For patients and their families

Source: https://www.cira.kyoto-u.ac.jp/e/faq/faq_patient.html Parent: https://www.cira.kyoto-u.ac.jp/e/pressrelease/news/260224-100000.html

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For patients and their families

Researches on \ Diseases Clinical Research Parkinson's disease \ research

Researches on Diseases

The following answers are based on information available as of January 2021.

Does CiRA make iPS cells using cells from patients?

Yes, since receiving approval from our institutional review board in June 2008, the laboratory of CiRA Deputy Director Tatsutoshi Nakahata has begun generating and conducting research on disease-specific iPS cells. The research plan involves the derivation of iPS cells from somatic cells contributed by patients suffering from diseases including Duchenne’s muscular dystrophy, amyotrophic lateral sclerosis, and Fanconi’s anemia. CiRA labs have generated iPS cells from patients with other diseases, and some of them have been distributed to other research institutes through RIKEN BioResource Center.

What type of research will be done with patient-derived iPS cells?

iPS cells generated from somatic cells donated by patients can be induced to differentiate into cell types of interest, which may make it possible to recreate disease states in vitro, enabling the study of disease mechanisms. Such cells may also be useful in studying drug efficacy and side effects, which may contribute to the development of new drugs.

Are there other research institutions apart from Kyoto University that are engaged in research based on generating iPS cells from patient cells?

Yes, at research institutions in and outside Japan, research using disease-specific iPS cells is in progress with the cooperation of patients. In Japan, publicly sponsored iPS cell-related projects are ongoing, with the participating research institutions engaged in wide-ranging studies. \ \ 【Major public iPS cell-related project websites】\ Please see below "How can I get the latest information on iPS cell research?."

How can I get the latest information on iPS cell research?

Research information at CiRA will be posted on the Research Results page of this website and be announced at symposiums and other events. You can obtain information from the websites of research institutes other than Kyoto University and from websites that provide research information on stem cells such as iPS cells, regenerative medicine and cell medicine. \ \ 【Website for iPS cell research, etc. 】

Research projects:

For Patients:

Clinical Research

The following answers are based on information available as of January 2021.

What is the plan for clinical Research or clinical trials at CiRA?

In cooperation with Kyoto University Hospital and others, we are engaged in several clinical studies or clinical trials in regenerative medicine (cell transplantation therapy) and drug discovery. When we decide to start clinical research or clinical trial, we will announce them on our website, etc.

Projects on regenerative medicine/cell transplantation therapy:

Projects related to drug development:

I would like to participate in clinical research or clinical trials for regenerative medicine (cell transplantation therapy) using iPS cells. What should I do?

When clinical research or clinical trials are to be started at CiRA, we will notify on our website.

CiRA | News and Events | News

I have seen on the Internet that there are medical institutions that carry out treatment using iPS cells and other stem cells. Are there any safety issues?

iPS cells are still at the research stage, and there are no medical institutions giving treatment using iPS cells for any disease, except the clinical research on age-related macular degeneration. Likewise, there are no medical institutions in Japan carrying out treatment using ES cells.\ \ However, there is development of new therapies in progress using somatic stem cells. Unlike iPS cells and ES cells, these stem cells are naturally present in the human body but can differentiate only into a limited range of cell types. For instance, blood stem cells can differentiate only into blood cells. In addition, the number of cells that can be sampled is small. For these and other reasons, they are not likely to have therapeutic effect across the whole range of disease. \ \ Some websites and publications do use language that suggests therapeutic benefit, and there are some medical institutions in Japan and overseas that use what they call somatic stem cells in injections and drip infusions. The therapeutic effect and safety of such treatments are often poorly supported by scientific papers or data, however. Furthermore, these organizations offer almost no information about safety control and patient protection. Because of this, the Japan Society for Regenerative Medicine (see link below) has called for caution regarding inappropriate stem cell therapy. When considering a consultation for stem cell therapy, patients are advised to confirm that the treatment’s safety has been scientifically demonstrated and approved by an official body. \ \ Meanwhile, the International Society of Stem Cell Research has produced a Patient Handbook on Stem Cell Therapies that deals with frequently asked questions about stem cells and stem cell therapies (see link below). The handbook, which can be downloaded as a PDF from the Society’s website, offers an essential minimum checklist of items that should be confirmed by people considering stem cell therapy. Patients are recommended to refer to it. \ \ In Japan, a law on safety control in regenerative medicine and related therapies was enacted in November 2013 as part of ongoing moves to put in place a legal framework to safeguard stem cell-based treatment.

ISSCR (International Society for Stem Cell Research) | \ Patient Resources

When will cell transplantation therapy using iPS cells become a reality?

When the safety and therapeutic effect of iPS cells have been confirmed, we will seek the cooperation of patients to proceed to clinical research. In fact, clinical research for age-related macular degeneration has already begun, as a patient has received a transplant of retinal cells derived from iPS cells. However, in order to offer cell transplantation therapy widely, large-scale cell processing technologies and treatment facilities are needed. These demands require cooperation with industry and hospitals, which makes it difficult to forecast a date for wide medical application. \ \ Therefore, at this time, it is difficult to give a definite answer as to when medical applications will become available. We will continue to promote research every day with the aim of bringing new treatment methods to patients as soon as possible.

Parkinson's disease research

The following answers are based on information available as of April, 2025. \ \ The term "clinical trial" refers to the iPS cell-based therapy for Parkinson’s disease conducted at Kyoto University Hospital.

Which patients will participate in the clinical trial?

Eligibility is given to patients diagnosed with Parkinson's disease and who also meet the following criteria. \ \

・Drug treatment does not adequately control symptoms

・Are between 50 and 69 years old at the time of consent

・Show signs of on-off phenomenon

・Score stage 3 or higher on the Hoehn and Yahr scale in the off phase

・Score stage 3 or lower on the Hoehn and Yahr scale in the on phase

・Responds well to L-dopa administration

* Note that patient recruitment is now complete.

How many patients will participant in the trial?

In total, 7 patients are expected to participate.

Where will the trial be conducted?

All patients will be treated at the Kyoto University Hospital.

How long will patients be admitted to the hospital?

Approximately one month.

How long will patients be observed after the cell transplant? How often will patients need to visit or be admitted to the hospital?

The follow-up period is two years. Initially, patients will visit the hospital monthly, but that will be reduced to every two months eventually. Patients will also be admitted 5 times during this two-year window. Follow-up will also be done after the trial ends.

How long are the hospital stays for the transplantation and follow-up observations?

The hospital stay will be 1 month for the transplantation and 1 week for the follow-up observations.

How can I apply to participate as a patient?

The clinical trial is no longer recruiting patients.

What type of surgery will be done?

Approximately 5 million neural progenitor cells differentiated from iPS cells will be transplanted into both the left and right putamen by stereotactic surgery.

Can deep brain stimulation (DBS) be combined with DBS?

Patients who have undergone DBS are not eligible for this clinical trial, but we expect the two treatments can be combined in the future.

When will the cell transplantation be available to all patients?

The use of the cell transplantation as an approved treatment will depend on the results of the clinical trial and cannot be predicted at this time.

If this cell transplantation treatment becomes mainstream, will it be eligible for insurance subsidies?

It is expected to be eligible for insurance subsidies.

What is the current status of the clinical trial?

For the clinical trial that started in 2018, we completed cell transplants for a total of 7 patients as planned by the end of 2021, and the monitoring of the patients after the cell transplantation also finished at the end of 2023. \ \ The results, published in the scientific journal Nature in April 2025, suggest both safety and clinical benefit.