The hectic nature of modern living, especially if you’re very much immersed in the changing world of business, can result in the accidental neglect of both your physical health and emotional well-being. Naturally, your health is the topmost priority and as such, making the switch to ensuring you’re taking care of...
Dr. Tauscher-Wisniewski,[/caption]
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.
Dr. Hatoum[/caption]
Alexander S. Hatoum, PhD
Research Assistant Professor
Institute for Behavioral Genetics
Washington University in St. Louis
MedicalResearch.com: What is the background for this study?
Response: It is well known that someone with one substance use disorder will have another sometime in their lifetime or concurrently. Further, individuals that do manifest two or more substance use disorders in their lifetime have the most morbid conditions. However, research often ignores the comorbidity and focuses on diagnosis of one substance use disorder at a time (i.e. opioid use disorder or alcohol use disorder). We set out to identify the biology behind the cross-substance liability.
Dr. Potter[/caption]
MedicalResearch.com Interview with:
Kelly Potter, PhD, RN, CNE
T32 Postdoctoral Scholar
CRISMA Center, Department of Critical Care Medicine
University of Pittsburgh
MedicalResearch.com: What is the background for this study?
Response: While it is well-recognized that survivors of critical illness often experience persistent problems with mental, cognitive, and physical health, very little is known about how these problems (collectively known as post-intensive care syndrome (PICS)) affect resumption of meaningful activities, such as driving.
Dr. Lillegraven[/caption]
Siri Lillegraven MD MPH PhD
Dr. Le Calvez-Kelm[/caption]
Florence Le Calvez-Kelm, Ph.D.
Genomic Epidemiology Branch
International Agency for Research on Cancer
Lyon, France and
[caption id="attachment_60206" align="alignleft" width="150"]
Dr. Levin[/caption]
Trevor Levin Ph.D.
Founder and CEO of Convergent Genomics that produces the Uroamp assay
San Francisco, CA
MedicalResearch.com: What is the background for this study?
Response: Bladder cancer is one of the most expensive and challenging to diagnose and treat. Therefore, identifying cost-effective urine bladder cancer biomarkers to complement or replace the gold-standard invasive and costly cystoscopy for the early detection and monitoring of this highly recurrent disease is crucial. At the international Agency for research on Cancer (IARC-WHO), we have developed a simple urine-based assay TERT promoter mutations, the most common mutations in bladder cancer, and showed that the urine biomarker could detect bladder cancer patients at diagnosis but many years prior to clinical diagnosis. However, in this study, we wanted to see whether a more comprehensive genomic profiling of urine samples collected years prior to clinical diagnosis of bladder cancer could identify even more patients before they develop any symptoms.
The study was based on the UroAmp test, a general urine test that identifies mutations in 60 genes, developed by the Oregon Health Science University spin out company, Convergent Genomics. Drawing on previous research to identify genetic mutations linked to bladder cancer, the research team narrowed the new test down to focus on mutations within just ten genes.
Working with colleagues from the Tehran University of Medical Sciences in Iran, they trialled the potential new test using samples from the Golestan Cohort Study, which has tracked the health of more than 50,000 participants over ten years, all of whom provided urine samples at recruitment. Forty people within the study developed bladder cancer during that decade, and the team were able to test urine samples from twenty-nine of them, along with samples from 98 other similar participants as controls.
This article is for informational purposes only and is not a substitute for professional medical advice, diagnosis or treatment. Contact a qualified medical professional before engaging in any physical activity, or making any changes to your diet, medication or lifestyle, Alcohol abuse is a serious problem that can have devastating...
Dr. Jonathan Silverberg[/caption]
Jonathan Silverberg, MD, PHD, MPH
Professor
Director of Clinical Research
Director of Patch Testing
George Washington University School of Medicine and Health Sciences
Washington, DC
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: Lebrikizumab was previously shown to be safe and effective as a treatment for moderate-severe atopic dermatitis in a phase 2 study. These Phase 3 randomized placebo-controlled trials are the largest studies to date of lebrikizumab in AD. They showed that lebrikizumab was safe and highly effective for the treatment of moderate-severe atopic dermatitis. These studies will hopefully support the approval of lebrikizumab in the United States later this year.
Dr. D'Orsogna[/caption]
Maria-Rita D'Orsogna Ph.D.
Professor, Mathematics
California State University, Northridge
Adjunct Associate Professor
Department of Computational Medicine at UCLA
MedicalResearch.com: What is the background for this study?
Response: Drug overdose deaths have been increasing in the USA for the past two decades. A ‘third wave’ of overdose fatalities started in 2013, with a shift from prescription opioids towards synthetic ones, in particular illicit fentanyl.
