Genetic Therapy Inc. said Wednesday it received FDA approvalto conduct the first human gene therapy trial involving thedirect administration of cells producing genetically alteredvectors into patients with brain tumors.

The Recombinant DNA Advisory Committee of the NationalInstitutes of Health approved the therapy. The trial is designedto treat patients with the most common form of brain tumor,primary glioma brain tumors, an always fatal disease. "Mostpatients have about nine months from diagnosis to death," saidMarc Schneebaum, vice president and chief financial officer ofthe Gaithersburg, Md., company (NASDAQ:GTII).

According to the protocol, GTI's vector system will transfer thegene for the enzyme thymidine kinase into dividing tumorcells. Patients will then be given the anti-viral drug ganciclovir,which attacks cells that produce or express thymidine kinase.Ganciclovir will also destroy the cells used to produce theoriginal vector.

The trial still must be approved by the FDA. Schneebaum saidthat he couldn't predict if the FDA would approve the therapy,but he said, "We expect them to. We currently have nine otherprotocols under way right now which are either gene transferor gene therapy." Schneebaum said GTI soon would file forapproval and that the FDA would probably respond within 30days.

GTI is collaborating with the NIH on this trial and retainsproprietary rights, but the NIH would receive royalties fromany financial gains to the resulting technology. Schneebaumwould not disclose the exact terms of the agreement with theNIH, but said that since no other companies are working on asimilar therapy, it "could have significant earning potential forthe company."

The new therapy could brighten the now-grim prospect forpatients with a primary glioma brain tumor. "Other treatmentsfor these tumors haven't been working," Schneebaum said. "Itis our hope that this therapy will reduce or eliminate thetumors.

"The skull is enclosed, so when something grows inside that isnot supposed to be there, it presses against other importantcells that have nowhere to go. Even if we just reduce the size ofthe tumor, you can imagine the benefit to the patient."

-- Steve Payne BioWorld Staff

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