HFEA awards licence for pioneering mitochondrial research

September 14, 2005

Brussels, 13 Sep 2005

Scientists in Newcastle, UK, have been granted a licence to carry out research aimed at preventing the transmission of mitochondrial DNA disease, a serious and distressing hereditary disorder that is passed from mothers to their children and, in some patients, leads to a variety of diseases affecting the brain, muscles, heart and other organs.

Such diseases arise from DNA found outside the nucleus, and thus inherited separately from DNA in the nucleus. Whilst the decision offers the first realistic hope of an effective treatment for an entire class of serious genetic diseases, the news has been received with concern by pro-life groups who fear a legal door leading to designer babies has been opened.

The scientists involved stress that this research is only the very first step in a very difficult process. The proposed technique has proved safe in animal embryos, but it needs to be determined whether it can be safely used in human eggs. Abnormally fertilised eggs from consenting couples undergoing IVF treatment will be used in the research project.

The licence awarded by the UK's Human Fertilisation and Embryology Authority, HFEA, allows a research group at Newcastle University Medical School and the Newcastle Fertility Centre at Centre for Life, to carry out work aimed at developing a technique for replacing defective mitochondria with normal mitochondria in the one-cell egg. The research team from Newcastle University plans to transfer the components of a nucleus of a human embryo by a man and woman into an unfertilised egg from another woman, thus preventing mother passing certain genetic diseases to the unborn baby.

The application was initially rejected by the HFEA's Research Licence committee. However, after hearing a range of evidence on the genetics behind the proposed research, the scientific process that the research would involve and how the HFE Act should be interpreted, the Appeal Committee satisfied itself that the research activities were 'necessary and desirable' and that the use of embryos was necessary for the research, thus ruling that a licence for this research should be granted.

Mitochondrias are small complex structures, which exist in every cell of the body, except red blood cells and act as the cell's energy factories, producing all the energy required by our cells. Unlike other DNA, mitochondrial DNA is transmitted to offspring only via the mother. If this DNA is faulty, a mitochondrial disease occurs. At present, no treatment for mitochondrial diseases exists. Studies in mice show it is possible to prevent the transmission of mitochondrial disease by moving the pronuclei - the genetic material which will go on to form a nucleus - from a fertilised egg containing defective mitochondria and putting it into another fertilised egg with healthy mitochondria. The UK research, funded by the Muscular Dystrophy Campaign, now plans to do the same in humans, and check that transplanting the pro-nuclei works and is safe. The resulting egg would never be allowed to develop into a baby.

Professor John Burn, Medical Director of the Institute of Human Genetics at the Centre for Life in Newcastle, explained on BBC TV News that mitochondria were the 'batteries' that powered each cell in the human body. Thus, the new technique would be like changing a battery in a computer without affecting the hard disk.

A public consultation is underway in the UK on the technology and techniques used to assist human reproduction. The Newcastle team's legal adviser at the appeal hearing, James Lawford Davies, declared: 'This is a good illustration of why the government's review of the law in this area is necessary. It has taken 18 months and three hearings to obtain a licence, largely because the 1990 Act is very ambiguous when applied to research of this kind.'

CORDIS RTD-NEWS / © European Communities
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