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Together, we can reach our goal and fund crucial work in 2021 -- promising drug trials, our national and international research programs, drug discovery and genetics programs, and medical education for our families and physicians.
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TOP THREE POTENTIAL NEW DRUGS FOR JM
I assessed the outcomes from Cure JM’s “New Drugs in Juvenile Myositis” symposium and the consensus reaction was, “Wow!”
The symposium informed pediatric rheumatologists and other doctors in JM care of new and better treatments that we hope will be available to them soon.
What we know so far is promising. Here are the biggest takeaways from the symposium:
Vamorolone: I call vamorolone the “anti-steroid” steroid. It is a drug that has all of the benefits of prednisone and virtually none of the side effects. Clinical trial results in Duchenne muscular dystrophy are forthcoming. Those results will be reviewed and hopefully approved by the FDA in 2021. Will it work in JM? Dr. Eric Hoffman, the CEO of the ReveraGen, the biomedical company that created vamorolone, presented an optimistic case that it will, and we will need to have our own clinical trial in JM to prove its effectiveness.
Baricitinib: Dr. Hanna Kim presented the findings of her four-patient clinical trial of baricitinib, a drug currently approved by the FDA for other inflammatory diseases, notably rheumatoid arthritis. Did it work in JM? In a word: YES. Of the four JM patients enrolled in the trial, all saw improvement in skin conditions, and two who suffered muscle weakness also saw significant improvements in muscle strength. Dr. Kim is planning a second trial with 20 JM patients in an effort to replicate the results and create a larger validated data set that doctors can use in the future to prescribe baricitinib for use in JM.
Abatacept: Dr. Rodolfo Curiel, the principal investigator of the drug’s trial and Director of the Cure JM Center of Excellence at George Washington University reported anecdotal information that suggests efficacy in JM.
It is clear to me that 2021 holds the potential to be a most important and pivotal year in Cure JM’s history. The clinical trials we need to conduct will focus on safety and efficacy in JM, and if successful, would represent great leaps forward. We’ll continue to keep you informed about these potential new treatments.
With appreciation,
James Minow
Cure JM Executive Director
ENCOURAGING RESULTS IN JM DRUG RESEARCH
New research at the NIH points to promising new drugs to treat Juvenile Myositis.
The Cure JM-funded research was conducted by Travis B. Kinder, PhD, Patricia K. Dranchak, PhD, and James Inglese, PhD, at the NIH's National Center for Advancing Translational Sciences (NCATS). The study tested thousands of drugs to identify which of those drugs could effectively treat JM.
The team genetically modified samples of human muscle cells to create conditions of chronic inflammation. Using a sophisticated robot, they ran thousands of drugs against the muscle cells in order to identify which drugs eased inflammation. Of the thousands tested, twelve drugs showed the most promise. Here's a short list of the most promising drugs:
Echinomycin
Ruxolitinib
Baricitinib
Tofacitinib
Panobinostat
Vorinostat
Doxorubicin
Givinostat
The drugs already approved by the FDA still need to go through clinical trials in JM patients to prove that they can safely and effectively treat JM.
For drugs not currently approved by the FDA, the next step is to test those drugs in animals.
The NCATS team will continue to screen more drugs and also explore potential environmental triggers for JM.
Watch this video to hear directly from Dr. Kinder in the NCATS lab where they conducted the research:
