Future of Mitochondrial Replacement Techniques Need to Be Decided by US Medical Community

An online commentary published by the New England Journal of Medicine argues that the U.S. medical community will be crucial in determining future applications of, and the ethical framework surrounding, mitochondrial replacement techniques (MRTs).

The United Kingdom recently addressed policy issues regarding MRTs. But the United States has a different regulatory process pertaining to the potential clinical use of MRTs, and the authors of the commentary – titled “Mitochondrial Replacement Techniques — Implications for the Clinical Community“ – point out that medical professional organizations, as well as practitioners and societies, will be fundamental in laying an ethically acceptable foundation for clinical applications of MRTs.

The authors – Marni J. Falk, Alan Decherney, and Jeffrey P. Kahn – were members of a committee at the Institute of Medicine that also released a recent report on MRTs, “Mitochondrial Replacement Techniques: Ethical, Social, and Policy Considerations.” The report was commissioned by the U.S. Food and Drug Administration (FDA).

Several debilitating diseases are related to mitochondrial DNA (mtDNA), and mitochondrial conditions are passed only from a mother to a child. With the help of MRTs, researchers hope to one day be able to exchange a woman’s mtDNA without altering her genomic DNA, so as to remove the mutated genes from a future child. The technique has so far only been tried in experimental animals.

“If and when initial investigations are undertaken, critical safety and efficacy questions will remain before regulatory approval or clinical use can occur,” the authors emphasized in their commentary.

They also highlighted that the framework on how to proceed with MRT research in humans could impact the use of other highly specialized therapies and methods, and noted that mitochondrial diseases could become a “poster child for highly targeted, ‘personalized’ medicine.”

The authors, however, also pointed out that any potential benefits of the technique will only aid the yet unborn, and will not help people who live with mitochondrial disease today. MRTs will also not prevent the occurrence of new mtDNA mutations.

“The first use of MRTs in humans is uncharted scientific territory, and requires caution and commitment to the safety of any potential offspring born through use of the techniques,” said Dr. Kahn in a press release. “Any path forward must assess and avoid not only health risks, but take account of psychological effects for the individuals born as a result of these techniques, as well as the social implications of such reproductive technologies.”

The commentary raises two major points from the FDA-commissioned report. First, the primary ethical concern is the safety of the child affected by the procedure. Second, in initial trials of the technique, only male embryos should be selected to lessen the risk of transferring potential flaws through the mtDNA.