With the momentum coming out of our 20th anniversary last year, we are doubling down on our critical work to improve the lives of our patients. Twenty years ago, the outcomes for a child diagnosed with juvenile myositis were bleak. The disease was like a cancer, spreading and rendering children immobile by attacking muscles and, for many, the heart and lungs. Thankfully, those days are largely behind us due to the work of Cure JM and the many pioneering researchers we supported in those earlier years. Indeed, today, we stand on the shoulders of those early visionaries who developed the life-saving treatments we have today.
While lifesaving, current treatments are imperfect and have complications and side effects of their own. High-dose corticosteroids and chemotherapy, the heart of today’s treatment standards, are effective in treating JM because they work by suppressing the immune system. They also leave children “immunocompromised” with a weakened ability to fight off infections, which puts our children at risk. The current treatments also cause a host of side effects and long-term health issues, of which I’m sure you are aware.
I recently spoke with a parent who shared with me that her daughter Olivia, six years old, has been receiving weekly chemotherapy injections for four years. The chemotherapy makes Olivia so sick that she often spends most of the weekend in bed due to nausea. In addition to the anxiety and nausea, doctors now believe that four years of this harsh treatment may be causing liver damage for Olivia. They may soon have to choose between controlling the juvenile myositis with these harsh treatments or protecting her liver. These are the kinds of difficult decisions our families make every day – leaving the JM unchecked is life-threatening, but the treatments can, in many ways, be worse than the JM itself.
I believe we can – and must – do better for our children.
This is why the core of our mission is to find better treatments for children with juvenile myositis. This is our focus. We must identify promising drug candidates in juvenile myositis and move those candidates into clinical trials to prove safety and effectiveness in juvenile myositis as quickly as possible.
To identify promising drug candidates, you may recall that Cure JM funded a drug discovery program at the NIH under the leadership of doctors Jim Inglese and Travis Kinder. In this program, researchers used cutting-edge, robotic screening to test more than 4,500 drugs and compounds to see which are effective in fighting JM.
The good news is that through this screening, we identified a dozen or so drugs or compounds that could effectively treat JM. The next step is to start clinical trials on these drugs so we can make them available for our children.
This drug is proven to block the over-expression of interferon, which leads to muscle inflammation in JM. The team’s clinical trial plan for children and adolescents with JM is in place and has been approved by the FDA.
We are ready to go—almost. I say “almost” because the pharmaceutical company that was to supply the drug as a clinical trial partner has now elected not to participate. I’m not totally surprised by this turn of events. Juvenile myositis is a rare disease, and in the dollars and cents world of big pharma, a market like JM is all too often seen as too small to bother with.
But we’re not giving up, even though this big pharma decision leaves the research team with a $150,000 budget gap that someone must fill.
Cure JM’s Research Committee recommends that we fill this gap and provide this funding— with an initial commitment of $150,000 — to allow the trial to move forward without pharma’s participation.
So, I am asking for your support today.
The trial plan is already approved by the FDA, and this new treatment could be very effective for our children. Without Cure JM to fill the funding gap, this trial cannot move forward.
Your donation will help us reach our goal of $150,000 to ensure this promising baricitinib clinical trial can start on time.
Checks can be made out to Cure JM and mailed to Cure JM, P.O. Box 45768, Baltimore, MD 21297 with “Clinical Trial” in the memo.
It takes an army of parents, grandparents, friends, and family to make Cure JM’s work possible. We’re grateful for any generosity you are able give to help us drive our mission to give children the better treatments they need to have the best shot at a brighter future ahead.
