Author Interviews / 13.05.2020

MedicalResearch.com Interview with: Celine Latulipe PhD Associate Professor University of Manitoba MedicalResearch.com: What is the background for this study? What are the main findings? Response: We wanted to find out how many hospitals offer proxy accounts for caregivers of adult patients. Most patient portal systems allow proxy accounts for parents of pediatric patients, so we know the underlying systems support the creation of proxy accounts. When we were starting this research, the two big healthcare systems where I was located did NOT offer such proxy accounts for caregivers of adult patients, and a staff person at one of those hospitals suggested adult patients share their passwords with their caregiver, if the caregiver needed access to the portal. As a computer scientist, I am well aware of the security and privacy risks associated with password sharing, and I was appalled by this advice. So we did this survey across the US and we found that 45% of the staff contacted in our study gave similar password sharing advice. This is hugely problematic. Caregivers using a patient's password means the caregiver can see everything in the medical record, including things the patient might not want the caregiver to know, such as past diagnoses of stigmatized illnesses, substance abuse or reproductive health decisions. Also, because password re-use is common across systems, a caregiver with a patient's portal password may now have access to the patient's online banking. (more…)
Author Interviews, Dermatology / 13.05.2020

MedicalResearch.com Interview with: Brian S. Kim, MD, MTR, FAAD Associate Professor of Medicine (Dermatology) Co-Director, Center for the Study of Itch and Sensory Disorders Division of Dermatology, Department of Medicine Washington University School of Medicine St. Louis, MO  MedicalResearch.com: What is the background for this study? What are the main findings? Response: Itch is the central and most debilitating symptom of atopic dermatitis. However, surprisingly, measuring itch or quality of life in clinical trials is not often a primary endpoint. Therefore, this study focuses in very detailed fashion on how ruxolitinib cream improves pruritus in a clinically meaningful way and its ultimate impact on quality of life. What patients want to know at the end of the day is how much will this drug change my life?  Not, whether it statistically beat out a placebo group. Indeed, what this study shows is that ruxolitinib cream has a major impact on itch in a meaningful way that is also tied to improvements in quality of life. (more…)
AACR, Author Interviews, Boehringer Ingelheim, Cancer Research / 13.05.2020

MedicalResearch.com Interview with: Dr. Udai Banerji, MD The Institute of Cancer Research and The Royal Marsden MedicalResearch.com: What is the background for this study? Response: Not only have I been working in the RAS mutations oncology world for a while, but I also have prior preclinical experience working with VS-6766 (RAF/MEK inhibitor) and defactinib (FAK inhibitor), the two drugs in the Phase 1 study that was presented at the American Association for Cancer Research (AACR) annual medical meeting on April 27th. It is important to know that there is a great significant medical need for novel treatments for KRAS mutant tumors, which are difficult to treat, aggressive, and quite common across advanced solid tumors, including low-grade serous ovarian cancer (LGSOC), non-small cell lung cancer (NSCLC) and colorectal cancer (CRC), resulting in the need for novel treatments in an area of significant medical need. I felt that early signals in preclinical research warranted a clinical trial; so that, combined with my RAS experience, made pursuing the Phase 1 study a clear fit. A clinical trial setting allowed us to explore RAF and RAS inhibitor combinations in multiple tumor trials, which was our aim. The data presented at AACR convey safety and dose response results from the dose-escalation portion and expansion cohorts from an open-label, investigator-initiated Phase 1 study evaluating the combination of VS-6766 (RAF/MEK inhibitor) and defactinib (FAK inhibitor) therapy in patients with LGSOC and KRAS mutant NSCLC. The introductory data described in the study suggest that a novel intermittent dosing schedule of RAF/MEK and FAK inhibitor combination therapy has promising clinical activity in patients with KRAS mutant LGSOC and KRASG12V mutant NSCLC, including patients formerly treated with a MEK inhibitor. Expansion cohorts remain ongoing.  (more…)
Author Interviews, COVID -19 Coronavirus / 12.05.2020

