ASCO, Author Interviews, Bayer, Cancer Research, Pediatrics / 03.06.2019

MedicalResearch.com Interview with: Douglas S. Hawkins, M.D. Hematology/Oncology Division Chief and Professor Pediatrics at Seattle Children's Hospital University of Washington School of Medicine MedicalResearch.com: What is the background for this study? Response: TRK fusion cancer is caused by a rare genomic alteration called a neurotrophic receptor tyrosine kinase (NTRK) gene fusion. Larotrectinib is a central nervous system (CNS) active, oral and highly selective TRK inhibitor used for the treatment of adult and pediatric patients with solid tumors that have a rare genomic alteration called an NTRK gene fusion. Larotrectinib was approved at the end of 2018 in the U.S. under the brand name VITRAKVI®, with European and worldwide regulatory submissions underway. At ASCO 2019, we will be presenting results from a new analysis specifically looking at the efficacy and safety of larotrectinib in pediatric patients (n=34) included in the expanded dataset from both adults and children across 24 tumor types, which was presented first at the European Society for Medical Oncology (ESMO) 2019 Annual Meeting.  (more…)
Author Interviews, Cancer Research, Prostate Cancer / 03.06.2019

MedicalResearch.com Interview with: Julie N. Graff, MD Associate Professor of Medicine Knight Cancer Institute Chief of Hematology/Oncology VA Portland Health Care System MedicalResearch.com: What is the background for this study? Response: Androgen deprivation therapy is often deployed in patients with a rising PSA after local therapy (such as radical prostatectomy or radiation therapy). With time, the prostate cancer can develop resistance to ADT, at which point it is called castration resistant prostate cancer (CRPC). There were 6 treatments for metastatic CRPC that have shown improved survival. However, in non-metastatic disease, there was nothing that showed improved survival. The SPARTAN study was designed to determine if a next generation androgen receptor antagonist could delay the time to metastatic disease. Overall survival was a secondary endpoint.  (more…)
ASCO, Author Interviews, Cancer Research, J&J-Janssen, Leukemia / 03.06.2019

MedicalResearch.com Interview with: Paul M. Barr, M.D. Associate Professor of Medicine and Director of the Clinical Trials Office Director of the Clinical Trials Office Wilmot Cancer Institute  MedicalResearch.com: What is the background for this study?   Response: When the study was designed, chronic lymphocytic leukemia (CLL)  treatment options were largely limited to chemotherapy and monoclonal antibodies. Ibrutinib had shown promise in early studies. The intent was to compare ibrutinib to a standard of care treatment option at that time, of atumumab, in patients with relapsed or refractory disease. The goal of the current analysis is to evaluate the durability of ibrutinib and report the long-term safety results. (more…)
Accidents & Violence, Author Interviews, Pediatrics, Pediatrics, Toxin Research / 03.06.2019

MedicalResearch.com Interview with: Dr. Gary Smith, MD MPH Director, Center for Injury Research and Policy Nationwide Children’s Hospital Columbus, OH MedicalResearch.com: What is the background for this study? What are the main findings? Response: Our 2016 study (https://pediatrics.aappublications.org/content/137/5/e20154529) investigated calls to US poison control centers related to laundry and dishwasher detergent exposures among children younger than 6 years old from 2013 through 2014 and found that poison control centers received more than 30 calls a day about children who had been exposed to a laundry detergent packet, which is about one call every 45 minutes. The current study investigated trends in calls to poison control centers across the country for exposure to liquid laundry detergent packets in order to evaluate the impact of the voluntary safety standard for this product with a focus on young children. The study found only a modest decrease (18%) in calls for children younger than 6 years of age following adoption of a 2015 product safety standard as well as an increase in calls for older children and adults. Exposures to the eyes also continued to climb. The observed decrease in exposures among young children is considerably less than the 40% to 55% decrease in toxic ingestions seen after passage of the Poison Prevention Packaging Act. This demonstrates that the current liquid laundry detergent safety standard is inadequate and needs to be strengthened. (more…)
Author Interviews, Emergency Care, Mental Health Research, Psychological Science / 03.06.2019

