Gene therapy with growth factor seems promising therapy for incurable muscle disorder ALS

ALS is an incurable paralysing muscle disorder affecting five in every one hundred thousand people. The disease mainly strikes healthy people in the most active period of their life, without any warning or family history. Researchers from VIB (the Flanders Interuniversity Institute for Biotechnology), lead by Prof. Peter Carmeliet (K.U.Leuven) already indicated the importance of the VEGF protein in this illness, on the basis of genetic studies. In cooperation with Oxford BioMedica, an Oxford-based biotech company, a new study of the VIB researchers indicates that gene therapy with VEGF appears to be one of the most promising therapies. By administering the gene that produces VEGF in the nerve trajectory of ALS mice, the researchers were able to slow down the development of the illness and increase their life expectancy by 30% - the largest therapeutic effect ever achieved for ALS.

ALS can affect anyone. Chinese leader Mao Tse Tung, Russian composer Dimitri Sjostakowitz, legendary Yankee baseball player Lou Gehrig and astrophysicist Stephen Hawkins were all affected by ALS. A large number of Italian top football players, pilots and soldiers in the Gulf War were also affected by this fatal disease. Around half of them die within three years, some even within a year, mostly in full possession of their faculties as a result of asphyxiation.

In patients with Amyotrophic Lateral Sclerosis (ALS) the nerves going to the muscles deteriorate, which is why the patient loses control of his muscles and is progressively paralysed, but remains disconcertingly in full possession of his faculties. The cause of this serious deterioration disease with an enormous clinico-social impact remains obscure. Up to now the disease was completely untreatable, because of which many ALS patients opt for euthanasia, a very controversial solution. Previously, genetic research by Peter Carmeliet and his team at K.U.Leuven has already led to the surprising insight that the vascular

Contact: Ann Van Gysel
VIB, Flanders Interuniversity Institute of Biotechnology

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