MedicalResearch.com Interview with: Peter Jüni, MD, FESC Director, Applied Health Research Centre Li Ka Shing Knowledge Institute of St Michael's Hospital Department of Medicine University of Toronto, Ontario  MedicalResearch.com: What is the background for this study? Response: It is unclear whether seasonal changes, school closures or other public health interventions will result in a slowdown of the current coronavirus disease 2019 (COVID-19) pandemic. We studied 144 geopolitical areas around the world with more than 375,000 COVID-19 cases by March 27 to determine whether epidemic growth is globally associated with climate or public health interventions intended to reduce transmission of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). (more…)
Author Interviews / 11.05.2020

MedicalResearch.com Interview with: David Spetzler, M.S., Ph.D., M.B.A. President and Chief Scientific Officer Caris Life Sciences MedicalResearch.com: What is the background for this announcement (WES)? Response: The announcement is about the launch of Caris’ latest molecular profiling test, called MI ExomeTM.  MI Exome a next-generation sequencing-based assay analyzing the whole exome of 22,000 DNA genes.  It is unique in that it will be run on every patient and will deliver unprecedented detail into the genomic characteristics of cancers, which will greatly improve our ability to understand cancer and provide insights needed to help physicians and patients make more informed treatment decisions that improve outcomes. (more…)
Author Interviews, Neurological Disorders, Neurology, Pain Research / 11.05.2020

MedicalResearch.com Interview with: Holly Yancy, DO Headache medicine specialist Banner – University Medicine Neuroscience Institute Phoenix, AZ Dr. Yancy comments on the recent Neurology journal article on the potential impact of yoga on migraine.  MedicalResearch.com: What is the background for this study? How might yoga reduce migraine intensity or frequency?  Response: The authors of this trial have studied the benefits of yoga when added to medical management of episodic migraine. They expand on prior, smaller reports of the potential benefit of yoga and mindfulness to migraine patients with a well-designed study that shows yoga, as an adjunct to preventive medication, can lower the intensity, frequency and impact of migraines. Participants even used less abortive medication. The authors propose multiple potential mechanisms of action, including an increase in parasympathetic / decrease in sympathetic nervous system activity, decreased muscle tension, and stress management. (more…)
Author Interviews, COVID -19 Coronavirus, Emergency Care, Infections, NEJM, Stroke / 09.05.2020

MedicalResearch.com Interview with: Akash Kansagra, MD, MS Assistant Professor of Radiology Neurological Surgery, and Neurology Director, Endovascular Surgical Neuroradiology Co-Director, Stroke and Cerebrovascular Center Washington University and Barnes-Jewish Hospital MedicalResearch.com: What is the background for this study? Response: Over the past five years, medicine has made enormous strides in stroke treatment. The effectiveness of these therapies has been absolutely astounding, and our ability to get patients to hospitals that can provide this life-saving care has also improved dramatically. (more…)
AACR, Author Interviews, Biomarkers, Cancer Research, MD Anderson, Pharmaceutical Companies / 09.05.2020

MedicalResearch.com Interview with: David S Hong, M.D MD Anderson Department of Investigational Cancer Therapeutics Division of Cancer Medicine University of Texas MedicalResearch.com: What is the background for this study? Response: Larotrectinib is a first-in-class, CNS active, oral TRK inhibitor exclusively designed to treat tumors with an NTRK gene fusion and does not have secondary targets. In previous presentations and published in The Lancet Oncology, larotrectinib demonstrated robust tumor-agnostic efficacy in an integrated dataset of 159 adult and pediatric patients with TRK fusion cancer across three clinical trials (Feb 2019 data cut-off date). In these studies, the objective response rate (ORR), according to investigator assessment, was 79% (95% confidence interval [CI], 72 – 85%), with a complete response rate of 16%. In this analysis presented at AACR 2020, we sought to evaluate the outcomes in patients from the integrated data set based on different baseline characteristics, including prior lines of therapy and Eastern Cooperative Oncology Group (ECOG) performance status. ECOG measures how the disease impacts a patient. ECOG describes a patient’s level of functioning with a numbering scale (0-5) so physicians can uniformly describe a patient’s ability to care for themselves, daily activity and physical activity (selfcare, walking, working, etc). (more…)
AACR, Author Interviews, Bayer, Cancer Research / 08.05.2020

