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Dear Cure JM Families and Friends,
I want to share with you briefly the two greatest gifts I’ve been given this Holiday Season.
The first gift came from last month’s American College of Rheumatology conference, which I was fortunate to attend. The gift was the excitement we could all feel around what is now being recognized as a “revolution” in juvenile myositis healthcare and treatment.
The revolution is driven by ever-increasing evidence that drugs known as Janus kinase inhibitors—or JAKs—truly are effective in treating juvenile myositis and have the potential to supplement or even replace current first-line treatments such as corticosteroids and methotrexate.
The revolution is also driven by CAR-T therapies. In CAR-T therapy, a JM patient’s “good” t-cells in the blood are modified to eliminate the “bad” b-cells, which are a root cause of juvenile myositis. Many researchers and doctors believe that CAR-T could be curative in JM, and results that we’ve seen in two teenagers with chronic JM toward eliminating the disease have been very encouraging.
This is one reason why Cure JM is partnering with Cabaletta Bio to conduct CAR-T clinical trials with actual JM patients starting in early 2025 in locations across the country. This is also the reason why Cure JM is partnering with NIH to conduct a clinical trial for Bristol Meyers Squibb's JAK inhibitor drug deucravacitinib, which will also enroll patients in early 2025.
This is our job, as stated in our mission: “To discover better treatments and cure for juvenile myositis.” My heartfelt gratitude goes out to all of our families and donors who have made this progress possible through your generous donations. Without the funding you have provided through the Holiday Challenge, these advances would not have happened.
The second gift arrived last week in a report to our families from two Cure JM-funded researchers—Dr. Jessica Turnier from the University of Michigan and Dr. Jess Neely from the University of California, San Francisco. Dr. Turnier and Dr. Neely are also attending JM physicians at their respective children’s hospitals.
You may recognize Jessica and Jess from their leadership of the Cure JM/Chan-Zuckerberg Initiative, a $2 million project to understand what changes take place in JM children at the basic cell level in muscle, blood, and skin. This kind of research is known as translational research, meaning that if we better understand the “what and why” of what happens in the body as a result of JM, we can better target future therapies.
Their update, entitled “Unlocking the Secrets Within: Inside the Cell - Discovering the Key to New Treatments and Cures,” provided astounding insight into the critical role of blood vessels and capillaries as JM courses through the body.
The “aha” moment for me was the realization through this research that healthy blood vessels and capillaries literally feed every organ of the body. In juvenile myositis, blood vessels can be compromised, and capillaries especially become gnarled and dysfunctional, impeding the successful transfer of oxygen and other nutrients to organs throughout the body.
In JM, symptoms most often show muscle weakness, skin rash, exhaustion, and diminished stamina. But importantly, other organs, notably the heart, lungs, and even the central nervous system can be starved and strangled by a diminished blood vessel highway.
Fixing that highway is at the heart of Jessica and Jess’s cell-level research. Indeed, their ambitious research goals are to connect what happens “inside the cell” with specific JM treatment outcomes by investigating a broad cross-section of skin and muscle biopsies, plasma, and blood. Understanding what cellular mechanisms drive JM-related inflammation will hopefully lead, as Dr. Neely concludes, that their research “helps to better track disease in the clinic and identify treatments that could target this part of the disease.”
I hope you see these research gifts as I do—important breakthroughs in research leading to better drugs and a therapy that could possibly be a cure for JM. Clinical trials in 2025 will start to prove the effectiveness and safety of newer and better options for JM patients. Your alliance with Cure JM has made past and future breakthroughs possible while improving the quality of care and quality of life for JM patients everywhere.
Best wishes for a wonderful Holiday Season.
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