CEO of Controversial Gene Therapy Company 'Talked From Both Sides of Her Mouth'

The death of a person involved in a gene therapy trial run by Seattle-based Targeted Genetics has recalled the tragedy of Jesse Gelsinger, who died in 1999 while receiving an experimental treatment for liver disease at the University of Pennsylvania.

Jesse's father, Paul, became an advocate for clinical trial reform. I called him today and asked if he had any comment on the latest case.

They were using an adeno-associated virus. My understanding of that virus from years ago is that human beings don't really have an immune response to it. In Jesse's case there was a massive immune response. But that doesn't mean there wasn't a reaction here. I don't know enough about it.

I have had experience with Targeted Genetics and H. Stewart Parker, their CEO. I met her in Washington, D.C., maybe five months after Jesse's death, at a Senate meeting looking into what had happened. This woman was very apologetic to me. She offered her condolences three times for Jesse's death. She was representing the Biotechnology Industry Organization at the time, not necessarily her company. Why, I wondered, was she being so apologetic?

In her testimony, she lambasted the researchers. She said that if the investigators were able to substantiate that the researchers had done wrong, they should be punished to full extent of the law. Turns out, five or six months later, her company bought Wilson's company. [Wilson is James Wilson, who led the trial that resulted in Jesse Gelsinger's death. The trial was conducted at the University of Pennsylvania, where
Wilson was a researcher, but he was also the founder of Genovo, the biotechnology company that developed the therapy. It was a gross conflict of interest.]

When that came out later, I said -- what is going on here. Talking out of both sides of their mouths. I found that I can't trust these people to tell the real truth. In our case, the real truth came out because of investigative reporting. The Washington Post dug deep, and put pressure on the FDA to put out the information. That's how the truth came out about Jesse.

When they looked into what happened to Jesse, they found out that the
FDA had failed. The researchers had been reporting adverse reactions to them, and the FDA did nothing to stop them from continuing. They dropped the ball. When you look at the record of how Penn reported, it wasn't adequate enough -- but it was sufficient to take a serious look at what was going on and to stop the clinical trial. There were adverse reactions and it did't stop. That's what led to Jesse's death: they kept escalating the dose.

Is that kind of stuff still going on? I'd be surprised if it was -- but then again, I wouldn't be surprised if it was.

It's important to note that the recent death could have been coincidental. Targeted Genetics and the FDA have released no details beyond the fact of the death and their concern about its chronological closeness to an injection of the therapeutic virus.

For more on the virus, known as an AAV, read this Seattle Times
article. It explains why gene therapists like to use AAVs -- they typically produce a mild immune response -- and mentions that an article published last Friday in Science described "how mice injected with a type of adeno-associated virus developed cancer."

Without more details, it's impossible to connect the death to these findings. However, that the paper's author said the cancer is
"presumably a phenomenon unrelated to the Targeted Genetics case and it has only been shown to occur in mice" is not reassuring.* *

Death clouds a gene-therapy's future [Seattle Times]

AAV Vector Integration Sites in Mouse Hepatocellular Carcinoma [Science]
*
*Portrait of Jesse Gelsinger from Jesse-Gelsinger.com. Wired covered the tragedy and the clinical trial reform it fueled.