Interview with Dr. Lou Vaickus, Tolerx, Question 1

DJ:  Why do you think a cure for Type 1 diabetes has yet to be commercialized?

LV:  Cure is a tall order in any chronic disease process. For Type 1, that involves both knowing when someone first has evidence of Type 1 (we do not have a good surrogate marker) and then having the right therapies to combat it before it destroys all of the insulin-producing beta cells in the pancreas.

Most folks have an ongoing autoimmune attack a relatively long time before diagnosis and so, when they first come to medical attention, damage has already been done, with only a small proportion of beta cells still intact and/or with reversible damage.

Regarding the right therapies, we have just begun to understand the autoimmune process — it is very complicated! — and it is very likely that it is heterogenous in how it affects one person vs another.  

However, we are exploring whether one experimental drug, otelixizumab, an antibody that binds to a surface protein on T lymphocytes called CD3, can halt the immune attack in patients who were just diagnosed with Type 1 diabetes. This exploration is a step in the direction of a cure and commercialization, and it could be the first in a class of products designed to stop the process that causes Type 1 rather than simply replace what’s lost.  

So, if someone presents with only 50% functioning beta cells but we can stop the autoimmune process from destroying more, do we call that a cure or a remission? Either way, it is the best we can do when someone first presents with Type 1 at present and other studies have shown that preserving even a small amount of endogenous insulin production will likely be of great benefit in preventing downstream complications.