Author Interviews, Neurological Disorders, Personalized Medicine / 19.06.2020

MedicalResearch.com Interview with: https://polyneuron.com/Pascal Hänggi, PhD Chief Scientific Officer Polyneuron Pharmaceuticals   MedicalResearch.com: What is the background for this study? Response: Anti-MAG neuropathy is a rare form of acquired demyelinating neuropathy. The disease onset normally presents after the age of 50 years and is 2.7 times more frequent in men than in women, with a prevalence of about 1 in 100,000. It is caused by the production of monoclonal anti-MAG IgM antibodies that recognize the HNK-1 epitope. The myelin-associated glycoprotein MAG is a mediator for the formation and maintenance of the myelin sheaths. There is strong evidence that the binding and deposition of anti-MAG IgM autoantibodies on myelin sheath is responsible for the demyelination, which clinically manifests itself as a peripheral neuropathy affecting primarily sensory nerves. However, the causes and the exact mechanisms behind the expansion of anti-MAG IgM producing B-cell and plasma cell clones are not fully understood. Most off-label treatments aim to reduce pathogenic autoantibody titers by depleting  autoantibody-producing B cell clones which interfere with antibody-effector mechanisms, or physically remove autoantibodies from the circulation. Most frequently, the anti-CD20 monoclonal antibody rituximab is used to treat anti-MAG neuropathy patients. However, all of these treatment options often lack of selectivity, efficiency, or can induce severe adverse effects in some patients. Polyneuron has designed PN-1007 to highly selectively target the IgM autoantibodies that cause anti-MAG neuropathy. PN-1007 is a glycopolymer that mimics the natural HNK-1 carbohydrate epitope found on myelin of peripheral nerves and binds to the circulating disease-causing antibodies. By eliminating these pathogenic antibodies, PN-1007 may protect the integrity of the neuronal myelin sheaths of anti-MAG neuropathy patients. (more…)
Author Interviews, Ophthalmology, Technology / 18.06.2020

MedicalResearch.com Interview with: Prof. FanProf. FAN Zhiyong PhD University of California, Irvine HKUST School of Engineering MedicalResearch.com: What is the background for this study? What are the main findings? Response: According to the report of The World Health Organization, there are over 252 million people suffering from visual impairment globally and 15 million of them are difficult to cure by conventional medical methods. However, today, even the best bionic eyes have only 200 clinical trials, less than 1 ppm of all the patients, mainly due to their poor performance and high cost. The huge gap in supply and demand triggers the study of bionic eyes with performance comparable to human eyes. One important reason for their poor performance is the mismatch in shape between the flat bionic eyes and concave sclera. To protect the soft tissue in eyes from being damaged by the bionic surface, the implanted bionic eyes have to be small. This has limited the sensing area and further the electrodes number, and finally yielded poor image sensing characters with low resolution and narrow field-of-view. In this work, we are trying to achieve high performance image sensing by biomimeticing human eyes. The high-density NWs are well aligned and embedded in a hemispherical template to serve as retina. The conformal attachment of bionic eyes with sclera enables the large sensing area and wide visual angle. In addition, each individual high-density nanowires can potentially work as an individual pixel. By addressing these challenges, our device design has huge potential to improve the image sensing performance of bionic eyes. (more…)
Author Interviews, Dermatology, Melanoma, Technology / 17.06.2020

MedicalResearch.com Interview with: Chi Hwan Lee PhD Assistant Professor of Biomedical Engineering and Mechanical Engineering, and by Courtesy, of Materials Engineering, and Speech, Language, & Hearing Sciences Purdue University  MedicalResearch.com: What is the background for this study? Response: Conventional melanoma therapiesincluding chemotherapy and radiotherapy, suffer from the toxicity and side effects of repeated treatments due to the aggressive and recurrent nature of melanoma cells. Less-invasive topical chemotherapies by utilizing miniaturized polymeric microneedles are emerged as an alternative, but the sustained, long-lasting release of drug cargos remains challenged due to the rapidly dissolving behavior of polymers (typically, within 15 min-2 hrs). In addition, the size of the microneedles is still large for small, curvilinear and sensitive areas of tissues such as cornea (for ocular melanoma). (more…)
Author Interviews, Diabetes, JAMA / 17.06.2020