MedicalResearch.com Interview with: Stephen L. Ristvedt, Ph.D. Associate Professor of Anesthesiology Washington University St. Louis, MO  63110-1093  MedicalResearch.com: What is the background for this study? Response: Having a usual source of healthcare – either with a regular doctor or a medical clinic – is the best way to manage one’s health in a proactive way.  Doctors and clinics can provide ongoing guidance with regard to the use of preventive medical screenings as well as the management of chronic illness.  Unfortunately, a significant proportion of US adults do not have a usual source of healthcare.  Also, many people often rely for their healthcare needs on a hospital emergency department, where there is neither sufficient continuity of care nor counseling for prevention. We wanted to investigate what factors might contribute to suboptimal utilization of healthcare resources.  We were particularly interested in looking at individual psychological factors that might play a role in the choices that people make when seeking healthcare.  One specific psychological characteristic proved to be important in our study.  That characteristic is called “threat sensitivity,” and it is measured with a simple questionnaire.  People who are relatively high in threat sensitivity are prone to experience high levels of anxiety in potentially threatening situations  (more…)
Author Interviews, Cannabis / 03.06.2019

MedicalResearch.com Interview with: Julie Bobitt, PhD Director Interdisciplinary Health Sciences College of Applied Health Sciences University of Illinois at Urbana Champaign Champaign, IL  61820 MedicalResearch.com: What is the background for this study? Response:  Older adults are using cannabis at an increasing rate but little is known about their attitudes about, and experiences – including outcomes- with, recreational and medical cannabis use. We believed a qualitative study, where we conducted focus group interviews, would provide a novel perspective to our understanding and help to identify the most salient themes concerning the use of medical and recreational cannabis by adults aged 60 and older living in Colorado. (more…)
Author Interviews, Breast Cancer, Cancer Research, Genetic Research, Novartis / 03.06.2019

MedicalResearch.com Interview with: Fabrice André, MD, PhD Research director and head of INSERM Unit U981 Professor in the Department of Medical Oncology Institut Gustave Roussy in Villejuif, France Global SOLAR-1 Principal Investigator. MedicalResearch.com: What is the background for this study? How does Piqray®  differ from other treatments for this type of advanced breast cancer? 
  • The US Food and Drug Administration (FDA) approved Piqray® (alpelisib, formerly BYL719) in combination with fulvestrant for the treatment of postmenopausal women, and men, with hormone receptor positive, human epidermal growth factor receptor-2 negative (HR+/HER2-), PIK3CA-mutated, advanced or metastatic breast cancer, as detected by an FDA-approved test after disease progression following an endocrine-based regimen.
  • Piqray is the first and only combination treatment with fulvestrant specifically for postmenopausal women, and men, with HR+/HER2- advanced or metastatic breast cancer with a PIK3CA mutation following progression on or after an endocrine-based regimen, bringing a biomarker-driven therapy option to this population for the first time.
  • Advanced breast cancer is incurable, and patients with all types need more treatment options. With this approval, physicians can now use an FDA-approved test to determine if their patients’ HR+/HER2- advanced breast cancer has a PIK3CA mutation and may be eligible for treatment with Piqray plus fulvestrant combination therapy. 
(more…)
Author Interviews, ENT / 03.06.2019

MedicalResearch.com Interview with: Edward McCoul, MD, MPH, FACS Associate Professor Director, Rhinology and Sinus Surgery Department of Otorhinolaryngology Ochsner Clinic New Orleans, Louisiana  MedicalResearch.com: What is the background for this study? What are the main findings? Response: Although the potential for doctors and patients to misunderstand each other has been noted in other areas of medicine, the meaning of the word "congestion" had not previously been the subject of study.  This paper calls attention to the relevance of potential communication gap in otolaryngology.  This is particularly important since congestion is a major diagnostic criteria of sinusitis, which ranks nationwide in the top 5 reasons for clinical encounters year after year.  If a communication gap is evident around this particular term, which is integral to establishing a diagnosis of sinusitis, then the likelihood increases that patients who present with "sinusitis" will be incorrectly diagnosed. The process of congestion refers to the microscopic accumulation of blood and/or fluid within cells or the spaces between cells in a particular tissue or body part.  When this occurs in the nose, the result is swelling inside the nose, which narrows the space for air to flow.  A patient would perceive this as blockage or obstruction of airflow.  This can be treated with anti-inflammatory medication that reduces swelling.  Medications that clear up mucus generally are not great at reducing swelling.  Many over-the-counter products are available that combine both types of medications, but using those products runs the risk of overmedication, which can have adverse consequences. (more…)
ASCO, Author Interviews, Breast Cancer, Cancer Research, Chemotherapy, Radiation Therapy / 03.06.2019

