Category Archives: Cell Medicine

Newswise Seventeen academics, researchers, and scientists are being recognized for their significant contributions to the anatomical sciences and the future of anatomy education and research by the U.S.-based international society representing 2,300 members in anatomy and anatomy-related disciplines. Through these awards, grants, and scholarships, the American Association for Anatomy (AAA) continues to elevate, celebrate, and advanceeven in a pandemicthe foundational science of anatomy and its application in healthcare, allied health, and beyond.

Although AAAs awards program culminates in celebrating these 17 honorees, awards, grants, and scholarships are available year-round. AAA is currentlythrough February 1accepting submissions for the Early-Career Anatomist Publication Awards, which recognize excellence in scientific research manuscripts by early-career researchers published in AAAs three peer-reviewed journals: The Anatomical Record, Anatomical Sciences Education, and Developmental Dynamics. Eligible applicants can find details and apply at anatomy.org/awards.

In the major categories already awarded, this years esteemed honorees are:

A.J. Ladman Exemplary Service Award

Lynne Opperman, PhD, FAAA, of Texas A&M University College of Dentistry in Dallas is Regents Professor, Head of the Department of Biomedical Sciences, and Director of Technology Development.

Dr. Opperman is Past President of AAA and current President of the Association of Anatomy, Cell and Biology and Neurobiology Chairpersons. An AAA member since 1998, she has served on numerous committees and task forces including, among others, 125th Anniversary, Program, and Advisory Committee for Young Anatomists (now CECA). An AAA Fellow since 2010, Dr. Opperman was pivotal in creating the Fellows Grant Award Program in 2016, which aims to advance federal funding of anatomical research.

With more than 5,700 research citations, Dr. Opperman is an oft-invited speaker on topics related to mentorship and her research, which includes craniofacial suture development and bone development growth and repair. She was elected a Fellow of the American Association for the Advancement of Science in 2019 in recognition of her research accomplishments, mentorship, and service.

Having mentored more than 140 students, Dr. Opperman trains not just academics, but academics who understand industry and the patent process notable because her company holds five US patents for devices related to bone reconstruction.

Dr. Opperman was born and raised in South Africa, where she completed her PhD before coming to the United States as a Postdoctoral Fellow. Her success in the United States provides an example for trainees and young faculty throughout the world for what is possible with hard work and a mind open to opportunity.

Henry Gray Distinguished Educator Award

Robert M. Klein, PhD, FAAA, of the University of Kansas School of Medicine is Vice Chancellor for Academic and Student Affairs and Chancellors Club Professor of Anatomy and Cell Biology.

For 45 years, Dr. Kleins students have recognized his passionate, student-oriented approach to preclinical curriculum. Said a colleague, For me, his enthusiasm and skill in linking the science and art of medicine was instrumental in sparking my interest in academic medicine as a career.

Dr. Kleins leadership was essential in changing KUs curriculum and in successful accreditation visits. In addition to administration, he remains an active educator and facilitator for small-group, case-based, collaborative learning sessions. Remarked Klein, I work to guide students to become life-long learners a requirement of future physicians.

Dr. Klein is a leader in the use of virtual microscopy for medical education, as well as in organizing national and international events advancing the teaching of anatomy, histology. and embryology. He co-authored two books and published numerous papers on advances in medical education.

An AAA member since 1977, Dr. Klein served on the Educational Affairs Committee and, in 2015, was recognized as a Fellow. He has been named a Charter Member of the Academy of Medical Educators and an Honorary Alumnus of the KU School of Medicine, and he has been recognized with numerous teaching awards including the Alpha Omega Alpha Robert J. Glaser Distinguished Teacher Award and the International Association of Medical Science Educators Master Teacher Award.

Henry Gray Scientific Achievement Award

Wayne Vogl, PhD, FAAA, of The Life Sciences Institute at The University of British Columbia is Professor in the Department of Cellular & Physiological Sciences and recipient of AAAs highest scientific honor for his countless contributions to the anatomical sciences.

Dr. Vogl is internationally recognized for his work using fluorescence microscopy and electron microscopy to explore the relationship between the three major cytoskeletal elements (actin filaments, microtubules, and intermediate filaments) in Sertoli cells and intercellular junctions in the seminiferous epithelium of the mammalian testis, and how the two systems together function to generate morphogenic events (translocation of developing sperm cells, sperm release) in the testis. Dr. Vogls work has led to a critical understanding of the role of the cytoskeleton in Sertoli cells during spermatogenesis and in male fertility.

Dr. Vogl has been cited more than 10,000 times, invited to participate in 55 lectures, and received numerous teaching awards, as well as the Basmajian Award. Consistently funded for more than 40 years, Dr. Vogl has been awarded more than $6 million to advance his work, from which he has published more than 130 peer-reviewed articles, 13 invited reviews and book chapters, and 150 abstracts.

In 2005, Dr. Vogl co-authored Grays Anatomy for Students with Richard L. Drake and Adam W.M. Mitchell. Now in its fourth edition, the text won first prize in Basic and Clinical Sciences at the 2009 British Medical Associations Medial Book Competition and has been translated into 13 languages.

Dr. Vogl joined AAA in 1984 and was named a Fellow in 2009.

Fellows

The rank of Fellow of the American Association for Anatomy (FAAA) honors distinguished members who have demonstrated excellence in science and in their overall contributions to the anatomical sciences. Since 2008, 100 members have been recognized as Fellows. This year, we add five including three Canadians and the first Latina. Below, meet this years esteemed Class of Fellows.

A member since 1999, Rebecca Fisher, PhD, is Professor & Interim Co-Chair of Basic Medical Sciences and Director of the Gross Anatomy Laboratory at the University of Arizona College of Medicine-Phoenix. Dr. Fisher has served as a research mentor for undergraduate, graduate, medical, and postdoctoral students, encouraging diverse trainees to pursue careers in science. Dr. Fisher studies the functional anatomy of mammals and cephalopods. Her current work on octopus-inspired robotics is funded by a grant from the Office of Naval Research. A former Basmajian Award recipient and Board Member, Dr. Fisher is the first Latina to be named an AAA Fellow. She has served in a number of volunteer leadership roles, and currently serves on the Diversity, Equity, and Inclusion Committee. (photo by Chris Richards)

A member since 1999, Julian Guttman, PhD, is Professor of Cellular Microbiology at Simon Fraser University in British Columbia, Canada. He previously received the Young Investigator Award for Morphological Sciences and a Young Anatomist Publication Award, and served two terms on the Board of Directors. Dr. Guttman has been an Associate Editor of The Anatomical Record since 2015 and is on the Editorial Boards of Cytoskeleton and Tissue and Cell. Dr. Guttmans research examines the molecular and morphological alterations that occur during bacterial infections of epithelial cells. He has published more than 60 articles and has served as Principal Investigator (PI) or Co-PI on research projects awarded in excess of $3 million.

Claudia Krebs, MD, PhD, is Professor of Teaching in the Department of Cellular & Physiological Sciences at The University of British Columbia. An AAA member since 2006, Dr. Krebs was elected to the Board in 2020, having previously served on the Educational Affairs Committee. Dr. Krebs continuously advances instruction of neuroscience while combatting neurophobia and innovating teaching modalities. For The HIVE (Hackspace for Innovation and Visualization in Education), she assembled an interdisciplinary team to create digital media for anatomy education, partnering with the Universitys Emerging Media Lab and industry partners like Microsoft. The initiative has developed 18 projects, with more in the pipeline. Additionally, she is the first author of Lippincotts Illustrated Review of Neuroscience, now in its second edition.

