How Scientists are Using iPSCs to Develop New Therapies

In 1998, scientists reported being able to derive cells from human embryos that could develop into almost any cell in the body. In 2007, the field took a huge leap when scientists discovered they could reprogram human adult skin cells to act like these embryonic stem cells.

Adult cells transformed into an embryonic state-;also called induced pluripotent stem cells-;are already being used to study ways to Parkinson's disease. Another burgeoning use is reprogramming healthy donor blood cells into iPSCs and creating billions of specialized cells to rejuvenate and provide for therapeutic applications. These cells can be used for treatments, such as to replace dead neurons in patients with Parkinson's disease or muscular dystrophy, or to create immune cells that act as delivery vehicles to treat cancers.

We also produce gene-modified iPSC lines. These cell lines could be used to target cancer cells more effectively, or to enhance function and transplantation of a regenerative cell therapy.

How do scientists produce stem cell and gene therapies?

We take adult cells, such as from the skin or blood of adults, or cells from umbilical cord blood, and we reprogram them back in developmental time into iPSCs. We do this by temporarily inserting various genes, growth factors and chemicals into the cells. The transformation process takes about a month.

These cells are then isolated and transferred to bigger petri dishes to allow them to multiply and expand. We create large cell "banks" from the cells, called stem cell lines. It takes another two to three months to create a cell line from a few transformed cells.

We can always go back to the cells, whether live or frozen, and create more copies of them in an almost infinite manner.

We also create gene-edited versions of iPS cells using the latest methods, like CRSPR/Cas9, to introduce new genes, delete existing genes, or modify abnormal genes.

Where do the cells go once they're produced or reprogrammed?

We make some iPS cell lines for investigators within Cedars-Sinai as well as for researchers at other universities and research institutions. We also supply cell lines to investigators at biotech and pharma companies. To date, we have generated over 1,200 iPSC lines.

Our team is also developing iPSCs and gene-edited iPSCs for specific cell therapies for early human clinical trials. We are also working with different types of cells. For example, we are supporting a clinical trial that is studying a potential new cell therapy involving adult retinal stem cells for dry age-related macular degeneration. For this trial, we generate the adult retinal cells in our center under strict U.S. Food and Drug Administration-regulated conditions, put them in a vial and ship them overnight to get transplanted into patients' eyes the next day.

Every process involved in the life cycle of a production of a cell therapy gets documented and recorded as per U.S. FDA requirements.

How large is the center?

We have about 25,000 square feet of physical laboratory and manufacturing space. We are staffed with 40 scientists, specialists, technicians and assistants, and 10 support staff including managers, administrators, facilities operators, quality assurance staff, and control staff.

What are some of the center's biggest accomplishments?

There are many, but to pick a few, in 2017, we were able to obtain approvals to build the facility. Construction was completed in 2020 before the COVID pandemic. We were commissioned and qualified to begin operations in 2021 and then licensed by the California Department of Public Health. We have created the world's largest repository of 1,000 iPSCs from patients with amyotrophic lateral sclerosis, also known as ALS, which are available to researchers through our portal. We have also produced the first set of retinal therapy cells that went into patients enrolled in a clinical trial for dry adult macular degeneration. We were also awarded a $3.12 million grant from the Department of Defense to enable creation of clinical-grade iPSC lines and methods to create clinically compatible iPSC-derived vascular endothelial cells to develop future regenerative stem cell treatments for diseases or conditions such as chronic wounds, ischemia, and sepsis.

Most recently we received a $2 million CIRM infrastructure grant to further the mission of the CBC.

How does the CIRM grant help the center meet its goals?

It will improve our operational efficiency, such as our ability to customize and implement electronic quality manufacturing systems for manufacturing cell therapies. The grant will also help us scale up by incorporating machine learning and artificial intelligence into the production of cell lines.

Lastly, the grant will allow us to partner with community colleges to create an internship and training certificate program focused on cell and gene therapies. We'll train the next generation of regenerative medicine scientists and support staff.

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