Публикация участника Betsy Pilon
Dr Pia Wintermark is truly Hope for HIE embodied. I have had the pleasure of working with her for a few years in various ways. She’s an absolute force of science — in all ways! Learn about her work, and her relentless pursuit through many obstacles, to accelerate science and hope for HIE.
Associate Professor of Pediatrics chez McGill University
https://lnkd.in/eDZkeumM The science: So happy and proud that this paper is published. SANE-01 is the first trial trying to REPAIR brain damage of babies with HIE, and not only prevent them. All the enrolled babies had brain damage before medication was started and we wanted first to ensure that it was feasible and safe to give a medication enterally for repairing their brain. The medication is sildenafil, most commonly known under the name of Viagra, and while it has many well-known effects in the adult world, the effects that pick my interest for babies was its role in preventing death of brain cells (apoptosis), promoting growth of new brain cells (neurogenesis), decreasing inflammation in the brain (neuroinflammation), and promoting specialization of brain cells (myelination). Our sample size is small, but our results are encouraging: -Among surviving neonates, partial recovery of injury, fewer cystic lesions, and less brain volume loss on day-30 MRI was present in 71% (5/7) in sildenafil group and in 0% (0/3) in placebo group -Rate of death or survival to 18-month with severe neurodevelopmental impairment was 57% (4/7) in sildenafil group and 100% (3/3) in placebo group. Now more studies are needed to prove efficacy. #Neurorestoration #RepairBabiesBrain #SildenafilForHIE #MoreToCome The story behind the science: Sometimes often research in academics takes long time. Got the idea the first time while I was a resident in Switzerland, where I was told I was too enthusiastic and should probably continue my journey abroad. Started the imaging study and got trained for animal work while I was in Boston. Then started the lab experiments when I moved to Montreal in 2010. Finally enrolled the first patient in this trial in 2016 1 week after having my own little one. 7 years later, after two cancers, two surgeries, a divorce, raising a toddler as a single mom, covid, clinical duties, and while running a second phase Ib study to find optimal dose (SANE-02, writing ongoing) and launching a similar study in Uganda (SANE-Uganda, enrollment ongoing), this first study is finally published! An accomplishment in itself! #Resilience #NeverGiveUp #WomenInSTEM Many thanks are in order for all the help and support received along the way. Thanks to the parents of these babies who trusted us despite being overwhelmed in the moment, thanks my fabulous NICU team B6S who recorded so many vital signs, and thanks to Anie Lapointe, Walter Haefeli, Robin Steinhorn, Gabriel Altit, Emmanouil Rampakakis, Patrick Colin and many others for your support all along. With the support of Instituts de recherche en santé du Canada | Canadian Institutes of Health Research, Fonds de recherche du Québec, Fondation Courtois, La Fondation de l’Hôpital de Montréal pour enfants, IR-CUSM | Institut de recherche du CUSM | #ircusm#ircusm, Hôpital de Montréal pour enfants | Montreal Children’s Hospital
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Enhancing Progressive Multiple Sclerosis Treatment with Abzyme Antibodies In his recently published book, Stephen Hauser, the director of the Weill Institute for Neurosciences and a neurology professor at the University of California, San Francisco, writes “The immune system, designed to protect us against invading microbes, misidentifies nerve cells and their myelin coverings as foreign. This leads to an autoimmune attack — the body turning against itself.” In addition to exploring the genetic roots of MS, Hauser and his team identified the critical role of B cells in attacking nerve cells, leading to the development of immunosuppressive drugs that target and destroy the B cells, thus reducing inflammation, slowing down nerve damage, and easing symptoms. These B-cell therapies were the first to be effective in treating progressive MS and have been enormously beneficial for those living with the disease. Instead of killing all antibody-producing B cells, Abzyme developed antibodies that block antibody-mediated complement activation. In the MS mouse model, we have shown that our antibodies Improve the neurological disability caused by multiplesclerosis. For more information, please contact [email protected] #multiplesclerosis #MS #abzymetx #antibodies #neurodegenerativediseases
Book Review: A Physician’s Quest to Treat Multiple Sclerosis
https://undark.org