To examine trends in drug overdose deaths by gender, race and geography in the United States during the period 2013-2020, we used an epidemiological database provided by the Centers for Disease Control and Prevention, extracting rates by race and gender in all 50 states plus the District of Columbia. We considered the impact of four main drug categories psychostimulants with addiction potential such as methamphetamines; heroin; prescription opioids and synthetic opioids such as fentanyl and its derivatives.
Dr. Koh[/caption]
Andrew Y. Koh, M.D.
Associate Professor, Pediatrics and Microbiology
University of Texas Southwestern Medical Center
Director of Pediatric Cellular and ImmunoTherapeutics Program
University of Texas Southwestern Medical Center and Children's Health
MedicalResearch.com: What is the background for this study?
Response: We asked the basic question how does a bacteria in your gut help your immune system fight a cancer outside the gut (extraintestinal tumor). Based on work that our group and others have published in the infectious diseases, microbiology, and inflammation fields, we knew that certain conditions (e.g. inflammation, infection) promote gut microbiota to move from the gut to the mesenteric lymph nodes. So we hypothesized that immune checkpoint therapy (which essentially induces inflammation to promote tumor killing) might induce gut microbiota translocation to the mesenteric lymph nodes and that this might be the first step by which gut bacteria can engage with host immune cells.
Prof. Rahimi[/caption]
Kazem Rahimi FRCP, DM, MSc, FESC
Professor of Cardiovascular Medicine and Population Health
University of Oxford
Consultant cardiologist
Oxford University Hospitals NHS Trust
MedicalResearch.com: What is the background for this study?
Response: The prevalence of hypertension has been rising worldwide. To mitigate the burden, identifying the modifiable environmental risk factors of hypertension and developing preventive interventions constitute important public health priorities. Despite the biological plausibility of the link between road traffic noise and the risk of hypertension, the quality of relevant evidence has been low, and the role of air pollution has been uncertain.
Dr. Turro[/caption]
Ernest Turro, PhD
Associate Professor Genetics and Genomics Sciences
The Turro group runs a research program on statistical genomics,
with a dual focus on rare diseases and blood-related traits at the Icahn School of Medicine
Mount Sinai Health System
MedicalResearch.com: What is the background for this study? Would you describe the Rareservoir database?
Response: The main motivation for our work is that only half of the approximately 10,000 catalogued rare diseases have a resolved genetic cause (or aetiology). Patients with these diseases are unable to obtain a genetic diagnosis which could otherwise inform prognosis, treatment for themselves and affected relatives.
One route towards resolving the remaining aetiologies is to enroll large numbers of rare disease patients into research studies so that statistical analyses can be performed comparing the genetic with the clinical characteristics of the study participants. One major endeavour, the 100,000 Genomes Project (100KGP), sequenced the genomes and collected clinical phenotype data for 34,523 UK patients and 43,016 unaffected relatives across 29,741 families.
The scale of this study is unprecedented, partly thanks to the ever-decreasing cost of DNA sequencing (25 years ago, it cost $1bn to sequence a whole genome, while now it costs only a few hundred dollars). Working with such large datasets is notoriously cumbersome. To overcome this, we developed a computational approach (the Rareservoir) that distills the most important information into a relatively small database, allowing us to apply our statistical methods nimbly.
We noted that the "genetic variants" that cause rare diseases are typically kept rare in the human population by natural selection because affected individuals tend to have few children, if any. This meant that we could discard the genetic information corresponding to variants that are common in the human population without throwing away the key disease-causing variants. By focussing on these "rare variants", we were able to perform our analyses using a small database (a `Rareservoir’), only 5.5GB in size, hastening our progress significantly.
Dr. Keyes[/caption]
Dr Helen Keyes PhD, AFBPsS, SFHEA
Head of School Psychology & Sport Science
Anglia Ruskin University
Cambridge
MedicalResearch.com: What is the background for this study? What types of sporting events?
Response: The data were collected as part of a large government study looking at a range of measures on health and activities across the UK population. Our study honed in on aspects of wellbeing – life satisfaction, loneliness, happiness, anxiety, a sense that life is worthwhile – as well as whether participants had attended a live sporting event in the last year. The data collected did not differentiate between different types of sport – the positive effects that we report for wellbeing are population-wide across a whole range of sports, from attending a local football match all the way up to elite sporting events.
Dr. Mosley[/caption]
Jonathan Mosley, MD, PhD
Associate Professor
Division of Clinical Pharmacology
Departments of Internal Medicine and Biomedical Informatics
Vanderbilt University Medical Center
MedicalResearch.com: What is the background for this study?
Response: Prostate cancer is an important source of morbidity and mortality among men. Earlier detection of disease is essential to reduce these adverse outcomes. Prostate cancer is heritable, and many single nucleotide polymorphisms (SNPs) associated with disease risk have been identified. Thus, there is considerable interest in using tools such as polygenic risk scores, which measure the burden of genetic risk variants an individual carries, to identify men at elevated risk of disease.
