial intelligence systems with high resolution, large-scale patient data creates an opportunity to fundamentally transform how medicine will be practiced, said Josep Bassaganya-Riera, director of the Nutritional Immunology and Molecular Medicine Laboratory and CEO of BioTherapeutics, in a statement. In this study and in our continuing efforts, we aim to be a leader in this developing field of precision, personalized medicine in infectious and autoimmune diseases.

Refining this model could ultimately minimize undesirable side effects and enable maximal efficacy of treatment in response to C. Diff and similar conditions.

The study appeared in the journal Artificial Intelligence in Medicine.

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Precision Medicine Method Could Lead to New C. Diff Treatment - R & D Magazine

Scientists have successfully regenerated cells in the retina of adult mice at the University of Washington School of Medicine in Seattle.

Their results raise the hope that someday it may be possible to repair retinas damaged by trauma, glaucoma and other eye diseases. Their efforts are part of the UW Medicine Institute for Stem Cell and Regenerative Medicine.

Many tissues of our bodies, such as our skin, can heal because they contain stem cells that can divide and differentiate into the type of cells needed to repair damaged tissue. The cells of our retinas, however, lack this ability to regenerate. As a consequence, injury to the retina often leads to permanent vision loss.

This is not the case, however, in zebrafish, which have a remarkable ability to regenerate damaged tissue, including neural tissue like the retina. This is possible because the zebrafish retina contains cells called Mller glia that harbor a gene that allows them to regenerate. When these cells sense that the retina has been injured, they turn on this gene, called Ascl1.

The gene codes for a type of protein called a transcription factor. It can affect the activity of many other genes and, therefore, have a major effect on cell function. In the case of the zebrafish, activation of Ascl1 essentially reprograms the glia into stem cells that can change to become all the cell types needed to repair the retina and restore sight.

The team of researchers in the new study were led by Tom Reh, University of Washington School of Medicine professor of biological structure. The scientists wanted see whether it was possible to use this gene to reprogram Mller glia in adult mice. The researchers hoped to prompt a regeneration that doesn't happen naturally in mammal's retina.

Their research findings appear online July 26 in the journal Nature. The lead author is Nikolas Jorstad, a doctoral student in biological structure and in the Molecular Medicine and Mechanisms of Disease program in the Department of Pathology.

Other UW Medicine researchers on the study are Matthew S. Wilken, Stefanie G. Wohl, Leah S. VandenBosch, Takeshi Yoshimatsu, William N. Grimes,Rachel O. Wong, all from the UW Department of Biological Structure, and Fred Rieke from the UW Department of Physiology and Biophysics and the Howard Hughes Medical Research Institute.

Like humans, mice cannot repair their retinas. Jorstad said that to conduct their experiment, the team "took a page from the zebrafish playbook." They created a mouse that had a version of the Ascl1 gene in its Mller glia. The gene was then turned on with an injection of the drug tamoxifen.

Earlier studies by the team had shown that when they activated the gene, the Mller glia would differentiated into retinal cells known as interneurons after an injury to the retina of these mice. These cells play a vital role in sight. They receive and process signals from the retina's light-detecting cells, the rods and the cones, and transmit them to another set of cells that, in turn, transfer the information to the brain.

In their earlier research, however, the researchers found that activating the gene worked only during the first two weeks after birth. Any later, and the mice could no longer repair their retinas. Reh said that at first they thought another transcription factor was involved. Eventually they determined that genes critical to the Mller glia regeneration were being blocked by molecules that bind to chromosomes. This is one way cells "lock up" genes to keep them from being activated. It is a form of epigenetic regulation -- the control of how and when parts of the genome operate.

In their new paper, Reh and his colleagues show that, by using a drug that blocks epigenetic regulation called a histone deacetylase inhibitor, activation of Ascl1 allows the Mller glia in adult mice to differentiate into functioning interneurons. The researchers demonstrated that these new interneurons integrate into the existing retina, establish connections with other retinal cells, and react normally to signals from the light-detecting retinal cells.

Reh said his team hopes to find out if there are other factors that can be activated to allow the Mller glia to regenerate into all the different cell types of the retina. If so, it might be possible, he said, to develop treatments that can repair retinal damage, which is responsible for several common causes of vision loss.