MedicalResearch.com Interview with: David S Hong, M.D Department of Investigational Cancer Therapeutics Division of Cancer Medicine MD Anderson, University of Texas MedicalResearch.com: What is the background for this study? Response: Larotrectinib is a first-in-class, CNS active, oral TRK inhibitor exclusively designed to treat tumors with an NTRK gene fusion and does not have secondary targets. In previous presentations and published in The Lancet Oncology, larotrectinib demonstrated tumor-agnostic efficacy in an integrated dataset of 159 adult and pediatric patients with TRK fusion cancer across three clinical trials (Feb 2019 data cut-off date). In these studies, the objective response rate (ORR), according to investigator assessment, was 79% (95% confidence interval [CI], 72 – 85%), with a complete response rate of 16%. A variety of NTRK genes have been identified in various tumor types including fusions and non-fusions (e.g., amplifications, rearrangements, deletions, slice variants). In the analysis presented at AACR 2020, we sought to evaluate this pooled data to determine the efficacy of larotrectinib in patients with non-fusion alterations in NTRK genes.  (more…)
Author Interviews, COVID -19 Coronavirus, Infections, Pulmonary Disease / 08.05.2020

MedicalResearch.com Interview with: Jehan Alladina MD Massachusetts General Hospital Pulmonary and Critical Care Medicine Boston, Massachusetts MedicalResearch.com: What is the background for this study? Response: During the pandemic, clinicians around the world have shared anecdotal experiences to help inform care of patients with COVID-19. However, these anecdotes and observations, without careful analysis, can bias clinicians; many clinicians have even recommended experimental therapies based on this information alone. To that end, the goal of our study was to rigorously examine the respiratory failure experienced by critically ill patients with COVID-19 and understand their response to the standards of care for respiratory failure. (more…)
Author Interviews, COVID -19 Coronavirus, Lancet, Rheumatology / 08.05.2020

MedicalResearch.com Interview with: Dr. Giulio Cavalli MD PhD & Prof. Lorenzo Dagna MD FACP Ospedale San Raffaele and Vita-Salute San Raffaele University Milan, Italy     MedicalResearch.com: What is the background for this study? Response: Upon encountering pathogens, our immune system produces pro-inflammatory mediators, called cytokines. Cytokines activate cells from the immune system. In most people, production of cytokines is an appropriate and protective response to infection. However, some individuals develop excessive and detrimental inflammatory responses, which are even more harmful than the pathogen itself to the host organism. We hypothesized that some patients with COVID-19 might develop excessive and detrimental inflammation, and that treatment with anti-inflammatory agents might be beneficial in this population. Anakinra is an inhibitor of the pro-inflammatory molecule interleukin 1 (IL-1). It was originally marketed for the treatment of rheumatoid arthritis, but is now mostly used to treat a variety of pediatric inflammatory diseases. (more…)
Author Interviews, Breast Cancer, Cancer Research, Genetic Research, JAMA / 08.05.2020

MedicalResearch.com Interview with: Anurag K. Singh MD Professor of Oncology Director of Radiation Research Leader, Cell Stress and Biophysical Therapy Program Associate Dean Graduate Medical Education, Research Roswell Park Comprehensive Cancer Center Buffalo NY MedicalResearch.com: What is the background for this study? Response: More than 40% of women with hormone receptor-positive, HER2-negative early stage breast cancer with high recurrence scores (RS) have RS of 26-30. Optimal adjuvant systemic therapy in this subgroup remains unclear, and national guideline currently recommends either chemoendocrine therapy or endocrine therapy alone. In addition, the difference in overall survival of a patient with a RS 26-30 versus RS >30 is unclear.   (more…)
Author Interviews, Colon Cancer, Gastrointestinal Disease, Infections / 08.05.2020

MedicalResearch.com Interview with: Ulrik Stenz Justesen, MD, DMSc Senior consultant at Department of Clinical Microbiology Odense University Hospital Denmark  MedicalResearch.com: What is the background for this study? Response: Anaerobic bacteria are bacteria that do not require oxygen for energy production, and live in various environments including the human gut, where they usually do not cause infections directly. Previous studies have reported an association between bacteria from the Bovis group streptococci, Clostridium septicum and colorectal cancer (CRC). Recently associations between different Bacteroides species., Fusobacterium nucleatum and CRC have also been reported. We aimed to investigate this further in a large-scale study.  (more…)
Author Interviews, COVID -19 Coronavirus / 08.05.2020

