ipilimumab

I just spent an afternoon discussing lung immunology, allergy, and a variety of basic immunological mechanisms and concepts with a scientist who turned out to have been one of the people who helped invent ipilimumab (Yervoy), the first drug ever to be able to help metastatic melanoma patients.

What was pretty amazing about his work is that before ipilimumab, as he put it rather bluntly, The standard of care was death.  Treatment made absolutely no difference.  Now, at least there is something that can be done, and a pretty large number of drugs in the pipeline.

The drug was approved because it appeared to double the number of patients who survive one or two years.  If you think of your immune cells as having a 'gas' pedal (to activate cells in response to bits of - for example - cancer proteins), CTLA-4 is the 'brake' that prevents it from getting out of control.  Ipilimumab basically takes off the brakes to allow the immune system to attack cancer cells more, with the major side effect being possible irritable bowel disease / diarrhea (IBD) or loss of skin pigment (vitilgo). The bit that I didn't know about the combination chemo/ipilimumab studies that he explained was that the rationale behind doing so is that giving chemo simultaneously kills/bursts the immune cells, releasing bits of protein which then allows the immune system to recognize the cancer cells.  Then ipilimumab takes off the brakes to allow the immune cells to attack the cancer more fiercely.

Science is not a perfect thing, and neither is medicine.  Both advance in small stages through everyone's contributions, but it is a fortunate scientist indeed who can see the results of his or her labor within a lifetime, particularly when it directly helps people (as corny as that sounds).  Most of us do science primarily because we enjoy the process itself, as the long-term fruits are too far away for most of us to perceive or remain motivated by.  I am biased as a medical scientist-in-training interested in translational research, but it is a rare and beautiful thing indeed when research ultimately fulfills its purpose to alleviate suffering.