Will This Sickle Cell Research Help Cure ADA SCID?

At some point in the past, we decided that it would be important to divide things up when we study them in school. For a long time, all science was classified as physics because it involved the study of the physical world. Philosophy, religion, and mythology were all studied together under the name of metaphysics (special thanks to Aristotle’s students and editor for not having a better name for the books that come after physics) because it is beyond or after the physical world. Despite the fact that these disciplines have been divided, there is much to be learned from crossing different fields.

An example of this can be found in the field of medical science. Researchers working on adenosine deaminase deficiency with severe combined immunodeficiency (ADA SCID) found a new idea for treatment by looking at something that sickle cell anemia researchers are doing.

It seems strange that a condition dealing with the immune system would look to the world of blood disorders for inspiration. ADA SCID is an inherited disorder that severely limits the effectiveness of a person’s immune system. Sometimes this condition is referred to as “bubble baby disease” because sufferers must be kept isolated from potentially dangerous bacteria and viruses. This vulnerability often causes repeated infections, some of which can be life threatening. Sickle cell disease is also a genetic disorder that causes some red blood cells to assume a crescent shape rather than the healthy disc shape.

A group of researchers at the University of California Los Angeles (UCLA) decided to conduct sickle cell disease research.

One of the latest treatment options for people with sickle cell disease is the use of stem cell transplants to rewire how red blood cells are created. In this case, the procedure involves taking stem cells out, manipulating them, and injecting them back into the person him- or herself. This idea of self-donation reduces the likelihood of rejection of the transplanted material as well as the need to find an exact match for donation.

The researchers at UCLA wanted to do something similar to boost the immune systems of children with ADA SCID. They found a way to help introduce the enzyme that these children are missing to stimulate their immune systems.

Thus far, the phases of the clinical trials have been quite successful. One study had ten children (nine babies and one teenager) undergo the procedure. All nine babies responded well. In fact, all nine were cured of the disease. The teenager, unfortunately, still needs enzyme replacement treatments, which he was using before the clinical trial. None of the children involved in the study experienced negative complications due to the procedure.

This is an exciting time for the field of immune deficient conditions. Looking outside the autoimmune community of related diseases may yield more revolutionary ideas for treatments.

Click here to read a post detailing the study on the UCLA website.