Fred Gage

Fred H. Gage from the Salk Institute for Biological Sciences and the University of California, San Diego, delivered a brainy keynote address. (Photo by Cristy Lytal)

Provost Michael Quick convened the inaugural USC Stem Cell Symposium with a straightforward truth about the future of regenerative medicine: “it will take a dedicated community of scholars across the disciplines to have maximum impact.”

The January 16 symposium brought together precisely such a community, with speakers hailing from USC’s schools of medicine, dentistry, gerontology and engineering and from The Saban Research Institute of Children’s Hospital Los Angeles (CHLA). Held at Aresty Auditorium at the Keck School of Medicine of USC, the event was hosted by USC Stem Cell (, a collaborative and multidisciplinary effort bringing together more than 100 researchers and clinicians working to translate discoveries into cures.

Andy McMahon, chair of the USC Stem Cell executive committee, shared his vision of engaging even more scholars from beyond the scientific realm: “An important part of the future is how USC Stem Cell is going to engage other schools — in business, in public policy, in law — as this area of medicine becomes increasingly more involved. So I’m looking forward to a future that embraces many more schools than are currently represented.”

McMahon also unveiled two new opportunities for young scientists to engage in collaborative and creative research projects. The new USC Stem Cell Hearst Fellowship will support exceptional junior postdoctoral fellows pursuing stem cell research at USC. The USC Stem Cell Student/Postdoc Collaborative Challenge Grant Program will provide $10,000 to one-year research projects bringing together students or postdocs in two or more labs.

“Particularly, we’re interested in interdisciplinary projects that bring people together across different areas of research that tend to have boundaries,” said McMahon. “The idea is to stimulate new interdisciplinary research and with this, to enhance the student’s or postdoc’s ability to be a creative and independent scientist.”

The USC Stem Cell Regenerative Medicine Initiative (RMI) Awards have enabled additional multi-investigator research collaborations among USC-affiliated faculty members. The three winning teams presented their progress in developing stem cell-based strategies to treat certain forms of deafness, bone defects and pediatric leukemia.

One of the day’s highlights was the keynote address by Fred H. Gage, the Vi and John Adler Chair for Research on Age-Related Neurodegenerative Disease at the Salk Institute for Biological Sciences and the University of California, San Diego. Gage introduced the concept of “mobile elements,” genetic material that that can move from one part of the genome to another. When these mobile elements insert into neural genes, they may potentially alter behavior, enhance genomic diversity and even speed evolution.

Several USC Stem Cell principal investigators also shared their most recent research:

  • David Warburton from CHLA and the Keck School addressed lung development and disease, which can have both genetic and environmental causes, such as smoking or pollution. He also shared his work on infant mortality in Ulaanbaatar, Mongolia, which consistently tops the list of cities with the worst air pollution in the world.
  • Paula Cannon from the Keck School talked about genetically modifying blood stem cells to cure HIV/AIDS. Her approach is inspired by the “Berlin patient,” a man cured of both HIV and leukemia through a bone marrow transplant from a donor with a gene mutation that confers natural immunity to HIV. The first human clinical trials will take place in collaboration with the City of Hope and Sangamo BioSciences.
  • Rong Lu from USC’s stem cell research center discussed “barcoding” individual blood stem cells by labeling them with a genetic marker. This allowed her to observe individual cells’ contributions to forming blood, which could determine strategies for more effective blood transfusions or bone marrow transplants.
  • Tracy Grikscheit — from Children’s Hospital Los Angeles (CHLA), the Keck School and the USC Viterbi School of Engineering — explained her strategy for “using science to cure babies” with injured or diseased intestine. Her approach is to take discarded human intestine from the operating room, break it up, and put it on a felt-like biodegradable tube in a special machine, to regrow into new intestine. Eventually, surgeons would reconnect this newly formed intestine to the patient’s digestive tract.
  • Senta Georgia from CHLA and the Keck School has different plans for patients’ intestines: inducing intestinal stem cells to make insulin by “turning on” key genes. The goal is to use these insulin-producing intestinal cells to treat diabetes.
  • Valter Longo from the USC Davis School of Gerontology introduced his research on fasting, which inhibits the genes responsible for both aging and overall growth in response to protein. Clinical trials are currently underway at the USC Norris Comprehensive Cancer Center, Mayo Clinic and elsewhere to explore whether fasting can improve outcomes in patients receiving chemotherapy for lymphoma as well as breast, prostate and colorectal cancers. He’s also conducting a clinical trial exploring whether a five-day fast can stimulate stem cell-based regeneration of multiple organ systems.
  • Min Yu — from USC’s stem cell research center and the USC Norris Comprehensive Cancer Center — recently isolated breast cancer cells circulating through patients’ blood streams and kept these cells alive in petri dishes. She is using these cells to identify mutated cancer strains and the right drugs to target them — ushering in personalized treatments for breast cancer patients.
  • Alan S. Wayne from CHLA and the Keck School talked about immunotherapy for patients with the most common form of childhood cancer, acute lymphoblastic leukemia, or ALL. In a recent clinical trial, his team trained patients’ immune cells to recognize and attack leukemia, and two-thirds of the children experienced complete remission.
  • Michael Bonaguidi from USC’s stem cell research center described the potential of neural stem cells within the adult brain. These stem cells can generate more stem cells or form new neurons and supporting cells, offering a possible avenue for treating symptoms associated with Alzheimer’s disease and other brain dysfunctions.
  • Justin Ichida from USC’s stem research cell center shared his progress in finding treatments for another fatal neurodegenerative disease: amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease. His lab is testing FDA-approved drugs on neurons formed by reprogramming skin cells from patients with ALS. Although these patient-derived neurons display the disease’s signature degeneration, the Ichida lab has already found FDA-approved drugs that keep the neurons alive in the petri dishes — and might do the same in patients.
  • Ruchi Bajpai — from the Ostrow School of Dentistry of USC and the Keck School — addressed why neural tube and craniofacial birth defects occur together in a congenital disorder known as CHARGE syndrome. She studies the genetics underlying this disease by differentiating stem cells into the so-called “neural crest cells” that form many parts of the brain and face.
  • David Cobrinik from CHLA and the Keck School discussed the formation of a childhood eye tumor called retinoblastoma, which he studies by creating retinal tissue from human stem cells. This research elucidates how the mutation of a tumor-suppressing gene called RB1 can lead to the formation of retinoblastoma.
  • Megan McCain from USC Viterbi and the Keck School introduced the concept of a “heart on a chip.” To overcome the limitations of using laboratory animals or human cells in petri dishes to study human disease, McCain’s laboratory engineers dynamic micro-scale mimics of human heart tissue.
  • Cheng-Ming Chuong from the Keck School addressed two related topics: how feather stem cells enabled the evolution of birds, and how hair stem cells might enable the “extinction” of male pattern baldness. His research has demonstrated that by plucking 200 hairs in a specific pattern and density, he can induce 1,000 replacement hairs to grow.
  • Yang Chai — director of the Center for Craniofacial Molecular Biology at the Ostrow School — explored stem cells that can maintain tooth growth and a possible way to help babies with fused skulls that can’t grow normally. Chai’s lab has found that the skull joints, known as sutures, contain a special population of stem cells that can help maintain and repair the skull.

At the end of the day, USC’s stem cell scientists left the symposium informed and inspired to translate discoveries into cures.

“It’s fantastic to see the diversity of different research that’s going on in the schools across USC,” said McMahon. “I very much look forward to meeting again next year.”

by Cristy Lytal