This article has been republished frommaterialsprovided by the University of Washington. Note: material may have been edited for length and content. For further information, please contact the cited source.

Reference:

Jorstad, N. L., Wilken, M. S., Grimes, W. N., Wohl, S. G., Vandenbosch, L. S., Yoshimatsu, T., . . . Reh, T. A. (2017). Stimulation of functional neuronal regeneration from Mller glia in adult mice. Nature. doi:10.1038/nature23283

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Retinal Cells Regenerated in Mice - Technology Networks

Imagine youre an oncologist faced with a difficult treatment decision for a patient. At your computer, you sift through all the data in their electronic health recordtheir demographics, cancer diagnosis, and clinical history. But one critical component is missing: the patients molecular profile, which is the key to personalized treatments. Then you hear the screech of the fax machine.

Most oncologists dont have to imagine this. Its standard practice for molecular test results to be faxed, or downloaded as a PDF then printed in a wide range of layouts that vary by testing provider. This makes it nearly impossible for oncologists to take full advantage of this crucial data, and deprives patients of the best care.

Molecular testing labs use some of the most advanced software-connected machine and analytics on earth, yet have managed to turn structured digital genomic data into paper!

At MedCitys CONVERGE conferenceJuly 31-August 1 in Philadelphia next week, Im speaking on this issue and other challenges that are holding back precision medicine.

Precision medicine already works, and health systems are using it successfully today to deliver better treatments to patients. But to truly scale precision medicine to providers across the country and fight cancer today, we need to modernize how molecular test results are structured, transferred, and shared. This would give providers, payers, and policymakers the real-world evidence needed to shift rapidly toward precision medicine.

Molecular tests, which give critical insights into the genomic makeup of a patients condition, are the foundation of precision medicine. But theyre newer and more complex than other lab tests, and the result data are not standardized, portable, consistent, or structured like common blood tests and almost all other routine lab results.

Standardizing this data will enable multiple health systems to communicate and exchange information. This improves the quality of care by helping physicians uncover new insights and saving them time that they can dedicate to more critical tasks.

Oncologists need to be able to easily access molecular data in the same system as the patients clinical datanot as PDF attachments or faxesso that patients can get the personalized care they deserve. They can then make more effective treatment decisions, and more accurately and quickly match patients to the right drugs or right clinical trials at the right time. Precision medicine is already changing how health systems treat certain forms of cancer, and standardizing molecular data will accelerate this.

Standardizing molecular data has broader implications, too.

One of the biggest questions around precision medicine is the economics. Can you deliver it cost-effectively? The answer is yes, and many case studies demonstrate the value to patients, providers, and payers. But for payers to buy in, they need real-world evidence on a much larger scale. Standardizing molecular data would help address this.

It would also facilitate better data sharing among providers. In the past year, health systems have rapidly moved toward sharing de-identified cancer data, largely spurred by former Vice President Bidens Cancer Moonshot initiative. But you cant share data at any meaningful scale when that data is faxed from department to department.

We now know that precision medicine not only works but can lead to better outcomes and quality of life. These benefits will expand exponentially as more providers adopt precision medicine, as they will have a much larger pool of real-world data to inform their insights. Now we need to make it easiernot harderfor organizations to access and share the real-world data that will enable this shift.

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Throw out the fax machines: The cancer world needs to modernize molecular test reporting - MedCity News

NEW HAVEN A Yale neurology and neuroscience professor who hopes to regrow neurons in patients with spinal cord injuries has secured enough funding for a human clinical trial.

Stephen Strittmatters New Haven-based company, ReNetX, also has a new CEO, Erika Smith, who joined the biomedical venture in June after leaving her post as director of Yale Universitys $10-million Blavatnik Fund for Innovation.

The company has raised about $20 million from the National Institutes of Health and other sources to carry out the first stages of a trial involving people with tetraplegia, paralysis of all four limbs and the torso.

Strittmatter said his approach works like a double negative. He identified a receptor that inhibits the growth of nerve fibers and then created a decoy to block it. That leaves the neurons free to grow or regrow, in this case naturally, as they do in early development.

We have this huge hospital and medical complex, Strittmatter said last week at his office in the Boyer Center for Molecular Medicine, 2 miles from the Yale New Haven Hospital Spine Center. We dont have a single drug that promotes neuroconnection. So if we get it to a trial and if the trials successful, it opens up a huge number of doors to all kinds of additional therapies and multiple diseases.

