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“There’s still a great need for additional treatment for this very dangerous condition,” Pui Lee, MD, PhD, emphasized.
Pui Lee, MD, PhD, explains the symptomology of Still’s Disease and systemic juvenile idiopathic arthritis (sJIA), what initially prompted his team to attempt to discover new pathways for treating patients with sJIA, and the study design of his recent trial. Lee is a pediatric rheumatologist at Boston Children’s Hospital and lead investigator of the study “mTORC1 connects the spectrum of pathology in experimental models of Still’s disease and macrophage activation syndrome.”
“It's a form of juvenile arthritis that we see in children, probably one of the rarer forms, but it is the form that is the most difficult to treat because in addition to the joint inflammation that other kids have, these kids have inflammation of the whole body," Lee noted. "They have lots of fever, they have skin rashes, they have inflammation of the heart, lungs, and in almost every single organ system can be involved.”
Adding to that, somewhere between 10%-20% of children are also affected by something called macrophage activation syndrome (MAS), in which the body is so inflamed that the organs start to fail, resulting in a 10%-20% mortality rate within this patient population.
“We've done a lot better over the last 10 years with the availability different biologics for treatment of systemic JIA, and it's much better now,” Lee highlighted. “Still, the limitation with some of the biologics that we have are that these are all parental administered medications. These are injections or infusions that we're committing these very young kids to for a long time and that's not particularly easy.
Lee emphasized that oral medications will have an advantage in this patient population, although he highlighted increased evidence that currently available medications may not be long-term answers for children who are refractory to treatment and bio-available biologics.
“There’s still a great need for additional treatment for this very dangerous condition,” Lee concluded. “We want to explore the physiology behind the disease further and see if we can have other treatment options.”
Huang Z, You X, Chen L, et al. mTORC1 links pathology in experimental models of Still's disease and macrophage activation syndrome. Nat Commun. 2022;13(1):6915. Published 2022 Nov 28. doi:10.1038/s41467-022-34480-6