Contradiction keeps US policy at standstill on mitochondrial therapy

June 09, 2016

Scientists believe they can now remove disease-causing mitochondrial DNA from human embryos, providing new cures for previously untreatable conditions, but the policy signals coming from Washington DC are in stark contradiction, according to a new Viewpoint essay published in JAMA June 9, 2016.

On Feb. 3, 2016, the Health and Medicine Division of the National Academies issued a report on mitochondrial replacement therapy (MRT) commissioned by the Food and Drug Administration. The division (formerly known as the Institute of Medicine) recommended that under certain conditions, MRT clinical trials should be allowed to proceed. But just six weeks before, President Obama signed an appropriations bill that included a bit of language essentially forbidding those trials, the JAMA authors wrote.

"One big step forward was taken by the IOM report when it concluded that it is "ethically permissible" to embark on first-in-human clinical trials of MRT subject to rigorous safety and efficacy imperatives," wrote Dr. Eli Adashi, former dean of medicine and biologic sciences at Brown University and I. Glenn Cohen, professor of law at Harvard University. "However, two steps back were taken with the enactment of a policy rider which precludes the FDA from further consideration of MRT."

In MRT, when a mother has mitochondria with problematic DNA, scientists propose to replace it in an embryo with that of a donor. The reconstituted embryo would then be implanted in the mother. If successful, that replacement procedure would ensure that the nuclear DNA of the resulting child would come from mom and dad, but would be paired with mitochondrial DNA (from a third party donor) that would not carry the risk of disease.

Because males do not pass down mitochondria from generation to generation, the National Academies report recommended that trials proceed only in male embryos. That would prevent altering whole lineages.

Meanwhile in the United Kingdom, the government has approved MRT clinical trials and some might begin this year. But despite the National Academies recommending a similar advance to the FDA, legislation has left U.S. policy at a standstill, wrote Adashi and Cohen.

"Whether or not the eventual births of disease-free children in the UK will change congressional hearts and minds remains to be seen," they wrote. "Failing such, progress in the prevention of mitochondrial DNA diseases will remain the domain of a biomedical enterprise an ocean away."
-end-


Brown University

Related Mitochondrial DNA Articles from Brightsurf:

Single-cell analysis provides new insights into mitochondrial diseases
Investigators led by a team at Massachusetts General Hospital (MGH) have made discoveries at the single cell level to uncover new details concerning mitochondrial diseases-- inherited disorders that interfere with energy production in the body and currently have no cure.

How to precisely edit mitochondrial DNA
A gene editing tool based on a bacterial toxin can make precise changes to mitochondrial DNA inside cells.

New molecular tool precisely edits mitochondrial DNA
The precision editing technologies that have revolutionized DNA editing in the cell nucleus have been unable to reach the mitochondrial genome.

First simulation of a full-sized mitochondrial membrane
Scientists from the University of Groningen have developed a method that combines different resolution levels in a computer simulation of biological membranes.

Cell biology -- maintaining mitochondrial resilience
Mitochondria cannot autonomously cope with stress and must instead call on the cell for help.

Increasing food intake by swapping mitochondrial genomes
To uncover the relationship between variation in genes and phenotypic diversity, geneticists use a set of fully sequenced fruit-fly genomes.

Structure of a mitochondrial ATP synthase
SciLifeLab researchers Alexander Mühleip and Alexey Amunts from Stockholm University solved the structure of a mitochondrial ATP synthase with native lipids.

New research tool for studying mitochondrial disorders and aging
Researchers at Karolinska Institutet in Sweden have developed a new research tool for studying how mitochondrial protein synthesis is affected by disease, pharmaceuticals, ageing and different physiological situations such as exercise and diet.

Why the lettuce mitochondrial genome is like a chopped salad
The genomes of mitochondria are usually depicted as rings or circles.

Researchers can finally modify plant mitochondrial DNA
Researchers in Japan have edited plant mitochondrial DNA for the first time, which could lead to a more secure food supply.

Read More: Mitochondrial DNA News and Mitochondrial DNA Current Events
Brightsurf.com is a participant in the Amazon Services LLC Associates Program, an affiliate advertising program designed to provide a means for sites to earn advertising fees by advertising and linking to Amazon.com.