Where Are We Going

Gene therapy: lessons learnt from the past decade

BMJ 1999; 319 doi: https://doi.org/10.1136/bmj.319.7220.1310 (Published 13 November 1999) Cite this as: BMJ 1999;319:1310
  1. Richard A Morgan, interim chief (rmorgan@nhgri.nih)a,
  2. R Michael Blaese, chief scientific officerb
  1. Clinical Gene Therapy Branch, National Institutes of Health, Bethesda, MD 20892, USA
  2. Kimeragen Inc, Newtown, PA 18940, USA
  1. Correspondence to: R A Morgan

    It is more than a decade since the first approved clinical trial to put genes into the cells of human beings was initiated. In that first trial, investigators at the US National Institutes of Health used a modified mouse leukaemia virus to insert a DNA marker into lymphocytes being used to treat cancer.1 A year later, a similar viral technique was used as treatment for two girls who had severe combined immunodeficiency with mutant adenosine deaminase.2 Ten years and more than 3000 patients later, it is appropriate to ask, “Where are we now?”

    Summary points

    The first approved human gene transfer experiments took place 10 years ago; more than3000 patients have been treated since

    Because the technical challenges were not fully understood, results from early trialsere perceived as unsuccessful

    Technical challenges—including in vivo delivery, the immune responses, and generegulation—have been researched vigorously and progress has been made

    The list of gene transfer vectors that can be used in gene therapy experiments is growing, as is understanding of the biology and immunology of these systems

    Gene therapy is now a robust scientific discipline with an array of new reagents which will soon be ready for specific clinical applications

    Where are we now?

    Initial speculation that gene therapy would quickly revolutionise medicine has clearly been wrong. As is often the case when entirely new areas of work develop, there was anoveroptimistic view of the pace of progress and an underestimation of the problems remaining to be overcome. In something of a knee jerk reaction, many scientific pundits declared gene therapy dead or, at best, a potentially useful research tool that was being aggressively oversold by a few biotechnology companies. In reality, the science of gene transfer was progressing quickly in a classic reiterative process, where lessons learned from the early clinical studies were redirecting the course …

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