Dr. Moses, D.O., J.D.[/caption]
Richard E. Moses, D.O., J.D.
Gastroenterologist, Associate Vice President, Mirikizumab Indication Lead
Global Medical Affairs, Eli Lilly and Company
MedicalResearch.com: What is the background for this study? Would you briefly describe how mirikizumab works in UC?
Response: First, this study specifically evaluated mirikizumab, a humanized IgG4 monoclonal antibody that selectively targets the p19 subunit of IL-23 and inhibits the IL-23 pathway. Inflammation due to over-activation of the IL-23 pathway plays a critical role in the pathogenesis of UC.
Regulatory decisions for mirikizumab as a potential treatment for adults with moderately to severely active UC in the U.S., E.U and other countries around the world are expected in 2023. If approved, mirikizumab has the potential to be the only UC treatment that selectively targets the p19 subunit of IL-23.
Gastroenterologists today benefit from having data from a range of endpoints, which can help them determine appropriate treatment options depending on their specific patients' needs and symptoms. In addition to clinical response and clinical remission – which are often used to determine the effectiveness of a treatment – we can also use combined endpoints like histologic-endoscopic mucosal improvement (which gauges remission and treatment effectiveness), histologic-endoscopic mucosal remission (the reduction of underlying inflammation visible endoscopically) and inflammatory biomarkers faecal calprotectin (fCal) and C-reactive protein (CRP) to inform our treatment strategies.
This analysis focused on patients treated with mirikizumab from the induction study who received intravenous mirikizumab every 4 weeks until Week 12 (LUCENT-1), and patients who responded to mirikizumab during 12-week induction period who were re-randomized for the maintenance period, receiving subcutaneous mirikizumab every four weeks up to Week 40 (LUCENT-2) for 52 weeks of continuous treatment.
This study explored the relationship between achieving histologic-endoscopic mucosal improvement (HEMI), histologic-endoscopic mucosal remission (HEMR) and improvement of biomarkers fCal and CRP levels at Weeks 12 and 52.
Dr. Tsirigos[/caption]
Aristotelis Tsirigos, Ph.D.
Professor of Medicine and Pathology
Co-director, Precision Medicine
Director, Applied Bioinformatics Laboratories“The American Diabetes Association (ADA) is the leading voice advocating for insulin affordability and is working to ensure that all people with diabetes have access to the care they need. The ADA has led the charge to enact cost-sharing limits on insulin in 22 states and D.C., and now the...
The FDA has the final say on the standards and requirements for medical device manufacturing. Its risk-based classification system is an important part of protecting the industry. Although there are several regulations to follow, compliance is not a problem for the many medical devices worldwide. The Demand Contract manufacturing for medical devices...
Dr. Kleiman[/caption]
Norman Kleiman, PhD, MS
Department of Environmental Health Sciences
Mailman School of Public Health
Columbia University, New York, NY
MedicalResearch.com: What is the background for this study?
Response: The 1986 Chornobyl nuclear disaster caused the evacuation of 300,000 persons from the cities and villages surrounding the nuclear power plant complex. Pets and belongings were left behind, and the Soviet authorities ordered all animals within the Chornobyl Exclusion Zone killed. Some dogs evaded destruction, and some 300+ descendants of these animals live primarily at two locations today, immediately surrounding the Nuclear Power Plant (NPP) complex and about 10 km away in Chornobyl city. What is relatively unknown to the general public is that Chornobyl is not a desolate, abandoned wasteland. Some thousands of individuals work there every day in continuing cleanup activities and at two new fuel reprocessing facilities built near the damaged reactor. These areas have been substantially remediated, and the average radiation levels are relatively modest. The dogs, which, while feral, are accustomed to human interaction, live near the workers and are not currently exposed to high radiation levels. In contrast to lower radiation levels, there is a toxic mixture of heavy metals, organics, pesticides, and unknown chemicals left over from years’ long cleanup efforts and the decay of a large former military-industrial complex at the NPP.
Since 2016, the NPP authorities have brought in teams of veterinarians and volunteers to spay, neuter, and vaccinate the dogs to protect the workers and deal with a growing population. At the same time, some scientists joined the teams to obtain various kinds of biospecimens (hair, urine, feces, blood, saliva, parasites) to examine the animals’ health and learn how this toxic environment may have affected them or their offspring. Since dogs are human companion animals and live closely with us, any information we learn about health risks to the dogs may be relevant to protecting human workers and inform us about the kinds of health risks posed by ecological and environmental disasters in the future.
Dr. Hulstrøm[/caption]
Veronica Hulstrøm MD, PhD
Senior Director
Clinical Project Lead for RSV Older Adults
GSK
MedicalResearch.com: What is the background for this study?
