UPENN Gene Therapy

The University of Pennsylvania in Philadelphia has developed a new "gene therapy" protocol that is designed to weaken the tumor cells. In the first phase, the patient takes biopsy samples from the lung and places a tube into the chest cavity near the tumors. The next step is where the actual treatment begins. A human gene in the form of a crippled cold virus called an adenovirus is injected into the pleural space via a chest tube. Once this serum is inside the lung, it is sloshed around, coating the tumors. The genetic serum triggers the production of the enzyme thymidine kinase, which is harmless to cells by itself. The final phase involves the use of ganciclovir several days later. Ganciclovir attacks only the "bad cells". The ganciclovir is turned into a poison by the enzyme, causing the tumor cell to self destruct. The mesothelioma cells divide three to four times faster than regular cells, making them especially vulnerable. The gene therapy treatment is designed to target and attack only the tumor, not the body's healthy cells. Preliminary results from animal studies are encouraging. To be eligible, patients must not have had any chemotherapy, surgery or radiation. There is no age cut-off and the treatment is free.

A Conversation Between Roger G. Worthington and
Dr. Daniel Sterman - August 20th, 1996
Regarding: The ongoing clinical trials at the University of Pennsylvania

Dr. Sterman, if you don't mind, I have a few questions for you about the gene therapy program. I have referred several clients with mesothelioma to the UPENN study. A few have been accepted. One, Mr. Bill Giblin, was originally accepted but died before he could take advantage. Is UPENN limiting the enrollment to 1-2 patients per month? If not, what is the monthly limit, if any?
We are limited by the FDA in treating 2 patients every 5-6 weeks and have had a 4-5 patient waiting list at any one time. The combination leads to an unavoidable 2-3 month delay between the time of acceptance into the trial and actual undertaking of the gene delivery experiment. It is quite unfortunate, but we have had several patients like Bill Giblin die while waiting for treatment. While this may seem cruel, it is beyond our control as this is a strictly regulated and highly scrutinized Phase I clinical trial. In addition, the truth of the matter is that our patients have tolerated the treatment quite well, but have not had any dramatic clinical responses or even impressive transfer of the suicide gene to the cancer cells. We are working out the bugs in the laboratory as fast as we we are able.

I was speaking the other day to a client who said he spoke to a 38 year old carpenter from Texas with mesothelioma in your waiting room. The latter was greatly concerned that his insurance company would not pay for your treatment. I know that every health insurance carrier is different, and this is certainly not your area of chief concern or interest. Do you have any feel for whether the costs of the UPENN protocol have been covered by any insurance carrier for any of your mesothelioma patients?
Our clinical trial has been estimated to cost $150,000.00 per patient of which approximately $145,000.00 is provided from Federal grants, the General Clinical Research Center funds, and the remainder generously donated by the University of Pennsylvania Medical Center. The remaining $5,000.00, which relates to the initial video thoracoscopy and outpatient pre-op evaluation, is billed to the patients insurance company, if possible. No insurance company has paid for the entire cost of the trial.

Again, we receive several calls a month from mesothelioma patients and I like to keep them current on what their treatment options are. We have included all of your articles in our information packet. We would appreciate your help on these matters.
We appreciate all of your referrals and will be more than happy to update you on the trial's progress. By the way, we will be hosting the biennial meeting of the International Mesothelioma Interest Group (IMIG-the international organization of physicians and scientists studying mesothelioma) in Philadelphia next May. You and/or some of your clients might be interested in attending. More details to follow. Thanks for the inquiry. I would happy to answer any additional questions that you might have.

Dr. Sterman can be reached at:

Center for Lung Cancer and Related Disorders
University of Pennsylvania Medical Center
3400 Spruce Street
Philadelphia, PA 19104
(215) 349-5613
(215) 349-5172 fax
sterman@mail.med.upenn.edu