Professor Norman C Nevin Chairman

The first gene therapy clinical trial to be approved started in September 1990. Since then, more than
400 clinical protocols have been approved worldwide. More than 10% of these have taken place in the UK, accounting for around 50% of all European studies. Applications to GTAC this year have focussed on gene therapy approaches to develop potential treatments for cancer.

As we reach the end of this first decade of gene therapy research, I am pleased to note encouraging
evidence of positive clinical results has emerged from trials for severe combined immune deficiency (SCID-IX) and for haemophilia. As well as for its successes, the past year will also be remembered as that in which the first gene therapy treatment-related death occurred in the USA. In September 1999 an 18-year-old man died as a consequence of participating in a phase I gene therapy trial using an adenovirus vector to correct an ornithine transcarbamylase deficiency (OTC), serving to highlight the importance of effective and efficient monitoring of patients involved in gene therapy trials.

In the spring of 1999 GTAC had already commenced a review of serious adverse events and issues related to the monitoring of patients involved in gene therapy research.As a consequence of information received in relation to the USA OTC study, the GTAC review focussed on the adenoviral gene therapy trials. Although no major life-threatening toxicity had occurred with the use of adenoviral vectors, it was decided to convene a working group to develop recommendations for patient monitoring in adenoviral gene therapy studies.

GTAC has been concerned about the lack of provision for long-term follow-up of patients who have received gene therapy. This year, with the co-operation of clinical investigators, GTAC have established a confidential mechanism which will operate via the National Health Service Central Register and provide for the long-term follow-up of UK participants in gene therapy research. Gene therapy clinical trials must continue to be conducted to the highest scientific and ethical standards with patient welfare and safety being paramount. GTAC’s first guidance notes were published in 1994 and I welcome the publication of GTAC’s updated guidance to proposers. I wish to take this opportunity to thank the NETS subgroup of GTAC for their input into updating the guidance notes for investigators and reviewing the new developments.

GTAC also owes enormous gratitude to the external experts who review proposals to conduct gene therapy research and to the expert advisors with whom the Committee consult. I would like to take this opportunity to thank all our external advisors for their efforts over the reporting year.

Finally, it gives me immense pleasure to welcome Dr Sohaila Rastan, Ms Caroline Benjamin and Mr Michael Harrison, who joined GTAC in December 1999.

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