Navigation Links
Growing corn to treat rare disease
Date:9/21/2012

The seeds of greenhouse-grown corn could hold the key to treating a rare, life-threatening childhood genetic disease, according to researchers from Simon Fraser University.

SFU biologist Allison Kermode and her team have been carrying out multidisciplinary research toward developing enzyme therapeutics for lysosomal storage diseases - rare, but devastating childhood genetic diseases for more than a decade.

In the most severe forms of these inherited diseases, untreated patients die in early childhood because of progressive damage to all organs of the body.

Currently, enzyme treatments are available for only six of the more than 70 diverse types of lysosomal storage diseases.

"In part because mammalian cell cultures have been the system of choice to produce these therapeutics, the enzymes are extremely costly to make, with treatments typically ranging from $300,000 to $500,000 per year for children, with even higher costs for adults," says Kermode, noting the strain on healthcare budgets in Canada and other countries is becoming an issue.

Greenhouse-grown maize may become a platform for making alpha-L-iduronidase, an enzyme used to treat the lysosomal storage disease known as mucopolysaccharidosis I, according to research published in this week's Nature Communications.

The findings could ultimately change how these enzyme therapeutics are made, and substantially reduce the costs of treating patients. The novel technology manipulates processes inside the maize seed that "traffick" messenger RNAs to certain parts of the cell as a means of controlling the subsequent sugar processing of the therapeutic protein.

In this way, the researchers have been able to produce the enzyme drug in maize seeds. The product could ultimately be used as a disease therapeutic, although it is still "early days," says Kermode, and several research goals remain to be accomplished before this can become a reality.

Kermode says the success of the work underscores the power of multidisciplinary research that included contributions from SFU chemistry professor David Vocadlo, and from UBC Medical Genetics professor Lorne Clarke. It further underscores the importance of connections between SFU and Australia's Griffith University, through collaborative researchers Mark von Itzstein and Thomas Haselhorst.

"In 2005, we had the basis of our story worked out," says Kermode. "Taking it to the next level involved their precise analyses to determine the sugar residues on the therapeutic enzyme produced by the modified maize seeds.

"When we first looked at the sugar analysis data we were amazed at how well the 'mRNA-trafficking strategy' had worked, and the high fidelity of the process for controlling the sugar-processing of the therapeutic protein. This is critical as sugar processing influences the characteristics of a protein (enzyme) therapeutic, including its safety, quality, half-life in the bloodstream, and efficacy. The work could well extend to forming a platform for the production of other protein therapeutics."

Kermode also credits SFU research associate Xu He, the first author of the Nature Communications paper. Her funding sources included NSERC Strategic grants and a Michael Smith Foundation for Health Research Senior Scholar Award, and in related research, a Canadian Society for Mucopolysaccharide and Related Diseases grant.


'/>"/>

Contact: Allison Kermode
kermode@sfu.ca
778-782-3982
Simon Fraser University
Source:Eurekalert

Related biology news :

1. Growing nitrous oxide levels explained
2. Growing risks from hatchery fish
3. What does a tree growing faster than its neighbor look like from outer space?
4. Learning from each other -- growing together
5. Ozone treated water v. lethal microbial material
6. Breakthroughs in Chikungunya research from A*STAR spell new hope for better treatment and protection
7. Collaborative preclinical efficacy studies suggest a new target for drug addiction treatment
8. Genetic research develops tools for studying diseases, improving regenerative treatment
9. Step forward in research into new treatments for brain edema
10. Nanotherapy: Treating deadly brain tumors by delivering big radiation with tiny tools
11. New hope for treating Alzheimers Disease: A role for the FKBP52 protein
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:5/12/2016)... 2016 WearablesResearch.com , a brand of ... results from the Q1 wave of its quarterly wearables ... consumers, receptivity to a program where they would receive ... insurance company. "We were surprised to see ... Michael LaColla , CEO of Troubadour Research, "primarily because ...
(Date:4/28/2016)... GOTHENBURG, Sweden , April 28, 2016 ... 1,491.2 M (139.9), up 966% compared with the first quarter of ... Operating profit totaled SEK 589.1 M (loss: 18.8) and the operating ... SEK 7.12 (loss: 0.32) Cash flow from operations was ... , The 2016 revenue guidance is unchanged, SEK 7,000-8,500 M. ...
(Date:4/15/2016)... 2016  A new partnership announced today will ... decisions in a fraction of the time it ... high-value life insurance policies to consumers without requiring ... Force Diagnostics, rapid testing (A1C, Cotinine and HIV) ... pressure, weight, pulse, BMI, and activity data) available ...
Breaking Biology News(10 mins):
(Date:6/23/2016)... ... , ... Supplyframe, the Industry Network for electronics hardware design ... Located in Pasadena, Calif., the Design Lab’s mission is to bring together inventors ... and brought to market. , The Design Lab is Supplyframe’s physical representation of ...
(Date:6/23/2016)... LOUISVILLE, Ky. , June 23, 2016 /PRNewswire/ ... from two Phase 1 clinical trials of its ... double-blind, placebo-controlled, single and multiple ascending dose studies ... and pharmacodynamics (PD) of subcutaneous injection in healthy ... APL-2 subcutaneously (SC) either as a single dose ...
(Date:6/23/2016)... 23, 2016 On Wednesday, June 22, ... down 0.22%; the Dow Jones Industrial Average edged 0.27% lower ... 2,085.45, down 0.17%. Stock-Callers.com has initiated coverage on the following ... Therapeutics (NASDAQ: NKTR ), Aralez Pharmaceuticals Inc. (NASDAQ: ... BIND ). Learn more about these stocks by accessing ...
(Date:6/23/2016)... ... June 23, 2016 , ... ... of intelligent tools designed, tuned and optimized exclusively for Okuma CNC machining centers ... The result of a collaboration among several companies with expertise in toolholding, cutting ...
Breaking Biology Technology: