After the death of a clinical research patient at the University of Pennsylvania, gene therapy has gained nationwide attention. At Duke, officials and researchers believe the Medical Center will continue to proceed with caution as the field develops slowly.
Researchers said Duke's medium-sized gene therapy program is concentrated in animal studies, and the Medical Center is moving with trepidation when it comes to human research.
There is one active protocol for a gene therapy study, but no patients have been enrolled in that study, said Dr. Russel Kaufman, vice dean for education at the School of Medicine.
"I think Duke has been pretty cautious compared to most places, as far as pushing the field of gene therapy into the clinic," said Bruce Sullenger, associate professor of experimental surgery.
Sullenger, whose research group is currently working to develop safer methods of gene therapy, said there is pressure for researchers to move on to testing human subjects.
However, Duke researchers and national experts said gene therapy must be treated responsibly, both at Duke and beyond.
Dr. Adil Shamoo, a bioethicist at the University of Maryland's medical school, said gene therapy "is no more or less dangerous than other research. The scare about gene therapy is due to lack of knowledge."
Shamoo, who is editor-in-chief of the journal Accountability in Research and has chaired international conferences on ethics and research on human subjects, said that with gene therapy trials-like all other kinds of research-there is a "cavalier attitude and a nonchalance" among scientists.
Although Duke researchers may not be at the point of clinical trials, some of them foresee testing on human subjects in the next few years. As the Medical Center becomes more involved with gene therapy, researchers said the field must be investigated carefully.
Andrea Amalfitano, an assistant professor of medical genetics in the pediatrics department, is currently studying the adeno virus and the way it can be manipulated to deliver genes to a variety of organs, including the liver and cardiac tissues.
One of the main focuses of Amalfitano's study is Pompe disease, a muscular disorder. His team tests the use of the virus to deliver genetic information that is missing and makes the virus safer to apply to any disease that affects an organ.
Although he has tested the adeno virus on animals, he said it might be a few years before he begins clinical research. "If you are going to pursue [gene therapy], you must cross the t's and dot the i's along the way," he said.
Amalfitano also said the protocols would be as stringent for gene therapy as for other types of research. "I don't know how extensive an effort gene therapy is [being given] at Duke, but the Institutional Review Board will apply the same principles to gene therapy as they do to all trials.... Those protocols are under the same scrutiny."
The level of scrutiny has increased at Duke since last May, when the Office of Protection from Research Risks suspended Duke's human subject research for four days because of procedural lapses. Kaufman stressed that Duke is enforcing the highest standards for research.
"The Office of Protection from Research Risks has been pleased with our quarterly progress reports and has decided that we no longer are required to submit additional reports," he said. "We will have a site visit by OPRR in May and hope to be on regular status after that visit. We believe OPRR has confidence in our programs."
The death at Penn jumpstarted the debate over gene therapy and clinical trials, both at Penn and at other academic research institutions.
The source of the controversy originated with the death of Arizona teenager Jesse Gelsinger. He fell into a coma and died in September at Penn's medical center after being injected with genes designed to correct an inherited liver disease.
On Jan. 21, the Food and Drug Administration shut down all gene therapy experiments at Penn and said there were, according to a press release, "serious deficiencies in the procedures in place for oversight and monitoring of clinical trials." In response, Penn President Judith Rodin appointed a committee to conduct a comprehensive review of the oversight and monitoring procedures at Penn's Institute for Human Gene Therapy.
Penn recently released a statement denying causing the death of the teenager.
In early February, Congress began examining gene therapy oversight procedures and guidelines enforced by the Food and Drug Administration and the National Institutes of Health.
And in light of the congressional hearing and the death at Penn, several hospitals, including Harvard-affiliated Beth Israel Deaconess Medical Center in Boston, temporarily suspended gene therapy experiments.
Researchers at Duke now find themselves with the task of expanding the field of gene therapy while maintaining acceptable standards for research.
Dr. Clayton Smith, associate professor of oncology, does gene transfer in blood stem cells for cancer and AIDS research, but he is not yet involved with clinical trials.
"I think that there is no question that [gene therapy] is a large part of the future of medicine... but the expectations in the early years were pretty unrealistic," he said.
Smith said he would be surprised if Duke did not have the same stringency that it does for other types of clinical trials, and he said that the recent events that have occurred around the nation should not impede the progress of researchers.
"I think to throw the baby out of the bathwater would be a huge mistake," he said. "It needs to be looked at carefully."
Get The Chronicle straight to your inbox
Signup for our weekly newsletter. Cancel at any time.