Yale

Erika Smith, CEO of ReNetX and former director of the Yale Blavatnik Fund for Innovation.

Erika Smith, CEO of ReNetX and former director of the Yale Blavatnik Fund for Innovation. (Yale)

ReNetX, formerly known as Axerion Therapeutics, went public with its new name, its funding progress and Smiths hire on Monday, though she joined the company June 30.

Sitting outside a New Haven coffee shop the next week, she recalled how people have told her the new logo looks retro, a bit 90s, with its teal and blue, space-age lettering.

I said, I like that. Its kind of Star Treky like this brave new world and thats kind of how we think about the company, too, Smith said.

Strittmatter the companys founder and science adviser is certainly exploring one of the final frontiers in medicine. No treatments available today can regenerate nerve cells in the adult brain and spinal cord, where neuron regrowth is extremely limited.

Spinal cord injury is one of the most significant unmet medical needs with an annual cost of more than $5 billion per year, Smith said. A treatment that could mitigate even only a part of the condition could improve quality of life of these patients.

ReNetXs treatment would be delivered as an injection into the spinal column, similar to an epidural.

The trial, which is pending regulatory approval, could begin in as little as 15 months and, if successful, the treatment could begin to restore some sensation and control to patients limbs within six months to a year. From there, the company would seek to apply the therapy to glaucoma and then stroke damage.

I wish things could move faster but on the other hand, its very encouraging to see the progress we do have, Strittmatter said, adding that he and others have spent 20 years trying to solve the problem of nerve fiber disconnection.

Its really sort of the culmination of probably hundreds of peoples work, hundreds of man-hours, woman-hours, he said. But thats what it takes to actually make a difference.

The fact that Smith has joined the company as CEO is further evidence that Strittmatters work is one step closer to leaving the lab for the marketplace.

Rebecca Lurye

Stephen Strittmatter, a neuroscience and neurology professor at Yale University and founder of ReNetX, works in his New Haven laboratory on Friday, July 21.

Stephen Strittmatter, a neuroscience and neurology professor at Yale University and founder of ReNetX, works in his New Haven laboratory on Friday, July 21. (Rebecca Lurye)

The two met when Strittmatter was applying for a grant from the fund that she directed, which seeks to bridge the gap between breakthrough research and commercialization. As Smith says, the fund tries to prevent an innovation from existing as just a paper.

Let it be something thats life changing, she said.

Strittmatter won the grant in May, $300,000 toward another of his areas of interest, Alzheimers disease. Soon after, Smith learned there was an opening for the top job at his company.

Shed spent more than 25 years as an investor and entrepreneur in life sciences, both at Yale and as senior director of investments with the Center for Innovative Technologies.

But no company tempted her away from her behind-the-scenes roles until ReNetX.

Theres amazing companies I had a chance to engage with, but this was just the right one at the right time with the right team, she said. It was just serendipity.

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Yale Scientist Aiming To Reverse Spinal Cord Injuries - Hartford Courant

The American Heart Association (AHA) has awarded Benjamin L. Prosser, PhD, a professor at the University of Pennsylvania, its Outstanding Early Career Investigator Award.

The AHA gave him the award to recognize his work on how to improve cardiac function in heart failure patients, according to a July 26 press release. He presented it earlier this month at the Councils 2017 Basic Cardiovascular Sciences meeting in Portland, Oregon.

His research demonstrated that by softening the internal cytoskeleton of heart cells in patients with heart failure, they could make their heart beats stronger.

In other research published last by Prosser, he found that in a study on rodents, microtubules provide sufficient, but not excessive resistance in health heart muscle. The findings were published in Science.

Prosser earned his bachelors degree in health and exercise from Wake Forest University in Winston-Salem, North Carolina, and his PhD in molecular medicine from the University of Maryland School of Medicine in College Park.

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AHA recognizes Penn Medicine cardiology researchers - Cardiovascular Business

July 27, 2017 This cross section image of the coronal brain region of a genetically mutant PlexA1 mouse shows evidence cortico motor-neuron cells eight days after researchers injected an altered rabies virus tracer into the animal's forelimbs. These connections are eliminated in normal wide mice as they mature. Scientists studying motor disabilities report in Science they blocked nerve pruning in the developing mutant mice, maintained these connections to adulthood. Credit: Cincinnati Children's

Researchers investigating why some people suffer from motor disabilities report they may have dialed back evolution's clock a few ticks by blocking molecular pruning of sophisticated brain-to-limb nerve connections in maturing mice. The result was mice with enhanced manual dexterity that grab and eat food much faster than regular wild-type mice, according to a study published July 28 in the journal Science.

