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Alert Number 242

Gene Therapies

Date: August 8, 2007

Gene Therapy.

Just the term conjures up visions of high tech medicine — Beverly Crusher of Star Trek Enterprise waving a medical tricorder over patients and curing them with a single (painless) shot of potent medicine that re-writes and corrects what ails them at the genetic level. How can it be anything but helpful, correcting the very mistakes in the genes that make us sick? How can it possibly be dangerous? It all sounds just a tad too good to be true, but patients who are sick to death of life-threatening diseases are often willing to suspend disbelief. We sell ourselves a bill of goods, more often than we are mislead.

Gene therapies of various sorts have been around for more than a decade. Not a single one of them has made it into mainstream medicine, each and every one of them is still experimental. I like to think the reason for this is actually very simple. We are still far from understanding the mind-bendingly complex interactions of the human body, the subtle feedback loops and push-pull controls, and multiple pathways that can short circuit any single road block we dare throw up. We are beginning to crack open the secrets, a little at a time, through the process of lessons learned painfully, often at the expense of patients who participate in early phase clinical trials. Some day, but not today, we will actually be able to re-write the disease causing genetic errors by means of “gene therapy”. Some day, but not today, we will know how to do it so that we do not cause more harm in the process of trying to cure.

The article below is not about CLL. But it underlines some of the risks associated with clinical trials, risks that I am not sure patients are aware of as they sign on the dotted line. For example, the patient consent form in the case below said the virus used in this particular experiment was unable to reproduce, and therefore posed little risk. I have heard that reassurance given often to patients. But as you can see from this case history, the mere fact the virus was not able to reproduce is insufficient guarantee, at least in this case. This is a report in the popular press, with some of the extra emphasis on tugging at your heartstrings. Nevertheless, there are important lessons here.

Washington Post Article

Most early phase trials have the language buried someplace in the patient consent form that says the experimental therapy used in the trial is not expected to be of therapeutic value to the patient. In other words, the patient is being encouraged to participate in the clinical trial in order to further our scientific understanding, to help create better therapies for future generations of patients. Nothing wrong with that! I have great admiration for the courage and generosity of patients who participate in early phase clinical trials for that reason. I just wonder how many patients kid themselves, sometimes with the help of a wink and a nod from the guy in the white coat, into thinking that the caveats are a lot of legal b.s that the researcher is forced to include – and that the experimental therapy will actually of benefit to them personally. I know of several CLL patients who were convinced the gene therapy clinical trials (phase 1 and 2) at UCSD were going to be just the ticket for them. Suffice to say, it has not cured any CLL patients yet, and I am not aware of any large scale and late stage trials well on the way to getting FDA approval in the near future.

Opportunity cost is something to consider, when you are making therapy decisions. Time is a precious commodity, especially for cancer patients with a clock ticking ever louder. Is there a cost in not treating your CLL with effective therapy when you need it, taking time out for a dog leg of ineffective therapy while the CLL continues to grow? You bet there is!

I am not suggesting you stay away from clinical trial participation. Quite the contrary. I think it is extremely important that our patients participate in well conducted clinical trials, our very lives depend on the information gathered from such trials. All I am saying is that patients need to be clear about the nature of the trial, the risks and rewards involved, and their own motivation for participation. Absolutely no one should sign up for a clinical trial because of high pressure sales tactics from individuals who may have financial stake in the investigational drug or therapy. Be sure you take the time to think it through, take the paperwork home and consult with your advisors and physicians who may be able to walk you through the complex jargon. We live in a complex world of conflicting agendas, and it is not always easy to sort things out. Taking a little time out, thinking things through, asking questions and making sure you understand the answers – these are things that might keep us from making costly mistakes.

This is an editorial opinion. My two cents, as it were, free of charge.

Be well,



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