Allergies, Author Interviews, Autism, JAMA, Pediatrics / 10.06.2018

MedicalResearch.com Interview with: Wei Bao, MD, PhD Assistant Professor, Epidemiology College of Public Health University of Iowa MedicalResearch.com: What is the background for this study? What are the main findings? Response: Previous studies indicated a possible link between immunologic dysfunction and autism. The current study, based on nationally representative large-scale surveys, showed that food allergy, respiratory allergy, and skin allergy, all relevant to immunological dysfunction, were associated with autism spectrum disorder among US children. (more…)
Author Interviews, Fertility, Nature / 09.06.2018

MedicalResearch.com Interview with: Tomer Avidor-Reiss, Ph.D.  Professor, College of Natural Sciences and Mathematics Department of Biological Sciences University of Toledo Toledo, OH 43606 MedicalResearch.com: What is the background for this study? What are the main findings? Response: Most dividing cells in the body need exactly two centrioles for normal cell division and development. Abnormalities in centriole number can cause cancer and devastating developmental defects. Because of this importance of centriole number and because all cells originate from the zygote – the product of the sperm and egg – it makes sense that the zygote should possess two centrioles, as well. However, the egg does not contain any centrioles, so the sperm is the sole contributor of the centrioles; and yet, it is currently thought that the sperm contains only one centriole. This is problematic because supposedly that leaves the zygote with only one centriole, even though it must propagate cells with two centrioles. In the past, we found that insect sperm have an atypical centriole that escaped discovery because it is so different. We therefore hypothesized that humans may also have an atypical sperm centriole. Our new paper shows that in human sperm there exists, in addition to the known centriole, a second centriole that deviates from the typical structural and composition that is expected from a centriole. Although it looks very different from any centriole ever described, we found that it functions in an in vitro functional assay. Furthermore, during fertilization, it performs the functions traditionally associated with centrioles. Together, this resolves a 50-year-long debate regarding the centrioles of human sperm; the sperm contains two functional centrioles, despite that one is atypical.  (more…)
Author Interviews, Genetic Research / 09.06.2018

MedicalResearch.com Interview with: “The human Brain” by Kristian Mollenborg is licensed under CC BY 2.0David Haussler PhD Investigator, Howard Hughes Medical Institute Distinguished Professor, Biomolecular Engineering Scientific Director, UC Santa Cruz Genomics Institute Scientific Co-Director, California Institute for Quantitative Biosciences  and Sofie Salama, PhD Research Scientist in BIomolecular Engineering Howard Hughes Medical Institute Senior Scientist MedicalResearch.com: What is the background for this study? What are the main findings? Dr. Haussler:  Researchers specializing in this area are interested understanding which evolutionary changes in our genome underlie human-specific brain features including our large (3X greater than chimpanzee) brain. It has been my personal dream to peer into human evolution at the level of individual genes and gene functions.  (more…)
Author Interviews, Ophthalmology / 09.06.2018

MedicalResearch.com Interview with: “pplkpr phone” by Kyle McDonald is licensed under CC BY 2.0Michelle Nguyen Senior Director, Consumer Marketing Lead Shire Ophthalmics MedicalResearch.com: What is the background for this initiative? How does screen time affect eye health?  Response: Screen responsibly is an educational initiative about the intersection between our screen use and eye health. This initiative is underscored by new national survey data and aimed at increasing our dialogue about how to love both our screens and our eyes. Screen responsibly is an extension of the eyelove campaign, which Shire launched in 2016 to elevate the importance of eye health and raise awareness of the signs and symptoms of Chronic Dry Eye, commonly referred to as Dry Eye. Since being glued to your screen can trigger Dry Eye symptoms, it is important that people are aware of ways to screen responsibly.  MedicalResearch.com:  How common is the problem of Dry Eye? How is it related to excessive screen time? Response: In the United States, nearly 30 million adults report symptoms consistent with Dry Eye. Dry Eye symptoms include a burning, itchy, stinging or gritty feeling in the eyes, episodes of blurred vision, eye redness and watery eyes. Shire is committed to addressing unmet needs within the Dry Eye patient community, and to do so, we sought to better understand their current behaviors, perceptions and knowledge around a common trigger of Dry Eye symptoms – intense screen usage. (more…)
Abuse and Neglect, Author Interviews, Breast Cancer, JAMA, MD Anderson / 08.06.2018

