Deciding whether to license new treatments to avoid passing on mitochondrial disease

Posted on March 1, 2011 by


The Human Fertilisation and Embryology Authority (HFEA – which regulates fertility clinics and all research using human embryos in the UK) is looking at methods of assisted conception that could be used to avoid passing on mitochondrial disease. They are doing a very quick review, aiming to send a report to the Department of Health in April to inform decisions over what treatments should be permitted. They’d like input on the science but will need it by 15 March.


Some genetic conditions are passed on by DNA in mitochondria. Research is ongoing to find methods to stop these unhealthy mitochondria from being passed on. Some of this research involves making changes to an embryo’s DNA by transferring the nucleus from an embryo with unhealthy mitochondria into an embryo with healthy mitochondria. This procedure is currently allowed for the purposes of research to see if it works but cannot be used to treat people. The HFEA’s Scientific and Clinical Advances Committee has been looking at these techniques to see if there is a case for them to be allowed for treatment. So far, they have concluded that more safety testing is needed.

What has happened now?

Research into these techniques is ongoing and advances are being made.  They offer huge potential for those at risk of passing on mitochondrial disease.

The Secretary of State has asked the HFEA to review the current expert understanding of the research. To inform this, the HFEA is asking for scientific experts to send them  information on the safety or effectiveness of methods to avoid the transmission of mitochondria disease through assisted conception.

They’ve set a very tight deadline of 15 March so they can gather evidence ahead of a workshop on 25 March and aim to report back to the Department of Health in April. More background and all the details on how to submit evidence here.

Why is this of interest to medical research charities?

If these techniques can be developed safely and effectively, they offer the potential to stop conditions such as muscle disease caused by mitochondrial myopathies being passed on and help lots of people. The Muscular Dystrophy Campaign has been funding some research at Newcastle University into these procedures – worth checking out their website here which gives more detail about the current research and how it works.

Posted in: Policy