MedicalResearch.com Interview with: Richard E. Pratley, MD AdventHealth Samuel E. Crockett Chair in Diabetes Research Medical Director | AdventHealth Diabetes Institute Senior Investigator and Diabetes Program Lead AdventHealth Translational Research Institute  MedicalResearch.com: What is the background for this study? Response: Historically, older individuals with T1D have been underrepresented or excluded from clinical trials. Older individuals with T1D are at particularly high risk for hypoglycemia. Because of their long duration of diabetes, they often have impaired counterregulatory responses and hypoglycemia unawareness. Hypoglycemia in older individuals is particularly dangerous because it can lead to falls and fractures, cognitive impairment and cardiovascular events, including death. There has been the misperception that older individuals are less able to use technology to manage their diabetes.  This study dispelled that notion.   (more…)
Abbvie, Author Interviews, OBGYNE / 16.06.2020

MedicalResearch.com Interview with: https://www.abbvie.com/   Ayman Al-Hendy, M.D., Ph.D. Investigator for the ELARIS UF-2 clinical trials Professor of Gynecology Director of Translational Research University of Illinois at Chicago   Dr. Al-Hendy discusses the recent announcement that the FDA has approved  ORIAHNN™ for the management of heavy menstrual bleeding due to uterine fibroids in pre-menopausal women. MedicalResearch.com: What is the background for this approval? Uterine fibroids, commonly referred to as uterine leiomyomas, are the most common type of non-cancerous tumor known to impact women of reproductive age (30-50 years old). In fact, studies show that uterine fibroids can occur in up to 70 percent of European American women and over 80 percent of African American women by age 50. As a result of uterine fibroids, women can experience a range of symptoms, the most common being heavy menstrual bleeding (i.e. prolonged and/or frequent bleeding), which can lead to other health effects such as anemia, fatigue, pelvic pain, urinary frequency etc. Uterine fibroid treatment recommendations have historically been based on the size and location of the fibroid(s). When treating larger and more complicated fibroids, healthcare providers have typically believed that surgery is their best course of action, which has made uterine fibroids the leading reason for the hysterectomies performed in the U.S. The FDA approval of ORIAHNN was based on improving care for uterine fibroid sufferers who have had a negative impact on their quality of life due to disruptive symptoms. What makes the approval of ORIAHNN so exciting, is that women now have an oral therapy to directly address heavy menstrual bleeding due to uterine fibroids.  (more…)
Author Interviews, Cancer Research, Leukemia / 16.06.2020

MedicalResearch.com Interview with: Courtney D. DiNardo, M.D., MSCE Department of Leukemia, Division of Cancer Medicine The University of Texas MD Anderson Cancer Center MedicalResearch.com: What is the background for this study? Response: At this year’s virtual European Hematology Association (EHA) meeting, we are presenting late-breaking data from the Phase 3 VIALE-A trial. The randomized double-blind, placebo-controlled trial evaluated venetoclax in combination with azacitidine in previously-untreated patients with acute myeloid leukemia (AML) who are ineligible for standard induction therapy compared to azacitidine plus placebo.  (more…)
Author Interviews, JAMA, Multiple Sclerosis, Neurology / 15.06.2020

MedicalResearch.com Interview with: Cris S Constantinescu,  MD, PhD, FRCP Professor, Division of Clinical Neuroscience Research Group in Clinical Neurology University of Nottingham Queen's Medical Centre Nottingham UK MedicalResearch.com: What is the background for this study? Response: The study is in some way a test of the hygiene or old friends hypothesis, whereby eradication, through improved hygiene, of some parasites that have existed in the human gut for thousands of years and have suppressed inflammatory reactions, leads to an increase in inflammatory conditions. This has been used to explain the increased autoimmune and inflammatory diseases in the developed world. Healthy volunteer studies at the University of Nottingham showed therapeutic hookworm infection to be safe and well tolerated up to about 50 larvae, and then safety studies in people with airway hyperreactivity and inflammatory bowel disease raised no concern. Following a study in Argentina showing that people with MS have milder disease when they have a natural co-existing asymptomatic infection with intestinal parasites, we (Professor Pritchard, immunoparasitologist and myself) decided to test hookworm in MS, and for the first time used 25 larvae in a patient study. (more…)
Author Interviews, Flu - Influenza, Pediatrics, Pediatrics, Vaccine Studies / 15.06.2020

