$1 Million to Be Paid Over Teen’s Death in Gene Therapy Study

The University of Pennsylvania and the Children’s National Medical Center have agreed to pay a total of more than $1 million to settle fraud allegations related to the death of a teenager during a gene therapy experiment.

Jesse Gelsinger, 18, of Tucson died on his fourth day in the study in 1999.

The teen had suffered from an inherited disorder that blocks the body from properly processing nitrogen. Researchers had hoped to cure him by injecting him with a modified virus carrying a gene that could replace the medications and special diet that had been controlling his condition.

The Food and Drug Administration concluded that the injection killed him.

After the death, Gelsinger’s family said the teen had been misled about the experiment’s potential risks.

Federal prosecutors alleged in a civil complaint that researchers should have discontinued the experiment once they realized it had unacceptable side effects. They also claimed that researchers had submitted reports to the government misrepresenting the study’s findings.

Under the settlement, the university will pay the government $517,496. The Children’s National Medical Center will pay $514,622.

The two institutions have maintained that Gelsinger’s death was unforeseen and that he was properly enrolled in the study based on the best scientific information available at the time. Neither institution was required to acknowledge any wrongdoing as part of the settlement.

Gelsinger family attorney Alan Milstein said the family was “extremely disappointed” because the agreement did not require the public release of all documents in the case.

Releasing the documents would ensure that “real changes could be made in the way human research is conducted in this country,” he said.

Penn officials said in a statement that in the five years since Gelsinger’s death, they had overhauled their rules for clinical research on human subjects.

“Out of this tragedy has come a renewed national effort to protect the safety of those who help to advance new treatments and cures through clinical research,” the statement said.

Three researchers – Dr. James M. Wilson, Dr. Mark Batshaw and Dr. Steven Raper – also will have restrictions placed on their work. Under the settlement they do not admit any wrongdoing.

The settlement requires that Wilson wait until 2010 before again leading research on humans. He has been barred from involvement in such studies since 2000.

Batshaw and Raper face lesser restrictions.