Cancer Immunotherapy (CAR-T)

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Cure rates for childhood acute lymphoblastic leukemia (ALL) have improved dramatically, but there is a subset of patients for whom no traditional therapies are effective. A global pediatric clinical trial led by this Frontier program contributed to the development of the first personalized cellular therapy for the treatment of B-cell precursor ALL that is refractory or in second or later relapse. The pioneering approach is called chimeric antigen receptor (CAR) T-cell therapy, tisagenlecleucel, where a patient's own immune cells are genetically altered to seek and destroy the cancerous cells in his or her body.

The combined efforts of investigators from CHOP, the Perelman School of Medicine at the University of Pennsylvania, and Novartis Pharmaceuticals paved the way for the first form of CAR T-cell therapy to receive U.S. Food and Drug Administration (FDA) approval in August 2017. Children who have received the treatment continue to show high rates of complete remission.
The Frontier program is developing cellular therapy strategies to treat several other forms of cancer that occur in children, adolescents, and young adults, with the hope of expanding its use to fight other forms of leukemia, solid tumors, and non-malignant diseases.

Here are some highlights of the Cancer Immunotherapy program's research efforts:

  • Began using a type of cellular therapy known as CAR T-cell therapy as an investigational treatment option for children with relapsed or refractory ALL in 2012. The first child treated remains cancer-free almost six years later.
  • Led clinical trials in the first form of CAR T-cell therapy (now called Kymriah™) to be approved by the FDA for the treatment of relapsed and refractory ALL.
  • Has treated more children with ALL with CD19-redirected CAR T-cell therapy than any other hospital in the world.
  • Identified and applied treatment with tocilizumab (Actemra®) to decrease the severity of potentially fatal toxic side effects of T-cell therapy called cytokine release syndrome (CRS). This approach is now used worldwide for children and adults treated with CAR T-cells and other new immunotherapies who develop CRS and was approved by the FDA for this use in 2017.
  • We have the only open trial for "retreatment" of patients with relapsed or refractory CD19+ leukemia and lymphoma who were previously treated with cell therapy.

The Cancer Immunotherapy Program received Frontier status in Fiscal Year 2016.