MedicalResearch.com Interview with: Allison Kempe, MD, MPH Ergen Family Endowed Chair in Pediatric Outcomes Research Professor of Pediatrics, University of Colorado School of Medicine Director of ACCORDS (Adult and Child Consortium for Health Outcomes Research and Delivery Science) University of Colorado School of Medicine | Children’s Hospital Colorado  MedicalResearch.com: What is the background for this study? Response: In 2019 the WHO designated vaccine hesitancy as one of the ten leading threats to global health. Although studies have assessed parental vaccine hesitancy in different localities and estimated vaccine refusals nationally, there is little recent US national data on the prevalence of hesitancy about routine childhood vaccines and national hesitancy rates for influenza vaccine have never been assessed. We used a hesitancy scale developed by the WHO to estimate levels of parental hesitancy for both routine childhood and childhood influenza vaccination  (more…)
Author Interviews, Diabetes, Duke, Telemedicine / 15.06.2020

MedicalResearch.com Interview with: Matthew J. Crowley, MD Core Investigator, Durham Center of Innovation to Accelerate Discovery and Practice Transformation (ADAPT) Affiliated Investigator, VA Office of Rural Health Staff Physician, Endocrinology Section, Durham VA Health Care System Elizabeth Kobe, BS Medical Student Durham VA Health Care System Duke University School of Medicine MedicalResearch.com: What is the background for this study? With whom were the telehealth sessions performed? (MDs, PAs, Dieticians etc). Response: Diabetes management in rural populations is especially challenging due to limited access to specialty care and self-management programs. Telehealth is a potential strategy for extending high-quality diabetes care to rural areas. The Veterans Health Administration (VHA) has a robust Home Telehealth (HT) system that is currently used for telemonitoring patient blood glucose values. In order to address the challenges of managing diabetes in rural areas in a clinically feasible manner, we strategically designed an intensive diabetes management intervention – Advanced Comprehensive Diabetes Care (ACDC) – for delivery using existing VHA HT infrastructure and clinical staffing. ACDC is a 6-month telehealth intervention that combines telemonitoring with module-based self-management support and medication management. ACDC is delivered entirely by existing clinical staff (a clinical HT nurse and a medication manager (typically a PharmD)) through bimonthly, 30-minute calls. Our initial randomized controlled trial found that ACDC improved HbA1c by a clinically and statistically significant  -1.0% relative to usual care at 6 months, while also improving blood pressure and diabetes self-care. Our goal with the present work was to improve diabetes care in clinical practice for rural Veterans whose type 2 diabetes remained uncontrolled despite receiving available services. To this end, we partnered with the VA Office of Rural Health to implement ACDC into VA sites across the country with large rural populations.  (more…)
Author Interviews / 15.06.2020

MedicalResearch.com Interview with: Nicholas J. Vogelzang, MD, FASCO, FACP Medical Oncologist at Comprehensive Cancer Centers of Nevada Associate Chair, US Oncology Research Genitourinary Committee  MedicalResearch.com: What is the background for this study? Response: According to the American Cancer Society, prostate cancer is the second leading cause of cancer death in men in the U.S. New approaches are needed to target the androgen receptor (AR), a critical driver of metastatic castration resistant prostate cancer (mCRPC). Current agents work by decreasing androgen levels (abiraterone) or blocking androgen binding to AR (enzalutamide). Despite rapid and dramatic responses to standards of care, all patients with metastatic disease progress to the castration resistant state and their tumors continue to be dependent on the AR signaling axis.1 Study design:
  • “3 + 3” dose escalation; starting dose = 35 mg, orally, once daily with food
  • Dose increases dependent on toxicities
    • Range 25% to 100% based on severity of AEs
Inclusion criteria:
  • Men with mCRPC, regardless of AR status
  • At least two prior systemic therapies, at least one of which was abiraterone or enzalutamide
  • Disease progression on most recent therapy
    • Rising PSA or 2+ new lesions upon bone scan
Endpoints:
  • Primary:
    • Define the maximum tolerated dose and recommended phase 2 dose
  • Secondary:
    • Pharmacokinetics
    • Anti‐tumor activity (PSA50, RECIST criteria)
  • Exploratory:
    • Biomarkers
      • ctDNA mutational profiling
      • AR levels in optional paired biopsies
      • AR and AR‐ V7 levels in circulating tumor cells (CTCs) 
(more…)
Author Interviews, Dermatology, Pediatrics, Pharmaceutical Companies, Regeneron / 15.06.2020

