ALS, Alzheimer's - Dementia, Author Interviews, Biomarkers, JAMA, Multiple Sclerosis / 27.06.2019

MedicalResearch.com Interview with: [caption id="attachment_50018" align="alignleft" width="200"]Charlotte E. Teunissen, Prof. Teunissen[/caption] Charlotte E. Teunissen, PhD Neurochemistry Laboratory, Department of Clinical Chemistry VU University Medical Centre, Neuroscience Campus Amsterdam Amsterdam, the Netherlands MedicalResearch.com: What is the background for this study? What are the main findings? Response: Several reports have shown increased in NfL in various neurological disorders, separately. We wanted to know how the levels are in these disorders relative to each other. Moreover, some reports showed absence of age effects in Multiple Sclerosis (MS) patients, which is normally present in controls. So, we thought that it would be good to study age effects in a large group of controls, and if these effects are absent in other diseases, similarly as in MS.
Author Interviews, Epilepsy, FDA / 29.05.2019

MedicalResearch.com Interview with: [caption id="attachment_49403" align="alignleft" width="200"]Dr. Steven S. Chung, MDExecutive Director and Program ChairNeuroscience Institute and Director of the Epilepsy ProgramBanner – University Medical Center Dr. Chung[/caption] 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.  
Author Interviews, Hospital Readmissions, JAMA, Neurology, Outcomes & Safety, University of Pennsylvania / 20.05.2019

MedicalResearch.com Interview with: [caption id="attachment_49220" align="alignleft" width="180"]Sameed Khatana, MDFellow, Cardiovascular Medicine, Perleman School of MedicineAssociate Fellow, Leonard Davis Institute of Health EconomicsUniversity of Pennsylvania Dr. Khatana[/caption] Sameed Khatana, MD Fellow, Cardiovascular Medicine, Perleman School of Medicine Associate Fellow, Leonard Davis Institute of Health Economics University of Pennsylvania MedicalResearch.com: What is the background for this study? Response: There has been a growing use of quality metrics and indices in the US healthcare system. Much attention has been paid to quality measurement programs used by public payors, however, the use of such programs by commercial payors is much less studied. "Centers of excellence" are one type of quality designation program that is growing in use by commercial payors where certain hospitals are determined to be "high quality" for a certain disease state or procedure based on meeting certain criteria. For some people, this is even impacting the choice of providers and hospitals they can use by payors. We evaluated centers of excellence programs from three large commercial payors, Aetna, Cigna and Blue Cross Blue Shield, targeted at cardiovascular diseases and interventions and examined publicly reported outcomes for all hospitals performing percutaneous coronary interventions (cardiac stenting) in New York State. 
Author Interviews, Education, Neurology, NYU/NYMC / 17.05.2019

MedicalResearch.com Interview with: Dr. Rebecca Stainman Dr. Arielle Kurzweil MD Adult Neurology Program Director New York University School of Medicine NYU Langone Health MedicalResearch.com: What is the background for this study? What are the main findings?  Response: Physician burnout is prevalent. Neurologists have among the highest burnout rates, ranked third among specialties in a 2011 study, and over half of US Neurologists report at least 1 symptom of burnout in a 2016 survey.  Efforts to address burnout in training programs have mostly been aimed at implementing wellness curricula and offering mental health resources. Training neurology residents to effectively identify, address, and help impaired colleagues is equally crucial in these efforts, yet there is a paucity of literature on this topic. We used simulation as a means of addressing this topic, via identifying and addressing an impaired colleague through an objective structured clinical examination (OSCE). 
Author Interviews, Brain Injury, Neurological Disorders, Neurology, Stroke / 12.05.2019

MedicalResearch.com Interview with: [caption id="attachment_49129" align="alignleft" width="128"]Thomas M Van Vleet PhDPosit Science  Dr. Van Vleet[/caption] Thomas M Van Vleet PhD Posit Science  Dr. Tom Van Vleet,  presented results on a common symptom of stroke and acquired brain injury (hemi-spatial neglect) at the American Academy of Neurology May 2019 MedicalResearch.com: What makes this study newsworthy? Response For the first time ever a highly-scalable intervention — computerized brain training (BrainHQ made by Posit Science) —was found to improve symptoms of hemi-spatial neglect, which is a common and often intractable and debilitating problem after stroke or other acquired brain injury. MedicalResearch.com: What can you tell us about the medical condition (hemi-spatial neglect) investigated in this study? Response About a third of patients with a brain injury exhibit a complex and debilitating array of neurological deficits known as the “neglect syndrome” (sometimes called, “hemi-spatial neglect” or “neglect”). The most apparent symptom of neglect is the inability of patients to efficiently process information on the side of space opposite the injury; often completely missing relevant events without awareness. As a result, patients often fail to adopt compensatory strategies or respond to other conventional rehabilitation protocols. The cost is significant, as patients with neglect experience longer hospital stays and have higher requirements for assistance, including greater skilled nursing home placements relative to patients with similar extent of brain injury without neglect. To date, there’s been no broadly-applicable and highly-scalable intervention for addressing neglect. An alarming reality given the increasing cost of stroke, which is currently estimated to exceed $34 billion per annum
Author Interviews / 07.05.2019

