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Development field

The Field of iPSC Regenerative Medicine

1. iPS cell–derived retinal pigment epithelial (iPS-RPE) cells

Healios undertakes research and development to provide RPE cell drugs made from differentiated iPS cells in the practical application of new treatment methods.
Based on our expertise with methods developed by researchers from RIKEN for the differentiation of RPE cells, we have succeeded in applying our own unique knowhow to establish an effective culturing method. Our Kobe laboratory has more than 20 staff, including an ophthalmologist and three PhDs, who research the formulation of these cells for surgery and the administering of drugs to people affected with age-related macular degeneration, while at the same time, developing safe and efficient production methods.

Development field

Since 2014, we have also been working with Osaka University in an industry-academic collaboration to further research and development of iPS cell-related technologies through a joint research chair in cell manufacturing system engineering.

2. Non-ophthalmology efforts

The overall research and development base for iPSC regenerative medicine has been expanding with the publication of related research results by numerous independent research entities. The Healios policy is to focus its efforts on pursuing promising work and conducting related joint research with the aim of developing practical applications. Based on this policy we have already begun joint research with Yokohama City University on human organs and, as a platform technology for various organs, plan to expand beyond the ophthalmologic field into research and development of treatment methods for other organs, the goal of which is to cause the least damage to the human body and prevent immunological rejection.

Somatic stem cell for regenerative medicine field

Somatic stem cell regenerative medicine utilizes somatic stem cells, which are present in various tissues of the body, with the aim of developing new treatments for diseases for which no effective treatments are currently available.

There are many types of somatic stem cells, including neural stem cells, mesenchymal stem cells, and hematopoietic stem cells, which are found in various tissues of the body. While some stem cells can only differentiate into certain types of cells, and others can differentiate into multiple types. In general, the range of cell types that somatic stem cells are able to differentiate into is more limited than that of iPS cells and so on.

■A pipeline for somatic stem cell for regenerative medicine (HLCM051)

Healios holds a development pipeline for treating ischemic stroke using the stem cell product HLCM051 (MultiStem®), for which the patent and licensing rights are held by Athersys, Inc.

In ischemic stroke (the target disease of this pipeline), blood vessel blockage in the brain prevents the supply flow of oxygen and nutrients, causing the nerve cells ahead of the blockage to gradually die off. It is estimated that 230 thousand to 330 thousand people experience ischemic stroke every year in Japan (our estimate is based on figures provided by the Ministry of Internal Affairs and Communications, Datamonitor and others), and it causes roughly 64 thousand deaths per year (Ministry of Health, Labour and Welfare Population Survey Report). Even those who survive often suffer impaired function, become bedridden or require assistance with daily activities.

Ischemic stroke is often treated with the thrombolytic agent t-PA, which dissolves the blood clots in the arteries of the brain. However, as thrombolytic agents may only be prescribed within 4.5 hours following the onset of symptoms, it is anticipated that the development of new drugs will expand the therapeutic time window for ischemic stroke treatment. MultiStem, the stem cell product developed by Athersys, Inc., is administered via intravenous injection, and is then conveyed to the spleen. It is believed that the product works by suppressing inflammatory immune cells, reducing inflammation and immune response and thereby minimizing damage to the nerve cells, as well as by producing neuroprotectants.

A Phase II trial for this product has already been conducted by Athersys, Inc. in the United States and Europe. The results of that trial suggest that a potential therapeutic benefit is associated with early treatment (<36 hours) with this product following acute ischemic stroke. Taking the results of this the United States and European Phase II trial into account, we are now moving forward with a Phase II/III study in Japan for the early treatment of acute ischemic stroke.

For more information about these clinical trials please refer to the website “ClinicalTrials.gov”(https://clinicaltrials.gov/ct2/show/NCT02961504).