MedicalResearch.com Interview with: Amy S Paller, MD Chair, Department of Dermatology Director, Skin Biology and Diseases Resource-Based Center Walter J. Hamlin Professor of Dermatology Professor of Dermatology and Pediatrics (Dermatology) Feinberg School of Medicine Northwestern University  Dr. Paller discusses the FDA approval of Dupixent® (dupilumab) for children aged 6 to 11 years with moderate-to-severe atopic dermatitis (eczema), whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable.  MedicalResearch.com: What is the background for this announcement? Would you briefly discuss what is meant by atopic dermatitis and how it affects children? Response: “Atopic dermatitis, the most common form of eczema, is a chronic inflammatory disease that often appears as a rash on the skin. Moderate-to-severe atopic dermatitis is characterized by rashes that can potentially cover much of the body and can include intense, persistent itching, skin lesions and skin dryness, cracking, redness or darkness, crusting and oozing. Itch is one of the most burdensome symptoms for patients and can be debilitating. This recent FDA approval expands the use of Dupilumab in the U.S. to include children aged 6 to 11 years with uncontrolled moderate-to-severe atopic dermatitis, making it the only biologic medicine approved for this use in this population. Dupilumab is also approved in the U.S. to treat patients aged 12 years and older with moderate-to-severe atopic dermatitis. Moderate-to-severe atopic dermatitis can place a particularly substantial burden on young children aged 6 to 11 years and their families. Limited treatment options leave many of these children to cope with intense, unrelenting itch and skin lesions. Families of these children can spend countless hours helping them to manage their disease.” (more…)
Author Interviews, Circadian Rhythm, COVID -19 Coronavirus, Sleep Disorders / 15.06.2020

MedicalResearch.com Interview with: Dr. Christine Blume PhD Centre for Chronobiology Psychiatric Hospital of the University of Basel Transfaculty Research Platform Molecular and Cognitive Neurosciences Basel MedicalResearch.com: What is the background for this study? Response: In modern societies, human rest-activity rhythms and sleep are between the often-conflicting poles of external social time (e.g., work hours and leisure activities) and an individual’s internal biological time. This can lead to so-called “social jetlag”, which has repeatedly been associated with detrimental health effects. With the restrictions to control the pandemic, social timing relaxed as people many started working from home and public life came to a standstill. In an online survey with 435 respondents, we investigated the effects of the phase with the strictest COVID-19 restrictions on the relationship between social and biological rhythms as well as sleep during a six-week period (mid-March until end of April 2020) in three European societies (Austria, Germany, Switzerland). (more…)
Author Interviews, Gout, Hepatitis - Liver Disease, Rheumatology / 15.06.2020

MedicalResearch.com Interview with: Brian LaMoreaux, M.D., M.S. Medical Director, Medical Affairs Horizon Therapeutics   MedicalResearch.com: What is the background for this study? Response: Hyperuricemia is associated with non-alcoholic fatty liver disease (NAFLD) but the relationship to fibrosis remains uncertain. Moreover, it is not known whether lowering serum urate will affect the course of NAFLD.   (more…)
Author Interviews, Gout, Rheumatology / 15.06.2020

MedicalResearch.com Interview with: Brian LaMoreaux, M.D., M.S. Medical Director, Medical Affairs Horizon Therapeutics  MedicalResearch.com: What is the background for this study? Response: Pegloticase is a PEGylated biologic therapy for patients with uncontrolled gout who have not improved on or could not tolerate conventional urate-lowering therapies. All biologics have the ability to engender anti-drug antibodies (ADAs) and it is known that some patients given pegloticase develop ADAs that cause them to stop treatment prior to receiving a complete course of therapy. In other rheumatic autoimmune diseases, DMARDs such as methotrexate or azathioprine are used as standard of care to prevent the development of ADAs to biologics. These DMARDs often allow patients to remain on biologic therapies longer and receive the full therapeutic benefits while minimizing adverse events. While pegloticase has been used traditionally as monotherapy, recent case series have demonstrated the therapeutic benefit of immunomodulator co-administration, allowing more patients to receive a full course of pegloticase therapy. Little has been published on how widespread this practice is and whether it has changed over time. (more…)
Author Interviews, Gout, Heart Disease / 14.06.2020