MedicalResearch.com Interview with: Anirban Basu, Ph.D. Stergachis Family Endowed Director and Professor The Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute University of Washington, Seattle MedicalResearch.com: What is the background for this study? What are the main findings? Response: The infection fatality ratio (IFR) of Covid19 infections is a key parameter to model the future burden of this pandemic. Case fatality rates at any point in time provide a biased estimate of IFR because of the undercounting in both the reported number of covid deaths (numerator) and the reported number of Covid19 cases (denominator). Instead, this study looked at the temporality or time trend of the CFRs within specific counties in the US (where data were deemed to be mature) to understand the underlying IFRs that these trends allude to. It estimates county-specific IFR to range from 0.5% to 3.6%, with a population average for the US at 1.3% (95% CCI: 0.6% - 2.1%).  (more…)
Author Interviews, Dermatology, Melanoma / 08.05.2020

MedicalResearch.com Interview with: Neelam A. Vashi, MD Associate Professor of Dermatology Director, Boston University Center for Ethnic Skin Director, Cosmetic and Laser Center Boston University School of Medicine and Daniela P.Sanchez BS Boston Medical Center Boston, MA 02118 MedicalResearch.com: What is the background for this study? Response: Although melanoma most commonly affects Caucasians, Hispanics are disproportionately affected by greater morbidity and mortality rates when diagnosed. Poor prognosis in Hispanic patients is likely multifactorial, and may be secondary to lack of knowledge or misconceptions about melanoma risk, atypical presentation, impaired access to care, and language barriers, ultimately resulting in a delay in diagnosis. (more…)
Author Interviews / 07.05.2020

Hemp-derived cannabidiol (CBD), or hemp extract, has been gaining in popularity in recent years. As long as CBD extract is produced using hemp plants, not marijuana, and contains less than 0.3% THC, it's considered legal in all 50 states. Some states enforce restrictions on buyers, but federally, general consumers who want to use CBD are in the clear.

Unfortunately, while the social stigma surrounding hemp extract has largely been eradicated in general populations, the athletic community hasn't fully embraced the benefits of CBD and some competitive fields still have strict regulations in place governing its use. Read on to find out what amateur, college, and professional athletes need to know.

CBD for Amateur Athletes

Amateur athletes don't have to worry about the restrictions put in place by regulatory bodies. They are free to take advantage of CBD's non-psychoactive therapeutic benefits, which include reduced joint pain and inflammation and improved muscle recovery after strenuous workouts. Guardian Athletic has more information about the benefits of cannabidiol for athletes looking to reduce recovery time and improve performance on their website.

Those who plan to engage in collegiate athletics or try out for semi-pro or professional sports teams should be aware that CBD can show up on a blood test for several days after consumption. They should also note that some hemp extracts contain up to 0.3% THC. This extremely low concentration is unlikely to produce a positive test result for marijuana, but it's still better to buy CBD from a reputable supplier that uses lab testing to ensure 0% THC concentrations.

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Asthma, AstraZeneca, Author Interviews / 06.05.2020

MedicalResearch.com Interview with: Ubaldo Martin MD VP Clinical Respiratory RIA Late Stage Development AstraZeneca MedicalResearch.com: What is the background for this study? Response: BORA was an extension study evaluating the long-term safety and specific aspects of efficacy in patients who had previously been in the benralizumab pivotal studies.  After the patients completed the pivotal studies (Calima, Sirocco and Zonda), they were eligible to join BORA which followed adults for an additional year and adolescent for an additional 2 years.  All patients receive one of two dosages of benralizumab.  The abstract reports the outcomes of adolescents in the BORA study who were followed for approximately 3 years in total. (more…)
Author Interviews, Gastrointestinal Disease / 06.05.2020

