Corn in fight against rare disease

Genetically engineered corn may hold the key to making the treatment of a life-threatening disease affordable.

An international research team led by scientists at Simon Fraser University and Griffith’s University’s Institute for Glycomics have inserted a section of DNA code into maize seed to produce alpha-L-iduronidase in the corn kernel.

Alpha-L-iduronidase breaks down sugar molecules and is deficient in people with the condition mucopolysaccheridosis I (MPS 1).

This is a so-called lysosomal storage disorder, in which sugary debris builds up in cells, damaging tissue in the heart, eyes, skeleton and brain. Without replacement enzymes, sufferers of MPS 1 often die in childhood.

The findings of this study have been reported in Nature Communications.

Professor Mark von Itzstein, Director of the Institute for Glycomics, said this multi-disciplinary study represents a significant breakthrough in the delivery of health care.

“My team at Griffith University’s Institute for Glycomics has been collaborating with Prof Kermode’s research team at Simon Fraser University to develop and characterize plant-produced humanized enzymes that may provide therapeutics for genetic-based diseases. This most recent advance is exciting and demonstrates the potential of the technology.” he said

The existing drug for MPS 1, laronidase (marketed as Aldurazyme) costs around $287,618 annually for children and $1 million for adults.

Further work would be needed to scale up volume, but this should not be too much of a problem and conventional techniques could be used, they add.

Severe MPS I occurs in approximately in one in 100,000 newborns, according to the MPS Australia website.

A milder form, called attenuated MPS I, occurs in about one in 500,000 births.