Closing?
The death of Jolee Mohr, here with his family, was unrelated to the gene therapy treatment she received, the sponsor of the study.
Mohr Family / AP Images
Federal regulators have given the green light to a test of arthritis gene therapy who was arrested last summer after that one patient died. New tests indicate that the therapy had no role in the death. The decision is a relief for gene therapy researchers who had worried about a potential new side of their field.
The trial by Targeted Genetics Corp. was arrested after the Death July 24 to 36 year old Jolee Mohr of Taylorville, Illinois, which received an injection of gene therapy to treat her rheumatoid arthritis in a knee 3 weeks earlier ( science , 3 August). During September 1 meeting of the Recombinant DNA Advisory Committee Federal (CAR), the experts noted that Mohr apparently died mainly from a fungal infection called histoplasmosis that his immune system was unable to fight ( Science NOW , September 17). Mohr immune system may have been compromised by a drug she was taking against arthritis, Humira, which blocks an inflammatory molecule called α tumor necrosis factor (TNF-a). The protein produced by the gene therapy is also a blocker of TNF-α, and if it spreads beyond the knee Mohr, combination with Humira can be left vulnerable to the fungus.
This was not the case, according Targeted Genetics. The company stresses the new test results from a survey conducted by the company and external researchers, which were presented at the American College of Rheumatology annual meeting earlier this month. The level of TNF-α blocker detected in the blood of Mohr with a binding assay was "well within the normal range expected" from the dose of Humira that she was taking, says President and CEO H. Stewart Parker. The new tests have also dismissed the idea that the gene therapy vector, adeno-associated virus reproduces in the body of Mohr. While the vector DNA turned in other tissues, the amounts were extremely low. Today, the company announced that the US Food and Drug Administration lifted the hold on the trial.
Others agree that the case is closed. "It does not look like gene therapy has been instrumental to the best of our knowledge," said the doctor Kyle Hogarth of the University of Chicago in Illinois, who treated Mohr and took part in. However investigation the Hogarth question whether it makes sense to include patients who are already taking TNF-blockers in the study, because it is difficult to distinguish between the effects of gene therapy product and drugs. Parker is disagreed, noting that rheumatologists say "there is a significant unmet need" for treatment for patients with joints that do not respond to systemic medications.
the company now plans to resume the trial of 127 patients but does give a second dose to as many as 35 patients waiting if they have a fever, as Mohr did. "We are being conservative," said Parker. She said the only change in the consent document will be lit to confirming death earlier. RAC will publish its final conclusions on the case at a meeting next week. RAC President Howard Federoff of Georgetown University in Washington, DC, declined to comment in advance of the meeting.
Related Sites
- Targeted Genetics press release
- Information 17 RAC meeting in September on the death arthritis
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