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Biotech / Medical : CLTX: Celsus Therapeutics -- Ignore unavailable to you. Want to Upgrade?

To: Miljenko Zuanic who wrote (22)	10/25/2001 9:11:17 PM
From: Miljenko Zuanic	Read Replies (1) \| Respond to of 40

<<I am still puzzled why is Street so slow to figure out...>> Maybe....??? FDA Urged to Collect More Long-Term Safety Data From Gene Therapy Trials -------------------------------------------------------------------------------- WASHINGTON (Reuters Health) Oct 24 - Expert advisors to the US Food and Drug Administration on Wednesday recommended that the agency require sponsors of gene therapy trials to track patients for at least 15 years, whether or not the therapy is delivered using a viral vector with the ability to replicate. At present, the FDA requires such long-term patient follow-up only for gene therapy that makes use of retroviral vectors. The committee's recommendation was meant to address the growing use of RNA- and DNA-based delivery mechanisms, for which there is no FDA policy. In making the recommendation, the Biological Response Modifiers Advisory Committee said that its primary concern was that all RNA- and DNA-based vectors could have long-term side effects. RNA- and DNA-based vectors have been shown to cause or be associated with malignancies such as leukemia, as well as liver failure, central nervous system disorders and autoimmune conditions such as multiple sclerosis. In some cases these conditions have developed months or years after initial administration of gene therapy. "The public, Congress expect it of us," said Dr. Daniel R. Salomon, the committee chair and an associate professor of Medicine at the Scripps Research Institute in La Jolla, California, while summing up the committee recommendation. "It will help move the field forward." Currently, an estimated 4,000 Americans are participating in about 100 trials of gene therapy, according to figures from the National Institutes of Health. But some investigators fear that in making the recommendation, the committee may have advised the agency to set too high a bar for sponsors in the field. While they support the need for long-term follow-up, these experts are concerned that the additional requirements will discourage patient participation and create a financial barrier that would be difficult for even the deep-pocketed sponsors of the experimental therapies to overcome. "It's a tough problem," Dr. Jolly Douglas, CEO of BioMedica Inc., a sponsor of gene therapy trials, told Reuters Health. Even in conventional clinical trials taking place over a span of several years, patients are often lost over time due to a move or other uncontrollable event, he noted. "It will take a lot of money to get done. It's a disincentive," he said. Dr. Douglas and other sponsors of gene therapy trials want the FDA to determine how much follow-up is needed and how that information is to be gathered on a case-by-case basis, which has been the agency's de facto policy. They also want either the FDA or another regulatory agency to collect the required long-term follow-up information in a central database to be managed by the government rather than by the sponsors. But even then, questions remain about how this information would be gathered. Committee members suggested that the task could be accomplished by sending a yearly postcard to patients. But the problem with that approach is that it would fail to account for any patients who died due to a side effect, the committee noted. Another proposed solution was to ask each patient's primary physician to note any possible long-term side effects. But that solution could discourage patients from participating because they would be required to allow the sponsor of the trial to question their doctor about their health history over almost two decades, the committee observed. The recommendations echoed suggestions made earlier this year by the same committee. At that April meeting, the panel suggested that the length of the follow-up period — which could potentially range from 5 years to about 20 years — should be tailored in part to the biological system that the gene therapy manipulates and to the risk that is likely to be associated with the treatment. For example, the committee suggested that a gene therapy that carries a risk of cancer should be monitored in patients for as long as 20 years, whereas a gene transfer product that could trigger an autoimmune response might need only a 5-year follow-up. While the committee's comments could lead to a broader general principle or policy, the details will need to be ironed out, Dr. Jay P. Siegel, director of the FDA's therapeutic research and review office, told Reuters Health. "What we are hearing is that there is no one-size-fits-all solution," he said.