Study of specific immune status of autoimmune diseases

This is the “Patient Centricity in Rare Disease R&D Program (abbreviation as PCRD²)” aiming at promoting patient-centered drug development that is jointly conducted by the University of Tokyo and 3H Holdings Inc. operating RareS.  (Click here for further information)

• Outline of research
• Purpose, significance and expected research results, etc.
• Types and information on testing samples which would like to be provided from patients
• How to study
• Withdrawal of consent to the use of testing samples, etc.
• Research results that can be provided to patients
• Conditions for those who can participate in the study
• Flow of participation in research
• Inquiry

Outline of research

Research title: Study of specific immune status of autoimmune diseases
Target diseases: Behcet’s disease, Ankylosing Spondylitis, Multiple Myositis and Dermatomyositis, Adult Still’s disease, Rapidly Progressive Glomerulonephritis, Ulcerative Colitis, Crohn’s disease, Rheumatoid Arthritis
*Recruitment of all diseases has been suspended due to full capacity (as of September 29, 2019)
Research Director: KOTAI Biotechnology Co., Ltd

Purpose, significance and expected research results, etc.

Immune cells (We examine T and B cells this time) are deeply involved in the development and progression of diseases in autoimmune diseases, and play an important role in many diseases, such as cancer and infectious diseases.

By analyzing the function of immune cell receptors (called T-cell receptors and B-cell receptors) which play an important role as limbs of T-cell and B-cell related to disease.

1. We will clarify the causes of “rare intractable autoimmune diseases” which are not clear currently and promote the creation of new therapeutic drugs for those diseases.
2. We will explore the use of therapeutic drugs that are effective in other diseases already for “rare intractable autoimmune diseases” that have not yet had therapeutic agents currently.

In this joint research with you, we will analyze receptors for immune cells (called T cells and B cells) in your blood.

The type of receptor is an astronomical number with 10 to the 18th power. The receptors work together like a right hand and a left hand. Taking a piano performance as an example, the combination of playing a chord with the left hand and playing a melody with the right hand would make one song. In other word, number of songs that can be played are same as the types of receptor and every person has repertoire of 10 to the 18th power. The study this time is to examine the types of receptor, which is called immune repertoire.

As each song has own image, immune cells usually play gentle songs, and when a foreign enemy comes, they play battle songs. In the case of autoimmune diseases, even though there are no bacteria or cancer cells that become foreign enemies in the body, immune cells that play battle songs increase and attack their own. We identify the cells and look for ways to quit playing the battle songs and play gentle songs.

On the other hand, when cancer is occurring, immune cells are trapped by the cancer cells not to attack them, so that can attack cancer cells by playing the battle songs play correctly.

This research on “Immune Repertoire” is the latest, and by conducting joint research with patients, we will obtain useful results for many patients suffering from “Rare Refractory Autoimmune Diseases” and lead to creation of new drugs.

Types and information on testing samples which would like to be provided from patients

As almost same volume, about 10cc, as the blood collected in health checkup.

How to study

With regards to blood, after isolation of immune cells (T cells and B cells), the gene sequence of immune cell receptors (right hand: TCR and BCR, left hand: HLA) are analyzed. We examine the situation of your immune repertoire that is active in your blood. Through this kind of research, we will search for drugs that can be used for your diseases among the drugs already in the world. Furthermore, it will lead to the creation of new therapeutic agents.

Withdrawal of consent to the use of testing samples, etc.

If you want to stop this joint research, you can cancel it at any time. In that case, we will discard your blood samples, etc. as well as all of your information and the results of your research.

Please contact “Contact for inquiries of RareS. 0120-330-800 (weekdays: 10:00-18:00)” to inform that you would like to withdraw your consent and discontinue joint research. Even if the consent of the joint research is withdrawn and it is canceled, there is no disadvantage to you at all.

However please note that we will not be able to provide feedback on the results of this joint research or the latest research information with you.

Research results that can be provided to patients

As a summary of the results obtained from this study, we will inform you of the similarities and differences (rough profiles) of each immunity related disease after completing the analysis, if you wish.

This joint research is the first step to find a drug for your treatment together. This joint research period will be short and preliminary, but it will not only you find a new drug to treat your disease in the long term, but also what lifestyle and existing treatments will be able to stabilize your health conditions. In the future, we also would like to conduct joint research with you in the long term.

Conditions for those who can participate in the study

• 18 years old or older
• Those who are suffering from the target diseases and are currently being treated
• Those who have documents that can prove that you are suffering from the disease
• Those who can visit the Tokyo Center Clinic (the nearest station: Tokyo Station)
• Target diseases: Behcet’s disease, Ankylosing Spondylitis, Multiple Myositis and Dermatomyositis, Adult Still’s disease, Rapidly Progressive Glomerulonephritis, Ulcerative Colitis, Crohn’s disease, Rheumatoid Arthritis

*Recruitment of all diseases has been suspended due to full capacity (as of September 29, 2019)

Flow of participation in research

The recruitment has been suspended because we have reached the planned number of patients for all diseases. In the future, there is a possibility that recruitment will be resumed, or research targeting other diseases may be started, so if don’t mind, we ask for your cooperation in the awareness survey and in pre-registration.