BETHESDA, Md. -- The era of gene therapy "is almost upon us,"buttechnical and regulatory complexities will delay itswidespread use, Bruce Merchant, vice president of ViageneInc., said Monday.

Merchant told a meeting of the Food and Drug Law Institutehere that gene therapy will be the next major development inpharmaceuticals. However, he said "it will take many decadesof effective development before it will have major impacts onthe way in which health care is provided to the public."

Merchant said that delivery problems will be among the mostformidable in creating effective gene therapies.

Although gene therapies are at an early stage of development,it is important to address the regulatory issues early on, saidPhilip Crowley, a regulatory attorney for Johnson & Johnson.Wide-ranging discussion in the scientific and regulatorycommunities paved the way for recombinant DNA research, henoted.

Speakers -- from industry and government -- described theunique set if regulatory hurdles that gene therapy protocolsmust overcome. All gene therapy protocols must be approvedby the FDA and can face examination by an advisory panel. Inaddition, gene therapy protocols that involve an institution thatreceives funding from the National Institutes of Health mustalso be approved by the NIH's Recombinant DNA AdvisoryCommittee (RAC).

Participants emphasized the importance of limiting genetherapy experimentation to somatic cell lines. "Thepredominant concern is that somatic gene therapy remainsexactly that" Merchant said. "Gene therapy must be limitedstrictly to that area and that domain. ... Any design of clinicalstudies must take into account very careful monitoring of thegenetic information that is provided to assure that it doesn'tactually end up going to germ line cells and thereforepotentially affecting future generations."

FDA officials said that the regulation of ex vivo gene therapiesposes new challenges. Regulation of in vivo gene therapy issimilar to drug and biologic regulation; the substance that isintroduced into the body is the object of regulation. In ex vivogene therapy, however, processes and facilities must beregulated.

-- Steve Usdin BioWorld Washington Bureau

(c) 1997 American Health Consultants. All rights reserved.