The first disease ever cured by gene therapy was severe combined immune disorder (SCID) — the so-called \”bubble boy\” disease in which the immune system is so severely compromised that children have to live in near-sterile environments to avoid life threatening disease. But now, there is new evidence that the cure for SCID may increase the risk of leukemia among children receiving it.

In 2002, one of the toddlers who received SCID gene therapy as a baby came down with leukemia, and officials in France and the United States temporarily stopped the procedure. Now, a three year old who received the treatment as an infant has also come down with leukemia. As a result, the United States has suspended all 27 existing gene therapy studies have been suspended pending new risk assessments.

The potential for this sort of leukemia problem accompanies all gene therapies that use retroviruses, but this is the first time where this hypothetical risk has become actual. According to an NPR report, both of the children involved are responding well to treatment for their leukemia.

Source:

Gene therapy causes \”leukemia-like side effect\”. Nando Times.