Saturday, June 22, 2013

The Root Cause of Type-1

Some researchers in Boston made a big splash recently by claiming to find the "root cause" of type-1 diabetes.  I've gotten at least two emails asking for my thoughts on the research, so here they are.
One thing that I try to do, is to read the original paper, or at least the abstract, and then describe what the researchers did, what they found, and why it is important.  I've found that the original research often contains valuable information that doesn't make it into press releases, news articles, blog postings, etc.  So I try hard to find that original work.

Unfortunately, I can't do that for this paper.  I've read the abstract a couple of times, and I can't make heads or tails of it.  The abstract assumes a level of knowledge way beyond me.  One thing that did make me nervous was this: it looks like this research was done on people and mice who had transplants to treat type-1 diabetes, and they studied how those people's bodies attacked the transplanted cells.  This makes me nervous because the immune system's attack on a transplanted organ is proper.  It's a foreign invader, after all.  This is different than the immune system's attack on it's own pancreas, which is an error.  I'm not sure learning about the causes of one, is going to teach us what causes the other.  On the other hand, I might be totally misunderstanding what they were doing.  The abstract is opaque to me.

How important is this?

I don't know.  I don't think anyone knows, as yet.  It often takes a few years to confirm that a breakthrough really is the big breakthrough that everyone hoped it was at the start.  Even after we know that this is a big breakthrough, we don't know if it will lead to a cure or not.  The discovery of the smallpox virus (for example) did not lead to the discovery of a cure for smallpox  (and the first smallpox vaccine predated knowledge of the smallpox virus).

In the future, if this leads to a cure (or preventative) for type-1 diabetes, then we will be able to look back and say, "this really was that important".  But there is no reasonable way for us to look into the future and know that this discovery is that important right now.

(I know that's kind of depressing.  Some people react to that by not donating money to research, because they never know which research is going to lead to a cure.  I view it the exact opposite, that it is important to give money to research, so that as many different options can be funded as possible, specifically because we don't know which will lead to a cure.)

What does this mean to me?

I care about a cure for type-1 diabetes, so what does this discovery mean to me?  Basically, a lot more research.  In order to get from a discovery of root cause to a cure, at least three things need to happen.  (a) they need to be sure the discovery is correct and use that discovery to figure out a cure. (b) they need to test that cure in petri dishes, tissue samples, and animals. (c) they need to test it in people.  Now (a) often takes a few years, although sometimes less, and (b) can take anywhere from years to decades, and (c) takes at least 10 years.  So making a few reasonable guesses, I would expect any cure that comes from this discovery to arrive in 15+ years (for an optimistic guess) to 25+ years (for a more reasonable guess).  If this discovery pans out as being important.  And that is a big "if".

One last word.

Curtis Lomax said "This one smells like a lot of hype."  I agree, and I couldn't put it better myself.  In fact, I'm linking to the Lynyrd Skynyrd song "That Smell" as the theme song for this posting:

Links to the news coverage:
I think this is the paper's abstract:
Joshua Levy
All the views expressed here are those of Joshua Levy, and nothing here is official JDRF or JDCA news, views, policies or opinions. My daughter has type-1 diabetes and participates in clinical trials, which I sometimes blog on. My blog contains a more complete non-conflict of interest statement. Thanks to everyone who helps with the blog. 
Clinical Trials Blog:
Cured in Mice Blog:


U can succeed ! said...

I agree with one thing. What does the break through really mean? I would say one has to look at the source this is the probably a real break through ( I mean we also understand how the aging process begins, but scientist can not reverse the aging processing). Therefore understanding how diabetes starts and how to stop it as you said Josh is something completely different. I personally believe it is really important break through, but again understanding why something happens and being able to treat cure or prevent it from happening is a whole different thing.

Aaron said...

Hi Josh, the blog and press surrounding this finding drew a lot of attention among my friends also and after looking into it here's what I told them:

The P2X pathway that they study is intriguing but I think they're hyping there findings to apply to more than the system in which they carried out he expts. They studied alloimmunity of transplanted islet(beta) cells in mice and found compelling evidence that P2X is involved (with some related human findings), but in the blog they claim to have found "the root cause of diabetes". That's yet to be proven IMO.

Thanks for looking into this and giving your insight into causality research.

Aaron said...

I really enjoy your blogs and appreciate the time and energy you must put into them!

I wanted to email you to point you to the reports about this finding but couldn't find your address here. Can you share your email address?