Gene Therapy Death in the News. Should We Be Alarmed?

Some sad news hit this week with the revelation that a patient died during a gene therapy trial. Should we be alarmed about this in the context of the upcoming gene therapy trials for thalassemia? My answer is no. let's look at some facts.

From http://www.washingtonpost.com/wp-dyn/content/article/2007/07/27/AR2007072702242.html

A gene therapy experiment that has triggered a federal investigation after the death of a patient on Tuesday raised a variety of concerns when it was first proposed to federal reviewers in 2003. Unlike the vast majority of such proposals, all of which aim to treat diseases by giving patients new genes, the plan to inject trillions of genetically engineered viruses into the joints of patients with arthritis was flagged for a special public review by the federal Recombinant DNA Advisory Committee, part of the National Institutes of Health.

At that Sept. 17, 2003, meeting, representatives of the sponsoring company, Targeted Genetics Corp. of Seattle, listened as a panel of experts wondered aloud why such a novel and possibly risky approach was to be offered to patients who were not especially ill, including some who had not even tried standard treatments.

Reviewers questioned the justification for the study, given that animal studies had found only a "limited correlation" between the treatment and any improvements in subjects' condition. And they asked for more assurance that the engineered viruses were not going to spread around the body or cause untoward immune system reactions in patients.Some also expressed concern that the informed consent document the researchers planned to use to describe the risks and benefits to participating patients was not upfront enough about the fact that the study was unlikely to help them and was designed merely to test the new approach's safety.

I also heard from Pat Girondi, CEO of Errant Gene Therapeutics, LLC™, concerning the death in this trial. He directed me to his response at 

http://blog.myspace.com/index.cfm?fuseaction=blog.view&friendID=148067567&blogID=292433562

After consulting our own gene therapy head and a European gene specialist I would like to convey the facts investigated.

More than 600 clinical trials for experimental gene therapies are ongoing or about to start in the United States. The diseases targeted range from cancer to blindness to heart failure.

The death of the patient in the AAV, Targeted Genetics Clinical Trial possibly had nothing to do with gene therapy.

If in fact the death is linked to gene therapy it is important to remember that EGT's Thalagen employs the lentiviral vector and not the AAV. Virxsys is almost 3 years into their trial with no incidents using a lentiviral vector. Vectors and Lentiviral vectors have been in 1,000's of patients to date...This unfortunate occurence should not be an issue. Thalagen trials should begin within 10 months.   "Gene Therapy is here to stay and will eventually better the lives of millions."

Thanks for the response Pat. I would like to add that there are always some setbacks along the way and also we need to remember that some of these therapies will have some inherent problems. I feel that the thal trials are in good hands and that the technology being used has worked well in animals and has tremendous potential for success.

Andy:
You are SO knowledgeable.  Where do you find so much info about Thal?  I try to be so proactive to help our little girl, but I am amazed at how much information you gather.
Thanks for doing so.

Hi Rebecca,

Most of what I reference about thal is information I have learned online. There is a vast wealth of information online and so many medical studies are published online. It's mostly a matter of finding the information and understanding it and its relevance to specific topics, and often filing things away that are found while searching for something else, until that information becomes needed.

I'm quite appreciative to have found something that stimulates my mind and also is of benefit to others. After so many years of the monotonous routines that running a retail store entails, it is a pleasure to be involved in something that is very challenging and also allows me to meet so many interesting people, both online and in person. The internet has changed the world and its potential to connect people with information and each other is immense. It may seem that I know a lot but most of it is a direct result of being connected to people (through this group), who ask the questions that lead to me connecting people with information. Without the members asking the questions, I wouldn't even be aware of most of what thal involves. So, thank you too. All of you. I'm just glad that what I do helps.