Tuesday, September 2, 2008

Discussion of recent press reports of type 1 cures

Over the last two weeks or so, there have been several press reports about various research into cures for type 1 diabetes. Since I often get asked about these, I thought I would post my thoughts on them. Note that none of these are human (or clinical) trails. They are all animal research.

There are two things to remember about all animal based research:
  1. Very few cures that work in animals, work in people. It is not possible to know the exact number, but my gut feel is that for most treatments, only 1% or 2% of the cures that work in animals end up being used in people. And the number for type-1 diabetes might be lower than that!
  2. It takes 10 years or more for a cure that works in animals to be fully tested in people and get all the approvals needed to be mass marketed for people.

With that in mind, here is the recent research:

This sounds like the perfect cure. Inject a drug (Leptin) into a type-1 diabetic, even a non-honeymoon diabetic, and then they are cured. And it worked in mice! Since this is being funded by JDRF, they should have no trouble getting funding for phase-I trials on people, if this really is as good as it sounds. For my part, this sounds very interesting, but also very different than previous cures. So I'm hopeful, but also nervious. I do think that this will make for a great phase-I trial, and we will know a lot more about it when that first human trial is done.


Direct Reprogramming
This is a very powerful idea, that basically allows you to reprogram a cell that is already in the pancrease to create insulin. You convert already existing cells into beta-cells. It has all the advantages of stem cells, but you don't need stem cells, instead you reprogram the cells that are already there. Obviously, to be a cure, you would have to stop the immune systems attack on these new beta-cells; either that or generate so many new ones, that you kept ahead of the destruction. So this might need to be combined with one of the "honeymoon" cures currently in phase-III trials, to be a complete cure. I discussed this more in a previous post. As above, since this is being funded by JDRF, they should have no trouble getting funding for phase-I trials, if this really is as good as it sounds, and it turns out we need beta cells in order to cure type-1.


Making Beta Cells
This guy is experimenting with making new beta cells outside of a body; sort of like a beta cell factory. Ideally, these can later be implanted back in a person. Has many of the same issues as "Direct Reprogramming" described above. You still need to stop the immune attack, and so on.


The National Acadamy of Sciences just published some of Faustman's pre-clinical studies in mice. Faustman is already doing a human trial based on this same research, and results are expected next year. The current human trials do not even measure BG or A1C levels, so no one's diabetes is going to be cured, or even improved, by the clinical trial currently underway. They are hoping to learn enough to effect BG and/or A1C in the next set of clinical trials.

Gastric Bypass for Type-1
Basically gastric bypass is a type of surgery used for type-2 diabetics. It shortens the digestive track so that you can eat the same amount of stuff, but still loose wait. For people who's type-2 diabetes is fueled by being overweight, this can be a viable treatment option. However, one particular doctor has this theory that the digestive system is involved in type-1 diabetes, and that this surgery will cure type-1 diabetics! It seems pretty nuts to me. He sees evidence that the type-2s he works on get good BG control "too fast" and "too well" for it to be caused just be losing weight, and so he thinks there must be a direct effect (which would work on type-1s) as well.

Because this is a surgery (and not a drug, implant, or device) he doesn't need to get any FDA approvals. He can try this surgery on anyone he wants. So he is currently looking for non-overweight type-1 diabetes to operate on.


Inflamation Based Cures
Last year there was some surprizing research pointing to inflamation as being a possible cause of type-1 diabetes (not a symptom of it, as previously thought). This suggested the possiblity that stopping inflamation would actually stop type-1 diabetes. However, I know of no human trials currently underway which are based on this theory. The closest would be DiaKine's research

But remember for all the research described above and any research which is not being done on people: it has only a 1% or 2% chance of working in people, and will take 10 years (or more!) to become generally available. And that is why I don't track these "cured type-1 in animals" press releases very closely. I've got 10 years to see if they get anywhere, and most of them never do. Even the really well thought out ones that sounds like a really good idea have a 98%+ failure rate.

Most of the research discussed above is not described on my web page (http://joshualevy.pbwiki.com/DiabetesCureReadyForHumanTrials) because that page only covers clinical trials: trials being done on people.