Human mitochondrial transplantation research

Mitochondrial transplantation is an emerging medical technique designed to restore energy to diseased cells by transferring healthy mitochondria to affected areas. One method involves autologous transplantation, where mitochondria are extracted from a patient’s own tissue, such as skeletal muscle, through processes like homogenization and centrifugation.

In a pioneering experimental procedure conducted under an FDA-authorized compassionate use protocol, researchers at the Icahn School of Medicine at Mount Sinai performed the first human transplant of autologous mitochondria to treat blindness. Doctors extracted healthy mitochondria from a 26-year-old patient’s thigh muscle and injected them into her eyes to provide energy to dormant retinal nerve cells following a severe brain hemorrhage.

While the procedure was biologically successful and showed no signs of rejection or inflammation, it did not restore functional vision. Although the patient’s pupils—which had been unresponsive for 71 days—began reacting to light within 48 hours, the effect was temporary and faded after approximately four weeks. Scientists noted that while the treatment provides cellular energy, it does not repair damaged neural pathways between the eye and the brain. However, the safety and biological tolerance demonstrated in this case may open new research avenues for treating degenerative conditions like glaucoma and other optic neuropathies.