At the University of Utah School of Medicine, David Morton, PhD, is Professor of Neurobiology and serves as Vice-Chair of Medical and Dental Education. He directs multiple courses and the gross anatomy lab, and teaches medical, dental, PA, PT, and OT students. He is an Academy of Health Science Educators Fellow and has received numerous teaching awards, including the UU Distinguished Teaching Award. Research interests include creation and incorporation of active learning activities and cadavers in medicine. Dr. Morton authored or co-authored multiple textbooks, including The Big Picture: Gross Anatomy. His video tutorials have received more than 8.5 million views on YouTube. Dr. Morton serves as visiting professor at three medical schools in Ghana. A 20-year member of AAA, he served on the Board of Directors 2014-2017.

A member since 2008, Bruce Wainman, PhD, fills many roles at McMaster University: Professor of Pathology and Molecular Medicine; Director of Anatomy, Education Program in Anatomy, and Surgical Skills Laboratory; and Adjunct Professor of Obstetrics and Gynecology and Surgery. Dr. Wainman has received numerous teaching awards and in 2016 was named a 3M National Teaching Fellow. He has mentored dozens of undergraduates, published 60 articles, and served as Principal Investigator (PI) or Co-PI on research projects awarded more than $5.2 million. He chaired the 2018 Regional Meeting (the most-attended to date), and is a member of the 2020 Nominating Committee. Research interests include interprofessional education, cognitive psychology, and use of augmented and virtual reality in education.

Basmajian Award

Haley OBrien, PhD, is Assistant Professor of Anatomy and Cell Biology at Oklahoma State University Center for Health Sciences, where she uses digital rendering techniques to study the function, physiology, development, and evolution of craniocervical systems. As course director of the Neurology block, she reformatted the nervous system course, resulting in average student improvement of one full letter grade. Since joining the Curriculum Oversight Committee, she developed the first Course Directors Handbook. Just five years post-doctorate, Dr. OBrien has already mentored nine PhD and MS students in research and teaching of human gross anatomy, along with 15 neuroanatomy research medical students. She recently won Oklahoma State University's App Competition for development of a nervous system educational app with a team of medical student researchers. Dr. OBrien has been recognized in the Federation of American Societies for Experimental Biology and AAA Annual BioArt Scientific Image Competition. She previously received two Education Outreach Grants and an Early-Career Publication Award for The Anatomical Record. The Basmajian Award acknowledges her many outstanding accomplishments early in her teaching career.

Early-Career Investigators

These awards recognize investigators in the early stages of their careers who have made important contributions to biomedical science through their research.

C.J. Herrick Award in Neuroanatomy

Andrea M. Gomez, PhD, of the University of California, Berkeley, is Assistant Professor of Neurobiology in the Department of Molecular & Cell Biology. Dr. Gomezs lab opened in 2020, studying how genetic programs balance order and variability in the brain, using electrophysiology, functional imaging, and molecular biology to decode the instructive cues that organize neural networks to discover how synaptic dysfunction manifests in conditions like autism, intellectual disability, and neurodegenerative disorders. In support of this groundbreaking neurological research, she was awarded a 2020 Young Investigator Grant from The Brain & Behavior Research Foundation. An advocate for diversity and inclusion in STEM, Dr. Gomez is Laguna Pueblo and Chicana.

H.W. Mossman Award in Developmental Biology

Eric Van Otterloo, PhD, of The University of Iowa is Assistant Professor in the Iowa Institute of Oral Health Research in the College of Dentistry. He researches craniofacial and neural crest cell development, and related pathologies, using both animal genetics and modern molecular biology approaches. Dr. Van Otterloo is the first author on seven publications and collaborated on nine others. He was among the first to receive an AAA Postdoctoral Fellowship, exploring the critical role of MEMO1 in the development of the facial skeleton. A member since 2010, he is also a member of the Society for Craniofacial Genetics and Developmental Biology, an AAA affiliate. Dr. Van Otterloos research is currently supported by the National Institute of Dental and Craniofacial Research.

R.R. Bensley Award in Cell Biology

Pulin Li, PhD, is a Member of the Whitehead Institute and, in 2020, was named the Eugene Bell Career Development Professor of Tissue Engineering at the Massachusetts Institute of Technology. Dr. Li uses quantitative approaches to gain new insights into tissue biology. Demonstrating abilities across the disciplines of developmental biology, chemical biology, synthetic biology, and imaging technology, Dr. Li studies cell communication circuits that enable multicellular functions. Her lab examines how communication provides positional information to cells within a tissue, how feedback circuits coordinate cell communication in space and time, and how communication systems adapt during evolution. Her contributions include developing tools for discovering novel signaling pathways in tissue development and repair from the top down, and reconstituting and rewiring communication circuits from the bottom up to decipher their design principles. (photo: Whitehead Institute)

Fellows Grant Award Program (FGAP)

This Program supports research proposals submitted to major funding agencies that, although well reviewed, did not receive funding. FGAP aims to help researchers revise grant applications in anticipation of resubmission for approval. This year, FGAP will fund two projects:

Martine Dunnwald, PharmD, PhD, of The University of Iowa is Research Associate Professor of Anatomy and Cell Biology. Her project, Arhgap29 in orofacial development, examines the role of Rho GTPase activating protein 29 (Arhgap29) in orofacial clefts (OFCs). NIH R01 reviewers noted, successful completion of the proposed studies would fill knowledge gaps in understanding the role of periderm [lining of the oral cavity] in pathogenesis of OFC, having significant impact in the field. Despite scientific rigor with inclusion of power analysis and sample size estimates, reviewers requested more data to differentiate between wild type and mutant cells. FGAP funding will support hiring a trainee from an underrepresented minority to generate Arhgap29 cell lines with patient-derived mutations and preliminary mechanistic data using those cell lines.

Heather Szabo-Rogers, PhD, of the University of Pittsburgh School of Dental Medicine is Assistant Professor in Oral and Craniofacial Sciences. Dr. Szabo-Rogers project is Prickle1 protein-protein interactions are required for craniofacial chondrocyte signaling and polarity. The FGAP award will expand an NIH R03 application to a competitive R01. The new application will focus on the development of the nasal capsule cartilages, in addition to determining the role of Prickle1 in primary ciliopathies and Robinow Syndrome (RS). FGAP funding will support hiring a trainee to analyze existing cutting-edge images and generate data from RS-patient cells and a mouse model. Once collected, the new data generated with FGAP funding will strengthen Dr. Szabo-Rogers strong preliminary data.

Postdoctoral Fellowships

This award provides salary support to postdoctoral trainees working in any aspect of biology relevant to the anatomical sciences, including both basic science research and education research.

Gary J. Farkas, PhD, is a Postdoctoral Fellow in the Department of Physical Medicine and Rehabilitation at the University of Miami Miller School of Medicine. His research, Exercise and Nutrition to Reduce Obesity-Induced Inflammation and Improve Cardiometabolic Health in Spinal Cord Injury (ENRIICH-SCI), adds to more than two dozen publications on spinal cord injury and rehabilitation. This research aims to define the impact of functional electrical stimulation leg cycle exercise and diet, versus diet alone, on epicardial and abdominal visceral adipose tissue and proinflammatory adipokines in persons with chronic motor complete C4-T4 spinal cord injury. A member since 2010, Dr. Farkas serves on the Educational Affairs Committee, co-created the educational abstract scoring rubric, and initiated the LGBTQ+ in Anatomy community.

Soma Dash, PhD, is a Postdoctoral Research Associate in the Trainor Lab at the Stowers Institute for Medical Research. Dr. Dash joined Stowers and AAA in 2018. The focus of her research is to determine a role for ribosome biogenesis, via RNA polymerase I subunits, Polr1a and Polr1c, and associated factor, Tcof1, in neural crest cells in enteric nervous system formation and gastrointestinal birth defects. Building upon previous studies published by the Trainor Lab related to cranial neural crest cells function and Treacher Collins syndrome, this new research has a high potential impact for therapeutic studies of Hirschsprung disease. I am driven to identify the nature and molecular mechanism of regulatory factors like ribosome biogenesis and their relation to developmental defects, said Dr. Dash.

Education Research Scholarship

Hei Ching Kristy Cheung is a graduate student at Western University in Ontario, Canada, where she is pursuing a Master of Science degree in Clinical Anatomy, as well as a Learning Design Certificate in Educational/Instructional Media Design. She has been a teaching assistant for half a dozen courses in gross and clinical anatomy, histology, and dentistry, and assisted with the development of e-learning modules for the medical program using articulate storyline software to revise and improve e-modules in gross anatomy and histology. Cheungs education research, Meaningful Motion: Development of a functional anatomy online resource for allied health professionals, will evaluate such modules in the wake of the dramatic shift to virtual learning. Cheung joined AAA in 2020.

See original here:
American Association for Anatomy Honors 17 Scientists Advancing Education and Discovery in Anatomical Sciences, Healthcare, and Related Fields -...

MEMPHIS, Tenn., Jan. 27, 2021 /PRNewswire/ -- Cognate BioServices, Inc., the premier commercial-ready global CDMO in the Cell and Gene Therapy industry, and Nucleus Biologics, LLC, The Cell Performance Company, announced today a partnership for custom and commercial medias and delivery systems for Cell and Gene Therapy clients.

"We're quite excited to be moving forward with Nucleus," said J. Kelly Ganjei, Cognate's Chief Executive Officer. "I am particularly pleased with this collaboration that has come to us from the front lines of product development. Seeing an opportunity to simplify the supply chain, our sales and business development team seized this opportunity. I applaud my team and Nucleus for their creativity and vision in getting this partnership off the ground."

Nucleus' AI formulation platform creates an optimized media offering that will allow Cognate's clients to reduce time in development and towards large scale production. The partnership between Cognate and Nucleus will improve stability, reproducibility and supply-chain integrity for clients by enabling an on-demand supply of proprietary media from clinical to commercial scale. "Nucleus' approach is a game changer in Cell and Gene Therapy manufacturing," added Mr. Ganjei.

This partnership will focus on both clinical and commercial clients, especially as Cognate expands its business with both allogeneic and autologous on-demand cell and gene therapies. The Nucleus platform is unique within the cell and gene therapy supply chain and provides a scalable solution to rapidly formulate optimized media and receive custom media lots. This remarkably robust platform will enable Cognate to rapidly implement this technology across its global capacity network.

"We're eager to get our partnership with Nucleus out to the market," said Mike Stella, Cognate's Chief Business Officer. "Nucleus' product pipeline will improve stability, reproducibility, and reduce time to large-scale production. Critical components like media have been front and center for a while in our industry, but the constraints have been further amplified with COVID. We believe this will become another key differentiator for our business."

Story continues

"We are grateful for the opportunity to work with Kelly and his team at Cognate," said David Sheehan, Founder, President and CEO of Nucleus Biologics. "Cognate understands the industry, the clients' needs, as well as the urgency with which the industry must create a new paradigm for media. Our approach promotes therapies getting into the clinic quicker, safer and more reliably, with less supply chain risk, and ultimately results in patients getting product more timely. We are laser focused on building disruptive products, especially as it pertains to the supply chain of advanced therapy developers, and we are excited to have Cognate as a partner."

About Cognate BioServices, Inc.

Cognate BioServices is the leading CDMO for the development and manufacturing of autologous and allogeneic cell and gene therapy products. We are a dynamic, results-driven, organization focused on providing the broadest range of commercialization services to regenerative medicine, cellular immunotherapy and advance cell therapy companies. Cognate provides a unique combination of custom services to companies across all points of clinical and commercial development specializing in mid to late stage clinical trials and supporting our clients through product scale-up into commercial manufacturing. Cognate applies the knowledge and expertise of its business, scientific and technical teams to successfully develop autologous and allogeneic products across multiple cell-based technology platforms from start to finish.

Cognate's business and expansion activities are supported by leading shareholder EW Healthcare Partners, as well as Medivate Partners, Blackrock, and a Middle East Sovereign Wealth Fund.

http://www.cognatebioservices.com

About Nucleus Biologics, LLC. Nucleus Biologics, The Cell Performance Company, is the leading provider of custom cell-growth media, tools and technologies for cell and gene therapy. Their mission is to speed the time from scientific discovery to cure by delivering innovative, transparent and cGMP products and services with the goal of disrupting the market and eliminating antiquated practices and products. Ultimately, Nucleus Biologics strives to create a new paradigm that serves both scientists and clinicians, while reducing the environmental footprint of cell culture. http://www.nucleusbiologics.com

For more information, please contact James Wilkerson at jwilkerson@cognatebioservices.com

View original content to download multimedia:http://www.prnewswire.com/news-releases/cognate-bioservices-and-nucleus-biologics-announce-partnership-in-cell-and-gene-therapies-301216405.html

SOURCE Cognate BioServices

Go here to see the original:
Cognate BioServices and Nucleus Biologics announce partnership in Cell and Gene Therapies - Yahoo Finance

CARLSBAD, Calif.--(BUSINESS WIRE)--Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies to address unmet medical needs, recently highlighted to members of the investment community its clinical and operational progress through a series of webinars hosted by FORCE Wealth with presentations by Brian M. Culley, Chief Executive Officer. The seminars covered all three of the companys clinical-stage product development programs: OpRegen, a retinal pigment epithelium (RPE) cell transplant for the treatment of dry age-related macular degeneration (AMD), OPC1, an oligodendrocyte progenitor cell transplant for the treatment of acute spinal cord injuries, and VAC2, a dendritic cell transplant for the treatment of cancer. Interested investors can now access all three presentations on the Media section of Lineages website.

Lineage Cell Therapeutics: The Eyes of the World A fireside chat hosted by Robert Rothman, M.D., Clinical Assistant Professor, Ophthalmology at the Zucker School of Medicine at Hofstra/Norwell and Principal, InFocus Capital Partners, an ophthalmology-focused venture capital firm, on January 14, 2021, discussing Lineages OpRegen program and the dry AMD therapeutic landscape and commercial opportunity.

Lineage Cell Therapeutics: A Fireside Chat on Regenerative Medicine A fireside chat hosted by Joseph Pantginis, Ph.D., Director of Research and Managing Director, Equity Research at H.C. Wainwright & Co. Inc., on December 8, 2020, discussing Lineages pipeline and the regenerative medicine landscape.