MedicalResearch.com Interview with: Naoto Tada Ueno, M.D., Ph.D., F.A.C.P. Executive Director, Morgan Welch Inflammatory Breast Cancer Research Program and Clinic Section Chief, Section of Translational Breast Cancer Research, Department of Breast Medical Oncology Division of Cancer Medicine The University of Texas MD Anderson Cancer Center Houston, TXNaoto Tada Ueno, M.D., Ph.D., F.A.C.P. Executive Director, Morgan Welch Inflammatory Breast Cancer Research Program and Clinic Section Chief, Section of Translational Breast Cancer Research, Department of Breast Medical Oncology Division of Cancer Medicine The University of Texas MD Anderson Cancer Center Houston, TX   MedicalResearch.com: What is the background for this study? What are the main findings? Response: The best outcome of inflammatory breast cancer (IBC) is dependent on achieving a pathological completed response after neoadjuvant chemotherapy for primary inflammatory breast cancer, which is the most aggressive type of breast cancer. We have conducted extensive preclinical work, which showed that EGFR is a potential therapeutic targets of IBC. Based on this preclinical data, we have conducted a phase II study to determine the pathological complete response rate of panitumumab plus neoadjuvant chemotherapy for HER2 negative primary inflammatory breast cancer.  (more…)
Author Interviews, Heart Disease, Occupational Health / 08.06.2018

MedicalResearch.com Interview with: Eleonor Fransson, PhD Associate Professor in Epidemiology Department of Natural Sciences and Biomedicine School of Health and Welfare JÖNKÖPING UNIVERSITY MedicalResearch.com: What is the background for this study? Response: Atrial fibrillation is a very common heart rhythm disorder affecting a large number of people in the population, but there is limited knowledge about risk factors for the disease. This is especially true when it comes to the role of occupational factors. MedicalResearch.com: What are the main findings? Response: We found that work stress measured as job strain, that is, a combination of having high psychological job demands and low control over the work situation, was associated with almost 50% increased risk of atrial fibrillation. When we combined the results from our study with two previously published studies on the same topic, we found that work stress was associated with 37% increased risk. (more…)
Author Interviews, Autism, Blood Pressure - Hypertension, JAMA, OBGYNE, Pediatrics / 08.06.2018

MedicalResearch.com Interview with: “Blood Pressure” by Bernard Goldbach is licensed under CC BY 2.0Ali Khashan, PhD Senior Lecturer in Epidemiology School of Public Health & INFANT Centre University College Cork Cork, Ireland MedicalResearch.com: What is the background for this study? What are the main findings? Response: There is some evidence to suggest an increased likelihood of neurodevelopmental disorders in relation to hypertensive disorders in pregnancy, however consensus is lacking. Considering hypertensive disorders in pregnancy are among the most common prenatal complication, we decided to synthesise the published literature on this topic by conducting a comprehensive systematic review and meta-analysis. Our main findings suggest that hypertensive disorders in pregnancy are associated with about 30% increase in the likelihood of autism spectrum disorders (ASD) and ADHD in the offspring, compared to offspring not exposed to hypertensive disorders in pregnancy. (more…)
Author Interviews, Brigham & Women's - Harvard, Dermatology, Environmental Risks, Melanoma, Transplantation / 08.06.2018

MedicalResearch.com Interview with: “Sunscreen” by Tom Newby is licensed under CC BY 2.0Rebecca Ivy Hartman, M.D Instructor in Dermatology Brigham and Women's Hospital Boston MA 02115 MedicalResearch.com: What is the background for this study? What are the main findings? Response: Organ transplant recipients (OTR) are at 100-fold higher risk to develop certain skin cancers compared to the general population due to immunosuppression, and thus preventing skin cancer in this population is critical. Our study found that in a high-risk Australian OTR population, only half of patients practiced multiple measures of sun protection regularly. However, after participating in a research study that required dermatology visits, patients were over 4-times more likely to report using multiple measures of sun protection regularly. Patients were more likely to have a positive behavioral change if they did not already undergo annual skin cancer screening prior to study participation. (more…)
Addiction, Alcohol, Author Interviews, Opiods / 07.06.2018

