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Archive - Jun 2017

June 8th

Day 2 of Personalized Medicine World Conference (PMWC) 2017 at Duke Begins with Panel Discussion on Value of Data Sharing

Day 2 (May 25) of the Personalized Medicine World Conference (PMWC) 2017 at Duke began with a panel discussion of “The Value of Data Sharing,” moderated by conference co-chair Geoff Ginsburg, MD, PhD, Professor of Medicine and Director of the Duke Center for Applied Genomic and Precision Medicine. The distinguished panelists were Kathy Giusti, MBA, a multiple myeloma patient and founder of the Multiple Myeloma Research Foundation (MMRF) in 1998, and Faculty Co-Chair of the Harvard Business School (HBS) Kraft Precision Medicine Accelerator--this $20M program is endowed by Robert Kraft and the Kraft Family Foundation and its mission is to speed precision medicine across all cancers; Bill Dalton (photo), MD, PhD, CEO of M2Gen, a health informatics solutions company that seeks to accelerate the discovery, development, and delivery of personalized medicine—the company was founded was founded in 2006 at the Moffitt Cancer Center in Tampa, Florida to operationalize the Total Cancer Care® Protocol, a unique approach to studying patients throughout their lifetime--in this effort, the company partners with the nation’s leading cancer centers via the Oncology Research Information Exchange Network (ORIEN)--Dr. Dalton was previously President, CEO, & Center Director of Moffitt Cancer Center--Moffitt and M2Gen have developed one of the largest cancer tumor bio-repositories and data warehouses in the U.S.

June 7th

Potential Therapeutic Use of Exosomes in Pancreatic Cancer Reported in Nature; Innovative Strategy Uses Engineered Exosomes Targeting Mutated KRAS Gene; MD Anderson Study Demonstrates That 'iExosomes' Shut Down Growth of Pancreatic Tumors in Mice

Genetic manipulation of exosomes, virus-sized particles released by all cells, may offer a new therapeutic approach to treating pancreatic cancer, according to a study at The University of Texas MD Anderson Cancer Center. Findings from the study, led by Valerie LeBleu, PhD, Assistant Professor of Cancer Biology, and Sushrut Kamerkar, PhD, Assistant Student in the MD Anderson UT Health Graduate School of Biomedical Sciences and the Cancer Biology Program, were published online on June 7, 2017 in Nature. The article is titled Exosomes Facilitate Therapeutic Targeting of Oncogenic KRAS in Pancreatic Cancer.” Earlier MD Anderson investigations demonstrated exosomes as a factor in detecting pancreatic cancer, but these latest findings reveal genetically altered exosomes as a potentially novel approach for direct and specific targeting of mutated KRAS, the cancer gene commonly linked to pancreatic cancer. In the study, exosomes, which are generated by all cells and are naturally present in blood, were modified as "iExosomes," capable of delivering small RNA to specifically target mutant KRAS, resulting in disease suppression and increased overall survival in mouse models. The investigators utilized a targeting method called RNA interference (RNAi) which, when delivered via these natural nanoparticles or exosomes, zero in on mutant KRAS in pancreas cancer cells, impacting tumor burden and survival in multiple pancreas cancer models. The team showed that exosomes could serve as an efficient carrier of RNAi, given that these nano-sized vesicles easily travel through the body and enter cells, including cancer cells.

June 6th

Newly Discovered Possible Mechanism for Type 2 Diabetes Involves Prominent Role for SOX5 Gene

