Alzheimer's - Dementia, Author Interviews, Genetic Research, Mental Health Research / 31.07.2018

MedicalResearch.com Interview with: The Jackson LaboratoryCatherine Kaczorowski, Ph.D. Associate Professor and Evnin Family Chair in Alzheimer's Research Kristen O’Connell, Ph.D., Assistant Professor Amy Dunn, Ph.D., Postdoctoral Associate The Jackson Laboratory MedicalResearch.com: What is the background for this study? What are the main findings?  Dr. Amy Dunn: “Alzheimer's disease is complex, with both genetic and environmental factors determining symptom onset and disease progression, though our current understanding of how genetic and environmental factors interact to influence disease risk is incomplete. We recently developed a panel of genetically diverse mice carrying human familial AD mutations (AD-BXDs) that better model human AD in order to determine how genetics and diet interact to modify disease onset and severity. We fed a high fat diet to AD-BXDs and monitored metabolic and cognitive function over the duration of the HFD feeding.  We observed accelerated working memory decline in most of the AD-BXD mouse strains, however, the impact of high fat diet on memory was dependent on individual genetic differences across the panel, with some AD-BXD strains maintaining cognitive function on high fat diet (resilient strains). Our data suggest that diet and genetic background interact to mediate vulnerability to AD pathogenesis, and that metabolic factors (e.g. obesity, body composition) that may contribute to cognitive decline differentially in normal aging versus AD. “ (more…)
Author Interviews, JAMA, Rheumatology, Statins / 31.07.2018

MedicalResearch.com Interview with: Dr Gillian E. Caughey PhD Senior Research Fellow | School of Medicine Discipline of Pharmacology THE UNIVERSITY OF ADELAIDE Australia MedicalResearch.com: What is the background for this study? What are the main findings? Response: Statins are one of the most commonly prescribed medications worldwide.  Muscular adverse effects (myalgia and myopathy) are well recognised adverse effects and symptoms resolve with cessation of statins. Idiopathic inflammatory myositis (IIM) is a heterogeneous group of autoimmune myopathies that may also be associated with statin use. IIM does not resolve with cessation of statin therapy, requires treatment with immunosuppressive agents and is a severe, debilitating condition. To date, there have been no epidemiological studies examining exposure to statins and the association of histologically confirmed IIM. In our case control study of 221 cases o fIdiopathic inflammatory myositis, there was an almost 2-fold increased likelihood of statin exposure in patients with IIM compared with controls (adjusted odds ratio, 1.79; 95%CI, 1.23-2.60). After observing a significant association of statin exposure with IIM, we conducted a sensitivity analysis where we excluded those patients with necrotizing myositis as recent studies have reported this type of IIM to be associated with statin use and the presence of autoantibodies against HMG-CoA reductase. Exclusion of these specific cases from the analysis did not change our study findings and an increased risk of Idiopathic inflammatory myositis with statin exposure remained (adjusted OR, 1.92; 95% CI, 1.29-2.86).  This suggest the potential association of all types of Idiopathic inflammatory myositis with statin exposure. (more…)
Author Interviews, Inflammation, Kidney Disease, Personalized Medicine / 31.07.2018

MedicalResearch.com Interview with: Michael P. Madaio, MD Sydenstricker Professor and Chairman Department of Medicine Medical College of Georgia at Augusta University MedicalResearch.com: What is the background for this study? Response: Glomerulonephritis is a inflammatory disease of the kidney.  Glomeruli are the filtering units in the kidney. This is most often immunologically mediated and are autoimmune. Most therapies are directed at inhibiting the Immune/autoimmune process (immunotherapy) systemically. (more…)
Author Interviews, Duke, Genetic Research, Neurology, Pediatrics / 30.07.2018

