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, Brain Injury, Neurological Disorders, Neurology, Stroke / 12.05.2019

MedicalResearch.com Interview with: 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 (more…)
Author Interviews, McGill, Multiple Sclerosis, Neurology / 09.05.2019

MedicalResearch.com Interview with: Douglas Arnold, MD The Montreal Neurological Institute & Hospital McGill University Montreal, QC, Canada MedicalResearch.com: What is the background for this study? Response: Diroximel fumarate (DRF) is a novel oral fumarate, with a distinct chemical structure that is being developed for relapsing forms of multiple sclerosis (MS). It is hypothesized that the distinct chemical structure of DRF may elicit less localized irritation in the gastrointestinal (GI) tract, potentially leading to improved GI tolerability. Diroximel fumarate is expected to have similar efficacy as dimethyl fumarate (marketed as TECFIDERA®), as both are converted to equivalent levels of monomethyl fumarate in the body. The EVOLVE-MS-1 study is primarily evaluating the safety of DRF and also exploring efficacy endpoints.   (more…)
Alzheimer's - Dementia, Author Interviews, Biomarkers, Neurological Disorders, Neurology, University of Pennsylvania / 08.05.2019

MedicalResearch.com Interview with: Lauren McCollum, MDCognitive and Behavioral Neurology FellowPenn Memory Center / Cognitive Neurology DivisionLauren McCollum, MD Cognitive and Behavioral Neurology Fellow Penn Memory Center / Cognitive Neurology Division MedicalResearch.com: What is the background for this study?   Response: Alzheimer’s Disease (AD) is a heterogenous condition, with considerable variability in cognitive symptoms and progression rates. One major reason for this heterogeneity is “mixed pathology,” – i.e., both AD- and non-AD pathology. Examples of non-AD pathology include cerebrovascular disease (CVD), Lewy Bodies, and TDP-43. Pathologically, Alzheimer’s Disease is defined by characteristic amyloid plaques and neurofibrillary tangles, which can be assessed for in living patients with CSF- or PET-based biomarkers for amyloid and tau, respectively. Classically, amyloid deposition begins years or even decades before pathologic tau accumulation, which is in turn associated with brain atrophy and cognitive decline. The recently developed NIA-AA “ATN” research framework allows for the classification of individuals with regard to 3 binary biomarkers: Amyloid (A), Tau (T), and Neurodegeneration (N). An individual’s ATN biomarker status indicates where along the “Alzheimer’s Disease continuum” they lie. Additionally, some ATN statuses are on the “typical AD” continuum, while others are not. Research has shown that 15-30% of cognitively normal older adults have elevated amyloid. It stands to reason that some portion of cognitively impaired individuals with elevated amyloid and neurodegeneration have something other than AD driving their neuronal injury. Within the context of the ATN research framework, this subset of people is the A+T-N+ group (i.e., people who have elevated amyloid and neurodegeneration, but are tau-negative), as amyloid alone (that is, amyloid without tau) is not thought to cause significant cognitive impairment or brain atrophy. Our hypothesis was that, compared to A+T+N+ (a set of typical-AD biomarkers), A+T-N+ have cognitive and neuroimaging profiles that deviate from a typical Alzheimer’s Disease pattern – i.e., with less memory loss and less atrophy in AD-signature regions – and may have biomarkers suggestive of alternate non-AD pathologies [e.g., white matter hyperintensities (WMHs), a marker of CVD]. (more…)
Author Interviews, Biomarkers, Multiple Sclerosis / 30.04.2019

MedicalResearch.com Interview with: Prof. Bernhard Hemmer MD PhD Director of the Neurology Clinic Technische Universität München  MedicalResearch.com: What is the background for this study? What are the main findings?  Response: The course of multiple sclerosis (MS) is still highly unpredictable and reliable markers to predict disability progression are largely missing. We found that patients with a high IgG Index, which means that the produce large amount of IgG within the CNS, have a higher risk of disease worsening during the first 4 years. I would consider patients with an elevated IgG index at a higher risk to run a more severe disease course. The marker could be used together with others to guide treatment decisions after multiple sclerosis diagnosis. (more…)
Author Interviews, Nature, Neurological Disorders, Nursing / 18.04.2019