MedicalResearch.com Interview with: Dr. Katie Witkiewitz PhD Professor, Department of Psychology University of New Mexico MedicalResearch.com: What are the main findings? Response: The main findings from our study indicate that individuals with alcohol dependence who misused opioids (e.g., used without a prescription or not as prescribed) had a significantly higher likelihood of relapse to heavy drinking during alcohol treatment and were drinking more alcohol during and following alcohol treatment. (more…)
Amgen, Author Interviews, Gastrointestinal Disease, Gluten / 07.06.2018

MedicalResearch.com Interview with: Markku Mäki, MD, PhD Professor (emeritus) at the University of Tampere and Presently research director at the Tampere University Hospital Tampere, Finland MedicalResearch.com: What is the background for this study? Response: The only treatment for this life-long gluten-induced autoimmune systemic disease is a strict avoidance of wheat, rye and barley, the food cereals which contain gluten, the environmental trigger and driving force in celiac disease.  Gluten causes intestinal inflammation, usually with (but sometimes without) gastrointestinal or nutritional symptoms or signs, and with frequent extra-intestinal diseases. However, it is impossible for celiac disease patients to avoid gluten entirely and indefinitely and a third of patients report symptoms on a strict gluten-free diet. Gut mucosal healing is not optimal in half of the patients, and inflammation and injury is detected for years after starting the diet, presumably due to contamination with gluten in the diet. This is why patients are requesting, and academia and industry are looking for novel adjunct therapies for celiac disease. Initially, these therapies are tested to prevent the consequences of hidden gluten; the ultimate goal being that also celiacs could one day eat safely wheat, barley and rye products. Some 20 novel experimental therapies are at present actively being investigated (modifying wheat or different drugs, devices and vaccines/immunotherapy). The present study investigated whether blocking interleukin 15, an important mediator of celiac disease, reduces or prevents gluten-driven ill health, both the inflammation and injury at the small intestinal mucosal level and gluten-induced symptoms. The experimental drug used was Amgen’s AMG 714, a human monoclonal antibody, used at a low and high dose, in the presence or absence of a high-dose gluten challenge. (more…)
Author Interviews, Dermatology, Hepatitis - Liver Disease, NEJM / 07.06.2018

MedicalResearch.com Interview with: Dr Christophe Corpechot Centre de Référence Maladie Rares: Maladies Inflammatoires des Voies Biliaires et Hépatites Auto-immunes (MIVB-H) Filière Maladies Rares: Maladies Rares du Foie de l’Adulte et de l’Enfant Hôpital Saint-Antoine (APHP) et Sorbonne Universités Paris MedicalResearch.com: What is the background for this study? Response: Primary biliary cholangitis (PBC, previously known as "primary biliary cirrhosis") is a rare, chronic, slowly progressive liver disease of unknown cause, mainly affecting women of middle age. It is characterized by serum marks of autoimmunity (specific auto-antibodies), chronic inflammation and destruction of small intra-hepatic bile ducts, and consequent bile secretion impairment (chronic cholestasis) leading to the progressive development of cirrhosis and liver failure. Ursodeoxycholic acid (UDCA) is the only first-line approved treatment for PBC. It improves the biochemical measures of cholestasis and prolongs survival without liver transplantation. However, 30% to 40% of UDCA-treated patients continue to have clinically significant abnormalities of their biochemical liver tests and those patients remain at high risk of developing end-stage liver disease complications. Recently (2016), obeticholic acid (OCA) in association with UDCA has been conditionally approved in patients with an inadequate response to UDCA. This approval (FDA, EMA) was based one the results of a 1-year randomized, double-blind, placebo-controlled trial of OCA in patients with an incomplete response or intolerance to UDCA (POISE trial). In this trial, OCA was shown to improve the biochemical features of cholestasis (alkaline phosphatase (ALP) level < 1.67 times the upper limit of the normal range and a reduction of at least 15% from baseline) but was associated with a significant increase of pruritus, a characteristic, potentially debilitating symptom of PBC. BEZURSO is the first ever placebo-controlled phase 3 trial of a fibrate (a class of drugs known to be agonists of the peroxisome proliferator-activated receptors alpha) in PBC. In this 2-year randomized double-blind trial, 100 patients with an incomplete response to UDCA were assigned to bezafibrate 400 mg/day (n=50) or placebo (n=50), all in association with continued UDCA therapy. (more…)
ASCO, Author Interviews, Cancer Research / 07.06.2018

