Japan medical institutions to develop tailored drugs for rare diseases

Tokyo Medical and Dental University and the National Center of Neurology and Psychiatry are planning to develop tailored drugs for patients with genetic disorders to find treatment for extremely rare diseases.

The two Japanese medical institutions will search for rare diseases that have the potential to be treated by nucleic acid drugs from a database of around 2,250 patients.

Nucleic acid drugs are an emerging class of therapeutics,

The development will be carried out based on technology that targets the expression of disease-causing proteins in the mutant gene.

Due to the price and challenges with financial viability, pharmaceutical companies are hesitant to develop medications for exceedingly rare disorders.

According to Takanori Yokota, a professor of neurology and neurological science at Tokyo Medical and Dental University, they hope to make effective patient-tailored nucleic acid drugs and find as many people who can be treated.

The Initiative on Rare and Undiagnosed Diseases, a study that examines the DNA of people whose diseases cannot be detected or for which there is no known cure, will be used to pick participants from among the patients registered in it.

The project catalogs approximately 1,700 linked gene sequences and covers a wide spectrum of illnesses, such as neurological, cardiac, and metabolic problems.

In Japan, only one or two family lines are affected by about 70% of the diseases, and many do not have any reliable treatments.

In addition to the causative gene, factors such as age, the severity of symptoms, and the speed at which the disease is progressing will be taken into account when choosing candidate patients.