In lab, drug-on-the-cob fights rare disease

Biologists in Canada have made a medical enzyme using genetically-engineered corn, a feat that could one day slash the cost of treating a life-threatening inherited disease, a journal reported on Tuesday.

Inserting a section of into maize seed caused them to make alpha-L-iduronidase in the endosperm, a nutritive tissue in the .

Alpha-L-iduronidase breaks down and is deficient in people with 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.

Until now, the therapy has been produced by coaxing cultures of cells taken from the ovaries of Chinese hamsters, and is hugely expensive.

The existing drug for MPS 1, laronidase (marketed as Aldurazyme) costs around $300,000 annually for children and $1 million for adults.

The research, led by Allison Kermode at Simon Fraser University in Burnaby, British Columbia, is published in the journal Nature Communications.

The results amount to "" for making the enzyme in laboratory conditions, the team say.

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 website Genetics Home Reference, which is supported by the US .

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


Explore further

Research supports newborn screening and early treatment for rare genetic disorder, MPS I

Journal information: Nature Communications

(c) 2012 AFP

Citation: In lab, drug-on-the-cob fights rare disease (2012, September 18) retrieved 11 August 2020 from https://medicalxpress.com/news/2012-09-lab-drug-on-the-cob-rare-disease.html
This document is subject to copyright. Apart from any fair dealing for the purpose of private study or research, no part may be reproduced without the written permission. The content is provided for information purposes only.
 shares

Feedback to editors

User comments