Focused Ultrasound–Facilitated Mitochondria Delivery Helps Treat Stroke


Key Points

  • A collaborative team used focused ultrasound to open the blood-brain barrier and enhance delivery of mitochondria to the brain cells in a preclinical model of stroke.
  • The treatment decreased the cerebral infarction volume and increased cell survival and function.

Intraarterial Transplantation of Mitochondria After Ischemic Stroke Reduces Cerebral Infarction

Intraarterially infused mitochondria permeate ischemic blood–brain barrier
Source: “Intraarterial Transplantation of Mitochondria After Ischemic Stroke Reduces Cerebral Infarction”

Could focused ultrasound help physicians offer a new treatment to patients who suffer from ischemic stroke? That is the question that researchers from the University of Virginia, St. John’s Neuroscience Institute, and the University of Oklahoma School of Medicine sought to answer in their recently published paper.

The team, led by neurosurgeon Mohammad Yashar S. Kalani, MD, recently published the results from their preclinical study, which showed that using focused ultrasound to open the blood-brain barrier (BBB) safely allowed the delivery of mitochondria into damaged brain tissue. In a model of stroke (cerebral artery occlusion), the mitochondrial treatment decreased the amount of damage and restored cellular function in the damaged tissue.

“Strokes are often debilitating and potentially fatal events,” said Dr. Kalani. “Stroke has a narrow treatment window, so we are working to create a treatment that can quickly reverse the damage caused when a stroke begins. Delivering a cellular component like mitochondria, which we know malfunction during stroke, to the damaged area of the brain allows the brain to rescue cells at risk for death and to protect itself from further injury.”

The intraarterial delivery method, which was facilitated by focused ultrasound, allowed the mitochondria to migrate across the BBB, enter the extracellular space, and integrate into various types of brain cells, where it made higher levels of adenosine triphosphate (ATP) and repaired the damaged area by reducing the volume of damage and increasing cellular viability. Importantly, the BBB-opening procedure did not create any complications.

The journal article offers a perspective for the clinical translation of this research. It notes that the approach is novel and that it demonstrates a clinically viable harvest and delivery method that “elevates the concentration of ATP, reduces infarct volume, and increases cell viability.” It notes that because the mitochondria are harvested from and received by the same organism, with minimal modification, the technique is more likely to be successful.

The advantages of this study are two-fold: 1. The mitochondria are autologous and unmodified, and 2. The mitochondria can be harvested and delivered in clinically viable time periods making translation of this study less cumbersome. A phase 1 study is in preparation at St. John’s Neuroscience Institute. 

See Stroke: Vascular and Interventional Neurology (Open Access)

New Scientist’s March 2023 article, “Restoring the Brain’s Mitochondria Could Slow Ageing and End Dementia,” mentions this research as one innovative solution to address neurodegeneration.