12 Jan Stem Cell Derived Growth Factors May Slow Progression of ALS
Posted at 22:15h
in ALS, Alzheimer's - Dementia, Author Interviews, JAMA, Multiple Sclerosis, Neurological Disorders, Stem Cells
[caption id="attachment_20586" align="alignleft" width="200"]
Prof. Dimitrios Karussis[/caption]
MedicalResearch.com Interview with:
Prof. Dimitrios Karussis M.D., Ph.D.
Professor of Neurology
Head, Multiple Sclerosis Center
Hadassah BrainLabs
Medical Research: What is the background for this study? What are the main findings?
Prof. Karussis: BrainStorm Cell Therapeutics is developing innovative, autologous stem cell therapies for highly debilitating neurodegenerative diseases such as Amyotrophic Lateral Sclerosis (ALS), Multiple Sclerosis (MS), and Parkinson’s Disease (PD). Our technology, NurOwn™ is a first-of-its-kind approach that induces autologous bone marrow-derived Mesenchymal Stem Cells (MSCs) to secrete Neurotrophic Growth Factors (NTFs). These MSC-NTF cells have been shown to be protective in several animal models of neurodegenerative diseases.
Data from the clinical trials described in the recent issue of the Journal of American Medicine – Neurology (JAMA Neurology), suggest that NurOwn can help patients with ALS. The two trials featured in the article, a phase 1/2 and a phase 2a, studied the transplantation NurOwn cells in ALS patients. These trials confirmed the excellent safety profile of NurOwn and suggest a clinically meaningful effect. The investigators used two well established clinical endpoints that measure disease activity in ALS, the Revised ALS Functional Rating Scale and Forced Vital Capacity, and were able demonstrate a slowing of disease activity in the period following treatment.
Dr. Fox[/caption]
MedicalResearch.com Interview with:
Dr. Caroline Fox, MD MPH
National Heart, Lung, and Blood Institute
Assistant Clinical Professor of Medicine
Harvard Medical School
Medical Research: What is the background for this study? What are the main findings?
Dr. Fox: There is evidence linking sugar sweetened beverages with obesity and type 2 diabetes. There is also evidence suggesting that specific adipose tissue depots may play a role in the pathogenesis of these diseases. We found that higher levels of 
Dr. Aaron Dawes[/caption]
MedicalResearch.com Interview with:
Aaron J. Dawes, MD
Fellow, VA/RWJF Clinical Scholars Program
Division of Health Services Research
University of California Los Angeles
Los Angeles, CA 90024
Medical Research: What is the background for this study? What are the main findings?
Dr. Dawes: We reviewed the published literature to answer three basic questions about bariatric surgery and mental health conditions.
First, how common are mental health conditions among patients being referred for or undergoing bariatric surgery?
Dr. Diana Schendel[/caption]
MedicalResearch.com Interview with:
Diana Schendel, Professor MSO
Department of Public Health
Institute of Epidemiology and Social Medicine and
Department of Economics and Business National Centre for Register-based Research
Aarhus University
Denmark
Medical Research: What is the background for this study? What are the main findings?
Dr. Schende: Elevated mortality has been reported in persons with autism spectrum disorder (ASD), especially with comorbid epilepsy and intellectual disability. The effect of comorbidity on the risk for mortality in ASD, however, has not been rigorously examined in large, population-based studies. Our study aim was to investigate ASD mortality patterns overall and to assess the specific effects of comorbid mental, behavioral, and neurologic disorders on ASD mortality into young adulthood. Our study comprised a nation-wide Danish cohort of 1.9 million children of whom 20,492 were diagnosed with ASD. We observed 68 deaths in persons with ASD; 83% of the persons with ASD who died had comorbid mental/behavioral or neurologic disorders. The risk for mortality was two-fold higher in persons with 
Dr. Boolbol[/caption]
MedicalResearch.com Interview with:
Susan K. Boolbol, MD, FACS
Chief, Division of Breast Surgery
Chief, Appel-Venet Comprehensive Breast Service
Co-Director, Breast Surgery Fellowship
Mount Sinai Beth Israel
Associate Professor of Surgery
Icahn School of Medicine at Mount Sinai
New York, NY 10003
Medical Research: What is the background for these new recommendations?
