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Provenge

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"On Prostate Cancer Immunotherapy and Recent Decisions by the FDA,"  May 2007  By George Cooper, MD

Let me preface this by saying that while I am a physician-scientist who has prostate cancer, such that I have a personal stake in the outcome of our approach to the development and approval process for new drugs, I have no financial relationship of any kind with any pharmaceutical or regulatory concern.

 That said, some investigators, physicians, and regulatory personnel who are tightly focused on the value of clinical trials, and this is a very important focus for the identification and advancement of new therapies, insist that statistical criteria for effectiveness be met with rigid exactitude before a drug is released by the FDA. One justification for this approach is the recent example of the small but significant risk for serious side effects of agents being used to treat diseases that are not inherently lethal, such as the usage of certain COX-2 inhibitors in treating osteoarthritis.

 The other side of this approach when it is applied simplistically and universally, however, is that it denies safe drugs which have reasonable data showing some degree of effectiveness to patients and family members, including children, living with the agony of end-stage malignant disease.

 Denial of approval for Provenge is a classic example of this problem, where an agency that was established to protect the public from dangerous and/or useless substances is instead taking on the role of dictating to dying patients and their families what they will and especially will not be given access to solely on the basis of cold statistical fine points, even when one is dealing with advanced malignancies wherein the lethal clinical outcome of placebo treatment in clinical trials or of standard therapy is known with near certainty. This is simply cruel, and as a physician I think that it is insupportable.

 The second problem with this overstepping of regulatory bounds, and this is very much more important in a practical sense, is that the impact of this decision is not confined to the single agent in question. Rather, capital and human resources that were rapidly gravitating to the field of cancer immunotherapy are now just as rapidly leaving. This is occurring at what was going to be the takeoff point for a field that has been gestating for decades and is only now showing the first signs of realistic promise in terms of effective therapies.

 No one in their right mind would think that Provenge was to be the sole such agent (and there are many others in development which will now inevitably have problems generating necessary funding and may well disappear); rather, it would begin a process that would hopefully, especially when used in patients with lesser tumor burdens in whom immunotherapy is most likely to be effective, provide the visibility and thus the impetus for the whole field to develop.

 After all, the data for the effectiveness of Provenge are hardly dramatic, but it is a start, it is an apparently safe start, and its wider usage in various stages of prostate cancer should provide information that would help in designing the next generation of more effective immunotherapeutic agents. As an analogy, penicillin was neither the only nor eventually the most effective antibiotic, but it was the foot in the door which provided the impetus for a new paradigm in terms of the antibiotic revolution for the effective treatment of infectious diseases. Thus, the present crushing of the prospects for this very promising new field of cancer therapeutics is the real long-term tragedy of this very poorly thought out, shortsighted decision.

 I do hope that wisdom and compassion emerge from the FDA. No one on either side of this debate is ill intended, but we really do need to consider together who we wish to be as a people and as a community.

 George Cooper, IV, M.D.

Distinguished University Professor
Medical University of South Carolina
Professor of Medicine, Pediatrics & Physiology
Director, Gazes Cardiac Research Institute
Chief of Cardiology

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