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Novartis' CAR-T Therapy for Pediatric Leukemia to Face FDA Advisory Panel

Alison Rodriguez
Novartisí chimeric antigen receptor T-cell (CAR-T) therapy for treating pediatric leukemia is on the cusp of being the first FDA-approved gene therapy, which will lead to new developments and utilizations of CAR-T therapy for treating other advanced blood cancers.
Novartis’ chimeric antigen receptor T-cell (CAR-T) therapy for treating pediatric leukemia is on the cusp of being the first FDA-approved gene therapy, which will lead to new developments and utilizations of CAR-T therapy for treating other advanced blood cancers.

Customizable CAR-T therapies are developed through harvesting a patients’ white blood cells, which are then genetically modified to target and attack cancer cells. Novartis’ therapy, tisagenlecleucel or CTL019, is specifically intended for treating children and young adults (3 to 25 years old) with relapsed or refractory B-cell acute lymphoblastic leukemia. CTL019 was originally developed at the University of Pennsylvania, and in 2012, the University partnered with Novartis in a global collaboration to continue research with efforts to advance and commercialize CAR-T therapies.

The ELIANA study demonstrated the efficacy of CTL019 and initiated the potential FDA approval. In the phase 2 study, 41 of 50 (82%) patients reached complete remission or complete remission with incomplete blood count recovery at 3 months following CTL019 treatment, according to Novartis. The phase 2 JULIET study was the second global CAR-T trial, following ELIANA, and focused on investigating the efficacy and safety of CTL019 in adult patients with relapsed or refractory diffuse large B-cell lymphoma.

“Even if a patient has difficult-to-treat relapsed/refractory leukemia, we have seen treatment with CTL019 in clinical trials put cancer into remission,” said Grupp, director of the Cancer Immunotherapy Frontier Program and director of Translational Research for the Center for Childhood Cancer Research at the Children’s Hospital of Philadelphia, in a press release following the study.

Results from the ELIANA and JULIET studies led the FDA to accept Novartis’ application for approval.

The FDA has few doubts regarding the efficacy of the therapy; however, the long-term effects are unknown, as seen in the FDA’s briefing documents from a treatment hearing. The FDA questions if the benefits of the treatment are worth the risk of the unknown and potentially harmful effects.

“The FDA seeks the opinion of the Committee regarding 1) post-marketing considerations for risk mitigation for short-term toxicities, particularly cytokine release syndrome, and 2) long-term follow-up for anticipated safety concerns related to the potential for insertional mutagenesis and secondary malignancies 3) whether the benefits justify the risks for a marketing approval of tisagenlecleucel for the proposed indication,” the FDA wrote.



 
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