Sue and Bill Gross Stem Cell Research Center celebrates 10 years of innovation

Recently, UCI celebrated 10 years of lifesaving innovation out of the Sue & Bill Gross Stem Cell Research Center. In this Q&A, center director Aileen Anderson discusses some key accomplishments and describes the importance of stem cell research – how it’s the key to fighting some of the most tragic illnesses, from neurodegenerative diseases such as Alzheimer’s and Parkinson’s to conditions such as blindness and the infectious disease of most concern today, COVID-19.

Why is stem cell research so important? Does it really play a role in fighting disease and helping create a healthier future?

The extraordinary potential of human pluripotent stem cells was immediately apparent when they first appeared on the scene in 1998. Pluripotent stem cells can be coaxed into becoming nearly any specialized cell type. But making that potential a reality required lots of hard work, and over the past decade, that work has been bearing fruit. Now we can see practical ways to take stem cells and mature them into highly functional cell types to treat injured or diseased organs or tissues.

How has stem cell research developed in the last 10 years?

Stem cell research has grown in both practical ways and in important new directions. We now know how to scale up stem cell production to create large numbers of cells for therapies. We have new sources of stem cells such as induced pluripotent stem cells, a technology that won the 2012 Nobel Prize and allows us to obtain stem cells that act like embryonic cells but are from adult cells. Another great new technology involves “organoids,” in which stem cells begin to organize themselves into tissues or organs.

What are a few of the common misunderstandings or myths about stem cell research?

The most common misconception is that all stem cells can do everything. Stem cells from early discarded embryos are different from stem cells derived from umbilical cord blood, and both are different from stem cells derived through genetic manipulation of adult skin cells. These cells are all quite different in their ability to grow, in their ability to mature into other cells, and in their ability to be transplanted. But many people are marketing adult stem cells for unapproved clinical uses with justifications based on what only embryonic stem cells can do.

How would you characterize your center’s main achievements over the past decade?

The Sue & Bill Gross Stem Cell Research Center has accomplished the difficult task of building its own comprehensive program. We have a pipeline that goes from basic science studies on how stem cells work at the molecular level to the bioengineering level, where scientists explore how stem cells interact within the body to form tissues and organs. We also study how to apply these findings at the preclinical level, where the stem cells are tested in disease models prior to testing in FDA-approved clinical trials.

What are some of the most important stem cell projects currently underway?

The use of stem cells to treat the blinding disease retinitis pigmentosa is in its third and most advanced clinical trial. The use of stem cells to develop a therapy for the incurable Huntington’s disease is moving toward a clinical trial. And the use of various types of stem cells to repair spinal cord injuries has been pioneered at UCI. The addition of four new faculty will expand the development of our program in tissue and organ engineering.

How is the work at the Sue & Bill Gross Stem Cell Research Center contributing to the fight against COVID-19?

Our scientists are using their stem cell skills and technology to help in many ways. The UCLA-UCI Alpha Stem Cell Clinic is participating in several clinical trials of new therapies for COVID-19 sponsored by the California Institute for Regenerative Medicine. Our scientists are studying both new antiviral drugs that might be useful and the basic biology of the coronavirus to find possible targets. Our team is helping in other ways too: Our core lab team, led by specialist Christina Tu, has been providing virus transport kits for testing when supplies available to UCI Health are limited. These kits are used for virus tests in the UCI Health pathology laboratories headed by stem cell center member Dr. Edwin Monuki. Thus, our center has been a great contributor to UCI’s COVID-19 program.

What center priorities are you focusing on and what investment and support will be necessary for the next decade of stem cell research discoveries and achievements?

Scientific fields develop incrementally as we build upon the knowledge gained. When we step back, we can see the breakthroughs that have happened from the accumulated knowledge – but breakthroughs are hard to see while they’re happening and even harder to predict. We can anticipate two broad themes in the coming years: First, our early successful clinical trials will move on to FDA approval and widespread use, while many more preclinical studies will be ready for human testing in clinical trials. The second theme is that our basic science and tissue engineering programs will move ahead in defining new approaches for using stem cells to repair or replace diseased or damaged tissues. The future of this field is bright and filled with promise.

How is the work at the Sue & Bill Gross Stem Cell Research Center contributing to the fight against COVID-19?

Our scientists are using their stem cell skills and technology to help in many ways. The UCLA-UCI Alpha Stem Cell Clinic (ASCC) is participating in several clinical trials of new therapies for COVID-19. Our scientists are studying both new antiviral drugs that might be useful and the basic biology of the coronavirus to find possible targets. Our Alpha Stem Cell Clinic (ASCC) is partnering with the City of Hope for a clinical study on the use of convalescent plasma as a potential treatment for COVID-19. In another study, SARS-CoV-2 positive subjects requiring hospital admission are being treated with immune (natural killer) cells to test if they are safe and have any impact on the disease. Severely ill patients, already on ventilators for breathing support, are being enrolled in a third clinical trial to see if a cellular therapy derived from donated human placental tissue is safe and effective for treating acute respiratory distress syndrome due to SARS-CoV-2. This work today, and for the last 10 years, has been fueled by the research engine that Californians created with the California Institute for Regenerative Medicine (CIRM). Our team is helping in other ways too. Through the efforts of the SCRC core lab team, especially Christina Tu and Allia Fawaz, we provided thousands of virus testing transport kit components for COVID-19 testing when supplies available to UCI Health were limited. These kits have been used for COVID-19 tests in the UCI Health pathology laboratories headed by SCRC member Dr. Edwin Monuki, Chair of UCI Pathology.

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Center faculty have obtained more than $170 million dollars in grant and foundation funds. Our research programs include an National Institutes of Health (NIH)-sponsored T32 training grant, state of the art shared equipment, research services programs and a seminar series.

How is the center funded?

Research at the Center is funded primarily by the California of Regenerative Medicine (CIRM), the National Institutes of Health (NIH) and philanthropic support from private donors. The administration of the center is supported by the university

Why is private support so important?

Philanthropic support is a vital to our success. Obtaining funding from major funding agencies like the National Institutes of Health can take years.  If we have a new idea, donor funds allow us to begin research right away. For patients suffering from diseases like Alzheimer’s, cancer, Huntington’s disease, heart disease or Parkinson’s disease and many other diseases, every day counts.

Why donate to the Center?

Stem cell research will transform the way that medicine is practiced and forever change the way that human diseases and injuries are treated. Few of us have the opportunity to truly impact the world we live in. Stem cell research promises to do just that. This is our opportunity to impact our world and we need to seize this moment. Please join the cause. Your support, whether it be by action or by financial support, can help the lives of people suffering from a wide variety of disease from Alzheimer’s to cancer, diabetes to heart disease, Parkinson’s disease to stroke.

What will my donation support?

All of your donation will be used to support research. The administrative costs of the Center are supported by the university. Your support can speed up the pace of research or provide support for new ideas and leverage support from Federal or State agencies and Foundations. Effectively, your donations can allow us to obtain more funding for stem cell research.

Can I specify what I would like my donation to go to?

Yes, absolutely. If you have an interest in a specific disease or injury or are interested in supporting the research of a particular researcher, you can specify that your support be used for that purpose.