Dr. Boolbol: To make this final recommendation, the Task Force conducted a comprehensive review of the science since its 2009 recommendation and considered the public comments it received on its 2015 draft recommendation statement. Based on all of this, the task force issued their recommendations.
Medical Research: What are the main changes from current guidelines?
Dr. Boolbol: Presently, there are several different guidelines and recommendations regarding screening mammography. Depending on the group issuing the guidelines, the recommendations vary from 
Dr. Victor Serebruany[/caption]
MedicalResearch.com Interview with:
Dr. Sergio Acuna[/caption]
MedicalResearch.com Interview with:
Sergio A. Acuna, MD
Graduate Student at St. Michael's Hospital and IHPME
University of Toronto
Medical Research: What is the background for this study?
Dr. Acuna: Solid organ transplant recipients are known to be at greater risk of developing cancers compared to the general population; however, because they are also at high increased risk of mortality from non-cancer causes, the risk of cancer morality in this population is unclear. As previous studies on this topic have reported disparate findings, the cancer mortality risk in this population remained uncertain.
Medical Research: What are the main findings?
Dr. Acuna: Our study provides conclusive evidence that solid organ transplant recipients are at increased risk of cancer mortality. Our findings demonstrate that solid organ transplant recipients are at increased risk of cancer death compared to the general population regardless of age, transplanted organ, and year of transplantation, and indicate cancer is a substantial cause of death in this population.
MedicalResearch.com Interview with:
Daniel Steffens, Ph.D.
The George Institute for Global Health
The University of Sydney
Medical Research: What is the background for this study?
Dr. Steffens: Back pain is a leading cause of disease burden globally. At present, a variety of interventions, such as getting a 
Dr. Green[/caption]
MedicalResearch.com Interview with:
Ariel R. Green, M.D., M.P.H
Assistant Professor of Medicine
Johns Hopkins University School of Medicine
Medical Research: What is the background for this study? What are the main findings?
Response: Implantable cardioverter-defibrillators (ICDs) are widely used to prevent sudden cardiac death in patients with systolic heart failure. Older adults with heart failure often have multiple coexisting conditions and are frail, increasing their risk of death from non-cardiac causes. Our understanding of outcomes in older patients with ICDs is limited.
Medical Research: What should clinicians and patients take away from your report?
Response: Our major finding was that more than 10% of patients currently receiving ICDs for primary prevention of sudden cardiac death (meaning that they have never had a potentially lethal arrhythmia but are at risk for one, usually due to systolic heart failure) are frail or have dementia. Patients with these geriatric conditions had substantially higher mortality within the first year after ICD implantation than those without these conditions. 
Dr. Sam Most[/caption]
MedicalResearch.com Interview with:
Sam P. Most, M.D., F.A.C.S.
Professor, Departments of Otolaryngology-Head & Neck Surgery and Surgery (Division of Plastic Surgery, by courtesy)
Chief, Division of Facial Plastic & Reconstructive Surgery
Stanford University School of Medicine
Stanford, CA
Medical Research: What is the background for this study? What are the main findings?
Dr. Most: Insurance companies often require patients to try a 6 or more week treatment with nasal steroids prior to allowing nasal surgery to proceed. This is true even in cases of physician-documented severe or extreme anatomic nasal obstruction that we know will not respond to medical therapy. We sought to examine this from a cost and quality-of-life perspective.
We found that while the up-front cost of surgery is obviously much higher than medical therapy, when viewed from an effect on improvement of quality of life (or lack thereof, in the case of medical therapy), the surgical therapy became more cost effective as years passed by.
Dr. Svetlana Popova[/caption]
MedicalResearch.com Interview with:
Svetlana Popova, M.D., Ph.Ds., M.P.H.
Senior Scientist
Social and Epidemiological Research, Centre for Addiction and Mental Health
Associate Professor, Epidemiology Division, Dalla Lana School of Public Health, University of Toronto
Associate Professor, Factor-Inwentash Faculty of Social Work, University of Toronto
Graduate Faculty Associate Member, Institute of Medical Science, University of Toronto
MedicalResearch: What is the background for this study?
