UCI Health stem cell transplant program poised for significant growth

UCI Health stem cell transplant program poised for significant growth

Posting its 200th transplant, Orange County's only adult program is taking off 'like a rocket' to deliver novel cellular therapies.

Orange, Calif. - The UCI Health bone marrow transplant program has reached a major milestone, recently performing its 200^th lifesaving procedure since it opened in May 2020.

It comes as the fast-growing program at the Chao Family Comprehensive Cancer Center is expanding access to cellular therapies, including chimeric antigen receptor (CAR) T cells, which offer hope to patients with some of the most difficult-to-treat blood cancers and solid tumors.

As Orange County's only adult bone marrow service, it also is one of the few U.S. programs to transplant hematopoietic stem cells in patients with multiple sclerosis and other autoimmune conditions. Patients with certain solid tumors, including testicular cancer, also have undergone transplants at UCI Medical Center, part of UCI Health.

"This program has taken off like a rocket," said Dr. Richard A. Van Etten, director of the Chao Family Comprehensive Cancer Center and a member of the transplant team.

In less than two years, Van Etten noted, the program accomplished an exceptional feat, accreditation for achieving the national standard of excellence for cellular therapy programs. It also is the only program in Orange County approved by CalOptima Health, the county's healthcare insurance plan for low-income adults, children and people with disabilities.

"We opened this program to provide our patients with much-needed access to these treatments close to home," he said. "Now we are seeing patients coming from all over the region and the country."

200+ transplants

To date, the Hematopoietic Stem Cell Transplant and Cellular Therapy Program has performed more than 200 blood and bone marrow-derived stem cell transplants, including 13 procedures for autoimmune diseases. These include autologous transplants using the patient's own cells and allogeneic procedures using matched, mismatched and half-matched (haploidentical) donor cells.

"Two hundred-plus patients is quite remarkable considering that most new programs initially offer only autologous transplants in their first several years," said Dr. Stefan O. Ciurea, a UCI Health hematologic oncologist and professor at the UC Irvine School of Medicine. He was recruited from the renowned MD Anderson Cancer Center in Houston to lead the program.

"In just four years, we have become a mature transplant and cell therapy service, performing all types of procedures associated with established programs," said Ciurea, a national leader in haploidentical transplantation, which make up the majority of the program's allogeneic transplants.

More treatment options

The team also is performing a growing number of FDA-approved CAR T-cell treatments, in which a patient's own immune cells are genetically modified in a laboratory, then returned to the patient to attack their cancer cells.

In addition, the team is enrolling numerous patients in highly promising early phase clinical trials of investigational CAR T and other cellular therapies, including several that have emerged from UCI scientists' discoveries. Other trials focused on improving transplant outcomes and success rates are underway, with more expected to open in coming months.

"We have very exciting studies, some unique to our cancer center, and others offered at only a few other academic medical centers in the country," Ciurea said.

Challenging procedure

Transplantation of hematopoietic stem cells - immature cells found in bone marrow and blood - can prolong the survival of and cure many patients with high-risk blood cancers, including leukemia, lymphoma and myeloma. A complex and challenging procedure, it requires considerable expertise and specialized facilities.

Treatment generally starts with the patient getting high doses of chemotherapy, often with radiation, to eliminate any remaining cancer cells. This usually eliminates the patient's bone marrow stem cells. New stem cells, either taken from the patient or a tissue-matched donor, are then infused to settle in the bone marrow and make healthy new blood cells.

Post-transplant, patients typically are hospitalized for several weeks to rebuild their blood and immune systems. Their progress and any side effects are carefully monitored in numerous follow-up visits.

The UCI Health program has an experienced transplant team of physicians. nurses, transfusion specialists, pharmacists and other support personnel. They also have a training program for physicians interested in stem cell transplantation and cellular therapies.

Preparing for growth

To accommodate the burgeoning CAR T and transplant patient population, Ciurea will add at least two fellowship-trained specialists to the team, as well as staff to operate a new processing laboratory.

