Using novel MIS-C therapy, team successfully treats two patients with multisystem inflammatory syndrome in children
A team of pediatricians at the Medical University of South Carolina (MUSC) was the primary within the nation to enroll patients with the multisystem inflammatory syndrome in children (MIS-C), a rare but life-threatening complication of COVID-19, during a trial of remestemcel-L. This investigational cell therapy, developed and made by Mesoblast, New York, New York, had previously been shown safe and effective for other inflammatory conditions. The MUSC team reports in Pediatrics that the 2 children enrolled so far showed significant improvement within 24 hours of remestemcel-L administration.
"While it appears to several folks that COVID is not any big deal in children, this potential complication, although rare, is very, very serious," said MUSC Children's Health pediatric communicable disease physician, Allison Eckard, M.D., who is leading the local trial. "In South Carolina, we've had over 70 cases of MIS-C and one death. that's far more than we'd wish to see in children."
Too often, MIS-C isn't recognized until children are already critically ill.
"The tricky part about MIS-C is that, in many cases, the families do not know that the youngsters ever had COVID because, generally, children have very mild symptoms," said Eckard.
MIS-C also can be missed because its symptoms are vague, then it can easily be mistaken for other illnesses.
Although the explanation for MIS-C is unknown, Eckard speculates that the body's reaction to COVID-19 likely triggers a cascade of unchecked inflammation.
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"Although inflammation features a role to play within the body, excessive inflammation is bad and causes organ damage, organ failure, and tons of other complications," she said.
The current standard of look after MIS-C is nonspecific. It consists of using steroids or antibodies derived from donated plasma to showdown all aspects of the system.
"The novel treatment we are using maybe a bit more specific," said Eckard.
Remestemcel-L may be a cellular therapy derived from a special sort of cell that forms within the bone marrow.
"These bone marrow cells, donated by healthy adults, are known to showdown inflammation," said Eckard. they will target the precise parts of the system that are most relevant to MIS-C.
The cells also are ready to improve the inner lining of the vessel, targeting the foremost serious and potentially long-term effects of MIS-C: cardiac and cardiovascular involvement. Remestemcel-L improves vital signs and therefore the flow of blood to where it must be.
"We believe the guts to pump blood adequately to all or any the opposite organs," said Eckard. "The same applies to blood vessels and therefore the circulatory system. once they don't work properly, your body can't pump blood effectively to vital organs."
Nationwide studies have already been conducted on the utilization of remestemcel-L to treat graft-vs-host disease in children, a condition which will develop after receiving a bone marrow transplant, and MUSC participated in a number of them. Other studies have also assessed its usefulness for treating cardiac complications in adults. However, MUSC is that the only institution so far to use remestemcel-L for MIS-C.
"We always worry in pediatrics about the security of latest therapies. But due to our experience here at MUSC with many children enrolled during a remestemcel-L trial for graft-vs. host disease, we felt specialized about its safety profile," said Eckard.
Thus far, two children are enrolled within the trial and treated with remestemcel-L. They were chosen because they still had underlying inflammation and cardiac dysfunction despite having received standard-of-care therapy. Eckard hopes that the investigational therapy will help to guard these children against long-term cardiovascular complications.
"People are always very hesitant to undertake new things if they think the present therapy is functioning. But what I tell that's we don't know the long-term effects of MIS-C," said Eckard.
Although it's too early to understand if the novel therapy will protect against long-term complications, Eckard is heartened by the therapy's short-term effects, which were reported within the Pediatrics article.
"There were some dramatic improvements within 24 hours of giving the treatment in both of the youngsters, which is what compelled us to publish those two cases," said Eckard.
The remestemcel-L trial to be used in MIS-C aims to enroll 50 children with cardiac dysfunction nationwide. If the investigational treatment proves effective at treating MIS-C, it'd even be studied in children with other inflammatory syndromes.
Future studies will aim to work out if the administration of remestemcel-L is alone liable for the development of patients or if it still must be administered with plasma antibodies, which is that the current but costlier standard of care. the present standard-of-care therapies even have unwanted side effects which may be avoided if remestemcel-L is employed instead.
"We want people to be more conscious of the condition, but we also want to point out folks that we've had success with this therapy, in terms of safety and effectiveness, and encourage others to think about using it at their own institutions," said Eckard.
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