A newly discovered mechanism behind reduced insulin production in type 2 diabetes is now being presented. In an article published online on June 6, 2017 in Nature Communications, researchers at Sahlgrenska Academy at the University of Gothenburg in Sweden describe how insulin-producing cells regress in their development, become immature, and do not work properly—a finding that opens the doors to new clinical treatments. “If you can affect things at the cellular level and restore the body’s own rapid regulation, you can more accurately adjust blood sugar compared to what is possible with insulin injections,” says Anders Rosengren, Associate Professor who is active at the Department of Neuroscience and Physiology as well as the Wallenberg Centre for Molecular and Translational Medicine at the University of Gothenburg. It has long been known that the insulin-producing cells fail in type 2 diabetes. The body does not get enough insulin and blood sugar rises. One theory argues that the insulin-producing cells become fewer in number, while another argues that their function is impaired. The new explanation, which combines the debated theories, states that the insulin-producing cells regress in their development and become immature. This reduces the number of functional cells. The open-access Nature Communications article is titled “Sox5 Regulates Beta-Cell Phenotype and Is Reduced in Type 2 Diabetes.” With the help of 124 tissue samples, of which 41 were from people with type 2 diabetes, the researchers were able to determine which genetic changes in the cells affected the course of the disease the most. Dr. Rosengren describes the analysis by comparing it to the world of air travel.

Pioneer & Luminary Awards Presented at Personalized Medicine World Conference (PMWC) 2017 at Duke

Day 1 of the two-day Personalized Medicine World Conference (PMWC) 2017 at Duke (May 24-25) ended with the presentation of Pioneer and Luminary awards to major contributors to the advance of precision medicine. The PMWC Luminary Award recognizes recent contributions of preeminent figures who have accelerated personalized medicine into the clinical marketplace. The PMWC Pioneer Award is given to rare individuals who presaged the advent of personalized medicine when less evolved technology and encouragement from peers existed, but still made major advances in the field. This year, the PMWC honored Francis Collins (Director, NIH), Elaine Mardis (Co-Director, Genomics Institute at Nationwide Children’s Hospital), and Keith Yamamoto (Vice Chancellor Research, UCSF) with the Luminary Award. For the Pioneer Award, Kathy Giusti (photo) (Founder of the Multiple Myeloma Research Foundation and Co-Chair, HBS Kraft Precision Medicine Accelerator), and Mark Levin (Co-Founder & Partner, Third Rock Ventures) were recognized. Francis S. Collins, MD, PhD, is a highly revered physician-geneticist who, as Director of the National Human Genome Research Institute, oversaw the successful 15-year multibillion-dollar effort to sequence the human genome. Often considered the most significant scientific undertaking of our time, researchers around the globe are now able to use genomic tools to expand understanding of human biology, combat disease and improve health through precision medicine. In 2009, Dr.

ME/Chronic Fatigue Syndrome Takes Center Stage on First Afternoon of Personalized Medicine World Conference (PMWC) 2017 at Duke

A very moving and informative session on myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS), was held in the mid-afternoon of Day 1 (May 24) at the Personalized Medicine World Conference (PMWC) 2017 at Duke. ME/CFS has been described by NIH Director Francis Collins as “the mysterious illness science has yet to unravel,” and, in this session, three compelling speakers sought to describe the disease, indicate where we are today in terms of research and knowledge of the disease, and suggest where we need to go in the future. The three speakers were session chair Zaher Nahle, PhD, MPA, Chief Scientific Officer and Vice President for Research at the Solve ME/CFS Initiative (SMCI) ( research & advocacy organization; Anthony L. Komaroff (photo), MD, Professor of Medicine, Harvard Medical School, and Senior Physician, Brigham and Women’s Hospital; and John Nicols, MBA, CEO of Codexis (, a protein-engineering company. Mr. Nicols’ wife Marcy, who was in the audience, has had ME/CFS for 25 years. Dr. Komaroff has cared for and studied patients with ME/CFS for 35 years, and is a member of the SMCI Research Advisory Council. Dr. Nahle oversees the research and scientific portfolio at the SMCI that includes the peer-review grant program for external investigators, specialized biobanking, and patient registry platforms, as well as numerous medical education initiatives for patients and healthcare professionals. He also directs a specialized investment program with medical centers, government agencies, and industry partners to spark innovation and accelerate the discovery process in the ME/CFS disease space. Dr. Nahle began the session by expressing his heartfelt gratitude to the PMWC for hosting this session on a very neglected disease.

