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Gene Therapy Clinical Trials for Sanfilippo A and B Coming in 2014
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Thread: Gene Therapy Clinical Trials for Sanfilippo A and B Coming in 2014

  1. #1
    Forum Helper - Moderator
    Join Date
    April 14, 2009
    Springfield, Illinois

    Gene Therapy Clinical Trials for Sanfilippo A and B Coming in 2014

    I have some questions about the Gene Therapy that has been posted recently by Team Sanfilippo.

    Here is a snippet of what has been posted on the Team Sanfilippo website:

    “A consortium of family foundations from around the world have come together to fund clinical trials in 2014 for Sanfilippo A and B using gene therapy. The trials will be conducted at Nationwide Children’s Hospital in Columbus, Ohio.

    The two lead products, ABX-A and ABX-B, uniquely deliver the therapeutic product to the CNS with the aim of preventing and/or reversing the effects of the genetic errors that cause the disease. ABX-A and ABX-B induce cells in the CNS and digestive tract to produce the missing enzymes and help repair and prevent damage caused to the cells. Safety studies conducted in large animal models have demonstrated that delivery of ABX-A and ABX-B are well-tolerated with minimal side effects. Importantly, efficacy studies in animals with Sanfilippo syndrome have demonstrated unprecedented therapeutic benefit months after treatment. A single dose of ABX-A or ABX-B significantly restored normal cell and organ function and increased the lifespan of animals with SF over 100% a year after treatment compared to untreated control animals. Similarly, animals treated with ABX-A and ABX-B demonstrated corrections of cognitive defects that remained months after drug administration. These results are consistent with studies from several laboratories suggesting ABX-A and ABX-B treatment could potentially benefit patients with Sanfilippo Syndrome Type A and B, respectively.

    ABX-A and ABX-B will be administered systemically for efficient drug delivery to the whole body and CNS – a significant advantage over current therapy options.”

    Jake is not in the Natural History Study, so I am not up to date on the latest and greatest. Some questions that come to mind are:

    1. Is this different than the Shire trial they are doing overseas? (I am guessing it is.)

    2. What is the vehicle used to administer these products into the CNS?

    3. Does anyone have any idea what will be involved with the trial, i.e. how many treatments?

    Mom to Hannah and Luke (9 - twins) unaffected and Jake (7) MPS IIIA

  2. #2
    I think this is different from history study but not sure.

  3. #3
    Senior Member
    Join Date
    June 11, 2006
    Upper Darby, PA.
    This sound so promising I am also curious about how they getting the treatment in the CNS.
    Aunt and Guardian:
    Karima (5-31-88) Age 24
    MPS VI Maroteaux-Lamy

  4. #4
    Forum Committee - Moderator
    Join Date
    February 26, 2004
    Baltimore, Maryland
    It is different that the one Shire did overseas, it's a one time IV injection at this point in a vein.
    God never gives me more than I can handle, though at times I wish the load was lighter :-)
    Love to you all!

    Patty and Jesse

    http://www.caringbridge.org/md/myjesse < click here for Jesse's website

  5. #5
    Forum Committee - Moderator
    Join Date
    October 4, 2004
    Upstate NY
    Gene therapy is different from the IT trials. In the IT trials, they are injecting the enzyme directly into the spinal fluid to allow it to reach the brain. With gene therapy, they splice the missing or mutated gene into a certain kind of virus (with the bad parts taken out). The virus travels through the body and actually puts that gene into the cells, allowing the body to produce the enzyme on its own. Depending on the virus that they use, it may have to be repeated periodically, but it wouldn't be as frequent as ERT or IT enzyme. In theory, gene therapy could be a cure rather than a treatment. This is my simplistic explanation- my husband could lecture on it for days! His early research in grad school was related to gene therapy (not for MPS).
    Mom to Avery, 14 yrs
    ^^Logan^^, MPS II (6/14/03-3/15/15)
    and Kara, 9 yrs

  6. #6
    Forum Committee - Moderator
    Join Date
    March 5, 2004
    Good explanation Robin!
    There is one other gene therapy trial that has already started in Paris involving 4 young Sanfilippo patients. There are no results yet, but this gene therapy of Dr. Fu's is far less invasive. that it will start punctually.
    Lots of love and good wishes
    Elizabeth, Peter, Daniel (MPSIIIA born Jan 91) and Samuel (born Nov 93)and Anna Nigina (born Nov 01)
    'I shall not die, but live and tell of the works of the Lord' Psalm 118 v. 1
    Video about Daniel and Sanfilippo,http://www.youtube.com/watch?v=RPG4w92RgbI
    Daniel's web site: http://www.caringbridge.org/visit/danielschuepbach

  7. #7
    I've been asking around (ie. sending an email to Team Sanfilippo, filling out a contact form for the children's hospital directly) to see how to get more information on how to be considered for the trial. Do any of you have info on how to become a candidate for it? Who do I get in touch with?

    Wife to: Ryan (my rock)
    Mommy to:
    Annika (Anni) MPS IIIA (Age 5, 5/28/08)-my laid-back, smiley, helpful and kind musical genius
    Emma (unaffected) (Age 2,5/14/11)-my go-getter, laughing, wake-up-happy little munchkin

    Psalm 139--When I don't know, I go to the One who does.

  8. #8
    Junior Member
    Join Date
    June 26, 2014
    Ottawa, Canada

    Natural Hist Study

    We have 2 children with Sanfilippo B, and I inquired about the natural hist study being carried out in Columbus Ohio. I received a call last night from Dr. Kevin Flanagan from Nationwide Children's Hospital. He explained that they used a virus as a vehicle.

  9. #9
    Senior Member
    Join Date
    April 16, 2009
    Amarillo TX
    I received a letter addressed to Ian's primary about this at the doctor. I was wondering if the one mentioned here is the one for SF B in Ohio.

    The letter stated there was a high need for SFB, and we did not enter.

    The virus vectored has always interested me. . . . .
    married to Derrick
    Mom to
    Ian MPS 3A
    Tesla Unaffected

    Give it to God.

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