Lineage Cell Therapeutics: From Paralysis to Pitching Improving Mobility After a Severe Spinal Cord Injury A presentation and Q&A session on October 22, 2020, discussing Lineages OPC1 program and the spinal cord injury disease landscape, treatment approaches, and the commercial opportunity of restoring mobility to individuals who have been paralyzed by a cervical spinal cord injury.

Lineage and some more recently established competitors in the field of allogeneic cell therapy are helping to demonstrate the viability and commercial potential of using allogeneic cell transplants to treat or cure serious diseases or conditions that represent major unmet medical needs and large market opportunities, stated Brian M. Culley, Lineage CEO. As the tools and methods used to manufacture and test these therapies in patients are reaching maturity, public policy and investor support for cell therapy has moved in a positive direction. As we prepare to advance our product candidates into later-stage clinical trials, we are working to position Lineage to benefit from this convergence of positive factors and help to accelerate the development and commercialization of this novel branch of medicine.

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineages programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed to either replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineages clinical programs are in markets with billion dollar opportunities and include three allogeneic (off-the-shelf) product candidates: (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase 1/2a development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of acute spinal cord injuries; and (iii) VAC, an allogeneic dendritic cell therapy platform for immuno-oncology and infectious disease, currently in clinical development for the treatment of non-small cell lung cancer. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

See more here:
Lineage Posts Series of Webinars Highlighting Recent Progress With its Three Clinical-Stage Allogeneic Cell Transplant Programs - Business Wire

Newswise New research from the University of Iowa and University Hospitals Cleveland Medical Center demonstrates that offspring can be protected from the effects of prenatal stress by administering a neuroprotective compound during pregnancy.

Working in a mouse model, Rachel Schroeder, a student in the UI Interdisciplinary Graduate Program in Neuroscience, drew a connection between the work of her two mentors, Hanna Stevens, MD, PhD, UI associate professor of psychiatry and Ida P. Haller Chair of Child and Adolescent Psychiatry, and Andrew A. Pieper, MD, PhD, a former UI faculty member, now Morley-Mather Chair of Neuropsychiatry at Case Western Reserve University and Investigator and Director of the Neurotherapeutics Center at The Harrington Discovery Institute at University Hospitals.

Stevenss lab studies the long-lasting impact of stress during pregnancy, which can lead to neuropsychiatric impairment in offspring during early life and in adulthood. Piepers lab focuses on discovery of neuroprotective treatments, exemplified by the pharmacologic agent used here, known as P7C3-A20, which has previously been shown to protect the adult brain from injury.

Schroeder spent time in both labs early in her graduate school career and was inspired to bring the two lines of research together in her own work, investigating the potential impact that P7C3-A20 might have in protecting the embryonic brain during adverse events in pregnancy. Her work is the first to explore the therapeutic potential of prenatal exposure to P7C3 compounds.

Prenatal stress increases the risk for offspring to have neurodevelopmental problems, Schroeder said. We wanted to know whether the P7C3-A20 compound protected the embryonic brain from damage. Our results show that offspring are protected from the negative effects of stress when the mothers are treated with P7C3-A20 during the same time.

The research was published online today in the journal Antioxidants & Redox Signaling.

Previous work by Piepers lab has shown that P7C3-A20 enables nerve cells to maintain normal levels of an energy molecule known as nicotinamide adenine dinucleotide (NAD+), under toxic or injury conditions that are otherwise overwhelming and energy-depleting for the cell.

Schroeders research showed that chronic prenatal stress in mice disrupted the embryonic brains NAD+-synthesis machinery, which led to degeneration of nerve cell axons, learning deficits, and depression-like behavior when the offspring reached adulthood. Schroeder demonstrated that when prenatally-stressed pregnant mice were simultaneously treated with P7C3-A20, their offspring were protected from these negative effects.

By stabilizing critical NAD+-producing mechanisms, we enabled the developing embryonic brain to continue developing normally despite the stress, Schroeder said.

Though there are many challenges associated with administering medicines during pregnancy, Rachel Schroeders discovery represents an exciting move forward in understanding how prenatal stress harms the brain, and strategies for protecting the developing embryo. said Pieper, who is also Psychiatrist at the Louis Stokes VA Medical Center in Cleveland.

This study represents an important proof of concept for a new approach to early prevention of neuropsychiatric problems, Stevens said. Neuropsychiatric problems are the most common chronic illnesses of young people, which means we need many more ways to protect the brain as it develops. Our lab is focused on mechanisms of brain development prenatally, a critical time when we could make a difference.

In addition to Schroeder, Pieper and Stevens, the research team included Lynn Nguyen and Alexandra Loren in the Stevens Lab at UI; Preethy Sridharan, Coral J. Cintrn-Prez, and Edwin Vzquez-Rosa in the Pieper Lab at the Harrington Discovery Institute; and Noelle S. Williams and Kavitha P. Kettimuthu at the University of Texas Southwestern Medical Center.

###

Funding was provided by the Brockman Foundation, the Elizabeth Ring Mather & William Gwinn Mather Fund, S. Livingston Samuel Mather Trust, G.R. Lincoln Family Foundation, Wick Foundation, Gordon & Evie Safran, the Leonard Krieger Fund of the Cleveland Foundation, the Maxine and Lester Stoller Parkinsons Research Fund, the Louis Stokes VA Medical Center resources and facilities, Project 19PABH134580006-AHA/Allen Initiative in Brain Health and Cognitive Impairment, a Junior Research Program of Excellence from the Roy J. Carver Charitable Trust, Nellie Ball Trust, NIH grant R01 MH122485-01, UI Environmental Health Science Research Center and the UI Graduate College.

About University of Iowa Health CareUniversity of Iowa Health Careis the states only comprehensive academic medical center, dedicated to providing world-class health care and health-related outreach services to all Iowans. Based in Iowa City, UI Health Care includesUniversity of Iowa Hospitals & Clinics, University of Iowa Roy J. and Lucille A. Carver College of Medicine, andUniversity of Iowa Physicians, the states most comprehensive multi-specialty physician group practice.

About University Hospitals / Cleveland, OhioFounded in 1866, University Hospitals serves the needs of patients through an integrated network of 19 hospitals, more than 50 health centers and outpatient facilities, and 200 physician offices in 16 counties throughout northern Ohio.The systems flagship academic medical center, University Hospitals Cleveland Medical Center, located in Clevelands University Circle, is affiliated with Case Western Reserve University School of Medicine. The main campus also includes University Hospitals Rainbow Babies & Children's Hospital, ranked among the top childrens hospitals in the nation; University Hospitals MacDonald Women's Hospital, Ohio's only hospital for women; University Hospitals Harrington Heart & Vascular Institute, a high-volume national referral center for complex cardiovascular procedures; and University Hospitals Seidman Cancer Center, part of the NCI-designated Case Comprehensive Cancer Center. UH is home to some of the most prestigious clinical and research programs in the nation, including cancer, pediatrics, women's health, orthopedics, radiology, neuroscience, cardiology and cardiovascular surgery, digestive health, transplantation and urology. UH Cleveland Medical Center is perennially among the highest performers in national ranking surveys, including Americas Best Hospitals from U.S. News & World Report. UH is also home to Harrington Discovery Institute at University Hospitals part of The Harrington Project for Discovery & Development. UH isone of the largest employers in Northeast Ohio with 28,000 physicians and employees.Advancing the Science of Health and the Art of Compassion is UHs vision for benefitting its patients into the future, and the organizations unwavering mission is To Heal. To Teach. To Discover.Follow UH on LinkedIn, Facebook @UniversityHospitalsand Twitter @UHhospitals. For more information, visitUHhospitals.org.

