News Releases & Research Results Development of novel treatment for autoimmune pulmonary alveolar proteinosis designated as an intractable disease based on understanding of the cause: cytokine inhalation therapy for the first time in Japan

Results of research and development conducted by the research group led by Professor Koh Nakata of Clinical and Translational Research Center, Niigata University Medical and Dental Hospital (participated by 12 medical institutions in Japan: Hokkaido University, Tohoku University, Niigata University, Chiba University, National Center for Global Health and Medicine, Kyorin University, Aichi Medical University, Kyoto University, National Hospital Organization Kinki-Chuo Chest Medical Center, Kobe City Medical Global Center Hospital, Kurashiki Municipal Hospital, and Nagasaki University)

• For autoimmune pulmonary alveolar proteinosis (*) designated as a rare disease, leading to accumulation of mucus called surfactant in the alveolar and bronchioiles before progression to respiratory failure, serum diagnosis and treatment have been developed following elucidation of the pathogenesis.
  *The estimated number of patients is ~3,300 in Japan. Males in the 50s are associated with higher incidence. A home oxygen therapy is required in 20% of the patients.
• Inhalation of cytokine (bioactive protein) GM-CSF was scientifically demonstrated to be effective for treatment for the first time in the world. Considering that whole lung washing, which places a significant burden on the patient, serves as a current effective therapy, it is expected that this developed therapy may reduce the burden on patients.
• GM-CSF has been already overseas launched as a drug (trade name: Leukine), but is not granted approval in Japan. In the future, recruitment of a company is planned aiming at submission for regulatory approval and NHI price listing.

This research and development was supported by Practical Research Project for Rare/Intractable Diseases of AMED.

These research and development results were published on September 5 in The New England Journal of Medicine.

Tazawa R., et al. Inhaled GM-CSF for Pulmonary Alveolar Proteinosis, The New England Journal of Medicine
DOI：10.1056/NEJMoa1816216