MedicalResearch.com Interview with: Gregory S. Sayuk, MD, MPH John T. Milliken Department of Medicine Division of Gastroenterology Associate Professor of Medicine and Psychiatry Associate Director, Fellowship Training Program Washington University School of Medicine MedicalResearch.com: What is the background for this study? Response: The primary objective of this analysis, “Plecanatide for Patients with Chronic Idiopathic Constipation and Irritable Bowel Syndrome‒Constipation: Analysis of Abdominal Pain from Four Randomized Phase 3 Clinical Trials,” was to determine the impact of plecanatide on abdominal pain in patients with chronic idiopathic constipation (CIC) and irritable bowel syndrome with constipation (IBS-C), in addition to comparing efficacy results in patient populations with minimal to mild pain versus moderate to severe pain at baseline. This was a post hoc analysis of data from the four large, phase 3 plecanatide pivotal trials in CIC and IBS-C. (more…)
Author Interviews, Brigham & Women's - Harvard, Nature, Weight Research / 05.05.2020

MedicalResearch.com Interview with: Ronald Kahn, MD Chief Academic Officer, Joslin Diabetes Center Mary K. Iacocca Professor of Medicine Harvard Medical School MedicalResearch.com: What is the background for this study? What are the main findings? Response: Adipose tissue is a heterogeneous organ and composed of several cell types, including mature adipocytes, preadipocytes, stem cells, endothelial cells, and various blood cells. Different adipose depots have distinct physiological functions associated with their anatomical location and cell composition. For example, accumulation of intra-abdominal (visceral) white adipose tissue is associated with insulin resistance and metabolic syndrome, whereas accumulation of subcutaneous adipose tissue is not metabolically detrimental and may be even associated with increased insulin sensitivity. Determining the mechanisms for these phenotypic differences could lead to development of novel therapies for diabetes, obesity, and their associated morbidities. A central challenging question in research of metabolic disease is whether disease risk for diabetes and metabolic syndrome is driven by a subset of fat cells that may interact with environmental stresses in disease pathogenesis in a way different from other fat cells. Indeed, previous studies from the Kahn lab have shown different fat cells in a single depot from the mouse may exhibit developmental heterogeneity. In this new study, we attempted to address this question for human white fat using a synergistic application of several methodologies: 1) single cell transcriptional profiling of human white fat during differentiation, 2) analysis of individual clones of white fat cells taken from humans at surgery, 3) novel computer based network analysis and 4) integration of the gene signatures across experimental models. Single-cell RNA sequencing is an ideal technique to profile gene expression of heterogeneous cell populations obtained from a single tissue, including fat tissue. (more…)
Author Interviews, JAMA, Pediatrics, Tobacco Research, USPSTF / 05.05.2020

MedicalResearch.com Interview with: Dr. Michael Silverstein M.D., M.P.H Professor of Pediatrics Director of the Division of General Academic Pediatrics Vice Chair of Research, Department of Pediatrics Boston University School of Medicine MedicalResearch.com: What is the background for this study? Response: The number of children and teens who use tobacco products continues to be a major issue in the U.S., driven largely by an increase in e-cigarette use, which makes preventing tobacco use among young people critical to the health of our nation. To help prevent kids and teens from starting to use tobacco, the Task Force recommends clinicians provide behavioral interventions, such as education or brief counseling.   (more…)
Author Interviews, Cancer Research, JAMA / 04.05.2020

MedicalResearch.com Interview with: Emerson Y. Chen, MD Assistant Professor of Medicine, Medical Oncology Knight Cancer Institute, Oregon Health & Science University Portland, OR 97239 MedicalResearch.com: What is the background for this study? Response: Our research group had previously studied how oncology drugs are approved in these two previous papers listed below. One is focused on the time delay trade-off from surrogate endpoints (i.e. response rate and progression-free survival) over definite endpoints (i.e. overall survival and quality of life). The other is focused on how promising the response rate of a drug candidate have to be to be considered for oncology drug approval.  (more…)
Author Interviews, Endocrinology, Weight Research / 04.05.2020