June 5th

Exosomes Are Focus of Lonza Investment for Next-Generation Life Science Applications; Global Biopharma Leader Acquires HansaBioMed & Makes Strategic Investment in Exosomics Siena

On May 16, 2017, Lonza, a global leader in the field of biopharmaceutical development and manufacturing, announced the acquisition of HansaBioMed Life Sciences OÜ (HBM-LS), based in Tallinn, Estonia, a start-up company dedicated to the research and development, manufacturing, and distribution of products for the exosomes research market. Lonza has also agreed to make a strategic investment in Exosomics Siena S.p.A. (Exosomics), an Italian start-up company developing exosome-based, early-stage cancer screening and molecular diagnostic tests. Exosomes are nano-sized vesicles released by all living cells in a finely regulated manner. They are found in biological fluids and contain genetic material, proteins, and other cellular molecules reflecting the characteristic of their cells of origin. Functioning as nano-bullets with multiple surface receptors for crossing biological barriers, tissue targeting, and delivery of bioactive molecules, they have the potential of being developed for various applications in life sciences. Exosomes’ acknowledged role as effectors in tissue regeneration, immune response, and inflammation shows their potential as the next generation of vaccine development, drug delivery, and off-the-shelf, cell-free regenerative therapies. “Exosomes represent a dynamically growing segment in life sciences with opportunities in research, diagnostics, and therapeutic applications,” said Dr. Uwe Gottschalk, Chief Technology Officer for Lonza Pharma & Biotech. “Exosomes may play an important role in the promising field of liquid biopsies and could become the next generation of cell-free therapies in regenerative medicine," he added. "With our financial support, technical and commercial expertise, Lonza is well suited to leverage these opportunities through HBM-LS and Exosomics.” Dr.

Time To Starting Cancer Therapy Is Increasing, Associated With Worsening Survival, Cleveland Clinic Researchers Report; Findings Based on Analysis of Common Solid Tumors in Study Population of Nearly 3.7 Million

After reviewing nearly 3.7 million patient records, Cleveland Clinic researchers have shown that newly diagnosed cancer patients are having to wait longer to begin treatment, a delay that is associated with a substantially increased risk of death. This research was presented today at the ASCO Annual meeting and is abstract 6557 ( The researchers used prospective data from the National Cancer Database and examined the number of days between diagnosis and the first treatment for those newly diagnosed with early-stage solid-tumor cancers from 2004 to 2013. The study population of 3,672,561 patients included breast, prostate, colorectal, non-small cell lung, renal, and pancreas cancers. The median time between diagnosis and treatment - referred to as "time to treatment initiation," or TTI - has increased significantly in recent years, from 21 days in 2004 to 29 days in 2013. Delays were more likely if patients changed treatment facilities or if they sought care at academic centers. Longer delays between diagnosis and initial treatment were associated with worsened overall survival for stages I and II breast, lung, renal, and pancreas cancers, and stage II colorectal cancers, with increased risk of mortality of 1.2 percent to 3.2 percent per week of delay, adjusting for comorbidities and other variables. Prolonged TTI of greater than six weeks was associated with substantially worsened survival. For example, five-year survival for stage I non-small cell lung cancer was 56 percent for TTI of less than or equal to six weeks, versus 43 percent for TTI greater than six weeks, and for stage I pancreas cancer was 38 percent versus 29 percent, respectively. "In addition to its impact on outcomes, delayed TTI can cause unnecessary stress and anxiety for patients," said Dr.

