Interview with Dr. Lou Vaickus, Tolerx, Question 3

DJ:  What are the tradeoffs?

LV: All drugs have side effects and almost all of these side effects are dependent on how much of the drug you get, especially for drugs that modulate the immune system. So, understanding the optimal dose is crucial.

The side effects can be short term, ones that appear only later, or ones that only a small fraction of people will experience.

The short term ones are very similar to having the flu in which you get one or more of a list of symptoms like headache, chills, myalgia, arthralgia, fatigue, nausea, and maybe vomiting. Like flu’s signs and symptoms, these side effects are mediated by cytokines, are transient, go away with time, and can be treated with standard medications such as ibuprofen.  

Longer term side effects may involve loss of control of infectious agents like viruses or alteration in lymphocyte number or function.  

The rare (idiosyncratic) ones appear to occur only in folks who have a certain type of genetic background / subclinical disease, or who don’t react the way most people will to an anti-CD3 antibody.  

So, to “get to know your antibody” you need to do a lot of difficult, complex, rigorously controlled, and expensive work (preclinical and clinical studies) in as many people with Type 1 diabetes as possible before you present all you know about your product to agencies like the Food and Drug Administration (FDA) and ask if they would give you the go-ahead to commercialize it and make it available to all eligible patients with new onset Type 1 diabetes. In other words, it takes a lot of time, thought, courageous patient volunteers, and effort.