MedicalResearch.com Interview with: Florian Keifer, M.D., Ph.D Associate Professor of Medicine Division of Endocrinology and Metabolism Department of Medicine III Medical University of Vienna MedicalResearch.com: What is the background for this study? Response: Brown fat, in contrast to white fat, burns significant amounts of chemical energy through heat production. In numerous animal models the activation of brown adipose tissue (BAT) increases energy expenditure and counteracts weight gain. Therefore BAT has been established as a promising target in the fight against obesity and related metabolic disorders. In humans, BAT can be activated by moderate cold exposure, however the function and relevance of BAT are incompletely understood. Using PET scans we identified two groups of individuals those with and without active BAT and studied their differences in energy expenditure and blood fatty acid composition.  (more…)
Author Interviews, Cancer Research, Genetic Research / 04.05.2020

MedicalResearch.com Interview with: Rebecca Nagy Vice President Medical Affairs Guardant Health  MedicalResearch.com: What is the background for this study? Response: Hormone receptor positive (HR+) breast cancer comprises roughly 75% of all cancers of the  breast. While many of these cancers can be cured through multi-modality therapy, there remain many deaths due to metastatic spread to distant organs. These metastatic cancers are marked by their resilience in the face of potent targeted therapies and chemotherapies, with many tumors displaying an initial drug response followed by resistance. Recently, genomic sequencing has identified recurrent, oncogenic alterations in HR+ metastatic breast cancer (MBC) with mutations in the catalytic alpha subunit of PI3K (PI3Kα, PIK3CA gene), in over 40% of cases. This has raised hopes for more durable disease control through precise inhibition of this driver oncogene. The SOLAR-1 Phase III study of alpelisib combined with fulvestrant in PIK3CA-mutated HR+ MBC showed a markedly improved PFS over fulvestrant monotherapy but pervasive resistance nonetheless. To characterize the basis for such resistance to combination hormone plus PIK3CA targeted therapy, we conducted a detailed, longitudinal analysis of tumor and plasma circulating cell-freetumor DNA (ctDNA) among patients with HR+ MBC who participated in a phase I/II dose escalation study of alpelisib in combination with letrozole or exemestane.  (more…)
Allergies, Asthma, AstraZeneca, Author Interviews / 01.05.2020

MedicalResearch.com Interview with: astrazenecaOlga Ryan, DrPH, MPH, MBA Regional Clinical Account Director, Southwest AstraZeneca  MedicalResearch.com: What is the background for this study? What are the main findings? Response: Asthma is common and imparts a substantial societal burden. It is well documented that asthma prevalence varies between males and females. Before puberty, more boys have asthma.  Following puberty, a greater proportion of women suffer with asthma. We also have observed that women experience greater morbidity from the illness, greater healthcare resource utilization and suboptimal response for guideline recommended therapies (ICS, ICS/LABA). Rationale for this study focused on describing asthma related outcomes between a well characterized severe asthma cohort, with intent in delineating differences among the sexes. With the advent of targeted biological medicines for severe asthma, as well as apparent gaps in knowledge, we wanted to understand potential sex-specific disease indicators in a well characterized severe asthma cohort. (more…)
Asthma, AstraZeneca, Author Interviews, Cost of Health Care / 01.05.2020

MedicalResearch.com Interview with: Yen Chung, PharmD Payer Evidence Director US Medical Affairs, AstraZeneca MedicalResearch.com: What is the background for this study? Response: Among patients with persistent asthma, use of systemic corticosteroids (SCS) is typically reserved for treatment of asthma exacerbations and as a supplemental maintenance therapy for patients whose disease remains uncontrolled with maximum maintenance controller therapies. However, SCS therapy comes with known risks for acute and chronic complications. It is well established that patients with severe asthma are responsible for a disproportionate amount of the economic burden of asthma;  however, less clear is the extent to which systemic corticosteroids use and its consequences specifically contributes to the cost burden of asthma. The purpose of this study was to use administrative claims to follow asthma patients with and without SCS treatment for up to 3 years and compare their complication rates, health care resource utilization, and costs.  (more…)
Author Interviews, Beth Israel Deaconess, COVID -19 Coronavirus, JAMA, Race/Ethnic Diversity / 01.05.2020