MedicalResearch.com Interview with: Brian LaMoreaux, M.D., M.S. Medical Director, Medical Affairs Horizon Therapeutics MedicalResearch.com: What is the background for this study? Response: This is an independent study by Dr. Gurkipal Singh for which Horizon provided support and funding. Heart failure is the eighth leading cause of death in the US, with a 38% increase in the number of deaths due to HF from 2011 to 2017. Dr. Gurkirpal Singh conducted this analysis on heart failure hospitalization rates in patients with gout in the US to estimate their clinical and economic impact. Gout and hyperuricemia have previously been recognized as significant risk factors for heart failure, but there is little nationwide data on the clinical and economic consequences of these comorbidities. (more…)
Author Interviews, Diabetes, Gout / 14.06.2020

MedicalResearch.com Interview with: Brian LaMoreaux, M.D., M.S. Medical Director, Medical Affairs Horizon Therapeutics MedicalResearch.com: What is the background for this study? Response: People with diabetes are known to have an increased risk of undergoing amputation procedures, however it was not known if patients with gout have an elevated independent risk for digit or limb amputations, or whether gout potentiates amputation rates in patients with diabetes. This analysis assessed and compared the rate of amputation procedures conducted in patients with gout, diabetes, both gout and diabetes, and neither gout nor diabetes via examining records from a large US claims database. (more…)
Author Interviews, Gout, Rheumatology / 14.06.2020

MedicalResearch.com Interview with: https://www.horizontherapeutics.com/Brian LaMoreaux, M.D., M.S. Medical Director, Medical Affairs Horizon Therapeutics    MedicalResearch.com: What is the background for this study? Response: Pegloticase is an infused biologic approved to treat uncontrolled gout. The drug is highly effective, but patients can develop anti-drug antibodies that may accelerate clearance of pegloticase from the circulation. Randomized clinical trials have shown that 42% of patients treated with bi-weekly pegloticase had a serum uric acid (sUA) below 6.0 mg/dl at 3 and 6 months. Mild-to-moderate immunomodulation has been shown to lower the prevalence of anti-drug antibody formation in patients with other autoimmune diseases (rheumatoid arthritis, Crohn’s disease, juvenile idiopathic arthritis). Case reports and case series in the literature suggest that low to-moderate doses of methotrexate or azathioprine may also decrease anti-drug antibody formation in uncontrolled gout patients treated with pegloticase.   (more…)
ASCO, AstraZeneca, Author Interviews, Cancer Research, Melanoma / 13.06.2020

MedicalResearch.com Interview with: Yuanbin Chen, MD, PhD Cancer & Hematology Centers of Western Michigan MedicalResearch.com: What is the background for this study? What are the main findings?
    • Response: The CASPIAN trial was a randomized, open-label, multi-center global Phase III trial in the first-line treatment of patients with extensive-stage small cell lung cancer (ES-SCLC). The trial compared IMFINZI in combination with etoposide and either carboplatin or cisplatin chemotherapy, or IMFINZI and chemotherapy with the addition of a second immunotherapy, tremelimumab, versus chemotherapy alone – the primary endpoint being overall survival (OS). After a median follow up of more than two years, the latest results for IMFINZI plus chemotherapy demonstrate a sustained and clinically meaningful OS benefit for patients with extensive-stage small cell lung cancer (ES-SCLC), maintaining a 25% reduction in the risk of death versus chemotherapy alone. Updated median OS was 12.9 months versus 10.5 for chemotherapy.
      • In a post-hoc analysis, 22.2% of patients treated with IMFINZI plus chemotherapy remained alive after 24 months, versus 14.4%, for chemotherapy alone.
      • Post-hoc analysis also showed that for IMFINZI plus chemotherapy, 11.0% of patients were alive and progression-free at 24 months versus 2.9% for chemotherapy alone.
      • IMFINZI plus chemotherapy maintained a high confirmed objective response rate (ORR) (68% versus 58%) and in a post-hoc analysis, duration of response (DoR) for IMFINZI at 24 months was 13.5% versus 3.9% for chemotherapy alone.
      • At 24 months, 12% of patients in the IMFINZI plus chemotherapy arm remained on IMFINZI treatment.]
(more…)
ASCO, AstraZeneca, Author Interviews, Cancer Research, Lung Cancer, Yale / 13.06.2020