MedicalResearch.com Interview with: Elsa F. Fouragnan PhD School of Psychology (Faculty of Health and Human Sciences) University of Plymouth MedicalResearch.com: What is the background for this study? What are the main findings?  Response: Counterfactual thinking is a psychological process that involves the tendency to create possible alternatives to life events that are currently happening. It is very important because it gives us the ability to switch away from uninteresting activities if better ones become available. For example, if you are working or doing the housework, you may be thinking about gardening or watching a movie later. As soon as your duties are finished, you may engage in these more exciting activities. In our study, macaque monkeys were tasked to find treats under several colored cups (on a screen). Some of these cups were better than others but were not always available, thus the animals had to retain what they had learnt about the good cups in case they became available again. We found that a frontal part of the brain called the anterior cingulate cortex was responsible for tracking which cups were the best in order to efficiently switch to them if the opportunity arose. If this part of the brain was not functioning properly, then animals were stuck in non-optimal choices. To reveal the causal role of the anterior cingulate cortex, we used a new neurostimulation method called low-intensity repetitive ultrasound to modulates activity in this part of the brain with millimetre accuracy. (more…)
Author Interviews, Epilepsy, Genetic Research, JAMA, Pediatrics / 12.04.2019

MedicalResearch.com Interview with: 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. (more…)
Author Interviews, Depression, Exercise - Fitness, JAMA, Parkinson's / 10.04.2019

MedicalResearch.com Interview with: Dr. Jojo Kwok  R.N., BN(Hons), MPH, Ph.D. School of Nursing, Li Ka Shing Faculty of Medicine The University of Hong Kong MedicalResearch.com: What is the background for this study? What are the main findings?  Response: Before the study, we knew that mind-body exercises such as yoga and stretching improves the physical health of patients with Parkinson’s disease (PD), however the benefits to their mental health was not known. This study concludes that mindfulness yoga alleviates psychological distress, improves spiritual well-being and quality of life, not to mention motor symptoms and mobility. When it comes to managing the stress and symptoms of Parkinson Disease, what is exciting, is that yoga has now been proven to be a better strategy than just stretching. Yoga draws together body, mind and spirit through mindful practice of 1) yoga posture, 2) breathing and 3) meditation. These form the three core components of our Mindfulness Yoga Program. Mindfulness is non-judgemental awareness of the present moment - of one’s physical sensations and thoughts, be they positive or negative. By adopting a mind-body approach, patients are much better positioned to reframe their illness journey than through physical training alone. By learning to relate non-judgmentally to their physical symptoms and emotions, they develop new coping skills that cultivate openness, acceptance and resilience to these symptoms. They feel better.  (more…)
ALS, Author Interviews, Statins / 15.02.2019

MedicalResearch.com Interview with: Alastair J. Noyce MD, PhD Preventive Neurology Unit, Wolfson Institute of Preventive Medicine Queen Mary University of London, Department of Clinical and Movement Neurosciences, University College London, Institute of Neurology, London UK MedicalResearch.com: What is the background for this study? What are the main findings?  Response: Amyotrophic lateral sclerosis (ALS) or motor neurone disease (MND) is a relentlessly progressive disorder that affects nerves which supply muscles. Over time the nerves die, leading to limb weakness, speech and swallowing problems, and ultimately breathing problems. Patients die on average 3-5 after diagnosis. There is no cure and the underlying disease processes are only understood in part. In this study, we adopted a large-scale approach to exploring causal risk factors for ALS. Causality is important because it implies that if one could modify or induce a change in a risk factor, one would observe a change in the risk of ALS. Observational studies struggle to prove causality definitely. Associations in observational studies can arise because: 1) the risk factor truly changes risk of ALS; or 2) something about ALS changes one’s exposure to the risk factor; or 3) the presence of another factor, which may or may not be known, can induce an association between a risk factor and ALS. Unless scenario 1 represents the truth, then changing the risk factor will not have any effect on risk of ALS. We used a proxy-based approach, known as Mendelian randomisation, to assess hundreds of possible risk factors for ALS for evidence of causality. What emerged from this was a very clear signal linking LDL cholesterol to risk of ALS. (more…)
Aging, Author Interviews, Neurological Disorders / 24.01.2019

