Juvenile arthritis describes autoimmune and inflammatory conditions that lead to symptoms of arthritis in children younger than 16. The symptoms include joint pain, stiffness, and swelling, and are caused by the immune system wrongly attacking healthy cells and tissues in the body, leading to damage and inflammation. The condition’s exact trigger is unknown and it currently has no cure, but research is ongoing to better understand its underlying cause and to find potential new treatments.
Research into the cause of juvenile arthritis
Without an understanding of its root cause, designing a treatment to prevent or cure juvenile arthritis is exceedingly difficult. Scientists worldwide are working to uncover that root cause, and many theories have been presented.
Research suggests that genetics may play a role in the development of juvenile arthritis. For example, a recent study published in the journal Arthritis & Rheumatology identified regions in the genome that are mutated in some common juvenile arthritis subtypes, called oligoarticular and polyarticular types. These regions are related to the immune system and could be involved in the disease.
Medication in clinical trials
Clinical trials are ongoing to test whether new or repurposed drugs may be effective in managing the symptoms and slowing the progression of juvenile arthritis.
Pfizer is currently recruiting participants for a randomized, double-blind, and placebo-controlled Phase 3 trial (NCT02592434) that is assessing the safety, tolerability, and efficacy of Xeljanz (tofacitinib citrate) in juvenile arthritis patients ages 2 to 17. Participants will also be able to enroll in an open-label extension study (NCT01500551), running through 2020, to test the long-term safety of the drug. Xeljanz is already approved to treat rheumatoid arthritis in adults, and results from a Phase 1 trial, (NCT01513902), in juvenile arthritis patients suggest that it is also safe and well-tolerated in children.
Other drugs in clinical trials for juvenile arthritis include:
- Arcalyst (rilonacept), developed by Regeneron Pharmaceuticals, has completed Phase 1 (NCT01803321) and Phase 2 (NCT00534495) clinical trials in patients with juvenile arthritis. Results, published in Arthritis & Rheumatism, suggest that the treatment is well-tolerated and could have a clinical benefit in juvenile arthritis.
- Kevzara (sarilumab), is an approved treatment for rheumatoid arthritis developed by Regeneron. It is currently under investigation in a Phase 2 trial for juvenile arthritis (NCT02776735).
Stem cell therapy
Stem cell therapy involves introducing new healthy cells into a patient to combat disease. There are many different types of stem cells, and they can produce different results.
Autologous hematopoietic stem cell transplantation (HSCT or ASCT) is being investigated as a therapy for some autoimmune diseases, including juvenile arthritis. HSCT resets the immune system’s “memory” by replacing regulatory immune cells with fresh ones. This aims to stop the immune system from targeting the healthy tissues and to prevent further damage. But as the underlying cause of the immune system targeting of healthy tissues is not known, HSCT cannot prevent this from happening again in the future. HSCT has been attempted in patients with juvenile arthritis, following immunosuppressive treatment. Studies suggest that it can result in sustained remission or a marked improvement in the disease symptoms, but the procedure can be risky.
Another type of stem cell therapy, using umbilical cord-derived mesenchymal stem cells (UC-MSCs), has been tested in a small trial involving 10 juvenile arthritis patients. Each received two doses of UC-MSCs, three months apart, and were monitored for up to two years following treatment. Results, published in the journal Stem Cells International, suggest that the treatment is safe and can reduce symptoms of the disease.
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