About Harrington Discovery Institute The Harrington Discovery Institute at University Hospitals in Cleveland, OHpart of The Harrington Project for Discovery & Developmentaims to advance medicine and society by enabling our nations most inventive scientists to turn their discoveries into medicines that improve human health.The institute was created in 2012 with a $50 million founding gift from the Harrington family and instantiates the commitment they share with University Hospitals to a Vision for a Better World. For more information, visit: HarringtonDiscovery.org.

Case Western Reserve University is one of the country's leading private research institutions. Located in Cleveland, we offer a unique combination of forward-thinking educational opportunities in an inspiring cultural setting. Our leading-edge faculty engage in teaching and research in a collaborative, hands-on environment. Our nationally recognized programs include arts and sciences, dental medicine, engineering, law, management, medicine, nursing and social work. About 5,100 undergraduate and 6,700 graduate students comprise our student body. Visitcase.eduto see how Case Western Reserve thinks beyond the possible.

Louis Stokes Cleveland VA Medical Center is the hub of VA Northeast Ohio Healthcare System, providing and coordinating primary, acute and specialty care. Focusing on treating the whole Veteran through health promotion and disease prevention, VA Northeast Ohio Healthcare System delivers comprehensive, seamless health care and social services for more than 112,000 Veterans at 18 locations across Northeast Ohio. VA Northeast Ohio Healthcare System contributes to the future of medicine through education, training and research programs. For more information visit http://www.cleveland.va.gov.

Read more from the original source:
Iowa and Ohio Researchers Discover New, Protective Strategy for Embryonic Development during Prenatal Stress in Animal Model - Newswise

REDWOOD CITY, Calif.--(BUSINESS WIRE)--Guardant Health, Inc. (Nasdaq: GH). Despite guideline recommendations for comprehensive genomic profiling (CGP) of all patients with advanced non-small cell lung cancer (NSCLC), profiling remains suboptimal due to continued reliance on invasive tissue biopsies for testing. A new study published in JCO Precision Oncology confirms previously reported data showing that the Guardant360 liquid biopsy is not only concordant to tissue genotyping, but detects significantly more informative alterations when used prior to tissue testing, and achieves similar treatment response rates and progression-free survival in patients with NSCLC. Publication link here.

The prospective study1 (n=186) compares comprehensive genomic profiling using the Guardant360 liquid biopsy versus standard-of-care tissue genotyping for first-line treatment decisions in advanced NSCLC. Patients with advanced NSCLC received targeted therapies based on the actionable biomarkers identified. Compared to tissue biopsy genotyping at time of diagnoses, the Guardant360 liquid biopsy was not only concordant with tissue biopsy but detected 23.6 percent more informative mutations when used first and before tissue biopsy.

The study adds to the growing body of evidence2-4 demonstrating that using a blood-first approach using the Guardant360 test for genomic biomarker detection can identify more actionable biomarkers than a tissue-first approach. Importantly, objective response rates and progression-free survival in biomarker-positive patients receiving targeted therapy was similar to previously reported registrational trials.

Despite the ever-growing availability of life-changing targeted drugs for treating patients with advanced lung cancer, many continue to be treated with chemotherapy or immunotherapy because first-line treatments are made without conducting comprehensive genotyping first, said the Principal Investigator of this study Dr. Rafael Rossell, Chief Medical Officer and President of the Dr. Rosell Oncology Institute. This publication outlines further evidence that the Guardant Health liquid biopsy is very effective in uncovering actionable genomic alterations, overcomes the challenges of tissue biopsies, and helps clinicians more easily customize treatments to improve the prognosis and survival of their patients.

Numerous clinical studies show that patients receiving targeted or personalized treatments have improved progression-free survival and higher overall response rates compared to chemotherapy or immunotherapy.5-11 Various factors contribute to clinical adoption of personalized medicine lagging behind recommended medical guidelines, including insufficient tissue for biopsy, which is the case for as many as 30 percent of solid cancer patients.12-14

Once again, the data show that our blood-first approach using our Guardant360 liquid biopsy has the advantage of increasing the number of patients receiving potentially life-changing targeted treatments without compromising treatment efficacy, said Helmy Eltoukhy, Guardant Health CEO. Sadly, research indicates that approximately 80 percent of advanced lung cancer patients do not receive comprehensive genotyping before starting treatment. I hope that with the recent FDA approval of our Guardant360 CDx liquid biopsy test, more clinicians will feel confident making the shift to liquid biopsies so we can reverse the serious trend of undergenotyping that exists today.

About Guardant Health

Guardant Health is a leading precision oncology company focused on helping conquer cancer globally through use of its proprietary blood tests, vast data sets and advanced analytics. The Guardant Health Oncology Platform leverages capabilities to drive commercial adoption, improve patient clinical outcomes and lower healthcare costs across all stages of the cancer care continuum. Guardant Health has launched liquid biopsy-based Guardant360, Guardant360 CDx, and GuardantOMNI tests for advanced stage cancer patients. These tests fuel development of its LUNAR program, which aims to address the needs of early stage cancer patients with neoadjuvant and adjuvant treatment selection, cancer survivors with surveillance, asymptomatic individuals eligible for cancer screening and individuals at a higher risk for developing cancer with early detection.

Forward-looking Statements

This press release contains forward-looking statements within the meaning of federal securities laws, including statements regarding the potential utilities, values, benefits and advantages of Guardant Healths liquid biopsy tests or assays, which involve risks and uncertainties that could cause the actual results to differ materially from the anticipated results and expectations expressed in these forward-looking statements. These statements are based on current expectations, forecasts and assumptions, and actual outcomes and results could differ materially from these statements due to a number of factors. These and additional risks and uncertainties that could affect Guardant Healths financial and operating results and cause actual results to differ materially from those indicated by the forward-looking statements made in this press release include those discussed under the captions Risk Factors and Managements Discussion and Analysis of Financial Condition and Results of Operation and elsewhere in its Annual Report on Form 10-K for the year ended December 31, 2019, in its Quarterly Reports on Form 10-Q for the periods ended March 31, 2020, June 30, 2020, and September 30, 2020, respectively, and in its other reports filed with the Securities and Exchange Commission. The forward-looking statements in this press release are based on information available to Guardant Health as of the date hereof, and Guardant Health disclaims any obligation to update any forward-looking statements provided to reflect any change in its expectations or any change in events, conditions, or circumstances on which any such statement is based, except as required by law. These forward-looking statements should not be relied upon as representing Guardant Healths views as of any date subsequent to the date of this press release.

References

Link:
Head-to-Head Study Shows Guardant360 Liquid Biopsy Outperforms Tissue Biopsy for Comprehensive Genomic Profiling in Advanced Non-Small Cell Lung...

HANOVER Shortly after Chinese researchers published the genetic sequence of the virus that causes COVID-19 a little more than a year ago, a group of U.S. researchers designed what would become key components of the Moderna and Pfizer vaccines that began making their way into peoples arms last month.

The speed of the vaccines development was possible because decades of research had laid the groundwork for them, said Jason McLellan, whose molecular biosciences laboratory was based at the Geisel School of Medicine at Dartmouth from 2013 to early 2018, before it moved to the University of Texas at Austin. His move was necessary, he said in a phone interview earlier this month, because UT has the multimillion-dollar microscopes he needs for his structural biology work.

