December 15, 2016

expert reaction to HFEA decision to permit the clinical use of mitochondrial donation techniques in the UK

The HFEA decided to permit the clinical use of mitochondrial donation techniques in the UK.

Dr David J Clancy, Lecturer, Lancaster University, said:

“As the body authorizing this treatment the HFEA must have applied a value to the benefit MRT provides. The benefit is that a woman with disease-causing mitochondria can have a child who is genetically related to her. The alternative, to avoid disease, is IVF by donor egg or adoption.

The reason they should have applied a value is that the technique they are enabling is not risk-free. If it were risk-free there would be no need to consider cost vs benefit. But there is a risk that the disease-causing mitochondria will be transferred and expand in number so that the offspring will suffer from the disease. We know this can happen.

With the technique as it is, producing a child with the disease-causing mitochondrial is more a probability than a possibility. We should be prepared to face this person when it becomes clear their life will be severely shortened or otherwise disabled and tell them that their situation was worth it because x number of women were able to have healthy children genetically related to them.

There might be a way to minimize culpability: only offer MRT to women who are adamant they want a child genetically related to them – women who refuse IVF by donor egg. For these women, for whom the need to have a genetically related child is a driving existential need, this technique could be offered. Although it is not clear, perhaps this group is who the HFEA describes as ‘parents at very high risk of having a child with a life-threatening mitochondrial disease’.”

Prof. Dagan Wells, Associate Professor at the NIHR Biomedical Research Centre, University of Oxford, said:

“Since the human genome was sequenced, over a decade ago, we have become increasingly good at diagnosing inherited disorders. However, the possibility of curing such conditions has remained frustratingly out of reach. Parents at risk of transmitting a disorder to their children can have their pregnancies tested, but if the foetus is found to be affected, the options are to terminate the pregnancy or continue, knowing that the baby will suffer from the condition, neither of which is an easy path to take.

“Mitochondrial donation offers a real opportunity to cure a class of potentially devastating inherited conditions and will bring hope to hundreds of affected families in the UK. The UK has led the world in terms of the debate over the ethical acceptability of the procedures and has taken a responsible, careful approach before finally approving clinical application. Research teams, such as those of Professors Herbert and Turnbull in Newcastle, have done sterling work to assess safety of methods for replacing defective mitochondria and their research has provided much reassurance in that regard. The HFEA’s decision to allow clinics to apply for permission to perform mitochondrial donation finally opens the way to begin using this technology for the benefit of families that have faced much heartbreak and hardship as a result of carrying a mitochondrial disorder.”

Prof. Adam Balen, Chair of the British Fertility Society said:

“Today’s decision by the regulator marks a major milestone in helping families to overcome mitochondrial disease, which can have devastating effects on people’s lives.

“There are many different medical disorders that can be described as mitochondrial disease. They range from mild to severe or life threatening.

However, mitochondrial DNA is only inherited from mothers and women can be at risk of passing on serious disease to their children. Preventing the transmission of mitochondrial disease will allow women carrying the mutations to give birth to children free of the disease.

“The treatment, which involves eggs donated from a healthy woman and the transfer of the fertilised nucleus with the genetic material from the mother and father of the affected family, will be carefully regulated and scrutinised by the HFEA.

“Clinics will now need to apply for a licence and the regulator will grant them taking into account each application extremely carefully. Scientists in Newcastle have lead the way with this ground-breaking research and the Centre for Life in Newcastle is likely to be the sole clinic offering this new technology at first.

“This marks a momentous and historic step and we hope families next year will begin their journey to eradicate these genetic diseases.”

Mr Alastair Kent, Director, Genetic Alliance UK, said:

“Mitochondrial replacement therapy is an increasingly promising new option for families to overcome the impact that these serious rare genetic conditions have on their lives. This step brings us closer to the end of a pathway which we hope will end at the delivery of a new treatment for couples that currently have no alternative.

A licence application should of course come at the point that a provider is confident of their ability to provide this service. We look forward to that next step!”

Mr Simon Stevens, Chief Executive of NHS England, said:

“The NHS has given the world medical innovations ranging from modern cataract surgery, new vaccines and hip replacements, and today we take a world-leading next step to harness ground-breaking science for the benefit of our children.

“As the country prepares for Brexit, this is another example of how the NHS both supports and benefits from our vibrant and internationally-competitive life sciences sector.”

Mr Steve Bates, CEO of the Bioindustry Association said:

“Today’s decision by a rigourous, democratically overseen, and appropriate regulatory regime in the HFEA not only paves the way to help affected families but increases the attractiveness of the UK as a destination for global investment in pioneering life science and its associated businesses.

Mr James Palmer, Clinical Director of Specialised Services at NHS England said:

“Mitochondrial diseases can be devastating and life limiting as well as hugely costly to the NHS to treat. This trial will, for the first time, give women living with mitochondrial disease the option of having a baby without passing on their condition and is a shining example of how the NHS is leading the world in developing cutting-edge innovative new medical interventions.”

