Although the FDA has found "serious deficiencies" with a University of Pennsylvania gene therapy laboratory and shut down eight experiments, investors say they're not worried about their multi-million dollar investments.
The FDA investigated the Institute for Human Gene Therapy at Penn following the September death of 18-year-old Jesse Gelsinger, who participated in an experimental gene therapy treatment for a rare liver disease. The investigators said researchers had violated FDA regulations for patient safety.
"The inspection revealed deficiencies in the procedures for oversight and monitoring of the clinical trial, so there was concern that there may be similar problems in [the institute's other trials] and that patients may be at risk," said an FDA spokeswoman
Gelsinger was the first person known to die as a result of gene therapy, a relatively new field of medicine that treats disease by injecting healthy genes into the body. He had a rare liver disorder called ornithine transcarbamylase deficiency (OTC), a genetic defect that causes ammonia to build up in the body.
Gelsinger was being treated with a genetically modified adenovirus – a common carrier of genetic therapies, which causes colds as well as severe infections such as pneumonia.
His death was a shock to researchers. During a three-day hearing last month, researchers said they were still reviewing the data to find out what went wrong. The FDA said the university must halt all of the experiments "until we have assurance the trials are being run in accordance with FDA regulations."
The university issued a statement saying it would "appoint a committee of scientists without affiliation to the university to review IHGT's oversight and monitoring of clinical trials ... and to recommend any additional remedial steps that may be necessary."
Genovo of Sharon Hill, Pennsylvania, is the lab's main investor, and was founded by Dr. James Wilson, who is also a founder of the lab.
The Philadelphia Inquirer reported that Genovo was considering taking its investment elsewhere. But company spokesman Fred Spar said the company had no such plans.
"The Philadelphia Inquirer article was wrong. They completely misunderstood," he said.
One of Genovo's main partners is Genzyme, a biotech company in Cambridge, Massachusetts. Genzyme also said it wasn't worried about the FDA action.
"The FDA action will not affect the work we're doing with Genovo. It's business as usual," said Genzyme spokesperson Bo Piela. "Our work with Genovo is in its early stages and has not yet reached a point where we will initiate clinical trials."
Piela did not say whether the company, which aims to develop therapies for rare genetic disorders, was concerned about potential clinical trials that could come out of the present research.
"Right now we're just focused on doing the early stage work, and it's one of the many research projects we have to develop the next generation of products."
Biotech firm Biogen, also of Cambridge, invested $35 million in Genovo according to the Inquirer. The company did not return phone calls requesting comment.
In its letter to the Penn lab, the FDA asked university officials to provide detailed descriptions of their plans for monitoring patients in clinical trials. Also, the university must review all gene therapy trials to make sure scientists are following their research plans and abiding by federal regulations.
The cancelled trials were to study potential treatments for diseases such as cystic fibrosis, melanoma, and breast cancer. Five of the eight had already enrolled patients, Wildes said.
The University said in a statement that it complies with the FDA thus far and would continue to do so "promptly and completely."
Dr. Marshall Summar, associate professor of Pediatrics and Molecular Physiology & Biophysics at Vanderbilt University Medical Center, said that the lab's implementation of exclusion criteria, which was brought up in the FDA's letter, could be a problem for Penn at the upcoming hearing. Exclusion criteria are rules by which a patient is either removed or not allowed to participate in a study.
"It is unclear if there were exclusion criteria for the study.... If [Gelsinger met the criteria], then he should not have been enrolled," Summar said. "If there were approved criteria, and he did not meet them, then his death is tragic, but a part of the risks of experimental medicine."
Summar was less concerned about the FDA's comments about the lab not having proper documentation for informed consent. He said that informed consent was obtained, but that the lab may have failed to fill out documentation to FDA standards. "In most any large study, one can find discrepancies in paperwork when close examination is applied."
The hearing is scheduled for 9:30 a.m. EST, 2 February, and will be chaired by Senator Bill Frist (R-Tennessee).
Reuters contributed to this report.
