Girl Cured Through Bone Marrow Transplant

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I think they're pretty confident it's an autoimmune disease. The stem cell treatments I've heard of involve destroying the patient's own immune system with chemotherapy, and then basically rebooting it with the stem cell/marrow transplant. Seems that if anything would be a cure, this would be.

That said, I do agree that it may be a little early to call it a cure, but it sells papers (and, truthfully, gets the word out about the treatment.)
 
"doctors discovered a unique mutated gene that was causing constant inflammation in Brygette’s gastrointestinal tract"

They found the cause for her.

If the treatment does well in other cases and their disease never returns, I'd probably call it a cure. I don't see how it would be any kind of a disservice. It'd be awesome.
 
Even though in her case it was a specific gene, I know they're doing trials with similar treatments for more general cases.
 
"doctors discovered a unique mutated gene that was causing constant inflammation in Brygette’s gastrointestinal tract"

They found the cause for her.

If the treatment does well in other cases and their disease never returns, I'd probably call it a cure. I don't see how it would be any kind of a disservice. It'd be awesome.


Yes, they say they found a mutated gene that caused it, and then say she's one of about 20 people with this mutation. As most of us know, having a gene for something doesn't mean you will get it because genes can be turned on and off. Also, there are lots more people with IBD and nobody knows the cause, so how can anyone prove the girls doesn't have what everyone else has?

To me, it seems like she is being helped with her autoimmune issues, but I'm skeptical that her bowel issues are Crohns disease or one of it's forms. I'm in no position to know anything, but I would be hesitant to make these kind of absolute claims. On the positive side of things, it is very encouraging to see such improvement with auto immune diseases! Looking forward to seeig what comes of this.
 
I think in this particular case, the inflammatory bowel disease (IBD) this 4 year old girl had WAS totally caused by that rare, mutated gene. I wonder how many cases of Crohns go by without doctors checking for this particular gene mutation. It may be that other folks (especially very young patients) are being diagnosed with Crohns when in fact it is this mutant gene in them. The fact that she is eating well, growing, playing like a normal child is good evidence that the bone marrow transplant that lead to her disease healing seems to have "cured" her. I know it is a 'rare' mutation, but given the sheer numbers of Crohns patients.... I'm wondering if this rare lightning has struck more than once.
 
Crohn's is currently defined by a basket of symptoms. There could be multiple causes, or more accurately there could be multiple diseases currently called Crohns.

Dan
 
There certainly are several diseases currently called Crohns. If not certain, near certain. Still, the success rate of immune modulators seems to demonstrate that autoimmune issues are relevant to a majority of sufferers.
 
The story I heard was that some Crohns patients who developed Leukemia and received bone marrow transplants went into remission (both crohns and leukemia). Which implies that immune system suppression or replacement is the way to go. remission usually lasts 10-15yrs. So I doubt that it's a true "cure".
 
I have read about some of this and the whole destroying the patient's own immune system scares the crap out me (no pun intended). I would guess it would be a last resort type treatment.
 
Crohn's is currently defined by a basket of symptoms. There could be multiple causes, or more accurately there could be multiple diseases currently called Crohns.

Dan

I agree that there may well be more than one disease bearing the catch-all label "Crohns". Isn't the defining diagnostic characteristic of Crohns the presence of non-caseating granulomas being present in the biopsy specimens histology?
 
I agree that there may well be more than one disease bearing the catch-all label "Crohns". Isn't the defining diagnostic characteristic of Crohns the presence of non-caseating granulomas being present in the biopsy specimens histology?



Yes and no. Yes it's a good indication, but no not all Crohnies have this. I never have.
 
I have the granulomas, but I also think I have Crohn's in its most common form. In another words I am nearly 100% my Crohn's is the type that is caused by the MAP bacteria, and also involves certain strains of E-Coli that are symbiotic with the MAP bacteria.

I think it is quite possible that what is currently diagnosed as Crohn's could be caused by other pathogens, but could still have the common denominator of a dysfunctional immune system. I really do not think "autoimmune" is an accurate description of the immune system problem.

Dan
 
Yeah, Crohns covers a multitude of diseases.... and whether by oversight or intention, the article referred to her problem as IBD, and not "Crohns" specifically. And we all know how hard it apparently is to get a correct diagnosis. Now, I've never undergone anything like what this little girl went thru... wiping out her immune system, the marrow transplant, etc.. A friend of mine went thru that, battling leukemia (lost the battle), and passed away. I don't know/can't recall if one had to take immuno suppressors after a marrow transplant. Thing is... if one has to continue taking immuno suppressors, that would alleviate symptoms anyway. If there is no continued immuno suppressors needed, and the mutant gene strain has been irradicated, unless it can somehow resurrect itself, I'd figure you'd call it cured.
 

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