MedicalResearch.com Interview with: Rachael D. Seidler, PhD Professor, Applied Physiology & Kinesiology University of Florida MedicalResearch.com: What is the background for this study? Response: There is accumulating evidence that spaceflight impacts the human brain: the brain is shifted higher within the skull and there are some regions of gray matter increases and decreases. To date, no studies have looked at the impact of spaceflight on human brain white matter pathways. Rodents flown in space show decreased myelination of white matter pathways. Here, we analyzed brain MRI scans pre and post spaceflight to quantify fluid shifts and white matter changes. (more…)
ALS, Author Interviews, Brigham & Women's - Harvard, Cognitive Issues, JAMA, Multiple Sclerosis / 08.01.2019

MedicalResearch.com Interview with: Michael Fralick, MD, FRCPC, SM, PhD (Cand) Clinical Associate, General Internal Medicine St Michael’s Hospital Phillipson Scholar, Clinician Scientist Program, University of Toronto PhD Candidate, IHPME, University of Toronto Affiliated Faculty, Program On Regulation, Therapeutics, And Law, Division of Pharmacoepidemiology and Pharmacoeconomics Brigham and Women’s Hospital, Harvard University MedicalResearch.com: What is the background for this study? What are the main findings? Response: This medication is a pill that combines two ingredients: dextromethorphan (the active ingredient in cough syrup) and quinidine (used to increase the concentration of dextromethorphan). The medication was primarily studied and evaluated in patients with amyotrophic lateral sclerosis (ALS)   or (multiple sclerosis) MS, but anecdotal evidence suggested it was being prescribed to patients with dementia. We used data from two nationwide healthcare databases to understand how the medication was being used in routine care. (more…)
Author Interviews, Immunotherapy, Multiple Sclerosis / 27.11.2018

MedicalResearch.com Interview with: Dietmar P. Berger, MD, PhD Head of Global R&D Atara Biotherapeutic MedicalResearch.com: What is the background for this study? Response: Epstein-Barr virus (EBV) is present in B lymphocytes, plasma cells and epithelial cells of over 95% of individuals over the age of 40.  Multiple studies have shown that nearly all patients with Multiple Sclerosis (MS) are EBV positive, including a recent presentation at the 2018 ECTRIMS Congress in Berlin that showed 100% of MS patients are positive for EBV (Ruprecht et. al). Current B cell directed therapies such as anti-CD20 therapies have demonstrated an effect on  Multiple Sclerosis activity. These therapies work by depleting B cells including those infected by EBV. Our belief is that loss of EBV-specific T cell function (e.g., T cell exhaustion) occurs in patients who develop Multiple Sclerosis, which results in the accumulation of EBV infected B and plasma cells in the CNS leading to the autoreactive immune cycle seen in MS patients. The increasing evidence of a link between EBV infection and the development of MS led to the initiation of a Phase 1 study to investigate the use of an autologous T-cell immunotherapy (ATA190) to selectively target and deplete EBV infected cells in patients with progressive MS. As T cell immunotherapies (like ATA190) are designed to penetrate the central nervous system, this approach was felt to be particularly useful in  Multiple Sclerosis where the inflammatory response and infected B lymphocytes and plasma cells are inaccessible inside the CNS to the vast majority of classic targeted agents. (more…)
Alzheimer's - Dementia, Author Interviews, Coffee, Parkinson's / 14.11.2018

