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Dr. Bruce H. Cohen
“We’re anxiously waiting to see what the FDA will now do with the new recommendations in hand but with the congressional prohibition also in place,” Dr. Dorsa said. “With the recommendations now out, we anticipate that pressure will mount for Congress to change its position because for now U.S. women with mitochondrial DNA disease will be precluded from this cutting-edge technology that could ensure they give birth to healthy children.”
Dr. Dorsa said that OHSU holds several patents related to MRT, but these patents have not been licensed to any commercial entity.*
Dr. Falk cited results from a 2015 survey of 92 women with mitochondrial DNA disease or at-risk carriers of mutated mitochondrial DNA who expressed their interest in MRT. Among the 92 respondents, 21 said that they were interested in having children. Of that smaller group, 90% said they would be interested in MRT when becoming pregnant.
Dr. Falk also noted that roughly 30,000-60,000 Americans have a mitochondrial disease, although many others might be affected but have never been diagnosed or undergone confirmatory sequencing of their mitochondrial DNA.* Many affected patients have a mutation in 1 of the 200 nuclear genes that can produce mitochondrial dysfunction and not in 1 of the 37 genes contained in mitochondrial DNA. Only women with a mutation in their mitochondrial DNA can benefit from MRT.
*Correction, 2/5/2016: An earlier version of this story failed to correctly distinguish all forms of mitochondrial disease from mitochondrial DNA disease.
*This story was updated 2/8/2016.
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