Navigation Links
New technology for a more efficient treatment of Pompe disease and other metabolic disorders

VIB researchers from UGent and Vrije Universiteit Brussel , together with a team of the firm Oxyrane have developed a new technology that can lead to a more efficient and possibly also cheaper therapy for diseases such as Pompe disease. Oxyrane will now start developing a clinical program for this therapy in Pompe disease.

Nico Callewaert (VIB-UGent): "By combining natural processes and proteins of two microbes and a plant we have improved the technology to produce enzymes that patients with metabolic disorders can't produce themselves. We played with the sugar structures on the enzymes. As a result these enzymes were directed much more efficient to the locations in the cells where they actually have to work. It shows once again what medical biotechnology can accomplish."

Han Remaut (VIB-VUB): "The lacking piece of the jigsaw in this technology was a protein we discovered in a soil bacterium. Thanks to the detailed picture we obtained from the 3D structure of this novel protein, we now understand how it comes to its unique reaction specificity and how we could further improve it".

Wouter Vervecken (Oxyrane): "Data suggest that this platform has the potential to enable production of more efficacious therapies in the years to come. The results are very encouraging the enzyme for the treatment of Pompe disease is absorbed 17 times as efficiently by patient cells in vitro than current treatments".

Lysosomal storage diseases

1 in 5000 newborns has a lysosomal storage disease, but it can also cause symptoms later in life. In all the cells of our body you can find lysosomes. They are responsible for breaking down cell components to be reused by the cell. In lysosomal storage diseases, one or more of the enzymes that accomplish this process are lacking or insufficient. The result is that the non-processed substances accumulate in the lysosomes and eventually poison the cell, leading to organ damage that keeps on worsening if left untreated. One example is Pompe disease, which affects the muscles.

Treatments for these illnesses

Lysosomal storage diseases can be treated with the lacking enzymes, provided they end up in the patient's lysosomes; this is known as Enzyme Replacement Therapy (ERT). This can be accomplished by adding a specific modified sugar to the enzymes, i.e. mannose-6-phosphate, but this is not easy to do. Until now such modified enzymes could only be produced more or less successfully in mammalian cells. The VIB researchers, together with Oxyrane, have developed a novel, more efficient method that uses yeast cells instead.

A new technology for a more efficient production

The result is impressive the enzyme for the treatment of Pompe's disease is absorbed 17 times as efficiently by patient cells in vitro than the current therapeutic drug. In a Pompe disease mouse model the enzyme also worked better than the current therapeutic product. Based on these results Oxyrane will start clinical trials for Pompe disease, this means that it will be tested in humans for the first time.

Contact: Kris Van der Beken
VIB (the Flanders Institute for Biotechnology)

Related medicine news :

1. Mechanical tissue resuscitation technology shows promise
2. New study identifies how information technology is used to solve global health challenges
3. Power generation technology based on piezoelectric nanocomposite materials developed by KAIST
4. Simulation technology allows users to safely practice phacoemulsification cataract surgery
5. The Casey Group's Sales Navigator Wins 2012 Best Mobility App in the New Jersey Technology Council's Mobile Application Competition
6. Swanson Health Products Introduces a New Alkalizing Drink Mix, Swanson® pH Balance Alka-Tone with Alka-Plex™ Technology
7. Miami Children’s Hospital Performs First Nerve Repair Surgery Using Avance® Nerve Graft Technology
8. FDA Approves SonixGPS Needle Guidance Technology for Vascular Access Procedures
9. IVR Technology Group Named Platimum VAR by TSYS
10. 3M™ Gripping Material Technology – Giving Golfers a Competitive Edge
11. Newer technology to control blood sugar works better than conventional methods
Post Your Comments:
(Date:11/24/2015)... , ... November 24, 2015 , ... It takes only ... a business, it is critical that the first impression be positive and reflects business ... not likely to buy anything or want to return. They will also share their ...
(Date:11/24/2015)... ... 24, 2015 , ... United Benefit Advisors (UBA), the nation’s ... as its newest Partner Firm. Based in Jefferson City, Missouri, their core philosophy ... regardless of whether that client is a business, a family, or an individual. ...
(Date:11/24/2015)... , ... November 24, 2015 , ... Serenity Point Rehabilitation, ... series of recent video interviews with some of the staff members at their recovery ... residential treatment facility, as well as some of the things that make their recovery ...
(Date:11/24/2015)... ... 2015 , ... Abington Hospital – Jefferson Health is pleased ... Surgery Facility for treating individuals living with morbid or extreme obesity. , Aetna ... its members to help them make informed decisions about their healthcare needs. In ...
(Date:11/24/2015)... ... November 24, 2015 , ... American Specialty ... Program have announced their endorsement of the Medical Fitness Network (MFN), a ... proud to have the MFN as one of our endorsed organizations,” said ASH ...
Breaking Medicine News(10 mins):
(Date:11/24/2015)... Nov. 24, 2015 iRhythm Technologies, Inc. , a ... today announced that it will participate in the 27th Annual Piper ... New York, NY . Kevin King , ... December 1, 2015 at 8:50am ET. --> ... . --> . --> iRhythm ...
(Date:11/24/2015)... Ontario , Nov. 24, 2015 Teledyne ... digital X-Ray image sensing technology, will introduce its CMOS ... Technical Exhibition , November 29 to December 3, at McCormick ... of dynamic detectors for diagnostic and interventional imaging will be ... Xineos family of advanced CMOS X-Ray detectors is ...
(Date:11/24/2015)... The uptake of recently approved and pipeline premium products for ... growth to 2021, says GBI Research . ... Type 1 Diabetes Mellitus (T1DM), will be a key driver of market ... --> The uptake of recently approved and pipeline premium products ... market growth to 2021, says GBI Research . ...
Breaking Medicine Technology: