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The source of this article is the Contra Costa Times: http://tinyurl.com/6qyyt Posted on Thu, Apr. 07, 2005 Parkinson's treatment left in limbo By Judy Silber CONTRA COSTA TIMES A shift in strategy by biotech company Avigen Inc. puts in jeopardy a preliminary but potentially promising clinical trial for Parkinson's disease. The Alameda-based company announced this week that it is abandoning its roots in gene therapy to develop more traditional pharmaceuticals, specifically therapies for neuropathic pain, the chronic discomfort sometimes caused by nerve damage. The change leaves in limbo a clinical trial for Parkinson's disease that started at UC San Francisco in December. Avigen will no longer fund the program, but says it is looking for alternate sources of money. The field of gene therapy, treatment to replace missing or defective genes with a modified virus, has had a bumpy road. Avigen's involvement is no exception. The company canceled a trial for hemophilia last year after five years of fits and starts. And it took many meetings and nearly a year of consideration by the FDA before Avigen received approval to start the Parkinson's trial. Despite 15 years of research, the U.S. Food and Drug Administration has yet to allow a single gene therapy product on the market. Even so, the Parkinson's trial was considered promising because of positive animal results showing that it might restore the effectiveness of a medication called L-Dopa. That would be helpful. L-Dopa initially acts as a miracle drug for many patients, but its effectiveness decreases within a few years and it also often causes discomfiting involuntary body movements. "I'm concerned about the program and the continuity of it," said Krys Bankiewicz, a professor of neurological surgery at UC San Francisco. It was Bankiewicz's laboratory research that laid the foundation for doing the trial in people, a process that took many years. Yet Avigen did not inform the scientist until 30 minutes before it issued its press release that it was withdrawing funding, and delivered the notification by e-mail. Bankiewicz expressed dismay and bafflement over Avigen's timing. The first patient in the trial experienced no complications, he said. It also appeared that the FDA might loosen restrictions and allow treatment of more patients in a shorter period of time. So why, just when the trial is getting off the ground, is the company throwing its future into doubt Bankiewicz asked. "Is it a responsible thing that they're doing? I don't think so," Bankiewicz said. "It really provides a great concern to patients, recruitment and the image of the trial. It's a big disappointment." The trial will most likely stop unless a sure source of funding steps forward. A few companies called Bankiewicz on Tuesday, inquiring about the trial and its status, but it's not clear whether any will buy the rights to pursue it. In a press release, Avigen said it has secured one funding source for the Parkinson's trial, but did not specify details. Avigen also said is examining three options to preserve the trial. One is to spin off the program into an independently backed company. The second is to combine assets with another gene therapy company. The third is to continue running the trial through universities, but end all of Avigen's efforts. Avigen executives could not be reached for further comment. Avigen has focused on gene therapy ever since its inception in 1992, but progress has been slow for both the company and the field. During a five-year period in its work on hemophilia, the company never moved beyond a phase I clinical trial, the most preliminary testing done in humans. Before this week, the latest setback for gene therapy occurred in March when Seattle-based Targeted Genetics announced that its clinical trial for cystic fibrosis had failed to help patients breathe better. The FDA also halted gene therapy trials in January for a rare and potentially fatal genetic disorder called severe combined immunodeficiency disease, after French scientists reported that three children developed cancer. In its press release, Avigen said its decision will curb its burn rate by more than 40 percent, from $23 million in 2004 to approximately $13 million. Avigen stock rose by 1.4 percent to $2.85 on Tuesday after the announcement and another 4.9 percent to $2.99 on Wednesday. ---------------------------------------------------------------------------- ---- Judy Silber covers biotechnology and the business of health care. Reach her at 925-977-8507 or [log in to unmask] ---------------------------------------------------------------------- To sign-off Parkinsn send a message to: mailto:[log in to unmask] In the body of the message put: signoff parkinsn