Author Interviews, Parkinson's / 25.07.2024

MedicalResearch.com Interview with: Gus Alva, MD, DFAPA Medical Director, ATP Clinical Research Medical Director, Senior Brain Health, Hoag Hospital, Newport Beach, Assistant Professor, Department of Psychiatry and Neuroscience University of California, Riverside, CA MedicalResearch.com: What is the background for this study? Response: This trial evaluated the effects of pimavanserin compared to placebo in frail older adults and elderly patients with neuropsychiatric symptoms related to Neurodegenerative disorder (NDD), such as hallucinations and delusions, to better understand the safety of pimavanserin in this population. The study was a phase 3b, 8-week treatment (study duration of up to 16 weeks) with the primary endpoint being safety and tolerability, measured by treatment-emergent adverse events (TEAEs). Secondary safety endpoints were change from baseline in motor and cognitive function; exploratory endpoints included suicidality, sleep quality, and neuropsychiatric symptoms. The reason for doing this study is that there is a high degree of interest in further understanding the safety of pimavanserin, as many antipsychotics used off label often have significant and serious adverse effects, including risk of falls, parkinsonism, and death. (more…)
Author Interviews, Brigham & Women's - Harvard, Infections, JAMA, Multiple Sclerosis, Neurological Disorders / 19.03.2024

MedicalResearch.com Interview with: Marianna Cortese, MD, PhD Senior Research ScientistDepartment of Nutrition Harvard T.H. Chan School of Public HealthBoston, MA 02115   MedicalResearch.com: What is the background for this study? Response: In a study published in Science in 2022, we reported compelling evidence that infection with the Epstein-Barr virus is the leading cause of Multiple Sclerosis. This is a follow-up study to investigate more in depth whether the antibody response to EBV is distinct in individuals with MS compared to individuals without MS and whether there is a part of EBV that the immune response is particularly targeting. For this purpose we assessed the immune response to all protein parts (peptides) of EBV and their association with MS. Previous studies could only look at parts of EBV and this is the first study looking at all EBV peptides. Antibodies to EBV (especially to a protein called EBNA1) are known to be overall higher in individuals with MS, so we also tested whether immune response overall or the immune response to specific EBV protein parts was more important. If the immune response to a specific EBV protein part (peptide) would be standing out or distinguishing individuals with MS, we hypothesized, it could point to a specific mechanism of how EBV may cause MS, i.e. it could point for example towards “molecular mimicry”, which is when antibodies targeting a pathogen start targeting a body-own structure (for example in the brain) which resembles the protein parts of the pathogen. (more…)
Author Interviews, Neurological Disorders, NYU, Pediatrics / 09.01.2024

MedicalResearch.com Interview with: Laura Gould, MSc, MA, PT Research Scientist SUDC Registry and Research Collaborative Comprehensive Epilepsy Center Department of Neurology NYU Langone Grossman School of Medicine     MedicalResearch.com: What is the background for this study? Response: Sudden Unexplained death in childhood (SUDC) is the unexplained death of a child on or after their 1st birthday that remains unexplained after a comprehensive death investigation. About 400 SUDC occur annually between the ages of 1-18, but more than half occur in toddlers, aged 1-4 years. Since most deaths are sleep related and unwitnessed with unremarkable autopsies, mechanisms of deaths have eluded our understanding. Febrile seizures are common in young children; ~ 3% of US children 6 months to 5 years will experience one. SUDC however has been associated with a 10-fold increase in febrile seizures; our study is the first to implicate them at time of death. The SUDC Registry and Research Collaborative (www.sudcrrc.org) at NYU Langone Health has enrolled over 300 cases of unexplained child death; seven with audiovisual recordings from the child’s bedroom during their last sleep period. More than 80% of the cases enrolled in the registry were children 1-4 years at the time of death. The seven cases with videos were aged 13-27 months with normal development and no pathogenic disease-causing variants by whole exome sequencing. (more…)
Author Interviews, Neurological Disorders, Science, UCLA / 29.09.2023

