Are three-person babies headed to the US?

Now that the UK has taken steps toward allowing three-person babies, we wonder “Could the same thing happen in the US?” The answer may surprise you.

Just as Great Britain has become the first country taking steps toward approving three-person babies to be conceived using DNA from a mother and father plus a second woman, many are wondering if the US could be next. US regulators have indeed been studying this digital health dilemma but have adopted a much more conservative approach.

At issue is mitochondrial replacement, a procedure that allows mothers to bear their own genetically-related children without passing on certain diseases that stem from mutations of their mitochondrial DNA.

Mitochondria are the “battery packs” of cells (outside their nuclei) that contain small amounts of DNA that are passed on to future children exclusively from the mitochondria in her eggs. During the procedure, the unhealthy mitochondria is extracted from the birth mother and replaced with healthy mitochondria from the female donor.

About one in 4,000 babies are born in the US every year with some form of mitochondria mutation that can result in diseases such as muscular dystrophy, heart, kidney and liver failure and severe muscle weakness. According to the Mitochondria Research Society, more than 50 million adults are affected by diseases where mitochondrial dysfunction is present.

FDA studying the issue

The Cellular, Tissue and Gene Therapies Advisory Committee of the FDA held hearings in February 2014 to discuss the issue that will undoubtedly attract the interest of digital health innovators and others involved in digital health and wellness.

The FDA committee “reviews and evaluates available data relating to the safety, effectiveness, and appropriate use of human cells, human tissues, gene transfer therapies and xenotransplantation products which are intended for transplantation, implantation, infusion and transfer in the prevention and treatment of a broad spectrum of human diseases and in the reconstruction, repair or replacement of tissues for various conditions.”

And…

Although no specific trial has been discussed, the FDA is aware of growing interest among scientists in advancing their research to include such a trial.

According to Scientific American, Dr. Shoukhrat Mitalipov and his colleagues at the Oregon Health & Science University have expressed some interest in conducting an American trial, although details remain “confidential,” according to the journal.

And although the role of the FDA advisory committee is to “review and evaluate”—not debate—a briefing given to the committee did express concerns that would have to be addressed as part of the review process.

Dr. Deborah Hursh, Office of Cellular, Tissue, and Gene Therapies at the Center for Biologics Evaluation and Research, called on the committee to consider previous animal trials as a starting point.

“Prior to human clinical investigations, animal and in vitro studies provide the primary data upon which safety assessment is made,” said Dr. Hursh.

“In the context of the available animal models or other experimental systems for mitochondrial manipulation technologies, please consider the specific objectives of studies that would be necessary to support the safety and prospect of benefit of mitochondrial manipulation technologies prior to first-in-human clinical trials.”

Overall concerns

Among the concerns related to mitochondrial manipulation are:

• Inadvertent damage to the embryo
• Potential for abnormal fetal or embryo growth, resulting in possible defects
• Methods for determining enrollment and selection criteria for first-in-human clinical trials
• Methods of determining when the embryo is ready to transferred
• Follow-up genetic tests after manipulation
• Long-term follow up for children and future generations

According to Scientific American, after last year’s initial review, the FDA committee asked the US Institute of Medicine to produce a consensus report exploring the ethical and social policy issues relating to mitochondrial manipulation.  

The IOM Board on Health Sciences Policy met in January and is expected to meet again in March and May with two-day public meetings planned.

The board is considering the following:

• Whether manipulation of mitochondrial content should be considered germline modification, or viewed differently from modification of nuclear DNA, from a social and ethical perspective
• The implications of manipulating mitochondrial content both in children born to women as a result of participating in these studies and in descendants of any female children
• Ethical issues in providing “consent” or “permission” to accept risks on behalf of a child who does not exist
• Ethical and social issues that arise if a child is born with genetic material from three individuals

A final report on this fascinating and challenging issue is expected sometime in 2016.

The nuviun industry network is intended to contribute to discussion and stimulate debate on important issues in global digital health. The views are solely those of the author.