Woman's death calls gene therapy into question

No disciplinary action
Details about the experiment’s other volunteers have not been released by Targeted Genetics. The government says the study recruited people through doctors in small cities — Springfield, Willow Grove, Pa., Glendale, Ariz., Ocala, Fla., and Coeur d’Alene, Idaho.

Tom Murray, president of the Hastings Center, an independent, nonprofit research institute on medical ethics, said seeking patients through smaller clinics tends to save money compared with doing the work at major medical centers.

“The good thing is more research is done at lower costs. The bad thing is you may not have the same depth of protections that the academic centers provide,” he said.

Trapp, who did not respond to several requests for an interview, enrolled “seven or eight” patients in the gene therapy study, according to his attorney, David Drake. He described Trapp’s enrollment procedures only as “standard,” and he declined to comment on how much money Trapp’s clinic received from Targeted Genetics, fees meant to reimburse costs.

Trapp, 59, has not faced state disciplinary action and his license is active. In 2002, he settled out of court with a patient who sued him for negligence after she developed eye problems known to be a side effect of an arthritis drug he had prescribed.

The investigation into Mohr’s death has steep consequences for Targeted Genetics. The company, which employs 70, has no products on the market and several years ago cut workers and several of its programs.

“A very exciting lead product with the possibility to meet significant unmet need could be jeopardized,” said CEO H. Stewart Parker when asked what was at stake for the company. “A program we’ve spent years on, one where there is a tremendous amount of preclinical data supporting it, is under examination.”

She said the company believes the study was done with the “highest level” of responsibility and that it followed federal guidelines. She said there’s never been any rush to get the study drug tested and on the market.

Parker also noted that key tests have not yet been done that could link Mohr’s death to the gene therapy study. A spokesman at the University of Chicago, which is conducting the tests, said they are under way but that several are needed to determine the cause of death.

Targeted Genetics believes Mohr might have died of a fungal infection that could be blamed on anti-arthritis drugs she was taking, Parker said.

The study included a Chicago site where Dr. Eric Ruderman of Northwestern University was in charge. He said none of the patients experienced any problems with the injections.

Ruderman also said the company paid him to cover the research costs at his site, as is customary in clinical research. He declined to disclose the amount.

“There are no bonuses for enrolling a specific number of patients or keeping patients in for the full study. Even if a sponsor were to offer this, I would not accept it,” he said.

Patients’ parking costs were covered, roughly $20 each, but they otherwise weren’t paid to participate, he said.

Industry involvement in gene therapy research is virtually a necessity, since experiments can cost millions of dollars, Friedmann said.

“It’s certainly an added complication when there’s an issue of transparency and profit issues that industry has to keep in mind,” Friedmann said.

Ruderman said he worries the public has the wrong impression about gene therapy research.

“I’m hopeful that people will look at what happened and realize that’s why you do a safety study, and not say, well, this kind of therapy is out the window,” he said.

Wondering why
Mohr’s family asked the University of Chicago to lead the investigation into her death, but the FDA is also investigating, spokeswoman Karen Riley said.

“If there are any implications for the safety of other gene therapy trials, we will take whatever actions that may be needed,” she said.

The NIH began developing guidelines on gene therapy in the mid-1970s and formed the Recombinant DNA Advisory Committee to review studies, procedures and safety.

Mohr’s death tops the agenda for the committee’s regularly scheduled quarterly meeting Monday at the NIH campus in Bethesda, Md. University of Chicago doctors will present autopsy results, though the cause of death and what role the gene therapy might have played are not expected to be ready by then.

Robb Mohr will be there.

“I think it’s great that there’s companies out there wanting to help people, but I also think there’s companies out there that are trying to make millions of dollars on people before they can truly understand what’s going on,” he said.

Two days before she fell ill, the family went boating. The last family snapshot of Jolee was taken on the boat by Toree, and Jolee beams at the young photographer.

“My wife has never looked so beautiful,” Robb Mohr said.

“She had nothing to gain from this study,” he said. “Why would a 36-year-old mother of a 5-year-old that’s going to kindergarten want to participate in something just to see how safe it is on her body? Nothing adds up.”

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