Dr. Popova: It has been known for many years that prenatal alcohol exposure is associated with a number of adverse health consequences for both the mother and developing fetus. Women who consume alcohol during pregnancy place their child(ren) at risk of developing Fetal Alcohol Spectrum Disorders (FASD), and can experience a number of other adverse pregnancy outcomes including stillbirth, spontaneous abortion, premature birth, intrauterine growth retardation, and low birth weight. It was also previously known that people with FASD have many comorbidities (the simultaneous presence of two or more chronic diseases or conditions in a patient) due to the permanent damage of prenatal alcohol exposure on the fetus. However, until now it was unknown how many and what type of diseases, and at what frequencies they occur. Therefore, we reviewed the medical and epidemiological literature to identify the disease conditions that have been found to occur in people with 
Kawther Hashem[/caption]
MedicalResearch.com Interview with:
Kawther Hashem MSc RNutr (Public Health)
Nutritionist and Researcher
Action on Sugar
Wolfson Institute of Preventive Medicine,
Queen Mary, University of London
London UK
Medical Research: What is the background for this study? What are the main findings?
Response: The calculations showed that a 40% reduction in free sugars added to Sugar Sweetened Beverages (SSBs) over five years would lead to an average reduction in energy intake of 38 kcal per day by the end of the fifth year. This would lead to an average reduction in body weight of 1.20kg in adults, resulting in a reduction in overweight and obese adults by approximately half a million and 1 million respectively. This would in turn prevent between 274,000-309,000 obesity-related type 2 diabetes over the next two decades. Policies such as this will reduce cases of overweight and obesity and type 2 diabetes, this will have a major clinical impact and reduce healthcare costs.
Dr. Dalane Kitzman[/caption]
MedicalResearch.com Interview with:
Dalane W. Kitzman, M.D.
Professor, Cardiology
Sticht Center on Aging
Gerontology and Geriatric Medicine
Translational Science Institute
Wake-Forest Baptist Health
Winston-Salem, NC
Medical Research: What is the background of the research?
Dr. Kitzman: Heart Failure With Preserved Ejection Fraction (HFPEF) is a relatively recently recognized disorder. It used to be thought that it was rare. However, we now realize that HFPEF is the dominant form of heart failure in America. It is also the fastest growing cardiovascular disorder. Interestingly, this disorder occurs almost exclusively among older persons, particularly women. The need is great because outcomes in persons with HFPEF (death, rehospitalization, health care costs) are worsening. This stands in contrast to most other cardiovascular disorders which are on the decline and / or are experiencing greatly improved outcomes. Remarkably, all of the large studies that have used medications in HFPEF that seemed they should be ‘sure bets’ showed no benefit for their primary outcomes. Thus, this is also the only major cardiovascular disorder where there is no proven medication treatment. That means physicians take ‘educated guesses’ in choosing treatment for this large group of patients.
The main symptom in patients with chronic HFPEF is shortness of breath and and fatigue with exertion. We showed in 2002 in JAMA that when we objectively measured this symptom with expired gas analysis (Peak VO2), this was as severely decreased in HFPEF as in patients with HFREF (severely reduced EF), the classic, well accepted form of heart failure. That and other studies helped lead to acceptance of HFPEF as a true Heart Failure disorder.
We first showed 5 years ago that 4 months of exercise training improves peak VO2 and quality of life in patients with HFPEF. In fact, exercise remains the only proven means to improve these patients’ chronic symptoms.
The goal of our study was to determine if weight loss diet also improved peak VO2 and quality of life in HFPEF patients, alone and in combination with exercise training. This was based on the under-recognized fact that over 80% of Heart Failure With Preserved Ejection Fraction patients are overweight or obese. It was already known that weight loss diet in other groups of older persons improves peak VO2 and quality of life. And small studies of 
Dr. Waters[/caption]
MedicalResearch.com Interview with:
Michael F. Waters, MD, PhD
Department of Neurology
Department of Neuroscience
McKnight Brain Institute
University of Florida College of Medicine
Gainesville, Florida
Medical Research: What is the background for this study? What are the main findings?
Dr. Waters: This study was based on a subgroup of medically managed patients with severe, symptomatic, intracranial, atherosclerotic disease. Historically we know that these patients have a very high rate of additional strokes, and multiple studies have attempted to determine the best management for these patients. In SAMMPRIS, we were able to prove that aggressive medical management was superior to stenting in these patients. However, certain patients in the medically managed group still had a very high rate of repeat strokes, as much as 30% of those with certain risk factors. This study was an attempt to identify those risk factors to determine which patients were at the greatest risk for another stroke.
