Gene Therapy in the United States: A Five-Year Status Report

作者：Gail Ross, Robert P. Erickson, Debra Knorr, Arno G. Motulsky, Robertson Parkman, Jude Samulski, Stephen E. Straus, Brian R. Smith · 发表于：Human Gene Therapy · 年份：1996 · DOI：10.1089/hum.1996.7.14-1781 · 被引用次数：96 · 研究领域：Virus-based gene therapy research、CRISPR and Genetic Engineering、Viral gastroenteritis research and epidemiology

At the time of the report, 94 of the 106 protocols receiving favorable RAC review had been approved by the NIH Director, with 12 awaiting NIH approval, pending submission of further information requested by the RAC. One protocol favorably reviewed by the RAC was disapproved by the NIH Director, and one single patient protocol, not recommended for approval, was ultimately permitted to proceed by the Director. Seventy-eight of the protocols were also approved by the FDA. Nineteen studies were closed because they had completed recruitment of patients; three studies were approved by the NIH but were never initiated; 55 were ongoing; and 14 were approved by the NIH and FDA but had not yet enrolled patients. As of June, 1995, 597 subjects in the United States had received experimental genetic material. This represents over 85% of the total patients worldwide who have received gene therapy (Marcel et al., 1995). Figure 1 illustrates the rate of increase in the cumulative number of protocols approved over the past 5 years. Although gene transfer studies were initiated in 1989, it was not until 1991 that there were multiple new projects approved in a single year. The total number of patients who underwent gene transfer is not evenly distributed among protocols. Two protocols (both involving HIV vaccination and both sponsored by Viagene, Inc.) account for 168 of the patients entered whereas all other protocols had entered 45 or fewer patients each. About 35, or 6%, of the patients have...