MedicalResearch.com Interview with: Rishi Wadhera, MD, MPP, MPhil Instructor in Medicine at Harvard Medical School Cardiologist,Beth Israel Deaconess Medical Center MedicalResearch.com: What is the background for this study? Response: With more than a million confirmed cases of COVID-19 in the United States, there is growing concern that low-income communities and racial/ethnic minorities may be disproportionately shouldering the burden of the pandemic. New York City, which is comprised of 5 boroughs (Bronx, Brooklyn, Manhattan, Queens, and Staten Island) with unique demographic and socioeconomic characteristics, has emerged as the epicenter of the COVID-19 outbreak. (more…)
Author Interviews, Biogen, Neurological Disorders, Pharmaceutical Companies / 30.04.2020

MedicalResearch.com Interview with: Toby Ferguson, M.D., Ph.D. Vice President, Head Neuromuscular Development Unit Biogen MedicalResearch.com: What is the background for this study? Would you briefly explain what is meant by SMA, who is primarily affected and incidence? Response: Spinal muscular atrophy (SMA) is a rare, genetic, neuromuscular disease characterized by a loss of motor neurons in the spinal cord and lower brain stem that can result in severe, progressive muscle atrophy and weakness. Approximately one in 10,000 live births have a diagnosis of SMA. It is a leading genetic cause of infant mortality; however, people of all ages are impacted by the disease. More than three years ago, SPINRAZA (nusinersen) became the first FDA-approved treatment option for SMA. The DEVOTE study, which recently treated its first patient, is designed to evaluate the safety and potential for even greater efficacy of SPINRAZA when administered at a higher dose than currently approved for the treatment of SMA. The Phase 2/3 randomized, controlled, dose-escalating study will be conducted at approximately 50 sites around the world and aims to enroll individuals of all ages with SMA. (more…)
Author Interviews, Gastrointestinal Disease, Hepatitis - Liver Disease, Occupational Health, Outcomes & Safety / 30.04.2020

MedicalResearch.com Interview with: Mishal Reja, MD,  MD Resident in Internal Medicine Robert Wood Johnson University Hospital MedicalResearch.com: What is the background for this study? Response: World Trade Center first responders were exposed to environmental toxicants that have resulted in negative health consequences. Gastrointestinal aerodigestive disorders such as GERD and Barrett’s esophagus have been frequently reported in this population. Additionally, an increasing body of literature has shown that fatty liver disease is not only secondary to metabolic syndrome, diabetes, and obesity, as previously thought, but can also result from environmental and industrial toxicants. Chemicals such as Vinyl Chloride, Tetrachloroethylene, Perchloroethylene, and many others are frequently found in industrial occupations and have resulted in fatty liver disease, steatohepatitis, cirrhosis, and hepatocellular carcinoma. Many of these chemicals were also present at ground zero, thus exposing many of first responders to the hepatotoxic effects of these chemicals. To date this is the first study to look at liver disease in World Trade Center first responders. (more…)
Author Interviews, C. difficile, Gastrointestinal Disease, Imperial College, Infections, Transplantation / 30.04.2020

MedicalResearch.com Interview with: Prof. Julian Marchesi PhD Professor of Digestive Health Faculty of Medicine, Department of Metabolism, Digestion and Reproduction   Dr. Benjamin Mullish PhD Faculty of Medicine, Department of Metabolism, Digestion and Reproduction NIHR Clinical Lecturer Imperial College London       MedicalResearch.com: What is the background for this study? Response: Many patients are colonized with bacteria that are resistant to nearly all the antibiotics that we currently have. This antibiotic resistance is a huge public health problem, not least because it may lead to the scenario where a bacterial species moves from the gut and into the bloodstream, causes an infection, and cannot be treated. Such scenarios particularly occur in patients who are particularly prone to getting multiple and frequent courses of antibiotics; this may include patients with particular kidney conditions (who may be vulnerable to recurrent urinary tract infections (UTIs)), and patients with blood cancers (such as leukaemia, who have weak immune systems and are therefore prone to infections). Furthermore, in both sets of patients, to help treat their disease, they may be offered transplants, either a new kidney or new bone marrow. When this transplant happens, the clinician needs to ‘switch off’ their immune system to allow the transplant to work. When the immune system is dialled down, it can no longer stop any invading bacteria, increasing the chance of antibiotic resistance bacteria causing infections, which frequently leads to patient death.  (more…)