Tuesday, August 7, 2012


Progress in the treatment of multiple myeloma is slow (let us not speak of "cures" here). Over the past couple of decades, the options developed, aside from the nuclear one (bone marrow or stem cell transplant), are the three FDA-approved agents thalidomide, lenalidomide (Revlimid), and bortezomib (Velcade). These, and various cocktails combining them with each other, with steroids, etc., comprise the universe of weapons commonly used against this cancer.

A few weeks ago, a fourth contestant entered the lists: carfilzomib, called by the brand name Kyprolis by its manufacturer, Onyx Pharmaceuticals, received FDA approval for the treatment of multiple myeloma. As usual, formal approval is for an absurdly narrow scenario, namely for patients who have developed resistance to two of the other therapies and who showed progression within 60 days of completing the last therapy. The clinical trial on which the approval was based involved just 266 such patients. On such slender scientific threads do our fates turn. Of course, the restrictions imposed by the approval are a polite fiction; having received approval for some usage, carfilzomib can now in reality be used by oncologists for any use that seems to them likely to provide some benefit, as indicated by further research. Fortunately.