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Rady Children’s Now Providing Pioneering Therapy That Re-engineers Patients’ Blood Cells to Attack Cancer

Kymriah® holds promise for treating refractory or relapsed B-cell acute lymphoblastic leukemia in children and young adults

SAN DIEGO – Friday, June 22, 2018 – Rady Children’s Hospital-San Diego is now one of a limited number of institutions across the United States certified to treat patients with the Novartis-manufactured immunotherapy Kymriah®.  The drug is intended for patients up to age 25 diagnosed with refractory or relapsing B-cell acute lymphoblastic leukemia (B-ALL), the most common form of pediatric cancer.

Immunotherapy is a rapidly evolving approach to cancer treatment that harnesses and augments a patient’s own immune system to treat disease. Kymriah, a type of immunotherapy called chimeric antigen receptor T-cell (CAR-T) therapy, genetically alters patients’ blood cells to attack cancer cells.

“In early multicenter clinical trials, 83 percent of patients were able to achieve remission within three months of Kymriah infusion,” says Deborah Schiff, M.D., pediatric hematologist-oncologist at Rady Children’s who oversees the Hospital’s Kymriah initiative. “We’re fortunate to have this opportunity to provide this potentially life-saving therapy for patients with a previously poor prognosis.”

Kymriah is the very first gene-modifying cancer therapy to be approved by the Food and Drug Administration (FDA), and certification for use in patients is rigorous. To meet requirements, staff in Rady Children’s Hematology/Oncology, Emergency Medicine, Pediatric Intensive Care and Pharmacy divisions participated in FDA-mandated training.

Medical centers treating patients with Kymriah are also required to have tocilizumab, which treats a potential side effect of CAR-T therapy, stocked before patients begin infusions.

“Because the revved up CAR-T cells can trigger an intense reaction called a cytokine release syndrome that can cause high fever, low blood pressure, respiratory distress, neurological symptoms and other life-threatening complications, “ explains Dr. Schiff, “medical staff members must be trained to manage these reactions.”

In an additional training step, the Rady Children’s partnered with the stem cell processing lab at the UC San Diego Center for Advanced Laboratory Medicine to complete mock product shipping and FDA audit scenarios.

For more information about CAR-T therapy, visit

About Rady Children’s Hospital-San Diego:

Rady Children’s Hospital-San Diego is a 551-bed pediatric care facility providing the largest source of comprehensive pediatric medical services in San Diego, southern Riverside and Imperial counties. Rady Children’s is the only hospital in the San Diego area dedicated exclusively to pediatric healthcare and is the region’s only designated pediatric trauma center. In June 2017, U.S. News & World Report ranked Rady Children’s among the best children’s hospitals in the nation in all ten pediatric specialties the magazine surveyed. Rady Children’s is a nonprofit organization that relies on donations to support its mission. For more information, visit and find us on FacebookTwitter and Vimeo.

Media Contact:
Ben Metcalf
(858) 966-8579(o) (619) 822-8593 (c)