MedicalResearch.com Interview with: Donald Weaver, PhD, MD, FRCPC, FCAHS Senior Scientist and Director, Research Institute Krembil Research Institute University Health Network Toronto, Canada MedicalResearch.com: What is the background for this study? What are the main findings? Response: First, we are seeking novel molecules that might have usefulness in the treatment of Alzheimer’s disease (AD). Since Mother Nature is a superb chemist, natural products are an ideal place to start looking for possible therapeutics. There is a long history (penicillin, digitalis …) of drugs identified from natural product sources. Moreover, in earlier work by us, we have shown that other natural products extracted from maple syrup have possible therapeutic efficacy against AD. Therefore, it was logical for us to look at extracts of coffee. We see similarities between maple syrup and coffee. In both of these natural products, the plant derived material (i.e. the coffee bean, or sap from maple syrup) is initially boiled or roasted prior to its use; thus, it is not a direct simple plant product, but one that has been heated (boiled or roasted). We suspect that the heating process “does more chemistry” enabling the generation of new molecules from the plant derived materials. In our study we show that a class of compounds (phenylindanes) from roasted coffee has the ability to inhibit the misfolding of two proteins (beta-amyloid, tau) whose misfolding and aggregation (“clumping”) is implicated in the disease process of AD. Second, as described below, there is already epidemiological evidence that coffee consumption may offer some protective effects against Alzheimer’s disease and Parkinson’s disease (PD), so by looking at the constituents of coffee for chemicals that might block the clumping of beta-amyloid and/or tau, was an attempt to seek a molecular link explaining the epidemiology. (more…)
Author Interviews, Gastrointestinal Disease, Parkinson's / 01.11.2018

MedicalResearch.com Interview with: Viviane Labrie, Ph.D. Assistant Professor Center for Neurodegenerative Science Van Andel Research Institute Grand Rapids, Michigan MedicalResearch.com: What is the background for this study? Response: Our lab has an interest in the early events and initiation of neurodegenerative diseases. Parkinson’s disease for a long time was thought to be a movement disorder driven by the destruction of dopamine neurons in a specific area of the brain, the substantia nigra. In the last 10 years it has become evident that Parkinson’s disease is not just a movement disorder but hosts a whole range of non-motor systems. One of the most common non-motor symptoms in Parkinson’s patients is issues with the gastrointestinal (GI) tract. GI symptoms often occur early in Parkinson’s disease; for many patients, GI symptoms precede the onset of motor symptoms by as many as 2 decades. Moreover, the GI is not only involved in the early signs of Parkinson’s but has been proposed to be a place in the body where Parkinson’s disease begins. The hallmark pathology of Parkinson’s disease in the brain is Lewy bodies, which contains a clumped form of a protein called alpha-synuclein. There is evidence that Parkinson’s disease pathology, this clumped alpha-synuclein protein, is detectable in the GI tract, even many years before the onset of Parkinson’s motor symptoms. Clumped alpha-synuclein is also capable of traveling across nerve cells. There is evidence that clumped alpha-synuclein can travel up the nerve that connects the GI tract to the brain and enter the brain. This could be disastrous because clumped alpha-synuclein can seed and spread in the brain, which has neurotoxic effects and can eventually lead to Parkinson’s disease. In fact, in the brain of Parkinson’s patients, one of the first places where alpha-synuclein clumps are detected is at the terminal where the gut nerve connects to the brain, and this pathology advances from this point to other brain areas as the disease progresses. This intriguing connection of the GI tract to the early processes of Parkinson’s disease had us interested in trying to understand how the gut could be involved in triggering Parkinson’s. But the GI tract is a very big place, and we first asked ourselves, where should we look to better understand GI involvement in Parkinson’s disease? (more…)
Author Interviews, JAMA, Neurology, Outcomes & Safety, Parkinson's, Pharmacology, University of Pennsylvania / 04.10.2018

MedicalResearch.com Interview with: 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. (more…)
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. (more…)
Author Interviews, Exercise - Fitness, JAMA, Neurology, Parkinson's / 23.09.2018

