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New FDA-approved paediatric cancer therapies offered at US health system

Atrium-Health

When pediatric cancer recurs, patients need access to innovative treatments and technologies that increase survival. However, these treatments require specialized expertise and complex care teams to ensure efficacy and safety.

Atrium Health Levine Children’s in Charlotte, North Carolina, of the United States is bringing renewed hope to patients and families with two new FDA-approved therapies: DANYELZA and chimeric antigen receptor (CAR) T-cell therapy.

Javier Oesterheld, MD, division chief of Levine Children’s Cancer and Blood Disorders, and Giselle Sholler, MD, pediatric oncologist and director of the Isabella Santos Foundation Solid and Rare Tumor Program at Levine Children’s, worked together to bring these therapies to their patients.

“We were able to implement these new treatment programs so quickly because we have a very robust team that works well together in support of our patients,” explained Dr. Oesterheld.

DANYELZA
DANYELZA (naxitamab-gqgk) is a drug used to treat patients who have experienced multiple relapses from neuroblastoma in the bone or bone marrow – and Levine Children’s is one of the first sites in the U.S. to offer it.

Each infusion takes an hour and requires close patient observation to monitor for signs of low or high blood pressure. Patients are treated weekly for at least six cycles with the goal of getting them back into remission. Phase II clinical trials revealed an overall response rate of 34% to 45% for partial or complete remission.

Dr. Oesterheld noted serious adverse reactions were reported in 32% of patients
treated with the infusions. “That’s why it requires a complex care team to support
patients both physically and emotionally during treatment,” he said.

For additional support, patients also have access to services from the Atrium
Health Levine Children’s Supportive Oncology program, including child life services, art therapy, chaplain services and Reiki therapy.

CAR T-cell therapy
Levine Children’s is the first in their region to offer CAR T-cell therapy, a type of immunotherapy used to treat relapsed B-cell acute lymphoblastic leukaemia in paediatric patients.

In CAR T-cell therapy, an apheresis machine collects and separates a patient’s blood cells. The T-cells are modified with a special receptor called chimeric antigen receptor (CAR), then given to the patient by infusion to attack cancer cells. “CAR T-cell therapy is considered the frontline therapy for relapsed B-cell acute lymphoblastic leukaemia, following bone marrow transplant,” said Dr. Oesterheld. “Most clinical trial patients emerged with no detectable cancer. However, redosing may be required to increase survival.”

According to Dr. Oesterheld, this is just the beginning of robust cellular therapy at Levine Children’s. “We plan to conduct research that will allow us to administer cellular and immunotherapy for brain tumours, solid tumours and other conditions,” he said. “Our cellular therapy program currently includes gene therapy for sickle cell anaemia. We’re also developing a new vaccine protocol for treating neuroblastoma and brain tumours.”

A world of care
Atrium Health Levine Children’s is headquarters of the Beat Childhood Cancer Research Consortium, a group of 40+ children’s hospitals that connects paediatric cancer patients to clinical trials from around the world. Though the North Carolina team works hard to provide world-class cancer care close to home, patients worldwide have access to Levine Children’s care through Atrium Health Global Healthcare Services. In addition to remote second opinions, Global Healthcare Services coordinates housing, food, transportation, concierge
services and insurance approvals.

“All the family has to focus on is caring for their child during their visit,” explained Dr. Oesterheld.

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