Lily Malinowsky was an energetic 1 year old, when last September she started having unexplained fevers and rashes, and was ultimately diagnosed with Systemic Juvenile Idiopathic Arthritis (or SJIA). She was started on Canakinumab (Ilaris) soon after diagnosis.
There’s an interesting article in Scientific American about a new trial for Rheumatoid Arthritis. The treatment involves a small pacemaker-like device that delivers tiny electrical shocks to the vagus nerve, a nerve that runs down the neck from the brainstem to several major organs, including the heart and gut.
Most SJIA kids whose disease is not controlled by biologic drugs take glucorticoid steroids of some kind (prednisone is the most common). Steroids work well in controlling inflammation, but come at a tremendous cost: stunted growth, fragile bones, cataracts, and weakened muscle are some of the side-effects. But we put up with it because the alternative of uncontrolled SJIA is worse.
Eric Hoffman, a researcher in another childhood disease (Duchenne Muscular Dystrophy), gave the keynote at our next-gen SJIA treatments conference in October. He is leading an effort at Reveragen to bring a steroid replacement called Vamorolone (generic name VBP15) to market. The goal of the drug is to replicate the good effects of steroids (primarily their anti-inflamatory effect) without the side effects.read more
TrialReach is an interesting new service for matching clinical trials with patientsto develop their service for matching patients with available clinical trials. This is a really cool idea. One of the things holding back research into rare diseases like SJIA is getting enough participants into the clinical trials so that the research can happen.read more