Pluristem Therapeutics Inc., a leading developer of placenta-based cell therapy products, has reported positive data from preclinical studies of its PLX-PAD cells in the treatment of Duchenne muscular dystrophy. The studies were conducted in conjunction with ADI, the Association Duchenne Israel, whose members are parents of children with Duchenne. They are committed to helping to find a cure for Duchenne muscular dystrophy through research, clinical trials, and advocacy.
Duchenne muscular dystrophy is the most common neuromuscular disorder, and affects roughly one in 3,500 boys. The disease causes progressive muscle weakness, and leads to severe disability and death. There is currently no cure.
Following Pluristem's announcement of positive results from a Phase II clinical trial of PLX-PAD as a treatment for muscle injury, the Association Duchenne Israel approached Pluristem with a request to study PLX-PAD cells in Duchenne muscular dystrophy. Pluristem donated PLX-PAD cells for the preclinical studies, and the association supported the research in cooperation with Science in Action Ltd.
The studies demonstrated that, in a mouse model of muscular dystrophy, PLX-PAD cells reduced creatine phosphokinase (CPK), a marker of muscle degeneration or injury, by approximately 50% as compared to placebo. CPK levels were measured via a blood sample taken 5 days after each intramuscular PLX-PAD injection made at day 15 and day 29 of the study. Histological analyses of quadriceps and diaphragm muscles show PLX-PAD reduced levels of inflammation and necrosis, a type of cell death, and induced regeneration of muscle tissue.
Hila Krupsky, CEO of ADI, the Association Duchenne Israel, stated, "These preclinical data suggest that PLX-PAD cells could possibly be a breakthrough therapy to help treat symptoms of Duchenne muscular dystrophy. We are thankful for Pluristem's donation of PLX-PAD and are eager to continue studying the cells since new therapeutic approaches are needed to manage this disease, save children's lives, and give them hope and a chance for the future."
"Because PLX-PAD cells have already displayed efficacy in muscle regeneration in a Phase II muscle injury study, we believe our cell therapy may potentially be beneficial in Duchenne muscular dystrophy in human clinical trials," said Pluristem Chairman and CEO Zami Aberman. "We admire the commitment of the Association Duchenne Israel to find a cure for Duchenne muscular dystrophy, and we will work closely with them in an effort to develop a treatment for the children around the world who suffer from this disease."