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A VEGF STORY: Why The Packard Center recruited Peter Carmeliet as its latest Scientific Advisor Several years ago, scientists in the ALS community were Normally, the gene, called VEGF, gives cells a blueprint for what’s called vascular endothelial growth factor, a protein that sparks rapid growth of new blood vessels in oxygen-deprived tissues. But alter the gene — a human one the lab animals were engineered to carry — and the animals’ muscles grew progressively weaker, much as they do in ALS. Spinal cord tissue was similarly injured. Following up on his study, Belgian researcher Peter Carmeliet, the lead scientist, teamed with colleague Wim Robberecht and others to examine VEGF genes in some 2,000 people in Belgium, Sweden and the U.K. They found three slightly different versions of the gene in that population. Two of the versions appeared to cause lowered levels of VEGF protein in the body. Then further analysis suggested — a surprising find — that low levels of VEGF correspond to a higher risk of developing ALS. More recently, and most important, Carmeliet and his team have shown VEGF is critical to the development of the nervous system. Also, they’ve shown the molecule plays an important part in nurturing and sustaining various nervous system cells. And finally, they have strong evidence that VEGF helps protect the nervous system against the sort of assault that’s typical of ALS, the so-called excitotoxic damage. Impressed by VEGF’s roles in healthy nervous systems, and by the apparent damage that can result when it’s in short supply, Carmeliet and his collaborators are exploring VEGF gene therapy for ALS. Preliminary animal studies are encouraging: given extra copies of the VEGF gene, SOD1 mice that model the disease have a greatly slowed onset of disease. Also, the animals’ life expectancy rose by 30 percent — a dramatic therapeutic effect when compared with other approaches. The work is continuing. The Packard Center is pleased to have Carmeliet as a new advisor. In that role, he’ll suggest new research avenues and evaluate the Center’s existing approaches. With the other advisors, he’ll also help monitor the quality of research by Center grantees. Already, Carmeliet has worked with Center scientist Alex Kolodkin and his research team, sharing expertise and lab materials. Kolodkin hopes to find the basis for VEGF’s protective effect. He believes it may be tied to an internal signalling system that maintains proper relationships between neurons and the muscles they stimulate. As an M.D., Ph.D. from Belgium, Peter Carmeliet is with the Center for Transgene Technology and Gene Therapy at Katholieke Universiteit, Leuven, Belgium. He was a research fellow at Harvard for a time and at MIT’s Whitehead Institute. His accomplishments have been recognized with many awards and honorary lectureships, including the prestigious Nobel Forum Lecture. Carmeliet holds more than a dozen patents and his work has been cited in nearly 10,000 scientific papers. His colleague, Wim Robberecht, has been a Packard Center advisor since it first opened. |
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intrigued by an unusual report from a university laboratory in Belgium.
It tied mutations in a gene well-known for triggering new blood vessel
growth to symptoms very much like ALS in the lab animals who carried it.
