Animal studies, even with 3 different species, only hint at what a human body will do to a drug. Pharma has found this out the hard way. After consistent animal studies they have had humans metabolize a test drug in a comparitively novel way, sometimes rendering the drug ineffective, sometimes making a drug poisonous, sometimes metabolizing a drug into a whole new (unexpected) drug. So - animal studies are helpful but by no means predictive. The biggest threat was toxicity, even at low dosages. Second cohort still at very low dose - it may be some time before we see positive effect. Continued lack of toxicity is a serious positive. So far so good. I have a LOT of confidence in this company, in this drug. I am trying hard not to evaluate with emotion but I feel so positive about it that my judgement may be a bit clouded.

When this drug leaves phase one and starts a phII/III trial, if the company allows (as they are in charge of each batch of drug manufactured for trial), individual physicians are allowed by FDA to file compassionate or expanded use application for patient with no viable treatment options. I had posted FDA link explaining procedure a little while back. It is cumbersome and due to minimal drug supply at this stage of development not always doable.