Reply #20: Don't get too excited YET. And before you tell someone with a [View All]

[Nothing Without Hope]

loved one dying of cancer about this, you need to find out much more. And even then, this drug is not going to be available for humans for some time, if it ever is. Phase I clinical trials are already underway, and the ones for efficacy won't be started until these results are in.

I have not gotten the actual journal article to see exactly what the researchers did, but since they are only now in a Phase I clinical trial in humans, it looks like it's only been tried in animal models, most likely immune-deficient mice. (That's standard - normal mice will reject human cells.) Phase I clinical trials are set up not for assessing efficacy but for testing safety for use in humans. The rather poorly written (since so little information is given) news article says that 56 patients with advanced and metastatic cancers -- people who basically have nothing to lose -- are being treated wth the drug in this Phase I trial. The researchers will be looking for signs of toxicity. If they are tolerable, then a Phase II or a combined Phase II/III trial will be started. THOSE would be for testing efficacy in human cancer patients. While it is possible that there may be some effect observed in the Phase I trial, that's not the goal and the patients are in a very advanced stage of the disease before treatment.

It may well be that only early-stage tumors or tumors of a certain type might respond, or perhaps there might need to be treatment with multiple drugs at once. It's just much to early to know what will happen and also much too early to plan on getting into a clinical trial quickly. The drug sounds promising, but no one knows what it will do in humans. Other drugs have been very promising in animal models as well. The animal model results sometimes don't transfer to humans, and apparently right now that's all there is. They are near the beginning of a long road, though the prospects look promising. It will be highly interesting to monitor how the trials go, but it's far too early to try to get the drug for someone you love.

So be careful about calling someone with a sick loved one with what sounds at first to be life-saving news.