Belgian researchers closing in on treatment for CMT

June 1, 2004

Brussels, 28 May 2004

Thanks to close collaboration between several Belgian universities and laboratories abroad, scientists are closer to a treatment for a little known hereditary disease of the nervous system, Charcot-Marie-Tooth (CMT) disorder.

Flemish researchers have discovered a tiny piece of the molecular puzzle which has brought science a step closer to developing new therapies for CMT, the most common hereditary illness of the peripheral nervous system which strikes one in every 2 500 people and leads to reduced function in the muscles, lower legs, feet and hands.

Scientists have searched for some time for the causes of neurodegenerative disorders, such as Lou Gehrig's disease and the lesser-known CMT. Two small proteins turn out to be crucial, say a team from the Flanders Interuniversity Institute for Biotechnology (VIB), and faults in the way the genes are coded lead to the disease's onset.

Today, there are no effective therapies to slow down or stop the onset and progress of CMT, only treatments aimed at supporting patients who have it. Severe cases, especially among older people, may even be confined to a wheelchair. While more research is needed to build a fuller picture of the diagnosis and potential treatments for the disease, the Belgian discovery is a good starting point.

Nerve centre

Researchers from the University of Antwerp's molecular genetics department recently found – in a number patients – that mutations in the genetic code of two 'heat shock' proteins (HSP) lead to an interaction that results in reduced neuronal viability. These findings, say the team, should pave the way to new therapies for not only CMT but also several other hereditary nervous disorders, including carpel tunnel syndrome.

The VIB is a joint research venture between four Flemish universities – Ghent University, the Catholic University of Leuven, the University of Antwerp and the Free University Brussels – and assembles the talents of 800 researchers from nine science departments into a single institute. The current research is a testament to the importance of scientific collaboration.

The Belgian findings come just one month after a team from Duke Centre for Human Genetics (US) reported, in the April issue of Nature Genetics, that defects in a gene critical to mitochondrial movement – known as mitofusin 2 – underlie CMT's symptoms. The current study appears in Nature Genetics May issue (Irobi et al and Evgrafov et al.) and, according to the VIB, can be found online from 2 May onwards.

DG Research
http://europa.eu.int/comm/dgs/research/i ndex_en.html
Item source: http://europa.eu.int/comm/research/headl ines/index_en.cfm

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