Held the 36th JPMA Policy Seminar Aiming to Improve Japan's Drug Discovery Capability: What Value Innovative New Drugs Bring to Society

Accelerating Innovation Contributing to Global Health

The 36th JPMA Policy Seminar" was held on March 17, 2025. Japan has created many innovative new drugs and has made significant contributions to global healthcare. Now, there is concern that its drug discovery capability is declining. What is the value of innovative new drugs? And what is needed to accelerate drug discovery innovation for a bright future? In this policy seminar, many recommendations were made, including the use of artificial intelligence (AI), promotion of digital transformation (DX), streamlining of clinical trials and clinical studies, and establishment of a drug discovery ecosystem. The following is a transcript of the seminar.

The venue

Speech] Drug Discovery from the Perspective of Medical Professionals: Its Impact and Challenges

Currently, the treatment of rheumatoid arthritis is based on the order in which conventional drugs are used first, and if they are not effective, another new drug is used. Patients have to wait for the new drug, and doctors are not objective in judging the effectiveness of the drug based on the patient's opinion. For this reason, efforts are being made worldwide toward "personalized medicine," in which a patient's blood is examined and evaluated objectively from the beginning, and a drug can be selected to suit the individual.

Drug discovery has so far focused on animal models and limited blood samples, but in the future, the trend will be to find biomarkers from patients' biological samples using advanced analytical techniques of data science. This will require cooperation and interrelationships between the medical field, academia, pharmaceutical companies, and ventures. It will be important for diverse personnel with different expertise and information to gather face-to-face and share the same themes and issues.

In the joint research between Keio University and a major pharmaceutical company, our physicians and researchers from the pharmaceutical company shared the issues and discussed what patients are seeking. Through industry-academia collaboration, we established a unique drug discovery platform, which enabled us to carefully follow the data of each patient before and after standard treatment, and we were able to obtain a series of patents and publish a series of papers. It is now a promising seed.

It is difficult for medical professionals to deliver medicines to patients without teaming up with pharmaceutical companies that possess the appropriate technology. Furthermore, it is of utmost importance to gain the understanding of patients and society to achieve therapeutic goals.

Speech] Drug Discovery and DX in Japan

We are moving forward in the direction of actually realizing my drug discovery DX platform concept that has been in the works for more than a decade, and we have started with early-stage basic development. For example, by entering a disease name and patient sample data, a drug target search can be performed to estimate the disease mechanism and target protein. It is also possible to estimate lead compounds by entering the name of the target protein.

Various such technologies have already been developed around the world, but the most important factor for the accuracy of AI is data. We have succeeded in developing an AI with world-class accuracy by collecting data from 17 Japanese pharmaceutical companies. We have already introduced a data sharing mechanism to 16 companies using federated learning, a method that allows multiple organizations to collaborate in learning AI models without leaving the data at each company.

This AI developed by industry and academia is scheduled to be provided to more than a dozen pharmaceutical companies for a fee starting in FY2025. The time required for calculation is less than two days from the input of a patient sample to the estimation of the target protein. The design of a compound also takes about three days, and the refinement of the compound through simulation takes about three days, making a total of about 10 days from the receipt of a patient sample to the design of the compound.

AI drug discovery ventures in Europe and the U.S. are able to receive tens of billions of yen or more in investment, but it is difficult for Japanese pharmaceutical companies to develop AI on that scale on their own. We believe that if this DX platform can be used as a common foundation in Japan, the field will be revitalized.

Lecture] Drug Discovery from a Startup Perspective: Cancer Immunotherapy and iPS Cells

On the other hand, however, this treatment is custom-made, so it is time-consuming and costly. In addition, special instruments are required to collect lymphocytes, making it highly difficult. It is also known that in some patients, T lymphocytes are weakened by anticancer drugs and other treatments and cannot tolerate genetic modification.

To overcome these problems, we have been working on T lymphocyte regeneration using iPS cell technology for more than 10 years. Even weak T lymphocytes can be reproduced as healthy T lymphocytes by generating iPS cells with cancer-detecting information and increasing them outside the body. Furthermore, the supply of iPS cells is inexhaustible because iPS cells themselves can be grown outside the body.

However, since the time and cost required for cell creation could not be solved, a methodology was devised in which cancer-detecting T lymphocytes are prepared in advance from the institute's iPS cell bank and used in patients who are suitable for cancer treatment. In a joint study with a major pharmaceutical company, basic data was obtained that receptor-loaded T lymphocytes can control subcutaneous tumors through a mouse model of solid tumors, and preparations for clinical trials are currently underway.

In 2013, they launched a joint start-up company, and in 2011, they received investment from a U.S. venture capital (VC) firm to become a U.S.-registered company and expand into the United States. They are continuing their research to develop a treatment that can cover a large number of patients with a single type of iPS cell, as well as a treatment that fights cancer by regenerating their own lymphocytes in a completely individualized way, anywhere in the world. We are continuing our research to make this treatment available anywhere in the world.

