02 October 2013

Rodent Studies – Do They Really Mean Anything?

I admit I am tired of reading about studies involving rodents that are heralded as a breakthrough. Most often that is the last we hear about them and then we see no human trials or studies using the rodent data. To me this means there is no viability for these to move forward to humans and thus no meaning for the rodent studies recently heavily promoted.

Now we have two more rodent studies, one about an approved cancer drug that could treat diabetes and another that could potentially solve the loss of beta cell features that contribute to the onset of diabetes. Now will we see any human studies confirming these findings? It will be interesting, but doubtful.

The most interesting is the adapting of the cancer drug and this is probably the most promising as the researchers do state, "Anecdotally, there have been reports that diabetic patients who have been prescribed VEGF inhibitors to treat their cancer are better able to control their diabetes." They also state, "Much work remains to translate these mouse studies to human patients, but it will be interesting to explore VEGF inhibitors or drugs that can stabilize HIF-2alpha, such as prolyl hydroxylase inhibitors, for diabetes treatment, possibly in combination with pre-existing therapies to minimize toxicities."

"Targeting the Phd3/HIF-2 pathway represents a new therapeutic approach for the treatment of diabetes with little toxicity," said one of the researchers. "These studies indicate that Phd specific inhibitors, especially Phd3, should be more widely developed for clinical development."

The second article about the protein Nkx6.1, which is a beta-cell enriched transcription factor, is essential for maintaining the functional state of beta cells. The study shows that loss of Nkx6.1 in mice caused rapid onset of diabetes. Scientists have revealed the critical role of this protein in the control of insulin biosynthesis, insulin secretion and beta-cell proliferation.

The researchers have found that the loss of Nkx6.1 activity had an immediate on the expression of genes that give beta cells their ability to synthesize and release in insulin biosynthesis.

Whether this rodent study is worth the time reading is debatable.

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