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Archive - Jun 2017

June 3rd

Increasing Patient Access to Investigational Drugs Is Focus of Early Afternoon Session on Day 1 at Personalized Medicine World Conference (PMWC) 2017 at Duke

PMWC 2017 at Duke Co-Chair Ralph Snyderman, MD, introduced the two panelists for a discussion of “Increasing Patient Access to Investigational Drugs,” as the two leading people in the country working to make such drugs available to patients, and noting that for patients this is perhaps the best of times and the worst of times—best in that we are learning so much so rapidly and worst due to the difficulty of patients gaining access to potentially helpful drugs. One panelist was Ellen V. Sigal (photo), PhD, Founder of Friends of Cancer Research (https://www.focr.org/), an organization that has been instrumental in the creation and implementation of policies ensuring patients receive the best treatments in the fastest and safest way possible. Dr. Sigal introduced herself by noting that her PhD is actually in history and she had worked as a real-estate developer until her sister was diagnosed with breast cancer and died two days after receiving a bone marrow transplant. Then, Dr. Sigel began her unstinting work to help cancer patients. Dr. Sigal is Vice Chair, and currently Acting Chair, of the Inaugural Board of Directors of the Reagan-Udall Foundation (http://www.reaganudall.org/), which was designed to be a vehicle for bringing an array of resources and perspectives to bear on high-priority FDA regulatory science projects. The foundation fosters collaborations between patient groups, industry, academia, and FDA scientists to design and conduct regulatory science research. Dr. Sigal also serves on the Board of the Foundation for the National Institutes of Health, where she chairs its Public Private Partnerships Committee. In 2016, Dr.

Yale Chemists Forge New Path in Search for New Antibiotics; Achieve Elusive Full Synthesis of Pleuromutilin

Yale University scientists have developed a novel chemical process that may lead to the creation of a new class of antibiotics. The discovery comes at a time when more types of bacteria are becoming resistant to existing antibiotics, increasing the occurrence of lethal infections. The ability to create new antibiotics would have significant ramifications for medical treatment and public health, said the researchers. "This is one way to focus our talents as synthetic chemists in a direction that can immediately help patients," said Seth Herzon, PhD, a Chemistry Professor at Yale and member of the Yale Cancer Center. Dr. Herzon is principal investigator of a new study published in the June 2, 2017 issue of Science. The article is titled “A Modular and Enantioselective Synthesis of the Pleuromutilin Antibiotics.” Yale postdoctoral fellow Stephen Murphy, PhD, and Yale graduate student Mingshuo Zeng are co-authors of the study. Both are members of the Herzon Lab. The new process makes it possible to create molecules related to the natural product pleuromutilin from simple commercial chemicals in the laboratory. Pleuromutilin is produced by a fungus and was found to have useful antibacterial properties in the early 1950s. Since then, scientists in academia and the pharmaceutical industry have created thousands of pleuromutilin derivatives by a process known as semisynthesis, which involves chemically modifying pleuromutilin itself. However, a large proportion of these derivatives only vary at a single position in the molecule. A practical full synthesis, which would make a wealth of additional antibiotics possible, has remained elusive. Dr. Herzon first attempted to find a solution in 2008.

Adding Abiraterone to Standard Treatment Improves Prostate Cancer Survival by 37 Percent

