Giving up on gene therapy is wrong reaction

Arthur Caplan, Ph.D. • E-mail

The recent death of Jolee Mohr is likely to have a seismic impact on the future of gene therapy research.

Biotech companies, private investors and government funders will shy away from sponsoring further research because Mohr died while a subject in an experiment using genetic engineering to treat disease.

But giving up on gene therapy is not the right lesson to learn from this tragedy.

Mohr had rheumatoid arthritis, an immune system disorder that caused pain and stiffness in her joints.

By using HUMIRA, a relatively new anti-inflammatory drug made by Abbott Laboratories, she was able to keep her symptoms under control. 

Despite her arthritis, Mohr played with her 5-year-old daughter, gardened, went boating and accompanied her husband, Robb, to church. She even volunteered at county fairs.

So how did it happen that this active 36-year-old woman wound up dead in a Chicago hospital?

Jolee Mohr died soon after her right knee was injected with genetically altered viruses as part of a research trial of gene therapy for rheumatoid arthritis. The viruses were aimed only at the cells in her arthritic knee, hopefully making this approach to treatment safer than the existing drug treatments. Targeted Genetics Corp., the Seattle-based company that sponsored the rheumatoid arthritis study, has halted the work and dozens of participants are being monitored.

The irony of gene therapy is that despite its name, it has achieved little in the past decade in terms of therapy but has, rather, been associated with serious problems including deaths.  Eight years ago, 18-year-old Jesse Gelsinger died in an early experiment with gene therapy at the University of Pennsylvania. Ironically, Targeted Genetics Corp. had acquired some of the genetic technology that had been used in the experiment in which Gelsinger died. 

The death of Jolee Mohr may be seen as the latest best reason to give up on gene therapy.

The finger-pointing about her death is well underway. Targeted Genetics Corp. has already hinted that maybe Mohr died not because of the gene therapy, but as a result of problems associated with HUMIRA, the arthritis drug she was taking before she signed up for the gene therapy trial. 

Robb Mohr says his wife did not understand that she had agreed to be in a safety study of gene therapy, which was not intended to benefit her in any way. Dr. Robert Trapp of the Arthritis Center of Springfield, Ill., who had been Jolee Mohr’s physician for seven years and recruited her into the study, says she signed a 15-page informed consent form and that he answered all her questions about the study. The study and its consent forms were reviewed and approved as U.S. law requires by an institutional review board, this one set up for the for-profit research.

If in fact Mohr did not understand the nature of the research she agreed to participate in, is this board partly to blame?

This question leads to another: Did the doctor closely monitor Mohr’s health before enrolling her in the gene therapy trial? And another: Shouldn’t someone other than her doctor have made the request for her to participate in a safety study of gene therapy since it might be easy for a patient to assume that a request from her doctor to try something would not be made unless the doctor thought there was some benefit in it?

I have had the chance to see the informed consent form that Jolee Mohr signed. In my opinion it is hard to read and not as clear as it should have been about the fact that she was being asked to participate in a safety study with risks but no expected benefits whatsoever for treating her arthritis. 

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