Joshua Levy's Update on Research to Find a Cure for Type-1 Diabetes

Here are the three research directions, that I think are most likely to lead to a cure, soonest. They have all made strong progress in the last few months, and over the last year.

Research to Watch

GAD65: Diamyd

Just got approval for a 300 patient, phase-3 trial in the US, and a seperate 300 patient, phase-3 trial in the EU. This is right on the heels of a successful phase-2 trial. The phase-3 trial is supposed to last 15 months, so if successful, this could lead to a generally available cure in 2-4 years. So far this cure has been tested for honeymoon type-1s only.

Anti-CD3: TolerRx and MacroGenics

Both of these companies are developing a treatment targeted at the same part of the immune system. Both are in phase-2 trials. MacroGenics is running several phase-2 trials at the same time, covering 100s of people all together. MacroGenetics has linked up with Eli Lilly to market whatever they develop. So far this cure has been tested for honeymoon type-1s only.

Ecapsulated Porcine: LCT

These guys are in the middle of a tiny phase-1 trial, but the results from the first 6 months seem to be very good, with people who were given small doses using much less insulin. But the study size is tiny, 2 in Russia, 2 in New Zealand. Official publication of some of the data is scheduled for 31-March-2008. They hope to do a phase-2 study in the US starting in 2009. If it works, this cure should work just as well on people who have had type-1 for years, as those in their honeymoon phase.

Some Common Themes

Honeymoon vs. Long Term

The Diamyd, TolerRx and MacroGenetics treatments have only been tested on Honeymoon type-1 diabetics. So we do not know if they will work on long term diabetics. The idea behind all three is that they stop the immune attack on the insulin cells, and there are enough surviving cells to reverse the diabetes. However, it is my personal opinion that non-honeymooners may also benefit from these cures, in one of these ways:

• Some recent research suggests that the pancrease is always making new cells, and the immune system is always killing them. If so, then even long term diabetics will slowly be cured with these treatments.
• There are several research areas trying to make the pancrease grow new cells. Currently, these are not considered promising, because the immune system would just kill the new cells. But if any of these cures work for honeymooners, then they could be paired with a cell growth treatment for a cure for long term type-1 diabetics. Such growth factors include human growth hormone, INGAP, etc.

Remember that once a drug is approved for any one purpose, that drug can be proscribed by a doctor for any other purpose. This is called "off label" prescription. If if any of these cures are approved even just for honeymooners, then some doctor, somewhere is going to prescribe it to non-honeymooners, and we will all quickly learn what happens.

New Players

I know little to nothing about any of research listed below. If you have heard of them, or know anything about them, please send me some email:

Bayhill Theraputics

Not really new, but new to me. And headquartered in Palo Alo. Does anyone know anything about their phase-1 trial, which has been underway for over a year?

Alba Therapeutics

IKDRC