Gene therapy – Horizon “Trial and Error”

The excitement about gene therapy received a serious blow in September 1999 with the death of Jesse Gelsinger.  At the time 18 year old Jesse was a participant in a clinical trial for gene therapy to overcome a genetic condition Ornithine Transcarbamylase (OTC) deficiency.  This documentary “Trial and Error” from the BBC’s flagship Horizon series tells the story of that trial and what went wrong.  Although the tone is sometimes unduly sensationalist, it is an excellent introduction to gene therapy.  The story is complicated by serious flaws in the conduct of the clinical trial, which can also make this a good vehicle for discussing appropriate procedures in biomedical research.  

This episode is from the 2003 season of Horizon.  It is frequently repeated on digital TV, particularly the UKTV Documentary channel.  It is also available as an off-air recording from the BUFVC (TRILT identifier 001D70CE).  Watching the whole episode is time well spent.  However, a four and a half minute clip starting at 4:40 with the voiceover “A medical revolution had begun…” and continuing through interviews with French Anderson and Dusty Miller explains the principles of using viral vectors for delivering genes into cells.  A transcipt of the programme is available on the BBC website.

5 Responses to Gene therapy – Horizon “Trial and Error”

  1. […] has had a chequered history (see, for example, the BioethicsBytes notes on the Horizon programme Trial and Error, and news stories about leukaemia risks from one gene therapy […]

  2. […] Despite success stories such as this one, gene therapy has not been without its problems. Several patients, including one at the London clinic featured in this programme, have developed leukaemia as a side-effect of the gene delivery process (see BBC news report), and Jesse Gelsinger, a volunteer in an unrelated American trial, sadly died as a result of complications (see BioethicsBytes post Gene therapy – Horizon ‘Trial and Error’). […]

  3. […] and in a separate trial a french patient developed leukaemia as a result of the gene therapy (See Gene therapy – Horizon “trial and error” and Designer Babies – three […]

  4. […] Gene therapy – the treatment being developed by GenSys is described as “gene therapy” (e.g.00:04:56 and 00:37:15). We are told that the agent has a viral delivery system, but beyond that we are not party to the detail of how the medication would supposedly work. This is far from the worst representation of gene therapy in the movies (that dubious honour still goes to Bond film Die Another Day). Discussion here could pick up on the difference between germ line therapy (Caesar is apparently permanently altered as a consequence of in utero exposure to ALZ112, whereas Rodman’s dad Charles (John Lithgow) requires repeated treatments. A discussion of the real state of gene therapy research could follow, including safety issues with immune response to viral vectors as experience here by Charles and, sadly, in the real life case of Jesse Gelsinger. […]

  5. […] Horizon: Trial and Error (Documentary review) […]

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