Yes, though based on what we can loosely call "FKBP-science," RAPID is indeed a conceptually fairly different animal versus ARGENT.
The following has some interesting comments, some of which touch on a topic brought up earlier in this forum:
cnn.com
Dr. Richard Furlanetto, scientific director of the Juvenile Diabetes Foundation, said the experimental technique "is very clever science" but might fall short.
"To be truly useful, it would have to be coupled to a system that would release the hormone in direct response to the levels of glucose in the blood," said Furlanetto.
However, Furlanetto said the technique could be very useful in treating conditions that require periodic secretion, or pulsed release, of some needed protein, such as growth hormone.
Yes, Dr. F, of course it would be nice if you implant the stuff and pretty much cure type I diabetes. But, if the system were to work in people as shown in the mouse model one would have what amounts to an oral, pill-based, version of current therapy with injected insulin. And this would not be truly useful? Am I missing something, not being a diabetes expert by a long shot?
Mind you, I am not saying it would be easy even to get to that stage with insulin. Most of the data presented in the Science article used IV delivery of the small molecule, and while "similar results" were said to be obtained with an oral version, it is also indicated that the efficiency of oral absorption is about 20% in mice. A lot of work is needed to make this work smoothly in people, and Ariad could use some help in the small molecule aspect from some pharmaceutical.
OTOH, one should note that in this system one also controls, to some extent, the protein target (the so-called CAD, a mutant form of FKBP12), so one can tweak the protein-small molecule interaction from either side. This is a luxury one does not have in "normal" drug optimization where the biological target is fixed. And, of course, insulin is far from the only protein one can conceive of delivering with this system. Other proteins may not need the very delicate balance on timing and concentration one apparently needs for insulin, and thus may represent easier avenues for clinical development.
PB |
