CIRM-funded USC+CHLA Alpha Clinic boosts capacity for clinical and translational research, community outreach, training and manufacturing
By Wayne Lewis
The California Institute for Regenerative Medicine (CIRM), the state’s stem cell agency, has awarded a five-year, $8 million grant to the Keck School of Medicine of USC and Children’s Hospital Los Angeles (CHLA). The USC+CHLA Alpha Clinic will expand personnel dedicated to advancing clinical trials of new cell and gene therapies, engaging underserved communities in such studies, and training the workforce that conducts them. It will also create opportunities to collaborate and exchange expertise with a network of CIRM-funded Alpha Clinics at eight other top biomedical research institutions statewide. The new program will advance CIRM’s goal of getting new cell and gene therapies to all Californians.
This new CIRM-supported program capitalizes on distinctive strengths and capabilities at the Keck School of Medicine and CHLA. The institutions have cultivated a large slate of cell and gene therapy research projects ranging from basic studies to investigational treatments poised to enter patient testing, and a track record of success engaging people from local underserved communities in research to improve their health.
“There’s a bridge of trust with our neighbors thanks to the outstanding work of our colleagues who lead community outreach,” said Tom Buchanan, MD, program director of the USC+CHLA Alpha Clinic, holder of USC’s Bernard J. Hanley Chair in Medicine and vice dean of research at the Keck School of Medicine. “We have a strong, growing portfolio of potential cell and gene therapies. The Alpha Clinic program will help us move that portfolio to approved treatments that will be available to Californians from all backgrounds.”
Approaches under development at USC and CHLA address conditions including cancer, vision loss, arthritis, fractures, burns, Alzheimer’s disease and a variety of pediatric diseases. Other investigations foster technologies for healing or replacing tissue in systems such as the skin, bone, cartilage, heart, gut and kidneys. These efforts benefit from preclinical research led by the Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research (USC Stem Cell) and other centers at USC and CHLA.
CHLA contributes special proficiency with cell therapies exemplified by its researchers’ role in the clinical development of CAR T cell therapy for pediatric leukemia. This breakthrough strategy re-engineers patients’ own immune cells so they fight blood cancers.
“We aim to translate discoveries into new therapies so that children — and individuals of all ages — can enjoy better health,” said Alan Wayne, MD, director of the Cancer and Blood Disease Institute at CHLA. “The USC+CHLA Alpha Clinic will dramatically speed progress in cell and gene therapy for real-world impact, right here in Los Angeles.” Wayne serves as site principal investigator for the CIRM grant at CHLA and associate director for pediatric oncology at USC Norris Comprehensive Cancer Center.
The USC+CHLA Alpha Clinic complements the activities of the Southern California Clinical and Translational Sciences Institute (SC CTSI), a collaboration led by Buchanan that unites the Keck School of Medicine, CHLA and the L.A. County Department of Health Services. SC CTSI offers a range of resources to move biomedical advances forward to clinical trials enrolling people from the city’s diverse communities. The USC+CHLA Alpha Clinic will also leverage the expertise and resources of USC Norris cancer center, which spans USC and CHLA.
Another asset connected to the USC+CHLA Alpha Clinic is the recently established USC+CHLA Cell Therapy Program, including a newly constructed facility for manufacturing cell and gene therapies under the Food and Drug Administration’s current good manufacturing practice (cGMP) standards. Launched through a partnership among the Keck School of Medicine, CHLA, Keck Medicine of USC and USC Norris cancer center, the program offers client-centric partnership to enable robust and cost-efficient product development to maximize successful translation of these innovative products for use in clinical care.
“The idea is to provide the complete support structure needed for investigators to translate scientific findings into clinical applications that benefit patients,” said Mohamed Abou-el-Enein, MD, PhD, MSPH, the USC+CHLA Alpha Clinic’s associate director, inaugural director of the USC+CHLA Cell Therapy Program, and director of the cGMP laboratory. “The cell therapy field is very dynamic, and with the current energy and momentum, I see a lot of opportunities for us to create cures for previously incurable diseases.”
The USC+CHLA Alpha Clinic will also help develop the workforce that runs clinical trials of cell and gene therapies. Courses will impart specialized skills in order to ensure safety and compliance with FDA requirements. The trainings will build upon modules from the Cell Therapy Program and SC CTSI, as well as expertise from the USC School of Pharmacy’s Department of Regulatory and Quality Sciences.
Participation of the USC+CHLA Alpha Clinic in the statewide CIRM Alpha Clinics Network will be coordinated by Juliane Glaeser, PhD, director of translational and collaborative research programs for USC Stem Cell. The Alpha Clinics Network creates new opportunities for USC and CHLA researchers to collaborate with peers at Cedars-Sinai Medical Center, City of Hope, Stanford University, UC Davis, UC Irvine, UCLA, UC San Diego and UC San Francisco. This will lead to new multi-institutional clinical trials of cell and gene therapies across California. Sites also commit to sharing knowledge in areas of strength. The USC+CHLA Alpha Clinic expects to contribute expertise in community engagement, the regulatory logistics of clinical trials, cell manufacturing and disease areas of specialized focus.
Buchanan extols the promise of the types of projects the USC+CHLA Alpha Clinic will advance.
“These cell- and gene-based strategies take advantage of really complex machinery that Mother Nature has developed over a billion and a half years of evolution,” he said. “They could change the way we practice medicine.”