MedicalResearch.com Interview with: Michael Sofroniew, MD, PhD Professor UCLA School of Medicine MedicalResearch.com: What is the background for this study? How did this study differ from your previous work on this topic? Response: After spinal cord injuries, nerve fibers that are damaged do not spontaneously regrow across injury sites. In previous studies, our group of collaborators identified a combination of interventions that could stimulate damaged nerve fibers to regrow for short distances across injuries, but we found that in spite of this short distance regrowth there was no recovery of functions. The present study examined what type of regrowth might be necessary to re-establish functions. (more…)
Author Interviews, Neurological Disorders, Parkinson's / 24.04.2023

MedicalResearch.com Interview with: Alberto J. Espay, MD, MSc, FAAN Professor of Neurology Director and Endowed Chair Gardner Family Center for Parkinson's disease and Movement Disorders University of Cincinnati Academic Health Center MedicalResearch.com: What is the background for this study? Response: This study was meant to address the gap that current oral levodopa formulations do not suffice to lessen motor fluctuations in people with Parkinson’s disease. IPX203 is a unique extended-release formulation of levodopa. (more…)
Author Interviews, Genetic Research, Neurological Disorders, Novartis / 29.03.2023

MedicalResearch.com Interview with: Sitra Tauscher-Wisniewski, MD Vice President Clinical Development & Analytics Novartis Gene Therapies MedicalResearch.com: What is the background for this study? Would you briefly describe the condition of Spinal muscular atrophy (SMA)? Response: At the 2023 Muscular Dystrophy Association Conference, we presented new data from two of our  Long-Term Follow-Up (LTFU) studies, LT001 and LT002, which show the continued efficacy and durability of Zolgensma across a range of patient populations, with an overall benefit-risk profile that remains favorable. LT001 is a 15-year ongoing observational LTFU study following the Phase 1 START patients, who were the very first patients to receive our gene replacement therapy. LT-002 is a voluntary Phase 4 15-year ongoing follow-up safety and efficacy study of Zolgensma IV and investigational intrathecal (IT) OAV101 in patients previously treated in the Phase 3 IV studies (STR1VE-US, STR1VE-EU, STR1VE-AP, SPR1NT) and the Phase 1 IT study (STRONG). Spinal muscular atrophy (SMA) is a rare, devastating genetic disease that leads to progressive muscle weakness, paralysis, and when left untreated in one of its most severe forms (SMA Type 1), permanent ventilation or death in 90% of cases by age 2. It is caused by a lack of a functional survival motor neuron 1 (SMN1) gene, and in the most severe forms results in the rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement. (more…)
Author Interviews, Genetic Research, Infections, Neurological Disorders / 14.12.2022

MedicalResearch.com Interview with: Dr. Eli Hatchwell, MA MB BChir (Cantab) DPhil (Oxon) BA (OU) Chief Scientific Officer Population Bio UK, Inc. Begbroke Science Park Begbroke Hill Begbroke, Oxfordshire United Kingdom MedicalResearch.com: What is the background for this study? Response: Progressive Multifocal Leukoencephalopathy (PML) is a devastating condition that is associated with a number of clinical situations, including treatment with a variety of drugs. Of these, the best known is natalizumab (Tysabri), which is a very successful drug in the treatment of MS (multiple sclerosis). Only a small proportion of patients treated with natalizumab develop PML and this has always been a mystery. The study was based on a hypothesis that some individuals have an underlying susceptibility to developing PML, based on the presence of variants in genes that are important in the immune system. The study identified several of these variants. (more…)
Alzheimer's - Dementia, Author Interviews, Infections, Parkinson's / 22.09.2022

