Mitochondrial donation: ‘Breakthroughs are possible when policy keeps pace with science’

‘The treatment was a success.’

For patients and families, these words mean everything. They are what hope is pinned on, in all reaches of healthcare.

Newcastle University and Newcastle Hospitals NHS Foundation Trust have shared such a success. Through pioneering science, they have given new hope to families affected by an inherited, otherwise incurable condition.

Eight babies – four boys and four girls – have now been born thanks to the innovative, licensed IVF technique, called pronuclear transfer, or mitochondrial donation. None show signs of having mitochondrial DNA disease.

This remarkable scientific achievement is a powerful demonstration of how long-term support for science and, especially for discovery research, can save lives. It highlights how, and why, the UK is a world leader in cutting-edge progress in life sciences.

Mitochondrial disease is a rare, complex condition, caused by faulty mitochondria. These tiny structures power our cells and are essential in giving in our bodies the energy we need. Around 1 in 5,000 babies are born with mutations that can cause mitochondrial disease. The disease – a genetic condition passed from mother to child – can result in many serious health conditions.

For women at risk of passing on this condition, there is now the chance to have children without this disease.

For the Newcastle team who have pioneered this treatment, it is a momentous achievement.

Success has been reached neither as a result of serendipity nor individual brilliance. As with all science at its best, progress came through rigorous research and close collaboration throughout between researchers, funders, regulators, and, critically, families affected.

The progress represents an almost lifetime’s work for many of the committed team – including authors of the latest research, Prof Doug Turnbull, Prof Mary Herbert, Dr Louise Hyslop and Prof Bobby McFarland.

In 2015, the UK became the first country to legally approve mitochondrial donation. Two years later, the Newcastle research team were given the first licence to carry out the treatment. And in 2018, the Human Fertility and Embryology Authority (HFEA) – the regulatory body – granted the first patient licences. The treatment is offered as part of a range of options, with mothers and babies followed closely through early years as part of the study.

Wellcome is proud to have been among the supporters, funding this pioneering research from its earliest stages to the clinical trial, advocating for the vital legislative change, encouraging ethical debate.

This success story has all the hallmarks of science to achieve potential in creating healthier futures.

It highlights the potential of integrating research in the NHS – a core ambition of the recently launched 10-year plan.

It reinforces the value of rigorous research – research which, correctly, comes with challenging questions for science.

It shows why as science advances we need regulators, policymakers, decisionmakers, working together to keep pace. Taking a robust and responsive approach ensures research can thrive, while maintaining public trust. Breakthroughs are possible when policy keeps pace with science.

It shows why the UK has been able to lead the way for decades in this research and across life sciences. To stay in step with science, enabling further innovation and to maintain a world leading position in this area, the government should also press ahead with the overdue review of the Human Fertilisation and Embryology Act.

For Wellcome, this research also represents principles at the core of our mission. It is why we commit to research over the long-term. It is why we fund projects without knowing what the answer might be.

In 2015, when approval was first granted, we could not be certain that mitochondrial donation would lead to a viable option for families. By asking that ‘what if’ question, we now have an answer. An answer best expressed by those affected: “After years of uncertainty this treatment gave us hope – and then it gave us our baby. We look at them now, full of life and possibility, and we’re overwhelmed with gratitude. Science gave us a chance.”