What are iPSCs?

What are iPSCs?

What are iPSCs?

“iPSCs” is an abbreviation for induced pluripotent stem cells and your stem cells for life and health, with ethical origins. It was developed by Professor Shinya Yamanaka of the Kyoto University iPS Cell Research Institute, who received the Nobel Prize in Physiology or Medicine in 2012 for his work. iPSCs are produced by introducing special factors into cells of the body, such as human skin and blood, and culturing them.

Main features of iPSC

By taking advantage of the following three features, we can get the necessary amount of various types of cells, such as human neurons, myocardia, T cells, etc. iPSCs are expanded and then converted to the target cell type, which can be used for regenerative medicine and drug discovery. Furthermore, by using one’s own iPSCs to generate cells for transplantation for cell therapy, the risk of an adverse immune response is significantly decreased.

1.Ability to be produced theoretically from any type of cell in the body.

iPSCs can be made by introducing special factors into various types of cells such as blood, skin, and hair cells and culturing them. With the current technology, iPSCs can be made with just 5 mL of blood.

1.Ability to be produced theoretically from any type of cell in the body.

iPSCs can be made by introducing special factors into various types of cells such as blood, skin, and hair cells and culturing them. With the current technology, iPSCs can be made with just 5 mL of blood.

2. Being able to transform into cells of various tissues and organs.

A major feature of iPSCs is that they have “pluripotency”. Pluripotency means being able to become cells of various types, such as neurons and cardiomyocytes.

2. Being able to transform into cells of various tissues and organs.

A major feature of iPSCs is that they have “pluripotency”. Pluripotency means being able to become cells of various types, such as neurons and cardiomyocytes.

3. Have the ability to proliferate almost indefinitely

Since iPSCs can divide and proliferate quite well, they can be cultured and expanded to obtain the amount of cells necessary with ease.

3. Have the ability to proliferate almost indefinitely

Since iPSCs can divide and proliferate quite well, they can be cultured and expanded to obtain the amount of cells necessary with ease.

Application of iPS cell technology

There are two main ways to apply iPSC technology. One is the so-called regenerative medicine, where cells such as neurons and heart cells from iPSCs are generated and transplanted. The other is to use patient-derived iPSCs and convert them into cells specific to their disease and use for research such as drug discovery screening, toxicity testing, and studying the mechanism of disease by reproducing the disease state.

1. Application for regenerative medicine

Regenerative medicine is a treatment that restores the function of a part of a body that has been lost due to injury or illness. iPSCs can theoretically be made from any cell in the body, and can be converted into a variety of cells that make up the body.

2.Drug development, investigating disease mechanism

Cells such as neurons and cardiomyocytes made from iPSCs made from patient’s blood, etc., can have the same phenotype as the patients’ cells in the body.

How will iPSCs be used for regenerative medicine, drug discovery, and elucidation of pathological mechanisms?
First, iPSCs are prepared by collecting cells from the patient’s blood and skin and introducing reprogramming genes. Since large numbers of cells are required for cell transplantation and drug discovery, once iPSCs are generated, they are expanded (to increase the number of cells). By transforming a large number of iPSCs to target cells such as neurons, cardiomyocytes, blood cells, bone, etc., it is possible to obtain enough cells for cell transplantation treatment and drug discovery. The resulting cells can be roughly divided into two uses. One is regenerative medicine that improves the functions of the body by transplanting cells such as the differentiated cardiomyocytes and neurons into the human body. Since Professor Shinya Yamanaka announced the successful generation of human iPSCs in 2007, various studies have been conducted for clinical applications in Japan and overseas. Examples of diseases for which clinical research has already begun include age-related macular degeneration, Parkinson’s disease, severe ischemic cardiomyopathy, and spinal cord injury. In addition, research is being conducted on application to diabetes, corneal disease, cerebral infarction, and liver and kidney diseases, among many others.

Also, CAR-T cell therapy research is attracting attention as one example of cancer treatment using iPSCs. This is an immunotherapy in which a patient’s blood-derived iPSCs are genetically modified to produce T cells that can recognize and attack specific cancer cells when returned to the patient’s body. The second application is to reproduce disease in a culture dish using iPSCs and use it to study disease mechanisms and develop new drugs. Utilizing iPSCs for drug discovery has two significant benefits. One is that it can be performed using human cells, as opposed to animals. Second, iPSCs can grow indefinitely, so researchers can get as many cells as needed. By successfully combining cells made from patient-derived iPS cells with animal experiments, we can efficiently develop new drugs in a short period of time and evaluate drug efficacy more accurately. In addition, the effects and side effects of individual drugs may differ due to differences in genetic background and environmental factors. With iPSC technology, optimal drug for each individual can be tested on patient-derived iPSC-based assays so personalized medicine is no longer a dream. In particular, iPSCs can greatly reduce the cost of orphan drug research and development.

History of iPSC

1958

Successful reprogramming of frog somatic cells
(Sir John Bertrand Gurdon)

1998

Establishment of human ES cells

2006

Establishment of mouse iPS Cells

2007

Establishment of human iPS Cells

2012

Sir John Bertrand Gurdon / Dr. Shinya Yamanaka
The Nobel Prize in Physiology or Medicine

Regenerative Medicine

2009

Human stem cell
First clinical trial approval
(ES Cell・Spinal injury)

2013

Jul  AMD Clinical trial (world’s first with iPSC technology)

2018

Sep   Platelet Clinical trial
Nov  Parkinson disease 1st Autotransplantation

2019

Feb  Spinal cord injury Clinical trial
Apr  AMD 5 transplantations completed one-year follow-up
Aug  Corneal disease 1st Transplantation
Dec  NK Cell Cancer 1st Transplantation

2020

Jan  Cartilage Clinical trial
Jan  Heart disease 1st Transplantation

Drug Discovery

2016

Jan  Spinal muscular atrophy Clinical trial

2017

Aug  Progressive ossifying fibrodysplasia  clinical trial

2016

Apr Pendred Syndrome clinical trial
Dec ALS clinical trial(total 3)

1958

Successful reprogramming of frog somatic cells
(Sir John Bertrand Gurdon)

1998

Establishment of human ES cells

2006

Establishment of mouse iPS Cells

2007

Establishment of human iPS Cells

2009

Human stem cell
First clinical trial approval
(ES Cell・Spinal injury)

2012

Sir John Bertrand Gurdon / Dr. Shinya Yamanaka
The Nobel Prize in Physiology or Medicine

Jul AMD Clinical trial
(world’s first with iPSC technology)

2018

2013

Sep  Platelet Clinical trial
Nov  Parkinson disease 1st Autotransplantation

2016

Jan  Spinal muscular atrophy Clinical trial

2017

Aug  Progressive ossifying fibrodysplasia  clinical trial

2019

Feb  Spinal cord injury Clinical trial
Apr  AMD 5 transplantations completed one-year follow-up
Aug  Corneal disease 1st Transplantation
Dec  NK Cell Cancer 1st Transplantation

2020

Jan  Cartilage Clinical trial
Jan  Heart disease 1st Transplantation

2018

Apr Pendred Syndrome clinical trial
Dec ALS clinical trial(total 3)

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