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First Clinical Trial of In Utero Stem Cell Transplant for Alpha Thalassemia

First Clinical Trial of In Utero Stem Cell Transplant for Alpha Thalassemia
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    We have an opportunity to cure a disease
    before a person's born.
    In our Fetal Treatment Center,
    we see many fetuses with birth defects all the time.
    Some of those birth defects can actually be treated
    with a stem cell transplant.
    If you do the stem cell transplant after birth,
    you have to give the baby a lot of different toxic medicines
    to make room in the bone marrow.
    And, that can cause diseases on its own.
    This is the world's first clinical trial
    of a blood stem cell transplantation in the fetus.
    If this treatment is successful,
    it could save the parents and the child
    from a lifetime of illness.
    (cello notes playing)
    So in this first clinical trial,
    we're focusing on a disease called Alpha Thalassemia Major.
    And we focused on that disease because it is fatal.
    Alpha Thalassemia Major is a disease in which
    the red blood cells don't form correctly.
    And so, the fetus gets very sick.
    When we take blood stem cells from the mother
    and we put them into the fetus,
    the hope is that they will go to the bone marrow
    and give rise to good red blood cells
    that can then carry oxygen and treat the anemia
    that the fetus is experiencing.
    (electronic notes popping)
    First, we have to harvest the stem cells from the mother.
    And then those cells are taken to the bone marrow
    stem cell processing lab where the blood stem cells
    are isolated and purified.
    And then, they're injected back into the umbilical vein.
    People used to think that the baby was enclosed
    in this impermeable space.
    But the truth is that the cells go back and forth
    between the mother and the fetus.
    And that trafficking it turns out,
    is actually really important in allowing
    the fetus' immune system to tolerate the mother's cells.
    (upbeat happy music)
    This is the first case in which we're using
    the mother's cells based on the biology that we discovered
    and using a high dose of cells placed directly
    into the fetus' blood stream.
    And we think that doing all three of those things
    will allow us to have more success
    compared to when it was tried before.
    If we find that this is safe and effective
    for this one disease,
    we hope that we can apply it
    to a host of other blood diseases,
    the most common of which would be Sickle Cell Disease.
    One of the things about being a doctor
    is that you're constantly seeing patients
    where you know that with the developments in science,
    you could help them better.
    You could help them more effectively.
    But those therapies aren't currently in the clinics.
    So I love being able to, in some weeks of the month,
    go into my research lab and test out therapies
    that we can finally bring to patients.
    (electronic beeps)
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