The Associated Press
The Associated Press
BETHESDA, Md. – A University of Pennsylvania researcher said he was ”fully comfortable” with a decision to include in a gene therapy experiment an Arizona teenager who later died.
Dr. James Wilson, head of a Pennsylvania research team, said yesterday he and his team decided to conduct the gene therapy on Jesse Gelsinger even though a blood test showed evidence that 18-year-old’s liver was not functioning well.
Gelsinger, who died in September, is the first death as the direct result of a gene therapy experiment.
Wilson was to formally defend his work today before a National Institutes of Health committee that is investigating Gelsinger’s death and considering measures to sharpen federal oversight of gene therapy medicine.
The Recombinant DNA Advisory Committee also is considering new guidelines that force public disclosure of any deaths or serious illnesses, called ”adverse events,” that occur among patients in the gene experiments.
Yesterday, Food and Drug Administration officials charged that Gelsinger ”did not meet the entry criterion” for taking part in the clinical trials and should not have been included by the Pennsylvania researchers.
The FDA also said the researchers failed to properly report other ”adverse events” from their gene therapy trials.
Wilson denied the allegations against his team’s research efforts.
Gelsinger’s death prompted the extraordinary three-day RAC hearing that has attracted an auditorium full of patients, researchers and drug company scientists.
The Tucson teen-ager suffered from a genetic liver disorder and was enrolled in an effort to correct his condition by inserting normal genes into his liver. He died within days following a gene injection.
Kathryn Zoon, chief of the FDA’s Center for Biologics Evaluation, said ”preliminary findings” of an FDA investigation show Gelsinger had elevated ammonia in his blood, an indication of a distressed liver, at the time he received the gene injection.
”The entry levels for ammonia (readings) were not met,” Zoon said.
Wilson, in a statement, said Gelsinger did meet the FDA requirements when he enrolled in the trial, but that his ammonia levels were ”slightly elevated” just before the gene therapy injection was made.
”We remain fully comfortable with the clinical decision to use alternate pathways to lower that level and proceed with the trial,” Wilson said.
Using genes to correct or even cure conditions from cancer to heart disease is considered one of the most promising therapies on the medical horizon.
He also said the FDA was notified of the two other patients who suffered liver damage.
The researcher issued a statement, but refused to answer questions yesterday.