Speech] The Future of Innovative Drug Discovery Achieved through Co-Creation with Patients

Promoting New Patient-Centered Clinical Trials

Muto: When we asked home care nurses to gather feedback from patients who use home care nursing in DCT, about 70% said they were glad they used it, and about 30% said they were rather glad they used it. Reduced time and physical burden, emotional relief, and reduced burden on family members were cited as advantages. There were also specific comments such as, "I was wondering whether to stop the clinical trial, but now I am positive about it," and "I feel like overcoming the disease," which also contributes to the improvement of patients' QOL (quality of life).

Speech] JPMA's Efforts to Strengthen Drug Discovery Capabilities

While small molecule drugs, in which Japan has excelled, can be discovered and commercialized by a relatively small number of players, biopharmaceuticals, which require a variety of drug discovery technologies, require the cooperation of multiple players. This is where the drug discovery ecosystem, which includes pharmaceutical companies, academia, startups and ventures, contract research organizations (CROs), and venture capital firms, comes into focus.

While a huge ecosystem has been formed in Europe and the U.S., such as the one in Boston, Japan's biotech clusters are scattered throughout the country and are small in scale, so it will be necessary to utilize AI and digital technologies to integrate their strengths.

In addition, the promotion of practical application of basic research by academia is also a key point for strengthening drug discovery capabilities. In order to cross this so-called "magic river," it is necessary to strengthen collaboration among the players in the drug discovery ecosystem and to establish the necessary facilities and institutional infrastructure, mainly by the national government. The knowledge and experience of the pharmaceutical industry will also be of great use.

The innovations created in this way will be evaluated appropriately, attracting new investment and creating the next innovative drug. Through this virtuous cycle, we hope to contribute to extending the healthy life expectancy of the Japanese people and to Japan's economic growth.

Panel Discussion

What is the value that innovative new drugs bring?

Moderator: Jun Ando, Editorial Writer, Nikkei Inc.

Panel discussion

Maintain drug discovery capabilities Support medical security

Ando: What are some of the innovative new drugs originating in Japan?

Takeuchi: For example, a humanized anti-IL-6 receptor antibody preparation approved for the treatment of rheumatoid arthritis. This is an innovative drug that originated in Japan, where the discovery of the receptor itself, the development of the antibody drug, and its clinical application were all led by Japan. It is so important that many medical treatments would not be possible without it.

Kaneko: Another example of a product from Japan is a regenerative medicine product using iPS cells. However, while Japan leads in basic research, Europe and the U.S. are catching up in clinical applications at a breakneck pace.

Ando: From the standpoint of patients, isn't it unnecessary to insist on products originating in Japan?

Mutoh: During the H1N1 coronavirus outbreak, it was difficult to obtain vaccines from overseas. As the world moves toward a more nationalistic approach, from the standpoint of medical security, it would definitely be better if Japan could create the ground for more and more innovative new drugs to be developed.

Okuno: Medicines save the lives of people all over the world. It is a source of pride for the Japanese pharmaceutical industry to produce such drugs. I believe that AI and DX will be of great help in delivering them to those who need them cheaply and reliably. However, AI and DX are not universal tools, but rather like a baseball field. They should be developed as a field where a variety of players, including pharmaceutical companies, can play an active role.

Ueno: In order to maintain and improve Japan's drug discovery capabilities, clinical trial and evaluation systems must be in place. Only when these systems are in place will innovative drugs be introduced from overseas. In addition, drug discovery contributes greatly to raising the level of basic science, and the higher the level of drug discovery, the more it will lead to the development of human resources who will play a key role in Japan's scientific capabilities.

Promote AI utilization, database development, and cluster collaboration.

Ando: What are the obstacles or lacks in accelerating drug discovery innovation?

Takeuchi: From a clinical perspective, there is a lack of a platform for sharing patient and medical information. Japan collects very detailed patient information and the quality of the information is high, but the data is not structured in a way that makes it easy to use. This delay in the creation of data is a disadvantage in promoting international joint clinical trials and other activities.

Muto: As reforms in work styles progress, medical professionals are spending less and less time on clinical trials, and the lack of progress in the creation of databases is creating an extra burden. In addition, the number of medical institutions for clinical trials is dispersed, resulting in inefficiencies.

Okuno: It is not a good idea to decentralize AI and data infrastructures. Unless the government takes the lead and concentrates the budget to develop a common AI and data infrastructure, we will not be able to compete with the rest of the world.

Kaneko: A framework for bringing together the diverse players in the drug discovery ecosystem is already in place, but the cautiousness of the Japanese people is undeniably putting the brakes on the process. If bad data is found in animal experiments, it would be better to improve the data and move on, but because the data was bad, it is no longer good enough and development tends to be terminated. It may be necessary to have a responsible person or organization to take the lead.

Ueno: Failure is an inevitable part of drug discovery, and failure is one piece of information that will lead to the next innovation. As the whole country becomes more and more interested in drug discovery and medical care, now is a good time to cultivate a culture that accepts failure as an opportunity. In Japan, there are excellent biotech clusters in various regions, and innovative research is being conducted, but I feel that each cluster is closed. Approaches from diverse perspectives are necessary for the utilization of new modalities and innovative drug discovery. It is necessary for the national government to take the initiative in creating a mechanism to link the various bioclusters in each region.

(Authored and produced by Nihon Keizai Shimbun, Inc. (2025 Nikkei e-Advertising Special Edition))

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