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Archive - Jan 19, 2017

NX Prenatal Appoints Leading Experts to Medical/Clinical Advisory Board; Company’s First Product Will Be Exosome-Based Blood Test to Stratify Risk of Preterm Birth

In a January 19, 2017 press release, NX Prenatal, Inc. announced the formation of its Medical/Clinical Advisory Board. This new board brings together leading clinicians and industry veterans who enhance the range and depth of expertise of the company as it accelerates development of its novel prenatal diagnostic tests. These experts are uniquely positioned to provide medical, clinical, and strategic guidance for the company's product development and commercialization activities. The first product will be a novel exosome-based blood test designed to stratify the risk of preterm birth in women as early as 10-12 weeks of pregnancy to provide physicians with a new decision support tool. Gail Page, NX Prenatal's Executive Chairperson, added, "We are pleased to be surrounded by advisors who have deep knowledge in the fields of maternal fetal medicine, obstetrics, proteomics, and biomarker test commercialization. We recognize the importance of working closely with key opinion leaders to ensure that our diagnostic tests are relevant and address an unmet need in the clinical community that will use them." The advisory board members include the following: KEVIN ROSENBLATT, M.D., PH.D. (CHAIR) Dr. Rosenblatt is Chief Medical & Scientific Officer of NX Prenatal. He previously served as CMO/CSO of CompanionDx Labs, a precision medicine/diagnostics company. Dr. Rosenblatt is recognized internationally for his proteomics and genomics expertise and has published over 60 papers on biomarker development and clinical proteomics. He served as an Associate Professor in the Division of Oncology, Department of Internal Medicine, Associate Professor at the Brown Foundation Institute of Molecular Medicine and as Director of the Proteomics Core for the Center for Clinical and Translational Sciences at UT Health.

Largest Study to Date Shows That "Precision Medicine for Pediatric Brain Tumors Can Now Be a Reality"

Precision medicine - in which diagnosis and treatments are keyed to the genetic susceptibilities of individual cancers - has advanced to the point where it can now impact the care of a majority of children with brain tumors, a new study by investigators at Dana-Farber/Boston Children's Cancer and Blood Disorders Center suggests. In the largest clinical study to date of genetic abnormalities in pediatric brain tumors, researchers performed clinical testing on more than 200 tumor samples and found that a majority had genetic irregularities that could influence how the disease was diagnosed and/or treated with approved drugs or agents being evaluated in clinical trials. The findings, reported online on January 19, 2017 in Neuro-Oncology, demonstrate that testing pediatric brain tumor tissue for genetic abnormalities is clinically feasible and that in many cases the results can guide patients' treatment. The need for new approaches to treating brain cancer in children is urgent, the study authors say. "Although there has been a great deal of progress over the past 30 years in improving survival rates for children with cancer, advances in pediatric brain cancer haven't been as dramatic," says co-lead author Pratiti Bandopadhayay, M.B.B.S., Ph.D., of Dana-Farber/Boston Children's. "In a recent study, brain tumors accounted for 25 percent of all pediatric deaths attributed to cancer. In addition, many of the current therapies can result in long-term difficulties in cognitive or physical functioning." Since emerging from research labs more than a decade ago, targeted therapies for cancer have significantly improved the treatment of certain types of leukemia, digestive system tumors, and breast cancer, among other malignancies.

Acute Myeloid Leukemia Study Supports Knowledge Bank Approach to Personalized Therapy in Cancer

An international collaboration led by clinical researchers at the Wellcome Trust Sanger Institute has shown proof-of-concept that truly personalized therapy will be possible in the future for people with cancer. Details of how a knowledge bank could be used to find the best treatment option for people with acute myeloid leukaemia (AML) were published online on January 16, 2017 in Nature Genetics. The article is titled “Precision Oncology for Acute Myeloid Leukemia Using a Knowledge Bank Approach.” AML is an aggressive blood cancer that develops in bone marrow cells. Earlier this year, the team reported that there are eleven types of AML, each with distinct genetic features. Now they report how a patient's individual genetic details can be incorporated into predicting the outcome and treatment choice for that patient. The scientists built a knowledge bank using data from 1,540 patients with AML who participated in clinical trials in Germany and Austria, combining information on genetic features, treatment schedule, and outcome for each person. From this, the team developed a tool that shows how the experience captured in the knowledge bank could be used to provide personalized information about the best treatment options for a new patient. There are two major treatment options for young patients with AML - a stem cell transplant or chemotherapy. Stem cell transplants cure more patients overall, but up to one in four people die from complications of the transplant and a further one in four experience long-term side effects. Weighing up the benefits of better cure rates with transplant against the risks of worse early mortality is a harrowing decision for patients and their clinicians. The team showed that these benefits and risks could be accurately calculated for an individual patient, enabling therapeutic choices to become personalized.

Genetically Altered Rabies Viruses Reveal Wiring in Transparent Mouse Brains; Viral Tool for Assessing Connectivity of Cell Transplants

Scientists under the leadership of the University of Bonn have harnessed rabies viruses for assessing the connectivity of nerve cell transplants: coupled with a green fluorescent protein, the viruses show where replacement cells engrafted into mouse brains have connected to the host neural network. A clearing procedure which turns the brain into a “glass-like state” and light sheet fluorescence microscopy are used to visualize host-graft connections in a whole-brain preparation. The approach opens exciting prospects for predicting and optimizing the ability of neural transplants to functionally integrate into a host nervous system. The results were published online on January 19, 2017 in Nature Communications. The open-access article is titled “Whole-Brain 3D Mapping of Human Neural Transplant Innervation.” Many diseases and injuries result in a loss of nerve cells. Scientists are working on tackling this challenge by transplanting neurons. In Parkinson’s disease, for instance, this is attempted with implanted dopamine-producing nerve cells. The key question for such techniques is whether the implanted cells actually connect with the existing neural network of the host brain and thus compensate the functional loss. “Previous methods only provided an incomplete or very small-scale insight into the functional integration of implanted neurons in the brain,” says Professor Oliver Brüstle from the Institute of Reconstructive Neurobiology at the University of Bonn and LIFE & BRAIN GmbH.