MedicalResearch.com Interview with: Jiangwei Sun PhD Postdoctoral researcher in Prof. Jonas Ludvigsson's group Department of Medical Epidemiology and Biostatistics Karolinska Institutet MedicalResearch.com: What is the background for this study? Response: A potential infectious etiology has been hypothesized for neurodegenerative diseases, as findings in animal studies have demonstrated that infectious processes might impact pathogenesis, phenotype, and progression of neurodegenerative disease. The extrapolation of such findings to a human context is however not straightforward. previous studies have mostly examined the role of specific pathogens on a specific neurodegenerative disease, e.g., herpesvirus for Alzheimer’s disease, and influenza, hepatitis C virus, and Helicobacter pylori for PD, with inconclusive results. Although several studies have also assessed associations between infectious diseases and risk of dementia and AD, influence of potential surveillance bias (greater-than-expected surveillance of disease after infections) and reverse causation (due to for example diagnostic delay of neurodegenerative diseases) on the associations was not always fully addressed. Therefore, whether infection is indeed a risk factor rather a comorbidity or secondary event of neurodegenerative disease remains unknown. In contrast to Alzheimer’s disease, and Parkinson’s disease, the potential link between infection and ALS has been less explored. (more…)
Author Interviews, Brigham & Women's - Harvard, Neurology, Parkinson's / 21.06.2022

MedicalResearch.com Interview with: Vikram Khurana, MD, PhD Chief of the Division of Movement Disorders Department of Neurology Brigham and Harvard Medical School Principal investigator, Ann Romney Center for Neurologic Diseases at the Brigham MedicalResearch.com:  What is the background for this study?   Response: Proteins abnormally accumulate in brain cells (neurons and glial cells) in all neurodegenerative diseases. In Parkinson’s disease and related disorders, the key protein that accumulates and aggregates is called “alpha-synuclein.” Presumably, when a protein like alpha-synuclein abnormally folds and aggregates, the abnormal form of the protein can become toxic to the neuron, eventually leading to cell death. Equally, the protein may no longer be able to carry out its normal function. This begs the question – what does alpha synuclein actually do? Most evidence to date points to alpha-synuclein being involved in the transport of other proteins and chemicals around the cell, by closely associating with vesicles that are small circular containers enclosed by fat (“lipid) membranes. But alpha-synuclein is not just found associated with these vesicle membrane. It is found away from the membrane and it’s been unclear what it does there. (more…)
Author Interviews, COVID -19 Coronavirus, Nature, Neurological Disorders / 07.04.2022

MedicalResearch.com Interview with: Tracy Fischer, PhD Associate Professor of Microbiology and Immunology Tulane National Primate Research Center    MedicalResearch.com:  What is the background for this study? Response: We investigated multiple regions of the brain from SARS-CoV-2 infected Rhesus macaques and African green monkeys for the presence of inflammation and other pathology that may result from COVID-19. Most animals were infected for approximately one month before our investigation, however, two of the African green monkeys developed acute respiratory distress syndrome (ARDS) prior to the study endpoint. (more…)
Author Interviews, Multiple Sclerosis / 19.01.2022

MedicalResearch.com Interview with: Marcus Koch MD PhD Associate Professor of Neurology Multiple Sclerosis Program University of Calgary MedicalResearch.com: What is the background for this study? Response: Primary progressive multiple sclerosis (PPMS) is the least common, but also the least treatable form of multiple sclerosis. PPMS does not react well to commonly used MS treatments. We believe that this is at least in part because PPMS is driven by other disease mechanisms. One disease mechanism that we believe is important in PPMS is microglial activation. Microglial cells are a type of cell in the brain and spinal cord that normally have beneficial functions, such as clearing debris or assisting repair after injury. In PPMS however, microglial cells are chronically active, and we believe that this chronic microglial activation contributes to tissue damage. (more…)
Author Interviews, Brigham & Women's - Harvard, Infections, Multiple Sclerosis, Science / 15.01.2022

MedicalResearch.com Interview with: Kassandra L. Munger Sc.D. Senior Research Scientist Alberto Ascherio MD Dr.P.H. Professor of Epidemiology and Nutrition Harvard T.H. Chan School of Public Health MedicalResearch.com: What is the background for this study? Response: An infectious cause of MS has been hypothesized for decades. Research over the past 20 years conducted by our group and others has strongly suggested a role for EBV infection including that EBV-negative individuals have a near zero risk of developing MS, having a history of infectious mononucleosis (caused by EBV infection) increases the risk of MS 2-fold, and healthy individuals have higher risks of MS with higher antibody levels against EBV antigens.  Ideally, to prove causality a randomized clinical controlled trial would be conducted; however, this not a feasible approach in this case. Given that nearly 95% of the adult population is infected with EBV and MS is a rare disease, we utilized the Department of Defense Serum Repository which stores over 60 million serum samples from over 10 million US Military active duty personnel. From this large resource, we were able to identify a cohort who were EBV negative when they joined the military and we followed them for whether they had a primary infection with EBV and then for who developed MS. (more…)
Author Interviews, Infections, JAMA, Multiple Sclerosis, Neurological Disorders / 27.10.2021

