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    Home > Active Ingredient News > Study of Nervous System > Received US$5 million funding from the National Institutes of Health, Alzheimer’s disease gene therapy advances to the clinic

    Received US$5 million funding from the National Institutes of Health, Alzheimer’s disease gene therapy advances to the clinic

    • Last Update: 2021-06-30
    • Source: Internet
    • Author: User
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    Recent popular reports from Yimaike ★Dry goodsIn-depth report on cell gene therapy outsourcing service industry ★Towards the ultimate cure Analysis of the clinical progress of AAV gene therapy Click on the picture to register now June 17, 2021 / Yimaike News eMedClub News/--Previous section Over time, the US FDA has been out against the crowd and approved aducanumab, the first new treatment for Alzheimer's disease in nearly 20 years, which has aroused many discussions
    .

    For many years, Alzheimer's disease (AD) has been one of the most active areas for new drug research and development.
    Numerous pharmaceutical companies and research institutions have deployed in this field, trying to break through this difficulty from different tracks
    .

    Recently, the National Institutes of Aging (NIA) of the National Institutes of Health (NIH) provided a five-year funding of up to $5 million for a clinical trial to promote the trial's entry into Phase 1 clinical trials
    .

    The trial will use an investigational gene therapy AAV2-BDNF to treat AD or mild cognitive impairment
    .

     AAV2-BDNF is an AAV gene therapy product developed by the University of California, San Diego (UCSD).
    The purpose of this trial is to use engineered AAV2 to deliver the gene BDNF to cells in specific areas of the brain, prompting cells to "read" Gene and produce BDNF protein, thereby improving the patient's symptoms
    .

    BDNF is the abbreviation of brain-derived neurotrophic factor, a growth factor that exists in the brain and other parts of the nervous system
    .

    As the name suggests, the signal sent by this protein can support the continued survival of mature neurons, promote the growth of new neurons, and promote the formation of new synapses (connections between individual neurons)
    .

    Recommended reading: AAV delivers brain-derived neurotrophic factor, Alzheimer's gene therapy initiates a phase 1 clinical trialYi Mai Meng broke the news that Professor of Neuroscience and Director of the Institute of Translational Neuroscience, University of California San Diego (UCSD) School of Medicine, Lead researcher Dr.
    Mark Tuszynski mentioned in the school’s press conference: “We found in early animal model studies that the delivery of BDNF to the part of the brain that was first affected by Alzheimer’s disease, namely the entorhinal cortex and hippocampus, can It reverses the loss of connections between neurons and prevents ongoing cell degeneration
    .

    ” Tuszynski pointed out that choosing to deliver BDNF to the brain is more effective than choosing nerve growth factor (NGF) because BDNF can act on the patient's degenerated neural circuits
    .

    In addition, the new delivery method can also deliver and distribute BDNF more effectively
    .

    NGF is the most important member of the nerve growth factor family, and NGF has been used in gene therapy for the treatment of AD before
    .

    UCSD researchers will test the therapy in a three-year phase 1 clinical trial
    .

    In the current experimental design, 12 people diagnosed with Alzheimer's disease or mild cognitive impairment will receive investigative treatment, and the other 12 will be used as controls for comparison
    .

    The trial is currently still recruiting subjects
    .

     In addition to the National Institutes of Health's NIA, the trial also received funding from the Alzheimer's Association and philanthropist Darlene Shiley
    .

     About Alzheimer's disease (AD)▲ Normal brain (left) and AD patient brain (right) (Source: SciencePhotoLibrary) AD is the most common type of dementia in the elderly and ranks among the top ten global disease burdens
    .

    Traditional AD treatment drugs are mainly based on symptomatic treatment, and cannot treat the root cause of AD
    .

    In addition to monoclonal antibody therapy, cell therapy and gene therapy also have their own place in the development of new drugs for AD
    .

     For more details, scan the QR code for a complete reading.
    The AD new drug track is still highly competitive
    .

    Although it is far from 2019 that Lugu Pharmaceutical’s new AD drug Phase 1 (Mannate Sodium Capsules) has been conditionally approved in China in 2019, and Aducanumab has recently been listed, the quantitative standards for the treatment of AD are still divergent.
    I hope that more new models will be introduced in the future.
    Therapies can have the opportunity to go to the clinic and to the market, bringing new hope to millions of patients
    .

    Reference materials: 1.
    https://alzheimersnewstoday.
    com/2021/06/14/5m-grant-supports-phase-1-trial-of-aav2-bdnf-potential-gene-therapy/?cn-reloaded=1Mak has always been committed to original news reports on cutting-edge technologies, industry trends, and industry insights in bio-innovative drugs.
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