MedicalResearch.com Interview with: Manjeet Chadha, MD, MHA, FACR, FASTRO Prof. Radiation Oncology Director of the Department of Radiation Oncology Mount Sinai Downtown  MedicalResearch.com: What is the background for this study? Response: Largely, the goal of cancer care among the elderly is to de-escalate therapy searching for a modality that is both an effective treatment and also associated with minimal toxicity. Approximately, 30% of new breast cancers diagnosed annually are among women older than 70 years of age. Age-adjusted trends note a relatively higher incidence of stage I breast cancer in women between the ages of 70-74 years. For this group of patients, it is imperative that we take a closer look at the evidence-base for our current practice standards, and evaluate opportunities to improve cancer care delivery in the elderly. Randomized trials have helped arrive at an acceptance of adjuvant endocrine monotherapy in older patients with ER positive, node negative breast cancer. However, in the older patients high rates of non-compliance to tamoxifen secondary to poor tolerance is widely recognized. Emerging data also detail the side effect profile of aromatase inhibitors. Most commonly observed symptoms of arthralgia, reduced bone mineral density, and increased risk of fractures throughout the duration of treatment are important considerations for an older population. At least a quarter of patients on aromatase inhibitors discontinue therapy specifically due to skeletal events and musculoskeletal symptoms. Overall, the side effects of ET contribute to a high rate of non-compliance and negative impact on patients’ quality of life. (more…)
Author Interviews, Dental Research / 02.06.2019

MedicalResearch.com Interview with: Dr. Katie Suda, PharmD, M.S.  Associate Professor College of Pharmacy University of Illinois at Chicago  MedicalResearch.com: What is the background for this study? Response: Dentists prescribe approximately 1 in every 10 antibiotics in the United States and are the top specialty prescriber. Dentists are the primary prescriber of clindamycin in the U.S., which is associated with a high risk of C. difficile infection (an overgrowth of bacteria in the GI tract that can cause a life-threatening infection). Clinical guidelines recommend that patients with specific cardiac conditions receive a dose of antibiotics prior to undergoing invasive dental procedures to prevent infective endocarditis (an infection of the heart values). Taking a dose of antibiotics prior to a dental visit is referred to as antibiotic prophylaxis. Starting in 2007, these guidelines were narrowed secondary to poor evidence on the effectiveness of antibiotic prophylaxis and the risk of antibiotic-related adverse events. Antibiotic adverse events include antibiotic resistance, C. difficile infection, and other general adverse events (nausea, vomiting, diarrhea). While significant research has been conducted on unnecessary prescribing of antibiotics by medical providers, little work has described appropriateness of prescribing by dentists. We assessed if antibiotics prescribed for prophylaxis prior to dental procedures were consistent with clinical guidelines. (more…)
ASCO, Author Interviews, Biomarkers, Melanoma, NYU / 02.06.2019

MedicalResearch.com Interview with: David Polsky, MD, PhD Dermatologist and Director of the Digmented Lesion Service Mahrukh M. Syeda, MS Research Associate Ronald O. Perelman Department of Dermatology NYU Langone Health MedicalResearch.com: What is the background for this study? Response: Several studies of metastatic melanoma patients have demonstrated that measuring circulating tumor DNA (ctDNA) associates with their disease burden and survival.  Generally, patients with detectable pre-treatment ctDNA levels and/or detectable ctDNA at various time intervals after starting treatment have shorter survivals than patients with lower pre-treatment or on-treatment ctDNA levels.  Studies have varied in their methods to detect ctDNA, the thresholds chosen to call a sample positive or negative, and the follow up time point for measurement, if any. In this study, we examined pre-treatment and week 4 on-treatment plasma samples from patients enrolled in Combi-D, the Phase III, randomized, double-blind trial of the BRAF and MEK inhibitors Dabrafenib and Trametinib, which led to FDA approval of the combination therapy for patients with unresectable stage III/IV melanoma. Only patients with BRAF V600E or V600K mutations identified from tumor genotyping were enrolled in the clinical trial. (more…)
Author Interviews, Brigham & Women's - Harvard, Exercise - Fitness, JAMA / 02.06.2019

