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.
The way that the drug works is by modifying steroids so that the transrepression effects are removed. Transrepression means the turning on and off of genes, and it is responsible for the majority of side effects in steroids.
Vamorolone is on a fast track. The drug has been tested in mice and has been tested for safety in healthy adults. Since it causes fewer side effects, it can be given in much higher doses than steroids are given, which could make it even more effective than steroids. A 48-person test with Duchenes children started in July of last year. And an 100-person test (with placebo and prednisone groups as well as Vamorolone groups) is starting in July of 2017. Given this schedule, it’s realistic to hope that the drug is approved by the FDA as early as 2019.
We are hopeful that this Vamorolone will be a good treatment for other diseases (many diseases are treated by steroids). And of course our main interest is in whether this drug will be useful in treating SJIA, especially the tough refractory cases that don’t respond to current biologic drugs. We don’t know of any researcher looking into this, but think its an interesting question given how many SJIA kids do respond well to steroids but cannot remain on it long term due to the side effects.
Also, Reveragen is not a profit-seeking corporation (they operate according to a venture philanthropy model), so if the drug does get approved, it will hopefully be reasonably priced. We’ll post more as we learn more about this fascinating new treatment, and whether it’s a useful treatment for SJIA.
Roberta Burwell says
I understand your interest is now in treating SJIA with Vamorolone. As a long-time user of prednisone and budesonide inhaler for asthma, and prednisone later in life for ulcerative colitis/crohn’s, I am wondering if this
new drug Vamorolone will be effective and available for ulcerative colitis/crohn’s. We all know the many adverse side effects of long-term prednisone use, and this new drug sounds wonderful.
please let us know when this drug will be in market. using prednislone for SLE and very depressed as i lost my hip to its side effects. Looking for a replacement with less side effects especially on the bone for postmenopausal women who suffer from osteoporosis.
Please kindly help us.
Maria Torresan says
I have a rare disease call Idiopathic Subglottic Stenosis. Where my body is attacking its self creating scar tissue that grows over my throat so I cant breath. Because it is rare not much is known about this condition. Speculation is that some type of Auto-immune. Approx every 4 months I have an opperation to open my throat again so I can breath. The only other treatment is steroids. I am hoping that this new medication will be the miracle I’m looking for.
I have had problems with my ears for the past 30 years due to inflammation.
I have been put on prednisone a number of times over the past 16 years or so. It does seem to help open my ears and sinuses but after taking the prednisone the inflammation comes right back.
Will this product help me?