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This morning I watched BBC breakfast time TV and grew increasing sad and frustrated with the interview with Jackie Steward taking about the devastating impact Alzheimer’s has on loved ones and the need for more Alzheimer’s research and support. The reason for my frustration is that just over a year ago, some innovative Alzheimer’s research was performed which believes it has identified the cause of Alzheimer’s and derived a potential treatment (and possibly a vaccine). This research goes against a lot of current thinking, where many current treatments are targeting the plaques seen in Alzheimer sufferers which is akin to applying calamine lotion to the spots caused by measles, that is addressing the consequence and not the source. At the time this was publicized widely including on LinkedIn by one of the Dragons’ Den team Tej Lalvani “POSITIVE DEVELOPMENT: A breakthrough Alzheimers jab could restore patients’ memories”. The paper (Bakrania, P., et al. (2021) Discovery of a novel pseudo β-hairpin structure of N-truncated amyloid-β for use as a vaccine against Alzheimer’s disease. Molecular Psychiatry. https://lnkd.in/ecE9iF7u) was in the top 1% of medical and life science papers published on Frontiers in Immunology’ read on their web site. My frustration is that nothing has happened with this research work since it was published as it has not been possible to find a pharmaceutical partner to carry out humans clinical trials. Based on the gentlemen, who earlier last year travelled with no appointment, from Italy to Leicester University to volunteer for trials, I think there will be no shortage of volunteers. The pandemic showed what can be done when the correct will, money and resources are in place for clinical trials. I accept that this work may not transfer from mice to human, but shouldn’t we find this out sooner rather that later, because imagine a world where this does translate … #alzheimers #alzheimersresearch
Discovery of a novel pseudo β-hairpin structure of N-truncated amyloid-β for use as a vaccine against Alzheimer’s disease – Molecular Psychiatry
nature.com
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November is Alzheimer’s Awareness Month, a time to acknowledge and support the tens of millions of people living with AD worldwide! More than 1000 structures around the world will shine the color teal, shown to be a #calming color. Importantly, AD is not a normal part of aging, but rather a progressive #brain disorder. Medical success stories are beginning; opportunities for more efficacious therapies, greater efficiency, and earlier detection abound. A recent report from the laboratory of Antonia Gutierrez [CIBERNED – Centro de Investigación Biomédica en Red] highlights the revolution offered by the concomitant use of transgenic mice and omics technologies that may lead to more rapid discovery coming from translational models (1). Sarah Gregory, Stina Saunders, and Craig Ritchie [Western General Hospital, The University of Edinburgh] advocate for shifting research to earlier disease stages to improve brain health outcomes and help to keep affected individuals dementia-free for as long as possible (2). Research at Hebei University of Technology applied the ECGenie to show that mice with AD have abnormal autonomic nervous system regulation manifest by significantly reduced #heart rate variability (HRV) even in early stages (3). Does correcting HRV mitigate symptoms of AD, in mice? In humans? As a primary, or adjunct therapy with some of the promising new drugs, whether #immunotherapy , cholinesterase inhibitors, or glutamate regulators? Mouse Specifics, Inc. is committed to diagnosing and treating AD; organizations with a published history in AD who cite this post will receive a 10% rebate from their purchase of instrumentation to support their #preclinicalresearch , and a travel scholarship of $500 to present ECG #digitalbiomarkers of AD at a scientific meeting. The ECGenie: Straight from the heart! https://lnkd.in/dd3f8anU To wit, researchers from University of Copenhagen (Københavns Universitet) recently identified several common ECG markers which were associated with AD, and which improved risk prediction for #alzheimersdisease (4). #wellness #data #innovation #3rs #healthcare References: 1. Raquel Sanchez Varo, et al. Transgenic Mouse Models of Alzheimer’s Disease: An Integrative Analysis. Int J Mol Sci. 2022 May 12;23(10):5404. 2. Gregory S, Saunders S, Ritchie CW. Science disconnected: the #translationalresearch gap between basic science, clinical trials, and patient care in Alzheimer’s disease. Lancet Healthy Longev. 2022 Nov;3(11):e797-e803. 3. 耿读艳, et al. Effects of Alzheimer’s disease of varying severity on cardiac and autonomic function. Braz J Med Biol Res. 2022 Jan 5;55:e11504. https://lnkd.in/eG9hUG3f 4. Jonas Isaksen, et al, et Jørgen K. Kanters. Association between primary care electrocardiogram markers and Alzheimer’s disease. J Neurol Sci. 2023 Apr 15;447:120581.
