FDA clears trial testing Mesoblast cell therapy in kids with DMD
76 children sought for study aimed at putting treatment on path to approval
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The U.S. Food and Drug Administration (FDA) has given Mesoblast the go-ahead to launch a clinical trial testing its cell therapy remestemcel-L-rknd in children with Duchenne muscular dystrophy (DMD).
The trial aims to enroll 76 kids with DMD ages 5-9 who are receiving standard-of-care therapies. Participants will be randomly assigned to receive infusions of the experimental cell therapy or a placebo seven times over the course of nine months.
The study’s main goal is to determine whether cell therapy improves performance on the time-to-stand test, a standard measure of motor function. The study will be designed as registrational, meaning that if results are positive, Mesoblast hopes to use the findings as a basis for applying for FDA approval of the therapy. The company said it will collaborate with advocacy group Parent Project Muscular Dystrophy (PPMD) to help identify trial participants.
Remestemcel-L-rknd contains mesenchymal stromal cells (MSCs) derived from the bone marrow of healthy donors. MSCs are naturally occurring cells that can grow into other cell types, such as connective tissue and fat cells. MSCs also secrete anti-inflammatory signaling molecules that can dampen inflammation.
Remestemcel-L-rknd is approved by the FDA under the name Ryoncil for children ages 2 months and older with hard-to-treat acute graft-versus-host disease, a disorder that can develop after a bone marrow transplant in which transplanted cells attack the recipient’s own healthy tissue. Remestemcel-L-rknd was the first MSC-based treatment to win FDA approval for any disease.
‘Unique approach’ to DMD treatment
The FDA’s authorization of the DMD trial was based in part on safety data showing that the therapy was generally well tolerated in children with graft-versus-host disease. The FDA’s decision also took into account preclinical data indicating efficacy in models of DMD, which is a genetic disorder marked by progressive damage to muscles. According to Mesoblast, remestemcel-L-rknd may help preserve muscle damage by dampening inflammation.
“We are very pleased to have received clearance to proceed directly to a registrational study for DMD based on our preclinical data in DMD animal models and our extensive safety data in children with” steroid-refractory acute graft-versus-host disease, Silviu Itescu, chief executive at Mesoblast, said in a company press release. “Our experience with Ryoncil suggests that we may have a unique approach to help with this devastating disease in children.”
Aravindhan Veerapandiyan, MD, director of the comprehensive neuromuscular program at Arkansas Children’s Hospital, will serve as principal investigator on the study.
“This study represents an important step forward in potentially addressing the inflammatory component of DMD, a major driver of disease progression,” Veerapandiyan said. “By leveraging the anti-inflammatory effects of Ryoncil, we aim to intervene at a stage where muscle tissue may still be preserved, potentially altering the trajectory of the disease.”