MedicalResearch.com Interview with: [caption id="attachment_49033" align="alignleft" width="200"]Lana Zhovtis Ryserson, MDAssistant Professor, Department of NeurologyNYU Langone Health Dr. Zhovtis Ryserson[/caption] Lana Zhovtis Ryserson, MD Assistant Professor, Department of Neurology NYU Langone Health MedicalResearch.com: What is the background for this study? What are the main findings? Response: Natalizumab is an effective therapy of relapsing remitting multiple sclerosis dosed at 300mg every 4 weeks. However it is associated with a potentially deadly infection - progressive multifocal leukoencephalopathy. In order to mitigate this risk, clinicians have adopted an approach of infusing the medication less frequently, a strategy which has become known as Extended Interval Dosing (EID). The TOUCH database is US mandatated risk evaluation and mitigation program which is the largest database available to assess PML risk for patients on EID schedule. Previous analysis of this database in 2017, showed a significant risk reduction of PML in patients utilizing extended interval dosing schedule. The aim of the current study was to update on this analysis with another year of data.
Author Interviews, Epilepsy, Genetic Research, JAMA, Pediatrics / 12.04.2019

MedicalResearch.com Interview with: [caption id="attachment_48481" align="alignleft" width="200"]Dr. Ahmad Abou Tayoun, PhDClinical Molecular GeneticistDirector of the Genetics LaboratoryAl Jalila Children’sUnited Arab Emirates Dr. Abou Tayoun[/caption] Dr. Ahmad Abou Tayoun, PhD Clinical Molecular Geneticist Director of the Genetics Laboratory Al Jalila Children’s United Arab Emirates MedicalResearch.com: What is the background for this study?   Response: In this study, we provide data in favor of using an exome-based testing approach, where parental samples can be readily accessible, for early onset epilepsy patients. The exome test includes all coding genes in the human genome. Although we perform exome sequencing on those patients, we demonstrate that a first tier analysis should include targeted interpretation of ~100 genes strongly associated with the disease. This analysis provides diagnoses in ~11% of the patients. Follow up parental testing on a limited number of patients (n=15) that had inconclusive results, revealed de novo (new mutations) variant status, leading to upgrade to positive reports in 7 patients and adding ~5% to the overall diagnostic yield.
Author Interviews, JAMA, Neurology, Outcomes & Safety, Parkinson's, Pharmacology, University of Pennsylvania / 04.10.2018

MedicalResearch.com Interview with: [caption id="attachment_45003" align="alignleft" width="148"]Allison W. Willis, MD, MS Assistant Professor of Neurology Assistant Professor of Biostatistics and Epidemiology Senior Fellow, Leonard Davis Institute Senior Scholar, Center for Clinical Epidemiology and Biostatistics University of Pennsylvania School of Medicine Dr. Willis[/caption] Allison W. Willis, MD, MS Assistant Professor of Neurology Assistant Professor of Biostatistics and Epidemiology Senior Fellow, Leonard Davis Institute Senior Scholar, Center for Clinical Epidemiology and Biostatistics University of Pennsylvania School of Medicine MedicalResearch.com: What is the background for this study? What are the main findings? Response: This study was motivated by my own experiences as a neurologist-neuroscientist. I care for Parkinson disease patients, and over the year, have had numerous instances in which a person was taking a medication that could interact with their Parkinson disease medications, or could worsen their PD symptoms.
ALS, Author Interviews, Pharmaceutical Companies / 27.09.2018

MedicalResearch.com Interview with: RetrotopeRobert J Molinari, Ph.D. President and CEO Retrotope, Inc.   MedicalResearch.com: What is the background for this study? How does RT001 differ from other treatments for neurodegenerative diseases?  Response: Lipid peroxidation in critical cell and mitochondrial membranes represents a common pathway of cell death in many neurodegenerative diseases, regardless of the initiating trigger of original damage, e.g. aberrant gene expression, misfolded proteins such as tau and amyloid, environmental insults, etc. This hypothesis was supported by work showing that stabilizing lipids (using virtually indistinguishable isotopic variants of the original dietary molecules) was able to mitigate disease-related cell dysfunction, and reverse disease in a variety of animal models, and more recently, in human patients enrolled in clinical trials. It has been known for decades that lipid peroxidation detritus of oxidized membrane fats are present in most all diseases of degeneration and aging, including Alzheimer’s disease, Parkinson’s disease, atherosclerosis, ALS, Huntington’s, and fatal inborn genetic errors resulting in neurodegenerative diseases (among others). The hypothesis that controlling such oxidation could provide therapeutic benefit was abandoned when numerous formal trials of classical antioxidants, e.g. Co-enzyme Q, Vitamin E, and others failed to provide meaningful benefit. We believed that the antioxidant mechanism used to attempt control of the membrane oxidation was flawed, but that the target itself was correct. Indeed, by using a new class of oral drugs that are lipids fortified against damage at the key susceptible bonds, we observed reduction in lipid peroxidation damage that halted, and even reversed neurodegenerative disease progression. Dosed in amounts and forms similar to omega 3 and 6 supplements, these drugs exhibited profound disease modification across a broad range of diseases in animal models, placebo controlled- and open label- human trials.
Author Interviews, Cannabis, Neurology, University Texas / 17.09.2018