MedicalResearch.com Interview with: Prof. Scott Montgomery Professor of medical science (clinical epidemiology) Örebro University, Sweden Director of Clinical Epidemiology and Biostatistics Örebro University Hospital, Sweden MedicalResearch.com: What is the background for this study? Response: Infections have been linked with increased risk of subsequent multiple sclerosis (MS), but it has been suggested this may be because the genetic or other family characteristics of people who go on to develop MS have a more severe response to infections: the infections would be more likely to be recorded in those who would subsequently develop MS, rather than being risk factors for the disease. To address this issue, we performed a large study of 2,492,980 people living in Sweden, and 5,867 of them had a diagnosis of MS after age 20 years. We identified who had a hospital diagnosis of infectious mononucleosis (caused by Epstein-Barr virus, EBV infection, and also known as glandular fever or the kissing disease). The new study was different from other studies of infection and MS risk, as it compared siblings in the same families. Siblings share much of their genetic make-up and have similar family lives. If glandular fever is associated with later MS when siblings are compared, then it is unlikely that the association is caused by genetics or other family characteristics that make infections worse in people more likely to develop future MS. (more…)
Author Interviews, Brigham & Women's - Harvard, Medical Imaging, Neurological Disorders / 02.07.2021

MedicalResearch.com Interview with: Michael Ferguson, PhD Instructor in Neurology | Harvard Medical School Lecturer on Neurospirituality | Harvard Divinity School Center for Brain Circuit Therapeutics Brigham and Women’s Hospital MedicalResearch.com: What is the background for this study? Response: Over 80% of the global population consider themselves religious with even more identifying as spiritual, but the neural substrates of spirituality and religiosity remain unresolved. MedicalResearch.com: What are the main findings? Where is this circuit located in the brain? What other effects does this circuit control or influence? Response: We found that brain lesions associated with self-reported spirituality map to a human brain circuit centered on the periaqueductal grey. (more…)
Author Interviews, Brigham & Women's - Harvard, Clots - Coagulation, Hematology, Neurological Disorders, Pain Research / 22.05.2021

MedicalResearch.com Interview with: Daniel Chasman, PhD Pamela Rist, ScD, Yanjun Guo, MD, PhD Division of Preventative Medicine Brigham and Women’s Hospital  MedicalResearch.com: What is the background for this study? What are the main findings? Response: There has been speculation in the field about relationships between coagulation and migraine susceptibility for some time, but previous research has been largely inconclusive. In this study, we leveraged Mendelian randomization, a mode of genetic analysis that can support or refute potential causal effects on a health outcome, to examine whether hemostatic factors may contribute to risk of MA. (more…)
Author Interviews, Genetic Research, Parkinson's / 06.05.2021

MedicalResearch.com Interview with: Clemens R. ScherzerClemens R. Scherzer, M.D. Center for Advanced Parkinson Research Harvard Medical School Brigham and Women’s Hospital Boston, MA MedicalResearch.com: What is the background for this study? Response: Parkinson's disease is the fastest growing brain disorder. The number of patients is projected to double to 14 million by 2040. The total cost of Parkinson's is $52 billion every year in the U.S. Yet, there are no medicines available to slow the disease. Current treatments temporarily alleviate symptoms, but do not address the underlying disease process, which continues to relentlessly progress. To begin to solve this puzzle, we searched the genome of 3,821 Parkinson's disease patients for genetic variants linked to rapid progression over time to dementia, which is a major determinant for a Parkinson's disease patient’s quality of life. These patients were deeply characterized in the International Genetics of Parkinson Disease Progression (IGPP) Consortium, a grass-roots, collaborative network of Parkinson’s investigators, with 31,578 longitudinal study visits over up to 12 years from disease onset.  (more…)
Author Interviews, Dermatology, JAMA, Parkinson's / 01.10.2020

