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

May 27, 2004

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 endothelium growth factor (VEGF) plays a major role in this process.

VEGF is a signal molecule controlling the growth of blood vessels. Tissue in need of oxygen produces a large degree of this protein, stimulating new blood vessel growth, and so the deficiency of oxygen declines. VEGF also helps nerve cells to survive in stressful circumstances. Last year the work of Peter Carmeliet's team indicated that people who produce an insufficient amount of VEGF - because of certain variations in the gene coding for VEGF - have an increased risk of developing ALS.

Now, a year later, Peter Carmeliet and Oxford BioMedica, indicate that an injection of the VEGF gene in the muscles slows down the onset and progression of ALS in mice. A treatment that started after the first paralysis symptoms appeared - and thus after neurons died off - is also achieving positive results. This is important for the clinical application because ALS cannot be detected before its onset in over 90% of the cases.

VEGF treatment increases the life expectancy of the ALS mice by 30% and there are no toxic side effects. This makes the treatment one of the most promising current therapies. Gene therapy is currently controversial, yet its researchers are hopeful that the method developed by the British company to bring the VEGF gene into expression in neurons, will be safe and useful - and possibly indispensable to this incurable muscle disorder.

Although these results are hopeful and promising, there is still a long way to go before a new medicine is developed. Regulated studies in ALS patients will have to indicate the therapeutic effect of VEGF for ALS, before it can become an available medicine. These procedures can easily take ten years.
As this research can raise many questions for ALS patients, we request you to refer to VIB's special e-mail address in your report or article. Everyone with queries about this and other medical research can contact patienteninfo@vib.be

VIB - Flanders Interuniversity Institute for Biotechnology