UCSF's Pediatric Cell Therapy Lab Powers New Treatments for Chronic Illnesses

Cellular therapies have transformed how children with cancer and life-threatening immune disorders are treated. Now, that expertise is reaching new parts of pediatric medicine.  

These advanced therapies have the potential to change the course of a broad range of lifelong diseases that start in childhood. Bringing them into clinical care requires highly specialized infrastructure, and the UCSF Pediatric Cell Therapy Lab (PCTL) is making that possible across the Department of Pediatrics.  

A New Option for Chronic Disease 

Among the first to benefit are children with chronic rheumatic conditions like lupus and juvenile dermatomyositis. For these patients, the current standard of care means decades of immunosuppressive medication and the accumulating burden of both disease and treatment. 

"Our collaboration with the PCTL has given us the opportunity to explore new treatment approaches that may offer durable remission and bring us closer to a goal that has long seemed out of reach: a cure for these diseases," says Susan Kim, MD, MMSc, a pediatric rheumatologist at UCSF. 

The approach uses CAR-T cell therapy – the same class of engineered T cells originally developed for pediatric leukemia – redirected to target the B cells that drive autoimmune disease. Clinical trials in adults have produced remarkable remissions, and UCSF is now bringing these therapies to children, with early patients already being treated through clinical trials supported by the PCTL. 

The Lab Behind the Innovation 

The living cells used for these therapies can’t be dispensed through a pharmacy. They require specialized collection, processing, cryopreservation, and delivery, with strict regulatory oversight throughout. 

The PCTL is one of the few dedicated pediatric laboratories in the country accredited to prepare these highly specialized therapies, and is directed by Chris Dvorak, MD, chief of the Division of Pediatric Allergy, Immunology, and Bone Marrow Transplantation.   

"We're enabling divisions like rheumatology, gastroenterology, and endocrinology to pursue cellular therapies that simply weren't available to them before," says Dvorak. "Having a dedicated pediatric lab with the regulatory infrastructure already in place makes that possible." 

Each therapy comes with its own manufacturing and handling requirements, and the lab provides the expertise needed to safely integrate them into pediatric care. At many academic medical centers, that work happens in a shared facility where pediatric needs compete with larger adult programs. The lab's dedicated focus on children means these complex therapies are managed by a team built entirely around pediatric patients. 

A Record of Transplant Excellence 

The PCTL's role in chronic disease builds on more than two decades of expertise in highly specialized cell processing for pediatric bone marrow transplantation. The team excels at technical procedures, including preparing cells for haploidentical transplants from half-matched family donors. That expertise has helped establish UCSF as a leader in reducing transplant rejection rates in children. 

The same infrastructure has also enabled UCSF to lead in newer cellular therapies. The institution was the first center in California certified to deliver CAR-T cell therapy to children outside of a clinical trial, creating the foundation for today's expansion into new areas of pediatric medicine. 

Looking Ahead 

For Dvorak and his team, the rheumatology partnership is just the beginning. The PCTL provides a platform that any division in the Department of Pediatrics can leverage to develop and trial new cellular therapies.  

"It is rare that the field of rheumatology sees advances of this magnitude," says Emily von Scheven, MD, MAS, chief of the Division of Pediatric Rheumatology. "With these new treatments, we are hopeful that young patients will be able to achieve prolonged periods of drug-free remission, or even cure." 

Learn how the UCSF Division of Pediatric Allergy, Immunology, and Bone Marrow Transplantation is advancing pediatric cellular therapies.