Three newly assembled disease teams within USC Stem Cell will take the early steps this year that might lead to future stem-cell based therapies for certain forms of deafness, bone defects and pediatric leukemia.
The teams are winners of USC’s Regenerative Medicine Initiative (RMI), a university-wide program kick-started by $1.2 million in funding from Carmen A. Puliafito, dean of the Keck School of Medicine of USC. Each RMI Award provides up to $200,000 per year for two years to multi-investigator research collaborations that harness the full potential of USC-affiliated faculty members.
The three winning teams were selected from 26 proposals involving 80 faculty members from Children’s Hospital Los Angeles (CHLA); the USC Viterbi School of Engineering; the USC Dornsife College of Letters, Arts and Sciences; the House Ear Institute; the USC School of Pharmacy; the Ostrow School of Dentistry of USC; and the Keck School.
One winning team aims at eventually curing deafness by using cellular reprogramming to create inner ear cells, including sensory hair cells. The death of these delicate structures, which humans and other mammals cannot regenerate, is the most common cause of deafness.
For this project, Neil Segil and Takahiro Ohyama — two researchers from the House Research Institute with appointments in USC’s Department of Cell and Neurobiology — will work with Justin Ichida, assistant professor of stem cell biology and regenerative medicine.
“The availability of these funds has stimulated a collaboration between investigators with different kinds of expertise and who have not previously worked together,” Segil said. “This kind of cross-fertilization is very stimulating, and we are all looking forward to the close collaboration that this project will require.”
Another winning team will focus on potential ways to repair human bones through lessons learned from two not-so-distant relatives: mice and zebrafish. When mouse ribs or zebrafish jaws are damaged, special cartilage cells transform themselves into bone-producing cells called osteoblasts. The research team hopes to show that these cells can more effectively heal severe bone damage that is a challenge to current therapeutic approaches.
The project brings together Jay Lieberman, a clinical orthopaedic surgeon and chair of the Department of Orthopaedic Surgery, and Gage Crump and Francesca Mariani, two researchers within the Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research.
“Both Francesca Mariani and myself are by training developmental biologists, whereas Jay Lieberman is an orthopaedic researcher who heads a surgical clinic,” Crump said. “So we’re hoping that we can bring a developmental mindset to the problem of repair so that we can make new headway into the problem.”
Another winning team will try to develop therapy for the most common form of cancer in children and adolescents, which is called B-precursor acute lymphoblastic leukemia. Some children with the disease have a genetic defect that results in the production of an abnormal form of the protein CD22E12, which causes their cancer stem cells to proliferate and resist chemotherapy. The team will leverage the insights gained from their studies of this abnormal protein to design new and more effective treatment strategies.
Fatih Uckun, professor in the Department of Pediatrics at the Keck School who heads the translational research in leukemia lymphoma at the Children’s Center for Cancer and Blood Diseases of CHLA, and Gregor Adams, assistant professor of cell and neurobiology, are co-principal investigators on the project. They’ll collaborate with Frank Alber, associate professor of biological sciences at USC Dornsife, and colleagues from other campuses.
“I don’t really consider this as an award but as an opportunity to work together with my colleagues,” Uckun said. “Only by working as a team can we accomplish the goal of helping these children who are in urgent need of a new treatment.”
Even in their early stages, these collaborative, multidisciplinary projects share an ambitious goal: to move basic research toward clinical applications.
“We hope these are the first of many future awards as we continue to develop new strategies to enable and empower researchers in regenerative medicine across USC,” said Provost Professor Andrew McMahon, who is spearheading USC Stem Cell and the RMI awards.