MINNEAPOLIS, MN- September 3, 2019 -The University of Minnesota (U of M) Medical School is at the forefront of developing a cell-based therapy for Duchenne muscular dystrophy (DMD), and now its team of researchers leading this work has been awarded a $945K grant from Duchenne UK to catalyze advancement of current preclinical discoveries. DMD is one of the most common fatal genetic diseases diagnosed in childhood. Patients who suffer from DMD cannot produce the protein that is vital for muscle strength and function resulting in a progressive deterioration of muscle.
"It is really exciting to see this work progress from bench to clinical manufacturing lab. At the completion of this work, we hope to be in a position to be ready to start a clinical, which is a dream come true," said Rita Perlingeiro, PhD, Professor of Medicine at the U of M Medical School, and the principal investigator of these pioneer studies. "It takes a number of experts with different skill sets to achieve what we have so far, and at the University of Minnesota, we are uniquely positioned to succeed in this cell therapy program."
Stem cells, called human induced pluripotent stem cells (iPSCs), replace the diseased muscle tissue with healthy muscle and can create healthy myofibers (mature muscle cells) which are able to regenerate. Perlingeiro and her team will use the grant to manufacture the stem cells and test dosing, safety, and toxicity. If the project is successful, the group will plan a Phase I Clinical Trial to test this approach on patients. Members of the collaborative team include the U of M Medical School's John Wagner, MD, professor in the Department of Pediatrics and Director of the Institute of Cell, Gene and Immunotherapy, David McKenna, MD, professor in the Department of Laboratory Medicine and Pathology and director of Molecular and Cellular Therapeutics, and Peter Karachunski, assistant professor in the Department of Neurology. Robert Schumacher, PhD, Scientific Director for the U of M Center for Translational Medicine is also part of the team.
"We are very excited to have the funding from Duchenne UK to continue this work and move our project to the next level," said Wagner. "If successful, this project could give hope for many patients with muscular dystrophy- including but not limited to DMD."
Duchenne UK funds more than 60 projects worldwide. These range from seed funding for gene therapy, to funding clinical trials, to expanding clinical trial capacity so more patients can access research. Perlingeiro and her group are one of four recipients of the DMD INSPIRE Major Grant Call, which invests $1.5 million in developing treatments for Duchenne Muscular Dystrophy (DMD).
"We are very excited to be supporting the work of the University of Minnesota's world-leading researchers," said Emily Crossley and Alex Johnson, co-founders of Duchenne UK. "Our organization is committed to accelerating therapies into clinical trials. This work has the potential to transform the lives of DMD patients and we are pleased to be able to support this ground-breaking research."
About the University of Minnesota Medical School
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