Philosophy & Approach
SJIA is a complex, systemic disease that can effect multiple organs if not under adequate control. While there are some biologic medications that are used for SJIA, there is no cure. The best that patients can hope for is for the disease to go into remission. The currently used biologic medications provide adequate control for only some of the patients. Other patients bounce from biologic to biologic, trying different combination of biologics and other immunosuppressive medications. A third group of patients are completely refractory, remaining under control only with systemic steroids.
The SJIA Foundation is focused on finding better treatments and a cure for all SJIA patients, with a special focus on patients who are refractory to current biologics.
SJIA being a rare disease, there are few medications developed specifically for it. Instead, many of the medications have come from other, more common inflammatory diseases , the current drugs have often come from other inflammatory diseases. As such a good place to look at is other inflammatory diseases for drugs to reposition or repurpose.
The diseases we currently track are
- Rheumatoid Arthritis (being a common disease, attracts a lot of drug development and clinical trials)
- HLH (is a good place to look for drugs for MAS)
- Other Auto-inflammatory diseases such as NLRC4 Mutation, CAPS (because the genetic basis is better understood than SJIA, targeted drug development is more possible)
- Still’s Disease (sometimes drugs are first tested in Still’s Disease since clinical trials with Pediatric patients are more complex)
The Next Gen Drugs in SJIA Conference (in October 2017) was the first effort to identify a pipeline of drugs that are in development for SJIA or similar diseases. At the conference, there were talks related to below drugs and cell therapies.
Currently the SJIA drugs and cell therapies we are tracking
- IL18-BP from AB2Bio: FDA just granted it breakthrough therapy status
- Emapalumab or NI-0501 from Novimmune
- Newer IL6 blockers
- Mesenchymal Stem Cells
- Hematopoietic Stem Cell Transplantation
- Jak Inhibitors: Tofacitinib & Baricitinib
- IL17 Blockers: Secukinumab, Brodalumab
SJIA Pipeline: (Coming Soon)
The SJIA Drug Pipeline is the primary way we decide what research we track what basic or translational research we want to focus on. The pipeline identifies the major drug programs in development, and tracks their progress from basic research through clinical trials.
How we support research
We support research projects that are focused on the goals listed above. Our support for research projects projects goes beyond grants. Any research project we partner with, we can provide patient input at any stage in the project, we can write letters of support for funding agencies, we can help get feedback from SJIA parents through surveys and other methods.
If you have a project that fits our goals, please write to us and we can discuss how we can help.
Projects we have supported in the past
JAK-STAT Signalling pathways in SJIA/MAS project at Cincinnati Childrens: Project focuses on identifying key signalling pathways in MAS. Principal Investigator: Alexi Grom.
Next Gen SJIA Treatment Conference (October 2016): Conference in Washington DC for researchers, doctors, and patients to identify novel treatments for SJIA.
SJIA-ILD project at Stanford: Funded a project focusing on Interstitial Lung Disease in Systemic JIA. Principal Investigator: Betsy Mellins.
SJIA-ILD working group (March 2016): Conference at Half Moon Bay for researchers and doctors who work with patients that have SJIA and Interstitial Lung Disease (ILD). Goal was to review the most severe lung disease cases and look for patterns.
SJIA-ILD Conference (March 2015): Conference at Stanford University for researchers and doctors who work with patients that have SJIA and Interstitial Lung Disease (ILD). Goal is to encourage cross-disciplinary work and share findings to enhance collaboration, and to understand the causes of and treatments for patients that have both of these diseases.
Consensus Treatment Plan in Systemic JIA: Funded the next phase of Carra’s Consensus Treatment Plan for Systemic JIA patients. Funds will be used to collect clinical data and blood from Systemic JIA patients in an effort to determine which treatments work best to control SJIA symptoms. Principal Investigator: Yukiko Kimura.