MedicalResearch.com Interview with: Axel Le Cesne, MD Institute de Cancerologie Gustave-Roussy Villejuif, France MedicalResearch.com: What is the background for this study? What are the main findings? Response: With the exception of a study in translocation-related sarcomas (TRS) (Kawai, TLO 2015), trabectedin was never compared to best supportive care (BSC) in a randomized study in patients with all STS histotypes. This trial required by French Health Authorities in 2014. The tumor control rate after 6 cycles of trabectedin is similar (30%) to previous study in French referral centers (T-DIS trial, Le Cesne, Lancet Oncol 2015) evaluating trabectedin in all subtypes of STS. As already reported, trabectedin was well tolerated with no cumulative toxicities This study met its first endpoint as a preplanned PFS analysis showed a significant improvement in median PFS with trabectedin  over BSC in patients with pretreated ASTS including multiple histologies (HR: 0.39). A major impact of trabectedin was observed in the L-STS cohort (liposarcomas and leiomyosarcomas) with a median PFS of 1.4 months in the BSC arm and 5.13 m (HR: 0.29, p<0.0001) in the trabectedin arm. respectively). The benefit observed with trabectedin in the L-STS cohort of patients is similar to those observed in the US trial in the same population (4.2 vs 1.5m for DTIC) (Demetri, JCO 2016) and in the Japanese trial mentioned above (5.8 vs 0.9m for BSC) (Kawai, TLO 2015) After the crossing over allowed by the protocol (trabectedin for patients progressing in the BSC arm), safety and efficacy profiles of trabectedin remains similar. We did not observe a difference in terms of OS between the two arms, probably due to the cross-over planned by the protocol.  (more…)
Author Interviews, Biomarkers, Orthopedics / 06.06.2018

MedicalResearch.com Interview with: Rick Sumner, PhD, FAAA The Mary Lou Bell McGrew Presidential Professor for Medical Research Chair, Department of Cell & Molecular Medicine (formerly, Anatomy and Cell Biology) Rush University Medical Center Chicago, IL  60612   MedicalResearch.com: What is the background for this study? What are the main findings? Response: The main cause of failure for total hip replacements is implant loosening which is often a consequence of particle-induced peri-implant osteolysis. Unfortunately, this condition is usually not diagnosed until it has progressed to the point of needing a revision surgery. We discovered two biomarkers that may be useful for identifying at risk patients much earlier than is currently possible. (more…)
ASCO, Author Interviews, Breast Cancer, Brigham & Women's - Harvard / 06.06.2018

MedicalResearch.com Interview with: Dr. Aditya Bardia  MD, MPH Assistant Professor, Medicine Harvard Medical School Attending Physician, Medical Oncology Massachusetts General Hospital MedicalResearch.com: What is the background for this study? What are the main findings? Response: Hormone receptor-positive (HR+)/ and human epidermal growth factor receptor 2-negative (HER2-) breast cancer is the most common sub-type of breast cancer. While metastatic HR+/HER2- breast cancer is initially treated with endocrine therapy-based combinations, including CDK 4/6 inhibitors, patients eventually have disease progression, but the response rate to standard chemotherapy is low (~10-15 percent, post-taxane setting). In particular, patients with visceral disease have a poor prognosis. In this trial, we evaluated the efficacy of sacituzumab govitecan in patients with metastatic HR+/HER2- breast cancer, who had measurable disease and had received prior therapies for metastatic breast cancer. We observed an overall response rate of 31 percent in a heavily pre-treated population (prior number of therapies for metastatic breast cancer = 5; number of patients with prior CDK 4/6 inhibitor use = 69 percent). The responses were durable (median duration of response = 7.4 months). Neutropenia was the main adverse event noted (grade 3 neutropenia = 42 percent), and two patients (3.7 percent) discontinued the clinical trial due to adverse events. The response rate in patients with visceral metastaseswas 27 percent.  (more…)
Author Interviews, Colon Cancer, Gastrointestinal Disease / 06.06.2018

