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“It took 20 years to realize the benefits of monoclonal antibody to treat human disease, and I believe that we are near a similar milestone in the field of gene therapy,” he adds
Years of animal tests show that the gene therapy is able to continually express the protein where it belongs in the brain for at least six years, according to one study at the University of California, San Francisco, Tuszynski says.
Both of Ceregene’s clinical trials of this drug were partly paid for by the Michael J. Fox Foundation, led by the actor who suffers from Parkinson’s. The company has also raised $69 million in venture capital through its history, from Alta Partners, MPM Capital, Investor Growth Capital, Hamilton Bioventures, California Technology Ventures, and South San Francisco-based Cell Genesys (NASDAQ: CEGE).
Ceregene’s first Parkinson’s trial, which looked at two different doses and involved 12 patients, found a 35 percent improvement in symptoms one year after the procedure, Tuszynski says. Nine of the 12 patients had what were considered clinically meaningful responses. Of those who responded, the average improvement in symptoms was 52 percent at the longest time of follow-up, which was two years for patients on a low dose, and 18 months on a high dose, Ceregene has said. By three months, researchers saw improvement, and that benefit was sustained, Tuszynski says. The higher dose was found to be safe and more effective, so that’s the one Ceregene chose to advance to the next stage.
The next trial, an even more meaningful event, is a Phase II study that will produce results by the end of the year. It enrolled 58 patients, with two-thirds of them getting the CERE-120, and one-third in a control group who got a sham surgery. An independent board of safety monitors hasn’t found any adverse events that would cause researchers to stop the trial, Tuszynski says. The big question will be how well it works after one year, compared with the sham procedure.
Whatever happens, the outcome will have significant ramifications for the field of gene therapy, and for Parkinson’s research. “People in the gene therapy field are well aware of this program. There’s also a buzz out there on Ceregene in the investment community,” Tuszynski says.
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