MedicalResearch.com Interview with: [caption id="attachment_44588" align="alignleft" width="149"]Dr. Francesca M. Filbey PhD Professor Program Head, Cognition and Neuroscience PhD Bert Moore Chair in BrainHealth UT Dallas Dr. Filbey[/caption] Dr. Francesca M. Filbey PhD Professor Program Head, Cognition and Neuroscience PhD Bert Moore Chair in BrainHealth UT Dallas MedicalResearch.com: What is the background for this study? What are the main findings? Response: The cannabis literature has generally focused on changes in brain function when engaged in a task. We were interested in examining whether these differences are present when not engaged in a task (i.e., during resting state) to understand baseline functional organization of the brain. Changes to baseline functional organization may reflect changes in brain networks underlying cognition. We also wanted to investigate whether specific brain waves, as measured by electroencephalography (EEG), are associated with measures of cannabis use, such as craving.
Author Interviews, Kidney Disease, Neurology, Sleep Disorders / 01.09.2018

MedicalResearch.com Interview with: [caption id="attachment_44254" align="alignleft" width="80"]Rachel Marie E. Salas, MD, MEHP, FAAN Associate Professor, Neurology and Nursing at Johns Hopkins Medicine Director, Interprofessional Education and Interprofessional Collaborative Practice Director, Neurology Clerkship Director, PreDoc Program Meyer/Neuro Sleep Baltimore, MD Dr Salas[/caption] Rachel Marie E. Salas, MD, MEHP, FAAN Associate Professor, Neurology and Nursing at Johns Hopkins Medicine Director, Interprofessional Education and Interprofessional Collaborative Practice Director, Neurology Clerkship Director, PreDoc Program Meyer/Neuro Sleep Baltimore, MD MedicalResearch.com: What is the background for this study? Can you briefly describe what is meant by RLS  and who suffers from it? Response: Restless Legs Syndrome (RLS) is a common neurological disorder characterized by an irritating, overwhelming urge to move (akathisia) the legs while at rest or sleep (conditions of diminished arousal), which almost immediately abates with mental or physical activity (conditions of maintained arousal). One of the most clinically-profound and scientifically relevant consequences of this disease process is an increased arousal state producing significant wake during sleep times and a relative sustainable degree of daytime alertness despite the degree of diseased-imposed sleep loss. The focus of most previous RLS research has been on the (limb) akathisia with associated periodic movements and reduction of these with dopaminergic treatment. Little research has been done to understand the broader biological dimensions​ of RLS. Patients with RLS have altered sleep-wake homeostasis with increased arousal and wakefulness (hyperarousal) not only driving the signature clinical symptoms (“the urge to move” and sleep loss) but also supporting arousal over sleep drive at night and in the day. We hypothesize that there is a basic glutamate-hyperarousal process producing both disrupted sleep (increased wake time) and cortical excitability (as demonstrated by transcranial magnetic stimulation (TMS)).​ 
Author Interviews, Neurology / 24.08.2018

MedicalResearch.com Interview with: MedicalResearch.comCaroline Schlüter, M.Sc. Psychologie Fakultät für Psychologie AE Biopsychologie Ruhr-Universität Bochum MedicalResearch.com: What is the background for this study? What are the main findings? Response: Individuals differ in their ability to initiate intended actions. While some people tend to put tasks off, others easily manage to tackle them directly. Although interindividual differences in what we call ‘action control’ make a major contribution to our everyday life by affecting our physical and mental health as well as our academic and occupational performance, their neural foundation was mostly unknown. Our study is the first to use both structural and functional neuroimaging methods to investigate the neural correlates of action control. We were able to show that poorer action control is significantly related to greater amygdala volume. The amygdala is considered to be a neuroanatomical hub for fear-motivated behavior. It processes sensory information in order to evaluate a given situation, behaviour or outcome. Hence, it is conceivable that individuals with a larger amygdala tend to evaluate future actions and their possible consequences more extensively. This, in turn, might lead to greater concern and hesitation, as observed in individuals with poorer action control. Further, we were able to show that weaker functional resting-state connectivity between the amygdala and the dorsal anterior cingulate cortex (dACC) is significantly associated with lower action control scores, which are typical for procrastinators. Previous studies indicate that the dACC has reciprocal connections with the amygdala, playing a significant role in purposive behaviour and self-control mechanisms. Thus, a weaker functional connection between both brain areas might hinder successful action control, as interfering negative emotions and 
Author Interviews, Duke, Genetic Research, Neurology, Pediatrics / 30.07.2018