MedicalResearch.com Interview with: Anas Raed, MD Section of General Internal Medicine Augusta University MedicalResearch.com: What is the background for this study? Response: Colorectal cancer (CRC) incidence and mortality rates have been decreasing in the US since mid 1980s, however, recent evidence shows that incidence and mortality rates of CRC in patients younger than 50 years have been increasing significantly. In spite of the increasing trend of colorectal cancer, routine screening of this population has not been addressed due to lack of evidence and cost-effectiveness. Administering screening colonoscopy for all individuals younger than 50 years might not be feasible and, therefore routine screening colonoscopy for specific age groups might reduce the disparity of the incidence in this disease. (more…)
Addiction, Author Interviews, JAMA, Race/Ethnic Diversity / 06.06.2018

MedicalResearch.com Interview with: Randall C. Swaim, Ph.D. Senior Research Scientist and Director Linda R. Stanley, Ph.D. Senior Research Scientist Tri-Ethnic Center for Prevention Research Department of Psychology Colorado State University                           MedicalResearch.com: What is the background for this study? Response: American Indian adolescents consistently report the highest levels of substance use compared with other US racial/ethnic groups. The harm associated with these high rates of use include higher risk of developing a substance use disorder, more alcohol-related problems, including alcohol-attributable death, and other negative outcomes such as school failure. These findings point to the importance of continuing to monitor this group, particularly given changing trends in perceived harmfulness of illicit substances as new statutes alter access to medical and recreational use of cannabis. (more…)
Author Interviews, Kidney Disease, Neurological Disorders, Pain Research, UCSF / 06.06.2018

MedicalResearch.com Interview with: Dr. Julie H. Ishida MD Department of Medicine, Division of Nephrology University of California, San Francisco and San Francisco Veterans Affairs Medical Center MedicalResearch.com: What is the background for this study? What are the main findings? Response: Gabapentin and pregabalin are used for the management of symptoms such as neuropathic pain, itching, and restless leg syndrome in patients receiving hemodialysis. However, hemodialysis patients may be particularly vulnerable to adverse events related to these agents, which are cleared by the kidney, but there is limited data evaluating their risk in this population. Gabapentin and pregabalin use were associated with risk for altered mental status, fall, and fracture, and in some cases, even at doses that would be considered safe for use in this population.  (more…)
Author Interviews, Mental Health Research, Pediatrics / 06.06.2018

MedicalResearch.com Interview with: Alfio Maggiolini, MD Minotauro, Milan MedicalResearch.com: What is the background for this study? What are the main findings? Response: Antisocial behaviour is common during adolescence and it incurs significant costs both for society and for the young people themselves. Persistent antisocial behaviour places a heavy burden on the community, the justice system and the public health system. Responses to juvenile crime have always seen a tension between a focus on the understanding and the rehabilitation of the youth and the need to enforce discipline and public safety through punishment and threat. The treatment of young offenders was traditionally deemed particularly difficult, and often ineffective. In recent years, therapeutic nihilism has given way to cautious optimism. While punitive-based approaches, at all levels, are hardly ever effective in the long term, the most popular and effective programs tend to focus on behaviour control. Common core elements of such programs include positive reinforcement, problem solving skills training and role playing, as behavioral problems are often assumed somewhat inherently wrong, or a “lack of something”, the programs aims at improving or changing. The study presents a developmental approach that understands behavioral problems as the result of intentions, values and goals that need to be taken in full consideration and that are usually legitimate, even though carried out in ways that prove dysfunctional for both the young person and society. In other words, we consider antisocial behaviors as maladaptive responses to legitimate developmental tasks, a deviant way of meeting positive goals and taking control of one’s life. In the program we describe, a developmental understanding is combined with a psychoanalytically informed perspective on treatment and delivered in multi-modal terms. It has been carried out in Italy for the past 20 years, with positive outcomes, both in private practice and within the juvenile justice services.  (more…)
Author Interviews, Infections, Social Issues / 05.06.2018

MedicalResearch.com Interview with: rat- wikipedia imageMícheál de Barra, PhD Lecturer in Psychology Brunel University London MedicalResearch.com: What is the background for this study? What are the main findings? Response: Disgust has been called the "intuitive microbiologist"  - it tracks the sources of infection in our environment. But so far, there has been little attempt to link the sources of disgust to the sources of infectious disease in a comprehensive way. So we developed a method for developing stimuli based on a random sample illness. We basically asked ourselves what the kinds of cues that might be associated with that kind of disease risk and asked people to rate disgust responses. The main motive for this was to contribute to a debate in the literature about if there are "kinds of disgust" and if so, how many. I results were a little ambiguous there I'm afraid. (more…)
ASCO, Author Interviews, Breast Cancer, Cancer Research / 05.06.2018

