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Genzyme bets on gene therapy as others steer clearReuters Thu Jun 21, 2007 2:24PM EDT By Toni Clarke BOSTON, June 21 (Reuters) - Genzyme Corp. is expanding its research into gene therapy, a controversial and cutting-edge approach to treating disease which most companies avoid. The company, which built itself into one of the world's biggest biotechnology companies by developing drugs for rare genetic disorders, said on Thursday it has entered a partnership with Ceregene Inc. to develop a treatment for Parkinson's disease. Gene therapy is a controversial method of injecting genes into cells to make proteins that can fight disease, typically using a genetically engineered virus to carry the gene to the cell. So far, no gene therapy product has been approved, and research has shriveled following the death in 1999 of Jesse Gelsinger, an 18-year-old participant in a gene therapy clinical trial. "Gene therapy makes everyone nervous and there are only a limited number of players," said Dr. Harry Tracy, publisher of the monthly journal NeuroInvestment. "This is a daring area of research, and Ceregene's program is one of the most sophisticated out there." Genzyme began work on gene therapy in the early 1990s as a potential treatment for cystic fibrosis. That program didn't work, but the company continued its research and is now in mid-stage clinical trials with a treatment which stimulates blood vessel growth and could be valuable for patients with conditions characterized by poor circulation, such as peripheral artery disease. Ceregene's program, CERE-120, is composed of an adeno-associated virus vector carrying the gene for neurturin, a protein known to protect or repair nerve cells which secrete a brain chemical known as dopamine that is involved in movement. Parkinson's disease is caused by a reduction in dopamine-containing nerve cells. Gene therapy, in theory, represents an attractive technology for treating neurological disorders because the treatment circumvents the blood-brain barrier, a cell structure that protects the brain from foreign substances in the blood -- including large molecules such as therapeutic proteins. "Parkinson's is very attractive because you can deliver the gene to a localized area, the cells will make the protein and hopefully do the job," said David Meeker, head of Genzyme's rare genetic diseases unit. Meeker said its Parkinson's research is the cornerstone for what the company expects will be a growing central nervous system disorders program, including work on treatments for ALS, also known as Lou Gehrig's disease, and Huntington's disease, a condition in which the degeneration of brain cells causes uncontrolled movement and loss of intellectual function. "This is an area of high interest for us," Meeker said. Cambridge, Massachusetts-based Genzyme said it will pay Ceregene $25 million up front, make as much as $125 million in payments as the companies reach development milestones, and provide 50 percent reimbursement for late-stage development costs. Genzyme will gain marketing rights in all markets outside the United States and Canada. Ceregene, which was launched in January 2001 and is a former subsidiary of Cell Genesys Inc. , is currently conducting a Phase II, or mid-stage, clinical trial which is enrolling 51 patients. In an early-stage trial, CERE-120 showed a 36 percent reduction in Parkinson's symptoms at 12 months following administration. "This is early data which is very encouraging," Meeker said, "but it is too early to draw meaningful conclusions about the relative efficacy to other treatments." ((Reporting by Toni Clarke, editing by Gerald E. McCormick; Reuters Messaging, [log in to unmask], 617-367-4165)) Keywords: GENZYME/GENETHERAPY For more on this therapy, see PD Pipeline Database www.pdpipeline.org__._,_.___ __,_._,___ ---------------------------------------------------------------------- To sign-off Parkinsn send a message to: mailto:[log in to unmask] In the body of the message put: signoff parkinsn