Mitochondrial Replacement Therapy: Let the Science Decide

Abstract

Mitochondrial replacement therapy (MRT) is an in vitro fertilization technique designed to prevent women who are carriers of mitochondrial diseases from passing on these heritable genetic diseases to their children. It is an innovative assisted reproductive technology that is only legal in a small number of countries. The United States has essentially stagnated all opportunities for research and clinical trials on MRT through a rider in H.R.2029 – Consolidated Appropriations Act, 2016. The rider bans clinical trials on all therapies in which a human embryo is intentionally altered to include a heritable genetic modification. This note argues that the rider should be amended to permit therapies such as MRT, which do not create artificial DNA sequences, while continuing to prohibit clinical trials on germline therapies that modify the sequence of a gene. MRT is distinct from the types of therapies that Congress intended to ban through the rider. Amending the rider would not automatically approve MRT trials, but rather allow the FDA to evaluate investigational new drug applications and determine whether individual trials may proceed. Without proper FDA oversight, carriers of mitochondrial diseases are denied access to a therapy that provides them with benefits they cannot enjoy by any other means, and researchers may look abroad to conduct the therapy illegally or dangerously. Further, the United States can look to other countries such as the United Kingdom as a model for how to proceed with research and trials on MRT in an ethical manner.