MedicalResearch.com Interview with: Fudi Wang, M.D., Ph.D. Qiushi Chair Professor Nutrition Discovery Innovation Center School of Public Health/School of Medicine Zhejiang University Hangzhou 310058, ChinaFudi Wang, M.D., Ph.D. Qiushi Chair Professor Nutrition Discovery Innovation Center School of Public Health/School of Medicine Zhejiang University Hangzhou  China MedicalResearch.com: What is the background for this study? What are the main findings? Response: Parkinson disease (PD) is the second most common neurodegenerative disease affecting approximately 10 million people around the world. To date, the cause of PD remains poorly understood. It is reported that 90% PD cases have no identifiable genetic cause. What’s worse, few therapeutic advances for the treatment of PD have been made in the past decades. Nevertheless, growing prospective longitudinal studies shed lights on the potential beneficial effect of lifestyle factors on reducing the risk of developing Parkinson disease. In this study, we performed a a dose-response meta-analysis of more than half a million participants. We found that physical activity, particularly moderate to vigorous physical activity, could significantly reduce PD risk. (more…)
Author Interviews, Cleveland Clinic, Multiple Sclerosis, NEJM / 30.08.2018

MedicalResearch.com Interview with: Robert J. Fox, MD, FAAN Principal Investigator | SPRINT-MS Trial Mellen Center for MS  |  Cleveland Clinic Cleveland, OH 44195  MedicalResearch.com: What is the background for this study? What are the main findings? Response: The current treatment options for progressive multiple sclerosis are very limited. The SPRINT-MS trial sought to obtain proof-of-concept evidence that ibudilast has beneficial activity in progressive multiple sclerosis. In a placebo-controlled, 96-week trial of 255 people living with progressive MS, treatment with ibudilast slowed the progression of brain atrophy (brain shrinkage) by 48% compared to placebo. Side-effects of ibudilast included gastrointestinal symptoms, headache, and depression.  (more…)
ALS, Author Interviews, Neurology / 21.08.2018

MedicalResearch.com Interview with: Pier Lorenzo Puri, M.D PhD Professor in the Development, Aging and Regeneration Program Sanford Burnham Prebys Medical Discovery Institute  MedicalResearch.com: What is the background for this study? What are the main findings? Response: My lab has been studying special repair cells called fibro-adipogenic progenitors (FAPs) and how these cells change in models of motor neuron diseases. These cells usually repair muscles after acute injury. But we are finding the FAPs change dramatically in disease settings. In this study we looked at these cells in models of spinal cord injury, ALS and spinal muscular atrophy, including muscle tissue from ALS patients. We found that FAPs change radically in several ways. Most importantly, the cells used a different signaling pathway, IL-6-STAT3, and when we blocked this signaling muscle atrophy and fibrosis halted. While further studies in humans are needed, this is a promising finding as FDA-approved medicines that block IL-6 and STAT3 are available.  (more…)
Author Interviews, Neurological Disorders, Neurology, Personalized Medicine, Radiology, Surgical Research / 13.07.2018

MedicalResearch.com Interview with: 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.   (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, CDC, JAMA, Neurological Disorders, Zika / 31.05.2018

MedicalResearch.com Interview with: 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.  (more…)
Author Interviews, Epilepsy, Neurological Disorders, NYU, Pharmaceutical Companies / 17.05.2018

MedicalResearch.com Interview with: https://www.gwpharm.com/epilepsy-patients-caregivers/patientsAnup Patel, M.D. Section Chief of Neurology Interim Division Chief of Neurology Nationwide Children’s Hospital MedicalResearch.com: What is the background for this study? Response: The study evaluated kids and adults with an epilepsy syndrome (Lennox Gastaut Syndrome – LGS) that is often difficult to treat and does not respond well to current medical treatment.  The study was a double blind randomized control trial evaluating how well a plant based, liquid solution, cannabidiol (CBD) product made by Greenwich Biosciences called Epidiolex helped to treat drop seizures (the most common seizure type in LGS) and how safe it was compared to placebo.  Two doses (10 mg/kg/day and 20 mg/kg/day) were evaluated compared to placebo. (more…)
Author Interviews, Emory, Hematology, JAMA, Neurological Disorders, Stroke / 23.04.2018

