Gene Therapy
This article was originally published in RAJ Devices
Executive Summary
US FDA halts gene therapy clinical trial after patient's death
US FDA halts gene therapy clinical trial after patient's death
The US Food and Drug Administration (FDA) has halted a gene therapy clinical trial of an arthritis treatment after a patient died following a severe adverse reaction1. Although the cause of the reaction is still unknown, the agency has placed a clinical hold on the trial. It is also reviewing all ongoing trials that use similar treatment genes.
The company conducting the trial, Seattle-based Targeted Genetics, notified the FDA of the adverse event, and is co-operating fully with the agency. The FDA is co-ordinating with the National Institutes of Health (NIH) to better understand the potential scientific and safety implications of the event, and the NIH will discuss the matter at the September 2007 meeting of its Recombinant DNA Advisory Committee.
This is another setback to the still experimental field of gene therapy. A teenage patient died in 1999 following a severe immune response to treatment, and a study in 2003 found that several children in a French gene therapy trial had developed cancer after being treated for a severe immunodeficiency disease2,3.
The trial that has been placed on hold - meaning the product may not be administered and new patients may not be enrolled - began in October 2005. Under the trial, 127 patients have received an initial dose of the drug or a placebo, and 55 have received two doses of the active drug4. Subjects will continue to be monitored.
The trial was designed to assess the safety and efficacy of tgAAC94, an investigational therapy injected directly into patients' joints for the treatment of inflammatory arthritis. The therapy uses an adeno-associated virus vector to deliver the gene encoding for a soluble form of the tumour necrosis factor-alpha (TNF-alpha) receptor, TNFR:Fc. The TNFR:Fc protein inhibits TNF-alpha, a key mediator of inflammation.
References
1. FDA press release, 26 July 2007, www.fda.gov/bbs/topics/NEWS/2007/NEW01672.html
2. The Regulatory Affairs Journal - Pharma, 2005, 16(2), 81
3. Gene Therapy FAQ, The Human Genome Project, accessed 1 August 2007, www.ornl.gov/sci/techresources/Human_Genome/medicine/genetherapy.shtml
4. Targeted Genetics press release, 26 July 2007, http://ir.targen.com/phoenix.zhtml?c=84981&p=irol-newsArticle&ID=1032343&highlight=