Scientists at Cincinnati Children's Hospital Medical Center who led the study stress they aren't trying to create a genetically superior species of rodents. They are testing the formation of nervous system connections during early development in genetically bred mouse models. Their goal is to understand how sophisticated nerve connections start to form in wild baby mice, disappear as the animals mature, and whether this information might one day help patients.

Their study points to a class of proteins called semaphorins, which control the formation of long thread-like nerves called axons and motor neuron connections in the mammalian corticospinal (CS) system. In particular the scientists identify a protein called PlexA1, a major receptor molecule that attracts semaphorins. Semaphorins prevent axons from forming in inappropriate regions of the nervous system.

In the case of micewhich spend most of their time on four paws - signaling between a semaphore protein called Sema6 and PlexA1 activates in young mice. This eliminates critical synaptic links between nerve cells to stop the formation of sophisticated CS neural connections and fine motor skills.

"We may have found a pivotal point in the evolution of the mammalian corticospinal (CS) system that leads to greater fine motor control in higher primates and people," said Yutaka Yoshida, PhD, lead study investigator in the Division of Developmental Biology at Cincinnati Children's. "Although we still need to explore this, it's possible that some patients with motor disabilities have upregulated expression of PlexA1 or activated PlexA1 signaling that diminish cortico-motor-neuron connections and fine motor skills. Inhibition of PlexA1 signaling during childhood might be a way to restore these skills."

Key collaborators on the study includes John H. Martin, PhD, Department of Molecular, Cellular, and Biomedical Sciences, City University of New York School of Medicine, N.Y., and Nenad Sestan, MD, PhD, Kavli Institute for Neuroscience, Yale School of Medicine, New Haven, Conn., and first author Zirong Gu, a graduate student in the Yoshida laboratory.

Building a Better Mouse

After learning the PlexA1 protein eliminates sophisticated motor neuron connections in maturing mice, the researchers bred mice that don't express the gene regulating it (gene designation PlexA1). As PlexA1 mutant mice mature into adulthood, they lack the elimination of CS synaptic and motor neuron connections.

In feeding tests involving both short narrow strands of pasta and food pellets, mutant PlexA1 mice were significantly more skilled and faster than normal mice at grabbing and eating food.

When researchers tested mutant PlexA1 mice in skilled walking tests (conducted on balance grid), mutant mice did not perform significantly better than normal wild-type mice, according to the authors.

To understand differences in PlexA1 levels in mice and humans, study authors compared genetic and molecular regulation of CS neural connections in the mouse and human motor cortex of the brain. This region controls voluntary movements and other critical tasks. Human tests of the motor cortex were performed with donated human brain tissue.

The scientists determined differing PlexA1 expression is caused by what are called cis-regulatory elements. These are regions of non-coding DNA that help regulate nearby genes. A transcription factor (genes that tell other genes what do to) called FEZF2 interacts with cis-regulatory elements and directs formation of neural transmitter connections in CS neurons.

These FEZF2-controlled cis-regulatory elements are found in human brain tissues and in those of other higher primates, according to the authors. They are not found in mice. These regulatory elements are also responsible for suppressing PlexA1 in the developing human CS connections, which prevents sophisticated motor neuron connections from being disrupted as infants mature over the years into adults.

Moving Forward

Yoshida and his colleagues emphasize that extensive additional research is needed before knowing whether these findings might eventually apply to clinical practice. But they add that data from the study provides a number of clues the scientists want to explore in their future work. This includes trying to determine whether people with various types of motor disabilities have mutations in the Sema6 -PlexA1 molecular signaling pathway.

Explore further: Study suggests genetic reason for impaired skilled movements

More information: Z. Gu el al., "Control of species-dependent cortico-motoneuronal connections underlying manual dexterity," Science (2017). science.sciencemag.org/cgi/doi 1126/science.aan3721

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Scientists block evolution's molecular nerve pruning in rodents - Medical Xpress