MedicalResearch.com Interview with: I-Min Lee, MD, ScD Professor of Medicine, Harvard Medical School Professor of Epidemiology Harvard T.H. Chan School of Public Health  MedicalResearch.com: What is the background for this study? Response: While we have many studies showing that physical activity is beneficial for health, there are few data on steps and health, particularly long-term health outcomes.  An expert committee – the 2018 Physical Activity Guidelines Advisory Committee, which reviewed the scientific evidence to support the recently released Physical Activity Guidelines for Americans, 2nd edition – noted this (i.e., the relation between steps and health outcomes) to be a critical gap in knowledge, since many individuals are using wearables and monitoring their step counts. We often hear the number 10,000 steps cited as a daily goal, but the basis for this number is unclear. It likely originated as a marketing tool: in 1965, the Yamasa Clock and Instrument Company, Japan sold a pedometer called “Manpo-kei” – “ten thousand steps meter” in Japanese. For many older people, 10,000 steps/day can be a very daunting goal; thus, we wanted to investigate whether this was necessary for lower mortality rates in older women.  Additionally, steps taken can be fast or slow, and there are no published studies on step intensity and long-term health outcomes.  Note that walking pace and step intensity are not the same concept: walking pace gauges intensity when walking purposefully (e.g., for exercise or transportation), while step intensity assesses an overall best natural effort in our daily life. (more…)
ASCO, Author Interviews, Cancer Research, J&J-Janssen, NEJM, Prostate Cancer / 01.06.2019

MedicalResearch.com Interview with: Dr. Kim Chi. MD Professor of Medicine Medical Oncologist and Medical Director at BC Cancer – Vancouver University of British Columbia, Principal Investigator of the TITAN Study. MedicalResearch.com: What is the background for this study? Response: For more than 70 years, androgen deprivation therapy (ADT) has been the standard of care therapy for patients with metastatic prostate cancer. The Phase 3 TITAN study looked at adding apalutamide (®®®®) to ADT compared with placebo plus ADT in a broad group of patients with metastatic castration-sensitive prostate cancer (mCSPC), regardless of disease volume or prior docetaxel treatment history. Metastatic castration-sensitive prostate cancer is prostate cancer that still responds to androgen deprivation therapy and has spread to other parts of the body. Patients with mCSPC tend to have a poor prognosis, with a median overall survival (OS) of less than five years, underscoring the need for new treatment options. The dual primary endpoints of this study were overall survival and radiographic progression-free survival (rPFS).  (more…)
Author Interviews / 01.06.2019

MedicalResearch.com Interview with: Erika Garcia, PhD, MPH Postdoctoral Scholar Division of Environmental Health Department of Preventive Medicine Keck School of Medicine University of Southern California Los Angeles, CA 90089-9237 MedicalResearch.com: What is the background for this study? Response: It is known that air pollution can exacerbate pre-existing asthma, but what is not yet established is whether air pollution exposure contributes to the development of new cases of asthma. There is increasing scientific evidence supporting a role of air pollution in asthma development. In this study we examined whether reductions in air pollution levels in Southern California were associated with subsequent reductions in rates of new-onset asthma in children. We separately evaluated four pollutants: nitrogen dioxide, ozone, coarse particulate matter (PM10), and fine particulate matter (PM2.5). (more…)
Author Interviews, Dermatology, JAMA / 01.06.2019

MedicalResearch.com Interview with: Jung Min Bae, MD, PhD Associate Professor, Department of Dermatology St. Vincent's Hospital College of Medicine, The Catholic University of Korea  MedicalResearch.com: What is the background for this study? Response: Vitiligo is a common chronic skin disease affecting 1% of the population, and it causes low self-esteem and social stigma. To date, there are no approved drugs for the treatment of vitiligo, even though growing evidence indicates favorable therapeutic responses of topical calcineurin inhibitors (TCIs) including tacrolimus and pimecrolimus. In this study, we conducted a systematic review and meta-analysis of all relevant prospective studies (n = 46) and identified remarkable therapeutic responses of TCI monotherapy and TCI plus phototherapy for vitiligo. (more…)
Author Interviews, Pharmaceutical Companies, Prostate, Urology / 31.05.2019

