I hate scleroderma. I think I may have mentioned this before. It seems that nothing seems to work in this condition, not even for the non-life-threatening but life-sucking symptoms like digital ulcers and calcinosis. In the fall, the ACR had two presentations on the use of imatinab (Gleevec). One small study suggested some small but encouraging benefit while the second study was extremely small because the investigators called the whole thing off. I decided to remain optimistic against all hope, but that all changed when I met one of the investigators from the second group at a recent conference. She was not at all wishy-washy, telling me that these patients did not tolerate the drug at all, even at sub-targeted doses. Forget it. Move along.
Well I have, and I've even found the next drug that probably won't work for scleroderma. Perfenidone, a novel, not-sure-how-it-works anti-fibrotic drug has just received a rather non-ringing endorsement from the FDA advisory committee for the treatment of idiopathic pulmonary fibrosis. In Vivo has an article on how one of the FDA referees voted no regarding safety and no regarding effectiveness but then voted yes for approval. The logic, which sounds familiar to any scleroderma patient or treating physician, was that even if it didn't impress him, there really isn't anything better out there. So I'm sure there are scleroderma trials underway trying to break the long streak of failures on record. We can only wish them luck. Lots of it.