MedicalResearch.com Interview with:  [caption id="attachment_43604" align="alignleft" width="128"]Paul C Marcogliese, Ph.D. Postdoctoral Associate, Laboratory of Dr. Hugo Bellen Department of Molecular and Human Genetics Baylor College of Medicine Houston, Texas 77030 Dr. Marcogliese[/caption] Paul C Marcogliese, Ph.D. Postdoctoral Associate, Laboratory of Dr. Hugo Bellen Department of Molecular and Human Genetics Baylor College of Medicine Houston, Texas 77030 [caption id="attachment_43603" align="alignleft" width="99"]Loren D. Pena, MD PhD Pediatric Medical Genetics Specialist Division of Medical Genetics, Department of Pediatrics Duke University School of Medicine, Durham, NC Dr. Peña[/caption] Loren D. Pena, MD PhD Division of Human Genetics Cincinnati Children's Hospital Medical Center Department of Pediatrics University of Cincinnati Cincinnati, OH 45229 MedicalResearch.com: What is the background for this study? What are the main findings? Response: The Undiagnosed Diseases Network (UDN) is a multi-site collaboration across the US that seeks to help diagnose patients with rare disorders that are ill-defined. Dr. Loren D.M. Pena and Dr. Vandana Shashi at the Duke-Columbia clinical site of the UDN had seen a patient with a severe neurological disorder. While the patient had no symptoms at birth, the patient began falling at about 3 years of age, eventually losing motor coordination and developing seizures. In the interim, the regression has progressed to a severely debilitating state. Re-analysis of the participant’s exome data by our site bioinformatician at Columbia (Nicholas Stong) in Dr. David Goldstein’s laboratory revealed a truncating variant in the single exon gene IRF2BPL that could be the candidate disease-causing gene. The UDN clinicians at Duke then contacted the UDN Model Organism Screening Center (MOSC) led by Dr. Hugo Bellen at Baylor College of Medicine and the Howard Hughes Medical Institute for functional analysis. In parallel, four more patients were found with truncating mutations causing a similar disorder though the UDN and GeneMatcher.org. Additionally, two patients with missense variants in IRF2BPL were identified that displayed seizures and some developmental delay or autism spectrum disorder but no motor regression. Work in MOSC by Dr. Paul Marcogliese using fruit flies revealed that the IRF2BPL truncating variants are severe loss of function mutations and one of the missense variants was a partial loss of function. Additionally, it was found that the fruit fly IRF2BPL gene, called pits, is expressed in the neurons of the adult fly brain. Lowering the levels of pits by about 50% in fly neurons leads to progressive behavioural abnormalities and neurodegeneration. By combining the human genetics, bioinformatics and model organism data, IRF2BPL was found to be a novel disease-causing gene in humans.
Author Interviews, JAMA, Neurology, Surgical Research / 30.07.2018

MedicalResearch.com Interview with: Jianning Zhang MD, Ph.D Chairman, II, VII Chinese Medical Association of Neurosurgery President, Tianjin Medical University General Hospital, China  Jianning Zhang MD, Ph.D Chairman, II, VII Chinese Medical Association of Neurosurgery President, Tianjin Medical University General Hospital, China   MedicalResearch.com: What is the background for this study? Response: The elderly population is growing dramatically world widely, especially in China. The incidence of chronic subdural hematoma has been rising over the past years. Although the surgery is not a difficult process, the risk of death and recurrence persist, and the affliction and economic expenditure of the patients are relatively higher in the elderly. For these reasons, it is urgent to develop novel pharmacological therapies with sufficient safety and efficacy.  It has been known that the high expression of VEGF and inflammatory factors in chronic subdural hematoma can lead to abundant angiogenesis of immature vessels on the wall of hematoma. In our previous study, patients with chronic subdural hematoma have impaired ability to promote vascular maturation. For example, the number of endothelial progenitor cells in circulating blood is about 67% of the healthy individuals with similar age.  Atorvastatin can mobilize endothelial progenitor cells to reduce inflammation. It increases the number of circulating endothelial cells that are inversely correlated with the volume of hematoma. We have demonstrated that atorvastatin can promote endothelial cell formation and reduce the leakage of endothelial cell barrier in vitro. Results from in vivo experiments in animal models of subdural hematoma suggest that atorvastatin can promote the maturation of blood vessels and reduce inflammation on the margin of hematoma, and thus improve the neurological outcome.
Author Interviews, Duke, JAMA, Schizophrenia / 26.07.2018

