Sunday, October 26, 2008

What is a real cure for T1D

The word Cure is getting thrown around a lot so it's reasonable to ask the question: What is a real cure for Type 1 Diabetes?

Diabetes is not the same for everyone, which leads to the conclusion that a cure for one person might not be applicable for someone else. Since Diabetes Type 1 (T1D) is a autoimmune decease stopping the autoimmunity should be the first course of action, either through immunosuppression (TolerX, MacroGenics), encapsulation (LCT) or immunotolerization (Diamyd).

Once the autoimmunity is stopped you can consider recreating the insulin producing beta-cells. This is still a major dark cloud and the reason why many (if not all) Phase III trials only include patients close to diagnosis and with remaining beta-cell mass. That way stopping the autoimmunity will in theory be a cure if the patient has sufficient beta-cell mass. So far no drug has proven to completely stop the autoimmunity, only to slow it down.

Lets have a closer look at the different approaches.

immunosuppression
Since it's all about suppressing the T-cells your body will be less resistant against infections and fever/flu-like symptoms are common. T1D often hit young children, and giving immunosuppressing drugs to children might not be taken lightly by both doctors and parents.

encapsulation
Transplanting islets and beta-cells is done and will work as a regular organ transplant. The risks of only transplanting the islets are smaller than a complete pancrea transplant. Even with immunosuppression drugs the new beta-cells will be a target for the immune system and soon get killed of. This is where encapsulation of the islets comes in to the picture. The islets will be protected without immunosuppression. This will work for "old" diabetics since there is no pre-requirement on any beta-cell function. Large downside is the amount of human islets available for transplants (LCT solves that by using pig islets), and the cost of treatment which might make this unavailable for poorer countries.

immunotolerization Can you teach the immune system not to kill your beta-cells? This is the way common vaccines work and by exposing the immune system to small quantities of the antigen you might turn off the autoimmunity. Diamyd works by exposing the body to one of the major auto-antigens in T1D, GAD65. Diamyd will not regenerate any beta-cells, so unless the body can regenerate beta-cells when the autoimmunity is cured, Diamyd will only work for diabetics with remaining beta-cell mass. Not all diabetics have auto-antibodies against GAD65, and if you don't you are excluded from the Diamyd treatment.


Hopefully within a few years (2011 for Diamyd), some therapies will start to become available. Some years later combination therapies will start to emerge and soon the cure(s) everyone is waiting for will eventually become available. Many have heard this before, a cure will soon be here, but we now live in the age of medicine with advances not thought possible.

Regarding newly diagnosed and pre-diabetics vs older diabetics, the first group will be the first to have a therapy available. Simply because they still have beta-cell mass left.

Two other large areas of interest are Stem Cell research and artificial pancreas. More about that later. I hope you have a nice day!

1 comments:

Claresa Levetan, MD said...

As an endocrinologist and full-time researcher in diabetes, a real cure for type 1 will mean a targeted immune tolerance agent, prior to using an islet neogenesis agent. None of the nine or so immune tolerance agents, thus far has proven that if you block the immune attack on the pancreas, that you can become insulin-free.

The process of islet neogenesis/new islet cell formation is extremely slow. Our group is introducing a new therapy with for islet neogenesis. Please visit CureDM.com The true cure will be here sooner than you think!
Claresa Levetan, MD
Fellow of the American College of Endocrinology
Chief Medical Officer
CureDM, Inc.
www.curedm.com