MedicalResearch.com Interview with: Roy S. Herbst, MD, PhD Ensign Professor of Medicine (Medical Oncology) Professor of Pharmacology Chief of Medical Oncology, Yale Cancer Center and Smilow Cancer Hospital; Associate Cancer Center Director for Translational Research Yale Cancer Center MedicalResearch.com: What is the background for this study? What are the main findings?
  • ADAURA is the first global trial for an EGFR tyrosine kinase inhibitor to show statistically significant and clinically meaningful benefit in adjuvant treatment of Stage IB, II, and IIIA EGFRm NSCLC. The results demonstrated unprecedented disease free survival (DFS) in the adjuvant treatment of these patients after complete tumor resection with curative intent. Osimertinib was assessed against placebo for a treatment duration of up to three years and then unblinded two years earlier than expected at the recommendation of the Independent Data Monitoring Committee (IDMC), based on its determination of overwhelming efficacy during a planned safety analysis.
  • In the primary endpoint of DFS in patients with Stage II and IIIA disease, adjuvant (after surgery) treatment with osimertinib reduced the risk of disease recurrence or death by 83% (based on a hazard ratio [HR] of 0.17; 95% confidence interval [CI] 0.12, 0.23; p<0.0001).
  • DFS results in the overall trial population, Stage IB through IIIA, a key secondary endpoint, demonstrated a reduction in the risk of disease recurrence or death of 79% (based on a HR of 0.21; 95% CI 0.16, 0.28; p<0.0001).
(more…)
ASCO, Author Interviews, Cancer Research / 12.06.2020

MedicalResearch.com Interview with: Alex Spira, MD, PhD, FACP Medical Oncologist Virginia Cancer Specialists and Chair of the US Oncology Research Executive Committee MedicalResearch.com: What is the background for this study? Would you explain what the conjugate consists of and what types of cancer it may target? What are the main findings? Response: The concept of the CX072 and CX2029 studies is that they use what’s called a probody molecule that gets broken down only at the tumor site. This is completely novel in that it helps diminish toxicity by not having less systemic absorption. In the case of CX2029, this target was previously undruggable, meaning the systemic toxicity was too high. By limiting it to activity at the tumor, that is significantly abated.   (more…)
ASCO, Author Interviews, OBGYNE, Ovarian Cancer / 12.06.2020

MedicalResearch.com Interview with: Robert L. Coleman, MD, FACOG, FACS Chief Scientific Officer US Oncology Research MedicalResearch.com: What is the background for this study? Response: For years, there has been general support for surgery in patients with recurrent ovarian cancer supported by reams of retrospective studies that suggest patients live longer if they have surgery preceding chemotherapy. Suggested hypotheses from these trials were that patients most likely to benefit from the procedure were those with good performance status (could tolerate the procedure), had long platinum-free interval (surrogate for potential for chemotherapy response) and those in whom all disease could be resected. Each of these are also characteristics that would portend a good prognostic cohort in general and would likely do better than other patients without these characteristics. So there was a strong selection bias in these retrospective surveys. Thus, the call for randomized trials. GOG-213 was launched in 2007 with 2 primary endpoints: 1. Determine the impact of adding bevacizumab to paclitaxel/carboplatin in patients with platinum-sensitive recurrent ovarian cancer, and 2. Determine if surgery increases overall survival. (more…)
Author Interviews, Dermatology / 12.06.2020

