Gene Therapy Study To Resume After Woman’s Death

Federal regulators have given permission for a company to resume a gene therapy study in which a woman died, the company is expected to announce today.

The company, Targeted Genetics, said the decision by the Food and Drug Administration is effectively an acknowledgment that the gene therapy did not cause the womans death.

I think its a vindication for the product, for the company and really for gene therapy, H. Stewart Parker, the chief executive of Targeted Genetics, said Friday in an interview.

The death had threatened to be another black eye for gene therapy, a field that has not had much success in treating disease and that suffered a setback in 1999 when a teenager died while participating in a clinical trial at the University of Pennsylvania.

A spokeswoman for the F.D.A. said the agency could not comment on its communications with companies involving drugs in clinical trials. The agency halted the clinical trial in July after the death of the woman, Jolee Mohr, 36, of Taylorville, Ill.

Her husband, Robb Mohr, said yesterday that he had not been convinced that the gene therapy was not a cause of Ms. Mohrs death and said the trial should not resume.

I think its very reckless, he said in an interview. They still dont know what caused her death. They still havent reported on her death.

Gene therapy involves implanting genes, usually carried by viruses, into a patients cells to induce them to produce some needed protein. Ms. Mohr, the mother of a young daughter, died three weeks after receiving her second injection of a gene meant to reduce inflammation and treat her rheumatoid arthritis.

The direct cause of her death appeared to be a fungal infection, histoplasmosis, according to testimony at a review of her case in September by an advisory committee to the National Institutes of Health. But the committee said it was too early to conclude that the gene therapy had not been a factor, perhaps by suppressing Ms. Mohrs immune system.

The committee, called the Recombinant DNA Advisory Committee, is scheduled to review the case again on Dec. 3.

But since September, Targeted Genetics has presented new data showing that the gene and the viral carrier, which were injected into Ms. Mohrs knee, had not spread widely through her body.

It has also presented data it says suggests that the level of immune suppression from the gene therapy was minuscule compared with that from Humira, an arthritis drug Ms. Mohr had been taking. Humiras label has a warning about the risk of fungal infections.

But Mr. Mohr said it was impossible to distinguish the immune suppression caused by the gene therapy from that caused by the Humira.

Ms. Parker, the Targeted Genetics executive, said patients wanting to continue in the trial would have to sign a new consent form, which was being revised to reflect Ms. Mohrs death. And review boards at all trial sites will have to approve new safeguards for the trial, like not treating patients who have fevers.

The early stage trial, which had 127 participants, was mainly devised to test the safety of the treatment. When it was halted, about 35 people had not yet received the second of two injections. Ms. Stewart said that while some patients could be reluctant to rejoin the trial, the company thought many would take part.

Dr. Mark A. Kay, a Stanford University professor active in gene therapy but not connected with Targeted Genetics, said the resumption of the trial was appropriate. Its really tragic this woman died, he said, but it really didnt appear to be due at all to the gene therapy.

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