Subject:   Concerns RE: Gene Therapy Experiment at NY Presbyterian Hospital
Date:   2003-09-06

A recent gene therapy experiment – the insertion of viruses directly into the brain of a person suffering from Parkinsonism – was uncritically presented to the public on national television. CIRCARE is concerned that there are are serious unknowns involved:

1. Only results with mice (with a disease that mimics Parkinson's) have been publicly discussed, but it is unknown how much we can extend these results to humans – as there is a great deal of difference between humans and mice, particulary in genomic expression.
2. Results with primate experiments have not been made available for any critical review. It would appear, unless otherwise presented, that there is a lack of rigorous trials with primates who are physiologically closer to the human – leaving safety unestablished before the procedure's use in humans.
3. Persons who are consenting are doing so in part because they don't like the idea of the alternative treatment – a stimulator implanted in the brain. However, this dislike of a device being implanted may create a false sense of safety for an organic implant. In the case of Jesse Gelsinger, a gene implant resulted in death, and in recent experiments with children in France several have become sick with leukemia.
4. It is not known if patients were informed of the advantages of a device – that a stimulator can be turned off, whereas a virus can not. How much to give of the virus implant is also unknown – was this established in primate studies? If too much virus is put in the brain there is no way to turn it off and more serious movement disorder can be created, as with wild flinging movements. When fetal cells were implanted in other experiments – in an attempt to more directly address the problem – they grew in a disorganized way and created a more serious movement disorder.

When so much doubt remains, CIRCARE advises not to proceed with human experimentation but with more rigorous animal testing. In addition, the results of animal studies should be placed in the public domain. Subjects may be naively accepting the insertion of viruses into their brains because they don't want a device implanted, versus acceptance based on findings that safety and a degree of efficiacy have been established in primate studies.

See the article in the NY Times: Gene Therapy Used to Treat Patient with Parkinson's. August 19, 2003. P. 1. *

Michael A. Susko MS, CIRCARE President
(410) 499-4895

Paul Gelsinger, Vice President, CIRCARE

* We apologize for providing incomplete information about the New York York Times article. We're attempting to locate the name of the reporter who wrote it.

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Last Updated: 2006-04-25

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