Adding abiraterone to hormone therapy at the start of treatment for prostate cancer improves survival by 37 per cent, according to the results of one of the largest-ever clinical trials for prostate cancer presented at the 2017 ASCO Annual Meeting in Chicago and published in the New England Journal of Medicine on Saturday, June 3. The NEJM article is titled “Abiraterone Acetate Plus Prednisolone for Hormone-Naïve Prostate Cancer.” The ASCO abstract (LBA5003)is titled “Adding Abiraterone for Men with High-Risk Prostate Cancer (PCa) Starting Long-Term Androgen Deprivation Therapy (ADT): Survival Results from STAMPEDE (NCT00268476). The results from the Cancer Research UK-funded STAMPEDE trial could change the standard of care for men with prostate cancer, making abiraterone a first-line treatment alongside hormone therapy. This part of the STAMPEDE trial recruited approximately 1,900 patients. Half the men were treated with hormone therapy, while the other half received hormone therapy and abiraterone. In men who were given abiraterone there was a 70 per cent reduction in disease progression. The drug is usually given to men with advanced prostate cancer that has spread and has stopped responding to standard to hormone therapy, but this study shows the added benefit to patients who are about to start long-term hormone therapy. Professor Nicholas James, Chief Investigator of the Cancer Research UK-funded STAMPEDE trial from the University of Birmingham, said: "These are the most powerful results I've seen from a prostate cancer trial -- it's a once-in-a-career feeling. This is one of the biggest reductions in death I've seen in any clinical trial for adult cancers. Abiraterone is already used to treat some men whose disease has spread, but our results show many more could benefit.

June 1st

Brown Fat Depot Similar to Human’s Identified in Mice; Study May Reveal Roles of Brown Fat in Humans

When it gets cold around you, your body turns up the heat to maintain its normal temperature. The heat is produced by brown adipose tissue, or brown fat, which also plays a role in how the body uses glucose and fat. However, scientists do not completely understand how brown fat carries out its functions both in health and disease, in part because of the lack of an appropriate animal model. In a paper published online on June 2, 2017 in the Journal of Clinical Investigation Insight, a team of researchers from several institutions, including Baylor College of Medicine, has filled this gap with the discovery that mice also have brown fat deposits similar to the largest depot found in people. The discovery opens the door to research that might lead to new ways of using brown fat to treat metabolic conditions such as obesity and type 2 diabetes in the future. The open-access JCI Insight article is titled Identification and Characterization of a Supraclavicular Brown Adipose Tissue in Mice.” "In addition to white adipose tissue, or white fat, people have brown fat, an important contributor to the body's energy balance via the generation of body heat and the participation in metabolic processes," said senior author Dr. Miao-Hsueh Chen, Assistant Professor of Pediatrics and Nutrition at Baylor College of Medicine and the USDA/ARS Children's Nutrition Research Center at Baylor and Texas Children's Hospital. Brown fat contains adipocytes, cells that are rich in small fat-filled droplets and in energy-producing structures called mitochondria. Brown fat adipocytes use fat and glucose as sources of energy. In mice, brown fat activated to produce heat markedly affects the energy balance.

Exosome Diagnostics Will Demonstrate the “Shahky” Point-of-Care Protein Detection Instrument at ASCO

In a May 31, 2017 announcement, Exosome Diagnostics, Inc. said it will have a demonstration unit of ShahkyTM, the world’s first point-of-care protein capture and analysis instrument, for ASCO attendees to view and learn about capabilities at the upcoming world meeting in Chicago, June 2-5. Shahky will be on display in Exosome Dx’s booth, # 25091 (nearby the posters section of the exhibit hall.) Earlier this year, Exosome Dx announced the first partnership that grants access to the Shahky instrument with Merck KGaA, Darmstadt, Germany. Exosome Dx will use the ASCO meeting as a forum to engage potential new partners for both the Shahky instrument and other proprietary platforms including exosomal long RNASeq, enabling actionable biomarkers for clinical use. The Shahky instrument achieves high sensitivity by selectively targeting disease-specific exosomes. The instrument was validated at a leading Boston Hospital in early January of 2017. The instrument’s capabilities make it a powerful technology for discovering, assessing, and validating clinical biomarkers. The system has been developed and overseen by Exosome Dx’s Regulatory Department, with design control and engineering practices that are in accordance with FDA and other applicable regulations. Exosome Dx recently licensed a multiplexed nanoplasmonic assay designed specifically to fit into clinical workflows from a research effort at Harvard-MGH (published in Science Translational Medicine, May 2017) that developed a prototype assay for early detection of pancreatic ductal adenocarcinoma (PDAC) cancer. The assay employs a liquid biopsy approach, leveraging low blood volumes and the clinical potential of exosome-based proteins and mRNA.