MedicalResearch.com Interview with: Steven A. Kaplan, M.D., FACS Professor of Urology Director, The Men's Health Program Icahn School of Medicine at Mount Sinai MedicalResearch.com: What is the background for this study? Response: PLUS is the first large-scale trial conducted in North America and Europe specifically designed to study the effects of mirabegron in controlling residual symptoms of urinary urgency and frequency in men with benign prostatic hyperplasia (BPH) using common agents such as tamsulosin (Flomax). We explored whether mirabegron (Myrbetriq), an agent approved for the treatment of overactive bladder (OAB), improved patient outcomes when added to tamsulosin. This was a randomized, double-blind, placebo-controlled, multi-center study enrolling 715 male patients 40 years of age and older. (more…)
Author Interviews, Gastrointestinal Disease, Heart Disease, Pharmacology / 31.05.2019

MedicalResearch.com Interview with: Ziyad Al-Aly, MD, FASN Assistant Professor of Medicine Director of the Clinical Epidemiology Center Chief of Research and Education Department of Veterans Affairs Health Care System Saint Louis  MedicalResearch.com: What is the background for this study?   Response: In 2017, we published a paper showing increased risk of death associated with Proton-pump inhibitors (PPI) use. Following the publication of that 2017 paper, several key stakeholders including patients, doctors, research scientists, medical media folks, mainstream media folks, and others asked us: what do these people die from? Did you study causes of death attributable to PPI use? In the study published today, we developed a causal inference framework to answer this question. (more…)
Author Interviews, Heart Disease, Supplements / 31.05.2019

MedicalResearch.com Interview with:

Lu Qi, MD, PhD, FAHA MD, PhD, FAHA HCA Regents Distinguished Chair and Professor Director, Tulane University Obesity Research Center Department of Epidemiology Tulane University School of Public Health and Tropical Medicine New Orleans, LA 70112 

MedicalResearch.com: What is the background for this study? Response: There is evidence from previous experimental studies or cross-sectional analyses in humans linking glucosamine and a variety potentially protective effects such as improving lipids, inhibiting inflammation, and mimic a low-carb diet.   (more…)
Author Interviews, Boehringer Ingelheim, NEJM, Pharmaceutical Companies, Pulmonary Disease / 30.05.2019

MedicalResearch.com Interview with: Donald Zoz, M.D., Senior associate director Clinical Development & Medical Affairs Boehringer Ingelheim Pharmaceuticals, Inc. MedicalResearch.com: What is the background for this study? How does nintedanib differ from other treatments for SSc-ILD? What are the main findings?  Response: SENSCIS is a Phase III double-blind, randomized placebo-controlled trial that included 576 patients in 32 countries. It is the largest trial to have been conducted in patients with systemic sclerosis associated interstitial lung disease (SSc-ILD). The primary endpoint was the annual rate of decline in forced vital capacity (FVC) over 52 weeks. At the end of the 52-week trial, patients receiving nintedanib had an adjusted annual rate of decline in FVC (mL/year) of -52.4 with nintedanib versus -93.3 with placebo (absolute difference 41.0mL/year [95% CI 2.9, 79.0]; p=0.04). This corresponds to a relative difference of 44% reduction in lung function decline. There are currently no approved treatments for SSc-ILD., BI conducted the SENSCIS study to evaluate in SSc-ILD patients the impact of nintedanib. Nintedanib, a selective tyrosine kinase inhibitor, is an antifibrotic agent. Results of the study, which were published in The New England Journal of Medicine and presented at the American Thoracic Society (ATS) International Conference, showed that nintedanib slowed the loss of pulmonary function by 44% in patients with SSc-ILD relative to placebo, as measured by FVC over 52 weeks.  (more…)
Author Interviews, Dermatology / 30.05.2019