MedicalResearch.com Interview with: Wenquan Zou, MD/PhD, Professor Department of Pathology Associate Director National Prion Disease Pathology Surveillance Center Case Western Reserve University School of Medicine Cleveland, Ohio 44106 MedicalResearch.com: What is the background for this study? Response: Parkinson’s disease (PD) is the second most common age-related neurodegenerative disorder. It is characterized by the accumulation of pathologically misfolded α-synuclein (αSynP) aggregates in the brain. Currently, a definite diagnosis relies on the detection of αSynP-containing Lewy bodies in the brain of PD patients. Development of a reliable and sensitive assay for αSynP in easily accessible peripheral tissue specimens is critical for early or differential diagnosis, determination of disease severity, and evaluation of therapeutic efficacy in clinical trials. Previous studies have revealed that the pathologically phosphorylated α-synuclein is detectable with traditional immunohistochemistry (IHC) and immunofluorescence (IF) microscopy but the sensitivity with IHC/IF is highly variable and inconsistent. Also the prion-like aggregation seeding activity of αSynP is detected in cerebrospinal fluid (CSF) of Parkinson’s disease patients with highly sensitive real-time quaking-induced conversion (RT-QuIC) and protein misfolding cyclic amplification assays (PMCA). But the lumbar puncture to collect CSF is more invasive compared to skin punch biopsy. (more…)
Author Interviews, Neurology, Parkinson's / 22.06.2020

MedicalResearch.com Interview with: Stewart A. Factor, D.O. Professor of Neurology Director of the Movement Disorders Program Vance Lanier Chair of Neurology Emory University School of Medicine MedicalResearch.com: What is the background for this study? Would you briefly explain what is meant by OFF episodes.  Response: Parkinson’s disease (PD) is a chronic, progressive neurodegenerative disease characterized by motor symptoms, including tremor at rest, rigidity and impaired movement, as well as significant non-motor symptoms, such as cognitive impairment, psychiatric symptoms and autonomic symptoms (i.e. urinary issues, constipation, low blood pressure). It is the second-most common neurodegenerative disease after Alzheimer’s disease and it is predicted that the prevalence of Parkinson’s disease will double by the year 2040. The symptoms of PD are in substantial part, due to loss of dopamine nerve cells in the brain. The current standard of care for PD includes replacing the dopamine loss by the use of oral carbidopa/levodopa. Levodopa is a precursor of dopamine, converted in the brain. OFF episodes have been a significant unmet need in Parkinson’s disease since the emergence of levodopa. Initially, levodopa controls PD symptoms in a continuous fashion throughout the day. With time the response becomes less predictable and patients experience a re-emergence or worsening of PD symptoms. These episodes are what we mean by OFF episodes. OFF episodes can be characterized, in part, by re-emergence of motor symptoms including tremor, stiffness or slowed movement that can happen at any point during the day. OFF episodes typically begin within the first five years of treatment and occur at the end of a dose. This is referred to as end of dose failure or wearing off. Within the first four to six years after diagnosis, regardless of disease severity, up to 60 percent of people with PD experience OFF episodes. With time these episodes become longer, more severe and disabling, more frequent and less predictable as PD progresses. They can take up more than half the day OFF episodes may alter a persons’ ability to perform everyday activities by slowing or even precluding their completion. The result is significant burden and distress for people living with Parkinson’s disease (PD) and their care partners. CTH-300 was a Phase 3, 12-week, randomized, double-blind, placebo-controlled, parallel group, study examining the efficacy, safety and tolerability of apomorphine hydrochloride sublingual film (KYNMOBI) in people with levodopa-responsive PD complicated by OFF episodes. The primary endpoint was a mean change in the score from pre-dose in the Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III Motor Examination at 30 minutes after dosing at the 12-week visit of the maintenance treatment phase. The key secondary endpoint was the percentage of people with PD with a patient-rated full ON (or best) response within 30 minutes at the 12-week visit of the maintenance treatment phase. (more…)
Author Interviews, Neurological Disorders, Personalized Medicine / 19.06.2020

