Gene Therapy Controversy
correspondent John Roberts reports on the controversy involving clinical trials in gene therapy after the death of a patient last year.
Fifty-six-year old Phil Pilla didn't want to follow in his father’s footsteps. Pilla has a hereditary heart disease that blocks the flow of blood to his heart, a disease that killed his father and nearly killed him.
"I've had bypass surgery, triple bypass surgery back in 1990 and ten years later they were all block up again," says Pilla.
Causing Pilla to suffer two heart attacks last August, doctors told Pilla that it was too risky to operate on his arteries again. So, in December, Pilla enrooled in an experimental gene therapy program at St. Elizabeth’s Medical Center in Boston.
"I had no recourse," says Pilla. "They inject protein DNA right into the center of my heart. What it was supposed to do was to rejuvenate blood vessels inside my heart that were damaged."
Pilla's wife Rhonda thought he was much better after a few treatments. "We've seen an improvement and our children have seen an improvement," says Rhonda. "They see a dad that has a lot more stamina, a lot more energy."
But this year, the Food and Drug Administration (FDA) investigated Dr. Jeffrey Isner, who was conducting the clinical trial. It found Isner had accepted patients with cancer in the study and had failed to properly report the death of one participant. Both are clear violations of research protocols. The FDA shut the trial down.
"The FDA is getting involved in this and I know that it's experimental and I know that they have rules but if something is working, leave it alone," says Pilla.
Gene therapy--attempts to use normal genes to replace faulty inherited genes-- has been under scrutiny since the death of 18-year-old Jesse Gelsinger in a study at the University of Pennsylvania last year. Of the 300 gene therapy clinical trials now under way, seventy have been investigated and a handful have been closed down.
"Safty should never be short changed and patients should never be put at risk at the expense of just adding another patient to a clinical trial," says Dr. Jane Henney, FDA Commissioner.
Henney wants stricter rules for gene studies and stiffer penalties for institutions and doctors who fail to follow them. "We've clearly gotten both the institutions’ and the investigators’ attention and assurances that they'll take corrective actions," says Henney.
Michael Grodin, a medical ethicist at Boston University, says that new rules will not ensure patient safety and he is calling for a moratorium on all clinical trials.
"All too often I think these things are over sold as if they are therapy and in fact we don't know that they work," says Grodin. "We don't know if they are safe we don't know if they are effective and in fact all the evidence is to the contrary that people haven't been helped bt harmed."
Dr. Robert Pergolizzi, who conducts gene studies, believes that science is promising. "We learn from experiments and we build on that and we go forward and there's nothing to apologize for," says Pergolizzi, director at North Shore LIJ Health System. "The fact that antibiotics is better now than they were during the Second World War doesn't mean that scientist then had anything to apologize for."
The first breakthrough in gene therapy came two months ago, scientists in France treated three children each born with a severely deficient immune system --sometimes called the bubble boy disease. The treatment worked so well that the children are all expected to live a normal life. That gives hope to Phil Pilla. "Heart disease you can't cure," says Pilla. "I was hoping that, you know, this may prolong my condition."
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