I was talking to my mom this morning about the cure for the boy in the bubble. Why can’t they just fix the faulty genes in Celiac and let’s move on. This brings up an interesting question – is gene therapy truly on the horizon?
We’ve all heard of the boy in the bubble. The boy in the bubble has a disease called X-linked severe combined immunodeficiency (SCID-X1). It causes babies to be born with little to no immune protection, making them prone to developing life-threatening infections. Shortly after birth, these children are put into sterile environments because their immune system cannot fight off even the slightest infection.
Up until recently, the only cure for this was a bone marrow transplant from a sibling or close relative. But the new therapy allowing these boys to live regular lives involves replacing the defective gene with a normal gene. Then that information is put into a modified HIV virus and injected into the patient. Many of these patients left the hospital after 3 or 4 months with completely normal immune systems. A small percentage also developed leukemia from this treatment, but it seems doctors knew this was a risk for this kind of treatment.
Now, would this work for Celiac disease? Simply replacing the defective genetic code with genetic code that works properly. I’m assuming it could be done – note I’m not a scientist, I just write a blog on the internet.
Should it be done or even explored as a method to solve Celiac? Celiac is not immediately life threatening like not having an immune system. While Celiac is a HUGE pain in the @ss and if left untreated is life threatening, but is it worth the risk of leukemia for a cure. This is a tough question and for me, I think leukemia is probably worse than Celiac.
I’m not sure I would be on board with altering my genetic code to make Celiac go away, but maybe over time as more and more diseases are cured this way it will become commonplace.
I’ve participated in a clinical trial to modify the way my immune system recognizes gluten, so I’m all about trying to fix things and make it better. I think the other drugs on the horizon are a better way to go for now. I’ll look forward to a genetic cure in 15-25 years.
PS – I’ve contacted the CeliacShield people regarding participation in their study. As soon as I talk with them, I’ll let you all know what I find out! I do know that you must the equivalent of 1/2 a slice of bread for 6 weeks in order to participate in this study and have a starting and ending endoscopy. You also have to go to their office 5 times during the study. I’m not sure if the beginning and ending endoscopy is included.