MedicalResearch.com Interview with: https://polyneuron.com/Pascal Hänggi, PhD Chief Scientific Officer Polyneuron Pharmaceuticals   MedicalResearch.com: What is the background for this study? Response: Anti-MAG neuropathy is a rare form of acquired demyelinating neuropathy. The disease onset normally presents after the age of 50 years and is 2.7 times more frequent in men than in women, with a prevalence of about 1 in 100,000. It is caused by the production of monoclonal anti-MAG IgM antibodies that recognize the HNK-1 epitope. The myelin-associated glycoprotein MAG is a mediator for the formation and maintenance of the myelin sheaths. There is strong evidence that the binding and deposition of anti-MAG IgM autoantibodies on myelin sheath is responsible for the demyelination, which clinically manifests itself as a peripheral neuropathy affecting primarily sensory nerves. However, the causes and the exact mechanisms behind the expansion of anti-MAG IgM producing B-cell and plasma cell clones are not fully understood. Most off-label treatments aim to reduce pathogenic autoantibody titers by depleting  autoantibody-producing B cell clones which interfere with antibody-effector mechanisms, or physically remove autoantibodies from the circulation. Most frequently, the anti-CD20 monoclonal antibody rituximab is used to treat anti-MAG neuropathy patients. However, all of these treatment options often lack of selectivity, efficiency, or can induce severe adverse effects in some patients. Polyneuron has designed PN-1007 to highly selectively target the IgM autoantibodies that cause anti-MAG neuropathy. PN-1007 is a glycopolymer that mimics the natural HNK-1 carbohydrate epitope found on myelin of peripheral nerves and binds to the circulating disease-causing antibodies. By eliminating these pathogenic antibodies, PN-1007 may protect the integrity of the neuronal myelin sheaths of anti-MAG neuropathy patients. (more…)
Author Interviews, JAMA, Multiple Sclerosis, Neurology / 15.06.2020

MedicalResearch.com Interview with: Cris S Constantinescu,  MD, PhD, FRCP Professor, Division of Clinical Neuroscience Research Group in Clinical Neurology University of Nottingham Queen's Medical Centre Nottingham UK MedicalResearch.com: What is the background for this study? Response: The study is in some way a test of the hygiene or old friends hypothesis, whereby eradication, through improved hygiene, of some parasites that have existed in the human gut for thousands of years and have suppressed inflammatory reactions, leads to an increase in inflammatory conditions. This has been used to explain the increased autoimmune and inflammatory diseases in the developed world. Healthy volunteer studies at the University of Nottingham showed therapeutic hookworm infection to be safe and well tolerated up to about 50 larvae, and then safety studies in people with airway hyperreactivity and inflammatory bowel disease raised no concern. Following a study in Argentina showing that people with MS have milder disease when they have a natural co-existing asymptomatic infection with intestinal parasites, we (Professor Pritchard, immunoparasitologist and myself) decided to test hookworm in MS, and for the first time used 25 larvae in a patient study. (more…)
Author Interviews, Neurological Disorders, Neurology, Pain Research / 11.05.2020

MedicalResearch.com Interview with: Holly Yancy, DO Headache medicine specialist Banner – University Medicine Neuroscience Institute Phoenix, AZ Dr. Yancy comments on the recent Neurology journal article on the potential impact of yoga on migraine.  MedicalResearch.com: What is the background for this study? How might yoga reduce migraine intensity or frequency?  Response: The authors of this trial have studied the benefits of yoga when added to medical management of episodic migraine. They expand on prior, smaller reports of the potential benefit of yoga and mindfulness to migraine patients with a well-designed study that shows yoga, as an adjunct to preventive medication, can lower the intensity, frequency and impact of migraines. Participants even used less abortive medication. The authors propose multiple potential mechanisms of action, including an increase in parasympathetic / decrease in sympathetic nervous system activity, decreased muscle tension, and stress management. (more…)
Author Interviews, Biogen, Neurological Disorders, Pharmaceutical Companies / 30.04.2020

