Submission to ERMA supporting AgResearch's Transgenic application

NZORD Submission to ERMA in support of AgResearch's Transgenic Cows application - May 2002

1. NZORD works to support information, clinical care and research into rare disorders.

2. Knowledge of the genetic causes of many rare diseases, and the disease process that follows from the incorrect protein expression, clearly demonstrates that one of the best potential options for effective treatment or cure of many rare disorders, is to reintroduce the enzyme or protein that is missing, or is produced by the body in reduced quantity.

3. Production of the complex human protein in a form that will be safe and effective as a therapy, will often require that the protein is derived from a mammalian system, to ensure the correct post-translational modifications. This is in order to provide the required levels of glycosylation, phosphorylation and other characteristics that will enable recognition and uptake of the protein by the patient's cells, and minimise the chances of rejection by the patient's immune system.

4. Other methods of production of such proteins, where this can be done, often fail to produce proteins of the required complexity, or alternatively, in order to achieve adequate characteristics, may  involve complex laboratory and industrial scale production process that ferment animal cell lines. Such processes are expensive, environmentally stressful, and do not always produce the ideal functioning protein.

5. Evidence shows that human proteins produced by transgenic animals can be effective in treating the disease process in one rare disorder, Pompe disease. This evidence demonstrates that the proposal to produce a variety of functional therapeutic proteins from transgenic animals, will offer great potential for advancement in the control and treatment of rare diseases in humans.

6. However there is a need to produce the protein in sufficient quantities to ensure supply, as well as with the required characteristics. A transgenic cow would be the best animal model to use that would offer quantities of milk to ensure good supply, and the production in the cow's mammary gland suggests a likely high correlation with the characteristics needed for efficient uptake by the patient's cells.

7. For these reasons we offer our strong support for the conceptual and methodological approach adopted by AgResearch in their application.

8. We also note and commend to ERMA the thorough approach taken in the application to all other relevant aspects such as consultation and ethical considerations, and the intention to undertake valuable associated research on such matters as the location of the introduced gene in the animal's genome.

9. In relation to the requirements of the Act, with the passing of the HSNO (GMO) Amendment Act, for ERMA to consider alternative methods of production, we note and support the information supplied by AgResearch in appendix 11 about the limitations of other production methods. We also refer to frequent media comments by the Green Party claiming that transgenic animals are unnecessary because of alternatives such as synthesis from micro-organisms, or cell culture. These claims by the Green Party have been specifically rebutted and discredited before the Royal Commission on Genetic Modification. The Royal Commission accepted the evidence of Lysosomal Diseases New Zealand that transgenic animals are more likely to produce medically useful proteins. (RCGM report Chapter 7, para 103, page 161). We urge ERMA to carefully note the evidence on these matters and to reject the claims of the Green Party and similar groups. Their continued public claims on these matters are ill-informed, at best, and deliberate and mischievous misinformation, at worst.

10. Other criticisms by the Green Party of AgResearch's transgenic programme have focused on their alleged lack of experience in medical matters. NZORD urges ERMA to note the well established collaborations between veterinary and human health research, the significant body of knowledge gained from animal research to the benefit of human health, and the existence of many animal models of human diseases (including several here in NZ) that are vital to understanding the human condition. The knowledge gain from AgResearch's proposal will be to a common body of biotechnology, from which human health frequently draws.ERMA should note that on this subject it is the Greens who lack sound knowledge of the issues, and their claims and criticisms should be rejected.

11. We particularly support that part of the application that would allow AgResearch to apply the process of transgenesis to a range of target functional proteins over time. The importance of this is that there are many complex proteins being identified and characterised, and the genetic constructs developed for the expression of these proteins. Efficient use of this knowledge and rapid human health gains, would be severely compromised if separate individual applications had to be made for every protein considered by AgResearch for development in its transgenic cows programme.

12. Finally, we submit that there are no human health risks or environmental risks of any significance, posed by this proposed development, and that the risk management process undertaken by AgResearch is more than adequate to deal with any risks, whether real or imagined.

John Forman
Executive Director