Dr. Charlton[/caption]
MedicalResearch.com Interview with:
Brittany M. Charlton, ScD
Instructor
Boston Children's Hospital and Harvard Medical School
Researcher, Harvard Chan School Department of Epidemiology
Boston, MA 02115
Medical Research: What is the background for this study? What are the main findings?
Dr. Charlton: Even though oral contraceptives can be over 99% effective with perfect use, almost 10% of women become pregnant within their first year of use. Many more women will stop using oral contraceptives when planning a pregnancy and conceive within just a few months. In both of those examples, a woman may inadvertently expose her offspring during pregnancy to exogenous sex hormones. We conducted a nationwide cohort study in Denmark in order to investigate whether oral contraceptive use shortly before or during pregnancy was associated with an increased risk of major birth defects in the offspring. Our main finding was that there was no increased risk of having a birth defect associated with oral contraceptive exposure. These results were also consistent when we broke down the birth defects into different subgroups, like limb defects.
Dr. Stefan Verlohren[/caption]
Stefan Verlohren, MD, PhD
Consultant and Senior Lecturer
Maternal-Fetal Medicine
Klinik für Geburtsmedizin / Department of Obstetrics
Charité Campus Mitte
Berlin
Medical Research: What is the background for this study? What are the main findings?
Dr. Verlohren: Preeclampsia affects 2–5% of pregnancies worldwide, and is a potentially life threatening syndrome for both mother and child. Treatment options for preeclampsia are very limited, with delivery being the only ‘cure’; however, early detection and monitoring are beneficial for improving maternal and fetal outcomes. Development of preeclampsia is very difficult to predict: its clinical presentation is variable and its signs and symptoms overlap with other conditions. There has been an unmet medical need for improved prediction of preeclampsia, i.e. predicting which women will develop 
Dr. Mary Hawn[/caption]
MedicalResearch.com Interview with:
Dr. Mary Hawn MD MPH
Chair, Department of Surgery
Stanford School of Medicine
Stanford, California
Medical Research: What is the background for this study? What are the main findings?
Dr. Hawn: Patients with known coronary artery disease are at higher risk for adverse cardiac events in the peri-operative period. Revascularization with coronary stents does not appear to mitigate this risk and in fact, may elevate the risk if surgery is in the early post-stent period. Drug eluting stents pose a particular dilemma as these patients require 12 months of dual anti platelet therapy to prevent stent thrombosis, thus elective surgery is recommended to be delayed during this period. In contrast, bare metal stents with early epithilialization are not at the same risk for stent thrombosis with anti platelet cessation. In our retrospective cohort study, however, we observed that stent type was not a major driver of adverse events in the early post-stent period and that underlying cardiac disease and acuity of the surgery explained most of the risk. We undertook this study to determine the influence of the underlying indication for the stent procedure on surgical outcomes over time following the stent.
Josephine Mollon[/caption]
MedicalResearch.com Interview with:
Josephine Mollon MSc
Department of Psychosis Studies
Institute of Psychiatry, Psychology, and Neuroscience
King’s College London
London, England
Medical Research: What is the background for this study? What are the main findings?
Dr. Mollon: Psychotic symptoms, such as hallucinations and delusions, are core features of psychotic disorders. A significant minority of the general population also reports subclinical psychotic experiences. Evidence suggests that these experiences may lie on a continuum with clinically significant psychotic symptoms. For example, cognitive deficits, which are a hallmark of psychotic disorders, are also seen in people with subclinical psychotic experiences. We used population-based survey data to characterize cognitive functioning in adults with psychotic experiences while adjusting for important sociodemographic characteristics and investigating the effect of age.
The 171 (9.7%) adults with psychotic experiences showed significant memory and verbal deficits, but not IQ or processing speed deficits. Only participants 50 years and older with psychotic experiences showed medium to large impairments in general IQ, verbal knowledge, working memory and memory after adjusting for socioeconomic status, cannabis use, and common mental disorders.
Dr. Sandeep Kumar[/caption]
MedicalResearch.com Interview with:
Sandeep Kumar, MD
Assistant Professor of Neurology
Harvard Medical School
Director, Inpatient Stroke Service
Department of Neurology, Stroke Division
Beth Israel Deaconess Medical Center
Boston, MA 02215
Medical Research: What is the background for this study? What are the main findings?
