The National Institutes of Health has a formal procedure forgranting requests for experimental gene therapy through itsRecombinant DNA Advisory Committee (RAC). So it wasunprecedented when NIH Chief Bernadine Healy bypassed itDec. 28 to grant a request for "compassionate" treatment for a51-year-old woman dying of a brain tumor.

This followed Healy's consultations with just a few of the NIH'sleading cancer researchers. The FDA had already approved thetreatment.

On Thursday the RAC voted 9-3, with one abstention, toapprove Healy's action as the outline for an interim procedure,pending development of a formal procedure for dealing withemergency requests, which is expected to happen at the RAC'sMarch 1 meeting. Researchers at institutions that receive fundsfrom NIH must submit protocols for experimental genetictreatment to the RAC.

Thursday's RAC meeting, said French Anderson, professor ofbiochemistry and pediatrics at the University of SouthernCalifornia School of Medicine, "was very constructive, but gotvery emotional." The patient's husband described his year-longefforts to obtain treatment for his wife, and pointed out theimportance of establishing emergency procedures at NIH, suchas those FDA already has.

But several RAC members expressed concern "that this mightopen the door for 100 requests tomorrow, and how do you dealwith it while being fair to everybody," Anderson recounted.

Gene therapy may open a huge market for the biotechnologyindustry. "If you consider the drugs that are most effective inmedicine, almost without exception they act outside of the cell,"said Robert Abbott, president and chief executive officer ofViagene Inc. "The drugs that have the least efficacy act insidethe cell. ... In oversimplified terms, because it produces theproduct inside the cell, gene therapy opens up a horizon ofmedical practice that is as large as that which has existed untilnow."

Gene therapy has come far, fast, said Anderson. "Three yearsago we were called 'weirdoes' even to be suggesting genetherapy, and now NIH is approving compassionate exemptions."Currently, about 50 patients have been injected with genetherapy products, said Abbott. But this particular case isprobably no more of a milestone "than the first patient thatFrench Anderson treated with adenosine deaminase (ADA)," hesaid.

The woman granted compassionate treatment had beendiagnosed a year ago and had undergone several operations,which had failed to arrest the spread of the tumor. Herestimated survival time is currently less than two months.

The purpose of this gene therapy, performed by Ivar Royston,is to highlight the tumor for the patient's immune system.Normally the immune system destroys tumor cells. Roystonremoved tumor cells from the patient, grew them in culture,inserted the gene for interleukin-2, and re-injected them intothe patient's bloodstream.

Another protocol for treating brain tumors has just begun atNIH. And several tumor vaccine protocols that have recentlybegun operate on the same general principles.

Although gene therapy has proven successful in animal studies,in humans "there is no evidence yet that it works for anythingbesides ADA deficiency," said Anderson.

But Anderson said that it should work. "That is the reason whyI have been a believer in gene therapy for 30 years. Theconcept is so simple, it's got to be right."

-- David C. Holzman Special to BioWorld

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

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