MedicalResearch.com Interview with: [caption id="attachment_43484" align="alignleft" width="199"]Richard Keefe PhD Professor in Psychiatry and Behavioral Sciences Duke Institute for Brains Sciences Dr. Keefe[/caption] Richard Keefe PhD Professor in Psychiatry and Behavioral Sciences Duke Institute for Brains Sciences MedicalResearch.com: What is the background for this study? What are the main findings? Response: A lot of studies have shown that cognitive deficits are present in young people at risk for psychosis. There have been calls for investigations of the idea that cognition declines over time in the young people who are at highest risk, but longitudinal studies are hard to conduct so not much work has been done to address this question. The main finding from our study is that the cognitive architecture – the way the various aspects of cognitive functioning appear to be organized in each individual’s brain based upon their pattern of performance – changes over time in those young people who are in the midst of developing psychosis. Interestingly, cognitive architecture also becomes more disorganized in those whose high-risk symptoms do not remit over a two year period, and is related to the functional difficulties they may be having. The young people whose high risk symptoms were present at the beginning of the study but remitted later actually improved cognitively over the two year period of the study.
Author Interviews, Neurological Disorders, Neurology, Personalized Medicine, Radiology, Surgical Research / 13.07.2018

MedicalResearch.com Interview with: [caption id="attachment_43125" align="alignleft" width="139"]Yasser Iturria-Medina, PhD Primary Investigator, Ludmer Centre for Neuroinformatics & Mental Health Assistant Professor, Department of Neurology and Neurosurgery Faculty of Medicine McGill University Dr. turria-Medina[/caption] Yasser Iturria-Medina, PhD Primary Investigator, Ludmer Centre for Neuroinformatics & Mental Health Assistant Professor, Department of Neurology and Neurosurgery Faculty of Medicine McGill University MedicalResearch.com: What is the background for this study? What are the main findings? Response: There are millions of patients following therapeutic interventions that will not benefit them. In this study, we aimed to illustrate that it is possible to identify the most beneficial intervention for each patient, in correspondence with the principles of the personalized medicine (PM). Our results show that using multimodal imaging and computational models it is possible to predict individualized therapeutic needs. The predictions are in correspondence with the individual molecular properties, which validate our findings and the used computational techniques. The results highly also the imprecision of the traditional clinical evaluations and categories for understanding the individual therapeutic needs, evidencing the positive impact that would have to use multimodal data and data-driven techniques in the clinic, in addition to the medical doctor's criterion/evaluations.  
Author Interviews / 12.07.2018

MedicalResearch.com Interview with: [caption id="attachment_43109" align="alignleft" width="200"]Dr-James Beck Dr. Beck[/caption] James Beck, Ph.D., Chief Scientific Officer Adjunct Associate Professor Department of Neuroscience and Physiology New York University Langone School of Medicine MedicalResearch.com: What is the background for this study? What are the main findings? Response: The most frequently cited study for prevalence in the US was based on a door-to-door survey conducted in 1978 in a rural county in Mississippi.  Only 26 cases of Parkinson’s disease (PD) were identified.  That has been extrapolated to our current US population of 330,000,000 people.  To give a sense of how long ago that was, Microsoft was considered a startup company.  Therefore, to provide an improved estimate of who has Parkinson’s disease, the Parkinson’s Foundation lead the Parkinson’s Prevalence study to do just that, using datasets that were from more geographically and ethnically diverse communities that can better reflect the US population as a whole. The main finding is that we know now that there are nearly 1,000,000 people living with PD – and that number is expected to increase dramatically as our population ages. (The biggest risk factor for Parkinson’s disease is age.)
Author Interviews, Neurology / 05.07.2018

MedicalResearch.com Interview with: Prof. David Adams Head of the French National Reference Centre for Familial Amyloidotic Polyneuropathy (NNERF)/APHP/INSERM Paris FranceProf. David Adams Head of the French National Reference Centre for Familial Amyloidotic Polyneuropathy (NNERF)/APHP/INSERM Paris France MedicalResearch.com:  What is the background for this study? Response: Hereditary transthyretin amyloidosis is an autosomal dominant, multisystemic, progressive, life-threatening disease caused by mutations in the gene encoding transthyretin (TTR ). The liver is the primary source of circulating tetrameric TTR protein. In hereditary transthyretin amyloidosis, both mutant and wild-type transthyretin deposit as amyloid in peripheral nerves and the heart, kidney, resulting in polyneuropathy and cardiomyopathy. Neuropathic changes result in profound sensorimotor disturbances, with deterioration in activities of daily living and ambulation, hypotension, diarrhea, impotence, and bladder disturbances. Until now,  only few patients have access to anti-amyloid therapy : Liver Transplantation or TTR stabilizers which are able only to slow progression of the disease at very early stage of the disease. Patisiran, an investigational RNA interference therapeutic agent, specifically inhibits hepatic synthesis of transthyretin and is specifically addressed to the liver by lipid nanoparticle (LNP) formulation. This study carried out a multicenter, international, randomized, double-blind, placebo-controlled, phase 3 trial of patisiran in patients with hereditary transthyretin amyloidosis with polyneuropathy.
Alzheimer's - Dementia, Author Interviews, JAMA, Neurology / 25.06.2018

