Replacing retroviruses

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http://www.biomedcentral.com/news/20030620/01

 

Replacing retroviruses

 

NIH advisory committee plans to explore alternative vectors for gene therapy |

By Hannah Kamenetsky

 

 

The National Institutes of Health Recombinant DNA Advisory Committee (RAC) will

consider a wide-ranging set of issues over the next year, including what gene

therapy vectors could replace retroviruses and what is required to win FDA

approval of new vectors so they can be used in clinical trials, members said at

a June 18 meeting in Bethesda, Maryland.

 

" We were looking in the broadest sense at the state of the art of retroviral

vectors, " said committee member Madison Powers, director of the Kennedy

Institute of Ethics at Georgetown University, in explaining the group's choice

of topics for future discussion. Another question that arises, Powers said, is

what safety modifications could be adopted for use of retroviral vectors in

clinical settings.

 

Although Powers didn't make the direct link in his comments, clinical trials

using retroviruses have attracted closer scrutiny since last fall, after

patients in French studies using retroviruses to insert new genes in blood stem

cells to treat X-linked severe combined immunodeficiency syndrome (X-SCID) were

found to have developed leukemia.

 

In February 2003, RAC recommended that retroviral gene transfer for X-SCID be

limited to patients for whom stem cell therapies had failed or for whom stem

cell donors couldn't be found. The committee did not go so far as to recommend

halting clinical trials using retroviruses to treat other diseases, including

non–X-linked SCID.

 

Looking ahead, Powers said, one large question is whether other vector types

would be appropriate substitutes for retroviruses, and if so, what is needed to

develop these new vectors in terms of clinical use, animal models, and Food and

Drug Administration approval. " I'd like to know what it takes from the beginning

to get a vector approved for use, " said Maxine Linial of the Fred Hutchinson

Cancer Research Center.

 

Another issue of interest is whether there are ways to make RAC's interaction

with institutional biosafety committees, institutional review boards, and

international oversight committees " more fluid and more useful, " Powers said.

 

Further topics that committee members want to put on their agenda include:

risk–benefit analyses of using retroviruses in clinical trials and whether there

are types of studies for which these vectors shouldn't be used, haploidentical

bone marrow transfer studies, large animal models, and statistical methodology

used in phase I clinical trials. The committee is scheduled to meet again

September 17–19, 2003.

Links for this article

National Institutes of Health Recombinant DNA Advisory Committee

http://www4.od.nih.gov/oba/rac/aboutrdagt.htm

 

B.A. Maher, " Gene therapy falters, " The Scientist, October 8, 2002.

http://www.biomedcentral.com/news/20021008/04/

 

B.A. Maher, " More gene therapy trials halted, " The Scientist, January 16, 2003.

http://www.biomedcentral.com/news/20030116/06/

 

A.P. Patterson, " Outcome of the RAC meeting and updated advice to investigators

regarding adverse events in the X-SCID gene transfer trial, " Letter to Principal

http://www4.od.nih.gov/oba/rac/XSCID_letter2.pdf

 

T.P. Toma, " Building better gene vectors, " The Scientist, September 18, 2002.

http://www.biomedcentral.com/news/20020918/02/

 

US Food and Drug Administration Center for Biologics Evaluation and Research

http://www.fda.gov/cber/gene.htm

 

 

 

 

 

 

 

 

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