Exosome Diagnostics Introduces Exosome Selection Platform at ASCO

In a June 1, 2017 press release, Exosome Diagnostics, Inc. announced that it will introduce its Exosome Dx Depletion (and/or) Enrichment (EDDE) platform to members of the American Society of Clinical Oncology (ASCO) at the ASCO annual meeting in Chicago (June 2-6). The patented EDDE platform can select exosomes derived from a specific tissue type, and, at the same time, significantly increase the signal to noise for any target. Exosome Diagnostics encourages attendees to stop by its booth to learn more about how the EDDE platform can improve biomarker discovery and patient stratification (booth #25091, nearby the posters section in the ASCO exhibit hall). “The EDDE platform is an important technological achievement to enrich for disease-specific vesicles. We have shown that this enables detection of targets not otherwise measurable on proteins and RNA. We are particularly excited to now combine the EDDE platform with our long RNAseq platform that efficiently enables sequencing of the transcriptome with a focus on mRNA, long non-coding RNA, and other clinically actionable targets from biofluids such as plasma and urine,” says Dr. Johan Skog, Chief Scientific Officer and Founding Scientist of Exosome Diagnostics. “We have already used the EDDE platform for immunotherapy targets like PD-1, as well as neuronal exosomes containing Tau,” continued Dr. Skog.

June 1st

North Carolina Governor & NIH Director Kick Off Precision Medicine World Conference (PMWC) 2017 at Duke

The 12th Personalized Medicine World Conference (PMWC) and the first on the East Coast of the United States was held May 24-25 at Duke University in North Carolina, and featured major biotech and medical figures from around the world. The meeting was opened by Program Co-Chair Ralph Snyderman, MD, Chancellor Emeritus, Duke University, James B. Duke Professor of Medicine, Duke University Medical School, and Director of the Duke Center for Research on Personalized Health Care. Dr. Snyderman said that with the completed sequencing of the human genome in 2003, there was a vision that health care would be transformed by new technologies and genomic advances, and that already remarkable progress has been made toward extending the healthy living and longevity of every person. He highlighted the strengths of Research Triangle Park and compared it favorably with Silicon Valley, particularly with respect to innovation. He paid tribute to the meeting co-hosts: Duke, Duke Health, Johns Hopkins University, Intermountain Healthcare, and UCSF, and then called upon Tal Behar, Co-Founder and President of PMWC International to say a few welcoming words to the over 400 meeting registrants. Tal said the PMWC was thrilled and honored to be at Duke for the first East Coast PMWC meeting and she thanked the meeting co-chairs, Dr. Snyderman and Geoff Ginsburg. MD, PhD, Professor of Medicine and Director of the Duke Center for Applied Genomic and Precision Medicine, for their efforts in organizing this truly spectacular meeting. She wished everyone two very productive days and then called on Dr. Ginsburg to make a few opening remarks. Dr. Ginsburg emphasized that precision medicine involves the convergence of many fields and focuses on the centrality of the patient.

Common Acne Medication Offers New Treatment for Recent-Onset Multiple Sclerosis; “Spectacular Outcome Will Positively Impact People's Lives Worldwide”