MedicalResearch.com Interview with: Dr. Giuseppe Del Priore, MD, MPH Chief Medical Officer of Tyme Inc. MedicalResearch.com: What is the background for this study? What are the main findings? Response: Metastatic breast cancer, sometimes also called “stage IV” or “advanced breast cancer,” is the most extensive stage of breast cancer. It is an invasive cancer that has spread to other parts of the body, most often bones, lungs, liver, and brain. The current standard of care for metastatic breast cancer is systemic drug therapies, such as hormone therapy, chemotherapy, targeted drugs or a combination of these.  Because they reach every cell in the body, they have side effects that can worsen the patient’s quality of life. Existing treatments cannot cure metastatic breast cancer and are palliative in intent. This presents a great unmet need and challenge in treating patients with metastatic breast cancer. SM-88 is a novel relatively non-toxic combination therapy that harnesses cancer’s unique cell metabolism and oxidative stress to selectively drive cancer cell death. Earlier studies with SM-88 therapy demonstrated its potential efficacy in breast and other metastatic cancers. In this current report, we assessed the efficacy of SM-88 in patients with metastatic breast cancer from the first in human “Phase 1” and compassionate use programs from 2012 to 2017. Data demonstrated the potential efficacy of SM-88 in metastatic breast cancer with favorable safety and quality of life profiles. In addition, there were no indications of cross-resistance based on hormone profile, previous treatments or metastatic site. This is an extremely important finding since most cancer deaths are due to resistance to subsequent therapies.  As predicted by the SM-88 mechanism of action, we could not detect this problem with SM-88 use. (more…)
ASCO, Author Interviews, Dermatology, Melanoma, Merck, University of Pittsburgh / 05.06.2018

MedicalResearch.com Interview with: Dr. Diwakar Davar, MD Assistant Professor of Medicine Division of Hematology/Oncology University of Pittsburgh  MedicalResearch.com: What is the background for this study? What are the main findings? Response: The optimal surveillance strategy to detect recurrence in cutaneous melanoma remains elusive. Risk of recurrence increases with higher stage, and is especially high for patients with stage IIIC disease. Although consensus guidelines agree on surveillance imaging for high-risk (stage IIB-IIIC) MEL, there is no consensus regarding optimal frequency/modality in these patients. NCCN guidelines suggest chest radiography (CXR) at 6- to 12-month intervals for stage IA-IIA melanoma  patients; although this is controversial. There exists a great deal of practice variation in the surveillance of these patients. (more…)
ASCO, Author Interviews, Cancer Research, Hematology / 05.06.2018

MedicalResearch.com Interview with: PharmaMarDr. Javier Gómez García Senior Manager. Biostatistics and Data Management PharmaMar Madrid Area, Spain MedicalResearch.com: What is the background for this study? What are the main findings? Response: Multiple myeloma accounts for approximately 1 percent of all cancers and slightly more than 10 percent of all hematologic malignancies. At present, more than 80,000 new cases are reported worldwide each year, and the disease prevalence is increasing. Plitidepsin is a synthetic cyclic depsipeptide isolated from the marine tunicate Aplidium albicans targeting the proto-oncogene eEF1A2, which is over-expressed in multiple myeloma cells. In ADMYRE trial 255 relapsed and refractory multiple myeloma patients with at least three but not more than six prior regimens, including at least bortezomib and lenalidomide/thalidomide, were randomized at 2:1 ratio to receive plitidepsin 5 mg/m2 D1 and 15 plus DXM 40 mg D1, 8, 15 and 22 (P+DXM), or DXM 40 mg D1,8,15 and 22 (DXM) every four weeks. P+DXM met the primary endpoint, progression-free survival (PFS) assessed by an Independent Review Committee, showing a 35% risk reduction in the probability of progression or death. Indeed, a 20% risk reduction in the probability of death was also observed in spite of the fact that 44% of patients from control arm (DXM) switched to P+DXM arm after progression. Therefore, survival in the control arm might have been extended by the effect of plitidepsin, increasing the post progression survival and overestimating the survival observed in the control arm and consequently underestimating the actual difference between treatment arms. Several methods were used to assess the impact of crossover and the robustness of the results even when penalizations were applied; PharmaMar believes that strong evidence in terms of survival benefit in favour of P+DXM has been established.  (more…)
Author Interviews, Boehringer Ingelheim, Pulmonary Disease / 05.06.2018