MedicalResearch.com Interview with:  Paul C Marcogliese, Ph.D. Postdoctoral Associate, Laboratory of Dr. Hugo Bellen Department of Molecular and Human Genetics Baylor College of Medicine Houston, Texas 77030 Loren D. Pena, MD PhD Division of Human Genetics Cincinnati Children's Hospital Medical Center Department of Pediatrics University of Cincinnati Cincinnati, OH 45229 MedicalResearch.com: What is the background for this study? What are the main findings? Response: The Undiagnosed Diseases Network (UDN) is a multi-site collaboration across the US that seeks to help diagnose patients with rare disorders that are ill-defined. Dr. Loren D.M. Pena and Dr. Vandana Shashi at the Duke-Columbia clinical site of the UDN had seen a patient with a severe neurological disorder. While the patient had no symptoms at birth, the patient began falling at about 3 years of age, eventually losing motor coordination and developing seizures. In the interim, the regression has progressed to a severely debilitating state. Re-analysis of the participant’s exome data by our site bioinformatician at Columbia (Nicholas Stong) in Dr. David Goldstein’s laboratory revealed a truncating variant in the single exon gene IRF2BPL that could be the candidate disease-causing gene. The UDN clinicians at Duke then contacted the UDN Model Organism Screening Center (MOSC) led by Dr. Hugo Bellen at Baylor College of Medicine and the Howard Hughes Medical Institute for functional analysis. In parallel, four more patients were found with truncating mutations causing a similar disorder though the UDN and GeneMatcher.org. Additionally, two patients with missense variants in IRF2BPL were identified that displayed seizures and some developmental delay or autism spectrum disorder but no motor regression. Work in MOSC by Dr. Paul Marcogliese using fruit flies revealed that the IRF2BPL truncating variants are severe loss of function mutations and one of the missense variants was a partial loss of function. Additionally, it was found that the fruit fly IRF2BPL gene, called pits, is expressed in the neurons of the adult fly brain. Lowering the levels of pits by about 50% in fly neurons leads to progressive behavioural abnormalities and neurodegeneration. By combining the human genetics, bioinformatics and model organism data, IRF2BPL was found to be a novel disease-causing gene in humans. (more…)
Author Interviews, Emergency Care / 30.07.2018

MedicalResearch.com Interview with: Benjamin H. Schnapp, MD BerBee Walsh Department of Emergency Medicine Assistant Professor (CHS) Assistant Emergency Medicine Residency Program Director University of Wisconsin MedicalResearch.com: What is the background for this study? What are the main findings? Response: Medical errors harm thousands of patients per year.  There’s already a lot we know about systems errors - the ways that care delivery can go wrong.  We know less about cognitive errors, or the ways in which doctors think that can lead to bad outcomes.  An Emergency Department can feel particularly vulnerable to this type of errors - it’s a chaotic environment with patients in various states of illness, many unaccompanied, without records, or too ill to communicate well. An Emergency Department with trainee physicians can feel even more chaotic - even though they are supervised by staff physicians, resident physicians in their first few months to years of training have not yet accumulated the same level of knowledge and experience as longer-tenured doctors.  Errors that get made on the hospital floor are errors of information processing - physicians have the right information, they just don’t always do the right thing with it.  We wanted to know what kinds of errors get made in an Emergency Department with trainees.  Are the errors related to the chaos and an inability to obtain reliable information from patients?  Are they related to the trainees not having enough knowledge and experience?  Or are they like the errors that get made on an inpatient floor? Our study found that the most frequent type of errors were errors of information processing - just like on the hospital floors.  The most common types of errors we saw were physicians settling on a diagnosis prematurely and weighing the importance of findings incorrectly. Patients with abdominal problems had the highest number of errors in our study.  Patients with certain risk factors, such as psychiatric disease or substance abuse, seemed to be particularly prone to errors. (more…)
Author Interviews, Brigham & Women's - Harvard, Gender Differences, Occupational Health, Sexual Health / 30.07.2018

MedicalResearch.com Interview with: Brittany M. Charlton, ScD, Assistant Professor Harvard Medical School Harvard T.H. Chan School of Public Health Boston Children's Hospital Boston, MA MedicalResearch.com: What is the background for this study? Response: Research has shown that nearly half of all sexual minorities (e.g., lesbian, gay, and bisexual individuals) experience employment discrimination in their lifetime, which may lead to many other disparities, including health insurance coverage, healthcare access, and ultimately health-related quality of life (e.g., pain, anxiety). (more…)
Author Interviews, Cancer Research, JAMA, Leukemia, Transplantation / 30.07.2018

MedicalResearch.com Interview with: Smita Bhatia, MD, MPH Gay and Bew White Endowed Chair in Pediatric Oncology Professor, Pediatric Oncology Vice Chair for Outcomes Research, Dept of Pediatrics Director, Institute for Cancer Outcomes and Survivorship School of Medicine University of Alabama at Birmingham Associate Director for Outcomes Research UAB Comprehensive Cancer Center  MedicalResearch.com: What is the background for this study? What are the main findings?  Response: Allogeneic bone marrow transplantation BMT is used with a curative intent for life-threatening malignant and non-malignant diseases of childhood. In this observational study, we describe the late mortality experienced by children undergoing BMT over the past 3 decades. Our cohort included 1388 BMT recipients who had undergone allogeneic BMT between 1974 and 2010 and survived 2 or more years. We found that, conditional on surviving the first 2 years after bone marrow transplantation, the probability of surviving an additional 20 years approached 80%. Risk of dying from non-relapse-related causes exceeded the risk of dying from relapse-related causes. The leading non-relapse-related causes of death were infection (with or without graft vs. host disease) and new cancers. Overall, the cohort was at a 14-fold greater risk of dying as compared with the general population (of similar age and sex). Further, this excess risk remained elevated even among those who had survived 25 years. On a positive note, the risk of late mortality has continued to decline over the past 3 decades.  (more…)
Accidents & Violence, Author Interviews, JAMA / 30.07.2018