MedicalResearch.com Interview with: Toby Ferguson, M.D., Ph.D. Vice President, Head Neuromuscular Development Unit Biogen MedicalResearch.com: What is the background for this study? Would you briefly explain what is meant by SMA, who is primarily affected and incidence? Response: Spinal muscular atrophy (SMA) is a rare, genetic, neuromuscular disease characterized by a loss of motor neurons in the spinal cord and lower brain stem that can result in severe, progressive muscle atrophy and weakness. Approximately one in 10,000 live births have a diagnosis of SMA. It is a leading genetic cause of infant mortality; however, people of all ages are impacted by the disease. More than three years ago, SPINRAZA (nusinersen) became the first FDA-approved treatment option for SMA. The DEVOTE study, which recently treated its first patient, is designed to evaluate the safety and potential for even greater efficacy of SPINRAZA when administered at a higher dose than currently approved for the treatment of SMA. The Phase 2/3 randomized, controlled, dose-escalating study will be conducted at approximately 50 sites around the world and aims to enroll individuals of all ages with SMA. (more…)
Author Interviews, Neurological Disorders, Parkinson's / 15.04.2020

MedicalResearch.com Interview with: Dr. Viviane Labrie, PhD Dr. Labrie is an associate professor in Van Andel Institute’s Center for Neurodegenerative Science, where she studies Parkinson’s, Alzheimer’s and other neurological diseases. MedicalResearch.com: What is the background for this study? Response: One of the most puzzling and persistent mysteries in neuroscience has been why some people are “right-brained” while others are “left-brained.” The two sides of the brain have different jobs. The left side is analytic and problem-solving, while the right side manages creativity and artistic talents. But despite their differences, the two sides are composed of the same cell types — essentially, brain neurons and their support cells. In this study, we sought to understand how it is possible for these cells to behave completely differently depending on what hemisphere they’re located in.  We also wanted to examine the reasons behind asymmetry in Parkinson’s disease; that is, why Parkinson’s symptoms typically start on one side of the body before the other. This asymmetry in neurodegeneration and symptoms in patients is one of the biggest unsolved puzzles in the Parkinson’s disease field — why do brain cells in one hemisphere begin dying before brain cells in the other hemisphere? (more…)
Abuse and Neglect, Alzheimer's - Dementia, Autism, Medical Imaging, Mental Health Research, MRI, Multiple Sclerosis, Neurology, Technology / 23.12.2019

MedicalResearch.com Interview with: Sebastian Magda, Ph.D Director of Science & Engineering CorTechs Labs, Inc MedicalResearch.com: What is the background for this study? Response: Previous studies have shown that the changes of brain structure volume and/or metabolic activity are associated with various neurological diseases. We have created an artificial intelligence clinical decision support tool based on brain volumetric and PET metabolic activity measurements as well as other clinical measurements. (more…)
Author Interviews, Epilepsy / 19.12.2019

MedicalResearch.com Interview with: Steve S. Chung MD Epilepsy Neurology Banner University Medical Center University of Arizona, Phoenix, AZ MedicalResearch.com: What is the background for this study? Response: Prior studies have shown that midazolam nasal spray (MDZ-NS) is superior to placebo in providing rapid, sustained seizure control and a favorable safety profile when administered to patients experiencing seizure clusters, a type of seizure emergency impacting an estimated 150,000 patients in the U.S. with uncontrolled epilepsy. To explore the influence of concomitant antiepileptic drugs (AEDs) on treatment outcomes, we conducted a post hoc analysis of a Phase III trial to evaluate the tolerability and efficacy of midazolam nasal spray in patients 12 years of age and older with seizure clusters, according to concomitant enzyme-inducing AED (EIAED)/non-enzyme-inducing AED (NEIAED) status and by the number of concomitant AEDs. (more…)
Author Interviews, Epilepsy / 19.12.2019

