Dr. LeRoy Walters, Chair, called the meeting of the Working Group on Hunan Gene 
Therapy to order at 9:13 a.m. on October 12, 1984. Dr. Walters we leaned the 
participants and asked them to identify themselves. 
Dr. Walters drew the attention of the working group to a draft document 
(Attachment II) which had been prepared by a volunteer subgroup (Ms. Areen, 
Mr. Capron, and Drs. Anderson, Gartland, Gorcvitz, Gottesman, Milewski, Miller, 
Murray, and Walters) of the working group. The subgroup had used four sources 
to develop the draft document: (1) The Department of Health and Human Services 
(DHHS) Hunan Subjects Regulations; (2) the document Splicing Life by the 
President's Canmission for the Study of Ethical Problems in Medicine and 
Biomedical and Behavioral Research; (3) the Pood and Drug Administration (FDA) 
IND Medical Review Guidelines (Attachment III); and (4) the FDA Points to 
Consider in the Characterization of Cell Lines Used to Produce Biologicals 
(Attachment IV). 
Dr. Walters asked the working group to begin by offering general canrrents on 
the draft document (Attachment II). He asked the working group to focus primarily 
on whether the document omitted important points or had skewed or wrong emphases. 
Dr. Varmus said the draft document (Attachment II) attanpts to elicit information 
but does not indicate guidelines for making decisions. For example, the document 
does not indicate what clinical goals the working group might consider legitimate. 
Dr. Walters agreed the draft document raises questions but gives no indication 
of what would be satisfactory answers. He asked the working group to consider 
whether this approach was appropriate. 
Dr. Anderson said at some time the working group might have to decide whether 
there might be a "right" answer for sane questions. He thought the working 
group could defer that type of discussion, however, until the first human gene 
therapy proposals have been received. 
Dr. Gottesman suggested the document should contain an introduction or preamble 
which helps to orient the investigator; the preamble would explain why the 
working group is looking for particular information and would indicate the 
amount of detail a submission should possess. She thought the preamble should 
state that the points to consider document w/as developed on the assumption that 
it applies to somatic cell therapy. 
Dr. Mot ul sky said although gene therapy has a special aura, it does not raise 
special or unique concerns. Most of considerations pertinent to gene therapy are 
also pertinent to other therapies. He said a preamble to the working group 
document should state that gene therapy differs from other therapies only in 
the use of DNA. 
Dr. Gorovitz said the public perceives gene therapy as special. He argued that 
the working group must do more than simply say a therapy presents "no special 
risk." He said genuine concern exists amoung the public and even ill-grcunded 
concerns can have a great impact. He offered as an example the moratorium the 
U.S. imposed on experiments to further knowledge in _in vitro fertilization of 
human embryos ; this moratorium was imposed because of ill-informed public 
concern and resulted in the technology being developed with private finds 
[ 90 ] 
