Passing the Torch: With the Lifting of the FDA Hold and the Recent NIH Award, the Re-Energized Gene Therapy Program Is Safely on Course under the Leadership of Dr. Isner' S Longtime Research Collaborator and Friend, Dr. Douglas Losordo
Perry, Patrick, The Saturday Evening Post
The Post interviewed Dr. Doug Losordo, associate professor of medicine at Tufts University School of Medicine, who was recently named successor to Dr. Isner as director of the St. Elizabeth's Medical Center's Gene Therapy Program in Boston. Dr. Losordo worked with Dr. Isner for the past 14 years and shares his longtime friend and colleague's commitment to his patients, medical research, and the gene therapy program.
Perry: How do you feel about continuing the work on the gene therapy research at St. Elizabeth's after the untimely death of Dr. Jeff Isner?
Losordo: I have been appointed to take over cardiovascular research here. Obviously, there is nothing sweet about it. The only positive thing about it is that it is a great honor, but it is with great sadness that I accept the position. Nevertheless, it is also with great resolve to see Dr. Isner's work continue.
Perry: Can you tell us about the NIH-funded new study?
Losordo: There are three clinical projects in the program project grant. One is investigating the use of VEGF gene transfer to treat diabetic peripheral neuropathy. The basis for that is the pioneering work that Jeff and Dr. Peter Schratzberger did in animal models, which revealed a rather striking improvement in diabetic neuropathy after VEGF gene transfer. That is now being used in an attempt to treat what is largely an untreatable condition for diabetics. Given the scope of the problem of diabetes, you can imagine that could potentially reach a large number of patients. That project is in the planning stages, working with the FDA and coming up with a protocol that is acceptable. The NIH obviously thought it was a worthwhile endeavor, having awarded Jeff a $10 million grant.
Perry: We learned that the NIH grant is for two coronary gene protocols and one peripheral protocol. Is the $10 million grant for all three programs?
Losordo: The grant is actually for a total of four. The other is a basic science program.
Perry: And the coronary gene therapy program will then resume?
Losordo: The coronary gene therapy program will resume in a number of iterations or programs. There will be 12 sites in this multicenter trial which should be the pivotal or definitive trial of gene transfer for myocardial angiogenesis. It will mirror essentially the small pilot study that was completed and for which we are presenting the data in Anaheim at the American Heart Association meeting. Despite the fact that it was a very small study of only 19 patients, it did reveal evidence for a significant benefit of treatment in a double-blinded, randomized, controlled study. The ability to show a statistically significant improvement in a study that is so small is really pretty striking and may be an indication of the fact that this therapy really does have the potential to make people a lot better who previously had no other options available.
Perry: It is hard for many lay people who read about the remarkable recovery of most of your patients to view these stories as anecdotal evidence of the ability of coronary gene therapy to work at restoring blood flow to the heart muscle.
Losordo: The patients certainly don't consider themselves anecdotal, and we don't, either. There was a lot of skepticism about what we were claiming to see when all patients were being treated. Because there was no placebo group, people were suspicious that a lot of the clinical benefit that we were observing was a placebo effect. Of course, we saw objective evidence of improved perfusion in addition to the subjective improvement that patients claimed. We aren't as suspicious. We believed our clinical instincts and believed that the patients were actually getting better. It is certainly scientifically correct to want to do things in a double-blinded study at some point, and that is where we are heading to answer those questions.
Perry: Will you be enrolling patients in this study? …