Sheba Medical Center is the site where the first patient in our Phase I/II clinical trial of Mitochondrial Augmentation Therapy (MAT) in Pearson syndrome, the first mitochondrial cell therapy trial ever conducted. The study is led by co-Principal Investigators, Professor Amos Toren M.D., Ph.D., MHA, head of the Division of Pediatric Hemato-oncology, and Elad Jacobi, M.D., head of the Pediatric Immunotherapy Center.
Sheba Medical Center also was the site of Minovia’s compassionate use program, through which three children with Pearson syndrome and one patient with Kearns-Sayre syndrome were treated. This program was the basis for the Phase I/II trial underway now, and results were presented at the annual meeting of the American Society of Hematology (ASH) and the United Mitochondrial Disease Foundation annual symposium. This report was named a “Best of ASH” presentation.