A group from Tokyo Medical and Dental University and others has started research to treat diseases for which drug development is difficult due to the extremely small number of patients and other reasons using a new technology called "nucleic acid medicine." After considering how to select patients who can be expected to be effective in treatment, we will aim for actual treatment in cooperation with medical institutions in the United States.
This research was initiated by a research center specializing in nucleic acid drugs at Tokyo Medical and Dental University and a group including AMED=Japan Agency for Medical Research and Development.
"Nucleic acid medicine" is a technology that regulates and treats disease-causing genes and proteins using a substance called "nucleic acid", which is the source of genes, and is expected to be able to make the optimal drug for each patient.
The group investigates nucleic acid medicines necessary for treatment for diseases in which it is difficult to develop drugs because there are only a few patients in Japan, and diseases that progress quickly and cannot be put into practical use in time.
If there is a patient who is expected to be effective, we will start administering nucleic acid drugs at affiliated medical institutions in the United States, and aim to build a system that can continue administration in Japan.
The group will select the first patient as early as this year.
Professor Takanori Yokota of Tokyo Medical and Dental University said, "Our major goal is to help patients with intractable diseases.