Key person interview
I want to expand through joint research
Head of Advanced Medical Research Center, Kobe Medical Industry Development Organization
University of Tokyo Faculty of Pharmaceutical Sciences Molecular Oncology Specially Appointed Researcher
Professor Toshio Kitamura
Question 1. What is the content of the joint research between the Institute of Medical Science, the University of Tokyo and SymBio Pharmaceuticals?
SymBio Pharmaceuticals' bendamustine (generic name for TREAKISYM) and rigosertib, the mechanisms of action of these drugs are very interesting, and we would like to study them together. In my laboratory, we are currently creating many mouse models, mainly of hematologic tumors, and we can administer rigosertib to them TREAKISYM We are also conducting research on what kind of medicines should be combined to produce greater effects, such as decitabine (unapproved in Japan) *1 and azacitidine (Vidaza) *2.
*1: Drug for myelodysplastic syndrome (MDS)
*2: Drug for myelodysplastic syndrome (MDS) and acute myeloid leukemia
Question 2. Could you tell us about bendamustine (TREAKISYM), including its potential for new indications?
First, I would like to say a few words about bendamustine (TREAKISYM). For the past 40 to 50 years, the treatment of malignant lymphoma using bendamustine (TREAKISYM) relied on a treatment called "CHOP." I added rituximab to it, and I got a little better with a treatment called "R-CHOP". It is wonderful that a treatment called RB, in which bendamustine (TREAKISYM) is added instead of CHOP, has been developed and that it is superior to the old CHOP or rituximab + CHOP. . RB is now the first choice and is becoming more commonly used. The mechanism of action of bendamustine (TREAKISYM) is not fully understood, so if we thoroughly investigate it in the future, I think it may be possible to use it for other cancers, including solid tumors. . In our mouse model, we have confirmed that it is also effective against myeloid tumors.
Question 3. What can we expect from the social collaboration course "Molecular Oncology Pharmacy" supported by SymBio Pharmaceuticals?
I have been doing research for a long time, but my focus was on basic research, including while I was living in the United States. About 15 years ago, although it would be interesting to continue basic research, I was originally a hematologist, so I decided to return to my field and study hematopoietic malignancies.
Until now, my research on hematopoietic malignancies has focused mainly on why they become ill, but from now on, I will also conduct development research on therapeutic aspects. We have more experience in many fields than other groups, such as immunology, cell biology, signaling research, and developing new methodologies. I am proud that I can make use of these experiences and achievements to conduct research that cannot be done by groups that are researching hematopoietic malignancies.
In terms of how we can develop research on hematopoietic malignancies through joint research with SymBio Pharmaceuticals, we have recently been working to elucidate the mechanism of action of drugs such as decitabine, and to explore the potential for combination therapy. I am doing research for development. Decitabine has been approved overseas and is mainly used for MDS, but its mechanism of action is actually not well understood. Everyone is interested in what drugs should be combined. We are introducing new methodologies in this field to study which drugs are combined with which drugs to give very good results.
It is a little difficult to explain the details of the principle of the research method that we use, but in simple terms, we randomly increase or decrease the expression of various genes in a cell, administer the drug. By examining the cells that remain after administration, we can find out which genes are more likely to be resistant to the drug, and which genes are more likely to be resistant to the drug. . By investigating this, we can understand the mechanism of action of the drug, and at the same time, we can understand what drugs should be used in combination.
If you do some research with existing drugs, you may find that a really unexpected combination can cure a disease. In the future, I would like to conduct such experiments centering on SymBio Pharmaceuticals' drugs, rigosertib and bendamustine (TREAKISYM), develop combination therapies, and further expand the range of diseases for which drugs can be administered. increase.
Question 4. Please tell us your thoughts on the future of drug development in Japan.
I have always been apprehensive about the delay in drug approval in Japan, but I believe that the development of the combination therapy I mentioned earlier can contribute to this. This is a drug that has not yet been put on the market, and if a mouse model is used to properly investigate the effects of combination with various drugs, it will be possible to conduct clinical trials before a new drug is put on the market in comparison with drugs that have already been developed. It is also possible to conduct clinical trials by combining them, and I believe that such a method is one way to catch up with overseas speed. Accurate knowledge of the drug's mechanism of action is also important when conducting clinical trials of combination therapy.
Question 5. What do you hope to achieve through joint research with SymBio Pharmaceuticals?
I myself have about seven years of experience as a doctor, and I have seen many patients who have suffered but have not made much progress, or who have died. I would like to be able to deliver an effective treatment method with as few side effects as possible to such drug-refractory patients, and I would like to conduct basic and applied research for that purpose.
Question 6. What do you think of the significance and value of this joint research?
I have been doing basic research for a long time, and I wanted to take it in a direction that would be useful to people. At that venture, we were able to create an antibody that would shrink cancer, but we were unable to form an alliance with a company beyond that. I thought that in order to conduct truly useful research, it might be better to proceed with joint research with companies from the beginning. In that sense, it is wonderful for me to be able to conduct development research together with SymBio Pharmaceuticals, and I would like to contribute to research that is useful to people in the future.
Question 7. What are your thoughts on SymBio Pharmaceuticals' philosophy of "delivering medicines to patients Underserved Therapeutic Areas"?
The philosophy of creating drugs in Underserved Therapeutic Areas to benefit patients is written on the website, and I think it is very consistent with what I want to do now. . Niche areas tend to have a small number of patients, and are difficult for major pharmaceutical companies to enter. I think that SymBio Pharmaceuticals will surely grow in the future, but I think that the idea of covering such areas is wonderful, and I would like to help with that, so I would like to continue this joint research relationship as long as possible. I think
Question 8. How will you address patients who are waiting for new drugs in future research?
In this super-aging society, clones of hematopoietic cells with only one gene mutation related to leukemia are gradually established, and the number of people who are over 65 years old is 10 people. I recently found out that there is one person in Such people are about 10 times more likely to develop leukemia, but there are also many other people with cerebral infarction, myocardial infarction, and cancer. It is known to have a high recurrence rate and a poor prognosis.
Our experimental results suggest that clonal hematopoiesis accelerates systemic aging. We believe that clonal hematopoiesis is a major problem in this super-aging society, and one of our research themes is clonal hematopoiesis. In order to prevent people with clonal hematopoiesis from progressing to leukemia, cancer, or cerebral infarction, it is important to think about what kind of treatment should be given to those who have already developed the disease. There is also a possibility that it can be applied, so I would like to proceed with such research in the future. In the field of clonal hematopoiesis, I think it would be great if we could do something together with SymBio Pharmaceuticals.