MedicalResearch.com Interview with: Christopher J. Ryerson, M.D. Assistant Professor Centre for Heart Lung Innovation University of British Columbia Vancouver, Canada MedicalResearch.com: What is the background for this study? What are the main findings? Response: A new Idiopathic pulmonary fibrosis (IPF) mortality analysis presented at the American Thoracic Society’s 2018 annual conference suggests that treatment with nintedanib may be associated with reduced risk of death in patients with the rare lung disease idiopathic pulmonary fibrosis (IPF). Pooled data from the two Phase II INPULSIS trials and the Phase II TOMORROW study compared the number of deaths observed versus the number predicted based on GAP stage over one year. GAP stage is used to predict IPF prognosis and is based on gender, age and lung function (as measured by forced vital capacity [FVC] decline predicted and DLco % predicted). Higher stages of GAP are associated with an increased risk of death. Across the population in the analysis (n=1,228), there were fewer deaths observed in each treatment group than predicted based on GAP stage at baseline (nintedanib: 42 vs. 89.9; placebo: 41 vs. 64.2). In the treated group, the number of observed deaths was 46.7% of the number predicted based on GAP stage, while in the placebo group the number of observed deaths was 63.9% of the number predicted. Based on these observations, the analysis suggests that nintedanib may be associated with a 26.8% relative reduction in the risk of death compared with placebo over one year.  (more…)
Author Interviews, Breast Cancer, Chemotherapy, JAMA / 05.06.2018

MedicalResearch.com Interview with: Dr. Guy Jerusalem, MD, PhD CHU Sart Tilman Liege and Liege University Liege, Belgium MedicalResearch.com: What is the background for this study? What are the main findings? Response: BOLERO-6 was conducted to fulfill postapproval regulatory commitments to the FDA and EMA to estimate treatment benefit with EVE + EXE vs EVE alone or CAP for ER+, HER2− ABC that had progressed on an NSAI. Everolimus plus exemestane has not previously been compared with everolimus alone or capecitabine in a randomized setting.Data describing everolimus alone are limited to a single phase 2 study of just 19 patients. Thus, the FDA deemed it important to ascertain the efficacy of everolimus alone for ER+ breast cancer, and to determine the contribution of exemestane to combination therapy with everolimus. Capecitabine is often the first chemotherapeutic agent given for ER+ breast cancer that has progressed on anti-estrogen therapy. It has a reported PFS of 4.1–7.9 months among patients with HER2-negative advanced breast cancer. However, it has a different safety profile to everolimus or exemestane, and a comparison of endocrine-based combination therapy with single-agent chemotherapy was yet to be conducted. The median PFS with EVE + EXE (8.4 months) was consistent with BOLERO-2 (7.8 months), and compared to EVE alone here (6.8 months) corresponded to an estimated 26% reduction of risk of disease progression or death (HR 0.74). A numerical median PFS difference was observed for CAP over EVE + EXE (9.6 vs 8.4 months), which may be attributed to various baseline characteristics favoring CAP and potential informative censoring. The median PFS with capacitabine was longer than expected based on previous trials. Interpretation of the results of BOLERO-6 must consider the limited sample size and open-label design.  (more…)
Author Interviews, Pain Research, Pharmaceutical Companies / 04.06.2018

MedicalResearch.com Interview with: Zosano Pharma Dr. Peter Schmidt, MD, MSc Senior Director, medical Affairs and Clinical Development Zosano Pharma   MedicalResearch.com: What is the background for this study? What are the main findings? Response: This was a post-hoc analysis of Zosano’s pivotal efficacy trial using its adhesive dermally-applied microarray (ADAM) zolmitriptan formulation, M207. The trial found that M207 was effective versus placebo for the co-primary endpoints of pain freedom and most bothersome symptom (MBS) freedom, both at two hours. The MBS endpoint was just ratified as a new endpoint in the FDA’s February 2018 guidance for acute migraine trials. The stated aim of this new endpoint is “…to better align the study outcome with the symptom(s) of primary importance to patients…” This is logical, as a given migraine patient may not experience all four previous symptom endpoints (pain, photophobia, phonophobia, nausea). (more…)
Author Interviews, Education, JAMA / 04.06.2018

