In a guest post earlier this week Alan Milstein, the attorney who represented Jesse Gelsinger’s family, wrote about his reaction to a recent editorial by James Wilson about gene therapy and informed consent in the journal Human Gene Therapy. The guest post prompted a number of comments, including one from Paul Gelsinger — Jesse’s father. Here’s Mr. Gelsinger’s comment in full:

To clear the air about what really happened, let me take you back nearly 9 years to the meeting we had with the principal investigator 3 months before Jesse’s actual participation in the “gene theray” clinical trial. Understanding the information regarding the gene therapy that Jesse was to receive wasn’t all that difficult for me, since I had extensive experience in science. The risks were fairly well spelled out. There was the possibility that he would have an immune response, that his liver could be seriously damaged, that a liver biopsy to be done a week after the vector infusion had the risk of serious side effects. When I questioned this investigator as to whether they had seen any ill effects, he stated that they had only had to deal with flu like symptoms. In further conversation, he remarked that the liver was a remarkable organ, the only organ in the body capable of regenerating itself. The risks were downplayed. There was no mention by the investigator or the consent form of any animals having died in the research.

A month later, a call from the expert on Jesse’s disorder, another of the principal investigators, led us to believe that the most recent patient had benefited from the vector infusion. He stated that she showed a 50% improvement in her ability to excrete ammonia following the gene therapy. When I said, “Wow, this really works”, he stated yes and that it would be for those for whom this therapy was designed, not kids like Jesse with mild otc, but newborns with the almost always fatal form. We totally dropped our guard and Jesse made arrangements to participate.

Three months ffter his death we discovered that there was never any efficacy, that the 50% improvement that the patient showed was not related, that the researchers had multiple toxicites at 1/10 the dose that Jesse received that were each a stop sign for the protocol (and the FDA knew of these), and that animals had died in the research. I had been very supportive of these men and their institution after his death, but with this new knowledge I was no long able to support them. Jesse had been doing very well (he was virtually normal except he had to take medication) on the standard drug therapy, and he only wanted to help those less fortunate than himself. You can not imagine how disillusioning it was to discover the lapses by the researchers and the government.

The financial conflicts of interest for both the sponsor (James Wilson) and the University were basically ignored by the University’s Conflict of Interest Committee. This led to a blindness on the part of all parties to the dangers they were seeing. These were not bad men doing evil. They were men blinded by ambition and greed. They have told me that they could not have foreseen Jesse’s death, yet their data was screaming at them to stop. We trusted a system that was untrustworthy, one that didn’t even pay attention to its own stops. So we sued and settled within 6 weeks. The quickness of that should tell how badly the other side wanted this to go away.

The government in the form of the FDA criminal division and the Department of Justice did a 5 year investigation at the end of which they fined the institutions involved a combined total of a little over a million dollars. They decided not to press any criminal charges and slapped the docs on the wrist with retraining and supervision. Wilson was not allowed to be a sponsor for 5 years and could only be involved with one trail at a time as an investigator for a specified period. The other docs were permitted to go about their business. We didn’t support the settlement… all we ever really wanted was an apology that wouldn’t be granted. As part of the deal, Wilson was to write a paper on “Lessons Learned” with my family having space in the same journal for a say on how we felt. It is now 3 years after the settlement and no “Lesson’s Learned” paper has been issued but he is editorialing on the issues surrounding what he did. Repeated efforts by my lawyer, Alan Milstein, and myself to get the DOJ to follow up have basically gone nowhere.

So, my son, doing the right thing, was killed by a sytem and people rife with conflicts of interest, and real justice has been found to be very lax. It’s essentially business as usual. You may think that I am bitter, but I am not. My son gave me the best possible example on how to be. The system showed me what everything is really all about. Hopefully, given enough time they’ll fix this, but I’m not holding my breath. Anyone considering joining a clinical trial needs to be aware that they are dealing with a system that is seriously flawed.