Testimony of Donald L. Morton at the Committe on Foreign Relations

Committee on Foreign Relations, Subcommittee on Western Hemisphere, Peace Corps, and Narcotics Affairs
June 19, 2002

Thank you for that kind introduction, Senator. I appreciate the opportunity to appear before this committee and provide comments and perspective on the topic of advances in medical research in Cuba. As a cancer surgeon and cancer immunology researcher, as well as a melanoma survivor, I have tremendous personal and professional interest in seeing that potential advances in the treatment of cancer from researchers located throughout the global community, including Cuba, be made available for the benefit of cancer patients in the U.S. and elsewhere. Politics should not get in the way of our being able to take full advantage of life-saving treatments- whatever the origin.

As Senator Dodd mentioned, my work in the field of cancer and immunology began over 40 years and 600 scientific articles ago. Ever since I became a physician, I've studied how the immune system can be stimulated to fight cancer. From my early work at the National Institute of Health, to UCLA, and now at the John Wayne Cancer Institute, my focus on cancer has been clear: trying to understand what happens to the immune system during cancer progression and to develop a rational basis for cancer immunotherapy with the goal of impacting the course of the disease to help patients live longer.

I have dedicated my career to advancing research and development of promising technologies, such as vaccines, for the treatment of cancer. Ultimately, I believe this will lead to breakthroughs in cancer treatments and potentially provide cures. Today, we appear to be on the threshold of translating cancer immunology research into real treatments for difficult cancers. With over half a million people dying of cancer every year in the US, at a public health cost of more than $100 billion, the search for treatments and cures must continue to be purposeful - and it must be relentless.

No doubt, cancer has touched the lives of everyone in this room today in one form or another, and I know that this distinguished group is working hard to promote the advancement of cancer research and treatment. Among a long list, I am aware that Senator Helms has jointly sponsored a bill to require health plans to cover the cost of colonoscopies for persons older than 50 to improve early detection of colorectal cancer; Senator Boxer is a strong supporter of the U.S. Department of Defense Breast Cancer Research Program, including funding for the Lawrence-Livermore labs to use laser expertise for breast cancer research. This kind of public policy in support of advancing research targeted at early diagnosis and treatment is crucial in our effort to conquer cancer. Today, we need your support more than ever.

I currently lead the John Wayne Cancer Institute as the Medical Director and Surgeon-in-Chief. I had the privilege of caring for John Wayne himself during his battle with cancer. JWCI has emerged as one of the world's leading cancer research centers, thanks to the support of the National Cancer Institute and many generous private donors. Each year, patients travel from more than 40 foreign countries and 48 states to receive treatment from John Wayne physicians, many of whom are listed among the world's top cancer specialists. The John Wayne Cancer Institute currently sponsors the third largest surgical oncology fellowship training program in the U.S. In terms of the amounts of peer-review funding, the JWCI is in the top 6% of the 2500 research centers receiving NIH funding. More cancer patients have received cancer vaccines on research protocols at the JWCI than any other research center in the world.

My own research efforts are currently focused on the development and improvement of therapeutic cancer vaccines, which are designed to stimulate the immune system to fight cancer. As a result of over thirty years of research, a vaccine I developed for the treatment of advanced stage melanoma, a rapidly progressing and deadly form of cancer, is being studied in large phase III pivotal international clinical trials, the final step before FDA approval. I serve on the board of directors and as a consultant to a biotechnology company (in which I have a financial interest) that is working to finalize the development of this vaccine and obtain regulatory approvals around the world to make the vaccine available to patients.

While on a day-today basis I am a surgeon, I strongly believe in the importance of basic research, and that's what brings me here today. I recently returned from a licensed scientific exchange to Cuba - my first visit there, and with all proper clearance received from the Treasury Department. I have also had the good fortune of having a Cuban research fellow visit the John Wayne Cancer Institute, during which time we gained knowledge of Cuban scientific research efforts. As well, during this trip, I engaged in a rewarding academic dialogue with Cuban scientists. While I've known about the quality of their work through published articles for some time, I was impressed by the depth and sophistication of their research, especially as it relates to cancer immunology.

I learned that the Cuban researchers have made some interesting and novel discoveries and have achieved significant progress in the development of cancer immunotherapies. Many of these discoveries are protected by patents, which have been issued in the United States, Europe and other countries. In addition, several of the immunotherapy products that they have developed are being tested in clinical trials in the United Kingdom and Canada.

One type of immunotherapy product being developed by the Cubans is based on monoclonal antibodies, which are immune system-derived proteins of exceptional purity and specificity that are being used to target cancer like "smart bombs". This is an area of significant interest for cancer researchers. The four monoclonal antibody products that have been approved in the US for the treatment of certain types of cancer have demonstrated improved efficacy with reduced side effects compared to previous standards of care.

Cuban researchers have also focused on developing therapeutic cancer vaccines that are designed to stimulate the immune system to develop antibodies that target epidermal growth factor receptors (EGFR) and gangliosides, molecules that are present on the surface of certain tumor cells. The vaccines being developed by the Cuban researchers have shown promise in the treatment of head and neck cancer, lung cancer, renal cell cancer, breast cancer, and melanoma, among others, all cancers with a poor prognosis for survival.

I left there very impressed with the quality of basic research with these technologies. I also left there very frustrated. The truth of the matter is that, while the research results that I have seen from the Cuban research institutes are very promising, unless we are permitted to test and evaluate these approaches in the U.S., using the rigorous standards for medical research that are customary in the US, we won't know how effective they may be in treating cancer. And, importantly, unless subjected to the development approval processes required by the US Food and Drug Administration, any products that emerge from the Cuban research will not be made available to cancer patients in the US.

To further underscore the potential merit of medical research being conducted in Cuba, it is my understanding that a vaccine that was developed by Cuban researchers for the prevention of meningitis, an infectious disease that causes substantial mortality in children, is currently being tested in international clinical trials. Further, I understand that the US Department of Treasury has given approval for a US company to participate in the development of this important vaccine. However, I also understand that it took two years for the approval to be forthcoming and it is my hope that these kinds of delays can be eliminated by Congressional action where life-saving vaccines are at stake. This is a great example of a decision in which the health and well-being of our children was made the highest priority.

Based upon my many years of experience in this field, I can assure you that it is tremendously difficult to develop therapies that can directly affect solid tumor cancers - and the Cuban scientists appear to have made important advances in precisely this area. I believe that all US citizens suffering from cancer deserve access to potentially life-saving therapies, and that such therapies should undergo the rigorous development and approval process of the US Food and Drug Administration.

Senators, I urge you to take the appropriate legislative steps to help us to thoroughly evaluate every possible option to save the lives of US citizens afflicted with cancer. US clinical oncologists should be given access to the broadest possible armamentarium of options to treat cancer, and US cancer patients deserve nothing less than the very best therapies available in the world.

Thank you all for all your continuing support of cancer research. Let us not stop short of our goal by restricting the evaluation of medical advances that have the potential to save lives. Senators, create this bridge for the benefit of US cancer patients, their families and friends. This is more than good public policy; it's the right thing to do for US patients desperate for new cancer treatments.