MedicalResearch.com Interview with: “GUNS” by Jessica Spengler is licensed under CC BY 2.0Kendrin R. Sonneville, ScD, RD Assistant Professor Department of Nutritional Sciences University of Michigan School of Public Health MedicalResearch.com: What is the background for this study? What are the main findings? Response: The results from this come out of MyVoice, a text message cohort of youth ages 14-24 representing every state in the country (http://hearmyvoicenow.org/). Each week we ask the youth in our cohort a series of open-ended to questions, with the goal of gathering the real-time opinions and experiences of youth across the U.S. This study reports the results of questions we asked MyVoice paritpatns between 07/2017 and 01/2018 about guns and gun control.  (1) What are your thoughts about having guns in your home? (2) Do you think gun control laws would affect mass shootings? Why? (3) Who, if anyone, should NOT be allowed to own guns? We found that about one-third of youth in our diverse sample were “against” guns in the home, and the remaining two-thirds were either “pro” or “conditionally pro” guns in the home, stating that gun ownership is acceptable under certain conditions, such as proper storage, or kept away from children. Youth in our sample largely believed that gun control laws could decrease mass shootings, but one-third felt that gun control laws would not be enough to impact mass shootings. (more…)
Author Interviews, JAMA, Neurology, Surgical Research / 30.07.2018

MedicalResearch.com Interview with: Jianning Zhang MD, Ph.D Chairman, II, VII Chinese Medical Association of Neurosurgery President, Tianjin Medical University General Hospital, China  Jianning Zhang MD, Ph.D Chairman, II, VII Chinese Medical Association of Neurosurgery President, Tianjin Medical University General Hospital, China   MedicalResearch.com: What is the background for this study? Response: The elderly population is growing dramatically world widely, especially in China. The incidence of chronic subdural hematoma has been rising over the past years. Although the surgery is not a difficult process, the risk of death and recurrence persist, and the affliction and economic expenditure of the patients are relatively higher in the elderly. For these reasons, it is urgent to develop novel pharmacological therapies with sufficient safety and efficacy.  It has been known that the high expression of VEGF and inflammatory factors in chronic subdural hematoma can lead to abundant angiogenesis of immature vessels on the wall of hematoma. In our previous study, patients with chronic subdural hematoma have impaired ability to promote vascular maturation. For example, the number of endothelial progenitor cells in circulating blood is about 67% of the healthy individuals with similar age.  Atorvastatin can mobilize endothelial progenitor cells to reduce inflammation. It increases the number of circulating endothelial cells that are inversely correlated with the volume of hematoma. We have demonstrated that atorvastatin can promote endothelial cell formation and reduce the leakage of endothelial cell barrier in vitro. Results from in vivo experiments in animal models of subdural hematoma suggest that atorvastatin can promote the maturation of blood vessels and reduce inflammation on the margin of hematoma, and thus improve the neurological outcome. (more…)
Author Interviews, Cannabis, McGill, Pulmonary Disease / 30.07.2018

MedicalResearch.com Interview with: Sara Abdallah, PhD Student, first author and Dennis Jensen, PhD Associate Professor, Department of Kinesiology and Physical Education Associate Dean – Infrastructure, Faculty of Education Director, McGill Research Center for Physical Activity and Health Canada Research Chair in Clinical Exercise & Respiratory Physiology Associate Member, Translational Research in Respiratory Diseases Program Research Institute of the McGill University Health Center MedicalResearch.com: What is the background for this study? What are the main findings?  Response: Many patients with chronic obstructive pulmonary disease (COPD) suffer from severe breathlessness at rest and on minimal exertion despite receiving optimal drug therapy for their underlying disease (e.g., bronchodilators). In these patients, breathlessness significantly diminishes exercise capacity and quality of life. Thus, research focused on identifying adjunct therapies for management of breathlessness in patients with advanced COPD is clinically relevant. A series of studies conducted in the 1970’s found that smoked cannabis caused bronchodilation (i.e., improved airway function) in healthy individuals and in patients with asthma. More recently, it has been demonstrated that delta-9 (∆9)-tetrahydrocannabinol (THC, the major cannabinoid constituent of cannabis) inhibits cholinergic contractions in isolated human bronchi and that a positive association exists between measure of lung function (e.g., forced expiratory volume in 1-sec) and cannabis use in patients with COPD. These studies lead us to hypothesize that inhalation of vaporized cannabis may alleviate exertional breathlessness and improve exercise tolerance in patients with advanced COPD by improving airway function at rest and during exercise. (more…)
Author Interviews, Breast Cancer, Cost of Health Care, Mammograms, Medical Imaging / 29.07.2018

