We are pleased to announce the launch of our website, entitled: “Intractable Adrenal Disease Research Project” This website was established to serve as a method of promotion for the project as well as an accessible source of information to the general public concerning intractable adrenal diseases.

For many years we have directed efforts toward advances in treatment and research of adrenal diseases. However, due to the sheer variety of these diseases—in addition to their rarity–we found it crucial to establish a more comprehensive, systematic, and sustainable documentation of these endeavors.

We first focused on “Pheochromocytoma.” This disease has been commonly thought to be a prime example of curable endocrine hypertension because surgical remove of the adrenaline-producing tumor is a safe and effective treatment. Unfortunately, we have several examples of young patients with spread of their disease (metastases) recognized a few years after they were thought to have been cured by surgery. These cases prompted us to think that we need a completely new approach. Consequently, in 2006, a working group was assembled and a project was started to take on this challenge as a key clinical research agenda for the Japan Endocrine Society. The progress was initially slow, but in 2009, a subdivision of the National Ministry of Health, Labor, and Welfare in charge of research directed at intractable diseases adopted “Malignant Pheochromocytoma” as one of its research projects and it was referred to as “PHEO-J” for the following 3 years. The research team achieved various accomplishments in relation to the diseases such as conducting a nation-wide epidemiological survey, holding public symposiums, creating guidelines, publishing textbooks directed towards medical personnel, creating an information brochure with the aim of informing the public, organizing and supporting patient associations, as well as building a disease registry using national clinical data gathered over the years. In particular, we believe that accomplishments such as the standardization of the clinical guidelines for the disease registry, the development of new diagnostic agents, and constructing a new biobank were practical and innovative outcomes which held significant clinical value.

Intractable diseases are defined through 4 characteristics: 1) Its etiology is unknown; 2) A standardized treatment for it remains unestablished; 3) The affected patient require long term care; and, 4) It is a rare disease. While these characteristics correspond to numerous adrenal diseases, these criteria exclude malignant tumors classified by the “Cancer Control Act.” Thus, diseases such as malignant and metastatic pheochromocytoma and adrenocortical carcinoma are not categorized as “Intractable Diseases” based on that strict definition. Consequently, diseases s such as pheochromocytoma and Cushing syndrome are classified as “Intractable Diseases” whereas adrenocortical carcinoma even associated with Cushing syndrome and malignant pheochromocytoma are classified as “Cancers”, resulting in differing administrative operations, and thus, different countermeasures.

However, for both malignant pheochromocytoma and adrenocortical carcinoma: 1) There remains no established methods of treatment after the diagnosis; and 2) Early differential diagnosis between benign and malignant tumors poses extreme difficulties for the pathologist and clinician. As individuals who constantly engage in clinical practice and research, we view that a more comprehensive and sustainable approach is mandatory in order to successfully address these diseases.

The “Intractable Adrenal Disease Research Project” was therefore established based on our experience and background. We seek to classify adrenal diseases difficult to overcome through present clinical diagnosis and surgical treatment as “Intractable Adrenal Diseases”. As a project with an aim of providing roles for various research platforms, it was planned with the cooperation of the “Japan Agency for Medical Research and Development” (AMED) as well as the “National Center for Global Health and Medicine” (NCGM). Under this project, we seek to advance various research objectives, produce high quality evidence, develop a higher standard for countering the target diseases, discover and optimize new diagnostic methods and medications while improving the QOL of the affected patients, ultimately leading to a contribution to the overall health of the people in Japan.

We sincerely ask for your understanding and cooperation to our approach for this ambitious initiative.

Research Institute of Clinical Endocrinology and Metabolism, Kyoto Medical Center,

National Hospital Organization, Kyoto, Japan

Special Research Fellow Mitsushide Naruse, MD, Ph.D