Biomedical Engineering Reference
In-Depth Information
be completed before a clinical trial of human gene transfer can begin.
15
In December 2000 and again in November 2001, the NIH proposed the
establishment of a Human Gene Transfer Safety Assessment Board to
evaluate adverse events in gene transfer trials in an organized, system-
atic manner and to report regularly to RAC.
16
This board received final
approval from the Office of Management and Budget in January 2002,
and the revision of the guidelines that authorizes its establishment
was published in the
Federal Register
in May 2002.
17
Moreover, in
September and December 2001, RAC engaged in an extended discussion
about the serious adverse events that had occurred in two clinical trials
designed to study gene transfer in subjects with hemophilia. In response
to the adverse events in the French trial of gene transfer for severe com-
bined immunodeficiency, RAC provided detailed public analyses in
December 2002 and February 2003. Finally, the number of RAC
members has been expanded beyond fifteen so that more areas of scien-
tific and clinical expertise can be represented on the committee.
For its part, the FDA announced in January 2001, during the waning
days of the second Clinton administration, its intention to make public
“certain data and information related to human gene therapy and xeno-
transplantation.”
18
Public comments on this proposal will be considered
before the new policy is enacted. The death of a healthy volunteer in an
asthma study being conducted at Johns Hopkins University in June 2001
reminded researchers and the public alike that research subjects can be
at serious risk even in seemingly innocuous trials.
19
Several months after
this volunteer's death, the FDA established a new Office for Good
Clinical Practice within the Commissioner's Office “to improve the
conduct and oversight of clinical research and to ensure the protection
of participants in FDA-regulated research.”
20
Like RAC, the FDA has
also analyzed the leukemias that occurred in the French gene transfer
trial—at public advisory committee meetings held in October 2002 and
February 2003.
Issues for the Future
When we look to the future, it is quite clear that the most critical issues
involving human gene transfer research will involve the brain (especially
behavioral traits), enhancement by genetic means, and the human germ
