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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.