MedicalResearch.com Interview with: Lara B. McKenzie, PhD, MA Principal Investigator in the Center for Injury Research and Policy Research Institute at Nationwide Children's Hospital Associate Professor in the Department of Pediatrics College of Medicine and the Division of Epidemiology College of Public Health at The Ohio State University MedicalResearch.com: What is the background for this study? What are the main findings? Response: Social media and other online tools have changed the way people seek and share health information. Recent consumer interest in natural, organic, and ethically-made personal care products has led to an increase of shared recipes for homemade products including sunscreen. The study found that nearly all (95%) pins, or bookmarks, for homemade sunscreen positively portrayed the effectiveness of homemade sunscreens and most (68%) recommended recipes for homemade sunscreens that offered insufficient UV radiation protection. Sun Protection Factor (SPF) claims were made in a third of pins with a range of SPF 2 to SPF 50. This is concerning because the ingredients recommended in homemade sunscreen pins offer minimal scientifically proven broad-spectrum protection from UV radiation yet are widely shared and promoted as safe alternatives to commercial sunscreens on Pinterest. Homemade sunscreen products are risky because they are not regulated or tested for efficacy like commercial sunscreens. When you make it yourself, you don’t know if it’s safe or effective. With rising skin cancer rates, the use of effective broadband sunscreen is critical to protect the skin from UV radiation and reduce incidence of skin cancer.  (more…)
Author Interviews, Clots - Coagulation, Duke, Genetic Research, Heart Disease, JAMA / 30.05.2019

MedicalResearch.com Interview with: Thomas J. Povsic, MD, PhD Interventional Cardiologist Duke Clinical Research Institute Duke University School of Medicine Durham, North Carolina  MedicalResearch.com: What is the background for this study?  Response: The background for this study is that it is unknown how mandatory reporting of CYP2C19 metabolizer status affects how doctors treat patients or to what degree provision of this information would affect choice of a P2Y12 inhibitor within a clinical trial. As part of the GEMINI-ACS trial, all patients underwent CYP2C19 metabolizer testing.  This trial enrolled patients with a recent acute coronary syndrome and randomized them to aspirin or a low dose of rivaroxaban.  All patients were also to be treated with ticagrelor or clopidogrel, which was at the discretion of the investigator.  Investigators were given information regarding the CYP2C19 metabolizer status about a week after randomization.  Importantly prior to randomization, all investigators were asked how they expected to use this information, and then we followed what they actually did. (more…)
Anesthesiology, Author Interviews, Duke, OBGYNE, Opiods, Pain Research, Surgical Research / 29.05.2019

MedicalResearch.com Interview with: Ashraf Habib, MDChief of the Division of Women’s Anesthesia and Professor of AnesthesiologyDuke University Ashraf Habib, MD Chief of the Division of Women’s Anesthesia Professor of Anesthesiology Duke University  MedicalResearch.com: What is the background for this study? What are the main findings? Response: This was a multicenter study conducted in 13 clinical sites in the United States enrolling patients undergoing elective Cesarean-section and receiving spinal anesthesia. 186 patients were enrolled and randomized to receive EXPAREL, a long-acting, non-opioid option to manage postsurgical pain, administered via transversus abdominis plane (TAP) field block, mixed with plain bupivacaine or TAP block with plain bupivacaine alone. A TAP block numbs the nerves that supply the abdominal wall. We presented the data at the 51st Annual Meeting of the Society of Obstetric Anesthesia and Perinatology (SOAP) in Phoenix, AZ. We aimed to collect clinical evidence that a multimodal postsurgical pain regimen using a TAP block with EXPAREL (bupivacaine liposome injectable suspension) together with regularly scheduled acetaminophen and nonsteroidal anti-inflammatory drugs (NSAIDs) could reduce opioid consumption more so than a standard multimodal pain control approach that combines TAP block with standard bupivacaine, regularly scheduled acetaminophen, and NSAIDs. (more…)
Author Interviews, Brigham & Women's - Harvard, Cancer Research, FDA, JAMA, Pharmacology / 29.05.2019