MedicalResearch.com Interview with: Kathleen M. Finn MD, MPhil Christiana Iyasere MD, MBA Division of General Internal Medicine Department of Medicine Massachusetts General Hospital, Boston MedicalResearch.com: What is the background for this study? Response: While the relationship between resident work hours and patient safety has been extensively studied, little research evaluates the role of attending supervision on patient safety. Beginning with the Bell Commission there have been increased calls for enhanced resident supervision due to patient safety concerns. At the same time, with the growth of the hospitalist movement more faculty physicians join daily resident work rounds under the assumption that increased supervision is better for patient safety and resident education. However, we know that supervision is a complex balancing act, so we wanted to study whether these assumptions were true. On the one hand patient safety is important, but on the other adult learning theory argues residents need to be challenged to work beyond their comfort level. Importantly, being pushed beyond your comfort level often requires appropriate space between teacher and learner. To investigate the role of attending supervision on patient safety and resident learning we studied the impact of two levels of physician supervision on an inpatient general medical team. Twenty-two teaching faculty were randomized to either direct supervision of resident teams for patients previously known to the team vs usual care where they did not join rounds but rather discussed the patients later with the team. Faculty participated in both arms of the study, after completing the first arm they then crossed over to the other arm; each faculty member participated in the study for a total of 4 weeks. (more…)
Author Interviews, Eating Disorders, Weight Research / 04.06.2018

MedicalResearch.com Interview with: Tomoko Udo, PhD Assistant Professor Department of Health Policy, Management, and Behavior School of Public Health University at Albany, State University of New York MedicalResearch.com: What is the background for this study?   Response: The National Epidemiologic Survey on Alcohol and Related Conditions III (NESARC III) was the largest epidemiological study on psychiatric disorders in US non-institutionalized adults that was conducted by the National Institution on Alcohol Abuse and Alcoholism, and the first one sinceDSM-5 came out. The last population-based study with US adults that examined eating disorders was the National Comorbidity Survey-Replication Study conducted by Hudson and his colleagues and published in 2007. We felt that it was important to obtain new prevalence estimates in a larger and representative sample especially because the DSM-5 included several changes to the criteria for eating disorders from the earlier DSM-IV. Thus, we thought it was important to provide updated and new prevalence estimates for eating disorders as well as how they are distributed across sex, ethnicity/race, and age.  Many  researchers and clinicians expected higher estimates than earlier studies as a result of “loosening” of diagnostic criteria for eating disorders. (more…)
ASCO, Author Interviews, Pancreatic / 04.06.2018

MedicalResearch.com Interview with: Dr. Marcus Smith Noel, MD University of Rochester James P. Wilmot Cancer Institute Strong Memorial Hospital MedicalResearch.com: What is the background for this study? What are the main findings? Response: Pancreatic cancer outcomes are poor even despite improvement in the overall prognosis for many cancers. Early detection of pancreatic cancer is uncommon because early stage pancreatic cancer often has few symptoms. Unfortunately, most cases are diagnosed at more advanced stages, which is in part why the disease is so lethal. Current standard of care treatments are highly toxic and not effective long-term, as about 90% of patients diagnosed with advanced or metastatic pancreatic cancer do not survive a year. SM-88 is a relatively non-toxic novel combination therapy designed to utilize cellular metabolism and oxidative stress to drive cancer cell death. This therapy has previously demonstrated activity in various metastatic cancers, such as pancreatic cancer, and is currently being evaluated in an ongoing Phase II trial for metastatic pancreatic cancer. This study is a trial in progress report of Tyme’s Phase II trial in patients with metastatic cancer. The Phase II trial is designed as an open-label, multi-center study of SM-88 in patients with metastatic pancreatic cancer who have failed at least one prior line of therapy. In the first stage of the trial, 36 patients will be randomized 1:1 to receive a dose of either a currently utilized active regimen or a double dose per day of SM-88. Primary endpoints are overall response rate (ORR) and overall survival (OS). Secondary endpoints include progression-free survival (PFS), disease control rate, duration of response and time to subsequent treatment. The purpose of the first stage of the study is to analyze the safety, efficacy and pharmacokinetics of SM-88 in patients.  The selected dose of SM-88 will be continued into the second stage of the trial for approximately 81 additional patients. (more…)