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Implications of Genetic Modification for the health of children: Article for "Children" magazine - June 2001

The New Zealand Organisation for Rare Disorders is a new umbrella group set up to represent the interests of support groups and individuals affected by rare conditions. NZORD believes that new genetic knowledge and technologies such as genetic modification offer great opportunities to improve the health and well being of our nation's children. Promoting research efforts to improve the treatment of rare disorders, and to work towards cures, are key goals of NZORD, along with the need to improve screening and diagnosis, upgrade the clinical expertise available to treat rare conditions, and supporting the support groups with more efficient information and communications.

Looking back at the history of health improvement, it is readily apparent that for thousands of years the human race was largely at the mercy of nature as it subjected many of us to diseases and genetic conditions that led to early death, or a lifetime of poor health, or physical or intellectual disability. Only in the last few hundred years has science been able to make really substantial inroads into nature's hold over us. We are now able to prevent or control diseases such as smallpox and polio, and even simple bacterial infections that claimed so many lives. As with most diseases, it was babies and children who were most vulnerable, and who are now the main beneficiaries of the advances made.

We learned too that some conditions that cause severe health problems or intellectual disability, such as PKU and hypothyroidism, could be detected at birth and effective measures put in place to prevent the onset of physical and intellectual disability. For these two conditions alone, the tests and treatments devised by scientists have meant that for many, many decades about 22 NZ babies every year have been able to achieve a much better health status and avoid the intellectual disability and other health problems that were otherwise inevitable. That is more than 220 NZ babies each decade, or 1100 Nzers and potentially 1,500,000 worldwide in the last 50 years, for those two conditions alone. Science has produced some truly wonderful results for our children, yet we often take this progress for granted.

But despite the rapid advances of the recent past, we still have a high mortality rate from genetic conditions. 28% of all deaths in babies under 12 months are caused by genetic conditions, about the same level as cot deaths and perinatal conditions.

Till now it has been too difficult to contemplate the idea of treatment or cure for many thousands of complex genetic conditions that have caused premature death or poor health and disabilities among children. The reason? The causes of the diseases were not fully understood, and methods of treatment too complex to develop or implement. Science was frustrated by the limits of its knowledge.

Recent advances in understanding the genetic origin of many rare conditions, has opened up the possibility that rare and very severe conditions like Rett syndrome, Hunter disease, Sanfillipo disease, Tay-Sachs and Batten disease, may become controllable or treatable. These early onset diseases mean that most affected children would not survive into their teens, yet it is now conceivable that they might be controllable, and perhaps even cureable in the future. The same possibility arises for many other diseases as well. Science can now lift its sights from just 500 diseases, mainly bacterial, metabolic and viral, that it can currently aim to treat, to a potential list of 5000 or more, mainly genetic diseases.

This hope stems from understanding the genetic defects that are the root cause of most rare diseases, the enzymes that are absent or deficient as a result, and the biological and biochemical effects this has, often on the brain and central nervous system as well as other organs. This recent understanding is as significant as the past discoveries of bacteria or micro-organisms causing infections, that allowed control mechanisms of hygiene and antibiotics to be conceptualised then implemented.

Already this genetic understanding, along with new technologies, especially genetic modification, is translating this hope into reality. We see practical application of this in some common conditions where GM hepatitis vaccine is preventing liver cancer, and GM insulin is producing a safer product to greater numbers of people with diabetes. And many hundreds of genetically modified medicines are at advanced stages of development for both rare and common conditions. Yet even as the possibilities are realised, there are debates about the appropriateness of genetic modification, and strong calls for its restriction or prohibition.

Most concerns about genetic modification can be traced to fear and uncertainty about all things new. Remember dire warnings about microwave ovens and pasteurised milk? In the current debate there is much emotion raised about genetic modification of food, despite evidence of benefits, and a lack of evidence of harm. All technologies have theoretical or actual risks. The appropriate response is to manage the risk, not to ban the technology.

If popular concerns about food modification do lead to any restriction on GM technology, it is inevitable that this will have negative impacts on the medical research we depend on to improve the health and quality of life of our sons and daughters. But more seriously, it will reduce our future ability to unlock the potential of so many children whose physical health or intellectual functioning is clouded through these genetic accidents. Instead of a lifetime of poor health and intellectual disability, GM will offer more of them a life with less disability, or even a normal life instead.

The possibility of being able to realise the full potential of each individual, is the motivation for many parents to support the maximum use of GM technology. A number of us representing our children affected by Rett, Fragile X, Batten, Mannosidosis and ML3 disorders, made a plea to the Royal Commission on GM to ensure the potential benefits of this technology are not restricted. We want and we expect the best for our children. We were also deeply impressed by the similar motivation of the scientists we have met, to stop unnecessary suffering, disability and stress, and to improve health and quality of life.

In the short term, there is unlikely to be full agreement in society about new and controversial technologies. There are also important ethical issues to be determined, rights to be protected, and appropriate safeguards to be applied to the research. But making a decision to restrict genetic modification would be a disaster for future generations. Without it, too many babies will continue to die in infancy, lots of young children will not reach their teens, and disabling conditions will blight the lives and restrict the potential of so many more. We must embrace Genetic Modification with enthusiasm, along with all innovative scientific advances which offer similar hope, and ensure every opportunity is taken to advance and improve the future health and wellbeing, physical and intellectual, of our nation's children.