MedicalResearch.com Interview with: Michal Horný PhD Assistant Professor Emory University School of Medicine, Department of Radiology and Imaging Sciences Emory University Rollins School of Public Health Department of Health Policy and Management Atlanta, GA 30322 MedicalResearch.com: What is the background for this study? Response: Increased breast tissue density is a common finding at screening mammography. Approximately 30-50% of women have so-called “dense breasts” but many of them are not aware of it. The problem is that the increased tissue density can potentially mask early cancers. In other words, if there is cancer hiding in dense breast tissue, it could be difficult to spot it. To improve the awareness of breast tissue density, a patient group called Are You Dense Advocacy, Inc., started lobbying state and federal policymakers to pass laws mandating health care providers to notify women about their breast density assessments. As a result, 31 states have already enacted some form of legislation regarding dense breast tissue. (more…)
Author Interviews, Biomarkers, NYU, Pediatrics, Pulmonary Disease / 29.07.2018

MedicalResearch.com Interview with: Dr. Mikhail Kazachkov MD Director of Pediatric Pulmonology Hassenfeld Children’s Hospital NYU Langone Medical Center  MedicalResearch.com: What is the background for this study? How common is the problem of chronic cough in children?  Is it more common in children with allergies, asthma or reflux? Response: Chronic cough is one of the leading causes of pediatric referrals to subspecialty physicians.  Its prevalence in the general pediatric population may approach 3% (Galassi et al, Epidemiol. Prev. 2005;29,Suppl.:9–13). It is important to recognize that the main causes of chronic cough in children are completely different for those in adults.  Specifically, gastroesophageal reflux and postnasal drip are not considered to be important causes of cough in children.  Cough variant asthma, although is a common cause of cough in adults, does not seem to be frequently diagnosed and a cause of chronic cough in children. The main cause of chronic wet cough in children is protracted bacterial bronchitis (Chang et al, Chest. 2017 Apr;151:884-890).  It is important to recognize that neurologically impaired children have completely different pathogenesis of chronic cough, which is mostly related to aspiration into the lower airway and development of aspiration-related lung disease. (more…)
Author Interviews, Genetic Research, Osteoporosis, PLoS, Stanford / 29.07.2018

MedicalResearch.com Interview with: Stuart Kim PhD Professor of Developmental Biology, Emeritus Bio-X Affiliated Faculty James H. Clark Center Stanford University  MedicalResearch.com: What is the background for this study? What are the main findings? Response: Osteoporosis is caused by a reduction in bone mass, and leads to a high incidence of bone fracture because the weakened bone is less able to withstand the stress of slips and falls. Osteoporosis affects millions of elderly, is responsible for as many as 50% of fractures in women and 25% of fractures in men over the age of 50, and accounts for $19 billion in annual health care costs in the US. Identification of people with an increased genetic risk for osteoporosis could reduce the incidence of bone fracture. Low BMD is also a risk factor for stress fractures. For athletes and military personnel undergoing harsh rigors of training, stress fractures are common injuries that limit playing time, military effectiveness and competitive success. Using data from UK Biobank, a genome-wide association study identified 1,362 independent SNPs that clustered into 899 loci of which 613 are new. These data were used to train a genetic algorithm using 22,886 SNPs as well as height, age, weight and sex as predictors. Individuals with low genetic scores (about 2% of those tested) showed a 17-fold increase in risk for osteoporosis and about a 2-fold increase in risk of fractures. (more…)
Aging, Alzheimer's - Dementia, Author Interviews / 27.07.2018

MedicalResearch.com Interview with: alkahestIan Gallager, MS Scientist at Alkahest Inc. San Francisco Bay Area  MedicalResearch.com: What is the background for this study? Response: Our research is aimed to develop novel therapeutics for age-related disorders from fundamental understandings of blood plasma. This expands upon work initially performed in the Wyss-Coray lab at Stanford utilizing a model of parabiosis. By surgically conjoining the blood supplies between a young and aged mouse, they established that beneficial effects were observed in the aged mouse brain, suggesting that there are proteins in young blood which have enhancing properties. The research presented at AAIC was the culmination of several years of model and dosing paradigm development utilizing both human plasma and a proprietary fractionated plasma product leading to advances for clinical application. (more…)
Author Interviews, Cancer Research, Cost of Health Care, JAMA / 27.07.2018

