You are hereJuly 23, 2020
Expanded Access Protocol Initiated for Stem Cell Treatment for Children With MIC-C Associated With COVID-19
MELBOURNE (AU), July 2020 — Mesoblast Ltd. has announced that an expanded access protocol (EAP) has been initiated in the United States for compassionate use of its allogeneic mesenchymal stem cell (MSC) product candidate remestemcel-L in the treatment of COVID-19 infected children with cardiovascular and other complications of multisystem inflammatory syndrome (MIS-C).
Patients aged between 2 months and 17 years may receive one or two doses of remestemcel-L within five days of referral under the EAP. The protocol was filed with the United States Food and Drug Administration (FDA) and provides physicians with access to remestemcel-L for an intermediate-size patient population1 under Mesoblast’s existing Investigational New Drug (IND) application.
According to the FDA, expanded access is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available. MIS-C is a life-threatening complication of COVID-19 in otherwise healthy children and adolescents that includes massive simultaneous inflammation of multiple critical organs and their vasculature. In approximately 50 percent of cases this inflammation is associated with significant cardiovascular complications that directly involve heart muscle and may result in decreased cardiac function. In addition, the virus can result in dilation of coronary arteries with unknown future consequences.
Recent articles from Europe and the United States have described this disease in detail. Mesoblast’s chief medical officer, Fred Grossman, M.D., said, “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. 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.”