Tuesday, November 25, 2014

Cure for "Bubble Baby" Disease

         Perhaps through the popular movie the Bubble Boy or the episode of Seinfeld that featured this disease you have heard about this disease. Although, those characters were fictional, the disease is very real. It is called the adenosine deaminase (ADA)-deficient sever combined immunodeficiency (SCID) or often called the "bubble baby" disease, this disease is very serious and if left untreated kills most newborns in the first year of life. There were very few options for children born with SCID ADA-deficient until now.
        Children born with (SCID) have inherited immunodeficiency, are diagnosed in the first six months, and are extremely vulnerable to infectious diseases. Children with SCID are also kept in controlled environments which is where the infamous "bubble" reputation came from.  A child with ADA-deficient SCID lack the proper enzyme ADA. This enzyme is critical in growth of healthy white blood cells, without these white blood cells the common cold could be fatal. Before this discovery was made there was only two options for children who were ADA-deficient SCID. The first was they had to get bi-weekly enzyme injections to try and bring their immune system to an optimum level. Which was an extremely expensive and life long process that might not even bring the immune system to the optimum level. The other option was to get a bone marrow transplant from a matched sibling, but to find the match is extremely difficult and the actual transplant is very painful. The marrow cells could also reject the child's normal body cells and turn against the child.
       UCLA researchers developed a new treatment using gene therapy to help cure these children. Dr. Donald Kohn's breakthrough was developed over three decades. It "safely restores immune systems in children with ADA-deficient SCID using the patient's own cells with no side effects." To date all 18 children with SCID that were in his clinical trials have been cured by receiving the stem cell gene therapy. Kohn and his team took the patient's blood stem cells from the bone marrow and genetically modified them to correct the defect. They then used a viral delivery system to put the corrected gene that now produces the missing enzyme back into the bone marrow of the patients. The team used "the powerful self-renewal potential of stem cells to repopulate the blood stream and the child develops their own new, fully-functioning immune system." The next step is to hopefully get FDA approval and get the treatment to all ADA-deficient SCID children.


       I find this whole discovery to be extremely fascinating. I just hope the FDA approves this gene therapy method and all of the children that need this disease get it before any other child dies.

Original article
http://www.medicalnewstoday.com/releases/285724.php 
Related Article
http://www.adagen.com/bone_marrow_transplant.html

2 comments:

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