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    Home > Active Ingredient News > Study of Nervous System > Express treatment of Parkinson's disease, Bayer's innovative cell therapy and gene therapy enter clinical trials

    Express treatment of Parkinson's disease, Bayer's innovative cell therapy and gene therapy enter clinical trials

    • Last Update: 2021-06-17
    • Source: Internet
    • Author: User
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    ▎ WuXi AppTec content team editor On June 8, 2021, Bayer announced that its BlueRock Therapeutics (BlueRock) company has successfully used the DA01 dopaminergic neuron therapy produced by pluripotent stem cells in a phase 1 clinical trial.
    Transplanted to a patient with Parkinson's disease
    .

    At the same time, its subsidiary Asklepios Biopharmaceuticals (AskBio) is also advancing gene therapy for Parkinson's disease, and is currently recruiting and evaluating patients in an ongoing phase 1b clinical study
    .

    Parkinson's disease is the most common neurodegenerative movement disorder, affecting more than 10 million people worldwide
    .

    It is caused by damage to nerve cells in the brain, which leads to a decrease in the level of dopamine in the brain.
    Dopamine is a neurotransmitter involved in processes such as memory or exercise
    .

    The disease usually begins with one-handed tremor.
    Other symptoms include muscle stiffness, cramps, and movement disorders (involuntary twisting movements of the face, arms, legs, or trunk)
    .

    Dopamine substitutes are commonly used to reduce disease symptoms, such as levodopa, but as the disease progresses, its effect diminishes
    .

    By targeting the root causes of the disease, cell and gene therapy aims to go beyond the treatment model that relieves symptoms
    .

    Using real dopaminergic neurons, BlueRock aims to re-establish the innervation of the affected areas of the human brain, thereby reversing the degeneration process and potentially restoring the patient's motor function
    .

    BlueRock's clinical trial will enroll 10 patients in the United States and Canada
    .

    In this study, patients will undergo surgery to transplant dopamine-producing cells into the putamen, a deep brain structure affected by Parkinson's disease
    .

    The main purpose of this phase 1 clinical trial is to evaluate the safety and tolerability of DA01 cells after 1 year of transplantation, and the secondary purpose is to evaluate the survival of transplanted cells and the effect on exercise performance 1 and 2 years after transplantation, as well as therapy The long-lasting safety and tolerability
    .

    AskBio's gene therapy uses AAV vectors to deliver the gene encoding human glial cell-derived neurotrophic factor (GDNF) to neurons in the putamen, resulting in the expression and secretion of GDNF protein in brain regions affected by Parkinson's disease
    .

    Long-term experiments using AAV-GDNF in rodents and non-human primate models have shown that continuous expression of GDNF can promote brain neuron regeneration and significant motor recovery
    .

    AskBio's Phase 1b clinical study is currently recruiting and evaluating patients in the United States to evaluate its safety and preliminary efficacy
    .

    Image source: 123RF "BlueRock and AskBio's clinical drug candidates have great potential in the treatment of Parkinson's disease
    .

    " said Dr.
    Wolfram Carius, Head of Cell and Gene Therapy at Bayer.
    "This may be the first time it is possible to halt and reverse this regression.
    Sexual diseases and truly help patients meet their highly unmet medical needs
    .

    "Reference: [1] Bayer to advance two first-of-its-kind cell and gene therapies for Parkinson's disease.
    Retrieved June 8, 2021, from https://media.
    bayer.
    de/baynews/baynews.
    nsf/id /Bayer-to-advance-two-first-of-its-kind-cell-and-gene-therapies-for-Parkinsons-disease Note: This article aims to introduce the progress of medical and health research, not a treatment plan recommendation
    .

    If you need to get treatment For program guidance, please go to a regular hospital for treatment
    .
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