Jennifer Couzin
and Jocelyn Kaiser
Five years after 18-year-old Jesse Gelsinger died in a gene therapy
experiment, the U.S. Department of Justice has reached a settlement
with the researchers and with their institutions. The department announced
last week that the University of Pennsylvania (U. Penn) will pay fines
of $517,496, and Children's National Medical Center in Washington, D.C.,
will pay $514,622. The settlement also restricts the clinical research
of the three investigators.
The Department of Justice
alleged that toxic reactions in humans should have halted the trial
earlier and that the lead investigators misrepresented clinical findings
to the study's overseers, such as the National Institutes of Health
(NIH) and the Food and Drug Administration (FDA). James Wilson of U.
Penn, who had a financial interest in a company that stood to profit
if the trial was successful, has agreed not to lead any FDA-regulated
clinical trials for 5 years and be monitored for 3 years. Steven Raper
of U. Penn and Mark Batshaw of Children's face less severe restrictions.
Under the agreement, the scientists do not admit responsibility for
Gelsinger's death. "Outrageous," responds Gelsinger family
attorney Alan Milstein, who said the family had hoped for a formal apology
and the release of the clinical trial documents.
While the Gelsinger case
drew to a close, the field of gene therapy suffered another setback
last month: A third child in a French trial for X-linked severe combined
immunodeficiency (X-SCID) developed leukemia, French authorities reported
on 24 January. Seventeen children have been successfully treated for
SCID using gene therapy, making it the field's bright spot. But two
patients in the French trial developed leukemia in late 2002 after a
vector inserted near an oncogene; one child died last October. In response
to the third leukemia case, the French trial has been halted again and
FDA has suspended three U.S. SCID trials, but a trial in Britain continues.
The two previous leukemia
cases in France occurred in infants treated at 3 months of age or less,
which led to speculation that cells with the oncogene insertion proliferate
more readily in very young children. But the third child was treated
at 9 months, suggesting that older children may also be at risk, says
Harry Malech of NIH, who heads one of the U.S. trials. Experts expect
to discuss the case when FDA's gene therapy advisory committee and NIH's
Recombinant DNA Advisory Committee meet in March.