MedicalResearch.com Interview with: Manali Patel MD MPH Assistant Professor of Medicine, Oncology Stanford Palo Alto Veterans Affairs Health Care System   MedicalResearch.com: What is the background for this study? What are the main findings?  Response: In prior work, many patients with advanced stages of cancer report a lack of understanding of their prognosis and receipt of care that differs from their preferences. These gaps in care delivery along with the unsustainable rise in healthcare spending at the end-of-life and professional healthcare provider shortages led our team to consider new ways to deliver cancer care for patients.  Based on input from focus groups with patients, caregivers, oncology care providers and healthcare payers, we designed a novel model of cancer care to address these gaps in care delivery.  The intervention consisted of a well-trained lay health worker to assist patients with understanding and communicating their goals of care with their oncology providers and caregivers. We found that patients who received the six-month intervention reported greater satisfaction with the care they received and their decision-making, had higher rates of hospice use, lower acute care use, and 95% lower total healthcare expenditures in the last month of life.  The intervention resulted in nearly $3 million dollars in healthcare savings. (more…)
Alzheimer's - Dementia, Author Interviews, Genetic Research / 27.07.2018

MedicalResearch.com Interview with: Gregory Carter, PhD Associate Professor at The Jackson Laboratory MedicalResearch.com: What is the background for this study? What are the main findings? Response: Animal models for late-onset Alzheimer’s disease (LOAD) will be of significant benefit for the discovery and characterization of links between specific genetic factors and the molecular pathways associated with the disease. To date, most animal models have been based on rare, early-onset Alzheimer’s disease genes that incompletely capture the complexity of LOAD and have not translated well to therapies. Therefore, developing and utilizing animal models based on genes hypothesized to play a role in LOAD will provide new insights into its basic biological mechanisms.  (more…)
Author Interviews, Cocaine, Gastrointestinal Disease, PLoS, Surgical Research, Vanderbilt, Weight Research / 27.07.2018

MedicalResearch.com Interview with: Aurelio Galli, Ph.D. Professor of Molecular Physiology & Biophysics and Psychiatry & Behavioral Science Associate Director for Research Strategy Vanderbilt Brain Institute MedicalResearch.com: What is the background for this study? What are the main findings? Response: The study builds on evidence that bile acids influence the brain’s reward system. Bile acids are normally released from the gall bladder into the upper part of the small intestine, where they emulsify fats for absorption, before being recycled further down the small intestine. In bile diversion surgery, an experimental treatment for weight loss, bile is released at the end of the small intestine, increasing the amount of bile acids that enter the general circulation. Mice treated with this surgery have less appetite for high-fat foods, which suggests that bile acids affect brain reward pathways. We demonstrated that mice receiving the surgery also showed less preference for the cocaine-associated chamber, indicating that cocaine was probably less rewarding. (more…)
Author Interviews, BMJ / 27.07.2018

MedicalResearch.com Interview with: Trudo Lemmens (LicJur, LLM bioethics, DCL) Professor and Scholl Chair in Health Law and Policy Faculty of Law, University of Toronto Toronto Ontario, Canada  MedicalResearch.com: What is the background for this study? Response: The study is part of a series of articles organized in collaboration between the BMJ and the Pan American Health Organization, with particular involvement of members of PAHO’s Advisory Committee on Health Research. The goal of the series is to highlight some of the key accomplishments in the PAHO region in the last decades following PAHO’s 2009 regional policy on research for health, the first WHO regional policy that aimed at contributing to WHOs Stragegy on Research for Health. One of the issues that have been identified in the last decades as an important challenge, particularly for research related to pharmaceutical product and medical device development, is the lack of transparency of clinical trials data. This has been associated with problems of hiding and misrepresentation of research results, duplication of research, and misleading or even outright fraudulent presentations of findings in medical publications. Various remedies have been proposed to address these problems, from trial registration, to results reporting, to broader data sharing that enables independent researchers to verify and challenge research results. I have been involved through PAHO and in my own research in the promotion of transparency measures at the national and international level. PAHO has been playing a leading role in this in the region, so it seemed more than fitting to have an article in the series on what has been accomplished and what challenges remain. (more…)
Author Interviews, CDC, Infections / 26.07.2018

MedicalResearch.com Interview with: Dr. Megin Nichols DVM, MPH, DACVPM Lead , Enteric Zoonoses Activity Division of Foodborne, Waterborne, and Environmental Diseases National Center for Emerging and Zoonotic Infectious Diseases CDC Veterinarian MedicalResearch.com: What is the background for this announcement? Response: Each year, CDC and multiple states investigate several multistate outbreaks of Salmonella infections linked to contact with live poultry in backyard flocks. Seventy outbreaks of Salmonella infections have been linked to contact with poultry in backyard flocks since 2000. (more…)
Author Interviews, Cancer Research, JAMA, Prostate Cancer / 26.07.2018

