Dear Families and Friends,
Cure JM was honored to be the only nonprofit invited to serve as a keynote speaker at Rare Disease Day at the National Institutes of Health this past February. This distinction reflects what is possible when a small, mission-driven rare disease organization commits to long term partnership and scientific collaboration. Over the past 23 years, thanks to your support, Cure JM has demonstrated how families, clinicians, federal researchers, and industry partners can work together to achieve results once thought out of reach.
I was honored to be joined on stage at the NIH by Dr. David Chang of Cabaletta Bio, one of our industry partners advancing the CAR-T clinical trial in JM. Together, we recognized the NIH and the National Center for Advancing Translational Sciences for their early leadership in advancing CAR-T therapy.
CAR-T therapy is one of many new treatments now offering real hope to families living with juvenile myositis and other rare autoimmune diseases. What is unique about CAR-T therapy is that it shows promise to be a “one-and-done” treatment, where a single infusion may “reset” the child’s immune system. The clinical trial is currently being conducted at Cure JM Clinical Care Network partner sites across the U.S., and our intention is to have 19 sites up and running by the end of the year.
Breakthroughs like CAR-T do not happen overnight, and they do not happen in isolation. They happen because families, clinicians, researchers, and funders stay committed over decades, investing early and often when others cannot.
That commitment is being tested right now.
We stand at the edge of a transformational period in the history of juvenile myositis research. Earlier this year, Cure JM’s Scientific Review Board reviewed the largest number of JM research proposals we have ever received. These studies represent meaningful opportunities to improve care today, including research to personalize treatment for children and young adults and innovations that allow families to track strength and progress from home.
At the very moment this pipeline of promising research is growing, federal funding cuts and belt-tightening across the country are threatening to slow or stop progress.
We recently met with a clinician whose federally funded JM clinical trial was eliminated due to budget cuts. Without Cure JM stepping in, that trial cannot move forward. Stories like this are becoming increasingly common as pediatric rheumatology programs across the country face tightening resources. When funding disappears, there is often no one else to fill the gap.
Except Cure JM.
Your support allows us to respond when research is at risk, clinics need support, and families are counting on progress. In the year ahead, Cure JM will invest at our highest level yet in pediatric rheumatology departments nationwide to help clinics maintain care, train clinicians, advance clinical trials, and continue research despite a shrinking pool of federal dollars.
And CAR-T is only the beginning.
With your support today for Cure JM’s Clinical Trial Fund, we will be able to invest directly in making the next generation of treatments a reality. Promising new treatments include JAK inhibitors, which show potential to reduce reliance on steroids and methotrexate and move JM care away from blunt force therapies toward more targeted, precision treatment with fewer long term side effects.
We are also advancing other highly promising therapies. One is a next generation therapy that experts call a “super-charged IVIG.” Another is a new treatment that has shown strong results in adults and was the turning-point for one child who did not respond to any other treatments. In fact, some clinicians have called this new treatment “life-saving.” Ensuring these therapies become accessible to children and adolescents is urgent.
Rare Disease Day was a powerful reminder that we are at a true turning point. We have the science, the talent, and the momentum to change the future for children with juvenile myositis. At the same time, external funding pressures mean that how quickly we move forward depends on our community stepping up.
I have heard many parents and grandparents say over the years, “If not us, who?” That question has never felt more urgent. When Cure JM cannot fill the gap, research is delayed, trials are cancelled, and progress stalls. We cannot allow that to happen.
I hope you will consider making a gift today to help ensure this momentum continues. Your support fuels research, strengthens care, and keeps hope alive for children and families who need it most.
With deepest gratitude and unwavering resolve,
Jim Minow
Executive Director, Cure JM Foundation
Feb-March 2026