Monday, July 21, 2008

LCT Reports Good Results from Phase-I Trials

LCT has just posted results from their Phase-I human trials. They are transplanting encapsulated pig islet cells. If successful, their cure will work on all type-1 diabetics. Even those who have been diabetic for years.

You can read the report here: my summary of it is below.

The Good News
No safety problems.
80% of people's A1c went down, average went from 8.5 to 6.8 (the one who went up was just .3)
All people's average insulin usage went down, on average by about 24% at end of study.
One person used no injected insulin at all for a time.

The Bad News
Study is very small, only six people.
Study is very short, a maximum of 12 months, most people were followed much less than that.
Study was done in Russia.

When you look at these results (and the more detailed results in the PDF paper linked above) remember that this is a Phase-I trial. That means it is designed to test safety, not effectiveness, and that they usually give a very small dose during Phase-I trials. It is usual to only get partial effectiveness in these trials, because you are testing for safety. Phase-II is where you should see the higher effectiveness, because you can use higher doses.

So, with that in mind, I think it is clear that LCT's treatment can lower the use a of insulin quite a bit, and also lower A1c numbers, and that should lead to fewer complications. And this is all in a low dose Phase-I trial!

The big issue for me is: How long will it last? And the news here is not so good. The two patients who were followed for 12 and 11 months ended up with about half the insulin production that they started with. So after about 11 months they were only generating about 1/2 the insulin as when they were first implanted. The two patients who were followed for 5 and 4 months, one stayed the same, and the other dropped about 30%. I think it is going to be critical to find out what happens to the 6 patients over the next four or five years. (Plus any research LCT can do to see why less insulin is generated after 11 months.)

There is also an issue in making sure the patient generates all of their own insulin, so they don't need to inject any, but I'm assuming that just requires more islet cells, and I'm kind of assuming that is not a big deal. They can put in 4x as many islets, and get 4x as much insulin. I hope.

The obvious question is: What is the next step? Will LCT start a higher dose, Phase-II trail? Will they follow their Phase-I patients for longer? Will the do more patients as part of their Phase-I trial? Will they do all of these things? I don't know. But when I do know, you will know.