MedicalResearch.com Interview with: Alison L. Allan, PhD Department of Oncology, Western University London Regional Cancer Program, London Health Sciences Centre London, Ontario, Canada  MedicalResearch.com: What is the background for this study? What are the main findings?  Response: This was an international collaborative study between Lawson Health Research Institute (London, ON), Memorial Sloan Kettering Cancer Center (New York), the Royal Marsden (London, UK) and molecular diagnostics company Epic Sciences (San Diego, CA). The study used a liquid biopsy test developed by Epic Sciences that examines circulating tumour cells (CTCs) in blood samples from patients with advanced prostate cancer who are deciding whether to switch from hormone-targeting therapy to chemotherapy. CTCs are cancer cells that leave a tumour, enter the blood stream and invade other parts of the body, causing the spread of cancer. The test identifies whether or not a patient’s CTCs contain a protein in the nucleus called AR-V7. The research team set out to determine whether the presence of this protein predicted which treatment would best prolong a patient’s life. They found that patients who tested positive for the protein responded best to taxane-based chemotherapy while those who tested negative for the protein responded best to hormone-targeting therapy with drugs called androgen-receptor signaling (ARS) inhibitors. These are the two most widely used drug classes to treat advanced prostate cancer. (more…)
Abbvie, Author Interviews, OBGYNE, Pain Research / 26.07.2018

MedicalResearch.com Interview with: Dr. Dawn Carlson MD MPH Vice President, General Medicine Development AbbVie  MedicalResearch.com: Please provide some background on this announcement. Would you briefly explain what endometriosis is? Whom does it affect and how does it interfere with quality of life? Response: Endometriosis is one of the most common gynecologic disorders in the U.S that affects an estimated one in 10 women of reproductive age. It occurs when tissue similar to the lining of the uterus starts growing outside of the uterus, where it doesn’t belong. The symptoms of endometriosis, including pain with menstrual periods and between periods, and with sexual intercourse, can be debilitating and significantly impact day-to-day activities of women’s lives, personally and professionally. Unfortunately, women with endometriosis can suffer for up to 10 years and visit multiple physicians before receiving a proper diagnosis. Unresolved endometriosis pain results in higher healthcare costs from emergency department visits and repeat surgeries.  (more…)
Author Interviews, Flu - Influenza, Genentech / 26.07.2018

MedicalResearch.com Interview with: Mark Eisner MD MPH Mark D. Eisner, MD, MPH Vice President, Product Development Immunology, Infectious Disease, and Ophthalmology Genentech   MedicalResearch.com: What is the background for this study? Would you briefly explain how baloxavir marboxil differs from other flu treatments?  Response: CAPSTONE-2 is a Phase III multicenter, randomized, double-blind study that evaluated a single dose of baloxavir marboxil compared with placebo and oseltamivir in people 12 years and older who are at a high risk of complications from the flu. The high risk inclusion criteria in CAPSTONE-2 were aligned with the Centers for Disease Control and Prevention (CDC), which defines people at high risk for serious flu complications to include adults 65 years of age or older, or those who have conditions such as asthma, chronic lung disease, diabetes or heart disease. For these people, flu can lead to hospitalization or even death. Participants enrolled in the study were randomly assigned to receive a single dose of 40 mg or 80 mg of baloxavir marboxil (according to body weight), placebo or 75 mg of oseltamivir twice a day for five days. The FDA recently accepted a New Drug Application (NDA) and granted Priority Review to baloxavir marboxil as a single-dose, oral treatment for acute, uncomplicated influenza in people 12 years and older. The NDA was based on results from the Phase III CAPSTONE-1 study of a single dose of baloxavir marboxil compared with placebo or oseltamivir 75 mg, twice daily for five days, in otherwise healthy people with the flu. Results from a placebo-controlled Phase II study in otherwise healthy people with the flu were included as supporting data in the NDA. The FDA is expected to make a decision on approval by December 24, 2018. Baloxavir marboxil is a first-in-class, single-dose investigational oral medicine with a novel proposed mechanism of action designed to target the influenza A and B viruses, including oseltamivir-resistant strains and avian strains (H7N9, H5N1). Unlike other currently available antiviral treatments, baloxavir marboxil is the first in a new class of antivirals designed to inhibit the cap-dependent endonuclease protein within the flu virus, which is essential for viral replication. By inhibiting this protein, baloxavir marboxil prevents viral replication earlier in the flu virus life cycle. (more…)
Author Interviews, Eli Lilly, Lancet, Pharmacology, Rheumatology, UCLA / 26.07.2018

