ABBEY MEYERS

	Abbey Meyers, president of the National Organization for Rare Disorders, talks about the FDA and Jesse Gelsinger's lasting legacy.

Q. Did the FDA make a mistake in approving the protocol? A. The FDA and/or the investigatror should have gone back to the RAC and said we really believe that it should be approved by allowing the genes to be injected into the liver. They should have come back to the RAC and discussed it with them. Instead, they didn't. The FDA made its own decision without using the expertise of the scientists who were members of the RAC, and their expertise was something that should have been appreciated and not ignored. Q. The FDA nonetheless believes that it did the right thing by approving the protocol with respect to the injection in the hepatic artery. And that at least insofar as the degree of compliance that the trial had with that part of the protocol, that there was not a problem. A. I'm not the scientist and so I can't say whether they were right or wrong. I can look at it the way a consumer would, which is number one, there were rules. Did everybody obey the rules? No. The rules said that they should have come back and reported to the RAC, told the RAC that they were going to approve the protocol in a way that the RAC had specifically told them not to approve. And number two, was complete information about all the dangers in the informed consent document that was given to the patients? Were they told the truth? In the years that I sat on the RAC, I would see these documents time after time, sometimes eight or ten of them would come in front of us at a meeting, and I saw lies, I saw omissions, I saw exaggerations. Patients were not being told the truth in the informed consent documents. Q. What were they being led to believe? A. they were being led to believe that gene therapy was definitely therapeutic, that it would help them in many instances, whereas none of the experiments in what we call phase one, the very first experiment, were intended to be therapeutic. There was just more safety. They were being told that there were no dangers, that other people had been - gone through this experiment before, and that there's never really been serious side effects from gene therapy. We knew that that was not true. And as we would review the informed consent documents, bioethesis and I would demand that the investigator change the informed consent document to tell the truth, to not omit the facts about - that there was absolutely no chance that it would be therapeutic, and that there were dangers, and that there were even unknown dangers, and possibly then, and we would ask them to change it voluntarily. And many times the investigator would say I can't change the informed consent document. I have no power not to change it. It's the institution review board at my institution who says I must say those things. And then we would ask the FDA, when this protocol comes before you, will you make the investigator change the informed consent document. And the FDA would say I don't have the authority to force a change in the informed consent document. Everybody would side-step the issue.

Jesse Gelsinger's lasting legacy

Q. What should be Jesse Gelsinger's lasting legacy on the field now of gene therapy research? Should it proceed but with a greater sense of caution than before, with more openness than before? A. It should absolutely proceed. The one thing that Jesse's death should not do is stop gene therapy research. That would be the worst outcome, and that's what the RAC has to keep in mind more than anything else, is that gene therapy research must go forward. It cannot be delayed. It's just too important. It's going to work. We know it's going to work. The scientists just have to find the right answer. And so if there's anything that it's done, we're going to look back I think 20 years from now at this incident and we're going to say that really turned things around. I think that there's very important things that are going to come out of this, and that is more of an understanding by the public that they have the right to know, and that sometimes when they're not given the right information in the local newspapers, or on the 30 seconds on the nightly news, that they really need to look into some of these questions. They really need to get all the information. Don't go out there and make your family planning decisions based on a newspaper article that says that hemophilia or cystic fibrosis is going to be cured in 20 years with gene therapy. Get on the Web and look at what's been published on gene therapy and try to understand it. I think and I hope that what happened to Mr. Gelsinger is something that is going to help all of science because it's not just gene therapy that is affected by the secrecy. There are many other areas of science that need this type of information. And we need access. We as consumers need access to information.