MedicalResearch.com Interview with: David Granville PhD, FAHA Professor, Pathology and Laboratory Medicine, UBC Associate Director, Vancouver Coastal Health Research Institute, VGH-UBC Associate Director, BC Professional Firefighters Burn & Wound Healing Group, Department of Surgery, UBC Principal Investigator, iCORD and UBC Centre for Heart Lung Innovation MedicalResearch.com: What is the background for this study? Response: Atopic dermatitis (aka. eczema) is a chronic inflammatory skin condition characterized by patches of dry, red, itchy skin. These patches can come and go - a process often referred to as 'flare ups'. Often when these flare ups occur, people avoid going out, or to work, resulting in lost productivity and reduced quality of life. While the cause of these flare-ups is not completely understood, a loss of the skin's protective barrier function is believed to be a triggering event. This is because the outer layer of skin (epidermis) acts as a barrier to allergens and other foreign entities from getting into the skin. When this outer barrier is lost, allergens are able to cross and penetrate the deeper layers of skin. This triggers an inflammatory response. The inflammatory response, in turn, can release factors that cause further disruption of the barrier thereby exacerbating the flare up. The outer skin barrier can be thought of in terms of a brick wall in which the 'bricks', or skin cells in this case, are held together by a molecular 'grout' known as adhesion proteins. If these adhesion proteins, which tightly anchor the skin cells together, are lost, the skin becomes more permeable to the outer environment, allowing foreign antigens to enter in, and conversely, moisture to escape out resulting in skin dryness and shedding (more…)
Author Interviews, Pediatrics / 12.06.2020

MedicalResearch.com Interview with: Dr Joanna Garstang Consultant Community Paediatrician / Designated Doctor for Child Death Allenscroft Children's Centre Kings Heath, Birmingham UK MedicalResearch.com: What is the background for this study? Response: Each year in England and Wales there around 3-400 babies die unexpectedly, in many cases the cause of death remains unexplained and these deaths are called Sudden Infant Death Syndrome (SIDS). Parents are understandably anxious about the risks for future children, the Care of Next Infant (CONI) programme was set up to offer support for families. In this study, we looked at records of families registered with CONI between 2000-2015 to determine the frequency and causes for repeat unexpected infant deaths. (more…)
Author Interviews, COVID -19 Coronavirus / 11.06.2020

MedicalResearch.com Interview with: Robin Gelburd President, FAIR Health  MedicalResearch.com: What is the background for this study? Response: As a result of the COVID-19 pandemic, healthcare professionals have been under prolonged stress, including the financial stress of widespread deferral of elective procedures. To investigate the pandemic’s impact on professionals’ utilization and revenue, FAIR Health analyzed data from its database of over 31 billion private healthcare claim records, the nation’s largest such repository, which is growing by over 2 billion claim records per year.  (more…)
Author Interviews, COVID -19 Coronavirus, Weight Research / 11.06.2020

MedicalResearch.com Interview with: Sarah Messiah, PhD, MPH Professor of epidemiology, human genetics, and environmental sciences UTHealth School of Public Health Dallas MedicalResearch.com: What is the background for this study? Response: Given that obesity is a prevalent, serious, complex, chronic, and relapsing disease, and severe obesity is a deepening crisis, it is important to pay special attention to these challenges during the COVID-19 pandemic. This will avoid placing an even greater burden on individuals, health systems, and society in the post-COVID-19 recovery phase. Thus, it is critically important to document, in real time, how socioecological determinants of health are impacting behaviors among those with obesity. Before our study, how the COVID-19 pandemic is impacting weight management, health behaviors, and psychosocial health in particular among people with obesity was unknown. (more…)
Author Interviews, Endocrinology, Thyroid Disease / 11.06.2020

MedicalResearch.com Interview with: https://www.horizontherapeutics.com/ Elizabeth H.Z. Thompson, Ph.D. Group vice president, Clinical Development and External Search Horizon Therapeutics MedicalResearch.com: What is the background for this study? Response: This study provides the first U.S.-based validation of the Graves' Ophthalmopathy Quality of Life (GO-QOL) questionnaire. For your background, Graves’ Ophthalmopathy is another term used to describe Thyroid Eye Disease (TED). The GO-QOL questionnaire includes eight questions, each on visual functioning and appearance-related impacts of TED on patients. Though widely used and validated in Europe, the relevance of the questions for patients in the United States hasn’t previously been explored.For this evaluation, 13 eligible TED patients completed the questionnaire and then underwent a separate cognitive QOL-related interview. (more…)
Author Interviews, COVID -19 Coronavirus, Environmental Risks, JAMA / 11.06.2020

