Regenerative Medicine Bets on iPS Stem Cells

8 Min. Read  |  March 2, 2021

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The type of stem cell called iPSCs (induced Pluripotent Stem Cells) has worked its way into potential therapies for virtually every part of your body, and the research is being done in thousands of labs around the world. In the less than 15 years since Shinya Yamanaka created the first iPSCs this versatile type of stem cell has become a favorite candidate for creating tissues to repair or replace parts of our bodies that just aren’t working right.

These cells—called “pluripotent” because they can make any tissue—result from delivering and briefly turning on in adult cells three or four genes that normally only have a role in embryos. That creates the iPSCs that can then be given genetic or chemical instructions to become any needed tissue. Theoretically iPSCs can be made from any cell in your body, but the efficiency of making them and their quality varies based on the cells you start with. The blood cells stored in the GoodCell personal biobank include one type of cell, the endothelial progenitor cell, that is particularly efficient at making more genetically stable iPSCs. And because that cell spends most of its time deep inside your body it has fewer sun-induced mutations than other cells. Cell banking these types of cells as early as possible preserves your best cells with the fewest age-generated mutations, making cell storage a potential means for taking advantage of cell therapies in the future.

Clinical Trials Show Progress

Proud of native son Yamanaka’s Nobel-Prize-winning accomplishment, the Japanese government and research institutions have pushed the fastest into iPSC clinical trials.  Teams there are testing therapies for heart failure, the leading cause of vision loss in adults, Parkinson’s disease, arthritic knees and several other conditions. World-wide there are more than 100 clinical studies using iPSCs.

Some of the Japanese trials use cells made from the patient and some use cells from banks of cells curated to be compatible with the majority of the population’s immune systems in Japan.  This is relatively easy in the genetically more homogenous population there, unlike in the U.S. Even then patients tend to be given potentially dangerous immune suppressive drugs to protect the donor tissue from rejection. In this country, scientists increasingly see the need to make sure their work reflects the wide genetic diversity of our population. Getting immunologically compatible donor cells here is much more difficult, leading many researchers here to work on personalized therapies made from individuals’ own cells, like those stored in the GoodCell personal biobank.

The National Institutes of Health has a clinical trial underway for the same vision loss as the Japanese trial, called Age-related Macular Degeneration. A Boston team treated a Parkinson’s disease patient with good results, and the Food and Drug Administration in the past month has given approval for large trials in Parkinson’s and diabetes. Other teams have trials underway or are in final stage of planning for heart failure, spinal cord injury, endometriosis, cartilage defects and several other conditions.

Stem Cells Are Powerful When Combined with Other New Technology

Bioprinting has emerged as one of the most exciting new fields in biotechnology. It’s very much as it sounds. A printer that uses cells as ink. Using various types of cells created by those chemical and genetic guides given to iPSCs, researchers can lay down complex layers of different cells and tissues. The technology recently produced functional kidney tissue following on success with several other organs. Each organ immediately becomes a tool for studying the underlying causes of the diseases afflicting the cell donors, but also begins to create a path to replacement of tissue and organs.

Many other tissue engineering advances have come from the simple concept of scaffolds, giving the new cells the structure and shape they need to create the desired tissue. A team in London recently used a scaffold to build a whole functioning thymus. That little discussed tiny gland helps mature our immune system T cells so they can get to work, and it is suddenly a very big deal during the COVID pandemic.  A poorly functioning thymus probably accounts for some of the more severe outcomes with the disease. 

Advances Make Getting the Right Tissue Easier

While it may sound relatively simple to nudge a gene or add a chemical and get iPSCs to turn into nerves, getting that right recipe has become one of the toughest parts of developing iPSC-derived therapies. But several groups in the past few months made major advances simplifying the task, with one team publishing a virtual recipe book available to scientists around the world for improving the creation of any tissue.

Then comes the task of getting the new cells to where they are needed. That final step, gearing up manufacturing to serve the thousands of patients needing therapies, is now being supported by a vast new industry that saw that saw a 20 percent increase in capital investment last year and is expected to reach $10 billion by 2030.  The FDA expects 10 to 20 new cell and gene therapies to be approved each year by 2025.

Harnessing iPSCs to Create New Drugs

Two members of GoodCell’s scientific advisory board (SAB) are among the world’s leaders in harnessing the power of iPSCs to understand disease and look for ways to intervene either with cell therapies or traditional drugs therapies. For many diseases, researchers have had a hard time figuring out what is really causing the malfunction. But by creating iPSCs from people with the disease, they can create, for example, nerves in a petri dish that have all the attributes of ALS (amyotrophic lateral sclerosis or Lou Gehrig’s disease).

Kevin Eggan, GoodCell SAB member and professor at the Harvard Stem Cell Institute, a few years ago developed such a model for ALS and found the patient’s nerves die from the exhaustion of over firing.  Subsequently they tested a drug known to calm over-active nerves and in a recent phase 2 trial it improved nerve function in ALS patients. Similarly, SAB member Joseph Wu from Stanford has used iPSC-derived heart cells from patients to uncover the basis of various cardiomyopathies and other cardiac conditions, and he expects to create paths to individualized therapies.

Having this world leading expertise on our SAB helps GoodCell remain prepared to make the best possible use of the cells stored in your Personal Biobank.