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📃Scientific paper: Developmental deficits and staging of dynamics of age associated Alzheimer’s disease neurodegeneration and neuronal loss in subjects with Down syndrome Abstract: The increased life expectancy of individuals with Down syndrome (DS) is associated with increased prevalence of trisomy 21–linked early-onset Alzheimer’s disease (EOAD) and dementia. The aims of this study of 14 brain regions including the entorhinal cortex, hippocampus, basal ganglia, and cerebellum in 33 adults with DS 26–72 years of age were to identify the magnitude of brain region–specific developmental neuronal deficits contributing to intellectual deficits, to apply this baseline to identification of the topography and magnitude of neurodegeneration and neuronal and volume losses caused by EOAD, and to establish age-based staging of the pattern of genetically driven neuropathology in DS. Both DS subject age and stage of dementia, themselves very strongly correlated, were strong predictors of an AD-associated decrease of the number of neurons, considered a major contributor to dementia. The DS cohort was subclassified by age as pre-AD stage, with 26–41-year-old subjects with a full spectrum of developmental deficit but with very limited incipient AD pathology, and 43–49, 51–59, and 61–72-year-old groups with predominant prevalence of mild, moderately severe, and severe dementia respectively. This multiregional study revealed a 28.1% developmental neuronal deficit in DS subjects 26–41 years of age and 11.9% AD-associated neuronal loss in DS subjects 43–49 years of age; a 28.0% maximum neuronal loss at 51–59 years of age; and a 11.0% minimum neuronal loss at 61–72 y. Discover the rest of the scientific article on es/iode ➡️https://etcse.fr/BsiAQ #alzheimer #science #health
Developmental deficits and staging of dynamics of age associated Alzheimer’s disease neurodegeneration and neuronal loss in subjects with Down syndrome
ethicseido.com
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📃Scientific paper: Developmental deficits and staging of dynamics of age associated Alzheimer’s disease neurodegeneration and neuronal loss in subjects with Down syndrome Abstract: The increased life expectancy of individuals with Down syndrome (DS) is associated with increased prevalence of trisomy 21–linked early-onset Alzheimer’s disease (EOAD) and dementia. The aims of this study of 14 brain regions including the entorhinal cortex, hippocampus, basal ganglia, and cerebellum in 33 adults with DS 26–72 years of age were to identify the magnitude of brain region–specific developmental neuronal deficits contributing to intellectual deficits, to apply this baseline to identification of the topography and magnitude of neurodegeneration and neuronal and volume losses caused by EOAD, and to establish age-based staging of the pattern of genetically driven neuropathology in DS. Both DS subject age and stage of dementia, themselves very strongly correlated, were strong predictors of an AD-associated decrease of the number of neurons, considered a major contributor to dementia. The DS cohort was subclassified by age as pre-AD stage, with 26–41-year-old subjects with a full spectrum of developmental deficit but with very limited incipient AD pathology, and 43–49, 51–59, and 61–72-year-old groups with predominant prevalence of mild, moderately severe, and severe dementia respectively. This multiregional study revealed a 28.1% developmental neuronal deficit in DS subjects 26–41 years of age and 11.9% AD-associated neuronal loss in DS subjects 43–49 years of age; a 28.0% maximum neuronal loss at 51–59 years of age; and a 11.0% minimum neuronal loss at 61–72 y. Discover the rest of the scientific article on es/iode ➡️https://etcse.fr/BsiAQ #alzheimer #science #health
Developmental deficits and staging of dynamics of age associated Alzheimer’s disease neurodegeneration and neuronal loss in subjects with Down syndrome
ethicseido.com
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📃Scientific paper: Developmental deficits and staging of dynamics of age associated Alzheimer’s disease neurodegeneration and neuronal loss in subjects with Down syndrome Abstract: The increased life expectancy of individuals with Down syndrome (DS) is associated with increased prevalence of trisomy 21–linked early-onset Alzheimer’s disease (EOAD) and dementia. The aims of this study of 14 brain regions including the entorhinal cortex, hippocampus, basal ganglia, and cerebellum in 33 adults with DS 26–72 years of age were to identify the magnitude of brain region–specific developmental neuronal deficits contributing to intellectual deficits, to apply this baseline to identification of the topography and magnitude of neurodegeneration and neuronal and volume losses caused by EOAD, and to establish age-based staging of the pattern of genetically driven neuropathology in DS. Both DS subject age and stage of dementia, themselves very strongly correlated, were strong predictors of an AD-associated decrease of the number of neurons, considered a major contributor to dementia. The DS cohort was subclassified by age as pre-AD stage, with 26–41-year-old subjects with a full spectrum of developmental deficit but with very limited incipient AD pathology, and 43–49, 51–59, and 61–72-year-old groups with predominant prevalence of mild, moderately severe, and severe dementia respectively. This multiregional study revealed a 28.1% developmental neuronal deficit in DS subjects 26–41 years of age and 11.9% AD-associated neuronal loss in DS subjects 43–49 years of age; a 28.0% maximum neuronal loss at 51–59 years of age; and a 11.0% minimum neuronal loss at 61–72 y. Discover the rest of the scientific article on es/iode ➡️https://etcse.fr/pabX #alzheimer #science #health
Developmental deficits and staging of dynamics of age associated Alzheimer’s disease neurodegeneration and neuronal loss in subjects with Down syndrome
ethicseido.com
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Chief Advocacy, Services & Science Officer at the National MS Society | Connecting people, resources and strategy to create a world free of Multiple Sclerosis 5 мес.
