An innovative new stem cell treatment may be used to cure a severe immune system disease commonly known as “bubble boy” syndrome.

The condition’s full name is severe combined immunodeficiency disease (SCID). It is a rare genetic disorder which impairs immune system function and makes people extremely vulnerable to infectious diseases. The term “bubble boy” was coined by the media when they were reporting on David Vetter, a young boy born with the disease in 1971.  Unfortunately David died at the age of 12 from Burkitt’s lymphoma.

Young mother Alysia Vaccaro recently had one of her daughters diagnosed with the condition. Alysia first realised something was wrong when one of her twin daughters, Evangelina, had slower than normal growth. Doctors diagnosed her with SCID. The condition meant her daughter’s immune system couldn’t fight off any diseases including the common cold. 

The condition is extremely rare, only affecting between 1 in 50,000 to 1 in 100,000 newborns in the United States. It occurs when the genetic mutations disturb the development of healthy T cells and B cells, which are both lymphocytes that play an important role in immune system response. The condition is the most severe form of immunodeficiency. Scientists have identified nine different genes that lead to mutations that cause the disease.

Alysia said the family immediately began wearing facial masks and washing their hands frequently to prevent the spread of disease to their daughter.

Doctors suggested that a new bone marrow transplant technique might be useful for treating her condition and enrolled Evangelina in a new clinical trial. The doctors took some bone marrow from Evangelina, purified it, and then cloned a gene to replace what she was missing. The stem cells with the repaired gene were then transplanted back into Evangelina where they could create healthy blood cells and a fully functional immune system.

Dr. Donald Kohn from the Broad Stem Cell Research Center at UCLA explains the procedure, saying: “Those stem cells are given back to the patient where they can go back to the bone marrow and make the blood cells for the rest of the patient’s life.”

23 patients from the clinical trial have achieved restored immune system function thanks to the treatment. Evangelina is now 3-years old and completely healthy!

Source: Doctor Uses Stem Cells to Cure ‘Bubble Baby’ Disease

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