Cure JM is dedicated to funding and collaborating on the most promising juvenile myositis research clinical trials to shape a future free of JM. We are dedicated to supporting better treatment options through the clinical trial process to improve the lives of generations of JM patients.
CAR-T Myositis Clinical Trial
We are supporting clinical trials for promising innovative new treatments like CAR-T, a potential “immune system reset” for juvenile myositis. Early data shows promise for this therapy in cancer and autoimmune diseases like JM.
Cure JM partner Cabaletta Bio has recently opened up an adult CAR-T myositis trial for myositis patients over 18 years old, with an expected adolescent trial in the coming month, so check back regularly for updates. Learn more about this clinical trial and Cabaletta Bio’s early promising results at https://www.curejm.org/car-ttherapypotential/
JAK Inhibitor Clinical Trial – Deucravacitinib
In 2017, we partnered on a project with the NIH to screen thousands of U.S.-registered drugs to uncover compounds and drugs that are the strongest candidates for best treating juvenile myositis with fewer side effects.
The outcome of this screening identified several JAK inhibitors, with a drug named deucravacitinib (Sotyktu), a promising candidate to be safe and effective in treating JM, with the hope of reducing reliance on steroids and chemo treatments. Deucravacitinib shows promise in JM with a low side effect profile.
Cure JM’s research partners at the NIH will open enrollment by early 2025. The trial will begin with patients aged 18 and older, and we expect adolescents to be added to it in 2025.
Vamorolone – A Potential Steroid Alternative
We are funding pre-trial work on vamorolone with key researchers at Cure JM’s Center of Excellence at Duke University to advance a clinical trial plan with the FDA. Vamorolone is a new steroid alternative that was recently approved by the FDA for treating Duchenne Muscular Dystrophy. Vamorolone shows promise in providing the powerful benefits of corticosteroids without the harsh side effects that are often associated with prednisone.
Cure JM is working closely with Duke University, the FDA, and the distributors of vamorolone on a clinical trial plan to test safety and efficacy of the drug in JM.
