VBP-15 for Duchenne Muscular Dystrophy

Prednisone has been used since the 1970s for delaying the otherwise obligatory progressive motor deterioration seen in  Duchenne Muscular Dystrophy (DMD.

A good deal of data has been acquired over the years.  In fact there are even ongoing studies looking at different dosing regimens.

The drug is typically started between ages 4-6 at a dose of 0.75 mg/Kg.  However, steroid cause may side effects to Duchenne boys, including weight gain and behavioral problems.

How does it work?  We’re not sure, but we think the medication stabilizes muscle membranes and inhibiting cytotoxic T-cells.

Side effects are mediated by binding sites in the cell nucleus which lead to initiation of metabolic pathways :
NM2

The new drug VBP-15 is molecularity very similar to Prednisone, but lacks the 11 beta hydroxy arm, which reduces the metabolic side effects, without affecting the beneficial membrane stabilizing effects:

nm3

VBP-15 has been shown to improve muscle strength and function in an animal model of DMD without the metabolic side effects of Prednisone:

nm4

Clinical trials in humans are expected to begin in 2013-14.

Watch this space for more information.

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9 thoughts on “VBP-15 for Duchenne Muscular Dystrophy

  1. Email from webblog:

    I am the patient of muscular dystrophy ( LGMD), 28yrs of female.I have done the treatment of Stem cell therapy 6 months ago,but no improvement.can I use the VBP15 oral drugs? Is it tb. or syp? plz sir tell me it’s usage ( dose). plz also tell me how it efffects on patients & patients history of those who use the VBP 15?

    plz sir reply me as soon as possible, I’m anxiously waiting for your reply.
    Thanks

      • first of all I want to say thanks for replying me. please sir tell me the dose of VBP15? I want to start VBP15 till gene therapy get successfull results b/c it is on a trail process? would I start to take VBP 15 till gene therapy come with your permission? can I? plz plz sir tell me the dose? Where it is available ( In which country)? what it’s price?

        Thanks

      • Neelum, I am not aware of any VBP15 drug availability outside of drug company trials. Research is still at a pre-clinical level. The next step will be a clinical trial. Even if the drug were available outside a drug trial, I would not recommend you take it that way. We do not yet know (a) if the drug works and (b) if it is safe. The purpose of the clinical trial is to answer these questions, and protect participants as much as possible from potential adverse effects. I would suggest that you keep an eye out here, on the drug company website, and on clinicaltrials.gov for study opportunities.

  2. Pingback: Duchenne Muscular Dsytrophy | Neurology Update

  3. Comment received by email:

    I have a son with 14 years old who is fighting with DMD ,now i want

    realy know if i can be hope to get this drug ( VBP15) in near

    future for my son ,if yes, please inform me when.

    Thanks

  4. hi,
    dear sir,
    i want to know that from where we can get VBP15, i am in kuwait now. this is available in wich countey.

    regards
    zahid sajjad

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