Nationwide Children’s Hospital Becomes Certified Site for CAR-T Cell Therapy

Kymriah available for pediatric and young adult patients with B-cell acute lymphoblastic leukemia

July 20, 2018

COLUMBUS, Ohio – Nationwide Children’s Hospital has been designated a certified treatment center for the FDA-approved Novartis CAR-T cell therapy Kymriah™ (tisagenlecleucel) and can now offer the therapy to patients.

In August 2017, the U.S. Food and Drug Administration (FDA) approved Kymriah, which is manufactured by pharmaceutical company Novartis, for the treatment of patients up to 25 years of age who suffer from refractory or relapsed B-cell acute lymphoblastic leukemia (ALL). It is the first gene transfer therapy approved by the FDA.

With the FDA and Novartis’ designation, Nationwide Children’s becomes one of 33 pediatric facilities throughout the U.S. that is certified to have the advanced facilities and specially-trained staff necessary for the administration of Kymriah.

“This is a potentially life-saving treatment for the many patients who suffer from B-cell acute lymphoblastic leukemia,” said Timothy Cripe, MD, PhD, division chief of Hematology, Oncology, and Bone Marrow Transplantation at Nationwide Children’s and a faculty member at The Ohio State University College of Medicine. “Prior to this novel therapy, prognosis for relapsed patients was poor, despite rounds of chemotherapy and radiation. Now, we have a novel, less invasive treatment option for families, and equally as important, we have the opportunity to provide families with hope,” continued Dr. Cripe, also a member of the FDA advisory panel that recommended the approval of Kymriah.

CAR-T cell therapy is a type of immunocellular therapy in which a patient’s T cells are altered to attack cancerous cells. After T cells are removed from the patient, the gene of a receptor, known as chimeric antigen receptor (CAR), is added to the T cells in a laboratory. When the T cells are infused back into the patient, the chimeric antigen receptor on the T cells binds to the patient’s cancer cells, effectively using the patient’s immune system to destroy the cancer cells.

“The one-time treatment with Kymriah is the type of personalized and targeted therapy that clinicians and research scientists have been working on for decades for the betterment of their patients,” said Dean Lee, MD, PhD, director of the joint Cellular Therapy and Cancer Immunotherapy Program at Nationwide Children’s and The Ohio State Comprehensive Cancer Center. “In addition, this pioneering immunotherapy has set the groundwork for applying this kind of approach to many other types of cancers.”

In the first global pediatric CAR-T open-label, multicenter, single arm clinical trial sponsored by Novartis, 68 patients were infused with Kymriah with 63 evaluated for efficacy. Eighty-three percent (52 of 63) of patients who received Kymriah attained complete remission or complete remission with incomplete blood count recovery within three months of infusion. After one year, a majority of patients who responded to treatment still had no cancer detectable in in their blood.1

About Hematology/Oncology/Bone Marrow Transplantation at Nationwide Children’s Hospital
Each year, more than 150 patients are diagnosed with cancer at Nationwide Children’s Hospital. Annually, inpatient admissions and outpatient visits exceed 13,000 total. We offer world-class diagnostic, treatment and research facilities that provide comprehensive, multi-disciplinary care for patients with cancer and acute and chronic blood disorders. Ranked No. 5 on the U.S. News & World Report’s 2018-19 Best Children’s Hospitals specialty rankings, Hematology/Oncology & BMT at Nationwide Children’s is a member of the Children’s Oncology Group, a National Marrow Donor Program Center, and is the largest facility of its kind in Ohio and one of the largest in the United States.

About Nationwide Children’s Hospital
Named to the Top 10 Honor Roll on U.S. News & World Report ‘s 2018-19 list of “Best Children’s Hospitals,” Nationwide Children’s Hospital is one of America’s largest not-for-profit freestanding pediatric health care systems providing wellness, preventive, diagnostic, treatment and rehabilitative care for infants, children and adolescents, as well as adult patients with congenital disease. Nationwide Children’s has a staff of more than 13,000 providing state-of-the-art pediatric care during more than 1.4 million patient visits annually. As home to the Department of Pediatrics of The Ohio State University College of Medicine, Nationwide Children’s physicians train the next generation of pediatricians and pediatric specialists. The Research Institute at Nationwide Children’s Hospital is one of the Top 10 National Institutes of Health-funded freestanding pediatric research facilities. More information is available at NationwideChildrens.org.

Reference
1Kymriah (tisagenlecleucel) Prescribing information. East Hanover, New Jersey, USA: Novartis Pharmaceuticals Corporation; August 2017.

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Cancer
Hematology
Hematology, Oncology & BMT

About Nationwide Children's Hospital

Named to the Top 10 Honor Roll on U.S. News & World Report ‘s 2018-19 list of “Best Children’s Hospitals,” Nationwide Children’s Hospital is one of America’s largest not-for-profit freestanding pediatric health care systems providing wellness, preventive, diagnostic, treatment and rehabilitative care for infants, children and adolescents, as well as adult patients with congenital disease. Nationwide Children’s has a staff of more than 13,000 providing state-of-the-art pediatric care during more than 1.4 million patient visits annually. As home to the Department of Pediatrics of The Ohio State University College of Medicine, Nationwide Children’s physicians train the next generation of pediatricians and pediatric specialists. The Research Institute at Nationwide Children’s Hospital is one of the Top 10 National Institutes of Health-funded freestanding pediatric research facilities. More information is available at NationwideChildrens.org.