People shouldnt be alarmed by how quickly these vaccines were developed, McLellan said, adding that much of the speed was possible due to the simultaneous manufacturing of the vaccines while clinical trials were ongoing, knowing that if they didnt work they would be tossed.

McLellan and his collaborators, including teams at Scripps Research and the Vaccine Research Center at the National Institute of Allergy and Infectious Diseases, had previously described coronavirus spike proteins or that club-like protein that sticks out from the virus, as McLellan explained. They focused on the coronavirus family because earlier infectious disease outbreaks such as Middle East respiratory syndrome (MERS) and severe acute respiratory syndrome (SARS) were caused by other members of the same family and they knew another could strike again.

The researchers described both how these spikes look and how they act, which is like transformers to bind to a cell and infect it, he said.

They also knew, based on their prior work, that they could manipulate the spike proteins and stabilize them, preventing them from binding to cells or causing infection. The stabilized proteins can stimulate the immune system to produce antibodies to respond in the event that it is faced with the virus.

All this background work meant that when the Chinese researchers published the genetic sequence of SARS-CoV-2, the virus that causes COVID-19, on Jan. 10, 2020, McLellans team was ready to determine the structure of the spike protein and identify how to stabilize it.

The mRNA included in the Pfizer and Moderna vaccines, which are now gradually being distributed across the United States and elsewhere, instruct immune cells to make the stabilized protein to stimulate an immune response. The stabilized proteins McLellan and others developed also are part of the Johnson & Johnson and Novavax vaccines, which are still going through clinical trials.

Charles Barlowe, chair of biochemistry and cell biology at Geisel, credited McLellan with being an exceptional scientist and described McLellans finding of how to introduce mutations to stabilize spike proteins as a very significant advance.

Barlowe also credited McLellan with being a wonderfully collaborative scientist who works in large groups to answer questions.

One of the members of McLellans team is Daniel Wrapp, a post doctoral fellow in McLellans lab who earned his Ph.D. from Dartmouth in November. Wrapp followed McLellan to Austin, as a graduate student, but remained enrolled at Dartmouth.

Wrapp said he enjoyed his time at Geisel in part because of the collaborative spirit engendered by the Upper Valleys relative isolation.

I think having that sort of physical isolation fosters a real sense of community amongst people working right next door, he said.

In some ways the scientists behind-the-scenes work has remained the same amid the pandemic, Wrapp said in a phone interview. But, he said, it has added urgency. And when some of his friends who are physicians began getting the shots and texting him about it last month, that was really exciting, Wrapp said.

McLellans wife, mother and sister all got their first shot of the Pfizer vaccine earlier this month, he said, noting that his wife and sister work in health care. Wrapp, who is 27, said he expects he wont get it for a while and even his grandmother in Florida has been having trouble getting a shot.

Im definitely sympathetic to the desire to get back to normal, Wrapp said.

McLellan said hes experienced an odd mixture of emotions this year. Its been exciting to see his work directly translate to have a positive impact on human health. His group also has been involved in the development of Eli Lillys bamlanivimab antibody treatment for COVID-19. But in the meantime he and his team have to work in the lab in shifts to limit their contact with others. And hes watched as hundreds of thousands of people have died the U.S. recently surpassed 400,000 deaths due to COVID-19.

The researchers future goals include developing a universal coronavirus vaccine that would inoculate people against all viruses in the family.

Science takes time, McLellan said. We fail a lot. Over time the field moves in the right direction.

Nora Doyle-Burr can be reached at ndoyleburr@vnews.com or 603-727-3213.

Follow this link:
COVID-19 researcher has ties to Geisel School of Medicine - Valley News

A chain reaction led by cells lining the intestines tips the immune system off to the presence of the parasite Cryptosporidium, according to a study led by researchers in the School of Veterinary Medicine.

To effectively combat an infection, the body first has to sense its been invaded, then the affected tissue must send out signals to corral resources to fight the intruder. Knowing more about these early stages of pathogen recognition and response may provide scientists with crucial clues when it comes to preventing infections or treating inflammatory diseases resulting from overactive immunity.

That was the intent behind a new study, led by researchers at the University of Pennsylvania School of Veterinary Medicine, examining infection with the parasite Cryptosporidium. When the team looked for the very first danger signals emitted by a host infected with the parasite, they traced them not to an immune cell, as might have been expected, but to epithelial cells lining the intestines, where Cryptosporidium sets up shop during an infection. Known as enterocytes, these cells take up nutrients from the gut, and here they were shown to alert the body to danger via the molecular receptor NLRP6, which is a component of whats known as the inflammasome.

You can think about the inflammasome as an alarm system in a house, says Boris Striepen, a professor in the Department of Pathobiology at Penn Vet and senior author on the paper, which is publishing in the journal Proceedings of the National Academy of Sciences. It has various components--like a camera that watches the door, and sensors on the windows--and once triggered it amplifies those first signals to warn of danger and send a call for help. Cells have these different components as well, and now weve provided maybe the clearest example yet of how a particular receptor in the gut is acting as a sensor for an important intestinal infection.

Typically, Striepen says, researchers have focused on immune cells, like macrophages and dendritic cells, as being the first to detect foreign invaders, but this new finding underscores that cells not normally thought of as part of the immune system--in this case intestinal epithelial cells--are playing key roles in how an immune response gets launched.

There is a growing body of literature that is really appreciating what epithelial cells are doing to help the immune system sense pathogens, says Adam Sateriale, first author on the paper who was a postdoc in Striepens lab and now leads his own lab at the Francis Crick Institute in London. They seem to be the first line of defense against infection.

Striepens lab has devoted considerable attention to Cryptosporidium, which is a leading cause of diarrheal disease that can be deadly in young children in resource-poor areas around the world. Cryptosporidium is also a threat to people in well-resourced environments, causing half of all water-borne disease outbreaks in the United States. In veterinary medicine, its known for infecting calves, stunting their growth. These infections have no effective treatment and no vaccine.

In the current work, Striepen, Sateriale, and colleagues took advantage of a naturally occurring species of mouse Cryptosporidium that they recently discovered mimics human infection in many respects. While the researchers knew T cells help control the parasite in later stages of infection, they began looking for clues as to what happens first.

One important clue is the unfortunate linkage between malnutrition and Cryptosporidium infection. Early infection with Cryptosporidium and the inflammation of the intestine that goes along with it predisposes children to malnutrition and stunted growth; at the same time, children who are malnourished are more susceptible to infection. This can lead to a downward spiral, putting children at greater risk of deadly infections. The mechanisms behind this phenomenon are not well understood.

That led us to think that maybe some of the danger-sensing mechanisms that can drive inflammation in the gut also play a role in the larger context of this infection, adds Striepen.

Together these linkages inspired the research team to look more closely at the inflammasome and its impact on the course of infection in their mouse model. They did so by removing a key component of the inflammasome, an enzyme called caspase-1. It turns out that animals that are missing this had much higher levels of infection, Sateriale says.

Further work demonstrated that mice lacking caspase-1 just in intestinal epithelial cells suffered infections as high as those lacking it completely, demonstrating the crucial role of the epithelial cell.