Dr. Kumar: Transient deficits that start suddenly and typically last for a few minutes to a few hours are the hallmark of a transient ischemic attack (TIA) or a minor ischemic stroke. In this single-center observational study, we have reported similar clinical presentation in some patients with intracerebral hemorrhage (ICH) that are difficult to distinguish from cerebral ischemia based only on clinical signs and symptoms.
Tara Gomes[/caption]
Medical Research: What is the background for this study? What are the main findings?
Response: Surveillance of the harms associated with chronic opioid use is imperative for clinicians and policy-makers to rapidly identify emerging issues related to this class of medications. However, data regarding opioid-related deaths is difficult to obtain in Canada as it is collected by local coroners and is not widely available to researchers. We conducted a validation study to evaluate whether regularly collected vital statistics data collected by Statistics Canada can be used to accurately identify opioid-related deaths in Canada.
We compared deaths identified from charts abstracted from the Office of the Chief Coroner of Ontario to those identified using several coding algorithms in the Statistics Canada Vital Statistics database. We found that the optimal algorithm had a sensitivity of 75% and a positive predictive value of 90%. When using this algorithm, the death data obtained from the Vital Statistics database slightly underestimated the number of opioid-related deaths in Ontario, however the trends over time were similar to the data obtained from the coroner’s office.
Dr. Trinh[/caption]
MedicalResearch.com Interview with:
Quoc-Dien Trinh MD
Assistant Professor, Harvard Medical School
Brigham and Williams Hospital
Medical Research: What is the background for this study? What are the main findings?
Dr. Trinh: Among elderly Medicare beneficiaries with metastatic prostate cancer, surgical castration is associated with lower risks of any fractures, peripheral arterial disease, and cardiac-related complications compared to medical castration using GnRH agonists.
Dr. David Gallego Ortega PhD[/caption]
MedicalResearch.com Interview with:
David Gallego Ortega, PhD
Group Leader, Tumour Development Group Cancer Division
Garvan Institute of Medical Research
Conjoint Lecturer, St Vincent’s Clinical School, Faculty of Medicine, UNSW, Australia
National Breast Cancer Foundation and Cure Cancer Foundation Australia Fellow
Medical Research: What is the background for this study? What are the main findings?
Dr. Ortega: We have identified a protein that 'goes rogue’ in breast cancer. The protein, called Elf5, ‘tricks' the immune system producing inflammation so that the immune cells now help the breast cancer cells to spread throughout the body.
Cancer spread, or metastasis, is the ultimate cause of death of 
Dr. Gágyor[/caption]
MedicalResearch.com Interview with:
Ildikó Gágyor MD
Senior researcher in primary care
Department of General Practice
University Medical Center Göttingen
Göttingen, Germany
Medical Research: What is the background for this study? What are the main findings?
Dr. Gágyor: Uncomplicated urinary tract infection is a common problem for women. Affected patients are usually treated with antibiotics to combat both unpleasant symptoms and to combat infection. However, prescription of antibiotics for a self-limiting condition, contributes to increased resistance rates posing a serious long-term threat to public health. In a double blind randomised controlled trial we examined whether symptomatic treatment of uncomplicated urinary tract infection with ibuprofen reduces the rate of antibiotic prescriptions without a significant increase in symptoms, recurrences, or complications. In all, 494 women were randomly assigned to receive: either ibuprofen for three days and antibiotics only if symptoms are persistent; or antibiotic treatment with fosfomycin. Results showed that antibiotic use could be reduced significantly: of the 248 women in the ibuprofen group two thirds recovered without antibiotics and one third received antibiotics subsequently. Women in the ibuprofen group had a higher symptom burden but in both groups, symptoms decreased within the first week (Figure 1). Six cases of pyelonephritis occurred, one in the fosfomycin group, five in the ibuprofen group.
Dr. Eberth[/caption]
MedicalResearch.com Interview with:
Jan Marie Eberth, PhD
Assistant Professor, Department of Epidemiology and Biostatistics
Deputy Director, SC Rural Health Research Center
Core Faculty, Statewide Cancer Prevention and Control Program
Arnold School of Public Health
University of South Carolina
Columbia, SC 29208
Medical Research: What is the background for this study?
Dr. Eberth: With the breakthrough findings of the National Lung Screening Trial released in 2011, professional organizations have largely embraced population-based screening guidelines for patients at high risk for