Prof. Mary Herbert, Professor of Reproductive Biology, Newcastle University, said:

“We welcome today’s decision from the HFEA and it is enormously gratifying that our many years of research in this area can finally be applied to help families affected by these devastating diseases.

“The development of the new techniques depended entirely on the willingness of women in the North East to donate their eggs for research.  Our research to improve the techniques further is still ongoing and we will continue to need donated eggs for this.

“Importantly, now that that we are moving forward towards clinical treatments, we will also need donors to donate eggs for use in treatment to prevent affected women transmitting disease to their children.”

Prof. Sir Doug Turnbull, Director of the Wellcome Centre for Mitochondrial Research at Newcastle University said:

“We are delighted by today’s decision as it paves the way offering mitochondrial donation as part of an NHS-funded package of care for families affected by mitochondrial DNA disease.

“Newcastle is a major referral centre for the women with mitochondrial DNA mutations in the UK and it will be hugely welcomed as it provides them reproductive choice.

“In Newcastle, we will be aiming to treat up to 25 carefully selected patients a year with the mitochondrial donation technique as a clinical risk reduction treatment. We will also provide long term follow up of any children born.”

Prof. Frances Flinter, Consultant of and Professor in Clinical Genetics at Guy’s and St Thomas’ NHS Foundation Trust & member of the 4th scientific review group, said:

“This is wonderful news for families who have, in some cases, waited years or even generations for the chance of having a healthy baby. Mitochondrial disorders can be very serious, progressive conditions and some couples know that they will never be able to have a healthy child of their own without trying this new therapeutic approach.

“I am delighted that the HFEA is willing to consider license applications now. It is infinitely preferable that the early clinical trials should be done in a tightly regulated system in the UK, with long term follow-up of any children born, rather than in countries where there is no regulation or oversight.”

Prof. Sir Mark Walport, Government Chief Scientific Adviser, said:

“I welcome this careful and considered assessment by the HFEA. The UK leads the world in the development of new medical technologies. This decision demonstrates that, thanks to organisations like the HFEA, we also lead the world in our ability to have a rigorous public debate around their adoption.”

Prof. Sir Robert Lechler, President of the Academy of Medical Science, said:

“We are very pleased with the HFEA decision to introduce mitochondrial replacement techniques into clinical practice and fully support their decision.

“For many couples, mitochondrial replacement techniques will offer the only chance to conceive a biologically related child, free from mitochondrial disease.

“An expert panel recently found these techniques are sufficiently safe and appropriate for clinical use in patients, and the HFEA will provide a robust regulatory framework for their use. We are also pleased to see that cases will be reviewed individually and procedures carried out only in clinics that are approved to do so.

“Medical science exists to improve lives. Today’s decision by the HFEA means this groundbreaking research can now be translated from theory into practice and transform lives in the clinic.”

Mr Robert Meadowcroft, CEO of Muscular Dystrophy UK, said:

“Today, this historic decision will open the door to the first licensed treatments being offered to eligible women affected by mitochondrial disease.

“Families have, understandably, had to wait through years of thorough ethical, safety and public reviews.

“We know of many women who have faced heartache and tragedy, and the sorrow of stillbirths, while trying to start their own family, and this decision gives them new hope and choice for the first time.

“We recognise this approach is not without some uncertainty, and, in any trial, success cannot be guaranteed. However, it is important that women are able to make informed choices by understanding the risks and the potential benefits.”

Dr Jeremy Farrar, Director of the Wellcome Trust, said:

“This is a landmark day for people living with mitochondrial disease.

“For over 10 years Wellcome has supported brilliant researchers at Newcastle University pioneering mitochondrial donation. It’s right there’s been such a thorough process to get to this stage. Our long-term support for the science, and close co-ordination with patient groups, policy-makers and regulators have been critical in reaching today’s outcome. Through our funding of the Wellcome Centre for Mitochondrial Research we look forward to continuing to support this work.

“We must recognise, though, that for some affected families this decision may have come too late. The scientific community must continue to work closely with policy and the regulatory system so future innovative, cutting-edge techniques reach patients faster.”

Dr Meenakshi Choudhary, Consultant Gynaecologist, Newcastle Fertility Centre, & associate lecturer at Newcastle University, said:

“Newcastle has been at the forefront of this pioneering technology which has in a great part been due to the generosity of the women in this region. Their help has enabled the research to progress to this point where the UK is going to be able to treat women affected for the first time.

“Egg donation for mitochondrial donation treatment differs from other forms of egg donation in that the donor’s nuclear genetic material will not be used for treatment.”

Dr Jane Stewart, Consultant in Reproductive Medicine and Gynaecology & Head of Department, Newcastle Fertility Centre, said:

“We are delighted that the wealth of research undertaken is now coming to fruition.  As you can imagine we have been preparing for licence application for some time and look forward to moving into the treatment phase of this important work.”

