Posted by on Jun 18, 2013 in Medicine | 0 comments

By nature I’m a skeptic, but when it comes to science and medicine, I’m a wild-eyed optimist. So you can imagine my mixed feelings of excitement and concern when I read the recent item out of Boston Children’s Hospital stating that their researchers had found the root cause of Type 1 diabetes. While this is cause for celebration, I think we’d better take a closer look at the implications for the future.

Just in case you missed the news, you can check out the organization’s own blog post on the subject. I’ll sum it up for you: Dr. Paolo Fiorina, a researcher in the Nephrology Division at Boston Children’s Hospital, and his team have studied hundreds of pathways in animals with diabetes, and finally isolated one – known as ATP/P2X7R – which triggers T-cell attacks on the pancreas. These attacks make the pancreas unable to produce insulin, which gets the whole process of Type 1 diabetes underway.

The whole blog post is worth reading, as it discusses other attempts at treating this tragic and painful disease – not just insulin injections, mind you, but transplants of pancreatic islet cells, used in an attempt to get the body producing insulin again so that injections are no longer necessary. Getting at the actual pathway for the attacks that cause the pancreas to become disabled could allow us to figure out a way to stop the disease process in its tracks. No children, then, might ever need to know the heartbreak that comes with sticking themselves with a needle multiple times a day, not eating cake and ice cream at birthday parties (even their own!) or other times, and more. I’m not even getting into all the nasty complications of the disease!

I’m smiling and all but jumping up and down with joy at this news…but I’m tempering that with a small dose of reality. Let me explain.

The first point is that this pathway was discovered in animals. If the team follows the usual approach, as I understand it, they’ll need to figure out how to block the pathway in animals before they can try it out in humans. In the blog post itself, Fiorino notes that it will be a few years before they test any therapies they come up with, based on this discovery, in children. I don’t have an issue with this; caution is certainly warranted. Because – and I’d like to emphasize that I’m speaking from a LACK of medical knowledge here – we need to make sure that this pathway works the same way in humans. We should also consider whether or not this is the ONLY pathway that triggers T-cell attacks on the pancreas. If it isn’t, we may eventually be treating and curing only some forms of Type 1 diabetes, not all.

Indirectly, this brings me to my second point: there is more than one form of diabetes. The much more prevalent form is Type 2, not Type 1 – and the way that disease takes hold in the body is entirely different. The pancreas of a patient with Type 2 diabetes may produce plenty of insulin – much more than a non-diabetic, in fact – but their body’s cells have become less receptive to the substance. Therefore, while this breakthrough (and it IS a breakthrough) will likely help the five percent  of diabetes sufferers with Type 1, and perhaps even lead to a cure for them, it won’t help the rest of us.

Or maybe it will. There’s a form of diabetes called latent autoimmune diabetes in adults (LADA) that displays signs of both Type 1 and Type 2 diabetes. A key point is that its patients possess antibodies against the insulin-producing cells of the pancreas, just like ordinary Type 1 diabetics – and as many as 10 percent of those diagnosed with Type 2 diabetes might have this variant of the disease. If some researchers are correct about the cause of LADA, then a treatment that cures Type 1 diabetes by disrupting the pathway that causes T-cells to attack the pancreas might slow the progression of the disease in many LADA patients – putting off or (dare one hope?) even avoiding eventual insulin injections altogether.

My guess is that we’re going to find out that this wonderful answer from Boston Children’s Hospital will go a long way towards a cure, but also raise even more questions about this complex disease. That’s how and why science is done. What do you think will be the result of this discovery?