FDA OKs stem cell therapy for kids with inflammatory syndrome linked to COVID-19
An expanded access protocol filed with the FDA gave clinicians access to an investigational allogenic mesenchymal stem cell product to use as therapy for multisystem inflammatory syndrome in children, or MIS-C, a complication of COVID-19.
The announcement was made by Mesoblast Limited, which manufactures the investigational therapy, remestemcel-L.
Under the expanded access protocol (EAP), children with MIS-C who are aged between 2 months and 17 years may receive one or two compassionate use doses of remestemcel-L within 5 days of referral, the company said. Access is available under the company’s existing investigational new drug application.
“The extensive body of safety and efficacy data generated to date using remestemcel-L in children with graft-versus-host disease suggest that our cellular therapy could provide a clinically important therapeutic benefit in MIS-C patients, especially if the heart is involved as a target organ for inflammation,” Mesoblast Chief Medical Officer Fred Grossman, DO, FAPA, said in a statement. “Use of remestemcel-L in children with COVID-19 builds on and extends the potential application of this cell therapy in COVID-19 cytokine storm beyond the most severe adults with acute respiratory distress syndrome.”
Remestemcel-L uses culture-expanded mesenchymal stem cells from the bone marrow of an unrelated donor, Mesoblast said. It is administered through a series of IV infusions.
According to the company, the therapy “is believed to have immunomodulatory properties to counteract the inflammatory processes that are implicated in several diseases by down-regulating the production of proinflammatory cytokines, increasing production of anti-inflammatory cytokines and enabling recruitment of naturally occurring anti-inflammatory cells to involved tissues.”
According to the FDA, expanded access potentially creates a pathway for patients who have an immediate life-threatening condition to be able to gain access to an investigational medical product for treatment outside of clinical trials.
Perspective
Back to TopThis is an interesting advance in the potential treatment of MIS-C, further targeting the immune reaction to recent infection as the cause of the organ dysfunction. Multiple reports have been recently published highlighting the heterogeneous and sometimes very severe presentation of MIS-C. In severe cases, especially in those with significant cardiac involvement, this syndrome has resulted in fatalities. The current treatment has centered on IV immune globulin and steroids, which it is hoped will modify the immune response and possible cytokine storm. Remestemcel-L would be another step in this immune modification. I would imagine that this treatment would be prioritized for the most severe cases.
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