MedicalResearch.com Interview with: Daniel J. Wallace M.D., FACP, MACR Associate Director, Rheumatology Fellowship Program Board of Governors, Cedars-Sinai Medical Center Professor of Medicine, Cedars-Sinai Medical Center David Geffen School of Medicine Center at UCLA In affiliation with Attune Health Beverly Hills, Ca 90211 MedicalResearch.com: What is the background for this study? What are the main findings?   Response: This is the first positive study of systemic lupus erythematosus (SLE) using baricitinib,  a small oral molecule that blocks the JAK system. The human kinome consists of 500 genes and helps regulate cell surface receptor interaction. While agents that inhibit certain pathways are approved for rheumatoid arthritis and certain malignancies, this is the first study of its kind in SLE. (more…)
Author Interviews, Duke, JAMA, Schizophrenia / 26.07.2018

MedicalResearch.com Interview with: Richard Keefe PhD Professor in Psychiatry and Behavioral Sciences Duke Institute for Brains Sciences MedicalResearch.com: What is the background for this study? What are the main findings? Response: A lot of studies have shown that cognitive deficits are present in young people at risk for psychosis. There have been calls for investigations of the idea that cognition declines over time in the young people who are at highest risk, but longitudinal studies are hard to conduct so not much work has been done to address this question. The main finding from our study is that the cognitive architecture – the way the various aspects of cognitive functioning appear to be organized in each individual’s brain based upon their pattern of performance – changes over time in those young people who are in the midst of developing psychosis. Interestingly, cognitive architecture also becomes more disorganized in those whose high-risk symptoms do not remit over a two year period, and is related to the functional difficulties they may be having. The young people whose high risk symptoms were present at the beginning of the study but remitted later actually improved cognitively over the two year period of the study. (more…)
Alzheimer's - Dementia, Author Interviews, Heart Disease, JAMA, Metabolic Syndrome / 26.07.2018

MedicalResearch.com Interview with: Maude Wagner, PhD Student Biostatistics Team Lifelong Exposures, Health and Aging Team Bordeaux Population Health Research Center Inserm Univ. Bordeaux MedicalResearch.com: What is the background for this study? What are the main findings?  Response: Many studies haves shown associations between cardiometabolic health and dementia in midlife, but associations later in life remain inconclusive. This study aimed to model concurrently and to compare the trajectories of major cardiometabolic risk factors in the 14 years before diagnosis among cases of dementia and controls. This study showed that demented persons presented a BMI decline and lower blood pressure (specifically systolic blood pressure) several years before dementia diagnosis that might be a consequence of underlying disease. In contrast, cases presented consistently higher blood glucose levels up to 14 years before dementia suggesting that high glycemia is a strong risk factor for dementia. (more…)
Author Interviews, HIV, JAMA / 25.07.2018

MedicalResearch.com Interview with: Michael S. Saag, MD Professor,Division of Infectious Diseases UAB MedicalResearch.com: What is the background for this study? What are the main findings? 
  • An update of prior recommendations made by the IAS-USA, which have been updated every 2 years since 1996
  • Cover ARVs for prevention and treatment of HIV infection
  • Developed by an international panel of 16 volunteer experts in HIV research and patient care appointed by the IAS–USA
    • Members receive no compensation and do not participate in industry promotional activities while on the panel
  • Primarily for clinicians in highly resourced settings; however, principles are universally applicable
  • Reviewed data published or presented from September 2016 through June 2018
  • Rated on strength of recommendation and quality of evidence
(more…)
AHA Journals, Author Interviews, Gender Differences, Geriatrics, Heart Disease / 25.07.2018

MedicalResearch.com Interview with: Quoc Dinh Nguyen, MD MA MPH Interniste-gériatre – Service de gériatrie Centre hospitalier de l’Université de Montréal – CHUM MedicalResearch.com: What is the background for this study? What are the main findings? Response: Randomized trials are the best evidence basis we have to treat patients. It is known for more than 20 years that older adults and women are disproportionately excluded from randomized trials in cardiology diseases. As the current US population is fast aging, we examined whether this underrepresentation improved or worsened in the last 20 years in the most influential studies published between 1996 and 2015. The main finding is that the women and older adults continue to be underrepresented in cardiology trials. Overall, the mean age was 63 years and the percentage of women was 29%. For coronary heart disease, women comprise 54% of the US population in need of treatment, yet are only 27% of the trial population. For heart failure, the median age of older adults in the US population is 70 years whereas it is only 64 years in the trial population. Our results indicate that the gap has very slowly narrowed in the last 2 decades. However, based on current trends, reaching proportionate enrollment would require between 3 and 9 decades. This persistent lack of representation has significant impacts on the ability of clinicians to provide evidenced based care for these segments of the population. Physicians and other health care professionals are forced to extrapolate study results from younger and male-predominant populations. This is problematic since we know that older adults and women may react differently to medications and to interventions.  (more…)
Alzheimer's - Dementia, Author Interviews, Genetic Research / 25.07.2018