Cures for diseases are found when people and organizations work together. The National MS Society is proud to partner with the American Brain Foundation, the American Academy of Neurology, the NFL Players Association, The Encephalitis Society, Gates Ventures, and the WoodNext Foundation on the Cure One, Cure Many award. Through this collaborative effort, we will fund cross-disciplinary research to understand and treat inflammation that affects brain health. This is a major and still poorly understood aspect of many brain diseases. Less brain inflammation means better brain health and better quality of life for millions who have a brain disease today and in the future. By working together we will leverage our collective strengths and unlock insights about brain inflammation that can unlock new treatments and cures that will benefit people across a number of brain diseases. Together we are stronger. #ms #multiplesclerosis https://lnkd.in/ez_68z4G
Research Initiative Established to Understand Neuroinflammation’s Impact on Brain Diseases
https://www.americanbrainfoundation.org
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📅 Today, February 29, 2024 is #RareDiseaseDay At the HIH, researchers like Prof. Simone Mayer and Prof. Matthis Synofzik focus on different rare diseases: Pontocerebellar hypoplasia type 2 (#PCH2) is a very serious #neurodevelopmental disorder in children. It is caused by a single #DNA building block being swapped. The tiny #genetic change, which only occurs in about one in a million people, causes a disruption in the development of certain areas of the brain, which leads to significant limitations in psychomotor development. Most affected children die before they reach adolescence. Prof. Simone Mayer, head of the “#Molecular #Brain #Development” research group, and her team are researching #brain #organoids that are created from donated skin cells from children affected by #PCH2a. These tissue structures can grow in three dimensions outside the human body and mimic the #cellular architecture and certain functional aspects of brain areas. Brain organoids give researchers insights into early brain development and the development of neurological and psychiatric diseases. Prof. Matthis Synofzik, head of the “#Translational #Genomics of #Neurodegenerative Diseases” section at the HIH, is leading a very unique treatment at the Tübingen Hertie Center for Neurology: For the first time in Europe, a #gene-based therapy is being administered that has been specifically tailored to the individual #genetic defect of a 5-year-old child: 5-year-old Poyraz suffers from ataxia telangiectasia, a very rare and serious disease that causes a progressive loss of the ability to walk and stand. Together with an international research team, Synofzik is taking an innovative path. The scientists produced antisense oligonucleotides (ASO) in the test tube, short pieces of RNA. The researchers created them so that they can lie exactly over the mutated area in the patient’s genome. This means that the cells can read the gene correctly again and the important protein is produced at normal levels. “This tailored, highly individualized approach is revolutionary in medicine,” says Matthis Synofzik. #RareDiseaseDay2024 #BrainResearch #Gehirnerforschen Gemeinnützige Hertie-Stiftung
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The Alex Manfull Fund is proud to announce our significant grant to the Pediatric Neuropsychiatry and Immunology Program at Massachusetts General Hospital. Dr. Kyle Williams will investigate the relationship between cytokines IL-17 and IL-23 and OCD symptoms by studying a population of OCD patients with comorbid autoimmune conditions (e.g., psoriasis and arthritis) who are receiving treatment with monoclonal antibody therapies (e.g., Cosentyx). This research seeks to demonstrate a link between immune dysfunction (e.g., autoimmunity) and psychiatric disorders (e.g., OCD) thus creating validation for researching the role of the immune system in other immune-mediated neuropsychiatric disorders such as PANDAS/PANS. Confirming such a connection could, eventually, have great implications for diagnosis and new treatment approaches for a subset of PANDAS/PANS that could move from bench to bedside more quickly. Be a part of our efforts to further understand the underlying mechanisms of immune mediated disorders. A gift of any size is welcome and appreciated. Click here: https://lnkd.in/einNRS9t #Pandaspans #panspandas #immunemediatedneuropsychiatricdisorders #panspandasresearch #ocdresearch #cytokines #psoriasis
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Biochemist & Cancer Researcher || Data Analyst || Excel || Power BI | R 3 мес.
🗞️Great Progress in Parkinson’s Research Scientists at Oxford’s Nuffield Department of Clinical Neurosciences have pioneered a blood-based test to identify the onset of Parkinson’s disease before symptoms manifest. This innovative test, highlighted in JAMA Neurology, assesses alpha-synuclein levels in extracellular vesicles, enabling early detection and potential screening for individuals at heightened risk. ✅Boasting a 90% accuracy in discerning high-risk cases, this advancement paves the way for timely precision therapies, addressing a key challenge in Parkinson’s clinical trials. 🧠💉 This is goodnews. #ParkinsonsInnovation #MedicalAdvancements #EarlyRecognition #PrecisionCare https://lnkd.in/d6gW5Mv4
Researchers develop a blood test to identify individuals at risk of developing Parkinson’s disease
ox.ac.uk
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