Consistent with this idea, the Penn Vet-led team showed that, out of a variety of candidate receptors, only loss of the NLRP6 receptor leads to failure to control the infection. NLRP6 is a receptor restricted to epithelial barriers previously linked to sensing and maintaining the intestinal microbiome, bacteria that naturally colonize the gut. However, experiments revealed that mice never exposed to bacteria, and thus lacked a microbiome, also activated their inflammasome upon infection with Cryptosporidium--a sign that this aspect of danger signalling occurs in direct response to parasite infection and independent of the gut bacterial community.

To trace how triggering the intestinal inflammasome led to an effective response, the researchers looked at some of the signalling molecules, or cytokines, typically associated with inflammasome activation. They found that infection leads to the release of IL-18, with those animals that lack this cytokine or the ability to release it showing more severe infection.

And when you add back IL-18, you can rescue these mice, Sateriale says, nearly reversing the effects of infection.

Striepen, Sateriale, and colleagues believe theres a lot more work to be done to find a vaccine against Cryptosporidium. But they say their findings help illuminate important aspects of the interplay between the parasite, the immune system, and the inflammatory response, relationships that may inform these translational goals.

Moving forward, they are looking to the later stages of Cryptosporidium infection to see how the host successfully tamps it down. Now that we understand how the infection is detected, wed like to understand the mechanisms by which it is controlled, Sateriale says. After the system senses a parasite, what is done to restrict their growth and kill them?

(This story has been published from a wire agency feed without modifications to the text. Only the headline has been changed.)

Follow more stories on Facebook and Twitter

Originally posted here:
Gut cells sound the alarm when parasites invade: Study - Hindustan Times

Researchersarepoised to makeunprecedentedbreakthroughsinhuman health thanks toadvancesin biomedical and computational sciencesthathave drivencritical tools and technologiessuch as genetic engineering,synthetic biology, andartificial intelligence.

Thats the messageDr. VictorDzau, president oftheU.S.National Academy ofMedicine, delivered to Medicine by Designs fifthannual symposium on Dec. 7 and 8.

Thevirtual event, whichattracted more than 500 registrants from across North America, focused on the theme of better science throughconvergence theintegration of approaches from engineering, science, medicine and other fields to expand knowledge and spark innovation.

I think for younger people, there is really not a more exciting time, in my opinion, to do research than now, because we can really see that some of the initial concepts that people have about health and medicinecan be realizedand truly transform the way we do health andmedicine.

In his talk, Dzau focused on the National Academy of MedicinesHealthy Longevity Global Challenge,an international competition that aims to catalyze transformative ideas and breakthroughs that will extend human healthand lifespan.

That program is one of the inspirations for Medicine by DesignsGrand Questions Program, which seeks to fund bold research that promises dramatically better health outcomes by changing the future of regenerative medicine.

Through our Grand Questions Program, we are thinking about what comes next and how to overcome fundamental problems in regenerative medicine,saidMichael Sefton, executive director of Medicine by Design andUniversity Professorin the department of chemical engineering and applied chemistry and theInstitute of Biomedical Engineeringat the University of Toronto.

We have a broad definition of regenerativemedicine, andpreventing degeneration can be as important as the next cell therapy.

Sefton pointed out that the symposium theme of better science through convergencefocusedon a key aspect of Medicine by Design:That we combine campus and hospital investigators, transformative science and translational elements, junior and senior investigators, and local and international collaborators, to address fundamental problems in regenerative medicine.

Thesymposium also featured a talk byRobert Langer, David H. KochInstitute Professorin the department of chemical engineeringat the Massachusetts Institute of Technology. The most highly cited engineer in history, he spoke about lessons helearnedfromhisscientific and business successes and how he decidedto be his own champion after facing criticism for his novel ideas early in his career.

If you try to do things whether its convergence, or things that a lot of people disagree with you have tohang in there, Langer said.Having good intellectual property has been key toraising the funds to do these things, and medicine is an incredibly expensive thing.

And finally, you really need teams that are super driven, and I think these startup companies have been a wonderful way to do this.

The symposium was organized around four sessions: translation, inflammation, biomaterials andimmunoengineering.Invited speakers from across North AmericaincludedKim Warren(AVROBIO),Kenneth Walsh(University of Virginia),Sarah Heilshorn(Stanford University)andMegan Levings(University of British Columbia).

All speakers fromU of T and its partnerhospitals were lead investigators on Medicine by Designs multi-disciplinary, multi-institution team projects. They included:John Dick,Clinton RobbinsandShaf Keshavjee(University Health Network (UHN));Molly Shoichet(department of chemical engineering and applied chemistry and Institute of Biomedical Engineering);Juan CarlosZiga-Pflcker(Sunnybrook Health Sciences Centre);andAndras Nagy(Sinai Health System).

Ted Sargent, vice-president of research and innovation, and strategic initiatives,and a University Professor in the Edward S. Rogers Sr. department of electrical and computer engineering,opened the symposium by congratulatingMedicine by Design on its successful mid-term review, which was conducted in early 2020 by a panel of international experts and theCanada First Research Excellence Fund(CFREF), which funds Medicine by Design.

Medicine by Design has amplified existing areas ofexcellenceatU of Tandour partner hospitals (Toronto Academic Health Sciences Network),and pushed the boundaries of regenerative medicine to tackle cell-based therapies, strategies for endogenous repair and the use of a stem cell lens to target the triggers of disease,Sargent said. In fact, Medicine by Design is such a compelling collaborative, cross-disciplinary initiative that itis a template fora new class of initiatives at the University ofToronto theInstitutional Strategic Initiativesportfolio whosepurpose is to mobilize ambitious,groundbreaking, collaborative, multi-institutional research networks that tackleimportantresearch problems, buildmajorexternal partnershipsboth with industry and emerging companies as well as with global academic peers;and foster societal impact.

They support the pursuit of grand challenges and bold ideas across disciplinary boundaries,further elevate U of Ts profile in high priority research areas of strategic importance,and enable us to realize transformational impacts on issues of major societal import.

The symposium also offered an opportunity for almost 45trainees to present their research during a poster session.KerstinKaufmann, a post-doctoral fellow in the laboratory ofJohn Dick(Princess Margaret Cancer Centre,UHN), won first place.JonathanLabriola, apost-doctoral fellowinSachdev Sidhuslab(Donnelly Centre for Cellular and Biomolecular Research, U of T), placed second, whileSabihaHacibekiroglu, a post-doctoral fellow in the lab ofAndras Nagy(Lunenfeld-Tanenbaum Research Institute, UHN)placed third.The awards were sponsored by STEMCELL Technologies.

YasamanAghazadeh,a post-doctoral fellow in the labsofCristina Nostro(McEwen Stem Cell Institute, UHN)andSara Nunes Vasconcelos(Toronto General Hospital Research Institute,UHN),won theCCRMTranslation Awardfor the poster with the greatest translational potential.AndAi Tian, a post-doctoral fellow fromJulien Muffatslab (The Hospital for Sick Children), won thePeoples Choice Award, a new award this year that wasdetermined byvotingby symposium attendeesand sponsored byBlueRockTherapeutics.

Funded by a $114-million grant from CFREF, Medicine by Design brings together more than 145principal investigators at the University of Toronto and its affiliated hospitals to work at the convergence of engineering,medicineand science. It builds on decades of made-in-Canada excellence in regenerative medicine dating back to the discovery of stem cells in the early 1960s by Toronto researchers James Till andErnest McCulloch.

