A drug for a lysosomal storage disease, which could previously only be manufactured by expensive cell culture techniques, may now be produced by greenhouse-grown maize, according to research published in this week’s Nature Communications. The findings could dramatically decrease the cost of treating patients with a rare, but life-threatening inherited disease.
Allison Kermode and her team created transgenic maize seeds which can synthesize alpha-L-iduronidase, an enzyme used to treat mucopolysaccharidosis I. Sufferers of this disease endure progressive damage to the heart, brain, and other organs, which if left untreated, results in death in early childhood. Currently, the enzyme is extremely costly to produce, with treatments typically ranging from $300,000 to $500,000 per year for children and much higher for adults, since the enzyme can only be made by cultures of mammalian cells that are capable of performing the biochemical tweaks required to make the enzyme function. By finding a way to trick the maize into making certain modifications, they were able to produce the drug in maize seeds, highlighting a process that could ultimately be used in disease treatment.