MedicalResearch.com Interview with: Bishal Gyawali  MD PhD
  • Program on Regulation, Therapeutics, and Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham and Women’s Hospital and Harvard Medical School, Boston, Massachusetts
  • Department of Oncology, Department of Public Health Sciences, and Division of Cancer Care and Epidemiology, Queen’s University, Kingston, Ontario, Canada
MedicalResearch.com: What is the background for this study? What are the main findings? Response: Accelerated approval pathway from the FDA allows cancer drugs to come to market sooner by showing improvement in surrogate measures such as change in tumor size. Surrogate measures do not reflect clinical benefit in terms of living longer or feeling better. So, when a drug receives accelerated approval, the drug is required to undergo a confirmatory trial to confirm that true clinical benefit from the drug actually exists. Last year, a paper from the FDA argued that accelerated approval pathway is working effectively because 55% of such drugs confirmed clinical benefit. However, we saw that most of those drugs were actually improving only a surrogate measure even in confirmatory trials. So the confirmatory trials were not confirming clinical benefit but actually confirming benefit in a surrogate endpoint. We investigate that issue in our study using updated results from the confirmatory trials that were ongoing at the time of FDA review. Our main finding is that only one-fifth of cancer drugs that received accelerated approval actually improved overall survival later in confirmatory trials. For, 20% of other drugs, the confirmatory trials tested the same surrogate endpoint as did the preapproval trial. For another 21%, the confirmatory trial showed benefit in a surrogate endpoint different from the one used in preapproval trial. Furthermore, when drugs fail to confirm clinical benefits in confirmatory trials, they still continue to remain on market.  (more…)
Author Interviews, Exercise - Fitness, JAMA, Mental Health Research, Pediatrics, UCLA / 29.05.2019

MedicalResearch.com Interview with: Molly C. Easterlin, MD Fellow, UCLA National Clinician Scholars Program Clinical Instructor, Pediatrics, Cedars-Sinai Medical Center  MedicalResearch.com: What is the background for this study? Response: Adverse childhood experiences or ACEs (including physical or emotional neglect or abuse, sexual abuse, domestic abuse, exposure to household substance misuse or mental illness, parental separation or divorce, and parental incarceration) are common with about half of children experiencing 1 and one-quarter of children experiencing 2 or more. Children exposed to adverse childhood experiences have worse mental health throughout life, including higher rates of depression and anxiety. However, little is known about what factors improve long-term mental health in those exposed to ACEs. Additionally, as far as we are aware, no studies have looked at team sports participation as a potential factor that may be associated with improved mental health among those with adverse childhood experiences. (more…)
Author Interviews, Social Issues / 29.05.2019

MedicalResearch.com Interview with: Peter Belmi PhD Assistant Professor of Leadership and Organizational Behavior Darden School of Business University of Virginia MedicalResearch.com: What is the background for this study?  Response: We wanted to understand how socioeconomic inequalities perpetuate from one generation to the next. Some scholars have suggested that social inequalities persist because of systemic prejudice that make it difficult for those at the bottom to improve their standing. Other scholars have suggested that structural inequalities may be hard to dismantle because those who wield the most influence are motivated to preserve their advantages. And other scholars have suggested that inequality may perpetuate when mainstream institutions do not acknowledge the values and norms of individuals from underrepresented groups. (more…)
Author Interviews, CDC, Flu - Influenza, Vaccine Studies / 29.05.2019

MedicalResearch.com Interview with: Megan C. Lindley, MPH Deputy Associate Director for Science Immunization Services Division CDC MedicalResearch.com: What is the background for this study? Response: Despite longstanding recommendations from the Advisory Committee on Immunization Practices, healthcare personnel influenza vaccination coverage remains below the Healthy People 2020 target of 90%. Healthcare employers use a variety of strategies to promote influenza vaccination among healthcare personnel, including facility-level mandates for vaccination. Several U.S. states have also enacted laws related to healthcare personnel influenza vaccination, but the effect of these laws on vaccination uptake is unclear. Our study used influenza vaccination coverage data reported by over 4,000 U.S. hospitals to examine three kinds of laws: (1) Assessment laws, which require hospitals to assess healthcare personnel influenza vaccination status; (2) Offer laws, which require hospitals to offer the influenza vaccine to healthcare personnel; and (3) Ensure laws, which require hospitals to require healthcare personnel to demonstrate proof of influenza vaccination. (more…)
Author Interviews, Epilepsy, FDA / 29.05.2019

