19 
Drs. Friedman and We ns ink agreed a discussion qroup on retroviruses would 
be helpful. Mr. Mitchell said any RAC members interested in participating 
in such a discussion group should contact ORCA. 
VII. REPORT OF WORKING GROUP ON HUMAN GENE THERAPY 
Dr. Walters, Chair of the RAC Working Group on Human Gene Therapy, said 
the working group had developed a guidance document (tabs 1201, 1213, 
1214, 1215) entitled "Points to Consider in the Design and Submission of 
Human Somatic-Cell Gene Therapy Protocols" (Attachment III). 
Dr. Walters said this guidance document begins with an introduction to human 
somatic-cell gene therapy. Part I, entitled "Description of Proposal", 
requests first the objectives and rationale of the gene therapy protocol. 
The document subsequently poses a series of issues which parallel the 
general DHHS regulations for all research involving huiran subjects including 
research design, anticipated risks and benefits, selection of subjects, 
informed consent, and privacy and confidentiality. Part II of the document 
raises issues that are not usually evaluated by local review committees. 
One such issue is appropriate provision for sharing information with the 
public in a timely and accurate fashion. Part III of the points to consider 
outlines requested documentation and Part IV, reporting requirements. 
Dr. Walters said the points to consider document had been published for 
public comment in the January 22, 1985, Federal Register ; fifteen letters 
were received in response to this announcement . 
Some modifications (tab 1213) have been introduced into the document in 
response to these comments. For example, the document's introduction more 
clearly accents the difference between somatic-cell and germ line gene 
therapy. The introduction reviews the multi-year process of public discus- 
sions concerning gene therapy. It now includes a quote from the 
December 1984 Office of Technology Assessment paper. Human Gene Therapy , 
vhich states: 
"Civic, religious, scientific, and medical groups have all accepted, in 
principle, the appropriateness of gene therapy of saratic cells in 
humans for specific genetic diseases. Somatic cell gene therapy is 
seen as an extension of present methods of therapy that might be 
preferable to other technologies." 
Dr. Walters said the revised document now asks about information on prior 
laboratory studies in non-human primates and/or other laboratory animals. 
The phrase "and/or other laboratory animals" has been added to this version 
of the points, and indicates the working group’s willingness to consider 
human gene therapy proposals on a case-by-case basis before the ccrrpletion 
of laboratory studies in multiple species of animals. 
Dr. Walters said the document has also been modified to request an agreement 
by prospective patients or their guardians to permit long-term follow-up and 
[139] 
