Very interesting. Am I understanding this correctly: it's an insulin replacement? Those treated would have effectively no insulin in their blood stream?
If so, wouldn't those treated by this gene therapy still suffer a persistent hormone imbalance? Hormones are complicated. Insulin (and the lack thereof) is tied in up-down regulation of lots of other hormones. Glucagon, for one, would always be higher in your body meaning you'd be prone to break down your body's fat stores. While that might actually be a positive side effect, I wouldn't be surprised if there were many negative side effects too.
Still, the side effects from this hypothetical hormone imbalance is very likely a huge improvement over constantly having advanced glycation end-products slowly destroying your body (which is what slowly happens to us type 1 diabetics).
It's a bit poorly-worded...but looks like it says the therapy does 2 things: 1) It expresses the insulin-producing genes (insulin is the protein hormone that binds with glucose in the blood to convert it to ATP, or energy), and 2) it expresses the Glucokinase genes (Glucokinase is a hormone that regulate glucose uptake from the blood).
(I know you're type 1 - me, too - but wanted to explain it further for those who don't know how it works.)
Agreed on the hormone imbalance side of things. The Glucagon issue seems very real. However, if it's in essence stimulating insulin production as well as improving the glucose uptake, then that in turn should balance the blood sugar effects of Glucagon. As for the other effects, though...that definitely remains to be seen.
It looks like the therapy introduces the genes for production of both insulin and glucokinase, so yes, there is still insulin, but not injected insulin.
Without a degree in Biochemistry, this appears to be a proof of concept. A demonstration that it's possible and the challenge is adapting it to humans, instead if starting from scratch. Seems to be pretty huge news for people with diabetes.
Exactly. There are thousands of these proofs of concept in mice. How many times have we heard of various cancers being cured in mice?
Keep in mind, too, a dog's metabolism is very different from ours. Yes, this is good news, but we're talking about an animal that would be killed by eating too much chocolate. I'll get really excited about this once we've cured some diabetic chimps.
Comparing cancer to diabetes is a reach. The balancing of hormones in diabetes is difficult but nothing compared to the complexities of each individual patient's cancers.
Since here we're dealing with a non-targeted genetherapy with a relatively well understood metabolic pathway the extension to humans will be simpler. The actual vector of getting the genes in will be challenging to convert but once we have that, we can use it for many other gene therapies.
There was a recent article in Science News [1] about the challenge of applying mouse studies to humans, followed by the NY Times slightly more exaggerated coverage [2]. Studies on non-humans for human issues can be misleading.
Agreed. They certainly can be misleading for those who expect them to translate over to humans without a hitch.
However, if one goes into trials on mice, dogs, and other animals with the understanding that you're comparing apples to oranges, there can be a lot gained.
This approach, for instance, is something completely different than I've ever seen WRT Type 1 diabetes. It's a 180-degree approach from islet cell transplants, semi-permeable protective membranes, and immuno-suppressive drugs. As a proof of concept, I think they can probably learn a lot from it - even if they can't simply copy it to humans exactly.
If so, wouldn't those treated by this gene therapy still suffer a persistent hormone imbalance? Hormones are complicated. Insulin (and the lack thereof) is tied in up-down regulation of lots of other hormones. Glucagon, for one, would always be higher in your body meaning you'd be prone to break down your body's fat stores. While that might actually be a positive side effect, I wouldn't be surprised if there were many negative side effects too.
Still, the side effects from this hypothetical hormone imbalance is very likely a huge improvement over constantly having advanced glycation end-products slowly destroying your body (which is what slowly happens to us type 1 diabetics).