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A tragic death that should not deter gene therapy researchers

4 October 1999
By Juliet Tizzard
Director, Progress Educational Trust
Appeared in BioNews 28
Jesse Gelsinger's parents are no doubt still reeling from the news of their son's death. But as they try to get on with the rest of their lives, a public and professional debate about why he died is raging in the United States. Jesse, who was 18 years old, died after experimental genetic therapy to treat a rare genetic disease, called ornithine transcarbamylase (OTC) deficiency, which had blighted him all his life. Now, a full-scale enquiry is taking place to examine the circumstances of Jesse's death.

The tragedy of the situation hardly needs stating. When someone dies whilst taking part in research which may help others, the sacrifice the participant made is poignant enough. But in Jesse Gelsinger's case, the sacrifice was particularly acute. He had a mild form of the disease and his symptoms had been brought under control by a drug regimen and a strict, non-protein diet. Jesse took part in the trial because he wanted to help other people who suffer from OTC deficiency. His own need for a cure was, although evident, less pressing than that of other sufferers.

Anyone taking part in a clinical trial has to take into account the risks and benefits of doing so. Participants have to balance their desire to help advance medical science with a concern for their own health and wellbeing. No doubt, Jesse Gelsinger did this: he weighed up the pros and cons and decided to take part - along with 17 other OTC sufferers - in the gene therapy trial.

In the public debate in the US, questions have already been asked about the safety of gene therapy and about the process by which clinical trials are approved. The results of the trial will need to be scientifically assessed and the approval process should be monitored, to make sure it's systems are rigorous. But ultimately, the scientific community should not be deterred from continuing with gene therapy research and trials. More importantly, the public should not lose faith in the potential of gene therapy to treat a range of debilitating genetic diseases - even though current work may take some time to bear fruit. Some people have lost their lives for the sake of the advancement of treatments that are now commonplace. Let's hope that, some years from now, gene therapy is regarded as a routine intervention and people like Jesse Gelsinger can live as normal a life as the rest of us.

8 May 2018 - by Dr Charlott Repschlager 
Personalised medicine is a topic that pops up frequently in the news and has been for what feels like a long time...
20 August 2007 - by Ailsa Stevens 
Last month a gene therapy trial for arthritis, carried out by US company Targeted Genetics, was halted when following the death of 36-year-old participant Jolee Mohr. Although the exact cause of her death is still unknown, the usually mild fungal infection found throughout Mrs Mohr's body...
11 February 2005 - by BioNews 
Two US institutions that ran a gene therapy trial in which a teenager died in 1999 agreed to pay more than $1 million in a civil settlement last week. The government had alleged that the researchers, based at the University of Pennsylvania and the Children's National Medical Center, had failed...
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