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

September 18, 2012

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: NIH grant for the move toward clinical trials targeting the lysosomal storage disease MPSIIIB

Related Stories

NIH grant for the move toward clinical trials targeting the lysosomal storage disease MPSIIIB

May 25, 2011
Investigators at Nationwide Children's have received a grant from the National Institutes of Health (NIH) to help move a therapy for MPS IIIB that has been shown effective in mice toward clinical trials in humans.

Recommended for you

Novel approach to track HIV infection

August 18, 2017
Northwestern Medicine scientists have developed a novel method of tracking HIV infection, allowing the behavior of individual virions—infectious particles—to be connected to infectivity.

Faulty gene linked to obesity in adults

August 18, 2017
Groundbreaking new research linking obesity and metabolic dysfunction to a problem in the energy generators in cells has been published by researchers from the Harry Perkins Institute of Medical Research and The University ...

Two lung diseases killed 3.6 million in 2015: study

August 17, 2017
The two most common chronic lung diseases claimed 3.6 million lives worldwide in 2015, according to a tally published Thursday in The Lancet Respiratory Medicine.

New test differentiates between Lyme disease, similar illness

August 16, 2017
Lyme disease is the most commonly reported vector-borne illness in the United States. But it can be confused with similar conditions, including Southern Tick-Associated Rash Illness. A team of researchers led by Colorado ...

Addressing superbug resistance with phage therapy

August 16, 2017
International research involving a Monash biologist shows that bacteriophage therapy – a process whereby bacterial viruses attack and destroy specific strains of bacteria - can be used successfully to treat systemic, multidrug ...

Can previous exposure to west Nile alter the course of Zika?

August 15, 2017
West Nile virus is no stranger to the U.S.-Mexico border; thousands of people in the region have contracted the mosquito-borne virus in the past. But could this previous exposure affect how intensely Zika sickens someone ...

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.