Global Health Asia-Pacific March 2020 | Page 55

their parents, they acquire mitochondria only from their mothers because the sperm mitochondria don’t make it into the female egg during fertilisation. As a result, the fertilised egg develops into an embryo carrying only its original female mitochondria. “A woman with mitochondrial disease can pass it on to her offspring, but it’s not a sure thing,” Dr Marni Falk, executive director of the mitochondrial medicine frontier program at Children’s Hospital of Philadelphia, told Global Health Asia-Pacific. “It depends on the percentage of mitochondrial DNA with the mutation.” This is because there are about 250,000 mitochondria in an egg cell, and each one of them stores hundreds of mitochondrial DNA copies. If a significant percentage of these copies are abnormally mutated, say 80 percent, then women are likely to have severe symptoms of mitochondrial disease and to pass the condition on to their babies. However, when the figure doesn’t exceed 10 percent, the disease is unlikely to be passed on. Some women with mitochondrial DNA mutations may never know they have them because they’re healthy and show no symptoms, but this doesn’t mean their kids are protected from transmission. While symptomatic women have up to a 50 percent chance of conceiving a child with mitochondrial disease, the likelihood is one in 25 for women without any symptoms. Although next-generation sequencing for genetic testing can work out the mutation level a woman carries, it’s still a matter of chance whether babies will be affected. “It’s like a jar of red and green jellybeans, with the red being the mutated genes and the green being the normal ones. If you put your hand in the jar, it’s impossible to predict with certainty how many reds you’ll pull out,” explained Dr Falk. “If there’s only one red among thousands, it’s very unlikely you’ll get it, but if there are 99 out of 100, you’re very likely to pull them out.” “They’re an important part of the human genome and are potentially involved not only in mitochondrial disease but also other conditions,” he said. In addition, the energy-producing structures have a special relationship with their corresponding nuclear genes. “A very important part of mitochondrial DNA regulation occurs at the hands of nuclear genes. Therefore, it’s crucial to keep intact this interaction between nuclear and mitochondrial genomes,” he added. One potential problem is that, if not all donated mitochondria can provide a suitable match for the mother’s nuclear genes, nuclear genome transfer could then lead to unknown complications. “A mismatch between nuclear and mitochondrial genes is a potential source of risk. We just don’t know to what degree,” said Dr Falk. According to Dr Keefe, some animal experiments suggest nuclear genome transfer could even lead to neuropsychiatric symptoms, like depression, and sterility. Potential risks must be considered in light of the availability of alternative ways to have an unaffected child. “There’s no strictly medical justification [to perform the procedure] as the main purpose is to have genetically-related babies,” pointed out Darnovsky. “Understandably, this is something important to some people, but it’s not a medical issue, it’s a social benefit. If you accept that understanding of the technique, you’ll want a very high level of assurance that you’re not putting babies at risk for future problems.” Efficacy, risks, and alternatives to nuclear genome transfer One way of viewing mitochondrial replacement therapy (MRT), another more common name for the controversial procedure, is to consider it as a slight upgrade to improve the body’s efficiency. This idea is often supported by the argument that the donated mitochondria make up just a tiny bit of the engineered embryo while their key function is simply to provide energy for biological processes. Supporters of MRT often downplay the significance of the cellular changes, comparing them to replacing the batteries in a camera, said Marcy Darnovsky, executive director of the not-for-profit Centre for Genetics and Society, noting that this was a big oversimplification. Dr Keefe agrees that characterising mitochondria as simple power plants is widely incorrect because they also play metabolic and immune functions. GlobalHealthAndTravel.com MARCH 2020 53