Global Health Asia-Pacific March 2020 | Page 59

individuals will be passed down to their future generations without their offspring’s consent. This is why the committee suggested women undergoing the procedure be implanted with only male embryos as the males born from them wouldn’t be able to transmit the donated mitochondria further on, preventing “potential adverse and uncertain consequences of MRT from being passed on to future generations.” The report concluded that “a slow, cautious approach to MRT is justified, with the requirement of deliberate and continued attention to ethical, social, and policy issues.” Despite its recommendations, MRT is banned in the US due to legislation prohibiting embryo manipulation in a way that leads to “heritable genetic modification.” Although similar bans are in place in many other countries, the UK was the first to legalise the procedure in 2015, within a strict regulatory framework that allows women carrying a high risk of passing down mitochondrial disease to undergo it at Newcastle Fertility Centre, the only institution with a licence to perform the procedure. Before the centre can treat any patients eligible for mitochondrial donation, it has to get permission from the UK Human Fertilisation & Embryology Authority, which grants it on a case-by-case basis. “Before couples are accepted for mitochondrial donation there is a rigorous process that includes counselling to ensure they are well prepared for it,” explained Curtis. Living with mitochondrial disease With nuclear genome transfer unable to treat mitochondrial conditions, affected patients have to rely on approaches that at best can manage their symptoms, but some of them are still at risk of an early death. This was the fate of Lily, Curtis’ third daughter, who was diagnosed with a mitochondrial condition at seven weeks and suffered repeated seizures as well as a cardiac arrest because of it. She eventually passed away in April 2007, at the age of eight months. “The experience was very isolating because there was little information available about the condition or support for families,” recalled Curtis. “It was like the worst nightmare in every respect as no one was able to give us any prognosis. You know that you are going to lose your child, but you don’t know when. So, we set the charity up to address these issues.” On its web page, the Lily Foundation provides a wealth of information on mitochondrial disease and has a medical board to give patients quality advice. It also helps families affected by the condition reach out to others who have gone through similar experiences. “We put families in contact with each other because mitochondrial disease can feel very isolating, and talking to someone who has experienced the same problems is a great help for GlobalHealthAndTravel.com There are still unknowns about the health of babies born through mitochondrial donation those affected,” she said. Since Curtis went through her ordeal, however, things have definitely improved for people with mitochondrial disease, at least in the UK. “It’s definitely changed for the better,” she stressed. “We now support over 750 families across the UK, and we work in collaboration with the NHS [National Health Service], which puts people diagnosed with mitochondrial disease in contact with us.” The foundation also puts a lot of money into medical research in the hopes of finding better diagnostics and, most importantly, a cure. Despite the bleak prognosis of some patients, many others can still have a good quality of life. “Getting care for mitochondrial disease can dramatically improve people’s well-being,” said Dr Falk. With enormous variability in terms of disease course, some patients start experiencing symptoms when they’re born, while others do so much later on, even when they reach their 60s. “There are people who only have heart problems or only have vision problems, and there are people who have multi-organ system dysfunction or severe neurologic problems,” said Dr Falk. “It’s hard to be specific, except that they’re all energy failure problems, and they all do improve with specific nutrition and exercise regimens. “And there are a lot of novel therapies emerging now that are based on preclinical studies on how to improve energy production and function when the genetic code of mitochondrial function is impaired. So, there’s a lot of room for hope.” n Despite the bleak prognosis of some patients, many others can still have a good quality of life MARCH 2020 57