MedicalResearch.com Interview with: [caption id="attachment_42615" align="alignleft" width="152"]Dr. Paul Foster Dr. Foster[/caption] Paul Foster BMedSci(Hons) BMBS PhD FRCS(Ed) FRCOphth FRCS(Eng) Professor of Glaucoma Studies & Ophthalmic Epidemiology Research Theme Lead Integrative Epidemiology & Visual Function UCL Institute of Ophthalmology & Moorfields Eye Hospital London  MedicalResearch.com: What is the background for this study?  Response:  Dementia is the medical challenge of the moment – increasingly common, adversely impacting quality of life for millions, and a great worry for all. Efforts to identify treatments or interventions rely on being able to identify those people at greatest risk. Our motivation was to help identify those people, primarily to aid in the development of treatments through clinical trials.
Author Interviews, Kidney Disease, Neurological Disorders, Pain Research, UCSF / 06.06.2018

MedicalResearch.com Interview with: [caption id="attachment_42216" align="alignleft" width="150"]Dr. Julie H. Ishida MD Department of Medicine, Division of Nephrology University of California, San Francisco and San Francisco Veterans Affairs Medical Center Dr. Ishida[/caption] 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. 
Author Interviews, CDC, JAMA, Neurological Disorders, Zika / 31.05.2018

MedicalResearch.com Interview with: [caption id="attachment_41893" align="alignleft" width="125"]Dr. Emilio Dirlikov, PhD Office of Epidemiology and Research, Puerto Rico Department of Health Epidemic Intelligence Service Division of Scientific Education and Professional Development CDC Dr. Dirlikov[/caption] Dr. Emilio Dirlikov, PhD Office of Epidemiology and Research, Puerto Rico Department of Health Epidemic Intelligence Service Division of Scientific Education and Professional Development CDC MedicalResearch.com: What is the background for this study? What are the main findings? Response: After reporting local Zika transmission in December 2015, the Puerto Rico Department of Health (PRDH), US Centers for Disease Control and Prevention (CDC), and University of Puerto Rico began identifying cases of Guillain-Barré syndrome (GBS), testing specimens, and conducting follow-up telephone interviews after patients left the hospital. Through these efforts, we were able to characterize acute clinical features and long-term disability of GBS associated with Zika infection by analyzing data from GBS patients with and without evidence of Zika infection. This investigation increases scientific and medical understanding of Guillain-Barré syndrome following Zika infection, provides insight into the disease processes involved in GBS following Zika infection, and adds to growing evidence of a causal association between Zika and GBS. 
Author Interviews, Duke, Mental Health Research / 21.05.2018

MedicalResearch.com Interview with: [caption id="attachment_41880" align="alignleft" width="125"]Maxwell Elliott Clinical psychology PhD student Working with Ahmad Hariri and the Moffitt & Caspi lab Duke University Maxwell Elliott[/caption] Maxwell Elliott Clinical psychology PhD student Working with Ahmad Hariri and the Moffitt & Caspi lab Duke University MedicalResearch.com: What is the background for this study? What are the main findings? Response: The traditional clinical science model identifies individuals who meet specific criteria for mental illness diagnoses (e.g. Depression, Anxiety) and compares them to “healthy” controls to find brain correlates of mental illness.  However, this approach often overlooks the high rates of comorbidity and shared symptamatology across mental illnesses. Emerging research has identified a general factor of psychopathology that accounts for shared risk among internalizing, externalizing, and thought disorders across diverse samples. This general factor of psychopathology has been called the p-factor. In our study we investigate the brain correlates of the p-factor using a data-driven analysis of resting state functional connectivity. We find that higher p-factor scores and associated risk for common mental illness maps onto hyper-connectivity between visual association cortex and both frontoparietal and default mode networks.
Author Interviews, Epilepsy, JAMA / 10.04.2018

MedicalResearch.com Interview with: [caption id="attachment_41045" align="alignleft" width="183"]Dr Hayley Gorton PhD MPharm MRPharmS FHEA Research Associate Centre for Pharmacoepidemiology and Drug Safety Research Division of Pharmacy & Optometry| Faculty of Biology, Medicine & Health University of Manchester Dr. Gorton[/caption] Dr Hayley Gorton PhD MPharm MRPharmS FHEA Research Associate Centre for Pharmacoepidemiology and Drug Safety Research Division of Pharmacy & Optometry| Faculty of Biology, Medicine & Health University of Manchester MedicalResearch.com: What is the background for this study? What are the main findings? Response: It is already known that people with epilepsy are at a higher risk of death than those without epilepsy but we didn’t know much about the risks of different types of death. Unnatural death (mainly accident and suicide) accounts for a very small number of all deaths but, compared to people without epilepsy, people with epilepsy are three times more likely to die by accident and twice as likely to die by suicide. Within these broad categories, persons with epilepsy are five times more likely to die specifically by accidental poisoning with medication, and three times more likely to die by intentionally poisoning themselves with medication. Opioid painkillers and medicines for mental illness were the ones most commonly used in poisoning deaths among people with epilepsy and those without epilepsy. Antiepileptic drugs were taken relatively infrequently-they were involved in about 10% of poisoning deaths in people with epilepsy. 
Alzheimer's - Dementia, Author Interviews, JAMA, Parkinson's / 27.03.2018