The Cellular Processing Lab at the medical center in Orange is designed to prepare cell products for patient use, including hematopoietic stem cells and immune cells, such as CAR T, for both FDA-approved commercial therapies and investigational cancer clinical trials at UCI Health.

The investigator-initiated trials also are supported by a state-of-the-art regenerative medicine manufacturing center on the university campus. The Good Manufacturing Practice facility, a suite of ultraclean lab and quality-control rooms, was built to create FDA-approved next-generation gene and cellular products for patient treatment and clinical research.

'Very good survival numbers'

Ciurea is especially proud of the program's outstanding results and very low error rate of 0.8% as reported to the Center for International Blood & Marrow Transplant Research (CIBMTR) compared with its benchmark of 3%. CIBMTR, a research collaboration between the National Marrow Donor Program and the Medical College of Wisconsin, collects data on all hematopoietic cell transplant procedures.

"We have very good survival numbers and our confidence is high that this will translate to superior transplant outcomes."

He also is eager for this month's opening of the Chao Family Comprehensive Cancer Center - Irvine and its large infusion facilities, which will greatly expand access for outpatient transplant procedures and cellular therapies. It also permits better continuity of care closer to home for patients living in coastal and south Orange County.

"For folks who are receiving intensive treatments, being close to home is very important," he said.

Looking to the future

When the UCI Health - Irvine medical complex opens a 144-bed acute care hospital in 2025, it will become the transplant program's new home. There, a more advanced processing lab will offer more opportunities to investigate novel cellular therapies.

Ciurea credits the program's sound footing and rapid growth to the "extraordinary institutional support" from Van Etten, Dr. Michael J. Stamos, dean of UC Irvine School of Medicine, and Chad Lefteris, president and CEO of UCI Health.

"We also have an exceptionally dedicated team, without which none of this would have been possible," he said.

"I could never really have imagined being where we are today. We will continue to expand to serve the many patients who need transplant and cellular therapy in Orange County and beyond. And we will continued to develop more innovative approaches to improve treatment outcomes for our patients."

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About UCI Health

UCI Health, one of California's largest academic health systems, is the clinical enterprise of the University of California, Irvine. The system comprises its main campus UCI Medical Center, a 459-bed, acute care hospital in in Orange, Calif., four hospitals and affiliated physicians of the UCI Health Community Network in Orange and Los Angeles counties and ambulatory care centers across the region. Recognized as a Top Hospital by The Leapfrog Group, UCI Medical Center provides tertiary and quaternary care and is home to Orange County's only National Cancer Institute-designated comprehensive cancer center, high-risk perinatal/neonatal program and American College of Surgeons-verified Level I adult and Level II pediatric trauma center, gold level 1 geriatric emergency department and regional burn center. UCI Health serves a region of nearly 4 million people in Orange County, western Riverside County and southeast Los Angeles County. Follow us on Facebook, Instagram, LinkedIn and Twitter.

About UCI Health - Irvine

UCI Health - Irvine, a new medical complex under construction at the north end of the UC Irvine campus, will bring unparalleled expertise and the finest evidence-based care that only an academic health system can offer to the communities of coastal and south Orange County. As part of UCI Health - which includes the flagship UCI Medical Center in Orange, Orange County's only National Cancer Institute-designated comprehensive cancer center and multiple outpatient care locations - the new 1.2 million-square-foot campus will offer key clinical programs in oncology, digestive health, neurology, neurosurgery, orthopedics and spine surgery. The nation's first all-electric, carbon-neutral medical center, UCI Health - Irvine is home to the Joe C. Wen & Family Center for Advanced Care, a five-story, 168,000-square-foot medical facility offering the full range of multidisciplinary specialty care for children and adults under a single roof, urgent care services, the Center for Children's Health and the UCI Health Center for Autism & Neurodevelopmental Disorders. Opening this summer: The Chao Family Comprehensive Cancer Center and Ambulatory Care, a 225,000-square-foot facility. Coming in 2025: A seven-story, 350,000-square-foot, acute care hospital.