The drug treatments for the rare lysosomal storage disease are incredibly expensive — we're talking hundreds of thousands of dollars a year for enzyme replacement therapy. But a new breakthrough technique could massively reduce that, by producing the drug in corn.
Currently, if you suffer from mucopolysaccharidosis I, getting the replacement alpha-L-iduronidase enzyme you need is $300,000-$500,000 a year for a child, more for an adult, because each dose has to be made in a mammalian cell culture in order to make the enzyme function. A newly described method could slash that cost thanks to finding a way to create the protein in maize seeds.
It's a long way from getting your daily drug dose as corn on the cob, but what it is is a massively more affordable method of producing what's now an extremely expensive drug for a very rare disease. By tweaking the maize, it was able to function as a home for the enzyme, and still work in humans. Hopefully, this technique could be adapted for other illnesses too, and save people from being faced with a $1 million pricetag for their treatment.
Photo by jster91.