Skip to content

    Parkinson's Disease Health Center

    Font Size
    A
    A
    A

    Gene Therapy May Hold Promise for Advanced Parkinson's Disease

    Study says new dopamine-producing brain cells help control motor function

    WebMD News from HealthDay

    By Steven Reinberg

    HealthDay Reporter

    THURSDAY, Jan. 9, 2014 (HealthDay News) -- A new, preliminary treatment involving triple-gene therapy appears safe and effective in helping to control motor function in Parkinson's disease patients, according to new research.

    The therapy, called ProSavin, works by reprogramming brain cells to produce dopamine, the chemical essential for controlling movement, the researchers said. Lack of dopamine causes the tremors, limb stiffness and loss of balance that patients with the neurodegenerative disease suffer.

    "We demonstrated that we are able to safely administer genes into the brain of patients and make dopamine, the missing agent in Parkinson's patients," said researcher Kyriacos Mitrophanous, head of research at Oxford BioMedica in England, the company that developed the therapy and funded the study.

    ProSavin also helps to smooth out the peaks and valleys often produced by the drug levodopa, the current standard treatment, Mitrophanous said.

    The treatment uses a harmless virus to deliver three dopamine-making genes directly to the area of the brain that controls movement, he explained. These genes are able to convert non-dopamine-producing nerve cells into dopamine-producing cells.

    Although the study results are promising, the researchers suggest they should be "interpreted with caution" because the perceived benefits fall into the range of "placebo effect" seen with other clinical trials.

    Hoping to improve on their results, the researchers have since re-engineered the therapy. "We have a new version which makes more dopamine in patients, and this new version is undergoing safety studies before we initiate trails in patients," he said.

    Experts reacted positively but cautiously to the findings, which were published online Jan. 10 in The Lancet. While the treatment seems safe, its potential as a replacement for current therapy still must be proved, they noted.

    "The ProSavin study was a positive and important first step for a potential gene therapy for Parkinson's disease," said Dr. Michael Okun, national medical director at the National Parkinson Foundation. "The results of this preliminary study revealed a promising safety profile, and it will be interesting to observe longer-term benefits and how ProSavin will compare to other therapies such as deep brain stimulation."

    Today on WebMD

    Parkinsons disease illustration
    Causes, symptoms, and treatments.
    hands on walker
    How does the disease progress?
     
    man with serious expression
    8 common questions and answers.
    intelligence quotient illustration
    What are the advantages of DBS?
     
    Parkinsons Disease Medications
    Article
    Questions Doctor Parkinsons
    Article
     
    Eating Right
    Article
    Parkinsons Exercise
    Article
     
    daughter consoling depressed mother
    Article
    senior man's hands
    Article
     
    Parkinsons Daily
    Article
    Acupunture
    Article