MedicalResearch.com Interview with: [caption id="attachment_40768" align="alignleft" width="147"]Benjamin Dawson, B.Sc.  MD Candidate 2020 Benjamin Dawson[/caption] Benjamin Dawson, B.Sc. MD Candidate 2020 MedicalResearch.com: What is the background for this study? What are the main findings? Response: Dementia in Parkinson’s Disease is one of its most feared complications, and may happen eventually to most patients if they reached advanced age. Identifying those at especially high risk of dementia has important potential implications - it would facilitate clinical counselling, it has treatment implications (e.g. knowing a person is likely to get dementia in the near future would probably steer you away from certain medications and towards others).  Most critically, it can help select patients for trials to prevent dementia. While several factors that show high risk for dementia in Parkinson’s disease have previously been described, these have yet to shape patient-care, either because they are not very strong predictors, or they are not user-friendly.  So, we designed a very simple clinical screening tool, called the Montreal Parkinson’s Risk of Dementia Scale (MoPaRDS).  It took predictors of dementia that were established from large-scale studies and boiled them down into a simple 8-point scale that uses information that you can get in a simple office visit.  The 8 predictors were being over 70, being male, having a blood pressure drop with standing, showing early mild cognitive changes, having a symmetric bilateral disease (that is, one side not clearly worse than the other), experiencing falls or freezing, having experienced hallucinations, and having symptoms of REM sleep behavior disorder ('acting out' the dreams at night). When we tested the scale in a combined cohort of 607 patients with Parkinson’s (of whom 70 developed dementia over mean follow-up of 4.4-years) a positive MoPaRDS screen (≥4 out of 8 items) identified 14-fold increased risk of dementia compared to a negative screen. We recommend dividing the scale into three categories; low-, intermediate- and high-risk. Those in the highest score group (MoPaRDS, 6-8) had a 14.9% risk of developing dementia each year, while those with the lowest scores (MoPaRDS, 0-3) had only 0.6% annual risk.  So, these simple measures can be pretty powerful predictors of dementia.
Author Interviews, Lancet, Neurological Disorders, Neurology / 23.03.2018

MedicalResearch.com Interview with: David Birnkrant, MD Professor of Pediatrics, Case Western Reserve University School of Medicine Director of Pediatric Pulmonology & Student Education, MetroHealth Medical Center MedicalResearch.com: What is the background for this study? What are the main findings? Response: This study updates guidance on all aspects of the multi-disciplinary care of patients with Duchenne muscular dystrophy (DMD). The project was funded by the CDC’s National Center on Birth Defects and Developmental Disabilities and the results were recently published as three articles in The Lancet Neurology. The project was guided by a 25-member steering committee. Eleven expert committees worked over a period of three years to develop guidelines based on the RAND/UCLA appropriateness method, in which assessments and interventions were evaluated for appropriateness and necessity. The recommendations update those originally published in 2010. Duchenne muscular dystrophy is transmitted by X-linked recessive inheritance and thus affects primarily boys and men. Patients affected by DMD do not produce functional dystrophin protein, resulting in progressive weakness of skeletal, respiratory, and heart muscles, causing a shortened life span. Teens and young men may require surgery for curvature of the spine, a ventilator device to assist breathing, and a feeding tube to help ensure adequate nutrition. The approach of the various subspecialties involved in DMD management has evolved, with more anticipatory assessment and therapy, identifying and addressing predictable medical complications as early as possible for optimal patient outcomes. With this kind of multi-disciplinary care, people with DMD now live into their 30s and beyond. Along with the emergence of new genetic and molecular therapies, the recognition that people with DMD are living longer was one of the main motivations behind the need for these updated care considerations. Patients with DMD, their families and their advocacy organizations are driving a new emphasis on optimizing quality of life, not just prolongation of survival. Thus, there was a need to address issues related to transitions of care from childhood to adulthood, coordination of care across subspecialties, and other topics related to education, vocation, independence, personal relationships, emotional health, and intimacy. The updated care considerations thus include eleven topic areas, eight of which were part of the 2010 guidelines. These are: (1) diagnosis, (2) neuromuscular management, (3) rehabilitation management, (4) gastrointestinal and nutritional management, (5) respiratory management, (6) cardiac management, (7) orthopedic and surgical management, and (8) psychosocial management. Three topics are new: (9) primary care and emergency management, (10) endocrine management (including growth, puberty, adrenal insufficiency, and bone health), and (11) transitions of care across the lifespan.
Author Interviews, Mayo Clinic, Neurology / 13.02.2018

MedicalResearch.com Interview with: [caption id="attachment_40022" align="alignleft" width="120"]Eoin Flanagan, M.B., B.Ch. Mayo Clinic Rochester, Minnesota Dr. Flanagan[/caption] Eoin Flanagan, M.B., B.Ch. Mayo Clinic Rochester, Minnesota  MedicalResearch.com: What is the background for this study?   Response: Traditionally it has been thought that infections (e.g., viruses)  account for the majority of encephalitis cases. Recent discoveries of neural autoantibody markers of encephalitis (e.g., NMDA receptor autoantibodies) have led to an appreciation that encephalitis cases can be caused by the body’s own immune system attacking the brain, what we term autoimmune encephalitis.
Author Interviews, Genetic Research, Neurology / 25.01.2018

