Promising results in the battle against incurable ALS muscle disease

t the vascular endothelial growth factor (VEGF) plays a major role in this disease.

VEGF: a promising track to follow

VEGF is a signaling substance that controls the growth of blood vessels. To a large extent, a tissue in need of oxygen manufactures the protein, thereby developing new blood vessels so that the need for oxygen again diminishes. VEGF also helps neurons survive under stressful conditions. Last year, the work of Peter Carmeliet's team showed that persons who produce too little VEGF - due to certain variations in the gene that codes for VEGF - have a greater chance of developing ALS. Earlier this year, their research proved that gene therapy with the VEGF gene increased the life expectancy of ALS mice by 30%. But gene therapy is still a controversial method of treatment, whose path to the clinic can be quite long.

VEGF prolongs the life of ALS rats

So, investigation into a possible treatment using the VEGF protein has never been abandoned. Now, research from Erik Storkebaum and Diether Lambrechts, under Peter Carmeliet's direction, has shown the effectiveness of such a treatment on ALS rats. 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 contracted the disease later than the untreated animals, and they continued to live considerably longer.

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 - by means of a small pump - directly into the cerebrospinal fluid (the fluid that circulates around the brain and the spinal cord) was quite effective.

New hope for patients

Furthermore, this technique permits a patient-oriented approach by enabling the administered dose of the VEGF protein to be easily controlled. Te

Contact: Sooike Stoops
VIB, Flanders Interuniversity Institute of Biotechnology

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