Mr James Lawford Davies, Partner, Hempsons, said:

“Last year Parliament passed regulations which made it possible for the HFEA to permit the use of mitochondrial donation in clinical practice. The HFEA has today confirmed that it will now consider applications from specialist centres to provide this ground-breaking treatment, making the UK the first country in the world to formally licence the technology.”

Prof Allan Pacey, Professor of Andrology, University of Sheffield, said:

“Mitochondrial disease has a devastating effect on the lives of families and currently there is no cure. Therefore, I am delighted to hear that the HFEA have today given the final go-ahead for licences to be issued which will allow women whose families are affected by mitochondrial disease to have access to the new technique of mitochondrial donation. This decision is a testament to the hard work of the doctors and scientists in the UK who have undertaken years of painstaking work in order to get to this stage, as well as the support of our parliamentarians and regulators who have spent time carefully considering the issue. I truly hope that 2017 sees the technique used to revolutionise the lives of UK families who have suffered as a consequence of this debilitating disorder. What a great end to the year.”

All our previous output on this subject can be seen at this weblink: http://www.sciencemediacentre.org/tag/mitochondrial-dna/

Declared interests

Prof Dagan Wells: No conflicts of interest.

Mr Alastair Kent: I am employed by Genetic Alliance UK, the national charity working to improve the lives of patients and families affected by all types of genetic conditions. We are an alliance of over 160 patient organisations. Our aim is to ensure that high quality services, information and support are provided to all who need them. We actively support research and innovation across the field of genetic medicine.

Policy and practice in generating resources to support the work of Genetic Alliance UK (including that carried out as part of our projects SWAN UK and Rare Disease UK) is determined by the Trustee Board of the Charity, the members of which are nominated and elected by the patient organisations which comprise Genetic Alliance UK’s membership.

Having determined a strategy and a work plan Genetic Alliance UK then seeks resources to implement it from a wide range of potential funders including National Governments, the EU, the pharmaceutical and medical devices industry, the Medical Research Council, Wellcome Trust, The Big Lottery and others. Patient organisations also pay a subscription according to their size. Genetic Alliance UK does not accept unsolicited grants that are contingent on the organisation carrying out work on behalf of a third party that would be counter to the interests of patients and families with genetic disorders or which would hinder the effective delivery of the strategy endorsed by the Trustee Board.

Full details of our funding policy can be found here: http://www.geneticalliance.org.uk/ethicalcollaborationpolicy.htm

All our income is reported in our annual reports available here: http://www.geneticalliance.org.uk/annual-report.htm

I am invited to speak at a wide range of conferences and meetings on issues arising from my role as director of Genetic Alliance UK, the expenses for which are met directly or reimbursed to me by the organisers. These include public, private and voluntary sector bodies in the UK and internationally. I am also the Chair of the UK Rare Diseases Forum, the body set up by the four health ministers of the UK to monitor implementation of the UK Strategy for Rare Diseases and to report on progress every two years, and a member of NHS England’s Rare Disease Advisory Group.

Prof Mary Hebert: Mary Herbert is employed by Newcastle University as Professor of Reproductive Medicine based at Newcastle Fertility and Wellcome Trust Centre for Mitochondrial Research.

Prof Mary Hebert: Mary Herbert is employed by Newcastle University as Professor of Reproductive Medicine based at Newcastle Fertility and Wellcome Trust Centre for Mitochondrial Research.

Prof Sir Doug Turnbull: Doug Turnbull receives funding from the Wellcome Trust for research into mitochondrial donation techniques and other research into mitochondrial disease. His other research is supported by MRC, BBSRC and NHIR.

Prof Frances Flinter: No conflicts of interest

Prof Sir Mark Walport: None received

Prof Sir Robert Lechler: None received

Mr Robert Meadowcroft: “The Muscular Dystrophy Campaign does not have a financial interest in mitochondrial research.  We have been a long-term funder of research into mitochondrial disease, conducted by Professor Doug Turnbull and his team at Newcastle University.  Professor Turnbull was appointed a Vice President of the Muscular Dystrophy Campaign in 2013, an honorary position which carries no payment or reward.”

Dr Jeremy Farrar: None received

Dr Meenakshi Choudhary: None received

Dr Jane Stewart: None received

James Lawford Davies: None received

Prof Allan Pacey: “Chairman of the advisory committee of the UK National External Quality Assurance Schemes in Andrology, Editor in Chief of Human Fertility and Trustee of the Progress Educational Trust (all unpaid). Also, recent work for the World Health Organisation, British Broadcasting Corporation, Purple Orchid Pharma (paid consultancy with all monies going to University of Sheffield). Co-applicant on a research grant from the Medical Research Council (ref: MR/M010473/1).”