MedicalResearch.com Interview with: Mohammad Sajadi, MD Associate Professor Institute of Human Virology Global Virus Network Center of Excellence University of Maryland School of Medicine Baltimore, MD 21201  MedicalResearch.com: What is the background for this study? Response: Because of geographical proximity and significant travel connections, epidemiological modeling of the epicenter predicted that regions in Southeast Asia, and specifically Bangkok would follow Wuhan, and China in the COVID-19 epidemic. When we saw this did not happen, we suspected that SARS-CoV-2 might be acting like a seasonal respiratory virus.  (more…)
Author Interviews, JAMA, Pain Research / 11.06.2020

MedicalResearch.com Interview with: Tobias Kurth, MD, ScD (Pronouns: he/him) Professor of Public Health and Epidemiology Director, Institute of Public Health Charité – Universitätsmedizin Berlin  MedicalResearch.com: What is the background for this study? Response: Migraine (with aura) has been associated with increased risk of cardiovascular disease but its absolute contribution in relation to other major vascular risk factors was not unclear. (more…)
AstraZeneca, Author Interviews, Rheumatology / 11.06.2020

MedicalResearch.com Interview with: Richard Alan Furie, MD Professor, Center for Autoimmune, Musculoskeletal and Hematopoietic Diseases Feinstein Institutes for Medical Research Chief, Division of Rheumatology, Northwell Health Professor of Medicine, Donald and Barbara Zucker School of Medicine Hofstra/Northwell MedicalResearch.com: What is the background for this study? Response: It has been known for decades that type I interferons play a role in SLE pathogenesis, and therefore the burning question has been whether inhibitors of these pro-inflammatory cytokines would reduce SLE disease activity and could be used as a therapeutic.  There are several strategies for inhibiting the type I interferon pathway, but a conventional approach is to create an antibody against the target protein. The first few clinical trials in SLE evaluated monoclonal antibodies to alpha interferon.  Results were modest at best.  Since this approach only inhibited one (alpha) of five type I interferon subtypes, there were still four subtypes unaffected that could provoke inflammation.  A rather crucial piece of information is that all five subtypes bind to the same receptor.  Therefore, if the receptor is blocked as opposed to a single cytokine, the entire type I interferon family of proteins would be prevented from binding the receptor. This was accomplished with anifrolumab. The phase 2 study in SLE (known as MUSE), which yielded very robust results, was reported several years ago.  It served as a foundation for the phase 3 program, which consisted of two pivotal studies known as TULIP-1 and TULIP-2. Both studies were reported at the 2019 American College of Rheumatology meeting in November, 2019.  Although TULIP-1 did not achieve the primary endpoint, several secondary endpoints were met.  TULIP-2 was successful.  Between all three studies, approximately 1000 patients were enrolled.  Taking advantage of these large numbers, additional analyses of the combined datasets afforded our ability to answer questions about the effects of anifrolumab that were not previously addressed with greater power. In the narrative that accompanied my presentation, I stated “In lupus, disease activity begets damage, and damage begets more damage.  The long-term sequelae of heightened disease activity, better known as flare, are significant.  Regardless of how flare is defined or measured, a major goal is to prevent flare. It is quite justified to think a drug that reduces lupus disease activity should also prevent flares. Well, the proof is in the pudding. In this analysis, we evaluated the effects of anifrolumab on flares.  Recall that anifrolumab targets the type I interferon receptor, blocking all 5 type I subtypes.  The phase 2 MUSE study yielded robust results as did the phase 3 TULIP-2 study.  While, the phase 3 TULIP-1 study did not achieve its primary endpoint, many secondary endpoints showed benefit. In this study, we focused on flare, and examined TULIP-1 and 2 individually as well as pooled data from both studies.”  (more…)
Author Interviews, JAMA, OBGYNE, Pediatrics / 11.06.2020

MedicalResearch.com Interview with: Colm Travers, M.D., MSPH Assistant Professor Department of Pediatrics University of Alabama at Birmingham MedicalResearch.com: What is the background for this study? Response: It is known that black mothers are much more likely to deliver preterm and low birth weight infants. The purpose of this study was to determine whether racial/ethnic disparities in care practices and outcomes were decreasing or increasing among extremely preterm infants. These are infants born from 22 to 27 weeks of gestation who have a high risk of death and major illnesses.  (more…)