Regenerative medicine uses stem cells to replace diseased tissues and organs, creating therapies in which cells are the biological product. Regenerative medicine can also mean triggering stem cells that are already present in the human body to repair damaged tissues or to modulate immune responses. Increasingly, regenerative medicine researchers are using a stem cell lens to identify critical interactions or defects that prepare the ground for disease, paving the way for new approaches to preventing disease before it starts.

(Photo of Robert Langer by Jason Alden)

Go here to see the original:
Medicine by Design symposium highlights importance of convergence in regenerative medicine and human health - News@UofT

Dec. 28, 2020 13:04 UTC

BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced its New Drug Submission for TRIKAFTA, Vertexs investigational triple combination medicine, has been accepted for Priority Review by Health Canada for the treatment of cystic fibrosis (CF) in people ages 12 years and older.

We are pleased this submission has been accepted for Priority Review by Health Canada, and we anticipate this accelerated review process will enable access for patients as early as possible, said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex.

With Priority Review, the conventional review timeline of 300 days is reduced to 180 days. The expected approval target by Health Canada is in the first half of 2021.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes one from each parent to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create medicines for people with serious diseases. The company has multiple approved medicines that treat cystic fibrosis (CF) a rare, life- threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertexs history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Carmen Bozic in this press release, including expectations for patient access to our medicine, and statements regarding the anticipated timing of the expected approval target by Health Canada. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that the New Drug Submission to Health Canada may not be approved in the expected timeline, or at all, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under the heading Risk Factors in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at http://www.sec.gov and available through the company's website at http://www.vrtx.com. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

View source version on businesswire.com: https://www.businesswire.com/news/home/20201228005171/en/

See the article here:
Vertex Announces New Drug Submission for Investigational Triple Combination Medicine for the Treatment of Cystic Fibrosis Has Been Accepted for...

The treatment landscape of lymphoid malignancies space is rich with available immunotherapy agents, said Joshua Brody, MD. However, the field continues to push toward improving response rates by refining established modalities, such as CAR T-cell therapy and checkpoint inhibitors, as well as introducing novel modalities.

During the5th AnnualInternational Congress on Immunotherapies in Cancer, a program run by Physicians Education Resource (PER), Brody, an assistant professor of medicine, hematology, and medical oncology and director of the Lymphoma Immunotherapy Program at The Tisch Cancer Institute at Mount Sinai, gave a presentation on established and upcoming immunotherapies in the lymphoid malignancy pipeline.

We are somewhat lucky, and maybe a bit spoiled in lymphoid malignancies, such as lymphomas and some B-cell malignancies, to have [seen] a huge amount of progress [with immunotherapy], said Brody.

However, lymphoid malignancies are highly heterogeneous. As such, not all patients are able to derive responses from the same therapy, Brody explained.

CAR T-cell therapy represents a significant advance in the treatment paradigms of several hematologic malignancies, including lymphoid malignancies, said Brody.

The results [of CAR T-cell therapy treatment] for patients with aggressive B-cell lymphomas have been amazing, Brody said. [CAR T-cell therapy] has shown higher response rates [in this space] than any other immunotherapy for any type of cancer; response rates are above 80%. Many of those [responses] are long lasting, complete remissions [CRs].

Notably, it is thought that patients who remain in CR for at least 6 to 12 months after CAR T-cell therapy infusion are likely cured of their lymphoma, Brody said. For example, it is likely that around 35% to 40% of patients with diffuse large B-cell lymphoma (DLBCL) who receive CAR T-cell therapy are effectively cured.

Third-line [DLBCL], the most common type of lymphoma, [was thought to be] an incurable disease setting, said Brody. We [thought we could] cure people in the first- or second-line setting, but not beyond. Im not sure how we define a miracle, but it seems miraculous to me to change the incurable to partly curable.

Now, ongoing research efforts are attempting to refine CAR T-cell therapies to enhance responses and expand the utility of the modality to other hematologic malignancies beyond DLBCL, acute leukemia, and mantle cell lymphoma, explained Brody. For example, developing armored CAR T cells and fourth-generation CAR T cellsT cellsredirected for antigenunrestricted cytokineinitiated killingmay allow for improved delivery of cytotoxic payloads to liquid as well as solid tumors.

Additionally, novel CAR T-cell therapies may overcome some of the ongoing challenges observed with current, autologous products, such as toxicity, accessibility, and feasibility. For example, utilizing an allogeneic approach to CAR T-cell therapy may allow for an off-the-shelf option that could alleviate the risk of cytokine release syndrome and neurotoxicity.

Beyond CAR T-cell therapy, bispecific monoclonal antibodies, such as the investigational CD20/CD3-directed REGN1979 agent, could potentially be used in patients who progress after CAR T-cell therapy. Findings from a phase 1 trial (NCT02290951) demonstrated high objective response rates and CR rates among patients with heavily pretreated, relapsed/refractory NHL who were treated with REGN1979.1

Although the toxicity challenges of CAR T-cell therapies are likely not going to be solved with bispecific antibodies, the novel agents may offer an alternative option that can decrease the risk of adverse effects.

At least with [bispecific antibodies], we can tweak and optimize [dosing] a bit, explained Brody. CAR T-cell therapy is usually a one-shot therapy, [whereas] bispecifics can be given in low, medium, or high doses. We can up-titrate the therapy to try to minimize or avoid some of these toxicities.

Additionally, bispecific antibodies are potentially able to avoid antigen escape, which is a common concern with CD19/CD20-directed CAR T-cell therapies, Brody explained.

PD-1/PD-L1 inhibitors have transformed the treatment landscape in melanoma, lung cancer, renal cell carcinoma, and bladder cancer. However, Hodgkin lymphoma is the most responsive disease to antiPD-1 agents because chromosomal amplifications or translocations are hardwired to overexpress PD-1, Brody said.

As such, responses with checkpoint inhibitors, such as nivolumab (Opdivo), are seen in around 70% of patients with Hodgkin lymphoma who progress following autologous stem cell transplant.2

The introduction of checkpoint inhibitors to this space has been particularly encouraging for younger patients who fail curative-intent chemotherapy and who, historically, had dismal prognoses.

Currently, ongoing efforts are attempting to bring PD-1 inhibitors to the second-line setting and, potentially, up-front settings in combination with chemotherapy.

Although it is true that hot tumors are more likely to respond to immunotherapy compared with cold tumors, it is not the only factor that contributes to whether a tumor will respond. As such, alternative methods of improving immune response in patients with lymphoid malignancies are being evaluated.

For example, one investigational method is to utilize radiotherapy or other means to mobilize dendritic cells loaded with tumor antigens to antigen-presenting cells. Ultimately, this compound could serve as a therapeutic anti-cancer vaccine to deliver the antigens, as well as costimulatory signals, to the cancer cells.

As such, an ongoing trial (NCT03789097) led by Brody is currently testing an in situ vaccine combining radiation therapy, the immune cell growth factor Flt3L/CDX-301, the immune-cell activating factor Poly-ICLC, and pembrolizumab (Keytruda).3 Initial findings from the study have yielded encouraging results, according to Brody.

In situ vaccines such as this may be able to cross prime T cells and increase the effectiveness of immunotherapy for patients, Brody concluded.

View original post here:
Immunotherapy Continues on Positive Trajectory in Lymphoid Malignancies - OncLive