Accelerating Therapies For Refractory SJIA
(with support from Rare as One grant from CZI)
Children with refractory SJIA, especially lung complications have a high mortality rate. Their disease is not controlled by any of the typical SJIA medications. Many children are on a strong cocktail of different immunosuppressive medications, but lung disease keeps progressing or MAS cannot be controlled. Several patients are looking into Bone Marrow Transplantation as a last option.
To find safe and effective therapies for this group of patients, whether new drugs or repurposed.
For the past few years, SJIA Foundation has collaborated with researchers at Cincinnati Children’s and other institutions to find safe & effective treatments for SJIA. With the Rare As One grant, we will renew that work with increasing vigor, work with more institutions and researchers and find treatments for these patients.
- Study patients during flares & quiescent disease (from multiple centers)
- Study organ biopsy tissue when available
- Apply modern bioinformatics method to finding patterns
- Build up a robust drug pipeline of new drugs
- Identify “repurposed” drugs with established safety profile
- Rashmi Sinha, SJIA Foundation
- Jon Boutelle, SJIA Foundation
- Alexei Grom, Cincinnati Children’s Hospital
- Grant Schulert, Cincinnati Children’s Hospital
Our Approach with Patients
We are a patient-first organization and are ultimately driven by helping patients. Since 2016, we have helped at least 50 SJIA patients get second opinion appointments.
- Build up a patient contact registry (launched June 2019)
- Annual Family Conference & Sample Collection (launched May 2017)
- Helping patients get second opinions at expert centers
- Longitudinal Sample Collection (repeat samples on same patients)
- Get samples from biopsies when possible
Collaborating with Other Organizations
It takes a village to solve a disease. Here are some organizations and efforts we hope to work with
- CARRA (Childhood Arthritis and Rheumatology Research Alliance), specifically their project on Lung Disease in SJIA
- Resyst: a collaborative study identifying molecular pathways underlying refractory Systemic Juvenile Idiopathic Arthritis. Focused on Europe (main contact: Bas Vastert & Fabrizio DeBenedetti)
- SJIA Parents Network on Facebook to organize and help patients with SJIA (main contact: Leah Bush)
- Autoinflammatory Alliance and their Facebook group to help patients with diseases similar to SJIA (IL18opathies and interferonopathies). Main contact: Karen Durrant