Phase one clinical trials escalate drug dosage to determine toxicity and potential efficacy.

“so these are typically very small studies usually less than 100 people and they're typically done in cohorts so you might say well the first you know 12 people are going to be at 0.1 milligrams per kilogram and assuming we see no adverse effects there we'll go up to 0.15 milligrams per kilogram for the next 12 people and if we have no issues there we'll escalate it to 0.25”

“now you'll notice bob i said nothing in there about does the drug work right so right these are going to be patients with cancer if this is a drug that's being sought as a treatment for colon cancer these are going to be patients that all have colon cancer they're often going to be patients who have metastatic colon cancer right so these are going to be patients who have progressed through all other standard treatments and who are basically saying look sign me up for this clinical trial i realize that this first phase is not going to be necessarily giving me a high enough dose that i could experience a benefit and that you're really only looking to make sure that this drug doesn't hurt me but nevertheless i want to participate in this trial”

“the first phase of that which is called phase one is geared specifically to dose escalate this drug from a very very low level so to determine what the toxicity is across a range of doses that will hopefully have efficacy so these are typically very small studies uh usually less than 100 people and they're typically done in cohorts”

“now you'll notice Bob I said nothing in there about does the drug work right”

“phase one is only for safety phase one is is it safe to give humans this molecule is it safe to give it once is it safe to give it multiple times and there's an art to knowing where to start the dose because it's an escalating dose trial”