MedicalResearch.com Interview with : Dr. Hyacinth I Hyacinth MD Aflac Cancer and Blood Disorder Center, Emory Children’s Center, Department of Pediatrics, Emory University School of Medicine Atlanta, GA 30322 MedicalResearch.com: What is the background for this study? This study was conducted against the backdrop of a significantly higher risk for stroke among African Americans compared to non-Hispanic Whites, despite adjusting for traditional risk factors. Also, sickle cell disease is a well-known genetic risk factor for stroke and recent studies show that sickle cell trait is a risk factor for chronic kidney disease, venous thromboembolism and pulmonary embolism, all of which are potential risk factors for stroke. (more…)
Alzheimer's - Dementia, Author Interviews, Cognitive Issues, Epilepsy / 12.04.2018

MedicalResearch.com Interview with: Britta Haenisch, PhD Pharmacoepidemiology in Neurodegenerative Disorders German Center for Neurodegenerative Diseases, DZNE  MedicalResearch.com: What is the background for this study? What are the main findings? Response: Antiepileptic drugs (AEDs) have been shown to affect cognition by suppressing neuronal excitability and increasing inhibitory neurotransmission. Previous studies suggested that AEDs may be associated with cognitive adverse effects. Therefore, we evaluated the association between AED use and incident dementia and Alzheimer’s disease (AD). We utilized large longitudinal datasets from Finnish health registers and from German health insurance data. The case-control analyses was adjusted for several potential confounders like comorbidities and polypharmacy. The inclusion of a lag time between . Antiepileptic drugs use and dementia diagnosis allowed minimization of protopathic bias. Our study provides an association between regular prescription of  antiepileptic drugs with known cognitive adverse effects and the occurrence of dementia and AD in patients aged 65 years and older.  (more…)
Author Interviews, Epilepsy, JAMA / 10.04.2018

MedicalResearch.com Interview with: 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.  (more…)
ALS, Alzheimer's - Dementia, Author Interviews, JAMA / 09.04.2018

MedicalResearch.com Interview with: Celeste Karch, PhD Assistant Professor of Psychiatry Molecular mechanisms underlying tauopathies Washington University School of Medicine St Louis MedicalResearch.com: What is the background for this study? What are the main findings? Response: Nearly half of all patients with amyotrophic lateral sclerosis (ALS), a fatal neuromuscular disorder, develop cognitive problems that affect memory and thinking. Why a disease that primarily affects movement also disrupts thinking has been unclear. Our findings suggest that genetic connections between the two disorders may explain why they share some of the same features and suggest that some drugs developed to treat ALS also may work against frontotemporal dementia and vice versa. We used a statistical method in almost 125,000 individuals with ALS, frontotemporal dementia (FTD), progressive supranuclear palsy, corticobasal degeneration, Alzheimer’s disease and Parkinson’s disease to determine whether there are common genetic variants that increase risk for multiple neurodegenerative diseases. We found that common variants near the MAPT gene, which makes the tau protein, increases risk for ALS. MAPT has previously had been associated with diseases including frontotemporal dementia and Alzheimer’s disease. But the gene hadn’t been linked to ALS. We also identified variations in a second gene, BNIP1, which normally plays an important role in protecting against cell death, increased the risk of both ALS and frontotemporal dementia. ImportantlyBNIP1 mRNA levels were altered in people who had ALS and in patients with frontotemporal dementia, suggesting the BNIP1 may be a potential therapeutic target for both disorders. (more…)
Alzheimer's - Dementia, Author Interviews, JAMA, Parkinson's / 27.03.2018

MedicalResearch.com Interview with: 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. (more…)
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. (more…)