MedicalResearch.com Interview with: Eun Jung Choi, MD, PhD; Senior Director, New Product Launch, Neurology UCB MedicalResearch.com: What is the background for this study? Response: Patients experiencing seizure clusters, a type of seizure emergency impacting an estimated 150,000 patients in the U.S. with uncontrolled epilepsy, are at risk for potentially serious consequences including hospitalization, mortality, physical injury, neurological damage, and status epilepticus. Although unmet needs of seizure clusters are intuitively well accepted, the burden of seizure clusters has not been well characterized and evaluated in a systemic matter. As a result, the seizure cluster patients are overlooked, and burden of seizure clusters is under-recognized. So we systematically assessed the clinical, humanistic, and economic burden associated with seizure clusters to provide the comprehensive body of evidence about burden of seizure clusters. (more…)
Author Interviews, Epilepsy, NEJM, NIH / 11.12.2019

MedicalResearch.com Interview with: Robin Conwit, M.D Program Director, Division of Clinical Research NIH: National Institute of Neurological Disorders and Stroke MedicalResearch.com: What is the background for this study? Response: Approximately one-third of patients with intractable status epilepticus do not respond to benzodiazepines, often the first line medications given in the emergency department, leaving doctors to decide among three commonly prescribed anti-convulsants. In this study all of the anti-convulsants were equally effective. The trial gives doctors a way to clear the air of arguments that one of these drugs works any better than the others, or that any one of them is a lot safer.  It gives doctors a reason to choose a dosing strategy in status epilepticus for levetiracetam, which has otherwise been controversial and non-standard.   (more…)
Author Interviews, Lipids, NEJM, Neurological Disorders, Stroke / 18.11.2019

MedicalResearch.com Interview with: Pierre Amarenco, MD Professor and Chairman Paris University, Paris, France INSERM Department of Neurology and Stroke Centre Bichat Hospital Paris, France MedicalResearch.com: What is the background for this study? Response: The rationale of the Treat Stroke to target trial was that after we published the SPARCL trial in 2006 (atorvastatin 80 mg/day vs placebo in patients with stroke) which showed a 16% relative risk reduction of recurrent stroke, we performed several pre specified and post hoc analyses, showing that in SPARCL patients randomized with "atherosclerotic disease" the risk reduction for the primary endpoint was much higher (33%), and in in patients achieving a LDL cholesterol of less than 70 mg/dL as compared to those achieving a LDL cholesterol 100 mg/dL or higher, the risk reduction was 28%. Therefore to confirm this findings, we designed the TST trial, which was an investigator initiated trial funded by the french ministry of health,  and enrolled patients with an ischemic stroke due to atherosclerotic stenosis and randomized them to either a target LDL cholesterol of less than 70 mg/dL or a target LDL cholesterol of 90 to 110 mg/dL. To achieve these goals, the investigators could use any statin available on the market, and titrate the dosage of the statin to get to the assigned target. They could also use ezetimibe on top of statin therapy if a high dosage of statin was not sufficient to get to the target level assigned by randomization. (more…)
Author Interviews, Epilepsy, Nutrition / 23.07.2019

MedicalResearch.com Interview with: Geoffrey W. Abbott PhD Department of Physiology and Biophysics, Bioelectricity Laboratory, School of Medicine, University of California MedicalResearch.com: What is the background for this study? Response: The main focus of my laboratory is the study of potassium ion channels - proteins that coordinate electrical activity in all organisms. When human potassium channels do not function properly, it can result in pathologically discordant electrical activity, and diseases such as cardiac arrhythmia, myotonia, and epilepsy - depending on whether the affected potassium channel is in the heart, skeletal muscle or brain, for example. T here are existing drugs that directly regulate ion channels for therapeutic benefits, including one - retigabine - that opens neuronal potassium channels in the KCNQ family, to treat epilepsy. Retigabine causes side effects including turning the skin blue, and was withdrawn from clinical use in 2017. Retigabine may make a comeback because a form of epilepsy was recently discovered, arising from mutations in the KCNQ2 gene, that is associated with severe developmental delay and seizures. In my lab, we are interested in discovering new therapeutic agents that might more safely fix dysfunction in KCNQ2 and other potassium channels. We turned to plants as a possible source of compounds. We are interested both in explaining the underlying mechanism of traditional botanical medicines, and also discovering unanticipated therapeutic chemicals synthesized naturally by plants.  (more…)