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 Improving memory with magnets
28.03.2017 | McGill University

nachricht Graphene-based neural probes probe brain activity in high resolution
28.03.2017 | Graphene Flagship

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: A Challenging European Research Project to Develop New Tiny Microscopes

The Institute of Semiconductor Technology and the Institute of Physical and Theoretical Chemistry, both members of the Laboratory for Emerging Nanometrology (LENA), at Technische Universität Braunschweig are partners in a new European research project entitled ChipScope, which aims to develop a completely new and extremely small optical microscope capable of observing the interior of living cells in real time. A consortium of 7 partners from 5 countries will tackle this issue with very ambitious objectives during a four-year research program.

To demonstrate the usefulness of this new scientific tool, at the end of the project the developed chip-sized microscope will be used to observe in real-time...

Im Focus: Giant Magnetic Fields in the Universe

Astronomers from Bonn and Tautenburg in Thuringia (Germany) used the 100-m radio telescope at Effelsberg to observe several galaxy clusters. At the edges of these large accumulations of dark matter, stellar systems (galaxies), hot gas, and charged particles, they found magnetic fields that are exceptionally ordered over distances of many million light years. This makes them the most extended magnetic fields in the universe known so far.

The results will be published on March 22 in the journal „Astronomy & Astrophysics“.

Galaxy clusters are the largest gravitationally bound structures in the universe. With a typical extent of about 10 million light years, i.e. 100 times the...

Im Focus: Tracing down linear ubiquitination

Researchers at the Goethe University Frankfurt, together with partners from the University of Tübingen in Germany and Queen Mary University as well as Francis Crick Institute from London (UK) have developed a novel technology to decipher the secret ubiquitin code.

Ubiquitin is a small protein that can be linked to other cellular proteins, thereby controlling and modulating their functions. The attachment occurs in many...

Im Focus: Perovskite edges can be tuned for optoelectronic performance

Layered 2D material improves efficiency for solar cells and LEDs

In the eternal search for next generation high-efficiency solar cells and LEDs, scientists at Los Alamos National Laboratory and their partners are creating...

Im Focus: Polymer-coated silicon nanosheets as alternative to graphene: A perfect team for nanoelectronics

Silicon nanosheets are thin, two-dimensional layers with exceptional optoelectronic properties very similar to those of graphene. Albeit, the nanosheets are less stable. Now researchers at the Technical University of Munich (TUM) have, for the first time ever, produced a composite material combining silicon nanosheets and a polymer that is both UV-resistant and easy to process. This brings the scientists a significant step closer to industrial applications like flexible displays and photosensors.

Silicon nanosheets are thin, two-dimensional layers with exceptional optoelectronic properties very similar to those of graphene. Albeit, the nanosheets are...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

Event News

International Land Use Symposium ILUS 2017: Call for Abstracts and Registration open

20.03.2017 | Event News

CONNECT 2017: International congress on connective tissue

14.03.2017 | Event News

ICTM Conference: Turbine Construction between Big Data and Additive Manufacturing

07.03.2017 | Event News

 
Latest News

Transport of molecular motors into cilia

28.03.2017 | Life Sciences

A novel hybrid UAV that may change the way people operate drones

28.03.2017 | Information Technology

NASA spacecraft investigate clues in radiation belts

28.03.2017 | Physics and Astronomy

VideoLinks
B2B-VideoLinks
More VideoLinks >>>