ps418

A new paper describes a successful 'cure' of diabetes in a mouse model. The cured mice were treated with the genes Neurod and betacellulin, which were delivered using adenovirus vector. The mice remained normoglycemic throughout the 4 months of the experiement. Apparently the gene therapy induces the formation of insulin-producing islet beta cells in the liver (they are normally found in the pancreas). Its certainly interesting, but I wonder if gene-therapy trials for diabetes medicines would be authorized, given that effective medicines already exist for the treatment of diabetes?

Patrick

Kojima et al, 2003. NeuroD-betacellulin gene therapy induces islet neogenesis in the liver and reverses diabetes in mice. Nature Medicine Published online: 21 April 2003, doi:10.1038/nm867.
abstract

Press Release

Godot

Why not? We're not talking about a new treatment modality here, we're talking about a potential "cure". If the proposed trials meet ethics requirements (which is likely to happen after a few more animal trials are concluded), there is little barring human trials.
Of course, there is the possibility that diabetic gene-therapy in humans will be about as effective as the whole leptin thing. We just won't know if a treatment effective in a rat population will be equally viable in a human one until we subject it to clinical trials.

doghouse

I'm sure it would if it promises to be safe and effective. There are still a lot of problems with current treatment. First is the dailyordeal of injections, monitoring blood glucose, etc. Second, many diabetics eventually suffer serious complications, such as kidney failure, blindness, or peripheral nerve disease.

Kinross

Why wouldn't it. The current treatment for diabetes only allows you to manage the disease which is cumbersome and error prone. This gives the possibility of a cure allowing the body to regain control of blood sugar levels.

capsaicin67

In addition to the prior comments regarding reasons why a cure versus the current modes of management would be important and justified, some cases of diabetes also result in amputation, and some individuals are extremely brittle diabetics. Current management practices certainly are significant and important, but are far from without problems.

Another point might be offered that some individuals are simply lousy at managing and monitoring their diabetes, and comply poorly with their treatment regimen. This impacts not only themselves, but those around them, and the healthcare system itself, adversely. It seems like there are many reasons why a cure would be a fantastic and helpful option.

punta

I posted this news on another board and by two replies people were talking about the Jesse Gelsinger case.

not to diminish that uneccesary tragedy but it seems many people are more okay with the suffering and deaths of those who might be cured than taking a well informed (in the Gelsinger case it wasn't) and voluntary risk?

it seems easier to just wash our hands when the suffering is not staring us in the face, and cling to our self-absolving notions of "tampering with nature" and "playing God."

ps418

Does anyone know where to find information on the guidelines for gene therapy, that is, the requirements for initiating a human trial? I'm all for gene-therapy cures of diabetes, of course, but my understanding was that there are almost insurmountable regulatory hoops that need to be jumped through in order to initiate a human trial. For instance, would one fatal reaction in monkies rule out a human trial for that therapy?

Patrick

ps418

Yet another very interesting finding with significance for diabetes treatment is described in the April 29 issue of PNAS reports that Glucagon-like peptide (GLP) 1 induces glucose-dependent insulin production in mouse intestinal epithelial cells, by upregulating the transcription factor neurogenin 3 (ngn3 -- involved in the differentiation of endocrine system cells). Transplanation of the treated epithelial cells to diabetic mice reverses the diabetes.

Suzuki et al, Glucagon-like peptide 1 (1-37) converts intestinal epithelial cells into insulin-producing cells. PNAS 2003 100: 5034-5039.

Patrick

God Fearing Atheist

This is the closest thing I could find, and its not much..

From the FDA.