A Canadian clinical trial led by researchers at the University of Calgary's Hotchkiss Brain Institute (HBI), at the Cumming School of Medicine (CSM), shows that minocycline, a common acne medication, can slow the progress of relapsing-remitting multiple sclerosis (MS) in people who have recently experienced their first symptoms. In addition to being an unexpected discovery - an acne drug benefitting a neurological disorder - the discovery is significant as it offers a safe and affordable treatment option for those with early onset MS. This discovery could impact thousands of newly diagnosed MS patients around the world. The results of the Phase 3 clinical trial were published in the June 1, 2017 issue of the New England Journal of Medicine. The trial included 142 participants between the ages of 18 and 60 across 12 Canadian sites including: Vancouver, Burnaby, Calgary, Edmonton, Winnipeg, Ottawa, Toronto, London, Montreal, Quebec City, and Halifax. MS is thought to be an autoimmune disease of the central nervous system (brain, spinal cord). The disease attacks myelin, the protective covering of the nerves, causing inflammation and often damaging the myelin. The drug works by reducing the inflammation. The NEJM article is titled “Trial of Minocycline in a Clinically Isolated Syndrome of Multiple Sclerosis.” In Canada, the cost of current therapies for relapsing-remitting MS typically falls in the range of $20,000 to $40,000 per year; the minocycline treatment would cost a fraction of that at about $600 per year. In the United States, MS treatment often costs about three times as much as in Canada. Researchers say the potential cost saving will be tremendous and will improve access to treatment for people with MS.

Gene Finding Will Allow Eradication of Severe Blistering Disorder of the Skin Found in Dogs

Researchers at the University of Helsinki in Finland have identified a novel gene defect that causes a hereditary blistering disorder of the skin, epidermolysis bullosa, in dogs. Epidermolysis bullosa, found in the Central Asian Shepherd dog breed, occurs also in humans due to an identical gene found in both canines and humans. Hereditary blistering disorders of the skin are found with varying severity in both humans and animals. A symptom typical of all disease types is fragile skin that is easily damaged, followed by blistering and abrasion of the skin. This is caused by a defect in or deficiency of the structural proteins of the skin. "The project got its start when blistering in the skin and mucosal membranes was detected in different parts of the body in newborn littermates of Central Asian Shepherd dogs. This finding aroused suspicions of a hereditary blistering disorder of the skin," says researcher Marjo Hytönen, a member of the research group led by Professor Hannes Lohi. Pathological tests conducted by the Finnish Food Safety Authority Evira confirmed the diagnosis as epidermolysis bullosa. In genetic studies conducted by gene researchers at the University of Helsinki, the gene defect causing the disease was found in the COL7A1 gene. The gene is responsible for producing collagen, an important component in preserving the elasticity of the skin. This finding helped specify the diagnosis as the dystrophic form of the recessive blistering disorder of the skin known as epidermolysis bullosa. Four different structural types of the disease are known: simplex, junctional, dystrophic, and Kindler syndrome.

Raising Age Limit for Genetic Testing for Lynch Syndrome (Hereditary Nonpolyposis Colon Cancer) Could Decrease Mortality from This Cancer

Raising the age limit for routine genetic testing in colorectal cancer could identify more cases of families affected by Lynch syndrome, a condition that accounts for approximately 5% of all colon cancers, according to new research presented at the annual conference of the European Society of Human Genetics (ESHG) in Copenhagen, Denmark, on May 29, 2017. Professor Nicoline Hoogerbrugge, Head of the Radboud University Medical Centre Expert Centre on Hereditary Cancers, Nijmegen, The Netherlands, will tell the conference that there is an urgent need to find families carrying a mutation for Lynch syndrome in order to decrease mortality from the disease. "We know that, at present, only between 20% and 30% of people with Lynch syndrome have been identified. Most countries rely on detection through family history and early age at diagnosis, and this leads to significant under-diagnosis. We have shown that, by raising the age limit for testing we are able to detect new affected families who would not have been identified previously," she says. The researchers studied results of mismatch repair (MMR) testing in patients up to 70 years of age with colorectal cancer from 14 pathology laboratories. Previously, in The Netherlands, such testing was carried routinely in patients who were aged up to 50. Of 87 patients whose results suggested that they are at high risk of Lynch syndrome, 35 were referred for genetic counseling. After further testing, Lynch syndrome mutations were definitely identified in 13 of 32 patients with complete genetic testing, and 11 of these patients came from families in which the disease had previously not been detected. Eight of them were aged between 50 and 70 and did not comply with previous referral criteria for genetic testing based on age and family history.