Right now, British and American scientists have developed techniques to prevent life threatening and incurable diseases that affect tens of thousands of people around the world. And this week, the Parliament of the United Kingdom voted to pass regulations that will bring clinics one step closer to performing this technique, mitochondrial replacement therapy. Yet, for the more than 12,000 women with mitochondrial diseases in the United States at risk of having children with mitochondrial diseases, they face a ban on this lifeline to have healthy children. This issue, so critical to public health, is being half ignored, half slighted by Congress, the president and the relevant agencies. This is Washington gridlock at its worst, and it has to stop.
Ironically, the techniques to prevent mitochondrial diseases were developed both in the United States and in the United Kingdom, but only women in the U.K. will have access to these treatments after British government intervention this week. The U.K. has taken a leading role in the debate, regulation and approval of MRT, and its efforts should be commended.
The New York Stem Cell Foundation research institute, Columbia University Medical Center and the Oregon Health and Science University are three of the leading groups, along with Newcastle University in the U.K., that developed these techniques. And yet, the United States has largely ignored the value of MRT to the women in our country at risk of passing these devastating diseases to their children.
A woman with a family history of mitochondrial diseases or who has had a child with such a disease has few options if she wants to have healthy children. She may elect to have no children or to adopt a child. She can undergo an egg retrieval process at an in-vitro fertilization clinic, where pre-implantation genetic diagnosis can select eggs with the lowest number of mutant mitochondria to potentially reduce — but not fully eliminate — the risk of the child developing mitochondrial disease.
These options are not acceptable to many future parents, knowing the technology could be made available to allow them to have genetically related, healthy children.
In February 2014, the Food and Drug Administration’s Cellular, Tissue, and Gene Therapy Advisory Committee met to discuss MRT but a year later, there has been little additional progress from the government.
Indeed, several animal studies on MRT have been conducted, yet many in the U.S. have asked for further animal investigation. The New York Stem Cell Foundation Research Institute and others continue to perform additional studies, but the scientific community should be looking at the best ways of conducting these additional studies while monitoring the progress of human trials in parallel — not waiting for safety and efficacy confirmation that will never come without actually doing the trials.
This debate over mitochondrial replacement therapy, like the human embryonic stem cell debate in years past, cannot live in limbo for years, delaying our ability to save lives now. Action by the federal government and Congress is needed to break the logjam and stymie any efforts that slow the progress of science. If introduction of a bill such as the Stem Cell Research Enhancement Act or an executive order signed by the president is needed then it must happen. During the debate over the Stem Cell Research Enhancement Act, members from both sides of the aisle worked together to advance science and offer hope to patients. We are grateful the U.K. parliament had the foresight to pass legislation that will advance this important science to save lives. This is not only an issue of the FDA and our regulatory bodies; our federal government and Congress cannot cede this work to other countries. They must send a clear message to our scientists and the FDA that this work must happen here and now. The United States must lead the way in innovation in science and offering hope to so many Americans suffering from disease and disabling conditions.
The U.S. needs to take a page out of the U.K.’s book and take advantage of the years they spent assessing the benefits and risks of MRT. The women who are suffering from these diseases and are at risk of passing them on to their children deserve the autonomy and the right to make their own informed decisions on how they will build their families. With the advent of new technologies, including MRT, it is time for the U.S. to once again be a world leader in offering our citizens the best medical care.
Susan L. Solomon is CEO of The New York Stem Cell Foundation.