Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

First trial in patients with a potential treatment of the incurable ALS muscle disease

01.12.2008
Project of VIB, UZ Leuven and NeuroNova

Permission has been granted to start the first safety and tolerability trial on patients for a remedy for ALS. ALS is an incurable, paralyzing neurodegenerative disorder that strikes 5 persons in every 100,000.

The disease commonly affects healthy people in the most active period of their lives - without warning. Researchers from VIB at the K.U.Leuven have previously shown the possibilities for the use of VEGF in the treatment of ALS through work in animal models. The Swedish Biopharmaceutical company NeuroNova has already built upon this research. Together with UZ Leuven they’ll start the first evaluation of safety and tolerability of the drug in patients by the end of this year. This is an important step in the development of a new treatment. It will take several years before the protein can be made available as a medicine.

An incurable disease of the muscles
Amyotrophic Lateral Sclerosis (ALS) can strike anyone. The Chinese leader Mao Tse Tung, Russian composer Dimitri Shostakovich, the legendary New York Yankee baseball player Lou Gehrig, and astro-physicist Stephen Hawking have all been afflicted with ALS. About half of the patients dies within three years - some even in the first year - usually as a consequence of suffocation.

In ALS, the patient’s nerve bundles that extend to the muscles deteriorate. As a result the patient loses control of the muscles, and progressively becomes paralyzed. The originating mechanism of this deadly disease of deterioration - which has an enormous medical and social impact - remains obscure. At present, the disease is totally untreatable.

VEGF: a promising candidate drug
VEGF is a substance that controls the growth of blood vessels. Unexpectedly, VEGF also helps neurons survive under stressful conditions. In 2001 Peter Carmeliet’s team showed that too little VEGF causes ALS-like symptoms in mice. Later the group of Diether Lambrechts, Wim Robberecht and Peter Carmeliet showed that persons who produce too little VEGF - due to certain variations in the gene that codes for VEGF - have a higher risk of developing ALS. This was the starting point of a search for a possible treatment with the VEGF protein.

Testing the treatment on rats with a severe form of ALS and on rats with a milder form, the researchers found that, in both groups, the VEGF-treated rats manifested the disease later than the untreated animals, and they lived considerably longer.

Using a pump
The researchers also investigated what the optimal technique would be for administering VEGF. An ordinary injection proved to be ineffective. But continuous administration of the VEGF protein directly into the cerebrospinal fluid (the fluid that circulates around the brain and the spinal cord) was quite effective. This was possible by means of a small pump that continuously pumps the VEGF protein in the brain. Furthermore, this technique permits a patient-oriented approach by enabling the administered dose of the VEGF protein to be easily controlled.
A story with several players
These encouraging and promising results were only the first steps on the way to a new remedy. Anders Haegerstrand and his team of the Swedish company NeuroNova have taken the development of the treatment further. After additional studies this research has reached the stage of starting the first trial in patients.

Wim Robberecht (UZ Leuven) and Markus Jerling (NeuroNova) will co-ordinate this first trial which is intended to evaluate the safety and tolerability of the drug and the infusion system. It is planned to start at the end of this year, and the investigator Dr Robberecht is currently looking for patients who are eligible for participation. These regulated studies on ALS patients will have to demonstrate the safety of the VEGF administration, and in a later stage the efficacy of VEGF as ALS therapy, before the protein can be made available as a medicine. Such procedures can easily last several years.

Sooike Stoops | alfa
Further information:
http://www.vib.be
http://www.neuronova.com

More articles from Health and Medicine:

nachricht Penn vet research identifies new target for taming Ebola
12.01.2017 | University of Pennsylvania

nachricht The strange double life of Dab2
10.01.2017 | University of Miami Miller School of Medicine

All articles from Health and Medicine >>>

The most recent press releases about innovation >>>

Die letzten 5 Focus-News des innovations-reports im Überblick:

Im Focus: Designing Architecture with Solar Building Envelopes

Among the general public, solar thermal energy is currently associated with dark blue, rectangular collectors on building roofs. Technologies are needed for aesthetically high quality architecture which offer the architect more room for manoeuvre when it comes to low- and plus-energy buildings. With the “ArKol” project, researchers at Fraunhofer ISE together with partners are currently developing two façade collectors for solar thermal energy generation, which permit a high degree of design flexibility: a strip collector for opaque façade sections and a solar thermal blind for transparent sections. The current state of the two developments will be presented at the BAU 2017 trade fair.

As part of the “ArKol – development of architecturally highly integrated façade collectors with heat pipes” project, Fraunhofer ISE together with its partners...

Im Focus: How to inflate a hardened concrete shell with a weight of 80 t

At TU Wien, an alternative for resource intensive formwork for the construction of concrete domes was developed. It is now used in a test dome for the Austrian Federal Railways Infrastructure (ÖBB Infrastruktur).

Concrete shells are efficient structures, but not very resource efficient. The formwork for the construction of concrete domes alone requires a high amount of...

Im Focus: Bacterial Pac Man molecule snaps at sugar

Many pathogens use certain sugar compounds from their host to help conceal themselves against the immune system. Scientists at the University of Bonn have now, in cooperation with researchers at the University of York in the United Kingdom, analyzed the dynamics of a bacterial molecule that is involved in this process. They demonstrate that the protein grabs onto the sugar molecule with a Pac Man-like chewing motion and holds it until it can be used. Their results could help design therapeutics that could make the protein poorer at grabbing and holding and hence compromise the pathogen in the host. The study has now been published in “Biophysical Journal”.

The cells of the mouth, nose and intestinal mucosa produce large quantities of a chemical called sialic acid. Many bacteria possess a special transport system...

Im Focus: Newly proposed reference datasets improve weather satellite data quality

UMD, NOAA collaboration demonstrates suitability of in-orbit datasets for weather satellite calibration

"Traffic and weather, together on the hour!" blasts your local radio station, while your smartphone knows the weather halfway across the world. A network of...

Im Focus: Repairing defects in fiber-reinforced plastics more efficiently

Fiber-reinforced plastics (FRP) are frequently used in the aeronautic and automobile industry. However, the repair of workpieces made of these composite materials is often less profitable than exchanging the part. In order to increase the lifetime of FRP parts and to make them more eco-efficient, the Laser Zentrum Hannover e.V. (LZH) and the Apodius GmbH want to combine a new measuring device for fiber layer orientation with an innovative laser-based repair process.

Defects in FRP pieces may be production or operation-related. Whether or not repair is cost-effective depends on the geometry of the defective area, the tools...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

Event News

12V, 48V, high-voltage – trends in E/E automotive architecture

10.01.2017 | Event News

2nd Conference on Non-Textual Information on 10 and 11 May 2017 in Hannover

09.01.2017 | Event News

Nothing will happen without batteries making it happen!

05.01.2017 | Event News

 
Latest News

Multiregional brain on a chip

16.01.2017 | Power and Electrical Engineering

New technology enables 5-D imaging in live animals, humans

16.01.2017 | Information Technology

Researchers develop environmentally friendly soy air filter

16.01.2017 | Power and Electrical Engineering

VideoLinks
B2B-VideoLinks
More VideoLinks >>>