Voices from award winners 1

We have now started to see light about the feasibility of radical therapies for leukemia, and also for the eradication of leukemia using prophylactic vaccination. Keep your hopes up and keep on fighting against the disease.

Dr. Haruo Sugiyama, Professor of Graduate School of Medicine/Faculty of Medicine, Osaka University

Voices from award winners

*Dr. Haruo Sugiyama won the Takashi Ogimura Special Award of the Japan Leukemia Research Fund in Heisei 8 (1996) with the research theme, "Establishment of a detection method of minimal residual leukemia using WT1 assay and the development of new treatment for leukemia using this method." Here is Dr. Sugiyama's insight into the concrete outcomes of the research.

- Dr. Sugiyama explained the theme of the award-winning research.

"I noted that a type of protein called WT1 frequently develops inside leukemia cells, and in 1992, prior to other researchers, I found a method to detect the onset of leukemia relapse at an early stage, by quickly detecting leukemia cells that remain active even after undergoing leukemia treatment using WT1 as the marker. However, a number of articles challenged my theory, which was not easily accepted in the beginning. Even under such circumstances, I believed that vaccination of patients with the WT1 protein would help to cure leukemia, so I prepared my research proposal to apply for the Japan Leukemia Research Fund."
"Although a number of doctors were skeptical about my theory back then, doctors from the management committee of Japan Leukemia Research Fund somehow decided to trust me. I am so grateful for that. The research subsidy I received then has given me the opportunity to speak at international academic conferences, etc. Thanks to those opportunities, I have come to be trusted by medical practitioners across the world, and decided to launch the WT1 International Conference in 2004, which in turn gave me the chance to get to know co-researchers who have the same aspiration all over the world."

- How much of the objective of your award-winning research been accomplished so far?

"The detection method of minimal residual disease in leukemia using WT1 has already been launched on the market by major pharmaceutical companies, and is also covered by health insurance. The method is also disseminating across western nations, and is regarded as a significant guideline to determine the direction of treatment."

- Dr. Sugiyama showed us some pictures of the appearance of erythema on the skin similar to a tuberculin test. According to Dr. Sugiyama, the more intense the erythema that appears on the patient's skin, the earlier the treatment will be effective in the patient.

"In leukemia treatment using WT1, only the portion of peptides that can be recognized by the human immune system is extracted from proteins called WT1, and is subcutaneously injected to patients. The WT1 will be detected in the patient's body, and the number of killer cells will then increase and start attacking the leukemia cells with WT1 on the surface, leading to the elimination of the leukemia cells from the body. This is WT1 peptide vaccination therapy."

Currently, major pharmaceutical companies are proceeding with clinical trials to commercialize this therapy in Japan, the USA, and other Asian nations.
- What is the rationale why you think that the therapy could be a radical treatment for leukemia?

"Research of the WT1 peptide vaccination therapy has come to show effectiveness to prevent leukemia from recurring in patients." Cancer research has shown that leukemia stem cells that can trigger the disease are also present in leukemia. Eradicating all of these leukemia stem cells is difficult with conventional treatments, thus the recurrence of leukemia cannot be avoided. The leukemia stem cells carry the WT1 peptide on the surface, and can be eliminated by the WT peptide vaccination therapy. That is to say, eradicating the root of the disease is likely to be feasible."

- That is an excellent treatment procedure, but are there any adverse drug reactions associated with the therapy?

"The main purpose of this therapy is to improve immunity, and severe adverse drug reactions have not been reported yet. It seems that the effectiveness of the therapy does not deteriorate even when used concurrently with other anticancer drugs." "Nevertheless, it seems that the effectiveness varies from person to person. The wide range of different degrees of effectiveness is apparently due to the different amounts of immune strength of each individual. For example, it is now clear that the outcome of patients will improve dramatically if the WT1 peptide vaccination therapy is performed in patients following bone-marrow transplant."

- What direction do you think the research will take from now on?

"In the initial phase of the research, I tried to perform WT1 peptide vaccination therapy in patients with refractory disease." While the patients were receiving the therapy, their condition did not deteriorate. Therefore, I gradually started to implement the therapy at earlier stages of the treatment. When the therapy was implemented at early stages, the effectiveness became more apparent. I think that the time will surely come when WT1 peptide vaccines are administered as soon as patients are diagnosed with leukemia." "Not only leukemia cells but most cancer cells have WT1 on the surface. The effectiveness of WT1 peptide vaccination therapy has also started to be witnessed."
"I think all citizens should receive WT1 peptide vaccine as prophylactic immunity against cancer on their birthday at a certain age in the future. When this kind of system is established, it will no longer be a dream to eradicate leukemia or cancer but a feasible possibility, and we probably will be able to prolong the human life-span."

- Comments to patients as well as ordinary citizens

"Medical treatments are advancing rapidly. Wonder drugs will definitely be found. We researchers are also doing the best we can, so please keep up your hope." "I would like to ask as many citizens as possible to help Japan Leukemia Research Fund make a contribution to realize the hope of each patient as soon as possible."

- There must be various ways to realize the dream of eradicating leukemia, but through this interview, I have realized that this is not only a dream but an actual issue to be solved.
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