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Mitochondrial Therapy: Hope or Hype?

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Recent studies on mitochondrial therapy have sparked a mix of enthusiasm and skepticism within the scientific community. Koning Shen, a postdoctoral researcher specializing in mitochondrial biology at the University of California, Berkeley, remarked on the intriguing aspects of this field. She is also a coauthor of a comprehensive review discussing the signaling roles of mitochondria published in the 2022 Annual Review of Cell and Developmental Biology. Shen pointed out that significant challenges remain in scaling up the extraction of mitochondria and developing effective methods for storing and preserving these organelles for broader therapeutic use.

Another expert in the field, Navdeep Chandel from Northwestern University, expressed caution regarding the implications of mitochondrial transplants. He noted that while there are many knowledgeable individuals thoroughly examining the subject, the primary question revolves around the exact mechanisms at play. Chandel remains skeptical that donor mitochondria can merely replace dysfunctional ones, suggesting instead that mitochondrial donation may activate stress and immune responses that could offer indirect healing benefits to damaged tissues.

Research involving animal models indicates that the efficacy of mitochondrial therapy may depend on the functionality of the mitochondria used. Lance Becker, who heads the emergency medicine department at Northwell Health in New York and studies mitochondria’s role in cardiac arrest, conducted an experiment comparing the effects of fresh mitochondria, thawed frozen mitochondria, and a placebo on rats after cardiac arrest. The findings revealed that the 11 rats treated with fresh mitochondria experienced enhanced brain function and a higher survival rate three days post-treatment, in stark contrast to the placebo group. The frozen-thawed mitochondria did not yield similar benefits.

Researchers emphasize the need for further investigation into the mechanisms of mitochondrial therapies, refinement of delivery methodologies, larger clinical trials, and a track record of successful outcomes before these therapies can seek FDA approval for widespread application in treating ischemia-reperfusion injuries. The aspiration is to establish a centralized bank of stored mitochondria, enabling a readily available source for various healthcare providers.

Becker acknowledged the nascent stage of this research, stating, “We’re so much at the beginning—we don’t know how it works. But we know it’s doing something that is mighty darn interesting.”

This article was originally published in Knowable Magazine, a nonprofit organization committed to making scientific knowledge accessible. Subscribe to Knowable Magazine’s newsletter.

 

Mitochondrial Therapy: Hope or Hype?
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