Human Gene Therapy- Dr. Ledley



We met many years ago when you were on the Recombinant DNA Advisory Committee (RAC) and the Human Gene  Therapy Subcommittee, and my team at the Baylor College of Medicine proposed an early trial of gene transfer involving hepatocyte transplantation. We did get an approval (from the RAC) and while we never conducted our proposed trial, it was a seminal experience in my career. Ever since then, I have been a tremendous admirer of everything you represented and have accomplished.


If I can tell you a story.  For a year before we submitted our proposal to the RAC, my team attended many of the meetings of the RAC and Human Gene Therapy Subcommittee to understand the process and be sure our proposal addressed the questions that were being asked. Our conclusion was that our science was as good as anyone else’s (not as good as we wanted it to be, but then state-of-the-art), that we had extensive safety data, and that the scientific questions we would be asked were largely predictable. We also concluded that the most important questions anyone on the committee was asking were yours, which addressed the larger context of responsible investigation, the rights of the individual, and the implications for society. It became a mantra in our laboratory every time we did an experiment or wrote a section of our proposal to ask ourselves out loud “What would Abbey think?” or “What would Abbey want us to do?”  As a result, our proposal included research not only on gene transfer and hepatocyte transplantation, but also the personal and social context of the proposed trial. When we presented our proposal to the Human Gene Therapy Committee, and you were among the first to express your support, it was all our team could do not to start cheering; yours was the opinion we valued the most.



Congratulations on your new book.


Best wishes,


Fred Ledley, M.D.

Professor, Departments of Natural & Applied Science, Management

Director, Center for Integration of Science and Industry

Bentley University

Waltham, MA 


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