MedicalResearch.com Interview with: Gregory Carter, PhD Associate Professor at The Jackson Laboratory MedicalResearch.com: What is the background for this study? What are the main findings? Response: Late-onset Alzheimer’s disease (LOAD) is the most common form of the disease and the major cause of dementia in the aging population. To date, the complex genetic architecture of LOAD has hampered both our ability to predict disease outcome and to establish research models that effectively replicate human disease pathology. Therefore, most basic research into Alzheimer’s disease has focused on early-onset forms caused by mutations in specific genes, which has provided key biological insights but to date has not translated to effective disease preventatives or cures. Our study analyzes both common and rare human genetic variants to identify those significantly associated with .late-onset Alzheimer’s disease, beginning with a large data set from the Alzheimer’s Disease Sequencing Project. We also analyzed RNA sequencing data from post-mortem human and mouse model samples to prioritize candidate genes. We found a new common coding variant significantly associated with disease, in addition to those in genes previously associated with late-onset Alzheimer’s disease. We also found five candidate genes conferring a significant rare variant burden.  (more…)
Aging, Author Interviews, Dermatology, Environmental Risks, JAMA, Technology / 25.07.2018

MedicalResearch.com Interview with: Startup Screen Dermatology APPDr. med. Titus Brinker Head of App-Development // Clinician Scientist Department of Translational Oncology National Center for Tumor Diseases (NCT) Department of Dermatology University Hospital Heidelberg Heidelberg MedicalResearch.com: What is the background for this study? What are the main findings? Response: ​While everyone in the dermatologic community appears to agree on the importance of UV-protection for skin cancer prevention, busy clinicians often lack time to address it with their patients. Thus, the aim of this study was to make use of waiting rooms that almost every patient visiting a clinic spends time in and address this topic in this setting by the means of modern technology rather than clinicians time. We used our free photoaging app "Sunface" which shows the consequences of bad UV protection vs. good UV protection on the users' own 3D-animated selfie 5 to 25 years in the future and installed it on an iPad. The iPad was then centrally placed into the waiting room of our outpatient clinic on a table and had the Sunface App running permanently. The mirroring of the screen lead to a setting where every patient in the waiting room would see and eventually react to the selfie taken by one individual patient which was altered by the Sunface App. Thus, the intervention was able to reach a large proportion of patients visiting our clinic: 165 (60.7%) of the 272 patients visiting our waiting room in the seven days the intervention was implemented either tried it themselves (119/72,12%) or watched another patient try the app (46/27,9%) even though our outpatient clinic is well organized and patients have to wait less than 20 minutes on average. Longer waiting times should yield more exposure to the intervention. Of the 119 patients who tried the app, 105 (88.2%) indicated that the intervention motivated them to increase their sun protection (74 of 83 men [89.2%]; 31 of 34 women [91.2%]) and to avoid indoor tanning beds (73 men [87.9%]; 31 women [91.2%]) and that the intervention was perceived as fun (83 men [98.8%]; 34 women [97.1%]). (more…)
Author Interviews, Global Health, HIV / 25.07.2018

MedicalResearch.com Interview with: Professor Donna Spiegelman ScD Susan Dwight Bliss Professor of Biostatistics Director, Center for Methods in Implementation and Prevention Science (CMIPS)­­­­­­, Yale School of Public Health Professor, Department of Statistics and Data Science, Yale University Director, Interdisciplinary Methods Core, Center for Interdisciplinary Research on AIDS Yale School of Medicine MedicalResearch.com: What is the background for this study? Response: HIV infections can be transmitted from mothers to their infants during pregnancy, childbirth, and  breastfeeding.  Without access to a package of health services that includes antiretroviral medicines and counseling on best breastfeeding practices, it is estimated that 25% of children born to HIV-positive mothers become infected with HIV.In low-resource settings, 50% of these children die before their second birthday. A 32% increase in under-five mortality between 1988 and 2003 prompted the Kenyan government to establish Prevention of Mother to Child Transmission of HIV (PMTCT) programs in over 10,000 health facilities. This achievement was supported by U.S.President’s Emergency Fund for AIDS Relief (PEPFAR), the which contributed over $248 million to PMTCT programs in Kenya between 2004 and 2014. Although this investments in PMTCT coincided with a remarkable halving of Kenya’s under-five mortality rate, it is unknown whether this improvement can be causally attributed to PEPFAR funding for PMTCT. During the 2000s, child mortality decreased across most of sub-Saharan African countries.  These regional trends, rather than PEPFAR funding, may explain all or part of Kenya’s reduction in over 10,000 in child mortality. To help identify whether PEPFAR’s investments in PMTCT made a causal contribution to this reduction in child mortality, we used statistical methods to assess whether the amount or “dose” of PEPFAR funding provided to different provinces in Kenya was associated with increased HIV testing among pregnant women, which is a critical first step in identifying which women need PMTCT, and reduced infant mortality in Kenya. (more…)