Early Research Suggests First Immunotherapy for Mesothelioma on Horizon

Malignant pleural mesothelioma or MPM is a rare cancer, but its incidence has been rising. This cancer is usually associated with asbestos exposure, and patients have a median life expectancy of only 13-15 months. All patients relapse despite initial chemotherapy, more than 50% of them within six months after stopping treatment. There are currently no effective therapeutic options for patients with MPM. Early findings from an ongoing phase II clinical trial in France, MAPS-2, show that immunotherapy may slow the growth of MPM after relapse. At 12 weeks, cancer had not worsened in 44% of patients who received nivolumab (Opdivo) and in 50% of those who received nivolumab with ipilimumab (Yervoy). The study will be featured in a press briefing on June 5, 2017 and presented at the 2017 American Society of Clinical Oncology (ASCO) Annual Meeting. "Our findings suggest that immunotherapy may provide new hope to patients with relapsed mesothelioma," said lead study author Arnaud Scherpereel, MD, PhD, Head of the Pulmonary and Thoracic Oncology Department at the University Hospital (CHU) of Lille in Lille, France. "This randomized phase II trial may be enough to support the use of immune checkpoint inhibitors in this setting, but it is too early to conclude whether nivolumab alone or the combination of nivolumab and ipilimumab is better." This multi-center clinical trial enrolled 125 patients with advanced MPM who had received up to two prior treatments, including standard platinum-based chemotherapy. The majority of patients (80%) were male, and the median age was 72 years. The patients were randomly assigned to treatment with nivolumab alone or nivolumab with ipilimumab until the cancer worsened; 70% of patients received at least 3 cycles of either treatment.

GRAIL Announces Data from Early Research Using Its High-Intensity Sequencing Approach on Circulating Cell-Free DNA (cfDNA); Data Will Inform Future Development of Blood Tests for Early Detection of Cancer

On June 3, 2017, GRAIL, Inc., a life sciences company focused on the early detection of cancer, announced results from a study evaluating a novel high-intensity sequencing approach for detecting tumor signals in the bloodstream. Historically, research has focused on detecting a small number of clinically-actionable mutations in circulating cell-free DNA (cfDNA). This study evaluated the ability of a high-intensity sequencing approach to detect a more diverse range of tumor signals, including those that are less common. This broader and deeper approach will be required to develop tests that can detect the tiny amounts of tumor DNA in the blood, and accurately capture the diversity in genomic alterations that characterize the tumors of every person with cancer. In the study, blood and tumor tissue samples from 124 patients with various advanced cancers were sequenced with a 508-gene panel, yielding approximately 100 times more sequencing data than previous approaches. In 89 percent of patients, at least one of the mutations detected in the tumor tissue was also detected in the blood. When evaluating all genetic variations, including those present at high levels in tumor tissue as well as those at low levels, 627 of 864 mutations (73 percent) detected in tumor tissue were also detected in patients’ blood. “These encouraging results showed our high-intensity sequencing approach is able to detect a broad range of tumor mutations in the bloodstream with high levels of concordance with all mutations detected in tumor tissue,” said Alex Aravanis, MD, PhD, Head of Research & Development at GRAIL. “These important foundational data support the feasibility of our approach and will inform further development of blood tests to detect early cancer.

June 4th

Phase III Trial of PARP Inhibitor (Olaparib) Shows First Evidence of Improved Outcomes in Breast Cancer

The first phase III trial of a PARP inhibitor used to treat breast cancer reported promising data at the annual meeting of the American Society of Clinical Oncology (ASCO) in Chicago. Mark Robson, MD, Clinic Director of the Clinical Genetics Service and medical oncologist at Memorial Sloan Kettering Cancer Center (MSK), led the multicenter, international trial. He presented the study results in the plenary session of the ASCO annual meeting on June 4. “This landmark study is the first to demonstrate that a PARP inhibitor improves outcomes in women with BRCA-mutation-associated breast cancer compared with standard treatment,” explained Dr. Robson. “At MSK, we like to say that breast cancer is often described as a book with many chapters. We see this treatment option as an early chapter in a woman’s journey. The study showed that olaparib is a well-tolerated treatment that can extend the time until chemotherapy is necessary. Olaparib helps patients preserve their quality of life in the crucial early stages of their treatment journey.” Called OlympiAD, the randomized, open-label phase III study assessed the efficacy and safety of olaparib (Lynparza™), an oral PARP inhibitor, versus standard single-agent chemotherapy treatment. All of the patients in the trial had metastatic breast cancer that was caused by an inherited mutation in the gene BRCA1 or BRCA2 — either hormone receptor–positive or triple negative. They all had previously received anthracycline and taxane treatments and, if their cancer was hormone receptor–positive, a hormonal treatment. A total of 205 women in the study received olaparib and 91 received chemotherapy. The median patient age was 44, as women with BRCA mutations are significantly younger than the average breast cancer patient.