MedicalResearch.com Interview with: Ona Bloom PhD “Duluth Boat Show - Sea Lamprey Booth” by USFWSmidwest is licensed under CC BY 2.0Associate Professor, Center for Autoimmune, Musculoskeletal and Hematopoietic Diseases, The Feinstein Institute for Medical Research Associate Professor, Department of Molecular Medicine, Donald and Barbara Zucker School of Medicine at Hofstra/Northwell MedicalResearch.com: What is the background for this study? What are the main findings? Response: Scientists have known for years that an ancient species of fish called the lamprey has a remarkable ability to rebuild their spinal cord after it’s been severed. After the lamprey spinal cord is cut, they recover from paralysis to fully swimming again in about twelve weeks, without taking any medicines or other treatments. We are studying the lamprey because we want to know the recipe of molecular ingredients that supports successful recovery after spinal cord injury. The genome of this animal was reported about 5 years ago, in a publication led by my colleagues Dr. Jeramiah Smith at the University of Kentucky and Dr. Weiming Li at Michigan State University.  It turns out that many aspects of the lamprey genome are similar to ours, particularly in the central nervous system. Therefore, we think it is a reasonable expectation that what we learn from lamprey could give us some relevant clues about what might be different about the responses in mammals and other animals that are not good at regenerating their spinal cord. In this study, we found that the expression of many genes in the spinal cord and brain of lampreys change during their recovery from spinal cord injury. Some of the genes that get activated are similar to what happens when our peripheral nervous system is injured, which is better at regenerating than the central nervous system. We also identified that a pathway called the Wnt pathway plays an important role in the regeneration and recovery process. This is a large, complex network of genes that are important in many biological processes, from embryological development in fruit flies to cancer in humans.
Author Interviews, Parkinson's / 28.12.2017

MedicalResearch.com Interview with: Dr. Frances M. Weaver PhD Hines VA Hospital Center of Innovation for Complex Chronic Healthcare Hines, IL 60141 MedicalResearch.com: What is the background for this study? What are the main findings? Response: Research has shown that deep brain stimulation (DBS) for Parkinson’s disease (PD) improves motor function and this improvement is sustained. There is also improvement in quality of life after DBS. However, it is not known whether DBS also effects survival. A few studies that have examined survival have had mixed results. In the current study we compared survival for a large cohort of persons with Parkinson’s disease who underwent DBS to a match group of persons with PD who were managed medically. We found a modest improvement in survival for persons with Parkinson’s disease who underwent DBS compared to individuals who did not.
Author Interviews, Cocaine / 27.12.2017

“cocaine photo” by Imagens Evangélicas is licensed under CC BY 2.0MedicalResearch.com Interview with: Mary Kay Lobo, PhD Associate Professor University of Maryland School of Medicine Department of Anatomy and Neurobiology Baltimore, MD 21201  MedicalResearch.com: What is the background for this study? What are the main findings? Response: Altered energy balance has been studied in drug abuse but the fundamental source of energy, mitochondria, has not been well examined.  In this study we found that a molecular regulator of mitochondrial fission (division) is increased in the nucleus accumbens, a major brain reward region, of rodents exposed to repeated cocaine and postmortem samples of cocaine dependent individuals.  We further found that mitochondrial fission is increased in a nucleus accumbens neuron subtype in rodents that self-administer cocaine. Pharmacological blockade of mitochondrial fission can prevent physiological responses to cocaine in this neuron subtype while reducing cocaine-mediated behaviors.  Finally, genetic reduction of mitochondrial fission in this neuron subtype in the nucleus accumbens can reduce drug (cocaine) seeking in rodents previously exposed to cocaine. In contrast, increasing mitochondrial fission, in this neuron subtype, enhances cocaine seeking behavior.
Author Interviews, JAMA, Psychological Science / 18.12.2017

MedicalResearch.com Interview with: [caption id="attachment_38996" align="alignleft" width="100"]Dr. Foltynie Dr. Foltynie[/caption] Thomas Foltynie MD PhD Senior Lecturer and Honorary Consultant Neurologist Unit of Functional Neurosurgery Institute of Neurology and National Hospital for Neurology and Neurosurgery University College London MedicalResearch.com: What is the background for this study? What are the main findings? Response: Stimulation of the Nucleus Basalis of Meynert can enhance cholinergic innervation of the cortex in animal models and has been previously reported to have beneficial cognitive effects in a single patient with Parkinson’s Disease dementia. In this double blind crossover trial, six patients with Parkinson’s Disease underwent low frequency stimulation to the NBM bilaterally.  While there were no consistent objective improvements in cognitive performance, there was a marked reduction in visual hallucinations in two of the participants. .