Neurodegeneration and Mitochondria Organelle Transplantation: “A Technology That Proof of Principle Suggest Is Ready for Prime Time”

It is known that mitochondrial dysfunction is associated with neurodegenerative diseases including amyotrophic lateral sclerosis (ALS). Researchers have tested the therapeutic efficacy of many mitochondrial targeted agents; however, results have been disappointing without significant impact on disease survival. Several groups have demonstrated that mitochondrial transfer of isolated normal healthy mitochondria to defective calls can restore functional recovery. Our experience with mitochondria organelle transplantation (MOT) in cancer cells led to investigating the technology for neurodegenerative diseases (NDs), especially ALS. The rationale was that if the uptake of normal mitochondria into cancer cells inhibited proliferation and glycolysis; then MOT might be a cell-based therapy for NDs. In this communication, we will present background research on MOT in vitro and in vivo cell culture and animal models respectively. This research evidence showed proof of principle of the technology. This fact led us to try the procedure on a desperate human ALS patient.