MedicalResearch.com Interview with: Dr. Steven S. Chung, MD Executive Director and Program Chair Neuroscience Institute and Director of the Epilepsy Program Banner – University Medical Center MedicalResearch.com: What is the background for this study? How is Nayzilam different from other treatments for epilepsy? Who/How is it administered?  Response: NAYZILAM is the first medication and only FDA-approved nasal option for treating seizure clusters. NAYZILAM allows for administration by a non-healthcare professional to patients when a seizure cluster occurs, which could provide significant value to patients who currently have limited treatment options for SC. The effectiveness of NAYZILAM was established in a randomized, double-blind, placebo-controlled trial (Study 1; NCT 01390220). Study 1 was conducted in two phases: an open-label Test Dose Phase followed by a randomized, double-blind, placebo-controlled, Comparative Phase. In the Test Dose Phase, tolerability was assessed in 292 patients. Patients were excluded from participation in the Comparative Phase if they failed to meet pre-defined blood pressure, heart rate, sedation, electrocardiogram, and peripheral oxygen saturation criteria. In the Comparative Phase, 201 patients treated a single seizure cluster episode in an outpatient setting. Numerical differences in favor of NAYZILAM were observed on each of the components of the treatment success responder definition; termination of seizure(s) within 10 minutes after initial dose of study drug (80.6 versus 70.1%) and the absence of seizure recurrence between 10 minutes and 6 hours after the initial dose of study drug (58.2 versus 37.3%). Study 1 also evaluated the occurrence and time to next seizure after the initial blinded dose of study drug. A smaller proportion of NAYZILAM-treated patients experienced the next seizure within 24 hours after the initial blinded dose of study drug (37.3% versus 46.3%). NAYZILAM-treated patients experienced a statistically longer time-to-next-seizure than the placebo group.   (more…)
Author Interviews, Baylor University Medical Center Dallas, Gastrointestinal Disease / 28.05.2019

MedicalResearch.com Interview with: Dr. Rhonda Souza, MD Baylor University Medical Center Center for Esophageal Research Dallas, TX 75246  MedicalResearch.com: What is the background for this study?   Response: Eosinophilic esophagitis (EoE) is a modern disorder of the esophagus caused by an allergy to certain foods. EoE causes esophageal symptoms like difficulty swallowing and heartburn and is diagnosed when biopsies of the esophagus taken during endoscopy show numerous eosinophils, which are a type of inflammatory blood cell.  There are few established treatments for EoE. One such treatment is a diet that eliminates the offending food allergens, and another is to use steroids to reduce the number of eosinophils in the esophagus. However, the most common treatment for adults with eosinophilic esophagitis is to use proton pump inhibitors (PPIs), which block the proton pumps in stomach cells that make acid.  In earlier studies, we found that PPIs also can block proton pumps in esophageal cells.  Those proton pumps are activated by chemicals that the body produces in response to allergens including interleukin (IL)-13 or IL-4.  Il-13 and IL-4, which cause the esophagus to produce eotaxin-3, a molecule that attracts eosinophils. What remained unknown, however, was the mechanism whereby these interleukins activate proton pumps in the EoE esophagus. In our present study, we explored whether IL-4 works by increasing calcium levels in esophageal cells from EoE patients. (more…)
Author Interviews, Dermatology / 28.05.2019

MedicalResearch.com Interview with: Venkateswarlu (Venkat) NelabhotlaPresident & Board MemberVyome Therapeutics Inc., Venkateswarlu (Venkat) Nelabhotla President & Board Member Vyome Therapeutics Inc.,  MedicalResearch.com: What is the background for this study? Response: There are only a handful of antibiotics that are approved for use against the bacterial strain P.acnes that causes acne vulgaris. Because of the lack of new antibiotic treatment options, similar antibiotics are overused resulting in 1 out of 3 patients developing resistance against treatment, ultimately causing a decline in response rates. There is a clear unmet medical need for these patients; currently, patients do not have access to topical antibiotic treatments capable of an effective response without generating resistance. This study was designed to test the efficacy of VB-1953 in such moderate to severe acne patients, who were carrying resistant acne-causing bacteria and had failed to respond to currently approved antibiotics. VB-1953, a bactericidal topical gel formulated antibiotic, differs from these older generation antibiotics in its novel mechanism of action, through which it can kill both sensitive as well as resistant bacteria, while also reducing inflammation . The earlier generation of antibiotics, including treatments like clindamycin, are bacteriostatic in action, i.e. they merely prevent the growth of acne-causing bacteria. VB-1953 is the first bactericidal antibiotic for the treatment of acne that kills acne-causing bacteria. VB-1953 also retards the development of resistance, and reduces inflammation associated with acne via an independent immunomodulatory effect.  (more…)