Response: The AReSVi-006 phase III trial is designed to investigate the efficacy and safety of GSK’s respiratory syncytial virus (RSV) vaccine candidate for adults aged 60 years and above. The phase III trial is a randomized, placebo-controlled, double-blind, international trial with 24,966 participants who received either the investigational vaccine or placebo.
Hyuna Sung, PHD
Senior Principal Scientist, Cancer Surveillance Research
American Cancer Society
Kennesaw, GA 30144
Dr. Gulati[/caption]
Nicholas Gulati, MD, PhD
Director, Early Detection of Skin Cancer and Oncodermatology Clinic
The Kimberly and Eric J. Waldman
Department of Dermatology
Mount Sinai Health System
New York, New York
MedicalResearch.com: What is the background for this study? What is dupilumab primarily used for?
Response: Dupilumab is a monoclonal antibody that inhibits a specific part of the immune system known as Th2 cells, which are important in the development of various diseases including atopic dermatitis (eczema) and asthma. Therefore, dupilumab has become one of the major treatments for these conditions. Given the increasing use of this drug, it is important to understand the safety of it in terms of cancer development, as that is currently largely unknown.
Prof. Myeong-Ki Hong[/caption]
MedicalResearch.com Interview with:
Myeong-Ki Hong, MD PhD
Professor of Cardiology
Yonsei University College of Medicine
Severance Cardiovascular Hospital
Seoul, Korea
MedicalResearch.com: What is the background for this study?
Response: The background of this study was to compare the long-term clinical outcomes between the two distinct strategies regarding statin intensity in patients with coronary artery disease (CAD).
One is to titrate statin intensity to meet a target low-density lipoprotein cholesterol (LDL-C) level (treat-to-target strategy), the other is to maintain high-intensity statin without a target goal (high-intensity statin strategy).
Dr. Lazarus[/caption]
Philip Lazarus, PhD
Boeing Distinguished Professor, Pharmaceutical Sciences
Professor, Dept of Pharmaceutical Sciences
College of Pharmacy and Pharmaceutical Sciences
Washington State University
Spokane, WA 99210
MedicalResearch.com: What is the background for this study?
Response: Smoking and tobacco use remains a major health issue. Smokers use cigarette over the course of the day because the levels of nicotine, the addictive agent in cigarettes and other forms of tobacco, dimmish with time in the bloodstream due to the breakdown of nicotine by enzymes in the body. By inhibiting the breakdown of nicotine in smokers, one would expect that the levels of nicotine would remain higher after smoking a single cigarette, and that these individuals may not require lighting up another cigarette so quickly, reducing the number of cigarettes smoked over the course of a day. This could have a profound effect on reducing the overall harm incurred from smoking or from using other forms of tobacco.
In a single previous study, smokers who used a CBD inhaler exhibited a 40% reduction in cigarette use. In addition, while cannabis users are often smokers, previous studies have indicated that they smoke less cigarettes than non-cannabis-using cigarette smokers. In previous studies published in 2021, we found that major cannabinoids present in cannabis like THC and CBD inhibit major metabolizing enzymes in our body, including several that are important in drug metabolism. We hypothesized that CBD and its major active metabolite, 7-hydroxy (OH)-CBD, may also be inhibiting one or more of the enzymes important in the metabolism (or breakdown) of nicotine.
Dr. Roca[/caption]
Anna Roca PhD
MRC Unit The Gambia at the London School of Hygiene and Tropical Medicine
Fajara, The Gambia
MedicalResearch.com: What is the background for this study?
Response: Context specific interventions are needed to decrease the high burden of severe neonatal morbidity and mortality in sub-Saharan Africa. Severe bacterial infections are a main cause of neonatal mortality in the continent. Oral intra-partum azithromycin is a cheap intervention easily scalable. Before embarking on this trial, we conducted a proof-of-concept trial that showed the intervention reduced maternal and neonatal bacterial carriage of the most prevalent bacteria causing neonatal sepsis in the continent.
Dr. Khullar[/caption]
Dhruv Khullar, M.D., M.P.P.
Director of Policy Dissemination
Physicians Foundation Center for Physician Practice and Leadership
Assistant Professor of Health Policy and Economics
Weill Cornell Medicine, NYC
MedicalResearch.com: What is the background for this study?
Response: From prior research, we know that there are racial/ethnic differences in the acute impact of COVID-19, including higher rates of hospitalization and death among Black and Hispanic individuals compared to white individuals. Less is known about whether there are differences in the rates or types of long COVID by race and ethnicity.
Alexia Aguilar[/caption]
Alexia Aguilar
Geisinger Commonwealth School of Medicine
Scranton, PA
MedicalResearch.com: What is the background for this study?
Response: Traditional antidepressants like Zoloft and Lexapro have three major drawbacks.